--> BMRN
BioMarin Pharm misses by $0.08, beats on revs; guides FY17 revs below consensus
- Reports Q4 (Dec) loss of $0.16 per share, excluding non-recurring items, $0.08 worse than the Capital IQ Consensus of ($0.08); revenues rose 31.6% year/year to $300 mln vs the $294.95 mln Capital IQ Consensus.
- Co issues downside guidance for FY17, sees FY17 revs of $1.25-1.30 bln vs. $1.32 bln Capital IQ Consensus with $30-70 mln in non-GAAP net loss.
- "our regulatory filings for approval of Brineura, for the treatment of Batten disease, were accepted and validated in both the U.S. and EU. With the Prescription Drug User Fee Act (PDUFA) goal date for an FDA approval decision of April 27, 2017, we hope to have an approved treatment option for this devastating childhood disease in the near future." Mr. Bienaimé continued, "In addition, in 2016 we moved our vosoritide program forward based on Phase 2 results in children ages 5-14 with achondroplasia. At the end of 2016, we initiated a one-year, randomized, placebo-controlled Phase 3 study in children with achondroplasia ages 5-14 using a daily 15µg/kg dose. We recently provided encouraging preliminary results with our earliest clinical-stage program BMN 250 for the treatment of MPS IIIB, or Sanfilippo Syndrome, Type B. We are now moving to the expansion phase of the development program that will assess the impact of treatment with BMN 250 on the neurocognitive function in this rapidly progressive pediatric brain disease. Finally, in addition to the filing of the BLA for pegvaliase for the treatment of phenylketonuria expected in the second quarter of 2017, we expect to start to turn the corner to profitability with the achievement of positive non-GAAP results for the full year 2017."
- BMN 270 gene therapy product for hemophilia A: Today the Company announced that in the ongoing Phase 1/2 study, the three additional patients to be enrolled in the study will be dosed at the same 4 x 1013 vg/kg dose as the 3 most recently enrolled patients. Consistent with the dosing regimen of the 3 most recently enrolled patients, the 3 additional patients will be dosed without prophylactic corticosteroids. In October 2016, the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom approved continued enrollment into the open-label Phase 1/2 study of BMN 270 for severe hemophilia A. The agency also approved the Company's proposed amendments to the study, which included eliminating the requirement for prophylactic corticosteroids and increasing potential additional enrollment from up to three additional patients to up to six additional patients. In the fourth quarter of 2016, three patients were dosed at 4 x 1013 vg/kg with BMN 270.