Corporate Presentation (Nasdaq: COLL) June 1, 2015

Transcription

1 Corporate Presentation (Nasdaq: COLL) June 1, 2015

2 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the company s current expectations. For example, there can be no guarantee that we will obtain approval for Xtampza or any of our other product candidates from the U.S. Food and Drug Administration ( FDA ) or foreign regulatory authorities; even if Xtampza is approved, we may not be able to obtain the label claims that we are seeking from the FDA. Furthermore, we are subject to patent infringement litigation relating to Xtampza and may, in the future, be subject to additional litigation relating to our other product candidates, which may be expensive to defend and delay the commercialization of Xtampza or our other product candidates. Management s expectations and, therefore, any forwardlooking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: our ability to commercialize our product candidates; the size and growth potential of the markets for our product candidates, and our ability to service those markets; our ability to develop sales and marketing capabilities, whether alone or with potential future collaborators; the rate and degree of market acceptance of our product candidates; the success, cost and timing of our product development activities, studies and clinical trials; the success of competing products that are or become available; and our expectations regarding our ability to obtain and adequately maintain sufficient intellectual property protection for our product candidates. These and other risks are described under the heading Risk Factors in the registration statement on Form S-1 (commission file number ), which was declared effective by the Securities and Exchange Commission ( SEC ) on May 6, Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release. 2

3 Developing Proprietary, Abuse-deterrent Pharmaceuticals Leveraging our Drug Delivery Platform Technology Proprietary DETERx Technology Platform Microspheres made of drug, fatty acid and waxes creates ER properties Inactive components are made of hydrophobic, waxy materials Potential significant therapeutic and abuse-deterrent benefits Potential best-in-class technology Each microsphere is extendedrelease and abuse-deterrent Development Portfolio Focused on Chronic Pain PRE-CLINICAL IND CLINICAL NDA OXYCODONE COL-172 OXYMORPHONE COL-195 HYDROCODONE COL-196 MORPHINE COL-171 METHYLPHENIDATE Lead program NDA filing accepted Feb. 10, 2015 PDUFA goal date Oct. 12, 2015 Developing commercial infrastructure for launch Second opioid product clinic ready by year end Methylphenidate out-licensing opportunity 3

4 Key 2015 Milestones NDA Accepted for FDA Review Raised $50M Mezzanine Round of Financing Completed IPO Raising $80M Initiated Commercial Build with Hiring of Key Leadership 4

5 The Epidemic: Chronic Pain and Prescription Drug Abuse Chronic pain represents a public health crisis of epidemic proportions¹ Over 100M people in the U.S. suffer from chronic pain; > heart disease, cancer and diabetes combined Over $560BN in healthcare and productivity costs per year Prescription opioids remain the primary treatment for chronic pain 29M TRx/year annually for extended-release (ER) and long acting (LA) opioids 2 OxyContin OP has a 20% U.S. market share (annual ER/LA TRx); $2.5BN U.S. Sales but deaths in the U.S. from prescription opioid overdoses have grown from ~4,000 in 1999 to ~16,000 in Abuse of painkillers cost payers over $72BN per year in direct healthcare costs 1 ¹ American Journal of Managed Care Prescription Opioid Abuse: Challenges and Opportunities for Payers. 2 IMS Data National Center for Health Statistics / CDC, National Vital Statistics Report, Final death data for each calendar year (June 2014). 5

6 Significant Support for Abuse-deterrent Opioids FDA Action FDA final guidance Abuse-Deterrent Opioids Evaluation and Labeling FDA creating incentives for improved ADF technologies Government STOPP Act (Stop Tampering of Prescription Pills Act) States starting to mandate that insurers cover abuse-deterrent opioids Health Canada proposed regulations requiring all opioids to have abusedeterrent properties Industry Groups Industry groups (BIO and PhRMA) lobby FDA to reject new opioid generics that aren t ADF and remove non-adf generics from the market where there is an alternative Ultimately, FDA looks forward to a future in which all or substantially all opioid medications are less susceptible to abuse than the conventional formulations that dominate the market today. 1 1 FDA Federal Register, September 23, Development and Regulation of Abuse-Deterrent Formulations of Opioid Medications; Public Meeting. 6

7 % of Oxycodone Released Reformulated OxyContin OP Not Crush Resistant and Can Be Easily Defeated by Household Tools OxyContin OP Tablet $2.5 Billion in 2014 U.S. Sales 5.5 Million Prescriptions $6.39 Pill Crusher Abuseable Fine Powder in 16 Seconds + = 100% 80% 60% 40% 20% 0% % of Oxycodone Released 1 Hour 1 17% OxyContin OP Intact 1 Collegium in vitro dissolution study. 77% OxyContin OP Crushed BLACK BOX WARNING Instruct patients to swallow OxyContin tablets intact. Crushing, dissolving, or chewing the tablet can cause rapid release and absorption of a potentially fatal dose of oxycodone. [Section of Label: 2.5, 5.1] Implications for patient misuse and intentional abuse 7

8 Reformulated OxyContin OP has Decreased Abuse by Injection and Snorting but Oral Abuse has Increased Initial findings on abuse rates and routes of administration among individuals assessed for substance use treatment following introduction of reformulated OxyContin OP 1 Percent of Abusers 100% 80% 60% 40% 20% 0% 89% 35% Original OxyContin Before (n=2,015) Reformulated OxyContin After (n=490) 35% 14% 61% Source: Butler, et al. 2012; NAVIPPRO 2 nd Annual Scientific Meeting 28% 8% 3% 52% Any non-oral Inject Snort Smoke Oral 75% Abusers converted to oral abuse or defeated ADF mechanism and injected or snorted 2 1 Butler, et al. 2012; NAVIPPRO 2nd Annual Scientific Meeting 2 Abuse-Deterrent Formulations and the Prescription Opioid Abuse Epidemic in the United States: Lessons Learned From OxyContin; JAMA Psychiatry. doi: /jamapsychiatry ; March (n=88) 8

9 Types of Abuse-Deterrent Strategies Hard Tablet / Gelling Features Limitations Particle size reduction leads to significant increase in drug release Pre-treatment diminishes gelling activity Examples OxyContin OP (oxycodone ER) Hysingla ER (hydrocodone ER) Agonist / Antagonist Combination Potential for withdrawal Potential for decreased pain relief Embeda (morphine/naltrexone) Targiniq (oxycodone/naloxone) Soft Hydrophobic Microspheres 9

10 DETERx: Potential Best-in-Class Abuse-deterrent Technology DETERx Design Elements Microspheres made of drug, combined with fatty acid and wax excipients creates ER properties Microspheres are micron size, uniform in shape, soft and insoluble in water Drug is homogeneously dispersed within each microsphere Drug binds chemically with inactive components 10

11 Xtampza ER (oxycodone extended-release capsules) is being developed for the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. Regulatory Status Fast Track Designation granted 505(b)(2) regulatory pathway EOP2 and Pre-NDA meetings completed Numerous FDA advice letters NDA filing accepted Feb. 10, 2015 PDUFA goal date Oct. 12, 2015 Studies to Support NDA Bioequivalence and bioavailability clinical trials Phase 3 safety/efficacy clinical trial Abuse-deterrent studies to support differentiated labeling Supporting data for sprinkle and NG/G tube administration REMS program consistent with classwide REMS 11

12 Oxycodone Concentration (ng/ml) Oxycodone Plasma Concentration (ng/ml) Oxycodone Concentration (ng/ml) is designed to be used safely by patients with chronic pain with dysphagia (difficulty swallowing) Large Unmet Medical Need 11M patients in U.S. with chronic pain with dysphagia OxyContin OP cannot be used in this patient group Inadequate treatment options Sprinkle Food Studies Feeding Tube Studies Chewing PK Data Crushing PK Data Soft Food PK Data Xtampza DETERx Capsules Capsules Intact 40 Xtampza DETERx Capsule Microspheres Chewed 30 Chewed Time (hrs) Source Data: CP-OXYDET Xtampza Intact Capsules 50 Crushed Microspheres Crushed Time (hrs) Source Data: CP-OXYDET-25 Xtampza Microspheres Hours Source Data: CP-OXYDET-27 DETERx Capsules Intact Xtampza Capsules DETERx Microspheres Sprinkled on Food Xtampza Microspheres (Applesauce) 12

13 Pivotal Phase 3 Efficacy and Safety Clinical Trial Study Design vs Placebo, randomized withdrawal design 12 week study in patients with moderate to severe chronic lower back pain Opioid naïve and experienced subjects (N=389 randomized) Rescue medication: acetaminophen Results Achieved primary and secondary endpoints Sensitivity analyses all significant Safe and well tolerated 1 FDA approved Collegium s SAP for Study CP-OXYDET-08. Change from Baseline Change from Randomization Baseline to Week 12 / Early Discontinuation Average Daily Pain Intensity Score 1, DETERx p< Placebo 13

14 Comprehensive Evaluation of vs OxyContin OP Developed to be Consistent with FDA Guidance 1 Category 1 Lab based in vitro manipulation and extraction studies Category 21 Laboratory Pharmacokinetic based in vitro manipulation Clinical Trials and extraction studies Category 3 Human Abuse Potential Clinical Trials Numerous physical and chemical manipulation studies Numerous route specific extraction studies Five Clinical Trials CP-17 (oral) CP-19 (nasal) CP-21 (nasal) CP-24 (oral) CP-25 (OxyContin) Two Clinical Trials CP-21 (nasal) CP-24 (oral) 1 FDA Final Guidance, Guidance for Industry, Abuse-Deterrent Opioids Evaluation and Labeling, issued in April

15 Smoking Route Specific IV Injection Manipulation Chemical Physical Category 1: Extensive Evaluation Completed OxyContin OP vs. Summary of laboratory based in-vitro manipulation and extraction studies Experiment Objective Particle Size Reduction (PSR) studies Effect of household tools on increasing drug release (validated in independent third party laboratory) Extraction studies Drug released into solvents under multiple conditions Small volume extraction for IV injection Direct injection and melting Syringeability Extraction of drug into injectable amounts of water Ability to suspend in liquid and inject or melt and inject Drug passed through syringe after crushing and exposing to water Simulated smoking abuse study Drug vaporized for the purpose of smoking 15

16 Category 1: Attempts to Inject Attempt to Suspend in Liquid and Inject Attempt to Melt and Inject 18 gauge 25 gauge 27 gauge Xtampza melted and attempted to draw into syringe Xtampza quickly solidifies and clogs needle Negligible amounts of microspheres pass through the largest needle Melt clogs syringe 16

17 % Drug Released % Drug Released Category 1: Attempts to Extract and Inject OxyContin OP vs. Injection Study: versus OxyContin OP (40 mg strength) Crushed Xtampza OxyContin OP Crushed, Microwave Xtampza OxyContin OP 1 Crushing method identified as most effective for each product in PSR study; Extraction following boiling temperature in water. 2 Extraction in boiling water following pretreatment of solid powder in microwave after 8 minutes of exposure. Injecting OxyContin OP Its super easy to bang the new OP formula. U just file it down to a powder, put on spoon, burn till it turns brown, add water and stir. U can then place a small piece of cotton in it and draw up. Same high as before.. * OxyContin label warning that injection of the inactive ingredients can result in local tissue necrosis, infection, and increased risk of heart disease 17

18 Oxycodone Plasma Concentration (ng/ml) Oxycodone Plasma Concentration (ng/ml) Category 2: Oral and Nasal PK Clinical Trials Demonstrate xxxxxxxx Abuse-deterrence Oral PK 1 Nasal PK Cmax (ng/ml) 80 Cmax (ng/ml) Bioequivalent Cmax decreases when microspheres are snorted IR Oxycodone Control Xtampza Intact Xtampza Chewed Xtampza Crushed 0 IR Oxycodone Control Xtampza Intact Xtampza Crushed 1. CP-OXYDET-17; Oral tamper PK study, naltrexone blocked subjects (N=44). 2. CP-OXYDET-21; Intranasal HAP study, non-naltrexone blocked subjects (N=36). 3. Bioequivalence as defined as 90% Confidence Interval within 80% to 125%. 18

19 Oxycodone Oxycodone Plasma Plasma Plasma Concentration Concentration (ng/ml) (ng/ml) (ng/ml) Oxycodone Plasma Concentration (ng/ml) Oxycodone Plasma Concentration (ng/ml) Oxycodone Plasma Concentration (ng/ml) Category 2: vs. OxyContin OP in PK Clinical Trial; OxyContin OP Demonstrates Dose Dumping OxyContin OP 1 Xtampza 1 Treatment Arm Cmax Tmax OxyOP Treatment Intact Arm Tablet Cmax 64.9 Tmax 5.0 OxyContin Intact OxyContin OxyContin Manipulated Intact IR OxyContin Manipulated Intact Manipulated OxyContin IR Manipulated Manipulated OxyOP Treatment OxyOP Crushed Intact ArmTablet Tablet Cmax Tmax IR OxyOP Crushed Intact Crushed Tablet Tablet Tablet OxyOP IR Crushed Crushed Tablet Tablet IR Manipulated IR Crushed Tablet Time 6(hrs) Crushing Time 6(hrs) results 8 in loss of ER properties Time (hrs) Time 6(hrs) Time (hrs) Time 6(hrs) Time (hrs) Treatment Arm Cmax Tmax DETERx Xtampza Intact Capsule DETERx Xtampza Manipulated Crushed Microspheres IR IR Manipulated Crushed Tablet Intact and Crushed curves Bioequivalent with similar Tmax Time (hrs) Time (hrs) BLACK BOX WARNING Instruct patients to swallow OxyContin tablets intact. Crushing, dissolving, or chewing the tablet can cause rapid release and absorption of a potentially fatal dose of oxycodone. [2.5, 5.1] CP-OXYDET-25 was a randomized (n=36), open-label, active-controlled, 5-period, naltrexone-blocked, healthy subject, crossover study. 19

20 AQ (ng/ml/hr) Category 2: vs OxyContin OP in PK Clinical Trial: Abuse Quotient (Cmax/Tmax) ~4x Baseline Lower than Baseline 0 DETERx Xtampza Intact DETERx Manipulated OxyContin Intact OxyContin Manipulated IR Manipulated Xtampza Crushed OxyContin Crushed IR Crushed Error bars = standard deviation AQ (Cmax/Tmax) provides a rough measure of rate of rise in plasma concentration AQ for OxyContin OP crushed and IR crushed are similar Source: data from CP-OXYDET-25 study. 20

21 Drug Liking Emax (Peak Effect) Drug Liking Emax (Peak Effect) Category 3: Intranasal and Oral Human Abuse Potential (HAP) Clinical Trials ( Drug Liking ) Intranasal HAP 1 Oral HAP Drug Liking E max (Peak Effect) P< P= Drug Liking E max (Peak Effect) P< P< P= Xtampza Crushed DETERx Crushed IN Snorted Xtampza Intact IR Oxycodone DETERx Intact Oral Crushed IN Oral Snorted Placebo 40 Xtampza Intact Xtampza Chewed IR Oxycodone Crushed Placebo Error Bars = ± Standard error of the mean Error Bars = ± Standard error of the mean 1 Data from CP-OXYDET-21 clinical trial (n=36). 2 Data from CP-OXYDET-24 clinical trial (n=36). 21

22 Label Differentiation Strategy Section of Label Dosage and Administration Pharmacology Black Box Warning and Precautions Throughout Label Drug Abuse Other Potential Differentiation Additional language: Open capsule and sprinkle, administer via NG/G tubes Addition of PK data from Abuse-deterrent sprinkle clinical trials Removal of the following: Instruct patients to swallow OxyContin tablets intact. Crushing, dissolving, or chewing the tablet can cause rapid release and absorption of a potentially fatal dose of oxycodone*. * Included in label sections black box, 2.5, 5.1, 5.2, 17 Label will be consistent with FDA Abuse-Deterrent Guidance: Category 1 In Vitro Claims 1 Category 2 PK Claims (Oral and Nasal) Category 3 HAP Claims (Oral and Nasal) 2 Removal of warnings about difficulty swallowing, risks of IV injection, etc. 1. OxyContin label includes limited in vitro tamper data. 2. OxyContin label includes data from only one intranasal human abuse potential study. The data did not show statistically significant reduction compared with IR (i.e. original OxyContin or oxycodone HCL powder). 22

23 Product Positioning Early Adoption Targets vs OxyContin OP: 1 2 Will Not Take or Prescribe OxyContin OP Superior Abuse- Deterrence / Safety 3 Cannot Swallow or Difficulty Swallowing Tablets 23

24 Compelling Market Opportunity Chronic Pain Population Candidates for ER Opioids CPD Dysphagia Population 29 Million TRx per year 11 Million Patients in the U.S. Potential large revenue opportunity in the existing oral ER opioid market and dysphagia market Safety Ease of Swallowing Abuse-deterrence Effective pain relief Potential Best-in-Class Abuse-Deterrent Technology Potentially the Only Abuse- Deterrent ER Opioid Addressing Patients with CPD 24

25 Large ER/LA Opioid Market / Narrow Prescriber Focus ER/LA Opioids 29 Million TRx / $6BN U.S. Sales in M Chronic Pain & Dysphagia Segments 6.5M 5.5M 3.6M 1.0M 0.7M 1.0M Morphine Fentanyl Oxycodone Methadone Oxymorphone Buprenorphine Other Products Avinza, Kadian, MS Contin, Generic ERs Duragesic, Generic ERs OxyContin Generic LAs OPANA ER, Generic ERs Butrans Zohydro, Ultram, Nucynta, Exalgo, Generics ERs Sales $1.1BN $1.2BN $2.5BN $80M $470M $210M $310M Prescribers (Deciles 6-10) 10,000 8,500 3,500 Significant overlap in prescribing base Source: 2014 IMS Data. 25

26 Commercialization Strategy Preparing for potential Q U.S. commercial launch > Key commercial leadership in place > Launch planning process underway Launch with a sales team of approximately reps targeting the approximately 10,000-12,000 prescribers that write more that 50% of the branded ER opioids Clinically focused specialty sales force targeting institutions focused on CPD segment > Target palliative care, long-term care, hospital-based clinics Achieve broad Tier 3 payer coverage on Commercial plans and aggressively contract with Medicare and Medicaid > Co-pay reduction programs 26

27 Strong IP Portfolio Nine patents covering the DETERx technology platform Seven U.S. patents are expected to be listed in Orange Book at approval Additional three issued patents in Ex-US territories: Japan, Australia and Canada Issued patents are projected to expire in 2025 Eight additional patent applications in prosecution with the USPTO and International patent offices that could broaden and extend coverage beyond 2030 Proprietary manufacturing processes 27

28 Hatch-Waxman Litigation Status FTO opinion completed Collegium submitted Paragraph IV certification to Purdue for all 11 Orange Book listed patents Non infringement position Purdue filed suit in March currently invalid Orange Book listed patents 1 non-orange Book listed patent Collegium is extremely confident in its non-infringement position and intends to vigorously defend its position 28

29 Investment Highlights Differentiated Product Superior abuse-deterrence to OxyContin OP as demonstrated in clinical trials Opportunity to address large unmet medical need in patients with CPD Platform Technology Additional product opportunities Target 2 nd product candidate entering clinic Q Licensing opportunities (e.g. ADHD) Proprietary Manufacturing Process Proprietary processes support microsphere matrix formulation Commercially scalable Strong IP Portfolio Patents cover DETERx platform technology Issued patents up to 2023 and 2025 Additional patent applications extend coverage up to 2030 International Opportunities Near-term opportunity to expand in Canada, Japan, Europe and Australia Future opportunities in Latin America and Asia Business Development Identify and license, co-promote or acquire products being developed for pain indications and other complementary products 29