Presentation on theme: "MIGSYS Ian Evans, Genetics Max Musicant, SOM Grant Patterson, EPH Jia Kang, Med Informatics Colin Shaw, Nursing YBPS Case Competition November 20, 2009."— Presentation transcript:
MIGSYS Ian Evans, Genetics Max Musicant, SOM Grant Patterson, EPH Jia Kang, Med Informatics Colin Shaw, Nursing YBPS Case Competition November 20, 2009
Opportunity 2: Science Behind GTX-001 Mechanism of Action: mAb against 1 1 integrin Identification High Throughput Screening Target expression is more T H 1 specific (question of use in UC patients) In Vitro Abolished Interaction with Collagen In Vivo IV toxicity at 6mg/kg Effective at 2mg/kg every other day Reduced inflammation at 4-6 weeks
Released in 2004 for MS, Tysabri was then removed from market in 2005 due to cases of PML Reintroduced in 2005 and since restricted to smaller patient segment – To date 24 cases of PML found in Tysabri users GTX-001 is in the same class as Tysabri – mAb against 4-integrin – Decreases extravasation PML (Progressive Mulitfocal Leukoencephalopathy): a rare neurological disease Due Diligence
Recommendation: GTX-002 Pros – A new class of kinase inhibitor Cons – Existing market well served by many products – Low market prices for immunomodulators – Signs of side effects in pre-clinical studies – Financial models show inviability Recommendation: NO GO
Recommendation: GTX-001 Pros – Large moderate and severe segments are underserved – Biologics command high prices in market – Large therapeutic window – Financial model shows significant profitability Cons – Similar mechanism to existing treatment (Tysabri) Significant medical risk associated with this class of molecules Recommendation: NO GO
Thank You from MIGSYS Ian Evans, Genetics Max Musicant, SOM Grant Patterson, EPH Jia Kang, Med Informatics Colin Shaw, Nursing
Tysabri, A Risky Proposition PML in patients who have received at least 24 infusions ranges from 0.4 to 1.3 per 1,000 in MS patients CD patients make up only 2% of the Tysabri users EMEA rejected Tysabri twice in 2007 Approved for the indication of CD in 2008 Strong warnings not to use Tysabri with PML in family history Case Report: N Engl J Med 2005;353:362-8. Previous FDA Alert: [issued 2/2006] The FDA has lifted the clinical hold on Biogen-IDEC's trials of natalizumab for patients with multiple sclerosis (MS). Biogen-IDEC can now resume administration of natalizumab to patients with relapsing-remitting MS who had previously been treated with the drug in clinical trials. Biogen-IDEC had previously suspended marketing of natalizumab and all further dosing of patients in on-going clinical trials. This decision was made after confirmation of one fatal case and one additional case of severely disabling progressive multifocal leukoencephalopathy (PML) in patients receiving natalizumab for MS. A third case of PML, this one fatal, in a patient with Crohn's Disease had been identified shortly thereafter.