With its IPO pending, Eyetech Pharmaceuticals Inc. disclosed limited Phase III data regarding Macugen last week at a scientific meeting, and the news about its treatment for age-related macular degeneration was not good.
"They're in a difficult situation," said analyst Douglas Miehm, with RBC Capital Markets in Toronto, who called the data "positive but underwhelming" in a research note.
Neither Eyetech nor partner Pfizer issued press releases, but QLT Inc., of Vancouver, British Columbia - which markets Visudyne (verteporfin) for the condition - did, noting that the top-line results show that Macugen "appear[s] to provide no improvement" over Visudyne for patients with choroidal neovascularization due to AMD.
"I still think this product is going to be approved," Miehm told BioWorld Today, pointing out that Eyetech has a deal for Macugen with New York-based Pfizer Inc., "one of the best marking partners in the world." A year ago, the companies signed a potential $750 million deal. (See BioWorld Today, Dec. 19, 2002.)
Miehm allowed that QLT's Visudyne partner Novartis AG, of Basel, Switzerland, is "also strong," and QLT seems to have a leg up for the moment, even though Visudyne is said to be effective in only about 30 percent of patients.
QLT noted that although complete data were not presented at the American Academy of Ophthalmology meeting in Anaheim, Calif., results with the anti-vascular endothelial growth factor aptamer Macugen appear no better than Visudyne's original data from QLT's TAP (which stands for "treatment of AMD with photodynamic therapy") trial in all AMD lesion types.
Now, it's all about what the FDA will want from New York-based Eyetech, and whether the agency will let the company file for a new drug application based on all the data, or will advise a filing only for some subgroup of AMD patients in which Macugen was particularly effective.
AMD cases are divided into predominantly classic, minimally classic and occult, based on how clearly defined they are.
More trials with Macugen (pegaptanib sodium) might even be in the cards, but Miehm doubts the FDA will require them. "It wouldn't help anyone if [Eyetech] had to go back and do that," he said. "I think they'll be able to go ahead with what they have."
Eyetech filed for its IPO in September, and last week set its price range at $18 to $20 and number of shares at 6.5 million, which at the top end would raise $130 million. (See BioWorld Today, Sept. 16, 2003.)
AMD, which is the leading cause of blindness for people more than 50 years of age, comes in two forms. The "wet," or neovascular, type is characterized by new blood vessels forming behind the eye as the body attempts to get more blood to retinal tissue. When the blood vessels break, the tissue around them is damaged.
The much more common and much more gradual "dry" form of the condition involves the forming of drusen - yellowish deposits - in the retina and loss of pigment.
Although the wet form of AMD makes up only 10 percent to 15 percent of cases, it's responsible for most blindness caused by the condition, with vision loss starting about age 55.
There are other late-stage product candidates in wet AMD. In September 2002, Alcon Research Ltd. began enrolling a 500-patient Phase III study comparing its modified steroid, anecortave acetate, to Visudyne. Genentech Inc., of South San Francisco, has Lucentis (ranibizumab), another anti-VEGF product, in Phase III trials for AMD, partnered with Novartis.