Disease

Retinitis pigmentosa and erythrocytic microcytosis

Late-stage therapeutic development

Also known as RPEM, retinitis pigmentosa and erythrocytic microcytosis; RPEM.

Clinical trials

Associated genes

Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate

Key developments

4 clinical trials expected to report results, the earliest in Q2 2029.

Upcoming Milestones

Major developments

Upcoming trial readoutHigh impact

A Phase 3, Double-Blind, Randomized Trial Evaluating the Efficacy and Safety of Elritercept (TAK-226) Compared to Placebo in Participants With Myelofibrosis and Anemia on Concurrent Ruxolitinib Therapy

Results expected Q4 20292026-10-01

Upcoming trial readoutHigh impact

A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation With Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

Results expected Q4 20292026-07-30

Clinical Milestones12View all 12

Results expected 4

Recently completed 4

Other updates 4

+4 more in the activity timeline below

Activity timeline12

2029-12-01ClinicalEnarodustat Plus CsA Versus CsA Monotherapy in the Treatment of Newly-diagnosedTD-NSAA: a Single-center Randomized TrialWithdrawn
2026-10-01ClinicalA Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Once Daily Oral Vadadustat for the Treatment of Pediatric Subjects With Anemia of Chronic Kidney Disease After Conversion From an Erythropoiesis-stimulating AgentWithdrawn
2026-10-01ClinicalA Phase 3, Double-Blind, Randomized Trial Evaluating the Efficacy and Safety of Elritercept (TAK-226) Compared to Placebo in Participants With Myelofibrosis and Anemia on Concurrent Ruxolitinib TherapyResults expected Q4 2029
2026-08-12ClinicalA Randomized, Open-Label, Active-Controlled Study Evaluating the Efficacy of Three Times Weekly Vadadustat Compared to Standard of Care ESA in Patients With Anemia of CKD Receiving In-Center HemodialysisCompleted
2026-07-30ClinicalA Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation With Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)Results expected Q4 2029
2026-07-22ClinicalA Phase 3, Multicenter, Open-Label, Randomized Trial to Compare the Efficacy and Safety of Elritercept Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in ESA-naïve Adult Participants Who Require Red Blood Cell TransfusionsResults expected Q2 2029
2026-06-30ClinicalA Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis PigmentosaPrimary completion
2026-06-15ClinicalNAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis PigmentosaResults expected Q2 2029
2026-03-31ClinicalIRONICA: IRON Repletion in Congestive Heart Failure - A Randomized Controlled Trial Comparing Oral Versus IV ApproachesTerminated
2026-01-14ClinicalPreventing Malaria in School Children to Protect the Whole Community in Rural Blantyre District, MalawiCompleted

Clinical trials

15 sponsors · 3 new · 3 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

Completed

Late-stage studies

Phase 3 · Recruiting · Takeda

A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis Pigmentosa

Phase 2/3 · Active, not recruiting · Beacon Therapeutics

NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis Pigmentosa

Phase 3 · Active, not recruiting · Johns Hopkins University

A Phase 3, Multicenter, Open-Label, Randomized Trial to Compare the Efficacy and Safety of Elritercept Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in ESA-naïve Adult Participants Who Require Red Blood Cell Transfusions

Phase 3 · Recruiting · Takeda

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

Phase 3 · Active, not recruiting · Agios Pharmaceuticals, Inc.

Phase 3 · Active, not recruiting · Boston Children's Hospital

Recruiting

Phase 3 · Recruiting · Takeda

Recently completed

A Randomized, Open-Label, Active-Controlled Study Evaluating the Efficacy of Three Times Weekly Vadadustat Compared to Standard of Care ESA in Patients With Anemia of CKD Receiving In-Center Hemodialysis

Phase 3 · Completed · Akebia Therapeutics

Optimizing Dosing Strategies in Oral Iron Supplementation

Phase 4 · Completed · Luzerner Kantonsspital

A Multicenter, Randomized, Double-blind, Parallel Controlled Clinical Study on the Efficacy and Safety of Compound E Jiao Jiang(cEJJ）in the Treatment of Postpartum Anemia

Phase 4 · Completed · DongE E Jiao Coporation Limited

Preventing Malaria in School Children to Protect the Whole Community in Rural Blantyre District, Malawi

Phase 4 · Completed · Liverpool School of Tropical Medicine

Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda: A Single Arm Open Label Trial, "BRAIN SAFE II"

Phase 3 · Completed · Global Health Uganda LTD

The Effect of Preoperative Ferric Carboxymaltose Administration on Mortality in Geriatric Hip Fractures

Phase 4 · Completed · Istanbul University

Associated genes

5 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

TRNT10.76 CRBN0.51 RUSC10.04 CD300LD0.04 NTAQ10.03

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Protein cereblonvia CRBN0.51

Reference

Authoritative identity, definition & identifiers.

Synonyms

RPEM, retinitis pigmentosa and erythrocytic microcytosis; RPEM

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
Related entities are derived from literature co-mention (studied together) — associative, not causal.