| Sponsored by  | | |  | Hello from JPM Week 2023! We’re coming to you live from San Francisco, where healthcare’s annual convocation has re-gathered after two years in Covid-19 isolation. It’s nice to be back, although the weather? The precipitation is plentiful. Our morning newsletter has switched to an afternoon format so that our team of STAT reporters on the ground — Adam Feuerstein, Matthew Herper, Allison DeAngelis, Jonathan Wosen, Jason Mast, Bob Herman, and Mohana Ravindranath — can bring you all the news, analysis, and cocktail party gossip. Let’s do this! | | | 
At J.P. Morgan, A metaphorical statement from the literal ocean (Adam Feuerstein) Gloomy weather — and attitudes — hover over San Francisco What should be a triumphant return to the global biotech industry’s biggest annual event, “JPM Week,” after a two-year hiatus is turning as gloomy as Bay Area weather, with industry executives forecasting economic troubles in the year ahead.
STAT asked more than 100 biotech and pharma executives, investors, and other insiders how they’re feeling about the state of the sector heading into 2023 and on the eve of the J.P. Morgan Healthcare Conference in San Francisco. The biggest takeaway: Nearly 70% of respondents said financing — raising venture capital, launching an IPO, or selling additional stock to further fund R&D and other operations — will be a headwind in 2023.
But biotech insiders are not entirely glum. STAT’s survey shows optimism for science and innovation, with 40% telling us that R&D productivity is a positive tailwind for 2023.
Read more. | BioMarin hopes it has finally, finally answered all the FDA’s questions Biomarin’s gene therapy for hemophilia A, Roctavian, was approved by European regulators in August. But the company has been waiting through a protracted review with the FDA as it has waited through delay after delay on what its executives have said for years could become its biggest seller.
On Sunday morning the company issued a press release with two updates that could help speed things up before the FDA’s current decision deadline of March 31. First, Roctavian had positive three-year results in a 134-patient study, with patients’ annualized bleeding rate down 80%. And BioMarin said the FDA’s pre-license inspection of its manufacturing facility is complete, potentially removing another reason the FDA’s decision on the gene therapy could be delayed again. | Sponsor content by Amgen 5 ways biotech can help support patients and their communities As a company, Amgen’s mission is to serve patients, and while innovating new medicines may be at the core of what they do every day, staff are always excited for new ways to serve not only patients, but also the communities where they live and work. Learn more about how the people of Amgen came together in 2022 to help patients, families and communities live happier, healthier lives. | Don’t forget about the providers and health insurers Biotech and pharma aren’t the only subsectors angling for attention at JPM. A cornucopia of hospitals, health insurers, and other provider-related companies are back in person as well, and they have a familiar pitch to the crowd: Most health care dollars are flowing through or to them.
Of the $4.2 trillion Americans spent on health care in 2021, almost 60% went toward hospitals, outpatient centers, and doctors — nearly identical to prior years. In other words, spending on hospitals and physician care didn’t slow down even though admissions and procedures have receded since the pandemic. Health insurers also have been big beneficiaries of those lower volumes, pocketing record profits and paydays.
Our colleagues Bob Herman and Tara Bannow will be closely tracking 17 not-for-profit hospitals as well as a host of other public and privately traded companies. A handful of the private provider companies may be using this JPM to test the waters for going public. Read their walk-up to see which of those companies they're watching, and why. | Promising data for a common pediatric brain cancer Updated data from Day One Biopharmaceuticals show that its targeted therapy shrank tumors by at least 50% in a majority of children with the most common form of pediatric brain cancer, the company said today, results that could support regulatory approval.
In the small trial, of the 69 children with low-grade glioma who had been evaluated, 44, or 64%, saw their tumors dwindle to less than half their original size, including three patients whose tumors disappeared. Their responses lasted 8.4 months at the median, and that number may rise as the trial continues. As of Sept. 28, when the data were collected, 77% of patients were still taking Day One’s drug, known as tovorafenib.
“The most remarkable thing about this study is that most of the patients had received numerous prior lines of therapy,” said Craig Erker, a pediatric oncologist at Dalhousie University in Halifax, Novia Scotia, who was not affiliated with the work. “And they still see a good overall response.”
Day One plans to submit tovorafenib for approval in the U.S. in the first half of this year.
Read more. | Can Cologuard hold off the cancer-detecting blood tests? Last month, investors were largely disappointed by the colorectal-cancer-screening blood test developed by Guardant Health. SVB Securities analyst Puneet Suda predicted that would help Exact Sciences, the maker of the Cologuard test to detect colorectal cancer.
Exact shares are still down 37% over 12 months. Maybe this will help: Exact pre-announced Sunday that its fourth quarter revenue should top $550.7 million, 9% higher than investors expected. Total revenue for 2022 will be about $2.08 billion, an increase of 18%, with $1.4 billion coming from screening tests. | Someone has to pay for all those free drinks For those that need reminding, the J.P. Morgan Healthcare Conference is an investment banking conference. Meaning, the biotech and pharma CEOs presenting at the Westin St. Francis hotel over the next four days were not selected because of their suave personalities. They are here because their companies are fee-paying clients of J.P. Morgan — or its bankers are wooing them to be fee-paying clients in the near future. Jamie Dimon is not running a charity, folks.
Two Januaries ago, CytoKinetics spent JPM Week appearing virtually at a D-list banker’s confab. Tomorrow morning, CEO Robert Blum has a highly coveted speaking slot inside the Westin. Consider it a thank-you from the bankers that helped Cytokinetics raise $300 million in July 2021. Cytokinetics is also developing a very promising, and potentially lucrative, heart drug with a pivotal study readout later this year. Acquisition candidate? Monday’s primo slot says yes.
Arrowhead Pharmaceuticals is not a J.P. Morgan client, but was also given a presentation slot on Monday morning. Draw your own conclusions. | More reads - FDA approves Alzheimer’s drug shown to moderately slow cognitive decline in early stages of the disease, STAT
- Chinese-made mRNA vaccine starts trial production, Reuters
- Fate Therapeutics plans mass layoffs, after early end to cell therapy deal with Janssen, STAT
- Billionaires for a 'hot minute': Biotech slump whipsaws Boston founders, Bloomberg
That's all for the weekend. See you tomorrow for a recap of Day 1! | | | |
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