Praxis Precision Medicines Reports Positive Topline Results from PRAX-944 Phase 2a Study in Essential Tremor Patients

PRAX-944 demonstrated clinically meaningful functional improvement in essential tremor patients in Part B of Phase 2a study

Mean improvement at Day 42 was 42% as measured by Modified ADL, followed by statistically significant difference in the placebo-controlled randomized withdrawal phase

PRAX-944 continues to be generally well-tolerated

BOSTON, May 09, 2022 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced positive topline results from Part B of its Phase 2a study evaluating the safety and efficacy of PRAX-944 for the treatment of essential tremor (ET). In the study, treatment with PRAX-944 resulted in clinically meaningful improvements in function, which were supported by improvements in tremor amplitude.

In the open-label period through Day 42, patients treated with PRAX-944 demonstrated mean improvement from baseline of 42% in the Modified Activities of Daily Living (ADL) score (N=11, nominal p<0.05). Following randomization, the difference between patients who remained on treatment (N=6) through Day 56 and those randomized to placebo (N=5) was clinically and statistically significant. The Modified ADL is a composite score based on TETRAS ADLs with the addition of spiral drawings and handwriting from the TETRAS performance scale.

“The data from the PRAX-944 Phase 2a study offer hope to people living with essential tremor, for a treatment developed specifically for ET,” said Bernard Ravina, M.D., chief medical officer of Praxis. “In the study, PRAX-944 demonstrated clear functional benefit while on treatment that was lost once treatment was withdrawn, with no new safety findings. Following these exciting proof-of-concept results, we intend to update the primary endpoint for the Phase 2b Essential1 study from safety to efficacy, putting us one step closer to a potential new treatment option for people living with ET.”

Part B of the Phase 2a study of PRAX-944 for the treatment of ET included both an open-label and randomized withdrawal period. In the open-label period, participants were to be titrated up to a maximum dose of 120 mg over 28-days prior to a stable period at the highest dose reached from Day 29 to Day 42. Participants who remained in the study through Day 42 were then randomized one-to-one to either active drug or placebo from Day 43 to Day 56, with a subsequent safety follow-up visit at Day 70.