Metagenomi, Inc. (Nasdaq: MGX) (Metagenomi), a precision genetic
medicines company committed to developing curative therapeutics for
patients using its proprietary gene editing toolbox, today provided
a business update and reported third quarter 2024 financial
results.
“Our strong pace of innovation and execution continued in the
third quarter, highlighted by the nomination of our first DC,
MGX-001, intended as a one-time curative gene editing therapeutic
for both adults and children with hemophilia A,” said Brian C.
Thomas, PhD, CEO and founder of Metagenomi. “Supporting this DC
nomination, we achieved successful proof-of-concept in NHPs,
demonstrating site specific integration and durable Factor VIII
activity levels through 12 months. This preclinical study provided
an important validation of our gene editing platform, supporting
our potential to overcome a key limitation of gene therapies that
have struggled to achieve long term persistence of Factor VIII
expression in patients. Pre-clinical data from our hemophilia A
program has been accepted for oral presentation at the American
Society of Hematology meeting in December and we remain on track to
file an IND for MGX-001 in 2026.”
“Metagenomi’s collaboration with Ionis is progressing, with one
to two planned DC nominations in 2025, focused on cardiometabolic
development programs, where gene editing could represent a
transformative option for patients. Our gene editing toolbox
continues to differentiate on multiple fronts. We recently unveiled
our novel ABE platform which we believe has superior targetability,
precision and efficiency. Additionally, our ultra small SMART
editing systems showed compatibility with single-AAV delivery,
potentially unlocking novel in vivo extrahepatic therapeutic
development opportunities in the neuromuscular space.”
Third Quarter 2024 Business Updates
Therapeutic Pipeline Updates
- Metagenomi continues to advance its
wholly-owned lead program in hemophilia A, designed as a one-time
curative treatment for both adults and children.
- Preclinical data utilizing a
cynomolgus Factor VIII (FVIII) construct showed site-specific
integration and durable FVIII activity levels in NHPs over 12
months and the study remains ongoing.
- Updated preclinical data has been
accepted for an oral presentation at the American Society of
Hematology (ASH) 66th Annual Meeting and Exposition in San Diego in
December.
- The company initiated manufacturing
activities to support IND enabling studies, and MGX-001 remains on
track for an IND filing in 2026.
- Building on the hemophilia A program,
the company is advancing additional wholly-owned therapeutic
candidates targeting secreted protein disorders, leveraging the
MGX-001 editing system with the goal of achieving targeted and
durable gene expression.
- All four therapeutic targets in the
first wave of the company’s collaboration with Ionis are advancing
in lead optimization.
- In vivo rodent proof-of-concept was
achieved in all four wave 1 genetic targets, including
transthyretin (TTR) for transthyretin amyloidosis and
angiotensinogen (AGT) for refractory hypertension as well as two
undisclosed programs in significant cardiometabolic
indications.
- The company remains on track to
nominate one to two development candidates in 2025.
Technology Platform Updates
- Using PAM interacting domain
engineering, Metagenomi’s ABE platform is able to target over 95%
of the human genome's base pairs, a significantly wider range of
sites than first-generation SpCas9 base editors. The ABE achieved
over 95% triplex protein knockdown at key gene targets in primary T
cells. Genome-wide analyses confirmed no detectable translocations
and no changes in stress-related gene expression post-editing.
- The company’s SMART platform
includes genome editing systems that are small enough to be
packaged into single AAV vectors even when additional effector
domains are included for base editing. The company demonstrated
50-fold optimization of an ultra small base editing system using
its proprietary metagenomics database and AI tools, potentially
allowing for therapeutic levels of editing at targets in the
neuromuscular space.
Upcoming Events Metagenomi plans to participate
in the following investor conferences during the fourth quarter of
2024:
- Jefferies London Healthcare
Conference Corporate presentation on Thursday, November 21, 2024 at
1:30pm GMTA live webcast and replay will be available in the
investor section of the company's website at
https://ir.metagenomi.co/
- Piper Sandler 36th Annual
Healthcare Conference, NYC Tuesday, December 3, 20241x1 investor
meetings only
Metagenomi plans to participate in the following scientific
conferences during the fourth quarter of 2024:
- ASH 66th Annual Meeting and
Exposition in San DiegoOral presentation “Site-Specific Insertion
of Factor VIII Gene Results in Durable Factor VIII Expression in
Nonhuman Primates”Monday, December 9, 2024, 5:30pm PT
- Nature conferences: RNA at the
Bench and Bedside IV, The Salk Institute La Jolla December 11,
3:05pm PT
Third Quarter 2024 Financial Results
Cash Position: Cash,
cash equivalents, and available-for-sale marketable securities were
$274.6 million as of September 30, 2024.
R&D Expenses:
Research and development (R&D) expenses were $26.3 million for
the three months ended September 30, 2024, compared to $26.8
million for the three months ended September 30, 2023.
G&A Expenses:
General and administrative (G&A) expenses were $7.6 million for
the three months ended September 30, 2024, compared to $7.9
million for the three months ended September 30, 2023.
About Hemophilia A
Hemophilia A is the most common X-linked inherited bleeding
disorder, caused by a large variety of mutations in the FVIII gene
leading to a loss of functional FVIII protein. Intracranial
bleeding is of greatest concern as this can lead to major morbidity
and mortality. Bleeding into joints leads to cumulative joint
damage and is a major cause of morbidity. Diagnosis of severe
disease typically occurs in infancy due to exaggerated bleeding in
response to minor injury or routine medical procedures. Prevalence
is estimated to be up to 26,500 patients in the US and more than
500,000 patients globally according to the World Federation of
Hemophilia, with the vast majority of patients being male.
About Metagenomi
Metagenomi is a precision genetic medicines company committed to
developing curative therapeutics for patients using its
proprietary, comprehensive metagenomics-derived toolbox. Metagenomi
is harnessing the power of metagenomics, the study of genetic
material recovered from the natural environment, to unlock four
billion years of microbial evolution to discover and develop a
suite of novel editing tools capable of correcting any type of
genetic mutation found anywhere in the genome. Its comprehensive
genome editing toolbox includes programmable nucleases, base
editors, and RNA and DNA-mediated integration systems (including
prime editing systems and clustered regularly interspaced short
palindromic repeat associated transposases). Metagenomi believes
its diverse and modular toolbox positions the company to access the
entire genome and select the optimal tool to unlock the full
potential of genome editing for patients. For more information,
please visit https://metagenomi.co.
Metagenomi intends to use the Investor Relations section of its
website as a means of disclosing material nonpublic information and
for complying with its disclosure obligations under Regulation FD.
Accordingly, investors should monitor Metagenomi’s website in
addition to following its press releases, SEC filings, public
conference calls, presentations, and webcasts.
Cautionary Note Regarding Forward‐Looking
Statements
This press release contains “forward-looking statements” within
the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934, each as
amended. Such statements, which are often indicated by terms such
as “anticipate,” “believe,” “could,” “estimate,” “expect,”
“goal,” “intend,” “look forward to,” “may,” “plan,”
“potential,” “predict,” “project,” “should,” “will,” “would”
and similar expressions, include, but are not limited to, any
statements relating to our growth strategy and product development
programs, including the timing of and our ability to conduct
IND-enabling studies and make regulatory filings such as INDs,
statements concerning the potential of therapies and product
candidates, including our development candidate, MGX-001,
statements concerning the timing of data presentations and
publications, statements regarding our cash runway and any other
statements that are not historical facts. Forward looking
statements are based on management’s current expectations and are
subject to risks and uncertainties that could negatively affect our
business, operating results, financial condition, and stock value.
Factors that could cause actual results to differ materially from
those currently anticipated include: risks relating to our growth
strategy; our ability to obtain, perform under, and maintain
financing and strategic agreements and relationships; risks
relating to the results of research and development activities;
risks relating to the timing of starting and completing clinical
trials; uncertainties relating to preclinical and clinical testing;
our dependence on third party suppliers; risk related to our
ability to attract, integrate and retain key personnel; risks
related to the early stage of product candidates under development;
risks related to our need for substantial additional funding; risk
related to our estimates of our cash runway, including that our
assumptions underlying our cash runway may be incorrect; risks
related to regulatory matters; risks related to patent and
intellectual property matters; risks related to the competitive
nature of our industry; as well as other risks described in “Risk
Factors,” in our most recent Form 10-K and our most recent 10-Qs on
file with the Securities and Exchange Commission. We expressly
disclaim any obligation or undertaking to release publicly any
updates or revisions to any forward-looking statements contained
herein to reflect any change in our expectations or any changes in
events, conditions or circumstances on which any such statement is
based, except as required by law, and we claim the protection of
the safe harbor for forward-looking statements contained in the
Private Securities Litigation Reform Act of 1995.
Investor Contact:Simon Harnest - CIO, SVP
Investor Relationssimon@metagenomi.co
Media Contact:Ashlye Hodge - Communications
Managerashlye@metagenomi.co
|
|
|
|
Condensed Financial StatementsCondensed
Consolidated Balance Sheet
Data(Unaudited) |
|
|
|
|
|
|
|
September 30, |
|
|
December 31, |
|
| (in thousands) |
|
2024 |
|
|
2023 |
|
|
Cash, cash equivalents and available-for-sale marketable
securities |
|
$ |
274,587 |
|
|
$ |
271,182 |
|
| Total assets |
|
$ |
358,348 |
|
|
$ |
364,842 |
|
| Total liabilities |
|
$ |
102,575 |
|
|
$ |
149,668 |
|
| Redeemable convertible
preferred stock |
|
$ |
— |
|
|
$ |
350,758 |
|
| Total stockholders’ equity
(deficit) |
|
$ |
255,773 |
|
|
$ |
(135,584 |
) |
| Total liabilities, redeemable
convertible preferred stock and stockholders’ equity (deficit) |
|
$ |
358,348 |
|
|
$ |
364,842 |
|
|
|
|
|
Condensed Consolidated Statements of
Operations(Unaudited) |
|
|
|
|
|
|
|
Three Months EndedSeptember 30, |
|
|
Nine Months EndedSeptember 30, |
|
| (In thousands, except share
and per share data) |
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
|
Collaboration revenue |
|
$ |
11,514 |
|
|
$ |
12,363 |
|
|
$ |
42,681 |
|
|
$ |
32,357 |
|
| Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
26,256 |
|
|
|
26,837 |
|
|
|
86,015 |
|
|
|
69,648 |
|
|
General and administrative |
|
|
7,641 |
|
|
|
7,921 |
|
|
|
24,944 |
|
|
|
21,005 |
|
|
Total operating expenses |
|
|
33,897 |
|
|
|
34,758 |
|
|
|
110,959 |
|
|
|
90,653 |
|
| Loss from operations |
|
|
(22,383 |
) |
|
|
(22,395 |
) |
|
|
(68,278 |
) |
|
|
(58,296 |
) |
| Other income (expense): |
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income |
|
|
3,616 |
|
|
|
3,866 |
|
|
|
11,526 |
|
|
|
11,836 |
|
|
Change in fair value of long-term investments |
|
|
(2,055 |
) |
|
|
— |
|
|
|
(2,055 |
) |
|
|
2,870 |
|
|
Other expense, net |
|
|
(57 |
) |
|
|
(85 |
) |
|
|
(158 |
) |
|
|
(70 |
) |
|
Total other income, net |
|
|
1,504 |
|
|
|
3,781 |
|
|
|
9,313 |
|
|
|
14,636 |
|
| Net loss before benefit
(provision) for income taxes |
|
|
(20,879 |
) |
|
|
(18,614 |
) |
|
|
(58,965 |
) |
|
|
(43,660 |
) |
|
Benefit (provision) for income taxes |
|
|
2,106 |
|
|
|
(1,206 |
) |
|
|
4,305 |
|
|
|
(5,301 |
) |
| Net loss |
|
$ |
(18,773 |
) |
|
$ |
(19,820 |
) |
|
$ |
(54,660 |
) |
|
$ |
(48,961 |
) |
| Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(0.51 |
) |
|
$ |
(5.82 |
) |
|
$ |
(1.73 |
) |
|
$ |
(14.38 |
) |
| Weighted average common shares
outstanding, basic and diluted |
|
|
36,766,309 |
|
|
|
3,404,585 |
|
|
|
31,601,825 |
|
|
|
3,404,585 |
|
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