Homology Medicines Announces Plans for Three Clinical Programs in 2021 Spanning Phenylketonuria (PKU) and Hunter Syndrome (MPS II)
- Clinical Data from pheNIX Gene Therapy Phase 2 Dose Expansion Trial Expected by End of Year; Trial Currently Recruiting Patients -
- Company Plans to Nominate Additional Development Candidate in New Therapeutic Area -
- Management Highlights Anticipated 2021 Milestones in Webcast Available on Homology’s Website -
“We ended 2020 with positive data from the dose-escalation phase of the world’s first PKU gene therapy clinical trial, pheNIX, and validation of both our PKU gene therapy and gene editing programs with an equity investment from Pfizer, a leader in genetic medicines,” stated
Company management discussed its 2021 plans in a webcast that is now available on Homology’s website in the Investors section. Homology also plans to participate in the following events at the upcoming
- Virtual fireside chat (webcast will be available at
6:00 a.m. ETon Homology’s website)
- Panel titled, “Clinical Trials - Considerations in the Current Environment and its Impact on Future Design and Maintenance,” hosted by former Commissioner of the
U.S. Food and Drug Administration( FDA) Scott Gottlieb, M.D., at 12:00 p.m. ET.
Today’s webcast and next week’s fireside chat will be available on Homology’s website for 90 days.
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; expectations regarding the Phase 2 dose expansion phase of the pheNIX trial; plans to initiate two additional Phase 1/2 dose-escalation trials; plans to nominate an additional development candidate in 2021; 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; 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; 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; failure to obtain
|Chief Communications Officer
|and Corporate Communications|
Source: Homology Medicines, Inc.