China’s NMPA has given Belief Biomed Inc. the official go-ahead to start testing its investigational gene therapy, BBM-H901, for the potential treatment of hemophilia B in the country, marking the first time an I.V. gene therapy for a rare disease has been approved in China. The company plans to advance the phase I/II trial for the candidate shortly, it said.
Fulcrum Therapeutics Inc. shares (NASDAQ:FULC) closed at $18.77, up $10.44 or 125%, on word of positive interim results from a phase I trial in healthy adult volunteers with oral FTX-6058 for sickle cell disease (SCD). The firm has “already achieved maximal target engagement [MTE] at all three doses,” said Christopher Morabito, the company’s chief medical officer. “I don’t think we’ll exceed that.”
The new case of myelodysplastic syndrome (MDS) that overshadowed Bluebird Bio Inc.’s earnings and resulted in a clinical hold by the FDA will be addressed similarly to an earlier hitch in the sickle cell disease program, said Andrew Obenshain, the company’s head of severe genetic diseases. “Essentially, we will try and do the same thing,” and exonerate the lentiviral vector, he said during a conference call with investors. “At this point, we don't have tumor cells or leukemia to analyze,” he noted.
Sinocelltech Group Ltd. won market approval from China’s NMPA for SCT-800, a B-domain deleted recombinant human coagulation factor VIII, for the prophylactic treatment of severe hemophilia A in adolescent and adult patients. This marks the first homegrown drug for treating hemophilia A in China.
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