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This presentation and our remarks based upon it,
including responses to questions made during and
following the presentation, may include forward-looking
statements. Such statements are subject to the risks and
uncertainties we discuss in detail in our reports filed with
the Securities & Exchange Commission, including in our
quarterly report on Form 10-Q filed November 8, 2012.
We expressly disclaim any obligation to release publicly
any updates to forward-looking statements made during
the course of this presentation.
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Two late stage clinical programs
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Enobosarm (Ostarine; GTx-024), a SARM, for the prevention and treatment
of muscle wasting in patients with non-small cell lung cancer:

Eight clinical trials completed to date involving approximately 600 subjects

Granted fast track status January 2013

Enrollment completed 4Q 2012 for two pivotal Phase III clinical trials with
approximately 650 non-small cell lung cancer (NSCLC) patients – top line results
expected summer 2013

DSMB reviewed safety data in October 2012 and agreed trial could continue as
planned
Capesaris (GTx-758), an oral selective ER alpha agonist, for combination
primary ADT & secondary hormonal treatment of advanced prostate cancer:

Eight clinical trials conducted to date involving approximately 500 subjects

Phase II 712 clinical trial evaluating Capesaris® (GTx-758) for secondary
hormonal treatment in men with metastatic castration resistant prostate cancer
currently enrolling
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Enobosarm (Ostarine; GTx-024)
Selective Androgen Receptor Modulator (SARM)
for the prevention and treatment of muscle wasting
in patients with lung cancer
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Muscle wasting is an important cancer related
symptom in patients with advanced NSCLC
•
At diagnosis, nearly 50% of advanced NSCLC patients have severe
muscle loss and approximately 70% of NSCLC patients will lose muscle
 88% have lower body functional limitations including the ability to climb stairs, lift
and carry 10 lbs, walk ¼ mile, and stoop, crouch or kneel
•
Performance status is a predictor of a patient’s ability to tolerate
chemotherapy, and poor performance status is a primary reason patients
are not offered treatment
 Performance status also predicts the likelihood of hospitalization, ability to
maintain independence, and survival
Baracos VE, et al. Am J Clin Nutr. 2010 Apr;91(4):1133S-1137S. Baracos VE. 2011 data on file. Bruera E. BMJ. 1997;315(7117)1219-22. Schootman M, et al.
Cancer. 2009 Nov 15;115(22):5329-38. Braithwaite D, et al. J Natl Cancer Inst. 2010;102(191):1468-77. Prado CMM, et al. The Lancet Oncology 2008;9(7):629-35.
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Phase IIb clinical trial in cancer patients:
Enobosarm increased lean body mass & improved physical function in NSCLC
Subset analysis: 61 NSCLC patients
• Mean weight loss at entry was 9.7%
• Enobosarm treated patients had a mean 18% improvement in stair climb power from
baseline and 1.1 kg improvement in LBM
• No statistically significant improvements in physical function or LBM in placebo group
Ad Hoc Phase IIb Responders Analysis – NSCLC*
Endpoint
Placebo
Enobosarm
Physical Function
19%
42%
Lean Body Mass
24%
42%
*Based on ITT including 30% drop out rate
Safety- NSCLC
Placebo
(n=21)
1 mg
(n=21)
3mg
(n=19)
Deaths
6 (29%)
8 (38%)
3 (16%)
SAE
10 (48%)
10 (48%)
6 (32%)
•
Reported incidence of tumor progression similar
across groups
•
The most common AEs were fatigue, anemia,
nausea and diarrhea
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International pivotal Phase III clinical trials: POWER 1 and 2
Indication: Prevention and treatment of muscle loss in patients with NSCLC
• Stage III/IV NSCLC patients initiating 1st line chemotherapy
• Co-primary endpoints (responders analysis): (1) no loss of lean body mass (LBM) by DEXA; (2) at least
10% improvement in Stair Climb Power (SCP)
• Each endpoint α=0.05, power >93%
• Assumes 30% drop out rate by 3 months
Co-primary endpoints
Secondary endpoints
•Lean body mass
•Physical function SCP
@ 3 months
• Durability of effect
@ 5 months
• Overall survival
Enobosarm
150 patients
3 mg
platinum +
taxane
Other endpoints
Placebo
150 patients
Enobosarm
150 patients
3 mg
platinum +
non taxane
Placebo
•QoL – FAACT,
FACIT fatigue scales
•Healthcare resource
utilization
•Adherence to
chemo plans
•Tolerance to chemo
150 patients
Input from FDA, MHRA (England) and MPA (Sweden) on Phase III clinical development plans and
Fast Track granted 1/13
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Enobosarm
Anticipated clinical development plan
2011
1Q
2Q
3Q
2012
4Q
1Q
2Q
3Q
2013
4Q
1Q
2Q
3Q
2014
4Q
1Q
2Q
3Q
4Q
Phase III-POWER 1
Enobosarm 3 mg vs placebo in 300 pts
with NSCLC receiving platinum + taxane
chemotherapy (5 month study)
NDA
Phase III-POWER 2
Enobosarm 3 mg vs placebo in 300 pts
with NSCLC receiving platinum + non taxane
chemotherapy (5 month study)
First patient
enrolled
Last patient
out
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Enobosarm
Large market opportunity
Stage III-IV NSCLC is the majority of NSCLC diagnosed and, based on perceptions of
oncologists, at least 50% of these patients have muscle wasting
United States
5 EU
NSCLC
NSCLC
207 K
186 K
Stage III-IV
NSCLC
161 K (78%)
hyperK+
Rates
Stage III-IV
NSCLC
148 K (79%)
hyperK+
Rates
Muscle Wasting
Muscle Wasting
81K (~50%)
74K (~50%)
Source: Datamonitor 2011; projected market size in 2012, US and EU Qualitative market research
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Enobosarm
Large market opportunity- US Payors’ perspective*
•
Improvement in physical function and increase in muscle mass were considered the two most
favorable clinical attributes of enobosarm
•
All economic endpoints being evaluated are acceptable by the majority of Payors

Hospitalizations, ER visits, mobility devices, home health services, and hospice
•
80% of Payors would cover enobosarm with or without restriction
•
At a monthly WAC of $1500 - $3000 the majority of Payors would cover enobosarm as a Tier 3
with a prior authorization
•
Currently, several drugs treating cancer related symptoms are priced between $30 and $50 per
day
•

Xgeva (CTIBL and SRE) $55 per day

Zometa (CTIBL and SRE) $40 per day

Neulasta (neutropenia) $5600 per dose
GTx estimates muscle wasting indication in patients with NSCLC
could be a $750 million opportunity in US alone
*Based
on US Managed Healthcare Landscape and Assessment of Enobosarm, Managed Solutions, LLC April 2012
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Enobosarm
Intellectual property
•
79 enobosarm composition of matter and method of use patent
applications issued, approved, or pending in U.S. and rest of world with
expiration dates in 2024.

Includes issued composition of matter and method patents in US and Japan
•
As a new chemical entity, issued patents should be eligible for patent
term extension of up to 5 years (2029)
•
GTx has 350 patents issued, approved or pending worldwide for all
SARMs including enobosarm
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Capesaris® (GTx-758)
Selective ERα agonist for the treatment of
advanced prostate cancer
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Evolving treatment options for advanced
prostate cancer
Advanced Prostate Cancer
Castration Resistant Prostate Cancer (CRPC)
Hormone
Sensitive
Primary hormone
therapy
(LHRH agonists
or antagonists)
Capesaris
First
Secondary
Hormone Tx
Capesaris
Enzalutamide
Second
Secondary
Hormone Tx
Capesaris
Enzalutamide
=approved
Third
Secondary
Hormone Tx
Abiraterone +
Prednisone
Capesaris
Chemotherapy
Docetaxel
Post
Chemotherapy
Enzalutamide
Abiraterone/
Prednisone
Cabazitaxel
750,000
patients
80,000
patients
36,000
patients
Market
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Multiple potential mechanisms of action for a
selective ERα agonist to treat prostate cancer
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Higher SHBG reduces available free (unbound) T
Adapted from Dunn JF et al. (1981) J Clin Endo and Metabolism, 53:58-68
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Phase II studies in men with advanced prostate
cancer confirms the mechanism of action*
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Phase II open label loading dose finding 705 clinical study in
advanced prostate cancer comparing1500mg BID and 1000mg BID
loading doses followed by 1000mg or 2000mg maintenance doses
(n=55)
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•
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Phase II open label maintenance dose finding 710 clinical study in
advanced prostate cancer comparing Lupron, Capesaris 1000mg
PO qd and Capesaris 2000mg PO qd (n=164)

2000mg Capesaris and Lupron arms
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Castration rate greater than 90% for both arms
Capesaris increased SHBG and decreased free T
Maintained castration by Kaplan-Meier estimates >95.5%
Similar testosterone escapes
Capesaris increased SHBG and decreased free T
Improvement in hot flashes, bone turnover markers and insulin resistance
Safety- VTE incidence rate increased for Capesaris
*Studies terminated prior to completion
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Phase II, open label, 712 clinical study of secondary
hormonal treatment in men with metastatic CRPC
started 3Q 2012
Subjects
Primary Endpoint:
Secondary Endpoints:
• mCRPC
• Maintain ADT
• Serum PSA response
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GTx-758
25 patients
125 mg
30 d
GTx-758
250 mg
30 d
25 patients
GTx-758
500 mg
Day
0
PSA progression
Progression free survival
Free T/SHBG
Adrenal (DHEA&DHEAS)
Estrogen deficiency side effects
SRE
25 patients
Day
90
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Capesaris
Steering Committee
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Medical oncology






•
Evan Yu* (U Washington)
Johann DeBono* (Royal Marsden)
Dan Petrylak* (Columbia/ Yale)
Chuck Ryan* (UCSF)
Phil Kantoff* (Dana Farber Harvard)
Thomas Flaig* (U Colorado)
Urology
 Badri Konety (U Minnesota)
•
Advocacy group
 Tom Kirk (Us TOO)
* Confirmed participation
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Capesaris
Anticipated clinical development plan
2011
1Q
2Q
2012
3Q
4Q
1Q
2Q
2013
3Q
4Q
1Q
2Q
3Q
2014
4Q
1Q
2Q
3Q
4Q
Phase IIb
705 clinical study
Maintenance dose finding
Phase II
710 clinical study
Loading dose finding
Phase II
Phase II
707 clinical study
Secondary hormonal
therapy in CRPC patients
712 clinical study
Secondary hormonal therapy in
mCRPC patients (lower doses)*
*Estimated based on projected enrollment
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Capesaris
Intellectual Property
•
Approximately 43 composition of matter and method of use patent applications
and patents, which are either issued, allowed or pending in the US and rest of
world with expiration dates of January 2029 in the US (issued) and November
2026 in the ROW
•
As a chemical entity, issued US patent should be eligible for additional patent
term extension of up to 5 years (for a maximum term of November 2034), as
may be determined following FDA approval of Capesaris
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Financial summary
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Shares outstanding: 62.8 M
•
Cash, cash equivalents and short-term investments
at December 31, 2012: $56.1M
•
No debt, no warrants
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