The BUC just reinforced what he discussed on the TC? GC hasn't backed down, backed off or shied away from everything he's been saying.
Twitter Conference-
WCG : What 3 milestones will occur to allow AMBS to up-list?
GC : LymPro partnership, Eltoprazine first patient in, MANF orphan drug designation.
LymPro Partnership
This services agreement extends over four years and covers LymPro’s central laboratory requirements under CLIA (under CLIA, only 1 laboratory facility can run a Laboratory Developed Test), in addition to consulting support for Eltoprazine’s further clinical development and our MANF program as we move it towards human trials....
With this agreement, we also gain access to a global distribution network for LymPro, including central laboratories in Europe, Singapore, India and China that will allow LymPro to enter these new markets seamlessly through an already established distribution framework.
JN, CFA: Any thoughts you can share on the design of the P2 #eltoprazine study? Size? Location? Endpoints?
Phase 2b trial, 200 patients, Europe & US, Unified Dyskinesia Rating Scale.
JN, CFA: Does it make sense to pursue to separate Orphan designations on MANF in both RP and Wolframs? Yes.
JN, CFA: What is the timeline for the first Orphan Drug application with MANF?
2H-2014
JN, CFA: Has Orphan Drug designation ever been granted on a preclinical drug? Will the FDA want to see human studies under first?
Yes. The FDA does not need human data to grant ODD.
United States
Orphan Drug Act
Under the law, companies that develop such a drug (a drug for a disorder affecting fewer than 200,000 people in the United States) may sell it without competition for seven years,[3] and may get clinical trial tax incentives.
EU
Orphan drug status granted by the European Commission gives marketing exclusivity in the EU for 10 years after approval.
Other countries
In addition to the United States and the European Union, legislation has been implemented by Japan, Singapore, and Australia that offers subsidies and other incentives to encourage the development of drugs that treat orphan diseases.
What would happen if he announces a Worldwide Partnership for LymPro companion use? (what would BP pay to have worldwide access for their ALZ studies exclusively? Would GC give worldwide exclusivity to just one BP?) It becomes a very different landscape quite quickly.
To say GC hasn't created Shareholder value is misleading a bit. It just hasn't shown itself in share price yet...when will it? To me, looks to be unfolding soon.
Twitter Conference-
WCG : What 3 milestones will occur to allow AMBS to up-list?
GC : LymPro partnership, Eltoprazine first patient in, MANF orphan drug designation.
LymPro Partnership
This services agreement extends over four years and covers LymPro’s central laboratory requirements under CLIA (under CLIA, only 1 laboratory facility can run a Laboratory Developed Test), in addition to consulting support for Eltoprazine’s further clinical development and our MANF program as we move it towards human trials....
With this agreement, we also gain access to a global distribution network for LymPro, including central laboratories in Europe, Singapore, India and China that will allow LymPro to enter these new markets seamlessly through an already established distribution framework.
JN, CFA: Any thoughts you can share on the design of the P2 #eltoprazine study? Size? Location? Endpoints?
Phase 2b trial, 200 patients, Europe & US, Unified Dyskinesia Rating Scale.
JN, CFA: Does it make sense to pursue to separate Orphan designations on MANF in both RP and Wolframs? Yes.
JN, CFA: What is the timeline for the first Orphan Drug application with MANF?
2H-2014
JN, CFA: Has Orphan Drug designation ever been granted on a preclinical drug? Will the FDA want to see human studies under first?
Yes. The FDA does not need human data to grant ODD.
United States
Orphan Drug Act
Under the law, companies that develop such a drug (a drug for a disorder affecting fewer than 200,000 people in the United States) may sell it without competition for seven years,[3] and may get clinical trial tax incentives.
EU
Orphan drug status granted by the European Commission gives marketing exclusivity in the EU for 10 years after approval.
Other countries
In addition to the United States and the European Union, legislation has been implemented by Japan, Singapore, and Australia that offers subsidies and other incentives to encourage the development of drugs that treat orphan diseases.
What would happen if he announces a Worldwide Partnership for LymPro companion use? (what would BP pay to have worldwide access for their ALZ studies exclusively? Would GC give worldwide exclusivity to just one BP?) It becomes a very different landscape quite quickly.
To say GC hasn't created Shareholder value is misleading a bit. It just hasn't shown itself in share price yet...when will it? To me, looks to be unfolding soon.
