A compact summery of presented information by MediGene in the years 2010 - 2012 with personal comments on progress made. Marketed Products: Veregen: - Global progress in 2012 - Approval in additional 17 EU countries (done)
- Price negotiations in Netherlands, France and Romania (ongoing)
- Market launch in Serbia, Israel, Nordic countries and CEE countries (planed)
- Already marketed in US, Germany, Austria, Spain and Switzerland (done)
- Additional Sales Representatives available in US in Q3 / 2012 (done)
- Approvals and market launches in additional countries (ongoing)
- Additional marketing and partnership agreements (ongoing)
- Revenue growth (ongoing)
Eligard: - Full European rights sold for 25 mio. Euro in cash and 2% royalties (done)
- 2% royalty stream monetized in 2012 for 14.1 mio. Euro (done)
Oracea: - Medigene receives up to 24 Mio. Euro successive milestone payments depending on sales revenues achieved by Galderma (awaited)
Pipeline and Business Development Adapt and restructure operations: - Restructuring completed
- Focus on clinical development and CMC (done)
- Focus expenses to reduced project portfolio (done)
- Adapt headcount to reduced project portfolio (done)
- Streamline processes and reporting lines (done)
- Reduce external spending (Spin-Out - done, Studies - ongoing process)
Next Milestones in Business Development: - Adaption of structures - rigorous cost control - reach break even. (ongoing)
- Focus expenses on strategic objectives, i.e. key programs and assets (ongoing)
- Assure sufficient cash to maintain key programs development (Eligard - completely done in 2012)
- Selective additions to oncology pipeline through strategic transactions (planed)
- M&A with complementary Biotech companies (planed as an option)
Clinical Development and CMC Strategy: Leverage liposomal know-how and complement portfolio with selective partnering - Step-wise,value-optimized focus on oncology and immunology (RhuDex, AAVLP, EndoTAG-1) (done)
- Spin-out of all non-core technologies (Immunocore Phase I ongoing, Catherex/Markert - Phase I - M002 / IL-12 expressing HSV-1 planed for 2013) (done)
- EndoTAG-1 Partnering partly done
Focus on EndoTAG-1, Prerequisite to achive attractive deal parameters with potential partner(s) - Evaluation of spray-drying for commercial process (done)
- TNBC to provide second proof of concept (done)
- Prepare commercial manufactoring (CMC) process (done)
New event in 2012 Partner for EndoTAG-1 switch to lead indikationTNBC: - TNBC Phase III development of EndoTAG-1 with partner (planed)
- NDA filing expected for 2018 (planed)
- TNBC Phase III study design already discussed with EMEA. (done)
- Scale-Up of spray drying process for commercial use after approval and in phase III trial (planed)
- Discussions with additional potential partners ongoing for EU / US (planed)
- TNBC Phase II study started - Investigator Initiated Trial (phase II neoadjuvant study) with EndoTAG®-1/paclitaxel combination in HER2-negative breast cancer. (ongoing - results now expected in Q1 2013)
- Perform small explorative phase I/II trials in selected indications and patient populations (planed)
- Leveraging of liposomal technology in other indications (SCLC, Ovarian, esophageal, pancreatic ca - neoadjuvant - Orphan, little competition, high medical need, highly vascularized, chemo-sensitive, fast read-out, check additional combinations p.e. platinium analogue (unknown)
RhuDex: - New RhuDex Formulation successfully developed in 2012 (done)
- Additional non-clinical studies started to support clinical development in RA (ongoing)
- Start prove-of-concept PBC trial end of 2012 with new RhuDex formulation (planed)
- Outlicensing RhuDex after proof of concept in PBC Indication (planed)
AAVLP: - Development of a broadly protective vaccines using AAVLP display technology (ongoing)
- Large markets with possible Indikations in autoimmune disorders, allergic reactions and multiple cancer indications with receptors like L1, HER2, CD20, NY-ESO, MAGE-3
- Partnering planed
Major change in strategy - Pancreatic Cancer Phase III Trial suspended: - Phase III in pancreatic cancer will be launched in H1/2011 using new spray-dried material. (failed)
- PC Phase III development of EndoTAG-1 with partner for launch in 2015 (failed)
- PC Phase III study design finalized and accepted by EMEA & FDA
- PC Phase III Network of clinical opinion leaders and research organization
- High medical need, attractive market potential
- Positive phase II data in pancreatic cancer are a solid base for phase III trial
- Good tolerability for palliative setting
- Favorable time-to-market
- Orphan drug designation
- Feasibility study demonstrated little competition and high interest
- EndoTag-1 Phase III setup of the pivotal Pancreatic Cancer Trial:
- Study design defined
- Medical steering committee established
- Country / site feasibility study completed
- Scientific advice from BfArM, MPA, EMEA, FDA completed
- Study design accepted by EMEA and FDA
- FDA Orphan drug designation for panreatic cancer granted
- Option: Start of a pancreatic cancer study in a neo-adjuvanten Setting (?)
EndoTAG next generation applications (possibly suspended ?): - Explore new product opportunities in further cancer indications
- Combination with novel (IP-protected, high potency) actives having shown clinical proof of concept in oncology
- "Enhancement" of existing activities
- Improve anti-tumor efficacy and safety (improved benefit/risk profile based on more efficient liposomal carrier)
- Expand application Interval (>= two weeks)
- Reduce amount of liposomes per application (Improved API loading capacity)
- Reduce interaction with serum components
- Develop high capacity, robust manufacturing process
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