prize4life - november 14th

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P R I Z E 4 L I F E . O R G P R I Z E 4 L I F E . O R G 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

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Page 1: Prize4life - November 14th

P R I Z E 4 L I F E . O R G P R I Z E 4 L I F E . O R G

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

Page 2: Prize4life - November 14th

P R I Z E 4 L I F E . O R G P R I Z E 4 L I F E . O R G

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)

What is ALS (Lou Gehrig’s disease)?

Page 3: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

• No disease modifying therapies available

• ALS strikes as many people as Multiple Sclerosis, and more people than Huntington’s Disease, but because ALS is so rapidly fatal, at any given time 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 emotional burden

• $160,000/year for in-home treatment; US$430,000/year for institution-based treatment

High cost

Fit with

Teva

No treatment

Need increasing

ALS presents a clear unmet medical need

Page 4: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

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The challenges facing ALS are typical of those in the broader neuro space

So what’s holding us back?

Incentives are misaligned

Fit with Teva

Drug development is hard

Basic research is hard

The brain is hard to access High fail rate Expensive Complex and unpredictable regulatory environment Hard to cross the “Valley of Death”

The nervous system is REALLY complex Animal models have not yet led to treatments Multiple levels of analysis difficult to integrate

Publish or perish Incentives for data sharing often lacking Few data standards Clinical research viewed as less attractive than basic research

Page 5: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

“Valley of

Death”

Research – Development - Access:

“Valley of Death” = gap between academic research and industry

involvement

Without industry commitment, basic research does not get

translated into tangible results for patients

Universities/ Research

Institutions

Biotech – Pharma

Health Systems Hospitals

Health Services

P R I Z E 4 L I F E . O R G

Due to these challenges, innovation in basic research

often isn’t translated into direct patients’ benefit

Page 6: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

The drug discovery gap: valley of death

• Academic researchers have typically focused on understanding biological fundamentals and disease mechanisms

• Industry focus is now on the later stages of drug development

• This gap is particularly worrisome in the context of ALS and other neurodegenerative disorders

Basic Research

Disease Mechanism

Target Identification

Assay Development

Screening

Lead Optimization

Preclinical Development

Clinical Trials

Submission/ Approval

Academia

Industry

Valley of Death

Page 7: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

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: an organization with the sole purpose of being terminated

Page 8: Prize4life - November 14th

P R I Z E 4 L I F E . O R G CONFIDENTIAL

Mission Statement

To accelerate the discovery of treatments and a cure for ALS by using powerful incentives to

attract new people and drive innovation

P R I Z E 4 L I F E . O R G

From the get-go, Prize4Life’s mission statement was developed to be both inspirational and pragmatic

Page 9: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

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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”)

Prize4Life’s approach: how can we make ALS breakthroughs more likely

Page 10: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

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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

Over the years, Prize4Life has been launching incentive prizes and spearheaded several infrastructure program

Ongoing

Page 11: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

• $1M ALS Treatment Prize; Re-launched June 2012

• Supported by the ALSGene, The ALS forum and the ALS mouse colony.

• 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, Sanofi Aventis, Novartis, Regeneron and NEALS; to be launched early 2013

• 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 more

• Avichai Kremer awarded 2012 Prime Minister award for innovation and entrepreneurship in a non profit

• 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

Pre clinical science

Promoting research funding

PRO ACT database

Prize4Life: focus on current programmatic portfolio

Page 12: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

Drug and Biotech

companies

currently

investing in ALS

R&D

Prize4Life is constantly monitoring global ALS research so it can develop the best programs to accelerate research

Page 13: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

What is an incentive prize?

Benefits of an incentive prize

• Democratic funding model which does not

pre-select a winner/idea based on any

particular criteria (anyone can compete)

unlike other up-front funding models,

which are often predicated on prior results

or pedigree or networks

• Attracts new minds, new media, and new

money

• Encourages outside-of-the-box thinking

• Complimentary to existing funding models

• Results focused (you get what you want

or you don’t pay)

• Efficient from a funder/donor perspective

What is an incentive prize

• An effective form of crowd-

sourcing/open innovation

• Crowd-funding

• Open source movement

• A results-based reward for a very

specific pre-determined need (not a

recognition prize like the Nobel Prizes)

• An example of “pull” funding vs. “push”

(upfront) funding (e.g. grant or contract)

• A mechanism to create excitement,

momentum, competition, and attention

• Prize purses can range from 0 to

millions of dollars

Page 14: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

Large incentive prizes throughout history

Page 15: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

18% CAGR

Source: Set of 219 prizes worth >$100,000 from “And the Winner Is …”, McKinsey, 2009

Aggregate prize purse, prizes over $100,000 Large prize purses by sector

18% CAGR

The incentive prize model has grown exponentially over the last fifteen years

Page 16: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

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)

History of the ALS biomarker prize challenge

Page 17: Prize4life - November 14th

P R I Z E 4 L I F E . O R G 17

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

History of the ALS biomarker prize challenge – cont’d

Page 18: Prize4life - November 14th

P R I Z E 4 L I F E . O R G CONFIDENTIAL 18 P R I Z E 4 L I F E . O

R G

CONFIDENTIAL

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 world

Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) Project: Prediction Prize >> Won yesterday!!

Page 19: Prize4life - November 14th

P R I Z E 4 L I F E . O R G

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

Key lessons learned