LONDON – A significant body of research indicates inhibition of the mitochondrial permeability transition pore (PTP) would reduce neuroinflammation and protect neurons, but the difficulty of finding inhibitors that cross the blood-brain barrier to regulate the pore has left the therapeutic potential largely untapped.
A month after Astrazeneca plc’s $39 billion takeover of Alexion Pharmaceuticals Inc. was finalized, the merged company has hit its first speed bump after the new unit discontinued a phase III rare disease trial. Alexion, of Boston, said it axed the 382-patient CHAMPION-ALS trial of its long-acting C5 complement inhibitor Ultomiris (ravulizumab) in amyotrophic lateral sclerosis (ALS).
As amyotrophic lateral sclerosis continues to make headlines, candidates bearing varied approaches proliferate and the indication likely allows for multiple players, given the prospect of a combo regimen.
Amylyx Pharmaceuticals Inc.’s oversubscribed, $135 million series C financing led by Viking Global Investors will help lay the groundwork for commercializing in Canada and Europe, as well as for a second phase III study in the U.S. with lead candidate AMX-0035 for amyotrophic lateral sclerosis.
In search of new therapies for amyotrophic lateral sclerosis (ALS), Eli Lilly and Co. has agreed to pay Verge Genomics $25 million in up-front, equity and potential near-term payments, plus as much as $694 million in milestones, to discover and validate up to four new targets for treating the disease over the course of a three-year collaboration. Verge retained rights to its internal lead ALS program, poised to enter the clinic next year.
Trading in shares in AB Science SA was suspended Tuesday after a safety signal prompted a voluntary hold on two late-stage clinical studies of its masitinib, in mastocytosis and amyotrophic lateral sclerosis, respectively.
Despite two recent clinical trial failures, Orphazyme A/S’s arimoclomol is still on track for its June 17 PDUFA date in treating Niemann-Pick disease type C. The newest stumble is in the pivotal study of arimoclomol for treating amyotrophic lateral sclerosis (ALS) as it failed to hit its primary and secondary endpoints.
Brainstorm Cell Therapeutics Inc. said FDA senior leadership told the company the level of clinical data in the Nurown (neurotrophic factor-producing mesenchymal stem cells) phase III trial in amyotrophic lateral sclerosis (ALS) does not provide the threshold of substantial evidence needed to support a BLA.
Shares of Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) fell 66.3% to $4.02 on Nov. 17 following news that its autologous cell therapy candidate, Nurown, missed the primary efficacy endpoint of a phase III amyotrophic lateral sclerosis (ALS) study.
Mutations in the annexin A11 gene contribute to motor neuron degeneration in amyotrophic lateral sclerosis by disrupting cellular calcium ion homeostasis and stress granule protein disassembly contributing to ALS neurodegeneration.
