NCT01235936
- Objective
- Not yet disclosed
- Design
- Not yet disclosed
- Participants
- Not yet disclosed
- Primary endpoint
- Not yet disclosed
- Results
- Results not yet reported
pharma · Anemia · Anemia Associated With Chronic Kidney Disease · AKBA
Akebia Europe
Akebia Europe Limited
Akebia Europe is a pharma organization headquartered in Cambridge, USA. It trades on NYSE under ticker AKBA. Primary therapeutic focus areas include Anemia, Anemia Associated With Chronic Kidney Disease, Anemia of Chroni
Cambridge, USA HQ
2007 Founded
271 Employees
Public company Type
AKBA · NYSE Ticker
Company details
- Status
- Public
- HQ
- Cambridge, USA
- Founded
- 2007
- Employees
- 271
- Programs
- 25
- Drugs
- 13
- Patents
- 28
AKB-6548
Phase 2 · small molecule · Anemia
AKB-6548 is a small-molecule therapeutic candidate developed by Akebia Europe Limited for the treatment of anemia. The program is currently in Phase 2 development with a completed status as of July 2022. The drug's specific mechanism of action and molecular target have not been publicly disclosed. Akebia's development
Internal code AKB-6548-CI-0011
At a glance
- Sponsor
- Akebia Europe Limited
- Phase
- Phase 2
- Modality
- small_molecule
- Indication
- Anemia
- Status
- completed
- Trials
- 4
Executive summary
AKB-6548 is a small-molecule therapeutic candidate developed by Akebia Europe Limited for the treatment of anemia. The program is currently in Phase 2 development with a completed status as of July 2022. The drug's specific mechanism of action and molecular target have not been publicly disclosed. Akebia's development strategy for AKB-6548 involves a clinical trial portfolio spanning multiple Phase 2 studies, as evidenced by four registered clinical trials (NCT01235936, NCT01381094, NCT01906489, NCT02260193). The most recent milestone was recorded on July 21, 2022, though the nature of this milestone has not been disclosed. The program operates without disclosed partnership arrangements or licensing agreements. Regulatory approval status, peak sales projections, and consensus analyst positioning remain undisclosed. The competitive landscape for anemia treatment includes multiple approved therapies targeting iron supplementation, erythropoiesis-stimulating agents, and emerging Phase 3 candidates with novel mechanisms.
Why this program matters
Anemia remains a significant clinical and commercial indication with substantial unmet medical need across multiple patient populations, including those with chronic kidney disease, cancer-related anemia, and hereditary anemias. The anemia therapeutics market encompasses multiple mechanistic approaches, reflecting the heterogeneous nature of anemia etiology and treatment response. AKB-6548's development occurs within a competitive landscape that includes well-established approved therapies such as epoetin alfa, epoetin beta, and methoxy polyethylene glycol-epoetin beta, as well as emerging candidates in Phase 3 development including Reblozyl and Mitapivat. The commercial significance of the anemia market is underscored by the presence of multiple pharmaceutical sponsors including Hoffmann-La Roche, Takeda, United Therapeutics Europe Ltd, and Celgene Europe Limited. Patient populations affected by anemia span chronic disease states, acute conditions, and rare genetic disorders, creating diverse commercial opportunities. The Phase 2 completion status of AKB-6548 suggests the program has generated sufficient preliminary efficacy and safety data to inform subsequent development decisions, though the specific clinical outcomes and competitive positioning relative to approved and investigational comparators remain undisclosed.
Drug intelligence
Drug Class: Small-molecule therapeutic for anemia treatment
Modality: Small molecule
Mechanism of Action: Not yet disclosed
Molecular Target: Not yet disclosed
Route of Administration: Not yet disclosed
Related Therapies: The anemia treatment landscape includes erythropoiesis-stimulating agents (epoetin alfa, epoetin beta, methoxy polyethylene glycol-epoetin beta, darbepoetin alfa), iron supplementation agents (ferric carboxymaltose, ferumoxytol, iron and zinc combinations), and emerging mechanistic classes represented by Phase 3 candidates
First Approval: Not yet disclosed
Patent Status: Not yet disclosed
anemia
Also known as: anaemia (disease), anemia (disease)
Overview
A reduction in the number of red blood cells, the amount of hemoglobin, and/or the volume of packed red blood cells. Clinically, anemia represents a reduction in the oxygen-transporting capacity of a designated volume of blood, resulting from an imbalance between blood loss (through hemorrhage or hemolysis) and blood production. Signs and symptoms of anemia may include pallor of the skin and mucous membranes, shortness of breath, palpitations of the heart, soft systolic murmurs, lethargy, and fatigability.
Treatment landscape
ClinicalTrials.gov lists 98 registered studies for Anaemia, (AACT aggregate).
Phase breakdown: NA (35), PHASE3 (21), PHASE1 (18), PHASE2 (12), PHASE4 (11), PHASE2/PHASE3 (1)
Common investigational therapies:
- GSK1278863
- Daprodustat
- Placebo
- rhEPO
- GSK1278863A
- Darbepoetin alfa
- Iron therapy
- Daprodustat (GSK1278863)
- Ferinject ® (Ferric carboxymaltose)
- Normal saline (0.9%)
Disease data sourced from MONDO Disease Ontology (MONDO:0002280), NCT00466297, NCT00767702, NCT01043133, NCT01317979, NCT01477281, AACT (ClinicalTrials.gov aggregate), ClinicalTrials.gov, NCT00140517, NCT00238043, NCT00258024, NCT00259142, NCT00276224, Open Targets Platform (CC BY 4.0).
Clinical development timeline
- Phase 2TBD
Phase 2 studies initiated
Four Phase 2 clinical trials registered (NCT01235936, NCT01381094, NCT01906489, NCT02260193).
- Phase 22022-07-21
Phase 2 program completed
Latest milestone recorded; specific outcomes not yet disclosed.
Competitive landscape
The anemia therapeutics market includes multiple established approved therapies and emerging Phase 3 candidates. Approved erythropoiesis-stimulating agents include epoetin alfa and epoetin beta (United Therapeutics Europe Ltd and Hoffmann-La Roche), methoxy polyethylene glycol-epoetin beta/Mircera (Hoffmann-La Roche), and darbepoetin alfa/Dynepo (Takeda). Iron supplementation therapies include ferric carboxymaltose and ferumoxytol (United Therapeutics Europe Ltd and Lacuna Pharma Pty Ltd), and combination iron and zinc formulations. Emerging Phase 3 candidates include Reblozyl (Celgene Europe Limited), Mitapivat (Lacuna Pharma Pty Ltd), and additional iron formulations in Phase 3 development. The competitive positioning of AKB-6548 relative to these approved and investigational therapies cannot be determined from disclosed information, as the drug's mechanism of action, efficacy profile, and safety characteristics remain undisclosed. The diversity of mechanisms represented in the competitive set—including erythropoiesis stimulation, iron metabolism modulation, and emerging novel targets—suggests multiple pathways to anemia treatment, potentially creating differentiated opportunities for AKB-6548 depending on its undisclosed mechanism and clinical profile.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| Rabbit ATG, (Genzyme) | Xiyuan Hospital of China Academy of Chinese Medical Sciences | small_molecule | approved |
| Ferric carboxymaltose | United Therapeutics Europe Ltd | small_molecule | approved |
| Epoetin Alfa | United Therapeutics Europe Ltd | small_molecule | approved |
| Ferinject 50 mg/ml dispersión inyectable y para perfusión | The George Institute | small_molecule | approved |
| Dynepo | Takeda | small_molecule | approved |
| methoxy polyethylene glycol-epoetin beta [Mircera] | Hoffmann-La Roche | small_molecule | approved |
| iron and zinc combined | United Therapeutics Europe Ltd | other | approved |
| epoetin beta [NeoRecormon] | Hoffmann-La Roche | small_molecule | approved |
| 0.9 % w/v Sodium Chloride Injection, Reblozyl 25 mg powder for solution for injection, Reblozyl 75 mg powder for solution for injection | Celgene Europe Limited | small_molecule | phase_3 |
| MITAPIVAT, MITAPIVAT, "Placebo to Match Mitapivat Tablets, 5 mg and 20 mg, are supplied as film-coated, blue, round tablets for oral administration Placebo to Match Mitapivat Tablets, 50 mg or 100 mg, are supplied as film-coated, blue, oblong tablets for oral administration. Placebo to Match Mitapivat Granules, 1 mg, are supplied as film-coated, white, round granules for oral administration", MITAPIVAT | Lacuna Pharma Pty Ltd | small_molecule | phase_3 |
| Venofer 20 mg iron / ml, solution for injection or concentrate for solution for infusion., Ferumoxytol | Lacuna Pharma Pty Ltd | small_molecule | phase_3 |
| VOXELOTOR | — | Hemoglobin HbA positive modulator | Approved |
| TRIAMCINOLONE ACETONIDE | — | Glucocorticoid receptor agonist | Approved |
| SUTIMLIMAB | — | Complement C1s inhibitor | Approved |
| RUXOLITINIB PHOSPHATE | — | Tyrosine-protein kinase JAK2 inhibitor | Approved |
| RAVULIZUMAB | — | Complement C5 inhibitor | Approved |
| PREDNISONE | — | Glucocorticoid receptor agonist | Approved |
| PREDNISOLONE SODIUM PHOSPHATE | — | Glucocorticoid receptor agonist | Approved |
| PREDNISOLONE ACETATE | — | Glucocorticoid receptor agonist | Approved |
| PREDNISOLONE | — | Glucocorticoid receptor agonist | Approved |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
Regulatory intelligence
FDA Status: Not yet disclosed
EMA Status: Not yet disclosed
PMDA (Japan) Status: Not yet disclosed
NMPA (China) Status: Not yet disclosed
Approval History: Not yet disclosed
AKB-6548 remains in Phase 2 development with no disclosed regulatory submissions, approvals, or interactions with major regulatory authorities. The program's regulatory pathway, anticipated submission timeline, and regulatory strategy have not been publicly disclosed.
Clinical evidence summary
NCT01381094
- Objective
- Not yet disclosed
- Design
- Not yet disclosed
- Participants
- Not yet disclosed
- Primary endpoint
- Not yet disclosed
- Results
- Results not yet reported
NCT01906489
- Objective
- Not yet disclosed
- Design
- Not yet disclosed
- Participants
- Not yet disclosed
- Primary endpoint
- Not yet disclosed
- Results
- Results not yet reported
NCT02260193
- Objective
- Not yet disclosed
- Design
- Not yet disclosed
- Participants
- Not yet disclosed
- Primary endpoint
- Not yet disclosed
- Results
- Results not yet reported
Key questions answered
What is AKB-6548 used for?
AKB-6548 is a small-molecule therapeutic candidate in development for the treatment of anemia. Its specific mechanism of action and target patient population have not been publicly disclosed.
Who manufactures AKB-6548?
AKB-6548 is developed by Akebia Europe Limited, a subsidiary or affiliate of Akebia Therapeutics. No manufacturing partners have been disclosed.
What is the current development status of AKB-6548?
AKB-6548 is in Phase 2 development with a completed status as of July 21, 2022. The specific nature of this completion and next development steps have not been disclosed.
How does AKB-6548 work?
The mechanism of action of AKB-6548 has not been publicly disclosed. The drug's molecular target and therapeutic pathway remain undisclosed.
Is AKB-6548 approved by the FDA?
No, AKB-6548 is not approved by the FDA. The drug remains in Phase 2 clinical development and has not submitted a regulatory application.
What clinical trials support AKB-6548?
Four Phase 2 clinical trials have been registered for AKB-6548: NCT01235936, NCT01381094, NCT01906489, and NCT02260193. Detailed trial results and outcomes have not been publicly disclosed.
Does AKB-6548 have any partnerships or licensing agreements?
No partnerships or licensing agreements have been disclosed for AKB-6548. The program appears to be developed independently by Akebia Europe Limited.
What is the route of administration for AKB-6548?
The route of administration for AKB-6548 has not been disclosed. This information may be revealed upon publication of clinical trial data or regulatory submissions.
What are the main competitors to AKB-6548?
Approved competitors in the anemia market include epoetin alfa, epoetin beta, methoxy polyethylene glycol-epoetin beta, and ferric carboxymaltose. Phase 3 competitors include Reblozyl and Mitapivat.
When was AKB-6548 first disclosed?
The initial disclosure date for AKB-6548 has not been recorded in available sources. The program's first public appearance may have occurred through clinical trial registration.
What is the projected peak sales potential for AKB-6548?
Peak sales projections for AKB-6548 have not been disclosed by the sponsor or analyst consensus. Commercial potential cannot be assessed without efficacy and safety data.
What is the molecular target of AKB-6548?
The specific molecular target of AKB-6548 has not been publicly disclosed. This information would typically be revealed through scientific publications or regulatory submissions.
Has AKB-6548 advanced to Phase 3 development?
As of the latest disclosed milestone (July 2022), AKB-6548 remains in Phase 2 development. No Phase 3 initiation has been announced.
What patient populations are being studied in AKB-6548 trials?
The specific patient populations enrolled in AKB-6548 trials have not been disclosed. Anemia affects multiple populations including those with chronic kidney disease, cancer-related anemia, and hereditary anemias.
What are the primary endpoints in AKB-6548 clinical trials?
The primary endpoints for the four Phase 2 trials have not been publicly disclosed. Typical anemia trial endpoints may include hemoglobin levels, transfusion requirements, or quality of life measures.
Is there a lead investigator or research institution for AKB-6548?
The lead investigator and primary research institutions for AKB-6548 have not been disclosed in available sources.
Entity relationship graph
AKB-6548 → Drug → Target → Indication → Company → Trials → Competitors
Companies
Indication
Competitors
- Rabbit ATG, (Genzyme)
- Ferric carboxymaltose
- Epoetin Alfa
- Ferinject 50 mg/ml dispersión inyectable y para perfusión
- Dynepo
- methoxy polyethylene glycol-epoetin beta [Mircera]
- iron and zinc combined
- epoetin beta [NeoRecormon]
- 0.9 % w/v Sodium Chloride Injection, Reblozyl 25 mg powder for solution for injection, Reblozyl 75 mg powder for solution for injection
- MITAPIVAT, MITAPIVAT, "Placebo to Match Mitapivat Tablets, 5 mg and 20 mg, are supplied as film-coated, blue, round tablets for oral administration Placebo to Match Mitapivat Tablets, 50 mg or 100 mg, are supplied as film-coated, blue, oblong tablets for oral administration. Placebo to Match Mitapivat Granules, 1 mg, are supplied as film-coated, white, round granules for oral administration", MITAPIVAT
- Venofer 20 mg iron / ml, solution for injection or concentrate for solution for infusion., Ferumoxytol
- VOXELOTOR
- TRIAMCINOLONE ACETONIDE
- SUTIMLIMAB
- RUXOLITINIB PHOSPHATE
- RAVULIZUMAB
- PREDNISONE
- PREDNISOLONE SODIUM PHOSPHATE
- PREDNISOLONE ACETATE
- PREDNISOLONE
Analyst insights
Development Status: AKB-6548 completed Phase 2 development as of July 2022. The lack of disclosed milestone details or subsequent Phase 3 announcements suggests either continued evaluation of Phase 2 data, strategic reassessment, or potential program deprioritization. The absence of disclosed next-milestone expectations indicates uncertainty regarding advancement timeline.
Competitive Positioning: Without disclosed mechanism of action or efficacy/safety data, competitive positioning cannot be assessed. The anemia market's maturity and presence of multiple approved therapies creates a high bar for differentiation. Emerging Phase 3 candidates (Reblozyl, Mitapivat) represent potential competitive threats if they achieve approval with novel mechanisms or superior efficacy-safety profiles.
Strategic Implications: Akebia's development of AKB-6548 occurs within a crowded therapeutic space. The program's future depends on demonstration of clinical advantages over established therapies or identification of specific patient populations with unmet needs. The lack of partnership disclosure suggests Akebia is pursuing independent development, which may limit resources for late-stage development and commercialization.
Future Catalysts: Potential catalysts include Phase 2 data disclosure, Phase 3 initiation announcement, regulatory feedback, or strategic partnership announcement. The extended interval since the last disclosed milestone (July 2022) without announced progression suggests the program may face development challenges or strategic reconsideration.
Quick answers
Concise, citable answers optimized for AI answer engines.
- What is AKB-6548?
- Small-molecule therapeutic candidate for anemia in Phase 2 development by Akebia Europe Limited.
- Is AKB-6548 approved?
- No, AKB-6548 is not approved. It remains in Phase 2 clinical development.
- What indication is AKB-6548 being developed for?
- Anemia treatment.
- Who develops AKB-6548?
- Akebia Europe Limited.
- What is the mechanism of action of AKB-6548?
- Not yet disclosed.
- What is the molecular target of AKB-6548?
- Not yet disclosed.
- What is the route of administration for AKB-6548?
- Not yet disclosed.
- What is the current development phase of AKB-6548?
- Phase 2 (completed as of July 2022).
- How many clinical trials are registered for AKB-6548?
- Four Phase 2 trials: NCT01235936, NCT01381094, NCT01906489, NCT02260193.
- Does AKB-6548 have any development partners?
- No partners have been disclosed.
- What is the projected peak sales for AKB-6548?
- Not yet disclosed.
- What are the main competitors to AKB-6548?
- Epoetin alfa, epoetin beta, methoxy polyethylene glycol-epoetin beta, ferric carboxymaltose, Reblozyl, Mitapivat.
- When was AKB-6548 last updated?
- July 21, 2022 (Phase 2 completion milestone).
- Is AKB-6548 a small molecule or biologic?
- Small molecule.
- Has AKB-6548 published clinical trial results?
- Results have not been publicly reported or disclosed.
- What is the internal code for AKB-6548?
- AKB-6548-CI-0011.
- Is AKB-6548 approved in Europe?
- No, regulatory approval status in Europe has not been disclosed.
- Is AKB-6548 approved in Japan?
- No, regulatory approval status in Japan has not been disclosed.
- Is AKB-6548 approved in China?
- No, regulatory approval status in China has not been disclosed.
- What unmet medical need does AKB-6548 address?
- Anemia treatment with potential advantages over existing approved therapies; specific unmet need not disclosed.
- When is the next expected milestone for AKB-6548?
- Not yet disclosed.
- What is the consensus analyst position on AKB-6548?
- Not yet disclosed.
Evidence & sources
- ClinicalTrials.gov NCT01235936 (clinicaltrials)
- ClinicalTrials.gov NCT01381094 (clinicaltrials)
- ClinicalTrials.gov NCT01906489 (clinicaltrials)
- ClinicalTrials.gov NCT02260193 (clinicaltrials)
- Source: phase (source_attribution)
- MONDO Disease Ontology (MONDO:0002280) (mondo)
- NCT00466297 (clinicaltrials_gov)
- NCT00767702 (clinicaltrials_gov)
- NCT01043133 (clinicaltrials_gov)
- NCT01317979 (clinicaltrials_gov)
- NCT01477281 (clinicaltrials_gov)
- AACT (ClinicalTrials.gov aggregate) (aact)
- ClinicalTrials.gov (clinicaltrials_gov)
- NCT00140517 (clinicaltrials_gov)
- NCT00238043 (clinicaltrials_gov)
- NCT00258024 (clinicaltrials_gov)
- NCT00259142 (clinicaltrials_gov)
- NCT00276224 (clinicaltrials_gov)
- Open Targets Platform (opentargets)
Intelligence compiled from public regulatory and clinical sources (FDA, EMA, ClinicalTrials.gov and company disclosures). Figures may be editorial or analyst estimates; verify against primary sources before relying on them.