Ionis Pharmaceuticals Highlights Pipeline Progress and Financials in Q3 2024
Key Insights
Ionis Pharmaceuticals (search) reports strong progress with WAINUA launch for ATTRv-PN (search), generating $5 million in royalties for Q3 2024, and securing approvals in the UK and a positive CHMP opinion in Europe.
The company is advancing olezarsen for FCS (search), with a PDUFA date set for December 19, 2024, and donidalorsen for HAE (search), with a PDUFA date of August 21, 2025, positioning them for potential launches.
Positive Phase 2/3 DEVOTE study data for higher dose nusinersen in SMA (search) was presented, with global regulatory applications planned, alongside QALSODY's marketing approval in China for SOD1-ALS (search).
Ionis Pharmaceuticals (search), Inc. (Nasdaq: IONS) has announced its financial results for the third quarter of 2024, highlighting significant advancements in its pipeline and commercial achievements. The company is on track to deliver a steady cadence of transformational medicines to people with serious diseases.
Commercial Success and Regulatory Milestones
The U.S. launch of WAINUA™ (eplontersen) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (search) (ATTRv-PN (search)) is progressing well. WAINUA generated sales of $23 million in Q3 2024, resulting in royalty revenue of $5 million. The drug has also been approved in the UK and received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).
SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (search) (SMA (search)) continues to be a strong revenue generator, with global sales of $381 million in Q3 2024, resulting in royalty revenue of $57 million. Positive Phase 2/3 DEVOTE study data from higher dose nusinersen was presented, and global regulatory applications are planned.
QALSODY® (tofersen) has been granted marketing approval in China for the treatment of SOD1-ALS (search), marking another significant milestone for Ionis' marketed medicines.
Late-Stage Pipeline Advancements
Olezarsen is positioned to potentially treat two patient populations with urgent unmet needs: familial chylomicronemia syndrome (search) (FCS (search)) and severe hypertriglyceridemia (search) (sHTG (search)). The NDA for 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 pivotal development program for sHTG is on track for data in H2 2025.
Donidalorsen Phase 3 data positions it to potentially launch next year as (search) the first RNA-targeted prophylactic treatment for people with hereditary angioedema (search) (HAE (search)). The FDA accepted the NDA for HAE with a PDUFA date of August 21, 2025. Positive Phase 2 open label extension (OLE) study data in patients treated up to three years with every four weeks or every eight weeks dosing was presented.
Zilganersen Phase 3 study is fully enrolled as (search) a potential treatment for Alexander disease (search), with data expected in 2025. The drug has been granted Fast Track designation by the FDA.
ION582 achieved important clinical and regulatory milestones enabling initiation of Phase 3 development in Angelman syndrome (search) (AS (search)) in H1 2025. Positive Phase 1/2 data in patients with AS was presented at the Angelman Syndrome Foundation (ASF) Family Conference, and a positive End-of-Phase 2 discussion with the FDA, included alignment on Phase 3 design, was completed.
Positive data was reported from the Phase 2 study of IONIS-FB-LRx in patients with immunoglobulin A nephropathy (search) (IgAN (search)); Roche continues to advance IONIS-FB-LRx in the Phase 3 IMAGINATION study.
Financial Performance
Ionis' revenue for the three months ended September 30, 2024, was $134 million, compared to $144 million for the same period in 2023. Revenue for the nine months ended September 30, 2024, was $479 million, compared to $463 million for the same period in 2023. The company's cash, cash equivalents, and short-term investments increased to $2.5 billion as (search) of September 30, 2024, compared to $2.3 billion at December 31, 2023. Ionis reaffirmed its 2024 P&L financial guidance and increased its cash guidance to $2.2 billion, reflecting proceeds from an equity offering.
"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.