Atopic dermatitis patients now have a new option that comes as a topical cream. The FDA approved Incyte’s drug, Opzelura, as a treatment for the inflammatory skin condition, but the regulatory nod comes with a black box warning on the label that broadly covers the entire class of JAK inhibitors.
Nantes, France, Naobios, a CDMO (Contract Development and Manufacturing Organization) providing bioprocess development and GMP production of clinical batches of viral vaccines BSL2/BSL3, oncolytic viruses and viral vectors, today announces the next phase in its partnership with FluGen, Inc., a clinical-stage vaccine company transforming vaccine efficacy in infectious respiratory diseases. Naobios will initiate the GMP …
Shots: The P-I/II dose expansion studies evaluate the safety & preliminary anti-tumor efficacy of VT1021 as monothx. in patients with dose-escalation & expansion cohorts with rGBM & PC. The results of both studies are expected in Q4’21 In the rGBM expansion cohort, the therapy showed a complete tumor regression & PR, patients remain on trial …
Shots: The P-III COSMIC-311 trial evaluates Cabometyx (60mg, qd) vs PBO in a ratio (2:1) in 258 patients aged ≥12yrs. with previously treated radioactive iodine-refractory DTC across 164 sites globally An updated analysis showed ORR (11% vs 0%) as assessed by BIRC in ITT population including 1 CR, m-OS (19.4mos.) for Cabometyx but not estimable …
The Seagen and Genmab antibody drug conjugate (ADC), named Tivdak, is approved to treat cases of cervical cancer that have returned or spread following treatment with chemotherapy. Tivdak is Seagen’s third ADC; for Genmab, the drug will become its first commercialized product.
The FDA has approved the first biosimilar that references the blockbuster Roche drug Lucentis. The Biogen and Samsung BioLogics joint venture that developed the biologic product have approval to treat three eye conditions that lead to vision loss.
A lower-dose version of Pfizer and BioNTech’s Covid-19 vaccine showed safety and efficacy results in children 5 to 11 that were comparable to those in an older age group. With these preliminary data, the companies said they plan to seek emergency use authorization of the shots.
Shots: The P-III CASPIAN trial evaluates Imfinzi + CT or Imfinzi + CT + tremelimumab vs CT alone in 805 patients with ES-SCLC in 200+ centers across 23 countries, including the US, EU, South America, Asia & Middle East The results showed OS benefits & sustained efficacy after a median follow-up of ≥3yrs. with a …
Shots: The P-III KEYNOTE-826 trial evaluates Merck’s Keytruda (200mg, IV, q3w for up to 35 cycles) + Pt-based CT vs PBO + CT with/out bevacizumab in 617 patients aged b/w 22- 82yrs. with persistent, recurrent, or metastatic cervical cancer The results showed a 33% reduction in the risk of death; m-OS (24.4 vs 16.5mos.), m-PFS …
Shots: The P-III EMPOWER-Lung 3 study evaluates Libtayo (350mg, q3w for 108wks.) + Pt doublet CT vs CT alone in a ratio (2:1) in 466 patients with LA/mNSCLC irrespective of histology & across all PD-L1 expression levels, 84% had an ECOG 1 performance status The trial met its 1EPs & 2EPs i.e., improvement in OS …
Shots: The P-I/II KRYSTAL–1 study evaluates adagrasib (600mg, bid) as monothx. and in combination with cetuximab in patients with heavily pretreated colorectal cancer harboring a KRASG12C mutation following prior systemic therapy The therapy showed the clinical activity and broad disease control. As of May 25 & July 9, 2021, 80% of patients received adagrasib mononthx. …
An FDA advisory panel voted down a proposal for booster shots of the Pfizer/BioNTech Covid-19 in the general population, opting instead to support third shots in certain high-risk groups, such as the elderly. Panelists want to see more information supporting the need for a booster, as well as data about a heart safety risk the …
FDA panel backs booster shots in high-risk groups after rejecting broader proposal Read More »
Rune Labs developed software that aggregates brain data, providing insights for clinical care and clinical research. Health systems are starting to adopt the technology, and the startup has now raised $22.8 million to grow that part of the business while also expanding use of the technology in drug development.
Shots: The P-III JUPITER-06 trial evaluates toripalimab + CT vs PBO + CT in a ratio (1:1) in 514 treatment-naive patients with advanced or metastatic ESCC The study met its co-primary 1EPs i.e., improvements in PFS & OS compared to CT alone. At a prespecified interim analysis on Mar 22, 2021 @median follow-up of 7.4 …
Shots: The P-II CONVEY withdrawal study evaluates efficacy & safety of vixotrigine (200/350 mg, bid) vs PBO in 265 patients with SFN for 12wks. The study met its 1EPs i.e., 200mg dose showed a reduction in mean ADP & worst daily pain score @12wks. while 350mg dose did not meet its 1EPs of mean change …
Shots: The companies collaborated to evaluate the synergistic effects of BI 1701963 (SOS1::pan-KRAS inhibitor) + Lumakras (KRAS G12C inhibitor) in a P-I trial for adult patients with LA or metastatic NSCLC The companies will jointly share costs & manage the clinical development for combined therapy. Amgen will sponsor the trial The preclinical data showed that …
Takeda Pharmaceutical drug mobocertinib was approved by the FDA to treat non-small cell lung cancer in patients whose tumors carry a rare genetic signature. With the approval, Takeda can offer an oral alternative to a recently approved Johnson & Johnson drug that targets the same mutation but is given as an infusion.
Shots: The P-III CheckMate -214 trial evaluates Opdivo (3mg/kg) + Yervoy (1mg/kg, q3w for 4 doses) vs sunitinib in 1096 patients with previously untreated advanced/mRCC Results: @median follow-up of 67.7mos., combination showed superior OS in intermediate, poor risk patients & randomized or ITT population; m-OS (47.0 vs 26.6mos.) & (55.7 vs 38.4mos.); ORR (42% vs …
Shots: The P-Ib/II CodeBreaK 101 study evaluates Lumakras + Vectibix in 31 patients with KRAS G12C-mutated advanced CRC The results demonstrated ORR (27%) among 26 patients in the efficacy analysis, DCR (81%), 33% of patients experienced a response in the expansion cohort, no patients experienced dose-limiting toxicities during the 28 days following initial treatment. The …
Shots: The companies collaborated to evaluate Hookipa’s HB-200 (arenaviral immunotherapeutic) + Merck’s Keytruda (anti-PD-1 therapy) in a P-II trial for patients with advanced HNSCC, based on an ongoing P-I/II trial of HB-200 for advanced HPV16+ cancers patients HB-201 monothx. showed an ORR (18%), m-PFS (3.45mos.) in heavily pretreated head & neck cancer patients. Preliminary data …
After closing a series C round led by 8VC, Flywheel used a portion of the funds to acquire St. Louis-based Radiologics. Both have platforms to help researchers process medical imaging data.
Dice Therapeutics raised $204 million from its IPO to support development of oral drugs that could compete against biologic drugs that are injected or infused. Also debuting on the public markets were Tyra Biosciences and Procept BioRobotics.
What You Should Know: Global digital wound care provider Swift Medical, today announced the launch of a new application specifically designed to help pharma and med device researchers conduct decentralized clinical trials. Since patients are now doing more trials at home, study coordinators have had trouble tracking skin side effects remotely — or trusting patients …
Swift Medical Launches AI Digital Wound App to Support Clinical Trials Read More »
The clinical trial failure of Theravance Biopharma’s most advanced internal program, a drug for a rare blood pressure disorder, is leading to a companywide restructuring. Going forward, the biotech will focus on developing drugs for respiratory disorders.
Shots: The companies collaborated to evaluate IO’s IO102-IO103 + MSD’s Keytruda vs Keytruda alone in the P-III trial for patients with previously untreated unresectable or MM. Biomarker studies will also be conducted while IO will sponsor the trial & MSD will provide Keytruda The P-I/II trial of combination therapy in 30 patients with the same …
Shots: The P-II/III study evaluates opaganib +SoC vs PBO + SoC in a ratio (1:1) in 475 patients with COVID-19 pneumonia requiring hospitalization & treatment with supplemental oxygen The preliminary data showed that the study did not meet its 1EPs while efficacy EPs showed consistent trends. The therapy demonstrated good tolerability with balanced AEs & …
The death occurred less than two weeks after Astellas Pharma reported that the patient developed liver problems after being dosed with the experimental gene therapy for a rare neuromuscular disorder. Astellas said the cause of death is still under investigation, and the FDA has placed a clinical hold on the study.
Such treatments are extraordinarily promising and costly. Will the Biden administration commit to spending that could speed clinical trial results?
Such treatments are extraordinarily promising and costly. Will the Biden administration commit to spending that could speed clinical trial results?
Shots: The P-III CHOICE-01 trial evaluates toripalimab + CT vs CT alone in 465 patients in a ratio (2:1) with advanced sq./nonsq. NSCLC without driver mutations The interim analysis met its 1EPs i.e., improvement in PFS, superior ORR & DoR, OS data are still maturing. The safety profile was consistent with PD-1 inhibitor while final …
What You Should Know: – Medidata, a Dassault Systèmes company, today announced a first-of-its-kind partnership with Labcorp using the Medidata Sensor Cloud to introduce a new model to advance the use of medical-grade sensors in clinical trials and accelerate biomarker discovery. – As part of the partnership, Medidata will receive and process medical-grade sensor data within drug, vaccine …
AbbVie will take the lead on commercializing Regenxbio’s gene therapy in wet AMD and diabetic retinopathy. It’s AbbVie’s second major gene therapy deal this year, coming months after it committed $90 million to preclinical-stage Capsida Biotherapeutics.
Shots: The P-III CheckMate -743 trial evaluates Opdivo (3mg/kg, q2w) + Yervoy (1mg/kg, q6w) vs CT in 605 patients with previously untreated unresectable MPM. The results will be presented at ESMO 2021 The result showed an improvement in OS with a reduction in risk of death, patients were alive (23% vs 15%) @3yrs., improvement in …
Shots: The P-III POSEIDON trial evaluates Imfinzi (1500mg, q3w, FD, for 4 cycles) + Pt-based CT or Imfinzi + Tremelimumab + CT vs CT alone in 1013 patients with mNSCLC The results showed an improvement in OS & PFS, 28% reduction in risk of cancer progressing or death with an m-PFS (6.2 vs 4.8mos.), 23% …
Acknowledging the demand for a Covid-19 vaccine for children, the FDA said it expects regulatory review could take weeks, not months, depending on the timing of a submission from vaccine developers. U.S. health officials told Reuters they expect the Pfizer/BioNTech vaccine will be the first one ready for an FDA submission.
The FDA issued a warning letter to Kaleido Biosciences alleging the company tested its experimental microbiome product in Covid-19 patients without submitting a formal clinical trial application. Kaleido claims the filing was not required because the product is a medical food, but the regulator doesn’t buy that argument.
Shots: The P-III study evaluates Byooviz (0.5 mg, IVT) vs ranibizumab in 634 patients with nAMD. The results will be presented at EURETINA 2021 The 1EPs are changed from baseline in BCVA @8wks. and change in CST @4wks. with both EPs, followed by 52wks. The post-hoc analysis showed that the baseline age, BCVA, CST, and …
Shots: The first patient has been dosed in a P-III program that consists of P-III ADORING 1/2 and long-term extension ADORING 3 study evaluates the safety and efficacy of tapinarof (1% dosed, qd) vs vehicle-controlled cream in patients aged ≥2yrs. with AD The 1EPs of ADORING 1/2 studies will be the proportion of patients achieving …
Shots: The P-Ib/II trial evaluates the safety and preliminary efficacy of onvansertib + Folfiri and Avastin in 2L treatment of patients with KRAS-mutated mCRC As of July 2, 2021, 42% patients achieved a partial response at RP2D (15 mg/m2 dose); confirmed PR (37%); ORR (5-13%); mPFS has not yet reached. Additionally, 38% patients achieved an …
Shots: The P-III MANDALA & DENALI trial evaluates the efficacy & safety of PT027 (180/160mcg & 180/80mcg doses) vs albuterol & albuterol and budesonide aged ≥4yrs. in 3132 & 1001 patients with asthma Both trials met its 1EPs i.e., reductions in risk of severe exacerbations in the MANDALA trial, improvement in lung function as measured …
Shots: The P-III PEGASUS trial evaluates pemphigus (BTK inhibitor) vs PBO in 131 patients with newly diagnosed or relapsing mod. to sev. pemphigus in 19 countries globally The trial did not meet its 1EPs or 2EPs i.e., complete remission from 29 to 37wks. with minimal doses of CS (≤10/mg day). The safety profile remained consistent …
Shots: The companies initiate P-III SANOVO study to evaluates the efficacy & safety of Orpathys in combination with AstraZeneca’s Tagrisso (irreversible EGFR TKI) vs Tagrisso alone in patients with NSCLC whose tumors harbor EGFR mutation and overexpress MET. The first patient was dosed on Sept 7, 2021 The 1EPs of the study is m-PFS as …
Shots: The P-IIIb PONENTE study evaluates the safety & efficacy of Fasenra (30mg, SC, q8w) in adult patients with sev. eosinophilic asthma on high-dose ICS + LABA & long-term use of OCS therapy with/out additional asthma controlled Results: 97% of patients maintained a low OCS dose of ≤5mg across blood eosinophil counts @6mos., 3% of …
Shots: The BREEZE study evaluates Tyvaso DPI vs nebulized Tyvaso in 51 patients with PAH who were transitioned from Tyvaso to Tyvaso DPI at a corresponding treprostinil dose The results demonstrated safety and tolerance with improvements in 6MWD compared to baseline, improvements in overall satisfaction, PAH Impact & PROs. Additionally, 96% completed 3wks. treatment phase …
Prescriptions for ivermectin have jumped to more than 88,000 per week, some pharmacists are reporting shortages and people are overdosing on forms of the drug meant for horses.
Unleashed from restrictive norms for recruiting and interacting with patients, researchers are finding ways to bring more people into trials by simply making enrollment and participation accessible beyond restraints like geography and availability during “business hours.”
Shots: The first patient has been enrolled at the first site in P-II MSC-COV-202BR efficacy study evaluates COVI-MSC vs PBO in 100 patients with COVID-19-induced ARD or ARDS in Brazil The primary objective of the study is to evaluate the efficacy of COVI-MSCs in patients with COVID-19-induced ARD or ARDS Additionally, the company plans to …
Researchers ended a large trial in South Africa after finding that an experimental vaccine offered little protection.
Shots: The P-III NAVIGATOR study involves assessing Tezepelumab + SOC vs PBO +SOC in adults (18–80yrs.old) and adolescents (12–17yrs. old) with severe, uncontrolled asthma. The study showed improvements in lung function and nasal polyp symptoms The pre-specified exploratory analysis evaluated the effect of tezepelumab in NAVIGATOR patients with/ out reported nasal polyps (NP+ or NP−) …
Shots: The P-IIb PAGANINI study involves assessing the efficacy, safety & tolerability of eliapixant (25, 75 or 150mg, bid) vs PBO in 310 patients with RCC for 12wks. and it lasted for 18wks. in total The study met its 1EPs i.e. reduction in the 24hrs. cough count of up to 27% with 75mg dose and …
Shots: The FDA’s clinical hold is based on interim safety findings from a pre-clinical study which showed that 6/7 animals treated with BMN 307 (2e14 Vg/kg) had tumors on liver necropsy @52wks. after dosing with evidence for integration of portions of AAV vector into the genome No lesions were observed in any mice @24wks. 5 …
BioMarin Reports FDA’s Hold on P-I/II Phearless Trial of BMN 307 in Adults with PKU Read More »
Shots: The discontinuation is based on the interim analysis of data from the CIRRUS-1 study demonstrating that CFZ533 (iscalimab)-based treatment is less efficacious compared to tacrolimus-based treatment in the prevention of organ rejection in patients receiving a kidney transplant In the study, both CFZ533 and tacrolimus were used in combination with other immunosuppressive therapies (induction …
The agency that regulates drug safety has become too cozy with the industry it’s supposed to oversee.
The agency that regulates drug safety has become too cozy with the industry it’s supposed to oversee.
Clinical trials have repeatedly failed to show that the drug is effective against the virus. Still, U.S. demand is high.
Clinical trials have repeatedly failed to show that the drug is effective against the virus. Still, U.S. demand is high.
Clinical trials have repeatedly failed to show that the drug is effective against the virus. Still, U.S. demand is high.
The drug has spared hundreds of millions of Africans from serious parasites, but clinical trials have repeatedly failed to show that it is effective against Covid. Still, U.S. demand is high.
The FDA improved an Impel NeuroPharma migraine treatment, a nasal spray intended to bring pain relief more quickly than drugs administered in other ways. It marks the first approval for the Seattle company, which has developed a proprietary nasal delivery technology.
Tools like eConsent provides organizations with an efficient and streamlined way to garner consent from study participants. Removing this barrier can help improve engagement and deeper the pool of potential participants.
Shots: The P-II clinical trial evaluates FB-401 vs PBO in patients with AD The trial did not meet its 1EPs of EASI-50 (50% improvement in AD disease severity as measured by EASI) Additionally, positive trends were identified in key 2EPs i.e., improvement in disease severity @16wks., patients achieving the EASI-90 EPs (27.6% vs 20.5%) while …
Shots: The company initiates a P-III RENOIR trial to evaluate RSVpreF vs PBO in 30,000 adults aged ≥60yrs. with RSV The 1EPs of the study is to evaluate the safety & efficacy for the prevention of mod. to sev. LRTI during the first RSV season In Nov’18, the vaccine has received FTD from FDA for …
A Roche drug that failed as a treatment for neurological disorders is now the lead program for Disc Medicines. CEO John Quisel said Roche’s clinical data showed the small molecule’s promise addressing a rare blood disorder and now the biotech startup has $90 million to advance that drug and another one into Phase 2 testing.
The company rolled out a tool to help pharmaceutical companies pull together information about clinical trial sites and identify the best physician investigators for a trial.
The agency that regulates drug safety has become too cozy with the industry it’s supposed to oversee.
Shots: Astellas discontinue the dosing in the ASPIRO trial that evaluates AT132 in 24 participants (7 at the 1.3×1014 vg/kg dose and 17 at the 3.5×1014 vg/kg dose) with XLMTM. The participant has a history of intermittent cholestasis The discontinuation is due to serious AEs in a study that demonstrated abnormal LFTs in patients who …
Shots: The P-III PANTHER trial evaluates pevonedistat + azacitidine vs azacitidine alone in patients with higher-risk MDS, CMML and AML The study did not achieve its pre-defined 1EPs of EFS, the safety profile was consistent with previously reported data of the combination regimen Pevonedistat is an NAE inhibitor that leads to cancer cell death by …
The FDA’s review of a post-marketing study for Pfizer blockbuster anti-inflammatory drug Xeljanz found a higher risk of cardiovascular problems and cancer, and the agency is requiring additional warnings flagging those risks. The updated warnings extend to AbbVie and Eli Lilly medicines in the same drug class.
Jai Singh, SVP of Growth at Reify Health Clinical trials were front and center in 2020, as pharma companies raced to develop and prove the safety and efficacy of their COVID-19 vaccines. Despite this increased awareness, many potential participants and referring clinicians remain unaware of available trials and how to become involved. This negatively impacts …
Clinical Trials: 4 Strategies to Maximize Patients Referrals Read More »
Shots: The company initiates a P-III MOVe-AHEAD trial to evaluate molnupiravir (800 mg, q12h for 5 days) vs PBO in ~1,332 participants aged 18yrs. who reside in the same household as someone with laboratory-confirmed SARS-CoV-2 infection with symptoms. The trial is being conducted globally The 1EPs of trial i.e., proportion of participants with COVID-19 (laboratory-confirmed) …
Five months after raising $55 million to back a new technology and a promising lead cancer immunotherapy, Asher Biotherapeutics has reeled in $108 million more. CEO Craig Gibbs said investors were enticed by encouraging new data suggesting Asher Bio’s lead program is superior to a competitor’s.
Shots: The company has decided to discontinue the P-IIb Imbokodo, POC efficacy study that evaluates HIV vaccine regimen in ~2600 young women across five countries in sub-Saharan Africa The results showed that the HIV vaccine regimen did not provide sufficient protection against HIV infection. The vaccine was found to be safe with no serious AEs …
The use of consistent terminology within and between studies is critical to enabling an efficient trial process. CDISC standards such as CDASH, SDTM, SEND and ADaM standardize the structures to use when collecting and submitting data, but what about the data values themselves? This article gives an overview of CDISC’s standardized controlled terminology and how it …
Using NCI controlled terminology for standardizing data Read More »
Shots: The company initiates P-III OASIS program to evaluate the efficacy and safety of elinzanetant (120mg, qd) in 1300 patients with vasomotor symptoms during menopause at 200+ centers across 20+ countries The design and dosing of the P-III clinical development program is based on the results from two P-II studies i.e., P-Ib/IIa RELENT-1 & P-IIb …
Shots: The P-II Lauriet study evaluates the safety & efficacy of semorinemab (anti-tau mAb) vs PBO in 272 adult patients with AD across 43 centers globally. The therapy is being developed by Genentech The trial met its one co-1EPs i.e 43.6% reduction in cognitive decline from baseline as measured by ADAS-Cog11 @49wks. The 2EPs were …
Johnson & Johnson reported results from a mid-stage study in southern Africa showing just 25% efficacy for its HIV vaccine. J&J developed the HIV vaccine with the same technology used in its successful Ebola and Covid-19 vaccines.
Novartis was unsuccessful developing bimagrumab as a treatment for a rare muscle disorder, but additional tests showed encouraging results in fat reduction. Startup Versanis Bio licensed rights to the antibody and plans to use its Series A financing for clinical trials testing the drug in obese patients.
Shots: The P-III EMPEROR-Preserved trial evaluates the safety & efficacy of empagliflozin (10mg, qd) vs PBO in 5988 patients with chronic HFpEF The results demonstrated a 21% reduction in risk for composite 1EPs of CV death or hospitalization for HF. The 2EPs showed a 27% reduction in risk of first & recurrent hospitalizations for HF …
Researchers ended a large trial in South Africa after finding that an experimental vaccine offered little protection.
Shots: The P-III GRIPHON & P-IIIb TRITON trials evaluate Uptravi vs control group in 404 & 245 patients with PAH, respectively The results from a post-hoc pooled analysis showed a 52% reduction in the risk of disease progression @6mos., Additionally, 48% reduction in the risk of disease progression among patients who received triple therapy group, …
For the healthcare ecosystem to drive research and care, clarity is needed about the opportunities and challenges of ECAs and their value in bringing therapies to patients faster, say COTA Viraj Narayanan and Karla Feghali from ConvergeHEALTH by Deloitte. External control arms (ECAs) – also known as synthetic control arms – are based on real …
External control arms and debunking real-world data myths Read More »
Shots: The P-III study involves assessing JTA-004 (enhanced viscosupplement) vs PBO and the active comparator to reduce pain measured by the WOMAC pain subscale in patients with osteoarthritic pain in the knee The study showed that all treatment arms have similar efficacy, no significant difference in knee pain reduction b/w JTA-004, PBO, and active comparator …
Shots: The study will evaluate the safety and immunogenicity of GBP510 vs AZ/Oxford University’s COVID-19 vaccine and will enroll ~ 4,000 participants across multiple countries The initiation of the P-III study follows positive interim P-I/II data that showed all participants who received the GBP510 developed strong neutralizing Ab responses, demonstrating a 100% seroconversion rate The …
As children’s hospital beds fill up, the F.D.A. shouldn’t delay.
As children’s hospital beds fill up, the F.D.A. shouldn’t delay.
Prescriptions for ivermectin have jumped to more than 88,000 per week, some pharmacists are reporting shortages and people are overdosing on forms of the drug meant for horses.
Shots: The head-to-head P-III JADE DARE study involves assessing the efficacy of Abrocitinib (200mg, qd, PO) vs dupilumab (300mg, SC, q2w followed by 600mg induction dose) in adult patients on background topical therapy with moderate to severe AD The study met its co-1EPs of at least a 4-point improvement in the severity of PP-NRS4 @2wks. …
Shots: The P-II/III LIBERTY AD PRESCHOOL (part B) involves assessing the efficacy & safety of Dupixent (200/300mg) + low-potency TCS vs TCS alone in 162 children aged 6mos. to 5 years with uncontrolled mod. to sev. AD Result: clear or almost-clear skin (28% vs 4%); 75% or greater overall disease improvement (53% vs 11%); improvement …
Prescriptions for ivermectin have jumped to more than 88,000 per week, some pharmacists are reporting shortages and people are overdosing on forms of the drug meant for horses.
The European Commission has approved a BioMarin Pharmaceutical drug that treats achondroplasia, a rare genetic disorder that slows bone growth and is the most common cause of dwarfism. The commission also granted conditional approval to a cancer drug from partners Incyte and MorphoSys.
Shots: The P-III HUYGENS study evaluates Repatha + optimized statin therapy vs optimized statin therapy alone in patients with ACS for 52wks. The study met its 1EPs i.e., improvement in the features of plaque morphology by increasing the fibrous cap thickness (75% vs 39%) as measured by OCT. The therapy also improved all 2EPs includes …
The startup is working with Curebase to launch five clinical trials to test its VR-based therapeutic for chronic pain. They plan to enroll a total of more than 2,000 people.
What You Should Know: – Curebase, a company committed to democratizing access to clinical studies, and AppliedVR, a pioneer advancing the next generation of digital medicine, today announced a one-year partnership where AppliedVR will deploy the Curebase platform to run five clinical trials assessing the effectiveness of virtual reality-based (VR) therapy to treat chronic pain. …
Curebase, AppliedVR Partner on Multiple VR Therapy Clinical Trials Read More »
The royalty agreement with HealthCare Royalty Partners will support ADC Therapeutics’ plans to commercialize its newly approved cancer drug and develop the next one in its pipeline. The biotech’s drug Zynlonta is the first of its type approved by the FDA as a treatment for diffuse large cell B-cell lymphoma
Shots: The P-III FoCus trial evaluates the efficacy and safety of ALXN1840 vs SoC in 214 patients aged ≥12yrs. in a ratio (3:1) with Wilson disease The trial met its 1EPs i.e., improvement in daily mean copper mobilization from tissues defined as daily mean AUEC for directly measured dNCC @48wks. The therapy was generally well-tolerated …
Everyone is familiar with the phrase ‘patient-centricity’ but, where it might once have been seen as a ‘checkbox exercise’, it has gained preeminent status in the era of COVID-19, says Prime Global’s Ross Jackson. The pandemic has transformed many aspects of our industry and one area that change has come to is patient engagement within …
Amplifying the patient voice for better engagement, recruitment and retention Read More »
Shots: The P-III PNEU-PED study evaluates immunogenicity, safety & tolerability of Vaxneuvance (4-dose regimen) vs PCV13 in 1720 infants aged b/w 42-90 days with IPD Vaxneuvance showed a non-inferior immune response to PCV13 for 12 & 13 shared serotypes @ 30days, following PD3 & for all 13 based on serotype-specific IgG GMCs, following PD4. The …
As U.S. plans for Covid-19 booster shots unfold, Johnson & Johnson is angling to join the mix, reporting data that it says support a second shot of its vaccine. But the data the company points to are sparse and it’s not entirely clear they support booster shots.
What You Should Know: – ConcertAI, a leader in RWD and enterprise AI tech for oncology, announced the expansion of its multi-year collaboration with Janssen. The expanded collaboration aims to further the novel work that the two companies’ are doing to broaden access to trials in new sites and strengthen trial diversity. – This expanded …
ConcertAI, Janssen Partner to Address Health Disparities in Clinical Trials Read More »
Chronic kidney disease patients who experience pruritus after dialysis can now get relief from the first FDA-approved treatment for the condition. Cara Therapeutics received the FDA’s nod green light for its injectable drug, Korsuva.
The Theravance Biopharma drug is the lead program in an alliance with Johnson & Johnson subsidiary Janssen Biotech, which paid $100 million up front to develop the JAK inhibitor for intestinal diseases. Following the Phase 2 failure, Theravance said it will minimize spending on that program.
Shots: The P-III BELINDA study evaluates the efficacy, safety, & tolerability of Kymriah vs SOC in patients with aggressive B-cell NHL who had primary refractory disease or relapsed within 12mos. of 1L treatment in 73 sites across 18 countries globally The study did not meet its 1EPs of EFS while the safety profile was consistent …
Shots: Merck KGaA discontinues its P-II INTR@PID BTC 055 study evaluating bintrafusp alfa with gemcitabine + cisplatin in patients with locally advanced or metastatic BTC The discontinuation is based on the IDMC’s recommendation following a review of data which concluded that the trial fails to meet the primary objective of OS & no new safety …
Shots: The P-IIb dose-finding induction study evaluates izencitinib (PO, qd) vs PBO in 239 patients with a mod. to sev. active UC for 8wks. The results from the P-II study for CD is expected in late Q4’21 or early Q1’22 The study did not meet 1EPs & 2EPs of change in total Mayo score & …
Though Gilead Sciences leads the group of companies developing immunotherapies that target the cancer protein CD47, Pfizer contends that molecules it is getting through the Trillium Therapeutics acquisition have safety and efficacy advantages that could make them the best in this emerging drug class.
Shots: The IDMC has recommended the discontinuation of the P-III CHAMPION-ALS trial for Ultomiris (IV, q8w) in adults with ALS. Additionally, enrolled patients will discontinue the study medication and complete any required follow-up assessment The discontinuation is due to lack of efficacy based on the pre-specified interim analysis. No new safety was identified while the …
Vigil Neuroscience led the way with a $90 million round of funding, one of four biotech companies to close Series B financing rounds in the past week. The fresh capital comes as each of the companies looks ahead to bringing their respective drugs into the clinic.
An AstraZeneca antibody drug for Covid-19 has clinical data showing the drug reduced the risk of infection by 77%. With the results, the pharmaceutical giant pulls ahead of other companies developing injectable versions of antibody drugs for Covid-19 prevention.
Alexion Pharmaceuticals drug Ultomiris is already approved to treat a rare blood disorder, but the company hoped the antibody’s approach could also work in amyotrophic lateral sclerosis. Despite failing in ALS, Alexion, recently acquired by AstraZeneca, brings additional clinical-stage programs for rare diseases.
Shots: The P-I CHRYSALIS study evaluates the safety, PK, and preliminary efficacy of Rybrevant (1050/1400 mg) as a monothx. and in combinations with lazertinib in 19 patients with NSCLC with METex14 mutations The initial data showed anti-tumor activity and a consistent safety profile with reported experience at the approved P-II dose. Disease response was evaluated …
Shots: The P-III PROVENT, pre-exposure prophylaxis trial evaluates safety & efficacy of AZD7442 (300mg, IM) vs PBO in 5197 patients in a ratio (2:1) with COVID-19 across 87 sites in the US, UK, Spain, France & Belgium Results: AZD7442 (1st LAAB combination) showed a 77% reduction in risk of developing symptomatic COVID-19, 75% of patients …
Shots: The companies collaborated to evaluate ADG106 + Keytruda in dose-escalation and expansion clinical study to treat advanced or metastatic solid tumors & hematological malignancies In preclinical studies, ADG106 showed antitumor activity and was well tolerated as a monothx. and in combination with other immuno-oncology therapies ADG106 is a fully human, ligand-blocking, agonistic anti-CD137 IgG4 …
A common set of standards for randomised clinical trials (RCTs) aims to help researchers develop the drugs and interventions of the future – no matter what they do or where they are based. The Good Clinical Trials Collaborative (GCTC) has published a draft guidance document, and is asking for the life sciences sector to make …
Does draft RCT guide set the scene for future trials? Read More »
Shots: The P-III CHOICE-01 trial evaluates toripalimab + CT vs PBO + CT in 465 patients in a ratio (2:1) with advanced sq./nonsq. NSCLC. The results of the study will be presented at WCLC 2021 Results: improvement in PFS with m-PFS (8.3 vs 5.6mos.), m-OS (21.0 vs 16.0mos.). In sq. & nonsq. NSCLC patients – …
Shots: The P-III ADVANCE trial evaluates efficacy, safety & tolerability of atogepant (PO, 10/30/60 mg, qd) vs PBO in 910 patients with EM who experienced 4-14 migraine days/mos. Results: patients achieving a ≥50% reduction in monthly migraine days (55.6%/58.7%/60.8% vs 29.0%) @12wks. & all doses were well tolerated. Additionally, 30/60 mg doses showed improvements in …
GlaxoSmithKline drug Jemperli is now FDA approved for treating solid tumors throughout the body as long as they have a particular genetic signature. Expanding the drug’s approval means the drug can treat more patients who have more types of cancer, and it also gives GSK a way to grow revenue in its thin oncology portfolio.
Shots: The company reported 2yrs. & 1 yrs. results from P-III KITE & KINGFISHER studies evaluating Beovu (6mg, q4w) vs aflibercept (2mg) in 926 patients with DME across 36 countries The results showed non-inferiority to aflibercept in a change in BCVA from baseline @100wks. in KITE & 52wks. in KINGFISHER studies. The therapy also demonstrated …
Verily’s recent moves have signaled the company’s interest in supporting decentralized clinical trials and real-world studies. Now the life sciences subsidiary of Alphabet is bolstering its capabilities in this these areas with the acquisition of startup SignalPath.
Gritstone bio is developing Covid-19 vaccines that use self-amplifying messenger RNA, which is intended to further boost the immune response compared to mRNA alone. The company also says its vaccine candidates may be able to protect against variants of the novel coronavirus.
Over the past two years, the pharma industry’s demand for automated research solutions has increased significantly and, particularly when it comes to clinical trial design, the need has never been greater. This shift to digitised approaches has been triggered by some persistent challenges faced by those companies still reliant on applying manual processes. “A few …
From start to finish – digitising clinical trials Read More »
Shots: The FDA decision to lift the hold of P-I clinical trial for RP-A501 is based on the modification of the trial protocol and other supporting documents with revised guidelines for patient selection and management The FDA allows the company to restart the patient enrolment. Additionally, the company is expected to initiate the dosing of …
Shots: The ongoing P-III ADvocate 1 & 2 studies evaluate lebrikizumab as monothx. vs PBO in adult & adolescent patients aged 12 to less than 18 yrs. with mod. to sev. AD for 52wks. The 1EPs & 2EPs of study i.e >75% change from baseline in EASI score @16wks., improvement in skin clearance & itch …
FDA approval of the Merck drug, belzutifan, covers treatment of tumors caused by von Hippel-Lindau disease, an inherited disorder associated with several types of cancer. Merck added the small molecule to its pipeline two years ago as part of a $1.2 billion acquisition.
I was mentioning the number of unusual therapeutic modes that are being explored these days, and one of those is (broadly) RNA-based approaches. The ones that directly feed into coding for proteins get a lot of attention (the mRNA vaccines, for example), and everything that’s currently approved is some sort of antisense or siRNA species. …
Covid-19 vaccine booster shots are now authorized for organ transplant patients and others who have weakened immune systems. The FDA based its update on the results of two small studies that tested third shots of the mRNA Covid vaccines from Moderna and Pfizer/BioNTech.
Clinical trials are an essential part of the drug development process. Worldwide, more than 2 million clinical trials are registered and the number is steadily increasing over the past several years. While clinical trials are pivotal to analyze the efficacy of any new therapy or drug, every year, a lot is spent on clinical trials. …
Reimagining Clinical Trials: Why are Virtual Clinical Trials Becoming the New Normal? Read More »
Shots: The withdrawal follows the recent discussion with the US FDA regarding the palovarotene’s NDA review that initiated in May’21 The additional analysis will be required to progress and complete the review process from Ipsen’s P- III MOVE and FOP program. Ipsen plans to resubmit to the FDA, following completion of the required data Palovarotene …
Shots: The US FDA has put a partial clinical hold on Aprea’s clinical program that evaluates eprenetapopt + acalabrutinib or with venetoclax and rituximab in patients with lymphoid malignancies. The FDA’s decision is related to the safety & efficacy data from the P-III MDS study The patient enrollment will be pause until the clinical hold …
Aprea Reports the FDA’s Hold on Clinical Trials for the Treatment Lymphoid Malignancy Read More »
FDA approval of a therapy that pairs drugs from Eisai and Merck allows the combination to become a first-line treatment for renal cell carcinoma. It’s the latest approval for the partners, which have been working together since 2018.
Shots: The P-III trial evaluates the efficacy and safety of GC5107 (IV, every 21 or 28 days for 12 mos.) in 49 patients aged 3-70yrs. with PI. The trial was conducted at 17 study sites (10 in the US & 7 in Canada) The trial met its1EP & 2EPs i.e., <1 aSBI occurred among the …
Shots: The ongoing P-II/III study evaluates safety, efficacy & cytokine levels of plonmarlimab as monothx. (6mg/kg) vs PBO for CRS in patients with COVID-19 The interim analysis showed preliminary results i.e., @day 30, higher MVF rate (83.6% vs 76.7%), lower mortality rate (4.9% vs 13.3%), higher recovery rates (68.9% vs 56.7%) @day 14 & (80.3% …
I-Mab Reports Interim Data of Plonmarlimab (TJM2) in P-II/III Study to Treat COVID-19 Read More »
Shots: The P-III CREDIT-2 study evaluates the efficacy and safety of IBI306 vs PBO in 148 Chinese patients with HeFH, prior treated with statins and ezetimibe. The study provides high-quality clinical evidence for patients with the same indication The study met its 1EPs i.e., reduction in the LDL-C levels @12wks. of continuous treatment & demonstrated …
Shots: The NDA is based on P-III PSOARING 1 & 2 along with interim results from PSOARING 3 OLE study evaluating the safety & efficacy of tapinarof vs vehicle in patients with PsO In PSOARING 1 & 2 results: patients achieved a PGA score of 0 or 1 (35.4% vs 6% & 40.2% vs 6.3%) …
Technological innovations have enabled the adoption of decentralized and hybrid clinical trial models (DCTs) at […]
Reify, which makes software for clinical trial recruitment and decentralized clinical trials, raised $220 million in a funding round led by Coatue Management.
Shots: The company has completed the enrolment in the P-III HELIOS-B study evaluates the efficacy and safety of vutrisiran (25 mg, SC, q3M, for ~ 36 mos.) vs PBO in 600 patients in a ratio (1:1) with ATTR amyloidosis with cardiomyopathy across 123 activated sites in 32 countries The 1EP of the study is to …
Shots: The ongoing BE BRIGHT, OLE trial evaluates the safety, tolerability, and efficacy of bimekizumab (q4w/q8w) in 989 patients with the mod. to sev. PsO The interim data showed that @16wks., 9/10 patients achieved clear or almost clear skin (IGA 0/1), 8/10 patients achieved complete skin clearance (PASI 100) and maintained responses through 2yrs. with …
Shots: The P-III POLARIX trial evaluates the efficacy, safety, and PK of Polivy + R-CHP vs R-CHOP in 879 patients in a ratio (1:1) with previously untreated DLBCL for 6 cycles, followed by Rituxan for 2cycles; or R-CHOP + Polivy PBO for 6 cycles, followed by 2 cycles of Rituxan. The trial is being conducted …
Shots: The P-III ORIENT-11 study evaluates the efficacy & safety of sintilimab (200mg) + pemetrexed and platinum CT vs PBO + CT in 397 patients in a ratio (2:1) as 1L treatment for advanced or metastatic nonsq. NSCLC without sensitizing EGFR mutations or ALK rearrangements The results demonstrated an OS benefit with a median follow-up …
A study of women’s participation in cardiovascular clinical trials revealed that only 38% of participants were women. But if women comprise 50 percent of the population, that should be reflected in clinical trials too.
Boosters may not be necessary yet, many experts say, and the pursuit of additional shots raises ethical questions.
Patient enrollment is a common issue across many therapeutic areas in clinical research. Psychiatric disorders in particular represent a trial area with significantly high drop-out rates and poor patient recruitment. The reasons behind this problem, however, are rarely studied or understood. Despite this, new strategies devised over the last five years are beginning to reverse the …
Predicting and improving clinical trial enrolment: A problem for psychiatry Read More »
Shots: The P-III HELP OLE study evaluates the safety & efficacy of Takhzyro (300mg, q2w) in 212 patients aged ≥12yrs. with HAE attacks for ~2.5yrs. Results: 87.4% mean reduction in attack rate compared to baseline; attacks requiring acute treatment (93.4%); mod. or sev. attacks (84.3%); HAE attack-free (97.7%) with a duration of the attack-free period …
The ketamine story is an excellent example of why CNS drug discovery is. . .well, how would the HR department put it? Well-stocked with personal growth opportunities? Full of areas in which to innovate? Those of us in the lab-research end of things consider it an extraordinary maze of conflicting clues which we currently can …
Shots: The P-III EMPOWER-Lung 3 trial evaluates Libtayo (350mg, IV, q3w for 108wks.) + platinum-doublet CT (q3w for 4 cycles) vs platinum-doublet CT alone in a ratio (2:1) in 466 patients with metastatic or LA tumors with either sq./nsq. histology across all PD-L1 expression levels The results demonstrated an improvement in OS (22 vs 13mos.), …
Shots: The P-III trial evaluates REGEN-COV (1200mg, SC) vs PBO in a ratio (1:1) in 1505 patients who lived in the same household with COVID-19 patients diagnosed within the prior 4 days The trial met its 1EPs i.e. 81% reduction in the risk of symptomatic infections, 93% reduction after 1wks. with no dose-limiting toxicities. REGEN-COV …
Shots: The P- IIb/III ALLEGRO trial evaluates ritlecitinib {50/30mg, (with/out one month of initial treatment with ritlecitinib, 200 mg), 10mg, PO, qd} vs PBO in 718 patients aged ≥12yrs. with alopecia areata The trial met its 1EPs of improving scalp hair regrowth, patients with 30/50mg dose had ≤ 20% scalp hair loss @24wks., the safety …
Shots: The companies collaborated to evaluate F-star’s FS120 (tetravalent bispecific Ab targeting CD137 & OX40) + MSD’s Keytruda (anti-PD-1 therapy) in patients with cancer The P-I study evaluates the PK & PD of FS120 as monothx. in dose escalation in patients with advanced cancer while MSD will supply Keytruda for a combination arm in the …
F-star Collaborates with MSD to Evaluate FS120 + Keytruda for the Treatment of Cancer Read More »
The short answer is that cancer vaccines represent no silver bullet but, in combination with other immunotherapy and drug cocktails, can prove potent.
The need for automated metadata management software has never been greater. The number of global clinical trials being run is increasing every year. And, they are becoming increasingly complex to manage. There’s a vast amount of data that needs to be collected and analyzed. There are more regulations in place than ever before and industry …
Shots: The P-IIb clinical trial involves evaluating the efficacy and safety of eliapixant (25/75/150mg, PO, bid) vs PBO in 300 patients with refractory chronic cough for 12wks. The trial met its 1EPs that showed an improvement in 24hrs. cough counts @12wks. of treatment. The results showed a favorable safety and tolerability profile of the therapy …
The artificial intelligence-based technology of Iterative Scopes brings computer vision analysis to endoscopic images. The startup’s technology was initially developed to assist gastroenterologists in finding pre-cancerous polyps but CEO and founder Jonathan Ng said it’s also finding additional use helping pharmaceutical companies identify patients for clinical trials.
Sanofi is acquiring Translate Bio, its messenger RNA R&D partner for the past three years, in a $3.2 billion deal. The pharmaceutical and vaccines giant said the acquisition will speed up development of partnered vaccine programs and spark work on new ones in areas such as cancer, immunology, and rare diseases.
Shots: The P-III COV-BARRIER substudy evaluates baricitinib + SoC vs PBO + SoC in 101 adult patients with COVID-19 who are on mechanical ventilation or extracorporeal membrane oxygenation (ECMO) The results showed reduction in mortality (46% & 44%) @ Day 28 & 60. The findings are consistent with the reduction in mortality in the overall …
Shots: The FDA decision to lift the partial clinical trial hold of the OAV-101 IT clinical program is based on data from a nonclinical toxicology study in NHP The company plans to start P-III STEER study to evaluate the efficacy & safety of OAV-101 IT vs sham controls in treatment naïve patients aged b/w 2-18yrs. …
Novartis Lifts Partial Clinical Trial Hold on OAV-101 Clinical Program to Treat SMA Read More »
An AstraZeneca lupus drug has been awarded FDA approval for treating the most common form of the autoimmune disorder. The regulatory decision gives AstraZeneca a product that can compete head-to-head with GlaxoSmithKline’s lupus drug, Benlysta.
Of the 20 companies that went public in the last week of July, 11 of them represent some aspect of the life sciences. The tally of newly public companies made the week the biggest for IPO activity in more than 20 years.
Icosavax’s research developing a vaccine for a virus that can lead to deadly respiratory infections in the very young and the very old has found an additional application in the pursuit of a vaccine for the novel coronavirus. The biotech’s $182 million IPO gives it the capital to move forward with clinical research on multiple …
Icosavax’s $182M IPO haul is key for RSV, but eyes are on its Covid vaccine too Read More »
Patients taking new Alzheimer’s disease drug Aduhelm will be evaluated in a real-world study lasting up to five years. Biogen announced plans for the observational study, which is one of three post-marketing clinical programs planned to generate more data about the drug
With a string of new drugs for Alzheimer’s disease making their way through late-stage clinical testing, the demand for suitable subjects to enrol into trials is running at an all-time high – and threatens to outstrip supply. Now, the National Institute on Aging (NIA) – part of the US National Institutes of Health (NIH) – …
NIH digital tool aims to boost enrolment into Alzheimer’s studies Read More »
Shots: The company has reported the results from Part 2 of the FIREFISH study evaluating the efficacy and safety of Evrysdi in 41 infants aged 1-7 mos. with symptomatic Type 1 SMA The study met its 1EP i.e., 29% of infants sitting without support for at least 5 sec., 93% of infants were alive and …
Shots: The P-III SEQUOIA trial evaluating efficacy & safety of Brukinsa vs bendamustine + rituximab (B+R) in patients with treatment-naïve CLL or SLL whose tumor did not exhibit the deletion of chromosome 17p13.1 (del[17p]) The interim analysis of trial met its 1EPs @ median follow-up of 25.8 mos, demonstrates improvement in PFS as assessed by …
The long list of side effects that follow ads for the newer expensive drugs to treat Type 2 diabetes sometimes include an unusual warning: They might cause weight loss. That side effect is one that many people — especially those with Type 2 diabetes, which is associated with obesity — may desperately want. So it’s …
Diabetes Drug’s New Weight Loss Formula Fuels Cost-Benefit Debate Read More »
Ardelyx drug tenapanor was rejected by the FDA as a treatment for a complication experienced by chronic kidney disease patients. The regulatory decision came two weeks after the company received an FDA letter citing deficiencies in the drug application.
Boosters may not be necessary yet, many experts say, and the pursuit of additional shots raises ethical questions.
Shots: The study A of P-III LIBERTY CUPID trial evaluates Dupixent vs SoC in 138 patients aged ≥6 yrs. with CSU who remained symptomatic despite antihistamine use and who were not previously treated with anti-IgE therapeutics The trial met its 1EPs & 2EPs @24wks. i.e. showed a reduction in itch severity (63% vs 35%), reduction …
Shots: The companies will initiate P-I/II study evaluating the safety and preliminary efficacy of BBP-398 in combination with both Opdivo as dual therapy & Opdivo + KRASG12C inhibitor as triplet therapy in NSCLC patients with KRAS mutations, as 1L & 2L treatment options BridgeBio will sponsor the study while BMS will provide nivolumab to the …
AC Immune is bolstering the Parkinson’s disease portion of its drug pipeline with a deal to acquire a therapeutic vaccine that Affiris is developing for the disorder. Switzerland-based AC Immune plans to advance its new asset to Phase 2 testing.
Shots: BioNTech unveils the plan to develop the first mRNA-based vaccine to prevent malaria with anticipated initiation of the clinical study by the end of 2022 The company intends to set up a large vaccine production hub in Africa with the help of local partners and transfer its mRNA technology to Africa. These efforts of …
Shots: Prothena presents new data for PRX012 (anti-amyloid beta (Aβ) Ab) and the company’s dual Aβ-tau vaccine program being developed for the prevention and treatment of AD The preclinical data of PRX012 (SC) highlights significant ex vivo clearance of both pyroglutamate-modified and -unmodified Aβ plaque from AD brain at concentrations expected to be reached in …
Prothena Presents Preclinical Data of its Alzheimer’s Candidates at AAIC 2021 Read More »
The F.D.A. wants Pfizer and Moderna to increase the number of 5- to 11-year-olds who participate in trials of their coronavirus vaccines to ensure there is enough data about rare side effects.
Bristol Myers Squibb blockbuster drug Opdivo is being pulled from the market after failing to meet the main goal of a confirmatory study. The voluntary withdrawal comes amid broader FDA scrutiny of cancer drugs that were given accelerated approval.
An Incyte drug developed as a treatment for a rare type of anal cancer has fallen short of FDA approval. The regulator said it needs to see more clinical data that demonstrating clinical benefit for the antibody drug.
Shots: Denali reports data from the P-I/II study evaluating DNL310 (qw, IV) for both CNS and peripheral MPS II. All patients received doses of DNL310 after switching from idursulfase enzyme replacement therapy on Day 1 Results of Cohorts A & B showed the levels of heparan sulfate in CSF normalized in all patients analyzed with …
A lack of racial diversity in clinical trials is a long-standing, well-documented problem that contributes to the stark health inequalities that have been brought into sharp focus by COVID-19. Tackling it, however, has not been easy, thanks in no small part to the reasons being as complex as they are multi-faceted. But, according to the …
Tackling the ‘disease of systemic racism’ in clinical trials Read More »
Shots: The P-III GEMINI 1 study involves assessing AGN-190584 (pilocarpine 1.25%, qd) ophthalmic solution in 323 patients in a ratio (1:1) with presbyopia for 30days. The study demonstrated that AGN-190584 met both its 1EPs & 2EPs with patients achieving near & intermediate vision gains. In addition, AGN-190584 showed no loss of distance vision, rapid onset …
Adverum Biotechnologies still doesn’t know if its experimental gene therapy caused eye problems reported in April in one patient in its diabetic macular edema study. But four others have since developed similar problems, and the company will no longer continue to develop the therapy for that indication.
Caribou Biosciences’ CRISPR approach to cell therapy sparked strong investor interest, enabling the clinical-stage biotech to upsize its IPO and raise $304 million in the biggest life sciences IPO this week. Absci, Sophia Genetics, and Cytek Biosciences also priced IPOs.
Research into these often mysterious and debilitating disorders has languished. That’s changing.
Shots: The company has completed enrollment in the P-II CATALINA study evaluating the safety and efficacy of NGM621 (IVT injections, q4w/ q8w) vs sham control in 320 patients with geographic atrophy secondary to AMD. The results are expected in H2’22 The 1EPs of the P-II CATALINA study is the rate of change in geographic atrophy …
Arvinas’ early clinical data for its targeted protein degradation drug for breast cancer drew partnering interest from several companies. Pfizer beat them all with a deal that pays the biotech $1 billion to share in the development and commercialization of this therapy.
Shots: The P-Ib MAD trial involves assessing IMT-002 (350 /1050 mg, qd/bid over a 2wks.) vs PBO in 30 T1D patients preselected for HLA-DQ8 aged b/w 18-45yrs. and were interspersed in the 4 groups and involves a 1wk. follow-up after dose completion The therapy was safe & well-tolerated with no SAEs & did not increase …
IM Reports Results of IMT-002 in P-Ib MAD Trial for the Treatment of Type 1 Diabetes Read More »
The FDA has approved Bylvay, an Albireo Pharma drug that treats the severe itching experienced by patients with progressive familial intrahepatic cholestasis (PFIC). Prior to this approval, the only treatment options for the rare liver disorder were surgical.
Shots: The company has completed the enrollment in the P-III C.L.E.A.R. program evaluating SP-102 vs PBO in 400 patients with lumbosacral radicular pain or sciatica at 40 sites across 25 states in the US. The results are expected in Q4’21 The 1EPs of the study is mean change in the numeric pain rating scale for …
Shots: The ongoing P-IIa trial evaluating the safety, tolerability & PK of islatravir (60/120mg, PO, once-monthly for 6 mos.) vs PBO in a ratio (2:2:1) in 242 patients with PrEP of HIV infection for 24 wks. The PK analysis showed that the level of islatravir (60/120 mg monthly doses) in PBMCs continues to remain above …
An amyotrophic lateral sclerosis drug from Amylyx Pharmaceuticals is being prepared for Phase 3 clinical testing on track to begin later this quarter. To support that research, the biotech has raised $135 million in financing.
Though some of its own senior officials said there was little evidence of benefit for patients, the F.D.A. nonetheless greenlighted Biogen’s Aduhelm, or aducanumab.
Though some of its own senior officials said there was little evidence of benefit for patients, the F.D.A. nonetheless greenlighted Biogen’s Aduhelm, or aducanumab.
A Cytokinetics drug in development for treating a rare heart disorder has promising Phase 2 data suggesting it could compete against a Bristol Myers Squibb drug currently under FDA review. Cytokinetics plans to advance its small molecule to Phase 3 testing by the end of this year.
An intranasal Covid-19 vaccine candidate from Meissa Vaccines could offer advantages compared to injectable ones. The company is still in early clinical development but the company now has data from monkey studies that further build the case for the vaccine.
Research organizations can participate in ethical data sharing today by considering participants’ privacy and security and balancing the need to protect their commercial interests with the need to share enough data to benefit medical research.
Shots: The P-IIIb STASEY study evaluates the safety of Hemlibra (qw for ~2 yrs.) in 193 patients with hemophilia A with factor VIII inhibitors. The results of the study are consistent with a P- III HAVEN program The results from the final analysis of a P-IIIb STASEY study showed no new thrombotic microangiopathy or thrombotic …
Shots: The ongoing P-II/III CAPELLA trial evaluating the antiviral activity of lenacapavir (SC, every 6mos.) + other antiretroviral agents vs PBO in 36 patients in a ratio (2:1) with multi-drug resistant HIV-1 infection. The trial is being conducted at research centers in North America, the EU & Asia The study demonstrated high rates of virologic …
Shots: The P-III ASCEND program includes 5 studies i.e ASCEND-ND, D, ID, NHQ & TD studies evaluating efficacy & safety of daprodustat vs ESA in ~8,000 patients with anemia due to CKD for ~ 3.75yrs. The program met its 1EPs in 5 studies i.e improvement in Hgb levels in untreated patients and maintaining Hgb levels …
Vaxneuvance, a Merck pneumococcal vaccine, is now approved by the FDA. The intramuscular shot protects against 15 pneumococcal strains—five fewer than the 20 covered by a Pfizer pneumococcal vaccine that the FDA approved in June.
Cancer drug developer Erasca, whose mission is to “erase cancer,” has raised $300 million from its IPO. The clinical-stage biotech addresses a single elusive cancer target; it has multiple programs taking multiple approaches, two of them in human testing and the rest on track to join them.
The FDA has yet to approve a therapy for memory loss. Nia Therapeutics is developing a medical device that stimulates the brain to bring it back to a “good memory state,” and it was judged the winner in the life sciences track of the INVEST Precision Medicine Pitch Perfect Competition.
Shots: The 1st patient has been dosed in the P-II MARCH trial evaluating safety, tolerability & efficacy of VIR-2218 + VIR-3434 (SC) in ~90 patients aged 18-65yrs. with chronic HBV infection for 4-20wks. & follow-up period of ~116 wks., depend on dosing cohort The 1EPs of a trial is the proportion of patients with AEs …
Shots: The P-III study evaluates the safety and efficacy of Ultomiris vs PBO in 175 adults in a ratio (1:1) with gMG with no prior treatment with a complement inhibitor medicine across North America, EU, Asia-Pacific, and Japan for 26 wks. The study met its 1EPs of change from baseline in MG-ADL total score @26wks. …
Wugen, a biotech developing “off-the-shelf” natural killer and CAR T cell therapies, has raised $172 million in Series B financing. The company’s lead program has already reached human testing and the new capital will be used to continue that research and advance other pipeline programs to the clinic.
Shots: The ongoing P-I/IIa sub-study involves evaluating safety, reactogenicity & immunogenicity of COVID-19 vaccine (IM, given as single-dose or two-dose, 8 wks. apart) in adults across multiple clinical sites in Belgium & the US The results demonstrated durable humoral & cellular immune responses in single-shot COVID-19 vaccine @8mos. after immunization & elicits neutralizing Ab activity …
Shots: The P-III REACH3 study involves the safety & efficacy of ruxolitinib vs BAT in patients with steroid-refractory chronic GVHD The study showed improvements in 1EPs i.e ORR (49.7% vs 25.6%) & BOR (76.4% vs 60.4%) @24wks. & also showed the improvements in 2EPs i.e patients achieved longer FFS than BAT, m-FFS (not reached vs. …
Shots: The P-Ib study evaluates GLPG3667 (low/high dose) vs PBO in 31 patients in a ratio (1:1:1) in patients with moderate to severe plaque psoriasis for 4wks. Results: 40% of patients with the high dose of GLPG3667 showed improvement of 50% in PASI response from baseline @4wks. and was well tolerated with no serious AEs. …
Galapagos Reports Results of GLPG3667 in P-Ib Study for the Treatment of Psoriasis Read More »
The major medical center’s rejection of the new Alzheimer’s drug is one of the starkest signs of concern over its effectiveness.
Shots: The first patient has been dosed in the P-I ANTLER trial evaluating CB-010 in patients with r/r B-NHL. In the dose-escalation part of the study, patients who have failed at least 2L of CT or immunotherapy will receive the therapy following lymphodepletion The initial purpose of the dose escalation is to evaluate the safety …
Shots: The open-label, HYPHOP study involves assessing Keveyis vs PBO in patients with PPP, following 9wks. controlled study. The results are published in the journal, Muscle & Nerve The results from post hoc analyses of the HYPHOP study confirmed that the therapy is safe and effective for chronic use, efficacy was maintained @61wks., reduction in …
At 15, Autumn Fuernisen is dying. She was diagnosed at age 11 with a rare degenerative brain disorder that has no known cure or way to slow it down: juvenile-onset Huntington’s disease. “There’s lots of things that she used to be able to do just fine,” said her mom, Londen Tabor, who lives with her …
Dying Patients With Rare Diseases Struggle to Get Experimental Therapies Read More »
Shots: The P-IIa AMBITION study assessing CRV431 (75/ 225mg, qd, PO given as soft gelatin capsules) vs PBO to presumed F2 and F3 NASH patients for 28days, followed by a 14-day observation period for safety The study met its 1EPs i.e. safety, tolerability & PK with no SAEs at both doses while some patients experienced …
Hepion Reports Results of CRV431 in P-IIa AMBITION Trial for the Treatment of NASH Read More »
By paying $125 million up front for rights to multiple sclerosis drug candidate orelabrutinib, Biogen secures a place among those companies trying to treat the disease by targeting a particular enzyme highly expressed in the central nervous system. But Biogen still has some catching up to do.
Shots: The P-II clinical trial involves evaluating ASP377 (a novel 24 valent MAPS Vaccine) vs Prevnar 13 & Pneumovax 23 in 503 adults aged 65-85yrs. with Streptococcus pneumoniae. The vaccine was developed using Affinivax’s MAPS platform technology The results showed that the ASP3772 was well tolerated, induced a robust immune response in 24 pneumococcal serotypes …
The regulated content and labeling software firm has made three new customer-facing appointments and created a brand-new Customer Experience Team to provide enhanced, end-to-end customer support for medical device and clinical trial organizations. Chief Executive Richard Adams says the creation of the new team reflects the company’s desire to improve the hands-on advice and assistance …
PRIYSM ID boosts customer service offer with new customer experience team Read More »
Novo Nordisk is paying $100 million up front to acquire an experimental Prothena drug being developed to treat heart problems stemming from a misfolded protein. The deal is part of a broader strategy to expand into drugs for cardiovascular disease.
Shots: The P-III UNIFI study involves assessing the safety & efficacy of Stelara (90mg, q8w/q12w) as induction and maintenance therapy vs PBO in 588 patients with UC with inadequate response & were unable to tolerate conventional therapies Results: in IIT population, 55.2% were in symptomatic remission & 96.4% of the patients in symptomatic remission were …
Shots: The P-III SELECTION trial comprises 2 induction trials & a maintenance trial evaluating filgotinib (200/100 mg) vs PBO in adult patients with active UC Induction studies results: improvements in PROs of SF & RB in biologic-naïve & biologic-experienced patients (200mg dose). In induction study A & B (18.8% vs 9.5% & 10.7% vs 4.2%) …
Karen Petrou invented a new funding model for curing blindness. Proposed legislation aims to apply it to medical research more generally.
Shots: The first patient has been dosed in a P-I CIBI323A101 study evaluating IBI323 (anti-LAG-3/PD-L1 bispecific Ab) in patients with advanced solid tumors. The trial is being conducted in China The purpose of the P-I dose-escalation and expansion study is to evaluate the safety, tolerability, optimal dosage, and preliminary efficacy of IBI323 in patients with …
Shots: The P-III HELP OLE study evaluating the safety & efficacy of Takhzyro (300mg, q2w) in 212 patients aged ≥12yrs. with HAE attacks for ~2.5 yrs. Results: 87.4% mean reduction in attack rate compared to baseline & median reduction was 97.7%. Additionally, @70 day steady-state, attack rates were further reduced to a mean of 92.4% …
The FDA has approved Bayer’s Kerendia to treat chronic kidney disease in patients with type 2 diabetes. The drug is the first in its class, giving the pharma giant an alternative way to compete against medicines already marketed by AstraZeneca and Bayer.
A hemophilia A patient in a Sigilon Therapeutics clinical trial developed a well-known complication to treatments for the blood disorder. The potential problem for Sigilon is that its cell therapy is designed to avoid triggering such immune responses.
Scientists were critical of the announcement, pointing to evidence that the current two-dose regimen is powerfully effective against the coronavirus.
Scientists were critical of the announcement, pointing to evidence that the current two-dose regimen is powerfully effective against the coronavirus.
Xilis is developing technology that creates a living model of a patient’s tumor, then applies artificial intelligence to determine the best drug or drug combinations to treat the cancer. Clinical trials are planned to test this precision medicine approach and the biotech startup has raised $70 million to fund its research.
The agency faced criticism for approving Aduhelm for all Alzheimer’s patients. Now it recommends that the drug be given only to those with mild symptoms.
The agency faced criticism for approving Aduhelm for all Alzheimer’s patients. The new label recommends that the drug be given only to patients with mild symptoms.
Israel informó que la vacuna de Pfizer tenía una efectividad del 64 por ciento contra la variante Delta. Otras investigaciones sugieren una tasa más alta.
I blogged earlier this year about some interesting androgen antagonist results in coronavirus therapy. The idea is that the TMPRSS serine protease needs to be present at the cell membrane for the entire cell-entry pathway of the virus to work, and that androgen antagonists decrease its expression sharply. As the references in that earlier post …
An Update on Anti-Androgen Therapy for the Coronavirus Read More »
Israel reported that the Pfizer vaccine was 64 percent effective against the Delta variant. Other studies suggest a higher rate.
Israel informó que la vacuna de Pfizer tenía una efectividad del 64 por ciento contra la variante Delta. Otras investigaciones sugieren una tasa más alta.
For community cancer centers that rely on patient reimbursement to stay afloat, a smart data-driven approach to clinical trials provides a foundation for future growth. Brenda Noggy Dr. Tandy Tipps By TANDY TIPPS and BRENDA NOGGY Covid-19’s tragic, devastating impact on cancer treatment is now well documented. Cancer screenings dropped by almost 90 percent at …
Researchers are trying to discern whether vaccines work on the Delta variant.
Researchers are trying to discern whether vaccines work on the Delta variant.
A NeRRE Therapeutics drug that failed a clinical trial for refractory or unexplained chronic cough is getting a second shot as a potential treatment for chronic cough in patients with idiopathic pulmonary fibrosis, a rare lung disorder. The biotech raised £20M to proceed with Phase 2 testing.
Shots: The P-III STRATA2016 trial evaluates the efficacy & safety of StrataGraft treatment vs autograft in 71 patients aged >18 yrs. with deep partial-thickness thermal burns across 12 burn centers in the US. The trial was supported by BARDA The study met its both co-primary efficacy EPs i.e 96% did not require autografting & 83.1% …
Mallinckrodt Reports Results of StrataGraft in P-III STRATA2016 Study for Thermal Burns Read More »
Shots: The companies collaborated to evaluate OncoSec’s TAVO + Merck’s Keytruda vs SoC in a P-III KEYNOTE-C87 trial in ~400 patients with metastatic melanoma who are refractory to immune checkpoint therapy across the US, Canada, EU & Australia The companies will responsible for internal costs with OncoSec covering the third-party costs. The trial is intended …
Shots: The P-II trial will assess the efficacy & safety of RTX across several dose groups vs PBO in patients with a mod. to sev. OAK pain. The study follows the P-Ib trial results of RTX The P-Ib data demonstrated the significant efficacy supporting RTX for long term refractory OA pain, pain relief observed in …
Two months after Eli Lilly began an alliance with MiNA Therapeutics, a startup developing a new type of RNA therapy, the pharmaceutical giant is making a $15 million equity investment in its partner. MiNA is developing a new class of medicines called small activating RNA therapies.
Israel reported that the Pfizer vaccine was 64 percent effective against the Delta variant. But other studies suggest a higher rate.
Provention Bio needs to show the FDA that the drug substance produced from its contract manufacturer is comparable to the Eli Lilly-made drug substance that was evaluated in clinical trials. A larger study testing the prospective type 1 diabetes therapy teplizumab is ongoing and could provide the information the agency needs.
Shots: The company has initiated a P-I open-label study to evaluate the safety, tolerability, PK & preliminary efficacy of HMPL-295 & to determine MTD and RP2D in patients with advanced malignant solid tumors. The 1st patient has been dosed on 02 Jul, 2021 Following the initial dose-escalation stage, 10 to 15 patients will enroll at …
Shots: The P-III ENSURE (1 &2) program evaluates IMU-838 (30mg, qd) vs PBO in ~ 1,050 patients with active RMS. The 1EPs of both trials is time to 1st relapse @72wks. & 2EPs include a volume of T2-lesions, time to confirm disability progression, changes in cognition & measure brain volume The P-II CALLIPER trial assess …
Shots: The P-III study involves assessing Covaxin (COVID-19 vaccine candidate) vs PBO in 25,798 patients aged 18-98 yrs include 2,750 >60 yrs. of age & 7,065 with comorbidities in over 25 centers across India and is being conducted in partnership with ICMR & NIV The results from the P-III study demonstrates overall efficacy of 77.8% …
Ocugen and Bharat Biotech Present Results of Covaxin in P-III Study Against COVID-19 Read More »
Four years after being taken private, Parexel has been sold again, with Goldman Sachs’ private equity arm and EQT paying $8.5 billion to claim ownership of the contract research organisation. The handover comes four years after Pamplona Capital Management bought the company for around $5 billion, and just a few weeks after lab tech company …
Private equity firms play ‘pass the Parexel’ with $8.5bn handover deal Read More »
Shots: The company will present data from the final analysis of P-IIIb STASEY study of Hemlibra (emicizumab) reinforcing the safety & efficacy of Hemlibra in people with hemophilia A with factor VIII inhibitors Spark will share data from the P-I/II study of SPK-8011, demonstrating that hepatocyte expression of factor VIII can be stable and durable …
Shots: The company reported that all the P-I studies assessing insulin aspart (GL-ASP), insulin lispro (GL-LIS), and insulin glargine (GL-GLA) met their primary PK & PD analyses and demonstrated that PK/PD bioequivalence b/w each of the proposed biosimilar and their respective reference compounds The safety profiles were comparable b/w each of the three proposed biosimilar …
France is handing out third shots of the two-dose vaccines to cancer patients and others with immune system impairments. In the United States, patients like these are on their own.
France is handing out third shots of the two-dose vaccines to cancer patients and others with immune system impairments. In the United States, patients like these are on their own.
The Pfizer and Moderna vaccines are safe and effective. Full approval from the F.D.A. will help stop the spread of Covid-19.
The Pfizer and Moderna vaccines are safe and effective. Full approval from the F.D.A. will help stop the spread of Covid-19.
The Pfizer and Moderna vaccines are safe and effective. Full approval from the F.D.A. will help stop the spread of Covid-19.
Acumen Pharmaceuticals’ IPO raised $160 million to fund clinical development of its Alzheimer’s disease drug candidate. Though that drug goes after the same target as Biogen’s recently approved Aduhelm, Acumen says its antibody has potential dosing and safety advantages.
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