- On Track for Updates on pheEDIT and juMPStart Programs by End of 2022 -
- Prioritized Pipeline, Shifting Resources to HMI-103 PKU Gene Editing Trial in Adults While Pausing Enrollment in HMI-102 PKU Gene Therapy Trial Also in Adults -
- Received Clearance from Health Canada for juMPStart Trial of Investigational HMI-203 Gene Therapy for Hunter Syndrome and U.S. FDA Orphan Drug Designation -
- Announced Optimized MLD Gene Therapy Development Candidate with Plans to Seek a Partner for Program -
- Finished Second Quarter 2022 with Strong Cash Position and Extended Runway with Pipeline Prioritization -
BEDFORD, Mass., Aug. 15, 2022 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today financial results for the second quarter ended June 30, 2022, highlighted recent accomplishments, and provided a business update.
“Homology has a strong cash position that is now expected to take us into the fourth quarter of 2024. The extended cash runway is, in part, based on our recent strategic decision to divert our resources to the ongoing pheEDIT clinical trial evaluating gene editing candidate HMI-103 in adults with PKU, while pausing enrollment in our HMI-102 pheNIX gene therapy trial that was recruiting for the same patient population,” said Arthur Tzianabos, Ph.D., Chief Executive Officer of Homology Medicines. “We believe this, together with additional steps that we are taking, including plans to partner our optimized MLD gene therapy program, will position the Company well to move select programs forward to near-term catalysts while focusing our resources and conserving cash. We continue to expect to provide program updates on pheEDIT and our Hunter syndrome gene therapy trial, juMPStart, at the end of the year.”
Albert Seymour, Ph.D., President and Chief Scientific Officer of Homology Medicines, added, “While we continued to see biologic activity in our pheNIX gene therapy trial for PKU, we believe focusing our efforts on the gene editing program for PKU is the best approach for the Company at this time. This was confirmed at the recent ASGCT Meeting where we shared the unique mechanism of action of HMI-103, including data showing durability and increased expression in preclinical models, coupled with the potential to treat adults and ultimately younger patients. With several sites currently active and open for enrollment, we believe we can more rapidly progress recruitment for the pheEDIT trial, which will evaluate a steroid-sparing immunosuppressive regimen, including a T-cell inhibitor, versus the time it would take to resume enrollment of the pheNIX trial. We also continued to advance HMI-104, which uses our GTx-mAb technology, into IND-enabling studies. This program represents a third part of Homology’s platform, and we believe it provides the opportunity to address potentially larger market indications.”
Second Quarter 2022 and Recent Accomplishments
Second Quarter 2022 Financial Results
Upcoming Events
About Homology Medicines, Inc.
Homology Medicines, Inc. is a clinical-stage genetic medicines company dedicated to transforming the lives of patients suffering from rare diseases by addressing the underlying cause of the disease. The Company’s clinical programs include HMI-103, a gene editing candidate for phenylketonuria (PKU); HMI-203, an investigational gene therapy for Hunter syndrome; and HMI-102, an investigational gene therapy for adults with phenylketonuria (PKU). Additional programs focus on metachromatic leukodystrophy (MLD), paroxysmal nocturnal hemoglobinuria (PNH) and other diseases. Homology’s proprietary platform is designed to utilize its family of 15 human hematopoietic stem cell-derived adeno-associated virus (AAVHSCs) vectors to precisely and efficiently deliver genetic medicines in vivo through a gene therapy or nuclease-free gene editing modality, as well as to deliver one-time gene therapy to produce antibodies throughout the body through the GTx-mAb platform. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a focus on rare diseases. Homology believes its initial clinical data and compelling preclinical data, scientific and product development expertise and broad intellectual property position the Company as a leader in genetic medicines. For more information, visit www.homologymedicines.com.
Forward-Looking Statements
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 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; our plans to engage in future collaborations and strategic partnerships; 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 U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; securities class action litigation; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs incurred as a result of operating as a public company. These and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 and our other filings with the Securities and Exchange Commission (SEC) could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.
- Financial Tables Follow -
HOMOLOGY MEDICINES, INC. | |||||||
CONDENSED CONSOLIDATED BALANCE SHEETS | |||||||
(in thousands) | |||||||
(Unaudited) | |||||||
As of | |||||||
June 30, 2022 | December 31, 2021 | ||||||
Cash, cash equivalents and short-term investments | $ | 225,523 | $ | 155,873 | |||
Assets held for sale | — | 28,907 | |||||
Equity method investment | 29,298 | — | |||||
Property and equipment, net | 1,720 | 2,252 | |||||
Right-of-use assets | 15,151 | 15,607 | |||||
Other assets | 12,917 | 9,082 | |||||
Total assets | $ | 284,609 | $ | 211,721 | |||
Accounts payable, accrued expenses and other liabilities | $ | 16,702 | $ | 13,772 | |||
Accrued income taxes | 862 | — | |||||
Operating lease liabilities | 23,997 | 23,934 | |||||
Deferred revenue | 2,760 | 4,364 | |||||
Stockholders' equity | 240,288 | 169,651 | |||||
Total liabilities and stockholders' equity | $ | 284,609 | $ | 211,721 | |||
HOMOLOGY MEDICINES, INC. | |||||||||||||||||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | |||||||||||||||||
(in thousands, except share and per share amounts) | |||||||||||||||||
(Unaudited) | |||||||||||||||||
Three months ended June 30, | Six months ended June 30, | ||||||||||||||||
2022 | 2021 | 2022 | 2021 | ||||||||||||||
Collaboration revenue | $ | 802 | $ | 2,187 | $ | 1,604 | $ | 31,492 | |||||||||
Operating expenses: | |||||||||||||||||
Research and development | 21,075 | 23,697 | 45,348 | 45,452 | |||||||||||||
General and administrative | 8,034 | 9,042 | 22,181 | 17,703 | |||||||||||||
Total operating expenses | 29,109 | 32,739 | 67,529 | 63,155 | |||||||||||||
Loss from operations | (28,307 | ) | (30,552 | ) | (65,925 | ) | (31,663 | ) | |||||||||
Other income: | |||||||||||||||||
Gain on sale of business | - | — | 131,249 | — | |||||||||||||
Interest income | 474 | 52 | 506 | 90 | |||||||||||||
Total other income | 474 | 52 | 131,755 | 90 | |||||||||||||
Income (loss) before income taxes | (27,833 | ) | (30,500 | ) | 65,830 | (31,573 | ) | ||||||||||
Benefit from (provision for) income taxes | 105 | — | (862 | ) | — | ||||||||||||
Loss from equity method investment | (1,361 | ) | — | (1,952 | ) | — | |||||||||||
Net income (loss) | $ | (29,089 | ) | $ | (30,500 | ) | $ | 63,016 | $ | (31,573 | ) | ||||||
Net income (loss) per share-basic | $ | (0.51 | ) | $ | (0.54 | ) | $ | 1.10 | $ | (0.59 | ) | ||||||
Net income (loss) per share-diluted | $ | (0.51 | ) | $ | (0.54 | ) | $ | 1.09 | $ | (0.59 | ) | ||||||
Weighted-average common shares outstanding-basic | 57,385,578 | 56,497,461 | 57,334,078 | 53,429,634 | |||||||||||||
Weighted-average common shares outstanding-diluted | 57,385,578 | 56,497,461 | 57,869,443 | 53,429,634 | |||||||||||||
Company Contacts
Theresa McNeely
Chief Communications Officer
and Patient Advocate
[email protected]
781-301-7277
Media Contact:
Marisa Citrano
Manager, Corporate Communications
[email protected]
617-335-2841
Business Development Contact:
Kristi Sarno
Vice President, Business Development
and Corporate Strategy
[email protected]
617-429-1643
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