BioMarin JPM 2025.pdf

1、BioMarin Pharmaceutical Inc.Alexander Hardy,President and Chief Executive OfficerJanuary 13,202543rd AnnualJ.P.Morgan Healthcare Conference2Forward Looking StatementsThis presentation and the associated conference call and webcast contain forward-looking statements about the business prospects of Bi

2、oMarin Pharmaceutical Inc.(BioMarin),including,without limitation,statements about:future financial performance,including the expectations of Total Revenues,Non-GAAP Operating Margin percentage,Operating Cash Flow and Revenue Compound Annual Growth Rate(CAGR)for future periods and the underlying ass

3、umptions;BioMarins plans and expectations regarding innovation and growth,including plans regarding business development and clinical-stage external innovation,as well as the anticipated benefits of such plans;ability of BioMarins approved products,including VOXZOGO and BioMarins enzyme therapies,to

4、 drive long-term revenue growth;the clinical development and commercialization of BioMarins product candidates and commercial products,including plans and expectations regarding(i)the ability to expand BioMarins leadership in achondroplasia with VOXZOGO and leverage VOXZOGO in other skeletal conditi

5、ons,including hypochondroplasia,idiopathic short stature,Noonan Syndrome,Turner Syndrome and SHOX deficiency;(ii)development of BMN 333 for the treatment of achondroplasia and hypochondroplasia,(iii)expansion of PALYNZIQ for the treatment of adolescents with phenylketonuria(PKU),(iv)development of B

6、MN 390 for the treatment of PKU,(v)development of BMN 351 for the treatment of Duchenne Muscular Dystrophy,(vi)development of BMN 349 for the treatment of alpha-1 antitrypsin deficiency,and(vii)development of BMN 370 for the treatment of von Willebrand disease;the expected benefits and availability