CytoDyn announces voluntary withdrawal of BLA for HIV-MDR due to CRO data management issues

VANCOUVER, Washington, Oct. 28, 2022 — CytoDyn Inc. (OTCQB: CYDY) (“CytoDyn” or the “Company”), a biotechnology company developing leronlimab, a CCR5 antagonist with the potential for multiple therapeutic indications, today announced that it has voluntarily withdrawn its pending Biologics License Application (BLA) for leronlimab as a combination therapy in persons living with HIV with resistance to highly active antiretroviral therapy (HAART) in the HIV multi-drug resistant population (HIV-MDR).

The decision to voluntarily withdraw was based on various factors, including systemic issues related to the quality of the data collection and monitoring of the pivotal clinical trials by the clinical research organization (CRO) contracted to manage the trials, resulting in significant concerns with achieving a successful U.S. Food and Drug Administration (FDA) BLA approval. The Company is of the opinion that FDA approval for the HIV-MDR indication is not feasible without significant additional investment to remedy the issues. CytoDyn plans to publish soon the safety and efficacy data in which it met its primary endpoint, in its Phase 2b/3 randomized, double-blinded, placebo-controlled trial for the HIV-MDR population, in a peer-reviewed journal.

The Company believes the data it currently possesses is sufficient to complete and submit its responses to the FDA to seek the removal of the clinical hold placed on the Company’s HIV program. Further, the Company will continue to leverage the performance of leronlimab in these and other studies to advance leronlimab in other HIV-related, non-alcoholic steatohepatitis (NASH), and oncology indications – where compelling data has been generated – that may benefit a greater number of patients and result in significant shareholder value creation. For example, the Company plans to continue to pursue other underserved HIV-related indications, where it can potentially be first to market.

Cyrus Arman, Ph.D., President of CytoDyn, stated, “We have decided to voluntarily withdraw our BLA for the HIV-MDR population at this time only after extensive review and deliberation, including audits from three external independent regulatory quality firms. While the Company met its primary endpoints in these pivotal trials, which we think is a clear indication that leronlimab performs well in the clinic, we believe the issues identified in each of the three independent audits related to the quality of the data collection and oversight by the CRO make it difficult to support a successful BLA regulatory submission. Further, we have filed a claim against the CRO seeking damages resulting from its breach of the Master Services Agreement and related agreements and reimbursement of our attorney fees and costs associated with the action. As previously discussed, we are focusing on continued development in other HIV indications, NASH, and oncology, where we have Fast Track designation for metastatic triple-negative breast cancer. We plan to reenter the clinic in those indications and believe these steps will allow us to further build on the strong signals we have seen in these indications. I am very excited and quite optimistic about these opportunities, which are what ultimately attracted me to leronlimab and CytoDyn. I believe we have a unique opportunity to impact a significant number of patient lives while creating long-term value for our shareholders.”

Tanya Urbach, CytoDyn Board Chair, said, “While this is a difficult decision, the Board supports management and believes this is the best path forward for the Company, study participants, and shareholders. We are grateful to have the expertise of Dr. Arman and our new Board members to identify, evaluate, and guide the Company through difficult decisions such as these to advance successful regulatory approvals. We are very excited about the potential and future promise of leronlimab; the management team and Board are committed to execution.”

About the HIV-MDR BLA and withdrawal of the BLA Submission

The Company pursued the regulatory approval of leronlimab in HIV-MDR based on positive data from its Phase 2b/3 clinical trial for leronlimab as a combination therapy with HAART for highly treatment-experienced HIV patients, as well as information discussed in meetings with the FDA. In July 2020, the Company received a Refusal to File letter from the FDA regarding its BLA submission for leronlimab as a combination therapy with HAART for highly treatment-experienced HIV patients. The FDA informed the Company that the BLA did not contain certain information and data needed to complete a substantive review, and therefore, the FDA would not file the BLA. The deficiencies cited by the FDA included administrative deficiencies, omissions, data presentation and related analyses, and clarifications regarding the manufacturing processes. In November 2021, the Company resubmitted the non-clinical and chemistry, manufacturing, and controls (CMC) sections of the BLA. As of March 2022, the FDA had commenced its review of the CMC section. The Company is in a legal dispute with its former CRO, which was engaged to manage the clinical trials. In the context of the litigation, the Company obtained an order requiring the CRO to release the Company’s clinical data related to the BLA and other clinical trials, which the CRO had been withholding. Further, the order granted the Company the right to perform an audit of the CRO’s services. Additionally, in March of 2022, the FDA placed the HIV program on a partial clinical hold, which could affect our ability to resubmit the BLA. The Company performed evaluations of the data, results of the audits, and implications of the partial clinical hold. The Company determined the likelihood of FDA approval of the BLA was low and voluntarily withdrew its BLA due to issues with the quality of the collection and monitoring of the data by the CRO contracted to manage the clinical studies and the costs needed to remedy the issues. However, the Company believes it does possess the necessary data to submit to the FDA in connection with its response to the clinical hold on the HIV program.

Read the full press release here.