Disc Medicine Advances Bitopertin NDA for Erythropoietic Protoporphyria as Pipeline Progress Continues
核心洞察
Disc Medicine has initiated the APOLLO confirmatory trial for bitopertin in erythropoietic protoporphyria (搜索) (EPP) and remains on track to submit an NDA under accelerated approval pathway in the second half of 2025.
The company is advancing its hematologic disease pipeline with DISC-0974 studies in myelofibrosis (搜索) and chronic kidney disease (搜索) anemia, with data readouts expected in H2 2025.
Following a successful $259 million public offering in January, Disc Medicine reports a strong financial position with $695 million in cash, providing runway into 2028.
Disc Medicine has initiated the APOLLO confirmatory trial for bitopertin in erythropoietic protoporphyria (搜索) (EPP) and remains on track to submit a New Drug Application (NDA) in the second half of 2025, the company announced in its first quarter financial results.
The clinical-stage biopharmaceutical company, focused on developing treatments for serious hematologic diseases, has made significant progress across its pipeline programs while strengthening its financial position with a $259 million public offering completed in January.
Bitopertin Program Advances Toward NDA Submission
The company's lead program, bitopertin, a GlyT1 inhibitor that modulates heme synthesis, has reached a critical milestone with the initiation of APOLLO, a double-blind, placebo-controlled confirmatory trial in adults and adolescents with EPP. This rare genetic disorder causes severe phototoxic reactions and potentially life-threatening complications.
"We believe that bitopertin has the potential to be a life-altering therapy and thank the EPP physician, advocate, and patient communities for their ongoing enthusiasm and partnership in the development process," said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc Medicine.
The company plans to submit an NDA for bitopertin in EPP in the second half of 2025 under the FDA's accelerated approval pathway, using protoporphyrin IX (PPIX) reduction as a surrogate endpoint. This submission will be based on existing clinical data, including results from the BEACON and AURORA Phase 2 trials.
Disc Medicine has already achieved alignment with the FDA's Office of Pharmaceutical Quality on key Chemistry, Manufacturing, and Controls (CMC) components required for the NDA package. The company has scheduled a pre-NDA meeting to align with the FDA on the format and content of the submission.
Expanding Pipeline in Hematologic Diseases
Beyond bitopertin, Disc Medicine continues to advance its pipeline targeting fundamental biological pathways of red blood cell biology:
DISC-0974 Program (Anti-Hemojuvelin (搜索) Antibody):
- The company is progressing a Phase 2 study in patients with anemia of myelofibrosis (MF), with initial data expected in the second half of 2025
- A Phase 1b study in patients with anemia of non-dialysis dependent chronic kidney disease (NDD-CKD) is ongoing, with multiple-dose data expected in H2 2025
- The company will host a virtual MF Anemia KOL Day on May 9, featuring key opinion leaders to discuss DISC-0974's potential role in treating anemia in MF patients
DISC-3405 Program (Anti-TMPRSS6 (搜索) Antibody):
- On track to initiate a Phase 2 study in polycythemia vera (PV) in the first half of 2025
"Backed by a solid financial foundation which provides a cash runway into 2028, we are well-positioned to progress our commercial preparation efforts for bitopertin as well as development of the rest of our pipeline," Quisel noted.
Strong Financial Position
Disc Medicine reported a robust financial position with $694.7 million in cash, cash equivalents, and marketable securities as of March 31, 2025, which is expected to fund operational plans into 2028.
For the first quarter of 2025, the company reported:
- Research and Development expenses of $27.8 million, compared to $23.7 million for the same period in 2024
- Selling, General and Administrative expenses of $12.2 million, compared to $7.8 million for the same period in 2024
- Net loss of $34.1 million, compared to $26.9 million for the same period in 2024
The increase in expenses reflects the company's expanding clinical programs and preparations for potential commercialization, including increased headcount and infrastructure development.
Looking Forward
With multiple clinical milestones anticipated in 2025, Disc Medicine is positioned to make significant advances in addressing unmet needs in hematologic diseases. The company's focus on targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis, represents a potentially transformative approach for patients with serious blood disorders.
As the company progresses toward potential regulatory approval for bitopertin, it continues to build its pipeline of innovative therapies that could address a wide spectrum of hematologic diseases with significant unmet medical needs.