Following positive Phase I/II trials with its recombinanthuman Factor IX treatment, Genetics Institute Inc. said itbegan Phase III studies of the blood clotting drug forpatients with hemophilia B, a less common form of thedisease than hemophilia A.
Dennis Harp, spokesman for Genetics Institute, said theCambridge, Mass.-based company expects to haveenough data from the Phase III trials to file for marketapproval of the Factor IX drug in 12 to 18 months in theU.S. and Europe.
Hemophilia B patients have a deficiency in theproduction of Factor IX and are subject to uncontrolledbleeding. Current treatments involve plasma-derivedFactor IX. Genetics Institute's recombinant version of theblood-clotting protein, produced in mammalian cellcultures, reduces supply constraints and contaminationrisks associated with products from donor plasma.
Harp said the Phase III studies will involve 20 to 30patients at eight medical centers in the U.S., U.K.,Belgium, France and Germany. Eleven patientsparticipated in the Phase I/II trials comparing therecombinant drug to the natural human protein. GeneticsInstitute said its Factor IX controlled spontaneousbleeding episodes in all patients receiving the drug.
Hemophilia B affects about 10,000 people worldwide.Sales of plasma-derived Factor IX are about $150 millionannually.
The most common form of the disease is hemophilia A,whose approximately 50,000 victims have a deficiency ofthe blood-clotting Factor VIII. Genetics Institute'srecombinant Factor VIII is sold globally by BaxterHealthcare Corp., of Deerfield, Ill.
Harp said Genetics Institute's sales of the product toBaxter in 1994 totaled $43 million and for the firstquarter of 1995 were $23 million. Genetics Institute alsoreceives royalties from Baxter.
Of the $800 million in Factor VIII sales each year, therecombinant version of the blood clotting proteinaccounts for about half. In addition to Baxter, Germany-based Bayer AG also sells a recombinant Factor VIII.
Harp said Genetics Institute intends to market Factor IXitself in North America and is evaluating options for saleselsewhere.
Genetics Institute's stock (GENIZ) closed Tuesdayunchanged at $35. n
-- Charles Craig
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