Microsoft powerpoint - mpex pharmaceuticals - non-confidential website deck - 04-08-10.pptx

11535 Sorrento Valley Blvd, San Diego, CA
www.mpexpharma.com
 Aeroquin (proprietary aerosol formulation of levofloxacin) a reduced risk clinical program; 100% of rights retained • Compelling results from Phase 2b trial in Cystic Fibrosis • Preparing to initiate Phase 3 trial in Cystic Fibrosis  Separate broad technology platform focused on efflux pump inhibitors (EPIs) • Collaboration with GSK fully funds this program  < 40 employee private company with supportive VC investors  Well-funded; completed a $40 million series D in early 2009 Mpex 2010
Important pharmacological and physical properties of new aerosol antibiotics for lung infections  Broad Spectrum Activity: Cover all relevant bacterial pathogens found  Good PK/PD Profile: Achieve sufficient lung levels to not only have maximum killing but also to reduce the potential to cause resistance  Activity in Lung Environments: Retain activity in biofilms and anaerobic environments and not be deactivated in sputum  Compliance: Short nebulizer delivery time, infrequent dosing and  Safety and Tolerability: Low systemic exposure and side effects Aeroquin is well positioned to satisfy all of these requirements
Mpex 2010
Aeroquin is a proprietary formulation of  Levofloxacin’s combination of potency, spectrum, solubility, and physico-  Novel formulation masks bitter taste associated with inhaled fluoroquinolones and increases AUC in the lung (to improve efficacy)  Manufactured in ready to use blow-fill-seal polyethylene ampules Mpex 2010
 Patent applications filed to cover multiple aspects of Aeroquin are  Exclusive license in place to use patented Pari eFlow nebulizer with • Drug/Device specific co-labeling – Aeroquin will only be approved for use with branded eFlow device which provides additional regulatory protections  Orphan Drug Status granted for Aeroquin in CF • 7 year exclusivity in U.S.
• 10 year exclusivit Mpex 2010
Cystic Fibrosis is a high value indication with  60,000 patients in the U.S. and E.U.
been shown to significantly improve lifespan for CF patients  Patients are chronically infected with bacterial pathogens (50%  Aerosol antibiotics are the preferred approach to care because very • Not feasible with oral or i.v. antibiotics • Max killing at site of infection with reduced resistance development • Lower systemic exposure and side effects Mpex 2010
Significant unmet clinical need for new and improved  Recent market research study conducted by a globall consulting firm has confirmed that there is still significant unmet clinical need for new and improved therapies for the treatment of CF  Additional inhaled antibiotic classes; provides additional options for  New Antibiotics for treating patients intolerant to or experiencing diminished responses to existing therapies  Shorter dosage duration and infrequent dosing schedules to help Mpex 2010
Goal for inhaled antibiotic treatment of chronic lung infections in CF is 12 months of treatment per year  Clinical goal of continuous therapy is the reduction of pulmonary  In order to best achieve 12 months of inhaled antibiotic treatment, • e.g. aminoglycosides, beta lactams, fluoroquinolones reduced resistance development, reduced class related side effects, and improved long term efficacy antibiotics of the same class (e.g. TOBI & Amikacin) or different formulations of the same antibiotic (e.g. TOBI & DPI TOBI) Mpex 2010
Objective:
 To evaluate the efficacy, safety and tolerability of Aeroquin administered daily for 28 days, compared to placebo in CF patients infected with Pseudomonas Study Design:
 Randomized, double blind, placebo controlled  51 clinical sites in the US and Europe  Patients required to have at least 3 courses of inhaled antibiotics in previous  Patients were allowed to remain on other medication shown to improve lung Mpex 2010
Aeroquin’s Successful Phase 2 CF Trial (Cont’d)  Aeroquin met the primary endpoint of reducing bacterial counts of Pseudomonas aeruginosa in sputum after  Clinically and statistically significant improvements versus placebo • Time to need for anti-pseudomonal antibiotics Mpex 2010
Aeroquin’s Successful Phase 2 CF Trial (Cont’d) in this trial, with higher doses showing improved responses  No significant change in antibiotic resistance was observed in this Mpex 2010
Aeroquin was well received by CF clinicians  A globally recognized consulting firm completed 51 physicians and  Physicians in both the U.S. and EU were excited about the use of Aeroquin as an inhaled maintenance therapy for CF patients with pulmonary infections  According the consulting firm Aeroquin received unusually high attractiveness ratings from surveyed physicians • An average of ~5.9 out of 7 on an attractiveness scale  Key benefits of Aeroquin are the reduction of exacerbations, dosing and treatment duration, and broad spectrum of activit Mpex 2010
Globally recognized consulting firm forecasts revenue of ~$350 million for Aeroquin in CF  Completed Q3, 2009; U.S. & Big 5 E.U., 51 physicians & 16 payor WW Aeroquin Non-Risk Adjusted Revenue in CF (2012-22)
f Dollars
o
Millions
* Assumes ~45% share of CF patients are treated with approximately 3-4 courses of Aeroquin per year Mpex 2010
 Statistically and clinically significant CF Phase 2b data Mpex 2010
 Efflux Pumps
• Reduce intracellular concentrations of membrane
• Responsible for multi-drug resistance in • Largely responsible for inability to develop  Efflux Pump Inhibitors (EPIs) as Drugs
resistance and increases antibiotic susceptibility • Suppresses the emergence of resistance• Enhances antibiotic • Can improve the “developability” of novel Inner
membrane
Mpex 2010
 Collaboration on combinations with both existing commercial  $15 million upfront in cash and equity funds lead optimization  Early milestones cover all costs of Mpex taking compounds through  Milestones of $200 – 250 million per product; $1.5 billion in total Mpex 2010
The Mpex management team is highly experienced  Dan Burgess – President and CEO, Director
 Mark Wiggins – Chief Business Officer
EVP Global Business Development, Biogen Idec  Dr. Mike Dudley
y – SVP of R&D and Chief Scientific Officer
VP Preclinical and Clinical Sciences, Diversa Corporation  Dr. Jeff Loutit – Chief Medical Officer
VP Clinical Science, InterMune, Inc.
 Liz Morgan – VP Clinical Operations
VP Clinical Operations, Nereus Pharmaceuticals  Dana Krzyston, CPA – VP Finance & Admin
 Dr. Olga Lomovskaya – VP Biology
Associate Director of Biology, Essential Therapeutics  Dr. Scott Hecker – VP Chemistry
Mpex 2010

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