Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Merger or Acquisition (December 2024)
24 slides from Pharming Group Nv's merger presentation, Dec 16, 2024
Slide 1: Proposed Acquisition of Abliva AB
Pharming Group N.V. Proposed Acquisition of Abliva AB December 16, 2024 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 2: Pharming to Acquire Abliva AB
Pharming to Acquire Abliva AB Strategic Rationale and Offer Sijmen de Vries, Chief Executive Officer KL1333 for mtDNA Mitochondrial Disease Anurag Relan, Chief Medical Officer Commercial Overview Stephen Toor, Chief Commercial Officer Conclusion Sijmen de Vries, Chief Executive Officer Q&A 2
Slide 3: Forward-looking statements
Forward-looking statements This presentation may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual resu
Slide 4: Strategic Rationale and Offer
CEO Sijmen de Vries, MD Chief Executive Officer Strategic Rationale and Offer
Slide 5: Building a Leading Global Rare Disease Biopharma Company
Building a Leading Global Rare Disease Biopharma Company Market RUCONEST® for acute Global approvals and Ongoing pipeline development HAE attacks in key markets – commercialization of Joenja® and management of rare U.S. focus (leniolisib) for ADPS disease assets Positive cash flow from RUCONEST® Suc
Slide 6: Acquisition Terms and Financial Details
Acquisition Terms and Financial Details Acquisition Terms Financial Details Timing Acquisition through a public tender Acquisition of shares with available The acceptance period is expected offer under Swedish Takeover Act cash to commence on or around January and Nasdaq Stockholm Takeover 16, 2025,
Slide 7: KL1333 Adds Value to Pharming's Pipeline
KL1333 Adds Value to Pharming’s Pipeline 7
Slide 8: KL1333 for mtDNA mitochondrial disease
CMO Anurag Relan, MD Chief Medical Officer KL1333 for mtDNA mitochondrial disease
Slide 9: Attractive Rare Disease Opportunity Fits Pharming's Business Model
Attractive Rare Disease Opportunity Fits Pharming’s Business Model Mitochondria are the “powerhouses” of cells • Producing chemical energy to power the human body, in particular muscle cells Primary mitochondrial diseases are rare disorders affecting the mitochondria’s ability to generate energy • P
Slide 10: Dysfunctional Mitochondria Produce Less ATP
Dysfunctional Mitochondria Produce Less ATP Primary Mitochondrial Disease (PMD) Mitochondria, often described as the “powerhouses” Dysfunctional Mitochondria of cells, are crucial for energy production Mutation Impaired ETC protein expression Mitochondrial diseases are a group of genetic disorders c
Slide 11: Heavy Patient Burden with No Approved Therapies
Heavy Patient Burden with No Approved Therapies Presentation and Diagnosis Patients present to their primary care doctor and then often get referred to a neurologist for musculoskeletal issues Either the neurologist or a referral to a metabolic geneticist will result in a diagnosis Many patients are
Slide 12: KL1333 Corrects the Underlying Pathophysiology
KL1333 Corrects the Underlying Pathophysiology KL1333 normalizes conversation of NADH to NAD+ via NQO1 Normalizes the NAD+/NADH Ratio Restored Energy Metabolism Electron Transport Chain Complex III Complex IV Mitochondrial Membrane Q Synthase ATP Complex I Complex II X NADH NAD+ ADP + Pi ATP Restore
Slide 13: KL1333: First-in-Disease Small Molecule with Unique MOA
KL1333: First-in-Disease Small Molecule with Unique MOA Attributes Outcomes Directly increases the NAD+/NADH ratio Improved energy regulation and ETC via NQO1 function Unique MoA works upstream from all competing MoA in PMD Stimulation of mitochondria biogenesis Oral, small molecule, BID dosing Fati
Slide 14: Phase 1b Demonstrated Significant Activity vs. Placebo
Phase 1b Demonstrated Significant Activity vs. Placebo The placebo-controlled Phase 1b study demonstrated that KL1333 reduced patients’ fatigue and myopathy after only 10 days, 50 mg/day Fatigue Reduction Muscle Function Improvement Changes from baseline to day 10 Changes from baseline to day 10 KL1
Slide 15: Pivotal Study Design Based on Regulatory and Patient Advocacy Input
Pivotal study Design Based on Regulatory and Patient Advocacy Input Study Schematic Regulatory Feedback Study Design Screening Both FDA and EMA accepted Methodology study as registrational • Randomized, double-blind, parallel-group, placebo- FDA said achieving one of the controlled pivotal study 3:2
Slide 16: FALCON Study – Positive Interim Analysis
FALCON Study – Positive Interim Analysis Pivotal FALCON Study Interim Futility Analysis: Positive outcome achieved, with both primary endpoints having WAVE 1 – Fully enrolled passed futility 40 patients recruited across six countries (U.S., UK, France, Spain, Belgium, Denmark) Promising differences
Slide 17: Commercial overview
CCO Stephen Toor Chief Commercial Officer Commercial overview
Slide 18: First-In-Disease Rare Disease Therapy with Blockbuster Potential
First-In-Disease Rare Disease Therapy with Blockbuster Potential Builds on Pharming’s existing rare disease expertise and infrastructure KL1333 positioned to become first standard of care in mtDNA mitochondrial disease Large commercial opportunity with >30,000 mtDNA diagnosed patients in US, EU4 and
Slide 19: Significant Revenue Opportunity for KL1333
Significant Revenue Opportunity for KL1333 PMD Prevalence 1 per 4,3001 US 78,200; EU4 and UK 76,400 Diagnosed mtDNA 9.6 per 100,0002 US 32,300; EU4 and UK 31,600 FALCON Trial Inclusion Criteria* 51% US 16,500; EU4 and UK 16,100 >30,000 diagnosed mtDNA mitochondrial disease patients addressable in th
Slide 20: Majority of Patients Diagnosed and Treated in US Centers of Excellence
Majority of Patients Diagnosed and Treated in US Centers of Excellence or Academic Institutions Mitochondrial Care Network – Centers: www.mitonetwork.org/centers 20
Slide 21: Conclusion
CEO Sijmen de Vries, MD Chief Executive Officer Conclusion
Slide 22: Building a Leading Global Rare Disease Biopharma Company
Building a Leading Global Rare Disease Biopharma Company Market RUCONEST® for acute Global approvals and Ongoing pipeline development HAE attacks in key markets – commercialization of Joenja® and management of rare U.S. focus (leniolisib) for ADPS disease assets Positive cash flow from RUCONEST® Suc
Slide 23: Q&A
Q&A Sijmen de Vries, MD Anurag Relan, MD Stephen Toor Chief Executive Officer Chief Medical Officer Chief Commercial Officer
Slide 24: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
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