Rejuvenate Bio is advancing its lead gene therapy for treating mitral valve disease (MVD) in dogs and believes its pipeline has blockbuster potential.
The California-based start-up is developing a “revolutionary approach” to treating age-related diseases. It claims to have identified several longevity genes with robust safety profiles that can be used to treat these chronic conditions and believes its gene expression technology has a significant opportunity to treat a large patient pool in both animals and people.
Speaking at the recent Kansas City Animal Health Corridor 2021 Digital Animal Health Summit, company founder and chief executive Daniel Oliver said: “We think these two markets fit together very well. Our work in animal health provides early revenues and validation for human applications, and since dogs suffer from similar age-related diseases, it has also served as pre-clinical models for human gene therapy.
“Although we’re using novel technology to enter these key indications, gene therapy is actually very well understood, particularly in dogs. There has been a number of products in a similar vein approved for use in humans and the vast majority of those used dogs as their pre-clinical model. There’s quite a bit of data out there suggesting gene therapy is effective at treating disease in dogs.”
Rejuvenate Bio will be taking advantage of the newly expanded conditional approval pathway in the US to accelerate clinical development of its combination gene therapy for canine MVD. The firm claims MVD is one of a number of different blockbuster indications it will target in animal health, including osteoarthritis. The business is starting with a focus on MVD because with the current standard of care, it is “a death sentence”. Mr Oliver said dogs are often diagnosed in the later stages of the disease and prescribed small molecule therapeutics, which typically slow the progression of heart failure but do not stop it.
Mr Oliver noted MVD affects about 7% of all dogs and certain breeds have a high disposition to it, including the Cavalier King Charles Spaniel. In fact, Rejuvenate Bio’s R&D has been backed by a partnership with the American Cavalier King Charles Spaniel Club, which has shared knowledge about the condition in the breed, sponsored the firm’s work with over $200,000 and is helping to enrol dogs for an ongoing pilot trial at the Cumming’s School of Veterinary Medicine at Tufts University.
Rejuvenate Bio selects DNA that ‘turns on’ the genes that halt the progression of MVD, and encapsulates it in an adeno-associated virus (AAV) – the same type of virus that has been approved for use in humans. The therapy is then administered as a one-time intravenous injection. Mr Oliver said through preclinical work for human applications, AAV gene therapies have been shown to last more than 10 years in dogs.
In 2019, the firm published a paper that showed its lead therapy was efficacious in treating four age-related conditions in mice, including heart failure, kidney failure, diabetes and obesity. Since being progressed to canines, over 50 dogs have been treated with the therapy. The candidate has demonstrated efficacy in weight loss and the reversal of key heart failure metrics. Mr Oliver said no safety problems have been observed, either from the initial injection or long-term expression of key proteins.
Rejuvenate Bio’s technology is protected by two patents. Its key patent is licensed exclusively from Harvard University and covers all combinations of the firm’s three lead proteins in gene therapies targeting age-related diseases in dogs and humans.
Rejuvenate Bio sees an opportunity to address the 1.4 million dogs diagnosed with stage B2 MVD each year. Mr Oliver said if the company is able to capture just 2% of that market, it would represent around $100 million in revenues.
Mr Oliver commented: “We think this price point is right in line with other common treatments for life-threatening diseases. It’s very similar to what it would be if you had your dog on Vetmedin, the current standard of care, which is not a cure but just slows the progression of the disease for two years. It’s also similar to any kind of surgery for a life-threatening illness, and it’s also similarly or lower priced than the typical cancer interventions.”
He also revealed Rejuvenate Bio is already in discussions with “large animal health companies” for sales and distribution agreements. He pointed out 71% of wealthy pet owners are concentrated in the top six metro areas in the US, meaning the company’s initial customers are reachable with a small sales force. Additionally, Mr Oliver noted the firm’s partnership with its existing investor Mars Petcare will enable rapid expansion. Mars owns both the VCA and Banfield Pet Hospital clinic networks.
Funding and next steps
Mr Oliver told IHS Markit Animal Health the start-up raised approximately $13.5m through a series A funding round. Combined with data from its ongoing trials and accelerated market entry through the expanded approval pathway, the firm is looking at a 2023 commercialization date for its MVD therapy.
Mr Oliver said: “Our current priorities are transferring our manufacturing process to a third-party manufacturer then running our target animal safety trial. We have an ongoing pilot trial with Tufts veterinary school where we’ve been giving our therapy to people’s pets that have MVD. In following them, we’ve had dogs on the therapy now for over a year. Our first therapy has also shown promise in treating other cardiac conditions as well as other metabolic conditions, and we’re continuing to investigate that. For us though, the primary goal is to get it to market – we think that’s the most important thing.
“We’re taking advantage of the expanded approval statute here in the US, which allows us to take our MVD product to market, as we are concurrently running an efficacy trial, so that gives us a head start here. The second and third markets are going to be a bit delayed because we need to run that efficacy trial. We’re very focused on getting this US conditional approval because that allows us to get to market much quicker, but unfortunately a number of the other countries’ regulatory bodies don’t have similar programs to this expanded approval statute.
“For our second combination therapy, which has shown some ability to treat things like osteoarthritis, we are in the process of running pilot trials to verify its efficacy in large animals, i.e. verifying the results from rodents.
“We are always interested in partnerships, particularly where the partners bring different skill sets. We do believe we could bring the MVD product to market ourselves but we respect and believe that there are a number of potential partners out there who have quite a bit of experience bringing novel therapies to market, particularly in the cardiac space.”
Reprinted with permission of IHS Markit