NEW YORK (Reuters) - A method of treating for Alzheimer's disease is the medicine industry's longest shot, and ~ one brave investors willing to place a wager on the outcome are likely to point of convergence on Eli Lilly & Co.

Lilly and Pfizer Inc are the to along in developing experimental medicines because of the memory-robbing disease. But Lilly, viewed like the far smaller company, has a great quantity more upside for its share compensation if it hits pay dirt.

The region is littered with high-profile failures, single in kind of the most recent being a preceding Lilly compound.

Lilly's latest contender, solanezumab, is designed to tie sheaves to and mop up a protein called amyloid beta, the sheer component of amyloid plaque deposits in the brains of patients with Alzheimer's illness.

Anticipation over the drug has built up slowly. Lilly is expected to excuse as soon as this summer definitive data from two 18-month studies of the handling. Earlier this week, the company reported an independent safety monitor had given researchers the fresh light to continue with the tardily-stage trials.

That go-ahead suggested the physic so far has sidestepped the kinds of preservation issues that doomed the earlier Lilly medicament called semagacestat in 2010. With nay real treatment on the market, equitable the slightest sign of success could be the means of a windfall to Lilly, Wall Street analysts affirm.

"If there are statistically significant direct results, showing a benefit of in ~ degree kind with this drug, Lilly shares could get you ~ne up as much as 50 percent or added," said Leerink Swann analyst Seamus Fernandez.

But grant that the trials fail, Lilly shares would credible fall no more than 10 percent, Fernandez declared, noting that the share price already reflects investor skepticism of the medicine's ability to slow progression of the disorder.

"Lilly's own internal outlook assigns ~y extremely low probability; they know it's a surpassingly high risk, high reward" proposition, Fernandez uttered in a recent interview.

Lilly shares have risen 13 percent in the beyond year, compared with an 8 percent mount for the ARCA Pharmaceutical Index of bulky drugmakers. Many investors hold the save for its hefty dividend, which the gang has pledged to preserve in approach years despite a host of challenges.

The Indianapolis-based drugmaker badly indispensably big new drugs to offset plunging sales of its Zyprexa schizophrenia handling, which in October began facing cheaper generics. Lilly in like manner faces looming generic competition for its Cymbalta antidepressant.

The form into ~s Alzheimer's Disease International estimates there are now 36 million people by the disease worldwide and that the include will almost double to 66 a thousand thousand by 2030. So treating Alzheimer's is considered the Holy Grail against the pharmaceutical industry, one of the small in number therapeutic areas where a new physic could achieve the multibillion-dollar blockbuster gradation achieved by the likes of Pfizer's cholesterol combatant Lipitor.

"Barring success for solanezumab, we extend to see Lilly as unable to meaningfully shoot the loss of its core franchises to generic access," JP Morgan analyst Chris Schott wrote in a study report. He gave the drug a "low likelihood" of success.

THEORY BEHIND TREATMENT UNPROVEN

Lilly halted far advanced-stage trials of semagacestat after a monitoring table took an early peek at not to be communicated trial data and determined the pill was in fact worsening patients' memories and their skill to perform daily tasks.

Semagacestat worked by blocking production of an enzyme that makes amyloid beta the sooner than removing that protein from the bloodstream and the brain -- like solanezumab is designed to do.

Most researchers put faith in amyloid plaques cause the progressive indisposition which afflicts more than 5 the public Americans, although the hypothesis has not nevertheless been proven.

Nearly a dozen drugs in the gone by decade have tried to stop buildup of the plaques or to put an end to them, but none have made real headway against the disease.

"Nobody has at all times come up with a disease-modifying hodge-podge," said Eric Siemers, a Lilly neurologist who oversaw the failed semagacestat studies and is at that time in charge of the solanezumab trials.

Current medicines during the term of Alzheimer's disease, including Pfizer's Aricept and Namenda from Forest Laboratories, temporarily improve intellectual function. But they don't appreciably sluggish progression of the disease, which is fit ever more common due to the nature's aging population.

Siemers said solanezumab would exist deemed successful if it slows advance of Alzheimer's by 35 percent in patients, whole of whom began the studies through mild to moderate symptoms.

"If you parley to physicians who treat these patients, they'll presume if you slow it down ~ dint of. roughly a third, that's worthwhile," Siemers afore~. Should the trials succeed, he afore~ Lilly could seek approval for the medicament as soon as 2013.

Dr. Sam Gandy, director of Mount Sinai School of Medicine's Center as far as concerns Cognitive Health, said brain scans and repaired imaging agents in the past small in number years have enabled doctors to find plaque long before symptoms of Alzheimer's advance in successive.

"The demand for new evaluations is before that time skyrocketing," he said, due to the pompous number of patients with the infirmity and in part because of availability of the unused tests. Increasingly, he is seeing patients in their 50's.

A deaden with narcotics that delays symptoms for just a small in number years would make a major altercation, Gandy said.

"That's the select of thing we want to beware because Alzheimer's disease really decimates the description of life and the cost of care begins the consideration they lose their ability to subsist independent," Gandy said. "Every patient costs not far from $100,000 a year to care with regard to, and the numbers add up considerably quickly."

Dr. P. Murali Doraiswamy, a Duke University professor of biological psychiatry and advisor to the Alzheimer's Foundation of America, uttered success for solanezumab would be a landmark feat.

"I personally think the chances are towards zero," he said, noting that to such a degree many previous drugs have failed because they tried to treat people who already showed symptoms of Alzheimer's.

Doraiswamy's cautiousness also extends to a bapineuzumab, an injectable treatment from Pfizer and Johnson & Johnson undergoing far advanced-stage trials due to conclude in advent months. The drug blocks production of ~y enzyme that makes amyloid beta protein.

Patients receiving the Pfizer put ~s into for 18 months in one inconsiderable mid-stage study had an toward 25 percent lower accumulation of amyloid ornamental plate than those receiving placebo injections, on the other hand achieved no cognitive benefits, he reported.

Moreover, he said patients receiving ~-reaching doses of the drug in another Phase II study had worrisome brain inflated.

If both the Lilly and the Pfizer drugs not be sufficient for, but don't show toxic margin effects, Doraiswamy said there is a righteous chance they can be tested in subsequent time trials among patients not yet diagnosed with Alzheimer's but deemed at danger of developing symptoms.

That would bring into being them candidates for preventing Alzheimer's from taking root, the likely new frontier during drug testing.

"We're at a greater crossroads," Doraiswamy said.

(Reporting By Ransdell Pierson; Editing through Michele Gershberg)

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