does business, having Shkreli to rail against all year could be, well, just what the doctor ordered.
All right, so I predicted in January that drug pricing would remain a hot topic all year. Big whoop. What about my other big questions for 2015? Let’s have a look.
—“What happens when cell-based cancer immunotherapy has its first big setback?” I asked that question as the immunotherapy frenzy was helping the entire biotech sector make its best bull run ever. Since then, the markets have deflated. Juno Therapeutics (NASDAQ: [[ticker:JUNO]]), Kite Pharma (NASDAQ: [[ticker:KITE]]), and Novartis, whose clinical programs for a form of cell therapy known as CAR-T had already shown early promise, made more progress in 2015. Novartis was the first to report Phase 2 data from a CAR-T program, for example. There were no big setbacks, but there was acknowledgment that patients with blood-borne cancers will likely do better with an extra dose of chemotherapy before receiving CAR-T therapy. Other obstacles for CAR-T, such as large scale manufacturing or making the leap to treat solid tumors such as breast, lung, and ovarian cancers, have yet to be solved, as my colleague Ben Fidler reported in September.
Investors hate negative surprises, and there weren’t any whoppers in the CAR-T field this year. One positive surprise came from Cellectis (NASDAQ: [[ticker:CLLS]]), a French drug developer that established a U.S. beachhead in New York this year. Using its version of gene editing called TALEN, the company has created “off the shelf” T cells to treat blood cancer. The DNA of a donor’s T cells are snipped with molecular scissors to make them friendly to a recipient’s immune system. When the cells are infused into the recipient, they attack the cancer, not the patient’s healthy cells. That is the goal, at least: Cellectis’s first product, UCART19, has not been approved yet for clinical trials.
Then news emerged in November that an infant in desperate straits with leukemia was treated with UCART19. It was a special request from her parents, not part of a trial. UCART19 helped bring the baby girl, Layla Richards, back from the brink and allowed doctors to try again a bone marrow transplant, which hadn’t worked previously. As of November, Layla had recovered. In other words, it worked, but it wasn’t part of a trial, and other factors were involved. The halo around Cellectis’s stock wore off quickly. Just like the shares of Juno, Kite, and Bellicum Pharmaceuticals (NASDAQ: [[ticker:BLCM]]), all making personalized, self-donated “autologous” CAR-T products, Cellectis’s shares tumbled in early December around the year’s big hematology conference.
To see if Layla’s recovery is a fluke or part of a promising therapeutic future for others, Cellectis will have to watch from the sidelines. Next year, two larger companies, Servier and Pfizer (NYSE: [[ticker:PFE]]), will take UCART19 into clinical trials, because Cellectis sold Servier the global rights in 2014, which in turn sold U.S. rights to Pfizer last month. The first trial could start in the U.K. soon.
Other CAR-T programs to watch include those from Novartis, Kite, and Juno, in pivotal trials for children and adults with acute lymphoblastic leukemia, or ALL, meaning the data could be enough to spur an FDA approval in 2016 or 2017. “ALL is really the low-hanging fruit of CAR-T,” says independent investor Brad Loncar, who has created a stock index of companies developing CAR-T and other forms of cancer immunotherapy.
After ALL come weightier challenges. Kite is also aiming for pivotal data next year in non-Hodgkins lymphoma. Another program to watch is CAR-T against multiple myeloma, the second most prevalent blood cancer. Bluebird Bio (NASDAQ: [[ticker:BLUE]]) could have a cell therapy in the clinic in 2016, while the NIH continues a Phase 1 trial with an earlier version of Bluebird’s therapy.
Also proving more challenging to treat with CAR-T are solid tumors, such as breast, ovarian, and pancreatic, but 2016 should bring the first significant data sets from early trials in a few indications.
—“Is it time to stop worrying and love the IPO window?” After I asked that question, eight more months passed before the public markets slowed. From January 1 to August 31, about 40 life sciences companies went public in the U.S. Since September 1, 19 have debuted, according to IPOScoop.com. I ask you: Is that the popping of a bubble, or the slow descent of a feather on a windless day? Contemplating that metaphor is like being at a Zen retreat, except you’re reading a column on the Internet.
Biomedical scientists and investors are always wary of changes in regulation and policy. Those risks have been reduced in what one might call “the Hamburg Era.” Peggy Hamburg took the reins of the FDA in 2009 and presided over a drug approval boom. In her final full year, 2014, the agency approved 41 new and novel drugs, the highest total since 1996 and a big leap over the years before Hamburg took the post. Hamburg has handed the baton to Rob Califf, a Duke University cardiologist whose ties to industry have been a point of contention. So far in 2015, the FDA has approved 45.
Although drug pricing has become a talking point, the U.S. political apparatus is squarely (and, if you’re a critic, disturbingly) aligned with getting more drugs to market faster. That won’t change with the blockbuster legislative package known as “21st Century Cures.” It includes FDA reform and more NIH funding, and it passed through the House of Representatives this summer but has since stalled in a Senate committee. According to committee chair Lamar Alexander (R-TN), speaking on C-SPAN, one hang-up is whether the NIH funding should be guaranteed for
