Angal Biote Secures Exclusive License for Neurovision's TGFBRII-Targeting ASO Candidate to Treat ALS and Fibrotic Diseases in China

Recently, Anku Biotechnology (Suzhou) Co., Ltd. formally signed a License-in agreement with Germany’s Neurovision Pharma GmbH to exclusively introduce the latter’s independently developed antisense oligonucleotide candidate drug. The agreement grants Anku Biotechnology the rights to develop and commercialize the drug in China (including the Hong Kong Special Administrative Region) for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, and fibrotic diseases.

Antisense oligonucleotides (ASOs) are single-stranded nucleotide chains that bind to complementary target RNA sequences, thereby modulating transcript levels and ultimately the levels of target proteins. ASOs hold particular significance for drug development because, in principle, they can target any mRNA regardless of the structure and function of the encoded protein. Moreover, ASOs exhibit high stability, which can substantially reduce the frequency of therapeutic administration.

In the TGF-β signaling pathway, TGFBRⅡ specifically recognizes and binds to extracellular signaling molecules, coordinating cellular development and differentiation. Aberrations in the TGF-β signaling pathway are often closely associated with the pathogenesis and progression of fibrotic diseases and may contribute to motor neuron loss in amyotrophic lateral sclerosis (ALS). Anku Bio has recently secured the development rights for antisense oligonucleotide (ASO) candidates targeting the TGFBRⅡ receptor. By suppressing TGFBRⅡ expression levels in target organs, this initiative aims to address these urgent unmet clinical needs.

As a “stubborn holdout” among neurodegenerative diseases, the development of therapeutic drugs for amyotrophic lateral sclerosis (ALS) faces significant challenges. To date, only three drugs have received FDA approval: riluzole, edaravone, and Relyvrio (a fixed-dose oral combination of sodium phenylbutyrate and taurursodiol, also known as AMX0035), which was recently approved by Amylyx Pharmaceuticals. Although orphan drugs target only a small patient population, their market size is substantial; currently, they account for 16% of the global prescription drug market, with a value exceeding $70 billion.

China bears the heaviest global burden of liver disease, with more than one-fifth of its population affected by hepatic disorders. Liver fibrosis, triggered by chronic diseases, is characterized by a high incidence rate and irreversibility, posing a severe threat to patients’ lives. As a type of fibrosis highly associated with metabolic disorders, the development of anti-fibrotic drugs for liver fibrosis faces numerous difficulties and challenges. This is primarily due to the complex formation process and mechanisms of liver fibrosis, the multitude of influencing factors, and significant inter-individual variability. Consequently, there are currently no effective chemical drugs or biologics available for clinical treatment.Neurovision Pharma’s antisense oligonucleotide (ASO) candidate has demonstrated its ability to effectively inhibit the TGF-β signaling pathway through both in vivo and in vitro experiments. Furthermore, a 13-week toxicology study involving intrathecal administration in cynomolgus monkeys confirmed its safety and potential as a candidate drug for treating neurodegenerative diseases. In addition, given the broad application prospects of TGF-β signaling pathway inhibitors in the treatment of liver, lung, and kidney fibrosis, Ancure Bio will leverage this acquired ASO drug development project to explore its clinical applications in the substantial domestic market for liver fibrosis therapy.

Neurovision Pharma GmbH, a German intellectual property holding company, was co-founded by two neurology experts and a patent attorney specializing in the drafting, prosecution, and defense of pharmaceutical patents. The company holds exclusive patents for antisense oligonucleotides (ASOs) targeting the TGFBRII receptor in more than 35 countries worldwide. Its portfolio comprises six pairs of ASO candidates that cover all available target sites of TGFBRII, addressing approximately 50 diseases, including indications such as fibrotic diseases, dementia, Alzheimer’s disease, Parkinson’s syndrome, amyotrophic lateral sclerosis (ALS), and central nervous system aging.

Anku Bio, a novel drug R&D company established in Suzhou through joint investment by Europe’s JLP Health, the Jiangsu Industrial Technology Research Institute, and Suzhou New District Hi-Tech Industrial Co., Ltd., is driven by world-class technologies for precise analysis of drug targets and proprietary drug screening platforms. The company primarily focuses on the early-stage development of innovative therapeutics in oncology, antiviral treatments, and medicines for metabolic and neurodegenerative diseases. Anku Bio has exclusively licensed ASO candidate drugs targeting TGFBR II from Germany’s Neurovision Pharma, aiming to strengthen its pipeline in metabolic and neurodegenerative disease treatments, develop novel therapies for ALS and fibrotic diseases, address significant unmet clinical needs in China, and alleviate patient suffering.