One stock I am quite conflicted about is PTIE.
On the downside, their launching a Phase III trial in irritable bowel after the skimpiest of Phase II's (single dose, open label, 50 people), suggests something other than a respectable biotech. (Not to mention that in their presentation they somehow forgot to mention that this was just a very low dose of an existing approved drug)
On the other side, their recently announced Phase II trial top line results (all that has been disclosed so far) shows a statistically significant (p<.006) improvement over oxycodone in a 21-day pain study. That's very hard to do, especially given the variability of pain studies, and indicates to me that they have something real. All they really have to do is show efficacy, as safety cannot be a real issue (except for the FDA of course) given the minuscule amount of naltrexone they use.
On the theoretical side, a year-or-so ago I heard second- or third-hand reports from two top pain experts, one who thinks they have something and the other who doesn't believe their work for a moment.
Here's their recent PR:
Pain Therapeutics Launches Pivotal Phase III Study With
Oxytrex(TM)
- Jun 30, 2003 07:15 AM (PR Newswire)
- finance.lycos.com
===========================================================================
350 Patient Phase II Study Meets Primary Endpoint
SOUTH SAN FRANCISCO, Calif., June 30 /PRNewswire-FirstCall/ --
Pain Therapeutics, Inc. (Nasdaq: PTIE), a medical research company, announced
today the initiation of a Phase III clinical study with Oxytrex(TM), the
Company's investigational new drug to treat chronic pain. This pivotal study
will enroll up to 700 patients with chronic, severe low-back pain.
The initiation of this pivotal study follows the successful completion of
a 21-day Phase II study. The Phase II study met its primary efficacy
endpoint, showing a statistically significant reduction in chronic pain using
Oxytrex(TM). In this study, Oxytrex(TM) was superior to oxycodone (p=0.006)
and to placebo (p<0.001) in reducing severe osteoarthritic pain during a
21-day treatment period. These results showcase the ability of Oxytrex(TM) to
outperform oxycodone in a multi-dose, chronic model of pain.
"Today marks a major milestone in the growth of Pain Therapeutics," said
Remi Barbier, president and chief executive officer. "From our pioneering
roots in drug development, I can now begin to envision the Company as a
commercially-focused organization."
Details of the Pivotal Phase III Study
This Phase III study is designed to compare the analgesic effects of
Oxytrex(TM) against oxycodone and placebo over a three-month treatment period.
The trial will enroll up to 700 patients with chronic, severe low-back pain at
20-30 sites across the United States. Patient enrollment may take up to a
year to complete. The study is randomized, double-blinded and
placebo-controlled. The primary endpoint in this study is analgesic efficacy
and will be measured using the same industry-wide standard metrics of pain
that the Company used in its Phase II studies, such as pain reduction and
global assessment.
"In recent years, we have systematically generated a significant body of
data on various opioid agonist/low-dose antagonist compounds," said Nadav
Friedmann, PhD, MD, Pain Therapeutics' chief medical and operating officer.
"We started with novel biology, then worked with animal models, then moved
into single-dose human studies, followed by the successful conclusion of Phase
II studies with Oxytrex(TM). We believe the collective data offer ample
evidence that Oxytrex(TM) is well-tolerated and offers superior analgesia
compared to oxycodone."
Details of the Completed Phase II Study
This study evaluated the safety and efficacy of Oxytrex(TM) relative to
oxycodone over 21 days in patients with severe chronic pain due to
osteoarthritis of the hip or knee. The study was randomized, double-blinded
and placebo-controlled. Over 350 male or female patients were enrolled in
multiple centers across the United States. Patients were titrated with an
initial dose of Oxytrex(TM), oxycodone or placebo and were dose-escalated
during the 21-day treatment period. Dose and dose escalation were consistent
across all treatment arms. Safety, pharmacokinetic and efficacy parameters
were assessed at baseline and during the treatment period. The primary
efficacy endpoint was prospectively defined as pain reduction from baseline to
21 days. The study met its primary endpoint with statistical significance.
Oxytrex(TM) was clinically superior to both oxycodone (p=0.006) and to placebo
(p<0.001) in reducing chronic pain over 21 days. In addition, Oxytrex(TM) was
well-tolerated throughout the study. No unusual adverse events or safety
issues were observed. The Company expects to release further details of this
study (pending additional safety and efficacy analysis) at an appropriate
scientific meeting or publication in a peer reviewed journal.
About Opioid Drugs
Opioid drugs (sometimes called 'narcotic painkillers') are derived from
the opium poppy plant. These drugs are widely used throughout the world to
treat severe pain. In the United States, for example, sales of opioid drugs
exceed $3 billion per year. Despite widespread use, opioid drugs have
debilitating effects that limit their true potential to treat pain. Acute use
often results in side effects, such as nausea or vomiting. Chronic use may
lead to tolerance, dependence, abuse, or, more rarely, addiction. As a
result, some patients prefer to suffer through pain rather than to endure the
ill effects of opioid drugs. The under-treatment of chronic pain is a serious
and growing problem in the U.S. For example, according to the National
Institutes of Health, over 40 million Americans are unable to find relief from
their pain.
About Pain Therapeutics, Inc.
We are a medical research company specializing in the discovery and
development of novel drugs. We believe one of our lead drug candidates,
Oxytrex(TM), may offer more pain relief (with no increase in side-effects) or
lower tolerance/dependence, withdrawal effects or addiction potential compared
to conventional forms of oxycodone. The market for oxycodone exceeded
$1.5 billion in the United States in 2002. Another drug candidate, PTI-901,
is aimed at treating patients who suffer from abdominal pain associated with
Irritable Bowel Syndrome (IBS). The U.S. market for IBS is believed to exceed
$1 billion.
Peter |
