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RCKT-News
RCKT-Events
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."
Rocket Pharmaceuticals Sells PRV for $180 Million
Rocket Pharmaceuticals announced a strengthened financial position following the sale of its Rare Pediatric Disease Priority Review Voucher, PRV. Rocket has entered into a definitive agreement to sell its PRV for $180 million. The PRV was awarded following the U.S. Food and Drug Administration, FDA, accelerated approval of Kresaldi. The transaction reflects the continued strategic value of PRVs, as well as Rocket's disciplined approach to capital formation and allocation in support of its prioritized cardiovascular gene therapy programs. "The monetization of our PRV, following the FDA approval of Kresaldi, provides meaningful non-dilutive capital and extends our cash runway into the second quarter of 2028," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. "This strengthens our ability to advance key clinical milestones across our cardiovascular gene therapy pipeline, with all programs on track."
Rocket Pharmaceuticals Receives Accelerated Approval for Kresladi
Rocket Pharmaceuticals announced that the U.S. Food and Drug Administration, FDA, has granted accelerated approval for Kresladi, an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant. This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression. Confirmation of clinical benefit will be based on the evaluation of longer-term follow-up data of treated patients in the ongoing clinical study and through a post-marketing registry. With the approval of Kresladi, the FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher, a program designed to encourage development of therapies for rare pediatric diseases. The Company intends to evaluate strategic options to monetize the PRV in a manner designed to enhance financial flexibility and maximize shareholder value.
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