Clene disclosed Thursday the FDA has determined that biomarker Neurofilament Light Chain reduction in its Phase II programs “were insufficient to support accelerated approval at this time.”
Pictured: Rejected stamp put on a document/iStock, Pavel Muravev
Clene revealed Thursday that the FDA has decided not to grant accelerated approval for the company’s amyotrophic lateral sclerosis drug candidate.
The FDA in a recent meeting with Clene determined that the biomarker Neurofilament Light Chain (NfL) reduction findings in Phase II programs “were insufficient to support accelerated approval at this time,” according to the company’s announcement. Clene’s stock tumbled 33% in premarket trading Thursday in response to the news.
Despite the FDA rejection, Clene said it intends to provide “supplemental data for further engagement” with the regulator in the first half of 2024, providing additional long-term clinical evidence and biomarker results of the treatment benefit for amyotrophic lateral sclerosis (ALS) patients.
Clene is hopeful regarding the clinical development of CNM-Au8, an oral suspension of catalytically active gold nanocrystals. Phase II clinical and biomarker results showed the treatment reduced NfL in ALS patients, with evidence of long-term survival data. Safety data for CNM-Au8 has identified no safety signals for the drug candidate, according to the company.
In September 2023, Clene posted promising long-term survival data showing CNM-Au8 reduced the risk of death in ALS patients by 49% with no serious adverse events.
“We are also encouraged that the recently disclosed long-term NfL biomarker decreases are consistent with delayed clinical time-to-event outcomes,” Benjamin Greenberg, Clene’s head of medicine, said in a statement.
In April 2023, the FDA approved Biogen and Ionis’ Qalsody (tofersen) under its accelerated approval pathway for ALS patients with a mutation in the SOD1 gene. The approval was based on the drug’s ability to lower blood levels of NfL, which drug developers hoped would help pave the way for their drug candidates that do the same.
Clene isn’t alone in dealing with an FDA setback in the ALS space. In September 2023, an FDA advisory committee voted 17-1 against BrainStorm Cell Therapeutics experimental treatment ALS NurOwn. The agency took issue with the manufacturing plan for the cell therapy and said the company failed to demonstrate substantial efficacy evidence in its BLA.
As it continues to seek regulatory approval for CNM-Au8, Clene is supporting two compassionate use programs and adding a third, thanks to a $45 million grant from the National Institutes of Health. The company will also launch a Phase III ALS confirmatory study in 2024. CNM-Au8 is also in late-stage trials for multiple sclerosis and Parkinson’s disease.
Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.
