On April 16, 2024 Astrazeneca reported updated exploratory results from the TOPAZ-1 Phase III trial showed that Imfinzi (durvalumab) in combination with standard-of-care chemotherapy demonstrated a clinically meaningful long-term overall survival (OS) benefit at three years for patients with advanced biliary tract cancer (BTC) (Press release, AstraZeneca, APR 16, 2024, View Source [SID1234642077]).

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These results from TOPAZ-1, which are the longest survival follow-up ever reported for a global, randomised Phase III trial in this setting, will be presented on 18 April at the 2024 Cholangiocarcinoma Foundation Conference in Salt Lake City, Utah.

At more than three years (median follow-up of 41.3 months), results showed Imfinzi plus chemotherapy reduced the risk of death by 26% versus chemotherapy alone (based on a hazard ratio [HR] of 0.74; 95% confidence interval [CI], 0.63-0.87). The median OS was 12.9 months for Imfinzi plus chemotherapy versus 11.3 months for chemotherapy alone. More than twice as many patients on the Imfinzi-based regimen were alive at three years versus chemotherapy alone (14.6% versus 6.9%).

The TOPAZ-1 trial met the primary endpoint of OS in October 2021 at a planned interim analysis, showing that the combination reduced the risk of death by 20% versus chemotherapy alone (based on a HR of 0.80; 95% CI, 0.66-0.97; 2-sided p=0.021 at a statistical significance threshold of 0.03).

Do-Youn Oh, MD, PhD, Professor, Division of Medical Oncology, Department of Internal Medicine at Seoul National University Hospital and Seoul National University College of Medicine, and principal investigator in the trial, said: "The latest data from TOPAZ-1 show that twice as many patients with advanced biliary tract cancer were still alive at three years with durvalumab and chemotherapy, an especially meaningful advance in a setting where historically the prognosis has been poor. These results reinforce the long-term benefit of this immunotherapy-based combination as a standard of care for patients with this devastating disease."

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: "TOPAZ-1 raised the bar for the treatment of advanced biliary tract cancer, showing a remarkable survival benefit for Imfinzi added to chemotherapy with a well-tolerated regimen. These data represent the longest survival follow-up reported for immunotherapy in this setting, and the three-year landmark survival improvement underscores our commitment to improving long-term outcomes in gastrointestinal cancers."

Stacie Lindsey, CEO, Cholangiocarcinoma Foundation said: "AstraZeneca’s longer survival data in advanced biliary tract cancer represents a meaningful milestone in that we are seeing three-year survival data for the first time for these patients. The data spurs hope that research will continue to improve outcomes for patients living with these challenging and rare cancers."

Summary of updated survival results: TOPAZ-1i

OSi,ii

Imfinzi + chemotherapy

(n=341)

Chemotherapy

(n=344)

Median OS (95% CI in months)

12.9

(11.6-14.1)

11.3

(10.1-12.5)

HR (95% CI) iii

0.74 (0.63-0.87)

OS rate at 36 months (95% CI) (%)iv

14.6

(11.0-18.6)

6.9

(4.5-10.0)

i. 26 months of additional follow-up (data cut-off: 23 October 2023) after the primary analysis, with 89% overall OS event maturity

ii. At data cut-off for this analysis, median (95% CI) follow-up time in all patients calculated using reverse Kaplan-Meier technique was 42.9 (39.8-44.3) months for Imfinzi plus chemotherapy and 41.8 (36.7-46.2) months for chemotherapy

iii. HR and 95% CI calculated using Cox proportional hazards model

iv. OS rates calculated using Kaplan-Meier technique

Imfinzi plus chemotherapy continued to be well-tolerated, with no new safety signals observed with longer follow-up. Results showed 15.4% of patients experienced treatment-related serious adverse events with Imfinzi plus chemotherapy versus 17.3% with chemotherapy alone.

Notes

Biliary tract cancer
Biliary tract cancer (BTC) is a group of rare and aggressive gastrointestinal (GI) cancers that form in the cells of the bile ducts (cholangiocarcinoma), gallbladder or ampulla of Vater (where the bile duct and pancreatic duct connect to the small intestine).1,2 Approximately 50,000 people in the US, Europe and Japan and about 210,000 people worldwide are diagnosed with BTC each year.3-5 These patients historically have a poor prognosis, with approximately 5% to 15% of patients with BTC surviving five years. For patients with metastatic disease, the five-year survival rate drops to less than 5%.7

Cholangiocarcinoma is more common in China and Thailand and is on the rise in Western countries.1,6 Gallbladder cancer is more common in certain regions of South America, India and Japan.8

Early-stage BTC affecting the bile ducts and gallbladder often presents without clear symptoms and most new cases of BTC are therefore diagnosed at an advanced stage, when treatment options are limited and the prognosis is poor.6,8,9

TOPAZ-1
TOPAZ-1 is a randomised, double-blind, placebo controlled, multicentre, global Phase III trial of Imfinzi in combination with chemotherapy (gemcitabine plus cisplatin) versus placebo in combination with chemotherapy as a 1st-line treatment in 685 adult patients with unresectable, locally advanced or metastatic BTC including intrahepatic and extrahepatic cholangiocarcinoma, and gallbladder cancer. Patients with ampullary carcinoma were excluded.

The primary endpoint is overall survival and key secondary endpoints included progression-free survival, objective response rate and safety. The trial was conducted in 105 centres across 17 countries including in the US, Europe, South America and several countries in Asia including South Korea, Thailand, Japan and China

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

In addition to its indications in unresectable, Stage III NSCLC and ES-SCLC, Imfinzi is currently approved in a number of countries in combination with a short course of Imjudo and chemotherapy for the treatment of metastatic NSCLC.

Imfinzi is also approved in a number of countries in combination with chemotherapy in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the EU and in previously treated patients with advanced bladder cancer in a small number of countries.

Since the first approval in May 2017, more than 220,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, bladder cancer, breast cancer, several gastrointestinal cancers and other solid tumours.

On April 16, 2024 Imugene, Ltd. ("Imugene")(ASX: IMU) and Kincell Bio, LLC ("Kincell"), reported a strategic manufacturing and process development partnership, which includes the sale of Imugene’s North Carolina Current Good Manufacturing Practice (CGMP) manufacturing facility and the transfer of process and analytical development activities to Kincell (Press release, Imugene, APR 16, 2024, https://mcusercontent.com/e38c43331936a9627acb6427c/files/3e21bcbd-4451-a69a-db91-69476faaf963/Imugene_and_Kincell_Bio_Announce_Strategic_Partnership.pdf [SID1234642076]).

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Under the terms of an asset purchase agreement between Imugene and Kincell, Kincell will acquire Imugene’s CGMP-compliant cell therapy manufacturing facility in North Carolina for a total consideration of up to $6M USD in upfront and milestone-driven payments. Both parties have entered into a manufacturing supply agreement whereby Kincell will manufacture Imugene’s Azer-cel to support ongoing clinical trials. Imugene will also transfer process and analytical development of Azer-cel to Kincell in order to support process and method optimization for commercial readiness.

Leslie Chong, Managing Director and Chief Executive Officer of Imugene, commented, "We are delighted to have found a strong partner for the development and manufacturing of our CAR T Azer-cel program. We are confident that this strategic partnership with Kincell will enable Imugene to reach key upcoming data inflection points and extend the company’s cash runway to 2026. Moreover, this partnership allows us to focus on our key capabilities, namely the development of novel cancer treatments. With the transaction, we look forward to continuing to work with many of our former manufacturing colleagues in a new relationship as our contract development and manufacturing organization partner."

Bruce Thompson, CEO of Kincell, said, "The acquisition of this facility and experienced team, which is actively manufacturing CGMP-compliant products that can support late-stage and/or pivotal clinical trials, accelerates our ability to expand our service offerings for cell therapy developers. The facility’s location in Research Triangle Park (RTP) will facilitate access to talent in a fast-growing and attractive biotech hub. Additionally, we’re excited that the manufacturing supply agreement enables us to partner with Imugene, an innovative immunotherapy company, to optimize and progress an allogeneic CAR T product candidate into later-stage development for patients with significant unmet medical needs."

The 32,800-square-foot, state-of-the-art, CGMP-compliant facility is designed with the flexibility to expand in capacity and scope to support the manufacture of cell-based therapies. Kincell intends to evolve the site capabilities while leveraging enterprise-wide expertise to manufacture a broad portfolio of autologous and allogeneic products.

On April 15, 2024 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported the Phase III STARGLO study met its primary endpoint of overall survival (Press release, Genentech, APR 15, 2024, View Source [SID1234642080]). The study demonstrated that people with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL), who have received at least one prior line of therapy and are not candidates for autologous stem cell transplant, lived longer when treated with Columvi (glofitamab-gxbm) in combination with gemcitabine and oxaliplatin (GemOx) versus Rituxan (rituximab) in combination with GemOx. Safety of the combination appeared consistent with the known safety profiles of the individual medicines. The data will be submitted to health authorities and shared at an upcoming medical meeting.

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"People with this aggressive lymphoma facing relapse or progression after initial treatment have limited options – particularly those who are ineligible for stem cell transplant," said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. "Building on Columvi’s established benefits, these data demonstrate the potential of this combination regimen to improve survival outcomes in earlier lines of treatment."

Columvi was the first fixed-duration bispecific antibody to receive accelerated approval by the U.S. Food and Drug Administration and conditional marketing authorization from the European Commission to treat people with R/R DLBCL after two or more lines of systemic therapy. These approvals were based on positive results of Columvi as a monotherapy from the pivotal Phase I/II NP30179 study in patients with R/R DLBCL who had previously received two or more prior treatments.

Columvi is a CD20xCD3 T-cell engaging bispecific antibody designed to be off-the-shelf and ready for infusion, so patients can start treatment soon after diagnosis. This is particularly important for patients with highly aggressive disease who are at risk of rapid disease progression. Columvi is given as a fixed-duration treatment, offering people with R/R DLBCL who have failed two or more lines of therapy a treatment end date and the possibility of a treatment-free period, unlike continuous treatments.

About the STARGLO Study

The STARGLO study [GO41944; NCT04408638] is a Phase III, multicenter, open-label, randomized study evaluating the efficacy and safety of Columvi (glofitamab-gxbm) in combination with gemcitabine plus oxaliplatin (GemOx) versus Rituxan (rituximab) in combination with GemOx in patients with diffuse large B-cell lymphoma who have received at least one prior line of therapy and who are not candidates for autologous stem cell transplant. Outcome measures include overall survival (primary endpoint), progression-free survival, complete response rate, objective response rate, duration of objective response (secondary endpoints), and safety and tolerability.

About Diffuse Large B-Cell Lymphoma

Diffuse large B-cell lymphoma (DLBCL) is an aggressive (fast-growing) blood cancer and is the most common form of non-Hodgkin’s lymphoma (NHL) in the U.S. While many people with DLBCL are responsive to treatment, the majority of those who relapse or are refractory to subsequent treatments have poor outcomes. DLBCL not otherwise specified is the most common category of large B-cell lymphoma (LBCL) and accounts for about 80% or more of cases. It applies to cases that do not fall into any specific disease subgroups of LBCL.

About Columvi (glofitamab-gxbm)

Columvi is a CD20xCD3 T-cell engaging bispecific antibody designed to target CD3 on the surface of T cells and CD20 on the surface of B cells. Columvi was designed with a novel 2:1 structural format. This T-cell engaging bispecific antibody is engineered to have one region that binds to CD3, a protein on T cells, a type of immune cell, and two regions that bind to CD20, a protein on B cells, which can be healthy or malignant. This dual-targeting brings the T cell in close proximity to the B cell, activating the release of cancer cell-killing proteins from the T cell. A clinical development program for Columvi is ongoing, investigating the molecule as a monotherapy and in combination with other medicines for the treatment of people with B-cell non-Hodgkin’s lymphomas, including diffuse large B-cell lymphoma and other blood cancers.

Columvi U.S. Indication

Columvi (glofitamab-gxbm) is a prescription medicine to treat adults with certain types of diffuse large B-cell lymphoma (DLBCL) or large B-cell lymphoma (LBCL) that has come back (relapsed) or that did not respond to previous treatment (refractory), and who have received 2 or more prior treatments for their cancer.

It is not known if Columvi is safe and effective in children.

The conditional approval of Columvi is based on response rate and durability of response. There are ongoing studies to establish how well the drug works.

What is the most important information I should know about Columvi?

Columvi can cause Cytokine Release Syndrome (CRS), a serious side effect that is common during treatment with Columvi, and can also be serious and lead to death.

Call your healthcare provider or get emergency medical help right away if you develop any signs or symptoms of CRS, including:

fever of 100.4°F (38°C) or higher
chills or shaking
fast or irregular heartbeat
dizziness or light-headedness
trouble breathing
shortness of breath
Due to the risk of CRS, you will receive Columvi on a "step-up dosing schedule".

A single dose of a medicine called obinutuzumab will be given to you on the first day of your first treatment cycle (Day 1 of Cycle 1).
You will start the Columvi step-up dosing schedule a week after the obinutuzumab dose. The step-up dosing schedule is when you receive smaller "step-up" doses of Columvi on Day 8 and Day 15 of Cycle 1. This is to help reduce your risk of CRS. You should be hospitalized during your infusion and for 24 hours after receiving the first step-up dose on Day 8. You should be hospitalized during your infusion and for 24 hours after receiving the second step-up dose on Day 15 if you experienced CRS during the first step-up dose.
You will receive your first full dose of Columvi a week after the second step-up dose (this will be Day 1 of Cycle 2).
If your dose of Columvi is delayed for any reason, you may need to repeat the "step-up dosing schedule".
If you had more than mild CRS with your previous dose of Columvi, you should be hospitalized during and for 24 hours after receiving your next dose of Columvi.
Before each dose of Columvi, you will receive medicines to help reduce your risk of CRS and infusion-related reactions.
Your healthcare provider will monitor you for CRS during treatment with Columvi and may treat you in a hospital if you develop signs and symptoms of CRS. Your healthcare provider may temporarily stop or completely stop your treatment with Columvi if you have severe side effects.

Carry the Columvi Patient Wallet Card with you at all times and show it to all of your healthcare providers. The Columvi Patient Wallet Card lists the signs and symptoms of CRS you should get emergency medical help for right away.

What are the possible side effects of Columvi?

Columvi may cause serious side effects, including:

Cytokine Release Syndrome.
Neurologic problems. Columvi can cause serious neurologic problems that may lead to death. Your healthcare provider will monitor you for neurologic problems during treatment with Columvi. Your healthcare provider may also refer you to a healthcare provider who specializes in neurologic problems. Tell your healthcare provider right away if you develop any signs or symptoms of neurologic problems, including:
headache
confusion and disorientation
difficulty paying attention or understanding things
trouble speaking
sleepiness
memory problems
numbness, tingling, or weakness of the hands or feet
dizziness
shaking (tremors)
Serious Infections. Columvi can cause serious infections that may lead to death. Your healthcare provider will monitor you for signs and symptoms of infection and treat you as needed. Tell your healthcare provider right away if you develop any signs of an infection, including: fever, chills, weakness, cough, shortness of breath, or sore throat.
Growth in your tumor or worsening of tumor related problems (tumor flare). Tell your healthcare provider if you get any of these signs or symptoms of tumor flare:
tender or swollen lymph nodes
pain or swelling at the site of the tumor
chest pain
cough
trouble breathing
The most common side effects of Columvi include: CRS, muscle and bone pain, rash, and tiredness.

The most common severe abnormal lab test results with Columvi include: decreased white blood cells, decreased phosphate (an electrolyte), increased uric acid levels, and decreased fibrinogen (a protein that helps with blood clotting).

Your healthcare provider may temporarily stop or completely stop treatment with Columvi if you develop certain side effects.

Before receiving Columvi, tell your healthcare provider about all of your medical conditions, including if you:

have an infection
have kidney problems
are pregnant or plan to become pregnant. Columvi may harm your unborn baby
Females who are able to become pregnant:
Your healthcare provider should do a pregnancy test before you start treatment with Columvi.
You should use effective birth control (contraception) during treatment and for 1 month after your last dose of Columvi. Talk to your healthcare provider about what birth control method is right for you during this time.
Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Columvi.
are breastfeeding or plan to breastfeed. Columvi may pass into your breast milk. Do not breastfeed during treatment and for 1 month after your last dose of Columvi.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What should I avoid while receiving Columvi?

Do not drive, operate heavy machinery, or do other dangerous activities if you develop dizziness, confusion, shaking (tremors), sleepiness, or any other symptoms that impair consciousness until your signs and symptoms go away. These may be signs and symptoms of neurologic problems.

These are not all the possible side effects of Columvi. Talk to your health care provider for more information about the benefits and risks of Columvi.

You may report side effects to the FDA at (800) FDA-1088 or View Source You may also report side effects to Genentech at (888) 835-2555.

Please see Important Safety Information, including Serious Side Effects, as well as the Columvi full Prescribing Information and Medication Guide or visit View Source

On April 15, 2024 NiKang Therapeutics Inc. ("NiKang"), a clinical stage biotech company focused on developing innovative small molecule oncology medicines to help patients with unmet medical needs, reported that the first patient has been dosed in a phase 1/1b, open-label, first-in-human dose escalation and expansion study of single agent NKT3447, a small molecule that inhibits cyclin-dependent kinase 2 (CDK2) (Press release, NiKang Therapeutics, APR 15, 2024, View Source [SID1234642079]). NKT3447 is designed to treat patients with cancers driven by cyclin E amplification or overexpression, which is present in many different tumor types.

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The Phase 1/1b trial (NCT06264921) is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical activity of NKT3447 in adult patients with advanced or metastatic solid tumors driven by cyclin E or CDK2.

"The initiation of dosing in this study marks a major milestone for NiKang, as NKT3447 is the first of our pipeline programs targeting the cell cycle to begin clinical evaluation," said Zhenhai Gao, Ph.D., co-founder, president, and CEO of NiKang. "We have strong conviction that CDK2 is a key oncology target and have taken a holistic approach to build an industry-leading portfolio that also includes a CDK2-selective degrader and a CDK2/4 dual degrader. While there has been clinical success with drugs targeting the cell cycle, it has been challenging to identify inhibitors of CDK2 that spare CDK1 and do not cause a compensatory increase of cyclin E which is a driver of tumor cell proliferation. NKT3447 binds inactive monomeric CDK2, disrupting the CDK2/cyclin E complex without impacting CDK1. Furthermore, its interaction with CDK2 results in suppression of activating phosphorylation of CDK2 on Thr160 and a substantial downregulation of cyclin E, potentially preventing a mechanism of resistance."

"We are excited to initiate clinical trials of NKT3447, which has unique features that have led to sustained pharmacodynamic effects and significant anti-tumor activity in various cyclin E amplified tumor models," said Joanne Jenkins Lager, M.D., Chief Medical Officer of NiKang. "CDK2 and cyclin E are deregulated in many human cancers, and we believe NKT3447 has the potential to change the standard of care for people with cyclin E amplified or overexpressing cancers including ovarian cancer, endometrial cancer and gastric cancer."

On April 15, 2024 Norgine B.V. reported its first marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom (Press release, Norgine, APR 15, 2024, View Source [SID1234642078]). This milestone supports Norgine’s efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology.

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Norgine and USWM, LLC (dba US WorldMeds), a Kentucky-based specialty pharmaceutical company, have an exclusive licensing agreement by which Norgine will register and commercialise eflornithine, also referred to as DFMO, in Europe, Australia and New Zealand.

On 13 December 2023, the US Food and Drug Administration (FDA) approved eflornithine as the first oral maintenance therapy for high-risk neuroblastoma (HRNB), indicated to reduce the risk of relapse in adult and paediatric patients who have received certain prior therapies.1 The approval decision was based on findings from a trial comparing outcomes from Study 3b (NCT02395666; investigational arm)2,4 and Study ANBL0032 (NCT00026312; clinical-trial-derived external control arm)3,4, where use of eflornithine resulted in improved event-free survival and overall survival when compared to outcomes for patients with high-risk neuroblastoma treated with the standard of care (SoC) without the drug.1

Dr David Gillen, Chief Medical Officer at Norgine, added, "These submissions via Project Orbis represent an important first step in the regulatory process for eflornithine and re-emphasise Norgine’s passion and commitment in attempting to secure additional treatment options for patients living with HRNB, a condition with a high level of unmet medical need."

Project Orbis

Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health).

HRNB background

Children diagnosed with HRNB undergo an intense SoC regimen that still leaves them vulnerable to relapse and death, a risk that is particularly acute during the first two years.5 Approximately 30% of patients who attain remission following upfront therapy will relapse, resulting in a poor prognosis and low likelihood of long-term survival (e.g. estimates as low as 15% of patients will live for five years after relapsing).6 Avoiding relapse is key to long-term survival, yet outside of the United States there are no approved therapies for sustaining remission following SoC treatment. The data with eflornithine demonstrate using it as maintenance therapy extends remission and reduces risk of relapse in patients with HRNB.