Acuitas Therapeutics is a biotech company that develops and provides lipid nanoparticle (LNP) delivery systems to enable nucleic acid-based therapies and vaccines. Pulse 2.0 interviewed Acuitas Therapeutics President and CEO Dr. Thomas Madden to gain a deeper understanding of the company.
Dr. Thomas Madden’s Background
Screenshot
Could you tell me more about your background? Madden said:
“I have a Ph.D. in biochemistry and pursued an academic career initially, including a position as an Adjunct Professor at the University of British Columbia (UBC). Our research had clinical applications, and I had worked with different biotech companies part-time while at UBC before deciding to move full-time to industry.”
“I spent approximately fifteen years at INEX Pharmaceuticals (a biotech company I co-founded with Drs. Pieter Cullis, Michael Hope, and others) with responsibilities in various areas including IP, licensing, partner liaison, project management, technology development, CMC, and regulatory affairs.”
Formation Of The Company
How did the idea for the company come together? Madden shared:
“After leaving INEX Pharmaceuticals after a merger with another company, I founded Acuitas Therapeutics with Drs. Pieter Cullis and Michael Hope with whom I had worked for many years, both at UBC and at INEX Pharmaceuticals. We wanted to continue to develop next-generation lipid nanoparticle delivery systems for RNAi therapeutics.”
Favorite Memory
What has been your favorite memory working for the company so far? Madden reflected:
“There have been many memorable moments at Acuitas and I am particularly proud of the team we have built there and our culture. There are two particular memories which are close to my heart.”
“First when Onpattro was approved by the FDA and we celebrated this achievement with our colleagues at Alnylam Pharmaceuticals. Onpattro is used to treat individuals with a rare, genetic disease, transthyretin amyloidosis (ATTR). This disorder is fatal and symptoms generally start to arise in people in their late twenties or early thirties. As part of the celebration, our Alnylam colleagues played a video of a young woman with the disease treated by Onpattro. On camera she thanked the scientists who had developed the drug for saving her life. Although we had been aware of the disease and its consequences, this message from an actual patient had a huge emotional impact on us.”
“The second was in November 2020. We had been working incredibly hard during that pandemic year supporting our partners, BioNTech and Pfizer, in development of the mRNA COVID-19 vaccine. It was early in the morning and I was having breakfast when my wife told me to check the BBC news. The topline results of the phase 3 studies showing a response rate of 95% had just been announced. The magnitude of this efficacy was shocking, particularly in the context of the unfolding pandemic and its global impact to that point. I knew then that the end of the pandemic, while still some distance away, was within sight.”
Core Products
What are the company’s core products and features? Madden explained:
“Acuitas provides delivery technology that enables a range of new therapeutics based on nucleic acids. Our lipid nanoparticles encapsulate the nucleic acid, protecting it after administration and delivering it into cells where it works.”
Challenges Faced
Have you faced any challenges in your sector of work recently? Madden acknowledged:
“Acuitas is partly shielded from some of the challenges impacting the biotechnology sector but we see the consequences of these challenges in terms of how they affect our partners. These include issues relating to vaccine hesitancy and online misinformation, a weak financing market, and recent changes made by the US administration, which severely reduce research funding and create uncertainty around US regulatory affairs. The overall consequence of these challenges is that our partners are taking fewer products into clinical development and/or struggling to maintain their current research programs.”
Evolution Of The Company’s Technology
How has the company’s technology evolved since its launch? Madden noted:
“Acuitas invests heavily in continuous innovation. We never rest on our laurels but rather are always striving for further improvements in the potency and safety of our LNP delivery systems and new clinical applications. Recently, we have been developing targeted LNP carriers that are specifically directed to cells in the blood compartment, including T-cells. These targeted LNP have applications in the areas of cancer and auto-immune disease.”
Significant Milestones
What have been some of the company’s most significant milestones? Madden cited:
“As noted above, our most significant milestones have been the marketing approvals for Onpattro and Comirnaty. In addition, Acuitas technology supported the development of the base editing therapy used to treat baby KJ Muldoon at the Children’s Hospital of Philadelphia.”
Customer Success Stories
Can you share any specific customer success stories? Madden highlighted:
“Our success is always based on our partners’ success. Our technology enables their clinical candidates, and we jointly contribute to each positive clinical outcome.”
Total Addressable Market
What total addressable market (TAM) size is the company pursuing? Madden assessed:
“The TAM would be large depending on the number of partners we are working with and the commercial potential of each product.”
Differentiation From The Competition
What differentiates the company from its competition? Madden affirmed:
“We believe we are the global leader in LNP technology, providing the highest potency and widest therapeutic index for nucleic acid therapeutics. We also provide support and expertise across the full spectrum from preclinical development to commercialization.”
Future Company Goals
What are some of the company’s future goals? Madden concluded:
“To continue providing scientific leadership in the LNP space, expand our partner base, and enable an increasingly broad range of clinical therapeutics.”
