On May 15, 2020 Precision BioSciences, Inc. (Nasdaq: DTIL), a life sciences company dedicated to improving life through the application of its pioneering, proprietary ARCUS genome editing platform, reported financial results for the first quarter ended March 31, 2020, and provided a business update (Press release, Precision Biosciences, MAY 15, 2020, View Source [SID1234558127]).
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"So far in 2020, we have continued to make significant progress across our portfolio. This includes advancing the ongoing Phase 1/2a trial of our lead allogeneic CAR T program candidate, PBCAR0191, in relapsed/refractory (R/R) NHL and B-ALL; dosing the first patient in a Phase 1/2a trial of PBCAR20A in R/R NHL, CLL and SLL; and preparing to launch a Phase 1/2a clinical trial of our third allogenic CAR T candidate, PBCAR269A, in multiple myeloma," said Matt Kane, CEO and co-founder of Precision BioSciences. "Amid COVID-19’s impact on the healthcare ecosystem, we believe this progress is not only a testament to our team’s dedication, but also a reminder of the significant disease burden and poor prognosis faced by late-stage cancer patients who have failed previous treatment lines. As evidenced by early data from our Phase 1/2a trial of PBCAR0191, our differentiated, off-the-shelf CAR T approach may offer meaningful clinical benefit for these patients, while potentially avoiding safety and logistical challenges faced by other cell therapies, including autologous CAR T. We continue to look forward to reporting updated data from the PBCAR0191 program later in 2020."
Recent Developments and Upcoming Milestones:
Allogeneic CAR T Portfolio:
PBCAR0191: PBCAR0191 is an investigational allogeneic CAR T candidate targeting CD19, currently being evaluated in a Phase 1/2a study in relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL) or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). The NHL cohort will include patients with mantle cell lymphoma (MCL), an aggressive subtype of NHL, for which Precision has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). In the first quarter of 2020, after discussion with the FDA, Precision implemented an amendment to the PBCAR0191 trial protocol designed to further optimize clinical activity. The amended trial design is intended to specifically address key clinical questions, which include assessing the impact of higher total doses of cells on clinical activity and/or the impact of modified lymphodepletion on the ability to achieve durable clinical benefit with
associated CAR T cell expansion and persistence. The PBCAR0191 clinical trial continues to progress, and no dose limiting toxicities or serious adverse events have been observed to date. Precision expects to present updated interim clinical data from both the NHL and B-ALL cohorts of this trial during 2020. PBCAR0191 is being developed in collaboration with Servier, an international pharmaceutical company.
PBCAR20A: PBCAR20A is a wholly-owned investigational allogeneic CAR T candidate targeting CD20 for the treatment of hematological malignancies. In April 2020, Precision dosed the first patient in its Phase 1/2a clinical trial evaluating PBCAR20A in two patient cohorts: R/R NHL, and R/R chronic lymphocytic leukemia (CLL) or R/R small lymphocytic lymphoma (SLL). Similar to the Phase 1/2a study of PBCAR0191, the NHL cohort for PBCAR20A will include patients with MCL, which has also received ODD from the FDA. Based on the safety profile observed from early dose levels in Precision’s ongoing Phase 1/2a study of PBCAR0191, the FDA authorized dosing in this Phase 1/2a study of PBCAR20A to begin at what was originally designed to be Dose Level 2 (1×106 cells/kg), with the subsequent dose level expected to be 3×106 cells/kg.
PBCAR269A: PBCAR269A is a wholly-owned investigational allogeneic CAR T candidate targeting B-cell maturation antigen (BCMA) for the treatment of R/R multiple myeloma, for which Precision has also received ODD. In January 2020, the FDA cleared Precision’s Investigational New Drug (IND) application for PBCAR269A, and the Company expects to begin dosing patients in a Phase 1/2a clinical trial in 2020.
In Vivo Gene Correction Portfolio:
PH1 Program: In January 2020, Precision announced that its first wholly-owned in vivo gene correction program will apply its ARCUS genome editing technology to knock out the HAO1 gene as a potential one-time treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disease. In 2020, Precision expects to select a clinical candidate for this program to advance into human trials.
ASGCT 2020: At the American Society of Gene & Cell Therapy 23rd Annual Meeting held May 12-15, 2020, multiple presentations were made by Precision and its collaborators supporting the Company’s ARCUS genome editing platform and emerging pipeline applying this technology in vivo. Posters and talks included:
Engineering a Self-Inactivating Adeno-Associated Virus (AAV) Vector for ARCUS Nuclease Delivery (Abstract #654)
Gene Editing Approach to Eliminate Hepatitis B Virus Using ARCUS Meganucleases (Abstract #1057)
Therapeutic Efficacy of ARCUS Meganuclease Gene Editing – Arrest of Rod Degeneration and Restoration of Rod Function in a Transgenic Pig Model of Autosomal Dominant Retinitis Pigmentosa (Abstract #2)
Evaluation of the Long-term Effects of AAV-Meganuclease Genome Editing of PCSK9 in Macaque Liver (Abstract #518)
Corporate
COVID-19: In April 2020, Precision provided an update regarding its clinical trials and business operations amid the COVID-19 pandemic. This includes steps taken in line with guidance from public
health officials to protect the health and safety of its employees and to ensure continuity of its clinical trials. Precision’s work-from-home policy and restriction of on-site activities to certain manufacturing functions and limited laboratory and support activities remain in effect. To date, Precision has not experienced material delays to its planned or ongoing clinical trials.
Senior Leadership and Board Appointments: Precision further strengthened its senior leadership team with the appointment of Dora Alvarado as Senior Vice President, Human Resources. The Company also recently welcomed Geno Germano, President and CEO of Elucida Oncology, and former head of Pfizer’s Global Innovative Pharmaceutical business, to its Board of Directors.
Quarter Ended March 31, 2020 Financial Results
Cash and Cash Equivalents: As of March 31, 2020, Precision had approximately $154.2 million in cash and cash equivalents. The Company expects that existing cash and cash equivalents will be sufficient to fund operating expenses and capital expenditure requirements into the second half of 2021.
Revenues: Total revenues for the quarter ended March 31, 2020 were $7.0 million, compared to $5.5 million for the quarter ended March 31, 2019. This increase was due to increases in both Therapeutic and Food segments.
Research and Development Expenses: Research and development expenses were $24.9 million for the quarter ended March 31, 2020, as compared to $20.0 million for the same period in 2019. This increase of $4.9 million was primarily due to increases in direct research and development expenses, as well as platform development and early stage research expenses, including increases in personnel costs, laboratory supplies and services and expenses to support Precision’s technology platform development and manufacturing capabilities.
General and Administrative Expenses: General and administrative expenses were $9.6 million for the quarter ended March 31, 2020, as compared to $5.0 million for the same period in 2019. The increase of $4.6 million was primarily due to an increase in employee-related costs for additional personnel and facility costs associated with the Company’s growing infrastructure needs.
Net Loss: Net loss was $26.8 million, or $(0.52) per share, for the quarter ended March 31, 2020, compared to a net loss of $31.8 million, or $(1.99) per share, for the same period in 2019.