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 first quarter 2024 financial
results and provided operational highlights.
“The first quarter of 2024 kicks off another transformative year
for 4DMT with exceptional progress across our product pipeline and
platform in multiple therapeutic areas, particularly our lead
program 4D-150 in large market VEGF-driven retinal diseases,” said
David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.
“With robust clinical activity from 4D-150 demonstrated in our
initial interim 24-week results from the Phase 2 PRISM Dose
Expansion cohort in the severe disease activity wet AMD patient
population, we set the stage for additional important data readouts
in 2024, including 24-week results from the Phase 2 PRISM
Population Extension cohort in the broader wet AMD patient
population expected to be announced at ASRS in July and 24-week
results from the Phase 2 SPECTRA Dose Confirmation cohort in DME
expected to be announced in the fourth quarter. We believe the
emerging profile of 4D-150 shows its potential to be the
best-in-class, long-acting genetic medicine for broad market use in
VEGF-driven retinal diseases, and we look forward to completing
alignment with the FDA and EMA to rapidly advance 4D-150 into its
first pivotal trial for global development. Building on the success
of intravitreal R100, we are also encouraged by the preclinical
results from 4D-175 that we shared today at the 2024 Association
for Research in Vision and Ophthalmology (ARVO) Annual Scientific
Meeting and are looking forward to bringing the program into the
clinic for treatment of geographic atrophy in the second half of
the year, demonstrating the power of our modular vectors and
product engine. In addition, we continue to advance towards
important milestones for 4D-710 for cystic fibrosis, 4D-725 for
A1AT deficiency and 4D-310 for Fabry disease cardiomyopathy. Our
successful financing puts us in a strong position to execute on all
our corporate objectives, with expected cash runway into the first
half of 2027.”
Recent Highlights in Large Market Ophthalmology
Portfolio
- 4D-150 for Wet Age-Related
Macular Degeneration (wet AMD):
- Rapidly advanced intravitreal 4D-150
for wet AMD in the Phase 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 in February (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, high dose of 4D-150
(3E10 vg/eye) resulted in robust reductions in overall treatment
burden of 89%, percent of patients with 0-1 injections of 84%,
percent of patients injection-free of 63%, plus improved retinal
anatomical control vs. the aflibercept control arm
- Durable responses demonstrated
beyond one year in Phase 1 3E10 vg/eye dose cohort, with three
patients injection-free through 80–104 weeks (up to 2 years) of
follow-up
- Announced preliminary pivotal Phase
3 clinical trial design with additional regulatory interactions
with the U.S. Food and Drug Administration (FDA) and European
Medicines Agency (EMA) under RMAT (Regenerative Medicine Advanced
Therapy) and PRIME (Priority Medicines) designations underway
- Successfully completed the qualified
person (QP) inspection of our GMP manufacturing facilities and
operations resulting in issuance of a QP Declaration enabling
distribution of investigational medicinal products (IMP) in the EU
territories
- 4D-175 for Geographic
Atrophy:
- Preclinical data for 4D-175 for
geographic atrophy presented in an oral presentation at the 2024
ARVO Annual Scientific Meeting in May:
- Short-form complement factor H
(sCFH) transgene-derived protein showed functional activity
consistent with wild type full-length CFH in in vitro
experiments
- Dose-dependent transgene expression
and inhibition of alternative complement pathway by 4D-175
demonstrated in human retinal pigment epithelium (RPE) cells
- Intravitreal administration of
4D-175 to non-human primates (NHP) was safe and well tolerated and
resulted in robust transgene expression in the retina and
RPE/choroid, resulting in sCFH levels above target therapeutic
concentrations in the aqueous humor
- Presentation can be found on our
website under Scientific Presentations
Recent Highlights in Pulmonology Portfolio
- 4D-710 for Cystic Fibrosis
(CF) Lung Disease:
- Nine patients dosed to-date in Phase
1/2 AEROW clinical trial across four dose levels (range of 2.5E14
to 2E15 vg)
- Conducted initial pivotal study
interactions with the FDA and EMA; Company has clear registration
path for 4D-710 for treatment of cystic fibrosis (CF) lung disease
in people with CF who are ineligible for or cannot tolerate
approved CF modulator therapies
- Completed in-house process
development of a suspension Good Manufacturing Practice (GMP) ready
manufacturing process for 4D-710 at 500 liter scale for the pivotal
study
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 in February:
- 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
Expected Upcoming Milestones
- 4D-150 for Wet AMD:
- Phase 2 PRISM Population Extension
cohort (N=32) in the broader wet AMD patient population:
- Initial interim 24-week landmark
analysis expected to be presented at the ASRS Annual Scientific
Meeting on July 17-20, 2024
- Phase 3 planning:
- Update on Phase 3 clinical trial
design expected in Q3 2024
- First 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
analysis expected in Q4 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:
- Phase 1/2 AEROW clinical trial
interim clinical data expected at the ECFS Conference on June 5-8,
2024, including safety, lung biomarker and clinical activity data
on all nine patients dosed across four dose level cohorts
- Phase 2 Expansion cohort dose
selection expected to be shared in conjunction with interim data at
ECFS
- Phase 3 pivotal trial initiation
expected in H2 2025
- 4D-725 for
Alpha-1-Antitrypsin (A1AT) Deficiency Lung Disease:
- Program update 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
Q1 2024 Financial Results
Cash and Cash Equivalents and Marketable Securities: Cash and
cash equivalents and marketable securities were $589 million as of
March 31, 2024, as compared to $299 million as of December 31,
2023. The net increase in cash was primarily a result of cash
inflows from approximately $316 million of net proceeds from our
public offering of common stock completed in February including
partial exercise of underwriters’ option to purchase additional
shares. 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 $27.9
million for the first quarter of 2024, as compared to $22.4 million
for the first quarter of 2023. 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 $10.3
million for the first quarter of 2024, as compared to $8.0 million
for the first quarter of 2023.
Net Loss: Net loss was $32.4 million for the first quarter of
2024, as compared to net loss of $28.7 million for the first
quarter of 2023.
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®, 4D®, 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, as well as the plans, announcements and
related timing for the clinical development of, 4DMT’s 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 to be filed
on or about the date hereof, 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) |
|
|
|
Three Months Ended March 31, |
|
|
2024 |
|
|
2023 |
|
Collaboration and license revenue |
|
$ |
28 |
|
|
$ |
298 |
|
Operating expenses: |
|
|
|
|
|
Research and development |
|
|
27,870 |
|
|
|
22,412 |
|
General and administrative |
|
|
10,294 |
|
|
|
7,992 |
|
Total operating expenses |
|
|
38,164 |
|
|
|
30,404 |
|
Loss from operations |
|
|
(38,136 |
) |
|
|
(30,106 |
) |
Other income (expense), net: |
|
|
5,735 |
|
|
|
1,424 |
|
Net loss |
|
$ |
(32,401 |
) |
|
$ |
(28,682 |
) |
Net loss per share, basic and
diluted |
|
$ |
(0.66 |
) |
|
$ |
(0.88 |
) |
Weighted-average shares
outstanding used in computing net loss per share, basic and
diluted |
|
|
49,271,984 |
|
|
|
32,723,530 |
|
4D Molecular Therapeutics, Inc.Balance
Sheet Data (Unaudited)(in
thousands) |
|
|
|
|
|
March 31, |
|
December 31 |
|
|
|
2024 |
|
|
|
2023 |
|
Cash and cash equivalents and marketable securities |
|
$ |
588,853 |
|
|
|
$ |
299,186 |
|
Working capital |
|
|
519,186 |
|
|
|
|
277,637 |
|
Total assets |
|
|
629,884 |
|
|
|
|
339,891 |
|
Total liabilities |
|
|
29,323 |
|
|
|
|
32,062 |
|
Accumulated deficit |
|
|
(447,728 |
) |
|
|
|
(415,327 |
) |
Total stockholders’
equity |
|
|
600,561 |
|
|
|
|
307,829 |
|
|
Contacts:
Media:Katherine SmithInizio Evoke
CommsKatherine.Smith@inizioevoke.com
Investors:Julian PeiHead of Investor Relations
and Corporate CommunicationsInvestor.Relations@4DMT.com
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