Akcea Therapeutics

Akcea Therapeutics. Akcea Therapeutics is a biopharmaceutical company focused on developing and commercializing drugs for patients with serious and rare diseases, utilizing antisense technology. Founded as a subsidiary of Ionis Pharmaceuticals, the company advanced several therapies through clinical development and regulatory approval. Its operations have been closely integrated with its parent company, leading to a strategic merger to consolidate resources and pipeline development.

History

The company was established in 2015 by Ionis Pharmaceuticals to specifically develop and commercialize a portfolio of drugs derived from Ionis's proprietary antisense technology. Key early leadership included industry veteran Paula Soteropoulos, who served as its first Chief Executive Officer. A significant milestone was reached in 2018 when Akcea, in collaboration with its parent company, secured Food and Drug Administration approval for TEGSEDI (inotersen) for the treatment of hereditary transthyretin-mediated amyloidosis. That same year, the company also gained European Medicines Agency approval for both TEGSEDI and WAYLIVRA (volanesorsen). In 2020, Ionis Pharmaceuticals announced a merger to fully integrate Akcea's operations, a move completed to streamline the development of their shared pipeline and commercial efforts.

Products and pipeline

The company's commercial portfolio included two approved therapies. TEGSEDI is an antisense oligonucleotide designed to treat polyneuropathy in adults with hereditary transthyretin amyloidosis. WAYLIVRA was approved in the European Union for the treatment of familial chylomicronemia syndrome, though its New Drug Application in the United States received a Complete Response Letter from the FDA. The clinical pipeline featured investigational candidates like AKCEA-APOCIII-LRx, a drug targeting hypertriglyceridemia developed with Novo Nordisk, and AKCEA-TTR-LRx, a next-generation therapy for ATTR amyloidosis. These programs leveraged advanced LICA technology to enhance drug potency and dosing convenience.

Collaborations and partnerships

A foundational partnership existed with its parent company, Ionis Pharmaceuticals, which provided the core antisense technology platform. A major strategic collaboration was formed with Novo Nordisk in 2019 to develop and commercialize drugs for cardiometabolic diseases, including AKCEA-APOCIII-LRx and AKCEA-ANGPTL3-LRx. The company also worked with Pfizer on the development and global commercialization of vupanorsen. Commercialization efforts for TEGSEDI involved partnerships with various specialty pharmacy and distribution networks across North America and Europe to reach patients with rare diseases.

Financial performance

Following its initial incorporation, Akcea completed an initial public offering on the NASDAQ stock exchange in 2017, trading under the ticker symbol AKCA. Its primary revenue streams were generated from the global sales of its approved product, TEGSEDI, and collaboration revenues from partners like Novo Nordisk. The company reported net product revenues alongside significant research and development expenditures required to advance its clinical pipeline. The 2020 merger with Ionis Pharmaceuticals was structured as a stock-for-stock transaction, dissolving its status as a separate publicly traded entity on the NASDAQ.

Leadership and governance

The company was initially led by Paula Soteropoulos, who served as Chief Executive Officer and was a member of its Board of Directors. Following her departure, Damien McDevitt, previously the Chief Operating Officer, was appointed CEO. Other key executives included Sarah Boyce as President and Jeffrey Goldberg as Chief Financial Officer. The Board of Directors included representatives from Ionis Pharmaceuticals, such as B. Lynne Parshall and Stanley T. Crooke, the founder of Ionis. This governance structure ensured close strategic alignment with its parent company throughout its operational history.

Category:Biotechnology companies of the United States Category:Companies based in Boston Category:Pharmaceutical companies established in 2015