Ionis announces Q3 2024 financial results

Ionis Pharmaceuticals, Inc. recently released its financial results for the third quarter of 2024, highlighting the company's ongoing efforts in drug development and commercialization. CEO Brett P. Monia emphasized that Ionis is on the brink of significant milestones, including the co-commercialization launch of WAINUA and the anticipated independent launch of olezarsen for treating familial chylomicronemia syndrome (FCS), a rare condition lacking approved treatments in the U.S., scheduled for an FDA action date in December 2024. Additionally, donidalorsen for hereditary angioedema (HAE) is poised for FDA review by August 2025.

Ionis' pipeline remains robust, with Phase 3 results expected next year for olezarsen’s second indication in severe hypertriglyceridemia and pelacarsen in cardiovascular disease driven by Lp(a). Furthermore, Phase 3 data for eplontersen’s secondary indication in ATTR cardiomyopathy is anticipated by the second half of 2026. The company is also advancing ION582 for Angelman syndrome, projecting Phase 3 development by the first half of next year after positive Phase 2 discussions with the FDA.

Financially, Ionis noted a 7% decrease in revenue for the three months ended September 30, 2024, compared to the same period last year, though revenue for the nine months increased by 3%. This variation is attributed to a diverse revenue stream, including royalties from WAINUA’s launch in the U.S. in early 2024. Operating expenses rose as planned, reflecting investments in late-stage developments and commercialization efforts for WAINUA, olezarsen, and donidalorsen.

WAINUA has achieved several milestones, including generating significant sales and receiving positive regulatory opinions in Europe and the UK. Commercial efforts for this treatment are also underway in Canada following its approval and reimbursement. SPINRAZA (nusinersen) for spinal muscular atrophy generated substantial global sales, contributing significantly to Ionis' royalty revenue. QALSODY (tofersen) was approved in China for treating SOD1-ALS, further expanding Ionis' market footprint.

Olezarsen is targeted at two patient groups with unmet needs—FCS and severe hypertriglyceridemia (sHTG). The FDA has given priority review to the New Drug Application (NDA) for FCS, with a decision expected in December 2024. Regulatory review by the EMA is also ongoing. The sHTG program is on track to provide pivotal data by the second half of 2025.

Donidalorsen is positioned as a potential first RNA-targeted prophylactic treatment for people with HAE. The FDA accepted the NDA with an action date set for August 2025. Positive Phase 2 study data have been presented, reinforcing the drug’s potential efficacy. Zilganersen for Alexander disease is fully enrolled in a Phase 3 study, with data anticipated in 2025, and has received Fast Track designation by the FDA.

ION582 is advancing towards Phase 3 development for Angelman syndrome in the first half of 2025, following positive Phase 2 results and discussions with the FDA. Additionally, Ionis reported favorable Phase 2 data for IONIS-FB-LRx in treating immunoglobulin A nephropathy (IgAN), with Roche advancing this treatment in Phase 3 trials.

Other pipeline advancements include sapablursen for polycythemia vera, which has received orphan drug designation and completed Phase 2 enrollment, and IONIS-MAPTRx (BIIB080) for early Alzheimer's disease, with Phase 2 data expected in 2026. Several new neurological disease treatments have entered early human trials, including ION356 for Pelizaeus-Merzbacher disease and ION269 for Alzheimer's disease in patients with Down syndrome.

Financially, Ionis has strengthened its balance sheet, ending September 2024 with $2.5 billion in cash and short-term investments, following an equity offering that raised $500 million. The company plans to continue investing in its growth opportunities, including pre-commercialization activities and advancing its pipeline of innovative medicines.

CFO Elizabeth L. Hougen highlighted the company’s strategic investments to prepare for upcoming product launches and scale its capabilities in line with the potential of its key medicines. Ionis is focused on sustainable growth, supported by its rich pipeline and recent achievements, positioning the company to deliver new treatments for serious diseases.