Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/TH2 Cell Therapy (RAPA-501) of Amyotrophic…

Description

Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/TH2 Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis - ALS Is a Lethal Neurodegenerative Disease Accelerated by Neuroinflammation. Current FDA-Approved Therapies Have Modest Benefits and Do Not Address Inflammation. To Address This, Rapa Therapeutics, LLC (Rapa) Has Developed an Autologous T Cell Therapy (RAPA-501) That Reduces Inflammation, with the Goal of Reducing ALS Morbidity and Mortality. RAPA-501 Are Manufactured Ex Vivo to Attain Dual TREG/TH2 Anti- Inflammatory Activity and a T-Stem Phenotype That Permits T Cell Therapy Without Conditioning Chemotherapy. IN an Ongoing Clinical Trial of RAPA-501 in People with ALS (Pwals) (NCT04220190), RAPA-501 Cells Were Found to Be Safe (No Product-Related Adverse Events), Biologically Active (Diverse Anti-Inflammatory Effects in Pwals), and Showed Early Trends Toward Stabilizing Pulmonary Function Decline. A Phase 2/3 Expansion Cohort Was Added to the Trial to Assess Whether RAPA-501 Is Efficacious in Standard-Risk Pwals. We Will Extend RAPA-501 Therapy to Pwals Not Eligible for This Ongoing Phase 2/3 Trial or Other ALS Trials, Which Nearly Universally Require That Participants Have a Slow Vital Capacity (SVC) Value of =50% of Predicted Normal. The Proposed EAP Will Enroll Pwals Who Have SVC Values <50%. This Population of Pwals Is Considered “High Risk” (~50% Chance of Respiratory Failure or Death Within 180 Days) and Thus Particularly Suitable for Experimental Immune Therapies Such as RAPA-501. IN Addition, the RAPA-501-EAP Will Not Exclude Pwals Who Have a Prolonged Time from ALS-Related Symptoms or Low Alsfrs-R Scores. Participants Will Receive Four RAPA-501 IV Infusions (Every 42-DAYS at Established Safe Dose, 80 X 106 Cells/Infusion). This RAPA-501-EAP Will Further Evaluate the Safety of This Therapy, Expand an Understanding of the RAPA-501 Therapeutic Mechanism of Action, and Evaluate Signals of Efficacy in This Real-World Population of Pwals Using Standard Methods and Origent Data Sciences Machine Learning ALS Prediction Algorithms. The RAPA-501 EAP Will Be Led by Investigators at Mass General Hospital (Mgh; Drs. Berry, Babu, and Paganoni) and Sponsored by Rapa, Which Is Responsible for RAPA-501 Manufacturing and FDA Regulatory Filings Under Existing Ind 019480 (Dr. Fowler, Sponsor). Clinical Trial Site Investigators Have Experience with RAPA-501 Therapy (Mgh; Hackensack University Medical Center; and Mayo Clinic Arizona) or Other Cells Therapies. Sites Are Geographically Diverse and Likely to Accrue a Significant Number of Underserved Pwals (U of Iowa; U of Idaho; Providence Hospital, Portland, Oregon; Uc-Irvine; Columbia, Nyc). IN Addition, Several Research Collaborations Will Emanate from the Intensive Study of the Clinically-Annotated, Valuable Research Samples Obtained from the RAPA-501 Eap.

Award details