Malignant Hyperthermia

— Companies will co-develop inhibitors of a dark kinase target (NEK1) identified for cancer dependency by Turbine's Simulated Cell™ platform — LONDON and BUDAPEST, Hungary and NEW YORK, April 1, 2024 /PRNewswire/ -- Turbine, a company that is building the world's first predictive simulation of patient biology, and Harmonic Discovery (Harmonic), a therapeutics company building an integrated computational and experimental platform for kinase drug discovery and targeted polypharmacology, have entered into a collaboration to co-develop novel cancer therapies that will inhibit a dark kinase identified for cancer dependency using Turbine's Simulated Cell™ platform. "We believe that the dark kinase NEK1 holds great potential as a drug target for developing cancer therapies addressing important resistance challenges in the clinic," said Daniel Veres, M.D., Ph.D., Chief Scientific Officer and Co-Founder of Turbine. "Coupling Harmonic's deep expertise in multi-specific kinase-targeted drug design with our unique ability to guide drug discovery by running simulated experiments with causal interpretation in a vast biological search space will help realize a more innovative and efficient process than traditional approaches. We are excited for the opportunity to demonstrate the power of our platforms to collectively boost R&D success in the clinic." "Our fully integrated kinase drug discovery platform aims to challenge current drug discovery paradigms by building a machine learning-first infrastructure that integrates multiple aspects of kinase drug discovery," said Rayees Rahman, Ph.D., CEO and Co-Founder of Harmonic Discovery. "Turbine's exciting biological simulation technology is highly complementary to our approach. We are confident that integrating both companies' innovative platforms will yield a radically more effective approach to selecting and advancing kinase-targeted therapies that can substantially improve patient outcomes in oncology." The collaboration provides a framework in which biological and mechanistic insights from Turbine's simulations will directly inform Harmonic's generative chemistry models – with the goal of bringing forward new, differentiated, and effective kinase inhibitor therapies for the patients who need them most. Harmonic will assume responsibilities for computational chemistry and medicinal chemistry activities, while Turbine will be responsible for in silico simulations and wet-lab validation of target biology, including the identification of both synergistic kinase co-targets to NEK1 and patient populations most likely to benefit from therapies. Financial terms of the collaboration are not disclosed. About Turbine Countless resources and time get expended globally to research and advance novel therapies that end in clinical failure and no patient benefit. Imagine a world where it is possible to predict any potential drug's effect on translatable biological models – including those that may not even be available for lab-based testing – while accurately representing patient biology. Turbine is revolutionizing the status quo by working to ensure that we always run the right wet lab experiments. With machine learning that understands the logic by which human cells make decisions, Turbine has built the world's first predictive simulation of patient biology. The Simulated Cell™ platform models the protein signaling that decides cell fate and facilitates in silico experiments at scales that are impossible in the physical world, to empower the biopharma industry by guiding experiments that identify and confirm disease driving mechanisms. Turbine's platform has been validated with leading pharma companies such as AstraZeneca, Ono Pharmaceutical, Cancer Research Horizons, and Bayer. In addition, Turbine has received funding from industry leaders such as MSD (Merck & Co, Inc. Rahway NJ, USA) Global Health Innovation Fund, Accel, and MassMutual Ventures, among others. For more information, visit or follow Turbine on LinkedIn. About Harmonic Discovery Harmonic Discovery (the "Company") is a precision pharmacology company applying its generative chemistry platform to advance next-generation kinase inhibitors. The Company's platform is trained on multiple layers of proprietary data and is integrated with domain expertise to design safer and more efficacious kinase therapeutics. The Company's lead programs are focused on malignant hematology indications, with exploratory discovery efforts in additional therapeutic areas including immuno-oncology, immunology and inflammation. The Company has raised $8 million in Seed Financing to date from Innovation Endeavors, Fifty Years, Y Combinator and other select investment firms. The Company was founded in 2021 and is headquartered at Johnson & Johnson's JLABS ("JLABS@NYC") in New York City, New York. To learn more, visit SOURCE Harmonic Discovery 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:

Europe's Committee for Medicinal Products for Human Use has signed off on Novo Nordisk's one-weekly insulin for diabetes patients, in addition to providing positive opinions on Novartis' Fabhalta and Pfizer's antibiotic combo Emblaveo. Sales are booming for Novo Nordisk’s GLP-1 treatments for diabetes and obesity. But that doesn’t mean the Danish company has moved on from its previous generation of blood sugar modulators. Though it’s been on the market for a century, there are still advances to be made with good old insulin. With a positive recommendation from Europe’s Committee for Medicinal Products for Human Use (CHMP), Novo has come a step closer to bringing its revolutionary Awiqli (once-weekly basal insulin icodec) to the market. The treatment has been tested successfully in both Type 1 and Type 2 diabetes patients. “The world has never seen a weekly insulin,” Novo CEO Lars Fruergaard Jørgensen told Fierce Pharma in August of 2021. Less than three years later, it's close to arriving. The company expects the FDA to sign off on Awiqli next month and to get a nod in Europe by the end of May. By reducing the frequency of dosing, Awiqli could improve convenience, adherence and quality of life. “Awiqli has the potential to become the insulin of choice for people with type 2 diabetes initiating insulin treatment," Martin Holst Lange, Novo’s EVP for development, said in a release. The positive CHMP opinion is based on results from six phase 3 studies of 4,000 patients. In people with Type 2 diabetes, Awiqli achieved superior blood sugar reduction and time spent within a recommended blood sugar range compared with daily basal insulin. In those with Type 2 who have not been treated with insulin, observed rates of hypoglycemia were below one event per year with both Awiqli and the comparators. In people with Type 1 diabetes, Awiqli showed non-inferiority in reducing blood sugar but with a higher estimated rate of hypoglycemia compared with daily insulin. CHMP says "yes" to other new drugs   Also getting a positive opinion from the CHMP was Novartis’ Fabhalta (iptacopan), a treatment for the rare genetic blood disorder paroxysmal nocturnal hemoglobinuria, which was approved by the FDA three months ago. The CHMP further recommended Pfizer’s antibiotic combo regimen Emblaveo (aztreonam-avibactam) to treat a variety of infections. And Outlook Therapeutics of New Jersey notched a CHMP recommendation for Lytenava (bevacizumab) for the treatment of wet age-related macular degeneration (AMD). Nogine B.V. of the Netherlands got a CHMP nod for Agilus (dantrolene sodium, hemiheptahydrate), a treatment for malignant hyperthermia, a life-threatening condition in which skeletal muscles are over-stimulated. BrePco Biopharma of Ireland got a CHMP thumbs up for pediatric use of Neoatricon (dopamine hydrochloride) for hypotension. The CHMP also signed off on three biosimilars, including two osteoporosis treatments from Sandoz—Wyost and Jubbonti, which are copycats of Amgen’s Prolia and Xgeva (denosumab). Celltrion’s biosimilar Omlyclo, which is a knockoff of Novartis’ asthma drug Xolair (omalizumab), also made the grade. Astellas gets thumbs-up for Xtandi expansion   Besides approval recommendations for new medicines, the CHMP signed off on Astellas’ blockbuster Xtandi (enzalutamide) as a monotherapy or in combination with androgen deprivation therapy for the treatment of adult men with high-risk biochemical recurrent (BCR) non-metastatic hormone-sensitive prostate cancer (nmHSPC) who are unsuitable for salvage radiotherapy. The FDA signed off on Xtandi in the indication in November. Astellas hopes for a marketing authorization endorsement in Europe by the end of May.  The CHMP also issued positive opinions that would expand the labels for UCB’s inflammatory treatment Bimzelx, Ipsen’s pancreatic cancer medicine Onivyde, Eli Lilly’s thyroid cancer therapy Retsevmo and Daiichi Sankyo’s cholesterol drugs Nilemdo and Nustendi.