Biodexa Pharmaceuticals plc · NASDAQ:BDRX
Biodexa Pharmaceuticals — Investor Presentation (June 2024)
28 slides from Biodexa Pharmaceuticals's investor presentation, June 2024
Slide 1: Corporate Presentation
Corporate Presentation NASDAQ:BDRX June 2024
Slide 2: Disclaimer & cautionary language regarding forward-looking statements
Disclaimer & cautionary language regarding forward-looking statements The following presentation, including a hard copy of these slides/the talks given by the presenters, the information communicated during any delivery of the presentation and any question and answer session and any document or mate
Slide 3: Company Strategy
Company Strategy BUILD DEVELOP PARTNER Build pipeline of assets Develop assets through License out to pharma focussed on the oncology next stage/s of validation partners / rare orphan space Progress through to Partners to sponsor Target assets in late pre- completion of phase 1/2 phase 3 programme a
Slide 4: Expanded pipeline
Expanded pipeline Grant Indication Preclinical Phase 1 Phase 2 Phase 3 Funding eRapa Familial Adenomatous Up to approx. (rapamycin) Polyposis (FAP) Orphan $20 million Non-Muscle Invasive eRapa Bladder Cancer $2.8 million (rapamycin) (NMIBC) Tolimidone Type 1 Diabetes (Lyn Kinase activator) MTX110 Di
Slide 5: Rapamycin a/k/a sirolimus
Rapamycin a/k/a sirolimus • Rapamycin is a well characterized and approved mTOR inhibitor • mTOR = mammalian Target Of Rapamycin • mTOR is a central regulator of cell metabolism, growth, proliferation and survival • mTOR pathway is activated during various cellular processes including tumor formatio
Slide 6: eRapa – superior formulation of rapamycin
eRapa – superior formulation of rapamycin Novel dosage form allows wider clinical applications • Unique formulation of rapamycin • Nanoparticles (200-300 nm) • Coated in pH sensitive polymer[s] • By-passing stomach results in: • Enhanced bioavailability / lower dose • Less variable pharmacokinetics
Slide 7: Familial Adenomatous Polyposis (FAP)
Familial Adenomatous Polyposis (FAP) No approved therapy • Orphan Drug Designation • Manifests as mass proliferation of polyps in the gastrointestinal tract, usually in mid-teens • Significant hereditary component (single gene mutation) • If untreated, all patients go on to develop colorectal cancer
Slide 8: eRapa – preclinical: reduced polyp count in FAP model
eRapa – preclinical Reduced polyp count, extended survival in FAP model Polyp count at death eRapa treated mice: 50 • Demonstrated reduced polyp count at death 40 • Experienced limited toxicity Polyp Number 30 0 ppm • Outlived untreated mice 14 ppm 20 42 ppm 10 0 8
Slide 9: Phase 2 in FAP: prevention of progression
Phase 2 in FAP Prevention of progression in FAP 6 month data 256%138% 100 Cohort 1 80 Cohort 2 Change from baseline (%) 60 Cohort 3 PD 40 20 0 SD -20 -40 -60 Progressors Non-progressors n=5/30 (17%) n=25/30 (83%) PR -80 -100 Mean overall polyp burden, 24% reduction, p=0.04 (paired T test) 9
Slide 10: eRapa Phase 3
eRapa Phase 3 Based on successful EOP2 FDA meeting DESIGN FAP PATHOLOGY • Placebo-controlled, double-blind, ~30 sites, (n=140) • Inclusion: high risk pts with germline / phenotypic FAP diagnosis • Stratification: • Intact colon vs post-surgery • Stage 3 / higher duodenal polyposis • Primary endpoint
Slide 11: eRapa – non-orphan indications / clinical studies
eRapa – non-orphan indications / clinical studies Safety profile established, potential in multiple indications PHASE 2 NMI BLADDER CANCER (ongoing) PHASE 1 PROSTATE CANCER (completed) • Placebo-controlled, two centers, (n=166,121 enrolled as of • Open label dose escalation, single center, (n=14) 08
Slide 12: Tolimidone in Type 1 Diabetes
Tolimidone in Type 1 Diabetes The potential to restore β-Cell Function
Slide 13: Type 1 Diabetes Epidemiology and Market Size
Type 1 Diabetes Epidemiology and Market Size The worldwide incidence of type 1 diabetes mellitus (T1DM) has increased in recent decades PREVALENCE/INCIDENCE MARKET 8.4 million Global T1DM market estimated 500,000 People living with T1DM at US$7.59bn in 20223 New cases per year1 worldwide in 20211
Slide 14: Lyn kinase as target in T1D
Lyn kinase as target in T1D • Signaling protein which modulates key intracellular functions such as: • Proliferation • Differentiation • Apoptosis • Migration, and • Metabolism • In fat cells it increases utilization of insulin and thus decreases blood sugar • In pancreatic islets: • activation of l
Slide 15: Tolimidone effects in T1D mouse model
Tolimidone effects in T1D mouse model • Tolimidone increases β-cell mass Improved glucose control in non-obese mouse model (NOS model) with preserved effect after • β-cell function preservation may treatment termination – protective effect of be due to a direct protective tolimidone activity of toli
Slide 16: Clinical development of tolimidone
Clinical development of tolimidone Phase IIa: • Efficacy dose confirmation study. Safety data complemented by earlier Pfizer/Melior studies • N ≈ 12-15 patients, 3 dose/escalations • Runs in parallel with preparations for Phase IIb Phase IIb: • Aiming to open recruitment within 12 months • Double-bl
Slide 17: MTX110
MTX110 (panobinostat with Convection Enhanced Delivery) 17
Slide 18: MTX110 Rationale for panobinostat
MTX110 Rationale for panobinostat • In a screen of 83 drugs panobinostat was among the most effective agents1 • Convection-enhanced delivery (CED) to directly infuse panobinostat into the pons of a DMG orthotopic xenograft model showed: • A marked effect on the rate of DMG xenograft growth1 • Signif
Slide 19: MTX110 Product concept
MTX110 Product concept Delivered directly Drug bypasses into tumor blood brain barrier Re-fillable implanted pump Optimized Therapeutic Dose with Minimal Potential for Systemic Exposure 19
Slide 20: Diffuse Midline Glioma (DMG) Opportunity
MTX110 Diffuse Midline Glioma (DMG) Opportunity • Significant unmet medical need • About 300 children diagnosed in the US pa1, over 1,100 worldwide2 • Median survival of 8-11 months3 • Resection not possible, no effective treatment4 • Phase I programme: • ‘201 study (UCSF) n=7 results published • ‘2
Slide 21: DMG – clinical data from study
MTX110 Diffuse Midline Glioma (DMG) – clinical data from 201 study The median overall survival (OS) for cohort was 26.1 months (95% CI) Median survival based on a cohort of 316 cases was 10.0 months 1 1. Jansen et al, 2015. Neuro-Oncology 17(1):160-166 21
Slide 22: Glioblastoma (GBM) Opportunity
MTX110 Glioblastoma (GBM) Opportunity • Most common and aggressive form of brain cancer in adults • Survival with standard of care treatment ranges from approximately 13 to 30 months depending on MGMT methylation 1. • Standard of care: resection, radiation, limited drug options • Near 100% recurrenc
Slide 23: Glioblastoma (GBM) preclinical data
MTX110 Glioblastoma (GBM) preclinical data 1400 Control TumVolume (mm3) 1200 MTX110 • Problem: 1000 800 • Many drug failures due to poor BBB penetration 600 400 • Potential Solution: 200 • MTX110 is delivered intra-tumorally at high doses via 0 0 5 10 15 20 25 30 35 40 CED, by-passing BBB Days Area
Slide 24: Multiple near-term clinical milestones
Multiple near-term clinical milestones Opportunities for value inflection eRapa Tolimidone Tolimidone eRapa Tolimidone Licensing Initiation of Phase 2a Topline data from Initiation of Phase 3 Initiation of Phase agreement with dose-confirming Phase 2a study in study in FAP 2b study in T1D Emtora stu
Slide 25: Appendix
pharmaceuticals Appendix biodexa 25
Slide 26: MTD217
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Slide 27: MTD217 Leptomeningeal Disease (LMD) Background
MTD217 Leptomeningeal Disease (LMD) Background • Cancer in the cerebrospinal fluid and leptomeninges caused by metastases • Most commonly arises from breast and lung cancers • Approximately 5% of people who have cancer develop1 LMD • No effective treatments currently exist for LMD • With treatment,
Slide 28: MTD217 hypothesis and clinical approach
MTD217 hypothesis and clinical approach Remsik et.al 2020 CANCER REPORTS Literature suggests cancer cells in LMD exist in a metabolic equilibrium Leptomeningeal between adherent and floating states metastatic cells adopt two phenotypic states Hypothesis: Simultaneous OXPHOS inhibition (with water-so
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