Krystal Biotech (NASDAQ:KRYS) Portfolio, Stock and Financial Analysis

Krystal Biotech Company Highlights

- Biotechnology company that specializes in gene therapy 
- Utilizes proprietary HSV virus for delivery of genes to the target tissue
- Multiple clinical programs and early research  pipeline including  dystrophic epidermolysis bullosa, autosomal recessive congenital ichthyosis & cystic fibrosis
- Cash, cash equivalents, and short-term investments of $286.4 million as of September 30, 2020

Krystal Biotech, Inc. is a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from rare diseases.

Krystal Biotech Platform

Skin TARgeted Delivery platform, or STAR-D, that consists of an engineered HSV-1 vector and skin optimized gene transfer technology, suitable for introducing into the patient one or more therapeutic genes relevant to treating skin disease. The modified HSV-1 is a replication-defective, non-integrating viral vector that can efficiently penetrate a broad range of skin cells.

Stock Symbol: (NASDAQ:KRYS)

Krystal Biotech Portfolio

Beremagene Geperpavec (B-VEC) for Dystrophic epidermolysis bullosa (DEB)
DEB is a fatal skin blistering condition caused by mutations in the gene encoding type VII collagen, or COL7, a protein that provides critical structural adhesion between skin layers in a normal individual. Beremagene geperpavec (B-VEC or KB103) is a replication-defective, non-integrating HSV-1 engineered to deliver wild-type human type VII collagen genes directly to a DEB patient's dividing and non-dividing skin cells, and providing gene correction of the underlying genetic deficiency. The enrollment of the pivotal GEM-3 study is ongoing and anticipated to complete in early 2021.

KB105 for TGM1-Deficient Autosomal Recessive Congenital Ichthyosis (TGM1-ARCI)
KB105 uses the STAR-D technology to deliver functional TGM1 genes directly to the skin in order to express the transgultaminase-1 enzyme therein. Phase 1/2 study of KB105 in patients with TGM1 deficient autosomal recessive congenital ichthyosis (ARCI) is ongoing. Update is expected in H12020

KB407 for Cystic Fibrosis
Cystic fibrosis (CF), the most common inherited genetic disorder in the United States, is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR). Lack of functional CFTR in secretory airway epithelia results in defective Cl-, bicarbonate, and thiocyanate secretion, coupled with enhanced Na+ absorption and mucus production, leading to dehydration and acidification of the airway surface liquid. The in vivo data from the ongoing preclinical development of KB407 showed successful nebulization, distribution of vector and functional protein expression throughout the lung in mice and a nonhuman primate. Pre-clinical validation work is ongoing, and the Company is on track to initiate a clinical study in the first half of 2021.

KB301 for Aesthetic Indications
Positive preclinical data supporting the ongoing development of KB301 in aesthetic indications supported clinical development. Initial Phase 1 safety data from this study is anticipated in early 2021, followed by initial efficacy data in 2H21.

KB104 for Netherton Syndrome
KB104, is engineered to deliver functional SPINK5 directly to the skin of patients with Netherton Syndrome. KB104 utilizes the STAR technology for gene delivery. The company continues to work towards an IND filing, which is anticipated in 2021.

Kyrstal Biotech Financial Highlights Q3 2020

  • Cash, cash equivalents and short-term investments totaled $286.4 million on September 30, 2020.
  • Research and development expenses for the third quarter ended September 30, 2020 were $5.1 million, compared to $3.9 million for third quarter 2019.
  • General and administrative expenses for the third quarter ended September 30, 2020 were $4.6 million, compared to $1.5 million for third quarter 2019.
  • Net losses for the quarters ended September 30, 2020 was $9.6 million