Can a blood pressure drug protect the brain from Parkinson's?

medicalnewstoday | April 23, 2019

A prescription drug already in use for the treatment of high blood pressure could be effective against conditions such as Parkinson's, Alzheimer's, and Huntington's, in which toxic proteins build up in brain cells. Scientists at the University of Cambridge in the United Kingdom and the Guangzhou Institutes of Biomedicine and Health in China suggest that the hypertension drug felodipine could be a promising candidate for "repurposing" as a treatment for neurodegenerative conditions. In experiments with zebrafish and mice, they showed that felodipine can prompt a cellular recycling process called autophagy to clear away toxic proteins in brain cells, or neurons. "Our data suggest," they write in a recent Nature Communications paper, "that felodipine induces autophagy in neurons and enhances removal of a range of disease-causing proteins: mutant huntingtin, mutant [alpha]-synuclein, and tau."

Spotlight

A recent e-symposium presented insights into the latest
research and innovative solutions to help shorten timeto-market, reduce development costs, increase yield,
and assure quality and pharmacopoeia compliance
throughout the drug development process.


Other News
PHARMA TECH

Three iA Employees Honored with Top Women in Health Awards

iA | November 18, 2022

iA, a provider of software-enabled pharmacy fulfillment and automation solutions, celebrated three of its employees being recognized at Drug Store News’ Fourth Annual Top Women in Health, Wellness & Beauty Awards held in Chicago, Illinois. Rachael Clark, Vice President of Commercial Sales, received a Rising Star award. As Vice President of Commercial Sales, Rachael works with large-scale retail chain grocery stores, mainstream pharmacies, and digital pharmacies, as well as independent mom-and-pop operations. Rachael is responsible for a team of account executives that work to promote iA’s technology across North America and globally by developing long-term strategies, while also organizing and maintaining sales operations. Bobbi Jamriska, Senior Vice President, Product Management, received an award for Business Excellence. Bobbi leads the Product Management team at iA that is responsible for the iA portfolio of solutions, where she manages the strategy, development, and go-to-market planning. She has been a pharmacy tech for 12 years, and has led global product management initiatives as well as new product development experiences. Stacey Weber, Senior Director of Customer Operations, received an award for Business Excellence. Stacey oversees the customer support division, where she manages a team of 35 remote technicians who assist customers in using iA’s Rx fulfillment solutions, which include automated prescription filling robots and innovative warehouse management systems. In addition to ensuring customers receive timely and helpful assistance, she also oversees standardization and process improvement for iA’s call center and for the customers that use iA solutions. “We are incredibly proud of our team members. Rachael, Bobbi, and Stacey all represent the best of iA, and we are very lucky to be able to work with them every day.” Tom Utech, President and Chief Operating Officer About iA iA is a leading-edge provider of software-enabled pharmacy fulfillment solutions. With over 30 years in pharmacy fulfillment, iA has invented and developed a suite of software-driven pharmacy automation solutions. From NEXiA software to automated modular hardware and sophisticated counting and collation devices, iA pharmacy automation solutions and technology empower pharmacists. iA can run the prescription fulfillment process from end to end, helping pharmacies manage fulfillment and inventory to help lower costs, improve efficiency and provide comprehensive Rx tracking and real-time support.

Read More

VIEWS AND ANALYSIS

Altamira Therapeutics Provides Business Update and First Half 2022 Financial Results

Altamira Therapeutics Ltd | December 01, 2022

Altamira Therapeutics Ltd. a company dedicated to developing therapeutics that address important unmet medical needs, today provided a business update and reported its first half 2022 financial results. “We continue to make good progress with the transformation of Altamira into an RNA delivery technology company. We are optimistic of reaching an agreement to divest or partner our Bentrio nasal spray for key markets in North America and Europe by year end. Last month, we agreed to divest part or all of our inner ear therapeutics programs to a European family office. Following some delay and under slightly amended terms, we expect that transaction to close in December. Thomas Meyer, Altamira Therapeutics’ founder, Chairman and CEO “Heading towards 2023, we look forward to focusing exclusively on the many emerging opportunities in the fast-growing RNA therapeutics market. We are increasingly well positioned to advance our RNA delivery technology throughout 2023.” As Altamira is going through the final stages of a major corporate transformation, management intends to hold its next investor call upon finalization of its partnering / divestiture projects. On that call, the Company will also provide its outlook for 2023. RNA delivery platform update Altamira continued to make solid progress with the development of its patented, peptide-based platform for RNA delivery to extrahepatic tissues. In recent months, the RNA team led by Chief Development Officer Covadonga Pañeda, Ph.D., and Chief Scientific Officer Samuel Wickline, MD, have advanced various projects, including selection and optimization of siRNA sequences, formulation, process development and manufacturing. Starting with project AM-401 for the treatment of KRAS-driven tumors, the Company added a second project, AM-411 for the treatment of rheumatoid arthritis. AM-411 nanoparticles comprise siRNA targeting NF-kB a key checkpoint in RA inflammation. The Company is developing both AM-401 and AM-411 with the objective of out-licensing the drug products at a later stage. They serve as a “showcase” for the application of Altamira’s RNA delivery technology; the Company’s strategy will be to out-license the technology to pharma and biotech companies for use with their own RNA molecules. In this context, Altamira has been intensifying its efforts to raise awareness about OligoPhore/SemaPhore within science and industry. In recent months, members of Altamira’s leadership team gave oral presentations at multiple international conferences, highlighting the ability to deliver RNA molecules to extrahepatic tissues and achieve efficient and rapid endosomal release inside target cells. Concurrently, further data on RNA delivered with Altamira’s delivery technology has been published by independent research groups in peer-reviewed scientific journals. Altamira anticipates entering into its first partnering agreements in 2023. Bentrio Update Earlier today, Altamira reported that its licensee and distribution partner Nuance Pharma has launched Bentrio nasal spray in Hong Kong to help provide protection against airborne viruses as well as allergens. This will be the first step to distributing Bentrio in the other Nuance-licensed territories which is comprised of mainland China, Macau and South Korea. As part of its strategy to focus exclusively on RNA delivery, Altamira has been in discussions with several well-established OTC consumer health companies for the partnering of Bentrio. Those discussions intensified following the 510(k) clearance of the product by the FDA and have advanced well, including due diligence by interested parties. The Company anticipates entering into a partnering transaction before year end. In the context of those partnering discussions, Altamira suspended preparations for launching the product in the US on its own as well as pausing major marketing initiatives in Europe. This restraint provides the prospective strategic partner for Bentrio with maximum flexibility to fit the product into its business plan. Beginning in early October, the Bentrio nasal spray was relaunched in Europe for allergic rhinitis. Previously, the Company had ceased marketing the product for the indication of viral infection in the EU and Switzerland although Bentrio’s mode of action is the same regardless of whether it provides a barrier against airborne virus or allergen particles. This had been demonstrated in various relevant in vitro assays. However, certain countries and regions require specifically clinical performance data to clear Bentrio for this indication, in particular related to COVID-19. Such data are expected to become available through the COVAMID trial. In September, Altamira announced that it had reached its extended enrollment target of 160 confirmed subjects in its COVAMID clinical investigation to evaluate the safety, tolerability, and efficacy of its Bentrio nasal spray in patients with acute COVID-19. The read-out of top-line data remains on track for the current quarter. The Company plans to seek an expansion of its product label to also include viral infections in those countries requiring supportive clinical data. In September, the Company also announced that its “NASAR” clinical trial in seasonal allergic rhinitis (SAR) resumed enrollment as the new pollen season started in Australia. The NASAR trial is expected to enroll a total of 100 patients suffering from SAR and is designed to compare the safety and efficacy of Bentrio against a (control) saline nasal spray. The primary endpoint will be the comparison of the reflective Total Nasal Symptom Score under treatment with Bentrio against control. The NASAR trial was initiated in the fall of 2021. It was suspended in spring 2022 as the pollen season came to an end before the enrollment target could be met. Interim data from the trial were used in support of the 510(k) clearance of Bentrio by the US FDA. Unless an interim analysis performed upon reaching 50% of the enrollment target to check the validity of the statistical powering assumptions requires a change to the target size of 100 patients, the Company expects to complete enrollment into the NASAR trial by year-end or in early 2023 with a read-out of top-line data in late 1Q-23.

Read More

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.

Read More

BUSINESS INSIGHTS

Illuccix® Available on High Activity Gallium Generator Technology to Meet High Demand

Telix Pharmaceuticals Limited | November 23, 2022

Telix Pharmaceuticals Limited announces Illuccix® with up to 50mCi for radiolabeling is available for use on 100mCi gallium generators, an important development in radioisotope production. High activity generators from Eckert & Ziegler Strahlen- und Medizintechnik AG increase activity from 50mCi to 100mCi and will be integral to expanding patient access to gallium-based radiopharmaceuticals as demand for PSMA-PET imaging in the United States continues to grow. This technology increases the number of daily elutions and activity in each elution, enabling hospitals and radiopharmacies to increase the number of daily doses produced, expand their service area and gain greater production efficiency. "As the only commercially available kit that can take advantage of high activity generators for up to 50mCi radiolabeling, Illuccix gives hospitals, pharmacies, and physicians added confidence that their dose scheduling needs – and the needs of their patients – can be met today and in the future as the potential of 68Ga PSMA-11 is realized in the diagnostic market. We are pleased to continue our collaborative relationships with IRE ELiT and EZAG to continue to make 68Ga PSMA-11 accessible and convenient to use for customers across the United States." Dr. Christian Behrenbruch, Group CEO and Managing Director of Telix Jay Simon, Managing Director of Eckert and Ziegler North America said, "The high activity GalliaPharm® generator may allow for broader availability and expanded service areas. It is about delivering doses when and where they are needed, whether you're a remotely located clinic, a busy metropolitan hospital or a pharmacy. We are pleased to be working with Telix to harness this innovation to maximize the efficiency for our customers and ultimately, their patients." Jean Bonnet, Head of Sales, Strategy and Marketing at IRE Group said, "While current generator technology is sufficient to meet today's demands, the rollout of the higher activity 100mCi generators are future-proofing the ability to meet demand in a market which is growing exponentially. The use of high activity generators has the potential to increase daily elutions and increase the activity in each elution. When radiolabeled up to 50mCi with Illuccix, the result could potentially provide more flexibilitty in meeting the growing demand for 68Ga radiopharmaceuticals while giving customers the benefit of greater dose scheduling flexibility and efficient production." About Telix Pharmaceuticals Limited Telix is a biopharmaceutical company focused on the development and commercialisation of diagnostic and therapeutic radiopharmaceuticals. Telix is headquartered in Melbourne, Australia with international operations in the United States, Europe (Belgium and Switzerland), and Japan. Telix is developing a portfolio of clinical-stage products that aims to address significant unmet medical need in oncology and rare diseases. Telix is listed on the Australian Securities Exchange.

Read More