Gene editing company Graphite Bio announced a voluntary moratorium on the phase 1/2 CEDAR study
of nulabeglene autogedtemcel (nula-cell, GPH101) in sickle cell disease (SCD).
Due to the occurrence of a serious adverse event in the first patient taking Nula-Cel, the Company believes that this adverse event may be related to
this therapy.
As a result, the company will not be able to meet its guidance for initial proof-of-concept data by mid-2023
.
Nula-cel therapy is a next-generation gene-edited autologous hematopoietic stem cell therapy designed to directly modify HBB gene mutations to treat sickle cell disease
.
However, patients experience prolonged periods of low blood counts after treatment, which requires ongoing transfusion and growth factor support
.
Although adverse events did not meet the requirements to discontinue the clinical study, the patient achieved study-defined neutrophil implantation, and there was no evidence of myelodysplastic abnormalities, Graphite Bio reported the event to the FDA and discontinued the clinical study
after concluding that the adverse event may be related to treatment.
Notably, in addition to suspending the above studies, Graphite Bio also said that by mid-2023, the company will no longer meet the guidance requirements for nula-cel's initial proof-of-concept data, nor will it submit an IND application
for GPH102, a drug candidate for the treatment of β-thalassemia, until mid-2024.
The company said it will work to improve operational efficiency to maintain the company's cash flow through 2026
.
In fact, drug research and development has always had the characteristics of
long process, large investment and high failure rate.
Every year, major pharmaceutical companies around the world will have a number of clinical trials
that attract a lot of attention, but they come to an abrupt end at a critical stage.
Many multinational pharmaceutical companies have also announced clinical failures of new drugs and suspended trials
in recent months.
For example, a month ago, Relmada Therapeutics announced that its drug REL-1017 for the treatment of major depressive disorder did not meet the primary endpoint in the phase III RELIANCE-I clinical study, and the patient's depression did not improve significantly, that is, the clinical trial of depression drugs failed
again.
It is understood that REL-1017 is a novel NMDA receptor channel blocker that preferentially targets the GluN1-GluN2D hyperactive channel and maintains physiological glutamatergic neurotransmission
.
The FDA has granted REL-1017 Fast Track designation as an adjunct treatment
for major depression.
According to the results of the trial published by Relmada, on a rating scale measuring symptoms of depression, taking its four-week course of medication with standard care did not result in a statistically significant improvement
compared to placebo.
Prior to this, Roche released news that its phase III clinical study of Alzheimer's disease (AD, Alzheimer's) drug (ganteneruma) failed, and the official caliber was that it failed to have a statistically significant treatment effect
.
That is, it fails to significantly slow down the progression of dementia and solve the problems
of memory loss and decreased executive ability in early Alzheimer's disease.
In addition, Japan's Fujifilm has also announced its decision to stop the development of its anti-influenza drug Favipiravir tablets (Avigan) for the treatment of new coronavirus pneumonia.
.
.
In general, going from the lab to the launch of a new drug is a long and complex process, and every step in the process has the potential to lead to failure
.
Therefore, under the continuous innovation and research and development of pharmaceutical companies, failure will be inevitable
.
The industry proposes that in the future, if pharmaceutical companies want to solve this problem, they may be able to promote resource integration and international cooperation
.
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