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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