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Homology Medicines, Inc. (FIXX)

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Neutralpattern detected
Previous Close7.39
Open7.40
Bid7.45 x 1800
Ask7.43 x 800
Day's Range7.32 - 7.45
52 Week Range5.94 - 17.34
Volume95,361
Avg. Volume730,907
Market Cap422.707M
Beta (5Y Monthly)-0.32
PE Ratio (TTM)N/A
EPS (TTM)-2.80
Earnings DateAug 09, 2021 - Aug 13, 2021
Forward Dividend & YieldN/A (N/A)
Ex-Dividend DateN/A
1y Target Est23.60
  • Homology Medicines Announces Presentations on its Expanding Genetic Medicines Platform and Internal GMP Manufacturing at the American Society of Gene & Cell Therapy Annual Meeting
    GlobeNewswire

    Homology Medicines Announces Presentations on its Expanding Genetic Medicines Platform and Internal GMP Manufacturing at the American Society of Gene & Cell Therapy Annual Meeting

    - Data From New GTx-mAb Platform Demonstrated Proof of Principle for AAVHSCs to Deliver One-Time In Vivo Gene Therapy to Produce Antibodies in Humanized Murine Model - - Additional Data for HMI-203 Gene Therapy in Hunter Syndrome and HMI-103 Gene Editing in PKU Support Planned Phase 1/2 Clinical Trial Initiations by End of 2021 - - On Track to Report Initial Phase 2 Data From pheNIX PKU Clinical Trial by Year End - - Conference Call / Webcast Today, May 13 at 8:15 a.m. ET - BEDFORD, Mass., May 13, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of data in multiple disease areas supporting the Company’s human hematopoietic stem cell-derived adeno-associated virus vector (AAVHSC) platform. Among Homology’s presentations are data from its new GTx-mAb platform targeting complement protein 5 (C5), which showed proof of principle in paroxysmal nocturnal hemoglobinuria (PNH). The Company also presented data from its gene therapy program for MPS II (Hunter syndrome) and gene editing program for phenylketonuria (PKU), both of which are on track to enter the clinic this year. Presentations also focused on the Company’s internal commercial GMP manufacturing process and platform. These data were featured at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting. “Our new GTx-mAb platform is designed to leverage our AAVHSCs to deliver one-time in vivo gene therapy to produce antibodies from the liver and secrete them throughout the body,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “These data demonstrated once again that our vectors are highly efficient in targeting the liver. With this approach, we showed our vectors enabled the liver to produce antibodies that resulted in sustained expression levels in the serum of a humanized murine model that inhibited red blood cell destruction using an ex vivo assay. These PNH data are exciting, given the high unmet need, and underscore the potential to address other complement-related diseases, as well as diseases with larger patient populations. We are on track to name our first development candidate from the GTx-mAb platform in PNH this year.” Dr. Seymour continued, “Our multiple presentations at ASGCT highlight the maturation of our pipeline, which is expected to yield two additional clinical programs this year, including our Phase 1/2 clinical trials in Hunter syndrome and our first in vivo gene editing candidate for PKU. We plan to share additional details about our GTx-mAb platform and our ASGCT data during our conference call and webcast this morning.” Highlights from Homology’s 2021 ASGCT PresentationsHomology’s first-ever data from its GTx-mAb platform in the presentation “Transducing the Liver as an Antibody Factory using AAVHSCs” showed: Proprietary vector containing a full-length antibody against C5 was delivered via AAVHSCs and engineered to express in the liverExpression levels in the serum of two mouse models were consistent with C5 antibody therapeuticsSustained and robust immunoglobulin G (IgG) expression in vivo for the duration of the study in mice (up to 20 weeks)In vivo expression of C5mAb had potent functional activity, as shown in an ex vivo hemolytic assay In the presentation “Long-Term Expression of HMI-203: Investigational Gene Therapy Candidate for Mucopolysaccharidosis Type II (MPS II), or Hunter Syndrome,” a single I.V. dose of HMI-203 in the murine model: Demonstrated long-term transduction and expression in the brain, kidney, heart, lung, liver and spleenResulted in sustained secretion of iduronate-2-sulfatase (I2S) into the serumReduced glycosaminoglycan heparan sulfate (GAG-HS) in all tissues testedAchieved phenotypic correction of joints and skeletal featuresSignificantly reduced GAG-HS levels in cerebrospinal fluid (CSF) at all doses at 12 weeks post dose In the presentation “Investigational Genetic Medicine Approaches for Phenylketonuria (PKU),” a single I.V. dose of AAVHSC15-based gene therapy or gene editing resulted in a sustained reduction of phenylalanine (Phe) in a murine model of PKU on a normal diet, supporting the potential to address the underlying cause of PKU in individuals with either a fully developed or growing liver. In the study, the AAVHSCs: Achieved a sustained reduction of Phe in PAHenu2 mice on a normal dietIntegrated into the human target PAH locus with the human-specific gene editing vectorShowed integration levels that were similar with the human- and mouse-specific gene editing vectors and at rates that have resulted in sustained reduction of serum Phe in a murine model In “Functional Characterization of AAVHSCs Compared to AAV serotypes: Activation of Cellular Pathways In Vitro and In Vivo Transduction Properties,” the characterization of AAVHSCs compared to AAV capsids 1-8 in human induced pluripotent stem cells (iPSC) showed AAVHSCs: Did not significantly elicit p53-mediated cell death or impact cell division (AAVHSC9/15/16/17) compared to non-Clade F vectorsDid not alter the cell cycle and cell division in iPSC or primary human fibroblasts (AAVHSC15) compared to non-Clade F vectorsHad different intracellular transport and nuclear accumulation kinetics (AAVHSC15) compared to AAV2Were determined to be the most efficient liver transducers, alongside AAV8 and AAV9, among all capsids tested as determined by luciferase expression and vector genomesResulted in higher vector genomes and luciferase expression at the F8 locus when tested in vivo in mice compared to mice transduced with AAV6 On the manufacturing front, “Next Generation AAV Drug Products: Enhanced Stability & Clinical Ease for High Titer Preparations” demonstrated the marked stability of AAVHSCs in the liquid state and the impact of novel formulations on capsid stability, which: Achieved concentrations in excess of 1E14 vg/mL and demonstrated stability for a minimum of one year at 2-8°C and more than six months at room temperature, reducing the need for -80°C-storage infrastructure and simplifying the clinical supply chain by enabling 2-8°C storage at the manufacturing site and clinical pharmacy In “Wildtype AAV2 Rep Protein Produces Higher Titer AAVHSC Vectors with Improved Packaging Profiles Compared to Clade F Associated Chimeric Rep,” wildtype AAV2 Rep protein was shown to be superior when compared with Clade F associated chimeric Rep (AAV9) protein. Vector produced with WT AAV2 Rep resulted in: Increased productivity in both adherent and suspension platforms across multiple AAV genome constructs, with an up to 88% increase in vector genomes and 40% increase in calculated full capsidsIncreased productivity across multiple production scalesImproved or consistent quality attributes in vector packaging profile, infectivity and purity In “Gene Therapy Candidate for Metachromatic Leukodystrophy (MLD): Summary of Preclinical In Vivo Data Following an Intravenous Delivery of HMI-202,” a single I.V. dose of HMI-202 showed a direct motor benefit in MLD mice as evidenced by distribution patterns required to show a functional effect. In the MLD mouse model, data showed: Long-term transduction, expression and ARSA activity in the peripheral and brain tissuesReduced sulfatides to normal levels in the brainPhenotypic rescue in the rotarod assay In non-human primates (NHPs): AAVHSCs crossed the blood-brain barrier (BBB) and blood-nerve barrier (BNB) with expression in relevant cell types For more information about the presentations, visit Homology’s website at www.homologymedicines.com/publications. Webcast/Conference CallIn addition to the presentations at ASGCT 2021, Homology management will host a conference call and webcast at 8:15 a.m. ET to discuss the new GTx-mAb platform and share data highlights from the Company’s pipeline. The webcast will be accessible on Homology’s website in the Investors section, and the webcast replay will be available on the website for 90 days following the presentation. To access using the conference call line, dial (866) 244-8091 (U.S./Canada toll-free) or (602) 563-8623, with Conference ID 5548861. About Homology Medicines, Inc. Homology Medicines, Inc. is a clinical-stage 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. The Company’s intellectual property covers its family 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 StatementsThis 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 the potential of our gene therapy and gene editing platforms, including our new GTx-mAb platform; our plans to name a development candidate in a new therapeutic area and potential thereof; our plans and timing for the release of additional preclinical and clinical data and to name a developmental candidate and potential thereof; 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; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; 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 quarter ended March 31, 2021 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 ContactsTheresa McNeelyChief Communications Officer and Patient Advocatetmcneely@homologymedicines.com781-301-7277 Media Contact:Marisa CitranoSenior Corporate Communications Associatemcitrano@homologymedicines.com617-335-2841

  • Homology Medicines (FIXX) Reports Q1 Loss, Tops Revenue Estimates
    Zacks

    Homology Medicines (FIXX) Reports Q1 Loss, Tops Revenue Estimates

    Homology Medicines (FIXX) delivered earnings and revenue surprises of 96.92% and 3681.29%, respectively, for the quarter ended March 2021. Do the numbers hold clues to what lies ahead for the stock?

  • Homology Medicines Reports First Quarter 2021 Financial Results and Recent Highlights
    GlobeNewswire

    Homology Medicines Reports First Quarter 2021 Financial Results and Recent Highlights

    - On Track to Report Initial Phase 2 Data From pheNIX PKU Clinical Trial by Year End - - Announced New GTx-mAb Platform With Proof of Concept Data to Be Presented at Upcoming ASGCT Annual Meeting - - Presented Data Demonstrating Nuclease-Free Gene Editing of Retinal Cells in Two Targets - - Strengthened Financial Position - BEDFORD, Mass., May 06, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic medicines company, announced today financial results for the first quarter ended March 31, 2021, and highlighted recent accomplishments. “We are in the midst of a transformational time at Homology as we continue to mature and expand our genetic medicines platform and we are on track to report initial Phase 2 data from our pheNIX clinical trial and move two additional programs into the clinic this year,” stated Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “We were excited to unveil our new GTx-mAb platform last week, from which we will feature data that demonstrated proof of principle targeting complement protein 5 with a one-time treatment in a humanized murine model, alongside data from our multiple gene therapy and gene editing programs at the ASGCT Annual Meeting next week. The GTx-mAb platform expands our capability to address diseases with unmet needs, including those with larger patient populations.” Dr. Tzianabos continued, “With multiple catalysts ahead of us this year and next, we recently completed a modest financing to ensure that we are able to keep pace with our advancing programs, and importantly, remain focused on continuing to achieve our goals. We plan to provide an update from our pipeline and showcase highlights from our presentations at ASGCT next week in a conference call and webcast.” First Quarter 2021 and Recent Accomplishments Progressed the dose expansion phase of the Phase 1/2 pheNIX trial evaluating HMI-102 gene therapy for the treatment of adults with phenylketonuria (PKU), which has the potential to be converted to a registrational trial. Following encouraging clinical data from the dose-escalation phase of pheNIX, Homology continued to engage additional sites to enroll patients. Unveiled the Company's new GTx-mAb platform, which leverages its AAVHSCs to deliver vectors at a high efficiency to the liver and secrete antibodies throughout the body through a one-time administration of in vivo gene therapy, as shown in a humanized murine model. Proof of concept data targeting complement protein 5 (C5) that support this platform, in addition to new data from across the Company’s programs, including PKU, MPS II (Hunter syndrome) and metachromatic leukodystrophy (MLD), and advancements from its internal GMP manufacturing process and platform, are the subject of seven presentations, including two oral presentations, at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting May 11-14. The Company plans to name a development candidate in a new therapeutic area from the GTx-mAb platform in 2021.Shared the first-ever data from the Company’s nuclease-free gene editing technology platform that demonstrated the ability of AAVHSCs to transduce relevant cell types in both non-human primate (NHP) and human cells and nuclease-free gene editing in two ophthalmic targets in human retinal explants in a co-presentation with Novartis at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting. An evaluation of 11 AAVHSC capsids from in vivo studies with NHPs and ex vivo studies with human retinal cells following a single intravenous (I.V.) or subretinal dose, respectively, found that: All capsids crossed the blood-retinal and blood-brain barriers in NHPs.AAVHSC15 achieved cross-species transduction of human and NHP photoreceptor cells.Molecular methods confirmed seamless editing in two independent loci and detection of hybrid transcript in human retinal cells. Highlighted data from Homology’s PKU and Hunter syndrome gene therapy programs in presentations at the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, including presentations on the pheNIX trial and the Company’s development candidate for Hunter syndrome, HMI-203. The Company expects to initiate Phase 1/2 dose-escalation clinical trials with HMI-203 and its in vivo gene editing candidate for PKU, HMI-103, in 2021.Showcased new and long-term data from IND-enabling studies for Homology’s central nervous system platform, including HMI-203 and the gene therapy development candidate HMI-202 for MLD, respectively, in presentations at the 17th Annual WORLDSymposium™ Meeting.Participated in patient advocacy-focused events, including Cure MLD’s Standards of Care Conference and Project Alive’s Hunter syndrome Community Conference.Completed a $50.0 million underwritten public offering of shares of common stock in April, strengthening Homology’s financial position in support of key development milestones anticipated in each of the Company’s programs.Promoted Tim Kelly to Chief Operating Officer. First Quarter 2021 Financial Results Net loss for the quarter ended March 31, 2021 was $(1.1) million or $(0.02) per share, compared to a net loss of $(35.3) million or $(0.78) per share for the same period in 2020.Collaboration revenues for the quarter ended March 31, 2021 were $29.3 million, compared to $0.6 million for the quarter ended March 31, 2020. Collaboration revenues for the three months ended March 31, 2021 includes the recognition of approximately $28.5 million of deferred revenue and reimbursement of R&D expenses under the Company’s strategic collaboration with Novartis, which Novartis decided to conclude in February 2021 following a portfolio review. Also included in collaboration revenues is revenue recognized under the Company’s stock purchase agreement with Pfizer.Total operating expenses for the quarter ended March 31, 2021 were $30.4 million, compared to $37.1 million for the quarter ended March 31, 2020, and consisted of research and development expenses and general and administrative expenses.Research and development expenses for the quarter ended March 31, 2021 were $21.8 million, compared to $29.3 million for the quarter ended March 31, 2020. Research and development expenses decreased due to higher expenses in the prior year related to the accelerated procurement of raw materials for manufacturing drug product for the Phase 1/2 pheNIX clinical trial and other development programs as part of Homology’s risk mitigation efforts in response to the COVID-19 pandemic. Additionally, the continued optimization of the Company’s ‘plug and play’ manufacturing process and platform has created greater than 50% efficiencies in subsequent programs that directly reduced spend for clinical trial materials. Costs incurred with Homology’s contract research organization (CRO) to conduct and manage the pheNIX trial with HMI-102 continued to increase, as well as personnel costs to support ongoing development programs, research initiatives, technology platforms and manufacturing capabilities.General and administrative expenses for the quarter ended March 31, 2021 were $8.7 million, compared to $7.8 million for the quarter ended March 31, 2020. General and administrative expenses increased due to personnel costs as a result of new hires, increased audit and legal costs and increased costs associated with expanded operations.As of March 31, 2021, Homology had approximately $188.6 million in cash, cash equivalents and short-term investments. Based on current projections, Homology expects cash resources, together with the approximately $49.4 million in net proceeds received from a follow-on offering of its common stock in April 2021, to fund operations into the first quarter of 2023. Upcoming Events American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting: May 11-14Homology Medicines Corporate & Pipeline Update Conference Call & Webcast: May 13RBC Capital Markets Healthcare Conference: May 18-19Bank of America Securities 2021 Napa Biopharma Virtual Conference: June 14-16National PKU Alliance 2021 Virtual Mini-Conference: July 9-10 About Homology Medicines, Inc. Homology Medicines, Inc. is a clinical-stage 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. The Company’s intellectual property covers its family 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; the potential of our gene therapy and gene editing platforms, including our new GTx-mAb platform; our plans to name a development candidate in a new therapeutic area and potential thereof; plans and timing for the release of additional preclinical and clinical data, including initial Phase 2 data from the pheNIX clinical trial; 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; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; 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 quarter ended March 31, 2021 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. - Financial Tables Follow - HOMOLOGY MEDICINES, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(in thousands, except share and per share amounts)(Unaudited) Three months ended March 31, 2021 2020 Collaboration revenue $29,305 $588 Operating expenses: Research and development 21,755 29,310 General and administrative 8,661 7,770 Total operating expenses 30,416 37,080 Loss from operations (1,111) (36,492)Other income: Interest income 38 1,161 Total other income 38 1,161 Net loss $(1,073) $(35,331)Net loss per share-basic and diluted $(0.02) $(0.78)Weighted-average common shares outstanding-basic and diluted 50,363,579 45,151,265 HOMOLOGY MEDICINES, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands) (Unaudited) As of March 31, 2021 December 31, 2020 Cash, cash equivalents and short-term investments $188,649 $217,431 Property and equipment, net 36,153 37,002 Right-of-use assets 5,618 5,897 Other assets 4,277 3,407 Total assets $234,697 $263,737 Accounts payable, accrued expenses and other liabilities $10,301 $14,525 Operating lease liabilities 14,821 15,442 Deferred revenue 8,746 37,775 Stockholders' equity 200,829 195,995 Total liabilities and stockholders' equity $234,697 $263,737 Company ContactsTheresa McNeelyChief Communications Officerand Patient Advocatetmcneely@homologymedicines.com781-301-7277 Media Contact:Marisa CitranoSenior Corporate Communications Associatemcitrano@homologymedicines.com617-335-2841