4D-150 Phase 2 SPECTRA clinical trial Diabetic Macular Edema Enrollment expected to open in Q3 2023
First Phase 1 PRISM clinical trial with 4D-150 in wet age-related macular degeneration further validates the potential of R100 intravitreal vectors for other large-market eye diseases, including geographic atrophy
Interim PRISM data for dose cohorts 1, 2, and 3 (n=15) will be presented at the 2023 Society for Research in Vision and Ophthalmology (ARVO) Annual Meeting.
The randomized Phase 2 portion of the Phase 1/Phase 2 PRISM clinical trial with 4D-150 in wet AMD is now enrolling.
EMeryville, Calif., Feb. 2, 2023 (GLOBE NEWSWIRE) — 4D Molecular Therapeutics (Nasdaq: FDMT, “4DMT”) is a clinical-stage biopharmaceutical company that harnesses the power of directed evolution of targeted gene medicines. There is an FDA Investigational New Drug Application (IND) for 4D-150, an R100 vector-based intravitreal gene therapy agent for the treatment of patients with diabetic macular edema (DME).
The Phase 2 SPECTRA clinical trial will evaluate 4D-150 in patients with DME. The study design consisted of a dose-confirmation phase followed by a masked dose-expansion phase, in which patients were treated with one of the two dose levels of 4D-150 or aflibercept in a 1:1:1 ratio. Patients will be randomized to receive internal injections (n=54 patients). DME-evaluated doses are expected to be between 6E9 and 3E10 vg/eye. The IND approval will allow the SPECTRA clinical trial facility to start, and 4DMT plans to begin enrollment in the third quarter of 2023.
Initial Cohort 1 data (n=5) from the Phase 1 portion of the Phase 1/Phase 2 PRISM clinical trial with 4D-150 for wet AMD showed a decrease in injection rate. This further validates the potential of our intravitreal R100 vectors for other large market eye diseases such as geographic atrophy.
On January 9, 2023, 4DMT announced that it has initiated the randomized Phase 2 portion of the Phase 1/2 PRISM clinical trial of 4D-150 in patients with wet AMD. This part of the trial is currently enrolling patients. Additionally, the company plans to present interim data from dose cohorts 1, 2, and 3 (n = 15) at his 2023 ARVO Annual Meeting on April 23-27, 2023. At the time of ARVO data release, all patients are expected to have at least 6 months of follow-up after her 4D-150 treatment.
“This is the sixth US IND filed by 4DMT, and all six have been FDA approved. , and the continued excellence of our regulatory team,” said David Kirn, MD, co-founder and CEO of 4DMT. “To initiate enrollment for the Phase 2 portion of his PRISM trial in wet AMD to evaluate the potential of 4D-150 in his DME patients who require frequent anti-VEGF injections and who have a high treatment burden.” I am pleased to
About 4D-150 and Wet AMD and DME
4D-150 consists of a targeted, evolved intravitreal vector R100 and a payload expressing both aflibercept and VEGF-C RNAi. The R100 was invented with his 4DMT through his own Therapeutic Vector Evolution platform. We created this platform using the principle of directed evolution, a Nobel Prize winning technology. This dual transgene payload inhibits four angiogenic factors: VEGF A, B, C, and PlGF. The 4D-150 is designed for single low-dose intravitreal delivery.
Wet AMD is a highly prevalent disease with an estimated incidence of 200,000 new cases annually in the United States, according to published data. Wet AMD is a form of macular degeneration in which abnormal blood vessels (choroidal neovascularization or CNV) grow into the macula, the central region of the retina. As a result, CNV causes retinal swelling and edema, hemorrhage and scarring, leading to visual distortion and loss of vision. Abnormal vascular proliferation and leakage are stimulated by VEGF. This process can distort, destroy central vision, and can lead to blindness if left untreated.
DME is a highly prevalent disease with significant unmet medical need. According to published data, it is estimated that there are approximately 1 million DME patients in the United States. DME is characterized by swelling of the macula due to leakage from blood vessels. This can blur your vision. DME is usually treated with intravitreal anti-VEGF agents given approximately every 4 to 12 weeks.
4DMT is a clinical-stage biopharmaceutical company that harnesses the power of directed evolution of gene-targeted medicines. 4DMT aims to unlock the full potential of gene medicines with its unique invention platform, Therapeutic Vector Evolution. It combines the Nobel Prize-winning technology of directed evolution with approximately one billion synthetic AAV capsid-derived sequences to invent targeted and evolved inventions. Vector for use in product candidates. All of our vectors are proprietary to 4DMT and were invented at 4DMT, including vectors used in our clinical-stage and preclinical pipeline product candidates R100, A101, and C102. The company will initially focus on his five clinical-stage product candidates in his three therapeutic areas of rare and large-market conditions: ophthalmology, pulmonology, and cardiology (Fabry cardiomyopathy). increase. 4DMT-directed evolved vectors are delivered at relatively low doses via a clinically routine, well-tolerated, and minimally invasive route of administration, effectively targeting diseased cells in target tissues. to reduce immunogenicity and, where relevant, resistance to pre-existing antibodies. 4DMT currently has five product candidates in clinical development. 4D-150 for wet AMD and DME, 4D-710 for cystic fibrosis lung disease, 4D-310 for Fabry cardiomyopathy, 4D-125 for XLRP, and 4D-110 for choroidemia. . 4D preclinical product candidates in development are 4D-175 for geographic atrophy and 4D-725 for alpha-1 antitrypsin deficiency.
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 other regulatory agencies. yeah. No statements are made about the safety or efficacy of 4D-150, 4D-710, 4D-310, 4D-125, or 4D-110 for the therapeutic uses for which they are being studied.
4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, implied and express statements regarding the therapeutic potential and clinical benefits of 4D-150. contains a description of Phase 2 SPECTRA clinical trial development and timing. “could”, “could”, “could”, “could”, “could”, “could”, “expect”, “plan ‘, ‘anticipate’, ‘intend’, ‘believe’ and ‘expect’ are used interchangeably with ‘estimate’, ‘seek’, ‘predict’, ‘future’, ‘project’ and ‘potential’. , “continue,” “target,” and similar words or expressions are intended to identify forward-looking statements, not all of which may appear to be forward-looking. Statements contain these identifiers. The forward-looking statements in this press release are based on management’s current expectations and beliefs and many risks, uncertainties and actual events or outcomes are expressed or implied by the forward-looking statements. are subject to important factors that may cause them to differ significantly from those presented. It includes risks and uncertainties that are described in more detail in the section entitled “Risk Factors” of 4D Molecular Therapeutics’ Most Recent Quarterly Report on Form 10-Q. exchange committee. In addition, forward-looking statements represent only 4D Molecular Therapeutics’ views as of today and should not be relied upon as representing views at any later date. 4D Molecular Therapeutics expressly disclaims any obligation to update any forward-looking statements. No representations or warranties (whether express or implied) are made as to the accuracy of such forward-looking statements.
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August J. Moretti
Life Science Advisor