Palatin Technologies, Inc. · AMEX:PTN
Palatin Technologies — Investor Presentation (May 2014)
46 slides from Palatin Technologies's investor presentation, May 2014
Slide 1: Palatin Technologies
Palatin Technologies May 2014 Carl Spana, Ph.D. President & CEO Stephen T. Wills, CPA/MST CFO / COO
Slide 2: Forward-Looking Statement
Forward-Looking Statement The statements in this presentation that relate to future plans, events or performance are forward-looking statements, which are made pursuant to the safe harbor provisions of Section 21E of the Securities Exchange Act of 1934. Such forward- looking statements involve signi
Slide 3: Company Profile
Company Profile Palatin Technologies, Inc. (NYSE MKT: PTN) is a biopharmaceutical company developing targeted, receptor-specific peptide therapeutics for the treatment of diseases with significant unmet medical need and commercial potential. 3
Slide 4: Palatin Opportunity
Palatin Opportunity Well positioned to drive growth through internal pipeline and collaborations Lead product: bremelanotide (BMT) for the treatment of female sexual dysfunction (FSD) ◦ Unmet medical need with multi-billion dollar market opportunity ◦ Phase 2B clinical trial positive – Data presen
Slide 5: Pipeline
Pipeline Preclinical Phase 1 Phase 2 Phase 3 Melanocortin Receptor Programs Bremelanotide for Female Sexual Dysfunction AstraZeneca Obesity, Metabolic Syndrome & Diabetes Collaboration Next Generation Female Sexual Dysfunction & Erectile Dysfunction Peptide Melanocortin Inflammatory & Dermatologic D
Slide 6: Female Sexual Dysfunction Program
PALATIN Female Sexual Dysfunction Program
Slide 7: Female Sexual Dysfunction Overview
Female Sexual Dysfunction Overview Female Sexual Dysfunction (FSD) is defined as persistent or recurring problems during one or more of the stages of sexual response with associated distress FSD is prevalent in U.S. adult women (pre and post-menopausal) ◦ ~44% experiencing some symptoms of FSD ◦
Slide 8: FSD: Significant Untapped Market Opportunity
FSD: Significant Untapped Market Opportunity No FDA approved treatments available >60 million premenopausal women in US o 22% reported a sexual problem (PRESIDE) o 11% were distressed by their sexual problems – One-third sought formal care Market size – U.S. o Potential presenting premenopausa
Slide 9: Bremelanotide Profile
Bremelanotide Profile First-in-class melanocortin receptor-4 agonist for FSD On-demand use with rapid onset of activity and well-tolerated Novel mechanism of action activating endogenous pathways involved in sexual arousal response Evaluated in 31 clinical studies showing efficacy in both FS
Slide 10: Bremelanotide Subcutaneous Development
Bremelanotide Subcutaneous Development Demonstrated optimal pharmacokinetics ◦ Subcutaneous on-demand formulation: less variation in patient exposure ◦ Good therapeutic dosing window to avoid potential adverse events (gastrointestinal and blood pressure effects) associated with over-exposure Com
Slide 11: Female Sexual Response Cycle
Female Sexual Response Cycle M O T Sexual Sexual Stimuli I Receptivity V A T Multiple I reasons for O N engaging in sex Desire Subjective Arousal Sexual satisfaction Non-Sexual rewards Responsive sexual arousal and desire Key points for drug intervention are the sites of bremelanotide action 11
Slide 12: Approaches to Treating FSD
Approaches to Treating FSD Peripheral vasodilators: PDE5 inhibitors and topical prostaglandins ◦ Multiple Phase 2 studies in Female Sexual Arousal Disorder (FSAD) patients, minimal efficacy ◦ Genital vasodilatation not key to sexual response Hormone replacement: low dose transdermal testosterone
Slide 13: Approaches to Treating FSD
Approaches to Treating FSD CNS active agents ◦ Flibanserin is a serotonin 5-HT1A agonist, 5-HT2A antagonist that requires chronic dosing – Phase 3 complete in premenopausal women with HSDD positive results – NDA withdrawn after negative FDA Advisory Panel • Failed to meet co-primary endpoint of da
Slide 14: Bremelanotide Phase 2B FSD Clinical Trial
Bremelanotide Phase 2B FSD Clinical Trial Dose ranging 16 week at-home placebo-controlled study ◦ Subcutaneous dosing: placebo and 0.75 mg, 1.25 mg and 1.75 mg bremelanotide ◦ Target enrollment: 400 patients Used FDA accepted and validated endpoints ◦ Increase in Satisfying Sexual Events (SSEs)
Slide 15: 2B FSD Clinical Trial Patient Population
2B FSD Clinical Trial Patient Population Premenopausal women experiencing acquired sexual dysfunction with associated distress ◦ At least 21 and premenopausal as defined by modified STRAW criteria ◦ Previously normal sexual function ◦ Willing to engage in sexual activity at least once per month fo
Slide 16: Phase 2B Clinical Data
Phase 2B Clinical Data Analysis of the primary and key secondary endpoints of 327 pre-menopausal patients shows clinically meaningful and statistically significant effects for bremelanotide vs. placebo. Analysis was for 1.25 mg & 1.75 mg pooled vs. placebo. Primary endpoint improvement in the nu
Slide 17: Phase 2B Clinical Data
Phase 2B Clinical Data SSE FSFI FSDS-DAO 0.9 5 0 0.8 * *** 1.75mg 1.25mg placebo 0.7 4 -2 0.6 -4 0.5 3 * -6 0.4 0.3 2 -8 0.2 -10 * 0.1 1 0 -12 0 -14 *** 17
Slide 18: Phase 2B Clinical Data
Phase 2B Clinical Data 395 premenopausal FSD patients were randomized to receive drug or placebo 26 patients met the predefined blood pressure withdrawal criteria and were evenly distributed among placebo and active arms of the study 12 patients withdrew for adverse events (2 on placebo), pred
Slide 19: Responder Analysis
Responder Analysis PLACEBO SUBTRACTED 60 30 50 25 40 20 % RESPONDERS % RESPONDERS 30 15 20 10 10 5 0 0 PLACEBO 1.25 mg 1.75 mg 1.25 mg 1.75 mg 19
Slide 20: Return to Normal Function
Return to Normal Function PLACEBO SUBTRACTED 45 25 40 35 20 % RESPONDERS 30 25 15 20 10 15 10 5 5 0 0 PLACEBO 1.25 mg 1.75 mg 1.25 mg 1.75 mg 20
Slide 21: Summary of Bremelanotide FSD Program
Summary of Bremelanotide FSD Program Multiple Phase 2 studies established proof-of-concept in FSD Phase 2B study met all key objectives and supports transition to Phase 3 development Subcutaneous formulation has consistent pharmacokinetics and is well-tolerated Bremelanotide continues to dem
Slide 22: Study 54 Patient Testimony
Study 54 Patient Testimony “[FSD] affects everything. I wonder if he’s happy or not? Am I? It makes you have doubts.” – 062-0053 “It definitely got better. We were more physical with each other. We felt more confident again, like we used to.” – 077-0007 “It changed how we were with each other. We we
Slide 23: FDA Phase 3 Pivotal Program Comments
FDA DRUP Phase 3 Pivotal Program Comments Key Outcomes for Phase 3 program o Patient population HSDD & HSDD with arousal symptoms o 24 week treatment evaluation period o Co-primary endpoints of SSE and FSFI Desire sub-domain (28 day recall) o Key secondary endpoint FSDS-R question #13 o Single bli
Slide 24: Phase 3 Population and Endpoints
Phase 3 Population and Endpoints SSE FSFI-Des FSDS-Q13 1.2 P=0.03 1.2 P=0.001 0 1 1 -0.2 0.8 0.8 -0.4 0.6 0.6 -0.6 0.4 0.4 -0.8 0.2 0.2 -1 0 P=0.008 0 -1.2 - Phase 3 population: HSDD with or without arousal component - Data is from phase 2B Study 54 24
Slide 25: Phase 3 Program Summary
Phase 3 Program Summary Functional Group Detail 2 Pivotal Trials (~1200 patients) N. America 1 Pivotal Trial (~900 patients) EU Clinical 1 Open Label Study Population PK analysis 9 DDI / Ancillary Studies NDA prep/Regulatory Consulting Education/Marketing Regulatory / Marketing / Other Development C
Slide 26: Timelines
Timelines U.S. ◦ End of Phase 2 meetings with FDA - completed 2Q2013 – Final design/protocols pending ◦ Commence Phase 3 trials 2H2014 ◦ Ancillary/DDI studies 2H2014 ◦ FDA NDA submission 1H2016 ◦ FDA approval 1H2017 European ◦ EMA/CHMP guidance - completed 1Q2014 ◦ Commence Phase 3 trial 1Q2015
Slide 27: Bremelanotide Corporate Partnering
Bremelanotide Corporate Partnering Option agreement for European rights ◦ $1M Initial Option Fee; option exercised April 2014 ◦ European pharmaceutical company focused on female health ◦ EMA/ CHMP guidance on the EU phase 3 program received ◦ Jointly working on EU phase 3 clinical development prog
Slide 28: Target Product Profile
Target Product Profile TARGET PRODUCT PROFILE Bremelanotide Subcutaneous Injection for the Treatment of Premenopausal Women with Female Sexual Dysfunction Characterized by Reduced Desire Associated with Distress Bremelanotide is a melanocortin-4 receptor agonist that activates endogenous pathways
Slide 29: Melanocortin Receptor-1 Agonists: Inflammation and Dermatologic Indications
Melanocortin Receptor-1 Agonists Inflammation and Dermatologic Indications
Slide 30: Melanocortin Receptor-1 Agonist Peptides
Melanocortin Receptor-1 Agonist Peptides Developed new families of peptides that are highly specific and selective for the melanocortin receptor-1 (MC1r) ◦ Limited or no off-target effects Broad US and international patent applications MC1r involved in a wide range of physiological responses ◦
Slide 31: Immunological effects of α-MSH
Immunological effects of α-MSH Anti-inflammatory properties of α-MSH have been explored in vitro and in vivo ◦ Effects mediated by inhibition of NF-κB, an inflammatory mediator ◦ Increased production of IL-10, an anti-inflammatory cytokine ◦ Inhibition of proinflammatory cytokines (IL-1, IL-2, IL-
Slide 32: Mouse TNF-a inhibition via MC1r agonists
Mouse TNF-a inhibition* via MC1r agonists Results 3000 Dose – mg/kg SC pmole/mL TNFa 2000 1000 0 1 1 e 10 10 5 cl @ @ @ @ @ hi 76 77 a Ve 76 77 ex 81 81 81 81 D PL PL PL PL *TNF-α stimulated by LPS 32
Slide 33: Melanocortin Receptor-1 Agonist Peptides
Melanocortin Receptor-1 Agonist Peptides Active preclinical research program ◦ Rational design and synthesis of new compounds ◦ Evaluation in in vitro studies ◦ Evaluation in animal studies for number of different indications Lead clinical candidate PL-8177 selective MC1r agonist ◦ Initiated pre
Slide 34: Melanocortin Receptor-4 Agonist: Obesity & Diabetes
Melanocortin Receptor-4 Agonist Obesity & Diabetes
Slide 35: AstraZeneca Obesity Collaboration
AstraZeneca Obesity Collaboration Exclusive global licensing and research collaboration ◦ $10M upfront and $12M milestones received ◦ $145M in additional potential milestones ◦ Royalties upon commercialization Clinical proof-of-concept studies for MC4r mechanism have been completed ◦ Primary obj
Slide 36: Melanocortins and Obesity Overview
Melanocortins and Obesity Overview Hypothalamus arcuate nucleus Stimulates MC4r – decrease in food intake & weight loss POMC Brainstem & Cortex Peripheral Signals MC4r signals Leptin Grelin GLP-1 Down stream effects on Insulin food intake, weight CCK and energy expenditure AGRP Inhibits MC4r – incre
Slide 37: Natriuretic Peptide Receptor Program
PALATIN Natriuretic Peptide Receptor Program
Slide 38: Natriuretic Peptide System
Natriuretic Peptide System Prohormone CNP Neuropeptide hormone system that BNP plays an important role in the regulation ANP of cardiovascular homeostasis Corin GTP Physiological Effects • Down regulate Renin-Angiotensin-Aldosterone System • Suppression of Cardiac Hypertrophy & Remodeling • Stimulat
Slide 39: Overview Natriuretic Program
Overview Natriuretic Program Design and develop commercial candidates selective for NPR-A, NPR-B & NPR-A/B PL-3994 Lead Product candidate ◦ Selective for NPR-A ◦ Increased metabolic stability ◦ Once daily SC patient administration ◦ Phase 2 drug development candidate Validated in clinical stud
Slide 40: Daily Administration of PL-3994 Significantly Reduces Cardiac Remodeling
Daily Administration of PL-3994 Significantly Reduces Cardiac Remodeling Attenuation of RAAS Activation Heart Weight /Body Weight following 6 weeks of treatment 100000 0.6 2K1C (saline) ** ** Plasma aldosterone 10000 2K1C (PL3994) 0.4 (pg/ml) 1000 HW/BW 100 0.2 10 1 0.0 14 42 14 42 7 7 -7 -7 ) 4) )
Slide 41: PL-3994 in Asthma Disease Models
PL-3994 in Asthma Disease Models 100 Isolated guinea-pig trachea 15 Intra-tracheal instillation of PL-3994; ∆ in pulmonary inflation effect on inflation pressure in guinea pigs pressure (cmH 2O) 80 % Inhibition of contraction PL3994 PL5731 10 60 Vehicle 40 5 20 0 control 100 µg/kg 10 µg/kg 1 µg/kg 0
Slide 42: PL-3994 Clinical Results
PL-3994 Clinical Results Phase 1 & 2a placebo-controlled dose escalation studies ◦ Placebo and escalating doses of PL-3994 delivered SC ◦ Endpoints – Safety, ↓SBP, ↑diuresis, ↑natriuresis, ↑cGMP plasma levels Results ◦ Well tolerated - no adverse or severe adverse events ◦ Clear dose response fo
Slide 43: Multiple Opportunities
Multiple Opportunities Treatment of cardiac hypertrophy in patients with low systemically active natriuretic peptides ◦ High clinical need ◦ Strong preclinical support ◦ Emerging clinical profile indicates high probability of treatment benefit Acute Severe Asthma ◦ High clinical need for therapy
Slide 44: Milestones
Milestones Bremelanotide Phase 2B study in FSD patients ◦ Positive Top-line data released 4Q12 ◦ EOP2 meetings with FDA completed 2Q13 ◦ Initiate Phase 3 Pivotal Trials (as early as) 2H14 ◦ Corporate collaboration 2H14 AstraZeneca MCR development obesity/diabetes program ◦ Clinical candidate
Slide 45: Financial Snapshot
Financial Snapshot Ticker Symbol NYSE MKT: PTN Shares Outstanding – March 31, 2014 ◦ Common 39.3M ◦ Fully Diluted 135.1M Cash and Cash Equivalents Balance – March 31, 2014 $16.7M 45
Slide 46: Thank You
Thank You
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