One innovation area for mental health

Aug 13, 2024

With help from Erin Schumaker

Driving The Day

Bristol Myers Squibb's pharmaceutical plant of French group UPSA is pictured.

Bristol Myers Squibb has developed an antipsychotic drug therapy for schizophrenia, which will be up for FDA review next month. | Georges Gobet/AFP via Getty Images

THE FUTURE OF MENTAL HEALTH DRUGS — Psychiatrists and some drug companies are bullish on novel drugs for intractable mental health conditions, like post-traumatic stress disorder and schizophrenia, even as the FDA denied one firm’s bid to pair psychedelic drug MDMA with talk therapy as a PTSD treatment.

The agency is slated to decide by Sept. 26 whether to approve Bristol Myers Squibb’s KarXT, which could be the first new antipsychotic in decades to treat schizophrenia. Another biotech is waiting in the wings to bring a longer-acting version of the drug to market by the end of the decade.

“I would describe this moment as a mini-boomlet in drug discovery because it’s been quiet for a really long time in terms of novel treatments,” Dr. Ken Duckworth, chief medical officer for the National Alliance on Mental Illness, said.

The novel candidate: KarXT is thought to improve a range of schizophrenia symptoms. Most antipsychotics on the market address only “positive” symptoms, Duckworth said, or symptoms that patients shouldn’t experience, like auditory hallucinations. They’re also linked to side effects like weight gain and involuntary body tics.

KarXT is expected to also treat the disease’s “negative” symptoms — social withdrawal, lack of motivation — or the absence of things that a patient should be able to do, he said.

The gold-standard drug today for treatment-resistant schizophrenia is clozapine, a medication first trialed in the 1960s. Many patients partially respond to the therapy, Duckworth said, so they might not hear voices as loudly but still have trouble completing tasks.

The drug is also subject to the FDA’s risk-mitigation protocol because it can cause abnormally low white blood cell count, with patients required to take blood tests before using it. However, the agency is considering changes to the program, with an advisory committee meeting scheduled for Nov. 19.

The “prodrug” approach: Terran Biosciences is gearing up to innovate on KarXT, which patients would take twice daily if approved, by developing a once-daily pill and a long-acting injection.

Terran is targeting a so-called prodrug — an inactive compound that metabolizes in the body and becomes an active drug — dubbed TerXT to compete with KarXT. The company got its prodrug patented earlier this year.

CEO Dr. Sam Clark said he expects the compound to be eligible for an accelerated FDA approval pathway that could see a decision in five years — just as BMS’ marketing exclusivity for KarXT ends.

IT’S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. So much for a quiet August! Are you planning to attend Thursday’s drug pricing event (more on that below)?

Let us know and send tips to David Lim (dlim@politico.com or @davidalim) and Lauren Gardner (lgardner@politico.com or @Gardner_LM).

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Industry Intel

F Psilocybin mushrooms stand ready for harvest in a humidified "fruiting chamber"

Compass Pathways has developed a therapy for treatment-resistant depression combining psychological support with psilocybin, also known as magic mushrooms. | John Moore/Getty Images

PSYCHEDELICS HORSE RACE — British biotech Compass Pathways is gaining ground in the quest to secure FDA approval for psychedelic medicine thanks to back-to-back losses for rival Lykos Therapeutics, Erin writes.

As part of its rejection of MDMA on Friday, the FDA told Lykos it must conduct an additional late-phase trial of the regimen's safety and efficacy for its application to be reconsidered.

Next up: Compass Pathways has a different psychedelic treatment in Phase III trials. Its treatment-resistant depression regimen combines psilocybin, or psychedelic mushrooms, with psychological support. Like Lykos, the FDA gave Compass Pathways' treatment breakthrough therapy status, a label meant to expedite its path toward approval.

“Robust monitoring and rigorous standards are at the core of our programs at Compass and we believe that generating clear and compelling clinical evidence is the best way to bring new medicines to patients as quickly as possible,” Chris Williams, Compass’ chief communications officer, said in a statement.

Psychedelics advocates have criticized Compass for developing synthetic psilocybin and patenting it, a business-savvy move they view as counter to the ethos of the psychedelics movement.

Big picture: Given interest in psychedelics treatments for mental illness and significant need — in 2021, 14.5 million American adults had at least one major depressive episode and 13 million people had PTSD, according to federal data — Lykos’ FDA struggles might prove instructive, instead of discouraging, to its rivals.

Related retraction: The journal Psychopharmacology announced over the weekend that it’s retracting studies of MDMA-assisted therapy by Lykos’ nonprofit arm due to “protocol violations amounting to unethical conduct” at one of the study sites.

“Lykos disagrees with this retraction and believes the identified issues should have been addressed through corrections,” the company said in a statement.

Drug Pricing

DRUG PRICE INTEL COMING — Mark your calendars: The White House is expected to divulge details from the inaugural Medicare drug price negotiations Thursday, Lauren and POLITICO’s Adam Cancryn scooped.

Administration officials are expected to describe the results of the talks covering 10 prescription drugs available under Medicare Part D, according to three people with knowledge of the planning who were granted anonymity to disclose private conversations.

It’s unclear whether that means the final prices will be announced — CMS and the nine drugmakers involved wrapped up the discussions earlier this month — or the Biden administration will quantify in some other way the savings patients can expect beginning in 2026. The move comes one day before the second anniversary of the Inflation Reduction Act, which created the program.

CMS must publish the final prices by Sept. 1, but it has until March to explain how it arrived at those numbers.

Legal lookout: Most of the lawsuits challenging the program at the federal district court level have been dismissed, including industry association attempts to draw opinions from arguably favorable jurisdictions like Ohio and Texas.

But most if not all have been or will be taken to the appellate court level — the next stop on the path toward the Supreme Court, where most observers expect at least some cases to end up.

Eye on the FDA

ASCENDIS GETS APPROVAL — The FDA approved a new medicine Monday for a rare hormone disorder known as hypoparathyroidism that affects around 70,000 to 90,000 people in the U.S.

The new drug, made by Danish drugmaker Ascendis Pharma, helps treat a lack of the parathyroid hormone. But it will not be available immediately; the company says manufacturing plans for the U.S. mean that initial supply is expected by early next year.

If the FDA grants the company permission to commercialize its existing product, Ascendis said the drug could be introduced in the fourth quarter.

Pharma Moves

Mary Moody Johnson is now a senior vice president at SL Strategies. She previously worked as a health policy director for Senate HELP ranking member Bill Cassidy (R-La.).

WHAT WE'RE READING

Light alcohol consumption was associated with an increase in cancer deaths in older adults in the United Kingdom, The New York Times' Roni Caryn Rabin reports.

Researchers are optimistic about the potential of GLP-1s to prevent common cancers exacerbated by obesity, NPR’s Yuki Noguchi reports.

Johnson & Johnson has more than 75 percent support from cancer claimants for a $6.5 billion settlement plan, Bloomberg’s Jef Feeley and Damian Garde report.

Document Drawer

The FDA announced it will renew its Peripheral and Central Nervous System Drugs Advisory Committee for two years.

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