CytoDyn & FDA Will Meet to Potentially Finalize Protocol for Pivotal Monotherapy Trial

CytoDyn Inc. recently announced the US FDA has requested an in-person meeting to discuss and potentially finalize the company’s recently submitted protocol for a pivotal monotherapy trial with leronlimab. The company will announce further details when available.

Management is also developing a clinical trial collaboration agreement to initiate a HIV prevention study to be conducted by and fully funded by a public health agency in Southeast Asia. CytoDyn’s President and CEO, Nader Pourhassan, PhD, stated “We believe there are over 130,000 individuals using a currently approved drug for the prevention of HIV. The side effects and long-term morbidities are well known, which include, among others, kidney and liver complications, bone density issues, headaches, abdominal pain, and weight loss. Based upon leronlimab’s mechanism of action, our collaborating partners are very excited to initiate a prevention study, and we share their enthusiasm about the possibilities.”

“The potential role of the CCR5 co-receptor in the prevention of and cure for HIV has been very well documented and has been the subject of major media attention,” added Dr. Pourhassan. “There is only one self-injectable humanized monoclonal antibody, leronlimab, which is near approval that targets CCR5. Leronlimab is one dose-a-week, sub-cutaneous injection that has all the characteristics of potentially becoming a major therapeutic contributor to the field of HIV and perhaps in cancer and auto-immune diseases. The support of our shareholders has enabled our Company to advance leronlimab towards an anticipated FDA approval, in addition to exploring many opportunities in various cancer indications, any of which could offer significant benefits and rewards for patients throughout the world and for all of our stakeholders.”

The FDA granted a Fast Track designation to two clinical trials being conducted by CytoDyn for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second a trial for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases including NASH. Leronlimab has successfully completed nine clinical trials in over 700 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays an important role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by >98% in a murine xenograft model. CytoDyn is therefore conducting a Phase 2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.  Additional research is being conducted with leronlimab in the setting of cancer and NASH with plans to conduct additional clinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation and may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to further support the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD and that blocking this receptor from recognizing certain immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted “orphan drug” designation to leronlimab for the prevention of graft-versus-host disease (GvHD).

CytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and in immune-mediated illnesses, such as graft-vs-host disease (GvHD) and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in 2019 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab (PRO 140) as a once-weekly monotherapy for HIV-infected patients and, plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab (PRO 140) can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than four years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and has received clearance to initiate a clinical trial with leronlimab in metastatic triple-negative breast cancer. For more information, visit www.cytodyn.com.