ALE1
An investigational treatment for Hypophosphatasia.
The same compound appears under different names depending on the context. Here is how to identify ALE1 wherever you encounter it, plus the key facts at a glance.
- Generic name
- ALE1
- Development code
- ALE1
- Drug class
- Oral small molecule (pyrophosphate-lowering)
- Manufacturer
- BioMarin Pharmaceutical (acquired from Alesta Therapeutics, September 2026)
- How it's taken
- Oral (investigational; dose and schedule not yet set)
An investigational pill for hypophosphatasia that aims to lower the pyrophosphate build-up behind the disease without injections. BioMarin bought it from Alesta Therapeutics in 2026; a Phase 1/2a safety study in healthy volunteers and adults with HPP is recruiting.
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Where ALE1 fits
A possible future third option after Strensiq and efzimfotase alfa, and the only one that would be a pill. It is at the earliest clinical stage, with no efficacy data, so it is not a treatment choice today; its relevance now is as a trial for adults with a confirmed ALPL variant who have never had enzyme replacement.
How ALE1 works
Every treatment for HPP so far has replaced the missing alkaline phosphatase enzyme by injection. ALE1 takes a different route. BioMarin says it is designed to inhibit a target that regulates inorganic pyrophosphate (PPi), the substance that accumulates when the enzyme is missing and stops calcium and phosphate from hardening bone; by lowering PPi from another direction it aims to restore healthier bone and mineral metabolism across the full spectrum of HPP[1].
The company has not named the target. If it works, it would be the first oral therapy for HPP and the first that does not depend on an enzyme infusion or injection[1].
Mechanism: Oral small molecule designed to inhibit an undisclosed target that regulates inorganic pyrophosphate (PPi) levels, lowering the excess PPi that blocks bone mineralization in HPP
Side effects and safety
No safety results have been released. The first-in-human study measures adverse events, blood counts, blood chemistry, heart rhythm (ECG) and vital signs over the first 16 days after dosing in each group[3]. Until data are presented, nothing can be said about what side effects to expect.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking ALE1
An oral pill. The Phase 1/2a study tests single and multiple ascending doses, with and without food, so no dose or schedule has been chosen[3].
Availability and cost
Years from pricing. The appeal to BioMarin, in its own words, is a potential oral alternative to the injectable therapies available today and a larger rare disease market than most of its products serve.
Access and eligibility
The patient part of the Phase 1/2a study takes adults aged 18 to 50 with a documented ALPL variant and no prior enzyme replacement, gene therapy or osteoporosis drugs. People who have taken Strensiq are excluded.
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
NCT07179640 is a randomized, placebo-controlled, double-blind Phase 1/2a study that began on September 30, 2025 and plans to enroll about 120 people[3]. Part 1 gives single and multiple rising doses to healthy adults. Part 2 enrolls adults aged 18 to 50 with a documented ALPL gene variant who have never received enzyme replacement therapy, gene therapy or osteoporosis treatment, have no hyperparathyroidism, and have had no new fracture in the previous 12 weeks[3].
The primary endpoints are safety measures through day 16; primary completion is estimated for January 2027[3]. As of October 18, 2026 the trial was recruiting and the sponsor of record was still Alesta Therapeutics.
Development history
ALE1 was developed by Alesta Therapeutics, a clinical-stage company led by Ilan Ganot. On August 18, 2026 BioMarin agreed to buy Alesta for $275 million upfront plus up to $215 million in development and regulatory milestones, with Alesta spinning out its other programs and staff into a separate company before closing[1]. BioMarin said the deal closed in a release dated September 1, 2026, and the program now sits in its Skeletal Conditions Business Unit beside Voxzogo for achondroplasia[2].
BioMarin cites more than 9,000 people diagnosed with HPP in the US as the addressable group[1].
Explore Hypophosphatasia trials
Other Hypophosphatasia treatments
Where to access ALE1
Investigational: available only through clinical trial enrollment
ALE1 has not been approved by the FDA and is not available by prescription. The only way for a patient to receive ALE1 today is by enrolling in an active clinical trial and meeting that trial's eligibility criteria. Decisions about trial participation should be made with the specialist who manages the patient's condition and knows their current treatment.
Primary active trial
ClinicalTrials.gov record NCT07179640
View full trial record on ClinicalTrials.gov →Trial status, site list, and eligibility are pulled from ClinicalTrials.gov. Confirm current recruitment status and nearest site directly with the study team before traveling.
Common questions about ALE1
▸What is ALE1?
An investigational pill for hypophosphatasia that aims to lower the pyrophosphate build-up behind the disease without injections. BioMarin bought it from Alesta Therapeutics in 2026; a Phase 1/2a safety study in healthy volunteers and adults with HPP is recruiting.
▸How does ALE1 work?
Every treatment for HPP so far has replaced the missing alkaline phosphatase enzyme by injection. ALE1 takes a different route. BioMarin says it is designed to inhibit a target that regulates inorganic pyrophosphate (PPi), the substance that accumulates when the enzyme is missing and stops calcium and phosphate from hardening bone; by lowering PPi from another direction it aims to restore healthier bone and mineral metabolism across the full spectrum of HPP[1]. The company has not named the target. If it works, it would be the first oral therapy for HPP and the first that does not depend on an enzyme infusion or injection[1].
▸What are the side effects of ALE1?
No safety results have been released. The first-in-human study measures adverse events, blood counts, blood chemistry, heart rhythm (ECG) and vital signs over the first 16 days after dosing in each group[3]. Until data are presented, nothing can be said about what side effects to expect.
▸How is ALE1 taken?
An oral pill. The Phase 1/2a study tests single and multiple ascending doses, with and without food, so no dose or schedule has been chosen[3].
▸Is ALE1 FDA approved?
ALE1 is currently in phase 1 clinical trials for Hypophosphatasia. It has not yet received FDA approval.
▸Is ALE1 available for hypophosphatasia?
No. It is in a Phase 1/2a first-in-human study and has no efficacy results. The only way to receive it is through that trial, which enrolls healthy volunteers and adults aged 18 to 50 with an ALPL variant who have never had enzyme replacement therapy.
▸Why does BioMarin own ALE1?
BioMarin agreed on August 18, 2026 to acquire Alesta Therapeutics for $275 million upfront plus up to $215 million in milestones to get ALE1, and said the deal closed on September 1, 2026. BioMarin wants an oral option for HPP alongside its skeletal medicines such as Voxzogo.
▸How would a pill treat HPP?
Strensiq and efzimfotase alfa replace the missing enzyme by injection. ALE1 instead blocks a target that controls inorganic pyrophosphate, the substance that builds up in HPP and stops bone from mineralizing. BioMarin has not disclosed the target or any human results.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- BioMarin Pharmaceutical · 2026-08-18. BioMarin to Acquire Alesta Therapeutics to Gain ALE1, a Potential First Oral Therapy for Hypophosphatasia. https://www.biomarin.com/news/press-releases/biomarin-to-acquire-alesta-therapeutics-to-gain-ale1-a-potential-first-oral-therapy-for-hypophosphatasia-adding-an-important-clinical-program-to-biomarins-pipeline/
- BioMarin Pharmaceutical · 2026-09-01. BioMarin Announces Close of the Alesta Therapeutics Acquisition. https://www.biomarin.com/news/press-releases/biomarin-announces-close-of-the-alesta-therapeutics-acquisition/
- ClinicalTrials.gov · 2026-08-03. A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adult Subjects and Adult Patients With Hypophosphatasia. https://clinicaltrials.gov/study/NCT07179640