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Voyager Sinks as Sanofi Genzyme Opts Out of Parkinson’s Gene Therapy Program

Written by: | | Dated: Tuesday, October 31st, 2017


By Alex Keown


Shares of Voyager Therapeutics have fallen more than 12 percent in premarket trading after the company announced Sanofi Genzyme walked away from its $845 million gene therapy treatment for Parkinson’s disease.

The two companies forged the agreement in 2015 to develop deno-associated virus (AAV) gene therapies for the treatment of severe CNS disorders, such as Parkinson’s and Huntington’s disease. But that came to an end on Monday, Voyager announced.

Now Voyager has full control of VY-AADC and intends to launch a Phase II/III program later this year.

In its announcement, Voyager said Sanofi Genzyme decided not to exercise its development and commercial rights to VY-AADC outside the United States. From what Voyager announced, it appears that Sanofi Genzyme’s concern with the deal was that the rights to the gene therapy drug did not include commercialization rights inside the United States. However, according to the terms of the deal when it was first announced, Voyager always had U.S. rights to the Parkinson’s disease drug. That raises some questions as to why Sanofi Genzyme walked away from this particular program.

In September, Voyager announced positive results from an ongoing Phase Ib trial VY-AADC01 in advanced Parkinson’s disease. The results “demonstrated durable, dose-dependent and time-dependent improvements across multiple measures of patients’ motor function after a one-time administration of the gene therapy,” the company said in its announcement.

Voyager Chief Executive Officer Steven Paul said the company is pleased to regain worldwide rights to VY-AADC.

“There is a significant unmet need for Parkinson’s disease patients, particularly during the advanced stages of the disease where there are hundreds of thousands of patients globally. With a one-time administration that has been well tolerated, VY-AADC could penetrate the estimated 10,000 deep brain stimulation procedures worldwide each year for Parkinson’s disease and the further tens of thousands of advanced patients who do not elect to undergo deep brain stimulation,” Paul said in a statement.

VY-AADC consists of the adeno-associated virus-2 capsid and a cytomegalovirus promoter to drive AADC transgene expression. The therapy is designed to deliver the AADC gene directly into neurons of the putamen where dopamine receptors are located. By doing so, that bypasses the substantia nigra neurons, which enables the neurons of the putamen to express the AADC enzyme to convert levodopa into dopamine, according to Voyager.

While Sanofi Genzyme has walked away from the Parkinson’s disease program, Paul said the company remains “an important collaborator” to the company and its Huntington’s disease and Friedreich’s ataxia programs.

Paul said Voyager intends to describe its next steps regarding the Parkinson’s disease program during its third quarter conference call.

As of June 30 Voyager had cash and cash equivalents of $141.3 million. Based on the company’s current operating plan, Voyager said it anticipates ending 2017 with cash and cash equivalents between $90 and $100 million. That funding should be sufficient to continue operations into 2019, the company said.

Voyager closed at $22.73 on Monday, but fell to a low of $18.09 in late trading.


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