Scottish biopharmaceutical company Mironid has pocketed $46m in Series B funding raise to progress the development of its lead rare kidney disease candidate.
The financing, which was contributed to by Roche Venture Fund and Sofinnova Partners, as well as the Scottish National Investment Bank, Epidarex Capital and BioGeneration Ventures, will help Mironid progress its lead small molecule autosomal dominant polycystic kidney disease (ADPKD) closer to the clinic.
Following the extension of its Series A raise in 2023, Mironid’s CEO, Neil Wilkie, tells Pharmaceutical Technology that the company has delivered on the objectives of the raise by completing investigational new drug (IND)-enabling studies and expanding its patent portfolio. Now, with new capital in hand, Wilkie says that the company should be set cash-wise through to the generation of initial clinical data.
Mironid progresses its pipeline as ADPKD impacts around 12.4 million individuals worldwide – making it the most common inherited kidney disorder, despite its status as a rare disease. The condition, which is primarily caused by mutations in the PDK1 or PDK2 genes, is characterised by the uncontrolled growth of fluid-filled cysts on the kidney, leading to kidney failure in as many as half of those suffering from the disease by the age of 60.
Treating ADPKD: the challenges and opportunities
Currently, the only available targeted treatment option for ADPKD is Otsuka Pharmaceutical’s Jynarque (tolvaptan), which secured approval for use in the condition back in 2018. However, there are several side effects associated with the drug, including excessive thirst, frequent urination and potential severe liver damage – meaning some patients may choose to discontinue treatment.
Unlike Jynarque, which acts as a selective vasopressin V₂ receptor antagonist, Mironid’s lead candidate targets cyclic AMP (cAMP), a secondary messenger molecule that plays a key role in regulating physiological processes in the body. When cAMP is activated at abnormally high levels, it can also play a significant role in all stages of ADPKD, Wilkie says.
By designing a small molecule that can activate the degradation of cAMP through the activation of long-form PDE4 enzymes, Mironid hopes that its lead candidate could curb both new cyst formation and existing cyst growth – addressing one of the root causes of the disease.
Mironid also proposes that the drug may have a better side effect profile than Jynarque, potentially offering patients a more tolerable treatment option. This sentiment is backed by preclinical data, with the company claiming that studies have demonstrated the drug’s potential to reduce the number of cysts and kidney volume, while exhibiting a favourable safety profile.
Abnormally elevated levels of cAMP are not just found in ADPKD, Wilkie adds, as several other diseases are also characterised by heightened cAMP signalling. This means that Mironid’s approach could hold potential across both other kidney diseases and conditions in different disease areas – something Wilkie says the company is currently investigating.
