4DMT Presents Positive Interim Data from Intravitreal 4D-150 Phase 1/2 PRISM Clinical Trial in Patients with Wet AMD at ARVO 2023
27 Apr 2023
Phase 1Clinical Result
EMERYVILLE, CA, USA I April 27, 2023 I 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT, or the Company), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, today announced positive interim clinical data from the Phase 1 Dose Exploration stage (three doses, n=15) of the 4D-150 Phase 1/2 PRISM clinical trial for wet age-related macular degeneration (wet AMD)AMD). 4D-150, a novel genetic medicine that utilizes 4DMT’s evolved and customized R100 vector for intravitreal delivery to the retina, was well- tolerated at all doses. All doses demonstrated clinical activity, including a reduction in anti-VEGF injection burden, stable or improved retinal edema and thickness, and stable visual acuity. A dose response was demonstrated in favor of the high dose 3E10 vg/eye. Interim data from the study will be presented today in an oral presentation titled, “Interim results for the Phase 1/2 PRISM Trial evaluating 4D-150, a dual-transgene intravitreal genetic medicine in individuals with neovascular (wet) age-related macular degeneration,” at the 2023 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in New Orleans, LA at 11:30 a.m. ET.
“4D-150 is the first retinal genetic medicine that is designed to inhibit all four VEGF-related molecules that drive disease in wet AMDAMD,” said Robert Kim, M.D., Chief Medical Officer. “We are impressed by the clinical activity reported today, especially in the high dose cohort where 80% of patients did not require anti-VEGF injections through 36 weeks. We continue to execute relentlessly on enrolling the Phase 2 Dose Expansion stage of the PRISM trial in an effort to advance this potentially transformative therapy into late-stage development as soon as possible.”
Arshad M. Khanani, M.D., M.A., FASRS, Managing Partner and Director of Clinical Research at Sierra Eye Associates, Clinical Associate Professor at University of Nevada, Reno, and a Principal Investigator in the 4D-150 PRISM clinical trial, added, “I continue to be encouraged by the tolerability and clinical activity of a single intravitreal injection of 4D-150 in high-need patients with wet AMDAMD. Given the stabilization or improvements seen in retinal anatomy and maintenance of vision up to 9 months, I believe 4D-150 has the potential to be a transformative treatment for patients with wet AMDAMD and to greatly reduce their treatment burden.”
“These clinical data on 4D-150 represent another important milestone for patients with wet AMDAMD and for 4DMT,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “We believe these results further validate the potential of our intravitreal R100 vector, for the safe delivery of effective transgene payloads at relatively low doses, and our Therapeutic Vector Evolution platform. These promising interim clinical data and excellent progress on Phase 2 enrollment further demonstrate the power of our product design and development engine.”
Phase 1/2 PRISM Clinical Trial for Patients with Wet AMD: Design and Baseline Characteristics
Phase 1 Dose Exploration Interim Safety Data
Phase 1 Dose Exploration Interim Clinical Activity Data
Phase 1 Dose Exploration Interim Aflibercept Biomarker Data
1) Injection-free at 12 weeks, sample results available at time of data cutoff
Future Directions for 4D-150 & Large Market Ophthalmology Portfolio with the R100 Vector
Webcast Information:
4D Molecular Therapeutics will host a webcast today, Thursday, April 27, 2023 at 8:00 a.m. Eastern Time to discuss clinical data and development plans. Dr. Arshad Khanani will also join today's webcast.
An archived copy of the webcast will be available for up to one year by visiting the “Investors & Media” section of the 4DMT website at https://ir.4dmoleculartherapeutics.com/events.
4D-150 is comprised of our customized and evolved intravitreal vector, R100, and a transgene payload that expresses both aflibercept and a VEGF-C inhibitory RNAi. This dual transgene payload inhibits 4 angiogenic factors that drive wet AMDAMD and DME: VEGF A, B, C and PlGF. R100 was invented at 4DMT through our proprietary Therapeutic Vector Evolution platform; we created this platform utilizing principles of directed evolution, a Nobel Prize-winning technology. 4D-150 is designed for single, low-dose intravitreal delivery.
About Wet AMD and DME
Wet AMD is a highly prevalent disease with estimated incidence rate of 200,000 new patients per year in the United States. Wet AMD is a type of macular degeneration where abnormal blood vessels (choroidal neovascularization or CNV) grow into the macula, the central area of the retina. As a consequence, CNV causes swelling and edema of the retina, bleeding and scarring, and causes visual distortion and reduced acuity. The proliferation and leakage of abnormal blood vessels is stimulated by VEGF. This process distorts and can potentially destroy central vision and may progress to blindness without treatment.
DME is a highly prevalent disease with significant unmet medical need. It is estimated that there are approximately one million individuals with DME in the United States. DME is characterized by swelling in the macula (central retina) due to leakage from blood vessels. This can lead to blurred vision. DME is typically treated with intravitreal anti-VEGF agents administered approximately every 4-12 weeks.
About 4DMT
4DMT is a clinical-stage biotherapeutics company harnessing the power of directed evolution for genetic medicines targeting large market diseases. 4DMT seeks to unlock the full potential of genetic medicines using its proprietary invention platform, Therapeutic Vector Evolution, which 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 product candidates. All of our vectors are proprietary to 4DMT and were invented at 4DMT, including the vectors utilized in our clinical-stage and preclinical pipeline product candidates: R100, A101, and C102. The Company is initially focused on five clinical-stage product candidates in three therapeutic areas for both rare and large market diseases: ophthalmology, pulmonology, and cardiology (Fabry disease cardiomyopathy). The 4DMT customized and evolved vectors were invented with the goal of being delivered at relatively low doses through clinically routine, well-tolerated, and minimally invasive routes of administration, transducing diseased cells in target tissues efficiently, having reduced immunogenicity and, where relevant, having resistance to pre-existing antibodies. 4DMT is currently advancing five product candidates in clinical development: 4D-150 for wet AMDAMD and DME, 4D-710 for cystic fibrosis lung disease, 4D-310 for Fabry disease cardiomyopathy, 4D-125 for XLRP, and 4D-110 for choroideremia. The 4D preclinical product candidates in development are: 4D-175 for geographic atrophy and 4D-725 for AATLD.
4D-150, 4D-710, 4D-310, 4D-125, and 4D-110 are our product candidates in clinical development and have not yet been approved for marketing by the US FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of 4D-150, 4D-710, 4D-310, 4D-125, or 4D-110 for the therapeutic uses for which they are being studied.
SOURCE: 4D Molecular Therapeutics
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