UniQure is looking for a path forward with its experimental gene therapy for Huntington’s disease. The company said the FDA has “strongly recommended” conducting a randomized and placebo-controlled study before submitting AMT-130 for approval. But in an earnings call Monday, company executives said a sham-controlled study may be difficult to enroll, if not outright impossible. “Some might even consider this trial design to be unethical,” chief medical officer Walid Abi-Saab said.

UniQure’s stock got caught up in the chaos, plunging in the days after FDA Commissioner Marty Makary appeared to criticize uniQure’s Huntington’s program in a CNBC interview, despite not mentioning it by name. Analysts on uniQure’s earnings call asked for more information about how the FDA wants the placebo-controlled study to be conducted, but uniQure didn’t provide a clear answer. The company said it will need to follow up with the FDA in additional discussions, and executives are shooting for a second-quarter meeting.

Under Makary, the agency has trumpeted efforts to accelerate rare disease drug approvals. In last week’s CNBC interview, Makary said he brings a “sense of urgency” for addressing rare diseases to his role at the agency. Following uniQure’s announcement, some were skeptical. “Whatever one thinks about the [uniQure] data package for AMT-130, broadly speaking it continues to seem like this FDA is not serious about exerting flexibility and accelerating treatments in rare disease,” Stifel analyst Paul Matteis wrote in a note to investors on Monday. 

After a steady stream of GLP-1 data last week, the news kept flowing this week when Sciwind Biosciences got clearance from Chinese regulators for its GLP-1 injectable for obesity. Pfizer will commercialize the drug in that country following a deal last month.

But the obesity attention this week was more focused on the amylin front. The mechanism, which some believe should lead to better quality weight loss than the megablockbuster GLP-1s, has caught the attention of many big pharma and biotech investors over the past few years.

And it was one of those pharma deals — Roche’s up to $5.3 billion bet on Zealand Pharma’s petrelintide — that caught the industry by storm last year. Now, the Danish biotech’s stock is being wiped by investor displeasure in Phase 2 data that dropped Thursday evening.

In a global race to develop cheaper and potentially better alternatives to the world’s 10 most expensive medicines, China has pulled ahead, Endpoints’ Jared Whitlock wrote this week. Last April, Chinese regulators approved Belief BioMed’s gene therapy for the bleeding disorder hemophilia B. It’s the first hemophilia gene therapy in China to be developed and manufactured entirely by a domestic company. Belief priced the treatment at $350,000, one-tenth the cost of a rival therapy that sells for $3.5 million in the US.

The company plans to dose its first commercial hemophilia B patient this month, and it’s working to secure approval in the US and additional countries. It’s also conducting clinical trials for gene therapies that would compete with two top-priced therapies for hemophilia A and the rare disorder Duchenne muscular dystrophy. 

Belief is joined by other Chinese companies that are chasing after the world’s most expensive drugs. An Endpoints News analysis of clinical trial databases shows that China accounts for 48 of the 77 programs targeting a group of ultra-expensive gene therapies. Even if only a fraction of these lower-cost alternatives reach the market, it could upend the economics of a gene therapy system built on multimillion-dollar prices, Jared wrote.

One company’s mission to create individualized therapies has ended for now. EveryOne Medicines, which was a pioneering biotech in the fight to develop custom medicines for ultra-rare genetic diseases, is closing its doors, Ryan Cross and Jared Whitlock exclusively reported.

EveryOne made the decision shortly after US health regulators outlined long-awaited guidance on the path for individualized therapies to gain FDA approval. A person familiar with the matter told Endpoints that the draft policy didn’t go as far as what EveryOne had hoped. Commercialization in the US wouldn’t be as feasible as initially anticipated.

Despite the setback, EveryOne CEO Julia Vitarello said there’s still been plenty of progress in the past year. “Where we are today is a significantly better place,” she said in an interview. But “changing the system is hard. It is not linear,” she added.

The San Diego and Shanghai biotech, whose CEO has set a goal of proving whether T cell engagers can wipe out autoimmune diseases, will go public via a reverse merger. Rather than taking the traditional IPO path, which has gained some traction so far this year, Candid is going with an alternative route that sees an up-and-coming private biotech take the place of a struggling Nasdaq-listed company.