Pioneering Medicine to Preserve Kidney Health

John B Wirthlin.Founder/Chief Operating Officer

November, 2014

© AlloCure 2014615 Arapeen Dr. Salt Lake City, Utah 84108 Phone (801) 583 8450

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While the Goal of Drug Development Is Laudable, The Challenge of the Task Is Often Daunting

The Problem: Acute Kidney Injury (AKI)

• AKI occurs in the hospital, often in but not limited to, the intensive care unit setting

• Characterized by a rapid decline in kidney function, most often in patients with acute insults on top of underlying co-morbidities

• Risk factors include CKD, hypertension, diabetes and age

• There is no approved therapeutic solution other than patient support

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Common Contributors to AKI CABG and valve replacement surgery with

cardiopulmonary bypass as well as complex surgical procedures in numerous clinical settings

Contrast imaging agents and nephrotoxic drugs

Sepsis and hypotension

Consequences

Extended ICU and hospital stay

Increased need for short and often long term dialysis

Progression of CKD, including ESRD

Treatment of AKI is a Critical Unmet Medical Need

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Lewington et al Raising awareness of acute kidney injury: a global perspective of a silent killer Kidney International. 2013 84, 457-467

Unfortunately, Single Metabolic Pathway Interdictions in AKI Have Been Unsuccessful

• Natriuretic peptides

• N-acetylcysteine

• Low dose dopamine

• IGF-1

• rHuEPO

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The AlloCure Solution: AC607 Bone Marrow Derived Mesenchymal Stem Cells

SDF-1

VEGF, HGF,IGF, PGE2

Humphreys and Bonventre. Ann. Rev. Med. 2008. 59:325–39

• AC607 transiently resides in the injured kidney and delivers VEGF, HGF, IGF, PGE2 and other mediators that treat AKI via multiple processes (local, transient, poly-pharmacy effect):

Anti-apoptotic Mitogenic Anti-inflammatory Angiogenic

• AC607 does not divide and repopulate the injured kidney

• Immune privileged- no donor matching

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AC607 Phase 1 Trial in Cardiac Surgery Completed

• 16 subjects scheduled for elective CABG and/or valve replacement and at high risk for AKI

• Following surgery, AC607 administered via catheter into the suprarenal aorta

• Primary endpoint was safety; additional endpoints evaluating kidney function including assessment of AKI and post operative course 3 year follow up complete; AC607 was safe and well tolerated No SAEs related to study drug

• Historical control data were examined to enable a preliminary assessment of efficacy

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Incidence of AKI, Hospital Readmission Rates and Length of Stay Lower in AC607 Subjects Vs. Historical Controls*

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RIFLE (%) AKIN (%) Readmission Rate (%) Hospital Length of Stay (Days)

0

5

10

15

20

25

30

Phase 1 Subjects (n=16) Matched Controls (n=64)

*matched for age, risk factors, surgical characteristics

Doty et al., Abstract. Am Soc Neph Kidney Week, 2011. Phil, PA.

AlloCure Developed a Robust Manufacturing Process for AC607

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MSC Isolation

Multiple Cell

Factories

MSC Expansion MSC Harvest & Vialing

MediaMedia Cell Factory

Healthy Donor Bone

Marrow

Healthy Donor Bone

Marrow

Cell Factory

Culture

Multiple Cell

Factories

ExpansionConcentrate & Wash

Fill & FinishAC607 Doses

MasterCell Bank

AC607Production

• Closed system for culturing and downstream processing • Free of animal derived raw materials• Fully compliant and tested in accordance FDA guidelines

AC607 Proof of Concept Phase 2 Trial: ACT-AKI

Design Parameters

Design Randomized, double-blind, multi-center, placebo-controlled, two arm study

Sample Size N = 200

Primary Endpoint Time to kidney recovery

Secondary Endpoints Composite of incidence of dialysis and mortality, safety

Exploratory Endpoints Length of stay (ICU and hospital), hospital readmission rates, short and long term measures of kidney function

AC607

n = 100

Placebo

3 Month Evaluation Period Long Term Follow-up

n = 100

⁄ ⁄

⁄ ⁄

Study Population: Subjects undergoing CABG and/or valve

surgery with a 0.5 mg/dL rise in SCr within 48 hours

of surgery

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ACT-AKI Phase 2 Trial Update

• AlloCure recently completed the data analysis from its phase 2 trial

• The clinical data was not strong enough to justify further clinical development of AC607

• Final clinical results will be presented at the American Society of Nephrology next week

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AlloCure Accomplishments

• Raised $45MM from top tier VCs and corporate partners

• Approval of the first IND using stem cells to treat AKI

• Awarded Fast Track designation by the FDA

• Conducted one of the largest trials in AKI

• Advanced the science of stem cells

• Developed a next generation robust manufacturing process

• Contributed to the clinical knowledge of AKI

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Lessons Learned

• Attracting Capital

• Capital Efficient Organization

• Agile Clinical Development

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Attracting Capital

• Approached investors who had experience with stem cells

• Specifically tailored the message

• Show progress in hitting milestones during the funding negotiation

• Tranching

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Capital Efficient Organization: Keep it Lean

•Employees• Hire individuals who can do more than one job function• High emotional intelligence

• Outsource high cost functions: • Human Resources• Accounting • IT• Clinical operations• Manufacturing

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Agile Clinical Development

• Early and rapid response to change• High functioning executive team

• Creative problem solving • Clear communication • Trustful execution

• Develop a culture of excellence• Work environment that is exciting, challenging and fun • Employees who are dedicated to reaching the goal • Commitment to “do whatever it takes”

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Questions