| Sponsored by  | | |  | Hello from not-San Francisco! The second-straight virtual J.P. Morgan Healthcare Conference got underway this morning, and here’s the first of your daily recaps from the industry’s largest annual meeting. We’ll have more tomorrow and all week. | | | Pfizer talks up an mRNA future When you expect to sell $31 billion worth of Covid-19 vaccine in 2022, the future starts to look a lot like the technology behind that vaccine — that is, mRNA.
Witness Pfizer’s news from J.P. Morgan: The company not only spent $300 million up front on the gene editing technology being developed by Beam Therapeutics, which is based on mRNA, but also licensed rights from Acuitas to use the lipid nanoparticle packaging that’s used to get mRNA into the body. And it licensed technology it says could speed up the creation of new therapeutics, potentially cutting the time to produce a new Covid variant vaccine from three months to two. “This would be the first step in a pretty rapid sprint to build this comprehensive growth engine of mRNA-based products,” Pfizer’s chief scientific officer, Mikael Dolsten, told STAT in an interview.
The hope, in short, is that Pfizer can turn a once-in-a-lifetime pandemic windfall into a sustainable business. | Vertex CEO dismisses cystic fibrosis competitor That would be Reshma Kewalramani, who must have swallowed an extra dose of feisty before using her podium time Monday to warn Abbvie that it has no business being considered a viable rival to Vertex’s dominant franchise of cystic fibrosis treatments. Kewalramani didn’t mention Abbvie by name, of course, but the message was still delivered loud and clear.
Abbvie is developing a cocktail of drugs to treat patients with cystic fibrosis and will read out results from mid-stage studies later this quarter. But whatever those early Abbvie data show, Kewalramani reminded investors that Vertex’s flagship treatments, led by Trikafta, have already established a “tremendously high bar” of long-term benefits for people with cystic fibrosis, including meaningfully lower risks of death, lung transplant, and pulmonary exacerbations.
It will take years for any competing cystic fibrosis treatment to match Vertex’s data, if ever, she said. The company is expected to report $7.5 billion in sales this year, with growth well into the middle of the decade, Kewalramani said. Vertex’s closest competitor is actually Vertex, she added, noting that its improved version of Trikafta is further along in clinical trials than anything else. Translated: Abbvie, sit down. | Sponsor content by ICON How can a strong regulatory strategist support your unique needs? Innovative biotech products present a complex regulatory environment. For this reason, a formidable regulatory strategist is a critical member of the drug development team. A regulatory strategist assists in navigating the regulatory maze of global development and ensures a tight focus on regulatory pathways. In a new whitepaper, learn how a strong regulatory strategy can support small biotech through every phase of drug development. | Another Covid treatment? Very little has gotten investors excited so far this J.P. Morgan. One thing that did: new data on a Covid treatment from Molecular Partners, a Swiss biotech. Novartis exercised an option to license the product, giving Molecular Partners 150 million Swiss Francs, or about $160 million.
The drug, ensovibep, is a type of protein therapy called a DARPin that is easier to manufacture than a monoclonal antibody. In a placebo group of 99 patients, 6% were hospitalized, sent to the ER, or died. By comparison, 1.3% of the 301 patients who received the drug were hospitalized or visited the ER. None of those in the drug group died. The study predated the Omicron wave.
But that statistical analysis was not prespecified, and will leave the Food and Drug Administration and other regulators with the challenge of deciding whether to authorize a drug based on such a small dataset. Still, the story of Covid so far has shown that options that seem like they’re on the back of the shelf can become a first choice rather quickly. Example: With monoclonal antibodies ineffective or scarce, some doctors are giving Gliead’s remdesivir to patients who have not yet been admitted to the hospital to keep them out. In a global pandemic, there might always be room for another tool. | How Biogen went from 'wrong' to 'courageous' Here are some words people have used to describe Biogen’s $56,000-a-year price tag for the Alzheimer’s disease treatment Aduhelm: “unacceptable,” “unconscionable,” “absurd.” Biogen CEO Michel Vounatsos sees it differently. Speaking at JPM this morning, he defended the original price as “evidence-based,” adding “if you look at comparators, you’ll see that this is a price that eventually we could defend.” Nonetheless, the scathing backlash from physicians, payers, and patients proved Biogen was “wrong,” Vounatsos said, which is why the company cut Aduhelm’s price by roughly half last month. “This is courageous,” he said. “We could have waited and dictated the need to change over time based on competitive pressure on access decisions.” What remains to be seen is whether the reduced price will change the minds of physicians and payers who have serious doubts as to whether Aduhelm actually benefits patients, regardless of its cost. Meanwhile, Medicare is scheduled to release a draft coverage decision that could make or break Aduhelm’s future by Wednesday. Alisah Alaimo, president of Biogen’s U.S. operations, said the company would see the draft Medicare decision at the same time it’s released publicly, and she declined to speculate on the outcome. Read more. | M&A MIA Monday passed without the announcement of a blockbuster acquisition, so sound the sad trombones. The most widely followed index of biotech stocks, the XBI, didn't exactly soar, ending the day .15% above its open. For reference, it's down 30% over the past 12 months.
It’s unrealistic to expect a mega-M&A deal during every JPM Week, but hopes were running higher this year given Big Pharma’s bulging balance sheets and their need to restock drug pipelines. The deals could come eventually. The CEOs of Bristol Myers Squibb and Novartis both reiterated their optimism for “bolt-on” deals — a high priority, they both said. But then Bristol announced a $5 billion acquisition of its own stock. Monday’s best individual stock performance came courtesy of BioCryst Pharmaceuticals, which pre-announced better-than-expected 2021 sales, and higher 2022 sales guidance, for its recently approved rare-disease treatment.
Shares of Amicus Therapeutics fell the most Monday after reporting disappointing 2021 sales of its rare disease drug and a weaker-than-expected outlook for 2022. | Moderna has $17 billion and a plan for vaccine domination Moderna, which made nearly $18 billion from its Covid-19 vaccine last year, has a plot to turn its superlatively successful vaccine into a product with eternal demand.
As CEO Stéphane Bancel explained this morning, the idea is to craft a “pan-respiratory annual booster shot” that would evolve each year. Right now it’s an mRNA directed against Covid-19. If clinical trials go well, it will become a combination that protects against SARS-CoV-2 and the year’s dominant flu strain. In time Moderna would add an mRNA for respiratory syncytial virus, and then for a common coronavirus called OC43. Eventually, the company would have an omnibus mRNA and market it to governments around the world as a must-have annual injection.
Whether those plans come to fruition is years away from resolution. But in the meantime, Moderna has $17 billion in cash and a nearly $100 billion valuation, with another $20 billion or so in revenue expected in 2022. The company’s doing so well that its once-vaunted pipeline of mRNA therapeutics, which has made little progress over the past three years, barely featured in the investor Q&A. | Oak Street Health looks toward expansion Executives with Oak Street Health, a provider of virtual and in-person primary care to Medicare patients, said they will open the throttle on their expansion plans in 2022, adding 70 new health centers.
That would give the company a total of 200 clinics nationwide, in addition to a virtual specialty consultation business it added through the acquisition of RubiconMD. Chief Financial Officer Tim Cook estimated the company’s adjusted EBITDA will top out at $1.3 billion this year.
Oak Street is also facing some headwinds, however, including a Department of Justice inquiry into whether the company violated the False Claims Act related to third-party marketing campaigns to Medicare beneficiaries. CEO Mike Pykosz said risks to the company’s core business are minimal, noting those marketing programs account for less than 10% of new patients. | Thanks for reading! More tomorrow. | |
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