On January 8, 2025, MSQ Ventures hosted Dr. Joan Fallon, Founder and CEO of Curemark, for an insightful webinar titled Advancements in Autism Treatment: Curemark's Pioneering Approaches. The session explored groundbreaking advancements in autism treatments, highlighting Curemark’s innovative work on CMAT, a drug targeting the core symptoms of autism through a gut-brain connection.

Curemark’s Mission and Origins

Founded in 2007, Curemark is a biopharmaceutical company addressing serious neurodevelopmental diseases, particularly autism. The company’s flagship product, CM-AT, is a first-in-class disease-modifying therapy. Curemark’s pipeline also includes treatments for ADHD, Parkinson’s, and schizophrenia, leveraging enzyme deficiency research to develop innovative therapies.

Scientific Innovation: CM-AT’s Mechanism

CM-AT addresses a deficiency in chymotrypsin, a critical enzyme for protein digestion, which indirectly impacts neurotransmitter production such as serotonin and dopamine. The drug’s novel lipid-encapsulation technology ensures effective delivery, particularly for children who struggle with traditional medication forms.

Rigorous Clinical Trials

Curemark conducted two double-blind, placebo-controlled Phase 3 trials, demonstrating CM-AT’s efficacy in reducing core autism symptoms, including irritability, social withdrawal, and inappropriate speech. A delayed-start analysis revealed CM-AT’s potential as a disease-modifying treatment, marking a significant milestone in autism research.

Addressing Global Needs

Autism prevalence continues to rise, with significant unmet needs worldwide. Curemark aims to position CM-AT as the standard of care for children diagnosed as early as age three, providing long-term improvements in communication, behavior, and socialization. Curemark’s research also supports potential therapies for ADHD, Parkinson’s, and schizophrenia, with plans for Phase 2 trials in these areas.

Future Outlook

With FDA fast-track designation, Curemark anticipates U.S. regulatory approval by late 2025. The company is exploring global partnerships to ensure equitable access to CM-AT while advancing its pipeline for other neurodevelopmental conditions.

Dr. Fallon emphasized Curemark’s mission to provide transformative solutions for children and families, ensuring that every child in need has access to these groundbreaking treatments.