Slides
Page 1
Pioneering Precision Peptides for Endocrine and Metabolic Diseases January 2026 J.P. Morgan Healthcare Conference
Page 2
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, future results of operations and financial position, strategy and plans, industry environment, potential growth opportunities, and our expectations for future operations, are forward looking statements The words “ believe,” “ may,” “will,” “ estimate,” “ continue,” “ anticipate,” “ design,” “ “ expect,” “ could,” “ plan,” “ potential,” “ predict,” “ seek,” “ should,” “would,” or the negative version of these words and similar expressions are intended to identify forward looking statements. We have based these forward-looking statements on our current expectations and projections about future events and trends that we believe may affect our financial condition, results of operations, strategy, short- and long- term business operations and objectives, and financial needs. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including but not limited to, our ability to develop and advance our programs and product candidates, our regulatory approvals and filings, and other risks, uncertainties and assumptions identified in our filings with the Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment. New risks emerge from time to time, and it is not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward looking statements we may make. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this presentation may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward looking statements as predictions of future events. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither we nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements. We undertake no obligation to update publicly any forward-looking statements for any reason after the date of this presentation to conform these statements to actual results or to changes in our expectations, unless required by law. This presentation contains estimates and other information concerning our industry, our business and the markets for our product candidates. Information that is based on estimates, market research or similar methodologies, including prevalence studies which are extrapolated to broader populations, is inherently subject to uncertainties, and actual events or circumstances may differ materially from events and circumstances that are assumed in this information. Unless otherwise expressly stated, we obtained this industry, business, market and other data from our own internal estimates and research as well as from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data and similar sources. Industry publications and surveys generally state that the information contained therein has been obtained from sources believed to be reliable. Although we believe the industry and market data to be reliable as of the date of this presentation, this information could prove to be inaccurate. Industry and market data could be wrong because of the method by which sources obtained their data and because information cannot always be verified with complete certainty due to the limits on the availability and reliability of raw data, the voluntary nature of the data gathering process and other limitations and uncertainties. While we are responsible for the accuracy of such information and believe our internal company research as to such matters is reliable and the market definitions are appropriate, neither such research nor these definitions have been verified by any independent source.
Page 3
$373.7 million in cash provides runway into 20291 Program Milestone Anticipated Timing Canvuparatide (MBX 2109) End-of-Phase 2 FDA Meeting Q1 2026 Avail Phase 2 presentation and one-year OLE data Q2 2026 Phase 3: Initiation Q3 2026 MBX 4291 (GLP-1/GIP) Phase 1: 12-week MAD results Q4 2026 MBX 5XXX (amycretin) Nominate development candidate Q2 2026 MBX 6XXX (GLP-1/GIP/GCGR) Nominate development candidate Q3 2026 Imapextide (MBX 1416) STEADI Phase 2a: Results Q2 2026 1 Unaudited cash, cash equivalents and marketable securities as of December 31, 2025 MBX: Catalyst-Rich Year Ahead
Page 4
Clinically Validated Precision Endocrine Peptide (PEP ) Platform Created by MBX and Scientific Founder Richard DiMarchi, PhD INNOVATIVE PEPTIDE DESIGN With a goal to optimize: • Multiple mechanisms of action within a single peptide • Increased potency • Enhanced physical properties, including stability and solubility PROGRAMMABLE PRODRUG Designed to provide: • Gradual, controlled release of active drug • Slow rise to maximum exposure • Flattened exposure FATTY ACYLATION With a goal to optimize: • Longer time action • More convenient dosing Combining PEP technologies to deliver differentiated and best-in-class medicines for patients
Page 5
Canvuparatide Investigational Once-Weekly PTH Replacement Therapy for Hypoparathyroidism
Page 6
Chronic Hypoparathyroidism: A Serious Endocrine Disease Affecting More than 250,000 Patients in the U.S. and Europe • Conventional therapy includes large and frequent doses of calcium and active vitamin D • Majority of patients are insufficiently managed and may experience: • Cramps • Fatigue • Seizures • Anxiety or depression • Long-term complications including kidney stones and chronic kidney disease Source:Mannstadt M, et al. Nat Rev Dis Primers. 2017;3:17055. Vadiveloo T, et al. J Bone Miner Res. 2018;33(3):478-485. Clarke BL, et al. J Clin Endocrinol Metab. 2016;101(6):2284-99. Powers J, et al. J Bone Miner Res. 2013;28(12):2570-6. Underbjerg L, et al. J Bone Miner Res. 2013;28(11):2277-85. Soibelman D, Ronen O. Clin Otolaryngol. 2025;50(2):205-219. Cianferotti L, et al. Calcif Tissue Int. 2018;103(2):144-150. Swartling O, et al. J Clin Endocrinol Metab. 2022;107(10):e4098-e4105.
Page 7
Canvuparatide Data Support Potential Best-in-Class Profile for Hypoparathyroidism • All patients completed the 12-week Avail trial and 94% entered the 2-year open-label extension (OLE) • Once-weekly canvuparatide was well tolerated, with no treatment-related serious adverse events or discontinuations during the 12-week trial • 96% of responders at Week 12 remained responders at 6 months2 1 Analysis based on patients with available data for each component of the composite criteria, defined as independence from active vitamin D, independence from oral calcium (≤600 mg/day), and serum AdjCa within the normal range (8.2–10.6 mg/dL) at 6 months. 2 Based on patients with available data (23 out of 24) Initiation of Phase 3 trial anticipated in Q3 2026 63% Responder Rate at Week 12 in Avail* *Statistically significant vs. placebo 79% Responder Rate at 6 Months in OLE1
Page 8
Once-Weekly Dosing Drives Preference for HCPs and HP Patients 80% 100% of Endocrinologists chose once-weekly over daily 90% of Nurses chose once-weekly over daily 100% of HP Patients chose once-weekly over daily of endocrinologists said they would be “extremely likely” to switch from daily to once-weekly if it were approved/available HP = Hypoparathyroidism Source: Primary US market research study (n=10 endocrinologists, n=10 nurses responsible for injection training and initiation, n=20 HP patients); conducted by Bold Insight; August - September 2024; assumes otherwise same product profile
Page 9
• Significant milestones upcoming, including one- year follow-up data in Q2 2026 • Phase 3 trial preparations underway, initiation anticipated in Q3 2026 • Commercial readiness preparation ongoing • MBX retains global commercial rights, patent protection to 2041 and beyond Once-Weekly Canvuparatide: Paving the Way for a Potential New Standard of Care in Hypoparathyroidism
Page 10
Obesity Portfolio
Page 11
Obesity Opportunity: Once-Monthly Dosing with Improved Tolerability MBX obesity candidates are designed using proprietary PEP platform for once-monthly dosing with the goal of more gradual, flattened and sustained exposure and improved tolerability Source for tirzepatide concentrations: CPT Pharmacometrics Syst Pharmacol. 2024 Mar;13(3):494-503. Tirzepatide is the active ingredient in Zepbound. Source for MET-0971i concentrations: Metsera, Inc. Form S-1, filed January 10, 2025. T1/2Cmax calculated as time to 50% of Cmax 0 7 14 21 28 35 10 100 1,000 Time (days) Concentration (ng/mL) Tirzepatide MET-097i (GLP-1 Monoagonist) MBX Target Product Profile T1/2Cmax ~ 5 to 6 days T1/2Cmax ~ 20 to 21 days T1/2Cmax > 21 days
Page 12
Pipeline Designed to Address Broad Range of Obesity Patient Needs ✓ Significant weight loss ✓ Differentiated mechanism of action ✓ Improved convenience MBX 4291 GLP-1/GIP Agonist ✓ Weight loss with improved tolerability ✓ Improved convenience MBX 5XXX Amycretin MBX is developing a robust obesity portfolio with potential to drive strong optionality across patient segments ✓ Significant weight loss ✓ Muscle preservation ✓ Differentiated mechanism of action ✓ Improved convenience MBX 6XXX GLP-1/GIP/GCGR Agonist
Page 13
MBX 4291: Engineered for Gradual Release, Long Exposure and Dual GLP-1/GIP Agonism MBX scientific founder, Richard DiMarchi, PhD
Page 14
MBX 4291 Proof of Concept: Flattened and Steady Exposure 1 2 3 4 5 6 7 8 0.001 0.01 0.1 1 22 23 24 25 26 27 28 29 Time (Days) Concentration (μM) 0.4323 mg/kg MBX 4291 (Active) 1.33 mg/kg MBX 4291 (Active) 4.33 mg/kg MBX 4291 (Active) 13.3 mg/kg MBX 4291 (Active) Week 1 Week 4 Mean Concentration (µM) Once-weekly dosing in non-human primates
Page 15
MBX 4291: 12-Week MAD Data with Once-Monthly Dosing on Track for Q4 2026 15 Primary: • Establish safety and tolerability focusing on competitive gastrointestinal tolerability and streamlined dose titration Secondary: • Determine pharmacokinetics suitable for monthly injection schedule • Demonstrate pharmacodynamics (i.e., weight loss) • Identify doses and titration regimen for Phase 2 * May add a second cohort to evaluate additional doses/dosing regimens Participants BMI ≥ 30 Design N = 30 Cohorts 1* MBX 4291 N = 20 Placebo N = 10 Weekly dosing followed by once-monthly dosing Endpoints12-Week Multiple Ascending Dose (MAD)
Page 16
New Development Candidates Aimed at Exciting Obesity Targets Amycretin Nomination Q2 2026 GLP-1/GIP/GCGR Nomination Q3 2026 Single dose administration evaluated by MBX in an in vivo model in non-human primates (NHPs); Represents active drug concentrations Designed to address full spectrum of disease, each with once-monthly dosing
Page 17
Imapextide (MBX 1416) Long-Acting GLP-1 Receptor Antagonist for Post-Bariatric Hypoglycemia
Page 18
Post-Bariatric Hypoglycemia (PBH): A Rare, Serious and Chronic Complication of Bariatric Surgery Estimated >125,000 patients in U.S.1,2,3,4 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 18 • Rapid transit of nutrients into intestines stimulates excess GLP-1 and insulin secretion; occurs after a meal • PBH presents six months to years after roux-en-Y gastric bypass and sleeve gastrectomy CAUSE1,2 • Severe Hypoglycemia • Neuroglycopenia symptoms including seizures, loss of consciousness, confusion, weakness, dizziness and blurred vision SYMPTOMS • Unpredictable timing and frequency • Social isolation • Diminished quality of life, including disability • Glucagon injection may be required PATIENT IMPACT
Page 19
• Once-weekly imapextide designed to: ✓ Provide daily and nightly prevention of severe hypoglycemia and associated risks ✓ Offer convenient weekly dosing ✓ Improve quality of life ✓ Eliminate need for rescue therapy (glucagon) and surgical intervention • Data from Phase 2a STEADI trial anticipated in Q2 Imapextide: A Potential Best-in-Class Therapy for PBH No Currently Approved Treatments
Page 20
Once-Weekly Dosing Drives Preference for HCPs and PBH Patients Source: Primary research based on blinded target product profile (n=11 endocrinologists, n=12 PBH patients) 70% of Endocrinologists chose once-weekly over daily 92% of Patients chose once-weekly over daily 73% of Patients would switch from daily if given chance
Page 21
MBX: Catalyst-Rich Year Ahead $373.7 million in cash provides runway into 20291 Program Milestone Anticipated Timing Canvuparatide (MBX 2109) End of Phase 2 FDA Meeting Q1 2026 Avail Phase 2 presentation and one-year OLE data Q2 2026 Phase 3: Initiation Q3 2026 MBX 4291 (GLP-1/GIP) Phase 1: 12-week MAD results Q4 2026 MBX 5XXX (amycretin) Nominate development candidate Q2 2026 MBX 6XXX (GLP-1/GIP/GCGR) Nominate development candidate Q3 2026 Imapextide (MBX 1416) STEADI Phase 2a: Results Q2 2026 1 Unaudited cash, cash equivalents and marketable securities as of December 31, 2025
Page 22
Thank You www.mbxbio.com investors@mbxbio.com www.mbxbio.com investors@mbxbio.com