By Michael Quigley, VP of Investor Research, LSN
Due to a recent FDA decision, immuno-oncology has proven to be a key area for personalized medicine approaches. In this article, Next Phase will explore what this could mean for new technology development.
Many currently marketed cancer immunotherapies struggle due to high toxicity and response patterns that differ when compared to traditional therapies (1). Both in a research and clinical setting, identifying those patients most likely to respond to these and other new therapeutic approaches represents a massive opportunity for pharma companies. This has given rise to increasing interest and investment in the field of companion diagnostics.
In May, the FDA approved Keytruda as the first immunotherapy cancer drug based on tumor specific molecular characteristics. While there have already been such approvals for genetic based markers, the FDA’s approval of Keytruda based on tumor specific characteristics offers shows the regulatory environment’s positive sentiment on other personalized approaches as well. This is the first of such successes of a long developed shift away from the more traditional organ based approvals/research for cancer therapeutics.
While this shows obvious promise for large pharma companies developing these types of therapeutics it also shines a very positive light on companies looking at innovative ways of profiling tumor and patient subtypes. These include including sequence-based diagnostics, multiple gene and protein signatures, multiplexed and digital PCR, microfluidic systems and novel signal detection approaches among others. These methods for identifying predictive biomarkers and patient stratification stand to move the field of immunotherapy forward, away from its current toxicity and efficacy issues and into an era of more personalized medicine.
Looking farther into the future implications of this approval is another positive sign into the early research current being done into what is known as “theranostics”. Where companion diagnostics are generally developed for an existing therapeutic agent the development of theranostics is a process involving the co-creation of therapeutics and a diagnostic to identify which patients will respond most positively. This is the next frontier for personalized medicine, where therapeutics are designed inherently to be effective in sub-populations of a disease or condition. Though this approach would potentially shrink the potential market for a new drug rather than aiming for “blockbuster” status, it also presents the opportunity to dramatically improve research and clinical trial efficiency for these compounds.
Overall this latest Keytruda approval provides a signal that the FDA is on board with this trend towards personalized medicine that researchers and drug developers have been moving towards for years. In an industry that is so filled with risk and uncertainty, any form of reassurance from the regulatory environment is always welcome, and investors may see this as a positive signal for the sector.
Congratulations on a great RESI meeting in San Diego last week. I have been attending biomedical conferences for longer than I’d like to admit – ABC, ACA, ACOG, ASA, ASCO, BIO, DMD, H&Q, JPM, MnM, PhRMA, and many others, some whose acronyms are slowly being lost to history.
Congratulations on running yet another successful RESI conference! I have attended several over the past 3 years and they just keep getting better. The partnering system makes it easy to identify leads and I always have a packed meeting schedule with potential investors and partners that I could not have otherwise found through the usual channels. While I did not see immediate success, you and I both know this is, in part, a numbers game and persistence pays. It has taken a lot of work, but this past March I closed with one investor group and in July I will close with another group that has committed to invest. From the recent San Diego conference, I have several new high-quality investor leads and partnering opportunities to follow-up on in the coming weeks. Given the enthusiastic response I received to Curtana’s story, I anticipate that we will continue to benefit from the relationships we have made at the RESI conferences.
Attending numerous RESI from a few years back, I can say that the events are getting increasingly better and a must go to event to meet quality companies. Attendees from the investor side has also been increasing in quantity and quality, almost feeling like a 1:1 attendee ratio. The partnering system is intuitive to use and makes identifying promising companies to meet quite easy. From the event I have met many new business relationships that I expect will flourish in not only in the business setting but also as friends. Most importantly the follow-up rate from meetings held at RESI is also quite high, proving to me that the quality and dedication of these companies are certainly a par above. The RESI team has always been helpful in addressing any concerns and helping build new leads. I am certain that we will be attending future RESI events and look forward to seeing RESI flourish. Congrats to the RESI team on making another great event!
At the outset of your life science startup company, you may have great technology and a well-thought-out business plan, however oftentimes your company ends up looking much different than you had imagined. The long development timelines and capital-intensive nature of the life science sector usually doesn’t allow for much in terms of testing product/market fit, meaning these startups had better have done extensive research on their target market – various stakeholders, investor sentiment, market dynamics, end user and patient preferences, regulatory environment for target indication, and other important factors. However, at times even the most prepared company may encounter the need to pivot.
