Receptos 8K Regulation FD Disclosure | RCPT 15 Sep 14

Phase 2 Results of the RADIANCE Trial: A Randomized,

Double-Blind, Placebo-Controlled Trial of Oral RPC1063

in Relapsing Multiple Sclerosis

Amit Bar-Or, MD

September 13, 2014

Cleveland Clinic, Mellen Center for MS Treatment and Research, Cleveland, OH, United States

NeuroRx Research, Montreal, QC, Canada

McGill University, Montreal, QC, Canada

San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Neurology, Milan, Italy

Montreal Neurological Institute, Department of Neurology and Neurosurgery, Montreal, QC, Canada

Receptos, Inc., Clinical Development, San Diego, CA, United States

Medical University of Lodz, Department of Neurology, Lodz, Poland

J Cohen

, DL Arnold

2,3

, G Comi

, A Bar-Or

, S Gujrathi

, JP Hartung

, A Olson

M Cravets

, PA Frohna

, K Selmaj

Exhibit 99.1

2014 Joint ACTRIMS-ECTRIMS Meeting

Boston, Massachusetts

13 September 2014

ACTRIMS-ECTRIMS 2014

Conflict of Interest Statements

Amit Bar-Or

financial

support

from

Biogen

Idec,

DioGenix,

Genentech,

GlaxoSmithKline, Guthy-Jackson Charitable Foundation, Medimmune,

Merck Serono, Novartis, Ono Pharmaceuticals, Receptos, Roche, Sanofi-

Aventis, and Teva Neuroscience

Cohen

financial

support

from

EMD

Serono,

Receptos,

Genzyme/Sanofi,

Innate Immunotherapeutics, Novartis

DL Arnold

financial

support

from

Receptos

Comi

financial

support

from

Receptos

K Selmaj

financial

support

from

Biogen

Idec,

Novartis,

Genzyme,

Synthon, Roche, Merck, Receptos, Teva

S Gujrathi,

Hartung,

Olson,

Cravets

Frohna

are

full-time

employees of Receptos

13 September 2014

ACTRIMS-ECTRIMS 2014

Introduction

Immunomodulation through the sphingosine 1-phosphate

(S1P) receptor family is a validated therapeutic approach

for the treatment of relapsing multiple sclerosis (RMS)

RPC1063 is a novel, oral S1P

1,5R

modulator in clinical

development for RMS by Receptos, Inc., which has only

been previously tested as single and multiple doses in

healthy volunteer subjects

The Phase 2 results presented today are from the

RADIANCE trial: an ongoing international, combined

Phase 2/3 trial of RPC1063 in adult patients with RMS

13 September 2014

ACTRIMS-ECTRIMS 2014

RADIANCE Patient Population: Key Inclusion Criteria

MS, as diagnosed by the revised McDonald criteria (2010)

Exhibiting

relapsing

clinical

course

consistent

with

RMS

and

history

of brain MRI lesions consistent with MS

Ages 18-55 years, inclusive

EDSS at Baseline between 0 and 5.0 points, inclusive

Meet 1 of the following RMS disease activity criteria:

At least 1 documented relapse within the last 12 months

At least 1 documented relapse within last 24 months plus at least 1 GdE

lesion

13 September 2014

ACTRIMS-ECTRIMS 2014

RADIANCE Phase 2 Trial Design

24-Week Placebo-Controlled

Treatment Period

30-day

Screening

Period

7-day

Titration

Period

Randomization

1 mg RPC1063

0.5 mg RPC1063

Placebo

Week

-4

Safety

Only

Baseline

Blinded Safety-

Extension

1 mg RPC1063

0.5 mg RPC1063

1 mg RPC1063

0.5 mg RPC1063

Yearly

2°

Efficacy Endpoints:

Number of GdE lesions at Week 24

Cumulative new or enlarging T2 lesions

Annualized Relapse Rate (ARR)

MRI

1°Endpoint: GdE Lesions

13 September 2014

ACTRIMS-ECTRIMS 2014

Patient Disposition

Randomized (N=258)

Placebo (N=88)

RPC1063 0.5 mg (N=87)

RPC1063 1 mg (N=83)

Discontinued

3 (3.4%)

Adverse event

Lost to follow-up

1 (1.1%)

Protocol violation

Consent withdrawn

2 (2.3%)

Discontinued

2 (2.3%)

Adverse event

Lost to follow-up

Protocol violation

1 (1.1%)

Consent withdrawn

1 (1.1%)

Discontinued

1 (1.2%)

Adverse event

Lost to follow-up

Protocol violation

Consent withdrawn

1 (1.1%)

Completed

85 (96.6%)

Completed

85 (97.7%)

Completed

82 (98.8%)

Very high rate of study compliance and completion

Similar completion rates across the treatment groups

13 September 2014

ACTRIMS-ECTRIMS 2014

Patient Demographics and RMS Disease History

Demographics

Placebo

(N=88)

RPC1063 0.5 mg

(N=87)

RPC1063 1 mg

(N=83)

Female –

n (%)

62 (70.5)

60 (69.0)

59 (71.1)

Age (yrs)

39.0 (8.7)

38.1 (9.2)

38.4 (9.8)

RMS Disease History, mean (SD)

Years since MS diagnosis

4.6 (5.1)

2.8 (5.0)

3.6 (4.4)

EDSS Score at Baseline

2.85 (1.27)

2.94 (1.29)

2.86 (1.21)

Relapses, last 12 months

1.3 (0.6)

1.5 (1.2)

1.3 (0.7)

Relapses, last 24 months

1.8 (1.0)

2.0 (1.8)

1.9 (1.1)

Gd-Enhancing lesions at Baseline

1.4 (3.4)

0.9 (1.4)

1.3 (2.8)

Gd-Enhancing lesion free at Baseline, n (%)

62 (70.5)

53 (60.9)

52 (62.7)

13 September 2014

ACTRIMS-ECTRIMS 2014

Primary Endpoint: Cumulative Number of Total GdE

Lesions From Weeks 12 to 24

p < 0.0001

86% decrease

11.1

1.5

Treatment Group

Placebo (N=88)

RPC1063 0.5 mg (N=87)

RPC1063 1 mg (N=83)

13 September 2014

ACTRIMS-ECTRIMS 2014

Secondary Endpoint (1):

Mean Number of GdE Lesions at Week 24

p < 0.0001

91% decrease

94% decrease

3.2

0.3

0.2

0.5

1.5

2.5

3.5

Treatment Group

Placebo (N=88)

RPC1063 0.5 mg (N=87)

RPC1063 1 mg (N=83)

13 September 2014

ACTRIMS-ECTRIMS 2014

Secondary Endpoint (2): Cumulative Number of New

or Enlarging T2 Lesions From Weeks 12 to 24

p < 0.0001

84% decrease

91% decrease

9.0

1.4

0.8

Treatment Group

Placebo (N=88)

RPC1063 0.5 mg (N=87)

RPC1063 1 mg (N=83)

13 September 2014

ACTRIMS-ECTRIMS 2014

Secondary Endpoint (3):

Annualized Relapse Rate

p = 0.271

31% decrease

p = 0.053

53% decrease

0.5

0.35

0.24

0.1

0.2

0.3

0.4

0.5

0.6

Treatment Group

Placebo (N=88)

RPC1063 0.5 mg (N=87)

RPC1063 1 mg (N=83)

13 September 2014

ACTRIMS-ECTRIMS 2014

Pharmacodynamic Effect of RPC1063:

Mean ±

SE Percent Change in Absolute Lymphocyte Counts

Study Week

-20

-40

-60

50%

60%

Program: E:\SASData\rpc1063\201a\csr\prog\g_lymph.sas

08JUN2014

13 September 2014

ACTRIMS-ECTRIMS 2014

SAFETY:

Treatment Emergent Adverse Events, n (%)

(All 24-week Double blind phase)

Placebo

(N=88)

RPC1063 0.5 mg

(N=87)

RPC1063 1 mg

(N=83)

Number of subjects experiencing

1 TEAE

52 (59.1)

56 (64.4)

47 (56.6)

1 moderate or severe TEAE

23 (26.1)

24 (27.6)

13 (15.7)

1 severe TEAE

1 (1.1)

2 (2.3)

1 (1.2)

1 possible, probable, or related TEAE

11 (12.5)

19 (21.8)

15 (18.1)

1 related TEAE

1 (1.1)

Preferred Term

Placebo

(N=88)

RPC1063 0.5 mg

(N=87)

RPC1063 1 mg

(N=83)

Nasopharyngitis

12 (13.6%)

11 (12.6%)

5 (6.0%)

Headache

8 (9.1%)

5 (5.7%)

3 (3.6%)

Urinary Tract Infection

2 (2.3%)

6 (6.9%)

2 (2.4%)

Most Common TEAEs in RPC1063-treated Patients

13 September 2014

ACTRIMS-ECTRIMS 2014

SAFETY: Serious TEAEs

Only 3 Serious TEAEs (reported as unrelated) during the study

*No change in EDSS post IV methyprednisolone treatment; did not qualify as a protocol-defined

relapse but was considered related to the patient’s MS

Preferred Term

Placebo

(N=88)

n (%)

RPC1063

0.5 mg

(N=87)

n (%)

RPC1063

1 mg

(N=83)

n (%)

Total number of Serious TEAEs

Number

of Subjects with

1 Serious TEAE

3 (3.4)

Optic

neuritis*

(1.1)

Somatoform autonomic

dysfunction

1 (1.1)

Uterine

cervical squamous metaplasia

1 (1.1)

13 September 2014

ACTRIMS-ECTRIMS 2014

SAFETY: Non-Serious TEAEs

No notable cardiac, pulmonary, ophthalmologic, infectious

or malignancy TEAEs were observed

3 TEAEs

elevated

liver

enzymes

(

ULN)

without

clinical signs/symptoms were observed

All were isolated ALT elevations (without AST or Bilirubin elevations)

None

5x ULN

Transient elevations despite continued treatment

Parameter

Placebo

(N=87)

RPC1063 0.5 mg

(N=85)

RPC1063 1 mg

(N=83)

Alanine Aminotransferase (ALT)

3x ULN, n (%)

2 (2.4%)

1 (1.2%)

Aspartate Aminotransferase (AST)

3x ULN, n (%)

13 September 2014

ACTRIMS-ECTRIMS 2014

Placebo

(N=88)

RPC1063 0.25 mg

(N=170)

Subjects with 2

Degree AVB, type I

2 (2.3%)

4 (2.4%)

Subjects

with

Sinus

Pause

(

seconds)

1 (1.1%)

1 (0.6%)

Day 1: Cardiac Findings on 24-hr Holter

SYSTEM ORGAN CLASS

Preferred Term

Placebo

(N=88)

RPC1063 0.5 mg

(N=87)

RPC1063 1 mg

(N=83)

CARDIAC DISORDERS

2 (2.3%)

1 (1.2%)

Angina Pectoris

1 (1.2%)

First Degree AV Block

1 (1.1%)

Palpitations

1 (1.1%)

2 (2.3%)

Overall Incidence of Cardiac TEAEs

Cardiac Safety Profile of RPC1063

13 September 2014

ACTRIMS-ECTRIMS 2014

Conclusions

Both doses of RPC1063 demonstrated large,

significant and consistent reductions of all MRI

measures of MS disease activity

The overall tolerability and safety profile of RPC1063,

including cardiac and hepatic safety results, suggests

a favorable risk-benefit profile in the treatment of RMS

These results support the ongoing Phase 3 portion of

the RADIANCE trial and the second Phase 3 trial of

RPC1063 vs. IFN

-1a in RMS (SUNBEAM)

Receptos (RCPT) Inactive 8-KRegulation FD Disclosure