Praxis Precision Medicines reports positive results in epilepsy study By

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BOSTON – Praxis Precision Medicines Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company, announced positive findings from its Phase 2a proof of concept study for PRAX-628, a treatment for epilepsy patients with photoparoxysmal response (PPR).

The study showed a complete response rate of 100% in the 45 mg cohort and an 80% complete response rate in the 15 mg cohort, with the remaining 20% achieving a partial response.

The PPR study is designed to measure electroencephalogram (EEG) signatures after photic stimulation, which serves as an indicator of anti-seizure efficacy. The study’s results demonstrated strong and consistent responses across both dosing cohorts, with a safety profile in line with previous studies. Pharmacokinetic analysis confirmed the expected therapeutic exposures of the drug.

The company has indicated that these results will inform the planning of an efficacy study for focal onset epilepsy, which is slated to begin in the second half of 2024. Praxis extended gratitude to the patients who participated in the PPR study.

In the study, patient EEG signatures were assessed over a 24-hour period following administration of either placebo or PRAX-628. The response was classified as complete if there was a reduction to zero in the number of generalized PPR events at any assessment period versus baseline. A partial response was noted if there was a reduction, but not to zero, in the number of generalized PPR events.

The safety and pharmacokinetic (PK) profiles of PRAX-628 were monitored throughout the observation period. The drug, which is being developed as a once-daily oral treatment for adult focal onset epilepsy, has shown promising results in preclinical studies and was well-tolerated in a Phase 1 dose escalation study in healthy volunteers.

PRAX-628 is characterized as a precision sodium channel modulator, aiming to target the hyperexcitable state of sodium channels in the brain. In vitro studies have demonstrated its selectivity for disease-state NaV channel hyperexcitability, and in vivo studies have shown significant potency in a model predictive of efficacy in focal epilepsy.

Praxis will host a call to discuss the study results, featuring Dr. Daniel Friedman from NYU Grossman School of Medicine. These findings are based on the company’s press release statement and are part of Praxis’s ongoing efforts to develop therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance.

InvestingPro Insights

Praxis Precision Medicines (NASDAQ: PRAX) has recently released promising results from its Phase 2a study for PRAX-628, which could have significant implications for the company’s financial health and stock performance. According to real-time data from InvestingPro, Praxis has a market capitalization of $668.54 million, indicating a substantial investor interest in its potential growth in the biopharmaceutical sector.

Despite the company’s exciting clinical advancements, it’s important to note that Praxis is not currently profitable, with a negative P/E ratio of -2.73 and an adjusted P/E ratio for the last twelve months as of Q4 2023 at -5.42, reflecting the high-risk nature of investing in clinical-stage biopharma companies.

InvestingPro Tips reveal that Praxis holds more cash than debt on its balance sheet, which may provide some financial flexibility as it continues to fund its research and development activities. However, the company is quickly burning through cash, which is a critical factor for investors to consider. Additionally, analysts have revised their earnings upwards for the upcoming period, potentially indicating a positive outlook on the company’s future earnings potential.

InvestingPro also highlights that Praxis’s gross profit margin is deeply negative at -3445.81% for the last twelve months as of Q4 2023, underscoring the challenges the company faces in reaching profitability. Despite this, the stock has experienced a high return over the last year, with a 312.03% price total return, showcasing the market’s optimism about its long-term prospects. For investors intrigued by Praxis’s potential and seeking further insights, there are 15 additional InvestingPro Tips available at: To access these insights, consider using the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.

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