FIXX Homology Medicines Inc.

15.42
+0.08  (+1%)
Previous Close 15.34
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Latest News

  1.  -  Methods Showed Efficient On-Target Gene Integration with No Unintended
    DNA Modifications -

    BEDFORD, Mass., May 26, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today the peer-reviewed publication of methods used to evaluate the on-target efficiency and precision of Homology's in vivo phenylketonuria (PKU) gene editing program. These quantitative molecular methods provide a framework to characterize homologous recombination-based, nuclease-free gene integration and evaluate whether any unintended on-target mutations or viral insertions occurred.

    "As we continue to develop our AAVHSC-based gene editing technology into new treatment options and potential cures for patients, we believe…

     -  Methods Showed Efficient On-Target Gene Integration with No Unintended
    DNA Modifications -

    BEDFORD, Mass., May 26, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today the peer-reviewed publication of methods used to evaluate the on-target efficiency and precision of Homology's in vivo phenylketonuria (PKU) gene editing program. These quantitative molecular methods provide a framework to characterize homologous recombination-based, nuclease-free gene integration and evaluate whether any unintended on-target mutations or viral insertions occurred.

    "As we continue to develop our AAVHSC-based gene editing technology into new treatment options and potential cures for patients, we believe that it is important to employ quantitative molecular methods to characterize changes to the genome," stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. "We used multiple molecular methods to assess our gene editing technology, which demonstrated precise PAH gene integration into the target location without introducing any unintended mutations or viral components. We believe that the framework described herein represents a major advancement in the field of AAV-based gene editing and, going forward, should now enable the ability to make direct data comparisons across studies."

    In the publication, one of Homology's family of 15 adeno-associated viral vectors derived from human hematopoietic stem cells (AAVHSC15) was used to deliver the human PAH gene into the murine model with human hepatocytes. This construct is designed to leverage the body's natural DNA repair process of homologous recombination, without requiring a nuclease to cut the DNA.

    Key findings in the publication include:

    • Single I.V. administration of the AAVHSC15-based gene editing construct resulted in seamless integration of the human PAH gene in human hepatocytes in the murine model
      To determine whether gene integration occurred at the human PAH locus, the study employed a targeted integration (TI) PCR, and the TI amplicon was then purified and Sanger sequenced. The resulting data matched with reference, confirming PAH gene integration.

    • Gene integration was precise without de novo mutations
      Next-generation sequence analysis spanning the full integration site revealed there were no unintended on-target mutations.
                   
    • AAVHSC15-mediated gene editing occurs via homologous recombination
      Analysis of the target location showed a pattern of DNA strand cross-over that supports homologous recombination as the pathway for gene integration. These findings are consistent with published work demonstrating AAV-mediated gene integration occurs via the homologous recombination pathway.

    • No inverted terminal repeats (ITRs) integrations
      Long-read sequencing was used to determine that there were no ITRs at the integration site, which also supports that AAVHSC15-mediated gene editing is due to homologous recombination.
       
    • Gene integration efficiency was characterized at the DNA level
      Two independent quantitation assays directly assessed the DNA, including linkage analysis by droplet digital PCR (ddPCR) and 3-primer next-generation sequencing, and each demonstrated approximately 6% gene integration. This level of editing is an improvement over the efficiencies published in others' nuclease-free gene editing studies.

    The integration efficiencies in this publication are consistent with efficiencies seen in the PKU Pahenu2 murine model following a single administration of Homology's nuclease-free gene editing construct, which corrected the disease phenotype.i Homology Medicines is currently in IND-enabling studies with a lead gene editing development candidate, HMI-103, for the treatment of pediatric patients with PKU. Since the liver rapidly divides throughout childhood, a gene editing approach, which unlike gene therapy makes a permanent correction to the genome, could offer a potential cure for children with PKU.

    The publication, "Molecular Characterization of Precise In Vivo Targeted Gene Integration in Human Cells Using AAVHSC15," was peer-reviewed and published in the journal PLOS ONE. For more information, please visit https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0233373 or www.homologymedicines.com/publications.

    About Homology Medicines, Inc.
    Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology's proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit www.homologymedicines.com.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential of our gene editing framework and the ability to make direct data comparisons across studies; the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; our goal of delivering potential cures to patients; beliefs about preclinical data; and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

    Company Contacts:
    Theresa McNeely
    Chief Communications Officer
    and Patient Advocate

    781-301-7277
     
    Media Contact:
    Cara Mayfield
    Senior Director, Patient Advocacy
    and Corporate Communications

    781-691-3510

    _____________________________

    iEllsworth J, Smith L, St. Martin T, et al. Nuclease-free genome editing by AAVHSC vectors leads to in vivo genome correction and amelioration of disease phenotype in a mouse model of phenylketonuria (PKU). Human Gene Therapy. Nov 2019. A1-A221.

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  2. - Molecular Methods Quantified Precision and Efficiency of Nuclease-Free Gene Editing for PKU -

    - Manufacturing Enhancements Led to Improved Productivity, Quality and Scalability of Commercial Process, Confirmed in 2,000L Bioreactor -

    - Data Highlight Unique Characteristics of AAVHSC Genetic Medicines Platform -

    BEDFORD, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today the presentation of data at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting. Among Homology's seven presentations are data from its in vivo nuclease-free gene editing program for phenylketonuria (PKU) and in vivo gene therapy program for metachromatic leukodystrophy (MLD), both…

    - Molecular Methods Quantified Precision and Efficiency of Nuclease-Free Gene Editing for PKU -

    - Manufacturing Enhancements Led to Improved Productivity, Quality and Scalability of Commercial Process, Confirmed in 2,000L Bioreactor -

    - Data Highlight Unique Characteristics of AAVHSC Genetic Medicines Platform -

    BEDFORD, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today the presentation of data at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting. Among Homology's seven presentations are data from its in vivo nuclease-free gene editing program for phenylketonuria (PKU) and in vivo gene therapy program for metachromatic leukodystrophy (MLD), both of which are in IND-enabling studies. Presentations also focus on the Company's commercial manufacturing platform, as well as data on the differentiating characteristics of Homology's family of AAVHSC vectors, particularly when compared to other AAVs, which highlight the potential of the Company's dual gene therapy and editing platform.

    "Homology has made substantial progress in understanding the unique properties of our AAVHSC-based technology and this enables us to move our dual genetic medicines platform forward to develop potential treatments, or cures, for patients," stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. "We are pleased to share data here that describe the molecular methods we have developed to characterize in vivo, nuclease-free gene editing efficiency and precision at the DNA level. Additional data from our in vivo MLD gene therapy program demonstrates the impact on key biomarkers in two species, as well as the durability of effect in the murine model of disease with data out to 52 weeks. Underpinning all our programs is our internal GMP process and manufacturing capabilities, where we have now confirmed our commercial HEK293 suspension platform at the 2,000-liter scale, bringing our total internal capacity to 3,500 liters. Additionally, we are presenting data showing improved AAVHSC packaging as compared to the non-Clade F vector AAV5."

    Highlights from Homology's 2020 ASGCT Presentations

    The presentation, "Molecular Characterization of Precise In Vivo Targeted Gene Editing in Human Cells using AAVHSC15, a New AAV Derived from Hematopoietic Stem Cells (AAVHSC)," describes quantitative molecular methods to measure efficiency and precision of nuclease-free, homologous recombination-based gene editing. The studies, which used a single I.V. administration of a gene editing construct to insert the human PAH gene, which is mutated in people with phenylketonuria (PKU), in a humanized liver murine model, show:

    • Precise gene editing with no unintended on-target mutations or viral integration, supportive of the homologous recombination pathway
    • Six percent gene editing efficiency using two independent molecular assays. This level of editing is an improvement over the efficiencies published in others' nuclease-free gene editing studies, and it has been shown to be sufficient to normalize Phe levels in the murine model.

    Two posters related to Homology's internal commercial manufacturing platform will be presented.
    In "Molecular Design and Characterization of Packaging Plasmid Sequences for Improved Production of Novel Clade F AAVHSCs," the data demonstrate:

    • AAVHSCs were suitable for packaging a wide range of AAV genome sizes and yielded more intact packaged genomes than AAV5, a non-Clade F serotype
    • Improved productivity and optimized upstream process following enhancements to the packaging plasmid sequences

    In "Development and Scalability of Transfection-Based Production and Purification of Novel Clade F Adeno-Associated Viruses Isolated from Human Hematopoietic Stem Cells (AAVHSCs)," Homology describes high-quality productivity and scalability of its mammalian, suspension-based manufacturing, including:

    • Executed 2,000-liter bioreactor scale using Homology's commercial process, with comparable productivity and product quality to smaller scale runs
    • Improved product quality using anion exchange purification, which can be leveraged across Homology's family of AAVHSC vectors and product candidates

    Related to Homology's HMI-202 investigational gene therapy for MLD, the presentation, "Gene Therapy for Metachromatic Leukodystrophy (MLD) That Crosses the Blood-Nerve and Blood-Brain Barriers in Mice and Non-Human Primates," details that a single I.V. administration:

    • Crossed the blood-brain-barrier and blood-nerve barriers in the MLD murine model and in non-human primates, with human ARSA (hARSA) enzyme detected in both species
    • Showed hARSA biodistribution in the murine model that resembled endogenous murine ARSA in age-matched controls
    • Produced durable hARSA levels in the murine model meeting or exceeding normal human brain ARSA activity levels out to 52 weeks
    • Positively impacted key MLD biomarkers, including reducing sulfatides and LAMP-1 (lysosomal-associated membrane protein 1), as well as increased MAL (myelin and lymphocyte protein)

    In collaboration with Children's Hospital of Philadelphia (CHOP), Homology also presents, "In Vivo Transduction of Murine Hematopoietic Stem Cells after Intravenous Injection of AAVHSC15 and AAVHSC17," which shows:

    • AAVHSC15 and AAVHSC17 - two of Homology's family of 15 AAVHSCs that were tested in the studies - can target murine hematopoietic stem cells in vivo
    • Transduced cells were viable, self-renewed and actively contributed to hematopoiesis over time

    As Homology has advanced its AAVHSC technology, it is presenting mechanistic data on the platform, including the following two presentations.
    In "Role of Terminal Galactose in Cellular Uptake, Intracellular Trafficking, and Tissue Tropism Using Adeno-Associated Viruses Isolated from Human Stem Cells (AAVHSCs)," the data show:

    • AAVHSC transduction efficiency improved with increased galactose binding, a finding that has been previously described with Clade F vector AAV9
    • Nucleotide differences in AAVHSCs led to differences in transduction efficiencies related to galactose binding

    In "AAVHSCs Transduction Does Not Significantly Elicit p53-Mediated Apoptosis or Alter Cell Cycle in Human iPSCs and Primary Cells When Compared to Non-Clade F AAV Vectors," the studies demonstrate that AAVHSCs:

    • Have differentiating properties than those vectors in Clades 1-8, and did not induce p53 or CHK2 activation, which are responsible for apoptosis (cell death)
    • Even at high multiplicities of infection (MOI), AAVHSCs did not elicit cell death or alter cell cycle             

    For more information about the presentations, visit Homology's website at www.homologymedicines.com/publications.

    About Homology Medicines, Inc.
    Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology's proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit www.homologymedicines.com.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; our beliefs regarding our manufacturing capabilities; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

    Company Contacts
    Theresa McNeely
     
    Chief Communications Officer
    and Patient Advocate
     

    781-301-7277
     
     
    Media Contact:  
    Cara Mayfield  
    Senior Director, Patient Advocacy
    and Corporate Communications
     

    781-691-3510
     


     

     

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  3. - Progressed the pheNIX Trial for PKU and Added Clinical Sites, Completed Clinical Supply for Phase 1/2 and Began Manufacturing Pivotal Supply -

    - Executed Commercial Manufacturing Process at 2,000-Liter Bioreactor Scale -

     - Implemented Plans to Minimize Potential COVID-19 Impact on Employees and Operations -

    BEDFORD, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today financial results for the first quarter ended March 31, 2020, and highlighted recent accomplishments.

    "The Homology team's experience, agility, and forward-thinking capabilities are a tremendous asset to the Company as we operate within this global health crisis, and as a result, our programs have continued…

    - Progressed the pheNIX Trial for PKU and Added Clinical Sites, Completed Clinical Supply for Phase 1/2 and Began Manufacturing Pivotal Supply -

    - Executed Commercial Manufacturing Process at 2,000-Liter Bioreactor Scale -

     - Implemented Plans to Minimize Potential COVID-19 Impact on Employees and Operations -

    BEDFORD, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today financial results for the first quarter ended March 31, 2020, and highlighted recent accomplishments.

    "The Homology team's experience, agility, and forward-thinking capabilities are a tremendous asset to the Company as we operate within this global health crisis, and as a result, our programs have continued to move forward," stated Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. "In taking proactive steps early on, we believe we were able to minimize the potential impact to our business by procuring sufficient raw materials, accelerating manufacturing and implementing safety measures for employees. In addition, we have established home-based care and monitoring services for patients in our Phase 1/2 pheNIX gene therapy trial for PKU, making their safety and continuity of the study priorities. We will continue to evaluate these measures and make adjustments as needed. Our plan is still to report additional data from our pheNIX clinical trial mid-year. Given the evolving nature of the COVID-19 pandemic, we will continue to work closely with trial sites in our efforts to achieve this goal."

    Dr. Tzianabos concluded, "Our pipeline is also advancing, and we recently presented data further demonstrating the potential of our AAVHSC platform for diseases of the central and peripheral nervous systems. As leaders in the field of gene therapy and gene editing manufacturing, we expanded the internal capacity of our mammalian, suspension-based system to 1,500 liters, and we executed our commercial process at the 2,000-liter bioreactor scale."

    First Quarter 2020 Financial Results and Recent Accomplishments

    • Progressed the dose-escalation phase of the pheNIX clinical trial evaluating HMI-102 gene therapy for the treatment of adults with phenylketonuria (PKU).
      • Following encouraging initial clinical data from the trial announced in December 2019, additional PKU centers across the U.S. were initiated, bringing the total to eight trial sites.
      • Produced all the HMI-102 supply for the dose-escalation and expansion phases of the trial using Homology's commercial manufacturing process and began producing supply for a pivotal trial.
      • As previously announced, Homology plans to provide an update on the trial mid-year when a dose is selected for the expansion phase, which has the potential to be converted to a registrational trial.
    • Highlighted preclinical data supporting the HMI-102 program in a peer-reviewed publication in Molecular Therapy: Methods & Clinical Development. The data demonstrated HMI-102 restored the normal biochemical pathway in the established PKU murine model on normal protein diet; initial data from the pheNIX trial announced in December 2019 suggest that the increased PAH enzymatic activity seen in the preclinical model was also observed in the clinical study following a single I.V. administration of HMI-102.
    • Presented data demonstrating that Homology's AAVHSC vectors crossed the blood-brain-barrier and blood-nerve-barrier in the metachromatic leukodystrophy (MLD) murine model and non-human primates and that the HMI-202 gene therapy candidate for MLD reduced key biomarkers of disease and produced normal levels of human ARSA protein in the murine model. These data were presented at the WORLDSymposium™ 2020 Conference and support the further development of AAVHSC-based gene therapies for diseases of the nervous system.
    • Announced upcoming presentations on Homology's in vivo gene therapy and nuclease-free gene editing platform across multiple disease areas, mechanistic data further characterizing AAVHSC capsids, and details on Homology's commercial manufacturing process and internal GMP production capabilities at the virtual American Society of Gene & Cell Therapy Annual Meeting during the poster sessions May 12-14.
    • Increased GMP manufacturing capacity using the commercial process as well as executed 2,000-liter bioreactor scale in the Company's internal manufacturing facility.
    • Implemented business continuity plans to minimize the impact of COVID-19 on business operations:
      • Worked with pheNIX clinical trial sites to mitigate potential COVID-19-related disruptions and to ensure the safety of patients and healthcare professionals.
        • Deployed home-health services, home visits for close monitoring and reporting, and centralized laboratory testing for enrolled patients.
        • Homology will continue to evaluate the ongoing global pandemic, which has and may continue to affect patient visits, enrollment and resources at many of our clinical trial sites, and will adjust as needed.
      • Developed and implemented a shift schedule for employees whose work in Homology's internal manufacturing facility and laboratories requires an on-site presence and instituted a work-from-home policy for all other employees.
        • Developed a modified office layout and traffic flow pattern to increase spacing capabilities, reduce inter-office risks and allow for continuity of activities.
        • Increased cleaning protocols and restricted visitors to the office.
        • Cancelled all business travel.
      • Accelerated procurement of raw materials for future manufacturing and research and development needs to minimize potential supply chain interruptions.

    First Quarter 2020 Financial Results

    • Net loss for the quarter ended March 31, 2020 was $(35.3) million or $(0.78) per share, compared to a net loss of $(23.9) million or $(0.64) per share for the same period in 2019.
    • Collaboration revenues for the quarter ended March 31, 2020 were $0.6 million, compared to $0.3 million for quarter ended March 31, 2019 and consisted of revenue recognized under the Company's strategic collaboration with Novartis. Collaboration revenues are being recognized over time consistent with the pattern of performance of research and development activities under the collaboration agreement. Homology and Novartis continue to work together on ophthalmic programs and seek to identify new targets for the collaboration based on the collaboration's exploratory research component.
    • Total operating expenses for the quarter ended March 31, 2020 were $37.1 million, compared to $25.4 million for the quarter ended March 31, 2019, and consisted of research and development expenses and general and administrative expenses.
    • Research and development expenses for the quarter ended March 31, 2020 were $29.3 million, compared to $20.5 million for the quarter ended March 31, 2019. Research and development expenses increased due to a rise in direct research expenses, including costs related to manufacturing preclinical study and clinical trial materials, costs incurred with Homology's contract research organization to conduct and manage the Phase 1/2 pheNIX clinical trial, and development costs in advancing HMI-202 and HMI-103 through IND-enabling studies. Increased costs also reflected additional personnel to support ongoing development programs, research initiatives, technology platform enhancements and manufacturing capabilities, as well as increased expenses related to the accelerated procurement of raw materials for future manufacturing, and research and development needs in response to the COVID-19 pandemic.  
    • General and administrative expenses for the quarter ended March 31, 2020 were $7.8 million, compared to $4.9 million for the quarter ended March 31, 2019. General and administrative expenses increased due to personnel costs as a result of new hires, increased consulting costs and additional costs associated with expanded operations.
    • As of March 31, 2020, Homology had approximately $234.9 million in cash, cash equivalents and short-term investments. Based on current projections, Homology expects cash resources to fund operations into the fourth quarter of 2021.

    Upcoming Virtual Events

    • BofA Securities 2020 Health Care Conference: Fireside Chat on May 12 at 11:00 a.m. ET
    • American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting on May 12-15
    • RBC Global Healthcare Conference 2020: Fireside Chat on May 20 at 1:20 p.m. ET

    About the Phase 1/2 pheNIX Clinical Trial in Phenylketonuria (PKU)
    The pheNIX trial is the first gene therapy clinical trial ever conducted for people with PKU. pheNIX is designed to evaluate the safety and efficacy of a single intravenous administration of HMI-102 in adult patients with PKU aged 18-55. The study design allows for expansion of the number of patients in any dose cohort pending review by the Data Monitoring Committee and the Homology Internal Data Review Team. A decision to expand would trigger the addition of the randomized, concurrently controlled Part B of the trial, which has the potential to be converted to a registrational trial. The primary efficacy endpoint of the expansion part is incidence of sustained plasma Phe concentration ≤360 μmol/L as demonstrated by two measurements ≤360 μmol/L between 16 and 24 weeks.

    About Homology Medicines, Inc.
    Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology's proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit www.homologymedicines.com.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of clinical data from the Phase 1/2 pheNIX trial, including the Part B expansion part and the potential for conversion to a registrational trial; the anticipated impact of the COVID-19 pandemic on our business and operations; our collaboration activities with Novartis; our beliefs regarding our manufacturing capabilities; our position as a leader in the development of genetic medicines; the sufficiency of our cash, cash equivalents and short-term investments to fund our operations; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

     
    HOMOLOGY MEDICINES, INC.
    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
    (in thousands, except share and per share amounts)
    (Unaudited)
             
        Three months ended March 31,
          2020       2019  
    Collaboration revenue   $ 588     $ 270  
    Operating expenses:        
    Research and development     29,310       20,536  
    General and administrative     7,770       4,857  
    Total operating expenses     37,080       25,393  
    Loss from operations     (36,492 )     (25,123 )
    Other income:        
    Interest income     1,161       1,271  
    Total other income     1,161       1,271  
    Net loss   $ (35,331 )   $ (23,852 )
    Net loss per share-basic and diluted   $ (0.78 )   $ (0.64 )
    Weighted-average common shares outstanding-basic and diluted     45,151,265       37,384,507  
             



    HOMOLOGY MEDICINES, INC.
    CONDENSED CONSOLIDATED BALANCE SHEETS
    (in thousands)
    (Unaudited)
             
        As of
        March 31, 2020   December 31, 2019
    Cash, cash equivalents and short-term investments   $ 234,929   $ 262,388
    Property and equipment, net     42,566     42,716
    Other assets     11,293     5,463
    Total assets   $ 288,788   $ 310,567
             
    Accounts payable, accrued expenses and other liabilities   $ 14,347   $ 21,109
    Operating lease liabilities     17,152    
    Deferred revenue     30,748     30,951
    Stockholders' equity     226,541     258,507
    Total liabilities and stockholders' equity   $ 288,788   $ 310,567
             


    Company Contacts
     
    Theresa McNeely  
    Chief Communications Officer and Patient Advocate  
       
    781-301-7277
     
     
    Media Contact:  
    Cara Mayfield  
    Senior Director, Patient Advocacy and Corporate Communications  
       
    781-691-3510  

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  4. BEDFORD, Mass., April 29, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today upcoming presentations demonstrating the broad applicability of its in vivo gene therapy and nuclease-free gene editing platform across multiple disease areas. Presentations will also include mechanistic data further characterizing Homology's novel AAVHSC vectors and details on the commercial manufacturing process and internal GMP production capabilities at Homology. These data will be presented during the virtual American Society for Gene & Cell Therapy (ASGCT) 23rd Annual Meeting in the online poster sessions May 12 - 14, 2020.

    "We are looking forward to sharing data that demonstrate the potential of our…

    BEDFORD, Mass., April 29, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today upcoming presentations demonstrating the broad applicability of its in vivo gene therapy and nuclease-free gene editing platform across multiple disease areas. Presentations will also include mechanistic data further characterizing Homology's novel AAVHSC vectors and details on the commercial manufacturing process and internal GMP production capabilities at Homology. These data will be presented during the virtual American Society for Gene & Cell Therapy (ASGCT) 23rd Annual Meeting in the online poster sessions May 12 - 14, 2020.

    "We are looking forward to sharing data that demonstrate the potential of our AAVHSC genetic medicines platform to treat rare genetic diseases such as PKU and MLD," stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. "Our presentations represent how we continue to advance our understanding of Homology's proprietary AAVHSC vectors through characterization of cellular transduction, impact on cell cycle, and biodistribution to disease-relevant cell types after a single I.V. administration. Homology's leadership in gene therapy and gene editing manufacturing will be the subject of presentations highlighting our efficient commercial process and scalable internal GMP manufacturing platform, which currently serves the needs of our clinical and preclinical programs. Additionally, our contributions to the field of gene editing will be featured in Homology data detailing molecular methods used to measure homologous recombination-based genomic integration, methods that we believe are important to characterize changes to the genome, measure integration efficiency and compare results across studies and platforms."

    Homology's ASGCT 2020 presentations include:

    In Vivo, Nuclease-Free Gene Editing for PKU
    Molecular Characterization of Precise In Vivo Targeted Gene Editing in Human Cells using AAVHSC15, a New AAV Derived from Hematopoietic Stem Cells (AAVHSC)

    • Tuesday, May 12; 5:30 - 6:30 p.m.
    • Abstract #: 227

    Scalable Manufacturing
    Molecular Design and Characterization of Packaging Plasmid Sequences for Improved Production of Novel Clade F AAVHSCs

    • Tuesday, May 12; 5:30 - 6:30 p.m.
    • Abstract #: 445

    Development and Scalability of Transfection-Based Production and Purification of Novel Clade F Adeno-Associated Viruses Isolated from Human Hematopoietic Stem Cells (AAVHSCs)

    • Thursday, May 14; 5:30 - 6:30 p.m.
    • Abstract #: 1248

    In Vivo Gene Therapy for MLD
    Gene Therapy for Metachromatic Leukodystrophy (MLD) That Crosses the Blood-Nerve and Blood-Brain Barriers in Mice and Non-Human Primates

    • Wednesday, May 13; 5:30 - 6:30 p.m.
    • Abstract #: 590

    In Vivo Transduction of Hematopoietic Stem Cells
    In Vivo
     Transduction of Murine Hematopoietic Stem Cells after Intravenous Injection of AAVHSC15 and AAVHSC17

    • Wednesday, May 13; 5:30 - 6:30 p.m.
    • Abstract #: 600

    AAVHSC Platform
    Role of Terminal Galactose in Cellular Uptake, Intracellular Trafficking, and Tissue Tropism Using Adeno-Associated Viruses Isolated from Human Stem Cells (AAVHSCs)

    • Wednesday, May 13; 5:30 - 6:30 p.m.
    • Abstract #: 570

    AAVHSCs Transduction Does Not Significantly Elicit p53-Mediated Apoptosis or Alter Cell Cycle in Human iPSCs and Primary Cells When Compared to Non-Clade F AAV Vectors

    • Thursday, May 14; 5:30 - 6:30 p.m.
    • Abstract #: 1011

    The abstracts are available on the ASGCT website.
     
    About Homology Medicines, Inc.
    Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology's proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit www.homologymedicines.com.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of preclinical data; our beliefs regarding our manufacturing capabilities; beliefs about preclinical data; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property; the impact of the COVID-19 pandemic on our operations, the continuity of our business, including our preclinical studies and clinical trials, and general economic conditions; and significant costs as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2019 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

    Company Contacts:
    Theresa McNeely
    Chief Communications Officer
    and Patient Advocate

    781-301-7277

    Media Contact:
    Cara Mayfield
    Senior Director, Patient Advocacy
    and Corporate Communications

    781-691-3510

    Primary Logo

    View Full Article Hide Full Article
  5. - Data Package Supported Initiation of Ongoing pheNIX Clinical Trial for Adults with PKU -

    BEDFORD, Mass., March 16, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today the peer-reviewed publication of preclinical data that supports Homology's HMI-102 investigational gene therapy program for the treatment of adults with phenylketonuria (PKU). HMI-102 is currently being evaluated in the pheNIX Phase 1/2 clinical trial, and the Company plans to provide an update on the trial when selecting the dose for the expansion part, which is currently anticipated in mid-2020.

    The published data shows that a single administration of HMI-102 (AAVHSC15-PAH) produced a sustained reduction in phenylalanine…

    - Data Package Supported Initiation of Ongoing pheNIX Clinical Trial for Adults with PKU -

    BEDFORD, Mass., March 16, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (NASDAQ:FIXX), a genetic medicines company, announced today the peer-reviewed publication of preclinical data that supports Homology's HMI-102 investigational gene therapy program for the treatment of adults with phenylketonuria (PKU). HMI-102 is currently being evaluated in the pheNIX Phase 1/2 clinical trial, and the Company plans to provide an update on the trial when selecting the dose for the expansion part, which is currently anticipated in mid-2020.

    The published data shows that a single administration of HMI-102 (AAVHSC15-PAH) produced a sustained reduction in phenylalanine (Phe), the key biomarker in the diagnosis and management of PKU, for the lifespan of the established murine model for PKU. The data also demonstrated a concomitant increase in tyrosine (Tyr), a metabolite of Phe and precursor to neurotransmitters, indicating enzymatic activity. Additionally, brain levels of Phe, 5-HIAA (downstream serotonin metabolite) and coat color were normalized, further indicating restoration of the Phe metabolic pathway.

    "We developed a robust preclinical data package for our investigational HMI-102 gene therapy, which supported the initiation of our ongoing Phase 1/2 pheNIX clinical trial for adults with PKU," stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. "These published data demonstrated that a single dose of HMI-102 was able to restore the normal biochemical pathway in the established PKU model on normal protein diet. Initial data from the pheNIX trial suggests that the increased PAH enzymatic activity after administration of HMI-102 seen in the preclinical model was also observed in the clinical study."

    Key data in the publication include:

    • A single IV administration of HMI-102 reduced serum Phe concentrations to normal levels within one week in the PKU murine model, and mean levels remained below 120 µM (the normal range) over the course of the 48-week study.
    • A corresponding increase in Tyr concentration was also observed, indicating restoration of the Phe/Tyr metabolic pathway.
    • HMI-102 administration was also associated with an increase in 5-HIAA and a decrease in Phe in the brain to normal levels.

    The publication, "Sustained Correction of a Murine Model of Phenylketonuria Following a Single Intravenous Administration of AAVHSC15-PAH," was peer-reviewed and published in the journal Molecular Therapy: Methods & Clinical Development. For more information, please visit www.homologymedicines.com/publications.

    About the Phase 1/2 pheNIX Clinical Trial in Phenylketonuria (PKU)
    The pheNIX trial is the first gene therapy clinical trial ever conducted for people with PKU. pheNIX is designed to evaluate the safety and efficacy of a single intravenous administration of HMI-102 in adult patients with PKU aged 18-55. The study design allows for expansion of the number of patients in any dose cohort pending review by the Data Monitoring Committee and the Homology Internal Data Review Team. A decision to expand would trigger the addition of the randomized, concurrently controlled Part B of the trial, which has the potential to be converted to a registrational trial. The primary efficacy endpoint of the expansion part is incidence of sustained plasma Phe concentration ≤360 μmol/L as demonstrated by two measurements ≤360 μmol/L between 16 and 24 weeks.

    About Homology Medicines, Inc.
    Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology's proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit www.homologymedicines.com.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of clinical data from the Phase 1/2 pheNIX trial, including the Part B expansion; plans and timing for the release of clinical data; our beliefs regarding our manufacturing capabilities; advancing our novel platform and pipeline; our goal of delivering potential cures to patients; beliefs about preclinical data; our position as a leader in the development of genetic medicines; the sufficiency of our cash, cash equivalents and short-term investments; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2019 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

    Company Contacts:
    Theresa McNeely
    Chief Communications Officer
    and Patient Advocate

    781-301-7277

    Media Contact:
    Cara Mayfield
    Senior Director, Patient Advocacy
    and Corporate Communications

    781-691-3510

    Primary Logo

    View Full Article Hide Full Article
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