4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company),
a leading clinical-stage genetic medicines company focused on
unlocking the full potential of genetic medicines to treat large
market diseases, today reported full year 2023 financial results
and provided operational highlights.
“2023 was a transformative year for 4DMT with incredible
progress across our product pipeline and platform in multiple
therapeutic areas, especially for our lead program 4D-150 in wet
age-related macular degeneration (wet AMD) and diabetic macular
edema (DME),” said David Kirn, M.D., Co-founder and Chief Executive
Officer of 4DMT. “Robust enrollment ahead of schedule across
multiple cohorts in the PRISM clinical trial of patients with wet
AMD and the SPECTRA clinical trial of patients with DME have set
the stage for multiple important data readouts in 2024, starting
with our recent positive interim data at Angiogenesis, Exudation,
and Degeneration 2024 Conference in the hardest-to-treat wet AMD
patients. As of the most recent data update, 110 patients have been
dosed with 4D-150 with no clinically significant inflammation,
which continues to build on the emerging profile of 4D-150 as a
potentially best-in-class long-acting genetic medicine with the
opportunity for broad market adoption. We look forward to further
updates in the broad wet AMD population and DME patients in the
second half of 2024, setting the stage for our first Phase 3
clinical trial in wet AMD currently expected to initiate in the
first quarter of 2025. We also look forward to providing updates on
other key programs beyond 4D-150 in 2024, including 4D-175 for
geographic atrophy, 4D-710 for cystic fibrosis and 4D-310 for Fabry
disease cardiomyopathy. Our recent financing and resulting cash
position allows us to execute on our vision of becoming a fully
integrated, next-generation genetic medicines company.”
Recent Highlights in Large Market Ophthalmology
Portfolio
- 4D-150 for Wet Age-Related
Macular Degeneration:
- Rapidly advanced intravitreal 4D-150
for wet AMD in the Phase 1/2 PRISM clinical trial
- Randomized Dose Expansion cohort
(N=51) in patients with severe disease activity and high treatment
burden presented at Angiogenesis, Exudation, and Degeneration 2024
Conference (data cutoff January 19, 2024):
- Favorable safety profile
demonstrated, with no significant or recurrent intraocular
inflammation, and no 4D-150–related serious adverse events (SAEs)
or study eye SAEs. All patients were off steroids
- At 24 weeks, 4D-150 resulted in
robust reductions in overall treatment burden, % of patients with
0-1 injections, % of patients injection-free, plus improved retinal
anatomical control vs. the aflibercept control
- Durable responses demonstrated
beyond 1 year in Phase 1 3E10 vg/eye dose cohort, with 3 patients
injection-free through 80–104 weeks (up to 2 years) of
follow-up
- Enrollment completed ahead of
schedule in Phase 2 PRISM Population Extension cohort (N=32)
evaluating 4D-150 in patients with broad disease activity and
treatment burden versus the Dose Expansion cohort
- Received RMAT and PRIME
designations, enabling increased collaboration between the FDA and
EMA on regulatory planning
- Announced preliminary pivotal Phase
3 clinical trial design and plans
- 4D-150 for Diabetic Macular
Edema:
- Enrollment and dosing completed
ahead of schedule in Dose Confirmation cohort (N=22) of the Phase 2
SPECTRA clinical trial in DME
Recent Highlights in Pulmonary Portfolio
- 4D-710 for Cystic Fibrosis
Lung Disease:
- Presented positive interim data from
the Phase 1/2 AEROW clinical trial at the North American Cystic
Fibrosis Conference
- Dose ranging continues (5E14 – 2E15
vg) with lung biopsy CFTR expression significantly above normal
controls, demonstrating the feasibility of efficient delivery and
CFTR transgene expression at lower doses; first participant dosed
in lower dose Cohort 3 (5E14 vg)
- Received Rare Pediatric Disease
Designation in January 2024 and Orphan Drug Designation from FDA in
February 2024
Recent Highlights in Cardiology Portfolio
- 4D-310 for Fabry Disease
Cardiomyopathy:
- Interim clinical and biopsy data
from Phase 1/2 INGLAXA clinical trials presented in the
late-breaking session at WORLDSymposium™ 2024:
- 4D-310 demonstrated clinically
meaningful improvements on multiple cardiac endpoints through 12-24
months in five evaluable patients
- Cardiac biopsies from one patient at
week 6 and 24 showed robust and durable delivery, transgene
expression and clearance of Gb3 substrate in cardiomyocytes
- Safety and tolerability profile
maintained; previously reported cases of atypical hemolytic uremic
syndrome (aHUS) (n=3) have fully resolved
- Reached agreement with FDA on a
proposed plan to address clinical hold. Single non-human primate
safety study evaluating intravenous 4D-310 with the
rituximab/sirolimus immunosuppressive regimen underway
Strategic Agreements & Partnerships
- In July 2023, entered license
agreement with Astellas Pharma for their use of R100 vector for
genetic targets implicated in rare monogenic ophthalmic diseases
and received $20 million upfront with potential future milestones
of up to $942.5 million, including potential near-term development
milestones of $15 million for the initial target
- In December 2023, entered strategic
partnership with Arbor Biotechnologies to engineer, co-develop and
co-commercialize AAV-delivered CRISPR/Cas-based therapeutics for up
to six product candidates in the CNS therapeutic area
Expected Upcoming Milestones
- 4D-150 for Wet AMD:
- Phase 2 PRISM Population Extension
cohort (N=32) in patients with broad disease activity and treatment
burden:
- Initial interim 24-week landmark
analysis expected in Q3 2024
- Phase 3 Planning:
- Additional FDA and EMA regulatory
interactions under RMAT and PRIME planned in Q2 2024; update
expected in Q3 2024
- Phase 3 clinical trial initiation
expected in Q1 2025
- 4D-150 for DME:
- Phase 2 SPECTRA clinical trial Dose
Confirmation cohort (N=22):
- Initial interim 24-week landmark
data expected in H2 2024
- 4D-175 for Geographic
Atrophy:
- IND filing expected in Q2 2024
- Phase 1 initiation expected in H2
2024
- 4D-710 for CF Lung
Disease:
- Update on FDA feedback on
development plan for monotherapy and approved CF modulator
combination regimens expected in Q1 2024
- Interim update from Phase 1/2 AEROW
clinical trial expected in mid-2024
- 4D-725 for A1AT Deficiency
Lung Disease:
- Program update and initiation of
IND-enabling studies expected in 2024
- 4D-310 for Fabry Disease
Cardiomyopathy:
- FDA submission of preclinical NHP
safety data in combination with rituximab/sirolimus
immunosuppression regimen expected in Q2 2024
- 4D-110 for Choroideremia and
4D-125 for X-Linked Retinitis Pigmentosa:
- Program updates expected in
2024
Full Year 2023 Financial Results
Cash and Cash Equivalents and Marketable Securities: Cash and
cash equivalents and marketable securities were $299 million as of
December 31, 2023, as compared to $218 million as of December 31,
2022. The net increase in cash was primarily a result of cash
inflows from $129 million of net proceeds from our public offering
of common stock completed in May, $19 million of net proceeds under
our Open Market Sales Agreement, and the $20 million upfront
payment in connection with the Astellas License Agreement and was
partially offset by cash used in operations. In addition, in
February 2024 we completed a public offering of common stock and
prefunded warrants that resulted in us receiving net proceeds of
approximately $281 million. We currently expect cash and cash
equivalents to be sufficient to fund operations into the first half
of 2027.
R&D Expenses: Research and development expenses were $97.1
million for 2023, as compared to $80.3 million for 2022. This
increase was driven by the progression of our existing clinical
trials, primarily 4D-150 in wet AMD and DME, along with increased
payroll and stock-based compensation expense due to higher
headcount.
G&A Expenses: General and administrative expenses were $36.5
million for 2023, as compared to $32.9 million for 2022.
Net Loss: Net loss was $100.8 million for 2023, as compared to
net loss of $107.5 million for 2022.
About 4DMT
4DMT is a leading clinical-stage genetic medicines company
focused on unlocking the full potential of genetic medicines to
treat large market diseases in ophthalmology and pulmonology.
4DMT’s proprietary invention platform, Therapeutic Vector
Evolution, combines the power of the Nobel Prize-winning
technology, directed evolution, with approximately one billion
synthetic AAV capsid-derived sequences to invent customized and
evolved vectors for use in our wholly owned and partnered product
candidates. Our product design, development, and manufacturing
engine helps us efficiently create and advance our diverse product
pipeline with the goal of revolutionizing medicine with potential
curative therapies for millions of patients. Currently, 4DMT is
advancing five clinical-stage and two preclinical product
candidates, each tailored to address rare and large market diseases
in ophthalmology, pulmonology, and cardiology. In addition, 4DMT is
also advancing programs in CNS through a gene editing partnership.
4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™,
and the 4DMT logo are trademarks of 4DMT.
All of our product candidates are in clinical or preclinical
development and have not yet been approved for marketing by the FDA
or any other regulatory authority. No representation is made as to
the safety or effectiveness of our product candidates for the
therapeutic uses for which they are being studied.
Learn more at www.4DMT.com and follow us on LinkedIn.
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 regarding the therapeutic potential, and
clinical benefits of 4DMT’s product candidates, as well as the
plans, announcements and related timing for the clinical
development of our clinical and preclinical product candidates, and
statements regarding our financial performance, results of
operations and anticipated cash runway. 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 risks and
uncertainties that are described in greater detail in the section
entitled "Risk Factors" in 4D Molecular Therapeutics’ 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 4D Molecular Therapeutics'
views only as of today and should not be relied upon as
representing its views as of any subsequent date. 4D Molecular
Therapeutics 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.
4D Molecular Therapeutics, Inc.Statements
of Operations (Unaudited)(in
thousands, except share and per share
amounts) |
|
|
|
Year Ended December 31, |
|
|
2023 |
|
|
2022 |
|
Collaboration and license revenue |
|
$ |
20,723 |
|
|
$ |
3,129 |
|
Operating expenses: |
|
|
|
|
|
Research and development |
|
|
97,096 |
|
|
|
80,253 |
|
General and administrative |
|
|
36,494 |
|
|
|
32,908 |
|
Total operating expenses |
|
|
133,590 |
|
|
|
113,161 |
|
Loss
from operations |
|
|
(112,867 |
) |
|
|
(110,032 |
) |
Other
income (expense), net: |
|
|
12,030 |
|
|
|
2,538 |
|
Net
loss |
|
$ |
(100,837 |
) |
|
$ |
(107,494 |
) |
Net loss
per share, basic and diluted |
|
$ |
(2.58 |
) |
|
$ |
(3.32 |
) |
Weighted-average shares outstanding used in computing net loss per
share, basic and diluted |
|
|
39,130,067 |
|
|
|
32,351,221 |
|
4D Molecular Therapeutics, Inc.Balance
Sheet Data (Unaudited)(in
thousands) |
|
|
|
As of December 31, |
|
|
|
2023 |
|
|
2022 |
|
Cash and cash equivalents and marketable securities |
|
$ |
299,186 |
|
|
$ |
218,462 |
|
Working
capital |
|
|
277,637 |
|
|
|
204,780 |
|
Total
assets |
|
|
339,891 |
|
|
|
261,846 |
|
Total
liabilities |
|
|
32,062 |
|
|
|
30,509 |
|
Accumulated deficit |
|
|
(415,327 |
) |
|
|
(314,490 |
) |
Total
stockholders’ equity |
|
|
307,829 |
|
|
|
231,337 |
|
|
|
|
|
|
|
|
|
|
Contacts:
Media:
Katherine SmithInizio Evoke
CommsKatherine.Smith@inizioevoke.com
Investors:
Julian PeiHead of Investor Relations and Corporate
CommunicationsInvestor.Relations@4DMT.com
4D Molecular Therapeutics (NASDAQ:FDMT)
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