Annual General Meeting KPMG Level 36, 727 Collins St Melbourne Tuesday 21 November am
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1 Annual General Meeting 2017 KPMG Level 36, 727 Collins St Melbourne Tuesday 21 November am
2 Forward looking statements Certain statements in this presentation relate to the future, including forward looking statements relating to Hexima s future expectations, beliefs, goals, plans, prospects, financial position and strategy. These forward looking statements involve known and unknown risks, uncertainties, assumptions and other important factors that could cause the actual results, performance or achievements of Hexima to be materially different from future results, performance or achievements expressed or implied by such statements. Neither Hexima nor any other person gives any representation, assurance or guarantee that the occurrence expressed or implied in any forward looking statements in this document will actually occur and you are cautioned not to place undue reliance on such forward looking statements. Subject to any continuing obligations under applicable law, Hexima disclaims any obligation or undertaking to disseminate any updates or revisions to any forward looking statements in this document to reflect any change in expectations in relation thereto or any change in events, conditions or circumstances on which any such statement is based.
3 AGM 2017 Dr Nicole van der Weerden Chief Executive Officer
4 Key achievements for 2017 Successfully closed $3.9 mil rights issue to fund clinical development of HXP124 as a treatment for fungal nail infections. Demonstrated that HXP124 has excellent activity in an Infected Nail Model Completed pre-clinical toxicology program for HXP124. Repeat dose dermal toxicity study in minipigs Received HREC approval to conduct a phase I/IIa clinical trial to test the safety and efficacy of HXP124 as a treatment for fungal nail infections. Patient recruitment to commence in December Dosing to commence in January 4
5 Key achievements for 2017 Three patent families granted in multiple Jurisdictions Improved variants of defensins (human and plant applications) USA, Japan, China, Mexico, Europe (previously granted in Australia) Defensin-defensin synergy (human and plant applications) Australia, South Africa Defensin synergy for onychomycosis USA Government grants. $50k Research Connections grant to study effect of Hexima s lead defensins in a rat model of Vulvovaginal candidiasis. $50k Research Connections grant to continue development of the enzymatic ligation technology. SIEF STEM+ Business Fellowship to fund Dr James McKenna to investigate the effect of defensins on Candida infections and biofilms. 5
6 HXP124 Hexima is developing a novel therapeutic (HXP124) for the treatment of fungal nail infections (onychomycosis). HXP124 has the potential to be superior to current therapies. Potent, broad-spectrum antifungal molecule Member of the Plant Defensin class of molecules Readily penetrates nails Rapidly kills the fungus 6
7 Global onychomycosis market US$3.06 billion in 2015 and projected to reach US$4.7 billion by Major deficiencies in current therapies. Poor efficacy rates Long treatment times Oral therapies can be toxic Expensive Estimated that between 50 and 90% of individuals with fungal nail infections are not receiving treatment. Large potential for rapid growth in the market with an effective product. 7
8 HXP124 kills fungi better than current treatments for nail infections 100% 90% Kills fungal cells within 30 min. Inefficient killing by the drugs currently on the market means the fungus is likely to become resistant during long treatment times and may regrow when treatment is stopped. Percent living cells 80% 70% 60% 50% 40% 30% 20% 10% 0% HXP124 Efinaconazole (Jublia ) Tavaborole (Kerydin ) Terbinafine Ciclopirox (Penlac ) Fluorescence Associated Cell Sorting (FACS) of Propidium Iodide stained cells was used to identify living and dead Candida albicans cells after 30 min treatment with various antifungal molecules. 8
9 HXP124 is as effective as efinaconazole in an infected nail model MedPharm (UK) tested HXP124 in an infected nail model to provide additional confidence that HXP124 passes through nails and kills fungal cells. Industry standard assay. Nail and fungal growth conditions more representative of clinical condition. 2. HXP124 applied topically nail adapter human nail 1. fungal infection developed on underside of nail Jublia and Penlac were used as comparator products in this study. Jublia is the current industry gold standard. 3. Viability of fungal cells measured 9
10 HXP124 is as effective as efinaconazole in an infected nail model HXP124 killed over 95% of fungal cells within 7 days and was as efficient as Jublia in this model. 40% 2. HXP124 applied topically nail adapter human nail 1. fungal infection developed on underside of nail ATP levels* (% relative to infected control) 30% 20% 10% 3. Viability of fungal cells measured 0% HXP124 Jublia Penlac HXP124 Jublia Penlac HXP124 Jublia 7 days 14 days 21 days *ATP levels are used as a measure of cell survival 10
11 FY 2017 progress Pre-clinical animal toxicology studies are complete and clinical trials have been initiated. CY 2017 CY 2018 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Pre-clinical animal toxicology studies Complete Phase I/IIa human clinical safety and efficacy studies On-track United States Food and Drug Administration Investigational New Drug (IND) application Business Development Continuing Proof of concept studies for new fungal disease indications Continuing 11
12 HXP124 was not toxic in a dermal irritation minipig study 42-day GLP repeat-dose dermal toxicity study in minipigs No Observable Adverse Effect Level (NOAEL) of 50 mg/kg (highest dose) Very low absorption through the skin No systemic toxicity effects at the highest dose (50 mg/kg) Very slight dermal irritation at dose site also present in the placebo group 12
13 Pathway to ethics approval In vitro pharmacology & efficacy studies Toxicology in animals GMP manufacture Formulation Safety pharmacology Drug stability studies Container closure Dosage and administration Clinical trial Protocol Advertising and recruitment Patient compliance and consent Insurance and indemnity Reviewed and approved by Human Research Ethics Committee Clinical trial
14 Clinical trial to begin recruiting patients in Dec 2017 Randomised, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study in Healthy Volunteers with Mild to Moderate Onychomycosis. Approval received from Human Research Ethics Committee (Nov 2017) Patient screening to commence in early December First dosing in early January Patients will treat nails daily with HXP124 (or placebo) for 42 days Trial conducted by Linear Clinical Research (Perth) Trial managed by Clinical Network Services (Australia)
15 Clinical trial design 7 days 7 days Cohort 3 HXP124 2% N=6 patients (randomised 2:1) Cohort 2 HXP124 1% N=6 patients (randomised 2:1) Cohort 1 HXP % N=6 patients randomised 2 (drug):1(placebo) 12-week follow up Cohort 4 Highest safe dose N=24 patients (randomised 3:1) 12-week follow up 52-week follow up 42 days daily dosing Jan 2018 May-Jun 2018 Sep-Oct 2018 *Note timelines depend on recruitment rates 15
16 Phase I/IIa clinical trial endpoints Primary endpoint Safety and tolerability Secondary endpoints Preliminary efficacy data Mycological cure (clearance of fungi from the nail) Partial cure (clear nail growth) Complete cure (mycological cure and clear nail growth at 12 months) Infected area Nail matrix Day 0 Day 90 Clear nail growth Schematic representation of method to assess clear nail growth. Low patient numbers Preliminary efficacy results unlikely to be statistically significant BUT - Inform the design of further phase II clinical trials 16
17 Recruitment strategy Clinical trial sites Internal databases (Linear has 14,000 patients on file) Targeted advertising campaign Recruitment vendor (Clinical Trial Group): Dedicated website Facebook ads and split testing Facebook page campaign Targeted patient populations and digital marketing Pre-screening of potential applicants 17
18 HXP124 commercialisation plan Proof-of-concept efficacy data is a major value creation step during drug development. If positive clinical efficacy data is obtained, Hexima intends to licence HXP124 to a pharmaceutical companie(s) to generate upfront and future milestone payments and licensing revenue. Proof-of-concept efficacy data is expected to be obtained in mid-late
19 Licencing HXP124 in Japan Hexima is continuing discussions with several Japanese companies regarding a licence for HXP124. Hexima believes that the large, proven Japanese market for onychomycosis makes it a key target market for HXP124. Hexima believes that a co-development process with a Japanese partner is the most effective way to obtain approval for HXP124 in the Japanese market. Discussions will continue over the coming year Licence negotiations will occur once clinical data is available. 19
20 Summary Hexima is developing HXP124 as a superior therapeutic for onychomycosis. Better nail penetration. Rapidly kills fungal cells. Anticipating shorter treatment regimen. 6 weeks vs 48 weeks. Good safety profile A phase I/IIa clinical trial has been initiated. Approved by HREC Recruitment to begin in December, dosing in January 20
21 Hexima s defensin platform is applicable to other fungal diseases Vulvovaginal candidiasis (thrush) HXP124 and other Hexima lead candidates rapidly kill Candida spp HXP124 is stabile in a topical formulation which is a significant advantage for this application $50k Research Connections grant received Systemic candidiasis SIEF STEM+ Fellowship for Dr James McKenna (La Trobe University) to conduct preliminary research Fungal infections of traumatic injuries/wounds HXP124 and other lead candidates kill non-dermatophyte moulds, the most common pathogens in fungal infected wounds. Fungal skin infections and dandruff Fungal sinusitis Veterinary applications 21
22 Defensins are active in a rat model of vaginal thrush Topical treatment with defensin reduced the number of C. albicans cells in vaginal fluid by 70-87% within 3 days Less effective than miconazole after 5 days BUT - Miconazole is fungistatic and yeast cells re-emerge after 9-12 days Need to assess lead defensin over longer period to ensure cell number continues to decrease Any follow-up will be conducted after clinical results for HXP124 in onychomycosis 22
23 C. albicans biofilm development Candida biofilms on medical device implants are a major cause of systemic infections Help protect the fungus from antifungal drugs Hexima s defensins kill established Candida biofilms Growth and development of a C. albicans based biofilm schematic illustrating the stages of C. albicans biofilm development. Attachment: yeast cells adhere to a substrate forming a basal layer of cells. Proliferation: cells reproduce and form germ tubes. Mature biofilm: hyphae are formed and extracellular matrix accumulates. Dispersal: the mature biofilm releases cells to seed new locations. Modified from Tsui et al SEM of C. albicans in a biofilm. Modified from Tsui et al
24 Dow-DuPont Pioneer Insect Project Project is funded by Dow-DuPont until December 2018 ~9000 diverse plant samples and 46,500 bacteria have been collected (in 3.8 yrs) Novel leads continue to progress through the development pipeline at Dow-DuPont Implications of the Dow-DuPont merger not yet clear No immediate impact anticipated
25 Focus for 2018 HXP124 clinical trial Patient recruitment and enrolment Dosing and safety assessments for dose escalation Preliminary efficacy assessment Business development activities for HXP124 Engaging with BD and licencing experts Licencing discussions with preferred partners to begin once clinical data is obtained FDA Investigational New Drug (IND) application-enabling activities for HXP124 Formulation stability studies Mechanism of action studies Improving production yield for HXP124 to reduce cost of goods Dow-DuPont Insect Project Expand the collection of Australian plants and bacteria Continue development of novel leads 25
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