TENGION INC - FORM 8-K - EX-99.1 - EXHIBIT 99.1

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8-K - TENGION, INC. FORM 8-K - TENGION INC	tengion8k.htm

Exhibit 99.1

Tengion Analyst & Investor Meeting

October 27, 2010

Forward Looking Statements

Certain statements in this presentation may constitute forward looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995. Although Tengion believes that
these statements are based upon reasonable assumptions within the bounds of its knowledge of
its business and operations, there are a number of factors that may cause actual results to differ
from these statements.

For instance there can be no assurance that: (i) the Company will be able to obtain the capital it
needs to develop its product candidates or continue as a going concern; (ii) the Company will be
able to successfully enroll patients in its clinical trials, including its Phase I clinical trial for the
Neo-Urinary Conduit; (iii) patients enrolled in the Company’s clinical trials will not experience
adverse events related to the Company’s product candidates, which could delay clinical trials or
cause the Company to terminate the development of a product candidate; (iv) the results of the
clinical trial for the Neo-Urinary Conduit will support further development of that product
candidate; (v) data from the Company’s ongoing preclinical studies will continue to be
supportive of advancing its preclinical product candidates; and (vi) the Company will be able to
progress its product candidates that are undergoing preclinical testing, including the Neo-
Kidney Augment, into clinical trials.

For additional factors which could cause actual results to differ from expectations, reference is
made to the reports filed by the Company with the Securities and Exchange Commission under
the Securities Exchange Act of 1934, as amended. The forward looking statements in this
presentation are made only as of the date hereof and the Company disclaims any intention or
responsibility for updating predictions or expectations in this presentation.

Agenda

Company Overview

Steven Nichtberger, MD, President and CEO

Tengion’s Regenerative Medicine Platform and Product Development Pathway

Tim Bertram, DVM, PhD - Executive Vice President Science & Technology and Chief Scientific Officer

Neo-Urinary Conduit - Clinical

Sunita Sheth, MD, Chief Medical Officer, Vice President Clinical & Regulatory Affairs

Neo-Urinary Conduit - Commercial Opportunity

Mark Stejbach, Vice President & Chief Commercial Officer

- Q&A

Neo-Kidney Augment - Preclinical Research Program

Sharon Presnell, PhD, Senior Vice President Regenerative Medicine & Biology

Neo-Kidney Augment - Research to Clinic

Deepak Jain, PhD, Senior Vice President, Bioprocess Research & Development

Neo-Kidney Augment - Clinical Development Considerations

Sunita Sheth, MD, Chief Medical Officer, Vice President Clinical & Regulatory Affairs

Conclusion

Steven Nichtberger, MD, President and CEO

- Q&A

Tengion products harness the body’s natural ability to regenerate

§ Unique and productive research platform

Lead clinical product targets patients with bladder cancer

§ First patient enrolled, biopsied and implanted successfully

– 1 quarter later than anticipated

§ Investigators anticipate more timely enrollment going forward

§ Significant unmet need drives billion dollar market opportunity

Lead preclinical program delays progression of kidney failure

§ Four key animal models of chronic renal failure

– Initial proof of concept data - AJP/RP: Sept 10

– Diabetic renal failure study - ISCT: Sept 10

– Human cells implanted into nude rats with renal failure - TERMIS Dec 10

– Large animal study - American Society of Transplantation Oct 10

§ Strong and growing foundation of evidence for product development

Company Highlights

Catalyzing Regeneration in the Body

INTEGRATED

PLATFORM

Industrialization

Cells

Biomaterials

Implantation

Humans have limited capacity to regenerate

Our platform uniquely catalyzes human tissue regeneration

Tengion’s Regenerative Medicine Platform and

Product Development Pathway

Tim Bertram, DVM, PhD

Regenerative solutions to address unmet medical needs

safely and efficaciously

Accelerated Product Development Pathway
Translation of Regenerative Medicine Products

§ Product-directed scientists addressing unmet medical need

§ Optimized product formulation for optimal outcomes

§ Established development pathway to commercialization

§ Scale-up manufacturing to meet market demands

Platform - Cells

Defined regenerative cells taken from the patient

§ Autologous cells

– Identify, characterize and expand selected regenerative cells

– Indifferent as to tissue source or cell type - focus is optimizing regeneration

– Avoids risks associated with allogeneic and other sources

Fat biopsy

Selected Regenerative
Cells (SMC)

GMP shipping conditions

INTEGRATED

PLATFORM

Industrialization

Biomaterials

Cells

Kidney Augment: Chronic Kidney Disease

Conduit: Bladder cancer patients requiring bladder removal

Platform - Biomaterials

Biomaterials for enhancing formulation and outcomes

Optimized formulation

Cellular control

Biodegradable scaffold

Cellular alignment

Functional regeneration

§ Focus on materials with clinical precedent to support regeneration

§ Optimize formulation for maximal product stability

Industrialization

Biomaterials

Cells

Kidney Augment: Chronic Kidney Disease

INTEGRATED

PLATFORM

Conduit: Bladder cancer patients requiring bladder removal

* Regeneration demonstrated in preclinical models

Platform - Industrialization

GMP compliant scale-up and focus on cost of goods

§ Integrated, GMP-compliant processing, bio-analytical methods and delivery devices to
produce scalable, cost-efficient products

Expansion

Production bioreactor

Production facilities

Expansion and isolation

Production facilities

Production bioreactor

INTEGRATED

PLATFORM

Industrialization

Biomaterials

Cells

Kidney Augment: Chronic Kidney Disease

Conduit: Bladder cancer patients requiring bladder removal

Platform - Implantation

3-Dimensional tissue and organ regeneration

Delivery/Transport

system

Regenerative outcome

Neo-Urinary
Conduit

Delivery system

Minimally invasive

Regenerative outcome

NX + NKA

Healthy

§ Key opinion leaders have performed preclinical surgeries to inform product design

§ Implant procedure being standardized for consistent outcomes

INTEGRATED

PLATFORM

Industrialization

Biomaterials

Cells

Conduit: Bladder cancer patients requiring bladder removal *

Kidney Augment: Chronic Kidney Disease

* Actively evaluating in clinical program

§ BLA with CBER in the lead and CDRH collaborating

§ IND accepted in 30 days

§ Neo-Bladder Augment experience in US and Europe is instructive for
conduit

§ FDA interactions planned 2011

§ Combination product development pathway expected

§ Previous development experience instructive for optimal plan

Regulatory Pathway - Combination Products

CBER lead with CDRH collaboration

Conduit: Bladder cancer patients requiring bladder removal

Kidney Augment: Chronic kidney disease

IP and Barriers to Entry

Issued patent protection to 2021, plus recent applications pending

§ 37 US and 130 international patents and patent applications

§ Core patents cover composition, design and methods of manufacture*

Plus know-how, trade secrets and integrated capabilities associated with
the discovery, development and manufacturing of our product candidates

CELLS

DEVICE /

MATERIAL

PROCESS

THX USE

Urologic Franchise

= Patent Applications

= Issued Patents

Neo-Kidney Augment

CELLS

DEVICE /

MATERIAL

PROCESS

THX USE

* Individual patents may cover multiple elements

Neo-Urinary Conduit

Clinical Update

Sunita Sheth, MD

When bladder removal is needed, a urinary diversion procedure is performed…

§ Native bladder removed

§ Section of bowel isolated, with blood supply maintained

§ Bowel continuity re-established without the removed segment

§ Ureters connected to the bowel segment, which is connected to
abdominal wall for ostomy bag drainage

Non-continent Urinary Diversion Conduit (20,000 annually in the US & EU)

§ Native bladder removed

§ Section of bowel isolated, with blood supply maintained

§ Bowel continuity re-established without the removed segment

§ Isolated bowel segment fashioned into a pouch

§ Ureters connected to the bowel segment, which is connected to
urethra

Orthotopic Neo-bladder (1,600 annually in the US & EU)

Bladder Cancer Management
Urinary diversion procedures

Current Standard of Care - Common Side Effects

Absorption Issues

Electrolyte and metabolic imbalances,
drug re-absorption issues

Infection

Persistent infections in
~23% of conduit patients

Cancer

Malignancy may result from
use of bowel tissue

Mucus Secretion

Bowel tissue secretes
mucus into urine

Bowel Complications

Leaks, fistulas, obstructions, anemia
and neurological abnormalities

Stone Formation

Recurrent stones in ~10%
of conduit patients, due to
mucus secretion

Neo-Urinary Conduit
Expanding an established regeneration technology platform

SMC from Adipose

Scaffold

Construct

Surgical Implantation

In vivo Regeneration

Urinary diversion

Neo-Urinary Conduit

Open label study to define surgical procedure and safety profile

§ 5 patients with primary bladder cancer requiring bladder removal

– U of Chicago and Johns Hopkins screening

§ Selective enrollment criteria

– Bladder cancer, stage T1/T2 and N0, M0 only

– Not obese

– No history of pelvic radiation

– No uncontrolled diabetes or hypertension

§ Primary efficacy assessment

– Conduit integrity and patency at 1 year

§ Quarterly interim assessments

– Weekly visits in post-operative period

Neo-Urinary Conduit

Clinical trial

Neo-Urinary Conduit Trial
Incorporating years of clinical development experience

Selected entry criteria for initial clinical study

§ Enroll a less complicated and more homogeneous population

– Minimize risk of unrelated events

Standardize surgical approach and post-op stoma care

§ Same 2 surgeons at each surgery for the first 3 patients

Sequential patient enrollment first 3 patients

§ Minimum of 8 weeks between implants for first 3 patients

§ Permits optimization of surgical approach prior to the next patient

First patient has been implanted at U of Chicago

§ Biopsied, manufactured product to specifications, shipped, implanted

§ 1 quarter longer than anticipated to identify first patient

Investigators expect more timely enrollment going forward

§ First patient with Neo-Urinary Conduit now implanted

§ Two sites now actively screening rather than one

Actively evaluating

§ Broadening of patient selection criteria

§ Addition of more sites (minimal incremental cost per site)

Neo-Urinary Conduit

Enrollment

Neo-Urinary Conduit
Commercial Opportunity
Mark Stejbach

Conduit Commercial Opportunity
Efficacy without the morbidity of using bowel

Compelling potential advantages across stakeholders

§ Eliminates need to harvest bowel from the patient

§ May reduce / eliminate many acute and chronic complications

§ Value proposition driven by targeted safety profile

Sources: US: NIS, average 2003-5; EU: National health records for UK,
France, Germany, (2004-6), population-based estimates for others

11,000
Procedures*

13,000
Procedures*

*Annual procedures for all indications

Today, these patients get either a conduit or a bladder replacement

created from their own gastrointestinal tissue

Other conditions include:

radiation cystitis

pelvic trauma

diabetic neurogenic bladder

~15% of Conduits are placed for conditions
other than bladder cancer

Cystectomy Procedures (IMS 2005-6)
Provides a sound basis for current market assumptions

ICD-9 Code Description Procedures

57.71 Radical Cystectomy 12,778

BASE: Total physicians performing cystectomies, n=204)
PPQ1. What advantages, if any, do these products have over existing options?

BioVid survey, Jul 08

Most Important Importance Rankings Least Important

“If this works,
why would I ever
do the traditional
procedure?”

Market Research Reinforces Rapid Uptake Expectations

Based on the product profile, urologists were asked
to rate the Neo-Urinary Conduit compared to the
standard of care

BASE: MDs (n=30) performing 3-25 ileal diversions per year.

Q19. How advantageous would this procedure be as opposed to the current
standard of care?

Very advantageous

Advantageous

No difference

Disadvantageous

Very Disadvantageous

20%

40%

60%

80%

100%

52%

45%

GLG Survey, Dec 08

Percent of respondents

Direct Sales Force

§ Sequenced product launches through centers of excellence

§ >65% of market covered by a specialty sales force of ~ 20 reps

Commercial Model

§ Certified urologist requests biopsy kit

§ Reimbursement confirmation by Tengion

§ Upon pre-clearance, biopsy kit sent

§ Biopsy received at Tengion, cells isolated, conduit grown

§ Neo-Urinary Conduit sent to hospital for implant

US Commercial Strategy
Fully accessible market internally or through partnering

Conduit Commercial Assessment
Summary

Most of the 24,000 US / EU annual conduit procedures are for bladder cancer

Market uptake driven by elimination of bowel surgery

§ Market research with urologists is unambiguous

§ Potential for acute and chronic benefits

§ Potential for new standard of care by eliminating the use of bowel tissue

Intend to model offsets based on clinical trial experience including:

§ Length of OR procedure and hospital stay

§ Peri-op, post-op and long-term complications

§ Impact on QoL and activities of daily living

Payer preference for new products with narrow populations and well-defined
needs, strong clinical support and some direct costs offset

Targeted and consultative hospital call point

Neo-Kidney Augment
Preclinical Research
Sharon Presnell, PhD

100,000 new dialysis patients each year in the US

§ 350,000 currently on dialysis

§ 20% annual mortality

§ $60,000 1st year cost per patient

§ $22 billion in direct US costs annually for end stage kidney disease

Neo-Kidney Augment (NKA) Overview

Intended to delay the need for dialysis or transplantation

***Kelley et al, Am J Physiol Renal Physiol 2010

(Published online 9/6/2010)

*Selected Regenerative Cells used in the NKA

** In development

*Kelley et al, Am J Physiol Renal Physiol 2010 (In Press)

**Presnell et al., presented at ISCT (San Francisco) 9/28/2010

Neo-Kidney Augment Preclinical Research
Summary

Proof-of-concept established for regenerative renal cells in three
independent small animal models of chronic kidney disease (CKD)

§ 5/6 Nephrectomy model of terminal renal insufficiency*

§ ZSF1 model of metabolic syndrome and diabetic nephropathy**

§ Human cells in a nude rat model of I-R/G, chronic after acute renal failure

Early observations from ongoing large animal studies are consistent with
early small animal results

§ Isolation and delivery of renal cells from canines with renal insufficiency

§ Early results indicate in vivo function of regenerative cellular components

Isolation of renal cells from human kidneys with CKD

§ Reproducible isolation from core needle biopsies

§ Supports autologous sourcing strategy from target patient population

*Kelley et al, Am J Physiol Renal Physiol 2010 (In Press)

**Presnell et al., presented at ISCT (San Francisco) 9/28/2010

Neo-Kidney Augment Preclinical Research
Summary

Proof-of-concept established for regenerative renal cells in three
independent small animal models of chronic kidney disease (CKD)

§ 5/6 Nephrectomy model of terminal renal insufficiency*

§ ZSF1 model of metabolic syndrome and diabetic nephropathy**

§ Human cells in a nude rat model of I-R/G, chronic after acute renal failure

Early observations from ongoing large animal studies are consistent with
early small animal results

§ Isolation and delivery of renal cells from canines with renal insufficiency

§ Early results indicate in vivo function of regenerative cellular components

Isolation of renal cells established from human kidneys with CKD

§ Reproducible isolation from core needle biopsies

§ Supports autologous sourcing strategy from target patient population

Rodent 5/6 Nx-induced

model of renal failure

5/6 Nephrectomy Model of CKD
Renal disease secondary to reduced kidney mass

Terminal progressive renal failure

§ 0% survival at 6M post-nephrectomy

§ Progressive decline in GFR

§ Uremic

§ Anemic

§ Proteinuria

§ Hypertensive

§ Weight loss

§ Progressive glomerular sclerosis

§ Progressive tubulointerstitial fibrosis

Renal cells delivered after chronic
disease state established

§ sCREAT sustained at >200%

§ BUN sustained at >150%

Selected regenerative renal cells
(NKA) outperform unfractionated
mixture (UNFX) and improve multiple
physiologic parameters

§ Enhanced survival

– (100% (NKA) vs. 0% (Nx and UNFX)

§ Stabilized filtration (sCreatinine)

§ Improved protein retention

§ Reduced phosphatemia

Kelley et al, Am J Physiol Renal Physiol 2010

(Published online 9/6/2010)

NKA

SHAM NX

HEALTHY NX NX + NKA

Renal Cell function Validated in vivo
In rodent 5/6 nephrectomy model of chronic renal failure

*Kelley et al, Am J Physiol Renal Physiol 2010 (In Press)

**Presnell et al., presented at ISCT (San Francisco) 9/28/2010

Neo-Kidney Augment Preclinical Research
Summary

Proof-of-concept established for regenerative renal cells in three
independent small animal models of chronic kidney disease (CKD)

§ 5/6 Nephrectomy model of terminal renal insufficiency*

§ ZSF1 model of metabolic syndrome and diabetic nephropathy**

§ Human cells in a nude rat model of I-R/G, chronic after acute renal failure

Early observations from ongoing large animal studies are consistent with
early small animal results

§ Isolation and delivery of renal cells from canines with renal insufficiency

§ Early results indicate in vivo function of regenerative cellular components

Isolation of renal cells established from human kidneys with CKD

§ Reproducible isolation from core needle biopsies

§ Supports autologous sourcing strategy from target patient population

Tofovic et al., Ren. Fail. 22:387 (2000)

ZSF1 rodent model of

diabetic nephropathy

Obese ZSF1 Rats Model Progressive Nephropathy
Renal disease secondary to diabetes mellitus and hypertension

Aggressive Metabolic Syndrome

§ Morbid obesity (leptin-receptor deficient)

§ ~50% mortality at ~1yr

§ Multiple co-morbid conditions

– Hyperglycemia

– Vasculopathy

– Hypertension

§ Progressive disease occurs throughout
the nephron

– Renal hypertrophy

– Progressive glomerular sclerosis

– Progressive decline in GFR

– Tubular / interstitial fibrosis

– Severe proteinuria

STABLE

DECLINE

FAILURE

CKD 3

CKD 4

CKD 5

Validating Renal Cells in Chronic Disease
Renal failure secondary to obesity and Type 2 diabetes (ZSF1)

Intervention windows:

§ Stage 3 CKD with no control of hyperglycemia

§ Stage 4 CKD with moderate control of hyperglycemia

§ Stage 5 CKD with moderate control of hyperglycemia

§ Lean ZSF1 = positive control

Intervention strategies:

§ Syngeneic diseased donors

§ Treated (1) or both kidney(s)

Polynomial fit w/ 95% C.I.

Deaths in

untreated

p = 0.033

Untreated

OB ZSF1

+ NKA

Renal Cells Improved Function Throughout the Nephron
Glomeruli, tubules and collecting ducts

At > 1 year of age:

§ 35% improvement in eGFR

(Filtration)

§ 15% reduction in phosphatemia

(Tubular Function)

§ 10% improvement in uOSM

(Concentration)

Treatment Group	63-week Survival
Untreated OB ZSF1	20% (1/5)
OB ZSF1 + NKA	100% (5/5)

p = 0.042

Renal Cells Reduced Hypertension and Improved Survival
ZSF1 rats at >1 year of age

§ Renal Cells reduced mean
arterial blood pressure (MABP)
significantly

§ Renal Cells support survival
beyond 50% mortality time
point for OB ZSF1

*Kelley et al, Am J Physiol Renal Physiol 2010 (In Press)

**Presnell et al., presented at ISCT (San Francisco) 9/28/2010

Neo-Kidney Augment Preclinical Research
Summary

Proof-of-concept established for regenerative renal cells in three
independent small animal models of chronic kidney disease (CKD)

§ 5/6 Nephrectomy model of terminal renal insufficiency*

§ ZSF1 model of metabolic syndrome and diabetic nephropathy**

§ Human cells in a nude rat model of I-R/G, chronic after acute renal failure

Early observations from ongoing large animal studies are consistent with
early small animal results

§ Isolation and delivery of renal cells from canines with renal insufficiency

§ Early results indicate in vivo function of regenerative cellular components

Isolation of renal cells established from human kidneys with CKD

§ Reproducible isolation from core needle biopsies

§ Supports autologous sourcing strategy from target patient population

Nude Rat Model of CKD

Enables functional assessment of bioactive human cells

Rodent model of CKD
after acute injury

Chronic renal insufficiency

§ Renal failure due to Ischemia-
Reperfusion + Gentamicin (I-R/G)

§ Rats that fail to recover 3M post-injury
are utilized (chronic after acute)

§ Model characterized by stable
insufficiency with partial recovery

§ Ischemia-Reperfusion + Gentamicin
injury in nude rats enables functional
testing of human cellular components

Human-derived cells
prevent renal failure in
CKD Nude Rats for 3 mo.

Human Kidney Tissue Regeneration in Nude Rats

Delayed progression of CKD and stabilized renal function

Human-derived cells
improve CKD Nude Rat
nephron function

I-R/G Injury +
Human Renal Cells

I-R/G Injury

Treatment

1 4 12

weeks post-treatment

* p<0.05

Human

Renal Cells

NoTx

*Kelley et al, Am J Physiol Renal Physiol 2010 (In Press)

**Presnell et al., presented at ISCT (San Francisco) 9/28/2010

Neo-Kidney Augment Preclinical Research
Summary

Proof-of-concept established for regenerative renal cells in three
independent small animal models of chronic kidney disease (CKD)

§ 5/6 Nephrectomy model of terminal renal insufficiency*

§ ZSF1 model of metabolic syndrome and diabetic nephropathy**

§ Human cells in a nude rat model of I-R/G, chronic after acute renal failure

Early observations from ongoing large animal studies are consistent with
early small animal results

§ Isolation and delivery of renal cells from canines with renal insufficiency

§ Early results indicate in vivo function of regenerative cellular components

Isolation of renal cells established from human kidneys with CKD

§ Reproducible isolation from core needle biopsies

§ Supports autologous sourcing strategy from target patient population

Canine 5/6 Nx-induced
model of renal failure

INTACT

POLE REMOVAL A

POLE REMOVAL B

RIGHT KIDNEY REMOVED

STEP 1

STEP 2

Modified 5/6 Nephrectomy Canine Model of CKD
Renal disease secondary to reduced kidney mass

Chronic renal insufficiency

§ >50% reduction in GFR

§ Mild hypertension

§ Uremia

§ Mild anemia

§ Progressive proteinuria

§ Gradual weight loss

Caudal Renal

Pole Exposed

Caudal Renal

Pole Injected

NKA Product

R-Kidney
(Control), 40x

L-Kidney
(NKA), 40x

L-Kidney
(NKA), 600x

Compensation

Focus of tubular regeneration

Renal Cell Delivery Verified
In dogs with single kidney (n=2)

No adverse events

No systemic evidence
of renal injury

§ Serum chemistry

Normal histology (1M
post-implant)

§ Normal post-
nephrectomy
compensation

§ Foci of tubular
regeneration at
delivery site

*Kelley et al, Am J Physiol Renal Physiol 2010 (In Press)

**Presnell et al., presented at ISCT (San Francisco) 9/28/2010

Neo-Kidney Augment Preclinical Research
Summary

Proof-of-concept established for regenerative renal cells in three
independent small animal models of chronic kidney disease (CKD)

§ 5/6 Nephrectomy model of terminal renal insufficiency*

§ ZSF1 model of metabolic syndrome and diabetic nephropathy**

§ Human cells in a nude rat model of I-R/G, chronic after acute renal failure

Early observations from ongoing large animal studies are consistent with
early small animal results

§ Isolation and delivery of renal cells from canines with renal insufficiency

§ Early results indicate in vivo function of regenerative cellular components

Isolation of renal cells established from human kidneys with CKD

§ Reproducible isolation from core needle biopsies

§ Supports autologous sourcing strategy from target patient population

Human Translation of Cellular Components
Isolation, characterization and expansion from diseased human kidney

§ Standard core needle biopsy procedure
(0.02g tissue)

§ Cells can be expanded and
cryopreserved

§ Salient phenotypic attributes are
preserved

§ Supports autologous sourcing strategy

Presnell et al., Tissue Engineering Part C, 2010

(Published online 9/20/2010)

*Kelley et al, Am J Physiol Renal Physiol 2010 (In Press)

**Presnell et al., presented at ISCT (San Francisco) 9/28/2010

Validation of Renal Cell Function in Four Models
Increases probability of success through development

1. Renal cells demonstrated durable function in 5/6 Nx rodent model of CKD due to renal
mass insufficiency*

§ Robust therapeutic effect (animals followed 6M post-implant)

§ Reproducible across independent studies

2. Renal cells function in a model of CKD secondary to obesity and Type 2 Diabetes
(Active)**

§ Evaluation of intervention at CKD Stages 3-4 and followed to >1 year of age

§ Renal cells derived from diseased donors

3. Will human-derived renal cells function in vivo? (Active)

§ Ischemia-Reperfusion-Gentamicin (I-R/G) model of kidney damage (chronic after acute)

§ Feasibility established in I-R/G model with human renal cells

4. Will the renal cells function in a large animal model of CKD? (Active)

§ Isolation and delivery of renal cells from canines with renal insufficiency

§ Early results indicate in vivo function of regenerative cellular components

Neo-Kidney Augment
Research to Clinic
Deepak Jain, PhD

Neo-Kidney Augment
Research to clinic

INTEGRATED

PLATFORM

Industrialization

Cells

Biomaterials

Implantation

Manufacturing/Product Release

Product Delivery

Selected Regenerative Cells

Product Formulation

INTEGRATED

PLATFORM

Industrialization

Biomaterials

Cells

Cell Factory (10 stack)

INTEGRATED

PLATFORM

Industrialization

Biomaterials

Cells

COBE

Tissue Culture Flasks

Neo-Kidney Augment
Cells: selected regenerative cell sourcing

Cell Sourcing

§ Kidney needle biopsy

Cell Isolation

§ Enzyme digestion of biopsy

§ Cell washing and plating

Cell Expansion

§ By serial passage

§ Separated by density gradient
centrifugation

Cell-biomaterial formulations optimized in
combinatorial screening platform

Renal Cell - Biomaterial Formulations

INTEGRATED

PLATFORM

Industrialization

Biomaterials

Cells

Neo-Kidney Augment
Biomaterials: product formulations

Neo-Kidney Augment Program

Selected Regenerative Renal Cell Population

§ Demonstrated efficacy

Biomaterial Formulation Provides

§ Cell stability and durability for transport

§ Predictable and persistent delivery of cells

§ Structure - architecture for cell interactions

§ Space - displacement of fibrous tissue

Product Characterization and
Quality Release Criteria:

Cell Function

Product Integrity

Final Construct

Cell Phenotype

Cell Viability

Total Viable Cells

Cell Morphology

Product Release Criteria

Quality Attribute

INTEGRATED

PLATFORM

Industrialization

Biomaterials

Cells

Neo-Kidney Augment
Industrialization: manufacturing

Manufacturing:

§ Closed system

– aseptic, scalable manufacturing

§ Maintain integrity during transport

§ User-friendly handling in the OR

INTEGRATED

PLATFORM

Industrialization

Biomaterials

Cells

Neo-Kidney Augment
Implantation: Product Delivery

Delivery of Injectable Product:

§ Syringe and needle

§ Catheter

Product Implantation:

§ Effective delivery

Neo-Kidney Augment

Clinical Development Considerations

Sunita Sheth, MD

Sources: NHANES (2004) and US Renal Data System (2008)

CKD Prevalence by Stage

(000s)

Primary Diagnosis at ESRD

44%

28%

13%

Diabetes

Hypertension

Glomerulonephritis

Cystic Kidney

Disease

Urologic Disease

Other known

Unknown

Missing data

Chronic Kidney Disease Affects Over 20 million in US
Expected to double by 2020, driven by diabetes

Clinical Development Pathway
Informed by preclinical studies and regulatory history

Clinical Benefit

§ Improve kidney function in patients at risk of requiring dialysis

§ Delay progression of kidney failure or time to onset of dialysis

Target Population

§ Stage of kidney failure

– Stage 3 and 4

§ Underlying disease

– Diabetes, hypertension, other

Efficacy Measures

§ Functional changes

– Improvement in GFR or change in rate of decline in GFR

– Time to doubling of the serum creatinine

– Reduction in proteinuria

§ Event reduction

– End-stage renal disease

• Defined as initiation dialysis or transplant

– Mortality

Neo-Urinary Conduit Trial in Bladder Cancer Patients

§ Successful first implant

– Biopsy, manufacturing, quality release and implant procedure

§ Investigators optimistic regarding future enrollment timelines

– First patient successfully implanted

– Two sites actively screening

§ Anticipate update on clinical progress by next implant

Neo-Kidney Augment to Delay Progression or Eliminate Need for Dialysis

§ Strong and growing foundation of evidence for product development

– Optimizing product formulation

– Continuing follow-up from ongoing studies

§ Anticipate discussions with FDA in 2H11 regarding path to clinical trials

Conclusions

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