Sanofi, Regeneron asthma prospect struggles against Dupixent

fiercebiotech | June 21, 2019

A phase 2 asthma trial of Sanofi and Regenerons REGN3500 has met its primary endpoint. But the IL-33 antibody performed numerically worse than Dupixent and showed little promise in combination with the approved drug. Sanofi and Regeneron are putting REGN3500, also known as SAR440340, through a suite of asthma, atopic dermatitis and chronic obstructive pulmonary disease clinical trials to assess the effect of inhibiting the IL-33 protein in these patient populations. IL-33 is thought to be central to type 1 and type 2 inflammation, leading the partners to conclude REGN3500 may improve outcomes in a range of diseases. The asthma trial enrolled 296 people with moderate-to-severe forms of the disease that were poorly controlled by inhaled corticosteroid and long-acting beta-agonist therapy, making them suitable candidates for biologic intervention. REGN3500 outperformed placebo in terms of the proportion of patients who suffered loss of asthma control, resulting in the trial hitting its primary endpoint. And it triggered statistically significant gains in lung function as measured by FEV1. The comparison to Dupixent, which won approval in asthma last year, was less flattering. While the trial was not powered to show differences between the active treatment arms, Sanofi and Regeneron noted patients who received Dupixent performed numerically better than their peers on REGN3500 across all endpoints. A Dupixent-REGN3500 combination did no better than Dupixent monotherapy. Sanofi and Regeneron are yet to share efficacy data from the trial, but the top-line findings suggest REGN3500 may struggle to supersede Dupixent. The partners noted that REGN3500 worked best in patients with blood eosinophil levels above 300 cells/microliter. But Dupixent is also most effective in this subpopulation, raising doubts about the scope for REGN3500 to carve out a space in the indication.

Spotlight

CRISPR or clustered regularly inter-spaced short palindromic repeats has become a pretty big deal in the world of biotechnology. In this video we will take a look at the very basics of CRISPR and a couple of examples of its applications so that we can build a base understanding of the concepts of this form of biotechnology which can revolutionize gene therapy and genomics.

Spotlight

CRISPR or clustered regularly inter-spaced short palindromic repeats has become a pretty big deal in the world of biotechnology. In this video we will take a look at the very basics of CRISPR and a couple of examples of its applications so that we can build a base understanding of the concepts of this form of biotechnology which can revolutionize gene therapy and genomics.

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MEDTECH

Versantis to Present Positive Phase 1b Data at AASLD for VS-01 in Patients with Decompensated Cirrhosis

Versantis | November 15, 2021

Versantis, a clinical-stage biotechnology company developing novel therapies for orphan liver and pediatric diseases, announced that positive Phase 1b clinical data from a study of its lead investigational, orphan-designated product, VS-01, in 12 hospitalized patients with decompensated liver cirrhosis will be presented at the American Association for the Study of Liver Diseases (AASLD) annual meeting. The data show that VS-01 was safe and well tolerated in these patients, and demonstrated promising early indications of efficacy in this clinical study. VS-01 is a potentially lifesaving, multi-organ support therapy that aims to timely reverse Acute-on-Chronic Liver Failure (ACLF) by enhancing the clearance of ammonia and other toxins following paracentesis. The Abstract selected for oral presentation is as follows Oral Presentation Title: Safety and Preliminary Efficacy and Pharmacokinetics of Intraperitoneal VS-01 Infustions in Patients with Decompensated Liver Cirrhosis: A First-in-Human, Open-label, Phase 1b Clinical Trial Presenter: Dr. Frank Erhard Uschner, Section for Translational Hepatology, Department of Internal Medicine I, Goethe University Clinic Frankfurt, Session Date/Time: Monday, November 15, 2021, 12:30 p.m. EST The annual Liver Meeting, held by AASLD, brings together attendees from around the world to exchange the latest research, discuss new developments in treatments, and network with others in the field. The primary objective of this single-center first-in-human study was to evaluate the safety and tolerability of i.p.-administered VS-01 on top of standard of care in cirrhotic patients with ascites and mild hepatic encephalopathy following single and multiple intraperitoneal administrations. The secondary objectives were to gather preliminary PK, PD, and clinical efficacy data. In total, all 12 patients completed treatment in the Department of Internal Medicine I, Goethe University Frankfurt and were assigned to receive either a single dose of VS-01 (Part A; n=9) or four consecutive doses (Part B; n=3). Following the drainage of ascites in these patients, VS-01 was then infused into the peritoneal cavity via the existing paracentesis catheter and removed after a dwell time of 2-3 hours. No treatment-related serious adverse events were reported and no patient discontinued treatment due to an adverse event. VS-01 also demonstrated promising clinical efficacy results, including a high and dose-dependent ammonia clearance, promising improvement in hepatic encephalopathy based on psychometric tests, and increased peritoneal clearance of ACLF-related metabolites. “The data show that VS-01 is safe and well tolerated in cirrhotic patients with ascites and covert (mild) hepatic encephalopathy, so very promising. We were able to administer VS-01 using standard hospital equipment via the therapeutic paracentesis catheter, which we believe can easily be incorporated into standard of care for patients There are very few treatments available for these patients and VS-01 is complementary to those, so we are excited to continue advancing it’s development and hopefully generate the data supporting this ground-breaking clinical approach.” Prof. Dr. Trebicka, the principal investigator and head of the Section of Translational Hepatology in Goethe University Frankfurt “VS-01 represents a promising new therapeutic for the potential treatment of patients with ascites and acute complications of cirrhosis. The data from this early study were very promising and importantly show that VS-01 appears to be safe and well tolerated in these patients. The amomonia and ACLF metabolites clearance data is particularly encouraging,” added Vincent Forster, CEO and co-Founder of Versantis. “The successful completion of this study supports future development of VS-01. We are now preparing to initiate a multi-center Phase 2a study in patients with ACLF, a seriously underserved and under-resourced indication. Together with our pipeline of innovative products, we are committed to developing and commercializing new treatment options for patients suffering from acute liver diseases.” About Acute-on-Chronic Liver Failure (ACLF) ACLF is an underserved medical condition, which, despite best possible available care, is associated with high short-term mortality. It is characterized by an abrupt life-threatening worsening of a pre-existing chronic liver disease (e.g., cirrhosis) resulting in liver and extrahepatic organ failure rapidly progressing into coma and death. Every year at least 150’000 patients are hospitalized with ACLF in the US and EU. The incidence is growing due to a higher prevalence of diabetes, obesity, fatty liver diseases, and alcohol consumption. By timely reversing ACLF and the multi-organ complications arising from cirrhosis, VS-01 aims to improve outcomes in these patients and relieve the growing health and economic burden of this advanced liver disease. About Versantis Versantis is a clinical stage biotechnology company focused on addressing the growing, un-met medical need in liver diseases. With a pipeline of drug and diagnostic products covering chronic and acute indications, Versantis believes it can revolutionize current standard of care for patients suffering from acquired and genetic hepatic deficiencies. Versantis’ lead program, VS-01, is in clinical development as a first-line therapy for the timely reversal of acute-on-chronic liver failure (ACLF). It harnesses Versantis’ proprietary scavenging liposomes to extract toxins from the body and, if approved, will be the first drug to take advantage of the intraperitoneal route to potentially support the liver, kidneys and brain, the organs that most often fail in cirrhotic patients. VS-01 has received orphan drug designation (ODD) from the EMA and U.S. FDA, as well as a Rare Pediatric Diseases Designation from the U.S. FDA for Urea Cycle Disorders. Founded by scientists from ETH Zurich with entrepreneurial drive, Versantis has built a team and Board of seasoned industry executives with a proven ability to advance novel therapies from the idea stage into clinical development. The company is headquartered in Zurich, Switzerland.

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MEDICAL

GT Molecular, Rapidly Expanding Molecular Diagnostics Biotech, Launches Two Highly Sensitive Tests for the Dangerous UK Variant of SARS-CoV-2

GT Molecular | January 13, 2021

GT Molecular, a quickly extending sub-atomic diagnostics biotech, has dispatched two profoundly delicate tests for the risky UK Variant of SARS-CoV-2 (B.1.1.7). These tale examines expand upon GT Molecular's clinical foundation and technology for profoundly exact and solid qPCR and advanced PCR recognition of SARS-CoV-2 virus and are offered in two formats. The fee-for-service testing of wastewater for the presence of SARS-CoV-2 and the variations available for use furnishes networks with a quick, exceptionally savvy technique to comprehend the measure of viral transmission inside a local area. Everybody inside a local area gives an example as a flush to the wastewater treatment office and a solitary wastewater test can cover a large number of individuals for a simple portion of what a clinical test for an individual expenses. Moreover, the presence of the infection in wastewater can go before nearby flare-ups locally by as long as seven days, giving wellbeing authorities time to prepare and increase testing. By applying this technique for local area wastewater checking to the recently coursing UK variation, GT Molecular is the first to go above and beyond and really "genotype the sewer".

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Equillium Acquires Bioniz Therapeutics Significantly Expanding Pipeline of Novel Immunomodulatory Drug Candidates

Equillium, Inc. | February 17, 2022

Equillium, Inc. a clinical-stage biotechnology company focused on developing novel therapeutics to treat severe autoimmune and inflammatory disorders with high unmet medical need, today announced that it has acquired Bioniz Therapeutics, Inc., a privately held clinical-stage biotechnology company. Bioniz developed its novel structured-domain peptides, including BNZ-1 and BNZ-2, entirely in-house from its proprietary product discovery platform. The Bioniz lead product candidates are multi-specific inhibitors of key disease-driving, clinically validated cytokine targets aimed at addressing unmet needs across a range of immuno-inflammatory indications. Transaction Details With the acquisition of Bioniz, Equillium obtained exclusive worldwide rights to all current and future Bioniz products. The transaction consideration is comprised of an all-stock upfront payment of 5,699,492 unregistered shares of common stock of Equillium issuable to Bioniz stockholders, which represents approximately 19.3% of Equillium’s outstanding capital stock. Approximately 97% of the issued Equillium stock is subject to a standard 6-month lock-up provision and thereafter will be released ratably each month over the following 6 months. Equillium is also obligated to pay Bioniz stockholders up to $57.5 million in potential development milestone payments across three Bioniz clinical candidates (BNZ-1, BNZ-2, and BNZ-3) beginning upon first U.S. marketing approval, and up to $250 million in sales milestone payments based upon BNZ-1 achieving calendar year global net sales of $500, $1,000, $1,500, and $2,000 million. Other than the aforementioned milestones, Equillium does not have any third-party obligations with respect to milestones or royalties related to the Bioniz products or platform. Equillium has sole discretion over the timing and extent of advancing clinical development of the Bioniz products. Bioniz was estimated to have modestly positive net working capital at closing. Webcast and Conference Call Management will host a conference call accompanied by a slide presentation to discuss the acquisition of Bioniz for analysts and institutional investors, at 8:00 am ET today, February 16, 2022. To access the call, please dial (888) 350-3846 or (646) 960-0251 and, if needed, provide confirmation number 8770084. A live webcast of the call will also be available on the company’s Investor Relations page at https://ir.equilliumbio.com/events-and-presentations. The webcast will be archived for 180 days. About Itolizumab Itolizumab is a clinical-stage, first-in-class anti-CD6 monoclonal antibody that selectively targets the CD6-ALCAM pathway. This pathway plays a central role in modulating the activity and trafficking of T cells that drive a number of immuno-inflammatory diseases. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited. About Bioniz Bioniz is a clinical-stage biopharmaceutical company developing precision cytokine targeted therapies for the treatment of immuno-inflammatory diseases. Bioniz’s platform incorporates world class expertise in cytokine biology to advance a novel therapeutic approach to selectively inhibit functionally redundant cytokines while leaving the rest of the cytokine network intact. Bioniz is developing a robust pipeline of product candidates in multiple autoimmune indications. About Equillium Equillium is a clinical-stage biotechnology company leveraging deep understanding of immunobiology to develop novel products to treat severe autoimmune and inflammatory disorders with high unmet medical need. Equillium is developing itolizumab for multiple severe immuno-inflammatory diseases, including acute graft-versus-host-disease (aGVHD), lupus/lupus nephritis and uncontrolled asthma.

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