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

Summary

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