On March 7, 2019 Rocket Pharmaceuticals, Inc. (Nasdaq:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported financial results for the year ended December 31, 2018, and provides an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, MAR 7, 2019, View Source [SID1234534079]).

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"2018 was a great year for Rocket," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We successfully established ourselves as a public gene therapy company, funded our pipeline with more than $250 million in capital, achieved clinical proof of concept for our lead lentiviral gene therapy program in FA, expanded our pipeline with a new AAV gene therapy program in Danon disease, and received FDA clearance of three Rocket-sponsored INDs in only three months. These accomplishments are a testament to our ability to execute on our goals and commitment to bring new therapies to patients who desperately need them."

Dr. Shah added, "Looking ahead, Rocket is entering an exciting period in our evolution as we anticipate treating more patients with our gene therapy products across our pipeline. We look forward to at least two clinical readouts this year that, if successful, may support advancing our gene therapies that have the potential to transform the treatment paradigm in diseases that are limited to toxic bone marrow or organ transplant regimens. We believe we are well-positioned and prepared to meet all of these exciting challenges and opportunities."

Full Year 2018 and Recent Highlights

Expanded Pipeline with First AAV Gene Therapy Program Targeting a Monogenic Heart Failure Syndrome. In November 2018, the Company presented preclinical proof of concept data from its first adeno-associated viral vector (AAV)-based gene therapy, RP-A501, targeting Danon disease. RP-A501 is being developed in collaboration with Dr. Eric Adler, Director of Cardiac Transplant and Mechanical Circulatory Support at UC San Diego Health and Professor of Medicine at University of California San Diego School of Medicine. Preclinical studies show RP-A501 improves patient survival and corrects the disease phenotype with dose-dependent improvements in molecular, structural, and functional endpoints with a clean safety and tolerability profile. The Company simultaneously announced an exclusive, worldwide license agreement with REGENXBIO Inc. for Rocket to develop and commercialize gene therapy treatments for Danon disease using REGENXBIO’s NAV AAV9 vector, as well as exclusive options for two additional NAV AAV vectors for the treatment of Danon disease. A Phase 1 clinical trial of RP-A501 is planned to commence in the second quarter of 2019.
Initial Clinical Proof of Concept Achieved in FA. Long-term clinical data from the ongoing Phase 1/2 clinical trial of RP-L102 for Fanconi Anemia (FA) utilizing "Process A", the first-generation non-optimized process, were presented at ESGCT in November 2018. Results showed durable engraftment at 30 months post treatment, stabilization of previously-declining blood counts and progressive increases in corrected versus non-corrected peripheral blood leukocytes. Moving forward, the Company plans to initiate a Phase 1 clinical trial of RP-L102 at Stanford University utilizing "Process B" which incorporates higher cell doses, transduction enhancers, and commercial-grade vector manufacturing and cell processing.
Three Gene Therapy INDs Cleared by the FDA. In a three-month period, the FDA cleared three Rocket-sponsored Investigational New Drug (IND) applications, paving the way for Phase 1 clinical trials to begin in FA and Danon disease along with a Phase 1/2 adaptive clinical study to begin in Leukocyte Adhesion Deficiency-I (LAD-I).
Nine Regulatory Designations Across Pipeline – RMAT, Fast Track, ATMP, Orphan Drug and Rare Pediatric Disease.
The Company received notification that the following programs received regulatory designations:
RP-L102 for FA received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Rare Pediatric Disease designations from the FDA, as well as Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA).
RP-L201 for LAD-I received Fast Track and Rare Pediatric Disease designations from the FDA, as well as ATMP classification from the EMA.
RP-L401 for Infantile Malignant Osteopetrosis received Orphan Drug designation from the FDA.
RP-A501 for Danon disease received Orphan Drug designation from the FDA.
Each designation provides numerous incentives to support the development of Rocket’s programs, including prescription drug user fee waivers, increased access to the regulatory authorities, expedited review timelines and tax credits towards the cost of clinical trials.
Approximately $150 Million Secured in Two Oversubscribed Public Offerings. Following the successful completion of its reverse merger with Inotek Pharmaceuticals in January 2018 that capitalized the Company with $97.6 million in cash, Rocket completed two oversubscribed underwritten public offerings of its common stock for gross proceeds of approximately $150 million over the course of the year.
Anticipated Milestones

FA (RP-L102)
Initiation of Phase 1 clinical trial of RP-L102 utilizing "Process B" (2Q19)
Additional data from RP-L102 utilizing "Process A" (2H19)
Initial data from Phase 1 clinical trial of RP-L102 utilizing "Process B" (2H19)
Regulatory alignment on final endpoints for registration (2H19)
LAD-I (RP-L201)
Initiation of Phase 1/2 clinical trial of RP-L201 in support of registration (2Q19)
Initial data from Phase 1 portion of clinical trial of RP-L201 (2H19)
PKD (RP-L301)
Initiation of Phase 1 clinical trial of RP-L301 (2H19)
Danon (RP-A501)
Initiation of Phase 1 clinical trial of RP-A501 (2Q19)
Upcoming Investor Conferences

Cowen and Company 39th Annual Health Care Conference. Rocket is scheduled to participate in a fireside chat on Tuesday, March 12, 2019, at 10:40 a.m. Eastern Time.
Alliance for Regenerative Medicine Cell & Gene Investor Day. Rocket is scheduled to present on Thursday, March 21, 2019, at 9:55 a.m. Eastern Time.
Fourth Quarter and Full Year 2018 Financial Results

Cash position. Cash, cash equivalents and investments as of December 31, 2018, were $213.1 million.
Debt. Our cash position includes a $52.0 million fully convertible debenture which matures in August of 2021.
R&D expenses. Research and development expenses were $23.7 million and $53.3 million for the three and twelve months ended December 31, 2018, compared to $4.9 million and $14.9 million for the three and twelve months ended December 30, 2017.
G&A expenses. General and administrative expenses were $2.9 million and $17.9 million for the three and twelve months ended December 31, 2018, compared to $1.7 million and $4.9 million for the three and twelve months ended December 31, 2017.
Net loss. Net loss was $27.3 million and $74.5 million or $(0.66) and $(1.89) per share (basic and diluted) for the three and twelve months ended December 31, 2018, compared to $6.7 million and $19.6 million or $(0.98) and $(2.88) per share (basic and diluted) for the three and twelve months ended December 31, 2017.
Shares outstanding. Approximately 45.2 million shares of common stock were outstanding as of December 31, 2018.
Financial Guidance

Cash position. Based on its current operating plan, Rocket expects its cash, cash equivalents and investments as of December 31, 2018, will be sufficient to run its operations into the second half of 2020.