Although AEterna Laboratories Inc. missed the primary endpoint in a Phase III trial of Neovastat in renal-cell carcinoma, the company Wednesday said a subgroup of earlier-stage cancer patients demonstrated a significant survival benefit.
Nevertheless, the U.S. market didn't view the data favorably, as the company's stock (NASDAQ:AELA) plunged $2.17 or 33.5 percent, to close at $4.30.
The Phase III study, designed to evaluate 305 patients who were refractory to immunotherapy, failed to hit the primary endpoint of improving overall median survival time by 50 percent. However, a 38-patient subgroup of healthier patients with clear cell histology and only a single metastatic site showed a median survival time of 26.3 months in the treatment arm, compared to 12.6 months in the placebo group (p=0.0236). Neovastat has FDA orphan status in renal-cell carcinoma.
In a conference call Wednesday, Gilles Gagnon, president and CEO of Quebec City, Quebec-based AEterna, characterized the findings as "very interesting," saying the data appear to confirm that when the product is administered at an early stage, it might increase efficacy chances. The data also likely will be helpful in supporting an ongoing Phase III trial of Neovastat in non-small-cell lung cancer.
"This confirms what we have learned over the past few years," Pierre Champagne, AEterna's medical director and vice president, clinical affairs, told BioWorld Today. "The less sick the patients are, and the ones with only one site of metastases, the better they are doing. In NSCLC we are only treating patients without metastases - we are treating them first line. But more than that, my medical feeling is that the role of anti-angiogenesis will be to work with standard treatment [before] metastases."
Neovastat consists of anti-angiogenic components extracted from marine cartilage.
Echoing Gagnon's comments during the conference call, Champagne later said the company received the kidney trial data only Tuesday, thus it is too early to discuss the company's direction regarding Neovastat in renal-cell carcinoma.
When asked by two analysts on the call whether the company would conduct an additional Phase III in the kidney cancer population if requested by the FDA, company officials said they could not respond to hypothetical questions.
As for the next step, Gagnon said the trial data, are "a door opener for discussions with the FDA," adding that he's unsure when the company will meet face to face with the agency. But in the near future, the company will focus on the NSCLC trial.
The renal-cell carcinoma Phase III was a randomized, double-blind, placebo-controlled, three-year trial involving 305 patients who had failed to respond to immunotherapy. According to the company, 153 patients were treated with Neovastat and 152 were given placebo. Overall, the median survival time in the Neovastat group was 12.4 months compared to 12.3 months for the placebo group.
Meanwhile, in NSCLC, AEterna has accrued about one-third of the targeted 760 patients sought for the trial.
Champagne said the company expects to conduct an interim analysis of the trial toward the end of 2005, with an expected completion date of 2006.
Beyond Neovastat, the company's lead compound in endocrinology is Cetrotide, a product sold in the in vitro fertilization market in the U.S. and Europe. The compound is in clinical testing in endometriosis, uterus myoma and enlarged prostate. The company is sponsoring an additional seven clinical programs with various compounds.