Homology Medicines Reports Second Quarter 2021 Financial Results and Recent Highlights
- On Track to Report Initial Results from Phase 2 pheNIX Gene Therapy Trial for Adults with PKU in 2021-
- Plans to Initiate First-Ever Gene Editing Trial for PKU with HMI-103 This Year -
- Expects to Commence Clinical Trial with HMI-203 In
- Named Clinical Development Candidate from GTx-mAb Platform, HMI-104 -
“We continue to demonstrate the breadth and depth of our genetic medicines platform with three clinical programs expected this year, which span in vivo gene therapy and gene editing trials for rare liver and CNS-based diseases,” stated
Second Quarter 2021 and Recent Accomplishments
- Progressed Phase 2 of the pheNIX clinical trial evaluating a single intravenous (I.V.) administration of HMI-102 gene therapy in adults with phenylketonuria (PKU), with initial results expected by year end.
- Phase 2 is designed with the potential to be converted to a registrational trial.
- Homology is working closely with pheNIX sites to mitigate the potential impact of a COVID-19 resurgence.
- On track to initiate a gene therapy clinical trial for Hunter syndrome (MPS II), which is designed to evaluate a one-time I.V. administration of HMI-203 in adults, by year end. Data from IND-enabling studies were presented at the
American Society of Gene & Cell Therapy(ASGCT) Annual Meeting and showed:
- Long-term transduction and expression in multiple organs and sustained secretion of I2S enzyme in the serum;
- Reduction of glycosaminoglycan heparan sulfate (GAG-HS), including in the cerebrospinal fluid; and
- Phenotypic correction of joints and skeletal features.
- Shared first-ever data from Homology’s new GTx-mAb platform, which is designed to deliver one-time in vivo gene therapy to produce antibodies from the liver and secrete them throughout the body. Building off the preclinical data presented at ASGCT, Homology named a development candidate, HMI-104, for paroxysmal nocturnal hemoglobinuria (PNH).
- Concluded IND-enabling studies with HMI-103, Homology’s in vivo, nuclease-free gene editing candidate for PKU, which is on track to enter the clinic by the end of 2021. Preclinical data at ASGCT demonstrated HMI-103 integrated into the human PAH locus of a humanized liver model with integration rates similar to those that have resulted in sustained reduction of serum phenylalanine (Phe) in the Pahenu2 PKU murine model.
- Presented data at ASGCT demonstrating the stability, at high concentrations, of Homology’s novel formulation at cold storage temperatures, which simplifies the supply chain by eliminating the need for -80C frozen storage.
- Appointed gene therapy pioneer and ASGCT president
Beverly Davidson, Ph.D., as Chair of Homology’s Scientific Advisory Board, on which Dr. Davidsonhas served since 2018.
- Spoke alongside gene therapy industry leaders during a panel at the 2021
BIO International Conventionand participated in national and regional PKU patient advocacy-focused events.
- Completed a follow-on underwritten public offering of common stock with gross proceeds of
Second Quarter 2021 Financial Results
- Net loss for the quarter ended
June 30, 2021was $(30.5) millionor $(0.54)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
June 30, 2021were $2.2 million, compared to $0.6 millionfor the quarter ended June 30, 2020. Collaboration revenues for the second quarter 2021 included the recognition of deferred revenue and reimbursement of R&D expenses under the Company’s collaboration with Novartis, in addition to revenue recognized under Homology’s stock purchase agreement with Pfizer.
- Total operating expenses for the quarter ended
June 30, 2021were $32.7 million, compared to $36.3 millionfor the quarter ended June 30, 2020, and consisted of research and development expenses and general and administrative expenses.
- Research and development expenses for the quarter ended
June 30, 2021were $23.7 million, compared to $27.5 millionfor the quarter ended June 30, 2020. Research and development expenses decreased due to the completion of manufacturing of drug product in the prior year for the Phase 1/2 pheNIX clinical trial. Additionally, enhancements to the Company’s ‘plug and play’ internal manufacturing process and platform created greater than 50 percent efficiencies in subsequent programs that directly reduced spend for clinical trial and other materials. These included:
- Leveraging the highly developed and optimized ‘plug and play’ platform;
- Developing a novel transfection system that yields up to 230 percent more vector; and
- Operational execution and expertise delivering a 100 percent cGMP production run success rate.
These decreases were partially offset by increases in personnel costs to support ongoing development programs and new clinical programs, research initiatives, technology platform expansion and manufacturing capabilities.
- General and administrative expenses for the quarter ended
June 30, 2021were $9.0 million, compared to $8.8 millionfor the quarter ended June 30, 2020. General and administrative expenses increased due to personnel costs as a result of new hires and increased insurance expense, partially offset by decreased consulting expense and legal fees.
- As of
June 30, 2021, Homology had approximately $213.3 millionin cash, cash equivalents and short-term investments. Based on current projections, Homology expects cash resources to fund operations into the first quarter of 2023.
- PKU Northwest Family Camp: Presentation on HMI-102 gene therapy program for PKU on
August 21 AGBT Precision Medicine Conference: September 9-11 Georgia PKU Connect Summer Camp: Presentation on HMI-102 on September 11 H.C. Wainwright23rd Annual Global Investment Conference: Available on-demand September 13at 7:00 a.m. ET
- Baird 2021 Global Healthcare Conference:
- Intermountain PKU & Allied Disorders Lunch and Learn: Presentation on HMI-102 on
- Chardan’s 5th Annual
Genetic Medicines Conference: October 4-5
- Cell & Gene Meeting on the Mesa:
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; 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 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
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash, cash equivalents and short-term investments||$||213,293||$||217,431|
|Property and equipment, net||34,379||37,002|
|Accounts payable, accrued expenses and other liabilities||$||13,385||$||14,525|
|Operating lease liabilities||14,189||15,442|
|Total liabilities and stockholders' equity||$||258,918||$||263,737|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(in thousands, except share and per share amounts)|
|Three months ended
||Six months ended
|Research and development||23,697||27,471||45,452||56,780|
|General and administrative||9,042||8,793||17,703||16,563|
|Total operating expenses||32,739||36,264||63,155||73,343|
|Loss from operations||(30,552||)||(35,697||)||(31,663||)||(72,188||)|
|Total other income||52||357||90||1,517|
|Net loss per share-basic and diluted||$||(0.54||)||$||(0.78||)||$||(0.59||)||$||(1.56||)|
|Weighted-average common shares outstanding-basic and diluted||56,497,461||45,207,934||53,429,634||45,180,096|
Chief Communications Officer
Vice President, Patient Advocacy
and Corporate Communications
Source: Homology Medicines, Inc.