How flexible should the U.S. FDA evidentiary standards be for a therapy addressing a significant unmet need in a disease such as amyotrophic lateral sclerosis (ALS)? That’s the question the agency’s Cellular, Tissue and Gene Therapies Advisory Committee will ponder at the Sept. 27 meeting as it looks at the data for Brainstorm Cell Therapeutics Inc.’s Nurown (debamestrocel), a mesenchymal stromal cell therapy targeting ALS. The FDA’s release of its briefing document for the adcom resulted in a nearly 55% drop in Brainstorm’s (NASDAQ:BCLI) value this morning, as shares plunged as low as 45 cents in heavy trading before leveling off a few cents higher. Brainstorm had closed at almost 82 cents on Sept. 22. Just a month ago, the company was trading at $1.66, and its one-year target is $15.
Valo, Novo enter AI cardiometabolic tie-up worth up to $2.7B
Valo Health Inc.’s deal with Novo Nordisk A/S to discover and develop treatments for cardiometabolic diseases brings an up-front payment and a potential near-term milestone reward totaling $60 million. Boston-based Valo is eligible to bank more milestone payments for up to 11 programs, adding up to as much as $2.7 billion, plus R&D funding and would-be royalty payments down the line. The partnership is set up to exploit Valo’s substantial human dataset and computation powered by artificial intelligence.
Newco news: Integra Therapeutics opening a new chapter in gene editing
The rapid migration of gene editing technologies from the bench to the clinic has opened up new therapeutic possibilities for patients with previously intractable genetic diseases and difficult-to-treat cancers. But mobilizing gene editing components into a target cell or organ remains a critical step for the field – and, although real progress has been achieved, at least in certain organ systems, this is still not a trivial problem. Viral and non-viral methods each have their advantages and disadvantages, in terms of toxicity, costs, packaging limits, transduction efficiency and tissue selectivity. Once inside the cell, minimizing or eliminating off-target edits is also crucial. Regardless of the modality chosen, optimizing all of these parameters is a complex engineering challenge. Integra Therapeutics SL, an early stage Spanish firm, is now engaged in that process with a novel gene writing platform.
Scynexis recalls its antifungal Brexafemme
Fearing cross contamination might have occurred during manufacturing, Scynexis Inc. is recalling Brexafemme (ibrexafungerp) from the market and putting a temporary hold on the drug’s clinical trials. The company said it has not received any reports of adverse events. Scynexis became aware of a potential problem with the antifungal for treating vulvovaginal candidiasis after a recent review by its partner GSK plc. The company’s stock (NASDAQ:SCYX) took a dramatic drop on Sept. 25, with shares trading 32% lower at midday at $2.24 each. Ibrexafungerp is in the phase III Mario study for invasive candidiasis and other refractory fungal infections.
Abbvie drops full CD47 collaboration, as I-Mab pushes ahead with phase III
I-Mab Biopharma Co. Ltd. has regained full rights to its CD47 antibody program from Abbvie Inc., including lemzoparlimab, the most advanced candidate. The move, disclosed in a Sept. 22 U.S. SEC filing, eliminates the potential $1.295 billion in milestones associated with the amended collaboration deal signed in 2022.
Chinabio 2023: Big pharma scouts post-pandemic ‘fast China’ for innovation
Despite China’s near-frozen startup scene and increasingly cautious foreign investors following the COVID-19 pandemic, multinational pharmaceutical firms continued to scout for innovative up-and-coming Chinese biotechs in Shanghai at Chinabio Partnering Forum 2023 over its two-day run. On Sept. 21, six biotech startups delivered fast-paced, four-minute pitches at a spotlight competition in hopes of grabbing the panelists’ attention and feedback, along with a 10% discount to the Adock laboratory and a complimentary ticket to an event hosted by the EBD group.
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