A Rare Request, an Orphan Offer

A press release last week has prompted me to get on my “rare disease” soap box again.  In previous posts, I noted that drug development for rare and orphan diseases (but not the neglected diseases of poverty that are rare in the US) has become one of the saviors of the pharma industry (or at least its profits) with one-third of the drugs approved in 2011 being for rare disease (“Orphaned and Neglected” in February 2012).  Of course, the venture capital industry took note of this opportunity and has been investing in early-stage companies developing such drugs, but last week’s announcement showed that VC-types are also good at structuring new business models to accelerate and “de-risk” drug development.  My request (naïve hope) is that they apply their smarts to drug development for the orphan, neglected diseases (NDs) of the rest of the world and add some push for the pharma/biotech industry into the global health business.

The press release was from a new company called Cydan located in our local biotech hotbed of Cambridge.  While the company website has little additional information (Cydan), the release noted that Cydan will be “an orphan drug accelerator that identifies and de-risks programs with therapeutic and commercial potential” with such programs coming from academia and other companies but presenting “well-understood biology and proof-of-concept data in in vivo models” (Cydan PR).  Cydan will conduct its preclinical studies via contract development outfits to “inform definitive ‘go’ or ‘no go’ development decisions” and will start up companies based on the most promising programs.  Cydan’s management has strong drug development credentials.  Cristina Csimma, the CEO, has start-up and big pharma experience (I know her slightly from our overlapping stints at Genetics Institute/Wyeth) and James McArthur, CSO, and Deborah Geraghty, Vice President, Project and Portfolio Development, have multiple start-up company experience.  More importantly the company has good initial financial backing, $16 million led by the VC firm, New Enterprise Associates (NEA), which has the deepest pockets among the VCs for biotech investment.  NEA’s raising of $2.6 billion in 2012 means it could funnel more than $700 million into biotech companies in the coming years (FierceBiotech article).  The other participants are Pfizer Venture Investments, suggesting a corporate acquisition pipeline, and Alexandria Real Estate Equities, Inc., the dominant Kendall Square (Cambridge) lab space developer with more than 2 million square feet occupied and 1.7 million planned and approved (Alexander Real Estate PR).

Will Cydan refute conventional wisdom and take up ND projects where the profit margins are likely less than those for the rare diseases and more difficult to assess?  Not likely but NEA is not totally unfamiliar with the global pharma market, having offices in India and China and having made a investments in Chinese service companies, a few anti-infectives and needle-less delivery companies, and in one US company, Liquidia Technologies, that has a collaboration with PATH on pneumonia vaccines (NEA portfolio and Liquidia press release).  Also the NEA principals could be inclined to try a ND opportunity or two knowing that assessing commercial potential for programs that will result in drugs, even with accelerated development, in 6-10 years is difficult and a market for ND drugs could develop by then.  They are also likely aware that US insurers are increasingly expecting new drugs to provide measurable improvements in quality of life and that there will be competition even for rare disease drug price coverage (e.g., Sanofi and Agenion are competing on price for their recently approved products for familial hypercholesterolemia [FiercePharma article]).

Undoubtedly, the founders of Cydan have list of rare disease program opportunities they are working, but I read the press release as implying that interested parties may submit their nominees for consideration.  What global health/neglected disease opportunities are there that may meet Cydan’s criteria of having with well-understood biology, proof-of-concept data in in vivo models, and a “characterized genetic etiology?”  The last is a sticky point and the only one I can think of is sickle cell anemia in which suffers have retained expression of a form of hemoglobin which confers some protection against malaria but also decreases blood flow and increases the severity of infections in African kids (see my post, “A Really Neglected Disease”‘ July 2010).  However, if the last criterion includes diseases in which the target organism has genetic characterization, there are more opportunities.  One may be drugs for hepatic C virus infection for which there are many early- (and late-) stage drug development programs and for which I wrote up a not too-unreasonable justification of commercial potential outside the developed markets (“Rolling the Dice” Aug 2012).  There are also the several families of compounds being pursed by the non-profit neglected disease drug developers that may be stuck in the preclinical stage for lack of funding or expertise (e.g., DNDi portfolio and MMV portfolio).  There are also a bunch of academic labs trying their hand at drug development for neglected diseases, e.g. Michael Pollastri at Northeastern (Pollastri lab) and the many listed in the BVGH Global Health BVGH Primer (BVGH Primer).  If anyone has a program she/he wants to submit for Cydan’s consideration, I’m happy to help with the pitch.



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