Ladenburg Thalmann launched coverage of Rocket Pharmaceuticals (NASDAQ:RCKT) with a “buy” rating and $30 price target. The stock closed at $23.96 on Sept. 12.
Rocket is developing potentially curative first-in-class gene therapies for rare monogenic genetic diseases.
Analyst Wangzhi Li writes that the company has licensed from leading academic institutes a large pipeline of four ex-vivolentiviral (LV) gene therapy programs for fanconi anemia (FA), leukocyte adhesion deficiency-I (LAD-I), pyruvate kinase deficiency (PKD) and infantile malignant osteopetrosis (IMO), as well as one in vivo AAV gene therapy for a monogenic multi-organ disease.
Lead program, RP-L102 for FA (about 2,500 patients in the U.S. plus EU with early bone marrow failure and high risk of cancer) is in a Phase 1 trial that has already showed early proof-of-concept data and expects to advance into a potential registration trial in 2019, he added.
Mr. Li said RP-L201 for LAD-I (ultra-rare) and RP-L301 for PKD (about 32,000 patients in the U.S. plus EU) also are expected to enter the clinic in the first half of 2019.
An IND filing for RP-L401 for IMO (ultra-rare) is expected in 2020, he said, adding that the AAV program, RP-A501, is expected to enter the clinic in the first half of 2019, with its identity and indication (15,000-30,000 patients in the U.S. plus EU) to be disclosed in the fourth quarter of 2018.
“We see Rocket as on track to have four gene therapy programs in the clinic by the end of 2019,” Mr. Li said.
