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Marwan G. Fakih, MD, explains the implications of the findings from the phase 2 trial of balstilimab plus botensilimab for the treatment of patients with microsatellite stable metastatic colorectal cancer without liver metastases. 


Fakih Discusses Phase 2 Trial of Balstilimab Plus Botensilimab in MSS mCRC

Marwan G. Fakih, MD, provides an overview of the study design, rationale, and findings from a phase 2 trial of balstilimab with botensilimab for the treatment of patients with microsatellite stable metastatic colorectal cancer without liver metastases.

Preliminary Data Show the Potential of Balstilimab Plus Botensilimab in MSS mCRC

ASH 2024 Preview for Community Oncologists: The Show and After Show

The American Society of Hematology (ASH) Annual Meeting and Exposition aims to educate oncology providers across an array of hematologic malignancies, with a focus on novel agents, drugs across the FDA pipeline, and more.

, co-hosts Kristie L. Kahl and Andrew Svonavec offer a glimpse at some of the presentations guaranteed to spark one’s curiosity, spur debate, and enhance the field even further. Some key highlights include:

 Continued advancements in CAR T-cell therapy are expected to revolutionize the treatment of hematologic malignancies.

 Bispecific antibodies, such as Epcoritamab (Epkinly), offer a novel approach to target multiple antigens on cancer cells.

 The assessment of MRD is becoming increasingly important in predicting treatment response and overall survival.

Advances in Acute Lymphoblastic Leukemia (ALL)

Blinatumomab (Blincyto) in pediatric ALL:

 The addition of blinatumomab to chemotherapy has shown promise in improving disease-free survival in high-risk pediatric ALL. This combination therapy may offer a more effective treatment option for young patients.

Acalabrutinib (Calquence) in mantle cell lymphoma (MCL):

 The FDA is set to review the approval of acalabrutinib with bendamustine or rituximab (Rituxan) for previously untreated MCL, based on data from the phase 3 ECHO trial (NCT02972840).

Asciminib (Scemblix) in chronic phase chronic myelogenous leukemia (CML-CP):

 The FDA recently granted accelerated approval to asciminib for newly diagnosed CML-CP. This tyrosine kinase inhibitor has shown favorable safety and tolerability profiles.

 isn't just about data, as the duo also offers food recommendations, tourist attractions and fun activities to partake in, as well as interesting facts about San Diego.

This is just a taste of the wealth of information and inspiration waiting at the 2024 ASH Annual Meeting. Check back for future meeting previews with 

Targeted Oncology co-hosts Kristie L. Kahl and Andrew Svonavec highlight abstracts for community oncologists to look out for at the upcoming ASH Annual Meeting in San Diego, with some additional tidbits to round out the main event.

News

The biologics license application (BLA) of the subcutaneous formulation of nivolumab (Opdivo) coformulated with human hyaluronidase (rHuPH20) has been accepted by the FDA.

Findings from the CheckMate -67T (NCT04810078) support this BLA of subcutaneous nivolumab as the trial met its coprimary pharmacokinetic end points and key secondary end point of overall response rate (ORR) vs intravenous (IV) nivolumab in clear cell renal cell carcinoma.

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date for December 29, 2024.

The FDA has accepted the BLA of the subcutaneous formulation of nivolumab coformulated with rHuPH20, also known as subcutaneous nivolumab, for all previously approved adult solid tumor indications of nivolumab as a monotherapy, monotherapy maintenance following completion of the combination therapy with nivolumab plus ipilimumab (Yervoy), or in combination with chemotherapy or cabozantinib (Cabometyx).

Results from the phase 3 CheckMate -67T trial of subcutaneous nivolumab support this application. In the trial, experts sought to evaluate subcutaneous nivolumab vs its intravenous (IV) formulation.

 Here, the subcutaneous form demonstrated noninferior pharmacokinetics and efficacy, as well as consistent safety compared with the IV formulation of nivolumab among patients with advanced or metastatic clear cell renal cell carcinoma (ccRCC) who have received prior systemic therapy.

A PDUFA goal date has been set for December 29, 2024. With this, subcutaneous nivolumab shows promise to become the first and only subcutaneously administered PD-1 inhibitor.

“We believe subcutaneous nivolumab has the potential to make a significant difference in the lives of patients, which is reinforced by the FDA’s acceptance of our application,” said Gina Fusaro, PhD, vice president, global program lead, Bristol Myers Squibb, in a press release.

“[Nivolumab] is a foundational PD-1 inhibitor approved for many different types of cancer, and our continued investment in research that puts patients first remains a priority. If approved by the FDA, the subcutaneous administration of nivolumab would provide patients and their physicians with a new option that delivers the same well-known benefits as IV [nivolumab] but with the improved convenience of an injection administered in 3 to 5 minutes rather than a 30- to 60-minute infusion,” Fusaro continued.

The phase 3, randomized, open-label CheckMate -67T trial is exploring subcutaneous administration of nivolumab coformulated with rHuPH20 vs IV nivolumab for the treatment of patients with advanced or metastatic ccRCC who were previously treated with systemic therapy.

In the study, 495 patients were randomly assigned to receive either subcutaneous nivolumab or IV nivolumab. Enrollment was open to patients with a histological confirmation of ccRCC, advanced RCC not amenable to curative or radiation therapy, metastatic RCC, those who received no more than 2 prior systemic therapies, those with intolerance or progression after last treatment regimen within 6 months of randomization, and those with a Karnofsky performance score ≥ 70 at screening.

Experts assessed the coprimary end points of time-averaged serum concentration over 28 days and trough serum concentration at steady-state of subcutaneous nivolumab vs IV nivolumab. A key secondary end point of the trial was ORR.

The study met its coprimary pharmacokinetic end points and key secondary end point of ORR with subcutaneous nivolumab in this patient population.

 Subcutaneous nivolumab was noninferior in regard to time-averaged nivolumab serum concentration over 28 days and steady-state trough serum concentration vs treatment with IV nivolumab. Additionally, subcutaneous nivolumab also demonstrated a noninferior ORR compared with IV nivolumab.

1. U.S. Food and Drug Administration accepts Bristol Myers Squibb’s application for subcutaneous nivolumab (nivolumab and hyaluronidase). News release. Bristol Myers Squibb. May 6, 2024. Accessed May 6, 2024. 

2. Phase 3 Check-Mate -67T trial of subcutaneous nivolumab (nivolumab and hyaluranisade) meets co-primary end points in advanced or metastatic clear cell renal cell carcinoma. News release. Bristol Myers Squibb. October 19, 2024. Accessed May 6, 2024. 

3. A study of subcutaneous nivolumab versus intravenous nivolumab in participants with previously treated clear cell renal cell carcinoma that is advanced or has spread (CheckMate-67T). ClinicalTrials.gov. Updated October 12, 2023. Accessed May 6, 2024. 

https://www.clinicaltrials.gov/study/NCT04810078

The FDA is considering a subcutaneous nivolumab formulation for various solid tumors based on promising data from the phase 3 CheckMate -67T trial. A target action date has been set for December 29, 2024.

Clinical

FDA Accepts BLA for Subcutaneous Nivolumab Across Various Solid Tumors

How do you monitor and manage anemia in patients with primary myelofibrosis prior to starting Janus kinase (JAK) inhibitor therapy?​

How do you monitor and manage anemia in patients with myelofibrosis even prior to starting a JAK inhibitor? If you're initially evaluating someone, they come in with hemoglobin of 7 or 8 g/dL or something like that, how does that impact your thinking, and how do you work that up?

The first thing is to make sure that they don't have something easily fixable [such as] iron, B12, or folate deficiency. Theoretically, if you don't have any nutritional cause or any immune causes, you could use erythropoietin analogs, but they are very tricky because they don't help with the spleen, at least in my experience, and they may increase the risk of clots. I try to avoid them if possible. If I have to start a JAK inhibitor, if they have symptoms, either I go away from ruxolitinib [Jakafi] or I start a low dose of ruxolitinib. Sometimes I use anabolic steroids and prednisone. We have the thalidomide/prednisone [regimen] that now is kind of forgotten about.

Erythropoietin-stimulating agents [ESAs] are limited in what they do, and certainly not helping other aspects of the disease. You did mention one of my favorite regimens, thalidomide/prednisone, which is looked over a bit nowadays, but has quite a potential for efficacy in terms of improving hemoglobin. But it’s probably something that's gone by the wayside in many practices.

As Dr Lekakis says, there are some other options that you can use. Danazol is sometimes something you can use perfectly in this patient population. From the JAK inhibitors, there is momelotinib [Ojjaara]. But a priority for me is to refer a patient for a stem cell transplant; when I was a fellow, we did the first allogeneic bone marrow transplant at the University of Illinois, Chicago, and it was a very nice experience.

Dr Lekakis] also mentioned steroids, [and we are] thinking about things like danazol, androgen derivatives. We saw from the MOMENTUM study [NCT04173494] looking at momelotinib that danazol is an active agent.

 It was a large, randomized trial, one of the first randomized trials with danazol. The focus there was on momelotinib, but there's also a lot to learn about what danazol could do as well from that study.

You're mainly talking about anemia here, and [if], the patient is not a candidate for a transplant or a clinical trial [there are] the 

, which basically double the response and reduction of the spleen.

Yes, these emerging therapies to some degree may change how we approach this disease. But I think that with some of these older therapies, especially when you're when you're first talking about someone who's coming in with anemia, you need to think about ESA, danazol, and thalidomide/prednisone.

I think that ESA is probably the most prominently used. How many of you are checking serum erythropoietin levels in these patients to assess their candidacy for ESAs, is that a common practice?

Now in terms of the patient who develops anemia while receiving a JAK inhibitor, ruxolitinib or fedratinib [Inrebic], how do you then approach that patient? This is the someone on therapy who may not have had prominent anemia before, but now that anemia is a little bit more obvious.

I would reduce the dose. I cannot stop [ruxolitinib] easily because of the withdrawal symptoms, but I would reduce the dose.

I probably would be giving a lower dose or initiating at a lower dose if they are anemic, and then I can go up later if I have to.

With the option of reducing dose, and now newer JAK inhibitors that are available, how do you decide between changing therapy vs reducing the dose of ruxolitinib?

I would try to maximize the use of ruxolitinib and then go to the next drug.

Hemoglobin levels do not deter me from initiating ruxolitinib

The most common answer in about half of the responders is that a hemoglobin of 7 to 8 g/dL is a concerning level. We’ve hit on this before that if ruxolitinib drops hemoglobin on average by 1 to 2 g/dL, if you start in the 7s, that means you're going to drop to the 6s at least, and that’s an uncomfortable spot to be. Others have said less than 7 g/dL, some 8 to 9 g/dL, and then at least one for hemoglobin levels not deterring from ruxolitinib initiation. But I think in general, the 7 to 8 g/dL makes sense from a pragmatic point of view, given what we know about ruxolitinib, and I've heard that answer quite frequently.

Kuykendall previously disclosed consulting fees from CTI Biopharma, GSK, and Imago Biosciences; payment or honoraria from BMS, Incyte, and MorphoSys; travel support from AbbVie and MorphoSys; and advisory board participation with Incyte; no other relevant participant disclosures are known.

1. Verstovsek S, Gerds AT, Vannucchi AM, et al. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study. 

. 2023;401(10373):269-280. doi:10.1016/S0140-6736(22)02036-0

2. Pemmaraju N, Mead AJ, Somervaille T, et al. Transform-1: a randomized, double-blind, placebo-controlled, multicenter, international phase 3 study of navitoclax in combination with ruxolitinib versus ruxolitinib plus placebo in patients with untreated myelofibrosis. Blood. 2023;142(suppl 1):620.doi:10.1182/blood-2023-173509

During a live event, Andrew Kuykendall, MD, discussed management of anemia before and during use of JAK inhibitor for myelofibrosis in the second of 2 articles. 

Reevaluating Available Options for Anemia in Myelofibrosis

Marlise R. Luskin, MD, MSCE, discusses the trial of tagraxofusp in BPDCN, which showed high response rates and overall survival.

Tagraxofusp Shows Encouraging Response Rate in Patients With BPDCN

Marlise R. Luskin, MD, MSCE, a member of the Adult Leukemia Program at Dana-Farber Cancer Institute, associate program director of the Dana-Farber/Mass General Brigham Hematology/Oncology Fellowship Program, and associate professor of medicine at Harvard Medical School, discusses the data observed with tagraxofusp (Elzonris) in patients with blastic plasmacytoid dendritic-cell neoplasm (BPDCN).

In the phase 1/2 study with 47 patients enrolled (NCT02113982), 32 patients had no prior treatment, and the rest had received previous treatments. The phase 2 dose from earlier in the trial showed a high response rate according to Luskin, with a 90% overall response rate (ORR) and a 72% complete remission rate. This led to a 2-year overall survival rate of 50%, which Luskin says is promising compared to historical controls.

Additionally, the treatment allowed half of the patients to proceed to stem cell transplantation, aiming for a more durable remission. Responses to the treatment were observed quickly, often after 1 to 2 cycles, and some responses were found to be durable, providing evidence for the drug's effectiveness in both transplant-eligible and ineligible patients.

 The initial publication reported on 47 patients [who] were mostly without previous treatment, 32 of the 47, with the rest having had previous treatment. For untreated patients who received the recommended phase 2 dose, there was a really high response rate, 90% ORR, and 72%, almost three quarters, achieving a complete remission. In those patients, that translated to an overall survival [rate] at 2 years of 50%, which is really encouraging based on historical controls.

 This regimen also allowed half of these patients to proceed to transplantation, which is offered to patients who are able to receive this treatment with the goal of a more durable remission and cure. The other interesting thing is that the responses in this study were seen really quickly, generally after 1 or 2 cycles, allowing patients to receive this treatment and then move, when possible, to transplantation relatively quickly. Long-term outcomes suggest that in a portion of patients, the responses are durable, which provides additional data for this drug in patients, whether or not they are transplant eligible or ineligible.

Recent analyses have shown a stark imbalance in the distribution and access to renal cell carcinoma (RCC) clinical trials, with the studies conducted heavily skewed toward high-income countries. According to Regina Barragan-Carrillo, MD, out of 357 analyzed trials, 76% were conducted exclusively in high-income countries, while low-income countries had 0 trials available over a 5-year period, leaving entire populations without access to potentially life-saving treatments.

These data come from research presented at the 2025 American Society of Clinical Oncology Genitourinary (ASCO GU) Cancers Symposium. Barragan-Carrillo, postdoctoral fellow at the genitourinary clinic at the medical oncology and medical therapeutics at City of Hope Comprehensive Cancer Center in Duarte, CA, shared that pharma-led trials in non-high–income countries are mostly late-phase, meaning early-stage research remains concentrated in wealthier nations. This creates significant delays in bringing new therapies to underserved regions.

Because of this, patients with cancer in low- and middle-income countries are faced with worse outcomes due to limited access to innovative treatments. Additionally, the lack of trials in these regions means there’s no data on how new therapies perform in diverse populations, further delaying drug approvals and worsening survival disparities.

“Survival rates for renal cell carcinoma in high-income countries are improving thanks to clinical research, but low- and middle-income countries are still dying at higher rates because they don’t have access to the same innovations. I think that’s quite an unacceptable gap,” Barragan-Carrillo told Targeted Oncology

In the interview, Barragan-Carrillo further highlighted these global disparities in RCC clinical trials.

Targeted Oncology: Can you provide some background on this research and this abstract?

 The biggest takeaway from this abstract is that access to renal cell carcinoma clinical trials is heavily skewed towards high-income countries, while low- and middle-income countries are largely left behind. Overall, we analyzed a total of 357 renal cell carcinoma clinical trials, and the numbers tell a very clear story. Out of these 357 trials, 76% were conducted exclusively in high-income countries, while 23% included countries classified as upper-middle income, and only 3% included countries from lower-middle income economies. The worst part is that not a single renal cell carcinoma trial was found to be active in low-income countries. That means that entire populations have zero access to clinical research in this disease setting.

We also found that pharma-led trials were much more frequently involved in non–high-income countries compared to academic or government-sponsored trials. But there’s a catch: these pharma-led trials in non–high-income countries are mostly late-phase trials, including phase 3 and phase 4, meaning that early-stage research, mostly phase 1, is still happening almost entirely in wealthier nations. This creates a huge delay in bringing new therapies to patients located in underserved areas. While pharma is technically present in non-high-income countries, research is happening much later in the process, after the drugs have already been tested in high-income populations, which is not exactly equitable.

How does the availability of RCC clinical trials vary across different income levels of countries, and why is that important to talk about?

The numbers paint a pretty stark picture of inequality. As I mentioned, out of the 357 trials we analyzed, 273—which is 76%—were exclusively available in high-income countries. When we looked at how those trials were distributed across different income levels, the pattern was very clear: the wealthier the country, the more trials it had available. Among high-income countries, 60% had at least 1 renal cell carcinoma trial available. But the numbers dropped very fast as you move down the economic scale. Only 24% of upper-middle-income countries had a trial, and just 7.7% of lower-middle-income countries had any renal cell carcinoma trial available at the point of analysis. As I mentioned before, regarding low-income countries, 0 trials were available in the 5-year span we analyzed.

In an interview, Regina Barragan-Carrillo, MD, highlighted the ongoing global disparities in renal cell carcinoma clinical trials. 

Marwan G. Fakih, MD

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