RMAT designation follows positive Phase 2 proof-of-concept data published in the Journal of Clinical Oncology (JCO) for Viralym-M, an allogeneic, off-the-shelf multi-virus specific T-cell therapy
Viralym-M Phase 3 registrational studies planned for treatment of severe and life-threatening virus-associated diseases, including virus-associated hemorrhagic cystitis
AlloVir, a late-clinical stage allogeneic T-cell therapy company, today announced that it has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for Viralym-M (ALVR105), its lead allogeneic, off-the-shelf, multi-virus specific T-cell therapy, for the treatment of hemorrhagic cystitis (HC) caused by BK virus in adults and children following allogeneic hematopoietic stem cell transplantation (HSCT). Following positive Phase 2 clinical data published in the Journal of Clinical Oncology (Tzannou, JCO, 2017), AlloVir is in the process of planning Phase 3 registrational studies of Viralym-M. AlloVir is an ElevateBio portfolio company.
The FDA may grant RMAT designation to drug candidates with preliminary clinical evidence indicating the potential to address unmet medical needs in patients with serious or life-threatening conditions. Similar to Breakthrough Therapy designation, RMAT designation provides a number of important benefits in the drug development process, including early interactions with the FDA to discuss clinical trial design and other actions to expedite development and review.
“We are pleased to have received this RMAT designation for Viralym-M, which follows the initial clinical results achieved with Viralym-M in patients with BK virus-associated hemorrhagic cystitis after HSC transplant. We look forward to working closely with the FDA as we continue to develop Viralym-M for these patients, who currently lack effective treatment options,” said David Hallal, Chief Executive Officer of AlloVir and co-founder of ElevateBio. “Viralym-M is specifically designed to restore natural T-cell immunity in immunocompromised patients, helping to fight or prevent severe and life-threatening virus-associated diseases such as hemorrhagic cystitis until the patient’s own immune system kicks back in. We are on track to initiate Phase 3 studies for Viralym-M and to advance ALVR106, AlloVir’s second allogeneic, off-the-shelf multi-respiratory virus specific T-cell therapy, into the clinic over the next 12 months.”
The observed incidence of BK virus-associated HC is 8%-25% and 7%-54% in pediatric and adult patients, respectively, and is higher after allogeneic HSCT than after autologous HSCT, particularly after haploidentical HSCT with post-transplant exposure to cyclophosphamide as prophylaxis for graft versus host disease (GVHD). HC is associated with significant and prolonged morbidity and may result in hospitalization, renal dysfunction, and increased mortality. Over half of all patients with HC present with clot formation and/or severe bladder hemorrhage with renal impairment. Bleeding may be life-threatening requiring urologic interventions including cystectomy. There are no FDA approved treatments for BK virus-associated HC.
About Viralym-M (ALVR105)
Viralym-M is the lead therapeutic candidate in AlloVir’s pipeline of allogeneic, off-the-shelf multi-virus specific T-cell therapies designed to treat active virus-associated diseases in immunocompromised patients, including in patients following HSCT, solid organ transplant, or in patients suffering with primary immunodeficiencies, cancer, or HIV. AlloVir has been developing Viralym-M to treat a range of such active virus-associated diseases, including BK hemorrhagic cystitis, cytomegalovirus, adenovirus, Epstein-Barr virus, JC virus and human herpesvirus 6. In a positive Phase 2 proof-of-concept study, published in the Journal of Clinical Oncology (Tzannou, JCO, 2017), greater than 90% of patients who failed conventional treatment and received Viralym-M demonstrated a complete or partial clinical response based on predefined criteria, and most exhibited complete elimination of detectable virus in the blood and resolution of major clinical symptoms.
AlloVir (formerly ViraCyte), founded in 2013 by researchers at Baylor College of Medicine’s Center for Cell and Gene Therapy, is the leader in the development of novel cell therapies with a focus on restoring natural T-cell immunity against life-threatening virus-associated diseases in patients with severely weakened immune systems. The company’s technology platforms deliver commercially scalable solutions by leveraging off-the-shelf, allogeneic, multi-virus specific T-cells targeting devastating viral pathogens for immunocompromised patients under viral attack. AlloVir’s technology and manufacturing process enables the potential for the treatment and/or prevention of up to six devastating viruses with its lead allogeneic product, Viralym-M (ALVR105), and allows potentially hundreds of patients to be treated with virus-specific T-cells manufactured from a single donor, using a proprietary cell selection strategy to match the company’s bank of third-party donor-derived cell lines to patients. AlloVir is advancing multiple mid- and late-stage clinical trials across its product portfolio. More information can be found at www.allovir.com.
ElevateBio, LLC, is a Cambridge-based biotechnology company, established to create and operate a broad portfolio of cell and gene therapy companies with leading academic researchers, medical centers and entrepreneurs. ElevateBio builds single- and multi-product companies by providing scientific founders with fully integrated bench-to-bedside capabilities including world-class scientists, manufacturing facilities, drug developers and commercial expertise. ElevateBio BaseCamp, a company-owned Cell and Gene Therapy Center of Innovation, will serve as the R&D, process development and manufacturing hub across the entire ElevateBio portfolio while also supporting selected strategic partners. ElevateBio’s lead investors are the UBS Oncology Impact Fund (OIF) managed by MPM Capital, as well as F2 Ventures. Investors also include EcoR1 Capital, Redmile Group, and Samsara BioCapital. For more information, please visit https://www.elevate.bio.