Hematology companies
23 listed hematology companies, largest first, with lead program and the next catalyst each has disclosed.
| Company | Ticker | Lead program | Indication | Next catalyst |
|---|---|---|---|---|
| Incyte Corp | INCY | JAKAFI (ruxolitinib) and portfolio of marketed products | myelofibrosis, polycythemia vera, GVHD (plus multiple other marketed indications) | Q4 2026 — Potential EU approval (MAA submitted end of 2025) |
| Haemonetics Corp | HAE | NexSys PCS plasma collection system / Plasma franchise | automated plasma collection (source plasma) | — |
| Grifols SA | GRFS | Fibrinogen concentrate (BT524) | Congenital fibrinogen deficiency (US BLA approved 2025); acquired fibrinogen deficiency (EU approved, US Phase III to start 2026) | 2026 — US commercial sales start |
| CRISPR Therapeutics AG | CRSP | CASGEVY (exagamglogene autotemcel) | sickle cell disease and transfusion-dependent beta thalassemia | — |
| Disc Medicine, Inc. | IRON | bitopertin | erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) | Q4 2026 — Initial Phase 1b data in sickle cell disease |
| Beam Therapeutics Inc. | BEAM | risto-cel (BEAM-101) | Sickle cell disease | Q4 2026 — BLA submission |
| Agios Pharmaceuticals, Inc. | AGIO | mitapivat (PYRUKYND/AQVESME) | PK deficiency hemolytic anemia; alpha/beta-thalassemia anemia (sickle cell disease sNDA under review) | H2 2026 — confirm proof of mechanism in PKU patients |
| Hemab Therapeutics Holdings, Inc. | COAG | sutacimig (HMB-001) | Glanzmann thrombasthenia (Phase 1/2) and Factor VII deficiency (Phase 2), prophylaxis | H2 2026 — commence Phase 3 trial |
| Damora Therapeutics, Inc. | DMRA | DMR-001 | Essential thrombocythemia / myelofibrosis (mutCALR) | H2 2026 — Additional regulatory submissions to expand Phase 1/1b globally |
| Silence Therapeutics plc | SLN | Divesiran (SLN124) | Polycythemia vera | 2027 — Potential IND filing |
| Omeros Corp | OMER | YARTEMLEA (narsoplimab-wuug) | hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) | 2026 — Enrollment to begin in two investigator-sponsored studies (hyperinflammatory ARDS; prophylactic pediatric TA-TMA) |
| City Therapeutics, Inc. | CTY | CITY-FXI | thromboembolic disease (secondary stroke prevention, DOAC-ineligible AFib, VTE prophylaxis, cancer-associated thrombosis) | H2 2026 — Report additional Phase 1 data |
| Rigel Pharmaceuticals Inc | RIGL | TAVALISSE (fostamatinib) and other marketed products | chronic immune thrombocytopenia (ITP); R/R AML mIDH1; RET+ NSCLC/thyroid cancer; ESR1m breast cancer | H2 2026 — Complete dose-expansion enrollment and select recommended Phase 2 dose |
| Geron Corporation | GERN | imetelstat (RYTELO) IMpactMF | intermediate-2 or high-risk relapsed/refractory myelofibrosis | H2 2026 — Interim overall survival analysis |
| Immix Biopharma, Inc. | IMMX | NXC-201 (CAR-T) | relapsed/refractory AL amyloidosis | date not stated — NEXICART-2 final readout followed by BLA submission |
| Nuvectis Pharma, Inc. | NVCT | NXP100 (ciprocopan / HSK39297) | paroxysmal nocturnal hemoglobinuria; IgA nephropathy; lupus nephritis | date not stated — Start of combination study with ALK inhibitor lorlatonib |
| Electra Therapeutics, Inc. | ETRA | ipsoprubart (formerly ELA026) | secondary hemophagocytic lymphohistiocytosis (sHLH) | H1 2027 — Phase 1 healthy-volunteer data |
| Cerus Corp | CERS | INTERCEPT Blood System (platelet, plasma, cryoprecipitation/IFC; red blood cell system in development) | Pathogen reduction in donated blood components for transfusion | H2 2026 — Preliminary results from US Phase 3 RedeS study |
| X4 Pharmaceuticals, Inc | XFOR | Mavorixafor (4WARD trial) | chronic neutropenia (congenital, acquired primary autoimmune, idiopathic) with recurrent/serious infections | H2 2027 — Top-line data |
| Rallybio Corp | RLYB | RLYB116 | complement-mediated diseases (C5 inhibitor, once-weekly subcutaneous) | 2026 — Reverse merger closing and concurrent $215.0M financing (corporate event) |
| Metagenomi Therapeutics, Inc. | MGX | MGX-001 | Hemophilia A (site-specific Factor VIII gene integration, AAV + LNP) | Q4 2026 — Metagenomi plans to submit IND for MGX-001 in hemophilia A in Q4 2026. |
| Exicure, Inc. | XCUR | GPC-100 (with propranolol) | Hematopoietic stem cell mobilization in blood cancer / multiple myeloma patients eligible for transplant | — |
| Cadrenal Therapeutics, Inc. | CVKD | CAD-1005 | heparin-induced thrombocytopenia (HIT); also cardiac surgery-associated acute kidney injury | 2029 — NDA submission, contingent on obtaining funding to run the pivotal Phase 3 |
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.