X4 Pharmaceuticals (XFOR) Presents At BMO Capital Markets Prescription For Success Healthcare Conference - Slideshow

Company Overview - X4 Pharmaceuticals is developing novel therapies targeting diseases resulting from CXCR4 pathway dysfunction to improve immune cell trafficking[6] - The lead product candidate, mavorixafor (X4P-001), is a first-in-class, oral, small molecule allosteric antagonist of chemokine receptor CXCR4[6] - The company is conducting multiple clinical trials, including a global registrational Phase 3 trial of mavorixafor in WHIM syndrome[6] Mavorixafor Clinical Trials and Indications - Mavorixafor is being investigated as a targeted treatment for diseases driven by immune-cell trafficking deficits, with lead indications including WHIM syndrome (Phase 3), Waldenström's Macroglobulinemia (Phase 1b), and Severe Congenital Neutropenia (Phase 1b)[11] - In a Phase 2 open-label extension study for WHIM syndrome, mavorixafor 400 mg orally once daily was well tolerated for over 2 years[33] - The Phase 2 WHIM trial demonstrated at least a 4.5-fold increase in TATANC (Time Above Threshold of Absolute Neutrophils Count) versus lower doses[34] - In the WHIM Phase 2 open-label extension, infection rates decreased from 4.63 to 2.14 (a 54% reduction) at the 400 mg dose[37] - The average wart burden was reduced by 75% after 14 months on 400 mg mavorixafor, compared to baseline[37] - A global registrational Phase 3 trial in WHIM syndrome is underway, randomizing patients 1:1 to mavorixafor (N=9) or placebo (N=9), with topline data expected in 2022[41, 42] - A Phase 1b trial of mavorixafor in combination with ibrutinib is underway in patients with MYD88 + CXCR4 mutations who have failed prior treatment for Waldenström's Macroglobulinemia, with initial data expected in 2H 2020[51] - A Phase 1b trial in Severe Congenital Neutropenia (SCN) is underway, enrolling up to 45 patients to assess responders to mavorixafor[61] Market Opportunity and Financials - The company estimates a significant market opportunity, targeting >10,000 total patients with rare diseases, including >3,500 with WHIM syndrome, 4,000-5,000 with CXCR4-mutant Waldenström's Macroglobulinemia, and 2,000-3,000 with Severe Congenital Neutropenia[64, 65, 67, 69] - As of March 31, 2020, the company had $117 million in cash, expected to fund operations into 2022[78]