RARE Daily

Ionis Drops Development of Two Rare Disease Programs

May 4, 2023

Rare Daily Staff

Ionis Pharmaceuticals is cutting two rare disease programs, cimdelirsen for acromegaly and sapablursen for beta-thalassemia, as the company continues to focus its R&D pipeline, according to the company’s first quarter 2023 financial report.

Although both programs had a favorable safety and tolerability, efficacy results did not meet Ionis’ minimum target product profile, the company said in an analyst call. However, Ionis will continue to advance the phase 2 sapablursen study for polycythemia vera.

“2023 is off to a strong start. With Qalsody’s approval, it joins Spinraza as a new groundbreaking medicine to treat a devastating neurological disease, further validating our RNA-targeting therapeutic platform. We also achieved another important milestone with our recent positive eplontersen phase 3 data. We believe the positive efficacy and safety data, and the attractive self-administered dosing profile, position eplontersen to be an important treatment for ATTRv-PN patients, who today are underserved. We look forward to the first potential approval of eplontersen in the U.S. in December,” said Brett Monia, CEO of Ionis.

Photo: Brett Monia, CEO of Ionis Pharmaceuticals

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