Ionis Drops Development of Two Rare Disease Programs

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