Ionis Pharmaceuticals Analysis: $4.5B Raised
What is Ionis Pharmaceuticals?
Pioneer in RNA-targeted medicines with five marketed drugs and a leading neurology/cardiology pipeline
Employees
1001-5000
Founded
1989
Valuation
4.52B
HQ Full Address
Carlsbad, California, United States
Revenue Growth Rate
34%
Product Features & Capabilities
- RNA-targeted therapeutics
- Donidalorsen for rare neurological disorders
- Olezarsen for familial chylomicronemia syndrome
- Zilganersen for Alexander disease
- Proprietary antisense chemistry
- Clinical development in neurology and cardiometabolic diseases
Investment Focus
Ionis Pharmaceuticals is primarily focused on therapeutic areas including neurology, cardiology, and select areas of high patient needs. They aim to provide new solutions for serious diseases, particularly in common intractable neurologic conditions and cardiometabolic disorders.
Other Considerations
Approved donidalorsen in U.S. as first and only RNA-targeted therapy for rare neurological condition; Olezarsen approved in EU for familial chylomicronemia syndrome; Positive topline results from pivotal study of zilganersen in Alexander disease; Two new medicines launched in under nine months; Pipeline focused on neurology and cardiometabolic diseases; NDA submission planned for zilganersen in Q1 2026
Latest Funding Round
Ionis Pharmaceuticals recently completed a public offering, pricing 11,500,000 shares of its common stock at $43.50 per share, raising a total of $500.3 million. This funding round was announced on September 10, 2024.
Key Innovations
- Spinraza (Nusinersen) - a treatment for spinal muscular atrophy.
- Tegsedi (Inotersen) - used for the treatment of hereditary transthyretin amyloidosis.
- Waylivra (Volanesorsen) - for the treatment of familial chylomicronemia syndrome.
Awards Recognition
Ionis Pharmaceuticals has received several awards and recognitions in the biotech field, including:
1. The Prix Galien USA Award for the Best Biotechnology Product in 2020 for TEGSEDI, which is the first and only self-administered, subcutaneous treatment for the polyneuropathy of hereditary ATTR amyloidosis in adults.
2. The Xconomy Building a Biotech Award in 2020, recognizing the company for allowing employees to pursue research.
3. The Prix Galien Best Biotechnology Product Award for SPINRAZA in collaboration with Biogen.
1. The Prix Galien USA Award for the Best Biotechnology Product in 2020 for TEGSEDI, which is the first and only self-administered, subcutaneous treatment for the polyneuropathy of hereditary ATTR amyloidosis in adults.
2. The Xconomy Building a Biotech Award in 2020, recognizing the company for allowing employees to pursue research.
3. The Prix Galien Best Biotechnology Product Award for SPINRAZA in collaboration with Biogen.
Partnerships
- Collaboration with Novartis - Ionis entered a collaboration and license agreement with Novartis to advance a program targeting Lp(a) for cardiovascular disease, building on an existing collaboration for pelacarsen, which is currently in a Phase 3 study.
- Expansion of Agreement with AstraZeneca - Ionis expanded its agreement with AstraZeneca to include the licensing of eplontersen in Latin America, with potential earnings of up to $200 million.
- Partnership with GSK - Ionis and GSK have been collaborating on the development of bepirovirsen, which has advanced into Phase 3 clinical trials.
- Collaboration with Chinook Therapeutics - In May 2023, Ionis partnered with Chinook Therapeutics to develop an antisense therapy for rare, severe chronic kidney disease.
- Agreement with Roche - Ionis entered an agreement with Roche for two novel RNA-targeted programs, granting Roche exclusive rights to develop and commercialize the investigational medicines.
- Multi-Target Partnership with Scipher Medicine - Ionis announced a multi-disease area collaboration with Scipher Medicine in July 2023.
Regulatory Approvals
- TRYNGOLZA™ (olezarsen) - Approved by the U.S. Food and Drug Administration (FDA) as the first-ever treatment for adults with familial chylomicronemia syndrome (FCS) as an adjunct to diet, with the approval date on December 19, 2024.
- WAINZUA™ (eplontersen) - Approved in the European Union for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
- QALSODY™ (tofersen) - Approved by the FDA as the first treatment targeting SOD1-ALS.
