Treatment of AdV infection is posoleucel’s second potential indication to receive RMAT designation
Phase 3 registrational study of posoleucel for AdV treatment is now open for enrollment
Proof-of-concept study of ALVR106 for the treatment of multiple respiratory viral infections is also open for enrollment
WALTHAM, Mass.--(BUSINESS WIRE)-- AlloVir, a late-clinical stage allogeneic T-cell immunotherapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted its lead multi-virus specific T cell (VST) therapy, posoleucel (Viralym-M, ALVR105), Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of adenovirus (AdV) infection following allogeneic hematopoietic stem cell transplant (allo-HCT). The designation is based on positive results from the Phase 2 CHARMS study.
RMAT designation recognizes the potential for posoleucel to address the unmet medical need posed by AdV, a potentially life-threatening condition with no approved treatment options. This designation enables early interactions with the FDA to discuss clinical trial design and other actions to expedite development and review. The FDA previously granted RMAT designation to posoleucel for the treatment of hemorrhagic cystitis (HC) caused by BK virus in adults and children following allo-HCT.
“We are pleased that the FDA has granted posoleucel RMAT designation for a second treatment-related indication,” said Ercem Atillasoy, M.D., Chief Regulatory and Safety Officer, AlloVir. “The two RMAT designations reinforce the potential of posoleucel in areas of urgent patient need. We look forward to working closely with FDA as we continue to advance posoleucel through late-stage clinical development.”
A Phase 3 registrational study (NCT05179057) of posoleucel for the treatment of AdV viremia is now open and enrolling pediatric and adult patients following allo-HCT. This study is the second Phase 3 registrational study of posoleucel, following the initiation of the Phase 3 study for the treatment of virus-associated HC last year.
Separately, the company also announced the initiation of a Phase 1/2 clinical trial (NCT04933968) of ALVR106, its investigational, allogeneic, off-the-shelf, multi-VST therapy for the treatment of infections caused by human metapneumovirus (hMPV), influenza, parainfluenza virus (PIV) and respiratory syncytial virus (RSV). This trial extends AlloVir’s VST platform to tackle respiratory viruses that pose a considerable risk for autologous and allo-HCT patients.
"The initiation of these posoleucel and ALVR106 studies represents important progress in our effort to bring forward much needed treatment options for immunocompromised patients,” said Richard Riese, M.D., Ph.D., Senior Vice President, Clinical Research, AlloVir. “These investigational multi-VST therapies aim to restore immunity against viruses that, if left untreated, can have devastating consequences. We look forward to working with study investigators to enroll these studies and demonstrate the potential for VST therapy in areas of urgent unmet need.”
About Adenovirus and the Posoleucel Phase 3 Study
AdV is a potentially life-threatening viral infection that has no approved treatments. AdV viremia occurs in approximately one third (32%) of pediatric allo-HCT patients and 6% of adult allo-HCT patients. The spectrum of AdV-associated disease in HCT patients ranges from mild gastroenteric or respiratory symptoms to pneumonia, hepatitis, severe hemorrhagic enteritis or cystitis, multi-organ failure or death. The current standard of care is off-label use of an antiviral that has demonstrated limited efficacy and significant toxicity.
The Phase 3 registrational trial (NCT05179057) of posoleucel is a randomized, double-blind, placebo-controlled study assessing the efficacy and safety of posoleucel for the treatment of AdV. The study is enrolling 80 pediatric and adult allo-HCT patients with high-level viremia (viral load ≥10,000 copies/mL) or with consistently rising viral load and immune deficiency (viral load ≥1,000 copies/mL and