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BOSTON - Praxis Precision Medicines, Inc. (NASDAQ: PRAX) has announced promising topline results from its Phase 2 study of relutrigine, a potential treatment for developmental and epileptic encephalopathy (DEE). During the double-blind period of the trial, relutrigine demonstrated a placebo-adjusted 46% reduction in monthly motor seizures. Notably, over 30% of patients achieved seizure freedom while on the drug, a significant milestone for those affected by SCN2A and SCN8A DEE.
The study, known as EMBOLD, included male and female participants aged 2-18 years diagnosed with early onset SCN2A-DEE or SCN8A-DEE. The trial design was double-blind, placebo-controlled, and included a subsequent open-label extension. Out of sixteen patients randomized, fifteen were eligible for efficacy assessments. Dose adjustments were allowed, with seven patients increasing their daily dose during the study period. No patients discontinued due to adverse events, which included infections, vomiting, pyrexia, somnolence, and constipation.
Improvements were also observed in caregiver and clinician assessments of patients' alertness, communication, and seizure severity. In the long-term extension of the study, eight patients showed a median reduction in motor seizures of 75%, with five achieving a 28-day seizure-free status, compared to none on placebo.
Relutrigine, developed as a preferential inhibitor of persistent sodium current, has shown potential in addressing seizure symptoms in DEE. The drug has received Orphan Drug Designation and Rare Pediatric Designation from the FDA, and Orphan Drug Designation from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE.
The company plans to discuss the study results and next steps in a call on Tuesday morning. This news is based on a press release statement and represents a significant development in the treatment of pediatric epilepsy, offering hope to patients and families affected by these severe conditions.
In other recent news, Praxis Precision Medicines has been the subject of significant developments and analyst activity. The company's recent second-quarter earnings call highlighted their upcoming interim Essential3 Part1 analysis, expected in the fourth quarter of 2024, and the anticipated release of the relutrigine EMBOLD data in the third quarter of 2024. Praxis Precision also discussed the potential of PRAX-628's RADIANT study and the design choices for the POWER1 and POWER2 trials.
Analysts from Piper Sandler, H.C. Wainwright, and Guggenheim have maintained positive ratings on Praxis Precision shares, with Piper Sandler reaffirming its Overweight rating and a price target of $270.00, H.C. Wainwright raising its price target to $120, and Guggenheim maintaining a Buy rating and increasing the price target to $170.
Oppenheimer initiated coverage on Praxis Precision with an Outperform rating, citing the potential of the company's central nervous system drugs, particularly ulixacaltamide for essential tremor and PRAX-628 for focal onset seizures. Jefferies raised its stock target for Praxis from $128 to $141, maintaining a 'Buy' rating, fueled by the potential outcomes of the EMBOLD study involving PRAX-562. Baird also initiated coverage on Praxis, assigning an 'Outperform' rating. These recent developments reflect the ongoing interest in Praxis Precision Medicines from a variety of analyst firms.
InvestingPro Insights
Praxis Precision Medicines (NASDAQ: PRAX) has shown positive clinical progress, which could be a catalyst for the company's stock. The promising results from the Phase 2 study of relutrigine are a testament to the company's potential in developing treatments for complex neurological disorders. However, it's important for investors to consider the financial health and market performance of the company as well.
InvestingPro Data indicates that Praxis Precision Medicines has a market capitalization of 943.79 million USD, which reflects investor confidence to a degree. Despite not being profitable over the last twelve months, with a negative P/E ratio of -5.46, the company holds more cash than debt on its balance sheet, which is a positive sign of financial stability. Additionally, the company's stock price has experienced a high return over the last year, with a year-to-date price total return of 138.55%, suggesting strong investor interest.
InvestingPro Tips highlight that analysts have revised their earnings upwards for the upcoming period, indicating optimism about the company's future performance. However, it is also noted that analysts anticipate a sales decline in the current year. This juxtaposition of analyst expectations suggests that while the short-term outlook may have positive aspects, potential challenges lie ahead.
Investors looking for more insights can find additional InvestingPro Tips for Praxis Precision Medicines, which could help in making more informed decisions. Currently, there are 10 additional tips listed on InvestingPro for PRAX, available at https://www.investing.com/pro/PRAX. These tips could provide a deeper understanding of the company's financial health, market performance, and future prospects.
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