Next Generation Immunotherapy
for Improved Oncology Outcomes

Targeting the causal Pathophysiology of febrile neutropenia By enhancing innate immunological defenses 

Akthelia Pharma is a preclinical-stage biopharma company targeting the large unmet market opportunity to improve cancer treatment and cell/organ transplant outcomes through enhancing the efficacy and reducing the leading risk factor associated with current cytotoxic chemotherapy. 

  • Low neutrophil count resulting from cytotoxic chemotherapy is associated with an increased risk of severe bacterial and fungal infections. This has now been causally linked to mucositis - cytotoxic damage to the lining of the GI tract and other mucosa, which leads the immunocompromised host to be vulnerable to invasion by infectious pathogens from their own microbiota. The current treatment paradigm of prophylaxis with fluoroquinolone antibiotics is unpopular with oncologists not only because of drug toxicity, but also because it leads to increased spread of drug-resistant bacteria and dysbiosis of the intestinal microbiota, which further encourages colonisation by professional pathogens. An antimicrobial approach is also fundamentally ineffective because it does not halt the translocation of infectious organisms through the GI tract wall.
  • Our therapeutic strategy, which is based on a depth of field-leading cell biology expertise from our science founders, is to eliminate bacterial translocation by enhancing the innate immunologic barrier resistance of the GI tract. We do this through upregulating endogenous proteins that are naturally expressed within epithelia and normally act to contain the microbiota within the gut lumen in a healthy individual. This holds the promise of a new standard of care treatment with potential to replace prophylactic use of antibiotics in patients at risk for febrile neutropenia, with reduced risk of SAEs, AMR, and microbiome dysbiosis. We have encouraging data that shows that our lead NCE candidate effectively knocks down translocation in murine models and are currently completing pre-clinical studies to demonstrate translatable survival benefit. This highly differentiated and patented approach has been enthusiastically endorsed by top KOLs in oncology.Akthelia's disruptive, patented immunological enhancement platform and lead therapeutic program has broad application and addresses the growing threat of antibiotic bacterial resistance worldwide.

  • Our clinical strategy targets the underlying causal pathophysiology of febrile neutropenia, the most common (~1% of chemo- and radiotherapy patients) and serious, life-threatening complication associated with patients receiving chemotherapy for cancer or who are immunocompromised to avoid transplant rejection. Febrile neutropenia is often dose-limiting in treatment with significant mortality rates -- 5% up to 50% in high-risk populations, with >60,000 hospitalizations and >4,000 deaths annually costing ~$2.8B (2012) in the US alone, >8% of all cancer-related hospitalization costs.

Differentiation over existing/competitive approaches and the supporting data

  • Combined strengthening of epithelial barrier(tight junctions) and upregulation of host defence peptides in epithelial cells and macrophages.
  • Broad spectrum therapy with a different mode of action from antibiotics - immunomodulation that induces production of host defense peptides which then kill the bacteria.
  • Pathogens are unlikely to become resistant, as production of multiple host defense microbial factors is induced. This is in contrast to antibiotics, which act directly on the microbes, thus quickly selecting for resistant strains.
  • Minimal impact on microbiota.