The sNDA filing is based on P-III ADVANCE trial data, assessing Otezla (30 mg, bid) vs PBO in 595 patients in a ratio (1:1) with mild-to-moderate plaque psoriasis
The P-III ADVANCE study results: @16wks., improvement in its 1EPs of sPGA score; achieve 2EPs i.e, 75% improvement in BSA, change in PASI score
Otezla is a selective PDE4 inhibitor, approved therapy for mod. to sev. plaque psoriasis who are candidates for phototherapy or systemic therapy, for active PsA, and for with oral ulcers associated with Behçet’s Disease
Click here to read full press release/ article | Ref: PRNewswire | Image: Conejo Valley Guide
How is a Rare disease defined? Well, it depends on the geography in question, since there is no universal definition, even so, the definition revolves around the point of prevalence or incidence of rare disease. However, despite the changing attributes in the definition, there is one common ground every country faces‒challenges in mapping the patient pool, R&D, development of therapies, and investments & funding. Let us have a look at the unique challenges that Rare diseases pose that impact healthcare, regulatory agencies, but mainly patients.
Lack of knowledge.
The first and foremost challenge is the struggle in identifying and understanding the pathophysiology of the disease. With varying demographics, race, and ethnicity, symptoms and causes may vary, and it often gives rise to chaos and frustration. Rare disease patients are vastly spread and heterogeneous in disease subtypes, symptoms, stages, and exposure to prior treatment. The limited number of patients who have reported the disease limits the number of medical practitioners dealing with it. Data recorded is restricted, and it does not offer the chance to fully examine and analyze the disease.
Rare is frequent.
Rare diseases are individually rare, however, en masse, they are agreeably vast. Many timesit takes years of consultations, hospital visits, and tests for an accurate diagnosis of any rare disease. According to reported data, there are around 7000 identified rare diseases, with several being discovered all the time. Rare diseases are difficult to diagnose. Many have non-specific symptoms; while others have unusual symptoms. Many progress gradually and slowly within the body. There is no way to find out when the journey will end unless the prescribed therapies start to improve the condition.
Patients and their misery.
For patients, the path towards an appropriate diagnosis is bleaker than any figure represents. The challenges to patients with a rare disease are that they keep on struggling with the disease, manifesting the symptoms, and find a knowledgeable physician who would accurately diagnose and prescribe treatment for it. Cases of misdiagnosis and prescription of wrong treatment regimen are umpteen. Further, after diagnosis, the huge cost of the treatment as well as long-distance travel to get specific treatment drill holes in their pockets. In the fullness of time, every second of wait or search can proportionate to a missed opportunity to save a life.
Dilemma of physicians.
The challenges to physicians attending rare disease patients mirror the concerns faced by the patients. We discussed how late and misdiagnoses of rare disease is a common sight, and how it adds to the medical bills, physical stress, and mental burden for the patients and their families. However, physicians find it equally challenging and difficult while dealing with rare diseases. The exposure any physician is ought to get with rare disease patients is not very high owing to the dispersed patient pool of rare diseases. Knowledge and experience of the clinicians build over time while they deal with patients, which is not the case in rare diseases owing to fewer patients that report such diseases.
Screening and diagnosing?
Despite technological and medical advances in recent years, misdiagnosis or underdiagnosis of rare diseases forms a significant challenge. Often patients spend years, however, many neither get the name right nor treatment. Diagnosed without treatment is better than being underdiagnosed or misdiagnosed because in former cases, at least, patients get a chance to undergo the right treatment than none at all. However, diagnostic tests themselves cost a lot. There is a clear lack of services, technology, and understanding of the diagnostics and screening methodologies that can accurately detect and differentiate among a myriad of rare diseases.
Rare is costly.
In many cases, the treatment of rare diseases is extremely costly. Caretakers and families of patients shell out thousands of dollars yearly to buy orphan drugs, as designated by the USFDA. Pharmaceutical companies sell their orphan drugs at astronomical prices such that many patients are not able to afford them. Past years saw an increase in the entry of Orphan drugs in the market – at higher rates than ever before, and it is hurting the families and patients.
Funding is an issue!
A rare disease is a costly affair. High costs associated with launching a new drug in the market that has a limited patient pool discourages potential sponsors and pharma enterprises to invest in it. Therefore, rare disease patients were ‘orphaned’ by them and left without treatment. However unfortunate this is, it can be understood. Developing a new drug requires years of research, time, and money; and in cases where winds are against the market forces and profit margins appear shallow, conducting and bearing the cost for such trials can prove to be a pretty penny.
Conducting clinical trials for rare diseases?
Conducting clinical trials for orphan diseases is as important as for any other disease, however, in this case, it is nothing but a tough row to hoe. The clinical trials and patient studies are vital to deciding on effective and ineffective treatments; however, given the minuscule patient populations, the designing of the trials, recruitment of patients in the trials, and evaluating the results is extremely difficult. It makes it hard to rely on results obtained from short cohorts. Further, it gets expensive both in terms of time and money to run trials for rare diseases whose patients are widely spread all over the world.
Aftermath of clinical trials.
The struggle continues even after the designing and conducting of trials. Mapping the journey through the trials, and deriving an outcome from it is equally challenging. Further, the nod from regulatory agencies to run the trial, post-approval process, and assessment of the drug play a greater role that can not be neglected or compromised. Studies demonstrate varying results owing to varying age, the pace of progression of the disease, ethnicity, race, age, and severity. Thus, lost in diversity of data, researchers often find themselves in turmoil while measuring clinical trial outcomes in rare disease patients.
Thus, the crux of the matter is that lack of knowledge compounded with other challenges invariably lands patients, their caretakers, clinicians, and investigators in a difficult place. On the flipside, rare diseases project a shedload of opportunities as well. Medical research for orphan conditions has opened innumerable avenues to chart into the waters, explore the unexplored world of the rare disease, and emerge with novel therapies to lessen down their burden. Over time, several pharma companies such as Celgene, Takeda, Eli Lilly, Novartis, BMS, Alexion, Sanofi, Vertex Pharmaceuticals, Roche, bluebird bio, Amgen, Biogen, and many more have come up with novel approaches and novel orphan drugs that include oral, inhalable, and injectable therapies; drug-device combination products, novel gene therapies, diagnostics methods, and others to bring relief to the patients.
The health regulatory agencies all over have issued several guidelines, released strategies, and amended regulations to expedite the drug delivery process in the rare disease market landscape. Keeping in mind unique challenges, including low patient numbers, limited understanding of disease pathology and progression, variability in disease presentation, and a lack of established endpoints, regulatory bodies, and advisory bodies have taken a flexible stance. Undoubtedly, strides of improvements are made in healthcare, however, there remains a lot to traverse.
The biopharma industry saw numerous deal terminations in 2020. Clinical and regulatory results, change in control limitations, and strategic reprioritizations were among the most common reasons for deal termination.
Sanofi and Hanmi’s agreement in 2015 ranked first under which Hanmi regained WW rights to its protein/peptide discovery technology, lapscovery. The second position goes to the Merck KGaA termination of its development and commercialization deal with Pfizer
This article is based on the 2020 biopharma deals data provided by Chris Dokomajilar of DealForma. PharmaShots has compiled a list of the top 20 terminations of 2020 based on total deal value
Sanofi Terminated its 2015 Agreement with Hanmi
In Nov. 2015, Hanmi granted Sanofi exclusive, worldwide rights to its protein/peptide discovery technology, lapscovery to develop and commercialize a late-stage GLP1-RA agonist, efpeglenatide weekly insulin, and a fixed-dose weekly GLP-1-RA/insulin drug combination for the treatment of diabetes. Additionally, Hanmi had an exclusive option to co-commercialize the products in Korea and China. Hanmi received $434.7M up front and was eligible for up to $3.8B in development, regulatory, and sales-based milestones, plus double-digit royalties. In May 2020, Sanofi terminated the agreement and Hanmi regained worldwide rights.
Merck KGaA Terminated its Development and Commercialization Deal with Pfizer
Merck KGaA partnered with Pfizer to jointly develop & commercialize MSB0010718C for multiple types of cancer. The companies would develop the Ab as a single agent and in combination with Pfizer’s and Merck KGaA’s portfolio of approved and investigational oncology therapies. Both companies would collaborate on up to 20 immuno-oncology clinical development programs expected to commence in 2015, including up to 6 trials in P-II or P-III. Pfizer and Merck KGaA also partnered separately to co-promote Pfizer’s Xalkori in the US and other markets. Merck KGaA received $850M up front and was eligible to receive up to $2B in regulatory and sales milestones. Both companies would jointly fund all development and commercialization costs and split profits. On Mar. 19, 2019, Merck & Pfizer discontinued the P-III trial (JAVELIN Ovarian PARP 100) due to clinical trial results to develop avelumab in combination with CT followed by maintenance therapy of avelumab in combination with PARP inhibitor (talazoparib) for LA or metastatic ovarian cancer (Stage III or Stage IV). On Mar. 13, 2020, the companies terminated their P-III JAVELIN Head and Neck 100 trial of Bavencio (avelumab) with CRT for LA SCCHN as it failed to hit its 1Eps.
AbbVie (Allergan) Terminated its Development and Commercialization Deal with Assembly Biosciences
In Jan. 2017, Assembly Biosciences granted Allergan exclusive, worldwide rights to co-develop and commercialize ABI-M201 and ABI-M301 for the treatment of ulcerative colitis and Crohn’s disease, plus an additional two drugs for the treatment of Irritable Bowel Syndrome with Diarrhoea, with Constipation (IBS-C), or Mixed (IBS-M). Allergan and Assembly shared development costs until proof-of-concept studies. Assembly received $50M up front and was eligible for up to $2.77B in milestones, plus royalties. In May 2020, AbbVie acquired Allergan and on Jun. 18, 2020, AbbVie, terminated the agreement with Assembly Biosciences.
Eli Lilly Terminated its 2017 Agreement with CureVac
In Oct. 2017, CureVac granted Eli Lilly rights to develop and commercialize its 5 mRNA cancer vaccines using CureVac’s RNActive technology for up to 5 cancer vaccines that target neoantigens across multiple tumor types. Lilly was responsible for target identification, clinical development, and commercialization. CureVac was responsible for mRNA design, formulation, and manufacturing of clinical supplies. CureVac had the option to co-promote vaccines in Germany. CureVac received $50M up front, an equity investment of $51M (€45M), and CureVac would be eligible to receive more than $1.7B in development and sales milestones if all 5 vaccines are successfully developed, plus tiered royalties. In Jun. 2020, Eli Lilly terminated the agreement.
Voyager Terminated its Option and License Deal with AbbVie
In Feb. 2019, Voyager granted AbbVie options to license exclusive, worldwide rights to vectorized antibody-based gene therapies for Parkinson’s and other neurodegenerative diseases. Voyager would perform preclinical development to vectorize antibodies directed against alpha-synuclein designated by AbbVie. AbbVie would select one or more vectorized antibodies to advance into IND-enabling studies and clinical development. Voyager would be responsible for research, IND-enabling, and P-I clinical activities and costs. After completing P-I, AbbVie had an option to license the vectorized alpha-synuclein antibody program for further clinical development and global commercialization for Parkinson’s disease and other synucleinopathies. Voyager received $65M up front and was eligible for up to $245M in preclinical and P-I option payments, up to an additional $728M in development and regulatory milestones for each alpha-synuclein vectorized antibody compound, and up to $500M in total sales milestones, plus tiered royalties. On Aug. 3, 2020, Voyager Therapeutics terminated the agreement with AbbVie.
AstraZeneca Terminated its Development and Commercialization Deal with Allergan
In Oct. 2016, AstraZeneca’s global biologics research and development arm, MedImmune, granted Allergan exclusive, worldwide rights to develop and commercialize MEDI2070. The IL-23 monoclonal antibody was being developed for the treatment of Crohn’s disease and ulcerative colitis and was developed in partnership with Amgen Inc. under a 2012 deal. AstraZeneca received $250M up front and was eligible for up to $1.27B in milestones, plus royalties. AstraZeneca would share one-third of all payments and royalties with Amgen Inc. Amgen would also receive a single-digit inventory royalty on MEDI2070. On Jan. 27, 2020, AstraZeneca and Allergan mutually terminated the agreement, Allergan funded the ongoing study of brazikumab in CD and UC including CDx.
Eli Lilly Terminated its Research Partnership and Option to License Agreement with NextCure
In Nov. 2018, Lilly signed a research partnership with NextCure with an option to license immuno-oncology therapies using NextCure’s FIND-IO platform. Lilly had the exclusive option to license the antibodies resulting from the study. NextCure received $25M up front and Lilly invested $15M in NextCure. If Lilly exercised its option to license the drug, NextCure would be eligible to receive up to $1.4B in milestones, plus royalties. On Jan. 10, 2020, Eli Lilly terminated the agreement with NextCure effective Mar. 3, 2020.
Bridge Biotherapeutics and Boehringer Ingelheim Mutually Terminated their Development and Commercialization Deal
In Jul. 2019, Bridge granted Boehringer exclusive, worldwide rights to develop and commercialize BBT-877 for the treatment of fibrosing interstitial lung diseases such as idiopathic pulmonary fibrosis (IPF). The Bridge received $50.48M up front and was eligible for up to $1.23B in milestones, plus royalties. On Nov. 9, 2020, Bridge Biotherapeutics and Boehringer mutually terminated the agreement.
Voyager Terminated its License Option Deal with AbbVie
In Nov. 2018, Voyager granted AbbVie an option to license the development and commercialization of its vectorized antibodies targeting tau by combining AbbVie’s monoclonal antibody expertise and Voyager’s gene therapy platform and expertise to generate AAV vectors for the treatment of neurodegenerative diseases, including Alzheimer’s disease. Both companies planned to identify 5 antibodies based on Voyager’s gene therapy platform and select 3 antibodies for development. AbbVie had an option to license up to 2 antibodies following the completion of P-I and would be fully responsible for development costs. Voyager received $69M up front, and was eligible for up to $155M in preclinical and P-I option payments, and was eligible for up to $895M in development and regulatory milestones, plus royalties. On Aug. 3, 2020, Voyager terminated the agreement with AbbVie.
Amgen Terminated its 2006 Research Partnership with Cytokinetics
In Dec. 2006, Cytokinetics signed a research partnership with Amgen to develop CK-1827452 for the treatment of heart failure. Amgen had an exclusive option to license the drug candidate worldwide excluding Japan before the completion of a P-IIa study. Cytokinetics was responsible for initial development. Upon exercising its option, Amgen was responsible for all further development costs. Also, the partners developed cardiac myosin as a backup compound. Cytokinetics received $75M up front, including $33M in its common stock at a premium of $6.9M. Upon option exercise, Cytokinetics received a $50M option exercise fee and was eligible for up to $600M in development, regulatory, and sales milestones, plus escalating royalties. Cytokinetics had the option to co-fund the P-III study in exchange for additional royalties. If Cytokinetics decided to co-fund the study, it could co-promote the drug in North America. In 2013, Amgen licensed Japan rights, and in 2016 Amgen sublicensed the rights for Europe and the Commonwealth of Independent States to Servier. On Nov. 23, 2020, Amgen terminated the agreement and Cytokinetics regained worldwide rights for omecamtiv mecarbil and backup compound AMG 594. The termination was effective May 20, 2021. The sublicense agreement between Amgen and Servier would remain effective post-termination.
Roivant Terminated its Development and Commercialization Deal with Poxel
In Feb. 2018, Poxel granted Roivant Sciences and its subsidiary Metavant rights to develop and commercialize its oral therapy, Imeglimin, for T2 Diabetes. Poxel received $35M up front and was eligible for up to $600M in milestones, plus royalties. Roivant invested $15M in Poxel’s common stock by purchasing 1,431,399 ordinary shares at €8.5 per share. Roivant was responsible for worldwide development and commercialization. Poxel planned to contribute $25M to the development and had an option to co-promote the product. In Nov. 2020, Metavant terminated the agreement with Poxel for the development of Imeglimin following a strategic analysis.
Alexion Terminated its Development and Commercialization Deal with Affibody
In Mar. 2019, Affibody granted Alexion rights to develop and commercialize ABY-039 for IgG-mediated autoimmune diseases. Affibody received $25M up front and was eligible for up to $625M in development and sales-based milestones, plus tiered low double-digit royalties. Additionally, Affibody had an option to co-promote the bivalent antibody-mimetic targeting the neonatal Fc receptor (FcRn) in the US. In Feb. 2020, Alexion terminated the agreement following unfavourable early-stage data.
Boehringer Ingelheim Terminated its Development and Commercialization Deal with Zealand
In Jun. 2011, Zealand granted Boehringer Ingelheim exclusive, worldwide rights to develop and commercialize glucagon/GLP-1 dual agonists, including ZP2929, for the treatment of type-2 diabetes and obesity. Zealand received $52.4M up front, including reimbursements cost and $5.6M in research funding, and was eligible for up to $532.2M in development and commercial milestones, plus double-digit royalties. In Mar. 2020, Boehringer Ingelheim terminated the agreement and Zealand regained worldwide rights.
BMS (Celgene) Terminated its Development and Commercialization Deal with Jounce
In Jul. 2019, Jounce granted Celgene exclusive, worldwide rights to develop and commercialize JTX-8064 targeting the LILRB2 receptor on macrophages. Jounce received $50M up front and was eligible for up to $480M in milestones, plus royalties. In Jun. 2020, Bristol Myers Squibb terminated the agreement.
Cytokinetics Terminated its Development and Commercialization Deal with Astellas
In Jun. 2013, Cytokinetics granted Astellas exclusive, worldwide rights to co-develop and commercialize CK-2127107 for skeletal muscle weakness. Cytokinetics had the option to co-promote products in the US and Canada. Cytokinetics received $16M up front, $24M in R&D reimbursement, and was eligible for over $250M in development and sales milestones for collaboration products, including up to $112M for CK-2127107 and up to $200M in sales milestones, plus undisclosed royalties. On Dec. 22, 2014, Cytokinetics restated the agreement and granted Astellas exclusive, worldwide rights to co-develop & commercialize CK-2127107 and other products for SMA and other neuromuscular indications. Cytokinetics was responsible for P-II in patients with SMA and other neuromuscular diseases. Both companies would co-develop and co-commercialize CK-2127107 and other skeletal troponin activators in neuromuscular indications. Cytokinetics had the option to co-fund certain development costs, co-promote & conduct commercial activities for CK-2127107 in non-neuromuscular and neuromuscular indications in the US, Canada, and EU. On Jul. 27, 2016, Cytokinetics extended the agreement and granted Astellas additional rights to develop and commercialize tirasemtiv and CK-2127107 for ALS. Cytokinetics received a $100M option exercise fees and was eligible for additional milestones, plus royalties based on sales of tirasemtiv in Astellas’ countries. Also, Astellas was eligible for royalties based on Cytokinetics’ sales of tirasemtiv in its territory. On Apr. 23, 2020, the companies mutually terminated the agreement, Astellas contributed one-third of the development cost, or ~$12M, in an exchange of royalties in the US, Canada, and the EU for 10 years or reduced royalties until 2034. Also, Astellas returned all inventory and IP related to FRSA compounds and agreed not to engage in any research and development activities on FSRA compounds for 4 years.
Calithera Terminated its Development and Commercialization Deal with Incyte
In Jan. 2017, Calithera granted Incyte worldwide rights to develop and commercialize its arginase inhibitor, CB-1158, for the treatment of hematology and oncology indications. Incyte would fund 70% of the development cost while Calithera would fund 30%. Incyte and Calithera would share profits and losses, with Incyte at 60% and Calithera at 40%, based on U.S. sales. Incyte and Calithera planned to co-detail the drug in the U.S. and Calithera was eligible for up to $483M, including upfront and milestones, plus royalties ex-U.S. In Sep. 2020, Calithera terminated the agreement and regained worldwide rights.
Idorsia Terminated its Option and License Deal with Santhera for Vamorolon
In Nov. 2018, Idorsia granted Santhera an option to sublicense the development and commercialization of vamorolone for the treatment of DMD worldwide, excluding Japan and South Korea. Idorsia received 1M new registered shares of Santhera and an upfront cash payment of $20M. Idorsia became the largest shareholder in Santhera with a 13.3% equity position. The option was exercisable upon receipt of data from the P-IIb VISION-DMD study (VBP15-004). If exercised, Idorsia would receive a one-time payment of $30M and was eligible for regulatory & commercial milestones of up to $80M for the DMD indication and four one-time sales milestone payments of up to $130M in total. Idorsia was also eligible for regulatory milestone payments of up to $205M for three additional indications. On Sep. 2, 2020, Santhera exercised its option to license vamorolone for all indications including DMD, and revised the terms of the agreement. Vamorolone was originally developed by ReveraGen, and Actelion had the option rights. In Jun. 2017, Actelion spun off Idorsia as a separate entity along with vamorolone rights before its merger with Johnson & Johnson. According to the revised terms of the agreement, Idorsia received 366,667 shares in Santhera’s common stock and CHF10M in the form of convertible notes, up to 65% was converted in the form of Santhera common stock and transfer the control rights to its original developer ReveraGen BioPharma. Santhera had replaced Idorsia as party to the ReveraGen agreement.
AMAG Terminated its Development and Commercialization Deal with Palatin Technologies
In Jan. 2017, Palatin Technologies granted AMAG Pharmaceuticals rights to develop and commercialize Rekynda (bremelanotide) for the treatment of female sexual disorders in North America. Palatin received $60M up front, up to $380M in milestones, and $25M in reimbursement payments, plus royalties. In Jul. 2020, AMAG terminated the agreement and Palatin Technologies regained the rights plus $16.3M in termination fees.
Gilead Terminated its Development and Commercialization Deal with Precision BioSciences
In Sep. 2018, Precision granted Gilead rights to develop and commercialize therapies for Hepatitis B Virus targeting HBV cccDNA and integrated HBV DNA present in human hepatocytes using Precision’s ARCUS platform. Precision would focus on the development, formulation, and preclinical studies. Gilead was responsible for clinical studies and commercialization. Precision received research funding and was eligible for up to $445M in milestone payments and royalties. In Sep. 2020, Gilead terminated its agreement with Precision BioSciences.
ReveraGen Terminated its Option and License Deal with Actelion (Idorsia after the spin-off from Actelion)
In Nov. 2016, Actelion signed a research partnership with ReveraGen BioPharma with an option to license vamorolone for the treatment of DMD and other indications. Actelion would co-develop the compound for the next 28 months and would contribute $1M annually. ReveraGen received $10M up front, up to $165M in dev. and reg. milestones for DMD, and up to $190M for an additional three indications, plus tiered double-digit royalties. On Jun. 16, 2017, Actelion was acquired by Johnson & Johnson. Actelion spun off Idorsia as a new company. In 2018, Idorsia maintained the option rights for vamorolone and restructured the agreement. It agreed to contribute the research for an additional year (until mid-2020). ReveraGen initially received $15M to maintain the agreement, a $20M option fee, and would be eligible for up to $75M in milestones for the DMD indication, and three additional sales milestones of up to $120M. Royalties and milestone payments for other indications remain unchanged. In Nov. 2018, Idorsia granted Santhera an option to sublicense the development and commercialization of vamorolone for DMD worldwide, excluding Japan and South Korea. On Sep. 2, 2020, Santhera exercised its option to license vamorolone for all indications including DMD with the control rights transferred to ReveraGen BioPharma.
The BT designation is based on P-II CodeBreaK 100 study assessing Sotorasib in 126 patients with KRAS G12C-mutated locally advanced or metastatic NSCLC who have received at least one prior systemic therapy. The findings will be presented at the IASLC 2020 WCLC Presidential Symposium
The company is currently recruiting in a P-III study (CodeBreaK 200) assessing sotorasib vs docetaxel in patients with KRAS G12C-mutated NSCLC. Amgen has several P-Ib combination studies across various advanced solid tumors (CodeBreaK 101) open for enrolment
Sotorasib is being studied in the a clinical program exploring 10 combinations with global sites across the four continents
Click here to read full press release/ article | Ref: Amgen | Image: BioSpace
The P-II cohort of the CodeBreaK 100 clinical study involves assessing Sotorasib (AMG 510, 960mg, qd) in 126 patients with KRAS G12C-mutated advanced NSCLC. The findings will be presented at the IASLC 2020 WCLC
Results: @median follow up of 12.2 mos., ORR (37.1%); DCR (80.6%); mDoR (10 mos.), PFS (6.8 mos.). Median tumor shrinkage among all responders was 60% and complete responses observed
Sotorasib is 1st KRAS G12C Inhibitor to enter the clinic and is being studied in the broadest clinical program exploring 10 combinations with global sites spanning five continents
Click here to read full press release/ article | Ref: PRNewswire | Image: Fierce Pharma
Biosimilars are developed to be highly similar versions of approved biologics in terms of safety, purity, and potency.
Biosimilars are expected to be a cost-effective alternative to the high-priced branded biologics, offering significant and much-needed cost savings to both payers and the patients. Hence, the providers are more likely to adopt biosimilars as a “reference product to biologics” possessing similar therapeutic properties.
During the month of December, Celltrion receives CHMP’s positive opinion for CT-P17 (biosimilar, adalimumab) while Samsung Bioepis initiated P-II study SB16. Our team at PharmaShots has summarized 10 key events of the biosimilar space of Dec 2020
NMPA has approved HLX03 for the treatment of RA, AS, and PsO. It is the first China-developed biosimilar, approved both in China and in the EU
The approval marks the expansion of the Henlius’ commercial pipeline into the field of autoimmune diseases and the scope of patients befitted with the therapy
Henlius will continue optimizing and upgrading the product, and cooperating with Wanbang Biopharma to promote the commercialization of biosimilar, to bring high-quality treatment options to patients suffering from autoimmune diseases
Biomm to get exclusive rights to distribute & market the therapy in Brazil. Bio-Thera will be responsible for the development & commercial supply of BAT1706 out of its manufacturing facilities in Guangzhou, China
Bio-Thera’s BAT1706 has completed a global P-III comparative clinical study assessing the efficacy, safety, PK, and immunogenicity of BAT1706 vs EU-bevacizumab + CT in patients with advanced nsq. NSCLC
Biomm will be responsible for filing the dossier in Brazil. The partnership will leverage Biomm’s presence, sales, and marketing capabilities in Brazil
Health Canada has authorized Hyrimoz on Nov 4, 2020 for marketing in Canada. Hyrimoz has been approved for use in all same indications as reference Humira, including rheumatology, gastroenterology and dermatology
A patient support program will be available to patients treated with Hyrimoz providing guidance with reimbursement navigation, financial assistance, administrative support & education for patients
Hyrimoz is a fully human TNF blocker. The notice of compliance has been issued for 3 SC dosage forms: 40 mg/0.8 mL & 20 mg/0.4mL in prefilled syringe, 40 mg/0.8 mL in autoinjector
The approval is based on trial assessing Riabni (375 mg/m2, IV) vs Rituxan once weekly for 4wks. followed by dosing @12wks. & 20wks. in 256 patients in a ratio (1:1) with grade 1, 2, or 3a follicular B-cell NHL & low tumor burden
The WAC of Riabni will be 23.7% lower than the Rituxan in the US and will be available at a WAC of $716.80/ 100mg and $3,584.00/ 500mg single-dose vial
Riabni is a biosimilar to Rituxan, approved for the treatment of NHL, CLL, GPA, MPA and ill be made available in the US in Jan 2021
The approval is granted in pediatric plaque psoriasis and non-infectious intermediate uveitis, posterior uveitis and panuveitis in adults, who do not respond adequately to corticosteroids.
These are fifth and sixth approved indications of Sulinno in China and it was first approved by NMPA on Sep 2, 2020
Sulinno is an adalimumab biosimilar which is a recombinant human anti-TNF-α mAB developed by Innovent and are indicated in rheumatoid arthritis, ankylosing spondylitis, psoriasis, and polyarticular juvenile idiopathic arthritis
Forum participants analyzed key barriers and misinformation around biosimilar adoption to bridge gaps in provider knowledge, generate RWE evidence, streamline biosimilar approval processes, and improve education across safety and efficacy for biological drugs
Participants suggested multiple strategies around education, language, RWE, benefit design, legislation, regulations, confidence, and thought sharing to navigate the complexities of the biosimilar terrain
All findings & recommendations from the AMCP Partnership Forum will be published in an upcoming issue of AMCP’s Journal of Managed Care + Specialty Pharmacy
In its report released last Tuesday, a nonprofit drug pricing research group claims that seven drugs have prices that do not align with any newly discovered increase in clinical benefit, leading to over $1.2 billion in excess drug spending in 2019, alone.
US cost effectiveness watchdog ICER found 10 examples of substantial price rises for top-selling medicines in 2019, and concluded that seven of those were not backed by any clinical evidence.
The cost to the American taxpayer from those increases? Around $1.2 billion for the seven drugs alone, says the organisation, which also found that for all but one the list price increase was at least double the US rate of inflation in that year.
Amgen’s venerable TNF inhibitor Enbrel (etanercept) for arthritis and other inflammatory conditions topped the list of offenders in terms of the added cost to the healthcare system, which ICER – the Institute for Clinical and Economic Review – estimated to be $403 million.
Enbrel’s list price rose 5.4%, but the net price was up almost 9% once adjusted for other factors, which could include rebates, wholesale fees and other variables like patient assistance programmes.
Second place was Johnson & Johnson’s schizophrenia treatment Invega Sustenna/Invega Trinza (paliperidone), with a 6.8% increase in the list price – and 10.7% net – which added $203 million to US drug spending.
The biggest price increases were seen with Salix’ irritable bowel syndrome therapy Xifaxan (rifaximin) – up 8.4% and 13.3% respectively – which added $173 million to the national spend.
The rest of the seven were: Bristol-Myers Squibb’s arthritis drug Orencia (abatacept) at a cost of $145 million; Biogen’s multiple sclerosis therapy Tecfidera (dimethyl fumarate) at $118 million; AbbVie’s TNF drug Humira (adalimumab) at $66 million; and UCB’s Vimpat (lacosamide) for epilepsy which swelled spending by $58 million.
“Several of these treatments have been on the market for many years, with scant evidence that they are any more effective than we understood them to be years ago,” said ICER’s chief medical officer David Rind.
The increases came against a backdrop of relatively modest price increases overall in 2019, a time when the US administration had recently published a series of proposals to tackle high prescription drug prices, although many of those failed to come to fruition.
It’s worth noting that the scale of the increase identified in the latest report is considerably lower than was seen in ICER’s last edition in 2019. That also identified seven hefty price hikes for products – including Humira and Tecfidera – but cumulatively they all added a massive $4.8 billion to the US drugs bill.
According to a 2019 report by the OECD group of industrialised nations, the US spends roughly twice the average amount spent by other member countries on pharmaceuticals per head.
Three drugs also saw sizeable price increases, but ICER says those may have been justified by new clinical evidence – although the organisation notes it hasn’t run a full cost-effectiveness analysis.
The three were Novartis’ heart failure therapy Entresto (sacubitril/valsartan), Takeda’s Entyvio (vedolizumab) for ulcerative colitis and Astellas’ prostate cancer therapy Xtandi (enzalutamide).
Evoq to receive ~$240M up front & milestones along with royalties on the sales of therapies emerges from the collaboration
The companies collaborated on preclinical development, while Amgen will be responsible for clinical development and commercialization
The collaboration will bolster Amgen’s autoimmune offerings as its portfolio contains innovative medicines, including Otezla and Enbrel, and biosimilar products, such as Amgevita (a biosimilar to Humira) and Avsola (a biosimilar to Remicade)
Click here to read full press release/ article | Ref: PRNewswire | Image: Stat
“Lockdown’ declared Collins Dictionary word of the year. The year 2020, well known as COVID-19 year has been a busy year for global pharma and biotech companies involved in M&A, option & licensing agreements, and gaining approvals. Our team has compiled a list of 30 most read life sciences news on PharmaShots in 2020.
Dare to receive up front, $20M as option exercise fee, $310M as commercial milestones, royalties on sales of the product along with access to Bayer’s clinical and market capabilities and remain responsible for development & regulatory activities of Ovaprene
Bayer to get exclusive right to commercialize Ovaprene in the US, once approved by the FDA. Dare is expected to file IDE for the therapy in H1’20 and the initiation of its clinical study in H2’20 following FDA’s review and clearance of the IDE
Ovaprene is an investigational hormone-free monthly vaginal contraceptive, currently in development for the prevention of pregnancy and if approved, will be the first monthly non-hormonal contraceptive therapy
Theramax acquires commercialization rights of Zoely, allowing it to commercialize the therapy in 50+ countries globally. Earlier, Theramax has right to commercialize Zoely in eleven countries in the EU while MSD retains rights in the US and Canada
The acquisition of further rights of Zoely expands Theramex global footprints by providing innovative therapies to maintain the healthcare of women
Zoely is a combined oral contraceptive therapy consisting of two steroid hormones: 17-beta estradiol and nomegestrol acetate
Rockfeller to receive up front, milestones plus royalties on sales and will retain rights to perform non-clinical and early-stage clinical research on the portfolio of HIV Abs. Gilead to get exclusive rights to develop and commercialize Rockefeller’s full portfolio of HIV bNAbs
The focus of the agreement is to enhance Gilead’s HIV pipeline and will advance academic programs into potential future products
3BNC117 and 10-1074 are clinical-stage products with the ability to be used as HIV long-acting therapies for treatment and prevention
Fujifilm’s CAD EYE receives CE mark, backing the real-time detection of colonic polyps during colonoscopy utilizing AI, will be available with software EW10-EC01 and the compatible expansion unit EX-1 in combination with the ELUXEO 7000 system
CAD EYE utilizes FUJIFILM’s REiLi AI technology and can perform complex segmentation of 2D/3D images, spot lesions & is compatible with various imaging modalities. It automatically engages with white light or LCI mode to improve the accuracy of lesion detection
CAD EYE is customized detection support used with the ELUXEO system, aimed to improve lesion detection in the colon at the expert level and is expected to be available with EX-1 in Mar’2020
The EMA has accepted Type II Variation (T2V) for Zejula as maintenance therapy in a 1L setting for women with advanced platinum-responsive advanced OC, regardless of biomarker status. The validation indicates the acceptance of MAA and the initiation of CHMP’s formal review process
The submission is based on P-III PRIMA study assessing Zejula vs PBO in women in a ratio (2:1) as 1L therapy for stage III/ IV platinum-responsive advanced OC. The study demonstrated clinical outcomes of Zejula
Zejula (PO, qd) is a PARP inhibitor, indicated as a monothx. for the maintenance treatment of patients with platinum‑sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response to platinum-based CT
The approval is based on P-III trial Fixed Combination Drug Product LDL-C Lowering program, involves assessing of Nexlizet vs PBO when added on to maximally tolerated statins
The study resulted in well-tolerated data and lowered LDL-C by 38%, when added on to maximally tolerated statins. Also, the results are published in The European Journal of Preventative Cardiology
Nexlizet is an oral qd, non-statin LDL-cholesterol lowering medicine approved by the US FDA on Feb 21, 2020 and will be available in Jul 2020. Nexletol (bempedoic acid) is a novel ATP Citrate Lyase inhibitor involves lowering of LDL-C by cholesterol biosynthesis and up-regulating the LDL receptors, will be available from Mar 30, 2020
The P-III CASPIAN study involves assessing of Imfinzi + SOC (etoposide and carboplatin/ cisplatin CT) or Imfinzi + Tremelimumab vs CT as monothx. as a 1L treatment for 805 patients with ES-SCLC in 200+ centers across 23 countries
The first arm (Imfinzi + SOC) has met its 1EPs of OS in Jun’2019 while the second arm (Imfinzi + tremelimumab) did not meet its 1EPs of OS. The safety profile of each therapy is consistent with the known safety profiles
Imfinzi (durvalumab) is mAb targeting PD-L1, acts by blocking the interaction of PD-L1 with PD-1 & CD80 and is currently under review in combination with etoposide and carboplatin/cisplatin as 1L treatment for ES-SCLC in the US, EU and Japan with its anticipated PDUFA date in 2021
Health Canada has expanded the approval of Fiasp (10mL vials) to include use in insulin infusion pumps for the improvement of glycemic control in pediatric patients aged ≥2yrs with diabetes (T1D/T2D both)
The approval is based on data from 7 clinical studies that verified the safety & efficacy of Fiasp in children. The label change for use in insulin infusion pumps is based on Health Canada’s review of data from 5 clinical studies which demonstrated the efficacy of Fiasp used in insulin infusion pumps in adults with diabetes
Fiasp is the first and only fast-acting mealtime insulin injection, administered at the beginning of a meal or within 20 minutes after starting a meal and has received FDA’s approval as an IV infusion or SC MDI in adults with diabetes
The approval is based on P-III CHANGE II study assessing extreme regimen (ERBITUX + cisplatin + 5-FU, followed by ERBITUX as maintenance therapy) vs Pt. based CT (cisplatin + 5-FU) in 243 patients aged ≥18 yrs. with R/M SCCHN, prior not treated with any systemic therapy in China
The P-III CHANGE II study results: improvement in PFS (5.5 vs 4.2mos.); OS (10.2 vs 8.4mos.), ORR (50% vs 27%) with no new safety findings
ERBITUX is an IgG1 mAb targeting the EGFR and is an approved therapy in 100+ countries for RAS wild-type m-CRC and for HNSCC
The clinical study will evaluate the safety and efficacy of Virazole + SOC in hospitalized patients aged ≥18yrs. with respiratory distress due to COVID-19
The clinical study has been approved by Health Canada and is expected to initiate within the next few weeks. The company is working with multiple health authorities including the US FDA regarding additional studies to assess Virazole as a treatment for COVID-19 infection
Virazole (ribavirin for inhalation solution, USP) aerosol is indicated only for lower respiratory tract infection due to RSV. The Bausch Foundation is working with health authorities in Italy to make Virazole for inhalation available free of charge in compassionate use in hospitals
Adaptive to expand its platform for selection of B cell receptors of recovered patients from COVID-19. Amgen will utilize its Ab engineering and drug development capabilities to select and develop Abs designed to bind and neutralize SARS-CoV-2. Additionally, Amgen ‘s subsidiary DeCODE Genetics located in Iceland, will provide genetic information from patients infected with COVID-19
The focus of the collaboration is to combine expertise to discover and develop fully human neutralizing Abs for SARS-CoV-2 virus to treat COVID-19 where Adaptive’s immunological medicine platform will help in identification of virus neutralizing Abs
Additionally, the Abs can be used to treat patients with COVID-19 and can be administered to patients with who are at increased risk of exposure to SARS-CoV-2
The companies reported that the first cohort of BioNTech’s P-I/II clinical trial has dosed 12 participants with BNT162 in Germany since dosing began on Apr 23, 2020. Following the regulatory approvals, both the companies plan to initiate the clinical study for BNT162 in the US
The dose-escalation portion of the P-I/II study will include ~200 healthy participants aged 18-55yrs. and will target a dose range of 1-100 µg, focusing on determining the optimal dose for further studies and to evaluate the safety and immunogenicity of the vaccine
The study will evaluate the effects of repeated vaccination following a prime injection for 3 vaccine candidates that contain uRNA or modRNA. The fourth vaccine candidate contains saRNA will be evaluated following a single dose of vaccine. Additionally, BioNTech is collaborating with Fosun Pharma to develop BNT162 in China, where the companies expect to conduct clinical studies
Fresenius Kabi and Medec collaborated to offer IDACIO as an additional therapy option for rheumatologists and dermatologists to treat rheumatic illnesses. From Jun 01, 2020, Medec’s will market the biosimilar therapy
Last year, Fresenius Kabi launched IDACIO in the EU for arthritis and psoriasis. The collaboration offers patients and doctors new benefits and synergies in therapy offerings as well as consulting
Medac provides methotrexate (metex PEN, metex FS) as the parenteral treatment of patients with chronic inflammatory diseases, the affected patients are treated with a combination of methotrexate and adalimumab
Idorsia to receive $45M upfront in cash, $365M for development & regulatory milestone, one-time sales threshold and royalties on sales. Additionally, will receive $7M in funding to discover, identify and develop additional novel T-type calcium channel blockers
Neurocrine exercises its option to license rights for ACT-709478 (post IND acceptance from the US FDA on Apr 30, 2020) for rare pediatric epilepsy. In 2019, Neurocrine and Idorsia signed a preclinical research collaboraion for ACT-709478 to treat rare pediatric epilepsy
ACT-709478 is an selective, orally-active and brain penetrating T-type calcium channel blocker also received the US FDA’s Rare Pediatric Disease designation for rare pediatric epilepsy with completion of P-I in 2019 and expected P-II initiation in in H2’20
The companies intend to establish manufacturing suites at Lonza’s facilities in the US and Switzerland to manufacture mRNA-1273 at both sites. The collaboration will deploy Lonza’s global expertise in technology transfer and manufacturing while the technology transfer expected to begin in Jun’2020
The focus of the collaboration is to enable the manufacturing of mRNA-1273 up to 1B doses/year and anticipates the manufacturing of the first batches of mRNA-1273 at Lonza US site in Jul’2020, assuming the currently expected dose of 50µg
Manufacturing operations at Lonza US site is covered by Moderna’s agreement with BARDA under which BARDA will support late-stage clinical development programs of mRNA-1273. On Apr 27, 2020, Moderna has submitted IND to the US FDA for P-II studies with its expected initiation in Q2’20
The approval is based on two P-III SAkuraStar & SAkuraSky studies involve assessing Enspryng (120mg, SC, q4w) as a monothx & as an add-on therapy to baseline IST vs PBO in 95 & 83 patients aged 20-70 & 13-73yrs. in a ratio (2:1) & (1:1) administered at week 0,2 & 4 in patients with NMOSD respectively
In overall population: reduction in the risk of relapse (62% & 55%); In the pre-specified subgroup of AQP4-IgG seropositive patients: reduction in the risk of relapse (79% & 74%) respectively
Enspryng is a mAb targeting IL-6 and is under PR in Canada for NMOSD patients who are AQP4-IgG seropositive. In Oct’2019, the FDA & EMA has accepted the MAA for the therapy with expected CHMP & FDA’s decision in 2020
The US FDA has issued a EUA to Gilead for emergency use of remdesivir to treat hospitalized COVID-19 patients. In May, Gilead has extended a voluntary non-exclusive license to Cipla to manufacture and market Cipla’s Remedisvir called CIPREMI
Cipla has received DCGI’s approval for restricted emergency use in India as part of the accelerated approval process. Cipla will provide training on the use of the drug, informed patient consent documents, conduct post-marketing surveillance as well as to conduct a P-IV clinical trial on Indian patients
As per ACTT-1 study, 1063 patients were treated with Remdesivir vs PBO over 60 centers across the US, EU and Asia demonstrated faster time to clinical recovery in hospitalized patients with the mortality rate as (7.1% vs 11.9%)
Sorrento has reported that its EUA is under review at the US FDA for its COVI-TRACK in vitro diagnostic test kit for the detection of IgG and IgM Abs in sera of patients exposed to the SARS-CoV-2 virus
Following the issuance of an EUA, the COVI-TRACK test will be available for distribution to clinical testing sites nationwide. The assay develops three clear lines that confirm the assay validity and the qualitative detection & differentiation of IgM and IgG Abs to the COVID-19
Sorrento has secured manufacturing capacity to ramp up the production of up to 5M test kits/ month with the availability of results in ≤8mins. The assay showed specificity > 97% and diagnostic sensitivity of > 94% in an analytical validation
Fujifilm to receive upfront, license fee along with royalties on sales of the therapy. Dr. Reddy’s and GRA to get the exclusive right to develop & commercialize Avigan globally (Ex- Japan). Additionally, Dr. Reddy’s would have exclusive rights for the therapy in India
Fujifilm will provide pre/ clinical data of Avigan to Dr. Reddy’s and GRA for utilizing it in clinical studies targeting COVID-19. Moreover, Dr. Reddy’s will get right to use Avigan’s patents of formulation and manufacturing method and will establish a setup for developing drug-like Avigan and utilizes the GRA’s global sales network to supply the manufactured drugs
Fujifilm is currently conducting a clinical study on Avigan targeting COVID-19 patients in the US and Japan and is collaborating with multiple companies to increase the drug’s production
The approval is based on P-III BRIGHTE study assessing Rukobia (600mg, ER) + OBT in 371 HTE adults living with multidrug-resistant HIV. Participants were enrolled in either a randomized or nonrandomized cohort
In the randomized cohort, 60% adults achieved undetectable HIV viral load and clinically meaningful improvements to CD4+ T-cell count @96wks., HIV-1 RNA <40 copies/mL @24 & 96wks. (53% & 60%); changes in CD4+ cell count (90 & 205 cells/mm3) respectively
In the nonrandomized cohort, 37% achieved HIV-1 RNA <40 copies/mL @24 & 96wks.; HIV-1 RNA <200 copies/mL (42% & 39%); mean changes in CD4+ cell count (41 & 119 cells/mm3) respectively. Fostemsavir is a first-in-class HIV-1 attachment inhibitor, currently under EMA’s review with additional submissions to regulatory authorities anticipated in 2020 & 2021
The company has launched Remdec at a price of $37.41 (Rs. 2800) for a 100mg lyophilized injection. The generic version is the most economical Remdesivir brand in India
In Jun’2020, Zydus signed a non-exclusive agreement with Gilead to manufacture and commercialize Remdesivir for severe COVID-19 in India. The API of the therapy has been developed and manufactured at the group’s API manufacturing facilities in Gujarat
The drug will be made available across India via Zydus’ strong distribution chain reaching out to government and private hospitals treating COVID patient
J&J acquires Momenta in all-cash transaction at a price of $52.50/ share, making a total deal value as $6.5B. The transaction is expected to be closed in H2’20
The acquisition allows J&J to expand its portfolio for autoimmune diseases with the addition of Momenta’s Nipocalimab (M281) to its pipeline. In addition to nipocalimab, Janssen will acquire Momenta’s pipeline of clinical and pre-clinical assets
Janssen plans to retain Momenta’s presence in Cambridge, Massachusetts which will increase J&J footprint and capabilities in the key innovation hub. Nipocalimab provides an opportunity for Janssen to deliver transformative treatments in autoantibody-driven autoimmune diseases
The EU label update includes additional categorization of retinal vasculitis and/or retinal vascular occlusion, usually in the intraocular inflammation. The approval follows Novartis completion of safety review and initiation of an update to the Beovu prescribing information globally
The label update is applicable to all 27 EU member states as well as UK, Iceland, Norway, and Liechtenstein. Beovu is now approved for wet AMD treatment in 40+ countries including in the US, EU, UK, Japan, Canada, and Australia
Beovu (brolucizumab) is the clinically advanced humanized single-chain Ab fragment (scFv) that enhances tissue penetration, rapid clearance from the systemic circulation, and drug delivery characteristics. Novartis has established a multidisciplinary panel of internal experts collaborating with external advisors to examine the root cause, potential risk factors, and mitigation of AEs
The P-II trial will assess BI 764198 (qd for ~4wks.) in patients hospitalized for COVID-19 with expected enrollment initiation in Oct’2020. The 1EPs will be the percentage of patients who are alive and free of mechanical ventilation at day 29 of treatment while other EPs include clinical improvement, oxygen saturation & ICU admission
The therapy has shown a reduction in cellular damage and lung edema in preclinical studies and may provide similar benefits in patients with severe SARS-CoV-2 infection. BI 764198 was well tolerated in P-I study in healthy adults
BI 764198 is potent & selective inhibitor of TRPC6, focusing to reduce the need for ventilator support and to improve patient recovery rate
The approval was based on the P-III IMbrave150 study (n=501) assessing the combination of Tecentriq (1200 mg, IV) and Avastin (15 mg/kg, IV) or sorafenib (400 mg, bid) in unresectable HCC patients who had not received prior systemic therapy which included analyses of a cohort of Chinese patients (n=194) from the same study
Results: Tecentriq in combination with Avastin reduced the risk of OS by 56% (among Chinese patients) and 42% (global results) & the PFS risk by 40% (among Chinese patients) and 41% (global results) as compared with sorafenib
IMbrave150 is the 1st P-III cancer immunotherapy study to show an improvement in OS and PFS in people with unresectable or metastatic HCC compared with sorafenib. Additionally. in May 2020, the US FDA approved Tecentriq in combination with Avastin for the treatment of people with unresectable or metastatic HCC who have not received prior systemic therapy
Sanofi will sponsor the clinical trials while MSD will provide KEYTRUDA. Additionally, Sanofi is separately evaluating the activity of THOR-707 in combination with other anti-PD-1 antibodies, including Libtayo (cemiplimab-rwlc) and with anti-EGFR and anti-CD38 antibodies for various types of cancer tumors
In preclinical studies, THOR-707 demonstrated the ability to induce the expansion of CD8+T-cells resulting in anti-tumor effects both as a single agent as well as in combination with an anti-PD1 mAb
THOR-707 is currently being evaluated by Sanofi in an ongoing P-I dose escalation and expansion trial assessing THOR-707 and determining its recommended P-II dose alone and in combination with anti-PD-1 and anti-EGFR antibodies
Fusion to receive up front, as well as development milestones and other payments. The companies will jointly discover, develop, and have an option to co-commercialize novel TATs in the US while AstraZeneca will lead commercialization in the ROW with equal profit & loss sharing globally
The collaboration leverages Fusion’s TAT platform and expertise in radiopharmaceuticals with AstraZeneca’s leading portfolio of Abs and cancer therapies, including DDRis
Additionally, the companies will exclusively explore certain specified combination strategies between TATs (including Fusion’s FPI-1434) and AstraZeneca’s therapies for the treatment of multiple cancers. Both companies will retain full rights to their respective assets
The Lucira’s COVID-19 all-in-one test kit test has been authorized for home use with self-collected nasal swab samples in individuals aged≥ 14yrs. who are suspected of COVID-19 by their HCPs
It is also authorized for use in POC settings for all ages, but samples must be collected by an HCP when the test is used at the POC to test individuals <14yrs. The test is currently authorized for prescription use only
Lucira plans to amend its EUA or file a new EUA so people who think they’re infected with COVID-19 can communicate with a medical professional online through a website to arrange a prescription and overnight delivery of the test kit by Q2’21
The acquisition will bolster UCB’s pipeline program, capabilities, and platforms in the gene therapy space. The Handl Therapeutics will continue to be based in Leuven, Belgium while working closely with UCB’s international research teams
In addition, the UCB collaborated with Lacerta to focus on CNS diseases, under which Lacera will lead research, preclinical activities, and the early manufacturing process development, while UCB will complete IND-enabling studies, manufacturing, and clinical development
The collaboration will allow UCB to access Lacerta’s expertise in AAV-based CNS targeted gene therapies, fortifying UCB’s ability to produce effective treatments for neurodegenerative diseases
Janssen acquires rights to Hemera’s HMR59, administered as a one-time, outpatient, IVT inj. to help preserve vision in patients with geographic atrophy
The acquisition will boost Janssen’s eye disease portfolio & strengthens its gene therapy capabilities
HMR59 is designed to increase the ability of retina cells to make a soluble form of CD59, helping to prevent further damage to the retina and preserve vision. The P-I study of the therapy for patients with geographic atrophy is completed while the P-I study exploring HMR59 in patients with wet-AMD is currently conducting follow-up visits to evaluate the long-term safety
Amgen has decided that a drug for leprosy and tuberculosis it inherited as part of its acquisition of Celgene’s psoriasis blockbuster Otezla last year would fare better in the hands of a non-profit company, dedicated to bringing affordable medicines to poorer countries.
AMG 634 – a PDE4 inhibitor being investigated for TB and leprosy complication erythema nodosum leprosum (ENL) – is being licensed to Australia’s Medicines Development for Global Health (MDGH), with Amgen surrendering all rights to the drug.
The US biotech will continue to provide support for two phase 2 trials of AMG 634 in TB and ENL which are due to get underway next year by producing and supplying the drug and funding the ENL trial.
AMG 634 acts as an anti-inflammatory, so it aims to tackle the complications of these chronic infections rather than attacking the infectious organisms themselves.
TB – caused by Mycobacterium tuberculosis – remains a massive public health problem worldwide, affecting 10.4 million patients every year and causing over a million deaths. About a quarter of the world’s population carry the bacterium, and have a 5% to 15% chance of developing the disease.
Current treatments are often inadequate, with resistance emerging to many of the mainstay drugs used to treat the infection, and if it isn’t treated effectively can leave patients with permanent, clinically significant lung damage.
Leprosy meanwhile generally doesn’t hit the headlines like TB, but despite being curable with antibiotic therapy still causes more than 200,000 infections every year, according to the World Health Organization.
Caused by the bacterium Mycobacterium leprae, the infection develops slowly – sometimes it can take a year or more for symptoms emerge – if untreated it can cause progressive and permanent damage to the skin, nerves, limbs, and eyes.
Treatment typically requires months or even years of multidrug therapy with antibiotics – generally with dapsone, rifampicin and clofazimine – but compliance over that long period can be problematic, particularly in countries with less sophisticated healthcare systems.
A significant number of patients go on to develop ENL, an autoimmune complication that can occur many years after being cured of leprosy and can cause permanent nerve damage and disability.
MGDH has already demonstrated that it can bring drugs to market for diseases that mainly afflict low- and middle-income countries, launching moxidectin – the first new treatment for river blindness (onchocerciasis) in 30 years – in 2018.
Its founder and managing director, Mark Sullivan, said the company is “honoured to take over the stewardship of this compound from Amgen… and we will now undertake full development of AMG 634 in hopes of bringing it to patients in need of a treatment for their disease.”
AstraZeneca and Amgen looked on course to cruise to approval of their severe asthma drug tezepelumab with a pair of positive clinical trials, but the failure of a third threatens to derail the programme.
The new study – called SOURCE – was supposed to back up the encouraging readouts from the phase 3 NAVIGATOR trial and phase 2b PATHWAY, which found that the antibody benefited asthma patients whose symptoms were so bad they needed oral corticosteroid (OCS) therapy.
SOURCE has changed the script however by missing its primary objective, proving unable to significantly reduce the daily dose of OCS needed by patients in the 150-subject trial, although AZ and Amgen still think the drug is approvable based on the “totality of evidence”.
AZ and Amgen have only released the top-line data, so it could be a while before an explanation for the failure emerges, although AZ’s head of biopharmaceuticals R&D Mene Pangalos suggested it may have resulted from flaws in the trial design.
Just a month ago, AZ and Amgen trumpeted the result of the NAVIGATOR trial of tezepelumab, a first-in-class inhibitor of the cytokine thymic stromal lymphopoietin (TSLP) that analysts have said could have blockbuster potential if approved, perhaps reaching as much as $2.5 billion in annual sales.
NAVIGATOR found that the antibody reduced the asthma exacerbations compared to placebo when added to standard care, and crucially also seemed to have a positive impact on patients with low levels of white blood cells called eosinophils.
Biologics like GlaxoSmithKline’s Nucala (mepolizumab) and AZ’s Fasenra (benralizumab) – both IL-5 inhibitors – and Sanofi/Regeneron’s IL-4 and IL-13 blocker Dupixent (dupilumab) are already approved to treat severe eosinophilic asthma but treatment options are limited for patients with non-eosinophilic forms.
Tezepelumab claimed a breakthrough designation in non-eosinophilic asthma from the FDA in 2018, on the promise that it could provide an option for these patients, who account for around a third of all severe asthma cases.
The drug acts further upstream in the inflammatory cascade than its rivals and so could be effective for a broader range of patients, according to its developers.
AZ and Amgen say they still plan to press ahead with plans for regulatory filings for tezepelumab next year, and will prevent detailed results from NAVIGATOR and SOURCE at a future medical conference.
The two partners started working together on tezepelumab in 2012, and was one of five drugs covered by that alliance. It has previously failed a mid-stage study in atopic dermatitis, but is still in development for that indication as well as chronic obstructive pulmonary disease (COPD).
The approval is based on trial assessing Riabni (375 mg/m2, IV) vs Rituxan once weekly for 4wks. followed by dosing @12wks. & 20wks. in 256 patients in a ratio (1:1) with grade 1, 2, or 3a follicular B-cell NHL & low tumor burden
The WAC of Riabni will be 23.7% lower than the Rituxan in the US and will be available at a WAC of $716.80/ 100mg and $3,584.00/ 500mg single-dose vial
Riabni is a biosimilar to Rituxan, approved for the treatment of NHL, CLL, GPA, MPA and ill be made available in the US in Jan 2021
Click here to read full press release/ article | Ref: PRNewswire | Image: Adweek
Amgen has filed its groundbreaking KRAS inhibiting drug sotorasib with the FDA for a group of lung cancer patients with an aggressive form of the disease.
The drug was the first targeted at the mutation known as KRAS to show activity in the clinic and provided the biggest talking point at the American Society of Clinical Oncology (ASCO) conference in 2019.
Since then Amgen has been gathering evidence to support a filing in a group of patients with advanced or metastatic KRAS G12C mutated non-small cell lung cancer.
The FDA is reviewing sotorasib under its Real-Time Oncology Review (RTOR) programme and could be the first to be approved in this indication, which covers around 13% of NSCLC patients.
Amgen’s filing is on track with a schedule laid out at the beginning of the year, following a top-line read out from a phase 2 trial in October.
These results came from the CodeBreaK 100 clinical study, which tested the drug in patients whose cancer had progressed despite prior treatment with chemotherapy and/or immunotherapy.
In the study, treatment with sotorasib provided durable anticancer activity with a positive benefit-risk profile, Amgen said, although detailed results have yet to be announced.
Full results will be presented at the International Association for the Study of Lung Cancer (IASLC) 2020 World Conference on Lung Cancer (WCLC) Presidential Symposium next month.
KRAS is a target that has long evaded pharma companies but early trial results in solid tumours at ASCO led to a round of deal-making involving rivals.
Mirati, a biotech from California, specialises in drugs targeting KRAS and is a step behind Amgen with its rival adagrasib.
Novartis signed a deal to evaluate Mirati’s drug soon after ASCO and Merck & Co and Boehringer Ingelheim are among those who have signed KRAS deals.
Although it looks like the molecules developed so far will work only in lung cancer, rather than the wider range of cancers with KRAS mutations, there is hope the drug will provide a new treatment option for an aggressive and deadly form of the disease.
David Reese, executive vice president of Research and Development at Amgen, said: “Sotorasib was the first KRASG12C inhibitor to enter the clinic and now is on track to potentially be the first approved targeted therapy for patients with advanced NSCLC harbouring the KRAS G12C mutation.”
The BT designation is based on P-II CodeBreaK 100 study assessing Sotorasib in patients with advanced NSCLC with KRAS G12C mutation whose cancer had progressed despite prior treatment with CT and/or immunotherapy
The company is currently recruiting in a P-III study (CodeBreaK 200) assessing sotorasib vs docetaxel in patients with KRAS G12C-mutated NSCLC. Amgen has several P-Ib combination studies across various advanced solid tumors (CodeBreaK 101) open for enrollment
Sotorasib has also accepted into FDA’s Real-Time Oncology Review Pilot Program. Additionally, the company plans to submit the NDA to the US FDA by the end of 2020
Click here to read full press release/ article | Ref: PRNewswire | Image: BioSpace
Amgen, UCB, and Takeda reported that the first patient has been enrolled in the COMMUNITY trial. The study will test whether Amgen’s Otezla, Takeda’s lanadelumab, and UCB’s zilucoplan can reduce the severity of COVID-19 in hospitalized patients by moderating the immune system’s response to the disease
The focus of the trial is to identify an effective treatment for hospitalized COVID-19 patients, who are Grade 2 to Grade 5 on a clinical severity status 8-point ordinal scale. Trial sites are being established in hospitals across the globe to support enrollment in communities
This is the first time that the global pharmaceutical has come together to launch an adaptive clinical trial
Click here to read full press release/ article | Ref: Takeda | Image: Stat
The P-III NAVIGATOR study involves assessing Tezepelumab + SOC vs pbo + SOC in adults (18–80yrs.) & adolescents (12–17yrs.) with severe, uncontrolled asthma, who were receiving treatment with medium/high dose ICS + at least 1 additional controller medication with or without OCS
Trial met its 1EPs i.e. reduction in AAER @52wks. in the overall population. The study also met 1EPs in patients with low levels of eosinophils i.e. <300 & 150 cells/microlitre
Tezepelumab is mAb that inhibits the action of TSLP and has received US FDA’s BT designation in Sept’2018 for patients with severe asthma, without an eosinophilic phenotype
Click here to read full press release/ article | Ref: AstraZeneca | Image: The Indian Express
Novartis’ injectable migraine prevention antibody Aimovig has been shown to be more effective than topiramate – a go-to oral therapy for people with chronic migraine – in a head-to-head trial.
The HER-MES trial found that Aimovig (erenumab) was more effective at preventing migraine attacks and also better tolerated than topiramate, a generic epilepsy drug which is known to have side effects like sleepiness, dizziness, diarrhoea and nausea.
In the 777-patient study, fewer patients on Novartis’ drug discontinued treatment due to side effects, while more of them met the objective of a 50% reduction in the number of days in a month they had a migraine compared to high-dose topiramate.
Topiramate also needs to be taken twice a day, while Aimovig is given as an injection once a month and is available in a self-injector pen device. Both drugs can take up to three months for their effect on migraine prevention to fully kick in.
Novartis recorded Aimovig sales of $108 million in the first nine months of 2020, a rise of 44% on the same period of 2019, and says it is the most prescribed drug in the CGRP inhibitor class with 480,000 patients using it worldwide.
Quarterly sales have however fallen from a strong launch in 2018, and the drug can’t seem to generate the momentum needed to meet blockbuster sales expectations voiced during its development.
Part of that is the entry into the market of CGRP rivals, with three injectable drugs (Teva’s Ajovy and Eli Lilly’s Emgality) and one six-monthly infusion (Lundbeck’s Vyepti) now jostling for position in the migraine prevention market.
Meanwhile, two oral CGRP inhibitors – AbbVie’s Ubrelvy and BioHaven’s Nurtec – have been launched for the on-demand treatment of acute migraine attacks. These don’t compete with Aimovig and the other prevention therapies directly, but are raising the profile of the CGRP class among migraine sufferers and their doctors.
This year of course there have also been access issues caused by the coronavirus pandemic that have had a particularly big impact on neurology prescribing, as well as a continued challenge in persuading doctors to switch to the new class from older drugs like topiramate and AbbVie/Allergan’s Botox.
It’s estimated that CGRP drugs have only penetrated 15% of the migraine prevention market, and that’s why the HER-MES results are so important to Novartis.
“These results further emphasise its potential to provide significant relief from migraine with an infrequent dosing compared with the oral treatment,” said Estelle Vester-Blokland, the company’s global head of neuroscience medical affairs.
The company has exclusive rights to the sell Aimovig outside the US, where Amgen records sales, with the exception of Japan. Novartis and Amgen are however locked in a legal battle over marketing rights to the drug.
Bristol-Myers Squibb could be on the verge of a major coup in psoriasis after its deucravacitinib pill outperformed Amgen’s rival Otezla.
BMS had to sell off Otezla (apremilast) as a condition of its merger with Celgene and made Amgen pay $13.4 billion in cash for the popular pill treatment, which is not quite as effective as injections such as J&J’s Stelara (ustekinumab) but more convenient.
The reason why competition regulators were concerned about Otezla was BMS’ deucravacitinib, which was in Bristol’s pipeline when the $74 billion merger was being finalised last year.
Also known as BMS-986165, BMS was developing the drug as a potential best-in-class oral therapy with a different mechanism of action and cleaner safety profile.
Latest phase 3 results show that BMS may have played a blinder by getting a eye-watering price for Otezla while retaining the better asset: deucravacitinib has outperformed placebo in the POETYK PSO-1 phase 3 trial and met several secondary goals showing it outperformed Otezla while showing a safety profile similar to that seen in phase 2.
Results from the trial testing deucravacitinib in patients with moderate to severe disease showed more patients achieving 75% skin clearance compared with placebo at week 16.
Using a global scale, doctors scored more patients on deucravacitinib as clear or almost clear compared with placebo, meaning the trial’s other main endpoint was met.
BMS said deucravacitinib also beat Otezla on the 75% skin clearance score and the global doctors’ assessment.
BMS added that the overall safety profile of deucravacitinib in the POETYK PSO-1 trial was consistent with previously reported phase 2 results, where there was little to differentiate placebo and treatment groups.
Results of an almost identical phase 3 study, POETYK PSO-2 are due in the first quarter of next year and phase 2 findings will be presented at the American College of Rheumatology conference later this week.
Deucravacitinib has a different mechanism of action than other psoriasis drugs on the market – it is a tyrosine kinase 2 inhibitor that changes intracellular signalling and is also in development for several other inflammatory and immune diseases.
Immunology is an important branch of science which deals with the study of the immune system. The immune system is a highly regulated and balanced system and when the balance is disturbed, the disease can result. A lot of this work has importance in the development of new therapies and treatments that can handle or heal the condition by modifying the way the immune system is working or, in the case of vaccines, instructing the immune system and enhancing the immune reaction to specific pathogens. In the top 20 ledgers, AbbVie again ensured the top position with total revenue of $19.57B with its blockbuster drug, Humira (adalimumab) from its immunological segment. Our team at PharmaShots has compiled a list of the top 20 immunology companies based on their 2019 immunology revenue
Immunology Segment Revenue: $0.01B
Founded Year: 1979
Market Cap: ~$0.49B
Total Employees: ~178
Headquarter: New Jersey, United States
Stock Exchange: NASDAQ
AntaresPharma is an American pharmaceutical company focus on developing and commercializing therapies for rheumatology, urology, endocrinology, and neurology. Antares has reported a total sale of $0.01B from its immunology segment in 2019
Immunology Segment Revenue: $0.24B
Founded Year: 2008
Market Cap: ~$15.70B
Total Employees: ~1,200
Headquarter: Dublin, Ireland
Stock Exchange: NASDAQ
Horizon Therapeutics is an Ireland based biopharmaceutical company focused on developing and commercializing therapies for the treatment of gout, rheumatoid arthritis, and rare diseases. Horizon has generated the sale of $0.24B from its four approved immunology products including Tepezza, Rayos, Duexis and Vimovo. Horizon’s Tepezza was selected for “2020 R&D World R&D 100 Award”
Immunology Segment Revenue: $0.49B
Founded Year: 1993
Market Cap: ~$2.57B
Total Employees: ~5,047
Headquarter: Shenyang, China
Stock Exchange: HKD
3SBio is a fully integrated Chinese biotechnology company with market-leading biopharmaceutical franchises in oncology, auto-immune diseases, nephrology, metabolic diseases, and dermatology. There are three approved drugs in its immunology portfolio including Yisaipu, Tpiao and Xenopax. 3SBio’s Tpiao used to treat chemotherapy-induced thrombopenia (approved in 2005) and immune thrombocytopenia has generated global sales of $0.33B in 2019.
Immunology Segment Revenue: $0.73B
Founded Year: 1978
Market Cap: ~$42.57B
Total Employees: ~7,400
Headquarter: Massachusetts, United States
Stock Exchange: NASDAQ
Biogen is a global biopharma company focused on neurology, hematologic, and autoimmune diseases. Biogen has a total of six products in its immunology segment with four approved drugs including Tysabri, IMRALDI, FLIXABI, BENEPALI. Biogen’s lead drug Tysabri recorded a revenue of $1.89B. Biogen revealed the positive result of BIIB059 in the Phase 2 LILAC study for cutaneous lupus erythematosus and systemic lupus erythematosus.
Immunology Segment Revenue: $0.81B
Founded Year: 2000
Market Cap: ~$88.14
Total Employees: ~99,000
Headquarter: Brentford, United Kingdom
Stock Exchange: LON
GlaxoSmithKline (GSK) is a global healthcare company serving the world with drugs, vaccines & consumer healthcare products. With only approved products, Benlysta, GSK has generated a revenue of $0.81B in 2019. In Jul 2019, GSK initiated the phase 3 study of otilimab for rheumatoid arthritis. In Sep’19, EMA granted a positive CHMP opinion for intravenous Benlysta in children with lupus and was approved in Oct 2019.
Immunology Segment Revenue: $1.11B
Founded Year: 2007
Market Cap: ~$34.6B
Total Employees: ~7,228
Headquarter: Osaka, Japan
Stock Exchange: TYO
Mitsubishi Tanabe is a Japanese pharma company focused on autoimmune diseases, diabetes and kidney diseases, neurological disorders, and vaccines. Mitsubishi has reported a total sale of $1.11B from its immunology segment in 2019.
Immunology Segment Revenue: $1.24B
Founded Year: 1891
Market Cap: ~$197.46B
Total Employees: ~71,000
Headquarter: New Jersey, United States
Stock Exchange: NYSE
Merck & Co. is a global health care company delivering innovative health care products with its Prescription medicines, Oncology drugs, Vaccines, Biologic therapies, and Animal Health care products. Merck has recorded the sale of $1.24B in 2019 from its five approved drugs in its immunology portfolio including Simponi, Remicade, Renflexis, Brenzys, Hadlima. Simponi and Remicade was co-commercialized by Merck and Johnson & Johnson and Simponi recorded the revenue of $0.83B in 2019.
Immunology Segment Revenue: $1.68B
Founded Year: 1991
Market Cap: ~$19.38B
Total Employees: ~1,300
Headquarter: Delaware, United States
Stock Exchange: NASDAQ
Incyte Corp is a global biopharmaceutical firm focused on developing therapies in two categories Oncology and Inflammation & Autoimmune. Incyte has generated a revenue of $1.68B from its immunological segment. In Jan’19, Incyte reports results of Itacitinib in GRAVITAS-301 P-III study for patients with treatment-naive acute graft-versus-host disease.
Immunology Segment Revenue: $1.79B
Founded Year: 2005
Market Cap: ~ $26.49B
Total Employees: ~15,883
Headquarter: Tokyo, Japan
Stock Exchange: TYO
Astellas Pharma is a Japanese multinational pharmaceutical company focused on the therapeutic fields of urology, immunology including transplantation and infectious diseases, oncology, neuroscience and DM complications, and metabolic diseases. Astellas has four drugs in its immunology portfolio including two approved drugs Smyraf And Prograf and has recorded the sale of $1.79B in 2019. In Jul’19, Astellas Pharma launched Smyraf 50 mg and 100 mg tablets for rheumatoid arthritis.
Immunology Segment Revenue: $1.79B
Founded Year: 1901
Market Cap: ~$135.16B
Total Employees: ~33,625
Headquarter: Indiana, United States
Stock Exchange: NYSE
Eli Lilly and Company is a global pharmaceutical firm focused on delivering therapies in two divisions Human Pharmaceutical Products and Animal Health products. The pharmaceutical portfolio offers products for Cardiovascular, Endocrinology, Immunology, Neuroscience, and Oncology. Eli Lilly has two approved drugs including Taltz and Olumiant. Lilly’s Taltz, an approved drug for plaque psoriasis or psoriatic arthritis has generated a revenue of $1.6B in 2019. In Apr’19, Eli Lilly signs research and licensing agreement with avidity biosciences to develop therapies in immunology.
Immunology Segment Revenue: $1.92B
Founded Year: 1928
Market Cap: ~$20.49
Total Employees: ~7,600
Headquarter: Brussels, Belgium
Stock Exchange: EBR
UCB is a global biopharmaceutical company focused on neurology, inflammatory, gastrointestinal and autoimmune disorders. UCB has recorded the sale of $1.92B in 2019 from its immunology segment with its only approved drug, Cimzia indicated for psoriatic arthritis (PsA). In Jul’19, Cimzia was approved by China’s NMPA.
Immunology Segment Revenue: $2.53B
Founded Year: 1973
Market Cap: ~$122.35B
Total Employees: ~100,000
Headquarter: Paris, France
Stock Exchange: EPA
Sanofi is a global healthcare leader in vaccines providing healthcare solutions in 170+ countries around the world. Sanofi is ranked third in the global market and first in EU and Latin America. Sanofi has four drugs in its immunology portfolio including one approved drug Kevzara, developed in partnership with Regeneron. In Dec’19, Sanofi presented the positive result from its pivoted phase 3 study of sutimlimab for cold agglutinin disease. Additionally, Sanofi restructured its agreement with Regeneron to obtain worldwide rights for Kevzara.
Immunology Segment Revenue: $2.97B
Founded Year: 1887
Market Cap: ~$135.30B
Total Employees: ~30,000
Headquarter: New York, United States
Stock Exchange: NYSE
Bristol-Myers Squibb is an American pharmaceutical company focused on Oncology, Cardiovascular, Immuno-Science, and Fibrosis. BMS has two approved drugs Orencia and Nulojix. BMS’ Orencia is a protein indicated to treat adult rheumatoid arthritis, juvenile idiopathic arthritis, and adult psoriatic arthritis has generated the highest revenue of $2.97B in 2019. The acquisition of Celgene in 2019, has boosted up BMS’ Immunology pipeline.
Immunology Segment Revenue: $3.66B
Founded Year: 1925
Market Cap: ~$52.60B
Total Employees: ~49,578
Headquarter: Osaka, Japan
Stock Exchange: TYO
Takeda is a global biopharma company focused on Oncology, Gastroenterology (GI), Neuroscience, Immunology, and Rare Diseases. With three approved drugs including Immunoglobulin, Albumin, and Entyvio, Takeda has generated a $3.66B sale in 2019. In Apr’19, EMA accepted the application for a subcutaneous formulation of Entyvio in Crohn’s disease, and in Oct 2019, Takeda acquired CNP-101 from COUR Pharmaceuticals. In Feb’20, Takeda acquired PvP Biologics to strengthen its immunology pipeline. Additionally, Takeda got approval for Entyvio from China’s NMPA for Crohn’s disease in Mar 2020.
Immunology Segment Revenue: $4.22B
Founded Year: 1996
Market Cap: ~$205.93B
Total Employees: ~109,000
Headquarter: Basel, Switzerland
Stock Exchange: SIX Swiss Exchange, NYSE
Novartis is a multinational group of companies specializing in research, development, manufacturing, and marketing with a broad range of healthcare solutions including generic and ophthalmic therapies. The company is focused on Immunology, Hepatology, Dermatology, Oncology, Neurology, and Ophthalmology. Novartis has the uppermost number of immunology drugs with eight approved products including ACZ885/Ilaris, AIN457/Cosentyx, Myfortic (Renal transplant), Neoral, Simulect, and Zortress. Novartis’ Cosentyx (secukinumab) used to treat Psoriasis, ankylosing spondylitis and psoriatic arthritis have generated global sales of $3.55B in 2019. In Apr’19 Novartis acquired IFM Tre to enhance its immunologic portfolio with its NLRP3 inhibitors for $1.5B.
Immunology Segment Revenue: $4.73B
Founded Year: 1849
Market Cap: ~$206.05B
Total Employees: ~83,000
Headquarter: New York, United States
Stock Exchange: NYSE
Pfizer is a research-based, global biopharmaceutical company having a vast portfolio including Oncology, Medicines, vaccines, and other health care products for the prevention & treatment of untreated diseases. With 3 approved drugs including Xeljanz, Enbrel (outside the US and Canada), and Inflectra (Biosimilar), Pfizer has generated $4.73B sale from its immunology portfolio indicated for rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, Behcet’s disease, and ulcerative colitis. Pfizer’s Xeljanz has generated revenue of $2.24B in 2019. In Jan’2019, CytoReason signed a research partnership with Pfizer to develop drugs using CytoReason’s cell-centered models of the immune system.
Immunology Segment Revenue: $5.39B
Founded Year: 1980
Market Cap: ~$134.11B
Total Employees: ~23,400
Headquarter: California, United States
Stock Exchange: NASDAQ
Amgen is one of the leading biotechnology company developing novel therapies focused on cardiology, oncology, neurology, nephrology, and inflammatory diseases. Amgen has generated a total sale of $5.39B in 2019 with its drugs Otezla, Avsola, Enbrel, Nplate, and Prolia. Amgen’s Otezla used to treat certain types of psoriasis and psoriatic arthritis has generated sales of $1.6B.
Immunology Segment Revenue: $8.79B
Founded Year: 1896
Market Cap: ~$281.59B
Total Employees: ~98,000
Headquarter: Basel, Switzerland
Stock Exchange: SWX
Roche Holding AG is a Swiss multinational healthcare company that operates worldwide under two divisions: Pharmaceuticals and Diagnostics. The immunology department focus on rheumatoid arthritis (RA), systemic juvenile idiopathic arthritis, polyarticular juvenile idiopathic arthritis, and giant cell arteritis including severe persistent allergic asthma (AA), chronic idiopathic urticaria (CIU), and idiopathic pulmonary fibrosis (IPF) with 3 approved drugs including Actemra, CellCept and Mabthera and has generated the sale of $8.97B in 2019. In Dec’2019, Roche signed an exclusive global option and license agreement with Rheos Medicines to develop and commercialize therapies for immune metabolism.
Immunology Segment Revenue: $13.95B
Founded Year: 1887
Market Cap: ~$378.94B
Total Employees: ~132,100
Headquarter: New Jersey, United States
Stock Exchange: NYSE
Johnson & Johnson (J&J) is an American multinational healthcare company focused on the development and commercialization of pharmaceutical, medical device, and consumer packaged products. The pharmaceutical portfolio offers products for Cardiovascular, Endocrinology, Immunology, Neuroscience, and Oncology. J&J has generated $13.95B from its Immunology portfolio with 5 approved products including Remicade, Simponi, Stelara, Tremfya, Simponi Aria. Remicade was jointly marketed by J&J and Merck and has generated a revenue of $4.38B in 2019. In Nov 2019, J&J’s Tremfya meets the primary endpoint in the phase 3 study for Psoriatic Arthritis. Additionally, J&J submit two applications with the USFDA for Polyarticular Juvenile Idiopathic Arthritis and Juvenile Psoriatic Arthritis. FDA approved Tremfya for Psoriatic Arthritis and Simponi Aria for polyarticular juvenile idiopathic arthritis and active psoriatic arthritis for patients 2 years of age and older in Jul 2020.
Immunology Segment Revenue: $19.57B
Founded Year: 2012
Market Cap: ~$146.29B
Total Employees: ~30,000
Headquarter: Illinois, United States
Stock Exchange: NYSE
AbbVie is a global, research and development-based biopharmaceutical company focused on developing innovative advanced therapies. The company is focused on developing products in immunology, oncology, virology, and neuroscience, dermatology. AbbVie has generated the sale of $19.57B in 2019 from its immunological segment with 3 approved drugs including RINVOQ, SKYRIZI, HUMIRA. AbbVie’s blockbuster drug HUMIRA recorded a revenue of $19.16B. In Apr’2019, AbbVie received EMA approval of SKYRIZI for Plaque Psoriasis. Its RA drug RINVOQ received FDA approval in Aug 2019 and EMA approval in Dec 2019. Additionally, RINVOQ achieved positive results in primary and key secondary endpoints for Psoriatic Arthritis and subsequently submitted the regulatory applications with the FDA and EMA.
The P-lll GALACTIC-HF study involves the assessing of Omecamtiv mecarbil (25mg, bid with the maintenance dose of 50 mg, 37.5mg, or 25mg, bid) vs PBO in 8,256 patients with HFrEF
Result: The study met its 1EPs of reduction in CV death or HF events (HF hospitalization and other urgent treatment for HF) while the study did not meet its 2EPs of reduction in time to CV death, presented at AHA 2020
Omecamtiv mecarbil is a cardiac myosin activator, targeting the contractile mechanisms of the heart, being developed under a collaboration between Amgen and Cytokinetics, with funding and strategic support from Servier
Click here to read full press release/ article | Ref: Amgen | Image: The Times
Shares in Amgen were down nearly 7% after close of trading yesterday after the company’s heart failure drug omecamtiv mecarbil disappointed in a large phase 3 trial.
Take a look at Amgen’s portfolio and it becomes apparent how important this potential new drug is: the company is relying on its ageing inflammatory diseases drug Enbrel for a large chunk of its revenue.
It is also hoping to steal market share from rivals with biosimilars, which are comparatively cheaper versions of biologic drugs.
Psoriasis drug Otezla is also bringing in the bucks – but Amgen paid $13.4 billion for this after Celgene was forced to sell it as a condition of its merger with Bristol-Myers Squibb.
Amgen looks in need of fresh home-grown revenues, but investors don’t think these will come from omecamtiv mecarbil, based on the findings of the phase 3 GALACTIC-HF trial.
The trial met its primary endpoint by demonstrating a statistically significant effect to reduce cardiovascular death or heart failure events compared with placebo in patients treated with standard care.
But there was no reduction in the secondary endpoint measuring only cardiovascular death in the trial, which with 8,256 patients with reduced ejection fraction in 35 countries is one of the largest phase 3 cardiovascular outcomes studies in heart failure ever conducted.
Adverse events, including major ischemic cardiac events, were balanced between treatment arms, Amgen said.
With such a large cohort of subjects, Amgen and partners Cytokinetics and Servier have nowhere to hide – they cannot claim the trial was not powered to produce a result against this important secondary endpoint measuring the impact on mortality.
Merck & Co and Bayer are trying to develop a rival heart failure drug in patients with reduced ejection fraction, vericiguat, but that has also produced similar results, scoring against a composite measure of deaths and hospitalisation but failing to outperform placebo at reducing deaths.
Developed by Cytokinetics, omecamtiv mecarbil is a cardiac myosin activator that works by increasing interactions between filaments in the heart muscle to improve its pumping.
Amgen has been working on the drug with Cytokinetics since 2006, and Servier bought European rights in 2013.
The company did not provide data, but said results for patients who received sotorasib at the 960mg dose were consistent with those seen in the Phase I portion of the Phase I/II study, which showed an overall response rate of 35.3%. ……
The P-ll CodeBreaK 100 study involves assessing of Sotorasib (proposed INN for AMG 510, 960mg) in 126 patients with KRAS G12C-mutant advanced NSCLC, whose cancer had progressed despite prior treatment with CT and/or immunotherapy
Result: ORR is consistent with previously reported P-I study while other measures of efficacy including DoR are promising and 50%+ of the responders were still on treatment and continuing to respond as of the data cutoff date whereas safety & tolerability is similar to previous P-I data
Detailed results of the P-II study will be submitted to the IASLC 2020 World Congress on Lung Cancer while the company has begun recruiting in P-III CodeBreaK 200 study evaluating Sotorasib vs docetaxel in KRAS G12C-mutant NSCLC patients
Click here to read full press release/ article | Ref: Amgen | Image: Reuters
The OLE P-II study involves assessing of Aimovig monthly (70mg) vs PBO in 383 eligible adult patients with an episodic migraine for 12wks. 250 patients increasing their dosage to 140mg monthly after a protocol amendment to assess the long-term safety of the higher dose
The results of the OLE study showed the therapy helped patients to achieve 5.3days reductions in MMD and 4.4 days reduction in the use of AMSM, and has the longest duration of safety and efficacy trial data for any anti-CGRP pathway therapy, will be presented at the Migraine Trust Virtual Symposium
Amgen will also present additional studies that include interim results of the LIBERTY OLE study, as well as efficacy and safety results of Aimovig in the EMPOwER study. Aimovig is the first and only FDA and EMA-approved migraine preventive treatment targeting CGRP receptor
Click here to read full press release/ article | Ref: Amgen | Image: CNBC
Three of the companies making drugs used in the Phase II I-SPY COVID-19 study – Amgen, AbbVie and Takeda – announced the patient enrollments Monday. The study, which will enroll up to 1,500 critically ill patients, could test around 10 drugs.
The average life expectancy span of Human Beings are increased due to better medical facilities and drugs developed by Biopharma companies. Pharmaceutical products or drugs or medicines are being produced for a wide range of medical sectors. It includes the lifesaving drugs or the major therapy area including immunology, cardiology, and neurology but are they not limited to only these indications and are rapidly increasing with the increasing medical needs. The drugs are developed targeting with the motive to cure, vaccinate, and alleviate the symptoms.
We have compiled a list of global top 20 drugs blockbuster prescription drugs based on their sales for last year i.e. 2019. The top position was maintained by AbbVie’s blockbuster drug Humira with $19.16B another drug grasped the second position headed by Merck’s Keytruda with $11.08B following the third was occupied by BMS’s Revlimid with $9.37B while ended at the low end by Gilead’s Truvada.
If you have any questions or see something we might have missed? Please reach out to Senior Editor, Shiwani Sharma by email.
Product – Truvada
First Approved – US (Aug 02, 2004), EU (Feb 20, 2005)
Indications Approved – HIV-1
Company – Gilead Sciences
Total Revenue – $2.81B
Truvada is a combination of tenofovir disiproxil fumarate (tenofovir DF) and emtricitabine used for HIV treatment and pre-exposure prophylaxis for PrEP (pre-exposure prophylaxis) that can help reduce the risk of getting HIV-1 through sex. On July 23, 2019, Gilead presented new findings on the profile of Descoy for potential use as HIV Pre-exposure Prophylaxis compared with Truvada. Descovy and Truvada are only 2 FDA-approved pills for PrEP.
First Approved– US (Jan 31, 2002), EU (Aug 22, 2002)
Indications Approved – Febrile Neutropenia
Neulasta is a PEGylated form of the recombinant human granulocyte colony-stimulating factor analogue filgrastim. Neulasta has been steadily losing market share due to the launch of multiple biosimilars and the utilization of Neulasta’s OnPro on-body injector (the dominant player at > 55%) has been relatively steady.
Product– Victoza Company– Novo Nordisk
First Approved– US (Jan 25, 2010), EU (Jun 30, 2009) Total Revenue– $3.29B
Indications Approved – Glycemic control in type 2 diabetes mellitus patients, Reduce adverse cardiovascular events in adults with type 2 diabetes mellitus
Victoza or liraglutide belongs to a class of drugs called glucagon-like peptide-1 agonists (GLP-1). Recently Novo Nordisk’s patent on Victoza is been reviewed as Mylan has claimed that its invalid because it covers an obvious invention. Mylan has challenged the Victoza patent with an aim to launch the low-cost copy of the drug.
Product– Lyrica Company– Pfizer
First Approved– US (Dec 30, 2004), EU (Jul 05, 2004) Total Revenue– $3.32 B
Lyrica is an anticonvulsant or an anti-seizure drug that can treat a range of conditions, including epilepsy, fibromyalgia, and nerve pain. The recent update on May 28, 2019, Pfizer announced that its epilepsy drug Lyrica (pregabalin), failed to meet the primary endpoint in P-III study, assessing it as adjunctive therapy in epilepsy patients (aged 5 to 65 years) with primary generalized tonic-clonic (“PGTC”) seizures.
Product– Imbruvica Company– Johnson & Johnson
First Approved– US (Nov 13, 2013), EU (Nov 21, 2014) Total Revenue– $3.41B
Imbruvica is an oral therapy which inhibits Bruton’s tyrosine kinase (BTK) and has received its 10 FDA approval. In Apr 2020, US FDA approved Imbruvica (ibrutinib) plus Rituximab for the treatment of patients with Chronic Lymphocytic Leukemia (CLL)
Product– Genvoya Company – Gilead Sciences
First Approved – US (Nov 05, 2015), EU (Nov 19, 2015) Total Revenue– $3.93 B
Indications Approved – HIV-1
Genvoya is a combination of elvitegravir, cobicistat, emtricitabine, and tenofovir alafenamide targeting HIV. In Aug 2018, The CNDA approved Gilead’s Genvoya (elvitegravir 150 mg/cobicistat 150 mg/emtricitabine 200 mg/tenofovir alafenamide 10 mg or E/C/F/TAF) for treating HIV Infection.
Product – Remicade Company – Johnson & Johnson
First Approved– US (Aug 24, 1998), EU (Aug 13, 1999) Total Revenue– $4.38B
Remicade or infliximab is a tumour necrosis factor (TNF-alpha or TNF-α) blocker and a chimeric monoclonal IgG1 antibody. In June 2019, Janssen’s Remicade was issued a Civil Investigative Demand to Johnson & Johnson by FTC for investigating whether its contracting practices violate federal antitrust.
Product – Ibrance Company – Pfizer
First Approved– US (Feb 03, 2015), EU (Nov 09, 2016) Total Revenue– $4.96B
Indications Approved – HER2 Negative Advanced Breast Cancer
Ibrance PO is CDKs 4 and 6 inhibitors indicated for HR+, HER2- advance or mBC and has been prescribed up xto 160,000 patients with approval in 85 countries worldwide. On May 29, 2020, the Data Monitoring Committee (DMC) of the collaborative P-III early breast cancer PALbociclib CoLlaborative Adjuvant Study (PALLAS) determined that the trial failed in the primary endpoint of invasive disease-free survival (iDFS). Eli Lilly’s Verzenio has been chasing Ibrance in the metastatic setting since it hit the market and it now its ahead of Ibrance which recently took a big blow.
Product – Enbrel Company – Amgen
First Approved– US (Nov 02, 1998), EU (Feb 02, 2000) Total Revenue– $5.22B
Enbrel is a TNF inhibitor drug that treats autoimmune diseases by interfering with tumour necrosis factor by acting as a TNF inhibitor. Enbrel was the drug of choice for multiple autoimmune indications. On Jul 01, 2020, the US Court of Appeals for the Federal Circuit held in Amgen’s favour on the validity of 2 patents that describe and claim Enbrel’s methods.
Product – Prevnar 13 Company – Pfizer
First Approved– US (Feb 24, 2010), EU (Dec 12, 2009) Total Revenue– $5.84B
Indications Approved – S. Pneumoniae Infection
Prevnar 13 or Pneumococcal 13-valent Conjugate Vaccine [Diphtheria CRM197 Protein is a vaccine approved for adults 18 years of age and older for the prevention of pneumococcal pneumonia and invasive disease caused by 13 Streptococcus pneumonia strains. In June 2020, Pfizer started four P-III clinical trials for investigational vaccines including Prevnar 13.
Product – Herceptin Company – Roche
First Approved – US (Sep 25, 1998), EU (Aug 28, 2000) Total Revenue – $6.23 B
Indications Approved – Metastatic Breast Cancer, Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma
Herceptin or trastuzumab is a mAb that binds to HER2 receptors on the surface of HER2-positive tumor cells, blocking them from receiving growth signals and flagging them for destruction by the immune system. It is on the WHO’s List of Essential Medicines, the safest and most effective medicines needed in a health system. On 29 June 2020, Roche got the approval of Phesgo, a fixed-dose combination of Perjeta (pertuzumab) and Herceptin with hyaluronidase, administered by SC injection in combination with IV chemotherapy, for the treatment of early and metastatic HER2-positive breast cancer. This is the first time that Roche has combined two mAbs that can be administered by a single SC injection.
Product – Stelara Company – Johnson & Johnson
First Approved – US (Sep 25, 2009), EU (Jan 15, 2009) Total Revenue – $6.36B
Stelara or ustekinumab is a mAb with a novel mechanism of action that targets the p40 subunit of cytokines interleukin-12 (IL-12) and interleukin-23 (IL-23). In Apr 2020, Johnson & Johnson received NICE’s positive Final Appraisal Document (FAD) recommending Stelara (ustekinumab) for the treatment of ulcerative colitis.
Product – Rituxan Company – Roche
First Approved – US (Nov 26, 1997), EU (Jun 02, 1998) Total Revenue – $6.69B
Rituxan or rituximab is a mAb used to target cancer cells with CD20 markers in patients. In Sep 2019, The US FDA approved Roche’s Rituxan (rituximab) in combination with glucocorticoids for treating granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) disorders.
Product – Opdivo Company – Bristol-Myers Squibb
First Approved – US (Dec 22, 2014), EU (Jun 19, 2015) Total Revenue – $7.20B
Indications Approved – Metastatic Melanoma, Non-Small Cell Lung Cancer,Melanoma, Small Cell Lung Cancer, Renal Cell Carcinoma, Hodgkin Lymphoma, Squamous Cell Carcinoma of the Head and Neck, Urothelial Carcinoma, Mismatch Repair Deficient Metastatic Colorectal Cancer, Hepatocellular Carcinoma, Esophageal Squamous Cell Carcinoma
Opdivo or nivolumab is a PD-1 immune checkpoint inhibitor targeted for cancer cells and is approved in 65 countries including the US, EU, Japan & China. In Jun 2020, the US FDA granted approval for Opdivo (nivolumab) following the P-III ATTRACTION-3 study results evaluate it in comparison with taxane CT (docetaxel/paclitaxel) to treat patients with unresectable advanced, recurrent or ESCC, refractory/ intolerant to at least one prior fluoropyrimidine & platinum-based CT.
Product – Avastin Company – Roche
First Approved – US (Feb 26, 2004), EU (Jan 12, 2005) Total Revenue – $7.30B
Avastin or bevacizumab is a tumour-starving (anti-angiogenic) therapy targeted for preventing tumour growth. In Jun 2020, Roche received the US FDA’s approval for a combination of Avastin (bevacizumab) + Tecentriq (atezolizumab) to treat patients with Unresectable Or Metastatic Hepatocellular Carcinoma (HCC) who have not received prior systemic therapy, which lead to a novel immunotherapy approval for the indication. The approval followed the P-III IMbrave150 study results assessing the combination.
Product – Eylea Company – Regeneron Pharmaceuticals
First Approved – US (Nov 18, 2011), EU (Nov 21, 2012) Total Revenue – $7.54B
Eylea or aflibercept is one form of anti-VEGF therapy administered by injection into the eye. Recently, Bayer launched Eylea (aflibercept) pre-filled syringe in all 27 states of the EU including the UK, Iceland, Norway, and Liechtenstein in Apr 2020. In May 2019, Regeneron’s Eylea injection received the US FDA’s approval to treat all stages of Diabetic Retinopathy (DR) further reducing the risk of blindness.
Product – Eliquis Company – Bristol Myers Squibb
First Approved – US (Dec 28, 2012), EU (May 18, 2011) Total Revenue – $7.92B
Indications Approved – Stroke, Systemic Embolism, Deep Vein Thrombosis, Pulmonary Embolism, Recurrence of DVT and PE
Eliquis or apixaban is an anticoagulant that reduces blood clotting. Eliquis stood on the fourth-highest selling product with $7.92B in 2019. In late 2019, the US FDA approved BMS’ Eliquis tablets to reduce the risk of stroke and systemic embolism in patients with nonvalvular atrial fibrillation.
Product – Revlimid Company – Bristol-Myers Squibb
First Approved – US (Dec 27, 2005), EU (Jun 14, 2007) Total Revenue – $9.37B
Revlimid or lenalidomide is a thalidomide analogue candidate which reported WW sales of $9.37B. In Feb 2020, BMS’ Revlimid received the National Institute for Health and Care Excellence (NICE) approved it targeting to treat previously treated follicular lymphoma in combination with MabThera (rituximab).
Product – Keytruda Company – Merck & Co
First Approved – US (Sep 04, 2014), EU (Jul 17, 2015) Total Revenue – $11.08B
Keytruda or pembrolizumab is a mAb serving as an anti-PD-1 therapy for the tumour cells blocking the interaction between PD-1 and its ligands, PD-L1, and PD-L2. It initially received FDA accelerated approval for refractory, advanced melanoma in September 2014. Subsequently, it has received approval for the treatment of many other oncologic conditions, and many more are currently in clinical development.
Keytruda acquired the second position in the WW sales list of 2019. In Jul 2020, the US FDA granted Priority Review to Merck’s Keytruda for its sBLA to treat patients with 2L+ Relapsed or Refractory Classical Hodgkin Lymphoma (cHL). The designation follows P-III KEYNOTE-204 study results and the expected PDUFA date as Oct 30, 2020. In Jun 2020, Merck’s Keytruda receives the US FDA’s approval to treat patients with 1L Unresectable or Metastatic MSI-H or dMMr Colorectal Cancer.
Product Name – Humira Company Name – AbbVie
First Approved – US (Dec 31, 2002), EU (Aug 09, 2003) Total Revenue – $19.16B
Humira (adalimumab) is a mAb blocking TNF-a protein targeted for the inflammatory response of immune-mediated diseases. Humira has recorded the highest selling product sales with a generated revenue of $19.16B in 2019. Humira continues to be the leader of the commercial drug market and keeping its first position with the crown of the most lucrative drug in the history of the pharmaceutical market. In Jun 2020, AbbVie reported results of ABBV-3373, an ADC comprising novel glucocorticoid receptor modulator (GRM) vs Humira in patients with Moderate to Severe Rheumatoid Arthritis and resulted in similar profile with the original product.