1st ALS patient enrolled in MN-166 expanded access program
Trial enrolling 200 patients ineligible for ongoing Phase 2b/3 study
The first patient has been enrolled in an expanded access program (EAP) trial evaluating MN-166 (ibudilast), Medicinova’s oral anti-inflammatory drug, in people with amyotrophic lateral sclerosis (ALS).
EAPs, also known as compassionate use programs, allow patients with serious or life-threatening conditions to access investigational therapies outside of clinical trials when no other alternatives are available.
In addition to providing a greater number of patients with potentially lifesaving or life-extending treatment, EAPs offer opportunities to collect additional clinical data on experimental therapies.
The MN-166 program will enroll about 200 patients at several locations in the U.S. who are not eligible for the ongoing COMBAT-ALS trial (NCT04057898), including those with more advanced disease, to evaluate the therapy’s safety and efficacy in a broader patient population.
It is funded by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), under the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS). ACT for ALS was signed into law in 2021 and provides $100 million annually through fiscal 2026 for efforts to improve ALS prevention, diagnosis, and treatment.
Targeting proteins to reduce inflammation
“We are honored to support this EAP trial, which will provide MN-166 to more individuals in advanced stages of ALS who are not eligible to our current Phase 2/3 COMBAT-ALS trial,” Yuichi Iwaki, MD, PhD, president and CEO at Medicinova, said in a company press release.
In ALS, the nerve cells responsible for controlling voluntary muscle movements, called motor neurons, become progressively damaged and die. The exact causes of ALS remain unclear, but abnormal brain inflammation is thought to play a central role.
MN-166 is an oral small molecule designed to reduce that inflammation and boost the survival and growth of nerve cells by targeting several different proteins involved in those processes. It is expected to slow the progression of ALS and potentially extend survival.
The ongoing Phase 2b/3 COMBAT-ALS trial is evaluating MN-166 in about 230 adults who began experiencing ALS symptoms up to 1.5 years before entering the study.
Participants are being given either MN-166 or a placebo twice daily for 12 months, after which all will be able to enter an optional, open-label extension and receive the experimental therapy for an additional six months.
An interim analysis from the first 217 patients, presented last year, suggested that MN-166 may slow ALS progression, extend survival, and reduce the decline in key physical functions. These include bulbar functions like speech and swallowing, fine motor skills such as writing, and gross motor abilities like walking or climbing stairs.
The data remain blinded, meaning investigators don’t know which patients are receiving NM-166 and which are on a placebo, so more data will be needed to know for sure if the experimental therapy has an impact on those outcomes.
MN-166 has received orphan drug designation in the U.S. and European Union to treat ALS. The U.S. Food and Drug Administration (FDA) has also granted it fast track designation. These designations are intended to support and expedite the development of promising treatments by offering incentives such as regulatory support, market exclusivity, and potential expedited review.
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