- Positive opinion issued by EMA Committee for Orphan Medicinal Products for CB 2679d/ISU304, a next-generation Factor IX for subcutaneous prophylactic treatment of Hemophilia B
SOUTH SAN FRANCISCO, Calif., May 31, 2017 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (Nasdaq:CBIO), a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending orphan medicinal product (orphan drug) designation for CB 2679d/ISU304 for the treatment of hemophilia B.
Currently approved therapies for treating hemophilia B rely on frequent intravenous infusions where adherence and convenient access to peripheral veins is difficult, often requiring the use of a central venous access devices that have associated risks of infection and thrombosis. CB 2679d, a highly potent next-generation coagulation Factor IX variant, has demonstrated, in preclinical studies, the potential to normalize human Factor IX levels with a daily subcutaneous injection.
“Obtaining orphan drug designation is an important part of our regulatory approval strategy for CB 2679d, and the receipt of the positive COMP opinion puts us one step closer to this goal,” said Nassim Usman, Ph.D., President and Chief Executive Officer of Catalyst.
Catalyst’s South Korean collaborator, ISU Abxis, plans to initiate a Phase 1/2 proof-of-concept study of CB 2679d in individuals with severe hemophilia B in June 2017 in South Korea.
About Orphan Designation
Applications for orphan designation are initially reviewed by the COMP. Positive opinions are forwarded to the European Commission, which is responsible for formally granting the orphan medicinal designation. Orphan designation in the EU is given to products that are intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating; where prevalence of the condition in the EU is less than 5 in 10,000; and where the product represents a significant benefit over existing treatments.
Orphan Designation benefits include protocol assistance, reduced EU regulatory filing fees and 10 years of market exclusivity. Designated orphan medicines are also eligible for conditional marketing authorization. Detailed information on orphan designation can be found here.
About Factor IX
CB 2679d/ISU304 is a next-generation coagulation Factor IX variant that is IND-approved in South Korea. Catalyst believes that CB 2679d/ISU304 may allow for subcutaneous prophylactic treatment of individuals with hemophilia B and maintain higher FIX activity levels than currently available products. Learn more about Factor IX.
About Hemophilia and Factor Replacement Therapy
Hemophilia, for which there is no cure, is a rare but serious bleeding disorder that results from a genetic or an acquired deficiency of a protein required for normal blood coagulation. Individuals with hemophilia suffer from spontaneous bleeding episodes as well as substantially prolonged bleeding times upon injury. Learn more about hemophilia.
About Catalyst
Catalyst is a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications. Catalyst is focused on the field of hemostasis, including the subcutaneous prophylaxis of hemophilia and facilitating surgery in individuals with hemophilia. For more information, visit www.catalystbiosciences.com.
Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statement of historical facts, included in this press release regarding our strategy, the potential uses and benefits of CB 2679d/ISU304 and development plans for this product candidate are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the anticipated granting of orphan drug designation of CB2679d/ISU304 in the EU by the European Commission, clinical trial timelines, including the anticipated initiation of a Phase 1/2 proof-of-concept study for CB 2679d/ISU304 in June of 2017, the potential uses and benefits of subcutaneously dosed CB 2679d/ISU304. Actual results or events could differ materially from the plans and expectations and projections disclosed in these forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Catalyst makes, including, but not limited to, the risk that the European Commission will not follow the recommendation of the European Medicines Agency Committee for Orphan Medicinal Products, the risk that trials and studies may be delayed and may not have satisfactory outcomes, that human trials will not replicate the results from animal studies, that potential adverse effects may arise from the testing or use of Catalyst’s products, including the generation of antibodies, the risk that costs required to develop or manufacture Catalyst’s products will be higher than anticipated, competition, and other factors described in the “Risk Factors” section of the Company’s most recent Annual Report on Form 10-K and our other periodic reports filed with the SEC. Catalyst does not assume any obligation to update any forward-looking statements, except as required by law.
Contacts: Investors: Fletcher Payne, CFO Catalyst Biosciences 650.871.0761 investors@catbio.com Media: Denise Powell 510.703.9491 denise@redhousecomms.com
