Ionis Pharmaceuticals, a US-based developer of RNA-targeted therapies, has launched the OASIS-HAE phase 3 clinical trial for donidalorsen (formerly IONIS-PKK-LRx) in patients having hereditary angioedema (HAE).

Donidalorsen, which is an investigational antisense drug, is designed to lower the production of prekallikrein, which has a major role in triggering inflammatory mediators pertaining to acute attacks of hereditary angioedema.

The antisense drug candidate has been designed based on the LIgand-Conjugated Antisense (LICA) technology of Ionis Pharmaceuticals.

Kenneth Newman — Ionis Pharmaceuticals vice president of clinical development said: “Initiating the Phase 3 program for donidalorsen moves us one step closer to bringing a potential best-in-class prophylactic treatment to market for people with HAE globally experiencing recurrent painful and severe HAE attacks.

“Advancing this program underscores our commitment to the HAE patient community to deliver transformative treatments.”

OASIS-HAE is designed to be a double-blind, randomized, placebo-controlled clinical trial involving up to 84 hereditary angioedema patients, including those with Type 1 and Type 2.

Patients will be randomly grouped to be administered monthly or bi-monthly subcutaneous donidalorsen for a duration of 25 weeks. After the placebo-controlled portion of the study, patients could move into the 52-week open-label extension study.

Recently, Ionis Pharmaceuticals released data from a phase 2 clinical trial of donidalorsen, which showed that the drug candidate could deliver overall reduction in moderate to severe attacks, and in reducing the attacks starting with the second dose.

Ionis Pharmaceuticals said that for the last month of the mid-stage study, all the patients treated with donidalorsen were attack-free and the drug candidate was found to be safe and well tolerated.

  donidalorsen, hereditary angioedema, Ionis Pharmaceuticals, IONIS-PKK-LRx, Kenneth Newman, OASIS-HAE clinical trial, USA