GRF-6019

It was a busy week for clinical trial news. Read on for more information. COVID-19-Related Johnson & Johnson announced topline efficacy and safety data from the Phase III ENSEMBLE trial of its single-dose COVID-19 vaccine in development by Janssen Pharmaceutical Companies. It met all primary and key secondary endpoints. The data suggested the vaccine was 66% effective overall in preventing moderate to severe COVID-19, 28 days after vaccination, with protection observed as early as day 14. There were variations in country-to-country studies against moderate to severe COVID-19 infection 28 days after vaccination: U.S. (72%); Latin America (66%); South Africa (57%). Against severe disease, it was 85% effective in all regions 28 days after vaccination, with efficacy increasing over time with no cases reported after day 49. It demonstrated complete protection against COVID-19-related hospitalization and death, 28 days after vaccination. Merck announced that it was going to stop developing its two COVID-19 vaccine candidates, dubbed V590 and V591, after poor responses in the Phase I trials. They are shifting their attention to advancing two therapeutic drugs for COVID-19, MK-4482 and MK-7110. Merck is developing MK-4482 with Ridgeback Biotherapeutics. In the Phase I trials, V590 and V591 were generally well tolerated, but the company reports that immune responses were inferior to what was observed in people who recovered naturally from COVID-19 infections. Eli Lilly and Company announced the results of its Phase III BLAZE-2 COVID-19 prevention trial, conducted with the U.S. National Institute of Allergy and Infectious Diseases (NIAID) and the COVID-19 Prevention Network (CoVPN). The trial evaluated the company’s bamlanivimab (LY-COV555), its neutralizing antibody, in COVID-19 in residents and staff at nursing homes in the U.S. At eight weeks of follow-up, there was a significantly lower frequency of symptomatic COVID-19 in the bamlanivimab treatment arm compared to placebo. Eli Lilly’s bamlanivimab and esesivimab together significantly decreased COVID-19-related hospitalizations, hitting the primary endpoint of the Phase III BLAZE-1 trial. Bamlanivimab is a recombinant, neutralizing human IgG1 monoclonal antibody directed against the COVID-19 virus spike protein. Etesevimabis a recombinant fully human monoclonal neutralizing antibody that specifically binds to the SARS-CoV-2 surface spike protein receptor binding domain with high affinity and can block the binding of the virus to the ACE2 host cell surface receptor. Achiko AG and Regenacellx.sl announced the completion of their Phase I trial of its nanoparticulate DNA aptamer conjugate and diagnostic testing technology, Project Gumnuts. The COVID-19 testing study demonstrated in field tests 80% sensitivity and 63% specificity. Applying software and machine learning increased it to 91% sensitivity and 85% specificity. Regeneron Pharmaceuticals announced positive initial data from its ongoing Phase III trial of its antibody cocktail, REGEN-COV, as a passive vaccine to prevent COVID-19 in people at high risk of infection from household exposure. The trial is being run with NIAID. Exploratory analysis found that the cocktail demonstrated 100% prevention of symptomatic infection in the 400 patients that could be evaluated. There was also about a 50% lower overall rate of infection, symptomatic and asymptomatic. The lower number of infections in the group on the cocktail were all asymptomatic, with decreased peak virus levels and a short duration of viral shedding, suggesting they were less likely to spread the disease. Vir Biotechnology, Eli Lilly and GlaxoSmithKline inked a three-way deal to evaluate Vir’s monoclonal antibody, VIR-7831, in combination with Lilly’s bamlanivimab in low-risk mild to moderate COVID-19 patients. Lilly expanded its ongoing BLAZE-4 study to include Vir’s compound. Vir and GSK were already partnered on the development of VIR-7831. Dr. Reddy's Laboratories and Global Response Aid FZCO terminated its Avigan Trial Study in Kuwait focused on moderate to severe COVID-19 patients in a hospital setting. The Phase III study in outpatient setting in North America in partnership with Appili Therapeutics and Global Response Aid will continue. The Kuwait study did not show a statistically significant difference for the primary endpoint for Avigan compared to placebo. Avigan (favipiravir) is an antiviral drug marketed by Fujifilm Toyota Chemical Co. Valneva completed enrollment in its Phase I/II trial of its COVID-19 vaccine candidate. VLA2001 is an inactivated vaccine candidate, meaning it uses an inactivated SARS-CoV-2 virus with an adjuvant to further stimulate the immune system. Initial data is expected in April. EmitBio announced that its clinical trial of EmitBIo device achieved a 99.9% reduction in average viral load of SARS-CoV-2 and accelerated time to symptom resolution by more than 48 hours compared to the control group. The device uses a small handheld device that directs precise wavelengths of safe, visible, targeted light to the back of the throat and surrounding tissues. Novavax reported that NVX-CoV2373, its COVID-19 vaccine, demonstrated an efficacy of 89.3% in its Phase III trial in the UK. It also announced positive data from its Phase IIb trial in South Africa. Non-COVID-19-Related Roche announced topline data from two identical Phase III trials, TENAYA and LUCERNE, of faricimab in neovascular or “wet” age-related macular degeneration (nAMD). Both trials hit their primary endpoint, demonstrating visual acuity outcomes that were non-inferior to aflibercept injections. They received faricimab injections at fixed intervals up to every 16 weeks compared to every eight weeks for aflibercept. Aflibercept is marketed by Regeneron Pharmaceuticals as Eylea. Faricimab is a bispecific antibody, meaning it is designed to recognize two different antigens. In this case, it targets angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A), both of which are associated with several diseases of the retina. Abeona Therapeutics announced it held a successful Type B meeting with the FDA over its co-primary endpoints for the pivotal Phase III VIITAL trial of EB-101 in recessive dystrophic epidermolysis bullosa (RDEB). The drug is an autologous, gene-corrected cell therapy. RDEB is a rare connective tissue disorder with no approved therapies. Vir Biotechnology announced initial topline data from its ongoing Phase I trial of VIR-3434 in chronic hepatitis B virus (HBV) infection. The early data demonstrated a mean reduction in serum hepatitis B virus surface antigen by day eight. VIRI-3434 is an HBV-neutralizing monoclonal antibody designed to block entry of all 10 genotypes of HBV into hepatocytes and decrease the level of viral and subviral particles in the blood. Rhythm Pharmaceuticals announced positive proof-of-concept data from multiple cohorts in its Phase II Basket Study of setmelanotide in patients with severe obesity due to genetic variants in the melanocortin-4 receptor (MC4R) pathway. Setmelanotide (Imcivree) is indicated for chronic weight management in adults and children age six years and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilism/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. Exilixis initiated the first-in-human Phase I trial of XL1-2 alone or in combination with other therapies in patients with inoperable locally advanced or metastatic solid tumors. XL102 is a potent, selective and orally bioavailable inhibitor of cyclin-dependent kinase 7 (CDK7). Pfizer announced co-primary endpoint data from a recently wrapped post-market safety study of tofacitinib at two doses compared to a TNF inhibitor in patients with rheumatoid arthritis who were 50 years of age or older with at least one additional cardiovascular risk factor. The co-primary endpoints were non-inferiority of tofacitinib compared to TNFi in relation to major adverse cardiovascular events (MACE) and malignancies (excluding non-melanoma skin cancer (NMSC). The non-inferiority criteria were not met for the endpoints, with no evidence of a difference between the two tofacitinib treatment groups. Agios Pharmaceuticals reported its Phase III ACTIVATE-T trial of mitapivat in regularly transfused adults with PK deficiency demonstrated a statistically significant and clinically meaningful decrease in transfusion burden. Mitapivat is a first-in-class oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes. NervGen Pharma Corp announced it was adding an Alzheimer’s disease patient cohort to its Phase I trial of NVG-291. The Phase I program was originally going to be in healthy volunteers. The drug is a specific and selective protein tyrosine phosphatase sigma inhibitor. EyePoint Pharmaceuticals dosed the first patient in its Phase I trial of EYP-1901 as a potential twice-yearly sustained delivery anti-VEGF treatment for wet age-related macular degeneration (wet AMD). The drug uses the company’s Durasert drug delivery technology that is already approved in four FDA-approved products. Organicell Regenerative Medicine announced the FDA had approved its IND for Zofin in COPD. The approved Phase I/II clinical trial will evaluate the safety and efficacy of IV Zofin for COPD. Zofin is an acellular, biologic derived from perinatal sources and is engineered to retain naturally occurring microRNAs. Takeda Pharmaceutical announced new data from the Phase I/II trial of TAK-788 (mobocertinib) in previously treated patients with EGFR Exon20 insertion+ metastatic non-small cell lung cancer (mNSCLC). The data showed the drug resulted in clinically meaningful responses and a “noteworthy” duration of response in this patient population. Mobocertinib is an oral tyrosine kinase inhibitor (TKI) designed to target EGFR Exon20 insertion mutations. Alklahest published data from its Phase II trial of GRF6019 for Alzheimer’s disease. It met the primary endpoint, showing both dose levels were safe and well tolerated. Patients also demonstrated a lack of cognitive decline and limited functional decline over the 24-week study period. The drug is a proprietary plasma fraction that has enhanced neurogenesis and improved age-related deficient in learning and memory, as well as decreased neuroinflammation in preclinical animal studies. Orion Biotechnology Canada presented results from their Phase I trial of OB-002H at the HIVR4P virtual meeting. It evaluated OB-002h Gel for prevention of HIV. OB-002 is a CCR5 inhibitor with best-in-class in vitro potency and is also being studied in oncology and COVID-19. Amgen announced results from the Phase II cohort of the CodeBreaK 100 trial of sotorasib in 126 patients with KRAS G12C-mutated advanced non-small cell lung cancer (NSCLC). The drug demonstrated a confirmed objective response rate (ORR) and disease control rate (DCR) of 37.1% and 80.6%, respectively, and a median duration of response of 10 months. Sotorasib is an investigational KRASG12C inhibitor. Janssen Pharmaceutical Companies, of J&J, announced new data from the Phase I CHRYSALIS trial of amivantamab in metastatic or unresectable non-small cell lung cancer (NSCLC) and EGFR exon 20 insertion mutations whose disease progressed on or after platinum-based chemotherapy. The drug is an investigational, fully-human EGFR and MET bispecific antibody. The findings demonstrated robust activity and durable responses. Most Read Today