Ionis plans next steps for Angelman drug rejected by Biogen

Ionis Pharmaceuticals is advancing its rare disease drug for Angelman syndrome into late-stage testing following positive outcomes from a smaller preliminary trial. Angelman syndrome is a genetic disorder characterized by severe learning and functional disabilities. The trial involved 51 participants who received one of three doses of the drug, ION582. The company reported that the treatment was generally safe, well-tolerated, and provided significant benefits in communication, cognition, and motor functions.

Researchers evaluated the effects of ION582 using four different scoring systems. One of the scoring methods revealed a 97% improvement in Angelman symptoms among participants who received medium to high doses. Another method showed that enhancements in cognition, communication, and motor skills surpassed those documented in natural history studies of similar Angelman patients.

With these promising results, Ionis plans to discuss the design of a pivotal Phase 3 trial with drug regulators. The company aims to commence this critical trial phase in the first half of the next year. Following the announcement, Ionis' stock saw a more than 4% increase, trading just below $50 per share. Over the past year, the stock has risen nearly 23%.

Ionis Pharmaceuticals specializes in antisense oligonucleotides, a type of medication that modifies the genetic instructions used to produce proteins. The company has already introduced five antisense therapies to the market, including two developed in collaboration with Biogen. Interestingly, Biogen recently decided not to license ION582.

Ionis is now positioning ION582 as a significant addition to its portfolio of unpartnered products. CEO Brett Monia stated that the drug is poised to become a cornerstone of Ionis' next wave of transformative, wholly owned treatments for neurological disorders. According to the company, approximately 1 in every 21,000 people is affected by Angelman syndrome. The disorder results from the malfunction of a gene on the maternal chromosome, which disrupts the production of an essential enzyme involved in protein degradation and nervous system development.

ION582 aims to increase enzyme production by binding to RNA molecules that control the gene's expression on the paternal chromosome. Although this gene is usually inactive in the brain, Ionis' drug is designed to reactivate it. William Blair analyst Myles Minter commented that the newly released data is a "clear positive" for Ionis. The company’s Phase 3 plans bring it close to Ultragenyx Pharmaceutical, which is also developing a similar therapy expected to enter late-stage testing this year.

Minter noted that although Ionis is slightly behind Ultragenyx in development, it has significantly narrowed the timeline gap. He suggested that ION582 could stand out in terms of safety, making it an appealing asset for future growth, especially given the current lack of approved therapies for Angelman syndrome.

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