Biodexa Pharmaceuticals plc · NASDAQ:BDRX
Biodexa Pharmaceuticals — Ladenburg Thalmann Symposium 2024
21 slides from Biodexa Pharmaceuticals's conference presentation, Dec 12, 2024
Slide 1: Ladenburg Thalmann Symposium
Ladenburg Thalmann Symposium December 12, 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 only and and any document or mat
Slide 3: Highlights
Highlights NASDAQ listed Focused on rare / orphan diseases Funded Phase 3 program in FAP* De-risked, re-purposed molecules Semi-virtual organization Near term milestones – incl. potential “Conditional Approval” in EU 4Q25 * Familial Adenomatous Polyposis 3
Slide 4: Strategy
Strategy BUILD DEVELOP PARTNER Build pipeline of assets Develop assets through License out to pharma focused on: next stage/s of validation partners - rare/orphan - oncology Progress through to Partners to sponsor completion of phase 1/2 phase 3 program and Target assets in late pre- proof-of-concep
Slide 5: 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 6: eRapa
eRapa First to market potential in Familial Adenomatous Polyposis (FAP)
Slide 7: 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 8: Rapamycin – Approved Formulations
Rapamycin – Approved Formulations • Approved in US and Europe in 1999 • Indication: prevention of organ rejection after kidney transplantation • Marketed by Pfizer as Rapamune® • LAM indication added to label in 2015 8
Slide 9: eRapa – Superior Formulation Of Rapamycin
eRapa – Superior Formulation Of Rapamycin Novel dosage form allows wider clinical applications • Unique formulation of rapamycin • Low strength 0.5mg dosage form • Nanoparticles (200-300 nm) • Coated in pH sensitive polymer[s] • Avoids acid degradation in the stomach • By-passing stomach results in:
Slide 10: Familial Adenomatous Polyposis (FAP)
Familial Adenomatous Polyposis (FAP) No approved therapy • Orphan Drug Designation expected in Europe (process underway) • Manifests as mass proliferation of polyps in the gastrointestinal tract, usually in mid-teens • Significant hereditary component (single gene mutation) Colectomy resection speci
Slide 11: Rationale for mTOR as a target in FAP
Rationale for mTOR as a target in FAP • mTOR has a well known role in tumorgenesis • In vivo studies demonstrate the role of mTOR in the Apc pathway • Overexpression of mTOR in FAP polyps • Inhibition of mTOR and suppression of polyp growth in Apc knockout mice • Polyp reduction and restoration of n
Slide 12: Phase 2 in FAP – six month data
Phase 2 in FAP – six month data Prevention of progression in FAP 256%138% Cohort 1 Cohort 2 Cohort 3 100 80 60 PD Change from baseline (%) 40 20 0 SD -20 -40 -60 Progressors Non-progressors PR n=5/30 (17%) n=25/30 (83%) Median Response -80 28% reduction P=0.04 (paired T test) -100 Mean overall polyp
Slide 13: Phase 2 in FAP – 12 month data
Phase 2 in FAP – 12 month data 13
Slide 14: eRapa Phase 2 study – adverse events
eRapa Phase 2 study – adverse events 14
Slide 15: Phase 2 study in FAP - conclusions
Phase 2 study in FAP - conclusions • eRapa is safe with minimal related SAEs or DLTs • Tolerable with 95% compliance at 12 months • 75% non-progression rate at 12 months • Every other week dosing (cohort 2) produced durable response of only 11% progression at 12 months 15
Slide 16: eRapa Phase 3
eRapa Phase 3 Based on successful EOP2 FDA meeting DESIGN TIMING • Placebo-controlled, 2:1 double-blind, ~30 sites, (n=168) • USA submission Q1’ 25 • Inclusion: high risk pts with germline / phenotypic FAP • EU submissions Q2’ 25 diagnosis • FPFV target Q2’ 25 (USA) • 18 mos enrollment • Stratificat
Slide 17: eRapa in Non Muscle Invasive Bladder Cancer
eRapa in Non Muscle Invasive Bladder Cancer • Bladder cancer confined to the inner layers of the bladder without invading deeper muscle tissues • Main treatment involves transurethral resection of bladder tumour (TURBT) usually followed by intravesical Bacillus Calmette-Guérin (BCG) therapy • Ongoin
Slide 18: Business plan
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Slide 19: eRapa partnering strategy
eRapa partnering strategy Potential source of non-dilutive fi nancing • US market most valuable: • European market more complex • Single market / higher prevalence • Most accessible • Initiate partnering outreach at BIO • Japanese BD consultancy appointed • Highest price Europe, Nov 4-6, 2024 • Aim
Slide 20: Upcoming milestones - eRapa
Upcoming milestones - eRapa Tolimidone eRapa eRapa eRapa eRapa Initiation of Phase 2a FAP Orphan Drug Fast Track Target partnerships EMA decision on dose-confirming study Designation Europe Designation in US for Europe / Japan “Conditional in T1D Approval” in FAP eRapa eRapa eRapa Type C meeting wit
Slide 21: EMA Conditional approval guidelines
EMA Conditional approval guidelines 21
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