References 1 Kiernan, UK and Naylor, S. Emerging Paradigm of Integrated Platform-Drug Discovery and Development Companies. Drug Discov. World. Fall Edition, 33-43, (2018). 2 Ben-Joseph, O and Manning, S. Platforms Versus Products in the Life Sciences Sector. thepharmaletter. 19th January (2017). https://www.thepharma versus-products-in-the-life- sciences-sector. 3Thong, R. Bioscience Platform Companies and Their Lifecycle. SciTechStrategy. /01/bioscience-platform- companies-and-their-lifecycle/ (2015). 4Thong, R. Everything You Need to Know About Biotech Business Models. ung-biotech-business-models/ (2018). 5Terry, M. Moderna Therapeutics Sets Record for Biggest IPO. BioSpace cle/moderna-therapeutics- biggest-ipo-in-biotech-history/ 7th December (2018). 6 Oakes, B and Amer, Y. One of Biotech’s Biggest Investors Says the Industry Must be Unreasonable. WBUR Website- Bioboom. http://www.wbur. org/commonhealth/2018/06/07 /noubar-afeyan-interview 7th June (2018). 7 Mukherjee, S. Moderna Had the Biggest Biotech IPO Ever. Here’s What That Says About the Industry's Future. Fortune moderna-ipo-biotech-future/ 8th December (2018). 8 Smith, S. One Hundred and Twenty Seven Startups Using Artificial Intelligence in Drug Discovery. BenchSci. ups-using-artificial-intelligence- in-drug-discovery 18th March (2019).

However, the current valuation of BenevolentAI is predicated on its AI-DDD efforts providing services and drug candidates to third party clients. Ben- Johnson and Manning would vociferously support such focused efforts2. In contrast it could be argued that CureHunter is testimony to a company lacking focus. The company struggled to attract equity investments in both its AI-DDD and AI-PD3 endeavours. However, in this case it would be an oversimplification. CureHunter was almost a decade ahead of all competitors in terms of AI- DDD. It enjoyed some real success in terms of its Evidence-Based Medicine subscription model, but struggled to convince investors as to the value of AI- DDD before it became the ‘shiny new bouncing ball’ that everybody now chases today. Thus part of the lesson from consideration of the CureHunter situa- tion is the matter of timing and the punishing effects of the Technology Hype Cycle1. Verge Genomics is a dedicated AI-PD3 company.

This young company is still building out its corpo- rate infrastructure and developing alliances with major academic institutions to pursue the develop- ment of drug candidates in ALS and Parkinson’s disease. It is too early to determine the trajectory of this company, at present, but the investment com- munity enthusiasm for AI-PD3 has facilitated the company’s fund raising efforts with a war chest of $36 million. A number of other AI-PD3 companies find themselves in a similar predicament and includes a number of other dedicated AI-PD3 com- panies such as AI Therapeutics, Auransa Inc, Berg Health, Insilico Medicine and ReviveMed. Future evaluation of this sector will determine whether PD3 companies are a fad or a real trend.

Drug Repurposing PD3 companies Drug Repurposing (DRPx) is used as a catch-all term that is defined as ‘the concept of an active pharmaceutical ingredient, at any stage of the life cycle and regardless of the success or misfortune it has encountered, serving a therapeutic purpose that is significantly different from the originally intend- ed one’. We have written extensively on this subject in past editions of Drug Discovery World10. A number of DRPx companies qualify as PD3 corpo- rate entities and several are discussed below.

Continued on page 51 50

Biovista ( is a pioneering com- pany in both DRPx and PD3, long before both approaches were included in the popular lexicon. The company was founded in 1993 by the Persidis brothers and is based in Charlottesville, Virginia, USA, as well as Athens, Greece. It currently has three employees, with annual revenues of an esti-

mated $2.2 million in 2018 and is still a privately- held company. The company was originally an AI- PD3 company with a vibrant drug candidate pipeline. However, more recently it has transi- tioned to an AI-DDD company providing opera- tional and drug candidate services to the pharma sector using its proprietary AI systematic drug- repositioning platform.

Recursion Pharmaceutical (www.recursionphar- is a dedicated DRPx-PD3 company found- ed in 2013 and based in Salt Lake City, Utah, USA. The company has approximately 120 employees with ~ $5 million in revenue for 2018. The compa- ny has raised $141.3 million in a combination of equity investments as well as debt financing. The current pipeline consists of 10 drug candidates with two in Phase I trials and the remaining eight in pre- clinical stage with a current valuation of an estimat- ed $275 million.

Melior (; www.melior- is a DRPx-PD3 company predicated on its theroTRACE mouse phenotype model screening platform. The company was founded in 2005 and is based in Exxon, Pennsylvania, USA. The company has ~25 employ- ees (Melior Discovery) with an annual revenue of ~$10 million in 2018. The company raised ~$10 million in a Seed Round for Melior Discovery. However, since then Melior Discovery has provided service-generated revenues to support the PD3 efforts. The current business model is different from other DRPx-PD3 companies. Each time a drug can- didate is identified, a new corporate entity (Melior Pharmaceuticals I, II, etc) is formed that holds the IP and drug candidate development responsibilities. Currently, two Melior Pharmaceutical companies exist focused on Type II diabetes (completed Phase II) as well as NASH (preclinical evaluation) in Melior Pharmaceutical I, and Parkinson’s disease (entering phase II) in Melior Pharmaceuticals II. The valuation of these companies combined is pred- icated on the innovative PD3 self-sustaining model of Melior Discovery and the drug candidates in the pipeline of Melior Pharmaceuticals I and II.

Biovista started as a DRPx-PD3 company before

the concept was even articulated. This was done in concert with service provisions as a source of rev- enue. Biovista demonstrated innovation, creativity and initial success with a pipeline of drugs candi- dates. However, problems ensued in attracting investment funds to develop drug candidates. The parallels with CureHunter are striking and another

Drug Discovery World Spring 2019

Page 1  |  Page 2  |  Page 3  |  Page 4  |  Page 5  |  Page 6  |  Page 7  |  Page 8  |  Page 9  |  Page 10  |  Page 11  |  Page 12  |  Page 13  |  Page 14  |  Page 15  |  Page 16  |  Page 17  |  Page 18  |  Page 19  |  Page 20  |  Page 21  |  Page 22  |  Page 23  |  Page 24  |  Page 25  |  Page 26  |  Page 27  |  Page 28  |  Page 29  |  Page 30  |  Page 31  |  Page 32  |  Page 33  |  Page 34  |  Page 35  |  Page 36  |  Page 37  |  Page 38  |  Page 39  |  Page 40  |  Page 41  |  Page 42  |  Page 43  |  Page 44  |  Page 45  |  Page 46  |  Page 47  |  Page 48  |  Page 49  |  Page 50  |  Page 51  |  Page 52  |  Page 53  |  Page 54  |  Page 55  |  Page 56  |  Page 57  |  Page 58  |  Page 59  |  Page 60  |  Page 61  |  Page 62  |  Page 63  |  Page 64