FDMT 4D Molecular Therapeutics Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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4D-110
Choroideremia
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Phase 1
Phase 1
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4D-310
Fabry Disease
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Phase 1/2
Phase 1/2
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
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4D-125
X-linked retinitis pigmentosa (XLRP)
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Phase 1/2
Phase 1/2
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Phase 1/2 initial results showed that dosing was well tolerated in all patients treated to-date (n=8), with no dose-limiting toxicities, no serious adverse events and no chronic inflammation - noted Oct 10, 2021
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4D-710
Cystic fibrosis
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Phase 1/2
Phase 1/2
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Phase 1/2 to be initiated before year-end 2021.
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4D-150
Wet age-related macular degeneration (AMD)
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Phase 1/2
Phase 1/2
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Phase 1/2 trial to be initiated before year-end 2021.
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4D-710
Cystic fibrosis
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Phase 1
Phase 1
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Phase 1 trial to be initiated 4Q 2021.
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Latest News
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- 4D-125 was well tolerated in all patients treated to-date (n=8), with no dose-limiting toxicities, no serious adverse events and no chronic inflammation
- Clinical activity observed through anatomical measurements of reduced photoreceptor loss in treated vs untreated control eyes on ellipsoid zone area endpoints
- Clinical activity observed through functional improvements in treated vs untreated control eyes on two microperimetry endpoints: (1) mean retinal sensitivity and (2) number of loci with >7 dB improvement
- 4DMT plans to continue enrollment at the 1E12 vg/eye in the dose expansion cohort, including in less advanced patients
- 4DMT to host conference call and webcast on Monday, October 11, 2021 at 8:00 a.m EDT
EMERYVILLE, Calif., Oct. 10, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ:FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced interim safety and clinical activity data from the Phase 1/2 clinical trial of intravitreal 4D-125 in patients with advanced X-linked retinitis pigmentosa (XLRP). The interim data were presented today in a late-breaking presentation at the American Society of Retina Specialists (ASRS) 39th Annual Meeting.
"These are the first clinical data reported with a product invented from our Therapeutic Vector Evolution platform at 4DMT, and these interim data demonstrate clinical proof-of-concept for safety, tolerability and clinical activity," said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. "These data support our belief that 4D-125 is well tolerated, and has the potential to both slow the progressive loss of photoreceptors in patients with XLRP after a single intravitreal injection and to improve visual function. We believe these results validate the potential of our platform, and of the R100 vector, which is also deployed in 4DMT targeted product candidates designed to treat a variety of large market diseases. Consistent with our prior guidance, we expect our first R100-based large-market product candidate, 4D-150 for wet AMD, to enter clinical development before year-end."
"Given the encouraging data we have seen with our intravitreal gene therapy, we plan to continue enrolling patients at the top dose level of 1E12 vg/eye in the dose expansion cohort, including treatment of patients with less advanced disease who are evaluable for both anatomical and functional endpoints," said Dr. Robert Kim, M.D., Senior Vice President & Clinical Therapeutic Area Head, Ophthalmology. "We designed 4D-125 to be administered via intravitreal administration, which targets the entire surface of the retina and thereby enables the potential to treat broader patient populations, including early stage patients, more effectively than is feasible with subretinal approaches. In the case of XLRP, we believe that early stage patient populations may have the potential to benefit from our gene therapy even more than advanced patients given our goal of preserving photoreceptors. We plan to explore development paths that include treating both early stage and advanced patient populations."
"XLRP is a slowly progressing inherited retinal dystrophy that leads to vision loss and ultimately blindness," said Dr. Cagri Besirli, M.D., Ph.D., University of Michigan, Kellogg Eye Center and a principal investigator on the 4D-125 Phase 1/2 clinical trial. "There are currently no treatment options available to these XLRP patients. 4DMT's gene therapy is a promising approach because, unlike other gene therapy approaches, it is delivered via intravitreal injection, a routine clinical route of administration that targets the entire surface of the retina. These interim clinical data suggest that 4D-125 is well-tolerated and has the potential to both slow the loss of the photoreceptor ellipsoid zone area and enhance retinal sensitivity."
4D-125 Interim Clinical Data Summary
The data described are from the first-in-human, on-going Phase 1/2 dose escalation and dose expansion clinical trial assessing intravitreal 4D-125, 4DMT's targeted and evolved R100-based product candidate for XLRP. As of the data cutoff date (September 1, 2021), eight patients with clinically advanced XLRP due to RPGR gene mutation had been enrolled. A standard 3+3 dose escalation design was used, followed by a dose expansion cohort. Patients were enrolled in one of three dose cohorts: dose-escalation cohort 1 (3E11 vg/eye; n=3), dose-escalation cohort 2 (1E12 vg/eye n=3) and the dose expansion cohort (1E12vg/eye; n=2 to date). Patients enrolled in the dose escalation cohorts of the first-in-human clinical trial had clinically-advanced XLRP, with patients having limited or no measurable remaining photoreceptor area or retinal sensitivity. As of the data cutoff date, two dose escalation patients (n=1 at 3E11 vg/eye; n=1 at 1E12 vg/eye) were evaluable for clinical activity defined as having both measurable ellipsoid-zone area (EZ Area) by spectral domain optical coherence tomography (SD-OCT) and retinal sensitivity by microperimetry in both the treated and untreated control eye with at least six months follow-up; dose expansion cohort patients (n=2) had not yet reached six months follow-up but are expected to be evaluable with sufficient follow up. On-going enrollment in the dose expansion cohort is expected to enroll patients with less advanced disease than those enrolled in dose escalation, and who we expect to be evaluable for clinical activity based on central reading center confirmation at screening.
Interim Safety Data Summary
- 4D-125 was well-tolerated in all eight XLRP patients, including in five patients at the top dose level of 1E12 vg/eye.
- No dose-limiting toxicities or serious adverse events were observed.
- No chronic inflammation was observed.
- Transient, grade 1+ anterior chamber and/or vitreous cells were observed in two of the eight patients at a single protocol-defined assessment timepoint (SUN1 & NEI2 grading scales)
- One patient had grade 1+ vitreous and anterior chamber cells
- One additional patient had grade 1+ vitreous chamber cells
Interim Clinical Activity Summary
- In the two dose escalation patients who were evaluable for clinical activity in both the treated and untreated control eyes, interim data from both patients demonstrated anatomical retina preservation as measured by EZ Area progression, a measurement of intact photoreceptors, in the treated eye as compared to the untreated control eye in the same patient. In addition, interim data from both patients demonstrated functional improvements as measured by increases in the mean retinal sensitivity in the treated eye, and a greater number of loci gaining ≥ 7 dB sensitivity in the treated eye, as compared to the untreated control eye in the same patient.
- Patient 3 (3E11 vg/eye cohort – 9 months follow-up): Decreases from baseline EZ Area were -12.4% in the treated eye compared to -16.2% in the untreated control eye (~23% lower relative progression rate). An increase in mean retinal sensitivity was demonstrated, with an increase of +1.65 dB in the treated eye from baseline compared to +0.25 dB in the untreated control eye. The number of loci gaining greater than ≥ 7 dB sensitivity were six in the treated eye compared to one in the untreated control eye.
- Patient 5 (1E12 vg/eye cohort – 6 months follow-up): Decreases from baseline EZ Area were -20.2% in the treated eye compared to -28.7% in the untreated control eye (~30% lower relative progression rate). An increase in mean retinal sensitivity was demonstrated, with an increase of +0.90 dB in the treated eye from baseline compared to +0.10* dB in the untreated control eye. The number of loci gaining greater than ≥ 7 dB sensitivity were three in the treated eye compared to zero in the untreated control eye.
*note: microperimetry data for this patient's untreated eye were evaluable at 4 months but not available at month 6 due to an inability to fixate with the untreated control eye at that visit.
- Patient 3 (3E11 vg/eye cohort – 9 months follow-up): Decreases from baseline EZ Area were -12.4% in the treated eye compared to -16.2% in the untreated control eye (~23% lower relative progression rate). An increase in mean retinal sensitivity was demonstrated, with an increase of +1.65 dB in the treated eye from baseline compared to +0.25 dB in the untreated control eye. The number of loci gaining greater than ≥ 7 dB sensitivity were six in the treated eye compared to one in the untreated control eye.
1. Standardization of Uveitis Nomenclature Grading Scheme - SUN Working Group 2005 (Jabs et al., 2005)
2. National Eye Institute Grading System for Vitreous Cells - (Mahendradas, Khanna, Kawali, & Shetty, 2014)
Conference Call and Webcast Information
4DMT will host a live conference call and webcast tomorrow, Monday, October 11, 2021, at 8:00 a.m. EDT. Listeners can access the live webcast by visiting the 4DMT "Investor" section of the 4DMT website at www.4dmoleculartherapeutics.com.
To access the live call by phone, dial (833) 540-1164 (domestic) or (929) 517-0354 (international). The conference ID is 1234677. The recorded webcast will be available for at least two weeks following the call.
About 4DMT
4DMT is a clinical-stage company harnessing the power of directed evolution for targeted gene therapies. 4DMT seeks to unlock the full potential of gene therapy using its platform, Therapeutic Vector Evolution, which combines the power of directed evolution with approximately one billion synthetic capsid sequences to invent evolved vectors for use in targeted gene therapy products. 4DMT is initially focused in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The 4DMT targeted and evolved vectors are invented with the goal of being delivered 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 conducting three clinical trials: 4D-125 is in a Phase 1/2 clinical trial for XLRP patients, 4D-110 is in a Phase 1 clinical trial for choroideremia patients and 4D-310 is in a Phase 1/2 clinical trial for Fabry disease patients.
4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT.
Cautionary Note Regarding 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 plans and timelines for the clinical development of 4D-310, 4D-125, 4D-110, 4D-150 and 4D-710, including the therapeutic potential and clinical benefits thereof; whether 4D-125 has the potential to slow the progressive loss of photoreceptors in patients with XLRP after a single intravitreal injection and to also improve visual function; the timing of 4D-150 entering clinical development; and 4DMT's clinical development plans for 4D-125 . 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, without limitation, risks associated with: the impact of COVID-19 on countries or regions in which we have operations or do business, as well as on the timing and anticipated results of our clinical trials, strategy and future operations; the delay of any current or planned clinical trials for the development of 4D Molecular Therapeutics' drug candidates, the risk that the results of our clinical trials may not be predictive of future results in connection with future clinical trials; 4D Molecular Therapeutics' ability to successfully demonstrate the safety and efficacy of its drug candidates; the timing and outcome of our planned interactions with regulatory authorities; and obtaining, maintaining and protecting our intellectual property. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics' most recent Quarterly Report on Form 10-Q filed on August 12, 2021, 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.
4D-310, 4D-125 and 4D-110 are our product candidates in clinical trials 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-310, 4D-125, or 4D-110 for the therapeutic use for which they are being studied.
Contacts:
Media:
Carolyne Zimmermann
Chief Business Officer
czimmermann@4dmt.comInvestors:
Mike Zanoni
VP, Investor Relations
mzanoni@4dmt.com
- 4D-125 was well tolerated in all patients treated to-date (n=8), with no dose-limiting toxicities, no serious adverse events and no chronic inflammation
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EMERYVILLE, Calif., Oct. 06, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ:FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for 4D-150 for wet age-related macular degeneration (wet AMD). The active IND enables the initiation of 4D-150 Phase 1/2 clinical trial sites, which is expected before year-end.
"4D-150 is a dual-transgene intravitreal gene therapy encompassing the R100 capsid which was invented through Therapeutic Vector Evolution with the goal of robust and safe delivery to all regions and major cell types within the retina at relatively low doses," said David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT. "We believe that 4D-150's design, which targets four distinct angiogenic factors with dual transgenes, has the potential for broad, robust and durable efficacy after a single low dose intravitreal administration in patients with wet AMD. 4D-150 has the potential to be administered at significantly lower doses compared to other intravitreal AAV gene therapy approaches, and 4D-150 clinical development builds on the favorable tolerability profile to date with the same R100 capsid utilized in our 4D-125 X-linked retinitis pigmentosa (XLRP) program at significantly higher doses. While 4D-125 has been well-tolerated at 1E12 vg/eye, we believe 4D-150 has the potential for clinical activity at substantially lower doses."
"4D-150 represents numerous firsts in the AAV gene therapy space and underscores 4DMT's commitment to innovation," said Peter Francis, M.D., Ph.D., Chief Scientific Officer. "4D-150 is the first R100-based product candidate to enter clinical development in a large market disease. In addition, it is not only the first clinical-stage dual-transgene AAV gene therapy utilizing an evolved vector, but also the first vectorized RNAi AAV gene therapy product candidate utilizing an evolved vector to enter clinical development."
The Phase 1/2 clinical trial is a dose-escalation and randomized, controlled, masked expansion trial of intravitreal 4D-150 and is expected to enroll approximately 60 adults with wet AMD. In the dose-escalation phase, multiple dose levels of 4D-150 will be examined in an open-label, 3+3 design with an initial dose of 3E10 vg/eye. In dose expansion, patients (n=50) will be randomized 2:2:1 to receive one of 2 dose levels of 4D-150 (n=20 for each dose level) or aflibercept (n=10). The primary endpoints of the study are safety and tolerability. Secondary endpoints include the number of supplemental aflibercept injections received, and change from baseline in best corrected visual acuity (BCVA) over time.
About 4D-150 and wet AMD
4D-150 is a dual-transgene, intravitreal gene therapy designed to inhibit four distinct VEGF factors and prevent angiogenesis and vascular permeability for the treatment of wet AMD. We believe that targeting four distinct angiogenic factors with dual transgenes in patients with these retinal diseases has the potential for greater efficacy and/or lower required doses versus therapies that target a single VEGF factor, including in patients refractory to currently approved anti-VEGF therapies. Intravitreal delivery of biologics to the eye is routine, and a single dose intravitreal gene therapy that could provide long-term efficacy in patients would be an advantage for patients who struggle with treatment burden and/or treatment resistance. 4D-150 builds on the excellent tolerability to date of the R100 capsid in the 4D-125 XLRP clinical program at doses up to 1E12 vg/eye. In the non-human primate retinal laser-induced CNV model data presented in May 2021 at the annual conference of the American Society of Gene and Cell Therapy, at doses ranging from 1E11 to 1E12 vg/eye, 4D-150 intravitreal injection resulted in 100% suppression of grade 4 angiogenic lesions.
Wet AMD is a type of macular degeneration in which abnormal blood vessels grow into the macula and cause visual distortion, reduced acuity and, in some cases, blindness. The proliferation of abnormal blood vessels in the retina is stimulated by VEGF family members. There are approximately 200,000 new incidences of wet AMD per year in the United States alone. High expression levels of VEGF appear to play a causal role in the symptoms of wet AMD.
About 4DMT
4DMT is a clinical-stage company harnessing the power of directed evolution for targeted gene therapies. 4DMT seeks to unlock the full potential of gene therapy using its platform, Therapeutic Vector Evolution, which combines the power of directed evolution with approximately one billion synthetic capsid sequences to invent evolved vectors for use in targeted gene therapy products. The company is initially focused in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The 4DMT targeted and evolved vectors are invented with the goal of being delivered 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-310 for Fabry disease, 4D-125 for XLRP, 4D-150 for wet AMD, 4D-710 for cystic fibrosis and 4D-110 for choroideremia.
4D-310, 4D-125, 4D-150, 4D-710 and 4D-110 are our product candidates in clinical development and have not yet been approved for marketing by the U.S. FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of 4D-310, 4D-125, 4D-150, 4D-710 or 4D-110 for the therapeutic use 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 press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as "aim," "anticipate," "assume," "believe," "contemplate," "continue," "could," "design," "due," "estimate," "expect," "goal," "intend," "may," "objective," "plan," "positioned," "potential," "predict," "seek," "should," "target," "will," "would" and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. All statements other than statements of historical facts contained in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, statements about: 4D-150's potential as a therapeutic product and the number of patients to enroll in 4D-150's Phase 1/2 dose-escalation and randomized, controlled, masked expansion trial. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: the company's history of net operating losses and limited operating history; the company's ability to obtain necessary capital to fund its clinical programs; the risk and uncertainties inherent in the clinical drug development process; the early stages of clinical development of the company's product candidates and the limited regulatory and clinical experience to date for novel AAV gene therapy product candidates; the effects of COVID-19 or other public health crises on the company's clinical programs and business operations; the company's ability to obtain regulatory approval of and successfully commercialize its product candidates; any undesirable side effects or other properties of the company's product candidates; the company's reliance on third-party suppliers and other service providers; the outcomes of any current or future collaboration and license agreements; and the company's ability to adequately maintain intellectual property rights for its product candidates. These and other risks are described in greater detail under the section titled "Risk Factors" contained in the company's most recent Quarterly Report on Form 10-Q filed as of August 12, 2021, as well as any subsequent filings with the Securities and Exchange Commission. Any forward-looking statements that the company makes in this press release are made pursuant to the Private Securities Litigation Reform Act of 1995, as amended, and speak only as of the date of this press release. Except as required by law, the company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise.
Contacts:
Media:
Carolyne Zimmermann
Chief Business Officer
czimmermann@4dmt.comInvestors:
Mike Zanoni
VP, Investor Relations
mzanoni@4dmt.com
-
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EMERYVILLE, Calif., Oct. 06, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ:FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for 4D-710 for the treatment of patients with cystic fibrosis. The active IND enables the initiation of 4D-710 Phase 1/2 clinical study sites, which is expected before year-end.
"4D-710 is an aerosol-delivered gene therapy that has promise as a mutation agnostic treatment for patients with cystic fibrosis lung disease," said David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT. "4D-710 is designed to express high levels of the cystic fibrosis transmembrane conductance regulator (CFTR) protein directly within target cells lining the airway, enabling CFTR mutation-independent activity, and was also invented for resistance to pre-existing antibodies in humans. We plan to focus initially on the approximately 10-15% of all patients whose disease is not amenable to existing modulator medicines that target the CFTR protein. Ultimately, we believe 4D-710 has the potential to treat a broad cystic fibrosis patient population, including those patients treated with current CFTR modulators, all of which require daily dosing over the patient's lifetime and generally result in only partial correction of lung function."
"4D-710 is comprised of our targeted and evolved vector, A101, and a microCFTR transgene," said Robert Fishman, M.D., Chief Medical Officer of 4DMT. "A101 was invented not only for aerosol delivery diffusely throughout the lung airways and alveoli, but also for penetration through the mucus barrier and for resistance to pre-existing antibodies, both of which are potentially key attributes for successful treatment of these patients. 4D-710 has the potential to be a differentiated therapy for the treatment of cystic fibrosis lung disease due to its potentially corrective mechanism, expected ability to treat patients independent of CFTR mutation and resistance to AAV antibodies."
The Phase 1/2 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion trial of 4D-710 in patients (n=~18) with cystic fibrosis who are ineligible for CFTR modulator therapy or who have discontinued therapy due to adverse effects. The primary endpoint of the study is safety and tolerability. Secondary endpoints include assessments of clinical activity including lung function, plus transgene transfer and microCFTR expression as measured within bronchoscopic biopsies and brushings.
As a result of the IND clearance for 4D-710 and in accordance with the terms of our collaboration with the CF Foundation, CF Foundation will purchase 125,715 shares of our common stock, for aggregate proceeds of approximately $4 million. The proceeds of the CF Foundation equity investment will be used to further support development of 4D-710.
About 4D-710 and Cystic Fibrosis
4D-710 is comprised of our targeted and evolved vector, A101, and a codon-optimized microCFTR transgene. 4D-710 has the potential to treat a broad range of patients with cystic fibrosis lung disease, independent of the specific CFTR mutation, and is designed for efficient aerosol delivery to achieve CFTR expression within lung airway epithelial cells. 4D-710 is being initially developed in the approximately 10-15% of patients whose disease is not amenable to existing medicines targeting the CFTR protein. In patients with CFTR mutations whose disease is amenable to modulator medicines, these modulators do not fully restore normal lung function in most patients. Further, these chronic therapies require daily dosing for the patient's lifetime. We therefore expect to eventually develop 4D-710 in this broader patient population, as a single agent and/or in combination with these CFTR modulator small molecule medicines.
Cystic fibrosis is the most common life-shortening inherited disease in the United States and results from mutations in the CFTR gene. Cystic fibrosis causes impaired lung function, inflammation and bronchiectasis and is commonly associated with persistent lung infections and repeated exacerbations due to the inability to clear thickened mucus from the lungs. These complications result in progressive loss of lung function and hospitalizations, and ultimately lead to end-stage respiratory failure. According to the CF Foundation, more than 30,000 people in the United States and more than 70,000 people worldwide are living with cystic fibrosis, with approximately 1,000 new cases of cystic fibrosis diagnosed in the United States each year. Patients with cystic fibrosis require lifelong treatment with multiple daily medications. Those with end-stage lung disease may undergo lung transplantation.
About 4DMT
4DMT is a clinical-stage company harnessing the power of directed evolution for targeted gene therapies. 4DMT seeks to unlock the full potential of gene therapy using its platform, Therapeutic Vector Evolution, which combines the power of directed evolution with approximately one billion synthetic capsid sequences to invent evolved vectors for use in targeted gene therapy products. The company is initially focused in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The 4DMT targeted and evolved vectors are invented with the goal of being delivered 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-310 for Fabry disease, 4D-125 for XLRP, 4D-150 for wet AMD, 4D-710 for cystic fibrosis and 4D-110 for choroideremia.
4D-310, 4D-125, 4D-150, 4D-710 and 4D-110 are our product candidates in clinical development and have not yet been approved for marketing by the U.S. FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of 4D-310, 4D-125, 4D-150, 4D-710 or 4D-110 for the therapeutic use 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 press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as "aim," "anticipate," "assume," "believe," "contemplate," "continue," "could," "design," "due," "estimate," "expect," "goal," "intend," "may," "objective," "plan," "positioned," "potential," "predict," "seek," "should," "target," "will," "would" and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. All statements other than statements of historical facts contained in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, statements about: 4D-710's potential as a therapeutic product and the number of patients to enroll in 4D-710's Phase 1/2 multicenter, open-label, dose-escalation and dose-expansion trial. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: the company's history of net operating losses and limited operating history; the company's ability to obtain necessary capital to fund its clinical programs; the risk and uncertainties inherent in the clinical drug development process; the early stages of clinical development of the company's product candidates and the limited regulatory and clinical experience to date for novel AAV gene therapy product candidates; the effects of COVID-19 or other public health crises on the company's clinical programs and business operations; the company's ability to obtain regulatory approval of and successfully commercialize its product candidates; any undesirable side effects or other properties of the company's product candidates; the company's reliance on third-party suppliers and other service providers; the outcomes of any current or future collaboration and license agreements; and the company's ability to adequately maintain intellectual property rights for its product candidates. These and other risks are described in greater detail under the section titled "Risk Factors" contained in the company's most recent Quarterly Report on Form 10-Q filed as of August 12, 2021, as well as any subsequent filings with the Securities and Exchange Commission. Any forward-looking statements that the company makes in this press release are made pursuant to the Private Securities Litigation Reform Act of 1995, as amended, and speak only as of the date of this press release. Except as required by law, the company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise.
Contacts:
Media:
Carolyne Zimmermann
Chief Business Officer
czimmermann@4dmt.comInvestors:
Mike Zanoni
VP, Investor Relations
mzanoni@4dmt.com
-
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EMERYVILLE, Calif., Sept. 29, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ:FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that management will participate in Chardan's 5th Annual Genetic Medicines Conference being held virtually on Monday, October 4. Details of the fireside chat are as follows:
Event: Chardan's 5th Annual Genetic Medicines Conference
Date & Time: Monday, October 4, 2021 at 2:30 p.m. ET
Format: Fireside Chat
A live audio webcast of the fireside chat will be available by visiting the "Investors & Media" section of the 4DMT website at www.4dmoleculartherapeutics.com. A replay of the webcast will be available for at least two weeks following the live event.
About 4DMT
4DMT is a clinical-stage company harnessing the power of directed evolution for targeted gene therapies. 4DMT seeks to unlock the full potential of gene therapy using its platform, Therapeutic Vector Evolution, which combines the power of directed evolution with approximately one billion synthetic capsid sequences to invent evolved vectors for use in targeted gene therapy products. The company is initially focused in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The 4DMT targeted and evolved vectors are invented with the goal of being delivered 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 conducting three clinical trials: 4D-125 is in a Phase 1/2 clinical trial for XLRP patients, 4D-110 is in a Phase 1 clinical trial for choroideremia patients and 4D-310 is in a Phase 1/2 clinical trial for Fabry disease patients.
4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT.
4D-310, 4D-125 and 4D-110 are our product candidates in clinical trials 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-310, 4D-125, or 4D-110 for the therapeutic use for which they are being studied.
Contacts:
Media:
Carolyne Zimmermann
Chief Business Officer
czimmermann@4dmt.comInvestors:
Mike Zanoni
VP, Investor Relations
mzanoni@4dmt.com
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EMERYVILLE, Calif., Sept. 24, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ:FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced a late-breaking presentation of preliminary clinical data from the on-going 4D-125 Phase 1/2 trial in patients with X-Linked Retinitis Pigmentosa (XLRP) will be presented at the upcoming Annual Society of Retina Specialists Annual Meeting (ASRS) 2021 that will take place in San Antonio, TX on October 8-12, 2021.
Details of the ASRS presentation are as follows:
Title: Phase 1/2 Clinical Trial of Intravitreal 4D-125 AAV Gene Therapy in Patients with Advanced XLRP: Interim Safety & Preliminary Activity
Speaker: Dr. Cagri G. Besirli M.D., Ph.D, Kellogg Eye Center, University of Michigan
Session: Surgical Techniques & Maneuvers Symposium (Late Breaker Acceptance)
Date and Time: Sunday, October 10, 2:56 p.m. CDT
The presentation will be available for viewing by registered participants during the conference via the ASRS mobile meeting website on October 10, 2021.
About the 4D-125 Phase 1/2 Clinical Trial
4DMT is currently enrolling patients in an on-going Phase 1/2 dose-escalation and dose-expansion clinical trial assessing intravitreal 4D-125, 4DMT's targeted and evolved R100-based product candidate for XLRP. The study employs a standard 3+3 dose-escalation design, followed by dose expansion. Patients are enrolled in one of two dose cohorts: 3E11 vg/eye and 1E12 vg/eye. The dose expansion phase of the study is enrolling patients at the 1E12 vg/eye dose. The primary objectives of this trial are to evaluate the safety and maximum tolerated dose of 4D-125. Secondary endpoints include assessments of clinical activity, including both visual field function and anatomical endpoints.
About XLRP
XLRP is a rare inherited X-linked recessive genetic disorder that causes progressive vision loss and blindness in boys and young men. There are currently no approved therapies for XLRP. Seventy percent of cases are caused by mutations in the retinitis pigmentosa GTPase regulator ("RPGR") gene. The estimated worldwide prevalence of XLRP due to RPGR variants is approximately one in 25,600 people, which represents approximately 24,000 patients in the United States, and France, Germany, Italy, Spain and the United Kingdom (together, EU-5). It is characterized by dysfunction and degeneration of photoreceptors in the retina. Symptoms of XLRP are initially characterized by night blindness, followed by loss of peripheral visual field, decreasing visual acuity and eventually blindness.
About 4DMT
4DMT is a clinical-stage company harnessing the power of directed evolution for targeted gene therapies. 4DMT seeks to unlock the full potential of gene therapy using its platform, Therapeutic Vector Evolution, which combines the power of directed evolution with approximately one billion synthetic capsid sequences to invent evolved vectors for use in targeted gene therapy products. The company is initially focused in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The 4DMT targeted and evolved vectors are invented with the goal of being delivered 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 conducting three clinical trials: 4D-125 is in a Phase 1/2 clinical trial for XLRP patients, 4D-110 is in a Phase 1 clinical trial for choroideremia patients and 4D-310 is in a Phase 1/2 clinical trial for Fabry disease patients.
4D-310, 4D-125 and 4D-110 are our product candidates in clinical trials 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-310, 4D-125, or 4D-110 for the therapeutic use for which they are being studied.
4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT.
Contacts:
Media:
Carolyne Zimmermann
Chief Business Officer
czimmermann@4dmt.comInvestors:
Mike Zanoni
VP, Investor Relations
mzanoni@4dmt.com