We just received this and wanted to share the information with the community…
Hi all,
We received some discouraging news today about the DEFEND 1 Study.
Tolerx, Inc. and GlaxoSmithKline (GSK) today announced that the Phase 3 DEFEND-1 study of otelixizumab, an investigational humanized anti-CD3 monoclonal antibody, did not meet the primary efficacy endpoint of change in C-peptide at month 12 in patients with new-onset autoimmune type 1 diabetes.
The good news is that there were no new or unexpected treatment-related safety concerns have emerged during the DEFEND-1 study.
So what does this mean for DEFEND-2?
GSK will continue to explore additional dosing regimens to inform decisions about the future clinical development programme for otelixizumab. New recruitment and dosing in the DEFEND-2 study, the ongoing confirmatory Phase 3 study with a design similar to DEFEND-1, has been suspended pending review of the DEFEND-1 results.
Read more at:
Tolerx and GlaxoSmithKline Announce Phase 3 Defend-1 Study of Otelixizumab in Type 1 Diabetes Did Not Meet Its Primary Endpoint
This is my blog entry on this news:
Otelixizumab (Tolerx / GlaxoSmithKline) Fails Phase-III Trial
The official quote is “did not meet the primary efficacy endpoint”. Basically, the the people who got the drug did not do better than those who did not, in the the most important measurement of success. The primary endpoint for this experiment was C-peptide generation, which is a marker for natural insulin production. Basically, they were hoping that giving this drug would help honeymoon type-1 diabetics generate more of their own insulin, but it did not.
In terms of actions: they have stopped enrollment in their second phase-III trial (DEFEND-2), which shows that they think that there is little to no hope of moving forward with this drug at this time.
So that is about as dead as a phase-III trial can get.
Press release: http://classic.cnbc.com/id/42028160
Tolerx blog: http://tolerx.com/index.php?page=greenchair&entry=committed-to-the-promise-of-our-normalization-immunotherapy-platform
DEFEND-1: http://www.clinicaltrials.gov/ct2/show/NCT00678886
DEFEND-2: http://www.clinicaltrials.gov/ct2/show/NCT01123083
A Little Discussion
This is not completely unexpected, because there was another similar drug (Teplizumab), which was also an anti-CD3 monoclonal antibody which was also in phase-III clinical trials and just a few months ago, it failed as well. And for the same reason: did not cure/improve people. Neither trial had any safety problems.
There is still one anti-CD3 monoclonal antibody out there. It is NI-0401 by NovImmune, and is in phase-II clinical trials. Unfortunately, I’ve never been able to find results for their phase-I study, so I don’t hold out much hope. If you know anything about NovImmune’s NI-0401 results, or where they were published, then please tell me.
Previous blogging on Teplizumab:
http://cureresearch4type1diabetes.blogspot.com/search/label/Teplizumab
Previous blogging on NI-0401:
http://cureresearch4type1diabetes.blogspot.com/search/label/NI-0401
So where are we now?
Years ago, there was only one treatment in phase-III trials: DiaPep277. Last year, there were four. Since then, two have failed and none have entered, so we are down to two: GAD65 and DiaPep277. GAD65 is expected to announce their first phase-III results in the next 3 months, but DiaPep277 results are much farther away.
Joshua Levy
Blog: http://cureresearch4type1diabetes.blogspot.com