Causeway Therapeutics is a clinical-stage biotech company developing novel microRNA-based therapies — principally their lead candidate, designed to repair tendon structure, relieve pain, and restore function in chronic tendon disease. Pulse 2.0 interviewed Causeway Therapeutics CEO and Executive Chairman Declan Doogan to gain a deeper understanding of the company.
Declan Doogan’s Background
Could you tell me more about your background? Doogan said:
“I graduated from Glasgow University Medical School and later went into Pharma. I spent 25 years at Pfizer. Early in my career, I oversaw the development of Zoloft, and later I was the Head of Worldwide Development. After leaving Pfizer, I decided to get involved in a start-up biotech. I served as Head of R&D and acting CEO of Amarin (AMRN: Nasdaq) and then went on to co-found Biohaven (BHVN: NYSE). I found that I enjoyed building teams. I also understood the perils of falling in love with the science when you are too close. You have to step back and look dispassionately at the data and the likelihood of translational success as well as commercial reality.”
Formation Of The Company
How did the idea for the company come together? Doogan shared:
“As multi-pathway regulators, we know that microRNA-based therapies are ideally suited for the treatment of complex multi-genic disorders, where conventional single-target therapies have failed. Derek Gilchrist and Neal Miller had made the discovery that microRNA-29a was disordered in tendon disease and that they could design a synthetic oligonucleotide mimic of microRNA-29a that could restore the levels in impacted tendons in mice and horses.”
“The robustness of the translational story gave some level of confidence in the possibilities of the same effects in humans. I initially joined as an advisor, having known the team through my affiliations with Glasgow University Medical School and my experiences within the local scientific community. We identified grant funds to support research around an asset based on the discovery, which is now called TenoMiR.”
“We then had to raise the capital for the large clinical proof of concept study. I was optimistic and we were able to form a syndicate of investors to fund this. Once we brought in new blood with commercial drug expertise we could begin to think of the evolution of the company and invested in commercial analyses that showed the massive unmet medical need that exists in tendon disease, affecting millions of patients with very few therapeutic options.”
Favorite Memory
What has been your favorite memory working for the company so far? Doogan reflected:
“Seeing the Phase 2 results emerge for TenoMiR in lateral epicondylitis (LE). On the surface, it looked mixed because we missed our endpoints. After the team further analyzed the data, it revealed terrific efficacy in patients that appropriately received it. This reassured us that the other animal data lined up well with the clinical data and that it was not a fluke. This is the underpinning for the next phase, which will include further trails of TenoMiR in LE and for the rotator cuff of the shoulder.”
Core Products
What are the company’s core products? Doogan explained:
“Our lead program is TenoMiR. TenoMiR is a chemically synthesized mimic of miR-29a, developed to target the underlying molecular cause of tendon disease, not just alleviate symptoms, by stimulating natural healing. TenoMiR is administered via intratendinous injection.”
“We recently completed a phase 2 study of TenoMiR in patients experiencing lateral epicondylitis (LE), commonly known as tennis elbow.”
“The results of this TenoMiR trial are highly encouraging. Patients felt a significant reduction in pain both on the numeric rating scale [NRS] and the ASES-E [American Shoulder and Elbow Surgeons Elbow Questionnaire] elbow pain subscale. Patients also experienced improvement in upper limb and elbow-specific function as assessed by QuickDASH. There was sustained improvement in tendon structure over 90 days, suggesting disease modification, measured by greyscale ultrasound. There is a significant correlation of improved tendon structure with patients receiving TenoMiR and reduction in pain and improvement in function – validating the clinical significance of tendon healing.”
“We plan to advance TenoMiR to a phase 3 trial for treatment of LE and a phase 2 trial for treatment of tendinopathy in the rotator cuff.”
Challenges Faced
Have you faced any challenges in your sector of work recently? Doogan acknowledged:
“There’s always a lot to learn when you’re first in the arena. To date, no microRNA therapies have been approved. It’s highly complicated and many strategies have failed. We have both the advantages and disadvantages of being one of the early first movers of programs using this approach.”
“We’ve learned from our work to date, including recently with our phase 2 trial, about the intricacy of appropriate delivery for this therapy. It is critical that it be injected directly into the affected area in the tendon. While we have extremely positive data from patients who appropriately received the injection, we didn’t meet our endpoints because of administration at select sites. It’s critical that as we move this forward, we work with investigators who are well versed in musculoskeletal medicine.”
“And, of course, it’s difficult to get investor attention in an already difficult financing environment. But we know that we have a solid product with significant unmet need.”
“It’s a lot of work, but tendinopathy profoundly impacts patients’ lives and costs the healthcare system millions, making it a worthy pursuit.”
Significant Milestones
What have been some of the company’s most significant milestones? Doogan cited:
“The data from our clinical studies is validating. We were encouraged by the phase 1b results that we saw in 2024 with clear and consistent translational success from animal to human models. And now the phase 2 readout builds on that.”
“This Phase 2 trial was a multi-site – in the US and UK – randomized, double-blind, placebo-controlled, parallel-group study evaluating the efficacy, safety and pharmacokinetics of TenoMiR, administered via intratendinous injection in 123 subjects with lateral epicondylitis. Two dose levels of TenoMiR given as a single injection were tested, and subjects were followed for 90 days. Of the 123 subjects enrolled in the study, 73% received the correctly delivered dose. We now have proof of concept through an evaluable population of 90 patients. This is strong data and has caught the attention global KOLs in musculoskeletal disease therapy. We are also discussing our development approach with ongoing discussions with regulators.”
Funding/Revenue
Are you able to discuss funding and/or revenue metrics? Doogan revealed:
“Yes. The company has raised approximately $25 million to date, made up of grants, tax credits, and private investment. We’ve been lean and focused. We’re currently engaging with investors as we seek to raise a Series A to move forward with the further studies we have planned.”
Differentiation From The Competition
What differentiates the company from its competition? Doogan affirmed:
“To our knowledge, we are the most clinically advanced micro-RNA treatment currently in development. There are no other companies taking the approach that we’re taking for the indications that we seek to treat.”
“Today, a patient with tennis elbow may be offered standard of care with pain relief – such as NSAIDs – and physiotherapy. They may end up undergoing surgery. We’re developing a therapy that treats tendon disease at the molecular level – this is not simply a pain killer. We have been able to show convincing effects in three domains of pain, function and tendon repair. This has not been shown with any other drug treatment.”
“Our team has expertise both in drug development and commercialization to take TenoMiR all the way through development and into the market place. We also have strong relationships with key opinion leaders and understand that there is a concentration of expert sites in certain US regions. This is a significant opportunity to bring TenoMiR to patients efficiently upon approval.”
Future Company Goals
What are some of the company’s future goals? Doogan emphasized:
“First, we want to initiate the phase 3 trial for TenoMiR in for LE and the phase 2 trial for rotator cuff. But, Causeway has much more beyond these indications. TenoMiR could be studied in certain fibrotic conditions and in intervertebral disc disease for example. And, microRNA technology can be targeted in other conditions in cardiology and dermatology, for example. Causeway plans to be a fully-integrated, end-to-end company with knowledge and experience to take candidates from discovery through to commercialization. So, the opportunities are huge – and we are open to working with partners who can help us bring TenoMiR to patients most effectively and efficiently.”
Additional Thoughts
Any other topics you would like to discuss? Doogan concluded:
“We seek to enable patients to reclaim their active lives and perform at their best. The pathway in drug development is never clear and straightforward but we feel confident that our technology works. Thanks for the opportunity to share with you and your readers.”
