Homology Medicines Reports Fourth Quarter and Full Year 2020 Financial Results and Recent Highlights
- Phase 2 PKU Gene Therapy Trial Recruiting Patients With Initial Data Expected by Year End -
- On Track to Initiate Phase 1/2 Trials With In
- Plans to
“In 2020, we remained focused on our mission, proactive in our preparations and successfully advanced our genetic medicines platform to meet our goals,” stated
Fourth Quarter 2020 and Recent Accomplishments
- Outlined plans to have three clinical programs during 2021, including:
- Advancing Homology’s pheNIX clinical trial for adults with phenylketonuria (PKU), the world’s first PKU gene therapy clinical trial, with initial data from the Phase 2 randomized, concurrently controlled, dose expansion phase of the trial expected this year. This has the potential to be converted to a registrational trial.
- Plans to initiate two additional Phase 1/2 dose-escalation trials; one with Homology’s first gene editing candidate, HMI-103, which is for PKU, and one with HMI-203, Homology’s gene therapy candidate for Hunter syndrome.
- Announced plans to unveil a development candidate in a new therapeutic area, unlocking the potential of the AAVHSC platform to target diseases with larger patient populations.
- Presented the first data from IND-enabling studies with HMI-203 at the 17th Annual WORLDSymposium™ Meeting. A single I.V. administration of HMI-203 in the adult murine model of Hunter syndrome:
- Led to robust biodistribution and sustained human I2S enzyme expression, which resulted in:
- Significant reductions in key Hunter syndrome biomarkers of heparan sulfate glycosaminoglycans (GAGs) and lysosomal-associated membrane protein 1 (LAMP1) in the brain, liver, heart, spleen, lung and kidneys compared with vehicle.
- Significant reductions in heparan sulfate GAGs in the cerebrospinal fluid (CSF) compared with vehicle.
- Ameliorated paw deformities, as shown by significant changes in measurements of ankle depth and width and paw depth and width, compared with vehicle.
- Led to uptake of human I2S from the serum of the HMI-203-treated model in human cell lines, demonstrating potential for cell cross-correction.
- Led to robust biodistribution and sustained human I2S enzyme expression, which resulted in:
- Shared long-term preclinical data with Homology’s HMI-202 in vivo gene therapy program for metachromatic leukodystrophy (MLD), which showed a single I.V. administration:
- Crossed the blood-brain barrier and blood-nerve barrier in the murine MLD disease model and in non-human primates (NHPs), with human ARSA (hARSA) detected in neuronal and glial cells.
- Showed durable hARSA activity in the central nervous system of the disease model, with distribution levels resembling those of Arsa in normal age-matched controls.
- Demonstrated significant changes in key MLD biomarkers of LAMP1, glial fibrillary acidic protein (GFAP), MAL transcript and neuronal sulfatides in the disease model compared with vehicle, similar to age-matched wild type controls.
- Announced a
$60 millionequity investment from Pfizer Inc. with the purchase of 5,000,000 shares of common stock at a price of $12.00per share, which closed on November 9, 2020. The investment includes a right of first refusal on potential future transactions involving PKU programs: HMI-102 gene therapy and HMI-103 gene editing.
- Presented at the
EveryLife Foundation for Rare Diseases2020 Scientific Workshopon innovative strategies and potential advantages and outcomes of implementing home health services in the first-ever PKU gene therapy clinical trial during the pandemic.
- Supported National PKU Alliance’s (NPKUA) #wearblueforPKU2020 campaign for PKU Awareness Day, and partnered with TinySuperheroes, an organization that empowers children overcoming an illness or disability, in recognition of Rare Disease Day 2021.
- Regained worldwide exclusive rights from Novartis to research, develop, manufacture and commercialize Homology’s proprietary nuclease-free gene editing technology platform for an ophthalmic target. The companies believe studies conducted under the collaboration provide early proof-of-principle and support a nuclease-independent approach to editing of relevant cell types in the eye after sub-retinal injection. Results of the studies are the subject of a planned presentation at an upcoming scientific meeting.
Fourth Quarter 2020 and Full Year Financial Results
- Net loss for the quarter ended
December 31, 2020was $(29.8) millionor $(0.62)per share, compared to a net loss of $(24.2) millionor $(0.55)per share for the same period in 2019. Net loss for the year ended December 31, 2020was $(128.7) millionor $(2.80)per share, compared to a net loss of $(103.9) millionor $(2.47)per share for the same period in 2019.
- Collaboration revenues for the three and twelve months ended
December 31, 2020were $1.0 millionand $2.7 million, respectively, as compared to $0.6 millionand $1.7 millionfor the comparable periods in 2019. Collaboration revenues consisted primarily of revenue recognized under the Company’s strategic collaboration with Novartis, which Novartis decided to conclude in February 2021following a portfolio review. Also included in collaboration revenues is revenue recognized under the Company’s stock purchase agreement with Pfizer.
- Total operating expenses for the three and twelve months ended
December 31, 2020were $30.8 millionand $133.0 million, respectively, as compared to $26.1 millionand $111.6 millionfor the comparable periods in 2019, 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, 2020were $23.2 millionand $100.4 million, respectively, as compared to $20.3 millionand $89.4 millionfor the comparable periods in 2019. Research and development expenses increased due to a rise in direct research expenses, including costs incurred with Homology’s contract research organization (CRO) to conduct and manage the Phase 1/2 pheNIX clinical trial with HMI-102, costs related to manufacturing clinical trial materials and direct research expenses related to advancing the Company’s other development-stage programs, including HMI-203 for Hunter syndrome and increased personnel costs to support ongoing development programs, research initiatives, technology platform and manufacturing capabilities.
- General and administrative expenses for the three and twelve months ended
December 31, 2020were $7.6 millionand $32.6 million, respectively, as compared to $5.8 millionand $22.2 millionfor the comparable periods in 2019. General and administrative expenses increased due to personnel costs as a result of new hires, increased audit and legal costs and increased costs associated with expanded operations.
- As of
December 31, 2020, Homology had approximately $217.4 millionin cash, cash equivalents and short-term investments. Based on current projections, Homology expects cash resources to fund operations into the third quarter of 2022.
- Project Alive – Presentation on HMI-203 gene therapy program for Hunter syndrome:
- Guggenheim Healthcare Talks Genomic Medicines and Rare Disease Day:
- 20th Annual
Needham Virtual Healthcare Conference: April 12-15 American Collegeof Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting– Presentations on pheNIX gene therapy clinical trial and HMI-203 gene therapy program: April 13-16
- 5th Annual Chardan Genetic Medicines Manufacturing Summit:
April 26-27 Association for Research in Vision and Ophthalmology(ARVO) 2021 Annual Meeting – Presentation on biodistribution of AAVHSCs and gene editing in ophthalmology research: May 1-7 Bank of America Merrill Lynch Health Care Conference: May 11-13 American Society of Gene & Cell Therapy(ASGCT) 24th Annual Meeting: May 12-15 RBC Capital Markets Healthcare Conference: May 18-19
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; our plans to name a development candidate in a new therapeutic area and potential thereof; plans and timing for the release of additional preclinical and clinical data; our beliefs regarding our manufacturing capabilities; 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. 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 capabilities 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
- Financial Tables Follow -
|CONSOLIDATED STATEMENTS OF OPERATIONS
|(in thousands, except share and per share amounts)
|For the Three Months Ended
||For the Years Ended
|Research and development||23,195||20,342||100,392||89,398|
|General and administrative||7,587||5,780||32,573||22,211|
|Total operating expenses||30,782||26,122||132,965||111,609|
|Loss from operations||(29,802||)||(25,559||)||(130,263||)||(109,943||)|
|Total other income||11||1,392||1,569||6,027|
|Net loss per share-basic and diluted||$||(0.62||)||$||(0.55||)||$||(2.80||)||$||(2.47||)|
|Weighted-average common shares outstanding-basic and diluted||48,112,174||44,077,777||45,910,787||42,117,690|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash, cash equivalents and short-term investments||$||217,431||$||262,388|
|Property and equipment, net||37,002||42,716|
|Accounts payable, accrued expenses and other liabilities||$||14,525||$||21,109|
|Operating lease liabilities||2,501||—|
|Operating lease liabilities, net of current portion||12,941||—|
|Total liabilities and stockholders' equity||$||263,737||$||310,567|
Chief Communications Officer
Senior Corporate Communications Associate
Source: Homology Medicines, Inc.