Mironid Ltd.

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.

Mironid Presents Translational Data in Oral Presentation at the European Renal Association Congress LoAc® compound lowers renal cAMP after single or multiple doses and demonstrates efficacy in a preclinical model of ADPKDMironid will select a development candidate from its LoAc® programme to advance as a treatment for ADPKD 28 May 2024, Glasgow, Scotland – Mironid, a biopharmaceutical company developing small molecule therapeutics for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), a serious hereditary kidney disease, presented preclinical data on the effects of a compound from its LoAc® pipeline on a model of ADPKD on 25 May 2024 at the European Renal Association (ERA) Congress, held in Stockholm, Sweden. The presentation, by Mironid Chief Scientific Officer Dr Adele Rowley, was entitled ‘Targeting cAMP in ADPKD by small molecule activation of long form PDE4 enzymes’. ADPKD is the most common hereditary kidney disorder and affects over 12 million people worldwide. ADPKD is characterized by the uncontrolled growth of fluid-filled cysts in the kidney that can lead to kidney enlargement, loss of function, and associated complications. By activating long form PDE4 enzymes and increasing cAMP hydrolysis, LoAc®, Mironid’s first-in-class small molecules1 directly target the increased kidney cAMP levels that are known to drive cyst formation in ADPKD, resulting in decreased cyst growth in vitro and in vivo. The presentation highlighted translational data demonstrating that a LoAc® compound results in a significant reduction of cAMP levels in ADPKD models. By directly targeting cAMP, LoAc® compounds have the potential to slow renal disease progression, and to delay or prevent the need for dialysis, offering a potential new treatment option for ADPKD patients. Mironid is working to select a development candidate from its LoAc® programme to advance as a treatment for ADPKD. Omar, F., et al. (2019) Small-molecule allosteric activators of PDE4 long form cyclic AMP phosphodiesterases. PNAS 116(27): 13320-13329 -ENDS- Enquiries:Mironidenquiries@mironid.com ICR ConsiliumSukaina Virji / Lindsey Neville / Evi UsehMironid@consilium-comms.com About MironidMironid is a biopharmaceutical company developing small molecule therapeutics for the treatment of life-threatening hereditary kidney disease. Its lead programme is for Autosomal Dominant Polycystic Kidney Disease (ADPKD), which is characterized by uncontrolled growth of fluid-filled cysts and is the most common hereditary kidney disorder, but has limited treatment options. Mironid’s first-in-class small molecule LoAc® drug candidates directly target the abnormally high kidney cAMP levels that drive cyst formation. The Company is led by an industry-experienced management team and supported by a strong advisory network and blue-chip investors including Roche Venture Fund, Epidarex Capital, Sofinnova Partners and BioGeneration Ventures. The University of Strathclyde, Scottish Investment Bank and the European Investment Fund (EIF) are also investors in Mironid.

Mironid appoints Dr Catherine Kelleher as Chief Medical Officer October 24 2023, Glasgow, Scotland – Mironid, a biopharmaceutical company developing small molecule therapeutics for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), a serious hereditary kidney disease, today announces the appointment of Dr Catherine (Cass) Kelleher as Chief Medical Officer. Dr Kelleher is a physician with more than 14 years experience in the biopharmaceutical industry and a further 14 years in academic medicine as an internist, nephrologist and geriatrician. She started her industry career at Amgen and has worked in multiple senior positions in drug development including nephrology and rheumatology. Cass most recently served as Chief Medical Officer at Anteris Bio and previously as Senior Vice President of Clinical Development at ChemoCentryx. Prior to joining industry, Cass was Associate Professor of Medicine at the University of Colorado. “Mironid has the potential to improve the ADPKD disease space by offering a first-in-class, innovative molecule to treat patients with ADPKD and possibly other rare diseases,” commented Dr Cass Kelleher, Chief Medical Officer of Mironid. “I look forward to helping make this a reality, working closely with the executive team at Mironid as we work to progress our treatment into the clinic.” “I am delighted to welcome Cass as Chief Medical Officer to the team here at Mironid,” commented Dr Neil Wilkie, Chief Executive Officer of Mironid. “Her extensive experience in drug development in rare diseases, as well as her broad medical and scientific knowledge, make her perfectly suited for this position. We continue to build a strong team with the shared goal of delivering a safe, well-tolerated and effective treatment option for all ADPKD patients.” -ENDS- Enquiries: MironidNeil Wilkie, Chief Executive Officerenquiries@mironid.com ICR ConsiliumMary-Jane Elliott / Sukaina Virji / Lindsey NevilleMironid@consilium-comms.com About MironidMironid is a biopharmaceutical company developing small molecule therapeutics for the treatment of life-threatening hereditary kidney disease. Its lead programme is for Autosomal Dominant Polycystic Kidney Disease (ADPKD), which is characterized by uncontrolled growth of fluid-filled cysts and is the most common hereditary kidney disorder, but has limited treatment options. Mironid’s first-in-class small molecule LoAc® drug candidates directly target the abnormally high kidney cAMP levels that drive cyst formation. The Company is led by an industry-experienced management team and supported by a strong advisory network and blue-chip investors including Roche Venture Fund, Epidarex Capital, Sofinnova Partners and BioGeneration Ventures. The University of Strathclyde, Scottish Investment Bank and the European Investment Fund (EIF) are also investors in Mironid.