Rocket Pharmaceuticals: Portfolio, Financial Analysis and Stock

  Highlights of Rocket Pharmaceuticals 

- Gene Therapy company with promising clinical development & research program
- Early and Late Stage Programs with potential growth 
- Pipeline includes Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Infantile Malignant Osteopetrosis (IMO)
- Strong Balance Sheet with $229 Million in Cash; Capitalized into 2Q 2022

About Rocket Pharmaceuticals, Inc.
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.

Stock Symbol: Nasdaq: RCKT

Platform
The company is developing multi-platform gene therapy targeting rare diseases with ex vivo lentiviral and in-vivo AAV mediated gene delivery 


Therapeutic Areas
Disease Focus Area and Pipeline


Ex-vivo Lentiviral vectors (LVV) 
Fanconi Anemia (FA) 
RP-L102 is a gene therapy product containing autologous (patient-derived) hematopoietic stem cells (HSCs) that have been genetically modified with a lentiviral vector to contain a functional copy of the FANCA gene. If given early in life, RP-L102 gene therapy has the potential to serve as a preventative measure, correcting bone marrow cells well before bone marrow failure.

Leukocyte Adhesion Deficiency-I (LAD-I) 
RP-L201 is a gene therapy product containing autologous (patient-derived) hematopoietic stem cells (HSCs) that have been genetically modified with a lentiviral vector to contain a functional copy of the ITGB2 gene. In an early-stage clinical trial, RP-L201 has been shown to increase levels of CD18, correcting the deficiency that is a hallmark of the disease.

Pyruvate Kinase Deficiency (PKD)
RP-L301 is a gene therapy product containing autologous (patient-derived) hematopoietic stem cells (HSCs) that have been genetically modified with a lentiviral vector to contain a functional copy of the PKLR gene. RP-L301 has the potential to correct multiple disease elements associated with PKD. Due to the number of patients who could benefit from RP-L301, Rocket’s PKD program has the potential to become its largest lentiviral vector program. Learn More.....

Infantile Malignant Osteopetrosis (IMO)
RP-L401 is a gene therapy product containing autologous (patient-derived) hematopoietic stem cells (HSCs) that have been genetically modified with a lentiviral vector to contain a functional copy of the TCIRG1 gene. In preclinical studies, data show that RP-L401 has the potential to correct osteoclast resorptive function.

In-vivo adeno-associated virus (AAV)
Danon Disease
RP-A501 is Rocket’s first AAV program. It is a gene therapy product consisting of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a functional version of the human LAMP2B transgene (AAV9.LAMP2B) which is administered as a single intravenous (IV) infusion. RP-A501 has the potential to restore normal cardiac function in patients with Danon Disease.

Third Quarter 2020 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.

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