Homology Medicines Reports First Quarter 2023 Financial Results and Recent Highlights

  • On Track to Report Initial Clinical Data from pheEDIT PKU Gene Editing Trial Mid-Year 2023
  • First Presentations of Preclinical Data with GTx-mAb Development Candidate HMI-104 for PNH, and Potential for Re-Dosing with AAVHSCs, at Upcoming ASGCT Meeting
  • Solid Financial Position with Anticipated Cash Runway into Fourth Quarter 2024

BEDFORD, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today financial results for the first quarter ended March 31, 2023, and highlighted recent accomplishments.

“We look forward to announcing the first gene editing data in people with PKU, as we are on track to report initial clinical data mid-year 2023 from our pheEDIT trial evaluating HMI-103,” said Albert Seymour, Ph.D., President and Chief Executive Officer of Homology Medicines. “I am pleased to share that the second participant was dosed in the pheEDIT trial, a third participant is in screening and there continues to be strong interest from the patient and physician community in this one-time gene editing approach for PKU.”

Continued Dr. Seymour, “We will have a big presence at ASGCT next week with presentations spanning our genetic medicines platform, including new preclinical data with the anti-C5 GTx-mAb development candidate, HMI-104 for PNH, which demonstrated sustained expression of functional C5 monoclonal antibody levels in multiple in vivo models. We will also present the first preclinical data sets demonstrating the potential to re-dose with AAVHSCs, as well as methods to identify genomic sites with improved gene editing integration, which highlight our commitment to scientific innovation in gene editing and gene therapy.”

First Quarter 2023 and Recent Accomplishments

  • Announced that the second participant has been dosed in the Phase 1 pheEDIT clinical trial evaluating in vivo nuclease-free gene editing candidate HMI-103 in adults with phenylketonuria (PKU), and a third participant is advancing through screening to complete the first dose cohort of the trial. Homology continues to expect initial clinical data from the trial mid-year 2023.
  • Continued recruitment efforts for the Phase 1 juMPStart trial evaluating in vivo HMI-203 gene therapy candidate in adults with Hunter syndrome (MPS II).
  • Progressed HMI-104, a C5 monoclonal antibody development candidate for paroxysmal nocturnal hemoglobinuria (PNH), through IND-enabling studies.
  • Published work on Homology’s metachromatic leukodystrophy (MLD) gene therapy program in the peer-reviewed Journal of Neuroscience.
  • Shared that six presentations on Homology’s gene editing, gene therapy and GTx-mAb programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, featuring:
    • IND-enabling studies with GTx-mAb candidate HMI-104 for PNH, and preclinical data with HMI-204 for MLD;
    • Preclinical data showing potential for re-dosing with AAVHSCs across different clades;
    • Ocular biodistribution of AAVHSCs in multiple preclinical models and routes of administration;
    • Preclinical support for the targeted immunosuppression regimen utilized in Homology’s ongoing gene editing and gene therapy clinical trials; and
    • Methodology for identifying genomic sites with improved gene editing efficiency.

First Quarter 2023 Financial Results

  • Net loss for the quarter ended March 31, 2023 was $(28.8) million or $(0.50) per share compared to net income of $92.1 million or $1.61 per share for the quarter ended March 31, 2022. Diluted net (loss) per share was $(0.50) compared to diluted net income per share of $1.59 for the same period in 2022. Net income for the three months ended March 31, 2022 was primarily due to a gain of $131.2 million recognized on the sale of the Company’s manufacturing operations.
  • Collaboration revenue was $0.8 million in each of the quarters ended March 31, 2023 and 2022. Collaboration revenue in both periods reflects revenue recognized under the Company’s Stock Purchase Agreement with Pfizer.
  • Total operating expenses for the quarter ended March 31, 2023 were $28.3 million, compared to $38.4 million for the quarter ended March 31, 2022, and consisted of research and development expenses and general and administrative expenses.
  • Research and development expenses for the quarter ended March 31, 2023 were $20.0 million, compared to $24.3 million for the quarter ended March 31, 2022. Research and development expenses decreased primarily due to lower employee-related costs as a result of transferring employees to OXB Solutions upon the sale of the Company’s manufacturing operations to Oxford in March 2022, along with decreased external development costs related to HMI-102 as the pheNIX trial was placed on clinical hold in February 2022 and enrollment was paused in August 2022. Partially offsetting these decreases was increased external development costs related to development-stage programs including higher spending on HMI-104, the Company’s GTx-mAb product candidate for PNH. External development costs for the Company’s HMI-203 and HMI-103 clinical programs also increased.
  • General and administrative expenses for the quarter ended March 31, 2023 were $8.3 million, compared to $14.1 million for the quarter ended March 31, 2022. The decrease in general and administrative expenses was largely due to higher professional fees incurred in the prior year in connection with the OXB transaction, which included strategic advisory, legal and audit fees.
  • As of March 31, 2023, Homology had approximately $150.0 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 2024.

Upcoming Events

  • ASGCT 26th Annual Meeting: May 16-20
  • World Orphan Drug Congress USA 2023: May 23-25

About Homology Medicines, Inc.
Homology Medicines, Inc. is a clinical-stage genetic medicines company dedicated to transforming the lives of patients suffering from rare diseases by addressing the underlying cause of the disease. The Company’s clinical programs include HMI-103, a gene editing candidate for phenylketonuria (PKU); HMI-203, an investigational gene therapy for Hunter syndrome; and HMI-102, an investigational gene therapy for adults with PKU. Additional programs focus on paroxysmal nocturnal hemoglobinuria (PNH), metachromatic leukodystrophy (MLD) and other diseases. Homology’s proprietary platform is designed to utilize its family of 15 human hematopoietic stem cell-derived adeno-associated virus (AAVHSCs) vectors to precisely and efficiently deliver genetic medicines in vivo through a nuclease-free gene editing modality, gene therapy, or GTx-mAb, which is designed to produce antibodies throughout the body. Homology established an AAV manufacturing and innovation business in partnership with Oxford Biomedica, which was based on Homology’s internal process development and manufacturing platform. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a focus on rare diseases. Homology believes its initial clinical data and compelling preclinical data, scientific and product development expertise and broad intellectual property position the Company as a leader in genetic medicines. For more information, visit www.homologymedicines.com.

Forward-Looking Statements
This press release contains forward-looking statements. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. 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 GTx-mAb platform; our plans and timing for the release of additional preclinical and clinical data; our plans to progress our pipeline of genetic medicine candidates and the anticipated timing for these milestones; 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. The words “believe,” “may,” “will,” “estimate,” “potential,” “continue,” “anticipate,” “intend,” “expect,” “could,” “would,” “project,” “plan,” “target,” and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions. 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 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, including for the manufacture of materials for our research programs, preclinical and clinical studies; 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; securities class action litigation; the impact of the COVID-19 pandemic and general economic conditions on our business and operations, including our preclinical studies and clinical trials; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property; and significant costs incurred 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, 2023 and our other filings with the Securities and Exchange Commission 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 -

(in thousands)  
    As of  
    March 31, 2023   December 31, 2022  
Cash, cash equivalents and short-term investments   $ 150,025   $ 175,026  
Equity method investment     23,036     25,814  
Property and equipment, net     1,062     1,078  
Right-of-use assets     20,200     20,563  
Other assets     3,558     5,989  
Total assets   $ 197,881   $ 228,470  
Accounts payable, accrued expenses and other liabilities   $ 16,527   $ 19,859  
Operating lease liabilities     29,101     29,477  
Deferred revenue     354     1,156  
Stockholders' equity     151,899     177,978  
Total liabilities and stockholders' equity   $ 197,881   $ 228,470  
(in thousands, except share and per share amounts)
    Three months ended March 31,
      2023       2022  
Collaboration revenue   $ 802     $ 802  
Operating expenses:        
Research and development     19,988       24,273  
General and administrative     8,325       14,147  
Total operating expenses     28,313       38,420  
Loss from operations     (27,511 )     (37,618 )
Other income:        
Gain on sale of business           131,249  
Interest income     1,469       32  
Total other income     1,469       131,281  
Income (loss) before income taxes     (26,042 )     93,663  
Provision for income taxes           (967 )
Loss from equity method investment     (2,802 )     (591 )
Net income (loss)   $ (28,844 )   $ 92,105  
Net income (loss) per share-basic   $ (0.50 )   $ 1.61  
Net income (loss) per share-diluted   $ (0.50 )   $ 1.59  
Weighted-average common shares outstanding-basic     57,716,344       57,279,963  
Weighted-average common shares outstanding-diluted     57,716,344       57,875,576  

Company Contacts:
Cara Mayfield
Vice President, Patient Advocacy
and Corporate Communications

Investor Contact:
Bill Slattery, Jr.
Vice President, Investor Relations

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