Homology Medicines Reports First Quarter 2022 Financial Results and Recent Highlights
- Presentations at Upcoming ASGCT Annual Meeting Include First-Time Details of Mechanism of Action of Nuclease-Free Gene Editing Candidate HMI-103 for PKU; Symposium to Feature Lead Principal Investigator for the pheEDIT Trial -
- Additional Presentations on GTx-mAb Platform and Characterization of Proprietary AAVHSC Capsid with Low Liver Tropism -
- Submitted Response to FDA with Information Requested for pheNIX Gene Therapy Trial Clinical Hold -
“During the first quarter of 2022, we took additional steps to pave the way for Homology’s success,” said
First Quarter 2022 and Recent Accomplishments
- Closed the deal with Oxford Biomedica (OXB) to establish an AAV Innovation and Manufacturing Business that incorporated Homology’s technical and manufacturing operations, team and GMP facility. Homology received
$130 millionin non-dilutive capital from OXB and maintains access as a preferred customer to the ‘plug and play’ AAV manufacturing process and platform, as well as shares in potential benefits as a 20% owner of the business.
- Announced upcoming presentations at the
American Society for Gene & Cell Therapy(ASGCT) 25th Annual Meeting May 16-19, 2022, including:
- Several presentations featuring HMI-103 investigational gene editing candidate for phenylketonuria (PKU), including optimization and mechanism of action, and genome-wide integration assays conducted to confirm no off-target editing;
- New information from the GTx-mAb platform, including data that further characterize the expression of C5 antibodies following a one-time administration;
- Characterization of a non-liver-tropic capsid and other details on the characterization of Homology’s family of 15 naturally derived AAVHSCs; and
- A symposium on
Wednesday, May 18at 7:30 a.m. ETto feature guest speaker Jerry Vockley, Ph.D., M.D., FACMG, Division Director, Genetic and Genomic Medicine, Professor of Pediatrics and Human Genetics, and Director, Center for Rare Disease Therapyat the University of Pittsburgh, and Lead Principal Investigator for the pheEDIT HMI-103 trial.
- Announced today that Homology submitted a response to the
U.S. Food and Drug Administration(FDA) regarding the clinical hold on the pheNIX gene therapy trial of HMI-102 in adults with PKU. As previously disclosed, the clinical hold related only to the pheNIX trial and pertained to elevated liver function tests (LFTs) observed in the trial, including a request for modified risk-mitigation measures. Homology plans to provide the next update on pheNIX when the path forward is established with the FDA.
- As part of the response, Homology included a protocol amendment to incorporate the same targeted immunosuppressive regimen in the ongoing pheEDIT clinical trial, which utilizes a T-cell inhibitor, is shorter in duration and reduces the length of steroid therapy. The new regimen is aligned with data that show that T-cell inhibitors dampen the immune response to AAV capsids in the clinical setting. Homology believes the regimen may also increase patient compliance.
- As previously reported, all instances of LFT elevations have resolved, and no hospitalizations were required.
Albert Seymour, Ph.D., to Presidentin addition to his role as Homology’s Chief Scientific Officer.
First Quarter 2022 Financial Results
- Net income for the quarter ended
March 31, 2022was $92.1 millioncompared to a net loss of $(1.1) millionfor the quarter ended March 31, 2021. Net income for the three months ended March 31, 2022was primarily due to a gain of $131.2 millionon the sale of the Company’s manufacturing operations, offset by operating expenses of $38.4 million. Diluted net income per share was $1.59compared to diluted net (loss) per share of ( $0.02) for the same period in 2021.
- Collaboration revenues for the quarter ended
March 31, 2022were $0.8 million, compared to $29.3 millionfor the quarter ended March 31, 2021. Collaboration revenues for the three months ended March 31, 2021included the recognition of approximately $28.5 millionof deferred revenue and reimbursement of R&D expenses as a result of concluding the Company’s collaboration with Novartis. Also included in collaboration revenues in both periods is revenue recognized under the Company’s Stock Purchase Agreement with Pfizer.
- Total operating expenses for the quarter ended
March 31, 2022were $38.4 million, compared to $30.4 millionfor the quarter ended March 31, 2021, and consisted of research and development expenses and general and administrative expenses.
- Research and development expenses for the quarter ended
March 31, 2022were $24.3 million, compared to $21.8 millionfor the quarter ended March 31, 2021. Research and development expenses increased due to higher external development costs for earlier stage programs, specifically related to the development of HMI-104, Homology’s GTx-mAb product candidate for PNH, and HMI-203, the Company’s investigational gene therapy in development for the treatment of adults with Hunter syndrome. Employee-related costs also increased due to additional employee headcount to support ongoing development programs and research initiatives, which resulted in increases in salaries, payroll taxes and stock-based compensation expense. Partially offsetting these increases was decreased direct research expenses for HMI-102 as the trial was placed on clinical hold.
- General and administrative expenses for the quarter ended
March 31, 2022were $14.1 million, compared to $8.7 millionfor the quarter ended March 31, 2021. The increase in general and administrative expenses was largely due to professional fees incurred in connection with the OXB transaction, which included strategic advisory, legal and audit fees related to the unique structure of the collaboration that provided for Homology’s 20% ownership in the new business, preferred customer status, and the transfer of assets including its technical operations team, GMP facility, and associated intellectual property.
- In March of 2022, the Company closed its transaction with OXB and recorded a gain of
$131.2 millionon the sale of Homology’s manufacturing operations. As a result, Homology recorded an income tax provision of $1.0 millionfor the three months ended March 31, 2022that predominately resulted from the sale, after utilization of net operating loss carryforwards and tax credits.
- As of
March 31, 2022, Homology had approximately $256.1 millionin cash, cash equivalents and short-term investments. Based on current projections, Homology still expects cash resources to fund operations into the second half of 2024.
- Homology ASGCT Symposium:
May 18at 7:30 a.m. ET H.C. Wainwright Global Investment Conference: Available on-demand May 24at 7 a.m. ET
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates, including our expectations surrounding our ability to resolve the clinical hold placed on the HMI-102 program and the timing of related updates; the potential of our gene therapy and gene editing platforms, including our GTx-mAb platform; our plans and timing for the release of additional preclinical and clinical data; our position as a leader in the development of genetic medicines; the sufficiency of our cash and cash equivalents to fund our operations; activities related to our agreements with
- Financial Tables Follow –
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash, cash equivalents and short-term investments||$||256,126||$||155,873|
|Assets held for sale||—||28,907|
|Equity method investment||30,639||—|
|Property and equipment, net||1,986||2,252|
|Accounts payable, accrued expenses and other liabilities||$||16,192||$||13,772|
|Accrued income taxes||967||—|
|Operating lease liabilities||23,983||23,934|
|Total liabilities and stockholders' equity||$||310,958||$||211,721|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(in thousands, except share and per share amounts)|
|Three months ended
|Research and development||24,273||21,755|
|General and administrative||14,147||8,661|
|Total operating expenses||38,420||30,416|
|Loss from operations||(37,618||)||(1,111||)|
|Gain on sale of business||131,249||—|
|Total other income||131,281||38|
|Income (loss) before income taxes||93,663||(1,073||)|
|Provision for income taxes||(967||)||—|
|Loss from equity method investment||(591||)||—|
|Net income (loss)||$||92,105||$||(1,073||)|
|Net income (loss) per share-basic||$||1.61||$||(0.02||)|
|Net income (loss) per share-diluted||$||1.59||$||(0.02||)|
|Weighted-average common shares outstanding-basic||57,279,963||50,363,579|
|Weighted-average common shares outstanding-diluted||57,875,576||50,363,579|
Chief Communications Officer
and Corporate Communications
Source: Homology Medicines, Inc.