Raptor Pharmaceuticals Corp. (NASDAQ: RPTP) saw its shares approach its 52-week low in Monday’s premarket. This huge downturn was due to missed results in a Phase 2 study. The company also announced that it may discontinue this study altogether.

The company announced top-line results from the Phase 2b CyNCh study, which did not meet its primary endpoint of improving nonalcoholic steatohepatitis (NASH) in children. The trial evaluated the safety and efficacy of RP103, or cysteamine bitartrate delayed-release capsules, in children with biopsy-confirmed NASH.

For some background: Raptor is a global biopharmaceutical company focused on the development and commercialization of life-altering therapeutics that treat rare, debilitating and often fatal diseases.

Julie Anne Smith, president and CEO of Raptor, commented on the results:

While these trial results replicated the serological improvements seen in the earlier Phase 2a study, they did not translate into a measurable effect on histology. We’re disappointed with this outcome given the paucity of treatment for these children with NASH. While we’ll work closely with the NIDDK to understand the full data set, we do not expect to advance this program based on topline results. This clarifies our near-term priorities, which are to maximize the reach of PROCYSBI in nephropathic cystinosis, further the development of RP103 in Huntington’s and mitochondrial diseases, prepare for QUINSAIR’S launch and initiate at least one trial in nontuberculous mycobacteria or bronchiectasis. We remain wholly devoted to developing and commercializing transformational treatments for people living with rare diseases.

So far in 2015, Raptor has outperformed the market and shares are up over 14%. However, over the past 52 weeks shares are up only 5%.

On the books, Raptor reported its cash and cash equivalents for the most recent quarter as 194.5 million. Also for the most recent settlement date, the company saw its short interest rise to 12.4 million, with 10.5 days to cover, from the previous level of 11.7 million with 9.9 days to cover.

Shares of Raptor closed Friday down 0.3%, at $12.03 in its 52-week trading range of $7.85 to $16.28. In early trading indications Monday, shares were down an additional 34% at $7.87. The stock has a consensus analyst price target of $18.00.

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