Tracking the trends

Learning which interesting drugs are in the clinical trial pipeline and which ones have garnered U.S. Food and Drug Administration (FDA) approval is critical for both scientists and business development professionals in the pharmaceutical and biotechnology arenas. One company offers a free look every quarter.

Thomson Reuters released its newest edition of "The Ones to Watch," a quarterly report on the most promising drug launches, approvals and changes in clinical phase. Since 2006, Thomson Reuters has used strategic data and insight from its Cortellis for Competitive Intelligence division to list and analyze drugs with the most potential for success. Published in August, the current report covers April through June.

"While certain categories of drugs seem to be key areas time after time, the challenge is to look outside of the likely areas and find drugs that are unusual and that treat unmet needs," explains Dr. Peter Robins, editorial and content manager of the report.

For example, the current report lists three drugs for ulcerative colitis, "each operating in a different way," Robins says, adding that it is a "challenge to modify this disease of unknown etiology." He explains that drug researchers have to make decisions about how easy a drug is to develop, versus the need, versus the opportunity. "This is a microcosm for other diseases," he says.

According to the report, ulcerative colitis affects somewhere "between eight and 246 individuals in every 100,000, causing considerable pain to sufferers and costing Western nations billions of dollars each year." While the three drugs are at different stages of development, "each has a different mechanism of action, highlighting the opportunities available while the precise causes of the disease continue to be explored," the report says.

One of the drugs, Hutchinson Medipharma Enterprises' HMPL-004, is a novel botanical extract. It entered Phase III development for ulcerative colitis and is currently being assessed in a Phase II trial for Crohn's disease.

Another key topic of the report is metastatic melanoma, for which the FDA has approved GlaxoSmithKline PLC's (GSK) Mekinist and Tafinlar. These personalized medicine therapies for advanced metastatic or unresectable melanoma represent a "two-pronged attack that acts on a small group of patients, rather than a wider population," according to Robins. He adds, "Until now, many cancer drugs have been a machine-gun approach that may not fit the targets."

Metastatic melanoma, "a devastating disease that presents a huge challenge," in the words of Robins, is the most aggressive form of skin cancer in which the cancer spreads beyond the surface of the skin to other organs. Affecting about 10,000 people globally each year, the disease has a one-year mortality rate as high as 66 percent.

According to the report, "about half of those suffering from the disease are positive for a BRAF mutation. The BRAF V600E mutation accounts for 85 percent of all BRAF V600 mutations, with BRAF V600K accounting for a further 10 percent."

To address this need, GSK developed two personalized therapies. Mekinist, the world's first approved MEK inhibitor, targets tumors expressing the BRAF V600E and V600K gene mutations. Tafinlar, a BRAF inhibitor, targets tumors expressing the BRAF V600E gene mutation. Both drugs were approved by the FDA in May 2013, along with a genetic test. Launch of both drugs is expected in 2013, and GSK submitted a supplemental New Drug Application for the use of the two drugs in combination. Mekinist and Tafinlar are the second and third personalized medicines approved for this indication. While personalized therapies have a limited target market, Mekinist and Tafinlar are expected to reach global sales of $556 million and $374 million, respectively, in 2018, according to forecast data from Cortellis Competitive Intelligence.

Another important topic in the report is obesity, which, as the report explains, can lead to cardiovascular disease, type 2 diabetes, some cancers, asthma, obstructive sleep apnea and reduced life expectancy. It cites the World Health Organization prediction that by 2015, approximately 2.3 billion adults globally will be overweight, and more than 700 million will be obese.

"The Ones to Watch," highlighted Arena Pharmaceuticals' Belviq in the third quarter of 2006 when it entered Phase III clinical trials. In the second quarter of 2013, it was approved in the United States for use as an adjunct to a reduced calorie diet for chronic weight management in adults. Belviq, which is only the second weight loss drug to be approved by the FDA in the last 13 years, is expected to command global sales of up to $647.3 million in 2017 and 19 percent of the obesity drug market by 2018. The oral small molecule activates the serotonin 2C receptor in the area of the brain critical for regulating metabolism and food intake.

While Robins says he enjoys seeing drugs that were considered interesting a few years ago, he also knows a lot can happen in the interim. Still, he thinks the most interesting section of the report is about the Phase I candidates.

"That's where the true novelty is," he says.