A patient with an autoimmune disease has died after receiving an experimental genetic medicine from a biotech startup in China, Endpoints’ Ryan Cross reported exclusively this week. The startup, called RiboX Therapeutics, is working on an in vivo CAR-T therapy based on a second generation of mRNA medicines known as circular RNA. RiboX said the patient, a man in his 50s, had “severe systemic sclerosis and significant pre-existing medical issues.” The company said it “promptly reported” the death in China to “the relevant ethics and regulatory authorities” and also reported it to the FDA. But it didn’t publicly disclose the death until questioned by Endpoints.

The fatality is the third uncovered in recent weeks in China’s investigator-initiated trials. IITs have become a popular way for researchers to quickly and cheaply test experimental drugs in China without oversight from the country’s national drug regulators. But recent deaths have put the trials under increased scrutiny. In May, the Chinese government instituted new rules that are supposed to narrow the use of IITs. But there is still no requirement to publicly report deaths from IITs, several Chinese biotech executives told Endpoints.

The deaths have become a flashpoint for several ongoing debates, including the transparency and trustworthiness of drug development in China and the wisdom of the country’s lenient IIT system, Ryan wrote. On Thursday, two Republican lawmakers asked the FDA to reject clinical data from China unless a trial site has been recently audited by the agency, and to institute a sweeping review of existing products’ reliance on Chinese clinical results.

The latest death is likely to raise questions not only about China IITs, but also about the two technologies RiboX is working on: in vivo CAR-T and circular RNA. RiboX would not provide additional information on the patient or their cause of death. An unnamed source said RiboX was in talks with multiple global pharma companies who were interested in buying the program, and the death put a damper on those talks. Meanwhile, RiboX recently received a green light from the FDA to begin a clinical trial of the treatment in the US, which the company announced on Aug. 8. Ryan has the full story here.

📈Is the next generation of immunotherapy here? Merck and Moderna certainly believe so, as the pair declared the first Phase 3 success for their personalized cancer vaccine in patients with certain stages of melanoma. Patients received the vaccine, called intismeran autogene, combined with Keytruda. The study hit its primary endpoint, reducing the risk of recurrence or death compared to Keytruda alone by a statistically significant margin. Specific data will be disclosed at a future medical conference, but the win marks a number of important firsts in cancer immunotherapy. Read more about the groundbreaking study here. 

For Merck, the trial success gives it another arrow in its quiver as it attempts to stave off Keytruda’s patent cliff, expected in 2028. It’s been a longstanding goal for Merck CEO Rob Davis to turn the Keytruda cliff into more of a “shallow dip,” and other recent developments have brought that closer to fruition.

Moderna, meanwhile, saw its stock surge 177% in Wednesday’s trading as the biotech is attempting to regain its footing after slashing costs once the Covid-19 pandemic let up. The result eases investor skepticism and also opens the door for Moderna’s broader ambitions in oncology and other areas. 

The companies did not give a timeline for when they might file for FDA approval. Trials are continuing in lung cancer, bladder cancer and kidney cancer, where they believe the treatment could prove beneficial.

President Donald Trump selected Heidi Overton to be the next FDA commissioner. Overton has been a top White House aide as the deputy director of the Domestic Policy Council following five years at the America First Policy Institute, a Trump-aligned DC think tank. Overton would replace Kyle Diamantas, who has served as acting FDA commissioner the last three months after Marty Makary resigned under pressure in May.

Who is Heidi Overton? Senior reporter Max Bayer took a closer look at Overton’s past work this week. She has both a medical degree and PhD, but no direct regulatory experience. Over the years, she’s advocated for increased hospital transparency, changes to how doctors prescribe opioids, and more oversight of the abortion pill. Overton also stood by Trump earlier in August as he announced a sweeping and contentious new executive order calling for amendments to the childhood vaccine schedule. 

The industry’s reaction so far has been mixed. Chris Meekins, a former Trump health official in his first term and now a Washington healthcare analyst for Raymond James, wrote to clients that she would be “like Makary, but probably worse.” Evan Seigerman of BMO Capital Markets, meanwhile, was a bit more tempered, calling her selection “a modest positive” because it removes the overhang of waiting on a new chief. Read more here. 

🤝Reverse mergers have become more popular recently, and industry experts expect the trend to continue. Avere Therapeutics collected $820 in investor commitments this month to take its China-sourced immunology drug onto the public markets via a reverse merger, surpassing Parabilis’ record-breaking IPO. And three more companies recently chose this avenue: Redx Pharma, Slate Medicines and Ambros Therapeutics.

It’s been the most active year since 2018, according to Raymond James bankers. That popularity is bred by their own success, the number of available shell companies, and the inherent advantages of the approach, such as timing and investor certainty, Kyle LaHucik wrote. As the Raymond James bankers put it: "The market is becoming less focused on how a company gets public and more focused on the quality of the company that arrives there.” Kyle breaks down the trend and its advantages here.