X4 PHARMACEUTIC. DL-,001

The European Commission has approved XOLREMDI (mavorixafor) from X4 Pharmaceuticals for once‑daily oral treatment of patients aged 12 and older with WHIM syndrome. The approval was granted under exceptional circumstances and is based on the Phase 3 4WHIM study; mavorixafor is thus the first and only approved therapy for WHIM in the US and the EU. Norgine will lead commercialization in Europe, Australia and New Zealand following regulatory approvals; under the licensing agreement X4 can receive up to €226 million in milestone payments plus tiered royalties. » Więcej na de.investing.com

At the Leerink Global Healthcare Conference, X4 Pharmaceuticals presented a strategic refocus on chronic neutropenia and the regulatory Phase‑3 study 4WARD for mavorixafor. Recruitment for the study is expected to be completed by the end of Q3 2026, with top‑line data anticipated in 2027 and commercialization for 2028 fully funded. The company reports significant cost reductions, personnel additions from CTI BioPharma, and positions mavorixafor as an oral alternative to G‑CSF for a U.S. market estimated at ~15,000 symptomatic patients. » Więcej na de.investing.com

At the Guggenheim Summit, X4 Pharmaceuticals outlined its priorities: focus on the Phase‑3 4WARD study for mavorixafor, operating cost cuts and workforce reductions to consolidate resources. The oral drug is expected to offer advantages over G‑CSF; the addressable market is seen at about 15,000 patients, with roughly 5,000 patients to be treated initially. Commercialization for WHIM syndrome has been postponed; the pricing strategy foresees a reduced but still premium level versus the current list price of about USD 500,000. Approval is targeted for 2028. » Więcej na de.investing.com

X4 Pharmaceuticals has strategically refocused development of mavorixafor on treating chronic neutropenia and raised nearly $240 million to complete the ongoing Phase‑3 FORWARD study and fund a launch. Study recruitment is more than one third complete; target to finish enrollment is end of Q3 2026, with top‑line data expected in H2 2027. The company aims for a U.S. launch by 2028, plans premium pricing and a collaboration with Norjin for Europe. Commercialization for WHIM syndrome has been deferred and the leadership structure was reorganized to accelerate development. » Więcej na de.investing.com

X4 Pharmaceuticals is offering 45.86 million common shares at $2.90 and pre-funded warrants for up to 700,000 shares at $2.899, and expects gross proceeds of about $135 million before costs. The offering includes a 30-day over-allotment option for up to 6.984 million shares; closing is scheduled for October 27, 2025. Proceeds are intended primarily to fund the phase‑3 development of mavorixafor for certain chronic neutropenic disorders and for general corporate purposes. In parallel, X4 announced a strategic restructuring including a 50% workforce reduction, leadership changes and expected annual cost savings of about $13 million. » Więcej na de.investing.com