Legend Biotech Corp. is entering what it no doubt hopes will be another fruitful collaboration with a much larger company. Legend’s wholly owned subsidiary, Legend Biotech Ireland Ltd., will work with Novartis AG in an exclusive global development and license agreement for chimeric antigen receptor T-cell therapies targeting DLL3. Legend is getting $100 million up front and could receive up to $1 billion in milestones and royalties. The deal includes LB-2102, a CAR T-cell candidate that Legend has in an interventional phase I study to treat extensive stage small-cell lung cancer or large-cell neuroendocrine lung cancer. Legend did well in a similar collaboration with Janssen Biotech Inc. that began in 2017 and led to the U.S. approval of Carvykti (ciltacabtagene autoleucel) for treating multiple myeloma. Legend received an up-front $350 million in the deal and brought in a $50 million milestone payment in April 2022.
Aclaris’ oral MK2 inhibitor fizzles in RA phase II study
Shares of Aclaris Therapeutics Inc. (NASDAQ:ACRS) plunged to trade midday at 62 cents, down $4.14, or 87%, after the firm disclosed top-line data from a phase II study with oral MK2 inhibitor zunsemetinib (ATI-450) in moderate to severe rheumatoid arthritis (RA). The drug fell short of primary and secondary endpoints. Wayne, Pa.-based Aclaris is quitting development of the compound.
Adverse events dampen early readout for Verve’s cholesterol-lowering base-editing approach
Verve Therapeutics Inc. grabbed headlines over the weekend, with a presentation at the American Heart Association Scientific Sessions highlighting the first human proof-of-concept data for a single-course in vivo base-editing treatment. Treatment with VERVE-101 led to promising dose-dependent reductions in low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolemia. However, as the markets opened Monday morning, the company’s stock (NASDAQ:VERV) took a hit, as investors focused on safety data from the first 10 patients, which included reports of elevated liver transaminases in one patient and a myocardial infarction in one patient deemed potentially related to treatment.
Wegovy cuts down on heart attacks, shows diverse cardio benefits
More details of Novo Nordisk A/S’ phase III Select trial indicate that risk reductions in major adverse cardiovascular events (MACE) were achieved regardless of age, gender, ethnicity and starting body mass index when obese and overweight patients were treated with Wegovy (semaglutide, 2.4 mg). Statistical significance was seen, however, only with the reduction of heart attacks and not other measures that were part of the trial. BioWorld reported on the top-line results in August showing the GLP-1 receptor agonist reduced the risk of MACE by a statistically significant 20%, sending shares (NYSE:NVO) of the Copenhagen, Denmark-based company up by 17.2% on Aug. 8 . The new data were released Nov. 11 at the American Heart Association Scientific Sessions in Philadelphia and simultaneously published in the New England Journal of Medicine.
SfN 2023: Lessons from Huntington’s successes, and failures
The gene for Huntington’s disease (HD) “was cloned in 1993, and everyone thought there was going to be a treatment right around the corner,” Sarah Tabrizi told the audience at the 2023 Annual Meeting of the Society for Neuroscience (SfN). Then, “it took 25 years for the first trial targeting the Huntington gene.” Tabrizi, who is the co-founder and director of the University College of London’s Huntington’s Disease Center, gave one of the presidential plenary lectures at the meeting. Compared to the complex and still puzzling genetics of other dementias, HD is straightforward. It is a monogenic disorder that results from a triplet expansion in the huntingtin gene. Theoretical considerations as well as large volumes of preclinical data suggested that targeting that triple expansion would be a disease-modifying therapy for HD.
Immvira moves intravenous oHSV into phase II for cancer
Immvira Group Co. plans to progress its intravenous oncolytic herpes simplex virus (oHSV) MVR-T3011-IV to phase II trials after completing a phase I study in the U.S. in patients with late-stage solid tumors. The Shenzhen, China-based company is the first to conduct clinical trials via intravenous administration of oHSVs. “Intravenous administration of oncolytic viruses has long been a challenge, as most oncolytic viruses cannot overcome multiple obstacles such as neutralization by innate immune response, and they can also potentially lead to cytokine storm in the body,” Immvira Chief Financial Officer Carl Yeung told BioWorld in an earlier interview.
US Senate patent reform bill draws mixed reviews
The America Invents Act of 2011 was designed to provide a durable overhaul of the U.S. patent system, but the inter partes review process has drawn fire from inventors as a patent-killing machine. The Senate is considering a new bill to address some of these concerns, but witnesses at a hearing this week were anything but united in their assessment of the status quo, making it difficult to forecast the fate of this latest effort at patent reform. The Senate Judiciary Committee’s subcommittee for intellectual property convened Nov. 8, with committee chairman Sen. Chris Coons (D-Del.) stating that small inventors are routinely subject to predatory litigation from one or more larger entities.
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