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Cancer is well represented in the Astellas Pharma portfolio and pipeline, but the drugmaker has made diversification a key part of its growth strategy. Newer products in its drug lineup include a vision-loss disorder drug and a first-in-class therapy for menopause symptoms. Cell and gene therapies are also a key part of Astellas’s growth strategy, building off of its 2020 acquisition of gene therapy developer Audentes Therapeutics. Within gene therapy, the Tokyo-based company is focused neuromuscular disorders, Chief Medical Officer Tadaaki Taniguci said in an interview last week during the annual J.P Morgan Healthcare Conference in San Francisco. “Our strategy in the past is more developing our capability,” Taniguci said. “We actually acquired Audentes, developing our capabilities toward gene therapy. But now we see that the more important thing for us is to really bring in clinical-phase assets to our pipeline.” Exclusive Improving the Healthcare Financial Experience to Help Care Flow Zelis CEO Amanda Eisel shares her perspective on how the company is solving the problems of a fragmented health financial system to benefit all. By Stephanie Baum The Astellas pipeline is split nearly 50/50 between internally developed drug candidates and assets from acquisitions or external collaborations. Audentes brought AT132, a gene therapy for the rare neuromuscular disease X-linked myotubular myopathy. This program has been beset by setbacks due to patient deaths in the trial. But Audentes also brought programs for two rare other rare disorders, Pompe disease, an inherited disorder that leads to muscle weakness, as well as Friedreich’s ataxia, which causes cardiomyopathy. Both programs are in early clinical development. Astellas is still expanding its gene therapy prospects through deals. Last October, the company paid AviadoBio $50 million for the option to license the biotech’s lead gene therapy, which is in early clinical development for frontotemporal dementia, a neurodegenerative disorder with no FDA-approved treatments. The deal structure is similar to a 2022 agreement that gave Astellas the option to license a Taysha Gene Therapies program for Rett syndrome, a disorder that leads to developmental problems. Phase 1/2 safety and efficacy data for this Rett therapy, TSHA-102, are expected in the first half of this year. Astellas remains willing to strike deals that give it new tools that expand its capabilities in gene therapy, Taniguci said. The company is already developing gene therapies delivered to their bodily destinations carried aboard adeno-associated viruses (AAV). These engineered viruses preferentially go to the liver. In 2021, Astellas began a research collaboration with Dyno Therapeutics focused on discovering novel capsids — the protein shells that envelop a genetic payload — for delivery to skeletal and cardiac muscle. Last month, Astellas began a partnership with Sangamo Therapeutics, securing the right to use one of that biotech’s proprietary capsids to penetrate the brain and reach neuronal targets, which Taniguci said fits with his company’s neuromuscular disease strategy. Penetrating the blood-brain barrier “is the one biggest challenge to overcome to target the [central nervous system],” he said. “So they help us to create much better access to the target organ. We see more platform development together with them to actually create the next generation of gene therapy.” Sponsored Post The Funding Model for Cancer Innovation is Broken — We Can Fix It Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches. By Eve McDavid - CEO of Mission-Driven Tech In oncology, the company’s top products are Xtandi, a small molecule drug for prostate cancer, and Padcev, an antibody drug conjugate for bladder cancer. A Phase 3 study is evaluating Padcev in combination with the Merck immunotherapy Keytruda as a treatment for muscle-invasive bladder cancer. Data are expected in Astellas’s next fiscal year, which starts on April 1. Additional clinical studies are testing Padcev in non-muscle-invasive bladder cancer and other solid tumors. Astellas is pursuing other approaches to cancer. Its in-house R&D has yielded ASP3082, the company’s lead targeted protein degrader drug for cancer. This drug candidate targets the cancer-driving protein KRAS G12D. At the European Society of Medical Oncology (ESMO) meeting last September, Astellas reported preliminary Phase 1 data showing anti-tumor activity in patients with pancreatic, colorectal, and non-small cell lung cancers. Taniguci noted the results indicate a dose-dependent degradation of the target protein. Astellas will need to show differentiation from other companies developing KRAS G12D-targeting drugs, such as Revolution Medicines and Bristol Myers Squibb, via the pharma giant’s acquisition of Mirati Therapeutics. But Astellas envisions potentially bringing its targeted protein degradation approach to multiple KRAS mutations. “This is a new technology that we started using for KRAS G12D, but we also have a pan-KRAS product coming to the clinic pretty soon,” Taniguci said. “We also started expanding more to other targets. We cannot disclose yet, but [there’s] a lot of excitement.” Astellas’s newest cancer drug is Vyloy, which in October became the first FDA-approved drug targeting claudin 18.2, a protein highly expressed in gastrointestinal cancers. This internally developed drug is a monoclonal antibody. The Astellas pipeline includes another program targeting claudin 18.2, but with a bispecific antibody. This program, ASP2138, is in Phase 1 testing. Women’s health is a newer piece of Astellas’s portfolio coming with the 2023 FDA approval of Veozah, a non-hormonal drug for treating vasomotor symptoms caused by menopause. The first-in-class therapy is a small molecule designed to block neurokinin 3, a receptor that plays a role in regulating body temperature. Last month, the FDA added a black box warning on the product’s label, flagging the risk of severe liver injury. Taniguci said liver toxicity is a known risk that was first observed in clinical trials. None of those cases were severe. In the market, the product has been used by about 100,000 patients. “Liver tox is relatively rare, but sometimes we see the severe cases,” Taniguci said. “That’s why I think it’s important to provide cautions for the patient and physician to use this important medicine. But of course we believe that the benefit/risk balance is really positive. So that’s why we still hear a lot of patients willing to use Veozah in treatment of [vasomotor symptoms].”

Bayer is preparing for two potential blockbuster launches in 2025. Although Bayer’s top-selling drug Xarelto lost market exclusivity in most geographies in 2024, the company still predicts it will see strong growth from the pharma business unit beginning in 2027.The optimism is rooted in new launches, Stefan Oelrich, head of Bayer’s pharma division, said Tuesday at the J.P. Morgan Healthcare Conference.Besides label expansions for in-market products such as prostate cancer drug Nubeqa and kidney disease therapy Kerendia, the German pharma is prepping for the potential rollouts of two key drugs: BridgeBio-partnered transthyretin amyloid cardiomyopathy (ATTR-CM) therapy acoramidis and menopause candidate elinzanetant.These launches are what Oelrich called “must-win battles” at Bayer as the Xarelto loss of exclusivity reaches “full swing” in 2025. In the third quarter of 2024, Xarelto already saw its sales drop about 24% to 802 million euros in what Oelrich called “a harbinger” for what to expect of the blood thinner in the following quarters.Bayer in-licensed European rights to acoramidis in April 2024 and secured a positive opinion from the European Medicines Agency in December.In the U.S., following an FDA approval in November and an official launch Dec. 11, BridgeBio this week revealed that it has recorded 430 Attruby (acoramidis) prescriptions for patients with the rare disease.BridgeBio’s early launch progress, which analysts viewed as a strong start, gives Bayer confidence in its own efforts.“It’s certainly a fast-growing, billion-plus type of market opportunity that we will address right where the sweet spot for us is,” Oelrich said. Bayer will leverage its existing commercial infrastructure to support the drug’s launch, which is initially planned for Germany and Switzerland, he said.With elinzanetant, Bayer is handling the drug’s commercialization globally, including the U.S. If approved—the FDA has set a target decision date for July—the drug would go up against Astellas’ Veozah.But, just a few months after an FDA nod in 2023, Astellas slashed its Veozah peak sales projection in half to between 150 billion Japanese yen ($1 billion) and 250 billion yen ($1.6 billion). The Japanese pharma cited insurance coverage as a major challenge to uptake for now, plus a lower-than-expected treatment rate in the overall vasomotor symptoms market. These market-wide insights have led to questions around elinzanetant’s potential.“We see this as a blockbuster candidate,” Oelrich said of elinzanetant. “And, quite frankly, otherwise we shouldn't be launching.”“But I think I mentioned it last year already: This is not a walk in the park, because there is a clear understanding by physicians that [hormone replacement therapy] is an effective treatment,” Oelrich continued. “So we need to find the right place where this is acceptable, both to women, [who] I think are more open to nonhormonals, but also to physicians.” Oelrich argued elinzanetant’s mechanism of action could help it differentiate itself. While Veozah is a neurokinin 3 (NK3) receptor antagonist, elinzanetant is a NK-1 and -3 receptor blocker. Besides, while women’s health is a new area for Astellas, it’s not for Bayer, which has a contraceptive portfolio, including the Mirena device family.“I have personally been part of a launch of HRT,” Oelrich said. “Because we have been a leader in the segment for over 100 years and have been also a leader for many, many years in the women’s health care space in the United States, we’re looking at this, eyes wide open.”