Transcript Phase 3 studies of enobosarm

Corporate Presentation
Safe Harbor
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 May 12, 2014. We expressly disclaim any obligation to release
publicly any updates to forward-looking statements made during the
course of this presentation.
2
Overview of Clinical Programs
PROGRAM
Enobosarm 9mg
(Ostarine®; GTx-024)
Enobosarm 3mg
(Ostarine®; GTx-024)
Capesaris®
(GTx-758)
PROPOSED INDICATION
AR+ and ER+ advanced
metastatic breast cancer
Prevention and treatment
of muscle wasting in
patients with non-small
cell lung cancer
Secondary hormonal
treatment for advanced
prostate cancer
DRUG
CLASS
PRECLINICAL
SARM
G200801
Phase I
Phase II
Phase III
NDA
POWER 1 (Taxane)
SARM
POWER 2 (Non-taxane)
ERα
Agonist
G200712
SARM- Selective Androgen Receptor Modulator
ERα Agonist- Estrogen Receptor Alpha Agonist
3
ENOBOSARM 9mg (OSTARINE®; GTx-024)
Selective Androgen Receptor Modulator (SARM)
to treat AR+/ER+ metastatic breast cancer
4
Targeting the Androgen Receptor in Breast
Cancer
• Prior to the introduction of SERMs and AIs, steroidal androgens had
been more extensively used for the treatment of breast cancer
•
After the development of SERMs, androgens fell out of favor due to the
improved efficacy of SERMs, potential for the aromatization of
androgens to estrogen and the virilizing side effects of androgens
•
SARMs provide a potential new hormonal approach for the treatment
of breast cancer without the virilizing side effects of other androgens
• Enobosarm is a non-steroidal, selective androgen receptor modulator
• Binds specifically to the AR with no binding to other steroidal receptors
• Cannot be aromatized to estrogen
•
Targeting the AR in patients with metastatic breast cancer may
provide at least similar response rates as seen historically with
androgens without the unwanted side effects
5
Phase II, Open Label Study to Examine AR Status and the
Activity of GTx-024 Hormonal Therapy in Women with ER+
and AR+ Metastatic Breast Cancer Who Have Previously
Responded to Hormone Therapy But Are Now Progressing
GTx-024
9 mg QD
22 patients
Day
0
•
•
•
Day
168
Primary Endpoint:
Key Secondary Endpoints:
Clinical Benefit (CR + PR + SD) at 6 monthsin AR+ Breast Cancer
 Clinical Benefit (CR + PR + SD) at 6 months in All Subjects
 PFS by Modified RECIST 1.1 or Death (at completion of trial)
Lead PI- Dr. Beth Overmoyer from Dana Farber
7 clinical sites in US have fully enrolled 22 patients (~60% enrolled by Dana Faber)
Threshold for success – 3 patients out of 14 AR+ meeting primary endpoint
6
Preliminary Results of Phase 2 Trial
• 17 out of 22 patients (77%) were AR+
• 6 of the 17 AR+ patients (35%) who received an assessment at 6
months met the clinical benefit criteria
• 7 out of 22 total patients (32%) who received an assessment at 6
months met the clinical benefit criteria
• After a median duration on study of 81 days, 9 out of 22 patients
(41%) had clinical benefit as best overall response
• Enobosarm was well tolerated with the majority (76%) of adverse
events being Grade 1; no adverse events exceeded Grade 3
• No serious adverse events were definitely related to study drug,
although one SAE has been designated as possibly related
7
AR+ Steering Committee Members
•
•
•
•
•
•
•
•
•
Beth Overmoyer (Dana Farber Harvard)
Hope Rugo (University of California San Francisco)
Hannah Linden (University of Washington)
Joyce O'Shaughnessy (Baylor-Sammons Cancer Center)
Adam Brufsky (University of Pittsburgh)
Hy Muss (University of North Carolina)
Chuck Vogel (University of Miami)
Wayne Tilley (University of Adelaide)
Carlo Palmieri (University of Liverpool)
8
ENOBOSARM 3mg (OSTARINE®; GTx-024)
Selective Androgen Receptor Modulator (SARM)
for the prevention and treatment of muscle wasting
9
US and Europe: Control for negative or positive effects of
chemotherapy
Endpoints
Add-on, placebo-controlled trials
G300504
New
study design slide
G300505
10
Status Update
• Consistent with the Phase 2 trial, enobosarm demonstrated the ability
to increase LBM in both Phase 3 trials
• In the taxane study, enobosarm improved physical function as
assessed by SCP
• Enobosarm was well tolerated in both trials
• No meaningful difference was observed in the pooled analysis of
overall survival
• GTx is evaluating next steps for regulatory submission in Europe and US
11
Enobosarm
Intellectual property
• 74 enobosarm composition of matter and method of use patent
applications issued, approved, or pending in U.S. and rest of world with
expiration dates of at least 2024
– Includes issued composition of matter and method patents in US, Europe
and Japan
– Method of use patents to treat breast cancer may extend patent
coverage to 2033
• As a new chemical entity, issued composition of matter patent should
be eligible for patent term extension of up to 5 years (2029)
• GTx has 248 patents issued, approved or pending worldwide for all
SARMs, including enobosarm
12
CAPESARIS® (GTx-758)
Selective ERα agonist for the treatment of advanced prostate cancer
13
GTx-758 Unique Mechanism of Action
• Increases sex hormone binding globulin (SHBG)
• SHBG binds to and removes free testosterone (T) from the blood,
thereby decreasing levels of the active form of testosterone
• Since GTx-758 is a small molecule with estrogenic activity, it may
ameliorate the estrogen deficiency side effects that are associated
with androgen deprivation therapy (ADT) for prostate cancer
including
•
•
•
•
Increased bone turnover leading to weaker bone and fractures
Hot flashes
Metabolic changes including fat redistribution
Reduced libido and cognitive properties
14
Phase 2, Open Label, Dose Finding, G200712
Clinical Study of Secondary Hormonal Treatment in
Men with Non-Metastatic and Metastatic CRPC
Subjects:
• mCRPC
• nmCRPC
• Maintain ADT
GTx-758
125 mg
Sequential
enrollment
Primary Endpoint:
• Serum PSA response
Secondary Endpoints:
•
•
•
•
•
•
PSA progression
Tumor progression
Free T and SHBG
Adrenal (DHEA&DHEAS)
Estrogen deficiency side effects
SRE
38 patients with metastatic CRPC –fully enrolled
GTx-758
250 mg
Day 0
38 patients with metastatic and nonmetastatic CRPC- enrolling
Day 90
15
Current Clinical Trial (G200712) Summary
• 125 mg dose cohort
• Enrollment complete
• No drug related SAEs or indications of vascular side effects
o Most common AE is gynecomastia (16%)
• SHBG elevated and free T decreased in >90% of patients
• Hot flashes and bone turnover biomarkers decreased
• 250 mg dose cohort
• Enrollment completion estimated Q4 2014
• Preliminary data estimated Q1 2015
• To date, no drug related SAEs or indications of vascular side effects
16
Capesaris
Intellectual Property
• Approximately 53 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 composition of matter 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
17
Financial Summary
• Shares outstanding: 75.2 M
*Warrants outstanding to purchase up to 10.2M shares at $1.67 per share
• Cash, cash equivalents and short-term investments
at $27.8 mm at March 31, 2014
18