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Homology Medicines Reports First Quarter 2020 Financial Results and Recent Highlights

- 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 7, 2020 – 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

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.

Company Contacts:
Theresa McNeely
Chief Communications Officer and Patient Advocate

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