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Octernal Stopping Clinical Testing, Reducing Workforce After Safety, Efficacy Issues in Two Trials

NEW YORK – Octernal Therapeutics on Thursday said it will stop all clinical product development, reduce staff, and explore strategic alternatives for several cancer drugs in its pipeline after a patient died in a CAR T-cell therapy trial and an investigational prostate cancer drug showed lackluster efficacy in another study.

In a filing with the US Securities and Exchange Commission, the company said it will lay off approximately 10 employees, or 37 percent of its workforce. Octernal Chief Medical Officer Salim Yazji will also stop serving in this role as of Oct. 1 as part of the workforce reduction.

The San Diego-based company was developing the autologous ROR1-targeting CAR T-cell therapy ONCT-808 in a Phase I trial in patients with refractory, aggressive B-cell lymphoma and testing ONCT-534, a dual-action androgen receptor inhibitor, in a Phase I trial involving metastatic castration-resistant prostate cancer patients resistant to androgen receptor pathway inhibitors.

While the CAR T-cell therapy showed anti-tumor activity at the tested doses in the Phase I trial, one patient died due to complications of shock at the highest dose. The dual-action androgen receptor inhibitor did not provide clinically meaningful improvement in prostate cancer patients' outcomes, including prostate-specific antigen levels, at interim analysis of its Phase I trial.

"Based on the available clinical data and capital requirements for continued development, the company has decided to terminate these studies," Octernal said in a statement, adding that it will explore strategic alternatives for ONCT-534 and ONCT-808, as well as two other assets, zilovertamab and ONCT-216.

The firm is testing the ROR1-targeting monoclonal antibody zilovertamab in various hematologic malignancies, including p53-mutated chronic lymphocytic leukemia.

ONCT-216 is an investigational inhibitor of the E26 transformation-specific family of oncoproteins including fusion proteins, which characterize most cases of Ewing sarcoma. Translocations or overexpression of E26 transformation-specific oncoproteins are also found in various leukemias, lymphomas, and prostate cancer. The firm is testing this drug in a Phase I/II Ewing sarcoma trial.

Octernal said it will explore selling or licensing these assets, as well as a merger, a reverse merger, an acquisition, and other options. The discontinuation of "all product development activities" and workforce reduction are part of the company's effort to preserve cash resources while it explores strategic options. 

In the SEC filing, Octernal said the workforce reduction will be completed in the third quarter and will result in $1.0 million in charges.