Nearly three months after winning a contentious bid to buy obesity drugmaker Metsera Inc. for $10 billion, Pfizer Inc. reported its first statistically significant data of the acquired lead, ultra-long-acting glucagon-like peptide-1 (GLP-1) receptor agonist, showing a mean placebo-adjusted weight loss of up to 12.3% at 28 weeks, as well as competitive tolerability. The findings signal a potential for once-monthly maintenance dosing, although the company’s move to a higher dose in an upcoming phase III “may speak to aspirations for more weight loss,” noted Evercore ISI analyst Umer Raffat. Released alongside Pfizer’s full-year 2025 earnings showing a 2% year-over-year dip in revenues of $62.6 billion, due primarily to reduced demand of its COVID-19 products, the phase IIb Vesper-3 study tested the candidate previously known as MET-097i – and now called PF-08653944 – in participants with obesity or overweight without type 2 diabetes.
Ultragenyx rolls out gene therapy data in Sanfillipo syndrome
Ultragenyx Pharmaceutical Inc. disclosed new long‑term data from clinical studies evaluating UX-111 (rebisufligene etisparvovec), an AAV9 gene therapy for type A Sanfilippo syndrome. Against the fatal neurodegenerative lysosomal storage disorder, UX-111 showed substantial and durable biomarker improvements and meaningful functional benefits compared with natural history, with consistent and highly statistically significant results across age and disease severity, Ultragenyx said. UX-111 was well-tolerated, and the safety profile remains favorable. The BLA for the compound, which garnered a complete response letter last summer related to CMC issues, has been resubmitted to the U.S. FDA and the PDUFA date is expected to land in the third quarter of 2026. Results with UX-111, which is undergoing phase I/II/III development, will be offered at the Worldsymposium this week in San Diego.
NMD reports positive phase II data in Charcot-Marie-Tooth
Following on from a successful phase IIa proof-of-mechanism study in myasthenia gravis, NMD Pharma A/S has added clinical data on a second neuromuscular disorder, reporting positive phase IIa results for ignaseclant, in both type I and type II Charcot-Marie-Tooth (CMT) disease. The CMT study did not meet the primary endpoint of an improvement in the six-minute walk test; however, there were significant improvements in secondary endpoints, including muscle strength, functional performance and patient-reported outcomes.
Genentech licenses Sanegene’s RNAi asset in $1.7B deal
Genentech Inc. is paying $200 million up front and up to $1.5 billion in milestone payments to license one of Suzhou Sanegene Bio Inc.’s RNAi programs. Metabolic and autoimmune-focused Sanegene did not disclose specifics around the licensed candidate, except that it was derived from its LEAD (Ligand and Enhancer Assisted Delivery) platform. The same platform was behind its $1.2 billion deal with Eli Lilly and Co. to develop RNAi candidates for metabolic diseases in November 2025.
Medtronic to acquire Cathworks for $585M
Medtronic plc is continuing to put its money where its mouth is as it plans to exercise its option to acquire Cathworks Ltd. for up to $585 million. The company said last month it was committed to expanding its pipeline through strategic investments and targeted acquisitions. The move for Cathworks comes on the heels of Medtronic’s $90 million investment in Anteris Technologies Global Corp.
Trumprx launch threatened by shutdown, kickback concerns
At a recent Cabinet meeting, U.S. Health and Human Services (HHS) Secretary Robert Kennedy reportedly said he expected Trumprx to probably go live within 10 days. That was Jan. 29, two days before HHS was once again forced to shut down many of its activities due to a congressional gridlock over a fiscal 2026 appropriations package to keep the department and several others open beyond Jan. 30. Although a funding fix is in the works, a few days of a shutdown could delay the launch of Trumprx. But the shutdown isn’t the only, or biggest, threat to the portal designed to steer patients to drug companies’ direct-to-consumer online programs that offer discounted cash prices for prescription drugs, cutting out the middlemen.
Switching off resistance: Lixa’s bold AMR bet
Lixa Pty Ltd. has formed a partnership with the Global Antibiotic Research & Development Partnership and announced a AU$28 million (US$20 million) series B round to take Neox-121 to the clinic to fight antimicrobial resistance (AMR). Perth, Australia-based Lixa has invented a scalable breakthrough platform, called Neox, that looks to reverse bacterial resistance and could preserve antibiotics to extend their clinical usefulness and longevity. Lixa CEO Maud Eijkenboom told BioWorld the funds will take the compound through phase I trials and hopefully to proof of concept.
Disarming bacterial immune systems to diversify phage therapy
Certain chemical compounds can disable the bacterial immune systems that protect them from viruses, making the bacteria vulnerable to infection. Scientists at Indiana University have discovered a promising compound that works in different types of bacteria and could be used to develop potent and diverse phage therapies against bacterial infections, ultimately giving clinicians more options against antibiotic resistance.
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