BioMarin

A leading, global rare disease biotech company focused on delivering medicines for people living with genetically defined conditions.

[BioMarin @BioMarin]

At BioMarin, we are driven by our purpose: to be the biotech leader that translates the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The BioMarin 8 – our core Operating Principles – provide the framework for how we work together every day to fulfill that purpose.

[BioMarin @BioMarin]

Several of our team members were on hand in Cambridge, MA last week as local startups made their case for a @LabCentral Golden Ticket, which provides emerging biotechs with residency at LabCentral and access to its innovative community and resources. Orthrus Therapeutics took home the BioMarin-sponsored Golden Ticket after demonstrating a promising novel approach to help enable long-lasting immune tolerance to chronically administered therapies.

We’re proud to support Lab Central’s Golden Ticket program for the sixth consecutive year to celebrate collaboration in advancing the life sciences, and are excited to see how Orthrus Therapeutics contributes to the future of genetic medicine.

Learn more about the LabCentral Golden Ticket program: https://t.co/y0edbkf2rA

[BioMarin @BioMarin]

Today we announced Q3 2026 results to the investor community, including a 32% year-over-year increase in total revenues compared to the same quarter last year. This was driven by strong underlying demand for our medicines around the world.

Read more in our press release: https://t.co/3Ofx0B024a

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[BioMarin @BioMarin]

We’re looking forward to sharing an update with investors on our Q3 results Monday, Nov. 2 at 4:30 p.m. ET / 1:30 p.m. PT. Join our earnings call and webcast here: https://t.co/CVtq4fO1N2

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[BioMarin @BioMarin]

Today we announced we have entered into an exclusive worldwide license agreement with GC Biopharma and Hanmi Pharm to develop and commercialize a potential next-generation enzyme replacement therapy for Fabry disease. The investigational long-acting medicine is currently being studied in a Phase 1/2 clinical trial and has the potential to be the first subcutaneous enzyme replacement therapy for Fabry disease.

Read more in our press release: https://t.co/cIctAhHMKl

[BioMarin @BioMarin]

For much of his career, Arpit Davé has been fascinated by an idea: Some of the biggest opportunities in biopharma lie not in the science alone, but in how organizations can most effectively learn from the vast amounts of data that science generates.

Long before today's excitement around AI, Arpit saw the potential for technology to help researchers uncover new insights, connect information and make better decisions. The challenge was that the ambition often outpaced the tools available to support it. But in recent years, he says, technology has caught up enough to turn potential into reality.

"My career journey has really been about cracking this complex problem of using technology and data, and now this powerful tool of AI, to allow companies to fulfill their mission, which is to serve patients,” Arpit says. “There has never been a time like the present for using technology to deliver on the promise of science to improve human health – now it’s on us to seize the moment.”

We recently sat down with Arpit, who joined BioMarin earlier this year as Chief Digital Information Officer, to discuss his career journey, what brought him here and how he envisions combining the company’s three decades of scientific expertise with emerging technology to accelerate impact for people with rare genetic conditions.

Read more: https://t.co/CG23I9YgeY

[BioMarin @BioMarin]

Today we announced new data at the American Society for Bone and Mineral Research (@ASBMR) Annual Meeting, highlighting advances in research across multiple skeletal conditions. These results add to a growing body of evidence deepening scientific understanding of skeletal dysplasias and reflect our ongoing commitment to improving outcomes for people living with these conditions.

Learn more about the updates we’re sharing at #ASBMR2026: https://t.co/HjI1lyUR8W

[BioMarin @BioMarin]

We’re thrilled to welcome Cara Bauer to BioMarin as our new Chief People Officer. Cara brings more than 25 years of experience as a leader in human resources across the biopharmaceutical and technology industries. She has a track record of building and scaling organizations through periods of rapid growth and change.

We look forward to the valuable perspective Cara will bring to the role, and thank Amy Wireman, who is retiring after 14 years at BioMarin. Amy has played a tremendous role in shaping the company and we wish her all the best in her retirement.

Read more: https://t.co/ZnSz6f6LBA

[BioMarin @BioMarin]

Today we announced new five-year Phase 3 data in adults with late-onset Pompe disease at the Annual Congress of the @WorldMuscleSoc.

These findings showcase long-term benefits for individuals living with this rare genetic condition, including people who had previously received enzyme replacement therapy and those who had not.

Learn more about the data presented here: https://t.co/R2CRaT7hk8

#WMS2026

[BioMarin @BioMarin]

We’re excited to be partnering with @ProfluentBio, an innovative AI lab for biology that will apply its foundational AI models to generate novel enzyme therapies for BioMarin.

Profluent will design and optimize potential medicines using the largest protein sequence dataset in the world, composed of more than 115 billion unique sequences. Our scientists will screen these protein designs and evaluate the most promising candidates to advance to preclinical models, accelerating our early stage research and development in metabolic conditions.

Read more: https://t.co/AK9EvMF6FK

[BioMarin @BioMarin]

Today we provided an update on our clinical program for Noonan syndrome. Read more: https://t.co/hUwD8GmamV

[BioMarin @BioMarin]

We’re excited to welcome Robert Plenge to our Board of Directors. Robert, who currently serves as Executive Vice President, Chief Research Officer and Head of Research at Bristol Myers Squibb, brings more than 25 years of experience across academia and biopharma and will provide an important scientific perspective to our Board as we build on our research capabilities and expand our pipeline.

Read more: https://t.co/Rfioeg9BSZ

[BioMarin @BioMarin]

Today we announced new Phase 3 data in children living with hypochondroplasia, which were published in NEJM Evidence and featured in a late-breaking oral presentation at #ESPE2026.

These findings reflect our continued commitment to advancing research in skeletal conditions and deepening scientific understanding of #hypochondroplasia, a form of skeletal dysplasia that can impact growth, physical function and everyday life for children and families.

Learn more: https://t.co/ABu72OI3mJ

[BioMarin @BioMarin]

We’re excited to introduce the BioMarin Skeletal Dysplasia (BISK) Research Awards, a new opportunity to advance investigator-initiated research focused on improving care and outcomes for people living with #skeletaldysplasia.

This program supports innovative, non-interventional studies that address gaps and barriers in care. Areas of interest include generating real-world data, patient-reported outcomes, quality-of-life evidence, and qualitative research such as surveys and interviews.

Eligible applicants are healthcare professionals, such as physicians, allied health practitioners and research scientists, working within the skeletal dysplasia space in the United States.

Letters of intent are due by Wednesday, Sept. 30.

Learn more and apply: https://t.co/RoOmm3EcIc

#RareDiseaseResearch #ClinicalResearch #MedicalResearch

[BioMarin @BioMarin]

We have completed our previously announced agreement to acquire Alesta Therapeutics. The acquisition will strengthen our skeletal conditions portfolio by adding ALE1, an oral small molecule for the potential treatment of hypophosphatasia currently being studied in a Phase 1/2a clinical trial.

Read more: https://t.co/0f259KVwmm

[BioMarin @BioMarin]

Today we announced a global settlement with Ascendis Pharma A/S, under which Ascendis will pay royalties to BioMarin on Yuviwel sales in the U.S., EU, Brazil and South Korea. Read more: https://t.co/yD1eYK2Afb

[BioMarin @BioMarin]

A global panel of experts has developed the first international consensus recommendations to help enable earlier and more accurate diagnosis of hypochondroplasia.

Published in Nature Reviews Endocrinology, the recommendations provide a comprehensive framework that integrates clinical, radiographic and genetic assessments, aiming to reduce diagnostic uncertainty and enable more timely, consistent care.

We were proud to support this initiative as part of our commitment to improving recognition and diagnosis of hypochondroplasia around the world, and we look forward to working with medical communities to ensure these guidelines are implemented.

Read more: https://t.co/QtJonyX1ES

[BioMarin @BioMarin]

Today we announced that we have entered into a definitive agreement to acquire Alesta Therapeutics to gain Alesta’s lead clinical stage asset, ALE1, an orally active, small molecule for the potential treatment of a rare genetic bone condition called hypophosphatasia (HPP). ALE1 has the potential to be the first oral therapy for HPP and is currently being evaluated in an ongoing Phase 1/2a clinical trial.

Read more: https://t.co/RrKHoxYnf7

[BioMarin @BioMarin]

Cristin Hubbard is still motivated by the same idea that sparked her earliest interest in healthcare and eventually led her to biopharma: getting medicines to the people who need them most.

At BioMarin, Cristin says she feels the impact of that commitment as strongly as ever.

“What I love about working in rare disease is the connection to the communities we serve,” says Cristin, BioMarin’s Chief Commercial Officer. “You feel like every contribution matters so deeply for patients and their families.”

From early work as a medicinal chemist to leading global commercial teams, her career has been shaped by a consistent focus on bringing innovation closer to patients. She’s applying those experiences to help BioMarin leverage its global footprint and capabilities and ultimately reach more people living with rare genetic conditions around the world.

We sat down with Cristin to reflect on her professional journey, her leadership philosophy and how her experience across a variety of roles has prepared her for this moment at BioMarin.

Read more in the full interview: https://t.co/01B24AFYut

[BioMarin @BioMarin]

Today we announced Q2 2026 results, including a 20% increase in total revenues compared to the same quarter last year. This was driven by our acquisition of Amicus Therapeutics and growth in skeletal conditions and phenylketonuria. We also increased full-year guidance for total revenues, revenue for skeletal conditions and non-GAAP diluted earnings per share. Read more in our press release: https://t.co/5w99sccFf8

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