Stock To Watch: Rocket Pharma On Track

(RTTNews) - Rocket Pharmaceuticals, Inc. (RCKT), on Sunday, reported positive preliminary results from its phase I study of commercial-grade gene therapy RP-L102 "Process B" for Fanconi Anemia (FA) at the 61st annual meeting of American Society of Hematology (ASH).

Fanconi anemia is a rare genetic disorder, that mainly affects the bone marrow resulting in the decreased production of all types of blood cells.

The data were that of two pediatric patients of age 5 and 6 years who were treated with "Process B" RP-L102 prior to the development of severe bone marrow failure. Treatment with 'Process B' RP-L102 at four to six months post-infusion showed early signs of engraftment and bone marrow restoration, noted the Company. Previously declining blood cell counts have been stabilized or even increased within six months of therapy.

"Process B" trial incorporates higher cell doses, transduction enhancers, and commercial-grade vector manufacturing and cell processing. No safety or tolerability issues have been reported.

The company expects to present additional long-term follow-up data from these patients in the first half of 2020.

A global registrational phase II study of "Process B" RP-L102 for Fanconi Anemia is currently underway, with primary endpoint of bone marrow MMC-resistance. The trial is expected to enroll five patients in the U.S. and five patients in Europe.

RCKT closed Friday's trading at $22.36, up 2.66%.