Our Pipeline


Pre-clinical POC

Phase 1

Phase 2

Phase 3

Breast Cancer
Research work is ongoing on the role of SFX-01 in relation to the development of resistance to certain CDK4/6 inhibitors. Read more +
Neurodevelopmental Disorders
Evgen Pharma has licensed the global rights for lead asset SFX‐01 in neurodevelopmental disorders and schizophrenia to Stalicla Read more +
Strong preclinical data has been generated in a new solid tumour indication with further preclinical work underway and designs for a Phase Ib/IIa trial being assessed. Read more +
Preclinical data has demonstrated that SFX-01 is effective in in vitro models of rhabdomyosarcoma. Further experiments have show efficacy of SFX-01 in vivo also. Read more +
NCE: Ox1 in Addiction Anxiety/BED/AUD/impulse disorders
NCE: DAT in Long COVID/MS Fatigue
NCE: DAT in Narcolepsy

Licensing & Partnerships

Licensing Strategy
Evgen is open to out-licensing and collaboration discussions on all of its programmes and is seeking to expand its pipeline through in-licensing or partnerships.

Please contact us for more information.

Existing Partnerships

Evgen has licensed the global rights for lead asset SFX‐01 in neurodevelopmental disorders and schizophrenia to Stalicla.

Stalicla will use its proprietary precision medicine platform to identify specific phenotypes of autism spectrum disorder (ASD). Evgen and Stalicla will collaborate initially on a clinical programme in ASD, with Stalicla funding all clinical development activities.

Evgen retains the global rights for all other indications.




Evgen is seeking to expand its presence through in-licensing or partnerships. Please contact us for more information.
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Breast Cancer

Breast cancer is the largest cause of cancer deaths in women worldwide.  In around 75% of breast cancers, the hormone oestrogen plays a key part in tumour growth.

Evgen sponsored a Phase II trial [STEM – SFX-01 in the Treatment and Evaluation of Metastatic Breast Cancer] in patients with ER+, HER2-ve metastatic breast cancer (mBC).  The study enrolled 46 patients with oestrogen-positive mBC who had all previously received treatment with either tamoxifen, an aromatase inhibitor (AI) or fulvestrant. Prior to entry to the STEM trial, patients previously responded to their current hormone therapy for at least six months but then presented with progressive disease, thereby demonstrating the start of resistance to the hormone therapy.

Once entered into the trial, patients continued to receive their failing hormone therapy in addition to SFX-01 and had regular scans through to week 24. Patients discontinued the trial when one of the scans shows disease progression, or at week 24. Those patients who did not progress by week 24 were allowed to continue to receive treatment in an extension phase until disease progression.

Positive results were announced in March 2019 and the headlines were:

  1. Evidence of anti-cancer activity via tumour shrinkage and disease stabilisation.

  2. 24% of patients showed a durable clinical benefit for at least six months, despite the late stage of disease and patients’ established resistance to hormone therapy. Of these, five patients received SFX-01 for over 12 months and one patient remained on treatment for over 18 months.

  3. A favourable side effect profile for an anti-cancer drug.

See full STEM information, including posters and presentations.

Evgen is continuing investigation into how SFX-01, in combination with other treatments, can improve outcomes for patients with HR+ breast tumours that have become resistant to other therapies.  This includes research into STAT3 and pSTAT3, a protein that controls transcription of information from DNA to messenger RNA; and SHP2, a non-receptor protein tyrosine phosphatase that is associated with many cancers including breast cancer.


Strong preclinical data has been generated in a new solid tumour indication, glioblastoma (GBM), with further preclinical work underway and designs for a Phase Ib/IIa trial being assessed.

Glioma is the most common form of brain tumour affecting around five per 100,000 people. The more severe, grade IV classification, glioblastoma, is a very serious form of brain tumour representing 45% of all cases and has a poor prognosis with median survival of around 14 months. The five-year survival of the severe grades is 5%.

The data generated for SFX-01 in standard preclinical models and orthotopic models (where glioma cells are implanted in brain tissue representing a more disease-relevant model) show tumour shrinkage and significantly extended survival times. SFX-01 was also found to potentiate (i.e. substantially increase) the therapeutic effect of radiotherapy in these models.

The therapeutic options for glioma are limited to surgery, radiotherapy and the one drug widely available, temozolomide. There is a clear unmet need for more treatments for use in conjunction with the current standard of care.

The US Food and Drug Administration (”FDA”) has granted an Orphan Drug Designation (“OD”) for the use of SFX-01 to treat malignant glioma.  This is usually granted when there are fewer than 200,000 patients with a given disease in the United States and there is a scientific rationale for potential use of the product in that condition. As well as recognising the relative rareness of a disease, OD confers intellectual property cover to an investigational drug in the form of data protection at the time of approval of a new drug application, which is additional to any patent cover in force.  Tax credits are also possible on eventual US sales of an approved orphan drug.


Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in childhood, representing about 3-4% of all paediatric cancers and 50% of all soft tissue sarcoma in children. Treatment can include any combination of surgery, radiotherapy and chemotherapy, however the 5-year event-free survival rate for RMS patients with metastatic disease is about 30%.

In vitro studies demonstrated the antitumor activity of SFX-01 in two preclinical models of RMS tumours. In particular, SFX-01 treatment significantly reduced the proliferation and migration of RMS cells and enhanced the effects mediated by radiotherapy. The results suggest that the combined approach using SFX-01 and radiotherapy  might have therapeutic benefits, mainly in the clinical management of patients with aggressive RMS disease.

A poster detailing the study was presented by Dr S Camero at the ESMO Sarcoma and Rare Cancer congress in March 2023.


SFX Series: Evgen Pharma holds the exclusive worldwide rights to a range of novel sulforaphane analogues developed by the medicinal chemistry group at the University of Seville in Spain. These compounds are in preclinical investigation.

Neurodevelopmental Disorders

Autism spectrum disorder is a group of neurodevelopmental disorders (NDDs) currently diagnosed based on core behavioural features, without specific biological criteria.

Previous studies with other sources of sulforaphane have shown evidence of clinical efficacy in improving symptoms of ASD (e.g. Singh et al 2014). However, patient heterogeneity provides a challenge in identifying those individuals most likely to respond to therapy.

Stalicla specialises in the identification of specific phenotypes of autism spectrum disorder (ASD) and has a unique, proprietary technology to identify ASD patients who are most likely to respond to SFX‐01.  This screening approach has already been used successfully to identify ideal patients for other ASD drug trials and is a key differentiator for Stalicla in developing drugs for such a wide spectrum disorder as ASD.

Evgen’s partnership with Stalicla will enable the targeting of patient groups most likely to benefit from SFX‐01, not only de‐risking the clinical development but potentially bringing a therapeutic option to those individuals who are currently underserved, in a quick and efficient manner.