Graphite Bio pauses early-stage blood disease therapy trial
05/01/2023 22:57
Jan 5 (Reuters) - Gene therapy firm Graphite Bio
GRPH.O said on Thursday it was pausing an early-to-mid-stage
trial of its blood disease therapy due to a serious adverse
event in the first patient dosed, sending its shares down nearly
48% in after hours trading.
After being given Graphite's therapy, nula-cel, the patient
showed prolonged low blood cell counts and required continued
transfusion of blood, which the company believes is likely
related to its treatment.
Graphite said it has reported the incident to the U.S.
Food and Drug Administration (FDA), and the patient has shown no
evidence of blood cancer. The company is continuing a detailed
assessment of the incident and also considering changes to the
manufacturing process of its therapy.
In light of the pause, Graphite said it no longer plans to
seek the U.S. health regulator's permission to test its other
experimental therapy, GPH102, in humans by mid-2024. The gene
therapy firm said it was also looking to identify ways to extend
its cash position to at least 2026, from previously reported
cash runway of fourth quarter 2024.
Nula-cel, which holds FDA's 'fast track' tag, was being
studied as a potential treatment for sickle cell disease, which
leads to a shortage of healthy blood cells due to the
sickle-shape instead of the round shape of red blood cells.
The condition impacts about 100,000 people in the United
States and can cause complications such as infection, acute
chest syndrome and stroke.
(Reporting by Bhanvi Satija in Bengaluru; Editing by Shailesh
Kuber)
((Bhanvi.Satija@thomsonreuters.com; Outside U.S. +91
9873062788;))