| Sponsored by  | | |  | Hello from Day 2 of the virtualized J.P. Morgan Healthcare Conference! If you happen to be reading this at or around 4 p.m. ET, join STAT for a live discussion of all things JPM, featuring Alnylam CEO Yvonne Greenstreet and a bunch of reporters. | | | Nearing a pivotal moment for a novel schizophrenia treatment In November 2019, Karuna Therapeutics announced that its experimental pill called KarXT reduced the acute psychosis and related symptoms experienced by people with schizophrenia. The positive results from the mid-stage clinical trial suggested a new way to treat the serious mental disorder without the side effects of current medicines. Karuna’s stock price rose quintupled.
Now, Karuna is nearing the finish line on two, pivotal Phase 3 studies of KarXT, with readouts set for the middle and second half of the year. If the results from these studies confirm the benefit seen two years ago, Karuna will seek regulatory approval.
“We expect the trials to read out positive, but of course, you’d expect me to say that,” said Karuna CEO Steve Paul, speaking today at the conference. The company’s confidence in KarXT is bolstered by the design and conduct of the Phase 3 studies, which don’t veer at all from the Phase 2. “We’re keeping it simple,” he said. | Hello, nice to meet you Tuesday brought a reminder that in the pre-Covid days, serendipitous face-to-face meetings between biotech people could make cool science and business deals happen. The research partnership between Amgen and the biotech startup Arrakis Therapeutics announced today had its roots in a friendly introduction and chat that Amgen scientist/executive Ray Deshaies struck up with Arrakis’ CEO/scientist Michael Gilman at a 2019 symposium in Berkeley, Calif. Now, the two companies are teaming up to develop a new class of oral drugs that selectively destroy RNA molecules that turn genetic instructions into disease-causing proteins.
Read more. | Sponsor content by ICON Funding emerging biotechs from promising discoveries to life-saving realities The discovery of tomorrow’s extraordinary therapies, with the potential to solve some of the most challenging diseases and to fulfill the most demanding unmet needs, will require collaboration among biotechnology companies, clinical research experts, and venture capital firms. ICON is committed to helping biotechnology companies launch their therapies throughout the drug development process by facilitating this partnership and collaboration. Learn how. | Illumina goes long These days, DNA sequencing giant Illumina is tied up in a long and complicated battle over its acquisition of Grail, which has developed a blood test to help screen for cancer. But it wasn’t that long ago — OK, it was November 2018 — that Illumina was working on another deal: its acquisition of Pacific Biosciences. Regulators succeeded in scuttling that merger. The point behind the Illumina/PacBio deal was to combined two types of DNA sequencing technology: Illumina’s short-read sequencing, which assembles inexpensive tiny readouts like a puzzle, and PacBio’s long-read technology, which gives researchers much longer readouts, making assembling DNA code easier. At J.P. Morgan, Illumina announced that it will unveil its own long-read technology, dubbed “Chemistry X.” Illumina shares rose 15%, and PacBio shares fell 11%. Also, a small piece of evidence Illumina might be on to something valuable with the whole cancer screening blood test thing: Roche invested $290 million in Freenome, a Grail competitor that is also pursuing such tests, bringing the total amount Freenome has raised to more than $1 billion. | Sanofi: First-generation mRNA is 'doomed to fail' against flu Sanofi, the global vaccine giant that famously lost modern history’s biggest vaccine race, believes the mRNA companies that triumphed over Covid-19 are in for a reality check on its home turf of influenza.
Moderna and partners Pfizer and BioNTech are pressing forward with flu vaccines based on the technology that worked against Covid. But the “mRNA pandemic generation is doomed to fail against flu,” said Thomas Triomphe, head of Sanofi’s vaccines division, at the company’s JPM presentation today. The issue is tolerability: People are willing to deal with the often unpleasant side effects of current mRNA vaccines because the alternative is exposure to a deadly pandemic virus. But when it comes to an annual flu vaccine, the bar for safety will be higher, Sanofi believes. It’s worth noting Triomphe’s biases. Sanofi is the multibillion-dollar incumbent in flu vaccines, giving it a vested interest in dismissing challenges to its throne. And, having missed out on the first wave of mRNA, the company is banking heavily on the second, having paid $3.2 billion for Translate Bio and its theoretically more benign approach to the space. Soon there will be data to determine who’s right. Sanofi expects to get its own mRNA flu vaccine into clinical trials this year, and Moderna and Pfizer/BioNTech have promised more readouts in the months to come. | BioMarin is ready to make some money BioMarin Pharmaceutical opened its doors in 1997 with the goal of building a sustainable business by developing medicines for ultra-rare disorders. Twenty-five years later, the company has launched seven medicines and amassed a $16 billion valuation. What it has never done is consistently turn a profit. But that’s going to change in 2022, the company said at JPM. BioMarin’s latest approved drug, a treatment for the most common cause of dwarfism, is on pace to be its biggest product ever, CEO Jean-Jacques Bienaimé told STAT. The treatment, called Voxzogo, will make between $80 million and $110 million in 2022, BioMarin said, and increase from there. The company also believes it’s on the verge of approval for the first gene therapy for hemophilia A, a product analysts expect to bring in about $1 billion a year by 2026.
Add all that up, and BioMarin is on pace for profitability in 2022. “The key word is sustainably profitable,” Bienaimé said. The next challenge is maintaining it, which will require bringing forward more and more drugs from BioMarin’s pipeline. “It’s definitely a turning point for the company,” Bienaimé said, “but one has to be careful not to be too exuberant.” | Boom times for testing At last year’s conference, health care companies that dove headlong into the Covid-19 testing game had to temper their expectations for investors. Investments in test development, manufacturing, and distribution were an obvious need, but with the first vaccine jabs just going into arms, there was no telling how long demand would last. This year, as the wave of Omicron has swept across the world, a number of companies blew past their initial projections as fourth quarter testing volume exploded, both for PCR tests and over-the-counter antigen tests — and they’re riding high on the influx of cash. CVS Health saw retail revenue outperform in the fourth quarter, largely attributed to higher-than-expected vaccination volume in addition to testing, “both sort of the traditional testing we've been doing, but now the OTC testing that really, really took off in December,” said chief financial officer Shawn Guertin. Abbott, maker of lab-based PCR tests, rapid molecular tests, and the BinaxNOW antigen test, is manufacturing more than 100 million tests a month, said CEO Robert Ford, after the company revised sales projections down mid-year, when the CDC was more optimistic about the need for testing in vaccinated individuals. “The part that we did get right was the importance of rapid testing, the importance of scale,” he said. “The timing of it was a little bit difficult. That’s going to be our challenge.”
Their calculus may change again, as the Biden administration will require private insurers to cover the cost of up to eight at-home tests every month as of Jan. 15. Ford described the new policy as an opportunity for Abbott’s rapid test line, but Guertin was more balanced: If people begin using more over-the-counter tests because of the guaranteed reimbursement, they may in part replace more expensive PCR tests that insurers were already required to pay for. | What Best Buy doesn't want to do in health care When Best Buy hired Deborah Di Sanzo in September 2020, it signaled the company was serious about health care. In the year and a half since, Di Sanzo, a former executive at Philips and IBM Watson Health, has absorbed the retailer’s consumer electronics DNA.
“What our customers are telling us is that they want to shop for a physician the same way they shop for a TV,” she said at a JPM side event.
Televisions are not doctors, but Di Sando wiggled her way to a description of how the company’s approach to health care is rooted in the sales and installation services it’s always provided with gadgets: It sells fitness equipment and wearable devices to consumers and remote patient monitoring devices like connected blood pressure cuffs to seniors and others who need at-home care. It helps people set the devices up and delivers support over the phone or at home when necessary. While some retailers have an interest in delivering care, Di Sanzo says Best Buy’s not going there.
“We have a big red line that we won't cross, and that is, we are not going to be a provider,” she said. “So we don't have pharmacies, we're not going to have pharmacies. We don't have clinics, we're not going to have clinics. That's not our role… we’re Best Buy. We help people with technology in their home and people need help with health technology.”
One final cryptic note: Di Sanzo hinted at some health system partnerships that are “a little bit in stealth mode.” Shhh… make sure you don’t tell thousands of investors about that. | Thanks for reading! More tomorrow. | |
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