View the full interview at https://checkrare.com/fda-approves-gazyva-to-reduce-relapse-risk-in-patients-with-idiopathic-nephrotic-syndrome/

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Ali A. Habib, MD, Associate Clinical Professor, University of California, Irvine, presented the results of an exploratory subanalysis of the NIMBLE trial. This work evaluated the ability of the C5-complement inhibitor cemdisiran to prevent hospitalization in patients with gMG. Dr. Habib described the investigation as an initial test of this exploratory endpoint.

As the medical community tries to unlock the potential of the targeted therapies on the inpatient treatment of myasthenic exacerbation and crisis, keeping the patient out of the hospital by preventing these events is a broader, extremely important goal.

The overall NIMBLE study was a phase 3 design, in which patients were randomized to receive one of the following regimens SC for 24 weeks:

Cemdisiran 600 mg q12wk
Pozelimab 200 mg q4wk
Cemdisiran + Pozelimab (200 mg q12wk each)
Placebo
For this exploratory analysis, 24-week data from the cemdisiran monotherapy and placebo groups only were analyzed, to determine hospitalization (from any cause), number of myasthenic crises, and the use of rescue therapy during the 24-week period.

Based on the safety analysis dataset, the average age of the 72 patients in the placebo group was 48.9 years compared with 53.4 years for the 79 patients in the cemdirisan group. The groups were well matched for race, gender, duration of gMG (8.7 vs 9.2 yr, respectively), and the presence of anti-AChR antibodies (94%-95%) and anti-LRP4 antibodies (6%–12%). The researchers evaluated days of hospitalizations and hospitalization days.

In the 2 years before study randomization, participants in the placebo arm had a mean 1.6 hospitalizations versus 1.3 hospitalizations in the active treatment group. Dr. Habib stated that only 1 participant in the cemdisiran group was hospitalized during the study period compared with 11 in the placebo group (1 day of MG-related hospitalization vs. 92 patient-days, respectively). “The median duration of each hospital stay was 1 day for the patients receiving cemdisiran compared with 7 days for the placebo group,” he said.

Of the participants taking cemdisiran, three (3.8%) experienced at least one actual or impending myasthenic crisis, compared with 11 (15%) experiencing crises in the placebo group. Related to this finding, two participants (2.5%) receiving active treatment were administered rescue therapy versus 11 (15%) receiving the placebo.

Dr. Habib noted that cemdisiran was generally well-tolerated. The incidence of treatment-emergent adverse events in this study was lower in the cemdisiran group than in the placebo group (34.2% vs. 41.7%, respectively). No serious infections were observed.

Dr. Habib concluded that, in this exploratory analysis, cemdisiran seemed to be associated with fewer hospitalizations (and hospitalization days) and myasthenic crises relative to patients who received placebo. This trial may pave the way for further investigations of resource use reduction in gMG, as well as support the evidence base for the preventive clinical benefit of this complement inhibitor.

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Naji Gehchan, MD, MBA, Chief Medical and Development Officer at Kyverna Therapeutics, discusses one-year data showing sustained benefits of KYV-101 (mivocabtagene autoleucel ; Miv-cel) in stiff person syndrome (SPS), along with longer-term follow-up data in generalized myasthenia gravis (gMG).

SPS and gMG are rare neurological and neuromuscular disorders:

SPS: SPS is a rare, progressive neurological autoimmune disease that causes muscle stiffness and painful spasms that can affect walking and mobility. Up to 80% of patients lose their mobility over time, and there are currently no FDA-approved treatments. 

gMG: gMG is a rare autoimmune neuromuscular disease characterized by muscle weakness and fatigue. As the disease progresses, it can interfere with daily activities, such as speaking, chewing, swallowing, and breathing. 

Positive one-year topline data from the KYSA-8 clinical trial (NCT06588491) were announced on September 24, 2026. KYSA-8 was a registrational, phase two, open-label, single-arm, multicenter study of miv-cel.

Miv-cel is a fully human, autologous, CD19-targeting CAR T-cell therapy with CD28 co-stimulation. It’s designed to achieve deep B-cell depletion, reset the immune system, and deliver durable drug-free, disease-free remission in autoimmune diseases with a single dose. 

A total of 26 adult patients who had an inadequate response to at least one immunotherapy treatment participated in the study. Each participant received a single dose of miv-cel. Among patients who achieved a clinically meaningful improvement in the timed 25-foot walk at primary analysis, 95% maintained that benefit at 12 months. Of the 12 patients who needed a walking aid before treatment, 67% still did not require assistance at the 12-month follow-up. Furthermore, 92% of patients remained off chronic immunotherapies for SPS. 

Also announced were longer-term follow-up data for its KYSA-6 trial (NCT06193889). KYSA-6 is a phase 2/3 trial evaluating miv-cel, administered as a single dose, in patients with gMG.  

A total of seven adult patients participated in the study. Follow-up now extends to one year and beyond (up to 1.5 years) in five patients, with one patient at 9 months and another at 6 months. All seven participants achieved clinically meaningful improvements in both the Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores at 24 weeks, while all five participants with at least one year of follow-up maintained clinically meaningful improvements. Additionally, six of the seven patients remained off immunosuppressant therapies at their latest follow-up.   

Miv-cel continued to be well-tolerated in both studies. In the KYSA-8 trial, no high-grade cases of cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) and no cases of immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) were reported through the 12-month follow-up.

In the KYSA-6  trial, no high-grade CRS, no ICANS, and no cases of IEC-HS were reported during the available follow-up.

For more information, visit: https://ir.kyvernatx.com/news-releases/news-release-details/kyverna-therapeutics-reports-positive-one-year-data

To learn more about stiff person syndrome, myasthenia gravis, and other rare neurological and neuromuscular disorders, visit: https://checkrare.com/diseases/neurology-nervous-system-diseases/

Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

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Should people be able to bet on clinical trial outcomes and FDA approvals?

Listen to the full episode at https://checkrare.com/gambling-on-clinical-trials-the-checkrare-brief/

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Nisha Joseph, MD, Associate Professor in the Department of Hematology and Medical Oncology at the Emory University School of Medicine, discusses how bispecific antibodies are changing multiple myeloma treatment. 

Multiple myeloma is a rare cancer of the plasma cells and the most common type of plasma tumor. Plasma cells are white blood cells that make antibodies to protect against infection. In multiple myeloma, these cells multiply rapidly, crowding out normal cells in the bone marrow and affecting the production of red blood cells, platelets, and other white blood cells. Symptoms include bones that break easily, frequent infections, fatigue, bruising, and bleeding. About 36,000 Americans are diagnosed with multiple myeloma each year.

Multiple myeloma is challenging to treat. Between 30% and 50% of patients don’t respond to first-line therapy, and almost everyone who initially achieves remission relapses or develops resistance over time. Because of this, researchers are always looking for new ways to combat the disease. 

Dr. Joseph said that bispecific antibodies are especially promising because they help a patient’s T cells recognize and attack the cancer. She described the process in simple terms. “It basically gloms onto the T cell, gloms onto the myeloma cell, and says, ‘Hey, look over here. Kill this guy.’” 

Furthermore, Dr. Joseph shared that bispecific antibodies can be combined with other myeloma drugs because they fight cancer in different ways. For example, some drugs, such as immunomodulatory agents and cereblon E3 ligase modulatory drugs (CELMoDs), stimulate the immune cells, while bispecific antibodies direct T cells to attack myeloma directly. Researchers are trying to determine whether combining these treatments can improve patient outcomes. 

Despite their potential benefits, Dr. Joseph said that undergoing bispecific antibody treatment requires short-term and long-term considerations. 

In the short term, bispecific antibodies are typically administered via a ramp-up period that takes about a week. Initial doses are small, but they increase over time, and that process requires several doctor visits. Dr. Joseph shared that this approach reduces the risk of cytokine release syndrome (CRS), which can cause fever, low blood pressure, and shortness of breath. Some patients may also experience neurological symptoms, which can require caregiver support.

The good news is that after the initial ramp-up period, Dr. Joseph said these side effects generally become much less of a concern.  

In the long term, bispecific antibodies can increase the risk of infection and low blood counts. Dr. Joseph noted that clinicians have learned that when it comes to using these drugs, “less is more.” She then explained that once a patient responds to treatment, doctors typically extend the time between doses to make it easier on the immune system. Preventive measures, such as antibody infusions and antibiotics, can also support these efforts.

Next, Dr. Joseph emphasized the importance of putting patients first when using bispecific antibodies. Even though these drugs are effective and typically well-tolerated, treatment is an ongoing process, requiring routine visits, something she described as “a marathon, not a sprint.” 

Even so, these drugs make multiple myeloma treatment more accessible. Unlike CAR T-cell therapy, which is primarily offered at larger academic centers with the necessary infrastructure, bispecific antibodies can be administered at smaller facilities, like community treatment centers. 

To learn more about multiple myeloma and other rare hematologic disorders, visit: https://checkrare.com/diseases/hematologic-disorders/

To learn more about Dr. Joseph’s background and areas of expertise, visit: https://winshipcancer.emory.edu/profiles/joseph-nisha.php

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In this episode, we discuss gambling on clinical trials, the FDA approval of a new formulation of Sjögren’s disease treatment, and an approval in IgA nephropathy.

Produced by CheckRare.

Explore additional CME activities, physician interviews, podcasts, and rare disease resources at CheckRare.com. Subscribe to the CheckRare Podcast Network for the latest rare disease education, expert interviews, and weekly news.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

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In this episode, we discuss gambling on clinical trials, the FDA approval of a new formulation of Sjögren’s disease treatment, and an approval in IgA nephropathy.

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In this episode, endocrinologist Margarita Ochoa-Maya, MD, speaks with endocrinologist Katherine Arakay, MD, of the University of California, Los Angeles (UCLA), about clinical trials for rare endocrine disorders. The conversation examines clinical trials from three perspectives: patients, clinical researchers, and sponsors.

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In this episode, endocrinologist Margarita Ochoa-Maya, MD, speaks with endocrinologist Katherine Arakay, MD, of the University of California, Los Angeles (UCLA), about clinical trials for rare endocrine disorders. The conversation examines clinical trials from three perspectives: patients, clinical researchers, and sponsors.

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