On August 13, 2020 Precision BioSciences, Inc. (Nasdaq: DTIL) a clinical-stage biotechnology company dedicated to improving life with its novel and proprietary ARCUS genome editing platform, reported financial results for the second quarter ended June 30, 2020 and provided a business update (Press release, Precision Biosciences, AUG 13, 2020, View Source [SID1234563569]).
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"We have continued to make important progress across our clinical portfolio, including the initiation of our Phase 1/2a study of BCMA-targeted PBCAR269A, bringing us to three CAR T candidates now in clinical trials. However, due to study site activation and patient enrollment delays related to the COVID-19 pandemic, we now anticipate reporting updated interim data from our lead candidate, PBCAR0191, targeting CD19 in heavily pretreated patients with R/R NHL or B-ALL, no earlier than the fourth quarter of 2020," said Matt Kane, CEO and co-founder of Precision BioSciences. "Our pre-clinical work also continued to progress, including our lead gene correction program targeting PH1, for which we look forward to nominating a clinical candidate later this year. We anticipate sharing more about this program as it advances towards the clinic, including what we believe are prudent safety and delivery advantages with ARCUS genome editing that support additional in vivo targets of interest."
Recent Developments and Upcoming Milestones
Allogeneic CAR T Portfolio
PBCAR0191: PBCAR0191 is an investigational allogeneic chimeric antigen receptor (CAR T) candidate targeting CD19 and is 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 includes patients with mantle cell lymphoma (MCL), an aggressive subtype of NHL, for which Precision has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). The PBCAR0191 clinical trial has continued to progress, although site activation and patient enrollment across all studies, including PBCAR0191, has slowed due to the COVID-19 pandemic. Precision expects to share an update of this trial no earlier than the fourth quarter of this year. 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. Precision’s Phase 1/2a clinical trial is evaluating PBCAR20A in two patient cohorts: R/R NHL, and R/R chronic lymphocytic leukemia (CLL) or R/R small lymphocytic lymphoma (SLL). The NHL cohort will include patients with MCL, an aggressive subtype of NHL, for which Precision has received Orphan Drug Designation from the FDA.
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 received Orphan Drug Designation from the FDA. In June 2020, the Company dosed the first patient in a Phase 1/2a study of PBCAR269A, at a starting dose of 6 x 105 CAR T cells/kg body weight. Subsequent cohorts will be treated with escalating doses to a maximum dose of 6 x 106 CAR T cells/kg body weight. This is the first study for which all clinical trial materials will be produced at the Company’s in-house manufacturing facility, which is compliant with current Good Manufacturing Practices. In preclinical disease models, PBCAR269A demonstrated potent in vivo clearance of BCMA+ tumor cells and overall volume reduction, with no evidence of graft-versus-host disease.
In Vivo Gene Correction Portfolio
PH1 Program: Precision’s lead, wholly-owned in vivo gene correction program applies 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. Precision expects to select a clinical candidate to advance into human trials for this program during 2020.
HBV Program: In July 2020, Precision announced it will regain full clinical development and commercialization rights and all data it generated for the in vivo chronic hepatitis B virus (HBV) program developed under its 2018 collaboration agreement with Gilead Sciences, effective September 4, 2020. The Company is exploring partnership or alternative opportunities that enable the continued development and potential commercialization of ARCUS-based HBV therapies.
Elo Life Systems
Dole Collaboration: On August 4, 2020, Elo Life Systems, a wholly-owned subsidiary of Precision BioSciences, announced its strategic collaboration with Dole Food Company, one of the world’s largest food producers of high-quality fresh fruit and vegetables, with the aim to develop banana varieties resistant to Fusarium wilt Tropical Race 4 (TR4). The TR4 strain of the fungal pathogen, Fusarium oxysporum threatens the continued cultivation of the Cavendish variety, one of the world’s most popular bananas. Under the terms of the collaboration, Dole will fully fund research and development efforts executed by Elo to co-develop banana varieties resistant to a pathogen that is otherwise unresponsive to any control measures. Elo is eligible to receive royalties on any commercialized plant product.
Quarter Ended June 30, 2020 Financial Results
Cash and Cash Equivalents: As of June 30, 2020, Precision had approximately $126.9 million in cash and cash equivalents. The Company expects that existing cash, cash equivalents and available credit will be sufficient to fund operating expenses and capital expenditure requirements into 2022.
Revenues: Total revenues for the quarter ended June 30, 2020 were $1.1 million, compared to $5.4 million for the quarter ended June 30, 2019. This decrease of $4.3 million was primarily due to a decrease in collaboration revenue recognized from Servier and Gilead.
Research and Development Expenses: Research and development expenses were $25.2 million for the quarter ended June 30, 2020, as compared to $22.8 million for the same period in 2019. This increase of $2.4 million was primarily due to increases in direct research and development expenses related to the commencement of our CD20 and BCMA Phase 1/2a clinical trials and our ongoing CD19 clinical program.
General and Administrative Expenses: General and administrative expenses were $8.7 million for the quarter ended June 30, 2020, as compared to $6.5 million for the same period in 2019. The increase of $2.2 million was primarily due to costs associated with the Company’s growing infrastructure needs.
Net Loss: Net loss was $32.7 million, or $(0.63) per share, for the quarter ended June 30, 2020, compared to a net loss of $19.4 million, or $(0.39) per share, for the same period in 2019.