RARE Daily

Homology Cuts Staff, Evaluates Options for its Genetic Medicines Programs

July 28, 2023

Rare Daily Staff

Homology Medicines said it is stopping further development of its rare disease programs, cutting its staff, and evaluating strategic alternatives, including selling the company, after the board of directors completed a review of its business.

Homology also reported encouraging initial data from the first dose level in the phase 1, dose-escalation trial evaluating gene editing candidate HMI-103 in adults with classical phenylketonuria (PKU), which showed it was generally well-tolerated in all three participants and resulted in a meaningful reduction in plasma phenylalanine (Phe) in two participants as of the data cut-off date of July 26, 2023. Despite these encouraging data, based on the current financing environment and Homology’s anticipated clinical development timelines, Homology will not be further developing its programs and will be instituting a related reduction in force while it explores options for the company and its assets, including HMI-103.

“We are pleased with the initial data from the first dose level in our PKU gene editing trial, which support dose-escalation; however, given today’s tough financing conditions and the expected clinical development timeline for HMI-103, we believe the best path forward for our shareholders is to evaluate all strategic options for the Company and our pipeline,” said Albert Seymour, president and CEO of Homology Medicines.

Homology has gene editing and gene therapy clinical-stage programs in PKU and Hunter syndrome (MPS II), a preclinical pipeline that includes a gene therapy candidate for metachromatic leukodystrophy and a GTx-mAb (vectorized antibody) candidate for paroxysmal nocturnal hemoglobinuria, as well as intellectual property on its family of 15 adeno-associated viruses. Additionally, the company has a 20 percent stake in Oxford Biomedica Solutions, an AAV manufacturing company based on Homology’s internal process development and manufacturing.

Homology is reducing its workforce by 87 percent and stopping further program development efforts outside of required actions, including continued collection of data from and monitoring of participants in its clinical trials, to significantly reduce the company’s ongoing operating costs. These measures are expected to extend Homology’s cash runway into 2026. As of March 31, 2023, Homology had approximately $150.0 million in cash, cash equivalents and short-term investments, and the company will provide an update in its second quarter 2023 financial results.

Strategic options may include but are not limited to, an acquisition, merger, reverse merger, sale of assets, strategic partnerships, or other transactions.

Photo: Albert Seymour, president and CEO of Homology Medicines


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