RCKT Rocket Pharmaceuticals Inc.

32.31
-0.25  -1%
Previous Close 32.56
Open 32.78
52 Week Low 22.035
52 Week High 67.48
Market Cap $2,081,725,513
Shares 64,429,759
Float 44,222,036
Enterprise Value $1,690,997,381
Volume 81,879
Av. Daily Volume 513,700
Stock charts supplied by TradingView

Upcoming Catalysts

Drug Stage Catalyst Date
RP-A501
Danon disease
Phase 1
Phase 1
Premium membership is required to view catalyst dates, analyst ratings, earnings dates and cash burn data. Click here to unlock and sign up to a 14-day FREE TRIAL.
RP-L201
Leukocyte Adhesion Deficiency-I (LAD-I)
Phase 1/2
Phase 1/2
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
RP-L301
Pyruvate Kinase Deficiency (PKD)
Phase 1
Phase 1
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
RP-L401
Infantile Malignant Osteopetrosis (IMO)
Phase 1
Phase 1
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
RP-L102
Fanconi Anemia (FA)
Phase 2
Phase 2
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.

Drug Pipeline

Drug Stage Notes
Trabodenoson
Primary open-angle glaucoma (POAG)
Phase 3
Phase 3
Phase 3 top-line data released January 3, 2017. Primary endpoint not met.
Trabodenoson and latanoprost
Glaucoma
Phase 2
Phase 2
Phase 2 trial failed - noted July 7, 2017.

Latest News

  1. Previously disclosed positive data from the low-dose (6.7e13 vg/kg) cohort of the ongoing Phase 1 trial presented at HFSA's Annual Scientific Meeting 2021 —

    Data demonstrated RP-A501 was well tolerated and showed increasing and durable benefit —

    Low-dose pediatric patient cohort expected to commence in Q3; full update anticipated Q4 —

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces an oral presentation of previously disclosed data from the ongoing Phase 1 trial of RP-A501 for the treatment of Danon Disease was given at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021. The…

    Previously disclosed positive data from the low-dose (6.7e13 vg/kg) cohort of the ongoing Phase 1 trial presented at HFSA's Annual Scientific Meeting 2021 —

    Data demonstrated RP-A501 was well tolerated and showed increasing and durable benefit —

    Low-dose pediatric patient cohort expected to commence in Q3; full update anticipated Q4 —

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces an oral presentation of previously disclosed data from the ongoing Phase 1 trial of RP-A501 for the treatment of Danon Disease was given at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021. The presentation overviewed data from the low-dose (6.7e13 vg/kg) cohort which demonstrated the investigational gene therapy RP-A501 was well tolerated and showed progressive and durable clinical benefit.

    "Patients with Danon Disease desperately need a treatment option that provides meaningful therapeutic benefit with a manageable safety profile, and we are excited to be a part of the HFSA Annual Meeting and to share this important work," said Jonathan Schwartz, M.D., chief medical officer of Rocket Pharma. "We continue to be encouraged by the safety and durable clinical benefit RP-A501 has demonstrated in the low dose, with one patient now followed to 24 months. Additionally, we are working expeditiously to commence treatment in the low-dose (6.7e13 vg/kg) pediatric patient cohort in the third quarter and expect to provide a comprehensive clinical update from the Phase 1 trial in the fourth quarter."

    Data from the Low-Dose Adult Cohort (n=3) of the Phase 1 Trial of RP-A501 in Danon Disease

    • RP-A501 was generally well tolerated with a manageable safety profile. All observed adverse effects were transient and reversible with no lasting clinical sequelae. Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.
    • All three low-dose patients demonstrated evidence of sustained cardiac LAMP2B expression by Western blot and/or immunohistochemistry with decreased vacuoles and improved tissue architecture on electron microscopy.
    • All three patients demonstrated improvements in the 6 minute walk test (6MWT). One patient improved from a pretreatment baseline of 443 meters (m) to 467 m at 24 months. The second patient improved from a pretreatment baseline of 405 m to 410 m at 18 months. The third patient improved from a pretreatment baseline of 427 m to 435 m at 15 months.
    • An improvement in NYHA class in two of the three low-dose patients was observed. In these two patients, a substantial improvement of a key marker of heart failure, B-type natriuretic peptide (BNP), was also observed. BNP decreased from a pretreatment baseline by 75 percent in one patient and 79 percent in the other. The third patient has demonstrated stabilization of NYHA class and BNP.

    About RP-A501

    RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure. It consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a functional version of the human LAMP2B transgene (AAV9.LAMP2B). RP-A501 is currently being evaluated in an ongoing Phase 1 clinical trial, from which preliminary data of the low-dose cohort showed it was generally well tolerated and provided evidence of improved cardiac function in patients.

    About Danon Disease

    Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and Europe. The only available treatment option for Danon Disease is cardiac transplantation, which is associated with substantial complications and is not considered curative. There are no specific therapies available for the treatment of Danon Disease.

    About Rocket Pharmaceuticals, Inc.

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket") is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The company's platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit www.rocketpharma.com.

    Rocket Cautionary Statement Regarding Forward-Looking Statements

    Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2021 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, Rocket's plans for the advancement of its Danon Disease program following the lifting of the FDA's clinical hold and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket's ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

    View Full Article Hide Full Article
  2. —Oral presentation on previously disclosed data from the ongoing Phase 1 trial of RP-A501—

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that previously disclosed data from the ongoing Phase 1 trial of RP-A501 in Danon Disease will be presented in a late-breaking oral presentation at the upcoming Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021. The meeting will take place September 10-13, 2021 at the Gaylord Rockies Hotel and Conference Center in Aurora, Colorado.

    Details for Rocket's oral presentation are as follows:

    Title: Results From First-In-Human Clinical Trial Of RP-A501…

    —Oral presentation on previously disclosed data from the ongoing Phase 1 trial of RP-A501—

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that previously disclosed data from the ongoing Phase 1 trial of RP-A501 in Danon Disease will be presented in a late-breaking oral presentation at the upcoming Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021. The meeting will take place September 10-13, 2021 at the Gaylord Rockies Hotel and Conference Center in Aurora, Colorado.

    Details for Rocket's oral presentation are as follows:

    Title: Results From First-In-Human Clinical Trial Of RP-A501 (AAV9:LAMP2B) Gene Therapy Treatment For Danon Disease

    Session Title: Late Breaking Clinical Trials

    Session Date: Monday, September 13, 2021

    Presentation Time: 11:00 a.m. - 12:00 p.m. MDT

    Presenter: Barry Greenberg, MD, FHFSA, Distinguished Professor of Medicine at UC San Diego School of Medicine and director of the Advanced Heart Failure Treatment Program at the Cardiovascular Institute at UC San Diego Health

    About RP-A501

    RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure. It consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a functional version of the human LAMP2B transgene (AAV9.LAMP2B). RP-A501 is currently being evaluated in an ongoing Phase 1 clinical trial, from which preliminary data of the low-dose cohort showed it was generally well tolerated and provided evidence of improved cardiac function in patients.

    About Danon Disease

    Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and the Europe. The only available treatment option for Danon Disease is cardiac transplantation, which is associated with substantial complications and is not considered curative. There are no specific therapies available for the treatment of Danon Disease.

    About Rocket Pharmaceuticals, Inc.

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket") is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The company's platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit www.rocketpharma.com.

    Rocket Cautionary Statement Regarding Forward-Looking Statements

    Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2021 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, Rocket's plans for the advancement of its Danon Disease program following the lifting of the FDA's clinical hold and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket's ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

    View Full Article Hide Full Article
  3. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Isabel Carmona, J.D., as chief human resources officer and senior vice president. Ms. Carmona brings more than 25 years of experience in human resources and organizational leadership including the last 15 years within the life sciences industry. Ms. Carmona will be responsible for all aspects of human resources, including talent acquisition, talent management and organizational development, and will serve as a key part of the leadership team.

    "I am delighted to welcome Isabel to Rocket and believe her rich experience leading organizational…

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Isabel Carmona, J.D., as chief human resources officer and senior vice president. Ms. Carmona brings more than 25 years of experience in human resources and organizational leadership including the last 15 years within the life sciences industry. Ms. Carmona will be responsible for all aspects of human resources, including talent acquisition, talent management and organizational development, and will serve as a key part of the leadership team.

    "I am delighted to welcome Isabel to Rocket and believe her rich experience leading organizational growth at life science companies will be a tremendous asset as we continue our exciting growth trajectory," said Gaurav Shah, M.D., chief executive officer of Rocket Pharma. "Her success in talent acquisition, talent management and organizational development at rapidly changing and expanding life science companies will be instrumental as we continue to add key capabilities and teams to support our long-term growth plans."

    Prior to joining Rocket, Ms. Carmona was chief human resources officer of Ichnos Sciences. There, she led talent and organizational development during the spin-off, setting up and growing Ichnos Sciences as an independent oncology biotechnology company with headquarters in the U.S. and research centers around the world. Prior to that, she served in positions of increasing leadership in the global operations and human resources teams at Teva Pharmaceuticals and Shire Pharmaceuticals.

    "Joining the Rocket family at such an exciting time is an incredible opportunity," said Ms. Carmona. "I am honored to be part of bringing potentially revolutionary gene therapy cures to patients living with rare, devastating diseases. I look forward to working with this tremendous leadership team to expand key capabilities while maintaining our passionate culture and entrepreneurial spirit."

    Ms. Carmona has developed her career across the U.S. and Europe, where she has held leadership positions in legal, compliance, operational excellence and human resources in multiple industries including life sciences, information technology and financial services. Ms. Carmona earned her J.D. degree with a specialty in business and international law from the Universidad de Sevilla in Seville, Spain.

    About Rocket Pharmaceuticals Leadership

    The Rocket leadership team is comprised of seasoned industry veterans with rich and extensive experience in drug development and approvals across public and private pharmaceutical and biotechnology companies of various sizes. Leadership team members have led the development and approval of numerous gene therapy and rare disease drugs on the market for previously unmet medical needs and have served in significant roles within the U.S. Food and Drug Administration (FDA). Uniquely, Rocket's multi-platform gene therapy approach necessitates the leadership of two Chief Development Officers, one specializing in lentiviral vector (LVV)-based gene therapy and a second specializing in adeno-associated virus (AAV)-based gene therapy.

    About Rocket Pharmaceuticals, Inc.

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket") is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The company's platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit www.rocketpharma.com.

    View Full Article Hide Full Article
  4. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces a securities purchase agreement with a fund affiliated with RTW Investments, LP, the Company's largest shareholder, for the purchase of an aggregate of 812,516 shares of common stock, par value $0.01 per share, at a purchase price of $32.48 per share, the closing price on August 27, 2021, for aggregate gross proceeds of approximately $26.4 million to the Company before offering expenses. The private placement is expected to close on or about August 31, 2021, subject to the satisfaction of customary closing conditions. Rocket expects to use the net proceeds from…

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces a securities purchase agreement with a fund affiliated with RTW Investments, LP, the Company's largest shareholder, for the purchase of an aggregate of 812,516 shares of common stock, par value $0.01 per share, at a purchase price of $32.48 per share, the closing price on August 27, 2021, for aggregate gross proceeds of approximately $26.4 million to the Company before offering expenses. The private placement is expected to close on or about August 31, 2021, subject to the satisfaction of customary closing conditions. Rocket expects to use the net proceeds from the private placement to continue to advance and expand its pipeline of product candidates, for research and development expenses and for working capital.

    The securities to be sold in the private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other applicable jurisdiction's securities laws, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions' securities laws. The Company has agreed to file a registration statement with the U.S. Securities and Exchange Commission (the "SEC") registering the resale of the common shares issued in the private placement upon the purchasers' request.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any offer, solicitation, or sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful. Any offering of the securities under the resale registration statement will only be made by means of a prospectus.

    About Rocket Pharmaceuticals, Inc.

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket") is advancing an integrated and sustainable pipeline of genetic therapies that aim to correct the root cause of complex and rare childhood disorders. The company's platform-agnostic approach enables it to design the best potential therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the potential treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit www.rocketpharma.com.

    Rocket Cautionary Statement Regarding Forward-Looking Statements

    Various statements in this release concerning the closing of the private placement, the anticipated use of proceeds therefrom and expected sufficiency thereof, Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2021 in light of COVID-19, the closing of the private placement and Rocket's uses therefrom, Rocket's expected cash runway and Rocket's ability to advance its integrated and sustainable pipeline of genetic therapies for rare childhood disorders, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket's ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

    View Full Article Hide Full Article
  5. Initiation of Pediatric Patient Dosing of RP-A501 Expected in Q3 —

    Comprehensive Clinical Update from Phase 1 Clinical Trial Planned for Q4 —

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company's Phase 1 clinical trial of RP-A501 for the treatment of Danon Disease, allowing patient enrollment to resume. The hold was removed after the Company addressed the FDA's requests to modify the trial protocol and other supporting documents with revised guidelines for patient selection and management. The Company has initiated…

    Initiation of Pediatric Patient Dosing of RP-A501 Expected in Q3 —

    Comprehensive Clinical Update from Phase 1 Clinical Trial Planned for Q4 —

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company's Phase 1 clinical trial of RP-A501 for the treatment of Danon Disease, allowing patient enrollment to resume. The hold was removed after the Company addressed the FDA's requests to modify the trial protocol and other supporting documents with revised guidelines for patient selection and management. The Company has initiated steps to resume the program as soon as possible and expects to commence dosing in the low-dose (6.7e13 vg/kg) pediatric patient cohort in the third quarter.

    "We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial. We look forward to progressing this critical work on behalf of all Danon patients," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "We are moving as quickly as possible to resume dosing and commence treatment this quarter. Additionally, given the activity observed among young adults in our low-dose cohort, in agreement with the FDA, we are now proceeding with the pediatric cohort. This is another important step forward as we believe the pediatric Danon population has the potential to realize the maximum benefit from our Danon Disease gene therapy program. We continue to anticipate reporting updated longer-term data from the low-dose (6.7e13 vg/kg) and higher-dose (1.1e14 vg/kg) young adult cohorts in the fourth quarter."

    Rocket's Danon Disease program was placed on clinical hold by the FDA in May of 2021 to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management. No new drug-related safety events were observed in the low-dose or higher-dose young adult cohorts as part of the clinical hold or during the hold.

    The non-randomized, open-label Phase 1 trial is designed to enroll both pediatric and young adult male patients to assess the safety and tolerability of a single intravenous (IV) infusion of RP-A501. Additional outcome measures include cardiomyocyte and skeletal muscle transduction by gene expression, histologic correction via endomyocardial biopsy and clinical stabilization via cardiac imaging and functional cardiopulmonary testing.

    About RP-A501

    RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure. It consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a functional version of the human LAMP2B transgene (AAV9.LAMP2B). RP-A501 is currently being evaluated in an ongoing Phase 1 clinical trial, in which the Company observed preliminary activity data of RP-A501 as well as a favorable tolerability profile.

    About Danon Disease

    Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for many male patients, death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and the Europe. The only available treatment option for Danon Disease is cardiac transplantation, which is associated with substantial complications and is not considered curative. There are no specific therapies available for the treatment of Danon Disease.

    About Rocket Pharmaceuticals, Inc.

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket") is advancing an integrated and sustainable pipeline of genetic therapies that aim to correct the root cause of complex and rare childhood disorders. The company's platform-agnostic approach enables it to design the best potential therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the potential treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit www.rocketpharma.com.

    Rocket Cautionary Statement Regarding Forward-Looking Statements

    Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2021 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, Rocket's plans for the advancement of its Danon Disease program following the lifting of the FDA's clinical hold and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket's ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

    View Full Article Hide Full Article
View All Rocket Pharmaceuticals Inc. News