EXO Biologics CEO Discusses Innovation in Developing Exosome Therapies

Hugues Wallemacq, CEO of EXO Biologics.

Editor’s note: This interview has been lightly edited for brevity and clarity.

At the BIO 2024 Conference in San Diego, Pharmaceutical Executive had the opportunity to sit down with Hugues Wallemacq, CEO of EXO Biologics, a pioneering company dedicated to advancing exosome therapies. Founded with a mission to advance treatment accessibility, EXO Biologics focuses on developing scalable and GMP-compliant exosome therapies derived from stem cells and various cell types. This interview offers a glimpse into EXO Biologics' innovative approach and ambitious plans to reshape therapeutic landscapes globally.

Pharmaceutical Executive: Can you tell us about EXO Biologics?

Wallemacq: The mission of EXO Biologics is to accelerate the development of innovative exosome therapies and make them available to patients. A key challenge in the exosome field is producing scalable, reproducible, and GMP-compliant exosomes. It's a new and disruptive field, and that's where we have focused all our efforts. Our goal is to build a platform capable of producing exosomes from stem cells as well as from different cell types, and to load them.

We believe this will accelerate the field, and that’s our purpose. We are the first company in the world running a trial approved in Europe. It’s for an orphan designation, bronchopulmonary dysplasia (BPD). We selected this disease because there is a good fit between exosome-derived stem cells and this complex inflammatory condition. Additionally, this approach allows us to leverage an accelerated regulatory pathway.

Pharmaceutical Executive: What is the current treatment for BPD?

Wallemacq: Bronchopulmonary dysplasia (BPD) primarily affects preterm infants. These babies are exposed to oxygen because, without treatment, they would not survive. The treatment is mainly supportive, with no specific therapy for BPD. Sometimes steroids are used, but neonatologists often reserve them for severe cases. As a result, the disease has remained stable over the last two decades. This stability is why we chose to focus on BPD.

Our goal was to accelerate the development of exosome therapies and clinical advancements. This led us to develop a platform to address BPD specifically. Beyond BPD, our platform has the potential to address over 100 other conditions, including complex inflammatory diseases like pulmonary fibrosis, COPD, and asthma. Outside the pulmonary field, it can address chronic inflammatory conditions such as osteoarthritis.

Pharmaceutical Executive: So that's the beauty of the exosome platform, right? You don't necessarily need to have a single target.

Wallemacq: Exactly. You can produce it, and it can be repurposed and reused for different indications. That's why we are currently focusing on bronchopulmonary dysplasia, but we are also starting a second indication related to skin, aiming to demonstrate the regenerative effect of these exosomes. With the same type of exosome, you don't need separate development tracks for each indication. In other words, you can use the same exosome for multiple purposes.

Right now, we are accelerating our efforts because our mission is to expedite development. We are forming partnerships with other entities in Europe and the US. We've signed several contracts to reuse the same exosome platform to speed up clinical trial demonstrations. The hope is to run the phase one trial in Europe and the phase two in the US. For all indications, we are targeting both the US and Europe because the US is the biggest market.

Pharmaceutical Executive: Tell us more about the science behind the platform and what piqued your interest in it as a scalable platform.

Wallemacq: We started with stem cell therapy initially. Our CSO, Marcin Jurga, PhD, worked on that project. The team sought to find a precise and efficient way to produce stem cells in a scalable manner using a bioreactor. Based on this initial asset, they designed the platform to be scalable in two ways—downsizing and upsizing—to ensure it could support technical and clinical trial phases, from phase one to phase three and eventually to commercial production. We use bioreactors and can extend the scale up to 500-fold based on the same principle.

Pharmaceutical Executive: Are you doing all the manufacturing internally, or are you partnering with a CDMO?

Wallemacq: We do it internally. We have created our own CDMO, and our GMP zone, which is a separate entity within the company called ExoXpert. It's so important to control the production in exosome development that we decided to keep it close to us.

Pharmaceutical Executive: Is the goal, as you're developing assets, the two entities will run in tandem and, and you'll license ExoXpert to other companies?

Wallemacq: Yes, exactly. With EXO Biologics, we focus on direct drug development based on our platform. It takes years and millions of dollars to get into clinical trials, so we created ExoXpert to license the platform and accelerate development. We signed our first partnership with a US company, NeuCore Bio, and we continue to gain traction. What I consider is ambidexterity: EXO Biologics accelerates drug development, while ExoXpert uses a licensing model to accelerate development and revenue for the group.

Pharmaceutical Executive: When do you anticipate getting clinical trial data?

Wallemacq: We are currently in a Phase I/II clinical trial with 36 babies currently in phase one. This is a dose-escalation study with 36 participants in the phase I part, but the advantage is that we have 100 historical controls because the disease is very stable. A large portion of the babies will develop BPD, allowing us to confirm safety and measure the effect of the exosome on preventing disease development in terms of incidence and severity. It's a proof of concept directly.

Pharmaceutical Executive: Are you working with regulatory agencies in both the EU and US to solidify the endpoints?

Wallemacq: Yes, but the endpoint here is a well-established clinical endpoint in both the US and EU. The timing is pretty soon after birth, between two to three months. The clinical diagnosis is independent of us; you are either diagnosed with BPD or not, and there is also a severity grade associated with it. This is harmonized between the FDA and EMA.

Pharmaceutical Executive: Assuming phase one goes well, what size do you anticipate for phase two?

Wallemacq: It will be between 100 and 200 patients, depending on the monitor size. If the data is positive, we expect to have a conditional market approval directly after phase two, given the orphan drug designation from the FDA and EMA. That's the goal: to go as fast as possible to show the benefit to the patient and get market approval.

Pharmaceutical Executive: Any thoughts on getting into oncology?

Wallemacq: We are not focusing on clinical trials in oncology, but we know that exosomes can deliver payloads to specific spaces or organs in the body. We have R&D evidence and IP on delivering anti-cancer drugs using exosomes, particularly for bone and osteosarcoma. That could be a potential niche in the future.