Clene Launches Second Expanded Access Program for ALS Therapy CNM-Au8

Robert Glanzman, MD, FAAN

Clene Nanomedicine has announced the launch of its second FDA expanded access program (CNMAu8.EAP02) with its lead drug candidate, CNM-Au8, an aqueous suspension of catalytically active, clean-surfaced, faceted gold nanocrystals, in patients with amyotrophic lateral sclerosis (ALS).¹

The expanded access program will be in conjunction with the Healey ALS Platform Trial, the first platform trial for the treatment of ALS. The breakthrough trial features CNM-Au8 and several other drug candidates and aims to help develop these therapies by reducing trial time, costs, and increasing patient participation. Additionally, the new program will provide an opportunity to those with ALS to access CNM-Au8 even if they are not eligible to enroll in the trial.

Robert Glanzman, MD, FAAN, chief medical officer, Clene, said in a statement, "We are honored to be collaborating with the Healey Center for ALS in this critically important effort of providing access to CNM-Au8 to people with ALS who may benefit. The Healey Center is an outstanding partner, and we proudly share its goal of developing lifesaving therapies for the treatment of ALS."

Three participating clinical trials sites in the Healey ALS Platform Trial will be available to patients in the expanded access program and are supported by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH). To date, Holy Cross Hospital in Fort Lauderdale, Florida, and the Hospital for Special Care in New Britain, Connecticut, are the first 2 sites to be included.

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The first expanded access program (CNMAu8.EAP01), also launched in partnership with the Healey & AMG Center, presently supports access to the investigational agent for 40 people living with ALS. Enrollment for this program began in September 2019 with long-term participants now treated for over 100 weeks.

"While CNM-Au8 advances through the registration platform trial with an aim to become available to all patients with ALS, expanded access programs enable more people with ALS to benefit today from the potential of our first-in-class neuroreparative energetic nanotherapeutic. Clene is committed to supporting communities living with devastating neurodegenerative diseases, and we are doing our utmost each day to reach and treat more patients," Rob Etherington, president and chief executive officer, Clene, said in a statement. 

CNM-Au8 demonstrated an ability to improve motor neuron function in patients with ALS in its phase 2, multicenter, double-blind, parallel-group, placebo-controlled trial, dubbed RESCUE-ALS (NCT04098406). Interim results presented at the Virtual 31st International Symposium on ALS/MND, December 9-11, 2020, showed that over 40% of the 43 participants experienced improvements as assessed by the motor neuron number index-4 (MUNIX[4]) score. Not only did the average MUNIX(4) score increase in both the CNM-Au8 and placebo groups, but the increase exceeded statistic modeling that predicted a linear decline in average MUNIX(4) score from study onset.²

The drug is currently in development for other neurological diseases including multiple sclerosis (MS) and Parkinson disease (PD). Clene announced topline results from the phase 2 REPAIR trials in August, which demonstrated that treatment with CNM-Au8 significantly improved brain energetic metabolism in patients with MS and PD. All told, the results for the primary end point, the mean change in the brain ratio of oxidized nicotinamide adenine dinucleotide (NAD+) to reduced nicotinamide adenine dinucleotide (NADH), demonstrated a statistically significant increase by an average of 0.589 units (10.4%) following the 12-week treatment period (paired t-test, P = .037).³

REFERENCE

1. Clene announces initiation of a second FDA expanded access program with CNM-Au8 for people living with amyotrophic lateral sclerosis. News release. Clene Nanomedicine. September 23, 2021. Accessed September 23, 2021. https://www.globenewswire.com/news-release/2021/09/23/2302037/0/en/Clene-Announces-Initiation-of-a-Second-FDA-Expanded-Access-Program-with-CNM-Au8-for-People-Living-with-Amyotrophic-Lateral-Sclerosis.html

2. Clene Nanomedicine presents blinded interim results from RESCUE-ALS phase 2 study at the 31st international symposium on ALS/MND. News release. Clene Nanomedicine. December 10, 2020. Accessed September 23, 2021. https://www.globenewswire.com/news-release/2020/12/10/2143338/0/en/Clene-Nanomedicine-Presents-Blinded-Interim-Results-from-RESCUE-ALS-Phase-2-Study-at-the-31st-International-Symposium-on-ALS-MND.html

3. Clene reports positive top-line results from its phase 2 REPAIR clinical trials in Parkinson disease and multiple sclerosis. News release. August 5, 2021. Accessed August 5, 2021. https://www.globenewswire.com/news-release/2021/08/05/2275392/0/en/Clene-Reports-Positive-Top-line-Results-from-its-Phase-2-REPAIR-Clinical-Trials-in-Parkinson-s-Disease-and-Multiple-Sclerosis.html