CORPORATE PRESENTATION - March 2019 I. CORPORATE HIGHLIGHTS II. LEADERSHIP IN NASH & PBC - GENFIT
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MARCH2019
CORPORATE
PRESENTATION
March 2019
I. CORPORATE HIGHLIGHTS
II. LEADERSHIP IN NASH & PBC
1
Disclaimer
Important Information and Forward Looking Statements
IMPORTANT NOTICE - YOU MUST READ THE FOLLOWING BEFORE CONTINUING
•THIS PRESENTATION HAS BEEN PREPARED BY GENFIT AND IS FOR INFORMATION PURPOSES ONLY.
•CERTAIN OF THE INFORMATION CONTAINED HEREIN CONCERNING ECONOMIC TRENDS AND PERFORMANCE IS BASED UPON OR DERIVED FROM
INFORMATION PROVIDED BY THIRD-PARTY CONSULTANTS AND OTHER INDUSTRY SOURCES. WHILE GENFIT BELIEVES THAT SUCH INFORMATION IS
ACCURATE AND THAT THE SOURCES FROM WHICH IT HAS BEEN OBTAINED ARE RELIABLE, GENFIT HAS NOT INDEPENDENTLY VERIFIED THE ASSUMPTIONS
ON WHICH PROJECTIONS OF FUTURE TRENDS AND PERFORMANCE ARE BASED. IT MAKES NO GUARANTEE, EXPRESS OR IMPLIED, AS TO THE ACCURACY AND
COMPLETENESS OF SUCH INFORMATION.
•THIS PRESENTATION CONTAINS CERTAIN FORWARD-LOOKING STATEMENTS OR INFORMATION. ALTHOUGH THE COMPANY BELIEVES ITS EXPECTATIONS
ARE BASED ON REASONABLE ASSUMPTIONS, THESE FORWARD-LOOKING STATEMENTS OR INFORMATION ARE SUBJECT TO NUMEROUS RISKS AND
UNCERTAINTIES, WHICH COULD CAUSE ACTUAL RESULTS TO DIFFER MATERIALLY FROM THOSE EXPRESSED IN, OR IMPLIED OR PROJECTED BY, THE
FORWARD-LOOKING STATEMENTS. THESE RISKS AND UNCERTAINTIES INCLUDE AMONG OTHER THINGS, THE UNCERTAINTIES INHERENT IN RESEARCH AND
DEVELOPMENT, INCLUDING RELATED TO BIOMARKERS, PROGRESSION OF, AND RESULTS OF CLINICAL DATA FROM, THE RESOLVE-IT TRIAL, REVIEW AND
APPROVALS BY REGULATORY AUTHORITIES, SUCH AS THE FDA OR THE EMA, REGARDING IN PARTICULAR, ELAFIBRANOR IN NASH AND PBC, AS WELL AS
OTHER DRUG CANDIDATES IN OTHER INDICATIONS, AND BIOMARKERS, THE SUCCESS OF ANY INLICENSING STRATEGIES, THE COMPANY’S CONTINUED
ABILITY TO RAISE CAPITAL TO FUND ITS DEVELOPMENT, AS WELL AS THOSE DISCUSSED OR IDENTIFIED IN THE COMPANY’S PUBLIC FILINGS WITH THE AMF,
INCLUDING THOSE LISTED UNDER SECTION 4“MAIN RISKS AND UNCERTAINTIES” OF THE COMPANY’S 2018 REGISTRATION DOCUMENT REGISTERED WITH
THE FRENCH AUTORITÉ DES MARCHÉS FINANCIERS (AMF) ON FEBRUARY 27, 2019 UNDER NO. D.19-0078, WHICH IS AVAILABLE ON GENFIT’S WEBSITE
(WWW.GENFIT.COM) AND ON THE WEBSITE OF THE AMF (WWW.AMF-FRANCE.ORG) OTHER THAN AS REQUIRED BY APPLICABLE LAW, THE COMPANY DOES
NOT UNDERTAKE ANY OBLIGATION TO UPDATE OR REVISE ANY FORWARD-LOOKING INFORMATION OR STATEMENTS.
•WE HAVE FILED A REGISTRATION STATEMENT WITH THE SECURITIES AND EXCHANGE COMMISSION ("SEC") IN CONNECTION WITH THE OFFERING TO
WHICH THIS PRESENTATION RELATES. BEFORE YOU INVEST, YOU SHOULD READ THE REGISTRATION STATEMENT, THE PRELIMINARY PROSPECTUS INCLUDED
WITHIN THE REGISTRATION STATEMENT, AND OTHER DOCUMENTS WE HAVE FILED WITH THE SEC FOR MORE COMPLETE INFORMATION ABOUT US AND
THIS OFFERING. YOU CAN OBTAIN THESE DOCUMENTS FOR FREE BY VISITING EDGAR ON THE SEC WEBSITE AT WWW.SEC.GOV. ALTERNATIVELY, COPIES OF
THE PRELIMINARY PROSPECTUS MAY BE OBTAINED BY CONTACTING SVB LEERINK LLC, ATTENTION: SYNDICATE DEPARTMENT, ONE FEDERAL STREET, 37TH
FLOOR, BOSTON, MA 02110, OR BY TELEPHONE AT (800) 808-7525, EXT. 6132, OR BY EMAIL AT SYNDICATE@SVBLEERINK.COM; OR FROM BARCLAYS
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BY TELEPHONE AT (888) 603-5847, OR BY EMAIL AT BARCLAYSPROSPECTUS@BROADRIDGE.COM.
2
I.
CORPORATE
HIGHLIGHTS
3
Company Profile
BACKGROUND
• Public company focused on metabolic diseases and associated complications,
including liver related disorders
• World-leading expert in nuclear receptor based drug discovery
• Lead drug candidate discovered in-house
• Founded in 1999 (Lille, Paris, France – Cambridge, United States) – 150+ employees
• Since 2006, Euronext Paris – compartment B (GNFT) – Market capitalization of ~€700M
• €207 million cash balance (End of Year 2018)
MAIN PROGRAMS
• Elafibranor: First-in-class molecule
• NASH – Phase 3 enrolled (Subpart H), accelerated approval process (Subpart H with
FDA; Conditional approval with EMA) and fast-track designation
• PBC – 12-week Phase 2 trial completed with positive top line results
• In-Vitro Diagnostic (IVD) for non-invasive diagnosis of NASH
• Strong IP protection with full worldwide rights
4
Experienced Management Team
and Highly Respected Advisory Board
• Jean-François Mouney, Chief Executive Officer, co-founded the company in 1999
• The executive team and board of directors have a deep experience at leading
biotech companies, large pharmaceutical companies and academic institutions
• Pr. Bart Staels, chair of the scientific advisory board and co-founder of the company,
is a world-renowned expert in nuclear receptors
• The scientific advisory board is comprised of internationally recognized key opinion
leaders in the field of metabolic and inflammatory diseases, with a particular focus
on the liver and gastroenterology
• The expertise, leadership and strength of the company’s relationships within the
academic and clinical communities are critical to its ability to execute on its mission
as it progresses its development pipeline
5
A Comprehensive Strategy
in Liver and Metabolic Healthcare
1. TREATMENT 2. DIAGNOSIS
Identifying
Providing PATIENTS ELIGIBLE FOR TREATMENTS
THERAPEUTIC SOLUTIONS
4. LAUNCH
EXCELLENCE
Preparing for
COMMERCIALIZATION
3. AWARENESS
Optimizing
STANDARD of CARE
6Robust Pipeline Focused on Liver and Metabolic Diseases
With Near-Term Clinical Milestones
• Currently finalizing analytical and clinical study designs • License agreement with LabCorp – January 2019
• 2018: alignment with FDA on path forward to validate NIS4 • LDT anticipated release in 2019
• Discovery of two key miRNA biomarkers in 2015 • Regulatory submission for IVD in 2020
7Upcoming Expected Catalysts
PBC (elafibranor) NASH (elafibranor)
PHASE 2 – DATA READOUT PHASE 3 – INTERIM DATA READOUT
for ACCELERATED MARKET APPROVAL
2018 2019 2020
PBC (elafibranor)
PHASE 3 – LAUNCH
NASH Pediatric (elafibranor)
PHASE 2 – 1st PATIENT
NASH Diagnostic (biomarkers) NASH Diagnostic (biomarkers)
PARTNERSHIP NIS4 – START COMMERCIALIZATION (LDT)
NASH Fibrosis (nitazoxanide)
PHASE 2 – POC START
8II.
LEADERSHIP IN
NASH AND PBC
9A Potential to Become
a Leader in NASH and PBC
1. TREATMENT
NASH & FIBROSIS
Elafibranor
Combinations
Nitazoxanide
PBC
Elafibranor
10NASH, a Disease Leading to Cirrhosis and HCC,
Represents a Large and Untapped Market
› Leading cause of liver disease in developed countries; ~20 million in the United States suffer from NASH and advanced fibrosis
› Cardiovascular events are the leading cause of death in NASH
› Multifaceted disease
› Market estimations: up to $20bn by 2025
Matteoni, Gastro 1999 – Adams, Gastro 2005 – Ekstedt, Hepatol 2006 – Ong, J Hepatol 2008 – Dunn, AJG 2008 – Sorderberg, Hepatol 2010 – Targher, NEJM 2010 – Williams, Gastro 2011
Chalasani, Gastro 2012 – Torres, Clin Gastro Hepatol 2012 – Wree, Nat. Rev Gastroenterol Hepatol 2013 – Rinella, JAMA 2015 – Bazick, Diabetes Care 2015
11Elafibranor, First-in-class,
Elafibranor, First-in-class, has
has Pluripotent
Pluripotent Activities:
Activities
PPARα/δ Regulate
PPARα and Multiple
δ Regulate Pathways
Multiple Pathways Essential
Essential inin NASH
NASH
12Elafibranor Is One of the Most Advanced NASH Product Candidates
2018 2019 2020 2021
PHASE 3 interim Potential NDA
FIRST wave
candidates
DATA READOUT Submission
• Elafibranor (Genfit)
• Ocaliva (Intercept)
• Selonsertib (Gilead)
• Cenicriviroc (Allergan)
PHASE 2
SECOND wave
candidates
DATA READOUT
• MGL-3196 (Madrigal)
• Aramchol (Galmed)
• NGM-282 (NGM)
• VK2809 (Viking)
13Evidence from Phase 2 NASH Trials
Elafibranor is the only product candidate from the first wave in NASH to have demonstrated all four of
(i) EFFICACY ON "NASH RESOLUTION WITHOUT WORSENING OF FIBROSIS" (ii) IMPROVEMENT
OF LIPID PROFILE (iii) IMPROVEMENT OF METABOLIC PROFILE (iv) TOLERABILITY
TOP LINE COMPARISON on EFFICACY for
"NASH RESOLUTION without worsening of fibrosis"
(approved/relevant endpoint for Phase 3 trials and market approval)
Elafibranor1 Ocaliva2 Selonsertib Cenicriviroc
1st wave (GENFIT) (INTERCEPT) (GILEAD) (ALLERGAN)
candidates 26% vs 5% 20% vs 6%
N/A N/A
(p-value 0.02) (p-value 0.03)
MGL-31962 Aramchol2 NGM-2822 VK28092
2nd wave (MADRIGAL) (GALMED) (NGM) (VIKING)
candidates 25% vs 6% 17% vs 5% 11% N/A
(p-value 0.03) (p-value >0.05) (no placebo)
(1) Ratziu et al, 2016 Gastroenterology (centers with randomization in all arms, to take into account the well known heterogeneity in the standard of care of NASH patients in different centers)
(2) Source: Corporate publications/presentations. Data not published in peer-reviewed scientific journals
14Elafibranor
Elafibranor in2b
Phase NASH
Results:
A Solid Ground for RESOLVE-IT
GOLDEN-505 Phase 3 trial
Phase 2 Results*
› Data published in peer-review journal
› Elafibranor resolved NASH without worsening of fibrosis, which has been recommended as
primary endpoint for Phase 3 pivotal trials in NASH1: Ballooning = 0; Inflammation = 0 (or 1);
No worsening of fibrosis (1 stage)
› Elafibranor showed improvement in the cardiometabolic risk profile of NASH patients
› Elafibranor showed favorable safety and tolerability results
(1) Draft guidance presented by the FDA on 12/3/18
15Elafibranor
Elafibranor Phasein2b
NASH
Results:
Additional Key Benefits
GOLDEN-505 Phasefor NASH Patients
2 Results*
Beneficial effect on Favorable
Beneficial effect on
- Glucose Homeostasis - Safety profile
Lipid Markers
- Insulin Sensitivity - Tolerability profile
in NASH patients
in T2D NASH Patients
ON TOP OF STANDARD of CARE ON TOP OF STANDARD of CARE
Crucial for a
chronic and silent
LDL-c ("bad” cholesterol) disease
HbA1c (glucose homeostatis)
TG (triglycerides) HOMA-IR (insulino resistance)
HDL-c (“good” cholesterol)
SAFETY
clinical outcomes
“Even using a low assumption for NAFLD prevalence
“It is imperative that any drug developed for
in T2D patients, it is estimated that 84MM people in
TOLERABILTY
NASH be at least neutral from a cardiovascular
risk perspective and ideally also reduce
the U.S. live with prediabetes or T2D and NAFLD. compliance
Moreover, the coexistence of NAFLD and T2DM
cardiovascular risks”
results in a worse metabolic profile and a higher efficacy in real world
(Hepatology 2015)
cardiovascular risk.“ (Bril, Cusi, Diabetes Care 2017)
16Elafibranor Phase 3 Design:
Details and Timing
> 250 centers ~ 1000 ~ 2000
(worldwide) patients patients
ACCELERATED MARKET AUTHORIZATION
• SUBPART H (FDA)
• CONDITIONAL APPROVAL (EMA)
FIRST TREATMENT PERIOD 18 MONTHS EXTENSION PERIOD
Placebo Placebo
Elafibranor 120mg Elafibranor 120mg
2:1 2:1
TRIAL INITIATION Q1 2016 72-WEEK INTERIM ANALYSIS END OF STUDY
Study population: patients at Histological primary endpoint Prevention of NASH associated
risk of progression to clinical NASH RESOLUTION WITHOUT clinical events, including cirrhosis
events WORSENING OF FIBROSIS
› NASH with a NAS ≥4 (central reading for all biopsies): cancer, all cause mortality
› Fibrosis stage F2 and F3 › Ballooning = 0
› (F1 + cardiometabolic risk) › Inflammation = 0 (or 1)
› Without worsening fibrosis (1 stage) Read-out ~2000 patients:
based on occurrence of a pre-defined
Histological key secondary endpoint number of events
End of enrollment first ~1000 improvement of histological fibrosis
patients for Subpart H: April 2018 (to be considered as an additional
labeling claim)
DSMB 18-month
DSMB 24-month
DSMB 30-month
Read-out first ~1000 patients: End of 2019
17Clinical Requirements for Future Combinations:
Elafibranor Shows Potential as Backbone Therapy
ANTI-NASH PURE ANTI-FIBROTIC
drug candidates drug candidates
1 Addressing NASH
(the underlying cause)
2
Addressing FIBROSIS
(the consequence)
Among product candidates in Phase 3, only ELAFIBRANOR and
OCALIVA have the potential to address both NASH and fibrosis
3 ELAFIBRANOR has demonstrated
Ensuring a clean
a favorable safety and tolerability profile
SAFETY/TOLERABILITY
in Phase 1 and Phase 2 clinical trials
18Proactive Evaluation of Potential "Add-on"
Drug Candidates for Elafibranor in NASH
BACKBONE Add-On
ELAFIBRANOR DRUG X
ELAFIBRANOR + NTZ
ELAFIBRANOR + FXR
ELAFIBRANOR + ACC
ELAFIBRANOR + ANTI-T2D
19Nitazoxanide (NTZ):
GENFIT’s Pure Anti-Fibrotic Drug Candidate
Nitazoxanide (NTZ)
› A new anti-fibrotic drug candidate, part of GNFT’s discovery program
› Currently approved as an anti-parasitic
› Early pre-clinical studies have shown promising anti-fibrotic activity 3
› A wholly owned IP position in NASH-related fibrosis
› Currently being evaluated in a Phase 2a investigator-led study
EASL 2017
CSAA CDAAc NTZ 30mpk NTZ 100mpk
Preventive protocol in the CDAA/c model (12 wks)
20Nitazoxanide (NTZ):
Investigator-initiated Phase 2a
OBJECTIVE
Evaluate the safety and efficacy of NTZ
NASH patients with fibrosis Stage 2 (significant fibrosis) or 3 (severe fibrosis)
Non-invasive readout related to fibrosis to assess the changes from baseline to the end
of treatment:
de novo collagen synthesis through Fractional Synthesis Rate of circulating plasma proteins
circulating markers of fibrosis
fibrosis scores based on circulating markers
imaging techniques
21PBC (Primary Biliary Cholangitis): Elafibranor Well Positioned to
Address Unmet Needs in this Severe Chronic Liver Condition
HIGH UNMET NEEDS RATIONALE for ELAFIBRANOR
• Cholestatic chronic autoimmune disease
• Affecting intrahepatic bile ducts
• Severe liver disease
• Prevalence in the general population: 0.05%
• Patient profile: women 40-60 years old
Elafibranor consistently showed positive effects
on ALP in all studied populations
+
Clinical evidence of beneficial effects induced by
PPARα and PPARδ in PBC populations, supported
by a pluripotent mechanism of action
+
Significant proportion of non/partial responders Recent evidence showing the potential of PPARα
with current treatments in PBC patient population to alleviate pruritus, a major symptom of PBC
Major symptom in PBC is pruritus and is not
addressed by current PBC therapies
22Phase 2a Study with Elafibranor
in PBC (Primary Biliary Cholangitis)
1st Patient enrolled May 2017 12-WEEK ANALYSIS
TREATMENT PERIOD 12 WEEKS
UDCA + Placebo
UDCA + Elafibranor 80mg
2:1
UDCA + Elafibranor 120mg
Study population Primary endpoint
adult patients with PBC effect of daily oral
and inadequate response to administration of elafibranor on
ursodeoxycholic acid serum alkaline phosphatase
(UDCA) (ALP) from baseline
End of enrollment 45 patients: Primary Endpoint achieved:
July 2018 December 2018
Secondary endpoints include:
ALP < 1.67 × upper limit of normal (ULN)
and total bilirubin within normal limit and > 15% decrease in ALP
Paris, Toronto, UK PBC scores
Pruritus and QoL (Quality of Life)
Safety of elafibranor in a PBC population
23Elafibranor
Elafibranor in NASH
Phase 2 Results:
Positive Data ReadoutPhase
GOLDEN-505 in PBC – Key Takeaways
2 Results*
› Elafibranor successfully meets PRIMARY ENDPOINT
(“change at week 12 in serum alkaline phosphatase (ALP) from baseline”) with:
› High statistical significance (pElafibranor
Elafibranor in NASH
Phase 2 Results:
AGOLDEN-505
Highly Competitive Profile
Phase 2 Results*
TOP LINE COMPARISON
EFFICACY in PHASE 2 (12-week data)
Elafibranor1 Ocaliva2 Seladelpar3
(GENFIT) (INTERCEPT) (CYMABAY)
80mg 120mg pbo 10mg pbo 5mg 10mg pbo
ALP
-48% -41% +3% -24% +3% -33% -45% N/A
(% change vs baseline)
% responders
ALPElafibranor
Elafibranor in NASH
Phase 2 Results:
Positive Data Readout
GOLDEN-505in PBC – Additional
Phase 2 Results* Key Benefits
Improvements in: Beneficial effect: Favorable:
Markers of PBC › Early indication of › Safety profile
› Gamma-glutamyl improvement in › Tolerability profile
transferases (GGT) pruritus to be
confirmed in a
Metabolic Markers longer study
› Total cholesterol, low-
density lipoprotein-C,
and triglycerides.
Clear clinical evidence to further advance Elafibranor in PBC
26A Pioneering & Proactive Approach
to Unlock the NASH Market
2. DIAGNOSIS
Towards a large scale industrial solution
27NASH Diagnosis:
A Need for Simple Blood-based Solutions
Current bottleneck
BIOPSY Imperfect "Gold Standard"
IMAGING TECHNIQUES Non-invasive, but limited
Ideal situation
“…there is an urgent unmet need to develop biomarkers that facilitate the
diagnosis, identification of populations at risk, assessment of disease progression
or regression, and/or response to treatment.”
Page 1401
BLOOD TEST Potential for large scale adoption in the clinic
28GENFIT’s Approach Designed to Ensure the NASH Market
Can Reach its Full Potential
Focus on a specific and relevant clinical question:
HEALTHY NAFLD CIRRHOSIS
NO NASH NASH
N/A TO BE TREATED
Steatosis ≥1 NAS ≥ 4
Ballooning ≥1 F2 or higher
Inflammation ≥1
LDT anticipated release in 2019
Regulatory submission for approval anticipated in 2020 (US, EU)
29NIS4 Commercialization:
Planned Strategy and Objectives
2019 2020 2021
PARTNERSHIP
U.S. Commercial
Licensing Agreement
• LDT for Clinical Research E.U. Commercial
• Expansive Research Licensing Agreement
REGULATORY
Prepare IVD for
FDA Submission
• IVD Approval
30The Future Patient Journey with IVD Test,
for Better Clinical Management of NASH Patients
31A Pioneering & Proactive Approach
to Unlock the NASH Market
3. AWARENESS
32The NASH Education ProgramTM
For BETTER CARE
NASH PHYSICIANS PATIENTS
(Non-Alcoholic Incl. DIABETOLOGISTS, Individuals at risk,
ENDOCRONOLOGISTS, Families
SteatoHepatitis)
CARDIOLOGISTS, GPs
1
Serious liver
condition Sub-Optimal
2 PATIENT CARE
Unprecedented
rise in prevalence
3
Silent, by nature
Yet still little
Diagnostic = bottleneck HIGH UNMET NEEDS
known, because…
No approved treatment yet
The NASH Education ProgramTM
www.the-nash-education-program.com 33A Worldwide Success for the Inaugural Edition of
International NASH Day, in 25+ Countries
ROADMAP 2019
A large coalition of 20+ stakeholders,
across the United States, Europe,
and LATAM, including:
• Patient associations
• Learned societies New and larger
• Companies leadership
Focus on education
34A Relevant Approach to Market Access
to Prepare for Commercialization
1. TREATMENT 2. DIAGNOSIS
Ideal drug profile Market Enabler
1st line treatment monotherapy (patient identification)
Cornerstone combination therapies
4. LAUNCH
EXCELLENCE
3. AWARENESS
Market Enabler
(physicians’ knowledge)
35A Strategy to Maximize Sales Uptake
and Transform the Company
Elafibranor: uniquely positioned in the first wave of NASH products
Objective: transform GENFIT into a biopharma, with a mixed revenue stream from:
Direct sales of elafibranor
Royalties from potential licensing deal
World class launch plan
Currently recruiting a team of experienced pharma leaders in the field of marketing
and market access
People who have joined the team have worked on several global launches and have
years of combined experience in big pharma in Europe and the US
Commercialization strategy
Open to explore potential alliance with large pharma company, preferably with a
solid footprint in metabolic diseases
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