Benevolentai Cambridge Ltd. has synthesized retinoid acid receptor α (RARα) and β (RARβ) agonists reported to be useful for the treatment of cancer and amyotrophic lateral sclerosis (ALS).
Less than a month after disclosing that its confirmatory phase III trial of Relyvrio (sodium phenylbutyrate plus taurursodiol) fell short of its endpoint, Amylyx Pharmaceuticals Inc. is withdrawing the amyotrophic lateral sclerosis (ALS) drug from the market.
Asha Therapeutics LLC has nominated a development candidate, ASHA-624, as a potential disease-modifying therapy for amyotrophic lateral sclerosis (ALS) with additional indications in chemotherapy-induced peripheral neuropathy, glaucoma, and traumatic brain and spinal cord injuries. ASHA-624 is expected to enter the clinic by year-end.
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease for which there is a 10% rate of familial cases, with the rest being sporadic cases. Both genetic and environmental factors contribute to the etiology of ALS, and more than 120 genes have been reported to be tied to the disease, but few with strong association. Thus, identifying additional genes contributing to ALS will help shed light on the disease and its related therapies.
Top-line data from Seelos Therapeutics Inc.’s phase II/III study of SLS-005 in amyotrophic lateral sclerosis (ALS) failed to meet statistical significance in its primary and secondary endpoints, continuing the stock’s nearly half-year downward trajectory.
Patients with amyotrophic lateral sclerosis (ALS) have a median survival of 2 to 5 years. There are 3 FDA-approved drugs for ALS (riluzole, edaravone and Relyvrio [phenylbuturate/taurursodiol]), but they only lead to modest benefit. There are several pathways involved in the disease, but all of them lead to neuroinflammation.
In cell and animal models of amyotrophic lateral sclerosis (ALS), the expression of toxic dipeptides in neurons led to changes in the extracellular matrix (ECM) as a protective response. The authors wrote that their findings, which appeared in Nature Neuroscience on Feb. 29, 2024, could suggest new strategies for how to approach ALS.
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.
Amyotrophic lateral sclerosis (ALS) is a fatal disease characterized by the death of motor neurons in conjunction with neuroinflammation and deposition of protein aggregates, such as TDP-43, in these neurons and oligodendrocytes. Progranulin is a growth factor that is essential for neuron survival and a regulator of anti-inflammatory responses.
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.
