Asklepion Pharmaceuticals

What Asklepion Pharmaceuticals does

Asklepion Pharmaceuticals, LLC is a privately held, Baltimore-based biopharmaceutical company founded in 2006 that develops and commercializes treatments for rare pediatric diseases with little to no existing therapeutic options. The company is incorporated in Delaware, operates with 11-50 employees, and is led by CEO Jeff Courtney, with Gurdyal Kalsi as Chief Medical Officer. Asklepion's portfolio is focused on endogenous pharmaceutical products—naturally occurring compounds such as L-citrulline, cholic acid, and ursodeoxycholic acid—targeting ultra-rare indications including bile acid synthesis disorders and acute lung injury induced by cardiopulmonary bypass in pediatric patients undergoing congenital heart surgery.

The company's principal technology platform is IV L-citrulline, a urea cycle intermediate and nitric oxide precursor, being developed to prevent acute lung injury in children undergoing cardiopulmonary bypass. Asklepion holds an exclusive worldwide license from Vanderbilt University for this technology, covered by US Patent 10,265,286 (issued April 2019) with coverage extending to June 2036. The Phase III program completed enrollment of 192 patients at 29 centers across the USA, Israel, Germany, and Austria in July 2019, and is supported by an FDA Orphan Drug Designation (granted January 2017) and a Special Protocol Assessment (December 2017). The company previously developed Cholbam (cholic acid), which received FDA approval in March 2015 as the first treatment for bile acid synthesis disorders, and Kolbam (cholic acid), which received a positive CHMP opinion from the EMA in April 2014; both products' commercial rights were subsequently divested to Retrophin.

Asklepion monetizes through a hybrid model combining direct commercialization of approved treatments, licensing/royalty arrangements, and partnerships. Following the 2015 sale of Cholbam worldwide rights to Retrophin and the 2016 sale of URSOFALK US rights, the company's principal near-term value driver is the IV L-citrulline program, with revenue largely dependent on retained licensing/royalty streams and contingent milestone payments. The company has not disclosed venture or private equity funding rounds and appears to operate as a capital-efficient, founder/management-controlled entity that leverages academic partnerships (Vanderbilt, University of Arkansas for Medical Sciences) and CRO relationships to advance development.

Name

Asklepion Pharmaceuticals

Legal name

Asklepion Pharmaceuticals, LLC

Website

https://asklepionpharm.com

Company type

Private

Founded year

2006

Operating status

Operating

Headcount range

11–50 employees

Short description

Asklepion Pharmaceuticals is a privately held Baltimore-based biopharmaceutical company founded in 2006 that develops endogenous pharmaceutical products—chiefly IV L-citrulline and cholic acid—for rare pediatric diseases with high unmet need, including bile acid synthesis disorders and acute lung injury in pediatric cardiac surgery.

Ownership category

akta.pro rank

Product category

Pediatric Rare Disease Pharmaceuticals

NAICS

Scientific Research and Development Services (5417), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

akta.pro secondary industries

Rare Gastroenterology & Hepatology Disorder Therapies (HLAIAIAL), Rare Cardiovascular & Vascular Disorder Therapies (HLAIAIAH)

Keywords

• Rare disease pharmaceuticals
• Pediatric drug development
• Clinical trial development
• Orphan drug development
• Bile acid disorders

Headquarters

HQ city

Brentwood

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pharmaceutical Product Sales: Direct commercialization of FDA-approved pharmaceutical products for rare diseases. In 2015, Asklepion obtained FDA approval for Cholbam (cholic acid) for bile acid synthesis defects.
2. Licensing and Royalty Revenue: Revenue from licensing agreements including sale of worldwide rights to Cholbam to Retrophin and sale of U.S. rights to ursodeoxycholic acid suspension.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels4 records

Core offering

Asklepion Pharmaceuticals is a pediatric, rare disease pharmaceutical development company that discovers, develops, and commercializes endogenous pharmaceutical products (L-citrulline and cholic acid-based treatments) for children with rare conditions who have limited therapeutic options. The company secured FDA approval for Cholbam (cholic acid) for bile acid synthesis disorders in March 2015, and is currently advancing IV L-citrulline through Phase III clinical trials for prevention of acute lung injury in pediatric patients undergoing cardiopulmonary bypass for congenital heart defect surgery.

Product overview

Asklepion Pharmaceuticals is a pediatric, rare disease pharmaceutical development company focused on discovering, developing, and commercializing treatments for patients with rare conditions who have limited therapeutic options. The company has developed and commercialized treatments including Cholbam (cholic acid) for bile acid synthesis disorders and currently has IV Citrulline in Phase III clinical development for acute lung injury prevention in pediatric cardiac surgery patients. Asklepion also holds rights to URSOFALK Suspension (ursodeoxycholic acid) for the US market and Kolbam (cholic acid) for the EU market.

Differentiator

Problem solved

Functional benefit

Products and services

• IV Citrulline (Intravenous L-Citrulline) Intravenous L-citrulline being developed for prevention of clinical sequelae of acute lung injury induced by cardiopulmonary bypass in pediatric patients undergoing surgery for congenital heart defects. Citrulline is a urea cycle intermediate and important precursor of nitric oxide. Pipeline product currently in Phase III clinical development.
• Cholbam (cholic acid) FDA-approved oral treatment for bile acid synthesis disorders due to single enzyme defects, and as adjunctive treatment of peroxisomal disorders including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat soluble vitamin absorption. Rights were acquired by Retrophin in March 2015.
• Kolbam (cholic acid) EMA-approved treatment for inborn errors of primary bile acid synthesis in infants from one month of age for continuous lifelong treatment through adulthood, encompassing single enzyme defects including sterol 27-hydroxylase deficiency (CTX), AMACR deficiency, and CYP7A1 deficiency.
• URSOFALK Suspension (ursodeoxycholic acid) Ursodeoxycholic acid suspension for which Asklepion obtained exclusive rights to market in the United States from Dr. Falk Pharma GmbH. U.S. rights were subsequently sold to Retrophin in June 2016.

Quantifiable outcome

• Phase 1b/2a study achieved sustained target plasma citrulline level of >100 µmol/L in patients receiving citrulline
• +1 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Six partnerships are on record, tiered core and minor.

• Frederick E. Barr, M.D. (University of Arkansas for Medical Sciences)coreStrategic or Co-development Partner · 19 December 2017Dr. Frederick E. Barr serves as coordinating investigator for Phase III trial. He is chair of the Department of Pediatrics and associate dean for child health in the University of Arkansas for Medical Sciences College of Medicine, and pediatrician-in-chief for Arkansas Children's.
• Pediatric Cardiac Intensive Care SocietyminorStrategic or Co-development Partner · 8 December 2017Asklepion presented Phase 1b/2a clinical trial data for IV-Citrulline at the PCICS 13th Annual International Meeting in Washington, D.C.
• RetrophinminorChannel Partner/ Reseller/ Distributor · 9 June 2016Asklepion sold its U.S. rights, titles, and ownership of ursodeoxycholic acid suspension to Retrophin.
• Retrophin (Nasdaq: RTRX)coreChannel Partner/ Reseller/ Distributor · 18 March 2015Retrophin exercised right to acquire all worldwide rights to Cholbam (cholic acid) from Asklepion Pharmaceuticals for treatment of bile acid synthesis defects. This included acquisition of all worldwide rights, titles, and ownership.
• Dr. Falk Pharma GmbHcoreChannel Partner/ Reseller/ Distributor · 23 October 2006Asklepion obtained exclusive rights to market URSOFALK Suspension (ursodeoxycholic acid) in the United States from Dr. Falk Pharma GmbH.
• Vanderbilt UniversitycoreTechnology or Integration · 14 July 2006Exclusive license from Vanderbilt University for intravenous L-citrulline technology. Vanderbilt researchers discovered that cardiopulmonary bypass significantly decreased blood levels of citrulline, compromising nitric oxide production. Asklepion holds exclusive worldwide rights to the patent family covering this technology.

Scale indicators5 records

Recent moves8 records

Expansion highlights4 records

Broad incumbents

• Genzyme (Sanofi): Pioneering rare disease business within Sanofi with deep pediatric metabolic and lysosomal franchises; relevant benchmark for the rare disease commercial model and partnership/late-stage acquisition dynamics.
• BioMarin Pharmaceutical: Larger rare disease commercial-stage biotech with multiple pediatric rare disease franchises (e.g., enzyme replacement therapies); comparable in therapeutic area focus and Orphan Drug–driven business model, though operating at much greater scale.
• Recordati Rare Diseases: Specialty pharma division focused on rare and orphan diseases with strong pediatric metabolic and gastroenterology assets; comparable overlap in endogenous-substance / metabolite-replacement therapies and U.S. rare disease distribution.
• Alexion Pharmaceuticals (AstraZeneca Rare Disease): Established rare disease franchise including pediatric and metabolic indications (e.g., complement-mediated and metabolic disorders); relevant comparable for orphan exclusivity strategy, pediatric rare disease commercialization, and licensing approach.
• Chiesi Farmaceutici: Private specialty pharma group with a rare disease division active in pediatric metabolic and hepatology; comparable in endogenous-substance, metabolite-replacement focus and partnership-led U.S. commercialization approach.

Direct peers

• Amicus Therapeutics: Rare disease biotech developing treatments for lysosomal storage and metabolic disorders; comparable in targeting rare metabolic conditions with high unmet need and pediatric patient populations.
• Ultragenyx Pharmaceutical: Commercial-stage rare disease biotech focused on ultra-rare and rare pediatric metabolic disorders; directly comparable to Asklepion in business model (orphan drug development, endogenous-substitute mechanisms, pediatric rare disease focus) and target physician/KOL audience.
• Travere Therapeutics (formerly Retrophin): Acquired Cholbam rights from Asklepion in 2015 and the ursodeoxycholic acid rights in 2016; operates a rare disease commercial model with overlapping pediatric metabolic and rare disease franchises, directly comparable as both a counterparty and a competitor in the same therapeutic space.

Emerging players

• Soligenix: Smaller, clinical-stage rare disease biopharmaceutical company focused on serious unmet needs in pediatrics; comparable in operational scale, Orphan Drug strategy, and pipeline-stage orientation.

Others

• Dr. Falk Pharma GmbH: European specialty pharma in hepatology and gastroenterology; Asklepion's historical licensing partner for URSOFALK (ursodeoxycholic acid) in the U.S. market — relevant as an adjacent hepatic/GI rare disease counterpart and channel partner.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

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Compliance4 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Asklepion Pharmaceuticals