Sutimlimab shows promise for hard-to-treat, rare blood disorder

In a first-in-human clinical trial reported today in Blood, the investigational drug sutimlimab appeared to be effective in treating cold agglutinin disease, a rare chronic blood disorder for which there are currently no approved treatments. Cold agglutinin disease is caused by a malfunction in the immune system that causes antibodies - components of the immune system that are produced in the blood and help the body fight off disease - to mistakenly latch onto and kill red blood cells. The disease is a type of hemolytic anemia, a condition that occurs when the bone marrow can't produce red blood cells as quickly as they are destroyed.

In the study, sutimlimab, a specific C1s inhibitor, rapidly halted the destruction of red blood cells, increased hemoglobin levels, eliminated patients' need for blood transfusions, and caused no serious adverse effects.

"The drug was well tolerated, produced clinically meaningful increases in hemoglobin levels, and precluded the need for transfusions, even in patients for whom multiple prior therapies had failed," said senior author Bernd Jilma, MD, of the Medical University of Vienna.

Cold agglutinin disease is thought to affect about 10,000 people in the United States and Europe. Most patients with the disease are over 50 years old.

To date, there are no U.S. Food and Drug Administration (FDA)-approved treatments for cold agglutinin disease, though rituximab-- a treatment for certain blood cancers -- has been used with or without chemotherapy with limited success.

This study included 10 patients 56-76 years old. Patients had cold agglutinin disease for a median of five years, and many had received multiple prior treatments that had been unsuccessful. At enrollment, all patients had below-normal levels of hemoglobin, a component of red blood cells that is responsible for transporting oxygen to cells and organs throughout the body. Six patients were receiving regular blood transfusions to control their symptoms.

Of those patients who responded, within the first week of treatment with a full dose of sutimlimab, destruction of red blood cells stopped and patients' hemoglobin levels significantly increased, said Dr. Jilma. Seven patients responded and had a median increase in hemoglobin levels of 4 grams per deciliter (g/dL) over a baseline of 7.5 g/dL within six weeks. Four patients had their hemoglobin levels return to normal.

When sutimlimab treatment was discontinued and the drug had left the patients' blood, hemoglobin levels dropped and destruction of red blood cells began again. However, when treatment resumed, these effects were once again reversed. The six patients who had been sustained by regular blood transfusions remained transfusion-free for up to 18 months while receiving sutimlimab treatment.

"Provided that safety results remain positive, sutimlimab could become the first approved treatment for cold agglutinin disease," Dr. Jilma said. "The drug clearly addresses an unmet medical need, as we have seen rapid, strong responses in patients for whom multiple prior therapies have failed."

Ulrich Jäger, Shirley D'Sa, Christian Schörgenhofer, Johann Bartko, Ulla Derhaschnig, Christian Sillaber, Petra Jilma-Stohlawetz, Michael Fillitz, Thomas Schenk, Gary Patou, Sandip Panicker, Graham C Parry, James C Gilbert, Bernd Jilma.
Inhibition of complement C1s improves severe hemolytic anemia in cold agglutinin disease: a first-in-human trial.
Blood 2018. doi: 10.1182/blood-2018-06-856930.

Most Popular Now

Aspirin green light for brain bleed stroke patient…

People who suffer a stroke caused by bleeding in the brain - known as brain haemorrhage - can take common medicines without raising their risk of another stroke, a major ...

Cancer cells are quick-change artists adapting to …

Until now, researchers have assumed that the growth of solid tumors originates from cancer stem cells characterized by specific surface markers, which develop in a fixed...

APRINOIA Therapeutics awarded grant from The Micha…

APRINOIA Therapeutics, a clinical-stage biotechnology company with a pipeline of therapeutics and imaging diagnostics for neurodegenerative diseases, announces today that...

Benralizumab not effective reducing exacerbations …

More than 15.3 million people in the U.S. suffer from chronic obstructive pulmonary disease (COPD), which is the third leading cause of death in this country, according t...

Pfizer announces top-line results from Phase 3 Tri…

Pfizer Inc. (NYSE: PFE) announced today that a Phase 3 study to assess the use of LYRICA® (pregabalin) as adjunctive therapy for epilepsy patients 5 to 65 years of age wi...

Novartis phase II data for new inhaled combination…

Novartis announced today that new phase II data for IND/GLY/MF (QVM149), an investigational, once-daily, fixed dose combination asthma treatment containing indacaterol ac...

Cannabis use among older adults rising rapidly

Cannabis use among older adults is growing faster than any other age group but many report barriers to getting medical marijuana, a lack of communication with their docto...

New data show Symbicort reduces attacks in mild as…

New data from Novel START, an open-label trial designed to reflect real-world practice, has demonstrated the effectiveness of Symbicort Turbuhaler (budesonide/formoterol)...

Merck pioneers new effort to see MS from the insid…

Merck, a leading science and technology company, joins the global multiple sclerosis (MS) community in recognition of World MS Day, an initiative created by the Multiple ...

Amgen joins with community oncology networks for n…

Amgen (NASDAQ: AMGN), along with leading community oncology networks, today announced the launch of Amgen Community Oncology Research Collaborators (ACORC), a new initiat...

Novartis Kisqali significantly extends life in wom…

Novartis announced statistically significant overall survival (OS) results for Kisqali in combination with endocrine therapy[1]. The Phase 3 MONALEESA-7 trial evaluated K...

Bayer receives U.S. FDA breakthrough therapy desig…

Bayer announced today that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for copanlisib (Aliqopa™) for the treatment of adult patie...