First Patient Dosed In Phase 3 REVEAL Study Of ION582 For Angelman Syndrome

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) is among the 11 Best Genomics Stocks to Buy According to Hedge Funds. The initial participant in the pivotal Phase 3 REVEAL trial, which is assessing ION582 for Angelman syndrome, a severe neurodevelopmental disease, has been dosed by Ionis Pharmaceuticals, Inc. (NASDAQ:IONS).

A scientist in a laboratory making a breakthrough discovery in biotechnology.

The randomized, placebo-controlled research will include approximately 200 people with UBE3A gene deletions or mutations. The main goal of the 52-week trial will be expressive communication, and quarterly 40 mg or 80 mg dosages of ION582 will be evaluated. The trial intends to finish enrollment in 2026 and is based on positive Phase 1/2 HALOS results.’

ION582 is an antisense RNA treatment that inhibits UBE3A-ATS to promote UBE3A protein. The FDA has designated it as a Rare Pediatric Disease, Fast Track, and Orphan Drug. Globally, 1 in 21,000 individuals suffer from Angelman syndrome, which results in severe physical and cognitive impairments.

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), which developed SPINRAZA and QALSODY, is expanding its neurology portfolio, with eight wholly owned treatments in development for rare and common disorders, including Alzheimer’s and Parkinson’s disease.

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