Krystal Biotech Reports First Quarter 2020 Financial Results and Provides Update on Operational Progress
KB105 interim Phase 1/2 clinical data for treatment of autosomal recessive congenital ichthyosis (ARCI) to be announced at the
B-VEC Phase 3 pivotal study anticipated to start in 1H 2020
B-VEC Phase 1/2 clinical data and statistical analysis to be presented at the Society for Investigative Dermatology (SID) Annual Meeting in
KB407 in vitro pharmacology data to treat cystic fibrosis to be announced at the
Ended the quarter with cash, cash equivalents and short-term investments of
“In 2020, we anticipate treating patients across three different clinical trials for skin diseases and conditions and remain committed to developing our pipeline to ensure that that we can translate its potential into treatments for patients affected by severe diseases,” said
- Presentation of “First-in-Human use of a Novel In Vivo Gene Therapy for the Treatment of Autosomal Recessive Congenital Ichthyosis: Results of Phase 1/2 placebo controlled trial,” by Dr.
Amy S. Paller, M.D. (Chair, Department of Dermatology, Northwestern University), at the Society for Investigative Dermatology(SID) annual meeting, May 13-16 2020.
- Presentation of “In vivo correction of dystrophic epidermolysis bullosa by direct cutaneous COL7A1 gene replacement: results of Phase 1/2 trial,” by Dr.
Peter Marinkovich, M.D. (Associate Professor of Dermatology, Stanford University), at the SID annual meeting.
- Poster presentation of “In vitro Pharmacology of KB407, an HSV-1 based gene therapy vector, for the treatment of cystic fibrosis” at the
American Society of Gene & Cell Therapy(ASGCT) 23rd Annual Meeting, May 12-15, 2020.
- Commence pivotal Phase 3 study on B-VEC for the treatment of dystrophic epidermolysis bullosa and organize commercial plans for potential future global launch of B-VEC.
- Advance Phase 1/2 study to include pediatric patients on KB105 for the treatment of ARCI, following safety review by the FDA.
- Initiate Phase 1 clinical safety and efficacy study on KB301 for an aesthetic indication.
- Issuance of a new
U.S.patent covering compositions of matter and methods of cosmetic use related to our product candidate KB301 after positive decision rendered by the US Patent & Trademark Officefor U.S.Pat. App. No. 16/395,896, entitled “Recombinant Nucleic Acids Encoding Cosmetic Protein(s) for Aesthetic Applications.”
- Work to file an IND for KB104 for the treatment of Netherton Syndrome.
- Continue pre-clinical efforts on KB407 for an anticipated IND filing in 2021.
Financial results for the quarter ended March 31, 2020
- Cash, cash equivalents and short-term investments totaled $186.7 million on March 31, 2020.
- Research and development expenses for the first quarter ended
March 31, 2020were $3.5 million, compared to $3.2 millionfor first quarter 2019.
- General and administrative expenses for the first quarter ended
March 31, 2020were $2.4 million, compared to $1.5 millionfor first quarter 2019.
- Net losses for the quarters ended March 31, 2020 and 2019 were $5.3 million and $4.1 million or (
$0.31) and ( $0.29) per common share (basic and diluted), respectively.
For additional information on the Company’s financial results for the year ended
About Krystal Biotech
B-VEC (Beremagene Geperpavec, previously “KB103”) is Krystal’s lead product candidate that seeks to use gene therapy to treat dystrophic epidermolysis bullosa, or DEB, an incurable skin blistering condition caused by a lack of collagen in the skin. B-VEC is a replication-defective, non-integrating viral vector that has been engineered employing Krystal’s STAR-D platform to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. HSV-1 is Krystal’s proprietary vector that can penetrate skin cells more efficiently than other viral vectors. Its high payload capacity allows it to accommodate large or multiple genes and its low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.
About Dystrophic Epidermolysis Bullosa, or DEB
Dystrophic epidermolysis bullosa, or DEB, is an incurable, often fatal skin blistering condition caused by a lack of collagen protein in the skin. It is caused by mutations in the gene coding for type VII collagen, or COL7, a major component of the anchoring fibrils, which anchor the epidermis to the underlying dermis, and provide structural adhesion in a normal individual. The lack of COL7 in DEB patients causes blisters to occur in the dermis as a result of separation from the epidermis. This makes the skin incredibly fragile, leading to blistering or skin loss at the slightest friction or knock. It is progressive and incredibly painful.
The most severe form of DEB is recessive DEB, or RDEB, which is caused by null mutations in the COL7A1 gene. DEB also occurs in the form of dominant DEB, or DDEB, which is considered to be a milder form of DEB. There are no known treatments, which affect the outcome of either form of the disease, and the current standard of care for DEB patients is limited to palliative treatments. Krystal is developing KB-103 for the treatment of the broad DEB population, including both recessive and dominant forms of the disease.
About Autosomal Recessive Congenital Ichthyosis, or ARCI
Transglutaminase 1 (TGM-1) is an essential epidermal enzyme that facilitates the formation of the epidermal barrier, which prevents dehydration, and protects the skin from unwanted toxins and surface microorganisms. The loss of TGM-1-activity results in the severe genetic skin disease autosomal recessive congenital ichthyosis (ARCI). Most patients with a TGM-1-deficiency exhibit life-long pronounced scaling with increased transepidermal water loss (TEWL). The scales are plate-like, often of a dark color, and cover the whole body surface area. Erythroderma is either absent or minimal. Such patients usually have ectropion and, at times, eclabium, hypoplasia of joint and nasal cartilage, scarring alopecia, especially at the edge of the scalp, and palmoplantar keratoderma. Additional complications include episodes of sepsis, fluid and electrolyte imbalances due to impaired skin barrier function, and failure to thrive, especially during neonatal period and infancy. Severe heat intolerance, and nail dystrophy are also frequently observed. TGM-1-deficient ARCI is associated with increased mortality in the neonatal period and has a dramatic impact on quality of life. No efficient treatment is available; current therapy only relieves some symptoms.
About the STAR-D Gene Therapy Platform
Krystal’s Skin TARgeted Delivery platform, or STAR-D platform, is a proprietary gene therapy platform consisting of an engineered viral vector and skin-optimized gene transfer technology that Krystal is employing to develop off-the-shelf treatments for dermatological diseases for which there are no known effective treatments. The company believes that the STAR-D platform provides an optimal approach for treating dermatological conditions due to the nature of the HSV-1 viral vector it has created. Certain inherent features of the HSV-1 virus, combined with the ability to strategically modify the virus in the form employed as a gene delivery backbone, provide the STAR-D platform with several advantages over other viral vector platforms for use in dermatological applications.
Any statements in this press release about future expectations, plans and prospects for
Ashley R. Robinson
Source: Krystal Biotech, Inc.