Krystal Biotech Announces First Patient Dosed in Phase 1 Clinical Trial of KB407 for the Treatment of Cystic Fibrosis

PITTSBURGH, July 03, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a biotechnology company focused on developing and commercializing genetic medicines for patients with debilitating diseases, announced today that the first patient has been dosed at the Cystic Fibrosis Institute of Chicago in the Company’s Phase 1 CORAL-1/US study evaluating KB407, an engineered HSV-1-based, aerosol-delivered, mutation agnostic, genetic medicine for the treatment of patients with cystic fibrosis (CF).

“By delivering full-length copies of CFTR directly to the lung via nebulization, KB407 has the potential to address the basic genetic defect present in cystic fibrosis,” said Steven R. Boas, M.D., Director of the Cystic Fibrosis Institute and Professor of Pediatrics at Northwestern University Feinberg School of Medicine. “As this therapy does not depend on the type of CFTR mutation present, KB407 may potentially benefit all individuals affected by cystic fibrosis.”

“Dosing our first patient in the KB407 Phase 1 clinical trial is an exciting step forward for the company and for the patients we aim to benefit,” said Hubert Chen, M.D., Senior Vice President of Clinical Development at Krystal Biotech. “Not only is the Phase 1 study designed to provide key insights into the safety and efficacy of KB407, it is also a critical step in expanding our vector platform to tissues beyond the skin. We look forward to continued enrollment into the Phase 1 study with anticipated data in 2024.”

CORAL-1/US Study Overview

The CORAL-1/US study is a multicenter, dose-escalation trial of KB407 in patients (n~20) with CF regardless of their underlying genotype. Each administration of KB407 will be nebulized in under 30 minutes. This study will include three cohorts and enroll five subjects each in the first two cohorts and ten subjects in the last cohort.

Cohort 1 participants will receive a single administration of KB407 on Day 0, and Cohort 2 participants will receive administrations of KB407 on Day 0 and Day 1. Cohorts 1 and 2 will be conducted on an open-label basis and up to 3 subjects in each of the first two cohorts may receive concurrent CFTR modulator therapy. Cohort 3 participants will be randomized 4:1 to receive administrations of KB407 or placebo on Day 0, Day 1, Day 2, and Day 3. Cohort 3 will follow a randomized, placebo-controlled, and double-blind design and will enroll only subjects who are not otherwise eligible for CFTR modulators. A Data Monitoring Committee will conduct a safety review after each cohort before proceeding to the next one.