Bioverativ (BIVV) plans to improve compliance rates for hemophilia patients further by launching therapies with lower dosage frequency.
On June 12, 2017, the FDA (U.S. Food & Drug Administration) accepted the company’s IND (Investigational New Drug) application for investigational therapy BIVV001. This prophylactic therapy is being studied as a once-weekly or less frequent dosing regimen for hemophilia A patients.
The binding of factor VIII with the von Willebrand factor has been the major challenge in developing therapies with lower dosing frequency for hemophilia A patients. Since the von Willebrand factor has a very short half life, it has proved difficult to develop a once-weekly dosing regimen.
However, in animal studies, BIVV001 has demonstrated double the half-life compared to Eloctate, the longest-acting factor VIII currently available in the market.
So considering the unmet market need, if BIVV001 manages to meet its clinical trial endpoints, it may have a favorable impact on Bioverativ stock as well as the Vanguard Extended Market ETF (VXF). Bioverativ makes up about 0.15% of VXF’s total portfolio holdings.
The company also plans to explore BIVV002, a subcutaneous formulation for hemophilia B patients.
To compete effectively with blood disorder players such as Novo Nordisk (NVO), Pfizer (PFE), and Shire (SHPG) and expand beyond the hemophilia indication, Bioverativ is working with Sangamo Therapeutics on a genome editing program for beta thalassemia and sickle cell diseases.
Bioverativ has also collaborated with the San Raffaele Telethon Institute for Gene Therapy to develop a gene-based therapy for hemophilia A and B patients.
Bioverativ is also planning to strengthen its research pipeline inorganically. It believes in adding early-stage assets since these investigational orphan drugs can be rapidly advanced to a proof-of-concept stage by understanding the major biomarkers.