Ionis Pharmaceuticals has announced plans to submit a new drug application (NDA) to the US Food and Drug Administration (FDA) after the Phase III OASIS-HAE study (NCT05139810) hereditary angioedema (HAE) study with donidalorsen met its primary endpoint.  

The trial successfully met its primary endpoint, demonstrating a reduction in angioedema attacks in patients receiving donidalorsen injections every four or eight weeks, compared to a placebo. In the 91-patient cohort, participants were randomly assigned in a 2:1 ratio to receive either the antisense drug donidalorsen  or a placebo.  Secondary endpoints were also achieved with donidalorsen showing a favourable safety profile with no adverse events reported. 

Ionis is preparing an NDA for the US, whilst its Japan-based commercialisation partner Otsuka plans to submit a marketing authorisation application in Europe. Under the terms of the deal, Otsuka made a $65m upfront payment and will make milestone payments to Ionis based on sales and regulatory goals. 

HAE is a rare genetic disorder characterised by recurrent and severe swelling in various body parts. The condition is caused by a deficiency or dysfunction in the C1 inhibitor protein, leading to an overproduction of bradykinin, a substance causing blood vessels to leak fluid.  Donidalorsen directly targets prekallikrein, a precursor to the kallikrein enzyme, which plays a role in inflammation, disrupting the pathway that triggers HAE attacks. 

According to GlobalData’s Pharma Intelligence Center, donidalorsen is forecast to generate $332m in sales in 2029. 

GlobalData is the parent company of Clinical Trials Arena. 

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By GlobalData

Ionis is also further investigating the therapy’s long term effects in an open-label extension study called OASIS-Plus (NCT04307381). Results from the study, expected later this year, will cover a distinct switch-cohort group of patients transitioning to donidalorsen from a different prophylactic medication.  

In the announcement accompanying the data, senior vice president at Ionis Kenneth Newman said: “Based on these results and the durable efficacy and favourable safety data seen in the ongoing Phase 2 open-label extension study, we believe donidalorsen, if approved, could be an attractive new treatment option for patients with HAE.” 

This marks another positive Phase III data readout for Ionis over the past year. In December 2023, Ionis and AstraZeneca gained FDA approval for their jointly developed rare neurodegenerative disease treatment Wainua (eplontersen). Ionis also teamed up with Novartis last year to develop a therapy for cardiovascular disease.