Novartis will temporarily suspend dosing of branaplam (LM1070) in a phase 2b Huntington’s disease (HD) study following a data review which revealed the drug might be causing side effects on patients’ nerves, the company announced.
Specifically, the review found that branaplam may cause peripheral neuropathy, which is the result of nerve injury outside the brain and spinal cord.
In a letter to the HD community, Novartis outlined that the study was specifically designed with ‘robust monitoring’ to detect early safety signals.
The company stated that the independent Data Monitoring Committee (DMC) did not recommend terminating the study at this time, and participants will continue to have regular study assessments per the protocol.
The company said in a statement: ‘We understand the devastating impact of HD on people living with the disease and their families, and will continue to work closely with those involved in the study and thoroughly assess all available information, including data to be collected in the coming weeks, to better understand these findings and inform next steps for the programme.’
HD is a rare, inherited disease that causes the progressive breakdown of nerve cells in the brain. The disease has a wide impact on a patient’s functional abilities and usually results in movement, cognitive and psychiatric disorders.
Existing pharmacologic treatments are limited and only address individual symptoms which have no effect on the course of the disease or life expectancy, underlining the unmet medical need for therapies that delay the onset or slow the progression of HD.
Branaplam is a once-weekly, orally administered, mRNA splicing modulator that targets the underlying pathophysiology in HD by modifying HTT mRNA throughout the brain and the body, resulting in lower levels of HTT protein. The treatment received fast track designation by the US Food and Drug Administration (FDA) in December 2021.
‘We wish to highlight how grateful we are to the people participating, and their families, and how important it is for those in the study to continue to be assessed. With this participation, the community will add to the body of knowledge about HD, help continue progress toward new treatments, and assist us in making the best decisions regarding the future development of branaplam,’ the company said in the letter.