FDA Grants PharmassêtX Orphan Drug Designation for Treatment of Rare Form of Inflammatory Bowel Disease with Active Component from Green Tea

Clinical trials slated to begin next year in collaboration with global leader

LOUISVILLE, Ky., July 10, 2024 /PRNewswire/ -- PharmassêtX Inc., a late-preclinical stage pharmaceutical development company, today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to epigallocatechin gallate (EGCG), a well-characterized component of green tea that the Company is developing as a high-purity, high-potency drug for the treatment of inflammatory bowel disease (IBD).

FDA granted PharmassêtX ODD for the treatment of pouchitis, a rare yet debilitating form of IBD experienced by a number of patients who have undergone surgery to remove their large intestine. There is currently no FDA-approved drug for the treatment of pouchitis.

"We are grateful to be positioned to provide a much-needed innovative drug, for which there is already substantial evidence of safety and effectiveness, to the underserved community of pouchitis patients," said Terry Minton, Co-Founder and CEO of PharmassêtX. "Our mission as an organization is to improve outcomes and enhance quality of life for IBD patients. The granting of this ODD is an important milestone along the critical path to accomplishing that mission."

Peer-reviewed evidence supporting EGCG's safety and effectiveness for an IBD indication was first published in the journal Inflammatory Bowel Diseases, a publication of the Crohn's and Colitis Foundation. A randomized, double-blinded, placebo-controlled, investigator-initiated clinical study of an EGCG-enriched green tea extract showed that patients suffering from mild to moderate ulcerative colitis, a common form of IBD, experienced clinical improvement, including endoscopic improvement, after treatment. No serious adverse events were reported.

Further peer-reviewed evidence of EGCG's safety and effectiveness was gathered in the course of an investigator-initiated retrospective study of pouchitis patients published in International Journal of Colorectal Diseases, a Springer Nature publication. The majority of refractory pouchitis patients experienced complete relief from their symptoms, with no serious adverse events reported.

Such studies, initiated and completed prior to the formation of the Company by one of its founders, Gerald W Dryden, Jr, MD, PhD, gastroenterologist/immunologist and Chief Medical Officer of PharmassêtX, established the basis for the Company's founding. Dr Dryden offered this perspective: "My clinical experience, consistent with the clinical experience of prominent and knowledgeable colleagues both in the US and globally, indicates that EGCG is safe and effective for the treatment of various forms of IBD. It is unfortunate that previous development efforts to harness partially pure EGCG for the chemoprevention of cancer – a noble goal, but a high bar indeed – were abandoned. That decision was made not because of lack of evidence of efficacy or safety; rather, a change in leadership at the former corporate sponsor, a prominent tea producer, led to their change of heart. The rigorous demands and high costs of pharmaceutical development fell outside of their corporate risk tolerance. Ever since, there has been a pressing need to revive the development of EGCG as a drug to treat conditions such as IBD, including pouchitis. PharmassêtX was founded to meet that unmet medical need."

EGCG has been shown to target NF-κB, a key regulator of mucosal immunity, which promotes the expression of multiple pro-inflammatory genes dysregulated in IBD. Additional effects may include decreased activation of the NLRP3 inflammasome, decreased secretion of pro-inflammatory interleukins, and promotion of intestinal barrier integrity. Studies in relevant IBD models suggest that targeting NF-κB can be an effective strategy in disease management, whether alone or as an adjunct in combination with other therapeutics.

To advance its proprietary ultra-pure EGCG drug PSX-514 into the clinic as efficiently as possible, PharmassêtX has recently partnered with Alimentiv, a Canada-based global contract research organization recognized as the leader in designing and executing clinical trials of novel therapeutics for gastrointestinal indications, including various forms of IBD such as Crohn's disease, ulcerative colitis, and pouchitis. The collaboration with Alimentiv is intended to expedite patient access to PSX-514 and advance the drug into the clinic next year. Such clinical validation is required for eventual regulatory approval and, importantly, so that public and private insurers will have data supporting coverage and payment for PSX-514 prescriptions on the basis of medical necessity. Per-insured per-year costs for IBD patients are significantly higher than for insured patients in general, indicating the need for more innovative and cost-effective therapeutics.

About inflammatory bowel disease: A groundbreaking 2023 study led by the Crohn's and Colitis Foundation showed that nearly 1 in 100 Americans has IBD. "IBD is an umbrella term used to describe disorders that cause chronic inflammation of the gastrointestinal (GI) tract. Symptoms include diarrhea, as well as abdominal pain, nausea, fever, loss of appetite, fatigue and at times rectal bleeding. The two most common forms of IBD are Crohn's disease and ulcerative colitis. There are currently no cures for IBD." See the Crohn's and Colitis Foundation website, https://www.crohnscolitisfoundation.org/groundbreaking-study-led-the-crohns-colitis-foundation-estimates-nearly-1-100-americans-has.

About pouchitis: "Pouchitis is the most common complication after restorative proctocolectomy with ileal pouch–anal anastomosis for ulcerative colitis." See "AGA Clinical Practice Guideline on the Management of Pouchitis and Inflammatory Pouch Disorders" at https://www.giboardreview.com/wp-content/uploads/2023/12/AGA-Clinical-Guidleine-Pouchitis-Jan-2024.pdf.

About Orphan Drug Designation: "Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including: tax credits for qualified clinical trials; exemption from user fees; potential seven years of market exclusivity after approval." See the FDA website, https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products.

About EGCG: Epigallocatechin gallate (EGCG) is a natural substance obtained from the tea plant Camellia sinensis. As evidenced by over four thousand peer-reviewed publications just in the past decade, EGCG has become the object of extensive research into its health effects and other properties. See the National Institutes of Health National Center for Biotechnology Information PubChem website, https://pubchem.ncbi.nlm.nih.gov/compound/Epigallocatechin-Gallate.

About Gerald W Dryden, Jr, MD, PhD: Dr Dryden is Professor of Medicine at the University of Louisville School of Medicine, inventor of a number of endoscopic devices, co-author of dozens of publications on IBD, and a frequent invited speaker at gastroenterology conferences focused on improving IBD treatment.

About Terry Minton: Mr Minton is an experienced healthcare executive, working most recently in pharmacogenomics and diagnostics, who has launched multiple startups including oncology drug developer Aptamera, which was acquired for a premium yielding a highly attractive ROI for its investors.

About Alimentiv: Alimentiv is an industry-leading specialty GI contract research organization (CRO) improving clinical trial outcomes and accelerating drug development timelines. As a global CRO offering clinical, medical imaging and precision medicine services, Alimentiv partners with pharmaceutical and biotechnology industries to advance the development of novel therapies. Alimentiv is headquartered in London, Ontario, Canada, with a global footprint across its operations in Canada, the United States, Europe, Asia-Pacific, and Latin America. For more information: www.alimentiv.com.

About PharmassêtX: Founded in 2024 with a mission to address the needs of underserved populations within the IBD community, Louisville, Kentucky-based PharmassêtX Inc. (pronounced "Farm-a-SET-x") is a privately held Delaware corporation. The Company welcomes substantive inquiries from interested parties.

Contact: Terry Minton, CEO, PharmassêtX, [email protected] or (502) 324-5111.

Note: This press release contains forward-looking statements, including without limitation statements about future developments, which are subject to significant risks and uncertainties. Actual future developments may differ materially due to various factors. PharmassêtX Inc. undertakes no obligation to update or revise any forward-looking statement. No reliance should be placed on any forward-looking statement contained in this press release.

SOURCE PharmassêtX Inc.