Ionis Q2 2024 Financial Results

Ionis Pharmaceuticals, Inc., based in Carlsbad, California, released its financial results for the second quarter of 2024. The company highlighted significant advancements in its pipeline and market performance, aiming to provide treatments for serious diseases.

Brett P. Monia, Ph.D., Ionis' CEO, stated that the company successfully launched WAINUA in the U.S. for hereditary ATTR polyneuropathy (ATTRv-PN) and received approval for the drug in Canada. Additionally, QALSODY, a treatment for a genetic form of ALS, has been approved in the European Union (EU). The company is also preparing for its first independent launch of olezarsen, a treatment for familial chylomicronemia syndrome (FCS), with a U.S. FDA action date set for December 19, 2024.

Olezarsen has shown positive results in Phase 3 trials for severe hypertriglyceridemia (sHTG), with data expected in the second half of 2025. Donidalorsen, another key drug for hereditary angioedema (HAE), has also demonstrated promising Phase 3 results and is being prepared for regulatory submissions in the U.S. and EU.

Revenue for the second quarter and first half of 2024 increased by 20% and 8%, respectively, compared to the same periods last year. This growth was primarily driven by increased research and development (R&D) revenue, showcasing the value of Ionis' pipeline and technology. Operating expenses rose due to strategic investments in late-stage development and commercialization efforts for WAINUA, olezarsen, and donidalorsen.

WAINUA generated $16 million in sales in the second quarter and $21 million in the first half of 2024, contributing to royalty revenue of $4 million and $5 million, respectively. SPINRAZA, a treatment for spinal muscular atrophy (SMA), achieved global sales of $429 million in the second quarter and $770 million in the first half of 2024, resulting in royalty revenue of $57 million and $95 million, respectively. QALSODY received marketing approval in the EU for SOD1-ALS.

Ionis' CFO, Elizabeth L. Hougen, emphasized the company's plans to invest strategically in its promising near- and longer-term portfolio. This includes preparations for the launches of olezarsen and donidalorsen and additional investments to scale capabilities for WAINUA and olezarsen. The company is also advancing its next wave of potentially transformational medicines, including ION582 for Angelman syndrome, which is set to enter Phase 3 development in the first half of 2025.

Commercial revenue for the second quarter and first half of 2024 included new royalty streams from the U.S. launch of WAINUA and royalties from QALSODY's sales in the U.S. and EU. R&D revenue increased due to amortization of upfront payments from new collaborations with Roche and Novartis and licensing fees from expanded agreements, such as the donidalorsen licensing agreement with Otsuka, which now covers the Asia-Pacific region.

Operating expenses rose in line with expectations, driven by the U.S. launch of WAINUA and preparations for the launches of olezarsen and donidalorsen. Despite these expenses, R&D costs remained relatively stable as several late-stage studies concluded.

As of June 30, 2024, Ionis' cash, cash equivalents, and short-term investments totaled $2.1 billion, down from $2.3 billion at the end of 2023. The company plans to end 2024 with $1.7 billion in cash, cash equivalents, and short-term investments, focusing on late-stage programs and new medicines.

Ionis continues to advance its pipeline with several key developments:
- Olezarsen achieved multiple clinical and regulatory milestones for FCS and sHTG.
- Donidalorsen demonstrated positive results in Phase 3 trials for HAE.
- ION582 showed positive Phase 2 data for Angelman syndrome and is preparing for Phase 3 development.
- ION224 presented positive Phase 2 data for metabolic dysfunction-associated steatohepatitis (MASH).
- The Phase 1/2 Orbit study of ION356 commenced for Pelizaeus-Merzbacher disease (PMD).

Ionis remains committed to delivering innovative treatments for serious diseases and expects its strategic investments to drive sustainable growth in the coming years.

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