- Achieved Regulatory Designations for Fanconi
Anemia (FA) in the U.S. and Europe; Registrational Trial on Track
for 2019 -
- Presented Promising Fanconi Anemia Data at
ASGCT -
- Adeno-associated Viral Vector Program
Disclosure on Track for Fourth Quarter of 2018 -
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) (“Rocket”), a leading
U.S.-based multi-platform gene therapy company, today reported
financial results for the quarter ended June 30, 2018, and provided
an update on the Company’s recent achievements, as well as upcoming
milestones.
“Rocket made significant progress on our clinical, regulatory
and corporate initiatives in the second quarter,” said Gaurav Shah,
M.D., Chief Executive Officer and President of Rocket. “We are
pleased with the positive clinical data from our FA program that
were presented at ASGCT and look forward to additional data over
the next 12-18 months. The momentum has continued with recent
regulatory designations for FA, including Rare Pediatric Disease
from the U.S. Food and Drug Administration (FDA) and Advanced
Therapy Medicinal Product (ATMP) by the European Medicines Agency
(EMA). These positive steps set the stage nicely for a global
registrational study in 2019.”
“Our additional gene therapy pipeline programs for devastating
rare diseases remain on track. These include our products for
Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency
(PKD) and our undisclosed adeno-associated viral vector (AAV)
program. We expect to disclose the indication and share preclinical
data from our AAV program later this year, and clinical data on up
two programs in 2019. Our progress to date has been a true
collaboration between the Rocket team, our partners, physicians,
and the patients we serve. We look forward to meeting the
milestones ahead.”
Recent Pipeline and Corporate Updates
- Rare Pediatric Disease Designation
for FA. In July 2018, the Company was notified that it received
Rare Pediatric Disease designation from the FDA for RP-L102 for the
treatment of FA Type A. The FDA defines a “rare pediatric disease”
as a serious and life-threatening disease that affects less than
200,000 people in the U.S. that are aged between birth to 18 years.
The Rare Pediatric Disease designation program allows for a Sponsor
who receives an approval for a product to potentially qualify for a
voucher that can be redeemed to receive a priority review of a
subsequent marketing application for a different product.
- Advanced Therapy Medicinal Product
Classification for FA. In June 2018, the Company was notified
that the EMA classified RP-L102 as an ATMP. The ATMP classification
recognizes and defines medicines for human use that are considered
gene-, tissue- or cell-based therapies. The key benefit of ATMP
classification is the early involvement and guidance from the EMA’s
Committee of Advanced Therapies, which is the regulatory reviewing
body for gene therapies.
- Phase 1/2 data of RP-L102 in FA
shows promising engraftment and chromosomal stability leading to
improved bone marrow functionality. At the ASGCT Annual Meeting
in May 2018, updated data from the ongoing Phase 1/2 clinical trial
of RP-L102 was presented and included data from four patients that
have been followed for 12-24 months and a fifth patient, treated
with transduction-enhanced RP-L102, that was followed for two
months. All patients demonstrated continued improvement in
engraftment following administration of RP-L102 with sustained
phenotypic reversals and earlier evidence of gene correction seen
in higher-dosed patients. The progressive increases of corrected
versus non-corrected peripheral blood leukocytes indicate the
potential of RP-L102 to restore the functionality of bone marrow
hematopoietic stem cells. The one patient that received
transduction enhanced RP-L102 showed the highest transduction
efficiency seen to date in all five patients treated, with a
preliminary drug product vector copy number (VCN) of ~2.5 – 3, and
a cell dose considered below the threshold level of 500,000K
CD34+/kg. Rocket plans to engage with regulatory authorities to
progress RP-L102 towards a potential global registrational study in
2019.
- Stanford University research
collaboration. In May 2018, Rocket and the Stanford University
School of Medicine announced a strategic collaboration to support
the advancement of FA and PKD gene therapy research. Under the
terms of the collaboration agreement, Stanford will serve as a lead
clinical trial research center in the U.S. for the planned FA
registrational trial and would also be the lead site for PKD
clinical trials. The project will also separately evaluate the
potential for non-myeloablative, non-genotoxic antibody-based
conditioning regimens as a future development possibility that may
be applied across bone marrow-derived disorders.
- Strengthened management team with
addition of former FDA Director of the Office of Orphan Products
Development (OOPD). Gayatri R. Rao, M.D., J.D., was appointed
Vice President, Regulatory Policy and Patient Advocacy, in May
2018. Dr. Rao most recently served as Director of the OOPD within
the FDA for the last five years where she was responsible for
implementing statutory programs focused on promoting the
development of medical products for rare diseases. In her new role
at Rocket, Dr. Rao will support the development of global
regulatory policies and strategies, patient advocacy initiatives,
and rare disease natural history studies.
Anticipated Milestones
- Preclinical data and disclosure of the
AAV-based gene therapy program (4Q18)
- Investigational Medicinal Product
Dossier (IMPD) filing in Spain for the LAD-I program (4Q18)
- IMPD filing in Spain for the PKD
program (Early 2019)
- Additional FA patient data (Next 12-18
months)
- Investigational New Drug (IND)
application filing in the U.S. for the AAV-based program
(2019)
- IND application filing in the U.S. for
the FA program (2019)
September Conferences
- Citi’s 13th Annual Biotech Conference –
September 5-6, 2018 in Boston, MA
- Morgan Stanley 16th Annual Global
Healthcare Conference – September 12-14, 2018 in New York, NY
- Oppenheimer Specialty Pharma & Rare
Disease Fall Summit – September 25-26 in New York, NY
- Jefferies Gene Therapy and Editing
Summit – September 27, 2018 in New York, NY
Second Quarter 2018 Financial Results
- Cash position. Cash, cash
equivalents and investments as of June 30, 2018, were $171.5
million, which includes a $52.0 million fully convertible debenture
which expires in 2021.
- R&D expenses. Research and
development expenses were $10.8 million and $16.5 million for the
three and six months ended June 30, 2018, compared to $2.8 million
and $5.1 million for the three and six months ended June 30,
2017.
- G&A expenses. General and
administrative expenses were $4.1 million and $12.8 million for the
three and six months ended June 30, 2018, compared to $0.7 million
and $1.3 million for the three and six months ended June 30,
2017.
- Net loss. Net loss was $15.8
million and $31.1 million or $(0.40) and $(0.82) per share (basic
and diluted) for the three and six months ended June 30, 2018,
compared to $3.3 million and $6.2 million or $(0.49) and $(0.91)
per share (basic and diluted) for the three and six months ended
June 30, 2017.
- Shares outstanding.
Approximately 39.5 million shares of common stock were outstanding
as of June 30, 2018.
Financial Guidance
- Cash position. Based on its
current operating plan, Rocket expects its cash, cash equivalents
and investments as of June 30, 2018, will be sufficient to run its
operations into 2020.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is an
emerging, clinical-stage biotechnology company focused on
developing first-in-class gene therapy treatment options for rare,
devastating diseases. Rocket’s multi-platform development approach
applies the well-established lentiviral vector (LVV) and
adeno-associated viral vector (AAV) gene therapy platforms.
Rocket's lead clinical program is a LVV-based gene therapy for the
treatment of Fanconi Anemia (FA), a difficult to treat genetic
disease that leads to bone marrow failure and potentially cancer.
Preclinical studies of additional bone marrow-derived disorders are
ongoing and target Pyruvate Kinase Deficiency (PKD), Leukocyte
Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis
(IMO). Rocket is also developing an AAV-based gene therapy program
for an undisclosed rare pediatric disease. For more information
about Rocket, please visit www.rocketpharma.com.
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 the safety, effectiveness and
timing of product candidates that Rocket may develop, including in
collaboration with academic partners, to treat Fanconi Anemia (FA),
Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency
(PKD) and Infantile Malignant Osteopetrosis (IMO), 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 successfully demonstrate
the efficacy and safety of such products and pre-clinical studies
and clinical trials, its gene therapy programs, the preclinical and
clinical results for its product candidates, which may not support
further development and marketing approval, Rocket’s ability to
commence a registrational study in FA within the projected time
periods, the potential advantages of Rocket’s product candidates,
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 and its licensors ability to
obtain, maintain and protect its and their respective intellectual
property, the timing, cost or other aspects of a potential
commercial launch of Rocket’s product candidates, Rocket’s ability
to manage operating expenses, Rocket’s ability to obtain additional
funding to support its business activities and establish and
maintain strategic business alliances and new business initiatives,
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, 2017. 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 June 30, Six Months Ended
June 30, 2018 2017
2018 2017 Revenue $ - $ -
$ - $ - Operating expenses: Research and development 10,772
$ 2,819 16,525 5,104 General and administrative 4,100
702 12,752 1,287 Total
operating expenses 14,872 3,521
29,277 6,391 Loss from operations (14,872 )
(3,521 ) (29,277 ) (6,391 ) Research and development incentives -
192 186 192 Interest expense (1,363 ) - (2,834 ) - Interest income
473 - 805 - Other income (5 ) - 10
- Net loss $ (15,767 ) $ (3,329 ) $ (31,110 )
$ (6,199 ) Net loss per share attributable to common shareholders -
basic and diluted $ (0.40 ) $ (0.49 ) $ (0.82 ) $ (0.91 )
Weighted-average common shares outstanding - basic and diluted
39,483,006 6,795,627 37,954,972
6,795,627 Selected Balance Sheet
Information (amounts in thousands)
June 30, December
31, 2018 2017 Cash,
cash equivalents and investments 171,466 18,142 Total assets
207,574 20,147 Total liabilities 50,498 4,628 Total shareholders'
equity 157,076 15,519
View source
version on businesswire.com: https://www.businesswire.com/news/home/20180808005190/en/
Claudine Prowse, Ph.D.SVP Corporate Strategy and IRORocket
Pharma, Inc.The Empire State Building, Suite 7530New York, NY
10118cp@rocketpharma.comwww.rocketpharma.cominvestors@rocketpharma.com
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