Stephen Squinto, Ph.D.
Executive Vice President and Head of Research & Development
“When we founded Alexion, we started with a goal of harnessing newly emerging technologies
in the biosciences so we could build an independent biotechnology company by creating
useful products for some pretty tough diseases. This was unusual for a new company
of our size – the typical approach would be to discover a compound with some potential,
then seek a partner who can usher it through the complete drug development process.
By deciding to tackle the entire process ourselves and build a company, we knew
we were in for a long, difficult journey. But our scientists were up for the challenge,
because we remained focused on developing products that could significantly improve
patients' lives – a goal we still live by today.
“From the start we set our sights on attacking the complement system, an immune
process that involves many proteins that both protect us against foreign particles
found in the blood but can also cause severe inflammation. There's a long history
of companies delving into the world of complement biology through more traditional
approaches, but no effective complement blocking drugs had been developed. We were
one of the first companies to renew interest in complement biology by stating that
we wanted to block a well-established inflammatory pathway that is most likely common
to many diseases including some very rare disorders like paroxysmal nocturnal hemoglobinuria
(PNH), which is characterized by the destruction of red blood cells.
“We started by trying to engineer proteins that naturally exist to block complement
on your cells. After a few years without success, we shifted our focus to using
monoclonal antibodies as a way of blocking complement, just to prove we had the
right technology. It turned out that nothing worked better than these antibodies,
which showed us the way forward.
“It was an amazing feeling when we finally learned the results of the Soliris Phase
3 studies in patients with PNH. A month prior we found out that another therapy
we had in development for cardiovascular disease had failed, so we went from dramatic
disappointment to jubilation when we saw the Soliris data and realized the potential
for patients with PNH.
“I remember thinking, ‘We've finally done it.’ Here we are, 15 years later, and
we've done what very few companies of our size have ever done – move a drug through
discovery and development all on our own.
“Soliris is a unique product for a very rare disease. As the first terminal
complement blocking drug, Soliris also represents an advance in our understanding
of a fairly common inflammatory pathway. We now have an opportunity to investigate
other rare and underserved diseases where research shows that complement is involved.
We're working with the medical community to conduct clinical trials and sponsor
investigator-initiated studies to explore this further.
“Our business is to provide medications that will be life-changing for very ill
patients with rare, life-threatening diseases. That's a pretty tall order, but we've
done it before, which gives me encouragement that we can do it again.”