Cothera Bioscience Announces Orphan Drug Designation for PC-002 for the Treatment of Burkitt Lymphoma

San Francisco December 7, 2023 – Cothera Bioscience, a clinical stage biotech company developing therapeutics for previously undruggable oncology targets with high unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PC-002, a first-in-class deubiquitinase (DUB) inhibitor that induces myc degradation, for the treatment of Burkitt lymphoma.

Burkitt lymphoma is a rare but highly aggressive form of non-Hodgkin lymphoma, driven by the mutation of c-Myc rearrangement. It can be fatal and requires intensive chemotherapy. According to the FDA, orphan drug designation is a status assigned to a medicine intended for use against a rare condition. Drugs that have obtained orphan drug status can enjoy corresponding policy benefits, such as 7 years of U.S. market exclusivity after approval, NDA application fee exemptions, tax deductions for clinical research fees, and possible exemptions from reporting requirements for some clinical data.

“We are very pleased to receive orphan drug designation for PC-002, which reflects the urgent need for new therapeutic options for patients with Burkitt lymphoma,” said Dr. Alex Wu, Co-founder and CEO of Cothera Bioscience. “PC-002 is the most advanced clinical stage DUB inhibitor which works by accelerating the degradation of Myc oncoprotein, a key driver of Burkitt lymphoma and other cancers. We look forward to advancing PC-002 into a global phase 2 clinical trial and delivering a breakthrough for this devastating disease.”

About Cothera Bioscience, Inc.

Cothera Bioscience is a clinical stage biotech company developing therapeutics for previously undruggable oncology targets with high unmet medical needs. The company’s strength is in its clinically validated translational platform exploiting synthetic legality and protein degradation pathways for cancer killing. It was formed by the core founding members of Crown Bioscience, a well-known Contract Research Organization specializing in translational oncology, together with veteran serial entrepreneurs from Silicon Valley.

Clinical Pipeline

Leveraging the powerful translational science linking the i-CR® technology platform and clinical patient drug response, Cothera Bioscience has developed several innovative anticancer candidates in the field of synthetic lethality, protein degradation and immunotherapy. Cothera Bioscience has applied for multiple international patents covering the drug candidates as well as the i-CR® technology platform.

• PC-002

Currently in Phase 2 studies for multiple indications, Cothera’s lead program PC-002 is a first-in-class small molecule inhibitor of the DUB family enzymes targeting Myc- mutated tumors. PC-002 has demonstrated initial efficacy as well as safety in an ongoing monotherapy trial in relapsed and refractory high-grade B-cell lymphoma and Burkitt lymphoma patients with myc rearrangement (NCT05263583). With more than 50% of human cancers showing increased expression, Myc is regarded as one of the most important yet “undruggable” cancer targets. With a unique mechanism of action, PC-002 selectively induces Myc protein degradation and cell apoptosis in Myc-dependent tumors, potentially targeting multiple indications in cancers involving Myc dysregulation. PC-002 is also known as sepantronium bromide and YM155.

• Zotiraciclib (ZTR/TG02)

The second compound the company is developing is zotiraciclib (ZTR/TG02). ZTR is a potent oral CDK9 inhibitor that crosses the blood-brain barrier and degrades short-lived, anti-apoptotic oncogene proteins. The compound is being developed for the treatment of high-grade glioma (HGG) and diffuse intrinsic pontine glioma (DIPG), both brain cancers with high unmet medical need. ZTR has successfully completed a Phase 1b clinical trial sponsored and conducted by the US National Cancer Institute (NCI) that confirmed the safety and initially demonstrated effectiveness of ZTR in combination with temozolomide (TMZ) in patients with recurrent high-grade glioma. Recently the compound also demonstrated initial efficacy as well as safety in an on-going monotherapy trial targeting IDH-mutant high-grade glioma sponsored by NCI (NCT05588141).

Proprietary i-CR® technology platform

Cothera Bioscience developed a proprietary i-CR® technology platform, a powerful method for individualized drug screening and new drug development. The platform combines a high content screening system with conditional reprogramming of primary cultured tumor cells, which enables efficient and undifferentiated expansion of primary patient-derived tumor cells in vitro while preserving the tumor’s heterogeneity. Through collaboration with leading oncology medical centers, Cothera Bioscience has carried out prospective clinical trials and demonstrated that the i-CR® system can effectively predict the actual clinical response of a drug, with potential to significantly improve the clinical efficacy and success rate in anticancer drug development. (Transl Oncol. 2021 Jan;14(1):100935).

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