Good News For Promising Carcinoid Syndrome Drug

Lexicon Pharmaceuticals announced today that the FDA accepted its  New Drug Application application for telotristat etiprate.

According to the company website, “The FDA has granted a Priority Review of the NDA filing and set a Prescription Drug User Fee Act (“PDUFA”) target action date of November 30, 2016.”

“The granting of priority review by the FDA underscores the need for improving the lives of the patients and caregivers who live with carcinoid syndrome on a daily basis,” said Lexicon President and Chief Executive Officer, Lonnel Coats. “If approved, telotristat etiprate would be the only approved therapy for patients who are no longer able to control their carcinoid syndrome with the current standard of care alone.”

Carcinoid syndrome is a rare disease affecting thousands of cancer patients with metastatic neuroendocrine tumors (mNETs) that have spread to the liver and other organs from the gastrointestinal tract. The condition is characterized by frequent and debilitating diarrhea that often prevents patients from leading active, predictable lives, as well as facial flushing, abdominal pain, fatigue and, over time, heart valve damage.

Telotristat etiprate is the first investigational drug in clinical studies to target tryptophan hydroxylase, an enzyme that triggers the excess serotonin production within mNET cells that leads to carcinoid syndrome.”

The current standard of care for carcinoid syndrome is  somatostatin analogue therapy given by injectables which reduces the release of serotonin outside of tumor cells.

Telotristat etiprate works in a different way, at the source to reduce serotonin production within the tumor cells. By specifically inhibiting serotonin production, telotristat etiprate seeks to control this important driver of carcinoid syndrome and, in turn, provide patients with more control over their disease.

In August, as the NANETS meeting in Austin,  the phase III TELESTAR study was reported. In this study  of patients with carcinoid syndrome, telotristat etiprate decreased mean daily bowel movements by 35% among participants (n=135) who received 500 mg of the drug three times a day and 29% among those who took 250 mg three times a day, compared with 17% for individuals who received a placebo. The results, measured from baseline to 12 weeks, were statistically significant
(P <.001). Click here to read more at OncLive.

Telotristat etiprate has received Fast Track and Orphan Drug designation from the U.S. Food and Drug Administration.

 

 

 

Advertisements

Practice Changing Clinical Trials Presented At The North American Neuroendocrine Tumor Society 2015 Meetings

logo_0.png

I had the opportunity recently to attend the 2015 scientific symposium of the North American Neuroendocrine Tumor Society in Austin, TX. It’s possible I was the only actual carcinoid/NET patient there.

The Healing net Foundation funded my educational experience as part of a grant program to spread information about carcinoid and neuroendocrine cancers to community physicians.

I had the opportunity to talk with leading NET physicians from around the world and listen to presentations on the state of the art of treatment and diagnosis of NET. My surgeon, Dr. James Howe chaired one of the sessions. Dr. Thomas O’dorisio my carcinoid doctor was present and participated on discussion panels.

Exciting results from treatment trials were presented.

As summarized by NANETS:

The results of three recent, practice-changing phase III clinical trials were presented for the first time in North America at the NANETS symposium which included: (1) TELESTAR (randomized phase III trial of Telotristat vs. Placebo in patients with carcinoid syndrome), (2) NETTER-1 (randomized phase III trial of 177-Lu-DOTATATE vs. high dose octreotide in patients with progressive midgut NETs) and (3) RADIANT-4 study (randomized phase III trial of everolimus vs. placebo in advanced non-functional GI and lung NETs). NANETS will continue to keep you informed on the progress of the regulatory submissions for all three of these treatments.

Following are brief descriptions of the data:

Trial 1: In the phase III TELESTAR study, telotristat etiprate decreased mean daily bowel movements by 35% among participants (n=135) who received 500 mg of the drug three times a day and 29% among those who took 250 mg three times a day, compared with 17% for individuals who received a placebo. The results, measured from baseline to 12 weeks, were statistically significant
(P <.001). Click here to read more at OncLive.

Trial 2: The phase III NETTER-1 trial compared the efficacy of 177-Lu-Dotatate (Lutathera) (n=11) with octreotide 60 mg (Sandostatin LAR) (n=11) in patients with advanced progressive disease. Results showed that the median progression-free survival (PFS), the trial’s primary endpoint, improved by nearly 80% (HR .21). The median with high-dose octreotide was 8 months and was not yet reached in the 177-Lu-Dotatate arm at a median follow-up of 18 months.
Click here to read more at OncLive.

Trial 3: The phase III RADIANT-4 trial was a study of advanced, progressive, nonfunctional lung/GI NETs. In the study, 302 patients with progressive, well-differentiated, nonfunctional lung/GI NETs were randomized 2:1 to receive best supportive care plus either everolimus at 10 mg per day
(n = 205) or placebo (n = 97). Tumors were located in the GI tract (n = 175), lung (n = 90), or were of unknown origin (n = 36). The primary endpoint was progression free survival (PFS); the progression free survival was 11 months on the everolimus arm compared to 3.9 months in the placebo arm. Click here to read more at OncLive.