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Nov 09, 2025, 23:00 ET Rona Therapeutics Presents Phase 1 Data for RN0361, a Long-Acting APOC3-Targeting siRNA, at the American Heart Association 2025 Scientific Sessions
DePaoli, MD, CMO. The randomized, placebo-controlled Single Ascending Dose (SAD) study in volunteers with baseline TG >80 mg/dl showed favorable tolerability, no serious adverse events, with mild, self-limited injection-site reactions (ISRs) and transient ALT and AST elevations
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Nov 09, 2025, 23:00 ET Rona Therapeutics Presents Phase 1 Data for RN0361, a Long-Acting APOC3-Targeting siRNA, at the American Heart Association 2025 Scientific Sessions
DePaoli, MD, CMO. The randomized, placebo-controlled Single Ascending Dose (SAD) study in volunteers with baseline TG >80 mg/dl showed favorable tolerability, no serious adverse events, with mild, self-limited injection-site reactions (ISRs) and transient ALT and AST elevations
More news about: Rona Therapeutics
Nov 09, 2025, 20:55 ET Kelun-Biotech Presented TROP2 ADC Sacituzumab Tirumotecan Results From Multiple Clinical Studies at the 2025 CCHIO Congress
sac-TMT 4 mg/kg group and 44 patients to the sac-TMT 5 mg/kg group. At data cutoff (May 21, 2025), median (range) follow-up was 11.7 months in the 4 mg/kg group and 21.8 months in the 5 mg/kg group. Confirmed objective response rate (ORR) was 30.7% and 34.1% in the 4 mg/kg and 5
More news about: Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd.
Nov 09, 2025, 03:00 ET Première thérapie mondiale d'édition génique ciblant l'APOC3 pour le traitement de l'hyperlipidémie
causée par des mutations bialléliques du gène de la lipoprotéine lipase (LPL) ou d'autres gènes régulateurs clés, avec des TG à jeun ≥ 10 mmol/L (885 mg/dL) et une prévalence mondiale de 1 cas sur 100 000-1 000 000 personnes. Le syndrome de chylomicronémie multifactorielle est le résultat d'une interaction
More news about: CorrectSequence Therapeutics
Nov 08, 2025, 10:40 ET Clinically meaningful pivotal study results for olezarsen in sHTG presented as a late breaker at AHA Scientific Sessions
the olezarsen group (pooled 50 mg and 80 mg dose groups). Olezarsen demonstrated a favourable safety and tolerability profile in the CORE and CORE2 studies. Adverse events were balanced across treatment arms (75% olezarsen 50 mg; 76% olezarsen 80 mg; 75% placebo). Serious adverse
More news about: Swedish Orphan Biovitrum AB
Nov 08, 2025, 10:15 ET AMGEN'S REPATHA® CUTS RISK OF FIRST MAJOR ADVERSE CARDIOVASCULAR EVENTS BY 25% IN LANDMARK PHASE 3 VESALIUS-CV TRIAL
attack or stroke, an LDL-C ≥ 90 mg/dL, or non-high-density lipoprotein cholesterol (non-HDL-C) ≥ 120 mg/dL, or apolipoprotein B ≥ 80 mg/dL; and treated with highest tolerated dose of statin and/or ezetimibe. The median baseline LDL-C was 122 mg/dL (IQR, 104-149 mg/dL) on local lab testing. Participants
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Nov 08, 2025, 08:30 ET AusperBio Announces Late-Breaking 48-Week Phase II Data of AHB-137 in Chronic Hepatitis B at AASLD 2025
including complete response (HBsAg < 0.05 IU/mL and HBV DNA < LLOQ) and partial response (HBsAg < 10 IU/mL and HBV DNA < LLOQ). The 300 mg, 24-week regimen showed the highest response rates, with efficacy consistent across baseline HBsAg strata. AHB-137 was well tolerated, and no new safety
More news about: AusperBio Therapeutics Inc.
Nov 08, 2025, 01:42 ET DEADLINE ALERT: Faruqi & Faruqi, LLP Investigates Claims on Behalf of Investors of Jasper
2025, Jasper issued a press release reporting updated data from the BEACON Study. The press release stated that "[r]esults from the 240mg Q8W and the 240mg followed by 180mg Q8W dose cohorts appear to be confounded by an issue with one drug product lot used in those cohorts, with 10 of the 13 patients dosed
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Nov 08, 2025, 01:33 ET DEADLINE ALERT: Faruqi & Faruqi, LLP Investigates Claims on Behalf of Investors of aTyr Pharma
corticosteroid (OCS) dose at week 48, with the OCS dose reducing by an average of 2.79mg for 5.0 mg/kg efzofitimod compared to 3.52 mg for placebo. Complete steroid withdrawal was achieved for 52.6% of patients treated with 5.0 mg/kg efzofitimod versus 40.2% on placebo. After aTyr Pharma
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Nov 07, 2025, 19:29 ET U.S. FDA approves KYGEVVI™ (doxecitine and doxribtimine), the first and only treatment for adults and children living with thymidine kinase 2 deficiency (TK2d)
years).1 The median duration of treatment was 4 years (range: 1 day to 12 years) and the median dose received was 762 mg/kg/day (range: 260 to 800 mg/kg/day).1 The most common adverse reactions (incidence ≥5%) are diarrhea, abdominal pain (including abdominal pain
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Nov 07, 2025, 14:18 ET CANDICE SWANEPOEL, EL ICÓNICO ÁNGEL VICTORIA'S SECRET® LLEGÓ A CIUDAD DE MÉXICO
Geraldine Galván, Paola Wise, Mónica Murillo, Lynn Vizcarra, Mónica Saldivar, Paris Danielle, Frida Sahagún, Maria Riestra, Annesy Lozano, Becky Mg, Diana Paulina, Valeria Rincón, Elena Escobar, Andrea Rodríguez, Cami Corona, Pamela Mcbeath, Natalya Galilea, America Planter and Marietta Rojas.
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Nov 07, 2025, 09:00 ET HanchorBio Presents Two Late-Breaking Abstracts at the Society for Immunotherapy of Cancer 2025 Demonstrating Best-in-Class Potential of HCB101 in Advanced Cancers
ongoing Phase 1a dose-escalation, no dose-limiting toxicities (DLTs) have been observed at doses up to 24 mg/kg. CD47 receptor occupancy ≥99% achieved at doses ≥5 mg/kg, with dose-proportional pharmacokinetics. Confirmed partial responses seen in head
More news about: HanchorBio Inc.
Nov 07, 2025, 09:00 ET CREATE Medicines Announces Positive First-in-Human Results for MT-302
were no events of grade 3 or higher CRS. Maximum tolerated dose: 0.10 mg/kg without steroid premedication. One Grade 4 ICANS event was observed at the highest dose tested (0.15 mg/kg). Pharmacokinetics: terminal half-life of pegylated and ionizable lipids
More news about: CREATE Medicines, Inc.
Nov 07, 2025, 08:52 ET Weltweit erste, auf APOC3 abzielende Gen-Editing-Therapie zur Behandlung von Hyperlipidämie
Mutationen im Lipoproteinlipase (LPL)-Gen oder anderen wichtigen regulatorischen Genen verursacht wird, mit einem Nüchtern-TG-Wert von ≥10 mmol/L (885 mg/dL) und einer globalen Prävalenz von 1 zu 100.000–1.000.000. MCS ist das Ergebnis eines komplexen Zusammenspiels von genetischen, lebensstilbedingten
More news about: CorrectSequence Therapeutics
Nov 07, 2025, 08:31 ET MetaVia Presents Positive New Phase 2a Data on Vanoglipel (DA-1241) in Patients with Presumed MASH at the AASLD The Liver Meeting® 2025
MASH and qualifying baseline alanine transaminase (ALT) and imaging analyses were randomized to receive placebo, vanoglipel 50 mg, vanoglipel 100 mg alone, or vanoglipel 100 mg with a DPP4 inhibitor once daily for 16 weeks in a 2:1:2:2 ratio. "The encouraging additional Phase 2a
More news about: MetaVia Inc.
Nov 07, 2025, 08:30 ET 騰盛博藥宣佈在《自然-醫學》發表ENSURE II期研究結果
IU/L）被分類為BRII-179抗-HBs應答者或無應答者，隨後在ENSURE研究中接受48周的elebsiran（100 mg Q4W）聯合每週PEG-IFNα治療。 該論文的關鍵結果包括： 隊列1-3：在治療結束後24周，在隊列2（elebsiran 200mg + PEG-IFNα）的19名參與者中4例（21.1%）實現了HBsAg清除；在隊列3（elebsiran 100 mg + PEG-IFNα）的18 名參與者中6例（33.3%）實現了HBsAg清除。相比之下，隊列1（PEG-IFNα單藥）18名參與者中僅有1例（5.6%）實現了HBsAg清除。
More news about: 騰盛博藥生物科技有限公司
Nov 07, 2025, 08:30 ET Brii Biosciences Announces Publication of Phase 2 ENSURE Study Results in Nature Medicine
Part I (Cohorts 1-3), participants naïve to BRII-179 were randomized to receive 48 weeks of PEG-IFNα alone or in combination with elebsiran (200 mg or 100 mg administered every 4 weeks [Q4W]). In Part II (Cohort 4), participants previously treated with 9 doses of elebsiran and BRII-179 in a completed
More news about: Brii Biosciences Limited
Nov 07, 2025, 08:24 ET La primera terapia de edición genética del mundo dirigida a APOC3 para la hiperlipidemia
común causado por mutaciones bialélicas en el gen de la lipoproteína lipasa (LPL) u otros genes reguladores clave, con TG en ayunas ≥10 mmol/L (885 mg/dL) y una prevalencia global de 1 en 100.000 a 1.000.000. MCS es el resultado de una interacción compleja de trastornos genéticos, de estilo de
More news about: CorrectSequence Therapeutics
Nov 07, 2025, 08:00 ET PRM Pharma Announces Nationwide Availability of HemiClor® (Chlorthalidone) 12.5 mg: The First FDA-Approved Low-Dose Chlorthalidone Tablet Aligned With Hypertension Guidelines
2025 /PRNewswire/ -- PRM Pharma, LLC announces the nationwide availability of HemiClor® (chlorthalidone) 12.5 mg tablets, the first and only FDA-approved 12.5 mg formulation of chlorthalidone for the treatment of hypertension in adults (
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Nov 07, 2025, 07:00 ET Alebund Presents Phase 3 Trial Results of AP301 at the American Society of Nephrology (ASN) 2025 Congress
difference of -0.02 mmol/L（95% confidence interval (CI): -0.10, 0.06）[-0.06 mg/dL (95% CI: -0.31, 0.20)]; the upper CI bound of 0.06 mmol/L (0.20 mg/dL) was below the pre-defined non-inferiority margin (NIM) of 0.19 mmol/L (0.59 mg/dL). (see Figure 1) At week 27, AP301 maintenance
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Nov 06, 2025, 21:22 ET 世界初の APOC3 を標的とした高脂血症向け遺伝子編集治療薬
mmol/L（885 mg/dL）以上、世界的な有病率は10万人に1人〜100万人に1人とされています。MCSは、遺伝的要因、生活習慣、または代謝性疾患の複雑な相互作用によって発症し、世界的な有病率は最大で600人に1人とされています。 現在のカイロミクロン血症の治療は主に、急性膵炎のリスク閾値（<5.7 mmol/L または 500 mg/dL）未満に空腹時トリグリセリド（TG）値を管理することを目的としています。しかし、既存のトリグリセリド低下薬では十分な効果が得られないことが多く、また超低脂肪食は長期的な継続が困難です。
More news about: CorrectSequence Therapeutics
Nov 06, 2025, 21:00 ET Argo Biopharma to Present Positive Phase I Results of siRNA Therapeutic BW-20805 for HAE at ACAAI 2025
led to rapid, profound, and durable reductions in plasma PKK levels, achieving mean maximum reductions of approximately 75% (50 mg), 87% (150 mg), 94% (300 mg), and 95% (600 mg). PKK suppression was sustained for up to 24 weeks, supporting the potential for 6-month dosing intervals.
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Nov 06, 2025, 19:10 ET ACAAI 2025 | CS2015 (OX40L/TSLP Bispecific Antibody) Makes International Debut
Outstanding accelerated stability under high-temperature stress (40°C); Low viscosity (scored at only 3.7 for a 100 mg/ml solution), promising for subcutaneous injection at high-concentration; Potent efficacy in the intranasal OVA/TSLP-induced
More news about: CStone Pharmaceuticals
Nov 06, 2025, 19:00 ET 里程碑突破！全球首個APOC3基因編輯臨床治療高血脂獲得成功
lipase，LPL）基因或參與調控LPL活性的關鍵基因發生雙等位基因突變，導致脂蛋白脂肪酶功能受損，使甘油三脂的分解代謝產生障礙，從而造成體內乳糜微粒的蓄積，通常患者的空腹TG水準≥10 mmol/L (885 mg/dL)，全球發病率在十萬分之一到百萬分之一之間；而MCS則由多種因素引起的病理狀態，其發病受遺傳背景、生活習慣或其他代謝疾病共同影響，全球發病率高達1/600。 目前，高乳糜微粒血症的主要治療策略是將患者的空腹甘油三脂（TG）水準控制在急性胰腺炎風險閾值以下（通常需<5.7 mmol/L，即 500 m
More news about: CorrectSequence Therapeutics
Nov 06, 2025, 17:07 ET DARZALEX FASPRO® is the first and only treatment approved by the U.S. FDA for patients with high-risk smoldering multiple myeloma
at Week 2 or subsequent infusions. In EQUULEUS, patients receiving combination treatment (n=97) were administered the first 16 mg/kg dose at Week 1 split over two days, ie, 8 mg/kg on Day 1 and Day 2, respectively. The incidence of any grade infusion-related reactions was 42%, with 36% of patients experiencing
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