Business


Examples of platform-DDD companies Historically, there have been numerous attempts at creating PD3 companies resulting in mixed out- comes. One of the first such innovative companies was a Noubar Afeyan (Flagship Ventures, now renamed Flagship Pioneering) creation, namely Beyond Genomics. This was the world’s first Systems Biology company that attempted a foray into PD3. The history, background and haphazard evolution of Beyond Genomics’ effort is provided in more detail in the side panel titled ‘The confused utilisation of the Beyond Genomics platform’. The problems encountered by Beyond Genomics were myriad in nature. While its systems biology plat- form was highly innovative, the company ultimate- ly failed in its PD3 efforts because it was not pur- pose-built


for DDD. In stark contrast,


CombinatoRx was a purpose-built PD3 company, and many consider it the archetypical example. This is explained in more detail in the side panel entitled ‘The rise and demise of the CombinatoRx platform-DDD efforts’. The


failure of


CombinatoRx was not due to its initial PD3 efforts and spectacular productivity of combination can- didate drugs for a variety of disease indications. The company floundered on the jagged rocks of drug development. In the case of both Beyond Genomics and CombinatoRx, analysis of their fail- ures is instructive. It highlighted the fact that to build a successful PD3 company requires a myriad of skill sets and expertise, and was certainly part of the reason why many analysts in the mid-2000s were skeptical about such endeavours as discussed in the opening section of this article. Currently, it is estimated that there are more than


100 different PD3 companies. At least five of these companies are classified as ‘Unicorns’, which refers to a privately-held start-up/early-stage entity valued at more than $1 billion. As we noted above, the plat- forms of such companies can be technology, biologi- cal, molecular or digital-based or some hybrid com- bination. The size and valuation of these companies spans a considerable range that includes behemoths such as publicly-traded entities like Ionis Pharmaceuticals (http://www.ionispharma.com/), Akcea Therapeutics (https://akceatx.com/) and Ablynx (http://www.ablynx.com/). However the list also contains much smaller, privately-held companies such as Sirenas (http://sirenasmd.com/) and Melior Pharmaceuticals (http://www.meliorpharmaceuti- cals.com/). More recently, the authors founded iMetabolic


Biopharma Corporation (https://imbiopharma. com). We are a precision medicine PD3 company intent on developing biological therapeutics for


Drug Discovery World Fall 2018


The confused utilisation of the Beyond Genomics platform


Noubar Afeyan, (CEO of Flagship Ventures) founded Beyond Genomics Inc in February 2000. One of us (Dr Stephen Naylor) was part of the scientific founding team and served as the CTO for the early growth phase of the company. Beyond Genomics was the world’s first systems biology company with a purpose-built plat- form, consisting of gene, protein and metabolite analysis capabilities, and bioinfor- matic, biostatistics, knowledge management and data visualisation tools. The proof- of-principle for this integrated platform was the systems analysis of the Apolipoprotein E3-Leiden transgenic mouse. Initially the company utilised the plat- form on independent, disease-specific research that included Alzheimer’s Disease, atherosclerosis and oncology. The company also collaborated with numerous phar- maceutical companies, leveraging its platform across a portfolio of applications in DDD, biomarker discovery and diagnostics. In June 2002, Beyond Genomics recruited Dr James Hauske as CSO. Hauske had


a rich pedigree in DDD, with companies such as Sepracor and Pfizer on his resume. This hire reflected intent by the company to utilise its in-house systems biology platform for DDD purposes. These activities were reinforced in February 2005 when the company changed its name to BG Medicine and hired a new President, Dr Pieter Muntendam. He was charged with leading the company’s business operations and strategy and to focus on high-value drug development applications using its pro- prietary ‘Systems Pharmacology’ platform. However, by 2008 another change in direction occurred and the company raised $40 million in new capital to help com- mercialise its first product, Galactin-3, a predictive biomarker of congestive heart failure. The company platform had morphed into a biomarker discovery engine. BG Medicine aggressively pursued development of Galactin-3 and it was granted FDA 501(k) approval in November 2010, followed shortly thereafter by a successful IPO, raising $40.25 million in February 2011. Initial efforts to implement a platform-driven DDD initiative proved to be elusive


for Beyond Genomics/BG Medicine. The systems biology platform developed by the company was innovative but was not purpose-built for DDD. A more pragmatic biomarker and diagnostics platform driven focus led to considerable business suc- cess and a successful IPO. However, it is noteworthy that its one product, Galectin- 3, was actually in-licensed from ACS Biomarker BV (The Netherlands). At the end of 2015, BG Medicine was delisted from the NASDAQ and the stock is now listed on the OTC market and currently trades at ~3-5 cents per share. Mixed fortunes for a highly-innovative company, that lacked an integrated and sustainable plan for its first-in-class platform utilisation, particularly in DDD.


the treatment of obesity-related diseases. Currently we are focused on the treatment of the hyperlipidemic


disorder Familial


Chylomicronemia Syndrome. We are assembling a platform that contains functional modules that span across a sophisticated Artificial Intelligence (AI) discovery capability coupled with a protein


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