The Targeted Pulse: Cancer Research Slowed by Funding Freezes and the FDA Takes Forward Steps

Nivolumab/Ipilimumab Combination Moves Forward in dMMR/MSI-H CRC

The FDA has accepted the supplemental biologics license application (sBLA) for nivolumab (Opdivo) plus ipilimumab (Yervoy), marking a potential first-line treatment option. The indication includes adult and pediatric (≥12 years) patients with microsatellite instability–high or mismatch repair–deficient unresectable or metastatic colorectal cancer. The sBLA submission is supported by data from the phase 3 CheckMate 8HW study. The Prescription Drug User Fee Act (PDUFA) target date for this indication is June 23, 2025.

“CheckMate 8HW has been [ongoing] for several years now as we have gotten updates from the study’s maturation, [evaluating] the combination of nivolumab and ipilimumab,” Mark A. Lewis, MD, told Targeted Oncology^TM. “It drives home the point of [identifying] biomarkers as early as possible, because if you can save a patient from chemotherapy, that is a wonderful thing to do,” Lewis is director of gastrointestinal oncology at Intermountain Health in Salt Lake City, Utah. For further information regarding this sBLA, access the full article here.

Funding Freezes Put Cancer Research on Ice, Halting Progress

Gwen Nichols, MD, discussed the ramifications of a rapidly implemented policy, that froze and then cut funding. She addressed concerns over the potential impact these reductions in federal funding may have on both community and academic centers that rely on grants from the National Institutes of Health (NIH). Nichols is particularly concerned for young investigators who depend on these grants to establish their careers. She explained that these cuts could have a trickle-down effect, slowing the next generation of innovation in cancer research.

“The freeze affected community health centers funded by the NIH, forcing some to temporarily close their doors and stop seeing patients until funding was restored. These were unintended consequences of a rapidly implemented policy,” Nichols explained. “This isn’t about the Leukemia & Lymphoma Society (LLS) taking a stance on the policy itself, but rather about how quickly these changes occurred, without sufficient time to adjust and find alternative solutions. Nichols is executive vice president and chief medical officer of the LLS in Rye Brook, New York. To access this information, we ask that you share some details about yourself so we can better tailor our offerings. Begin hereto get started and access this resource.

Maintenance Fedratinib After Transplant Shows Potential in MPN

Hany Elmariah, MD, discussed recent data from a phase 1 trial evaluating fedratinib (Inrebic) as maintenance therapy for myeloproliferative neoplasms following allogeneic hematopoietic cell transplant. Fedratinib is a JAK2 inhibitor that is approved for pre-transplant myelofibrosis and is now being evaluated as a post-transplant maintenance therapy to potentially reduce relapse risk and graft-vs-host disease. In the trial, a daily dose of 400 mg of fedratinib, the established maximum tolerated dose, was determined safe.

“Importantly, although the study was conducted at a large cancer center, fedratinib is a drug that can be prescribed and managed in the community setting,” Elmariah told Targeted Oncology^TM. “Many patients have their transplant at a major center like Moffitt but then return to their local oncologists for follow-up care. So, this drug could be used by community physicians to manage these patients post-transplant,” Elmariah is associate member at the Moffitt Cancer Center in the department of bone marrow transplant and cellular immunotherapy in Tampa, Florida. For more information about the data, access the full article here.

Petosemtamab/Pembrolizumab Combo Gains Breakthrough Status in HNSCC

The FDA has granted priority review to the combination of petosemtamab and pembrolizumab (Keytruda) as a first-line treatment for recurrent or metastatic head and neck squamous cell carcinoma with a PD-L1 combined positive score of at least 1. Findings from phase 2 of a phase 1/2 trial (NCT03526835) are the basis for this development. In addition, the phase 3 LiGeR-HN1 trial (NCT06525220), is another study evaluating the combination in this patient population.

“One of the interesting things I’ve learned about these molecules is that responses can be seen quite early, which is unusual for checkpoint inhibitors—we typically have to wait longer,” Cesar Augusto Perez, MD, explained in an interview with Targeted Oncology^TM. “For unknown reasons, this antibody shows activity early in the treatment course, which is crucial for symptomatic patients. This is especially important for those with life-threatening disease in the immediate term, as we aim not to lose patients.” Perez is a hematologist and medical oncologist at Florida Cancer Specialists & Research Institute’s Lake Nona Cancer Center & Drug Development Unit. For information regarding this development, access the full article here.

“Priority Review Illustrates Urgent Need” for Zongertinib in HER2-Mutant NSCLC

The FDA has granted priority review to zongertinib (BI 1810631) for patients with unresectable or metastatic non–small cell lung cancer harboring HER2 (ERBB2) mutations who have received prior systemic therapy. This decision is supported by data from the phase 1b Beamion LUNG-1 trial (NCT04886804). The PDUFA date is set for the last quarter of 2025.

“We believe zongertinib has the potential to transform the care of previously treated patients with HER2 (ERBB2)-mutant advanced NSCLC and are hopeful about the continued research in other tumor types and lines of therapy,” said Shashank Deshpande, in a press release. “Priority Review illustrates the urgent need in this patient population and the possibility for zongertinib to be a groundbreaking innovation for patients with limited treatment options.” Deshpande is a member of the managing directors board and head of human pharma at Boehringer Ingelheim. For more information on trial data, access the full article here.

Thank you for joining us for this week’s Targeted Pulse. Look out for more recaps to come.

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