Clene (NASDAQ:CLNN) executives told investors the clinical-stage biopharmaceutical company has secured an in-person meeting with the U.S. Food and Drug Administration (FDA) later this quarter as it continues to pursue an accelerated approval pathway for its lead program in amyotrophic lateral sclerosis (ALS).
FDA meeting granted after multiple recent interactions
CEO and President Rob Etherington said the company was granted a Type C meeting with the FDA “this quarter,” describing it as the fifth meeting with the agency in roughly 14 months following prior discussions in November, June, August, and September. Etherington said the purpose of the ongoing dialogue is to review Clene’s biomarker and survival datasets related to CNM-Au8, including analyses the company said were recommended by the FDA.
Biomarkers highlighted: neurofilament, GFAP, and others
Etherington pointed to reductions in neurofilament and glial fibrillary acidic protein (GFAP) as key findings discussed with regulators. He described neurofilament as associated with neuronal injury and GFAP as associated with astrocytes, and said both biomarkers can be detected in blood and cerebrospinal fluid, with Clene measuring them in blood. According to Etherington, the company has shown these biomarker improvements were “strongly associated with longer survival.”
He also referenced insulin-like growth factor binding protein as another biomarker Clene recently discussed, saying it moved “in lockstep” with the broader biomarker-survival thesis. Etherington said Clene observed a concordant response among individuals on its drug and described a “direct connection” between declines in neurofilament and GFAP.
Accelerated approval strategy centers on surrogate endpoints and confirmatory study
Etherington framed the company’s regulatory approach as a “three-legged stool,” describing three elements Clene is presenting to FDA reviewers:
- Evidence that biomarker benefits are “substantial and significant” and concordant, including neurofilament, GFAP, and insulin-like growth factor binding protein.
- Evidence that changes in neurofilament are independently associated with mortality.
- Evidence that a clinical survival benefit is consistent across datasets, supported by multiple statistical methods.
Etherington cited the FDA’s accelerated approval pathway—originally developed during the AIDS era—as an option when a trial misses its primary endpoint, using a surrogate endpoint such as a biomarker. He also pointed to Biogen’s Qalsody (tofersen) as an example of ALS approval based on neurofilament light outcomes, though he noted it applies to a small subset of patients.
Clene’s proposed path includes an NDA submission for accelerated approval and a confirmatory Phase III trial designed around survival, according to Etherington. He said the company has sent the FDA a 105-page dossier, submitted in December, and that the in-person meeting will take place at the FDA’s White Oak facility in Maryland, with Clene bringing key opinion leaders and biomarker experts.
Timing: meeting this quarter; potential NDA filing in Q2
On timing, Etherington said the meeting will be completed by the end of the current quarter. If the FDA concurs that the company’s biomarker and survival evidence meets expectations—including confirmation across multiple datasets and linkage between biomarker reduction and survival—Clene intends to file a New Drug Application seeking accelerated approval “in the course of the second quarter.”
He added that Clene expects to learn whether the FDA accepts the application in the summer, and if accepted, the company could be on a commercialization path “for the top of next year.”
Financing extends runway; tranche structure tied to milestones
CFO Morgan Brown said Clene recently completed a tranche financing that raised roughly $6 million at $6.50 per share, which he said extended the company’s runway by an additional quarter. Brown said the structure was designed to align funding with upcoming milestones, including NDA acceptance and potential approval.
Brown described a second tranche that could raise $7 million at $7 per share, and a final tranche triggered upon approval with a $12.50 strike price that would raise about $15 million. He said the financing involved three dedicated healthcare investors: Vivo, Castellan, and Boxer Capital.
In closing remarks, Etherington reiterated the company’s focus on ALS, citing an estimated 30,000-plus ALS patients in the U.S. and describing typical mortality without treatment as two to four years. He also referenced efforts to reauthorize congressional funding for ALS research and said the company is “very grateful” the FDA is willing to consider its potential application.
About Clene (NASDAQ:CLNN)
Clene (NASDAQ: CLNN), also known as Clene Nanomedicine, is a clinical-stage biopharmaceutical company developing proprietary nanoparticle therapies aimed at treating neurodegenerative and demyelinating disorders. The company’s flagship product, CNM-Au8, is a suspension of catalytic gold nanocrystals designed to enhance cellular energy metabolism, promote remyelination, and reduce oxidative stress. Clene’s platform leverages the unique physicochemical properties of its nanoparticles to support neuronal health, with a focus on diseases that currently lack effective disease-modifying treatments.
Clene’s lead candidate, CNM-Au8, is undergoing multiple clinical trials targeting conditions such as amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple sclerosis (MS).
