Ironwood Pharmaceuticals Inc. shares (NASDAQ:IRWD) were trading midday at $9.28, down $5.84, or 38%, after the Boston-based firm offered top-line results from its pivotal phase III Stars trial, which tested the efficacy and safety of once-weekly subcutaneous apraglutide in reducing parenteral support dependency in adult patients with short-bowel syndrome with intestinal failure (SBS-IF). The Stars outcome were positive enough that the company plans to submit an NDA for the glucagon-like peptide-2 analogue. SBS-IF, rare and severe, affects an estimated 18,000 adult patients in the U.S., Europe and Japan.

Kenai secures $82M to move iPSC drug for Parkinson’s into phase I

San Diego-based Kenai Therapeutics 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, to treat Parkinson’s disease. The series A was co-led by Alaska Permanent Fund Corp., Cure Ventures and The Column Group, with participation from Euclidean Capital and Saisei Ventures.

Pivotal trial next for Pharmaust after MND/ALS phase I success

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). Preliminary efficacy data show a 58% reduction in the rate of disease progression in the cohort of patients who took the higher dose. With these results, the company will now progress to a pivotal phase II/III trial by midyear, Pharmaust CEO Michael Thurn told BioWorld.

Despite positive news, BioWorld Infectious Disease Index closes 2023 down 43%

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. In January 2024, BIDI saw a 1.41% decline, with seven stocks gaining ground while and 10 experienced declines. Throughout 2023, five stocks within BIDI concluded the year with positive returns, while 12 stocks remained in the negative.

China NMPA approves 40 innovative new drugs in 2023

China’s National Medical Products Administration (NMPA) cleared 40 novel innovative drugs in 2023, of which nearly half were cancer therapies, marking a significant increase from the 21 new class 1 drugs approved in 2022. According to the latest report posted on Feb. 4, China’s health regulator approved the therapies as class 1 products, referring to novel drugs without prior regulatory clearance in China or overseas markets.

US price negotiations point of contention at rare disease hearing

Has the U.S. Inflation Reduction Act (IRA) with its Medicare drug price negotiation provision become the new partisan sacred cow that cannot be tweaked? Debate over whether the orphan drug carveout included in that 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 today in a hearing held in observation of Rare Disease Day. Republicans on the subcommittee called the law’s current carveout that’s limited to drugs with only one orphan indication an ”unintended consequence” of a bill that was rushed through the legislative process without the usual hearings and deliberations. Their Democratic peers pushed back, saying Republican-sponsored bills aimed at expanding the carveout is just a political attempt to undermine the IRA.

BIO CEO 2024: AI applications will only become broader

The flood of artificial intelligence (AI) companies and their approaches can be dazzling to companies wondering if jumping into the market will be practical or just following a day’s trend. In a panel discussion with executives at the 2024 BIO CEO conference this week in New York, the consensus emerged that the new technology is here to stay, despite its occasional moments of hype, as its applications continually grow. The executives said companies need to determine how it will best work for their drug development and let the data bring value to the process. A big question to answer came from Abe Heifets, CEO of Atomwise: “Does AI do something you couldn’t otherwise do?”

Zinc finger approach mutes the epigenome to reduce cholesterol

An Italian group of researchers has used zinc finger editing to silence the PCSK9 gene and improve blood cholesterol levels in mice by applying a single dose of their modifier. The epigenetic-based method could be an alternative to genome editing.

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Achieve, Adma, Akeso, Applied, Arcutis, Arthex, Avidity, Beigene, Bioaegis, Biolinerx, Blossomhill, Cassava, Celldex, Derm-Biome, Dyadic, Enveric, Fore, Gain, Gilead, GSK, Hemogenyx, Hyloris, IMC, Immpact, Incannex, Incyte, Janux, Kadimastem, Kineta, Kiniksa, Lutris, Milestone, Neurenati, Nicox, Obseva, Oculis, Orbis, Palatin, Pharmaust, Redx, Sato, Shoreline, Skyhawk, Spero, Tonix, UCB, Viking