Homology Medicines Reports Fourth Quarter and Full Year 2021 Financial Results and Recent Highlights
- Formed New AAV Manufacturing and Innovation Business with Oxford Biomedica to Leverage Homology’s
- Launched pheEDIT Gene Editing Trial for PKU and juMPStart Gene Therapy Trial for Hunter Syndrome; Program Updates Expected by Year-End -
- Company Updates Guidance on pheNIX Gene Therapy Trial for PKU -
“We recently closed our deal with Oxford Biomedica to form a new company that incorporates Homology’s expertise in AAV process development and manufacturing capabilities, team and GMP facility,” stated
Consistent with Homology’s prior announcement of a clinical hold on its pheNIX gene therapy trial for phenylketonuria (PKU), the Company received the anticipated letter from the
With the additional information requested by FDA on the pheNIX trial and the planned conversion to a more specific steroid-sparing immunosuppressive regimen, Homology estimates that it will require more time to submit and receive feedback on its proposed clinical risk-mitigation strategy. This also includes time needed to amend the pheNIX study protocol. As a result, the Company now expects to provide a program update when the path forward is established with FDA.
Fourth Quarter 2021 and Recent Accomplishments
- Formed a new Manufacturing and Innovation Business with
UK-based, global viral vector manufacturer Oxford Biomedica (OXB), which incorporates Homology’s leading internal technical and manufacturing operations and is led by Homology’s former Chief Operating Officer, Tim Kelly, as Chief Executive Officer and Board Chair. Key benefits to Homology include:
$130 millionfrom OXB, which will support the advancement of the three clinical programs and genetic medicines platform;
- Non-dilutive capital and significant reduction in manufacturing expenses that extends cash runway into 2H 2024;
- 20% ownership and a Board seat in the new company;
- Access to the AAV ‘plug and play’ process and platform, which met CMC requirements for Homology’s three INDs, as a preferred customer;
- Continuing to work with AAVHSC experts within the state-of-the-art manufacturing facility built by Homology; and
$50 millioninvestment from OXB in the new company that aims to leverage OXB’s existing client base and commercial expertise.
- Initiated the pheEDIT trial, a Phase 1 dose-escalation study in adults with PKU evaluating HMI-103, a one-time, in vivo product candidate that utilizes a nuclease-free gene editing approach for PKU. A program update is expected by year-end 2022.
- Presented data supporting the recently launched juMPStart trial evaluating a one-time, systemic administration of HMI-203 investigational gene therapy for Hunter syndrome. Presentations at WORLDSymposium™ included:
- Clinical trial design, select eligibility criteria and planned endpoints for the Phase 1 dose-escalation trial. A program update is expected by year-end 2022;
- Incorporated patient, caregiver and key opinion leader feedback into the clinical trial design, which related to unmet medical need and expectations for a one-time gene therapy that has the potential for enzyme replacement therapy (ERT)-independence; and
- Homology’s gene therapy approach to Hunter syndrome and other lysosomal storage diseases, including metachromatic leukodystrophy (MLD), which is developed to target both the peripheral as well as the central nervous system (CNS) manifestations of these multi-organ disorders.
- Received orphan medicinal product designation (OMPD) and advanced therapy medicinal product (ATMP) classification from the
European Medicines Agency(EMA) for both HMI-203 for Hunter syndrome and HMI-103 for PKU.
- Continued to advance HMI-104, a C5 antibody development candidate for paroxysmal nocturnal hemoglobinuria (PNH), which is currently in IND-enabling studies. The Company’s GTx-mAb platform is designed to deliver one-time in vivo gene therapy to produce antibodies from the liver and secrete them throughout the body.
Fourth Quarter 2021 and Full Year Financial Results
- Net loss for the quarter ended
December 31, 2021was $(33.6) millionor $(0.59)per share, compared to a net loss of $(29.8) millionor $(0.62)per share for the same period in 2020. Net loss for the year ended December 31, 2021was $(95.8) millionor $(1.73)per share, compared to a net loss of $(128.7) millionor $(2.80)per share for the same period in 2020.
- Collaboration revenues for the three and twelve months ended
December 31, 2021were $0.8 millionand $34.0 million, respectively, as compared to $1.0 millionand $2.7 millionfor the comparable periods in 2020. Collaboration revenues consisted primarily of revenue recognized as a result of concluding the Company’s collaboration with Novartis. 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, 2021were $34.4 millionand $129.9 million, respectively, as compared to $30.8 millionand $133.0 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, 2021were $23.6 millionand $93.1 million, respectively, as compared to $23.2 millionand $100.4 millionfor the comparable periods in 2020. Research and development expenses decreased in 2021 due to a reduction of direct research expenses for HMI-102 due to the completion of manufacturing of drug product in the prior year for the Phase 1/2 pheNIX clinical trial. Additionally, the continued optimization of Homology’s ‘plug and play’ manufacturing process and platform has created efficiencies across all of programs that directly reduced spend for clinical trial and other materials and limited reliance on outside contract manufacturing organizations. Further, direct research expenses related to our HMI-202 program decreased over the prior year as the Company was applying the learnings from the IND-enabling studies to further optimize a vector with a better therapeutic profile. Partially offsetting these decreases were increased direct research expenses for HMI-103 and HMI-203 as Homology advanced both programs into the clinic in 2021, as well as 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, 2021were $10.8 millionand $36.8 million, respectively, as compared to $7.6 millionand $32.6 millionfor the comparable periods in 2020. General and administrative expenses increased due to personnel costs as a result of new hires and increased legal costs and other professional fees.
- As of
December 31, 2021, Homology had approximately $155.9 millionin cash, cash equivalents and short-term investments. Based on current projections, Homology expects current cash resources, including the $130.0 millionreceived from Oxford Biomedica in March 2022, to fund operations into the second half of 2024.
H.C. Wainwright Gene Therapy and Gene Editing Conference: Available on-demand March 30at 7:00 a.m. ET
- 21st Annual
Needham Virtual Healthcare Conference: April 11at 9:30 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 timing and expectations surrounding communications with the FDA regarding the pheNIX trial clinical hold and related updates from the Company; 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; 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 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
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash, cash equivalents and short-term investments||$||155,873||$||217,431|
|Assets held for sale||28,907||—|
|Property and equipment, net||2,252||37,002|
|Accounts payable, accrued expenses and other liabilities||$||13,772||$||14,525|
|Operating lease liabilities||246||2,501|
|Operating lease liabilities, net of current portion||23,688||12,941|
|Total liabilities and stockholders' equity||$||211,721||$||263,737|
|CONSOLIDATED STATEMENTS OF OPERATIONS|
|(in thousands, except share and per share amounts)|
|For the Three Months Ended
||For the Year Ended
|Research and development||23,646||23,195||93,085||100,392|
|General and administrative||10,781||7,587||36,835||32,573|
|Total operating expenses||34,427||30,782||129,920||132,965|
|Loss from operations||(33,625||)||(29,802||)||(95,949||)||(130,263||)|
|Total other income||42||11||185||1,569|
|Net loss per share-basic and diluted||$||(0.59||)||$||(0.62||)||$||(1.73||)||$||(2.80||)|
|Weighted-average common shares outstanding-basic and diluted||57,150,079||48,112,174||55,283,318||45,910,787|
Chief Communications Officer
Vice President, Patient Advocacy
and Corporate Communications
Source: Homology Medicines, Inc.