Corticotropin

PRINCETON, N.J., Sept. 26, 2024 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. (ANI or the Company) (Nasdaq: ANIP) today announced that following final approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA), the Company launched Ketoconazole Shampoo, 2%. ANI’s Ketoconazole Shampoo, 2% is the generic version of the reference listed drug (RLD) Nizoral®. "We are proud to announce the FDA approval and commercialization of Ketoconazole Shampoo, 2%, another limited competition product. ANI’s talented R&D team continues to execute on our key strategic priority to consistently and effectively launch new products that address the needs of patients,” stated Nikhil Lalwani, President and Chief Executive Officer of ANI. U.S. annual sales for Ketoconazole Shampoo, 2% total approximately $69.2 million, based on July 2024 moving annual total (MAT) IQVIA data. About ANI Pharmaceuticals, Inc. ANI Pharmaceuticals, Inc. (Nasdaq: ANIP) is a diversified biopharmaceutical company serving patients in need by developing, manufacturing, and marketing high-quality branded and generic prescription pharmaceutical products, including for diseases with high unmet medical need. The Company is focused on delivering sustainable growth by scaling up its Rare Disease business through its lead asset Purified Cortrophin® Gel, strengthening its Generics business with enhanced research and development capabilities, delivering innovation in Established Brands, and leveraging its U.S. based manufacturing footprint. For more information, visit www.anipharmaceuticals.com. Forward-Looking Statements This press release contains not only historical information, but also forward-looking statements made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements represent the Company’s expectations or beliefs concerning future events, including statements regarding the benefits of the acquisition of Alimera Sciences. These forward-looking statements generally are identified by the words “believe,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “continue,” “strategy,” “future,” “opportunity,” “plan,” “may,” “should,” “will,” “shall,” “would” other words of similar meaning, derivations of such words and the use of future dates. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. The following factors, among others, could cause actual results to differ materially from those described in these forward-looking statements: (i) the ability to implement business plans, forecasts, and other expectations in connection with the acquisition and integration of Alimera Sciences, Inc. (“Alimera”) and identify and realize additional opportunities and, in particular, the possibility that the Company is unable to achieve anticipated synergies, (ii) costs and regulatory requirements relating to contract manufacturing arrangements, (iii) costs or delays associated with manufacturing (including the sources and any changes in sources thereof) of the Company’s products, (iv) delays or failures in retaining and obtaining continuing and future product approvals from the FDA, and other regulatory issues relating to the Company's business and products, (v) market trends for the Company’s products, including but not limited to, ILUVIEN, YUTIQ and Cortrophin Gel, and the ability to achieve anticipated sales for such products, (vi) risks that the acquisition of Alimera may disrupt current plans and operations of the Company and potential difficulties of the Company in retaining employees of Alimera and/or maintaining business relationships of Alimera, (vii) the impact of any litigation to which the Company is, or may become, a party, including in connection with the acquisition and integration thereof, (viii) volatility in the Company’s stock price, including as a result of the acquisition, (ix) changes in competitive and regulated industries in which the Company operates, variations in operating performance across competitors, changes in laws and regulations affecting the Company’s business, and changes in the Company’s capital structure as a result of the acquisition, (x) regulatory and other approvals relating to product development and manufacturing, (xi) the Company’s ability, and that of its suppliers, development partners, and manufacturing partners, to comply with laws, regulations and standards that govern or affect the pharmaceutical and biotechnology industries and/or the Company and its products, (xii) costs incurred in connection with the acquisition of Alimera and the possibility that the Company is unable to realize anticipated benefits of the acquisition or to realize estimated pro forma results and underlying assumptions, (xiii) delays in production, increased costs and potential loss of revenues if there is a change in manufacturers or manufacturing processes due to the limited number of suppliers for the Company’s raw materials, active pharmaceutical ingredients, excipients and other materials, (xiv) the Company’s reliance on single source third-party contract manufacturing supply for certain of its key products, including ILUVIEN, YUTIQ and Cortrophin Gel, (xv) changes in policy or actions that may be taken by the FDA, United States Drug Enforcement Administration, and other regulatory agencies, including among other things, drug recalls, regulatory approvals, facility inspections and potential enforcement actions, (xvi) the impact of legislative or regulatory reform on the pricing for the Company’s products, (xvii) the Company’ ability to maintain the services of its key executives and other personnel, and (xviii) general business and economic conditions, such as inflationary pressures, geopolitical conditions including, but not limited to, the conflict between Russia and the Ukraine, the conflict between Israel and Gaza, conflicts related to the attacks on cargo ships in the Red Sea, and the effects and duration of outbreaks of public health emergencies. More detailed information on these and additional factors that could affect the Company’s actual results are described in the Company’s filings with the Securities and Exchange Commission (“SEC”), including its most recent annual report on Form 10-K and quarterly reports on Form 10-Q, as well as other filings with the SEC. All forward-looking statements in this news release speak only as of the date of this news release and are based on the Company’s current beliefs, assumptions, and expectations. The Company undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise. Investor Relations:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Source: ANI Pharmaceuticals, Inc.

来源：药渡 撰文：哥哈骎     编辑：维他命 2024年第四季度，多款备受瞩目的创新疗法将迎来FDA的审批节点，可能为多个难治疾病带来突破性的治疗选择。这些疗法涵盖了从罕见遗传病到肿瘤和内分泌疾病的广泛领域，每一种都有望为患者提供显著的健康改善。 这些药物的批准，不仅对相关疾病的治疗方法可能带来深远影响，也体现了现代药物研发在精准医学、基因治疗和靶向疗法等领域的进步。 随着这些创新药物的上市决定进入最后审批阶段，患者、医疗从业者以及整个行业都对未来几个月的进展充满期待。每一项决定都代表着一个新的里程碑，预示着医学的未来可能更加个性化和精准化。这些即将做出的审批决定，也将为行业提供关键的催化剂，推动未来更多创新疗法的研发和应用。 01 Upstaza 研发企业：PTC Therapeutics 模态：基因疗法 适应症：AADC缺乏症 PDUFA日期：2024年11月13日 获批意义：首款FDA批准的AADC缺乏症药物 Eladocagene exuparvovec（Upstaza，艾哌依卡基）是一种基于腺相关病毒2型（AAV2）的基因治疗药物，能够表达人体芳香族L-氨基酸脱羧酶（AADC，aromatic L-amino acid decarboxylase），用于治疗AADC缺乏症。 AADC缺乏症是一种致命且罕见的遗传性疾病，导致儿童患者出现严重的功能障碍。AADC缺乏症通常会在婴儿出生后的头几个月导致严重的残疾和痛苦，影响生活的各个方面，包括身体、精神和行为。患有AADC缺乏症的儿童可能诱发癫痫样眼球旋转危象发作，导致眼睛在头部向上翻滚、频繁呕吐、行为问题和睡眠困难。受影响儿童的生命受到严重影响及缩短。 AADC缺乏症患者的多巴脱羧酶（DDC）基因发生突变，导致AADC酶的缺乏。DDC基因编码AADC酶，AADC酶负责将L-3,4-二羟基苯丙氨酸（L-DOPA）转化为多巴胺（图1），并将5-羟色氨酸转化为血清素。然而，AADC缺乏症患者体内的突变会导致AADC酶活性的减少或缺失，导致神经递质水平的下降，并导致无法达到发育里程碑。 图1. ADDC催化的3,4-二羟基苯丙氨酸脱羧产生多巴胺的化学过程 Eladocagene exuparvovec通过直接脑部输注至AADC缺乏症患者的大脑（壳核）中，促进AADC酶的生成并增加多巴胺水平。使用eladocagene exuparvovec可以改善AADC缺乏症患者的运动功能发育。 在临床试验中，接受Upstaza治疗的AADC患儿表现出此前未见的运动功能和认知技能进展，如抬头、坐立或站立以及交流能力，这些改善可持续长达十年。在三项临床试验中，所有患儿的运动发育在治疗后早至三个月内均有改善。Eladocagene exuparvovec于2022年7月获得了EMA的批准。在此基因疗法获批之前，AADC缺乏症患者的治疗选择非常有限，主要是通过增加单胺类神经递质的产生，抑制单胺氧化酶（MAO）以减少神经递质的分解代谢，以及缓解症状。 FDA尚未批准任何AADC缺乏症的治疗方案，Upstaza有望成为美国首个该病症的治疗方法。作为一次性治疗，Upstaza有潜力提供持久的疗效，减少持续性症状治疗和干预的需求。FDA已对其BLA授予优先审查资格，并指定2024年11月13日为PDUFA日期。 02 Zanidatamab 研发企业：Jazz Pharmaceuticals 模态：单抗 适应症：胆道癌 PDUFA日期：2024年11月29日 获批意义：首款专门针对胆道癌的HER2靶向疗法 Jazz Pharmaceuticals正寻求zanidatamab在美国的批准，用于治疗既往接受过治疗的、不可切除的、局部晚期或转移性HER2阳性胆道癌（BTC）。BTC包括胆囊癌和肝内胆管癌，占全球成人癌症的不到1%，且预后通常较差。人类表皮生长因子受体2（HER2）是其他癌症抗肿瘤治疗的成熟靶点。在美国、欧洲和日本，每年约有 12000人被诊断出患有HER2+BTC。 Zanidatamab是一种双特异性HER2靶向双特异性抗体，靶向HER2蛋白的两个不同表位（双表位靶向），该蛋白在某些癌细胞表面过度表达。这种独特的设计和增强的结合可产生多种作用机制，包括双重HER2信号阻断、从细胞表面去除HER2蛋白以及免疫介导的细胞毒性，从而促进患者的抗肿瘤活性。 在关键的IIb期HERIZON-BTC-01试验中，zanidatamab取得了令人印象深刻的成果，达到了主要临床终点，其客观缓解率为41.3%。Zanidatamab治疗的中位总生存期为15.5个月。历史数据显示，标准化疗在二线晚期胆道癌患者中的总生存期为6至9个月，因此急需能够改善生存期的靶向疗法。 虽然，目前已有针对FGFR2基因或IDH1基因突变的胆道癌二线患者的靶向疗法获批，但尚无针对HER2过表达患者的靶向疗法。如果获批，zanidatamab将成为首个专门针对胆道癌的HER2靶向疗法。 Zanidatamab由Jazz和百济神州根据Zymeworks的许可协议开发，Zymeworks是zanidatamab的首家开发者。FDA已授予zanidatamab突破性疗法认定，并授予 zanidatamab两项快速通道认定：一项作为难治性BTC的单一药物，另一项与标准治疗化疗联合用于1L胃食管腺癌 (GEA)。 此外，zanidatamab还获得FDA授予的治疗BTC和GEA的孤儿药认定，以及EMA授予的治疗BTC和胃癌的孤儿药认定。Zanidatamab还获得了国家药品监督管理局药品审评中心（CDE）授予的突破性疗法认定。FDA已授予zanidatamab的BLA优先审查资格，PDUFA日期为2024年11月29日。 03 Crinecerfont 研发企业：Neurocrine Biosciences 模态：小分子 适应症：先天性肾上腺皮质增生症（CAH） PDUFA日期：2024年12月29/30日 获批意义：首款CAH的非糖皮质激素疗法 Crinecerfont是一种口服选择性促肾上腺皮质激素释放因子1型受体（CRF1受体）拮抗剂，由Neurocrine Biosciences公司开发，用于治疗经典的21-羟化酶缺乏导致的先天性肾上腺皮质增生症（CAH）。 先天性肾上腺皮质增生症是一种罕见的遗传疾病，会导致酶缺乏，从而改变肾上腺激素的产生，而肾上腺激素是生命所必需的。大约95%的CAH病例是由突变引起的，该突变导致21-羟化酶 (21-OHD) 缺乏。这种酶的严重缺乏会导致肾上腺无法产生皮质醇，大约75%的病例无法产生醛固酮。如果不及时治疗，CAH会导致盐流失、脱水甚至死亡。 Crinecerfont是一种口服选择性促肾上腺皮质激素释放因子1型受体 (CRF1) 拮抗剂，通过不依赖糖皮质激素的机制减少和控制过量的促肾上腺皮质激素 (ACTH) 和肾上腺雄激素，用于治疗因21-羟化酶缺乏引起的CAH。 成人III期数据表明，crinecerfont治疗在第24周时显著减少糖皮质激素剂量，同时维持雄烯二酮水平控制。在2至17岁儿童中，crinecerfont治疗在第4周时显著降低雄烯二酮水平。 Neurocrine Biosciences已提交了crinecerfont胶囊剂型和口服剂型的NDA申请，用于治疗儿童、青少年和成人的CAH。FDA已对两项NDA授予优先审查资格，PDUFA日期分别为2024年12月29日和2024年12月30日。目前，FDA尚未批准任何用于CAH的非糖皮质激素治疗方法。如果获批，crinecerfont将成为70年来首个治疗CAH的新药。 参考文献： 1.PTC Therapeutics Announces FDA Acceptance and Priority Review of the BLA for Upstaza™. PTC Therapeutics Press Release. 14. 05. 2024. 2.Zanidatamab Granted Priority Review for HER2-Positive Metastatic Biliary Tract Cancer. Jazz Pharmaceuticals Press Release. 29. 05. 2024. 3.Neurocrine Biosciences Announces U.S. FDA Accepts New Drug Applications and Grants Priority Review for Crinecerfont for Pediatric and Adult Patients with CAH. PR Newswire. 01. 07. 2024. 专栏作者 哥哈骎 南开大学本科、硕士，德国比勒菲尔德大学博士。多肽化学、多肽API GMP生产专家、欧洲制药公司首席科学家； 拥有Lean Six Sigma黑带认证； 著有专著《Side Reactions in Peptide Synthesis》。 *声明：本文仅是介绍医药疾病领域研究进展或简述研究概况或分享医药相关讯息，并非也不会进行治疗或诊断方案推荐，也不对相关投资构成任何建议。内容如有疏漏，欢迎沟通指出！ 2024生物制药裁员潮：人数激增、肿瘤及神经病学成重灾区…… 恒瑞、石药、科伦老牌药企押注创新，创新药将成增长新引擎 K药抗癌版图再下一城，一线联合治疗恶性胸膜间皮瘤获FDA批准