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14 For ’17: Key Clinical Data to Watch For Next Year (Part 1)

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can’t lower dangerous cholesterol levels with statins, the generic standard of care. But these new drugs, called PCSK9 inhibitors, cost roughly $14,000 a year before discounts, and patients are supposed to take them once every month or two for life.

But they haven’t proven they help people with high cholesterol live longer. These massive trials—Amgen’s FOURIER has followed more than 27,000 patients, Regeneron/Sanofi’s ODYSSEY OUTCOMES more than 18,000—aim to do just that.

If the outcome studies are successful, there’s a chance insurers will pay for those costs. Until then, however, insurers have put a chokehold on coverage, forcing doctors to keep prescribing statins. In the first six months of 2016, evolocumab and alirocumab combined for less than $100 million in revenues. A third PCSK9 entrant, Pfizer, measured the landscape and its own drug’s weaknesses and ditched its Phase 3 program this year. Even with good outcomes, some observers think the companies will still have to lower their prices significantly, a message that infuriates the drug makers.

[Updated with info on REVEAL trial] Even if these studies prove successful, however, Merck could throw a big wrench into Regeneron/Sanofi and Amgen’s plans. Early next year, results from another big cardiovascular trial—Merck’s 30,000 patient study of anacetrapib, REVEAL—are expected as well. Other drugs of anacetrapib’s ilk, cholesterol lowering drugs known as CETP inhibitors, were potential threats to PCSK9 blockers, but all of them have failed. Merck’s is the only one remaining.

Disease Area: Cystic fibrosis

Company: Vertex Pharmaceuticals

Trials: Phase 2 and Phase 3

Data expected: Phase 3, First Half 2017; Phase 2, Fall 2017

Why we’re watching: In recent years, Vertex (NASDAQ: VRTX) has pivoted from losing its hepatitis C business to become the industry leader in cystic fibrosis. Its ivacaftor (Kalydeco) and ivacaftor-lumacaftor (Orkambi) are the first two drugs to directly address the molecular malfunction that underlies CF, not just the symptoms. But they are not cures. They only help improve patients’ lung function, and in some cases not that much. They’re only approved for patients with genetic mutations who comprise about 5 percent of the roughly 70,000 people worldwide with CF. And competition is on the way from Galapagos NV and AbbVie (NYSE: ABBV), and others like Concert Pharmaceuticals (NASDSAQ: CNCE) trying to break in.

Vertex will try to keep its pole position by developing a variety of multi-drug “cocktails” to improve upon its existing drugs and treat a wider swath of patients. Two crucial tests will come in 2017. The first, with data expected in the first half, is a Phase 3 trial combining experimental tezacaftor with ivacaftor. The second, expected in the fall, is Phase 2 data from a pair of triple combinations. One combination incorporates experimental drug VX-152, and the other VX-440.

Disease Area: Hemophilia

Companies: UniQure, BioMarin, Spark, Dimension

Trials: Multiple, phase 1/2

Data expected: Varied, 2017

Why we’re watching: Gene therapy has shown early and sometimes stunning results in treating the chronic blood diseases hemophilia A and hemophilia B. Experimental therapies from Spark Therapeutics (NASDAQ: ONCE), BioMarin Pharmaceutical (NASDAQ: BMRN), and UniQure (NASDAQ: QURE) have each shown potential to help patients avoid for months at a time dangerous bleeding episodes or the need for other drugs. But there are caveats: Results have come in small sample sizes, and they have varied patient to patient. Some people, for instance, have developed immune responses that neutralize the treatment. Longer studies are needed to test the durability and safety of the treatments.

In 2017, some of these questions will be answered. Sample sizes for trials will increase, as will the length of time patients are on treatment. It’s possible that bigger differences will emerge between each treatment, or that one of these companies heads towards a “pivotal” trial, the last required before approval. The first data from another hemophilia gene therapy, from Dimension Therapeutics (NASDAQ: DMTX), will also likely emerge in January.

Disease Area: Lung Cancer

Company: AstraZeneca

Trial: MYSTIC

Data expected: January 2017

Why we’re watching: The past few years, the standard of care for lung cancer—the leading cause of cancer death in the U.S.—began to shift, thanks to immunotherapy drugs called checkpoint inhibitors. In October, pembrolizumab (Keytruda) was approved for newly diagnosed patients whose tumors produce certain levels of the protein PD-L1. Nivolumab (Opdivo) is approved for those who have failed a round of chemotherapy.

But many patients get no benefit from checkpoint inhibitors, so researchers are pivoting to combination therapies that might represent the future of cancer care. One major study to provide data in early 2017 will be AstraZeneca’s combination of two “checkpoints,” durvalumab and tremelimumab, compared to chemotherapy in newly diagnosed lung cancer patients. Positive results could be worth billions of dollars to AstraZeneca, but the data are similarly important for cancer researchers. These drugs each block different proteins (PD-L1 for durvalumab and CTLA-4 for tremelimumab) that tumors use to evade the immune system, and MYSTIC will be an important test of how tumors respond when both proteins are blocked. It will also provide information about using a patient’s level of PD-L1 tumor protein to predict his or her response to checkpoint blockers.

(Part two of this list—multiple sclerosis, Zika infection, migraines, and more—coming tomorrow.)

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