What AM Pharma does

AM-Pharma is a clinical-stage biopharmaceutical company headquartered in Utrecht, the Netherlands, founded in 2001/2002, that develops a single proprietary therapeutic candidate, ilofotase alfa (recombinant human alkaline phosphatase, engineered from two naturally occurring human isoforms of the alkaline phosphatase enzyme). The compound acts via two mechanisms: enzyme replacement that reduces elevated pyridoxal-5′-phosphate (PLP) and inorganic pyrophosphate (PPi) substrates central to hypophosphatasia (HPP), and dephosphorylation of lipopolysaccharides (LPS) and extracellular ATP that inactivates pro-inflammatory ATP and produces adenosine acting on the immunosuppressive A2a receptor pathway. Following a positive Phase II STOP-AKI study (301 patients, published in JAMA, demonstrating >40% relative mortality reduction), the pivotal Phase III REVIVAL trial in sepsis-associated AKI was stopped in October 2022 for futility. AM-Pharma then pivoted development to adult HPP, where the Phase 1b study showed dose-dependent normalization of alkaline phosphatase activity and pharmacologic effects on all HPP-specific biomarkers (PPi, PLP, PEA) with no safety concerns, and has additionally initiated a Phase 2 trial in cardiac surgery-associated renal damage (enrollment completed October 2025).

The asset is protected by FDA and EMA orphan drug designation for hypophosphatasia and previously held FDA Fast Track for SA-AKI; ~1,000 patients have been exposed to ilofotase alfa across clinical studies. AM-Pharma has raised more than EUR 240 million in equity and debt from a European-led syndicate that includes LSP/EQT Life Sciences, Andera Partners, Forbion, Ysios Capital, Kurma Partners, Gilde Healthcare, Idinvest Partners, Pureos Bioventures, and corporate venture investors Pfizer, Takeda, and Nestlé Health Sciences, plus an innovation credit from the Netherlands Enterprise Agency (RVO).

AM-Pharma's revenue model is currently milestone-driven: the September 2021 exclusive license with Kyowa Kirin covers Japan and provides a EUR 20 million upfront, up to EUR 245 million in development, regulatory and sales milestones, tiered double-digit royalties, and drug supply fees. Outside Japan, AM-Pharma is building toward direct commercialization to hospitals and ICUs for acute indications (via IV) and to adult HPP patients via subcutaneous injection; a U.S. affiliate (AM-Pharma Inc.) was established in September 2021 and commercial-oriented hires (CFO, VP Commercial Strategy, VP Value & Market Access, CBO) have been added. The customer base for the eventual product is fragmented across hospital systems and rare-disease patient populations (HPP), with no commercial product currently generating revenue.

Name

AM Pharma

Legal name

AM-Pharma Holding B.V./AM-Pharma B.V.

Website

https://am-pharma.com

Company type

Private

Founded year

2002

Operating status

Operating

Headcount range

11–50 employees

Short description

AM-Pharma is a clinical-stage biopharmaceutical company developing ilofotase alfa, a proprietary recombinant human alkaline phosphatase, for adult hypophosphatasia and cardiac surgery-associated renal damage. Headquartered in Utrecht, Netherlands; partnered with Kyowa Kirin for Japan.

Ownership category

akta.pro rank

Product category

Biopharmaceutical Therapeutics

NAICS

Pharmaceutical Preparation Manufacturing (325412), Pharmaceutical and Medicine Manufacturing (3254), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Nephrology & Urology Specialty Pharmaceuticals (HLAIACAH)

akta.pro secondary industry

Immunology & Autoimmune Specialty Pharmaceuticals (HLAIACAB)

Keywords

• Biopharmaceutical therapeutics
• Enzyme replacement therapy
• Recombinant alkaline phosphatase
• Acute kidney injury treatment
• Rare disease therapeutics

Headquarters

HQ city

Bunnik

HQ country

Netherlands

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Supply Chain, Marketing or Sales

Revenue model

1. Licensing and Milestone Payments: AM-Pharma receives upfront payments, development and regulatory milestones, and sales milestones from licensing partners. Deal with Kyowa Kirin includes EUR 20 million upfront, EUR 30 million pre-submission milestones, up to EUR 195 million upon submission, NHI price listing and sales milestones, plus tiered double-digit royalties on sales and drug supply fees.
2. Pharmaceutical Product Sales: Revenue from direct commercialization of ilofotase alfa upon regulatory approval for treatment of sepsis-associated acute kidney injury (SA-AKI) and hypophosphatasia (HPP).

Go-to-market motion1 record

Distribution channels2 records

Marketing channels4 records

Core offering

AM-Pharma is a clinical-stage biopharmaceutical company developing ilofotase alfa, a proprietary recombinant human alkaline phosphatase (rhAP) enzyme replacement therapy. The compound is being advanced for adult hypophosphatasia (HPP) as a Phase 2-ready program, with prior development in sepsis-associated acute kidney injury (SA-AKI) and ongoing work in cardiac surgery-associated renal damage. The drug targets severe inflammatory and infectious diseases where there are no approved pharmacological treatments.

Product overview

AM Pharma is a clinical-stage biopharmaceutical company developing a single proprietary therapeutic compound, ilofotase alfa (recombinant human alkaline phosphatase). The drug is being developed for multiple indications: sepsis-associated acute kidney injury (SA-AKI) as the lead indication in Phase III (REVIVAL trial), hypophosphatasia (HPP) as Phase 2-ready, and cardiac surgery-associated renal damage in Phase 2. Ilofotase alfa acts as an enzyme replacement therapy addressing elevated levels of pyridoxal-5′-phosphate (PLP) and inorganic pyrophosphates (PPi), with a dual mechanism of action via dephosphorylation of DAMPs and PAMPs.

Differentiator

Problem solved

Functional benefit

Products and services

• Ilofotase alfa (recombinant human Alkaline Phosphatase) Proprietary recombinant human alkaline phosphatase (rhAP) therapeutic candidate constructed from two human isoforms. Being developed as enzyme replacement therapy for adult hypophosphatasia (HPP), with prior Phase III development for sepsis-associated acute kidney injury (SA-AKI) and Phase 2 development for cardiac surgery-associated renal damage. Administered via subcutaneous injection for HPP and IV for acute conditions. Demonstrated to be well-tolerated, stable, and highly active in multiple clinical trials.

Quantifiable outcome

• Phase II STOP-AKI: >40% relative reduction in mortality compared to placebo in sepsis-associated AKI patients
• +4 more outcomes

Named customers3 records

Segments2 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

One partnership is on record.

• Kyowa KirincoreOEM/ Whitelabel/ Licensing Partner · 8 September 2021Exclusive license agreement for development and commercialization of ilofotase alfa in Japan. AM-Pharma receives EUR 20 million upfront, EUR 30 million pre-submission milestones, up to EUR 195 million upon submission, NHI price listing and sales milestones, tiered double-digit royalties on sales, and drug supply fee. Kyowa Kirin responsible for regulatory approval and commercialization in Japan.

Scale indicators13 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• BioMarin Pharmaceutical: Global rare disease biotech with multiple approved enzyme replacement and biologics therapies for rare genetic conditions including HPP-adjacent metabolic disorders. Comparable in scale, clinical stage asset portfolio, and orphan drug strategy.
• Alexion (AstraZeneca Rare Disease): Markets Strensiq (asfotase alfa), the only approved enzyme replacement therapy for hypophosphatasia and the direct competitive incumbent to AM-Pharma's ilofotase alfa. Both companies develop biologics for rare genetic metabolic disorders and target the same HPP prescriber base.
• Amicus Therapeutics: Rare disease biotech focused on enzyme replacement therapies and gene therapies for devastating genetic conditions, with approved products and pipeline assets addressing similar patient populations. Closely comparable development model and therapeutic categories.
• Ultragenyx Pharmaceutical: Clinical-stage and commercial rare disease biotech focused on novel therapies for serious rare and ultra-rare genetic diseases, with multiple enzyme and protein replacement therapies in development or on market. Directly comparable business model, target indications (rare metabolic diseases), and development stage to AM-Pharma.
• Orchard Therapeutics: Clinical-stage gene therapy company focused on rare genetic metabolic and immune disorders, including programs that could compete or combine with enzyme replacement approaches like ilofotase alfa. Similar development stage and rare disease specialization.
• BridgeBio Pharma: Clinical-stage biotech developing targeted therapies for genetic diseases and cancers, including rare disease programs in similar developmental stages to AM-Pharma's pipeline. Comparable business model of advancing multiple indications from a focused genetic disease platform.
• Swedish Orphan Biovitrum (Sobi): Specialty biopharma focused on rare diseases, with a portfolio including enzyme replacement therapies and biologics for rare inflammatory and genetic conditions. Highly comparable target market, distribution model through specialty channels, and orphan drug focus.

Broad incumbents

• Pfizer: Global pharmaceutical incumbent that held an exclusive option to acquire AM-Pharma (paid $87.5M upfront in 2015, did not exercise). Operates broadly in rare diseases and nephrology-relevant therapeutic areas; relevant as both a current investor and potential future strategic acquirer.
• Takeda Pharmaceutical: Global pharma with a substantial rare disease franchise (post-Shire acquisition) and existing investor in AM-Pharma. Operates broadly across nephrology, metabolic, and rare disease indications overlapping with AM-Pharma's therapeutic focus.

Emerging players

• Chiesi Farmaceutici: Privately-held specialty pharma with a growing rare disease franchise (gained through the $1.85B acquisition of Amryt Pharma) focused on metabolic and enzyme deficiency disorders. Potential strategic acquirer of regional rights or partner for ilofotase alfa commercialization.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/am-pharma

Compliance3 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles20 records

Subsidiaries1 record

Funding overview

Funding rounds10 records

Investors17 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about AM Pharma