Business
Krystal Biotech Reports Third Quarter 2020 Financial Results and Provides Update on Operational Progress
- Pivotal GEM-3 study of B-VEC in DEB expected to complete enrollment in early 2021 - Initiated the Phase 1 Study of KB301 in Facial Wrinkles and Acne Scars
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"\n- Pivotal GEM-3 study of B-VEC in DEB expected to complete enrollment in early 2021\n\n- Initiated the Phase 1 Study of KB301 in Facial Wrinkles and Acne Scars\n\n- Today announced additional in vivo preclinical data supporting the development of KB407 in cystic fibrosis\n\n- Strong balance sheet with cash, cash equivalents and short-term investments of $286.4 million as of September 30, 2020\n\n PITTSBURGH--(BUSINESS WIRE)--\nKrystal Biotech, Inc. (Nasdaq:KRYS), a fully integrated gene therapy company driven by its proprietary, engineered herpes simplex virus type 1 vector (HSV-1) platform, today reported financial results and key operational progress updates for the third quarter ended September 30, 2020.\n\n“Despite the challenges presented by COVID in this quarter, we initiated a pivotal trial for DEB, a phase 2 trial in ARCI and a phase 1 trial in aesthetic skin conditions, and I thank my entire team for their efforts in these difficult times,” said Krish Krishnan, Chairman and CEO of Krystal Biotech, Inc. “I am particularly encouraged by progress with KB407 for the treatment of cystic fibrosis. The in vivo animal data reported today demonstrates our ability to deliver functional CFTR throughout the lung, which is encouraging in this indication and gives us confidence to explore additional diseases of the lung where delivery of a therapeutic transgene may be beneficial,” he added.\n\nHe further noted, “With the granting of Rare Pediatric Designation for this program by the FDA this quarter, we are now eligible to receive a Priority Review Voucher for KB407 as well as for B-VEC, KB105, KB104.”\n\nProgram Highlights & Upcoming Events\n\nBeremagene Geperpavec (B-VEC) for DEB \n\n\nEnrollment in the ongoing pivotal GEM-3 study is proceeding well and enrollment completion is anticipated in early 2021. The trial is a randomized, double-blind, intra patient placebo-controlled multicenter trial designed to evaluate the efficacy and safety of B-VEC for patients suffering from both recessive and dominant dystrophic forms of Epidermolysis Bullosa.\n\n\nDetails of the pivotal study can be found at www.clinicaltrials.gov under NCT identifier NCT04491604.\n\n\nTop line data and BLA filing are anticipated in 2021, in line with prior guidance. Data from this trial will also form the basis of an MAA filing in the EU which is anticipated t...