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Amicus Reports Disappointing Tests Results for Pompe Disease Drug

Amicus Therapeutics shares tanked Friday as analysts downgraded the biopharmaceutical company after it reported disappointing test results for its Pompe disease drug.

Pompe disease is a rare degenerative disorder that causes progressive muscle weakness. It can lead to death by disabling heart and skeletal muscles. Amicus said Phase 3 results for its AT-GAA treatment didn’t produce statistical significance in a primary endpoint.

To be sure, some analysts said the Food and Drug Administration may approve AT-GAA based on statistical significance in a secondary endpoint, Bloomberg reported.

In any case, Amicus recently traded at $12.64, down 32.52%. That left it down 14% for the past six months.

As for the analysts, SVB Leerink’s Joseph Schwartz cut his rating on the stock to market perform from outperform and slashed his price target to $15 from $30.

“The approvability of the drug comes into question, although management remains encouraged about their regulatory path forward for their crown jewel AT-GAA program and will still file for approval,” he wrote.

Cantor analyst Kristen Kluska dropped her rating to neutral from overweight and her price target to $17 from $30. “We believe AT-GAA could still see an approval this year, but have less conviction on this potential,” she wrote. “We expect the stock to be down 20-30% on the [endpoint] miss.”

Cowen left its rating at outperform but cut its price target to $29 from $31. “We think Pompe key opinion leaders and patients will see the totality of the data in a positive light and see a path to US and EU approval,” it said.