Takeda swings and misses with Ninlaro in AL amyloidosis, calls off phase 3 trial

fiercepharma | June 06, 2019

With its once-biggest seller Velcade bracing for generic competition, Takeda has big plans for its oral multiple myeloma drug Ninlaro. But now, the med has suffered two setbacks in the span of a few months. After Ninlaro hit a snag at the FDA in an expanded use back in February, the company this week canned a phase 3 trial in another rare disease. Ninlaro had been in phase 3 testing for patients with systemic light-chain amyloidosis, but an analysis showed the med didn't meet a primary endpoint. Investigators were testing a combo of Ninlaro and dexamethasone in patients who'd relapsed or failed to respond to another treatment. Compared with a doctor’s choice of standard chemotherapy, Ninlaro didn’t improve overall hematologic response, so Takeda called the trial off. In patients with the disease, certain plasma cells produce abnormal immunoglobulin light-chain fragments. Patients develop amyloid deposits in organs and tissues, particularly the kidneys, heart, liver and nerves, Takeda said. That, in turn, can lead to organ failure and death.

Spotlight

High standards have to be met by the pharmaceutical industry when it comes to drug quality and safety. These standards are documented in pharmacopoeias as officially recognised pharmaceutical rules, and published as legal tools of customer protection by authorities such as governments and medical societies.

Spotlight

High standards have to be met by the pharmaceutical industry when it comes to drug quality and safety. These standards are documented in pharmacopoeias as officially recognised pharmaceutical rules, and published as legal tools of customer protection by authorities such as governments and medical societies.

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PHARMA TECH

KiOmed Pharma and Hansoh Pharma Announce License Agreement for KiOmedinevsOne in China’s Mainland, Macau and Taiwan

KiOmed Pharma and Hansoh Pharma | September 27, 2022

KiOmed Pharma, a Belgian biotechnology company with a focus on the development, manufacture and distribution of safe and effective medical devices based on a medical-grade highly pure natural chitosan-derivative, and Hansoh Pharmaceutical Group Company Limited a leading biopharmaceutical company in Asia, today announced a license agreement for the development and commercialization of KiOmedinevsOne, a carboxymethyl chitosan injection that has been launched in Europe for the treatment of knee osteoarthritis, in China’s mainland, Macau and Taiwan. Under the terms of the agreement, KiOmed will receive an upfront payment and will be eligible to receive development, regulatory and commercial milestones of up to € 66 million, as well as tiered royalties on future product sales. Hansoh will receive exclusive rights to develop and commercialize KiOmedinevsOne and its future extensions in the territory. KiOmed retains the right to develop and commercialize KiOmedinevsOne in all other global markets. Osteoarthritis is a progressive and disabling disease affecting about 250 million people worldwide. According to a recent epidemiology report, the prevalence of knee osteoarthritis in China is 8.1%. As the population in China is aging rapidly, disease burden caused by knee osteoarthritis continues to rise while safe and effective treatment options are still lacking. “We are very excited to partner with Hansoh Pharma, one of the largest biopharmaceutical entities in Greater China. We believe that with Hansoh’s impactful presence in China, their support will be essential to bring this long-acting treatment to the large population of patients suffering from osteoarthritis in China. We launched KiOmedinevsOne last year in several regions of Europe, and the feedback we continue to receive from osteoarthritis patients and physicians is great. With just one single injection to the knee, it can provide symptoms relief including pain, stiffness, and function improvement for more than 6-month. It is the world-first next generation non-animal sourced chitosan injection with a potential safety advantage over animal-sourced chitosan,” added Dr. Houtaï Choumane, CEO and Managing Director of KiOmed. Mr. François Blondel, Founder and Executive Chairman of the Board of KiOmed Pharma “KiOmed is a leader and an expert in developing natural chitosan products to fulfill unmet medical needs in different spaces. Osteoarthritis negatively impacts an individual’s quality of life and creates a large burden for the patients and their families. We are very glad to see KiOmed’s product (KiOmedinevsOne) will help to solve this problem,” said Eliza Sun, Executive Director of the Board of Hansoh Pharma. “We are also very excited to partner with them and bring this unique product to much needed patients in China.” About KiOmedinevsOne KiOmedinevsOne is a new generation single injection for the treatment of knee osteoarthritis based on world-first exclusive animal free KiOmedine® CM-Chitosan. KiOmedine® CM-Chitosan is a highly purified polysaccharide derived from Agaricus Bisporus (button mushroom) and a patented technology made in Belgium, resulting from years of research and innovation. Different from hyaluronic acid, KiOmedinevsOne has dual mechanism of action to tackle osteoarthritis discomfort and other symptoms by reducing oxidative stress and enhancing joint lubrication. Clinical studies have shown that after a single injection of KiOmedinevsOne, significant pain reduction was observed within 2 weeks and the WOMAC pain score was reduced by 66%, with a long-lasting osteoarthritis symptom reduction for at least 6 months. KiOmedinevsOne is a regulated health product which bears the CE mark under this regulation. It has been launched in Europe in 2021. About KiOmed Pharma Based in Belgium, KiOmed Pharma has a history of innovation and expertise in exclusive natural chitosan chemistry. The company develops a unique pipeline of medical devices that address unmet medical needs in high impact pathologies and major social burdens such as osteoarthritis, skin aging and ophthalmology. KiOmed Pharma's innovative pipeline is based on a solid building block exclusive technology: KiOmedine®, a medical-grade highly pure natural chitosan-derivative. About Hansoh Pharmaceutical Group Hansoh Pharma one of the largest biopharmaceutical companies in China, is committed to discovering and developing life-changing medicines to help patients conquer serious diseases and disorders.

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BUSINESS INSIGHTS

Astellas and MBC BioLabs Announce Astellas Future Innovator Prize to Help Biotech Start-ups Accelerate Early Drug Discovery and Research Efforts

Astellas Pharma Inc. | September 20, 2022

Astellas Venture Management LLC a wholly-owned venture capital subsidiary of Astellas Pharma Inc. and Mission Bay Capital BioLabs a life-science incubator, today announced their collaboration on the "Astellas Future Innovator Prize" formerly known as the Astellas Golden Ticket competition. Building on over three successful years, the competition offers entrepreneurial scientists or emerging biotechnology start-ups one year's priority usage of MBC BioLabs' state-of-the-art lab facility and access to Astellas' research and development (R&D) capabilities and business leaders. With a shared commitment to discovering and advancing innovative science for the potential future benefit of patients worldwide, AVM and MBC BioLabs are continuing their partnership to support scientists and early-stage companies to accelerate their novel therapeutic programs, modalities or platforms. The 2022 competition will prioritize innovative research that compliment Astellas' Focus Area Approach and pipeline. "We are truly thrilled to host the Astellas Future Innovator Prize again this year. Through our long-term collaboration with MBC BioLabs, we aim to create an environment that encourages innovation to thrive. The Astellas Future Innovator Prize enables us to provide biotech companies with expertise and capabilities to turn an innovative idea into a real solution, which could transform the lives of patients in need around the world." William Watt, President, AVM "We are pleased to continue our partnership with Astellas to uncover the innovators of tomorrow," said Douglas Crawford, MBC BioLabs General Manager. "The past winners of the Astellas Future Innovator Prize show what can be achieved by combining Astellas' support, advice and expertise with our laboratory incubator, and we are excited to see the result of our ongoing collaboration." Entrepreneurial scientists, emerging life-science and biotechnology start-ups have until November 4, 2022 to enter the Astellas Future Innovator Prize. About the Astellas Future Innovator Prize at MBC BioLabs Astellas is offering up to two prizes for pioneering scientists with innovative research that complements Astellas' areas of interest in alignment with its Focus Area Approach and pipeline, including Blindness & Regeneration, Mitochondria, Genetic Regulation, Immuno-Oncology, Cell Therapy, and other areas. The 2021 Astellas Future Innovator Prize winners were Vcreate and Weatherwax Biotechnologies, chosen for the potential of their innovations to deliver therapeutic advances for unmet medical needs and their potential synergy with Astellas' Focus Area Approach. About Astellas Venture Management LLC AVM is the wholly-owned venture capital organization within Astellas, dedicated to supporting pre-clinical, cutting-edge science that can bring VALUE to patients. For over 15 years, AVM has provided equity investments to private, early-stage companies developing therapeutic programs and platform technologies, helping them to advance their innovations faster. AVM is a strategic investor, making investments in science that will enhance the current Astellas R&D pipeline or that could catalyze new directions in discovery research. About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. About MBC BioLabs MBC BioLabs is dedicated to helping life-science startups succeed. By renting space as small as a single bench and providing these entrepreneurial scientists with access to millions of dollars of equipment, MBC BioLabs allows companies to be fast, focused, and frugal. It has three sites: one in the Dogpatch neighborhood in San Francisco and two campuses in San Carlos, California. Each site has a complete molecular biology core facility that allows companies to do experiments on day one. MBC BioLabs has partnerships with leading pharmaceutical and life-science companies as well as a built-in venture capital firm, Mission BioCapital. These partnerships provide entrepreneurs with valuable insights about where to focus their efforts and accelerates the innovation pipeline. Since opening in 2013, MBC BioLabs has helped launch and grow 290 companies. These companies have brought 58 programs to the clinic, produced 13 approved diagnostics, and raised over $14 billion.

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BUSINESS INSIGHTS

NeuroBo Pharmaceuticals, Inc. and Dong-A ST Co. Ltd. Announce Strategic Collaboration

NeuroBo Pharmaceuticals, Inc. | September 16, 2022

NeuroBo Pharmaceuticals, Inc. and Dong-A ST Co., Ltd. announced that they have entered into a conditional exclusive license agreement for NeuroBo to develop and commercialize DA-1241 and DA-1726, which are currently being evaluated for the treatment of nonalcoholic steatohepatitis obesity and type 2 diabetes. DA-1241 is a novel G-Protein-Coupled Receptor 119 agonist, which promotes the release of key gut peptides GLP-1, GIP and PYY, which, in turn, play an important role in glucose metabolism, lipid metabolism and weight loss. DA-1241 is a synthetic, selective small molecule, suitable for oral administration and has been shown to be well tolerated in phase 1 studies. Further, its multimodal mechanism appears to induce strong anti-NASH effects, supported by potential best-in-class efficacy, as demonstrated in pre-clinical studies. DA-1726 is a novel oxyntomodulin analogue functioning as a glucagon-like peptide-1 receptor and glucagon receptor dual agonist. OXM is a naturally-occurring, 37-amino acid peptide hormone that is released from the gut after ingestion of a meal, activating both the GLP-1 and glucagon receptors, prompting reduced food intake as well as an increase in energy expenditure, potentially resulting in superior body weight loss compared to selective GLP-1 receptor agonists. The beneficial effects of this dual mechanism of DA-1726 on weight loss compared to selective GLP-1 activity has been demonstrated in animal models. Additionally, DA-1726 has shown the ability to improve hepatic steatosis, inflammation and fibrosis when compared to the GLP-1 agonist, semaglutide in these same models. Under the license agreement, NeuroBo will be responsible for global development, regulatory and commercial activities other than for certain Asian-Pacific geographies. Dong-A will manufacture clinical supplies and initial commercial supplies of the product at its manufacturing facility in Korea. "The acquisition of these two cardiometabolic assets marks a seismic shift for NeuroBo, providing us with a highly promising, diversified pipeline with several upcoming value inflection points in the NASH and obesity space -- areas with enormous market opportunity," stated Gil Price, M.D., President and Chief Executive Officer of NeuroBo. "Through this agreement, Dong-A, one of our largest shareholders, has reaffirmed its commitment to remain a long-term strategic partner of NeuroBo. Dong-A is dedicated to our success and we are grateful it has also committed to provide continued support to facilitate the clinical development of the licensed assets. Once the transaction has closed, which is contingent upon certain closing conditions, we will be uniquely positioned to initiate a phase 2a study of DA-1241 in NASH in the first half of 2023, with data expected in the second half of 2024. We also intend to initiate a phase 1a safety study of DA-1726 in the first half of 2023, for which data is expected in the second half of 2023. We are truly excited about the prospects of NeuroBo as we transition to a cardiometabolic company across the large and growing markets of obesity and NASH." "We are highly enthusiastic about this opportunity to accelerate development of our novel treatments in partnership with NeuroBo. Dong-A plans to continue to strengthen its R&D capability and to seek additional collaboration opportunities to establish ourselves in the US market", Min Young Kim, Chief Executive Officer of Dong-A About the Proposed Licensing Transaction Under the terms of the license agreement, Dong-A will receive an upfront payment of $22 million in Series A convertible preferred stock, which will automatically convert into common stock upon receipt of requisite stockholder approval, and will be eligible to receive commercial- and regulatory-based milestone payments, dependent upon the achievement of specific regulatory and commercial developments. Dong-A will also be entitled to single digit royalties on net sales of the two assets. Dong-A has also agreed to commit $15,000,000 toward financing the assets, subject to NeuroBo's ability to obtain additional financing under the terms of the license agreement. The license agreement has been approved by the board of directors of NeuroBo. The transaction is expected to close in the third quarter of 2022, subject to obtaining third party financing for development of the assets and other customary closing conditions. About NeuroBo Pharmaceuticals NeuroBo Pharmaceuticals, Inc., is a clinical-stage biotechnology company historically focused on therapies for neurodegenerative, infectious, and, upon closing of the license agreement, cardiometabolic diseases. Its therapeutics programs currently include ANA001, an oral niclosamide formulation, which is in Phase 2/3 clinical trials to treat patients with moderate coronavirus disease (COVID-19); NB-01 for the treatment of painful diabetic neuropathy; NB-02 for the treatment of symptoms of cognitive impairment and to modify the progression of neurodegenerative diseases associated with the malfunction of tau protein; and gemcabene currently being assessed as an acute treatment for COVID-19 in combination with ANA001. NeuroBo Pharmaceuticals, Inc. is headquartered in Boston, Massachusetts. About Dong-A Dong-A ST Co. is a leading healthcare company in South Korea with a business focus on developing, manufacturing and distributing pharmaceutical products and medical devices worldwide. Dong-A has successfully developed and marketed several products globally and continues to develop prospective clinical candidates. Dong-A also provides licensed-in and licensed-out drugs, and medical devices, including high-technology medical devices, custom-made products, and sets of artificial cardiac circuits for use in open-heart surgery. Dong-A has over 5,500 employees including 2,300 in the pharmaceutical sector. Dong-A was founded in 1932 and is headquartered in Seoul, South Korea.

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