| Sponsored by  | | |  | Greetings from 2021’s stay-at-home J.P. Morgan Healthcare Conference. Before we recap the day, a word about STAT Trials Pulse. It’s a new clinical trials intelligence platform, built by AI company Applied XL and vetted by STAT’s national biotech team, that helps life sciences professionals identify the most important updates happening in the clinical trial space. Visit statnews.com/trialspulse to learn more. And if you like what you see, enjoy your first four weeks free when you subscribe today. | | | Biogen is not having a great week A day after Biogen CEO Michel Vounatsos called his company “courageous” for lowering the price of an Alzheimer’s disease treatment with questionable benefits, Medicare proposed a plan that would dramatically restrict its coverage of the drug and torpedo the company’s efforts to increase its sales.
As STAT’s Rachel Cohrs reports, Medicare is proposing to cover Aduhelm only for certain patients and only when they’re enrolled in a placebo-controlled study. Biogen estimates that roughly 80% of patients eligible for Aduhelm are Medicare beneficiaries. That means that unless Medicare’s feelings change between now and the April deadline for a final policy, the drug’s commercial prospects are negligible.
While Biogen’s escalating misfortune has been a constant subplot of JPM, the Medicare situation has implications for the whole drug industry, Matthew Herper writes. FDA approval, like the one Aduhelm received in June, used to be the major milestone for new drugs. Biogen’s Medicare saga will test what happens when the decision to pay for a drug in the U.S. is further decoupled from the decision to make it available for patients to purchase — a prospect that appeals both to libertarians and advocates for stricter controls on the price of medicines. | Headspace gets into the chatbot game Headspace Health, which was formed last year with the merger of tele-mental health service Ginger and mindfulness app Headspace, today announced the acquisition of Sayana, an AI mental health app.
That’s a lot of apps! Let’s unpack this a little.
Headspace Health makes a similar pitch to many mental health startups: It’s using tech to address the inadequate supply of trained mental health professionals. During his remarks this morning at JPM, CEO Russell Glass said 80% of members don’t need clinical care, and he described how the services combined in last year’s merger plus the new addition of Sayana allow the company to serve very large populations.
“If you look at Headspace as prevention and as subclinical and then you look at Ginger as highly clinical and moving into the coaching and clinical care, Sayana really sits in the middle,” he said.
Sayana, a Y Combinator alum, engages users with an automated persona that asks about their moods and teaches lessons based on cognitive behavioral therapy and other techniques. Tech behind the scenes guides users to different content. The app shares similarities to the chatbot-based treatments being developed by Woebot, which last year raised $90 million.
Headspace Health, which has deals to serve the employees of 3,500 companies and the populations of a number of commercial health plans, leans on AI to get people to the right care and to help clinicians and coaches work faster. For example, it uses natural language processing to analyze what users write in messages and suggests smart replies, cutting back on the amount of time providers need to spend constructing their responses.
Given Headspace’s passion for tech-powered efficiency. Sayana’s completely automated treatment would seem to fit right in.
“They've used technology and content to solve for a number of more clinical needs through dialectic behavioral therapy and cognitive behavioral therapy,” said Glass. “And these experiences they've created help move us more upstream from a content perspective into these harder issues to manage.” | Sponsor content by ICON How small biotech can navigate the complexities of asset development Emerging biotech companies may require unique and specialized expertise to guide them through the development stages of their asset. In a new whitepaper, gain insight into the common questions small and emerging biotech companies face, and the importance of early engagement and regulatory affairs when developing an asset and considering a drug development partner. | Remdesivir redux Remember remdesivir, or, as Gilead calls it, Veklury?
A few months ago, you’d probably forgotten about this Covid drug. Monoclonal antibodies worked better early in the disease and Pfizer’s Paxlovid was on the way. But the monoclonals mostly whiffed against Omicron, and Paxlovid’s supply is still ramping up. And in December, the New England Journal of Medicine published a study about using remdesivir to prevent Covid patients from landing in the hospital.
Daniel O’Day, Gilead’s CEO, told STAT that discussions with the Food and Drug Administration are ongoing and that an emergency use authorization for remdesivir in outpatients could come in the coming weeks. Yes, the drug has to be given intravenously on three consecutive days, but for some patients, such as the immunocompromised, it might be useful. “I’m not suggesting that over time this would be the first choice of therapy, but under the current environment, it presents a real option,” O’Day said.
Gilead is also working on a remdesivir pill. “It’s not something that’s going to materialize in the course of this year, but we’re going to move with speed,” he said. | CAR-T comeback? Another blast from the past from Gilead: Could CAR-T, the cell therapy against cancer, finally have its day soon?
Remember that CAR-Ts from Gilead and Bristol-Myers Squibb outperformed standard second-line therapy — a stem cell transplant — in studies presented in November in B-cell lymphoma. O’Day, Gilead’s CEO, said he expected that adoption of the treatments as second-line therapies could take time, but he predicted it will happen.
Gilead says there are 11,000 B-cell lymphoma patients in the U.S. who do not respond to their first treatment, but that half of those are not considered good candidates for a grueling transplant. Of the remaining half, most could be considered for CAR-T therapy. Right now, only about 2,700 of those patients who are “transplant intended” make it to the actual transplant, the company said. | What does a drug’s launch price say about its future? On or before Feb. 17, Agios Pharmaceuticals is expected to secure U.S. approval for mitapivat, the first treatment for an inherited form of anemia called pyruvate kinase deficiency, or PKD. Approximately 3,000 patients in the U.S. are diagnosed with PKD, so like other marketers of drugs for rare diseases, Agios will charge a lot for mitapivat. The company hasn’t disclosed its price, but analyst consensus is roughly $300,000 per year.
Speaking Wednesday at JPM, Agios CEO Jackie Fouse expressed confidence in the coming mitapivat approval decision and said the company is preparing for the commercial launch.
But Agios is also developing mitapivat to treat beta-thalassemia and sickle cell disease, which are more common blood disorders. There are about 100,000 people in the U.S. with sickle cell disease, for instance, so if mitapivat proves to be an effective treatment, what will Agios do about the drug’s price?
Fouse said launching mitapivat first as a treatment for the rare PKD is an advantage, and the company might consider lowering the price if the drug is eventually approved for beta-thalassemia and sickle cell disease. She noted beta-thalassemia might be rare enough in the U.S. to keep the drug’s price high, adding the totality of the data and the extent to which patients benefit, particularly in sickle cell, will dictate mitapivat’s future pricing.
But there’s another way to look at a $300,000-per-year starting price for mitapivat: Agios isn’t confident the drug ever grows up to become a meaningful treatment for patients with sickle cell. That might reflect the early data, which were decidedly mixed and caused Agios’s stock price to fall. The drug also faces significant competition from other experimental treatments for sickle cell, which could prove to be more effective.
The real reason Agios might have decided to price mitapivat at $300,000 per year is because executives concluded it will never be more than a rare-disease drug. | The awkward business of biotech derailment Back in 2020, Generation Bio was among a crop of promising, early-stage biotech companies. Generation was yet to push any of its next-generation gene therapies into clinical trials, but it had a plan for doing so, and investors were willing to be patient. The company raised $230 million at a valuation exceeding $1 billion. By December 2020, Generation was worth twice that.
Then, last month, the company hit the reset button. Preclinical development was not going according to plan, and Generation rescinded all of its timelines for starting a first clinical trial. The stock price fell by about 60%, and now the company is worth about one-third of its IPO price.
All that made this morning’s JPM presentation a study in damage control. CEO Geoffrey McDonough reached for railroad imagery. While Generation cannot say specifically when it will be ready to move from animal testing to human trials, the process is like “bringing each system onto the rails,” he said. After that, “it becomes a little bit more like a train schedule: We know the path” to clinical trials.
Generation’s volatile post-IPO experience underscores a common question in biotech: It’s increasingly easy for early-stage firms to go public, but does that mean they should? In fairly recent biotech history, companies in the earliest stages of honing their science simply stayed private, in large part because the ups and more frequent downs of preclinical drug development can lead to unpredictable trading. | Capitalism, man A few months ago, STAT reporter Adam Feuerstein volunteered to fly to San Francisco to attend the J.P. Morgan confab in person. His only condition for accepting the assignment was being able to stay at a decent hotel, lest he come down with Covid and require the comfort of high-thread-count sheets.
So, he booked a room at the W Hotel just south of San Francisco’s financial district, for which his bosses were prepared to pay about $1,000 per night.
You know what happened next — Omicron and J.P. Morgan’s decision to take its conference entirely virtual. Feuerstein cancelled his travel plans. But out of curiosity, he checked to see what the W was now charging for the same hotel room — absent the demand from the biopharma and adjacent crowd.
$280 per night. | Thanks for reading! More tomorrow. | |
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