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Welcome back to Endpoints Weekly! Our team of reporters covered first-quarter earnings results from a handful of companies this week. See below for pharma executives’ comments on tariff exposure, dealmaking and more. We also have an assessment of the industry’s first quarter from Endpoints News’ founding editor John Carroll, who broke down numbers and charts on dealmaking activity, VC investment and more. We’ll be following more Q1 updates next week from Pfizer, Moderna, GSK and others, so stay tuned.
And keep an eye out for news from the American Association for Cancer Research Annual Meeting in Chicago, where our reporter Lei Lei Wu is on the ground covering the latest readouts and trends. — Nicole DeFeudis |
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Nicole DeFeudis |
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Editor, Endpoints News |
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Earnings season is upon us |
Some of the biggest names in the pharma industry reported their first-quarter earnings this week, including Sanofi, Roche, Merck, Bristol Myers Squibb, Gilead and AbbVie. Tariffs were top of mind for investors, with each company’s executives getting peppered with questions about how President Donald Trump’s trade war would affect the industry.
Most opted for a wait-and-see approach, but Roche CEO Thomas Schinecker notably took a proactive stance in warning of a negative impact on dealmaking. Any questions or unknowns about tariffs would make it “more difficult to make financial sense of any M&A deals,” he said Thursday. Roche estimates that four of its medicines make up about 92% of its tariff
exposure, though it wouldn’t specify which drugs those are.
Here’s a quick rundown of some of the other highlights:
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Makary on rare disease |
FDA Commissioner Marty Makary floated a potential new regulatory pathway for some rare disease treatments. In a SiriusXM interview, Makary highlighted that randomized controlled trials aren’t always feasible for extremely rare diseases. He told Megyn Kelly that drugs could potentially be cleared for use “based on a plausible mechanism” on a “conditional basis,” and the agency would closely monitor patients on the drugs until there’s a clearer signal in the data.
He reiterated the pathway in an interview with PBS’ “NewsHour” co-anchor and co-managing editor Amna Nawaz this week.
“If you're treating a condition that only affects 52 kids in the world, we shouldn't be requiring a randomized controlled trial. We may want to authorize or approve something for that condition based on what I call a plausible mechanism, a plausible scientific mechanism,” Makary said during the interview. Further details remain unclear at this time.
The idea echoes work by former CBER Director Peter Marks around accelerated approvals for rare disease gene therapies, as well as a bill from former Sen. Mike Braun (R-IN) and Sen. Kirsten Gillibrand (D-NY) to create a new way for the FDA to grant time-limited provisional approval to rare disease drugs that substantially demonstrate
early evidence of safety and efficacy. |
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Startup is first to inject CRISPR in the brain |
A Chinese biotech shared preliminary results from the first study to test a CRISPR-based treatment in the brain. Alvin Luk, CEO of HuidaGene Therapeutics, spoke to Endpoints’ Ryan Cross last week about the company’s first clinical trials and its business strategy.
What is MECP2 duplication syndrome? It’s a rare neurodevelopmental condition that can cause intellectual disability, movement problems and seizures. HuidaGene gave a low dose of its experimental therapy to a 9-year-old boy with MECP2. Three months later, the boy appeared to have improved eye contact, motor skills and walking ability, and was able to respond to simple directions such as “come to eat” or “go to the bathroom.”
The results are still early, but HuidaGene is now considering partnerships that could advance its programs. The company also recently unveiled early results for its experimental CRISPR therapy in Duchenne muscular dystrophy. The DMD and MECP2 therapies were both tested in an investigator-initiated trial, where academic or hospital researchers can move forward with less stringent requirements. But Luk said he plans to take the data to the FDA to get clearance for Phase 1 clinical trials in the US.
HuidaGene is working on a broad gene editing toolkit, from Cas12 enzymes for DNA cutting to Cas13 for RNA knockdown to tools for base editing and epigenetic editing.
“We can choose which tool is the best for the disease,” Luk said. It was a major factor in his decision to join the company as CEO in 2023. |
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John Carroll on biotech’s Q1 |
Endpoints’ founding editor John Carroll examined biotech’s first-quarter performance this week, pulling in the numbers and charts from Chris Dokomajilar at DealForma. Carroll notes that despite the XBI falling 27% since Trump won the November election, a massive FDA reorganization and broader HHS layoffs, there are some positive signs under the hood.
Most importantly, dealmaking activity remains active, even though IPOs and PIPEs remain cool. Mid- and late-stage assets continue to make attractive takeout options for bigger companies, while licensing deals and other partnerships are holding steady. But a big question remains: Can these trends hold up for the rest of the year as uncertainty lingers from Trump’s tariffs? |
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New fund to pressure biotech "zombies" |
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The number of barely surviving biotechs has reached a crisis level for some investors, and a handful of individuals are looking to do something about it. Nicholas Johnston, a longtime healthcare banker at JP Morgan and Perella Weinberg Partners, recently launched Alis Biosciences, which wants to work with C-suites and boards to make better use of their cash. They estimate over $30 billion in “trapped” capital on the books of an estimated 300 publicly traded biotechs. |
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