Rani Therapeutics Holdings, Inc. · NASDAQ:RANI
Rani Therapeutics — Investor Presentation
38 slides from Rani Therapeutics's investor presentation
Slide 1: RT-111 Phase 1 Data Corporate Presentation
Our mission at Rani is to end painful injections for the millions of patients suffering from chronic diseases. Rani Therapeutics Corporate Presentation RT-111 Phase 1 Data NASDAQ: RANI
Slide 2: Disclaimer
Disclaimer This presentation and the accompanying oral statements contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act. Forward-looking st
Slide 3: Rani Therapeutics is a public, clinical-stage biotech company
Talat Imran Svai Sanford Mir Hashim, Ph.D. Kate McKinley Rani Therapeutics is a public, Chief Executive Officer Chief Financial Officer Chief Scientific Officer Chief Business Officer clinical-stage biotech company developing a platform technology for the oral delivery of biologic drugs. Eric Groen
Slide 4: Our mission at Rani is to end painful injections
Our mission at Rani is to end painful injections for the millions of patients suffering from chronic diseases FOCUS: Oral Delivery of Biologic Drugs with Bioavailability Comparable to Parenteral Products • 3mg Capacity • 200 µL Capacity TECHNOLOGY: GO HC • Solid Drug Formulation (High Capacity) • Li
Slide 5: Development Pipeline
Development Pipeline INDICATION(S) FORMULATION PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 KEY MILESTONE* CORE PROGRAMS Phase 1 Study RT-111 Psoriasis Ustekinumab** Completed RT-102 Osteoporosis PTH-OP Initiate Phase 2 in 2024 RT-105 Psoriatic Arthritis Adalimumab** Initiate Phase 1 Hypo- RT-110 PTH-Hypo I
Slide 6: Development Pipeline
Development Pipeline INDICATION(S) FORMULATION PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 KEY MILESTONE* CORE PROGRAMS Phase 1 Study RT-111 Psoriasis Ustekinumab** Completed RT-102 Osteoporosis PTH-OP Initiate Phase 2 in 2024 RT-105 Psoriatic Arthritis Adalimumab** Initiate Phase 1 Hypo- RT-110 PTH-Hypo I
Slide 7: RT-111 Phase 1 Study Data
RT-111 Phase 1 Study Data 7
Slide 8: Study Overview
Study Overview A Phase 1 Study to Evaluate the Safety, Tolerability and Pharmacokinetics (PK) of RT-111 – RaniPill Capsule Containing Ustekinumab (CT-P43) – Administered Orally to Healthy Volunteers Objective To assess safety, tolerability and PK of Ustekinumab delivered via Oral RaniPill Study Popu
Slide 9: Study Design: Single Ascending Doses
Study Design: Single Ascending Doses • Study Groups – Control Group: 0.50 mg Stelara SC (N=15) – RT-111 Group: 0.50 mg CT-P43 in RaniPill (N=20) – RT-111 Group: 0.75 mg CT-P43 in RaniPill (N=20) • Protocol – Participants were given a single dose of the study drug (SC or Oral) after an overnight fast
Slide 10: Pharmacokinetics
‘A Pharmacokinetics
Slide 11: PK Profiles of Oral Ustekinumab Biosimilar (RT-111) vs SC Stelara
PK Profiles of Oral Ustekinumab Biosimilar (RT-111) vs SC Stelara Injection Pharmacokinetic Parameters 100 RT-111, 0.75 mg (N=16) Cmax Tmax AUC0-t RT-111, 0.50 mg (N=19) Group 80 (ng/mL) (days) (day.ng/mL) SC, 0.50 mg (N=15) Concentration (ng/mL) SC 0.5mg 60 56 ± 4 10 ± 0.8 1,566 ± 130 Ustekinumab (
Slide 12: Safety & Tolerability
LA Safety & Tolerability
Slide 13: Incidence of Adverse Events
Incidence of Adverse Events SC, 0.50 mg RT-111, 0.50 mg RT-111, 0.75 mg Adverse Events (N=15) (N=20) (N=20) All 1 2 0 Abdominal Bloating 0 1 (5%) 0 Drug-Related Injection Site Rash/Reaction 1 (6.7%) 0 0 Burning Sensation in RaniPill-Related N/A 1 (5%) 0 stomach* *Burning sensation in stomach perceiv
Slide 14: Incidence of Anti-Drug Antibodies (ADAs)
Incidence of Anti-Drug Antibodies (ADAs) Incidence of ADAs Stelara SC RT-111 RT-111 45 0.50 mg 0.50 mg 0.75 mg 40 Incidence of ADA (%) N=15 N=20 N=20 35 30 Drug Signal Detected N=15 N=19 N=16 25 20 Number of ADA Positive Cases 15 10 N 4 2 6 5 Total ADA 0 Positive* % 27% 11% 38% SC 0.50 mg RT-111 0.5
Slide 15: Summary
Summary No meaningful Oral delivery of RT-111 was difference in ADA Ustekinumab well-tolerated development via Rani biosimilar via RaniPill No SAEs route of delivery with compared to SC High Bioavailability injection 15
Slide 16: RT-111 Oral Ustekinumab Target Product Profile
RT-111 Oral Ustekinumab Target Product Profile
Slide 17: Psoriasis Competitive Landscape – Select Injectables
Psoriasis Competitive Landscape – Select Injectables(1) Potential RaniPill Humira Cosentyx Taltz Tremfya Skyrizi Stelara Opportunity Administration SC SC SC SC SC SC Oral Maintenance 3-Day Monthly Short Q2W Q4W Q4W Q8W Quarterly Quarterly Frequency Course Target TNF-α IL-17A IL-17A IL-23 IL-23 IL-12
Slide 18: Psoriasis Competitive Landscape – Select Orals
Psoriasis Competitive Landscape – Select Orals(2) JNJ-2113*** Potential RaniPill Otezla Sotyktu (PTG-200) Opportunity 3-Day Monthly Short Frequency BID Daily Daily / BID Course Target PDE4 TYK2 IL-23 IL-12/IL-23 $.1B Revenue* $2.2B NA -- (Q1-Q3 2023) Total Number of Pills 730 365 365 / 730 36 52 Wee
Slide 19: Historical Data Shows Potential Opportunity to Improve Efficacy and Safety with Higher Loading Doses
Historical Data Shows Potential Opportunity to Improve Efficacy and Safety with Higher Loading Doses Ustekinumab Efficacy at 12 Weeks* Four weekly Four weekly Dose Groups One 45mg dose One 90mg dose 45mg dose 90mg dose 75 PASI Score 52% 59% 67% 81% 90 PASI Score 23% 30% 44% 52% Patients with at 90%
Slide 20: Historical Data Shows Potential Opportunity to Improve Efficacy and Safety with Higher Loading Doses
Historical Data Shows Potential Opportunity to Improve Efficacy and Safety with Higher Loading Doses Ustekinumab Efficacy at 12 Weeks* Four weekly Four weekly Dose Groups One 45mg dose One 90mg dose 45mg dose 90mg dose 75 PASI Score 52% 59% 67% 81% 90 PASI Score 23% 30% 44% 52% Patients with at 90%
Slide 21: Historical Data Shows Potential Opportunity to Improve Efficacy and Safety with Higher Loading Doses
Historical Data Shows Potential Opportunity to Improve Efficacy and Safety with Higher Loading Doses Ustekinumab Efficacy at 12 Weeks* Four weekly Four weekly Dose Groups One 45mg dose One 90mg dose 45mg dose 90mg dose 75 PASI Score 52% 59% 67% 81% 90 PASI Score 23% 30% 44% 52% Patients with at 90%
Slide 22: The RaniPill Could Enable Higher Loading Phase Doses
The RaniPill Could Enable Higher Loading Phase Doses with the Convenience of Oral Administration Simulated RaniPill+Ustekinumab Loading Dose Serum Concentrations at 12 Weeks 25 Four Weekly 90mg Loading Doses Daily Oral Administration with RaniPill 12mg Daily 30-Day Loading Dose (RaniPill Dosing) Cou
Slide 23: Current Stelara Maintenance Phase
Current Stelara Maintenance Phase Current Stelara Dosing Serum Concentration 6 45mg Loading Dose at Week 0 and Week 4, 45mg Q12W Maintenance Dose • Current maintenance dosing for 5 Stelara is 1 injection every 12 weeks Concentration (µg/ml) 4 • Infrequency of injection results in serum concentration
Slide 24: RaniPill + Ustekinumab Proposed Maintenance Dosing
RaniPill + Ustekinumab Proposed Maintenance Dosing Proposed RaniPill Dosing 25 45mg Loading Dose at Week 0 and Week 4, 45mg 20 Q12W Maintenance Dose 12mg Daily Loading Dose for 30 Days, 7mg 3-Day Concentration (µg/ml) Target Therapeutic Concentration Monthly Short Course Maintenance Dose 15 12mg Dai
Slide 25: Target Dosing: RaniPill + Ustekinumab
Target Dosing: RaniPill + Ustekinumab Loading Phase 30-days of 7-12mg Daily Dosing DAILY • Potential for better early-onset clinical efficacy Maintenance Phase 7-12mg 3-day Monthly Short Course –Total of 36 pills per year per patient MONTHLY • Potential for tighter banding of therapeutic concentrati
Slide 26: Advantages of RaniPill Technology in Psoriasis
Advantages of RaniPill Technology in Psoriasis Other Oral Options Injectables RaniPill Targets • Less Efficacious than Biologics • Inconvenient & Painful to − Otezla, JAK Inhibitors Administer • Efficacy Comparable to Injectable Biologics • Additional Safety Concerns • Dosing Regimen not Maximizing
Slide 27: Celltrion Supply Agreement Includes ROFN
Celltrion Supply Agreement Includes ROFN Ustekinumab Biosimilar (RT-111) • Lead Indication: Psoriasis • Market Size: $9.7B in Stelara (ustekinumab) sales worldwide Combining proven, high value in 2022 (3) drugs with competitive and disruptive technology Adalimumab Biosimilar (RT-105) Two partner
Slide 28: Target Product Profile of RaniPill Products
Target Product Profile of RaniPill Products • Efficacy • Long Half-Life • Safety Goals Of a Monoclonal Antibody More convenient than small molecule Equal or better efficacy • Convenience compared to injectables • Dosing Flexibility • Patient Preference Potentially lower AEs Of a Pill than injectable
Slide 29: Anticipated Upcoming Milestones & Progress
Anticipated Upcoming Milestones & Progress • Initiate Phase 2 clinical trial with RT-102 in 2024 • Advance clinical development of RT-111 at higher doses • Progress development of RaniPill HC towards clinic • Evaluate platform further in strategic areas of focus 29
Slide 30: Thank You
Thank You Rani - Confidential 30
Slide 31: Additional Updates
Additional Updates
Slide 32: Transenteric Delivery of GLP-1/GIP/Glucagon Molecule Elicits Rapid Weight Loss in Beagle Dogs
Transenteric Delivery of GLP-1/GIP/Glucagon Molecule Elicits Rapid Weight Loss in Beagle Dogs Objective ∆ Body Weight (%) ∆ Serum Cholesterol (%) • To evaluate the PK-PD profiles of Blinded Triagonist (a unimolecular incretin agonist for GLP-1, GIP and Glucagon Group 1 Group 2 Group 1 Group 2 recept
Slide 33: Obesity Market Potential Opportunity
Obesity Market Potential Opportunity Potential Oral Wegovy Mounjaro Retatrutide Orfoglipron Danuglipron RaniPill Semaglutide Opportunity Administration SC SC SC Oral Oral Oral Oral Frequency Weekly Weekly Weekly Daily Daily Twice Daily Daily or Weekly GLP-1, GLP-1, Dual Single, Dual and Target GLP-1
Slide 34: Appendix
Appendix
Slide 35: RT-111 Phase 1 Additional Data
RT-111 Phase 1 Additional Data
Slide 36: Study Demographics – RT-111 Phase 1 Study
Study Demographics – RT-111 Phase 1 Study SC RT-111 RT-111 0.50 mg 0.50 mg 0.75 mg N 15 20 20 Mean Age, years 29.3 (19 - 49) 28.3 (20 - 39) 30 (20 - 58) Race Hispanic 0% (0/15) 25% (5/20) 5% (1/20) White-non-Hispanic 60% (9/15) 35% (7/20) 75% (15/20) Asian 40% (6/15) 35% (7/20) 20% (4/20) Body Mass
Slide 37: PK Profiles of Oral Ustekinumab Biosimilar (RT-111) vs SC Stelara
PK Profiles of Oral Ustekinumab Biosimilar (RT-111) vs SC Stelara Injection PK - ALL PK - ADA Negative 100 100 RT-111, 0.75 mg (N=10) RT-111, 0.75 mg (N=16) RT-111, 0.50 mg (N=17) 80 RT-111, 0.50 mg (N=19) 80 Concentration (ng/mL) Concentration (ng/mL) SC, 0.50 mg (N=11) SC, 0.50 mg (N=15) Ustekinum
Slide 38: References
References (1) Data of third party molecules are from separate studies published or disclosed by such third parties. Data are not from head-to-head studies. For Humira data and PASI 75 at 16 weeks, see tables 17-18 in U.S. prescribing information. For Humira PASI 75 at 48 weeks, see Table 3 of EMA S