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Mar 31, 2021
Rocket Q1 2021 Earnings Report
Rocket Pharmaceuticals reported financial results for the first quarter of 2021 and provided updates on key pipeline developments.
Key Takeaways
Rocket Pharmaceuticals reported a net loss of $40.2 million for the first quarter of 2021. The company's cash, cash equivalents, and investments totaled $466.4 million, expected to fund operations into the second half of 2023. Key pipeline developments include pausing the RP-A501 Danon Disease program for additional risk mitigation and advancing the fifth gene therapy program in Infantile Malignant Osteopetrosis into patient dosing.
RP-A501 Danon Disease program paused for additional risk mitigation requested by the FDA.
Low Dose RP-A501 treatment demonstrates durable expression and improvements in biomarkers.
RP-L301 for PKD shows normalization of hemoglobin levels in preliminary Phase 1 data.
First patient dosed with RP-L401 in Infantile Malignant Osteopetrosis Phase I trial.
Rocket
Rocket
Forward Guidance
Rocket Pharmaceuticals expects its cash, cash equivalents and investments to fund operations into the second half of 2023.
Positive Outlook
- Advancement of five clinical programs
- Buildout and initiation of AAV cGMP manufacturing capabilities
- Updated “Process B” data for Fanconi Anemia (RP-L102) in Q2 2021
- Longer-term Phase 2 data for LAD-I (RP-L201) in Q3 2021
- Initial Phase 1 data for IMO (RP-L401) in Q3 2021
Challenges Ahead
- RP-A501 Danon Disease program paused for additional risk mitigation
- Anticipate delay in enrollment by one quarter for Danon Disease program
- Dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates
- Actions of regulatory agencies may affect the initiation, timing and progress of pre-clinical studies and clinical trials
- Unexpected expenditures