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Financial Highlights ($ in millions, except per share data, unaudited) | ||||||
Three Months Ended June 30, | Six Months Ended June 30, | |||||
2014 | 2013 | % Change | 2014 | 2013 | % Change | |
Total BioMarin Revenue | $ 191.7 | $ 136.8 | 40.1% | $ 343.3 | $ 264.7 | 29.7% |
VIMIZIM Net Product Revenue | 14.3 | -- | 15.2 | -- | ||
Naglazyme Net Product Revenue | 98.3 | 69.9 | 40.6% | 178.4 | 139.3 | 28.1% |
Kuvan Net Product Revenue | 46.9 | 40.9 | 14.7% | 92.1 | 78.5 | 17.3% |
Aldurazyme Net Product Revenue | 24.1 | 17.5 | 37.7% | 42.2 | 34.2 | 23.4% |
Aldurazyme Royalty Revenue | ||||||
(excluding Net Product Transfer Revenue) - non-GAAP | 24.4 | 21.2 | 15.1% | 46.3 | 40.5 | 14.3% |
Firdapse Net Product Revenue | 4.6 | 4.1 | 12.2% | 9.3 | 7.7 | 20.8% |
Non-GAAP Net Income (Loss) | $ 10.8 | $ -- | $ 9.1 | $ (8.0) | ||
Non-GAAP Net Income (Loss) per Share - Basic | $ 0.07 | $ -- | $ 0.06 | $ (0.06) | ||
Non-GAAP Net Income (Loss) per Share - Diluted | $ 0.06 | $ -- | $ 0.05 | $ (0.06) | ||
GAAP Net Loss | $ (33.5) | $ (21.5) | $ (71.6) | $ (61.3) | ||
GAAP Net Loss per Share -- Basic | $ (0.23) | $ (0.15) | $ (0.49) | $ (0.46) | ||
GAAP Net Loss per Share -- Diluted | $ (0.23) | $ (0.16) | $ (0.50) | $ (0.46) | ||
Cash, cash equivalents and investments | $1,080.8 | $ 518.5 | 108.4% |
SAN RAFAEL, Calif., July 30, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced financial results for the second quarter ended June 30, 2014. Non-GAAP net income was $10.8 million, or $0.07 per basic share and $0.06 per diluted share for the second quarter of 2014, compared to non-GAAP net loss of $0.0 million for the second quarter of 2013. Non-GAAP net income was $9.1 million, or $0.06 per basic share and $0.05 per diluted share for the six months ended June 30, 2014, compared to a loss of ($8.0) million, or ($0.06) per basic and diluted share for the six months ended June 30, 2013.
GAAP net loss was ($33.5) million, or ($0.23) per basic and diluted share for the second quarter of 2014, compared to GAAP net loss of ($21.5) million, or ($0.15) per basic share and ($0.16) per diluted share for the second quarter of 2013. GAAP net loss for the six months ended June 30, 2014 was ($71.6) million, or ($0.49) per basic share and ($0.50) per diluted share, as compared to GAAP net loss of ($61.3) million, or ($0.46) per basic and diluted share for the six months ended June 30, 2013.
The increased non-GAAP net income for the second quarter of 2014 compared to the second quarter of 2013 was primarily due to strong uptake of VIMIZIM® in its first full quarter of commercial sales, and significant growth in revenues from our other commercial products including, Naglazyme®, KUVAN®, Aldurazyme® and Firdapse®. Naglazyme revenue growth of 41% in the quarter compared to the prior year reflected both continued growth in number of patients consistent with long term trends, and also included significant purchases from certain governmental entities which potentially reflects patient demand in future quarters. These revenue increases were partially offset by increased research and development expenses for PEG PAL, BMN 190 and BMN 673, as well as increased selling, general and administrative expenses related to VIMIZIM launch activities. Increased GAAP net loss was also driven by increased income tax expense and interest expense in the second quarter. These measures are excluded from the non-GAAP measurements. As of June 30, 2014, BioMarin had cash, cash equivalents and investments totaling $1,080.8 million, as compared to $518.5 million on June 30, 2013.
"With the strong launch of VIMIZIM and continued double-digit year over year growth of our other products, we believe the ultra-orphan business model is as robust as ever. We are extremely pleased with the momentum of the U.S. commercial launch of VIMIZIM. At the end of the second quarter, 132 patients had started receiving commercial VIMIZIM," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "Since receiving approval of VIMIZIM in the European Union in April, we are making good progress seeking reimbursement on a country by country basis. The excitement we are seeing in the Morquio community is driving our expectation of continued, rapid uptake of VIMIZIM across all our commercial markets. With E.U. approval in hand, we believe VIMIZIM sales combined with the strength of our other marketed products will help BioMarin reach over $1 billion in revenues over the next 2-3 years."
Net Product Revenue (unaudited)
Total Revenue Growth (in millions) | ||||||||
Three Months Ended June 30, | Six Months Ended June 30, | |||||||
2014 | 2013 | $ Change | % Change | 2014 | 2013 | $ Change | % Change | |
VIMIZIM | $ 14.3 | $ -- | $ 14.3 | -- | $ 15.2 | $ -- | $ 15.2 | -- |
Naglazyme (1) | 98.3 | 69.9 | 28.4 | 40.6% | 178.4 | 139.3 | 39.1 | 28.1% |
Kuvan | 46.9 | 40.9 | 6.0 | 14.7% | 92.1 | 78.5 | 13.6 | 17.3% |
Aldurazyme | 24.1 | 17.5 | 6.6 | 37.7% | 42.2 | 34.2 | 8.0 | 23.4% |
Firdapse | 4.6 | 4.1 | 0.5 | 12.2% | 9.3 | 7.7 | 1.6 | 20.8% |
Net product revenue | 188.2 | 132.4 | 55.8 | 42.1% | 337.2 | 259.7 | 77.5 | 29.8% |
Collaborative agreement revenue | 0.5 | 0.9 | (0.4) | 0.9 | 1.0 | (0.1) | ||
Royalty and license revenue | 3.0 | 3.5 | (0.5) | 5.2 | 4.0 | 1.2 | ||
Total BioMarin revenue - GAAP | 191.7 | 136.8 | 54.9 | 40.1% | 343.3 | 264.7 | 78.6 | 29.7% |
Less: Previously recognized Aldurazyme net product transfer revenue | (0.3) | (3.7) | 3.4 | (4.1) | (6.3) | 2.2 | ||
Total BioMarin revenues (excluding Aldurazyme net product transfer revenue) - Non-GAAP (2) | $ 192.0 | $ 140.5 | $ 51.5 | 36.7% | $ 347.4 | $ 271.0 | $ 76.4 | 28.2% |
Reconciliation of Aldurazyme Revenues (in millions) | ||||||||
Three Months Ended June 30, | Six Months Ended June 30, | |||||||
2014 | 2013 | $ Change | % Change | 2014 | 2013 | $ Change | % Change | |
Aldurazyme revenue reported by Genzyme | $ 62.3 | $ 53.6 | $ 8.7 | 16.2% | $ 118.2 | $ 102.1 | $ 16.1 | 15.8% |
Three Months Ended June 30, | Six Months Ended June 30, | |||||||
2014 | 2013 | $ Change | 2014 | 2013 | $ Change | |||
Aldurazyme Royalties due from Genzyme - Non-GAAP (2) | $ 24.4 | $ 21.2 | $ 3.2 | $ 46.3 | $ 40.5 | $ 5.8 | ||
Previously recognized net product transfer revenue (3) | (0.3) | (3.7) | 3.4 | (4.1) | (6.3) | 2.2 | ||
Total Aldurazyme net product revenue - GAAP | $ 24.1 | $ 17.5 | $ 6.6 | $ 42.2 | $ 34.2 | $ 8.0 | ||
(1) 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. | ||||||||
(2) BioMarin believes that this non-GAAP information is useful to investors, taken in conjunction with BioMarin's GAAP information because it provides additional information regarding the end-user demand for Aldurazyme. The Aldurazyme net product transfer revenue is the result of timing of deliveries to Genzyme Corp. and is therefore not representative of patient demand for the product. By providing information about both the GAAP and non-GAAP revenue measures, the Company believes that the additional information enhances investors' overall understanding of the Company's business and in particular allows for more consistent period to period evaluation of the revenue. | ||||||||
(3) To the extent units shipped to third party customers by Genzyme exceed BioMarin inventory transfers to Genzyme, BioMarin will record a decrease in net product revenue from the royalty payable to BioMarin for the amount of previously recognized product transfer revenue. If BioMarin inventory transfers exceed units shipped to third party customers by Genzyme, BioMarin will record incremental net product transfer revenue for the period. |
Priority Review Voucher Sold for $67.5 million
The Company also announced today that it had sold the Rare Pediatric Disease Priority Review Voucher (PRV) it obtained in February of this year. The Company received the voucher under an FDA program intended to encourage the development of treatments for rare pediatric diseases. BioMarin was awarded the voucher when it received approval of VIMIZIM®, a new biological product for patients with Mucopolysaccharidosis type IVA, also known as Morquio A syndrome. BioMarin received $67.5 million from Regeneron Ireland, an indirect, wholly-owned subsidiary of Regeneron Pharmaceuticals, Inc., in exchange for the voucher.
Updated 2014 Guidance, as of July 30, 2014
Revenue Guidance | ||
($ in millions) | ||
Initially Provided | Updated | |
Item | February 26, 2014 | July 30, 2014 |
Total BioMarin Revenues | $650 to $680 | $745 to $765* |
Naglazyme Net Product Revenue | $290 to $310 | $305 to $320 |
Kuvan Net Product Revenue | $180 to $200 | $180 to $200 |
VIMIZIM | $60 to $70 | $60 to $70 |
* Includes of $67.5 million for the sale of the Priority Review Voucher. | ||
Selected Income Statement Guidance | ||
($ in millions, except percentages) | ||
Initially Provided | Updated | |
Item | February 26, 2014 | July 30, 2014 |
Cost of Sales (% of Total Revenue) | 17.5% to 18.5% | 16.5% to 17.5% |
Research and Development Expense | $500 to $530 | $460 to $480 |
Selling, General and Admin. Expense | $265 to $285 | $280 to $295 |
GAAP Net Loss | $(255) to $(285) | $(180) to $(195)* |
Non-GAAP Net Loss | $(100) to $(130) | $(60) to $(80) |
* Includes $50 million net of taxes, related to the sale of the Priority Review Voucher. |
Anticipated Upcoming Milestones
4Q 2014: Enrollment completion of Phase 1/2 trial with BMN 190 for the treatment of Batten disease |
1Q 2015: IND filing or equivalent for BMN 270 for the treatment of Hemophilia A |
2Q 2015: Data on first three cohorts in Phase 2 with BMN 111 for the treatment of Achondroplasia |
1H 2015: Enrollment completion of Phase 2/3 trial with BMN 701 for the treatment of Pompe disease |
2H 2015: Results from Phase 1/2 trial with BMN 190 for the treatment of Batten disease |
2H 2015: Enrollment completion of Phase 3 trial with talazoparib (BMN 673) for the treatment of mBC |
Mid-2015: IND filing or equivalent for BMN 250 for the treatment of MPS IIIB |
4Q 2015: Results from pivotal Phase 3 trial with PEG PAL for the treatment of PKU |
1Q 2016: Submission of PEG PAL BLA to the FDA for the treatment of PKU |
Commercial and Regulatory Update on VIMIZIM for Mucopolysaccharidosis type IVA
Advanced Clinical Programs
Phase 3 with talazoparib (BMN 673) for gBRCA breast cancer: The Company is conducting a Phase 3 trial to study talazoparib for the treatment of deleterious germline BRCA mutation metastatic breast cancer and is currently enrolling patients. The Phase 3 trial is an open-label, 2:1 randomized, parallel, two-arm study of talazoparib as compared to monotherapy of physicians' choice (capecitabine, eribulin, gemcitabine or vinorelbine) in germline BRCA mutation subjects with locally advanced and/or metastatic breast cancer who have received no more than two prior chemotherapy regimens for metastatic disease. The global study will enroll approximately 429 subjects. The primary objective of the study is to compare progression-free survival (PFS) of subjects treated with talazoparib as a monotherapy relative to those treated with protocol-specific physicians' choice.
Early-stage Clinical Programs
Preclinical Programs
Non-GAAP Net Income (Loss) and Reconciliation
The results for the three and six months ended June 30, 2014 and June 30, 2013 and the guidance for 2014 include both GAAP net loss and non-GAAP net income/loss. As used in this release, non-GAAP net income/loss is based on GAAP earnings before interest, taxes, depreciation and amortization (EBITDA) and further adjusted to exclude certain non-cash stock compensation expense, non-cash contingent consideration expense and certain other nonrecurring material items (non-GAAP net income/loss).
The following table presents the reconciliation of GAAP to non-GAAP financial metrics:
Reconciliation of GAAP Net Loss to Non-GAAP Net Income (Loss) | |||||
(in millions) | |||||
(unaudited) | |||||
Three Months Ended June 30, |
Six Months Ended June 30, |
Year Ending December 31, |
|||
2014 | 2013 | 2014 | 2013 | 2014 Guidance | |
GAAP Net Loss | $ (33.5) | $ (21.5) | $ (71.6) | $ (61.3) | $(180.0) - $(195.0) |
Interest expense, net | 7.5 | -- | 15.5 | 1.0 | 31.3 |
Provision for (benefit from) income taxes | 5.8 | 1.2 | 9.3 | (3.5) | 14.5 - 19.5 |
Depreciation expense | 8.0 | 6.6 | 14.6 | 12.7 | 32.0 |
Amortization expense | 2.9 | 3.6 | 5.5 | 6.2 | 11.1 |
EBITDA Loss | (9.3) | (10.1) | (26.7) | (44.9) | (86.1) - (106.1) |
Stock-based compensation expense | 17.3 | 13.9 | 33.6 | 25.5 | 84.6 |
Contingent consideration expense (1) | 2.6 | (3.8) | 10.8 | 1.0 | 17.6 |
Non-recurring: | |||||
Sale of Priority Review Voucher (4) | (67.5) | ||||
Gain on termination of lease (2) | (0.5) | -- | (9.3) | -- | (9.3) |
Debt conversion expense (3) | 0.7 | -- | 0.7 | 10.4 | 0.7 |
Non-GAAP Net Income (Loss) | $ 10.8 | $ -- | $ 9.1 | $ (8.0) | $(60.0) - $(80.0) |
(1) Represents the expense associated with the change in the fair value of contingent acquisition consideration payable for the period, resulting from changes in estimated probabilities and timing of achieving certain developmental milestones. | |||||
(2) Represents the net gain due to the early termination of the Company's operating lease and the realization of the remaining balance in deferred rent upon acquisition of the San Rafael Corporate Center where the Company's corporate headquarters are located, as well as other facilities the Company has acquired that were previously under operating lease. | |||||
(3) Represents debt conversion expense associated with the early conversion of a portion of our 2017 convertible notes. | |||||
(4) Represents the total sales price of $67.5 million for the PRV, or approximately $50.0 million net of tax. |
BioMarin believes that this non-GAAP information is useful to investors, taken in conjunction with BioMarin's GAAP information because it provides additional information regarding the performance of BioMarin's core ongoing business, VIMIZIM, Naglazyme, Kuvan, Aldurazyme and Firdapse, and development of the Company's pipeline. By providing information about both the overall GAAP financial performance and the non-GAAP measures that focus on continuing operations, the Company believes that the additional information enhances investors' overall understanding of the Company's business and prospects for the future. Further, the Company uses both the GAAP and the non-GAAP results and expectations internally for its operating, budgeting and financial planning purposes.
Conference Call Details
BioMarin will host a conference call and webcast to discuss second quarter 2014 financial results today, Wednesday, July 30, at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.BMRN.com.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises five approved products and multiple clinical and pre-clinical product candidates. Approved products include: Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; KUVAN® (sapropterin dihydrochloride) Powder for Oral Solution and Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS); and VIMIZIM® (elosulfase alfa) for the treatment of Morquio A (MPS IVA). Product candidates include: BMN 165 (PEGylated recombinant phenylalanine ammonia lyase), also referred to as PEG PAL, which is currently in Phase 3 clinical development for the treatment of PKU; talazoparib (BMN 673), a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer; BMN 701, a novel fusion of acid alpha glucosidase (GAA) with a peptide derived from insulin like growth factor 2, which is currently in Phase 3 clinical development for the treatment of Pompe disease; BMN 111, a modified C-natriuretic peptide, which is currently in Phase 2 clinical development for the treatment of achondroplasia; and BMN 190, a recombinant human tripeptidyl peptidase-1 (rhTPP1), which is currently in Phase 1 for the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.
CONTACT: Investors: Traci McCarty BioMarin Pharmaceutical Inc. (415) 455-7558 Media: Debra Charlesworth BioMarin Pharmaceutical Inc. (415) 455-7451