Incentives for Starting Small Companies Focused on Rare and Neglected Diseases

Abstract

Starting biotech or pharmaceutical companies is traditionally thought to be based around a scientist, their technology platform or a clinical candidate spun out from another company. Between us we have taken a different approach and formed two small early stage companies after initially leveraging the perspective of a parent with a child with a life-threatening rare disease. Phoenix Nest (http://www.phoenixnestbiotech.com/) was co-founded to work on treatments for Sanfilippo syndrome a devastating neurodegenerative lysosomal storage disorder. In the space of just over 3 years we have built up collaborations with leading scientists in academia and industry and been awarded multiple NIH small business grants. The second company, Collaborations Pharmaceuticals Inc. (http://www.collaborationspharma.com/) was founded to address some of the other 7000 or so rare diseases as well as neglected infectious diseases. The Rare Pediatric Disease Priority Review Voucher is likely the most important incentive for companies working on rare diseases with very small populations. This may also be partially responsible for the recent acquisitions of rare disease companies with late stage candidates. Lessons learned in the process of starting our companies are that rare disease parents or patients can readily partner with a scientist and fund research through NIH grants rather than venture capital or angel investors initially. This process may be slow so patience and perseverance is key. We would encourage other pharmaceutical scientists to meet rare disease parents, patients or advocates and work with them to further the science on their diseases and create a source of future drugs.

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Abbreviations

eRA:

Electronic research administration

FDASIA:

Food and drug administration safety and innovation act

IND:

Investigational new drug application

MPS:

Mucopolysaccharidosis

NDA:

New drug application

NIH:

National institutes of health

NME:

New molecular entities

SBIR:

Small business innovation research

STTR:

Small business technology transfer

VC:

Venture capital

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ACKNOWLEDGMENTS AND DISCLOSURE

Much of what we have described would not have been possible without the generosity, collaboration and support of many colleagues globally who have played a major role from the beginning including: Dr. Alexey Pshezhetsky, Dr. Brian Bigger, Dr. Patti Dickson, Dr. Xiaoyi Zhang, Dr. Tsui-Fen Chou, Mr. Derek Moen, Dr. Steven Le, Dr. Shih-hsin Kan, Dr. Matthew Ellinwood, Dr. Alex Clark, Dr. Joel Freundlich, Dr. Ruben Flores, Susan Rubin, Allison Weber, Calvin Chen, Mary F. Ognibene, Allison Moore, Lori Sames, Rare Genomics Inst., Assay Depot, Taconic, Jonah’s Just Begun, JLK-Sanfilippo Research Foundation, Sanfilippo Sud, Sanfilippo Barcelona, Sanfilippo Portugal, National MPS Society, The Lukondi family, our scientific advisory board members, boards of directors and all the families with Sanfilippo Syndrome. Michele Rhee is kindly acknowledged for creating Fig. 2 and her support. SE and JW are co-founders of Phoenix Nest, Inc. SE is a founder of Collaborations Pharmaceuticals, Inc.

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Correspondence to Sean Ekins.

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NIH NINDS 1R41NS089061-01

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Ekins, S., Wood, J. Incentives for Starting Small Companies Focused on Rare and Neglected Diseases. Pharm Res 33, 809–815 (2016). https://doi.org/10.1007/s11095-015-1841-9

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KEY WORDS

  • entrepreneurship
  • neglected diseases
  • rare diseases
  • Sanfilippo syndrome
  • Small companies