In a U.S. Senate Finance Committee confirmation hearing today that was marked by shouted protests, outbursts of applause and tense exchanges on several issues beyond the reach of the Department of Health and Human Services (HHS), Robert F. Kennedy Jr. (RFK) tried to present himself as a man of science, not a conspiracy theorist or anti-vaxxer. Responding to news reports claiming that he’s anti-vax and anti-industry, President Donald Trump’s nominee for HHS secretary said, “I am neither.” Instead, RFK said, he’s disturbed the status quo by asking uncomfortable questions during his years of advocacy work on vaccines and other health-related issues. “I’m not going to apologize for that,” he added. Several senators on the committee weren’t buying his explanations, which they said were contradicted by his past statements. Some of them also pressed him on his commitment to the Medicare drug price negotiations. His answer was to reference an administration statement today supporting the negotiations while seeking more transparency in the process.

The science on vaccines and autism: no there there

At his confirmation hearing, Robert F. Kennedy Jr., the nominee for secretary of the U.S. Department of Health and Human Services, claimed that he is not anti-vaccine. But his record does not bear that out. Kennedy is a longstanding vaccine denier, and in 2021 was identified as one of the “Disinformation Dozen” – the 12 accounts responsible for the majority of disinformation about COVID-19 vaccines on social media platforms – by the British-American nonprofit Center for Countering Digital Hate. He has also spread disinformation about other vaccines including measles and polio vaccines.

Allografts can patch up failing heart

Implanted patches of iPS-derived heart muscle integrated with heart tissue in a primate model of heart failure, and in patients being treated in a clinical trial, marked progress toward a potential option for patients with advanced heart failure. “You can add muscle to the failing heart, and ... you can do so without safety concerns related to arrhythmia, for example, or tumor formation,” senior author Wolfram-Hubertus Zimmermann told reporters at a press conference announcing the findings, which the team reported online in Nature on Jan. 29, 2025. Zimmerman is the director of the Institute for Pharmacology and Toxicology at the University Medical Center Göttingen. Heart failure occurs when the heart cannot effectively supply the body with blood, because it cannot pump well or because it does not effectively fill with blood. The condition is progressive, and once it reaches an advanced stage, half of patients die within a year.

Regulus heads to phase III after more positive kidney disease data

Regulus Therapeutics Inc. disclosed further positive data from its ongoing phase Ib study testing RGSL-8429 in autosomal dominant polycystic kidney disease, but the bigger news might be its plans to move straight into a phase III trial later this year. A December 2024 meeting with the U.S. FDA resulted in an agreement for a single, pivotal study testing the drug, now with the generic name farabursen, with total kidney volume at one year as a primary endpoint for potential accelerated approval. Despite the positive update, shares of Regulus (NASDAQ:RGLS) were trading down 26% at midday.

Zentalis creates a path for its cancer study with layoffs

It’s one step backward in order to take two steps forward at Zentalis Pharmaceuticals Inc. In a restructuring to help fund the second part of its potentially registration-enabling Denali study, Zentalis has laid off 40% of its workforce. The clinical trial of azenosertib, Zentalis’ lead candidate and a WEE1 kinase inhibitor, is for treating advanced solid tumors and hematologic malignancies. The layoffs will come from the company’s business operations and R&D groups and, according to Zentalis, should be substantially completed in the second quarter of 2025. The company’s stock (NASDAQ:ZNTL) staggered at midday, with shares down 17% at $1.99 each.

Biopharma financings hit $102B in 2024, third highest year on record

Biopharma financings saw their third highest year, with companies collectively raising $102.15 billion in 2024, behind only 2020 and 2021. The amount marked a 44% jump from $70.97 billion in financings in 2023 and a 68% increase from $60.81 billion in 2022. The fourth quarter (Q4) contributed $15.45 billion, the lowest quarter of the year, following $16.38 billion in Q3, $23.07 billion in Q2, and a strong $47.25 billion in Q1.

VCs lead rising UK biotech investments in 2024, analysis shows

The final tally shows U.K. biotechs raised £3.5 billion (US$4.35 billion) in 2024, a stonking 93% more than in 2023, and surpassing the total in 2020, before the life sciences investment boom sparked by the pandemic. Of this, £2.1 billion was in venture capital, a 64.8% increase on 2023, while U.K biotechs attracted £1.5 billion in follow-on offerings, most of it raised by companies listed on Nasdaq. Once again, there were no IPOs in London. At a time when the U.K. government is fixated on trying to grow the economy, the numbers are an indication of how life sciences could contribute through an “exceptional capacity for growth and innovation,” which is “fueled by outstanding science and improving financial performance,” said Steve Bates, CEO of the Bioindustry Association (BIA), the industry group that compiled the figures.

FDA trial-diversity guidelines disappear after Trump DEI order

Without fanfare, the U.S. FDA has taken down from its website the guidelines regarding diversity action plans related to clinical trials. Last summer, the agency issued draft guidance on the matter, and Robert Califf, FDA commissioner, at the time called the move “an important step – and one of many ongoing efforts – to address the participation of underrepresented populations” in trials. President Donald Trump recently issued an executive order putting a stop to diversity, equity, and inclusion programs across the government.

FCA cases reach new high in 2024, but value of recoveries no outlier

False Claims Act (FCA) litigation in the U.S. spiked significantly according to a new report by Gibson, Dunn & Crutcher, LLP, at least in terms of the number of cases brought to the courts. However, the report states also that the total dollar value of these cases is in line with historical norms, suggesting that the average recovery amount has tapered off significantly.

Also in the news

Allogene, Alumis, Apellis, Astrazeneca, Atai, Atalanta, Beacon, Beximco, Beyondspring, Bioage Labs, Biogen, Calidi, Clarametyx, Commit, Concerto, CSL, Daiichi Sankyo, Eisai, Genentech, GSK, Helicore, Humacyte, Inbrain, Intensity, Invisishield Technologies, ITM, Kashiv, Leap, Lineage Cell, Lutris, March, Medexus, Metsera, Neurocrine, Nrx, Oncomatryx, Oncozenge, Orgenesis, OS Therapies, Precision, Processa, RLS, Roche, Sab, Saol, Scholar Rock, Seed, Takeda, Telix, Tris, Virpax