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BOSTON - Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company with a market capitalization of $820 million, announced significant progress in the development of treatments for developmental and epileptic encephalopathies (DEEs), a group of disorders marked by an imbalance in neuronal activity. According to InvestingPro data, the company maintains a strong financial position with a current ratio of 8.44, indicating robust liquidity to fund its development programs. During a virtual investor event on Friday, the company shared promising data on its clinical programs, including its leading candidate relutrigine.
The EMBOLD study’s initial patient cohort revealed that after 11 months of relutrigine treatment, participants experienced a mean seizure reduction of approximately 90% from baseline. The mean longest seizure-free period extended to 67 days, a considerable improvement from the 3 days recorded at baseline.
Praxis is also advancing the EMBRAVE3 trial design for its antisense oligonucleotide (ASO) therapy, elsunersen, targeting SCN2A gain-of-function mutations. The innovative trial design is notable for its brevity compared to other ASO studies and its focus on early intervention, with treatment potentially starting at birth.
The company’s ASO portfolio continues to evolve, with PRAX-100, aimed at mono-genetic autism, expected to reach development candidate status by mid-year. Praxis is also on track to nominate development candidates for PRAX-080 and PRAX-090, targeting different genetic mutations, by the end of 2025.
Relutrigine, a small molecule preferentially inhibiting persistent sodium current, has shown efficacy across various DEE models and has been well-tolerated in clinical studies. The FDA has granted Orphan Drug and Rare Pediatric Disease Designations for relutrigine for multiple DEEs.
Elsunersen, developed in collaboration with Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), and RogCon, Inc., has also demonstrated significant seizure reduction in SCN2A-DEE patients and boasts FDA Orphan Drug and Rare Pediatric Disease Designations, as well as EMA Orphan Drug and PRIME designations.
With a target U.S. market potential of at least $3 billion for relutrigine and a global market potential of $1 billion for elsunersen, Praxis estimates substantial commercial opportunities. The company’s comprehensive approach to DEE treatment, which includes potential combination therapies, underlines its commitment to addressing these challenging conditions. InvestingPro analysis shows the stock has gained over 10% in the past week, though investors should note its high volatility with a beta of 2.68. Get access to 10+ additional exclusive ProTips and comprehensive financial metrics with an InvestingPro subscription.
This update is based on a press release statement from Praxis Precision Medicines. While the company holds more cash than debt on its balance sheet, InvestingPro data indicates it’s not yet profitable, with analysts anticipating continued losses this year. Discover detailed financial analysis and Fair Value estimates by visiting InvestingPro.
In other recent news, Praxis Precision Medicines reported a net loss of $58.7 million for the fourth quarter of 2024, with a total net loss of $182.8 million for the year. The company also recorded $7.5 million in collaboration revenue for the same quarter. Praxis has $469.5 million in cash, cash equivalents, and marketable securities as of December 31, 2024, expected to fund operations into 2028. The Independent Data Monitoring Committee recommended halting the Essential3 program for futility, but Praxis plans to continue the study to its completion, with results anticipated in the third quarter of 2025. Despite this setback, the company is advancing other programs, including the EMERALD study for developmental epilepsies set to begin by mid-2025. TD Cowen and Truist Securities both adjusted their price targets for Praxis, with TD Cowen lowering it to $61 and Truist to $85, while maintaining a Buy rating. The analysts noted the importance of upcoming data releases for Praxis’ epilepsy treatments, which could be significant for the company’s future. Additionally, Praxis granted stock options and restricted stock units to six new non-executive employees as part of its 2024 Inducement Plan.
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