Kringle Pharma Inc.’s phase II trial evaluating its recombinant human hepatocyte growth factor ligand, oremepermin alfa, failed to meet both primary and secondary endpoints in a study of its potential to help people with amyotrophic lateral sclerosis (ALS).
Amylyx Pharmaceuticals Inc. got good news when the Institute for Clinical and Economic Review posted a revised evidence report Aug. 4 that assessed the comparative clinical effectiveness and value of the company’s AMX-0035 and Mitsubishi Tanabe Pharma America Inc.’s Radicava (edaravone) in treating amyotrophic lateral sclerosis.
No one denies that fewer new drugs will be a consequence of the Medicare drug pricing provisions the Democrats are trying to push through the U.S. Congress ahead of the midterm election campaign season. Less attention has been paid to the negative impact on drug-device combinations and new diagnostics that accompany innovative treatments.
Unless the U.S. FDA once again overrides its Peripheral and Central Nervous System Drugs Advisory Committee, it looks like the ALS community will have a longer wait for an additional tool against the fatal, degenerative disease. After hearing from both Amylyx Pharmaceuticals Inc. and FDA reviewers, along with 26 people testifying during the open public hearing, the committee voted March 30 against approval of AMX-0035, a fixed-dose combination of sodium phenylbutyrate and taurursodiol, as a much-needed treatment for amyotrophic lateral sclerosis (ALS).
Following another failure in amyotrophic lateral sclerosis (ALS), Biogen Inc. will discontinue its development of antisense oligonucleotide BIIB-078 with partner Ionis Pharmaceuticals Inc. The stumble is part of a mega-collaboration the two companies began 10 years ago that has also yielded a lot of success, including the blockbuster Spinraza (nusinersen).
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.
