William Blair launched coverage of Rocket Pharmaceuticals (NASDAQ:RCKT) with an “outperform” rating and fair value estimate of $32. The stock finished at $19.17 on July 9.
Rocket is focused on developing first-in-class gene therapies for rare diseases. Its lead candidate, RP-L102, is an ex vivolentiviral gene therapy being developed for the treatment of fanconi anemia (FA), with three additional ex vivolentivirus programs in earlier development for the treatment of leukocyte adhesion deficiency-I (LAD-I), pyruvate kinase deficiency (PKD), and infantile malignant osteopetrosis (IMO).
“We also expect the company to disclose itsin vivoadeno-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,” writes analyst Raju Prasad.
At the 2018 American Society of Gene and Cell Therapy, Rocket presented an RP-L102 Phase 1/2 update that Mr. Prasad said established proof-of-concept of the lentivirus platform and the potential for better outcomes with an optimized process and longer follow-up.
He said the company anticipates dosing more patients using the refined process in the near term, with a pivotal trial potentially beginning in 2019, using commercial manufacturing.
