ACG-301/401 (Oncology)

ACG-301 (DNAK inhibitor)

The overlap of our microbiota and cancer development, progression and treatment are a new field in cancer treatment development. For patients with rare cancers, or those that develop tolerance to current treatments, options are limited. Of particular interest to Aceragen is the emerging link between specific microbiologic alarmone products that interfere with chemotherapeutic efficacy. Aceragen is developing antagonists to these microbiologic products in hopes of enhancing the efficacy of available treatments for patients with few therapeutic options.

ACG-401 (leptin-receptor inhibitor)

A new paradigm for the treatment of cancer is integrating leptin signaling inhibition into cancer care. Leptin is produced by adipose tissue and is a regulator of body weight and energy balance. Leptin also plays a significant role in cancer risk, progression, and metastasis. Many cancer patients have higher levels of leptin, as well as their receptor (LEPR) which are linked to worse prognosis and survival. Aceragen is developing a leptin receptor antagonist, a first-in-class metabolic modulator that can impact multiple rare solid tumors.

Aceragen has a set of drug development programs with application to various oncology indications with definable orphan patient populations and high unmet medical need. These programs are progressing rapidly through pre-clinical development based on lead compounds with separate and novel mechanisms of action. Aceragen is collaborating with leading academic partners to collect in vivo or ex vivo proof of concept data in appropriate disease models to support indication selection and clinical entry.