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The search for a cure for Duchenne muscular dystrophy, a muscle-wasting degenerative disease, has been plagued by setbacks and controversy.
Last week, Pfizer The program was abolished DMD treatment drug Syfreo We’re screwed This is phase 3.
Sarepta Therapeutics’ drug, Erebidis, First approved gene therapy For this symptom, $334 million FDA approves in June 2023, recently Significant label expansionBut it is also Controversy For example, disappointing test data. question About the effectiveness of treatment The price is $3.2 million Per injection.
Linda Malvan, CEO of Capricor Therapeutics
Used with permission from Carpricor
But there is a silver lining to this turmoil. California-based biotech company Capricorn Therapeutics Recently Announced Capricor CEO Linda Malvan said the company’s investigational drug, Delamiocel (CAP-1002), slowed the progression of Duchenne muscular dystrophy by nearly 50% in patients treated for three years as part of the ongoing Phase 2 HOPE-2 open-label extension study. In particular, the drug improved heart function in patients who developed a form of cardiomyopathy for which there is currently no approved treatment and which is often fatal. Patients in the trial saw an overall improvement in ejection fraction, the gold standard for heart function, she said. And those whose hearts were healthiest at the start of the trial saw the most pronounced improvements.
Challenges of DMD
a Genetic mutation DMD is caused by a genetic mutation that causes a deficiency of the muscle-protecting protein dystrophin. Because the mutation is on the X chromosome, the disease affects mostly boys, who begin to show slower motor development and decline in athletic ability than their peers. 2nd or 3rd birthdayThe disease is progressive and often causes fatal cardiac and respiratory problems.
The FDA has approved drugs to treat DMD since 2016, and the market size is expected to grow by $18 billion by 2030Many of these are exon-skipping drugs, which help bridge the faulty section of genetic code and restore some production of the dystrophin protein.
Regenxbio is also testing a gene therapy, RGX-202, and recently saw positive results in that trial. Ongoing Phase 1/2 In clinical trials, the treatment appears to promote expression of a shorter but similar form of dystrophin called micro-dystrophin. Some treated patients saw up to a 77% increase in expression compared to controls after three months. However, it is unclear whether this increase translates into improved function. The company plans to release strength and function assessment data in the second half of 2024.
Slowing progression
Unlike many of these treatments, Capricol’s homologous cell therapy does not target the underlying causes of DMD, but aims to slow the progression of the disease. The therapy modulates the immune system to reduce inflammation, reduce fibrosis, and aid in cell replication and repair of damaged tissue. It may also complement other treatments and offer an advantage over steroids, as it appears to have few serious side effects and could potentially be used as a long-term treatment.
“One of the best things about this treatment is its safety,” she said.
Even after years of treatment, most patients experience mild symptoms such as body aches and headaches after the infusion. Malvan said the treatment, which is expected to cost around $1 million a year, similar to exon-skipping drugs, could help keep patients healthier for longer.
“We’re treating patients who are already on maximum doses of steroids, and we’re seeing more benefit, more preservation of function — the ability to maintain and perform tasks that they’d lost or were about to lose,” she said. “And that’s because with the breakdown of proteins from cell destruction, we need something to dampen inflammation and prevent fibrosis.”
The company is currently awaiting data from a Phase 3 clinical trial. Allogeneic cell therapyThat’s expected at the end of 2024. If all goes as planned, company executives could file a license application for the biologic by mid-2025, Malvan said. Capricor is also studying similar diseases in which the treatment might be effective, Malvan said.
“We’re looking very closely at Becker muscular dystrophy as our next target,” she said.
The company’s second-generation DMD product, CAP-2003, which Marban hopes will produce better results than current technologies, is currently in preclinical development.
