IntellaTurn, LLC

• The FDA refused to review Moderna’s application for a new influenza vaccine, a surprise decision that could raise concerns about the agency’s posture toward drug companies and the Trump administration’s policies on vaccines.

• Moderna, revealing the rejection, took the unusual step of releasing the letter it had received from Vinay Prasad, who heads the FDA’s biologics division.

• It also issued a strongly worded statement from its CEO Stéphane Bancel, who said the decision “does not further our shared goal of enhancing America’s leadership in developing innovative medicines.”

• At the heart of the dispute is what existing influenza vaccine Moderna should have used as a control when testing the efficacy of its new shot, which utilizes the same mRNA technology the company used in its Covid-19 vaccine. (STAT)

• After waiting through a three-month delay, Regenxbio’s investigational gene therapy RGX-121 has been rejected by the FDA for the treatment of Hunter syndrome, an ultra-rare neurodegenerative condition.

• In a complete response letter, the regulator cited “uncertainty” over the eligibility criteria that Regenxbio used for the study included in RGX-121’s data package.

• The agency also questioned the applicability of using natural history external controls and the validity of the surrogate endpoint—cerebrospinal fluid levels of the biomarker DS26—as basis for approval.

• For a resubmission, the FDA recommended running a new study, adding more patients, following them for longer periods and using an untreated comparator group.

• Regenxbio, however, called these options “challenging” in a release, given the ultra-rare nature of Hunter syndrome. (BioSpace)

• For all its success across a wide range of cancer types, Merck’s PD-1 superstar Keytruda has never been able to crack the code for one of the toughest-to-treat indications in oncology—ovarian cancer.

• But now, 13 years after the FDA initially approved it for skin cancer, the regulator has approved Keytruda as a second- or third-line treatment for patients with a certain type of ovarian cancer.

• With the green light, Keytruda becomes the first immune checkpoint inhibitor approved for this indication.

• Keytruda is to be used in combination with the chemotherapy paclitaxel and with or without Roche’s targeted cancer therapy Avastin.

• The indication covers those with platinum-resistant recurrent ovarian, fallopian tube or primary peritoneal carcinoma with PD-L1-positive tumors.

• The approval also covers its subcutaneous version, Keytruda Qlex, which the FDA approved five months ago. (Fierce Pharma)

• The website is part of the president’s plan to lower drug prices in the US and to counter political attacks by Democrats who have increasingly focused on rising health care costs.

Trump unveiled the site on February 5, saying “this launch represents the largest reduction in prescription drug prices in history by many many times, and it’s not even close.”

• The website touts steep discounts for many drugs, compared to their list prices.

• But an analysis by STAT reveals that’s far from the whole story.

• In some cases, consumers could save hundreds of dollars by going with the exact same generic medicine, instead of TrumpRx’s price.

• STAT’s analysis is similar to a finding from Sarah Karlin-Smith, a research director at the advocacy group Public Citizen, and from Brian Reid, founder of Reid Strategic and a senior fellow at Tufts’ Center for the Evaluation of Value and Risk in Health.

• STAT’s analysis adds to questions about whether TrumpRx will meaningfully improve patients’ ability to afford their prescriptions.

• There is no such disclaimer about generics. (STAT)

• Madrigal Pharmaceuticals is leaning further into its liver disease portfolio, inking a preclinical partnership with Chinese biotech Suzhou Ribo Life Science for six investigational siRNA programs aimed at the liver.

• Under the terms of the agreement, Madrigal is fronting $60 million and promising up to $4.4 billion in certain milestone payments across all programs. Ribo, which entered the alliance via its subsidiary Ribocure Pharmaceuticals, will also be eligible for royalties on net sales.

• Madrigal plans to test combination regimens of these siRNA therapies with its metabolic associated steatohepatitis (MASH) drug Rezdiffra, which scored the first FDA approval in the indication in March 2024.

• The company expects to start IND-enabling activities for initial candidates from the Ribo deal in 2026.

• SiRNAs—small stretches of non-coding RNA that bind to a target mRNA molecule, in turn preventing its translation into a protein—offer a precision gene silencing approach to MASH. (BioSpace)

• Roche detailed just how much its BTK inhibitor was deemed non-inferior compared to its blockbuster medicine Ocrevus in a late-stage trial in primary progressive multiple sclerosis.

• Fenebrutinib cut the risk of disability progression by 12% versus the CD20-targeting antibody Ocrevus in the phase 3 trial called FENtrepid.

• The number indicates a “nominal trend toward better efficacy, but the level is modest,” Jefferies analysts wrote in a note. (Endpoints)

• Hims & Hers caused a firestorm last week when it revealed that it planned to launch a knockoff version of Novo Nordisk's new oral weight loss medicine.

• But the reaction to the move has caused the company to quickly retreat.

• Following rebukes from Novo and the FDA commissioner earlier last week, Hims’ plan truly began to unravel on February 6, when HHS General Counsel Mike Stuart announced on X that his agency had referred the online health and wellness company to the Department of Justice (DOJ) for investigation.

• The probe concerns potential violations of the Federal Food, Drug, and Cosmetic Act “and applicable Title 18 provisions,” Stuart said.

  • The HHS official posted the notice amid growing backlash to Hims' announcement that it planned to launch a compounded, semaglutide-containing pill for obesity.

• Novo swiftly branded the move “illegal mass compounding,” drawing the safety of Hims’ pill into question on the basis of its allegedly “untested delivery mechanism.”

  • The Danish drugmaker pledged to take legal action, and its stance was echoed in a social media post by FDA Commissioner Marty Makary. (Fierce Pharma)

• For example, previous studies provide evidence that the shingles vaccine may help prevent cardiovascular conditions, such as heart disease and stroke.

• A study published last month even found that the shingles vaccine might help slow biological aging.

• Additionally, there has been a good deal of research focused on the shingles vaccine and its potential role in dementia prevention.

• This new study discovered evidence suggesting receiving the shingles vaccine may help reduce or delay dementia diagnosis in older adults.