On August 12, 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 second quarter ended June 30, 2021 and provided a business update (Press release, Precision Biosciences, AUG 12, 2021, View Source [SID1234586377]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"In the second quarter of 2021, we continued to execute on our ARCUS-edited allogeneic CAR T programs, including our anti-CD19 studies. We continue to monitor for durability of response for our PBCAR0191 dosing protocol with enhanced lymphodepletion following the interim results presented in June 2021. We also dosed the first patient in our Phase 1 study of PBCAR19B, our next-generation anti-CD19 candidate designed to evade the immune system by avoiding both T and NK cell rejection," said Matt Kane, Chief Executive Officer at Precision BioSciences. "This quarter we also advanced our preclinical in vivo gene editing programs and look forward to sharing more about our development strategy and plans to advance select in vivo programs into the clinic at our upcoming gene editing event in September."
Recent Developments and Upcoming Milestones:
Allogeneic CAR T Portfolio:
PBCAR0191: In June 2021, Precision reported updated data for its lead anti-CD19 CAR T candidate, PBCAR0191. As of May 21, 2021, 18 subjects with Relapsed/Refractory (R/R) non-Hodgkin lymphoma (NHL) completed Day 28 evaluation and received either enhanced lymphodepletion (eLD, n=12) or standard lymphodepletion (sLD, n=6) with Dose Level 3 (3.0 × 106 cells per kg) of PBCAR0191. After a single dose of PBCAR0191 following eLD, overall response rates and complete response rates were 75% and 50%, respectively, at Day ≥ 28. Five of nine responding patients (56%) who received PBCAR0191 cells following eLD remained progression-free, including 4/9 evaluable subjects with responses lasting greater than 4 months. PBCAR0191 with eLD continues to show acceptable tolerability with no evidence of graft versus host disease (GvHD) and with a similar frequency of immune effector cell-associated neurotoxicity syndrome (ICANS) and cytokine release syndrome (CRS) compared to patients who received sLD. A subset of this data set was presented at ASCO (Free ASCO Whitepaper) 2021.
PBCAR19B: In July 2021, Precision announced that the first patient in the Phase 1 study of PBCAR19B, the Company’s anti-CD19 immune-evading stealth cell candidate for patients with R/R NHL. PBCAR19B is being administered at flat dose levels, with the first dose level (2.7 × 108 CAR T cells per patient) comparable to Dose Level 3 of PBCAR0191. The primary objective of the study is to identify the maximum tolerated dose and any dose-limiting toxicities.
PBCAR20A: Precision has completed Dose Level 3 (4.8 × 108 cells per patient given as a fixed dose) of its Phase 1/2a anti-CD20 study of PBCAR20A and has paused the study until PBCAR0191 and PBCAR19B durability is demonstrated. Precision expects to provide an interim update for PBCAR20A in 2021.
PBCAR269A: Precision continues to enroll patients in its Phase 1/2a study of PBCAR269A targeting B-cell maturation antigen (BCMA) for patients with R/R multiple myeloma. In June 2021, Precision announced that it dosed the first patient in its combination arm with PBCAR269A and nirogacestat, a gamma secretase inhibitor (GSI) being developed by SpringWorks. Emerging preclinical and clinical data suggest that a GSI may increase antitumor efficacy of BCMA-targeted autologous CAR T therapy in patients with relapsed or refractory multiple myeloma. Precision expects to provide an interim update on the monotherapy arm of the study in 2021.
PBCAR269B: In April 2021, Precision announced it started conducting IND-enabling studies for PBCAR269B, its next-generation immune-evading stealth cell candidate targeting BCMA.
In Vivo Gene Correction Portfolio:
ARCUS to Target Mitochondrial DNA: In June 2021, Precision announced a paper was published in Nature Communications and highlighted the use of ARCUS genome editing to target mutant mitochondrial DNA. Led by Carlos T. Moraes, Ph.D., Esther Lichtenstein Professor in Neurology at the University of Miami Miller School of Medicine, with Ugne Zekonyte as first author, researchers reported effective use of a mitochondrial-targeted ARCUS nuclease (mitoARCUS) to edit mutant mtDNA. This is the first time ARCUS has been used to edit outside the nuclear genome and has done so with encouraging safety and efficacy in this mouse model. These results were also discussed during the United Mitochondrial Disease Foundation’s Mitochondrial Medicine 2021 Virtual "Meet the Scientific Program Faculty" in June 2021.
Gene Editing Event: Precision will host its first in vivo focused gene editing event on Thursday, September 9, 2021. The virtual event will include presentations on the Company’s in vivo gene editing business strategy, including pipeline development plans and timelines to the clinic for certain gene editing programs such as its wholly-owned PH1 program. The virtual event is expected to last approximately two hours and will be a live video webcast available through the Company’s website. Additional details for the event will follow.
Corporate:
Executive Leadership: In May 2021, Precision announced that Alex Kelly had been appointed as Chief Financial Officer, a role in which he had served in an interim capacity since January 2021. Alex oversees Precision’s finance, corporate communications, investor relations, IT, facilities and operations functions. Shane Barton, the Company’s Vice President and Corporate Controller, had been serving as interim principal accounting officer and will now serves as principal accounting officer.
Elo Life Systems:
Corporate Structure: Precision continues to explore strategic options and expects to complete any such spinout, sale or other treatment of Elo in 2021.
Quarter Ended June 30, 2021 Financial Results
Cash and Cash Equivalents: As of June 30, 2021, Precision had approximately $173.9 million in cash and cash equivalents. 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 second quarter ended June 30, 2021 were $68.8 million, as compared to $1.1 million for the same period in 2020. The increase of $67.7 million in revenues during the three months ended June 30, 2021 was primarily the result of a $62.0 million increase in revenue recognized under the Servier Agreement, as the performance obligation was deemed fully satisfied upon the execution of the Program Purchase Agreement. In the second quarter, the Company also recognized $5.3 million in revenue under the Lilly Agreements, compared to no revenue for the same period in 2020, as work commenced in 2021.
Research and Development Expenses: Research and development expenses were $37.2 million for the quarter ended June 30, 2021, as compared to $25.2 million for the same period in 2020. The increase of $12.0 million in research and development expenses was primarily the result of $11.3 million in expense related to the Servier Program Purchase Agreement that was recognized in the quarter ended June 30, 2021.
General and Administrative Expenses: General and administrative expenses were $9.9 million for the quarter ended June 30, 2021, as compared to $8.7 million for the same period in 2020.
Net Income (loss): Net income was $21.7 million, or $0.38 per share (basic) and $0.36 per share (diluted), for the quarter ended June 30, 2021, as compared to a net loss of ($32.7 million), or $(0.63) per share (basic and diluted) for the same period in 2020. Weighted average common shares outstanding for the quarter ended June 30, 2021 were 57,739,622 (basic) and 59,841,638 (diluted), compared to 51,909,240 (basic and diluted) for the same period in 2020.