I phar presentation-skolkovo_15.04.2013

IPHAR

DRUG DEVELOPMENT FOR FOREIGN

AND DOMESTIC MARKETS

2013 Copyright © iPhar

President

Veniamin KhazanovProfessor, MD, Ph.D., D.Sc.

IPHAR IS A HOLDING COMPANY

Main activities:

Development of innovative drugs for Russian and Global market

Contract services - preclinical and clinical studies of drugs

(over 60 studies for Russian and foreign customers in the last 3 years)


APIs screeningProject “packaging”

Attraction of investmentsIP protectionDrug formulationTechnology scalingPreclinical researchClinical trialsRegistrationPilot manufacturingCommercialization

Manufacturing and Sales

CustomersDevelopment andCommercialization


Russian and foreign drug manufacturers

Startup companies

IPHAR – DRUG DEVELOPMENT SERVICES

Commercialization department

(preparing projects, attraction of investments, IP

protection)Management

(planning, management and

control of all activities – from

idea to implementation)

Clinical department

)clinical trials of new drugs in

Russian clinical centers(

Technological department

)development and scaling drug

synthesis technology(

R&D center specializing in

preclinical trials of drug safety and

efficacyTeam)over

60 employees,6 D.Sc.,12 PhD(

STRUCTURE OF IPHAR


INTERACTION WITH OTHER ORGANIZATIONS

Substance synthesis:TSU, OOO “Novohim”

(Tomsk), NIOC SB RAS (Novosibirsk), OAO

“Organica” (Novokuznetsk), etc.

Laboratory analysis:TSU, TPU (Tomsk), institutes of SB RAS

(Novosibirsk), institutes of RAMS and RAS (Moscow)

Clinical trials:clinical centers of Tomsk,

Novosibirsk, Kemerovo and other Russian cities

Russian and foreign

pharmaceutical companies

IPHAR

2013 Copyright © iPhaR

ADVISORY BOARD

Professor Khazanov V. (iPhar) – pharmacologist, biochemist.

Developer of over 100 drugs. Creator of a group of companies specializing in drug development and manufacture.

– a prominent Russian chemist, inventor of 8 original drugs, author of 10 monographs on drug synthesis and development.

Prof. Salakhutdinov N. (Scientific Institute of Organic Chemistry, Novosibirsk) – major Russian specialist in medical chemistry and drug synthesis.

Prof. Gogvadze V. (Karolinska University, Sweden), D. Sci. – a major scientist in the field of molecular biology and toxicology.

Dr. Schwarz J. (Nano Essentials Inc., Canada) – formerly one of the heads of formulation department of TEVA (Israel).

Dr. Cleverley S. (ISIS Innovation, UK) – expert of Oxford University technology transfer center.

Prof. Granik V.


2013 2014 2015 2016

1. Antiulcer drug – peptic ulcer and GERD

2. Anti-inflammatory - rheumatism, arthritis

3. Anti-Parkinson drug - Parkinson's disease

4. Antiplatelet - cardiovascular disease

DEVELOPMENT OF INNOVATIVE DRUGS FOR GLOBAL MARKETS

2013 - 2015 IPHAR plans to complete preclinical studies of 4 innovative drugs and start clinical trials:


Product – new molecule based on pyridopyrazindione with a new mechanism of action on H+/K+ ATPase: a reversible potassium-independent proton pump inhibitor.

IP: Patent RU 2465274, priority date 05.03.2010; PCT/RU2011/000103; US application 13/602,239 dated 03.09.2012 (allowance for a patent dated 21.03.2013); EPO application 11750980.2 dated 04.10.2012; Ukraine and EAPO applications.

Competitive advantages compared to the best drugs on the market(proton pump inhibitors ):• reversibly inhibits enzymes of gastric mucosa;• does not suppress normal gastric acid secretion level;• does not inhibit gastrointestinal peristalsis;• reduces the risk of night-time acid “breakthrough” and “rebound” – sudden increase in acidity after cancelling the drug.

Consumer benefits – sustained normal digestion level in long-term treatment, reduction in adverse effects incidence and disease recurrence.

ANTIULCER DRUG


The drug is based on a new small molecule (terpenoid derivative), differing in its properties from the known antiparkinson compounds.

IP rights - OOO«Rionis»: patent RU 2418577, priority date 24.12. 2009; PCT/RU2010/000778; US application No. 13/518,814 dated 22.06.2012; EPO application 10844832.5 dated 20.07.2012; EAPO application.

Competitive advantages :•does not cause dyskynesia and other motor and non-motor adverse effects, present in levodopa;•effectiveness – as good as levodopa, unlike most competitors (dopamine agonists or MAO and COMT inhibitors);•can potentially have neuroprotective action and eliminate some non-motor disorders of Parkinson disease;•can be used to treat drug-induced parkinsonism.

Consumer benefits :1) Makes safe and long-term PD monotherapy possible;2) Slows down the disease progression;3) Eliminates non-motor disorders.

ANTI-PARKINSON DRUG


Product - the new patented compound - N-[3-(4-nitrophenylamino)-indole-2-ilmethylene]aminoguanidine hydrochloride with a new mechanism of action - selective inhibition of inducible NO-synthase.

IP: Patent RU 2478618, priority date 15.03.2010; PCT/RU 2011/000142; US application 13/634,840 dated 13.09.2012; EPO application 11756610.9 dated 12.10.2012; Ukraine and EAPO applications.

The main advantage of the drug over the worldwide used analogs (NSAIDs) is the absence of dangerous ulcerogenic effects, because its pharmacological target is NO-synthase and not cyclooxygenase.

Efficiency - the new compound exhibits anti-inflammatory action as potent as voltaren and indomethacin in peritonitis and arthritis models (ED50 – 50 mg/kg, mice, rats)

Safety - the compound has low toxicity (LD50>5000 mg/kg, mice).

Market. There are no direct market analogs of the new drug. World market of NSAIDs amounts to $10 billion.

ANTI-INFLAMMATORY DRUG


Product – a new molecule - indolinone-3 derivative with a new mechanism of action - a nitric oxide donor and a stimulator of soluble guanylate cyclase and cGMP synthesis.

IP: Patent RU (application № 2011144895 dated 08.11.2011); PCT/RU2012/000884.

Competitive advantages: Unlike aspirin, the new molecule does not have ulcerogenic side effect. Unlike the known antiplatelet drugs, it has anti-hypertensive and cardioprotective properties, reducing xenobiotic load, the incidence of adverse effects and costs of cardiovascular disease treatment.

Efficacy is proven in in vitro (GC activity) and in vivo models (platelet aggregation, parenchymal hemorrhage, hypertension). Effective doses: 10 -7 mol in vitro, 4-12 mg/kg (per os), 0,01-1,0 mg/kg (intravenously).

Low toxicity - LD50 = 8900 mg/kg in oral administration in mice.

Market. The world antiplatelet drug market amounts to $15 billion. The novel drug aims to be a possible alternative to clopidogrel and new antiplatelet drugs (prasugrel, ticagrelor) used in CVD treatment.

ANTIAGGREGANT )ANTIPLATELET DRUG(


Product – a new molecule – glycyrrhetic acid derivative, a structural analogue of the known compound - Bardoxolone methyl (Reata Pharmaceuticals/ Abbott Laboratories), which underwent Phase III clinical trials as antioxidant inflammation modulator for the treatment of chronic kidney disease in type 2 diabetes.

Mechanism of action – modulator of activity of transcription factors Nrf2k,NFkB and PPAR.

Promising areas of application:

1) Oncology;

2) Neurodegenerative diseases (Alzheimer's disease, Parkinson's disease, multiple sclerosis).

IP: Patent RU 2401273 dated 30.03.2009; Russian patent application № 2012129738 dated 13.07.2012

Efficiency: proven anti-tumor activity in vitro at doses of 0,5 – 5x10-6 mol

Low toxicity - LD50 > 5000 mg/kg in oral administration in mice.

ANTICANCER DRUG


IPHAR

President: Veniamin Khazanov,

Professor, MD, Ph.D., D.Sc.

Address: Elizarovykh str. 79/4, Tomsk, Russia

Tel./fax: +7(3822)248721

Mob.: +79138295599

e-mail: [email protected]

www.iphar.ru


Business

I phar presentation-skolkovo_15.04.2013