Spruce Biosciences, Inc. · NASDAQ:SPRB
Spruce Biosciences — Investor Day (August 2021)
68 slides from Spruce Biosciences's investor day presentation, Aug 25, 2021
Slide 1: Research and Development Day Tildacerfont for Adult and Pediatric Classic CAH
Research and Development Day Tildacerfont for Adult and Pediatric Classic CAH August 25, 2021 Exhibit 99.1
Slide 2: Forward-Looking Statements
FORWARD-LOOKING STATEMENTS This presentation contains forward-looking statements about Spruce Biosciences, Inc. (“we,” “Spruce” or the “Company”). All statements other than statements of historical facts contained in this presentation are forward-looking statements, including statements about our st
Slide 3: Agenda
AGENDA Classic CAH Overview Management of CAH About Tildacerfont Phase 2 Adult Classic CAH Development Program Late-stage Adult Classic CAH Development Program KOL Panel Discussion Pediatric Classic CAH Overview Phase 2 Pediatric Classic CAH Development Program
Slide 4: Today's Speakers
TODAY’S SPEAKERS Richard King Chief Executive Officer Spruce Biosciences Rosh Dias, MD, MRCP Chief Medical Officer Spruce Biosciences Paul Thornton, MD Medical Director, Endocrine and Diabetes Program Cook Children’s Hospital Rich Auchus, MD, PhD Professor of Internal Medicine and Pharmacology Unive
Slide 5: Spruce At-a-Glance: Key Investor Highlights
SPRUCE AT-A-GLANCE KEY INVESTOR HIGHLIGHTS Tildacerfont poised to transform treatment paradigm in classic CAH Strong IP protection Multiple expansion opportunities Significant commercial opportunity Highly experienced leadership team Two late-stage clinical studies initiated; Data expected in 2022.
Slide 6: Patient Views on Management of Adult CAH
PATIENT VIEWS ON MANAGEMENT OF ADULT CAH Based on survey of 59 patients. CAH, congenital adrenal hyperplasia; GC, glucocorticoid; HCP, healthcare professional. Spruce Biosciences. Data on file. The vast majority of patients (>90%) report GCs are effective in controlling CAH, but… Side-effects are co
Slide 7: Classic CAH Overview
Classic CAH Overview —— SPprucesiOosciENCES
Slide 8: CAH Is a Chronic Genetic Disease
CAH IS A CHRONIC GENETIC DISEASE Congenital adrenal hyperplasia encompasses a group of rare autosomal recessive disorders of the adrenal cortex Genetic mutations cause deficiency in one or more key enzymes involved in adrenal steroidogenesis (cortisol synthesis) Clinical features are linked to corti
Slide 9: CYP21A2 Mutation Is the Most Common Cause of CAH
21-OH, 21-hydroxylase; CAH, congenital adrenal hyperplasia; CYP, cytochrome. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]. CYP21A2 MUTATION IS THE MOST COMMON CAUSE OF CAH Mutations Involved in CAH Less common mutation
Slide 10: Of the 21-OH Deficient CAH Subtypes, Classic Is More Severe
21-OH, 21-hydroxylase; 21-OHD, 21-hydroxylase deficient; CAH, congenital adrenal hyperplasia; CYP, cytochrome. 1. White, P, et al. Endocr Rev. 2000;21:245-91.; 2. Livadas S, et al. Front Endocrinol. 2019;10:1-11; 3. Classic 21-OHD CAH1 More severe, life-threatening 1:18,000-10,000 births worldwide O
Slide 11: HPA Axis Functions as a Negative Feedback Loop
CRF from the hypothalamus stimulates the pituitary to produce ACTH CRF ACTH ACTH, adrenocorticotropic hormone; CRF, corticotropin releasing factor; HPA, hypothalamic-pituitary-adrenal. Engels M, et al. Endocr Rev. 2019;40:973-87. HPA AXIS FUNCTIONS AS A NEGATIVE FEEDBACK LOOP Kidneys Hypothalamus Pi
Slide 12: HPA Axis Functions as a Negative Feedback Loop
CRF ACTH 17-OHP, 17-hydroxyprogesterone; 21-OH, 21-hydroxylase; A4, androstenedione; ACTH, adrenocorticotropic hormone; CRF, corticotropin releasing factor; HPA, hypothalamic-pituitary-adrenal. Engels M, et al. Endocr Rev. 2019;40:973-87. Kidneys Hypothalamus Pituitary Cortisol Adrenal Glands Pituit
Slide 13: HPA Axis Functions as a Negative Feedback Loop
17-OHP, 17-hydroxyprogesterone; 21-OH, 21-hydroxylase; ACTH, adrenocorticotropic hormone; CRF, corticotropin releasing factor; HPA, hypothalamic-pituitary-adrenal. Engels M, et al. Endocr Rev. 2019;40:973-87. HPA AXIS FUNCTIONS AS A NEGATIVE FEEDBACK LOOP CRF ACTH Kidneys Hypothalamus Pituitary Cort
Slide 14: 21-OHD CAH: Loss of Negative Feedback
Deficiency in 21-OH prevents cortisol production, & reduces or prevents aldosterone production Lack of cortisol upregulates CRF & ACTH, which leads to overstimulation & hyperplasia of the adrenal glands 17-OHP is routed to the androgen pathway, resulting in excess androgens 21-OHD CAH: LOSS OF NEGAT
Slide 15: 21-OHD CAH Is Classified by Degree of Hormone Imbalance
Androgens Cortisol 21-OH, 21-hydroxylase ; 21-OHD, 21-hydroxylase deficient; CAH, congenital adrenal hyperplasia. 1. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 2. Nordenstrom A, et al. Eur J Endocrinol. 2019;180:R12
Slide 16: Hormone Imbalances Are Characteristic of 21-OHD CAH
Cortisol Aldosterone Cholesterol Progesterone ACTH 21-OH 21-OH Androgens (e.g., A4) 17-OHP HORMONE IMBALANCES ARE CHARACTERISTIC OF 21-OHD CAH 17-OHP, 17-hydroxyprogesterone; 21-OH, 21-hydroxylase; 21-OHD, 21-hydroxylase deficient; ACTH, adrenocorticotropic hormone; CAH, congenital adrenal hyperplas
Slide 17: Hormone Imbalances Are Characteristic of 21-OHD CAH
HORMONE IMBALANCES ARE CHARACTERISTIC OF 21-OHD CAH 17-OHP, 17-hydroxyprogesterone; 21-OH, 21-hydroxylase; 21-OHD, 21-hydroxylase deficient; ACTH, adrenocorticotropic hormone; CAH, congenital adrenal hyperplasia; TART, testicular adrenal rest tumor. 1. Claahsen-van der Grinten H, et al. Endocr Rev.
Slide 18: Hormone Imbalances Are Characteristic of 21-OHD CAH
HORMONE IMBALANCES ARE CHARACTERISTIC OF 21-OHD CAH 17-OHP, 17-hydroxyprogesterone; 21-OH, 21-hydroxylase; 21-OHD, 21-hydroxylase deficient; ACTH, adrenocorticotropic hormone; CAH, congenital adrenal hyperplasia; TART, testicular adrenal rest tumor. 1. Claahsen-van der Grinten H, et al. Endocr Rev.
Slide 19: Diagnosis of 21-OHD CAH
DIAGNOSIS OF 21-OHD CAH 17-OHP, 17-hydroxyprogesterone; 21-OHD, 21-hydroxylase deficient; ACTH, adrenocorticotropic hormone; CAH, congenital adrenal hyperplasia. 1. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 2. Liva
Slide 20: Management of CAH
Management of CAH —— SPprucesiOosciENCES
Slide 21: Management of Classic CAH Is a Three-Pronged Approach
MANAGEMENT OF CLASSIC CAH IS A THREE-PRONGED APPROACH ACTH, adrenocorticotropic hormone; CAH, congenital adrenal hyperplasia. SUPPRESS ACTH PRODUCTION & ANDROGEN EXCESS REPLACE CORTISOL REPLACE ALDOSTERONE CAH REPLACE ALDOSTERONE REPLACE CORTISOL SUPPRESS ACTH PRODUCTION & ANDROGEN EXCESS CAH
Slide 22: Aldosterone Is Replaced to Maintain Fluid & Electrolyte Balance
ALDOSTERONE IS REPLACED TO MAINTAIN FLUID & ELECTROLYTE BALANCE CAH, congenital adrenal hyperplasia; d, day; g, gram; mg, milligram. 1. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 2. Speiser P, et al. J Clin Endocrin
Slide 23: Low Dose Hydrocortisone Replaces Physiologic Cortisol
LOW DOSE HYDROCORTISONE REPLACES PHYSIOLOGIC CORTISOL CAH, congenital adrenal hyperplasia; d, day; HC, hydrocortisone; mg, milligram. 1. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 2. Bornstein S, et al. J Clin Endoc
Slide 24: Higher Doses of GC Are Required to Suppress ACTH & Androgens
HIGHER DOSES OF GC ARE REQUIRED TO SUPRESS ACTH & ANDROGENS ACTH, adrenocorticotropic hormone; AR, androgen receptor; GC, glucocorticoid; HC, hydrocortisone; mg, milligram; OCP, oral contraceptive pill; TART, testicular adrenal rest tumor. 1. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab0
Slide 25: This Presents a Difficult Choice in Treating Classic CAH
SHORT STATURE CARDIOVASCULAR DISEASE INSULIN RESISTANCE SHORT STATURE ABNORMAL AND/OR EARLY PUBERTY INFERTILITY PSYCHOLOGICAL EFFECTS HIRSUTISM, AMENORRHEA ACNE Excess GCs Excess Androgens OSTEOPOROSIS OBESITY THIS PRESENTS A DIFFICULT CHOICE IN TREATING CLASSIC CAH CAH, congenital adrenal hyperplas
Slide 26: Unmet Medical Needs According to Disease Status
UNMET MEDICAL NEEDS ACCORDING TO DISEASE STATUS CAH, congenital adrenal hyperplasia; GC, glucocorticoid. 1. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 2. Speiser P, et al. J Clin Endocrinol Metab. 2018;103:4043-88.
Slide 27: Unmet Medical Needs According to Disease Status
UNMET MEDICAL NEEDS ACCORDING TO DISEASE STATUS CAH, congenital adrenal hyperplasia; GC, glucocorticoid. 1. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 2. Speiser P, et al. J Clin Endocrinol Metab. 2018;103:4043-88.
Slide 28: Unmet Medical Needs According to Disease Status
UNMET MEDICAL NEEDS ACCORDING TO DISEASE STATUS CAH, congenital adrenal hyperplasia; GC, glucocorticoid. 1. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 2. Speiser P, et al. J Clin Endocrinol Metab. 2018;103:4043-88.
Slide 29: Unmet Medical Needs in the Current Management of Classic CAH
Novel therapies are needed to reduce the need for supraphysiologic GCs UNMET MEDICAL NEEDS IN THE CURRENT MANAGEMENT OF CLASSIC CAH Glucocorticoids – the mainstay of treatment since the 1950s1 – contribute to the burden of disease CAH, congenital adrenal hyperplasia; GC, glucocorticoid. 1. Hayek A,
Slide 30: Tildacerfont
Tildacerfont —— SPprucesiOosciENCES
Slide 31: Tildacerfont Is a Novel CRF1 Receptor Antagonist
Tildacerfont is an oral, second generation CRF1 receptor antagonist1 Tildacerfont binds to CRF1 receptors in the pituitary gland, blocking receptor stimulation by the hypothalamus1 TILDACERFONT IS A NOVEL CRF1 RECEPTOR ANTAGONIST ACTH, adrenocorticotropic hormone; CRF, corticotropin-releasing factor
Slide 32: Tildacerfont Is Designed to Reduce Adrenal Androgen Production
CRF1 receptor blockade by tildacerfont Reduced ACTH secretion Reduced 17-OHP levels Reduced A4 levels 21-OH Cortisol Effect of tildacerfont on HPA-axis function in CAH1,2 Tildacerfont inhibits excessive production of ACTH, 17-OHP and adrenal androgens1 By reducing excess adrenal androgens (e.g., A4)
Slide 33: Tildacerfont Is a Potent, Highly Selective CRF1 Receptor Antagonist
In cell-based radioligand binding assays, tildacerfont displayed a higher binding affinity for the hCRF1 vs. hCRF2 receptor Tildacerfont did not inhibit any clinically important target by >50% when tested at a concentration ~33,000-fold higher than the Ki for binding to the hCRF1 receptor TILDACERFO
Slide 34: Phase 2 Adult Classic CAH Clinical Development Program
Phase 2 Adult Classic CAH Clinical Development Program
Slide 35: Eight Clinical Studies of Tildacerfont Have Been Completed
PHASE 1 SPR001-103 I3C-FW-BLAA SPR001-105 SPR001-104 I3C-FW-BLAB SPR001-201 PHASE 2 SPR001-202 EIGHT CLINICAL STUDIES OF TILDACERFONT HAVE BEEN COMPLETED Spruce Biosciences, Inc. Investigator’s brochure for Tildacerfont (SPR001), Version 5.0, Dated 26 March 2021. SPR001-106
Slide 36: SPR001-201: Clinical Proof of Concept
SPR001-201: CLINICAL PROOF OF CONCEPT (PHASE 2 STUDY)1,2 17-OHP, 17-hydroxyprogesterone; BID, twice daily; PD, pharmacodynamics; PK, pharmacokinetics; PO, oral administration; QD, once daily. 1. Sarafoglou K, et al. J Clin Endocrinol Metab. 2021:dgab438. DOI: https://doi.org/10.1210/clinem/dgab438 [
Slide 37: SPR001-202: Twelve-Week, Open-Label Phase 2 Study
SPR001-202: TWELVE-WEEK, OPEN-LABEL PHASE 2 STUDY1,2 *Trial visits were conducted in the morning, at approximately 8 AM, prior to consumption of a morning GC dose at baseline (Day 1) and Weeks 2, 4, 6, 8, 10, and 12, and 30 days after the last dose. 17-OHP, 17-hydroxyprogesterone; A4, androstenedion
Slide 38: SPR001-202: Eligibility Criteria
SPR001-202: ELIGIBILITY CRITERIA1,2 *If screening occurred >3 months after the subject's final follow-up visit for SPR001-201, 17-OHP was measured at screening and patients were screened for any clinically significant unstable medical condition, medically significant illness, or chronic disease occu
Slide 39: SPR001-202: Patient Demographics and Baseline Characteristics
SPR001-202: PATIENT DEMOGRAPHICS AND BASELINE CHARACTERISTICS *Patients receiving dexamethasone (demonstrated by a post-hoc analysis to have the potential to confound efficacy assessments) were excluded from efficacy analyses but included in safety and pharmacokinetic analyses. ‡Combination therapy:
Slide 40: SPR001-202: Robust Reduction in ACTH in Poorly Controlled Disease
SPR001-202: ROBUST REDUCTION IN ACTH IN POORLY CONTROLLED DISEASE *One subject at week 2 prior to discontinuation from the trial and two patient during month 3. ACTH, adrenocorticotropic hormone; QD, once daily. Sarafoglou K, et al. J Clin Endocrinol Metab. 2021:dgab438. DOI: https://doi.org/10.1210
Slide 41: SPR001-202: Sustained Reduction in 17-OHP in Poorly Controlled Disease
SPR001-202: SUSTAINED REDUCTION IN 17-OHP IN POORLY CONTROLLED DISEASE A4, androstenedione; QD, once daily. Sarafoglou K, et al. J Clin Endocrinol Metab. 2021:dgab438. DOI: https://doi.org/10.1210/clinem/dgab438 [Epub ahead of print]. In poor disease control group, an initial drop in 17-OHP was seen
Slide 42: SPR001-202: Sustained Reduction in A4 in Poorly Controlled Disease
SPR001-202: SUSTAINED REDUCTION IN A4 IN POORLY CONTROLLED DISEASE A4, androstenedione; QD, once daily. Sarafoglou K, et al. J Clin Endocrinol Metab. 2021:dgab438. DOI: https://doi.org/10.1210/clinem/dgab438 [Epub ahead of print]. In poor disease control group, an initial drop in A4 was seen at week
Slide 43: SPR001-202: Treatment-Emergent Adverse Events
Preferred Term 400 mg QD (n=11) Participants with at least one TEAE, n (%) 9 (81.8) Upper respiratory tract infection 2 (18.2) Hypothyroidism 1 (9.1) Abdominal pain upper 1 (9.1) Diarrhea 1 (9.1) Nausea 1 (9.1) Vomiting 1 (9.1) Dysgeusia 1 (9.1) Glycosylated hemoglobin increased 1 (9.1) Hepatic enzy
Slide 44: Key Findings from Phase 1 and 2 Studies: Summary
Efficacy Two distinct patient populations:1 These form the basis of the late-stage clinical program KEY FINDINGS FROM PHASE 1 AND 2 STUDIES: SUMMARY ACTH, adrenocorticotropic hormone; ALT, alanine aminotransferase; AST, aspartate aminotransferase; CAH, congenital adrenal hyperplasia; QD, once daily;
Slide 45: Late-Stage Adult Classic CAH CAHmelia Program
Late-Stage Adult Classic CAH CAHmelia Program
Slide 46: CAHmelia-203: Adrenal Androgen Reduction Study
CAHmelia-203: ADRENAL ANDROGEN REDUCTION STUDY Study schema is not drawn to scale. SINGLE-BLIND RUN-IN (6 weeks on placebo) SAFETY FOLLOW-UP (30 days) PART B (12 weeks) PART A (12 weeks) PART C (46 weeks) RANDOMIZED PERIOD OPEN-LABEL EXTENSION R Tildacerfont 200 mg QD Tildacerfont 100 mg QD Tildacer
Slide 47: CAHmelia-203: Study Endpoints
CAHmelia-203: STUDY ENDPOINTS 17-OHP, 17-hydroxyprogesterone; A4, androstenedione; ACTH, adrenocorticotropic hormone; CAH, congenital adrenal hyperplasia; GC, glucocorticoid; QoL, quality of life; TART, testicular adrenal rest tumor; ULN, upper limit of normal. PRIMARY ENDPOINT Percentage change in
Slide 48: CAHmelia-204: GC Reduction Study
CAHmelia-204: GC REDUCTION STUDY A randomized, double-blind, placebo-controlled Phase 2b study to evaluate the efficacy and safety of tildacerfont in reducing supraphysiologic GC use in adult patients with classic CAH Study schema is not drawn to scale. GC CONVERSION (6 or 12 weeks) SAFETY FOLLOW-UP
Slide 49: CAHmelia-204: Study Endpoints
CAHmelia-204: STUDY ENDPOINTS 17-OHP, 17-hydroxyprogesterone; A4, androstenedione; ACTH, adrenocorticotropic hormone; BMI, body mass index; CAH, congenital adrenal hyperplasia; d, day; DXA, dual-energy X-ray absorptiometry; GC, glucocorticoid; HCe, hydrocortisone equivalent(s); HOMA-IR, homeostatic
Slide 50: KOL Panel Discussion
KOL Panel Discussion —— SPprucesiOosciENCES
Slide 51: Today's Panelists
TODAY’S PANELISTS Paul Thornton, MD Richard Auchus, MD, PhD Rosh Dias, MD, MRCP Moderator
Slide 52: Pediatric Classic CAH Overview
Pediatric Classic CAH Overview
Slide 53: Classic CAH Presents in Infancy & Early Childhood
CLASSIC CAH PRESENTS IN INFANCY & EARLY CHILDHOOD CAH, congenital adrenal hyperplasia 1. Falhammer H, et al. J Clin Endocrinol Metab. 2014; 99: E2715-E2721; 2. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 3. Merke D,
Slide 54: Classic CAH Presents in Infancy & Early Childhood
CLASSIC CAH PRESENTS IN INFANCY & EARLY CHILDHOOD ADHD, attention deficit hyperactivity disorder; CAH, congenital adrenal hyperplasia. 1. Falhammer H, et al. J Clin Endocrinol Metab. 2014; 99: E2715-E2721; 2. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/e
Slide 55: Classic CAH Presents in Infancy & Early Childhood
CLASSIC CAH PRESENTS IN INFANCY & EARLY CHILDHOOD CAH, congenital adrenal hyperplasia 1. Falhammer H, et al. J Clin Endocrinol Metab. 2014; 99: E2715-E2721; 2. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 3. Merke D,
Slide 56: Classic CAH Presents in Infancy & Early Childhood
CLASSIC CAH PRESENTS IN INFANCY & EARLY CHILDHOOD CAH, congenital adrenal hyperplasia; TARTs, testicular adrenal rest tumors. 1. Falhammer H, et al. J Clin Endocrinol Metab. 2014; 99: E2715-E2721; 2. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bna
Slide 57: Classic CAH Presents in Infancy & Early Childhood
CLASSIC CAH PRESENTS IN INFANCY & EARLY CHILDHOOD CAH, congenital adrenal hyperplasia. 1. Falhammer H, et al. J Clin Endocrinol Metab. 2014; 99: E2715-E2721; 2. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 3. Merke D,
Slide 58: Classic CAH Presents in Infancy & Early Childhood
CLASSIC CAH PRESENTS IN INFANCY & EARLY CHILDHOOD CAH, congenital adrenal hyperplasia. 1. Falhammer H, et al. J Clin Endocrinol Metab. 2014; 99: E2715-E2721; 2. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 3. Merke D,
Slide 59: Short Stature in CAH Is Caused by Androgens and GCs
SHORT STATURE IN CAH IS CAUSED BY ANDROGENS AND GCs CAH, congenital adrenal hyperplasia; GC, glucocorticoid. 1. Merke D, et al. N Engl J Med. 2020;383:1248-61; 2. Lui J. Endocr Dev. 2011;20:187-93; 3. Claahsen-van der Grinten H, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bn
Slide 60: Unmet Needs in Pediatric CAH: Strategies to Balance Androgens & GC Dose
UNMET NEEDS IN PEDIATRIC CAH: STRATEGIES TO BALANCE ANDROGENS & GC DOSE CAH, congenital adrenal hyperplasia; GC, glucocorticoid. 1. Claahsen-van der Grinten HL, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Epub ahead of print]; 2. Pijnenburg-Kleizen KJ, et al. J Pedi
Slide 61: Phase 2 Pediatric Classic CAH Development Program
Phase 2 Pediatric Classic CAH Development Program
Slide 62: Management Goals of Pediatric CAH Vary with Age
MANAGEMENT GOALS OF PEDIATRIC CAH VARY WITH AGE 17-OHP, 17-hydroxyprogesterone; CAH, congenital adrenal hyperplasia; d, day; GC, glucocorticoid; HC, hydrocortisone; MC, mineralocorticoid. 1. Claahsen-van der Grinten HL, et al. Endocr Rev. 2021;bnab016. DOI: https://doi.org/10.1210/endrev/bnab016 [Ep
Slide 63: Phase 2 Study in Pediatric CAH: To Be Initiated in 2021
PHASE 2 STUDY IN PEDIATRIC CAH: TO BE INITIATED IN 2021 Study schema is not drawn to scale. *Weight-based dosing at adult/effective dose equivalents. 17-OHP, 17-hydroxyprogesterone; A4, androstenedione; ACTH, adrenocorticotropic hormone; B, baseline; CAH, congenital adrenal hyperplasia; DSMB, Data S
Slide 64: Concluding Remarks
Concluding Remarks —— SPprucesiOosciENCES
Slide 65: Key Anticipated Milestones
KEY ANTICIPATED MILESTONES Initiate Phase 2 trial in pediatric classic CAH 2H2021 Initiate Phase 2 proof-of-concept trial in PCOS 2H2021 2H2022 Topline results in adult classic CAH (CAHmelia-204) 1H2022 Topline results in adult classic CAH (CAHmelia-203) Phase 2 results in pediatric classic CAH and
Slide 66: Investment Highlights
INVESTMENT HIGHLIGHTS KEY INVESTOR HIGHLIGHTS Tildacerfont poised to transform treatment paradigm in classic CAH Strong IP protection Multiple expansion opportunities Significant commercial opportunity Highly experienced leadership team Two late-stage clinical studies initiated; Data expected in 202
Slide 67: Q&A Session
Q&A Session Research and Development Day Tildacerfont for Adult and Pediatric Classic CAH August 25, 2021
Slide 68: SPrucesiOsciENCES
Ss SPrucesiOsciENCES