Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a biopharmaceutical company
focused on developing a new class of genetic medicines for both
rare and highly prevalent diseases, today reported financial
results for the full year ended December 31, 2023, and reiterated
its recent progress and anticipated milestones.
“Last year was a remarkable year for us as we
achieved multiple major milestones, including selecting KRRO-110 as
a development candidate, a potentially best-in-class therapeutic
for AATD, becoming a public company, and closing $117 million in
financing,” said Dr. Ram Aiyar, CEO and President of Korro. “2024
is off to a strong start and is poised to be a transformative year
for us. We expect multiple milestones throughout the year,
including a regulatory submission for KRRO-110 in the second half
of 2024, further progression of our pipeline programs, and
expansion of platform innovation. Our dedication to providing
potential disease-modifying therapies for patients in need, coupled
with our conviction in the pivotal role of RNA editing in the
development of these therapeutic options, uniquely positions us to
make a meaningful impact on patients’ lives.”
Pipeline and Business
Updates:
- Nominated KRRO-110 as first
development candidate for the potential treatment of AATD.
KRRO-110 is the first RNA editing oligonucleotide product candidate
from Korro’s platform called Oligonucleotide Promoted Editing of
RNA (OPERA™). KRRO-110 is designed to co-opt an endogenous enzyme,
Adenosine Deaminase Acting on RNA (ADAR), to edit the “A” variant
on SERPINA1 RNA, repair an amino acid codon, and restore secretion
of normal alpha-1 antitrypsin (AAT) protein. This repair of the
endogenous protein has the potential to clear protein aggregates
from within liver cells to create a potentially clinically
differentiated benefit for liver function and to preserve lung
function by providing an adequate amount of normal AAT
protein.
- Reported preclinical data
for KRRO-110 that supports continued development
as a potentially best-in-class therapeutic for AATD
patients. Preclinical data demonstrated high specificity
with no detectable bystander edits in human (MZ) primary
hepatocytes and secretion of ~50µM functional AAT protein after a
single dose in a preclinical human transgenic PiZ mouse model with
durability in mice based on an increase in AAT protein with the
inhibition of elastase activity.
- Advanced OPERA platform to
generate differentiated Customized High-Fidelity Oligonucleotides
for RNA Deamination, or CHORDs™. Aided by
proprietary algorithms, Korro developed CHORDs with enhanced in
vivo potency and durability. Using CHORDs, Korro achieved greater
than 50% editing using GalNAc conjugates with subcutaneous
administration to the liver in a preclinical mouse model.
- Demonstrated translation of
RNA editing efficiency from mice to NHPs in multiple
targets. For its AATD program, with a single dose, Korro
has shown greater than 40% editing in NHPs utilizing a surrogate
oligonucleotide designed to edit and create a de novo AAT protein
variant in NHPs in a transient manner. Additionally, Korro
demonstrated the creation of a de novo protein variant of an
undisclosed transcription factor, leading to sustained downstream
activity in NHPs lasting longer than 21 days, demonstrating the
potential to increase the half-life of the transcription factor and
enhance its function.
- Strong balance sheet with
cash runway into 2026. Cash and cash equivalents of
approximately $166.1 million are expected to fund operations and
multiple potentially value-creating milestones into 2026, including
advancement of KRRO-110 through a potential interim clinical
readout, progression of additional product candidates through the
Company’s pipeline, and demonstration of OPERA applicability across
additional tissue types.
- Expanded Board of Directors
with two independent director appointments. Added Dr.
Rachel Meyers, an industry veteran with decades of experience
developing RNA therapeutics while at Alnylam, and Tim Pearson, an
industry veteran and financial expert operating large and small
biotechs, both of whom bring unique perspectives and deep expertise
to Korro’s Board.
Upcoming Milestones:
- Regulatory filing for KRRO-110 in
AATD on track for the second half of 2024.
- Anticipated interim clinical
readout for KRRO-110 in AATD in the second half of 2025.
Full Year 2023 Financial
Results:
Cash Position: Cash, cash equivalents and
short-term investments were $166.1 million as of December 31, 2023,
compared to $55.2 million as of December 31, 2022. Korro expects
that its cash, cash equivalents and short-term investments to fund
operations into 2026.
Research and Development (R&D) Expenses:
R&D expenses were $57.2 million for the year ended December 31,
2023, as compared to $42.2 million for the year ended December 31,
2022. The increase in R&D expenses was driven primarily by
preclinical activities for the KRRO-110 AATD program and expenses
related to the advancement of its pipeline and platform.
General and Administration (G&A) Expenses:
G&A expenses were $27.3 million for the year ended December 31,
2023, as compared to $16.8 million for the year ended December 31,
2022. The increase is driven primarily by merger-related personnel
costs, as well as additional personnel and facility costs to
support growing R&D function.
Net Loss: Korro’s net loss was $81.2 million for
the year ended December 31, 2023, as compared to $58.0 million for
the year ended December 31, 2022.
About Korro
Korro is a biopharmaceutical company focused on
developing a new class of genetic medicines for both rare and
highly prevalent diseases using its proprietary RNA editing
platform. Korro is generating a portfolio of differentiated
programs that are designed to harness the body’s natural RNA
editing process to effect a precise yet transient single base edit.
By editing RNA instead of DNA, Korro is expanding the reach of
genetic medicines by delivering additional precision and
tunability, which has the potential for increased specificity and
improved long-term tolerability. Using an oligonucleotide-based
approach, Korro expects to bring its medicines to patients by
leveraging its proprietary platform with precedented delivery
modalities, manufacturing know-how, and established regulatory
pathways of approved oligonucleotide drugs. Korro is based in
Cambridge, Mass. For more information, visit
korrobio.com.
Forward-Looking Statements
Certain statements in this press release may
constitute “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995, as amended.
Forward-looking statements include, but are not limited to, express
or implied statements regarding expectations, hopes, beliefs,
intentions or strategies of Korro regarding the future including,
without limitation, express or implied statements regarding:
Korro’s planned regulatory filing for KRRO-110 in AATD and any
interim data readout; Korro’s cash runway; Korro’s ability to
advance its pipeline and the role of RNA editing technology in
developing therapeutic options; KRRO-110’s potential as a
best-in-class drug candidate for AATD; and the benefits of OPERA;
among others. In addition, any statements that refer to
projections, forecasts, or other characterizations of future events
or circumstances, including any underlying assumptions, are
forward-looking statements. The words “anticipate,” “believe,”
“continue,” “could,” “estimate,” “expect,” “intend,” “may,”
“might,” “plan,” “possible,” “potential,” “predict,” “project,”
“should,” “strive,” “would,” “aim,” “target,” “commit,” and similar
expressions may identify forward-looking statements, but the
absence of these words does not mean that statement is not forward
looking. Forward-looking statements are based on current
expectations and assumptions that, while considered reasonable are
inherently uncertain. New risks and uncertainties may emerge from
time to time, and it is not possible to predict all risks and
uncertainties. Factors that may cause actual results to differ
materially from current expectations include, but are not limited
to, various factors beyond management’s control including risks
inherent in biopharmaceutical development; risks associated with
pre-clinical studies and clinical trials; and other risks
associated with obtaining regulatory approvals and protecting
intellectual property; as well as risks associated with general
economic conditions; the inability to recognize the anticipated
benefits of the recently completed merger, which may be affected
by, among other things, competition, Korro’s ability to grow and
manage growth profitably, maintain relationships with customers and
suppliers and retain key employees; costs related to merger; the
possibility that Korro may be adversely affected by other economic,
business, and/or competitive factors; other risks and uncertainties
indicated from time to time in Korro’s filings with the SEC,
including Item 1A. “Risk Factors” in Korro’s Annual Report on Form
10-K filed with the SEC on the date hereof, as such may be amended
or supplemented by its other filings with the SEC. Nothing in this
press release should be regarded as a representation by any person
that the forward-looking statements set forth herein will be
achieved or that any of the contemplated results of such
forward-looking statements will be achieved. You should not place
undue reliance on forward-looking statements in this press release,
which speak only as of the date they are made and are qualified in
their entirety by reference to the cautionary statements herein.
Except as required by law, Korro does not undertake or accept any
duty to release publicly any updates or revisions to any
forward-looking statements to reflect any change in their
expectations or in the events, conditions or circumstances on which
any such statement is based. This press release does not purport to
summarize all of the conditions, risks and other attributes of an
investment in Korro.
Korro Contact Information
Investors IR@korrobio.com
Media Glenn Silver FINN
Partners Glenn.silver@finnpartners.com
Korro Bio, Inc.Condensed Consolidated
Statements of Operations(in thousands, except
share and per share
amounts)(unaudited) |
|
|
|
Years Ended December 31, |
|
|
|
2023 |
|
|
2022 |
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
$ |
57,250 |
|
|
$ |
42,201 |
|
General and administrative |
|
|
27,284 |
|
|
|
16,797 |
|
Total operating expenses |
|
|
84,534 |
|
|
|
58,998 |
|
Loss from operations |
|
|
(84,534 |
) |
|
|
(58,998 |
) |
Other income: |
|
|
|
|
|
|
Other income, net |
|
|
3,389 |
|
|
|
976 |
|
Total other income, net |
|
|
3,389 |
|
|
|
976 |
|
Loss before provision for
income taxes |
|
|
(81,145 |
) |
|
|
(58,022 |
) |
Provision for income
taxes |
|
|
27 |
|
|
|
10 |
|
Net loss |
|
$ |
(81,172 |
) |
|
$ |
(58,032 |
) |
Other comprehensive
income |
|
|
|
|
|
|
Unrealized gain on available-for-sale investments |
|
|
— |
|
|
|
2 |
|
Comprehensive loss |
|
$ |
(81,172 |
) |
|
$ |
(58,030 |
) |
Net loss per share, basic and
diluted |
|
$ |
(53.08 |
) |
|
$ |
(227.42 |
) |
Weighted-average shares used
in computing net loss per share, basic and diluted |
|
|
1,529,321 |
|
|
|
255,175 |
|
|
Korro Bio, Inc.Condensed Consolidated
Balance Sheet Data(in
thousands)(unaudited) |
|
|
|
December 31, |
|
|
|
2023 |
|
|
2022 |
|
Cash, cash equivalents and investments |
|
$ |
166,150 |
|
|
$ |
55,248 |
|
Working capital (1) |
|
|
153,245 |
|
|
|
48,382 |
|
Total assets |
|
|
221,663 |
|
|
|
73,742 |
|
Total liabilities |
|
|
51,752 |
|
|
|
8,910 |
|
Total stockholders' equity
(deficit) |
|
|
169,911 |
|
|
|
(99,031 |
) |
|
|
|
|
|
|
|
(1) Working capital is defined as current assets
less current liabilities. |
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