VIA THERAPEUTICS AWARDED PHASE I SBIR CONTRACT TO TREAT CHILDREN WITH TUBERCULOSIS

Current treatment regimens for Mycobacterium abscessus (Mab) lung infections require a combination of
multiple toxic drugs for prolonged periods, and exhibit success in only 30% of cases. Inhaled antibiotics are a
promising approach to increase antibiotic levels at the site of Mab infection while reducing systemic exposure,
but thus far inhaled monotherapy has proven ineffective for Mab treatment. Based upon promising in vitro
synergistic activity, we propose to evaluate a novel inhaled co-formulation of amikacin and clofazimine for the
treatment of Mab lung infections.

The incidence and mortality rates of cutaneous squamous cell carcinoma (cSCC) and skin cancer continues to increase. It is expected that 1 in 5 Americans will develop skin cancer in their lifetime. The delivery of drugs with reduction and prevention of tumor progression properties are able to be developed in a topical formulation that delivers the drug inside the skin while minimizing systemic absorption and adverse effects. 

Via Therapeutics is working ot develop a low-cost and shelf-stable minitablet formulation for the ease of treatment of tuberculosis in pediatric patients

Multidrug resistance tuberculosis (MDR-TB) is a global healthcare concern compounded by poor compliance associated with lengthy drug susceptible TB (DS-TB) treatment regimens. Clofazimine (CFZ) is highly active against MDR-TB, but oral use is limited by systemic toxicity (hyperpigmentation, GI effects, and QT prolongation). Inhalation of CFZ will overcome these limitations by achieving high local concentrations of drug and reducing systemic side effects.

Prostate cancer is the most common diagnosed cancer developing in men, developing in around 160,000 men
in the United States. While many men are successfully treated of the disease, nearly 30,000 continue to die each year to do an advanced cancer which continues to grow despite castration levels of androgen present. Niclosamide has recently been discovered to inhibit at least splice variant expression AR-V7, which in combination with next generation androgen receptor signal inhibitors has shown a synergistic effect. If successful, the proposed research would contribute to the development of a new treatment for patients with
castration-resistant prostate cancer, improving the progression free survival in these patients.

Rheumatoid arthritis is a chronic inflammatory disease that manifests as painful and swollen joints and subcutaneous nodules, among other symptoms. We recently developed a
new TNF-siRNA solid lipid nanoparticle formulation that is efficacious in treating RA in a mouse model of collagen antibody induced arthritis. However, a key issue with siRNA therapy is the toxicity associated with the acute inflammatory response induced by the siRNA. Our long-term goal is to develop a safe and effective siRNA based RA therapy using an innovative formulation approach.