Dive Brief:
- Roche will advance an experimental Parkinson’s disease drug with a mixed track record into late-stage development, announcing Monday that it is “encouraged by the efficacy signals” the medicine displayed in earlier testing despite missing its main objectives in two clinical trials.
- The Swiss drugmaker and partner Prothena said continued study of trial enrollees suggested that the antibody drug, known as prasinezumab, delayed progression of symptoms like tremors or joint stiffness in people with early-stage disease.
- Roche and Prothena have been developing prasinezumab for more than a decade, having first initiated trials shortly after signing a partnership deal in 2013. The drug targets alpha-synuclein, a protein that can misfold and build up in the brains of Parkinson’s patients, and that’s been the focus of an array of drug programs. Study results have largely disappointed, however.
Dive Insight:
Despite decades of research into Parkinson’s, drugmakers still haven’t made much progress with medicines designed to slow its progression. Typical treatments largely involve older drugs that can help keep symptoms such as tremors under control.
The discovery of alpha-synuclein’s involvement in Parkinson’s gave scientists one strategy to combat the disease, but success has been elusive. Programs from Biogen and AbbVie and, more recently, a UCB-developed pill licensed to Novartis, have all disappointed in clinical testing.
Roche and Prothena have also dealt with setbacks. Two separate mid-stage trials, called Pasadena and Padova, showed only a numerical benefit on motor function that didn’t achieve statistical significance. Yet upon the failure of the second study last December, Roche indicated that there were enough signs to “merit further exploration.”
On Monday, the company committed heavily to that assessment. In a statement, Chief Medical Officer Levi Garraway said the “totality of the data” suggests prasinezumab “may have the potential to become the first disease-modifying treatment for people with Parkinson’s disease.”
In its statement, the company said that “multiple endpoints” from the Padova trial, as well as open-label extension studies, “suggest a clinical benefit” when added to effective symptomatic treatments. The Padova study also showed evidence prasinezumab might be impacting underlying disease biology, Roche added.
A longer-term analysis of results from the Pasadena trial published in Nature last year pointed to a sustained treatment effect, though the study authors cautioned that those findings need confirmation in further testing.
Prothena received $30 million up front to begin its deal with Roche and was promised up to a total of $600 million in future payouts, along with a share of U.S. profits and royalties on sales elsewhere. Prothena has collected a total of $135 million from the deal so far. Company shares climbed 11% in Monday trading.
