Q: As CSO of Mironid, what drives your passion for scientific innovation, and how do you keep your team motivated through challenges?
I’ve been a scientist for nearly 30 years, and I feel incredibly fortunate to be in a career where you learn something new every day. At Mironid, I work alongside a talented and passionate team who inspire me as much as I hope to inspire them. Our work in Autosomal Dominant Polycystic Kidney Disease (ADPKD) is particularly motivating. With this being a disease that runs in families, it carries a significant emotional burden for patients and their loved ones, making the work we do even more meaningful. A large aspect of what keeps our team engaged is the very clear link we have observed between the mechanism of action of our LoAc® molecule and the disease itself – by lowering cyclic AMP, we have the potential to make a real difference. In a small company like ours, everyone sees the direct impact of their work, learns from challenges, and grows as scientists, which I believe is truly motivating.
Q: Mironid is advancing small molecule therapeutics for the treatment of ADPKD – what makes your approach unique in the industry?
Mironid’s approach is built on deep expertise in PDE4 structure-function relationships, allowing us to precisely modulate the activity of this enzyme with small molecules. PDE4’s natural role is to degrade cyclic AMP (cAMP), a key driver of cyst formation and expansion in ADPKD. Unlike conventional approaches, our LoAc® molecules are designed to selectively increase PDE4 activity, effectively lowering the aberrantly high cAMP levels that drive disease progression. This unique mechanism offers the potential to slow disease progression, delaying or even preventing the need for dialysis or transplant. By targeting the root cause of cyst growth, we aim to provide a transformative treatment that improves the quality of life for ADPKD patients and their families.
Q: Can you share an update on Mironid’s latest advancements and their potential impact?
We are excited to have selected our lead compound for development and are on track to advance it into Phase 1 clinical trials this year. A key milestone in our progress has been the successful optimisation of urine cyclic AMP measurement as a biomarker for LoAc® response, which will be an important tool in our clinical studies. This biomarker allows us to directly assess the pharmacodynamic activity of our compounds, strengthening our ability to translate preclinical findings into meaningful clinical outcomes. As we complete the final regulatory-enabling studies ahead of our CTA submission, we are proud of the progress made and remain committed to bringing this innovative treatment to patients as soon as possible.
Q: What are the biggest challenges in developing next-generation small molecule drugs, and how is Mironid overcoming them?
Small molecules remain vital in drug development, offering the advantage of oral administration, which is particularly important for ADPKD where lifelong treatment requires a simple, patient-friendly option. A key challenge is selecting the right target. With deep expertise in PDE4 biology, we know that increasing its activity to lower cyclic AMP directly addresses ADPKD’s underlying pathology, strengthening our likelihood of success. Another challenge is ensuring that a molecule is not only effective but suitable for long-term development. The team at Mironid possesses exceptional scientific expertise, and have carefully considered factors like formulation, manufacturing, and physicochemical properties from the outset. By combining scientific insight with a focus on real-world drug development, we are advancing a small molecule therapy designed to be both impactful and accessible for patients.
Q: Looking ahead, what are the next key milestones for Mironid, and how do you see the company evolving in the coming years?
Our key priority is advancing our lead compound into the clinic, with plans to initiate Phase 1 trials this year. We are currently completing CTA-enabling studies and preparing regulatory filings, both of which are critical milestones in bringing our therapy closer to patients. Beyond our lead program, we are also developing backup molecules to support the long-term success of our approach. This continued investment in chemistry ensures a strong pipeline and reinforces our commitment to delivering impactful therapies for ADPKD.