Monash University

NAARDEN, The Netherlands and MIAMI, Oct. 28, 2024 (GLOBE NEWSWIRE) -- NewAmsterdam Pharma Company N.V. (Nasdaq: NAMS or “NewAmsterdam” or the “Company”), a late-stage, clinical biopharmaceutical company developing oral, non-statin medicines for patients at risk of cardiovascular disease (“CVD”) with elevated low-density lipoprotein cholesterol (“LDL-C”), for whom existing therapies are not sufficiently effective or well-tolerated, today announced that it will present additional safety and efficacy data from the pivotal Phase 3 BROOKLYN study evaluating obicetrapib in patients with Heterozygous Familial Hypercholesterolemia in a late-breaking oral presentation at the 2024 American Heart Association (AHA) Scientific Sessions, taking place November 16 – 18, 2024 in Chicago, Illinois. Additionally, NewAmsterdam announced that company management will participate in the Jefferies London Healthcare Conference, taking place November 19 – 21, 2024 in London. Details are as follows: 2024 AHA Scientific Sessions Presentation Title: Safety and Efficacy of Obicetrapib in Patients with Heterozygous Familial Hypercholesterolemia (BROOKLYN)Session Title: Late-Breaking Science 8: New Targets and New Treatments: Advances in Lipid TherapeuticsPresentation Date and Time: Monday, November 18, 2024, 2:14 p.m. – 2:26 p.m. CST (3:14 p.m. – 3:26 p.m. ET)Presenter: Stephen Nicholls, M.B.B.S., Ph.D., Director, Monash Victorian Heart Institute and Professor of Cardiology, Monash University Jefferies London Healthcare Conference Fireside Chat Date and Time: Thursday, November 21, 2024, at 1:00pm GMTPresenters: Michael Davidson, M.D., Chief Executive Officer, NewAmsterdam Pharma, John Kastelein, M.D., Ph.D., FESC, Chief Scientific Officer, Ian Somaiya, Chief Financial Officer A live webcast of the Jefferies fireside chat will be available through the investor relations section of the NewAmsterdam Pharma website at ir.newamsterdampharma.com. Following the live webcast, an archived replay will be available on the Company’s website. About Obicetrapib Obicetrapib is a novel, oral, low-dose CETP inhibitor that NewAmsterdam is developing to overcome the limitations of current LDL-lowering treatments. In each of the Company’s Phase 2 trials, ROSE2, TULIP, ROSE, and OCEAN, as well as the Company’s Phase 3 BROOKLYN trial, evaluating obicetrapib as monotherapy or combination therapy, the Company observed statistically significant LDL-lowering combined with a side effect profile similar to that of placebo. The Company is conducting an additional Phase 3 pivotal trial BROADWAY, to evaluate obicetrapib as a monotherapy used as an adjunct to maximally tolerated lipid-lowering therapies to provide additional LDL-lowering for CVD patients, and TANDEM, to evaluate obicetrapib and ezetimibe as a fixed-dose combination. The Company began enrolling patients in BROADWAY in January 2022 and in TANDEM in March 2024; completing enrollment of BROADWAY in July 2023, and TANDEM in July 2024. The Company also commenced the Phase 3 PREVAIL cardiovascular outcomes trial in March 2022, which is designed to assess the potential of obicetrapib to reduce occurrences of major adverse cardiovascular events, including cardiovascular death, non-fatal myocardial infarction, non-fatal stroke and non-elective coronary revascularization. NewAmsterdam completed enrollment of PREVAIL in April 2024 and randomized over 9,500 patients. Commercialization rights of obicetrapib in Europe, either as a monotherapy or as part of a fixed dose combination with ezetimibe, for cardiovascular diseases have been exclusively granted to the Menarini Group, an Italy-based, leading international pharmaceutical and diagnostics company. About NewAmsterdam NewAmsterdam Pharma (Nasdaq: NAMS) is a late-stage biopharmaceutical company whose mission is to improve patient care in populations with metabolic diseases where currently approved therapies have not been adequate or well tolerated. We seek to fill a significant unmet need for a safe, well-tolerated and convenient LDL-lowering therapy. In multiple phase 3 studies, NewAmsterdam is investigating obicetrapib, an oral, low-dose and once-daily CETP inhibitor, alone or as a fixed-dose combination with ezetimibe, as LDL-C lowering therapies to be used as an adjunct to statin therapy for patients at risk of CVD with elevated LDL-C, for whom existing therapies are not sufficiently effective or well tolerated. Company Contact Matthew PhilippeP: 1-917-882-7512matthew.philippe@newamsterdampharma.com Media Contact Spectrum Science on behalf of NewAmsterdamBryan BlatsteinP: 1-917-714-2609bblatstein@spectrumscience.com Investor Contact Precision AQ on behalf of NewAmsterdamAustin MurtaghP: 1-212-698-8696austin.murtagh@precisionaq.com

MELBOURNE, Australia, Oct. 28, 2024 (GLOBE NEWSWIRE) -- Aravax, a clinical-stage biotechnology company developing next-generation immunotherapies designed to be safe, convenient, and precisely targeted, today announces it has completed patient recruitment into its Phase 2 study (AVX-201) of its novel, precision therapy PVX108 for the treatment of peanut allergy. Aravax’s PVX108 is an advanced immunotherapy designed to ‘re-train’ the immune system by administering engineered peptides to precisely target T cells and reverse the course of allergic disease. Unlike most treatments available, or under development for peanut allergy, PVX108 does not contain peanut proteins which put patients at significant risk of serious side effects, which leads to complex and burdensome dosing regimens. The study, which is being conducted under an U.S. FDA Investigational New Drug Application, has enrolled 95 patients across eight trial sites in the U.S. and six in Australia. Trial participants were children and adolescents aged four to 17 years with peanut allergy. The trial is a double-blind, placebo-controlled study designed to evaluate the safety and efficacy of two dose levels of PVX108 versus placebo, administered monthly for one year. In Phase 1 trials, researchers observed that PVX108 produced relevant changes in the immune response to peanut protein, which continued to develop once dosing had completed. The primary endpoint of this Phase 2 study is ratio of maximum tolerated dose of peanut protein in a controlled food challenge at the end of the study period relative to baseline. Headline results are expected in H1 2026. Dr Pascal Hickey, CEO of Aravax, said: “The timely completion of recruitment across multiple sites in the U.S. and Australia, is a significant step for Aravax. There remains a critical need for better treatments for serious food allergies. PVX108 has been designed to provide a safe and convenient treatment which has the potential to reverse the course of allergic disease. We look forward to continuing evaluation of PVX108 efficacy in this potentially Phase 3-enabling study.” About Aravax Aravax is a clinical stage biotechnology company focused on revolutionising the treatment of food allergies with next-generation specific immunotherapies which are safe, effective and convenient. Aravax applies proprietary technology and know-how to design highly targeted immunotherapies, which reset the immune system to tolerate a specific allergen without evoking allergic reactions during treatment. The lead product, PVX108, is being developed for the treatment of peanut allergy. Founded in 2015 based on technology from Alfred Health and Monash University, Aravax is a private company headquartered in Melbourne. Its investors include Brandon Capital, Tenmile, Novartis Venture Fund, Breakthrough Victoria, Uniseed, UniSuper and Agati Capital. For more information visit: www.aravax.com.au About PVX108 PVX108 is an innovative precision immunotherapy containing a mixture of synthetic peptides which are designed to precisely target the T cells which drive peanut allergy. Each peptide represents a dominant T cell epitope from major peanut allergens. PVX108 does not contain natural extracts or whole allergens and has been engineered to be incapable of triggering acute allergic reactions. It has been designed to provide a safe, convenient and long-lasting treatment with monthly dosing through essentially needle-free intradermal administration. Media – Australia Kirrily Davis, E: kdavis@bcpvc.com M: +61 (0)401 220228 Media – International Sue Charles, Charles Consultants E: sue.charles@charles-consultants.com M: +44 (0)7968 726585

近日，此前依靠新型精神病药物KarXT获得百时美施贵宝140亿美元天价收购后，从Karuna离职的核心员工现在又有了新动向。 新公司Seaport Therapeutics宣布在B轮融资中获得超额认购的2.25亿美元，而这一切仅仅发生在今年4月份时A轮融资后的6个月内，目前该公司总共已经获得了3.25亿美元的资金，开设了涉及精神疾病的三条管线。 掌舵Seaport的Karuna“老兵” Karuna本身来自于PureTech Health的孵化，而Seaport Therapeutics同样如此。 两位创始人中的Daphne Zohar本人就是PureTech Health的创始人兼前任CEO，可以见得母公司PureTech Health对这一项目的重视。 另外一位创始人兼董事会主席的Steve Paul博士主要贡献体现在技术方面，BMS收购案核心KarXT的诞生就和Steve Paul博士拖不开关系。 Steve Paul博士早先在礼来工作了17年，主要负责神经中枢系统疾病药物的开发，而KarXT复方成分之一的呫诺美林的早期验证，正是他的手笔。然而，实际应用到人体临床中却发现，因为肠道等外周系统中存在毒蕈碱受体，因此遭到激动后会诱发严重的肠胃道副作用，因此研发就此搁置。 而这时PureTech Health合伙人之一的Andrew Miller提出，如果采用只有外周活性的蕈碱受体抑制剂形成复方制剂曲司氯铵就能够正负抵消，让呫诺美林的效力只集中于中枢神经系统，而不会产生严重的副作用。 因此双方一拍即合，便诞生出了Karuna，核心管线KarXT也确实不负所望地推动公司市值增长直至被收购。 “老兵”掌舵的新技术 2017年时，PureTech获得了莫纳什大学Christopher Porter教授的技术Glyph平台授权，Christopher Porter教授的主要研究方向是内源脂质的转运，共同发明人还有Seaport和PureTech公司的成员Dan Bonner和Jamie Simpson。 Glyph平台有点类似于ADC的设计，不过和ADC的设计不同的是，ADC的结构是抗体-Linker-payload，而Glyph平台的结构采用的是甘油三酯-Linker-小分子。 正常的口服药物会通过毛细血管抵达肝脏进行代谢再释放到体循环，这是许多药物离不开肝毒性问题的原因。 而甘油三酯则可以通过肠道吸收进入淋巴系统，最后再在体循环中释放小分子，由于绕开了肝脏系统，让这一平台大大提升了生物利用度，减少了肝毒性。 目前该公司管线中进度最快的候选药物是SPT-300，这是一种别孕烷醇酮的口服前药，目前正在进入针对伴有或不伴有焦虑不安的重度抑郁症的IIb期研究。 采用口服前药自然是需要解决别孕烷醇酮本身的缺点。 别孕烷醇酮（商品名：Zulresso）是一种内源性神经甾体，2019年被FDA批准用于成人产后抑郁，但是治疗过程非常麻烦，需要静脉点滴注射，关键还存在过度镇静和意识丧失的副作用，患者需要住院治疗，严重阻碍了这一药物的应用推广，开发商Sage Therapeutics后来为此与Biogen合作开发了口服形式的Zuranolone，然而，Zuranolone仍然存在肝毒性和肾毒性的担忧，经常需要注意用量以及和其他药物之间存在交叉反应的问题。 Glyph平台正好可以解决肝毒性等问题，因此SPT-300可能具有替代Zulresso甚至Zuranolone的临床潜力。 而有趣的是，Steve Paul博士正好也是开发商Sage Therapeutics联合创始人和董事会成员，他可能将Zulresso的遗憾寄托于SPT-300上。 其他的管线也出于类似的机制，打算采用Glyph平台来实现其他药物本身肝毒性过强等遗憾。 SPT-320所采用的Agomelatine（阿戈美拉汀），Agomelatine就是一款被肝毒性和肾毒性阻碍的抗抑郁药物，长期过量服用就很容易导致肝肾损伤。 总结 总的来看，可以期待Glyph平台在实际使用中产生的效果，这可能使得未来有更多有药物肝毒性遗憾的企业上门合作，目前已在该公司出版物中观察到涉及肥胖症的治疗，表明肥胖赛道也是这家企业未来的布局之一，难怪先后获得了高额融资。 参考来源： https://seaporttx.com/news-papers/