- Precision to Receive $75 Million Upfront for
a Single Target; Precision Eligible to Receive up to an Additional
$1.4 Billion in Milestones and Tiered Royalties on Sales of
Licensed Products
- Precision to Develop a Single ARCUS®
Nuclease Designed for Safe and Efficient In Vivo Gene Insertion
- Collaboration Combines Precision’s
Proprietary ARCUS Genome Editing Platform and Gene Insertion
Capabilities with Novartis’ Drug Discovery and Gene Therapy
Expertise
- Extends Precision’s Cash Runway into Q2
2024
- Precision to Host Conference Call Tomorrow,
June 22, 2022, at 8:00 AM ET
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage
gene editing company developing ARCUS-based ex vivo allogeneic CAR
T and in vivo gene editing therapies, today announced it has
entered into an exclusive worldwide in vivo gene editing research
and development collaboration and license agreement with Novartis
Pharma AG (the “Agreement”). As part of the Agreement, Precision
will develop a custom ARCUS nuclease that will be designed to
insert, in vivo, a therapeutic transgene at a “safe harbor”
location in the genome as a potential one-time transformative
treatment option for diseases including certain hemoglobinopathies
such as sickle cell disease and beta thalassemia.
Under the terms of the Agreement, Precision will develop an
ARCUS nuclease and conduct in vitro characterization, with Novartis
then assuming responsibility for all subsequent research,
development, manufacturing and commercialization activities.
Novartis will receive an exclusive license to the custom ARCUS
nuclease developed by Precision for Novartis to further develop as
a potential in vivo treatment option for sickle cell disease and
beta thalassemia. Precision will receive an upfront payment of $75
million and is eligible to receive up to an aggregate amount of
approximately $1.4 billion in additional payments for future
milestones. Precision is also eligible to receive certain research
funding and, should Novartis successfully commercialize a therapy
from the collaboration, tiered royalties ranging from the
mid-single digits to low-double digits on product sales.
“We are excited to collaborate with Novartis to bring together
the precision and versatility of ARCUS genome editing with
Novartis’ gene therapy expertise and commitment to developing
one-time, potentially transformative treatment for hard-to-treat
inherited blood disorders,” said Michael Amoroso, Chief Executive
Officer at Precision BioSciences. “This collaboration will build on
the unique gene insertion capabilities of ARCUS and illustrates its
utility as a premium genome editing platform for potential in vivo
drug development. With this Agreement, Precision, either alone or
with world-class partners, will have active in vivo gene editing
programs for targeted gene insertion and gene deletions in
hematopoietic stem cells, liver, muscle and the central nervous
system showcasing the distinctive versatility of ARCUS.”
“We identify here a collaborative opportunity to imagine a
unique therapeutic option for patients with hemoglobinopathies,
such as sickle cell disease and beta thalassemia – a potential
one-time treatment administered directly to the patient that would
overcome many of the hurdles present today with other therapeutic
technologies,” said Jay Bradner, President of the Novartis
Institutes for Biomedical Research (NIBR), the Novartis innovation
engine. “We look forward to working with Precision and leveraging
the ARCUS technology platform, which could bring a differentiated
approach to the treatment of patients with hemoglobinopathies."
“The in vivo gene editing approach that we are pursuing for
sickle cell disease could have a number of significant advantages
over other ex vivo gene therapies currently in development,” said
Derek Jantz, Ph.D., Chief Scientific Officer and Co-Founder of
Precision BioSciences. “Perhaps most importantly, it could open the
door to treating patients in geographies where stem cell transplant
is not a realistic option. We believe that the unique
characteristics of the ARCUS platform, particularly its ability to
target gene insertion with high efficiency, make it the ideal
choice for this project, and we look forward to working with our
partners at Novartis to bring this novel therapy to patients.”
Upon completion of the transaction, Precision 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 Q2 2024.
Precision BioSciences Conference Call and Webcast
Information
Precision's management team will host a conference call and
webcast tomorrow, June 22, 2022, at 8:00 AM ET to discuss the
collaboration. 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 6252688.
Participants may access the live webcast on Precision's website
https://investor.precisionbiosciences.com/events-and-presentations
in the Investors page under Events and Presentations. An archived
replay of the webcast will be available on Precision's website.
About ARCUS and “Safe harbor” ARCUS Nucleases
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 nearly 100 patents to date.
Precision can use an ARCUS nuclease to add a healthy copy of a
gene (or “payload”) to a person’s genome. The healthy copy of the
gene can be inserted at its usual site within the genome, replacing
the mutated, disease-causing copy. Alternatively, an ARCUS nuclease
can be used to insert a healthy copy of the gene at another site
within the genome called a “safe harbor” that enables production of
the healthy gene product without otherwise affecting the patient’s
DNA of gene expression patterns.
About Sickle Cell Disease and Beta Thalassemia
Sickle cell disease (SCD) is a complex genetic disorder that
affects the structure and function of hemoglobin, reduces the
ability of red blood cells to transport oxygen efficiently and,
early on, progresses to a chronic vascular disease.1-4 The disease
can lead to acute episodes of pain known as sickle cell pain
crises, or vaso-occlusive crises, as well as life-threatening
complications.5-7 The condition affects 20 million people
worldwide.8 Approximately 80% of individuals with SCD globally live
in sub-Saharan Africa and it is estimated that approximately 1,000
children in Africa are born with SCD every day and more than half
will die before they reach five.9,10 SCD is also a multisystem
disorder and the most common genetic disease in the United States,
affecting 1 in 500 African Americans. About 1 in 12 African
Americans carry the autosomal recessive mutation, and approximately
300,000 infants are born with sickle cell anemia annually.11 Even
with today’s best available care, SCD continues to drive premature
deaths and disability as this lifelong illness often takes an
extreme emotional, physical, and financial toll on patients and
their families.12,13
Beta thalassemia is also an inherited blood disorder
characterized by reduced levels of functional hemoglobin.14 The
condition has three main forms – minor, intermedia and major, which
indicate the severity of the disease.14 While the symptoms and
severity of beta thalassemia varies greatly from one person to
another, a beta thalassemia major diagnosis is usually made during
the first two years of life and individuals require regular blood
transfusions and lifelong medical care to survive.14 Though the
disorder is relatively rare in the United States, it is one of the
most common autosomal recessive disorders in the world.14 The
incidence of symptomatic cases is estimated to be approximately 1
in 100,000 individuals in the general population.14, 15 The
frequency of beta-thalassemia mutations varies by regions of the
world with the highest prevalence in the Mediterranean, the
Middle-East, and Southeast and Central Asia. Approximately 68,000
children are born with beta-thalassemia.16
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
precise 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 ex vivo
“off-the-shelf” CAR T immunotherapy clinical candidates and several
in vivo gene editing candidates designed 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, as may
any related presentations, within the meaning of the Private
Securities Litigation Reform Act of 1995. All statements contained
in this herein and in any related presentation that do not relate
to matters of historical fact should be considered forward-looking
statements, including, without limitation, statements regarding the
goal of providing a one time, potentially curative treatment for
certain hemoglobinopathies, the success of the collaboration with
Novartis, including the receipt of any milestone, royalty, or other
payments pursuant to and the satisfaction of obligations under the
Agreement, clinical and regulatory development and expected
efficacy and benefit of our platform and product candidates,
expectations about our operational initiatives and business
strategy, expectations about achievement of key milestones, and
expected cash runway. In some cases, you can identify
forward-looking statements by terms such as “aim,” “anticipate,”
“approach,” “believe,” “contemplate,” “could,” “estimate,”
“expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,”
“potential,” “predict,” “project,” “should,” “target,” “will,”
“would,” 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 studies
and clinical trials; public perception about genome editing
technology and its applications; competition in the genome editing,
biopharmaceutical, and 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
COVID-19 pandemic and variants thereof, 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 Quarterly
Report on Form 10-Q for the quarterly period ended March 31, 2022,
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 page of
our website under SEC Filings at investor.precisionbiosciences.com.
References
1 Saraf SL, et al. Paediatr Respir Rev. 2014;15(1):4-12. 2
Stuart MJ, et al. Lancet. 2004;364(9442):1343-1360. 3 National
Institutes of Health (NIH). Sickle cell disease. Bethesda, MD. U.S.
National Library of Medicine. 2018:1-7. 4 Conran N, Franco-Penteado
CF, Costa FF. Hemoglobin. 2009;33(1):1-16. 5 Ballas SK, et al.
Blood. 2012;120(18):3647-3656. 6 Elmariah H, et al. Am J Hematol.
2014(5):530-535. 7 Steinberg M. Management of sickle cell disease.
N Engl J Med. 1999;340(13):1021-1030. 8 National Heart Lung and
Blood Institute: What Is Sickle Cell Disease? 9 Odame I.
Perspective: We need a global solution. Nature. 2014
Nov;515(7526):S10 10 Scott D. Grosse, Isaac Odame, Hani K. Atrash,
et al. Sickle Cell Disease in Africa: A Neglected Cause of Early
Childhood Mortality. American Journal of Preventive Medicine 41,
no. S4 (December 2011): S398-405 11 Sedrak A, Kondamudi NP. Sickle
Cell Disease. [Updated 2021 Nov 7]. In: StatPearls [Internet].
Treasure Island (FL): StatPearls Publishing; 2022 Jan-. 12 Sanger
M, Jordan L, Pruthi S, et al. Cognitive deficits are associated
with unemployment in adults with sickle cell anemia. Journal of
Clinical and Experimental Neuropsychology. 2016;38(6):661-671. 13
Anim M, Osafo J, Yirdong F. Prevalence of psychological symptoms
among adults with sickle cell disease in Korie-Bu Teaching
Hospital, Ghana. BMC Psychology. 2016;4(53):1-9. 14 NORD Rare
Disease Database: Beta Thalassemia 15 Galanello R, Origa R.
Orphanet J Rare Dis. 2010;5:11 16 Needs T, Gonzalez-Mosquera LF,
Lynch DT. Beta Thalassemia. [Updated 2022 May 8]. In: StatPearls
[Internet]. Treasure Island (FL): StatPearls Publishing; 2022
Jan-.
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Investor Contact: Alex Kelly Chief Financial Officer
Alex.Kelly@precisionbiosciences.com
Media Contact: Maurissa Messier Senior Director,
Corporate Communications
Maurissa.Messier@precisionbiosciences.com
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