Ionis Pharmaceuticals IONS announced that it has dosed the first patient in the phase I/II ASCEND study, which is evaluating its investigational RNA-targeted medicine, ION337, for treating patients with Dravet syndrome, a rare and severe neurological disorder.
ION337 is the first wholly owned therapy developed using Ionis' next-generation N-Methylacetamide (NMA) technology, designed to enhance potency and support infrequent intrathecal dosing.
Management believes that initiating dosing in the ASCEND study represents an important milestone in the development of a potential disease-modifying treatment for Dravet syndrome.
The open-label ASCEND study will assess the safety and tolerability of ION337 for the treatment of Dravet syndrome in children aged two to 12 years.
The study consists of two parts: an initial 6-month single ascending dose phase, followed by a 24-month multiple ascending dose phase in which ION337 is administered every six months. The study also includes an additional seven-month safety follow-up period.
IONS Price Performance
Year to date, Ionis's shares have plunged 28.3% compared with the industry’s decline of 1.5%.
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IONS’ Wholly-Owned Products Hold Promise
Ionis' collaborations with leading drugmakers and biotech companies provide a steady source of funding through license fees, upfront payments and milestone payments, helping advance its internal pipeline.
Ionis has two wholly-owned marketed medications — Tryngolza for severe hypertriglyceridemia and familial chylomicronemia syndrome, as well as Dawnzera for hereditary angioedema. The two drugs are also the company’s first independent product launches.
Some of Ionis’ wholly-owned candidates include zilganersen and obudanersen (formerly ION582), which are being evaluated in late-stage studies for treating Alexander disease (AxD) and Angelman Syndrome, respectively.
A regulatory filing seeking approval for zilganersen in AxD is currently under review in the United States, with a decision from the FDA expected on Sept. 22, 2026.
Other Players in the Dravet Syndrome Market
We also note that other companies are developing late-stage treatments for Dravet syndrome. Stoke Therapeutics STOK is evaluating its lead investigational candidate, zorevunersen, in late-stage studies for the indication.
The company believes zorevunersen is a novel proprietary antisense oligonucleotide with the potential to become the first disease-modifying therapy to address the genetic cause of Dravet syndrome.
Stoke Therapeutics has a strategic collaboration with drug giant Biogen BIIB to develop and commercialize zorevunersen.
In February 2025, Biogen in-licensed the rights to the candidate outside the United States, further expanding its rare disease pipeline.
Although ION337 is still in the early stages of development, the successful development and potential approval of zorevunersen could create competitive pressure for Ionis' candidate.
IONS’ Zacks Rank
Ionis currently has a Zacks Rank #4 (Sell).
You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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This article originally published on Zacks Investment Research (zacks.com).