Investing in emerging new technologies early on can generate market-crushing gains over the long haul. The lucky few that saw the potential of tech stocks like Apple and Microsoft in their infancy, for instance, can attest to this fact:
Right now, the treatment of cancer is being revolutionized by so-called immuno-therapies that have the power to melt tumors away in some cases.
Bristol-Myers Squibb (BMY -0.27%) and Merck & Co. (MRK 0.10%) ushered in the era of this game-changing new technology last year with their respective PD-1 inhibitors Opdivo and Keytruda. But with the market for immuno-therapies expected to grow to a staggering $50 billion by 2030, there are numerous companies racing to develop their own immuno-oncology pipelines.
Bellicum Pharmaceuticals (BLCM) and Kite Pharma (NASDAQ: KITE) are two small-cap companies working on a powerful form of immuno-therapy called CAR T cells that could be worth tens of billions of dollars one day, for example.
CAR T cells are modified white blood cells that have the ability to recognize tumors through specific proteins they produce. In theory, these genetically modified cells could provide lifetime immunity against certain forms of cancer.
Unfortunately, CAR T cells have produced some serious unwanted side effects, such as inflammation and extremely high fevers, during clinical testing, which has greatly slowed their development.
These two biotechs, though, think their next-generation CAR T programs have now solved these earlier safety problems and may even have boosted the platform's cancer-fighting ability at the same time. So, let's take a deeper look at these companies, and consider if their stocks represent compelling buys at present.
Bellicum looks to improve the safety profile and potency of CAR T cells
Although Bellicum's pipeline is only now entering clinical trials, the company has one of the most compelling approaches toward improving CAR T safety and efficacy. Specifically, the tiny biotech has incorporated "molecular switches" into its cell lines that can be triggered by a small molecule known as rimiducid. These switches can be used to either enhance a response or "turn-off" the cells, if a patient is having an adverse reaction.
Source: Bellicum Pharmaceuticals
Bellicum is developing five product candidates in total (not just CAR T cells) that should enter early stage testing by mid-2016. The intriguing part is that the company is going after a wide diversity of indications such as a host of blood disorders, solid tumors, and skin cancer, among many others.
Unfortunately, Bellicum is going to require patience for investors. And lots of it. Based on my projections, the company probably won't have a pivotal late-stage trial under way until 2017, meaning that its first shot at gaining a regulatory approval won't come until 2018.
One potential wild card that could speed up this process, however, is if the biotech can garner a Fast Track designation along the way, leading to a rolling submission and perhaps an accelerated approval. This scenario isn't totally out of the question given that both Bristol-Myers and Merck's PD-1 inhibitors were able to exploit this route to reach the market faster than normal.
Kite Pharma is pushing hard to become a leader in the CAR T space
Although Kite Pharma only IPO'ed last year, its stock has bolted higher based on the early success of its lead clinical candidate, KTE-C19, indicated as a therapy for aggressive non-Hodgkin's lymphoma.
Specifically, a small early stage study showed that the immunotherapy led to a 61% complete remission rate in patients with advanced B-cell malignancies. Moreover, every patient in the trial exhibited at least some form of clinical benefit in the form of either stable disease or partial remission. The company thus plans on pushing KTE-C19 into a midstage clinical trial soon, and hopefully launching the drug via an accelerated approval by 2017.
Kite has also greatly solidified its foundation by signing a lucrative research agreement with Amgen to develop additional immuno-oncology candidates to kick off the year, and by snapping up privately held T-Cell Factory B.V. for roughly $21 million. This latter business move gives the biotech a footprint in Europe, where it plans on expanding its operations moving forward.
Are these two cancer immunotherapies strong buys right now?
A quick look at these two stocks might leave you with the impression that they are speculative, high-risk biotechs, especially in light of how little clinical data they've generated thus far. But I think they have all the makings of future stars in the emerging field of immuno-therapy, and are therefore worth consideration by risk-tolerant investors.
My reasoning is based on the fact that CAR T cells could become a major leap forward in the treatment of a range of blood disorders, and may even have a place, as a part of a combo therapeutic approach, for solid tumors as well. Topping it off, cancer drugmakers, in general, tend to garner mind-boggling premiums that are resistant against downturns in the broader market.
That's why top hedge funds like Baker Brothers and Soros Fund Management have taken a keen interest in these stocks, as well as some of their peers.
Overall, I think both of these companies have a decent shot at producing mind-boggling gains as their immuno-therapy pipelines begin to produce data, making now a good time to take a deeper dive.
