Praxis Precision Medicines, Inc. Stock Price, News & Analysis

Praxis Precision Medicines (NASDAQ: PRAX) announced topline results from the Phase 2b Essential1 study of ulixacaltamide for treating essential tremor. The primary endpoint of improvement in modified Activities of Daily Living (mADL) did not reach statistical significance (p=0.126), but a secondary endpoint, the TETRAS-ADL, showed nominal significance (p=0.026). The drug was well tolerated, with no new safety findings reported. Praxis plans to engage with the FDA for an end-of-Phase 2 meeting and initiate a Phase 3 study in 2H23, as the unmet need for effective therapies for essential tremor persists.

Praxis Precision Medicines (NASDAQ: PRAX) provided a corporate update and financial results for Q4 and full year 2022, highlighting a transformative 2023 ahead with topline results from the ulixacaltamide Essential1 study expected in 1Q23. The company reported cash and investments of $100.5 million as of December 31, 2022, which funds operations into 1Q24. R&D expenses increased to $155 million in 2022, with net losses of $214 million.

Key milestones for 2023 include results from their clinical programs, including PRAX-562 and PRAX-628. A strategic collaboration with UCB could yield up to $100 million in milestone payments for the KCNT1 program.

Praxis Precision Medicines (NASDAQ: PRAX) has announced a strategic collaboration with UCB focused on developing small molecule therapeutics for KCNT1 related epilepsies, which currently have no approved treatments. The partnership will allow Praxis to leverage UCB's experience in epilepsy drug development. Praxis will receive an upfront payment and could earn up to approximately $100 million in milestone payments, along with royalties from any resulting products. This collaboration underscores Praxis' commitment to advancing treatments for genetic epilepsy disorders.

BOSTON, Nov. 28, 2022 – Praxis Precision Medicines (NASDAQ: PRAX) announced presentations regarding its epilepsy programs at the American Epilepsy Society (AES) 2022 Annual Meeting from December 2-6, 2022. The company will discuss its programs PRAX-222, PRAX-562, and PRAX-628, including first-in-patient studies and findings on developmental and epileptic encephalopathies (DEE). Key studies highlight PRAX-562's anticonvulsant activity across multiple models and insights into SCN2A and SCN8A-related DEE, potentially influencing future treatment endpoints and therapies.

Praxis Precision Medicines (PRAX) has received FDA authorization to proceed with the PRAX-562 Phase 2 EMBOLD study targeting pediatric patients with developmental and epileptic encephalopathies (DEEs). The study will commence in Q1 2023, focusing on SCN2A-DEE and SCN8A-DEE cohorts. Topline results are expected in late 2023. PRAX-562 aims to offer a precision treatment option, using a sodium channel inhibitor that has shown promising results in preclinical models. The company has received multiple orphan drug designations, underscoring the potential impact of this treatment.

Praxis Precision Medicines (NASDAQ: PRAX) reported a Q3 2022 net loss of $43.9 million, with cash and investments at $123.7 million, expected to fund operations until Q1 2024. Upcoming topline results for the PRAX-944 Essential1 study on essential tremor are anticipated in Q1 2023, while the PRAX-222 EMBRAVE study for SCN2A-DEE is set to initiate in Q4 2022. The company has adjusted its Parkinson's disease study design and plans to meet with the FDA regarding Phase 3 development following Essential1 results.

Praxis Precision Medicines (NASDAQ: PRAX) announced participation in two investor conferences. Management will join the Guggenheim’s 4th Annual Immunology and Neurology Conference on November 14, 2022, at 10:45 a.m. ET, and the Piper Sandler 34th Annual Healthcare Conference on November 29, 2022, at 1:00 p.m. ET. Both events will feature live webcasts available on the company’s website, with replays accessible for 30 days post-event. Praxis is focused on developing therapies for CNS disorders using genetic insights, with a portfolio that includes four clinical-stage product candidates.

Praxis Precision Medicines (NASDAQ: PRAX) announced its participation in a fireside chat at the Guggenheim Nantucket Therapeutics Conference on September 28, 2022, at 11:45 a.m. ET. The event will be broadcasted live on the company’s website, with a replay available for 30 days post-event. Praxis specializes in developing therapies for central nervous system disorders, utilizing genetic insights to address neuronal excitation-inhibition imbalances. The company boasts a diverse portfolio, including four clinical-stage product candidates aimed at treating various neurological conditions.

Praxis Precision Medicines has successfully utilized real-world data from Invitae's Ciitizen platform to support its IND application for PRAX-222, aimed at treating pediatric patients with SCN2A developmental and epileptic encephalopathy. This marks a significant advancement as it represents the first use of a patient-mediated data source for regulatory filings with the FDA. The comprehensive data collection method overcomes traditional challenges in rare disease research, offering a unique patient-centered approach. The trial has received FDA Orphan Drug and Rare Pediatric Designation.