Analyst Conference Summary

Biomarin Pharmaceuticals

conference date: February 25, 2016
for quarter ending: December 31, 2015 (fourth quarter 2015)

Forward-looking statements

Overview: Not a strong quarter for revenue, but good y/y net income improvement.

Basic data (GAAP):

Revenue was $227.9 million, down 1% from $230.1 million year-earlier.

Net income was $68.6 million, reversing a loss of $69.8 million year-earlier.

Diluted EPS was $0.39, up from negative $0.47 year-earlier.


For 2016 total revenue is expected between $1.05 and $1.10 billion. Cost of sales as % of revenue between 18% and 19%. Operating expenses $1.15 to $1.21 billion. Resulting in a non-GAAP net loss of $75 to $100 million and a GAAP net loss of $400 to $430 million. $150 to $175 million capital expense. Ranges mainly depend on whether Kyndrisa is approved and launched.

Conference Highlights:

Biomarin is expecting to generate over $1 billion in revenue in 2016. Two new product filings may be made in 2016, with 2 products launching in 2017. Plus development will continue on other products in the pipeline.

therapy Q4 2015
revenue (millions)
Q4 2014
revenue (millions)
y/y change

Biomarin notes that "Vimizim and Naglazyme revenues experience quarterly fluctuations due to the timing of government ordering patterns in certain countries. The Company does not believe these fluctuations reflect a change in underlying demand." Vimizim launch has gone well and expects $300 to $330 million in sales in 2016.

Believes naglazyme negative growth is a one-quarter fluctuation as continues to add patients and keep patients already on the drug.

There was a negative impact from FX effects.

Collaboration, royalty and other revenue was $1.5 million.

Non-GAAP net income was negative $70.0 million, down from negative $10.7 million year-earlier.

The non-GAAP EPS decline resulted from higher operating expenses.

GAAP net income in the quarter was positive due to the $369.5 million gain on the sale of talazoparib to Medivation. It also included a $47.9 million credit to contingent consideration expense due to the Kyndrisa complete response letter. There was a $198.7 million impairment due to the decline of the value of Kyndrisa.

Cash and equivalents ended at $1.02 billion.

In Q2 Biomarin hopes to receive a positive European, CHMP opinion on Kyndrisa for Duchenne muscular dystrophy amenable to exon 51 skipping.

Believes can reach $1.5 billion in product revenue by 2020. Believes can reach non-GAAP break even by 2017.

Milestones possible for the first half of 2016:

Cerliponase alfa for CLN2, late-infantile form of Batten disease: Complete results from the Phase 1/2 study for the treatment of patients with late-infantile neuronal ceroid lipofuscinosis type 2 (NCL-2), will be announced on March 2, 2016. If data are supportive, Biomarin plans to submit in the U.S. and E.U. for regulatory approval mid-year 2016.

Pegvaliase for phenylketonuria (PKU): expects to share top-line results from this study in the first quarter of 2016 and, if the data are supportive, submit a Biologics License Application (BLA) to U.S. FDA in the second half of 2016.

BMN 270 gene therapy product for hemophilia A: In the fourth quarter of 2015, the first patient was dosed in a Phase 1/2 trial with BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A. BMN 270 is an AAV-factor VIII vector, designed to restore factor VIII plasma concentrations. BioMarin will provide a program update at the R&D Day in April 2016.

Vosoritide for achondroplasia: In June 2015, the Company published results from the Phase 2 study showing a 50 percent in mean annualized growth velocity (speed at which growth in children occurs) in the cohort of 10 patients receiving a 15 µg/kg dose of vosoritide daily for six months compared with their own pre-treatment growth velocity (P-value= 0.01). In addition, a fourth cohort with 30 micrograms per kilogram daily completed enrollment in the fourth arm of the Phase 2 study. BioMarin will provide 12-month results with vosoritide an update on Phase 3 plans at the R&D Day in April 2016.

Kyndrisa (drisapersen) for Duchenne muscular dystrophy: The Committee for Medicinal Products (CHMP) is expected to provide an opinion on the application in the second quarter of 2016. If the CHMP provides a positive opinion, Kyndrisa could potentially be approved in the E.U. in the second half of 2016.

Reveglucosidase alfa for Pompe disease: In January 2016, the Company shared interim results from the single-arm Phase 2/3 trial with patients previously treated with alglucosidase alfa who were then switched to treatment with reveglucosidase alfa. The study showed an improvement from baseline in the respiratory parameter Maximal Inspiratory Pressure (MIP) as well as the secondary endpoint 6 minute walk test.


R&D on other exon skippers? We have 3 other exons in Phase 2. They will continue this year. We don't see a change in investment level in 2016. Going forward it would depend on the Kyndrisa results.

Revenue effect of FX in 2016? If rates stay stable from today the FX impact could be $20 to $40 million. The Euro could pick up, but third world currencies could weaken.

Gene therapy program scenarios? This is a first in man study, keys are safety and dose ranging vs. Factor 8 response. We are looking at the dose response curve and gene expression.

PKU attentiveness data: we are trying to show a trend, which is about all you can do given the number of patients available. We hope we will get an improvement in cognition. We do not have a concrete agreement on the FDA for the P value for this.

Pegvaliase, unlike Kuvan, is injected and can cause flu like symptoms, but it is manageable, you just need to pause while escalating the does. All of our drugs require considerable clinician support for patients. We have learned a lot about managing patients during our trials.

Pricing of drugs? Price increases do not exceed inflation. We would guide to only very modest price increases going forward.

We did have to change our plans because of the Kyndrisa complete response letter. That is why we were not giving guidance. Now we have the confidence to give guidance. We did rachet the target down a little, from non-GAAP profitable in 2017 to non-GAAP break-even in 2017. We did decide we could delay some investments a little bit.

Facebook Hemophilia blogger? We protect the identity of patients in our clinical trials. We are up to the third dose level and have been encouraged by what we have seen so far.

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Disclaimer: My analyst call summaries may include both our condensations of statements made by company representatives and my own analysis. They are not covered by any warranty. I cannot guarantee anything said by company representatives is true. I try not to make errors, but it is possible. This is investment journalism, not financial advice.

Copyright 2016 William P. Meyers