- Tucked in the Q1 earnings release, Rocket Pharmaceuticals Inc RCKT has paused the RP-A501 Phase 1 trial to treat Danon Disease to address the FDA request for additional risk mitigation methods in the protocol.
- The company says that the enrollment will delay by one quarter. No new safety events were observed.
- The agency asked to stop patient dosing and modify the protocol and other supporting documents with revised guidelines for patient selection and management.
- Rocket Pharma updated low dose results in RP-A501 for Danon disease, demonstrating durable expression and ongoing improvements in biomarkers, and evidence of a positive risk/benefit profile out to 18-months.
- Full data to be presented in Q4 2021.
- For Q1, the company ended the period with cash and equivalents of $466.4 million, expected to fund its operations into the second half of 2023.
- It posted a net loss of $40.2 million or $0.65 per share, higher than the $24.7 million or $0.45 loss posted a year ago.
- Price Action: RCKT shares are down 8.3% at $39.75 during the market trading session on the last check Tuesday.
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