On May 13, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies with its ARCUS genome editing platform, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Precision Biosciences, MAY 13, 2021, View Source [SID1234579942]).
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"In the first quarter, we have worked diligently to advance our ARCUS-based in vivo gene editing and allogeneic CAR T pipelines," commented Matt Kane, CEO and co-founder of Precision BioSciences. "In April, we seized the opportunity to reacquire all global rights previously granted to Servier for our CAR T programs, including PBCAR0191 and PBCAR19B. We believe that this repurchase, alongside our maturing datasets, will allow us to take full advantage of our CAR T programs as they progress through clinical development."
"We are on track to initiate the Phase 1 study of our PBCAR19B this month, our first candidate incorporating immune evading stealth cell technology as an alternative potential path to deeper and more durable responses. This will precede multiple anticipated clinical and research milestones across our portfolio in 2021, including interim updates from all three clinical-stage CAR T programs: PBCAR0191, PBCAR20A, and PBCAR269A. Additionally, we look forward to providing an update on our in vivo gene editing pipeline mid-year," concluded Mr. Kane.
Recent Developments and Upcoming Milestones:
Allogeneic CAR T Portfolio:
Reacquired Global Development and Commercialization Rights from Servier: In April 2021, Precision announced that it had entered into a Program Purchase Agreement to reacquire all global development and commercialization rights for all CAR T partnered programs covered under its Development and Commercial License Agreement with Servier. This includes its two CD19-targeting allogeneic CAR T candidates, PBCAR0191 and PBCAR19B stealth cell, plus four additional CAR T targets identified by Servier in 2020.
Under the terms of the Program Purchase Agreement, Precision paid $1.25 million in cash to Servier and agreed to waive earned, but as-yet unpaid milestones totaling $18.75 million that would have otherwise been payable to Precision. Servier is also eligible to receive milestones and low- to mid-single-digit royalties subject to product development achievement.
PBCAR0191: Precision has continued to advance its Phase 1/2a study of PBCAR0191 in patients with relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL) or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). Since the December 2020 interim data update, additional patients have been dosed with PBCAR0191 following enhanced lymphodepletion (eLD)1. Precision will continue to monitor the results for durability from this eLD regimen and expects to report updated interim results in June at ASCO (Free ASCO Whitepaper) 2021.
PBCAR19B: Precision has begun adding clinical trial sites for its Phase 1 study of PBCAR19B, the Company’s next generation, stealth cell, allogeneic CAR T candidate for patients with CD19-positive malignancies such as those with R/R NHL. The study is expected to begin by the end of May 2021. PBCAR19B will be administered at flat dose levels, beginning at 2.7 × 108 cells, with the ability to dose up to 8.1 × 108 cells per patient, following standard lymphodepletion2. The primary objective of the study is to identify the maximum tolerated dose and any dose-limiting toxicities.
PBCAR20A: Precision continues to enroll patients in its Phase 1/2a clinical trial of PBCAR20A, its anti-CD20 CAR T therapy for patients with R/R NHL, including patients with R/R chronic lymphocytic leukemia or R/R small lymphocytic lymphoma. In February 2021, the study began enrolling patients into dose level 3, a fixed dose of 480 × 106 cells with a max dose of 6.0 × 106 cells/kg. Precision expects to provide an interim update for the PBCAR20A study in 2021.
PBCAR269A: Precision continues to enroll patients in its Phase 1/2a study of PBCAR269A, its CAR T candidate targeting B-cell maturation antigen (BCMA) for the treatment of R/R multiple myeloma, for which Precision has received Fast Track Designation and Orphan Drug Designation from the FDA. In February 2021, the study began enrolling patients into its highest dose cohort, dose level 3 at 6.0 × 106 cells/kg and Precision expects to provide an interim update in 2021. Precision also expects to initiate the combination arm of its ongoing Phase 1/2a clinical study with PBCAR269A and nirogacestat, SpringWorks Therapeutics’ investigational gamma secretase inhibitor, in the first half of 2021.
PBCAR269B: In April 2021, Precision introduced PBCAR269B, a next-generation, BCMA-targeted candidate incorporating stealth cell technology, for treatment of R/R multiple myeloma. Precision is conducting IND-enabling studies for PBCAR269B and expects to file an IND application in early 2022.
In Vivo Gene Correction Portfolio:
Genome Editing Research Collaboration with Eli Lilly: In January 2021, Precision announced the closing of its agreement with Eli Lilly. Under the agreement, Precision will develop up to six in vivo therapies for genetic disorders using ARCUS, with an initial focus on Duchenne muscular dystrophy and two other undisclosed gene targets.
PH1: Pre-clinical research continues to progress with Precision’s wholly-owned in vivo gene correction program using 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. The Company expects to provide an update in mid-2021.
Corporate:
Executive Leadership: In April 2021, Precision announced that it has initiated a CEO transition plan for Co-Founder and Chief Executive Officer, Matt Kane. Mr. Kane is expected to continue in his current leadership capacity until his successor has been identified. He will also serve as an advisor for a period of time after the next CEO is hired ensuring a smooth transition.
Precision also announced the appointment of Alan List, M.D. as the Company’s Chief Medical Officer and a member of the senior leadership team. Dr. List is a world-renowned hematologist with extensive academic and clinical experience in the research and development of hematology and oncology products.
Board of Directors: Precision announced that it has strengthened its Board of Directors with the appointment of Stanley R. Frankel, M.D., a hematologist-oncologist with extensive academic and industry experience in the research, clinical development, and commercialization of immuno-oncology and cellular therapies. Dr. Frankel was most recently the Senior Vice President, Cellular Therapy Development at Bristol-Myers Squibb (BMS). Prior to the BMS acquisition of Celgene, he was Corporate Vice President, Head, Immuno-Oncology & Cellular Therapy, Clinical Research and Development Head, Cell Therapy Clinical Center of Excellence at Celgene.
Elo Life Systems:
Corporate Structure: In January 2021, Precision disclosed its intention to spin out its wholly-owned subsidiary, Elo Life Systems. Precision is continuing to explore its strategic options, and expects to complete any such spinout, sale or other treatment of Elo in 2021.
Quarter Ended March 31, 2021 Financial Results
Cash and Cash Equivalents: As of March 31, 2021, Precision had approximately $193.5 million in cash and cash equivalents, which includes the $100 million upfront cash payment and equity investment of $35 million received from Eli Lilly in January 2021. The Company expects that existing cash and cash equivalents, expected operational receipts, and available credit will be sufficient to fund its operating expenses and capital expenditure requirements into 2023.
Revenues: Total revenues for the first quarter ended March 31, 2021 were $16.3 million, as compared to $7.0 million for the same period in 2020.
Research and Development Expenses: Research and development expenses were $25.6 million for the quarter ended March 31, 2021, as compared to $24.9 million for the same period in 2020.
General and Administrative Expenses: General and administrative expenses were $9.5 million for the quarter ended March 31, 2021, as compared to $9.6 million for the same period in 2020.
Net Loss: Net loss was $18.7 million, or $(0.33) per share, for the quarter ended March 31, 2021, as compared to a net loss of $26.8 million, or $(0.52) per share, for the same period in 2020.