Azafaros: funding a revolutionary drug

Azafaros is a venture-backed Dutch biotech company developing a potentially first-in-class therapy for rare neurological metabolic diseases.

The drug they are developing – a compound called nizubaglustat – has shown results that lead to improved mobility, fewer seizures and better walking for their patients.

1 in 5,000 people are affected by rare diseases. They can interfere with the brain’s chemistry – which can affect movement, cognition, behaviour, and overall health.

“But I always say that while the number of patients may be limited, there are also families and carers heavily affected by these diseases,” says Stefano explaining that Azafaros has hired early on a colleague specifically to maintain relationships with families and patient organisations.

Nizubaglustat is now entering Phase 3 trials, which is the final step before possible regulatory approval. “Phase 3 basically measures the effectiveness of the drug – so this is a pivotal moment for us,” says Stefano. That means that Azafaros is building to launch. “We’re going to start working on the commercial plans and hiring.

We will increase our medical presence in several countries and start our transformation from a small clinical company to a larger commercial one,” says Stefano, with a glimpse of both excitement and cautious anticipation.

Azafaros started in 2018 but is built on decades of research at Leiden University and Amsterdam UMC. When asked about financing – and whether a drug aimed at a relatively small patient population can be profitable for investors – Stefano explains that Azafaros’ strength lies in the fact that the company already has a potential solution. “It’s feasible for a private company to launch in rare diseases when you are sitting on a potential solution that is first of its kind,” Stefano says.

They raised €132m in a Series B round and are backed entirely by European investors – including Jeito, a French investment company focused on ground-breaking medical innovation that has been supported by the European Tech Champions Initiative. “Our funding story is a sign that the European ecosystem is working and is quite strong,” says Stefano.

The other thing that's strong is the impact at the end of the day. As Stefano explains, “the highlight of my year is going to meetings with the families of patients and seeing how they take on the challenge of living with these rare diseases every day. Knowing that we may help these people one day is what drives me.”