Homology Medicines Reports Third Quarter 2021 Financial Results and Recent Highlights
- Achieved Goal of Three Clinical Programs Across Gene Therapy and Gene Editing Technology Platform This Year -
- Start of
- Initiated juMPStart Trial of One-Time I.V. Gene Therapy Candidate for Adults with Hunter Syndrome -
“We realized our goal to have three clinical programs underway this year by moving our gene editing candidate for PKU and gene therapy for Hunter syndrome into the clinic, both evaluating first-of-a-kind approaches for these diseases,” stated
Third Quarter 2021 and Recent Accomplishments
- Announced the pheEDIT clinical trial, a Phase 1 dose-escalation study of one-time, in vivo product candidate HMI-103 that utilizes a nuclease-free gene editing approach for phenylketonuria (PKU) and leverages learnings from the Company’s ongoing pheNIX PKU trial with the same AAVHSC vector. Once positive safety and efficacy results are established in adults, Homology plans to enroll younger patients in clinical trials.
- At the
American Society of Human Genetics (ASHG) Meeting, data supporting HMI-103 gene editing precision were presented, including molecular methods that demonstrated on-target integration and no evidence of integration into any other genomic location in a humanized murine liver model.
- HMI-103 received Fast Track designation by the
U.S. Food and Drug Administration(FDA).
- At the
- Initiated the juMPStart clinical trial, a Phase 1 dose-escalation study of one-time, in vivo gene therapy candidate HMI-203, the first I.V. gene therapy designed to address both peripheral and central nervous system (CNS) effects, in adults with mucopolysaccharidosis type II (MPS II), or Hunter syndrome.
- Data presented at ASHG showed adults with Hunter syndrome reported unmet medical needs despite the use of enzyme replacement therapy (ERT), particularly related to peripheral manifestations of the disease (e.g., range of motion and mobility, pain, etc.) and burden of chronic dosing. Preclinical data that supported the HMI-203 IND were also shared at scientific conferences, including systemic reduction of disease biomarkers in peripheral organs and the CNS, and prevention of skeletal deformaties.
- Provided an update on the ongoing pheNIX clinical trial, a Phase 2 dose expansion study evaluating HMI-102 gene therapy in adults with PKU. Specifically:
- Both doses were generally well-tolerated and showed evidence of biological activity, including clinically meaningful reductions in phenylalanine (Phe) levels, increases in tyrosine (Tyr) and reductions in the Phe-to-Tyr ratio;
- Added new clinical trial sites for a total of 13 with more expected shortly, and expanded Medical Affairs, Clinical Development and Operations teams to support ongoing trials; and
- Plans to provide a detailed data update in mid-2022 when more patients are expected to be enrolled in the trial.
- In addition to studies supporting three clinical programs, Homology highlighted preclinical data from its GTx-mAb development program for paroxysmal nocturnal hemoglobinuria (PNH) at the
European Society of Gene & Cell Therapy (ESGCT) Virtual Conference.
- Continued to support the rare disease patient community through educational efforts at national and regional patient advocacy-focused events.
Third Quarter 2021 Financial Results
- Net loss for the quarter ended
September 30, 2021was $(30.6) millionor $(0.54)per share, compared to a net loss of $(28.2) millionor $(0.62)per share for the same period in 2020.
- Collaboration revenues for the quarter ended
September 30, 2021were $1.7 million, compared to $0.6 millionfor the quarter ended September 30, 2020. Collaboration revenues for the third quarter 2021 included the recognition of all remaining deferred revenue and final reimbursement of R&D expenses under the Company’s former collaboration with Novartis, in addition to revenue recognized under Homology’s stock purchase agreement with Pfizer.
- Total operating expenses for the quarter ended
September 30, 2021were $32.3 million, compared to $28.8 millionfor the quarter ended September 30, 2020, and consisted of research and development expenses and general and administrative expenses.
- Research and development expenses for the quarter ended
September 30, 2021were $24.0 million, compared to $20.4 millionfor the quarter ended September 30, 2020. Research and development expenses increased due to higher external development costs as HMI-103 and HMI-203 advanced into Phase 1 clinical trials during the quarter, as well as increased personnel costs to support ongoing development programs and new clinical programs, research initiatives, technology platform expansion and manufacturing capabilities. Partially offsetting these increases was a decrease in 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.
- General and administrative expenses for each of the quarters ended
September 30, 2021and 2020 were $8.4 million.
- As of
September 30, 2021, Homology had approximately $187.6 millionin cash, cash equivalents and short-term investments. Based on current projections, Homology continues to expect cash resources to fund operations into the first quarter of 2023.
- Stifel 2021
Virtual Healthcare Conference: November 17at 9:20 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; 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 -
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash, cash equivalents and short-term investments||$||187,551||$||217,431|
|Property and equipment, net||32,697||37,002|
|Accounts payable, accrued expenses and other liabilities||$||13,538||$||14,525|
|Operating lease liabilities||13,541||15,442|
|Total liabilities and stockholders' equity||$||230,816||$||263,737|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(in thousands, except share and per share amounts)|
|Three months ended
||Nine months ended
|Research and development||23,987||20,417||69,439||77,197|
|General and administrative||8,351||8,423||26,054||24,986|
|Total operating expenses||32,338||28,840||95,493||102,183|
|Loss from operations||(30,661||)||(28,273||)||(62,324||)||(100,461||)|
|Total other income||53||41||143||1,558|
|Net loss per share-basic and diluted||$||(0.54||)||$||(0.62||)||$||(1.14||)||$||(2.19||)|
|Weighted-average common shares outstanding-basic and diluted||57,106,639||45,227,231||54,704,410||45,196,459|
Chief Communications Officer
Vice President, Patient Advocacy
and Corporate Communications
Source: Homology Medicines, Inc.