Ionis (IONS) Inks Deal to Develop Precision Genetic Medicines

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Ionis Pharmaceuticals, Inc. IONS announced that it has entered into a collaboration with a California-based privately-held gene editing company, Metagenomi, to add gene editing to its broad technology platform.

With this partnership, the companies are looking to leverage Ionis' expertise in RNA-targeted therapeutics with Metagenomi's next-generation gene editing systems to develop novel genetic targets.

Initially, the companies will jointly research and develop investigational medicines for up to four genetic targets, with IONS reserving the right to add four additional targets upon achieving certain pre-determined development milestones.

Per the agreement, Ionis will make an upfront payment of $80 million to Metagenomi along with potential future milestone payments and royalties

The collaboration looks like a good strategic fit for Ionis as it is likely to help the company to diversify its broad technology platform of RNA-targeted therapeutics and deliver precision genetic medicines with gene-editing technology.

Shares of Ionis have rallied 44.8% so far this year against the industry’s decline of 29.5%.

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We note that Ionis’ RNA antisense technology has allowed the company to form an impressive set of partnerships with leading drugmakers for the development and marketing of its drugs.

Ionis has collaboration deals with pharma giants such as Biogen BIIB, AstraZeneca AZN and Novartis NVS.

Ionis earns commercial revenues, primarily royalty payments on net sales of Biogen's Spinraza and R&D revenues from partnered medicines.

Biogen is responsible for commercializing Spinraza, which is approved for treating spinal muscular atrophy worldwide. Ionis receives royalties from BIIB on Spinraza’s sales. The companies are also developing several other candidates, including tofersen for treating SOD1-ALS (amyotrophic lateral sclerosis) and other neurological diseases.

Meanwhile, AstraZeneca and Novartis are Ionis’ partners for pipeline candidates eplontersen and pelacarsen.

Ionis and AZN will file a new drug application seeking approval of eplontersen for TTR polyneuropathy later in 2022. Ionis and AstraZeneca are also developing eplontersen for the treatment of cardiomyopathy in the phase III CARDIO-TTRansform study.

Ionis and Novartis are evaluating pelacarsen in the ongoing phase III cardiovascular outcome study, HORIZON, in patients with established cardiovascular disease and elevated lipoprotein(a), or Lp(a).

Zacks Rank

Ionis currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.


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