Sara Karlovitch is an associate editor with Targeted Oncology. She received a duel degree in Journalism and Government and Politics from the University of Maryland. 

Sara Karlovitch

Iron metabolism normalization is key mechanism of action of the synthetic mimetic of hepcidin, rusfertide (PTG-300), which has been shown to help achieve hematocrit control in patients with polycythemia vera (PV) without the need for periodic therapeutic phlebotomy.

During the 63rd American Society of Hematology Annual Meeting & Exposition (ASH), 2 presentations highlighted the promise and the need for further exploration of rusfertide in PV treatment.

According to earlier research, rusfertide regulates iron absorption, distribution, and storage, thereby controlling the body’s production of red blood cells. Rusfertide is meant to be more potent and offer greater stability and solubility compared with the natural hormone.1 When explored in a phase 2 study as induction therapy administered twice weekly, rusfertide was found to be effective in achieving the target hematocrit of < 45% quickly and without phlebotomy.

A recent phase 2 trial presented during ASH found that rusfertide was safe and tolerable in the PV patient population and did improve hematocrit levels.

“PV is a chronic myeloproliferative disease, one of several, it is associated often given its chronicity with a long period of survival, including sometimes up to 20 years post diagnosis. There are groups of patients both better categorized into low risk versus high risk. The low-risk patients are younger and do not have a history of thromboembolic events. The higher risk population is older and or has a history of a thromboembolic event. Patients are often treated with therapeutic phlebotomy, that's the mainstay of treatment, especially in the low-risk population,” said lead study author Yelena Ginzburg, MD, an associate professor of Medicine, Hematology, and Medical Oncology at Mount Sinai Hospital in an interview with Targeted Oncology™.

PV is often associated with fatigue, which can be severe, due to the increase in iron utilization by the disease. Iron deficiency worsens after repeated therapeutic phlebotomy. Thus, the management and treatment of symptomatic iron deficiency remains an unaddressed clinical need in PV patients. 

Rusfertide is being investigated in over 160 patients. It was placed on clinical hold after both benign and malignant subcutaneous skin tumors were observed in a mouse model. The hold was removed in October 2021 after a review.

The first study evaluating rusfertide in this patient population is a phase 2 study with an estimated enrollment of 20 participants and an estimated completion date of April 2023. The primary end point of the study is efficacy, measured as the proportion of patients with hematocrit < 45%. The secondary end point is safety.

The study is composed of a single arm, and all patients received the study drug. Dosing and schedule were adjusted every 2 to 4 weeks and treatment continued for up to 52 weeks.

At the time of data cutoff, 16 patients were enrolled. The mean age of the patients (12 male and 4 female) was 56.1, with the median time from diagnosis being 3.74 years. With the agent, a rapid decrease in hematocrit to below 45% without the use of phlebotomy was seen and continued to be well controlled after rusfertide was reduced.

Fifty percent of patients experienced adverse events. Injection site reactions were seen in 7 patients and were mild to medium in severity. Common injection site reactions included erythema (n = 7), induration (n = 5) and pruritis (n = 2). Hypertension, pyrexia, and thrombocytes were also observed. Two serious adverse events, which included worsening migraine and pleuritic chest pain, were considered unrelated to rusfertide.

“The adverse events were adjudicated to be related to the agent were all grade 1 and 2, there was one patient with asymptomatic thrombocytosis, that did not require any intervention. And the majority of the adverse events were injection site reactions, none of which led to the patients experiencing that side effect to come off of the study. And we're all relatively transient,” said Ginzburg.

Further analysis is needed in order to validate these phase 2 findings.

"The main point that we are hoping to see with the trial as it continues is both prolonged continuous, either phlebotomy free or phlebotomy reducing effect of using rusfertide in the chronic setting that can also ultimately allow us to evaluate a potential reduction in the thrombotic events in this patient population,” said Ginzburg

A phase 3 trial is currently underway to potentially confirm the previous phase 2 results. The global, multicenter, randomized trial aims to enroll approximately 250 participants to assess the primary end points of a confirmed HCT of 45% or less or a HCT of 48% or less.

“We borrow the experience from the phase 2 study. The phase 2 study was open to any patients, whether they are low risk and would usually receive only phlebotomy or is it for high-risk patients... as long as there are too many phlebotomies, these patients were eligible for the phase two open label study. And what we have seen is that almost everybody benefits right away by elimination of the phlebotomy and people feel better. Based on this information. The phase three study is similar in the design,” said Srdan Verstovsek, MD, PhD, the United Energy Resources, Inc. Professor of Medicine and hematologist-oncologist at MD Anderson Cancer Center in an interview with Targeted Oncology™.

The phase 3 study will include 3 parts. Part 1a will be a randomized, double-blind, placebo-controlled, add-on group period lasting 32 weeks. Patients will be stratified by ongoing PV treatment and randomized 1:1 to receive either rusfertide or placebo added-on to their ongoing PV treatment. Part 1b will be composed of subjects who completed part 1a successfully. They will receive rusfertide for 20 weeks. Part 2 will be a long-term extension phase, where all patients who complete part 1b will receive rusfertide for an additional 32 weeks.

“The key point would be to eliminate need for phlebotomy. That is, the value that we understand is the number 1 goal in PV patients. But along the way, because other issues may arise in life of the PV patients, we want to know and make sure that the therapy has other benefits. And you may understand where we are, the quality of life is the paramount, you want to have a therapy that not only normalize the blood numbers, but with that improve the quality of life,” said Verstovsek.

1.Ginzburg Y, Kirubamoorthy K, Salleh S, et al. Rusfertide (PTG-300) induction therapy rapidly achieves hematocrit control in polycythemia vera patients without the need for therapeutic phlebotomy. 

 2021; 138(1): 390. doi: 10.1182/blood-2021-149205

2. 4.PTG-300 in patients with polycythemia vera and elevated hematocrit. ClinicalTrials.gov. Accessed January 5, 2022.

3.Verstovsek S, Kuykendall A, Hoffman R, et al. A Phase 3 study of the hepcidin mimetic rusfertide (PTG-300) in patients with polycythemia vera. 

 2021; 138(1): 1504. doi: 10.1182/blood-2021-149219

Articles

With rusfertide, clinical trial investigators are poised to fill a clinical gap by addressing iron deficiency in patients with polycythemia vera.

Rusfertide Lowers Hematocrit in Patients With Polcythemia Vera

The combination of the anti-PD-1 agent, balstilimab and the anti-CTLA4 antibody, zalifrelimab showed promising clinical activity and an acceptable safety profile for the treatment of recurrent and/or metastatic cervical cancer, according to a study published in the 

Patients with cervical cancer who progress after first-line chemotherapy doublet plus bevacizumab (Avastin), current treatment remains suboptimal. Currently, pembrolizumab (Keytruda) is the only immunotherapeutic agent approved in this setting.

A phase 1/2 trial (NCT03495882) was designed to test the validity and safety of the balstilimab/zalifrelimab combination as secondline treatment for the advanced cervical cancer patient population. The study has an actual enrollment of 154 participants and an estimated study completion date of May 2023. The primary end point of the study is the objective response rate (ORR) of the investigational combination. Secondary end points include safety and tolerability, maximum drug concentration, minimum observed concentration at steady-state, area under the drug concentration-time curve, time to maximum concentration, terminal elimination of half-life, systemic clearance, volume of distribution, duration of response, disease control, duration of stable disease, time to response, progression-free survival, and overall survival.

Phase 1 was a dose-escalation study, which utilized a 3 + 3 dose escalation. Phase 2 was a dose expansion in advanced cervical cancer.

The median age of patients enrolled was 50 (range, 24-76) and 95.5% were White. Tumor histologies included squamous cell carcinoma (70.3%), adenocarcinoma (27.1%), adenosquamous (2.6%). Fifty-seven percent of patients had an ECOG performance status of 0, and 42.6% had an ECOG performance status of 1. Additionally, 58.8% were PD-L1 positive. Eighty-nine percent of patients received prior radiotherapy. The patients had received a median of 1 (range, 0-2) prior line of therapy. Prior antineoplastic agents included platinum (99.4%), taxane (78.75), and bevacizumab (32.9%).

The ORR was 25.6%. The complete response rate was 8%, the partial response rate was 17.6%, and the stable disease rate was 27.2%. Progressive disease was seen in 40% of patients. The DCR was 52%. The median DoR was not reached, with the median time to response being 2.7 months (range, 1.3-15.8). Twenty-five percent of responses lasted for 6 months or more, 20% of responses lasted for 9 months or more, and 12% of responses lasted for 12 months or more.

For patients who were PD-L1 positive, the ORR was 32.8%. For patients who were PD-L1 negative, the ORR was 9.1%, and 28% for those with an unknown PD-L1 status. For patients with squamous cell carcinoma, the ORR was 32.6%. For patients with adenocarcinoma, the ORR was 8.8%. No responses were seen in patients with adenosquamous histology.

Overall, the 12-month PFS probably was 21.%. The predicated 6-month OS was 69.2%, and the predicted 12-month OS was 53.3%.

In terms of safety, any grade treatment-emergent adverse events (TRAEs) occurred in 71% of patients. Common TRAEs included hypothyroidism (16.8%), diarrhea (14.2%), and fatigue (11.6). TRAEs grade 3 and higher occurred in 20% of patients. Common grade 3 or higher TRAEs included ALT increase (2.6%), diarrhea (1.9%), and anemia (1.3%). TRAEs lead to dose interruption in 12.3% of patients and lead to dose discontinuation in 7.7% of patients.

“The balstilimab plus zalifrelimab doublet elicited high ORRs, durable responses, and an acceptable safety profile as second-line treatment for patients with recurrent and/or metastatic cervical cancer. The high proportion of complete responders and activity irrespective of tumor PD-L1 status or histology were particularly promising outcomes that confirm the feasibility of dual-targeted immunotherapy for this disease,” wrote study authors.

O’Malley D, Neffa M, Monk B, et al. Dual PD-1 and CTLA-4 checkpoint blockade using balstilimab and zalifrelimab combination as second-line treatment for advanced cervical cancer: An open-label phase II study. 

 2021. Published online. doi: 10.1200/JCO.21.02067

In patients with recurrent and/or metastatic cervical cancer, the second-line combination of balstilimab and zalifrelimab may offer meaningful clinical benefit, but further research is needed. 

Balstilimab With Zalifrelimab Elicits High, Durable Responses in Recurrent/Metastatic Cervical Cancer 

Compared with sorafenib (Nexavar), nivolumab (Opdivo) does not significantly improve overall survival (OS), despite showing both clinical activity and favorable safety in patients with advanced hepatocellular carcinoma (HCC), according to results of the CheckMate 459 trial published in 

Previous studies established that single agent nivolumab produces durable responses and manageable safety in patients with advanced HCC. However, it’s efficacy compared to sorafenib was unknown.

The phase 3 CheckMate 459 trial (NCT02576509) was designed to compare the efficacy of the 2 agents head-to-head. The trial had an actual enrollment of 743 participants and an estimated completion date of June 2022. The primary end point of the study is OS. Secondary end points include objective response rate, progression-free survival (PFS), and efficacy based on PD-L1 expression.

In total, 371 were assigned to receive nivolumab and 372 were assigned to receive sorafenib. In total, 329 patients in the nivolumab arm discontinued treatment. Reasons for discontinuation included progressive disease (245), adverse events uncreated to drug (37), study drug toxicity (32), patient request (8), other reasons (2), withdrew consent (2), maximum clinical benefit (1), no longer meeting study criteria (1), and death (1). In total, 371 were analyzed for efficacy and 367 for safety.

In the sorafenib arm, 355 patients discontinued treatment. Reasons for discontinuation included disease progression (240), study drug toxicity (41), adverse events unrelated to the study drug (40), patient request (17), withdrawal of consent (7), other reasons (7), poor compliance (1), lost to follow-up (1), and death (1). In total, 372 patients were evaluated for efficacy, and 363 for safety.

The median age of patients in the nivolumab group is 65 (range, 57-71), 85% were male, and 54% were white. Seventy-three percent of patients had an ECOG performance status of 0. Barcelona Clinical Liver Cancer Stages included A (4%), B (14%), and C (82%). Sixty percent of patients experienced extrahepatic spread and 33% experienced vascular invasion. Ten percent experienced tumor invasion of the liver and 98% of patients had a Child-Pugh score A. Fifty-four percent of patients underwent surgery and 52% of patients underwent non-systemic treatment.

The median age of patients in the sorafenib group was also 65 (range, 58-72), 53% were male, and 53% were white. Seventy percent had an ECOG performance status of 0. Barcelona Clinical Liver Cancer stages included A (5%), B (17%), and C (78%). Fifty-six percent of patients were experiencing extrahepatic spread and 32% were experiencing vascular invasion. Forty-seven percent of patients had more than 3 liver modules and 9% had tumor invasion in the liver. Ninety-six percent of patients had Child-Pugh score A and 56% of patients underwent locoregional therapy.

Fifteen percent of patients in the nivolumab arm experienced an objective response, compared with 7% of patients in the sorafenib group. In the nivolumab group, 4% of patients experienced a complete response, 12% experienced a partial response, 35% achieved stable disease, 37% experienced progressive disease, and 55% achieved disease control. The median duration of disease control was 7.5 months (range, 6.5-10.7). The median time to response was 3.3 months (range, 1.9-3.8). The median duration of response was 23.3 months (range, 17-31.5).

In the sorafenib group, 1% of patients experienced a complete response, 6% experienced a partial response, 48% achieved stable disease, and 28% experienced progressive disease. Fifty-eight percent of patients achieved disease control. The median duration of disease control was 5.7 months (range, 5.6-7.4), and the median time to response was 3.7 months (range, 2.1-5.3). The median duration response was 23.4 months (range, 8.5-NE).

In the nivolumab group, the 12-month OS was 60%, the 18-month OS was 47%, and the 34-month OS was 37%. In the sorafenib group, the 12-month OS was 55%, the 18-month OS was 44%, and the 24-month OS was 33%.

In terms of PFS, the 12-month PFS rate in the nivolumab group was 22%, the 18-month rate was 17%, and the 24-month rate was 14%. In the sorafenib group, the 12-month PFS was 14%, the 18-month rate was 9%, and the 24-month rate was 6%.

In both groups, adverse events were overwhelmingly grade 1 through 3 in nature and very rarely resulted in discontinuation. Common grade 1/2 adverse events for both the nivolumab and the sorafenib groups, respectively, included fatigue (14% vs. 22%), rash (11% vs. 12%), and diarrhea (8% vs. 42%).

“The proportion of responders in the nivolumab group was also higher, although not significantly higher, in patients with tumor cell PD-L1 expression of 1% and greater compared with patients with PD-L1 expression less than 1%, consistent with findings from a biomarker analysis in the nivolumab CheckMate 040 study. Tumor cell PD-L1 expression was not predictive for overall survival or progression-free survival in the current study,” wrote study authors.

Yau T, Park J, Finn R, et al. Nivolumab versus sorafenib in advanced hepatocellular carcinoma (CheckMate 459): a randomised, multicentre, open-label, phase 3 trial. 

 2022;23:77-90. doi: 10.1016/ S1470-2045(21)00604-5

Although nivolumab did not significantly outperform sorafenib in the CheckMate 459 of patients with advanced hepatocellular carcinoma, positive resuls were shown for the secondary end points.

Nivolumab Achieves Durable Responses and Extends OS Versus Sorafenib in HCC

The FDA has granted both a fast-track designation and regenerative medicine advanced therapy designation to the autologous bi-specific CAR-T therapy that targets both CD19 and CD20, C-CAR039, for the treatment of relapsed or refractory diffuse large B cell lymphoma (DLBCL), according to a press release by Cellular Biomedicine Group Inc.

"This is great news for CBMG that the FDA has granted C-CAR039 both RMAT and fast track designations based on its potential to increase objective and complete response rates in r/r DLBCL. The clinical data based on our clinical trials in China continue to support the hypothesis that C-CAR039 is the best-in-class CAR-T asset for patients in this indication,” said Tony (Bizuo) Liu, chairman and chief executive officer, in a press release.

The agent was previously granted clearance for clinical development by the FDA for the treatment of relapsed or refractory non-Hodgkin lymphoma.

The clearance granted phase 1b clinical development of the agent. C-CAR039 has also been granted an orphan drug designation for the treatment of follicular lymphoma. A study of the agent conducted in China found that it had a promising efficacy and safety profile.

“We are excited about the potential of C-CAR039 as a best-in-class treatment in r/r B-NHL. C-CAR039 clearly showed potential superior efficacy and favorable safety results for patients with r/r B-cell NHL. Our C-CAR039 trial is a testament of the teams’ translational medicine capability. We look forward to bringing more potentially promising development to clinical trials,” said Yihong Yao, PhD, chief scientific officer of CBMG, in a press release.

As of April 2021, 34 patients had received C-CAR039, with 28 patients being evaluable for safety and 27 for efficacy. The median age of patients was 55.5 years, and 75% had cancer of Ann Arbor stage III/IV. The median number prior lines of therapy was 3. Nearly 18% of patients had received bridging therapy.

The best overall response rate was reported at 92.6%, with a complete response of 85.2%. The median time to response was 1 month. At a median follow up of 7 months, 74.1% remained in complete remission. The estimated 6-month progression-free survival was at 83.2% (95% CI, 69.1-100.0).

In terms of safety, 96% experienced cytokine release syndrome, with 92% of cases being grade 1 or 2. Only 1 patient experienced grade 3 cytokine release syndrome. All immune-effector cell-associated neurotoxicity syndrome were grade 1 or 2. No grade 3 or higher neurological events were reported.

Longer follow-up studies of C-CAR039 are currently planned, according to Liu.1 

“We are working towards initiating 1b/2 trials for C-CAR039 in the US soon. And we will work closely with the FDA to seek the best path forward to deliver the drug to patients in the US and EU," he stated, in a press release.

1 CBMG receives FDA regenerative medicine advanced therapy and fast track designations for bi-specific anti-CD19/CD20 CAR-T cell therapy for relapsed/refractory B-cell non-Hodgkin lymphoma. News release. CBGM. January 12, 2022. Accessed January 12, 2022.

2 CBMG receives FDA clearance of IND application for bi-specific anti-CD19/CD20 CAR-T cell therapy for relapsed/refractory B-cell non-hodgkin lymphoma. News release. CBGM. December 13, 2021. Accessed January 12, 2022. https://bit.ly/3fgaCaW.

The FDA granted both a regenerative medicine advanced therapy and fast track designation to C-CAR039 for relapsed or refractory diffuse large B-cell lymphoma. 

FDA Grants Fast Track Designation to C-CAR039 for DLBCL

The triplet combination of acalabrutinib (Calquence) plus venetoclax (Venclexta), and rituximab (Rituxan) may provide an alternative option for patients with treatment naïve mantle cell lymphoma (MCL) who are unable to receive chemotherapy, according to data initial safety and efficacy findings from a phase 1b.

In the ongoing, multicenter, open-label study (NCT02717624), the initial analysis looked at 21 patients with MCL who had a median age of 66. Based on promising efficacy and safety observed with Bruton’s tyrosine kinase inhibitor therapy combined with an anti-CD20 monoclonal antibody, investigators of the study believe that the combination has the potential to improve upon fontline convention chemoimmunotherapy

, Michael Wang, MD, a professor in the department of lymphoma and myeloma at MD Anderson Cancer Center, discussed the safety, efficacy, and tolerability of acalabrutinib plus venetoclax and rituximab in MCL.

TARGETED ONCOLOGY™: Can you give an overview of the safety and efficacy of acalabrutinib plus venetoclax and rituximab in patients with treatment naïve MCL?

 Our study showed the triple combination of acalabrutinib plus venetoclax, and rituximab is tolerated very well, in patients with treatment naive MCL. And of the 4 patient who died on the study, all died due to COVID unfortunately. Regarding the efficacy, we achieved a very unprecedented efficacy, with 100% of our patient responded to this triple combination. And the complete response rate was over 90% and the treatment with a higher rate of a moderate complete molecular response as well. So, the result of this analysis supports additional study.

The study was designed to enroll 20 to 30 patients with MCL who never treated it before. The primary end point is the safety analysis, and the secondary objectives includes efficacy, complete response, partial response, and overall response. Acalabrutinib was given from cycle 1 day 1 at 100 mg BID, until disease progression or intolerance. Venetoclax was given on cycle 2 day 1 with a 5 week ramp up period of dose escalation through cycle 5 to 25. Rituximab was given on day 1 of every cycle for 6 cycles and then after that, every other cycle until cycle 24. So, this is how dose limiting toxicity was assessed from cycle 2 day 1 to cycle 3, day 2.

What was most interesting about the results of the study?

What is striking and noteworthy for this study in that number 1, the combination is very safe. The patients tolerated the therapy very well. There are no toxins deaths, unfortunately 4 patients died from COVID without any relationship to our study therapy. The other issue is that the efficacy is very, very high. One hundred percent of our patients responded to therapy, and the complete response rate by PET scan was 90%.

What was the safety of the triplet? Did you see anything out of the ordinary?

The study therapy was tolerated very well. The most common side effects included diarrhea, headache, and fatigue, but the majority of those side effects are grade 1 or 2. There's hardly any grade 3 or 4 toxicities except diarrhea.

With ibrutinib, we are always concerned about atrial fibrillation. However, among the 21 patients, none of the patients had atrial fibrillation with a steady combination, and bleeding, accord in 33% of patients. And they were all grade 1 or grade 2, there was no grade 3 or 4 bleeding.

What unmet need is this combination filling in this population?

The unmet needs of this population, which is elderly patients with a newly diagnosed MCL, in the past and until now, internationally, we are treating these patients with chemotherapy. So, this study gave us a good result with chemo free therapy that has a very superb safety and efficacy profile. In order to replace chemotherapy, we have tried to doublets, we have tried to ibrutinib with rituximab in the frontline. We published this article recently in 

, and the response rate is very high. However, the cardiotoxicity was noted with atrial fibrillation.

So, although the doublet is very efficacious, we think that with the triplicated chemo free therapy in the frontline, we will have a better efficacy analysis, and hopefully, without severe toxicities. This study just showed that the response rate could be so high and 100%, it cannot be better. The complete response is 90% by PET scan, it can hardly be better. And the side effect profile was very tolerable and very tolerable. And that's, a good study, a wonderful combination with a safety profile and highest response rate that can be attained during the clinical trial. However, I want to note that we only have 21 patients in this trial. And in order to confirm this result, we need to we need more patients in the future trials.

Wang M, Robak T, Maddocks K, et al. Safety and efficacy of acalabrutinib plus venetoclax and rituximab in patients with treatment-naïve (TN) mantle cell lymphoma (MCL). Presented at: 2021 ASH Annual Meeting; December 9-14, 2021; Atlanta, GA. Abstract 3845.

In an interview with Targeted Oncology, Michael Wang, MD, a professor in the department of lymphoma and myeloma at MD Anderson Cancer Center, discussed the safety, efficacy, and tolerability of acalabrutinib plus venetoclax and rituximab in mantle cell lymphoma. 

Acalabrutinib Plus Venetoclax and Rituximab Signals Safety, Efficacy in MCL

Aspacytarabine (BST-236) for the treatment of newly diagnosed acute myeloid leukemia patients (AML) who are unfit for standard induction therapy, demonstrated acceptable safety, efficacy, and tolerability, according to a press release by Biosight Ltd.

Aspacytarabine is a novel anti-metabolite composed of cytarabine covalently bound to asparagine, thus acting as a prodrug of cytarabine. Cytarabine has been a main part of AML treatment for 45 years due to efficacy. However, it is associated with severe bone marrow, neurological, and gastrointestinal toxicities, limiting its use. Aspacytarabine allows for high-dose therapy with lower systemic exposure to free cytarabine while relatively sparing normal tissue.

The phase 2 trial (NCT03435848) of the agent had an estimated enrollment of 65 participants and an estimated study completion date of 2022. The primary end point of the study is complete remission 28 to 35 days after induction.

During the study, patients receive 1 to 4 courses of aspacytarabine Intravenously at 4.5 g/m2/d or 2.5 g/m2/d, for 6 days.

According to data presented at the 2021 American Society of Hematology Annual Meeting and Exposition, at the time of data cutoff, 65 patients from the phase 2b study with AML were included in the safety and efficacy analysis. All patients were unfit for standard induction chemotherapy and had a median age of 75 years. Thirty-eight percent of patients had an ECOG performance status of 2, 40% had secondary AML, and 17% received prior hypomethylating agents with or without venetoclax treatment.

The complete response rate was 37% across all evaluable patients, 27% in patients who received prior hypomethylating agents with or without venetoclax treatment, 44% in 

 AML patients, and 27% in secondary AML patients. Half of all evaluable complete responses were minimal residual disease negative. Additionally, rapid complete hematological recovery was observed in all patients who achieved a complete bone marrow response, with a median time of 25 days for complete neutrophil recovery. A complete platelet recover was observed for 26 days.

“These final complete remission data are extremely encouraging and strengthen our conviction in the potential of aspacytarabine as a potential standard-of-care treatment option for AML patients,” said Jessica Altman MD, professor, medicine, Hematology Oncology Division, Feinberg School of Medicine, Robert H. Lurie Comprehensive Cancer Center of Northwestern University, and lead study investigator in a press release. “Notably, a complete remission rate of 37% was achieved in a challenging patient population, including patients with secondary AML, prior hypomethylating agent treatment, and older age. While follow-up is still ongoing, the efficacy data across key measures, including duration of response and overall survival, combined with a favorable safety profile and a time-limited treatment of up to 4 courses, all form the basis for my confidence in aspacytarabine as a novel therapy for the treatment of patients with AML.”

In order to participate in the study, patients must be 18 years of age or older, not be eligible for standard induction therapy, and have an ECOG performance status of 2 or less at screening. Patients with relapsed or refractory AML, acute promyelocytic leukemia, an active malignant disease other than AML, congestive heart failure, or a life expectancy of less than 3 months are not eligible to participate.

“We continue to be very encouraged by the promising results of this larger data set, reinforcing our belief that aspacytarabine has the potential to become standard of care in patients with AML and may address a significant unmet need for those unfit for intensive chemotherapy. These results strongly support the continued progression of our aspacytarabine clinical trial program, with the recently announced expansion into additional Phase 2 studies in patients with relapsed or refractory AML and myelodysplastic syndrome,” said Ruth Ben Yakar, PhD, chief executive officer of Biosight, in a press release.

Biosight reports final primary endpoint data from phase 2b study of aspacytarabine (BST-236) for first-line acute myeloid leukemia therapy. News release. Biosight. January 6, 2022. Accessed January 10, 2022. https://bit.ly/3tix4Zl

Efficacy and safety of BST-236 in newly diagnosed acute myeloid leukemia patients, unfit for standard induction therapy (ELPIS). ClinicalTrials.gov. Accessed January 10, 2022. https://bit.ly/3JR9VTP

Aspacytarabine for acute myeloid leukemia showed a complete response rate of 37% in phase 2 study. 