MolMed Leading the way in Cell & Gene therapy Company Overview, FY18 Results update
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MolMed
Leading the way in Cell & Gene therapy
Company Overview, FY18 Results update
Company Overview - FY18 Results update
Disclaimer
The presentation contains certain forward-looking statements. Although the Company believes its expectations are based on
reasonable assumptions, these forward-looking statements are subject to numerous risks and uncertainties, including scientific,
business, economic and financial factors, which could cause actual results to differ materially from those anticipated in the
forward-looking statements.
The Company assumes no responsibility to update forward-looking statements or adapt them to future events or developments.
This presentation is not an offer of securities for sale in any country or jurisdiction, including the United States. Securities may
not be sold to the public in the United States, in Australia, in Canada, in Japan, or in other relevant jurisdictions without
complying with local registration requirements and other legal restrictions.
Company Overview - FY18 Results update 2
Agenda
Company overview
R&D proprietary pipeline
GMP Services (CDMO)
Financials and Shareholders ID
Upcoming milestones
Company Overview - FY18 Results update 3
MolMed overview Company Overview - FY18 Results update 7
A recognized leader in Cell & Gene research, development and manufacturing
Biotechnology company focused on research, development, manufacturing
and clinical validation of innovative anticancer and rare diseases therapies,
listed on the main market (MTA) of the Milan Stock Exchange since 2008
(MLMD.MI)
Pioneering research & development approach in viral vectors and cells
engineering
~ 200 scientists and support staff
Growing and diversified proprietary pipeline with autologous and allogeneic
CAR therapies
GMP manufacturing authorization for Cell & Gene Therapies for its
proprietary products as well as for third parties and/or in partnership
2 Authorized GMP manufacturing facility (~5,000sqm) for GMP
manufacturing, quality control activities for the production of clinical and
commercial products.
Company Overview - FY18 Results update 5
An established dual business model leveraging common technological assets
Proprietary Pipeline GMP Solutions (CDMO)
Ability to manage from pure research to clinical, Growing source of revenues to fund internal R&D
manufacturing, regulatory authorization, market
access and pricing & reimbursement Cutting edge technological asset to grant robust
development and manufacturing of internal products
Growing and diversified clinical and pre-clinical stage
onco-hematology cell & gene products pipeline
CAGR 2011-18
36%
Company Overview - FY18 Results update 6
Relevant achievements
9 proprietary patent families including 244 granted patents
Strimvelis, July 2016: EMA Market Authorization, MolMed is the exclusive
manufacturer for vector and medicinal product
Zalmoxis®, Aug 2016: EMA Conditional Market Authorization for 28
countries; market access in Italy and Germany (cost per patient ~ €300k)
CAR T CD44v6, Aug 2018: IMPD submitted for 1st in-man clinical trial
Enhanced CAR early stage pipeline both autologous and allogeneic (NK cells)
GMP development & manufacturing services: high profile network of
partners and solid revenue grow (+39% 2011-17 CAGR).
Company Overview - FY18 Results update 7
Management team
Carlo Incerti, MD - Chairman
MolMed’ Chairman
Head of Global Medical and Chief Medical Officer Affairs at Sanofi Genzyme (1991-2018)
Member of the Board of EuropaBio, the European Association for Bioindustries
Member of the Governing Board of IMI (Innovative Medicine Initiative)
Riccardo Palmisano, MD - CEO
Since 2015 CEO at MolMed S.p.A.
Since 2016 President of Assobiotec (Italian biotech industries Trade Association)
Vice President, Managing Director and GM at Genzyme Italy (2005 -15)
VP Commercial Retail Market at GSK Italy (2003 - 05)
Managing Director and GM at Shire Italy (2000 – 03)
Salvatore Calabrese - CFO
Since Sept 2018 CFO at MolMed
General Manager at Jazz Pharma Italy (2014-18)
COO and at Gentium (NASD) (2005-14)
Cell Therapeutics Europe (2003-05)
Manager at PWC (2000 – 2003)
Luca Alberici, PhD, MBA - CBO
Since 2015 CBO at MolMed
Bain & Co (2013-15)
Research Associate at Sanford B. P. Medical Discovery Institute (La Jolla, CA) (2011-12)
Company Overview - FY18 Results update 8
Scientific Advisory Board
Claudio Bordignon; MolMed Founder and SAB Chairman
Chairman of MolMed SAB
Member of the Scientific Council of the European Research Council
Professor of hematology at the University San Raffaele in Milan
Mohamad Mohty
Professor of Hematology and Head of the Hematology and cellular therapy
Department at the Saint-Antoine Hospital and University Pierre & Marie Curie, Paris
Malcolm K. Brenner
Director of the Center for Cell and Gene Therapy and
Professor of Medicine and of Pediatrics at Baylor College of Medicine, Houston, TX
Miguel-Angel Perales
Deputy Chief, Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center, NY, USA
Gianpietro Dotti
Member of the UNC Lineberger Comprehensive Cancer Center and Professor of the Microbiology and Immunology
Director of the UNC Immunotherapy Program at the Univ. of North Carolina, NC
Company Overview - FY18 Results update 9
MolMed
Proprietary pipeline
Company Overview - FY18 Results update 13MolMed Onco-hematology proprietary pipeline
Product portfolio includes proprietary anti-tumor cell & gene therapies in clinical and preclinical development:
Company Overview - FY18 Results update 14MolMed
Proprietary pipeline
Zalmoxis®
Company Overview - FY18 Results update 15Zalmoxis® Zalmoxis®: a first in class orphan drug with a specific mechanism of action to address the limits of partially compatible stem cell transplantation Zalmoxis® (TK) is an ex vivo cell therapy based on donor T cells genetically engineered to enable bone marrow transplants from partially compatible donors, inducing a rapid immune reconstitution Company Overview - FY18 Results update 13
Zalmoxis® Zalmoxis® efficacy: 100% of acute GvHD resolution Company Overview - FY18 Results update 14
Zalmoxis®
Zalmoxis®: clinical outcome as additional life saving therapy
New pair-matched analysis Non-relapse Overall
Chronic
1-year outcomes mortality survival
GvHD
Alive and relapse free at 21 days (NRM) (OS)
Controls (n=139) 46% 34% 23%
Zalmoxis (n=36) 20% 51% 6%
p-value^ 0.003 0.007 0.02
Contemporaneous haploidentical transplants (period 2000-2013), including 36 Zalmoxis and 139 controls (70 T-cell replete and 69 T-cell depleted) were
matched (1 to 4 ratio). 28 controls without information on cGvHD. *RI and NRM are competing risk events (when one competing event occurs, patients
are no longer at risk for the other event, with those with shorter survival being less likely to develop relapse) and NRM events occur earlier than relapse
events. ^Cox test stratified on match group (LFS and OS) and Gray test (RI, NRM and chronic GvHD)
Company Overview - FY18 Results update 15Zalmoxis®
European target population: haploidentical transplants
1158 (’16) 1 Haplo in Acute
Current CMA
indications AML and ALL are the target
(+36% -’14) leukemia indications also of TK008 PhIII
Study
MDD, MPN, CLL, Plasma cell
823 (’16) 2 Haplo in Other disorders (MM, others), Hodgkin
(+82% -’14) Hemat. Mal.
and non-Hodgkin lymphoma
MUD 9/10 clinically performs as
4000 (‘16) 3 MUD 9/10 High unmet
Upsides
mismatched thus haplo could be
preferred medical need
DLIs could significantly benefit opportunity
7900 (‘16) 4 DLI from TK suicide strategy
and
Most autologous and allogeneic CAR-T therapies may benefit from TK suicide gene machinery
Source: Company and EBMT (Passweg J et al ,Bone Marrow Transpl 2018)
Company Overview - FY18 Results update 16Zalmoxis®
Zalmoxis® track
~ 300K Eu / patient
European Commission CMA Next Steps
(Conditional Marketing Authorization)
Robust Core Value Dossier
Clear pricing policy and corridor AIFA P&R* dossier
Reimbursement Submission ONGOING ONGOING
price
Price/ Marketing Commercial partner
Exclusive commercial agreement Authorization Scouting
• TTY Biopharm (Asian countries) for
EU and US
Upfront + Milestones: 13.5M€
Pricing Price
• TTY Biopharm (Israel) MolMed - Dompé negotiation Confirmed
Commercialization & last round 130k € /infusion
Supply Agreement
for Europe
Closed in 4Q18
Aug Apr and Jun Jul Dec Jan Dec Jan
2016 2017 2017 2017 2018 2018 2019
* P&R: Pricing &
Reimbursement
Company Overview - FY18 Results update 17MolMed
Proprietary pipeline
CAR platform
Company Overview - FY18 Results update 21CAR-CD44v6
CD44v6 CAR T cells: an original late preclinical stage therapy, targeting
both hematological and solid tumors
CAR-T family: lymphocytes armed with chimeric receptors that have demonstrated high anti-tumor potential,
also against tumors, above all hematological, particularly aggressive and resistant to traditional therapies
CAR T CD44v6 features
Variant v6 of antigen CD44 is over-expressed in MM and AML
High safety profile (low skin toxicity and suicide gene)
High therapeutic potential also in hematological and solid tumors:
it specifically recognizes variant 6 (v6) of the antigen CD44 (CD44v6)
The LNGFR spacer allows selection and in vivo tracking of CD44v6 CAR T cells
Generation of CD44v6 antigen-loss variants is circumvented by the reduced growth of CD44v6 negative
tumor cells
Company Overview - FY18 Results update 19CAR-CD44v6
CD44v6 in vivo activity in hematological tumors: high tumor burden
hematological
model of AML-M5 (THP-1) tumours
Day 1 Day 15 Day 16 Day 40
AML T cells T cells Sacrifice
CAR-CD44v6 cures aggressive
leukemia in a mouse model: liver
Liver weight (gr)
CD44v6
Ctrl CAR CD44v6∆N
MolMed published results
Company Overview - FY18 Results update 20CAR-CD44v6
CD44v6 CAR-T cells do not significantly infiltrate the skin: low hematological
expected toxicity tumours
Use in combination with
MolMed’ proprietary
patent TK suicide gene
Company Overview - FY18 Results update 21CAR-CD44v6
CARTCD44v6 clinical trial within the EU-funded EURE-CART project hematological
tumours
Multi-center, first-in-man Phase I/IIa clinical trial to demonstrate the safety and the efficacy
of CAR-CD44v6 T-cell immunotherapy in:
• Acute Myeloid Leukemia (AML)
• Multiple Myeloma (MM)
Phase I - Dose escalation Phase II - Dose expansion
Objectives: Maximum Tolerated Objectives: Confirmation of Clinical
Dose and Clinical Activity Activity and Safety Profile
18 Pts (3 dose levels) up to 30 Pts 14 Pts (1 Dose level selected in Ph. I)
(BOIN Adaptive design) per indication (Simon design)
QI19 QI-Q2 2020
Company Overview - FY18 Results update 22CAR-CD44v6
The EURE-CART project hematological
tumours
MolMed leads a Team of clinical experts in oncology and pioneers in the field of Cell & Gene therapy:
IRCCS Ospedale San Raffaele (Italy)
Universitätsklinikum Würzburg (Germany) University Hospital Ostrawa (Czech Republic)
Ospedale Pediatrico Bambino Gesù (Italy) Istituto Superiore della Sanità (Italy)
L'Hospital de la Santa Creu i Sant Pau (Spain) Acromion GMBH (Germany)
ARTTIC SAS (France)
«MolMed Spa is uniquely endowed in the EU with the knowhow and experience necessary to meet
this ambitious objective, as demonstrated by its unparalleled track record»
«To be successful, EURE-CART proposes the early involvement of National regulatory authorities for
accelerating the approval of CAR T-cell immunotherapy, as well as the centralisation of its
production by the MolMed Spa» Horizon 2020 EURECART Project funding commission.
IMPD submitted to authorities in October ‘18 (expected outcome January ’19)
Company Overview - FY18 Results update 23CAR-CD44v6
CD44v6: expressed by several solid cancers Solid tumours
Most of the clinical studies conducted to date have used CAR specific for CD19 antigen,
limiting its use in patients with hematologic B cell malignancies (hematological tumor)
Variant v6 of antigen CD44 is over-expressed also in several solid epithelial tumors:
• Squamous Cell Carcinomas mainly from head & neck, esophagus, skin, and lung, ovary
• Adenocarcinomas mainly from breast, lung, pancreas and colon
• Sarcomas
Antitumor activity of CD44v6CAR T cells has successfully been demonstrated in preclinical models of human
lung and ovary carcinomas
STRONG RATIONALE TO DESIGN AND IMPLEMENT A BASKET TRIAL BY 3Q 2019
Company Overview - FY18 Results update 24CAR-CD44v6
CD44v6 in vivo activity in solid tumors: a human lung adenocarcinoma Solid tumours
model
CAR T CD44v& highly
infiltrates the tumor
Company Overview - FY18 Results update 25CAR-CD44v6 CD44v6 in vivo activity in solid tumors: effect against human lung Solid tumours adenocarcinoma Company Overview - FY18 Results update 26
CAR platform
MolMed criteria to select new targets for CAR therapies
Target unmet clinical needs
Target both hematological and solid tumors
Chose targets with safe and/or moderate risk expression profile
No follower approach on CD19 or other targets in advanced clinical development
by large companies
Evaluate IP freedom to operate
Target selection endorsed by Scientific Advisory Board
Company Overview - FY18 Results update 27CAR platform
MolMed approach to develop new-generation CAR therapies
proprietary pipeline
MolMed is one of the few biopharma worldwide having a diversified pipeline in both autologous and allogeneic CARs
Autologous CAR-T Platform Allogeneic CAR-NK Platform
CAR-NKs
CAR-Ts
PATIENT Healthy DONOR
PATIENTS
No GvHD risk NK cells exclude GvHD
Proven clinical efficacy Lower COGS/patient (significant benefits from both a technical
High production cost (1 batch = 1 patient) and logistic point of view)
Wider market potential (1 batch = multiple patients)
June 28th 2018: 3ys Master Agreement with AbCheck May 31st 2018: binding term sheet with Glycostem for the
for the development of new CARs targeting novel development and
tumor antigens manufacturing of allogeneic CAR-NK therapies
Company Overview - FY18 Results update 28CAR-NK platform
MolMed approach to select a new allogeneic CAR platform
CAR-NK cells are one of the most innovative pre-clinical investigations in cellular immunotherapy with much
less competition compared to autologous CAR-T
NK cells are cells of the innate immune system, capable to mediate anti-cancer effects without the risk of
inducing graft-versus-host disease (GvHD)
NK cells are well suited as off-the-shelf therapy capable, starting from a single batch produced by a healthy
donor, to treat a large number of patients with cancer
XX
MolMed has defined 3 specific targets products, using 3 different tumor antigen XY
receptors, in order to enlarge possible cancer indications, targeting both CAR-
NK CAR-
hematological and solid tumors and place itself in a leadership position inside YZ NK
the CAR-NK field
CAR-
NK
Company Overview - FY18 Results update 29The CAR NK platforms currently in development in the field have CAR-NK platform
important limitations
NK cells obtained from PBMCs display low transduction efficiency and reduced growth rate
The number of NK cells in UCB is very low
iPS derived NKs may have safety limitations and heterogenic phenotype
NK-92 cell line are immortalized and difficult to manipulate causing safety issue for the patient
MolMed is working on a new CAR-NK platform that overcomes these limitations
Company Overview - FY18 Results update 30CAR-NK platform
MolMed - Glycostem partnership for CAR-NK allogeneic platform
Partnership* between two leader companies in their own field gives strong potential to develop a new and original cancer therapy
Leader in Cell & Gene therapy:
high expertise in lentiviral vectors
development and cell transduction
Umbilical cord HSC Purification Transduction with Cells expansion
blood CAR
Leader in NK field:
proprietary novel differentiation
protocol in NK cells
Patient infusion NK cells differentiation and expansion Thowing
Company Overview - FY18 Results update 31GMP services (CDMO) Company Overview - FY18 Results update 35
CDMO
CDMO business experience in manufacturing of vector and
modified cells
Viral Vector GMP Manufacturing GMP Cell Engineering
Top level expertise from tech transfer to fill and finish
for clinical and commercial use
Up to 200L Vector
manufacturing We offer proprietary processes for gene modification
of HSC (CD34+) and T-cells
+ 150 GMP Vector
batch produced
Customization, development, qualification and + 250 Treated
validation activities carried out by top level Expertise patients
+ 20 Clinical Trials
Able to perform more than 100 analytical tests in-
house, resulting in containment of material, costs
and release timelines
Company Overview - FY18 Results update 33CDMO
Excellent GMP capacity
MolMed obtained the GMP manufacturing authorization for Cell & Gene Therapies for its proprietary
products as well as for third parties and/or in partnership
First Facility - Milan San Raffaele New Facility - Milan Bresso Open Zone
Bresso
1,500 SQM (16,000 SQF) and 9 grade B/C suites 3,300 SQM (36,000 SQF) and >20 Grade B/C suites
Authorized GMP manufacturing facility since 2003 Qualified Officina Farmaceutica, authorized for GMP
for clinical programs manufacturing and quality control activities for the
production of clinical and commercial products
Authorized GMP manufacturing facility since 2015
for the Commercial products Stream 2 opening in 2019/2020, for further services and
new collaborations
Company Overview - FY18 Results update 34CDMO GMP Development and Manufacturing partners Company Overview - FY18 Results update 35
GMP services continuous revenue growth
CAGR 2011-18
36%
Company Overview - FY18 Results update 36MolMed
Financials
Shareholders ID
Company Overview - FY18 Results update 40Financial results improved significantly in the last three years
Total FY18 Revenues of 29.9€ M, with Revenues from sales equal to Euro 28.5€ M, increased by 23.7% compared to 2017
Operating and Net Results considerably improved respectively by 52.6% and 51.5% compared to 2017
Human resources increased year by year, from 152 employees at the end of 2016 to 201 as of December 31st, 2018
Δ Δ
€/000 FY18 FY18 vs FY17 FY 2017 FY 2016 FY17 vs FY16
€ % € %
Operating Revenues 29,880 5,893 24.6% 23,987 22,825 1,162 5.1%
Revenues 28,447 5,447 23.7% 23,000 19,484 3,516 18.0%
Other operating revenues 1,433 456 46.2% 987 3,341 (2,354) (70.5%)
Operating costs (33,745) (1,610) 5% 32,135 36,411 (4,276) (11.7%)
Operating Results (3,865) 4,283 52.6% (8,148) (13,586) 5,438 40.0%
Net Result for the period (4,123) 4,374 51.5% (8,497) (13,876) 5,379 38.8%
Net Financial Position 16,466 18,111 19,702
Work Force (#) 201 15 186 181 5
Company Overview - FY18 Results update 38Shareholders’ structure
MolMed is listed on the main market (MTA) of the Milan Stock Exchange since 2008 (MLMD.MI)
Market cap ̴ 190M € (as of April 18th, 2019)
Outstanding shares: 463,450,672
Fininvest S.p.A. 23%
Airain Ltd. 5%
H-Equity S.r.l. 2%
H-Invest S.p.A. 2%
Market 68%
Company Overview - FY18 Results update 39MolMed
Strategic goals and upcoming
milestones
Company Overview - FY18 Results update 45MolMed’ strategic vision and dual business model: R&D and CDMO
REINVESTING
in innovative cell & gene DELIVERING
research products new clinical proof
of concepts
R&D CDMO
Upfront and royalty Stream of
streams from revenues from
licensing / GMP Services for
co-developing third parties
agreements
Company Overview - FY18 Results update 41Upcoming milestones
Contract Development and
Proprietary Product Pipeline Manufacturing
TK (Zalmoxis®): Milan new facility stream 2 authorization (2019/20)
2019: new partner for commercial development
Further expansion of clients base
CAR44v6 (hematological tumors)
1H 2019: 1st in man clinical trial Enlargement of current clients scope and services
2Q 2020: Clinical Activity and Safety Profile confirm
Continuous double digit growth
CAR44v6 (solid tumors)
2019: solid trial IMPD
2020: 1st in man clinical trial
New autologous and allogenic CAR pipeline:
2020: preclinical data
Company Overview - FY18 Results update 42Contacts
Ilaria Candotti
Investor Relations & Communication Manager
e-mail: investor.relations@molmed.com
MolMed S.p.A.
phone: +39 02 21277.205
Company Overview - FY18 Results update 43You can also read
