By Brady Huggett
Regeneron Pharmaceuticals Inc. put out its earnings Tuesday and provided follow-up data from its Phase II study of Axokine for obesity, but its heaviest piece of news was the initiation of a Phase III obesity trial for the same drug.
The initial trial will spearhead the overall Axokine Phase III program that is expected to enroll about 4,000 patients. About half of those 4,000 patients are expected to take part in the pivotal trial ¿ enrolling at more than 60 sites in the United States ¿ and that trial will play a large part in when Regeneron looks to file.
¿This trial will set the pace for the ultimate application,¿ said Murray Goldberg, senior vice president, finance and administration, and chief financial officer at Regeneron. ¿We are looking at 12 months of treatment and another 12 months of safety.¿ At the end of the treatment stage, Goldberg said, Regeneron would begin to sift through the numbers and prepare for regulatory filing as the safety data accrued.
The trial will be double blind, randomized and placebo controlled. The first 12 months will see patients receiving either Axokine, dosed at 1.0 mg/kg, or placebo, once daily. In the 12-month safety phase all patients will receive Axokine. The endpoints of the study are based on changes in body weight vs. baseline during the treatment period, and while Goldberg would not reveal specifics, he spoke about the FDA¿s requirements in this area.
¿The FDA has guidelines on obesity that have been out there for years,¿ Goldberg told BioWorld Today. ¿They evaluate drugs and look at two things: amount of weight lost, and the amount of patients that lose 5 percent of weight compared to placebo.¿
The follow-up Axokine Phase II data shed light on a problem experienced by some who shed pounds ¿ difficulty in keeping the weight off. The initial results released last year showed that the drug appeared to spur significant weight loss compared to placebo and the 24-week data showed that it stayed off, Goldberg said. (See BioWorld Today, Nov. 29, 2000.)
¿We are excited about the results ¿ both by the weight loss we saw in the 12 weeks of treatment and the observations that patients maintained the weight loss after treatment was stopped,¿ Goldberg said. ¿Six months is a long period of time, but now we¿ve seen [weight loss] maintained for 24 weeks after treatment, and there will be a 48-week observation point.¿
Helping keeping the pounds off is what makes Axokine different, Goldberg said.
¿Axokine goes through the bloodstream and reaches the hypothalamus, which is believed to control appetite,¿ he explained. ¿It acts in the hypothalamus by suppressing food intake. What appears to be unique about Axokine is the body doesn¿t retain a memory of that loss of weight, so when treatment stops, there is no immediate rebound in food intake and weight gain. The body doesn¿t overreact. Normally, when people are on a diet or on drugs, the body sends out hunger signals. The hunger signals are not eliminated, but they are abated.¿
In its early stages, Axokine was partnered with Procter & Gamble Co., of Cincinnati, until Phase I results indicated that perhaps the drug would be usable only in herpes simplex virus-negative patients. But Regeneron pushed ahead with an adjusted dosing schedule in HSV-positive and -negative patients alike, which eliminated that concern, and the drug is aimed at both today. It has remained unpartnered since then. (See BioWorld Today, Oct. 1, 1997, and Sept. 3, 1999.)
¿None of Regeneron¿s current programs are partnered but that doesn¿t mean we wouldn¿t find that beneficial in the future,¿ Goldberg said. ¿We certainly have enough financial capability to take this through Phase III and we have the infrastructure.¿
Regeneron, of Tarrytown, N.Y., had $290.5 million in cash and marketable securities as of June 30. The company posted a net loss for the quarter of $14.8 million, or 34 cents per share. But the most notable figure, said Charles Poole, vice president, investor relations, at Regeneron, is what the company spent to keep the pipeline filled and flowing.
¿The thing that is important to us is our investment in [research and development],¿ he said. ¿And we invest to do two things: to move products into clinical trials and to make sure they progress in a sufficient manner.¿ Regeneron spent about $19.6 million on research and development in the second quarter of 2001, up from approximately $15.4 million for the same period in 2000.
The news was released after the market closed Tuesday. Regeneron¿s stock (NASDAQ:REGN) rose 14 cents Tuesday to close at $26.87.