First-in-human trial launches in US to test drug for hard-to-treat AML
Developer evaluating safety, optimal dose of blood cancer treatment
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A first-in-human clinical trial testing Orum Therapeutics‘ infusion therapy ORM-1153 in people with hard-to-treat acute myeloid leukemia (AML), a form of blood cancer, has dosed its first participant.
This early study will be assessing whether the experimental therapy is safe and well tolerated in people with AML that has proven treatment-resistant. The main goals of the Phase 1 trial (NCT07809906) are to evaluate ORM-1153’s safety profile and identify the optimal dose(s) for future testing.
The trial expects to enroll an estimated 42 adults with AML that is relapsed or refractory, meaning the cancer has returned or failed to respond following prior lines of treatment. To be eligible to participate, participants’ cancer cells must express detectable amounts of a protein called CD123.
“This Phase 1 study will provide important initial information about ORM-1153’s safety, pharmacology, and potential activity in patients,” Andrew Sochacki, MD, associate director of hematology at START Midwest, the trial site in Grand Rapids, said in a press release from Orum.
The trial is expected to enroll participants at multiple sites in the U.S., including testing centers in Michigan and Texas. All participants will receive treatment with ORM-1153, administered at various doses by infusion into the bloodstream.
AML is an aggressive form of blood cancer, and when the cancer comes back or fails to respond after initial treatment, options for patients are limited.
“Patients with relapsed or refractory AML, particularly those with high-risk molecular features, continue to have poor outcomes and need additional treatment options,” Sochacki said.
Treatment candidate showed benefits in lab models of AML
ORM-1153 is a novel therapy consisting of an antibody targeting the CD123 protein that’s attached to a molecule capable of destroying the GSPT1 protein. AML cells rely on GSPT1 for their survival. The idea is that the antibody will deliver the GSPT1 degrader to AML cells, thus destroying this protein and ultimately killing the cancer cell.
“Targeted delivery of a GSPT1 degrader offers a different approach to treating these cancers,” Sochacki said.
We have built a substantial preclinical data package for ORM-1153, and our task now is to establish how those findings translate clinically.
According to Orum, ORM-1153 has shown promising results across various preclinical models of this cancer, with “demonstrated activity across … AML patient samples and [laboratory] models.”
Olaf Christensen, MD, the company’s chief medical officer, noted that Orum has been working closely with clinical site partners to make the transition from lab evaluations to clinical testing as quick and efficient as possible.
“We have built a substantial preclinical data package for ORM-1153, and our task now is to establish how those findings translate clinically. … The study will allow us to assess whether the improvements in payload delivery observed preclinically translate into a favorable balance of activity and tolerability in patients,” Christensen said.
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