Si interrompe la somministrazione di tominersen nella sperimentazione Generation HD1 di Roche per la malattia di Huntington
On March 22nd, 2021, a press release from Roche revealed that the phase III study of tominersen had been halted on the advice of the Independent Data Monitoring Committee (iDMC).
When a therapeutic trial is launched, an independent external commission (iDMC: independent Data Monitoring Committee) is nominated. This committee is a group of independent experts who are totally separate from the patients, physicians and drug companies involved in running the study. Their sole job is to monitor the trial periodically to determine whether or not it should continue.
The committee periodically analyzes the data collected from the point of view of safety and efficacy and assesses whether the study should continue or not. Importantly: neither the pharmaceutical company nor the investigators have access to this data.
On the advice of the Independent Data Monitoring Committee (iDMC) the study of tominersen had been halted in the Generation HD1 Phase III clinical trial. No specific new safety concerns were raised so far, but nevertheless the trial has been stopped prematurely.
Since no critical issues related to the safety of the drug have emerged, we can assume that the drug has not proven to be sufficiently effective. So it’s not worth the risk of exposing people to a new drug because the benefits aren’t clear.
Does this mean that lowering the levels of the protein is not the right strategy?
Not necessarily. There could be several explanations, for example: the protein has been reduced too little, compared to what is necessary, it may not have been reduced enough in the expected areas of the brain, this strategy may not be the most suitable for patients in the early stages of the disease (stage 1 and stage 2, according to the inclusion criteria) or the drug does not reach the brain well ...
These are obviously only hypotheses at the moment. We will know more when the data are made public. At the moment, not even the researchers at Roche know the answer to those hypothetical questions right now.
Roche's Huntington's disease clinical trials will not be stopped: they will continue, but without the lumbar puncture and without the dosing of tominersen.
Patients are therefore being asked to stay in the study and continue their visits, but without the drug/placebo administration.
We are aware of the enormous disappointment this news produces in you all. However, we should not be discouraged. There are still many initiatives that will appear in the trials’ panorama and there is time to intervene with new therapeutic strategies even in the same patients who have participated in Generation HD1. We must not give up: the path is longer and more tortuous than we thought, but it continues and slowly will arrive to the hoped destination.
Tomorrow afternoon, Wednesday 24/03/2021 at 15.30 we will be connected via zoom for anyone who wants to know more at the following link: https://bit.ly/3ccIMvR
Find attached the official Press Release by Roche.