On Monday, Rocket Pharmaceuticals, Inc. RCKT presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.Danon disease is a rare X-linked dominant genetic disorder that manifests with the clinical triad of cardiomyopathy (stiff heart muscles), skeletal myopathy (weakness), and intellectual disability.Also Read: FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock SlidesDanon disease is caused by mutatio ...

Rocket Pharmaceuticals, Inc. (RCKT) incurred a loss of 71 cents per share in the third quarter of 2024, narrower than the Zacks Consensus Estimate of a loss of 78 cents. In the year-ago quarter, the company had reported a loss of 75 cents per share.In the absence of any marketed product in its portfolio, Rocket Pharmaceuticals did not record any revenues during the third quarter.Stay up-to-date with all quarterly releases: See Zacks Earnings Calendar.More on RCKT's Q3 ResultsIn the reported quarter, general ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-36829 Rocket Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) Delaware 04-3475813 (State ...

Exhibit 99.1 Rocket Pharmaceuticals Reports Third Quarter 2024 Financial Results and Highlights Recent Progress Enrollment completed in September and dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease Updated Phase 1 data from RP-A501 for Danon disease anticipated at American Heart Association's 2024 Late-Breaking Scientific Sessions Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Preliminary data expected in the first half of 2025 Rolling BLA submissi ...

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Rocket Pharmaceuticals, Inc. (RCKT) announced that it has completed patient enrollment in a pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating male patients with Danon disease. Danon disease is a rare X-linked inherited disorder caused by mutations in the LAMP2 gene. Per the company, an estimated 15,000 to 30,000 patients suffer from Danon disease in the United States and Europe. Currently, the only available treatment for the disease is cardiac transplantati ...

Shares of Rocket Pharmaceuticals (RCKT) have plunged 32.9% in the past six months compared with the industry's decline of 3.4%. The company is developing its gene therapy candidate, Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder. In June 2024, the FDA issued a complete response letter (CRL) to Rocket's biologics license application (BLA) seeking approval for Kresladi to treat LAD-I. Per the CRL, the FDA sought "limited" add ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-36829 Rocket Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) Delaware 04-3475813 (State or o ...

Rocket Pharmaceuticals Reports Second Quarter 2024 Financial Results and Highlights Recent Progress Enrollment of patients ongoing in the pivotal Phase 2 study of RP-A501 for the treatment of Danon disease and the Phase 1 study of RP-A601 to treat PKP2 arrhythmogenic cardiomyopathy Working toward FDA-approval of KRESLADI for severe LAD-I; Commercial infrastructure and capabilities in place for launch Presented long-term KRESLADITM follow-up data from the global Phase 1/2 study for severe LAD-I, results from ...

Rocket Pharmaceuticals (RCKT) announced that the FDA issued a complete response letter (CRL) to its biologics license application (BLA) seeking approval for its gene therapy Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder. Per the CRL, the FDA sought 'limited' additional information on the therapy's Chemistry Manufacturing and Controls (CMC), which is a part of the BLA submission. The goal of CMC is to ensure that every batc ...