Commission calls for major investments to counter China's biotech rise

A federal commission is calling for $15 billion in new financing to reinforce U.S. biotech leadership amid encroaching competition from China.

The recommendation came as part of a sweeping report released today by the National Security Commission on Emerging Biotechnology, which was created by Congress 2022 to examine China’s influence in the industry.

The report also calls for $1 billion to create an investment fund, more than $6 billion for large-scale research challenges, and a build-up of biomanufacturing sites across the country.

Read more from STAT's Allison DeAngelis.

biotech

Cartesian's CAR-T may help autoimmune patients long term

From my colleague Allison DeAngelis: Over the last few years, the drug industry has rushed to retool CAR-T treatments for cancer patients into vanguard therapies for people with autoimmune diseases. But so far, there’s been scant data on just how long the treatment might help patients.

Well, Cartesian Therapeutics has jumped into the conversation, presenting new data from a Phase 2 trial of its CAR-T therapy for the autoimmune disorder myasthenia gravis, which causes muscle weakness. Cartesian reported last July that the trial had succeeded in meeting its goals, and today, followed up with data from a sub-group of 12 patients who received the treatment a year ago. Ten of the patients maintained a clinically meaningful response to treatment. Four of the trial subjects had few or no symptoms.

The best responses were seen in patients who hadn’t previously taken any biologic therapies like FcRn inhibitors.

Cartesian plans to start a Phase 3 trial in the next couple of months.

regulation

Bristol CMO warns of harms from FDA cuts

From my colleague Adam Feuerstein: The loss of key personnel at the FDA through layoffs, retirements, and resignations forced by HHS Secretary Robert F. Kennedy Jr. and Commissioner Marty Makary threaten to tip the agency into dysfunction by robbing it of experienced people needed to work closely with drugmakers, said Samit Hirawat, chief medical officer at Bristol Myers Squibb.

“It’s very concerning to see what is happening — nothing that has changed is for the good from a regulatory perspective,” he said, speaking Sunday evening at a STAT event in San Diego.

“You want people who are knowledgeable in the regulatory sciences, people who are not only knowledgeable, but responsible, who understand the disease, understand the process,” said Hirawat.

Bristol is monitoring the evolving situation at the FDA, he said, watching for signs the agency can’t do its job. Right now, there are more questions than answers.

“What is going to happen to the new drug applications or the biologic applications that we submit? What is going to happen to inspections of facilities? If you remember during Covid inspections could not happen, therefore drug approvals got delayed. Do we want that again?” he said.

politics

Survey shows vote of no confidence in RFK Jr.

From my colleague Adam Feuerstein: RFK Jr. will have a “dramatically negative impact” on science and should be removed as HHS secretary. Peter Marks should also be rehired, said an overwhelming majority of biopharma industry and Wall Street people surveyed by Josh Schimmer and Eric Schmidt, analysts at the investment bank Cantor Fitzgerald.

The analysts conducted this survey after they published a searing note on RFK Jr., warning of the destruction and chaos he will cause for the industry and for public health. (We will note that Cantor was run by Howard Lutnick until he became President Trump’s commerce secretary.)

More than 400 people participated in the survey conducted over the past week. Ninety-five percent of respondents said Kennedy will have a negative impact on science and 90% want to see him ousted from HHS.

Ninety-three percent of respondents want Peter Marks to return to the FDA in some form. Marks was forced to resign as the agency’s top regulator of vaccines, cell and gene therapies, and biologic drugs by RFK Jr. and FDA Commissioner Marty Makary. Many of Mark’s top deputies also left the FDA in the days following his ouster.

Half of the survey participants said the upheaval at the FDA will make it harder to get drugs and cell and gene therapies approved.

Politics

When will RFK Jr. face Senate questioning?

And where is RFK Jr. as this all plays out? Senate health committee Chair Bill Cassidy (R-La.) and ranking member Bernie Sanders (I-Vt.) have asked Kennedy to appear before the committee on April 10 to speak on the HHS layoffs.

But Kennedy is likely to delay his appearance by several weeks, my D.C. colleagues report.

Kennedy’s team confirmed receipt of the request but did not confirm attendance, according to committee staff. And after this week, the Senate is taking a two-week break; senators are due to return April 28.