What Orbus Therapeutics does

Orbus Therapeutics, Inc. is a private, late-stage biopharmaceutical company founded in 2012 and headquartered in Menlo Park, California. The company is focused exclusively on developing eflornithine (alpha-difluoromethylornithine, DFMO), an oral solution and the only irreversible inhibitor of ornithine decarboxylase (ODC), the rate-limiting enzyme in polyamine biosynthesis. The lead clinical indication is recurrent grade 3 IDH-mutant astrocytoma, where the Phase 3 STELLAR study (NCT02796261) in 343 patients demonstrated clinically meaningful improvements in overall survival (34.9 vs 23.5 months, HR=0.64) and progression-free survival (15.8 vs 7.2 months, HR=0.57) for eflornithine + lomustine versus lomustine alone in the pre-specified IDH-mutant subgroup, with results published in the Journal of Clinical Oncology in December 2025. Eflornithine carries FDA Breakthrough Therapy Designation, FDA Orphan Drug Designation, and EMA Orphan Medicinal Product status, and is protected by US and European method-of-use patents.

The company's go-to-market model is bifurcated: direct commercialization in North America through specialty pharmacy and hospital channels upon regulatory approval, paired with active out-licensing of ex-North America rights to corporate partners. Total funding raised is approximately $71 million in Series A tranches (2015 and 2020) from Longitude Capital, H.I.G. HealthBridge Partners, Adams Street Partners, and Abingworth. Beyond the lead glioma indication, Orbus holds an exclusive license (Jan 2023) for eflornithine use in Bachmann-Bupp Syndrome (BABS), a rare pediatric ODC1 genetic disorder, and initiated a Phase 1b study (NCT05879367) in newly diagnosed glioblastoma in September 2023. Leadership transitioned in February 2025 with founder Jason Levin assuming the President and CEO role following Bob Myers' departure. The company is pre-revenue with no approved products in any jurisdiction.

Name

Orbus Therapeutics

Legal name

Orbus Therapeutics, Inc.

Website

https://orbustherapeutics.com

Company type

Private

Founded year

2012

Operating status

Operating

Headcount range

11–50 employees

Short description

Orbus Therapeutics is a private, late-stage biopharmaceutical company developing eflornithine (DFMO), an oral irreversible ODC inhibitor, as a treatment for recurrent grade 3 IDH-mutant astrocytoma and other rare diseases, with planned direct commercialization in North America.

Ownership category

akta.pro rank

Product category

Oncology Pharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

Keywords

• Rare disease therapeutics
• Oncology pharmaceuticals
• Brain tumor treatment
• Polyamine pathway inhibitors
• Investigational drug development

Headquarters

HQ city

Palo Alto

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Eflornithine commercialization (pre-revenue): Orbus Therapeutics is a late-stage private biopharmaceutical company developing eflornithine for rare diseases. The company plans to commercialize eflornithine in North America and is actively seeking corporate partners for development and commercialization in other regions. Eflornithine is an investigational product not yet approved for any disease.
2. Licensing and partnership revenues: Orbus is pursuing a strategy of out-licensing or co-development partnerships for eflornithine in geographies outside North America, as well as exploring additional development-stage and commercial-stage opportunities to expand its portfolio.

Go-to-market motion2 records

Distribution channels2 records

Marketing channels4 records

Core offering

Orbus Therapeutics is a late-stage private biopharmaceutical company developing eflornithine (alpha-difluoromethylornithine, DFMO), an investigational oral solution that is the only irreversible inhibitor of ornithine decarboxylase (ODC). The lead indication is recurrent grade 3 IDH mutant astrocytoma, with additional programs in newly diagnosed glioblastoma and Bachmann-Bupp Syndrome (BABS), a rare pediatric genetic disorder.

Product overview

Orbus Therapeutics is a late-stage private biopharmaceutical company developing a single lead product candidate, eflornithine (DFMO), an oral solution for treating rare diseases, specifically recurrent grade 3 astrocytoma. The company is not structured as a platform-plus-modules architecture; it is focused on the development and potential commercialization of this one investigational drug candidate in North America and Europe. The product is an investigational therapy not yet approved for any disease in the United States or any other country.

Differentiator

Problem solved

Functional benefit

Products and services

• Eflornithine (DFMO) Oral Solution Investigational oral solution and irreversible metabolic inhibitor targeting ornithine decarboxylase (ODC), developed for the treatment of recurrent grade 3 IDH mutant astrocytoma in patients who have progressed after irradiation and adjuvant temozolomide chemotherapy. Also being evaluated in newly diagnosed glioblastoma and Bachmann-Bupp Syndrome.

Quantifiable outcome

• Median OS of 34.9 months vs 23.5 months (HR=0.64, p=0.01) in recurrent grade 3 IDH mutant astrocytoma patients treated with eflornithine + lomustine vs lomustine alone
• +3 more outcomes

Segments3 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Two partnerships are on record, tiered core and minor.

• Michigan State University (MSU) and Helen DeVos Children's Hospital (HDVCH, Corewell Health)coreStrategic or Co-development Partner · 10 January 2023Orbus Therapeutics entered an exclusive license agreement with Michigan State University and Helen DeVos Children's Hospital (part of Corewell Health) in January 2023. The agreement grants Orbus exclusive rights to intellectual property related to the method of use of eflornithine in the treatment of Bachmann-Bupp Syndrome (BABS), a rare pediatric condition. Researchers at MSU and HDVCH discovered the link between the ODC1 gain-of-function mutation and BABS, and showed that oral eflornithine improves physical coordination, attention, and interaction in affected children. Orbus plans to develop and commercialize eflornithine for BABS in parallel with its ongoing glioma program.
• Clinical Trial Centers (North America and Europe)minorOthersMore than 105 leading clinical trial centers in eight countries in North America and Europe participated in the Phase 3 STELLAR clinical trial evaluating eflornithine + lomustine in recurrent anaplastic astrocytoma patients. The Phase 1b study in newly diagnosed glioblastoma includes approximately 8 leading neuro-oncology clinical trial centers in the United States.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Emerging players

• Pharvaris: Late-stage rare disease biotech developing therapies for hereditary angioedema. Comparable as a small, focused clinical-stage rare disease company with a single late-stage asset and similar partnering/financing dynamics.
• Krystal Biotech: Rare disease biotech that launched VYJUVEK for dystrophic epidermolysis bullosa with a small specialty team. Comparable as a small-team rare disease company executing a direct specialty launch, illustrating the playbook Orbus would pursue for eflornithine in North America.

Broad incumbents

• BioMarin Pharmaceutical: Established rare disease biotech with multiple approved orphan drugs. Comparable as a model for commercializing rare disease therapies globally; also relevant as the former employer of Orbus CMO Jackie Walling.
• Ultragenyx Pharmaceutical: Commercial-stage rare disease biotech with multiple approved orphan therapies. Comparable as a rare disease commercial model with global reach and similar regulatory exclusivity-driven economics.
• Jazz Pharmaceuticals: Specialty pharma that has commercialized rare oncology and CNS products globally. Comparable as a North American rare oncology commercial model; also notable as the former employer of multiple Orbus executives including President/CEO Jason Levin and Head of Regulatory Jennifer Ekelund.
• SpringWorks Therapeutics: Commercial-stage rare oncology biotech with multiple orphan-designated programs including OGSIVEO for desmoid tumors. Comparable rare disease oncology strategy and similar regulatory pathway expertise, though at a more advanced commercial stage.

Direct peers

• Day One Biopharmaceuticals: Pediatric rare oncology company with tovorafenib approved for low-grade glioma. Closely comparable to Orbus in targeting rare CNS/pediatric brain tumors with FDA-approved orphan drug strategies and a similar small-team specialty launch model.
• Marinus Pharmaceuticals: Rare CNS disease biotech that received FDA approval for ZTALMY in CDKL5 deficiency disorder. Comparable as a small-cap rare CNS disease company pursuing orphan drug exclusivity and direct specialty commercialization.
• Kazia Therapeutics: Clinical-stage oncology biotech developing paxalisib for glioblastoma and other brain cancers. Directly comparable as a small-cap, late-stage brain tumor drug developer pursuing rare CNS oncology indications with similar regulatory and partnering pathways.

Others

• Servier Pharmaceuticals: Oncology-focused pharma that acquired Shire's oncology portfolio including ONIVYDE, which is used in CNS and other oncology indications. Relevant as a potential strategic acquirer or ex-NA commercial partner for rare oncology assets like eflornithine.

Market position

Strengths4 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/orbus-therapeutics-inc

Compliance3 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds3 records

Investors4 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 Orbus Therapeutics