Praxis study halted for futility, other programs advance

Published 28/02/2025, 14:06
Praxis study halted for futility, other programs advance

BOSTON - Praxis Precision Medicines Inc. (NASDAQ: PRAX) announced that the Independent (LON:IOG) Data Monitoring Committee (IDMC) has recommended halting Study 1 of the Essential3 program, which is evaluating ulixacaltamide for essential tremor, due to unlikely achievement of the primary efficacy endpoint. The news has contributed to a significant 12.7% decline in the stock price over the past week, according to InvestingPro data. Despite this setback, the company plans to continue the study to its completion, with final results expected in the third quarter of 2025.

The IDMC’s recommendation was based on a predefined decision framework, suggesting the statistical model’s underlying assumptions may have influenced the outcome. Praxis CEO Marcio Souza expressed disappointment but confirmed the company’s commitment to exploring alternative analysis methods for the final dataset.

In addition to the Essential3 program, Praxis provided updates on its other development programs. The company anticipates topline results from the RADIANT and POWER1 studies in focal onset seizures and generalized epilepsy with vormatrigine by mid-year 2025. The EMERALD registrational study of relutrigine in developmental epilepsies and encephalopathies is expected to begin by mid-2025.

Praxis also highlighted the progress of its Cerebrum™ Small Molecule Platform and Solidus™ Antisense Oligonucleotide Platform. The company remains on track to nominate development candidates for its early-stage antisense oligonucleotide therapeutic initiatives in 2025.

Financially, Praxis reported a robust position with $469.5 million in cash, cash equivalents, and marketable securities as of December 31, 2024. InvestingPro analysis shows the company maintains a strong liquidity position with a current ratio of 10.96 and more cash than debt on its balance sheet. This is expected to fund operations into 2028. The company recognized $7.5 million in collaboration revenue for the fourth quarter of 2024, primarily related to its agreement with UCB. Eight analysts have recently revised their earnings expectations downward for the upcoming period.

Research and development expenses increased by $65.6 million for the year 2024, primarily due to expenses related to the Cerebrum™ platform. General and administrative expenses also rose by $14.2 million, mainly due to personnel-related costs and professional fees. Praxis reported a net loss of $58.7 million for the fourth quarter of 2024 and $182.8 million for the full year. With a market capitalization of $1.25 billion and an EBITDA of -$163.59 million over the last twelve months, InvestingPro analysis suggests the stock is currently trading near its Fair Value. Subscribers can access 9 additional ProTips and comprehensive financial metrics to better evaluate the company’s investment potential.

The company’s pipeline includes multiple clinical-stage product candidates across epilepsy and movement disorders. This update is based on a press release statement from Praxis Precision Medicines.

In other recent news, Praxis Precision Medicines Inc. has been the subject of multiple analyst updates and developments. Deutsche Bank (ETR:DBKGn) initiated coverage on Praxis with a Buy rating and a price target of $111, highlighting potential catalysts despite previous setbacks in clinical trials. Jefferies maintains a Buy rating with a significantly higher price target of $305, expressing optimism about upcoming milestones in 2025, including the interim analysis of the Essential3 Study 1 for essential tremor. Truist Securities also raised its price target to $175, citing increased confidence in the success of Praxis’s ulixacaltamide, with a probability of success raised from 50% to 70%.

Piper Sandler reiterated its Overweight rating with a $270 price target, emphasizing the potential of Praxis’s pipeline, including ulixacaltamide’s interim results expected in early 2025. The firm also noted the importance of other drugs in development, such as vormatrigine and relutrigine, with results anticipated in the first half of 2025. Piper Sandler identified Praxis as one of the companies poised for significant catalysts in the coming year, with expectations for de-risked Phase 3 readouts. These developments reflect growing interest and varying degrees of optimism from analysts regarding Praxis’s future prospects in the biotech sector.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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