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Hello! Fighting back a hyper 6-year-old and his 3-year-old partner in crime as I attempt to write an intro here. My offspring say hello.
Today, we discuss the innovative way a mother is attempting to raise money for a gene therapy for her child, we see South African regulators drop their investigation into Vertex’s cystic fibrosis drug Trikafta, and more.
The need-to-know this morning
- Edgewise Therapeutics said an experimental drug designed to prevent muscle damage achieved the primary goal of a study involving participants with Duchenne muscular dystrophy, a rare neuromuscular disorder. The Edgewise drug significantly reduced a blood-based biomarker for muscle loss compared to placebo. Muscle-function tests showed improvements, although the results were not statistically significant.
- Neurocrine Biosciences won FDA approval for a drug to treat classic congenital adrenal hyperplasia, a rare disease involving the adrenal glands. The drug will be marketed under the brand name Crenessity.
- Abbvie acquired privately held Nimble Therapeutics for $200 million. Nimble is developing drugs for autoimmune diseases.
As the drug industry’s interest in gene therapy fades, one family takes an unusual approach
Gene therapy, in theory, could be harnessed to treat all kinds of rare diseases. But too often those conditions are so rare that they fail to capture the interest of the drug industry.
Amber Freed, a mother whose 7-year-old son has a rare genetic condition and might benefit from gene therapy, has come up with a creative solution: She hopes to raise $1 million to fund the manufacturing of a treatment as well as early trials — and is offering to name the treatment after the highest-bidding donor.
“If we are able to name the treatment after a funder, it’s truly a way to honor a person’s name forever,” said Freed, who was a financial analyst before she quit her job to chase a cure for the ultra-rare disease Maxwell has, SLC6A1. “You’re going to be saving lives and helping kids and a name can forever be associated with grit and perseverance.”
Viridian confirms benefit of drug for thyroid eye disease
An experimental treatment from Viridian Therapeutics improved the signs and symptoms of chronic thyroid eye disease — achieving the goals of a second Phase 3 clinical trial, the company reported today.
In the 15-week study, 56% of participants responded to treatment with Viridian’s drug, called veligrotug, compared to 8% in the placebo group. Response was defined as a meaningful reduction in eye bulging, a hallmark sign of thyroid eye disease. Hearing impairment, a closely watched side effect, was reported by 13% of the participants treated with veligrotug compared to 3% in the placebo arm.
In September, veligrotug, formerly known as VRDN-001, achieved the goals of a Phase 3 study in participants with active thyroid eye disease. Viridian will include the results of both studies in its marketing application to the FDA, expected in the second half of 2025.
Veligrotug is an antibody that targets insulin-like growth factor-1 receptor. It is based on the same mechanism as Amgen’s Tepezza, which was approved for thyroid eye disease in 2020 and is expected to generate nearly $1 billion in sales this year.
FTC declines to challenge Novo-Catalent deal
Novo Holdings will finalize its $16.5 billion acquisition of the contract drug manufacturer Catalent in the coming days, after months of scrutiny from regulators, including the FTC.
The acquisition was prompted by sporadic shortages of one of the world’s hottest-selling medications — Novo Nordisk’s weight loss treatment Wegovy — and designed, in part, to solve what has been a critical and seemingly intractable problem for Novo Nordisk.
But the prospect that Novo Holdings might soon control one of the biggest players in pharmaceutical contract work prompted speculation that the FTC would object on anti-competitive grounds.
South Africa: Vertex has provided sufficient access to cystic fibrosis treatment
South African regulators have concluded that Vertex Pharmaceuticals is offering sufficient access to its cystic fibrosis treatment Trikafta. Its conclusion ends a high-profile investigation into the company’s dealings there — even as advocacy groups insist more should be done.
Vertex has made Trikafta accessible via private health plans and a patient assistance program, but critics say that only patients with premium medical coverage can access it.
More reads
- Ozempic link to rare vision loss risk confirmed in study, Bloomberg