Iadademstat-Placebo Combo to Be Studied in Neuroendocrine Cancers

The first patient was enrolled in a phase 2 trial studying iadademstat (ORY-1001) plus paclitaxel in patients with relapsed/refractory, high-grade extrapulmonary (outside of the lungs) neuroendocrine carcinoma — a type of cancer that starts in the neuroendocrine system, which includes nerve and gland cells throughout the body — or relapsed/refractory small cell lung cancer.

The start of the trial was announced by Oryzon Genomics, the manufacturer of iadademstat.

The main goal of the trail is to determine overall response rate (percentage of patients whose tumors shrink as a result of treatment). Responses can be classified as a complete response, where there are no more signs of cancer left or partial response, where cancer is still present, but has shrunk.

Researchers on the trial will also analyze the rate of severe side effects, as well as progression-free survival, which is how long patients live after treatment before their disease gets worse.

“Relapsed and refractory (neuroendocrine carcinoma) and small cell lung cancer carry an unfortunately dismal prognosis and represent a major unmet medical need in oncology,” Dr. Douglas Faller, global chief medical officer of Oryzon said in a press release.

According to the release, neuroendocrine cancers — which include small cell lung cancer — can be very aggressive, depending on the subtype, and patients’ disease often progresses after frontline cytotoxic chemotherapy. To date, there is no second line treatment that is clearly efficacious, and there tends to be low response rates after frontline therapy.

To be eligible for the trial, patients must have metastatic or unresectable (unable to be removed via surgery) neuroendocrine cancer or small cell lung cancer. Trial participants must have undergone between one and three prior therapies, including a platinum-based chemotherapy regimen. They must have an ECOG status of 0 or 1, meaning that they have little — if any — restrictions on their activities of daily living, and have normal organ and marrow function, as defined by lab tests.

The researchers plan on treating patients and collecting data until August 2023, and completing the study in August 2026.

“Achieving first-patient-on in the study is an important milestone in our plan to utilize our epigenetic therapeutic drug, iadademstat, in combination with selected other agents, as a new approach to the treatment of NECs, with the potential to provide meaningful benefit to these patients,” Faller said.

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