Curis announces start of Roche-sponsored study of Erivedge for AML and MDS

LEXINGTON, Mass.—Oct. 4 saw Curis Inc., anoncology-focused company, announce that Roche initiated a Phase Ib/II study ofErivedge (vismodegib) in patients with relapsed/ refractory acute myelogenousleukemia (AML) and relapsed/refractory high-risk myelodysplastic syndrome(MDS). Roche and its wholly-owned subsidiary, Genentech, have developedErivedge under a collaboration agreement with Curis.

Aberrant activation of the Hedgehog signalingpathway has been reported to play a role in the development of certain cancers,including basal cell carcinoma and AML. Erivedge is an oral drug designed to blockabnormal Hedgehog pathway signaling, and already has been approved for thetreatment of advanced basal cell carcinoma in the United States, Europe and anumber of other countries. The current Phase Ib/II clinical trial is designedto examine the potential benefit from Erivedge treatment for AML and MDS cancerpatients. The companies involved believe that selective targeting and blockingof the Hedgehog signaling pathway may have an effect on leukemic (stem) cellproliferation and survival.

"We are extremely pleased with Roche'scommitment to the continued development of Erivedge for treatment of patientswith cancers where the Hedgehog pathway appears to be altered," said Dr. AliFattaey, Curis' president and CEO. "We believe that inhibition of Hedgehogpathway signaling by Erivedge has the potential to provide benefit for patientswith AML and MDS. Specifically, unlike basal cell carcinomas that are driven bymutations in the Hedgehog pathway, AML and MDS represent cancers whereligand-dependent abnormal signaling within this pathway is associated with thedisease."

According to Roche, the open-label, non-randomizedclinical study is expected to enroll approximately 60 patients into twocohorts. Patients in one cohort will receive 150 milligrams of Erivedge aloneonce daily, and patients in the other cohort will receive the same dose ofErivedge once daily in combination with the standard dose of cytarabineadministered for 10 days. The primary endpoint of the trial is the overallresponse rate after eight weeks of treatment. The secondary endpoints includeoverall response rate at any time during treatment, duration of response,overall survival, and safety and pharmacokinetics of the study drug(s).

Roche has developed Erivedge under a collaborationagreement with Curis. Erivedge was discovered by Genentech and jointlyvalidated by Genentech and Curis through a series of preclinical studies.Through this collaboration, Genentech in the United States, Roche outside theUnited States (excluding Japan) and Chugai Pharmaceuticals (in Japan) areresponsible for the clinical development and commercialization of Erivedge.Curis is eligible to receive cash payments assuming the successful achievementof specified clinical development and regulatory approval milestones, as wellas royalties upon commercialization of Erivedge.

In January 2012, Erivedge became the firstlicensed medicine for patients with advanced basal cell carcinoma when the U.S.Food and Drug Administration (FDA) approved it following a priority review.Erivedge has since also been approved in other countries, including Australia, Canada,Ecuador, European Union, Norway, Israel, Mexico, South Korea, Switzerland andUruguay.

SOURCE: Curis news release