1. EMERYVILLE, Calif., Aug. 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 financial results for the second quarter of 2021, and provided operational highlights.

    "Harnessing the power of directed evolution to develop targeted gene therapies is central to 4DMT, and this past quarter we continued to make substantial progress towards our goal," said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. "Importantly, we initiated the expansion of our cGMP manufacturing facilities to support commercial-scale production of our clinical product candidates. Notably, we believe we are the first AAV…

    EMERYVILLE, Calif., Aug. 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 financial results for the second quarter of 2021, and provided operational highlights.

    "Harnessing the power of directed evolution to develop targeted gene therapies is central to 4DMT, and this past quarter we continued to make substantial progress towards our goal," said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. "Importantly, we initiated the expansion of our cGMP manufacturing facilities to support commercial-scale production of our clinical product candidates. Notably, we believe we are the first AAV gene therapy company to successfully complete cGMP manufacturing of primate-evolved targeted vectors, and we achieved this milestone with three different clinical product candidates. This achievement underscores the promise of our Therapeutic Vector Evolution platform, which empowers us to invent targeted and evolved vectors that package efficiently during our proprietary manufacturing process. In addition, we enhanced our leadership team with the additions of Carolyne Zimmermann as Chief Business Officer and Nadine Greiner, Ph.D., as Chief Human Resources Officer. We remain on track to announce clinical data from each of our three ongoing clinical programs and to initiate clinical development with product candidates in wet AMD and cystic fibrosis by the end of this year."

    Recent Operational Highlights

    • Initiated the expansion of current Good Manufacturing Practices (cGMP) compliant manufacturing facilities and analytical laboratories at 4DMT headquarters in Emeryville, CA to support manufacturing and analytical testing of current and future product candidates. The expanded facility will enable commercial-scale manufacturing of our clinical product candidates and expansion of our analytical development capabilities, including laboratories dedicated for developing potency assays.
    • Appointed Carolyne Zimmermann as Chief Business Officer. Ms. Zimmermann brings over 27 years of leadership experience in life sciences corporate and business development, including from her prior roles at Johnson & Johnson Innovation and Novartis Pharmaceuticals.
    • Appointed Nadine Greiner, Ph.D. as Chief Human Resources Officer. Ms. Greiner has served as head of human resources for 25 years at organizations including California Pacific Medical Center, the Palo Alto Medical Foundation at Sutter Health, The Institute on Aging, and Bank of America. She holds a dual Ph.D. in Organization Development and Clinical Psychology.
    • Updated our rare disease ophthalmologic portfolio: reported positive initial safety data from the Phase 1 clinical trial of 4D-110 in choroideremia and the Phase 1/2 clinical trial of 4D-125 in X-linked retinitis pigmentosa (XLRP) and regained global rights to 4D-110. We expect to report initial clinical activity data from the Phase 1 clinical trial of 4D-110 in choroideremia and the Phase 1/2 clinical trial of 4D-125 in XLRP in the fourth quarter of 2021. We also plan to submit to the FDA safety and efficacy data from the 4D-110 Phase 1 clinical trial along with a new 4D-110 clinical study protocol in the fourth quarter of 2021.

    Expected Upcoming Milestones

    • Initial clinical data from the Phase 1/2 clinical trial of 4D-310 in Fabry disease expected in the fourth quarter of 2021
    • Initial clinical activity data from the Phase 1/2 clinical trial of 4D-125 in XLRP expected in the fourth quarter of 2021
    • Initial clinical activity data from the Phase 1 clinical trial of 4D-110 in choroideremia expected in the fourth quarter of 2021
    • Initiation of a clinical trial with 4D-150 in wet AMD expected in the fourth quarter of 2021
    • Initiation of a clinical trial with 4D-710 in cystic fibrosis lung disease expected in the fourth quarter of 2021

    Financial Results for the Second Quarter Ended June 30, 2021

    Cash and Cash Equivalents: Cash and cash equivalents were $243.7 million as of June 30, 2021. We expect cash and cash equivalents to be sufficient to fund operations into the second half of 2023.

    Revenue: Total revenue was $14.6 million for the quarter ended June 30, 2021, as compared to $3.6 million for the quarter ended June 30, 2020. The increase was primarily driven by increased revenue recognized under the Roche collaboration agreement as a result of adjustments to the transaction price and total budgeted costs due to the termination of the agreement which will become effective in the third quarter of 2021.   

    R&D Expenses: Research and development expenses were $15.2 million for the quarter ended June 30, 2021, as compared to $15.7 million for the quarter ended June 30, 2020. This decrease was primarily driven by decreased external manufacturing expense, which was partially offset by increased preclinical and clinical trial expense and payroll and stock-based compensation expense.

    G&A Expenses: General and administrative expenses were $7.0 million for the quarter ended June 30, 2021, as compared to $3.1 million for the quarter ended June 30, 2020. This increase was primarily due to increased payroll and stock-based compensation expense, business insurance expense and professional service expenses.

    Net Loss: Net loss was $7.6 million for the quarter ended June 30, 2021, as compared to $15.2 million for the quarter ended June 30, 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 initial clinical activity data being available for 4D-125's Phase 1/2 clinical trial, initial clinical activity data being available for 4D-110's Phase 1 clinical trial and initial clinical data for 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; whether the expansion of our manufacturing facilities will support commercial-scale production and expand our analytical development capabilities; 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 Statement of Operations and Comprehensive Loss
    (Unaudited)
    (in thousands, except share and per share amounts)
      Three Months Ended

    June 30,
     Six Months Ended

    June 30,
      2021 2020 2021 2020
    Revenue:                
    Collaboration and license revenue $14,580  $3,508  $16,580  $6,919 
    Collaboration and license revenue, related parties     125      249 
    Total revenue  14,580   3,633   16,580   7,168 
    Operating expenses:                
    Research and development  15,223   15,720   27,992   28,878 
    General and administrative  6,953   3,062   12,496   6,716 
    Total operating expenses  22,176   18,782   40,488   35,594 
    Loss from operations  (7,596)  (15,149)  (23,908)  (28,426)
    Other income (expense):  7   (4)  (87)  113 
    Net loss and comprehensive loss $(7,589) $(15,153) $(23,995) $(28,313)
    Net loss per share attributable to common stockholders, basic and

    diluted
     $(0.28) $(2.92) $(0.90) $(5.46)
    Weighted-average shares outstanding used in computing net loss

    per share attributable to common stockholders, basic and diluted
      26,739,149   5,183,955   26,715,014   5,183,900 



    4D Molecular Therapeutics, Inc.
    Condensed Balance Sheet Data
    (Unaudited)
    (in thousands)
      June 30, December 31,
      2021 2020
    Cash and cash equivalents $243,743  $276,726 
    Working capital  238,882   265,912 
    Total assets  254,934   288,331 
    Accumulated deficit  (159,674)  (135,679)
    Total stockholders' equity  240,646   256,387 

    Contacts:

    Media:

    Carolyne Zimmermann

    Chief Business Officer

    czimmermann@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article
    • 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…

    • 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-1104D-125Total
    Patient # enrolled6612
    Doses3E11 or 1E12 vg/eye3E11 or 1E12 vg/eye-
    Follow-up at data cut-off (months)1-9 months4-9 months-
    Dose-Limiting Toxicities (DLTs)0 (0%) 0 (0%)0 (0%)
    Serious AE0 (0%)0 (0%)0 (0%)
    Any CTCAE Grade ≥ 30 (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

    czimmermann@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article
  2. 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…

    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

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article
  3. 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…

    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

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article
  4. 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…

    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,

    2021
      December 31,

    2020
    Cash 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

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article
    • 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…

    • 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

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com

     



    Primary Logo

    View Full Article Hide Full Article
  5. EMERYVILLE, Calif., May 04, 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 new collaboration with investigators at the University of California, Berkeley focused on expanding the vector invention power of 4DMT's Therapeutic Vector Evolution platform by applying machine learning technology to the AAV vector capsid datasets generated from 4DMT's platform. This research will be conducted with Jennifer Listgarten, Ph.D., a global leader in machine learning and computational biology, and David Schaffer, Ph.D., a global leader in AAV directed evolution and gene therapy. Dr. Listgarten is a Professor in U.C…

    EMERYVILLE, Calif., May 04, 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 new collaboration with investigators at the University of California, Berkeley focused on expanding the vector invention power of 4DMT's Therapeutic Vector Evolution platform by applying machine learning technology to the AAV vector capsid datasets generated from 4DMT's platform. This research will be conducted with Jennifer Listgarten, Ph.D., a global leader in machine learning and computational biology, and David Schaffer, Ph.D., a global leader in AAV directed evolution and gene therapy. Dr. Listgarten is a Professor in U.C. Berkeley's Center for Computational Biology, the Berkeley Artificial Intelligence Research Lab and the Chan Zuckerberg Biohub. Prior to her current positions, she held leadership roles in machine learning within Microsoft Research for approximately 10 years. Dr. Schaffer is Professor of Chemical and Biomolecular Engineering, Molecular and Cell Biology and the Helen Wills Neuroscience Institute at U.C. Berkeley. He is also Co-founder, Director and Chief Scientific Advisor at 4DMT.

    "This cross-functional collaboration with world leaders in machine learning and AAV gene therapy technologies gives us the potential to dramatically expand the depth and breadth of targeted and evolved vectors invented through our Therapeutic Vector Evolution platform, significantly expanding our leading vector patent and product portfolios. 4DMT's industry-leading AAV capsid libraries encompass over one billion synthetic capsid sequences. To our knowledge, we have the largest capsid biodistribution datasets in the world as a result of the over 15 vector selection programs we have conducted in non-human primates," said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. "This partnership with Drs. Listgarten and Schaffer empowers 4DMT to dramatically expand our bioinformatics capabilities, enabling us to identify even more promising vectors both within, and even beyond, our existing libraries. This partnership reaffirms our commitment to relentless innovation in order to bring cures to patients."

    Therapeutic Vector Evolution combines the power of directed evolution with approximately one billion synthetic capsid sequences to invent evolved vectors for use in targeted gene therapy products. Using its proprietary Therapeutic Vector Evolution platform, to date 4DMT has generated an industry-leading 40 distinct capsid libraries, conducted more than 15 vector selections in non-human primates, and has filed patent applications on over 300 novel AAV vectors. Three of these proprietary vectors were used in clinical and/or IND product candidates in ophthalmology (4D-150, 4D-125, 4D-110), cardiology (4D-310) and lung (4D-710). Through these 15 vector selections, 4DMT has generated numerous industry leading and expansive datasets relating to capsid biodistribution and delivery to targeted tissues through routine routes of administration, efficient transduction of target cells, and/or resistance to neutralizing antibodies. The application of machine learning to 4DMT's Therapeutic Vector Evolution and its datasets represents an opportunity to leverage an enabling technology to invent new vectors for use in targeted gene therapy products.

    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; 4DMT's potential to dramatically expand the depth and breadth of the proprietary optimized vectors invented through its Therapeutic Vector Evolution platform and its ability to expand its vector patent portfolio; whether 4DMT's machine learning collaboration with U.C. Berkeley will expand its bioinformatics capabilities and whether 4DMT will be able to identify additional promising synthetic capsid sequences within its existing libraries; whether 4DMT's machine learning collaboration with U.C. Berkeley could drive additional value and result in the discovery of novel new capsids; 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.

    4D-310, 4D-125 and 4D-110 are 4DMT's 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:

    Theresa Janke

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article
  6. EMERYVILLE, Calif., April 30, 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 present at the BofA Securities 2021 Virtual Health Care Conference on Thursday, May 13 at 2:00 p.m. PT.

    A live audio webcast of the presentation 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…

    EMERYVILLE, Calif., April 30, 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 present at the BofA Securities 2021 Virtual Health Care Conference on Thursday, May 13 at 2:00 p.m. PT.

    A live audio webcast of the presentation 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

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article
  7. Abstracts on 4D-150 for wet AMD/DME and 4D-310 for Fabry disease accepted for oral presentations at ASGCT 24th Annual Meeting

    Abstract on 4D-710 for cystic fibrosis lung disease accepted for oral presentation at ATS 2021 International Conference

    EMERYVILLE, Calif., April 27, 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 preclinical data presentations from non-human primate (NHP) studies at two upcoming medical meetings, the American Society for Gene and Cell Therapy (ASGCT) 24th Annual Meeting, held virtually from May 11 – 14 and the American Thoracic Society (ATS) 2021 International Conference…

    Abstracts on 4D-150 for wet AMD/DME and 4D-310 for Fabry disease accepted for oral presentations at ASGCT 24th Annual Meeting

    Abstract on 4D-710 for cystic fibrosis lung disease accepted for oral presentation at ATS 2021 International Conference

    EMERYVILLE, Calif., April 27, 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 preclinical data presentations from non-human primate (NHP) studies at two upcoming medical meetings, the American Society for Gene and Cell Therapy (ASGCT) 24th Annual Meeting, held virtually from May 11 – 14 and the American Thoracic Society (ATS) 2021 International Conference, held virtually from May 14 – 19. The data presentations will address three of the company's wholly-owned product candidates leveraging three distinct targeted and evolved vectors across three therapeutic areas: 4D-150 for the treatment of wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME), 4D-310 for the treatment of Fabry disease and 4D-710 for the treatment of cystic fibrosis lung disease.

    For the first-time, 4DMT will describe the design of 4D-150's three distinct anti-angiogenic mechanisms and will present preclinical data demonstrating highly significant efficacy, durable aqueous anti-VEGF levels within the NHP eye, safety and tolerability in the laser-choroidal neovascularization (CNV) model.

    Details for the presentations at each conference are as follows:

    ASGCT 24th Annual Meeting

    Abstracts are available online and presentations and posters will be accessible through ASGCT's website at www.asgct.org.

    Abstract 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 Type: Oral Presentation

    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

    Abstract Title: A Targeted AAV Gene Therapy Product Candidate, 4D-310, for the Treatment of Fabry Disease: Intravenous Biodistribution, Transgene Expression and Safety in Non-Human Primates

    Session Type: Oral Presentation

    Session Date/Time: Friday May 14, 2021 12:15 PM - 2:00 PM EDT

    Session Title: Gene Therapy for Lysosomal Storage Disorders

    Abstract number: 220

    ATS 2021 Annual Conference

    Abstracts are available online and presentations and posters will be accessible through ATS's website at conference.thoracic.org.

    Abstract Title: Identification and Characterization of a Novel AAV Capsid and Product for the Treatment of Cystic Fibrosis

    Session Type: Mini Symposium (Oral)

    Session Date/Time: Sunday May 16, 2021 1:30 PM - 3:00 PM EDT

    Session Title: A010 Novel Discoveries in Lung Biology

    Abstract number: A1043

    Abstract Title: Development of Novel AAV-Based Gene Therapy for Cardiac Disease

    Session Type: Thematic Poster Session

    Session Date/Time: On Demand

    Session Title: TP083 Hey Jude – Novel Findings from Omic and Bioinformatic approaches in Pulmonary Hypertension

    Abstract Number: A3624

    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; 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 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 4DMT's drug candidates, the risk that the results of our 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 filed 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.

    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:

    Theresa Janke

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com

     



    Primary Logo

    View Full Article Hide Full Article
  8. SAN DIEGO, April 20, 2021 (GLOBE NEWSWIRE) -- Biosplice Therapeutics, Inc. ("Biosplice"), a clinical-stage biotechnology company pioneering therapeutics based on alternative pre-mRNA splicing for major diseases, announced today the addition of three independent directors to its board. New directors comprise Troy Cox, former CEO of Foundation Medicine, Susannah Gray, former EVP and CFO of Royalty Pharma Management, and Karen McGinnis, former VP and CAO of Illumina.

    "We are honored to have this caliber of experience and leadership join our new board at Biosplice," said Osman Kibar, PhD, Founder and Executive Chairman of Biosplice. "Ms. Gray, Ms. McGinnis, and Mr. Cox bring invaluable insight to our company as we continue the buildout of our…

    SAN DIEGO, April 20, 2021 (GLOBE NEWSWIRE) -- Biosplice Therapeutics, Inc. ("Biosplice"), a clinical-stage biotechnology company pioneering therapeutics based on alternative pre-mRNA splicing for major diseases, announced today the addition of three independent directors to its board. New directors comprise Troy Cox, former CEO of Foundation Medicine, Susannah Gray, former EVP and CFO of Royalty Pharma Management, and Karen McGinnis, former VP and CAO of Illumina.

    "We are honored to have this caliber of experience and leadership join our new board at Biosplice," said Osman Kibar, PhD, Founder and Executive Chairman of Biosplice. "Ms. Gray, Ms. McGinnis, and Mr. Cox bring invaluable insight to our company as we continue the buildout of our alternative splicing platform and navigate the final stages of clinical development for our lead osteoarthritis drug candidate, lorecivivint, and continue to develop our promising oncology and neurology pipelines."

    Troy Cox led Foundation Medicine (NASDAQ:FMI), as President and Chief Executive Officer, through the acquisition by Roche for $5.3 billion in 2018. Prior to Foundation Medicine, he served as Senior Vice President and Officer at Genentech, where he led US BioOncology for a period of unprecedented growth to over $12 billion while accountable to help steer the Roche Genentech R&D portfolio. Before Genentech, Mr. Cox held executive and senior roles, including President UCB BioPharmaceuticals, Senior Vice President Sanofi-Aventis and diverse foundational roles at Schering-Plough.   He also currently serves as a director of Zymeworks, Inc. (NYSE:ZYME), Sophia Genetics, LetsGetChecked, CM Life Sciences II (NASDAQ:CMIIU) and MassBio.

    Susannah Gray brings more than 30 years of biopharmaceutical experience, specifically in corporate finance and capital markets roles, most recently serving as Executive Vice President, Finance & Strategy of Royalty Pharma Management, LLC. Ms. Gray also spent 14 years as Executive Vice President and Chief Financial Officer of Royalty Pharma before retiring in 2019.   Prior to joining Royalty Pharma, she had a 14-year career in banking, working in various capacities within high yield and structured finance departments of Chase Securities, Merrill Lynch and CIBC World Markets. Ms. Gray also serves as a director of Maravai LifeSciences (NASDAQ:MRVI) and 4D Molecular Therapeutics (NASDAQ:FDMT).

    Karen McGinnis has over 20 years of experience in executive operational and finance roles at international public companies. She most recently served as Vice President & Chief Accounting Officer of Illumina, Inc. (NASDAQ:ILMN), a leader in sequencing- and array-based solutions for genetic and genomic analysis. Earlier in her career, Ms. McGinnis served as Director, President and Chief Executive Officer, and previously Chief Financial Officer, of Mad Catz Interactive, Inc. (OTC:MCZAF), a global provider of innovative interactive entertainment products. Prior to joining Mad Catz, Ms. McGinnis served as Chief Accounting Officer of Cymer, Inc., a leading supplier to the semiconductor industry and, previously, Chief Accounting Officer for Insight Enterprises, Inc. Ms. McGinnis also currently serves as a director of Alphatec Holdings, Inc. (NASDAQ:ATEC) and AbSci, Inc.

    Learn more about Biosplice's Board of Directors at https://www.biosplice.com/board-of-directors

    About Biosplice

    Biosplice is developing first-in-class, small-molecule therapeutics based on pioneering science of alternative pre-mRNA splicing. Stemming from foundational discoveries in Wnt pathway modulation, Biosplice has elucidated novel biology linking CLK/DYRK kinases to the therapeutic regulation of alternative splicing. Alternative splicing is an essential biological mechanism that regulates the diversification of proteins in a cell, which, in turn, determines cell type and function. Biosplice's target class governs the selection of tissue-specific mRNA splice sites, making them attractive, druggable targets within the cellular "command and control" center.

    Biosplice's drugs in clinical development include lorecivivint for osteoarthritis (in Phase 3), cirtuvivint for numerous cancers, and a broad pipeline that ranges from Alzheimer's disease to other degenerative conditions. Learn more at https://www.biosplice.com

    Corporate Contact:

    Erich Horsley

    Biosplice Therapeutics, Inc.

    erich.horsley@biosplice.com

    858-365-0200

     



    Primary Logo

    View Full Article Hide Full Article
  9. - First patient dosed in the 4D-310 Phase 1/2 clinical trial in Fabry disease

    - Intravitreal product candidates, 4D-125 for the treatment of XLRP and 4D-110 for the treatment of choroideremia, completed dose escalation portion of Phase 1/2 clinical trials (n=12 patients)

    - Intravitreal product candidate 4D-150 for the treatment of wet AMD and DME on track to initiate clinical trial in the second half of 2021

    - Cash and cash equivalents of approximately $277M as of Dec 31, 2020

    EMERYVILLE, Calif., March 25, 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 year ended December 31…

    - First patient dosed in the 4D-310 Phase 1/2 clinical trial in Fabry disease

    - Intravitreal product candidates, 4D-125 for the treatment of XLRP and 4D-110 for the treatment of choroideremia, completed dose escalation portion of Phase 1/2 clinical trials (n=12 patients)

    - Intravitreal product candidate 4D-150 for the treatment of wet AMD and DME on track to initiate clinical trial in the second half of 2021

    - Cash and cash equivalents of approximately $277M as of Dec 31, 2020

    EMERYVILLE, Calif., March 25, 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 year ended December 31, 2020 and provided operational highlights.

    "2020 was a transformational year for 4D Molecular Therapeutics," said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. "We transitioned into a clinical-stage company, with three product candidates currently in clinical development: 4D-125 for X-linked retinitis pigmentosa, 4D-110 for choroideremia, and 4D-310 for Fabry disease. In addition, we strengthened our leadership team and corporate governance, with the addition of key clinical development executives and four experienced board members, including our Executive Chairman John Milligan, Ph.D. With the proceeds from our IPO, we raised the capital necessary to expand our vision for developing transformative next-generation gene therapies in multiple therapeutic areas for both rare and large market diseases."

    Recent Operational Highlights

    • Dosed the first patient in the Phase 1/2 clinical trial of 4D-310 for the treatment of Fabry disease in March 2021. The Phase 1/2 open-label, dose-escalation and dose-expansion clinical trial is expected to enroll up to 18 Fabry disease patients. The primary endpoints of this trial are to evaluate safety and to define the maximum-tolerated/-feasible dose.

    • Completed the dose escalation portion of the Phase 1/2 clinical trial of 4D-125, an intravitreal R100 vector-based product candidate, in adult patients with X-linked retinitis pigmentosa (XLRP). Dose escalation was completed following the dosing of six patients in two cohorts. The dose expansion portion of the trial will enroll patients at the highest dose tested of 1E12 vg/eye. To date, 4D-125 has been well-tolerated and has not resulted in dose-limiting toxicity or serious adverse events. The primary endpoints of this trial are to evaluate safety and to define the maximum-tolerated/-feasible dose.

    • Completed the dose escalation portion of the Phase 1/2 clinical trial of 4D-110, an intravitreal R100 vector-based product candidate, in adult patients with choroideremia. Dose escalation was completed following the dosing of six patients in two cohorts. The dose expansion portion of the trial will enroll patients at the highest dose tested of 1E12 vg/eye. To date, 4D-110 has been well-tolerated and has not resulted in dose-limiting toxicity or serious adverse events. The primary endpoints of this trial are to evaluate safety and to define the maximum-tolerated/-feasible dose.

    • Initiated IND-enabling studies for 4D-150, an intravitreal R100 vector-based product candidate, and submitted preclinical dataset for presentation at upcoming medical meeting. 4D-150 is a wholly owned product candidate for wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME) and is engineered for three distinct mechanisms-of-action.

    • Initiated 4D-710 IND-enabling studies and submitted preclinical dataset for presentation at upcoming medical meeting. 4D-710 is a product candidate for cystic fibrosis lung disease which is designed for efficient single dose aerosol delivery in a broad range of patients.



    • Completed upsized initial public offering in December 2020 which raised $222 million in gross proceeds, including the full exercise of the underwriters option to purchase additional shares. Together with the completion of a Series C financing earlier in the year, the company raised approximately $298 million in gross proceeds in 2020.



    • Strengthened corporate governance with appointments to its board of directors including John Milligan Ph.D., as Executive Chairman, and Susannah Gray, Nancy Miller-Rich and Shawn Tomasello as independent members. As a result of becoming a publicly-traded company, the Series B investor representative, Tony Yao, M.D., Ph.D., notified the board of his intent to resign, effective March 20, 2021. Likewise, the Series A investor representative, William Burkoth, notified the board on March 19, 2021 of his intent to not stand for reelection at the company's 2021 annual meeting of stockholders.



    • Strengthened leadership team with key appointments in clinical research and development, including Chief Medical Officer & Therapeutic Area Head, Pulmonology Robert Fishman, M.D.; Senior Vice President & Therapeutic Area Head, Cardiology Raphael Schiffmann, M.D.; and Senior Vice President & Clinical Therapeutic Area Head, Ophthalmology Robert Kim, M.D., M.B.A.

    Expected Upcoming Milestones

    • Initial clinical data from the Phase 1/2 clinical trial of 4D-125 in XLRP expected in the second half of 2021



    • Initial clinical data from the Phase 1/2 clinical trial of 4D-310 in Fabry disease expected in the second half of 2021



    • Presentation of 4D-150 preclinical data expected at upcoming medical meeting and initiation of clinical trial with 4D-150 in wet AMD and diabetic macular edema expected in the second half of 2021



    • Presentation of 4D-710 preclinical data expected at upcoming medical meeting and initiation of clinical trial with 4D-710 in cystic fibrosis lung disease expected in the second half of 2021

    Financial Results for the Year Ended December 31, 2020

    Cash and Cash Equivalents: Cash and cash equivalents was $276.7 million as of December 31, 2020, as compared to $49.7 million as of December 31, 2019. The increase in cash and cash equivalents was primarily a result of the proceeds received from the December 2020 initial public offering and the issuance of our Series C redeemable convertible preferred stock in the second quarter of 2020, which was partially offset by cash used in operations. We expect cash and cash equivalents to be sufficient to fund operations into mid-2023.

    Revenue: Total revenue was $13.6 million for the year ended December 31, 2020, as compared to $7.0 million for the year ended December 31, 2019. The increase was primarily driven by the recognition of revenue under the Roche collaboration agreement entered into in 2017.

    R&D Expenses: Research and development expenses were $53.0 million for the year ended December 31, 2020, as compared to $38.7 million for the year ended December 31, 2019. This increase was primarily due to higher external costs incurred to advance our clinical and preclinical programs and higher payroll and stock-based compensation expenses.

    G&A Expenses: General and administrative expenses were $17.2 million for the year ended December 31, 2020, as compared to $13.9 million for the year ended December 31, 2019. This increase was primarily due to higher payroll and stock-based compensation expenses and higher professional service expenses.

    Net Loss: Net loss was $56.7 million for the year ended December 31, 2020, as compared to $49.3 million for the year ended December 31, 2019.

    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 Annual Report on Form 10-K to be filed on 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

    (in thousands, except share and per share amounts)

     Year Ended

    December 31,
     2020 2019
       
     (in thousands, except

    share and per share data)
     
    Statements of Operations and Comprehensive Loss Data:       
    Revenue:       
    Collaboration and license revenue$13,363  $6,960 
    Collaboration and license revenue, related parties 249   26 
    Total revenue 13,612   6,986 
    Operating expenses:       
    Research and development 53,038   38,718 
    Acquired in-process research and development    5,137 
    General and administrative 17,238   13,895 
    Total operating expenses 70,276   57,750 
    Loss from operations (56,664)  (50,764)
    Other income (expense) (29)  1,458 
    Net loss and comprehensive loss$(56,693) $(49,306)
    Net loss per share attributable to common stockholders, basic and diluted$(8.82) $(9.59)
    Weighted-average shares outstanding used in computing net loss per share attributable to common stockholders, basic and diluted 6,430,555   5,142,560 
     

    4D Molecular Therapeutics, Inc.

    Condensed Balance Sheet Data

    (in thousands)

     As of December 31,
     2020 2019
       
     (in thousands)
    Balance Sheet Data:       
    Cash and cash equivalents$276,726  $49,652 
    Working capital 265,912   39,553 
    Total assets 288,331   58,234 
    Accumulated deficit (135,679)  (79,025)
    Total stockholders' equity (deficit) 256,387   (72,970)

    Contacts:

    Media:

    Theresa Janke

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article
  10. EMERYVILLE, Calif., March 09, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ:4), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the first patient has been dosed in the Phase 1/2 clinical trial of 4D-310 for the treatment of Fabry disease. Fabry disease is an inherited lysosomal storage disease with high unmet medical need that results from loss of function mutations in the alpha-galactosidase (AGA) enzyme.

    "Through our Therapeutic Vector Evolution platform we apply the principles of directed evolution to invent targeted and evolved AAV vectors for the delivery of genes to specific tissue types. Dosing the first patient in the Phase 1/2 clinical trial…

    EMERYVILLE, Calif., March 09, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ:4), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the first patient has been dosed in the Phase 1/2 clinical trial of 4D-310 for the treatment of Fabry disease. Fabry disease is an inherited lysosomal storage disease with high unmet medical need that results from loss of function mutations in the alpha-galactosidase (AGA) enzyme.

    "Through our Therapeutic Vector Evolution platform we apply the principles of directed evolution to invent targeted and evolved AAV vectors for the delivery of genes to specific tissue types. Dosing the first patient in the Phase 1/2 clinical trial of 4D-310 marks the third product candidate to be administered to patients, all of which utilize proprietary vectors derived from our Therapeutic Vector Evolution platform," said David Kirn, MD, chief executive officer, co-founder and president of 4DMT. "4D-310 is designed with the goal of achieving a novel dual mechanism-of-action for these patients. 4D-310 is designed for both stable therapeutic AGA enzyme activity in the blood, as well as for intracellular production within affected tissues, including in cardiac muscle cells."

    "The unmet need for patients with Fabry disease is significant. Current standard of care enzyme replacement therapies are associated with a high-treatment burden, and due to the short half-life in the blood, patients lack therapeutic concentrations of AGA between infusions. Cardiovascular disease is still the leading cause of death in patients despite standard therapy. Gene therapy represents a promising therapeutic modality for patients with Fabry disease because of its potential as a one-time therapy that can deliver stable and sustained levels of AGA activity," said Dr. Raphael Schiffmann, MD, Senior Vice President & Therapeutic Area Head, Cardiology at 4DMT. "The targeted organ distribution of 4D-310 in Fabry disease makes it likely that this therapy can also potentially treat the cardiomyopathy associated with Fabry disease, a critical disease manifestation that other therapies have been unable to address."

    The Phase 1/2 open-label, dose-exploration and dose-expansion study is expected to enroll up to 18 Fabry disease patients. The study is designed to assess the preliminary safety, tolerability, and maximum-tolerated or maximum-feasible dose of 4D-310. Key secondary endpoints include the assessment of biological activity including AGA enzyme activity and substrate concentrations in the blood over time. The first patient was dosed at University of Pittsburgh Medical Center (UPMC), Children's Hospital of Pittsburgh under the direction of principal investigator Dr. Jerry Vockley MD, PhD, chief of genomic and genetic medicine.

    About Fabry Disease and 4D-310

    Affecting more than 50,000 people in the United States and European Union, Fabry disease is a genetic disorder of the GLA gene that results in the body's inability to produce an enzyme called alpha-galactosidase or AGA, causing the accumulation of the substrate globotriaosylceramide-3 (Gb3) in critical organs, including the heart, kidney and blood vessels. Such substrate accumulation can lead to life-threatening hypertrophic cardiomyopathy, heart failure, arrhythmias, various degrees of kidney dysfunction and cerebrovascular stroke. Progression of the disease causes significant reduction in the quality of life and significant economic burden associated with greater patient needs for supportive care.

    By using a targeted and evolved vector to deliver a functional copy of the GLA gene, we believe 4D-310 has the potential to be a promising treatment of Fabry disease. 4D-310 is administered via IV delivery and is designed to produce both high, stable AGA activity in the bloodstream and to generate AGA activity intracellularly within critical affected organs, including the heart.

    About 4DMT

    4DMT is a clinical-stage gene therapy company pioneering the development of product candidates using targeted and evolved AAV vectors. 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, 4D-110 is in a Phase 1 clinical trial for choroideremia and 4D-310 is in a Phase 1/2 clinical trial for Fabry disease.

    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, including the therapeutic potential and clinical benefits thereof; 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 or 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' Registration Statement on Form S-1 dated December 10, 2020, 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 is our product candidate in clinical trials and has not yet been approved for marketing by the U.S. Food and Drug Administration. No representation is made as to the safety or effectiveness of 4D-310 for the therapeutic use for which it is being studied.

    Contacts:

    Media:

    Theresa Janke

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article
  11. EMERYVILLE, Calif., Dec. 15, 2020 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics, Inc. (4DMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced today the closing of its initial public offering of 9,660,000 shares of its common stock, including 1,260,000 shares sold pursuant to the full exercise of the underwriters' option to purchase additional shares, at a price of $23.00 per share. The aggregate gross proceeds of the offering were approximately $222 million, before deducting underwriting discounts and commissions and other offering expenses. The shares began trading on the Nasdaq Global Select Market on December 11, 2020 under the ticker symbol "FDMT."

    Goldman Sachs…

    EMERYVILLE, Calif., Dec. 15, 2020 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics, Inc. (4DMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced today the closing of its initial public offering of 9,660,000 shares of its common stock, including 1,260,000 shares sold pursuant to the full exercise of the underwriters' option to purchase additional shares, at a price of $23.00 per share. The aggregate gross proceeds of the offering were approximately $222 million, before deducting underwriting discounts and commissions and other offering expenses. The shares began trading on the Nasdaq Global Select Market on December 11, 2020 under the ticker symbol "FDMT."

    Goldman Sachs & Co. LLC, BofA Securities and Evercore ISI acted as book-running managers for the offering.

    Registration statements relating to these securities have been filed with the Securities and Exchange Commission (SEC) and became effective on December 10, 2020. Copies of the registration statements can be accessed through the SEC's website at www.sec.gov. This offering was made only by means of a prospectus. A copy of the final prospectus relating to this offering may be obtained from: Goldman Sachs & Co. LLC, Prospectus Department, 200 West Street, New York, New York 10282, by telephone at (866) 471-2526, or by email at Prospectus-ny@ny.email.gs.com; BofA Securities, Attention: Prospectus Department, NC1-004-03-43, 200 North College Street, 3rd Floor, Charlotte, North Carolina 28255-001, or by email at dg.prospectus_requests@bofa.com; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, New York 10055, by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About 4DMT

    4DMT is a clinical-stage gene therapy company pioneering the development of product candidates using targeted and evolved AAV vectors. 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, 4D-110 is in a Phase 1 clinical trial for choroideremia and 4D-310 is in a Phase 1/2 clinical trial for Fabry disease.

    4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT.

    Contacts:

    Media:

    Theresa Janke

    tjanke@4dmt.com

    Investors:

    Mike Zanoni

    Endurance Advisors

    mzanoni@4dmt.com



    Primary Logo

    View Full Article Hide Full Article