Analyst Conference Summary

biotechnology

conference date: April 28, 2016 @ 1:30 PM Pacific Time
for quarter ending: March 31, 2016 (first quarter, Q1 2016)

[yet]
Forward-looking statements

Overview: Sales increased sequentially as well as y/y, a good sign. Lots of good pipeline news was reviewed, though most of it had been released on April 20.

Basic data (GAAP):

Revenue was $236.7 million, up 4% sequentially from $227.9 million, up 17% from $202.9 million year-earlier.

Net income was negative $85.1 million, down sequentially from $68.6 million, and down from negative $67.5 million year-earlier.

Diluted EPS was negative $0.53, down sequentially from $0.39, and down from negative $0.43 year-earlier.

Guidance:

Updated GAAP net-loss guidance for 2016.

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 $355 to $385 million. This assumes approval of Kyndrisa in the EU. If not approved should not affect non-GAAP results, but would affect GAAP through an impairment charge.

Conference Highlights:

Jean-Jacques Bienaimé, Chairman and CEO of BioMarin said, "Our commercial base business is robust and is expected to generate over one billion dollars in revenues this year. Prospects for new product launches in 2017 increased during the quarter due to positive data readouts for cerliponase alfa and pegvaliase that we expect will lead to two new product filings later this year."

Vimizim demand was strong and looks strong going forward. Kuvan was boosted by the beginning of PKU sales outside North America of $16.9 million.

Collaboration, royalty and other revenue was $1.3 million.

Non-GAAP net income was negative $27.2 million, up sequentially from negative $70.0 million, and down from negative $25.4 million year-earlier.

Cash and equivalents ended at $770 million.

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.

Total operating costs (GAAP) were $317.7 million, consisting of: cost of sales $43.1million, research and development $158.8 million, selling, general and administrative $105.3 million, and intangible amortization & contingent $10.4 million. Loss from operations was $80.9 million. Other expense net $8.2 million. Income tax benefit $4.0 million.

A pipeline update was given at the R&D day, April 20, see Biomarin 2016 R&D Day slides . A brief recapitulation was given.

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), showed a robust response. Biomarin plans to submit in the U.S. and E.U. for regulatory approval mid-year 2016.

Pegvaliase for phenylketonuria (PKU): Phase 3 results met the primary endpoint. Biogen will 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. Preliminary data released in April was encouraging.

Vosoritide for achondroplasia: Tthe Company updated results from the Phase 2 study showing a 46 percent in mean annualized growth velocity (speed at which growth in children occurs) over 12 months.

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.

Q&A:

Achondroplasia 30 microgram data release, efficacy goals? We have not revealed our plans for relase of the 30 µg cohort data. The endpoint is normalization of growth velocity. We have no decided on whether to start a 60 µg cohort. We believe the 15 µg dose is sufficient to warrant a registration-enabling trial.

The fundamental thought for achondroplasia is that it starts in the growth center and the other defects are secondary, so treating for growth should improve other endpoints over time.

Naglazyme buying patterns, Latin America political instability? We have no exposure in Venezuala, but do in Brazil and other countries. In the past the impacts have been from foreign exchange and irregular order patterns. Vimizim already had material buying in Q2. We don't expect Q2 buys to always be heavy.

Kyndrisa other exons plans? We are waiting for the EU decision before making decision about development plans for the other exons.

Hemophilia EU gating event? Due to an increase in liver enzymes in 2 patients we will not enroll further patients until we have completed discussions with EU regulators. This is an early safety precaution, we don't expect a substantial delay. These patients were not given prophylactic steroids, no problem with those given steroids.

Vimizim patient growth? We don't disclose patient number by quarter. Patients doubled in the past year, as of the end of Q1.

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