First Gene Therapy Clinical Trial to Address Genetic Blindness

PharmaNewsIntelligence | March 11, 2020

Oregon Health & Science University (OHSU) held the first-ever gene therapy clinical trial, BRILLIANCE, to address blindness-causing gene mutation, LCA10, according to a recent press release The clinical trial, sponsored by Allergan plc and Editas Medicine, is one of 14 different clinical trials investigating new genetic treatments for ophthalmic conditions and nearly 50 vision-related clinical trials. The procedure uses gene-editing tool CRISPR to edit human genes, also known as in vivo gene editing. Previously, gene-editing methods have edited genetic material after it was removed from the human body. Being able to edit genes inside the human body is incredibly profound,” said Mark Pennesi, MD, PhD, the Kenneth C. Swan, associate professor of ophthalmology in the OHDU School of Medicine and chief of the OSHU Casey Eye Institute’s Paul H. Casey Ophthalmic Genetics Division.

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The market for the use of biosimilars in the treatment of rheumatoid arthritis is rapidly expanding as many high-profile therapies come off patent. Despite the increasing number of biosimilars, there remain clinical, regulatory and market access challenges for developers.

Spotlight

The market for the use of biosimilars in the treatment of rheumatoid arthritis is rapidly expanding as many high-profile therapies come off patent. Despite the increasing number of biosimilars, there remain clinical, regulatory and market access challenges for developers.

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BUSINESS INSIGHTS

Merck Enters Collaboration and Option to License Agreement with Nerviano Medical Sciences to Develop Next-Generation PARP1 Selective Inhibitor

Merck | September 22, 2022

Merck, a leading science and technology company, announced a collaboration agreement with licensing option with Nerviano Medical Sciences S.r.l. for the next-generation highly selective and brain-penetrant PARP1 inhibitor, NMS-293. NMS-293 has strong potential in combination with a wide variety of DNA-damaging agents, including systemic or targeted chemotherapy or with DNA damage response inhibitors, in numerous tumor types. NMS-293 is in early clinical development for the treatment of patients with BRCA-mutated tumors as a single agent and in combination with temozolomide in recurrent glioblastoma. “Building on the therapeutic impact that PARP inhibitors have had over the last several years, we believe this new PARP1 program, if successful, could fill a significant unmet need for patients unresponsive to existing PARP inhibitors with an improved hematological adverse event profile. The work of NMS to discover and advance this next generation PARP1 selective inhibitor coupled with our deep expertise in developing therapies which modify DNA damage response mechanisms, creates a strong foundation to further develop this investigational therapy for patients.” Victoria Zazulina, M.D., Head of Development Unit Oncology for the Healthcare business of Merck PARP is key in the repair of DNA damage, and PARP inhibitors have been shown to be highly efficacious in the treatment of patients with tumors deficient in homologous recombination repair, such as breast, ovarian, prostate and pancreatic cancers with BRCA-mutations. Under the current agreement, Merck will make early payments (up-front and option exercise fees) of up to $65 million to NMS. Furthermore, NMS will receive payments for the achievement of certain development, regulatory and commercial milestones and tiered royalties on net sales by Merck. Upon exercise of the option, NMS will grant to Merck the exclusive rights to research, develop, manufacture, and commercialize NMS-293. During the option period, NMS and Merck will collaborate on the clinical development of NMS-293, with NMS designing, sponsoring, conducting, and funding global clinical trials. About NMS-293 NMS-293 is an orally available small molecule inhibitor of PARP1 and is currently in early clinical development for the treatment of patients with BRCA mutated tumors as single agent and with recurrent Glioblastoma (GBM), a brain tumor with very high medical need, in combination with temozolomide (TMZ). About Merck Merck, a leading science and technology company, operates across life science, healthcare and electronics. More than 60,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From advancing gene editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2021, Merck generated sales of € 19.7 billion in 66 countries.

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PHARMA TECH

Sirona Biochem Signs International Partnership Agreement with Wanbang Biopharmaceuticals

Sirona Biochem Corp. | November 28, 2022

Sirona Biochem Corp. announces that, subsequent to the LOI, Sirona and Wanbang Biopharmaceuticals have signed an expanded, international partnership agreement to collaborate on licencing Sirona’s SGLT2 inhibitor, TFC-039, as a pharmaceutical treatment in both animal and human health. The agreement adds human health to the partnership as a result of new licencing opportunities currently in due diligence. Wanbang and Sirona initially signed a licensing agreement for TFC-039, whereby Wanbang obtained the rights to develop the compound as a diabetes treatment in China and Sirona retained the global rights. Sirona has since been in discussions with animal health companies to advance TFC-039 as a treatment for diabetes and chronic kidney disease in companion animals. SGLT2 inhibitors provide an opportunity to treat inflicted animals with an oral medication as opposed to the traditional method of daily insulin injections. More recently, Sirona has entered into due diligence with a large pharmaceutical company with a regional interest in developing the compound for human diabetes. Together, Sirona and Wanbang share extensive knowledge and scientific results of TFC-039. Partnering will significantly increase the speed to third-party partnerships and commercialization. The shared data spans over 12 years of research and development, and includes in vitro and in vivo preclinical work, multiple clinical studies, advanced manufacturing process development and the ability to commercially manufacture TFC-039. “We have a long-standing relationship with Wanbang and are excited to combine our two companies’ expertise to license TFC-039. Wanbang has invested millions of dollars into the clinical stage research and development of the manufacturing processes for TFC-039. These pieces of data are critical to large organizations and will greatly increase the opportunities to move forward. The probability of a successful licensing agreement has been made much stronger by leveraging our alliance with Wanbang. We have had a successful year building Sirona’s pipeline, with positive movement on all projects and we’re looking forward to continuing this success with our SGLT2 inhibitor as well as our antiviral and anti-aging projects in 2023.” Dr. Howard Verrico, CEO About Wanbang Biopharmaceuticals and Fosun Pharmaceuticals Wanbang Biopharmaceuticals develops, manufactures, and markets drugs with indications for chronic disease treatment, antibiotics, and other endocrine diseases in China. Founded in 1981, the company is headquartered in Xuzhou, China, and is a subsidiary of Shanghai Fosun Pharmaceutical Group. Fosun is a leader in the pharmaceutical industry and is regarded as one of the top five domestic pharmaceutical companies in China. About Sirona Biochem Corp. Sirona Biochem is a cosmetic ingredient and drug discovery company with a proprietary platform technology. Sirona specializes in stabilizing carbohydrate molecules with the goal of improving efficacy and safety. New compounds are patented for maximum revenue potential. Sirona’s compounds are licensed to leading companies around the world in return for licensing fees, milestone fees and ongoing royalty payments. Sirona’s laboratory, TFChem, is located in France and is the recipient of multiple French national scientific awards and European Union and French government grants.

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BUSINESS INSIGHTS

Stevanato Group and Gerresheimer AG Announce Collaboration on the Development of an Innovative Ready-To-Use Vial Platform

Gerresheimer and Stevanato Group | September 08, 2022

Stevanato Group S.p.A. a global provider of drug containment, drug delivery and diagnostic solutions to the pharmaceutical, biotechnology and life sciences industries, and Gerresheimer AG, a leading global provider of healthcare & beauty and drug delivery systems for pharma, biotech and cosmetics, announce that they have jointly developed a high-end Ready-To-Use (RTU) solution platform with an initial focus on vials, based on Stevanato Group’s market-leading EZ-fill® technology. This collaboration is projected to help customers gain efficiencies, improve the quality standard, increase speed to market, reduce total cost of ownership (TCO) and help mitigate supply chain risk. Stevanato Group and Gerresheimer AG are addressing rising demand for RTU vials in the market and the partnership serves as a market enabler to fully support customers’ evolving needs and establish a gold standard in the industrial filling process. The collaboration aims to make RTU vials a standard, available to a wide number of pharma companies globally – offering premium quality solutions to patients and reducing complexity for pharma operations. The new solution will be available to other players in the market to standardize fill-finish operations from early phase drug development to commercialization. The new RTU solution platform from Stevanato Group and Gerresheimer AG will share the same secondary packaging, production process and sterilization method, ensuring consistent available capacity and a reliable double sourcing to the pharmaceutical industry. A hallmark of the new RTU platform is a significant reduction in particles, improving the overall quality and performance of the RTU solutions. In addition, customers can count on a high-end solution made by two major players in the drug containment industry with a long history of innovation. The new jointly developed vial platform and trademark will be presented in detail to the market at the CPhI Worldwide in Frankfurt in November 2022. “Our EZ-fill® vial platform is the market’s most established choice for pre-sterilized containers, and this enhanced version for RTU vials will bring to the market its full potential in terms of quality, flexibility and value for Pharmaceutical Companies and CMOs. Through our collaboration with Gerresheimer AG we are responding to market demand, and we expect to achieve another important milestone in creating a reliable ecosystem to support, accelerate and de-risk the conversion from bulk to EZ-fill® vials already underway in the market.” Mauro Stocchi, Chief Business Officer at Stevanato Group “The new developed RTU vial platform stands out in terms of quality, TCO and sustainability and takes the use of RTU solutions like vials and, in the future, cartridges to a new level,” said Dr. Lukas Burkhardt, Member of the Management Board of Gerresheimer AG. "Due to the strong reduction of the particle load, the quality is significantly increased. Our new innovative solution will convince the market to significantly accelerate the conversion from bulk to RTU vials.” About Stevanato Group Founded in 1949, Stevanato Group is a leading global provider of drug containment, drug delivery and diagnostic solutions to the pharmaceutical, biotechnology and life sciences industries. The Group delivers an integrated, end-to-end portfolio of products, processes and services that address customer needs across the entire drug life cycle at each of the development, clinical and commercial stages. Stevanato Group’s core capabilities in scientific research and development, its commitment to technical innovation and its engineering excellence are central to its ability to offer value added solutions to clients. About Gerresheimer AG Gerresheimer AG is the global partner for pharmaceutics, biotech, healthcare, and cosmetics with a very broad product range for pharmaceutical and cosmetic packaging solutions and drug delivery systems. The company is an innovative solution provider from concept to delivery of the commercial product. Gerresheimer achieves its ambitious goals through a high level of innovative strength, industrial competence and concentration on quality and customer focus. In developing innovative and sustainable solutions, Gerresheimer relies on a comprehensive international network with numerous innovation and production centers in Europe, America and Asia. Gerresheimer produces close to its customers worldwide with around 11,000 employees and generated annual revenues in 2021 of around EUR 1.5b. With its products and solutions, Gerresheimer plays an essential role in people's health and well-being.

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