Catalyst Biosciences Inc. (NASDAQ: CBIO) saw its shares practically triple in Tuesday’s session after the company announced a critical regulatory approval. The company reported that the Korean Ministry of Food and Drug Safety (MFDS) approved the Investigational New Drug Application (IND) for CB 2679d/ISU304, which works to treat individuals with hemophilia.
Hemophilia is a serious bleeding disorder that results in spontaneous bleeding episodes, as well as substantially prolonged bleeding times upon injury.
In terms of the drug, it is a highly potent next-generation coagulation Factor IX variant, has demonstrated the potential to normalize human Factor IX levels with a daily subcutaneous injection in preclinical studies. In simpler terms, CB2679d/ISU304 works to inhibit blood loss and can be used as a preventative treatment to individuals with hemophilia.
Catalyst’s collaborator, ISU Abxis, plans to initiate a Phase 1/2 proof-of-concept study in individuals with severe hemophilia B in the second quarter of 2017. After this Phase 1/2 study, ISU Abxis has an option for exclusive commercial rights in South Korea and a profit share on ex-South Korean commercialization. However, Catalyst would retain full development and commercial rights for the drug outside of South Korea.
Nassim Usman, Ph.D., president and CEO of Catalyst Bio, commented:
We are very pleased with the progress we and our collaborator ISU Abxis have made in advancing this Factor IX candidate towards the clinic and we look forward to the initiation of a Phase 1/2 clinical trial next quarter. We believe that stopping bleeding is good, but preventing bleeding is better. CB 2679d/ISU304 has the properties required, including high potency, to allow for daily subcutaneous injection with the potential to achieve stable and normal Factor IX clotting levels.
Shares of Catalyst Bio were trading up 196% at $15.24 on Tuesday, with a consensus analyst price target of $24.50 and a 52-week trading range of $0.45 to $18.88.