View all newsletters
Receive our newsletter - data, insights and analysis delivered to you
In association with Cytiva for Cell & Gene Therapy coverage
  1. News
July 22, 2021

FDA places clinical hold on Magenta’s Phase I/II leukaemia trial

The requirement for a bioassay to inform dose escalation was the only clinical hold factor identified.

The US Food and Drug Administration (FDA) has issued a clinical hold letter for Magenta Therapeutics’ investigational new drug application to conduct a Phase I/II clinical trial of MGTA-117.

Magenta filed the IND last month. The trial for the treatment of acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) is yet to commence.

The FDA asked the company to develop a bioassay for use with the pharmacokinetic/pharmacodynamic model to help make decisions on dose escalation, apart from safety assessments.

Magenta noted that the bioassay was the single clinical hold factor provided by the FDA and is not associated with MGTA-117’s toxicology or production.

The company has already begun bioassay development and will collaborate with the FDA on the application of the product for dose escalation.

Magenta Therapeutics president and CEO Jason Gardner said: “We are actively developing the bioassay requested by the FDA and do not expect significant technical challenges in its completion.

“We expect to request a ‘Type A’ meeting in the coming weeks and, if successful in resolving this remaining issue, we would anticipate opening the study in Q4 2021.”

MGTA-117 is an antibody-drug conjugate developed to specifically reduce hematopoietic stem cells (HSCs) before the transplant or HSC-based gene therapy.

It acts on the CD117 receptor found on the cell surface of HSCs and leukaemia cells, indicating potential use in treating several diseases such as blood cancers and inherited metabolic disorders.

The Phase I/II trial is designed to assess the safety, pharmacokinetics and pharmacodynamics of MGTA-117 as a monotherapy in patients with relapsed/refractory AML and MDS.

Following adequate results in this patient population, Magenta intends to change the trial to transplant-eligible patients.

In addition, the company plans to develop MGTA-117 as a targeted conditioning agent for genetic disease patients before the use of ex vivo gene treatments.

Magenta reported in May this year that Phase II trial of its MGTA-145 in combination with plerixafor in multiple myeloma patients met the primary endpoint.



Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

Free Whitepaper

Secure the cell therapy supply chain from bench to bedside

The development of cell therapies is changing healthcare, delivering new hope to thousands of patients around the world. The vein-to-vein workflow for these therapies, however, is not without challenges, many of which will increase as we scale up to treat more patients. Download this free guide from Cytiva to learn more about the challenges and risks associated with the cryogenic supply chain for cell therapies, and how supply chain disruptions can best be mitigated.
by Cytiva Thematic

By clicking the Download Free Whitepaper button, you accept the terms and conditions and acknowledge that your data will be used as described in the Cytiva Thematic privacy policy By downloading this Whitepaper, you acknowledge that we may share your information with our white paper partners/sponsors who may contact you directly with information on their products and services.

Visit our privacy policy for more information about our services, how we may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Topics in this article:
NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy


Thank you for subscribing to Clinical Trials Arena