Firibastat

With a presidential inauguration and a federal holiday, it wasn’t an enormously busy week for clinical trial news, but there was a fair amount, nonetheless. Read on to see. COVID-19-Related Gritstone Oncology, based in Emeryville, California, announced that it is advancing development of its own second-generation vaccine against COVID-19. It inked a deal with the National Institute of Allergy and Infectious Diseases (NIAID) to launch a Phase I trial. It will be run through the NIAID-supported Infectious Diseases Clinical Research Consortium (IDCRC), with support from the Bill & Melinda Gates Foundation. Innovation Pharmaceuticals received the greenlight from the FDA to initiate a Phase II trial of Brilacidin in hospitalized COVID-19 patients. Brilacidin has shown in vitro to be have antiviral properties against different SARS-CoV-2 strains and other human coronaviruses. The drug appears to inhibit IL-6, IL-1beta, TNF-alpha and other pro-inflammatory cytokines and chemokines. It also has robust antimicrobial properties. Researchers out of Brazil published a small study in the BMJ British Medical Journal that did not find the addition of Roche’s Actemra to standard of care to help the most severe cases. This is a different finding than in several previous trials, including a larger, 800-patient study that found significant benefit. The Brazilian study was designed to determine whether Actemra improved clinical outcomes for patients with severe or critical COVID-19. It was run at nine hospitals in Brazil from May 8 to July 17, 2020. It includes adults with confirmed COVID-19 on supplemental oxygen or mechanical ventilation with abnormal levels of at least two serum biomarkers, C reactive protein, D dimer, lactate dehydrogenase, or ferritin. The data monitoring committee recommended the trial be halted early after 129 patients had been enrolled, because there was an increased number of deaths at 15 days in the Actemra group. Eli Lilly announced that its BLAZE-2 clinical trial conducted with the U.S. National Institute of Allergy and Infectious Diseases (NIAID), of bamlanivimab (LY-CoV555) decreased the risk of COVID-19 among residents and staff of long-term care facilities, i.e., nursing homes. It evaluated 965 participants who tested negative for COVID-19 at baseline, 299 residents and 666 staff. All participants randomized to receive either 4,200 mg of bamlanivimab, Lilly’s neutralizing antibody, or placebo. Non-COVID-19-Related Biohaven Pharma completed a focused analysis of topline co-primary and key secondary data from its Phase II/III trial of troriluzole as a symptomatic treatment in mild-to-moderate Alzheimer’s disease. Additional secondary and exploratory efficacy analyses and biomarker data including neurofilament light chain (NfL), neurogranin, tau and amyloid are still pending. The drug did not differ statistically from placebo at 48 weeks on the prespecified co-primary endpoints on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 and the Clinical Dementia Rating Scale Sum of Boxes. Nor did it differentiate from placebo on the key secondary measure of hippocampal volume assessed by MRI. Celyad Oncology updated its Phase I alloSHRINK trial of CYAD-101 for refractory metastatic colorectal cancer. CYAD-101 is the company’s allogeneic NKG2D-receptor and T cell receptor inhibitory molecule-based, non-gene edited CAR-T candidate. It is being administered together with FOLFOX chemotherapy. Two patients achieved a confirmed partial response and nine patients achieved stable disease, with seven demonstrating disease stabilization lasting more than or equal to three months of duration. Ipsen announced results from new analyses of its pivotal Phase III trial to evaluate treatment intervals over repeat cycles of Dysport (abobotulinumtoxinA) in five patient populations. It shared 26 abstracts at the International Neurotoxin Association, including updates from the recently published surveys, data from the Phase IV ULIS-III trial, and 10 abstracts on basic science research into neurotoxins. Caladrius Biosciences treated the first patient in its Phase IIb FREEDOM trial of CLBS16 for coronary microvascular dysfunction. The trial will investigate 105 patients for the drug, which is made up of autologous CD34+ cells. Verrica Pharmaceuticals presented positive data from post-hoc pooled analyses of the pivotal Phase III CAMP trial of VP-102 in molluscum contagiosum. Patients treated with the drug with lesions in the upper extremities, head/neck, back/buttocks, and chest/abdomen at baseline demonstrated statistically significantly higher rates of complete clearance compared to the placebo group. VP-102 is a proprietary drug-device combination that contains a GMP-controlled formulation of cantharidim delivered by way of a single-use applicator. Brickell Biotech announced efficacy and safety data from its Phase III trial with Kaken Pharmaceutical of sofpironium bromide in Japanese patients with primary axillary hyperhidrosis. Hyperhidrosis is a severe case of sweating. Transgene and BioInvent received approval from France’s national regulatory agency to begin a Phase I/IIa trial of Vaccinia virus BT-001 in solid tumors. BT-001 is based on the patented Invir.IO oncolytic virus (VVcopTK-RR-) and engineered to encode both a Treg-depleting human recombinant anti-CTLA4 antibody and the human GM-CSF cytokine. Quantum Genomics launched its Phase III REFRESH trial in difficult-to-treat or resistant hypertension. The trial will evaluate a single dose of firibastat 1000mg compared to placebo, on top of the patients’ current therapy. Evelo Biosciences announced new positive confirmatory data from its Phase Ib trial of EDP1815 in mild and moderate atopic dermatitis. The drug was well tolerated and demonstrated clinically meaningful improvement in the Dermatology Life Quality Index (DLQI) and Patient-Oriented Eczema Measure IPOEM). The drug is an oral therapy for inflammatory diseases and effects multiple pathways, TH1, Th2 and Th17. Ocuphire Pharma published results from its ORION-1 Phase II trial of Nyxol in glaucoma and presbyopia. The drug produced statistically significant 20% mean reduction or about 1 mm in pupil diameters under daytime and nighttime lighting conditions, and 60% demonstrated statistically significant improvement in a 1-line or greater in near visual acuity compared to 20% on placebo. Histogen announced the FDA had verbally notified the company it had additional questions about its IND application for the planned Phase I/II trial of HST-003 for knee cartilage regeneration. The drug is a human extracellular matrix intended to regenerate hyaline cartilage for damaged joints. The company has not yet received written notice of its clinical hold from the FDA. INmune Bio announced results from its Phase Ib trial of XPro1595 in Alzheimer’s disease. The drug decreased biomarkers of neuroinflammation across multiple measures and assays, including cerebrospinal fluid and MRI. XPro1595 is a dominant-negative protein that neutralizes soluble TNF, which is to say, it’s a next-generation tumor necrosis factor inhibitor, but doesn’t behave the way currently existing TNF inhibitors do. Transgene dosed the first patient in its Phase I trial of TG4050 (myvac Platform) in a patient with head and neck cancer. TG4050 is an individualized immunotherapy for solid tumors based on the company’s myvac technology and leveraging NEC’s artificial intelligence expertise. It is a virus-based therapeutic vaccine that encodes neoantigens picked by NEC’s Neoantigen Prediction System. GlaxoSmithKline halted and discontinued its Phase II trial of GSK2831781 for ulcerative colitis. The drug was licensed from Immutep Limited. The drug is a monoclonal antibody designed to deplete recently activated LAG3 expressing T-cells in T-cell driven immuno-inflammatory disorders. The trial was halted and abandoned after an interim evaluation by an IDMC. 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PARIS and NEW YORK, Jan. 18, 2021 (GLOBE NEWSWIRE) -- Quantum Genomics (Euronext Growth - FR0011648971 - ALQGC), a biopharmaceutical company specializing in developing a new drug class that directly targets the brain to treat difficult-to-treat and resistant hypertension and heart failure today announced the launch of its Phase III REFRESH study in difficult-to-treat(1) or resistant(2) hypertension. This new study is part of firibastat’s Phase III clinical development and aims to assess long-term safety as well as the three-month efficacy after a single daily dose of firibastat 1000mg in treatment resistant hypertensive patients. “We are thrilled to launch this new pivotal study as a precursor to potential market approval of single dose firibastat, which we believe to be the best regimen for chronic treatment of resistant of difficult-to-treat hypertension,” said Jean-Philippe Milon, Chief Executive Officer at Quantum Genomics. “This study will also provide long-term safety data in accordance with Quantum Genomics’ development plan endorsed by the FDA.” (3) This multicenter, multinational study will enroll 750 patients with difficult-to-treat or resistant hypertension in Europe, Canada, US as well as collaboratively in Asia with our commercial partners in China, Taiwan and South-Korea. For the first randomized three-month period, 750 patients will receive firibastat (1000mg once-a-day) or placebo, on top of their current therapy. The primary endpoint will be reduction in systolic automated office blood pressure (AOBP) from baseline. After this first period, subjects will be treated and followed-up for six months (and 12 months for 100 patients) to assess long-term safety, data that are mandatory for New Drug Application (NDA) for a chronic treatment. Sites selection will start shortly, and regulatory submissions to competent authorities and ethics committees are in preparation for each country. First patient recruitment is planned in Q2 2021 and efficacy results and six-month safety results are expected for mid-2023, on time to submit New Drug Applications in 2023. Study costs are budgeted and financed thanks to the partnerships signed by Quantum Genomics. (1) Patients not controlled despite two antihypertensive classes, including a diuretic, at maximum tolerated doses. (2) Patients not controlled despite at least three antihypertensive classes, including a diuretic, at maximum tolerated doses. (3) FDA: Food and Drug Administration, US Regulatory Authority. About Quantum Genomics Quantum Genomics is a biopharmaceutical company specializing in the development of a new class of cardiovascular medications based on brain aminopeptidase A inhibition (BAPAI). Quantum Genomics is the only company in the world exploring this innovative approach that directly targets the brain. The company relies on 20 years of academic research from the Paris-Descartes University and the laboratory directed by Dr. Catherine Llorens-Cortes at the Collège de France (French National Institute of Health and Medical Research (INSERM)/ the Scientific Centre for National Research (CNRS)). The goal of Quantum Genomics is to develop innovative treatments for complicated, or even resistant, cases of hypertension (around 30% of patients have poor control of their condition or receive ineffective treatment) and for heart failure (one in two patients diagnosed with severe heart failure dies within five years). Based in Paris and New York, Quantum Genomics is listed on the Euronext Growth exchange in Paris (FR0011648971- ALQGC) and trades on the OTCQX Best Market in the United States (symbol: QNNTF). For more information, please visit , or follow us on Twitter and LinkedIn Contact