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by Zachary Jordan '21  The biotechnology industry is, by and large, dominated by large companies and people who have spent their entire lives (and many years’ worth of education) becoming the foremost experts in their given fields (4). In a recent survey of biomedical engineers, nearly 20 percent of respondents indicated that a doctorate was required for their position, and a full 35 percent asserted that a master’s degree was necessary (5). The field is heavily regulated and often requires millions of dollars upfront to develop and test a drug or device. By many, it is described as one of the hardest fields to become a part of – entering the market takes tenacity, good lab results, a little bit of luck, and many, many late nights (6). But for some, passion for science and the opportunity to make a difference in the lives of thousands of people drives the development of life-changing technology.  Meet Josh Cohen ‘14 and Justin Klee ‘13, Brown graduates who opted to take this path. In 2013, in the face of a painful job application process, a casual conversation between Josh and Justin became a serious business venture. As biomedical engineering and neuroscience concentrators, respectively, cofounders Josh and Justin had been exposed to neurodegenerative disease biology in classes and research. They began to take an interest in metabolic disease, and as they would describe it, “one thing led to another” (4). Today, their company Amylyx Pharmaceuticals, Inc. has raised more than 10 million dollars towards the advancement of its drug (AMX0035) from the ALS Accelerated Therapeutics Initiative, grants, venture capital funding, and private investments (8). It is soon to enter Phase II clinical trials, a major step towards the implementation of their drug. How did recent college graduates succeed in one of the most challenging industries to break into? In between their busy schedules, Josh and Justin found a few minutes to talk with me about their journey, give some advice for interested entrepreneurs and scientists, and reveal the truth of the industry as they see it. First off, what is Amylyx? In their own words, “Amylyx is an early-clinical stage pharmaceutical company dedicated to providing innovative, disease-modifying solutions to neurodegenerative diseases” (8). With only three full-time employees, the company is what is known as a ‘virtual biotech’. This means that all wet lab research and clinical trials are contracted out to third party companies. Each CMO (contract manufacturing organization) and CRO (contract research organization) has a specialty and a staff that can take care of all the legwork, which makes virtual biotechnology companies increasingly common. This allows Amylyx to focus on concepts rather than logistics, and helps people with good ideas bring new technology to market without having to rely on large pharmaceutical companies (4). For entrepreneurs that lack years of wet-lab experience in biotechnology, the advent of these third-party agencies has opened the door to innovation.  What about AMX0035? According to Josh and Justin, their company originally had its sights set on Alzheimer’s. Classes at Brown inspired them to look further into neurodegenerative diseases on their own time – after reviewing the literature, it was clear that the scientific community was far from reaching a consensus on how to treat the disease. For years, it was believed that amyloid clumps (protein aggregates that easily stick together, forming fibrils in the brain) were in some way responsible for the cognitive deterioration of Alzheimer's patients (2). However, the Alzheimer's Disease Neuroimaging Initiative (ADNI), a multimillion dollar study funded by the NIH, showed that amyloid levels don’t correlate as highly with decline in cognitive function as does brain shrinking (1). So, Josh and Justin looked for drugs that instead inhibited neuroinflammation and nerve cell death (responsible for this change in neurological tissue) – after reviewing the literature, they found two drugs that they thought would interact synergistically to achieve this goal: TUDCA and sodium phenylbutyrate. Instead of putting in millions of dollars to synthesize a new compound, the founders of Amylyx patented a proprietary combination of two existing drugs, both of which had been validated in previous studies by academic groups (4). By thinking creatively, they then only had to prove that the two work together better than individually to validate their provisional patent (a ‘bookmark’ of sorts giving you a year to prove that your idea has scientific validity). Later on, after talking with experts, they realized that proof-of-concept clinical trials for AMX0035 should focus on ALS (amyotrophic lateral sclerosis), a progressive neurodegenerative disease associated with the deterioration of nerve cells in the brain and spinal cord (7). Realizing that AMX0035 had strong data in this area (while the exact cause of Alzheimer’s is still unknown, making the efficacy of AMX 0035 with respect to Alzheimer’s more uncertain), Justin and Josh refocused their fundraising and company platform. Science First Josh notes that, “the science has driven the company from day one and continues to today” (4). Ultimately, for an entrepreneurial venture in the sciences (especially biotechnology) to succeed, you need a methodology, product, or perspective that differs from the others on the market. If you have an idea that you think could change people’s lives, be as thorough as possible on the front end – make sure that your idea isn’t too similar to another one to be patented, and that your scientific basis is well founded. CRISPR-Cas9 genome editing technology serves as a valuable case study for intellectual property protection. Two labs fought a multimillion dollar patent lawsuit over the potentially world changing technology (3); if you don’t have the money to do the same, it’s essential that you establish your patent and show in your experimental phase that your idea is unique and scientifically based. Josh commented that “the first experiments were very stressful. You raise a little money, use that money to get a little data, and use that data to raise more money. It took us a year to raise our first serious investor” (4). As soon as your experiments reach completion and your proof of concept is complete, then it’s time to focus on fundraising or looking for possible partnerships.  How do I know if my idea is viable?
Before you put in the money towards your first experiments, it’s essential that you hone them as much as possible if you want to succeed. To do so, it is critical that you reach out to as many experts as you can. Josh and Justin told me that at the end of every meeting they went to, they asked who they should talk to next in order to further refine their ideas (4). Because Josh and Justin were willing to reach out and ask for advice, Amylyx has a Board of Directors full of experienced scientists and businessmen. These include George Milne (former President of Central Research at Pfizer), Steve Chubb (former President and CEO of Allegro Diagnostics, Inc.), and Walter Gilbert (1980 Chemistry Nobel Prize winner and Professor Emeritus at Harvard), among many other distinguished scientists (8). By surrounding yourself with experts, you can quickly create a reliable consensus about the direction your research should be taking and what steps need to be taken next. Expect to hear fifty “NO’s” for each “YES”, but with dedication eventually you’ll see your product evolving to fit into the existing market (4). More and more experts will voice their approval, and after much hard work you’ll be ready to proceed to clinical trial. Do I need a once-in-a-lifetime idea to make it in such a tough market? The high level of competition in the biotechnology industry often deters interested parties because they aren’t sure of their idea. However, Josh and Justin offered a unique perspective on the issue: because it’s so expensive to develop a drug, there are innumerable drug combinations, compounds, and treatment mechanisms that have yet to be tried! They emphasize this as a valuable lesson to those interested in the field, as Justin comments, “The most important thing is believing in your idea – the fact is, the world doesn’t care unless you make it. Commit yourself, and have solid science to back you up” (4). If you’ve proven the science is successful and made connections with scientific authorities who concur, you can raise the money you need to proceed to clinical trials and ultimately to market. If your idea doesn’t hold water, hopefully you’ll be told so by the multitude of experts in the field that you’ve met with so that you don’t waste time and money. Let the science and data drive your development. Biotechnology – a uniquely challenging field Because of the above challenges, most of the entrepreneurs in the biotechnology field are professors or ex-employees of big pharma. In 2013-2014, the average age of the CEO of a newly public biotech company was 53 - with more than 70% in their 50’s or 60’s. Even among first time CEO’s, the average age is 51 at IPO (9). This is another reason why having a reputable network of advisors is important in establishing the credibility of your company, especially if you’re young. Josh and Justin are a part of the shifting demographic that has been made possible by the advent of virtual biotech companies. In addition to these challenges, the biotech industry is heavily regulated, and for every topic and field there’s an expert. Nevertheless, for those willing to think outside the box, be persistent, and make connections, there’s also boundless opportunity. By reaching out to experts to validate your plans and ideas, protecting your intellectual property, and doing your homework (both literally and figuratively), it’s possible for a dedicated Brown student to make a difference in the lives of thousands of people -- and that’s a cause worth all the late nights and stressful experiments in the world. References:
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