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Homology Medicines Reports Second Quarter 2020 Financial Results and Provides Business Update

Three Cohorts Enrolled in Phase 1/2 pheNIX Gene Therapy Trial for PKU

Progressed Into Later Stages of IND-Enabling Studies for MLD Gene Therapy and PKU Gene Editing Programs and Published Key Data

Successfully Executed Multiple Internal 2,000-Liter Bioreactor Runs Using Commercial Process and Platform

BEDFORD, Mass., August 10, 2020 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today financial results for the second quarter ended June 30, 2020, highlighted recent accomplishments and provided a business update.

“The pheNIX trial for adult patients with PKU is ongoing, and we continue to be encouraged by both the clinical data suggesting enzymatic activity and the safety profile of HMI-102. We have dosed patients in three cohorts in the dose-escalation phase of our trial, and this has taken more time than we anticipated due to the pandemic. We continue to assess the data, which includes a recently dosed patient, before we select the dose for our expansion phase. As a result, we are extending our timeline and still expect to provide an update once we have selected a dose for the expansion phase, which has the potential to be a registrational trial.” “In the second quarter, we achieved a milestone in our nuclease-free gene editing program with the peer-reviewed publication of the quantitative molecular methods that we used to show the precision and efficiency of our technology in preclinical studies of PKU. We also announced longer-term data demonstrating that our MLD gene therapy candidate produced durable human ARSA protein at normal levels in advanced IND-enabling studies. Additionally, we demonstrated that our commercial manufacturing process in a 2,000-liter bioreactor resulted in high quality and productivity, and that our AAVHSC vectors packaged more efficiently than non-Clade F AAV5,” concluded Dr. Tzianabos.

Second Quarter 2020 and Recent Accomplishments

  • Announced today an update to the Phase 1/2 pheNIX gene therapy clinical trial with HMI-102 for adults with phenylketonuria (PKU):
    • Since the Company’s initial trial data reported in December 2019:
      • Homology continued to observe encouraging clinical data that suggest PAH enzymatic activity.
      • High-dose Cohort 3 patients were enrolled and dosed.
      • No treatment-related serious adverse events were reported.
    • Homology is extending the timeline for the dose-escalation portion of the trial before selecting a dose for the expansion phase of the trial, and plans to provide an update when the dose is chosen.
    • As previously stated, Homology has all the expected supply on-hand for the dose-escalation and expansion phases of the pheNIX trial and continues to produce supply for the pivotal trial in its internal manufacturing facility.
  • Progressed IND-enabling studies with HMI-103, Homology’s in vivo, nuclease-free gene editing candidate for pediatric PKU, and published methods developed to show the program’s on-target efficiency and precision.
    • Multiple molecular methods showed seamless integration of the human PAH gene into the genome of human hepatocytes in a murine model without any unintended on-target mutations or viral insertions.
    • Gene integration levels in the humanized liver model were consistent with efficiencies seen following administration to the PKU Pahenu2 murine model, which corrected the disease phenotype.
    • Results were peer-reviewed and published in PLOS ONE and data were presented at the American Society of Gene & Cell Therapy (ASGCT) Meeting.
  • Executed additional 2,000-liter bioreactor runs, with multiple product candidates, in Homology’s internal facility using its commercial process and platform, and new data were presented at the ASGCT Meeting which demonstrated that:
    • AAVHSCs were suitable for packaging a wide range of genome sizes and yielded more intact packaged genomes than AAV5, a non-Clade F serotype.
    • Homology’s commercial process and internal manufacturing platform performs at the 2,000-liter bioreactor scale with similar high quality and productivity as smaller scale runs in the same facility.
  • Progressed into late-stage IND-enabling studies with in vivo central nervous system (CNS) gene therapy candidate HMI-202 for metachromatic leukodystrophy (MLD), and presented data at the ASGCT Meeting.
    • I.V. HMI-202 demonstrated durable 52-week expression of human ARSA levels in the MLD murine model that met or exceeded normal human brain ARSA activity levels.
    • Data expanded on prior studies that demonstrated HMI-202 crossed the blood-brain and blood-nerve barriers in murine and non-human primate models and impacted key disease biomarkers in the MLD murine model.
  • Highlighted the potential of Homology’s dual gene therapy and gene editing platform in additional presentations on the differentiating features of the Company’s AAVHSC vectors.
  • Continued to monitor the ongoing COVID-19 pandemic and updated business continuity plans, as needed.
    • Homology expanded home-health services, home visits and centralized lab testing for pheNIX clinical trial participants.
    • Employees followed shift schedules for requisite on-site work in Homology’s laboratories and manufacturing facility and a work-from-home policy for all others.
    • Homology developed on-site COVID-19 and antibody testing for all employees to promote health and safety, which is ongoing.
    • Procured additional raw materials for the manufacturing of drug product for the pheNIX trial ahead of the normal purchasing cycle to mitigate potential supply chain interruptions. 

Second Quarter 2020 Financial Results

  • Net loss for the quarter ended June 30, 2020 was $(35.3) million or $(0.78) per share, compared to a net loss of $(26.3) million or $(0.61) per share for the same period in 2019.
  • Collaboration revenues for the quarter ended June 30, 2020 were $0.6 million, compared to $0.4 million for the quarter ended June 30, 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 an ophthalmic program and seek to identify new targets for the collaboration based on the exploratory research component.
  • Total operating expenses for the quarter ended June 30, 2020 were $36.3 million, compared to $28.4 million for the quarter ended June 30, 2019, and consisted of research and development expenses and general and administrative expenses.
  • Research and development expenses for the quarter ended June 30, 2020 were $27.5 million, compared to $22.8 million for the quarter ended June 30, 2019. Research and development expenses increased primarily 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 June 30, 2020 were $8.8 million, compared to $5.5 million for the quarter ended June 30, 2019. General and administrative expenses increased primarily due to personnel costs as a result of new hires, increased consulting costs and additional costs associated with expanded operations.
  • As of June 30, 2020, Homology had approximately $206.6 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

  • BTIG Virtual Biotechnology Conference – Fireside Chat – August 11 at 10:30 a.m. ET
  • Canaccord Genuity 40th Annual Growth Conference – Fireside Chat – August 13 at 2:00 p.m. ET
  • Baird Global Healthcare Conference – September 9 at 12:15 p.m. ET
  • H.C. Wainwright & Co. 22nd Annual Global Investment Conference – September 14-16
  • Chardan’s 4th Annual Genetic Medicines Conference – October 5-6
  • Cell & Gene Meeting on the Mesa – October 14-16

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

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 for the release of clinical data from the Phase 1/2 pheNIX trial, including the dose-escalation phase and Part B expansion part; selection of a dose for the Part B expansion phase; 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 June 30, 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

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