NHS England and Vertex break stalemate over price of CF drugs

Pharmaceutical Technology | October 24, 2019

Four years after Orkambi was approved in the European Union, this life-saving drug will now be available to Cystic Fibrosis (CF) patients in England through the NHS. The reason for the delay was continued failing pricing negotiations between Orkambi's manufacturer, Vertex, and NHS England, leading to patient advocacy campaigns supported by Labour Party leader Jeremy Corbyn. Vertex and NHS England have announced they have come to an access agreement for all three of the former’s licensed Cystic Fibrosis (CF) drugs, Orkambi (lumacaftor/ivacaftor), Symkevi (tezacaftor/ivacaftor) and Kalydeco (ivacaftor). This means within 30 days NHS England physicians will be able to prescribe these drugs for eligible patients. Currently, this totals around 5,000 patients based on the approved indications of the CF drugs, but there is no limit to the number of patients as the access agreement is linked with all future indications of Orkambi, Symkevi and Kalydeco. Orkambi will be available to all patients over the age of two with two copies of the F508del mutation in the Cystic Fibrosis transmembrane conductance regulator (CFTR) gene, while Symkevi is indicated for patients aged 12 or older with either two copies of F508del mutation in the CFTR gene or one F508de mutation and another of 14 other mutations in the same gene.

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Elligo Health Research® Partners With Peoples Rx

Elligo Health Research | April 21, 2022

Elligo Health Research, the largest healthcare-enabling research organization, announced they are expanding partnerships to include Peoples Rx, an Austin, Texas-based pharmacy chain. “We’re in a unique position at Peoples Rx to help inform more people about how they can participate in clinical research opportunities. Working with Elligo to offer research as healthcare to our customers helps support our mission to empower people to make educated health decisions.” Stephen Erickson, Peoples Rx president One of the first studies Peoples Rx will be working to share with Elligo is a study for people to advance screening diagnostics for cancer. “Our patients may not hear about clinical research opportunities otherwise, and this empowers patients with knowledge to become more proactive in their own healthcare journey while simultaneously supporting the advancement of health sciences,” Erickson said. Elligo began its mission to provide easy access to clinical research as care to more patients nearly six years ago. Specifically, Elligo is focused on accelerating clinical trials through healthcare by maintaining the integrity of the trusted patient and physician relationship, building local healthcare communities, and leveraging electronic health records. “Accomplishing this mission is made possible by the partnerships we have with real-world healthcare,” said Elligo CEO John Potthoff, Ph.D. “Peoples Rx has a positive impact on our local community and is a trusted partner for healthcare. This is also Elligo’s first partnership with a pharmacy, and it is our hope that this will lead to further opportunities to collaborate with more providers in the healthcare spectrum on similar initiatives and continue contributing to the changing clinical research landscape.” About Elligo Health Research® Elligo Health Research accelerates clinical trials through healthcare with access to known patients and their HIPAA-compliant healthcare data, our IntElligo® Research Stack technology, and our hybrid enrollment model, PatientSelect. Coupled with the largest Known Patient Access Network, Elligo’s Site Solutions enable healthcare practices and research sites to participate in clinical trials. By adaptive engagement of known patients and physicians, we accelerate the development of new pharmaceutical, biotechnology, and medical device and diagnostic products. About Peoples Rx Peoples Rx has been the local favorite pharmacy and wellness headquarters in Austin for over 40 years. Our integrative approach to health and wellness starts with our knowledgeable and experienced pharmacists and wellness specialists, and our cutting-edge, holistic, and professional products. We also compound custom medications in our state-of-the-art lab to meet customers’ specific needs. And because food is medicine too, our own in-house delis serve up scratch-made meals and snacks with the highest quality ingredients. With a mission to empower people to make educated health choices, several of our 170+ employees, including clinical nutritionists, herbalists, naturopaths, and homeopaths also offer their expertise through private consultations and educational events.

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BUSINESS INSIGHTS

Agilent Announces the Innovative Seahorse XF Pro Analyzer

Agilent Technologies Inc. | January 25, 2022

Agilent Technologies Inc. announced the release of the Agilent Seahorse XF Pro Analyzer, featuring new pharma-oriented workflow solutions incorporating advanced experimental design and analysis tools. With enhanced performance, more robust and efficient workflows, and an optimized user experience, the XF Pro enables operators at any skill level to access the most advanced cellular metabolism analysis technology for understanding cellular fate, fitness, and function. Preclinical therapeutic discovery workflows place high demands on cellular analysis, with immunology and disease researchers increasingly using rare, ex vivo, and genetically engineered cells to build better disease models. To address the increasing interest in monitoring live cells in real-time, the new XF Pro Analyzer incorporates a suite of hardware and software enhancements that improve measurement performance and data interpretation. These enhancements will make it easier to identify novel drug targets, validate target effect on cellular function, optimize disease models, and determine drug safety and antitumor potential of T cell therapies. “With dedicated workflow solutions the XF Pro provides enhanced performance and customer experience, particularly within pharma and biopharma therapeutic development, and toxicity programs. This underlines Agilent’s commitment towards developing intentional end-to-end solution-focused innovations that continue to lead the way for applied analytical and research applications, including direct metabolic analysis.” Richard Fernandes, associate vice president and general manager of Agilent’s Cell Analysis, Seahorse, Luxcel, and microplates businesses "The launch of the XF Pro is very much a customer-driven event. Through collaboration and intentional conversations with our customers and key opinion leaders, we recognized the need for a live-cell metabolic analysis platform which makes sophisticated analysis even more accessible for both experienced users as well as novices,” added Chris Braun, associate vice president of Marketing for Agilent’s Cell Analysis Division. “Primarily targeting the pharma/biopharma sector, but beneficial to all our customers, the XF Pro incorporates critical metabolic insights into researchers’ discovery workflows to facilitate even higher quality data where it matters most.” The XF Pro Analyzer is expected to enhance critical aspects of live-cell analysis, particularly for researchers working in immunotherapy, early drug discovery, and preclinical safety assessment. By delivering better precision at a low oxygen consumption rate (OCR), the XF Pro Analyzer allows analysts to confidently interrogate more immune cell types, as well as cell types that are bioenergetically compromised. The instrument also features pharma-tailored workflows for harnessing robust cellular metabolism measurements while handling suspension cell types, along with simplified automation enablement and analytical instrument qualification (AIQ). About Agilent Technologies Agilent Technologies Inc. is a global leader in the life sciences, diagnostics, and applied chemical markets, delivering insight and innovation that advance the quality of life. Agilent’s full range of solutions includes instruments, software, services, and expertise that provide trusted answers to our customers' most challenging questions. The company generated revenue of $6.32 billion in fiscal 2021 and employs 17,000 people worldwide.

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PHARMACY MARKET

CASI PHARMACEUTICALS ANNOUNCES CNCT19 (CD19 CAR-T) GRANTED ORPHAN DRUG DESIGNATION BY THE U.S. FDA

CASI Pharmaceuticals, Inc. | January 19, 2022

CASI Pharmaceuticals, Inc. a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, announced today, that the U.S. Food and Drug Administration as granted Orphan Drug Designation for CNCT19, an investigational CD-19 directed CAR-T therapy, for the treatment of patients with Acute Lymphoblastic Leukemia. CNCT19 is currently being developed independently by Juventas to meet the urgent clinical needs of patients with hematologic malignancies globally. The National Medical Products Administration has granted CTA approval for CNCT19 in two indications in Nov. 2019. Currently, the Phase II clinical trials of CNCT19 are in progress. Positive clinical data for adult and pediatric patients with relapsed/refractory B-ALL has been presented at the December 2021 ASH annual meeting, which further demonstrated its safety and efficacy profile. CNCT 19 is expected to be the first domestic CD19 directed CAR-T product in China with independent intellectual property rights. "Our partner Juventas continues to make encouraging progress in developing their CD19 CAR-T therapy. The Orphan Drug Designation, from the FDA, and the Breakthrough Designation status, granted by the China Center of Drug Evaluation (CDE) in December 2020, represent significant milestones that demonstrate our belief that CNCT19's commercialization will not only be successful in China, but potentially on a global scale. CASI has worldwide co-commercial rights of CNCT19, and will start the global commercialization process according to CNCT19's regulatory progress outside China." Dr. Wei-Wu He, CASI's Chairman, and CEO About Juventas Juventas is a biopharmaceutical company headquartered in China dedicated to the development and commercialization of cell therapies globally. Utilizing innovative and integrated technology platforms, the company has developed a diverse pipeline of cellular immunotherapies for treatment of hematological malignancies, solid tumors, and other non-oncological conditions both in China and globally. At present, the company is conducting two pivotal clinical trials of CNCT19 for treating adult r/r-B-ALL and r/r-B-NHL in China. CNCT19 has the potential to become the first launched domestically developed CD19 CAR-T therapy in China and the first CAR-T product for the treatment of adult R/R B-ALL in China. About CASI Pharmaceuticals CASI Pharmaceuticals, Inc. is a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products in China, the United States, and throughout the world. The Company is focused on acquiring, developing and commercializing products that augment its hematology oncology therapeutic focus as well as other areas of unmet medical need. The Company intends to execute its plan to become a leader by launching medicines in the greater China market leveraging the Company's China-based regulatory and commercial competencies and its global drug development expertise. The Company's operations in China are conducted through its wholly-owned subsidiary, CASI Pharmaceuticals (China) Co., Ltd., which is located in Beijing, China. The Company has built a commercial team of more than 100 hematology oncology sales and marketing specialists based in China.

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PHARMACY MARKET

Sunovion and Otsuka Initiate Phase 3 Clinical Development of Non-Racemic Amisulpride for the Treatment of People with Bipolar Depression

Sunovion Pharmaceuticals Inc. | February 10, 2022

Sunovion Pharmaceuticals Inc. and Otsuka Pharmaceutical Development And Commercialization, Inc. announced that the first patient has been randomized in SEP380-301, a Phase 3 clinical study evaluating non-racemic amisulpride for the treatment of major depressive episodes associated with bipolar I disorder (bipolar depression). Non-racemic amisulpride is being jointly developed and commercialized as part of a collaboration between Sunovion, its parent company Sumitomo Dainippon Pharma and Otsuka Pharmaceutical Co., Ltd. SEP380-301, named RELEASE 1 is a six-week, global, multicenter, randomized, double-blind, placebo-controlled, parallel-group study examining the efficacy, safety and tolerability of non-racemic amisulpride in adults with bipolar depression. Patients will be randomized to receive fixed-dose, non-racemic amisulpride or placebo. The primary endpoint for RELEASE 1 is the reduction of depressive symptoms, as measured by the change from baseline in Montgomery-Åsberg Depression Rating Scale (MADRS) total score, compared to placebo, after six weeks of treatment. Following participation in the six-week study period, patients who complete RELEASE 1 may enroll in RELEASE 3, a 12-month, open-label extension study assessing the long-term safety, tolerability and effectiveness of non-racemic amisulpride. “Major depressive episodes associated with bipolar I disorder have a profound impact on those living with the condition. Depressive episodes are associated with mood impairment, fatigue, loss of interest in activities or social situations and, in some cases, suicidality. We are encouraged by our Phase 2 clinical results and look forward to enhancing our understanding of non-racemic amisulpride and its potential to help address unmet needs in the treatment of bipolar depression.” Armin Szegedi, M.D., Ph.D., Senior Vice President, Chief Medical Officer at Sunovion “Randomizing the first patient in the RELEASE Phase 3 clinical development program is a significant step in our collaboration with Sunovion and our shared goal of expanding neuropsychiatric treatment options to patients living with serious mental conditions,” said John Kraus, M.D., Ph.D., Vice President, Clinical Development Therapeutic Head, CNS, Otsuka Pharmaceutical Development & Commercialization, Inc. “Through our collaboration with Sunovion, we hope to advance SEP-4199, which we believe shows promise for the treatment of people with bipolar depression.” About Non-Racemic Amisulpride (SEP-4199) Non-racemic amisulpride is in Phase 3 clinical development for the treatment of major depressive episodes associated with bipolar I disorder (bipolar depression). Non-racemic amisulpride was designed to enhance 5-HT7 receptor antagonist activity and reduce dopamine D2 receptor antagonist activity compared to racemic amisulpride. Lower D2 receptor occupancy associated with non-racemic amisulpride is intended to maintain overall effectiveness for patients with mood disorders, including bipolar depression, while reducing the burden of D2-related side effects, including movement-related symptoms, such as extrapyramidal symptoms and akathisia.1 About Bipolar Disorder Bipolar disorder affects approximately 12.6 million individuals in the United States and an estimated 29 million people worldwide.2,3 A person is usually diagnosed with bipolar disorder after experiencing at least one manic/hypomanic episode, with symptoms that are not better explained by another mental health condition, such as schizophrenia.4 Bipolar disorder is characterized by debilitating mood swings, interspersed with periods of stable mood and behavior.5 When individuals with bipolar disorder are experiencing symptoms, most tend to be depressed rather than manic.4 Bipolar disorder is associated with significant impairment in personal and professional relationships, quality of life and ability to function. About Sunovion Pharmaceuticals Inc. (Sunovion) Sunovion is a global biopharmaceutical company focused on the innovative application of science and medicine to help people with serious medical conditions. Sunovion’s vision is to lead the way to a healthier world. The company’s spirit of innovation is driven by the conviction that scientific excellence paired with meaningful advocacy and relevant education can improve lives. With patients at the center of everything it does, Sunovion has charted new paths to life-transforming treatments that reflect ongoing investments in research and development and an unwavering commitment to support people with psychiatric, neurological and respiratory conditions. Headquartered in Marlborough, Mass., Sunovion is an indirect, wholly-owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sunovion Pharmaceuticals Canada Inc., based in Mississauga, Ontario, is a wholly-owned direct subsidiary of Sunovion Pharmaceuticals Inc. About Otsuka Otsuka Pharmaceutical Co., Ltd. is a global healthcare company with the corporate philosophy: “Otsuka–people creating new products for better health worldwide.” Otsuka researches, develops, manufactures, and markets innovative products, with a focus on pharmaceutical products to meet unmet medical needs and nutraceutical products for the maintenance of everyday health. In pharmaceuticals, Otsuka is a leader in the challenging areas of mental, renal, and cardiovascular health and has additional research programs in oncology and on several under-addressed diseases including tuberculosis, a significant global public health issue. These commitments illustrate how Otsuka is a “big venture” company at heart, applying a youthful spirit of creativity in everything it does. Otsuka established a presence in the U.S. in 1973 and today its U.S. affiliates include Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC) and Otsuka America Pharmaceutical, Inc. (OAPI). These two companies’ 1,700 employees in the U.S. develop and commercialize medicines in the areas of mental health, nephrology, and cardiology, using cutting-edge technology to address unmet healthcare needs. OPDC and OAPI are indirect subsidiaries of Otsuka Pharmaceutical Company, Ltd., which is a subsidiary of Otsuka Holdings Co., Ltd. headquartered in Tokyo, Japan. The Otsuka group of companies employed 47,000 people worldwide and had consolidated sales of approximately USD 13.3 billion in 2020. About Sumitomo Dainippon Pharma Co., Ltd. Sumitomo Dainippon Pharma is among the top-10 listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China, and other Asian countries. Sumitomo Dainippon Pharma aims to create innovative pharmaceutical products in the Psychiatry & Neurology area, the Oncology area and Regenerative medicine/Cell therapy field, which have been designated as the focus therapeutic areas. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 7,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate

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