Peter Voorhees, MD, Atrium Health and Levine Cancer Institute, Wake Forest University School of Medicine, discusses results from the CARTITUDE-1 study in patients with multiple myeloma (MM).

MM is a bone marrow-based plasma cell neoplasm characterized by a serum monoclonal protein and skeletal destruction. Common symptoms include osteolytic lesions, pathological fractures, bone pain, hypercalcemia, and anemia. The exact underlying cause of multiple myeloma is currently unknown.

Cilta-cel is a B-cell maturation antigen-directed genetically modified autologous T cell immunotherapy. The CARTITUDE-1 clinical trial was a phase 1b/2, open-label study evaluating cilta-cel in patients with relapsed/refractory MM who had been heavily pretreated. A total of 97 patients received a single infusion of cilta-cel.

Of the 97 patients treated, 32 remain alive and progression free after five years or more of receiving cilta-cel, without further treatment. Of these 32 patients, the median time from the start of their previous line of therapy to disease progression prior to trial enrollment was 4.0 months. Those who remained progression-free for 5 or more years, despite having high-risk cytogenetics or extramedullary disease, shared baseline characteristics comparable to those who experienced progression within 5 years.

Several biomarkers significantly correlated with ≥5-year progression-free status. These included a higher proportion of naïve T cells in the infused CAR-T product, a lower neutrophil-to-T cell ratio, elevated baseline hemoglobin and platelet levels, and a higher effector-to-target ratio (measured by peak CAR-T concentration to baseline sBCMA levels). At peak concentration, long-term responders also had significantly higher levels of CD4 central memory CAR+ T cells and CAR+ T cells expressing activation markers CD38, CD25, and PD-1.

At one single center, all 12 patients who were progression-free for ≥5 years were consistently MRD-negative at 10⁻⁶ and PET/CT-negative annually for 5 years.

Overall, at a median follow-up of 60.3 months in the full CARTITUDE-1 cohort (N=97), median overall survival was 60.6 months (95% CI, 41.9–not estimable). Three additional cases of second primary malignancies were reported, including one case of acute myeloid leukemia. No new instances of movement or neurocognitive disorders were observed. The median overall survival was 5 years and 33% of patients remain progression free for 5 or more years following a single cilta-cel infusion.

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Andrew T. Kuykendall, MD, Associate Member in the Department of Hematology at Moffitt Cancer Center, discusses results from the VERIFY clinical trial in polycythemia vera (PV).

PV is a rare condition characterized by an increased number of red blood cells in the bloodstream. Affected people may also have excess white blood cells and platelets. These extra cells cause the blood to be thicker than normal, increasing the risk for blood clots, including deep vein thrombosis (DVT). The condition has been associated with genetic changes in the JAK2 and TET2 genes.

Rusfertide is an investigational, first-in-class hepcidin mimetic peptide therapy with potential to regulate iron homeostasis and red blood cell production, therefore controlling hematocrit levels in patients with PV.

The VERIFY clinical trial is a phase 3, ongoing, three-part, global, randomized, placebo-controlled study evaluating rusfertide in patients with PV. The study enrolled 293 patients with PV who were dependent on frequent phlebotomy, with or without treatment with cytoreductive therapy. Patients were randomized to receive once-weekly, subcutaneously self-administered rusfertide or placebo, plus standard of care treatment.

Data from the clinical trial was recently presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Il. The primary endpoint, the proportion of patients achieving a clinical response, defined as absence of phlebotomy eligibility during weeks 20-32, was met. Results showed that 76.9% of patients treated with rusfertide plus standard of care achieved a clinical response compared to 32.9% in the placebo plus standard of care group.

All key secondary endpoints also met statistical significance including the mean number of phlebotomies; 0.5 in the rusfertide group, compared to 1.8 in the placebo group during weeks 0-32. Patients requiring a phlebotomy between weeks 0-32 was at 27% in the rusfertide group versus 78% in the placebo group. 62.6% of patients treated with rusfertide maintained hematocrit levels below 45% compared to 14.4% treated with placebo. Rusfertide also illustrated improvements in mean change from baseline to week 32 in PROMIS Fatigue and MFSAF Total Symptom Score.

Additionally, rusfertide had a favorable safety profile and was generally well tolerated. Most adverse events were low grade and non-serious. The most common adverse events were localized injection site reactions, anemia, and fatigue. No serious adverse events were reported. Rusfertide also showed no evidence of increased risk of cancer as compared to placebo.

Chapters:
Introduction 00:00
PV Overview 00:30
Current Management 1:59
Rusfertide Overview 3:13
VERIFY Clinical Trial 4:47
Take Home Message 8:33

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Jorge Cortes, MD, Director of the Georgia Cancer Center, offers advice to newly diagnosed patients with chronic myeloid leukemia (CML).

CML is a myeloproliferative neoplasm characterized by the expression of the BCR-ABL1 fusion gene. It presents with neutrophilic leukocytosis. Around 90% to 95% of patients have a shortened chromosome 22, or Philadelphia chromosome.

The condition can manifest at any age but most commonly affects middle aged and older individuals. Patients usually present with fatigue, weight loss, anemia, night sweats, and splenomegaly. Approximately 50% of patients diagnosed with CML were asymptomatic and diagnosed due to routine complete blood count.

Allogeneic stem cell transplantation and tyrosine kinase inhibitors are traditional first-lines of therapy for this condition. There are currently four U.S. Food and Drug Administration (FDA) approved tyrosine kinase inhibitors for first-line therapies for patients with CML: imatinib, bosutinib, dasatinib, and nilotinib. There are also several other therapies approved, or in development, as second- and third-line therapies for this rare cancer.

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Jorge Cortes, MD, Director of the Georgia Cancer Center, discusses treatment options for chronic myeloid leukemia (CML).

CML is a myeloproliferative neoplasm characterized by the expression of the BCR-ABL1 fusion gene. It presents with neutrophilic leukocytosis. Around 90% to 95% of patients have a shortened chromosome 22, or Philadelphia chromosome.

The condition can manifest at any age but most commonly affects middle aged and older individuals. Patients usually present with fatigue, weight loss, anemia, night sweats, and splenomegaly. Approximately 50% of patients diagnosed with CML were asymptomatic and diagnosed due to routine complete blood count.

Allogeneic stem cell transplantation and tyrosine kinase inhibitors are traditional first-lines of therapy for this condition. There are currently four U.S. Food and Drug Administration (FDA) approved tyrosine kinase inhibitors for first-line therapies for patients with CML: imatinib, bosutinib, dasatinib, and nilotinib. There are also several other therapies approved, or in development, as second- and third-line therapies for this rare cancer.

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Jorge Cortes, MD, Director of the Georgia Cancer Center, provides an overview of chronic myeloid leukemia (CML) and its current landscape.

CML is a myeloproliferative neoplasm characterized by the expression of the BCR-ABL1 fusion gene. It presents with neutrophilic leukocytosis. Around 90% to 95% of patients have a shortened chromosome 22, or Philadelphia chromosome.

The condition can manifest at any age but most commonly affects middle aged and older individuals. Patients usually present with fatigue, weight loss, anemia, night sweats, and splenomegaly. Approximately 50% of patients diagnosed with CML were asymptomatic and diagnosed due to routine complete blood count.

Allogeneic stem cell transplantation and tyrosine kinase inhibitors are traditional first-lines of therapy for this condition. There are currently four U.S. Food and Drug Administration (FDA) approved tyrosine kinase inhibitors for first-line therapies for patients with CML: imatinib, bosutinib, dasatinib, and nilotinib. There are also several other therapies approved, or in development, as second- and third-line therapies for this rare cancer.

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Jorge Cortes, MD, Director of the Georgia Cancer Center, provides an overview of chronic myeloid leukemia (CML), discusses treatment options, and offers advice to newly diagnosed patients.

CML is a myeloproliferative neoplasm characterized by the expression of the BCR-ABL1 fusion gene. It presents with neutrophilic leukocytosis. Around 90% to 95% of patients have a shortened chromosome 22, or Philadelphia chromosome.

The condition can manifest at any age but most commonly affects middle aged and older individuals. Patients usually present with fatigue, weight loss, anemia, night sweats, and splenomegaly. Approximately 50% of patients diagnosed with CML were asymptomatic and diagnosed due to routine complete blood count.

Allogeneic stem cell transplantation and tyrosine kinase inhibitors are traditional first-lines of therapy for this condition. There are currently four U.S. Food and Drug Administration (FDA) approved tyrosine kinase inhibitors for first-line therapies for patients with CML: imatinib, bosutinib, dasatinib, and nilotinib. There are also several other therapies approved, or in development, as second- and third-line therapies for this rare cancer.

Chapters:
Introduction 00:00
The CML Landscape 00:45
Choosing Treatment Strategies 2:07
Long Term Goals in Treatment 4:23
TKIs and Fasting 5:17
Advice to Newly Diagnosed Patients 6:45

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Jonathan Strober, MD, Director of the Neuromuscular Clinic and Professor of Pediatrics and Neurology at the University of California at San Francisco, discusses the implications of nipocalimab's approval to treat pediatric patients with myasthenia gravis (MG).

MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing. Weakness tends to increase during periods of activity and improve after periods of rest. The condition results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against acetylcholine. The exact reason this occurs is not known.

The U.S. Food and Drug Administration (FDA) recently approved Imaavy (nipocalimab) for the treatment of generalized MG in patients ages 12 years and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) antibody positive. Nipocalimab is a human monoclonal antibody FcRn-inhibitor designed to reduce immunoglobulin G (IgG) autoantibodies.

The approval was supported by data from the Vivacity-MG3 clinical trial (NCT04951622). The study illustrated superior disease control through 24 weeks, measured by MG Activities of Daily Living score, in patients treated with nipocalimab plus standard of care versus those treated with placebo plus standard of care. A rapid and sustained reduction in autoantibody levels by up to 75% from the first dose and throughout a 24-week period of monitoring was also observed. Additionally, improvements in patients treated with nipocalimab plus standard of care were maintained out to 20 months of follow-up in the open-label extension.

In addition to the above study, nipocalimab is also being tested in patients as young as 2 years of age. Results from the ongoing Vibrance phase 2/3 pediatric study (NCT05265273), patients aged 2 to 17 years, showed that nipocalimab plus standard of care met its primary endpoint with a 69% reduction in total serum IgG over 24 weeks. Improvements were also seen in MG Activities of Daily Living and Quantitative MG scores. The safety profile of nipocalimab has been favorable and consistent across both studies.

Chapters:
Introduction 00:00
MG Treatment Landscape 00:42
Nipocalimab Overview 2:48
Vivacity-MG3 Clinical Trial 3:38
Implications for Physicians 4:07

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Aravindhan Veerapandiyan, MD, Pediatric Neuromuscular Neurologist at Arkansas Children’s Hospital, discusses new data from the EMBARK part 2 study of Elevidys (delandistrogene moxeparvovec) in patients with Duchenne muscular dystrophy (DMD).

DMD is a rare genetic, neuromuscular disease that leads to progressive muscle wasting. DMD is an x-linked disorder and primarily impacts males. The symptoms of DMD include progressive weakness and atrophy of both skeletal and heart muscles. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. DMD is caused by genetic changes in the DMD gene.

Delandistrogene moxeparvovec is a single-dose, adeno-associated virus (AAV) gene therapy administered via intravenous infusion. It is designed to address the disease-causing gene mutations in the DMD gene that result in a lack of dystrophin protein by targeting production of micro-dystrophin in the skeletal muscle.

New data from the EMBARK trial was presented at the 2025 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. Patients included in this part 2 study had received placebo in part 1 and were aged 8 to 9 years at crossover. At one year post treatment with delandistrogene moxeparvovec, statistically significant improvements were observed including 4.75 points on North Star Ambulatory Assessment, a 6.87 second time-to-rise from floor, and a 4.76 second 10-meter walk/run compared to a control cohort. Additionally, no new safety signals were observed and micro-dystrophin expression and sarcolemmal localization were sustained from week 12 to week 64.

Chapters:
Introduction 00:00
DMD Treatment Landscape 00:41
Elevidys Overview 1:40
EMBARK Clinical Trial 3:10
Implications for Physicians and Patients 5:10

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Claudia Sommer, MD, Professor of Neurology at the University of Würzburg, Germany, and Alex Seluzhytsky, MD, Senior Global Medical Director at Sanofi, discuss data on riliprubart for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP).

CIDP is a rare neurological disorder that causes progressive weakness and impaired sensory function, most notably in the legs and arms. Symptoms often include tingling or numbness (first in the toes and fingers), weakness of the arms and legs, loss of deep tendon reflexes, fatigue, and abnormal sensations. Other symptoms may include pain, dysphagia, and diplopia. CIDP is considered to be an autoimmune disease that targets the myelin sheath of the peripheral nerves.

Riliprubart is an investigational IgG4 humanized monoclonal antibody designed to selectively inhibit activated C1 protein in the classical complement pathway.

Data on riliprubart was recently presented at the 2025 Peripheral Nerve Society Annual Meeting from a phase 2 multicenter, open-label, non-randomized, proof-of-concept study evaluating the efficacy, safety, and tolerability of riliprubart in adults with CIDP. Patients were split into three cohorts: those on standard of care, those refractory to standard of care, and those who were treatment-naïve. All three groups showed significant improvement in disease following use of riliprubart.

Next steps include phase 3 trials where riliprubart will be compared to placebo. Two of these trials are ongoing and currently recruiting patients.

Chapters:
Introduction 00:00
CIDP Overview 00:54
Diagnostic Journey 2:03
Red Flag Symptoms 3:31
Current Management 4:13
Riliprubart Overview 5:10
Clinical Trials 7:16
Next Steps 9:12

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