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 second quarter ended June 30,
2023.
“In recent months, we continued to advance our diversified
portfolio of Prime Editing programs while also executing against a
strategic partnering strategy aimed at further expanding the broad
therapeutic potential of Prime Editing,” said Keith Gottesdiener,
M.D., President and Chief Executive Officer of Prime Medicine.
“PM359, our product candidate for the treatment of CGD, is
progressing well, and at the ASGCT Annual Meeting in May, we
presented new preclinical data demonstrating its ability to
reproducibly correct the CGD disease-causing mutation in CD34+
cells ex vivo with no detectable off-target editing. These findings
further support our belief in the potential of this program to
change the trajectory of this recurrent debilitating condition, and
we look forward to sharing additional in vitro and in vivo data on
this program and others later this year.”
Dr. Gottesdiener continued, “Also in the second quarter, we
entered into a research collaboration with Cimeio Therapeutics,
gaining access to Cimeio’s powerful CD117 immunotherapy technology
for genetic diseases. We are pleased to be working together to
evaluate combining Prime Editing enabled protective shielding with
multiplexed therapeutic edits to potentially reduce the toxicity
and increase the efficiency of existing HSC transplant regimens.
These combined technologies may enable us to more gently and
effectively treat a wider range of genetic diseases than currently
possible, and future applications may include selection of in vivo
edited HSCs, which could allow for the treatment of genetic
diseases without transplantation. This partnership reflects the
tremendous breadth of our Prime Editing technology, as well as our
commitment to leveraging its versatility, precision and efficiency
to improve the care and treatment of patients worldwide.”
Recent Business Updates
Pipeline
- Prime Medicine continued to advance its strategic pipeline of
eighteen programs and remained on track to initiate investigational
new drug (IND)-enabling studies of PM359, its development candidate
for chronic granulomatous disease (CGD), in 2023.
- In May 2023, Prime Medicine presented new preclinical data at
the American Society of Gene and Cell Therapy (ASGCT) 26th Annual
Meeting. These data further demonstrated the potential for Prime
Editing to correct the causative mutation of CGD and showcased the
potential application of the Prime
Assisted
Site-Specific
Integrase Gene
Editing (PASSIGE™) platform to generate
multiplex-edited CAR-T cells for the treatment of certain cancers
and immune diseases. Read the full data here.
Corporate
- In June 2023, Prime Medicine announced a research collaboration
with Cimeio Therapeutics to develop Prime Edited Shielded Cell and
Immunotherapy Pairs™ (SCIP) for genetic diseases, acute myeloid
leukemia (AML) and myelodysplastic syndrome (MDS). The goal of the
research is to reduce the toxicity of conditioning regimens and
introduce new therapeutic options to meaningfully expand the
utility of hematopoietic stem cell (HSC) transplant and enable the
in vivo selection of edited HSCs to potentially remove the need for
transplantation entirely. Under the terms of the agreement, Prime
Medicine will develop a Prime Editor for Cimeio’s CD117 shielding
variant that will then be evaluated by both companies; if the
research collaboration is successful, the companies will grant
exclusive license options to each other for their technologies. If
the companies exercise their exclusive license options, they will
each be eligible to receive economics on net sales of licensed
products.
Anticipated Upcoming Milestones
Prime Medicine expects the following activities and next steps
to drive the Prime Editing platform forward:
Pipeline
- Initiate investigational new drug (IND)-enabling studies for
PM359 in CGD in 2023.
- Expand preclinical proof-of-concept in vivo data, with plans to
share data from in vivo rodent studies and large animal studies
from several programs in the second half of 2023.
- Share in vitro preclinical data in additional liver, eye and
neuromuscular programs.
- Complete first IND filing as early as 2024 and additional IND
filings anticipated in 2025.
Platform
- Continue to develop and optimize non-viral and viral delivery
systems and share additional proof-of-concept data from in vivo
rodent and large animal studies in the second half of 2023.
- Further demonstrate superior off-target profiles for Prime
Editing programs.
- 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.
Second Quarter 2023 Financial Results:
- R&D Expenses: Research and development
(R&D) expenses were $34.6 million for the three months ended
June 30, 2023, as compared to $18.9 million for the three months
ended June 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 $10.7 million for the three months ended
June 30, 2023, as compared to $7.4 million for the three months
ended June 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 our R&D function.
- Net Loss: Net loss was $42.4 million for the
three months ended June 30, 2023, as compared to $29.3 million for
the three months ended June 30, 2022.
- Cash Position: As of June 30, 2023, cash, cash
equivalents, investments and restricted cash were $221.1 million,
as compared to $263.0 million as of March 31,
2023.
Financial Guidance
Based on its current operating plans, Prime Medicine expects
that its cash, cash equivalents and investments as of June 30,
2023, will be sufficient to fund its anticipated operating expenses
and capital expenditure requirements into 2025.
About Prime Medicine
Prime 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
Statements
This 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
the initiation of IND-enabling studies for PM359 in 2023, its
ability to expand preclinical proof-of-concept in vivo data and
plans to share data from several programs in the second half of
2023, the timing of its regulatory filings, including its
anticipated initial IND application submission as early as 2024
with additional filings anticipated in 2025, and plans to share
preclinical in vitro data in additional programs; the potential of
PM359 to reproducibly correct the causative mutation of CGD, and
the capacity of its PASSIGE technology to edit CAR-T cells for the
treatment of certain cancers and immune diseases; its 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; the
research collaboration to combine Prime Medicine and Cimeio’s
respective technologies, including Prime Medicine’s Prime Editing
platform and Cimeio’s SCIP platform, and the goals of such
collaboration; the potential benefits of such collaboration and
technology thereunder, including the ability to cure various
diseases and replace existing treatments such as transplantation;
and the exercise of the exclusive options and payment of economics;
the implementation of its strategic plans for its business,
programs, and technology; 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 into 2025.
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 Quarterly Report on Form 10-Q, 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.
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) |
|
June 30, 2023 |
|
December 31, 2022 |
Cash, cash equivalents, and investments |
|
$ |
207,618 |
|
$ |
293,921 |
Total assets |
|
|
280,865 |
|
|
360,314 |
Total liabilities |
|
|
40,763 |
|
|
44,044 |
Total stockholders’
equity |
|
|
240,102 |
|
|
316,270 |
|
|
|
|
|
|
|
Condensed Consolidated Statement of
Operations (unaudited)
|
|
Three Months Ended June 30, |
(in thousands, except share and per share
amounts) |
|
2023 |
|
2022 |
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
$ |
34,599 |
|
|
$ |
18,940 |
|
General and administrative |
|
|
10,658 |
|
|
|
7,365 |
|
Total operating expenses |
|
|
45,257 |
|
|
|
26,305 |
|
Loss from operations |
|
|
(45,257 |
) |
|
|
(26,305 |
) |
Other income (expense): |
|
|
|
|
|
|
Change in fair value of short-term investment — related party |
|
|
263 |
|
|
|
(3,723 |
) |
Other income, net |
|
|
2,640 |
|
|
|
238 |
|
Total other income (expense), net |
|
|
2,903 |
|
|
|
(3,485 |
) |
Net loss before income
taxes |
|
|
(42,354 |
) |
|
|
(29,790 |
) |
(Provision for) benefit from
income taxes |
|
|
(31 |
) |
|
|
442 |
|
Net loss |
|
$ |
(42,385 |
) |
|
$ |
(29,348 |
) |
Cumulative dividend on preferred stock |
|
|
- |
|
|
|
(6,293 |
) |
Net loss attributable to
common stockholders |
|
$ |
(42,385 |
) |
|
$ |
(35,641 |
) |
Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(0.47 |
) |
|
$ |
(1.76 |
) |
Weighted-average common shares
outstanding, basic and diluted |
|
|
90,467,298 |
|
|
|
20,227,343 |
|
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