First CTCL Patients Enrolled in Phase 2 Trial of Topical Treatment WP1220

The Phase 2 clinical trial assessing Moleculin Biotech‘s STAT3 inhibitor, WP1220, for the topical treatment of cutaneous T-cell lymphoma (CTCL) lesions has enrolled the first two participants.

The trial, to assess WP1220‘s safety and efficacy, has been reviewed and approved by regulatory authorities in Poland, where the study will be conducted.

“CTCL is a potentially deadly form of skin cancer involving skin lesions that often have high levels of activated STAT3,” Don Picker, PhD, chief science officer of Moleculin, said in a press release. “As a potent inhibitor of [STAT3], we believe WP1220 may be ideally suited to treat these lesions through topical application, which is what this clinical trial is designed to evaluate.”

“There is a significant unmet need for improved topical therapies for CTCL, and we are excited to be the first clinic to evaluate this promising new drug in CTCL patients,” said study coordinator Malgorzata Sokolowska-Wojdylo, MD, PhD, an associate professor in the dermatology department at the Medical University of Gdańsk.

WP1220 is a small molecule that blocks the activity of the STAT3 signaling molecule, known to regulate immune cells’ activity and modulate several cancer-related proteins.

It was first developed as a treatment for psoriasis, an immune-mediated skin disease. But results from a U.S.-based Phase 2 trial (NCT01826201) showed only limited efficacy, with WP1220 having little effect on the underlying mechanisms involved in psoriatic plaque formation.

Despite the developmental setback, preclinical studies showed that WP1220, applied to the skin, may be effective at treating CTCL lesions. Thus, Moleculin initiated a collaboration with a Polish company to further explore the investigational compound as a topical treatment for early-stage CTCL.

“In this case, we are targeting CTCL with a topical STAT3 inhibitor in light of the significant role that STAT3 appears to play in CTCL skin lesions,” Walter Klemp, chairman and CEO of Moleculin, said in another press release.

Moleculin plans to conduct a preliminary evaluation of clinical data from the first five patients to determine whether topical use of WP1220 is viable for CTCL treatment. The results are expected this year.