Notizia - Recent Pharma, Healthcare and Biotech Happenings
May 13, 2025
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Eli Lilly and Company released detailed results from the Phase IIIb SURMOUNT-5 clinical trial, showing that ZEPBOUND (tirzepatide) significantly outperformed WEGOVY (semaglutide) in weight loss among adults with obesity or overweight and at least one weight-related condition, excluding diabetes. The findings, presented at the 32nd European Congress on Obesity and published in The New England Journal of Medicine, highlight a 20.2% average weight reduction with ZEPBOUND versus 13.7% with WEGOVY over 72 weeks, a 47% relative difference.
“Thanks to the latest advancements in obesity management medications, more physicians and patients are witnessing significant weight reduction beyond what they have seen before,” said Dr. Louis J. Aronne, principal investigator of SURMOUNT-5. “The SURMOUNT-5 head-to-head results demonstrated that tirzepatide led to greater weight reduction compared to semaglutide, providing further evidence to support tirzepatide as an effective option for obesity management.”
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The trial also demonstrated superiority of ZEPBOUND across all five key secondary endpoints. Nearly 65% of participants on ZEPBOUND achieved ≥15% weight loss compared to 40.1% with WEGOVY. In waist circumference reduction, ZEPBOUND patients lost an average of 7.2 inches compared to 5.1 inches with WEGOVY. On average, ZEPBOUND users lost 50.3 lbs (22.8 kg), while WEGOVY users lost 33.1 lbs (15.0 kg).
“In the SURMOUNT-5 trial, ZEPBOUND demonstrated a significantly higher magnitude of weight reduction compared to WEGOVY across all comparisons,” said Dr. Leonard Glass, senior vice president, global medical affairs at Lilly. “These data confirm ZEPBOUND as a leading treatment option for people living with obesity and equip healthcare providers with critical insights to make well-informed treatment decisions as part of a comprehensive obesity care plan.”
ZEPBOUND’s safety profile was consistent with previous trials, with most adverse events being mild to moderate and gastrointestinal-related. Treatment discontinuation due to side effects occurred in 6.1% of ZEPBOUND users versus 8.0% for WEGOVY. However, the trial wasn’t powered to compare safety between the two drugs directly.
ZEPBOUND is approved in the U.S. for adults with obesity or overweight and related medical conditions, while its counterpart, Mounjaro, is indicated for type 2 diabetes. WEGOVY and Ozempic (semaglutide) serve similar purposes under different labels.
In May 2025, Verastem Oncology announced that the FDA granted accelerated approval for AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib capsules and defactinib tablets) for adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. This marks the first and only FDA-approved therapy for this rare form of ovarian cancer, arriving well ahead of the PDUFA date of June 30, 2025. The oral combination will be commercially available in the U.S. as a co-packaged prescription treatment.
“Today’s approval of AVMAPKI FAKZYNJA CO-PACK for patients with KRAS-mutated recurrent low-grade serous ovarian cancer represents not only the first-ever FDA-approved treatment specifically for this rare cancer but also a new day for people living with this disease,” said Dan Paterson, President and CEO of Verastem Oncology.
The approval was based on the Phase II RAMP 201 trial, which showed a 44% overall response rate in patients with KRAS-mutant recurrent LGSOC. AVMAPKI FAKZYNJA CO-PACK has also received Breakthrough Therapy and Orphan Drug Designations, and continued approval may depend on results from the confirmatory Phase III RAMP 301 trial.
“The approval of avutometinib plus defactinib brings a much-needed therapeutic option to patients and establishes this combination as the new standard of care,” said Dr. Rachel Grisham of Memorial Sloan Kettering Cancer Center.
“Today we’re celebrating a milestone with the first-ever FDA-approved treatment option specifically for patients with recurrent LGSOC with a KRAS mutation,” added Nicole Andrews, Chair of the STAAR Low-Grade Serous Ovarian Cancer Foundation.
The approval of AVMAPKI FAKZYNJA CO-PACK represents a landmark achievement in gynecologic oncology, signaling progress for patients with RAS/MAPK-pathway-driven cancers.
Thermosome, a clinical-stage drug development company advancing targeted tumor therapies, has announced that its lead candidate, THE001, has been granted Orphan Drug Designation (ODD) by the FDA for the treatment of soft tissue sarcomas (STS). This designation adds to the ODD already granted by the European Medicines Agency (EMA), further validating the therapeutic potential of THE001.
The FDA’s ODD program is designed to encourage the development of treatments for rare diseases and offers key benefits, including seven years of market exclusivity upon approval, exemption from user fees, and tax credits for qualified clinical trials. The FDA acknowledged the clinical potential of Thermosome’s doxorubicin-encapsulating thermosensitive liposomes, which may demonstrate superior efficacy compared to standard non-liposomal doxorubicin.
“We see the U.S. Orphan Drug Designation as a strong regulatory validation of the potential of our innovative approach in soft tissue sarcoma,” said Dr. Pascal Schweizer, co-founder and CEO/CFO of Thermosome. “This recognition, based on preclinical and early clinical data from our Phase I study, marks an important milestone and is a further step into the U.S. market, the world’s most important market for patent-protected drugs. In parallel, we are evaluating strategic partnerships to advance THE001 and fully realize its therapeutic potential.”
In addition to this regulatory milestone, Thermosome announced the expansion of its intellectual property estate with the publication of four new PCT patent applications, bringing its total patent families to seven. These patents reinforce the company’s proprietary position around THE001 and its broader liposomal and lipid nanoparticle (LNP) technologies.
The newly published patents include:
Thermosome continues to work closely with external patent attorneys and has reported no freedom-to-operate concerns to date, underscoring its strong and secure innovation platform.
Beam Therapeutics Inc., a pioneer in precision genetic medicine using base editing, announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302 for the treatment of alpha-1 antitrypsin deficiency (AATD). BEAM-302 is a liver-targeted lipid nanoparticle (LNP) formulation that delivers a guide RNA and mRNA encoding a base editor to correct the genetic mutation causing AATD.
AATD is a serious inherited disorder affecting the lungs and liver, with limited treatment options currently available. The RMAT designation aims to accelerate the development of regenerative medicines for life-threatening diseases and offers benefits such as early and frequent FDA interactions, rolling review, and potential eligibility for priority review and accelerated approval.
“Just weeks after IND clearance, the FDA’s RMAT designation recognizes the transformative potential of BEAM-302 as a one-time treatment for AATD,” said Dr. Giuseppe Ciaramella, President of Beam Therapeutics. “This underscores the strength of our clinical data and the promise of base editing to directly address the underlying genetic mutation.”
Beam recently reported positive interim data from its Phase I/II trial of BEAM-302. In the first three single-ascending dose cohorts, the therapy was well tolerated and showed durable, dose-dependent correction of the disease-causing mutation. The 60 mg dose cohort achieved total AAT protein levels above the therapeutic threshold, establishing early proof of concept for in vivo base editing.
The company has initiated dosing in the fourth cohort at 75 mg and plans to begin dosing AATD patients with mild to moderate liver disease in Part B of the trial in the second half of 2025. Updated clinical data is expected to be presented at a medical conference later this year. Beam previously received FDA IND clearance for BEAM-302 in March 2025.
Capsida Biotherapeutics has announced FDA clearance of the Investigational New Drug (IND) application for CAP-002, a first-in-class, intravenously administered gene therapy for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE). CAP-002 is the first gene therapy candidate to enter clinical trials using an engineered AAV capsid capable of crossing the blood-brain barrier while detargeting off-target tissues such as the liver and dorsal root ganglia (DRG).
STXBP1-DEE is a rare and devastating pediatric neurological condition, affecting around 5,000 children across the U.S. and Europe. Caused by mutations in the STXBP1 gene, essential for normal neurotransmission, the disorder leads to early-onset seizures, developmental delays, motor dysfunction, and increased risk of SUDEP. Currently, there are no approved therapies targeting the underlying genetic cause.
CAP-002 was developed using Capsida’s proprietary capsid engineering and manufacturing platform. In preclinical studies involving non-human primates, CAP-002 demonstrated more than 70% neuronal transduction across critical brain regions while effectively avoiding liver and DRG uptake. Toxicology studies also indicated a favorable safety profile, supporting advancement into human trials.
The SYNRGY Phase I/IIa clinical trial is now underway, with first patient dosing anticipated in Q3 2025. CAP-002 was granted Orphan Drug Designation by the FDA in October 2024, highlighting its promise as a potential disease-modifying therapy for STXBP1-DEE.
“This milestone not only advances Capsida’s mission but represents hope for families affected by STXBP1-DEE,” said Dr. Swati Tole, Chief Medical Officer of Capsida. “We’re proud to launch the SYNRGY trial and explore the potential of CAP-002 to transform care for this underserved population.”
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