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REGENERON CNTF PHASE III TRIAL COMPLETION SLATED FOR MID-1994,

Executive Summary

REGENERON CNTF PHASE III TRIAL COMPLETION SLATED FOR MID-1994, company Chairman Leonard Schleifer indicated during a March 30 meeting at the New York Society of Security Analysts. The 700- patient trial at more than 30 sites will evaluate the safety and efficacy of Regeneron's recombinant human ciliary neurotrophic factor for treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) over nine months of treatment. "If successful, the data from the trial will be used to support the submission of a PLA," Regeneron said. "We already have identified over 500 patients who will be enrolled in this study," Schleifer said. Regeneron "should be able to have full enrollment...probably by the end of the second quarter." The Phase III trial will involve two doses of CNTF versus placebo in "early- to mid-" stage, rather than severe, ALS, Schleifer said. The study will attempt to demonstrate that CNTF can "slow the rate of decline of muscle strength" as measured by a Regeneron-developed scoring system, he added. The trial will also measure some "electrophysiological parameters." Regeneron and Synergen appear to be neck and neck in the race to develop CNTF for ALS. Synergen is beginning its pivotal trial of CNTF, which will enroll approximately 500 patients at 24 sites. Synergen has not disclosed how long the trial is expected to run. Both Regeneron and Synergen have orphan designation for CNTF: the first to win approval will have seven years of marketing exclusivity under current law. A potential marketing complication involves the issue of patents. Synergen has two issued patents covering the CNTF gene and a production process, Schleifer noted. Regeneron has received allowances for patents in the same areas, and the matter has been referred to the patent office interference branch, Schleifer said. No patents have yet been issued covering the CNTF protein or the use of CNTF in ALS, he added. During his presentation to NYSSA, Schleifer repeatedly sought to distinguish his company from other biotechs (including Synergen) that have not met analysts' expectations. Synergen canceled an April 7 appearance before NYSSA due to a management reshuffling (see following T&G). Schleifer was asked to discuss Regeneron's Phase II CNTF data. "We don't want to hype things based on studies we can't say definitively will support the licensing of a product," he responded. "We don't want to create false hopes for either patients or investors" by "prematurely talking about efficacy," he declared. The questioner pressed Schleifer, citing investors' need to evaluate the progress of the company. "The glib answer to that is that [Phase II data] hasn't helped you in the past," he said. "Our goal is not short-term blips [in the price of the stock] -- it is to get this product approved." Investors "have to be comforted by the fact that we have accomplished what we set out to do" so far in developing CNTF. Schleifer added that some CNTF data will be discussed at the American Academy of Neurology annual meeting in New York City later in April. Schleifer was asked if Regeneron was seeking cost- effectiveness data from the CNTF trial. In another oblique reference to Synergen's Antril experience, Schleifer said "at this point we haven't made that our focus. We don't want to outsmart ourselves" by concentrating on cost effectiveness rather than efficacy. Regeneron will have "an enormous database" at the end of the Phase III trial from which "we'll be able to extract that data," Schleifer maintained. Synergen publicized cost- effectiveness data from its Phase II study of Antril in sepsis ("The Pink Sheet" May 11, 1992, T&G-9), only to have its Phase III trial fail to show efficacy. Schleifer told the analysts that Regeneron is not publicly projecting the market for CNTF. He did, however, refer to analysts' estimates of a market size of $200-$500 mil. for the 15,000-30,000 ALS patients in the U.S. based on an estimated drug cost of $15,000 per patient per year. Regeneron continues to emphasize its relationship with Amgen in its dealings with the financial community. The two firms have a 50/50 joint venture in the U.S. for the development of brainderived neurotrophic factor (BDNF) and neurotrophin-3 (NT-3). BDNF is scheduled to start clinical trials for "diseases of the peripheral nervous system" by "the third quarter of this year," Schleifer said. An IND for NT-3 is expected to be filed by the end of the year, he added. In December, Regeneron signed an agreement with American Cyanamid to develop heparinbinding neurotrophic factor and midkine neurotrophic factor. "Not everything we work on is going to turn to gold," Schleifer cautioned. However, "we are here for the long haul."
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