BioMarin Announces Second Quarter 2017 Financial Results Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended June 30, Six Months Ended June 30, 2017 2016...

SAN RAFAEL, Calif. , Aug. 2, 2017 /PRNewswire/ --

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN ) today announced financial results for the second quarter ended June 30, 2017 . For the quarter ended June 30, 2017 , GAAP net loss was $36.8 million , or $0.21 per basic and diluted share, compared to GAAP net loss of $419.0 million , or $2.58 per basic and diluted share for the quarter ended June 30, 2016 . The reduction in GAAP net loss year over year was primarily due to the absence of the $599.1 million impairment of intangible assets associated with the discontinuance of the Kyndrisa and Reveglucosidase alfa programs in 2016, partially offset by increased selling, general and administrative expenses for the continued global expansion of Kuvan and the launch of Brineura.

Non-GAAP income for the second quarter ended June 30, 2017 was $26.6 million , compared to non-GAAP income of $17.0 million for the quarter ended June 30, 2016 . Non-GAAP income (loss) is defined by the Company as reported GAAP net income (loss), excluding net interest expense, provision for (benefit from) income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and certain other specified items as detailed below. Refer to non-GAAP Information beginning on page 9 of this press release for a complete discussion of the Company's non-GAAP financial information and reconciliations to the comparable GAAP reported information.

Total BioMarin Revenues were $317.4 million for the second quarter of 2017, an increase of 6% compared to the same period in 2016. For the second quarter of 2017, Kuvan net product revenues increased to $102.0 million , a 13% year over year increase. Growth was driven by an 11% increase in the number of patients on Kuvan therapy in the U.S and the continued growth in the ex-North American territories acquired in 2016. For the second quarter of 2017, Naglazyme net product revenues increased to $85.7 million , an increase of 9% year over year. The number of patients on Naglazyme therapy increased 7% year over year. Vimizim net product revenues were $103.2 million in the quarter, a 3% year over year decrease, primarily driven by the timing of government orders. The number of patients on Vimizim therapy increased 24% year over year. With these new patient starts, Vimizim remains on track to meet full year revenue target of $400 to $430 million , a growth rate of 13% to 21%.

As of June 30, 2017, BioMarin had cash, cash equivalents and investments totaling $1.2 billion , as compared to $1.4 billion on December 31, 2016 .

Commenting on the quarter, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "We achieved a number of important strategic milestones during the first half of 2017, including record revenues, the development and approval of Brineura in the U.S. and Europe in less than four years and unprecedented data with BMN 270, the first-in-human gene therapy product for the treatment of severe hemophilia A." Mr. Bienaimé continued, "We expect that trajectory to continue during the second half of 2017 as we execute our Phase 3 program developing both the 4e13 vg/kg and 6e13 vg/kg doses in two separate studies with BMN 270; filing the MAA for pegvaliase in Europe ; and providing updates across our development pipeline at R&D Day on October 18 . With these investments in our pipeline and commercial organization, while we expect to incur a GAAP net loss for 2017, we expect to achieve non-GAAP income for the full-year 2017, as planned."

Revenues (in millions of U.S. dollars, unaudited) Total BioMarin Revenues

Details of Net Product Revenues Attributable to Aldurazyme

Updated 2017 Financial Guidance

Revenue Guidance ($ in millions)

Select Income Statement Guidance ($ in millions, except percentages)

Key Program Updates

A total of six patients received a single dose of BMN 270 at the 4e13 vg/kg dose. Based on the most recent data, for the three patients who were given the 4e13 vg/kg dose in November/ December 2016 , at week 32, all are in or near to the normal range of Factor VIII activity levels, with both median and mean Factor VIII levels of 51%. For the cohort of three patients who were given the 4e13 vg/kg dose in February/ March 2017 , at week 20, their Factor VIII activity levels have all moved into the mild range and two of the three are continuing to trend upward. For all six patients who received a dose of 4e13 vg/kg, at week 20, the median Factor VIII level was 34% and the mean was 31%. According to the World Federation of Hemophilia rankings of severity of hemophilia A, the mild hemophilia A range of Factor VIII activity levels is between 5% and 40%, and the normal range of Factor VIII activity levels for people without disease is between 50% and 150%, in each case expressed as a percentage of normal factor activity in blood.

Based on these updated results, BioMarin plans to initiate two separate Phase 3 studies as soon as possible, one with the 4e13 vg/kg dose and one with the 6e13 vg/kg dose. In addition, the Company has commissioned its commercial gene therapy manufacturing facility and expects to start the Phase 3 program in the fourth quarter of 2017. (See press release on 4e13 dose update from earlier today)

6e13 vg/kg data as announced at ISTH July 11, 2017 : As of the May 31, 2017 data cutoff as announced at ISTH, all patients at the 6e13 vg/kg dose had reached 52 weeks of post-treatment follow-up. Median and mean Factor VIII levels from week 20 through 52 for the 6e13 vg/kg dose cohort have been consistently within the normal levels post treatment as a percentage calculated based on the number of International Units per deciliter (IU/dL) of plasma. At one year after dosing, the median and mean Factor VIII levels of the 6e13 vg/kg cohort continue to be above 50%. For the six patients who were on pre-study prophylaxis after receiving a single dose of BMN 270 at 6e13 vg/kg dose and after reaching a Factor VIII level above 5%, the mean Annualized Bleed Rate (ABR) was reduced by 97% from 16.3 to 0.5. The median ABR for those same patients was reduced from 16.5 to zero. The mean annualized Factor VIII infusions for the 6e13 vg/kg dose cohort were reduced by 94% from 136.7 to 8.5. The median annualized Factor VIII infusions for those same patients were reduced from 138.5 to zero.

In the open-label Phase 1/2 study, a total of 15 patients with severe hemophilia A 1 (defined by the World Federation of Hemophilia (WFH) as having Factor VIII activity levels less than 1%, expressed as a percentage of normal factor activity in blood) received a single dose of BMN 270, seven of whom were treated at a dose of 6e13 vg/kg and an additional six of whom were subsequently treated at a lower dose of 4e13 vg/kg. The other two patients in the study were treated at lower doses as part of dose escalation in the study and did not achieve therapeutic efficacy.

In 2016, the Company announced pivotal results for the Phase 3 PRISM-2 study (formerly referred to as 165-302) with pegvaliase demonstrating that the primary endpoint of change in blood Phe compared with placebo (p<0.0001) had been met. The pegvaliase treated group maintained mean blood Phe levels at 527.2 umol/L compared to their Randomized Discontinuation Trial (RDT) baseline of 503.9 umol/L, whereas the placebo treated group mean blood Phe levels increased to 1385.7 umol/L compared to their RDT baseline of 536.0 umol/L. The treatment effect demonstrated in this study represents an approximately 62% improvement in blood Phe compared to placebo.

On June 1 , the Company announced that the European Commission (E.C.) had granted marketing authorization for Brineura, the first treatment approved in the European Union for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. The dosing administration includes all ages from birth. With approval from the E.C., BioMarin received marketing authorization for Brineura in all 28 countries of the European Union, Norway , Iceland and Liechtenstein .

The commercial launch of Brineura is well underway in the U.S. and Europe . The Company recorded its first commercial sales of Brineura in the second quarter of 2017 in the U.S. and Argentina and in the third quarter of 2017, both France and Germany had their first commercial sales.

Under the terms of the settlement, BioMarin will grant Par a non-exclusive license to its patents related to Kuvan to allow Par to market a generic version of sapropterin dihydrochloride 100mg tablets and powder for oral solution in 100mg and 500 mg sachets in the U.S. for the indications approved for Kuvan beginning October 1, 2020 if Par is entitled to the statutory 180-day first filer exclusivity period; April 1, 2021 if Par is not entitled to the statutory 180-day first filer exclusivity period; or earlier under certain circumstances. Additional details of the agreement remain confidential. BioMarin continues to vigorously enforce its intellectual property related to Kuvan. BioMarin holds patents in Europe related to Kuvan that are valid until at least 2024.

The Phase 3 study is a randomized, placebo-controlled study of vosoritide in approximately 110 children with achondroplasia ages 5 -14 for 52 weeks. The study will be followed by a subsequent open-label extension. Children in this study will have completed a minimum six-month baseline study to determine their respective baseline growth velocity prior to entering the Phase 3 study. Vosoritide is being tested in children in the age range where their growth plates are still open. This is approximately 25 percent of people with achondroplasia.

The Company intends to provide an update on the most recent data available with vosoritide at the upcoming R&D Day event in New York City on October 18, 2017 .

Under the terms of the license and settlement agreements, Sarepta will make a one-time payment of $35 million to BioMarin as well as certain additional regulatory and commercial milestone payments and net sales royalties for exons 51, 45, 53 and possibly on future exon-skipping products. The Company expects to recognize the $35 million payment in the third quarter of 2017 as Royalty and other revenues and no amounts are expected to be deferred or amortized.

Conference Call Details

BioMarin will host a conference call and webcast to discuss second quarter 2017 financial results today, Wednesday, August 2, 2017 at 4:30 p.m. ET . This event can be accessed on the investor section of the BioMarin website at www.biomarin.com .

U.S. / Canada Dial-in Number: 866.502.9859 International Dial-in Number: 547.990.1362 Conference ID: 41574149

Replay Dial-in Number: 855.859.2056 Replay International Dial-in Number: 404.537.3406 Conference ID: 41574149

About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six approved products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com .

BioMarin ® , Vimizim ® , Naglazyme ® , Kuvan ® and Firdapse ® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. Brineura ™ and Kyndrisa ™ are trademarks of BioMarin Pharmaceutical Inc. Aldurazyme ® is a registered trademark of BioMarin/Genzyme LLC.

1 Source: World Federation of Hemophilia http://www.wfh.org/en/resources/annual-global-survey http://www.wfh.org/en/abd/prophylaxis/prophylaxis-administration-and-dosing-schedules

Non-GAAP Information

The results presented in this press release for the three and six months ended June 30, 2017 and 2016 include both GAAP information and non-GAAP information. As used in this release, non-GAAP income (loss) is defined by the Company as GAAP net loss excluding net interest expense, provision for (benefit from) income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and certain other specified items, as detailed below. In addition, BioMarin includes in this press release the effects of these adjustments on certain components of GAAP net loss for each of the periods presented. In this regard, non-GAAP income (loss) and its components, including non-GAAP Royalty, License and other revenues, non-GAAP Cost of sales, non-GAAP Research and development expenses, non-GAAP Selling, general and administrative expense, non-GAAP Intangible asset amortization and contingent consideration and non-GAAP Provision for (benefit from) income taxes are statement of operations line items prepared on the same basis as, and therefore components of, the overall non-GAAP measures.

Non-GAAP income (loss) and its components are not meant to be considered in isolation, as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the Company's results of operations as determined in accordance with GAAP. Investors should also note that these non-GAAP measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the Company may exclude for purposes of its non-GAAP measures; likewise, the Company may in the future cease to exclude items that it has historically excluded for purposes of its non-GAAP measures. Because of the non-standardized definitions, the non-GAAP measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.

The following table presents the reconciliation of GAAP Net Loss to non-GAAP Income (Loss):

The following reconciliation of the GAAP reported to non-GAAP information provides the details of the effects of the non-GAAP adjustments on certain components of the Company's operating results for each of the periods presented.

SOURCE BioMarin Pharmaceutical Inc.