FDA Approves Bristol Myers Squibb’s Reblozyl as First-Line Treatment of Anemia in Adults with Lower-Risk MDS Who May Require Transfusions

Bristol Myers Squibb announced that the Food and Drug Administration (FDA) has approved Reblozyl® (luspatercept-aamt) for the treatment of anemia in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions. Based on interim results from the pivotal Phase III COMMANDS trial, Reblozyl demonstrated superior efficacy of concurrent RBC transfusion independence (RBC-TI) and hemoglobin (Hb) increase compared to epoetin alfa, an ESA, regardless of ring sideroblast status. These findings support Reblozyl’s capacity to treat chronic anemia early in the treatment process in a broader variety of individuals.

For patients with lower-risk MDS, current standard therapies, including ESAs, have provided limited benefit in controlling anemia, with only 1 in 3 patients responding for a duration of 6-18 months,” said Guillermo Garcia-Manero, M.D., lead investigator and Chief of the Section of Myelodysplastic Syndromes at The University of Texas MD Anderson Cancer Center. “According to the findings of the COMMANDS study, nearly twice as many patients treated with Reblozyl achieved transfusion independence for at least 12 weeks and concurrent hemoglobin increase compared to epoetin alfa.” Today’s approval is a significant step forward for patients with low-risk MDS.

In the Phase III COMMANDS study, 58.5% (n=86) of Reblozyl patients vs. 31.2% (n=48) of epoetin alfa patients achieved the primary objective of RBC-TI of at least 12 weeks with a mean Hb rise of at least 1.5 g/dL within the first 24 weeks (p0.0001). Diarrhea, tiredness, hypertension, peripheral edema, nausea, and dyspnea were the most prevalent (>10%) side effects.

The COMMANDS study’s findings were presented in June as part of the press program at the American Society of Clinical Oncology (ASCO) Annual Meeting and as a plenary session at the European Hematology Association (EHA) Congress, with simultaneous publication in The Lancet. As of November 2021, Reblozyl will be developed and commercialized under a global agreement with Merck.

FDA Awards Orphan Drug Designation to NXC-201 for Multiple Myeloma

Nexcella, Inc. stated that the U.S. Food and Drug Administration (FDA) has given the Orphan Drug Classification (ODD) classification to NXC-201 for the treatment of multiple myeloma, a potentially fatal form of blood cancer. NXC-201, a next-generation CAR-T cell treatment, is now being examined in the NEXICART-1 (NCT04720313) Phase Ib/IIa clinical trial.

The FDA’s Office of Orphan Product Development bestows orphan designation status on medicines and biologics designed for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 persons in the United States. Following regulatory clearance, Orphan Drug Designation gives specific benefits, including financial incentives to encourage clinical research and the potential for up to 7 years of market exclusivity in the United States.

“We are pleased to receive the FDA’s orphan drug designation in multiple myeloma for NXC-201, the only clinical-stage BCMA-targeted CAR-T cell therapy with no neurotoxicity observed in over 50 patients dosed to date. We are thrilled to potentially expand therapeutic options for multiple myeloma patients while eliminating the most feared adverse effect of this therapeutic class, neurotoxicity.

Ilya Rachman, MD PhD, Nexcella’s Executive Chairman

Multiple myeloma is an incurable blood cancer of plasma cells that begins in the bone marrow and is distinguished by uncontrolled cell growth. As per the assessment done by DelveInsight, the total incident cases of multiple myeloma in the 7MM comprised more than 70,000 cases in 2022 and are projected to increase during the forecast period. DelveInsight estimates that the total incident multiple myeloma cases were the highest in the United States. Data suggests that roughly half of newly diagnosed multiple myeloma patients are ineligible for transplant, and around a third of eligible patients do not receive the transplant.

Janssen Submits Supplemental New Drug Application to the U.S. Food and Drug Administration Seeking Full Approval of BALVERSA

The Janssen Pharmaceutical Companies, a division of Johnson & Johnson, have officially submitted an additional application to the U.S. Food and Drug Administration (FDA). This application aims to secure full approval for BALVERSA (erdafitinib), a type of kinase inhibitor. BALVERSA is intended to be used in the treatment of adult patients who have locally advanced or metastatic urothelial carcinoma (mUC) with specific genetic alterations in the fibroblast growth factor receptor (FGFR)3 gene. These patients must have experienced disease progression either during or after treatment with at least one type of programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor in the locally advanced or metastatic setting, or within a year of neoadjuvant or adjuvant therapy.

BALVERSA had previously received Breakthrough Therapy Designation from the U.S. FDA in 2018 and was granted accelerated approval in 2019 for treating adults with locally advanced or mUC that had relevant genetic alterations in the FGFR3 or FGFR2 genes. These patients needed to have experienced disease progression after at least one round of platinum-containing chemotherapy, including within a year of neoadjuvant or adjuvant platinum-containing chemotherapy. This new supplemental application (sNDA) for BALVERSA aims to fulfill the regulatory requirements needed to establish the clinical benefits of the drug. This is based on the randomized data from Cohort 1 of the Phase 3 THOR study.

Dr. Peter Lebowitz, the Global Therapeutic Area Head for Oncology at Janssen Research & Development, LLC, emphasized that BALVERSA has shown promising results in clinical studies for patients with FGFR-altered metastatic urothelial cancer. These patients often have poor disease outcomes, and the goal is to positively impact their lives through targeted therapy.

The sNDA submission relies on information gathered from Cohort 1 of the Phase 3 THOR study, a multicenter study that assessed the efficacy and safety of BALVERSA. This study successfully met its primary goal of improving overall survival. Patients who were treated with BALVERSA achieved a median overall survival of more than one year at the interim analysis data cutoff point.

The interim outcomes satisfied the pre-established criteria for showing that BALVERSA was superior to chemotherapy. As a result, an independent data safety monitoring committee recommended halting the study and allowing patients who were assigned to chemotherapy to switch to BALVERSA treatment. The safety profile of BALVERSA observed in the THOR study aligned with the known safety information of the drug in mUC. The results from Cohort 1 were presented during a Late-Breaking Presentation Session at the 2023 American Society of Clinical Oncology Annual Meeting.

FDA Grants Fast Track Status to ALE.C04 for Recurrent or Metastatic CLDN1+ HNSCC

Alentis Therapeutics has announced that the US Food and Drug Administration (FDA) has given its approval for the Fast Track development program for ALE.C04. This program is intended to expedite the development process and hasten the evaluation of new medications that are designed to address severe or life-threatening conditions that lack adequate medical solutions. ALE.C04 is aimed at treating patients with recurrent or metastatic Claudin-1 positive (CLDN1-positive) Head and Neck Squamous Cell Carcinoma (HNSCC).

The Fast Track designation from the FDA emphasizes the potential of ALE.C04 to tackle a significant medical requirement in the realm of cancer, particularly HNSCC. Dr. Roberto Iacone, Alentis’ Chief Executive Officer, remarked, “The FDA’s decision to award Fast Track designation highlights the capability of ALE.C04 to address a critical medical necessity within cancer, specifically focusing on HNSCC.” He further noted, “We are consistently making progress in our portfolio of antibodies targeting Claudin-1, a highly promising target with therapeutic applications spanning various indications in oncology and organ fibrosis.”

Dr. Luigi Manenti, Chief Medical Officer at Alentis, also expressed enthusiasm, saying, “We are enthusiastic about the opportunity to accelerate the development of ALE.C04 for patients with recurrent or metastatic, CLDN1-positive HNSCC. The ongoing Phase 1/2 clinical trial for HNSCC will furnish valuable insights into ALE.C04’s safety, pharmacodynamic characteristics, as well as its potential to combat tumors both as a standalone therapy and when used alongside pembrolizumab.”

AusperBio Announces FDA Clearance of IND Application of AHB-137 in Chronic Hepatitis B Treatment

AusperBio Therapeutics, Inc. and Ausper Biopharma Co., Ltd. (collectively known as AusperBio) have revealed that on August 25, 2023, they received confirmation from the U.S. Food and Drug Administration (FDA) regarding the approval of their Investigational New Drug (IND) application. This clearance enables them to proceed with a clinical trial involving AHB-137.

AHB-137 represents an unconjugated antisense oligonucleotide (ASO) that holds promise as a foundational element in the pursuit of a functional cure for chronic hepatitis B (CHB). The U.S.-based clinical trial forms a component of a larger, multi-region initiative. This randomized, double-blind, placebo-controlled study aims to investigate the safety, tolerability, pharmacokinetics, and preliminary efficacy of AHB-137 among patients grappling with CHB (clinicaltrials.gov # NCT05717686).

Earlier on June 28, 2023, AusperBio announced approval from China’s Center for Drug Evaluation (CDE) for the Investigational New Drug (IND) application of AHB-137. This approval is intended for the purpose of utilizing AHB-137 in the treatment of chronic hepatitis B (CHB) with the objective of achieving a functional cure.

The FDA’s clearance of our IND application to initiate clinical evaluation of AHB-137 in CHB patients in the United States brings us one step closer to introducing a potential functional cure for people living with HBV. This milestone is a pivotal juncture for AusperBio, underscoring our consistent and outstanding execution in driving innovative therapies towards a functional cure for CHB. 

AusperBio CEO and Co-founder Dr. Guofeng Cheng

We are committed to expediting patient access to innovative treatments. Our focus now is on working closely with key opinion leaders to initiate the CHB patient study in the US.

Dr. Cheng Yong Yang, CSO and Co-founder of AusperBio

Affecting approximately 290 million individuals across the globe, chronic Hepatitis B (CHB) infection is a liver ailment that can lead to enduring complications like cirrhosis and hepatocellular carcinoma. As per DelveInsight, the total prevalent cases of Chronic Hepatitis B in the 7MM comprised approximately 56,333,000+ cases in 2022. Moreover, the total prevalent cases of Chronic Hepatitis B in the United States were around 22,56,700+ cases in 2022. In the United States, approximately 55% of the patient share is attributed to males, whereas around 40% of females suffer from Chronic Hepatitis B. Among the EU4, Germany accounted for the largest number of Chronic Hepatitis B cases, followed by France, whereas Spain accounted for the lowest number of cases in 2022. While existing treatment choices can stifle HBV replication, attaining a complete cure is a rare occurrence. Consequently, there persists a pressing necessity for the identification of a definitive remedy for CHB. Companies across the globe are consistently working toward the development of new therapies for the treatment of Chronic Hepatitis B.

Faron Receives FDA Orphan Drug Designation for Bexmarilimab in Acute Myeloid Leukemia

Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON) revealed on August 29, 2023, that its fully owned asset, bexmarilimab, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for addressing Acute Myeloid Leukemia (AML).

Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to develop bexmarilimab as a potential treatment for Acute Myeloid Leukemia. The designation represents a milestone in our development journey, one that we believe, when combined with standard of care, will lead to better patient outcomes and improved quality of life. 

Chief Medical Officer Dr. Marie-Louise Fjällskog

Bexmarilimab is presently undergoing evaluation in the Phase I/II BEXMAB study (ClinicalTrials.gov: NCT05428969) as part of a combination with the standard of care (SoC). This study aims to address the treatment of aggressive hematological malignancies in cases of relapsed/refractory Acute Myeloid Leukemia (AML) and myelodysplastic syndromes (MDS). In the previous month, Faron disclosed updated and positive results emerging from the Phase I segment of the trial. Within the cohort receiving a combination of 6 mg/kg bexmarilimab and azacitidine, three out of five patients achieved objective responses. Across all three dosing cohorts in the doublet approach, a total of eight objective responses were observed out of 15 patients, with one patient continuing treatment for a span of 13 months. The completion of dose escalation, readout of enrichment cohorts, and initiation of the Phase II portion of the BEXMAB trial are expected in Q4 2023.

As per DelveInsight’s latest Acute Myeloid Leukemia Market assessment report, in 2022, the total incident cases of Acute Myeloid Leukemia were 42,000+ in the 7MM, which might reach ~48,000+ cases by 2032. In the 7MM, the highest number of incident cases of Acute Myeloid Leukemia was observed in the US. Similarly, the rate of new cases of Acute Myeloid Leukemia is around 4.3 per 100,000 men and women per year in the United States. The rising number of cases and the unmet therapeutics need lead to a greater burden of Acute Myeloid Leukemia. Several major pharma and biotech companies are in the process of developing and delivering new therapeutic solutions to the market to overcome the existing challenges.