Biostage Receives FDA Orphan Drug Designation For Cellspan Esophageal Implant

HOLLISTON, Mass., Dec. 1, 2016 /PRNewswire/ -- Biostage, Inc. (Nasdaq: BSTG), ("Biostage" or the "Company"), a biotechnology company developing bioengineered organ implants to treat cancers and other life-threatening conditions of the esophagus, bronchus and trachea, today announced that its Cellspan Esophageal Implant was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) to restore the structure and function of the esophagus subsequent to esophageal damage due to cancer, injury or congenital abnormalities.

The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent 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 upon marketing approval, as well as certain incentives, including tax credits and a waiver of PDUFA filing fees.          

"The Orphan Drug Designation for our Cellspan Esophageal Implant is an important achievement in our regulatory strategy as we advance this novel product candidate that we believe, has the opportunity to create a significant treatment paradigm shift in regenerative medicine," said Jim McGorry, CEO of Biostage. "Based on preclinical, large-animal studies conducted to date, we believe our Cellspan esophageal implants may have the potential to offer improved outcomes for patients by reducing post-operative complications and providing an improved quality of life, by prompting regeneration of the patient's own esophagus."

The Company recently provided a regulatory update of its Cellspan esophageal implant following its substantive pre-IND meeting with the FDA. The feedback provided by the FDA to Biostage's proposed clinical protocol for its esophageal implant provided clarity for a clinical path forward for its intended Investigational New Drug (IND) application and the advancement into Phase 1 human clinical studies. The Company expects to file its IND in the third quarter of 2017 and commence its Phase 1 first-in-human clinical study in the fourth quarter of 2017.

"We believe this Orphan Drug Designation and the regulatory guidance we recently received from the FDA, provide a clear development pathway which may potentially enable us to offer a promising treatment outcome for patients suffering from esophageal cancer. Our focus is steadfast on emerging as rapidly as possible as a clinical-stage company," concluded Mr. McGorry.

About Cellframe Technology
The Company's proprietary Cellframe technology is designed to harness the full potential of the in vivo microenvironment to achieve tissue regeneration and restore organ function. It employs a multi-step process in which the patient's own stem cells are taken from a simple adipose/fat tissue biopsy, expanded and banked, and then seeded onto a proprietary scaffold that mimics the natural dimensions of the organ being regenerated. After several days in a rotating bioreactor, the biocompatible scaffold containing the stem cells is ready to be implanted. Preclinical studies suggest that the organ implant signals the stem cell niche in the surrounding native tissue to guide the regeneration of a biological structure. This technology is based on the concept of in situ tissue regeneration using the body's own biologic resources and reparative capability in combination with tissue-specific biomaterials implanted at the sites of disease or injury.

About Cellspan Esophageal Implants
Cellspan Esophageal Implants utilize the Company's proprietary Cellframe technology and may offer improved outcomes for patients by potentially simplifying surgical techniques to reduce post-operative complications and improve quality of life, by prompting regeneration of the patient's own esophagus. Cellspan implants are intended to offer numerous advantages over standard surgical resection including: eliminating the use of the stomach or intestine to create a mock esophagus, reduced complications and improve post-surgical morbidity.

About Biostage
Biostage is a biotechnology company developing bioengineered organ implants based on the company's new Cellframe technology which combines a proprietary biocompatible scaffold with a patient's own stem cells to create Cellspan organ implants. Cellspan implants are being developed to treat life-threatening conditions of the esophagus, bronchus or trachea with the hope of dramatically improving the treatment paradigm for patients. Based on its preclinical data, Biostage has selected life-threatening conditions of the esophagus as the initial clinical application of its technology.

Cellspan implants are currently being advanced and tested in collaborative preclinical studies. Preclinical, large-animal safety studies, conducted in compliance with the FDA Good Laboratory Practice (GLP) regulations, for the Company's Cellspan Esophageal Implant product candidate are ongoing, in support of Biostage's goal of filing an Investigational New Drug (IND) application with the U.S. FDA in the third quarter of 2017. The IND will seek approval to initiate clinical trials for its esophageal implant product candidate in humans.

For more information, please visit www.biostage.com and connect with the Company on Twitter and LinkedIn. 

Forward-Looking Statements:
Some of the statements in this press release are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements in this press release include, but are not limited to, statements relating to the development expectations and regulatory approval of any of our products, including those utilizing our Cellframe technology, by the U.S. Food and Drug Administration, the European Medicines Agency or otherwise, which expectations or approvals may not be achieved or obtained on a timely basis or at all; or success with respect to any collaborations, clinical trials and other development and commercialization efforts of our products, including those utilizing our Cellframe technology,  which such success may not be achieved or obtained on a timely basis or at all. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, our ability to obtain and maintain regulatory approval for our products; plus other factors described under the heading "Item 1A. Risk Factors" in our Annual Report on Form 10-K for the fiscal year ended December 31, 2015 or described in our other public filings. Our results may also be affected by factors of which we are not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. Biostage expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions or circumstances on which any such statement is based.

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SOURCE Biostage, Inc.