Marinus sinks after seizure treatment fails to meet high expectations

2024-04-15

临床3期上市批准优先审批

Shares of Marinus Pharmaceuticals plummeted over 80% on Monday after the company disclosed that an interim analysis of its seizure drug ganaxolone failed to meet early stopping criteria.

The Phase III RAISE trial is evaluating an IV version of ganaxolone in 100 patients with refractory status epilepticus (RSE). An oral formulation of the GABA receptor modulator was approved by the FDA in 2022 as Ztalmy to treat seizures associated with CDKL5 deficiency disorder CDKL5 deficiency disorder.

Ahead of the announcement, analysts from both Jefferies and H.C Wainwright were bullishly optimistic that the interim data would show 60%-70% response rates for ganaxolone.

“With the lack of other informative studies for RSE, RAISE has the potential to be a landmark study to establish ganaxolone as the standard of care,” H.C. Wainwright analyst Douglas Tsao wrote earlier this month, projecting peak sales of $600 million.

Investors will now have to wait until the summer – when topline results are expected to be released – to see if ganaxolone met or missed their high expectations.

“While we are disappointed that RAISE did not meet the early stopping criteria, we will only be able to determine the trial’s outcome once we unblind and analyse the full data set,” said chairman and chief executive Scott Braunstein.

Trial delays and cash runway

It’s not the first delay the RAISE trial has faced. In 2022, Marinus had to temporarily pause the study because of an "interruption of clinical supply material" caused by the Omicron wave.

However, Marinus may have been counting on an early trial stop as it nears the end of its cash runway – its current $113.3 million on hand is expected to fund operations only through the fourth quarter. The firm said Monday it will undergo cost reduction activities to extend that runway into 2025.

Meanwhile, Marinus said it is on track to complete enrollment in May for the Phase III TrustTSC trial, which is assessing an oral formulation of ganaxolone to treat tuberous sclerosis. If topline data, due in the fourth quarter, are positive, Marinus expects to submit the drug for FDA approval in the first half of 2025 with a request for priority review.

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