AlloVir Announces Viralym-M Granted Regenerative Medicine Advanced Therapy (RMAT) Designation for the Treatment of Hemorrhagic Cystitis Caused by BK Virus in Adults and Children Following Allogeneic HSCT

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

HOUSTON–(BUSINESS WIRE)–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.

About AlloVir

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.

About ElevateBio

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.

Contacts

Courtney Heath
ScientPR
[email protected]
617-872-2462