Ionis Pharmaceuticals is making significant strides across its pipeline, highlighted by the U.S. launch of WAINUA and upcoming regulatory decisions for olezarsen and donidalorsen. These advancements, coupled with ongoing Phase 3 trials, position Ionis to deliver a steady stream of transformational medicines for serious diseases. The company's third-quarter 2024 financial results reflect continued investment in late-stage development and commercialization efforts.
WAINUA Achieves Commercial and Regulatory Milestones
WAINUA (eplontersen), indicated for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN), has achieved several milestones. Sales of WAINUA generated $23 million in the third quarter and $44 million in the first nine months of 2024, resulting in royalty revenue of $5 million and $10 million, respectively. The European Medicines Agency (EMA) issued a positive Committee for Medicinal Products for Human Use (CHMP) opinion, and the drug was approved in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA) with an accelerated National Institute for Health and Care Excellence (NICE) recommendation, earning $30 million from AstraZeneca. Launch activities are also underway in Canada, following approval and reimbursement from Health Canada.
Olezarsen Poised to Address Unmet Needs in FCS and sHTG
Olezarsen is being developed to treat familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG). The New Drug Application (NDA) for FCS is under FDA Priority Review, with a PDUFA date of December 19, 2024. A marketing authorization application (MAA) is also under regulatory review by the EMA. The pivotal development program for sHTG is on track for data in the second half of 2025.
Donidalorsen Advances as Potential HAE Prophylactic Treatment
Donidalorsen is being developed as a prophylactic treatment for hereditary angioedema (HAE). The FDA 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, showing efficacy in patients treated for up to three years with every four weeks or every eight weeks dosing.
Progress in Other Pipeline Programs
Zilganersen, a potential treatment for Alexander disease, has fully enrolled its Phase 3 study, with data expected in 2025. ION582 for Angelman syndrome (AS) achieved clinical and regulatory milestones, enabling the initiation of Phase 3 development in the first half of 2025, following positive Phase 1/2 data and alignment with the FDA on Phase 3 design. Additionally, 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 the drug in the Phase 3 IMAGINATION study.
Financial Position and Outlook
Ionis reported a decrease in revenue for the three months ended September 30, 2024, compared to the same period last year, but an increase for the nine months ended September 30, 2024. This includes royalty revenue from the launch of WAINUA in the U.S. Operating expenses increased due to investments in late-stage development and commercialization efforts. As of September 30, 2024, Ionis' cash, cash equivalents, and short-term investments increased to $2.5 billion. The company reaffirmed its 2024 P&L financial guidance and increased its cash guidance to $2.2 billion, reflecting proceeds from an equity offering.
