Blood disorder therapeutics firm Bioverativ, which was spun out from Biogen earlier this year, announced a research collaboration with Bicycle Therapeutics focused on developing and commercializing new treatments for hemophilia and sickle cell disease, based on the U.K. firm's Bicycle® platform.
Bicycles are bicyclic peptides that are designed to combine the benefits of antibodies, small molecules, and peptides in one molecule, enabling high selectivity and affinity. Under terms of their partnership Bicycle will lead discovery and lead optimization activities through to candidate selection for two product development programs, with Bioverativ heading subsequent preclinical development through to commercialization and marketing.
Bicycle will receive a $10 million up-front fee and near-term R&D funding of $4.2 million. The firm could also earn up to $410 million in development, regulatory, and commercialization milestones for planned products under the two programs, plus royalties on future sales.
“We are constantly exploring new ways to do innovative science to find new molecules that can advance the care of people living with rare blood disorders,” Tim Harris, Ph.D., D.Sc., evp for R&D at Bioverativ. “This collaboration offers a unique opportunity to identify an entirely new therapeutic modality that may lead to meaningful new treatments and outcomes for people living with hemophilia and sickle cell disease.”
“Combining Bioverativ’s deep expertise in hematology with our powerful platform offers great promise for the development of novel, targeted therapies for patients,” added Kevin Lee, Ph.D., CEO at Bicycle Therapeutics. “This alliance provides the latest validation of our Bicycle platform and furthers our strategy to evaluate its potential in a wide range of new disease areas.”
Bioverativ was spun out of Biogen’s hemophilia business to develop new treatments for hemophilia, sickle cell disease, and other rare blood disorders. The Bioverativ pipeline spans partnered and in-house clinical, preclinical, and discovery-stage programs, inlcuding genome editing and small-molecule and biologic candidates against cold agglutinin disease (CAgD), beta thalassemia, sickle cell disease, hemophilia A, and hemophilia B. The firm’s in-house clinical pipeline has been headed by BIVV001, a novel Factor VIII therapy for hemophilia A, which is designed to allow once-weekly, or even less frequent, dosing. The FDA accepted the firm’s Investigational New Drug (IND) application for BIVV001 in June, and a Phase I/IIa study is expected to start during the second half of 2017.
Also in June, Bioverativ completed its potentially $825 million acquisition of True North Therapeutics, which has given Bioverativ a first-in-class humanized monoclonal antibody, BIVV009 (previously TNT009), for treating CAgD. BIVV009 has been granted breakthrough therapy designation by the FDA and orphan drug designation by both FDA and the European Medicines Agency for treating hemolysis in patients with primary CAgD. Bioverativ is planning its late-stage clinical development and registrational trials program for the candidate. Also acquired as part of the True North Therapeutics buyout is TNT020, a discovery-stage, follow-on monoclonal antibody with the potential for less frequent dosing and subcutaneous administration.
Bicycle Therapeutics is developing its bicyclic peptide platform for the potential treatment of cancer and other diseases. The firm's in-house development, focused on cancer, is headed by lead candidate BT1718, a membrane type 1 matrix metalloproteinase (MT1-MMP)-targeting Bicycle Drug Conjugate®, which is designed to deliver the cytotoxic drugs directly to tumors. Early clinical development is being funded through a partnership with Cancer Research UK.
Bicycle Therapeutics raised $52 million in a Series B round of fundraising in June 2017. In December 2016, the firm announced a collaboration with AstraZeneca, worth potentially more than $1 billion, to develop Bicycle bicyclic peptides against an undisclosed number of AstraZeneca targets across multiple nononcology disease areas.