Placebo-adjusted monthly motor seizure reduction of 46% during double-blind period Over 30% of patients achieved seizure freedom status while on relutrigine Meaningful gains observed in alertness, communication and seizure severity 75% reduction in median seizure rate seen for patients in the long-term extension Registrational phase of the EMBOLD study for SCN2A and 8A initiated BOSTON, Mass., Sept. 03, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceuti ...

BOSTON, Mass., Sept. 02, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that it will share the topline results from the Phase 2 EMBOLD study of relutrigine (PRAX562) in SCN2A and SCN8A Developmental and Epileptic Encephalopathies (DEEs) on Tuesday, September 3, ...

Praxis Precision Medicines, Inc. (PRAX) could be a solid choice for investors given its recent upgrade to a Zacks Rank #2 (Buy). This rating change essentially reflects an upward trend in earnings estimates -- one of the most powerful forces impacting stock prices. A company's changing earnings picture is at the core of the Zacks rating. The system tracks the Zacks Consensus Estimate -- the consensus measure of EPS estimates from the sell-side analysts covering the stock -- for the current and following yea ...

Praxis Precision Medicines, Inc. (NASDAQ:PRAX) Q2 2024 Earnings Conference Call August 13, 2024 8:00 AM ET Company Participants Marcio Souza - President and Chief Executive Officer Tim Kelly - Chief Financial Officer Daniel Ferry - Investor Relations, Life Science Advisors Conference Call Participants Yasmeen Rahimi - Piper Sandler Joon Lee - Truist Securities Francois Brisebois - Oppenheimer Yatin Suneja - Guggenheim Ritu Baral - TD Cowan Joel Beatty - Baird Ami Fadia - Needham Douglas Tsao - HC Wainwright ...

Financial Data and Key Metrics Changes - In Q2 2024, operating expenses were $37.8 million, with $27.3 million allocated to R&D and $10.6 million to G&A [16] - Operating cash spent increased to $27.4 million in Q2 from $20.9 million in Q1 2024, reflecting heightened activity for Essential3 studies [16] - Cash equivalents and marketable securities rose to $433.8 million from $81 million in December, primarily due to net proceeds from public offerings [16] Business Line Data and Key Metrics Changes - The lead clinical program, Ulixacaltamide, is progressing towards registration with pivotal Essential3 trials in essential tremor expected to yield topline results later this year [5][6] - PRAX-628 is undergoing a comprehensive clinical program with three interventional studies in epilepsy patients and an observational study in collaboration with the Epilepsy Study Consortium [5][10] - The RADIANT study for PRAX-562 in pediatric patients with developmental and epileptic encephalopathies is on track for topline results in the first half of 2025 [6][12] Market Data and Key Metrics Changes - The unmet needs for essential tremor patients are significant, with millions in the US requiring therapeutic options [7] - The Essential3 program is the largest and most comprehensive study conducted to date for essential tremor, incorporating a decentralized design to reduce patient burden [8][9] Company Strategy and Development Direction - The company aims to have up to four programs in the registrational phase by 2025, focusing on precision medicines for CNS disorders [5][6] - The strategy includes building a robust efficacy package for PRAX-628 through multiple studies, with a focus on both focal and generalized epilepsy [11][12] - The company is committed to filing an NDA for Ulixacaltamide next year, aiming to be the first pharmacologically approved treatment for essential tremor in a decade [10][40] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the design and execution of the Essential3 program, emphasizing the importance of maximizing the program's success [9][10] - The upcoming readouts for various studies are anticipated to provide critical data that could influence future regulatory submissions and market opportunities [14][30] - Management highlighted the potential for PRAX-628 to address a large number of genetically defined epilepsies, indicating a significant market opportunity [31][32] Other Important Information - The company is fully funded through several key readouts, with cash runway extending into 2027 [16] - The interim analysis for the Essential3 study is designed to enhance the probability of success without indicating any current issues with enrollment or program execution [20][22] Q&A Session Summary Question: Enrollment status and interim analysis for Essential3 - Management confirmed that enrollment is on track and the interim analysis is a housekeeping exercise to potentially increase sample size if necessary [19][20] Question: Expectations for relutrigine data in DEEs - Management anticipates a 20% to 30% seizure reduction as a significant outcome, with potential implications for a broader range of DEEs [30][31] Question: Differences in study designs for Essential Tremor - Management explained the rationale behind using both parallel and randomized withdrawal designs to better reflect real-world patient behavior [39][40] Question: Clarifications on RADIANT and POWER studies - RADIANT is not placebo-controlled, while POWER1 and POWER2 are. Dosing strategies differ to maximize efficacy and flexibility in patient management [52][73] Question: Feedback from regulators on Elsunersen - Management indicated ongoing discussions with global regulators regarding the initiation of pivotal studies in Brazil and plans for expansion in the US and Europe [75]

This company is expected to post quarterly loss of $2.39 per share in its upcoming report, which represents a year-over-year change of +67.5%. Revenues are expected to be $1.67 million, up 114.1% from the year-ago quarter. The stock currently carries a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks Rank #1 (Strong Buy) stocks here >>>> The sudden rise in the stock price was observed after its competitor firm, Jazz Pharmaceuticals, announced the failure of its mid-stage study evaluating ...

Core Insights - Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance [1][4] - The company granted stock options and restricted stock units to a new non-executive employee as part of its 2024 Inducement Plan, which is designed to attract new talent [1][2] Stock Options and Awards - The Compensation Committee granted non-qualified stock options to purchase 300 shares and restricted stock units covering 225 shares to a new employee [1] - The options have an exercise price of $47.05 per share, equal to the closing price on the grant date, and will vest over four years [3] - The vesting schedule includes 25% vesting on the first anniversary and the remainder in equal monthly installments over the following three years, contingent on continued employment [3] Company Overview - Praxis utilizes genetic insights to develop therapies for both rare and prevalent neurological disorders through its proprietary platforms, Cerebrum™ and Solidus™ [4] - The company has a diversified CNS portfolio with multiple programs across movement disorders and epilepsy, including four clinical-stage product candidates [4]

BOSTON, May 17, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that management will be participating in three upcoming conferences. Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating insights from genetic epilepsies into the de ...

BOSTON, May 17, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that management will be participating in three upcoming conferences. About Praxis Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating insights from genetic epilepsie ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 (Exact Name of Registrant as Specified in its Charter) Delaware 47-5195942 (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification No.) FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transiti ...