Rocket Pharmaceuticals Inc（RCKT）のニュースとイベントRCKTRocket Pharmaceuticals Inc

Rocket Pharmaceuticals Announces Positive Clinical Safety Update for RP-A501

Rocket Pharmaceuticals announced a positive clinical safety update regarding the initial three patients treated under the modified protocol for its global Phase 2 trial of RP-A501 in Danon disease. Early safety has been promising with the Company now engaging with the U.S. Food and Drug Administration, FDA, to align on the pathway for treating additional patients and completing the pivotal Phase 2 trial under the modified protocol. The patients received RP-A501 at the recalibrated dose of 3.8 x 10^133 GC/kg together with a refined immunomodulatory regimen comprised of rituximab, sirolimus, and corticosteroids. Treatment proceeded sequentially, with a minimum of four weeks between infusions. To date, no thrombotic microangiopathy, TMA, capillary leak syndrome, or other significant safety concerns have been observed in these patients. The pivotal Phase 2 trial was designed as a 12-patient, single-arm study. The Company is actively engaging with the FDA to align on the path to dosing additional patients and completing the trial and expects to provide an update on the regulatory pathway in the second half of 2026.

Rocket Pharmaceuticals Closes $180M Sale of Rare Pediatric Disease PRV

Rocket Pharmaceuticals announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher, PRV, for gross proceeds of $180M. The Rare Pediatric Disease Priority Review Voucher was granted by the FDA in March 2026 in connection with the approval of Kresladi. Rocket's gene therapy for severe leukocyte adhesion deficiency-I, a rare and life-threatening primary immunodeficiency. As previously reported, before the PRV sale Rocket had cash, cash equivalents and investments of $144.4M as of March 31, 2026. Following the $180M in non-dilutive proceeds from the PRV sale, pro forma cash, cash equivalents and investments increased to approximately $322.6M, which the Company expects to fund operations into the second quarter of 2028.

Gaurav Shah: Cash and Investments Reach $144.4 Million

Cash, cash equivalents and investments as of March 31, 2026, were $144.4 million, excluding PRV monetization. "During the first quarter, we continued advancing our cardiovascular gene therapy portfolio, including reinitiating dosing in the initial three-patient cohort of our pivotal Phase 2 study of RP-A501 for Danon disease, while also achieving the accelerated approval of KRESLADI for severe LAD-I," said Gaurav Shah. "The subsequent $180 million non-dilutive monetization of our Rare Pediatric Disease Priority Review Voucher further strengthened our balance sheet and extended our expected cash runway into the second quarter of 2028. We remain focused on disciplined execution across our deep cardiovascular pipeline and anticipate providing an update on the Danon program in the second half of the year."