- Ionis Pharmaceuticals Inc. is flying solo — at least for now — in developing and commercializing a pair of rare disease drugs now that GlaxoSmithKline plc has decided not to license them.
- With full rights in hand, Ionis plans to file inotersen, an investigational treatment for hereditary TTR amyloidosis (hATTR), for U.S. approval later this year and launch it in 2018. The Carlsbad, California-based company also intends to usher its immunology medicine IONIS-FB-LRxinto a Phase 2 study for dry age-related macular degeneration (AMD) in 2017 and studies for other indications next year.
- GSK and Ionis have worked together since 2010 to find and develop treatments for rare diseases. Though the British pharma is leaving inotersen and IONIS-FB-LRx behind, its collaborations with Ionis for two hepatitis B drugs remain in tact.
GSK is in the midst of an expansive overhaul of its R&D business. In presenting the company's second quarter earnings, new CEO Emma Walmsley disclosed the company would be axing roughly 30 clinical and preclinical programs.
Moving forward, Walmsley explained that GSK's main focuses will be respiratory, HIV, oncology and inflammation drugs. As for its rare disease business, the company recently completed a strategic review and is looking to sell the unit.
"Research into rare diseases will continue within our core therapeutic areas, as a fundamental element of drug discovery," a GSK spokeswoman said in an email to BioPharma Dive about the decision not to license inotersen and IONIS-FB-LRx. "This follows a thorough analysis of the available efficacy and safety data for inotersen after completion of the pivotal trial and in parallel with a strategic review of our portfolio to identify the medicines we believe have the greatest potential to benefit patients and deliver long term value for GSK."
"We have had a long-standing partnership with Ionis, and will continue to work with them to ensure a smooth transition of the inotersen project to enable them to progress this important work as rapidly as possible," she said.
The spokeswoman added that GSK is still committed to developing therapies for amyloidosis, and will continue to work on a combination treatment of its serum amyloid P component (SAP) monoclonal antibody, GSK2398852, plus an SAP depleter, GSK2315698, for the systemic form of the disease.
Ionis, meanwhile, doesn't appear to be sweating GSK's departure. A frequent collaborator, the company is developing a large crop of medicines with big drugmakers like Biogen, Novartis and Roche, and anticipates finding new partners for inotersen.
"We are actively considering forming a commercial subsidiary to commercialize or co-commercialize inotersen in North America, as well as other options,"&Sarah Boyce, Ionis' chief business officer, said in an Aug. 11 statement, also noting the company's previous success in building out its subsidiary Akcea Therapeutics Inc.
"We have substantial interest from potential partners and are in discussions with several parties. We believe that, together with the right commercial partner, we can maximize the commercial success of the drug worldwide," Boyce said.
Ionis investors appeared to share that optimism. While the drugmaker's stock was initially down about 4% to $43.83 per share at market's open Friday, it rallied back up to $46.16 per share in mid-morning trading.