Increasing Access to Treatments: Synapticure’s Role in the Clene EAP

Expanding Access to Treatments: What to Know About the Clene EAP

At Synapticure, we are committed to advancing research and ensuring individuals have access to promising treatments. One of the ways we’re helping to drive progress is through our participation in Expanded Access Programs (EAPs). Synapticure is an enrolling study site for Clene’s ACT-EAP, a groundbreaking initiative funded by the National Institutes of Health (NIH). This program not only provides access to CNM-Au8, an ALS treatment entering Phase III clinical trials, but also generates high-quality data that will contribute to future drug development. Below, we answer key questions about this study and Synapticure’s role.

Understanding the Expanded Access Program (EAP)

What is the purpose of this study?
The primary goal of this EAP is to offer individuals with ALS who are ineligible to enroll in ALS-related clinical trials access to CNM-Au8 beforePhase III trials while collecting critical safety and efficacy data. This data, along with biological samples, will support broader research initiatives and further our understanding of ALS. By participating, individuals contribute to a growing repository of information that researchers can use to accelerate the development of future treatments and potential cures.

Why was this study funded by the NIH?
The grant funding for this EAP is part of the implementation of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS). The EAPS funded under the ACT for ALS were designed to provide early access to promising therapies while ensuring rigorous data collection, similar to a clinical trial but without a placebo group. The study follows strict clinical trial standards, ensuring the data and samples collected will be valuable for future research.

How does this EAP differ from others?
Unlike traditional EAPs, NIH-funded EAPs adhere to a clinical trial-like process with rigorous data collection, oversight, and reporting requirements. These include detailed study protocols, external monitoring, and extensive safety measures. This results in both expanded treatment access and high-quality insights that advance both our understanding of ALS and the drug being investigated.

Participant Selection & Study Enrollment

How were participants selected at Synapticure?
The number of patients who joined Synapticure’s study waitlist exceeded the number of available enrollment spots allocated to the site even before study enrollment opened, and has continued to grow. Because Synapticure’s participation in the study is intended to increase patient access, screening priority was based on an algorithm that weighs a number of factors centered around access to ALS research clinics and clinical care, with the goal of ensuring that patients with the least access to these resources were given a chance to screen for the study and enroll remotely.  The first 70% of participants were selected using this weighted algorithm, while the remaining participants were chosen through random lottery selection, ensuring equal opportunity for participation.

What other sites are part of this study? ?
For information on enrolling sites, visit Clene’s study website. Please note that enrollment is currently full, and future openings are not guaranteed.

Study Logistics & Expectations

How long will the study last?
Each participant will be in the study for approximately 144 weeks (~2.5 years), with the full study expected to run over four years.

Is there a placebo group?
No, all participants in this EAP receive CNM-Au8. Unlike traditional clinical trials, EAPs are designed to provide treatment access without a placebo comparison group.

What costs are associated with participation?
All research-related costs, including study medication, research visits, and assessments, are covered under the NIH grant. However, participants are required to have clinical oversight from a neurologist at their enrolled site, which is billed to insurance.

What Comes Next?

Clene is preparing for a Phase III clinical trial of CNM-Au8, expected to begin enrollment later this year (subject to change). Data from both this EAP and the upcoming Phase III trial will be submitted to the FDA under the accelerated approval pathway, with an NDA submission planned for mid-2025.

Why This Matters

Through our work with this EAP, Synapticure is expanding access to groundbreaking treatments and ensuring that individuals from all backgrounds—including those in underserved areas—can contribute to research. To date, we have enrolled 90 patients from 35 states. 21% of these study participants live in socioeconomically disadvantaged areas, and on average, they reside over 100 miles from the nearest NEALS-affiliated clinical site. Without a virtual model like Synapticure’s, many of these individuals would not have had the opportunity to participate in research.

Additionally, while our enrollment rate started conservatively in the first few months to ensure the highest standards of data collection, we have since accelerated significantly—demonstrating both the efficiency and efficacy of our approach.

We are proud to be setting new standards for patient access to ALS research via this partnership with Clene and Columbia University, and we remain committed to bringing individuals closer to cutting-edge treatments through innovative, patient-centered care.