All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit Know AML.

The AML Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy

Introducing

Now you can personalise
your AML Hub experience!

Bookmark content to read later

Select your specific areas of interest

View content recommended for you

Find out more
  TRANSLATE

The AML Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the AML Hub cannot guarantee the accuracy of translated content. The AML Hub and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.

Steering CommitteeAbout UsNewsletterContact
LOADING
You're logged in! Click here any time to manage your account or log out.
LOADING
You're logged in! Click here any time to manage your account or log out.
2020-05-14T16:58:39.000Z

Phase III study of omidubicel in patients with high-risk hematologic malignancies meets primary endpoint

May 14, 2020
Share:

Bookmark this article

Positive results from a phase III study of omidubicel, a cell therapy product of expanded umbilical cord blood (UCB) cells, (NCT02730299) have been announced.1 The study investigated the safety and efficacy of omidubicel, compared to standard UCB transplant, in patients with high-risk hematological malignancies without a suitable matched donor.2

 Key features3,4

Omidubicel

  • An advanced cell therapy product consisting of nicotinamide (NAM)-expanded stem cells and differentiated immune cells from UCB
    • NAM inhibits differentiation and enhances the functionality of cultured hematopoietic stem and progenitor cells, which substantially increases bone marrow homing and engraftment potential
  • Can be cryopreserved and transplanted after myeloablative conditioning as a standalone hematopoietic stem cell graft
  • The first bone marrow transplant product to receive the United States Food and Drug Administration (FDA) breakthrough therapy designation and has also received orphan drug designation in the United States and European Union.
  • Proven in a phase I/II study, published in the Journal of Clinical Oncology, to induce rapid and durable engraftment, while being well tolerated

 Study design2

  •  Open-label, controlled, multicenter, international, randomized, phase III trial comparing stem cell transplantation with omidubicel vs standard UCB in patients with high-risk hematologic malignancies
  • Started in November 2016 and completed in December 2019
  • Included 125 patients aged 12–65 years with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome, lymphoma, or acute leukemia1
  • Primary endpoint: Time to neutrophil engraftment 

Top-line results from the study demonstrated that treatment with omidubicel significantly reduced the median time to neutrophil engraftment by 10 days compared to standard UCB transplant (p < 0.001) and was generally well tolerated. Furthermore, a greater percentage of patients achieved successful neutrophil engraftment compared with patients in the comparator group (96% vs 88%, respectively).1 

Full efficacy and safety results are expected to be presented later this year, and a biologics license application is due to be submitted to the FDA by the end of the year.

  1. gamidaCell.Gamida Cell announces positive topline data from phase 3 clinical study of omidubicel in patients with high-risk hematologic malignancies. https://investors.gamida-cell.com/node/7236. Published May 12, 2020. Accessed May 12, 2020.
  2. Clinicaltrials.gov. Stem cell transplantation with NiCord® (omidubicel) vs standard umbilical cord blood in patients with leukemia, lymphoma, and myelodysplastic syndrome (MDS). https://www.clinicaltrials.gov/ct2/show/study/NCT02730299 Updated April 14, 2020. Accessed May 12, 2020.
  3. gamidaCell. Omidubicel: An investigational advanced cell therapy designed to enhance the life-saving benefits of bone marrow transplant. http://www.gamida-cell.com/wp-content/uploads/Omidubicel-Fact-Sheet.pdf. Accessed: May 12, 2020.
  4. Horwitz ME, Wease S, Blackwell B, et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019;37(5):367-374. DOI: 1200/JCO.18.00053

Your opinion matters

Do you intend to implement next generation sequencing for measurable residual disease monitoring in AML patients?
0 votes - 5 days left ...

Newsletter

Subscribe to get the best content related to AML delivered to your inbox