US Health Body Funds ALS Access Initiative

The US National Institutes of Health has awarded a multimillion-dollar grant to the Healey & AMG Center for ALS to fund an Expanded Access Program for Neurizon Therapeutics’ drug candidate NUZ-001. This initiative aims to provide up to 200 people living with amyotrophic lateral sclerosis (ALS) access to the investigational therapy for up to 96 weeks. The grant, part of a multiyear commitment, shows the NIH’s focus on accelerating access to potentially transformative treatments for debilitating diseases like ALS.

The program, also known as Compassionate Use, offers a pathway for individuals with serious or life-threatening diseases to access treatments not yet approved by the US FDA. It will run alongside the Phase 2/3 ALS Platform Trial, which is evaluating NUZ-001 in approximately 250 participants. Compassionate Use programs are critical for patients who cannot participate in traditional trials due to eligibility criteria, geographic barriers, or disease progression, ensuring they still have a chance to benefit from cutting-edge therapies.

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Expanding Access and Data Collection

The Expanded Access Program is designed to generate long-term real-world data from a broader ALS population than typically represented in clinical trials. It will collect safety and tolerability data, along with survival rates, ALS-specific functional measures, patient-reported outcomes, and exploratory biomarkers. Specifically, the program will track ALS Functional Rating Scale-Revised (ALSFRS-R) scores and slow vital capacity (SVC) measurements, which are key indicators of disease progression and respiratory function. This data collection will provide a more full understanding of NUZ-001’s impact across diverse patient subgroups, including those in advanced stages of ALS.

This data will complement the controlled efficacy and safety data from the Phase 2/3 Platform Trial. Neurizon expects the program to significantly expand the long-term safety database for NUZ-001 and provide additional insights to inform its development. By bridging the gap between clinical trial data and real-world outcomes, the program aims to address critical questions about NUZ-001’s effectiveness in a less controlled but more representative patient population.

The program will be conducted through approximately 20 physical ALS clinical trial sites and a national virtual study site, ensuring potential access across all 50 US states. This approach aims to make participation easier for individuals in rural and underserved areas. The virtual component leverages telemedicine and remote monitoring technologies to reduce the burden of travel and accommodate patients with mobility challenges, a common issue in ALS.

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Collaboration and Funding

The NIH funding covers all costs associated with the program, including drug supply, manufacturing, and regulatory and safety monitoring expenses. Neurizon will serve as the regulatory sponsor and supply NUZ-001, while the Healey & AMG Center will lead the clinical and operational execution. Neurizon’s partnership with Elanco Animal Health ensures a stable and scalable supply of active pharmaceutical ingredients, critical for meeting the demands of both the EAP and the ongoing Phase 2/3 trial.

In a statement, Sergio Duchini, interim executive chairman of Neurizon, expressed pride in supporting the Healey & AMG Center in this initiative. He emphasized that the program will broaden the understanding of NUZ-001 and provide valuable complementary data. Duchini also highlighted the collaborative effort between industry, academia, and government, which he described as essential for advancing treatments for complex diseases like ALS.

For those directly impacted by ALS, this development means more than just data collection. It represents a chance for individuals who might not qualify for traditional trials to access a potentially life-altering treatment, offering a glimmer of hope in a condition often marked by limited options. Patients and caregivers have welcomed the initiative as a step toward greater inclusivity in clinical research, particularly for a disease with no cure and few approved therapies.

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Looking Ahead

The Expanded Access Program is expected to begin enrolling participants by Q1 CY27, with topline results from the Phase 2/3 trial anticipated in late Q2 CY27. The continuation of the program beyond these results depends on a positive trial outcome. Start-up activities, including site preparation and protocol finalization, are already underway to ensure a smooth launch.

Dr. Sabrina Paganoni from the Healey & AMG Center highlighted the importance of such programs for ALS patients who are ineligible for clinical trials. She expressed gratitude to the NIH for supporting this effort and advancing ALS research. Dr. Paganoni also noted that the EAP aligns with the center’s mission to accelerate the development of effective treatments while ensuring equitable access for all patients, regardless of their trial eligibility.