Disc Medicine

Disc Medicine will present data from multiple hematology programs at the European Hematology Association 2025 Congress, including long-term extension trial results for bitopertin in erythropoietic protoporphyria.

Disc Medicine has initiated the APOLLO confirmatory trial for bitopertin in erythropoietic protoporphyria (EPP) and remains on track to submit an NDA under accelerated approval pathway in the second half of 2025.

Alterity Therapeutics has raised A$40 million through a two-tranche placement to advance ATH434, their lead compound showing promising results in Multiple System Atrophy treatment.

• Gemini Therapeutics has successfully concluded a Type C meeting with the FDA, reaching agreement on the design of APOLLO confirmatory trial for bitopertin in EPP and XLP treatment. • The planned APOLLO trial will enroll approximately 150 patients across multiple continents in a randomized, double-blind, placebo-controlled study with a 1:1 allocation ratio. • The company aims to submit a New Drug Application for bitopertin under the Accelerated Approval Program in the second half of 2025, with trial initiation planned for mid-2025.

• Disc Medicine is preparing for an NDA submission for bitopertin in erythropoietic protoporphyria (EPP) following positive FDA feedback, potentially leading to accelerated approval. • Phase 2 trials for DISC-0974 in anemia of myelofibrosis (MF) and chronic kidney disease (CKD) are progressing, with initial data expected in the second half of 2025. • A Phase 2 study of DISC-3405 in polycythemia vera (PV) is set to begin in H1 2025, expanding Disc Medicine's portfolio in hematologic diseases. • Disc Medicine's strengthened financial position, with a cash runway into 2027, supports the advancement of its clinical programs and strategic objectives.

Alterity Therapeutics' ATH-434 demonstrated statistically significant slowing of clinical progression in early-stage multiple system atrophy (MSA) patients.

• Disc Medicine presented Phase 1b trial results of DISC-0974 for myelofibrosis-associated anemia at the ASH annual meeting, showing significant clinical benefits. • In non-transfusion dependent patients, 68% achieved a hemoglobin increase of ≥1.5 g/dL, while 100% of low transfusion dependent patients reduced transfusion needs by ≥50%. • DISC-0974 demonstrated a >75% reduction in hepcidin levels across all patient groups and was generally well-tolerated, with diarrhea being the primary adverse event. • Following these positive outcomes, Disc Medicine has initiated a Phase 2 trial to further evaluate DISC-0974 in myelofibrosis patients with anemia.

• Disc Medicine has obtained a $200 million non-dilutive term loan to support its clinical trials and enhance financial flexibility. • The funding will facilitate a confirmatory study of bitopertin for erythropoietic protoporphyria (EPP) and Phase II trials for DISC-0974 and DISC-3405 in anemia. • A multiple-dose trial in anemia of non-dialysis dependent chronic kidney disease (NDD-CKD) is also planned with the secured financing. • The company anticipates initiating a confirmatory trial of bitopertin in EPP by mid-2025, supported by this financial boost.

• Disc Medicine's bitopertin may receive accelerated FDA approval for erythropoietic protoporphyria (EPP) treatment based on existing clinical data. • The FDA has agreed to the design of Disc's Phase III APOLLO trial, which will serve as a confirmatory study for bitopertin. • Bitopertin aims to reduce protoporphyrin IX (PPIX) levels, potentially alleviating pain and burns from sun exposure in EPP and XLP patients. • The Phase III APOLLO trial is set to begin in mid-2025 and will assess bitopertin's safety and efficacy in patients aged 12 and older.

• Disc Medicine received positive feedback from the FDA regarding bitopertin for erythropoietic protoporphyria (EPP), potentially allowing for accelerated approval based on PPIX reduction. • Analyst ratings for Disc Medicine have been upgraded to 'Strong Buy' by Raymond James, with a price target of $110, citing the accelerated timeline for bitopertin's launch. • The company is expected to submit a New Drug Application (NDA) for bitopertin in the second half of 2025, with potential approval and commercial launch anticipated in 2026. • Phase 1b study of DISC-0974 showed significant reduction in hepcidin levels and improvement in iron mobilization and hemoglobin levels in patients with NDD-CKD and anemia.