Pipeline
eRapa
eRapa is a proprietary oral tablet formulation of rapamycin (sirolimus). Rapamycin is an mTOR (mammalian Target Of Rapamycin) inhibitor. mTOR is known to have a significant role in the signalling pathway that regulates cellular metabolism, growth and proliferation and is activated during tumorgenesis.
Tolimidone
Tolimidone is an orally delivered, potent and selective activator of lyn kinase. It demonstrates glycemic control via insulin sensitization in animal models of diabetes and has the potential to become a first in class blood glucose modulating agent.
Familial Adenomatous Polyposis
FAP is characterized as a proliferation of polyps in the colon and/or rectum, usually occurring in mid-teens. There is no approved therapeutic option for treating FAP patients, for whom active surveillance and surgical resection of the colon and/or rectum remain the standard of care. If untreated, FAP typically leads to cancer of the colon and/or rectum. There is a significant hereditary component to FAP with a reported incidence of one in 5,000 to 10,000 in the US and one in 11,300 to 37,600 in Europe. eRapa has received Orphan Designation in the US with plans to seek such designation in Europe. Importantly, mTOR has been shown to be over-expressed in FAP polyps – thereby underscoring the rationale for using a potent and safe mTOR inhibitor like eRapa to treat FAP.
The company’s lead programme, eRapa, is being readied for a phase 3 registration study.
Type 1 Diabetes
Type 1 diabetes, also known as juvenile diabetes, is a chronic metabolic disorder that affects individuals of all ages. This autoimmune disease occurs when the immune system mistakenly attacks and destroys the insulin-producing β cells in the pancreas, leading to insufficient insulin production. Without adequate insulin, glucose cannot enter the body’s cells for energy, resulting in high blood sugar levels. Type 1 diabetes requires daily insulin injections or the use of an insulin pump to maintain normal blood sugar levels. Individuals with this condition must carefully monitor their blood glucose levels, consume a balanced diet, and engage in regular physical activity to effectively manage their diabetes and prevent complications.
Gastrointestinal Stromal Tumours (GIST)
Gastrointestinal stromal tumors (GIST) are rare cancers that arise from the interstitial cells of Cajal or related stem cells in the wall of the gastrointestinal tract and are classified as mesenchymal, nonepithelial tumors. They account for less than 1% of all gastrointestinal tumors but are the most common mesenchymal tumors of the GI tract. Globally, GIST has an estimated incidence of roughly 10–15 new cases per million people per year (about 1–1.5 per 100,000), translating in the United States to approximately 4,000–6,000 newly diagnosed adults annually. GIST can occur anywhere along the digestive tract but most often develops in the stomach (around 60–65% of cases) and small intestine (about 25–35%). It typically affects adults between 50 and 70 years of age, with incidence rising with age and peaking in people over 70 years. Despite its rarity, the recognition of GIST has increased substantially over the past two decades with advances in diagnostic pathology and the development of targeted therapies, which have improved outcomes for many patients.
Diffuse Midline Glioma
Paediatric Diffuse Midline Glioma (DMG), K27M-mutated (previously known as Diffuse Intrinsic Pontine Glioma – DIPG) is a rare malignant tumour primarily located in the pontine and thalamic areas of the brain. DMG affects children of all age with an incidence of 1-2 cases per 100,000 population though it mainly is diagnosed between the ages of 5 and 7. It makes up to 15% of all brain tumours in children and up to 80% of all paediatric gliomas. The prognosis of DMG is dismal due to the absence of effective therapies. After diagnosis, median survival is usually nine months. Only 10% live for more than two years.
Current treatment options include radiotherapy (either conventional fractionated or hypofractionated), re-irradiation upon progression and palliative symptomatic treatment. Despite numerous treatments in development, none have so far demonstrated significant extension of survival in these patients.
Glioblastoma
Glioblastoma (GBM) is the most common and devastating primary malignant brain tumour in adults encompassing close to 15% of all primary brain and central nervous system neoplasms and over 50% of all malignant brain tumours. With an incidence of approximately 2-4 per 100,000 population in both the EU and USA, over 250,000 new cases of GBM are diagnosed per annum worldwide. Standard of care for treatment of primary GBM is typically maximal surgical resection followed by radiotherapy plus concomitant and maintenance temozolomide chemotherapy with or without the Optune® device. Notwithstanding, the multidisciplinary approach, almost all patients experience tumour progression with nearly universal mortality. The median survival from initial diagnosis is less than 21 months.
Medulloblastoma
Medulloblastoma is the most common malignant tumour in children and accounts for approximately 25% of all primary brain tumours. The tumour originates from embryonic cells and is a grade III-IV one, according to the latest WHO classification. Medulloblastoma is classified based on the presence of mutations into 4 groups: WNT-activated, SHH-activated, group 3 and group 4. Classifying individual tumours is important as this has significant impact on the prognosis level with WNT-activated having the mildest course and group 3 being the most aggressive, often manifesting with tumour spread into the leptomeningeal space. Medulloblastoma treatment consists of maximal safe resection, followed by radiotherapy in different modalities, chemo- and targeted therapy and stem cells treatment. Survival prognosis depends on group classification, age, level of spreading and ranges from 30 to 85% at 5 years. Upon recurrence, there is no standard treatment and survival rate is poor, usually less than a year.