4DMT Highlights Recent Clinical Pipeline Progress, Near-Term Milestones and Organizational Updates
January 04 2024 - 8:00AM
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 announced clinical pipeline progress,
updates to near-term milestones and organizational updates.
“2023 was a landmark year for 4DMT, highlighted by rapid
enrollment for 4D-150 in wet AMD and DME, strong CFTR protein
expression data for 4D-710 in lungs of people with CF, multiple
value-generating business development partnerships, and key
additions to the Executive Team,” said David Kirn, M.D., Co-founder
and Chief Executive Officer of 4DMT. “Looking forward to 2024, we
plan to release multiple important datasets from our lead programs
in wet AMD, DME and CF. We also plan to share guidance on the
design and timing of Phase 3 clinical trials. In addition, we
expect to initiate clinical development of 4D-175 in geographic
atrophy by the second half of the year. By the end of 2024, we
intend to progress clinical development in four large market
diseases, positioning us well in our strategy to become a fully
integrated large market genetic medicines company.”
Large Market Ophthalmology Portfolio Recent Updates and
Expected Upcoming Milestones
- 4D-150 for Wet AMD:
- Phase 2 PRISM Clinical Trial
- Randomized Dose Expansion arm (N=50)
in advanced high treatment need patients:
- Initial interim 24 week landmark
data to be presented at the Angiogenesis, Exudation, and
Degeneration 2024 Conference on Saturday, February 3, 2024 followed
by a corporate webcast with details to be announced at a future
date
- Population Extension arm (N≤45) in
broad population (non-advanced, standard treatment-need):
- Enrollment update expected in Q1
2024
- Initial interim 24 week landmark
data analysis expected in H2 2024
- Received both RMAT and PRIME in Q4
2023, enabling increased collaboration between the FDA and EMA on
regulatory approval planning, in addition to the opportunity for
expedited product development
- Update on Phase 3 trial plans
expected in February 2024 along with the interim randomized Phase 2
PRISM trial data
- 4D-150 for DME:
- Phase 2 SPECTRA Clinical Trial: Part
1 – Dose Confirmation Stage
- Completed enrollment in Q4 2023
- 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
Pulmonology Portfolio Expected Upcoming
Milestones
- 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 data 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
Cardiology Franchise Expected Upcoming
Milestones
- 4D-310 for Fabry Disease
Cardiomyopathy:
- Interim data update including
cardiac functional, imaging, quality-of-life and cardiac biopsy
data for all 6 patients dosed to be presented in the late-breaking
session at the WORLDSymposium™ 2024 in San Diego, California on
Friday, February 9 (10:15 a.m. to 11:15 a.m. PT) titled “Phase 1/2
clinical trial evaluating 4D-310 in adults with Fabry disease
cardiomyopathy: Interim analysis of cardiac and safety outcomes in
patients with 12-33 months of follow-up”
- FDA submission of data from the
non-human primate (NHP) study evaluating the safety and
biodistribution of intravenous (IV) 4D-310 with the
rituximab/sirolimus (R/S) immunosuppressive regimen compared to the
prior prednisone regimen expected in Q2 2024
Rare Inherited Retinal Disorders Portfolio Expected
Upcoming Milestones
- 4D-110 for Choroideremia and
4D-125 for X-Linked Retinitis Pigmentosa: The safety and
tolerability profiles for both product candidates remain unchanged
from prior data releases. All enrolled patients are expected to
reach 24 months of follow-up in 2024, and we plan to assess the
magnitude and durability of key imaging and functional endpoint
changes in evaluable patients. We expect to provide program updates
in 2024.
Expanded CNS and Gene Editing Capabilities Through
Partnership with Arbor Biotechnologies
- Partnership with a leader in
next-generation genetic medicines to engineer, co-develop and
co-commercialize (50:50 profit share) AAV-delivered
CRISPR/Cas-based therapeutics for up to six product candidates in
CNS
- Initial product candidate, led by
Arbor, will be developed for a target in amyotrophic lateral
sclerosis (ALS) with additional targets expected to be in diseases
with high unmet need in both rare and large patient
populations
- More information can be found in the
press release issued on January 3, 2024
Corporate Governance Update: New Chief Scientific
Officer (CSO) and Scientific Advisory Board (SAB)
- Appointment of Noriyuki
(Nori) Kasahara, M.D., Ph.D. as CSO:
- Noriyuki Kasahara (Nori), M.D.,
Ph.D., was appointed as Chief Scientific Officer of 4DMT, effective
January 2, 2024, and previously served as a member of 4DMT’s Board
of Directors and chaired the Board’s Science & Technology
Committee from September 2022 to December 2023. Dr. Kasahara was
most recently Professor & Alvera L. Kan Endowed Chair of
Neurological Surgery and Radiation Oncology at the University of
California, San Francisco Medical School (UCSF). He brings more
than 30 years of experience and has authored more than 150
peer-reviewed articles in the fields of gene therapy and genetic
engineering. As an academic researcher, he previously established
the first Vector Core & Shared Resource facilities for gene
delivery vector research at both the University of Southern
California and the University of California, Los Angeles. Most
recently, in addition to his professorship and Endowed Chair, he
served as a Principal Investigator in the UCSF Brain Tumor Center.
Dr. Kasahara’s research has focused on optimizing the application
of multiple and diverse viral vector delivery systems to achieve
maximal therapeutic potential, as well as developing
next-generation gene transfer technologies with greater therapeutic
efficacy in vivo.
- In his new role as CSO, Nori will
lead the creation and validation of new genetic medicine delivery
and transgene payload platforms. Our Chief Development Officer An
Song, Ph.D., will continue her role in leading nonclinical and
clinical scientific development functions, including AAV product
design and engineering, in vitro and in vivo pharmacology,
toxicology, immunology, biomarkers and bioanalytical development,
and further advancing the AAV-based Therapeutic Vector Evolution
platform.
- Newly Formed SAB Combines
the Expertise of Renowned Key Opinion Leaders in AAV Gene Therapy,
Immunology and Core 4DMT Therapeutic Areas
- Ronald Crystal,
M.D. is Professor and Chairman of the Department of
Genetic Medicine at the Weill Medical College of Cornell
University, where he is also the Bruce Webster Professor of
Internal Medicine, Director of the Belfer Gene Therapy Core
Facility and Attending Physician at the New York-Presbyterian
Hospital/Weill Cornell Medical Center. Dr. Crystal is a pioneer in
the field of gene therapy, where he was the first to use a
recombinant virus as a vehicle for in vivo gene therapy and has
carried out human trials of gene therapy for CF, cardiac ischemia,
cancer and central nervous system disorders.
- Amit Gaggar, M.D.,
Ph.D. is the William C. Bailey Endowed Chair of Pulmonary
Medicine and Professor of Medicine at University of Alabama at
Birmingham (UAB), where he serves as Scientific Director of the UAB
Lung Health Center. Dr. Gaggar is a physician-scientist with a long
standing interest in protease biology and innate immune signaling
in chronic lung disease such as chronic obstructive pulmonary
disease (COPD) and CF.
- Douglas Hanahan,
Ph.D. is Director Emeritus of the Swiss Institute for
Experimental Cancer Research at École Polytechnique Fédérale de
Lausanne (EFPL) and is a Distinguished Scholar at the Ludwig
Institute for Cancer Research. Dr. Hanahan is a pioneer in
angiogenesis, cancer research, and is a recipient of the AACR
Lifetime Achievement Award in Cancer Research.
- Richard Moss, M.D.
is Professor Emeritus at Stanford University School of Medicine in
the Department of Pediatrics’ Center for Excellence in Pulmonary
Biology. He is former Chief of the pediatric pulmonary and
allergy-immunology divisions, and former allergy-immunology and
pulmonary fellowship training programs Director at Stanford
Children’s Health/Lucile Packard Children’s Hospital Stanford. He
was Director of the Stanford Cystic Fibrosis Center from 1991 to
2009 and Principal Investigator for the CF Foundation’s
Therapeutics Development Network, where he also served as the
inaugural Chair of the TDN Protocol Review Committee. He has served
on CFF’s Clinical Research Committee, Translational Advisory Group
and Clinical Research Advisory Board.
- Amy Rosenberg,
M.D., a physician-immunologist, trained in basic
immunology in Al Singer’s lab in the Experimental Immunology Branch
of the NCI, NIH, following medical residency and specialty training
in Internal Medicine and Infectious Diseases. She then established
a laboratory and performed regulatory duties at the FDA and there,
for 33 years (14 as Director of the Division of Therapeutic
Proteins then Division III in the Office of Biotechnology
Products), regulated an array of biotechnology products including
cellular and protein therapeutics and cell-device combination
products. She is currently Senior Director of Immunology and
Protein Therapeutics at EpiVax, Inc.
- Ramesh Shivdasani, M.D.,
Ph.D. is Professor of Medicine / Medical Oncology at
Harvard Medical School, and serves as Deputy Director of the
Dana-Farber Cancer Institute. He is a gastrointestinal disease
expert, medical oncologist and laboratory investigator in the
Gastrointestinal Cancer Center at Dana-Farber and Brigham and
Women's Hospital, and is an internationally recognized expert in
cellular and tissue development and oncology.
- Daniel Takefman,
Ph.D. is Principal of Takefman Gene Therapy Advisors since
March 2020. Dr. Takefman provides expert regulatory advice for the
development and commercialization of cell and gene therapies, and
assists in due diligence assessments of gene therapy assets for
venture capital firms and is a member of multiple scientific
advisory boards. Previously Dr. Takefman was SVP and Head of
Regulatory Affairs at Spark Therapeutics for 5 years. At Spark, Dr.
Takefman supervised the submission through to approval of the FDA
and EMA Luxturna® marketing applications. Dr. Takefman also
supervised the regulatory process for multiple AAV based
investigation products including two additional Breakthrough
Designation products: SPK-9001 for the treatment of Hemophilia B
(marketing applications currently under review by FDA and EMA) and
SPK-8011 for the treatment of Hemophilia A. Dr. Takefman held
multiple positions during his 15-year career at FDA, ultimately
becoming Chief of the Gene Therapy Branch.
- Paul J. (PJ) Utz,
M.D. is Professor of Medicine at Stanford School of
Medicine in the Department of Medicine, Division of Immunology,
where he has directed a translational research lab for 24 years. He
co-founded several companies including Bayhill Therapeutics,
Tolerion and Yolo Immune, and has decades of experience as a
consultant and SAB member for many biotechnology and pharmaceutical
companies. Dr. Utz studies autoimmune diseases, including systemic
lupus erythematosus, rheumatoid arthritis, scleroderma, myositis,
Sjögren's disease, type I diabetes, vasculitis and multiple
sclerosis.
- More details on our members can be
found in the SAB section of our website
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 estimated cash or 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.
Contacts:
Media:
Katherine SmithEvoke CanaleKatherine.Smith@evokegroup.com
Investors:
Julian PeiHead of Investor Relations and Corporate
CommunicationsInvestor.Relations@4DMT.com267-644-5097
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