Investors still aren’t sold on UniQure’s gene therapy for Huntington’s

 

• Shares of UniQure dipped more than 10% Tuesday morning after the biotechnology company released more data from a small study testing an experimental gene therapy for Huntington’s disease.

• The new results include up to 30 months of follow-up from 39 patients enrolled in the study, which is evaluating two doses of UniQure’s therapy, called AMT-130. The company said there’s evidence both doses are generally safe and can preserve or improve neurological function in patients with the disease, which progressively impairs movement and cognition.

• While UniQure leadership describes the results seen so far as “very promising,” analysts on Wall Street have found them puzzling. The low dose, for example, has appeared more effective and less variable than the high dose. How long either stays effective is also unclear. Nevertheless, UniQure intends to engage with regulators early next year to talk through the data it’s collected and “potential strategies for ongoing development of AMT-130.”

Huntington’s is caused by mutations in the gene responsible for making the similarly named “huntingtin” protein. While helpful to brain function in its normal form, the protein, when mutated, bunches together into toxic clumps that damage nerve cells.

AMT-130 is designed to silence that malfunctioning gene, and has become one of UniQure’s most closely watched programs.

In its update, UniQure said neurological function in patients given the high dose of AMT-130 was preserved or improved 18 months after treatment, compared to the start of the study. That was measured by a scoring system known in short as cUHDRS, which assesses the motor function, brain function and behavior of people with Hungtinton’s.

In the low dose arm, two other scales found neurological function was preserved at 30 months, according to UniQure.

The company added that, across all three measurements, AMT-130 showed “favorable trends” on the expected rate of decline compared to a natural history cohort. The cohort, which UniQure created with assistance from a nonprofit research organization, includes 31 patients with early Huntington’s disease who were similar to the participants in the AMT-130 study. Drugmakers use natural history data to better understand how a disease progresses without intervention.

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