Ionis' Rare Development Disorder Drug Candidate 'Could Be Differentiated' From Peer Ultragenyx: Analyst

Ionis Pharmaceuticals Inc IONS released results Monday from the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582 in people with Angelman syndrome (AS).

AS is a rare neurodevelopmental disorder caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide.

The data demonstrated consistent and encouraging clinical improvement on assessing all functional domains, including communication, cognition, and motor function measures.

Also Read: Ionis Pharmaceuticals Reveals Late-Stage Donidalorsen Data, Analyst Says Results Show Competitive Hereditary Angioedema Profile.

Overall, 97% of people in the medium and high dose groups saw an improvement in overall AS symptoms.

In the 51-patient HALOS study, ION582 showed favorable safety and tolerability at all dose levels.

Ionis plans to meet with regulators later this year to review and confirm its Phase 3 study design. This puts the company on track for a pivotal study initiation in the first half of 2025. 

William Blair writes that the update is a clear positive for Ionis, positioning ION582 as a key asset in its wholly owned portfolio after recently reacquiring sole rights from Biogen Inc BIIB.

William Blair reiterates the Outperform rating on Ionis.

The analyst notes that the consistent improvements across various metrics in the robust dataset, which will likely be used as a primary endpoint for approval, boost confidence in the drug’s potential benefits.

While slightly behind a peer program from Ultragenyx Pharmaceutical Inc RARE, Ionis has largely closed the development timeline gap and could be differentiated from a safety perspective, making ION582 an attractive asset for future growth given lack of approved therapies for the indication currently.

In the meantime, investors are expected to focus on the launch of Wainua (eplontersen) in collaboration with AstraZeneca Plc AZN, and the potential approval and launch of the wholly-owned therapies olezarsen for familial chylomicronemia syndrome (PDUFA December 19, 2024) and donidalorsen for the prophylactic treatment of HAE (NDA expected to be filed this year).

The analyst believes the next 12 to 18 months could be transformative for Ionis due to the expansion of its marketed products. They also consider the company undervalued, especially in light of the potential of TTR silencers for TTR-cardiomyopathy, following Alnylam Pharmaceuticals Inc’s ALNY positive HELIOS-B trial results for vutrisiran.

Price Action: IONS stock is up 4.50%, trading at $49.97 at last check Monday.

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