Vironexis Biotherapeutics Launches with FDA Clearance of IND Application for First-Ever Clinical Trial of an AAV-delivered Cancer Immunotherapy
September 12 2024 - 7:00AM
Vironexis Biotherapeutics, focused on transforming the future of
cancer treatment by pioneering AAV-delivered T-cell immunotherapy,
launched from stealth today, unveiling its TransJoin™ AAV Gene
Therapy Platform and a pipeline of more than ten product candidates
for blood-based cancers, solid tumor metastasis prevention, and a
cancer vaccine. Vironexis’s $26 million seed financing was led by
Drive Capital and Future Ventures, with participation from
Moonshots Capital and Capital Factory. The company has received
clearance of its Investigational New Drug (IND) application from
the U.S. Food and Drug Administration (FDA) for VNX-101, its first
gene therapy product candidate, for the treatment of CD19+ acute
lymphoblastic leukemia. Vironexis anticipates initiating patient
enrollment of a Phase 1/2 trial of VNX-101 in the fourth quarter of
2024, which will mark the first-ever clinical trial of an
AAV-delivered cancer immunotherapy. VNX-101 has received both Fast
Track Designation and Rare Pediatric Disease Designation from the
FDA.
“We’re excited to launch Vironexis from stealth and reveal our
noteworthy progress advancing AAV-delivered T-cell immunotherapy,”
said Samit Varma, co-founder and CEO of Vironexis. “Our novel
technology builds on the power of T-cell immunotherapy while
overcoming key shortcomings and challenges of existing approaches
such as CAR-T and bispecific antibodies. We believe we have the
opportunity to dramatically improve upon the safety, efficacy and
durability of these drug classes, while streamlining manufacturing
and significantly lessening the burden of treatment for patients.
Our focus on execution has yielded an expansive pipeline and a
clinic-ready lead program in just three years. We’re working as
quickly as possible to transform the future of cancer treatments
for patients.”
TransJoin enables a patient’s body to express an engineered
transgene that redirects T cells throughout the body to tumor
cells. The proprietary technology requires only a single
dose and provides a bridge that joins together T cells and
tumor cells to promote long-term, continuous T
cell-mediated tumor killing. The foundational technology for
TransJoin was licensed from Nationwide Children’s Hospital. In
2022, research led by Timothy Cripe, M.D., Ph.D., Chief of the
Division of Pediatric Hematology/Oncology/Bone and Marrow
Transplant, describing the TransJoin technology was published in
Science Advances [link here to paper]. Dr. Cripe is one of
Vironexis’s co-founders, along with Mr. Varma, a seasoned
biotechnology and gene therapy company entrepreneur, and Brian
Kaspar, Ph.D., a notable gene therapy scientist-entrepreneur who
founded AveXis, Inc. (acquired by Novartis) and pioneered the AAV
gene therapy ZOLGENSMA®.
“AAV is a proven delivery technology with multiple approvals
since 2017. Recognizing the pivotal impact of AAV delivery for the
treatment of rare diseases, we believed its unique ability to
enable long-term, continuous expression of a therapeutic protein
could be the missing link to overcome the myriad challenges
associated with first-generation T-cell immunotherapies like CAR-T.
We subsequently demonstrated the potential of this approach in the
preclinical setting,” said Dr. Cripe. “It’s thrilling to now be on
the cusp of seeing how this technology translates in the clinical
setting. Our ultimate goal is to help a vastly broader population
of patients realize the tremendous benefits of T-cell
immunotherapy.”
Vironexis is in the process of obtaining pre-IND input from the
FDA for its second program, VNX-202, as a treatment for the
prevention of metastatic HER2+ cancer (including breast cancer and
other tumor types), and plans to start dosing patients in a Phase
1/2 clinical trial in 2025. The company’s other current product
candidates include treatments for BCMA/GPRC5D+ multiple myeloma,
CD19/20+ B-cell lymphoma; treatments to prevent metastases in GD2+
neuroblastoma, HER2+ gastric cancer, PSMA+ and MSLN+ pancreatic
cancer, B7H3+ ostersarcoma and GP350+ nasopharyngeal cancer; a
cancer vaccine for GP350+ nasopharyngeal cancer; and a treatment
for CD19+ systemic lupus erythematosus, which Vironexis plans to
partner for further development.
Molly Bonakdarpour, Partner at Drive Capital commented, “As a
portfolio investment, Vironexis offers an ideal blend of
groundbreaking technology, impressive preclinical data, the
potential for vast patient impact, and a founding team with deep,
relevant expertise and a proven track record of company formation,
strategic thinking, and successful execution. Vironexis’s notable
productivity in a very short timeframe has been remarkable. We look
forward to its upcoming transition to a clinical-stage
company.”
Steve Jurvetson, Co-Founder of Future Ventures, added, “We were
drawn not only to the novelty of the TransJoin technology but also
to its broad applicability, spanning treatments for blood-based
cancers, solid tumor metastasis prevention, and cancer vaccines, as
well as immune disorders. The versatility of this platform is truly
standout, and the Vironexis team’s progress in rapidly building a
pipeline that explores the expanse of these opportunities is
extraordinary.”
How TransJoin Works
Vironexis’s TransJoin technology is designed to enable the
expression of a secreted T cell engager that binds the tumor cell
on one side (changeable depending on the product candidate
indication target) and T cells via CD3 on the other side. CD3 is a
protein that helps promote T-cell recognition of and activation
against cancer cells. Following a single, one-time intravenous
infusion, the TransJoin technology instructs the liver to
continuously secrete the bispecific protein into the bloodstream to
redirect T cells to tumor cells. TransJoin provides a bridge that
joins T cells and tumor cells, resulting in long-term, consistent
serum levels of the therapy and, thus, long-term, consistent
T-cell-mediated tumor cell killing. TransJoin’s extremely low-dose
AAV delivery minimizes toxicity and adverse events.
About Vironexis Biotherapeutics
Vironexis is focused on transforming the future of cancer
treatment by pioneering AAV-delivered T-cell immunotherapy. Our
TransJoin™ AAV Gene Therapy Platform enables the creation of
off-the-shelf, single-dose gene therapies designed to overcome the
key challenges and shortcomings of current immunotherapies,
including CAR-T and bispecific antibodies. Our current pipeline
includes more than ten product candidates for blood-based cancers,
solid tumor metastasis prevention, and a cancer vaccine. Our lead
program, VNX-101, for the treatment of CD19+ acute lymphoblastic
leukemia, is anticipated to begin clinical trials in the fourth
quarter of 2024. Visit us at vironexis.com and follow us on
LinkedIn and X.
Vironexis Media Contact:Liz MeloneMelone
Communications, LLCliz@melonecomm.com