4DMT’s 4D-150 gene drug for the intravitreal treatment of wet AMD received FDA Regenerative Medicine Advanced Therapy (RMAT) designation, the first RMAT designation for wet AMD

• The designation follows interim Phase 1 PRISM clinical data for 4D-150 that demonstrated an encouraging safety, tolerability and clinical activity profile in patients with wet age-related macular degeneration (wet AMD)

• 4D-150 is the first therapeutic candidate to receive RMAT or breakthrough designation for wet AMD

• The RMAT designation follows the Priority Medicines (PRIME) designation granted by the European Medicines Agency (EMA) in October 2023

• Obtaining RMAT and PRIME enhances collaboration between FDA and EMA in regulatory approval planning, as well as opportunities to expedite product development

• Initial interim milestone data from the Phase 2 PRISM randomized dose expansion phase (N=50 at 24 weeks) of 4D-150 in patients with advanced, high treatment need wet AMD will be presented on Saturday, February 3 in Angiogenesis, Exudation and Transgender 2024 Conference to be announced in 2024 at 4:20 pm (ET)

EMERYVILLE, Calif., Dec. 21, 2023 (GLOBE NEWSWIRE) — 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company) is a leading clinical-stage genetic medicine company focused on delivering gene medicine therapies The full potential of large cell diseases. Ophthalmology and Pulmonary Market Diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted RMAT designation to investigational genetic drug candidate 4D-150 for the intravitreal treatment of wet age-related macular degeneration (wet AMD). The FDA granted RMAT designation to 4D-150 based on its potential to address unmet medical needs in this population.

The RMAT designation is part of the 21st Century Cures Act. The program was created to expedite the development and review of regenerative medicine therapies designed to treat, modify, reverse or cure serious diseases. Obtaining RMAT designation provides the sponsoring company with all the benefits of the Fast Track and Breakthrough Therapy designation programs, allowing early, close, and frequent interaction with the FDA to expedite drug development. The designation follows interim Phase 1 PRISM clinical data for 4D-150, which demonstrated an encouraging safety, tolerability and clinical activity profile. 4D-150 has the potential to address an unmet medical need to safely maintain long-term visual outcomes while avoiding the need for repeated intravitreal injections. 4DMT is currently working with the FDA and EMA to develop an initial Phase 3 clinical trial plan and expects to provide an update in February 2024 along with data from the 4D-150 interim randomized Phase 2 PRISM trial.

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“We are honored to receive the first-ever RMAT designation for the treatment of wet AMD with our intravitreal 4D-150 gene therapy,” said David Kirn, MD, co-founder and CEO of 4DMT. This title confirms our belief that 4D-150 has the potential to address the high unmet needs of patients with wet AMD, maintain their visual outcomes and reduce their treatment burden. We continue to work closely with the FDA and EMA to effectively advance the development of 4D-150 for wet AMD and diabetic macular edema, and having the RMAT and PRIME designations allows us to further rapidly collaborate in our efforts to advance development in these two regions.

Robert Kim, MD, chief medical officer of 4DMT, said patients with wet AMD are awaiting a durable treatment that has the potential to significantly reduce treatment burden and preserve vision long-term. We are grateful that two of the world’s largest pharmaceutical regulatory agencies recognized the potential of 4D-150 and granted unprecedented RMAT and PRIME designations in wet AMD. I am extremely proud of the 4DMT team for this achievement, and we look forward to sharing preliminary interim data from our randomized Phase 2 dose-expansion clinical trial in patients with the highest anti-VEGF need at the 2024 Angiogenesis Meeting in February.

In July 2023, 4DMT announced that the dose response was demonstrated in support of the highest tested dose of 3E10 vg/eye, including a promising reduction in supplemental anti-VEGF injections (4 of 4 evaluable patients did not require injections) and a clinically meaningful reduction in anti-VEGF injections. Mean central subzone thickness (CST) at 36 weeks in the patient group with higher VEGF requirements. As of July 2023, intravitreal 4D-150 remains well tolerated in all patients at all doses, with no grade 1 inflammatory cells, hypotonia, dose-limiting toxicities, or treatment-related events during 36 weeks of follow-up. Serious Adverse Events (SAE). All 15 patients. Additionally, 4DMT announced that the 3E10 vg/eye dose cohort demonstrated sustained reductions in supplemental anti-VEGF injections after 36 weeks, with 3 of 4 evaluable participants remaining injection-free after one year and one patient at the longest follow-up period. No changes in the safety profile were observed by remaining uninjected for up to 80 weeks.

About Wet AMD’s 4D-150

4D-150 consists of our customized and evolved intravitreal vector R100 and a transgenic cassette expressing aflibercept and VEGF-C inhibitory RNAi. This dual transgenic payload inhibits four members of the VEGF family of angiogenic factors that drive wet AMD and DME: VEGF A, B, C, and PlGF. R100 was invented on 4DMT through our proprietary therapeutic vector evolution platform; we developed this platform using the principles of directed evolution, a Nobel Prize-winning technology. 4D-150 is designed for single-shot, low-dose intravitreal delivery for retinal transgene expression without significant inflammation.

About Wet AMD

Wet AMD is a highly prevalent disease, affecting an estimated 200,000 new patients in the United States each year. It is estimated that the total prevalence of wet AMD in major markets including the United States, the European Union (major markets) and Japan will exceed 4 million in the next five years. Wet AMD is a form of macular disease in which abnormal blood vessels (choroidal neovascularization, or CNV) grow into the macula, the central area of ​​the retina. As a result, CNV can cause retinal swelling and edema, hemorrhage, and scarring, as well as visual distortion and loss of vision. The proliferation and leakage of abnormal blood vessels are stimulated by VEGF. This process distorts and can destroy central vision, which can lead to blindness if left untreated.

About 4DMT

4DMT is a leading clinical-stage genetic medicine company dedicated to unlocking the full potential of genetic medicines to treat large-market diseases in ophthalmology and pulmonary diseases. 4DMT’s proprietary invention platform, Therapeutic Vector Evolution, combines Nobel Prize-winning technology and the power of directed evolution with approximately 1 billion synthetic AAV capsid-derived sequences to invent custom and evolved vectors for use in our wholly-owned and collaborative product candidates. Our product design, development and manufacturing engines help us efficiently create and advance a diverse pipeline of products with the goal of revolutionizing medicine by delivering potential treatments to millions of patients. Currently, 4DMT is advancing five clinical-stage product candidates and two preclinical product candidates, each tailored for rare and large-market diseases in ophthalmology, pulmonology and cardiology. 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 received marketing approval from the FDA or any other regulatory agency. We make no representations regarding the safety or effectiveness for therapeutic use of our investigational product candidates.

For more information, visit 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, but not limited to, implied and express statements regarding the therapeutic potential and clinical benefit of the 4DMT product candidate and the clinical development of 4D-150 Plans, announcements and related timing of regulatory interactions. “Could”, “could”, “will”, “could”, “would”, “should”, “expect”, “plan”, “anticipate”, “intend”, “believe”, expect, “estimate” , seek, “forecast,” future, “project,” “potential,” “continue,” “goal” 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 could cause actual events or results to differ from those expressed or implied in any forward-looking statement. There are material differences in the risks and uncertainties contained in this press release, including in more detail in the section titled “Risk Factors” in 4D Molecular Therapeutics’ most recent Quarterly Report on Form 10-Q and any subsequent filings with the Securities and Exchange Commission. Describe risks and uncertainties. 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 expressly disclaims any obligation to update any forward-looking statements. No representation or warranty (express or implied) is made as to the accuracy of any such forward-looking statements.

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