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]

Company Insights - Praxis Precision Medicines is expected to report a quarterly loss of $2.39 per share, reflecting a year-over-year increase of 67.5%. Revenues are projected to be $1.67 million, which is an increase of 114.1% compared to the same quarter last year [1] - The stock price of Praxis Precision Medicines increased by 6.4% to close at $40.11, supported by higher trading volume, contrasting with an 11.4% loss over the previous four weeks [5] - The consensus EPS estimate for Praxis has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without earnings estimate revisions [3][6] Industry Context - Praxis Precision Medicines operates within the Zacks Medical - Biomedical and Genetics industry, where Harmony Biosciences, another company in the same sector, has seen its consensus EPS estimate change by -114.8% to -$0.04, representing a year-over-year decline of 107.1% [4][7] - Harmony Biosciences has a Zacks Rank of 3 (Hold) and experienced a 0.3% decrease in its stock price, with a return of -4.3% over the past month [4][7]

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 ...

Praxis Precision Medicines Provides Corporate Update and Reports First Quarter 2024 Financial Results Plans to initiate two efficacy studies investigating PRAX-628 in focal onset seizures in the second half of 2024 and first half of 2025, respectively Continued strong recruitment for the pivotal Essential3 trials in Essential Tremor (ET), with over 50,000 pre- screening forms and over 7,000 referrals received to date that meet pre-qualifying eligibility criteria; topline results expected in the second half ...

Praxis Precision Medicines Provides Corporate Update and Reports Fourth Quarter and Full-Year 2023 Financial Results Over 3,000 referrals received to date meet pre-qualifying eligibility criteria for ulixacaltamide Phase 3 studies in the Essential3 program for essential tremor (ET); enrollment on track to be completed in 1H 2024 with topline results in 2H 2024 Topline results from the PRAX-628 study in epilepsy patients with photo-paroxysmal response (PPR) expected in 1Q 2024; preliminary analysis of 15 mg ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from________ to_________ Commission File Number: 001-39620 PRAXIS PRECISION MEDICINES, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 47-5195942 (St ...