- Wholly owned programs on track for CTA and/or
IND submissions - PBGENE-HBV for Hepatitis B virus in 2024 and
PBGENE-PMM for primary mitochondrial myopathy in 2025
- First ARCUS in vivo gene editing program now
open for enrollment in the United Kingdom through partnered OTC
deficiency program; United States and Australia expected to
follow
- Exercised option to return three advanced
preclinical programs for development internally or with partners,
including a novel gene editing approach for Duchenne Muscular
Dystrophy
- Monetized CAR T assets through licensing
deals with TG Therapeutics and Caribou Biosciences, and completed
$40 million common stock offering extending expected runway into H2
2026
Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene
editing company utilizing its novel proprietary ARCUS® platform to
develop in vivo gene editing therapies for sophisticated gene
edits, including gene elimination, insertion, and excision,
announced financial results for the first quarter ended March 31,
2024, and provided a business update.
“In 2024, Precision is focused on strong execution as we advance
our wholly owned Hepatitis B virus (HBV) and primary mitochondrial
myopathy (PMM) in vivo gene editing programs towards the clinic as
soon as possible,” said Michael Amoroso, Chief Executive Officer at
Precision BioSciences. “In parallel, our partner, iECURE has
already advanced the first ARCUS in vivo gene editing program for
OTC deficiency into the clinic. We believe this not only validates
ARCUS novel editing approach for gene insertion, but also sets key
regulatory precedence for in vivo ARCUS gene editing programs
across global markets including the US.”
“We were pleased to regain control of three exciting development
opportunities from our work with Prevail Therapeutics, which take
advantage of ARCUS’ unique cut, size, and simplicity. Strong in
vivo proof-of-concept data generated to date have yielded three
advanced preclinical programs that we believe have first-in-class
and best-in-class potential across multiple therapeutic areas. We
are currently assessing which programs Precision will advance
internally and seek to re-partner and expect to provide an update
once plans have been finalized,” added Mr. Amoroso.
“In addition to the important operational progress we continue
to make with our wholly owned and partnered programs, we added cash
to our balance sheet by monetizing our CAR T assets and completing
a $40 million equity offering to extend our cash runway into the
second half of 2026 to fund continued development of our
programs.”
Wholly Owned Portfolio – Lead Programs
PBGENE-HBV (Viral Elimination Program): Precision is
developing PBGENE-HBV for the treatment of patients with chronic
hepatitis B. Currently, it is estimated that approximately 300
million people worldwide are afflicted with chronic Hepatitis B.
PBGENE-HBV is expected to be the first and only potentially
curative gene editing program to enter the clinic that is
specifically designed to eliminate cccDNA and inactivate integrated
HBV DNA.
In February 2024, Precision announced that the company had
received pre-IND regulatory feedback from the US Food and Drug
Administration (FDA) in addition to regulatory feedback from
agencies outside the US providing clarity and alignment on
PBGENE-HBV investigational new drug (IND)/clinical trial
application (CTA)-enabling preclinical plans and clinical strategy.
PBGENE-HBV is advancing through final toxicology studies and
Precision expects to submit an IND and/or CTA for this program in
2024.
PBGENE-PMM (Mutant Mitochondrial DNA Elimination
Program): PBGENE-PMM is a first of its kind potential treatment
for m.3243-associated PMM by targeting mutant mitochondrial DNA.
Mitochondrial diseases are the most common hereditary metabolic
disorder in the world. The m.3243 associated PMM that our program
intends to address is sizable, affecting up to 25,000 people in the
US alone. Precision scientists published new preclinical data in
Nature Metabolism highlighting the high specificity of ARCUS
nucleases designed to edit and eliminate mutant mitochondrial DNA
while allowing wild-type (normal) mitochondrial DNA to repopulate
in the mitochondria, thus improving cellular function. This is a
differentiated program because unlike CRISPR/Cas, base editors, and
prime editors, ARCUS nucleases are able to penetrate the
mitochondrial membranes because they are single-component editors
that do not require a guide RNA. The Company expects to submit an
IND and/or CTA for PBGENE-PMM in 2025 for this program.
Wholly Owned Portfolio – Under Assessment
As previously announced, Precision’s collaboration with Prevail
Therapeutics Inc., has concluded, and Precision exercised its
option to regain rights for the three programs developed under the
collaboration. The Company is in the process of conducting a
portfolio assessment for the newly returned programs for internal
development and/or development through new partners and expects to
provide an update as decisions are finalized. These programs
include:
- PBGENE-DMD – novel gene excision approach for treatment
of Duchenne Muscular Dystrophy utilizing a pair of ARCUS nucleases,
delivered by a single adeno-associated virus (AAV), that are
designed to excise an approximately 500,000 base pair mutation “hot
spot” region from the dystrophin gene to generate a functionally
competent variant of the dystrophin protein.
- PBGENE-LIVER – liver target for gene insertion with data
demonstrating that ARCUS is capable of 40% to 45% high efficiency
gene insertion at 1- and 3-months in nondividing cells, the most
challenging context for gene insertion, in adult nonhuman
primates.
- PBGENE-CNS – gene editing program targeting neurons to
address a disease of the central nervous system.
Partnered Programs – In Vivo Gene
Editing
iECURE-OTC (Gene Insertion Program): Led by iECURE,
ECUR-506 is the first ARCUS-mediated in vivo gene editing program
to advance into the clinic following regulatory approvals in the
US, the United Kingdom, and Australia for initiation of the
OTC-HOPE study. The OTC-HOPE study is a first-in-human Phase 1/2
trial evaluating ECUR-506 as a potential treatment for neonatal
onset ornithine transcarbamylase (OTC) deficiency and has begun
screening patients. iECURE has recently communicated that one site
in the United Kingdom is open and recruiting patients for the
OTC-HOPE study. Non-human primate (NHP) data presented by
researchers from the University of Pennsylvania’s Gene Therapy
Program demonstrated sustained gene insertion of a therapeutic OTC
transgene one-year post-dosing in newborn and infant NHP’s with
high efficiency. iECURE has received Fast Track designation from
the FDA for ECUR-506 and expects initial data from this trial to be
available in late 2024 or in 2025.
PBGENE-NVS (Gene Insertion Program): Precision continues
to advance its gene editing program with Novartis to develop a
custom ARCUS nuclease for patients with hemoglobinopathies, such as
sickle cell disease and beta thalassemia. The collaborative intent
is to insert, in vivo, a therapeutic transgene as a potential
one-time transformative treatment administered directly to the
patient to overcome disparities in patient access to treatment with
other therapeutic technologies, including those that are targeting
an ex vivo gene editing approach.
Business Updates – Monetization of CAR T Investments:
Completed Licensing Deal with TG Therapeutics for Cell
Therapy Azer-Cel in Autoimmune Diseases, and Other Indications
Outside of Cancer:
In January 2024, Precision announced the completion of a
transaction with TG Therapeutics (Nasdaq: TGTX) for certain
exclusive and non-exclusive license rights to develop Azercabtagene
Zapreleucel (azer-cel) for autoimmune diseases and other
indications outside of cancer. In exchange for these rights,
Precision received upfront and potential near-term economics valued
at $17.5 million. Precision is eligible to receive up to $288
million in additional milestone payments based on the achievement
of certain clinical, regulatory, and commercial milestones, in
addition to high-single-digit to low-double-digit royalties on net
sales of the licensed product.
Completed Non-Exclusive Patent License Agreement with Caribou
Biosciences:
In February 2024, Precision announced that it had granted
Caribou Biosciences (Nasdaq: CRBU) a non-exclusive, worldwide
license, with the right to sublicense, to one of Precision’s
foundational cell therapy patent families for use with CRISPR-based
therapies in the field of human therapeutics. The licensed patents
and applications include method and composition of matter claims
that relate to Precision’s approach for targeted insertion of a
sequence encoding an exogenous antigen binding receptor, such as a
CAR, into the T cell receptor alpha constant (TRAC) gene locus of
human T cells via a single gene edit. The licensed family, which
includes more than 20 granted US and international patents, expires
in October 2036.
This patent family is potentially also available for
non-exclusive license to other high-quality partners in the cell
therapy space.
Business Updates – $40 Million Offering:
On March 1, 2024, Precision completed a $40 million public
underwritten offering consisting of 2,500,000 shares of its common
stock and accompanying warrants to purchase up to 2,500,000 shares
of common stock at a combined offering price of $16.00 price per
share, for total gross proceeds of $40 million, before deducting
underwriting discounts and commissions. The financing included
participation from leading life sciences investors, including
Perceptive Advisors, Janus Henderson Investors, Aquilo Capital
Management, LLC and LYFE Capital.
Quarter Ended March 31, 2024 Financial
Results:
Cash and Cash Equivalents: As of March 31, 2024,
Precision had approximately $137.8 million in cash and cash
equivalents. Existing cash and cash equivalents, upfront and
potential near-term cash from CAR T transactions, along with
expected operational receipts, continued fiscal and operating
discipline, availability of Precision’s at-the-market (ATM)
facility, and available credit are expected to extend Precision’s
cash runway into the second half of 2026. The company’s cash runway
is expected to enable funding the development of our PBGENE-HBV and
PBGENE-PMM in vivo gene editing programs through Phase 1 read out
while enabling commencement of a new third wholly owned gene
editing program.
Revenues: Total revenues for the quarter ended March 31,
2024, were $17.6 million, as compared to $8.8 million for the same
period in 2023. The increase of $8.8 million was primarily driven
by upfront investments from cell therapy transactions with TG
Therapeutics and Caribou Biosciences.
Research and Development Expenses: Research and
development expenses were $13.3 million for the quarter ended March
31, 2024, as compared to $11.0 million for the same period in 2023.
The increase of $2.3 million was primarily due to increasing
investment in our wholly owned in vivo gene editing programs,
PBGENE-HBV and PBGENE-PMM, as the programs advance toward IND/CTA
filing. The increases in program spending were partially offset by
lower employee-related costs and lab supplies.
General and Administrative Expenses: General and
administrative expenses were $8.4 million for the quarter ended
March 31, 2024, as compared to $11.1 million for the same period in
2023. The decrease of $2.7 million was primarily related to lower
employee-related costs and a reduction of external operational
expenses.
Net Income/Loss from Continuing Operations: Net income
from continuing operations was $8.6 million for the quarter ended
March 31, 2024, inclusive of a $10.4 million non-cash gain on the
fair value of our warrant liability which does not impact our cash
runway, as compared to a net loss from continuing operations of
$14.0 million, for the same period in 2023. The improvement was
primarily related to the revenue growth compared to the prior
period as well as the non-cash gain related to the fair value
adjustments of our warrant liability and Elo equity investment.
Net Loss: Net income was $8.6 million, or $1.70 per share
(basic and diluted), for the quarter ended March 31, 2024, as
compared to a net loss of $25.1 million, or $(6.75) per share
(basic and diluted), for the same period in 2023. The net loss in
the first quarter of 2023 includes a $11.1 million loss from
discontinued operations related to the decision to exit CAR T in
August 2023.
Shares: Basic weighted-average common shares outstanding
for the first quarter of 2024 were 5,060,978 compared to 3,709,894
for the same period in 2023. Following the common stock offering,
we had 6,916,239 shares outstanding as of March 31, 2024.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is an advanced gene editing company
dedicated to improving life (DTIL) with its novel and proprietary
ARCUS® genome editing platform that differs from other technologies
in the way it cuts, its smaller size, and its simpler structure.
Key capabilities and differentiating characteristics may enable
ARCUS nucleases to drive more intended, defined therapeutic
outcomes. Using ARCUS, the Company’s pipeline is comprised of in
vivo gene editing candidates designed to deliver lasting cures for
the broadest range of genetic and infectious diseases where no
adequate treatments exist. For more information about Precision
BioSciences, please visit www.precisionbiosciences.com.
The ARCUS® platform is being used to develop in vivo gene
editing therapies for sophisticated gene edits, including gene
insertion (inserting DNA into gene to cause expression/add
function), elimination (removing a genome e.g. viral DNA or mutant
mitochondrial DNA), and excision (removing a large portion of a
defective gene by delivering two ARCUS nucleases in a single
AAV).
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 clinical development and expected safety,
efficacy and benefit of our and our partners’ product candidates
and gene editing approaches including editing efficiency; the
suitability of ARCUS nucleases for gene insertion and
differentiation from other gene editing approaches; the expected
timing of regulatory processes (including filings and studies for
PBGENE-HBV and PBGENE-PMM); expectations about our and our
partners’ operational initiatives, strategies, and further
development of our programs; expectations and updates around our
partnerships and collaborations and our ability to enter into new
collaborations, license agreements or other arrangements; our
expected cash runway and available credit; the sufficiency of our
cash runway extending into the second half of 2026; expectations
about achievement of key milestones and receipt of any milestone,
royalty, or other payments; expectations regarding our liquidity
and capital resources; and anticipated timing of clinical data. In
some cases, you can identify forward-looking statements by terms
such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,”
“could,” “designed,” “estimate,” “expect,” “goal,” “intend,”
“look,” “may,” “mission,” “plan,” “possible,” “potential,”
“predict,” “project,” “pursue,” “should,” “strive,” “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. These statements are neither promises nor
guarantees, and involve 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 to advance our programs; risks
associated with our capital requirements, anticipated cash runway,
requirements under our current debt instruments and effects of
restrictions thereunder, including our ability to raise additional
capital due to market conditions and/or our market capitalization;
our operating expenses and our ability to predict what those
expenses will be; our limited operating history; the progression
and 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; the risk that
other genome-editing technologies may provide significant
advantages over our ARCUS technology; our dependence on our ARCUS
technology; the initiation, cost, timing, progress, achievement of
milestones and results of research and development activities and
preclinical and clinical studies, including clinical trial and
investigational new drug applications; public perception about
genome editing technology and its applications; competition in the
genome editing, biopharmaceutical, and biotechnology fields; our or
our collaborators’ or other licensees’ ability to identify, develop
and commercialize product candidates; pending and potential product
liability lawsuits and penalties against us or our collaborators or
other licensees related to our technology and our product
candidates; the US and foreign regulatory landscape applicable to
our and our collaborators’ or other licensees’ development of
product candidates; our or our collaborators’ or other licensees’
ability to advance product candidates into, and successfully
design, implement and complete, clinical trials; potential
manufacturing problems associated with the development or
commercialization of any of our product candidates; delays or
difficulties in our and our collaborators’ and other licensees’
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; our or our
licensees’ ability to obtain orphan drug designation or fast track
designation for our product candidates or to realize the expected
benefits of these designations; our or our collaborators’ or other
licensees’ 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; the
rate and degree of market acceptance of any of our product
candidates; our ability to effectively manage the growth of our
operations; our ability to attract, retain, and motivate executives
and personnel; effects of system failures and security breaches;
insurance expenses and exposure to uninsured liabilities; effects
of tax rules; effects of any pandemic, epidemic, or outbreak of an
infectious disease; the success of our existing collaboration and
other license 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; effects of
natural and manmade disasters, public health emergencies and other
natural catastrophic events; effects of sustained inflation, supply
chain disruptions and major central bank policy actions; market and
economic conditions; risks related to ownership of our common
stock, including fluctuations in our stock price; our ability to
meet the requirements of and maintain listing of our common stock
on Nasdaq or other public stock exchanges; and other important
factors discussed under the caption “Risk Factors” in our Annual
Report on Form 10-K for the year ended December 31, 2023, 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.
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.
Precision Biosciences, Inc. Condensed Statements
of Operations (In thousands, except share and per share
amounts) (unaudited)
For the Three Months Ended March
31,
2024
2023
Revenue
$
17,584
$
8,780
Operating expenses Research and development
13,343
11,048
General and administrative
8,428
11,086
Total operating expenses
21,771
22,134
Operating loss
(4,187
)
(13,354
)
Other income (expense): Gain (loss) from equity method investment
1,713
(1,341
)
Loss on changes in fair value
(348
)
(769
)
Gain on change in fair value of warrant liability
10,386
—
Interest expense
(574
)
(522
)
Interest Income
1,663
2,043
Loss on disposal of assets
(65
)
(7
)
Total other income (expense)
12,775
(596
)
Income (loss) from continuing operations
$
8,588
$
(13,950
)
Loss from discounted operations
—
(11,110
)
Net income (loss)
$
8,588
$
(25,060
)
Net income (loss) per share Basic
$
1.70
$
(6.75
)
Diluted
$
1.70
$
(6.75
)
Weighted-average shares of common stock outstanding Basic
5,060,978
3,709,894
Diluted
5,063,406
3,709,894
Precision Biosciences, Inc. Condensed Balance
Sheets Data (In thousands, except share amounts) (Unaudited)
March 31, 2024 December 31, 2023 Cash and cash
equivalents
$
137,766
$
116,678
Working capital
127,490
86,372
Total assets
184,741
159,781
Total liabilities
147,535
140,920
Total stockholders' equity
$
37,206
$
18,861
Common stock outstanding
6,916,239
4,164,038
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240513276878/en/
Investor and Media Contact: Naresh Tanna Vice President,
Investor Relations Naresh.Tanna@precisionbiosciences.com
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