The Pipeline Report 2014: Class Seekers

The Pipeline Report 2014: Class Seekers
The Pipeline Report 2014: Class Seekers

These 16 agents, hand-picked from a total field of 164, are poised for approval, and possibly much more. Marc Iskowitz reports

Since the human genome was mapped in 2001, it's taken scientists another dozen or so years to really understand what those genes do and how they affect disease. Better understanding of genetic targets, plus pressure from the market to develop fully differentiated drugs, is leading to a new generation of breakthrough medicines.

But with all the talk of “drugging the genome,” as analysts from Leerink Swann describe the ongoing efforts to develop therapies aimed at restoring normalcy among mutated genes and signaling pathways in cancer, it's possible to overlook two trends.

The first is what many drugmakers are doing to create alternate versions of, and to lower the cost of, existing drugs—in this case biologics.

“Who the players will be in the US, when we start to get biosimilars, is yet unclear, but every day seems to bring news of new biosimilars trials starting and different companies announcing they will play in this space,” says Ben Weintraub, PhD, director of research at inThought.

The coming year will be important in terms of quantifying the impact, he notes. The bellwether will be biosimilar versions of J&J's Remicade, launched in Europe by Celltrion and Hospira, and Eli Lilly's biosimilar insulin glargine, which is pending approval there and still in Phase III on these shores.

Secondly, Weintraub sees examples “of good drugs that are not really changing the standard of care because the unmet need is less than it has been in past.” In other words, drug developers' success in therapeutic areas like oncology and autoimmune is setting the bar higher for new drugs–like Pfizer's RA pill Xeljanz, which he says lacks a compelling value proposition. “[Sanofi's] Lemtrada in the US will fall into that problem, too,” predicts Weintraub. “It's a good drug, but it's hard to figure out how it fits into the current armamentarium.”

With payers in the US, Europe and Japan increasingly scrutinous, difficulty defining a strong value proposition could spell trouble for any drug profiled here. However, many appear to be off to a good start.

The PD-1 inhibitors from BMS and Merck continue to yield early but oh-so-promising results in heavily pre-treated patients with skin, lung and kidney cancer. The PCSK9 inhibitors being developed by Sanofi and Amgen are emerging as an important new class for statin-refractory patients. And in immunology, IL-17 inhibitor secukinumab from Novartis looks poised to transform psoriasis treatment.

In addition to the autoimmune, cardiology, infectious disease, metabolic, neurology, and oncology sectors, the following pages highlight some of the most promising orphan therapies, including Biogen Idec's long-acting clotting factor for hemophilia A, as well as outlining late-stage agents in the respiratory and women's health areas, plus some in mid-stage.

Profiled agents are based on consultation with inThought, Adis R&D Insight, GfK HealthCare and various other experts. Original analysis is updated to reflect the latest data sets (as of press time), and is complemented by revenue forecasts, lists of other key products and, where available, the estimated month of launch, plus a quick way to gauge the likelihood of an FDA OK called the Approvability Index (anything above 50% stands a good chance).



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