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Biotech / Medical : Immunomedics (IMMU) - moderated -- Ignore unavailable to you. Want to Upgrade?

To: ghettogoulash who wrote (63193)	7/27/2025 9:33:43 PM
From: sukit	4 Recommendations Recommended By bodish erippetoe ghettogoulash jhcimmu Respond to of 63320

The Sarepta Mugging and Drug Innovation The Trump FDA tries to kill a therapy that has helped boys with a deadly diagnosis. American companies are racing to develop gene therapies that treat dreadful maladies, with often spectacular results. But progress isn’t inevitable, and the story of a drug for a rare condition suggests that the Trump Administration is taking a bad turn that will chill investment in new drugs. The Food and Drug Administration this month asked Sarepta Therapeutics to suspend shipments of its gene therapy Elevidys. The drug treats Duchenne muscular dystrophy, and few ailments are more wrenching: Young boys lose muscle function as the disease progresses and eventually need wheelchairs, often before they’re teenagers. Some die in their 20s. The putative reason for the pause is safety. Two patients have died from acute liver failure after receiving the drug. Most recently, a 51-year-old man with a different condition died after receiving an experimental gene therapy made by Sarepta. The FDA said Friday it is investigating the death of an 8-year-old boy, which the company says was previously known and ruled unrelated to the gene therapy. **A tip that something is off here is that the FDA is eliding distinctions about these tragedies. The company says nearly 1,000 patients have been treated with Elevidys, a large sample size for a nascent gene therapy. Both Duchenne patients who died suffered from advanced disease and could no longer walk when treated. Sarepta more than a month ago paused shipments to patients who can no longer walk. It assembled a panel of experts to understand the risks and look at adding an immunosuppressant regimen to prevent liver overreaction for some patients. The company says it will add a black-box warning to help clinicians and patients make an informed choice. Investors are unhappy that they learned about the clinical trial death belatedly, which was a corporate PR debacle. But the company says it informed the FDA weeks ago when it happened. Yet the FDA has demanded the company stop shipping the Duchenne drug for all patients. Younger boys who can still walk are in better health and receive a lower weighted dose. They also have more to gain from prompt treatment—potentially more days to throw a ball or climb stairs. Therapies always carry risks, but patients and their families often prefer them to the brutal certainty of decline. The company initially refused to halt all shipments but later agreed. Yet it isn’t obvious what the company can now do to receive a clean bill of health. A senior FDA official told the press last week that the drug faced an “arduous and treacherous” path back to market and that the company suspended shipments because the agency had “a gun to its head.” Partial points for honesty that this is a regulatory mugging. What adds to this perception is that a self-professed Bernie Sanders acolyte who dislikes the drug is running FDA’s gene therapy shop. Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research, has lambasted his predecessor Peter Marks for twice overruling agency staff to approve the Duchenne therapy. Dr. Marks made good decisions to prioritize speed and patient choice over certainty on the data. The FDA noted in an approval last year that a large, randomized trial didn’t meet its primary aim. But boys improved on secondary measures such as “time to rise from the floor, 10-meter walk/run, time to ascend four steps.” There is “nothing ambiguous about these improvements,” as a father of a boy who received the therapy put it at a FDA hearing. Dr. Marks noted in his memo approving the drug that such secondary endpoints had been used to support prior drug approvals as primary* endpoints. This is the risk tolerance the agency is supposed to show, particularly for devastating and rare diseases like Duchenne with few or no treatments. Dr. Prasad has called Dr. Marks a rubber stamp who did “more for Sarepta than the CEO” and complained on his Substack that the Duchenne drug is among “the most costly” in history. That lays bare the real argument here, which isn’t about whether the safety risks in gene therapies can be managed. It’s a claim that the drug is an expensive false hope for desperate families who Dr. Prasad thinks can’t be trusted to make their own decisions about risks and benefits. That paternalism is familiar from Democratic administrations and the FDA bureaucracy. President Trump had a different policy in his first term when he made a cause of the 2018 “right to try” law. That measure had practical limits but was important as a policy statement that patients should be the first priority of regulators. ***The stakes here for drug development are enormous if Mr. Trump’s regulators crush a drug that has already won FDA approval. Several early gene therapies aren’t home runs, but drug development is iterative and builds on trial and error. Forget about better drug iterations if the agency kills a company for an emerging safety warning in some patients. FDA Commissioner Marty Makary has been talking about faster approvals and more flexibility for innovative drugs, but investors in the next cure bet capital based on the agency’s actions. Meanwhile, families facing a terrible diagnosis are left wondering if the only gene therapy on the market will be an option for their sons. If Americans wanted fewer novel treatments, they could have elected Kamala Harris.

To: ghettogoulash who wrote (63193)	7/27/2025 9:42:27 PM
From: sukit	3 Recommendations Recommended By bodish ghettogoulash jhcimmu Respond to of 63320

Vinay Prasad Is a Bernie Sanders Acolyte in MAHA Drag A top FDA appointee who doesn’t think patients can be trusted to make their own healthcare decisions. Meet Vinay Prasad, a young disciple of Bernie Sanders who ranks as one of the most powerful officials in the federal government. He determines whether patients get access to many life-saving medicines. Or not. Think of him as a one-man death panel. Dr. Prasad was named by Marty Makary, the Food and Drug Administration commissioner, as head of the agency’s biologics division in May, and last month as its chief medical and scientific officer. More on why he was tapped to these posts later. But it isn’t an exaggeration to say that Dr. Prasad wields more power than Anthony Fauci ever did at the National Institutes of Health. Vinay Prasad. Photo: Food and Drug Administration Like Dr. Fauci, Dr. Prasad thinks he knows what’s best for people, and that it’s government’s job to make it happen. “I favor a strong regulatory state,” he proudly professes. And just as Dr. Fauci slowed experimental HIV treatments during the 1980s by rigidly adhering to strict trial protocols, Dr. Prasad is now scuttling potentially life-saving therapies. In recent weeks, the FDA has rejected three therapies for debilitating diseases that have shown promise in clinical trials. The agency has also forced off the market a gene therapy that can slow the degenerative loss of muscular function in young boys with certain genetic mutations. Behold America’s strong and arbitrary regulatory state at work. If you read Dr. Prasad’s paper—or follow his smash-mouth Substack and X.com feeds—the FDA’s recent actions are no surprise. Dr. Prasad has long criticized the FDA for approving too many treatments that, in his view, provide only marginal benefits. He’s also lambasted President Trump’s first-term “right to try” law, which lets terminally ill patients try experimental drugs not yet approved by the FDA. “It is crucial to question whether non-curative therapies . . . are worth it,” Dr. Prasad wrote in a 2021 paper about an FDA-approved cell therapy for multiple myeloma. The therapy reduced disease progression or death by half in patients with advanced cancers who hadn’t responded to already approved therapies. Impressive. But Dr. Prasad complained that the treatment was pricey (then $419,500 for a course) and may “only delay inevitable progression” in some patients. In other words, sick patients should just give up and die. Got that? In 2016 he wrote an op-ed titled “The case for rationing: Why we should limit public spending on cancer drugs.” He exalted the United Kingdom’s socialized health system for restricting access to new treatments until they demonstrate a high degree of efficacy in multiple trials and that their benefits—as determined by the government—exceed their costs. Such government rationing is why survival rates for hard-to-treat cancers are much lower in the U.K. than in America—and why British patients with the financial means cross the pond to receive innovative and often life-saving treatments. Tough luck to Brits of average means. Of course, Dr. Prasad insists he really has patients’ best interests at heart. Why would terminally ill patients want to waste their precious remaining time on earth schlepping to hospitals for treatments that may not cure them when they could be preparing for their deaths? That’s the gist of his 2022 paper, which estimated patients with advanced cancers spend 16 more hours a month accessing and receiving novel treatments than if they accepted hospice or home palliative care. “Time is a valuable resource for people who have cancer,” the paper noted. Yes, and that’s why they want to continue living. He has also argued that “genome-informed cancer medicine”—treatments targeted based on a patient’s genes or tumor mutations—“is mostly hype,” no matter that such treatments have produced most recent improvements in cancer survival. Take CAR T-Cell therapies that re-engineer a patient’s immune cells to target proteins on tumor cells. Such therapies can cure aggressive cancers that not long ago carried a death sentence, though Dr. Prasad has hyped their side effects. He has done the same for Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, which the agency this month forced off the market after two patients in the advanced stages of the disease died from apparent side effects. The deaths look to have been a pretext for Dr. Prasad to deep-six the drug, which he had previously criticized as too costly for its benefits. Most treatments carry rare, life-threatening side effects, but doctors and patients can weigh their risks against their benefits. Perhaps Dr. Prasad doesn’t trust people to do so any more than Dr. Fauci did. Dr. Prasad found common cause with Dr. Makary and conservatives in opposing paternalistic Covid policies including vaccine mandates and school shutdowns. But his other positions are at odds with Dr. Makary’s stated support for more flexible reviews of drugs that treat rare and deadly diseases and the MAHA ethos of patient empowerment. Dr. Makary in spring 2021 lambasted the FDA for using the “eternal excuse of safety” to pause the Johnson & Johnson Covid vaccine after a rare blood-clotting side effect cropped up, mostly in middle-aged women. “This is a life-saving medication,” he then wrote. “What ever happened to giving people the data and letting them make their own health decisions?”