Presenting: Dr. Milind Deshpande

Based in New Haven, Connecticut since 2001, Achillion Pharmaceuticals devotes their efforts to small molecule therapies. On behalf of the company, President and CEO Dr. Milind Deshpande discussed the recent particle development victories. Tracing back his journey as a scientist in the pharmaceutical industry, a good 20 years ago, he mentioned that the only thing known about the complement immune system was that “it existed”. Today, with the advancement in knowledge and science in general, we know about the many pathways involved in this system and the numerous proteins associated with it. Achillion’s remedies largely target complement-mediated diseases. Host cells damaged by complimentary proteins can lead to a number of diseases, such as multiple sclerosis, age macular degeneration, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), myasthenia gravis, and neuromyelitis optica, to name a few.

As Dr. Deshpande began to explain the complex complement system, he acknowledged that the protein names are logical but almost silly when he has to repeat words like 5b, 5bb, and 5bc. Amongst these proteins, Achillion’s target is the “Factor D” protein, which is a serine protease complement that activates an unique alternative complement pathway. By targeting Factor D, it is possible to inhibit activation of this pathway when other harmful complement proteins are in play. Developed by Achillion, ACH-4471 is a potent inhibitor of Factor D. Weighing in at 500 grams per mole, this novel drug plays the same role as an antibody with a molecular weight of 25,000 grams per mol. Specifically, ACH-4471 prevents the binding of C3b and Factor B protein complex to Factor D. Further, Factor B is not cleaved, leading to the inhibition of hemolysis.

Although this is just one brief overview of a complicated, internal malfunction, Achillion is working on several different immune diseases. They have discovered an assortment of antiviral treatments for the Hepatitis C virus (HCV). HCV can be very difficult to treat because many of the present medicines have serious side effects and the early stages are nearly undetectable. However, Achillion has prepared ACH-3102, a NS5A inhibitor. NS5A is essential to HCV’s RNA replication. There is a 100% cure of HCV by the end of treatment with ACH-3102. Additionally, they have evolved a nucleotide NS5B polymerase inhibitor, ACH-3422, and a NS3/4A protease inhibitor, sovaprevir. In the future, they plan to have a combination of NS5A, NS3, and nucleoside polymerase inhibitors that will very likely improve all aspects of the HCV treatment. They aspire to have completely oral treatments by 2017, with a very safe and short treatment of only 6-8 weeks.

The company has generated a profound partnership with Janssen Pharmaceuticals, a Johnson and Johnson company, to further develop and commercialize their compounds. Together, Achillion and Janssen are developing the 3-drug combination therapy for HCV.

More than $900 million is estimated for potential development, regulatory, and commercial costs to develop ACH-3102, ACH-3422, and sovaprevir. Excellent science has met capital as Janssen invests about $225 million in equity and is alone backing the development and commercialization costs. It’s a win-win for Achillion with such a fantastic partnership. Janssen’s strong global footprint and their one HCV drug already on the market makes them the ideal “sustainable partner” Achillion has searched for. Moreover, this means good news for us Connecticut residents, as with its global expansion plans, Achillion needs to hire!

The success of Achillion is grounded in the heart of the lab. As Dr. Deshpande puts it, “we have great virologists, and good science makes things easier.” Once the foundations of the product are established, the rest is paying close attention to business. “You have to have the right product profile, like a safe treatment and no side effects.” Deshpande says that raising a cure success rate from 6% to 20% is a great victory because they aim for any progress they can use to eventually strengthen and improve their industry. Achillion’s main mission is to “transcribe innovation into novel discoveries and meet patient needs.” Each new day brings advancements, set backs, and insights to the scientific world we belong in.


Co-edited with Varsha Pednekar