What's Ahead?

It's been a little over a year since Ventrus Biosciences Inc. (VTUS), which is developing drugs that address an unmet need in gastrointestinal diseases, went public at $6 per share. This specialty pharmaceutical company, whose stock now trades around $9, has two catalysts in the coming months.

For readers who are new to Ventrus, here's a brief overview of the company and the pivotal trials to watch out for this year...

Ventrus is focused on developing prescription drugs for gastrointestinal disorders, specifically, hemorrhoids, anal fissures and fecal incontinence. The company was formerly known as South Island BioSciences Inc.

The company's product pipeline includes,

* VEN 309 (Iferanserin ointment), a phase III product, for the topical treatment of hemorrhoids

* VEN 307 (Diltiazem cream), a topical treatment for the relief of pain associated with anal fissures, which is also a phase III product

* VEN 308 (Phenylephrine gel), a phase II product, for the treatment of fecal incontinence associated with ileal pouch anal anastomosis.

VEN 309, the investigational treatment of hemorrhoids, is a new chemical entity that targets a specific serotonin receptor (5HT2A) thought to be important in the disease, and is formulated as an ointment for intra-anal application. Ventrus completed its acquisition of all rights and title to VEN 309 from the licensor, Sam Amer & Co., last November.

Hemorrhoids, often known as piles, refer to a condition where veins in the lower portion of the rectum or anus become swollen, accompanied by primary symptoms of bleeding, itching and pain. It is estimated that about 10 million to 20 million Americans have hemorrhoids.

The first of two pivotal phase III trials of VEN 309 began last August and is currently recruiting participants.

The phase III trial of VEN 309 is designed to enroll about 600 patients at 70 centers in the U.S. who will be randomized into three arms - Iferanserin administered intra-anally twice daily for 7 days followed by placebo for 7 days, Iferanserin administered intra-anally twice daily for 14 days, and placebo for 14 days.

The primary endpoint is the cessation of bleeding by the end of day 7 through day 14 and the secondary endpoints are cessation of itching and pain by day 7 through day 14.

In the German phase IIb study of VEN 309, 57% of Iferanserin-treated patients had cessation of bleeding compared to only 20% of placebo-controlled patients. In the same study, 59% of iferanserin-treated patients had cessation of itching compared to just 32% of placebo-controlled patients , while pain ceased at day 7 and did not return by day 14 in 50% of Iferanserin-treated patients versus 18% of placebo-treated patients.

Enrollment in the ongoing US phase III trial of VEN 309 (Iferanserin) in hemorrhoids is expected to be completed around April, and top line data is anticipated in June of 2012.

There are no FDA approved prescription drugs for treating hemorrhoids. Some of the treatment options for hemorrhoids include, banding, sclerosing agents and even surgery. Only prescription topical steroid containing products, e.g., oral Daflon, are approved in the EU and Japan.

Anusol HC is the most common prescription hemorrhoid medication while Preparation H is a well-known over-the-counter treatment for hemorrhoids.

Yet another arrow in the company's quiver for gastrointestinal medications is VEN 307, a proprietary formulation of Diltiazem hydrochloride, for anal fissures. Ventrus licensed the North American rights to VEN 307 from Switzerland-based S.L.A. Pharma.

Diltiazem hydrochloride is a calcium-channel blocker that has been marketed in oral formulations for the treatment of angina and high blood pressure for over two decades.

Anal fissure, a common anal disorder, refers to a tear in the lining of the anal canal and is characterized by severe anal pain, associated with or after bowel movements. In June of 2011, the FDA approved Scotland-based ProStrakan's - now a subsidiary of Japan'sKyowa Hakko Kirin's, Rectiv (nitroglycerin ointment) to treat pain associated with chronic anal fissures. Rectiv is expected to be launched in the first quarter of 2012.

Diltiazem cream, specially mixed for each patient , is prescribed off-label by many gastroenterologists across the U.S. for the treatment of anal fissures. In topical formulation, Diltiazem is only minimally absorbed, at one-tenth the amount of the lowest dose used for cardiovascular treatment.

A phase III study of VEN 307 in patients with anal fissures was initiated in November 2010. Enrollment in this study, which is being conducted in 31 centers in Europe, was completed last December with 465 patients. The clinical trial has 3 arms with patients randomized to receive thrice a day -2% Diltiazem, 4% Diltiazem and placebo for 8 weeks of treatment.

The primary endpoint of the trial is reduction in pain on defecation at week 4 and is measured using a validated scale like Likert and NRS.

VEN 307 has 505(b)(2) regulatory pathway. The 505(b)(2) regulatory pathway allows a company to rely, at least in part, on the FDA's findings of safety and/or effectiveness for a previously approved drug (the "reference drug").

The data from the European phase III trial of VEN 307 for the treatment of anal fissures is expected in May 2012.

The third clinical product of Ventrus is VEN 308 (Phenylephrine gel) for the treatment of fecal incontinence associated with ileal pouch anal anastomosis, or IPAA, an FDA orphan indication. Ventrus licensed the exclusive North American rights to VEN 308 also from S.L.A. Pharma.

Fecal incontinence, which refers to the inability to control bowel movements, is a common consequence of ileal pouch anal anastomosis, or IPAA procedure. The IPAA procedure is a surgical procedure performed after the colon and rectum have been completely removed in patients with ulcerative colitis.

It is estimated that about seven million Americans have fecal incontinence. In May 2011, Solesta, an injectable gel, developed by Oceana Therapeutics, was approved by the FDA through premarket approval process as a Class III medical device to treat fecal incontinence. Solesta is also CE marked and marketed in Europe. Oceana was acquired by Salix Pharmaceuticals Inc. (SLXP) last November for $300 million.

The other treatment options for fecal incontinence include, bulk laxatives, fiber diets, Imodium, which is a treatment for diarrhea, and invasive surgical procedures.

VEN 308 is a gel formulation of Phenylephrine. VEN 308 also will be developed under the 505(b)(2) regulatory pathway because Phenylephrine has been available since the early 1940s in oral and nasal form for the treatment of nasal congestion.

The company plans to initiate the U.S. phase IIb dose ranging study of VEN 308 for IPAA-related fecal incontinence this year.

A quick look at the company's balance sheet...

Since inception on October 7, 2005, Ventrus has incurred losses, and being a development stage company, it has not derived any revenue from product sales to date.

In the nine months ended September 30, 2011, the company's net loss was about $15.5 million or $1.79 per share. At September 30, 2011, Ventrus had zero debt and $53.3 million in cash and cash equivalents.

Shares of Ventrus have gained 18 percent in the last three months and trade around $9. With two high value pivotal data expected in the coming months, VTUS may be worth keeping an eye on.