Homology Medicines Reports Third Quarter 2020 Financial Results and Recent Highlights
- Based on Positive Data, Advancing pheNIX Gene Therapy Clinical Trial for PKU to Dose Expansion Phase -
- Unveiled In Vivo
“At the end of last week, we announced an important milestone for the PKU community and Homology with the positive data from the dose-escalation phase of our pheNIX PKU gene therapy clinical trial and plans to initiate the dose expansion phase by early 2021,” stated
Third Quarter 2020 Financial Results and Recent Accomplishments
- Announced today 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 is expected to close on or about November 10, 2020. The investment includes a right of first refusal on future transactions for the Company’s phenylketonuria (PKU) product candidates: HMI-102 gene therapy candidate for adults with PKU and HMI-103 gene editing candidate for pediatric patients with PKU.
- As announced on
November 6, 2020, plans to progress the pheNIX gene therapy clinical trial for adults with PKU to the randomized, concurrently controlled dose expansion phase with two doses. The expansion phase has the potential to be converted to a registrational trial. In the dose-escalation phase:
- Development candidate HMI-102 was generally well-tolerated, and there were no treatment-related serious adverse events (SAEs).
- There were marked reductions in phenylalanine (Phe) and the Phe-to-tyrosine (Phe/Tyr) ratio in some patients in Cohorts 2 and 3 (mid- and high-dose cohorts).
- The mean percent change from baseline in Phe observed in patients in Cohorts 2 and 3 were significant, compared to low-dose Cohort 1*. These Phe reductions occurred while patients self-liberalized their diets.
- Expanded Homology’s gene therapy programs with HMI-203, an in vivo approach for mucopolysaccharidosis type II (MPS II, or Hunter syndrome). Homology has initiated IND-enabling studies with HMI-203 and has scaled the development candidate to 500 liters using Homology’s commercial manufacturing platform.
- Received Orphan Designations in the
U.S.and EU and Rare Pediatric Disease Designation in the U.S.for HMI-202, Homology’s in vivo gene therapy development candidate for metachromatic leukodystrophy (MLD).
- Presented data from IND-enabling studies with HMI-202 at the
American Society of Human Genetics(ASHG) virtual annual meeting, including:
- A single I.V. administration of HMI-202 crossed the blood-brain-barrier and blood-nerve-barrier and led to durable reduction of sulfatides in all brain regions of the disease model.
- HMI-202 was superior to an AAV9-based MLD gene therapy construct in terms of human ARSA enzyme activity, vector genome copies and biodistribution in the peripheral and central nervous systems of the disease model.
- Progressed later-stage IND-enabling studies with HMI-103, Homology’s in vivo, nuclease-free gene editing candidate for pediatric PKU.
- Continued to enhance Homology’s internal GMP manufacturing capabilities, and executed more than 450 unique constructs and more than 550 lots of vector up to 2,000 liters using Homology’s plug and play system that is designed to enable rapid development.
- Continued to monitor the COVID-19 pandemic with no supply chain disruptions. HMI-102 supply is on-hand for the pheNIX trial expansion phase, which includes two doses.
Third Quarter 2020 Financial Results
- Net loss for the quarter ended
September 30, 2020was $(28.2) millionor $(0.62)per share, compared to a net loss of $(29.6) millionor $(0.67)per share for the same period in 2019.
- Collaboration revenues for the quarter ended
September 30, 2020were $0.6 million, compared to $0.4 millionfor the quarter ended September 30, 2019and consisted of revenue recognized under the Company’s strategic collaboration with Novartis. Collaboration revenues are being recognized over time consistent with the pattern of performance of research and development activities under the collaboration agreement. Homology and Novartis continue to work together on an ophthalmic program and seek to identify new targets for the collaboration based on the exploratory research component.
- Total operating expenses for the quarter ended
September 30, 2020were $28.8 million, compared to $31.7 millionfor the quarter ended September 30, 2019, and consisted of research and development expenses and general and administrative expenses.
- Research and development expenses for the quarter ended
September 30, 2020were $20.4 million, compared to $25.7 millionfor the quarter ended September 30, 2019. Research and development expenses decreased due to a decrease in direct research expenses for HMI-102 related to the transition of the manufacturing of clinical trial materials for our Phase 1/2 pheNIX trial from a CMO to our internal GMP facility in 2020, which reduced costs with our CMOs compared to the prior year period. Research and development costs related to laboratory supplies and research materials for our early-stage research programs also decreased. Partially offsetting these decreases were increased costs due to additional employee headcount to support ongoing development programs, research initiatives, technology platform and manufacturing capabilities resulting in increases in salaries, payroll taxes, stock-based compensation expense and recruiting costs.
- General and administrative expenses for the quarter ended
September 30, 2020were $8.4 million, compared to $6.0 millionfor the quarter ended September 30, 2019. General and administrative expenses increased primarily due to personnel costs as a result of new hires, increased audit and legal costs and additional costs associated with expanded operations.
- As of
September 30, 2020, Homology had approximately $178.0 millionin cash and cash equivalents. Based on current projections, Homology now expects cash resources, together with the anticipated proceeds of $60.0 millionfrom the Pfizer equity investment, to fund operations into the third quarter of 2022.
Upcoming Virtual Events
The EveryLife Foundation Scientific Conference: December 15, 2020 H.C. Wainwright BioConnect Conference: January 11 - 14, 2021
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing surrounding the Phase 1/2 pheNIX trial, including the expansion phase and the potential for conversion to a registrational trial; our collaboration activities with Novartis; our beliefs regarding our manufacturing capabilities and supply of HMI-102 for the expansion phase of the pheNIX trial; our position as a leader in the development of genetic medicines; our expectations regarding the timing for the closing of the Pfizer investment; the sufficiency of our cash and cash equivalents, together with the anticipated proceeds of
*P<0.004; Post-hoc comparison of Cohort 1 vs Cohorts 2&3 using repeated measures MANOVA/regression analysis
- Financial Tables Follow -
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(in thousands, except share and per share amounts)|
|Three months ended
||Nine months ended
|Research and development||20,417||25,691||77,197||69,056|
|General and administrative||8,423||6,038||24,986||16,431|
|Total operating expenses||28,840||31,729||102,183||85,487|
|Loss from operations||(28,273||)||(31,288||)||(100,461||)||(84,384||)|
|Total other income||41||1,661||1,558||4,635|
|Net loss per share-basic and diluted||$||(0.62||)||$||(0.67||)||$||(2.19||)||$||(1.92||)|
|Weighted-average common shares outstanding-basic and diluted||45,227,231||43,904,812||45,196,459||41,503,545|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash, cash equivalents and short-term investments||$||178,003||$||262,388|
|Property and equipment, net||39,684||42,716|
|Accounts payable, accrued expenses and other liabilities||$||11,742||$||21,109|
|Operating lease liabilities||16,037||—|
|Total liabilities and stockholders' equity||$||228,362||$||310,567|
|Chief Communications Officer
and Corporate Communications
Source: Homology Medicines, Inc.