<AXYX - Phenserine>
I don't know anything about the company or the compound, but fast track for an Alzheimer drug is somewhat unlikely on the surface. Memantine didn't get fast track, for example.
Well FWIW the company begs to disagree with my analysis:
Reuters
Axonyx says Alzheimer's drug may be fast tracked
Tuesday September 30, 3:05 pm ET
By Deena Beasley
LOS ANGELES, Sept 30 (Reuters) - Axonyx Inc. (NasdaqSC:AXYX - News), a tiny biotech company that began 2003 as a penny stock, survived the sector's 2002 dry spell, recently securing enough cash to fund clinical trials of its experimental Alzheimer's drug.
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Axonyx said the compound, called Phensarine, could be given a quick review by regulators depending on results of late-stage testing now underway in Europe, but it still expects to launch a pivotal-stage U.S. trial of the drug.
The New York-based company was warned in December it could be delisted from Nasdaq due to a shortfall in shareholder equity, but its stock has risen nearly sixfold so far this year, compared with a 36-percent rise in the American Stock Exchange biotech index. On Tuesday, the shares were up 3.4 percent at $4.24 in late Nasdaq trading.
"We just raised $25 million," Dr. Marvin Hausman, chief executive at Axonyx, told Reuters after explaining that a pivotal-stage U.S. trial of the drug would cost some $13 million. The private placement of stock and warrants was announced earlier this month.
In the first half of this year, the company reported a net loss of $2.8 million.
"From a risk point of view, it is still an attractive investment opportunity," said Matt Kaplan, an analyst at Punk, Ziegel & Co. "They have a Phase III product in a known therapeutic class." He puts the stock's 12-month price target at $8.
Axonyx in June launched two European trials of Phensarine, an oral drug designed to treat Alzheimer's by interrupting a genetic message that tells cells to produce beta-amyloid, the toxic protein that accumulates in the brains of people who suffer from the memory-depleting disease.
The drug works by blocking an enzyme that breaks down acetylcholine, an important neurotransmitter in the brain, which is associated with improved memory and thinking ability. But it also inhibits production of beta-amyloid protein, which suggests that it has the potential to slow the progress of Alzheimer's, according to Axonyx.
"Phensarine goes into the enzyme like a key inside of a lock," Hausman said.
TRIAL RESULTS KEY
Alzheimer's drugs like Pfizer Inc.'s (NYSE:PFE - News) Aricept work by blocking the cellular opening, which helps to relieve symptoms but not to slow the rate of brain deterioration, he explained.
"The drug's profile looks very interesting ,,, but so far there is only limited Phase 2 data," Kaplan said.
Phensarine's binding action does not cause side effects, but blockage of the acetylcholinesterase enzyme causes nausea and vomiting at rates similar to those seen with other drugs in the same class. Phensarine is more likely to cause dizziness than the other drugs, because it is powered to get into the brain, Hausman said, adding that Axonyx is working to fine-tune the dose.
Regardless of new U.S. trials, Phensarine could get an expedited review by regulators if results from recently launched trials are strong, Hausman said.
"If our amyloid beta trial has good data, and the cognition data is positive we would have blockbuster results," he said.
Results from the first European trial of 75 patients -- designed to measure the drug's effect on levels of beta-amyloid -- are expected next March.
"In animal models they have shown that production of beta-amyloid is decreased by more than 50 percent," Kaplan said. "That's the Holy Grail in terms of Alzheimer's research."
Results from a larger, 375-patient, Phase 3 trial, which will measure memory, are not expected until December 2004.
Hausman said Axonyx chose to conduct these trials in Europe because costs will be about 30 percent lower than in the United States and it is easier to find Alzheimer's patients that have never been treated with drugs.
But the company does plan to launch a pivotal-stage U.S. trial. "We plan to follow the cookbook used by Aricept and Exelon," Hausman said. "It's a class of drugs that's known to work," the CEO said.
"The drug could be fast-tracked," he added, noting that Johnson & Johnson's (NYSE:JNJ - News) Alzheimer's drug Reminyl did not have a U.S. trial before being approved for sale in this country.
The CEO said Axonyx does not need a corporate partner to usher Phensarine through the regulatory process, but it will likely need to ally with a company experienced in marketing geriatric drugs. |
