Cell therapy weekly: Orphan Drug Designation for brain cancer CAR-T cell therapy
This week: Mustang Bio’s (MA, USA) targeted CAR T-cell therapy, MB-101, received an Orphan Drug Designation and Lexeo Therapeutics’ (NY, USA) gene therapy for Friedreich Ataxia, LX2006, is granted Breakthrough designation. Plus, Amarna Therapeutics (CA, USA) and NorthX Biologics (Matfors, Sweden) made progress on their partnership, aiming to provide a potential cure for Type 1 diabetes.
The news highlights:
- Orphan Drug Designation for brain cancer CAR T-cell therapy
- Breakthrough designation for Friedreich Ataxia gene therapy
- Progress in autoimmune gene therapy partnership
Orphan Drug Designation for brain cancer CAR T-cell therapy
The US Food and Drug Administration (FDA; MD, USA) has granted Orphan Drug Designation to Mustang Bio’s IL13Ra2-targeted CAR T-cell therapy, MB-101. MB-101 is indicated for the treatment of recurrent diffuse and anaplastic astrocytoma and glioblastoma, types of brain cancers.
MB-101 is currently in an ongoing Phase I trial and data reported so far has shown that the therapy was well tolerated and 50% of patients achieved stable disease or better.
Furthermore, Mustang Bio hopes to enhance the efficacy of MB-101 by combining it with its MB-108 oncolytic virus – together presented as MB-109, to potentially treat patients with malignant glioma, including recurrent glioblastoma and high-grade astrocytomas.
“Our novel therapeutic strategy, combining our MB-101 CAR T-cell therapy with our MB-108 oncolytic virus, leverages MB-108 to reshape the tumor microenvironment (“TME”) to make cold tumors “hot,” thereby potentially improving the efficacy of MB-101 CAR T-cell therapy,” explained Manuel Litchman, CEO of Mustang Bio. “This progress demonstrates our dedication to exploring new possibilities for improving outcomes in patients with challenging-to-treat cancers.”
Breakthrough designation for Friedreich Ataxia gene therapy
Lexeo Therapeutics has been granted Breakthrough Therapy designation by the FDA for LX2006, its AAV-based gene therapy for Friedreich Ataxia (FA). The designation follows on from encouraging interim data where LX2006 demonstrated improvements in cardiac and neurological function and increased frataxin expression.
“Receiving Breakthrough Therapy designation is a significant milestone, highlighting the potential of LX2006 and the strength of clinical evidence generated to date,” said Sandi See Tai, Chief Development Officer of Lexeo Therapeutics. “We are highly encouraged by the impact of LX2006 on key measures of cardiac health, especially given the lack of treatments for FA cardiomyopathy today, which is the leading cause of death in FA. We are also optimistic about the improvements we have observed in functional measures of FA more broadly, and we look forward to a continued partnership with the FDA through the Breakthrough Therapy designation and the [Controls Development and Readiness Pilot] program as we work to bring this potential treatment to patients as quickly as possible.”
Progress in autoimmune gene therapy partnership
Amarna Therapeutics and NorthX Biologics have finalized their agreement to advance Amarna’s gene therapy platform, Nimvec™, targeting immune diseases. Last year, the companies announced their partnership in which Amarna transferred its research-scale production process for Nimvec AM510 to NorthX Biologics.
Nimvec AM510 is designed to target proinsulin – an antigen that triggers autoimmune attack on beta cells, aiming to restore immune tolerance to insulin-producing beta cells.
“This agreement represents a major advancement in our partnership with NorthX Biologics and a critical step toward bringing Nimvec™ AM510 to patients,” said Henk Streefkerk, CEO of Amarna Therapeutics. “With this investment, we are accelerating our path to clinical trials and moving closer to providing a potential cure for Type 1 Diabetes. Collaborating with NorthX Biologics enables us to build a solid [Chemistry Manufacturing and Controls] foundation, ensuring we are well-prepared for the next phases of development. We’re eager to see our first clinical-grade batch produced.”