AM-Pharma — Stock, Pipeline & Market Cap

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Private Company

Total funding raised: $480M

Overview

AM-Pharma is a clinical-stage biotech developing ilofotase alfa, a recombinant alkaline phosphatase therapy for adult hypophosphatasia (HPP). The drug has demonstrated promising effects on disease biomarkers and a favorable safety profile in a Phase 1b proof-of-concept study, positioning it as a potential treatment for an underserved adult patient population. The company is privately held, pre-revenue, and is advancing its lead program based on a targeted enzyme replacement platform.

Rare Genetic DiseasesMetabolic Disorders

Technology Platform

Proprietary recombinant human alkaline phosphatase (ilofotase alfa) engineered from two human isoforms, designed as an enzyme replacement therapy without a bone-targeting sequence.

Pipeline

13

13 drugs in pipeline1 in Phase 3

Drug	Indication	Stage
Recombinant human alkaline phosphatase	Acute Kidney Injury Due to Sepsis	Phase 3
Ilofotase alfa + Placebo	Open Heart Surgery	Phase 2
Alkaline Phosphatase + Placebo	Sepsis	Phase 2
recAP	Acute Kidney Injury	Phase 2
Placebo + BIAP	Sepsis	Phase 2

Funding History

7

Total raised:$480M

Series C$116MLSPSep 15, 2020

Series D$100MForbion Capital PartnersJul 15, 2020

Series C$116MLSPJun 15, 2018

Series B$47MForbionJun 15, 2017

Opportunities

A significant unmet medical need exists in adult-onset hypophosphatasia (HPP), where there are currently no approved disease-modifying treatments.

Ilofotase alfa's design, which lacks a bone-targeting sequence, offers a potential point of differentiation by addressing systemic symptoms like pain and muscle weakness, potentially capturing a unique position in the market.

Orphan drug designations in the US and EU provide regulatory and commercial incentives.

Risk Factors

The company faces clinical development risk, as positive Phase 1b biomarker data must be confirmed in larger, pivotal trials demonstrating clinical benefit.

As a pre-revenue private company, it carries high financing risk to fund expensive late-stage studies.

Competitive and market risks include potential new entrants and the challenge of identifying and diagnosing the target adult HPP patient population.

Competitive Landscape

The primary competitor is Alexion's asfotase alfa (Strensiq), but it is only approved for pediatric-onset HPP, creating a direct gap in adult care. Other potential competition could come from companies developing gene therapies or other enzyme replacement approaches for HPP. AM-Pharma's key competitive hypothesis is that its non-bone-targeted enzyme may offer broader systemic benefits for adult patients.

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