Anima Biotech has partnered with Takeda Pharmaceuticals to develop small molecules that control mRNA translation — the process by which the information encoded in a person’s DNA is “translated” into a protein — as potential therapies for neurological disorders with a genetic cause, including Huntington’s disease.
Huntington’s is caused by an expansion of three nucleotides — the building blocks of DNA, in this case the nucleotides CAG — in the huntingtin (HTT) gene. While healthy people normally have CAG repeated 10 to 35 times in their genes, Huntington’s patients may have up to 120 repeats or more. This expansion leads to the formation of a mutated form of the protein.
DNA is first transformed into messenger RNA (mRNA), which is then converted into a protein in a process called translation. Increasing evidence suggests that malfunctions during the translation process are an underlying cause of many repeat-associated diseases, such as Huntington’s.
Accordingly, developing small molecules that correct these translation defects may lead to new treatments.
Anima is pioneering the development of small molecules that selectively control mRNA translation, either by accelerating or slowing it. That, in turn, will result in an increase or decrease of the corresponding protein. The company’s proprietary pipeline includes programs in fibrosis, cancer, and infectious and repeat-associated diseases.
“Anima Biotech’s expertise in understanding the complex regulatory mechanisms of protein expression in the central nervous system [brain and spinal cord] makes them an ideal partner to explore this novel area of drug discovery,” Ceri Davies, PhD, head of the neuroscience drug discovery unit at Takeda, said in a press release.
“By combining Anima’s platform with our strength in translational medicine and clinical development, we aim to develop medicines that deliver greater benefits to patients with genetically-defined neurological diseases where there are non-existent or ineffective treatment options,” Davies said.
In the new collaboration, Anima will use its Translation Control Therapeutics platform to develop small molecules targeting the mutated HTT. Two additional targets involved in neurological diseases, and not yet disclosed, will be selected by Takeda. Under the agreement, Takeda will retain the exclusive rights to develop and commercialize the small molecules.
Takeda will make an upfront payment of approximately $120 million to Anima, which includes preclinical research milestone payments. Additionally, Anima will be eligible for up to $1.1 billion in clinical and commercial milestones, depending on the success of the three developed molecules, as well as tiered royalties on net sales.
Takeda has a defined time window to decide whether to expand the collaboration to include up to three additional targets. This is subject to additional payments to Anima of up to $1.2 billion and tiered royalties, once again dependent on the therapy’s success.
“Partnering is a core strategy of Anima and our model is to build collaborations that maximize the probability of success by combining our discovery platform and expertise in translation control biology with the scientific, clinical and commercial capabilities of our partners,” said Yochi Slonim, co-founder and CEO of Anima.
“Takeda’s leadership and depth of scientific expertise in the CNS area is ideally suited for our second Pharma partnership in Neuroscience and further validates our leadership in mRNA translation control,” Slonim added. “We are looking forward to working together with Takeda in a true collaboration model.”
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