X4 Pharmaceuticals(XFOR) - 2020 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of June 30, 2020, X4 Pharmaceuticals had $105.6 million in cash, cash equivalents, and restricted cash, which is expected to fund operations into early 2022 [22][23] - Research and development expenses were $9.3 million for Q2 2020, compared to $8.9 million for the same period in 2019, while general and administrative expenses increased to $5.3 million from $4.6 million year-over-year [24] - The net loss for Q2 2020 was $15.1 million, up from a net loss of $13.4 million in Q2 2019 [24] Business Line Data and Key Metrics Changes - The company is conducting a pivotal Phase 3 trial for mavorixafor in patients with WHIM syndrome, with ongoing enrollment and progress noted as sites reopen due to COVID-19 [15] - A Phase 1b trial for mavorixafor in patients with severe congenital neutropenia is also ongoing, with initial data expected in 2021 [16] - The Phase 1b study for Waldenstrom's macroglobulinemia is expected to enroll between 12 and 18 patients, with initial results anticipated by the end of 2020 [19] Market Data and Key Metrics Changes - Updated research indicates that the estimated range of diagnosed and undiagnosed WHIM patients in the U.S. is greater than 3,500, significantly up from the previous estimate of approximately 1,000 genetically diagnosed patients [10] - The company has identified a concentrated target physician population primarily consisting of immunologists, hematologists, and infectious disease specialists [10] Company Strategy and Development Direction - The company aims to own and commercialize in the rare disease markets while remaining opportunistic regarding partnerships as situations arise [64] - The focus remains on advancing mavorixafor for WHIM syndrome and exploring its potential in other indications, including Waldenstrom's macroglobulinemia [19][20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing clinical trials despite challenges posed by the COVID-19 pandemic, emphasizing patient safety and engagement [52][36] - The company plans to report topline Phase 3 results for WHIM in 2022, with further clarity on timelines for both WHIM and SCN expected in the future [15][16] Other Important Information - Dr. Renato Skerlj was promoted to Chief Scientific Officer, overseeing research and non-clinical development functions [20] - The collaboration with Invitae for generating genetic tests is ongoing, with no specific guidance yet on outputs [54] Q&A Session Summary Question: Any scenario for dose escalation beyond 600 mg in the Waldenstrom's trial? - Management indicated that while flexibility exists due to the drug's safety profile, they currently believe the 600 mg dose is sufficient [26] Question: Updates on potential collaborative partners for mavorixafor in solid tumors? - The partnership with Abbisko is progressing, but no updates on other strategic fronts outside of China [30] Question: Scope of the Phase 1b Waldenstrom's data? - The Phase 1b trial is a dose-escalating trial, with every patient starting at 200 mg and potentially escalating based on safety [33] Question: Enrollment status in clinical studies given the current environment? - Enrollment is maintaining guidance, with strong engagement from patients and physicians despite varying COVID-19 restrictions [52] Question: Thoughts on partnerships with new WHIM data and upcoming Waldenstrom's data? - The strategic focus remains on owning and commercializing in rare disease markets, with opportunistic partnerships considered as they arise [64]