Cornerstone Pharmaceuticals

What Cornerstone Pharmaceuticals does

Cornerstone Pharmaceuticals is a privately held, clinical-stage biopharmaceutical company developing metabolism-directed anti-cancer therapies for rare and hard-to-treat cancers. The company's lead compound, CPI-613® (devimistat), is a first-in-class small molecule designed to selectively target mitochondria in cancer cells, disrupting energy production to deprive tumors of metabolic fuel while sparing normal tissue. Cornerstone also previously held CB-839 (telaglenastat), a glutaminase inhibitor, but sold all rights to Synhale on February 5, 2025, consolidating the pipeline around devimistat. CPI-613 has been tested across multiple cancer indications — including pancreatic cancer (AVENGER 500 Phase 3, fully enrolled August 2020), acute myeloid leukemia (ARMADA 2000 Phase 3), Burkitt's lymphoma, biliary tract cancer, soft tissue sarcoma, MDS, peripheral T-cell lymphoma, and clear cell sarcoma — and has received seven FDA Orphan Drug Designations, two FDA Fast Track Designations, and four EMA Orphan Drug Designations.

The company operates an enterprise-focused go-to-market built around investigator-initiated and sponsor-led clinical trials conducted at premier cancer centers, including Memorial Sloan Kettering, MD Anderson, City of Hope, and Massachusetts General Hospital. Cornerstone is pre-revenue; the future revenue model is structured around pharmaceutical development, out-licensing of regional rights (notably the 2019 agreement with Ono Pharmaceutical covering Japan and other Asian countries), and eventual commercialization royalties — no product is currently sold. Founded in 2009 (originally as Rafael Pharmaceuticals, renamed back to Cornerstone in May 2022), the company is led by CEO John Goldberg, MD (appointed March 26, 2024) and Chairman Howard S. Jonas, founder of IDT Corporation. Cornerstone is headquartered at 9 Deerpark Drive, Suite J-5, Monmouth Junction, New Jersey, and operates with 11–50 employees.

Name

Cornerstone Pharmaceuticals

Legal name

Cornerstone Pharmaceuticals, Inc.

Website

https://cornerstonepharma.com

Company type

Private

Founded year

2009

Operating status

Operating

Headcount range

11–50 employees

Short description

Cornerstone Pharmaceuticals is a privately held, clinical-stage biopharmaceutical company developing CPI-613 (devimistat), a first-in-class mitochondria-targeting anti-cancer compound, for rare and hard-to-treat cancers including pancreatic cancer and AML; it is pre-revenue with a pipeline focused on out-licensing and future commercialization.

Ownership category

akta.pro rank

Product category

Oncology Pharmaceuticals

NAICS

Pharmaceutical Preparation Manufacturing (325412)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Oncology Specialty Pharmaceuticals (HLAIACAA)

akta.pro secondary industry

CMC & Pharmaceutical Development (Formulation/Process/Analytical Development) (HLAIALAD)

Keywords

• Metabolic oncology therapy
• Cancer drug development
• Clinical stage biotech
• Orphan drug therapy
• Mitochondria targeting drugs

Headquarters

HQ city

Cranbury

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, Marketing or Sales

Revenue model

1. Pharmaceutical Development and Licensing: Pre-revenue biotech company focused on developing cancer therapies. Revenue will come from drug development and eventual commercialization of CPI-613 (devimistat) for multiple cancer indications.
2. Out-licensing Agreements: Company has entered into out-licensing agreements for drug development in specific geographic regions, such as the agreement with Ono Pharmaceutical for Japan and other Asian countries.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Cornerstone Pharmaceuticals is a clinical-stage biopharmaceutical company developing CPI-613 (devimistat), a first-in-class metabolism-directed anticancer compound designed to selectively target cancer cell mitochondria to disrupt tumor energy production. The drug is being tested across multiple rare and refractory cancer indications, including acute myeloid leukemia, pancreatic cancer, Burkitt's lymphoma, biliary tract cancer, soft tissue sarcoma, MDS, and peripheral T-cell lymphoma, supported by FDA Orphan Drug, FDA Fast Track, and EMA Orphan Drug designations. The company conducts its clinical program through partnerships with leading oncology research institutions and out-licenses regional rights to commercial partners such as Ono Pharmaceutical.

Product overview

Cornerstone Pharmaceuticals is a biopharmaceutical company focused on metabolic oncology. The company is developing CPI-613® (devimistat) as its lead drug candidate - a mitochondria-targeting compound designed to disrupt cancer cell energy production. The company previously held CB-839 (telaglenastat), a glutaminase inhibitor, but sold all rights to Synhale in February 2025. The therapeutic approach centers on exploiting metabolic differences between cancer cells and normal tissues.

Differentiator

Problem solved

Functional benefit

Products and services

• CPI-613® (devimistat) Cornerstone's lead compound, a first-in-class metabolism-directed anticancer agent designed to selectively target cancer cell mitochondria to disrupt energy production, intended for use in recurrent and hard-to-treat cancers including AML, pancreatic cancer, Burkitt's lymphoma, biliary tract cancer, soft tissue sarcoma, MDS, and peripheral T-cell lymphoma. Held under FDA Orphan Drug, FDA Fast Track, and EMA Orphan Drug designations; administered in clinical trial settings.

Quantifiable outcome

• FDA Orphan Drug Designation received for multiple indications including Burkitt's lymphoma, AML, pancreatic cancer, biliary tract cancer, soft tissue sarcoma, MDS, and peripheral T-cell lymphoma
• +2 more outcomes

Named customers4 records

Segments1 record

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Ten partnerships are on record, tiered core and minor.

• SynhalecoreOEM/ Whitelabel/ Licensing Partner · 5 February 2025Cornerstone sold all rights to Telaglenastat (CB-839) to Synhale. This divestiture transferred all development and commercialization rights for this asset.
• Sara's Cure and SARCcoreStrategic or Co-development Partner · 1 December 2020Partnership for the launch of Phase 2 clinical trial for CPI-613 in combination with hydroxychloroquine for patients with clear cell sarcoma of soft tissue.
• Roswell Park Comprehensive Cancer CentercoreStrategic or Co-development Partner · 5 May 2020Research collaboration to evaluate the effects of CPI-613 on esophageal cancer. Joint research initiative combining Cornerstone's drug compound with Roswell Park's research capabilities.
• Pancreatic Cancer Action Network (PanCAN)minorGTM or Marketing Partner · 7 November 2019Joined PanCAN's Scientific & Medical Affairs Industry Membership to support pancreatic cancer research and patient advocacy.
• National Institutes of Health (NIH)coreStrategic or Co-development Partner · 17 October 2019Collaboration to test CPI-613 in cytokine independent (acute) HTLV-1 associated adult T-cell leukemia/lymphoma.
• Ono PharmaceuticalcoreOEM/ Whitelabel/ Licensing Partner · 25 June 2019Out-licensing agreement to accelerate clinical development and commercialization of CPI-613 in Japan and other Asian countries. Cornerstone granted Ono Pharmaceutical rights to develop and commercialize the drug candidate in the licensed territory.
• Stand Up To CancercoreStrategic or Co-development Partner · 7 February 2019CPI-613 selected to be explored as part of Stand Up To Cancer's T-Cell Lymphoma Dream Team Research Grant for new combination treatment.
• Atlantic Health SystemcoreStrategic or Co-development Partner · 16 November 2017Collaboration for Phase I study of CPI-613 in combination with gemcitabine and nab-paclitaxel for patients with locally advanced or metastatic pancreatic cancer.
• Wake Forest UniversitycoreStrategic or Co-development Partner · 19 April 2010Partnership for clinical trial of CPI-613 for hematologic malignancies.
• U.S. National Cancer InstitutecoreStrategic or Co-development Partner · 22 March 2010Collaboration agreement for evaluation of Emulsiphan (company's drug candidate) in cancer treatment research.

Scale indicators4 records

Recent moves7 records

Expansion highlights4 records

Others

• Rafael Holdings: Former parent/related entity of Rafael Pharmaceuticals (now Cornerstone). Connected through shared leadership (Howard Jonas as Chairman) and history, with current CMO of Rafael Holdings serving as Cornerstone's CEO - representing an ecosystem relationship.
• Ono Pharmaceutical: Existing licensing partner for CPI-613 in Japan and other Asian countries. Connected through strategic partnership and would be comparable as both hold rights to Cornerstone's lead asset in different geographies.

Broad incumbents

• AstraZeneca: Global oncology leader with established AML franchise (e.g., azacitidine combinations) and pancreatic cancer programs. Comparable as a major player in Cornerstone's target indications and potential strategic acquirer given metabolic oncology interest.
• Merck & Co. Major oncology player with leading PD-1 inhibitor (Keytruda) used across multiple cancer indications. Comparable as a potential combination therapy partner for CPI-613 given extensive clinical infrastructure in oncology indications Cornerstone targets.
• Bristol Myers Squibb: Major oncology incumbent with extensive AML and pancreatic cancer franchises (e.g., azacitidine, nab-paclitaxel). Comparable as both compete in Cornerstone's lead indications and would be potential acquirers or partners if Phase 3 trials succeed.
• Servier Pharmaceuticals: Established oncology company with IDH-targeted therapies (vorasidenib, ivosidenib) for AML and other cancers. Comparable as a metabolic oncology incumbent and potential strategic acquirer or combination partner for CPI-613 programs.

Direct peers

• Calithera Biosciences: Cancer metabolism-focused biotech that originally developed CB-839 (telaglenastat) before Cornerstone acquired rights. Directly comparable as a small-cap metabolic oncology company with pipeline assets in glutaminase inhibition and tumor metabolism.
• Agios Pharmaceuticals: Clinical-stage biopharmaceutical company focused on cellular metabolism and oncology. Direct peer as both companies pursue metabolism-targeted cancer therapeutics, with Agios's IDH inhibitors (vorasidenib, ivosidenib) targeting similar metabolic vulnerabilities in cancer cells.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat3 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/CornerstoneInc_
• Facebookfacebook.com/CornerstonePharmaceuticals
• LinkedInlinkedin.com/company/cornerstonepharma

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Funding overview

Funding rounds

Investors

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 Cornerstone Pharmaceuticals