Darwin Biotech Presented Randomized Phase 1 ALS Study Data of Cell-Free ALT001 and U.S. Phase 1/2 Study Design at 2026 NEALS Annual Meeting

• ALT001 was well tolerated in randomized Phase 1 ALS study, with early signals on Function and NfL
• No treatment-related serious adverse events at any of three doses
• ALS function scores and plasma neurofilament light chain favored ALT001 over placebo at Day 31
• U.S. Phase 1/2a study planned to test higher doses

BEIJING & CLEARWATER, Fla.--(BUSINESS WIRE)--Darwin Biotech (“Darwin”), a clinical-stage company developing treatments for neurological diseases, today announced that data from its randomized, double-blind, placebo-controlled Phase 1 study of ALT001 in amyotrophic lateral sclerosis (ALS), including 12-week results from an ongoing open-label extension (OLE), were presented in a poster at the 2026 Network of Excellence for ALS (NEALS) Annual Meeting, taking place October 27-30 in Clearwater, Florida.

ALT001 is a novel, cell-free protein complex derived from human mesenchymal stem cells. The Phase 1 study, led by Principal Investigator Prof. Yilong Wang and team at Beijing Tiantan Hospital, Capital Medical University, enrolled 36 participants with ALS who were randomized 3:1 to ALT001 at 1.0, 2.0 or 4.0 μg/kg or placebo (27 ALT001; 9 placebo). Primary endpoints were safety, tolerability, maximum tolerated dose and recommended dose. Secondary endpoints included the ALS Functional Rating Scale–Revised (ALSFRS-R), the ALS Assessment Questionnaire-40 (ALSAQ-40), the Modified Norris Scale, forced vital capacity, muscle function and biomarkers including plasma neurofilament light chain (NfL).

Key Phase 1 findings include:

• Safety: ALT001 was generally well tolerated across all dose levels, with no treatment-related serious adverse events (SAEs) or significant intolerance events (SIEs) reported and no dose-limiting toxicities identified.
• ALSFRS-R: At Day 31, mean ALSFRS-R scores increased from baseline by 1.22, 0.78 and 0.44 points in the 1.0, 2.0 and 4.0 μg/kg groups, respectively, compared with a mean decline of 0.78 points with placebo.
• Plasma NfL: At Day 31, mean plasma NfL decreased from baseline by 5.02%, 17.16% and 17.05% in the 1.0, 2.0 and 4.0 μg/kg groups, respectively, compared with 2.23% with placebo.
• Other measures: ALSAQ-40 scores showed favorable trends, Modified Norris Scale scores were generally stable, and grip strength was better preserved at 1.0 and 2.0 μg/kg than with placebo.

Efficacy measures were secondary endpoints, and these results are preliminary given the study’s size and 31-day treatment period.

Following the Phase 1 study, 29 of 36 participants entered a 12-week OLE (11, 8 and 10 participants in the 1.0, 2.0 and 4.0 μg/kg cohorts, respectively), in which participants originally assigned to placebo received ALT001 at their cohort’s dose. Among participants originally treated with ALT001, mean ALSFRS-R change from OLE baseline to Week 12 was −1.5, −3.4 and −1.3 points in the 1.0, 2.0 and 4.0 μg/kg cohorts, respectively. ALSFRS-R trajectories suggested slower functional decline in OLE participants than in Phase 1 participants who did not enter the extension. These findings are exploratory and are based on small, non-randomized groups with different follow-up timelines.

“ALT001 was well tolerated at every dose we tested,” said Weiqi Chen, M.D., Ph.D., Associate Chief Physician and Associate Professor, Department of Neurology, Beijing Tiantan Hospital, Capital Medical University, presenting on behalf of Lead Principal Investigator Prof. Yilong Wang. “Over the month of treatment, patients on ALT001 held their function and saw their NfL levels come down, while patients on placebo declined. These are encouraging early results, and I look forward to studying ALT001 in larger trials.”

Darwin plans to further evaluate ALT001 in a seamless adaptive U.S. Phase 1/2a study. Part A is an open-label dose-escalation phase evaluating 1.0, 3.0 and 9.0 μg/kg in sequential cohorts of three to six participants to identify the recommended Phase 2 dose (RP2D). In Part B, 12 participants will be randomized 3:1 to ALT001 or placebo in a double-blind, placebo-controlled evaluation for up to 24 weeks. Primary endpoints are safety and tolerability, including RP2D selection; secondary endpoints include change from baseline in ALSFRS-R and plasma NfL.

“These results give us a clear path forward for ALT001 globally,” said Wang Yu, Founder and Chief Executive Officer of Darwin. “We are grateful to the patients, their caregivers and doctors at Beijing Tiantan Hospital who made this study possible. We are now planning a Phase 1/2a study in the U.S. that will test higher doses against placebo over a longer period. Our team at the NEALS Annual Meeting has been holding conversations with some of the most prominent U.S. members of the ALS medical community and we look forward to advancing our clinical program in the United States as well.”

Dr. Chen presented the poster on behalf of Principal Investigator Prof. Yilong Wang, titled “Darwin-ALT001 for Amyotrophic Lateral Sclerosis (ALS): Results from a Randomized Phase I Study and Open-Label Extension in China, and Design of a U.S. Phase I/IIa Study,” on Thursday, October 29, 2026, between 4:50 pm – 6:30 pm ET during Poster Session 2 (poster 208).

About ALT001

ALT001 is a novel, independently developed biologic. Its active component is a protein complex derived from the secretome of human umbilical cord-derived mesenchymal stem cells. The therapy is currently in clinical development for neurological conditions, including ALS and ischemic stroke, with ongoing research into its pharmacokinetics, biomarkers, and commercial-scale manufacturing.

About ALS

Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's Disease, is a rare and fatal neurodegenerative disorder characterized by the progressive degeneration of motor neurons in the brain and spinal cord. This leads to muscle weakness, atrophy, and eventually paralysis, typically resulting in death within 3-5 years of symptom onset due to respiratory failure. The global incidence of ALS is approximately 1.5-2.5 per 100,000 people per year, with a prevalence of about 4-6 per 100,000. While ALS can affect individuals of any age, it most commonly presents between the ages of 40 and 70. The disease affects both upper and lower motor neurons, leading to a combination of spasticity, hyperreflexia, muscle weakness, fasciculations, and atrophy. The lack of effective treatments highlights the urgent need for novel therapeutic approaches that can address the complex, multi-factorial pathophysiology of ALS.

About Darwin

Darwin Biotechnology is a clinical-stage biotechnology company that has developed a proprietary, first-of-its-kind platform for mesenchymal stem cell (MSC)-derived protein complexes, together with supporting manufacturing processes and quality control and release systems. Guided by Quality by Design (QbD) principles, the platform enables the development of cell-free, non-exosomal protein complex biologics derived from MSCs. It is designed to preserve the coordinated biological activity of multiple cell-derived proteins while improving manufacturing consistency, product stability, and control over scale-up, laying the foundation for lower production costs and broader patient access. In neurology, Darwin prioritizes serious central nervous system (CNS) and other neurological disorders, with lead clinical programs in amyotrophic lateral sclerosis (ALS) and ischemic stroke. These programs are evaluating the therapeutic potential of targeting multiple disease mechanisms to promote neuroprotection and functional recovery. Darwin integrates basic research, clinical translation, and global regulatory strategy while building its commercial manufacturing infrastructure. The company aims to bring quality-assured, affordable cell-free protein complex therapeutics to patients worldwide. Through collaborations with medical institutions and research teams in China and internationally, Darwin continues to investigate mechanisms of action, generate clinical evidence, and advance the clinical development and application of this innovative technology.

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