Baxalta IncorporatedBXLT announced initial top-line data from an uncontrolled, open-label, multi-center phase III study on its hemophilia A drug, Adynovate. The study was conducted to evaluate the safety and immunogenicity of Adynovate and enrolled 73 previously treated patients (PTPs) with severe hemophilia A younger than 12 years of age and assessed the treatment's hemostatic efficacy in prophylaxis and treatment of bleeding episodes.
Results showed that no patient developed inhibitory antibodies to Adynovate, thereby meeting the primary endpoint of the study. While more than 70% of the patients when treated with Adynovate experienced zero joint bleeds, almost 40% experienced zero bleeds. On the safety front, no treatment-related serious adverse events were reported.
Based on these encouraging study results, Baxalta plans to file for approval in Europe and also intends to file for a pediatric indication in the U.S. in early 2016. The company plans to present complete data from this study at a congress in 2016. Currently, Adynovate is under review in Japan, Canada and Switzerland.
We note that Adynovate is an extended circulating half-life recombinant factor VIII (rFVIII), based on Advate (Baxalta's hemophilia A drug), a full-length FVIII molecule. Adynovate was approved in the U.S. last month for the on-demand treatment and control of bleeding, and prophylaxis to reduce the frequency of bleeding episodes in adolescent and adults (12 years and older) suffering from hemophilia A.
Meanwhile, Baxalta is working on expanding Adynovate's label further. In addition to an ongoing study in the surgical setting, Baxalta's continues to progress with the continuation study to assess the long-term safety and efficacy in PTPs with severe hemophilia A. The company also recently announced the initiation of a study in previously untreated patients with severe hemophilia A as well as a study to evaluate pharmacokinetic-guided prophylaxis dosing with Adynovate (the PROPEL study).
We note that Baxalta developed Adynovate using Nektar Therapeutics' NKTR PEGylation technology.
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