Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has announced its third quarter 2024 financial results, highlighting significant advancements in its pipeline and commercial operations. The company is on track to achieve its 2024 financial guidance, with increased cash reserves reflecting proceeds from a recent equity offering.
WAINUA Achieves Commercial and Regulatory Success
WAINUA (eplontersen), for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN), has achieved multiple commercial and regulatory milestones. In the third quarter of 2024, WAINUA generated sales of $23 million, resulting in royalty revenue of $5 million. The drug received a positive Committee for Medicinal Products for Human Use (CHMP) opinion from the European Medicines Agency (EMA) and was approved in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA).
Olezarsen Poised for Potential Approval
Olezarsen is positioned to potentially treat two patient populations with urgent unmet needs: familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG). The New Drug Application (NDA) for patients with FCS is under FDA Priority Review with a PDUFA date of December 19, 2024. The Marketing Authorization Application (MAA) is also under regulatory review by the EMA. The ongoing pivotal development program for sHTG is on track for data in the second half of 2025.
Donidalorsen Advances as Prophylactic HAE Treatment
Donidalorsen's Phase 3 data positions it to potentially launch as the first RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE). The FDA has accepted the NDA for patients with HAE with a PDUFA date of August 21, 2025. Positive Phase 2 open label extension (OLE) study data were presented in patients treated up to three years with every four weeks or every eight weeks dosing.
Pipeline Expansion and Strategic Investments
Ionis is advancing its pipeline with several key programs. Zilganersen, as a potential treatment for Alexander disease, has fully enrolled its Phase 3 study, with data expected in 2025. ION582, for Angelman syndrome (AS), achieved important clinical and regulatory milestones, enabling the initiation of Phase 3 development in the first half of 2025. Positive data from the Phase 2 study of IONIS-FB-LRx in patients with immunoglobulin A nephropathy (IgAN) were reported, with Roche continuing to advance IONIS-FB-LRx in the Phase 3 IMAGINATION study.
"This year has been marked by strong delivery on our pipeline and business goals, which position Ionis to deliver on our vision of bringing a steady cadence of innovative medicines to patients in need," said Elizabeth L. Hougen, chief financial officer of Ionis. "Fully realizing these significant opportunities requires substantial investment. As a result, we recently executed an equity offering that extends our cash runway, enabling us to continue to invest in the numerous attractive opportunities ahead of us, including our near-term commercial launches with multi-billion-dollar revenue potential and our rich late and mid-stage pipeline."
