Cleveland Clinic toasts Novartis, PCSK9ers and immuno-oncology in its R&D top 10

The Cleveland Clinic's annual list of the most promising medical innovations reads a bit like a biopharma yearbook, name-checking a slew of potential blockbuster treatments and affirming the potential of some of the biggest targets in drug development.

Unveiled at the Cleveland Clinic's 2014 Medical Innovation Summit, the organization's yearly top 10 is billed as a list of the projects most likely to have major impact on improving patient care in 2015, selected by a panel of 110 physicians and scientists within the nonprofit. Perhaps unsurprisingly, 6 of this year's winners are drugs or biologics, headlined by two superlatively competitive therapeutic areas projected to bring in billions in revenue.

  • Checkpoint inhibitors: Cleveland's clinicians heralded the promise of an emerging class of treatments designed to galvanize an immune system attack on tumors by blocking a pathway called PD-1, which, left unchecked, allows cancerous cells to pass undetected. The first natural target is melanoma, for which Merck ($MRK) and its first-mover Keytruda won FDA approval this year, but investigators believe checkpoint inhibitors have exciting potential in lung, kidney and other cancers. Bristol-Myers Squibb ($BMY), Roche ($RHHBY) and AstraZeneca ($AZN) are among the other contenders working up inhibitors of PD-1 or the related PD-L1, racing to market with treatments expected to gross more than $35 billion a year combined.
  • PCSK9 blockers: More than a decade ago, scientists homed in on a members of French family who, no matter what they ate, were unable to maintain healthy levels of LDL (or "bad") cholesterol. The problem: overactive copies of a gene called PCSK9 that regulates the body's LDL receptors, overseeing the process by which the liver cleans bad cholesterol out of the blood. Now, after years of research, some biopharma innovators have developed antibodies to the PCSK9 protein, crafting injectable treatments that significantly slash LDL levels. Amgen ($AMGN) is locked in a race with partners Sanofi ($SNY) and ($REGN), each expecting FDA approval next year, while Pfizer ($PFE) brings up the rear with a similar treatment. Roche, Bristol-Myers , Alnylam ($ALNY) and others are working up earlier-stage approaches to the same target.
  • Novartis' LCZ696: The scourge of heart failure can often be successfully controlled with so-called ACE inhibitors, long-generic drugs that relax blood vessels. But the ailment still kills more than 55,000 people a year, underscoring the need for a more effective solution. The Cleveland Clinic believes Novartis ($NVS) has discovered just that in LCZ696, a combination of the well-established Diovan with a new entity called sacubitril. In Phase III trials, LCZ696 reduced the risk of cardiovascular death by 20% compared to enalapril, a gold-standard ACE inhibitor, and patients on Novartis' treatment experienced 21% fewer hospitalizations and a 16% improvement in all-cause mortality. That promise has spurred analyst projections of up to $10 billion a year for the drug, and Novartis CEO Joe Jimenez said he believes the treatment has the potential to be a "multi-blockbuster."
  • New treatments for IPF: For years, patients with idiopathic pulmonary fibrosis (IPF) had no options to treat their often-fatal ailments, a prognosis soon to change thanks to InterMune ($ITMN) and Boehringer Ingelheim. The two companies won tandem FDA approvals for new drugs that combat the root cause of the disease, a lung-scarring affliction that inhibits oxygen absorption and hampers pulmonary function. In pivotal trials, both treatments significantly improved lung function compared to placebo, signaling new hope for sufferers of a disease that kills about 40,000 people each year
  • Sanofi's dengue fever vaccine: It only took 20 years, but Sanofi is finally rolling toward regulatory filings for the world's first vaccine for dengue fever, a deadly tropical disease that infects as many as 100 million people each year. The plan is to submit the shot to global regulators as early as the first quarter of next year, CEO Chris Viehbacher said (shortly before he was fired), and the company hopes to begin administering its long-in-development vaccine by the end of 2015.
  • Antibody-drug conjugates: On paper, killing cancer cells is easy: Blast enough cytoxic material at any tumor and it's unlikely to survive. However, because such poisons are liable to damage healthy tissues as well, researchers have developed a homing system designed to create a more targeted attack. By attaching anti-cancer payloads to synthetic antibodies engineered to bind only to cancer cells, drug developers have forged antibody-drug conjugates (ADC), hybrid therapies with broad promise. Two ADC-powered treatments are already approved--Seattle Genetics' ($SGEN) Adcetris and Roche's Kadcyla--and scores more are in development across the industry.

The Cleveland Clinic's four other honorees skew to the med tech side of the industry, including cheap blood testing technology, a leadless cardiac pacemaker, a mobile stroke unit and intra-operative radiation therapy devices.

- read more

Special Reports: The top 15 late-stage blockbusters in the pipeline | Science pays: 2013's 10 best-compensated R&D chiefs in biopharma