CLINGLIO · Project

New Drug Candidate for Deadly Brain Cancer Advancing Through Late-Stage Clinical Trials

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Brain cancer called glioma is one of the deadliest cancers, and doctors have almost nothing that truly works against it. Researchers discovered that cancer cells have a specific "switch" in their outer membrane that keeps them growing — and they designed a drug called 2OHOA that flips that switch off. Early human trials showed the drug was safe, and about half the patients who took it for more than two months showed improvement. This project ran a larger clinical trial to prove the drug works well enough for European regulators to approve it.

By the numbers

EUR 6,155,125

EU funding for Phase IIB clinical trial

Consortium partners across 8 countries

<0.5 per 10,000

Glioma prevalence in EU (orphan disease threshold)

~50%

Patients showing positive response after >2 months treatment in Phase I/IIa

SMEs in the consortium

The business problem

What needed solving

Glioblastoma is the most aggressive form of brain cancer with one of the highest mortality rates, and doctors currently have almost no effective treatments. It is classified as an orphan disease in the EU, meaning patient numbers are small but the medical need is desperate — creating a commercially attractive niche for any company that can deliver a working therapy.

The solution

What was built

The project conducted a Phase IIB clinical trial of 2OHOA (a first-in-class drug activating sphingomyelin synthase 1) in patients with newly-diagnosed glioblastoma. It also produced biomarker research for diagnosis, prognosis, and treatment response, plus a clinical trial registration (EudraCT).

Audience

Who needs this

Pharma companies seeking late-stage oncology drug candidates with orphan designationBiotech firms looking to license rare disease therapeutics with EU market exclusivityCompanion diagnostics companies developing glioma biomarker testsNeuro-oncology clinics and hospital networks treating brain cancer patientsVenture capital or pharma investors focused on orphan drug commercialization

Business applications

Who can put this to work

Pharmaceuticals

enterprise

Target: Pharma companies with oncology pipelines seeking orphan drug assets

If you are a pharma company looking to expand your oncology pipeline — this project advanced 2OHOA through a Phase IIB clinical trial for glioblastoma, the most aggressive brain cancer. The drug already has EMA orphan drug designation and a clear conditional marketing authorization pathway. With approximately half of treated patients showing positive response in the earlier Phase I/IIa trial, this represents a late-stage asset in a market with very few treatment options.

Biotech & Drug Licensing

mid-size

Target: Biotech firms specializing in CNS or rare disease drug licensing

If you are a biotech company seeking licensing opportunities in rare diseases — CLINGLIO's drug 2OHOA targets a first-in-class mechanism (sphingomyelin synthase 1 activation) with EMA orphan drug status for glioma, which affects less than 0.5 per 10,000 people in the EU. The orphan designation provides market exclusivity and regulatory incentives. The project was led by an SME (Laminar Pharmaceuticals) which may be open to partnership or licensing deals.

Diagnostic & Companion Diagnostics

any

Target: Companies developing cancer biomarker diagnostics or companion tests

If you are a diagnostics company working on precision oncology — this project investigated biomarkers for glioblastoma diagnosis, prognosis, and treatment response prediction. These biomarkers could become the basis for companion diagnostic tests that identify which patients will respond to 2OHOA treatment. The consortium of 13 partners across 8 countries generated clinical biomarker data from a multi-site Phase IIB trial.

Frequently asked

Quick answers

What would it cost to license or acquire this drug candidate?

Licensing terms would need to be negotiated with the lead SME, Laminar Pharmaceuticals (Spain). The project received EUR 6,155,125 in EU funding and has completed Phase IIB clinical trials with EMA orphan drug designation, which significantly increases the asset's value. Orphan drug status also grants regulatory fee reductions and market exclusivity upon approval.

How close is this drug to reaching the market?

The drug 2OHOA completed a Phase I/IIa trial before this project and this project conducted a Phase IIB trial. The EMA provided written guidance that conditional marketing authorization could be granted if Phase IIB demonstrates statistically significant efficacy. This means one successful trial could lead to market approval.

Who owns the intellectual property and what is the licensing situation?

The coordinating SME, Laminar Pharmaceuticals SA (Spain), discovered the drug target and designed 2OHOA. As project coordinator and the originating company, they likely hold the core IP. EMA orphan drug designation provides 10 years of market exclusivity in the EU upon approval.

Can this be scaled to treat large patient populations?

Glioma is classified as an orphan disease with less than 0.5 cases per 10,000 inhabitants in the EU, so the target patient population is small by design. However, orphan drug pricing typically compensates for smaller volumes. The mechanism of action (targeting the lipid proliferation switch) could potentially be explored for other cancer types.

What regulatory approvals has this drug already obtained?

2OHOA has received EMA orphan drug designation for glioma treatment. The EMA also provided formal scientific advice and protocol assistance (reference SA/PA-EMA/CHMP/SAWP773534/2014) confirming the pathway to conditional marketing authorization. A completed Phase I/IIa trial (NCT01792310) demonstrated safety and efficacy in humans.

What clinical evidence supports the drug's effectiveness?

In the Phase I/IIa trial, approximately half of patients with glioma who received more than 2 months of treatment showed positive response. The drug also demonstrated safety in both preclinical GLP studies and the first-in-man trial. The Phase IIB trial conducted by this project aimed to provide the definitive efficacy data required for approval.

How does this integrate with existing cancer treatment protocols?

Based on available project data, the project investigated 2OHOA's mechanism of action and explored personalized treatment design. The project also researched therapeutic alternatives for non-responding patients, suggesting the drug could be part of a broader treatment strategy rather than a standalone therapy.

Consortium

Who built it

The CLINGLIO consortium brings together 13 partners from 8 countries (Spain, France, Hungary, Israel, Italy, Netherlands, UK, US), with a strong industry presence at 38% — including 4 SMEs and 5 industry partners total. The project is led by Laminar Pharmaceuticals, a Spanish SME that originated the drug and its underlying science. With 3 universities and 4 research organizations providing clinical and scientific expertise, plus the multi-country setup needed for a credible clinical trial, this consortium is structured for real drug development rather than purely academic research. The international spread across major EU markets and inclusion of US and Israeli partners suggests global commercialization ambitions.

LAMINAR PHARMACEUTICALS SACoordinator · ES
BIOKERALTY RESEARCH INSTITUTE AIEthirdparty · ES
UNIVERSITA DEGLI STUDI DI SALERNOparticipant · IT
INSTITUT GUSTAVE ROUSSYparticipant · FR
THE ROYAL MARSDEN NATIONAL HEALTH SERVICE TRUSTparticipant · UK
UNIVERSITY OF NEWCASTLE UPON TYNEparticipant · UK
SPECIALIZED MEDICAL SERVICES-ONCOLOGY BVparticipant · NL
RESEARCH FUND OF THE HADASSAH MEDICAL ORGANIZATION (R.A)participant · IL
UNIVERSITAT DE LES ILLES BALEARSparticipant · ES
FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTAparticipant · IT

How to reach the team

Laminar Pharmaceuticals SA (Spain) — contact their business development or licensing team for partnership inquiries

Order a report on this consortium See LAMINAR PHARMACEUTICALS SA profile →

Next steps

Talk to the team behind this work.

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