BioMarin Pharmaceutical, Inc.

SAN RAFAEL, Calif., Jan. 13, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the company has initiated a legal action against Ascendis Pharma A/S for infringement of European patent EP 3 175 863 B1 at the Unified Patent Court (UPC) in Munich, Germany. The patent covers long-acting variants of C-Type Natriuretic Peptide (CNP). The legal action, which would result in a decision in the next 12-15 months, is based on BioMarin's belief that Ascendis' TransCon CNP investigational product and its development program in Germany and elsewhere in Europe infringe BioMarin's patent. The BioMarin patent was confirmed by the European Patent Office's Opposition Division in 2024. The UPC, which formally started in June of 2023, has jurisdiction over European patents for 18 European member states that have ratified the Unitary Patents (UPs), including: Austria, Belgium, Bulgaria, Denmark, Estonia, Finland, France, Germany, Italy, Latvia, Lithuania, Luxembourg, Malta, Netherlands, Portugal, Romania, Slovenia and Sweden. "Intellectual property provides the foundation on which advances in the biotechnology industry are built, particularly for rare genetic conditions," said Alexander Hardy, president and chief executive officer of BioMarin. "We are committed to protecting the important inventions our scientists make for patients around the world, including the more than 15 years of scientific research that supported VOXZOGO® and BMN 333, our long-acting CNP." About BioMarin BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin pursues treatments that offer new possibilities for patients and families around the world navigating rare or difficult-to-treat genetic conditions. To learn more, please visit www.biomarin.com. Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the legal action BioMarin initiated against Ascendis Pharma A/S for infringement of one of BioMarin's European patents, including BioMarin's expectations regarding the timing and resolution of such legal action. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of the legal action BioMarin initiated against Ascendis Pharma A/S; the content and timing of decisions by the Unified Patent Court; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc. Contacts: Investors Media Traci McCarty Andrew Villani BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 455-7558 (628) 269-7393 View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-announces-legal-action-against-ascendis-pharma-as-in-european-unified-patent-court-302349367.html SOURCE BioMarin Pharmaceutical Inc.

Seasoned advisory group including RNA, translation and commercialisation luminaries. LONDON, Jan. 10, 2025 /PRNewswire/ -- Serna Bio, an AI drug discovery company pioneering RNA-targeting small molecules, today announced the members of its Scientific Advisory Board (SAB). Established in 2021, Serna Bio is a preclinical drug discovery company with lead programs in neuro metabolism and obesity. The company is backed by leading investors including but not limited to, Y-combinator, Hummingbird Ventures, and 50Years. "We have established a truly differentiated platform to discover a novel class of small molecules - translational enhancers - and are using this to advance the development of therapies for diseases with no existing treatments, with a focus on genetically validated targets. The strength of our platform and IP has enabled us to surround ourselves with industry veterans with expertise in RNA Biology, preclinical RNA-small molecule research, clinical development and drug commercialisation. I am honoured to work with such an exceptional Scientific Advisory Board. They will be a force amplifier as we progress our lead assets toward the clinic and strengthen our pipeline," said Dr. Khan, MBA PhD. founder and CEO of Serna Bio The Scientific Advisory Board includes leading academics such as Dr. Nahum Sonenberg, who identified and characterized various translation factors involved in translation initiation; Dr. Matthew Hayes, a world leader in obesity drug discovery; and Dr. Jay Schneekloth, an expert in RNA small molecule development. It also includes industry leaders, including Jeff Ajer, the former Chief Commercial Officer at BioMarin. The advisors will draw on their considerable expertise and depth of experience to advise the company and its leadership team on scientific and strategic matters at the intersection of machine learning, RNA biology and drug discovery to help Serna Bio design next-generation small molecules to enhance translation for diseases caused by insufficient protein expression. "Serna Bio has developed a unique approach to biological translation via a large library of next-generation small molecules targeting RNA, with the potential to improve outcomes for patients with a range of diseases otherwise not addressed by present therapies." said Giorgio Massimini, MD, "I am excited to work closely with Dr. Khan and her leadership team as the company builds on its discoveries and pursues its clinical aspirations." "Many patients with rare genetic diseases continue to lack treatment options. With Serna Bio's ability to enhance translation, the company is opening up a new mechanism of action, enabling the development of much-needed medicines for the treatment of previously intractable diseases" said Nick France, MD The full SAB comprises: Jay Schneekloth Senior Investigator at National Cancer Institute (NCI). A pioneer in the identification of selective RNA and DNA binding small molecules. His work has led to the discovery that RNA targeting, druglike compounds are suitable starting points for inhibitor discovery efforts while driving fundamental advances in understanding RNA-small molecule recognition. Giorgio Massimini formerly Vice President Merck KGaA. Over 30 years of experience in leading clinical research & development projects. He contributed to the development of MEK inhibitors, c-Met inhibitors, IDO/TDO inhibitors, MetAP2 inhibitors, DNA repair inhibitors, monoclonal antibodies and other tyrosine kinase inhibitors in the Merck Biopharma pipeline. Nahum Sonenberg James McGill professor of biochemistry at McGill University in Montreal, Quebec, Canada. He is best known for his seminal contributions to our understanding of translation and is notable for the discovery of the mRNA 5' cap-binding protein, eIF4E, the rate-limiting component of the eukaryotic translation apparatus. Nick France Vice President, Global Therapeutic Area Head for Genomic Medicines and Neurology at Alexion. A physician-scientist and rare disease drug development leader. He has worked across the biopharma ecosystem developing diverse therapies for rare disease. Jeff Ajer formerly Executive Vice President and Chief Commercial Officer at BioMarin. Mr. Ajer has more than 25 years of experience driving commercialisation for rare diseases and speciality medicines. He was integral in establishing BioMarin's commercial infrastructure and global footprint and played a leadership role in the launches and growth strategies for BioMarin's commercial brands. Emily Freeman Senior Director, Global Medical Affairs, Patient Focused Implementation Science at Gilead Sciences. She is interested in the development of evidence-based communication tools to facilitate shared decision-making for patients. She has experience at Bristol-Myers Squibb, Pfizer, Eli Lilly, AbbVie, and most recently Sanofi. Matthew Hayes Albert J Stunkard Professor in Psychiatry at the University of Pennsylvania and Vice Chair for Basic and Translational Neuroscience Research in the Department of Psychiatry, Perelman School of Medicine at the University of Pennsylvania. Dr. Hayes is considered a leading expert on the neuroendocrine systems that regulate energy balance. About Serna Bio Serna Bio is an AI-enabled drug discovery company developing the next generation of small molecules to enhance translation. By targeting functional RNA structures, the company's proprietary discovery platform enables selective upregulation of the production of a protein of interest, using orally bio-available small molecules. Serna Bio's approach is a paradigm shift in drug discovery, moving from classical "inhibition" of protein function to protein enhancement. This novel approach unlocks the potential to treat a range of diseases intractable by classical drug discovery. The company's programs are powered by a Generative AI discovery platform trained on proprietary RNA-small molecule datasets, enabling Serna Bio to generate molecules in novel chemical space, outperforming state-of-the-art methods such as NVIDIA MolMIMM for small molecules targeting RNA. For more information about Serna Bio, please visit the company website at serna.bio or follow us on LinkedIn. Logo - SOURCE Serna Bio WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

MUNICH & SAN FRANCISCO--( BUSINESS WIRE )-- CatalYm today announced the appointment of Scott Clarke as Chief Executive Officer. Mr. Clarke brings over two decades of executive leadership experience in driving company growth, developing products, and shaping and executing transactions in the biopharmaceutical industry. He takes the helm as CatalYm enters a new stage of corporate and clinical development, including the initiation of a broad Phase 2b program for its lead candidate, visugromab, in non-squamous non-small-cell lung cancer and additional tumor indications. Based in the US, he will oversee both EU and US operations. “Scott Clarke is a fantastic addition to CatalYm’s leadership team. The company is in a strong position following its oversubscribed $150 million Series D financing and its recent publication in Nature of positive visugromab data. These events provide significant momentum for Scott’s start as CEO,” said Jon Edwards, Interim Chair of CatalYm’s Board of Directors . “We are confident that his experience in oncology and partnering will be a substantial asset as we initiate CatalYm’s broad Phase 2b program with visugromab in hard-to-treat, metastatic solid tumor indications.” “I am impressed by CatalYm’s compelling clinical data demonstrating that visugromab is uniquely positioned as a novel cancer treatment capable of reversing key resistance mechanisms and reinstating an efficient anti-tumor response,” said Scott Clarke, Chief Executive Officer at CatalYm . “I am very excited to join the CatalYm team at such a pivotal stage. I am committed to realizing visugromab’s potential to deliver unprecedented cancer remission depth and durability for patients with very limited therapeutic options.” Scott Clarke joins CatalYm from Ambagon Therapeutics, where he oversaw the company’s $85 million Series A financing and the evolution of its discovery pipeline of molecular glues. Previously, he served as CEO at Tizona Therapeutics, overseeing significant transactions and the development of its first-in-class anti-CD39 antibody for advanced cancers. Earlier in his career, he led oncology partnering at Roche, and was responsible for product development at BioMarin, contributing to multiple approved medicines. Mr. Clarke earned a Bachelor of Science in Chemical Engineering at the University of California, Berkeley, a Master of Science in Biotechnology at Northwestern University, and an MBA at London Business School. About CatalYm CatalYm is developing visugromab, a first-in-class anti-GDF-15 antibody, in solid tumors and other indications. In its first-in-human Phase 1/2a study, visugromab demonstrated durable anti-tumor efficacy with long-lasting objective responses in relapsed and refractory metastatic solid tumor patients in combination with anti-PD-1 treatment. In addition, data from the same study demonstrated that visugromab can significantly counteract the effects of cachexia in these patients. This data was recently published in Nature and presented at the International Conference on Sarcopenia, Cachexia & Wasting Disorders. CatalYm is now advancing visugromab into multiple Phase 2b studies. Founded in 2016 and based in Munich, Germany and San Francisco, USA, CatalYm is backed by leading international investors including Canaan Partners, Bioqube Ventures, Forbion, Omega Funds, Gilde Healthcare, Jeito Capital, Novartis Venture Fund, Vesalius, Brandon Capital, Bayern Kapital, BioGeneration Ventures, and Coparion.