Orbus Therapeutics, Inc. Market Research Report

Background

Orbus Therapeutics, Inc., established in 2012, is a private, late-stage biopharmaceutical company dedicated to developing treatments for rare diseases with limited or no effective therapies. The company's mission is to improve the health and quality of life for patients affected by these conditions. Orbus Therapeutics focuses on the development and commercialization of eflornithine, targeting malignant gliomas, including anaplastic astrocytoma and glioblastoma.

Key Strategic Focus

Orbus Therapeutics' strategic focus centers on:

• Core Objectives: Advancing eflornithine through clinical development to provide new treatment options for patients with malignant gliomas.

• Areas of Specialization: Specializing in oncology, particularly in the treatment of rare central nervous system cancers.

• Key Technologies Utilized: Developing eflornithine, a cytostatic agent that irreversibly inhibits ornithine decarboxylase, a key enzyme in polyamine biosynthesis upregulated in certain cancers.

• Primary Markets Targeted: Addressing unmet medical needs in North America and Europe for patients with malignant gliomas.

Financials and Funding

Orbus Therapeutics has secured significant funding to support its clinical programs:

• Series A Financing: Raised $32.5 million in August 2015.

• Series A2 Financing: Secured an additional $18.5 million in March 2019.

• Series A3 Financing: Further expanded funding by $10 million in August 2020.

• Total Funds Raised: Approximately $71 million to date.

Notable investors include Longitude Capital, H.I.G. BioVentures, Adams Street Partners, and Abingworth LLP. The capital is primarily allocated to advancing the STELLAR Phase 3 study and initiating additional clinical studies in patients with malignant gliomas.

Pipeline Development

Orbus Therapeutics' pipeline is centered around eflornithine:

• Eflornithine:

• Indication: Treatment of recurrent anaplastic astrocytoma and newly diagnosed glioblastoma.

• Clinical Trials:

• STELLAR Study: A Phase 3, randomized, open-label trial evaluating eflornithine in combination with lomustine versus lomustine alone in patients with recurrent anaplastic astrocytoma. The study completed in 2024, demonstrating a clinically meaningful improvement in progression-free survival (PFS) and overall survival (OS) for a defined subgroup of patients with grade 3 astrocytoma.

• Phase 1b Study: Initiated in September 2023, this open-label, single-arm, dose-limiting toxicity trial evaluates the safety and pharmacokinetics of eflornithine combined with temozolomide in patients with newly diagnosed glioblastoma.

Technological Platform and Innovation

Orbus Therapeutics' innovation is exemplified by its development of eflornithine:

• Proprietary Technologies: Eflornithine is a novel cytostatic agent that irreversibly inhibits ornithine decarboxylase, a key enzyme in polyamine biosynthesis upregulated in certain cancers.

• Significant Scientific Methods: The company employs rigorous clinical trial methodologies to assess the efficacy and safety of eflornithine in combination with other chemotherapeutic agents for treating malignant gliomas.

Leadership Team

• Jason Levin: Co-founder and President. Prior to founding Orbus Therapeutics, Jason was Chief Business Officer of Sorbent Therapeutics, Inc., a company focused on the development of oral polymers to treat chronic heart failure. Prior to Sorbent, he was Chief Business Officer at Brain Cells, Inc., a CNS focused drug discovery and development company. Before BrainCells, Jason was one of the initial employees at Jazz Pharmaceuticals, Inc. and was Vice President, Corporate Development. Before joining Jazz Pharmaceuticals, Jason was Director of Business Development at ALZA Corporation, a wholly owned subsidiary of Johnson & Johnson. He received a BA from the University of California, San Diego and a MBA from the University of Texas, McCombs Graduate School of Business.

Leadership Changes

In February 2025, Jason Levin, co-founder and Chief Operating Officer, was appointed President of Orbus Therapeutics. Bob Myers, co-founder and President and CEO since 2012, stepped down to pursue other opportunities in the industry.

Competitor Profile

Market Insights and Dynamics

The market for treating malignant gliomas is characterized by:

• Market Size: In the United States, approximately 3,600 new cases of anaplastic glioma are diagnosed annually, with a median survival of just over three years despite current treatments.

• Growth Potential: There is significant growth potential due to the high unmet medical need and limited effective therapies for malignant gliomas.

• Industry Trends: Increasing focus on developing targeted therapies and combination treatments to improve patient outcomes in rare and aggressive cancers.

Competitor Analysis

Key competitors in the malignant glioma treatment space include:

• Enterome: A biopharmaceutical company specializing in microbiome-based therapies for cancer and immune diseases.

• Memgen: Focuses on developing immunotherapies for cancer treatment.

• NexImmune: Develops T cell immunotherapies targeting cancer and autoimmune diseases.

• Galera Therapeutics: Works on therapies to reduce radiation-induced toxicities and improve cancer treatment outcomes.

• Iovance Biotherapeutics: Specializes in tumor-infiltrating lymphocyte (TIL) therapies for solid tumors.

These companies are developing various innovative therapies, contributing to a competitive landscape in the treatment of malignant gliomas.

Strategic Collaborations and Partnerships

Orbus Therapeutics has engaged in significant collaborations to enhance its development efforts:

• Exclusive License Agreement: In January 2023, Orbus entered into an exclusive license agreement with Michigan State University and Helen DeVos Children's Hospital, granting rights to intellectual property related to the use of eflornithine for treating Bachmann-Bupp Syndrome (BABS), a rare pediatric condition.

Operational Insights

Orbus Therapeutics' strategic considerations include:

• Market Position: Positioned as a leader in developing therapies for rare malignant gliomas, focusing on unmet medical needs.

• Competitive Advantages: Proprietary development of eflornithine, a novel cytostatic agent with potential to improve survival outcomes in malignant glioma patients.

• Differentiators: Focused approach on rare diseases, strong clinical trial data, and strategic partnerships enhancing research and development capabilities.

Strategic Opportunities and Future Directions

Orbus Therapeutics' strategic roadmap includes:

• Regulatory Approvals: Pursuing regulatory approvals for eflornithine based on positive clinical trial outcomes.

• Pipeline Expansion: Exploring additional indications for eflornithine and potential combination therapies.

• Market Expansion: Extending market reach in North America and Europe to provide access to therapies for patients with malignant gliomas.

• Partnerships: Seeking strategic collaborations to enhance research, development, and commercialization efforts.

Contact Information

For more information, visit Orbus Therapeutics' official website: www.orbustherapeutics.com