Investors didn’t respond well to the U.S. FDA’s briefing document for the March 30 advisory committee meeting on Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis (ALS) candidate. Shortly after the materials for the adcom were posted Monday, Amylyx (NASDAQ:AMLX) dropped from a morning high of $25.68 per share to an all-time low of $10.49 in the heaviest trading since the company went public in January. With share volume exceeding 15 million, Amylyx rebounded somewhat, ending the day at $16.01, down nearly 36% from its March 25 close of $25.
LONDON – Aviadobio Ltd. has raised $80 million in a series A round to take a precision microdosed gene therapy for treating familial frontotemporal dementia into a phase I/II clinical trial.
Ilex Medical Ltd. has injected $3.2 million into cell therapy company Kadimastem Ltd., as it prepares to list on the Nasdaq. As part of the investment, Ilex Medical was granted a total of 1,407,130 options (60% coverage) in two series, at exercise prices of $2.2 and $2.7 per share (up to 100% from the share price at the transaction). If exercised, it would increase the total investment to $6.7 million.
Though Wall Street may not have caught on to the value of Clene Inc.’s phase II results with gold nanocrystal suspension CNM-Au8 in amyotrophic lateral sclerosis (ALS), CEO Rob Etherington said his firm is “truly excited” about the data, which bode well for the next stage of development, already underway.
Biogen Inc.’s phase III trial of tofersen in a form amyotrophic lateral sclerosis (ALS) has missed its main goal, but the company said it is still talking with regulators after seeing positive trends in the data.
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).
PERTH, Australia – Bioinformatic and cell technology company Genieus Genomics Ltd. is using the power of the human genome to build a platform for neurodegenerative diseases to develop diagnostic tests for personalized medicines.
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.
