Ionis Pharmaceuticals Advances Pipeline with Key Regulatory Milestones and Clinical Data
Key Insights
Ionis Pharmaceuticals (search) reports positive progress across its pipeline, highlighted by the U.S. launch of WAINUA and advancements in regulatory approvals.
Olezarsen's NDA for FCS is under FDA Priority Review with a PDUFA date of December 19, 2024, and the MAA is under review by the EMA, potentially addressing unmet needs.
Donidalorsen's Phase 3 data positions it as a potential first RNA-targeted prophylactic treatment for HAE, with an FDA action date set for August 21, 2025.
Ionis Pharmaceuticals (search), Inc. (Nasdaq: IONS) has announced its third-quarter 2024 financial results, showcasing significant advancements in its pipeline and commercial achievements. The company is on track to achieve its 2024 financial guidance, driven by the progress of key drugs and strategic investments in late-stage development.
Key Pipeline Developments
Ionis is poised for potential independent launches of olezarsen and donidalorsen, targeting familial chylomicronemia syndrome (search) (FCS) and hereditary angioedema (search) (HAE), respectively. Brett P. Monia, Ph.D., chief executive officer of Ionis, emphasized the company's readiness to bring olezarsen to patients with FCS, a serious rare disease lacking approved treatments in the U.S. The FDA has granted Priority Review to the NDA for olezarsen, with a PDUFA date of December 19, 2024. The Marketing Authorization Application (MAA) is also under regulatory review by the EMA.
Donidalorsen is positioned as a potential first RNA-targeted prophylactic treatment for HAE. The FDA has accepted the NDA for donidalorsen, with a PDUFA date of August 21, 2025. Positive Phase 2 open-label extension (OLE) study data, involving patients treated for up to three years with dosing every four or eight weeks, further supports its efficacy.
WAINUA Commercial and Regulatory Milestones
WAINUA (eplontersen), for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (search) (ATTRv-PN), has achieved multiple commercial and regulatory milestones. Sales of WAINUA generated royalty revenue of $5 million and $10 million in the three and nine months ended September 30, 2024, respectively. The drug received a positive Committee for Medicinal Products for Human Use (CHMP) opinion from the European Medicines Agency (EMA) and was approved in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA).
Advancing Neurological Disease Pipeline
Ionis is also making strides in its neurological disease pipeline. The Phase 3 study of zilganersen for Alexander disease (search) is fully enrolled, with data expected in 2025. ION582 for Angelman syndrome (search) (AS) achieved clinical and regulatory milestones, enabling the initiation of Phase 3 development in H1 2025, following positive Phase 1/2 data and alignment with the FDA on Phase 3 design.
Financial Performance
Ionis reported total revenue of $134 million for the three months ended September 30, 2024, and $479 million for the nine months ended September 30, 2024. Operating expenses for the three and nine months ended September 30, 2024, increased as planned, reflecting continued investments in late-stage development and commercialization efforts. The company's cash, cash equivalents, and short-term investments increased to $2.5 billion as of September 30, 2024.
Elizabeth L. Hougen, chief financial officer of Ionis, stated that the company's recent equity offering extends its cash runway, enabling continued investment in attractive opportunities, including near-term commercial launches and the late and mid-stage pipeline.
SPINRAZA and QALSODY Updates
SPINRAZA (nusinersen) for spinal muscular atrophy (search) (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. QALSODY (tofersen) received marketing approval in China for the treatment of SOD1-ALS (search).
Additional Pipeline Updates
Sapablursen for polycythemia vera was granted orphan drug designation by the FDA, with enrollment complete in the Phase 2 IMPRSSION study. IONIS-MAPTRx (BIIB080) enrollment is complete in the Phase 2 CELIA study for early Alzheimer's disease (AD).