Our future appears to be in good hands after all. We can even feel appreciated.
That confirmation comes via a conversation with Amylyx Pharmaceuticals co-founders, Justin Klee, President, and Joshua Cohen, CEO. Klee and Cohen started Amylyx six years ago, when they were 21 and 22 years old.
Among their reassuring attributes: honest humility; entrepreneur élan; strong smarts; genuine growth.
Following our earlier editorial with Klee and Cohen, here’s a broader measure of the motivations and modus operandi of two of the youth inhabiting our drug development industry as we head into the 2020s … a decade we hope they will conquer.
The Cure To Every Disease
Let’s start with an anecdote from Cohen, partly because I’ve a soft spot for those who learn in their own way while at university.
Cohen shared this with me in response to a question on how he and Klee – the proverbial two guys in a dorm room – arrived at the idea to start a company specifically to form a new compound for neurodegenerative diseases:
“I had a professor who made the comment, ‘The answers to all the diseases are already out there. They're just buried in papers. And not enough people read the papers.’ I think there's truth in that.
“There is so much published research, but everybody is so busy with their jobs and various responsibilities. What was unique, and allowed us to start Amylyx, was for a time period at university I decided to, shall we say, attend fewer classes. Instead, I read as many papers as I possibly could. I don't know that other times in life or positions you get the opportunity to read hours of papers day after day without distractions … without the things that come with life as life goes on.”
“The molecules that make up our lead candidate were overlooked, mostly because they require extremely high dosing. They violated a couple of Lipinski’s rules of five. There are a number of reasons why your classical drug development engine would look at these and immediately throw them in the trash bin.
“At the time we selected them, lucky for us is we were intrigued by the molecules and didn’t start with those concerns. We said, ‘Okay, but might they still work?’ We had the time so we could focus on what we needed to without the distractions that come later in life.”
The Evolution Of Motive
But what actually drove Klee and Cohen, while pursuing undergraduate degrees at Brown University, to form a drug development company? Was it that pure interest in science? Had a certain disease affected their lives in some way, and they were devoted to finding a cure? Or was it dreams of cashing in on the biotech boom?
“The motivation has evolved over time,” explains Klee. “At first, it was more of that genuine scientific curiosity. We were looking at Alzheimer's and ALS-type diseases that are really terrible. There have been so many attempts at treatment, but unfortunately, mostly failures. So our first thoughts were, ‘Can we think of something else? Can we come up with something different that might succeed where others have failed? There was that challenge. Even as we entered the clinic, our motivations centered on that curiosity, and scientific bend.
“But the real driving force became patients. You can't spend time with ALS or Alzheimer’s patients, their families, caregivers, nurses, doctors, and not feel so compelled to try to help in any way possible. And then to also realize that as an industry, because of the many past failures, most companies have moved away from trying to solve these recognized societal problems, at least for the time being.
“So what drives us today is we have both an opportunity and a responsibility to help these patients, to help the whole community, to provide that first spark of hope of treatment. Perhaps similar to the way oncology has developed over the past 15 or 20 years. Death sentences are becoming cures, and difficult outcomes are well-managed conditions. That's what gets us really excited, as well as keeps us going.”
Most Outsourced Pharma readers will agree: That’s what keeps us going as well. And it’s comforting to hear it renewed by our young entrepreneurs like Klee and Cohen. Moreover, it’s reassuring to hear of their respect for our specific segment of the industry. It’s acquiring the deserved time and the professional services from CROs and CDMOs that have allowed them to move into exciting phase two studies with lead compound AMX0035.
Standing On Outsourcing Shoulders
Klee and Cohen may not have realized it when we spoke, but their comments on the ability today for young men and woman to bootstrap a biotech, get hypothesis professionally tested at experienced labs with high-tech instrumentation, have materials procured, developed, formulated, and manufactured at plants with state-of-the-art equipment, receive sage advice from experienced professionals, validates the service CDMOs and others provide the entire, global drug development industry. You could say it makes us proud of the outsourcing community we have become.
“We certainly weren't the first company to outsource. For us, it was like, wow, there are these groups that are experts in Alzheimer's mouse models, or API manufacturing, or packaging and delivery, whatever we need practically. What an opportunity for us. We get to partner with these world experts and get just the same quality as any big company. Why wouldn't we outsource?
“We didn't go into this with an ‘outsourcing business model’ in mind, but we had this great advantage waiting for us. We could not have brought in or built out these capabilities. Outsourcing really allowed our business to thrive, and is a big part of why we developed a company the way we have.”
Let’s end with these two key reminders:
The next generation of focused and upstanding individuals is indeed coming along to refuel our drug discovery and development efforts, and continue to further treat and cure human disease.
And we’ve built an outstanding outsourcing infrastructure to allow them to thrive.
Good news for all generations.