Praxis Precision Medicines (PRAX) 2025 Conference June 04, 2025 08:10 AM ET Speaker0 Good morning everyone. Welcome to the Jefferies Global Healthcare Conference twenty twenty five. With me today I have the great pleasure of introducing Praxis Precision Medicine's CEO and dear friend Marcio Souza. Marcio, how are you doing this morning? Speaker1 Doing great. Living the dream. Speaker0 Amazing. Well, we'll just jump right into it because there's so much exciting work you guys are doing at Praxis. Top of mind ...

Praxis Precision Medicines (PRAX) FY 2025 Conference May 20, 2025 09:30 AM ET Speaker0 Okay. Welcome, everybody. I'm Doug Sal, senior analyst at H. C. Wainwright. We are thrilled to have with us Praxis Precision Medicines, represented by the company's CFO, Tim Kelly. And Marcio Sauso, the company's CEO, is supposedly en route, so he might join us midway through the presentation. So you don't have to worry if somebody barges in and sits down. Speaker1 So it's Marcio versus the trains this morning. So I I thi ...

BOSTON, May 20, 2025 (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 be participating in three upcoming investor conferences: About Praxis The Company will be available for one-on-one meetings during all events. Interested investors should contact their ...

CORPORATE OVERVIEW May, 2025 ® Forward-looking statements This presentation may contain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to express or implied statements regarding the current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, , including statements regarding the estimated market for our product ...

Praxis Precision Medicines (PRAX) Update / Briefing May 02, 2025 10:00 AM ET Speaker0 Good morning, everyone, and thanks for being with us here today in the office in Boston. I really appreciate it. And everyone else in the webcast as well for our GE portfolio updates at Praxis. As you can see here at the beginning, the way we tend to think about everything at Praxis is daring for more. So we're there for more today as well. I think it's a great show here in a not so sunny day in Boston, and hopefully, gonn ...

Financial Performance - The company reported a net loss of $69.3 million for the three months ended March 31, 2025, with an accumulated deficit of $906.0 million[101]. - Net loss for the three months ended March 31, 2025, was $69.3 million, compared to a net loss of $39.6 million for the same period in 2024[120]. - Net cash used in operating activities for the three months ended March 31, 2025, was $53.0 million, compared to $20.9 million for the same period in 2024[135]. - Total operating expenses for the three months ended March 31, 2025, were $74.7 million, up from $42.3 million in 2024[120]. - Collaboration revenue decreased by $0.4 million to $0 for the three months ended March 31, 2025, due to the completion of research service obligations under the Collaboration Agreement with UCB[121]. Research and Development - Research and development expenses for the three months ended March 31, 2025, totaled $60.8 million, a significant increase from $27.0 million in the same period of 2024[112]. - The Cerebrum™ platform incurred $42.8 million in research and development expenses for the first quarter of 2025, compared to $12.9 million in the prior year[112]. - Research and development expenses increased by $33.8 million to $60.8 million for the three months ended March 31, 2025, primarily driven by increased spending on the Cerebrum™ platform[122]. - The company plans to initiate the Phase 3 registrational study for elsunersen (formerly PRAX-222) in mid-2025, with topline results expected in the first half of 2026[99]. - The EMPOWER study for vormatrigine in epilepsy patients was initiated in Q3 2024, with topline results expected by mid-2025[97]. - The company anticipates nominating a development candidate for PRAX-100 by mid-2025, with additional candidates expected by the end of 2025[99]. - The company relies on external consultants and organizations for its research and development activities, operating in a "virtual" model since inception[100]. - The Independent Data Monitoring Committee recommended stopping the ulixacaltamide study for futility, but the company will continue both studies to completion[97]. Financial Position and Resources - The company has cash, cash equivalents, and marketable securities of $472.0 million as of March 31, 2025, expected to fund operations into 2028[106]. - Cash, cash equivalents, and marketable securities as of March 31, 2025, totaled $472.0 million[126]. - The company expects substantial increases in expenses related to ongoing research and development activities, particularly for clinical trials[139]. - The company anticipates that its cash resources will be sufficient to fund operations into 2028, based on current estimates[140]. - The company currently lacks committed external sources of funds, which may impact its ability to access capital during market volatility[145]. - If additional funds are raised through collaborations or licensing, the company may have to relinquish valuable rights to its technologies or future revenue streams[146]. - The company raised $1.2 billion in aggregate cash proceeds from various transactions since inception through March 31, 2025[126]. Accounting and Risk - The management's financial analysis is based on condensed consolidated financial statements prepared under U.S. GAAP, requiring estimates and assumptions that may differ from actual results[147]. - There have been no changes to critical accounting policies from those described in the Annual Report on Form 10-K filed on February 28, 2025, except as disclosed in the quarterly report[148]. - Recently issued accounting standards are not expected to have a material impact on the company's financial statements or operations[149]. - The company is exposed to market risk related to interest rate changes, but an immediate change of 100 basis points would not materially impact its financial position[150].

Financial Performance - Praxis reported a net loss of $69.3 million for Q1 2025, compared to a net loss of $39.6 million in Q1 2024[12]. - Net loss for Q1 2025 was $69,296,000, compared to a net loss of $39,553,000 in Q1 2024, representing an increase in loss of approximately 75.3%[26]. - Net loss per share attributable to common stockholders increased from $2.84 in Q1 2024 to $3.29 in Q1 2025, a rise of about 15.8%[26]. - Accumulated deficit grew from $836,740,000 on December 31, 2024, to $906,036,000 on March 31, 2025, an increase of about 8.3%[24]. Research and Development - Research and development expenses increased to $60.8 million for Q1 2025, up from $27.0 million in Q1 2024, primarily due to increased expenses related to the Cerebrum™ platform[10]. - Research and development expenses increased to $60,806,000 in Q1 2025, up from $26,984,000 in Q1 2024, reflecting a rise of approximately 125.5%[26]. - Total operating expenses rose to $74,728,000 in Q1 2025, compared to $42,317,000 in Q1 2024, an increase of about 76.4%[26]. - Praxis plans to initiate pivotal studies for relutrigine and elsunersen by mid-2025, targeting developmental and epileptic encephalopathy[1]. Cash and Investments - Praxis reported cash and investments of $472 million as of March 31, 2025, maintaining a runway into 2028[1]. - Cash and cash equivalents decreased by $49,805,000, from $215,372,000 to $165,567,000, representing a decrease of about 23.1%[24]. - Total assets decreased from $483,110,000 on December 31, 2024, to $478,736,000 on March 31, 2025, a decline of approximately 0.8%[24]. Collaboration Revenue - Praxis did not recognize any collaboration revenue in Q1 2025, a decrease from $0.4 million in Q1 2024, due to the completion of obligations under an agreement with UCB[9]. - Collaboration revenue dropped to $0 in Q1 2025, down from $431,000 in Q1 2024, indicating a significant decline[26]. Clinical Studies and Results - The registrational cohort of the EMBOLD study is on track for topline results no later than the first half of 2026[2]. - Topline results for vormatrigine from the RADIANT study are expected by mid-2025, with additional results from the POWER1 study anticipated in the second half of 2025[2]. - The EMPOWER registrational study has over 3,000 patients registered to date, supporting recruitment for the RADIANT and POWER1 studies[5]. - The Phase 1 food effect study for vormatrigine demonstrated that food intake does not significantly impact its absorption, enhancing dosing flexibility[3]. - The Essential3 study for ulixacaltamide is expected to complete topline readout in Q3 2025, despite an interim analysis recommending study stoppage for futility[5]. Other Income - Other income increased from $2,333,000 in Q1 2024 to $5,432,000 in Q1 2025, reflecting an increase of approximately 133.5%[26].

On track for six major study readouts across four programs over the next 12 months Ready to initiate pivotal studies in two developmental and epileptic encephalopathy (DEE) programs in mid-year 2025: EMERALD for broad DEEs with relutrigine and EMBRAVE3 for SCN2A GoF with elsunersen Vormatrigine continues to generate a best-in-class safety profile with new data demonstrating no food effect and higher dosing tolerability Praxis to host a virtual investor event on Friday, May 2, 2025 to discuss its DEE portfol ...

BOSTON, April 08, 2025 (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 be hosting a virtual investor event on Friday, May 2, 2025 from 10:00 a.m. to 11:30 a.m. EDT. The webinar, hosted by the Praxis management team, will focus on the company’s clinical p ...

BOSTON, April 03, 2025 (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 be participating in two upcoming investor conferences this April: Praxis management will be presenting a corporate overview at the 24th Annual Needham Virtual Healthcare Conference, ta ...