BRILACIDIN

Brilacidin is Innovation Pharma’s lead drug candidate in its Host Defense Protein (HDP)-mimetic franchise.

Modeled after HDPs, the “front-line” of defense in the body's innate immune system, Brilacidin is a de novo-designed (pdf) synthetic, non-peptidic, small molecule that kills pathogens swiftly, greatly reducing the likelihood of drug resistance developing. Brilacidin also functions in a robust immunomodulatory capacity, lessening inflammation and promoting healing.

Brilacidin has been tested in over 500 human subjects across multiple clinical indications.

COVID-19

Brilacidin has received FDA Fast Track for COVID-19 and was evaluated in a placebo-controlled Phase 2 clinical trial in hospitalized COVID-19 patients (see NCT04784897). While the trial’s primary endpoint of time to sustained recovery through Day 29 was not met, patients who started study treatment <7 days of onset of COVID-19 symptoms achieved sustained recovery more quickly (Brilacidin 5-dose group versus pooled placebo, p=0.03). Other beneficial treatment effects based on the trial’s primary endpoint of sustained recovery were also observed in patient subgroups with the highest baseline values for key COVID-19 biomarkers. On two secondary endpoints, more patients treated with Brilacidin (5-dose group) achieved clinical improvement by 10 days as measured by National Emergency Warning Score 2 (NEWS2) criteria, and the mean change from baseline in NEWS2 was greater for Brilacidin treatment groups at all assessment timepoints.

ORAL MUCOSITIS

Innovation Pharma has evaluated Brilacidin, under FDA Fast Track designation, in a Phase 2 clinical trial (see NCT02324335) as an oral rinse to attenuate Oral Mucositis (OM) in patients with Head and Neck Cancer (HNC) who have received chemoradiation. Study results indicated Brilacidin has a high potential for preventative treatment, as evidenced by a clear reduction of Severe OM (SOM) among patients on Brilacidin as compared to those on placebo. Additional secondary endpoint analysis showed Brilacidin delayed the onset, as well as reduced the duration, of SOM. The Company and the FDA have completed an End-of-Phase 2 meeting, with both parties agreeing to an acceptable Phase 3 development pathway. Provider and payer analysis further supports the potential commercialization of Brilacidin as a novel OM drug candidate.

INFLAMMATORY BOWEL DISEASE

Innovation Pharma has tested Brilacidin, administered with water in an enema, in patients with Ulcerative Proctitis/Ulcerative Proctosigmoiditis (UP/UPS) — a type of Ulcerative Colitis (UC), and, like Crohn's Disease, an Inflammatory Bowel Disease (IBD) — in a proof-of-concept Phase 2 clinical trial. Being developed as a novel, non-corticosteriod, non-biologic IBD treatment, a majority of UP/UPS patients treated with Brilacidin achieved induction of clinical remission. Based on these results, Alfasigma S.p.A licensed Brilacidin for this indication. Additional formulation development work for oral delivery of Brilacidin in IBD has been conducted. Also of note, the academic literature suggests a defensin deficiency in IBD, impacting the mucosal immune system, indicating Brilacidin may have a compensatory effect in this regard.

SERIOUS SKIN INFECTIONS

As a late-stage antibiotic drug candidate, Brilacidin has received FDA Qualified Infectious Disease Product (QIDP) designation — qualifying the drug candidate for Fast Track and Priority Review, as well as an extra 5 years of market exclusivity upon drug approval. A Phase 2b trial (see NCT02052388) was completed evaluating intravenous Brilacidin for treatment of Acute Bacterial Skin and Skin Structure Infection (ABSSSI). Trial data showed a single dose of Brilacidin to be comparable in safety and efficacy to a 7-day dosing regimen of FDA-approved daptomycin.

HARD-TO-TREAT FUNGAL DISEASES

In pre-clinical in vitro and in vivo studies, Brilacidin has been shown to synergize with marketed antifungals against pathogenic species of aspergillosis, candidiasis, cryptococcosis and mucormycosis, and further exhibited potent stand-alone efficacy against Cryptococcus neoformans. Research is ongoing, in collaboration with NIH/NIAID-affiliated and other academic researchers. Collectively, data generated to date suggest Brilacidin has potential to be developed as a novel antifungal agent. A corresponding scientific paper, published in Nature Communications, is available, as well as supporting scientific presentations — 16th Annual European Conference on Fungal Genetics and 2023 Gordon Research Conference on the Immunology of Fungal Infections.

“BRILACIDIN [...] HAS A HIGH LIKELIHOOD OF REACHING COMMERCIALIZATION.”

— Dr. Michael Zasloff