Annamycin shows encouraging early results in Phase 2/3 AML clinical trial
Interim data show 40% composite remission ahead of first unblinding
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Early results from the Phase 2/3 MIRACLE clinical trial suggest that the experimental chemotherapy drug annamycin may offer meaningful benefit as a second-line treatment for people with relapsed or refractory acute myeloid leukemia (AML), an aggressive and often difficult-to-treat blood cancer.
So far, 35 people have been treated, and Moleculin Biotech, which is developing annamycin and sponsoring the trial, said another 11 potential participants have been identified, with some already undergoing screening.
The trial is designed so that once 45 patients have been treated and their results have been evaluated and verified, the data will be unblinded — meaning researchers will learn which participants received annamycin and which received placebo. The company said it expects to treat the 45th patient in the coming weeks and unblind the data later this year.
“With the first unblinding now clearly in sight, we are entering what we believe is a transformational period for the company,” Walter Klemp, chairman and CEO of Moleculin, said in a company press release.
MIRACLE trial evaluates annamycin with cytarabine in AML
The MIRACLE clinical trial (NCT06788756) aims to enroll more than 300 adults with relapsed or refractory AML — meaning the cancer has not responded to or has returned after one prior treatment. All patients in the trial receive the approved chemotherapy medication cytarabine. Participants are randomly assigned to receive either annamycin or a placebo, in addition to cytarabine.
According to Moleculin, interim data from the first 30 participants showed that 30% achieved complete remission, meaning no detectable cancer and recovery of blood counts. Another 10% achieved complete remission with partial hematological recovery, meaning no detectable cancer but incomplete recovery of certain blood counts. The company said these response rates appear higher than what has historically been seen with cytarabine alone.
“The blinded efficacy rates we’re seeing in MIRACLE are exceptionally encouraging,” Klemp said. “And, of course, this includes the control arm of cytarabine plus placebo, which suggests that the Annamycin results should be better. Even with the control arm included, these preliminary, blinded results substantially outperform historical outcomes for [complete response] with cytarabine alone and reinforce our expectations of what Annamycin can become in the treatment of [relapsed/refractory] AML.”
Klemp also said many participants had genetic factors associated with high-risk disease. More than a third of the patients had relapsed or refractory AML following treatment with a regimen that included Venclexta (venetoclax). Patients who failed to respond to Venclexta-based regimens are “generally considered among the most challenging to address with 2nd line therapies,” Klemp said.
Annamycin designed to limit heart-related side effects
Annamycin belongs to a class of chemotherapies called anthracyclines, which work by damaging the DNA of cancer cells. A major drawback of currently available anthracyclines is their potential to cause heart damage, a side effect known as cardiotoxicity. Annamycin is a novel drug in this class that aims to retain anticancer potency without the risk of heart damage. According to Klemp, early data from MIRACLE are consistent with prior studies suggesting the therapy can target cancer cells without evidence so far of cardiotoxicity.
“[W]hen you factor in that we estimate that one third of the subjects treated so far [in MIRACLE] did not receive Annamycin, we believe it is likely that Annamycin is performing at or near the impressive level we saw in our last Phase 2 trial,” Klemp said.
“Coupling this with the recently released safety data from 90 total subjects in prior clinical trials demonstrating Annamycin’s lack of cardiotoxicity, we believe Annamycin has the potential to offer a new avenue for patients battling this challenging disease. We are looking forward to the upcoming significant milestone for the Company and the MIRACLE trial of the unblinding of each arm’s efficacy for the first 45 subjects.”
