News Release Details
Rocket Pharmaceuticals Announces Updated Positive Preliminary Clinical Data from Phase 1 Trial of RP-L301 for the Treatment of Pyruvate Kinase Deficiency
Mar 22, 2021
— Updated Data Demonstrate Sustained Improvements in Hemoglobin to
— Maintained Safety and Tolerability of RP-L301 up to 6-months Post-treatment —
— Long-Term Data on Track for 2H2021 —
“The positive updates on our PKD trial represent continued validation of RP-L301’s robust potential benefit in addressing the challenges of PKD. We are pleased to report that updated patient 1 results show that RP-L301 continues to safely normalize hemoglobin levels as demonstrated by an increase from an average of approximately 7.4 g/dL at baseline to 13.9 g/dL at 6-months post-treatment with no transfusion requirements after hematopoietic reconstitution,” said
Key findings are highlighted below. Further information about the study can be found here.
Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients
Two adult patients with significant anemia and transfusion requirements were treated with RP-L301, Rocket’s ex vivo lentiviral gene therapy candidate for PKD. Respectively, patients L301-006-1001 and L301-001-1002 were 31- and 47-years old at the time of enrollment and have been followed for 6- and 3-months post treatment at the time of data cutoff.
- RP-L301 continued to be well tolerated, with no serious safety issues or infusion-related complications observed up to 6-months post treatment
Patient L301-006-1001 received a cell dose of 3.9x106 cells/kilogram (kg)
- Normalized hemoglobin levels from an average baseline of ~7.4 grams (g)/deciliter (dL) to 13.9 g/dL at 6-months post treatment with RP-L301
- Significant improvement of bilirubin, which had been substantially elevated prior to study enrollment
Patient L301-001-1002 received a cell dose of 2.4x106 cells/kg
- Normalized hemoglobin levels from a baseline of ~7.0 g/dL to 13.8 g/dL at 3-months post treatment with RP-L301
- Normalization of bilirubin, which had been substantially elevated prior to study enrollment
About Pyruvate Kinase Deficiency
Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red blood cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in
RP-L301 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD).
Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended
SVP, Strategy & Corporate Development