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Innovate Seeks Orphan Drug Status for Ulcerative Colitis Therapy

By Barry Teater, NCBiotech Writer

Innovate Biopharmaceuticals, a clinical-stage biotechnology company based in Raleigh, has submitted an application to the U.S. Food and Drug Administration (FDA) seeking orphan drug designation for an oral therapy to treat pediatric ulcerative colitis.

The therapy, called INN-108, is an oral small molecule that combines two agents:  mesalamine, or 5-aminosalicylic acid, an approved agent for ulcerative colitis; and 4-aminophenylacetic acid, an immunomodulatory agent approved in Japan for rheumatoid arthritis.

Together, the chemically bonded agents could be a more effective drug combination than mesalamine treatments alone, according to preliminary data, the company said in a press release.

“Orphan drug designation would expedite our development of a therapy that we believe could bring relief to children and teenagers suffering from active ulcerative colitis,” said Innovate’s CEO, Chris Prior, Ph.D. “There is an unmet need for an effective treatment for this disease, and INN-108 is a promising potential therapy. Our FDA submission is a major milestone toward making this therapy available.”

Under the Orphan Drug Act of 1983, the FDA provides incentives for companies developing treatments that are expected to provide significant therapeutic advantage over existing treatments and that target rare medical conditions affecting fewer than 200,000 U.S. patients per year. Incentives include seven-year market exclusivity, tax credits on U.S. clinical trials, fast-tracking of regulatory proceedings, and exemption from certain fees.

INN-108 has successfully completed two Phase 1 clinical trials in the United States for mild to moderate ulcerative colitis in both healthy adult subjects and adults with ulcerative colitis. A Phase 2 trial will begin in 2018, Innovate said.

A liquid oral formulation of INN-108 is in development for pediatric use.

Ulcerative colitis is a chronic disease that mainly affects the large intestine, or colon. Symptoms typically include constant diarrhea mixed with blood, abdominal pain, increased bowel movements, and in severe cases, weight loss and fatigue. Children can also develop unique complications related to growth, development, nutrition, pubertal maturation, bone mineral density accretion and psychological impacts.

Though the disease can present at any age, it often begins in teenagers and young adults. Various studies estimate that there are between 13,700 and 87,600 cases of pediatric ulcerative colitis in the United States, with most of those occurring in children and teenagers aged 10 to 17.

More than 80 percent of patients have the mild to moderate form of ulcerative colitis that is currently treated with various formulations of mesalamine, a $2 billion market in the United States. Due to the high failure rates (40 to 50 percent) of mesalamine treatments, new therapies for ulcerative colitis are needed, Innovate said.

Other products in pipeline

Innovate is also developing a late-stage drug for celiac disease and expects to begin Phase 3 clinical trials later this year. The drug, larazotide acetate, or INN-202, is a novel oral peptide that has the potential to become the first approved medicine for celiac disease. It has been granted "Fast Track" designation from the FDA to speed its regulatory review.

Celiac disease, like ulcerative colitis, is a type of inflammatory bowel disease. It is triggered by the ingestion of gluten, a protein in certain food grains. Resulting inflammation damages the inner lining of the small intestine, causing intestinal pain, bloating and diarrhea.

These symptoms can be reduced, and sometimes eliminated, by avoiding foods that contain gluten. However, an estimated half of celiac patients remain symptomatic despite a gluten-free diet, most likely due to unintentional ingestion of gluten, according to a patient registry sponsored by the Celiac Disease Foundation.

These patients may benefit from drug therapy. Currently, there are no drugs available to treat celiac disease, which affects 3 million people in the United States and about 15 million worldwide.

Innovate also has an imaging aid, Secretin, in Phase 3 trials. Secretin is indicated for use in magnetic resonance cholangiopancreatography (MRCP) procedures to help visualize disease in the pancreatic ducts.

More than 400,000 MRCP procedures are performed annually in the United States.

Merger pending

Earlier this month, Innovate announced it had signed a definitive merger agreement with California-based Monster Digital under which the shareholders of privately held Innovate will become the majority owners of Monster Digital, subject to shareholder approval.

The combined company will be traded on the NASDAQ stock exchange as Innovate Biopharmaceuticals and will be led by Innovate's management team. The transaction is expected to close in 2017.

Jay Madan, Innovate’s co-founder and president, said the merger “provides us an opportunity to access public markets faster than a traditional IPO (initial public offering). Innovate’s main objective is to quickly advance our celiac disease and ulcerative colitis therapeutics through their respective clinical trials to bring much-needed therapies to the patients. As Innovate moves forward in the clinic, we are also working to enrich our pipeline by investigating other potential indications of our existing molecules and by in-licensing additional assets.”

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