Homology Medicines Reports Fourth Quarter and Full Year 2022 Financial Results and Recent Highlights
- On Track to Provide Initial Clinical Data from Gene Editing Trial for PKU Mid-Year with First Participant Dosed and Others in Screening -
- Initial Clinical Data from Gene Therapy Trial for Hunter Syndrome Anticipated in Second Half of 2023; Trial Recruiting in the
- Anticipated Cash Runway Into Fourth Quarter 2024 Enables Execution Against Key Milestones -
“We entered 2023 with strong momentum across our gene editing and gene therapy clinical trials for PKU and Hunter syndrome, and we anticipate initial data read-outs from both programs this year,” said
Fourth Quarter 2022 and Recent Accomplishments
- Dosed first participant in the Phase 1 pheEDIT clinical trial evaluating in vivo nuclease-free gene editing candidate HMI-103 in adults with phenylketonuria (PKU); additional participants are in screening. Homology continues to expect initial clinical data from the trial mid-year 2023.
- Shared preclinical data that showed murine surrogate of HMI-103 was ten times more potent than non-integrating gene therapy vector HMI-102 in the murine model of PKU; HMI-103 is designed with a unique mechanism of action (MOA) to maximize PAH enzyme through both genome integration and episomal expression, and it has the potential to treat adults and pediatric patients.
- Building on physician and patient interest, anticipate initial clinical data from the HMI-203 juMPStart gene therapy trial for Hunter syndrome in the second half of 2023.
- Presented data that support the targeted immunosuppressive regimen in Homology’s clinical trials. In non-human primates (NHPs), the combination of a T-cell inhibitor and steroid was most effective in reducing the immune response to AAVHSC and improving gene expression.
- Presented preclinical data with HMI-204, Homology’s optimized, in vivo gene therapy candidate for metachromatic leukodystrophy (MLD). A single I.V. dose in the murine model of MLD led to robust expression in the central nervous system (CNS), including sustained levels of enzyme activity reaching levels of normal human adults and predicted to lead to efficacy in vivo. Homology continues to seek a partner for the optimized product candidate, which is ready to enter IND-enabling studies.
- Progressed HMI-104, a C5 monoclonal antibody (mAb) development candidate for paroxysmal nocturnal hemoglobinuria (PNH), through IND-enabling studies. HMI-104 is the first candidate that utilizes the Company’s GTx-mAb platform and is focused on using the liver to express a C5 mAb with a one-time dose. Homology believes its GTx-mAb platform has the potential to address larger market indications.
- Announced today the promotion of
Julie Jordan, M.D., to Chief Medical Officer.
Fourth Quarter 2022 and Full Year Financial Results
- Net loss for the quarter ended
December 31, 2022was $(34.3) millionor $(0.60)per share, compared to a net loss of $(33.6) millionor $(0.59)per share for the same period in 2021. Net loss for the year ended December 31, 2022was $(5.0) millionor $(0.09)per share, compared to a net loss of $(95.8) millionor $(1.73)per share for the same period in 2021. The decrease in net loss was primarily due to a gain of $131.2 millionrealized in connection with the Company’s sale of its manufacturing business to Oxford Biomedica in order to establish Oxford Biomedica Solutions (“OXB Solutions”), an AAV Innovation and Manufacturing Business, in the first quarter of 2022, partially offset by lower collaboration revenues in 2022.
- Collaboration revenues for the three and twelve months ended
December 31, 2022were $0.8 millionand $3.2 million, respectively, as compared to $0.8 millionand $34.0 millionfor the comparable periods in 2021. Collaboration revenues in 2022 consisted of revenue recognized under the Company’s stock purchase agreement with Pfizer compared with collaboration revenues in 2021, which were primarily the result of concluding the Company’s collaboration with Novartis.
- Total operating expenses for the three and twelve months ended
December 31, 2022were $35.3 millionand $136.5 million, respectively, as compared to $34.4 millionand $129.9 millionfor the comparable periods in 2021, and consisted of research and development expenses and general and administrative expenses.
- Research and development expenses for the three and twelve months ended
December 31, 2022were $27.2 millionand $98.4 million, respectively, as compared to $23.6 millionand $93.1 millionfor the comparable periods in 2021. Research and development expenses increased by $5.3 millionin 2022 primarily due to increases in direct costs of $9.3 millionrelated to pheEDIT and $3.9 millionrelated to juMPStart, as we incurred costs to initiate sites and recruit patients. Additionally, there was a $5.8 millionincrease in direct research expenses related to our other development-stage programs, primarily due to higher spending on HMI-104. Partially offsetting these increases was a $15.6 milliondecrease in employee-related costs as a result of transferring employees to OXB Solutions in order to leverage the Company’s in-house manufacturing capabilities while establishing a 20% ownership stake and preferred customer status in the new business.
- General and administrative expenses for the three and twelve months ended
December 31, 2022were $8.1 millionand $38.1 million, respectively, as compared to $10.8 millionand $36.8 millionfor the comparable periods in 2021. General and administrative expenses increased in 2022 due primarily to professional fees associated with the establishment of OXB Solutions.
- As of
December 31, 2022, Homology had approximately $175.0 millionin cash, cash equivalents and short-term investments. Based on current projections, Homology expects current cash resources to fund operations into the fourth quarter of 2024.
Project Alive Hunter Syndrome Community Conference: March 11at 2:30 p.m. ET
- 2023 ACMG Annual Clinical Genetics Meeting:
March 14 - 18
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 plans to engage in future collaborations and strategic partnerships; 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 and cash equivalents 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
- Financial Tables Follow -
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash, cash equivalents and short-term investments||$||175,026||$||155,873|
|Assets held for sale||—||28,907|
|Equity method investment||25,814||—|
|Property and equipment, net||1,078||2,252|
|Accounts payable, accrued expenses and other liabilities||$||19,859||$||13,772|
|Operating lease liabilities||1,561||246|
|Operating lease liabilities, net of current portion||27,916||23,688|
|Total liabilities and stockholders' equity||$||228,470||$||211,721|
|CONSOLIDATED STATEMENTS OF OPERATIONS|
|(in thousands, except share and per share amounts)|
|For the Three Months Ended
||For the Year Ended
|Research and development||27,149||23,646||98,351||93,085|
|General and administrative||8,147||10,781||38,138||36,835|
|Total operating expenses||35,296||34,427||136,489||129,920|
|Loss from operations||(34,494||)||(33,625||)||(133,281||)||(95,949||)|
|Gain on sale of business||—||—||131,249||—|
|Total other income||1,455||42||134,479||185|
|Income (loss) before income taxes||(33,039||)||(33,583||)||1,198||(95,764||)|
|Benefit from (provision for) income taxes||101||—||(715||)||—|
|Loss from equity method investment||(1,357||)||—||(5,488||)||—|
|Net loss per share-basic and diluted||$||(0.60||)||$||(0.59||)||$||(0.09||)||$||(1.73||)|
|Weighted average common shares outstanding-basic and diluted||57,483,402||57,150,079||57,399,762||55,283,318|
Vice President, Patient Advocacy
and Corporate Communications
Chief Financial and Business Officer
Source: Homology Medicines, Inc.