Following the release of interim results from a mid stage trial, Kite Pharma, Inc. (NASDAQ: KITE) appears on track to become the first company to launch a chimeric antigen receptor T-cell, or CAR-T, therapy. If so, then the impressive efficacy observed in this trial may revolutionize how doctors tackle tough-to-treat lymphomas.
Delivering new hope
Diffuse large B-cell lymphoma (DLBCL) is the most common form of non-Hodgkin lymphoma (NHL). DLBCL represents about 30% of NHL cases and, unfortunately, there are limited treatment options for patients who fail to respond to chemotherapy or see their disease return following treatment. Sadly, about 30 people with advanced chemo-refractory DLBCL pass away every day.
In a bid to offer DLBCL patients new hope, Kite Pharma developed KTE-C19, a CAR-T therapy that engineers a patient's T-cells so that they can identify and bind to the CD19 protein that's commonly expressed in B-cell cancers.
Today, Kite Pharma reported that tough-to-treat patients participating in their Zuma-1 study saw an impressive level of response to KTC-C19. Specifically, an interim look at response rates in 51 patients with DLBCL shows that 33% of patients had a complete response at the three-month mark. That's significant because, historically, complete responders at the three-month mark tend to remain complete responders.
Management also reported that KTE-C19's was effective in 11 patients with two less common forms of NHL: transformed follicular lymphoma (TFL) and primary mediastinal B-cell lymphoma (PMBCL). The complete response rate observed at the three-month mark for these patients was 64%.
Overall, complete response rates for all patients participating in the study was 39%. For perspective, the complete response rate at the three-, six-, and nine-month mark for the seven patients who participated in KTE-C19's previous phase 1 trial was 43%.
Those complete response rates are remarkable given how sick patients participating in this study were. According to Kite Pharma, the median overall survival for chemorefractory advanced DLBCL patients is about six months, and up until now, the complete response rates have been just 8%.
There were two patient deaths in the phase 2 trial that are being attributed to cytokine release syndrome (CRS), which results from T-cell over activity. However, patient deaths aren't uncommon in trials involving this patient population and CRS was manageable in most patients.
Today's results were "interim" and additional information regarding the trial is planned for release at an upcoming conference. Management expects to sit down with the Food and Drug Administration soon to discuss KTE-C19's interim results.
The FDA may allow Kite Pharma to file for approval of KTE-C19 prior to having the complete results in hand early next year -- or the former may tell management to wait until it has all the data prior to filing. If the FDA allows a filing with the interim data, then it's possible that KTE-C19 becomes commercially available by mid-2017. If not, then a mid-year FDA filing for approval could push back a decision until late next year or early in 2018.
Kite Pharma is ramping up its production capacity ahead of an expected regulatory green light, and because of those efforts, management believes it can craft enough KTE-C19 to support 5,000 patients in year one. It hasn't hinted at how much KTE-C19 will cost, but recently approved cancer drugs commonly carry six-figure annual price tags.
Overall, 26,000 people will be diagnosed with DLBCL in the United States in 2016 and thousands more will be diagnosed with DLBCL in Europe. The company's potential to address the toughest-to-treat cases in both of these markets is significant. Since there's a massive need for new, life-extending drugs for these patients, I think KTE-C19 could justify a valuation that's much higher than Kite Pharma's current $3 billion market cap.