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Amylyx Pharmaceuticals: A Beam of Hope on the Pharmaceutical Horizon


Lending a helping hand to struggling patients is the one thing Amylyx does best!

Some statistics show that today in the US, close to 1 million people are living with some sort of neurodegenerative disease. Fortunately, there are dedicated companies and organizations that work tirelessly to make these people’s day-to-day life a whole lot easier.

Amylyx Pharmaceuticals, a fast-growing pharmaceutical company, was founded in 2014, with the initial idea and optimism to create a treatment for people living with neurodegenerative diseases. Since then, Amylyx has managed to invent and develop AMX0035, with the goal being a treatment for amyotrophic lateral sclerosis and other neurodegenerative diseases.

The two co-founders of the company, Justin Klee and Joshua Cohen, brought their minds together, created a team, and have collaborated with numerous other companies, organizations, scientists, and doctors to try to develop therapeutics for the treatment of neurodegenerative diseases.

Stay with us and read all about Amylyx’s success story.

Budding Years

One of the founders of Amylyx, Joshua Cohen went to high school at Milton Academy, in Milton, Massachusetts. Once he graduated high school, he managed to enroll at Brown University. In 2014, Cohen graduated and got his bachelor's in Biomedical Engineering.

The other co-founder of the company, Justin Klee, was a student at The Williams School, a private secondary school in New London, Connecticut. Once he graduated high school, Justin also enrolled at Brown University. Spending 4 years at the university from 2009 until 2013, Justin earned himself a Bachelors of Science (B.Sc.), in Neuroscience.

While brewing the idea of creating a company together, both Joshua and Justin had their respective jobs. For a brief time of four months, Joshua was an Engineering Researcher at The National Institute of Standards and Technology.

Justin, on the other hand, was a Neuroscience Researcher at Brown University for about two years, and later for another two years, acted as a Neurology Research Technician at Massachusetts General Hospital/Harvard Medical School. While being there, Justin specialized as an Alzheimer’s Disease Research Technician.

The Ball Starts Rolling

Back in 2013, as both Joshua and Justin had their own ideas and ambitions, they decided to sit down and discuss the opportunity to create and launch their own company. With Joshua specializing in biomedical engineering and Justin having a ton of experience in neuroscience, they decided that putting their minds together would bring about a brilliant idea, for sure.

Even when they launched Amylyx, at the same time, Justin was still an undergraduate neuroscience researcher, and wrote his thesis on "Neural Correlates of Psychophysical Performance in a Vibrissa-Dependent Detection Task". Later, he was even appointed Alzheimer's Disease Research Technician, at the Massachusetts General Hospital/Harvard Medical School, which increased his desire to create and develop a treatment for neurodegenerative diseases, such as Alzheimer’s and ALS.

Joshua and Justin both knew that only the two of them wouldn’t make the impact that they wanted, so they started creating a team around them. On top of that, since the launch of the company, they have also urged other companies and organizations to collaborate, developing something that will make a positive difference in the future.

The Only Way Is Up

When the company first launched in 2014, Cohen was already working on AMX0035. The launch of the company only boosted the work that was needed to fully develop the treatment. With the help of Justin, their team, and numerous other collaborators, Amylyx had developed a drug that was ready for clinical trials.

There have been two fully complete trials since the launch of the company, the CENTAUR Phase 2 Study, focusing on the safety and efficacy of AMX0035 in ALS, and PEGASUS Phase 2 Study, which evaluated the tolerability, safety, preliminary clinical efficacy, and neurobiological activity of AMX0035 in patients with mild cognitive impairments due to Alzheimer’s disease and dementia.

The CENTAUR Phase 2 study was the first official trial that Amylyx was able to do to evaluate the safety and efficacy of AMX0035, which is a coformulation of two different active compounds, sodium phenylbutyrate (PB) and taurursodiol (also known as ursodoxicoltaurine. There were a total of 137 adult US participants with amyotrophic lateral sclerosis (ALS).

Apart from testing the safety and efficacy of the treatment and impact that it had on the disease progression, further measures were also assessed which are critical to people with ALS such as muscle strength, biomarkers of neuronal degeneration, and lung vital capacity. The trial was officially completed in 2019, and the results were more than encouraging.

The PEGASUS Phase 2 Study was next in line, and it had 95 participants from the United States, from the ages of 55 to 89. This trial was a placebo-controlled, randomized, trial assessing the effects of AMX0035 on patients with dementia or mild cognitive impairment due to Alzheimer’s disease. All of the participants went through a 24-week treatment and in the end, showed a significant impact on multiple biomarkers.

Joshua and Justin presented the results of PEGASUS at the 14th Clinical Trials on Alzheimer’s Disease (CTAD) conference in Boston, where they stated that the biological knowledge that they have gained from recent trials, especially with the PEGASUS Phase 2 Study, has shown that AMX0035, with further testing, can be the solution to treat various neurodegenerative diseases.

Securing the Financial Grounds

Since late 2014 and early 2015, Amylyx has gathered close to $202 million in seven different funding rounds. The latest funding was raised not so long ago on July 20, 2021, where a total of 14 investors contributed to the company. The last funding raised a total of $135 million, with lead investors like Viking Global Investors.

Right at the start of 2022, Amylyx decided to join the public market. In the beginning, the company was ready to offer $8.75 million shares, however, at the very last moment, they decided on offering $10 million at $19 apiece. As the first day of trading was coming to an end the price came down to $18.07.

Being present in the world of pharmaceuticals means that one will be faced with fierce competition. Namely, companies like Mankind Pharma, Ferrer, and PCI Pharma Services are among the top Amylix competitors in the race for securing a healthier tomorrow for everyone. Throughout the years, ALS Finding a Cure, the ALS Association, and Morningside Venture Investments have all contributed to Amylyx, with a goal to continue the development of AMX0035.

Throughout the years, Amylyx has partnered up with more than 20 other organizations and companies that share its noble vision. Amylyx, with the help of numerous organizations like the ALS Association, ALS Finding a Cure, and many more, has worked to develop a treatment for multiple neurodegenerative diseases.

Future Ambitions

At the moment, with headquarters in Cambridge, Massachusetts, Amylyx is constantly trying to increase the number of its staff. There are more than 200 employees at Amylyx and hundreds more collaborating with the company to develop treatments for neurodegeneration diseases. Currently, there is an ongoing trial named PHOENIX Phase 3 Trial, which is still in the enrollment phase. To date, this will be the biggest controlled trial which will span across 70 sites in the United States and Europe. More than 600 participants are required, and the goal of this 48-week trial is to further assess the safety and efficacy of AMX0035.

The initial starting point was back on the 28th of October,2021 and the estimated study completion date is said to be in March 2024, more information on enrolling can be found at This trial could be the next big breakthrough, and Amylyx is hopeful that it too will show positive results like the previous studies and trials.

From the initial idea right until today, Amylyx has always wanted to do more for people that are living with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. There are constantly urging individuals and other like-minded companies and organizations to come together because collaboration will only increase the strength, assets, and ideas that will ultimately impact the lives of people that are suffering each day.

Whatever the future might hold for Amylix, one thing is for sure — the pharmaceutical industry will be one step closer to alleviating the struggles of patients suffering from neurodegenerative illnesses, and that’s pretty darn good news.