Incyte Reports the US FDA’s Acceptance of sNDA for Priority Review of Jakafi (ruxolitinib) to Treat Chronic Graft Versus Host Disease

Shots:

  • The sNDA submission is based on the P-III REACH3 study assessing ruxolitinib vs best available therapy in adult and pediatric patients ≥12yrs. with steroid-refractory chronic GVHD. The findings were recently presented at the 62nd ASH Annual Meeting & Exposition
  • The study showed greater ORR @24wks. i.e 1EPs (49.7% vs 25.6%); @24wks. mFFS (not reached vs 5.7 mos.); greater symptom improvement per mLSS (24.2% vs 1.0%); no new safety signals were observed while AEs were consistent
  • The PR shortens the review period from 10 to 6mos. The anticipated PDUFA date is Jun 22, 2021

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: The Business Journals

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Pfizer Signs a License and Research Agreement with Imcyse to Develop Imotopes for Rheumatoid Arthritis

Shots:

  • Imcyse to receive up front, ~$180M as milestones along with royalties. Pfizer to purchase an equity stake in Imcyse
  • Initially, the companies will collaborate to develop existing lead candidates and further optimize potential molecules. Later, Pfizer will lead clinical development and commercialization activities for the program
  • The Imotope platform is a curative approach to severe autoimmune diseases. Imcyse plan to initiate a P-II study with its lead candidate in T1D and MS in 2022

Click here ­to­ read full press release/ article | Ref: Accesswire | Image: Imcyse

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Janssen Reports CHMP’s Acceptance for Accelerated Assessment of Cilta-cel’s MAA to Treat Patients with Heavily Pretreated Multiple Myeloma

Shots:

  • The MAA submission is based on a P-Ib/II CARTITUDE-1 study assessing the safety and efficacy of cilta-cel in adults with r/r MM. The company is expected to submit MAA to EMA in H1’21
  • The accelerated approval milestone in EU follows the rolling submission for cilta-cel to the US FDA in Dec’2020
  • Cilta-cel is an investigational BCMA-directed CAR-T therapy in development for the treatment of adults with r/r MM. CHMP’s accelerated assessment can reduce the review timelines to evaluate an MAA

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Businesswire

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Shionogi Concludes a License Agreement with BioAge for BGE-175 to Control the Aggravation of COVID-19

Shots:

  • Shionogi to receive an up front, milestones as well as royalties on sales of the therapy. BioAge to get exclusive US and EU rights to develop and commercialize BGE-17 for COVID-19
  • Additionally, BioAge gets an exclusive right to negotiate a license for additional indications and plans to initiate P-II study of BGE-175 for COVID-19 in the H1’21
  • BGE-175 is a DP1 receptor antagonist, developed for allergic rhinitis. The results of multiple non-clinical/ clinical trials in 2,400+ subjects & have shown that BGE-175 has high affinity and selectivity to the DP1 receptor1 & is well tolerated & safe

Click here ­to­ read full press release/ article | Ref: Shionogi | Image: BusinessWire

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Incyte Reports Acceptance and Priority Review of BLA for Retifanlimab to Treat Patients with Squamous Cell Carcinoma of the Anal Canal (SCAC)

Shots:

  • The BLA submission is based on P-II POD1UM-202 trial involves assessing of retifanlimab (500 mg, q4w) in 94 patients with locally advanced or metastatic SCAC who have progressed on or are intolerant of standard platinum-based CT
  • The study demonstrates 14% ORR for retifanlimab monotherapy as determined by ICR using RECIST v1.1 and mDOR of 9.5mos
  • Retifanlimab has received FDA’s ODD for the treatment of anal cancer along with PR which shortens the review period by 4mos vs Standard Review. The PDUFA target action date is Jul 25, 2020

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Technical.ly

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Aurinia Lupkynis (voclosporin) Receives US FDA’s Approval to Treat Adult Patients with Active Lupus Nephritis

Shots:

  • The approval is based on the pivotal AURORA P-III study and AURA-LV P-II study involves assessing of Lupkynis + SoC in 533 patients to treat adult patients with LN
  • The study demonstrated significantly improved renal response rates vs SoC, improved response rates in all parameters across immunologically-active classes, 50 % reduction in UPCR twice as fast as SoC, complete renal response @24 wks vs SoC @1year
  • Lupkynis is the 1st FDA-approved oral therapy for LN and is now commercially available in the US

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Aurinia Pharmaceuticals

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MTPA and Aquestive Sign a License and Supply Agreement for Exservan (riluzole) to Treat ALS in the US

Shots:

  • Aquestive will receive upfront, milestones, royalties on net sales for Exservan (oral film formulation) in the US and will earn revenue pursuant to the exclusive supply agreement
  • MTPA to commercialize Exservan in the US while Aquestive will serve as the exclusive sole manufacturer and supplier for the product
  • Exservan is an oral film formulation of riluzole, expected to be available in the mid of 2021

Click here ­to­ read full press release/ article | Ref: MTPA | Image: PR Newswire

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ViiV’s Cabenuva (cabotegravir and rilpivirine) Receives the US FDA’s Approval as the First and Only Complete Long-Acting Regimen for HIV treatment

Shots:

  • The approval is based on pivotal phase III ATLAS and FLAIR study assessing Cabenuva in 1,100+ HIV-1 adults to replace the current ARV regimen in those who were virologically suppressed (HIV-1 RNA less than 50 copies/mL) on a stable regimen
  • Prior to initiating treatment of Cabenuva, oral dosing of cabotegravir and rilpivirine should be administered for ~1mos. to assess the tolerability of each therapy. The therapy reduces the treatment dosing days from 365 days to 12days/ yr
  • The company will begin shipping of Cabenuva to wholesalers and specialty distributors in the US in Feb’2021

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: ViiV Healthcare

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Servier and MiNA Therapeutics Collaborate to Develop saRNA Therapies for Neurological Diseases

Shots:

  • MiNA to receive $267.6M in up front, exclusivity fee on first neurological target and will be eligible to receive research, development, regulatory and commercial milestones along with royalties
  • Servier will lead pre/ clinical development of lead candidates and will have the rights to commercialize any products that emerges from the collaboration
  • The collaboration integrates Servier’s CNS expertise and MiNA’s small activating RNA technology to identify new potential treatments for restoring normal cell function in neurological disorders

Click here ­to­ read full press release/ article | Ref: Businesswire| Image: Nohat

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Bayer and Merck’s Verquvo (vericiguat) Receives the US FDA’s Approval to Treat Chronic Heart Failure

Shots:

  • The approval is based on pivotal P-III VICTORIA trial involves assessing of Verquvo (2.5mg, 5mg & 10mg) vs PBO in 5,050 adult patients with symptomatic CHF and LVEF less than 45%, following a worsening HF event
  • The study met the primary efficacy objective based on a time-to-event analysis & showed a 4.2% reduction in annualized absolute risk. The 1EPs is time to the first event of CV death or hospitalization for HF @median follow-up of 11 mos.
  • Verquvo is the first soluble guanylate cyclase stimulator, approved to treat HF

Click here ­to­ read full press release/ article | Ref: Business Wire| Image: Financial Times

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Regenxbio Reports the Initiation of First Pivotal Program for RGX-314 to Treat Wet AMD

Shots:

  • Regenxbio has reported that the ATMOSPHERE trial evaluating RGX-314 is active and patient screening is ongoing. The study will enroll 300 patients across two RGX-314 dose arms vs ranibizumab, with anticipated initiation of patients dosing in Q1’21
  • The second trial is expected to be similar in design to ATMOSPHERE with its expected initiation in H1’21. The company expects to submit a BLA based on these trials in 2024
  • The company has completed enrollment of patients in Cohort 1 of the P-II AAVIATE study for wet AMD. Moreover, Regenxbio reported a new program RGX-202 for DMD with its expected IND filing in mid-2021

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: BioHealth Capital Region

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Pfizer and OPKO Reports the US FDA’s Acceptance of BLA for Somatrogon to Treat Pediatric Patients with Growth Hormone Deficiency

Shots:

  • The BLA submission is based on P-lll study assessing the safety and efficacy of Somatrogon (0.66 mg/kg, qw) vs Somatropin (0.034 mg/kg, qd) in 224 pediatric patients in the ratio of (1:1) with GHD
  • The study met its 1EPs of non-inferiority as measured by annual height velocity @12mos.; least square mean (10.12 vs 9.78 cm/year); the treatment difference in height velocity was 0.33 cm/yr
  • The anticipated PDUFA date is Oct 2021 and is approved, the therapy is served as a once-weekly treatment option

Click here ­to­ read full press release/ article | Ref: Business Wire | Image: The Wire

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Chi-Med Initiates Rolling Submission of NDA to the US FDA of Surufatinib to Treat Advanced Neuroendocrine Tumors

Shots:

  • Chi-Med initiated the filing of NDA to the US FDA for surufatinib to treat pancreatic & non-pancreatic NET & plans to complete the NDA submission in the H1’21
  • The NDA is based on two P-III studies that demonstrated a reduction in risk of progression or death by 67% & 51%, extending PFS of non-pancreatic NET & pancreatic NET patients with an acceptable risk/benefit ratio
  • Surufatinib is an oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with VEGFR and FGFR. The US FDA has granted FTD to surufatinib for pancreatic and non-pancreatic NET in Apr’2020 and ODD for pancreatic NET in Nov’2019

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: PMLive

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Novartis’s Entresto Receives the US FDA’s Advisory Committee Recommendation to Treat Patients with HFpEF

Shots:

  • The US FDA’s CRDAC voted 12 to 1 supporting the use of Entresto (sacubitril/valsartan) in the treatment of patients with HFpEF
  • The decision was based on efficacy & safety analyses, including findings from a pre-specified subgroup analysis of PARAGON-HF (P-III study in HFpEF) and additional evidence from PARAMOUNT (P-II trial in HFpEF), as well as PARADIGM-HF (P-III trial in HFrEF)
  • Entresto (sacubitril/valsartan) is a prescription medicine used to reduce the risk of CV death & heart failure hospitalization in people with long-lasting heart failure

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: BioSpace

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Richter Acquires Janssen’s Evra Transdermal Contraceptive Patch Assets for $263.5M

Shots:

  • Richter signed an asset purchase agreement with Janssen for Evra transdermal contraceptive patch assets outside the US. The total deal value is $263.5M
  • The companies also signed a transitional business license agreement and a series of other related agreements to run the business without interruption during the period required to transfer the marketing authorizations to Richter
  • The acquisition will strengthen Richter’s position in women’s healthcare and boost its existing female healthcare franchise globally. Evra (qw) is the first transdermal hormonal patch to be approved, as well as the first non-invasive form of birth control that, when used correctly, is 99% effective

Click here ­to­ read full press release/ article | Ref: Richter | Image: Richter

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Shionogi Receives MHLW’s Approval for Xofluza’s sNDA to Treat Post-Exposure Prophylaxis of Influenza Virus Infection

Shots:

  • The approval is based on P-III BLOCKSTONE study assessing Xofluza (baloxavir marboxil, 10/20mg) vs PBO in patients in household members aged ≥12yrs. who was living with someone with an influenza infection confirmed by a rapid influenza diagnostic test
  • Result: The proportion of household members who developed influenza (1.9% vs 13.6%); well tolerated with no new safety signals identified
  • Xofluza inhibits the cap-dependent endonuclease in the PA protein and is a single-dose oral treatment for influenza which is different from all other currently available antiviral treatments

Click here ­to­ read full press release/ article | Ref: Shionogi | Image: Businesswire

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Merck KGaA Reports EMA’s Validation of MAA for Tepotinib to Treat Advanced NSCLC with METex14 Skipping Alterations

Shots:

  • The MAA is based on a P-II VISION study assessing Tepotinib as monothx. in patients with advanced NSCLC of METex14 skipping alterations, prospectively assessed by liquid biopsy or tissue biopsy
  • With the validation, the application is complete, and the EMA will now initiate the review procedure.
  • Tepotinib is an oral MET inhibitor that inhibits the oncogenic MET receptor signaling caused by MET alteration. The FDA is reviewing the application under PR and through the Real-Time Oncology Review pilot program

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: FIerce Pharma

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Henlius Report the NMPA’s Acceptance of HLX15 (biosimilar, Daratumumab) to Treat Multiple Myeloma

Shots:

  • The NMPA has accepted HLX15’s IND to be used in the treatment of multiple myeloma. HLX15 is Henlius’ second self-developed product around blood tumor treatment
  • The company evaluated the biosimilar in a head to head clinical studies demonstrating that HLX15 is highly similar to its reference daratumumab while the safety profiles are also similar
  • The company has developed the HLX15 in accordance with the technical guidelines of development and evaluation of biosimilar drugs and EMA guideline on similar biological medicinal products

Click here ­to­ read full press release/ article | Ref: Henlius | Image: Henlius

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Novavax Receives the US FDA’s Fast Track Designation for NVX-CoV2373 to Treat COVID-19

Shots:

  • The US FDA has granted FT designation to NVX-CoV2373 which is currently in late-phase clinical development. Novavax expects to initiate pivotal P-III study in the US & Mexico by the end of Nov’2020
  • The company’s ongoing P-lll trial in the UK to evaluate the efficacy, safety, and immunogenicity of NVX-CoV2373 is expected to be fully enrolled by the end of Nov. Depending on the overall COVID-19 attack rate, interim data in the UK trial is expected in Q1’21
  • NVXCoV2373 is a stable, prefusion protein made using Novavax’ nanoparticle technology and includes its proprietary MatrixM adjuvant

Click here ­to­ read full press release/ article | Ref: Novavax | Image: Newsroom Keck Medicine of USC

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Medtronic’s Abre Venous Stent Receives the US FDA’s Approval to Treat Venous Outflow Obstruction

Shots:

  • The approval is based on ABRE clinical study assessing the Abre stent in 200 patients with iliofemoral venous outflow obstruction across the spectrum of deep venous obstruction including those with the post-thrombotic syndrome, NIVL & those who presented with an aDVT. The study also includes a challenging patient population, 44% of whom required stents that extended below the inguinal ligament into the CFV
  • The study resulted in meeting its 1EP of safety with a 2% rate of MAEs within 30 days and also met its 1EPs of efficacy with an overall primary patency rate of 88.0%, no stent fractures, and no stent migrations were reported in the study.
  • Abre venous self-expanding stent system is the device, indicated for use in the iliofemoral veins in patients with symptomatic iliofemoral venous outflow obstruction and has received CE Mark approval in April’2017

Click here ­to­ read full press release/ article | Ref: Medtronic | Image: elEconomista.es

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Henlius Signs a License Agreement with Essex to Co-develop HLX04 (biosimilar, bevacizumab) for Ophthalmic Diseases

Shots:

  • Henlius to receive upfront, regulatory, and commercial milestone payments of ~$43M, eligible to receive $30M for every $1B above the first $600M of the cumulative net sales along with 10% royalties on net sales of the therapy
  • Henlius will be responsible for the pre/ clinical development of HLX04, whereas Essex is responsible for regulatory filings and commercialization of HLX04 globally and will be responsible for 80% of all costs and expenses concerning to the development of HLX04. Additionally, Essex has a right to grant a sublicense to third parties, for which Henlius will receive 20% of the related sublicensing revenue
  • Henlius is developing HLX04 for ophthalmic diseases, wAMD & DR. Henlius and Essex plan to conduct global clinical trials of HLX04 in China, Australia, EU & the US, & will apply MAA in different countries across the globe

Click here ­to­ read full press release/ article | Ref: Henlius | Image: BioSpectrum Asia

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Novartis’ Leqvio (inclisiran) Receives CHMP’s Positive Opinion for Approval to Treat Hypercholesterolemia

Shots:

  • The CHMP’s positive opinion is based on ORION program including P-III studies assessing Leqvio in 3,600+ patients on a maximally tolerated statin dose. Inclisiran demonstrated effective and sustained LDL-C reduction of up to 52% with 2doses/yr, after an initial dose and one @3mos., in adults with ASCVD, ASCVD risk equivalent or HeFH6
  • 80% of high-risk patients do not reach guideline-recommended LDC-L, targets despite the widespread use of statins. Reduction in LDL-C was achieved through 17mos. with a safety and tolerability profile like PBO
  • Additional post hoc analysis demonstrated 88% of them reached guideline-recommended targets at any timepoint during the study. Additionally, the therapy is also under review by the US FDA and other health authorities for primary hyperlipidemia in adults who have elevated LDL-C while being on a maximally tolerated dose of statin therapy

Click here ­to­ read full press release/ article | Ref: Novartis | Image: Easy Health Options

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Janssen Reports the NDA Submission to the US FDA for Uptravi (selexipag, IV) to Treat Pulmonary Arterial Hypertension

Shots:  

  • The NDA submission is based on P-III UPTRAVI IV study he safety and tolerability of 20 patients with PAH temporarily switching from oral UPTRAVI to UPTRAVI IV, and then transitioning back to the initial oral dose
  • Results demonstrated that Uptravi IV is suitable to maintain continuous dosing for short periods of time when the oral formulation is not feasible. Both the formulation was well tolerated with no unexpected safety findings
  • Uptravi is a selective, prostacyclin IP receptor agonist, approved in IV formulation for PAH, WHO Group I in adults with WHO functional class (FC) II–III, who are currently prescribed oral Uptravi but are temporarily unable to take oral therapy

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: Penn Today

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MC2 Therapeutics Wynzora Cream Receives the US FDA’s Approval to Treat Plaque Psoriasis in Adults

  • The approval is based on P-III trial assessing Wynzora Cream vs Taclonex Topical Suspension in 794 patients for the treatment of plaque psoriasis in adults 18 years of age or older
  • The study resulted in a PGA treatment of 21.6% vs 14.6%, patients achieved at least a 4-point improvement in the peak pruritus NRS score at @4wks. 60.3% vs 21.4%
  • Wynzora is a novel topical solution offering high efficacy, favorable safety and excellent and is filled for MAA application in EU

Click here ­to­ read full press release/ article | Ref: MC2 Therapeutics | Image: MC2 Therapeutics

SK bioscience Collaborate with Novavax to Supply Antigen for COVID-19 Vaccine

Shots:

  • SK bioscience signs a development and supply agreement for Novavax’ NVX-CoV2373 to supply it globally including the COVAX facility. SK bioscience will manufacture the vaccine antigen component for use in the final drug product
  • SK bioscience will utilize its cell culture and recombinant protein capability, will initiate the production of the NVX-CoV2373 antigen at its facility in Andong L-house, South Korea, expected to begin in Aug’2020
  • Additionally, the companies have signed a letter of intent with the Republic of Korea’s Ministry of Health and Welfare for equitable access to NVX-CoV2373 in global market and to make it available in South Korea

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: Novavax

Gilead Reports NDA Submission to the US FDA for Veklury (Remdesivir) to Treat COVID-19

Shots:

  • The NDA filing is based on P-III studies evaluating Veklury vs PBO, conducted by NIAID. The studies demonstrated that Veklury led to faster time to recovery and that a 5-day or 10-day treatment duration led to similar clinical improvement
  • Across multiple studies, Veklury was generally well-tolerated in both the 5-day and 10-day treatment groups, with no new safety signals identified
  • Veklury (remdesivir) is an investigational nucleotide analog with broad-spectrum antiviral activity both in vitro/ in vivo in animal models against multiple emerging viral pathogens. The therapy has been approved by multiple regulatory authorities across the globe, including the EU and Japan

Click here ­to­ read full press release/ article | Ref: Gilead  | Image: The Wire Science

Lupin and SunPharma Launch Generic Favipiravir to Treat Mild to Moderate COVID-19 in India

Shots:

  • Lupin has launched favipiravir in India under the brand name ‘Covihalt’ for the treatment of mild to moderate COVID-19
  • Covihalt will be available as 200mg tablets in the form of a strip of 10 tablets, priced at $0.65 (₹49/tablet) and has received DCGI authorization for emergency use
  • Additionally, SunPharma has launched antiviral drug favipiravir under the brand name ‘FluGuard’ to treat mild to moderate cases of COVID-19, at ₹35/tablet in India

Lupin, SunPharma ­to­ read full press release/ article  | Image: StraitTimes




LEO Pharma Receives the US FDA’s Fast Track Designation for Delgocitinib Cream to Treat Moderate-to-Severe Chronic Hand Eczema

Shots:

  • The US FDA has granted FT designation to LEO’s delgocitinib cream as the potential treatment for adults with moderate-to-severe CHE. There are currently no treatment options available in the US specifically developed and approved for CHE
  • The FDA’s FT designation facilitates the development and expedites the regulatory review of drugs to treat serious conditions and demonstrate the potential to address an unmet medical need
  • Delgocitinib (bid, topical formulation) acts by inhibiting the activation of the JAK-STAT pathway, currently being evaluated in two P-IIb studies for mild-to-severe CHE & AD. The company plans to submit the results of P-IIb CHE study in late 2020

Click here ­to­ read full press release/ article | Ref: LEO Pharma | Image: LEO Pharma




AstraZeneca Signs a License Agreement with Redx Pharma for RXC006 to Treat Idiopathic Pulmonary Fibrosis

Shots:

  • Redx to receive $17M in early payment by the time of successful commencement of a P-I study, $360M as development, a regulatory and commercial milestone along with royalties on sales of the RXC006
  • AstraZeneca to get an exclusive global license to further develop & commercialize RXC006 targeting fibrotic diseases including IPF
  • Redx continues to progress its lead oncology asset, RXC004 (porcupine inhibitor) targeting Wnt-driven tumors, currently being evaluated in P-I/II clinical trial, and RXC007, the oral Rock 2 inhibitor targeting fibrosis where first in human studies are expected in 2021

Click here ­to­ read full press release/ article | Ref: AstraZeneca | Image: SoPosted




UCB Canada’s Brivlera (brivaracetam) Receives Health Canada Approval to Treat Partial-Onset Seizures in Pediatric Epilepsy Patients

Shots:

  • The approval is based on the principle of extrapolation of its efficacy data in adults also supported by pharmacokinetic and safety data in children. Additionally, the safety and tolerability data of Brivlera in children 4 years and older were similar to observed in adults
  • The usage of Brivlera in pediatric and adolescent patients is supported by the placebo-controlled partial-onset seizure studies in adults, PK study in pediatric aged 4 to 17 years of and for the achievement of similar plasma concentrations as of adults weight-based dose adaptations were practiced
  • Brivlera is an anti-epileptic drug (AED) displays a high and selective affinity for synaptic vesicle protein 2A (SV2A) developing anticonvulsant effects, approved by Health Canada as adjunctive therapy for partial-onset seizures in patients 4 years of age and older

Click here to read full press release/ article | Ref: Newswire | Image: Twitter




Merck’s MK-6482 Receives the US FDA’s Breakthrough Therapy Designation to Treat Patients With Von Hippel-Lindau Disease-Associated Renal Cell Carcinoma

Shots:

  • The designation is based on P-II study evaluating MK-6482 in patients with VHL-associated RCC with nonmetastatic RCC tumors >3cms in size, unless immediate surgery is required
  • The FDA’s BT designation is granted to expedite the development and review of medicines that are intended to treat serious or life-threatening conditions and have demonstrated preliminary clinical evidence indicating that the medicine may provide a substantial improvement over available therapy on at least one clinically significant endpoint
  • MK-6482 (formerly PT2977) is an investigational oral HIF-2α inhibitor, currently being evaluated in a P-III  trial in advanced RCC (NCT04195750), a P-II trial in VHL-associated RCC (NCT03401788), and a P-I/II dose-escalation and dose-expansion trial in advanced solid tumors, including advanced RCC (NCT02974738)

Click here ­to­ read full press release/ article | Ref: Merck | Image: Market Watch




BioMarin Reports the Submission of MAA to EMA for Vosoritide to Treat Children with Achondroplasia

Shots:

  • The MAA is based on P-III study assessing the efficacy and safety of vosoritide, further supported by the long-term safety and efficacy from the ongoing P-II and P-III extension studies and extensive natural history data
  • Following the completion of EMA’s validation, BioMarin expects the initiation of the MAA review to commence in Aug’2020. The company is planning to file NDA to the US FDA in Q3’20
  • Vosoritide is an investigational, once-daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia and has received FDA’s and EMA’s ODD

Click here to read full press release/ article | Ref: BioMarin | Image: BioMarin




Daiichi Sankyo Signs a Research Agreement Gustave Roussy for DS-1062 and Patritumab Deruxtecan to Treat Lung and Breast Cancer

Shots:

  • Gustave Roussy to receive funding and support from Daiichi Sankyo for integrative research program including clinical, translational, and preclinical studies for DS-1062 and patritumab deruxtecan in lung and breast cancer respectively
  • The research program includes two P-II studies evaluating the efficacy, safety, and markers of response and resistance to DS-1062 & patritumab deruxtecan in patients with advanced NSCLC, progressed on anti-PD-1/PD-L1 therapy and Pt-doublet CT & HER3 expressing mBC respectively
  • Each study will enroll ~100 patients and will be conducted at several sites in France. The 1EPs of the studies will be ORR and 2EPs includes clinical benefit rate, PFS, DoR, OS, and safety measures. DS-1062 is a TROP2 directed DXd ADC while patritumab deruxtecan is a HER3 directed DXd ADC

Click here to read full press release/ article | Ref: Daiichi Sankyo| Image: ETHealthworld




Roche Collaborates with Jnana Therapeutics to Discover Novel Therapies to Treat Immune-Mediated and Neurological Diseases

Shots:

  • Jnana to receive $40M upfront payment in cash and is eligible to get ~$1B as research funding, preclinical, development, and commercialization milestone along with royalties on sales of the therapies
  • The partners will work on the discovery and preclinical development of therapies targeting key regulators of cellular metabolism to treat across immunology and neuroscience while Roche will further develop and commercialize the therapies exclusively
  • The collaboration leverages Jnana’s RAPID platform designed to overcome the challenges of directly targeting SLC transporters. The RAPID platform is a cell-based platform that can be used to screen small molecule libraries to identify novel modulators of SLC transporter

Click here to read full press release/ article | Ref: Businesswire | Image: Twitter




Eli Lilly Reports Results of Mirikizumab in P-III OASIS-2 Study to Treat Moderate to Severe Plaque Psoriasis

Shots:

  • The P-III OASIS-2 study involves assessing mirikizumab vs PBO & Cosentyx (secukinumab) in 1,465 patients with mod. to sev. PsO. The patients were randomized in a (4:4:4:1) ratio to one of the following induction and maintenance period treatments: mirikizumab (250mg) @ 0, 4, 8, 12wks. followed by 250 & 125mg, q8w starting @16wks; 300mg secukinumab @ 0, 1, 2, 3, 4wks. followed by 300mg, q4w starting @4wks.; PBO @ 0, 4, 8, 12 wks. followed by 250mg mirikizumab q4w starting @16wks. through 32wks. followed by q8w thereafter
  • Results: @16wks. sPGA(0,1) (79.7% vs 6.3% & 76.3%), PASI 90 (74.4% vs 6.3% & 72.8%); PASI 100 (37.7% vs 1.8% & 36.6%). In general, the study resulted in meeting its 1EPs and all key 2EPs vs PBO @16wks. and all key 2EPs vs Cosentyx (secukinumab) @16wks. (non-inferiority) and @52wks. (superiority)
  • Mirikizumab is an IgG4 mAb binding p19 subunit of IL-23 and being evaluated for immune diseases, including psoriasis, UC and CD

Click here to read full press release/ article | Ref: Eli Lilly | Image: Glassdoor




Kiniksa’s Rilonacept Receives the US FDA’s Orphan Drug Designation to Treat Pericarditis

Shots:

  • The US FDA has granted ODD for Kiniksa’s Rilonacept to treat pericarditis, which includes recurrent pericarditis. The company plans to submit sBLA in recurrent pericarditis to the US FDA in late 2020
  • Earlier, the company has reported the results of P-III RHAPSODY study that met its all 1EPs & 2EPs showing that the therapy improved clinical outcomes associated with the unmet medical need in recurrent pericarditis
  • Rilonacept (SC, qw) is a recombinant fusion protein that blocks IL-1α and IL-1β signaling and has received the US FDA’s BT designation in 2019. In 2017, Regeneron out-licensed rilonacept to Kiniksa for recurrent pericarditis under which Regeneron will receive equal profit on sales of the therapy, if approved in the US

Click here to read full press release/ article | Ref: Kiniksa | Image: Kiniksa




MorphoSys Reports Submission of Tafasitamab’s BLA to the US FDA to Treat Relapsed or Refractory Diffuse Large B Cell Lymphoma (r/r DLBCL)

Shots:

  • The BLA submission is based on L-MIND P-II trial data results assessing Tafasitamab + lenalidomide in patients with r/r DLBCL and retrospective observational matched control cohort Re-MIND evaluating efficacy outcomes of r/r DLBCL patients who received Tafasitamab + lenalidomide vs lenalidomide monothx
  • Re-MIND has met its 1EPs and has shown improved ORR of the tafasitamab/lenalidomide combination vs lenalidomide monothx. In Oct 2017, Tafasitamab + Lenalidomide received the US FDA’s Breakthrough Therapy Designation
  • Tafasitamab (MOR208) is an investigational Fc-engineered mAb directed against CD19 and is being evaluated in B-MIND P-III trial in combination with bendamustine vs rituximab + bendamustine in patients with r/r DLBCL

Click here to read full press release/ article | Ref: MorphoSys | Image: Behance

Acceleron Signs a License Agreement with Fulcrum for the Identification of Therapies to Treat Pulmonary Diseases

Shots:

  • Fulcrum to receive $10M upfront, reimbursement for R&D costs, research, development and commercial milestone of up to $295M for first product commercialized and additional payments $143.5M for all subsequent products commercialized plus royalties on sales of product
  • Acceleron to get rights to access Fulcrum’s product engine and target identification platform for identifying small molecule therapies controlling the expression of genes impacting specific pathways for pulmonary diseases and will take care of all development and commercial activities for developed products
  • The collaboration combines Fulcrum’s skill in identifying drug targets based on modulation of genetic pathways associated with disease and Acceleron’s deep expertise in TGF-beta for the generation of targeted therapies. Acceleron’s ACE-083 is a P-II locally acting myostatin+ agent targeted for Charcot-Marie-Tooth disease

Click here to read full press release/ article | Ref: Acceleron | Image: Signbox

Eli Lilly Opens its P-III LIBRETTO-531 Clinical Trial for LOXO-292 (selpercatinib) to Treat RET-Mutant Medullary Thyroid Cancer (MTC) Patients

Shots:

  • The second P-III LIBRETTO-531 involves assessing of selpercatinib vs physician’s choice of cabozantinib or vandetanib in 400 patients in ratio (2:1) with advanced or metastatic RET-mutant MTC who have received no prior systemic therapy for metastatic disease
  • The study will have efficacy endpoints as progression-free survival (PFS), treatment failure-free survival (TFFS), overall survival (OS), overall response rate (ORR), and duration of response (DoR)
  • LOXO-292 is an oral investigational new medicine targeted to inhibit native RET signaling as well as anticipated acquired resistance mechanisms and has also received Breakthrough Therapy designation in RET fusion-positive NSCLC, RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancers

Click here to read full press release/ article | Ref: PRNewswire | Image: Twitter