Praxis Precision Medicines Updates and Q1 2024 Financial Results

Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company focused on developing treatments for central nervous system disorders, announced several key updates and financial results for the first quarter of 2024.

Praxis reported significant progress in its clinical programs, particularly for PRAX-628, which is being developed as a once-daily oral treatment for focal onset seizures. Positive results were observed in a Phase 2a proof of concept study for PRAX-628 in epilepsy patients. In this study, 100% of patients responded, with a substantial number achieving complete responses at different dosage levels. Based on these encouraging outcomes, Praxis plans to initiate two additional efficacy studies for PRAX-628. The first of these studies is anticipated to begin in the second half of 2024, with topline results expected in 2025, and the second study is expected to start in the first half of 2025, with results expected in the first half of 2026.

The company's Essential3 Phase 3 trials for ulixacaltamide, a treatment for Essential Tremor (ET), also showed strong progress. Since recruitment began in November 2023, more than 50,000 pre-screening forms have been submitted, resulting in over 7,000 referrals meeting the pre-qualifying eligibility criteria. Praxis aims to release topline results from these trials in the second half of 2024, which will support a planned New Drug Application (NDA) submission in 2025.

Additionally, Praxis completed the randomization phase of its PRAX-562 Phase 2 EMBOLD study, which targets pediatric patients with developmental and epileptic encephalopathies (DEEs). Topline results from this study are expected in the third quarter of 2024. PRAX-562 is designed to inhibit persistent sodium current, a key driver of seizure symptoms in DEEs.

Financially, Praxis bolstered its resources through two public offerings completed in 2024, raising net proceeds of approximately $161.6 million and $215.8 million, respectively. As of April 30, 2024, the company had $451.2 million in cash and investments, which is expected to fund operations into 2027.

For the first quarter of 2024, Praxis reported $243.3 million in cash, cash equivalents, and marketable securities. Research and development expenses for the quarter were $27.0 million, up from $25.5 million in the same period last year, primarily due to increased personnel expenses and costs associated with the Cerebrum™ platform. General and administrative expenses rose to $15.3 million from $13.3 million in the previous year, attributed mainly to higher personnel-related costs. The net loss for the first quarter of 2024 was $39.6 million, compared to a net loss of $37.5 million for the same period in 2023.

Praxis has several promising candidates in its pipeline. Ulixacaltamide is a highly selective small molecule inhibitor targeting T-type calcium channels to mitigate tremor activity in ET. PRAX-628 is designed to target hyperexcitable voltage-gated sodium channels in the brain for treating focal onset seizures, demonstrating superior selectivity and potency in preclinical and clinical studies. Another candidate, Elsunersen, an antisense oligonucleotide (ASO), aims to decrease SCN2A gene expression to treat seizures and other symptoms in patients with SCN2A gain-of-function mutations.

Praxis is committed to advancing its diversified portfolio of CNS therapies, leveraging genetic insights to develop precision medicines. Through its Cerebrum™ small molecule platform and Solidus™ ASO platform, Praxis continues to make strides in addressing both rare and prevalent neurological disorders.