Email Response #2

All-

I started this morning at 4:45 a.m., first answering emails to patients and their families.  I noticed a remarkable number of emails coming from Seattle, Washington; hence, I thought I would send a more lengthy reply to the Seattle Mito Family as a group.

First- thank you. Your vocal support and advocacy is great and well-needed, exactly what I would do. It is the squeaky wheel that gets the oil, and it was exactly that type of passion that resulted in Edison Pharmaceuticals being started as a patient- and physician-founded company. "We cannot and will not wait in line for a treatment– period!" I hear and applaud your message.

Second, on to your question as to the delay at Seattle. Simple truth is that we never expected EPI-743 to exhibit the promising results, albeit preliminary ones, that have been generated to date. We initiated a very closely watched and controlled treatment in one little girl ~ 2 years ago, and from that we are now at the request of FDA treating close to 85 children worldwide with a variety of inherited respiratory chain diseases. Dr. Enns - who is the principal investigator of this study - has presented some of the results at the UMDF meeting with our extended EPI-743 team. This data has just been tallied and submitted for publication.

Edison has been working very closely with the FDA, physicians, and hospital administrators and ethics review boards to expand access to EPI-743. There are three aspects that must be addressed to make this a success. We must have in place the appropriate safety guidelines set forth by the FDA and oversight at each institution; we must manufacture drug according to very strict specifications; and we must agree on budgets at each institution and contracts that are lengthy and unfortunately expensive.

As many of you know Edison is not a typical pharmaceutical company. Our mission is defined by our tag line--- "more tomorrows." We were founded by patient families who were/are blessed to have the financial resources to fund such an endeavor, and today we continue to only "invite" investors who share our singular focus… treatment for "our kids." In the last weeks, we have raised a considerable amount of money to fund studies in Seattle and worldwide, and we are working with FDA as recently as today is gaining their go-ahead to expedite treatment and pivotal clinical trials hopefully paving the way for a 1st approved drug for mito disease. We will not stop our mission there- we will work until we exhaust all of our talents and resources, and until all mito kids and adults have the potential for clinically meaningful treatment. 

We will not hide behind any excuses at Edison- too much is at stake. We know we can never move as fast as not only we want, but as you and your doctors and families want. The Seattle Children's group has been tremendous, but the fact is each institution has their polices and procedures, as so does Edison, and we both must respect them, and navigate them, and come to an agreement on all aspects of oversight, review and budget before we can begin treating patients.

As of today's discussion with administrators at Seattle Children's we have an agreed upon budget, and ALL roadblocks from Edison's side have been resolved. Next steps are for Seattle Children's hospital and Dr. Saneto's team to mobilize - then  a first subject can start. We are awaiting a response from Seattle Children's on their time estimates for this process. I have copied Lorraine Gilmore and will ask her to schedule a group conference call next Monday so I can field any open questions folks may have.

Thank you again for your advocacy and vocal championing of your children. We will need this and very much more to be successful. 

Best/g

Guy Miller, MD, PhD
Chairman, CEO
Edison Pharmaceuticals, Inc







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A great big THANKS to all who joined me in writing a letter.
I even got emails from people that don't have mito in their families, but felt compelled to help anyways. I appreciate each and every one of you who took the time to help us out in this matter. I guess we will see what happens here in Seattle....



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Just got this from Childrens's after I forwarded Dr. Millers response....



Ohmygosh, Girl, I think you did it!!!!!  I mean, it’s not just “coincidence” that they decide this today after all the letters from here!
 
Russ is going to “mobilize his team”!!  Stay tuned!!
 




I did get a follow up email that Dr. S mobilizing his team will take a little time, but sounds like we definitely got things pushed ahead! Wahhhoooo!!!! This is such great news!