DTIL Precision BioSciences Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
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PBCAR0191
Relapsed or refractory B-cell precursor acute lymphoblastic leukemia and R/R non-hodgkin lymphoma
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Phase 1/2
Phase 1/2
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PBCAR269A
Multiple Myeloma
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Phase 1/2
Phase 1/2
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PBCAR20A
Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL)
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Phase 1/2
Phase 1/2
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
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PBCAR19B
Non-Hodgkin Lymphoma (NHL)
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Phase 1
Phase 1
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Phase 1 trial to commence mid-2021.
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PBCAR269A
Multiple myeloma
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Phase 1/2
Phase 1/2
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Phase 1/2 dosing of first patient announced June 8, 2020.
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Latest News
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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, today announced it will publish first quarter 2021 financial results and provide a business update on Thursday, May 13, 2021.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Company's pipeline consists of multiple "off-the-shelf" CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210506005331/en/
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Preclinical Research with ARCUS Genome Editing Rejuvenated Rod Photoreceptor Structure and Function in a Transgenic Pig Model of Human Blindness
Precision BioSciences, Inc. (NASDAQ:DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies, announced today that the following paper presentation, highlighting preclinical research using its ARCUS® genome editing platform for autosomal dominant Retinitis Pigmentosa (adRP), will be presented today at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.
Title: Rho 1-2 meganuclease, an allele-specific gene-editing therapy, rejuvenates rod photoreceptor structure and function in a pig model of autosomal dominant Retinitis Pigmentosa (adRP)
Paper Presentation: Gene Therapy - Physiology/Pharmacology, Abstract 3543926
Date/Time: Monday, May 3, 2021, 4:30 – 6:00 p.m. EDT
Presenting Author: Archana Jalligampala, Ph.D., Postdoctoral Associate, Department of Ophthalmology and Visual Sciences, University of Louisville
Co-Authors: Jennifer Noel1, James W. Fransen1, Wei Wang1, Maha H. Jabbar1, Nazarul Hasan1, Gobinda Pangeni1, Bhubanananda Sahu1, Whitney Lewis2, Jeff Smith2, Victor Bartsevich2, Kristi Viles2, Derek Jantz2, Maureen A. McCall1The P23H mutation in the rhodopsin gene represents the most common form of adRP in North Americans. Mutations in the rhodopsin gene account for 25 to 30 percent of all cases of adRP in the United States. This leads to the progressive loss of rods, which are responsible for vision at low light levels.
"This form of adRP, an inherited eye disease that leads to complete loss of central vision, is not amenable to conventional gene replacement therapies," said Archana Jalligampala, Ph.D., Postdoctoral Associate, Department of Ophthalmology and Visual Sciences, University of Louisville. "We used ARCUS nucleases to target the P23H human rhodopsin mutation and found that, at our latest scheduled structural and functional assessment at 44 weeks post-injection, rods in the treated P23H retinas were more numerous, had elongated outer segments, and correctly localized rhodopsin compared to the untreated area in the same P23H retina or to an untreated P23H retina. We're very encouraged by these results as they indicate that the gene editing approach using ARCUS has the potential to treat adRP in human patients with the same point mutation and, importantly, at late disease stages."
In this study, ARCUS nuclease-treated animals showed significant improvements in rod driven signals, namely an increase in electroretinography (ERG) b-wave response compared to untreated animals. This effect was observed as early as postnatal day (P) 60 and was maintained through P300. These results suggest that the level of gene editing achieved in this study may facilitate functional visual improvements.
"We are excited to see that this latest large animal, preclinical dataset using ARCUS to address adRP will be presented at the annual ARVO meeting," commented Derek Jantz, Ph.D., Co-Founder and Chief Scientific Officer at Precision BioSciences. "This work adds to the growing body of preclinical research where in vivo gene editing with ARCUS has resulted in sustained effective responses. We look forward to the continued development of ARCUS for rare genetic conditions like adRP that lack a safe and effective treatment option."
Video-recorded presentations from the ARVO 2021 meeting are available on the Pathable online platform.
About ARCUS
ARCUS® is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock-in), remove (knock-out), or repair DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA. Precision's platform and products are protected by a comprehensive portfolio including more than 75 patents to date.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the potential results, uses and advancement of our in vivo gene editing programs and ARCUS-based gene editing technology, including, without limitation, its attributes and effects upon autosomal dominant Retinitis Pigmentosa (adRP). In some cases, you can identify forward-looking statements by terms such as "aim," "anticipate," "believe," "could," "eligible," "expect," "should," "plan," "intend," "estimate," "target," "mission," "goal," "may,", "suggest", "will," "would," "should," "could," "target," "potential," "project," "predict," "contemplate," "potential," or the negative thereof and similar words and expressions.
Forward-looking statements are based on management's current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators' ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' development of product candidates; our or our collaborators' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators' ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators' ability to enroll patients; changes in interim "top-line" and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events effects of the outbreak of COVID-19, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at www.sec.gov and the Investors & Media page of our website at investor.precisionbiosciences.com.
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Company's pipeline consists of multiple "off-the-shelf" CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
1 University of Louisville, Louisville, KY, USA
2 Precision BioSciences, Inc., Durham, NC, USAView source version on businesswire.com: https://www.businesswire.com/news/home/20210503005367/en/
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Preclinical Study Shows ARCUS Genome Editing May Be a Promising Approach for the Treatment of Transthyretin Amyloidosis
Precision BioSciences, Inc. (NASDAQ:DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies, today announced that the following poster, highlighting a preclinical research collaboration using its ARCUS® genome editing platform for treatment of transthyretin amyloidosis (ATTR), will be presented at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, scheduled for May 11-14, 2021.
Title: Translation of an AAV-delivered gene editing approach for transthyretin amyloidosis in animal models
Poster Session: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases, Abstract 497
Date/Time: Tuesday May 11, 2021 8:00 AM - 10:00 AM
Presenting Author: Jenny A. Greig, Ph.D., Senior Director, Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania
Co-Authors: Cassandra Gorsuch2, Joanna K. Chorazeczewski1, Melanie K. Smith1, Thomas Furmanak1, Alexa N. Avitto1, Scott N. Ashley1, Wendy Sharer2, Hui Li2, Jeff Smith2, Peter Clark1, Camilo Breton1, Derek Jantz2, and James M. Wilson1Transthyretin amyloidosis is a rare disease caused by the progressive accumulation of misfolded transthyretin (TTR) protein into amyloid fibrils, which leads to peripheral neuropathy and/or cardiomyopathy. Research to be presented at the annual ASGCT meeting, led by Dr. Jenny A. Greig at the Perelman School of Medicine, University of Pennsylvania, used an AAV vector for in vivo delivery of ARCUS gene editing nucleases to knock out the TTR gene, which is responsible for ATTR.
"With this program, we are excited to continue building a dataset demonstrating in vivo gene editing in large animal models using ARCUS nucleases," said Derek Jantz, Ph.D., Chief Scientific Officer and Co-Founder of Precision BioSciences. "In this study, use of an optimized ARCUS nuclease to knock out the TTR gene was found to be effective in both mice and nonhuman primates, where we observed a good correlation between TTR gene editing in the liver and reductions of TTR in the serum. This approach addresses the root cause of the disease and results in genomic edits that are expected to be permanent. These results continue to demonstrate the power and versatility of ARCUS nucleases, particularly for in vivo editing."
Abstracts for the ASGCT 2021 Meeting are available on the meeting website.
About ARCUS
ARCUS® is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock-in), remove (knock-out), or repair DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA. Precision's platform and products are protected by a comprehensive portfolio including more than 75 patents to date.About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Company's pipeline consists of multiple "off-the-shelf" CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding statements regarding the potential results, uses and advancement of our in vivo gene editing programs and ARCUS-based gene editing technology, including, without limitation, its attributes and effects upon the transthyretin gene, transthyretin serum levels and transthyretin amyloidosis. In some cases, you can identify forward-looking statements by terms such as "aim," "anticipate," "believe," "could," "eligible," "expect," "expected", "should," "plan," "intend," "estimate," "target," "mission," "goal," "may," "will," "would," "should," "could," "target," "potential," "potentially", "promising", "project," "predict," "contemplate," "potential," or the negative thereof and similar words and expressions.Forward-looking statements are based on management's current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators' ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' development of product candidates; our or our collaborators' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators' ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators' ability to enroll patients; changes in interim "top-line" and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events effects of the outbreak of COVID-19, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at www.sec.gov and the Investors & Media page of our website at investor.precisionbiosciences.com.
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
1 Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA
2 Precision BioSciences, Inc., Durham, NC, USAView source version on businesswire.com: https://www.businesswire.com/news/home/20210427006142/en/
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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, today announced that Alan List, M.D. has been appointed as the Company's Chief Medical Officer and a member of the senior leadership team at Precision BioSciences. Dr. List is a world-renowned hematologist with extensive academic and clinical experience in the research and development of hematology and oncology products. He has led clinical development programs for multiple FDA-approved drugs to treat hematologic malignancies, including myelodysplastic syndromes, acute leukemia, multiple myeloma, and lymphoma.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210415006059/en/
Precision BioSciences Appoints Dr. Alan List Chief Medical Officer (Photo: Business Wire)
"We are very pleased to welcome Alan to the senior leadership team of Precision BioSciences," said Matt Kane, Chief Executive Officer and Co-Founder of Precision BioSciences. "With three hematology products in the clinic and a fourth entering clinical trials soon, Alan's deep clinical expertise in hematology and oncology, including pioneering work to develop novel products from the laboratory to the clinic, will be a strong addition to the Precision team. Over the past 12 months, Alan has been intimately involved in our clinical strategy to develop our lead allogeneic CAR T therapy, PBCAR0191, as well as the design of our clinical program for PBCAR19B, our next generation, stealth cell program. We believe that Alan's leadership will be critical to the successful execution of our clinical development strategy."
Since April 2020, Dr. List has been a strategic clinical advisor to Precision BioSciences, providing advice to the company and its Board of Directors on its clinical stage and pre-clinical allogeneic CAR T programs. Prior to joining Precision BioSciences, Dr. List served in various roles at the Moffitt Cancer Center, including as President and Chief Executive Officer from 2012 to 2019; Executive VP, Physician in Chief from 2008 to 2012 and Chief of the Malignant Hematology Division from 2003 to 2008. Dr. List is internationally recognized for his many contributions in the development of effective treatment strategies for myelodysplastic syndrome (MDS) and acute myeloid leukemia. His pioneering work led to the development of Revlimid (lenalidomide) a transformational treatment for patients with MDS and multiple myeloma.
Dr. List is the author of more than 425 peer-reviewed articles and books. He previously served as the President for the Society of Hematologic Oncology and a member of the MDS Foundation Board of Directors. Dr. List is an active member of the American Society of Clinical Oncology, the American Society of Hematology and the American Association for Cancer Research. He is a Charter Fellow in the National Academy of Inventors, an inductee in the Florida Inventors Hall of Fame, and holds 18 U.S. patents and >45 applications filed. Dr. List was 2016 recipient of the Celgene Career Achievement Award for Clinical Research in Hematology, and other recognitions including the General Motors Cancer Research Foundation Merit Award, the J.P. McCarthy Foundation International Prize, the Emil J. Freireich Award, the Joshua Lederberg Society, and the Aplastic Anemia & MDS International Foundation Leadership in Science Award.
Dr. List received B.S. and M.S. degrees from Bucknell University and earned his M.D. from the University of Pennsylvania. He is board certified in internal medicine, hematology, and medical oncology. He completed his residency in internal medicine at Good Samaritan Medical Center in Phoenix, Arizona and fellowships in hematology and medical oncology at Vanderbilt University Medical Center. Prior to joining the Moffitt Cancer Center in 2003, Dr. List held academic and clinical appointments at the University of Arizona.
"After advising Precision BioSciences on its clinical trial programs for nearly a year, I'm very excited to join as the Chief Medical Officer as we approach the launch of the first of our stealth CAR T programs. I look forward to working with the management team and the clinical team to advance the development of our potentially breakthrough allogeneic CAR T cell programs, including PBCAR0191 and PBCAR19B. I'm particularly optimistic about the potential opportunity to bring novel cell therapies to patients who suffer from hematologic malignancies, such as non-Hodgkin lymphoma, B-cell acute lymphoblastic leukemia, and multiple myeloma," said Dr. List. "I'm also looking forward to interacting with the clinical investigators who are conducting our clinical trials at leading academic centers across the country."
Dr. List will succeed Chris Heery, M.D., who is leaving Precision BioSciences to pursue other opportunities. Dr. Heery will continue to serve as a consultant to the company to ensure a smooth transition in the conduct of clinical trials underway at Precision.
"During Chris' time with the company, Precision successfully transitioned PBCAR20A and PBCAR269A into Phase 1 clinical trials and designed the Phase 1 clinical trial for PBCAR19B. We thank Chris for his contributions to Precision BioSciences and wish him success in his future endeavors," added Mr. Kane.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Company's pipeline consists of multiple "off-the-shelf" CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our leadership transition and expectations regarding our operational initiatives and success of our clinical programs. In some cases, you can identify forward-looking statements by terms such as "aim," "anticipate," "believe," "could," "expect," "should," "plan," "intend," "estimate," "target," "mission," "goal," "may," "will," "would," "should," "could," "target," "potential," "project," "predict," "contemplate," "potential," or the negative thereof and similar words and expressions. Forward-looking statements are based on management's current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators' ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' development of product candidates; our or our collaborators' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators' ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators' ability to enroll patients; changes in interim "top-line" and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events effects of the outbreak of COVID-19, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption "Risk Factors" in our Annual report on Form 10-K for the fiscal year ended December 31, 2020, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC's website at www.sec.gov and the Investors & Media page of our website at investor.precisionbiosciences.com.
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210415006059/en/
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- Precision Reacquires from Servier its Global Development and Commercialization Rights and Control of All Partnered CAR T Programs, Including PBCAR0191 and PBCAR19B Stealth Cell
- Servier Eligible to Receive Milestones and Royalties Based on Program Success
- Precision to Host Conference Call and Webcast Today at 5:00 p.m. ET
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, today announced it has 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 clinical stage CD19-targeting allogeneic CAR T candidates, PBCAR0191 and PBCAR19B stealth cell, as well as four additional product targets.
"We are excited about the potential of our allogeneic CAR T pipeline to deliver off-the-shelf treatments for patients with cancer," said Matt Kane, CEO and Co-Founder of Precision BioSciences. "We believe we are in a unique position with multiple near-term opportunities to achieve success with allogeneic CAR T cells targeting CD19. Our lead candidate, PBCAR0191, continues to look promising when paired with our enhanced lymphodepletion regimen, and our immune-evading PBCAR19B stealth cell candidate is poised to enter the clinic soon. We seized the opportunity to reacquire these two CD19 programs, as well as four additional targets that were selected by Servier in 2020 to regain global commercial rights and full control of our clinical programs, allowing us to focus our resources and enable rapid decision making."
Under the terms of the Program Purchase Agreement, Servier will receive $1.25 million in cash and Precision has 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. With respect to products directed to CD19, Servier has certain rights of negotiation if Precision elects to re-partner the programs.
"Precision was a very good partner for us, and we continue to believe their proprietary, single-step cell engineering technology has the potential to open the door to an off-the-shelf approach for addressing cancer," said Patrick Therasse, Deputy Head of R&D Oncology at Servier. "While we have made the strategic decision to refocus our R&D activities and we will no longer be taking an active role in the Precision programs, we continue to believe they have multiple opportunities, including the stealth cell approach, to benefit patients with hematologic malignancies and solid tumors, so we are pleased to be able to participate in the potential future success of these programs."
Subsequent to Precision's December 2020 interim Phase 1/2a study data update for PBCAR0191, enrollment in the study has continued, with a focus on dose level 3 (3 x 106 cells/kg) following enhanced lymphodepletion (eLD)1. Initial response rates are consistent with the high response rates reported by Precision in December 2020, and the safety profile continues to be acceptable. Precision intends to monitor the results for durability from this eLD regimen and report updated interim results by mid-2021.
By the end of May 2021, Precision expects to dose the first patient with PBCAR19B, its second CD19 allogeneic CAR T candidate that is engineered with Precision's proprietary stealth cell technology. Although it has not finalized its full financial results for the first quarter ended March 31, 2021, Precision BioSciences had cash and cash equivalents of approximately $193 million as of March 31, 2021 and continues to expect that cash and cash equivalents, expected operational receipts, and available credit will allow the Company to continue its operations into 2023.
Company-Hosted Conference Call and Webcast Information
Precision's management team will host a conference call and webcast at 5:00 p.m. ET, Thursday, April 15, 2021 to discuss today's announcement. The dial-in conference call numbers for domestic and international callers are (866) 996-7202 and (270) 215-9609, respectively. The conference ID number for the call is 8170529. Participants may access the live webcast and the accompanying presentation materials on Precision's website https://investor.precisionbiosciences.com/events-and-presentations in the Investors and Media section under Events and Presentations. An archived replay of the webcast will be available on Precision's website for approximately 30 days.
About PBCAR0191 (Clinical Trials Study Identifier: NCT03666000)
PBCAR0191 is an investigational allogeneic CAR T in a Phase 1/2a clinical trial for the treatment of patients with R/R NHL and R/R B-ALL. PBCAR0191 was designed using Precision BioSciences' novel and proprietary ARCUS® genome editing platform. It has been granted Fast Track Designation by the FDA for the treatment of R/R B-ALL. Precision also holds Orphan Drug Designation from the FDA for this program in mantle cell lymphoma, an aggressive subtype of NHL.
In December 2020, Precision BioSciences reported positive interim results from this study, in which 27 patients with R/R NHL or R/R B-ALL were dosed with PBCAR0191 CAR T therapy and showed no graft versus host disease, no grade ≥ 3 cytokine release syndrome, and no grade ≥ 3 neurotoxicity. For those NHL and B-ALL patients dosed with PBCAR0191, when combined with eLD, objective response rates reached 83% (5/6).
About PBCAR19B (Clinical Trials Study Identifier: NCT04649112)
PBCAR19B is a next-generation, stealth cell candidate for patients with CD19-positive malignancies such as R/R NHL. PBCAR19B is designed to improve the persistence of allogeneic CAR T cells following infusion by reducing rejection by T cells and NK cells. In addition to the CAR gene, the PBCAR19B stealth cell vector carries a short hairpin RNA that suppresses expression of beta-2 microglobulin, a component of Class I Major Histocompatibility Complex (MHC) molecules found on the cell surface. Reducing or knocking-down Class I MHC expression on allogeneic CAR T cells has been shown to reduce CAR T cell killing by cytotoxic T cells. The PBCAR19B vector also carries an HLA-E gene intended to reduce rejection of CAR T cells by NK cells that can be stimulated as a result of reduced MHC molecule expression on the cell surface.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Company's pipeline consists of multiple "off-the-shelf" CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding potential milestone payments under the purchase agreement with Servier, the expected timing of trials and results from clinical studies of CAR T product candidates and in vivo gene editing programs, and expectations regarding our liquidity, anticipated cash payments and our ability to fund operations. In some cases, you can identify forward-looking statements by terms such as "aim," "anticipate," "believe," "could," "eligible," "expect," "should," "plan," "intend," "estimate," "target," "mission," "goal," "may," "will," "would," "should," "could," "target," "potential," "project," "predict," "contemplate," "potential," or the negative thereof and similar words and expressions.
Forward-looking statements are based on management's current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators' ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' development of product candidates; our or our collaborators' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators' ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators' ability to enroll patients; changes in interim "top-line" and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events effects of the outbreak of COVID-19, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at www.sec.gov and the Investors & Media page of our website at investor.precisionbiosciences.com.
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
1 Fludarabine (30 mg/m/day for 4 days) and cyclophosphamide (1000 mg/m2/day for 3 days)
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