Josh Cohen

Amylyx Pharmaceuticals

For the past eight months, my business partner Justin Klee ’13 and I have been working on developing a therapy for Alzheimer’s disease. Alzheimer’s Disease is a terrible affliction that currently affects over 5 million patients in the United States alone, making it the sixth leading cause of death and the third amongst people 65 and older. The number of patients is expected to grow at an increasingly faster rate over the next forty years as the population ages. On an economic scale, Alzheimer's costs the US healthcare system an estimated 216 billion dollars (Alzheimer’s Association, 2013). Devastatingly, there is no cure and there have been more than 100 consecutive drug failures for Alzheimer’s disease over the past ten years. The few current treatments extend life by about 6 months and do nothing to stop the progression of pathologies. Yet drug companies are hesitant to continue to pursue this massive unmet need because of the past failures.

Alzheimer’s disease has typically been considered as a disease caused by two proteins: amyloid beta, and tau. These proteins are known to disrupt neuronal signaling in the brain. Current therapies have repeatedly targeted this pathology and despite success in affecting levels of these two proteins no drug has seen particular success. After an extensive literature search, Justin and I became convinced that Alzheimer’s disease was more metabolic in nature and that these dysregulated proteins may be downstream of an earlier pathology. Using this mechanism we scanned the mechanisms of action of known drugs and found a combination of two existing non-neurological drugs that might treat the disease. Hopefully this new approach will succeed where other technologies have failed.

Next, Justin and I sought out opinion leaders in the field to help us to validate our science. We have since built a strong team of both business and scientific advisors who both provide verification of our science and give us advice as we move forward. Needing intellectual property advice, we have also signed on Fish and Richardson as our retained patent law firm. We have also filed two provisional patents which have been reviewed by Fish and Richardson to aid in protecting our idea. They are conducting future work to produce full U.S. utility patents.

We are currently testing our compounds in two studies. These studies are two separate in vitro studies aiming to provide further validation to our idea. With successful studies we will aim to move into in vivo studies and begin compiling an IND package which will allow us to conduct future tests in humans.

So far, Justin and I have provided for the company out of pocket, incurring >$10K in costs to keep the venture afloat. In the immediate future we will have to pay lawyers fees as well as fees for one of the studies we are conducting. The $500 grant we are receiving from BVLF should help us progress and learn more as we continue the project. Hopefully with this grant we will be one step closer to providing a therapy for this debilitating disease.

Reflection - Explore:

Amylyx has recently completed our first study with Charles River labs. The total study cost was $8000 the $500 was used to support this cost. We are pleased with the results from the study and very thankful to Brown for providing us with money to support this study. Through this process we have built a strong relationship with Charles River Labs and gotten onto a first name basis with multiple of their scientists. We have learned what is happening in the field of assay development as well as been invited to a conference on information in the field which we attended. There, we learned about recent advances in mice studies of hypercholesterolemia as well as new approaches in antibiotic drug discovery. We also heard a speaker talk about the limitations of current pharmacokinetic measures in medicinal chemistry. 

We have grown and learned from the experience and we are thankful that the Brown Venture Launch Fund was able to help us make this happen. As we look forward to filing our patent as well as preparing for an in vivo study. The promising results that we received should also help us to garner investor support for our next round of financing.

Next steps - Expand:

We are thankful to Brown to have received the $1,500 in grant funding to continue our work at Amylyx. Now that we have completed our first study our main focus has become defending our results in the form of a U.S. utility patent. Last Thursday we discussed with our retained patent lawyers at Fish and Richardson how best to protect our ideas in the form of a patent. We learned from their experience significantly more about the patent filing process and proceeded to draft a first draft of the patent at their request. This process will save in patent costs on our first filing.

The first filing is likely to cost about $10,000 but our parents have agreed to support us with this cost. The additional $1,500 from Brown will lower our cost figure, and significantly help us in affording this milestone as well as allowing us to save money for future operations. We are again thankful to the Brown Venture Launch Fund for providing us with this opportunity.

Expand Grant Report:

On December, 24th 2013, Amylyx filed our first Utility patent with Fish and Richardson LLP. which grants us an additional level of protection over our idea. The patent cost and $10,000 and BVLF’s $1,500 expand funding was instrumental in covering this cost.  The patent was filed claiming priority on two provisional patent applications which we filed in March. Through the patent process we have become experienced in the process of breaking down an idea into a set of claims and descriptive language. This patent has been critical as we have sought to raise money to further the company. Our early data and developing intellectual property position are beginning to build a compelling argument to potential early stage investors.

Our next major steps include a mouse model efficacy study which will support the use of our therapy in patients suffering from Alzheimer’s Disease, the filing of a PCT application with Fish and Richardson LLP. to further advance our Intellectual Property position, and additional studies in collaboration with labs at Harvard Medical School/Massachusetts General Hospital. We are thankful to the BVLF and Brown University for providing us with funding and continued support.