Q&A with Dr Sinead Blaber, Director of Clinical Development and Medical Affairs at Regeneus

Q. What is your background and experience in the regenerative medicine and stem cell space? How did this lead you to Regeneus?

A. I started my PhD in 2009, investigating the mechanisms of bacterial spore formation with the overall aim to identify new antibiotic targets. My husband worked in the lab next door and his supervisor was branching out into the regenerative medicine space and had just co-founded Regeneus.

Through discussions with this group, I quickly became fascinated with how stem cells work and the therapeutic potential of the regenerative medicine space. Ultimately, I transferred my PhD and completed it in conjunction with Regeneus. My PhD involved investigating the therapeutic adipose-derived stem cells and their secretions. I subsequently took up a position as a Research Fellow, acting as a bridge between the University-based research and Regeneus, before branching into the clinical research space at the company.

Q. Tell us about your current role as the Director of Clinical Development and Medical Affairs at Regeneus?

A. I am very excited to have rejoined Regeneus at such a critical time in the company’s development. My role is multi-factorial as it covers both clinical development and medical affairs.

On the clinical development side, my role involves designing and executing the strategic clinical development programs for both Progenza™ and Sygenus. This involves leading high quality pre-clinical and clinical studies to support their development and interacting with regulatory bodies in jurisdictions of interest.

On the medical affairs side, my role involves being the link between Regeneus and the wider medical community. This includes representing Regeneus at a variety of forums, such as conferences, and establishing and managing relationships with external key opinion leaders. Regeneus has numerous collaborations with experts at the University of Adelaide, Monash University, the University of New South Wales, the University of Technology Sydney and the Agency for Science, Technology and Research in Singapore. These collaborations are valued by Regeneus and contribute to building the knowledge base on our products.

Q. You were previously employed at Regeneus in a scientific and clinical research capacity, including leading the Phase I Progenza™ STEP Trial. Why did you decide to return to the company?

A. I joined Regeneus when it was a small company and worked there for over 8 years in multiple roles across the scientific and clinical research areas. In that time, the team grew substantially and achieved significant milestones in developing and commercialising autologous cell therapies for animals and humans.

During this time, I led the Phase I Progenza™ STEP Trial which met its primary endpoint of safety and showed very encouraging signs of efficacy, including pain relief and halting cartilage degradation in patients with knee osteoarthritis. These were very exciting results for the entire Regeneus team. There are no disease-modifying treatments available for osteoarthritis. so our Phase I trial results highlighted the potential of Progenza™ to be the first disease-modifying treatment for this condition.

In the years I spent away from Regeneus, I gathered experience not only in running Phase I and II clinical trials, but also in the overall strategy for developing different therapeutic modalities, including in the scientific, clinical, regulatory, intellectual property and commercialisation areas across multiple indications.

Given the additional experience I had gained outside of Regeneus, – coupled with my knowledge of the potential of Progenza™ and Sygenus – I decided to rejoin the company. I believe that our products can make a difference to patients and I wanted to apply my skills to develop and deliver Progenza™ and Sygenus to patients in need. I am passionate about developing therapeutics with the potential to help patients and rejoin the company.

Q. What do you believe is next for regenerative medicine and what role can Regeneus’ technology play?

A. In recent years there has been an explosion in the regenerative medicine space with many cell and gene products being developed for a variety of indications. Additionally, regulatory agencies have become much more familiar with regenerative medicines and recognize their potential resulting in initiatives such as the Japanese regulatory body, the PMDA, offering an accelerated approval pathway for cell therapies.

Since I left Regeneus after the completion of the Phase I Progenza™ STEP trial, the company has made significant progress through the Progenza™ licence and collaboration agreement with Kyocera, which highlights the significant commercial value and confidence in our technology. This agreement provides a clear commercialisation pathway for Progenza™ in osteoarthritis. Approximately 25.6 million people in Japan suffer from osteoarthritis with the market expected to grow to US$350 million by 2026 in Japan and US$3.5 billion globally.

Given our positive safety and efficacy results in the Phase I STEP trial, coupled with the Kyocera agreement, Progenza™ is well placed to change patients’ lives through pain relief and halting cartilage degradation associated with osteoarthritis, a serious condition with an unmet medical need, given as yet no disease-modifying treatments are available.

Q. What are you most excited about for Regeneus’ technology and as a company? How does your role help support this?

A. Progenza™ is a unique cell therapy product. One of the ways mesenchymal stem cells (MSCs) exert their therapeutic effect is through the secretions they produce. These secretions include a mixture of factors that act on multiple cells in the body, including immune cells and cartilage cells within a joint. These secretion factors can shift the pro-inflammatory environment commonly seen in osteoarthritic joints to an anti-inflammatory environment conducive with repair, regeneration and a reduction in pain. MSCs continue to secrete these factors once they are administered to a patient.

Unlike other stem cell therapies in development, Regeneus freezes the stem cells with their secretions. We have found that in doing so, the stem cells have enhanced viability and functionality upon thawing. This means the stem cells in our Progenza™ product when thawed are healthier and can exert their therapeutic effect quickly when introduced into a patient.

Sygenus contains just the secretion factors produced by stem cells and is being developed as a topical product for the treatment of pain and inflammatory conditions. We have shown positive safety and efficacy results in human pilot studies in conditions such as acne.

Regeneus has significant potential and is well placed to deliver Progenza™ to Japanese osteoarthritis patients in collaboration with Kyocera. However, there is also potential for Progenza™ beyond the Japanese market and for Sygenus in a variety of pain and inflammatory indications. My role involves establishing and maintaining partnerships with collaborators to continue to build on the knowledge base for our unique products, coupled with driving strategic clinical development of our technology to support their commercialisation in markets and indications of interest.