The Cardiovascular Research Technologies 2024 conference in Washington this week demonstrated continued positive outcomes for patients who underwent transcatheter aortic valve replacement with devices made by Abbott Laboratories, Edwards Lifesciences Corp. or Medtronic plc.
After a disappointment with the same product in another indication about two years ago, Sensorion SA met the primary endpoint in its proof-of-concept phase IIa trial with SENS-401 for residual hearing preservation after cochlear implant.
Tweaks made to the design of the phase III trial called Phoenix (vs. the narrowly positive phase II Centaur study) with Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis (ALS) drug Relyvrio (sodium phenylbutyrate plus taurursodiol) didn’t work. Now, the Cambridge, Mass.-based firm is facing possible withdrawal of the treatment from the U.S. and Canada, where it’s known as Albrioza. Shares of Amylyx (NASDAQ: AMLX) closed March 8 at $3.36, down $15.61, or 82.3%, after the firm disclosed top-line results from Phoenix, a global, 48-week, randomized, placebo-controlled phase III effort with Relyvrio, also known as AMX-0035.
After spending 20 years at Novartis, Radiopharm Theranostics Ltd. CEO Riccardo Canevari told BioWorld that when he joined Radiopharm he wanted to focus on something different within radiopharmaceuticals where no one was playing. “I believe these new modalities are at the beginning of their potential, much like in the immuno-oncology space years ago. That’s a nice place to be,” he said, but it’s not only about competition, it’s also about understanding what other companies are doing and if there is a disease area or a mechanism of action that is not being explored, he said.
The phase II Initium study of Ultimovacs ASA’s therapeutic cancer vaccine in treating unresectable or metastatic malignant melanoma did not meet the primary endpoint. The company framed the loss by saying the UV-1 vaccine still has other indications where it could succeed.
Phase I data of oral amycretin, a co-agonist of GLP-1 and amylin receptors from Novo Nordisk A/S, showed a 13.1% reduction in body weight at 12 weeks vs. 1.1% for placebo, pushing shares of the Bagsvaerd, Denmark-based company to their highest levels to date.
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.
Exonate Ltd. is preparing to advance its eye drop candidate to treat retinal vascular diseases, EXN-407, into a phase IIb study following promising tolerability and early signs of efficacy in a phase Ib/IIa study. A small-molecule SRPK1 inhibitor, EXN-407 is designed to be administered twice daily and works by selectively targeting specific (proangiogenic) isoforms of VEGF that lead to vascular retinal disease progression via aberrant growth of leaky blood vessels within the eye.
Apogee Therapeutics Inc.’s phase I home run put IL-13-targeting antibody APG-777 on an accelerated development path in atopic dermatitis, and the company touted its similarity to further-along IL-13 competitor lebrikizumab, from Eli Lilly and Co., as a likely indicator of further success.
The RNAi therapeutic zilebesiran, which was the focus of a 2023 $2.8 billion deal between Alnylam Pharmaceuticals Inc. and Roche Holding AG, demonstrated significant systolic blood pressure reductions in patients with mild to moderate hypertension who were part of the Kardia-2 phase II study.
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