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Dec 31, 2021
Rocket Q4 2021 Earnings Report
Rocket Pharmaceuticals reported financial and operational results for Q4 and full year 2021, highlighting progress in gene therapy programs and a strong cash position.
Key Takeaways
Rocket Pharmaceuticals reported a net loss of $44.2 million for the fourth quarter of 2021. The company highlighted positive clinical data from ongoing trials for Danon Disease, Leukocyte Adhesion Deficiency-I (LAD-I), and Fanconi Anemia (FA). They anticipate in-house AAV cGMP manufacturing readiness in Q2 2022 and expect their cash resources to fund operations into the second half of 2023.
Reported positive Phase 1 data from Danon Disease gene therapy trial, demonstrating sustained benefit across clinical, functional, and biomarker endpoints.
Reported engraftment in all nine severe LAD-I patients treated in pivotal Phase 2 trial.
Demonstrated evidence of engraftment in six Fanconi Anemia patients in pivotal Phase 2 trial.
Company's cash position was $388.7 million, expected to fund operations into 2H’23.
Forward Guidance
Rocket Pharmaceuticals anticipates several milestones in 2022, including reporting data from pediatric patient cohort of Phase 1 trial for Danon Disease in Q3 2022, reporting top-line data from pivotal Phase 2 trial for Leukocyte Adhesion Deficiency-I (LAD-I) in Q2 2022 and for Fanconi Anemia (FA) in Q3 2022. They also expect to achieve in-house AAV current Good Manufacturing Practice (cGMP) manufacturing readiness in Q2 2022.
Positive Outlook
- Report data from pediatric patient cohort of Phase 1 trial for Danon Disease in Q3 2022.
- Initiate pivotal Phase 2 trial activities for Danon Disease in Q4 2022.
- Report top-line data from pivotal Phase 2 trial for Leukocyte Adhesion Deficiency-I (LAD-I) in Q2 2022.
- Report top-line data from pivotal Phase 2 trial for Fanconi Anemia (FA) in Q3 2022.
- Achieve in-house AAV current Good Manufacturing Practice (cGMP) manufacturing readiness in Q2 2022.
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