Safe Harbor Statement

Size: px
Start display at page:

Download "Safe Harbor Statement"

Transcription

1

2 Safe Harbor Statement These slides contain forward-looking statements based on estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. Forward-looking statements involve risks and uncertainties, including, but not limited to, economic, competitive, governmental and technological factors outside of our control, that may cause our business, industry, strategy or actual results to differ materially from the forward-looking statements. These risks and uncertainties may include those discussed under the heading Risk Factors in our most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commission, and other factors which may not be known to us. Any forwardlooking statement speaks only as of its date. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. Neurocrine Biosciences files Annual Reports, Quarterly Reports and other documents with the Securities and Exchange Commission. You should read the documents we have filed with the Securities and Exchange Commission for more complete information about Neurocrine Biosciences. You may get these documents for free by visiting EDGAR on the Securities and Exchange Commission web site at or by visiting the Neurocrine Biosciences website at 2

3 Delivering Significant Shareholder Value in 2015 Elagolix Positive top-line data on first Phase III trial for endometriosis disclosed Jan 2015 Top-line data on second Phase III trial for endometriosis late 2015 Report on Phase IIb study for uterine fibroids in mid 2015 VMAT2 (NBI-98854) Wholly-owned; will commercialize in US Fast track and breakthrough designation in Tardive dyskinesia Top line Phase III data in Tardive dyskinesia second half 2015 Top line phase Ib data in Tourette Syndrome second half 2015 CRF Antagonist (NBI-77860) FDA Orphan Designation granted for Congenital Adrenal Hyperplasia (CAH) Phase Ib and IIa trials expected in second half 2015 New IND for neurological disorder expected to be filed in Q Phase I clinical data anticipated in

4 Elagolix A Pipeline Within a Program Elagolix Primary Targeted Disease States Endometriosis Phase III Uterine Fibroids Phase II Additional Disease States PCOS PMDD Adenomyosis ART Menorrhagia Primary Dysmenorrhea 4

5 Violet Petal Study Positive Primary Endpoint at Month 3» 150mg qd positive for both Dysmenorrhea and Non-menstrual pelvic pain p<0.001» 200mg bid positive for both Dysmenorrhea and Non-menstrual pelvic pain p<0.001 Duration of Effect at Month 6» 150mg qd positive for both Dysmenorrhea and Non-menstrual pelvic pain p<0.001» 200mg bid positive for both Dysmenorrhea and Non-menstrual pelvic pain p<0.001 Safety Profile Consistent with Phase 2 Program Bone Mineral Density (BMD) of 150mg dose consistent with 603 (Petal) Study Data has High Internal Integrity 5

6 Pipeline within a Program VMAT2 Inhibition: Potential for Multiple Conditions Tourette Syndrome Prevalence: 400k Currently approved therapies: Orap (pimozide), Haldol (haloperidol) both with black box warnings Tardive Dyskinesia* Schizophrenia Prevalence: 500k Currently approved therapies: None VMAT2 Inhibitor Pipeline Prevalence: 2.2MM Currently approved therapies: Dopamine Receptor Antagonists IMS Health, Kantar Health, Datamonitor *Fast Track Status Received Jan 2012 *Breakthrough Designation Received Oct

7 NBI Kinect 3 Phase III Study AIMS assessments at screening and every two weeks thereafter Placebo (n=80) Screening 40mg (n=80) Blinded 1-Year Safety Extension: Placebo randomized to 40mg or 80mg of NBI mg (n=80) Week 6 Primary Endpoint 1-Year Safety Study (n=150) 7

8 NBI Tourette Phase 1b Design Adolescents y.o. n=18 High Dose PK/PD Assessment Independent review of safety and PK data Mid Dose Low Dose Children 6 11 y.o. n=18 Mid Dose High Dose Low Dose Week

9 Classic Congenital Adrenal Hyperplasia (CAH) A group of autosomal recessive disorders characterized by impaired cortisol synthesis Worldwide incidence of 1 in 13,000 live births.» Approximately 30,000 in the USA Caused by a defect or mutation in 21 a-hydroxylase leading to a marked decrease in cortisol and a dramatic increased androgen production» The lack of cortisol feedback at the level of the pituitary causes increased ACTH release» The lack of cortisol feedback at the hypothalamus increases CRF release which compounds the increased ACTH released from the pituitary 9

10 Classic Congenital Adrenal Hyperplasia (CAH) Disease with Poor Treatment Alternatives Classic CAH is a lifelong disease that presents at birth with severe consequences Morbidity associated with CAH is related primarily to the effects of an absence of glucocorticoids and an excess of androgens» Salt wasting» Virilization of females including need for surgery, infertility, precocious puberty, menstrual irregularity, short stature, and hirsutism Current treatment is high doses hydrocortisone, prednisolone or dexamethasone» Bone loss and growth impairment» Cushing s syndrome» Metabolic Syndrome 10

11 Hypothalamic-Pituitary-Adrenal Axis (Normal) CRF ACTH Cortisol Androgens CORTISOL NEGATIVE FEEDBACK LOOPS CONTROL CRF, ACTH AND ANDROGEN LEVELS 11

12 Hypothalamic-Pituitary-Adrenal Axis (CAH) CRF ACTH Cortisol Androgens NO CORTISOL NEGATIVE FEEDBACK LOOPS UNCONTROLLED CRF, ACTH AND ANDROGEN LEVELS 12

13 Specific Enzyme Inefficiencies are Associated with types of Congenital Adrenal Hyperplasia Cholesterol ACTH CRF 95% of CAH Pregnenolone 17-OH Pregnenolone Progesterone 17-OH Progesterone 21-a-Hydroxylase Deoxycorticosterone Deoxycortisol 11-b-Hydroxylase Corticosterone Cortisol DHEA Androstenedione Testosterone Estrogens 13

14 Specific Enzyme Inefficiencies are Associated with types of Congenital Adrenal Hyperplasia Hypothalamus Cholesterol ACTH CRF 95% of CAH Pregnenolone 17-OH Pregnenolone Progesterone 17-OH Progesterone 21-a-Hydroxylase Deoxycorticosterone Deoxycortisol 11-b-Hydroxylase Corticosterone Cortisol DHEA Androstenedione Testosterone Estrogens 14

15 NBI for Classic Congenital Adrenal Hyperplasia Initial single dose pilot study of NBI CRF 1 antagonist in eight patients with CAH.» Demonstrated a robust decrease in ACTH and 17- OHP» Single site study of eight refractory adult females» three separate overnight visits consisting of bedtime dosing with placebo or one of two active doses of NBI

16 % Change from Predose Effect of a Single Dose NBI on Plasma ACTH and 17-hydroxy-Progesterone (17-OHP) % Change from Predose Pilot Single Dose Exploratory Study - % Change from Pre-dose ACTH : All Subjects (N=8) 17-OHP : All Subjects (N=8) Placebo 300 mg 600 mg Placebo 300 mg 600 mg Morning Window 6 10 AM Morning Window 6 10 AM 16

17 NBI for Classic Congenital Adrenal Hyperplasia Second study initiated (1401 study)» 15 patient open-label, sequential cohort, single ascending dose pharmacokinetic/pharmacodynamic study» assessing three doses of NBI at bedtime» adolescent females with classic CAH» three equal cohorts» Biomarker measurements include ACTH, 17-OHP, androgen, and cortisol levels collected the morning after dosing.» Results later in 2015 Third study to initiate shortly 17

18 Delivering Significant Shareholder Value in 2015 Elagolix Positive top-line data on first Phase III trial for endometriosis disclosed Jan 2015 Top-line data on second Phase III trial for endometriosis late 2015 Report on Phase IIb study for uterine fibroids in mid 2015 VMAT2 (NBI-98854) Wholly-owned; will commercialize in US Fast track and breakthrough designation in Tardive dyskinesia Top line Phase III data in Tardive dyskinesia second half 2015 Top line phase Ib data in Tourette Syndrome second half 2015 CRF Antagonist (NBI-77860) FDA Orphan Designation granted for Congenital Adrenal Hyperplasia (CAH) Phase 1b and 2a trials expected in second half 2015 New IND for neurological disorder expected to be filed in Q Phase I clinical data anticipated in

19 Financial Highlights Year Ended December 31, 2014 Year Ended December 31, 2013 Revenues and other Income $ 3.9 million $ 6.5 million Operating Expenses $ 64.4 million $ 52.6 million Net Loss $ (60.5 million) $ (46.1 million) Cash, Investments, Receivables $ million $ million Net Proceeds from February 2015 capital raise of approximately $270 million 19

Neurocrine Biosciences, Inc. THE NEUROENDOCRINE COMPANY

Neurocrine Biosciences, Inc. THE NEUROENDOCRINE COMPANY Neurocrine Biosciences, Inc. THE NEUROENDOCRINE COMPANY Safe Harbor Statement In addition to historical facts, this presentation contains forward-looking statements that involve a number of risks and uncertainties.

More information

SCHOOL OF MEDICINE AND HEALTH SCIENCES DIVISION OF BASIC MEDICAL SCIENCES DISCIPLINE OF BIOCHEMISTRY & MOLECULAR BIOLOGY

SCHOOL OF MEDICINE AND HEALTH SCIENCES DIVISION OF BASIC MEDICAL SCIENCES DISCIPLINE OF BIOCHEMISTRY & MOLECULAR BIOLOGY 1 SCHOOL OF MEDICINE AND HEALTH SCIENCES DIVISION OF BASIC MEDICAL SCIENCES DISCIPLINE OF BIOCHEMISTRY & MOLECULAR BIOLOGY PBL SEMINAR: SEX HORMONES PART 1 An Overview What are steroid hormones? Steroid

More information

ULTIMATE BEAUTY OF BIOCHEMISTRY. Dr. Veena Bhaskar S Gowda Dept of Biochemistry 30 th Nov 2017

ULTIMATE BEAUTY OF BIOCHEMISTRY. Dr. Veena Bhaskar S Gowda Dept of Biochemistry 30 th Nov 2017 ULTIMATE BEAUTY OF BIOCHEMISTRY Dr. Veena Bhaskar S Gowda Dept of Biochemistry 30 th Nov 2017 SUSPECTED CASE OF CUSHING S SYNDROME Clinical features Moon face Obesity Hypertension Hunch back Abdominal

More information

Supernus Pharmaceuticals

Supernus Pharmaceuticals Supernus Pharmaceuticals Jefferies 2016 Healthcare Conference May 2016 1 Safe Harbor Statement This presentation and other matters discussed today or answers that may be given to questions asked include

More information

SEX STERIOD HORMONES I: An Overview. University of PNG School of Medicine & Health Sciences Division of Basic Medical Sciences PBL MBBS III VJ Temple

SEX STERIOD HORMONES I: An Overview. University of PNG School of Medicine & Health Sciences Division of Basic Medical Sciences PBL MBBS III VJ Temple SEX STERIOD HORMONES I: An Overview University of PNG School of Medicine & Health Sciences Division of Basic Medical Sciences PBL MBBS III VJ Temple 1 What are the Steroid hormones? Hormones synthesized

More information

Diagnostics for the early detection and prevention of colon cancer. Publication of DeeP-C Study Data in New England Journal of Medicine March 2014

Diagnostics for the early detection and prevention of colon cancer. Publication of DeeP-C Study Data in New England Journal of Medicine March 2014 Diagnostics for the early detection and prevention of colon cancer Publication of DeeP-C Study Data in New England Journal of Medicine March 2014 Safe Harbor Statement Certain statements made in this presentation

More information

ObsEva Reports Third Quarter 2018 Financial Results and Provides Business Update

ObsEva Reports Third Quarter 2018 Financial Results and Provides Business Update ObsEva Reports Third Quarter 2018 Financial Results and Provides Business Update IMPLANT 4 trial of nolasiban in IVF starting in Q4 2018, European MAA filing expected late 2019 24-week data from Phase

More information

May 10, 2016 Q & Business Update

May 10, 2016 Q & Business Update May 10, 2016 Q1 2016 Thank Financial you Results & Business Update Q1 2016 Financial Results Prepared Remarks Q1 Update Tom Hughes, Ph.D., Chief Executive Officer Clinical Update Dennis Kim, M.D., Chief

More information

Developing clinical stage small molecule therapeutics to treat hormonal and reproductive system disorders

Developing clinical stage small molecule therapeutics to treat hormonal and reproductive system disorders Developing clinical stage small molecule therapeutics to treat hormonal and reproductive system disorders Repros Disclaimer Any statements made by the Company that are not historical facts contained in

More information

Neurocrine Biosciences, Inc. THE NEUROENDROCRINE COMPANY

Neurocrine Biosciences, Inc. THE NEUROENDROCRINE COMPANY Neurocrine Biosciences, Inc. THE NEUROENDROCRINE COMPANY Safe Harbor Statement In addition to historical facts, these slides contain forward-looking statements that involve a number of risks and uncertainties.

More information

Jefferies Healthcare Conference June 8, 2017

Jefferies Healthcare Conference June 8, 2017 Jefferies Healthcare Conference June 8, 2017 1 Forward-Looking Statements and Non-GAAP Financial Information Some statements in this presentation may be forward-looking statements for purposes of the Private

More information

REPROS THERAPEUTICS. Dedicated to Treating Male and Female Reproductive Disorders. Corporate Presentation. August 2017

REPROS THERAPEUTICS. Dedicated to Treating Male and Female Reproductive Disorders. Corporate Presentation. August 2017 REPROS THERAPEUTICS Dedicated to Treating Male and Female Reproductive Disorders Corporate Presentation August 2017 Safe Harbor Any statements made by the Company that are not historical facts contained

More information

REPROS THERAPEUTICS. Dedicated to Treating Male and Female Reproductive Disorders. Corporate Presentation. September 2017

REPROS THERAPEUTICS. Dedicated to Treating Male and Female Reproductive Disorders. Corporate Presentation. September 2017 REPROS THERAPEUTICS Dedicated to Treating Male and Female Reproductive Disorders Corporate Presentation September 2017 Safe Harbor Any statements made by the Company that are not historical facts contained

More information

PATENCY-1 Top-Line Results

PATENCY-1 Top-Line Results PATENCY-1 Top-Line Results December 13, 2016 2016 Proteon Therapeutics, Inc. Cautionary Note Regarding Forward-Looking Statements This presentation contains statements that are, or may be deemed to be,

More information

ObsEva Reports Third Quarter 2017 Financial Results and Provides Business Update

ObsEva Reports Third Quarter 2017 Financial Results and Provides Business Update ObsEva Reports Third Quarter 2017 Financial Results and Provides Business Update - All Three Development Compounds Progressing with Key Clinical Milestones Over the Next 12 Months - Geneva, Switzerland

More information

ObsEva Reports First Quarter 2018 Financial Results and Provides Business Update

ObsEva Reports First Quarter 2018 Financial Results and Provides Business Update ObsEva Reports First Quarter 2018 Financial Results and Provides Business Update -Key First Quarter 2018 Clinical Milestones Achieved Patient randomization completed in Phase 2b EDELWEISS trial of OBE2109

More information

Small-Cap Research. Neurocrine Biosciences, Inc. (NBIX - Nasdaq) NBIX: NDA for Valbenazine in Tardive Dyskinesia to be Filed in 2016 SUMMARY DATA

Small-Cap Research. Neurocrine Biosciences, Inc. (NBIX - Nasdaq) NBIX: NDA for Valbenazine in Tardive Dyskinesia to be Filed in 2016 SUMMARY DATA Small-Cap Research May 9, 2016 David Bautz, PhD 312-265-9471 dbautz@zacks.com scr.zacks.com 10 S. Riverside Plaza, Suite 1600, Chicago, IL 60606 Neurocrine Biosciences, Inc. (NBIX - Nasdaq) NBIX: NDA for

More information

May 2018 August 9, 2018

May 2018 August 9, 2018 Presentation Millendo Therapeutics to Lorem and Ipsum OvaScience DolorMerger May 2018 August 9, 2018 Disclaimer This presentation includes forward-looking statements. All statements contained in this presentation

More information

Diurnal Group plc. Analyst Day 11 December Date of Preparation: December 2018 Code: CORP-GB-0023

Diurnal Group plc. Analyst Day 11 December Date of Preparation: December 2018 Code: CORP-GB-0023 Diurnal Group plc Analyst Day 11 December 2018 Date of Preparation: December 2018 Code: CORP-GB-0023 1 Analyst Day: Challenges and Current Treatment Options for Congenital Adrenal Hyperplasia Prof John

More information

Supernus Pharmaceuticals

Supernus Pharmaceuticals Supernus Pharmaceuticals Investor Presentation March 2017 1 Safe Harbor Statement This presentation and other matters discussed today or answers that may be given to questions asked include forward-looking

More information

AMBIGUOUS GENITALIA & CONGENITAL ADRENALHYPERPLASIA

AMBIGUOUS GENITALIA & CONGENITAL ADRENALHYPERPLASIA AMBIGUOUS GENITALIA & CONGENITAL ADRENALHYPERPLASIA BY Dr Numair Ali sheikh FCPS PGT I Department Of Pediatrics BBH RWP AMBIGUOUS GENITALIA Children born with ambiguous genitalia may be subdivided in to

More information

PRENATAL TREATMENT AND FERTILITY OF FEMALE PATIENTS WITH CONGENITAL ADRENAL HYPERPLASIA

PRENATAL TREATMENT AND FERTILITY OF FEMALE PATIENTS WITH CONGENITAL ADRENAL HYPERPLASIA PRENATAL TREATMENT AND FERTILITY OF FEMALE PATIENTS WITH CONGENITAL ADRENAL HYPERPLASIA Nguyen Ngoc Khanh, Vu Chi Dung et al Vietnam Children s Hospital (VCH) Hanoi, Vietnam Outline Intruduction Prenatal

More information

Transitions For the CAH Patient

Transitions For the CAH Patient Transitions For the CAH Patient Richard J. Auchus, M.D., Ph.D. Division of Metabolism, Endocrinology & Diabetes Department of Internal Medicine DSD Program University of Michigan Disclosures Contracted

More information

Committed to Transforming the Treatment Paradigm for Migraine Prevention

Committed to Transforming the Treatment Paradigm for Migraine Prevention June 14, 2018 Committed to Transforming the Treatment Paradigm for Migraine Prevention September 6, 2018 Forward-Looking Statements This presentation and the accompanying commentary contains certain forward-looking

More information

Diagnostics for the early detection and prevention of colon cancer. Fourth-Quarter 2014 Earnings Call February 24, 2015

Diagnostics for the early detection and prevention of colon cancer. Fourth-Quarter 2014 Earnings Call February 24, 2015 Diagnostics for the early detection and prevention of colon cancer Fourth-Quarter 2014 Earnings Call February 24, 2015 Safe Harbor Statement Certain statements made in this presentation contain forward-looking

More information

Adrenal Insufficiency During Pregnancy

Adrenal Insufficiency During Pregnancy Disclosures Adrenal Insufficiency During Pregnancy Research funding from Diurnal Limited via NIH CRADA mechanism Deborah P. Merke, M.D., M.S. Bethesda, MD Outline Primary Adrenal Insufficiency Physiological

More information

ComprehensivePLUS Hormone Profile with hgh

ComprehensivePLUS Hormone Profile with hgh OLEBound400: 801 SW 16th St Suite 126 Renton WA 98057 425.271.8689 425.271.8674 (Fax) ComprehensivePLUS Hormone Profile with hgh Doctor ID Patient Name 6206 Doe, Jane Age Sex Date of Birth 44 F Date Collected

More information

Committed to Transforming the Treatment Paradigm for Migraine Prevention

Committed to Transforming the Treatment Paradigm for Migraine Prevention Committed to Transforming the Treatment Paradigm for Migraine Prevention 36th Annual J.P. Morgan Healthcare Conference January 8, 2018 Forward-Looking Statements This presentation and the accompanying

More information

Investor Presentation March 2015

Investor Presentation March 2015 Investor Presentation March 2015 1 Safe Harbor Statement This presentation and other matters discussed today or answers that may be given to questions asked include forward-looking statements within the

More information

Building a Fully Integrated Biopharmaceutical Company. June 2014

Building a Fully Integrated Biopharmaceutical Company. June 2014 Building a Fully Integrated Biopharmaceutical Company June 2014 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation

More information

PROMISE 1 Top-Line Data Results. June 27, 2017

PROMISE 1 Top-Line Data Results. June 27, 2017 PROMISE 1 Top-Line Data Results dd June 27, 2017 Forward Looking Statements This presentation and the accompanying commentary contains certain forward-looking statements within the meaning of Section 27A

More information

Merck Announces FDA Approval of KEYTRUDA. Provided to Investors as a Reference

Merck Announces FDA Approval of KEYTRUDA. Provided to Investors as a Reference Merck Announces FDA Approval of KEYTRUDA Provided to Investors as a Reference Forward-Looking Statement This presentation includes forward-looking statements within the meaning of the safe harbor provisions

More information

ADRENAL GLANDS HORMONES

ADRENAL GLANDS HORMONES ADRNAL GLANDS HORMONS Glands Cortex 80% mesoderm Mineralococorticoids Glucocorticoids (phenylethanolamine N- methyl transferase) A Sex Hormones Catecholamines Medulla 20% PNMT, N neuroectoderm N PNMT V

More information

Medical management of Intersex disorders. Dr. Abdulmoein Al-Agha, Ass. Professor & Consultant Pediatric Endocrinologist KAAUH, Jeddah

Medical management of Intersex disorders. Dr. Abdulmoein Al-Agha, Ass. Professor & Consultant Pediatric Endocrinologist KAAUH, Jeddah Medical management of Intersex disorders Dr. Abdulmoein Al-Agha, Ass. Professor & Consultant Pediatric Endocrinologist KAAUH, Jeddah Is it a boy or a girl? The birth of an intersex infant is often viewed

More information

New Ideas. Better Medicines. Third Quarter Financial Results Conference Call

New Ideas. Better Medicines. Third Quarter Financial Results Conference Call New Ideas. Better Medicines. Third Quarter 2018 Financial Results Conference Call Forward-Looking Statements 2 This presentation contains forward-looking statements that involve substantial risks and uncertainties.

More information

Creating a Leading Global HBV Therapeutics Company. ARB-1467 Update Call December 12, 2016

Creating a Leading Global HBV Therapeutics Company. ARB-1467 Update Call December 12, 2016 Creating a Leading Global HBV Therapeutics Company ARB-1467 Update Call December 12, 2016 NASDAQ: ABUS www.arbutusbio.com Forward Looking Statements This presentation contains forward-looking statements

More information

PROMISE 2 Top-Line Data Results January 8, 2018

PROMISE 2 Top-Line Data Results January 8, 2018 PROMISE 2 Top-Line Data Results January 8, 2018 Forward-Looking Statements This presentation and the accompanying commentary contains certain forward-looking statements within the meaning of Section 27A

More information

International Journal of Health Sciences and Research ISSN:

International Journal of Health Sciences and Research   ISSN: International Journal of Health Sciences and Research www.ijhsr.org ISSN: 2249-9571 Original Research Article Congenital Adrenal Hyperplasia in Saudi Arabia: The Biochemical Characteristics Nasir A. M.

More information

Aldosterone synthase inhibitors. John McMurray BHF Cardiovascular Research Centre University of Glasgow

Aldosterone synthase inhibitors. John McMurray BHF Cardiovascular Research Centre University of Glasgow Aldosterone synthase inhibitors John McMurray BHF Cardiovascular Research Centre University of Glasgow Inhibition of aldosterone synthesis is hypothesized to be of benefit to patients with cardiovascular

More information

Reproductive DHEA-S Analyte Information

Reproductive DHEA-S Analyte Information Reproductive DHEA-S Analyte Information - 1 - DHEA-S Introduction DHEA-S, DHEA sulfate or dehydroepiandrosterone sulfate, it is a metabolite of dehydroepiandrosterone (DHEA) resulting from the addition

More information

REATA PHARMACEUTICALS, INC. ANNOUNCES SECOND QUARTER 2018 FINANCIAL RESULTS AND AN UPDATE ON DEVELOPMENT PROGRAMS

REATA PHARMACEUTICALS, INC. ANNOUNCES SECOND QUARTER 2018 FINANCIAL RESULTS AND AN UPDATE ON DEVELOPMENT PROGRAMS REATA PHARMACEUTICALS, INC. ANNOUNCES SECOND QUARTER 2018 FINANCIAL RESULTS AND AN UPDATE ON DEVELOPMENT PROGRAMS IRVING, Texas August 8, 2018 Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage

More information

USPSTF Draft Recommendations Investor Call. October 6, 2015

USPSTF Draft Recommendations Investor Call. October 6, 2015 USPSTF Draft Recommendations Investor Call October 6, 2015 v Safe Harbor Statement Certain statements made in this presentation contain forward-looking statements within the meaning of Section 27A of the

More information

SAVARA CORPORATE PRESENTATION (NASDAQ: SVRA) NOVEMBER 2018

SAVARA CORPORATE PRESENTATION (NASDAQ: SVRA) NOVEMBER 2018 SAVARA CORPORATE PRESENTATION (NASDAQ: SVRA) NOVEMBER 2018 SAFE HARBOR STATEMENT Savara Inc. ( Savara or the Company ) cautions you that statements in this presentation that are not a description of historical

More information

Q1 Results 2018 Webcast presentation 26 April 2018

Q1 Results 2018 Webcast presentation 26 April 2018 Q1 Results 2018 Webcast presentation 26 April 2018 Disclaimer This presentation contains forward-looking statements, including (without limitation) statements concerning the progress of our clinical pipeline,

More information

Jefferies Healthcare Conference. June 6, 2018

Jefferies Healthcare Conference. June 6, 2018 Jefferies Healthcare Conference June 6, 2018 Forward-Looking Statements and Non-GAAP Financial Information Some statements in this presentation may be forward-looking statements for purposes of the Private

More information

Aerpio Reports Fourth Quarter and Full Year 2017 Financial Results and Provides Business Update

Aerpio Reports Fourth Quarter and Full Year 2017 Financial Results and Provides Business Update Aerpio Reports Fourth Quarter and Full Year 2017 Financial Results and Provides Business Update TIME-2b Clinical Trial of AKB-9778 in Patients with Diabetic Retinopathy Remains on Track Conference Call

More information

Ganaxolone as a Treatment for Drug-Resistant Epilepsy in Children

Ganaxolone as a Treatment for Drug-Resistant Epilepsy in Children Ganaxolone as a Treatment for Drug-Resistant Epilepsy in Children ANTIEPILEPTIC DRUG and DEVICE TRIALS XIII May 13-15, 2015 Turnberry Isle Miami Hotel Gail M. Farfel, PhD Chief Development & Regulatory

More information

Corporate Presentation August 6, 2015

Corporate Presentation August 6, 2015 Corporate Presentation August 6, 2015 Creating the Next Generation of CNS Drugs Forward-Looking Statement This presentation contains forward-looking statements. These statements relate to future events

More information

Laura Stewart, MD, FRCPC Clinical Associate Professor Division of Pediatric Endocrinology University of British Columbia

Laura Stewart, MD, FRCPC Clinical Associate Professor Division of Pediatric Endocrinology University of British Columbia Precocious Puberty Laura Stewart, MD, FRCPC Clinical Associate Professor Division of Pediatric Endocrinology University of British Columbia Faculty Disclosure Faculty: Laura Stewart No relationships with

More information

Dr. Nermine Salah El-Din Prof of Pediatrics

Dr. Nermine Salah El-Din Prof of Pediatrics Dr. Nermine Salah El-Din Prof of Pediatrics Diabetes Endocrine Metabolism Pediatric Unit (DEMPU) Children Hospital, Faculty of Medicine Cairo University Congenital adrenal hyperplasia is a common inherited

More information

February 23, Q4 and Year-End 2016 Financial Results

February 23, Q4 and Year-End 2016 Financial Results February 23, 2017 Q4 and Year-End 2016 Financial Results 2 RETHINKING CNS Agenda Today s Speakers Paul Cox, Senior Director, Investor Relations Jeff Jonas, M.D., Chief Executive Officer Jim Doherty, Ph.D.,

More information

Rhythm Plus- Comprehensive Female Hormone Profile

Rhythm Plus- Comprehensive Female Hormone Profile Rhythm Plus- Comprehensive Female Hormone Profile Patient: SAMPLE REPORT DOB: Sex: F Order Number: K00000 Completed: Received: Collected: SAMPLE REPORT Sample # Progesterone (pg/ml) Hormone Results Oestradiol

More information

AR CS205 Clinical Pilot Study Topline Results

AR CS205 Clinical Pilot Study Topline Results AR-13324-CS205 Clinical Pilot Study Topline Results 1 Important Information The information in this presentation is current only as of its date and may have changed or may change in the future. We undertake

More information

Determined to realize a future in which people with cancer live longer and better than ever before Q Conference Call

Determined to realize a future in which people with cancer live longer and better than ever before Q Conference Call Reimagining Cancer Treatment Determined to realize a future in which people with cancer live longer and better than ever before Q1 2016 Conference Call 1 Forward-Looking Statements Disclosure This presentation

More information

Zafgen PWS Clinical Trial Program Overview. November 16, 2014

Zafgen PWS Clinical Trial Program Overview. November 16, 2014 Zafgen PWS Clinical Trial Program Overview November 16, 2014 2 Disclaimers Forward Looking Statements These slides and the accompanying oral presentation contain forward-looking statements and information.

More information

- Linzagolix overall efficacy and safety maintained or improved at week 24

- Linzagolix overall efficacy and safety maintained or improved at week 24 Final results of ObsEva SA Phase 2b EDELWEISS trial of Linzagolix show sustained efficacy and Bone Mineral Density safety, for the treatment of endometriosis-associated pain - Linzagolix overall efficacy

More information

Company Presentation June 2016

Company Presentation June 2016 Company Presentation June 2016 Millendo Therapeutics Highlights Establishing a leading endocrine company Pipeline of first-in-class, disease-modifying, clinical-stage therapeutics for endocrine diseases

More information

Santhera to Acquire Option from Idorsia for Exclusive Sub-License to First-in-class Dissociative Steroid Vamorolone. Webcast, 21 November 2018

Santhera to Acquire Option from Idorsia for Exclusive Sub-License to First-in-class Dissociative Steroid Vamorolone. Webcast, 21 November 2018 Santhera to Acquire Option from Idorsia for Exclusive Sub-License to First-in-class Dissociative Steroid Vamorolone Webcast, 21 November 2018 Disclaimer This presentation is not and under no circumstances

More information

Conflict of Interest Disclosure

Conflict of Interest Disclosure Incidence and Preventative Strategies of Adrenal Crisis in Congenital Adrenal Hyperplasia PadmasreeVeeraraghavan 1, Diala El-Maouche 1, Deborah P. Merke 1,2 1 National Institutes of Health Clinical Center,

More information

Jefferies Healthcare Conference. June 25, 2008

Jefferies Healthcare Conference. June 25, 2008 Jefferies Healthcare Conference June 25, 2008 Safe Harbor Statement Except for the historical information set forth herein, the matters set forth in this presentation,including without limitation statements

More information

CHOLESTEROL IS THE PRECURSOR OF STERIOD HORMONES

CHOLESTEROL IS THE PRECURSOR OF STERIOD HORMONES HORMONES OF ADRENAL CORTEX R. Mohammadi Biochemist (Ph.D.) Faculty member of Medical Faculty CHOLESTEROL IS THE PRECURSOR OF STERIOD HORMONES CONVERSION OF CHOLESTROL TO PREGNENOLONE MINERALOCORTICOCOIDES

More information

Determined to realize a future in which people with cancer live longer and better than ever before

Determined to realize a future in which people with cancer live longer and better than ever before Determined to realize a future in which people with cancer live longer and better than ever before 4Q 2016 EARNINGS PRESENTATION MARCH 2017 1 Forward-looking statements disclosure This presentation contains

More information

Anti-IL-33 (ANB020) Program

Anti-IL-33 (ANB020) Program Anti-IL-33 (ANB020) Program Phase 2a Peanut Allergy Clinical Trial Interim Data Update March 26 th 2018 NASDAQ: ANAB Safe Harbor Statement This presentation and the accompanying oral presentation contain

More information

Transforming Treatment for Migraine. 16 th Annual Needham Healthcare Conference April 4, 2017

Transforming Treatment for Migraine. 16 th Annual Needham Healthcare Conference April 4, 2017 Transforming Treatment for Migraine 16 th Annual Needham Healthcare Conference April 4, 2017 Forward Looking Statements This presentation and the accompanying commentary contain certain forward-looking

More information

Diagnostics for the early detection and prevention of colorectal cancer.

Diagnostics for the early detection and prevention of colorectal cancer. Diagnostics for the early detection and prevention of colorectal cancer. Company Presentation May 2013 Safe Harbor Statement Certain statements made in this presentation contain forward-looking statements

More information

Polycystic Ovary Syndrome HEATHER BURKS, MD OU PHYSICIANS REPRODUCTIVE MEDICINE SEPTEMBER 21, 2018

Polycystic Ovary Syndrome HEATHER BURKS, MD OU PHYSICIANS REPRODUCTIVE MEDICINE SEPTEMBER 21, 2018 Polycystic Ovary Syndrome HEATHER BURKS, MD OU PHYSICIANS REPRODUCTIVE MEDICINE SEPTEMBER 21, 2018 Learning Objectives At the conclusion of this lecture, learners should: 1) Know the various diagnostic

More information

Alexis Howerton On Starting Up From New Science

Alexis Howerton On Starting Up From New Science Magazine Article August 2, 2018 Alexis Howerton On Starting Up From New Science Source: Life Science Leader By Wayne Koberstein, Executive Editor, Life Science Leader magazine Follow Me On Twitter @WayneKoberstein

More information

Audit of Adrenal Function Tests. Kate Davies Senior Lecturer in Children s Nursing London South Bank University London, UK

Audit of Adrenal Function Tests. Kate Davies Senior Lecturer in Children s Nursing London South Bank University London, UK Audit of Adrenal Function Tests Kate Davies Senior Lecturer in Children s Nursing London South Bank University London, UK Introduction Audit Overview of adrenal function tests Education Audit why? Explore

More information

Adrenal Insufficiency

Adrenal Insufficiency Adrenal Insufficiency Normal adrenal physiology Clinical features, Laboratory findings Common causes of primary adrenal insufficiency Evaluation of suspected adrenal insufficiency Acute and chronic management

More information

VolitionRx Announces First Quarter 2016 Financial Results and Business Update

VolitionRx Announces First Quarter 2016 Financial Results and Business Update May 13, 2016 VolitionRx Announces First Quarter 2016 Financial Results and Business Update NAMUR, Belgium, May 13, 2016 /PRNewswire/ -- VolitionRx Limited (NYSE MKT: VNRX), a life sciences company focused

More information

THE VALUE OF 24 HOUR PROFILES IN CONGENITAL ADRENAL HYPERPLASIA

THE VALUE OF 24 HOUR PROFILES IN CONGENITAL ADRENAL HYPERPLASIA THE VALUE OF 24 HOUR PROFILES IN CONGENITAL ADRENAL HYPERPLASIA This leaflet is a joint production between Professor Peter Hindmarsh and Kathy Geertsma The series editor is Professor Peter Hindmarsh Professor

More information

Corporate Presentation. October 2017

Corporate Presentation. October 2017 AEPP Breakthrough technology for women s cancers Corporate Presentation October 2017 Oncolix 2017 1 Safe Harbor Certain of the statements and the projections set forth in this presentation constitute forward-looking

More information

When testes make no testosterone: Identifying a rare cause of 46, XY female phenotype in adulthood

When testes make no testosterone: Identifying a rare cause of 46, XY female phenotype in adulthood When testes make no testosterone: Identifying a rare cause of 46, XY female phenotype in adulthood Gardner DG, Shoback D. Greenspan's Basic & Clinical Endocrinology, 10e; 2017 Sira Korpaisarn, MD Endocrinology

More information

November 2, Q Financial Results

November 2, Q Financial Results November 2, 2017 Q3 2017 Financial Results Agenda Today s Speakers Paul Cox, Senior Director, Investor Relations Jeff Jonas, M.D., Chief Executive Officer Steve Kanes, M.D., Ph.D., Chief Medical Officer

More information

-- Single Global Phase 3 Trial Expected to Begin in First Half of

-- Single Global Phase 3 Trial Expected to Begin in First Half of Catabasis Pharmaceuticals Reports Edasalonexent Preserved Muscle Function and Substantially Slowed Duchenne Muscular Dystrophy Disease Progression Through More Than One Year of Treatment -- Consistent

More information

N a s d a q : I N S Y

N a s d a q : I N S Y N a s d a q : I N S Y 0 Safe Harbor Statement This presentation contains both historical information and forward-looking statements. Forward-looking statements are based on management's current expectations

More information

Therapeutic Cohort Results

Therapeutic Cohort Results Patient: JANE DOE DOB: December 31, 1968 Sex: F MRN: Order Number: Completed: February 26, 2016 Received: February 26, 2016 Collected: February 26, 2016 One Day Hormone Check - Salivary Profile Therapeutic

More information

Reproductive physiology

Reproductive physiology Reproductive physiology Sex hormones: Androgens Estrogens Gestagens Learning objectives 86 (also 90) Sex Genetic sex Gonadal sex Phenotypic sex XY - XX chromosomes testes - ovaries external features Tha

More information

Determined to realize a future in which people with cancer live longer and better than ever before

Determined to realize a future in which people with cancer live longer and better than ever before Determined to realize a future in which people with cancer live longer and better than ever before 3Q 2018 EARNINGS PRESENTATION NOVEMBER 2018 1 Forward-looking statements disclosure This presentation

More information

Reproductive Endocrinology

Reproductive Endocrinology Reproductive Endocrinology Reproductive Endocrinology Hypothalamic hormones Gonadotropin releasing hormone (GnRH) - stimulate release of FSH = follicle stimulating hormone LH = luteinizing hormone from

More information

Therapeutic Cohort Results

Therapeutic Cohort Results Patient: SAMPLE PATIENT DOB: Sex: MRN: Menopause Plus - Salivary Profile Therapeutic Cohort Results Hormone Average Result QUINTILE DISTRIBUTION 1st 2nd 3rd 4th 5th Therapeutic Range* Estradiol (E2) 8.7

More information

Slide 1. Financial update and closing remarks. Jesper Brandgaard EVP and CFO. RAFAEL DE JESÚS FLORES, Mexico Rafael has haemophilia A

Slide 1. Financial update and closing remarks. Jesper Brandgaard EVP and CFO. RAFAEL DE JESÚS FLORES, Mexico Rafael has haemophilia A Slide 1 Financial update and closing remarks Jesper Brandgaard EVP and CFO RAFAEL DE JESÚS FLORES, Mexico Rafael has haemophilia A Slide 2 Forward-looking statements Novo Nordisk s reports filed with or

More information

TELECONFERENCE FY February 2015

TELECONFERENCE FY February 2015 TELECONFERENCE FY 2014 5 February 2015 Company disclaimer This presentation contains forward-looking statements that provide our expectations or forecasts of future events such as new product introductions,

More information

4Q and Full Year 2017 Financial Results Call February 7, 2018

4Q and Full Year 2017 Financial Results Call February 7, 2018 4Q and Full Year 2017 Financial Results Call February 7, 2018 Agenda Topic Welcome Opening Remarks 4Q 2017 Results Commercial Progress Question & Answer Speakers Amy Sullivan, SVP, Corporate Affairs Greg

More information

Utrophin Modulation: A Universal Treatment Approach to DMD. End Duchenne Tour April 2018

Utrophin Modulation: A Universal Treatment Approach to DMD. End Duchenne Tour April 2018 Utrophin Modulation: A Universal Treatment Approach to DMD April 2018 Legal Disclaimer Statements in this presentation, other than statements of historical fact, constitute forward-looking statements within

More information

Amicus Establishes Gene Therapy Pipeline for Lysosomal Storage Disorders (LSDs) Conference Call and Webcast September 20, 2018

Amicus Establishes Gene Therapy Pipeline for Lysosomal Storage Disorders (LSDs) Conference Call and Webcast September 20, 2018 Amicus Establishes Gene Therapy Pipeline for Lysosomal Storage Disorders (LSDs) Conference Call and Webcast September 20, 2018 Introduction 2 Safe Harbor This presentation contains "forward-looking statements"

More information

2017 Year-end Results and Corporate Update

2017 Year-end Results and Corporate Update NASDAQ: GNMX 2017 Year-end Results and Corporate Update March 13, 2018 2018, Aevi Genomic Medicine Forward-Looking Statement This presentation includes certain estimates and other forward-looking statements

More information

Savient's Pegloticase Data in Treatment-Failure Gout Patients Presented at 72nd Annual Meeting of the American College of Rheumatology Conference

Savient's Pegloticase Data in Treatment-Failure Gout Patients Presented at 72nd Annual Meeting of the American College of Rheumatology Conference Savient's Pegloticase Data in Treatment-Failure Gout Patients Presented at 72nd Annual Meeting of the American College of Rheumatology Conference EAST BRUNSWICK, N.J., Oct 27, 2008 /PRNewswire-FirstCall

More information

Sunesis Pharmaceuticals Reports Second Quarter 2011 Financial Results

Sunesis Pharmaceuticals Reports Second Quarter 2011 Financial Results Investor and Media Inquiries: David Pitts Argot Partners 212-600-1902 Eric Bjerkholt Sunesis Pharmaceuticals Inc. 650-266-3717 Sunesis Pharmaceuticals Reports Second Quarter 2011 Financial Results SOUTH

More information

Slide 1. International Operations update. Mike Doustdar EVP International Operations. YASMIN FIEDLER, Germany Yasmin has type 1 diabetes

Slide 1. International Operations update. Mike Doustdar EVP International Operations. YASMIN FIEDLER, Germany Yasmin has type 1 diabetes Slide 1 International Operations update Mike Doustdar EVP International Operations YASMIN FIEDLER, Germany Yasmin has type 1 diabetes Slide 2 Forward-looking statements Novo Nordisk s reports filed with

More information

Prothena Corporation plc

Prothena Corporation plc Prothena Corporation plc PRX003 Investor Update: Phase 2 Development Strategy September 29, 2016 Agenda Dr. Gene Kinney, Chief Operating Officer Introduction Dr. Ken Flanagan, Senior Scientist Th17 and

More information

Credit Suisse 27 th Annual Healthcare Conference

Credit Suisse 27 th Annual Healthcare Conference CHANGING THE COURSE OF HUMAN HEALTH THROUGH BOLD PURSUITS IN SCIENCE Credit Suisse 27 th Annual Healthcare Conference November 14, 2018 Forward-Looking Statements and Adjusted Financial Information This

More information

Therapeutic Cohort Results

Therapeutic Cohort Results Patient: PAGE LOVE DOB: January 11, 1983 Sex: F MRN: 1232704193 Order Number: J9020008 Completed: July 08, 2016 Received: July 02, 2016 Collected: July 01, 2016 Aum Healing Center Sarika Arora MD 332 Newbury

More information

August 7, Q Financial Results

August 7, Q Financial Results August 7, 2018 Q2 2018 Financial Results 1 Agenda Today s Speakers Paul Cox, Senior Director, Investor Relations Jeff Jonas, M.D., Chief Executive Officer Steve Kanes, M.D., Ph.D., Chief Medical Officer

More information

Dr Stella Milsom. Endocrinologist Fertility Associates Auckland. 12:30-12:40 When Puberty is PCO

Dr Stella Milsom. Endocrinologist Fertility Associates Auckland. 12:30-12:40 When Puberty is PCO Dr Stella Milsom Endocrinologist Fertility Associates Auckland 12:30-12:40 When Puberty is PCO Puberty or Polycystic Ovary Syndrome? Stella Milsom Endocrinologist Auckland DHB, University of Auckland,

More information

BIOSYNTHESIS OF STEROID HORMONES

BIOSYNTHESIS OF STEROID HORMONES BIOSYNTHESIS OF STEROID HORMONES Sri Widia A Jusman Department of Biochemistry & Molecular Biology FMUI sw/steroidrepro/inter/08 1 STEROID HORMONES Progestins (21 C) Glucocorticoids (21 C) Mineralocorticoids

More information

Hyperandrogenism. Dr Jack Biko. MB. BCh (Wits), MMED O & G (Pret), FCOG (SA), Dip Advanced Endoscopic Surgery(Kiel, Germany)

Hyperandrogenism. Dr Jack Biko. MB. BCh (Wits), MMED O & G (Pret), FCOG (SA), Dip Advanced Endoscopic Surgery(Kiel, Germany) Hyperandrogenism Dr Jack Biko MB. BCh (Wits), MMED O & G (Pret), FCOG (SA), Dip Advanced Endoscopic Surgery(Kiel, Germany) 2012 Hyperandrogenism Excessive production of androgens Adrenal glands main source

More information

Corporate Overview June 2014 Jefferies Healthcare Conference NASDAQ: GLYC

Corporate Overview June 2014 Jefferies Healthcare Conference NASDAQ: GLYC Corporate Overview June 2014 Jefferies Healthcare Conference NASDAQ: GLYC Forward-Looking Statements To the extent that statements contained in this presentation are not descriptions of historical facts

More information

Paul Hofman. Professor. Paediatrician Endocrinologist Liggins Institute, The University of Auckland, Starship Children Hospital, Auckland

Paul Hofman. Professor. Paediatrician Endocrinologist Liggins Institute, The University of Auckland, Starship Children Hospital, Auckland Professor Paul Hofman Paediatrician Endocrinologist Liggins Institute, The University of Auckland, Starship Children Hospital, Auckland 14:00-14:55 WS #108: Common pubertal variants how to distinguish

More information