Praxis Precision Medicines, Inc. - PRAX STOCK NEWS

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.

Invitae and Praxis Precision Medicines have successfully utilized Invitae's Ciitizen platform to support Praxis' IND application for PRAX-222, a treatment targeting pediatric patients with SCN2A developmental and epileptic encephalopathy. This marks the platform's first use as a source of real-world data in a regulatory filing. The FDA has cleared the IND application for the initial dose cohort of the PRAX-222 EMBRAVE clinical study, emphasizing the importance of innovative data collection in addressing rare diseases where traditional studies are challenging.

Praxis Precision Medicines (NASDAQ: PRAX) announced participation in a fireside chat at the H.C. Wainwright 24th Annual Global Investment Conference on September 14, 2022, at 10:30 a.m. ET. This event will focus on the company's advancements in CNS therapies targeting neuronal excitation-inhibition imbalance. Investors can access the live webcast on the company's website, with a replay available for 90 days after the event. Praxis is developing multiple clinical-stage product candidates for various CNS disorders.

Praxis Precision Medicines (NASDAQ: PRAX) announced the initiation of the PRAX-222 EMBRAVE clinical study, targeting pediatric patients with SCN2A developmental and epileptic encephalopathy (DEE), following FDA clearance of its IND application. The study aims to gather safety and efficacy data for dose escalation. Additionally, Praxis plans to start the Phase 2 study for PRAX-562 outside the U.S. after a clinical hold by the FDA. PRAX-222 has demonstrated significant potential in preclinical models, receiving Orphan Drug Designation from the FDA, while PRAX-562 shows promising results in initial studies.

Praxis Precision Medicines (NASDAQ: PRAX) provided a corporate update and reported financial results for Q2 2022. The company has $165.4 million in cash as of June 30, supporting operations into Q1 2024. Key clinical highlights include encouraging topline results from the Phase 2a study of PRAX-944 for essential tremor and the completion of the Phase 1 study for PRAX-562, confirming its potential therapeutic window. However, the PRAX-114 Phase 2 study for major depressive disorder was discontinued due to failure to meet statistical significance.