4D Molecular Therapeutics, Inc.
4D-150 Phase 2 SPECTRA objective trial for diabetic macular edema enrollment is expected to initiate successful Q3 2023
Initial Phase 1 PRISM objective proceedings with 4D-150 for bedewed age-related macular degeneration further validates R100 intravitreal vector imaginable for different ample marketplace oculus diseases including geographic atrophy
Interim PRISM information for dose Cohorts 1, 2, & 3 (n=15) to beryllium presented astatine the 2023 Association for Research successful Vision and Ophthalmology (ARVO) Annual Meeting
Randomized Phase 2 information of the Phase 1/2 PRISM objective proceedings with 4D-150 for bedewed AMD is presently enrolling
EMERYVILLE, Calif., Feb. 02, 2023 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, “4DMT”), a clinical-stage biotherapeutics institution harnessing the powerfulness of directed improvement for targeted familial medicines, announced FDA clearance of the Investigational New Drug Application (IND) for 4D-150, an R100 vector-based intravitreal familial medicine, for the attraction of patients with Diabetic Macular Edema (DME).
The Phase 2 SPECTRA objective proceedings volition measure 4D-150 successful patients with DME. The survey plan consists of a Dose Confirmation signifier followed by a masked Dose Expansion stage, successful which patients volition beryllium randomized to person a azygous intravitreal injection astatine 1 of 2 dose levels of 4D-150 oregon aflibercept successful a 1:1:1 ratio (n=54 patients). The doses to beryllium evaluated successful DME are anticipated to beryllium betwixt 6E9 to 3E10 vg/eye. The IND clearance enables the initiation of SPECTRA objective survey sites, and 4DMT expects to statesman enrollment successful the 3rd 4th of 2023.
Initial Cohort 1 information (n=5) from the Phase 1 information of the Phase 1/2 PRISM objective proceedings with 4D-150 for bedewed age-related macular degeneration (wet AMD) demonstrated a simplification successful annualized anti-VEGF injection complaint by implicit 95%, further validating the imaginable of our intravitreal R100 vector for different ample marketplace oculus diseases specified arsenic geographic atrophy.
On January 9, 2023, 4DMT disclosed that the Company had initiated the randomized Phase 2 information of the Phase 1/2 PRISM objective proceedings for 4D-150 successful patients with bedewed AMD. This information of the proceedings is present enrolling patients. In addition, the Company intends to contiguous interim information for dose Cohorts 1, 2, & 3 (n=15) astatine the 2023 ARVO Annual Meeting taking spot April 23-27, 2023; astatine the clip of the ARVO information presentation, each patients are predicted to person astatine slightest six months of follow-up pursuing 4D-150 treatment.
“This is the sixth US IND submitted by 4DMT, and each six person been cleared by the FDA. The robustness and ratio of our merchandise plan and improvement motor are based connected sustained excellence by our manufacturing, preclinical, clinical, and regulatory teams,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “We are excited to person started enrolling the Phase 2 information of our PRISM proceedings successful bedewed AMD, and to measure the imaginable of 4D-150 successful patients with DME who besides necessitate predominant anti-VEGF injections and person a precocious attraction burden.”
About 4D-150 and Wet AMD and DME
4D-150 is comprised of our targeted and evolved intravitreal vector, R100, and a payload that expresses some aflibercept and a VEGF-C RNAi. R100 was invented astatine 4DMT done our proprietary Therapeutic Vector Evolution platform; we created this level utilizing principles of directed evolution, a Nobel Prize-winning technology. This dual transgene payload inhibits 4 angiogenic factors: VEGF A, B, C and PlGF. 4D-150 is designed for a azygous debased dose intravitreal delivery.
Wet AMD is simply a highly prevalent illness with an estimated incidence complaint of 200,000 caller patients per twelvemonth successful the United States, according to published data. Wet AMD is simply a benignant of macular degeneration wherever abnormal humor vessels (choroidal neovascularization oregon CNV) turn into the macula, the cardinal country of the retina. As a consequence, CNV causes swelling and edema of the retina, bleeding and scarring, and causes ocular distortion and reduced acuity. The proliferation and leakage of abnormal humor vessels is stimulated by VEGF. This process distorts and tin perchance destruct cardinal imaginativeness and whitethorn advancement to blindness without treatment.
DME is simply a highly prevalent illness with important unmet aesculapian need. It is estimated that determination are astir 1 cardinal individuals with DME successful the United States according to published data. DME is characterized by swelling successful the macula owed to leakage from humor vessels. This tin pb to blurred vision. DME is typically treated with intravitreal anti-VEGF agents administered astir each 4-12 weeks.
About 4DMT
4DMT is simply a clinical-stage biotherapeutics institution harnessing the powerfulness of directed improvement for targeted familial medicines. 4DMT seeks to unlock the afloat imaginable of familial medicines utilizing its proprietary invention platform, Therapeutic Vector Evolution, which combines the powerfulness of the Nobel Prize-winning technology, directed evolution, with astir 1 cardinal synthetic AAV capsid-derived sequences to invent targeted and evolved vectors for usage successful our merchandise candidates. All of our vectors are proprietary to 4DMT and were invented astatine 4DMT, including the vectors utilized successful our clinical-stage and preclinical pipeline merchandise candidates: R100, A101, and C102. The Company is initially focused connected 5 clinical-stage merchandise candidates successful 3 therapeutic areas for some uncommon and ample marketplace diseases: ophthalmology, pulmonology, and cardiology (Fabry illness cardiomyopathy). The 4DMT targeted and evolved vectors were invented with the extremity of being delivered astatine comparatively debased doses done clinically routine, well-tolerated, and minimally invasive routes of administration, transducing diseased cells successful people tissues efficiently, having reduced immunogenicity and, wherever relevant, having absorption to pre-existing antibodies. 4DMT is presently advancing 5 merchandise candidates successful objective development: 4D-150 for bedewed AMD and DME, 4D-710 for cystic fibrosis lung disease, 4D-310 for Fabry illness cardiomyopathy, 4D-125 for XLRP, and 4D-110 for choroideremia. The 4D preclinical merchandise candidates successful improvement 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 merchandise candidates successful objective improvement and person not yet been approved for selling by the US FDA oregon immoderate different regulatory authority. No practice is made arsenic to the information oregon effectiveness of 4D-150, 4D-710, 4D-310, 4D-125, oregon 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.
Forward Looking Statements
This property merchandise contains forward-looking statements wrong the meaning of the Private Securities Litigation Reform Act of 1995, arsenic amended, including, without limitation, implied and explicit statements regarding the therapeutic imaginable and objective benefits of 4D-150, including plans for the objective improvement and timing of the Phase 2 SPECTRA objective trial. The words "may," “might,” "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," “expect,” "estimate," “seek,” "predict," “future,” "project," "potential," "continue," "target," and akin words oregon expressions are intended to place forward-looking statements, though not each forward-looking statements incorporate these identifying words. Any guardant looking statements successful this property merchandise are based connected management's existent expectations and beliefs and are taxable to a fig of risks, uncertainties, and important factors that whitethorn origin existent events oregon results to disagree materially from those expressed oregon implied by immoderate forward-looking statements contained successful this property release, including risks and uncertainties that are described successful greater item successful the conception entitled "Risk Factors" successful 4D Molecular Therapeutics’ astir caller Quarterly Report connected Form 10-Q, arsenic good arsenic immoderate consequent filings with the Securities and Exchange Commission. In addition, immoderate forward-looking statements correspond 4D Molecular Therapeutics' views lone arsenic of contiguous and should not beryllium relied upon arsenic representing its views arsenic of immoderate consequent date. 4D Molecular Therapeutics explicitly disclaims immoderate work to update immoderate forward-looking statements. No representations oregon warranties (expressed oregon implied) are made astir the accuracy of immoderate specified forward-looking statements.
Contacts:
Media:
Katherine Smith
Evoke Canale
Katherine.Smith@evokegroup.com
Investors:
August J. Moretti
Chief Financial Officer
amoretti@4dmt.com
Irina Koffler
LifeSci Advisors
jkoffler@lifesciadvisors.com
917-734-7387
English (US)