Ionis Pharmaceuticals IONS signed a license agreement with Sweden-based Sobi. Per the terms, Sobi will acquire exclusive rights to market its wholly owned drug Tryngolza outside the United States (except Canada and China).

The FDA recently approved Tryngolzafor treating familial chylomicronemia syndrome (FCS), a rare genetic disease marked by extremely elevated triglyceride levels. This nod makes the drug the first approved treatment for FCS in the country. Italso marks Ionis’ first independent commercial launch. A regulatory filing for this FCS drug is currently under review by the EMA, with a final decision expected later this year.

Per the deal terms, Sobi will be responsible for all future regulatory filings and commercialization activities for the drug in ex-U.S. geographies except Canada (licensed to Theratechnologies) and China. Ionis will continue to market the drug in the United States independently.

In consideration of granting these rights, Ionis will receive an undisclosed upfront payment and be eligible for milestone payments. The company will also be entitled to receive a tiered royalty (up to the mid-20% range) on the drug’s annual net sales.

This new deal also marks the start of another partnership between the two companies. Sobi is currently Ionis’ commercialization partner in Europe for Waylivra, the only approved FCS drug in the region.

More on Ionis’ Tryngolza

Ionis is also evaluating the drug as a potential treatment for severe hypertriglyceridemia (sHTG) across three late-stage studies — CORE, CORE2 and ESSENCE.

While data from the ESSENCE study is expected in mid-2025, a readout from the CORE and CORE2 studies is expected in the second half of the year. Like FCS, Ionis also has a first-mover advantage in sHTG indication.

IONS Stock Performance

Year to date, shares of Ionis have lost 12% compared with the industry’s 4% decline.

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Ionis Boasts a Diverse Revenue Stream

Ionis has collaborations with leading drugmakers/biotech companies, such as AstraZeneca AZN, Biogen BIIB, GSK plc GSK and Novartis. These deals provide the company with funds in the form of license fees, upfront and milestone payments to invest in the development of its wholly owned pipeline.

Ionis earns commercial revenues in the form of royalty payments on net sales of Spinraza, approved in the United States to treat spinal muscular atrophy (SMA) worldwide. Ionis licensed this drug to Biogen, which is responsible for commercializing it. Ionis and Biogen also market Qalsody, which was approved by the FDA in April 2023 for amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations.

AstraZeneca, Novartis and GSK are its partners for Wainua, pelacarsen and bepirovirsen, respectively. The FDA approved Wainua in December 2023 to treat adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (ATTRv-PN) in the United States. The drug recently obtained approval in the EU for a similar indication and will be marketed under the name Wainzua.

While the GSK-partnered drug is being developed in two late-stage studies for chronic hepatitis B (CHB), the Novartis-partnered drug is being developed in a late-stage study for patients with cardiovascular disease due to elevated Lp(a) levels. Data from the Novartis and GSK-partnered drug studies are expected in 2025 and 2026, respectively.

Some of its other wholly owned candidates include donidalorsen (for hereditary angioedema), zilganersen (for Alexander’s disease) and ulefnersen (for ALS), which are being evaluated in late-stage studies. Ionis expects commercial launches for these drugs over the next three years, which could further lower its dependence on collaboration partners.

Ionis Pharmaceuticals, Inc. Price

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IONS’ Zacks Rank

Ionis currently carries a Zacks Rank #3 (Hold). 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).

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