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Ascentage Pharma Announces its 9th Orphan Drug Designation from the US FDA in 2020

Ascentage Pharma | January 05, 2021

Ascentage Pharma Announces its 9th Orphan Drug Designation from the US FDA in 2020
Ascentage Pharma (6855.HK), an internationally engaged, clinical-stage biotechnology organization occupied with creating novel treatments for malignancies, persistent hepatitis B (CHB), and age-related sicknesses, today reported that the US Food and Drug Administration (FDA) has allowed an Orphan Drug Designation (ODD) to the organization's novel Bcl-2 inhibitor, APG-2575, for the therapy of patients with intense myeloid leukemia (AML). This is the fourth ODD allowed to APG-2575 by the US FDA, following the past ODDs for the therapy of Waldenström macroglobulinemia (WM), ongoing lymphocytic leukemia (CLL), and numerous myeloma (MM). Until this point, four of Ascentage Pharma's investigational drug up-and-comers have been allowed an aggregate of nine ODDs by the US FDA, a record number for any Chinese biopharmaceutical organization.

AML is an exceptionally heterogenous hematologic threat that is more normal in the old populace with a middle age at analysis of 68 years1. The latest information from the Surveillance, Epidemiology, and End Results Program (SEER) of the US National Cancer Institute (NCI) assessed 19,940 new instances of AML and 11,180 passings from this infection in the United States in 2020. Notwithstanding the critical advances in therapeutics as of late, the 5-year endurance pace of AML stays at 25%–30%, which actually speaks to a huge neglected clinical requirement for treatments with more solid viability and a superior wellbeing profile.

The expression "vagrant medications" alludes to drug items produced for the counteraction, finding, and treatment of uncommon illnesses or conditions. In the United States, a vagrant sign is characterized as a sickness or condition with a pervasiveness of under 200,000 patients in the nation. Since the Orphan Drug Act was passed in 1983, the US government has given motivators and strategy backing to support advancement of vagrant medications. Treatments conceded ODDs by the FDA are equipped for different advancement motivators, including a tax break on consumptions brought about in clinical examinations, a waiver of the New Drug Application (NDA) charge, conceivable exploration award granted by the FDA, and above all, 7 years of US market restrictiveness upon endorsement.

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The briefing note at hand has been prepared to annotate with further conceptual clarity the structure and organization of the Framework for Action, with the objective of specifying the following: its strategic vision according to the determinants of people-centered health services; how needed transformations are reasoned beginning from a root cause analysis of the function itself; and the translation of the defined vision and reasoned determinants into the core components of the Framework.

Spotlight

The briefing note at hand has been prepared to annotate with further conceptual clarity the structure and organization of the Framework for Action, with the objective of specifying the following: its strategic vision according to the determinants of people-centered health services; how needed transformations are reasoned beginning from a root cause analysis of the function itself; and the translation of the defined vision and reasoned determinants into the core components of the Framework.

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PHARMA TECH

Bayer and Mammoth Biosciences to Collaborate on Novel Gene Editing Technology

Bayer and Mammoth Biosciences | January 10, 2022

Bayer AG and Mammoth Biosciences, Inc., which is harnessing the diversity of nature to power the next-generation CRISPR products, announced a strategic collaboration and option agreement for the use of Mammoth’s CRISPR systems to develop in vivo gene-editing therapies. Mammoth Biosciences’ groundbreaking gene-editing technology is a key enabling technology, as well as a stand-alone therapeutic modality. It will significantly enhance Bayer’s efforts to develop transformative therapies for patients faster and strengthen the company’s recently established new cell and gene therapy platform. Under the terms of the agreement the two companies will start their collaboration with a focus on liver-targeted diseases. “Bringing together Mammoth’s novel CRISPR systems with our existing gene augmentation and our induced pluripotent stem cell (iPSC) platforms will allow us to unleash the full potential of our cell and gene therapy strategy. Partnering with Mammoth’s cutting edge scientific team is a fundamental pillar for our company to improve the lives of patients suffering from conditions that are currently still difficult to treat.” Stefan Oelrich, Member of the Board of Management, Bayer AG and President of the Bayer’s Pharmaceuticals Division “We’re excited to be working together with Bayer, building on the technology leap of our novel CRISPR systems, along with Bayer’s expertise in successful drug development,” said Dr. Peter Nell, Chief Business Officer and Head of Therapeutic Strategy at Mammoth. “This joint effort has the potential to benefit patients by developing CRISPR-based approaches for the clinic with the appropriate urgency, while ensuring scientific excellence and safety.” Cell and gene therapies are the next step in the evolution of drug development. By addressing the root cause of diseases, they are potentially capable of permanently reversing diseases with a one-time treatment. Gene editing serves as a key enabler for cell therapies when used outside the living body and allows therapeutic targeting of a wide range of genetic diseases with a high unmet medical need when used inside the living body. Mammoth Biosciences’ proprietary toolkit of ultra-small Cas enzymes, including Cas14 and Casɸ, allows for expanded high-fidelity gene editing to be combined with targeted systemic delivery. Under the agreement, Bayer gains access to this novel gene-editing technology, which offers the potential of an advanced in vivo applicability due to the ultra-compact size of these novel CRISPR systems. Under the terms of the agreement, Mammoth Biosciences will receive an upfront payment of USD 40 million and is eligible to receive target option exercise fees as well as potential future payments in the magnitude of more than one billion USD upon successful achievement of certain research, development, and commercial milestones across five preselected in vivo indications with a first focus on liver-targeted diseases. In addition, Bayer will pay research funding and tiered royalties up to low double-digit percentage of net sales. The companies are also exploring work on ex vivo projects on a nonexclusive basis. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to drive sustainable development and generate a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2020, the Group employed around 100,000 people and had sales of 41.4 billion euros. R&D expenses before special items amounted to 4.9 billion euros. About Mammoth Biosciences Mammoth Biosciences is harnessing the diversity of life to power the next generation of CRISPR products. Through the discovery and engineering of novel CRISPR systems, the company is enabling the full potential of its platform to read and write the code of life. Mammoth aims to develop permanent genetic cures through best-in-class in vivo and ex vivo therapies and to democratize disease detection with on-demand diagnostics. By leveraging its internal research and development and exclusive licensing to Cas12, Cas13, Cas14, and Casɸ, Mammoth can provide enhanced diagnostics and genome editing for life science research, healthcare, agriculture, biodefense and more. Based in the San Francisco Bay Area, Mammoth Biosciences is co-founded by CRISPR pioneer and Nobel Laureate Jennifer Doudna and Trevor Martin, Janice Chen, and Lucas Harrington. The firm is backed by top institutional investors including Redmile Group, Foresite Capital, Senator Investment Group, Sixth Street, Decheng, Mayfield, NFX, and 8VC.

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Recursion Acquires Vium Strengthening Its Attempts to Industrialize Drug Discovery

Recursion, Vium | July 29, 2020

Recursion, a digital biology company leading in the industrialization of drug discovery, today announced its acquisition of Vium, a digital vivarium company. Vium’s platform combines computer vision, machine learning and sensor technologies to non-invasively collect and derive insights from digital biomarkers in preclinical in vivo research. Recursion’s acquisition of Vium signals continued and rapid integration of advanced technologies to grow its drug discovery pipeline of over 30 programs along with programs developed jointly with large pharmaceutical partners. “Technology is set to change the pace, scale and cost of drug discovery over the coming decades. At Recursion, we are building a vast machine to rapidly discover drugs through the integration of technology at every step in the process,” said Chris Gibson, Ph.D., co-founder and CEO of Recursion. “Bringing the fantastic team at Vium in-house will enable us to build on our core technology and vision, augmenting our huge in vitro datasets with large, complementary in vivo datasets, and advancing our mission to decode biology to radically improve patient lives.”

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AmerisourceBergen & Global Pharmaceutical Manufacturers Discuss “Moving Health Forward” at Second Annual ThinkLive Trade Virtual Conference

AmerisourceBergen | October 22, 2021

Today, AmerisourceBergen concluded its second annual ThinkLive Trade virtual event, a three-day online summit geared toward brand, specialty, generic and consumer-product manufacturers. Over the course of the event, AmerisourceBergen leaders discussed the ever-evolving challenges and considerations that these manufacturers face when bringing products to market, and how AmerisourceBergen works with its partners to drive supply chain sophistication, maximize patient access, and move health forward. AmerisourceBergen also introduced its new President of Manufacturer Services and Commercial Solutions, Willis Chandler, during the conference – a new role for the organization.“The last two years have proven how dynamic, fast-paced and essential the pharmaceutical industry and supply chain are to advancing global health—from launching new therapies, to maintaining consistent access to daily prescriptions, to providing frontline workers with the products and resources they need to stay safe amid a pandemic,” said Willis Chandler, who recently stepped into the new role as President of Manufacturer Services and Commercial Solutions from leading AmerisourceBergen’s health systems and specialty services organization. “ThinkLive Trade is an opportune time for us connect with manufacturers and dig deeper into their needs, challenges and opportunities in the year ahead. I plan to carry our key learnings from the conference forward, and I cannot wait to bring more novel solutions to the market in collaboration with our manufacturer partners.” This year’s conference programming included remarks from AmerisourceBergen Chairman, President & CEO Steve Collis, dynamic panel discussions, presentations, awards, virtual networking sessions and more. Content and meetings were facilitated through AmerisourceBergen’s online experience platform, AmerisourceBergenLive. On the first day of the summit, attendees heard directly from AmerisourceBergen executives and customers on the state of the industry, strategies to improve global health equity, and sustainable and responsible approaches to healthcare advancement. Day two of ThinkLive Trade featured discussions on technology and infrastructure enhancements to drive innovation, supply chain continuity​, and AmerisourceBergen’s strategy and focus for the year ahead. The final day of the summit focused on the intersection of healthcare and politics, featuring a fireside chat with, Mark McClellan, MD, PhD, founding Director of the Duke-Margolis Center for Health Policy at Duke University, as well as timely discussions on global generics, specialty pharmaceutical products, biosimilars, and more. “The resilience of the pharmaceutical supply chain in the face of a global pandemic and evolving market dynamics continues to reinforce the value of strategic relationships, Our partners are developing novel therapeutics, creating pharmaceuticals to manage chronic conditions in patients worldwide, producing essential generics to ensure cost-effective medicines are available in today’s marketing, developing consumer products that are essential to fighting this pandemic and beyond, and so much more. We are thankful for their partnership and to all who attended ThinkLive Trade. This week’s conference encouraged meaningful dialogue that will help us advance healthcare efficiency and supply chain reliability in the year ahead.” Janine Burkett, SVP & President, Strategic Global Sourcing at AmerisourceBergen. AmerisourceBergen will also host ThinkLive Commercialization early next year. The event will bring together emerging biotech and biopharma leaders to discuss the latest in launch strategies across different therapeutic categories. About AmerisourceBergen AmerisourceBergen fosters a positive impact on the health of people and communities around the world by advancing the development and delivery of pharmaceuticals and healthcare products. As a leading global healthcare company, with a foundation in pharmaceutical distribution and solutions for manufacturers, pharmacies, and providers, we create unparalleled access, efficiency, and reliability for human and animal health. Our 41,000 global team members power our purpose: We are united in our responsibility to create healthier futures. AmerisourceBergen is ranked #8 on the Fortune 500 with more than $200 billion in annual revenue

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