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.
Commercial Progress and Regulatory Milestones
Wainua (eplontersen), indicated for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN), has achieved key commercial and regulatory 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 for Wainua, 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 underway in Canada following approval and reimbursement from Health Canada.
Spinraza (nusinersen), for the treatment of spinal muscular atrophy (SMA), generated global sales of $381 million and $1.2 billion, resulting in royalty revenue of $57 million and $152 million in the three and nine months ended September 30, 2024, respectively. Positive Phase 2/3 DEVOTE study data from higher dose nusinersen were presented, with global regulatory applications planned.
Qalsody (tofersen) received marketing approval in China for the treatment of SOD1-ALS.
Late-Stage Pipeline Developments
Olezarsen is being developed to potentially treat familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG). The New Drug Application (NDA) for FCS patients is under FDA Priority Review with a PDUFA date of December 19, 2024. The Marketing Authorization Application (MAA) is under regulatory review by the EMA. The pivotal development program for sHTG is on track for data in the second half of 2025.
Donidalorsen's Phase 3 data positions it as a potential first RNA-targeted prophylactic treatment for hereditary angioedema (HAE). The FDA accepted the NDA for HAE patients with a PDUFA date of August 21, 2025. Positive Phase 2 open-label extension (OLE) study data were presented, showing patients treated for up to three years with dosing every four or eight weeks.
Zilganersen's Phase 3 study is fully enrolled as a potential treatment for Alexander disease, with data expected in 2025. The FDA granted Fast Track designation to the program.
ION582 achieved clinical and regulatory milestones, enabling the initiation of Phase 3 development in Angelman syndrome (AS) in the first half of 2025. Positive Phase 1/2 data in AS patients were presented at the Angelman Syndrome Foundation (ASF) Family Conference, and a positive End-of-Phase 2 discussion with the FDA was completed, including alignment on the Phase 3 design.
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.
Additional Pipeline Updates
Sapablursen for polycythemia vera was granted orphan drug designation by the FDA, and enrollment is complete in the Phase 2 IMPRSSION study, with data expected in 2025.
IONIS-MAPTRx (BIIB080) enrollment is complete in the Phase 2 CELIA study in patients with early Alzheimer's disease (AD), with data expected in 2026.
First-in-human studies were initiated with multiple medicines from the neurological disease pipeline, including ION356 (PLP1) for Pelizaeus-Merzbacher disease (PMD), ION269 (APP) for Alzheimer's disease (AD) in patients with Down syndrome (DS), and ION440 (MECP2) in patients with MECP2 duplication syndrome.
Financial Position
As of September 30, 2024, Ionis' cash, cash equivalents, and short-term investments increased to $2.5 billion, compared to $2.3 billion at December 31, 2023. The company reaffirmed its 2024 P&L financial guidance and increased its cash guidance to $2.2 billion, reflecting proceeds from an equity offering. Ionis plans to continue deploying its capital resources toward growth opportunities, focusing on its wholly-owned late-stage and next wave of innovative medicines.
