GIST

A phase Ib/II clinical trial of the experimental anti-mitochondrial drug devimistat in combination with cisplatin and gemcitabine has been initiated in patients with biliary tract cancer, according to a press release from Rafael Pharmaceuticals, Inc. The company will collaborate with Michigan Medicine to proceed with the randomized multicenter clinical trial.

The FDA has approved avapritinib for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor who harbor platelet-derived growth factor receptor alpha exon 18 mutation, including PDGFRA D842V mutations. This approval makes avapritinib the first precision medicine therapy for a genomically defined population of patients with GIST, according to a press release from the Blueprint Medicines Corporation.

Aiwu Ruth He, MD, discusses the current role of immune checkpoint inhibitors in upper gastrointestinal and gastric cancers and which patients are most likely to benefit from the anti–PD-1 agents.

In December 2019, the FDA approved a number of new treatments in lung cancer, pancreatic cancer, urothelial carcinoma, breast cancer, and prostate cancer. A tissue complete assay was also approved by the FDA for use in a clinical trial evaluating pembrolizumab.

Margaret von Mehren, MD, discusses the rationale for evaluating ripretinib in patients with heavily pretreated advanced GIST in the phase III INVICTUS trial, which was presented at the ESMO Congress 2019.

The first half-yearly follow-up&nbsp;results from a phase I study of ilixadencel monotherapy in patients with advanced gastrointestinal stromal tumors will be presented by Alex Karlsson-Parra, MD, PhD on February 6, 2020, during poster session A, at the Clinical Immuno-Oncology Symposium, according to a press release from Immunicum AB, where Karlsson-Parra serves as the chief executive officer.<br /> &nbsp;

The target number of patients with advanced gastrointestinal stromal tumors have been enrolled in the phase III VOYAGER study in China, which is evaluating the safety and efficacy investigational drug avapritinib in the third- or fourth-line settings, according to a press release from CStone Pharmaceuticals.<br /> &nbsp;

A New Drug Application for ripretinib was submitted to the FDA for the treatment of patients with advanced gastrointestinal stromal tumor who have received a prior treatment of imatinib, sunitinib, and regorafenib, according to a press release from Deciphera Pharmaceuticals, Inc.

Benjamin Weinberg, MD, assistant professor of medicine, and attending physician, Georgetown University Hospital, Medstar Health, discusses the overall prognosis of patients with gastrointestinal stromal tumors.

Timil Patel, MD, discusses how to sequence therapies in a patient with rectal cancer with a single lung nodule.

Given the frequency of <em>FGFR2</em>&nbsp;alterations in intrahepatic cholangiocarcinoma, investigators have identified it as a potential prognostic indicator of survival and a rational target of systemic cancer therapies.

Winston Tan, MD, discusses the emergence of novel agents for the treatment of patients with gastrointestinal stromal tumors, namely agents like avapritinib and ripretinib, which have shown promise in ongoing clinical trials.<br /> &nbsp;

TT-00420, an investigational spectrum-selective multiple kinase inhibitor received an orphan drug designation from the FDA for the treatment of cholangiocarcinoma, TransThera Biosciences Co. Ltd announced in a press release.

Daniel Catenacci, MD, discusses how he makes decisions with immunotherapeutic agents for the treatment of patients with gastroesophageal junction cancer. He says he bases his sequencing decisions on a number of factors.

The phase III VOYAGER study may soon introduce a new treatment option for patients with locally advanced metastatic or unresectable gastrointestinal stromal tumors, a patient population with an unmet need for novel agents that are durable and can target specific drivers. The trial is looking at the efficacy of novel drug avapritinib versus standard-of-care regorafenib.<br /> &nbsp;

The FDA plans to split up the submitted New Drug Application for avapritinib into 2 separate NDAs in the treatment of 2 different populations of patients with gastrointestinal stromal tumors, according to a press release from Blueprint Medicines.

Pamela L. Kunz, MD, discusses the most recent FDA approval of Lutathera (lutetium [Lu 177] dotatate) for the treatment of patients with gastroenteropancreatic NETs (GEP-NETs), including foregut, midgut, and hindgut tumors.

Following the 2019 ESMO Congress, experts across various fields highlighted some next steps and how these treatment options will improve the treatment landscape for patients with ovarian, lung, breast, GI, or GU cancers. Overall, the abstracts presented at this year&rsquo;s meeting will change the treatment paradigm in a number of patient populations.

In the phase III INVICTUS trial, ripretinib, a novel KIT and PDGFRA inhibitor, demonstrated a dramatic improvement in progression-free survival compared with placebo in heavily pretreated patients with heavily advanced gastrointestinal stromal tumors, according to data presented at the 2019 ESMO Congress.

An open-label phase II trial of regorafenib given continuously at a reduced dose of 100 mg daily to patients with refractory gastrointestinal stromal tumors found that nearly two-thirds of patients achieved disease control lasting for at least 12 weeks.<br /> &nbsp;

The risk of progression or death was reduced by 63% with&nbsp;ivosidenib as treatment of pretreated patients with IDH1-mutant advanced cholangiocarcinoma&nbsp;compared with placebo, according to data from the phase III&nbsp;ClarIDHy study that was presented at the 2019 ESMO Congress.

An objective response rate of 35.5% was seen with&nbsp;treatment with pemigatinib, a selective oral inhibitor of&nbsp;FGFR1, 2, and 3, in patients with&nbsp;previously treated, locally advanced or metastatic cholangiocarcinoma with an&nbsp;<em>FGFR2&nbsp;</em>rearrangement or fusion,&nbsp;according to findings from the phase II FIGHT-202 clinical trial presented at ESMO 2019.

Gulam A. Manji, MD, PhD, discusses the need to identify mutations that may be actionable in patients with colorectal cancer. The identification of such mutations can further impact patient survival and improve efficacy, says Manji, in both CRC and other gastrointestinal cancers.

Amol K. Narang, MD, discusses the unanswered questions that still need to be addressed to further expand the role of stereotactic body radiation therapy in gastrointestinal cancers.

Here&#39;s a look back on the FDA happenings for the month of August 2019, including FDA approvals, priority reviews, and breakthrough designations.