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CytoDyn files pivotal trial protocol with FDA for flagship drug leronlimab as monotherapy for HIV

After four years of monotherapy trials, the company said it has the data to now design a long-anticipated and pivotal Phase 3 trial
Patients being treated for HIV
CytoDyn CEO Nader Pourhassan says a monotherapy with leronlimab, if approved, may allow patients with 'pill fatigue to maintain a disciplined compliance regimen'

CytoDyn Inc (OTCMKTS:CYDY) announced Wednesday that the late-stage biotechnology company has filed the pivotal trial protocol for its flagship drug leronlimab as a monotherapy for HIV patients with the US Food and Drug Administration.

The Vancouver-based biotech said the latest investigative monotherapy trial for HIV has now produced “sufficient data” for the company to design a pivotal monotherapy Phase 3 trial.

The company’s main product candidate leronlimab is an injectable antibody that shows promise as an antiviral agent with fewer side effects, lower toxicity and less frequent doses than daily therapies now in use for the treatment of HIV.

DEEP DIVE: CytoDyn presses forward with HIV treatment after Phase 3

The trial will include 10 weeks of induction therapy to identify which patients are likely to respond and which patients can safely return to their original HAART, or highly active antiretroviral therapy regimen.

The company said the objective of the trial is to assess the treatment strategy and clinical safety of using a leronlimab subcutaneous (SC) 700mg weekly dose as single-agent induction therapy followed by leronlimab SC 525 or 700mg as a maintenance therapy for the chronic suppression of CCR5-tropic HIV-1 infection.

In the study, patients will receive a combination of existing retroviral regimen and leronlimab 700 mg (sub-cutaneous) for four weeks (the overlap phase) before shifting to weekly leronlimab 700 mg monotherapy for 10 weeks (the monotherapy induction phase).

Subsequently, patients will be randomized 1:1 to leronlimab 525mg (Group A) or leronlimab 700mg (Group B) for a 36-week monotherapy maintenance phase. Success with this monotherapy trial may allow the company to file a biologics license application (BLA) for label expansion in anticipation of leronlimab receiving FDA approval for HIV patients as a combination therapy with HAART.

Glowing patient testimony 

“The testimony of patients who have taken leronlimab as a monotherapy in our previous clinical trials is very clear; patients who respond to monotherapy simply love it, and after 48 weeks they ask for extension to continue on monotherapy,” said CytoDyn CEO Dr Nader Pourhassan in a statement. “We have not seen any extension patients ask to return to their HAART regimen of pills.”

Pourhassan said a monotherapy with leronlimab, if approved, may allow patients with “pill fatigue to maintain a disciplined compliance regimen.”

“In addition, it may provide patients concerned about the possibility of developing one or more drug class resistances, to potentially maintain a suppressed viral load without the typical side effects or toxicity associated with HAART,” added Dr Pourhassan.

Jacob P Lalezari, a director at Quest Clinical Research, said that as a longstanding investigator for CytoDyn he had seen many HIV positive patients express "a preference for the weekly subcutaneous leronlimab monotherapy and uniformly request extension treatment beyond 48 weeks" in order to avoid returning to the HAART therapy.

The FDA earlier granted fast track designation — which bring drugs that fill an unmet need to market faster — to leronlimab in combination with HAART, or highly active antiretroviral therapy, for HIV treatment. Leronlimab has successfully completed nine Phase 1/2/3 clinical trials in more than 700 people, the company said.

—(Updates with quotes from Quest Clinical Research director)— 

Contact Uttara Choudhury at [email protected]

Follow her on Twitter@UttaraProactive 



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