Praxis Precision Medicines Advances CNS Pipeline with Key Clinical Trial Milestones
Key Insights
Praxis Precision Medicines' PRAX-114 Phase 2/3 clinical trial for Major Depressive Disorder (search) (MDD) is set to begin in March 2021 after IND clearance.
Topline data from the PRAX-944 Phase 2a high dose cohort in essential tremor (search) is expected in mid-year 2021, informing further development.
A collaboration with The Florey Institute (search) expands Praxis's pipeline with three ASOs targeting rare epilepsies (search), enhancing their rare disease portfolio.
Praxis Precision Medicines is making strides in its central nervous system (CNS) pipeline, with several key milestones achieved and upcoming clinical trials. The company's focus on translating genetic insights into therapies for CNS disorders characterized by neuronal imbalance is driving progress across mood disorders, movement disorders, and rare diseases.
PRAX-114 for Major Depressive Disorder
The U.S. Food and Drug Administration (FDA) has cleared Praxis' Investigational New Drug (IND) application for PRAX-114, a GABAA (search) positive allosteric modulator, for the treatment of major depressive disorder (search) (MDD). The Phase 2/3 monotherapy MDD trial is expected to begin in the United States and Australia by the end of March 2021, with topline data anticipated in the first half of 2022. This trial is intended to serve as one of two registrational trials required by the FDA to support clinical efficacy of PRAX-114 for monotherapy treatment of MDD.
Marcio Souza, president and chief executive officer of Praxis, stated, "Increasing rates of depression worldwide highlight the urgent need to develop new, differentiated treatments for MDD like PRAX-114."
Praxis also plans to initiate a PRAX-114 Phase 2 dose range finding trial for adjunctive treatment of MDD in the third quarter of 2021. Data from Part C of the PRAX-114 Phase 2a clinical trial showed that PRAX-114 was generally well-tolerated, with a rapid and marked improvement in the HAM-D score, with LS Mean improvement of 11 points at Day 15 that remained stable through the end of the active treatment period.
PRAX-944 for Essential Tremor
Praxis is currently conducting a two-cohort, Phase 2a, open-label trial of PRAX-944 in essential tremor (search) (ET). Preliminary data from six participants in the low dose cohort showed tremor amplitude reduction of >40% in the upper limb. A second cohort has been initiated, in which participants will be titrated to a dose of up to 120 mg/day of PRAX-944. Preliminary topline open-label safety, tolerability, and efficacy data for the high dose cohort is expected in mid-year 2021.
The company plans to initiate a Phase 2b randomized, double-blind, placebo-controlled clinical trial of PRAX-944 in ET in the fourth quarter of 2021.
Advancements in Rare Disease Programs
Praxis has completed the single ascending dose (SAD) portion in its Phase 1 clinical trial of PRAX-562 in healthy volunteers and the study has advanced to multiple ascending dose (MAD) cohorts. The company expects to initiate the initial proof-of-concept trial of PRAX-562 in the second half of 2021 in patients with rare adult cephalgias, including Trigeminal Neuralgia (search) (TN), Short-lasting Unilateral Neuralgiform headache attacks with Conjunctival injection and Tearing (SUNCT), and Short-lasting Unilateral Neuralgiform headache with Autonomic symptoms (SUNA).
IND-enabling toxicology studies for PRAX-222, an antisense oligonucleotide (ASO) candidate designed to down-regulate SCN2A (search) expression in SCN2A gain-of-function mutations, are expected to be completed by the end of 2021.
In March 2021, Praxis entered into a research collaboration with The Florey Institute (search) of Neuroscience and Mental Health to develop three novel ASOs, including a lead program for the treatment of SCN2A (search) loss-of-function mutations.
Financial Position
As of December 31, 2020, Praxis had $296.6 million in cash and cash equivalents, which is expected to fund operations into the fourth quarter of 2022.