News Release Details
Rocket Pharmaceuticals Reports First Quarter 2018 Financial Results and Operational Highlights
May 11, 2018
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Four Gene Therapy Programs Potentially in Clinic in 2019 with Clinical Data from up to Two Programs
Regulatory Submissions on Schedule for LAD-I, PKD and AAV Programs Over the Next 12 Months
Development of a Long-term Sustainable Growth Company Leveraging a Multi-Platform Approach
“2018 is off to a great start for Rocket. During the first quarter, we
continued to make excellent progress advancing our pipeline of
lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene
therapy programs towards key value inflection points. We look forward to
presenting updated patient data from the Fanconi Anemia program later
this month, and to disclosing our AAV-based gene therapy program by the
end of the year,” said
Dr. Shah continued, “While near-term milestones are certainly important, Rocket's long-term strategy is based on a sustainable and integrated multi-platform approach. We remain focused on our mission of continued innovation to deliver best-in-class curative cell and gene therapies to patients living with devastating diseases.”
Anticipated Milestones Over the Next 12 -18 Months
-
Fanconi Anemia (FA): Updated patient data from the ongoing
Phase 1/2 study of RP-L102 conducted with CIEMAT is expected over the
next 12 months. The next update will be provided at the American
Society of Gene and Cell Therapy’s (ASGCT) 21st Annual
Meeting in
Chicago . The oral presentation is scheduled during the Presidential Symposium onFriday, May 18, 2018 , at2:45 p.m. Central Time . - Leukocyte Adhesion Deficiency-I (LAD-I): An Investigational Medicinal Product Dossier (IMPD) is expected to be filed in the fourth quarter of 2018. Clinical data is expected over the next 12-18 months.
- Pyruvate Kinase Deficiency (PKD): An IMPD application is expected to be filed in early 2019.
- Infantile Malignant Osteopetrosis (IMO): Preclinical studies continue to advance in support of filing a first-in-human clinical study.
- AAV: Disclosure of the disease indication and preclinical data is planned for the second half of 2018.
Upcoming Conferences
-
Bank of America Merrill Lynch Health Care Conference 2018. Rocket is scheduled to present onTuesday, May 15 , at9:20 a.m. Pacific Time . -
UBS Global Healthcare Conference . Rocket is scheduled to present onWednesday, May 23 , at9:30 a.m. Eastern Time . -
Jefferies Global Healthcare Conference . Rocket is scheduled to present onThursday, June 7 , at2:00 p.m. Eastern Time .
First Quarter 2018 Financial Results
-
Cash position. Cash, cash equivalents and short-term
investments as of
March 31, 2018 , were$182.7 million , which includes a$52.0 million fully convertible debenture which expires in 2021. -
R&D expenses. Research and development expenses were
$5.7 million for the quarter endedMarch 31, 2018 , compared to$2.3 million for the quarter endedMarch 31, 2017 . -
G&A expenses. General and administrative expenses were
$8.7 million for the quarter endedMarch 31, 2018 , compared to$0.6 million for the quarter endedMarch 31, 2017 . The increase in G&A expenses was primarily due to one-time merger related expenses of$5.3 million . -
Net loss. Net loss was
$15.3 million or$(0.42) per share (basic and diluted) for the quarter endedMarch 31, 2018 , compared to$2.7 million or$(0.39) per share (basic and diluted) for the quarter endedMarch 31, 2017 . -
Shares outstanding. Approximately 39.4 million shares of common
stock were outstanding as of
March 31, 2018 .
Financial Guidance
-
Cash position. Based on its current operating plan, Rocket
expects its cash, cash equivalents and short-term investments as of
March 31, 2018 , will be sufficient to run its operations into 2020.
About
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
products 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, 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
Selected Financial Information | |||||||||||||||
Operating Results: | |||||||||||||||
(amounts in thousands, except share and per share data) | |||||||||||||||
Three Months Ended March 31, | |||||||||||||||
2018 | 2017 | ||||||||||||||
Revenue | $ | - | $ | - | |||||||||||
Operating expenses: | |||||||||||||||
Research and development | 5,743 | 2,285 | |||||||||||||
General and administrative | 8,662 | 585 | |||||||||||||
Total operating expenses | 14,405 | 2,870 | |||||||||||||
Loss from operations | (14,405 | ) | (2,870 | ) | |||||||||||
Research and development incentives | 186 | 192 | |||||||||||||
Interest expense | (1,427 | ) | - | ||||||||||||
Interest income | 288 | - | |||||||||||||
Other income | 15 | - | |||||||||||||
Net loss | $ | (15,343 | ) | $ | (2,678 | ) | |||||||||
Net loss per share attributable to common shareholders - basic and diluted | $ | (0.42 | ) | $ | (0.39 | ) | |||||||||
Weighted-average common shares outstanding - basic and diluted | 36,137,120 | 6,795,627 | |||||||||||||
Selected Balance Sheet Information | |||||||||||||||
(amounts in thousands) | |||||||||||||||
March 31, | December 31, | ||||||||||||||
2018 | 2017 | ||||||||||||||
Cash, cash equivalents and short-term investments | 182,697 | 18,142 | |||||||||||||
Total assets | 217,165 | 20,147 | |||||||||||||
Total liabilities | 47,019 | 4,628 | |||||||||||||
Total shareholders' equity | 170,146 | 15,519 | |||||||||||||
View source version on businesswire.com: https://www.businesswire.com/news/home/20180511005223/en/
Source:
Claudine Prowse, Ph.D.
SVP Corporate Development and IRO
Rocket
Pharmaceuticals, Inc.
The Alexandria Center for Life Science
430
East 29 Street, Suite 1040
New York, NY 10016
cp@rocketpharma.com