By Brady Huggett

NeoTherapeutics Inc. said patients receiving its lead product, Neotrofin, over 90 days in a global Phase II study showed a statistically significant improvement in an Alzheimer's disease rating scale that measures behavioral symptoms, but more data are needed.

"This study confirms all the data we have through animal studies and preclinical trials," said Rajesh Shrotriya, president of NeoTherapeutics. "And it tells us we need to do long-term trials because Alzheimer's is a slow-progressing disease. If you want to make a claim that you are modifying the disease, you need to show over a long trial that the patients have a slope that is flatter than placebo."

Preliminary results were presented in July at the World Alzheimer's Congress 2000.

Patients receiving Neotrofin (AIT-082) in a berry-flavored suspension - a form that is key when dealing with Alzheimer's patients, Shrotriya said - showed improvement on the Neuropsychiatric Inventory, a rating scale that measures 12 Alzheimer's symptoms, including hallucinations, aggression, irritability, depression and apathy.

Also, patients suffering from moderate Alzheimer's that received a 500-mg dose of Neotrofin improved 1.5 points from baseline on average on the Alzheimer's Disease Assessment Scale-cognitive subscale, with placebo being baseline. The scale measures patients' cognitive abilities and memory. The drug was shown to be safe and well tolerated, with 389 of the 431 patients completing the entire dosing. However, the findings for this scale were not statistically significant, although Shrotriya said the results were encouraging.

"For cognition and memory, we saw an improvement in the right direction, and that is why we need to do a longer study," Shrotriya said. "My feeling is, if the patients had stayed on this drug for six months or a 12-month duration, I think we would have seen a greater difference."

Beyond this Phase II, NeoTherapeutics is enrolling 1,500 patients for a Phase II/III trial for Alzheimer's to take place in 165 sites and 17 countries around the world. NeoTherapeutics has a Phase IIb completed in the United States and will have those data analyzed in the next few months, Shrotriya said. (See BioWorld Today, March 30, 2000.)

"We are very excited about the possibilities for our drug because it works through the regeneration of neurons," he said. "It should have applications for all neurodegenerative diseases and also spinal cord injury patients."

NeoTherapeutics, of Irvine, Calif., focuses its research and development program on designing and developing small molecules for neurological and psychiatric diseases and conditions, including Alzheimer's and Parkinson's diseases, peripheral neuropathy, stroke and spinal cord injury. NeoTherapeutics' pipeline includes AIT-082 for other indications - it's in the research stage for stroke and preclinical stages for neurodegeneration and spinal cord injury. Its product AIT-034 for dementia is in the preclinical stage, as is AIT-203 for Parkinson's disease, AIT-297 for treating migraines and AIT-202 for depression and obesity.

The company raised $8 million in early October to fund clinical trials, but Shrotriya said it will have to go to the well again before it's all said and done. The company has about $14 million in cash, he said.

"We are constantly raising money," Shrotriya said. "We raised enough money so that we can run the programs through, but we'll need more for Phase III. Money is being offered to us even now."

NeoTherapeutics' stock (NASDAQ:NEOT) closed Thursday at $7.75, up 75 cents.