Tranzyme Pharma (NASDAQ:TZYM) can measure the phase 3 clinical trial failure of its gastrointestinal drug ulimorelin by counting hours. The preliminary results don’t suggest that the compound did not work at all. Rather, post-surgical hospital care has apparently improved recovery time quite a bit.
Time, more specifically time saved, has always been a focus for drug candidate ulimorelin. The compound was developed to restore normal bowel function following surgery. It’s common for GI surgery patients to experience bowel dysfunction after the surgery, a condition called postoperative ileus, or POI. Patients who have POI can’t eat solid food or have have normal bowel movements for a period that lasts anywhere from days to up to two weeks. All of that time is spent in a hospital.
POI’s best treatment has been time. But two weeks in a hospital costs a lot of money. Ulimorelin was developed with the expectation that reducing post-surgical recovery time would cut healthcare by shortening hospital stays. When Durham, North Carolina-based Tranzyme completed phase 2 clinical trials on ulimorelin four years ago, the time from surgery to the first bowel movement and tolerance of solid food was between 68 and 75 hours. Under a placebo, that time was 92 hours.
In designing the phase 3 trials, Tranzyme estimated that the placebo arm of the trial would see GI function restored in 96 hours, CEO Vipin Garg said on a conference call to discuss the trial results. The median time for resoration of GI function under ulimorelin was 80 hours. But 80 hours was also the median time for patients treated with placebo to see restored function.
“There seems to be a progression toward an evolving standard of care, there have been other data to support that,” Garg said. “That’s the hypothesis we’re working with right now, the effect of the progressively evolving standard of care.”
The phase 3 results announced Monday were the first of two phase 3 studies. Results of the second double blind study are expected by the end of the second quarter. But with no statistical significance distancing ulimorelin from placebo in the initial results of the first phase 3 trial, Tranzyme has halted plans to file a new drug application for ulimorelin with the U.S. Food and Drug Administration.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Tranzyme is now shifting its emphasis to TZP-102, a compound developed to treat diabetic gastroparesis. Gastroparesis is a condition in which the stomach muscular contractions to move food into the intestines either slows or stops. It can be caused by any disease that has a neuromuscular effect on the GI tract. Diabetes is among the leading causes of gastroparesis. No new treatment for diabetic gastroparesis has been introduced in nearly 20 years and the FDA has granted fast track status for the Tranzyme compound. A TZP-102 phase 2 trial is currently enrolling and top-line data is expected by the end of the year.
Anaylsts are concerned about any simliarities that TZP-102 might have to ulimorelin. The two compounds work by targeting the same receptor. But Garg said that the compounds are different and are covered by separate intellectual property. Garg sought to further distinguish TZP-102 from ulimorelin. Garg said that ulimorelin is a short term treatment. POI patients will ultimately reach the endpoint of normal GI function. Ulimorelin was supposed to help them get there faster. But diabetic gastroparesis is a chronic condition.
“These patients are not going to get better,” Garg said. “So while you expect placbo effect in a chronic setting like that, ultimately the drug, if it’s effective, will be able separate itself away from any placebo effect.”
In other words, time won’t be the main factor for evaluating TZP-102. It either works, or it doesn’t.
[Photo by stock.xchng user cozgrl05]
