Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company
committed to delivering a new class of differentiated one-time
curative genetic therapies, today reported financial results and
provided business updates for the third quarter ended September 30,
2023.
“In recent weeks, we announced compelling
preclinical data across our pipeline, including our first in vivo
demonstration of Prime Editing in non-human primates. The
consistency of these data provides proof-of-concept that our
technology can precisely and efficiently edit across a range of
target tissues, correcting pathogenic mutations at levels that may
be sufficient to reverse disease manifestations across severe
genetic conditions,” said Keith Gottesdiener, M.D., President and
Chief Executive Officer of Prime Medicine. “We also shared
presentations detailing the power of our proprietary delivery
systems, as well as new off-target analyses demonstrating the
potentially best-in-class safety profile of Prime Editors.
Together, these encouraging results mark meaningful progress toward
the clinic and support our strategy of simultaneously advancing
multiple programs forward. We look forward to leveraging the
modularity of our platform, as well as our many clinical,
manufacturing and regulatory learnings, to inform further
development of our pipeline with the goal of delivering one-time,
curative therapies to transform the lives of patients with a wide
spectrum of debilitating diseases.”
Recent Business Updates
Prime Medicine is advancing a strategic pipeline
of eighteen programs. The Company is initially focused on
indications with the opportunity for the fastest, most direct path
to the clinic and technical success in humans, as well as
indications that cannot be treated using other gene editing
approaches.
Chronic Granulomatous Disease (CGD)
- In August 2023, Prime Medicine
received Rare Pediatric Drug designation (RPDD) from the U.S. Food
and Drug Administration (FDA) for PM359. The FDA grants RPDD to
medicines intended for the treatment of serious and
life-threatening diseases that primarily affect children ages 18
years or younger and fewer than 200,000 people in the United
States. Companies that receive approval for a New Drug Application
or Biologics License Application for a rare pediatric disease may
be eligible to receive a voucher for priority review of a
subsequent marketing application for a different product. The
priority review voucher may be used by the Company or sold to a
third party.
Glycogen Storage Disease 1b (GSD1b)
- In October 2023, Prime Medicine
presented new in vivo data at the European Society of Gene and Cell
Therapy 2023 Congress, demonstrating the ability of Prime Editors
to precisely correct one of the most prevalent disease-causing
mutations of GSD1b in both non-human primate (NHP) and mouse
models. In these preclinical studies using single lipid
nanoparticle (LNP) administration, Prime Editors showed up to 50%
whole liver precise editing in NHPs (resulting in up to 83% of the
key target cells, liver hepatocytes, edited), with no observed
safety concerns. In addition, no detectable off-target effects were
observed following a comprehensive in vitro off-target screening
analysis. These data, the first evidence of Prime Editing in NHPs,
provide proof-of-concept for Prime Medicine’s LNP liver-targeted
delivery approach and support the further advancement of the
Company’s Prime Editors for liver-targeted programs. Read the full
data here.
Retinitis Pigmentosa / Rhodopsin
- In October 2023, Prime Medicine
presented new in vivo data at the International Symposium on
Retinal Degeneration, demonstrating that Prime Editors can
efficiently and precisely correct the predominant mutations that
cause rhodopsin associated autosomal dominant retinitis pigmentosa
(RHO adRP). In preclinical studies, single Prime Editors showed up
to 70% precise correction in photoreceptors, as well as favorable
tolerability. These data suggest that Prime Medicine’s proprietary
dual adeno-associated virus platform can effectively deliver Prime
Editors to the eye, with the potential to treat a range of retinal
diseases. Read the full data here.
Additional Pipeline and Prime Editing Platform
Updates
- Prime Medicine presented additional
preclinical data across its Prime Editing pipeline and technology
at several healthcare industry conferences in October and has plans
to share additional abstracts at medical meetings before year-end.
Highlights of the conference presentations included:
- An overview of Prime Medicine’s
comprehensive suite of assays to identify potential off-target
events, which has been expanded to include new, unbiased
genome-wide tools. These analyses continue to demonstrate minimal
to no detectable off-target edits, chromosomal rearrangements or
translocations. These preliminary analyses, across multiple editing
programs, suggest potentially best-in-class safety;
- The first presentation on Prime
Medicine’s universal LNP-RNA platform designed to deliver Prime
Editors to correct pathogenic mutations in the liver;
- An update on using Prime Editors
for the potential treatment of repeat expansion diseases, including
an update on Fragile X syndrome; and
- Initial proof-of-concept data for
Prime Medicine’s hotspot approach to developing Prime Editors to
correct the CFTR gene in patients with Cystic Fibrosis, as well as
new detail on Prime Medicine’s LNP and all-RNA delivery approaches
for the lung.
- In December, Prime Medicine will
present preclinical data showcasing the potential of the PASSIGE
platform to multiplex edit CAR-T cells at the 65th American Society
of Hematology (ASH) Annual Meeting, being held December 9-12, 2023,
in San Diego, CA. Details of the poster presentation are as
follows:
Abstract
Title: Multiplex Prime Editing and PASSIGE for Non-Viral
Generation of an Allogeneic CAR-T Cell ProductDate &
Time: Monday, December 11, 6-8 PM PTRoom:
San Diego Convention Center, Halls G-HSession
Title: Cellular Immunotherapies: Basic and
TranslationalPresenter: Emily Pomeroy
Corporate
- In October 2023, the U.S. Patent
and Trademark Office issued Prime Medicine’s third patent, No.
11,795,452, “Methods and Compositions for Editing Nucleotide
Sequences,” which covers Prime Editing systems that include a
PEgRNA, Prime Editor protein, and, optionally, a recombinase.
Anticipated Upcoming
Milestones
Year-to-date, Prime Medicine continues to make
significant progress, executing against key initiatives to drive
its Prime Editing platform forward. The Company initiated
IND-enabling studies for PM359 in CGD, and expanded preclinical
proof-of-concept data in vivo and in vitro across several programs
and target tissue types. In addition, as evidenced by in vivo data
shared in recent presentations, Prime Medicine continues to
optimize its non-viral and viral delivery systems, and to
demonstrate excellent off-target profiles for its Prime Editing
programs.
Prime Medicine expects the following activities
and next steps to drive the Prime Editing platform forward in the
coming months:
Pipeline
- Complete first IND filing as early
as 2024 with additional IND filings anticipated in 2025.
Platform
- Expand Prime Editing using
proprietary recombinase technologies for new and existing
programs.
- Maximize Prime Editing’s broad
therapeutic potential and create value through strategic business
development that extends the reach and impact of Prime Editing to
areas beyond Prime Medicine’s current areas of focus.
Third Quarter 2023 Financial
Results:
- R&D Expenses:
Research and development (R&D) expenses were $41.0 million for
the three months ended September 30, 2023, as compared to $25.0
million for the three months ended September 30, 2022. This
increase was primarily due to increases in lab supplies, personnel,
and facilities costs as the Company continues to expand and build
out its R&D activities and function.
- G&A Expenses:
General and administrative (G&A) expenses were $10.5 million
for the three months ended September 30, 2023, as compared to $6.6
million for the three months ended September 30, 2022. This
increase was primarily due to an increase in professional and
consultant costs and personnel costs primarily attributable to the
build-out of the Company’s G&A team to support its R&D
function.
- Net Loss: Net loss
was $50.7 million for the three months ended September 30, 2023, as
compared to $29.4 million for the three months ended September 30,
2022.
- Cash Position: As
of September 30, 2023, cash, cash equivalents, investments and
restricted cash were $178.8 million, as compared to $307.4 million
as of December 31, 2022.
Financial Guidance
Based on its current operating plans, Prime
Medicine expects that its cash, cash equivalents and investments as
of September 30, 2023, will be sufficient to fund its
operating expenses and capital expenditure requirements through the
end of 2024.
About Prime MedicinePrime
Medicine is a leading biotechnology company dedicated to creating
and delivering the next generation of gene editing therapies to
patients. The Company is leveraging its proprietary Prime Editing
platform, a versatile, precise and efficient gene editing
technology, to develop a new class of differentiated, one-time,
potentially curative genetic therapies. Designed to make only the
right edit at the right position within a gene while minimizing
unwanted DNA modifications, Prime Editors have the potential to
repair almost all types of genetic mutations and work in many
different tissues, organs and cell types.
Prime Medicine is currently progressing a
diversified portfolio of eighteen programs initially focused on
genetic diseases with a fast, direct path to treating patients or
with a high unmet need because they cannot be treated using other
gene-editing approaches. Over time, the Company intends to maximize
Prime Editing’s therapeutic potential and advance potentially
curative therapeutic options to patients for a broad spectrum of
diseases. For more information, please visit
www.primemedicine.com.
Cautionary Note Regarding Forward
Looking StatementsThis press release contains
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995, as amended, including,
without limitation, implied and express statements about Prime
Medicine’s beliefs and expectations regarding: the initiation,
timing, progress, and results of its research and development
programs, preclinical studies and future clinical trials, and the
release of data related thereto, including its ability to expand
preclinical proof-of-concept in vivo data; its ability to maintain
Pediatric Drug designation for PM359 and the potential to use a
priority review voucher or sell it to a third party; the timing of
its regulatory filings, including its anticipated initial IND
application submission for CGD as early as 2024 with additional
filings anticipated in 2025; the potential of Prime Editors to
reproducibly correct disease-causing genetic mutations across
different tissues, organs and cell types, and the capacity of its
PASSIGE technology to edit CAR-T cells for the treatment of certain
cancers and immune diseases; its continued development and
optimization of various non-viral and viral delivery systems; its
ability to demonstrate superior off-target profiles for Prime
Editing programs; its expansion of Prime Editing using proprietary
recombinase and/or retrotransposon and other proprietary
technologies; the expansion of Prime Editing’s therapeutic
potential and the creation of value through strategic business
development to extend the reach and impact of Prime Editing to
areas beyond Prime Medicine’s current areas of focus; its
expectations regarding the breadth of Prime Editing technology and
the implementation of its strategic plans for its business,
programs, and technology; the potential of Prime Editing to offer
curative genetic therapies for a wide spectrum of diseases; and its
estimates of expenses, capital requirements, and needs for
additional financing and its expectations regarding the ability to
fund its anticipated operating expenses and capital expenditure
requirements through the end of 2024. The words “may,” “might,”
“will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,”
“intend,” “believe,” “expect,” “estimate,” “seek,” “predict,”
“future,” “project,” “potential,” “continue,” “target” and similar
words or expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words.
Any forward-looking statements in this press
release are based on management’s current expectations and beliefs
and are subject to a number of risks, uncertainties and important
factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including, without
limitation, risks associated with: uncertainties related to the
authorization, initiation, and conduct of preclinical and
IND-enabling studies and other development requirements for
potential product candidates, including uncertainties related to
opening INDs and obtaining regulatory approvals; risks related to
the development and optimization of new technologies, the results
of preclinical studies, or clinical studies not being predictive of
future results in connection with future studies; the scope of
protection Prime Medicine is able to establish and maintain for
intellectual property rights covering its Prime Editing technology;
Prime Medicine’s ability to identify and enter into future license
agreements and collaborations; and general economic, industry and
market conditions, including rising interest rates, inflation, and
adverse developments affecting the financial services industry.
These and other risks and uncertainties are described in greater
detail in the section entitled “Risk Factors” in Prime Medicine’s
most recent Annual Report on Form 10-K, as well as any subsequent
filings with the Securities and Exchange Commission. In addition,
any forward-looking statements represent Prime Medicine’s views
only as of today and should not be relied upon as representing its
views as of any subsequent date. Prime Medicine explicitly
disclaims any obligation to update any forward-looking statements
subject to any obligations under applicable law. No representations
or warranties (expressed or implied) are made about the accuracy of
any such forward-looking statements.
© 2023 Prime Medicine, Inc. All rights reserved.
PRIME MEDICINE, the Prime Medicine logos, and PASSIGE are
trademarks of Prime Medicine, Inc. All other trademarks referred to
herein are the property of their respective owners.
Investor ContactHannah DeresiewiczStern
Investor Relations,
Inc.212-362-1200hannah.deresiewicz@sternir.com
Media ContactDan Budwick,
1ABdan@1ABmedia.com
Condensed Consolidated Balance Sheet Data |
(unaudited) |
|
|
|
|
|
|
|
|
|
(in thousands) |
|
|
September 30,2023 |
|
|
|
December 31,2022 |
|
Cash, cash equivalents, and
investments |
|
|
165,317 |
|
|
|
293,921 |
|
Total assets |
|
|
239,145 |
|
|
|
360,314 |
|
Total liabilities |
|
|
45,188 |
|
|
|
44,044 |
|
Total stockholders’
equity |
|
|
193,957 |
|
|
|
316,270 |
|
Condensed Consolidated Statement of
Operations |
(unaudited) |
|
|
|
|
|
|
|
Three Months EndedSeptember
30, |
(in thousands, except share and per share amounts) |
|
|
2023 |
|
|
|
2022 |
|
Operating expenses: |
|
|
|
|
Research and development |
|
$ |
40,967 |
|
|
$ |
25,047 |
|
General and administrative |
|
|
10,492 |
|
|
|
6,608 |
|
Total operating expenses |
|
|
51,459 |
|
|
|
31,655 |
|
Loss from operations |
|
|
(51,459 |
) |
|
|
(31,655 |
) |
Other income (expense): |
|
|
|
|
Change in fair value of short-term investment — related party |
|
|
(1,579 |
) |
|
|
1,789 |
|
Other income, net |
|
|
2,222 |
|
|
|
728 |
|
Total other income (expense), net |
|
|
643 |
|
|
|
2,517 |
|
Net loss before income taxes |
|
|
(50,816 |
) |
|
|
(29,138 |
) |
(Provision for) benefit from income taxes |
|
|
108 |
|
|
|
(212 |
) |
Net loss |
|
$ |
(50,708 |
) |
|
$ |
(29,350 |
) |
Cumulative dividend on preferred stock |
|
|
— |
|
|
|
(6,362 |
) |
Net loss attributable to common stockholders |
|
$ |
(50,708 |
) |
|
$ |
(35,712 |
) |
Net loss per share attributable to common stockholders, basic and
diluted |
|
$ |
(0.55 |
) |
|
$ |
(1.61 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
|
91,846,835 |
|
|
|
22,226,301 |
|
Prime Medicine (NASDAQ:PRME)
過去 株価チャート
から 4 2024 まで 5 2024
Prime Medicine (NASDAQ:PRME)
過去 株価チャート
から 5 2023 まで 5 2024