Companies partner to develop oral treatments for SCD, other diseases
Immvention, Novo Nordisk cite 'pressing need' for effective therapies
Immvention Therapeutix has partnered with Novo Nordisk to develop oral treatments for people with sickle cell disease (SCD) and other chronic conditions.
The U.S.-based Immvention called the deal “a strategic collaboration and license agreement” in a company press release.
“We are excited to partner with Novo Nordisk,” said Anil Goyal, PhD, CEO and cofounder of Immvention. “This collaboration represents a significant milestone for Immvention and reinforces our vision to address unmet needs in sickle cell disease and beyond.”
In SCD, genetic mutations lead to the production of an abnormal version of hemoglobin, which is the protein red blood cells use to carry oxygen through the body. The defective protein tends to form clumps inside red blood cells that cause them to adopt the sickle-like shape that gives the disease its name.
Symptoms of SCD include anemia, fatigue, swelling of the extremities, recurrent infections, and vision problems. Sickled red blood cells can also block blood flow, especially in small blood vessels, leading to painful vaso-occlusive crises and other serious complications.
Stem cell transplants are available, and have the potential to cure SCD. But such treatment carries significant risks and is not suitable for all patients. Other therapy options include pain management with painkillers, antibiotics to prevent infections, and therapies aimed at reducing the frequency of pain crises. In the last few years, gene therapies such as Casgevy (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel) have been introduced as new treatment options.
But “despite the current treatments and recent gene therapies, we believe that there remains a pressing need for globally accessible, effective, and convenient oral therapies for people with SCD,” Goyal said.
Treatments for SCD will target BACH1 protein regulator
The new partnership will leverage Immvention’s orally available small molecules, which are designed to block BACH1, a key protein regulator of cellular responses, inflammation, and oxidative stress — all of which drive multiple diseases. Oxidative stress is a type of cell damage caused by reactive oxygen-containing molecules.
According to Immvention, BACH1 inhibitors have the potential to boost the production of fetal hemoglobin — a form of hemoglobin produced during fetal development that transports oxygen more efficiently than its adult counterpart. After birth, fetal hemoglobin is gradually replaced by adult hemoglobin, which is defective in people with SCD. Enhancing fetal hemoglobin production is a therapeutic approach being explored to treat the genetic disease.
BACH1 is believed to be a biologically relevant target in sickle cell disease. … We are excited to partner … to advance this program and explore a promising therapeutic option for people living with sickle cell disease.
According to Jaya Goyal, corporate vice president of rare disease research at Novo Nordisk, “BACH1 is believed to be a biologically relevant target in sickle cell disease.”
“Immvention has identified investigational BACH1 inhibitors and generated relevant preclinical data,” Jaya Goyal said. “We are excited to partner with Immvention to advance this program and explore a promising therapeutic option for people living with sickle cell disease.”
Under the terms of the agreement, Novo Nordisk will gain exclusive worldwide access to Immvention’s BACH1 program, and will work together with its new partner to advance BACH1 inhibitors. Upon or before identifying a potential candidate for further development, Novo Nordisk will then take over all activities related to clinical development, regulatory submissions, and commercialization worldwide, according to Immvention.
Anil Goyal said Novo Nordisk brings its “expertise and commitment” to the deal.
Together, the two companies “are poised to advance our novel BACH1 inhibitors and hope to bring meaningful treatment options to patients,” Anil Goyal said.
Meanwhile, Immvention has retained the rights to develop certain BACH1 inhibitors that can pass the blood-brain barrier, a protective mechanism designed to block harmful substances from reaching the brain, but which also stops treatments. These compounds can potentially treat diseases such as Parkinson’s and Alzheimer’s, in which BACH1 dysregulation is believed to play a role.
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