Plus: A billionaire fighting brain disease |
In this week’s edition of InnovationRx, we look at the Haitian immigrant who built a $6 billion company treating brain diseases, the drugs RFK Jr. may target after Tylenol, Northwell’s new CEO, and more. (Did someone forward this to you? To get it in your inbox, subscribe here.)
On Tuesday, President Trump announced a new direct-to-consumer website called TrumpRx that would be operated by the federal government and offer drugs at discounted prices. Pfizer was the first pharmaceutical company to sign up in what Trump said would be a series of deals designed to get medications to Americans at cheaper prices.
How exactly the new TrumpRx site would work wasn’t immediately clear. There were few details during Trump’s press conference where he was joined by Pfizer CEO Albert Bourla, as well as Health & Human Services head Robert F. Kennedy Jr. and Dr. Mehmet Oz, administrator of the Centers for Medicare and Medicaid Services. Pfizer did offer a few examples of drugs that would be available, many at discounts of 80% or more, including menopause drug Duavee, eczema treatment Eucrisa and overactive bladder therapy Toviaz. In exchange, the pharma giant will receive a three-year grace period from threatened tariffs, among other considerations. Some details of the Administration’s announcement were first reported by The Washington Post and The Wall Street Journal. Will this actually lower prices that you pay for medicine? Well, that depends. That’s because drug pricing is its own rabbit hole of listed prices, negotiated prices and rebates. So the impact for patients with good corporate health insurance (who typically do not pay anything close to the drug’s list price) will be very different than for those who are uninsured or underinsured. The latter are likely to be most helped by TrumpRx, as they have been previously by other efforts to offer drugs at discounted cash prices, including Amazon Pharmacy and billionaire Mark Cuban’s CostPlus. “That’s really just one more cash pay option,” Eric Levin, CEO of Scripta Insights, told Forbes. “It may be more expensive for certain drugs or less expensive for certain drugs. It’s going to vary a lot depending on what insurance [people] have.” Some 200 million Americans have private health insurance from their employers or have Medicare Advantage plans; for these people, having their insurance cover the cost at a negotiated rate may still be a better deal (though their employer or union may disagree). For those without insurance or who have high-deductible plans, the cash prices may be lower. “How much are prices really coming down?” Levin said. “Is it coming down off the fake list price? Or is it coming down off the average insurance price that people would pay?” Jayne Hornung, chief clinical officer of pharmacy at healthcare data provider MMIT, told Forbes by email that additional questions remain, including whether specialty medications, prescribed for chronic or rare conditions like multiple sclerosis, would be included and whether patients would need to get receipts to submit to their HSAs or FSA for reimbursement. “The scope of TrumpRx is also unclear since the list of drugs available and the depth of discounts have not been finalized,” she said. It wasn’t entirely clear whether the government would be in the business of handling drug sales itself, including warehousing and shipping, or would simply be providing a website where pharmaceutical companies or other cash-pay providers could make their products available at discount prices. William Blair biotech analysts also questioned in a research note whether companies would need to provide this option through TrumpRx or whether their own direct-to-consumer plans like LillyDirect or BMS Patient Connect would suffice. “We expect more companies will announce direct-to-consumer channels to further appease the Trump Administration,” they wrote. Or as Cuban told Forbes by email: “As long as they list everyone, including Cost Plus Drugs, I think it’s great!” As for Pfizer, its stock rose 7% yesterday on what Rajiv Leventhal, an analyst at Emarketer, called a “pretty savvy” move. “These are not their high-selling products. They’re older drugs that they’re not making a ton of money on anyway,” he said. “Meanwhile they create certainty in an uncertain environment with respect to Most Favored Nation threats, drug pricing reform and tariffs.” |
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 | Axsome Therapeutics' Herriot Tabuteau Getty Images |
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When Herriot Tabuteau started a drug development company in 2012, he decided to do so differently. First, he’d focus on brain disorders, treatments for which are notoriously difficult to develop and whose efficacy can be hard to prove. And he’d be both CEO and the scientific founder, bringing to bear his decades of experience investing in biotech startups and his medical training. But he’d take no venture capital, self-funding with help from friends and family. “If you do things exactly the same way as everybody else, you’re going to have the same outcomes as everybody else. And we wanted to have outcomes that stand apart,” Tabuteau, 57, says, during his first-ever interview with a reporter about his company, Axsome Therapeutics. Named for two parts of a nerve cell, the “axon” and the “soma,” Axsome has come a long way from its beginnings in a windowless three-desk office in New York’s Rockefeller Center, affectionately remembered as “the broom closet.” Today, it has three drugs on the market and five in the pipeline, with the potential to help the estimated 150 million Americans suffering from conditions like depression, ADHD or Alzheimer’s disease. Revenue for the 12 months ended in June reached $495 million, up 70% from the same period in 2024. Axsome is not yet profitable, booking a net loss of $247 million in that time. The company trades on the Nasdaq with a market cap of $6.1 billion; Tabuteau is a billionaire thanks to his 15% stake in it, plus options. He figures Axsome could hit $16.5 billion in peak sales from its current drug portfolio, which would put it among the top 25 drug companies by revenue today. Read more here. |
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Tylenol may be only the beginning. For years, Robert F. Kennedy Jr. has railed against a number of medications and therapeutics claiming without scientific consensus or evidence that they cause some kind of harm — autism (Tylenol), suicidal thoughts (Ozempic). Now, as President Trump’s head of Health and Human Services, he’s begun turning his opinions into public policy. His Make American Healthy Again initiative has already made it harder for people to get vaccinated against COVID-19 and pushed unverified claims about Tylenol, and he’s just getting started. So what might be next? Kennedy’s already told us. Read more here. Plus: Publicly traded Danish biotech Genmab agreed to buy Merus, the maker of a promising head and neck cancer drug, for $8 billion. Genmab anticipates the drug, called petosemtamab, will launch in 2027 (subject to regulatory approvals) and reach at least $1 billion in sales by 2029, making it a blockbuster. In a note to investors, William Blair analysts forecast peak sales of $3 billion to $4 billion for head and next cancers alone. The purchase price represents a premium of some 41% over Merus’s closing stock price on Friday. |
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Brooklyn-based Inspiren, which provides AI solutions to senior living centers, raised $100 million led by Insight Partners. The company declined to disclose its valuation, but it was worth $125 million after its previous financing in May, according to VC database PitchBook. Former Green Beret and cardiothoracic nurse Michael Wang founded Inspiren in 2016. The company plans to use the cash to expand nationally and improve its software capabilities. Plus: Thyme Care, which helps cancer patients navigate their care process with doctors and insurance companies, raised $97 million from numerous existing investors at a valuation that PitchBook pegs at $1.5 billion The new funds bring the Nashville-based startup’s total capital raised to $275 million. Forbes previously wrote about the company in 2023. |
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Ansa Biotechnologies raised $45 million and secured an additional $9 million in a round led by Cerberus Ventures. The Emeryville, Calif.-based startup manufactures DNA sequences for scientific research and other purposes, competing with the likes of Twist Biosciences and Integrated DNA Technologies. Its differentiator? It uses an enzymatic process rather than a chemical one, enabling it to synthesize longer and more complex genetic structures than conventional methods, CEO Jason Gammack told Forbes. “Ansa has created the better mousetrap for how we manufacture DNA,” Gammack said. “And it changes all the rules.” Plus: The U.S. opened an investigation into medical equipment and device imports. Similar investigations under so-called Section 232 scrutiny have previously resulted in tariffs on steel and aluminum. Tariffs on medical equipment and devices could cover everything from wheelchairs to IV bags, making hospital supplies more expensive and putting pressure on their already strained balance sheets. |
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PUBLIC HEALTH + HOSPITALS |
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Today is the end of one era and beginning of a new one for Northwell Health as Michael Dowling, who built the health system as CEO since 2002, steps down, and Dr. John D’Angelo becomes the new CEO. The not-for-profit health system is the Northeast’s largest, with 28 hospitals across New York and Connecticut, including Lenox Hill Hospital. D’Angelo, who began his career at Northwell as an emergency room physician at Glen Cove Hospital 25 years ago, told Forbes his goal is “proactive and personalized care.” To get there, D’Angelo is focused on cleaning up Northwell’s digital health portfolio, as years of mergers have left it with competing software platforms. He also plans to embrace AI solutions that can help ease administrative burdens on doctors and nurses. D’Angelo is taking the reins when hospitals are being squeezed by both political and technological changes. “I joke that’s why they picked an ER doc as CEO, because we’re good at navigating uncertainty,” he said. |
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How Tylenol’s troubled history helped make it a target for Trump and his followers. Tylenol maker Kenvue is bracing for a wave of lawsuits after the Trump Administration warned about its use during pregnancy. Pfizer’s $7.3 billion deal for Metsera could herald a new wave of M&A for pharmaceutical companies “tiptoeing into obesity.” Millions of Medicaid enrollees may be able to avoid Trump’s work rules because they live in counties with high unemployment. President Trump shared and then deleted a seemingly AI-generated video promising Americans access to “medbeds,” a fictional technology that far-right conspiracy theorists claim holds miracle cures. Harvard faces potential suspension or debarment from receiving federal funds through the Department of Health and Human Services after the agency accused the university of ignoring antisemitic harassment. A September federal court ruling found that the Trump administration “used antisemitism as a smokescreen for a targeted, ideologically-motivated assault on this country’s premier universities.” Doctors and nurses significantly outperform ChatGPT when it comes to triaging patients, according to research presented at the European Emergency Medicine Congress this week. |
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