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Abexinostat showed a 69.5% objective response rate in relapsed follicular lymphoma patientsNew Drug Shrinks Stubborn Lymphoma Tumors After Other Treatments Fail

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Key Takeaway
Consider abexinostat for third-line or later-line relapsed follicular lymphoma based on phase 2 data.

This single-arm, multi-center phase 2 study enrolled 90 patients with relapsed or refractory follicular lymphoma (FL) who had previously received a minimum of two systemic treatment lines. The intervention was abexinostat 80 mg oral twice daily on a schedule of seven days on and seven days off, within 28-day cycles. No comparator was reported.

The primary outcome, objective response rate (ORR), was 69.5% (95% CI 58.4-79.2), based on 57 of 82 evaluable patients. Secondary outcomes included a complete response rate of 14.6% (12/82) and a disease control rate (DCR) of 91.5% (75/82). Tumor size reduction was observed in 89% (73/82) of patients. Median duration of response was 13.96 months (95% CI 9.20-not reached). Median progression-free survival (PFS) was 13.80 months (95% CI 9.69-30.26), and median overall survival (OS) was 47.18 months (95% CI 45.70-NR). The median follow-up was 30.55 months (95% CI 23.72-33.64).

Safety was characterized by adverse events including thrombocytopenia (85.6%), neutropenia (58.9%), and leukopenia (52.2%). Serious adverse events were not reported. Discontinuations occurred until unacceptable toxicity or disease progression. The safety profile was described as manageable.

Limitations include the single-arm design and lack of a control group. Funding or conflicts of interest were not reported. This evidence supports abexinostat as a new treatment option for the third-line or later-line treatment of relapsed or refractory FL, pending validation in randomized settings.

Sarah was told her lymphoma was back—again. She’d tried two treatments already. Both worked—for a while. Then the cancer returned. Like many people with follicular lymphoma, she felt stuck. Options were running out.

Follicular lymphoma is a type of blood cancer. It grows slowly but often comes back after treatment. Over time, it can become resistant to standard therapies. About 20,000 people are diagnosed each year in the U.S. Most live for years, but each relapse makes the next treatment harder.

Many patients end up needing a third or even fourth therapy. But few options exist once earlier ones stop working. That’s why doctors are urgently searching for new paths.

A new drug may change that story for some.

What changed this time? Researchers tested a drug called abexinostat. It works differently than most lymphoma treatments. Instead of attacking cancer cells directly, it targets how genes are turned on or off inside them.

Think of cancer cells like a factory with broken switches. Abexinostat helps reset the switches so the cell can “remember” how to stop growing out of control. It’s like rebooting a computer that’s frozen.

This drug belongs to a class called histone deacetylase inhibitors. That’s a mouthful. But the idea is simple: it changes how DNA is packaged in the cell, which can slow or stop cancer growth.

The study included 90 patients with follicular lymphoma. All had tried at least two other treatments that failed. They took abexinostat by mouth—twice daily for seven days, then seven days off, repeating every 28 days.

Tumor responses were tracked by independent experts who didn’t know who was in the study. This helps keep results fair and accurate.

Results were encouraging. Tumors shrank in 69.5% of patients. That’s nearly 7 out of 10. Even better, 91.5% of patients had their disease controlled—meaning tumors didn’t grow for a significant time.

Some patients had complete remissions. Their scans showed no signs of cancer. That happened in 14.6% of cases—about 1 in 7.

Tumors got smaller in 89% of patients. The average time tumors stayed under control was nearly 14 months. Some people stayed in remission much longer.

Survival data is still early. But the average overall survival was 47 months—more than three years—and still not reached for many.

But there’s a catch.

Side effects were common. Most patients had low blood counts—especially platelets and white blood cells. This can raise the risk of bleeding or infection.

About 86% had low platelets. Nearly 59% had low neutrophils. These issues often required dose adjustments or treatment breaks. But most side effects were manageable.

Doctors say the results are promising—not because the drug cures everyone, but because it works when few other options do.

This doesn't mean this treatment is available yet.

Experts say this drug could become a new option for third-line treatment. That means after two other therapies have failed. But it’s not approved yet.

Right now, abexinostat is only available in clinical trials. Patients who’ve tried other treatments and are looking for new options should talk to their doctor about whether a trial might be right.

The study had limits. It didn’t compare abexinostat to other drugs. Everyone got the same treatment, so we can’t say it’s better than alternatives. Also, the group was relatively small.

Most participants were white and treated in specialized centers. That means results might look different in other populations.

Still, for a disease that keeps coming back, even a temporary win matters.

What happens next? Larger trials are needed. Researchers will likely test abexinostat against current standard treatments in a randomized way. That’s the next step before approval.

Drug development takes time. Even if results hold up, it could be several years before abexinostat becomes widely available—if it does.

But for patients running out of options, this study offers something real: hope of more time.

Study Details

Study typePhase2
Sample sizen = 90
EvidenceLevel 3
Follow-up30.6 mo
PublishedApr 2026
View Original Abstract ↓
This phase 2, single-arm, multi-center study (NCT03934567) evaluates the efficacy and safety of abexinostat, a histone deacetylase inhibitor, in patients with relapsed or refractory (r/r) follicular lymphoma (FL). Eligible participants had previously received a minimum of two systemic treatment lines, such as cytotoxic agents and/or anti-CD20 monoclonal antibodies. Participants received abexinostat 80 mg oral twice daily on a schedule of seven days on and seven days off, within 28-day cycles, continuing until unacceptable toxicity or disease progression occurred. The primary endpoint was objective response rate (ORR), evaluated by an independent review committee (IRC). Secondary endpoints comprised duration of response (DoR), disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and safety. Between May 2, 2019, and November 19, 2023, 90 patients were enrolled. As of October 08, 2024, with a median follow-up of 30.55 months (95% confidence interval [CI] 23.72-33.64), 17.8% (16/90) of patients were still on study treatment. The ORR was 69.5% (57/82, 95% CI 58.4-79.2), with a complete response rate of 14.6% (12/82). The DCR was 91.5% (75/82, 95% CI 83.2-96.5). Tumor size reduced in 89% (73/82) of patients. The median DoR was 13.96 months (95% CI 9.20-not reached [NR]) and the median PFS was 13.80 months (95% CI 9.69-30.26). The median OS was 47.18 months (95% CI 45.70-NR). The most common treatment-emergent adverse events were thrombocytopenia (77/90, 85.6%), neutropenia (53/90, 58.9%), and leukopenia (47/90, 52.2%). The results of this study demonstrated that abexinostat had promising efficacy and manageable safety profile, supporting abexinostat as a new treatment option for the third-line or later-line treatment of r/r FL.
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