Homology Medicines Inc FIXX announced initial data from the first dose cohort in the pheEDIT Phase 1 dose-escalation trial evaluating gene editing candidate HMI-103 in adults with classical phenylketonuria (PKU).
As of the July 26, 2023, data cut-off, HMI-103, has been generally well-tolerated in all three participants.
Participant 1 experienced a reduction in plasma phenylalanine (Phe) levels to below the U.S. American College of Medical Genetics and Genomics (ACMG) PKU treatment guideline threshold of <360 μmol/L and the majority of Phe levels have been below 360 μmol/L through 31 weeks post-dose, including after the initiation of dietary protein supplementation.
Participant 2 has experienced a meaningful plasma Phe reduction of 49% at 17 weeks post-dose. Participant 3 was recently dosed.
The company said that 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.
Homology has retained TD Cowen as its strategic financial advisor.
Homology is reducing its workforce by 87% and stopping further program development efforts outside of required actions, including continued collection of data from and monitoring of participants in its clinical trials.
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.
Price Action: FIXX shares are up 23.40% at $1.16 during the premarket session on the last check Friday.
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