Tag

Phase 3 Trial

All articles tagged with #phase 3 trial

business14 days ago•2 min saved

Novartis DM1 drug flops in phase 3, clouding $12B Avidity deal

Novartis’ phase 3 Harbor trial of del-desiran failed to meet its primary endpoint in myotonic dystrophy type 1 (DM1), undermining the $12 billion acquisition of Avidity Biosciences and sending the stock lower in premarket trading. While some secondary signals were noted, the miss raises questions about del-desiran’s contribution to future sales; Novartis will review the full data with regulators to determine next steps. The company also continues with other Avidity assets—delpacibart zotadirsen for Duchenne and delpacibart braxlosiran for FSHD—pursuing next actions based on newer data and FDA discussions.

via Fierce Biotech|

#avidity-biosciences #business #del-desiran

health19 days ago•2 min saved

Angelman syndrome drug GTX-102 misses Phase 3 endpoint, deal setback for Ultragenyx

Ultragenyx said its experimental Angelman syndrome therapy GTX-102 showed no benefit versus a sham in a large Phase 3 trial, ending earlier hopes from earlier results and creating a major hurdle for the company's profitability plans in the rare-disease space.

via STAT|

#angelman-syndrome #biotech #gtx-102

health-and-medicine20 days ago•7 min saved

One-Night Pill Cuts Sleep Apnea Breathing Interruptions by 44%

A once-nightly oral pill called AD109 (aroxybutynin plus atomoxetine) reduced the apnea–hypopnea index by about 44% in a six-month phase 3 trial across 646 adults who could not tolerate CPAP, with improvements in nighttime oxygen metrics and overall disease severity (over 40% moved to a less severe category and 18% achieved complete control). Side effects were generally mild (dry mouth, nausea, insomnia, difficulty urinating), with about 21% discontinuing. An NDA has been submitted and FDA Fast Track designation suggests a potential 1Q 2027 action date if accepted for review.

via Science Daily|

#ad109 #cpap-alternative #fda-fast-track

health20 days ago•1 min saved

GSK gears up for phase 3 with mRNA flu vaccine after promising phase 2 data

GSK reports its mRNA influenza vaccine candidates elicited higher immune responses than standard and high-dose vaccines in a 971-participant phase 2 trial, with acceptable safety. The company plans a phase 3 study this month for the optimized FLUm3HA.b-3NA candidate (targeting HA and NA), aiming to boost its faltering flu business amid a market historically characterized by variable vaccine effectiveness. Moderna’s recent FDA approval of an mRNA flu vaccine underscores the growing role of mRNA approaches in flu protection.

via Fierce Biotech|

#gsk #health #influenza-vaccine

$Hypofractionated Salvage RT Not Superior for Prostate Recurrence, Adds GI Toxicity$

medicine24 days ago•3 min saved

Hypofractionated Salvage RT Not Superior for Prostate Recurrence, Adds GI Toxicity

In the phase 3 SHARE trial from South Korea, hypofractionated salvage radiotherapy (65 Gy in 26 fractions) did not improve 4-year biochemical progression-free survival compared with conventional salvage RT (66 Gy in 33 fractions) for biochemical recurrence after radical prostatectomy. Distant metastasis-free and prostate cancer–specific survival were similar between groups, while late GI toxicity was higher with hypofractionation (7.9% vs 0.7%), particularly without an endorectal balloon; quality of life was similar in both arms. Concurrent ADT and positive surgical margins were associated with better biochemical outcomes. The authors suggest hypofractionated RT could be a viable alternative given comparable outcomes, but longer follow-up and more data on balloon use are needed.

via Medscape|

#hypofractionation #medicine #phase-3-trial

health1 month ago•5 min saved

Personalized mRNA vaccine plus Keytruda shows promising Phase 3 melanoma results

Moderna and Merck report that adding the patient-specific mRNA cancer vaccine intismeran (mRNA-4157) to Merck’s Keytruda significantly extended recurrence-free and distant metastasis-free survival versus Keytruda alone in a Phase 3 trial of 1,137 melanoma patients. Interim results suggest meaningful benefit, with mature overall-survival data still pending; five-year Phase 2 data had previously shown substantial risk reductions. Full details will be presented at an upcoming conference.

via Ars Technica|

#health #immunotherapy #melanoma

health1 month ago•5 min saved

Personalized mRNA cancer vaccine clears Phase 3 hurdle in melanoma

A patient-specific mRNA vaccine (intismeran autogene) combined with Keytruda met the Phase 3 primary endpoint in a 1,137-patient trial of resected melanoma, improving recurrence-free survival and potentially paving the way for the first FDA-approved mRNA-based cancer therapy; full data are to be presented later, with safety profile consistent with Keytruda.

via washingtonpost.com|

#cancer-immunotherapy #health #keytruda

health1 month ago•3 min saved

KRAS-targeting drug offers renewed hope in advanced pancreatic cancer

An experimental KRAS-inhibiting pill, daraxonrasib, significantly extends median survival in advanced pancreatic cancer (13.2 months vs. 6.7 months with chemotherapy) in a phase 3 trial, and is available to eligible patients via the FDA Expanded Access Protocol at 10 Intermountain Health hospitals while full approval is pending; it’s a second-line option with manageable side effects and is not a cure.

via USA Today|

#daraxonrasib #expanded-access-protocol #health

health1 month ago•2 min saved

MoonLake's nanobody delivers strong phase 3 results in psoriatic arthritis

MoonLake Immunotherapeutics reported positive topline phase 3 IZAR-1 data for biologic‑naïve psoriatic arthritis patients treated with sonelokimab, with week‑16 endpoints meeting across ACR50 (42.1%), ACR20 (66.5%), minimal disease activity (41.2%), and PASI90 in patients with skin involvement (61%), though there is no active comparator arm and topline results will be followed by blinded analyses with full data due in H1 2027; IZAR-2 and other IL-17 nanobody programs are ongoing.

via Fierce Biotech|

#health #moonlake-immunotherapeutics #nanobody

business1 month ago•0 min saved

Novo Nordisk suffers Phase 3 setback as CV inflammation drug fails

Novo Nordisk’s anti-inflammatory drug for cardiovascular inflammation unexpectedly failed in a Phase 3 trial designed to prove a link between inflammation and heart disease, marking a setback for its cardiovascular program and potentially reshaping investor expectations.

via endpoints.news|

#biopharma #business #cardiovascular-inflammation

health2 months ago•4 min saved

Orforglipron tops oral GLP-1 rivals in 52-week diabetes trial

A 52-week Phase 3 trial in 1,698 adults with type 2 diabetes found that Eli Lilly’s orforglipron, an oral small‑molecule GLP-1 drug, reduced HbA1c by about 1.71–1.91 percentage points and produced greater weight loss (6.1–8.2 kg) compared with oral semaglutide (1.47 HbA1c reduction and ~5.3 kg weight loss). However, tolerability favored semaglutide, with around 59% reporting GI side effects on orforglipron versus 37–45% on semaglutide and higher discontinuation (~10% vs ~4–5%). Orforglipron’s small‑molecule, no‑refrigeration profile could improve access in lower‑income countries, but there’s no head‑to‑head with injectable GLP‑1s yet and long‑term adherence remains to be seen.

via ScienceDaily|

#glp-1 #hba1c #health

health3 months ago•3 min saved

RAS-targeting drug daraxonrasib nearly doubles median survival in pancreatic cancer trial

A phase 3 trial published in the New England Journal of Medicine finds the oral RAS-inhibitor daraxonrasib significantly improves survival for metastatic pancreatic cancer, with a median of 13.2 months versus 6.7 months with chemotherapy; the FDA has granted an early-access program ahead of potential approval later in 2026, marking a potential paradigm shift by directly targeting mutant RAS to halt cancer growth.

via USA Today|

#daraxonrasib #health #pancreatic-cancer

health3 months ago•10 min saved

First-in-class KRAS therapy boosts survival in metastatic pancreatic cancer

A new oral drug, daraxonrasib, targets the KRAS-driven mechanism in pancreatic cancer by engaging cyclophilin A to shut down KRAS signaling. In a Phase 3 trial of 500 patients with metastatic pancreatic cancer who had prior treatment, daraxonrasib nearly doubled median survival from 6.7 to 13.2 months and reduced the risk of death by about 60% versus standard chemotherapy. The most common side effects were a skin rash, stomatitis, diarrhea, nausea and vomiting, though discontinuation due to adverse effects was lower and quality of life improved. Regulatory review by the FDA and other global authorities is the immediate next step, with expedited review likely; if approved, the drug could reach clinics within months and herald a shift toward targeted, personalized therapy and potential combination approaches to prevent resistance.

via The Conversation|

#daraxonrasib #health #kras

health3 months ago•7 min saved

Nightly Pill Could Redefine Sleep Apnea Treatment

A six-month phase 3 study of AD109 (an oral combo of aroxybutynin and atomoxetine) in 646 adults with mild–severe obstructive sleep apnea showed a ~44% drop in the apnea-hypopnea index versus an 18% reduction with placebo, with improved oxygen metrics and about 40% moving to a lower OSA severity category and 18% achieving complete control; AD109 targets neuromuscular pathways to keep throat muscles active, offering an alternative for CPAP-intolerant patients. Safety was mostly mild; 21% discontinued due to side effects. The drug has FDA Fast Track status and an NDA is with the FDA, with potential action in early 2027.

via SciTechDaily|

#ad109 #cpap #fda-fast-track

health4 months ago•5 min saved

Oral Sleep Apnea Pill AD109 Nears FDA Sign-Off After Phase 3 Win

A phase 3 trial of the oral drug AD109 (a combo of aroxybutynin and atomoxetine) reduced the apnea-hypopnea index by about 44% versus 18% with placebo over 26 weeks in 646 patients with mild–severe obstructive sleep apnea, with roughly 42% moving to a lower severity category and about 18% no longer meeting OSA criteria. Side effects were mild (dry mouth, nausea, insomnia). The FDA has fast-tracked AD109, with a decision anticipated in 2027, potentially offering a convenient alternative to CPAP for patients unable or unwilling to use the machine.

via ScienceAlert|

#ad109 #cpap-alternative #fda-fast-track