Pharms, Andre: Company Profile

Background

Pharms, Andre is a pharmaceutical company specializing in the development and commercialization of protein replacement therapies and precision medicines for rare diseases. The company operates primarily in the United States, Europe, and internationally, focusing on treatments for conditions such as hereditary angioedema (HAE) and activated phosphoinositide 3-kinase delta syndrome (APDS).

Key Strategic Focus

Pharms, Andre's strategic focus centers on:

• Core Objectives: Developing innovative therapies for rare diseases, expanding its product portfolio, and enhancing global market presence.

• Areas of Specialization: Protein replacement therapies, precision medicines, and gene therapies targeting rare and life-threatening conditions.

• Key Technologies Utilized: Recombinant protein production systems, small molecule inhibitors, and ex-vivo autologous hematopoietic stem cell gene therapy.

• Primary Markets Targeted: Patients with rare diseases, healthcare providers, and global pharmaceutical markets.

Financials and Funding

Pharms, Andre has demonstrated significant financial growth, with total revenue reaching approximately $97.3 million in the third quarter of 2025. The company has strategically invested in expanding its product portfolio and pipeline development. Notably, in 2025, Pharms, Andre acquired Abliva AB for $66.1 million, adding KL1333, a pivotal-stage asset targeting primary mitochondrial diseases, to its pipeline.

Pipeline Development

Pharms, Andre's pipeline includes:

• RUCONEST®: A recombinant C1 esterase inhibitor for treating acute attacks in adult and adolescent patients with hereditary angioedema (HAE).

• Joenja® (leniolisib): An oral small molecule PI3Kδ inhibitor for treating activated phosphoinositide 3-kinase delta syndrome (APDS).

• OTL-105: An investigational ex-vivo autologous hematopoietic stem cell gene therapy for treating HAE.

• KL1333: A pivotal-stage asset targeting primary mitochondrial diseases, added through the acquisition of Abliva AB in 2025.

Technological Platform and Innovation

Pharms, Andre distinguishes itself through:

• Proprietary Technologies: A rabbit-based protein production system for recombinant proteins.

• Significant Scientific Methods: Advanced gene therapy techniques, including ex-vivo autologous hematopoietic stem cell gene therapy.

• AI-Driven Capabilities: Utilization of artificial intelligence in drug discovery and development processes.

Leadership Team

The leadership team comprises:

• Fabrice Chouraqui, Ph.D., Pharm.D.: Chief Executive Officer and Executive Director.

• Kenneth Lynard, EMBA: Chief Financial Officer.

• Mireille Sanders, M.Sc.: Chief Operations Officer.

• Susanne Embleton: Investor Relations Manager.

• Ruud Van Outersterp: Chief Legal and Compliance Officer.

• Michael Levitan: Vice President of Investor Relations and Corporate Communications.

• Ines Bernal: Chief People Officer.

• Dr. Anurag Relan, M.D., MPH: Chief Medical Officer.

• Dr. Alexander Breidenbach, M.B.A.: Chief Business Officer.

• Leverne Marsh: Chief Commercial Officer.

In January 2026, Leverne Marsh succeeded Stephen Toor as Chief Commercial Officer, reflecting the company's commitment to strengthening its commercial operations.

Competitor Profile

Market Insights and Dynamics

The rare disease pharmaceutical market is experiencing significant growth, driven by advancements in biotechnology and increasing global awareness of rare conditions. Pharms, Andre's focus on rare diseases positions it favorably within this expanding market.

Competitor Analysis

Key competitors include:

• BioCryst Pharmaceuticals: Specializes in rare disease treatments, with a focus on hereditary angioedema.

• KalVista Pharmaceuticals: Develops oral therapies for diabetic macular edema and other retinal diseases.

• CSL Behring: A global leader in biotherapies for rare and serious diseases.

Pharms, Andre differentiates itself through its proprietary technologies and comprehensive pipeline targeting multiple rare diseases.

Strategic Collaborations and Partnerships

Pharms, Andre has established strategic collaborations, including:

• Novartis: Development collaboration and license agreement.

• Orchard Therapeutics plc: Strategic collaboration for research, development, manufacturing, and commercialization of OTL-105.

Operational Insights

Pharms, Andre's operational strategy focuses on:

• Major Competitors: Monitoring and responding to developments from key competitors in the rare disease space.

• Market Position: Strengthening its position through innovative therapies and strategic partnerships.

• Competitive Advantages: Proprietary technologies, a diversified pipeline, and a focus on underserved rare diseases.

Strategic Opportunities and Future Directions

Pharms, Andre's strategic roadmap includes:

• Future Business Directions: Expanding its portfolio to address additional rare diseases.

• Opportunities for Expansion: Entering new geographic markets and enhancing global distribution networks.

• Achieving Future Objectives: Leveraging current strengths to drive growth and innovation in the rare disease sector.

Contact Information

• Official Website: Pharms, Andre official website.

• Social Media Profiles:

• LinkedIn: Pharms, Andre LinkedIn page.

• Twitter: Pharms, Andre Twitter account.

• Facebook: Pharms, Andre Facebook page.

• Headquarters Location: City, Country.