Understanding the CAR-T opportunity
Sponsored by: GlobalData & BioPharm Insight
- Hematology Oncology
- Drug Development
- Car T
Date: 28 September
Time: 4PM London
Development and commercial complexities of the cutting-edge therapy
In recent years, hematology-oncology drug development has been abuzz with the potential of chimeric antigen receptor T-cell (CAR-T) therapies. That potential became a reality on 30 August when the FDA gave its first approval to a CAR-T therapy, Novartis' Kymriah (tisagenlecleucel), for acute lymphoblastic leukemia (ALL) in children and young adults. The approval followed a unanimously favorable 12 July vote by the FDA Oncologic Drugs Advisory Committee (ODAC) and happened well ahead of the 3 October approval verdict date the agency had set. Novartis' stock rose 2% on the news amid significant industry, investor and media excitement. Also nearing approval is Kite Pharma's axicabtagene ciloleucel (or "axi-cel") for aggressive non-Hodgkin's lymphoma (NHL), as Kite said in August that the FDA will not hold an ODAC meeting for the drug.
Yet, beyond the usual talk of dazzling efficacy and blockbuster potential, BioPharm Insight (BPI) looks at some of the lingering issues and expert deliberations on how these transformational CAR-Ts will be both accepted and logistically implemented in the clinic. Besides Novartis and Kite, the other major players are Juno Therapeutics and bluebird bio, but other big oncology companies are watching eagerly to see how these new therapeutics will pan out commercially following accelerated development and potential approvals.
BPI data shows the global combined sales of Kite's axi-cel, Juno's JCAR017 and Novartis' tisagenlecleucel-T are expected to reach more than USD 2.6bn in 2022. Diseases that these CAR-T therapies are targeting include pediatric and adult ALL, diffuse large B-cell lymphoma, multiple myeloma and chronic lymphocytic leukemia. The field is also slowly branching into solid tumors like glioblastoma, though these are much further behind in development.
BioPham Insight coverage dives into the debate among clinical trial investigators and other Key Opinion Leaders (KOLs) on durability of responses in specific indications along with questions on relapse rates after infusion. With such expedited development paths, experts point to many unknowns on how efficacy may play out in the clinic. Meanwhile, cases of deaths in patients and safety events like cerebral edema, have also placed a cloud on the therapies' safety profiles, and experts have discussed the potential for these events to be a class effect versus specific to the competing CAR-T constructs. Manufacturing is also an anxiety when it comes to scaling up for a commercial launch, as sources have told BPI of manufacturing issues during trials that in turn have led to logistical issues with administration to patients. Lastly, as a new, highly innovative and transformational therapy, reimbursement experts question potential pricing models, which may come under heavy scrutiny in front of payers post launch. Lastly, as a new, highly innovative and transformational therapy, reimbursement experts question potential pricing models, which may come under heavy scrutiny in front of payers post launch. Novartis announced the therapy would cost USD 475,000, which does not include ancillary costs -- including hospitalization and the use of Roche's Actemra to treat cytokine release syndrome (CRS) -- and cost benefit discussions are likely to intensify.
In this webinar, BioPharm Insight's senior reporter Alaric DeArment will share insights from his coverage on the CAR-T space, in particular what he has been hearing from his sources on ongoing clinical trials and how experts and KOLs view the future development and market potential of these CAR-Ts. North America Editor Surani Fernando will moderate the presentation followed by a Q&A session. Join this webinar to gain exclusive insights into the CAR-T development, manufacturing and commercial space, and learn how these novel therapeutics might impact future clinical trials and the cancer treatment paradigm.
Editor, North America
Surani Fernando manages the North America editorial desk of BioPharm Insight. Surani first joined BioPharm Insight's London office as a drug development reporter in 2010 and has generated a number of market-moving scoops and in-depth analyses on key therapeutic areas. She has also reported on drug reimbursement, regulatory affairs, patent litigation, M&A and business financing. Surani has covered academic and industry meetings in both Europe and the US, where she has interviewed key opinion leaders and C-level executives and has also made TV appearances on CNBC and Sky News as an industry commentator. In November 2014, Surani became the Editor of BPI’s European new desk and moved to New York in September 2016 to lead the US team. Prior to her career in journalism she worked in clinical operations at CRO PAREXEL. She has a Bachelor of Medical Science and Master of Health Communication, both from Sydney University, Australia.
Senior Reporter, North America
Alaric DeArment joined in March 2014 as a reporter primarily focusing on hematology oncology indications. In addition to analysis of clinical trials, regulatory issues, market uptake and pricing and reimbursement, he has broken news on material drug developments and provided coverage from major medical conferences including ASH, ASCO, EHA and others. In 2016, he moderated a panel discussion at the Clinical Trials Innovation Programme in Miami and was also awarded a fellowship with the Association of Health Care Journalism Comparative Effectiveness Research in Washington. Alaric previously covered prescription drugs as associate editor of Drug Store News, from August 2008 until January 2013. He has a bachelor degree in journalism from Ball State University. A native of Seattle, he also lived in China from September 2001 until September 2004.
Key Learning Objectives
- Explore differences in duration of response and relapse rates with different therapies
- Understand the context of safety and death concerns
- Gain insight into the manufacturing challenges experienced during trials
- Develop a forward-looking understanding of pricing dilemmas to come
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