Policy & Regulation
Viralym-M Granted Regenerative Medicine Advanced Therapy Designation for the Treatment of Hemorrhagic Cystitis Caused by BK Virus in Adults and Children Following Allogeneic HSCT
13 June 2019 - - US-based late-clinical stage allogeneic T-cell therapy company AlloVir has received Regenerative Medicine Advanced Therapy designation from the US Food and Drug Administration for Viralym-M (ALVR105), its lead allogeneic, off-the-shelf, multi-virus specific T-cell therapy, for the treatment of hemorrhagic cystitis caused by BK virus in adults and children following allogeneic hematopoietic stem cell transplantation, the company said.
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.
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.
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.
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 a 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.
ElevateBio 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 centres and entrepreneurs.
The company 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 and 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 managed by MPM Capital, as well as F2 Ventures.
Investors also include EcoR1 Capital, Redmile Group, and Samsara BioCapital.


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