Hyperion Therapeutics, Inc.

BOSTON and MONTREAL, Dec. 19, 2023 /PRNewswire/ - enGene Holdings Inc. ("enGene,"Nasdaq: ENGN), a clinical-stage genetic medicines company whose lead program is in a pivotal study for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), today announced the appointment of Lota S. Zoth, CPA, as a member of the Board of Directors and Audit Committee Chair. "The addition of Lota to our Board is another milestone in our progression as a public genetic medicines company, and we are fortunate to welcome her counsel and depth of experience as a leader," said Jason D. Hanson, Chief Executive Officer of enGene. "Lota's distinguished history of operational and financial leadership strengthens our Board of Directors, and we look forward to her perspective and insights as we advance our pivotal trial of EG-70 in NMIBC, as well as our mission to mainstream gene therapy for the benefit of underserved patients." Ms. Zoth has more than two decades of experience in senior financial and accounting roles, including as Senior Vice President and Chief Financial Officer of MedImmune, Inc. and Senior Vice President, Controller and Chief Accounting Officer of PSINet, Inc. She also spent nearly a decade with Ernst & Young leading auditing efforts for flagship clients. Ms. Zoth is an experienced Board of Directors member, having served in various roles at other companies, including Board Chair, Lead Independent Director, and Audit Committee Chair, as well as on Compensation, Transaction, Finance and Special Strategic Committees. She is currently serving on the Boards of the publicly traded companies Lumos Pharma, Inovio Pharmaceuticals, and 89 Bio. Her past Board experience includes Zymeworks, Spark Therapeutics, Aeras, Orexigen Therapeutics, Circassia Pharmaceuticals, Hyperion Therapeutics, and Ikaria. "enGene is a truly differentiated company with a compelling vision, promising pipeline, and near-term potential to have a meaningful impact in the lives of patients living with BCG-unresponsive NMIBC," said Ms. Zoth. "I am honored to join the Board of Directors and to serve as Audit Committee Chair to help build a successful future for enGene and patients in need." About enGene enGene is a clinical-stage biotechnology company mainstreaming genetic medicines through the delivery of therapeutics to mucosal tissues and other organs, with the goal of creating new ways to address diseases with high clinical needs. enGene's lead program is EG-70 for patients with non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (Cis) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG), a disease posing a high clinical burden. EG-70 is being evaluated in an ongoing Phase 2 pivotal study. EG-70 was developed using enGene's proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA. enGene became a publicly traded company effective November 1, 2023, upon the completion of a business combination with Forbion European Acquisition Corporation, a special purpose acquisition company. For more information, visit enGene.com. Forward-Looking Statements Some of the statements contained in this press release may constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and "forward-looking information" within the meaning of Canadian securities laws (collectively, "forward-looking statements"). enGene's forward-looking statements include, but are not limited to, statements regarding enGene's expectations, hopes, beliefs, intentions, goals, strategies, forecasts and projections. The words "anticipate", "appear", "approximate", "believe", "continue", "could", "estimate", "expect", "foresee", "intend", "may", "might", "plan", "possible", "potential", "predict", "project", "seek", "should", "would", and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about: enGene's goal of mainstreaming gene therapy, enGene's product pipeline and the near-term potential to have a meaningful impact in the lives of patients living with BCG-unresponsive NMIBC. Many factors, risks, uncertainties and assumptions could cause the Company's actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, the Company's ability to recruit and retain qualified scientific and management personnel, establish clinical trial sites and patient registration for clinical trials and acquire technologies complimentary to, or necessary for, its programs; the Company's ability to address diseases with high clinical needs; the Company's ability to retain commercial rights to EG-70 in the United States and commercialize EG-70 independently, while selectively partnering outside of the United States; the Company's plans and ability to secure regulatory approval, to execute product development, manufacturing process development, preclinical and clinical development efforts successfully and on anticipated timelines; enGene's ability to design mainstream gene therapy and bring genetic medicines to community clinics across the globe; and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission ("SEC") on EDGAR, including those described in the "Risk Factors" section of the Company's Current Report on Form 8-K, filed with the SEC on October 31, 2023 (copies of which may be obtained at sedarplus.ca or ). You should not place undue reliance on any forward-looking statements, which speak only as of the date on which they are made. enGene anticipates that subsequent events and developments will cause enGene's assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. SOURCE enGene

10月6日，安进（Amgen）宣布已以116.50美元/股现金完成对Horizon Therapeutics（简称“Horizon”）的收购，总交易权益价值为 278 亿美元。两家公司原本于2022年12月披露收购消息， 创下当年医药行业最大的收购交易记录，如今历经10个月，终于尘埃落定。来自：安进官网NO.1安进与FTC的博弈时间回到2022年，彼时三大制药巨头强生、安进、赛诺菲均有意收购Horizon。不过在半个月后，赛诺菲和强生两家公司先后退出竞购案，安进成为唯一买家，宣布将以278亿美元的价格将Horizon纳入旗下。不过这桩收购案并未能如约进行。2023年5月，为了阻止安进收购Horizon，美国联邦贸易委员会（FTC）对安进提起诉讼，理由是该并购阻碍Horizon两款新药Tepezza（替普妥单抗，治疗甲状腺眼病）、Krystexxa（聚乙二醇单抗，治疗慢性痛风）的市场竞争。FTC认为，安进可能会利用其现有的产品组合来巩固Horizon治疗甲状腺眼病（TED）和慢性痛风（CRG）药物的垄断地位。对此，安进曾多次发布声明称，它“没有理由，能力或意图”将Tepezza或Krystexxa与其任何产品捆绑在一起进行销售，并承诺不会这样做。而且安进与Horizon的产品针对不同的疾病，不存在重叠。最终于2023年8月，在安进签订多项条款，保证不会在TED和CRG领域形成垄断地位后，FTC才决定撤销诉讼。今年9月1日，FTC与安进达成和解，允许其继续收购Horizon。当然安进不得不答应了FTC提出的一些条件：1.安进不得将其任何产品与Tepezza或Krystexxa捆绑销售，并且在销售Horizon的产品时，安进不得附加任何与安进产品有关的回扣或合同条款作为销售的条件。2.禁止安进在未经FTC许可的情况下，收购任何与TED和CRG有关的产品，生物类似药以及已经过FDA批准试验的还未商业化的产品；不得参与该领域产品的生产或销售。这项持续数月的收购案最终以安进妥协尘埃落定，FTC利用这些强势条款扼杀了安进垄断TED和CRG领域药品的可能性。那么，Horizon究竟是怎样的一家公司，竟引得三家制药巨头青睐？NO.2也曾“买买买”的明星公司HorizonHorizon创立于2005年，由斯坦福大学兼职教授George F Tidmarsh创办。2008年，第二任CEO Tim Walbert上任后，公司进入高速发展阶段，并于2011年成功登陆纳斯达克上市。其专注于开发治疗罕见病、自身免疫性疾病以及严重炎症性疾病的药物，以满足这些疾病领域的迫切需求。在Tim Walbert的执掌下，Horizon开始借助资本加快企业发展。2011年登陆纳斯达克时，IPO募资4950万美元。2014年3月，Horizon斥资6.6亿美元收购了爱尔兰专科药企Vidara Therapeutics；同年10月，Horizon以4500万美元收购NuvoResearch公司在美国的骨关节炎药物Pennsaid（双氯芬酸钠）的经销权；2015年3月，Horizon为了扩充孤儿药组合，斥资11亿美元收购Hyperion Therapeutics；2016年9月，Horizon以8亿美元收购Raptor Pharmaceutical，这是一家罕见病重症药物研发企业。通过此次收购，Horizon充实了自身罕见病药物管线；2021年，Horizon收购了Viela Bio，获得自免药物管线。而最值得一提的还是Horizon通过1.45亿美元收购了River Vision。通过此次收购，Horizon获得了Tepezza这款重磅药物。这是一款全人源化胰岛素样生长因子1受体（IGF-1R）单抗，通过与IGF-1R结合并阻断其激活和信号转导，从而减少炎症并阻止细胞的过度生长。2020年1月，FDA批准Tepezza用于TED的治疗。TED是一种罕见的自免疾病，它是由人体攻击自身过表达IGF-1R的眼眶成纤维细胞，使得眼后的肌肉和脂肪组织发炎，导致眼睛向外凸出。Tepezza是FDA批准的首个治疗TED的药物，上市首年其销售额即超过8亿美元，2022年销售额接近20亿美元。Refinitiv分析预测，2028年Tepezza销售额将达到38.5亿美元。另一款重磅药物Krystexxa由Mountain View Pharmaceuticals授权引进，这是一款用于治疗成人慢性痛风的药物，于2010年获FDA批准上市。2022年7月，FDA又批准Krystexxa的补充生物制品许可申请（sBLA），将进一步拓展Krystexxa的适应症，使其可与甲氨蝶呤共同注射使用，以帮助更多不可控痛风患者达到治疗目的。2022年，Krystexxa的销售额为7.16亿美元。Refinitiv预测，2028年这款药物销售额将达到13.6亿美元。NO.3安进等MNC面临专利悬崖危机此次278亿美元的收购案刷新了安进的历史收购记录。安进之所以对Horizon志在必得，其中很大一个原因是其面对专利悬崖的压力。在2030年之前，安进旗下多款创新药专利即将到期，届时安进将面临收入严重下降的可能。如安进的地舒单抗（Prolia和Xgeva），销售额常年保持在40亿美元以上，2022年销售额为56.42亿美元，同比增长7%，是安进的重磅核心药物。不过该药的专利将于2027年到期，目前已有多家药企在开发其生物类似药。Prolia的“接班人”Evenity（romosozumab）是一款全人源化单抗，通过抑制骨硬化蛋白的活性发挥作用，既能增加骨形成，又能减少骨吸收，从而增加骨密度，目前已获全球37个国家/地区批准，是骨质疏松领域的重要治疗药物，不过其专利将于2029年到期。另外安进的关节炎治疗药物Enbrel(依那西普)，作为全球首款全人源的肿瘤坏死因子（TNF）拮抗剂，已获FDA批准6项适应症，其销售额曾稳定在40亿美元左右，为安进贡献了较大份额的业绩收入，不过其专利也将于2029年到期。因此，安进不得不通过收购，来弥补因重磅产品专利悬崖即将产生的收入降低；以及美国《通胀削减法案》(IRA)价格谈判的潜在风险。收购Horizon，将与安进的传统业务和专长领域的长期管线布局形成协同。据Jefferies分析师预计，Horizon将为安进在2024年到2030年之后，增加20-50亿美元的新产品收入。与安进相比，诺华的情况似乎更不乐观。公开资料显示，3年内，诺华将有超过10款产品面临专利到期。其中包括年销售峰值可能达到50亿美元的抗心衰明星药物Entresto（沙库巴曲缬沙坦钠）、用于ALK+ 非小细胞肺癌（NSCLC）患者的一线治疗药物Zykadia（色瑞替尼）、升高慢性免疫性(特发性)血小板减少性紫癫患者的血小板数量的Promacta（艾曲泊帕），治疗晚期肾细胞癌的Votrient（帕唑帕尼），乙型肝炎药物Tyzeka（替比夫定）等，局势非常紧张。对此，诺华宣布将心血管、血液学、实体瘤、免疫学和神经科学五个治疗领域以及一小部分技术平台作为其研发活动的重点，同时，诺华放弃了10%的研发项目。“宇宙大厂”辉瑞也有近10产品在三年内直面“专利悬崖”，包括治疗乳腺癌的重磅药物Ibrance（palbociclib），治疗类风湿性关节炎等的口服JAK抑制剂Xeljanz（托法替布），治疗成人转甲状腺素蛋白淀粉样变性心肌病的Vyndaqel（氯苯唑酸葡胺）等等。为此，辉瑞加大了对并购资产的布局。今年5月，辉瑞以116亿美元收购了Biohaven来填补内科医学板块；8月，辉瑞以54亿美元收购Global Blood Therapeutics，获得镰刀型红血球疾病治疗药物管线。默沙东也以13.5亿美元的价格收购了Imago BioSciences，来应对其肿瘤免疫疗法Keytruda（帕博利珠单抗）专利即将到期的局面。另外，艾伯维、阿斯利康、杨森、勃林格殷格翰等多款产品都将面临仿制药竞争的局面。不论是原研药企，还是仿制药企，都需要在有限的市场资源争夺战中来调整自己的应对策略。不管是加大研发投入，拓展BD合作，还是延长自己的专利优势，都是药企们保持竞争力的探索，无论哪一条路，都布满了荆棘。参考来源https://www.amgen.com/newsroom/press-releases/2023/10/amgen-completes-acquisition-of-horizon-therapeutics-plc.Amgen/Horizon: swoop on rare disease specialist will push up debt load；The Financial Times.https://www.fiercepharma.com/pharma/amgens-28b-horizon-buyout-cleared-takeoff-after-ftc-states-settle.European Commission Approves EVENITY® (romosozumab) For The Treatment Of Severe Osteoporosis In Postmenopausal Women At High Risk Of Fracture.END👇关注药渡数据媒体矩阵

IRVINE, Calif., April 24, 2023 (GLOBE NEWSWIRE) -- Tarsus Pharmaceuticals, Inc. (NASDAQ: TARS), whose mission is to focus on unmet needs and apply proven science and new technology to revolutionize treatment for patients, starting with eye care, today announced the appointment of Jeff Farrow as Chief Financial Officer and Chief Strategy Officer, effective immediately. Mr. Farrow succeeds Leo Greenstein who served as CFO since 2020 and will be leaving Tarsus to pursue other professional interests. Mr. Farrow is a seasoned finance and corporate strategy executive with more than two decades of experience successfully leading teams in the life science industry, including several publicly traded commercial biotech companies. Most recently, Jeff served as Chief Financial Officer at Global Blood Therapeutics, Inc., where he was an integral part of the leadership team that secured regulatory approval and successfully commercialized Oxbryta for the treatment of sickle cell disease. Prior to that, he held other CFO roles at ZS Pharma and Hyperion Therapeutics, Inc., where he provided strategic and financial leadership for product development and the commercialization of category-creating therapies for disease areas with large unmet needs. “I’m delighted to welcome Jeff to Tarsus and look forward to his leadership as we enter the next stage of growth and transition into a commercial organization,” said Bobak Azamian, M.D., Ph.D., Chief Executive Officer and Chairman of Tarsus. “Jeff’s robust experience in finance, corporate strategy and business development – as well as his proven track record in supporting companies as they evolve into successful commercial enterprises – will help position us to transform into a leading eye care company.” “I’m excited to join Tarsus at this very important and exciting time in the company’s evolution, where we have the opportunity to create and build an entirely new category in eye care and potentially help millions of patients suffering from Demodex blepharitis,” said Mr. Farrow. “I am enthusiastic about joining this extremely talented and motivated team to help take Tarsus to the next level and deliver value to patients and shareholders.” “We are extremely grateful for Leo’s commitment and contributions to Tarsus. With his leadership, Tarsus secured multiple rounds of funding, completed a successful IPO, and executed a significant partnership in China for our lead candidate, TP-03,” added Dr. Azamian. “Leo played an important role in helping Tarsus establish a strong finance team and public company foundation that will serve the organization well as we prepare to commercialize our first therapy. On behalf of the entire company, we extend Leo our well wishes and tremendous success in his future professional endeavors.” About TP-03 TP-03 (lotilaner ophthalmic solution, 0.25%) is a novel, investigational therapeutic designed to resolve the signs of Demodex blepharitis by targeting and eradicating the root cause of the disease – Demodex mite infestation. Lotilaner is a well-characterized anti-parasitic agent that paralyzes and eradicates Demodex mites by selectively inhibiting the GABA-Cl channels. It is a highly lipophilic molecule, which may promote its uptake in the oily sebum of the eye lash follicles where the mites reside. TP-03 was evaluated in two pivotal trials collectively involving more than 800 patients. Both trials met the primary endpoint and all secondary endpoints, with statistical significance and no serious treatment-related adverse events. Both trials also demonstrated that TP-03 was generally safe and well-tolerated. If approved, TP-03 may offer treatment for millions of patients with Demodex blepharitis. TP-03 is now also being studied for the treatment of MGD in patients with Demodex mites. About Demodex Blepharitis Blepharitis is a common lid margin disease that is characterized by eyelid margin inflammation, redness and ocular irritation. Demodex blepharitis is caused by an infestation of Demodex mites, the most common ectoparasite found on humans and accounts for over two-thirds of all blepharitis cases. Demodex blepharitis may affect as many as 25 million Americans based on an extrapolation from the Titan study indicating 58% of patients presenting to U.S. eye care clinics have collarettes, a pathognomonic sign of Demodex infestation, and that at least 45 million people annually visit an eye care clinic. Demodex blepharitis can have a significant clinical burden and negative impact on patients’ daily lives. The Titan study also showed that current management tools, such as tea tree oil and lid wipes, are ineffective at treating Demodex blepharitis. Currently, there are no FDA-approved treatments for Demodex blepharitis. About Tarsus Pharmaceuticals, Inc. Tarsus Pharmaceuticals, Inc. applies proven science and new technology to revolutionize treatment for patients, starting with eye care. Tarsus is advancing its pipeline to address several diseases with high unmet need across a range of therapeutic categories, including eye care, dermatology, and infectious disease prevention. Tarsus is studying three investigational medicines in clinical trials. Its lead product candidate, TP-03, is a novel therapeutic which has demonstrated positive results in two pivotal trials for the treatment of Demodex blepharitis and the New Drug Application for TP-03 has been accepted by the U.S. Food & Drug Administration (FDA) with a PDUFA target action date of August 25, 2023. TP-03 is also being developed for the treatment of Meibomian Gland Disease. In addition, Tarsus is developing TP-04 for the potential treatment of papulopustular rosacea and TP-05, an oral, non-vaccine therapeutic for the prevention of Lyme disease. TP-04 and TP-05 are both currently being studied in Phase 2a clinical trials to evaluate safety, tolerability, and proof-of activity. Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding the potential market size for TP-03, future events and Tarsus’ plans for and the anticipated benefits of its product candidates including TP-03, the timing, anticipated regulatory and development milestones, including the potential commercialization of the Company’s products, and the quotations of Tarsus’ management. The words, without limitation, “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” or “would,” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these or similar identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Further, there are other risks and uncertainties that could cause actual results to differ from those set forth in the forward-looking statement and they are detailed from time to time in the reports Tarsus files with the Securities and Exchange Commission, including Tarsus’ Form 10-K for the year ended December 31, 2022 filed on March 17, 2023 with the SEC, which Tarsus incorporates by reference into this press release, copies of which are posted on its website and are available from Tarsus without charge. However, new risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. Any forward-looking statements contained in this press release are based on the current expectations of Tarsus’ management team and speak only as of the date hereof, and Tarsus specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Contacts: Media Contact: Adrienne Kemp Sr. Director, Corporate Communications (949) 922-0801 akemp@tarsusrx.com Investor Contact: David Nakasone Head of Investor Relations (949) 620-3223 DNakasone@tarsusrx.com