Ultra-Rare to Rare: Two Execs Talk About Alexion's Shift

biospace | May 09, 2019

Alexion Pharmaceuticals is a company focused on ultra-rare diseases, although it is working on a shift into rare diseases. This may sound like something of a minor shift, but it represents developing drugs for more diseases with larger patient populations. The company’s success has been built primarily on its Soliris (eculizumab), a complement inhibitor. The drug has been approved for paroxysmal nocturnal hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS) and for generalized Myasthenia Gravis (gMG). Soliris, as well as the company’s next-generation complement inhibitor, Ultomiris, underline the company’s expertise in complement-related diseases. The complement system is part of the immune system made up of about 30 different proteins. As such it plays a role in numerous diseases at some level, and alone in several diseases, including PNH, aHUS and others.

Spotlight

With complex development processes and stringent regulatory requirements, the path to developing a combination product can seem uncertain and fraught with risk. All elements of the delivery system must comply with the highest quality and regulatory standards.

Spotlight

With complex development processes and stringent regulatory requirements, the path to developing a combination product can seem uncertain and fraught with risk. All elements of the delivery system must comply with the highest quality and regulatory standards.

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PHARMACY MARKET

Exscientia Highlights "The Future of AI-enabled Drug Discovery" at SLAS Europe

Businesswire | May 23, 2023

Exscientia plc announced two abstracts for oral and poster presentation, as well as the closing keynote to be delivered at the Society for Laboratory Automation and Screening (SLAS) Europe Conference and Exhibition being held May 22-26, 2023, in Brussels, Belgium. These presentations highlight Exscientia’s recent advancements in technology, including the development of computer-aided biology (CAB) approaches, encompassing design of experiments (DoE) and automation for biochemical assay optimisation. The SLAS Conference is Europe’s premiere venue for industry, government and academic researchers, developers and providers of laboratory automation technology innovation. “At Exscientia, our mission is to encode and automate drug discovery. We believe that within this decade the design of all new drug candidates will be augmented with AI. I am delighted to showcase at SLAS Europe how our recent technology advances enable and drive our projects,” said Dr Garry Pairaudeau, Chief Technology Officer at Exscientia. Integrating laboratory automation of synthesis and screening with its AI-driven computational design platform is the logical next step for the company. “This will facilitate the next transformation in speed and productivity, as we aim to bring better medicines to patients faster. We’re looking forward to opening our purpose-built automation studio in Oxfordshire, U.K., later in 2023.” The company’s presentation at SLAS will demonstrate how far along Exscientia is in automating the drug discovery processes. “We believe automation has the potential to radically improve the timely creation of high-quality drug candidates, so that future patients may benefit from innovative science faster,” Pairaudeau stated. Closing Keynote Presentation Title: The future of AI-enabled drug discovery Room/Location: Plenary Theater Date/Time: Thursday, May 25 / 16:30 – 17:30 PM CET This presentation will highlight Exscientia’s distinct approach to molecular design, using an end-to-end patient centric, AI-enabled platform, which has led the way in the precision design of AI-generated molecules resulting in the first AI-designed compounds to enter clinical trials. To reach Exscientia's goal of fully encoding and automating drug discovery, the company is assembling an automated physical laboratory alongside its suite of sophisticated computational design tools, methods and scalable engineering platform. This presentation will highlight Exscientia's current progress in the automation field and the critical steps needed in the implementation of automated, digital laboratories. Abstracts Accepted for Presentation Oral Presentation Title: Computer-aided biology: DoE solutions Track: Frontiers in Technology Room/Location: 231 Date/Time: Thursday, May 25 / 15:30 – 16:00 PM CET Exscientia is building an automated ecosystem of chemistry, biology and compound management, including developing a pipeline of fully automated biochemical assays from assay development onwards. This presentation highlights how CAB has been leveraged to achieve this pipeline, combining DoE (a statistical methodology) with automation to rapidly determine optimum conditions to run assays efficiently through analysis of multi-factorial experimental results. Utilising CAB also allows Exscientia to increase capacity and efficiency across FTEs, time and reagents required to run assays, increase assay robustness and investigate areas of biology not normally studied. Poster Title: Improving assay optimisation and pharmacological sensitivity of a nuclease by employing a computer-aided biology approach Session/Track: Session A; Frontiers in Technology Abstract Number: 1034-A Date/Time: Wednesday, May 24 / 13:30 – 14:30 PM Exscientia researchers demonstrated the practical use of CAB to simultaneously improve an assay’s performance and reduce the reagent requirement, cost and labour for assay development. This presentation highlights the application of CAB to optimise the buffer of a nuclease assay, resulting in a 15-fold enhanced assay signal in low salt buffer conditions, alongside over a three-fold decrease in protein requirement​ and reduced assay development cycle times. The low salt requirement is a previously unforeseen result from CAB use which would have not been predicted from the literature. This highlights the potential for technology to improve assay performance in ways a human alone would not have been able to predict. About Exscientia Exscientia is an AI-driven precision medicine company committed to discovering, designing and developing the best possible drugs in the fastest and most effective manner. Exscientia developed the first-ever functional precision oncology platform to successfully guide treatment selection and improve patient outcomes in a prospective interventional clinical study, as well as to progress AI-designed small molecules into the clinical setting. Our internal pipeline is focused on leveraging our precision medicine platform in oncology, while our partnered pipeline broadens our approach to other therapeutic areas. By pioneering a new approach to medicine creation, we believe the best ideas of science can rapidly become the best medicines for patients.

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BUSINESS INSIGHTS, PHARMACY MARKET

Endo Launches Authorized Generic Version of Noxafil® (posaconazole) Oral Suspension

Prnewswire | April 04, 2023

Endo International plc announced that one of its operating companies, Par Pharmaceutical, Inc., has begun shipping the authorized generic (AG) version of Merck's Noxafil® (posaconazole) 200 mg/5 mL oral suspension in the U.S. "Our partnership with Merck underscores our reputation as an industry-leading supplier," said Scott Sims, Senior Vice President and General Manager, Injectable Solutions & Generics at Endo. "This newly available Noxafil® AG dosage form is the latest addition to our generics portfolio—providing patient access to high-quality, lower-cost treatment options." Endo subsidiary Endo Ventures Limited entered into an exclusive U.S. supply and distribution agreement with a subsidiary of Merck & Co., Inc. of Rahway, N.J. allowing Par to launch and distribute an authorized generic version of the product. The agreement also included the authorized generic version of Noxafil® delayed-release tablets, 100 mg, which launched in 2019. Noxafil® is a registered trademark of Merck Sharp & Dohme LLC. About Endo International plc Endo is a specialty pharmaceutical company committed to helping everyone we serve live their best life through the delivery of quality, life-enhancing therapies. Our decades of proven success come from passionate team members around the globe collaborating to bring treatments forward. Together, we boldly transform insights into treatments benefiting those who need them, when they need them.

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BUSINESS INSIGHTS, PHARMA TECH

CONTINUUS Pharmaceuticals Adopts Digital Validation

CONTINUUS Pharmaceuticals | March 15, 2023

On March 14, 2023, ValGenesis, Inc., a leading enterprise validation lifecycle management solutions provider, announced that CONTINUUS Pharmaceuticals has successfully implemented the ValGenesis Validation Lifecycle Management System (VLMS) for efficient management of validation processes across the company in a 100% digital format. CONTINUUS Pharmaceuticals is a rapidly growing small business specializing in end-to-end integrated continuous manufacturing (ICM) of pharmaceuticals. As a spin-out company resulting from a long-term collaboration between MIT and Novartis, CONTINUUS Pharmaceuticals is committed to enhancing the accessibility and affordability of pharmaceutical products worldwide by exploring advanced manufacturing paradigms that expedite the development of raw materials and APIs to final dosage forms. Due to the immense validation requirements involved in the production of different drug products, CONTINUUS Pharmaceuticals needed an electronic solution that determines the disposition of their assets while switching amongst product lines. By leveraging the ValGenesis VLMS, CONTINUUS Pharmaceuticals has successfully implemented facilities, equipment validation, computer system and utility validation. The ValGenesis VLMS is the system of record for CONTINUUS Pharmaceuticals' engineering team and will be expanded to other teams in the future. The highly automated, real-time, and compliant system provided by ValGenesis minimizes the potential for Good Manufacturing Practice (GMP) non-compliance while accelerating time to market. About CONTINUUS Pharmaceuticals CONTINUUS Pharmaceuticals is an end-to-end integrated continuous manufacturing (ICM) company specializing in pharmaceutical production. The company is committed to enhancing the accessibility and affordability of pharmaceutical products worldwide by exploring advanced manufacturing paradigms that expedite the development of APIs and raw materials to final dosage forms. It leverages a novel continuous manufacturing platform called Integrated Continuous Manufacturing (ICM), initially developed through a $65 million joint research project between Novartis and MIT. With ICM, it aims to transform and streamline the supply chain of pharmaceuticals by delivering high-quality, lower-cost drugs "on-demand."

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