About Us | ScientistFDMT 4D Molecular Therapeutics Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
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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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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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Latest News
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- 4D-110: Initial clinical safety data at both of the two dose levels in the Phase 1 clinical trial indicate that 4D-110 was well-tolerated and did not result in any dose-limiting toxicity (n=6; all patients followed between one and nine months)
- 4D-125: Initial clinical safety data at both of the two dose levels in the Phase 1 portion of a Phase 1/2 clinical trial indicate that 4D-125 was well-tolerated and did not result in any dose-limiting toxicity (n=6; all patients followed between four and nine months)
- 4DMT will regain full-rights to 4D-110 as a result of Roche's termination of the Collaboration and License Agreement under which 4DMT had licensed to Roche certain rights to 4D-110
EMERYVILLE, Calif., June 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 an update on their rare disease ophthalmology product candidate portfolio including Phase 1 dose escalation clinical trial safety and tolerability data with 4D-110 for choroideremia and 4D-125 for X-linked retinitis pigmentosa (XLRP) (n=12 patients total), and that the company received a notice of termination of the Collaboration and License Agreement by 4D-110 licensee Roche resulting in full rights to 4D-110 reverting to 4DMT.
Update on Roche Collaboration and License Agreement
Roche requested that 4DMT conclude the Roche-funded 4D-110 trial in advanced choroideremia patients as a result of Roche's assessment of a change in the risk-benefit profile. Subsequently, Roche sent a notice of termination without cause of the Collaboration and License Agreement, effective as of September 16, 2021. As a result, 4DMT will regain full rights to 4D-110.
4DMT has not changed its position on the potential of 4D-110 for choroideremia, a devastating blinding disease with no approved therapies. Based on the totality of the data generated to date, 4DMT intends to continue clinical development. The company plans to submit to FDA safety and efficacy data from the completed Phase 1 clinical trial along with a new clinical study protocol as soon as possible. 4DMT will conclude the Roche-funded clinical trial under the collaboration and plans to subsequently transfer previously treated patients onto a 4DMT-sponsored long-term follow-up study to continue monitoring biologic activity endpoints, safety and tolerability.
4DMT Rare Disease Ophthalmology Product Candidate Portfolio Update
"We believe the initial clinical tolerability and adverse event profile data in twelve patients from our two intravitreal rare disease ophthalmology clinical trials, performed under 4DMT INDs, demonstrate the potential of our intravitreal product platform. We expect to release initial 4D-110 biologic activity data in the fourth quarter of this year when at least six months of follow-up are available for all currently enrolled patients, and after the 90-day transition period with Roche is completed. We are pleased to regain full rights to our 4D-110 product candidate for choroideremia, and to develop it further within our wholly-owned ophthalmology product portfolio," said David Kirn, M.D., Chief Executive Officer, President and Co-founder. "We would like to thank our Roche colleagues for a highly productive collaboration and funding support for the 4D-110 choroideremia program. The 4D-110 program would not be where it is today without their contributions and outstanding commitment supporting the development of innovative new therapies for ophthalmology patients. Patients with these diseases, many of whom are children, are all eventually blinded by these devastating diseases that have no approved therapies."
"We are pleased to have completed all Phase 1 dose escalation trial enrollment on the Roche-funded 4D-110 clinical trial. We plan to conclude the Roche-funded clinical trial under the collaboration and subsequently transfer previously treated patients onto a long-term follow-up study to continue monitoring biological activity endpoints and safety," said Robert Kim, M.D., Senior Vice President of Clinical Research, Head of Clinical Ophthalmology at 4DMT. "We are committed to designing and initiating the next 4D-110 clinical trial, including treatment of earlier-stage patients, as soon as possible after reviewing the clinical data with our investigators and the FDA."
Initial Phase 1 Dose Escalation Safety and Tolerability Data Summary: 4D-110 for choroideremia and 4D-125 for X-linked retinitis pigmentosa
Clinical trial designs and enrollment
Both clinical trials employed standard "3+3" dose-escalation designed to assess the safety, tolerability and biologic activity of a single intravitreal injection of either 4D-110 or 4D-125 at two dose levels (3E11 or 1E12 vg/eye). A total of twelve patients were enrolled across dose escalation cohorts, including six who received 4D-110 (three at each dose level) and six who received 4D-125 (three at each dose level). Patients received a standard immunosuppression regimen with taper; adjustments were determined by investigators. The results described today are based on data cut-offs as of April 12, 2021 for 4D-110 and April 27, 2021 for 4D-125.
Initial Tolerability and Adverse Event Profile
4D-110 and 4D-125 were both well-tolerated as outlined in the treatment-emergent adverse event (AE) summary table below:
4D-110 4D-125 Total Patient # enrolled 6 6 12 Doses 3E11 or 1E12 vg/eye 3E11 or 1E12 vg/eye - Follow-up at data cut-off (months) 1-9 months 4-9 months - Dose-Limiting Toxicities (DLTs) 0 (0%) 0 (0%) 0 (0%) Serious AE 0 (0%) 0 (0%) 0 (0%) Any CTCAE Grade ≥ 3 0 (0%) 0 (0%) 0 (0%) Retinal AE (Any Grade) 0 (0%) 0 (0%) 0 (0%) Uveitis CTCAE Grade 2 (moderate) 1/6 (17%) 1/6 (17%) 2/12 (17%) Uveitis CTCAE Grade 1 (mild) 4/6 (67%) 2/6 (33%) 6/12 (50%) Expected Upcoming Milestones
- 4D-125: Initial biologic activity data from the Phase 1/2 clinical trial of 4D-125 in XLRP are expected in the fourth quarter of 2021, following at least nine months follow-up for all currently enrolled patients.
- 4D-110: Initial biologic activity data from the Phase 1 clinical trial of 4D-110 in choroideremia are expected in the fourth quarter of 2021, following at least six months follow-up for all currently enrolled patients and completion of the 90-day transition period with Roche. Additionally, after regaining full-rights to 4D-110, 4DMT plans to submit to FDA safety and efficacy data along with a new clinical study protocol, and to initiate a new clinical trial as soon as possible, that enrolls earlier stage patient populations.
- 4D-310: Initial clinical data from the Phase 1/2 clinical trial of 4D-310 in Fabry disease are expected in the second half of 2021.
- 4D-150: Initiation of a clinical trial with 4D-150 in wet AMD and diabetic macular edema is expected in the fourth quarter of 2021.
- 4D-710: Initiation of a clinical trial with 4D-710 in cystic fibrosis lung disease is expected in the fourth quarter of 2021.
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.
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; the estimated timing of clinical data being available for 4D-125's Phase 1/2 clinical trial, 4D-110's Phase 1 trial and 4D-310's Phase 1/2 clinical trial; the estimated timing of initiating the clinical trials for 4D-150 and 4D-710, and the estimated timing of initiating the next clinical trial for 4D-110; expectations regarding current and future interactions with the U.S. Food and Drug Administration (FDA); the estimated next steps in the development of 4D-110; 4D Molecular Therapeutics' ability to demonstrate the potential of its intravitreal product platform; the timing and whether 4D Molecular Therapeutics regains the rights to 4D-110 under the Roche Agreement; and 4D Molecular Therapeutics' strategy, business plans and focus. 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, including any initial data therefrom, may not be predictive of future clinical trial results, including those from current and 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 May 13, 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
[email protected]Investors:
Mike Zanoni
Endurance Advisors
[email protected]
- 4D-110: Initial clinical safety data at both of the two dose levels in the Phase 1 clinical trial indicate that 4D-110 was well-tolerated and did not result in any dose-limiting toxicity (n=6; all patients followed between one and nine months)
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EMERYVILLE, Calif., June 07, 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 the appointment of Carolyne Zimmermann as Chief Business Officer. Ms. Zimmermann brings nearly 20 years of leadership experience in life sciences corporate and business development from her prior roles at Johnson & Johnson Innovation and Novartis Pharmaceuticals.
"Carolyne's extensive experience in biotechnology corporate and business development, external innovation, pre-commercial planning, product pipeline strategy and venture investing brings valuable skillsets to 4DMT," said David Kirn, M.D., Co-founder, President and Chief Executive Officer of 4DMT. "Her appointment reflects our commitment to unlocking the full potential of our Therapeutic Vector Evolution gene therapy platform, and further empowers the company to achieve its goal of becoming a fully integrated biopharmaceutical leader in gene therapy. Carolyne will play a major role in realizing our vision of converting the power of our directed evolution platform into potentially transformative products for patients."
Ms. Zimmermann joins 4DMT from Johnson & Johnson Innovation where she served as Vice President, Transactions. In this role, she led the transaction team on all deal-related matters, including sourcing and executing transactions as well as supporting J&J's Lung Cancer Initiative's early innovation deal making efforts. Earlier in her career, she held leadership roles in the Global Business Development & Licensing group at Novartis Pharmaceuticals for over 13 years, where she gained extensive experience in structuring and negotiating strategic transactions with both pharmaceutical and biotechnology entities, including collaborations, licensing deals, acquisitions, and equity investments. At Novartis, she led the Global Cardio-Metabolic Franchise's Business Development and Licensing efforts, where she was responsible for driving the external strategy and securing strategic assets to expand the portfolio. Previously, she was also General Partner at dRx Capital, a $100mn venture fund founded in 2015 by Novartis and Qualcomm Ventures focused on early stage digital health investing.
Carolyne earned her B.S. in Engineering Sciences from the University of California, San Diego and her M.B.A. from Columbia Business School, Columbia University.
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.
Contacts:
Media:
Theresa Janke
[email protected]Investors:
Mike Zanoni
Endurance Advisors
[email protected] -
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EMERYVILLE, Calif., June 03, 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 the Goldman Sachs 42nd Annual Global Healthcare Conference on Tuesday, June 8 at 2:30 p.m. PT.
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.
Contacts:
Media:
Theresa Janke
[email protected]Investors:
Mike Zanoni
Endurance Advisors
[email protected] -
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EMERYVILLE, Calif., May 13, 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 financial results for the first quarter of 2021, and provided operational highlights.
"We continue to relentlessly execute and innovate as demonstrated by achievements in our first full quarter as a public company," said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. "The company remains on track to announce initial clinical data from both our 4D-310 Fabry disease product candidate and our 4D-125 XLRP product candidate in the second half of this year. In addition, we remain on track to initiate clinical trials in the second half of this year for 4D-150, our wet AMD and DME product candidate, and for 4D-710, our cystic fibrosis lung disease product candidate. We also recently expanded our technology platform to include applications of machine learning, and yesterday, at the annual ASGCT conference, we presented preclinical non-human primate data from 4D-150."
Recent Operational Highlights
- Presented preclinical data from non-human primate (NHP) studies at the American Society for Gene and Cell Therapy (ASGCT) 24th Annual Meeting. For the first-time, 4DMT described the design of 4D-150, a dual transgene, intravitreal gene therapy designed to inhibit four distinct VEGF family members for the treatment of wet age-related macular degeneration (AMD) and diabetic macular edema (DME). Preclinical NHP studies demonstrated significant efficacy in the laser-induced choroidal neovascularization (CNV) model, including complete CNV suppression at the lowest dose of 1E11 vg/eye. In addition, a preclinical acute biodistribution study demonstrated high anti-VEGF levels within the NHP eye, with no evidence of uveitis or retinal abnormality.
- Entered into a collaboration focused on applying machine learning technology to the AAV vector capsid datasets generated from 4DMT's Therapeutic Vector Evolution platform. This research will be conducted with U.C. Berkeley investigators Jennifer Listgarten, Ph.D and David Schaffer, Ph.D., global leaders in machine learning, computational biology, AAV directed evolution and gene therapy.
Expected Upcoming Milestones
- Initial clinical data from the Phase 1/2 clinical trial of 4D-310 in Fabry disease expected in the second half of 2021
- Initial clinical data from the Phase 1/2 clinical trial of 4D-125 in X-Linked Retinitis Pigmentosa (XLRP) expected in the second half of 2021
- Initiation of a clinical trial with 4D-150 in wet AMD and diabetic macular edema expected in the second half of 2021
- Initiation of a clinical trial with 4D-710 in cystic fibrosis lung disease expected in the second half of 2021
Financial Results for the First Quarter Ended March 31, 2021
Cash and Cash Equivalents: Cash and cash equivalents were $259.9 million as of March 31, 2021. We expect cash and cash equivalents to be sufficient to fund operations into mid-2023.
Revenue: Total revenue was $2.0 million for the quarter ended March 31, 2021, as compared to $3.5 million for the quarter ended March 31, 2020. The decrease was primarily driven by decreased revenue recognized under the Roche collaboration agreement.
R&D Expenses: Research and development expenses were $12.8 million for the quarter ended March 31, 2021, as compared to $13.2 million for the quarter ended March 31, 2020. This decrease was primarily driven by decreased external manufacturing expense, which was partially offset by higher payroll and stock-based compensation expense.
G&A Expenses: General and administrative expenses were $5.5 million for the quarter ended March 31, 2021, as compared to $3.7 million for the quarter ended March 31, 2020. This increase was primarily due to higher payroll and stock-based compensation expense and higher business insurance expense.
Net Loss: Net loss was $16.4 million for the quarter ended March 31, 2021, as compared to $13.2 million for the quarter ended March 31, 2020.
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.
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; the estimated timing of clinical data being available for 4D-125's Phase 1/2 clinical trial and 4D-310's Phase 1/2 clinical trial; the estimated timing of initiating the clinical trials for 4D-150 and 4D-710; expectations on how long our cash and cash equivalents can fund operations; expectations regarding current and future interactions with the U.S. Food and Drug Administration (FDA); and 4D Molecular Therapeutics' strategy, business plans and focus. 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 to be filed on or about the date hereof, 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.
4D Molecular Therapeutics, Inc.
Condensed Statements of Operations and Comprehensive Loss
(Unaudited)
(in thousands, except share and per share amounts)Three Months Ended
March 31,2021 2020 Revenue: Collaboration and license revenue $ 2,000 $ 3,411 Collaboration and license revenue, related parties — 124 Total revenue 2,000 3,535 Operating expenses: Research and development 12,769 13,158 General and administrative 5,543 3,654 Total operating expenses 18,312 16,812 Loss from operations (16,312 ) (13,277 ) Other income (expense) (94 ) 117 Net loss and comprehensive loss $ (16,406 ) $ (13,160 ) Net loss per share attributable to common stockholders, basic and diluted $ (0.61 ) $ (2.54 ) Weighted-average shares outstanding used in computing net loss per share attributable to common stockholders, basic and diluted 26,690,167 5,183,845 4D Molecular Therapeutics, Inc.
Condensed Balance Sheet Data
(Unaudited)
(in thousands)March 31,
2021December 31,
2020Cash and cash equivalents $ 259,865 $ 276,726 Working capital 250,585 265,912 Total assets 270,114 288,331 Accumulated deficit (152,085 ) (135,679 ) Total stockholders' equity 242,431 256,387 Contacts:
Media:
Theresa Janke
[email protected]Investors:
Mike Zanoni
Endurance Advisors
[email protected] -
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- 4D-150 designed as an intravitreal gene therapy with dual transgenes expressing aflibercept and VEGF-C RNAi for the treatment of wet AMD and DME
- Preclinical NHP studies of 4D-150 demonstrated significant efficacy in the laser-induced choroidal neovascularization (CNV) model, including complete suppression of CNV lesions at the lowest dose of 1E11 vg /eye
- Preclinical acute biodistribution study demonstrated high anti-VEGF levels within the NHP eye with no evidence of uveitis or retinal abnormalities
EMERYVILLE, Calif., May 12, 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 new preclinical data from non-human primate (NHP) studies of 4D-150, a dual transgene, intravitreal gene therapy inhibiting four distinct VEGF family members for the treatment of wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME).
The data are being presented today in an oral presentation by Peter Francis, M.D., Ph.D., Chief Scientific Officer of 4DMT, at the American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting.
Highlights from the oral presentation include:
- For the first time, 4DMT described the design of 4D-150: a dual transgene, intravitreal gene therapy inhibiting four distinct VEGF family members for the treatment of wet AMD and DME. The combination of transgenes independently encoding for VEGF-C RNAi and for aflibercept provides a multi-mechanistic approach to inhibiting angiogenesis.
- In the NHP laser-induced CNV model, a single intravitreal injection of 4D-150 resulted in 100% suppression of CNV lesions 4-weeks after laser administration, the primary endpoint of the study, including at the lowest dose tested of 1E11 vg/eye; no uveitis or retinal abnormalities were reported at this 1E11 vg/eye dose level.
- In an acute biodistribution study of 4D-150 in NHP, a single intravitreal injection resulted in both high levels of ocular aflibercept expression and VEGF-C miRNA expression within the retina at 4 weeks, with no evidence of uveitis or retinal abnormalities observed.
- A single intravitreal injection of a 4D-150 prototype at two dose-levels (1E11 & 1E12 vg/eye) resulted in sustained, durable ocular anti-VEGF expression through 12 months in the NHP laser-induced CNV model.
4D-150 Oral Presentation at the ASGCT 24th Annual Meeting
Title: A Multi-Mechanistic Anti-Angiogenic AAV Gene Therapy Product Candidate, 4D-150, for the Treatment of Wet Age-Related Macular Degeneration (wAMD) and Diabetic Macular Edema (DME): Intravitreal Biodistribution, Transgene Expression, Safety and Efficacy in Non-Human Primates
Session Date/Time: Wednesday May 12, 2021 5:30 PM - 7:15 PM EDT
Session Title: AAV Biology, Engineering, Immunology and Animal Modeling
Abstract number: 64
About wet AMD, DME and 4D-150
Wet AMD is a type of macular degeneration where abnormal blood vessels (CNV) grow into the macula and cause visual distortion and reduced acuity. The proliferation of abnormal blood vessels in the retina is stimulated by VEGF. There are on average 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.
Diabetic eye disease is a leading cause of vision loss and blindness in working-age adults and often occurs due to the development of DME. The prevalence of DME is high, affecting approximately 1.1 million adults in the United States.
4D-150 is designed as a dual transgene, intravitreal gene therapy inhibiting four distinct VEGF family members to prevent angiogenesis for the treatment of wet AMD and DME. 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 a single anti-VEGF therapy, including in patients with resistance to currently approved anti-VEGF therapies. Intravitreal delivery of biologics to the eye is routine, and therefore would be an advantage for a single dose therapy that could provide long-term efficacy in patients for whom compliance, or treatment resistance, is a problem.
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.
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 4DMT's approach with 4D-150 has the potential for greater efficacy versus a single anti-VEGF therapy, including for patients with resistance to currently approved anti-VEGF therapies; whether intravitreal delivery of biologics to the eye would be an advantage and whether it could provide long-term efficacy in patients for whom compliance, or treatment resistance, is a problem;and 4DMT's strategy, business plans and focus. 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 4DMT has operations or does business, as well as on the timing and anticipated results of its clinical trials, strategy and future operations; the delay of any current or planned clinical trials for the development of 4DMT's drug candidates, the risk that the results of its clinical trials may not be predictive of future results in connection with future clinical trials; 4DMT's 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 4DMT's most recent Annual Report on Form 10-K that was filed on March 25, 2021, as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4DMT's' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4DMT 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.
Contacts:
Media:
Theresa Janke
[email protected]Investors:
Mike Zanoni
Endurance Advisors
[email protected]