Rocket Pharma Stock Drops After FDA Institutes Clinical Hold On RP-A501 Danon Disease Trial

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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