Analyst Conference Summary

biotechnology

conference date: February 21, 2019 @ 1:30 PM Pacific Time
for quarter ending: December 31, 2019 (fourth quarter, Q4 2018)

Forward-looking statements

Overview: While Q4 revenue was down 1% y/y, full 2018 revenue was up over 2017. The value is largely in the pipeline.

Basic data (GAAP):

Revenue was $353 million, down slightly sequentially from $ million, and down 1% from $358 million year-earlier.

Net income was negative $3.6 million, up sequentially from negative $ million, and up from negative $51.4 million year-earlier.

Diluted EPS was negative $0.03, up sequentially from negative $ and up from negative $0.30 year-earlier.

Guidance:

2019 commercial revenue expected between $1.68 and $1.75 billion. Expects a GAAP net loss of $45 to $85 million, but non-GAAP net income of $130 to $170 million.

Conference Highlights:

Jean-Jacques Bienaimé, Chairman and CEO of BioMarin said, "In November of 2018, we showcased a number of our other pipeline and research programs at BioMarin's annual Research and Development Day. Specifically, we provided a 42-month update on vosoritide for the treatment of achondroplasia that our ongoing Phase 2 study demonstrated an average additional cumulative height gain of 5.7 centimeters. Based on these results, we are encouraged that vosoritide could potentially be the first approved treatment option for children with achondroplasia. Finally, we were pleased to share initial pre-clinical data for BMN 307, our gene therapy product for PKU, which demonstrated lifetime normalization of Phe in a validated PKU mouse model. We plan to complete preclinical studies in the first half of 2019 with an anticipated IND filing planned for the second half of 2019."

The Q4 y/y revenue drop was driven by a decrease in Aldurazyme sales volume.

Non-GAAP net income was negative $10.8 million, up sequentially from $ million, and down from $5.2 million year-earlier. Stock-based compensation excluded was $ million.

Cash and equivalents ended at $1.32 billion, down sequentially from $ billion. Short term convertible debt $830 million. Total cash usage in 2018 was $87 million.

Total operating costs (GAAP) were $391 million, consisting of: cost of sales $75 million, research and development $175 million, selling, general and administrative $164 million, and intangible amortization & contingent consideration of $7 million. There was a $30 million gain on sales of intangible assets. Operating income was negative $38 million. Other expense net $5 million. Income tax benefit $40 million.

Palynziq first quarter of revenue, EU CHMP opinion expected in Q1 2019.

New adult patients who would have started Kuvan are instead starting Palynziq. For 2019 Kuvan revenue is expected between $420 and $460 million. Full 2018 revenue was $434 million.

Brineura (Cerliponase alfa) for CLN2, late-infantile form of Batten disease was approved by the FDA in April 2018.

Palyziq for phenylketonuria (PKU) (was Pegvaliase) received FDA approval on May 24, 2018. Believes could be a $1 billion product.

"A gene therapy product will be the next IND candidate for the treatment of PKU in 2019. PKU is an autosomal recessive disorder in which phenylalanine hydroxylase, the enzyme that metabolizes the amino acid phenylalanine (Phe), is deficient. PKU leads to high levels of neurotoxic phenylalanine, which would affect neurocognitive development, if left untreated. In preclinical models, BioMarin's PKU gene therapy product candidate demonstrated sustained, normalized Phe levels without hypophenylalanemia in an ongoing study and out to 53 weeks at the last observation. The product candidate will be an AAV vector containing the DNA sequence that codes for the phenylalanine hydroxylase enzyme that is deficient in people with PKU."

BMN 270 (Valoctocogene roxaparvovec, often "ValRox") gene therapy product for hemophilia A completed enrollment in a Phase 3 study. Has an accelerated approval endpoint strategy.

BMN 250 (renamed tralesinidase alfa) for MPS IIIB (Sanfilippo Syndrome Type B) updated preliminary Phase 1/2 data released in February showed normalization of heparan sulfate biomarker. Newer patients are safely at 300 mg. Study will now move to the expansion phase.

Vosoritide global Phase 3 trial in children with achondroplasia completed enrollment with data expected in 2019. Primary endpoint is growth velocity in children. Demonstrated a sustained increase in annualized growth rate at 30 months of treatment in Phase 2. An infant (Age 0-5 years) study will begin next year.

BMN 290 for Friedrich's Ataxia should have an IND submitted in the second half of 2018, followed by a Phase 1 trial initiation.

Anticipates a continuing flow of new drug candidates from the research organization. In particular believes will be expanding the gene therapy pipeline.

Q&A summary:

Hypothesis on pullback of expression levels in hemophilia program? Several considered, endoplasmic reticulum stress; cortical steroid false elevation; silencing; but we saw no evidence of them. So only left is the ? of the vector.

NPS6 market penetration issues? We are at over 85% penetration of known patients for several years for Naglazyme. But for morpheo 8 segment gating factors, we are working on that.

40 dose for hemophilia? Patients want the 60 dose, after that segment is full we can likely recruit more to the 40 dose.

Looking at other gene therapies and how payment structures might be done. Some might prefer to pay upfront, some over time. At first would likely be a one-time payment. By the time we get approval we should have 4 or 5 years experience with Phase 2 patients, which should help with pricing and reimbursement decisions.

Palyziq drop out rate? With a low sample size to date, tolerability has been at or lower than what we saw in the clinical trials. The overall dropout rate is relatively low, has more to do with logistics. The anaphylaxis rate has been relatively low. Dropout rate is not overly concerning so far.

Durability of Factor VIII levels vs. bleeding rates? FDA communicated we can get approval if assay is a valid measure of activity and you can document their bleeding rate is reasonably likely to predict in clinical outcomes. We expect to get confirmation of continuing efficacy. Durability of expression has not been a big concern of the FDA.

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