FDA Grants Orphan Drug Designation to March Biosciences for T-Cell Lymphoma Cell Therapy
On the back of FDA’s designation, March Biosciences is preparing to advance MB-105, a CD5-targeted CAR-T cell therapy, to a Phase II clinical trial in early 2025.
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US-based March Biosciences (March Bio), a clinical-stage biotechnology company, has been granted orphan drug designation by FDA for MB-105, the company's CD5-targeted chimeric antigen receptor T cell (CAR-T) cell therapy, for treating relapsed/refractory CD5-positive T-cell lymphoma (TCL). MB-105 is March Bio's lead program, which it launched from the Center for Cell and Gene Therapy (CAGT), a collaboration between experts at Baylor College of Medicine, Houston Methodist Hospital, and Texas Children's Hospital, according to a Jan. 28, 2025 company press release (1).
According to March Bio, MB-105 is a first-in-class, autologous, CD5-targeted CAR-T cell therapy which the company is developing for CD5-positive hematologic malignancies. In addition to TCL, it is in development for treating T-cell acute lymphoblastic leukemia (T-ALL), chronic lymphocytic leukemia, and mantle cell lymphoma. Using a proprietary CAR design, the therapy can selectively target malignant cells while preserving some normal T-cell function. MB-105 is currently in a Phase I clinical trial (NCT03081910) for relapsed/refractory TCL and T-ALL, where it has demonstrated a 44% overall response rate in TCL patients (1).
“Beyond an important regulatory milestone, securing orphan drug designation for MB-105 from [FDA] underscores the critical need for new therapeutic options for patients with T-cell lymphoma,” said Sarah Hein, co-founder and chief executive officer of March Biosciences, in the press release.
In October 2024, the company closed a $28.4 million Series A financing that will allow it to advance MB-105 into Phase II development as well as strengthen its manufacturing capabilities to support future commercialization (2).
“Currently, patients with treatment-resistant or recurrent T-cell cancers face an extremely poor prognosis. The MB-105 Phase I trial has shown promising safety and efficacy signals in relapsed/refractory T-cell lymphoma patients. This designation further validates our development strategy as we prepare to initiate our Phase II clinical trial in early 2025,” Hein said in the release.
March Bio’s pipeline beyond MB-105 includes cell therapies aimed at further expanding the company’s potential to offer better treatment options for difficult diseases and malignancies. Funding raised by the company to date totals more than $52 million and includes venture financing, support from the Cancer Prevention & Research Institute of Texas, and the National Institutes of Health Small Business Innovation Research program.
Meanwhile, the CAGT collaboration aims to provide an infrastructure consisting of research, manufacturing, and clinical care that is needed to develop and apply innovative and cost-effective cell and gene therapies. The goal of the center is to “facilitate and support collaboration among experts in all areas of human disease so that cellular and genetic therapies may be used as safely and as rapidly as possible” and “ensure that cell and gene therapy patients have continued access to the latest and most effective cellular and genetic treatments available” (3).
The CAGT facility includes high-tech features, such as an adult unit specifically designed to offer specialized care to immunocompromised patients and which includes the use of an advanced air filtration system; transplant units for pediatric and adult bone marrow and stem cell transplants (in collaboration with other leading institutions); and an Adult Stem Cell Transplant unit that houses a family care setting, which includes an activity center, computer business center, and entertainment/dining area. The center also contains laboratory space that can support basic and translational research regarding cell and gene therapy (3).
References
1. March Biosciences. March Biosciences Receives FDA Orphan Drug Designation for MB-105, a First-in-Class CD5 CAR-T Cell Therapy, for T-Cell Lymphoma. Press Release. Jan. 28, 2025.
2. March Biosciences. March Biosciences Closes Oversubscribed $28.4 Million Series A Financing. Press Release. Oct. 23, 2024.
3. Houston Methodist Hospital. Center for Cell & Gene Therapy. houstonmethodist.org (accessed Feb. 11, 2025).
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