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Intra-Cellular approval keeps focus on FDA's neuroscience review process

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Dive Brief:

  • The Food and Drug Administration on Monday approved Intra-Cellular's lumateperone as a treatment for adults with schizophrenia, a decision that supports speculation the agency is taking a more flexible approach when evaluating neuroscience medicines.

  • Lumateperone, which will be sold as Caplyta, showed conflicting results in late-stage testing. One study found patients given a higher dose of the drug as opposed to placebo had significantly improved scores on a scale measuring schizophrenia severity. And yet, both a higher and a lower dose of Intra-Cellular's drug failed to outperform placebo in another Phase 3 study.

  • In a note to investors, Stifel analyst Paul Matteis wrote that the Caplyta approval affirms how the FDA sees mixed clinical data for central nervous system drugs with a "glass half full lens." That could have positive implications for other neuroscience drug developers, he added, including Sage Therapeutics, Acadia Pharmaceuticals, and Karuna Therapeutics.

Dive Insight:

Historically, drugs are most likely to secure FDA approval when backed by at least two positive studies that enrolled relatively large numbers of patients. But that's not always the case, as demonstrated by a couple of neuroscience-related therapies cleared for the market in the last year.

In March, Johnson & Johnson won approval for its depression treatment Spravato (eskematine), despite the drug having support from just one short-term Phase 3 trial and an accompanying withdrawal study.

The clinical package for Sarepta Therapeutics' Vyondys 53 (golodirsen) also raised some eyebrows, as it was unclear whether the small biomarker benefit was seen in treated patients actually translates into functional gains.

The Spravato, Vyondys 53 and now Caplyta approvals raise questions about how the FDA evaluates neuroscience medicines and whether that process is more nuanced than other areas of drug development. Notably, clinical trials for brain and central nervous system treatments often use subjective endpoints, which can make them more difficult to interpret.

According to Matteis, the Caplyta decision may bode well for a Sage drug that recently failed a late-stage study in major depressive disorder. While the study missed its primary endpoint, Matteis wrote that "there is still a shot" regulators view it as supportive to approval based on positive data seen at multiple time points.

He also wrote that, while the FDA could be more flexible with Acadia's Nuplazid (pimavanserin) and Karuna's KarXT, the same can't be said for aducanumab, Biogen's closely watched treatment for Alzheimer's disease. Not only did aducanumab fail one late-stage trial, but the one in which it succeeded — the one in which Biogen hangs its approval hopes — paints a hazy picture of whether the drug's benefits outweigh its risks.

To that point, the FDA's flexibility has limits. In February, the agency rejected a depression drug from Alkermes backed by shaky evidence of clinical efficacy. 

With approval in hand, Intra-Cellular intends to start marketing Caplyta late in the first quarter of 2020. The biotech hasn't issued a list price for the drug. A company spokesperson wrote in an email to BioPharma Dive that pricing information will be disclosed closer to the launch, but couldn't provide additional details.

RBC Capital Markets estimates that assuming U.S. net pricing of about $900 per month, stateside Caplyta sales will be $60 million next year and greater than $400 million by 2024.

Intra-Cellular shares were trading a little over $41 apiece late Monday morning, up 230% from Friday's market close.

Correction: A previous version of this article incorrectly stated Spravato was approved in February. The drug was approved in March.

Written by: Jacob Bell

Published on: Dec. 23, 2019

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