NeoTargets
NeoTargets – Providing targets and drugs with a curative potential for targeted treatments of cancer.
Background
The survival rate for a broad range of cancers remains unacceptably low. In addition, 97% of clinical studies against cancer fail, mainly because of high toxicity and low efficacy of the drug candidate. A major reason behind this low success rate in clinical studies can in most cases be traced back to poor assessment during preclinical research. There is a lack of systematic assessment of drug targets, and routinely model systems with low predictive value that are insufficient to assess the efficacy of targets and drugs are engaged.
At NeoTargets we can increase the probability that candidates selected will not fail in clinical studies, by selecting the best targets against the disease and followingly validate them before the clinical studies are initiated, thereby saving precious time, money and ultimately also lives.
The team behind NeoTargets has long-standing expertise in cancer biology, drug target discovery and drug discovery with a proven ability to bring successful projects to the clinics. We have established a pioneering oncotarget discovery platform, consisting of an in-silico target assessment tool for systematic identification and prioritisation of targets with optimal characteristics (e.g. “druggability” and selectivity against cancer cells).
Selected candidate targets are functionally validated in a stepwise fashion on:
1. Relevant cancer cells.
2. Translational cell models for toxicity.
3. Well-characterised patient samples, including cancer-initiating cell populations.
4. Patient-derived xenograft (PDX) mouse models.
Validated targets are further investigated for mechanisms of action and potential molecular biomarkers.
We will provide validated targets and preclinical drugs with proven efficacy against primary cancer cells, including cancer-initiating cells but display minimal effects on vital tissues. In this way we can provide targets and preclinical drugs with a curative potential that can be used for development of new targeted treatments.
At NeoTargets, we also have our own pipeline of preclinical drug development program.
Vision, mission and strategy
Mission
Our mission is to deliver optimal therapeutic targets and to develop drugs with a curative potential for the treatment of cancer.
Vision
Our vision is to become a leading developer of targeted and effective therapeutic approaches for patients with unmet medical needs.
Strategy
Our strategy is to identify and validate optimal therapeutic targets via our pioneering oncotarget discovery platform. Validated targets will form the basis for preclinical drug development projects that will be conducted by NeoTargets or via partners and collaborators. Commercialisation of drug targets and preclinical drugs will be pursued via R&D collaboration agreements with established drug companies or via sales and licensing agreements of validated drug targets or drugs.
Research and Technology
Introduction
At NeoTargets, we have established a unique oncotarget discovery platform. It consists of a unique proprietary bioinformatic tool that is used for identification of optimal candidate targets followed by functional validation of the targets in translational model systems, including toxicity models, patient samples and animal models.
01
The in-silico target assessment tool
About 15% of all clinical trials are terminated due to target toxicity from harmful targets. At NeoTargets, we have developed a unique in silico assessment tool and identified cancer-specific and safe targets with optimal characteristics for more than 130 different cancer types. By comparing data from our targets with clinical data, we can completely eliminate all harmful targets. This is significantly better than any other available tools we have tested to date (e.g. DepMap).
02
Validation of cancer dependencies
Firstly, identified candidate targets are validated for their importance in different relevant cancer cell lines for the indication of interest. Typically, more than 90% of the candidates pass this validation step.
03
Functional assessment of toxicity in normal healthy cells
Toxicity in vital organs such as cardiovascular, liver, hematologic and the nervous system is a major challenge in cancer treatment. Thus, to minimise the risk for toxicity in downstream drug testing as early as possible, all targets are investigated for harmful effects in normal blood cells, cardiomyocytes, hepatocytes, and neurons.
04
Validation of targets using primary cancer cells
The targets will be validated for their importance in well-characterised patient samples, allowing identification of targets that can be used for battling specific subtypes of cancer (e.g. P53 mutated cancer). Strategies targeting cancer driving cells have a curative potential and therefore targets that can be used to eradicate this type of cells will be prioritised.
05
Validation of candidate targets in xenograft animal models
To determine if inhibition of the targets can prevent disease progression and increase survival rates, they will be validated using patient-derived xenograft (PDX) mouse models.
06
The molecular mechanisms behind the selective importance in cancer cells
The final candidates will be investigated for mechanisms of action and predictive markers, using functional genomics.
07
Projects and therapeutic areas
Collaboration projects
Acute myeloid leukemia
Acute myeloid leukemia (AML) is an aggressive haematological disorder with dismal prognosis. The standard treatment for AML is chemotherapy but this regimen causes severe side effects and have low curative potential. In collaboration with the Swedish drug development company Sprint Bioscience, we are validating optimal therapeutic targets for AML using our technology platform.
Pipeline of projects available for partnerships
Multiple myeloma
Multiple myeloma is an incurable blood malignancy will low life expectancy. The main treatment consists of proteasome inhibitors and chemotherapies, and bone marrow transplantation. All treatments are associated with severe side effects.
In this project, 25 promising target candidates have been identified and are currently being validated using our technology platform.
Selected characteristics of the candidate drug targets:
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All targets are required for viability of multiple myeloma cells but not for normal cells.
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One third of the targets are linked to poor overall survival when the target gene is overexpressed in multiple myeloma.
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50% of the targets have an available high-resolution crystal structure and several with substrates or investigational compounds.
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Investigational drugs or molecules are available for more than half of the targets.
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Before filtering out the most optimal targets, approximately 20% of them have been enrolled in clinical trials, suggesting that the selection criteria of the candidates are valid.
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Several of the targets are linked via physical interactions, components of the same pathways and display functional correlations, etc.
Glioblastoma multiforme
Glioblastoma multiforme is a deadly brain cancer and only 3-5% of the patients survive more than a year. The treatments are inefficient, and approximately 200 000 deaths are reported per year.
In this project, 25 promising candidates have been identified and will soon be validated in patient-derived cancer cells and animal models.
Nondisclosed projects
Other cancer types than the above with unmet medical needs with existing translational model systems.
NeoTargets is continuously broadening the portfolio of indications and additional projects are currently being added to the pipeline.
Collaborations and partnerships
To secure a professional management and further successful development of the company, NeoTargets has partnered with the Danish company, Marigold Innovation. By merging the senior biotech executive team from Marigold with the scientific group from NeoTargets, we have established a dynamic group with complementary competences and strong execution power.
Research collaborations and partnerships are essential in successful R&D, and a central part of NeoTargets’ strategy to maximise quality, progress, and commercial goals for our projects. In addition to our expertise, well-established model systems, and unique technology, NeoTargets collaborates closely with various researchers, partners, and clinicians for complementing biological and clinical expertise as well as state-of-the-art model systems with high translational value.
NeoTargets has an ongoing research collaboration project in acute myeloid leukemia with the Swedish listed drug development company, Sprint Bioscience.
Business model
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