The U.S. Food and Drug Administration has agreed to review Clene Inc.’s New Drug Application for CNM‑Au8, the company’s oral nanocrystal therapy for amyotrophic lateral sclerosis (ALS). The agency’s Type C meeting minutes, released by Clene Nanomedicine Inc., indicate that the FDA believes the biomarker data—particularly reductions in neurofilament light (NfL)—may support an accelerated approval pathway under Subpart H of 21 CFR 314.510.
The FDA will evaluate a data package that includes results from the Phase 2 HEALEY ALS Platform Trial, the RESCUE‑ALS extension data, and the NIH‑sponsored expanded access program. The agency has requested additional information linking the magnitude of NfL reduction to clinical benefit, underscoring the importance of the biomarker in the approval discussion.
Clene plans to file the NDA in the third quarter of 2026, with a Phase 3 confirmatory study slated to begin in the first quarter of 2027. The accelerated approval pathway is the only viable route to market without a costly Phase 3 trial, given the company’s limited cash runway.
The company’s $28 million financing plan, announced in March 2026, was oversubscribed and is expected to unlock the remaining tranches of its capital raise. The infusion of capital is critical to fund the confirmatory study and sustain operations until a potential approval.
Clene’s financial performance has been characterized by net losses, with a Q1 2025 loss of $0.8 million versus $11.1 million in Q1 2024, and a full‑year 2025 loss of $26.2 million compared to $39.4 million in 2024. Cash and cash equivalents stood at $9.8 million as of March 31 2025, highlighting the urgency of the financing plan to extend the company’s runway.
Rob Etherington, President and CEO of Clene, said, "We are encouraged by the FDA's careful evaluation of the benefits and risks associated with Clene's ALS drug candidate, CNM‑Au8, including the biomarker data the Company provided. The filing of an NDA submission represents an important milestone for CNM‑Au8 and for the ALS community. We are committed to working with the Agency on this filing and are conducting the Phase 3 confirmatory study for CNM‑Au8, which we intend to commence in the first quarter of 2027." Sandra Abrevaya, I AM ALS Co‑Founder and Board Member, added, "People living with ALS cannot afford to wait. Flexible, science‑driven regulatory approaches such as this can play a critical role in accelerating access to new therapies for a fast‑progressing, fatal disease. We're grateful for..."
The market reacted positively to the FDA’s willingness to consider NfL as a reasonably likely surrogate endpoint, reflecting investor confidence in the accelerated approval pathway and the potential for a first‑in‑class ALS therapy.
CNM‑Au8 has previously received orphan drug designation, providing incentives for development. The FDA’s decision marks a pivotal regulatory milestone that could transform Clene’s financial trajectory and competitive standing in the neurodegenerative disease space, provided the company can secure the necessary confirmatory data and manage its cash burn.
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