Spexis provides business update and announces financial results for the full year 2022 - Seite 2

In April 2023, Spexis reported the closing of a capital commitment of USD 4.5 million from SPRIM Global Investments (SGI) to enable the initiation of the ColiFin Phase 3 COPILOT study. COPILOT has study start-up activities ongoing and is expected to enroll its first patient in Q3 2023 with data expected in the first quarter of 2024. Additional financing will be required to continue the clinical development of ColiFin into the COPA pivotal study.

The clinical development of ColiFin is supported by an FDA Orphan Drug Designation for treatment of respiratory infection in patients with cystic fibrosis, Qualified Infectious Disease Product (QIDP) Designation for ColiFin for the treatment of PA lung infections in CF patients, and Fast Track Designation.

Balixafortide

Spexis announced positive results from the Phase 1 clinical trial of balixafortide (BLX) in patients with renal impairment in September 2022. The Phase 1 trial was designed to investigate the pharmacokinetics, safety and tolerability of BLX in subjects with mild (n=8), moderate (n=8), or severe (n=7) renal impairment compared to a control group (n=8) with normal renal function. Each person received a single 2-hour intravenous infusion at the previously studied clinical dose of 5.5 mg/kg of BLX. The data from this trial indicate that BLX doses substantially higher than 5.5 mg/kg, the highest dose previously tested, and can potentially be safely administered while stimulating higher levels of stem cell mobilization for longer durations well beyond 24 hours. Increase of functional stem cells, also called mobilization, is an important prerequisite to the collection and transplantation of bone marrow cells in patients with hematologic malignancies.

Inhaled murepavadin

In January 2023, Spexis reported promising safety and pharmacokinetics results from a first-in-human study with inhaled murepavadin (iMPV), a novel macrocycle compound. The proprietary compound was derived from the company’s macrocycle platform and targets the outer membrane of Pseudomonas aeruginosa (PA) including resistant strains present in cystic fibrosis infections. The Phase 1 trial was a single center, double blind, randomized placebo-controlled trial to investigate the safety, tolerability, and pharmacokinetics of single ascending doses of iMPV in healthy volunteers. At the highest single dose tested, systemic bioavailability of MPV was lower than 5%, and peak plasma concentrations were observed 1-2 hours post start of inhalation. Pharmakokinetic results showed the concentration of iMPV at the 24-hour timepoint was still above the concentration that would inhibit the growth of 90% of PA isolates (MIC90) obtained from people with CF. The data suggested that iMPV leads to concentrations in the airways of the lung sufficient to target PA, while remaining lower than intravenous administration. This favorable tolerability, safety, and concentration profile provides support for future clinical trials of iMPV in people with CF or non-CF bronchiectasis.