On November 7, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported financial results for the quarter ended September 30, 2019, and provides an update on the Company’s recent pipeline developments, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, NOV 7, 2019, View Source [SID1234550663]).

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"In the third quarter, we made meaningful progress towards our clinical and regulatory milestones," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We received IMPD and IND clearance for our global Phase 1 trial for PKD, treated our first patient for LAD-I and continued enrollment of our Phase 1 trial of RP-A501 for Danon disease. We also announced promising long-term data from our Phase 1/2 trial of first-generation RP-L102 for Fanconi Anemia supporting sustained clinical improvement with robust engraftment exceeding the 10% threshold agreed to by regulators for the upcoming global Phase 2 trial. We hope to further optimize these responses with our ‘Process B’ of RP-L102 designed to enable consistent results with commercial-grade product without cytotoxic conditioning. We look forward to data from ‘Process B’ later this year, along with initial Phase 1 data from our RP-L201 trial for LAD-I."

Dr. Shah continued, "We now have four gene therapy candidates in the clinic, meeting one of our 2019 corporate goals and reinforcing our multi-platform strategy. As we advance our first candidate into late-stage development, we remain dedicated to our pursuit of potentially curative treatments for patients contending with rare genetic diseases."

Recent Pipeline Developments

Commencement of registration-enabling Phase 2 study for Fanconi Anemia (FA). Patient enrollment is ongoing in the global Phase 2 trial. The study initiation follows recent alignment from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) on the trial design and the primary endpoint. Resistance to mitomycin-C, a DNA damaging agent, in bone marrow stem cells at a minimum time point of one year will serve as the primary endpoint and potentially as a surrogate endpoint for accelerated approval. Patient dosing is anticipated to begin in the fourth quarter with preliminary Phase 2 data anticipated in 2020.
Long-term Phase 1/2 clinical data of RP-L102 for FA "Process A" presented at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress. Long-term patient follow-up data demonstrate evidence of increasing and durable engraftment leading to bone marrow restoration exceeding the 10% threshold agreed to by the FDA and EMA for the ongoing registration-enabling Phase 2 trial. In patient 02002, who received what we consider adequate drug product, hemoglobin levels are now similar to those in the first year after birth, suggesting hematologic correction over the long term. Preliminary results from the first two patients receiving "Process B" of RP-L102 are anticipated in the fourth quarter.
Global Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD) initiates enrollment. Rocket received Investigational Medicinal Product Dossier (IMPD) clearance and Investigational New Drug (IND) application approval of RP-L301 during the quarter. The open-label, single-arm, clinical trial will enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe. The trial is designed to assess the safety, tolerability and preliminary efficacy of RP-L301. Preliminary data are anticipated in 2020.
Patient dosing continues in Phase 1/2 registrational trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). The Phase 1 portion of the trial is expected to enroll two patients and will assess the safety and tolerability of RP-L201. The Phase 2 portion of the trial will evaluate overall survival. Preliminary Phase 1 data will be presented in the fourth quarter.
Patient dosing continues in first cohort in the Phase 1 clinical trial of RP-A501 for the treatment of Danon disease. Rocket continues to enroll patients in the trial and anticipates reporting Phase 1 data in 2020. The study is designed to assess the safety and tolerability of a single infusion of RP-A501. Pediatric dosing will initiate pending determination of safety in a patient population comprised of older adolescents and young adults.
Anticipated Milestones

FA (RP-L102)
Global Phase 2 trial first patient treatment (4Q19)
Initial Phase 1 data for "Process B" (4Q19)
Additional Phase 1 "Process B" data (1H20)
Preliminary Phase 2 data (2H20)
Danon Disease (RP-A501)
Phase 1 data (2020)
LAD-I (RP-L201)
Initial Phase 1 data (4Q19)
Phase 1 data update (1H20)
Commence enrollment of Phase 2 study (2H20)
PKD (RP-L301)
Preliminary Phase 1 data (2H20)
IMO (RP-L401)
Initiation of clinical study (2H20)
Upcoming Investor Conferences

Barclays Gene Editing & Gene Therapy Summit—November 13, 2019 in New York, N.Y.
Evercore ISI 2nd Annual HealthCONx Conference—December 3, 2019 in Boston, M.A.
Piper Jaffray’s 31st Annual Healthcare Conference—December 5, 2019 in New York, N.Y.
Third Quarter 2019 Financial Results

Cash position. Cash, cash equivalents and investments as of September 30, 2019, were $240.6 million.
Debt. Our balance sheet includes a $52.0 million fully convertible debenture which matures in August of 2021.
R&D expenses. Research and development expenses were $14.8 million for the three months ended September 30, 2019, compared to $13.1 million for the three months ended September 30, 2018. The increase was primarily driven by an increase in clinical trial costs of $1.3 million.
G&A expenses. General and administrative expenses were $4.3 million for the three months ended September 30, 2019, compared to $2.3 million for the three months ended September 30, 2018. The increase was primarily driven by an increase in non-cash stock compensation expense of $1.4 million and an increase in compensation and benefits expense of $0.3 million as a result of increased headcount.
Net loss. Net loss was $19.3 million or $0.38 per share (basic and diluted) for the three months ended September 30, 2019, compared to $16.1 million or $0.40 per share (basic and diluted) for the three months ended September 30, 2018.
Shares outstanding. 50,376,030 shares of common stock were outstanding as of September 30, 2019.
Financial Guidance

Cash position. As of September 30, 2019, we had cash, cash equivalents and investments of $240.6 million. Rocket expects such resources will be sufficient to fund its operations into the first half of 2021.