Clene Inc. (Nasdaq: CLNN) (along with its subsidiaries, “Clene”) and its wholly owned subsidiary Clene Nanomedicine Inc., a late clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), today announced that it has entered into an agreement with German-based APST Research GmbH (APST) to utilize its extensive NfL database to support the FDA-recommended analyses of CNM-Au8®’s effect on NfL decline in participants in ongoing Expanded Access Protocols (EAPs).
APST maintains one of the largest comprehensive ALS repositories of people living with ALS, including demographic data, clinical data, ALS motor phenotypes and biomarker data, specifically, serum neurofilament light chain (sNfL). The database in the repository comprises extensive sNfL data from over 4,300 ALS patients as well as self-reported ALSFRS-R. The APST platform offers digitized data on thousands of individuals living with ALS to comprehensively view the disease’s progression by gathering information on ALS phenotypes, biomarker data, and patient-reported outcomes. This effort utilized cutting-edge data collection and analytics to provide a comprehensive long-term natural history of NfL change. Comparisons to the APST NfL dataset will be a crucial element of the FDA-recommended statistical analysis plan for NfL evaluations of NIH-sponsored EAP participants.
The NfL dataset being analyzed by Clene includes data from more than 1625 ALS patients, aligning the biomarker data of NfL to clinical ALSFRS-R assessments, slow vital capacity (SVC), and clinical events such as ventilation support and nutrition intervention. The objective of the planned analyses is to compare NfL change observed in the NIH-sponsored EAP participants to matched controls using the APST dataset, and to demonstrate that the rate of NfL change is associated with survival in people living with ALS.
Clene has supported the treatment of nearly 500 people living with ALS through its three (3) ALS EAP programs to collect Real-World Evidence (RWE) of the effects of CNM-Au8 in ALS. RWE evidence collected from EAP participants may be supportive of the observed survival improvement and NfL decline observed in the HEALEY ALS Platform Trial with CNM-Au8 30 mg treatment. Clene plans to submit the statistical analysis plan for the NIH-sponsored EAP NfL analyses to the FDA shortly. Analyses of NIH-sponsored EAP NfL reduction, if positive, will support the planned NDA submission for potential Accelerated Approval of CNM-Au8 in ALS, planned in the second half of 2025.
Across over 800 participant years of treatment with CNM-Au8, no significant safety concerns or safety trends have been identified. No serious adverse events (SAEs) have been identified as related to CNM-Au8 treatment by any investigator to date.
“We are excited to enter into this endeavor with one of the world’s largest ALS NfL datasets in order to supplement available biomarker NfL data from our NIH-sponsored EAP, and to support the existing clinical study data for the potential review of an application for approval of CNM-Au8 in ALS via an accelerated regulatory pathway,” said Clene’s CEO, Rob Etherington. “We remain dedicated to the ALS community and are honored to continue our efforts to help critically ill patients and their families.”
“Our extensive and robust data collection empowers pharmaceutical companies to advance their clinical research and trials, driving us toward a deeper understanding of ALS disease progression” said APST’s founder and CEO, Thomas Meyer, a renowned ALS key opinion leader.
