Homology Medicines Reports Third Quarter 2022 Financial Results and Recent Highlights
- On Track for Updates on pheEDIT and juMPStart Programs by Year-End -
- Strong Financial Position with Cash Runway into Fourth Quarter 2024 -
“We remain on track to provide an update from our pheEDIT and juMPStart clinical trials, including site initiations and enrollment status, by the end of this year,” said
Third Quarter 2022 and Recent Highlights
- Announced a pipeline prioritization, which included shifting resources from the pheNIX gene therapy trial evaluating HMI-102 for phenylketonuria (PKU) and pausing enrollment to focus on generating data from the pheEDIT clinical trial that is evaluating in vivo gene editing candidate HMI-103 for PKU. The mechanism of action of this gene editing candidate has the potential to treat adults and ultimately pediatric patients.
- Continued to advance the juMPStart clinical trial evaluating in vivo gene therapy candidate HMI-203 for MPS II, or Hunter syndrome. Homology remains on track to provide an update on enrollment and site status for this trial and the pheEDIT trial by the end of this year.
- Continued to advance IND-enabling studies of HMI-104, a C5 antibody development candidate for paroxysmal nocturnal hemoglobinuria (PNH), which utilizes the Company’s GTx-mAb platform that has the potential to address larger market indications.
- Announced intention to partner the optimized in vivo gene therapy candidate HMI-204 for metachromatic leukodystrophy (MLD), which demonstrated the ability to cross the blood-brain-barrier as well as reach the central nervous system and key peripheral organs involved in MLD following a single I.V. administration.
- Presented design of the pheEDIT gene editing trial for HMI-103 and supporting preclinical data, as well as data that demonstrated the use of next-generation sequencing methods that can lead to improved AAVHSC vector design, which were featured at the
American Society of Human Genetics Meeting.
Albert Seymour, Ph.D., to Chief Executive Officer of Homology and to the Board of Directors, succeeding Arthur Tzianabos, Ph.D., who served as CEO and Board member since 2016 and who was appointed Chair of the Board in conjunction with this transition.
- Announced peer-reviewed publication in Scientific Reports, a Nature journal, on the use of long-read sequencing methods that could be applied to further characterize Homology’s AAVHSCs and optimize vector design.
- Spoke alongside industry leaders on panels focused on innovations in gene editing and AAV manufacturing during the Cell & Gene Meeting on the Mesa.
Third Quarter 2022 Financial Results
- As of
September 30, 2022, Homology had approximately $201.1 millionin cash, cash equivalents and short-term investments. Based on current projections, Homology expects cash resources to fund operations into the fourth quarter of 2024.
- Net loss for the quarter ended
September 30, 2022was $(33.7) millionor $(0.59)per share, compared to a net loss of $(30.6) millionor $(0.54)per share for the quarter ended September 30, 2021.
- Collaboration revenues for the quarter ended
September 30, 2022were $0.8 million, compared to $1.7 millionfor the quarter ended September 30, 2021. Collaboration revenue in each period included deferred revenue recognized under Homology’s stock purchase agreement with Pfizer. Collaboration revenues for the third quarter 2021 also included the recognition of deferred revenue and reimbursement of R&D expenses under the Company’s former collaboration with Novartis.
- Total operating expenses for the quarter ended
September 30, 2022were $33.7 million, compared to $32.3 millionfor the quarter ended September 30, 2021, and consisted of research and development expenses and general and administrative expenses.
- Research and development expenses for the quarter ended
September 30, 2022were $25.9 million, compared to $24.0 millionfor the quarter ended September 30, 2021. Research and development expenses increased primarily due to increased development costs for our ongoing HMI-103 and HMI-203 clinical-stage programs and earlier-stage programs. Partially offsetting these increases were lower employee-related costs as a result of transferring employees to Oxford Biomedica Solutions, the AAV Innovation and Manufacturing Business that Homology established with Oxford Biomedica in the first quarter of 2022 in order to leverage its in-house manufacturing capabilities while establishing a 20% stake and preferred customer status in the new business.
- General and administrative expenses for the quarter ended
September 30, 2022were $7.8 million, compared to $8.4 millionfor the quarter ended September 30, 2021. General and administrative expenses decreased primarily as a result of finance, human resources, IT and legal services provided by the Company to OXB Solutions under the transitional services agreement that provided for these services to be reimbursed, as well as decreased depreciation expense as the Company’s leasehold improvements were transferred to Oxford Biomedica Solutions. Partially offsetting these decreases was an increase in audit and legal fees.
- Stifel 2022 Healthcare Conference:
November 15, 2022at 4:10 p.m. ET– Fireside Chat
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. 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 position as a leader in the development of genetic medicines; the sufficiency of our cash and cash equivalents to fund our operations; our plans to engage in future collaborations and strategic partnerships; 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: 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 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||$||201,074||$||155,873|
|Assets held for sale||—||28,907|
|Equity method investment||27,132||—|
|Property and equipment, net||1,415||2,252|
|Accounts payable, accrued expenses and other liabilities||$||21,805||$||13,772|
|Operating lease liabilities||29,827||23,934|
|Total liabilities and stockholders' equity||$||262,651||$||211,721|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(in thousands, except share and per share amounts)|
|Three months ended
||Nine months ended
|Research and development||25,854||23,987||71,202||69,439|
|General and administrative||7,810||8,351||29,991||26,054|
|Total operating expenses||33,664||32,338||101,193||95,493|
|Loss from operations||(32,862||)||(30,661||)||(98,787||)||(62,324||)|
|Gain on sale of business||-||—||131,249||—|
|Total other income||1,269||53||133,024||143|
|Income (loss) before income taxes||(31,593||)||(30,608||)||34,237||(62,181||)|
|Benefit from (provision for) income taxes||46||—||(816||)||—|
|Loss from equity method investment||(2,179||)||—||(4,131||)||—|
|Net income (loss)||$||(33,726||)||$||(30,608||)||$||29,290||$||(62,181||)|
|Net income (loss) per share-basic||$||(0.59||)||$||(0.54||)||$||0.51||$||(1.14||)|
|Net income (loss) per share-diluted||$||(0.59||)||$||(0.54||)||$||0.51||$||(1.14||)|
|Weighted-average common shares outstanding-basic||57,447,192||57,106,639||57,372,399||54,704,410|
|Weighted-average common shares outstanding-diluted||57,447,192||57,106,639||57,901,298||54,704,410|
Chief Communications Officer
Vice President, Patient Advocacy
and Corporate Communications
Source: Homology Medicines, Inc.