BASILEA.9.pptx

CLÉMENT DUBOS
JULIETTE PAILLARD
MARÍA ALCÁZAR DUQUE
SIHEM ALLOTI
SOSSANE NOURESTANI
BUSINESS
MODEL
ANALYSIS
PRESENTATION MAP
About the company
Governance
Pipeline
Financial statement
SWOT
About The Company
Features
• Headquarter: Basel, Switzerland.
• Innovative pharmaceutical products
Bacterial infections, fungal infections, oncology.
Resistance and nonresponse.
• Founded in 2000 as spin-off of Roche.
• Publicly traded since 2004 on the SIX Swiss Exchange.
About The Company
Basilea
Pharmaceuticals ltd.
Basilea Pharma SAS
Basilea Pharmaceuticals
iberia s.l.
Basilea
Pharmaceuticals A/S
Basilea Pharmaceutica
Deutschland gmbh
Basilea Pharmaceutica
China ltd.
Basilea
Pharmaceutica ltd.
Basilea
Pharmaceutica s.r.l.
About The Company
Research Center In China
-Wholly-owned subsidiary of Basilea
-Near Shangai, Jiangsu Province
People’s Republic of China
-Basilea’s key R&D projects
● Chemical synthesis
● Analytical development
● Process research and development
-Awards:
● High-tech Enterprise
● “A” class for safety operation
● Best Safety Performance
● Development of the local R&D
Governance
Board of Directors
Mr. Domenico Scala, Chairman
Dr. Thomas M.
Rinderknecht
Dr. Martin
Nicklasson
Prof. Daniel Lew
Mr. Steven D.
Skolsky
Dr. Thomas Werner
Governance
Management Committee
Ronald Scott, CEO
Dr. Günter
Ditzinger
Prof. Achim
Kaufhold
Dr. Laurenz
Kellenberger
Ms. Heldi
McDaid
Mr. Donato
Spota
Mr. David
Veitch
Pipeline - overview
Pipeline - overview
ANTIFUNGALS
Medical Need
ASPERGILLUS
New Antifungal Market :
USD 2.35 billion sales
MUCORALES
Mortality rates for invasive fungal infections
COMMERCIAL POTENTIAL
WW sales (mn standard units)
CANDIDA
Mechanism of action
Acetyl CoA
Weakening of
fungal cell
membrane
structure &
function
Prodrug activation
process
Plasma esterase
Squalene
Lanosterol
Lanosterol 14α-demethylase
Ergosterol
Fungi cell
• Completed three phase 3 clinical trials:
ACTIVE
SECURE
VITAL
Cell
membrane
CANDIDA
ASPERGILLUS
MUCORALES
DEVELOPMENT &
REGULATORY STATUS
Europe
•
Currently marketed by Basilea in Germany,
Italy, the United Kingdom, Austria and France,
 Collaboration with Quintiles
•
Orphan Drug designation
 10 years of market exclusivity
the USA
•
Marketed by Basilea’s license partner :
 Astellas Pharma US
•
2Q16 sales = USD 12 million
•
Orphan drug status + Qualified Infectious
Disease Product designation
 12 years of market exclusivity
MENA
Latin America
•
Distribution agreement with HIKMA
•
License agreement with GBT
ANTIBIOTICS
Medical Need
Over the last 30 years, no major new types of antibiotics have
been developed
•
Frequent Methicillinresistant
Staphylococcus aureus
(MRSA) is one of the
most frequent causes of
hospital-acquired
bacterial pneumonia
•
Between 10-30% of
patients with
Staphylococcus aureus
bacterieia will die from
the infection
MRSA
HAP
•
HAP is the second
most common
nosocomial infection
•
Among the highest
mortality rates (62 %)
•
Main causes of HAP:
MRSA +++ (> 20 %)
•
CAP up to 60 %
hospitalisations
COMMERCIAL POTENTIAL
Zevtera®/
Mabelio®
Mechanism of action
•
•
First broad-spectrum anti-MRSA
cephalosporin authorized for HAP (excl.
VAP) + CAP (Community Acquired
Pneumonia)
Single agent with broad-spectrum
bactericidal activity that includes MRSA and
Pseudomonas spp
Prodrug activation
Ceftobiprole
Modecaril (sodium)
Ceftobiprole
Enzymatic cleavage
(Plasmatique esterase)
Synthesis of
Peptidoglycane
Ceftobiprole
Bacterial Lysis
Diacetyl
DEVELOPMENT &
REGULATORY
STATUS
Europe
The USA
•
•
•
Currently marketed by Basilea in Germany,
Italy, the United Kingdom , France , Austria, and
Switzerland
→ Collaboration with Quintiles
Marketed in the Nordic Countries sept 2016
→ Collaboration with Unimedic Pharma
Market access activities ongoing in Spain
•
Not approved yet → Two cross-supportive
phase 3 trials (ABSSSI and Bacteriema)
•
Special Protocol Assessment’s agreement by
the FDA on going → phase III development
program H1 2017
Qualified Infectious Disease Product
Designation (ABSSSI and CABP)
→ 10 years of market exclusivity
•
Latin America
•
Licence agreement
with Biotoscana S.L
•
Fundings: Biomedical Advances Research and
Development Authority (BARDA) under
contract
Intellectual Property
EU MA 2014
MA 2015
Industrial property
Industrial property
PATENT
EU
ORPHAN
ORPHAN
1
Commercial exclusivity
Commercial exclusivity
USA
SPC
PATENT
QIDP
PIP
NCE
QIDP
ANTIBIOTICS
Medical Need
Infections caused by
multidrug resistant
Gram negative
bacteria with newly
acquired resistance
mechanisms  Chronic
bacterial pulmonary
infection: serious
concern in patients
with
• cystic fibrosis
• bronchiectasis
Cystic Fibrosis prevalence
70,000 and 100,000 people
worldwide
COMMERCIAL POTENTIAL
BAL30072
•
Novel siderophore (iron-binding) sulfactam
antibiotic potent bactericidal activity againts
GN pathogens.
•
Stable towards many of the extendedspectrum beta lactamase enzymes and
metalo-beta-lactamase.
Mechanism of action
Non Cinical data
Potent antibacterian in vitro- in vivo
activity against:
• Enterobacter spp.,
• Klebsiella spp,
• Acinetobacter spp.
• Pseudomonas aeruginosa
Synergy with
Meropenem
Clinical studies
•
•
Development of IV formulations with the
US BARDA
Inhaled formulation : ND4BB Program
15 august 2016
•
•
Completed PH1 study
Discontinuation of the inhaled
formulation
ONCOLOGY
Global cancer burden
12.7 mn new cases in 2008, 21.4 mn by 2030
•
2/3 of all cancer diagnoses occurring in
LMIC
Some Unmet Medical Needs
Growth sector
•
TNBC
•
NSCLC
•
Pancreatic cancer
•
Glioblastoma
Balisea’s product targets
markets such as the USD
3.5 bn taxane market
COMMERCIAL POTENTIAL
increasing need for new
agents in taxane-resistant
cancer patients.
Some Unmet Medical Needs
Growth sector
•
TNBC
•
NSCLC
•
Pancreatic cancer
•
Glioblastoma
Balisea’s product targets
markets such as the USD
3.5 bn taxane market
COMMERCIAL POTENTIAL
increasing need for new
agents in taxane-resistant
cancer patients.
Some Unmet Medical Needs
Growth sector
•
TNBC
•
NSCLC
•
Pancreatic cancer
•
Glioblastoma
Balisea’s product targets
markets such as the USD
3.5 bn taxane market
COMMERCIAL POTENTIAL
increasing need for new
agents in taxane-resistant
cancer patients.
BAL101553
Tumor checkpoint controller
Highly water-soluble lysine
prodrug
•
No other approved oncology agent
targets BAL27862’s binding site
•
Address the issue of resistance to
(MTA’s)
Highly water-soluble lysine
prodrug
•
Non-clinical data :
•
Good oral bioavailability
•
Passes through the blood-brainbarrier
•
Potent activity in many tumor cell
•
Activity in treatment-refractory
glioblastoma models. (AARC)
BAL101553
lines
Protease
•
BAL27862
Clinical trials :
•
IV : phase 2 : Potential 
colorectal, gastric, non-small cell
lung, ovarian, pancreatic and
triple- negative breast cancer
•
Oral : Phase 1/2a, patients with
advanced solid tumor cancers +
glioblastoma new arm by year-end
biomarker strategy
What’s next?
•
•
•
Optimize dose selection
Identify cancer patient groups
more likely to respond.
Could trigger a lucrative partnering
agreement with a major cancer
player :
 phase III development
 broader range of phase IIb
indications
BubR1
EB1
•
Forecast : Peak sales of CHF 500+ mn
ONCOLOGY
Medical Need
BRAF mutant in 50% of the melanoma
Estimated rates of deaths by melanoma worldwide (2012)
BAL3833
Commercial potential
Vemurafenib
ROCHE
Dabrafenib/Tram
etinib NOVARTIS
●
●
●
Launch
Profit (2015)
2011
US, CA,
EU
CHF 214
millions
2014
US, EU
CHF 453
millions
Vemurafenib: inhibition of mutant BRAF
Dafrabenib: inhibition of mutant BRAF
Trametinib: inhibition of MEK1 and MEK2
Research:
University of Manchester at The Institute of
Cancer Research
Fundings:
Wellcome trust
Cancer Research UK Cancer Institute
Mechanism of action
BAL3833: targets BRAF, CRAF and SRC.
Orally active drug targeting kinase inhibitors
Preclinical studies
-Results presented at the AACR
conference
-Mutant BRAF cancers: melanoma,
colorectal, ovarian
● Treatment of refractory
melanoma
● Inhibition of mutant BRAF
and cRAF/SRC
-Potential in K-RAS driven cancers :
pancreatic, colorectal, non-smallcell lung
● New therapeutic option
● Inhibition of panRAF/and
SRC kinase
Phase 1/2a study
•
•
Currently ongoing for patients
with advanced solid tumors
Biomarker evaluation initiated :
tumor type selection
Commercial Strategies
Funding partners & Academic institutions :
DISARM
iABC
BARDA
ADAPT
SPA
NCE
QIDP
BBND
Action Plan
against
antimicrobial
resistance
Commercial Strategies
Partnering and Licensing deals :
Financial Statement
(in CHF
thousands)
2012
2013
2014
2015
Revenue
58 336
41 376
42 634
52 825
R&D
(58 863)
(53 350)
(54 377)
(60 075)
100.9%
128.9%
127.5%
113%
Net profit
(53 033)
(33 019)
(41 520)
(61 520)
Equity
105 502
70 874
57 931
14 856
Cash
223 956
118 898
156 125
313 064
Earning per share
(5.53)
(3.40)
(4.17)
(6.09)
Burn rate
(in years)
2.0
1.6
1.8
2.7
Financial Statement
Financial Statement
Evolution of stock price in 2016 :
Financial Statement
TOCTINO’s timeline
License
agreement
with
Stiefel (GSK),
→ Contract with 200 CHF
million
and
subsequent
milestones for FDA approval
FDA denied the approval in 2016
GSK isn’t willing to
developing Toctino
Developped by Basilea in the
early 2000’s
continue
Impacted the stock price of
Basilea (-35%)
Basilea is considering buying
it back in order to find
another partner in the future
Bottom line :
Even though it didn’t get the FDA’s approval, it still
was a great help in Basilea’s development strategy
Financial Statement
Financial Statement
•
•
•
•
•
•
•
2 products on the
market
3 products in
development
Several therapeutic
areas with high
medical needs
Numerous partners
and license
agreement
Growth
employment
Communication
force
Cash increase
•
•
•
•
•
Commercialization •
dependent on
external partners •
No dedicated sell
force outside major •
EU countries
Loss of WW
Cresemba license
in 2027
•
Toctino’s
discontinuation
Discontinuation of
BAL30072
•
Positive regulatory
environment
Unmet medical
needs
Incentives &
encouraging
initiatives across the
globe
BAL101553 : solid
preclinical & early
clinical data → could
trigger major cancer
player
Implantation in China
•
•
•
•
•
Price and
reimbursement
policies
reinforcement
Antimicrobial
stewardship
Oncology R&D
(solid tumors) :
highest failure rate
for NME, higher
development costs
Upcoming drugs
M&A activity →
Basilea becoming
a potential target