And speaking of skimpy Phase II's prior to Phase III's, here's what I would consider the ultimate poster child:
Press Release Source: Pain Therapeutics, Inc.
Pain Therapeutics Initiates 1,200 Patient Phase III Program in Irritable Bowel Syndrome
Tuesday November 25, 7:31 am ET
SOUTH SAN FRANCISCO, Calif., Nov. 25 /PRNewswire-FirstCall/ -- Pain Therapeutics, Inc. (Nasdaq: PTIE - News), a biopharmaceutical company, today announced the initiation of a large Phase III clinical program with PTI-901. PTI-901 is the Company's investigational new drug to treat men or women who suffer from irritable bowel syndrome (IBS), a chronic, painful abdominal disorder that predominantly affects women. The program will enroll a total of 1,200 IBS patients in 80 clinical sites across the United States.
"This Phase III program marks a significant milestone in the development of PTI-901," said Remi Barbier, president and chief executive officer. "We believe PTI-901 offers a potential break-through strategy to treat patients who suffer from IBS. There are over 20 million such patients in the United States. A safe and efficacious drug to treat this patient population could represent a $1 billion opportunity."
Pain Therapeutics and the U.S. Food and Drug Administration met recently to discuss the Company's Phase III program. During these discussions both parties agreed upon the design and appropriateness of this Phase III program with PTI-901.
"PTI-901 really represents a new approach to treat patients who suffer from IBS," said Nadav Friedmann, Ph.D., MD, chief operating and medical officer. "At this time there are no approved drugs for men with IBS and there are limited choices for women. With the right clinical results, and regulatory approval, we believe PTI-901 can address a huge unmet medical need."
Study Detail
The Company's Phase III program in IBS consists of two clinical studies that are identical in all respects except for gender. One study will enroll only men while the other will enroll women. The two studies will enroll a total of 1,200 patients: 600 men and 600 women diagnosed with IBS by a gastroenterologist according to the Rome II Criteria. Each study is randomized, double-blinded, placebo controlled and is designed to compare the clinical effects of PTI-901 against placebo during a three-month treatment period. Male and female patients with constipation-predominant, diarrhea-predominant or alternating symptoms of IBS will be considered for this study.
Patients will be evaluated for abdominal pain, bowel habits and stool consistency at baseline and during the treatment period. Treatment consists of a single, daily, 0.5 mg oral dose of PTI-901 or matching placebo. The primary efficacy endpoint is relief of IBS symptoms, as measured by patients' observations of Subject Global Assessment of Relief. Secondary efficacy endpoints include improvements in abdominal pain, bloating, bowel habits and quality of life. Safety will be evaluated through the assessment of various adverse events.
About PTI-901
PTI-901 (low-dose naltrexone HCI) is the first in a new class of drugs designed to restore the balance of opioid activity in the gut. It is believed that an imbalance of opioid activity in the gut contributes to the symptoms that comprise IBS. Such imbalance may be triggered by emotional stress, metabolic disorders or intrinsic release of opioids from neurons in the gut. By restoring this balance, PTI-901 may relieve abdominal pain and other symptoms frequently observed in patients with IBS. Pain Therapeutics holds exclusive, worldwide commercial rights to a family of issued patents and patent applications directed to the long-term treatment of IBS patients with proprietary opioid antagonists, such as PTI-901.
Results from a 50 patient pilot study with PTI-901 in men and women were presented at the American College of Gastroenterology meeting in October 2003. In this study, patients with IBS reported a 76% response rate to PTI-901. This response rate was observed in men and women and occurred without drug-related safety issues.
About Irritable Bowel Syndrome
IBS is a chronic, painful abdominal disorder that leads to major changes in bowel habits. IBS causes some patients to have constipation, diarrhea or in some cases both. The cause of IBS is not known, and as yet there is no cure. People with chronic IBS may be unable to attend social events, hold a job, or travel away from home. Over 10 percent of the U.S. population suffers from IBS. For unknown reasons, IBS predominantly affects women.
Now I'm considerably conflicted here - this approach is very interesting and the stock will be a better than 10-bagger if the trial works. But consider that the single Phase II was a non-randomized, single-dose, non-placebo-controlled, foreign, unblinded 50-person study:
Study Design and Clinical Results
This open-label study was designed to assess the safety and efficacy of PTI-901 in IBS patients. A total of 50 male or female patients, all diagnosed with IBS by a gastroenterologist according to Rome II Criteria, were enrolled in the study. Treatment consisted of a 0.5 mg dose of PTI-901 given daily over a four-week period. Patients were evaluated for global response to treatment, abdominal pain, bowel habits and stool consistency at baseline, in weeks 1 through 4 and during a subsequent four-week follow-up period. The study was conducted by the staff at the Department of Gastroenterology and Liver Disease at the Sourasky Medical Center in Israel, under an Investigational New Drug (IND) Application filed with the FDA by the Company.
The efficacy of PTI-901 was assessed in weeks 1 through 4 using the patients' observations of "Global Assessment of Adequacy of Treatment." The primary endpoint was prospectively defined as patients reporting a weekly global assessment score of fair or better at week 4 compared to baseline. The male and female response rate to the primary endpoint was 77.8% (N=18) and 75% (N=28) respectively. Secondary efficacy endpoints included measurements of abdominal pain and bowel habits. Patients taking PTI-901 reported over 140% increase in number of pain-free days at week 4 compared to baseline (N=42). Clinical improvements in bowel urgency, stool consistency and number of stools per day were also reported at week 4 in both genders. PTI-901 was well tolerated by all patients during the entire treatment period.
(The company argued that the near 80% response rate is way higher than the placebo rate in comparable studies, which is indeed true).
Despite all this I confess to owning a (very little) stock bought at lower levels. What can I say by way of disclaimer? Don't try this at home? <g>
Peter |
