CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for zilganersen, a potential treatment for Alexander disease (AxD), an ultra-rare and fatal neurological disorder. This status, announced today, is intended to accelerate the development and review process for drugs that address serious conditions and fulfill an unmet medical need.
The investigational medicine, zilganersen, is currently the only one in clinical development for both adult and pediatric patients with AxD, a leukodystrophy that affects the brain's white matter. With an estimated global prevalence between one in one million and one in three million, AxD leads to a decline in neurological functions and typically results in death within 14 to 25 years from the onset of symptoms. Diagnosis is based on clinical symptoms, MRI findings, and genetic testing.
Ionis' executive vice president and chief clinical development officer, Eugene Schneider, M.D., underscored the significance of the Fast Track designation, stating that it reflects the gravity of AxD and the substantial need for therapies. He expressed the company's anticipation for the pivotal study data expected in the latter half of 2025 and the goal to introduce the first approved treatment for AxD.
Zilganersen, also known by its lab name ION373, is an antisense oligonucleotide designed to reduce the production of the glial fibrillary acidic protein (GFAP), which accumulates due to disease-causing gene variants. Prior to this designation, the FDA had granted zilganersen Orphan Drug and Rare Pediatric Disease designations, while the European Medicines Agency (EMA) had also granted it Orphan Drug designation in 2019.
Ionis, a company specializing in RNA-targeted therapies, has a history of developing medicines for serious diseases, boasting five marketed medicines and a robust pipeline focused on neurology and cardiology. The pivotal Phase 1-3 study for zilganersen has completed enrollment, with trials conducted across 13 sites in eight countries.
This news is based on a press release statement from Ionis Pharmaceuticals. Zilganersen has not yet been approved for any indication by regulatory authorities, and its safety and efficacy remain under investigation. Ionis has not provided any information on the potential commercialization of the drug or its impact on the company's financial future.
In other recent news, Ionis Pharmaceuticals announced robust progress in its 2024 product pipeline, with steady financial results aligning with its annual guidance. The company launched two new treatments, WAINUA for hereditary ATTR polyneuropathy and QALSODY for ALS, in the US and Europe. Ionis is also preparing for the launch of Olezarsen for FCS and global expansion. Despite discontinuing development programs for ION541 for ALS and IONIS-FB-LRx for geographic atrophy, the company remains optimistic about its diverse pipeline.
Furthermore, Ionis Pharmaceuticals has proposed a registered underwritten public offering of its common stock, expected to amount to $500 million, with an additional $75 million potentially available for underwriters. Managed by Morgan Stanley and Goldman Sachs & Co. LLC, the funds will be directed towards funding commercial launches, clinical programs, research and development, and general corporate needs.
RBC Capital maintained its Outperform rating and $70.00 price target on Ionis Pharmaceuticals, highlighting the company's potential in several therapeutic areas. The company also expressed excitement about potentially launching their first solo drug later this year, with a PDUFA date set for December 19th. This development could mark a significant milestone for the company in the cardiovascular disease space.
InvestingPro Insights
As Ionis Pharmaceuticals (NASDAQ:IONS) advances its promising treatment for Alexander disease, investors may find additional context from InvestingPro's data and tips valuable. The company's market capitalization stands at $6.31 billion, reflecting investor confidence in its pipeline and marketed products.
Despite the positive news on zilganersen, InvestingPro data shows that Ionis has faced some financial challenges. The company's gross profit margin for the last twelve months as of Q2 2023 was -12.86%, indicating struggles with profitability. This aligns with an InvestingPro Tip noting that Ionis "suffers from weak gross profit margins."
However, it's important to note that Ionis has demonstrated strong revenue growth, with a 29% increase in the last twelve months as of Q2 2023. This growth could be attributed to its existing marketed products and potentially reflects investor optimism about its pipeline, including zilganersen.
An InvestingPro Tip highlights that "liquid assets exceed short term obligations," suggesting that Ionis maintains a solid financial position to support its ongoing research and development efforts, including the pivotal study for zilganersen expected to yield results in late 2025.
For investors seeking a more comprehensive analysis, InvestingPro offers 10 additional tips for Ionis Pharmaceuticals, providing a deeper understanding of the company's financial health and market position.
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