DRUG TEST RESULTS ON ALS PATIENTS DISAPPOINT AMGEN.

Byline: Dave McNary Daily News Staff Writer

Biotechnology giant Amgen Inc., in a setback to efforts to expand its product line, announced Friday that extensive testing of its BDNF drug to treat Lou Gehrig's disease showed the drug was ineffective.

Thousand Oaks-based Amgen and partner Regeneron Pharmaceuticals disclosed that testing of BDNF, or brain-derived neurotrophic factor, showed there was no improvement in the breathing capacity in a Phase III, or late-stage, trial of about 1,000 patients with amyotrophic lateral sclerosis or ALS, also known as Lou Gehrig's disease in memory of the baseball star who died of it in 1941.

Loss of breathing capacity is often the cause of death from ALS. There is no known cure for the disease, which causes muscles to fail and waste away.

``Obviously, we are disappointed with these results,'' said Gordon Binder, chairman and chief executive officer. ``We had hoped this trial would show benefit (for) patients with this tragic disease.''

An estimated 30,000 to 40,000 Americans have the disease.

Binder said the companies have halted development of BDNF for beneath-the-skin delivery but would continue to work on small Phase I trials investigating injection of the drug into a patient's spinal fluid along with injections for nerve complications stemming from diabetes.

Given the severity of the disease, which kills about 5,000 people annually, and the large size of the Phase III trial, BDNF could have been placed on a fast track for regulatory approval had the test results been positive.

``Obviously, this is very disappointing for Amgen,'' said Carl Gordon, an analyst with Mehta & Islay. ``BDNF could have been a blockbuster if it had really worked well.''

If it had worked well, analysts said, BDNF could have taken a major part of the potential market for ALS drugs, estimated at $250 million to $500 million.

Shares of Amgen fell $2.8125, or 5 percent, to $55.0625 while Regeneron was clobbered, plunging by $8.25 to $10.875. Amgen owns 18 percent of Tarrytown, N.Y.-based Regeneron, and its agreement with Regeneron caps its potential investment at 20 percent.

James McCamant, editor of the Medical Technology Stock Letter, said Wall Street had not been overly optimistic that BDNF would work against ALS. ``It was a 50-50 chance,'' he said. ``ALS is a tricky disease, and BDNF is a really interesting molecule, but they still haven't quite figured out what it will work on and how to make it work.''

Amgen has spent aggressively on research in recent years to expand its portfolio beyond its flagship red-cell booster Epogen and white-cell stimulator Neupogen. McCamant noted that Amgen has a strong financial outlook, with annual revenues of more than $2 billion. ``This is not a serious blow, but it is disappointing because they have a relatively thin pipeline of new products,'' he said.