BioMarin Pharmaceutical Inc.’s (BMRN) shares were up almost 3% after the company raised its 2014 guidance for Vimizim sales and announced encouraging interim data from an ongoing phase I/II pivotal study on its pipeline candidate, BMN 190. Overall, shares are up 3.6% since the announcement.
BioMarin presented the data and guidance at the annual J.P. Morgan Healthcare Conference.
BMN 190 Interim Results Encouraging
BioMarin is evaluating BMN 190 for the treatment of patients suffering from late infantile CLN2 disorder, a form of Batten disease.
The open-label, dose-escalation phase I/II pivotal study (n=24) is being conducted in late infantile Batten disease patients to evaluate the safety and tolerability of BMN 190 and to assess the effectiveness of using a CLN2 disorder-specific rating scale score in comparison with natural history data (patients typically lose 1 point every 6 months) over a treatment period of 48 weeks.
Interim analysis showed that out of nine patients who have been treated with BMN 190 for at least six months or up to 15 months, six patients showed no net change in their CLN2 rating scale score, while the other three showed a decline of one point. Additionally, seven of the nine patients with matched baseline age and disease severity fared better than their natural history counterparts.
Based on the initial promising data, the company plans to work with regulatory authorities to see if this single study will be enough to support approval. Final data from the study is expected in the fourth quarter of 2015.
Vimizim 2014 Guidance Raised
BioMarin increased its guidance for potential blockbuster drug, Vimizim, on the back of continued impressive performance. The company now expects Vimizim sales in the range of $75 million to $77 million (old guidance: $65 million to $70 million) in 2014 with fourth quarter sales expected in the range of $34.6 million to $36.6 million.
We remind investors that Vimizim is approved both in the U.S. and EU for the treatment of mucopolysaccharidosis type IVA (MPS IV A) or Morquio syndrome type A.
With more than 1,650 MPS IVA patients identified, Vimizim is already off to a strong start. The product should continue to perform well in 2015 as reimbursement in additional EU and secondary markets pick up.
In addition to this, BioMarin informed that its Prosensa (RNA) acquisition is well on track and it expects 2015 to be a pivotal year in terms of multiple data readouts across the pipeline.
BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Alexion Pharmaceuticals, Inc. (ALXN) and AMAG Pharmaceuticals, Inc. (AMAG). Both carry a Zacks Rank #1 (Strong Buy).
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