👩‍🍼Hazel Marquart was born with cystic fibrosis, but with none of the disease’s symptoms. Her mother, Stephanie Marquart, started taking Vertex’s therapy Trikafta while she was pregnant, treating her baby in the womb. Endpoints’ senior biopharma correspondent Andrew Dunn looked at the new gap in care created by the success of drugs like Trikafta, and how the Marquarts took matters into their own hands. 

Such treatments have changed cystic fibrosis from a death sentence to a manageable chronic condition for the vast majority of patients. But Trikafta is only approved for children aged 2 and up, even though the disease’s damage begins in the womb. Stephanie didn’t want to wait two years to begin her unborn daughter’s treatment. An adult pulmonologist wrote Stephanie a prescription to take the drug during pregnancy, but insurance wouldn’t cover it at first. At the time, Trikafta cost roughly $27,000 for a month’s dosage. Stephanie sourced her first dose via Facebook. 

Vertex is not running a clinical trial to test the drug during pregnancy. It has also refused to provide free drug to physicians who’d like to study it in this setting to generate quality data to guide care decisions. Meanwhile, Scott Sagel, a pediatric pulmonologist at Children’s Hospital Colorado, is seeing more and more babies like Hazel. After two and a half years, the toddler still has no complications from the disease. “We are now getting contacted by families on an almost monthly basis,” Sagel told Endpoints. “It is a new frontier for us.”

Sagel is running a prospective, observational study, asking moms who inquire about fetal treatment to enroll, whether or not they actually take any therapy. The Cystic Fibrosis Foundation is also funding an observational study and planning another. Sagel hopes collecting those cases will be helpful, but he doesn’t believe it’s sufficient. Rigorous, controlled clinical trials remain absent. You can read Andrew’s full story here for more details on the Marquarts’ journey and the lack of information about medicine use during pregnancy.

🎤Amid an attempted culture reboot, Novo CEO Mike Doustdar spelled out his strategic vision to investors at the company’s capital markets day in London this week. Top of mind for Doustdar: The Danish drugmaker needs to diversify. Doustdar unveiled plans to start clinical trials of drugs with new mechanisms and pursue bolt-on deals, moves that observers have desired for a while now. But perhaps some were hoping for bigger swings, as Novo’s stock price fell about 5% in response to the event. And this week's modest drug delivery tie-up with Nanexa might have trouble moving the needle.

Novo’s biggest need is to not rely so significantly on semaglutide, the GLP-1 drug that makes up the company’s Ozempic, Wegovy and Rybelsus franchises. Roughly in the 2030 time frame, the company aims to launch five “multi-blockbusters” and bring its two triple agonists to market. On the dealmaking front, Novo will continue to pursue bolt-on deals along the lines of its Akero Therapeutics and Forma Therapeutics acquisitions. 

Key to Doustdar’s goals will be Novo’s ability to build confidence among investors, and Doustdar knows this. Make sure to keep following Elizabeth’s coverage as Novo charts this course.

👁️Merck reported a Phase 2b/3 win this week for a program in its deal with EyeBio. An experimental drug called remigromig hit the mark in its first pivotal trial in patients with diabetic macular edema, proving non-inferior to Lucentis after one year. The success represents Merck’s first win in ophthalmology since the company exited the space in 2014. 

Merck and EyeBio teamed up in 2024, with Merck spending $1.3 billion upfront and promising another $1.7 billion if all milestones are reached. The deal is part of Merck’s push to stave off the steep revenue declines that could happen as soon as 2028 when Keytruda loses exclusivity. Remigromig is in another DME pivotal trial, as well as a Phase 2 test in other eye disorders. There’s also another asset from EyeBio named MK-8748.