Rocket Pharma - Initiation of Research Coverage by William Blair & Company Announced


From William Blair & Company: Initiation of Research Coverage for Two Gene Therapy Companies – 

William Blair & Company initiated research coverage of two gene therapy companies—Krystal Biotech, Inc. (KRYS $15.07) and Rocket Pharmaceuticals, Inc. (RCKT $19.17).

Analyst Raju Prasad said that shares of Krystal Biotech hold a positive risk/reward profile ahead of clinical data expected by the end of the year. Krystal is developing novel gene therapies for skin diseases; lead candidate KB103 is in a Phase I/II trial for the treatment of dystrophic epidermolysis bullosa (DEB), a rare and severe genetic disease affecting the skin and mucosal tissues caused by one or more mutations in the COL7A1 gene, with no currently approved therapies. KB103 leverages Krystal’s Skin TARgeted Delivery (STAR-D) platform, which consists of a proprietary engineered viral vector based on herpes simplex virus 1 (HSV-1) that is both non-integrating and non-replicating.
Prasad said that the positive risk/reward profile is “a result of KB103’s mechanism of action and preclinical data, recent proof-of-concept with gene-corrected autologous skin grafts, and Amgen’s Imlygic, an HSV-1-based product already approved for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery.”
Prasad noted that Rocket Pharmaceuticals’ lead candidate, RP-L102, is an ex vivo lentiviral gene therapy being developed for the treatment of Fanconi anemia (FA), with three additional ex vivo lentivirus programs in earlier development for the treatment of leukocyte adhesion deficiency-I (LAD-I), pyruvate kinase deficiency (PKD), and infantile malignant osteopetrosis (IMO). He also expects the company to disclose its in vivo adeno-associated virus (AAV) gene therapy indication by year end, as the company anticipates having up to four programs in the clinic (likely FA, LAD-I, PKD, AAV) and data from two (likely FA, LAD-I) in 2019.
In his report, Prasad wrote, “The company is targeting rare diseases with a focus on bone marrow health and monogenic disorders with little to no competition. We believe this sound strategic approach somewhat de-risks the company’s preclinical programs. Rocket expects to have at least four clinical programs in 2019 (FA, LAD-I, PKD, and AAV indication), with data available from at least two of them during the year (we anticipate FA and LAD-I), which we view as ambitious goals for a relatively young company, and execution on this strategy could unlock significant value creation for shareholders.”
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