Bayer chooses UK for its AI-focused R&D hub

pharmaphorum | October 10, 2019

Bayer has continued its policy of setting up R&D hubs to get closer to innovative companies with a UK site that will focus on applying artificial intelligence to drug discovery and disease diagnosis. The cluster – based at Reading’s Green Park – is the seventh in the German company’s LifeHub network, located at R&D hot spots around the globe, and is further evidence of the UK’s leading position in AI technology. According to the Tech Nation 2019 report, the UK is today the world’s third biggest market for AI investment after the US and China with funding rising six-fold since 2014. Bayer already has one company getting ready to set up a unit at LifeHub UK. Sensyne Health, which started working with the German group earlier this year, will base a project focusing on applying AI to automate image evaluation in radiology at the site. The project will analyse research from three million anonymised, ethically sourced NHS patient records and data provided through Sensyne’s partnerships with NHS trusts, with the NHS remaining the controller of all patient data.

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In today’s hypercompetitive pharmaceutical and life sciences (PLS) industry, strategic partnerships are more critical than ever. A smaller life sciences company may have
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BUSINESS INSIGHTS, VIEWS AND ANALYSIS

Mirion Technologies to Divest Biodex Physical Medicine

Biodex Medical Systems | November 30, 2022

Mirion Technologies, Inc. announced that it has entered into a definitive agreement to sell the physical medicine assets of Biodex Medical Systems, Inc. to Salona Global Medical Device Corporation for $5 million in cash at closing and up to an additional $3 million in deferred cash payments. The deferred cash payments are contingent on the performance of the assets during the 12-month period following closing. The assets generated approximately $18 million in revenue with estimated gross margins of 30% during the most recent trailing twelve-month period. “Divesting the physical medicine assets of Biodex enables Mirion Medical to focus on our strategic priorities and better aligns the group with Mirion’s mission of harnessing our unrivaled knowledge of ionizing radiation for the greater good of humanity. We are pleased to see the physical medicine business in the hands of an owner whose core mission and strategy is better aligned with investing in the future of the product line and finding valued employees the right home for their skills and contributions,” Mike Rossi, President of Mirion Medical About Mirion Mirion Technologies is a leading provider of detection, measurement, analysis and monitoring solutions to the nuclear, defense, medical and research end markets. The organization aims to harness its unrivaled knowledge of ionizing radiation for the greater good of humanity. Many of the company's end markets are characterized by the need to meet rigorous regulatory standards, design qualifications and operating requirements. Headquartered in Atlanta (GA – USA), Mirion employs around 2,800 people and operates in 13 countries. About Biodex Medical Systems Biodex Medical Systems, Inc. is part of Mirion Medical, Inc., a group of healthcare-focused brands within Mirion Technologies. Mirion Technologies, Inc. uses science and technology to drive treatment innovation across physical medicine, nuclear medicine and medical imaging categories. With a history of manufacturing and engineering excellence that spans more than 60 years, the Biodex™ mission is to provide innovative solutions and customer-driven support to medical facilities and wellness centers around the globe.

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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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PHARMACY MARKET, PHARMA TECH

MTTI Obtains FDA Allowance of Investigational New Drug (IND) for Hürthle Cell Thyroid Cancer

Molecular Targeting Technologies, Inc | December 09, 2022

Molecular Targeting Technologies, Inc. a clinical stage radiopharmaceutical therapy company focused on therapies for rare diseases, announced today the allowance of an Investigational New Drug application by the U.S. Food and Drug Administration. It is now waiting for Institutional Review Board approval. Once approved by IRB, it will enable a Phase I/II clinical study of the Safety, Dosimetry and Efficacy of EBTATE in adult patients with metastatic, radioactive iodine non-responsive Hürthle cell thyroid cancer.” EBTATE is a new generation of peptide receptor radiotherapeutic drug that has demonstrated potential clinical superiority over standard of care. It selectively binds to somatostatin receptor 2 on neuroendocrine and other tumors, which are then killed by the radionuclide. EvaThera platform products were designed to bind to serum albumin, due to the Evans blue moiety, extending in vivo residence time, enabling lower, less frequent dosing of the radiopharmaceutical and reducing risk of renal injury vs. the current standard of care. Dr. Joanna Klubo-Gwiezdzinska, MD, Ph.D, MHSc, acting section chief of Thyroid Tumors and Functional Thyroid Disorders in the National Institute of Diabetes and Digestive and Kidney Diseases, part of the National Institutes of Health (NIH) and study author commented: “Hürthle cell thyroid cancer, which currently lacks effective treatment options, is characterized by a particularly high expression of SSTR2*. With this phase I/II clinical trial, we hope to implement individualized dosimetry-based dosing for each patient with Hürthle cell thyroid cancer that spread outside of the thyroid gland and analyze how much of an active agent is being accumulated in the tumor tissue to establish a threshold associated with the best efficacy and safety.” “The clearance of our IND is an important milestone for MTTI. Having solidified our clinical trial preparedness and manufacturing readiness, we are well-positioned to advance EBTATE to target Hürthle cell thyroid cancer.” Dr. Chris Pak, President & CEO of MTTI About Molecular Targeting Technologies, Inc. (MTTI) Molecular Targeting Technologies, Inc., is a privately held, rapidly growing, well financed, clinical-stage biotech company developing next-generation targeted radiotherapeutics and diagnostics for rare cancers. Evathera platform technology product has shown application for glioblastoma multiforme (GBM) cancer patients overexpressing integrin. MTTI is committed to building value by acquiring and translating innovative imaging, radiopharmaceutical and theranostics assets to improve human health, reduce healthcare costs and reward stakeholders. MTTI expects to be orchestrating multiple clinical trials in 2023. The content in this release is the sole responsibility of the authors and does not necessarily represent the official views or imply endorsement of the National Institutes of Health.

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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.

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