Ionis Pharmaceuticals will receive a $75 development milestone payment from Bayer, relating to the continued clinical development of the antisense drug IONIS-FXIRx and the start of a clinical program for a second antisense candidate, IONIS-FXI-LRx. Ionis says it plans to start a Phase IIb study with IONIS-FXIRX in patients with end-stage renal disease who are on hemodialysis. “We recently completed a Phase II study in patients with end-stage renal disease on hemodialysis, in which IONIS-FXIRx demonstrated robust reductions in Factor XI activity and no treatment-related major bleeding, ” stated B. Lynne Parshall, COO at Ionis Pharmaceuticals. The firm will also take IONIS-FXI-LRx through Phase I development. Under terms of the agreement with Bayer, once these studies have been carried out, and if Bayer decides to progress the programs, the German drugs giant will take over responsibility for all global development, regulatory, and commercialization activities for both drugs. Ionis will be eligible for development milestones, plus tiered royalties up to the high 20% range, on gross margins of both drugs combined.
IONIS-FXIRx and IONIS-FXI-LRx are antisense drugs designed to reduce the production of Factor XI. IONIS-FXI-LRx has been developed using Ionis’s Ligand Conjugated Antisense (LICA) platform. “We are pleased that Bayer has decided to expand our collaboration and initiate development of a LICA antisense drug targeting Factor XI,” Parshall added. “Our LICA technology enables flexible, low, and infrequent doses and dose regimens, which may be preferred for a drug targeting broad indications.”
Earlier this month Ionis earned a $5 million milestone payment from partner Biogen following the validation of a neurological disease target. Biogen and Ionis have a broad drug development collaboration in the field of neurological disorders. In December 2016, the FDA approved the firms' antisense drug SpinrazaTM for the treatment of spinal muscular atrophy in pediatric and adult patients. During January of this year, Novartis agreed to a potentially $1B global option and collaboration agreement to develop the Ionis subsidiary Akcea Therapeutics's cardiovascular disease candidates AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx.