In a panel discussion with executives at the 2024 BIO CEO conference this week in New York, the consensus emerged that artificial intelligence is here to stay, despite its occasional moments of hype, as its applications continually grow.
Is the U.S. Inflation Reduction Act with its Medicare drug price negotiation provision the new legislative sacred cow that cannot be tweaked? Debate over whether the orphan drug carveout included in the negotiation provision should be extended to drugs with more than one rare disease indication was the major discord in an otherwise bipartisan discussion the House Energy and Commerce Subcommittee on Health had in a hearing held Feb. 29 in observation of Rare Disease Day.
San Diego-based Kenai Therapeutics Inc. raised $82 million in a series A round to move its disease-modifying cell therapy for Parkinson’s disease into the clinic. The company, which leverages induced pluripotent stem cell (iPSC) technology, will advance its next-generation allogeneic neuron replacement cell therapies for neurological diseases, specifically completing a clinical proof-of-concept trial for its lead candidate, RNDP-001.
Positive phase III data from Ironwood Pharmaceuticals Inc. with once-weekly glucagon-like peptide-2 analogue apraglutide failed to impress Wall Street, and shares of the Boston-based firm (NASDAQ:IRWD) closed Feb. 29 at $9.43, down $5.69, or 37.6%. The experiment called Stars tested apraglutide to reduce parenteral support in adults with short bowel syndrome with intestinal failure. Rare and severe, the condition affects about 18,000 adults in the U.S., Europe and Japan. Based on the latest data, Ironwood plans to submit an NDA to the U.S. FDA and other regulatory filings.
In 2023, the BioWorld Infectious Disease Index (BIDI) concluded with a 43.48% decline, showing improvement from the 83.57% drop observed in 2022. While an uptick was seen from October's year-to-date performance, down 47.98%, BIDI still lagged both the Nasdaq Biotechnology Index and the Dow Jones Industrial Average for the year, which saw gains of 3.74% and 13.7%, respectively.
Pharmaust Ltd.’s monepantel met its primary safety endpoints and showed positive signals of potential efficacy in a phase I trial in patients with motor neuron disease (MND)/amyotrophic lateral sclerosis (ALS). With these results, the company will now progress to a pivotal phase II/III trial by midyear, Pharmaust CEO Michael Thurn told BioWorld.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Applied Therapeutics, Arthex, Hyloris, Incyte, Lutris, Spero, UCB.
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Arcutis, Beigene, Cassava, Derm-Biome, Dyadic, Enveric, Immpact, Kadimastem, Nicox, Obseva, Sato, Shoreline.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Akeso, Biolinerx, Gain, Incannex, Oculis, Palatin, Pharmaust, Redx, Skyhawk, Tonix.
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