Krystal Biotech expects interim results from GEM-3 trial in 1H20
The company said, "In September 2019, we initiated a Phase 1/2, first in-human trial of our second product candidate, KB105, an HSV-1 based gene therapy engineered to deliver a human transglutaminase-1 gene to patients with TGM1-deficient autosomal recessive congenital ichthyosis. TGM1-deficient ARCI is a debilitating rare skin disease characterized by excessive, thick scaling of the skin, causing multiple chronic health conditions. There are approximately 23,000 cases of TGM1-deficient ARCI worldwide and about 400 new cases per year globally. We have dosed two patients in the GEM-3 trial and anticipate announcing interim results in 1H 2020. In October 2019, the FDA granted Fast Track designation to KB105, the company's HSV-1 based gene therapy engineered to deliver a functional human TGM1 gene in patients with TGM1 deficient ARCI. KB105 is currently in a Phase 1/2 clinical study with interim data expected in mid-2020. Fast Track Designation is granted to drugs being developed for the treatment of serious or life-threatening diseases or conditions where there is an unmet medical need. The purpose of the Fast Track Designation provision is to help facilitate development and expedite the review and potential approval of drugs to treat serious and life-threatening conditions. Sponsors of drugs that receive Fast Track Designation have the opportunity for more frequent interactions with the FDA review team throughout the development program. These can include meetings to discuss study design, data required to support approval, or other aspects of the clinical program. Additionally, products that have been granted Fast Track Designation may be eligible for priority review of a New Drug Application and the FDA may consider reviewing portions of an NDA before the sponsor submits the complete application. In October 2019, the European Medicines Agency Committee for Orphan Medicinal Product granted orphan drug designation to KB105 for the treatment of TGM1 deficient ARCI. Orphan designation in the EU allows Krystal Biotech to benefit from a number of key incentives, including reduced regulatory fees, protocol assistance, and market exclusivity, to develop a medicine for the treatment of a rare disease affecting not more than five in 10,000 people in the European Union. KB105 was previously granted orphan drug designation by the FDA in August 2018 and is eligible for the pediatric review voucher."