Agios to Present Clinical Data from Ongoing AG-120 Phase 1 Trial

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    After the initial set of scans yesterday, 8 weeks into the AG-120 trial, the doctors were largely encouraged. The sobering news is that we were told there is a tumor in the lungs that has grown by 8% but we were told that all other tumors are stable and some in the liver have even shrunk. Dr. Harding at MSK has strongly encouraged her to stay on the trial based on these results, which they are considering to be “stable disease” at this point. There is some good news here but the pessimistic view is that I’m not sure we have any other option. I hate that there is growth of a tumor and she has been coughing but the rest of the news seems very good (other than swollen belly which may be accedes plus swollen liver plus other side effects and her continued recovery from the compression fracture, which seems to be largely healed; and she may also have a slight rib fracture or two that the doctors did not find significant enough to even discuss until questioned about it) and she feels good and all other blood tests are showing good signs. We are still awaiting PET Scan results and she is going for another biopsy on Friday, which is part of the trial protocol. I suppose the only logical alternative at this point may be Keytruda but none of the doctors are advocating that at this point. I wanted to report the news in case this might help anyone and, of course, if anyone has any suggestions or advice, please do not hesitate to share.

    Thanks and best to all!




    Thank you for the update and sending best wishes to you and Mom-




    Sending prayers for you and your mom that scan results are more than positive! Hugs to you both!


    Steven…thanks for sharing and wishing for absolute success with the treatment. Fingers are crossed for perfect scan results. Looking forward to your next posting.


    Hello All, my mother Kathleen, about whom I have posted a number of times previously, has been participating in this trial for Agios 120 (AG-120) at MSK in NYC since the beginning of December. She had been on FOLFOX chemo since August 2015 after we discovered in July that the CC had returned (stage 4). She was reaching the end of her tolerance of FOLFOX, which was beating her up but also seeming stabilizing the cancer, although she suffered a spinal compression fracture from the metastasis near the spine that the doctors think had spread there before she started FOLFOX. Side effects have been minimal although she has significant swelling in her bellly along with a protruding mass which the doctors indicated to be a side effect of the AG-120. She will be scanned two weeks from tomorrow (Weds). I will keep everyone posted with any noteworthy results.

    Very best to all,


    From Agios Sunday investor presentation today –

    Agios Extends Reach of Cancer Metabolism Drug into Solid Tumors.

    “Another 20 patients had intrahepatic cholangiocarcinoma, a type of liver cancer. Here, there was a single partial response durable beyond six months and 11 reports of stable disease.”



    Hi Jason,

    Thanks for that and great to see you on here again. 25% up in one day. Like you, I would be guessing that some got wind of the trial results to move up that much in one day. The IBB was up 3.25% today so it was a strong day anyway for the sector but 25%, someone must have heard something I would argue.

    ArQule also releasing some info and trial details on the 7th November –

    “About FGFR and ARQ 087

    ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (“FGFR”) family with demonstrated efficacy in FGFR2 amplified tumors. The FGFR pathway is disrupted in several ways in human cancer, thus providing numerous therapeutic targets for an inhibitor of this pathway. ARQ 087 has demonstrated inhibition of tumor growth and downstream signaling in vivo in tumors whose growth is driven by these targets.

    Signals of single agent activity with this compound were observed in Phase 1a testing. Phase 1b expansion cohorts with ARQ 087 include patients with cholangiocarcinoma and adrenocortical tumors, as well as those with FGFR translocations, amplification and mutations. Clinical development of ARQ 087 has advanced into Phase 2 for intrahepatic cholangiocarcinoma (“iCCA”) following the observation of two confirmed partial responses in this patient population in the Phase 1 portion of the program.

    Fingers crossed for good news from both camps!



    Agios to Present Clinical Data from Ongoing AG-120 Phase 1 Trial in Advanced Solid Tumors at AACR-NCI-EORTC Investor Lunch and Webcast on Sunday, November 8, 2015.

    This might bear watching. The drug AG-120 targets the IDH-1 mutation that is fairly common in Intrahepatic CC. I am pretty sure they included some CC patients in the trial, so maybe some data to come out in a week or two on whether it helps ICC patients.

    Here is the link to the press release:

    Agios stock was up 25% today, so perhaps the data were good news?

    I hope so,


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