For biotech companies developing an immuno-oncology (IO) therapy, the complexity of oncology clinical trials takes on new dimensions.
Advances in translational science, new therapeutic platforms, unique mechanisms of action, and novel trial designs are just a few of the influences shaping the dynamic IO clinical trial space.
IO-focused biotechs can still employ traditional oncology clinical trial parameters and processes, but considerations distinct to IO studies present unique scientific and operational challenges for even the most seasoned biotech executives. This white paper reviews the current IO landscape and offers guidance for biotech companies preparing for IO studies. The following topics will be addressed:
- Protocol development and regulatory approvals
- Selecting sites, educating staff, and managing data
- Enrolling the right patients
- Planning for logistics and product production
- Measuring response
FLOURISHING IO SECTOR
Cancer is an insidious disease that can manifest as a collection of distinct molecularly defined pathways. While mutationally-targeted therapies have been effective in stemming certain cancers, treatment resistance is common via the cancer harnessing alternative pathways, often leading to recurrence with lethality. Rather than target specific genetic aberrations, attention has turned to harnessing the power of our own immune system, which can find and eliminate cancerous cells no matter the genetic makeup. As a result, a boom has emerged with immuno-oncology (IO) therapeutics, starting with the first US approval of ipilimumab in 2011 and two other checkpoint inhibitors in 2014. Since then the use of IO therapeutics and their development have grown exponentially, in many cases completely redefining cancer treatment paradigms.
Confidence in IO therapies, fueled by significantly enhanced response rates and overall survival as well as entry into newer markets like China1 , is anticipated to drive the global cancer IO market to an estimated $171 billion by 2028, almost half of the total oncology drug market. Investment in IO start-ups is robust, having surpassed $1 billion in total capital raised.
This enthusiasm expanded the global IO pipeline in several dimensions. From 2017 to 2018, the number of IO agents grew 67 percent to nearly 3,400, representing 62 percent preclinical and 38 percent clinical-stage agents. By mid-2019, nearly 3,000 clinical trials involving an IO agent were registered,4 reflecting the vigor of examining new and existing IO therapies alone or in combination with other IO or traditional cancer treatments.
The goal of an IO therapy is to stimulate or induce a tumor-specific immune reaction. Progress in cancer biology and translation into clinical programs has led to a field of more than 400 targets, up 50 percent from 2017. Currently, more than 500 IO therapies and next generation biotherapeutics in development explore at least 80 different mechanisms of action.5 Given the diversity of these agents, it is useful to discuss them as broad groups.