Medexus Pharmaceuticals (MDP) investor relations material

Strategy and business model

• Rare-disease portfolio of ~14 marketed products generates just under $100M in U.S. revenue and positive EBITDA; ~75% of revenue is U.S.-based, expected to rise toward 90%.

• Strategy is shifting toward hematology-oncology; ~50% of revenue is already from the targeted growth area, while mature products provide cash flow for investment.

• North American commercial infrastructure is built out; growth priorities include organic product uptake and licensing or acquisitions suited to the existing footprint.

• Focused transplant market includes ~180 U.S. institutions and ~17 in Canada; a concentrated hospital base enables a relatively small commercial footprint.

• Product development centers on label and lifecycle improvements, not drug discovery; the model takes commercial rather than development risk.

GRAFAPEX growth opportunity

• GRAFAPEX, approved in Jan. 2025, is the first and only product approved for AML and MDS transplant conditioning; first-year U.S. revenue was reported at $11.5M, with $11.6M cited for the prior year elsewhere in the presentation.

• About half of hospitals had ordered and used the product; reimbursement was described as broad, with Medicare NTAP support and no reported clinician prescription rejections.

• FY net revenue guidance is $30M–$32M, versus $11.6M in the prior year; management cited institution-level uptake tracking and Canadian launch experience as support for the aggressive outlook.

• Peak U.S. revenue potential is $100M–$175M, based on a 29%–42% share of allogeneic transplants; seven years of orphan-drug exclusivity, with lifecycle efforts to extend exclusivity.

• Treosulfan gross margin is ~80%, versus ~60% for the blended portfolio; a Canadian real-world study mirrored Phase III results showing 30% better overall survival at two years.

Pipeline and portfolio

• UM171 was licensed for Canada as a cell-source product in the transplant pathway; additional opportunities include graft-versus-host disease treatments for the same patients and institutions.

• IXINITY, a factor IX treatment for hemophilia B, is the largest current product; patent protection extends to 2028, and management cited limited biosimilar economics as a barrier to generic competition.

• Rasuvo/Metoject regained U.S. share after a competitor exited, making it the sole methotrexate auto-injector cited; management expects stability until exclusivity loss and generic entry around 2028–2030.

• Mature products are being deprioritized as growth investments shift toward hematology and stem cell transplantation; non-strategic mature assets could be sold to fund that expansion.