Two late stage clinical programs

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Mitchell Martin
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2 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 annual report on Form 10-K filed March 5, We expressly disclaim any obligation to release publicly any updates to forward-looking statements made during the course of this presentation. 2

3 Two late stage clinical programs Enobosarm 3mg (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 3Q 2013 The last blinded DSMB reviewed safety data in April 2013 and agreed trial could continue as planned Enobosarm 9mg (Ostarine; GTx-024), for the treatment of ER+ and AR+ metastatic breast cancer: Phase II 801 clinical trial currently enrolling 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 3

4 Enobosarm (Ostarine; GTx-024) Selective Androgen Receptor Modulator (SARM) for the prevention and treatment of muscle wasting in patients with non-small cell lung cancer

Enobosarm increased lean body mass and improved physical function in three efficacy clinical trials Phase IIb cancer cachexia trial: 159 subjects with cancer cachexia, 4 months tx Change from

5 Enobosarm increased lean body mass and improved physical function in three efficacy clinical trials Phase IIb cancer cachexia trial: 159 subjects with cancer cachexia, 4 months tx Change from baseline (kg) LEAN BODY MASS Placebo Enobosarm 3 mg Change from baseline (%) 30% 25% 20% 15% 10% 5% 0% 5% 10% PHYSICAL FUNCTION Placebo Enobosarm 3 mg STAIR CLIMB Phase II POC clinical trial: 120 elderly men and postmenopausal women, 3 months tx Change from baseline (kg) Placebo Enobosarm 3 mg Change from baseline (%) 25% 20% 15% 10% 5% 0% 5% 10% 15% Placebo Enobosarm 3 mg STAIR CLIMB Phase Ib sarcopenia trial: 88 postmenopausal women, 3 months tx Change from baseline (kg) Placebo Enobosarm 3 mg Dobs, et al. Lancet Oncol. 2013;14(4): Dalton, et al. J Cachexia Sarcopenia Muscle. 2011;2: Marcantonio, et al. Endocrine Reviews. 2010;31(3):(suppl 1)S872. Change from baseline (lbs) Placebo Enobosarm 3 mg BILATERAL LEG PRESS 5

6 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 Apr;91(4):1133S-1137S. Baracos VE data on file. Bruera E. BMJ. 1997;315(7117) Schootman M, et al. Cancer Nov 15;115(22): Braithwaite D, et al. J Natl Cancer Inst. 2010;102(191): Prado CMM, et al. The Lancet Oncology 2008;9(7):

7 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 7

8 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 1 st 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 Lean body mass Physical function 3 months Secondary endpoints Durability of 5 months Overall survival Enobosarm 3 mg 150 patients platinum + taxane platinum + non taxane Placebo Enobosarm 3 mg Placebo 150 patients 150 patients 150 patients Other endpoints QoL FAACT, FACIT fatigue scales Healthcare resource utilization Adherence to chemo plans Tolerance to chemo Input from FDA, MHRA (England) and MPA (Sweden) on Phase III clinical development plans and Fast Track granted 1/13 8

9 Enobosarm Anticipated clinical development plan Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 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) Phase III-POWER 2 Enobosarm 3 mg vs placebo in 300 pts with NSCLC receiving platinum + non taxane chemotherapy (5 month study) NDA First patient enrolled Last patient out 9

10 Enobosarm Steering Committee Medical Oncology/Palliative Care Jeff Crawford (Duke University)-chair Richard Gralla (Quality of Life Research Associates) Phil Bonomi (Rush University Medical Center) Arti Huria(City of Hope) Kavitha Ramchandran (Stanford University) Carla Prado (Florida State University) Aminah Jatoi (Mayo Clinic) Advocacy Bonnie Addario (Bonnie J. Addario Foundation) Other Simon Pickard (University of Illinois) Dave Cella (Northwestern) 10

11 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 $ $3000 the majority of Payors would cover enobosarm as a Tier 3 with a prior authorization * Based on US Managed Healthcare Landscape and Assessment of Enobosarm, Managed Solutions, LLC April

12 Enobosarm Large market opportunity Lung cancer is the most common malignancy Worldwide - estimated 1.6 million new cases and 1.4 million deaths each year United States 226,160 new cases and 160,340 deaths in % present with advanced disease In US, 170,000+ advanced NSCLC patients initiate chemotherapy each year Indication being pursued is prevention and treatment of muscle wasting in patients with NSCLC, a cancer related symptom 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 12

13 Potential new indications for Enobosarm Muscle wasting End stage renal dise Cardiac cachexia COPD Rehab- hip replacement Rehab- knee replacement Glucocorticoids wasting Stroke *AR+ Breast cancer Enobosarm (or other SARM) Male Hypogonadism Gel formulation Endometriosis Anabolic agent Osteoporosis: PMO Male osteoporosis Glucocorticoid induced Dry eyes- Drops formulaton Animal health *Phase II 801 clinical study of enobosarm 9mg currently enrolling 13

14 Enobosarm Intellectual property 307 enobosarm composition of matter and method of use, synthesis and formulation patent applications issued, allowed, or pending in US and rest of world with expiration dates in 2024 for composition of matter and method claims (some method claims have later expiration dates). Includes patents granted in US, EU and Japan As a new chemical entity, issued patents should be eligible for patent term extension in the US for up to 5 years (2029), as may be determined following FDA approval of enobosarm. 14

15 Capesaris (GTx-758) Selective ERα agonist for the treatment of advanced prostate cancer

16 Evolving treatment options for advanced prostate cancer Advanced Prostate Cancer Hormone Sensitive Castration Resistant Prostate Cancer (CRPC) Primary hormone therapy (LHRH agonists or antagonists) Capesaris First Secondary Hormone Tx Capesaris Enzalutamide Second Secondary Hormone Tx Capesaris Enzalutamide Third Secondary Hormone Tx Abiraterone + Prednisone Capesaris Chemotherapy Docetaxel Post Chemotherapy =approved Enzalutamide Abiraterone/ Prednisone Cabazitaxel 750,000 patients 80,000 patients 36,000 patients Market 16

17 Multiple potential mechanisms of action for a selective ERα agonist to treat prostate cancer 17

18 GTx-758 (Capesaris ) Increases SHBG, Reducing Unbound (Free) T Towards Undetectable (%) 18 Adapted from Dunn JF et al. (1981) J Clin Endo and Metabolism, 53:58-68

19 Phase II studies in men with advanced prostate cancer confirms the mechanism of action * 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) Castration rate greater than 90% for both arms Capesaris increased SHBG and decreased free T 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 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 19

20 Phase II, secondary hormonal therapy 707 clinical study in chemotherapy naive CRPC * Inclusion/Exclusion Criteria: Serum PSA > 2ng/ml Castrate (total T <50ng/dl) ECOG 0-2 Maintain primary ADT Endpoints: Serum PSA response > 50% (Primary Endpoint) Serum PSA Progression (PSA > 2 & >25%) Serum free T and SHBG Bone and Soft Tissue metastases Capesaris 2000 mg PO q d 25 patients Day 90 Percent of baseline Serum PSA Response (N=7) Day 1 Day 15 Day 30 Day 60 1S001 1S003 2S001 5S001 5S003 5S004 5S002 Serum PSA responders (>50% reduction) Mean SHBG = 399% ± 85% -90 Days *Studies terminated prior to completion 20

21 Phase II, open label, 712 clinical study of secondary hormonal treatment in men with metastatic CRPC started 3Q 2012 Subjects mcrpc Maintain ADT Primary Endpoint: Serum PSA response Secondary Endpoints: PSA progression Progression free survival Free T/SHBG Adrenal (DHEA&DHEAS) Estrogen deficiency side effects SRE GTx mg 25 patients 30 d GTx mg 25 patients 30 d GTx mg 25 patients Day 0 Day 90 21

22 Hormone naive advanced prostate cancer Combination primary ADT (LHRH + GTx-758) Regulatory approval is based on total testosterone, not PFS or OS FDA established cutoff of <50 ng/dl is based upon limit of detection of older assays Up to 17% of patients on current primary ADT do not achieve this level New consensus cutoff of 20 ng/dl more closely reflects surgical castration 37-50% of patients on primary ADT do not achieve this level Current ADT labels are Palliative treatment of advanced prostate cancer --not PFS or OS Free testosterone (2% of total T) - unbound T is the active fraction Morote, et al suggest that a free T level of 1.7 pg/ml is reasonable cutoff for castration Significant circulating free T remains available for prostate cancer growth with current primary ADT creating a need for maximal suppression of free T Can GTx-758, combined with LHRH agonist, provide greater suppression of free T than Lupron alone for men with suboptimal castration? Co Primary Endpoints Free T and Total T Secondary endpoint improvement of estrogen deficiency side effects Rove K. Urology. 2012;80;4: Novara G, et al. Urol Int. 2009;82: Oefelein A, et al. Urology. 2000;56: Zlotta A, et al. Eur Urol Suppl. 2005;4: Tombal B. Urol Suppl. 2008;7: Morote J, et al. Int J Biol Markers. 2005;20:

23 Capesaris (GTx-758) clinical program Phase II Study 712 Secondary Hormone Therapy Primary endpoint- PSA response Select dose with best efficacy and VTE rate Q Q Phase III Hormone naive prostate cancer (suboptimal castration) Combination ADT Can GTx-758 combined with Lupron provide greater reduction in T than Lupron alone Co Primary Endpoints Total T and Free T Secondary Endpoint Improved estrogen deficiency side effect profile nmcrpc Primary Endpoint- rpfs 23

24 Capesaris Steering Committee Medical oncology Phil Kantoff (Dana Farber Harvard)- Chair Evan Yu (U Washington) Johann DeBono (Royal Marsden) Dan Petrylak (Yale) Chuck Ryan (UCSF) Robert Dreicer (Cleveland Clinic) Thomas Flaig (U Colorado) Urology Badri Konety (U Minnesota) Dan Lin (U Washington) Eric Klein (Cleveland Clinic) Advocacy group Tom Kirk (Us TOO) 24

25 Capesaris Intellectual Property Approximately 43 composition of matter and method of use patents and patent applications 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 new 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 *On account of patent term extension by the USPTO, the term for the US patent on composition of matter was extended 778 days to January 14,

26 Capesaris Anticipated clinical development plan Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q Phase IIb 705 clinical study Maintenance dose finding Phase II 710 clinical study Loading dose finding Phase II 707 clinical study Secondary hormonal therapy in CRPC patients Phase II 712 clinical study Secondary hormonal therapy in mcrpc patients (lower doses)* *Estimated based on projected enrollment 26

27 Financial summary Shares outstanding: 62.8 M Cash, cash equivalents and short-term investments at March 31, 2013: $42.2M No debt, no warrants 27

28 Two late stage clinical programs: milestones Enobosarm (Ostarine; GTx-024), a SARM, for the prevention and treatment of muscle wasting in patients with non-small cell lung cancer: Enrollment completed 4Q 2012 for two pivotal Phase III clinical trials in approximately 650 non-small cell lung cancer (NSCLC) patients top line results expected 3Q 2013 Granted Fast Track designation January 2013 Enobosarm 9mg (Ostarine; GTx-024), for the treatment of ER+ and AR+ metastatic breast cancer: Phase II 801 clinical trial currently enrolling Capesaris (GTx-758), an oral selective ER alpha agonist, for combination primary ADT & secondary hormonal treatment of advanced prostate cancer: Currently enrolling Phase II 712 clinical trial evaluating Capesaris (GTx-758) for secondary hormonal treatment in men with metastatic castration resistant prostate cancer 28

Two late stage clinical programs

Two late stage clinical programs 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