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CytoDyn Files for Orphan Drug Designation for Use of PRO 140 in Graft Versus Host Disease

VANCOUVER, Wash., Dec. 22, 2015 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTCQB:CYDY), a biotechnology company focused on the development of new therapies for combating human immunodeficiency virus (HIV) infection, today announced that it has filed for Orphan Drug Designation with the FDA for its first non-HIV indication, Graft versus Host Disease (GvHD). CytoDyn currently is conducting a Phase 3 pivotal trial with its lead product candidate, PRO 140, for the treatment of HIV infection.

The GvHD clinical trial is a Phase 2, randomized, double-blind, placebo-controlled, multi-center study. It evaluates the safety and efficacy of PRO 140 for prophylaxis of acute GvHD in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) undergoing allogeneic stem-cell transplantation. The trial is a 100-day study with 60 patients.

Positive results from this study would allow the Company to file for a breakthrough designation to expedite the commercialization of PRO 140 for this clinical indication. As previously reported, PRO 140 is considered safe and well tolerated with negligible toxicities or side effects. The Company believes these attributes make it an ideal candidate for treatment of GvHD, a life-threatening disease for transplant recipients with compromised immune systems.

The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Please reference: (https://rarediseases.info.nih.gov/files/fda%20orphan%20drugs.pdf)

The Company has selected a transplantation indication called GvHD as its first non-HIV clinical indication. The CCR5 receptor, the target for PRO 140, is an important mediator of GvHD, especially in the organ damage that is the usual cause of death. The only approved CCR5 inhibitor, Maraviroc, is currently in a Phase 2 study in GvHD. The Company believes that PRO 140 has significant advantages over Maraviroc in more favorable dosing and pharmacokinetics, less toxicity and side effects, and no direct stimulation (agonist activity) of the CCR5 receptor.

PRO 140 is a monoclonal antibody that targets the chemokine receptor, CCR5, expressed on a variety of cells that play a central role in inflammatory responses. PRO 140 binds to this receptor in a way that prevents HIV from using it as an entry gateway, while not activating the immune function of the receptor. The Company’s recent data indicate that PRO 140 interferes with (antagonist activity) activation of the receptor by its ligand, named CCL5. The CCL5 is a chemokine mediator which has been shown to be a central figure in many inflammatory disease processes, and blocking its interaction with CCR5 is believed to be of therapeutic benefit.

Dr. Nader Pourhassan, President and CEO, commented: “GvHD is our first expansion of PRO 140 into non-HIV clinical areas. We plan to explore further opportunities to expand the clinical applications of PRO 140 to those indications where CCR5 plays an important role, namely certain autoimmune diseases and cancer.”

About Graft versus Host Disease
Graft-versus-host disease (GvHD) is a complication that can occur after a stem cell or bone marrow transplant. With GvHD, the newly transplanted donor cells attack the transplant recipient's body. GvHD may occur after a bone marrow or stem cell transplant in which an individual receives bone marrow tissue or cells from a donor. This type of transplant is called allogeneic. The new, transplanted cells regard the recipient's body as foreign. When this happens, the newly transplanted cells attack the recipient's body. GvHD does not occur when an individual receives his or her own cells during a transplant. Before a transplant, tissue and cells from possible donors are tested to determine how closely they match the person having the transplant. GvHD is less likely to occur, or symptoms will be milder, when the match is close. The chance of GvHD, at varying degrees, can be approximately 30% to 40% when the donor and recipient are related and approximately 60% to 80% when the donor and recipient are not related. There are two types of GvHD: acute and chronic. Symptoms in both acute and chronic GvHD range from mild to severe. Acute GvHD usually happens within the first three months after a transplant. Chronic GvHD usually starts more than three months after a transplant, and can last a lifetime.

In the United States, in 2011, over 7,500 unrelated donor bone marrow transplants were performed and another 10,000 conducted outside the U.S. One-year survival is now approximately 60% with the most common causes of death being relapse or GvHD. The market in the U.S. is expected to reach $500 million in the next several years.

About CytoDyn
CytoDyn is a biotechnology company focused on the clinical development and potential commercialization of humanized monoclonal antibodies for the treatment and prevention of Human Immunodeficiency Virus (HIV) infection. The Company has one of the leading monoclonal antibodies under development for HIV infection, PRO 140, which has finished Phase 2 clinical trials with demonstrated antiviral activity in man and is currently in Phase 3. PRO 140 blocks the HIV co-receptor CCR5 on T-cells which prevents viral entry. Clinical trial results thus far indicate that PRO 140 does not negatively affect the normal immune functions that are mediated by CCR5. Results from six Phase 1 and Phase 2 human clinical trials have shown that PRO 140 can significantly reduce viral burden in people infected with HIV. A recent Phase 2b clinical trial demonstrated that PRO 140 can prevent viral escape in patients during several weeks of interruption from conventional drug therapy. CytoDyn intends to continue to develop PRO 140 as a therapeutic anti-viral agent in persons infected with HIV. For more information on the Company, please visit www.cytodyn.com.

About PRO 140
PRO 140 belongs to a new class of HIV/AIDS therapeutics -- viral-entry inhibitors -- that are intended to protect healthy cells from viral infection. PRO 140 is a fully humanized IgG4 monoclonal antibody directed against CCR5, a molecular portal that HIV uses to enter T-cells. PRO 140 blocks the predominant HIV (R5) subtype entry into T-cells by masking this required co-receptor, CCR5. Importantly PRO 140 does not appear to interfere with the normal function of CCR5 in mediating immune responses. PRO 140 does not have agonist activity towards CCR5 but does have antagonist activity to CCL5 which is a central mediator in inflammatory diseases. PRO 140 has been the subject of seven clinical trials, each demonstrating efficacy by significantly reducing or controlling HIV viral load in human test subjects. PRO 140 has been designated a “fast track” product candidate by the FDA. The PRO 140 antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements as compared to daily drug therapies currently in use.

Forward-Looking Statements
This press release includes forward-looking statements and forward-looking information within the meaning of United States securities laws, including statements regarding the Company’s Phase 3 study, GvHD study and its results and completion, as well as other studies. These statements and information represent CytoDyn’s intentions, plans, expectations, and beliefs and are subject to risks, uncertainties and other factors, many beyond CytoDyn’s control. These factors could cause actual results to differ materially from such forward-looking statements or information. The words “believe,” “estimate,” “expect,” “intend,” “attempt,” “anticipate,” “foresee,” “plan,” and similar expressions and variations thereof identify certain of such forward-looking statements or forward-looking information, which speak only as of the date on which they are made.

CytoDyn disclaims any intention or obligation to publicly update or revise any forward-looking statements or forward-looking information, whether as a result of new information, future events or otherwise, except as required by applicable law. Readers are cautioned not to place undue reliance on these forward-looking statements or forward-looking information. While it is impossible to identify or predict all such matters, these differences may result from, among other things, the inherent uncertainty of the timing and success of and expense associated with research, development, regulatory approval, and commercialization of CytoDyn’s products and product candidates, including the risks that clinical trials will not commence or proceed as planned; products appearing promising in early trials will not demonstrate efficacy or safety in larger-scale trials; future clinical trial data on CytoDyn’s products and product candidates will be unfavorable; funding for additional clinical trials may not be available; CytoDyn’s products may not receive marketing approval from regulators or, if approved, may fail to gain sufficient market acceptance to justify development and commercialization costs; competing products currently on the market or in development may reduce the commercial potential of CytoDyn’s products; CytoDyn, its collaborators or others may identify side effects after the product is on the market; or efficacy or safety concerns regarding marketed products, whether or not scientifically justified, may lead to product recalls, withdrawals of marketing approval, reformulation of the product, additional pre-clinical testing or clinical trials, changes in labeling of the product, the need for additional marketing applications, or other adverse events.

CytoDyn is also subject to additional risks and uncertainties, including risks associated with the actions of its corporate, academic, and other collaborators and government regulatory agencies; risks from market forces and trends; potential product liability; intellectual property litigation; environmental and other risks; and risks that current and pending patent protection for its products may be invalid, unenforceable, or challenged or fail to provide adequate market exclusivity. There are also substantial risks arising out of CytoDyn’s need to raise additional capital to develop its products and satisfy its financial obligations; the highly regulated nature of its business, including government cost-containment initiatives and restrictions on third-party payments for its products; the highly competitive nature of its industry; and other factors set forth in CytoDyn’s Annual Report on Form 10-K for the fiscal year ended May 31, 2015 and other reports filed with the U.S. Securities and Exchange Commission.

CONTACT: Media: Dr. Nader Pourhassan Office: 360-980-8524 E-mail: npourhassan@cytodyn.com

Source:CytoDyn Inc.