Novartis' SMA gene therapy would not be cost-effective if priced over $1.5M: ICER

FiercePharma | April 04, 2019

Novartis' SMA gene therapy would not be cost-effective if priced over $1.5M: ICER
The pricing analysis done by the Institute for Clinical and Economic Research (ICER) on new therapies for spinal muscular atrophy (SMA) has been no small undertaking. One of the therapies ICER reviewed—Zolgensma from Novartis’ AveXis unit—isn't yet FDA-approved or priced, forcing the influential agency to assess it using hypothetical price ranges. Now, after considering input from Novartis, ICER has released its final word on the subject—and it hasn't changed. The agency stood by its initial assessment, saying a price of $900,000 or so would make the most sense, but it definitely shouldn't carry a price higher than $1.5 million. The FDA is expected to decide on Zolgensma in May, and Novartis will reveal its pricing plans then. But Dave Lennon, president of AveXis, has already had plenty to say on the subject. Lennon, who argues that gene therapy cures need an entirely different type of evaluation, created quite a stir last year when he said the gene therapy would be cost-effective at $4 million to $5 million.

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Hemogenyx Pharmaceuticals and Selexis SA Will Advance Hemogenyx’s Acute Myeloid Leukemia (AML) CDX Bispecific Antibody to Human Trials

Hemogenyx Pharmaceuticals plc | January 13, 2022

Hemogenyx Pharmaceuticals plc the biopharmaceutical group developing new therapies and treatments for blood diseases, and Selexis SA, a JSR Life Sciences company, have signed a service agreement to develop the cell line for Hemogenyx’s CDX bispecific antibody for the treatment of acute myeloid leukemia (AML). Under the agreement, Hemogenyx will leverage Selexis’ proprietary SUREtechnology Platform™, a suite of cell line development tools and technologies that significantly reduces the time, effort, and costs associated with developing high-performance mammalian cell lines. The CDX bispecific is made using the Hemogenyx’s proprietary humanised monoclonal antibody against a target on the surface of AML cells. CDX was co-developed by Hemogenyx Pharmaceuticals and Eli Lilly and Company (“Lilly”). This cutting-edge application of immune therapy offers a potentially more benign and effective form of treatment that, if successful, could have a significant impact on treatment and survival rates for AML. CDX is planned to be the Company’s second therapeutic candidate to enter clinical trials. Following the completion of the co-development phase, Lilly granted the Company an exclusive worldwide license to certain intellectual property developed by Lilly related to the CDX bispecific antibody for all uses, including the treatment of AML and other blood cancers. “We are delighted to partner with Selexis, and access its proprietary protein expression tools and technologies, IP and know-how. The partnership is key to advancing our CDX programme into clinical trials and accelerating the timeline to deliver this innovative therapy to patients in need of a more benign and effective treatment for AML.” Dr. Vladislav Sandler, Chief Executive Officer and co-founder of Hemogenyx Pharmaceuticals Mr. Dirk Lange, CEO of Selexis, added, “There’s an urgent need for effective treatments for AML, and we at Selexis are pleased to apply our technologies to help Hemogenyx advance the CDX bispecific antibody to the clinic. We’ve built a reputation for delivering cell lines rapidly and cost-effectively, without compromising safety. This is an exciting milestone for Hemogenyx and we welcome the opportunity to join the company on its journey toward delivering a promising and effective therapy for patients with AML.” Selexis’ modular SUREtechnology Platform™ facilitates the rapid, stable, and cost-effective production of recombinant proteins and vaccines, providing seamless integration of the development continuum from discovery to commercialization. About Hemogenyx Pharmaceuticals plc Hemogenyx Pharmaceuticals is a publicly traded company headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility. The Company is a pre-clinical stage biopharmaceutical group developing new medicines and treatments to treat blood and autoimmune disease and to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development. For more than 50 years, bone marrow transplantation has been used to save the lives of patients suffering from blood diseases. The risks of toxicity and death that are associated with bone marrow transplantation, however, have meant that the procedure is restricted to use only as a last resort. The Company’s technology has the potential to enable many more patients suffering from devastating blood diseases such as leukemia and lymphoma, as well as severe autoimmune diseases such as multiple sclerosis, aplastic anemia and systemic lupus erythematosus (Lupus), to benefit from bone marrow transplantation. About Selexis SA Selexis SA, a JSR Life Sciences company, is the global leader in cell line development with best-in-class modular technology and highly specialized solutions that enable the life sciences industry to rapidly discover, develop and commercialize innovative medicines and vaccines. Our global partners are utilizing Selexis technologies to advance more than 146 drug candidates in preclinical and clinical development and the manufacture of eight commercial products. As part of a comprehensive drug development process, the Company’s technologies shorten development timelines and reduce manufacturing risks.

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Cullinan Oncology Forms Strategic Collaboration Agreement with Mount Sinai to Advance Oral HPK1 Protein Degrader for Cancer Treatment

Cullinan Oncology, Inc. | January 10, 2022

Cullinan Oncology, Inc. a biopharmaceutical company focused on developing a diversified pipeline of targeted therapies for cancer patients, today announced the Company has entered into a collaboration agreement with the Icahn School of Medicine at Mount Sinai to develop novel small molecule immune modulators.​ This exclusive option and multi-year collaboration agreement will be focused on the optimization and development of oral protein degraders targeting hematopoietic progenitor kinase 1 (HPK1), a key regulator of immune cell activation and a high-priority target in immune-oncology. The collaboration aims to accelerate the development of novel, best-in-class HPK1 degraders that stimulate robust anti-tumor immunity. The research will be conducted by leading scientists at both Cullinan and Icahn Mount Sinai. Cullinan will fund the collaboration and has an exclusive option to license the intellectual property for further development and commercialization. The Icahn Mount Sinai team will be co-led by Steven J. Burakoff, M.D., Lillian and Henry M. Stratton Professor of Cancer Medicine, Dean for Cancer Innovation, and Chief, Pediatric Oncology at Icahn Mount Sinai, and Jian Jin, Ph.D., Mount Sinai Professor in Therapeutics Discovery and Director, Mount Sinai Center for Therapeutics Discovery, at Icahn Mount Sinai. “Our team is excited to work with the talented and experienced cancer drug developers at Cullinan. Our research has already demonstrated that a degrader approach to targeting HPK1 may control tumor growth more effectively compared to simply inhibiting HPK1 kinase activity. We believe this collaboration will help us identify novel, differentiated oral HPK1 degraders and can bring future benefit to cancer patients worldwide.” Dr. Jin “We are proud to partner with Icahn Mount Sinai, which is at the forefront of cancer research and patient care,” said Leigh Zawel, Chief Scientific Officer, Small Molecules, of Cullinan Oncology. “We are very excited to build on the synergy between the deep scientific expertise of the Icahn Mount Sinai team and the strong drug development capabilities at Cullinan to advance therapies that have the potential to significantly improve the lives of patients with cancer. We look forward to a fruitful collaboration in this novel area of science.” About Cullinan Oncology Cullinan Oncology is a biopharmaceutical company that is developing a diversified pipeline of targeted therapeutic candidates across multiple modalities in order to bring important medicines to cancer patients. The Company’s strategy is to source innovation through both internal discovery efforts and external collaborations, focusing on advanced stage assets with novel technology platforms and differentiated mechanisms.

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Stallergenes Greer Strengthens Precision Medicine Approach in Allergen Immunotherapy With Imperial College London Research Collaboration

Stallergenes Greer | January 17, 2022

Stallergenes Greer, a global healthcare company specialising in allergen immunotherapy (AIT), today announced that the company has entered into a research collaboration on the discovery of biomarkers of AIT efficacy with Imperial College London, a global top ten university with a world-class reputation in science, engineering, business and medicine. This long-term research collaboration, which combines the long-standing expertise of Stallergenes Greer in AIT with the prestigious research capabilities of Imperial College London, aims to push the boundaries of allergic patient care by identifying biomarkers of AIT efficacy and expanding the knowledge of the pathophysiology of allergic diseases and their treatment with AIT. “We are thrilled about this collaboration with Imperial College London which will contribute to both deciphering the mode of action of AIT and identifying key markers that can be used in the routine practice of allergology for the benefit of patients and healthcare practitioners. Based on the understanding of specific phenotypes and the responses of patients with allergies, allergologists will be able to tailor treatment modalities with the right dosing at the right time for each individual. This underscores our commitment to advancing precision medicine for allergy sufferers”, Amer Jaber, Executive VP Operations Europe and International and President of Stallergenes SAS “This research collaboration is a remarkable opportunity for a fruitful partnership between Imperial College London, a pioneer and world-leading university in the biomarker field, and Stallergenes Greer. It will build on the recent advances of biomarker research to further develop precision medicine in AIT for the benefit of patients with allergic diseases,” said Dr Mohamed Shamji, Head of the Immunomodulation and Tolerance Group, Imperial College London. The results of this research collaboration will be shared in international congresses and peer review publications. About Allergen Immunotherapy Allergies are the most prevalent and fastest growing chronic diseases in the industrialised world, affecting over one billion people worldwide. Allergen immunotherapy uniquely alters the natural course of respiratory allergies. It is the only therapeutic class capable of modifying disease progression and potentially preventing the onset of the disease by inducing tolerance in the immune system. About Stallergenes Greer Headquartered in London (UK), Stallergenes Greer Ltd is a global healthcare company specialising in the diagnosis and treatment of allergies through the development and commercialisation of allergen immunotherapy products and services. Stallergenes Greer Ltd is the parent company of Greer Laboratories, Inc. About Imperial College London Imperial College London is one of the world's leading universities. The College's 20,000 students and 8,000 staff are working to solve the biggest challenges in science, medicine, engineering, and business. Imperial is University of the Year 2022 in the Times and Sunday Times Good University Guide. It is the world’s fifth most international university, according to Times Higher Education, with academic ties to more than 150 countries. Reuters named the College as the UK's most innovative university because of its exceptional entrepreneurial culture and ties to industry.

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OPTEL ACQUIRES PHARMA TRACK-AND-TRACE UNIT OF KÖRBER

OPTEL Group | January 12, 2022

OPTEL GROUP, already a world leader in supply chain traceability, is expanding its expertise and global reach with the recent acquisition of the track-and-trace unit of the Körber Business Area Pharma, formerly known as Traxeed, based in Germany. Körber is a leading international technology group. The acquisition, announced today, will allow OPTEL to leverage the unit's expertise in pharmaceutical and agrochemical track-and-trace technologies, provide the company with a second foothold in the European marketplace and contribute to its diversification plans. "Creating a strong presence in the German market is key to the success of our strategic objectives because Germany is a well-established manufacturing hub, with a strong history of developing innovative manufacturing efficiency solutions and Industry 4.0 technologies," Louis Roy, founder and president of OPTEL "We are pleased that we have found a solid and future-oriented new owner with a broad international market access, from whose global network and years of experience in the highly specialized track-and-trace sector customers will clearly benefit. We are furthermore convinced that the new strategic owner can open up attractive development opportunities for the team," said Dr. Jürgen Krebs, Chief Technology Officer and Chief Operating Officer in the Körber Business Area Pharma. The acquisition allows OPTEL to welcome highly skilled pharma track-and-trace experts from Körber, complementing OPTEL's existing strengths in traceability, vision technologies, software and hardware. Furthermore, it will solidify and expand the company's presence in the European market by providing a second base of operations after Limerick, Ireland. In addition to the acquisition, a close collaboration between OPTEL and Körber's Business Area Pharma is planned in the future. The two companies plan to sign a partnership agreement whereby OPTEL becomes Körber's preferred partner for track-and-trace solutions for the pharmaceutical industry. Since its founding in 1989, OPTEL has become the world's foremost provider of track-and-trace solutions to the pharmaceutical industry. Its acquisitions in recent years have positioned it as the only company with the ability to provide complete, full-stack supply chain traceability. The Canadian multinational has since diversified into other industries, including consumer-packaged goods, metals and minerals, and agrochemicals. The acquisition of the track-and-trace unit of the Körber Business Area Pharma is expected to accelerate further expansion. ABOUT OPTEL OPTEL is a leading global provider of traceability systems whose goal is to use its innovative technologies to build a sustainable world through the Intelligent Supply Chain. OPTEL is the only company with the ability to offer complete end-to-end traceability, providing granular data at every step of the supply chain – from raw materials to the consumer and beyond. Founded in 1989, OPTEL is a Certified B Corporation headquartered in Canada, with facilities in Ireland, India and Brazil, as well as employees worldwide. ABOUT KÖRBER We are Körber – an international technology group with about 10,000 employees, more than 100 locations worldwide and a common goal: We turn entrepreneurial thinking into customer success and shape the technological change. In the Business Areas Digital, Pharma, Supply Chain, Tissue and Tobacco, we offer products, solutions and services that inspire. We act fast to customer needs, we execute ideas seamlessly, and with our innovations we create added value for our customers. In doing so, we are increasingly building on ecosystems that solve the challenges of today and tomorrow. Körber AG is the holding company of the Körber Group.

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