There's Hope For BioMarin Beyond Kyndrisa

BioMarin Pharmaceutical Inc. BMRN announced that it has withdrawn Kyndrisa’s marketing authorization application in the EU, following expectations of a negative opinion from CHMP.

Barclays’ Geoff Meacham maintained an Overweight rating on the company, with a price target of $105.

Update Was Positive

“Given the earlier CRL from FDA, a negative opinion from CHMP was not a surprise,” Meacham mentioned, while adding, “The company is also discontinuing its work on follow-on products BMN044, BMN045, and BMN053.”

The analyst expects this to lead to a meaningful non-cash impairment charge of $580 million in 2Q16, although the update is viewed as “generally positive,” since no new resources would need to be allocated to Kyndrisa or the company’s other first generation exon-skipping products.

BioMarin Pharma continues to expect to achieve non-GAAP breakeven by 2017.

Catalysts For 2016

Meacham believes that possibility of additional data in achondroplasia and hemophilia could act as catalysts during 2016.

“We could see six-month efficacy data for the higher dose cohort of 30µg/kg. In April, four month data suggested that this dose was safe. BioMarin also plans on sharing plans for the phase 3 trial by 4Q,” the analyst stated.

Additional high-dose data for hemophilia is also expected by the year end, with the company intending to commence a registrationaldirect trial in 2017.