Thetis Receives Orphan Drug Designation For TP-252 In Familial Adenomatous Polyposis
SOUTHPORT, CT--(Marketwired - Jan 19, 2016) - Thetis Pharmaceuticals LLC (Thetis or the Company), a privately-held biopharmaceutical company focused on gastrointestinal (GI) and cardiometabolic (CM) diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to TP-252, the Company's development candidate for the treatment of familial adenomatous polyposis (FAP).
"This is an important milestone for Thetis," stated Dr. Frank Sciavolino, President and Chief Scientific Officer of the Company. "Prophylactic colectomy in adolescent FAP patients imposes a substantial psychosocial burden on their lives along with multiple post-surgical complications. Based on its novel mechanism of action for reducing polyp burden, TP-252 has the potential to become a safe and effective therapy to delay colectomy in this young population seeking to transition into adulthood with an intact colon."
About Orphan Drug Designation
The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent orphan diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain financial incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.
About FAP
Familial adenomatous polyposis is a rare genetic disease characterized by the formation of numerous large polyps in the bowel in late childhood or adolescence. Unless these polyps are removed, FAP patients inevitably develop colorectal cancer, typically in their late 30s or early 40s. There are no drugs approved by FDA for the treatment of FAP. Screening, surveillance and prophylactic colectomy constitute the current standard treatment for the management of this disease. Prophylactic surgery, which is usually performed between the ages of 16 and 20 years, carries significant risk of gastrointestinal problems, reproductive issues, and psychosocial burden.
About TP-252
TP-252 is a novel, mineral amino acid-based derivative of eicosapentaenoic acid (EPA) that delivers high plasma levels of EPA free fatty acid (EPA-FFA). Clinical proof-of-concept demonstrating that EPA-FFA reduces polyp burden in FAP patients has been reported in a Phase II/III randomized, double-blind, placebo-controlled trial completed in 2008 (West et. al. Gut 2010; 59:918-25). Thetis expects to begin clinical trials in 2017 to study TP-252 as adjunct therapy to endoscopic surveillance for the treatment of young FAP patients.
About Thetis Pharmaceuticals
Thetis Pharmaceuticals is a privately held biopharmaceutical company applying its HEALER™ technology to discover and develop innovative drugs. Thetis chemically transforms bioactive fatty acids into solid, stable, small molecule pharmaceuticals that target unmet medical needs in the GI and CM areas. The early-stage Thetis pipeline, which has composition-of-matter patent protection and potential first-in-class therapeutic credentials, includes TP-252 for treatment of familial adenomatous polyposis, TP-452 as adjunct therapy to statins for management of LDL-cholesterol, TP-113 for treatment of type 2 diabetes, and TP-4311 for treatment of post-operative ileus.
"This is an important milestone for Thetis," stated Dr. Frank Sciavolino, President and Chief Scientific Officer of the Company. "Prophylactic colectomy in adolescent FAP patients imposes a substantial psychosocial burden on their lives along with multiple post-surgical complications. Based on its novel mechanism of action for reducing polyp burden, TP-252 has the potential to become a safe and effective therapy to delay colectomy in this young population seeking to transition into adulthood with an intact colon."
About Orphan Drug Designation
The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent orphan diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain financial incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.
About FAP
Familial adenomatous polyposis is a rare genetic disease characterized by the formation of numerous large polyps in the bowel in late childhood or adolescence. Unless these polyps are removed, FAP patients inevitably develop colorectal cancer, typically in their late 30s or early 40s. There are no drugs approved by FDA for the treatment of FAP. Screening, surveillance and prophylactic colectomy constitute the current standard treatment for the management of this disease. Prophylactic surgery, which is usually performed between the ages of 16 and 20 years, carries significant risk of gastrointestinal problems, reproductive issues, and psychosocial burden.
About TP-252
TP-252 is a novel, mineral amino acid-based derivative of eicosapentaenoic acid (EPA) that delivers high plasma levels of EPA free fatty acid (EPA-FFA). Clinical proof-of-concept demonstrating that EPA-FFA reduces polyp burden in FAP patients has been reported in a Phase II/III randomized, double-blind, placebo-controlled trial completed in 2008 (West et. al. Gut 2010; 59:918-25). Thetis expects to begin clinical trials in 2017 to study TP-252 as adjunct therapy to endoscopic surveillance for the treatment of young FAP patients.
About Thetis Pharmaceuticals
Thetis Pharmaceuticals is a privately held biopharmaceutical company applying its HEALER™ technology to discover and develop innovative drugs. Thetis chemically transforms bioactive fatty acids into solid, stable, small molecule pharmaceuticals that target unmet medical needs in the GI and CM areas. The early-stage Thetis pipeline, which has composition-of-matter patent protection and potential first-in-class therapeutic credentials, includes TP-252 for treatment of familial adenomatous polyposis, TP-452 as adjunct therapy to statins for management of LDL-cholesterol, TP-113 for treatment of type 2 diabetes, and TP-4311 for treatment of post-operative ileus.