Top-line from Orphazyme A/S’ phase II/III trial of arimoclomol for treating inclusion body myositis, a muscle-wasting disease, failed to hit its primary and secondary endpoints. The data caused investors to pull back sharply as shares of Copenhagen-based Orphazyme (NASDAQ:ORPH) had dropped 28.97% on March 29 to close at $8.80 per share.
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.
French researchers have shown that modifying peripheral macrophages could quell microglial activation in the central nervous system, slowing the rate of decline and extending survival in mouse models of amyotrophic lateral sclerosis.
Scribe Therapeutics Inc., another CRISPR-based genome editing firm out of the Jennifer Doudna stable, launched with $20 million in series A funding and a deal with Biogen Inc. in amyotrophic lateral sclerosis (ALS), which brings in another $15 million up front and up to $400 million in development and commercial milestones.
In the past 10 years, the advances in understanding the etiology of neurodegenerative diseases have been dramatic. “The development of novel biomarkers and other tools as well are key in aiding diagnostic potential and the ability to track disease progression have been phenomenal,” Isaac Veinbergs, CEO of newly created Libra Therapeutics Inc., told BioWorld.
Though Quralis Corp., of Cambridge, Mass., has been developing its pipeline for more than three years, the company now has a $42 million series A in hand to continue researching and developing therapies for amyotrophic lateral sclerosis and frontotemporal dementia.
What’s new inevitably includes an element of the old. Clene Nanomedicine Inc., which just completed enrollment and dosed the first patient in its phase II trial in amyotrophic lateral sclerosis (ALS), literally contains an element of the old in its lead nanocatalytic therapy: gold.