Aerovate Therapeutics, Inc.

Novartis has terminated a mid-stage trial of an oral treatment candidate for idiopathic pulmonary fibrosis, the company confirmed to Endpoints News . The Swiss pharma giant axed a Phase 2 study of LTP001 last Thursday, according to the federal trials registry. The study cull was cited as a “sponsor decision.” A spokesperson added the company will “continue to evaluate future potential for IPF.” Investigators were testing the experimental medicine against placebo as a once-daily medicine for patients with IPF, in which lung scarring disrupts breathing and can lead to death within a few years of diagnosis. Some patients in the trial also received one of the two standard-of-care medicines, Roche’s Esbriet and Boehringer Ingelheim’s Ofev, both of which were FDA-approved in 2014. Generic versions of Esbriet are available and Roche confirmed in February it was looking at “potential options for Esbriet,” including a potential sale. Multiple other biotechs are working on potential treatments for IPF, including Pliant , PureTech and Avalyn Pharma . Boehringer is planning to send another IPF medicine, the oral drug nerandomilast, to the FDA for regulatory approval based on Phase 3 results last month. LTP001 is also being studied in pulmonary arterial hypertension, or PAH, a rare lung condition that recently received a key approval with Merck’s Winrevair. PAH impacts about 40,000 people in the US and causes high blood pressure in the lungs and heart. In Novartis’ July earnings presentation, PAH was listed as the lead indication for LTP001. Shortly thereafter, the company ended a placebo-controlled Phase 2 and an extension study of the candidate in PAH. On Monday, the spokesperson reiterated the company continues to advance LTP001 in PAH. “There have been no concerning or new safety signals observed to date,” the spokesperson added. LTP001 is an inhibitor of SMURF1, which has been found to be elevated in pulmonary arteries in animal models. Johnson & Johnson received an FDA green light for a single-tablet combination treatment for PAH earlier this year. Aerovate’s treatment candidate recently flunked a mid-stage test, which led to lay offs . Gossamer Bio and Diagonal Therapeutics are among other biotechs exploring this space.

While the Phase II portion of the study evaluating AN2’s epetraborole met the primary endpoint, it failed in a “key” secondary endpoint prompting trial termination. Image Credit: kora_sun / Shutterstock. AN2 Therapeutics has implemented cost-saving measures after “disappointing” topline results from Phase II part of the Phase II/III trial evaluating its lead antibiotic, epetraborole. The antibiotic’s development suffered a major setback a few months ago after AN2 voluntarily stopped enrolment in the Phase III portion of the trial. The decision followed a blinded aggregate data analysis indicating “potentially lower than expected efficacy”. Following the news, AN2’s stock was down by over 22.7% in premarket trading today (9 August), compared to the market close on 8 August. The company’s stock has been floundering since it took a nosedive in February after AN2 announced plans to pause enrolment in the Phase III portion of the trial. AN2 plans to cut 50% of its workforce by the end of the year. The restructuring is expected to cost approximately $2m – $3m. Furthermore, AN2’s chief medical officer, Dr Paul Eckburg, will leave the company by 30 August as part of the restructuring. The cost savings measures are expected to provide AN2 a cash runway through 2027. With the announcement of job cuts, AN2 joins the likes of FibroGen , Aerovate Therapeutics and Allakos , all of which have reduced their headcounts after trial failures this year. See Also: Bavarian Nordic wins positive CHMP opinion to amend Imvanex approval EAN 2024: anti-CGRPs more effective than onabotulinumtoxinA in chronic migraine prevention in real world Epetraborole is a bacterial leucyl-tRNA synthetase inhibitor. AN2 licensed the therapy from Pfizer , who gained it as part of a $5.2bn acquisition of Anacor Pharmaceuticals . AN2 was evaluating epetraborole as a treatment for refractory mycobacterium avium complex (MAC) lung disease. The placebo-controlled Phase II/III trial (NCT05327803) enrolled 80 patients with treatment refractory MAC lung disease. Although the Phase II portion met its primary endpoint of patient-reported response, there was no significant difference between the treatment and placebo arms in a “key” secondary endpoint of sputum culture conversion at six months. Whilst AN2 has shut down the development of epetraborole in MAC lung disease, it plans to start a Phase II trial evaluating the therapy in melioidosis, a bacterial infection. Another therapy in AN2’s pipeline is AN2-502998 for the treatment of Chagas disease , an infectious illness caused by a parasite transmitted by ticks.

1514字6图，阅读大约需要4分钟。 数据新进展：处于1/2期阶段CD20自体CAR-T细胞疗法MB-106用于治疗WM（华氏巨球蛋白血症），披露最新数据，ORR为90%；处于2b期阶段IBAT抑制剂Volixibat用于治疗胆汁酸淤积，包括PBC（原发性胆汁性胆管炎）和PSC（原发性硬化性胆管炎），公布两项2b期VANTAGE研究和VISTAS研究积极中期结果 数据新挫折：处于1/2期阶段WEE1抑制剂Azenosertib用于治疗实体瘤，FDA暂停3项临床研究，ZN-c3-001研究（1期剂量递增）、DENALI研究（2期铂耐药性卵巢癌）和TETON研究（子宫浆液性癌），由于在DENALI研究出现2例疑似因败血症而死亡的案例，第二天反弹26%；处于1b/2期PPAR⍺拮抗剂TPST-1120用于一线治疗HCC（肝细胞癌），更新OS数据，基线不平，OS的HR较之前有所降低且95%CI超1，PFS的HR较大；处于3期阶段CH24H抑制剂soticlestat用于治疗DS（Dravet综合征）和LGS（Lennox-Gastaut综合征），SKYLINE研究（DS)勉强达到主要终点，p=0.06，SKYWAY研究（LGS）未达主要终点；处于2b/3期阶段干粉吸入伊马替尼制剂AV-101用于治疗PAH（肺动脉高压），公布2b期顶线结果，未达PVR（肺血管阻力）主要终点     20240617-20240621 美股龙虎榜     20240617-20240621 美股蛇猫榜 1 Mustang Bio/MBIO 2024年6月17日，Mustang公司宣布处于1/2期阶段CD20自体CAR-T细胞疗法MB-106用于治疗WM（华氏巨球蛋白血症）的最新数据。 ●该队列的ORR为90%，且观察到持久缓解；一名患者在31个月时仍处于CR状态 ●所有患者均接受过大量BTK抑制剂治疗或对其有耐药性，只有一名患者在接受MB-106治疗后开始接受新的抗WM治疗     ●允许门诊治疗，且被认为是可行的 ●目前尚无FDA批准的针对WM的CAR-T疗法 6月20日，折价配股筹集250万美元，下跌34%。 2 GeoVax Labs/GOVX 2024年6月18日，GeoVax宣布处于2b期阶段MVA病毒载体COVID-19疫苗GEO-CM04S1用于预防COVID-19，获BARDA资助2430万美元，可能增至4500万美元，最高达3.43亿美元，6月21日继续涨23%。 3 Sarepta Therapeutics/SRPT 2024年6月20日，Sarepta公司宣布处于商业化阶段AAV基因疗法ELEVIDYS用于治疗DMD（杜氏肌营养不良症），获FDA完全批准门诊患者及加速批准无法行走的患者。 4 Mirum Pharmaceuticals/MIRM 2024年6月17日，Mirum公司宣布处于2b期阶段IBAT抑制剂Volixibat用于治疗胆汁酸淤积，包括PBC（原发性胆汁性胆管炎）和PSC（原发性硬化性胆管炎）的两项2b期VANTAGE研究和VISTAS研究积极中期结果。 ●VANTAGE PBC中期分析显示，瘙痒的主要终点较基线下降3.8个百分点，经安慰剂调整后下降2.3个百分点（p=0.0026）     ●VISTAS PSC中期分析结果超出继续研究的疗效阈值 5 Zentalis Pharmaceuticals/ZNTL 2024年6月18日，Zentalis公司宣布处于1/2期阶段WEE1抑制剂Azenosertib用于治疗实体瘤，FDA暂停3项临床研究，ZN-c3-001研究（1期剂量递增）、DENALI研究（2期铂耐药性卵巢癌）和TETON研究（子宫浆液性癌），由于在DENALI研究出现2例疑似因败血症而死亡的案例，第二天反弹26%。 该公司表示，迄今为止，已有500多名患者接受了azenosertib单药治疗，我们认为我们的数据表明治疗指数良好，可能为面临PROC（铂耐药性卵巢癌）和US（子宫浆液性癌）的女性带来有意义的益处。我们已经完成了DENALI研究第1b组的招募，招募了一百多名患者，进一步证明了我们对azenosertib等新型口服疗法的支持。 6 Longeveron/LGVN 2024年6月17日，Longeveron公司宣布折价配股筹集440万美元，该司核心品种为处于2期阶段同种异体MSCs细胞疗法Lomecel-B用于治疗HLHS（左心发育不全综合征）。 7 Tempest Therapeutics/TPST 2024年6月20日，Tempest公司宣布处于1b/2期PPAR⍺拮抗剂TPST-1120用于一线治疗HCC（肝细胞癌）的OS数据。     ●TPST-1120组的mOS为21个月，对照组为15个月 ●HR为0.65，与10个月之前观察到的0.59相比保持不变 ●生存曲线在早期分离并保持持续分离 ●OS是一线HCC治疗的主要监管终点 基线不平，OS的HR较之前有所降低且95%CI超1，PFS的HR较大。 8 Sonnet BioTherapeutics/SONN 2024年6月20日，Sonnet公司宣布折价配股筹集340万美元，该司核心品种为处于1期阶段IL-12白蛋白结合区融合蛋白SON-1010用于治疗实体瘤。 9 Ovid Therapeutics/OVID 2024年6月17日，Ovid公司宣布处于3期阶段CH24H抑制剂soticlestat用于治疗DS（Dravet综合征）和LGS（Lennox-Gastaut综合征），SKYLINE研究（DS)勉强达到主要终点，p=0.06，SKYWAY研究（LGS）未达主要终点。     10 Aerovate Therapeutics/AVTE 2024年6月17日，Aerovate公司宣布处于2b/3期阶段干粉吸入伊马替尼制剂AV-101用于治疗PAH（肺动脉高压） 2b期顶线结果，未达PVR（肺血管阻力）主要终点。 药渡媒体商务合作 媒体公关 | 新闻&会议发稿 张经理：18600036371（微信同号） 点击下方“药渡“，关注更多精彩内容 免责声明 “药渡”公众号所转载该篇文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请及时告知，我们会在24小时内删除相关信息。 微信公众号的推送规则又双叒叕改啦，如果您不点个“在看”或者没设为"星标"，我们可能就消散在茫茫文海之中~点这里，千万不要错过药渡的最新消息哦！👇👇👇