Homology Medicines Reports First Quarter 2021 Financial Results and Recent Highlights
- On Track to Report Initial Phase 2 Data From pheNIX PKU Clinical Trial by Year End -
- Announced New GTx-mAb Platform With Proof of Concept Data to Be Presented at Upcoming ASGCT Annual Meeting -
- Presented Data Demonstrating Nuclease-Free Gene Editing of Retinal Cells in Two Targets -
- Strengthened Financial Position -
“We are in the midst of a transformational time at Homology as we continue to mature and expand our genetic medicines platform and we are on track to report initial Phase 2 data from our pheNIX clinical trial and move two additional programs into the clinic this year,” stated
First Quarter 2021 and Recent Accomplishments
- Progressed the dose expansion phase of the Phase 1/2 pheNIX trial evaluating HMI-102 gene therapy for the treatment of adults with phenylketonuria (PKU), which has the potential to be converted to a registrational trial.
- Following encouraging clinical data from the dose-escalation phase of pheNIX, Homology continued to engage additional sites to enroll patients.
- Unveiled the Company's new GTx-mAb platform, which leverages its AAVHSCs to deliver vectors at a high efficiency to the liver and secrete antibodies throughout the body through a one-time administration of in vivo gene therapy, as shown in a humanized murine model. Proof of concept data targeting complement protein 5 (C5) that support this platform, in addition to new data from across the Company’s programs, including PKU, MPS II (Hunter syndrome) and metachromatic leukodystrophy (MLD), and advancements from its internal GMP manufacturing process and platform, are the subject of seven presentations, including two oral presentations, at the
American Society of Gene & Cell Therapy(ASGCT) Annual Meeting May 11-14. The Company plans to name a development candidate in a new therapeutic area from the GTx-mAb platform in 2021.
- Shared the first-ever data from the Company’s nuclease-free gene editing technology platform that demonstrated the ability of AAVHSCs to transduce relevant cell types in both non-human primate (NHP) and human cells and nuclease-free gene editing in two ophthalmic targets in human retinal explants in a co-presentation with Novartis at the
Association for Research in Vision and Ophthalmology(ARVO) Annual Meeting. An evaluation of 11 AAVHSC capsids from in vivo studies with NHPs and ex vivo studies with human retinal cells following a single intravenous (I.V.) or subretinal dose, respectively, found that:
- All capsids crossed the blood-retinal and blood-brain barriers in NHPs.
- AAVHSC15 achieved cross-species transduction of human and NHP photoreceptor cells.
- Molecular methods confirmed seamless editing in two independent loci and detection of hybrid transcript in human retinal cells.
- Highlighted data from Homology’s PKU and Hunter syndrome gene therapy programs in presentations at the
American Collegeof Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, including presentations on the pheNIX trial and the Company’s development candidate for Hunter syndrome, HMI-203. The Company expects to initiate Phase 1/2 dose-escalation clinical trials with HMI-203 and its in vivo gene editing candidate for PKU, HMI-103, in 2021.
- Showcased new and long-term data from IND-enabling studies for Homology’s central nervous system platform, including HMI-203 and the gene therapy development candidate HMI-202 for MLD, respectively, in presentations at the 17th Annual WORLDSymposium™ Meeting.
- Participated in patient advocacy-focused events, including Cure MLD’s Standards of Care Conference and Project Alive’s Hunter syndrome
- Completed a
$50.0 millionunderwritten public offering of shares of common stock in April, strengthening Homology’s financial position in support of key development milestones anticipated in each of the Company’s programs.
Tim Kellyto Chief Operating Officer.
First Quarter 2021 Financial Results
- Net loss for the quarter ended
March 31, 2021was $(1.1) millionor $(0.02)per share, compared to a net loss of $(35.3) millionor $(0.78)per share for the same period in 2020.
- Collaboration revenues for the quarter ended
March 31, 2021were $29.3 million, compared to $0.6 millionfor the quarter ended March 31, 2020. Collaboration revenues for the three months ended March 31, 2021includes the recognition of approximately $28.5 millionof deferred revenue and reimbursement of R&D expenses 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 quarter ended
March 31, 2021were $30.4 million, compared to $37.1 millionfor the quarter ended March 31, 2020, and consisted of research and development expenses and general and administrative expenses.
- Research and development expenses for the quarter ended
March 31, 2021were $21.8 million, compared to $29.3 millionfor the quarter ended March 31, 2020. Research and development expenses decreased due to higher expenses in the prior year related to the accelerated procurement of raw materials for manufacturing drug product for the Phase 1/2 pheNIX clinical trial and other development programs as part of Homology’s risk mitigation efforts in response to the COVID-19 pandemic. Additionally, the continued optimization of the Company’s ‘plug and play’ manufacturing process and platform has created greater than 50% efficiencies in subsequent programs that directly reduced spend for clinical trial materials. Costs incurred with Homology’s contract research organization (CRO) to conduct and manage the pheNIX trial with HMI-102 continued to increase, as well as personnel costs to support ongoing development programs, research initiatives, technology platforms and manufacturing capabilities.
- General and administrative expenses for the quarter ended
March 31, 2021were $8.7 million, compared to $7.8 millionfor the quarter ended March 31, 2020. 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
March 31, 2021, Homology had approximately $188.6 millionin cash, cash equivalents and short-term investments. Based on current projections, Homology expects cash resources, together with the approximately $49.4 millionin net proceeds received from a follow-on offering of its common stock in April 2021, to fund operations into the first quarter of 2023.
American Society of Gene & Cell Therapy(ASGCT) 24th Annual Meeting: May 11-14
- Homology Medicines Corporate & Pipeline Update Conference Call & Webcast:
May 13 RBC Capital Markets Healthcare Conference: May 18-19 Bank of America Securities2021 Napa Biopharma Virtual Conference: June 14-16 National PKU Alliance2021 Virtual Mini-Conference: July 9-10
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; the potential of our gene therapy and gene editing platforms, including our new GTx-mAb platform; 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, including initial Phase 2 data from the pheNIX clinical trial; our beliefs regarding our manufacturing capabilities; 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. 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; 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; failure to obtain
- Financial Tables Follow -
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(in thousands, except share and per share amounts)|
|Three months ended
|Research and development||21,755||29,310|
|General and administrative||8,661||7,770|
|Total operating expenses||30,416||37,080|
|Loss from operations||(1,111||)||(36,492||)|
|Total other income||38||1,161|
|Net loss per share-basic and diluted||$||(0.02||)||$||(0.78||)|
|Weighted-average common shares outstanding-basic and diluted||50,363,579||45,151,265|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash, cash equivalents and short-term investments||$||188,649||$||217,431|
|Property and equipment, net||36,153||37,002|
|Accounts payable, accrued expenses and other liabilities||$||10,301||$||14,525|
|Operating lease liabilities||14,821||15,442|
|Total liabilities and stockholders' equity||$||234,697||$||263,737|
Chief Communications Officer
Senior Corporate Communications Associate
Source: Homology Medicines, Inc.