Mbx Biosciences, Inc. · NASDAQ:MBX
Mbx Biosciences — Investor Presentation (June 2025)
37 slides from Mbx Biosciences's investor presentation, Jun 9, 2025
Slide 1: Precision Endocrine Peptides Company Overview June 2025
Precision Endocrine Peptides™ Company Overview June 2025 Exhibit 99.1
Slide 2: Disclaimer
Disclaimer This presentation includes forward looking statements. All statements other than statements of historical facts contained in this presentation, including statements regarding our product candidates, preclinical study and/or clinical trial timelines, including projected data announcements,
Slide 3: Pioneering Precision Peptides for Endocrine and Metabolic Diseases
Pioneering Precision Peptides for Endocrine and Metabolic Diseases Developing treatments to improve clinical outcomes and simplify disease management Canvuparatide (MBX 2109) for Hypoparathyroidism (HP): PTH peptide prodrug with FDA Orphan Drug Designation Designed to deliver continuous, infusion-li
Slide 4: MBX's World-class Leadership Team
MBX’s World-class Leadership Team Kent Hawryluk PRESIDENT & CEO Rick Bartram, CPA CHIEF FINANCIAL OFFICER Mike Dorato, PhD SENIOR VICE PRESIDENT DISCOVERY & NONCLINCAL DEVELOPMENT Matt Gambino VICE PRESIDENT MARKETING Michelle Graham CHIEF HUMAN RESOURCES OFFICER Sam Azoulay, MD CHIEF MEDICAL OFFICE
Slide 5: Expanding a Pipeline of PEPs in Significant Rare Disease and Obesity Markets
Candidate MOA Indication Discovery IND Enabling Phase 1 Phase 2 Phase 3 Anticipated Milestones Endocrine Portfolio Canvuparatide (MBX 2109) PTH Prodrug Hypoparathyroidism Hypo-parathyroidism Phase 2 topline results in Q3 2025 MBX 1416 GLP-1 Receptor Antagonist Post-Bariatric Hypoglycemia Post-bariat
Slide 6: Precision Endocrine Peptide (PEP) Platform
Precision Endocrine Peptide™ (PEP™) Platform Proprietary tools and know-how with the potential to enable design and development of transformative peptide therapeutics INNOVATIVE PEPTIDE DESIGN Designed to precisely time chemical conversion of prodrug to active drug to reduce peak-to-trough ratios an
Slide 7: Canvuparatide (MBX 2109) Investigational Once-Weekly PTH Replacement Therapy for Hypoparathyroidism
Canvuparatide (MBX 2109) Investigational Once-Weekly PTH Replacement Therapy for Hypoparathyroidism
Slide 8: Canvuparatide for Hypoparathyroidism – A Serious Endocrine Disease
Canvuparatide for Hypoparathyroidism – A Serious Endocrine Disease Estimated >250,000 patients in U.S. and EU combined1,2 PTH=parathyroid hormone 1. Clarke B et al JCEM 2016, 2. Powers J et al JBMR 2013, 3. Khan A et al JBMR 2022, 4. Yao L et al Complications Symptoms JBMR 2022 Hypocalcemia: Tetany,
Slide 9: Managing Hypoparathyroidism: PTH Replacement Aims to Address Shortcomings of Standard of Care
Managing Hypoparathyroidism: PTH Replacement Aims to Address Shortcomings of Standard of Care PTH=parathyroid hormone; QoL=quality of life 1. Khan A et al. JBMR 2022 Calcium and active vitamin D1 supplementation Daily PTH replacement therapy Investigational once-weekly PTH replacement therapy STANDA
Slide 10: Canvuparatide: Prodrug Chemically Converts to Active Drug at a Controlled Rate
Canvuparatide (MBX 2109): Prodrug Chemically Converts to Active Drug at a Precisely Controlled Rate Under Physiologic Conditions PTH=parathyroid hormone Biologically active peptide analog is converted and inactive metabolite eliminated Canvuparatide (MBX 2109) PRODRUG Dipeptide ACTIVE DRUG Fatty aci
Slide 11: Canvuparatide (MBX 2109) Phase 1 Trial
Canvuparatide (MBX 2109) Phase 1 Trial SAD=single ascending dose; MAD=multiple ascending dose; SC=subcutaneous; QW=every week 1. ClinicalTrials.gov Identifier: NCT05158335 Single and multiple ascending doses of MBX 2109 in healthy adult subjects1 Endpoints Primary: Secondary: To evaluate safety and
Slide 12: TEAEs by Preferred Term Following Repeat Administration of Canvuparatide
TEAEs by Preferred Term Reported in ≥5% Subjects Following Repeat Administration of Canvuparatide (MBX 2109) or Placebo n=number of subjects; TEAEs = treatment emergent adverse events MBX 2109 Preferred Term Placebo (N = 8) n (%) 200 µg (N = 8) n (%) 400 µg (N = 8) n (%) 600 µg (N = 8) n (%) 900 µg
Slide 13: Canvuparatide Phase 1: Safety Results
Canvuparatide (MBX 2109) Phase 1: Safety Results AE = adverse event; TEAE = treatment-emergent adverse event 1. Most common TEAEs defined in those occurring in ≥ 3 patients in any treatment group. Canvuparatide was generally well-tolerated No dose limiting toxicity or off-target AEs No related serio
Slide 14: Plasma Concentration-time Profile Demonstrated Infusion-like Profile
Plasma Concentration-time Profile for Active Drug Demonstrated Infusion-like Profile and Supported Once-weekly Dosing QW = once weekly; AUC0-τ = area under the concentration-time curve over a dosing interval; Cmax = maximal observed concentration; Tmax = time of the maximum observed concentration; T
Slide 15: Maximal Increases in Serum Calcium After Weekly Injections
Maximal Increases in Serum Calcium Levels (Emax,adj) After Canvuparatide (MBX 2109) Weekly Injections Demonstrated Dose-and Time-dependent Effect Emax,adj were observed approximately 48 hours after injection Emax, adj (mg/dL) (mean (SD)) Placebo 200 μg MBX 2109 400 μg MBX 2109 600 μg MBX 2109 900 μg
Slide 16: Endogenous PTH in Healthy Subjects Decreased After Weekly Injections
Endogenous PTH in Healthy Subjects Decreased After Canvuparatide (MBX 2109) Weekly Injections in a Dose- and Time-dependent Fashion Parathyroid Hormone, Intact (pg/mL) (mean (SD)) Placebo 200 μg MBX 2109 400 μg MBX 2109 600 μg MBX 2109 900 μg MBX 2109 Lower Limit of Normal 1 4 12 19 1 4 12 19 1 4 12
Slide 17: Canvuparatide Phase 1: PK / PD results
Canvuparatide (MBX 2109) Phase 1: PK / PD results The half-life of the canvuparatide active drug across all doses was approximately 7.7 to 8.9 days, which we believe supports a once-weekly dosing regimen Mean peak-to-trough ratios of the active drug following the last dose ranged from 1.47 to 1.79,
Slide 18: Phase 2 Trial Overview: Canvuparatide (MBX 2109)
(Phase 2) Trial Overview: Canvuparatide (MBX 2109) ClinicalTrials.gov Identifier: NCT06465108 Enrollment complete; TLR anticipated in Q3 2025 Endpoints Primary: Normalization of albumin-adjusted serum calcium while independent from active vitamin D and calcium supplements (<600 mg/day) at Week 12 Se
Slide 19: MBX 1416: Long-Acting GLP-1 Receptor Antagonist for Post-bariatric Hypoglycemia
MBX 1416: Long-Acting GLP-1 Receptor Antagonist for Post-bariatric Hypoglycemia
Slide 20: Post-bariatric Hypoglycemia: a Rare, Serious and Chronic Complication of Bariatric Surgery
Post-bariatric Hypoglycemia (PBH): a Rare, Serious and Chronic Complication of Bariatric Surgery 1. Fischer et al Surg Obes Relat Dis. 2021, 2. Michaels et al Obes Surg. 2017, 3. Lee et al Obesity 2015, 4. Internal Estimates Estimated >90,000 patients in U.S.1,2,3,4 Severe Hypoglycemia Neuroglycopen
Slide 21: Managing PBH: No Currently Approved Pharmacotherapies
Managing PBH: No Currently Approved Pharmacotherapies 1. Suhl E et al Surg Obes Relat Dis 2017, 2. Salehi M et al JCEM 2018 Includes restricted diet, off-label medications and surgery Once-daily PBH investigational therapy Once-weekly PBH investigational therapy STANDARD OF CARE1,2 Frequent, small m
Slide 22: MBX 1416 Phase 1 Trial
MBX 1416 Phase 1 Trial Endpoints Primary: Secondary: To evaluate safety and tolerability Pharmacokinetic (PK) profile of MBX 1416 Pharmacodynamic (PD) – Response to Mixed Meal Tolerance Test (MAD) SAD (n=32) COMPLETE MAD (n=23) COMPLETE n=8 subjects/cohort (2 placebo: 6 active) Treatments: placebo o
Slide 23: Overall Summary of Adverse Events
Overall Summary of Adverse Events Single dose cohorts (SAD and DDI): No Grade 3 or 4 AEs Multiple dose cohorts (MAD): No Grade 4 AEs; 5 subjects (4 subjects in the 30 mg and 1 subject in the 10 mg cohorts) experienced Grade 3 AEs (all ISRs) after the 3rd or 4th dose based only on erythema at the inj
Slide 24: Injection Site Reactions (ISRs) Assessment Summary
Injection Site Reactions (ISRs) Assessment Summary Given injectable peptides are known to cause ISRs, we proactively collected all ISRs, including mild reactions, using a dedicated form and the FDA Toxicity Grading Scale ISRs were observed in 73% of subjects who received MBX 1416 compared to 0% of s
Slide 25: Pharmacokinetics of Single and Multiple Doses of MBX 1416
Pharmacokinetics of Single and Multiple Doses of MBX 1416 MBX 1416 exposures were reproduceable and increased dose-proportionally Half-life of approximately 90 hours, supporting once-weekly dosing MBX 1416 concentrations increased dose-proportionally over the 10 to 200 mg dose range Steady-state a
Slide 26: Pharmacodynamic Effect of MBX 1416 Observed During MMTT
Pharmacodynamic Effect of MBX 1416 Observed During MMTT Increase in GLP-1 reaches statistical significance at early timepoints on Day 9 and Day 23 As expected in healthy subjects, no meaningful changes from baseline or between MBX 1416 and placebo were observed in the other parameters measured durin
Slide 27: Key Highlights from Phase 1 Study of MBX 1416
Key Highlights from Phase 1 Study of MBX 1416 MBX 1416 was generally well tolerated with a favorable safety profile No MBX 1416 dose-related serious adverse events were observed, and the majority of TEAEs were mild or moderate injection site reactions MBX 1416 concentrations increased dose-proportio
Slide 28: Obesity Portfolio
Obesity Portfolio
Slide 29: Obesity: a Global Epidemic with Large Potential Commercial Opportunity
Obesity: a Global Epidemic with Large Potential Commercial Opportunity Obese=(BMI>30 kg/m2); Overweight= (BMI 25.0-29.9 kg/m2) 1. 2020 U.S. adult population www.census.gov; 2. www.cdc.gov/nchs/data/hestat/obesity-adult-17-18/obesity-adult.htm MBX scientific founder, Richard DiMarchi, PhD Multiple ca
Slide 30: MBX 4291: Long-acting GLP-1/GIP Receptor Co-agonist Prodrug in Development for Obesity
MBX 4291: Long-acting GLP-1/GIP Receptor Co-agonist Prodrug in Development for Obesity Press Release: Dec. 4, 2024: Lilly's Zepbound® (tirzepatide) superior to Wegovy® (semaglutide) in head-to-head trial showing an average weight loss of 20.2% vs. 13.7% Designed as a high potency GLP-1/GIP receptor
Slide 31: In vitro Receptor Binding of MBX 4291 Active Drug vs Tirzepatide
In vitro Receptor Binding of MBX 4291 Active Drug has Similar Activity in Binding to the GLP-1 and GIP Receptors as Tirzepatide1 1. tirzepatide, a GLP-1/GIP co-agonist, is marketed as Zepbound® for the treatment of obesity hGLP-1R hGIPR tirzepatide MBX 4291 (active drug) CPS (mean) Concentration, nM
Slide 32: MBX 4291 Extended Time-action Profile in NHPs Compared to Tirzepatide
MBX 4291 Active Drug Demonstrated Extended Time-action Profile in NHPs Compared to Tirzepatide that may Support Once-monthly Dosing NHP = non-human primates TZP = tirzepatide
Slide 33: MBX 4291 Infusion-like Profile in Once-weekly Repeat Administration in NHPs
MBX 4291 Infusion-like Profile Demonstrated in Once-weekly Repeat Administration in NHPs NHP = non-human primates
Slide 34: MBX 4291 Reductions in Body Weight and Food Intake in DIO Mouse Model
Bodyweight (g) (mean) Time (Days) Time (Days) MBX 4291 Active Drug Produced Similar Reductions in Body Weight and Food Intake as Tirzepatide in DIO Mouse Model DIO = diet-induced obesity Dosing = 3 nmol/kg daily Days 0-4, 1.5 nmol/kg daily Days 5-10 Vehicle tirzepatide MBX 4291 (active drug) Bodywei
Slide 35: MBX 4291 Demonstrated Significant Weight Loss vs. Baseline in NHPs
MBX 4291 Demonstrated Significant Weight Loss vs. Baseline in NHPs NHP = non-human primates 1
Slide 36: Substantial Value Inflection Opportunities in 2025
PROGRAM MILESTONE ANTICIPATED TIMING Canvuparatide (MBX 2109) Avail™ (Phase 2) Enrollment Completion Complete Avail™ (Phase 2) Topline Results Q3 2025 MBX 1416 Phase 1 Topline Results Complete End of Phase 1 Meeting Mid-2025 Phase 2 Initiation 2H 2025 MBX 4291 IND Submission Q2 2025 Phase 1 Initiati
Slide 37: Thank You
Thank You www.mbxbio.com investors@mbxbio.com