Prize4life - November 14th


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Prize4life - November 14th

  1. 1. THE NEXT ALS BREAKTHROUGH COULD BE YOURS Prize4Life: on the road to finding a cure to ALS disease Presentation to MIT Enterprise Forum Israel Nov. 2012 PRIZE4LIFE.ORG
  2. 2. What is ALS (Lou Gehrig’s disease)?  A neurodegenerative disease, Amyotrophic lateral sclerosis (ALS) is also referred to as ‘motor neurone disease‘  Rapid and progressive paralysis of unknown cause, 100% lethal  Death within 2-5 years from diagnosis (respiratory failure)  First clinically identified in 1869--over 140 years ago  Only existing FDA-approved treatment prolongs life by 2-3 months  Affects both men and women primarily in mid- life but can strike at any adult age (including rarely in teenagers) PRIZE4LIFE.ORG
  3. 3. ALS presents a clear unmet medical need No • No disease modifying therapies available treatment • ALS strikes as many people as Multiple Sclerosis, and more people than NeedFit with Huntington’s Disease, but because ALS is so rapidly fatal, at any given time increasingTeva there are fewer people alive with ALS • Orphan disease with estimated 30,000 individuals affected in the U.S. at any given time and 600,000 worldwide • In the industrialized world, more than one in 500 people die of ALS • With aging, prevalence of ALS- and associated costs- are likely to increase • Disease cost is extremely high for patients and families – financial and High cost emotional burden • $160,000/year for in-home treatment; US$430,000/year for institution-based treatment PRIZE4LIFE.ORG
  4. 4. So what’s holding us back? The challenges facing ALS are typical of those in the broader neuro space Drug development  The brain is hard to access  High fail rate is hard  Expensive  Complex and unpredictable regulatory environment  Hard to cross the “Valley of Death”  The nervous system is REALLY complex Fit with Teva Basic research  Animal models have not yet led to treatments is hard  Multiple levels of analysis difficult to integrate Incentives are  Publish or perish  Incentives for data sharing often lacking misaligned  Few data standards  Clinical research viewed as less attractive than basic research 4 PRIZE4LIFE.ORG
  5. 5. Due to these challenges, innovation in basic researchoften isn’t translated into direct patients’ benefit Research – Development - Access: Universities/ “Valley Health Systems Research of Biotech – Hospitals Institutions Death” Pharma Health Services “Valley of Death” = gap between academic research and industry involvement Without industry commitment, basic research does not get translated into tangible results for patients PRIZE4LIFE.ORG
  6. 6. The drug discovery gap: valley of death Basic Research • Academic researchers Academia have typically focused on Disease Mechanism understanding biological fundamentals and Target Identification disease mechanisms • Industry focus is now on Assay Development the later stages of drug development Valley of Death Screening • This gap is particularly Lead Optimization worrisome in the context of ALS and other Preclinical Development neurodegenerative disorders Clinical Trials Industry Submission/ Approval PRIZE4LIFE.ORG
  7. 7. Prize4Life: an organization with the sole purposeof being terminated  Prize4Life is an innovative, results-oriented 501(c)(3) not-for- profit organization focusing exclusively on ALS  Founded by a group of Harvard Business School students in 2006 after one of their classmates—Avichai (Avi) Kremer— was diagnosed with ALS at the age of 29  Prize4Life has embraced the Incentive Prize Model to change the landscape for ALS research  We are small but have grown quickly from 2 staff (2007), to our current staff of 6 (2012). P4L has raised +$9M for ALS since 2005 PRIZE4LIFE.ORG
  8. 8. From the get-go, Prize4Life’s mission statement wasdeveloped to be both inspirational and pragmatic Mission Statement To accelerate the discovery of treatments and a cure for ALS by using powerful incentives to attract new people and drive innovation CONFIDENTIAL PP R I Z E 4 L I F E . O R G RIZE4LIFE.ORG
  9. 9. Prize4Life’s approach: how can we make ALSbreakthroughs more likely  Triple mission  Accelerate existing efforts in ALS research o Create new tools o Promote new collaborations  Bring new ideas and new minds into the field  Complement existing funding models o Draw attention to ALS research o Raise funding from previously untapped sources  Leverage is our operating principle  Inclusive approach  All of Prize4Life’s programs are designed to accelerate ALS therapy development across the board and are open to all (“lift all boats”, “we don’t bet on one horse, we focus on improving the entire track”) 9 PRIZE4LIFE.ORG
  10. 10. Over the years, Prize4Life has been launching incentiveprizes and spearheaded several infrastructure program Ongoing Incentive Prize Programs  $1M ALS Biomarker Prize (2011 Awarded)  $1M ALS Treatment Prize  ALS Mouse Colony  ALS Mouse Manual  ALS Prediction Prize (algorithm/”big data” challenge, awarded November 13, 2012(!)) Infrastructure Programs  PRO-ACT Database  ALS Forum Portal  ALSGene Database  Alzforum Science Writer Collaboration  Bi-weekly E-Newsletter 10 PRIZE4LIFE.ORG
  11. 11. Prize4Life: focus on current programmatic portfolio • $1M ALS Treatment Prize; Re-launched June 2012 Pre clinical • Supported by the ALSGene, The ALS forum and the ALS mouse colony. science • Allows P4L to understand, define, engage and incentivize the ALS preclinical community and to bridge between the ALS research community and relevant industry • PRO-ACT Database: Over 8,500+ records of patients from 20+ clinical trials from Teva,PRO ACT Sanofi Aventis, Novartis, Regeneron and NEALS; to be launched early 2013database • ALS Prediction Prize; Launched July 2012 and won Nov. 2012. Attracted over 750 solvers; Can reduce clinical trials variability by up to ~25%. • Allows P4L to support finding biomarkers, progression algorithms, stratification for personal medicine, stratification of drug responses and morePromoting • Avichai Kremer awarded 2012 Prime Minister award for innovation andresearch entrepreneurship in a non profitfunding • Avichai has been involved in fostering ALS research such as through ISF • Prize4Life is developing advocacy efforts regarding an Israeli “Orphan Disease Act” and efforts to promote foreign investment in Israeli orphan disease R&D. • Allows P4L to support funding and remove barriers for R&D of orphan diseases PRIZE4LIFE.ORG
  12. 12. Prize4Life is constantly monitoring global ALS research so itcan develop the best programs to accelerate research Drug and Biotech companies currently investing in ALS R&D PRIZE4LIFE.ORG
  13. 13. What is an incentive prize?What is an incentive prize Benefits of an incentive prize • An effective form of crowd- • Democratic funding model which does not sourcing/open innovation pre-select a winner/idea based on any • Crowd-funding particular criteria (anyone can compete) • Open source movement unlike other up-front funding models, which are often predicated on prior results • A results-based reward for a very or pedigree or networks specific pre-determined need (not a recognition prize like the Nobel Prizes) • Attracts new minds, new media, and new money • An example of “pull” funding vs. “push” (upfront) funding (e.g. grant or contract) • Encourages outside-of-the-box thinking • A mechanism to create excitement, • Complimentary to existing funding models momentum, competition, and attention • Results focused (you get what you want • Prize purses can range from 0 to or you don’t pay) millions of dollars • Efficient from a funder/donor perspective PRIZE4LIFE.ORG
  14. 14. Large incentive prizes throughout history PRIZE4LIFE.ORG
  15. 15. The incentive prize model has grown exponentially overthe last fifteen yearsAggregate prize purse, prizes over $100,000 Large prize purses by sector 18% CAGR 18% CAGR Source: Set of 219 prizes worth >$100,000 from “And the Winner Is …”, McKinsey, 2009 PRIZE4LIFE.ORG
  16. 16. History of the ALS biomarker prize challenge  Prize4Life launched the $1,000,000 ALS Biomarker Prize in November 2006  Challenge was for an ALS Biomarker capable of reducing the cost of ALS clinical trials by 50% or greater  Five $15,000 “idea prizes” awarded in May 2007 to researchers who submitted theoretical solutions to finding a biomarker including: o Plant biologist o Chemist o Dermatologist o Small biotech o Anonymous solver  54 teams from 18 different countries around the world actively competed  27 of these teams initiated interdisciplinary collaborations  65% of teams competing for the prize came from a discipline other than ALS (new minds!)  60% of teams competing for the prize came from academia and 40% from other domains (industry, gov’t, individuals) PRIZE4LIFE.ORG
  17. 17. History of the ALS biomarker prize challenge – cont’d  ALS Biomarker Prize closed in November 2008  12 submissions received from 7 countries  In 2009 Prize4Life awarded $100,000 in Progress Prizes to two teams for a: o Novel skin-based biomarker o Novel electrophysiological based biomarker  Relaunched the $1,000,000 Prize4Life ALS Biomarker Prize in May 2009  Over 1000 potential teams viewed the prize criteria  Received 4 submissions within the first year  Submissions vetted by Prize4Life’s SAB, expert biostatisticians, and several additional outside experts  One submission was deemed to have met all 5 basic requirements and 5/7 desirable features  ALS Biomarker identified and $1M Prize Challenge officially awarded in February, 2011 17 PRIZE4LIFE.ORG
  18. 18. Pooled Resource Open-Access ALS Clinical Trials(PRO-ACT) Project: Prediction Prize >> Won yesterday!! ALS Prediction Prize Overview  The ALS Prediction Prize was designed to capitalize on the creation of the PRO-ACT Database and attract widespread attention to the database (and ALS) from various quantitative communities (statisticians, computer scientists, bioinformatics specialists, computational neuroscientists, etc.). The 50K prizes were given for the best prediction algorithm(s) enabling prediction of the degree of change in a patient’s disease status over the following year (with an ultimate goal of improving ALS clinical trials). Prize Details:  The algorithm needs to predict a patient’s individual progression, using data only from the first three months of clinical data collection  The prize was launched on July 15th in collaboration with IBM’s DREAM Project (Dialogue for Reverse Engineering Assessments and Methods) and using the InnoCentive platform  Three winners announced Nov. 13 out of ~1040 solvers registered for the competition and 25 unique solvers who have submitted an algorithm for testing and ranking on a leaderboard  Active solvers currently competing from AMIA, IBM, Microsoft, Google, MIT, Stanford, Princeton, and many other leading institutions around the world18 CONFIDENTIAL P R I ZI E 4 L ILFI E . O O G PR ZE4 FE.R RG
  19. 19. Key lessons learned  There are multiple kinds of crowdsourcing; prizes are just one example  Crowdsourcing can be used successfully to address complex biological questions/problems but it isn’t easy  It is ok to increment a challenge/divide a problem into intermediate steps and milestones  Defining and articulating your goal/question/challenge carefully is key  Lower barriers to entry whenever possible (to maximize the benefits of using the incentive prize model you want a large diverse pool of solvers)  Marketing  IP policy  Access to resources  Maintain momentum (especially for large/multi-year challenges)  Big and small incentives can both work (depends on the complexity and resource requirements of the desired solution)  The “community” formed by crowdsourcing competitions is typically ephemeral but ties to the challenge/problem, as well as to the entity posting the challenge, can be lasting PRIZE4LIFE.ORG