| | | |   | We have a bump in the road for Sarepta's next-gen Duchenne drug, another public company going private, and more. Happy Friday! - Meghana | | | Sarepta’s new Duchenne drug on clinical hold The FDA has placed a clinical hold on a second-generation drug for Duchenne muscular dystrophy from Sarepta Therapeutics. A patient taking the experimental drug, called SRP-5051, experienced a “serious” decrease in blood-based magnesium, a condition called hypomagnesemia. The patient didn’t require hospitalization, and the condition cleared up within three days. SRP-5051 is meant to replace Exondys 51, Sarepta’s controversial first drug approved for Duchenne’s that treats about 13% of patients with the genetic disease. The new drug was designed to better penetrate cells, and produce more of the muscle protein dystrophin. Read more. | Uniqure’s gene therapy for Huntington’s has potential Uniqure’s gene therapy for Huntington’s disease is showing some promise in a small study — triggering a stock jump of 27% yesterday. The results from the low-dose intracranial infusions “exceeds our expectations,” SVB analysts say. Twelve months after treatments, four patients who received AMT-130 saw levels of mHTT, the protein that causes the neurodegenerative disease, decline between 44% and 71%. In comparison, the three patients who received a placebo treatment had results ranging from a 35% increase in mHTT to a decrease of 47%. Although it’s encouraging that the gene therapy can lower levels of the toxic protein, the next step is determining whether that will halt the disease: “Genetics is destiny for Huntington’s,” Uniqure’s R&D chief told STAT. “But of course, we don’t know whether just removing the stimulus is enough to prevent degeneration from happening.” Read more. | Over a decade later, what’s next for the future of immunotherapy? Since 2011, the FDA has approved over three dozen novel immunotherapy treatments for patients with various forms of cancer. While these therapies have improved outcomes for some patients, there are others whose tumors don’t respond well enough to these medicines. Learn how the next generation and personalization of immunotherapies will help deliver more options for people with cancer. | Just how powerful is the FDA? Is Merck about to spend $40 billion? And what’s a “Puff Bar"? We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. STAT Washington correspondent Nicholas Florko joins us to discuss the FDA’s decision to ban the sale of Juul Labs vaping products and a proposal to reduce the amount of nicotine in cigarettes. We’ll also explain the latest news in the life sciences, including a rumored blockbuster buyout and the next Theranos verdict. Listen here. | This is why you pay attention to Adam’s take STAT’s Adam Feuerstein can attract some hostility on Twitter — with day traders more than happy to pit their views of biotech stocks against his. That didn’t happen this time. One day trader, Jake (not his real name), told Adam he lost $200,000 by trading and investing in Ampio Pharmaceuticals. “I should have listened to you,” he told Adam, referring to a 2018 column that warned against the osteoarthritis drugmaker. So Jake has some advice for other erstwhile casual investors in biotech: “Stay disciplined, and don’t get get stuck holding a biotech stock because someone is telling you there’s a buyout just around the corner that will turn your $100,000 into $3 million,” he said. “Those guys bragging about trading a fortune are really just sitting in their basements.” Also, Jake warned: “Stay off social media.” Read more. | Radius Health bought by private investors Radius Health is going private, bought up by two investment firms in a deal worth $890 million. The publicly traded company, whose lead product Tymlos treats osteoporosis, reported revenue of $230 million last year, down from $239 million in 2020, FiercePharma writes. Radius Health’s stock jumped 22% on the news that it was selling itself to Gurnet Point Capital and Patient Square Capital. Just recently another drugmaker went a similar route. Private equity firm EQ Healthcare Partners bought TherapeuticsMD, a maker of hormonal treatments for women, in a $177 million deal. Are we seeing a new trend here? | More reads - A CVS whistleblower lawsuit comes at a pivotal moment for the FTC’s probe of pharma and PBMs. STAT
- FDA unveils 5-year action plan for neurodegenerative drugs, with focus on ALS. (FierceBiotech)
| Thanks for reading! Until next week,  | | | |
No comments