Business
Homology Medicines Reports Third Quarter 2020 Financial Results and Recent Highlights
- Based on Positive Data, Advancing pheNIX Gene Therapy Clinical Trial for PKU to Dose Expansion Phase - - $60 Million Equity Investment from Pfizer Inc. - -
About this update from Q32 Bio Inc.
[{"type":"text","content":"- Based on Positive Data, Advancing pheNIX Gene Therapy Clinical Trial for PKU to Dose Expansion Phase -\n - $60 Million Equity Investment from Pfizer Inc. - - Unveiled In Vivo Gene Therapy Program for Hunter Syndrome - BEDFORD, Mass., Nov. 09, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today financial results for the third quarter ended September 30, 2020, and highlighted recent accomplishments. “At the end of last week, we announced an important milestone for the PKU community and Homology with the positive data from the dose-escalation phase of our pheNIX PKU gene therapy clinical trial and plans to initiate the dose expansion phase by early 2021,” stated Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. Dr. Tzianabos added, “We also recently announced that we have expanded our CNS portfolio by unveiling a new gene therapy program for Hunter syndrome. Our in vivo approach with HMI-203 leverages the potential of AAVHSCs to target peripheral organs and central and peripheral nervous systems following an I.V. administration. We believe this advantage, coupled with our team’s direct development and commercialization experience in Hunter syndrome, provides an opportunity to address the cognitive as well as systemic effects of this fatal disorder. HMI-203 has already been scaled up to 500 liters in our internal manufacturing facility, leveraging our plug and play system that is designed to enable rapid development across our gene therapy and gene editing programs.” Third Quarter 2020 Financial Results and Recent Accomplishments Announced today a $60 million equity investment from Pfizer Inc. with the purchase of 5,000,000 shares of common stock at a price of $12.00 per share, which is expected to close on or about November 10, 2020. The investment includes a right of first refusal on future transactions for the Company’s phenylketonuria (PKU) product candidates: HMI-102 gene therapy candidate for adults with PKU and HMI-103 gene editing candidate for pediatric patients with PKU.As announced on November 6, 2020, plans to progress the pheNIX gene therapy clinical trial for adults with PKU to the randomized, concurrently controlled dose expansion phase with two doses. The expansion phase has the potential to be converted to a registration...