Annual General Meeting KPMG Level 36, 727 Collins St Melbourne Tuesday 21 November am

Size: px
Start display at page:

Download "Annual General Meeting KPMG Level 36, 727 Collins St Melbourne Tuesday 21 November am"

Transcription

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

Interim report January September 2017

Interim report January September 2017 Interim report January September 2017 Conference call, November 13 th, 2017 at 3:00 p.m. (CET) Dial-in number: SE: +46 8 566 425 08, US: +1 646 502 51 18 Peter Wolpert, CEO & Founder Anna Ljung, CFO Disclaimer

More information

Second Quarter 2016 Financial Results. August 4, 2016

Second Quarter 2016 Financial Results. August 4, 2016 Second Quarter 2016 Financial Results August 4, 2016 Cautionary Note Regarding Forward-Looking Statements This presentation and various remarks we make during this presentation contain forward-looking

More information

International Transfers of Personal Data at sanofi-aventis R & D

International Transfers of Personal Data at sanofi-aventis R & D International Transfers of Personal Data at sanofi-aventis R & D Pierre-Yves Lastic, PhD Senior Director, Standards Management & Data Privacy Sanofi-aventis R&D CONFERENCE ON INTERNATIONAL TRANSFERS OF

More information

Rebiotix: Business, Regulatory and Clinical Challenges in Commercializing a Microbiota Based Drug. May 2016

Rebiotix: Business, Regulatory and Clinical Challenges in Commercializing a Microbiota Based Drug. May 2016 Rebiotix: Business, Regulatory and Clinical Challenges in Commercializing a Microbiota Based Drug May 2016 Rebiotix Pioneer in Microbiome Therapeutics Privately held clinical stage biotechnology company

More information

CORPORATE NEWS EARNINGS PAION AG PUBLISHES GROUP QUARTERLY STATEMENT FOR THE FIRST NINE MONTHS OF 2016

CORPORATE NEWS EARNINGS PAION AG PUBLISHES GROUP QUARTERLY STATEMENT FOR THE FIRST NINE MONTHS OF 2016 CORPORATE NEWS EARNINGS PAION AG PUBLISHES GROUP QUARTERLY STATEMENT FOR THE FIRST NINE MONTHS OF 2016 Positive remimazolam data in pivotal U.S. Phase III study for procedural sedation during colonoscopy

More information

Office for Human Subject Protection. University of Rochester

Office for Human Subject Protection. University of Rochester POLICY 1. Purpose Outline the responsibilities and regulatory requirements when conducting human subject research that involves the use of drugs, agents, biological products, or nutritional products (e.g.,

More information

Samatasvir (IDX719), a Potent Pan-Genotypic NS5A Inhibitor, for the Treatment of Hepatitis C Virus (HCV) Infection

Samatasvir (IDX719), a Potent Pan-Genotypic NS5A Inhibitor, for the Treatment of Hepatitis C Virus (HCV) Infection Samatasvir (IDX719), a Potent Pan-Genotypic NS5A Inhibitor, for the Treatment of Hepatitis C Virus (HCV) Infection Douglas Mayers, MD December 11, 2013 1 Idenix: Advancing All-Oral, Pan-Genotypic Combination

More information

OPT-302: a VEGF-C/VEGF-D Trap for wet AMD

OPT-302: a VEGF-C/VEGF-D Trap for wet AMD OPT-302: a VEGF-C/VEGF-D Trap for wet AMD Ophthalmology Innovation Summit, Nov 12 2015 Circadian Technologies (ASX:CIR, OTCQX:CKDXY) Megan Baldwin PhD, CEO & MD megan.baldwin@opthea.com 2 Disclaimer Investment

More information

Early Phase Education WHITEPAPER. Three major items to consider when moving from preclinical to clinical development

Early Phase Education WHITEPAPER. Three major items to consider when moving from preclinical to clinical development Early Phase Education WHITEPAPER Three major items to consider when moving from preclinical to clinical development Three major items to consider when moving from preclinical to clinical development Dr.

More information

CORPORATE PRESENTATION

CORPORATE PRESENTATION A New Path for Antifungal Treatments Overview of SCY-078 -- First Representative of a Novel Oral/IV Triterpenoid Antifungal Family CORPORATE PRESENTATION September 2017 Forward-Looking Statements Certain

More information

Agios Pharmaceuticals, Inc. (Exact Name of Registrant as Specified in Charter)

Agios Pharmaceuticals, Inc. (Exact Name of Registrant as Specified in Charter) UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of report (Date of earliest event

More information

The Drug Development Process and Design of Clinical Trials

The Drug Development Process and Design of Clinical Trials The Drug Development Process and Design of Clinical Trials Darlene Kitterman, MBA Director, Investigator Support & Integration Services September 24, 2014 Clinical Trial Design Guidance Clinical Trial:

More information

Drug Development: Why Does it Cost so Much? Lewis J. Smith, MD Professor of Medicine Director, Center for Clinical Research Associate VP for Research

Drug Development: Why Does it Cost so Much? Lewis J. Smith, MD Professor of Medicine Director, Center for Clinical Research Associate VP for Research Drug Development: Why Does it Cost so Much? Lewis J. Smith, MD Professor of Medicine Director, Center for Clinical Research Associate VP for Research Drug Development Process by which new chemical entities

More information

Developing innovative new medicines for acute and chronic neurological and psychiatric conditions Larry Glass, CEO

Developing innovative new medicines for acute and chronic neurological and psychiatric conditions Larry Glass, CEO Developing innovative new medicines for acute and chronic neurological and psychiatric conditions Larry Glass, CEO (lglass@neurenpharma.com) Neuren Pharmaceuticals Created to commercialize University of

More information

Volunteering for Clinical Trials

Volunteering for Clinical Trials Volunteering for Clinical Trials Volunteering for Clinical Trials When considering volunteering for a clinical trial, it is important to make an informed decision. Below are answers to frequently asked

More information

THE FDA, THE DRUG APPROVAL PROCESS, AND THE PATIENT VOICE

THE FDA, THE DRUG APPROVAL PROCESS, AND THE PATIENT VOICE THE FDA, THE DRUG APPROVAL PROCESS, AND THE PATIENT VOICE Ali Mohamadi, M.D. Senior Director, Global Regulatory Patient Engagement and Policy BioMarin Pharmaceutical 30-July-2016 Goals of Today s Presentation

More information

Injectable modified release products

Injectable modified release products Guideline on the pharmacokinetic and clinical evaluation of modified release dosage forms (EMA/CPMP/EWP/280/96 Corr1) Injectable modified release products Dr Sotiris Michaleas, National Expert for the

More information

Management Discussion and Analysis for the Second quarter of FY

Management Discussion and Analysis for the Second quarter of FY Management Discussion and Analysis for the Second quarter of FY 2016 17 Revenue Figures Consolidated (Rs. In Millions) Average conversion rate in Q2 FY 2016 17 considered is 66.85 /USD 1.00 Average conversion

More information

Comparability Studies for Autologous Cell Therapy Products. Chris Shen July, 2017

Comparability Studies for Autologous Cell Therapy Products. Chris Shen July, 2017 Comparability Studies for Autologous Cell Therapy Products Chris Shen July, 2017 Forward Looking Statements/Safe Harbor To the extent statements contained in this presentation are not descriptions of historical

More information

Copyright. Jeremiah J. Kelly (2015). All rights reserved. Further dissemination without express written consent strictly prohibited.

Copyright. Jeremiah J. Kelly (2015). All rights reserved. Further dissemination without express written consent strictly prohibited. Statutory Framework for Biologics Drugs Investigational Use Application IND Pre-Market Approval Applications 505(b)(1) NDA 505(b)(2) NDA 505(j) ANDA Over-the-Counter (OTC) Non- Rx Drugs Monograph Biologics

More information

The Promise and Challenge of Adaptive Design in Oncology Trials

The Promise and Challenge of Adaptive Design in Oncology Trials THE POWER OFx Experts. Experience. Execution. The Promise and Challenge of Adaptive Design in Oncology Trials Clinical oncology trials are more complex and time consuming than those in any other therapeutic

More information

INVESTOR PRESENTATION FULL YEAR 2007 RESULTS. February 2008

INVESTOR PRESENTATION FULL YEAR 2007 RESULTS. February 2008 INVESTOR PRESENTATION FULL YEAR 2007 RESULTS February 2008 FORWARD LOOKING STATEMENTS This presentation contains forward looking statements. The words believe, expect, anticipate, intend, will, may, would,

More information

Industry Academic Collaboration: A Key to Successful Involvement of Patients Early in Clinical Development

Industry Academic Collaboration: A Key to Successful Involvement of Patients Early in Clinical Development Industry Academic Collaboration: A Key to Successful Involvement of Patients Early in Clinical Development Aernout van Haarst PhD Director, European Corporate Development Feb 2016 Industry Academic Collaboration

More information

INTERIM RESULTS AS OF MARCH 31, 2017

INTERIM RESULTS AS OF MARCH 31, 2017 INTERIM RESULTS AS OF MARCH 31, 2017 1 FORWARD-LOOKING STATEMENTS This presentation includes forward-looking statements that involve risks, uncertainties and other factors, many of which are outside of

More information

Delivering on the Promise of RNA- Based Therapeu;cs

Delivering on the Promise of RNA- Based Therapeu;cs Delivering on the Promise of RNA- Based Therapeu;cs Parent Project Muscular Dystrophy Webinar June 20, 2011 Forward- Looking Statements This presenta,on includes forward- looking statements, including

More information

DUCHENNE MUSCULAR DYSTROPHY CLINICAL DEVELOPMENT PROGRAM

DUCHENNE MUSCULAR DYSTROPHY CLINICAL DEVELOPMENT PROGRAM DUCHENNE MUSCULAR DYSTROPHY CLINICAL DEVELOPMENT PROGRAM Dana R. Martin, PharmD Sarepta Therapeutics PPMD ANNUAL CONNECT CONFERENCE ORLANDO, FLORIDA JUNE 27, 2016 FORWARD LOOKING STATEMENTS This presentation,

More information

Second Quarter 2017 Financial Results. August 8, 2017

Second Quarter 2017 Financial Results. August 8, 2017 Second Quarter 2017 Financial Results August 8, 2017 Agios Conference Call Participants Prepared Remarks Introduction RENEE LECK, Sr. Manager, Investor Relations Business Highlights & 2017 Key Milestones

More information

BIOPHARMA SOLUTIONS TM Expedite Your Drug Development Program

BIOPHARMA SOLUTIONS TM Expedite Your Drug Development Program BIOPHARMA SOLUTIONS TM Expedite Your Drug Program Maximize the Value of Your Asset The journey of drug development can be complex stressful. But it doesn t have to be that way. Join more than 600 biopharmaceutical

More information

Assembly Biosciences Jefferies 2015 Microbiome Summit. December 16, 2015

Assembly Biosciences Jefferies 2015 Microbiome Summit. December 16, 2015 Assembly Biosciences Jefferies 2015 Microbiome Summit December 16, 2015 Forward-Looking Statements This presentation contains forward-looking statements regarding future events. Forward-looking statements

More information

Moberg Pharma AB A DIFFERENT KIND OF PHARMA COMPANY

Moberg Pharma AB A DIFFERENT KIND OF PHARMA COMPANY Moberg Pharma AB A DIFFERENT KIND OF PHARMA COMPANY Corporate Presentation Remium Health Care Capital Market Day Dec 2, 2013 Peter Wolpert, CEO and Founder Disclaimer Statements included herein that are

More information

Eurofins ADME BIOANALYSES Your partner in drug development

Eurofins ADME BIOANALYSES Your partner in drug development Eurofins ADME BIOANALYSES Your partner in drug development Since 1987, you have placed your confidence in us. Our responsibility, to which my staff and I commit, is to consistently provide you with relevant

More information

Regulation of Microbiota- Based Products

Regulation of Microbiota- Based Products Regulation of Microbiota- Based Products LCDR Matthew Steele, PhD Team Leader, Regulatory Review Branch 1 Division of Vaccines and Related Products Applications CBER/OVRR My presentation is an informal

More information

INVESTOR PRESENTATION

INVESTOR PRESENTATION INVESTOR PRESENTATION March 2008 FORWARD LOOKING STATEMENTS This presentation contains forward looking statements. The words believe, expect, anticipate, intend, will, may, would, could and plan and similar

More information

The Australian Clinical Trial Handbook

The Australian Clinical Trial Handbook The Australian Clinical Trial Handbook A simple, practical guide to the conduct of clinical trials to International standards of Good Clinical Practice (GCP) in the Australian context About the Therapeutic

More information

Disclaimer. 2

Disclaimer.  2 www.valirx.com 1 Disclaimer This Document comprises an institutional presentation (the Presentation ) which has been prepared by and is the sole responsibility of ValiRx plc (the Company ). The content

More information

Int. J. Pharm. Sci. Rev. Res., 31(2), March April 2015; Article No. 04, Pages: A Review on Drug Approval in Regulated and Non-Regulated Markets

Int. J. Pharm. Sci. Rev. Res., 31(2), March April 2015; Article No. 04, Pages: A Review on Drug Approval in Regulated and Non-Regulated Markets Review Article A Review on Drug Approval in Regulated and Non-Regulated Markets Vemuri Pavan Kumar, N Vishal Gupta* Pharmaceutical Quality Assurance Group, Department of Pharmaceutics, JSS College of Pharmacy,

More information

World Medical Association Declaration of Helsinki

World Medical Association Declaration of Helsinki World Medical Association Declaration of Helsinki Ethical Principles for Medical Research Involving Human Subjects Adopted by the 18th WMA General Assembly Helsinki, Finland, June 1964 and amended by the

More information

WMA Declaration of Helsinki - Ethical

WMA Declaration of Helsinki - Ethical WMA Declaration of Helsinki - Ethical Principles for Medical Research Involving Human Subjects Download the PDF file Adopted by the 18 th WMA General Assembly, Helsinki, Finland, June 1964 and amended

More information

Clinical Trials How, Why and When? Candidate to Market 16 th May 2012 Kirsty Kwiatkowski

Clinical Trials How, Why and When? Candidate to Market 16 th May 2012 Kirsty Kwiatkowski Clinical Trials How, Why and When? Candidate to Market 16 th May 2012 Kirsty Kwiatkowski Agenda Why are clinical trials conducted? History Guidelines and Regulations When are trials conducted? Requirements

More information

8. Clinical Trial Assessment Phase II

8. Clinical Trial Assessment Phase II 8. Clinical Trial Assessment Phase II Junko Sato, PhD Office of New Drug I, PMDA Disclaimer: The information within this presentation is based on the presenter s expertise and experience, and represents

More information

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP)

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) European Medicines Agency Pre-authorisation Evaluation of Medicines for Human Use London, 22 February 2006 EMEA/CHMP/BMWP/94528/2005 COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) ANNEX TO GUIDELINE

More information

Adaptive Design for Medical Device Development

Adaptive Design for Medical Device Development Adaptive Design for Medical Device Development A guide to accelerate clinical development and enhance portfolio value Executive Summary In May 2015, the FDA released a draft guidance document regarding

More information

Maximizing opportunities towards achieving clinical success D R U G D I S C O V E R Y. Report Price Publication date

Maximizing opportunities towards achieving clinical success D R U G D I S C O V E R Y. Report Price Publication date F o r a c l e a r e r m a r k e t p e r s p e c t i v e Early Stage Drug Safety Strategies & Risk Management Maximizing opportunities towards achieving clinical success D R U G D I S C O V E R Y Report

More information

TECHNOLOGIES & SERVICES FOR THERAPEUTIC ANTIBODY DEVELOPMENT

TECHNOLOGIES & SERVICES FOR THERAPEUTIC ANTIBODY DEVELOPMENT Specialist in tissue analysis by Histology, Immunohistochemistry and In Situ Hybridization TECHNOLOGIES & SERVICES FOR THERAPEUTIC ANTIBODY DEVELOPMENT CONTENTS Histalim: who we are Our areas of expertise

More information

Novartis Business Services HR University Relations. Clinical Sciences and Innovation. Postgraduate Program

Novartis Business Services HR University Relations. Clinical Sciences and Innovation. Postgraduate Program Novartis Business Services HR University Relations Clinical Sciences and Innovation Postgraduate Program 2 CLINICAL SCIENCES AND INNOVATION CLINICAL SCIENCES AND INNOVATION 3 The CS&I Postgraduate Program

More information

Antisense Therapeutics Ltd ASX:ANP January 2017

Antisense Therapeutics Ltd ASX:ANP January 2017 Antisense Therapeutics Ltd ASX:ANP January 2017 Forward Looking Statements This presentation contains forward-looking statements regarding the Company s business and the therapeutic and commercial potential

More information

Pharmaceutical Development (Drug Substance & Drug Product) for Visceral Leishmaniasis candidate DNDI-6148

Pharmaceutical Development (Drug Substance & Drug Product) for Visceral Leishmaniasis candidate DNDI-6148 Request for Proposal Pharmaceutical Development (Drug Substance & Drug Product) for Visceral Leishmaniasis candidate DNDI-6148 Dated: October 12 th 2015 Page 1 Table of Contents 1. PURPOSE... 3 2. RFP

More information

HIKMA PHARMACEUTICALS PLC. Merrill Lynch Middle East & North Africa (MENA) Conference 13 December r 2005

HIKMA PHARMACEUTICALS PLC. Merrill Lynch Middle East & North Africa (MENA) Conference 13 December r 2005 HIKMA PHARMACEUTICALS PLC Merrill Lynch Middle East & North Africa (MENA) Conference 13 December r 2005 Important notice This document and its contents are confidential and may not be redistributed or

More information

Recombinant Biglycan for the Treatment! of Duchenne Muscular Dystrophy!!! PPMD Annual Conference, Jun 2013! By Joel B. Braunstein, MD!

Recombinant Biglycan for the Treatment! of Duchenne Muscular Dystrophy!!! PPMD Annual Conference, Jun 2013! By Joel B. Braunstein, MD! Recombinant Biglycan for the Treatment! of Duchenne Muscular Dystrophy!!! PPMD Annual Conference, Jun 2013! By Joel B. Braunstein, MD! Tivorsan Overview Commercial operations began in 2010 around biglycan

More information

Summary of Provisions in 21 st Century Cures Act (H.R. 6) as passed by full House of Representatives, July 10, 2015

Summary of Provisions in 21 st Century Cures Act (H.R. 6) as passed by full House of Representatives, July 10, 2015 Pediatric-Specific Provisions Summary of Provisions in 21 st Century Cures Act (H.R. 6) as passed by full House of Representatives, July 10, 2015 Requires the NIH to complete a strategic plan, and in the

More information

For personal use only

For personal use only Company Announcement 31 May 2011 Phosphagenics Limited JUNE 2011 NEWSLETTER Attached for release to the market is a copy of the Company s June 2011 Newsletter to shareholders. In addition to receiving

More information

Advancing New Treatments for DMD and C. difficile Infection

Advancing New Treatments for DMD and C. difficile Infection Advancing New Treatments for DMD and C. difficile Infection 34 th Annual Canaccord Genuity Growth Conference August 13, 2014 Legal Disclaimer FORWARD-LOOKING STATEMENTS This Document contains forward-looking

More information

When to Stop Dose Escalation: MTD, MLD or?

When to Stop Dose Escalation: MTD, MLD or? When to Stop Dose Escalation: MTD, MLD or? Henri CAPLAIN First Joint Annual Meeting, AGAH Club Phase 1 STRASBOURG, France 1 So called MTD STOP FIM No MTD? 2 WHY WE NEED TO REACH THE MAXIMUM TOLERATED DOSE

More information

35th Annual J.P. Morgan Healthcare Conference

35th Annual J.P. Morgan Healthcare Conference 35th Annual J.P. Morgan Healthcare Conference Investor Presentation Nachum Homi Shamir President and Chief Executive Officer January, 2017 Safe Harbor Statement Certain statements made during the course

More information

GLOBAL HEALTH SUPPLY CHAIN QUALITY ASSURANCE

GLOBAL HEALTH SUPPLY CHAIN QUALITY ASSURANCE GLOBAL HEALTH SUPPLY CHAIN QUALITY ASSURANCE Finished Pharmaceutical Product Questionnaire This questionnaire is used to collect information from vendors with regards to finished pharmaceutical products

More information

Spark Therapeutics, Inc. (Exact Name of Registrant as Specified in its Charter)

Spark Therapeutics, Inc. (Exact Name of Registrant as Specified in its Charter) UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event

More information

Stem Cell Services. Driving Innovation for Stem Cell Researchers

Stem Cell Services. Driving Innovation for Stem Cell Researchers Driving Innovation for Stem Cell Researchers Stem Cell Services Partner with us and have access to the most advanced and comprehensive stem cell services available today. 675 W. Kendall St. Cambridge,

More information

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) GUIDELINE ON THE NON-CLINICAL DEVELOPMENT OF FIXED COMBINATIONS OF MEDICINAL PRODUCTS

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) GUIDELINE ON THE NON-CLINICAL DEVELOPMENT OF FIXED COMBINATIONS OF MEDICINAL PRODUCTS European Medicines Agency London, 24 January 2008 Doc. Ref. EMEA/CHMP/SWP/258498/2005 COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) GUIDELINE ON THE NON-CLINICAL DEVELOPMENT OF FIXED COMBINATIONS

More information

uniqure Announces First Clinical Data From Second Dose Cohort of AMT-060 in Ongoing Phase I/II trial in Patients with Severe Hemophilia B

uniqure Announces First Clinical Data From Second Dose Cohort of AMT-060 in Ongoing Phase I/II trial in Patients with Severe Hemophilia B uniqure Announces First Clinical Data From Second Dose Cohort of AMT-060 in Ongoing Phase I/II trial in Patients with Severe Hemophilia B -- Second-dose Cohort Demonstrates Dose Response with All Patients

More information

Definitive Merger Agreement Conference Call

Definitive Merger Agreement Conference Call Definitive Merger Agreement Conference Call Committed to providing products and services that ensure the best possible patient outcomes August 15, 2017 NASDAQ: SKLN Forward-Looking Statements This presentation

More information

Pharmaceutical Industry Research Credit Audit Guidelines

Pharmaceutical Industry Research Credit Audit Guidelines Pharmaceutical Industry Research Credit Audit Guidelines 1. Preface and Overview 2. Summary of Recommended Audit Procedures 3. Overview of IRC Sections 174 and 41 4. Product Development Process a. Stage

More information

Efficacy Report Summarization for SoClean 2

Efficacy Report Summarization for SoClean 2 Efficacy Report Summarization for SoClean 2 October 2017 SoClean Inc 36 Town Forest Road Oxford, Massachusetts 01540 USA Tel. 508-363-0418 Email info@soclean.com SoClean 2 is USA FDA Registered 3009534409

More information

Long-Term Follow-Up in Gene Transfer Clinical Research

Long-Term Follow-Up in Gene Transfer Clinical Research Long-Term Follow-Up in Gene Transfer Clinical Research Jan P. Vleck, MD CIP Institutional Biosafety Committee Services A Division of WIRB www.ibcservicepoint.com ibcs@wirb.com What is LTFU? the collection

More information

Blood procurement: Process development, clinical trials and the market

Blood procurement: Process development, clinical trials and the market Teaching your cells to treat your disease Blood procurement: Process development, clinical trials and the market Christelle Boniface, MSc. Miguel Forte, MD PhD ISCT annual meeting 2016, 25 May 2016 1 Disclaimer

More information

Session 7 Clinical Trial Assessment Bioequivalence Studies

Session 7 Clinical Trial Assessment Bioequivalence Studies L1 Session 7 Clinical Trial Assessment Bioequivalence Studies Presentation to APEC Preliminary Workshop on Review of Drug Development in Clinical Trials Celia Lourenco, PhD, Manager, Clinical Group I Office

More information

ECCMID SCY-078 Scientific Data Presentation Conference Call

ECCMID SCY-078 Scientific Data Presentation Conference Call A New Path for Antifungal Treatments ECCMID SCY-078 Scientific Data Presentation Conference Call April 25, 2017 4:05pm ET Forward-Looking Statements Certain statements regarding SCYNEXIS, Inc. (the Company

More information

Pharmacology. Chatchai Chinpaisal, Ph.D. Department of Pharmacology and Toxicology, Faculty of Pharmacy, Silpakorn University.

Pharmacology. Chatchai Chinpaisal, Ph.D. Department of Pharmacology and Toxicology, Faculty of Pharmacy, Silpakorn University. Pharmacology Chatchai Chinpaisal, Ph.D. Department of Pharmacology and Toxicology, Faculty of Pharmacy, Silpakorn University. 1 PHARMACODYNAMIC STUDIES A. Primary pharmacodynamics primary action in target

More information

Guidelines: c. Providing the investigational drug for the requested use will not interfere with the initiation, conduct, or completion of clinical

Guidelines: c. Providing the investigational drug for the requested use will not interfere with the initiation, conduct, or completion of clinical Guidelines for the Submission of an Expanded Access IND to Permit Diagnosis, Monitoring or Treatment of Intermediate-size Patient Populations with an Investigational Drug or a REMS-restricted, Approved

More information

Expanded Access. to Investigational Drugs & Biologics. for Treatment Use

Expanded Access. to Investigational Drugs & Biologics. for Treatment Use SJMHS Research Compliance Office Guidance Document Expanded Access to Investigational Drugs & Biologics for Treatment Use May 2015 1 Expanded Access to Investigational Drugs & Biologics for Treatment Use

More information

Canadian Cancer Clinical Trials Network Portfolio Eligibility Criteria and Guidelines

Canadian Cancer Clinical Trials Network Portfolio Eligibility Criteria and Guidelines Canadian Cancer Clinical Trials Network Portfolio Eligibility Criteria and Guidelines 1. Introduction The Canadian Cancer Clinical Trials Network (3CTN) will support a portfolio of academic clinical trials

More information

ICH Topic E16 Genomic Biomarkers Related to Drug Response: Context, Structure and Format of Qualification Submissions. Step 3

ICH Topic E16 Genomic Biomarkers Related to Drug Response: Context, Structure and Format of Qualification Submissions. Step 3 European Medicines Agency June 2009 EMEA/CHMP/ICH/380636/2009 ICH Topic E16 Genomic Biomarkers Related to Drug Response: Context, Structure and Format of Qualification Submissions Step 3 NOTE FOR GUIDANCE

More information

IMP Management and Accountability

IMP Management and Accountability This is a controlled document. The master document is posted on the JRCO website and any print-off of this document will be classed as uncontrolled. Researchers and their teams may print off this document

More information

PRELIMINARY OXIDE HEAP LEACH TESTWORK DELIVERS EXCELLENT RESULTS AT THE SOUTH HOUNDÉ PROJECT

PRELIMINARY OXIDE HEAP LEACH TESTWORK DELIVERS EXCELLENT RESULTS AT THE SOUTH HOUNDÉ PROJECT NEWS RELEASE APRIL 7, 2014 PRELIMINARY OXIDE HEAP LEACH TESTWORK DELIVERS EXCELLENT RESULTS AT THE SOUTH HOUNDÉ PROJECT VANCOUVER, CANADA. Sarama Resources Ltd. ( Sarama or the Company ) is pleased to

More information

LTX 109. BIO International Philadelphia 2015

LTX 109. BIO International Philadelphia 2015 LTX 109 BIO International Philadelphia 2015 Lytix Biopharma company background Established in 2003 to develop and commercialize medical applications of lytic peptides originating from the University of

More information

Bioavailability and Bioequivalence Studies

Bioavailability and Bioequivalence Studies Bioavailability and Bioequivalence Studies Standard Approach Part I: Design and Conduct H. Rettig, Ph.D. LLC www.ivivc.com Note for Guidance on the Investigation of Bioavailability and Bioequivalence CPMP/EWP/QWP/1401/98

More information

Reflections on a career in Pharmaceutical Statistical Sciences: My Journey. James R. Johnson, PhD (UD04)

Reflections on a career in Pharmaceutical Statistical Sciences: My Journey. James R. Johnson, PhD (UD04) Reflections on a career in Pharmaceutical Statistical Sciences: My Journey James R. Johnson, PhD (UD04) 1 My Journey 7 Different Pharmaceutical Companies 30 years (1983 to 2013) Retired to Consulting and

More information

PRODUCT SAFETY RESEARCH POLICY

PRODUCT SAFETY RESEARCH POLICY For more information about Colgate-Palmolive please go to www.colgate.com or call our Consumer Affairs Department at 1 (800) 763-0246 PRODUCT SAFETY RESEARCH POLICY Delivering Safe and Effective Products

More information

Regulatory Pathways. Devices vs. Drugs Are there roles for registries? John Laschinger, MD CDRH/ODE/DCD/SHDB

Regulatory Pathways. Devices vs. Drugs Are there roles for registries? John Laschinger, MD CDRH/ODE/DCD/SHDB Regulatory Pathways Devices vs. Drugs Are there roles for registries? John Laschinger, MD CDRH/ODE/DCD/SHDB johnlaschinger@fda.hhs.gov 1 Disclosures and Disclaimer John C. Laschinger, M.D. I am a full

More information

Smart Parking Limited ASX:SPZ AGM Presentation. CEO Paul Gillespie 10th November 2017

Smart Parking Limited ASX:SPZ AGM Presentation. CEO Paul Gillespie 10th November 2017 Smart Parking Limited ASX:SPZ AGM Presentation CEO Paul Gillespie 10th November 2017 Business Overview World-leading technology for parking design, development & management Management Services UK Profitable

More information

Structure and content of an IMPD. What is required for first into man trial?

Structure and content of an IMPD. What is required for first into man trial? What is required for first into man? The EU IMPD Thomas Sudhop, MD Scope Structure and content of an IMPD What is required for first into man trial? Only for IMPs that do not have a marketing authorisation

More information

Investor Presentation: Phase II Data for LCP Tacro. March 3, 2008

Investor Presentation: Phase II Data for LCP Tacro. March 3, 2008 Investor Presentation: Phase II Data for LCP Tacro March 3, 2008 FORWARD LOOKING STATEMENTS This presentation contains forward looking statements. The words believe, expect, anticipate, intend, will, may,

More information

GMO Technology Conference

GMO Technology Conference GMO Technology Conference The regulation of Clinical Trials on humans involving therapies containing or consisting of genetically modified organisms The Printworks, Dublin Castle 10 th & 11 th October

More information

CRO partner in Rx/CDx Co-Development

CRO partner in Rx/CDx Co-Development CRO partner in Rx/CDx Co-Development DEDICATED DIALOGUE A sponsored roundtable discussion published in Pharmaceutical Executive Two Covance executives discuss a CRO s role in supporting Companion Diagnostics

More information

Regulatory Challenges of Global Drug Development in Oncology. Jurij Petrin, M.D. Princeton, NJ

Regulatory Challenges of Global Drug Development in Oncology. Jurij Petrin, M.D. Princeton, NJ Regulatory Challenges of Global Drug Development in Oncology Jurij Petrin, M.D. Princeton, NJ Topics General global R&D issues Regulatory issues with global oncology drug development US FDA initiatives

More information

The New News in Stem Cell Research Andrés Bratt-Leal, PhD 12/1/2017

The New News in Stem Cell Research Andrés Bratt-Leal, PhD 12/1/2017 The New News in Stem Cell Research Andrés Bratt-Leal, PhD 12/1/2017 Cell Therapy and Parkinson s Disease Very specific neural degeneration >50% of DA neurons are gone by diagnosis 1 million in the USA,

More information

MEDICAL DEVICE CLINICAL INVESTIGATIONS AND ISO 14155

MEDICAL DEVICE CLINICAL INVESTIGATIONS AND ISO 14155 MEDICAL DEVICE CLINICAL INVESTIGATIONS AND ISO 14155 EXECUTIVE SUMMARY Medical device regulations around the world generally require manufacturers of most types of medical devices to supply data as part

More information

A full-service CRO with integrated early-stage capabilities

A full-service CRO with integrated early-stage capabilities CHARLES RIVER TO ACQUIRE ARGENTA and BIOFOCUS A full-service CRO with integrated early-stage capabilities James C. Foster Chairman, President & CEO Thomas F. Ackerman Executive Vice President & CFO March

More information

Human Research Protection Program Good Clinical Practice Guidance for Investigators Regulatory File Essential Documents

Human Research Protection Program Good Clinical Practice Guidance for Investigators Regulatory File Essential Documents Guidance for s Regulatory File Essential s All principal investigators must maintain a regulatory binder or file system, which contains all study documentation. These records may be reviewed at the time

More information

Fujifilm s Initiatives in Regenerative Medicine

Fujifilm s Initiatives in Regenerative Medicine Fujifilm s Initiatives in Regenerative Medicine Shigetaka Komori Chairman and CEO FUJIFILM Holdings Corporation March 30, 2015 Priority Business: Healthcare Expand business with focus on six core business

More information

FDA S GUIDANCE FOR INDUSTRY ANDAS: STABILITY TESTING OF DRUG SUBSTANCES AND PRODUCTS

FDA S GUIDANCE FOR INDUSTRY ANDAS: STABILITY TESTING OF DRUG SUBSTANCES AND PRODUCTS FDA S GUIDANCE FOR INDUSTRY ANDAS: STABILITY TESTING OF DRUG SUBSTANCES AND PRODUCTS 02-December-2014 San Diego, CA Kim Huynh-Ba Executive Director PHARMALYTIK Kim.huynhba@pharmalytik.com Overview Stability

More information

Expanded Access to Investigational Imaging Drugs

Expanded Access to Investigational Imaging Drugs Expanded Access to Investigational Imaging Drugs Phillip B. Davis, MD FDA/CDER/ODEIV/DMIP 6/09/2016 1 Overview Expanded Access (EA) Defined Requirements for all EA authorizations Types of EA Individual

More information

1.4 Applicable Regulatory Requirement(s) Any law(s) and regulation(s) addressing the conduct of clinical trials of investigational products.

1.4 Applicable Regulatory Requirement(s) Any law(s) and regulation(s) addressing the conduct of clinical trials of investigational products. 1.1 Adverse Drug Reaction (ADR) In the pre-approval clinical experience with a new medicinal product or its new usages, particularly as the therapeutic dose(s) may not be established: all noxious and unintended

More information

How to rescue a clinical trial

How to rescue a clinical trial How to rescue a clinical trial Clinical trial conduct is demanding and rigorous, especially in orphan indications where patient populations are small. Even well-qualified, specifically selected Contract

More information