Krystal Biotech reports news on its commercial genetic-medicines business, led by VYJUVEK, a redosable gene therapy approved in the United States, Europe, and Japan for dystrophic epidermolysis bullosa. Company updates commonly cover VYJUVEK revenue and global commercialization, manufacturing and platform developments, and regulatory actions tied to its HSV-1-based gene delivery technology.
Recurring clinical and scientific updates address investigational programs including KB407 for cystic fibrosis, KB707 for advanced or metastatic non-small cell lung cancer, KB111 for Hailey-Hailey disease, KB803 for corneal abrasions in DEB patients, and additional HSV-1-based vectors. News also includes quarterly operating results, conference presentations, FDA designations, and investor-event participation.
Krystal Biotech reported financial results for Q1 2021, highlighting a strong balance sheet with cash, cash equivalents, and short-term investments totaling $403.4 million. The company completed enrollment in the pivotal GEM-3 study for B-VEC in dystrophic epidermolysis bullosa (DEB). Anticipated top-line data is expected in 4Q21. Additionally, Krystal is on track to provide updates for their KB105 and KB407 programs in 1H21 and 3Q21, respectively. Notably, R&D expenses increased to $6.2 million, and the net loss for the quarter was $15.8 million ($0.74 per share).
Krystal Biotech (NASDAQ: KRYS) has appointed Andy Orth as Chief Commercial Officer. With over 25 years of experience in biotechnology, Orth previously led U.S. operations at Alnylam Pharmaceuticals. His expertise in launching genetic medicines is seen as vital for Krystal’s growth, especially for its investigational program B-VEC aimed at treating dystrophic epidermolysis bullosa (DEB). The company aims to enhance its commercial capabilities to bring essential treatments to patients.
On April 27, 2021, Krystal Biotech (NASDAQ: KRYS) announced a digital poster presentation at the American Society of Genetic & Cell Therapy (ASGCT) Annual Meeting from May 11-14, 2021. The presentation will showcase preclinical data from its vector-encoded antibody platform, highlighting in vitro efficacy with KB501 and KB502 targeting antibodies for atopic dermatitis. The session is scheduled for May 11, 2021. Suma Krishnan, COO of Krystal, emphasized the potential of their approach for treating chronic conditions.
Krystal Biotech (NASDAQ: KRYS) announced updates to the Statistical Analysis Plan for its Phase 3 GEM-3 study of B-VEC, a treatment for dystrophic epidermolysis bullosa (DEB), following FDA guidance. The randomized trial aims to evaluate the safety and efficacy of B-VEC, with 31 enrolled patients. Adjustments made include the primary outcome measure of complete wound healing at specific weeks and the use of the McNemar test for analysis. Previous studies showed promising results with a 78.6% healing rate in B-VEC treated wounds. The trial continues to be double-blind until data lock in Q4 2021.
Krystal Biotech (NASDAQ: KRYS) announced positive results from a GLP toxicology study of its inhaled gene therapy KB407 for cystic fibrosis, showing it was well-tolerated in nonhuman primates. Key findings include a high No-Observed-Adverse-Effect Level (NOAEL) and successful distribution in lung tissues. The company is also advancing KB408 for alpha-1 antitrypsin deficiency, with promising initial preclinical results. Krystal plans to initiate a Phase 1 study of KB407 in 3Q21 and will present more detailed data for KB408 at an upcoming scientific conference.
Krystal Biotech (NASDAQ: KRYS) has completed enrollment for the pivotal GEM-3 study of B-VEC, a treatment for dystrophic epidermolysis bullosa (DEB). The study enrolled 31 patients, with 61% being pediatric. The company plans to report top-line results in Q4 2021, marking a significant milestone for potential treatment options for DEB. B-VEC is designed to address the root cause of DEB through a topical application in a double-blind, placebo-controlled setting.
Krystal Biotech (NASDAQ: KRYS) has announced its participation in two virtual conferences. The first is the Guggenheim Healthcare Talks: 2021 Genomic Medicines & Rare Disease on April 1, where Krystal's leadership will join a panel discussion at 4:00 PM ET. The second event is the 2021 Virtual Cell & Gene Meeting on the Mediterranean from April 6-9, featuring on-demand presentations. Krystal focuses on redosable gene therapies for rare diseases and is committed to developing innovative treatments.
Krystal Biotech Inc. (NASDAQ: KRYS) launched a new subsidiary, Jeune, Inc., focusing on aesthetic gene therapies. Initial data from the Phase 1 PEARL-1 study for their lead candidate, KB301, indicates safety and tolerability following repeat injections. Results from seven healthy subjects showed no safety signals, with mild, transient adverse events. KB301 effectively expressed COL3A1 collagen gene, crucial for skin health. Jeune plans to advance KB301 and other products targeting aging skin, with detailed data to be presented at the SID Annual Meeting in May.
Krystal Biotech reported key operational updates and financial results for Q4 2020. The company anticipates completing enrollment in the pivotal B-VEC GEM-3 study in Q1 2021, with topline data expected in Q4 2021. The European Medicines Agency granted orphan drug designation for KB407, aimed at treating cystic fibrosis. As of December 31, 2020, the company had a strong balance sheet with $271.3 million in cash and equivalents. Net losses for 2020 totaled $32.2 million, with a loss per share of $(1.71). A public offering in February raised $135 million in net proceeds.
Krystal Biotech Inc. (NASDAQ: KRYS) announced that Krish S. Krishnan, CEO, will participate in a fireside chat at the 41st Annual Cowen Health Care Conference from March 1-4, 2021. The presentation is scheduled for March 4, 2021, at 9:50 AM EST. Interested individuals can access the webcast via this link. A recording will be available for 90 days on the company’s website. Krystal Biotech focuses on developing therapies for rare diseases through its innovative gene therapy platform.