On behalf of patients and families impacted by cholangiocarcinoma (CCA), the Cholangiocarcinoma Foundation (CCF) today hailed progress in the development journey for TIBSOVO® (ivosidenib tablets) a targeted therapy for IDH1-mutated cholangiocarcinoma (CCA) patients, as Agios announced that it has submitted a Supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA). This filing is based upon the positive primary analysis of progression free survival and the recent encouraging overall survival (OS) analysis from Agios' global Phase 3 ClarIDHy trial of TIBSOVO® in previously treated CCA patients with an isocitrate dehydrogenase 1 (IDH1) mutation.

CCF has been involved in the global effort to advance this approach for treating the IDH1-positive subset of CCA patients – which accounts for approximately 20% of Intrahepatic CCA cases -- since the beginning of the Agios development program, providing patient insights and input on the design of the trial. CCF's initial engagement was predicated on the significant unmet need of this patient population, as there are no approved systemic therapies. Limited chemotherapy options are available in the advanced setting. Gemcitabine-based chemotherapy is often recommended for newly diagnosed advanced or metastatic disease.

"This is an important milestone for this community and specifically for patients with an IDH1 mutation. We want to acknowledge all who participated in this trial, and thank them for playing such a critical role. The ClarIDHy Phase 3 study data shows that TIBSOVO® has the potential to lengthen time to disease progression for these patients and provides hope that they will have access to a viable treatment option" said Stacie Lindsey, Founder and CEO, CCF.

"Cholangiocarcinoma is a rare, aggressive cancer with limited effective therapies, and patients are in desperate need of new treatment options – particularly those who experience disease progression after chemotherapy," said Chris Bowden, M.D., chief medical officer at Agios. "We are proud of the work we have done on behalf of these patients, and we look forward to working closely with the FDA during the review of the first oral therapy targeting an IDH1 mutation for patients with previously treated IDH1-mutated cholangiocarcinoma. Thank you to the Cholangiocarcinoma Foundation for providing important patient insight and information on unmet needs as we developed this program."

"Facing a stage 4 cholangiocarcinoma diagnosis, my future was unclear. Tibsovo gave me a future, it gave my life back! For the last two and half years, I have lived a normal life, without tumor progression, drug or cancer side effects. I feel like I hit the cancer lotto with Aigos' TIBSOVO®," said Kathy Barboza, cholangiocarcinoma patient and AG 120 trial participant.

The sNDA is currently under review with the FDA and a decision is expected by the end of 2021.

This is an important milestone for this community and specifically for patients with an IDH1 mutation. We want to acknowledge all who participated in this trial, and thank them for playing such a critical role. The ClarIDHy Phase 3 study data shows that TIBSOVO® has the potential to lengthen time to disease progression for these patients and provides hope that they will have access to a viable treatment option.

Stacie Lindsey
Founder and CEO
Cholangiocarcinoma Foundation