UPDATED INVESTOR PRESENTATION June 2015

Transcription

1 UPDATED INVESTOR PRESENTATION June 2015

2 Forward-looking Statements All statements in this presentation other than those of historical fact, including statements regarding our clinical development plans for RPC1063 and RPC4046, our research and other development programs, our ability to undertake certain activities and accomplish certain goals, projected timelines for our research and development activities and possible regulatory approvals, if any, our expectations regarding the relative benefits of our product candidates versus competitive therapies, our expectations regarding the possibility of licensing or collaborating with third parties regarding our product candidates or research, and our expectations regarding the therapeutic and commercial potential of our product candidates, research, technologies and intellectual property, are forwardlooking statements. The words believe, may, will, estimate, continue, anticipate, design, intend, expect, potential and similar expressions, as well as the negative version of these words and similar expressions, are intended to identify forward-looking statements. Our forward-looking statements do not constitute guarantees of future performance, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those anticipated or implied in such statements. Our forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated as a result of various risks and uncertainties which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and risks inherent in the effort to build a business around such drugs. Although we believe our expectations are reasonable, we do not in any way guarantee future results, level of activity, performance or achievements. In addition, neither we nor any other person assumes responsibility for the accuracy and completeness of any forwardlooking statements. Our forward-looking statements in this presentation speak only as of the date this presentation is actually delivered by us in person. We assume no obligation or undertaking to update or revise any statements to reflect any changes in our expectations or any change in events, conditions or circumstances on which any such statement is based. Before you invest, you should read the prospectus and prospectus supplement, including the risk factors set forth or incorporated therein, and review disclosure that further describes risks and uncertainties relevant to us in additional detail in our filings with the Securities and Exchange Commission. You may get these documents for free by visiting EDGAR on the SEC web site at 2

3 Receptos Key Investment Highlights Ozanimod (formerly RPC1063): Promising new oral S1P1 receptor modulator addressing multibillion dollar opportunities in relapsing multiple sclerosis (RMS) and ulcerative colitis (UC), with potential follow-on indications RMS: Positive Phase 2 results; Phase 3 initiated under SPA UC: Positive Phase 2 results; currently planning Phase 3 in UC and Phase 2 in Crohn s Disease World-wide rights on ozanimod with expected patent coverage through 2029 plus potential for extension Building innovative pipeline to create long-term value Strong balance sheet (~$644 million as of 3/31/15)

4 Receptos is Developing Potentially High-Value, Differentiated Assets Programs and Indications Ozanimod (S1PR modulator) Research Preclinical Phase 1 Phase 2 Phase 3 Commercial Rights Worldwide Relapsing Multiple Sclerosis (RMS) Phase 3 Initiated with SPA Q Ulcerative Colitis (UC) Phase 3 to be Initiated 2015 Crohn s Disease (CD) Phase 2 to be Initiated 2015 Other Immune Indications RPC4046 (anti IL-13 antibody) AbbVie Option Eosinophilic Esophagitis (EoE) Phase 2 Initiated Q GLP-1R positive allosteric modulator Worldwide Type 2 Diabetes IND-enabling Studies to Commence 2015

5 Ozanimod Program Relapsing Multiple Sclerosis 5

6 RMS Market Valued at $19B and Growing Trend Toward Oral Therapy Transforming RMS Market Positioning of MS Treatment Options Beta Interferons / Copaxone Ozanimod* Tecfidera Gilenya Aubagio Daclizumab Lemtrada Ocrelizumab JC virus negative patients Tysabri Newly Diagnosed 1 st Line Treatment Cycling New therapies will be introduced 2 nd Line Treatment 1 st line failures Tolerability Issues Last Line Treatment Salvage treatment * Current company expectation for therapeutic applicability; subject to FDA approval

7 The RMS Market is Currently Valued at ~$19 Billion 2014 MS Market: ~$19B MARKET SIZE 12% CAGR ( ) Tecfidera 15% Aubagio 3% Tysabri 10% Interferons 36% CURRENT THERAPIES* Interferons ($6.8B) Copaxone ($4.2B) Tysabri ($2B) Gilenya ($2.5B) Tecfidera ($2.9B) Gilenya 13% Copaxone 22% UNMET NEEDS Safe and efficacious therapies More tolerable & convenient therapies Agents that halt or reverse damage Source: 3 rd Party Q Quarterly and Annual Reports

8 Oral Share of RMS IMS Scripts Increasing Gilenya and Tecfidera driving increases in oral market share gains 100.0% 90.0% 80.0% 70.0% 60.0% 50.0% 40.0% Gilenya Launch Q Aubagio Launch Q Tecfidera Launch Q Orals ABCRs 30.0% 20.0% 10.0% 0.0% Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q Note: RMS Therapies Include Avonex, Plegridy, Betaseron, Copaxone, Extavia, Rebif, Gilenya, Aubagio & Tecfidera ; Orals = Gilenya, Aubagio & Tecfidera ; ABCRs = Avonex, Plegridy, Betaseron, Extavia, Copaxone & Rebif Source: IMS Scripts NPA

9 Differentiation of Ozanimod is Based on Intrinsic Properties Which May Drive Favorable Clinical Outcomes Ozanimod Potential Best-in-Class S1P1 Receptor Modulator Potential for Improved Cardiac Profile (HR, QTc, BP) Potential for Reduced Hepatotoxicity Rapid Lymphocyte Recovery Intrinsic Properties Pharmaceutic Properties Selectivity Potency

10 Change in Mean Heart Rate (bpm) First Dose Effect on Mean Heart Rate Hours 1 to 6* 4 2 RPC mg Phase Hours After First Dose *Data estimated from Novartis material at MSBoston Data from separate studies; not head-to-head study comparisons Gilenya 0.5 mg* FREEDOMS Gilenya 0.5 mg* TRANSFORMS

11 Other S1PR Modulators have Demonstrated Hepatotoxicity in Clinical Trials 16% 14% 12% 12.0% 10% 10.0% 8% 8.0% 6% 4% 2% 2.4% 1.2% 0% 0.5mg 1mg Ozanimod * Data from separate studies; not head-to-head study comparisons 0.5mg 1.25mg Gilenya (Phase 3) (Phase 2) 5.0mg

12 Short Half-Life Confers Multiple Potential Safety Advantages Rapid Lymphocyte Recovery Through Short Half-Life Ozanimod Gilenya Siponimod Ponesimod T½ (hr) Time to Lymphocyte Recovery (70% Lymphocyte Reduction) 3 days 4-8 wks <1 wk 3-6 days Gilenya has a prolonged half-life and PD effect relative to ozanimod and other S1PR modulators Rapid lymphocyte recovery may provide numerous advantages: Flexibility in retreatment with other immunosuppressive/immunomodulatory agents Ability to address RMS flare Ability to treat opportunistic infections, address other treatment-related complications Advantages to address pregnancy concerns

13 Status of Ozanimod Program in RMS Phase 2 RADIANCE trial results (both dose groups): Met primary efficacy endpoint with statistical significance (p<0.0001) after 24 weeks Met key secondary MRI endpoints (p<0.0001) with favorable trend on ARR Safety data supportive of differentiated product profile (overall AEs, CV, hepatic) Phase 3 program status: First trial (RADIANCE) initiated December 2013 Primary objective: Assess superiority against Avonex in reducing ARR after 24 months (N=1200) Completed enrollment in March 2015 Second trial (SUNBEAM) initiated December 2014 Primary objective: Assess superiority against Avonex in reducing ARR after 12 months (N=1200) SPA agreement with FDA covering both Phase 3 trials EMA has indicated that with minor adjustments, the proposed development program could be sufficient to support an MAA in the EU

14 Cumulative Number of GdE Lesions Primary Endpoint: Cumulative Number of Total GdE Lesions From Weeks 12 to % decrease 86% decrease p < p < Treatment Group Placebo (N=88) RPC mg (N=87) RPC mg (N=83)

15 Adjusted Annualized Relapse Rate Annualized Relapse Rate % decrease p = % decrease p = Treatment Group Placebo (N=88) RPC mg (N=87) RPC mg (N=83)

16 Precedents Exist for Avoidance / Removal of Class Labeling Diuretics for Hypertension FDA approved most recent drug without a black box despite acknowledged class risk for hyperkalemia Inspra (Pfizer) approved without a black box Midamor (Merck) and Dyrenium (WellSpring) had previously been approved, both with black box warnings Endothelin Receptor Antagonists for PAH FDA re-evaluated black box for liver toxicity Tracleer (Actelion) approved with black box warning Letairis (Gilead) approved with black box warnings, then removed after commercial experience, follow-up studies Opsumit (Actelion) subsequently approved without black box warning

17 OZANIMOD (RPC1063) IBD Indication 17

18 TOUCHSTONE Phase 2 Trial (Ozanimod in Ulcerative Colitis) Summary of Top-line Results Phase 2 randomized, double-blind, placebo controlled study in moderate-to-severely active UC 199 patients randomized 1:1:1 across two ozanimod dose groups and placebo 57 sites in 13 countries Efficacy: Met primary efficacy and all secondary endpoints for patients on 1 mg dose after 8 weeks of treatment (induction) Met primary efficacy and all secondary endpoints for patients on 1 mg dose after 32 weeks of treatment (maintenance) Safety: Consistent with favorable profile observed in Phase 2 trial in RMS Incidence of adverse events across active treatment groups and placebo appeared similar No concerning signals in the AEs of special interest Changes in heart rate generally modest during first 6 hours after administration Rates of liver enzyme elevations observed were low and consistent with the earlier Phase 2 trial in RMS

19 Percentage of Subjects in Remission Remission Rates: Ozanimod Data Compared to Recently Approved Treatments Note: Data from Separate Studies; Not Head-to-Head Study Comparisons 20% 16% Δ = 11.4% Δ = 7.2% Δ = 11.5% 17.8% 16.5% 16.9% Δ = 10.8% 16.4% 12% 9.3% 8% 6.4% 5.4% 6.2% 4% 0% PBO 200/100 mg PBO 160/80mg PBO 300 mg N=251 N=253 N=246 N=248 N=149 N=225 Simponi Humira Entyvio RPC1063 Phase 3 Phase 3 Phase 3 PBO 1 mg N=65 N=67 Phase 2 Note: treatment differences are adjusted for stratification factors

20 Ozanimod May be Positioned in Early Lines Against Immunosuppressants as well as Last Line Against Anti-TNFs Positioning of UC Treatment Options 5-Aminosalicylates Immunosuppressants and Corticosteroids Remicade, Humira Entyvio (vedolizumab) Ozanimod* Tysabri 1 st Line Treatment 2 nd Line Treatment 3 rd Line Treatment Last Line Treatment * Positioning based on Receptos sponsored market research. Respondents were provided with a product profile for ozanimod and vedolizumab that assumed comparable efficacy improved to anti-tnfs with specified improvements in safety profile. Actual clinical results for late stage trial and the FDA marketing label for ozanimod, if approved, and vedolizumab, approved in May 2014, may be different than the profile that was assumed for market research; pricing and reimbursement were assumed to be equal among ozanimod and all other UC treatments Source: Kantar 2013, US Gastroenterologist Quantitative Survey (N = 101)

21 IBD Market Currently $12B, Poised to Grow Rapidly Advent of oral therapy may drive accelerating growth 3-4x Moderate-to-Severe Patient Prevalence in IBD as Compared to RMS 3,000 2,000 1,000 0 Prevalence ( 000), Major Markets 1, RMS IBD RMS IBD RMS IBD US Ex-US Total Price Differential Between RMS and IBD Diminished with Entyvio Launch 75,000 60,000 45,000 30,000 15,000 Highest Annual Drug Pricing $66,000 $45,000 0 RMS Pricing IBD Pricing Source: Datamonitor Epidemiology Forecast December Ex-US represents EU5 + Japan. IBD market epidemiology forecasts represent moderate-to-severe patient populations.

22 RPC4046 Eosinophilic Esophagitis 22

23 Eosiniphilic Esophagitis (EoE) is an Orphan Disease with Significant Unmet Need Endoscopic Image Disease Overview EoE is a chronic, immune-mediated atopic GI-related disease ~160K patients in the US, ~145K in EU with growing prevalence Disease presents in children and adults and is marked by eosinophilic infiltration and structural changes in esophagus No FDA approved drugs for treatment; standard-of-care swallowed steroids are not effective in controlling disease Histologic Image Scientific Rationale for targeting anti-il13 Preclinical and human tissue studies point to a central role for IL-13 cytokine driving disease pathogenesis RPC4046 targets anti-inflammatory and anti-fibrotic pathways Anti-IL-13 validated in asthma with lebrikizumab (Genentech) Biomarker/diagnostic strategy exists for selecting responders

24 Receptos Continues to Deliver Against Milestones Timing RMS top-line Phase 2 data 6/14 RMS detailed Phase 2 data (MS Boston podium presentation) 9/14 Initiation of EoE Phase 2 trial 10/14 UC top-line Phase 2 data 10/14 UC detailed Phase 2 data 1H 2015 UC Phase 2 exploratory maintenance data 2H 2015 EoE Phase 2 trial results 1H 2016 Crohn s disease Phase 2 results 2H 2016 MS Phase 3 results 1H 2017 Receptos has established a track record of strong execution Status

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