Somatropin (Pfizer)

随着生物技术的飞速发展和人们对健康需求的日益增长，生长激素类药品市场近年来呈现出蓬勃发展的态势。为何发展快？“长高焦虑”成了商机？首先就身高而言，以下是1960年代至2010年代中国男孩和女孩的平均身高变化趋势表根据遗传、营养、医疗及社会环境的变化及可推断以下趋势：（以上数据主要来源：新浪新闻、小花生网（营养篇）、预高儿科、参考文献《婴儿和青春期前儿童的正常生长模式》作者：Julieana Nichols, MD, MPH）等）身高变化/差异的主要影响因素总结（以上数据主要来源：新浪新闻、小花生网（营养篇）、预高儿科、参考文献《婴儿和青春期前儿童的正常生长模式》作者：Julieana Nichols, MD, MPH）等）以上数据显示中国男孩和女孩的平均身高在过去几十年中稳步增长，预计未来婴幼儿身高将继续增长，但增速可能放缓。遗传、营养、环境、医疗保健、体育锻炼和睡眠质量是影响身高的主要因素。20世纪90年代末至21世纪初生长激素逐渐开始被广泛应用和普及的。随着医学技术的发展和对生长激素研究的深入，其在儿童身高的应用也逐渐被认可和推广。1生长激素类药品市场概况生长激素类药品市场规模持续扩大。根据最新市场研究报告，全球生长激素类药品市场在2022年已达到数十亿美元规模，并预计在未来五年内保持年均8%以上的增长率，到2028年市场规模可能超过150亿美元。这一增长主要得益于生物技术的不断进步、适应症的拓展以及患者对生长激素治疗认知度的提高。(图片来源于GMI)在市场结构方面，目前，市面上的生长激素主要有三大类别，分别是需每天注射一次的重组人生长激素注射用冻干粉针剂（粉剂）和重组人生长激素注射液（水剂），以及需每周注射一次的聚乙二醇重组人生长激素注射液（长效剂）。其中，重组人生长激素占据市场主导地位，广泛应用于儿童生长激素缺乏症、特发性矮小症等疾病的治疗。推动市场增长的关键因素包括：人口老龄化趋势加剧，导致与生长激素相关的代谢性疾病发病率上升；生活水平提高和健康意识增强，促使更多患者寻求生长激素治疗；此外，制药企业持续加大研发投入，不断推出新型长效制剂和更便捷的给药方式，也极大地提升了市场接受度。2市场需求分析生长激素类药物的发展是医学进步与市场利益共同作用的结果。亚太地区是生长激素市场的主要消费地区，占据了全球市场份额的40%以上。在应用领域方面，医疗领域是生长激素市场的主要消费领域，占据了市场份额的60%以上。生长激素类药品的市场需求主要来源于多个方面，包括儿童生长激素缺乏症、成人生长激素缺乏症、特发性矮小症、Turner综合征等。儿童生长激素缺乏症是生长激素类药品的主要应用领域，随着家长对孩子身高和发育的重视程度不断提高，这一领域的市场需求持续增长。成人生长激素缺乏症则主要与老龄化社会相关，随着全球人口老龄化趋势加剧，这一领域的市场潜力巨大。患者群体特征方面，儿童患者主要集中在5-15岁年龄段，家长对治疗效果和 全性有较高要求；成人患者则以40岁以上人群为主，更关注生活质量的改善和代谢功能的提升。不同患者群体的需求差异明显，儿童患者更注重身高增长和发育改善，而成人患者则更关注体成分改善、骨密度增加和心血管健康。市场需求的区域差异也十分显著。在发达国家，如北美和欧洲，由于医疗水平较高和医保覆盖较广，生长激素类药品的使用较为普及。而在发展中国家，如中国和印度，经济增长和医疗水平提升正在带动市场快速增长。特别是在中国，随着二孩政策的实施和家长对孩子身高重视程度的提高，儿童生长激素缺乏症的治疗需求呈现爆发式增长。3主要代表类生长激素药品分析在生长激素类药品市场中，有几个主要代表产品，包括诺和诺德Norditropin、辉瑞的Genotropin、礼来的Humatrope、维昇药业（VISEN Pharmaceuticals）的Skytrofa、诺华的Omnitrope以及长春金赛药业的重组人生长激素注射液。这些产品在市场上占据重要地位，各有其独特的优势和劣势。以下是全球生长激素市场主要企业的市场份额、代表药物及特点的整理（数据截止2024年）：备注：以上市场份额数据为大致估算，具体数据可能会因不同研究机构和市场调研报告而有所差异。生长激素类药品的优劣势比较：从疗效方面来看，各主要产品的临床数据均显示出良好的治疗效果。Norditropin和Genotropin在长期临床使用中积累了大量的有效性数据，特别是在儿童生长激素缺乏症治疗方面表现出色。Humatrope则在成人生长激素缺乏症治疗领域有较多研究支持。金赛药业的产品在国内临床应用中表现出与进口产品相当的效果，且在某些适应症上具有独特优势。安全性方面，所有主要产品都经过严格的临床试验和长期使用验证，总体安全性良好。然而，不同产品在不良反应发生率上可能存在细微差异。例如，Norditropin的预充式注射笔设计可能减少注射部位反应的发生且，首次使用后可以保存在冰箱外部长达21天。而Genotropin的双腔注射器设计则降低了药物污染的风险。金赛药业的产品在国内大规模使用中表现出良好的安全性，但在国际市场上的长期安全性数据仍需进一步积累。价格和可及性方面，进口产品通常价格较高，可能限制其在一些发展中国家的使用。相比之下，金赛药业的产品具有明显的价格优势，且受益于本土化生产，供应稳定，在国内市场的可及性较高。然而，在国际市场上，金赛药业的产品仍需面对激烈的价格竞争和复杂的市场准入壁垒。4竞争格局与趋势分析以下是全球主要生长激素生产企业及其代表药物的详细分析（数据截至2024年），结合产品类型、优势与竞争力综合整理：01技术路径差异• 长效剂型：长春金赛主导国内市场，其长效剂型技术领先，2024年长效产品收入占比提升至28%。• 维昇药业等新兴企业通过引进海外技术（如Ascendis Pharma的隆培促生长素）打破原有格局，推动市场增长。• 长春金赛（聚乙二醇修饰）与维昇药业（TransCon技术）形成技术竞争，后者因保留原型药物结构被视为潜在“Best-in-Class”。• 技术进步让用药更方便：以前每天打针，现在每周打一针就行，甚至未来可能出无痛贴片。02中国市场特殊性• 长春金赛凭借政策壁垒（唯一长效上市企业）和渠道优势主导市场，但面临集采降价压力（粉针/水针价格降幅超50%）。• 维昇药业通过差异化技术切入，预计打破长效市场垄断，2024年市场份额约3%。03剂型趋势长效剂型因依从性优势（每周注射一次）成为未来发展方向，短效水针仍占主流（全球60%以上），水针因便捷性逐步替代粉针，但价格敏感性市场仍依赖粉针（如辉瑞Genotropin）。04联合疗法生长激素与GLP-1类药物联用（如减重时减少肌肉流失）等新型疗法，提升了其临床价值。05全球市场集中度诺和诺德、辉瑞、礼来等五家跨国药企合计占全球90%份额，呈寡头垄断；中国企业（长春金赛）通过本土化优势跻身全球前十。06未来发展方向• 长效化趋势：全球60%以上市场仍为短效剂型，但长效产品因依从性优势加速替代，预计2030年中国长效市场规模达286亿元。• 适应症拓展：企业通过开发成人GHD、烧伤修复等新适应症扩大市场，如金赛药业布局肿瘤和代谢疾病领域。• 技术竞争升级：TransCon、聚乙二醇修饰等技术路径的临床效果与安全性对比将决定市场格局。— 总结与展望 —生长激素类药品市场未来仍具有广阔的发展前景。从市场需求来看，随着人们对身高和健康管理的重视程度不断提高，生长激素类药品的应用范围将持续扩大。特别是在中国等新兴市场，经济增长和医疗水平提升将带动更多患者接受治疗。新兴应用领域为市场增长提供了新的动力。除了传统的生长激素缺乏症治疗外，生长激素在抗衰老、运动损伤修复、代谢性疾病治疗等领域的应用潜力正在被逐步发掘。例如，有研究表明生长激素可能对阿尔茨海默病、骨质疏松等老年性疾病具有改善作用，这为产品线延伸提供了新的方向。技术进步将推动市场格局的变革。基因工程技术、纳米技术等新兴技术的应用，有望开发出更安全、更有效的新型生长激素制剂。同时，人工智能和大数据技术的引入，将提高药物研发效率和个性化治疗水平。此外，新型给药方式如口服制剂、透皮贴剂等的研发成功，将极大改善患者用药体验，提高治疗依从性。生长激素的使用需要在专业医生的指导下进行，因为它涉及严格的适应症和使用规范。此外，生长激素的治疗费用相对较高，这也限制了其在一些地区和家庭中的普及程度。近年来，随着人们对儿童生长发育的关注度提高，以及医疗技术的不断进步，生长激素在儿童身高的应用得到了更广泛的认可和应用。同时，相关的医疗保障政策也在逐步完善，以帮助更多需要治疗的儿童获得有效的身高管理。需要注意的是，生长激素并不是适用于所有儿童身高的问题，其使用需要经过详细的医学评估和诊断。因此，在考虑使用生长激素助力儿童身高时，应遵循专业医生的建议，并结合具体情况做出决策。避免家长对身高“内卷”导致非医学需求的滥用，未来需加强监管，平衡医疗需求与商业扩张。参考来源：https://www.gminsights.com/zh/industry-analysis/growth-harmone-markethttps://www.askci.com/reports/20200611/1730168107883641.shtmlhttps://www.genscigroup.com/https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/020280s090lbl.pdfhttps://www.dxy.cn/bbs/newweb/pc/post/25795192

Conference call begins at 4:30 p.m. Eastern time todayMIAMI, Nov. 07, 2024 (GLOBE NEWSWIRE) -- OPKO Health, Inc. (NASDAQ: OPK) reports business highlights and financial results for the three and nine months ended September 30, 2024. Highlights from the third quarter of 2024 and recent weeks include the following: Completed sale of select assets of BioReference Health to Labcorp for $237.5 million. OPKO completed the sale of BioReference Health’s laboratory testing businesses focused on clinical diagnostics and women’s health, excluding operations in New York and New Jersey, for $237.5 million. BioReference Health will continue to offer oncology and urology diagnostic services nationwide, as well as maintain its full operations in New York and New Jersey. This transaction streamlines BioReference Health’s laboratory services business while retaining its core operations to better position the division for sustained growth and profitability. Net sales from operations continuing at BioReference Health exceeded $400 million in 2023.Entered into a $250 million note purchase agreement with HealthCare Royalty secured by profit share payments related to NGENLA. Under the terms of the note purchase agreement, in the near term OPKO retains a significant portion of the profit share payments from Pfizer received pursuant to its license agreement relating to NGENLA with upside over the long term, as well as the full $100 million of remaining potential milestone payments.OPKO’s Board of Directors authorized a $100 million share repurchase program. Under the program, OPKO may repurchase shares of its common stock from time to time through open-market purchases, block trades, privately negotiated transactions, accelerated share repurchase transactions and/or pursuant to Rule 10b5-1 plans, in compliance with applicable securities laws and other legal requirements. The Company repurchased and retired 14.9 million shares for approximately $23.8 million during the three months ended September 30, 2024, and thereafter, through November 6, 2024 repurchased and retired an additional 8.7 million shares for approximately $13.0 million. In addition, the Company repurchased approximately $3.5 million in principal of its Convertible Notes.Enrollment and dosing underway in the MDX2001 Phase 1 trial for the treatment of solid tumor cancers. MDX2001, a tetraspecific antibody, is designed to optimize T-cell function while preventing tumor antigen escape. This Phase 1 open-label trial is expected to enroll up to 45 patients at four clinical trial sites. The Phase 1a portion is primarily designed to evaluate the safety and immunogenicity of ascending doses of MDX2001 in patients with various solid tumors.Awarded $51 million of additional funding under an existing BARDA contract to develop COVID multispecific antibodies and to initiate an influenza program. ModeX Therapeutics was awarded $26.9 million of additional funding under an existing contract with the Biomedical Advanced Research and Development Authority (BARDA). This funding will support the development of a second novel multispecific antibody to SARS-CoV-2 from preclinical through Phase 1 trials, as well as preclinical work on gene-based expression of multispecific antibodies to SARS-CoV-2 including mRNA and/or DNA vectors. In addition, BARDA activated an option for the second phase of funding totaling $24.1 million for ModeX to begin development of influenza multispecifics with gene and/or protein delivery modalities. Together, these funds bring the total support awarded to ModeX to $110 million, with up to $205 million in total, if all options are executed.Announced promising results of an orally delivered oxyntomodulin analog. OPKO is continuing to progress development of its long-acting oxyntomodulin analog in both its subcutaneous and oral formulations. The polyethylene glycol (PEG) linked peptide was studied and progressed to Phase 2 clinical trials before it was suspended due to dose limiting its formulation. The same key peptide as an acylated compound, OPK-88006, has been confirmed in in vitro assays and animal disease models to be a strong candidate for once-weekly subcutaneous administration. Entera and OPKO recently announced results from their ongoing collaborative research combining OPK-88006 and Entera’s proprietary N-Tab™ technology. The program is focused on developing the first oral dual-agonist GLP-1/glucagon peptide as a potential once-daily treatment for patients with obesity and metabolic disorders. In vivo studies in rodent and pig models showed single dose administration, delivered orally, resulted in a desirable PK profile and bioavailability. Third Quarter Financial Results Consolidated: Consolidated total revenues for the third quarter of 2024 were $173.6 million compared with $178.6 million for the comparable period of 2023. Operating income for the third quarter of 2024 was $14.2 million compared with operating loss of $64.4 million for the 2023 quarter. The third quarter of 2024 included a gain of $121.5 million from the sale of certain BioReference assets and a gain of $10.5 million from the sale of shares of GeneDx, as well as non-cash other income of $35.4 million compared with non-cash other expense of $8.3 million in the year-ago quarter related to the change in the fair value of the GeneDx Holdings investment. As a result, net income for the third quarter of 2024 was $24.9 million, or $0.03 per diluted share, compared with net loss of $84.5 million, or $0.11 per share, for the 2023 quarter.Pharmaceuticals: Revenue from products in the third quarter of 2024 was $39.1 million compared with $40.7 million in the third quarter of 2023, reflecting lower Rayaldee sales and foreign currency exchange fluctuations. Revenue from sales of Rayaldee was $5.8 million compared with $7.3 million in the same period in 2023. Revenue from the transfer of intellectual property and other was $13.2 million in the third quarter of 2024 compared with $6.2 million in the 2023 period, primarily attributable to $5.5 million related to the BARDA contract. Gross profit share and royalty payments for NGENLA and Pfizer's Genotropin was $7.0 million in the 2024 quarter compared with $4.9 million in the same period for 2023. Total costs and expenses increased to $84.6 million in the third quarter of 2024 from $72.3 million in the prior-year period primarily due to higher research and development expenses related to increased activity within the ModeX development programs. Operating loss was $32.2 million in the third quarter of 2024, which included $18.0 million of depreciation and amortization expense, compared with $25.4 million in the third quarter of 2023, which included $17.8 million of depreciation and amortization expense.Diagnostics: Revenue from services in the third quarter of 2024 was $121.3 million compared with $131.7 million in the prior-year period, with the decrease primarily due to lower clinical test volume, principally as a result of the Labcorp transaction and a reduction in reimbursement rates. Total costs and expenses, net of the gain on the sale of assets, were $62.7 million in the third quarter of 2024 compared with $160.8 million in the third quarter of 2023. The decrease in costs and expenses were primarily attributable to a $121.5 million gain on the Labcorp transaction, partially offset by severance expense of $26.3 million and costs related to the closure of an office building, which reflect the continued progress with cost-reduction initiatives. The third quarter of 2024 included revenue from services of approximately $19.9 million and total costs and expenses of approximately $31.7 million related to assets acquired by Labcorp. Operating income was $58.5 million in the third quarter of 2024 compared with a loss of $29.1 million in the 2023 period and included $6.1 million and $8.4 million of depreciation and amortization expense, respectively.Cash, cash equivalents, marketable securities and restricted cash: Cash and cash equivalents were $400.1 million, marketable securities, principally shares of GeneDx were $92.2 million and restricted cash and escrow was $43.7 million (including $6.3 million of current restricted cash) as of September 30, 2024. In the third quarter, OPKO entered into a $250 million note purchase agreement secured by OPKO’s profit share payments to be received from Pfizer relating to NGENLA. In addition, OPKO received $237.5 million upon closing of the Labcorp transaction, of which $23.7 million was deposited in an escrow account and will be released upon the one-year anniversary of the transaction close less any outstanding adjustments or claims. Conference Call and Webcast Information OPKO’s senior management will provide a business update, discuss third quarter financial results, provide financial guidance and answer questions during a conference call and live audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call here. Callers who pre-register will receive a unique PIN to gain immediate access to the call and bypass the live operator. Participants may register at any time, including up to and after the call start time. Those unable to pre-register may participate by dialing 833-630-0584 (U.S.) or 412-317-1815 (International). A webcast of the call can also be accessed at OPKO’s Investor Relations page and here. A telephone replay will be available until November 14, 2024, by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 6323665. A webcast replay will be available beginning approximately one hour after the completion of the live conference call here. About OPKO Health OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies. For more information, visit www.opko.com. Cautionary Statement Regarding Forward Looking Statements This press release contains "forward-looking statements," as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA), which statements may be identified by words such as "expects," "plans," "projects," "will," "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding expected financial performance and expectations regarding the market for and sales of our products, whether our product development efforts will be successful and whether the expected benefits of our products will be realized, including whether enrollment in a Phase 1 clinical trial for MDX2001 will be successful and whether the data will be positive, whether efforts to develop a daily oral dual-agonist GLP-1/glucagon peptide will succeed, whether and how many of our shares we will repurchase under a buyback program, whether NGENLA profits will be sufficient to provide long term upside after satisfying our obligations under the note purchase agreement, whether we can successfully progress the development of oxyntomodulin in both subcutaneous and oral formulations, whether the relationship with our commercial and strategic partners will be successful, whether our commercial and strategic partners will be able to commercialize our products and successfully utilize our technologies, our ability to market and sell any of our products in development, whether we will continue to successfully advance products in our pipeline and whether they can be commercialized, whether BioReference will be able to streamline its laboratory services business and better position the division for sustained growth and profitability, whether BioReference’s attempts at returning its core business to profitability will be successful, as well as other non-historical statements about our expectations, beliefs or intentions regarding our business, technologies and products, financial condition, strategies or prospects. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements. These factors include those described in our Annual Reports on Form 10-K filed and to be filed with the Securities and Exchange Commission and under the heading “Risk Factors” in our other filings with the Securities and Exchange Commission, as well as the continuation and success of our relationship with our commercial partners, liquidity issues and the risks inherent in funding, developing and obtaining regulatory approvals of new, commercially-viable and competitive products and treatments. In addition, forward-looking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, among other factors. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligation to update forward-looking statements. We intend that all forward-looking statements be subject to the safe-harbor provisions of the PSLRA. Contacts:Alliance Advisors IRYvonne Briggs, 310-691-7100ybriggs@allianceadvisors.com orBruce Voss, 310-691-7100 bvoss@allianceadvisors.com —Tables to Follow— OPKO Health, Inc. and SubsidiariesCondensed Consolidated Balance Sheets(in millions)Unaudited As of September 30, 2024 December 31, 2023Assets: Cash, cash equivalents, and current restricted cash$406.4 $95.9 Accounts receivable, net 106.6 123.4 Other current assets 116.6 90.2 Total current assets 629.6 309.5 In-process research and development and goodwill 730.9 793.3 Other assets 895.6 908.9 Total Assets$2,256.1 $2,011.7 Liabilities and Equity: Accounts payable$62.7 $69.7 Accrued expenses 122.8 90.1 Current portion of convertible notes 0.2 0.0 Other current liabilities 25.9 40.3 Total current liabilities 211.6 200.1 Long-term portion of convertible notes 178.7 214.3 Senior secured notes 245.4 0.0 Deferred tax liabilities, net 128.4 126.8 Other long-term liabilities, principally leases, and lines of credit 88.6 81.3 Total Liabilities 852.7 622.5 Equity 1,403.4 1,389.2 Total Liabilities and Equity$2,256.1 $2,011.7 OPKO Health, Inc. and SubsidiariesCondensed Consolidated Statements of Operations(in millions, except share and per share data)Unaudited For the three months endedSeptember 30, For the nine months endedSeptember 30, 2024 2023 2024 2023Revenues Revenue from services$121.3 $131.7 $377.5 $391.1 Revenue from products 39.1 40.7 117.7 124.6 Revenue from transfer of intellectual property and other 13.2 6.2 34.3 165.9 Total revenues 173.6 178.6 529.5 681.6 Costs and expenses Cost of service revenues 108.8 106.4 325.8 333.5 Cost of product revenues 24.7 24.5 69.8 74.7 Selling, general and administrative 98.2 72.3 237.2 227.7 Research and development 28.8 19.4 74.8 70.2 Contingent consideration 0.0 (1.1) 0.0 (1.0)Amortization of intangible assets 20.4 21.5 62.3 64.5 Gain on sale of assets (121.5) 0.0 (121.5) 0.0 Total costs and expenses 159.4 243.0 648.4 769.6 Operating income (loss) 14.2 (64.4) (118.9) (88.0)Other income (expense), net 34.2 (14.0) 73.6 (23.8)Income (loss) before income taxes and investment losses 48.4 (78.4) (45.3) (111.8)Income tax provision (23.5) (6.1) (21.9) (10.5)Loss before investment losses 24.9 (84.5) (67.2) (122.3)Loss from investments in investees (0.0) (0.0) (0.0) (0.1)Net income (loss)$24.9 $(84.5) $(67.2) $(122.4)Income (loss) per share, basic$0.04 $(0.11) $(0.10) $(0.16)Income (loss) per share, diluted$0.03 $(0.11) $(0.10) $(0.16)Weighted average common shares outstanding, basic 694,622,466 751,525,007 699,675,944 751,716,692 Weighted average common shares outstanding, diluted 998,363,636 751,525,007 699,675,944 751,716,692