What Is Batoclimab?
Batoclimab is a type of medication known as a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody. This means it is a specially engineered protein designed to target and block the FcRn protein in the body. The FcRn protein plays a crucial role in preventing the breakdown of certain antibodies, including those that can cause autoimmune conditions. By blocking FcRn, Batoclimab helps to reduce the levels of these disease-causing antibodies. This mechanism of action suggests its potential to treat various autoimmune diseases where excessive or harmful antibodies contribute to the illness. Batoclimab is currently being investigated in clinical trials for several autoimmune conditions.
Uses and Conditions Under Study
Batoclimab is currently under investigation in clinical trials for several autoimmune conditions. These conditions involve the immune system mistakenly attacking the body's own tissues.
The conditions being studied include:
- Thyroid Eye Disease: This autoimmune condition, studied in 3 trials, involves the immune system attacking tissues around the eyes, leading to inflammation and damage. Batoclimab's mechanism of reducing certain antibodies may help to lessen this autoimmune response.
- Graves Disease: Investigated in 1 trial, Graves Disease is an autoimmune disorder that causes an overactive thyroid. Batoclimab aims to modulate the immune response involved in this condition.
- Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): This rare neurological disorder is being studied in 2 trials. CIDP involves the immune system attacking the myelin sheath of nerve fibers, leading to muscle weakness and sensory problems. Batoclimab aims to reduce the autoimmune attack on nerves.
- Generalized Myasthenia Gravis (gMG): Studied in 1 trial, gMG is a chronic autoimmune neuromuscular disease characterized by fluctuating muscle weakness. By modulating antibody levels, Batoclimab aims to reduce the autoimmune attack on muscles in this condition.
Dosing
Clinical trials for Batoclimab have investigated various dosing regimens and forms. One specific regimen studied is Batoclimab 340 mg administered subcutaneously (SC) once weekly (QW). This indicates an injectable form of the medication, given under the skin. Studies have explored different treatment periods, including induction and maintenance phases, with varying doses referred to as "Dose 1" and "Dose 2" across different cohorts. For example, trials included "Treatment Period 1: Cohort A, Dose 1" and "Treatment Period 1: Cohort A, Dose 2", as well as similar variations for Cohorts B, C, and D. There were also "Withdrawal Period 2" regimens, which included Batoclimab at "Dose 2" or placebo, and "LTE Period" (Long-Term Extension) regimens for participants with or without relapse. These different dosing strategies are designed to evaluate the most effective and safe way to administer Batoclimab for the conditions under study, considering both initial treatment and longer-term management.
Side Effects
In a 12-week study of patients with irritable bowel syndrome with constipation (IBS-C) (NCT04079555), the most common side effect reported by patients taking Batoclimab was nausea. 11.6% of patients taking Batoclimab experienced nausea, compared to 6.7% on placebo. Other common side effects included:
- Diarrhea: 8.5% of patients taking Batoclimab experienced diarrhea, compared to 4.0% on placebo.
- Headache: 6.8% of patients taking Batoclimab experienced headache, compared to 5.0% on placebo.
- Abdominal pain: 6.1% of patients taking Batoclimab experienced abdominal pain, compared to 4.7% on placebo.
- Upper respiratory tract infection: 5.8% of patients taking Batoclimab experienced an upper respiratory tract infection, compared to 4.3% on placebo.
- Dizziness: 4.8% of patients taking Batoclimab experienced dizziness, compared to 2.3% on placebo.
In a separate 4-week study of patients with hyperphosphatemia undergoing dialysis (NCT03399345), specific side effects related to dialysis were observed. 10% of patients taking Batoclimab experienced an AV fistula complication, compared to 0% on placebo. Hyperkalemia (high potassium levels) was reported by 7% of Batoclimab patients, while no placebo patients experienced it. Hypophosphatemia (low phosphate levels) was also reported by 7% of Batoclimab patients, compared to 0% on placebo.
In an open-label extension study for IBS-C patients, where no placebo comparison was available, common events observed over 52 weeks included hypertension (8.5%), upper respiratory tract infection (7.8%), and headache (7.5%).
Clinical Trial Results
Irritable Bowel Syndrome with Constipation (IBS-C)
A 12-week Phase 2 study (NCT04079555) evaluated Batoclimab in patients with IBS-C. The primary goal was to determine the percentage of "Overall Responders," defined as patients experiencing at least a 30% reduction in weekly worst abdominal pain and at least one complete spontaneous bowel movement (CSBM) per week for at least 6 of the 12 weeks. In this study, 44% of patients taking Batoclimab met the criteria for an Overall Responder, compared to 33% of patients on placebo. This represented an 11% difference between the groups.
Key secondary outcomes also showed positive results:
- Abdominal pain responder: 52% of patients on Batoclimab experienced at least a 30% reduction in weekly worst abdominal pain for at least 6 of 12 weeks, compared to 42% on placebo.
- Stool consistency improvement: 55% of patients on Batoclimab showed at least a 1-point increase from baseline on the Bristol Stool Scale for at least 6 of 12 weeks, compared to 43% on placebo.
- CSBM responder: 50% of patients on Batoclimab had at least one CSBM per week for at least 6 of 12 weeks, compared to 40% on placebo.
Hyperphosphatemia in Dialysis Patients
A 4-week Phase 2 study (NCT03399345) investigated Batoclimab in patients with hyperphosphatemia (high phosphate levels) who were undergoing dialysis. The primary endpoint measured the change in serum phosphate levels from baseline to week 4. Patients treated with Batoclimab experienced a mean reduction of 1.8 mg/dL in serum phosphate, indicating an improvement. In contrast, patients on placebo had a mean increase of 0.2 mg/dL in serum phosphate. The difference between the groups was a 2.0 mg/dL reduction with Batoclimab.
An important secondary outcome was the percentage of patients who achieved target phosphate levels (below 4.5 mg/dL). 50% of patients receiving Batoclimab reached this target, while none (0%) of the patients on placebo achieved it.
Currently Recruiting Trials
Patients interested in contributing to medical research for Batoclimab have an opportunity to participate in a clinical trial designed to further understand its long-term effects. These studies are crucial for gathering comprehensive data on new treatments and advancing therapeutic options for various conditions.
One such trial, identified as NCT07188844, is an open-label extension study focusing on adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). CIDP is a rare neurological disorder characterized by progressive weakness and impaired sensory function due to damage to the myelin sheath of peripheral nerves. This Phase 2 study aims to evaluate the long-term safety and tolerability of Batoclimab in individuals who have already completed a previous study, IMVT-1401-2401 (NCT05581199).
In this ongoing trial, participants will receive a dosage of 340 mg of Batoclimab, administered subcutaneously once weekly. The study is designed to provide extended access to the investigational treatment for those who may have benefited from it during the initial trial. Open-label extension studies like this one are vital as they allow researchers to monitor the effects of a drug over an extended period, providing valuable insights into its sustained benefits and potential side effects beyond the initial treatment phase. For patients living with CIDP, understanding the long-term profile of a treatment like Batoclimab is essential for managing a chronic condition that significantly impacts quality of life. The sponsor, Immunovant Sciences GmbH, is targeting an enrollment of 108 participants for this important extension study, contributing to a deeper understanding of Batoclimab's role in managing CIDP.
Where to Participate
The current recruiting trial for Batoclimab, NCT07188844, is seeking participants across five different states in the United States. This geographic spread helps ensure a diverse representation of patients in the study.
Study sites are located in:
- New Haven, Connecticut
- Ormond Beach, Florida
- Nicholasville, Kentucky
- Charlotte, North Carolina
- Austin, Texas
To be eligible for participation in this specific trial, individuals must be adults, aged 18 years or older. The study is open to participants of all genders. It is important to note that this trial is not seeking healthy volunteers; participants must have a diagnosis of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and have completed the prior study. Children are not eligible to participate.
Development Timeline
The journey of Batoclimab in clinical development began on June 3, 2022, with its first clinical trial. Since then, Immunovant Sciences GmbH has been the sole sponsor, driving the research and development efforts for this investigational drug. Over this period, a total of seven clinical trials have been initiated, involving a cumulative enrollment of 1,040 participants.
Batoclimab's development pipeline has shown a strategic expansion in its target conditions. Initially, the focus was on conditions such as Irritable Bowel Syndrome with Constipation (IBS-C) and hyperphosphatemia. As research progressed, the scope broadened to include other significant autoimmune disorders. The program expanded to investigate Batoclimab for Generalized Myasthenia Gravis and Graves Disease, reflecting its potential across a wider range of conditions.
The development has also seen progression through different clinical phases, with three trials conducted in Phase 2 and four trials advancing to Phase 3. This progression indicates a growing understanding of Batoclimab's efficacy and safety profile. The latest trial is projected to conclude by September 23, 2025, marking a significant milestone in its ongoing development.