A large portion of the volatility that we are seeing in the biotech sector right now is rooted in the release of the set of abstracts for the upcoming American Society of Hematology (ASH) Annual Meeting. The meeting takes place in December (in this instance, December 9-12, 2017, in Atlanta, Georgia) and is a major event on the health care roster. Just as is the case with most of these major events, the abstracts hit press a few weeks ahead of the event, giving markets an opportunity to digest the presentations (to a certain degree, the abstracts don’t offer up 100% of the detail) and, in turn, to reflect on the implications of the updates in the market capitalizations of the companies in question.
So, of the recent abstract releases, which are the biggest in terms of impact on the share price of the companies that have released them?
One of the major movers this week is Juno Therapeutics, Inc. (NASDAQ:JUNO).
The company is going to be presenting a grand total of 15 different abstracts at the upcoming event, with a host of different target indications up for review. What markets are most looking forward to, however, and the presentation that will no doubt draw the most attention as and when it comes around, is that relating to a study called TRANSCEND.
The company set the study up to demonstrate the safety and efficacy of an asset called JCAR017 in patients with relapsed or refractory (r/r) aggressive B-cell non-Hodgkin lymphoma (NHL). The study includes patients with DLBCL and that represent a highly refractory population based on some key factors that are associated with a poor prognosis including an older age, having a double or triple hit, and being chemorefractory.
So, why are the markets so excited about this presentation?
Well, because the upcoming results will show that the drug looks incredibly strong in its target indication.
As per the presentation abstract, the study managed to show a 3-month overall response rate (ORR) of 80% (12/15) and a 3-month complete response (CR) rate of 73% (11/15) in the core group. Data support a dose-response relationship. Dose level 1 (DL1 = 50 million cells) showed 3-month ORR of 52% (11/21) and a 3 month CR rate of 33% (7/21).
That’s pretty impressive, especially in an indication like DLBCL, which is really tough to treat in this refractory population.
Further, across both doses in the core group, the best overall response was 84% (41/49) and the best overall CR rate was 61% (30/49).
Additionally, from a safety perspective, things look relatively clean. The most common treatment-emergent adverse events other than CRS and NT that occurred at ≥25% in the full group included neutropenia (41%), fatigue (30%), thrombocytopenia (30%), and anemia (26%). It’s important to recognize that these sort of safety issues may seem severe in other indications but when the target indication is cancer, the picture changes somewhat.
Why? Because the opportunity cost of not taking the treatment that brings about the side effects is generally death. That’s why people are willing to accept the side effects associated with chemotherapy treatment, which, as is widely publicized and well understood, can be completely debilitating for patients.
The FDA takes this into consideration when it analyzes a regulatory application from a risk-benefit perspective and this means that a drug gets a little more wiggle room on the safety side of things when it comes to PDUFA, assuming it has been shown to be effective in its target population. This leeway amplifies somewhat if the population in question doesn’t already have access to any degree of substantially effective treatment options, as is the case here.
So what’s next?
Subsequent to the presentation, the assumption is that Juno will use this data to underpin a regulatory submission to the FDA in the US. The trial was billed as pivotal, which suggests that the company doesn’t feel it needs to collect any more data to move forward with its submission and, in turn, to warrant a solid approval.
We’ll probably see an NDA submitted to the agency in the US before the end of the year, which sets the drug up for an approval during the third quarter of 2018, perhaps slightly later.