Amgen, Regeneron Say BDNF Isn’t Effective

Amgen Inc. and its partner Regeneron Pharmaceuticals Inc. struck out Friday in their long effort to develop a biotechnology drug to treat Lou Gehrig’s disease--announcing that their test drug BDNF is not effective against the fatal nerve disease.

Amgen had hoped the test drug would be among its first successful new products in six years, but BDNF was unable to significantly improve the condition of 1,000 patients suffering from Lou Gehrig’s disease, the two firms reported.

“This was a high-profile program and a failure,” said Stephen Sabba, analyst with Sturza’s Institutional Research in New York. “It’s a big disappointment for both Amgen and Regeneron.”

Amgen also announced Friday that another of its own developmental drugs, Stem Cell Factor, performed successfully in human tests to help combat toxic side effects from chemotherapy. Amgen said it plans to apply for FDA approval. Analysts said Amgen’s stem cell drug could become a $50 million a year product.

However, Wall Street paid more attention to the disappointing results. Amgen’s stock slid $2.8125, and closed Friday at $55.0625 per share on the Nasdaq. Trading in the stock was halted briefly before the company released its test results.

Meanwhile, Regeneron, which has no drugs approved for sale, got hammered, with its shares down $8.25 on Nasdaq, and closed at $10.875 for the day. Amgen entered a joint research deal in 1990 with Regeneron, which is based in Tarrytown, N.Y.

Amgen, based in Thousand Oaks, was hoping for encouraging test results on BDNF that could have led to FDA approval by late this year.

Lou Gehrig’s disease is named after the baseball Hall of Famer who was diagnosed with the illness in 1939. Gehrig died two years later at the age of 37; the disease is formally known as amyotrophic lateral sclerosis, or ALS.

About 5,000 people a year in the U.S. are diagnosed with ALS, and 30,000 people currently have the disease. The disease strikes the nervous system, usually in young people, and although their minds stay sharp, they gradually become paralyzed and typically die within five years.

Many ALS patients suffocate when their breathing muscles stop. It was hoped that BDNF would help slow these breathing problems.

There is only one ALS drug now for sale, and analysts estimate the market for ALS drugs at $250 million to $500 million a year.

“I thought [BDNF] really had a good chance. But it’s a tough disease to treat,” said Jim McCamant, editor of the Medical Technology Stock Letter in Berkeley.

Amgen will continue a small project to test BDNF on ALS patients by injecting the drug into their spinal fluids. The company is also testing the drug on diabetic patients who suffer numbness in their limbs.

Amgen’s stem cell drug was tested on 205 patients with breast cancer. The Phase 3 trials showed that patients who were given SCF, along with Amgen’s old drug Neupogen, required one-third fewer blood collection procedures.

In current therapy, cancer patients often have some of their peripheral blood cells removed and then frozen. After intensive chemotherapy, the blood components are injected back into the patient in the hopes of reducing infections and side effects.

McCamant said Amgen’s SCF drug could also reduce the need for bone marrow transplants.

Amgen is the world’s most successful biotech company. But its wealth stems from only two drugs. One triggers red blood cells that treat anemia in kidney disease; the other is Neupogen, a white blood cell drug that combats infections in everything from cancer to AIDS.