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0001193125-14-439080.txt : 201412100001193125-14-439080.hdr.sgml : 2014121020141210171233ACCESSION NUMBER:0001193125-14-439080CONFORMED SUBMISSION TYPE:SC TO-CPUBLIC DOCUMENT COUNT:51FILED AS OF DATE:20141210DATE AS OF CHANGE:20141210GROUP MEMBERS:BIOMARIN FALCONS B.V.

SUBJECT COMPANY:

COMPANY DATA:COMPANY CONFORMED NAME:Prosensa Holding N.V.CENTRAL INDEX KEY:0001574111STANDARD INDUSTRIAL CLASSIFICATION:PHARMACEUTICAL PREPARATIONS [2834]IRS NUMBER:000000000STATE OF INCORPORATION:P7FISCAL YEAR END:1231

FILING VALUES:FORM TYPE:SC TO-CSEC ACT:1934 ActSEC FILE NUMBER:005-87624FILM NUMBER:141278713

BUSINESS ADDRESS:STREET 1:J.H. OORTWEG 21CITY:2133 CH LEIDENSTATE:P7ZIP:00000BUSINESS PHONE:31 0 713320100

MAIL ADDRESS:STREET 1:J.H. OORTWEG 21CITY:2133 CH LEIDENSTATE:P7ZIP:00000

FORMER COMPANY:FORMER CONFORMED NAME:Prosensa Holding B.V.DATE OF NAME CHANGE:20130410

FILED BY:

COMPANY DATA:COMPANY CONFORMED NAME:BIOMARIN PHARMACEUTICAL INCCENTRAL INDEX KEY:0001048477STANDARD INDUSTRIAL CLASSIFICATION:PHARMACEUTICAL PREPARATIONS [2834]IRS NUMBER:680397820STATE OF INCORPORATION:DEFISCAL YEAR END:1231

FILING VALUES:FORM TYPE:SC TO-C

BUSINESS ADDRESS:STREET 1:105 DIGITAL DRIVECITY:NOVATOSTATE:CAZIP:94949BUSINESS PHONE:4155066700

MAIL ADDRESS:STREET 1:105 DIGITAL DRIVECITY:NOVATOSTATE:CAZIP:94949

SC TO-C1d836571dsctoc.htmSC TO-C

SC TO-C

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

SCHEDULE TO

(RULE 14d100)

Tender Offer Statement Pursuant to Section14(d)(1) or 13(e)(1)

of the Securities Exchange Act of 1934

ProsensaHolding N.V.

(Name of Subject Company)

BioMarin Falcons B.V.

(Offeror)

BioMarinPharmaceutical Inc.

(Parent of Offeror)

(Names of Filing Persons)

ORDINARYSHARES, PAR VALUE 0.01 PER SHARE

(Title of Class of Securities)

N71546100

(Cusip Numberof Class of Securities)

G. Eric Davis

Senior Vice President, General Counsel

BioMarin Pharmaceutical Inc.

770 Lindaro Street

SanRafael, California 94901

(415) 506-6700

(Name, Address and Telephone Number of Person Authorized to Receive Notices and Communications on Behalf of Filing Persons)

With a copy to:

Kevin Espinola

JonesDay

3161 Michelson Dr., Suite 800

Irvine, California 92612

(949) 851-3939

CALCULATIONOF FILING FEE

Transaction Valuation*Amount of Filing Fee*

N/A*N/A*

*A filing fee is not required in connection with this filing as it relates solely to preliminary communications made before the commencement of the tender offer.

Check the box if any part of the fee is offset as provided by Rule 0-11(a)(2) and identify the filing with which the offsetting fee was previously paid. Identify the previous filing by registration statement number, orthe Form or Schedule and the date of its filing.

Amount Previously Paid: n/aFiling Party: n/a

Form of Registration No.: n/aDate Filed: n/a

xCheck the box if the filing relates solely to preliminary communications made before the commencement of a tender offer.

Check the appropriate boxes below to designate any transactions to which the statement relates:

xThird-party tender offer subject to Rule 14d-1.

Issuer tender offer subject to Rule 13e-4.

Going-private transaction subject to Rule 13e-3.

Amendment to Schedule 13D under Rule 13d-2.

Check the following box if the filing is a finalamendment reporting the results of the tender offer.

If applicable, check theappropriate box(es) below to designate the appropriate rule provision(s) relied upon:

Rule 13e-4(i) (Cross-Border Issuer Tender Offer)

Rule 14d-1(d) (Cross-Border Third-Party Tender Offer)

This filing relates solely to preliminary communications made before the commencement of a tender offer (theOffer) by BioMarin Falcons B.V., (Buyer) a private company with limited liability organized under the laws of The Netherlands and an indirect wholly owned subsidiary of BioMarin Pharmaceutical Inc., a Delawarecorporation (BioMarin or the Company), to purchase all of the issued and outstanding ordinary shares, par value 0.01per share (the Shares), of Prosensa Holding N.V., a publiclimited liability company organized under the laws of The Netherlands (Prosensa), at a purchase price of $17.75 per Share, net to the seller in cash, plus one non-transferrable contingent value right per Share, which representsthe contractual right to receive a cash payment of up to $4.14 per Share upon the achievement of certain product approval milestones, in each case, without interest thereon and less any applicable withholding taxes, upon the terms and subject to theconditions contained in the Purchase Agreement, dated as of November23, 2014, by and among Buyer, BioMarin and Prosensa.

ADDITIONAL INFORMATION

The Offer described in this communication and related exhibits has not yet commenced, and this communication and related exhibits is neither an offerto purchase nor a solicitation of an offer to sell any ordinary shares of Prosensa or any other securities. On the commencement date of the Offer, a tender offer statement on Schedule TO, including an offer to purchase, a letter of transmittal andrelated documents, will be filed with the United States Securities and Exchange Commission (the SEC). Thereafter, Prosensa will file a solicitation/recommendation statement on Schedule 14D-9 with the SEC. The offer to purchaseordinary shares of Prosensa will only be made pursuant to the offer to purchase, the letter of transmittal and related documents filed as a part of the Schedule TO.

INVESTORS AND SECURITY HOLDERS OF PROSENSA ARE URGED TO READ BOTH THE SCHEDULE TO (AND THE INCLUDED OFFER TO PURCHASE) AND THE SOLICITATION/RECOMMENDATIONSTATEMENT, AS THEY MAY BE AMENDED FROM TIME TO TIME, AND OTHER RELEVANT DOCUMENTS FILED WITH THE SEC WHEN THEY BECOME AVAILABLE BEFORE THEY MAKE ANY DECISION WITH RESPECT TO THE TENDER OFFER, BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT THETERMS OF THE OFFER, THE PROPOSED TRANSACTIONS AND THE PARTIES THERETO.

The tender offer statement will be filed with the SEC by BioMarin and Buyer,and the solicitation/recommendation statement will be filed with the SEC by Prosensa. Investors and security holders may obtain a free copy of these statements (when available) and other documents filed with the SEC at the website maintained by theSEC at www.sec.gov or by directing such requests to the Information Agent for the tender offer that will be named in the tender offer statement on Schedule TO.

Cautionary Note Regarding Forward-Looking Statements

Anystatements contained in this document that are not historical facts are forward-looking statements as defined in the U.S. Private Securities Litigation Reform Act of 1995. Words such as anticipate, believe,estimate, expect, forecast, intend, may, plan, project, predict, should and will and similar expressions as they relate tothe Company, Buyer or Prosensa are intended to identify such forward-looking statements. These forward-looking statements involve risks and uncertainties concerning the parties ability to initiate the tender offer, close the transaction,terminate the Purchase Agreement, the expected closing date of the transaction, the anticipated benefits and synergies of the proposed transaction, anticipated future combined operations, products and services. Actual events or results may differmaterially from those described in this document due to a number of risks and uncertainties. These potential risks and uncertainties include,

among others, the outcome of regulatory reviews of the proposed transaction, the ability of the parties to complete the transaction, and other risks detailed in the Companys andProsensas SEC filings, including those discussed in the Companys Annual Report on Form 10-K for the year ended December31, 2013 and in any subsequent periodic reports on Form 10-Q and Form 8-K and Prosesas Annual Report onForm 20-F for the year ended December31, 2013 and in any subsequent reports on Form 6-K, each of which is on file with the SEC and available at the SECs website at www.sec.gov. The Company is not obligated to update these forward-lookingstatements to reflect events or circumstances after the date of this document. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of their dates.

EXHIBIT INDEX

Exhibit99.1Slides Presented at Investor Conference, dated December10, 2014

EX-99.12d836571dex991.htmEX-99.1

EX-99.1

Exhibit 99.1

WELCOME BIOMARIN ANALYST AND INVESTOR DAY 2014 Jean-Jacques Bienaime Chief Executive Officer Hank Fuchs Chief Medical Officer

ANALYST DAY 2014 Safe Harbor Statement This non-confidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc.,including potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarins product programs, actions of regulatory authorities, availability of capital,future actions in the pharmaceutical market and developments by competitors, and those factors detailed in BioMarins filings with the Securities and Exchange Commission such as 10-Q, 10-K and 8-K reports. 2

ANALYST DAY 2014 Additional Information The offer by BioMarin to purchase ordinary shares of Prosensa Holding N.V. (Prosensa) contemplated by the Purchase Agreement, datedNovember 23, 2014, between BioMarin, Prosensa and BioMarin Falcons B.V. (the Offer) has not yet commenced. This communication is neither an offer to purchase nor a solicitation of an offer to sell any ordinary shares of Prosensa or anyother securities. On the commencement date of the Offer, a tender offer statement on Schedule TO, including an offer to purchase, a letter of transmittal and related documents, will be filed with the United States Securities and Exchange Commission(the SEC). Thereafter, Prosensa will file a solicitation/recommendation statement on Schedule 14D-9 with the SEC. The offer to purchase ordinary shares of Prosensa will only be made pursuant to the offer to purchase, the letter oftransmittal and related documents filed as a part of the Schedule TO. INVESTORS AND SECURITY HOLDERS OF PROSENSA ARE URGED TO READ BOTH THE SCHEDULE TO (AND THE INCLUDED OFFER TO PURCHASE) AND THE SOLICITATION/ RECOMMENDATION STATEMENT, AS THEY MAYBE AMENDED FROM TIME TO TIME, AND OTHER RELEVANT DOCUMENTS FILED WITH THE SEC WHEN THEY BECOME AVAILABLE BEFORE THEY MAKE ANY DECISION WITH RESPECT TO THE TENDER OFFER, BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT THE TERMS OF THE OFFER,THE PROPOSED TRANSACTIONS AND THE PARTIES THERETO. The tender offer statement will be filed with the SEC by BioMarin and the solicitation/recommendation statement will be filed with the SEC by Prosensa. Investors and security holders may obtain afree copy of these statements (when available) and other documents filed with the SEC at the website maintained by the SEC at www.sec.gov or by directing such requests to the information agent for the tender offer that will be named in the tenderoffer statement on Schedule TO. 3

ANALYST DAY 2014 2014: BioMarins Most Productive Year to Date 2015 will be more productive 5 Marketed Products to Generate over $700M in 2014 Revenues Most Diverse Product Pipelinein Orphan-drug Industry 5 Potential Products Currently in the Clinic; 8 in 2015* Vimizim to Generate ~$100M in First Four Quarters of Sales Prosensa Acquisition Could Drive Significant Near-term Upside * 12 including drisapersen and other clinicalcandidates from Prosensa acquisition 4

ANALYST DAY 2014 Prosensa Acquisition Highlights Great strategic fit and value creator for BioMarin Drisapersen has a large body of clinical data that could support early regulatoryapproval Leverages BioMarins extensive experience getting orphan drugs approved DMD is one of the largest monogenetic disorders (approx. 75,000 WW) Leverages our global commercial infrastructure and leadership in orphan disease market Addswhat could be our largest product to a well diversified product portfolio With early approvals, accretive to operating and GAAP earnings in 2017 A potentially transformative investment that is risk managed

ANALYST DAY 2014 Why Prosensa? First to file in the U.S. for the treatment of Duchenne muscular dystrophy Our belief that the totality of drisapersen data combined with the significantunmet medical need of this patient population provide a path to early approvals Largest data set in DMD comprising three placebo-controlled studies and two long-term open label studies, treating over 300 patients New Drug Application (NDA) withBreakthrough Therapy designation currently under rolling review based on existing data; complete NDA submission expected 1Q15; MAA in EMA expected 2015 In-line with FDA guidance, two confirmatory post-approval studies to support accelerated approvalplanned to begin 1H15 Issued U.S. patents through 2023; EU through 2021; potential for extensions Low dose that supports sub-cutaneous route of administration Anticipated low COGS and available manufacturing capacity to meet market demand

ANALYST DAY 2014 Drisapersen: Timeline of Regulatory Events 4Q14 NDA rolling submission commenced with FDA 1Q15 anticipated completion of NDA submission with FDA 2Q15 expected MAAapplication for drisapersen with EMA 1H15 initiation of two confirmatory studies to support potential accelerated approval for drisapersen

ANALYST DAY 2014 History of Strong Revenue Growth Core Commercial Products Expected to Continue Growing VIMIZIM Naglazyme Kuvan Aldurazyme + Other $26 2005 $84 2006 $122 2007 $297 2008$325 2009 $376 2010 $441 2011 $501 2012 $549 2013 $700+ $65- $70 $305- $320 $190- $200 2014 (revenues in millions) Outlook 8

ANALYST DAY 2014 Whats New Today? Drisapersen Potential near-term registration opportunity in large market with unmet need Seamless fit within BioMarins clinical, regulatoryand commercial expertise BMN 111 for Achondroplasia First 3 cohorts enrolled Leveraging the natural history study to inform our understanding of our patients and analytic approaches BMN 190 for CLN2, Late Infantile Form of Batten Disease Enrollmentcompletion imminent Current study exhibiting excellent safety profile to date BMN 270 for Hemophilia A Launching Phase 1 study 1H15 on track two years after licensing Expanded pre-clinical proof of concept to include real world(clotting) assays

ANALYST DAY 2014 Whats New Today? PEG PAL for PKU Phe lowering in patients from enriched study at 26 weeks is robust at ~60% Enhanced strategies in place to facilitatecommercialization Pivotal data now expected 1Q16 BMN 701 for Late-onset Pompe Disease Improved manufacturing process with better purity, potency and COGS Modifying program to leverage to be commercialized process Proof of concept in 20patients of switch study; enrollment 1Q15/results 4Q15 Pivotal study resumes 4Q15 Talazoparib for mBRCA BC Enrolling more trials, including talazoparib in combination, neoadjuvant, etc. BMN 250 for MPS IIIB (Sanfilippo B syndrome) Launching naturalhistory study with patient accrual expected 2015 Phase 1 study in MPS IIIB patients 2H15

ANALYST DAY 2014 Agenda and Welcome External Speakers Cary O. Harding, M.D., F.A.C.M.G. Professor of Molecular and Medical Genetics and Pediatrics at Oregon Health & ScienceUniversity Medical Director of the OHSU Biochemical Genetics Laboratory Alfried Kohlschutter, M.D. Expert in NCL disorders (Neuronal Ceroid Lipofuscinoses, Batten disease) Specialty Clinic for Degenerative Brain Disorders in Children andAdolescents, associated with the Department of Pediatrics, University Medical Center in Hamburg, Germany Cathleen Raggio, M.D. Attending Orthopedic Surgeon, Co-Director, The Kathryn O. and Alan C. Greenberg Center for Skeletal Dysplasias at Hospitalfor Special Surgery, New York Craig McDonald, M.D. Professor and Chair, Department of Physical Medicine & Rehabilitation, Center for Healthcare Policy and Research U.C. Davis Childrens Hospital Expert in the clinical management andrehabilitation of neuromuscular diseases including muscular dystrophies and the development of novel outcome measures for clinical trials Glenn Pierce, M.D., Ph.D. Expert in the field of hemophilia with more than 20 years of experience Previouslyserved as the Chief Medical Officer and Senior Vice President of Biogen Idec Hemophilia Therapeutic Area and senior vice president of Hematology, Cell and Gene Therapies at Biogen Idec Inc. 11

PKU PROGRAM OVERVIEW FOCUS ON PEG PAL BIOMARIN ANALYST AND INVESTOR DAY 2014 Wolfgang Dummer, M.D., Ph.D., Vice President, Clinical Development BioMarin

Drisapersen: an overview of the clinical program in Duchenne muscular dystrophy

Craig McDonald, MD Professor and Chair Physical Medicine& Rehabilitation Professor of Pediatrics

Disclosures

Consulting work on Duchenne musculardystrophy clinical trials for

PTC Therapeutics

Sarepta

Prosensa

Pfizer

Eli Lilly

Halo Therapeutics

Bristol MyersSquib

Novartis

Italfarmaco

Mitokyne

Cardero Therapeutics

Site investigator for phase 3 trials for ataluren and eteplirsen Site investigator and study PI for DEMAND V study of drisapersen Site investigator for drisapersen extension trial

Introduction

Duchenne muscular dystrophy (DMD) isa severely debilitating and ultimately fatal childhood disease

Affects 1 in 35005000 newborn boys1,2

Characterized by a progressive decrease in muscle function3 leading to loss of ambulation, respiratory failureand cardiomyopathy Caused by mutations in the dystrophin gene, resulting in disruption of the open-reading frame, dystrophin deficiency and muscle fibre degeneration4

Exon skipping:

A therapeutic strategy forrestoring partial functional dystrophin expression

Aims to convert the more severe DMD into a milder Beckermuscular dystrophy (BMD) phenotype

Drisapersen:

A 2-O-methyl-phosphorothioate antisense oligonucleotide that induces skipping of exon 51 in the dystrophin pre-mRNA

Can be used to correct mutations occurring in approximately 13% of boys with DMD5

1. Ellis JA, et al. Neuromuscul Disord 2013;23:6829. 2. Mendell JR, et al. Muscle& Nerve2013;48:216. 3. Emery AE. Duchenne Muscular Dystrophy (3rd ed). Oxford University Press; Oxford, UK: 2003. 4. Aartsma-Rus A, et al. Muscle& Nerve 2006;34:13544. 5. Hammond SM, Wood MJ. Curr Opin Mol Ther 2010;12:47886.

2

Responsiveness of 6MWD to dystrophin restoration

-14 meters

- 44.1 meters

Minimal clinically importantdifference (MCID) in 6MWD in DMD has been calculated to be 30 meters (McDonald et al 2013)

MCID definitionMCID for 6MWD in DMD Reference

a. Baseline SD (1 r) 28.5 m McDonald 2013;

Wyrwich 1999

b. Baseline SD/3 31.7 m McDonald 2013;

Yost 2005

3

Decline in 6MWD predicts loss of ambulation over 2 years

Percent ambulatory over 2 years

80 60 40 20 0

-30m -30m

-30m

-30m

-30m

-30m

7 Baseline 6MWD > 350 m N=42, N=40, N=36

Excludes n=1, n=3, and n=7 with loss of ambulation by

Age > 7 Baseline 6MWD > 350 m N=43

Includesthose with

Age > 7 Baseline 6MWD < 350 m N=22, N=12, N=11

Excludes n=3, n=13, and n=14 with loss of ambulation by

Age > 7 Baseline 6MWD < 350 m N=25

Includes those with loss of ambulation

Effect of Baseline 6MWD and Loss of Ambulation

(Data Courtesy of Eugenio Mercuri)

Time from baseline

100

48-week 2-year 3-year 75 study design? Follow-up? Follow-up?

aseline 50

b 25 0 from 0 1 yr 1.5 2 yr 2.5 3 yr -25 WD -50

6M -75

in

-100 nge -125 Cha -150 -175

-200

-225

Age > 7 Baseline 6MWD > 350 m N=42,N=40, N=36

Excludes n=1, n=3, and n=7 with loss of ambulation by

1, 2, and 3 yrs respectively

Age > 7 Baseline 6MWD > 350 m N=43

Includesthose with loss of ambulation

Age > 7 Baseline 6MWD < 350 m N=22, N=12, N=11

Excludes n=3, n=13, and n=14 with loss of ambulation by

1, 2, and 3 yrs respectively

Age > 7 Baseline 6MWD < 350 m N=25

Includesthose with loss of ambulation

(Data Courtesy of Professor Eugenio Mercuri)

DMD114673 vs. Natural History

both including andexcluding patients who lost ambulation

=+33 m

=-25 m

DMD114673 Drisapersen 6 mg/kg (n=8)

Baseline 6MWD= 417 m Excludes n=2 with loss of ambulation

, ,

DMD114673 Drisapersen 6 mg/kg (n=10)

Baseline 6MWD = 386 m Including 2 patients with lost ambulation

Age > 7 Baseline 6MWD > 350 m N=42, N=40, N=36

Excludes n=1, n=3, and n=7 with loss of ambulation by

1, 2, and 3 yrs respectively

Age > 7 Baseline6MWD > 350 m N=43

Includes those with loss of ambulation

Natural history data provided by Professor Eugenio Mercuri (Catholic University Policlinico Gemelli, Rome, Italy).Note: Direct comparisons between extension drisapersen from DMD114673 and the natural history data cannot be made. They are presented together for illustrative purposes only.

26

Safety:

Drisapersen was generally well tolerated

Drisapersen clinical trial program: key safety data

Drisapersen is generally well tolerated, with an adverse event (AE) profile consistent with that described previously forthis class of molecule

59 serious AEs reported on drisapersen, most considered unrelated : 15/59 serious AEswere considered related or possibly related to drisapersen

12 of >300 subjects have withdrawn permanentlyfrom treatment owing to AEs

Most commonly reported AEs include injection-site reactions and renalabnormalities (including subclinical proteinuria)

Some cases of (moderate to severe) thrombocytopaenia havebeen reported

AEs of special interest, n (%):

Any AE of special Injection-site

Study Treatment N Renal abnormalities Inflammation Hepatic Thrombocytes interest reactions*

Placebo 18 13 (72%)6 (33%)7 (39%)9 (50%)1 (6%)0

DMD114117

Drisapersen

18 16 (89%)14 (78%)13 (72%)10 (56%)2 (11%)0 6 mg/kg/week

Placebo 16 9 (56%)5 (31%)5 (31%)1 (6%)1 (6%)0

DMD114876

Drisapersen

18 16 (89%)13 (72%)5(28%)5 (28%)1 (6%)0 6 mg/kg/week

Placebo 61 37 (61%)10 (16%)20 (33%)16(26%)0 0

DMD114044

Drisapersen

125 114 (91%)97 (78%)80(64%)33 (26%)7 (6%)0 6 mg/kg/week

*Includes discolouration, erythema, rash, induration;Includes proteinuria, red blood cells in urine; Two subjects in DMD114044 receiving drisapersen 6 mg/kg/week had severe AEs reported by the investigator as drug-related (glomerulonephritis, intracranial venous sinus thrombosis and spinalpain). No severe AEs were reported in the placebo group.

28

Conclusions

Comprehensive clinical trial program(>300 subjects) has been conducted to assess safety and efficacy of drisapersen in DMD, comprising both placebo-controlled and OLE studies The clinical programme efficacy data suggest: benefit with drisapersen versus placebo in boys with lesssevere disease clinically meaningful benefit after longer duration of drisapersen treatment in boys who are, on average, more severely affected

Totality of the data warrant a filing for Accelerated Approval with the FDA and Conditional approval with the EMA

29

Conclusions

Prosensa and now Biomarin areleveraging what they have learned from natural history studies to design confirmatory studies of drisapersen in DMD characterized by

a) choice of the correct dose based on phase 2 studies; b) longer trial duration based on our experience with the multiple extension studies; and c) an emerging natural history comparisoncohort

Drisapersen was generally well tolerated

AEs include sub-clinical proteinuria, local injection-site reactions and thrombocytopenia Addressed by phamacovigilenceand emerging dosing regimes (Intermittent dosing after loading; IV route of administration) Overall safety profile is consistent with that described previously for this class of molecule

30

Glenn Pierce, M.D.

Former Chief Medical Officer,Senior Vice President of Biogen Idec Hemophilia Therapeutic Area

BIOMARIN ANALYST AND INVESTOR DAY 2014

BioMarin ANALYST DAY 2014 Whats New Today? Drisapersen Potential near-term registration opportunity in large market with unmet need Seamless fit within BioMarinsclinical, regulatory and commercial expertise BMN 111 for Achondroplasia First 3 cohorts enrolled Leveraging the natural history study to inform our understanding of our patients and analytic approaches BMN 190 for CLN2, Late InfantileForm of Batten Disease Enrollment completion imminent Current study exhibiting excellent safety profile to date BMN 270 for Hemophilia A Launching Phase 1 study 1H15 on track two years after licensing Expandedpre-clinical proof of concept to include real world (clotting) assays

Whats New Today? PEG PAL for PKU Phe lowering in patients from enriched study at 26 weeks is robust at ~60% Enhanced strategies in place to facilitate commercialization Pivotal data now expected 1Q16 BMN 701 for Late-onset Pompe Disease Improved manufacturing process with better purity, potency and COGS Modifying program to leverage to be commercialized process Proof ofconcept in 20 patients of switch study; enrollment 1Q15/results 4Q15 Pivotal study resumes 4Q15 Talazoparib for mBRCA BC Enrolling more trials, including talazoparib in combination, neoadjuvant, etc. BMN 250 for MPS IIIB (Sanfilippo Bsyndrome) Launching natural history study with patient accrual expected 2015 Phase 1 study in MPS IIIB patients

Financial Outlook Detailed financial guidance to be provided in February 2015 with year-end release Consistent with past practice Will allow for Prosensa budgetexercise 2015 Revenues: Generally comfortable with consensus estimates 2015 Operating Expenses: Consensus R&D and SG&A expenses without Prosensa approximately 5% to 10% too low GAAP Operating expenses include substantialnon cash expenses 2014 non GAAP loss guidance: $50 million to $65 million 2015 non GAAP loss to be 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