Astellas’ Okamura: The bottleneck isn’t money — it’s translation

CEO Naoki Okamura on Japan’s biotech sector, cell and gene therapy, and Astellas’ playbook for menopause, China and Xtandi’s patent cliff

Why does great science so often stall before it becomes a medicine? Astellas Pharma CEO Naoki Okamura argues the bottleneck isn’t capital — it’s translation: a shortage of people, processes and platforms that can move ideas across the boundaries of biology, manufacturing, regulation and commercialization.

That theme threaded through his conversation on The BioCentury Show, surfacing most clearly in his diagnosis of what Japan’s biotech ecosystem lacks — and in his rationale for why Astellas Pharma Inc. (Tokyo:4503) is doubling down on cell and gene therapy as other multinational pharmas pull back.

The discussion also touches on Astellas’ journey in commercializing a women’s health product, the company’s China strategy, and its plans for life after Xtandi’s patent cliff.  

Toward a thriving biotech ecosystem in Japan

Astellas’ home base in Japan gives Okamura a close view of a familiar gap: strong academic science, but a start-up sector that has struggled to consistently turn that science into competitive products.

While VCs have recently taken notice of new government incentives designed to bolster investment in Japanese start-ups, Okamura argued that money isn’t the key issue holding back biotech.

“There is a challenge in Japan about talent fluently moving around the different sectors: academia, industry, regulatory space — that exactly is the biggest hurdle,” argued Okamura. 

The path from discovery to product is blocked by silos, he said, and by a shortage of people who can carry a program across them. “It is not about money. Money can follow good science, good assets.” Rather, “it is all about” whether that science can “find the path to become a real pharmaceutical product.” The implication: Incentives may help at the margin, but they don’t substitute for the practical development expertise — regulatory, clinical, manufacturing and commercial — that turns a promising mechanism into a viable medicine.

Astellas, he said, is trying to make that know-how more accessible, supporting start-ups not just with capital but with operating expertise through joint ventures and academic collaborations. He also pushed back on the notion that Japan’s opportunity is synonymous with cell and gene therapy. The idea that Japan is the place for those technologies is “a little bit overemphasized,” he said, noting the country has “many different sciences,” and what matters is building the translational capacity that makes those opportunities more visible — first to Japanese venture capital, and then to investors “anywhere in the world.”

Astellas stays the course in cell and gene therapy

A similar translation lens shapes Astellas’ approach to cell and gene therapy — a field where the biology is often compelling, but the gap between concept and scalable medicine is especially wide. As other multinationals retrench after setbacks in safety, delivery and commercialization, Okamura framed Astellas’ commitment as a bet that those barriers are solvable if companies build the right enabling platforms. Those who do, he said, will have an advantage in diseases where cell replacement or gene delivery may be the only path to meaningful benefit.

Acknowledging the “limitations” of AAV vectors, Okamura said the company is working systematically to reduce risk and expand where these therapies can be used. Astellas is trying to lower effective dose by improving the ratio of empty to full capsids, and by designing capsids that home more precisely to the intended organ to minimize off-target toxicity. It is also using cell-selective promoters to restrict gene expression to the right cells. None of these, he suggested, is a final fix; progress will come through iteration. “There is no perfection,” he said. “There’s no completion of those innovations.”

In the near term, Okamura described the eye as “a good entry point” for these therapies, reflecting the advantages of localized delivery, such as safety. He noted that while Astellas’ flagship cell therapy program targets the eye, its flagship gene therapy program, AT845 for Pompe disease, is a systemically delivered AAV8 program designed to drive GAA expression primarily in skeletal and cardiac muscle using muscle-restricted promoter elements.

Women’s health, China and life after Xtandi

Okamura also discussed three other priorities for Astellas: building demand for a women’s health launch, staying close to China’s innovation engine, and navigating loss of exclusivity for Xtandi enzalutamide without resorting to expensive stopgap deals.

Okamura said Astellas learned an unexpected lesson launching Veozah fezolinetant: in menopause, demand generation can’t run ahead of the medical system. The company initially thought patient awareness would drive rapid uptake of a non-hormonal therapy, but he said adoption required groundwork with physicians and payers first, convincing prescribers it differs from decades of hormone-based practice and securing coverage. Otherwise, patients show up asking about a drug their doctors don’t yet understand — an “embarrassing situation” that frustrates both sides. Astellas is doing direct-to-consumer outreach, he said, but selectively and iteratively, using early feedback to refine targeting while pacing demand to prescriber readiness.

On China, Okamura described a role reversal: it used to be that Chinese companies helped multinationals move programs faster, but “now they become the source of innovation for us.” He called China an “epicenter for innovation” and pointed to Astellas’ new R&D center in Beijing and the company’s in-licensing of a CLDN18.2-targeting ADC from Evopoint Biosciences Co. Ltd. (信诺维) as evidence of a strategy to embed in that ecosystem while reinforcing oncology franchises where Astellas already has a footprint.

Xtandi’s loss of exclusivity is the next big test for Astellas. Okamura called the prostate cancer franchise a “privilege,” noting it has served more than 1.3 million men globally, but said the company has prepared by building a portfolio rather than chasing a single replacement. Instead of patching near-term revenue with large “rescue” transactions, he emphasized five strategic brands with blockbuster potential, alongside several coming catalysts. The reason, he said, is that a rescue deal is “simply a cash flow type of transaction” that tends to be expensive, with “very limited room for us to add value to the asset.”