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 first quarter ended March 31,
2023.
“The first quarter and recent months were marked by a number of
important milestones across our business as we execute against our
mission of delivering a new class of one-time, potentially curative
genetic therapies,” said Keith Gottesdiener, M.D., President and
Chief Executive Officer of Prime Medicine. “We are particularly
encouraged by recent progress across our diversified pipeline.
Earlier this year, we nominated our first development candidate,
PM359, and, at the American Society of Gene and Cell Therapy
(ASGCT) Annual Meeting in May, we will showcase new preclinical
data further supporting the potential of PM359 to correct the
causative mutation of chronic granulomatous disease (CGD). Also at
ASGCT, we will share new data demonstrating the power of our
PASSIGE™ technology to generate CAR-T cells, potentially unlocking
the opportunity for Prime Editing to address a number of cancers
and immune diseases. Together, these presentations underscore our
belief that Prime Editing is unrivaled in its breadth and potential
to offer curative treatments for many diseases.”
Dr. Gottesdiener continued, “Additionally, we recently
strengthened our corporate position with additions to our
intellectual property portfolio. The recent patent issuance and
patent application allowance mark a significant step toward our
goal of securing the broadest intellectual property protection for
our platform technology. We are also pleased to announce the
appointment of Jeff Marrazzo to our Board of Directors. With his
experience as co-founder and former Chief Executive Officer of a
commercial gene therapy company, Jeff brings expertise and a unique
perspective that we believe will complement the skills of our
leadership team. We look forward to partnering with him as we
pursue our shared vision of pushing the frontier of genetic
medicine to deliver better outcomes to patients.”
Recent Business Updates
Pipeline
- Today, Prime Medicine announced that it will present
preclinical data from its Prime Editing platform at the ASGCT 26th
Annual Meeting, being held May 16-20, 2023 in Los Angeles. In an
oral presentation, Prime Medicine will highlight new findings
further demonstrating the potential of Prime Editing to correct the
causative mutation of CGD and, in a poster presentation, will
showcase the potential of the PASSIGE platform to multiplex edit
CAR-T cells for the treatment of certain cancers and immune
diseases. Details of the presentations are as follows:
Abstract Title: (101) Prime Editing of Human CD34+
Long-Term Hematopoietic Stem Cells Precisely Corrects the Causative
Mutation of p47phox Chronic Granulomatous Disease and Restores
NADPH Oxidase Activity in Myeloid ProgenyDate &
Time: Wednesday, May 17, 2023, 5:15 – 5:30 p.m.
PTRoom: Room 515 ABSession Title:
Genome Editing Therapies & Safety IPresenter:
Jennifer GoriAbstract Title: (602) An All-Prime
Editing One-Step Approach for Non-Viral Generation of a
Multiplex-Edited Allogeneic CAR-T Cell ProductDate &
Time: Wednesday, May 17, 2023, 12:00 p.m.
PTSession Title: Wednesday Poster
SessionPresenter: Emily Pomeroy
Corporate
- In May 2023, Prime Medicine appointed Jeff Marrazzo, co-founder
and former Chief Executive Officer of Spark Therapeutics, Inc., to
its Board of Directors. Jeff founded and built Spark Therapeutics
from an idea incubated within the Children’s Hospital of
Philadelphia (CHOP) into the world’s first fully integrated,
commercial gene therapy company with over 850 employees at the time
of his departure. Under Jeff’s leadership, Spark developed and
launched LUXTURNA® for a rare blinding disorder, the first U.S.
Food and Drug Administration-approved gene therapy for a genetic
disease in the U.S., spearheading the creation of novel
reimbursement models to ensure patient access to genetic medicines.
While at Spark, Jeff shepherded multiple gene therapies into the
clinic for patients with conditions ranging from inherited retinal
diseases to rare bleeding and neuromuscular disorders, raising $1
billion in capital and establishing major partnerships with Pfizer
and Novartis. Jeff’s orchestration of Spark’s $4.8 billion sale to
Roche in 2019 marked a 100-fold increase in the company’s market
value over six years.
- In May 2023, the U.S. Patent and Trademark Office (PTO) issued
Prime Medicine’s second patent, No. 11,643,652, which covers
composition of matter for Prime Editor guide RNAs (PEgRNAs). In
April 2023, Prime Medicine received a notice of allowance from the
U.S. PTO for its third patent application, No. 17/751,599, which
covers Prime Editing systems that include a PEgRNA, Prime Editor
protein, and, optionally, a recombinase.
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 and/or
retrotransposon 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.
First Quarter 2023 Financial Results:
- R&D Expenses: Research and development
(R&D) expenses were $30.9 million for the three months ended
March 31, 2023, as compared to $13.7 million for the three months
ended March 31, 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 $9.2 million for the three months ended
March 31, 2023, as compared to $6.2 million for the three months
ended March 31, 2022. This increase was primarily due to an
increase in facility costs and professional costs primarily
attributable to the build-out of the company’s G&A team to
support business growth.
- Net Loss: Net loss was $39.4 million for the
three months ended March 31, 2023, as compared to $23.8 million for
the three months ended March 31, 2022.
- Cash Position: As of March 31, 2023, cash,
cash equivalents, investments and restricted cash were $263.0
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 March 31,
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, the potential of
PM359 to 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 ability to advance its
portfolio toward additional preclinical proof-of-concept readouts;
its ability to demonstrate, and the timing of, preclinical
proof-of-concept in vivo for multiple programs; 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 and potential to offer curative
treatments for disease-causing genetic mutations; the timing of its
regulatory filings, including its anticipated initial IND
submission as early as 2024 with additional filings anticipated in
2025; 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 other development requirements for
potential product candidates, including uncertainties related to
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.
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) |
|
March 31, 2023 |
|
December 31, 2022 |
Cash, cash equivalents and investments |
|
$ |
249,518 |
|
|
$ |
293,921 |
|
Total assets |
|
|
316,946 |
|
|
|
360,314 |
|
Total liabilities |
|
|
38,145 |
|
|
|
44,044 |
|
Total stockholders’
equity |
|
|
278,801 |
|
|
|
316,270 |
|
Condensed Consolidated Statement of
Operations(unaudited) |
|
|
|
|
|
Three Months Ended March 31, |
(in thousands, except share and per share
amounts) |
|
|
2023 |
|
|
|
2022 |
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
$ |
30,880 |
|
|
$ |
13,677 |
|
General and administrative |
|
|
9,153 |
|
|
|
6,221 |
|
Total operating expenses |
|
|
40,033 |
|
|
|
19,898 |
|
Loss from operations |
|
|
(40,033 |
) |
|
|
(19,898 |
) |
Other income (expense): |
|
|
|
|
|
|
Change in fair value of short-term investment — related party |
|
|
(1,701 |
) |
|
|
(4,485 |
) |
Other income, net |
|
|
2,135 |
|
|
|
11 |
|
Total other income (expense), net |
|
|
434 |
|
|
|
(4,474 |
) |
Net loss before income
taxes |
|
|
(39,599 |
) |
|
|
(24,372 |
) |
Benefit from income taxes |
|
|
(202 |
) |
|
|
(532 |
) |
Net loss |
|
$ |
(39,397 |
) |
|
$ |
(23,840 |
) |
Cumulative dividend on preferred stock |
|
|
- |
|
|
|
(6,224 |
) |
Net loss attributable to
common stockholders |
|
$ |
(39,397 |
) |
|
$ |
(30,064 |
) |
Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(0.44 |
) |
|
$ |
(1.58 |
) |
Weighted-average common shares
outstanding, basic and diluted |
|
|
89,064,895 |
|
|
|
19,050,385 |
|
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