Alaxia, overview of our knowledge & products developed

1. Alaxia
A company dedicated to orphan drugs
Meveol program supported by
ALAXIA SAS, France

2. Location and Primary Focus
• Incorporated in 2008, ALAXIA is a private
biotech company based in Lyon, France.
• Main Focus: Development of medicinal products
targeting respiratory diseases, Products exiting
our Peroxidase plate-form.

3. Organizational Structure
Board
Dr Ernest NUSSBAUMER
President
Philippe BORDEAU
CEO
Dr Jean Paul PERRAUDIN
CSO
SCIENTIFIC COMMITEE
(G Bellon, P Diot, S Elborn,… )
Annie Claude BENICHOU, MD
Medical Advisor
Dr Maria CHAVARINO
PharmD
Head Regulatory Affairs
Meveol dvpt program Pilot
Dr Emmanuelle GEORGI
Dr Sandrine PERROTTO Dr Naima BENABDELLAH
PhD PharmD Philippe NUSSBAUMER
MD External relations (NPO,…)
R&D Lab Pharmaceutical Affairs Medical Affairs
External advisers
Dr Philippe DAVIOUD (Pharm), Dr Behrouz KASSAI (Med),,
Dr Annie Claude Benichou (MD), Dr Joël Guillemain (Tox),
Dr Yves Dumas (Reg), ERA ( FDA)

4. Intellectuel Property and Corporate Assets
• Intellectual Property
• 6 Patents
• Patents on formulation
• Patents on application (Infectious disease, CF, COPD)
• Patents on active ingredients manufacturing
• 2 Brands
• Orphan medicinal product designations (2009) in regards to our
novel compound Meveol for Cystic Fibrosis
• EU/3/09/654
• US #09-2946
• Expertise
• Plate-form on Peroxidase
• AMP (Antimicrobial Proteins)

5. Executive Summary of why to
Partner with Alaxia
• Alaxia prepare phase 1 for Meveol
• Meveol is safe (Safety pharm, gentox,… NO TOX)
• Meveol can be breathed for respiratory diseases or ingested for gastric
• Secured U.S. & E.U orphan drug designation and fully committed to molecule,
regulatory approval and launch
• Alaxia seeking funding/partnership to expedite clinical program, regulatory
filings, and co-marketing
• Other products under development

6. Drug Pipeline Strategy
Pipeline strategy
1) Develop Meveol® as orphan drug for Cystic Fibrosis
▪ ~100,000 patients affected by pulmonary infections
2) Strengthen other programs currently under discovery, dvpt, …
3) License products, program
▪ URTI (Sore throat, sinusitis), LRTI
▪ COPD

7. Alaxia’s Pipeline
Application Discovery Preclinic PhaseI PhaseII PhaseIII To Patient
Cystic
MEVEOL
Fibrosis
ALX-08 COPD
ALX-10 Lung Cancer
Gastric
ALX-G01
Cancer
Crohn‘s
ALX-G02
disease
ALX-06 Influenza
ALX-09 Tuberculosis
Oral & Nose
ATC-01
Care

8. MEVE L
Cathy, Jacky
Pilou, Cezar
www.alaxia-pharma.eu
ALAXIA SAS
3-11 Rue Perlerie
F- 69120 Vaulx en Velin
Phone : +33.(0)4.72.81.09.26
Fax : +33.(0)4.78.41.17.79
Email : contact@bioalaxia.eu
Program supported by
www.meveol.eu

9. Meveol : Alaxia’s First Drug in Development
• Attributes of Meveol for Cystic Fibrosis
• 1st product exiting in our peroxidase plateform
• Antimicrobial, wide spectrum,
– Pseudomonas Aeruginosa , Burkholderia cepacia, MRSA, Mycobacterium abscessus…
• Mechanism of action:
1) Delivers the missing antimicrobial compound,( OSCN-),
directly on the lung epithelium
2) Disrupts established biofilm (Lactoferrin)
• No antibiotic resistance risk
• Very low toxicity
• Inhalation use
• Strong scientific background in respiratory
– Cystic Fibrosis
– URTI, LRTI,…

10. Recent Articles of Interest
“Recent Discovery“ done by Banfi, Conner, Childers, Rogan *
on innate lung defenses
• Childers M, Eckel G, Himmel A, Caldwell J. A new model of cystic fibrosis pathology: lack of
transport of glutathione and its thiocyanate conjugates. Med Hypotheses. 2007;68(1):101-12.
SCN- doesn’t cross CFTR
• Moskwa P, Lorentzen D, Excoffon KJ, Zabner J, McCray PB, Nauseef WM, Dupuy C, Bánfi B. A
novel host defense system of airways is defective in cystic fibrosis. Am J Respir Crit Care Med.
2007;175(2):174-83 OSCN- natural antimicrobial compound miss in CF
• Conner GE, Wijkstrom-Frei C, Randell SH, Fernandez VE, Salathe M. The lactoperoxidase system
links anion transport to host defense in cystic fibrosis. FEBS Lett. 2007;581(2):271-8
Lactoperoxidase system innate host defense can’t work in CF
• Rogan MP, Taggart CC, Greene CM, Murphy PG, O’Neil SJ, McElvaney NG . Loss of microbicidal
activity and increased formation of biofilm due to decreased lactoferrin activity in patients with
cystic fibrosis. J. Infec. Dis. 2004; 190:1245-53
Lactoferrin activity is decreased in CF
Shows high interest of OSCN- + Lactoferrin (Meveol® API)
* not linked researchers team

11. CFTR, also SCN- channel
(an halogen channel)
Childers et al, 2007
SCN- alone is not able to reach airway peroxidase.
H2O2 generator, Duox, are inhibited by pyocyanin, Thus OSCN- cannot be produced locally
Note : Level of SCN- in CF patients saliva is statistically impaired compared to healthy non smokers or healthy smokers,
Minarowski, 2008

12. Cystic Fibrosis Overview
Airways, Peroxidases and OSCN-
Rada et al, 2008
Fischer, 2009
Childers et al, 2007

13. MEVEOL®
Meveol® (OSCN-/Lactoferrin) works by following mechanisms
- Disrupts microbial biofilm (Lactoferrin)
OSCN- Lactoferrin
- Kills bacteria (OSCN-/Lactoferrin)
- Targets to compensate and to restart airway
peroxidase system (OSCN-) using endogenous H2O2
Biofilm
OSCN-
Airway peroxidase
Pseudomonas
CFTR
H2O2 O2 Respiratory tract
SCN- DUOX
Airway epithelium

14. Meveol® Some targets (Non exhaustive list)
Bacteria
• Acinetobacter species • Shigella sonnei
• Aeromonas hydrophila • Staphylococcus aureus, Staph. Aerogenes (+Gram)
• Bacillus brevis, Bacillus cereus (+Gram) • Streptococcus agalactiae, Strept. Faecalis (+Gram)
• Bacillus megaterium, Bacillus subtilis (+Gram) • Streptococcus mutans (+Gram)
• Burkholderia cepacia • Wolinella recta
• Campylobacter jejuni • Xanthomonas campestris
• Clostridium perfringens (+Gram)
• Yersinia enterocolitica
• Capnocytophaga ochracea
• Corynebacterium xerosis (+Gram)
• Enterobacter cloacae
• Escherichia coli
• Haemophilus influenzae
• Helicobacter Pylori
Virus
• Klebsiella oxytoca , Klebsiella pneumoniae • Herpes simplex virus
• Legionella • Immunodeficient virus
• Listeria monocytogenes (+Gram) • Respiratory syncytial virus
• Mycobacterium abscessus, smegmatis, tuberculosis • Influenza virus
• Neisseria species
• Pseudomonas aeruginosa
• Salmonella species Tested by Alaxia or Alaxia’s partners

15. Antibacterial activity vs. Pseudomonas aeruginosa
Active compounds at T0

16. Antibacterial activity vs. Burkholderia cepacia
Active compounds at T0

17. Antibacterial activity vs. MRSA
Active compounds at T0

18. Recent Trials: Mycobacterium abscessus
Meveol vs Mycobacterium abscessus
Meveol at T0 only
12
10
8
log CFU/ml
Control
6 Meveol
4
2
0
0 5 10 15 20 25 30 35 40 45
Contact Time (Hours)
Active compounds at T0
In growth media solution

19. Recent Trials: MEVEOL®
• In Vivo Preliminary results
• Species : Mice
• Breed C57Bl6
• Infection by mucoid Pseudomonas aeruginosa
(isolated from Cystic Fibrosis patients)
• Meveol treatment started 24 hours after infection
• Second treatment 48 hours after infection
• Sacrifice 72 hours after infection
Protocol agreed by CER Ethic committee on
animal welfare

20. In Vivo efficacy
5,0
4,5 Mice lung 72h after infection by 106 CFU mucoid pseudomonas aeruginosa
4,0
3,5
Lung bacteria (log CFU/g)
3,0
2,5
≠1,6 log
2,0
1,5
1,0
N=9
Average : 3,1 N=12
0,5 Average : 1,5
SD : 0.4
SD : 0.5
0,0
Infected control Treated
2 trials, same efficacy acheived

21. Key Investment/Partnership Highlights

22. Unique Characteristics of Company
• Compounds missing in Cystic Fibrosis patients (Meveol)
• Intellectual Protection
– Own Patents & Brands (Alaxia®, Meveol®)
– Internal R&D peroxidase plateform
• Orphan drug designation www.alaxia-pharma.eu
– EU/3/09/654 & US 09-2946
• Strong scientific background
– Recent US discovery (Banfi, Childers, Conner, Rogan, Rada)
– Proof of concept validation (Vitro, Vivo)
– Biofilm destruction, Eradication of bacteria targeted (Dartmouth Medical College)
• Unique know how
– Hypothiocyanite (OSCN-), Lactoferrin, Peroxidase approach
• A new compound with No Tox (Safety pharm, Gentox,…)
• Strong Scientific Committee (KOL)
• Interesting Pipeline
• License/formulations/program available