O, Canada Like his across-the-aisle rivals, Trump looks to imports to cut drug costs

Fiercepharma | July 30, 2019

President Donald Trump and his 2020 challengers are in a heated skirmish to prove to voters who is the toughest candidate on Big Pharma. Now, Trump has found even more common ground with Democrats on lowering the cost of drugs. The Trump administration is drafting a plan to allow the Department of Health and Human Services (HHS) to “safely and effectively” import branded drugs from Canada. Freeing up Canadian imports would help lower drug prices by providing U.S. consumers with the “benefit of the deals that pharma themselves are striking with other countries,” HHS Secretary Alex Azar told CNBC’s “Squawk Box” on Tuesday. In most cases—often with the exception of chronic shortages—federal law prevents foreign drug importation. Azar, Trump's appointee, has balked at the idea in the past, as have previous FDA chiefs and many GOP politicians. But at least once each congressional session over the past decade or more, the idea ends up in a bill or two, only to be voted down. Now, though, the Canada import scheme represents a rare point of common cause between Trump and the Democratic candidates challenging him for the presidency in 2020.

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An online survey was conducted by the Analytical Scientist (a Texere Publication) on behalf of Thermo Fisher Scientific. The intent was to learn more about the application of ion chromatography (IC) in the bio/pharmaceutical industry and the level of awareness and applicability of pharmacopeial monograph modernization. The qualified responses collected in the survey are the basis for the data shared in this infographic.


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VIEWS AND ANALYSIS

Innovent and IASO Bio Present Updated Data of BCMA CAR-T Cell Therapy (Equecabtagene Autoleucel) at EHA 2022

Innovent Biologics | June 13, 2022

Innovent Biologics, Inc. a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, autoimmune, metabolic, ophthalmology and other major diseases, and IASO Biotherapeutics a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, today jointly announced that the updated data from phase 1/2 study of Equecabtagene Autoleucel (Innovent R&D code: IBI326, IASO Bio R&D code: CT103A), a fully-human anti-B cell maturation antigen chimeric antigen receptor T-cell therapy for the treatment of relapsed and/or refractory multiple myeloma (R/R MM), was presented in the form of an oral presentation at the 27th European Hematology Association Annual Meeting in Vienna on June 9-12, 2022. The updated data from the Phase 1/2 study with a longer duration of follow-up in more patients has showed durable and deepening efficacy, manageable safety and long-term in vivo persistence, indicating that Equecabtagene Autoleucel has the potential to be a breakthrough therapy for patients with R/R MM. The updated data is from the 14 clinical sites involved in the Phase 1/2 clinical study of Equecabtagene Autoleucel (ChiCTR1800018137, NCT05066646) in the treatment of patients with R/R MM. As of the data cutoff date of January 21, 2022, 79 patients received recommended phase 2 dose of 1.0×106 CAR-T cells/kg with the median follow-up of nine months (range 1.2, 19.6) and median prior five lines of therapy (range 3,23). Among the 79 patients, 34.2% (27/79) had high-risk cytogenetic abnormalities, 34.2%(27/79)had extramedullary multiple myeloma (EMM), and 15.2%(12/79)had received prior CAR-T therapy. Equecabtagene Autoleucel demonstrated a favorable and manageable safety profile: Among the 79 patients, 94.9% (75/79) experienced cytokine release syndrome (CRS). The majority experienced 1~2 CRS, and no patient experienced grade 3 CRS. The median time to CRS onset was six days after infusion, and the median duration of CRS was five days. Only two patients experienced immune effector cell-associated neurotoxicity syndrome (ICANS), including one patient who experienced grade 1 ICANS and one who experienced grade 2 ICANS. All patients with CRS or ICANS have recovered. Equecabtagene Autoleucel showed favorable and durable efficacy: Among the 79 patients, the overall response rate (ORR) was 94.9% (75/79), with "89.9% (71/79)" of those patients achieving very good partial response (VGPR) or deeper responses, and the complete response/stringent complete response (CR/sCR) rate was 68.4% (54/79). Equecabtagene Autoleucel also demonstrated favorable efficacy in 10 patients with EMM, achieving an ORR of 100% (10/10) and a CR/sCR rate of 90.0% (9/10). In all 79 patients, 92.4% (73/79) achieved minimal residual disease (MRD) negativity, all CR/sCR subjects achieved MRD negativity, and the median duration of MRD negativity was not reached. Equecabtagene Autoleucel demonstrated favorable efficacy in patients who had received prior CAR-T therapy: Among the 12 patients who previously received CAR-T therapy, the ORR was 75.0% (9/12), with 41.7% (5/12) of those patients achieving CR/sCR. Equecabtagene Autoleucel demonstrated robust expansion and prolonged persistence: The expansion of Equecabtagene Autoleucel in peripheral blood reached the peak at a median of 12 days, with a median Cmax of 92,000 copies/ug DNA. Equecabtagene Autoleucel was still detectable in 62.3% (38/61) and 53.3% (8/15) of the subjects who completed 6-months and 12-month follow-ups after infusion. Soluble BCMA in peripheral blood of patients rapidly declined after Equecabtagene Autoleucel infusion and persistently remained below the detectable limit. Equecabtagene Autoleucel has low immunogenicity: 16.5% (13/79) of the subjects tested anti-drug antibody (ADA)-positive after Equecabtagene Autoleucel infusion. Among them,1.3% (1/79) tested ADA-positive before Equecabtagene Autoleucel infusion, and 2.5% (2/79) tested ADA-positive within three months. Prof. Chunrui Li, MD, PhD, from Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology, stated "Chimeric antigen receptor (CAR)-T cell therapy is a revolutionary new pillar in cancer treatment. In our previous studies, Equecabtagene Autoleucel has shown excellent efficacy and manageable safety profiles. Its CAR structure contains fully human single-chain fragment variables (scFvs) to bypass potential anti-CAR immunogenicity of the host while retaining antitumor activity. At the 27th EHA conference, we updated the data on the efficacy and safety of Equecabtagene Autoleucel in R/R MM patients with longer median follow-up extended to 9.0 months, the CR/sCR deepened to 68.4%, compared with the CR/sCR of 58.2% with a median follow-up of 7.0 months, which were released at 63rd ASH conference in 2021. The updated data showed long-lasting safety and deepening efficacy of Equecabtagene Autoleucel. We are glad that Equecabtagene Autoleucel also shows favorable efficacy on patients who have relapsed after receiving prior CAR-T therapy. This has meaningful clinical value and is worthy of further exploration in the clinic to potentially bring forth new hope to patients with R/R MM." About Multiple Myeloma (MM) Multiple Myeloma is a deadly blood cancer that often infiltrates the bone marrow causing anemia, kidney failure, immune problems, and bone fractures. For multiple myeloma patients, common first-line drug treatments include proteasome inhibitors, immunomodulatory drugs, and alkylating agents. While treatment may result in remission, most patients will inevitably enter the relapsed or refractory stage as there's currently no cure. As a result, there is a significant unmet need for patients with relapsed/refractory multiple myeloma. In the United States, MM accounts for nearly 2% of all cancer cases, and more than 2% of cancer-related deaths. According to Frost & Sullivan, the number of new MM cases in the United States rose from 30,300 in 2016 to 32,300 in 2020 and is expected to increase to 37,800 by 2025. Additionally, the total number of patients diagnosed with MM increased from 132,200 in 2016 to 144,900 in 2020 and is expected to rise to 162,300 by 2025. In China, the number of new MM cases rose from 18,900 in 2016 to 21,100 in 2020 and is expected to increase to 24,500 by 2025. The total number of patients diagnosed with MM in China increased from 69,800 in 2016 to 113,800 in 2020 and is expected to rise to 182,200 by 2025. About Equecabtagene Autoleucel Equecabtagene Autoleucel is an innovative therapy co-developed by Innovent and IASO Bio, with a fully-human anti- BCMA CAR-T cell therapy which uses lentivirus as a gene vector to transfect autologous T cells. The CAR contains a fully-human scFv, CD8a hinge and transmembrane, and 4-1BB-mediated co-stimulation and CD3ζ activation domains. Based on rigorous screening and comprehensive in vivo and in vitro evaluation, Equecabtagene Autoleucel is proven to have potent and rapid anti-myeloma activity and outstanding safety, efficacy, and persistence results.

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PHARMACY MARKET

HKSTP and Boehringer Ingelheim Venture Fund Launch Co-Incubation Programme to Fast-track R&D in Infectious Diseases and Immunology

BIVF | June 18, 2022

Hong Kong Science and Technology Parks Corporation is partnering global research-driven biopharmaceutical leader, Boehringer Ingelheim Venture Fund Limited in a strategic co-incubation collaboration to promote and nurture startups on research and development for infectious diseases and immunology. HKSTP is dedicated to collaborating with sector leaders in building the strongest I&T eco-system to help startups via business development, mentorship and investment initiatives. In the last five years, the number of biotech companies at HKSTP have tripled from 50 to over 150, which has also synched with the Government’s strategic focus on biomedical technology with allocation of HK$10 billion to develop life and health technology as a key future growth sector for Hong Kong. This partnership between HKSTP and BIVF marks a key milestone to drive the Hong Kong’s biomedical technology development. Both local and global qualified startups in infectious diseases and immunology can apply to the incubation programme to access the full capabilities of the HKSTP ecosystem and BIVF’s extensive biotech funding network. Incubatees can access one-on-one coaching and assessment to track key research milestones, while receiving expert guidance from HKSTP on commercialisation, manufacturing, scaling-up and marketing strategy to ensure successful innovation, plus vital funding opportunities and investment insight from BI. “Hong Kong is now Asia’s largest and the world’s second largest fundraising hub for biotech. As HKSTP marks our 20th year of propelling success and innovation, our mission is to drive the growth of biotech to another level with world class leaders like BIVF. We will maximise the GBA growth opportunities for high-potential tech talents and early-stage startups to ensure the region emerges as a global I&T powerhouse.” Mr. Albert Wong, CEO of HKSTP Dr. Grace Lau, Head of Institute for Translational Research of HKSTP, said: “Our partnership with BIVF, provides early-stage startups and promising university spinoffs with vital support at the most critical stage of their long and challenging biotech innovation journey. Incubatees will have access to our Incu-Bio Programme, with total incubatees doubling up in the last five years. The startups can also access funding support of up to HK$6 million, with financial subsidies and upfront grants to cover regulatory activities such as clinical trials.” “The world has faced growing public health challenges in recent years. As Boehringer Ingelheim’s strategic investment arm, BIVF focuses on the development of new science in areas with huge unmet medical needs such infectious diseases and immunology,” said Dr. Frank Kalkbrenner, Global Head of the BIVF. “The co-incubation program jointly initiated by HKSTP and BIVF will enable us to identify more breakthrough technologies in the early stage of development. With the funding and infrastructure support offered by HKSTP and BI‘s expertise and experiences in the successful development of breakthrough mediactions for patients, we foster the startup companies and bridge the gap between science and industry for the local ecosystem. We look forward to developing together with HKSTP next-generation therapies in the fields of infectious diseases and immunology with long term partnerships.” “The innovation competency of biopharmaceuticals in Asia is rising quickly. To grab the opportunities in this market, Boehringer Ingelheim has set up the External Innovation Hub in China which brings our Research Beyond Borders, Business Development & Licensing and Venture Fund groups under one umbrella. Now we’re thrilled to see that the biopharmaceutical industry is picking up rapidly in the Guangdong–Hong Kong–Macau Greater Bay Area, it’s a great opportunity for us to partner with HKSTP to develop the local ecosystem, and further enhance China’s dual-circulation scheme. We hope to offer our continuous support to more home-grown innovations to be recognized on the global market and eventually benefit the patients worldwide.” said Mr. Felix Gutsche, President & CEO, Boehringer Ingelheim China. Programme incubatees will also benefit from HKSTP’s rapidly-growing biotech R&D capabilities including the HKSTP Institute for Translational Research (ITR), enabling biomedical startups to turn their innovative biomedical technologies to life-changing impact for patients and society. Also available to startups is HKSTP’s Incu-Bio program providing dedicated biotech mentorship, business matching, entrepreneur-in-residence, and access to R&D facilities and the Science Parks’ diverse talent pool. About Hong Kong Science and Technology Parks Corporation Hong Kong Science and Technology Parks Corporation (HKSTP) has for 20 continuous years committed to building up Hong Kong as an international innovation and technology hub to propel success for local and global pioneers today and tomorrow. HKSTP has established a thriving I&T ecosystem that is home to three unicorns and Hong Kong’s leading R&D hub with over 11,000 research professionals and over 1,000 technology companies focused on healthtech, AI and robotics, fintech and smart city technologies. Established in 2001, we attract and nurture talent, accelerate and commercialise innovation and technology for entrepreneurs on their journey of growth in Hong Kong, to the Greater Bay Area, Asia and beyond. Our growing innovation ecosystem is built around our key locations of the Hong Kong Science Park in Shatin, InnoCentre in Kowloon Tong and three modern INNOPARKs in Tai Po, Tseung Kwan O and Yuen Long. The three INNOPARKs are realizing a vision of re-industrialisation for Hong Kong. The goal is sectors like advanced manufacturing, electronics and biotechnology are being reimagined for a new generation of industry. Through our infrastructure, services, expertise and network of partnerships, HKSTP will help establish innovation and technology as a pillar of growth for Hong Kong, while reinforcing Hong Kong’s international I&T hub status as a launchpad for global growth at the heart of the GBA innovation powerhouse. About BIVF Created in 2010, the Boehringer Ingelheim Venture Fund GmbH invests in groundbreaking therapeutics-focused biotechnology companies to drive innovation in biomedical research. searching for significant enhancements in patient care through pioneering science and its clinical translation by building long-term relationships with scientists and entrepreneurs. BIVF’s focus is to target unprecedented therapeutic concepts addressing high medical needs in immuno-oncology, regenerative medicine, infectious diseases and digital health. These may include novel platform technologies to address so far undruggable targets, new generation vaccines and/or new biological entities, such as oncolytic virotherapy. About Boehringer Ingelheim External Innovation Hub China Established in 2020, Boehringer Ingelheim External Innovation Hub China unites three external innovation functions including Research Beyond Borders (RBB), Business Development & Licensing and the Boehringer Ingelheim Venture Fund. Through this innovative business model, Boehringer Ingelheim External Innovation Hub China is committed to providing one-stop solutions for biomedical innovators in China and. It is also dedicated to enriching the company's global R&D pipeline through external innovation collaborations so as to benefit more patients worldwide.

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BUSINESS INSIGHTS

Foundation Medicine Announces Strategic Collaboration with Arvinas

Foundation Medicine | June 06, 2022

Foundation Medicine, Inc., a pioneer in molecular profiling for cancer, announced a collaboration with Arvinas, Inc., to develop FoundationOne®Liquid CDx as a companion diagnostic for use with Arvinas’ bavdegalutamide an investigational novel PROTAC® protein degrader targeting the androgen receptor (AR). Arvinas’ bavdegalutamide is being developed for the potential treatment of men with metastatic castration resistant prostate cancer who have progressed on existing therapies. Arvinas is a clinical-stage biotechnology company and a pioneer in the rapidly growing field of targeted protein degradation. Arvinas’ proprietary PROTAC® targeted protein degraders, or proteolysis-targeting chimeras, work by harnessing the body’s natural protein disposal system to selectively and efficiently degrade and remove disease-causing proteins. AR activity is a key driver of prostate cancer, which makes the ability to regulate AR signaling an important factor in controlling disease progression. “We look forward to collaborating with Foundation Medicine to develop a companion diagnostic aimed at improving patient access. Foundation Medicine’s deep understanding of cancer genomics, scalable solutions, and regulatory expertise makes them an ideal partner for us as we develop bavdegalutamide as a potential new therapy for men with prostate cancer.” Ron Peck, M.D., chief medical officer at Arvinas Foundation Medicine’s portfolio of FDA-approved comprehensive genomic profiling tests offer physicians both blood- and tissue-based testing options for detecting genomic alterations that help guide personalized treatment decisions. As companion diagnostics, FoundationOne®CDx and FoundationOne®Liquid CDx allow oncologists to identify patients who may be appropriate for FDA-approved targeted therapies. “We are proud to serve as an end-to-end partner for Arvinas as they pioneer this new approach to treat cancer,” said Sanket Agrawal, chief biopharma business officer, Foundation Medicine. “Bringing our capabilities to this emerging area of biotechnology sets us on an exciting path to deepen our collective understanding of cancer biology and deliver more novel treatment options to patients now and in the future.” Foundation Medicine is an essential partner for biopharma organizations navigating the complexity of cancer care and research. This latest collaboration adds to its more than 65 global biopharma and biotechnology partnerships aimed at getting targeted cancer treatments to patients faster. About FoundationOne®CDx FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed, paraffin-embedded (FFPE) tumor tissue specimens. FoundationOne CDx is for prescription use only and is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Some patients may require a biopsy. About FoundationOne®Liquid CDx FoundationOne Liquid CDx is a qualitative next generation sequencing based in vitro diagnostic test for prescription use only that uses targeted high throughput hybridization-based capture technology to analyze 324 genes utilizing circulating cell-free DNA (cfDNA) isolated from plasma derived from anti-coagulated peripheral whole blood of advanced cancer patients. The test is FDA-approved to report short variants in over 300 genes and is a companion diagnostic to identify patients who may benefit from treatment with specific therapies (listed in Table 1 of the Intended Use) in accordance with the approved therapeutic product labeling. Additional genomic findings may be reported and are not prescriptive or conclusive for labeled use of any specific therapeutic product. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Patients who are negative for companion diagnostic mutations should be reflexed to tumor tissue testing and genomic alteration status confirmed using an FDA-approved tumor tissue test, if feasible. About Foundation Medicine Foundation Medicine is a pioneer in molecular profiling for cancer, working to shape the future of clinical care and research. We collaborate with a broad range of partners across the cancer community and strive to set the standard for quality, scientific excellence, and regulatory leadership. Our deep understanding of cancer biology helps physicians make informed treatment decisions for their patients and empowers researchers to develop new medicines. Every day, we are driven to help our partners find answers and take action, enabling more people around the world to benefit from precision cancer care.

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BUSINESS INSIGHTS

Prime Medicine Expands Leadership Team with Key Appointments to Support Continued Growth and Advancement of Prime Editing Technology and Portfolio

Prime Medicine | July 29, 2022

Prime Medicine, Inc. a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, Prime Editors, to address the widest spectrum of diseases by deploying Prime Editing technology “Prime Medicine is advancing a first-of-its-kind technology intended to expand the number of patients who may benefit from genetic medicines. We are thrilled to welcome Richard, Niamh and Fubao to the team. Together, they bring strong business acumen, extensive clinical and regulatory experience and leadership of successful teams that position us well to achieve our mission. These esteemed leaders have deep expertise across their respective focus areas, each of which is key to the advancement of our novel Prime Editing technology.” Keith Gottesdiener, M.D., Chief Executive Officer of Prime Medicine Richard Brudnick, Chief Business Officer, brings extensive industry experience, including leading successful business development transactions, across multiple stages of development and an array of therapeutic areas. Mr. Brudnick was previously Chief Business Officer and Head of Strategy at Codiak BioSciences and, before that, was Executive Vice President of Business Development and Alliance Management at Bioverativ, Inc., a company he helped found in 2016. Mr. Brudnick joined Bioverativ at the time of its spinoff from Biogen where, over the course of nearly 15 years, he initiated, led and completed numerous transactions, including for several of the company's marketed products and late-stage pipeline, including Tecfidera, Spinraza and its biosimilars joint venture with Samsung. Previously, Mr. Brudnick was the Chief Executive Officer of a regional pharmaceutical distribution business that he sold to a strategic buyer, co-founded two companies, and was a strategy consultant at Bain & Company. Mr. Brudnick earned his undergraduate degree in management science from MIT and his M.S. in management from the Sloan School of Management at MIT. Niamh Alix, Chief Human Resources (HR) Officer, is a human resources leader who brings nearly 20 years of experience helping to grow teams from small, early-stage organizations to global enterprises. She joins Prime from Orchard Therapeutics, where she was Vice President, Global Talent and HR Business Partner. Prior to that, Ms. Alix held multiple global and regional roles at Lonza, including HR Business Partner for Americas, Mexico, and Canada where she led the HR team and people strategy for a 5,000+ employee population across corporate and manufacturing clients, and across multiple scientific platforms including cell and gene therapy. Prior to that, she held senior HR roles of increasing responsibility at Novartis and Bristol Myers Squibb. She holds a master’s degree in HR strategies from Dublin City University and a B.Sc. in management from the Technological University of Dublin. Fubao Wang, Ph.D., SVP, Head of Regulatory, has substantial experience leading product development of genomic medicine products, as well as regulatory strategies and execution for both clinical and commercial-stage products. Before Prime, Dr. Wang served as Senior Vice President, Head of Regulatory Affairs at Asklepios BioPharmaceutical where he built and led the regulatory team to support the development of clinical-stage AAV-based gene therapy products. Earlier, he was Vice President, Head of Regulatory CMC at Sarepta Therapeutics, where he supported the development of AAV gene therapy and RNA products, including the FDA approvals of Vyondys and Amondys. He also served as Associate Vice President, U.S. Site Head, CMC Dossiers at Sanofi to support the BLA approval of Dupixent, a monoclonal antibody product. Prior to that, he held a 16-year tenure at Merck serving in research, development and regulatory roles of increasing responsibility, most recently as director, global and emerging markets regulatory affairs, led and contributed to the discovery and development of a variety of vaccine and biologics products. Dr. Wang holds a Ph.D. in molecular biology from Heidelberg University and completed his postdoctoral work in molecular biology at Stanford University. About Prime Medicine Prime Medicine, Inc. is a biotechnology company committed to delivering a new class of differentiated, one-time, curative genetic therapies to address the widest spectrum of diseases. The company is deploying Prime Editing technology, a versatile, precise, efficient and broad gene editing technology, which is designed to make only the right edit at the right position within a gene. With the theoretical potential to repair approximately 90 percent of known disease-causing genetic mutations across many organs and cell types, medicines based on Prime Editing, if approved, could offer a one-time curative genetic therapeutic option to a broad set of patients.

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Spotlight

An online survey was conducted by the Analytical Scientist (a Texere Publication) on behalf of Thermo Fisher Scientific. The intent was to learn more about the application of ion chromatography (IC) in the bio/pharmaceutical industry and the level of awareness and applicability of pharmacopeial monograph modernization. The qualified responses collected in the survey are the basis for the data shared in this infographic.

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