Orgenesis wins Orphan Drug designation for AIP cells for treating severe diabetes

CEO Vered Caplan says the designation will reduce time and costs of clinical development, helping the company get its AIP cell therapy to market faster

Cell therapy
Orgenesis is also a pioneer in transdifferentiation, which involves direct reprogramming of an adult tissue or cell into another type of specialized cell with its distinct function

Orgenesis Inc (NASDAQ:ORGS), a developer of advanced cell therapies, announced Monday that the US Food and Drug Administration has granted Orphan Drug designation to its Autologous Insulin Producing (AIP) cells as a cell replacement therapy for the treatment of severe diabetes following a pancreatectomy, a surgical procedure performed to treat chronic pancreatitis.

The Germantown, Maryland-based biopharmaceutical company’s Israeli subsidiary, Orgenesis Ltd, is also targeting insulin-dependent diabetes, said the company.   

Orgenesis is a pioneer in transdifferentiation, which involves direct reprogramming of an adult tissue or cell into another type of specialized cell with its distinct function.

Transdifferentiation potentially provides the use of a patient's own adult tissues to restore any damaged organs or tissue. This ability to convert one cell type to another holds great promise in the stem-cell field.

Orgenesis stock shot up nearly 6.8% to $4.58 in premarket trade. 

READ: Orgenesis leads the charge at an exciting time for cell therapy

Orgenesis founder and chief scientific officer Professor Sarah Ferber is the inventor of the company’s groundbreaking transdifferentiation platform technology.

The company said Monday that the technology is being exclusively licensed by Orgenesis Ltd from Tel Hashomer Medical Research Infrastructure and Services Ltd (THM) and is based on the work of Professor Sarah Ferber who is a researcher at THM.

Converting a diabetic’s own tissue into insulin-producing cells has the potential to provide a cure for insulin dependence and overcoming donor shortages, costs and risks involved with transplant rejection.

“This is a cell taken from your own body, utilized and manipulated, so it can go back and do an even better job,” said Orgenesis CEO Vered Caplan.

Novel Technology platform

Orgenesis has developed its transdifferentiation process and its Point of Care liver expansion technology to transform liver cells into AIP cells. The cells are first derived from a small sample of the patient’s liver cells and expanded in a liver cell bank based on Orgenesis’ unique POCare cell expansion capabilities.

At the appropriate time, the cells may be converted into functional glucose-regulated AIP cells through the company’s proprietary transdifferentiation process and returned to the patient’s liver via a transfusion, said the company. The goal is to provide a “practical cure” for various types of insulin-dependent diabetes.

“This represents a major milestone for both Orgenesis and patients who have to suffer the tremendous hardships associated with total pancreatectomy,” said Caplan. “Orphan Drug designation has the potential to reduce the time and costs required to bring our AIP cell therapy to market and should help streamline the approval process.”

Collaborating with multiple clinical sites

Caplan said the therapy is a great example of what the company hopes to achieve on a broader scale and for numerous indications through its POCare strategy.

“By collaborating with multiple clinical sites to collect and process the liver biopsies from the relevant patient populations, we are building the framework for the creation and maintenance of our liver cell banks that will be the basis for the transdifferentiation process,” said Caplan.

She said collaborations such as the one the company has cemented with the New York Blood Center in the US and the Medical University of Graz in Austria will allow the company to maintain liver cell banks at a “level required for clinical use” and should become a vital resource for clinical development.

“While the treatment of patients with hypoglycemia-prone diabetes resulting from total pancreatectomy is our immediate focus, we see significant opportunities ahead to expand our indications to include treatment for other causes of diabetes,” said Caplan.

The FDA’s Orphan Drug designation gives the drug sponsor to various development incentives, including eligibility for seven years of market exclusivity upon regulatory approval, exemption from FDA application fees, tax credits for qualified clinical trials, and other assistance in the drug development process.

Contact Uttara Choudhury at [email protected]

Follow her on Twitter@UttaraProactive 

Quick facts: Orgenesis

Price: 5.6 USD

Market: NASDAQ
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