October 2011
NON-CONFIDENTIAL

Business
Model
• Targeting large markets characterized by great unmet medical needs
• In-licensing drug candidates with investor exit in mind; 4-year timeframe
Lead product
FPI-01
• Developed at Memorial Sloan Kettering Cancer Center
• Compelling survival data in pilot study for first-remission Acute
Myeloid Leukemia (AML)
Management
• Operating as a virtual company; 3 FTEs + 5 contractors initially
• Seasoned management team, with on average > 20 years of functional-area experience
• Seeking a $0.5 million Series A, preceding $13MM Series B
2
NON-CONFIDENTIAL

3
Name / organization
Present affiliation
Functional role years of experience
Area of expertise Past affiliations
Maurits Geerlings,
MD, MBA (*)
Chief Executive Officer;
Co-Founder
>17 yrs
General management in lifesciences industry; investor relations; corp. development
Infinity Pharma, Cephalon, Prism
Pharma, Alexion Pharma, Actinium
Pharma, Memorial Sloan Kettering
Cancer Center
Giorgio Mosconi,
Steve Feder,
GenCounsel LLC
Eric Steager,
Esq.
MBA
MD, PhD (*)
Ann Lee Cahill
Clinical Development Group LLC
President & COO; Co-
Founder
>20 yrs
SVP, Clinical
Development
> 17 yrs
General Counsel &
Secretary
>20 yrs
SVP, Finance &
Treasurer
> 19 yrs
Pharmaceutical operations; regulatory affairs (US & EU); business development
Pierrell S.p.A., Acureon, Vicuron;
BristolMyersSquibb; Biosearch
Italia; Marion Merrell Dow
Clinical Development &
Operations
Vion Pharmaceuticals, Schering
Plough, New Britain General
Hospital
Corporate legal affairs, deal making , compliance
Safeguard Scientifics, Pepper
Hamilton LLP, Ballard Spahr LLP,
White and Williams LLP
Financial management, corporate finance, financial planning & analysis
GPX Enterprises, Safeguard
Scientifics, Ankaa Capital Partners,
Banc One, Ernst & Young, Capital
Valuation Group
NON-CONFIDENTIAL

• Corporate
– Martyn Greenacre – formerly, Chairman SmithKlineBeecham-Europe *
– Dave Williams – formerly, Chairman & CEO of Sanofi Pasteur
– Frederick (“Fred”) Frank – Vice Chairman at Peter J. Solomon Company
– Tommy Thompson – formerly, US Secretary of Health & Human Services
– Adelene Perkins – CEO, Infinity Pharmaceuticals
– Warren Cooper – formerly CEO, Prism Pharmaceuticals
– Richard (“Dick”) Sherman – Venture Partner, SCP Partners
– Mickey Flynn – formerly, President & current Vice-Chair of Pennsylvania-BIO
– Steve Goodman – Partner at Morgan Lewis Bockius, LLP
• Clinical & Regulatory (partial list)
– Bruce Burlington – formerly Deputy Director, CBER Division of FDA; formerly
EVP of Regulatory Affairs & Quality Controls at Wyeth Pharma
– Andrea Biondi – Professor, University of Milan; San Gerardo Hospital
4 * Also on Formula Board of Directors
NON-CONFIDENTIAL

Completed phase-1 trial at MSKCC:
Median overall survival
not yet reached at 54+ months
MSKCC pilot study: 5 of 9 patients were ≥ 64 years old; 7 of 9 patients had intermediate-risk cytogenecity
Expected median overall survival in comparable patients receiving standard of care: 9 – 12 months
Age < 56 yo 56-65 yo 66-75 yo >75 yo
No. of patients 368 246 274 80
Median survival, mo.
(95% CI)
18.8
(14.9-22.6)
9.0
(8.1-10.2)
6.9
(5.4-7.7)
3.5
(1.4-6.1)
5
Kantarjian H et al. Cancer 2006; 106:1090-1098 Appelbaum FR et al. Blood 2006;
107:3481-3485
NON-CONFIDENTIAL

An effective target-specific immune response
• Four synthetic peptides targeting Wilms ’ Tumor 1 (WT1) antigen
• WT1 over-expressed in many hematologic and solid tumors
• Designed to make for a powerful immune response against WT1
• Treating patients with minimal residual disease following standard chemotherapy and who cannot further tolerate cytotoxic therapy
• Preventing relapse = extending overall survival
• No products approved in the US for 1 st remission AML
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NON-CONFIDENTIAL

•
•
•
• Combinable with standard of care (safe; no change in practice)
• Targeting minimal residual disease settings
Composition of matter patent protection
√
√
√
• Off-the-shelf product
Measurable biomarkers specific for product
Clear regulatory guidance for clinical development path
√
√
√
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NON-CONFIDENTIAL

Combined Revenue Forecast in 7 largest markets
FPI-01 in AML Forecast Assumptions
Launch
Yearly Incident Growth
2018
0.8%
>60 Years AML
WT1 Expression
CR1
$/Injection
Price Growth
Average Injections
Revenue per Patient
Peak Penetration (2022)
75%
80%
40% - 70%
$8,000
1%
8
$64,000
50%
Yearly Incidence 1
United States
Japan
G7
2002 2010f 2013f 2016f 2019f
11,578 13,023 13,593 14,167 14,740
3,054 3,173 3,204 3,228 3,242
26,586 29,119 30,039 30,931 31,790
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(1) Datamonitor, Pipeline Insight: Leukemias, March 2010
NON-CONFIDENTIAL

• Limited competition exists for agents seeking to prevent a relapse following the 1 st remission in AML patients
1 st Line
2010 Patient incidence (G7)
Standard of care
29,000
Anthracycline/cytarabine
1 st Remission
20,500 none
Agents in development by stage
Phase III: 3
Phase II: 20
Phase I: 11
Pre-phase III: 1
(Ceplene)
Phase II: 2
Phase I: 2
Total agents in development
35 5 (*)
2 nd Line
24,000
IDAC, HDAC, LDAC, HSCT
Hypomethylating agents
Phase III: 3
Phase II: 35
Phase I: 22
61
Sources: Datamonitor, Medtrack, Kelley WN (ed): Textbook of Internal Medicine .
(*) Among development-stage competitors for1 st remission AML most are autologous, dentritic cell-based vaccines
NON-CONFIDENTIAL

Phase I
First-remission AML Completed
Mesothelioma
Melanoma
Elderly ALL
Ovarian cancer
Completed
Phase II
Formula start 2Q2012
MSKCC started
1Q2011
MSKCC started
2Q2011
Planned
Planned
Planned
10
MSKCC sponsored trials
NON-CONFIDENTIAL

2011 2012 2013 2014 2015
Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2
$1.1MM
$10MM
$5.4MM
.
1/1/2012 beginning cash
$12.6MM operating budget
$3MM
$4.3MM
$2.9MM
11
Start
Immune
Response
SOPs
Start Event
Complete Event
Start companysponsored
Ph2 AML trial
Orphan
Status application
Complete ph-2 AML trial enrollment
Start second indication phase-2 trial
Complete
Ph2 AML trial
M&A or
Strategic
Partnership
Regulatory Event
Expense
Business Event
NON-CONFIDENTIAL
Start Ph3

• FPI01 program designed with investors’ exit strategy in mind
• Potential for up to 10x return, upon positive completion of Formula’s planned randomized controlled phase-2 trial
• Management has deep experience in strategic licensing and M&A
Recently closed deals by companies roughly comparable with Formula’s projected business and product profile:
12
NON-CONFIDENTIAL

• Strategic partnering with pharma to finance phase-2 trial and to broaden clinical indications
– Preliminary discussions with various large multinational biopharmaceutical companies have initiated
• Broaden pipeline by acquiring additional affordable high-quality product candidates
– Formula has identified promising acquisition candidates that could fit with it’s investment philosophy (near-term exit, high-value inflection point)
• Raising non-dilutive capital from government- (e.g. SBIR, HHS, NCI) and Non-Government Organizations (e.g. CVAF, LLS, CPRIT)
• Sign-off from FDA on clinical development path towards approval, following recent positive FDA meeting
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NON-CONFIDENTIAL

• Virtual company developing a clinical stage product that has shown a compelling survival benefit in AML patients
• Strong scientific and clinical rationale, supported by scientific integrity of Memorial Sloan Kettering Cancer Center
• Experienced operational and business team
• Straight development path to exit within a 4 year timeframe
• Seeking $500,000 to supplement recently raised Series A round, which precedes an $13 million Series B financing by 4Q2011
• Sustainable business model through additional product candidates
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NON-CONFIDENTIAL

Formula Pharmaceuticals, Inc.
Three Westlakes, 1055 Westlakes Drive, 3 rd Floor
Berwyn, PA 19312 http://www.formulapharma.com
15
Maurits W. Geerlings, MD, MBA
Chief Executive Officer
Ph: 610-727-4172
Fx: 610-23-0111
Mobile: 301-461-7828 mgeerlings@formulapharma.com
NON-CONFIDENTIAL