2014 guidance of $55-$65 million of net sales for PROCYSBI ®
- Net product sales for PROCYSBI ® for the fourth quarter ended December 31, 2013 were $10.2 million and $16.9 million for the full year. PROCYSBI became commercially available in the U.S. in June 2013.
- As of December 31, 2013, there were 236 new prescriptions for PROCYSBI, 165 unique patients on therapy, with another 71 patients in the process of reimbursement verification.
- Top line results from a planned 18 month analysis of an ongoing 3 year Phase 2/3 study in Huntington's disease showed a positive trend towards slower progression of Total Motor Score (TMS) in patients treated with RP103 vs. those patients on placebo, and statistically significant slower progression of TMS in patients receiving RP103 without tetrabenazine, following 18 months of treatment.
- Full enrollment in Phase 2b NASH/NAFLD announced in January 2014. Data is anticipated the first half of 2015.
- Raptor expects net product sales for PROCYSBI of $55-$65 million and non-GAAP operating expense, excluding non-cash stock-based compensation expense, of $80-$90 million in 2014.
- Cash and cash equivalents as of December 31, 2013 were $83.1 million.