Ionis Pharmaceuticals, Inc. (IONS) has received some exciting news regarding its investigational RNA-targeted therapy, zilganersen. The FDA has granted zilganersen Fast Track designation for the treatment of Alexander disease (AxD), an ultra-rare neurological disorder. This designation is a significant boost for Ionis as it signifies the FDA’s commitment to expediting the development and review of drugs that address serious conditions with unmet medical needs. The goal of the Fast Track program is to get these treatments to patients who desperately need them as quickly as possible.
AxD is a rare neurological disease that affects astrocytes, a type of cell in the brain crucial for supporting neurons and oligodendrocytes. Individuals living with AxD experience cognitive dysfunction and progressive neurological deterioration, impacting their ability to swallow and control muscle movements for large actions. Zilganersen represents a beacon of hope for these patients as it is the first investigational medicine in clinical development for AxD, a condition currently without any approved therapies.
The FDA has previously recognized the potential of zilganersen by granting it Orphan Drug designation and Rare Pediatric Disease designation for treating AxD. This continued support from the FDA underscores the urgent need for a treatment option for this debilitating disorder.
Ionis is currently conducting late-stage studies to evaluate zilganersen’s effectiveness in treating adults and children with AxD. In July, the company completed full patient enrollment for a pivotal Phase III study. Top-line data from this study is anticipated in the second half of 2025. The primary endpoint of the study is to assess the percent change from baseline in gait speed using the 10-Meter Walk Test (10MWT) at the end of the 60-week treatment period.
Zilganersen is one of Ionis’ wholly-owned pipeline candidates, and the company intends to launch it independently in the United States. This signifies Ionis’ commitment to bringing its innovative therapies directly to patients.
Beyond zilganersen, Ionis has a promising portfolio of wholly-owned candidates. Olezarsen, for familial chylomicronemia syndrome and severe hypertriglyceridemia, is nearing the market. In April 2024, Ionis submitted a New Drug Application (NDA) to the FDA seeking approval for olezarsen in the FCS indication. The FDA is expected to make a decision by December 19, 2024. If approved, olezarsen will become Ionis’ first independently launched medicine. Ionis is also preparing to file an NDA for donidalorsen, a treatment for hereditary angioedema, with the FDA later in 2024. The company expects donidalorsen to be its second independent commercial launch upon potential approval.
Ionis’ dedication to its internal pipeline development is further emphasized by its collaborations with leading drugmakers and biotech companies. These partnerships provide Ionis with valuable funding in the form of license fees, upfront payments, and milestone payments, allowing them to continue investing in their groundbreaking research and development initiatives.
While Ionis’ future appears bright, it’s important to note that the company’s stock has declined 22.8% this year, compared to the industry’s 1.3% fall. This decline highlights the inherent volatility in the biotech sector and the challenges associated with bringing new therapies to market. However, with the FDA’s support and Ionis’ dedication, zilganersen and its other promising pipeline candidates offer a ray of hope for patients battling rare and debilitating diseases.
