Rocket Q1 2021 Earnings Report
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