Investor Presentation $13 Million Series B Round

November 2011

NON-CONFIDENTIAL Formula Pharmaceuticals – Investment Opportunity

• Lead product has peak sales potential exceeding $500 million for each targeted disease indication with great unmet need Opportunity • Investor exit opportunity possible within 3 to 4 years • Additional pipeline products through in-licensing, based on strong academic and corporate relations

• Developed at Memorial Sloan Kettering Cancer Center Lead • Early survival data in pilot study for first-remission Acute Myeloid product Leukemia (AML): 54+ months vs. 3-19 months for standard of care FPI-01 • Phase-2 MSKCC-sponsored trials started in AML & mesothelioma • Composition of matter patents issued (2026 expiry) and pending

• Strong and experienced team operating within a virtual structure Management • Seeking a Series B financing of $13MM to fund Phase-2 clinical development

2 NON-CONFIDENTIAL Formula Pharma – Significant Developments In 2011

• Series A financing closed in July, providing runway into mid-2012

• Positive FDA feedback on phase-2 clinical development plan

• Partnering interest from several large pharmaceutical companies

• Industry leaders serving as Directors and as Corporate Advisors

• Cancer vaccine field has progressed, showing sizable investor exits

• Attractive near-term exit planned, based on a $13MM Series B round

3 NON-CONFIDENTIAL Formula’s Executive Leadership

Name / organization Functional role Area of expertise Past affiliations Present affiliation years of experience

Infinity Pharma, , Prism Chief Executive Officer; General management in life- Pharma, Alexion Pharma, Actinium Co-Founder sciences industry; investor Maurits Geerlings, MD, MBA Pharma, Memorial Sloan Kettering >17 yrs relations; corp. development Cancer Center

President & COO; Co- Pharmaceutical operations; Pierrell S.p.A., Acureon, Vicuron; Giorgio Mosconi, MD, PhD Founder regulatory affairs (US & EU); BristolMyersSquibb; Biosearch Italia; >20 yrs business development Marion Merrell Dow

SVP, Clinical Clinical Development & Vion Pharmaceuticals, Schering Ann Lee Cahill Development Operations Plough, New Britain General Hospital Clinical Development Group LLC > 17 yrs

General Counsel & Safeguard Scientifics, Pepper Hamilton Corporate legal affairs, deal Steve Feder, Esq. Secretary LLP, Ballard Spahr LLP, White and making , compliance GenCounsel LLC >20 yrs Williams LLP

GPX Enterprises, Safeguard SVP, Finance & Financial management, Scientifics, Ankaa Capital Partners, Treasurer corporate finance, financial Eric Steager, MBA Banc One, Ernst & Young, Capital > 19 yrs planning & analysis Valuation Group

4 NON-CONFIDENTIAL Formula Key Advisors

• 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 Pharma – Andrea Biondi – Professor, University of Milan; San Gerardo Hospital

5 * Also on Formula Board of Directors NON-CONFIDENTIAL Our lead drug candidate, FPI-01 – why did we in-license?

• Phase-1 clinical trial successfully completed

– Median overall survival exceeding 54 months in AML and counting (1)

– Compares with median overall survival ranging between 3 – 19 months, using standard of care (2,3)

– Favorable safety and tolerability profile; combines well with standard of care treatment regimen

• Pilot study; data compelling because of poor prognostic factors (elderly patients; unfavorable histology)

• Potential for synergistic activity between WT1 peptides and approved cancer therapies

(1) Maslak, et al. Blood, 15 July 2010 Vol 116, No 2 (3) Appelbaum FR et al. Blood 2006; 6 (2) Kantarjian H et al. Cancer 2006; 106:1090-1098 107:3481-3485 NON-CONFIDENTIAL FPI-01 – Unique Positioning

Targeting a most significant unmet need in oncology:

Preventing recurrence of cancer

in patients

who achieved complete remission

with standard of care (chemotherapy ± surgery)

7 NON-CONFIDENTIAL

Our Product and Therapeutic Target

• Unique, first-in-class, four-peptide immunotherapeutic that targets Wilms’ Tumor 1 (WT1) antigen

• WT1 over-expressed in many hematologic and solid tumors • AML, acute lymphocytic leukemia (ALL), mesothelioma, ovarian- and various other cancers (expression in up to 93% of patients) • The National Cancer Institute recently recognized WT1 as the most relevant target for cancer immunotherapy 1

• Together, the four peptides make for a powerful mechanism, as they: • Elicit both CD4+ and CD8+ T-cell responses, which act synergistically • Cause T-cells to recognize native WT1, thereby overcoming immune tolerance • FPI-01 is non-HLA restricted; Therefore it applies to approximately 80% of the Western patient population

• Solid phase manufacturing; “single drug” from FDA perspective

8 (1) Clin Cancer Res 2009;15(17):5323–37 NON-CONFIDENTIAL

How FPI-01 Fits Within Standard of Care for AML

• Standard chemotherapy achieves complete remission (CR) in 40-80% of patients, but disease relapses in 80% within weeks to months

• FPI-01 seeks to prevent disease relapse, thereby prolonging survival

Induction Consolidation Relapse

Cytarabine plus an Chemotherapy (e.g. Combination salvage anthracycline (3+7) high-dose ) chemotherapy

e.g. cytarabine plus HSCT if donor HSCT in younger idarubicin, available and patient is patients cytarabine plus fit/young enough daunorubicine Off-label single-agent therapy. E.g. LDAC, HDAC, IDAC, hypomethylators

9 NON-CONFIDENTIAL FPI-01 – Clinical Results From Phase I Pilot Study

Completed phase-1 trial MSKCC pilot study: 5 of 9 patients were ≥ 64 years old; 7 of 9 at MSKCC: patients had intermediate-risk cytogenecity Median overall survival not yet reached at 54+ Expected median overall months 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. 18.8 9.0 6.9 3.5 (95% CI) (14.9-22.6) (8.1-10.2) (5.4-7.7) (1.4-6.1)

Kantarjian H et al. Cancer 2006; 106:1090-1098 Appelbaum FR et al. Blood 2006; 10 107:3481-3485 NON-CONFIDENTIAL What large pharma wants to see – key product features

• 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 √

11 NON-CONFIDENTIAL

Formula’s Pipeline Overview

Phase I Phase II

First-remission AML Completed Formula start 2Q2012

MSKCC started 1Q2011

Mesothelioma Completed MSKCC started 2Q2011

Melanoma Planned

First-remission ALL MSKCC started 1Q2011

Ovarian cancer Planned

12 MSKCC sponsored trials NON-CONFIDENTIAL Formula’s Planned Phase 2 Randomized Trial

• Target Population: AML patients > 60 yrs in first complete remission – Primary objective: median LFS for patients treated with FPI-01 vs. median LFS for patients treated with standard care (observation alone) – Secondary objective: Immune response, safety profile of FPI-01, WT1

expression, and OS • Design – Two arms: 30 patients on FPI-01; 30 patients observation only – Dosing: Six vaccinations over 12 weeks (D 1, 15, 29, 43, 57 and 71), plus monthly thereafter x 6 – Estimated enrollment: 60 patients to be enrolled in 15 US sites • Timing – Estimated starting date: 2Q2012; Estimated completion date: 1Q2015

13 NON-CONFIDENTIAL

FPI-01 in AML Alone – Global Revenue Forecast

• WW Peak Revenue (AML): ~$500MM (1)

FPI-01 in AML Forecast Assumptions Yearly Incidence (2) 2002 2010f 2013f 2016f 2019f Launch 2018 United States 11,578 13,023 13,593 14,167 14,740 Yearly Incident Growth 0.8% Japan 3,054 3,173 3,204 3,228 3,242 >60 Years AML 75% G7 26,586 29,119 30,039 30,931 31,790 WT1 Expression 80% CR1 50% $/Injection $8,000 Price Growth 1% Average Injections 8 Revenue per Patient $64,000 Peak Penetration (2022) 50%

(1) Does not include revenues from potential long-term or life-time boosting regimen, providing for sustained sales growth 14 (2) Datamonitor, Pipeline Insight: Leukemias, March 2010 NON-CONFIDENTIAL FPI-01 - Competition in AML

• Currently no FDA approved products in first remission AML

• FPI-01 has competitive advantages compared to other peptide vaccines in development: • Potent and synergistic CD4+ and CD8+ T-cell responses • Overcomes immune tolerance (heteroclitic design) • Broad HLA targeting profile • Composition of matter patent protection

• Two other potential competitors of note • Ceplene™ (histamine dihydrochloride plus low-dose IL-2) approved in 2009 in Europe; Filing SPA for Phase 3 trial in 2011 • GSK has a whole protein-based WT1 vaccine in phase 1 since 2008; significant disadvantages compared to FPI-01

15 NON-CONFIDENTIAL AML Competitive Landscape

• Limited competition exists for agents seeking to prevent a relapse following the 1st remission in AML patients

1st Line 1st Remission 2nd Line 2010 Patient 29,000 20,500 24,000 incidence (G7) Standard of IDAC, HDAC, LDAC, HSCT Anthracycline/cytarabine none care Hypomethylating agents

Pre-phase III: 1 Agents in Phase III: 3 Phase III: 3 (Ceplene) development Phase II: 20 Phase II: 35 Phase II: 2 by stage Phase I: 11 Phase I: 22 Phase I: 2

Total agents in 35 5 (*) 61 development

Sources: Datamonitor, Medtrack, Kelley WN (ed): Textbook of Internal Medicine. (*) Among development-stage competitors for1st remission AML most are autologous, dentritic cell-based vaccines NON-CONFIDENTIAL FPI-01 – Development Activities Performed By MSKCC

Activity Timing

Discovery of Class-I and Class-II peptides √ Composition of matter patents filed and issued √ Phase I trial completed in AML (n = 9) √ Phase I trial completed in mesothelioma (n = 10) √ cGMP grade manufacturing batches completed for trials (2 separate lots) √

Phase II single arm trial for first-remission AML planned Started 1Q 2011 Funded by MSKCC grant *

Phase II randomized, controlled trial for mesothelioma planned Started 2Q 2011 Funded by US-Department of Defense grant *

Phase II trials planned for Acute Lymphocytic Leukemia (ALL) and ovarian cancer t.b.d.

17 * Formula will be beneficiary of clinical data NON-CONFIDENTIAL FPI-01 – Development and Regulatory Activities Performed and Planned by Formula Activity Timing

Type B meeting held with FDA – positive confirmation / guidance on clinical √ development requirements for approval in first-remission elderly AML ( 2 / 2 0 1 1 )

Development of standard operating procedures for immune response assays, and S t a r t selection of standardized WT1 expression assay, to be used in planned phase-2 trial 4Q 2011

S t a r t Filing of Orphan Drug applications in the US and Europe 3Q 2012

S t a r t Phase-2 randomized, controlled, multi-center trial (60 patients; 15 US centers) 2Q 2012

Second-indication phase-2 trial (e.g. ovarian cancer; non-elderly AML or acute Start by lymphocytic leukemia) using non-dilutive capital sources 1Q2013

European Scientific Advice 2H 2014

18 NON-CONFIDENTIAL Total Investment, Milestones & Timeline

2011 2012 2013 2014 2015 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2

$12.6MM operating budget

$1.1MM $10MM $3MM $5.4MM $4.3MM $2.9MM . 1/1/2012 beginning cash

Complete ph-2 AML trial enrollment Complete Start Ph2 AML trial Start company- Immune sponsored Orphan Start second M&A or Response Ph2 AML Status indication Strategic SOPs trial application phase-2 trial Partnership Start Ph3

Start Event Regulatory Event Business Event 19 Complete Event Expense NON-CONFIDENTIAL Formula - Investor Exit

• FPI-01 program designed with investors’ exit strategy in mind

• Potential for up to 10x return, upon positive completion of Formula’s planned single-arm, open-label 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:

20 NON-CONFIDENTIAL Cancer Vaccines Sector Gaining Substantial Momentum

• In January 2011, acquired Biovex’s OncoVex program for $425MM upfront, plus $575MM in milestone payments – Valuation was based on phase-2 non-randomized clinical data in 40-some patients with head & neck cancer and melanoma – OncoVex program entered into phase-3 in December 2010

• Dendreon’s market cap exceeding $1.5B, as a single product company, despite the recent market turmoil

• BMS’s Yervoy (Ipilimumab) approved in 2011 for metastatic melanoma, with first three-months sales of $95MM

21 (1) Source: Datamonitor, Dec 2009 NON-CONFIDENTIAL Formula - Prior Financings & Use of Proceeds of Series B

• Since inception through August 2011, Formula’s activities have been funded by the Company’s founders and through a government grant

• $2MM in financing raised (including Series A) in 2011

• The proceeds of the $13MM Series B financing will be employed by the Company to: – Select a standardized, validated assay for measuring WT1 in patients – Select standardized, validated assays for measuring immune responses – Complete phase-2 development plan for lead indication (AML) – File Orphan Drug and Fast Track status – Initiate a second-indication, company sponsored trial (with non-dilutive capital) – Achieve near-term exit for investors through M&A or corporate partnership – Pursue at least one in-licensing opportunity involving other drug candidate(s)

22 NON-CONFIDENTIAL Potential Risk-Reducing Factors For Investors

• Strategic partnering with pharma to finance phase-2 trial and to broaden clinical indications (optional; subject to investor interest) – 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) – Also support for second indication (e.g. ovarian cancer; lymphocytic leukemias)

• Sign-off from FDA on clinical development path towards approval, following recent positive FDA meeting

23 NON-CONFIDENTIAL Formula Pharmaceuticals – Investment Highlights • Targeting a most significant unmet need within oncology: preventing recurrence of cancer in patient treated with standard of care

• FPI-01 has demonstrated safety and compelling efficacy in humans

• Seasoned management team with multi-disciplinary experience

• FPI-01 will experience little competition in the maintenance of complete remission space within AML

• Well-defined biomarker strategy with WT-1 supports differentiation and reduces clinical risk

• Already in phase-2 stage clinical development in two indications; potential for expanded indications through strategic partnering

• Seeking an $13 million Series B financing by 1Q2012 that will fund the Company to exit by early 2015

24 NON-CONFIDENTIAL Company address

Formula Pharmaceuticals, Inc. Three Westlakes, 1055 Westlakes Drive, 3rd Floor Berwyn, PA 19312 http://www.formulapharma.com

Maurits W. Geerlings, MD, MBA Chief Executive Officer Ph: 610-727-4172 Fx: 610-23-0111 Mobile: 301-461-7828 [email protected]

25 NON-CONFIDENTIAL