Mondobiotech Biotech Showcase2011.pdf
Mondobiotech Biotech Showcase2011.pdf
Mondobiotech Biotech Showcase2011.pdf
You also want an ePaper? Increase the reach of your titles
YUMPU automatically turns print PDFs into web optimized ePapers that Google loves.
an open-source<br />
bioscience technology company<br />
delivering pipeline for rare diseases<br />
<strong>Biotech</strong> Showcase 2011 – San Francisco<br />
Paolo Bassanini, Investor & External Relations
Disclaimer<br />
The shares of mondoBIOTECH holding AG have been traded since August 26 th , 2009<br />
on the SIX Swiss Exchange with the symbol RARE<br />
This document expressly or implicitly contains<br />
certain forward-looking statements concerning<br />
mondoBIOTECH holding AG and its business.<br />
Such statements involve certain known and<br />
unknown risks, uncertainties and other factors,<br />
which could cause the actual results, financial<br />
condition, performance or achievements of<br />
mondoBIOTECH holding AG to be materially<br />
different from any future results, performance<br />
or achievements expressed or implied by such<br />
forward-looking statements. mondoBIOTECH<br />
holding AG is providing this communication as<br />
of this date and does not undertake to update<br />
any forward-looking statements contained<br />
herein as a result of new information, future<br />
events or otherwise.<br />
This document does not constitute or form<br />
part, or all, of any offer or invitation to sell<br />
or issue, neither in the United States of<br />
America nor elsewhere, or any solicitation of<br />
any offer to purchase or subscribe for, any<br />
securities, nor shall part, or all, of this<br />
document or its distribution form the basis<br />
of, or be relied on in connection with, any<br />
contract or investment decision in relation to<br />
any securities.<br />
Finding Therapies for Rare Diseases 2
Who is <strong>Mondobiotech</strong><br />
<strong>Mondobiotech</strong> is a Swiss independent open-source bioscience technology<br />
company delivering pipeline for rare diseases using a proprietary<br />
bio-mathematical methodology<br />
Search&Match is our proprietary open-source technology platform which<br />
enables searching and redirecting existing compounds (human peptides)<br />
into rare diseases<br />
mondoBIOTECH community is a powerful global group of interacting expert<br />
researchers, who share a scientific ecosystem organized by <strong>Mondobiotech</strong><br />
in the field of rare diseases<br />
Finding Therapies for Rare Diseases 3
Achievements to date<br />
2001 <strong>Mondobiotech</strong> is founded in a basement in<br />
Lugano, Switzerland<br />
2002 Licensing-out agreement with NASDAQ-listed<br />
Intermune, Inc. for Idiopathic Pulmonary<br />
Fibrosis (IPF)<br />
2004 Strategic partnership with SIX-listed Bachem<br />
for peptides as biomedicines<br />
2006 Licensing-out agreement with NASDAQ-listed<br />
Biogen Idec, Inc. for Pulmonary Arterial<br />
Hypertension(PAH)<br />
1 Patent<br />
granted<br />
EP 0 795 332 B1<br />
5 Awards with<br />
international<br />
recognition<br />
7 Licensing-out<br />
agreements<br />
7 Orphan drug<br />
designations<br />
in EU and US<br />
2007 Licensing-out agreement with NASDAQ-listed<br />
United Therapeutics, Inc. for Methicillin-<br />
Resistant Staphylococcus Aureus in Cystic<br />
Fibrosis (MRSA in CF)<br />
2008 <strong>Mondobiotech</strong> awarded Technology Pioneer<br />
2008 by the WEF (World Economic Forum)<br />
2009 Strategic partnership with 23andMe, Inc. for<br />
genotyping patients affected by rare diseases<br />
2010 Licensing–out agreement with NASDAQ-listed<br />
United Therapeutics, Inc. for Pulmonary<br />
Arterial Hypertension (PAH), Acute<br />
Respiratory Distress Syndrome (ARDS),<br />
Idiopathic Pulmonary Fibrosis (IPF) and<br />
Sarcoidosis<br />
101 Patent<br />
applications<br />
filed<br />
135 Medicinal<br />
product<br />
candidates<br />
312 Peptides to<br />
redirect in rare<br />
diseases<br />
Finding Therapies for Rare Diseases 4
Focus on rare diseases<br />
30’000 is the number of known diseases today of which:<br />
7’000 diseases are neglected or rare<br />
affecting 6% to 8% of the world population<br />
In USA 30 million affected<br />
In Europe 35 million affected<br />
In the World >500 million affected<br />
(3 rd largest population after China and India)<br />
ONLY 52 RARE DISEASES BENEFIT FROM A THERAPY<br />
Finding Therapies for Rare Diseases 5
<strong>Biotech</strong>: high risk of drug development<br />
•! A typical biotech company has between 1 and 3 products in clinical development<br />
at any given time<br />
•! To develop a product a company must endure long series of pre-clinical and<br />
clinical testing spending tens to hundreds of million and lasting on average 10<br />
years<br />
•! Moreover the is a real risk of compromizing the business due to the fact that<br />
only 10% of products entering clinical phases do reach market authorization<br />
•! Showed below are examples of stock values changes of listed biotech companies<br />
in 2010 just after the announcement of failure or success of clinical trial phases<br />
Stock plunge after phase-2 failure Stock plunge after phase-3 failure Stock raise after phase-3 success<br />
Finding Therapies for Rare Diseases 6
Our approach to drug discovery<br />
•! In today’s world, information is extremely fragmented and dispersed throughout millions of<br />
publications from the academy world<br />
•! Yet, thanks to technology, it is easily accessible and retrievable at very limited costs<br />
•! Thus, we are changing the way drugs are developed as a vast amount of validated and highly<br />
qualified information can be accessed<br />
•! As companies are cutting R&D and drug development costs, <strong>Mondobiotech</strong> is delivering a steady<br />
stream of medicinal products at early clinical phases<br />
Finding Therapies for Rare Diseases 7
Diversifying the risk in drug discovery<br />
•! Our model allows drug discovery and development at very limited costs<br />
•! The risk is diversified through the capacity to license our products to many<br />
partners<br />
•! Our extended pipeline comprises 135 drugs for rare diseases of which:<br />
•! 6 products already licensed<br />
•! 11 biomedicines ready for licensing<br />
•! 118 new biomedicinal candidates in development<br />
Finding Therapies for Rare Diseases 8
<strong>Mondobiotech</strong> discovery platform: Search&Match<br />
Search<br />
31000 journals and 70million publications<br />
Search algorithms that:<br />
•! Access privileged qualified data sources<br />
for collecting all knowledge on human<br />
peptides and rare diseases<br />
•! Elaborate all relevant information<br />
•! Populate our database to feed the<br />
bio-mathematical model for the Match<br />
Existing scientific information<br />
Physical<br />
Records<br />
Libraries of Periodicals<br />
& Textbooks<br />
Electronic<br />
Records<br />
Academia<br />
Publications in Diagrams & Images<br />
Multiple Languages<br />
Lab Results<br />
Peptidess<br />
Match<br />
312 peptides with 7000 rare diseases<br />
Match algorithms that:<br />
•! Apply our bio-mathematical model to<br />
link the gathered information on<br />
peptides physiological pathways and<br />
resulting biological activities with the<br />
rare diseases patho-physiological signs<br />
& symptoms<br />
•! Create multiple peptide-disease<br />
matches generating the best possible<br />
medicinal platforms candidates<br />
<strong>Mondobiotech</strong>’s evaluation process<br />
Scientific Attributes & Properties: Rankings<br />
Finding Therapies for Rare Diseases 9<br />
Rare DDiseassees<br />
Proposed therapies
Why peptides<br />
Peptides characteristics makes<br />
them perfect candidates for<br />
Search&Match:<br />
•! Human origin<br />
•! Well researched by the global<br />
scientific community<br />
•! Mode of action through ligation<br />
to specific receptors<br />
•! Known biological activity<br />
•! Proven safety profile<br />
•! Therapeutic platform potential<br />
•! Synthetically manufactured<br />
Finding Therapies for Rare Diseases 10
Case study 1: DK-1000 redirection in PAH<br />
Product description:<br />
Known biological activity:<br />
•! Antinflammatory<br />
•! Immunoregulatory<br />
•! Antiproliferative<br />
•! Antibiotic<br />
•! Antiapoptotic<br />
Safety:<br />
•! Approved in combination with<br />
phentolamine in erectile<br />
dysfunction<br />
•! Used in various clinical trials in<br />
lung diseases over more than 2<br />
decades and hundreds of<br />
patients<br />
Homology with other species:<br />
•! Identical in the following<br />
species: human, cow, rat, pig,<br />
dog, goat, sheep, mouse,<br />
monkey<br />
Rare indications and route of<br />
administration:<br />
•! PAH via inhalation<br />
•! Sarcoidosis via inhalation<br />
•! ARDS via injection<br />
•! IPF via inhalation<br />
Finding Therapies for Rare Diseases<br />
Male DK-1000 knock-out mice develop PAH<br />
and die (Circulation 2007;115;1260-1268)<br />
Distribution of DK-1000 in patients after<br />
intravenous injection<br />
(New Engl J Med 331:1116-1121, 1994)<br />
DK-1000 is severely missing in PAH patients<br />
(J Clin Invest 111; 1339-1346, 2003)<br />
DK-1000 in PAH clinical benefit via inhalation:<br />
113m after 12 weeks and 129m after 24 weeks<br />
(J Clin Invest 111; 1339-1346, 2003)<br />
11
Case study 2: DK-0112 redirection in Sarcoidosis<br />
Product description<br />
Known biological activity:<br />
•! Antinflammatory<br />
•! Immunoregulatory<br />
•! Antiproliferative<br />
Safety:<br />
•! Has been approved in DiGeorge<br />
Syndrome<br />
•! Used in various clinical trials in<br />
many diseases over more than 2<br />
decades and hundreds of<br />
patients<br />
Homology with other species:<br />
•! Identical in the following<br />
species: human, mouse<br />
Rare indications and route of<br />
administration:<br />
•! Sarcoidosis via injection<br />
•! Sezary Syndrome via injection<br />
Finding Therapies for Rare Diseases<br />
Patients with Sarcoidosis (Cutaneous Sarcoidosis or Erythema Nosodum)<br />
treated with DK-0112 via injection show improvements in skin as well as<br />
other organs (Clin Immunol Immunopathol. 1983 Mar;26(3):350-60)<br />
Patient Sex Age<br />
(Years)<br />
Skin Organs involved<br />
other than skin<br />
Duration of<br />
trial (weeks)<br />
Clinical results<br />
M.F. F 38 CS None 12 Improvement (some CS<br />
cleared, others flattened)<br />
M.S. M 34 CS None 12 Improvement (some CS<br />
cleared, others flattened)<br />
N.B. M 39 CS Liver 12 Improvement (CS flattened)<br />
D.F. M 26 CS None 6 Improvement (most CS<br />
cleared, others flattened)<br />
J.DU. F 34 CS Bilateral hilar<br />
adenopathy,<br />
lung (stage 1)<br />
L.C. F 24 EN Bilateral hilar<br />
adenopathy,<br />
liver<br />
J.DI. F 35 EN Bilateral hilar<br />
adenopathy<br />
F.G. M 21 EN Bilateral hilar<br />
adenopathy,<br />
liver<br />
12 Skin improvement (CS<br />
flattened), hilar adenopathy<br />
cured (Week 6), lung improved<br />
(Week 6)<br />
6 EN cured (3 weeks),<br />
hilar lymphoma cured<br />
(Week 9)<br />
6 EN cured (3 weeks),<br />
adenopathy improved<br />
(Week 12)<br />
6 EN cured (3 weeks),<br />
adenopathy improved<br />
(Week 12)<br />
12
mondoBIOTECH community<br />
Rare diseases are spread throughout the world and consequently experts in<br />
those diseases are dispersed and close to the patients!<br />
To benefit from the skills of this<br />
expert we developed a<br />
multidisciplinary open-source<br />
bioscience technology platform<br />
where:<br />
•! Every Researcher (PhD) is part of<br />
the discovery and development of<br />
the products candidates<br />
•! Key opinion leader MDs<br />
specialized in rare diseases<br />
validate the products candidates<br />
•! Researchers and MDs can submit<br />
their projects to the community to<br />
be evaluated as Medicinal Product<br />
Candidates<br />
30 million in USA<br />
35 million in Europe<br />
Unique genetic<br />
information<br />
RARE DISEASES<br />
PATIENTS AND<br />
ADVOCACIES<br />
Bio-mathematical model<br />
discovering medicines for<br />
rare diseases<br />
SEARCH&MATCH<br />
MONDOBIOTECH<br />
SCIENTIFIC<br />
COMMUNITY<br />
REDIRECTION<br />
INTO NEW RARE<br />
INDICATIONS<br />
Medicinal candidate<br />
validation &<br />
commitment to drug<br />
development<br />
New therapeutics<br />
discovered through<br />
well researched and<br />
peer-reviewed<br />
publications.<br />
Our drug development validation<br />
Finding Therapies for Rare Diseases 13
Licensing box<br />
Products ready to enter negotiation for<br />
licensing are accompanied by a box containing<br />
full product documentation that interested<br />
parties can request<br />
Box includes:<br />
Product Candidate<br />
Development<br />
Disease<br />
Financial Information<br />
IP & Legal<br />
Offered Services<br />
Dossier, API information, Published information<br />
In vitro activities, Pre-clinical activities, Clinical activities<br />
Disease dossier, Centers of excellence, Patients associations & other key entities,<br />
Published data, Development plan<br />
Key competitor, Other market information, Projected sales<br />
Protection documentation, PCT application, ISR (if available), National applications<br />
(if available): USA, Europe, Australia, Canada, Japan, Singapore, United Arab Emirates,<br />
Korea, Russia. Orphan drug designations/Orphan drug application and granting<br />
(EU/US), EU Regulations, US Regulations, Standard licensing contract and conditions,<br />
Standard service contract<br />
API, Protocol assistance, Orphan drug & Regulatory assistance, Networking with<br />
<strong>Mondobiotech</strong> Community (Scientific, Medical, Centers of excellence, Laboratory,<br />
Research organizations and other experts), Scientific & Medical Workshops for<br />
developing and prompting the project, Co-marketing, other development services upon<br />
request<br />
Finding Therapies for Rare Diseases 14
Products licensed<br />
DasKloster 1001-01<br />
Idiopathic Pulmonary Fibrosis (IPF) InterMune Inc.<br />
DasKloster 1000-01<br />
Pulmonary Arterial Hypertension (PAH) Biogen Idec Inc. & Lung Rx, LLC<br />
DasKloster 1000-02<br />
Sarcoidosis Lung Rx, LLC<br />
DasKloster 1000-03<br />
Idiopathic Pulmonary Fibrosis (IPF) Lung Rx, LLC<br />
DasKloster 1000-04<br />
Acute Respiratory Distress Syndrome (ARDS/ALI) Lung Rx, LLC<br />
DasKloster 0138-01<br />
Methicillin-Resistant Staphylococcus Aureus in Cystic Fibrosis Lung Rx, LLC<br />
Lung Rx, a wholly-owned subsidiary of United Therapeutics (Nasdaq: UTHR),<br />
has acquired the right to choose between additional 4 MPC within our pipeline at its discretion<br />
In development<br />
Ready for licensing<br />
Licensed<br />
Finding Therapies for Rare Diseases 15
Products ready for licensing<br />
DasKloster 0210-01<br />
Chronic Beryllium Disease (CBD)<br />
DasKloster 0247-01<br />
Chronic Thromboembolic Pulmonary Hypertension (CTEPH)<br />
DasKloster 0249-01<br />
Drug Resistant Tuberculosis In collaboration with Pharmarare SA<br />
DasKloster 0274-01<br />
Pseudomonas Aeruginosa in Cystic Fibrosis<br />
DasKloster 0014-01<br />
Hepatitis B Virus induced Hepatocellular Carcinoma<br />
DasKloster 0112-01<br />
Sarcoidosis<br />
DasKloster 0080-01<br />
Lymphangioleiomyomatosis (LAM)<br />
DasKloster 0039-01<br />
Asbestosis<br />
DasKloster 0501-01<br />
Scleroderma<br />
DasKloster 0182-01<br />
Sarcoidosis<br />
DasKloster 0228-01<br />
Idiopathic Pulmonary Fibrosis (IPF)<br />
In development<br />
Ready for licensing<br />
Licensed<br />
Finding Therapies for Rare Diseases 16
Products in development<br />
19 In Development for Proof of Concept<br />
Targets not disclosed<br />
99 Search&Match in validation<br />
Targets not disclosed<br />
177 Medicinal Product Candidates<br />
Targets not disclosed<br />
In development<br />
Ready for licensing<br />
Licensed<br />
Finding Therapies for Rare Diseases 17
Model validation<br />
For our vision, business model and potential impact on world health,<br />
the World Economic Forum selected us as Technology Pioneer 2008<br />
“To be selected as a Technology Pioneer,<br />
a company must be involved in the<br />
development of life-changing technology<br />
innovation and have potential for<br />
long-term impact on business and society.<br />
In addition, it must demonstrate visionary<br />
leadership, show the signs of being<br />
a long-standing market leader<br />
– and its technology must be proven.”<br />
(World Economic Forum’s Technology Pioneer program )<br />
Technology Pioneers were Google, Mozilla, the Wikimedia Foundation and Twitter<br />
Finding Therapies for Rare Diseases 18
mondoBIOTECH holding AG<br />
DasKloster<br />
Mürgstsrasse 18<br />
CH-6370 Stans<br />
Switzerland<br />
www.mondobiotech.com<br />
Paolo Bassanini<br />
Investor & External Relations<br />
T. +41 840 200 010<br />
F. +41 840 200 011<br />
investor@mondobiotech.com<br />
Finding Therapies for Rare Diseases 19
Change language