Malignant Hyperthermia

Acquisition of Theravia from Mérieux Equity Partners represents meaningful step forward in Norgine's strategy for sustainable growth, adding a complementary portfolio of rare disease medicines. Transaction further establishes Norgine's position as a partner of choice for commercialising rare and specialty pharmaceuticals in Europe. PARIS, April 15, 2025 /PRNewswire/ -- Norgine and Theravia announced today that they have entered into a definitive agreement under which Norgine will acquire Theravia, an international pharmaceutical company specialising in cutting-edge treatments for patients with rare and debilitating conditions. Recent deals with Fennec Pharma for PEDMARQSI® and X4 Pharma for mavorixafor demonstrate Norgine's strong emphasis on driving growth in rare disease and specialty medicines through acquisitions and in-licensing. Theravia's innovations have resulted in successful launches of life-saving medications which have changed patient outcomes worldwide. Theravia has several products in the rare disease space, including SIKLOS®, for adults and children with sickle cell disease, and ORPHACOL®, a medicine for adults and children who have a genetic disorder that affects bile production by the liver. These products span both the rare haematology and rare hepatology therapeutic areas and are complementary to Norgine's existing rare and specialty portfolio. The development of Theravia in the past few years has been supported by its majority shareholder Mérieux Equity Partners, a healthcare dedicated investment firm based in France. said Janneke van der Kamp, Chief Executive Officer, Norgine. Franck Hamalian, Chief Executive Officer, Theravia, added: With the acquisition of Theravia, Norgine will now have six core products in its rare disease portfolio (PEDMARQSI®, eflornithine, mavorixafor, AGILUS®, SIKLOS® and ORPHACOL®) thereby comprising a franchise of critical scale with the ability to be a key growth driver in the medium-to-long-term. Norgine looks forward to building on this transaction as we continue to strengthen our platform for future acquisitions and in-licensing opportunities to drive growth. The transaction remains subject to obtaining customary regulatory approvals from the competent authorities.

TARRYTOWN, N.Y. and WASHINGTON, March 11, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Society for Science (the Society) announced that Matteo Paz,18, of Pasadena, CA, won the top award of $250,000 in the 2025 Regeneron Science Talent Search, the U.S.’s oldest and most prestigious science and math competition for high school seniors. Now in its 84th year, the competition celebrates and rewards young innovators who are applying their Science, Technology, Engineering and Math (STEM) talent and leadership skills to push the boundaries of discovery and address today’s pressing challenges. Forty finalists, including Matteo, were honored this evening during an award ceremony at the National Building Museum in Washington, D.C, where they were awarded more than $1.8 million in prizes for their groundbreaking research, exceptional problem-solving skills and potential to shape the future of STEM. Matteo Paz, 18, of Pasadena, CA, won first place and $250,000 for designing machine-learning algorithms to efficiently comb through 200 billion entries of raw NEOWISE infrared full-sky data. By analyzing tiny changes in infrared radiation, the AI sorted the objects into 10 classes. He found 1.5 million new potential objects. Second place and $175,000 went to Ava Grace Cummings, 18, of Smithfield, NC, for creating a fruit fly model of STAC3 disorder, or Native American myopathy (a rare genetic muscle disease). She found that the common nettle herb, alone or combined with the experimental drug Tirasemtiv, improved movement in both adult flies and larvae. Third place and $150,000 went to Owen Jianwen Zhang, 18, of Bellevue, WA, for solving a long-standing math problem about objects called 3-uniform hypergraphs. He proved a maximum value for how many 3-uniform hypergraphs can have similar structures but differing connections. Owen's results have applications in computer science. "Congratulations to the winners of this year's Regeneron Science Talent Search," said Maya Ajmera, President and CEO, Society for Science and Executive Publisher, Science News. "The remarkable creativity and dedication of these students bring renewed hope for our future. Driven by their ingenuity, these young scientists are developing groundbreaking solutions that have the potential to transform our world and propel society forward." The Regeneron Science Talent Search provides a national platform for high school seniors to showcase original, innovative STEM research that proposes novel solutions to real-world issues. Finalists are evaluated for their scientific rigor, originality, critical thinking, leadership potential and commitment to drive meaningful impact across crucial STEM fields. “The Science Talent Search changed my life. At my high school, STS winners were treated like star athletes, and I never imagined I would belong in such an amazing group of kids who were operating at a whole different level than I had ever seen,” said George D. Yancopoulos, co-Founder, Board co-Chair, President and Chief Scientific Officer of Regeneron and a 1976 Science Talent Search finalist and top winner. “The experience of competing in STS and being named a top winner gave me the confidence to devote my life to science. So, congratulations to this year’s finalists and winners, you are America’s best and brightest. I hope this moment inspires you to push boundaries, challenge assumptions and use your brilliance to change the world.” Other top honors from the competition include: Fourth Place: Logan Lee, 18, of Honolulu, HI received a $100,000 award for helping sterile male mosquitoes survive in the wild. These males are important in mosquito control. Logan improved their survival by giving them a transplant of wild mosquito bacteria. His transplant helped the sterile mosquitoes grow faster and survive better in the wild. Fifth Place: Rivka Lipkovitz, 18, of San Francisco, CA received a $90,000 award for using statistical modeling to study U.S. voter ID laws. She found that presidential election turnout dropped by 2.4% in states that passed strict laws after 2008. Turnout for midterm elections increased. Knowing how laws affect turnout can help shape future policies. Sixth Place: Melody Heeju Hong, 17, of Wantagh, NY received a $80,000 award for developing a powerful, flexible statistical model for mapping sites called trans-methylation quantitative trait loci (trans-mQTL) within the human genome. These sites are key to understanding the interplay between genes and environment in disease and aging. Seventh Place: Kevin Shen, 18, of Olympia, WA received a $70,000 award for building a custom flight computer to control a 3D-printed airplane with oblique wings. These aircraft can be more fuel-efficient but are hard to control. His oblique-wing aircraft and flight computer improved flight efficiency by 9.2%. Eighth Place: Minghao Zou, 18, of Santa Clara, CA received a $60,000 award for simulating protons to probe environments that produce subatomic particles called neutrinos. He created an algorithm mimicking extreme astrophysical conditions, such as electromagnetic and gravitational forces and interactions with nearby particles. He verified it using known cases of particle motion. Ninth Place: Thanush Patlolla, 17, of Cary, NC received a $50,000 award for approximating the density of electrons using a finite nuclear model. Using a mathematical strategy called a density function, he created a model to map electrons in a nuclear simulation. The map increased the accuracy of energy distribution predictions by 0.6%. Tenth Place: Ray Zhang, 17, of Chantilly, VA received a $40,000 award for studying how to better treat drug-resistant Fusarium fungal infections. Ray studied how the fungus builds sticky communities of cells that resist drug treatment. He also found that using a combination of drugs better controlled the fungus. Akilan Sankaran, 17, of Albuquerque, NM was named the Seaborg Award winner and selected to speak on behalf of the Regeneron Science Talent Search Class of 2025. The 40 finalists chose Akilan as the student who best exemplifies their class and the legacy of nuclear chemist Glenn T. Seaborg, who won the Nobel Prize for Chemistry in 1951 and served on the Society’s Board of Trustees for 30 years. All other finalists received $25,000. All 40 finalists join a distinguished group of Science Talent Search alumni, many of whom have gone on to achieve world-changing careers in STEM, with some earning esteemed honors, including the Nobel Prize, National Medal of Science, and MacArthur Fellowship. In total, Regeneron awarded $3.1 million in prizes, including $2,000 to each top scholar and their school. Learn more about Regeneron Science Talent Search at https://www.societyforscience.org/regeneron-sts/ and learn about all their research projects at our Virtual Public Showcase. For media resources, visit https://www.societyforscience.org/2025-regeneron-science-talent-search-media-kit About Society for Science Society for Science is a champion for science, dedicated to promoting the understanding and appreciation of science and the vital role it plays in human advancement. Established in 1921, Society for Science is best known for its award-winning journalism through Science News and Science News Explores, its world-class science research competitions for students, including the Regeneron Science Talent Search, the Regeneron International Science and Engineering Fair and the Thermo Fisher Scientific Junior Innovators Challenge, and its outreach and equity programming that seeks to ensure that all students have an opportunity to pursue a career in STEM. A 501(c)(3) membership organization, Society for Science is committed to inform, educate and inspire. Learn more at www.societyforscience.org and follow us on Facebook, Twitter, Instagram and Snapchat (Society4Science). About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, hematologic conditions, infectious diseases and rare diseases. Regeneron believes that operating as a good corporate citizen is crucial to delivering on our mission. We approach corporate responsibility with three goals in mind: to improve the lives of people with serious disease, to foster a culture of integrity and excellence and to build sustainable communities. Regeneron is proud to be included on the Dow Jones Sustainability World Index and the Civic 50 list of the most “community-minded” companies in the U.S. Throughout the year, Regeneron empowers and supports employees to give back through our volunteering, pro-bono and matching gift programs. Our most significant philanthropic commitments are in the area of science education, including the Regeneron Science Talent Search and the Regeneron International Science and Engineering Fair (ISEF). For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X. Media Contacts Joseph Brown, Regeneron 386-283-1323, joseph.brown2@regeneron.com Gayle Kansagor, Society for Science 703-489-1131, gkansagor@societyforscience.org Source: Regeneron Pharmaceuticals, Inc.

AMSTERDAM, March 26, 2024 /PRNewswire/ -- Norgine B.V. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of NPJ5008 (dantrolene sodium hemiheptahydrate) for the treatment of malignant hyperthermia. The European Commission (EC) will now review the CHMP's recommendation. If approved, the centralized marketing authorization would be valid in all 27 EU member states, as well as in Iceland, Liechtenstein, and Norway and would be the first new treatment in Europe for malignant hyperthermia in over forty years. Submissions to the UK MHRA and Swiss Medic are ongoing. Dr David Gillen, Chief Medical Officer of Norgine, added: "We are delighted to have received a positive CHMP opinion for NPJ5008. Malignant hyperthermia (MH) is thankfully a rare medical emergency following anaesthesia. Nevertheless, it is clear that effective management of MH is critically important to improve clinical outcomes. NPJ5008 will allow faster administration compared to the existing dantrolene formulation and therefore, we believe, better outcomes for all European patients with MH." For further information please contact: [email protected] Follow us @norgine Notes to Editors: About Malignant Hyperthermia Malignant hyperthermia (MH) is a rare life-threatening pharmacogenetic disorder of skeletal muscles that presents as an exaggerated hypermetabolic response to volatile anaesthetic agents and depolarizing muscle relaxants.[1] About NPJ5008 NPJ5008 is a novel formulation of dantrolene that has been developed by Norgine to shorten preparation and administration times versus DANTRIUM IV (dantrolene sodium). Each vial of NPJ5008 contains 120mg of dantrolene and is reconstituted in 20 mL of water for injections for intravenous administration.[2] About Norgine Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access. Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021. Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world. NORGINE and the sail logo are trademarks of the Norgine group of companies. References: Kaur et al. Mo Med. 2019;116(2):154–159. Ng Kwet Shing RH, et al. Eur J Anaesthesiol. 2024;41:1–10. Logo: