JPM: BioMarin Anticipates Billions In Revenue
This article was originally published in Scrip
BioMarin may not be optimistic about the prospects for the approval of its Duchenne muscular dystrophy drug in the US, but the company is giving investors billions of other reasons to be excited.
The biotech company told investors during the JPMorgan healthcare conference in San Francisco that an FDA decision regarding Kyndrisa, more widely known as drisapersen, is expected any day now, after the agency gave no decision on its PDUFA date of Dec. 27. The FDA delay came about a month after the FDA's Peripheral and Central Nervous System Drugs Advisory Committee issued a negative opinion regarding approval. While the agency is not required to follow the recommendation of the panel, it often does.
BioMarin CEO Jean-Jacques Bienaime, affectionately known as JJ by analysts, tried to conduct damage control during the company's presentation at the conference – noting that 85% of DMD patients are outside of the US in BioMarin's other territories.
Drisapersen is also awaiting regulatory authority in Europe with a decision expected in the third quarter. Bienaime notes that about 70% of patients are in Europe.
Billions In Revenue
"We are about to achieve the next phase of significant growth for the company. Our base business is on track to achieve more than $1bn in revenues this year," the exec told the room as he noted that this number doesn't include an approval of drisapersen or any of the products in its pipeline.
Bienaime is now contending that it has four drugs in the pipeline that are each a $1bn opportunity, including the already-approved Vimizim, which is expected to have at least $300m in revenues this year.
Pegvaliase, a follow-on to Kuvan (sapropterin dihydrochloride)� in patients with phenylketonuria (PKU) is one of the drugs with $1bn in market potential. Previously, the company had estimated that pegvaliase and Kuvan together could reach the blockbuster threshold.
Bienaime now estimates that pegvaliase will garner 50% of its patients in the US and the other half internationally, building on the success of Kuvan so far.
Meanwhile, BMN-111 for achondroplasia and BMN-270, a gene therapy for hemophilia A, are the other drugs with blockbuster potential that are in the pipeline – both are still early.
Positive Results
The biotech also announced results from a Phase II study of BMN-701 (reveglucosidase alfa) in patients with Pompe disease. The trial shifted patients off of the standard of care and onto the drug for 24 weeks to see if it could improve respiratory function as well as results in a six minute walk test.
The 18 of 20 patients who completed the study showed a 2.2 percentage point improvement from baseline in maximal inspiratory pressure (MIP), as well as a 26.1 meter improvement in the six minute walk test.
Evercore ISI analyst Mark Schoenebaum points out in a Jan. 11 note to investors that the trial is not a head-to-head comparison with alglucosidase alfa and therefore cannot show superiority, but that the results seem good.
Bienaime said the company will now host discussions with regulatory authorities to discuss a Phase III plan for the drug and a path forward.