SciClone Pharmaceuticals (China) Co., Ltd

Introduction: The CD47 and SIRPα checkpoint pathway has garnered much interest within the anti-cancer research community, with multiple experimental checkpoint inhibitors targeting CD47 and SIRPα in development. The use of such checkpoint inhibitors may however be limited by hematologic toxicity.Areas covered: We report on RRx-001, the first known small molecule downregulator of CD47 and SIRPα, which has shown a lack of hematologic toxicity in clinical trials.Expert opinion: RRx-001 is the first reported small molecule downregulator of CD47 and SIRPα and lacks any notable hematologic or systemic toxicity as demonstrated in clinical trials to date. Small molecule RRx-001 could be used in combination with or in place of CD47 targeting antibodies for anti-cancer treatment.

RRx-001, a CD47 antagonist via its inhibition of MYC and the γ-subtype of the peroxisome proliferator-activated receptor (PPAR) has been associated to date with minimal toxicity.The aim of this post-hoc anal. was to evaluate the toxicity and efficacy of RRx-001 in Asian patients since RRx-001, in the context of multiple Phase 3 studies, will be administered in China and Chinese territories as well as potentially throughout the rest of Asia.Patients received 4 mg of RRx-001 in three different antitumor clin. trials with chemotherapy and/or radiation and a retrospective subset efficacy and toxicity anal. was conducted for patients with Asian ancestry in comparison to patients with other ethnic backgrounds.The toxicity and efficacy data from these studies were similar between Asians and the rest of the treated patients.While the sample sizes are too small to draw definitive conclusions, at a dose of 4 mg, when RRx-001 is combined with chemotherapy, no apparent differences in terms of safety and efficacy are observed in cancer patients with Asian ancestry.