Eyes on Asia: Astellas bags gene therapy, AZ a lipoprotein, and a Japanese incubator

Astellas options dementia gene therapy from AviadoBio  

Tokyo’s Astellas Pharma signed an option to acquire global rights for neurology gene therapy candidate AVB-101from AviadoBio. AVB-101 is an adeno-associated virus (AAV) based gene therapy in a Phase I/II trial for patients who have frontotemporal dementia with mutations in progranulin (FTD-GRN).  

Astellas will invest $20 million in AviadoBio and pay $30 million in upfront payments to acquire the option. If the option is exercised, AviadoBio is eligible for up to $2.2 billion in license fees and milestones plus royalties. 

After Alzheimer’s disease, frontotemporal dementia (FTD) is the most common cause of early-onset dementia. It is the leading cause of dementia under the age of 65. Several genetic mutations are known to cause family-caused FTD, with mutations in progranulin (GRN) contributing 20–25% of familial FTD cases and about 10% of total FTD cases.  

AVB-101, AviadoBio’s lead drug, aims to restore the progranulin gene GRN, which causes the loss-of-function mutations in FTD-GRN. It delivers the GRN gene by infusion directly into the thalamus, causing GRN protein production. AVB-101 is a single treatment designed to halt disease progression. 

Astellas has five gene therapies in its portfolio, four of them gene replacement candidates. Two candidates have started clinical trials. 

The news comes as Astellas announces it will close a gene therapy manufacturing facility in South San Francisco, moving the work to another US facility in North Carolina. 

AstraZeneca bags CSPC cardio therapy rights 

AstraZeneca has in-licensed YS2302018, a preclinical small molecule lipoprotein (a) (Lp(a)) disruptor from CSPC Pharma – one of China’s largest pharmas . CSPC has been listed on the Hong Kong Stock Exchange since 1994.  

Lp(a) is similar to low-density lipoprotein (LDL), the “bad cholesterol.”  It is a type of fat in the blood that carries cholesterol, causing arterial blockages similar to low-density lipoprotein (LDL). Lp(a) hasn’t been a drug target until recently. CSPC will receive a $100 million upfront payment and could receive up to $1.9 billion in milestones plus tiered royalties. 

AstraZeneca may already be behind the Lp(a) curve. Eli Lilly has been testing a similar candidate that reduced levels of the lipoprotein by up to 65% in a Phase I study. It has also completed a Phase II trial and will report the results before the end of the year. 

YS2302018 is said to have shown that it can prevent Lp(a) from forming blockages. Lp(a) is similar in some ways to low-density lipoprotein (LDL), adding to artery blockages, but it is not the same. Lp(a) is caused by genetics, while LDL is a lifestyle condition. 

YS2302018 will be tested either alone or in combination with AZ’s oral small molecule PCSK9, also a cholesterol therapy that showed efficacy in a Phase I trial. The company has very high hopes for its PCSK9, expecting that the drug will become a $5 billion blockbuster.

Ciconia forms JV to open Japan drug incubator 

Tokyo’s Ciconia Bioventures has emerged as a joint venture incubator focused on everything from early drug discovery research to forming biotech startup companies.  

The company will work with partners Takeda, Astellas, and Sumitomo Mitsui Banking and will identify promising drug discovery seeds from Japanese academia, biotech startups, and pharmaceutical companies.  

The company intends to develop these seeds into high-potential assets with a better chance of success. Ciconia’s well-connected partners will certainly help.  

Ciconia plans to add R&D funding and experienced management teams to launch new innovative startups. The goal of the outreach is to expand the number of biotech ventures emerging from Japan and to deliver the country’s discovery ecosystem to the world. 

Akeso banks $250m for clinical trials 

Akeso Biopharma closed a $250 million share placement to advance its large and diverse portfolio of 16 biologic drug candidates. Approximately 70% of this will be committed to supporting clinical trials in China and the rest of the world. 

The company is conducting trials for 23 indications, including its lead drug, cadonilimab, which is approved in China for two indications (cervical and gastric cancers) and has started four additional trials.  

Earlier this year, the company raised $152 million in a similar sale of shares. Akeso now has over $1 billion on hand, which it will need to keep the lights on for its long list of its clinical trials.  

In late 2022, Guangzhou Akeso outlicensed ex-China rights to ivonescimab, its lead bispecific PD-1/VEGF combination, in a deal that could be worth up to $5 billion. Summit Therapeutics of Menlo Park, CA paid $500 million upfront and will be responsible for up to $4.5 billion in milestones, plus single digit royalties for ivonescimab rights.