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Minerva schizophrenia drug rejected by FDA

Dive Brief:

  • The Food and Drug Administration has rejected an experimental schizophrenia drug from Minerva Neurosciences, denying the biotechnology company’s attempt to secure approval over the regulator’s objections.
  • In a Tuesday statement, Minerva said the FDA found clinical trial evidence for the drug to be insufficient, and cited other shortcomings in the company’s application. The agency indicated Minerva’s submission lacked enough data on the drug’s safety over one year of treatment, for example.
  • Minerva plans to request a follow-up meeting with the FDA to see if it can address the regulator’s concerns. Currently, the agency is asking Minerva to obtain positive results from another “well-controlled” clinical trial.

Dive Insight:

The FDA’s decision sets back many years of effort by Minerva to secure approval of its drug for schizophrenia, called roluperidone and designed to treat so-called negative symptoms of the condition.

The company, which licensed rights to roluperidone from Mitsubishi Tanabe nearly two decades ago, advanced the drug into Phase 3 testing in late 2017 after promising mid-stage study results.

However, an initial phase of that study turned up negative data, prompting skepticism from FDA officials that Minerva had enough evidence to seek approval. The company pressed on, studying roluperidone in an open-label extension and submitting an application to the FDA in August 2022.

The FDA refused to accept Minerva’s filing, but in 2023, the biotech successfully appealed via a process designed to resolve disputes between drug sponsors and the regulator.

Tuesday’s decision indicates the FDA’s view of roluperidone hasn’t changed. Minerva outlined four main objections raised by the agency, most notably its determination that the positive Phase 2 study was “insufficient on its own to establish substantial evidence of effectiveness.”

The FDA also cited a lack of data on study participants’ use of other antipsychotic drugs alongside roluperidone, and proof that the effects associated with treatment would be clinically meaningful. Finally, agency reviewers indicated Minerva needed to submit data on a greater number of people exposed to roluperidone for at least one year.

The company also said the FDA “provided comments on, among other items, clinical pharmacology, product quality, biopharmaceutics, and nonclinical issues.”

Shares in Minerva, which were worth $6.80 apiece Monday, fell in value by about 60% during Tuesday morning trading. The company is now valued at less than $20 million, below the roughly $41 million in cash Minerva reported it had on hand at the end of December.

“[W]e will review FDA’s feedback and consider our potential paths forward, including continuing to work closely with the FDA and providing any additional information as needed,” said Minerva CEO Remy Luthringer, in the company’s statement.

Schizophrenia has been a difficult disease for drugmakers to crack. Recently, though, a new type of medicine known as muscarinic receptor agonists has drawn attention and investment. Bristol Myers Squibb’s and AbbVie’s respective acquisitions of Karuna Therapeutics and Cerevel Therapeutics were motivated in large part by those two companies’ experimental muscarinic drugs.

Acadia Pharmaceuticals, meanwhile, is studying its approved drug Nuplazid for use treating negative symptoms of schizophrenia.

In contrast to symptoms like hallucinations or delusions, negative symptoms describe the withdrawal and emotional suppression that some people with schizophrenia experience. Negative symptoms are considered harder to treat with existing antipsychotics.