· Supporting the development of its proprietary MIC-Lx Modified Immune Cells technology as a novel approach to reduce lifelong systemic immunosuppression and improve long-term outcomes for transplant recipients
· Strengthening MIC-Lx’s regulatory and commercial positioning in Europe
Heidelberg, Germany, July 13, 2026. TolerogenixX, a German clinical-stage biopharmaceutical company developing personalized cell therapies inducing antigen-specific immune tolerance, today announced that the European Commission has granted MIC-Lx, the Company's lead product candidate, Orphan Drug Designation (ODD) for living-donor kidney transplantation following a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).
The Orphan Drug Designation follows the
recent granting of EMA PRIME designation for MIC-Lx, marking a second key regulatory
milestone and further strengthening the Company's pathway toward full regulatory
approval.
Both designations recognize the significant
unmet medical need in kidney transplantation addressed by MIC-Lx. Despite major
advances in transplantation medicine, recipients today generally require
lifelong systemic immunosuppressive therapy to prevent organ rejection. While
effective, these therapies are associated with substantial long-term
complications, including infections, malignancies, cardiovascular disease and
chronic kidney damage, which continue to limit long-term patient outcomes.
Unlike conventional immunosuppressive
therapies, MIC-Lx is designed to induce donor-specific immune tolerance before
transplantation, enabling targeted protection of the transplanted organ while
preserving the patient's overall immune competence. Based on TolerogenixX's
proprietary Modified Immune Cell (MIC) technology, MIC-Lx aims to reduce
dependence on lifelong systemic immunosuppression and improve graft survival and
patients' long-term quality of life.
"Receiving both PRIME and Orphan
Drug Designation from the EMA is an important recognition of our first-in-class
approach and supports the ongoing development of MIC-Lx," said Prof. Dr. Matthias Schaier, Chief
Executive Officer of TolerogenixX. "Our goal is to enable long-term
graft protection without lifelong systemic immunosuppression, thereby addressing
one of the key challenges in transplantation medicine.”
“Kidney transplant
recipients still face the long-term burden of systemic immunosuppression,
including infection risk, malignancies, cardiovascular complications, and
chronic graft injury,” said Prof. Dr. Christian Morath, Chief Scientific Officer of
TolerogenixX. “MIC-Lx is designed to induce donor-specific immune tolerance
before transplantation, with the goal of protecting the graft while preserving
broader immune competence. The Orphan Drug Designation further supports the
clinical rationale for advancing this approach.”
In addition to recognizing the therapy's
clinical rationale, Orphan Drug Designation provides regulatory and commercial
incentives that support the development of innovative medicines for rare
diseases. These include protocol assistance from the EMA, significant fee
reductions during development and regulatory review, and, subject to marketing
authorization, up to ten years of market exclusivity in the European Union.
MIC-Lx is currently being evaluated in a
randomized, controlled Phase IIb TOL-2 trial in living-donor kidney
transplantation. Following the successful treatment of all 63 donor-recipient
pairs, the study has entered its follow-up phase, with topline results expected
in the first half of 2027.
MIC-Lx has already completed a clinical Phase
Ib trial (TOL-1) in kidney transplant recipients demonstrating sustained safety
and tolerability after a single administration while preserving normal immune
responses.
About TolerogenixX TolerogenixX is a privately held biopharmaceutical company
developing novel, personalized therapies to induce antigen-specific immune
tolerance in transplantation and autoimmune diseases, creating the foundation for
a broader immune tolerance platform. Its proprietary MIC (modified immune
cells) technology aims to modulate the immune system in a targeted manner,
potentially reducing the need for long-term systemic immunosuppression.
The Company’s lead candidate MIC-Lx has
completed a Phase Ib clinical trial (TOL-1) in kidney transplant recipients
demonstrating sustained safety and tolerability after a single administration
while preserving normal immune responses. A Phase IIb study (TOL-2) is
currently ongoing. TolerogenixX was founded in 2016 and is headquartered in
Heidelberg, Germany.
About MIC treatment TolerogenixX's proprietary MIC treatment is a
personalized cell therapy designed to modulate the immune system through a
novel mode of action. It not only benefits transplant recipients, but it could also
benefit patients with autoimmune diseases such as systemic lupus erythematosus and multiple sclerosis. Unlike
conventional immunosuppressive therapies, the platform is designed to preserve
the patient's overall immune competence.
MIC production is fast, safe, and effective.
Using cells obtained by leukapheresis, MIC can be manufactured within 24 hours.
Thanks to its standardized process, MIC production can be easily scaled up and supplied
globally using the proprietary approach developed by TolerogenixX. Forward-Looking Statements This press release contains
forward-looking statements, including statements regarding the expected timing
of topline data from the TOL-2 study, the potential clinical benefits of
MIC-Lx, future regulatory interactions, development plans, scale-up production,
partnering opportunities and the broader potential of the MIC Platform. These
statements are based on current expectations and involve risks and uncertainties.
Actual results may differ materially from those expressed or implied by these
forward-looking statements. TolerogenixX is under no obligation to update
forward-looking statements except when required by applicable law.
Contacts TolerogenixX GmbH Prof. Dr. med. Matthias Schaier Im Neuenheimer Feld 162 69120 Heidelberg, Germany Tel. +49 162 2638005
akampion Ines-Regina Buth Managing Partner Tel. +49 40 88 16 59 64 Tel. +49 30 23 63 27 68
