• Raptor Pharmaceutical Corp. (NASDAQ: RPTP) shares have plunged 43 percent in the last month, and are trading close to the lower end of their 52-week range of $6.82 - $16.28.

• Citi’s Joel Beatty downgraded the company from Buy to Neutral, while reducing the price target from $20 to $8.

• The rating and price target revisions follow the failure of the company’s RP103 to meet its primary endpoint in the phase 2b trial, Beatty said.

Raptor Pharmaceutical announced that the phase 2b trial of RP103 for pediatric fatty liver disease failed to meet its primary endpoint. Analyst Joel Beatty said that pediatric NASH had been removed from their model.

Beatty added, however, “RP103 reached a p-value of 0.34 on the primary endpoint of a two-point decrease in NAS and no worsening of fibrosis. We see this as a modest positive read-through for ICPT, as it supports that antioxidant activity (one of RP103’s multiple effects) alone is not enough to substantially improve liver histology in NASH.”

The next major catalyst for Raptor Pharmaceutical could be the clinical and regulatory update for RP103 in Huntington’s disease [HD], which is expected by the end of this year. There is a 40 percent probability of success for RP103 in HD, Beatty said.

Citi noted, “our base case is that another ph3 trial will be needed, with approval in HD in 2020. Approval based on 36-mo data would be upside from our estimates, and we believe this would be more likely in the EU than the US.”

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