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Praxis Precision Medicines, Inc. (NASDAQ: PRAX) is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for central nervous system (CNS) disorders characterized by neuronal imbalance. By leveraging genetic insights, Praxis aims to address conditions ranging from major depressive disorder to epilepsy.
The company's lead product candidates include:
- Prax-114: An extrasynaptic-preferring GABAA receptor positive allosteric modulator, currently in Phase IIa trials for treating major depressive disorder and perimenopausal depression.
- Prax-944: A selective small molecule inhibitor of T-type calcium channels, in Phase IIa trials for essential tremor treatment.
- Prax-562: A persistent sodium current blocker in Phase I trials aimed at severe pediatric epilepsy and adult cephalgia.
- Prax-222 (Elsunersen): An antisense oligonucleotide targeting gain-of-function SCN2A epilepsy, under collaboration with Ionis Pharmaceuticals and RogCon, Inc.
- PRAX-628: A next-gen small molecule targeting sodium-channels in the brain, being developed as a treatment for focal epilepsy.
- Ulixacaltamide: A small molecule inhibitor for essential tremor, currently in late-stage development.
Recent news highlights include a partnership with Tenacia to extend ulixacaltamide's reach to Greater China, and advances in the PRAX-628 program demonstrating its potential as a best-in-class epilepsy drug.
Financially, Praxis has shown significant progress. As of the latest reports, the company has sufficient funding to continue operations into 2027, driven by strategic public offerings and a lean operational model.
For more details, visit www.praxismedicines.com and follow them on social media channels.
Praxis Precision Medicines (NASDAQ: PRAX) outlined its 2025 corporate strategy, highlighting three late-stage clinical trials with blockbuster potential and four pivotal readouts expected in 2025. The company anticipates four commercial assets by 2028.
Key developments include: Study 1 of Essential3 program for ulixacaltamide in essential tremor with Q1 2025 interim analysis; EMBOLD study of relutrigine progressing towards 2026 NDA filing; vormatrigine ENERGY program advancing with RADIANT and POWER1 readouts in 2025. UCB has exercised its option to license the KCNT1 small molecule candidate.
The company reports approximately $470 million in cash and investments at the end of 2024, supporting operations into 2028. Multiple clinical programs are advancing, including treatments for essential tremor, epilepsies, and developmental and epileptic encephalopathies (DEEs).
Praxis Precision Medicines (NASDAQ: PRAX) has announced its participation in the 43rd Annual J.P. Morgan Healthcare Conference. The company, which focuses on developing therapies for central nervous system (CNS) disorders, will present a corporate overview at the conference on January 15, 2025, at 2:15pm PST at the Westin St. Francis Hotel in San Francisco.
The presentation will be accessible through a live webcast, and a replay will remain available for 30 days through the company's website at www.praxismedicines.com in the Investors + Media section under 'Upcoming & Recent Events'.
Praxis Precision Medicines (NASDAQ: PRAX) has received Rare Pediatric Disease Designation (RPDD) from the FDA for relutrigine in treating Dravet syndrome, a genetic developmental and epileptic encephalopathy. This marks the third RPDD for relutrigine, following previous designations for SCN2A and SCN8A DEEs.
The company plans to initiate an all-DEE trial (EMERALD) in 1H2025. Recent clinical results from the EMBOLD study showed promising outcomes, including a 46% placebo-adjusted monthly motor seizure reduction, over 30% of patients achieving seizure freedom, and a 77% reduction in median seizure rate during long-term extension. Praxis is currently enrolling patients in a second, registrational cohort for SCN2A and SCN8A patients, with topline results expected in 1H2026.
Praxis Precision Medicines (NASDAQ: PRAX) has announced its participation in two major investor conferences in December 2024. The company will present at the Piper Sandler 36th Annual Healthcare Conference in New York on December 4, featuring a fireside chat at 2:00pm ET. Additionally, Praxis will participate in the Oppenheimer Movers in Rare Disease Summit on December 12, where management will join a panel discussing rare disease companies with near-term catalysts.
Both events will take place in New York, with one-on-one meeting opportunities available for interested investors. A live webcast of the Piper Sandler presentation will be accessible through the company's website, with a replay available for 90 days afterward.
Praxis Precision Medicines (NASDAQ: PRAX) announced its participation in the 2024 American Epilepsy Society Annual Meeting, where it will present data from three epilepsy programs. The company will showcase updates through five poster presentations featuring their clinical-stage assets, including relutrigine and vormatrigine, described as the most potent and selective anti-seizure medications developed to date. The presentations will focus on preclinical and clinical data, including results from the EMBOLD study in childhood epilepsies. The event will be held from December 6-10, 2024, in Los Angeles, with Praxis exhibiting at booth #1235 and hosting a scientific exhibit.
Praxis Precision Medicines (NASDAQ: PRAX) has announced its participation in three major investor conferences in November 2024. The company will present at the Truist Securities BioPharma Symposium in New York on November 7 at 9:40am ET, the Guggenheim Securities Inaugural Healthcare Innovation Conference in Boston on November 11 at 1:00pm ET, and the Jefferies London Healthcare Conference in London on November 20-21.
Management will participate in fireside chats at both Truist and Guggenheim events, with the Guggenheim session being available via live webcast. One-on-one meetings will be available at all three conferences. The Guggenheim webcast will be accessible through the company's website for 90 days after the event.
Praxis Precision Medicines reported Q3 2024 financial results and provided corporate updates. The company maintains cash runway into 2027 with $411.2 million in cash and equivalents. Key highlights include planned interim analysis for ulixacaltamide's Essential3 Phase 3 program in Q1 2025, positive topline results from EMBOLD cohort 1 showing 46% placebo-adjusted reduction in monthly motor seizures, and advancement of vormatrigine trials. Q3 financial results showed a net loss of $51.9 million, with R&D expenses at $41.9 million and G&A expenses at $15.3 million. The company recognized $0.3 million in collaboration revenue.
Praxis Precision Medicines (NASDAQ: PRAX), a clinical-stage biopharmaceutical company focused on developing CNS disorder therapies, announced it will release its Q3 2024 financial results on Wednesday, November 6, 2024, before market open. The company will host a live webcast at 8am ET on the same day. Investors can access the webcast through a registration link or the company's website. A replay will be available for 90 days on the company's website.
Praxis Precision Medicines (NASDAQ: PRAX) has announced its participation in the Movement Disorders Society (MDS) 2024 International Congress in Philadelphia, Pennsylvania, from September 27 to October 1, 2024. The company will present a poster and platform talk showcasing its Phase 3 clinical program for ulixacaltamide, a potential therapy for adults with essential tremor (ET).
The presentations will focus on the Essential3 program, an innovative multi-study Phase 3 initiative evaluating the efficacy and safety of ulixacaltamide. Praxis will be available at booth #1008 and will deliver a platform presentation on September 28 and a poster presentation on September 29. Presentation materials will be made available on the company's website following the conference.
Praxis Precision Medicines (NASDAQ: PRAX), a clinical-stage biopharmaceutical company focusing on central nervous system (CNS) disorders, has announced its participation in two upcoming investor conferences. The company will present at the H.C. Wainwright 26th Annual Global Investment Conference on September 9, 2024, at 12:00pm EDT in New York, NY. Additionally, Praxis will attend the Baird Global Healthcare Conference on September 11, 2024, at 2:35pm EDT, also in New York, NY.
Both presentations will be in a fireside chat format. The company will be available for one-on-one meetings with interested investors during these conferences. A replay of the presentations will be available on Praxis' website for 90 days after the events.
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