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Home , Macrolide, Solithromycin

Developing Well- Differentiated to Meet Medical Needs

Cempra Corporate Presentation

Prabhavathi Fernandes, Ph.D. President & CEO

November 2014 Forward Looking Statement

This presentation contains forward-looking statements regarding future events. These statements are just predictions and are subject to risks and uncertainties that could cause the actual events or results to differ materially. These risks and uncertainties include, among others: risks related to the costs, timing, regulatory review and results of our studies and and those of our strategic partners; our need to obtain additional funding and our ability to obtain future funding on acceptable terms; our anticipated capital expenditures and our estimates regarding our capital requirements; our and our strategic partners’ ability to obtain FDA and foreign regulatory approval of our product candidates; our dependence on the success of and TAKSTA; the possible impairment of, or inability to obtain, intellectual property rights and the costs of obtaining such rights from third parties; the unpredictability of the size of the markets for, and market acceptance of, any of our products, including solithromycin and TAKSTA; our ability to produce and sell any approved products and the price we are able to realize for those products; our ability to retain and hire necessary employees and to staff our operations appropriately; our ability to compete in our industry; innovation by our competitors; and our ability to stay abreast of and comply with new or modified laws and regulations that currently apply or become applicable to our business. Please refer to the documents that we file from time to time with the Securities and Exchange Commission.

2 Highlights

. Cempra has two differentiated antibiotics with broad and large commercial potential ̶ Both in late stage of development

Solithromycin – Potent 4th Generation , First Fluoroketolide

. First Oral and IV macrolide for monotherapy in CABP . Suspension for pediatrics – First in over 2 decades . Potential for broad adult and pediatric use

TAKSTA – Long History of Use in EU for Prosthetic Joint (PJI)

. U.S. development for oral, chronic treatment of bone and joint infections replacing long-term IV and multiple surgeries

3 Cempra’s Portfolio

Product Indication Formulation Preclinical Phase I Phase II Phase III Milestones

Oral

Community Acquired IV-to-Oral Bacterial

Oral Suspension / Pediatric Solithromycin (CEM-101) Biodefense Animal Rule Oral / Suspension

Urethritis Oral

Anti-inflammatory / NASH Oral

Oral-Chronic Prosthetic Joint Infections Taksta Acute and Chronic Oral-ABSSSI Treatment of MRSA

Oral Suspension / Pediatric

Non- Diabetic Gastroparesis and GERD Macrolide

4 Investing in Antibiotics Now

Increasing antibiotic resistance to generic drugs

Few products approved / in development Many are hospital intravenous use only

CDC / FDA / WHO New laws to help antibiotic developers

New regulatory guidance, increased development efforts, increased Pharma interest

5 Large Macrolide Market Opportunity

2013 US Retail Antibiotic Prescriptions Total 264 MM Annual Rxs , a macrolide, is the most widely prescribed treatment for CABP 21.3 38.7 and other RTIs – >60% of market Beta-lactams 28.0 Fluoroquinolones . Broad spectrum of activity 78.8 61.6 . Good safety Other antibacterials . Excellent tissue/intracellular distribution and 36.0 / anti-inflammatory activity

Source: IMS Health (Retail) AMR Hospital Data (Inpatient)

. 51 Million prescriptions were written for azithromycin in the U.S. in 2013 2013 IMS New Prescription Audit Increasing resistance - 40% of U.S. pneumococci a; 96.4% in China b a Jones, RN. DMID. 2013;75:107-109; b Kim, SH. AAC. 2012;56:1418-1426.. Treatment failure of macrolide resistant pneumonia results in increased cost, and hospital admission Health Policy Institute, Univ. of California, Irvine. Reynolds et al, Resistance and Control 2014: 3:16

6 Need For a New Macrolide

. In vitro activity of solithromycin and azithromycin against 927 pneumoniae from RTI samples collected in 2012-2013

MIC Percentage Antibiotic Region (µg/mL) S R 90% Europe (418) 100 0 0.06 Solithromycin NA (380) 100 0 0.25 Asia (129) 100 0 0.5 Europe (418) 71.3 28.0 > 1 Azithromycin NA (380) 56.3 42.6 > 1 Asia (129) 28.7 70.5 > 1

Morrissey, I. ECCMID 2014. Abstr. P1584.

7 Macrolides Have Broad Hospital & Primary Care indications . CABP and respiratory tract infections (RTIs) represent > 35MM hospital treatment days and > 115 MM prescriptions in the communitya

2013 AMR Hospital Days of Therapy 2013 IMS Retail Scripts

35 RTIs: ~115 MM scripts annually 40 ~34MM 30 ~29MM days 35 Millions ~28MM

Millions ~28MM 25 30 20 25

15 20 ~13MM 15 Scripts ~10MM 10 ~6MM days 10 5

Days of Therapy Therapy of Days ~420k days ~400k days 5 0 CABP Bronchitis 0 Sinusitis Otitis MediaBronchitis Pharyngitis CABP Hospital Days of Therapy Retail Scripts a Source: AMR Hospital Data, IMS NPA and NDTI

. Outpatient treatment failures from resistance to generic antibiotics, resulting in more and more hospitalizations for IV therapy, risk of improperly treated infection, HAI b b Antimicrobial Resistance and Infection Control 2014: 3:16

. A new primary care antibiotic for RTIs that is safe and effective is an urgent need

8 What is Solithromycin? Currently Approved Macrolide Antibiotics

Solithromycin - the first fluoroketolide

Binds 23S RNA at 3 regions

Llano-Sotelo B, Dunkle J, Kleoacki D, Zhang W, Fernandes P, Cate JH and Mankin AS. AAC 2010, 4961-4970

9 Solithromycin Highlights

Clinical Trials . Phase 3 Oral, CABP completed enrollment Q3 2014, data expected Q1 2015 . Phase 3 intravenous-oral switch study; enrolling . Phase 3 in gonorrhea enrolling. Phase 2 completed with 100% cure in culture proven cases. . Phase 1 in pediatrics, enrolling

Strategic . BARDA HHS: $58MM contract – Development of Soli for pediatric use Partnerships and against bioterror . Toyama Chemical (FujiFilm): Global development program – exclusive license for Japan. $20MM upfront / milestones received; up to $50MM in additional milestones; tiered royalties based on sales

Regulatory & IP . Qualified Infectious Disease Products (QIDP) granted by FDA designations; oral and IV formulations for CABP . Provides priority review – 8 months . QIDP for gonorrhea . NCE patent to 2025 plus PTEs; Polymorph patent to 2032, and additional patents

10 CABP – Most Frequent Infectious Disease in the U.S. . Community Acquired Bacterial Pneumonia (CABP) – #1 cause of death from an infection

. 5 to 10 million CABP cases annually, 1.1 million patients hospitalized per yeara ─ More common in older adults (>65 years) and young children

Pneumococcal infections cause more deaths per year in U.S. than breast or prostate cancer. Xu, et al. Deaths: Final Data for 2007. Natl Vital Stat Rep. 2010;58:1-51. Respiratory disease incidence is increasing; growing numbers of COPD and asthma patients Drug Discovery News, May 2012.

. Appropriate empiric therapy is critical to positive outcomes . Multiple pathogens can be involved - Pneumococcus – the most frequent cause

a Freeman, MK. CABP: A Primer for Pharmacists: US Pharmacist July 1, 2013

11 Rising Hospital Discharges for CABP

. CABP remains the leading cause mortality in the US

Source: 2011 HCUP, ARHQ.gov 12 Safety and Efficacy Deficiencies of Current CABP Therapies

Current IDSA/ATS recommendation to give broad spectrum, empiric coverage:

1) A β-lactam plus . Requires intravenous Mortality rates a macrolide (e.g., ceftriaxone) in hospitalized and hospitalization CABP patients ─ No oral switch therapy is 23% replacement – 30-day ratea . Azithromycin for Legionella and a Freeman, MK. US Pharmacist. July 1, 2013 Cost and hazards of HAI b Magill, SS. And CDC and Emory Authors. NEJM 2014. 1198-1208, 2014 Or 2) A fluoroquinolone . IV and Oral available – and have broad spectrum activity, (e.g., Levaquin, however, they are not in favor for treating CABP because: Avelox)

─ FQ treatment failure higher than ̶ Treatment failures from resistant strain selection ceftriaxone plus azi a C.difficile ─ Higher mortality when FQs used ̶ Kill bowel flora – associated with colitis without a macrolide b ̶ Adverse tendonitis, Achilles tendon rupture, hepatotoxicity and peripheral ─ FQs no longer used in CABP in neuritis, retinal detachment several countries ̶ Not approved for use in pediatrics a Fuller, DF, Low, EL.,CID 2005. 41: 118-121 b Martınez, JA., et al. CID. 2003, 36: 389-395 The only oral option is fluoroquinolone. but there is growing concern among physicians because of safety 13 Solithromycin – Spectrum of Activity that Addresses CABP Pathogens

. Solithromycin has class-leading potency & spectrum in vitro against CABP pathogens

Gram Organisms Solithromycin Azithromycin Cephalosporin Fluoroquinolone

Positive    

Negative    

Staphylococcus Positive aureus    

Legionella Atypical pneumophila    

Atypical  /   

Chlamydophila Atypical pneumoniae    

. Interacts with bacterial at three sites – Resistance rare and it could only occur if mutations occur at three distinct sites

14 Solithromycin Has Distinct Advantages Over Azithromycin

Solithromycin is being developed as oral capsules, pediatric oral suspension and intravenous formulations

ACTIVITY PK . 4-16 fold more active in vitro . Best oral bioavailability . Active against azithromycin-resistant . No trailing blood levels strains . Better intracellular activity . Bactericidal for many pneumococcus . Better amniotic fluid and fetus exposure . Stronger anti-inflammatory effects

DRUG STABILITY TOLERABILITY / SAFETY & EFFICACY . More stable – no cladinose in . Better tolerated (less nausea) solithromycin . Safer – Negative QT, no Tinnitus . Ready to use IV bags in development . Monotherapy . Ophthalmic solutions stable

15 Solithromycin: Phase 3 CABP Studies

. NDA on 2 products: Oral capsules and Intravenous ̶ Combined safety and efficacy data from two CABP Phase 3 studies ̶ Both studies approximately 800 patients each ̶ Comparator Avelox ()

Phase 3 Study 1: SOLITAIRE Oral SOLI- Oral -5 d Completed Enrollment –

MOXI Oral -7 d Secondary: MITT Q3 2014 SFU 5-10 days after last dose Data expected Q1 2015

Primary: Early Response ITT

Phase 3 Study 2: SOLITAIRE IV to Oral SOLI IV to Oral

MOXI IV to Oral Secondary: MITT Enrolling SFU 5-10 days after last dose

Primary: Early Response ITT

16 Bacterial Urethritis Phase 3 Trial Enrolling

Solithromycin was 100% effective in all culture proven cases of gonorrhea in a Phase 2 trial

. Phase 3 is enrolling 300 patients with gonorrhea with or without Chlamydia

. Patients receiving a single dose of solithromycin 1000 mg PO or ceftriaxone 500 mg IM plus azithromycin 1000 mg PO

. Primary efficacy endpoint – Culture negative at 7 days (TOC); secondary end point is eradication of GC and Chlamydia, and safety and tolerability

. NDA is expected to be submitted with the CABP NDA

17 Market Research1 Suggests a High Unmet Need for a Novel Antibiotic to Treat CABP

Products to Overcome . Respondents (80%) want a new antibiotic to overcome rising Increasing resistance Resistance

Improved Safety / . Respondents agreed that new, safe antibiotics are needed (QT Tolerability negative, not associated with C. difficile diarrhea)

Products Offering . Hospital PharmDs and inpatient physicians emphasize the lack of IV to Oral Step- options that allow patients to transition to an oral formulation of Down Therapy the IV antibiotic

Antibiotics for . Numerous physicians report difficulty treating patients with kidney Patients with disease Kidney Disease

1. Source: Trinity qualitative primary market research with PCPs, infectious disease specialists, pulmonologists, hospitalists, ICU intensivists, hospital pharmacy directors and commercial payors.

18 Commercialization Phases

. Disease awareness campaign . Profile key accounts and influencers ONGOING PRE-LAUNCH . Segmentation to prioritize opportunity . Coming Soon ads

. Introduce SOLI, establishing it as best AT choice for CABP LAUNCH APPROVAL . Hospital and managed care formulary plans . Promotional Med Ed campaign

EXPANDED . Continue to build brand awareness POST- RTI’s and . Promotional campaign aligned to new indications APPROVAL Other Indications . Expanded physician specialty reach

19 Solithromycin’s Broad Use Potential

Respiratory Tract Infections (RTI) CABP HAP, Simple RTI’s, Pharyngitis, Sinusitis, Bronchitis, Acute Exacerbation of Chronic Bronchitis (AECB) Primary Indication Antibacterial and Anti-inflammatory COPD, Cystic fibrosis, Panbronchiolitis, NASH Special Populations BARDA funded Pediatrics and Pregnancy No pediatric drug with broad potential in development Infections in pregnancy – neonatal Infections In Utero – Premature, Cerebral palsy, Autism

Biodefense Multiple Unidentified Pathogens BARDA funded Anthrax, Tularemia

Sexually Transmitted Genital Infections (Gonorrhea and Chlamydia) Diseases Major public health crisis – multi drug resistance, no oral therapy

GI & Others Other Infections Ophthalmic Helicobacter gastritis, Campylobacteria, tick and insect borne diseases, diarrhea, and ophthalmic drops

20 TAKSTA™ (Fusidic Acid)

An Oral Antibiotic for MRSA Infections Being Developed for Chronic Use in Bone and Joint Infections in the U.S.

21 Taksta Highlights

What is Taksta? . Cempra’s proprietary fusidic acid dosing regimen . 40 years of safety and efficacy in acute and chronic oral use in staph infections (including MRSA) ex-U.S.

. Unique structure, no known cross resistance with any other antibiotic

Clinical Trials . Phase 2 PJI study data reported, study stopped . The pivotal study is being discussed with FDA

Regulatory . Orphan Drug Designation for PJI granted by FDA (October 2013) . Orphan Drug designation benefits Cempra’s loading dose ̶ 7 - year exclusivity 120 ̶ Tax credit for 50% of clinical trial cost 100 and PDUFA fees exempted 80

. GAIN MRSA-Potential QIDP 60

40 . Loading dose patent into 2029 and PTEs European dosing Intellectual . EU Dose 500 mg dose Concentration (mg/L) Concentration 20 . Cempra dose 1200 mg Q12h Property . Well tolerated in ABSSSI Phase 2 study; Day followed by 600 mg Q12h 0 no resistance seen 0 24 48 72 96 120 Time (hrs)

22 TAKSTA for Chronic Oral Use in Bone and Joint Infections . Compassionate use cases of bone/prosthesis infections in North America

. Total Joint and Hardware Procedures – 3,286,000/yeara,b ̶ 200,000 Hip Replacements; 550,000 Knee Replacements in 2007* ̶ 1% of hips and 2% of knees develop PJI's* *Del Pozo J.L. & Patel R. NEJM 361: 787794, 2009 . Potential use in osteomyelitis, septic arthritis, and diabetic foot

. Phase 2 study, Stopped April 2014. Phase 3 meeting with FDA expected in Q4 2014

a Life Science Intelligence market research report. U.S. Markets for Large Replacement Technologies in 2012. March, 2012. b Life Science Intelligence market research report. U.S. Markets for Small Joint Implants and Hardware for the Extremities. January, 2012.

23 Achieved and Projected Milestones

 1Q 14: QIDP designation by FDA for solithromycin for gonorrhea

 1Q 14: Negative QT results for solithromycin

 1Q 14: Pediatric trial initiated for solithromycin

 2H 14: Initiated Phase 3 gonorrhea study for solithromycin

 3Q 14: Enrollment completed, Phase 3 oral for solithromycin

. 4Q 14: Meet with FDA to define pivotal study for Taksta

. 4Q 14: Initiate Phase 2 COPD study for solithromycin

. 1Q 15: Data expected SOLITAIRE Oral Phase 3 CABP

24 Capitalization

Cash & Equivalents (9/30/14) $74.2MM

Long-Term Debt (9/30/14) $18.1MM

Shares Outstanding (10/24/14) 35.8MM

25 Proven Management Team

Prabhavathi Fernandes, PhD . Azactam (aztreonam) . Biaxin () President & CEO . Dificid ()

. IPO and M&A Mark Hahn, CPA . Athenix-Bayer CropScience CFO . Charles & Colvard (CTHR) . E&Y

. Levaquin David Moore, MBA . Topamax CCO . Ultram . Nucynta

Gary Horwith, MD . S. aureus vaccine EVP Regulatory . Abelcet

David Oldach, MD . Viread GILEAD . GS-9190 SVP Clinical . Combinations against HCV

. Injectable David Pereira, PhD . Dobutamine HCl injection SVP Chemistry . Ranitidine injection

26 Take Away Notes

. Cempra has two differentiated antibiotics in clinical development

. Solithromycin is a next generation macrolide being developed as adult oral, intravenous and pediatric formulations

– Available in oral capsules / powder for suspension / Intravenous

. Late-stage clinical development - Enrollment in the oral Phase 3 trial completed, data expected 1Q 2015 . Cash sufficient, based on current assumptions, to run current operations into 2016

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