A new method for precision drug delivery: painting

Phys.org | February 27, 2019

If traditional drug delivery were a type of painting, it might be akin to paintball. With good aim, a majority of the paint ends on the bullseye, but it also drips and splashes, carrying streams of paint across the target. If the drug needs to enter the bloodstream and circulate throughout your body for treating disease wherever it may be, this paintball-like delivery system may work. But it won't work for targeted and precise drug delivery. A more acute delivery approach would look more like "painting by numbers," a technique that would allow precise delivery of a certain amount of drugs to an exact location. Researchers at the James McKelvey School of Engineering and the School of Medicine at Washington University in St. Louis are developing the tools necessary for such a drug delivery system, which they call cavitation dose painting.
Their research was published online this week in Scientific Reports. Using focused ultrasound with its contrast agent, microbubbles, to deliver drugs across the blood-brain barrier (FUS-BBBD), the research team, led by Hong Chen, assistant professor of biomedical engineering at McKelvey School of Engineering, an assistant professor of radiation oncology at the School of Medicine, was able to overcome some of the uncertainty of drug delivery.

Spotlight

he recent evolution of Fedegari Group is present on integrated projects where various process machines interact with robotized handling systems for fully automatic high-throughput manufacturing lines. This video provides an overview of our last project: an automated production line with robotized handling solution for pharmaceutical syringes and vials. For more information, contact us: fedegari@fedegari.com


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PHARMACY MARKET, PHARMA TECH

GoodRx Expands Pharmacy Services Platform With $150M vitaCare Acquisition

GoodRx | October 21, 2022

To circumvent overpriced prescription drugs and grow its robust pharmacy services platform, GoodRx finalized its vitaCare acquisition. Based on a transaction that started in the first quarter of 2022, GoodRx agreed to secure vitaCare for $150 million in cash. Based on vitaCare’s financial performance through 2023, an additional $7 million consideration is also possible. A report cited by Globe Newswire showed that the global pharmacy market size has a forecasted value of $1,627.74 billion by 2030. Pharmaceutical platforms, with accessible clinical programs and digital adoptions, have a positive impact on today’s healthcare system, with vitaCare as one of the promising brands. vitaCare, a healthcare service platform, offers cost-effective solutions to patients. More precisely, the platform helps individuals navigate insurance coverage and its optimized benefits to attain lower-priced medications. Rising investments in pharmacy services have sparked interest in vitaCare, including that of GoodRx. The acquisition of vitaCare is in line with GoodRx’s mission, though. Co-CEO Doug Hirsch states that the company seeks to relieve the financial burden that comes with securing medication. Pharma manufacturers have constantly observed barriers to proper patient care, especially for affordable medicine and complicated reimbursement processes. With vitaCare at the helm, new tools are utilized to ensure more patients are linked to relevant savings programs and transparent pricing information. Hirsch hopes that the acquisition will facilitate a smoother brand prescription process overall. Profit-wise, GoodRx predicts the deal will contribute under 1% of its total revenue in 2022, though there is no substantial long-term discern. This acquisition is the latest in GoodRx’s commitment to delivering consumer-focused healthcare services. The company has a history of connecting individuals to accessible, research-based health information. One of the most notable ones is its partnership with Wheel in 2021, which generated better outcomes for patient care. Telemedicine software provider Wheel offers flexible clinical services to a broad patient base. Wheel’s platform equips clinics with the capability of synchronous and asynchronous consults. It utilizes network virtualization to create a seamless experience for healthcare providers and patients. Our post ‘Network Virtualization: The Future of Businesses and Networks’ previously discussed how virtualization empowers a software’s scalability — and this certainly manifests in Wheel’s platform. Providers can choose from a range of services that enhance patient care. One of the services is ePrescription medications, which remotely administers prescriptions. Following the integration of GoodRx’s technology into Wheel’s platform, health providers and patients alike can meet reduced costs of care. As it stands, GoodRx’s coupon price API operates with over 200 billion pricing data points daily. Consumers use these to identify lower-priced medications. With the integration, Wheel-mobilized clinics can offer their patients a GoodRx coupon. GoodRx can also adapt its services to remote healthcare activities through the partnership. After all, GoodRx is essentially a marketplace. Ultimately, GoodRx champions solutions that provide consumers with affordable healthcare access and medication. With vitaCare among its ranks today, the company can focus on promoting better patient adherence to treatment plans.

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BUSINESS INSIGHTS, PHARMACY MARKET

insitro Appoints Philip Tagari, Industry-Leading Scientist and Drug Hunter, as Chief Scientific Officer

insitro | December 02, 2022

insitro, a machine learning-powered drug discovery and development company announced that Philip Tagari has been appointed as chief scientific officer. Tagari joins insitro following a 24-year career at Amgen, where he has led the organization's research platforms for over a decade as vice president, research - therapeutic discovery. “I am deeply excited to partner with Philip on insitro’s journey to build a transformative biology platform for the efficient discovery and development of effective medicines for patients in need. Philip brings unparalleled experience in drug development, including building and utilizing cutting-edge platforms, profound scientific expertise across therapeutic areas and modalities, and most importantly, the humility and courage to transcend the drug discovery status quo and do things differently.” Daphne Koller, Ph.D., founder and CEO of insitro Tagari joins a cross-functional team of experts in biology and technology at insitro, part of a uniquely collaborative culture that spans the boundaries of both fields. “Philip’s inspiring track record of fusing science and technology to build innovative platforms and leading therapeutic programs from bench to approval will guide us in the next step of insitro’s journey, as we deploy our expertise to identify exciting new targets and design therapeutic molecules,” said Koller. “insitro is completely redefining our understanding of disease biology and developing novel therapeutic strategies for a variety of grievous illnesses. Using machine learning on genetics and multi-modal phenotypic data at scale from human cohorts and cellular systems, their cutting edge laboratories are rapidly generating differentiated approaches to currently intractable unmet needs,” Tagari said. “I’m thrilled to partner with Daphne and insitro’s world-class scientists and technologists in achieving a patient-driven vision brought to life in a different kind of drug company.” Tagari joins insitro in early 2023 from Amgen, where he has held a variety of roles since 1998, and served as vice president, research - therapeutic discovery, since 2012. During his time at Amgen, Tagari built and led a global organization of over 600 scientists that delivered numerous experimental and marketed therapies in neurology, inflammation, cardiovascular disease and oncology. Throughout his career, which includes a decade at Merck and research positions at McGill University and Oxford University, Tagari made significant contributions toward multiple first-in-class, disease-modifying, life-changing therapies including Lumakras, Repatha, Singulair, Aimovig, Evenity, Tarlatamab, Acapatamab, Efavaleukin alfa, and AMG 133. With more than 30 years of research experience, he brings expertise across neurobiology, metabolic disease, hematology/oncology, immunology and inflammation, cell and molecular biology, antibody discovery and biologics engineering, medicinal, peptide and oligonucleotide chemistry, analytical chemistry and biochemistry, structural biology, pharmacokinetics, safety pharmacology, laboratory automation and information technologies. Tagari has published or contributed to more than 70 peer-reviewed publications. “I had the privilege of working with Philip for more than 15 years, first at Merck, and thereafter at Amgen, and can say unequivocally that he ranks among the most accomplished, and most innovative, scientific leaders in the industry,” said Roger M. Perlmutter, M.D., Ph.D., currently president, chief executive officer, and chairman of Eikon Therapeutics, Inc., (formerly EVP R&D both at Amgen and at Merck), and is a member of insitro’s Board of Directors. “With Philip as chief scientific officer and partner to Daphne, insitro will be well positioned to ensure that insights from its machine learning-enabled discovery platform contribute to the development of important new medicines that will make a meaningful difference for patients.” About insitro insitro is a data-driven drug discovery and development company using machine learning and data at scale to transform the way that drugs are discovered and developed for patients. insitro is developing predictive machine learning models to discover underlying biologic states based on human cohort data and in-house generated cellular data at scale. These predictive models are being brought to bear on key bottlenecks in pharmaceutical R&D to advance novel targets and patient biomarkers, design therapeutics and inform clinical strategy. insitro is advancing a wholly owned and partnered pipeline of biologic insights and molecules in metabolism and neuroscience. Since formation in mid 2018, insitro has raised over $700 million from top tech, biotech, and crossover investors, and from collaborations with pharmaceutical partners.

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BUSINESS INSIGHTS

NanOlogy Publishes Review Article of Preclinical and Clinical Research Supporting LSAM-DTX

NanOlogy | September 21, 2022

NanOlogy LLC, a clinical-stage interventional oncology drug company, announced that a review article of preclinical and clinical research supporting its investigational drug, large surface area microparticle docetaxel has been published in Drug Delivery and Translational Research. The review article entitled Local administration of large surface area microparticle docetaxel to solid carcinomas induces direct cytotoxicity and immune‑mediated tumoricidal effects: preclinical and clinical studies provides a review of preclinical research that supports tumor-directed LSAM-DTX as well as promising results from the high-risk nonmuscle invasive bladder cancer arm of a clinical trial of local LSAM-DTX in urothelial carcinoma, which were published earlier this year in The Journal of Urology. The articles also follows an earlier review of preclinical and clinical research supporting another NanOlogy investigational drug, large surface area microparticle paclitaxel published in DDTR in 2020. In addition to LSAM-DTX, NanOlogy clinical programs have advanced tumor-directed LSAM-PTX in pancreas, lung, peritoneal, ovarian, prostate, and dermal cancers. The NanOlogy therapeutic platform is based on a proprietary supercritical precipitation technology that converts active ingredients into stable large surface area microparticles (LSAMs) of pure drug optimized for tumor-directed therapy and continuous drug release to maximize drug delivered to the tumor and minimize systemic toxicity. Taxane particles are covered by composition of matter patents issued in the US Canada, Europe, Japan, China, Hong Kong, South Korea, Australia, Indonesia, and Russia valid through June 2036. The composition patents form the foundation of an extensive intellectual property portfolio protecting NanOlogy investigational drugs, formulations, methods, and technology. About NanOlogy NanOlogy, LLC is a private clinical-stage interventional oncology drug company formed in 2015 to improve the treatment of solid tumors based on a proprietary particle engineering technology platform for tumor-directed drug therapy to maximize drug in tumor and minimize systemic toxicity.

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BUSINESS INSIGHTS

BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A

BioMarin Pharmaceutical Inc. | November 24, 2022

BioMarin Pharmaceutical Inc. announced advancements in the U.S. Food and Drug Administration review of the Biologics License Application of ROCTAVIAN™ for adults with severe hemophilia A. The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date. The Company also remains on track to host the scheduled FDA Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility located in Novato, CA. "The review of a BLA is a dynamic process, and we appreciate FDA's ongoing engagement as we work toward delivering a potentially transformative treatment choice to those patients with severe hemophilia A. We look forward to further dialogue with the Agency as it reviews our application." Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin About valoctocogene roxaparvovec The FDA granted Regenerative Medicine Advanced Therapy designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017. In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN™ on August 24, 2022. Robust Clinical Program BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A. Also ongoing is a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors. About Hemophilia A People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate or mild disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life. Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases.

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Spotlight

he recent evolution of Fedegari Group is present on integrated projects where various process machines interact with robotized handling systems for fully automatic high-throughput manufacturing lines. This video provides an overview of our last project: an automated production line with robotized handling solution for pharmaceutical syringes and vials. For more information, contact us: fedegari@fedegari.com

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