A Potential Best-in-Class Anti-Cancer Akt-1 Inhibitor

RX-0301 is a potential best-in-class, potent inhibitor of the protein kinase Akt-1, which we believe plays a critical role in cancer cell proliferation, survival, angiogenesis, metastasis, and drug resistance. RX-0301 works by specifically inhibiting the cancer cell signaling protein Akt-1, which is highly over-expressed in cancer cells. We believe RX-0301 is different from other Akt-1 inhibitors by its ability to inhibit both activated and inactivated forms of Akt-1, and as a result, it is not expected to lead to drug resistance. RX-0301 has received orphan drug designation from the FDA for renal cell carcinoma (RCC), glioblastoma, ovarian cancer, stomach cancer, and pancreatic cancer.

Inhibiting both Activated and Inactivated Forms of Akt-1

Akt-1 is over-activated in patients with multiple forms of cancer, including breast, colorectal, gastric, pancreatic, prostate, and melanoma cancers. Akt-1 activity may be inhibited by signaling molecules upstream of Akt-1 in cancer cells through the use of vascular endothelial growth factor and epidermal growth factor receptor inhibitors, but this treatment only indirectly affects the activity of native Akt-1. Because signal transmission for cancer progression and resistance occurs when Akt-1 is activated, inhibition of activated Akt-1 has the potential to prevent cancer progression.

Candidate & Indication Collaborators Preclinical Phase 1 Phase 2


Hepatocellular Carcinoma

Preclinical in progress
Phase 1 not started
Phase 2 not started

Clinical Development

Phase 2a Proof-of-Concept

Phase 2a proof-of-concept clinical trial of RX-0301 began in January 2014 to study its safety and efficacy in combination with Afinitor® (everolimus) in patients with metastatic RCC. This trial was conducted in two stages. Stage 1 was a dose ranging study, with up to three dose groups with three RCC patients each, to determine its maximum tolerated dose (MTD) in combination with everolimus. In January 2016, Rexahn completed Stage 1 of the study and a maximum tolerated dose of RX-0301 was identified for further study.

The results from Stage 1 of the Phase 2a clinical trial showed that, in RCC patients who had previously received multiple anti-cancer therapies, RX-0301 treatment produced both stable disease (which persisted for up to 383 days) and a reduction in tumor burden. Compared to baseline CT scans, three patients experienced reductions in the size of their tumors of up to 36%. At the lowest dose level of RX-0301 administered (125 mg/m2/day), one patient had a 16% tumor reduction after four cycles of treatment. At the second dose level (200 mg/m2/day) one patient experienced a 36% tumor reduction after two cycles of treatment. At the highest dose level (250 mg/m2/day), which has been determined to be the maximum tolerated dose, one patient had a 32% tumor reduction following six cycles of treatment. RX-0301 appeared to be safe and well tolerated with minimal side effects in patients with advanced cancers, where Grade 3 fatigue was the only dose-limiting toxicity observed. In addition, the most commonly reported adverse event in patients taking the combination of RX-0301 and everolimus was thrombocytopenia.

Stage 2

Stage 2 of the study was initiated in 2016, but terminated in January 2018 to focus on hepatic cell carcinoma (HCC) as the lead indication.

RX-0301 is now in preclinical development for HCC in collaboration with Zhejiang Haichang Biotechnology Co. Ltd.

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