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lost its legal battle in 1997 and was effectively put out of business. The judge in the legal trial accused CellPro’s outside counsel of rendering “deficient” patent opinions and deemed its efforts “a weak pass at quality work.”
2) The technology might not work. Data produced to date look remarkably promising, but the number of patients treated is limited, as are the disease types. Specifically targeting cancer cells remains a tricky business, no matter the approach, because identifying unique molecules on those cells is challenging. Side effects have been significant in a number of cases using T cell-based therapies (a result of the large-scale death of tumor cells), but ways are being found to mitigate them.
3) Manufacturing costs may be so high so that it could be difficult to sell the drug for a profit. Dendreon was forced into bankruptcy by a huge upcoming debt payment, but putting that aside, it appears that it was unable to sell sipuleucel-T at a profit, even with a $93,000 price tag. A new generation of effective, personalized immunotherapy treatments will likely cost much more than that.
4) Pricing pressure and reimbursement by payers. The next wave of personalized immunotherapy treatments is going to be very expensive. This is going to put increased pressure on payers, who will be watching how the specialty drug market (i.e. drugs for rare diseases or small subsets of serious diseases like cancer) is taking up an increasingly large percentage of healthcare dollars. How will the drug get paid for? Ultimately, what gets paid will be tied to just how efficacious the treatments turn out to be.
5) New technology or medicines may render their approach obsolete. Any oral treatment, for example, that produced similar clinical results would present a serious challenge to Juno and its competitors’ business plans. Similarly, having an “off-the-shelf” immunotherapy treatment that didn’t have to be custom-made for each patient would be a huge step forward over individualized treatments. The field is still in its early days, and we don’t know how these things will sort themselves out.
6) Potentially limited patient populations. Rare disease companies (e.g. Genzyme, Alexion Pharmaceuticals, Shire) have shown that you don’t need to have large patient populations to run a profitable and successful business. Having said that, all of their patients get the exact same treatments, which is not the case for these immunotherapy approaches. The American Cancer Society estimates that there were 1,665,540 cases of cancer diagnosed in the U.S. in 2014.
However, many of the immunotherapy companies are targeting (at least initially) the same small cancer subsets. Juno, Kite Pharma (NASDAQ: KITE), UPenn/Novartis, Bellicum Pharmaceuticals (NASDAQ: BLCM), and Cellectis are all targeting patients with acute lymphocytic leukemia (about 6,000 new cases in the U.S. diagnosed each year) and chronic lymphocytic leukemia (14,000 new cases per year). Efficacy of their treatments has been demonstrated mostly in patients with liquid tumors. This could leave a relatively small market to be carved up by a number of companies with somewhat similar technologies if this treatment approach does not expand to new areas. Most of the immunotherapy groups are also looking ahead to clinical trials in solid tumors, including glioblastoma, sarcoma, breast, ovarian, non-small cell lung, prostate, and colorectal cancers. Successfully treating these other cancer types would provide a much larger opportunity for all of these organizations.
7) Keeping egos and hubris in check. Raising lots of venture capital does not guarantee success. Another Seattle cancer immunotherapy company, Xcyte, raised $150 million during the go-go 90s to advance its T cell-based cancer therapy. Xcyte, too, was led by a number of highly regarded industry veterans. Unfortunately, it wound up shutting down operations after nine years in 2005 and selling off its core technology for only $5 million.
We can look back at Dendreon’s (and Xcyte’s) mistakes and think that by recognizing them, the same errors won’t be made again. That’s probably true, but new challenges will arise that will test the leadership of immunotherapy dilettantes and veterans. This includes those folks who were previously employed at Dendreon, Xcyte, and other (now defunct) companies when things were going wrong. It’s worth noting that Juno, specifically, has two former Dendreon executives in senior roles (Hans Bishop, its CEO, was formerly Dendreon’s COO and Mark Frohlich, Juno’s executive VP of R&D, held this same title at Dendreon and was also its chief medical officer). How the executives at each company handle these problems when they crop up will separate the winners (who will need to be both smart and humble) from the losers. As Admiral Hyram Rickover once put it, “It is necessary for us to learn from others’ mistakes. You will not live long enough to make them all yourself.”
Ultimately, the effectiveness of the drug (its “value,” in current industry parlance), the cost to make it, and the price that can be charged will be key factors in the success or failure of all of these immunotherapy companies. As Dendreon fades into history and is laid to rest in the Seattle biotech graveyard, we should see that it gets a proper burial. Let’s give it a three-infusion salute as it is laid to rest. Dendreon’s lasting legacy will be as a true pioneer in cancer immunotherapy. The path it took was not an easy one, having to step over the bleached bones of innumerable failed studies accumulated over a century of treatments designed to boost the power of the immune system.
Dendreon’s initial success in winning FDA approval for sipuleucel-T undoubtedly paved the way for a large increase in venture investing in numerous immunotherapy companies. Despite Dendreon’s missteps and untimely demise, those who follow in its footsteps will hopefully learn from its mistakes, and emerge triumphantly from the large shadow it cast.
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