analysis of licensing gtx-001 and gtx-002 and gastrex team 7 - animal pharm jason dwyer scott gleim...

Analysis Of Licensing

GTX-001 And GTX-002

and Gastrex

Team 7 - Animal Pharm

Jason Dwyer Scott Gleim Ph.D.

Samantha KennedyXin Li M.S.

Robert LindnerJohn Nelson

Ph.D. Candidate, BBSInternal MedicineMBA/MPH Candidate, 2012M. A Statistics Candidate M.S. Mol. Biotech CandidateMBA Candidate, 2011

YBPS Healthcare Case Competition, 2009

Agenda

Inflammatory Bowel Disease (IBD):Crohn’s Disease (CD) Ulcerative Colitis (UC)

Gastrex GTX-001 and GTX-002:Unmet Needs, Target Product Profile

Signals For Success Future Clinical Trial Timeline

Market AnalysisFinancial Projection

Licensing Recommendation

GTX-001(Biological) (Small Molecule)


Inflammatory Bowel Disease

What is IBD?• Inflammatory disorder of the intestinal

mucosa• Crohn‘s Disease (CD) : small & large

intestine• Ulcerative Colitis (UC): colon, rectum

• Symptoms• Diarrhea, abdominal pain• Anemia, bleeding

• Multifactorial and multigenic etiology• Mucosal integrity• Hypersensitivity to intestinal microbes• Aberrant TH-Cell mediated immune

activation

CD UC


GTX-002: Unmet Needs

ASACOL, LIALDA• Weak efficacy in Crohn’s Disease• High daily pill burden• Poor patient compliance

PREDNISONE, ENTOCORT• Can cause intestinal bleeding• Psychiatric disturbances• Nausea and/or vomiting, seizures

IMURAN, 6-MP• 20% of patients have severe

intolerance• Slow onset (6-8 months)• Loss of bone marrow

5-ASAs

Steroids

Immuno-modulators

Treatment of mild-moderate CD/UC relies on combinations of 5-ASAs, steroids and immunomodulators:

Mild46%

Moderate38%

Severe16%

Disease Severity


GTX-002 Target Product Profile

5-ASAs

Steroids

IM’s

Novel Mechanism of ActionDirectly Targets T-cell

ApoptosisActs Downstream of TNF a

Low Pill Burden

SaferMore Effective

Rapid Onset

Orally AvailableGTX-002

Competitive Advantage


Problems with anti-TNF Drugs: - All require black box warning for fungal infection - Potential for serious/fatal infections

- Solid-tissue cancers- Liver injury

GTX-001: Unmet Needs

INFLIXIMAB• Requires IV administration

every 8 weeks• Development of neutralizing

antibodiesADALIMUMAB• Requires dosing every 2

weeks• Risk of blood disordersCERTOLIZUMAB PEGOL• Produces only modest efficacy• Does not extend remission

time

45-70% of severe patients never respond or stop responding to anti-TNF treatment

Biologics used for treating severe CD/UC:


GTX-001 Target Product Profile

GTX-001 Competitive Advantage

Novel Mechanism of Action

Not TNF a DependentOnly Attacks Activated T-Cells

Fully Humanized

SaferMore Effective

PEGylated Half-lifeIncrease

SubcutaneousSelf-

Administration

GTX-001


Signals and Clinical Trial Timeline

2010 2032

January 2010Lead Optimization

TargetsChem/Eng

April 2010Preclinical Development

PK/PD/ADMEPharm/ToxFormulation/Mfg

April 2012IND/CTA

July 2012Phase ISafety &

ToleranceApril 2013Phase 2aSafety & Efficacy

July 2014Phase 2bSafety & Efficacy

July 2016Phase 3

Major Efficacy Trial(s)

January 2018NDA/BLA Filing

October 2019NDA/BLA Approval

January 2020Product Launch

2020 - 2032Post-Market Surveillance

Pharmacokinetics Safety Efficacy

GTX-001 SC administrationt1/2 ≥ 4 wks

AntigenicityImmunosuppression

≥ infliximabRefractory population

GTX-002 Oral administrationOAD or BID

Liver toxicityKidney toxicity

Rapid onsetConcomitant Tx


5-ASA, $1.3B37%

Steroid, $33.8M

1%

IM, $105M

3%

Treatment Sales by Drug Type

(US and EU)

Biologics$2.1B59%

Market Analysis

IBD Severity Rates

Mild 46%

Moderate 38%

Severe 16%

IBD Severity Rates

Mild 46%

Moderate 38%

Severe 16%

GTX-001 GTX-002

High Price, Low Volume Includes treatment refractory

segment Generic biologics more

challenging 3 major competitors

Low Price, High Volume

Established generics 5+ major competitors Potential to change

market?


Assumptions (per yr)- Discount Rate: 10%

- GTX-001 Price: $15k- GTX-002 Price: $2.5k

- CD Market Growth: 0.61%- UC Market Growth: 0%

ScenarioMarket Share

Market Share Growth

Profit Margin

NPV ($)

GTX-001 GTX-002High 24% 5% 25% 639,682,775 6,679,056Mid 18% 3% 20% 127,981,673 (213,724,869)Low 12% 1% 15% (198,022,889) (351,124,037)

Net Income Sensitivity Analysis


Licensing and Contract Recommendations

License GTX-001 Positive NPV Attractive Target Profile Strong Market Opportunity Important therapy for patients

Do not license GTX-002 under current agreement………

Contract Renegotiation Options Decrease milestone payments for GTX-002 or both -001 and -002 More indications for use: Rheumatoid Arthritis, Psoriasis Profit sharing vs. royalties


THANK YOU!

Team 7 - Animal Pharm

Jason Dwyer Scott Gleim Ph.D.

Samantha KennedyXin Li M.S.

Robert LindnerJohn Nelson

Ph.D. Candidate, BBSInternal MedicineMBA/MPH Candidate, 2012M. A Statistics Candidate M.S. Mol. Biotech CandidateMBA Candidate, 2011

http://www.vionpharm.com/

http://www.mckinsey.com/

http://www.taro.com/

http://www.yale.edu/ybps/www.covidien.com


Competitive Drugs in Development

Drug Mechanism Phase CompanyDelmitide INF-γ, TNF-α, IL-12 Inhibitor I Genzyme

HE-3286 Synthetic steroid I Hollis-Eden Pharma

PP-008 I Inflbloc Pharma

mAb beta 7 I Genentech

CCX282B CCR9 antagonist II ChemoCentryx

AIN-457 Anti-IL17 (mAb) II Novartis

AM-3301 II Amalyte

Apilimod IL-12 Inhibitor II Synta Pharma

Briakinumab Anti-IL-12/23 (hmAb) II Cambridge Antibody

MDX-1100 Anti-CXCL10 II Medarex

Ustekinumab Anti-IL-12/23 (hmAb) II Medarex/Centocor

ATN-103CP-690550Oprevekin

JAK-3 InhibitorRecombinant hIL-11

IIIIII

Pfizer

Vedolizumab Anti-LPAM-1 III Millennium Phamra

Alicaforsen ICAM-1 antisense III Isis Pharma


DRUG 1 DRUG 2, A DRUG 2, BCD UC CD UC CD UC

Market SizeSegment 1 0 0 0 0 0 0Segment 2 0 0 142000 0 142000 352000Segment 3 0 0 155000 135000 155000 135000Segment 4 0 0 245000 259000 245000 259000Segment 5 145000 99000 0 0 145000 99000Segment 6 65000 40000 0 0 65000 40000TOTAL 210000 139000 542000 394000 752000 885000

Drug 1 Drug 2Selling Price 15000 2500

Profit Margin, Initial 20%Market Share 18%Market Share growth 3%CD Market Growth 0.61%Milestone payment 400,000,000.00

UC Market Growth 0%


DRUG 1 1/1/2010 1/1/2020 1/1/2021 1/1/2022 1/1/2023 1/1/2024 1/1/2025 1/1/2026 1/1/2027 1/1/2028 1/1/2029 1/1/2030 1/1/2031 1/1/2032

Market Size 349000 362120.434 363459.3057 364803.1277 366151.9182 367505.6957 368864.4784 370228.285 371597.134 372971.0441 374350.0339 375734.1223 377123.3281 378517.6703Market Share % 18% 19% 19% 20% 20% 21% 21% 22% 23% 23% 24% 25% 26%

Total Patients 65,181.68

67,385.36

69,663.53

72,018.74

74,453.56

76,970.70

79,572.95

82,263.17

85,044.34

87,919.54

90,891.94

93,964.84

97,141.62

Sales $ 977,725,171.71

$ 1,010,780,329.24

$ 1,044,953,023.15

$ 1,080,281,035.35

$ 1,116,803,425.12

$ 1,154,560,572.24

$ 1,193,594,221.68

$ 1,233,947,529.72

$ 1,275,665,111.68

$ 1,318,793,091.25

$ 1,363,379,151.47

$ 1,409,472,587.47

$ 1,457,124,360.97

Less 10% to Gastrex $ (97,772,517.17)

$ (101,078,032.92)

$ (104,495,302.31)

$ (108,028,103.54)

$ (111,680,342.51)

$ (115,456,057.22)

$ (119,359,422.17)

$ (123,394,752.97)

$ (127,566,511.17)

$ (131,879,309.12)

$ (136,337,915.15)

$ (140,947,258.75)

$ (145,712,436.10)

Upfront Licensing agreement

$ (10,000,000.00)

Late Stage development Costs

Milestone Cost $ (40,000,000.00)

Promotional Costs $ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

$ (100,000,000.00)

NET INCOME $ (50,000,000.00)

$ 155,990,530.91

$ 161,940,459.26

$ 168,091,544.17

$ 174,450,586.36

$ 181,024,616.52

$ 187,820,903.00

$ 194,846,959.90

$ 202,110,555.35

$ 209,619,720.10

$ 217,382,756.42

$ 225,408,247.26

$ 233,705,065.75

$ 242,282,384.97

NPV $ 127,981,673.63 discount rate 10%

13%

DRUG 2, A 1/1/2010 1/1/2020 1/1/2021 1/1/2022 1/1/2023 1/1/2024 1/1/2025 1/1/2026 1/1/2027 1/1/2028 1/1/2029 1/1/2030 1/1/2031 1/1/2032

Market Size 936000 971188.3272 974779.1122 978383.1735 982000.5601 985631.3213 989275.5066 992933.1655 996604.348 1000289.104 1003987.484 1007699.537 1011425.316 1015164.869Market Share % 18% 19% 19% 20% 20% 21% 21% 22% 23% 23% 24% 25% 26%

Total Patients 174813.8989 180724.0474 186834.0076 193150.5347 199680.6124 206431.4605 213410.5428 220625.5755 228084.5357 235795.6702 243767.5044 252008.8523 260528.8256

Sales 437034747.2 451810118.5 467085018.9 482876336.7 499201531 516078651.2 533526357 551563938.8 570211339.3 589489175.5 609418761.1 630022130.8 651322063.9

Less 10% to Gastrex -43703474.72 -45181011.85 -46708501.89 -48287633.67 -49920153.1 -51607865.12 -53352635.7 -55156393.88 -57021133.93 -58948917.55 -60941876.11 -63002213.08 -65132206.39

Upfront Licensing agreement

(1,000,000.00)

Late Stage development Costs

Milestone Cost $ (40,000,000.00)

Promotional Costs -100000000 -100000000 -100000000 -100000000 -100000000 -100000000 -100000000 -100000000 -100000000 -100000000 -100000000 -100000000 -100000000

NET INCOME (41,000,000.00) 58,666,254.50

61,325,821.33

64,075,303.41

66,917,740.61

69,856,275.58

72,894,157.22

76,034,744.25

79,281,508.98

82,638,041.08

86,108,051.58

89,695,377.00

93,403,983.54

97,237,971.51

analysis of licensing gtx-001 and gtx-002 and gastrex team 7 - animal pharm jason dwyer scott gleim...

Documents

agenda gtx

competitive advantage

target product profile

statistics candidate

biotech candidatemba

cd market growth

uc market growth

high price