RCKT Rocket Pharmaceuticals Inc.

31.85
+0.89  (+3%)
Previous Close 30.96
Open 31.3
52 Week Low 9.01
52 Week High 35
Market Cap $1,759,196,466
Shares 55,233,798
Float 35,163,275
Enterprise Value $1,550,150,386
Volume 299,766
Av. Daily Volume 328,768
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Upcoming Catalysts

Drug Stage Catalyst Date
RP-L301
Pyruvate Kinase Deficiency (PKD)
Phase 1
Phase 1
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RP-L102
Fanconi Anemia (FA)
Phase 2
Phase 2
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RP-L201
Leukocyte Adhesion Deficiency-I (LAD-I)
Phase 1/2
Phase 1/2
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RP-A501
Danon disease
Phase 1
Phase 1
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RP-L401
Infantile Malignant Osteopetrosis (IMO)
Phase 1
Phase 1
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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. — Rocket Management to Discuss Data Updates from the Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD) Programs—

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that it will host a webcast on Monday, December 7 at 6:00PM EST to discuss presentations at the 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. The ASH presentations will highlight data from the Fanconi Anemia, Leukocyte Adhesion Deficiency-I and the Pyruvate Kinase Deficiency programs.

    Following the discussion, Rocket management and key opinion…

    — Rocket Management to Discuss Data Updates from the Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD) Programs

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that it will host a webcast on Monday, December 7 at 6:00PM EST to discuss presentations at the 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. The ASH presentations will highlight data from the Fanconi Anemia, Leukocyte Adhesion Deficiency-I and the Pyruvate Kinase Deficiency programs.

    Following the discussion, Rocket management and key opinion leaders will be available for a brief Q&A session.

    To access the live webcast and presentation, please click here. The webcast replay will be available on the Rocket website following the completion of the call.

    Investors may listen to the call by dialing (866) 866-1333 from locations in the United States or +1 (404) 260-1421 from outside the United States. Please refer to conference ID number 50038102.

    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 data updates at ASH, its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, filed November 6, 2020 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.

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  2. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces participation at the following upcoming conferences:

    • Piper Sandler 32nd Annual Virtual Healthcare Conference
      • Tuesday, December 1, 2020
    • Evercore ISI 3rd Annual HealthCONx Conference
      • Gaurav Shah, M.D., President and CEO, is scheduled to participate in a fireside chat on Wednesday, December 2, 2020, at 3:30 p.m. Eastern Time.

    A live audio webcast of the presentation will be available on the Investors section of the company's website, www.rocketpharma.com. A replay of the presentation will be archived on the Rocket website following the conferences…

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces participation at the following upcoming conferences:

    • Piper Sandler 32nd Annual Virtual Healthcare Conference
      • Tuesday, December 1, 2020
    • Evercore ISI 3rd Annual HealthCONx Conference
      • Gaurav Shah, M.D., President and CEO, is scheduled to participate in a fireside chat on Wednesday, December 2, 2020, at 3:30 p.m. Eastern Time.

    A live audio webcast of the presentation will be available on the Investors section of the company's website, www.rocketpharma.com. A replay of the presentation will be archived on the Rocket website following the conferences.

    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.

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  3. ~$3.7 Million Clinical Grant to Fund U.S. Phase 1 Study of RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis —

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the California Institute for Regenerative Medicine (CIRM) has awarded Rocket a $3.7 million CLIN2 grant award to support the clinical development of its lentiviral vector (LVV)-based gene therapy, RP-L401, for the treatment of Infantile Malignant Osteopetrosis (IMO), a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure. The CIRM was founded…

    ~$3.7 Million Clinical Grant to Fund U.S. Phase 1 Study of RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis —

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the California Institute for Regenerative Medicine (CIRM) has awarded Rocket a $3.7 million CLIN2 grant award to support the clinical development of its lentiviral vector (LVV)-based gene therapy, RP-L401, for the treatment of Infantile Malignant Osteopetrosis (IMO), a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure. The CIRM was founded in 2004 following the passing of Proposition 71 or the California Stem Cell Research and Cures Initiative, which allowed $3 billion in state funding for stem cell research conducted in California. This will be Rocket's second CIRM grant after receiving one in 2019 for the development of the company's gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I).

    "We're grateful the CIRM has recognized the promise of RP-L401 for IMO, a devastating pediatric rare disease for which the primary treatment option is allogeneic bone marrow transplant," said Jonathan Schwartz, M.D. Chief Medical Officer and Senior Vice President of Rocket. "RP-L401 could be a potentially curative treatment for this devastating disorder that affects children at a young age, and we are thankful to have this meaningful support from the CIRM to move our program forward for these families."

    Rocket's Investigational New Drug Application (IND) for RP-L401 was accepted by the U.S. Food and Drug Administration (FDA) in June of 2020, and the gene therapy received Fast Track designation from the FDA in August 2020. Proceeds from the grant will help fund clinical trial costs, as well as provide manufactured drug product for Phase 1 patients enrolled at the U.S. clinical trial site, University of California, Los Angeles, led by principal investigator Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles. The non-randomized, open-label Phase 1 clinical trial will enroll two pediatric patients, one month of age or older. The trial is designed to assess safety and tolerability of RP-L401, as well as preliminary efficacy, including potential improvements in bone abnormalities/density, hematologic status and endocrine abnormalities. Further information about the clinical program is available here.

    About Infantile Malignant Osteopetrosis

    Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.

    RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover. Rocket's IMO research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-12095). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

    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 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, filed November 6, 2020 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.

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  4. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces presentations at the upcoming 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. There will be two oral presentations highlighting clinical data from the Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs as well as a poster presentation highlighting preliminary clinical data from the Pyruvate Kinase Deficiency (PKD) program. All three programs utilize Rocket's "Process B" manufacturing platform.

    Details for Rocket's presentations are as follows:

    Oral Presentations
    Title: 
    Gene…

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces presentations at the upcoming 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. There will be two oral presentations highlighting clinical data from the Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs as well as a poster presentation highlighting preliminary clinical data from the Pyruvate Kinase Deficiency (PKD) program. All three programs utilize Rocket's "Process B" manufacturing platform.

    Details for Rocket's presentations are as follows:

    Oral Presentations

    Title: 
    Gene Therapy for Fanconi Anemia, Complementation Group A: Updated Results from Ongoing Global Clinical Studies of RP-L102

    Session Title: Gene Editing, Therapy and Transfer I

    Presenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of Medicine

    Session Date: Monday, December 7, 2020

    Session Time: 11:30 a.m. - 1:00 p.m. (Pacific Time)

    Presentation Time: 12:15 p.m. (Pacific Time)

    Title: Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

    Session Title: Gene Editing, Therapy and Transfer I

    Presenter: Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles

    Session Date: Monday, December 7, 2020

    Session Time: 11:30 a.m. - 1:00 p.m. (Pacific Time)

    Presentation Time: 12:30 p.m. (Pacific Time)

    Poster Presentation

    Title: Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

    Session Title: Gene Editing, Therapy and Transfer: Poster II

    Presenter: José Luis López Lorenzo, M.D., Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain

    Session Date: Sunday, December 6, 2020

    Session Time: 7:00 a.m. – 3:30 p.m. (Pacific Time)

    About Fanconi Anemia

    Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a ‘gold standard' test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patient's blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as ‘natural gene therapy' provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

    About Leukocyte Adhesion Deficiency-I

    Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

    Rocket's LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

    About Pyruvate Kinase Deficiency

    Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

    RP-L301 was in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).

    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 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, 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-Q for the quarter ended June 30, 2020, filed August 5, 2020 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.

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  5. —Positive Phase 1 Study Results of RP-L201 for the Treatment of LAD-I Demonstrate Ongoing Efficacy, Durability and Validation of Rocket's "Process B" Lentiviral Platform—

    —Data Readouts in PKD, FA "Process B" and Danon Disease on Track for December—

    —First Patient Treated in Higher Dose Cohort of Phase 1 Study of RP-A501 for the Treatment of Danon Disease—

    —Strong Balance Sheet with $229 Million in Cash; Capitalized into 2Q 2022—

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial results for the quarter that ended September 30, 2020, along with an update on the Company's key pipeline developments…

    —Positive Phase 1 Study Results of RP-L201 for the Treatment of LAD-I Demonstrate Ongoing Efficacy, Durability and Validation of Rocket's "Process B" Lentiviral Platform—

    —Data Readouts in PKD, FA "Process B" and Danon Disease on Track for December—

    —First Patient Treated in Higher Dose Cohort of Phase 1 Study of RP-A501 for the Treatment of Danon Disease—

    —Strong Balance Sheet with $229 Million in Cash; Capitalized into 2Q 2022—

    Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial results for the quarter that ended September 30, 2020, along with an update on the Company's key pipeline developments, business operations and upcoming milestones.

    "We are incredibly pleased with the steady progress we made across all five of our clinical programs this quarter. The data from our RP-L201 trial for LAD-I demonstrated the potential for a very robust and attractive profile for our ‘Process B' lentiviral gene therapy pipeline. We also treated our first higher dose patient in our Phase 1 study of RP-A501 for the treatment of Danon Disease, and presented compelling preclinical data in IMO," said Gaurav Shah, M.D., President and Chief Executive Officer of Rocket. "In addition, I am proud that against the backdrop of a global pandemic, while there have been some delays in patient follow up and data collection, we continued to enroll and treat patients in our LVV and AAV gene therapy programs as well as expand our clinical trials internationally. Finally, we made strong progress in the build-out of Rocket's AAV R&D and manufacturing facility, with the ability to produce GMP product anticipated in 2021."

    Dr. Shah continued, "We are looking forward to sharing additional updates as we enter the fourth quarter. These data announcements include an update on the Phase 1 and 2 trials for FA ‘Process B', and for the first time, Phase 1 clinical data from our two largest indications: RP-L301 for PKD and RP-A501 for Danon Disease. We believe we are advancing closer to our goal of taking drug products from the discovery phase to BLA submission and launch so we may help improve the lives of patients facing these rare and devastating childhood diseases."

    Key Pipeline Developments and Operational Updates

    • Positive clinical update for the Company's Leukocyte Adhesion Deficiency Program (LAD-I) presented at the European Society for Immunodeficiencies (ESID) 2020 Meeting along with preclinical data from the Company's Infantile Malignant Osteopetrosis (IMO) program.
      • An oral presentation provided positive longer-term follow-up data from the Phase 1/2 clinical trial of RP-L201 for LAD-I. The data presented in the oral presentation are from two pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%. Both patients were treated with RP-L201, Rocket's ex-vivo lentiviral gene therapy candidate. Patient L201-003-1001 was 9 years of age at treatment and has been followed for 12 months and Patient L201-003-1004 was 3 years of age at treatment and has been followed for 4 months. Treatments were well tolerated, and no safety issues were reported with infusion or post-treatment. Both subjects achieved hematopoietic reconstitution in less than 4 weeks. Patient L201-003-1001 demonstrated durable CD18 expression of 40%, peripheral blood VCN levels of 1.3, visible signs of improvement in existing skin lesions and no new infections reported 12 months post-treatment. Patient L201-003-1004 demonstrated CD18 expression of 28% and early peripheral blood VCN trending similarly to the first patient, reported 4 months post-treatment.
      • An e-poster highlighted preclinical trial data on RP-L401 for IMO. Preclinical data on IMO indicate that a modest level of engraftment corrects the disease phenotype in vivo, with increased long-term survival, tooth eruption, weight gain and normalized bone resorption. Results support acceleration into clinical development for RP-L401.
    • First patient treated in the higher dose cohort of the Phase 1 dose-escalation clinical trial of RP-A501 for the treatment of Danon Disease. The first patient was treated at the higher dose level of 1.1×1014 genome copies/kilogram after clearance from the U.S. Food and Drug Administration (FDA) and the Independent Data Safety Monitoring Committee (IDSMC) to move to the higher dose cohort of the study. Preliminary Phase 1 data are anticipated in December assuming no further delays in patient data collection due to COVID-19.
    • Opened the Research & Development (R&D) and Chemistry, Manufacturing and Controls (CMC) facility in Cranbury, New Jersey. Approximately half of the newly constructed 103,720 square foot facility will be dedicated to adeno-associated virus (AAV) Current Good Manufacturing Practice (cGMP) manufacturing. As previously guided, the first cGMP clinical product release is expected in 2021.
    • Received additional regulatory designations for IMO and Danon Disease programs from U.S. Food and Drug Administration. RP-L401 gene therapy for IMO received Fast Track Designation and RP-A501 for Danon Disease received Rare Pediatric Disease Designation. The FDA's Fast Track program facilitates the development of products intended to treat serious conditions that have the potential to address unmet medical needs. The designation enables greater access to the FDA for the purpose of expediting the product's development, review and potential approval. The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S.
    • Expanded clinical sites for Fanconi Anemia (FA), Danon, LAD-I and IMO trials, adding to global centers of excellence. The newly added centers, which include some of the leading gene therapy research programs in the world, include: Great Ormond Street Hospital (GOSH), Children's Hospital of Philadelphia (CHOP), the University of Minnesota and the University of California, Los Angeles (UCLA). We anticipate these additional sites will expand patient access to Rocket's clinical trials worldwide.
    • Hosted third Pyruvate Kinase Deficiency (PKD) Day in October. Continuing its commitment to the patient communities it serves, Rocket facilitated the first virtual edition of PKD Day. The event, intended for patients with PKD and their families, gave attendees an introduction to PKD, gene therapy and Rocket's clinical program and provided time for a Q&A session with experts in the field and Rocket team members.

    Anticipated Milestones

    • FA (RP-L102)
      • Preliminary "Process B" data (12/20)
      • Updated "Process B" data (1H21)
    • Danon Disease (RP-A501)
      • Preliminary Phase 1 data (12/20)
      • Updated Phase 1 data (2H21)
    • LAD-I (RP-L201)
      • Phase 2 data (1H21)
    • PKD (RP-L301)
      • Preliminary Phase 1 data (12/20)
      • Phase 1 data update (2H21)
    • IMO (RP-L401)
      • Phase 1 data (2H21)

    Upcoming Investor Conferences

    • Piper Sandler 32nd Annual Healthcare Conference, December 1, 2020
    • Evercore ISI 3rd Annual HealthCONx Conference, December 2, 2020

    Third Quarter Financial Results

    • Cash position. Cash, cash equivalents and investments as of September 30, 2020 were $228.7 million.
    • Debt. Our balance sheet includes $52.0 million of fully convertible notes.
    • R&D expenses. Research and development expenses were $21.7 million for the three months ended September 30, 2020, compared to $14.8 million for the three months ended September 30, 2019, primarily due to increases in manufacturing and development costs, clinical trial expenses, license fees, and compensation and benefit expenses due to increased R&D headcount.
    • G&A expenses. General and administrative expenses were $5.7 million for the three months ended September 30, 2020, compared to $4.3 million for the three months ended September 30, 2019, primarily due to increases in non-cash stock compensation expense and an increase in compensation and benefit expenses due to increased G&A headcount.
    • Net loss. Net loss was $29.0 million or $0.53 per share (basic and diluted) for the three months ended September 30, 2020, compared to $19.3 million or $0.38 per share (basic and diluted) for the three months ended September 30, 2019.
    • Shares outstanding. 55,204,127 shares of common stock were outstanding as of September 30, 2020.

    Financial Guidance

    • Cash position. As of September 30, 2020, we had cash, cash equivalents and investments of $228.7 million. Rocket expects such resources will be sufficient to fund its operations into the second quarter of 2022.

    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 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, 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-Q for the quarter ended June 30, 2020, filed August 5, 2020 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.

    Selected Financial Information

    Operating Results:

    (amounts in thousands, except share and per share data)

     

     

    Three Months Ended

    September 30,

     

    Nine Months Ended

    September 30,

     

     

    2020

     

    2019

     

    2020

     

    2019

    Revenue

    $

    -

     

    $

    -

     

    $

    -

     

    $

    -

     

     
    Operating expenses:
    Research and development

     

    21,657

     

     

    14,829

     

     

    55,345

     

     

    43,955

     

    General and administrative

     

    5,730

     

     

    4,336

     

     

    19,720

     

     

    12,547

     

    Total operating expenses

     

    27,387

     

     

    19,165

     

     

    75,065

     

     

    56,502

     

    Loss from operations

     

    (27,387

    )

     

    (19,165

    )

     

    (75,065

    )

     

    (56,502

    )

    Research and development incentives

     

    -

     

     

    -

     

     

    -

     

     

    250

     

    Interest expense

     

    (1,967

    )

     

    (1,466

    )

     

    (5,326

    )

     

    (4,615

    )

    Interest and other income net

     

    518

     

     

    979

     

     

    1,913

     

     

    2,522

     

    (Amortization of premium) accretion of discount on investments - net

     

    (244

    )

     

    368

     

     

    (306

    )

     

    930

     

    Net loss

    $

    (29,080

    )

    $

    (19,284

    )

    $

    (78,784

    )

    $

    (57,415

    )

    Net loss per share attributable to common shareholders - basic and diluted

    $

    (0.53

    )

    $

    (0.38

    )

    $

    (1.43

    )

    $

    (1.19

    )

    Weighted-average common shares outstanding - basic and diluted

     

    55,188,956

     

     

    50,364,649

     

     

    55,077,254

     

     

    48,270,771

     

     
    Selected Balance Sheet Information

    (amounts in thousands)

    September 30,

    2020

    December 31,

    2019

    Cash, cash equivalents and investments

     

    228,690

     

     

    304,115

     

    Total assets

     

    335,496

     

     

    372,121

     

    Total liabilities

     

    93,655

     

     

    64,824

     

    Total stockholders' equity

     

    241,841

     

     

    307,297

     

     

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