December 10, 2024

The need-to-know this morning

New Amsterdam Pharma said that its CETP inhibitor — a class of drugs the industry once invested heavily in, then abandoned — succeeded in its third Phase 3 trial. The experimental drug successfully lowered levels of bad cholesterol in certain patients with uncontrolled heart disease.
GSK struck a deal with computational biotech startup Relation to develop drugs for fibrotic disease and osteoarthritis.
Cardiff Oncology said that, in a small clinical trial, 64% of metastatic colon cancer patients who received a high dose of its drug saw their tumors shrink, compared to 33% on the control arm. The drug, CRDF-004, is meant to block an oncogene called RAS.

politics

Pharma isn't lobbying against RFK Jr.'s nomination

RFK Jr., President-elect Trump's pick to run the Department of Health and Human Services, has repeatedly blasted the pharmaceutical industry, accusing drugmakers of corruption and poisoning the American public. But about a month ahead of when confirmation hearings could begin, the industry has not yet lobbied senators to stop his nomination.

It's a signal that drugmakers are hoping to turn a new leaf with the Trump administration. They see an opportunity to water down drug pricing reforms that were passed under the Biden administration and also address a long list of desired FDA and PBM policy changes.

Read more from STAT's Rachel Cohrs Zhang and John Wilkerson.

financing

New oncology startup launches with $52 million

Cure Ventures, a new venture firm, has launched its first startup, Tasca Therapeutics, with $52 million in funding.

Tasca, which is developing a new breed of cancer therapies, had originally set out to raise as much as $70 million. But "the market is tough. There's no doubt about it," said Milenko Cicmil, CEO of the startup. He described the current biotech startup field as an "austerity-driven environment."

Tasca is developing small molecule treatments that target and bind to specific pockets on the pockmarked surface of proteins. The hope is that the medicines can lock onto cancer-associated proteins and kill the cancer cells.

Read more from STAT's Allison DeAngelis.

oncology

GSK myeloma drug builds case for comeback

New trial results show that a blood cancer treatment that GSK had previously withdrawn actually helped patients live longer, findings that support GSK's attempts revive the drug.

In the study, a treatment combination that included the drug, called Blenrep, cut the risk of death by 42% among patients with multiple myeloma, according to detailed results presented at ASH yesterday.

Blenrep won accelerated approval from the FDA in 2020, but GSK withdrew it in 2022 after it failed to meet its goal in a confirmatory study.

oncology

Early use of J&J drug may prevent mulitple myeloma

Another new study presented at ASH suggests that early treatment with J&J's Darzalex may delay or prevent multiple myeloma in high-risk patients.

Doctors have long debated whether treatment should be given to these high-risk patients with so-called "smoldering myeloma." These patients technically don't have cancer yet, and many never develop active myeloma either.

The study found that after five years, 63.1% of patients who received Darzalex had not progressed to active myeloma, versus 40.8% of patients who didn't get the drug. J&J said it's submitted these data to regulators for approval.

gene therapy

Uniqure gets FDA OK to seek accelerated approval

From STAT's Jason Mast: Uniqure said today that the FDA agreed it could seek accelerated approval for its Huntington's disease gene therapy, as opposed to requiring a large, placebo-controlled trial.

The announcement is the latest sign of changes within the agency, as Peter Marks, head of the center for biologics, pushes his reviewers and reshuffles the division to be more flexible when it comes to genetic medicines for devastating rare diseases. (How such an approach fares under the coming Trump administration remains to be seen.) The stance could both hasten the development of powerful treatments in rare or slow-moving diseases and allow ineffective products to reach patients.

The agency has previously asked for randomized trials for Huntington's, which slowly robs patients of both muscle control and cognition around mid-life. A closely watched Ionis trial failed in 2021, despite promising early signs. Uniqure said the agency will allow it to file for approval based on years-long follow-up data from its Phase I/II trial, which has enrolled over 40 patients. The agency will primarily look at whether patients declined more slowly than the disease's natural history would suggest.

That path, said Uniqure CEO Matt Klein, both makes medical sense and, going forward, would change how investors consider Huntington's disease. It could dramatically reduce the cost and timelines for clinical trials. "I think it definitely changes the calculus," he said.

health tech

A tech startup wants drugmakers to study GLP-1s in Parkinson's

Amid the boom in GLP-1 drugs and efforts to study them in a range of diseases, one health tech startup called Koneska Health has been pitching drugmakers to study the treatments in Parkinson's disease.

Why? Well currently in Parkinson's trials, doctors manually measure tremors of patients to quantify how severe their symptoms are. Koneksa has technology that it argues can measure tremors much more precisely.

But for its tools to start being used in trials, Koneska can't start studies all on its own. It needs interested drug companies to help foot the bill. Its pitch to study GLP-1s highlights the predicament of digital health companies trying to push innovation in clinical trials that are ultimately at the mercy of drug developers who pay for their services.