The Targeted Pulse: A Review of Last Month’s FDA Prominent Actions and Key Data Updates Advancing Treatment Across Disease Types

Exploring Promising Therapies and Defining Disease States in MDS

David Sallman, MD, discusses recent breakthroughs in the field of myelodysplastic syndromes (MDS). Specifically, investigators have gained a better understanding of MDS in order to design more effective clinical trials. Sallman highlights developments in the VERONA trial (NCT04401748), which is evaluating azacitidine with venetoclax [Venclexta] vs azacitidine therapy alone and the SELECT-MDS-1 (NCT04797780) trial, which is evaluating azacitidine/tamibarotene [Amnolake] vs azacitidine alone. These data are highly anticipated and if positive, may create a paradigm shift in MDS treatment.

“There has been a significant understanding of disease states, classifications, and prognostics, but clinical drug development has been much slower. I really hope we will have a drug to push us forward so we can start thinking about that again,” explained Sallman during an interview with Targeted Oncology^TM. Sallman is myeloid section head and associate member of the Malignant Hematology Department, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.

A One Stop Shop For Exploring October’s Focal FDA Updates Across Cancers

This article provides a thorough rundown of the prominent FDA actions that occurred in October 2024. Some of the major updates include the approval of acalabrutinib (Calquence) for first-line treatment of mantle cell lymphoma, the FDA’s priority review of fam-trastuzumab deruxtecan-nxki (T-DXd; Enhertu) for HER2-low/ultra-low breast cancer, and the launch of Cologuard Plus for non-invasive colorectal cancer screening. If you may have missed any crucial approvals or updates last month, this article provides a brief description of each FDA action and a link to the initial coverage.

“Although endocrine therapies are widely used in the initial treatment of hormone receptor-positive metastatic breast cancer, most patients see limited benefit with additional lines of treatment and subsequent chemotherapy is associated with poor response rates and outcomes. The results from DESTINY-Breast06 [NCT04494425] show that Enhertu has the potential to evolve the current hormone receptor-positive treatment landscape and become the first targeted treatment for patients with HER2-low or HER2-ultralow expression following endocrine therapy,” Susan Galbraith, executive vice president, Oncology R&D, AstraZeneca, stated in a press release.

Significant OS Benefit Shown With Pembrolizumab Plus Chemotherapy in TNBC

The combination of neoadjuvant pembrolizumab (Keytruda) combined with chemotherapy, followed by adjuvant pembrolizumab demonstrated significant overall-survival improvement in patients with early-stage triple-negative breast cancer. The combination was evaluated in the randomized, double-blind phase 3 KEYNOTE-522 trial (NCT03036488). In this article, Peter Schmid, MD, PhD, of Barts Cancer Institute at Queen Mary University in London, United Kingdom, discussed the results.

“[In the trial], we used 12 weeks of weekly paclitaxel and carboplatin, followed by 12 weeks of [doxorubicin or epirubicin and cyclophosphamide]. The trial has 2 coprimary end points. The short-term end point [is] pathological complete response [pCR], which means the complete disappearance of all invasive cancer at the time of surgery, and then the long-term end point is event-free survival, which means the reduction in recurrences, which is obviously the long-term benefit of the treatment,” Schmid told Targeted OncologyTM in the interview.

Lenvatinib Combination Boosts PFS in Intermediate-Stage HCC

According to data from the phase 3 double-blinded, active-controlled, multicenter LEAP-012 trial (NCT04246177), lenvatinib (Lenvima) with pembrolizumab (Keytruda) and transarterial chemoembolization (TACE) significantly improved progression-free survival in patients with intermediate-stage hepatocellular carcinoma (HCC). In the trial, the combination was compared with placebo and TACE. Study investigator, Josep M. Llovet, MD, PhD, provided deeper insight into the updated trial data in an interview with Targeted Oncology^TM. Llovet is director of the Liver Cancer Program and professor of medicine in the Division of Liver Diseases at the Icahn School of Medicine at Mount Sinai Hospital in New York, New York.

“This is the most robust data reported over the years in intermediate HCC, combining systemic therapies with local/regional therapies. Lenvatinib/pembrolizumab certainly provides an alternative treatment that we propose as a frontline option for patients with intermediate HCC, which represents 30% of patients and includes those with liver-only disease without dissemination,” Llovet told Targeted Oncology^TM.

Upcoming NY Lung Symposium Set To Explore Perioperative Data in Lung Cancer

This year’s 19th Annual New York Lung Cancers Symposium® will focus on treatment strategies in early-stage lung cancers, exploring immune checkpoint inhibitors in the perioperative space. The meeting is planned for November 16, 2024, and will be led by cochairs Balazs Halmos, MD, MS and Mark G. Kris, MD. Prior to the conference, Kris discussed data from the CheckMate 816 (NCT02998528), IMpower010 (NCT02486718), PEARLS (NCT02504372), and ADAURA (NCT02511106) trials, highlighting the effects of perioperative use with immune checkpoint inhibitors.

“We work hard to make this a very interactive meeting. It is geared toward practitioners, but we call upon researchers to present the most up-to-date data to the oncologists on the front lines. “It is not a dry data review. For people who attend the conference, they will walk away with expert opinion, and not just a list of possible treatment solutions,” Kris told Targeted Oncology^TM in the interview.

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

In case you missed it, here is last week’s Targeted Pulse.