Genentech update on T-DMI early access program

[chrisy]

Genentech has informed us that screening of new patients for enrollment
in T-PAS (T-DM1 Patient Access Study) will be discontinued on
September 6, 2011. Please read this message from Genentech for details:

"This is a difficult decision, but we believe continuing with T-PAS enrollment
would make it difficult to enroll the clinical trials and collect the overall
survival data we believe will be needed for full trastuzumab emtansine (TDM1)
approval in the future. People currently enrolled in T-PAS will
continue to receive trastuzumab emtansine per the protocol.
In addition, Genentech is opening a new Phase III, registrational clinical
trial for people with previously treated HER2-positive mBC (third-line
setting, known as the TH3RESA clinical trial). Screening of new patients in
T-PAS will be discontinued on September 6. This coincides with the
opening of enrollment of the third line trial, TH3RESA, for at least one U.S.
site. TH3RESA will help support FDA approval of trastuzumab emtansine,
as it is designed to confirm if trastuzumab emtansine can improve the
survival of people who have already been treated with other HER2-
targeted medicines. Closing new enrollment in T-PAS was a difficult
decision. However, we believe it is in the best interest of the majority of
people with HER2-positive mBC, as this is necessary to help us conduct
the clinical trials needed to support FDA approval so that access to T-DM1
will not be an issue."

[chrisy]

Some additional, bottom line info not clear from the "release" above and not ALL bad news:

1. Patients who ENTER screening for a T-PAS (EAP) before September 6 will be able to enter the T-PAS if they qualify. So if you're on the fence, my advice is get the ball rolling now, there is still a chance to get in.

2. Gene is still working on amendments to the T-PAS so that patients who are IN one of the EAP studies can roll over into an extension study or another study and continue to have access to T-DM1 if they are stable and tolerating treatment with T-DM1. The effect of this is there will be about double the number of trial sites, thus reducing the travel burden especially for Boston/NY and Northwest area patients.

3. The new 3rd line MBC study (TH3RESA) which will open in August/September has the same "inclusion" criteria as the T-PAS did (prior anthracycline, taxane, capecitabine and herceptin and tykerb regimens). The main difference is that this is a randomized study so patients might not be randomized to the T-DM1 arm. BUT it is a 2:1 randomization so you have a 2 in 3 chance instead of 50/50.

Overall my personal opinion is that I do understand that the requirement to show OS (overall survival) benefit complicates efforts to give patients access - and that the main goal is to not jeopardize the ultimate approval of TDM1. For me, the finger points to the FDA as the barrier to access although I know there are others who disagree.

However I am exceedingly disappointed and frustrated that this means additional delays and reduced access for those who need it NOW, not in another 3 years!

[schoonder]

New 2-arm multi-center T-DM1 trial set to get underway by September.

http://www.clinicaltrials.gov/ct2/sh...m=t-dm1&rank=9

[StephN]

This may be the new study I heard about yesterday at my followup visit to Seattle Cancer Care Alliance.

But I understood that it is for FIRST LINE mets treatment. So, perhaps I got it wrong or this is not the same trial.

[hutchibk]

The trial Chrisy linked is the new phase 3 that will start enrolling soon and opening soon I believe. It will be available in many many locations.

I have initiated inquiries already this week w/ folks about moving my treatment back to Texas. Saw my home Onc today and he believes that he/his clinic will be getting this new phase 3 trial and that I may be able to get my TDM1 TPAS treatments there... I hold out hope cautiously, but never count on it until the deal is sealed. If so, it would save us a bunch of travel money, but I would miss my Denver clinic and doctor sooooo much. They are stellar, but so is my home doctor and clinic. Sigh. It's rough having too many good choices! I wish everyone had such problems.

I hope all who are interested and qualify will start the ball rolling NOW! It is a great drug.

I agree 110% Chrisy, that the finger points to the FDA as the barrier to access

[kykeon22]

I'm worried and concerned about the starting of the Theresa phase III trial, does this means that all stage IV breast cancer her2 must wait the end of this trial and its results in order to get access toT-DM1 or there is a hope for the drug being approved sooner than the daunting 3.5 years time frame for the trial to be closed?

I'm not sure what this means, but how did the things go from fast tracking, to 1-2 years waiting and now this whooping 3.5 years or more delay?

[hutchibk]

It means that if you fit into the inclusion criteria of the trial, you can get into the trial. Otherwise, one must wait until they gain FDA approval.

That was my story 8 years ago when I was first diagnosed and did not fit the criteria for the Herceptin phase 3 trial, and did not get it when I was first diagnosed. I had to wait until I recurred to get Herceptin.

[schoonder]

kykeon22
Roche has indicated that by mid-2012 they expect to file for approval of T-DM1 based on results from the Emilia trial.

[yanyan]

I signed the petition as well. Can't believe it takes this long to bring a life saving drug on the market. I feel fortunate that herceptin is already available when i was diagnosed in 2011. But again, i feel so sad for those whose lives could have been saved by this drug. I am thankful to the researchers who devote themselves in saving human beings. We, are so tiny in this whole universe yet we are so powerful because we acheive things that were considered impossible. From pennicilin to cancer fighting drug, from a boat to airplane. I am amazed when i was lying on the chair looking at the sky the other night.

[hutchibk]

UPDATE as of today, 9/15/2011, from Genentech:

Genentech would like to provide an update on the protocol amendment to the T-DM1 patient access study (called T-PAS or TDM4884g) that may allow some patients enrolled in the study to receive T-DM1 closer to their homes. We submitted the proposed amendment to the FDA on August 8, 2011, and have received no questions or concerns to date that would require re-evaluation or revision. As a result, we are proceeding with this amendment. All of the study sites involved have been informed, and the next step is for each site to approve and implement it within their own institutions.

The T-PAS protocol amendment provides T-PAS patients the option to enroll into another, ongoing Genentech/Roche study (called the “extension study” or TDM4529g), which, in essence, doubles the number of sites and locations where T-PAS patients can receive T-DM1.. This option is only available to patients currently enrolled in T-PAS. Transferring to one of the extension study sites is optional and available to patients who have completed at least six cycles (18 weeks) of treatment, are determined stable by their doctor (i.e., not progressing and tolerating T-DM1 without major side effects) and meet other required criteria for the TDM4529g study. Current T-PAS patients interested in receiving T-DM1 at a site closer to home should contact the T-PAS study coordinator at their current site. The study coordinator can help determine if one of the extension study sites is closer and begin the transfer process to the new site. Since the amendment requires IRB approval at each of the original T-PAS and extension study sites, some sites may be able to accommodate the transfer of T-PAS patients more quickly than others.

People not enrolled in T-PAS but still interested in T-DM1 should talk to their doctor about their eligibility for the other T-DM1 studies currently enrolling patients.

Dear Chrisy,

Agree! Why the delay, even my oncologist does not understand why the FDA has not approved this drug as of yesterday!

Love,

Adriana