Merck’s neuroscience portfolio is thin and there hasn’t been much in this part of the pharmaceutical giant’s pipeline since the failure of an Alzheimer’s disease drug in a pivotal clinical trial five years ago. Biotech company Cerevance has had success discovering and advancing a Parkinson’s disease drug candidate and Merck now wants to tap the startup’s technology to see if it can yield similar results in Alzheimer’s.
The pharmaceutical giant is paying Cerevance $25 million up front to begin a collaboration focused on the discovery of new targets for the memory-robbing disease.
Cerevance discovers drugs using technology that analyzes healthy and diseased port-mortem tissue from human brains. Those samples come from donors ranging from age 8 to 104. Those samples also represent multiple regions of the brain. Cerevance contends that analyzing human brain tissue exposes biological pathways of brain disorders that would be difficult to see in animal models or even using differentiated human stem cells. The technology is called Nuclear Enriched Transcript Sort sequencing (NETSseq).
Under the Merck collaboration, Cerevance will use its technology to discover new targets for potential Alzheimer’s disease drugs. The multi-year alliance already starts with one program. As part of the collaboration agreement, Cerevance will out-license to Merck one of its discovery-stage programs. Depending on the progress of the partnered research, the Boston-based biotech could receive development and commercial milestone payments reaching up to $1.1 billion, plus royalties from sales.
Cerevance’s most advanced internal program is its Parkinson’s candidate, CVN424. By targeting an indirect pathway associated with the disease, the small molecule is intended to generate the effects of levodopa and deep brain stimulation, two standard Parkinson’s treatments. Levodopa (L-dopa) is a commonly prescribed Parkinson’s drug that the brain converts into dopamine, the neurotransmitter whose deficiency is believed to be at the root of the disorder.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
In March, Cerevance reported data from a placebo-controlled Phase 2 study enrolling 135 patients. Results showed that treatment with its drug led to “significant and meaningful” dose-dependent reduction in off time, which are times during the day when Parkinson’s symptoms recur despite treatment with L-dopa and other medications.
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