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Homology Medicines Reports Second Quarter 2019 Financial Results and Recent Highlights

- Phase 1/2 pheNIX Study of HMI-102 Gene Therapy Candidate for Adults with PKU Continues, Additional Sites Enrolling and Initial Data Expected by Year End -

- Fast Track Designation Granted in the U.S. for HMI-102, Adding to Orphan Drug Designation in Both the U.S. and the E.U. -

BEDFORD, Mass., August 8, 2019 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today financial results for the quarter ended June 30, 2019, and highlighted recent accomplishments.

“We initiated our Phase 1/2 gene therapy trial for PKU in June, and we remain on track to report initial clinical data on HMI-102 by the end of this year,” said Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “Our next gene therapy program and our first gene editing program are advancing through IND-enabling studies, and we plan to share details on these programs later in the year.”

Dr. Tzianabos added, “On the manufacturing front, we have run multiple GMP lots in our own facility and are operating on a commercial GMP gene therapy and gene editing platform that can supply our trial all the way through to commercial scale. This capability is an important strategic advantage for Homology as we progress our lead program in PKU in the clinic and advance our pipeline. The potential to expedite our PKU gene therapy program requires a strong financial position. Therefore, we strengthened our balance sheet in the second quarter by raising $144 million from existing investors and attracting support from new, top-tier funds.”

Second Quarter 2019 and Recent Accomplishments

  • Initiated the Phase 1/2 pheNIX study for HMI-102 gene therapy candidate for adults with phenylketonuria (PKU)
  • Presented data during three oral presentations and two poster sessions at American Society of Gene and Cell Therapy (ASGCT) which demonstrated:
    • In vivo preclinical proof of concept for PKU gene editing showing reduced Phe to therapeutic levels and efficient and selective editing
    • Tolerability of HMI-102 PKU gene therapy candidate in non-clinical studies in multiple species
    • AAVHSCs crossed the blood-brain-barrier and achieved therapeutic enzyme levels in the metachromatic leukodystrophy (MLD) disease model
    • Homology’s scalable HEK293 manufacturing process for AAVHSCs was superior to a baculovirus system and AAVHSC15 showed improved in vivo efficacy compared to AAV5 in PKU model
    • In a 5-year retrospective study of PKU patients at two specialized clinics, phenylalanine levels remained above the well-controlled threshold while on a restricted diet, highlighting the unmet need for new treatment options

These and other data highlights were also presented at key scientific meetings, including the European Society of Human Genetics Conference (ESHG) and ACMG Annual Clinical Genetics meeting.

  • Presented data at the Association for Research in Vision and Ophthalmology (ARVO) 2019 Annual Meeting showing that AAVHSCs target and enter therapeutically relevant cells in the eye in three models using multiple routes of administration, including intravenous, intravitreal and subretinal injections
  • Received Fast Track Designation for HMI-102 from the U.S. Food and Drug Administration (FDA), adding to orphan drug designations for HMI-102 in the United States and European Union
  • Raised $144.0 million in gross proceeds through a public equity offering
  • Appointed Alise Reicin, M.D., President, Global Clinical Development at Celgene Corporation, to the Board of Directors

“We have been pleased by the positive reception we are getting from additional clinical sites eager to participate in our trial, as well as from patients with PKU looking for a new solution,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology. “As a company founded and focused on developing our science into potential cures for the benefit of patients, we continue to present our data at peer-reviewed meetings, as evidenced by the 11 presentations by Homology at scientific conferences during the second quarter alone.”

Second Quarter 2019 Financial Results

  • Net loss for the quarter ended June 30, 2019 was $(26.3) million, or $(0.61) per share, compared to a net loss of $(12.4) million, or $(0.34) per share, for the same period in 2018.
  • Collaboration revenue for the quarter ended June 30, 2019 was $0.4 million, compared to $1.4 million for the quarter ended June 30, 2018, and consisted of revenue recognized under the Company’s strategic collaboration with Novartis. The Company recognizes revenue 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 its exploratory research component. Total operating expenses for the quarter ended June 30, 2019 were $28.4 million, compared to $14.8 million for the same period in 2018, and consisted of research and development expenses and general and administrative expenses.
  • Research and development expenses for the quarter ended June 30, 2019 were $22.8 million, compared to $10.3 million for the same period in 2018. The increase of $12.5 million was due to a rise in direct research expenses, including contract manufacturing costs related to our HMI-102 program, increased direct research expenses related to HMI-202 and HMI-103 external development costs, increased personnel costs to support ongoing development programs, research initiatives, technology platform and manufacturing capabilities, as well as increased expenses related to laboratory supplies, research materials and services for the further advancement of early-stage research programs.
  • General and administrative expenses for the quarter ended June 30, 2019 were $5.5 million, compared to $4.5 million for the same period in 2018. The increase of $1.0 million was due to increased personnel costs as a result of new hires and increased costs associated with expanded operations.
  • As of June 30, 2019, Homology had approximately $295.8 million in cash, cash equivalents and short-term investments, which the Company expects will fund operations into the fourth quarter of 2021.

Upcoming Events

  • BTIG Biotechnology Conference: August 12, 2019, in New York, NY
  • Citi’s Annual Biotech Conference: September 4–5, 2019, in Boston, MA
  • H.C. Wainwright Annual Healthcare Conference: September 8–10, 2019, in New York, NY

About the Phase 1/2 pheNIX Clinical Study in PKU
Homology’s proprietary suite of human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) enabled the selection of AAVHSC15 to efficiently deliver a functional copy of the phenylalanine hydroxylase (PAH) gene to the liver cells, where there is a missing or mutated PAH gene. This in vivo gene therapy approach is intended to enable the production of the PAH enzyme responsible for metabolizing Phe. People with PKU are not able to metabolize Phe properly, resulting in significantly elevated and potentially toxic levels of Phe, and if left untreated, PKU can lead to severe neurological impairment. Phe reduction is an established clinical endpoint for PKU registrational trials. Homology is conducting a Phase 1/2 gene therapy study of HMI-102 in adults with PKU, called pheNIX. Additional information about the pheNIX study can be found at

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; 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; and the sufficiency of our cash, cash equivalents and short-term investments. 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 Quarterly Report on Form 10-Q for the quarter ended June 30, 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.

- Financial Tables Follow –

Investor Contact:
Theresa McNeely
SVP, Corporate Communications and Patient Advocacy

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