- Two additional subjects completed Catalyst Biosciences' ongoing hemophilia Phase 2/3 trial without any bleeding or developing any anti-drug antibodies (ADAs), the company said in a statement Wednesday.
- The San Francisco, California-based biopharma initially released interim data in July on the first subject in the study, which aims to enroll 12 total participants. Consistent with the first patient, all three who have completed the trial have yet to exhibit bleeding or develop ADAs. The company also said two more subjects are currently enrolled.
- Catalyst is developing MarzAA, short for Marzeptacog alfa (activated), as a subcutaneous Factor VIIa therapy for treating hemophilia A or B with inhibitors. They presented the new data Wednesday in Boston at the 2018 Hemophilia Drug Development Submit.
In the last few years, hemophilia has turned into one of the most competitive areas among blood disorder therapies. Drugmakers are now seeking not just effective hemophilia treatments, but longer-lasting and safer options.
Currently, Roche AG's Hemlibra (emicizumab) is the front-runner in that regard, although the Swiss pharma disclosed in March that five patients with hemophilia A died since 2016 while undergoing treatment, though the causes of death were shown to be unrelated to the drug.
But new data from a late-stage program released in May impressed Wall Street, showing Hemlibra as a powerful hemophilia A treatment with or without inhibitors.
Gene therapies are also in development, although they have had their own speed bumps in the path to approval and the market. Spark Therapeutics, for instance, lost $1 billion in market value earlier this month when it released mixed Phase 2 results for its hemophilia gene therapy.
While it's too early in the study to draw any conclusions on the longevity of Catalyst's treatment, the initial results from the three subjects who have completed the trial are encouraging.
The study is formatted to take a patient through an individual dose escalation, but only if a breakthrough bleed occurs in the patient. The first stage is 50 days with 30 µg/kg; the second stage is 50 days with 60 µg/kg; the third stage is 50 days with 90 µg/kg; and the final stage is 50 days with 120 µg/kg.
"We have not observed any bleeds or anti-drug antibodies in the two additional subjects, who most recently completed dosing with 30 µg/kg MarzAA, as well as in the previously reported individual who completed 50 days of dosing with 60 µg/kg MarzAA," Catalyst CEO Nassim Usman said in a statement.
Long-acting hemophilia drugs were chosen as BioPharma Dive's 2017 Disruption of the Year, driven by the fact that the global market is expected to surpass $25 billion by 2024.