I got an e.mail from Summit with an updated Q&A of SMT C 1100.
These are the main two questions:
. When could patient trials commence
If the Phase I trial in healthy volunteers is successful, a patient study could start during
2013 although will still be dependent on a number of other factors including ensuring the
design of the study is correct and financing.
Where will patient studies take place?
At this stage, it is not known where any subsequent patient studies will take place although
it could involve a number of countries. The key will be to ensure the design of the patient
trials will provide SMT C1100 with the best possible chance of succeeding and being made
available to all DMD patients as quickly as possible. In addition the harmonisation
programme between the US and European regulators means that trial data from one region
can be used in applications in the other.
Summit was one of the presenter in PPMD'S annual conference.I heard the presentation through the livestreaming.(thanks to PPMD)Before coming there they were also present in Boston's Bio conference for partnering issue.
Sharon, do you have any idea whether or not they got financing for ph 2 study. Considering the fact that they already reached the milestone in the ph1 and in dose escalating part.
I am afraid if they have to depend on partner for ph2 financing, the partner may not be as committed as Summit ( We all aware of Biomarin case) and will take their own time.So, it will be better if they get funding from inside DMD community.Do you have any proposal from Summit?
I sincerely believe that PPMD is not only for exon skipping boys.My son will never ever benefited from exon skipping as he has a long chain deletion.Exon skipping can cover 70% to 80% of boys when all skippable exons will be there in trial.I am not sure how many % of boys are there who's only hope is UTROPHIN.
So, my son's disease modifying treatment could be SMT C 1100.Other two utrophin drug i.e Biglycan and PTC'S are still in preclinical stage.
Thank you for posting this update. Good questions, too.
I received the following attachment from my questions to Summit.
So, my point is that what we parents could do at this point to ensure ph2 as quickly as possible.This is the universal disease modifying treatment and could be the safest drug as it's oral formulation.As it is small molecule drug it can easily pass regulatory hurdle.