Daraxonrasib is safe and effective in patients with previously treated, advanced, RAS-mutated pancreatic ductal adenocarcinoma. Researchers evaluated whether daraxonrasib, an oral RAS(ON) multiselective inhibitor that targets guanosine triphosphate-bound mutant and wild-type RAS, would be safe and effective in patients with previously treated, advanced, RAS-mutated PDAC. “These results support the ongoing RASolute 302 study, a global randomized phase 3 trial of daraxonrasib as compared with chemotherapy as second-line treatment in patients with metastatic PDAC (ClinicalTrials.gov number, NCT06625320),” the researchers concluded. We want you to take advantage of everything Cancer Therapy Advisor has to offer. To view unlimited content, log in or register for free. ... Register now at no charge to access unlimited clinical news, full-length features, case studies, conference coverage, and more. Current PDAC therapies provide only modest benefit, study researchers noted. Because activating RAS mutations occur in more than 90% of PDAC cases, the researchers examined whether daraxonrasib, an oral RAS(ON) multiselective inhibitor that targets guanosine triphosphate-bound mutant and wild-type RAS, would be safe and effective in these patients. This phase1/2 trial (NCT05379985) included 168 patients from 16 US sites.

Original title: “Daraxonrasib Appears Safe and Effective in Previously Treated RAS-Mutated PDAC - Cancer Therapy Advisor”

The phase 1/2 data led to the phase 3 RASolute 302 trial which supported the FDA's allowance of early access to daraxonrasib in PDAC. Broader RAS G12/G13/Q61 cohort yielded 29% ORR with 8.1-month mPFS and 15.6-month mOS, suggesting activity beyond G12-only disease. Across 168 PDAC patients (≤300 mg), treatment-related AEs occurred in 96%; grade ≥3 events in 30%, dominated by rash, diarrhea, nausea, mucositis, vomiting, and fatigue. RASolute 302 demonstrated median OS 13.2 vs 6.7 months versus chemotherapy in the ITT population (HR 0.40; P<.0001) and met PFS/OS in RAS G12. Global randomization to daraxonrasib 300 mg daily versus investigator-choice cytotoxic therapy allowed enrollment across G12D/V/R, other RAS variants, and RAS wild-type, with QoL as a key secondary end point.SHOW MORE “Data from the Phase 1/2 trial show that daraxonrasib demonstrated promising clinical antitumor activity and durable responses, with an acceptable safety and tolerability profile, in patients with previously treated metastatic RAS mutant PDAC. These results, along with those from our Phase 3 trial, RASolute 302, strengthen our confidence in daraxonrasib’s potential to establish an important new treatment option for patients with pancreatic cancer and other RAS-addicted cancers,” added Sandler. These early-phase results led to the launch of the phase 3 RASolute 302 trial (NCT06625320), which supported the FDA’s decision to allow expanded access to daraxonrasib prior to the drug’s approval.3 · “RAS mutations are a central driver of disease across multiple solid tumors, including particularly pancreatic ductal adenocarcinoma. Grade 3 or higher treatment-related events occurred in approximately one-third of patients, at a rate of 30%. The adverse events occurring in at least 10% of patients included rash, diarrhea, nausea, stomatitis or mucositis, vomiting, and fatigue. This safety profile is generally consistent with other RAS-targeted agents and considered manageable in the context of the disease's severity and the historical lack of effective options in this setting.

Original title: “Daraxonrasib PDAC Data Published in NEJM as FDA Allows Expanded Access | Targeted Oncology - Immunotherapy, Biomarkers, and Cancer Pathways”

Despite understanding the link ... many rare diseases, the underlying cause of stiff person syndrome is not well understood. This makes it very difficult to diagnose and treat it, explained Gehchan. “There is a significant need for therapies that deliver durable and substantial clinical benefit while eliminating the burden and risks of chronic, off-label treatment,” said Gehchan. At present, there is only one noteworthy therapy in clinical trials for stiff ... Despite understanding the link between GAD65 and GlyR antibodies and the condition, like with many rare diseases, the underlying cause of stiff person syndrome is not well understood. This makes it very difficult to diagnose and treat it, explained Gehchan. “There is a significant need for therapies that deliver durable and substantial clinical benefit while eliminating the burden and risks of chronic, off-label treatment,” said Gehchan. At present, there is only one noteworthy therapy in clinical trials for stiff person syndrome, and it is California-based Kyverna Therapeutics’ CAR T-cell therapy miv-cel. No medicines exist for stiff person syndrome yet, but a new CAR T-cell therapy from Kyverna Therapeutics is moving closer to approval. “Current off-label treatments fail the majority of people with stiff person syndrome, require chronic dosing, and do not stop the progression of the disease,” said Gehchan. “There is a significant need for therapies that deliver durable and substantial clinical benefit while eliminating the burden and risks of chronic, off-label treatment.” No currently available medicines treat the root cause of the syndrome, but Kyverna Therapeutics’ cell therapy has been making steady progress and is poised for regulatory approval. Extremely rare, stiff person syndrome affects around one in a million people. It was first identified by two neurologists at the Mayo Clinic in Minnesota in 1956, and was described, at the time, as a “mystery illness that stiffens victims.” Public awareness grew when the singer Celine Dion announced her diagnosis in 2022.

Original title: “Kyverna CAR-T therapy for stiff person syndrome nears approval”

Aardvark Therapeutics had previously voluntarily suspended studies of ARD-101—and a related asset called ARD-201—after detecting anomalous echocardiographic readings in healthy volunteers that could indicate reduced heart efficiency.

Original title: “Future of Aardvark’s Prader-Willi drug in doubt as FDA slaps full hold on program”

One of the company’s more closely watched research assets, BIIB080 is now heading to late-stage testing based on “the strength of the biomarker and efficacy data” seen in a Phase 2 trial.

Original title: “Biogen pushing tau drug forward despite Alzheimer’s study failure”

While Biogen’s tau-targeting therapy didn’t demonstrate improvement on a dementia severity scale, the company touted biomarker and cognitive improvements from the Phase 2 study, leaving analysts eager for more data.

Original title: “Biogen takes tau Alzheimer’s therapy to Phase 3 despite mid-stage fail, prompting cautious optimism”

Beqalzi is the first BCL2 inhibitor approved for relapsed or refractory mantle cell lymphoma.

Original title: “BeOne wins mantle cell lymphoma approval, opening new therapy class”

Responders achieved weight loss and improvements in physical function

Original title: “Novo Nordisk data shows oral semaglutide delivers significant weight loss in phase 3 trial”

Recent clinical investigations, identified by ClinicalT […] Researchers are exploring how these new pathways can be leveraged to develop more effective treatments for children facing serious health challenges. The focus remains on translating these discoveries into tangible improvements in pediatric care. ... For any suggestion and feedback, please contact us. Date: May 13, 2026 ©www.geneonline.com All rights reserved. Collaborate with us: [email protected] ... Ted Turner’s Brain Disease May Be More Common Than Other Rare Neurological Conditions, Study Finds Recent clinical investigations, identified by ClinicalTrials.gov identifiers NCT02459327 and NCT03945552, have revealed potential new therapeutic pathways for critical pediatric conditions. The studies suggest that innovative molecular approaches and advanced clinical strategies could significantly alter current treatment methods.

Original title: “Clinical Trials NCT02459327 and NCT03945552 Reveal Potential New Therapeutic Pathways for Critical Pediatric Conditions - GeneOnline News”

EMA's office is closed from 18:00 on 13 May 2026 until 08:30 on 18 May 2026.The product emergency hotline is available outside working hours and on public holidays. To reach…

Original title: “EMA business hours over Ascension Day holidays, 14 and 15 May”

Indirect comparisons between BridgeBio’s Attruby and Pfizer’s tafamidis products showed a numerical survival benefit with the biotech’s drug.

Original title: “BridgeBio stands up to Pfizer in ATTR-CM as late-stage data hint at better survival”

Enfortumab vedotin and pembrolizumab recommended for NHS Scotland

Original title: “SMC backs first-line metastatic bladder cancer combination after trial results”

− Reductions in All-Cause Mortality and Recurrent Cardiovascular Events Maintained Across Key Patient Subgroups, Clinical benefits were similarly consistent regardless of comorbidity burden or concomitant use of disease-modifying therapies, including tafamidis and heart failure medications such as SGLT2 inhibitors, MRAs, ß-blockers and ACEi/ARB/ARNI. Consistent effects were also observed in women, a historically underrepresented population in ATTR-CM trials. A separate pooled analysis of clinical trial and post-marketing safety data evaluated the relationship between transthyretin-lowering RNAi therapies and vitamin A deficiency-related adverse events. Patients treated with vutrisiran and patisiran are suggested to take the recommended daily allowance of vitamin A. The analysis included more than 25,000 patient-years of treatment exposure across vutrisiran and patisiran programs. “These new HELIOS-B analyses show that the clinical benefits of vutrisiran were maintained across these clinically complex patient groups, as well as in patients receiving background therapies, including TTR stabilizers and disease-modifying heart failure therapies, reinforcing both the consistency of the treatment effect and its relevance in real-world clinical practice. The data presented at Heart Failure 2026, the annual congress of the Heart Failure Association of the European Society of Cardiology, show consistent clinical benefit across patient populations commonly encountered in clinical practice, including those with a high disease burden, supporting its use as a first-line treatment option for this rapidly progressive and life-threatening disease.

Original title: “New Data Analyses Presented at Heart Failure 2026 Demonstrate Robust and Consistent Clinical Benefit of Vutrisiran as a First-Line Treatment Option Across ATTR-CM Patient Populations, Including Patients with a High Disease Burden | Press Releases | daily-tribune.com”

Second national grant strengthens company’s expanding global IP estate

Original title: “Poolbeg secures Canadian patent for POLB 001 in cancer immunotherapy‑induced CRS”