Hypophosphatasia

Pipeline Programs

Companies

Clinical Trials

2 recruiting

Approved Products

Pipeline by Development Stage

Preclinical

Phase 1

Phase 1/2

Phase 2

Phase 2/3

Phase 3

On Market

Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

STRENSIQApproved

asfotase alfa

AstraZeneca

Tissue-nonspecific Alkaline Phosphatase [EPC]injection2015

259M Part D

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Pre

P1/2

P2/3

Mkt

AstraZenecaCAMBRIDGE, United Kingdom

5 programs

STRENSIQ(asfotase alfa)APPROVED_FOR_MARKETING5 trials

ALXN1850Phase 31 trial

ENB-0040Phase 21 trial

ALXN1850Phase 11 trial

Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)N/A1 trial

Active Trials

NCT02496689Approved For Marketing

NCT06015750Withdrawn0Est. Mar 2030

NCT05234567Recruiting30Est. Jul 2028

+6 more trials

Innovation PharmaceuticalsMA - Wakefield

1 program

Asfotase AlfaPhase 21 trial

Active Trials

NCT02456038Completed13Est. Jul 2015

SandozAustria - Kundl

1 program

BPS804Phase 21 trial

Active Trials

NCT01406977Completed8Est. Sep 2012

AM-PharmaNetherlands - Utrecht

1 program

ilofotase alfaPhase 21 trial

Active Trials

NCT05890794Completed12Est. Jul 2023

Alesta TherapeuticsNetherlands - Leiden

1 program

ALE1Phase 1/21 trial

Active Trials

NCT07179640Recruiting120Est. Jan 2027

CENTOGENEGermany - Rostock

1 program

Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)N/A

NovartisBASEL, Switzerland

1 program

BPS804PHASE_2

Ultragenyx PharmaceuticalNOVATO, CA

1 program

BPS804PHASE_2

Mereo BioPharmaUK - London

1 program

BPS804PHASE_2

Trial Timeline

Clinical trial activity over time

2021

2022

2023

2024

2025

2026

2027

2028

2029

2030

AstraZenecaasfotase alfa

AstraZenecaALXN1850

AstraZenecaasfotase alfa

Innovation PharmaceuticalsAsfotase Alfa

SandozBPS804

AstraZenecaasfotase alfa

AstraZenecaENB-0040

AstraZenecaasfotase alfa

Alesta TherapeuticsALE1

AM-Pharmailofotase alfa

Showing 15 of 20 trials with date data

Clinical Trials (21)

Total enrollment: 441 patients across 21 trials

NCT06015750 AstraZenecaasfotase alfa

Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia

Start: Jul 2026Est. completion: Mar 20300

Phase 4Withdrawn

NCT04189315 AstraZenecaasfotase alfa

Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease

Start: Apr 2020Est. completion: Aug 20220

Phase 4Withdrawn

NCT02531867 AstraZenecaasfotase alfa

Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan

Start: Jun 2015Est. completion: Nov 201513 patients

Phase 4Completed

NCT06079359 AstraZenecaALXN1850

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

Start: May 2024Est. completion: Aug 202830 patients

Phase 3Active Not Recruiting

NCT01176266 AstraZenecaasfotase alfa

Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)

Start: Jul 2010Est. completion: Sep 201669 patients

Phase 2/3Completed

NCT02797821 AstraZenecaasfotase alfa

Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)

Start: Jun 2016Est. completion: Jun 201727 patients

Phase 2Completed

NCT02456038 Innovation PharmaceuticalsAsfotase Alfa

Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)

Start: Aug 2014Est. completion: Jul 201513 patients

Phase 2Completed

NCT01406977 SandozBPS804

Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)

Start: Jul 2011Est. completion: Sep 20128 patients

Phase 2Completed

NCT01163149 AstraZenecaasfotase alfa

Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)

Start: Jun 2010Est. completion: Jun 201619 patients

Phase 2Completed

NCT01203826 AstraZenecaasfotase alfa

Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)

Start: Apr 2010Est. completion: Jun 201612 patients

Phase 2Completed

NCT00952484 AstraZenecaasfotase alfa

Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)

Start: Sep 2009Est. completion: Jul 201013 patients

Phase 2Completed

NCT00894075 AstraZenecaENB-0040

Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)

Start: Jul 2009Est. completion: Dec 20140

Phase 2Withdrawn

NCT01205152 AstraZenecaasfotase alfa

Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

Start: Apr 2009Est. completion: Aug 201610 patients

Phase 2Completed

NCT07179640 Alesta TherapeuticsALE1

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

Start: Sep 2025Est. completion: Jan 2027120 patients

Phase 1/2Recruiting

NCT05890794 AM-Pharmailofotase alfa

Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia

Start: May 2023Est. completion: Jul 202312 patients

Phase 1/2Completed

NCT00744042 AstraZenecaasfotase alfa

Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)

Start: Sep 2008Est. completion: May 201011 patients

Phase 1/2Completed

NCT04980248 AstraZenecaALXN1850

Study of ALXN1850 in Participants With Hypophosphatasia (HPP)

Start: Sep 2021Est. completion: Aug 202215 patients

Phase 1Completed

NCT02496689 AstraZenecaasfotase alfa

Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)

N/AApproved For Marketing

NCT05234567 AstraZenecaasfotase alfa

A Prospective Sub-Study of the Global Hypophosphatasia Registry

Start: Aug 2022Est. completion: Jul 202830 patients

N/ARecruiting

NCT04925804 AstraZenecaUnraveling Genetics of HypoPhosPhatasia (HPP Genetics)

Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)

Start: Jun 2021Est. completion: Dec 202116 patients

N/ACompleted

NCT03418389 AstraZenecaasfotase alfa

Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia

Start: Sep 2018Est. completion: May 202123 patients

N/ACompleted

Phase Legend

Preclinical— Lab & animal studies

Phase 1— Safety & dosing

Phase 2— Efficacy testing

Phase 3— Large-scale trials

On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals

2 actively recruiting trials targeting 441 patients

9 companies competing in this space

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