Core Viewpoint - Praxis Precision Medicines has experienced significant stock performance improvements due to positive clinical trial results and analyst upgrades, indicating strong investor confidence in the company's future prospects [1][5]. Stock Performance - PRAX's stock has increased by 520% over the past three months, primarily driven by successful late-stage studies of ulixacaltamide for essential tremor treatment [2][5]. - The current stock price is $264.06, reflecting a slight decrease of 2.55% or $6.92 from previous levels [3][5]. - The stock has shown considerable volatility over the past year, with a high of $278.44 and a low of $26.70 [3]. Analyst Ratings - Jefferies upgraded PRAX's stock to a "Buy" rating, raising the price target from $300 to $450 [1][5]. Market Capitalization and Trading Volume - Praxis Precision Medicines has a market capitalization of approximately $5.58 billion, with a trading volume of 238,597 shares [4][5]. Clinical Development - The company's progress on pre-New Drug Application steps and positive data for relutrigine have contributed to the recent stock rally, with a regulatory filing anticipated in early 2026 [4].

Key Takeaways PRAX surged on pivotal Essential3 results showing ulixacaltamide met all key endpoints in essential tremor.Efficacy appeared early and was sustained, with Study 2 confirming a maintained response versus placebo.Progress on pre-NDA steps and positive relutrigine data added further fuel to PRAX's recent stock rally.Shares of Praxis Precision Medicines (PRAX) have skyrocketed 519.5% in the past three months. The sharp rally in the stock was largely fueled by encouraging top-line data from two piv ...

Investors with a lot of money to spend have taken a bearish stance on Praxis Precision Medicine (NASDAQ:PRAX).And retail traders should know.We noticed this today when the trades showed up on publicly available options history that we track here at Benzinga.Whether these are institutions or just wealthy individuals, we don't know. But when something this big happens with PRAX, it often means somebody knows something is about to happen.So how do we know what these investors just did? Today, Benzinga's option ...

Praxis Precision Medicines Inc. (NASDAQ:PRAX) shares surged 24.81% in pre-market trading on Friday, climbing to $237.10.Check out the current price of PRAX stock here.PRAX closed Thursday's regular session at $189.97, up 2.05%, according to Benzinga Pro data.Early Trial Halt For EfficacyThe Boston-based clinical-stage biopharmaceutical company reported positive results from the registrational cohort of its EMBOLD study, which is evaluating relutrigine for SCN2A and SCN8A developmental and epileptic encephal ...

Core Viewpoint - A short seller is challenging Praxis Precision Medicines Inc. regarding the validity of its Phase 3 trial results for ulixacaltamide, suggesting that the data may not withstand regulatory scrutiny [1][2]. Group 1: Trial Results and Claims - Praxis reported a statistically significant 4.3 point mean improvement in the mADL11 score at Week 8 of the Phase 3 Essential3 program for essential tremor, with all key secondary endpoints achieving statistical significance [3]. - The short seller claims that Praxis manipulated the trial data by changing the primary endpoint without FDA approval and using optimistic assumptions for patients who discontinued the trial [5][6]. Group 2: Regulatory and Approval Concerns - The short seller argues that the issues raised could jeopardize ulixacaltamide's approval prospects ahead of a key FDA meeting later this year [4][7]. - The report suggests that the FDA will recognize the problems with the trial data, leading to significant downside risk for the stock [7]. Group 3: Valuation and Market Concerns - Nearly all of Praxis's market value is tied to ulixacaltamide, which was acquired for only $1 million in 2018, raising concerns about the drug's potential [8]. - The short seller describes ulixacaltamide as having "virtually no path forward" and labels the expectations surrounding it as resting on an unstable foundation [6][12]. - Following the release of the "positive" Phase 3 results, Praxis raised $567 million in new capital, with analysts valuing ulixacaltamide near $3 billion, constituting the majority of Praxis's market capitalization of approximately $4.5 billion [11].

Summary of Praxis Precision Medicines FY Conference Call Company Overview - **Company**: Praxis Precision Medicines (NasdaqGS:PRAX) - **Date of Conference**: November 10, 2025 - **Key Executives Present**: CEO Marcio Souza, SVP Finance Lauren, Matt Krogo Key Points Discussed Pipeline and Upcoming Developments - The company is preparing for a potential New Drug Application (NDA) filing in the next few months, particularly for essential tremors (ET) and ulixacaltamide [6][10] - There is a focus on the Essential 3 program, which has shown positive results, and the company is looking to leverage these results for future drug applications [7][8] - The company is moving forward with the SCN2A EMBOLD program, with plans for an NDA submission early next year [9][10] FDA Interactions - The upcoming FDA meeting is crucial for discussing the NDA submission and the adequacy of the evidence from the Essential 3 program [12][15] - The company has had multiple interactions with the FDA, which have been positive, indicating good continuity in the review process [11][20] - The key question for the FDA meeting is whether the current data package constitutes substantial evidence of effectiveness for approval [15][16] Market Opportunity - There are approximately 7 million Americans living with essential tremor, with at least 2 million actively seeking treatment, indicating a significant market potential [37][38] - The company estimates that with conservative market penetration, potential sales could reach $10 billion in the U.S. alone, and $13 billion to $15 billion when including ex-U.S. markets [39][40] - The pricing strategy is expected to be competitive, with a potential 25%-50% discount compared to existing treatments [38][39] Commercial Strategy - The company is building infrastructure to better understand and recruit patients, aiming to expand its database from 200,000 to about 1 million patients before launch [41][42] - The commercial organization is being strengthened, leveraging the experience of executives who have previously launched multiple drugs [42][43] Other Assets and Future Plans - The company is also focusing on relutrigine, which has received breakthrough designation, and is preparing for an interim analysis that could lead to an NDA submission [44][46] - The market for SCN2A and 8A is projected to be significant, with estimates of over $1 billion at launch [50][51] - Upcoming data from the RADIANT study is expected to provide insights into the effectiveness of relutrigine in generalized patients [54][55] Additional Insights - The company emphasizes the importance of FDA's role in ensuring drug safety and efficacy, and it aims to provide substantial evidence to support its applications [20][21] - There is a recognition of the need to overcome biases in market assumptions to accurately reflect the potential of their treatments [37][39] This summary captures the essential discussions and strategic directions of Praxis Precision Medicines as presented in the conference call.

Core Insights - Praxis Precision Medicines announced positive results from two pivotal Phase 3 Essential3 studies of ulixacaltamide HCl in essential tremor, with a pre-NDA meeting with the FDA scheduled for Q4 2025 [1][2] - The company plans to accelerate the development of relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies (DEE) after a comprehensive Type B meeting with the FDA [1][2] - Recruitment for the POWER1 study of vormatrigine in focal onset seizures has been completed, with topline results expected in the first half of 2026, while the POWER2 study has commenced [1][2] Clinical Development Updates - Ulixacaltamide demonstrated significant improvements in symptoms for essential tremor, with a mean improvement of 4.3 points in the modified Activities of Daily Living 11 (mADL11) at Week 8 [3][4] - The first study met all primary and secondary endpoints, while the second study showed 55% of patients on ulixacaltamide maintained their response compared to 33% on placebo [3][4] - Vormatrigine showed a 56.3% median reduction in seizure frequency in the RADIANT study, with 22% of patients achieving a 100% reduction in the last 28 days [4][15] Financial Overview - As of September 30, 2025, Praxis had $389.2 million in cash and marketable securities, down from $469.5 million at the end of 2024, primarily due to cash used in operating activities [6][7] - The company completed a public offering in October 2025, generating net proceeds of approximately $567 million, expected to fund operations into 2028 [7][8] - Research and development expenses increased to $65.8 million for Q3 2025, up from $41.9 million in Q3 2024, largely due to investments in the Cerebrum™ platform [9][10] Market Position and Future Prospects - Praxis is positioned to establish itself as a leader in innovative therapies for CNS disorders, with multiple clinical-stage product candidates across movement disorders and epilepsy [2][18] - The company is on track to nominate development candidates for early-stage antisense oligonucleotide (ASO) therapeutic initiatives in the first half of 2026 [13][18] - The EMBOLD study for relutrigine is expected to provide substantial evidence for NDA submission in early 2026, contingent on a successful interim analysis [4][16]

Core Insights - Praxis Precision Medicines, Inc. (NASDAQ:PRAX) has been identified as one of the 12 best-performing stocks over the last three months, with Oppenheimer raising its price target from $115 to $250 while maintaining an Outperform rating [1][2]. Group 1: Clinical Development - The positive topline results from the Essential3 trial of ulixacaltamide for essential tremor (ET) have contributed to the stock's performance, as there is currently no approved treatment for this condition [2]. - The drug met all endpoints in Study 1 and its pre-specified primary endpoint in Study 2, demonstrating general safety and tolerability without serious drug-related side effects [3]. Group 2: Future Prospects - Oppenheimer anticipates that Praxis Precision Medicines, Inc. will submit a New Drug Application in early 2026, with a potential commercial launch of ulixacaltamide in 2027 following FDA approval [3]. - The company is focused on developing therapies for central nervous system disorders, indicating a strategic emphasis on addressing unmet medical needs in this area [4].

Core Insights - Praxis Precision Medicines experienced a significant stock surge of 251% following positive phase 3 trial results for ulixacaltamide, a treatment for essential tremor patients [2][3] - The essential tremor condition affects approximately 10 million Americans, with only one approved treatment currently available, indicating a substantial market opportunity for new therapies [5] - Ulixacaltamide has shown promising results in clinical trials, but the company is still in the pre-commercial stage, which may pose risks for investors [3][15] Company Overview - Praxis Precision Medicines is focused on developing treatments for neurological disorders, with ulixacaltamide targeting essential tremor and vormatrigine aimed at epilepsy [13][14] - The company has a market capitalization of approximately $4 billion, reflecting investor interest in its late-stage drug candidates [12][16] - Praxis recently raised $525 million in a secondary offering, bolstering its cash reserves to support ongoing development efforts [15] Clinical Trial Results - In the first phase 3 study, patients treated with ulixacaltamide showed an average improvement of 4.3 points on a modified Activities of Daily Living test, compared to a 1.7-point improvement in the placebo group [7] - The second study indicated that patients who continued treatment with ulixacaltamide had a significantly higher likelihood of maintaining a response and reported improved disease outcomes [8] - Despite the positive results, a notable discontinuation rate of 35.6% in the first study and 38.1% in the second study raises concerns about treatment tolerability [10][11] Market Potential - The potential annual sales for ulixacaltamide could reach billions, given the large population of underserved essential tremor patients [3] - Up to 77% of essential tremor patients feel inadequately treated, and nearly 50% are not receiving any treatment, highlighting a significant unmet medical need [6] Future Prospects - Praxis is also advancing other drug candidates, including vormatrigine for epilepsy, which has shown promising phase 2 results with a 60% reduction in seizure rates for treated patients [13] - The company is preparing to enter phase 3 trials for vormatrigine, indicating a robust pipeline of potential therapies [14] - Investors with a high-risk tolerance may find Praxis an attractive opportunity due to its focus on underserved populations and multiple late-stage candidates [16]

Core Insights - Praxis Precision Medicines, Inc. (PRAX) shares experienced a significant rally of 183.7%, closing at $162.71, attributed to higher trading volume compared to typical sessions [1] - The stock has gained 30.3% over the past four weeks, indicating strong investor interest [1] Study Results - Positive topline results from two phase III studies of ulixacaltamide for treating essential tremor (ET) were announced, with Study 1 showing a 4.3-point mean improvement in daily functioning at Week 8 compared to placebo, meeting the primary endpoint [2] - Study 2 revealed that 55% of ulixacaltamide-treated patients maintained response compared to 33% on placebo, confirming sustained benefit and meeting the primary endpoint [2] - The drug was well tolerated, with no serious drug-related adverse events reported, positioning ulixacaltamide as a potential first-in-class treatment for ET, which affects around 7 million Americans [2] Regulatory Outlook - Praxis has submitted a pre-NDA meeting request to the FDA, indicating a path toward commercialization, with plans to submit a regulatory application for ulixacaltamide in early 2026 [2] Financial Expectations - The company is expected to report a quarterly loss of $3.45 per share, reflecting a year-over-year change of -25.5%, while revenues are anticipated to be $0.33 million, up 10.7% from the previous year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, suggesting that stock price movements may not continue without trends in earnings estimate revisions [4] Industry Context - Praxis Precision Medicines is part of the Zacks Medical - Biomedical and Genetics industry, where Royalty Pharma (RPRX) also operates, finishing the last trading session 0.4% higher at $36.34 [4] - Royalty Pharma's consensus EPS estimate for the upcoming report has changed by +0.8% over the past month to $1.11, representing a year-over-year change of +6.7% [5]