The US Food and Drug Administration (FDA) has granted an orphan drug designation to Immune-Onc Therapeutics’ IO-202 for the treatment of a rare blood cancer – chronic myelomonocytic leukaemia (CMML).

IO-202 had previously received a fast track designation from the US FDA as a treatment of relapsed or refractory (r/r) CMML in 2023. The therapy was also granted both orphan drug and fast track designations as a treatment for acute myeloid leukaemia (AML) by the FDA in 2020 and 2022, respectively.

The orphan drug designations allow for an extended market exclusivity of seven years in the US and developmental incentives such as an exemption of FDA application fees and tax credits for qualified clinical testing. The fast track designations enable earlier interactions with the FDA for the pursuit of accelerated approval. The status also opens chances of rolling reviews.

IO-202 is a monoclonal antibody that targets leukocyte immunoglobulin-like receptor subfamily B member 4 (LILRB4), which can suppress immunity in solid tumours. It also mediates the T-cell suppression and infiltration of AML cells.

It is being investigated as a monotherapy and in combination with chemotherapy, azacytidine, along with targeted therapy AbbVie/Genentech’s Venclexta (venetoclax), in a Phase I trial (NCT04372433). The study is expected to enrol approximately 106 patients with either r/r AML or r/r CMML. It is expected to conclude in 2026, as per

A second Phase I trial (NCT05309187) investigating IO-202 in combination with a PD-1 immune checkpoint inhibitor, MSD’s Keytruda (pembrolizumab) is also currently underway. The study enrolled approximately 200 patients with advanced solid tumours and is expected to be completed in April.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of 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.

Monoclonal antibodies have been identified as a key innovation area for cancer therapy by GlobalData, with multiple companies investing to develop antibody therapies for various indications. In December 2023, AbbVie partnered with BigHat Biosciences to identify and develop antibody therapies in oncology and neuroscience indications.

GlobalData is the parent company of Pharmaceutical Technology.

Almirall is developing an interleukin-21 (IL-21)-hindering monoclonal antibody, NN-8828, for the treatment of immune-inflammatory dermatological ailments. The company acquired the therapy from Novo Nordisk last week for an undisclosed upfront payment and will be entitled to developmental and commercial milestones, as well as tiered royalties on future global sales.