Hematology Video Recap—March 9, 2026

LucidQuest

20 minutes ago

This biweekly Hematology video recap highlights regulatory approvals, priority review milestones, clinical trial progress, financing partnerships, and regional expansion across blood disorders and rare hematologic diseases.

🎯 Watch Our Video Summary Capturing Hematology News from the Last Two Weeks

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Week 23 February–1 March 2026
Week 2 –8 March 2026

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Transcript

Welcome to the latest edition of Hematology Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.

Fulcrum Therapeutics reported 12-week results from the 20 milligram cohort of the Phase 1b PIONEER study evaluating pociredir in severe sickle cell disease. In the open-label cohort of twelve evaluable patients, the therapy increased mean fetal hemoglobin from 7.1 percent to 19.3 percent and raised the proportion of F-cells. Investigators also observed improvements in hemolysis markers and an increase in total hemoglobin of 1.1 grams per deciliter. No treatment-related serious adverse events were reported in the cohort.

Bristol Myers Squibb announced positive top-line results from a registrational Phase 2 study of Reblozyl, or luspatercept, in adults with alpha-thalassemia. The study includes both non-transfusion-dependent and transfusion-dependent patient cohorts. The therapy met the primary endpoint in each group, demonstrating increased hemoglobin levels in non-transfusion-dependent disease and reductions in transfusion burden among transfusion-dependent patients. Key secondary endpoints were also achieved.

Blackstone Life Sciences and Johnson and Johnson announced a co-funding agreement to support the development of bleximenib, an investigational oral menin inhibitor for acute myeloid leukemia. The collaboration will provide funding for both ongoing and future clinical trials as part of the broader development program. The agreement signals continued investment in targeted therapies designed to address genetically defined leukemia populations.

Oryzon Genomics received authorization from the European Medicines Agency to begin a Phase II clinical study of iadademstat in essential thrombocythemia. The IDEAL study will enroll adults who are resistant or intolerant to hydroxyurea and will be conducted in Spain. The trial will evaluate the LSD1 inhibitor over a treatment period of up to 24 weeks with the option for an extension phase.

Johnson and Johnson received United States FDA approval for the combination of TECVAYLI, also known as teclistamab, with DARZALEX FASPRO, or daratumumab, for adults with relapsed or refractory multiple myeloma who have received at least one prior line of therapy including a proteasome inhibitor and an immunomodulatory agent. The approval was supported by results from the Phase 3 MajesTEC-3 study, which demonstrated improved progression-free survival and overall survival compared with standard regimens. Three-year overall survival reached 83.3 percent in the teclistamab plus daratumumab arm.

Takeda and Protagonist Therapeutics announced that the United States FDA has accepted the New Drug Application for rusfertide in polycythemia vera and granted Priority Review. The filing is supported primarily by the Phase 3 VERIFY study, along with longer-term data from the Phase 2 REVIVE and THRIVE studies. According to the companies, the VERIFY trial met its primary endpoint and all four key secondary endpoints.

Agios announced that PYRUKYND, or mitapivat, has been approved in the United Arab Emirates for adults with alpha- or beta-thalassemia. The approval covers both non-transfusion-dependent and transfusion-dependent disease and is supported by results from the Phase 3 ENERGIZE and ENERGIZE-T studies. The company stated that mitapivat is currently the only approved therapy for this patient population in the UAE.

Hemab Therapeutics received Breakthrough Therapy Designation from the United States FDA for sutacimig for the prevention of bleeding episodes in patients with Glanzmann thrombasthenia. The designation is based on a Phase 1 and Phase 2 clinical data package. In the completed multiple-ascending-dose portion of the Phase 2 study, the company reported clinically meaningful reductions in bleeding events in this rare platelet disorder.

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Why it matters

Hemoglobinopathies were a major theme this week, with new clinical or regulatory milestones for sickle cell disease, alpha-thalassemia, polycythemia vera, and broader thalassemia care [1][2][6][7].
Multiple myeloma treatment moved earlier in line, with Johnson & Johnson adding a bispecific-based combination after just one prior therapy [5].
Capital continues to flow into targeted hematology development, shown by Blackstone Life Sciences joining Johnson & Johnson on bleximenib in AML [3].
European and Gulf regulators featured prominently, with EMA trial clearance for iadademstat and UAE approval for mitapivat [4][7].
Rare bleeding disorders also advanced, with Hemab gaining Breakthrough Therapy Designation for a potential prophylactic option in Glanzmann thrombasthenia [8].

🗓️ Explore weekly details and sources

Week 23 February–1 March 2026
Week 2 –8 March 2026

📚 See the full Hematology archive on our research hub page.

FAQ

What did Fulcrum report for pociredir in sickle cell disease?

Fulcrum said the 20 mg Phase 1b PIONEER cohort showed higher fetal hemoglobin, improved hemolysis markers, and higher total hemoglobin at 12 weeks, with no treatment-related serious adverse events reported in the cohort summary [1].

What is new for Reblozyl in alpha-thalassemia?

Bristol Myers Squibb reported positive top-line results from a registrational Phase 2 study, with both non-transfusion-dependent and transfusion-dependent cohorts meeting primary and secondary endpoints. Detailed congress data are still pending [2].

What does the Blackstone–Johnson & Johnson agreement mean for bleximenib?

It adds external development funding to support ongoing and future AML studies of bleximenib, an investigational oral menin inhibitor. The release does not disclose financial terms [3].

What changed for iadademstat in essential thrombocythemia?

Oryzon said EMA authorized the CTA for the IDEAL Phase II study in Spain, allowing the company to begin testing iadademstat in adults with ET resistant or intolerant to hydroxyurea [4].

Why is the TECVAYLI plus DARZALEX FASPRO approval important?

It moves a BCMA-directed bispecific regimen into earlier relapsed/refractory multiple myeloma, after at least one prior line. Johnson & Johnson said approval was based on significant progression-free and overall survival gains in MajesTEC-3 [5].

What does rusfertide’s Priority Review mean for polycythemia vera?

Takeda and Protagonist now have an FDA review underway, with a Q3 2026 PDUFA target. The filing is based primarily on Phase 3 VERIFY plus longer-term Phase 2 data [6].

Entities / Keywords

Fulcrum Therapeutics, pociredir, PIONEER, sickle cell disease, SCD, fetal hemoglobin, HbF, Bristol Myers Squibb, Reblozyl, luspatercept, alpha-thalassemia, Blackstone Life Sciences, Johnson & Johnson, bleximenib, JNJ-75276617, menin inhibitor, acute myeloid leukemia, AML, Oryzon Genomics, iadademstat, LSD1 inhibitor, essential thrombocythemia, ET, IDEAL, TECVAYLI, teclistamab, DARZALEX FASPRO, daratumumab, relapsed/refractory multiple myeloma, RRMM, MajesTEC-3, Takeda, Protagonist Therapeutics, rusfertide, polycythemia vera, PV, VERIFY, REVIVE, THRIVE, Agios, PYRUKYND, mitapivat, UAE, ENERGIZE, ENERGIZE-T, Hemab Therapeutics, sutacimig, Glanzmann thrombasthenia, GT, Breakthrough Therapy Designation.