Astellas Pharma, Inc.

Leon Wang, AstraZeneca’s Executive Vice President International and President of AstraZeneca China, is currently under investigation by Chinese authorities, the drugmaker confirmed today. The company has stated that Wang is fully cooperating, though details about the nature of the investigation remain undisclosed. AstraZeneca’s operations in China will continue as usual under the leadership of its General Manager. The announcement adds another layer to mounting scrutiny faced by AstraZeneca’s China operations. Last month, Chinese authorities detained five current and former AstraZeneca employees in the oncology division as part of an ongoing investigation into alleged illegal activities, as reported by Bloomberg. The employees, all Chinese nationals, were reportedly detained in Shenzhen earlier this summer over two suspected violations: potential breaches of China’s stringent data privacy laws through misuse of patient data and involvement in importing an unapproved liver cancer drug into China. AstraZeneca confirmed that a small number of its employees are under investigation but withheld further details, and Chinese authorities have not commented on the case. AstraZeneca’s challenges with Chinese regulatory bodies are not unprecedented. In 2022, the company was accused of tampering with gene-testing data, allegedly to make certain treatments eligible for government health insurance, adding to AstraZeneca’s already complex regulatory landscape in China. This latest probe comes amid a broader crackdown on data security and compliance in foreign companies operating in China. These investigations highlight a growing regulatory risk for multinational companies in China. Recently, a Japanese executive from Astellas Pharma faced similar challenges when he was detained in China on espionage charges, sparking concern among foreign firms navigating the country's newly intensified data and anti-spying regulations.

AMD is a significant cause of central vision loss in the elderly, often affecting both eyes. Credit: GBJSTOCK/Shutterstock. Astellas Pharma has announced the withdrawal of its marketing authorisation application for avacincaptad pegol intravitreal solution (ACP) to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD) from the European Medicines Agency (EMA). The withdrawal follows discussions with the EMA’s Committee for Medicinal Products for Human Use (CHMP). AMD is a significant cause of central vision loss in the elderly, often affecting both eyes. ACP, an investigational synthetic aptamer, inhibits the complement C5 protein. The overactivity of the complement system and C5 protein contributes to the scarring and vision loss associated with GA secondary to AMD. See Also: Pharma companies must adapt to keep pace with AI developments, say experts AF stroke patients could benefit from earlier anticoagulant administration By acting on C5, ACP reduces the activity of the complement system, which is implicated in the degeneration of retinal cells, potentially slowing the progression of GA. Astellas ophthalmology development vice-president and head Marci English stated: “We would like to emphasise our confidence in ACP’s clinical profile as demonstrated in two randomised sham-controlled trials and its potential to benefit people living with geographic atrophy (GA). “While we are disappointed with the CHMP’s response, we have seen the impact this medicine has had for GA patients in the US and remain committed to serving unmet patient needs globally.” Astellas is examining the possible financial effects of this issue for the fiscal year ending 31 March 2025. Despite the withdrawal, Astellas maintains a positive stance on ACP. The company continues to have confidence in ACP’s clinical profile and its potential to slow the disease. There are no currently approved treatments for GA secondary to AMD outside the US, where ACP was approved under the name IZERVAY in August 2023. Astellas will work with regulatory authorities to explore all available options to make ACP accessible to patients with GA globally, including those in Europe. Astellas recently received US Food and Drug Administration approval for VYLOY (zolbetuximab-clzb), in conjunction with chemotherapy, for the treatment of advanced gastric and gastroesophageal junction cancer.

The eye drop maker Ocuphire Pharma and startup Opus Genetics are merging in an all-stock deal, the companies told Endpoints News . Ocuphire $OCUP will acquire privately-held Opus and adopt its name, and will trade under the new stock ticker $IRD effective Thursday, reflecting its focus on inherited retinal diseases. The combined company will have a late-stage eye drop and a pipeline of seven AAV-based gene therapies. Ocuphire CEO George Magrath will remain in his post and Opus CEO Ben Yerxa will become president. Magrath said that at a recent conference, a speaker from the FDA “mentioned that the biggest unmet need in ophthalmology right now [is] treatments for inherited retinal disorders, so that was music to my ears.” Ocuphire shareholders will own about 58% of the combined company and Opus’ shareholders will get about 42%, according to the announcement. The combined company will have about 20 employees. Michigan-based Ocuphire went public through a reverse merger with Rexahn Pharmaceuticals in the fall of 2020. North Carolina-based Opus came out of stealth three years ago, backed with $19 million in seed funding. The company’s science is based on work from Jean Bennett, who co-founded Spark Therapeutics and co-developed its treatment Luxturna. Bennett will become a board member of the combined company. After its founding, Opus bought two preclinical gene therapies from Iveric Bio for $500,000 upfront in December 2022. Opus has raised a total of about $26 million, Yerxa told Endpoints Tuesday. Opus’ lead gene therapy is OPGx-LCA5, which is in Phase 1/2 for an early-onset retinal degeneration condition known as Leber congenital amaurosis 5. On Tuesday, the companies said three of three adults in the trial had visual improvement at six months. “We took a gamble to use our little bit of available capital to get that early clinical data,” Yerxa said. “That was really key to get to that proof-of-concept to show that this idea of curated gene therapies, hand-selected, that they would read out in a meaningful way.” The next step entails testing the therapy in three pediatric patients, and data could come in the third quarter of next year. Magrath said that if those data show similar improvement, “the door is open to discuss with the FDA what the accelerated approval would look like.” Ocuphire has experience with regulatory approval from its eye drop, which was cleared by the FDA last year for pharmacologically-induced mydriasis. The drug has been out-licensed to Viatris, which is also fully funding two Phase 3 tests of the phentolamine ophthalmic solution in dim light vision disturbances and presbyopia, a common condition that causes blurry vision. Those studies are slated to have data readouts in the first quarter and first half of 2025, respectively. Ocuphire is running the clinical development and is responsible for regulatory filings before handing off to Viatris to lead commercialization, Magrath said. The presbyopia market has had its challenges. AbbVie pulled back on promoting a product for the disease and cut a next-generation drug, while the company Orasis hasn’t yet started selling its approved drug. On Monday, Lenz Therapeutics said the FDA set an approval deadline of Aug. 8 for its eye drop LNZ100. After the merger, Ocuphire will stop working on the Ref-1 inhibitor APX3330, an oral small molecule that had failed a Phase 2 trial in diabetic retinopathy last year. It plans to look for a partner for the drug, as well as for its follow-on Ref-1 inhibitors APX2009 and APX2014 for geographic atrophy and other retina conditions, Magrath said.