[Updated, 4:28 pm ET, see below] After years of research, frustration, hype, and millions (sometimes hundreds of millions) of dollars invested, a drug program boils down to the clinical data—usually from big, late-stage studies—that can sometimes make or break a company.
There were high profile successes in 2016. Biogen (NASDAQ: BIIB) and Ionis Pharmaceuticals (NASDAQ: IONS) presented data supporting what could become the first approved drug for spinal muscular atrophy. Tesaro (NASDAQ: TSRO) reported data for a drug that, if regulators approve, could help fend off the recurrence of ovarian cancer. There were also plenty of high-profile failures in 2016: an Alzheimer’s drug from Eli Lilly, an age-related vision loss drug from Ophthotech (NASDAQ: OPHT), a brain cancer vaccine from Celldex Therapeutics (NASDAQ: CLDX), a microbiome therapy from Seres Therapeutics (NASDAQ: MCRB), and an RNA interference drug from Alnylam Pharmaceuticals, to name a few.
So what’s in store for 2017? Xconomy has put together a list of some of the most noteworthy clinical data expected next year, in heart disease, cancer, cystic fibrosis, and more. Part one is here. Part two is coming tomorrow. [Editor’s note: Alex Lash and Frank Vinluan contributed to this report.]
Disease area: Asthma
Companies: Regeneron and Sanofi
Trial: LIBERTY ASTHMA QUEST
Data expected: Late 2017
Why we’re watching: Dupilumab, from partners Regeneron Pharmaceuticals (NASDAQ: REGN) and Sanofi (NYSE: SNY), is nearing an FDA approval decision for eczema. But it also might have an impact on other diseases; next year its big test will come in asthma. About 24 million people in the U.S. have the respiratory disorder, according to the Centers for Disease Control and Prevention. For 10 to 20 percent, existing treatments don’t help, leading to hospital visits and other problems. Dupilumab, an antibody drug injected once every two weeks, could be another option if it comes through in Phase 3. In previous testing, dupilumab taken with inhalable drugs lowered asthma attacks and increased lung function with mostly mild or moderate side effects. But those results will have to be replicated in the larger study—1,858 patients, according to Clinicaltrials.gov. Its main goal is to reduce severe asthma attacks over the course of a year.
Disease Area: Alzheimer’s
Companies: Axovant Sciences and Merck
Trials: MINDSET and EPOCH
Data expected: Mid-to-late 2017
Why we’re watching: Both MINDSET and EPOCH are Phase 3 trials. Considering the dire need of Alzheimer’s, the late stage alone is enough to generate attention. But there are other intriguing reasons to watch. MINDSET sponsor Axovant (NASDAQ: AXON) had tongues clucking when it reeled in 2015’s biggest biotech IPO despite the fact that its only asset is a drug bought from GlaxoSmithKline for $5 million—part of founder Vivek Ramaswamy’s plan to find deep discounts on pharma’s dusty shelves and develop them quickly with loads of new cash. (He repeated the trick with Myovant Sciences, and is at it again with newly formed Dermavant Sciences.)
The Glaxo drug, now called RVT-101, is aimed at boosting cognition in mild-to-moderate Alzheimer’s patients already taking donepezil, one of only two drugs currently approved for Alzheimer’s. It’s a more modest goal than altering the root cause of the disease. But disease-modifying drugs have all failed. Indeed, the entire Alzheimer’s field has a near 100 percent failure rate. If Axovant shows RVT-101, which is taken as a pill, can help stave off decline even a little longer than the two current standards, the 31-year-old hedge-fund upstart Ramaswamy will have done something that drug giants spending billions of dollars have not been able to do: Give Alzheimer’s patients hope.
One of those giants is Merck (NYSE: MRK). Its 2200-patient EPOCH trial aims to show the pill verubecestat can improve cognition in mild-to-moderate patients. Similar goal as Axovant, but different method. Verubecestat disrupts a protein called amyloid-beta that clumps up in patients’ brains. Unlike recent amyloid failures, verubecestat works to prevent formation of the amyloid fragments that accumulate into the sticky clumps called plaques. Other amyloid drugs try to soak up the fragments or bust up the plaques.
Merck’s big gamble: Reaching mild-to-moderate patients already deep into the disease has proven fruitless with other amyloid-disrupting drugs. What’s more, older so-called BACE inhibitors have failed before because of unacceptable side effects. The new generation, like verubecestat, must prove they can be safe enough and improve the conditions of patients who, some researchers believe, might be beyond help already.
Disease Area: Beta-Thalassemia/Sickle Cell Disease
Company: Bluebird Bio
Trials: NORTHSTAR-2 and HGB-206
Data Expected: TBD 2017
Why we’re watching: Bluebird (NASDAQ: BLUE) is a key player in gene therapy’s renaissance, but it’s had a bumpy ride the past year or so. It reported positive early data in 2014 and ’15 with its LentiGlobin therapy in a handful of patients with chronic blood diseases beta-thalassemia and sickle cell disease. But Bluebird has since been unable to meet that high bar. Turns out some beta-thalassemia patients do not respond as well as others, depending on underlying genetics. Follow-on results in sickle cell patients have also disappointed.
In response, Bluebird in 2016 said it made key manufacturing improvements to lead to a more effective therapy with less variable results. That remains to be seen: The first substantive evidence should come this year, when Bluebird reports the initial results from a trial called HGB-207, or NORTHSTAR-2, and the latest data from an ongoing sickle cell study named HGB-206.
Disease Area: Cardiovascular Disease
Companies: Regeneron/Sanofi and Amgen
Trials: ODYSSEY OUTCOMES and FOURIER
Data expected: 2017
Why we’re watching: It’s the battle over U.S. drug prices in a nutshell. Evolocumab (Repatha) from Amgen (NASDAQ: AMGN) and alirocumab (Praluent), from partners Regeneron and Sanofi, have shown in big trials that they significantly lower cholesterol. The FDA approved them in 2015 for a small slice of patients who … Next Page »