Homology Medicines Announces FDA Lifted Clinical Hold on pheNIX Gene Therapy Trial for PKU
- Company Increased Risk-Mitigation Strategies, Including a Steroid-Sparing
Immunosuppression Regimen -
“We believe our ability to quickly and successfully respond to the FDA is a testament to our experienced clinical and regulatory teams who also applied key learnings from the gene therapy field and our clinical program to update the pheNIX trial protocol,” said
As previously disclosed, the clinical hold on the pheNIX trial pertained to elevated liver function tests observed in the trial, which were all resolved with no hospitalizations required. Homology’s response to the FDA included changes to the protocol intended to enhance risk-mitigation measures and a new immunosuppression regimen with the T-cell inhibitor tacrolimus, which will be used in combination with a reduced duration of prophylactic steroids. The same approach is being used in Homology’s ongoing pheEDIT gene editing trial for PKU and juMPStart gene therapy trial for Hunter syndrome. Similar regimens have been shown to dampen the immune response to AAVs in the clinical setting.
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 the protocol amendments and the additional risk-mitigation measures in place for our pheNIX trial, as discussed above; our plans and timing for the release of additional preclinical and clinical data; our expectations surrounding the potential, safety, and efficacy of our product candidates; the potential of our gene therapy and gene editing platforms; and our position as a leader in the development of genetic medicines. 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
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Source: Homology Medicines, Inc.