VivoSeq
Metabolic & Endocrine

Hypophosphatasia

11

Pipeline Programs↗

3

Active Trials↗

9

Companies Involved↗

Hypophosphatasia is an emerging landscape with 11 programs across 9 companies. The most active players are Ultragenyx, AM-Pharma, Alesta Therapeutics. 5 therapies are approved with 1 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

39/100

Pipeline Velocity

11/100

Mech. Concentration

50/100

Completion Rate

82/100

Attrition Rate

14/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

Ultragenyx logo

Ultragenyx

1 program

Phase 2A

AM-Pharma

1 program

Phase 2A

Alesta Therapeutics

1 program

Phase 2AstraZeneca logo

AstraZeneca

3 programs

Phase 3Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

1 program

ApprovedTakeda logo

Takeda

1 program

Approved

Pipeline

Programs by Phase

Analysis

Modality Breakdown

70%

Small molecule

7 of 10 classified programs — the dominant modality here.

30%ALL OTHERSProtein / enzyme220%Monoclonal antibody110%

Small molecule leads; the ring shows all 10 classified programs, each share of the total. Modality classified for 93% of 14 pipeline programs.

Analysis

Trial Status

22

clinical trials

Completed14 · 64%
Withdrawn3 · 14%
Recruiting2 · 9%
Approved for marketing1 · 5%
Unknown status1 · 5%
Active, not recruiting1 · 5%

Clinical Trials

Trial Activity

22 trials

A Prospective Sub-Study of the Global Hypophosphatasia Registry
Recruiting·AstraZeneca logoAstraZeneca·N/A·NCT05234567·30 enrolled
A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1
Recruiting·AAlesta Therapeutics·Phase 1/2·NCT07179640·120 enrolled
A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 (Recombinant Alkaline Phosphatase) Administered Subcutaneously in Adolescent (12 to < 18 Years of Age) and Adult Participants With Hypophosphatasia Who Have Not Previously Been Treated With Asfotase Alfa
Active, not recruiting·AstraZeneca logoAstraZeneca·Phase 3·NCT06079281·124 enrolled
Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT02797821·27 enrolled
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
Completed·AstraZeneca logoAstraZeneca·Phase 2/3·NCT01176266·69 enrolled
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT01205152·10 enrolled
Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)
Completed·TTranslational Research Center for Medical Innovation, Kobe, Hyogo, Japan·Phase 2·NCT02456038·13 enrolled
Study of ALXN1850 in Participants With Hypophosphatasia (HPP)
Completed·AstraZeneca logoAstraZeneca·Phase 1·NCT04980248·15 enrolled
Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
Completed·AstraZeneca logoAstraZeneca·N/A·NCT04195763·50 enrolled
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT01163149·19 enrolled
Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)
Completed·Ultragenyx logoUltragenyx·Phase 2·NCT01406977·8 enrolled
Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT00952484·13 enrolled
Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan
Completed·AstraZeneca logoAstraZeneca·Phase 4·NCT02531867·13 enrolled
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT01203826·12 enrolled
Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)
Completed·AstraZeneca logoAstraZeneca·Phase 1·NCT00739505·6 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)