GLP-1R x AMYR

Conference call scheduled for 4:30 p.m. ET today End-of-Phase 2 Meeting for Subcutaneous VK2735 for Obesity Planned for 4Q24 Phase 2 Study of Oral VK2735 in Obesity Expected to Begin 4Q24 Results of Phase 2b VOYAGE Study of VK2809 in NASH/MASH With Fibrosis Selected for Oral Presentation at AASLD Positive Phase 1b Results from VK0214 Study in X-ALD Demonstrate Safety, Tolerability and Reductions in Very Long-Chain Fatty Acids and Plasma Lipids Strong Quarter-End Cash Position of $930 Million SAN DIEGO, Oct. 23, 2024 /PRNewswire/ -- Viking Therapeutics, Inc. ("Viking") (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced its financial results for the third quarter and nine months ended September 30, 2024, and provided an update on its clinical pipeline and other corporate developments. Highlights from the Quarter Ended September 30, 2024, and Other Recent Events: "The first three quarters of 2024 have been a productive period for Viking, with positive results announced from four different clinical trials and promising initial findings reported from a new preclinical program," stated Brian Lian, Ph.D., chief executive officer of Viking. "In Q1, we announced results from the Phase 2 VENTURE trial evaluating subcutaneous VK2735 for obesity, which demonstrated impressive reductions in body weight after 13 weeks of treatment. We also reported the initial results from a Phase 1 trial evaluating an oral formulation of VK2735 in healthy volunteers, which showed encouraging reductions in body weight and excellent tolerability after 28 days of dosing. During the second quarter, the company announced positive 52-week histology results from the Phase 2b VOYAGE trial evaluating VK2809 in patients with NASH/MASH and fibrosis. This study successfully achieved its primary, secondary, and key exploratory endpoints showing reductions in liver fat as well as improvements in NASH/MASH resolution rate and fibrosis. Finally, the company recently announced positive results from a Phase 1b clinical trial of VK0214 in patients with X-linked adrenoleukodystrophy. Results from this study showed VK0214 to be safe and well-tolerated following once-daily dosing over the 28-day study period. In addition, significant reductions were observed in plasma levels of very long-chain fatty acids and other lipids. Moving forward, with $930 million in cash and equivalents, we believe we have the financial resources to achieve multiple important milestones with our clinical programs." Pipeline and Recent Corporate Highlights End-of-Phase 2 Meeting for Subcutaneous VK2735 Planned for 4Q24. VK2735 is a wholly owned dual agonist of the glucagon like peptide-1, or GLP-1 receptor, and the glucose dependent insulinotropic polypeptide, or GIP receptor, for the potential treatment of obesity and other metabolic disorders. During the first quarter of 2024, Viking announced positive top-line results from its Phase 2 VENTURE study in obesity. This study was a randomized, double-blind, placebo-controlled multicenter study designed to evaluate the safety, tolerability, pharmacokinetics, and weight loss efficacy of VK2735, administered subcutaneously, once weekly, for 13 weeks. The VENTURE trial successfully achieved its primary and all secondary endpoints, with patients receiving VK2735 demonstrating statistically significant reductions in body weight compared with placebo. With respect to the primary endpoint, patients receiving VK2735 demonstrated statistically significant reductions in mean body weight from baseline, ranging up to 14.7%, as well as statistically significant reductions in mean body weight relative to placebo, ranging up to 13.1%. Statistically significant differences compared to placebo were observed for all doses starting at Week 1 and were maintained throughout the course of the study. Weight loss in all treated cohorts appeared to be progressive through 13 weeks and did not show evidence of plateauing. The company believes further weight loss could be achieved through extended dosing beyond the 13-week treatment period of this study. VK2735 also demonstrated encouraging safety and tolerability in the VENTURE study, with the majority of observed adverse events (AEs) being reported as mild or moderate. Treatment and study discontinuation rates among VK2735 cohorts were well-balanced compared with placebo. Of gastrointestinal (GI) related AEs, 95% were reported as mild or moderate. Across all cohorts in the VENTURE study, GI-related AEs were most prevalent during the first week of treatment, with observed rates generally declining through the remainder of the study. The results of the VENTURE study will be presented in a poster session on November 3, 2024, at ObesityWeek® 2024, the annual meeting of The Obesity Society, in San Antonio, TX. During the second quarter, Viking received written responses to a U.S. Food and Drug Administration (FDA) Type C meeting packet, submitted to the agency earlier in the quarter. Based on agency feedback, the company plans to advance VK2735 into a Phase 3 program for obesity. The company has scheduled an End-of-Phase 2 meeting with the FDA during the fourth quarter, which will help inform next steps for the Phase 3 program. Initiation of Phase 2 Study of Oral VK2735 in Obesity Expected in 4Q24. In parallel with the development of a subcutaneous formulation of VK2735, Viking is also developing an oral tablet formulation of this compound. The company believes a tablet formulation could represent an attractive treatment option for patients who are hesitant to initiate injection-based therapy, or for those seeking to maintain the weight loss they have already achieved. A key advantage in this regard is the potential to transition patients from the subcutaneous formulation to an oral formulation which utilizes the same molecule. Viking believes this may reduce the risk of unexpected safety or tolerability challenges and could be an attractive option for both patients and clinicians. During the first quarter, Viking reported the initial data from a randomized, double-blind, placebo-controlled Phase 1 trial in healthy adults with a minimum BMI of 30 kg/m2, evaluating once-daily oral doses ranging from 2.5 mg to 40 mg. The primary objective of the study was to evaluate the safety and tolerability of VK2735 administered as an oral tablet once daily for 28 days. The secondary objective was to evaluate the pharmacokinetics of orally administered VK2735 in healthy subjects. Exploratory pharmacodynamic measures included assessments of changes in body weight and other metrics. Oral VK2735 was shown to be safe and well tolerated following once daily dosing for up to 28 days, at dose levels up to 40 mg. Among subjects receiving oral VK2735, all treatment emergent adverse events (TEAEs) were reported as mild or moderate in severity, with the majority, 76%, reported as mild. Similarly, all GI-related AEs in this study were reported as mild or moderate, with the majority, 79%, reported as mild. Mild nausea was reported in 14% of subjects receiving VK2735. No vomiting was reported among subjects receiving VK2735. Diarrhea was reported in 3% of VK2735 treated subjects, compared with 20% of subjects receiving placebo. Overall, no clinically meaningful differences were reported for GI-related AEs among subjects treated with VK2735 compared with placebo. An exploratory assessment of change in body weight showed that subjects receiving oral VK2735 demonstrated dose dependent reductions in body weight, ranging up to approximately 5.3% from baseline. Placebo-adjusted reductions in body weight reached up to approximately 3.3% from baseline. Body weight reductions compared with baseline and placebo were statistically significant at the highest dose evaluated. Weight loss in the 28-day window of this study was progressive at the 20 mg and 40 mg dose levels, with no plateau observed. Given the promising weight loss signal demonstrated after 28 days of once daily dosing, along with the excellent tolerability profile observed at doses of up to 40 mg, further dose-escalation has been evaluated at daily doses of up to 100 mg. Results from this study have been accepted for poster presentation at the upcoming ObesityWeek meeting on November 3, 2024, in San Antonio, TX. Based on observations in the Phase 1 study, Viking believes that further benefits from oral dosing of VK2735 might be anticipated from longer dosing periods. To this end, the company plans to initiate a 13-week Phase 2 trial in patients with obesity in the fourth quarter of this year. Successful Phase 2b VOYAGE Trial in NASH/MASH Reported in 2Q24; Data to be Featured in Oral Late Breaker Presentation at The Liver Meeting 2024. VK2809 is an orally available, small molecule agonist of the thyroid hormone receptor that is selective for liver tissue, as well as the beta isoform of the receptor. VK2809 was evaluated in the Phase 2b VOYAGE study, a randomized, double-blind, placebo-controlled, multicenter, international trial designed to assess the efficacy, safety and tolerability of VK2809 following 52 weeks of dosing in patients with biopsy-confirmed NASH and fibrosis. Enrollment included patients with at least 8% liver fat content as measured by magnetic resonance imaging, proton density fat fraction, as well as F2 and F3 fibrosis. The primary endpoint of the study evaluated the change in liver fat from baseline to Week 12 in patients treated with VK2809 compared to patients receiving placebo. Secondary and exploratory endpoints assessed histologic changes, such as NASH/MASH resolution and fibrosis improvement, following 52 weeks of treatment. In 2023, the company reported that VOYAGE had successfully achieved its primary endpoint, with patients receiving VK2809 demonstrating statistically significant reductions in liver fat content from baseline to Week 12 as compared with placebo. The median relative change from baseline in liver fat among patients treated with VK2809 ranged from 38% to 55% after 12 weeks. In addition, up to 85% of patients receiving VK2809 experienced at least a 30% relative reduction in liver fat. In the second quarter of 2024, Viking announced additional results from the VOYAGE study, demonstrating the successful achievement of the trial's secondary endpoints evaluating histologic changes assessed by hepatic biopsy after 52 weeks of treatment. On the secondary endpoint of NASH/MASH resolution without worsening of fibrosis, VK2809-treated patients demonstrated NASH/MASH resolution rates ranging from 63% to 75%, compared with 29% for placebo. On the secondary endpoint evaluating the proportion of patients demonstrating at least a one-stage improvement in fibrosis with no worsening of NASH/MASH, the proportion of VK2809-treated patients demonstrating improvements in fibrosis ranged from 44% to 57%, compared with 34% for placebo. On the secondary endpoint evaluating the proportion of patients experiencing both the resolution of NASH/MASH and at least a one-stage improvement in fibrosis, the proportion of VK2809-treated patients achieving both measures ranged from 40% to 50%, compared with 20% for placebo. Consistent with prior studies, patients receiving VK2809 in VOYAGE demonstrated statistically significant improvements in plasma lipids. Placebo-adjusted reductions in low-density lipoprotein cholesterol (LDL-C) ranged from 20% to 25%, and reductions in triglycerides and atherogenic proteins such as apolipoprotein B, lipoprotein (a), and apolipoprotein C-III, were also significantly improved relative to placebo. These lipids have been correlated with cardiovascular risk, suggesting that treatment with VK2809 may offer a long-term cardio-protective benefit. VK2809 also demonstrated an encouraging safety and tolerability profile through 52 weeks of treatment, with minimal differences compared with the previously reported results from 12 weeks. The majority, 94%, of treatment related adverse events among patients receiving VK2809 were reported as mild or moderate. Discontinuations due to adverse events were low and balanced across placebo and treatment arms. VK2809 demonstrated excellent GI tolerability through 52 weeks of treatment, with similar rates of nausea, diarrhea, stool frequency, and vomiting among VK2809-treated patients as compared to placebo. The company believes the Phase 2b VOYAGE data demonstrate VK2809's best-in-class efficacy on both NASH/MASH resolution and fibrosis improvement, along with the potential for cardiovascular benefit through improvement in plasma lipids. Results from the VOYAGE study have been selected for oral presentation at The Liver Meeting, the annual meeting of the American Association for the Study of Liver Disease, on November 19, 2024, in San Diego. Positive Phase 1b Results from VK0214 Study in X-ALD Demonstrate Safety, Tolerability and Reductions in Very Long-Chain Fatty Acids and Plasma Lipids. VK0214 is a novel, orally available thyroid hormone receptor beta agonist that is being evaluated as a potential treatment for X-linked adrenoleukodystrophy (X-ALD), a rare neurogenerative disease for which there are currently no pharmacologic treatment options. Subsequent to the end of the third quarter, the company announced positive results from a Phase 1b study evaluating VK0214 in patients with the adrenomyeloneuropathy (AMN), form of X-ALD, which is the most common form of the disorder. The study enrolled patients across three cohorts: placebo, 20 mg daily, and 40 mg daily. The primary objectives were to evaluate the safety and tolerability of VK0214 in subjects with AMN, when administered once-daily over a 28-day dosing period. Secondary objectives included an evaluation of the pharmacokinetics of VK0214 in this population. An exploratory objective was to evaluate the effects of VK0214 on plasma levels of very long-chain fatty acids (VLCFAs) in subjects with AMN. Results from the Phase 1b study showed VK0214 to be safe and well-tolerated following once-daily dosing over 28 days. TEAEs were reported as mild to moderate; one subject in the placebo cohort experienced a wrist fracture that was characterized as a severe adverse event. GI-related adverse events were higher among placebo subjects (33%) compared with VK0214-treated subjects (11%). Treatment with VK0214 resulted in significant reductions in mean plasma VLCFA levels at both doses evaluated, 20 mg/day and 40 mg/day, compared to placebo. Importantly, cohorts receiving VK0214 demonstrated reductions in mean plasma levels of the 26 carbon lysophosphatidyl choline (C26:0-LPC) derivative, a key diagnostic marker. In addition to VLCFA changes, subjects who received VK0214 experienced statistically significant reductions in other plasma lipids. Mean reductions relative to baseline and placebo were observed for LDL-C, apolipoprotein B, and lipoprotein (a) following 28 days of treatment with VK0214. Viking is continuing to evaluate the complete data set, after which it will consider next steps for this program. Novel Amylin Agonist Program Continues to Advance. During the second quarter, Viking presented preclinical data at the American Diabetes Association's (ADA's) annual Scientific Sessions from an internally developed dual amylin and calcitonin receptor agonist program. As the amylin receptor plays an important role in food intake and metabolic control, Viking believes it represents an attractive potential target for therapeutic intervention in obesity. The company's ADA presentation highlighted the effects of treatment on body weight, food intake and metabolic profile in both healthy rats and in diet-induced obese mice. The study demonstrated that Viking's dual amylin and calcitonin receptor agonists reduced food intake in lean rats in the period from 0 – 72 hours following a single subcutaneous dose. At 72 hours following a single dose, Viking's compounds resulted in up to 8% body weight reductions compared to vehicle-treated animals. In a rodent model of diet-induced obesity, treatment with Viking's compounds for 24 days resulted in up to 10% weight loss from baseline. The company believes that these results, as well as those from other preclinical studies, support the continued development of amylin agonists for obesity. Viking expects to file an investigational new drug (IND) application for this program in 2025. Upcoming Scientific Presentations and Investor Events. Viking management will participate in the following upcoming scientific and investor events: Scientific Presentations Results From the 13-Week VENTURE Phase 2a Study of the GLP-1/GIP Co-Agonist VK2735 in Obese Subjects Obesity Week, Poster Presentation San Antonio, TX November 3, 2024 First-in-Human Study of an Oral Formulation of the GLP-1/GIP Co-Agonist VK2735 in Healthy Adults Obesity Week, Poster Presentation San Antonio, TX November 3, 2024 Results from the 52 Week Phase 2b VOYAGE Trial of VK2809 in Patients with Biopsy-Confirmed Non-Alcoholic Steatohepatitis and Fibrosis: A Randomized, Placebo-Controlled Trial The 75th Liver Meeting (AASLD), Late Breaking Oral Presentation 5013 San Diego, CA 11:00 am PT, November 19, 2024 Investor Events Truist Healthcare Conference New York, NY November 7, 2024 UBS Global Healthcare Conference Rancho Palos Verdes, CA November 11 – 14, 2024 Stifel Healthcare Conference New York, NY November 18 – 19, 2024 Jefferies Healthcare Conference London, UK November 19 – 21, 2024 JP Morgan Healthcare Conference San Francisco, CA January 13 – 16, 2025 Third Quarter and Nine Month 2024 Financial Highlights Third Quarter ended September 30, 2024 and 2023 Research and development expenses were $22.8 million for the three months ended September 30, 2024, compared to $18.4 million for the same period in 2023. The increase was primarily due to increased expenses related to manufacturing for the company's drug candidates, stock-based compensation, salaries and benefits, and regulatory services, partially offset by decreased expenses related to preclinical studies and clinical studies. General and administrative expenses were $13.8 million for the three months ended September 30, 2024, compared to $8.9 million for the same period in 2023. The increase was primarily due to increased expenses related to stock-based compensation, legal and patent services, services provided by third-party consultants and insurance. For the three months ended September 30, 2024, Viking reported a net loss of $24.9 million, or $0.22 per share, compared to a net loss of $22.5 million, or $0.23 per share, in the corresponding period in 2023. The increase in net loss for the three months ended September 30, 2024, was primarily due to the increase in research and development expenses and general and administrative expenses, noted previously, partially offset by increased interest income, compared to the same period in 2023. Nine Months Ended September 30, 2024 and 2023 Research and development expenses for the nine months ended September 30, 2024, were $70.7 million compared to $43.3 million for the same period in 2023. The increase was primarily due to increased expenses related to manufacturing for our drug candidates, clinical studies, stock-based compensation, preclinical studies and salaries and benefits. General and administrative expenses for the nine months ended September 30, 2024, were $34.0 million compared to $28.2 million for the same period in 2023. The increase was primarily due to increased expenses related to stock-based compensation, salaries and benefits, services provided by third-party consultants and insurance, partially offset by decreased expenses related to legal and patent services. For the nine months ended September 30, 2024, Viking reported a net loss of $74.5 million, or $0.69 per share, compared to a net loss of $61.3 million, or $0.66 per share, in the corresponding period in 2023. The increase in net loss for the nine months ended September 30, 2024, was primarily due to the increase in research and development expenses and general and administrative expenses, noted previously, partially offset by increased interest income, compared to the same period in 2023. Balance Sheet as of September 30, 2024 At September 30, 2024, Viking held cash, cash equivalents and short-term investments of $930 million, compared to $362 million as of December 31, 2023. Conference Call Management will host a conference call to discuss Viking's third quarter 2024 financial results today at 4:30 pm Eastern. To participate in the conference call, please dial (844) 850-0543 from the U.S. or (412) 317-5199 from outside the U.S. In addition, following the completion of the call, a telephone replay will be accessible until October 30, 2024, by dialing (877) 344-7529 from the U.S. or (412) 317-0088 from outside the U.S. and entering conference ID # 9473630. Those interested in listening to the conference call live via the internet may do so by visiting the Webcasts page of Viking's website at . An archive of the webcast will also be available on the Webcasts page of Viking's website for 30 days. About Viking Therapeutics, Inc. Viking Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel first-in-class or best-in-class therapies for the treatment of metabolic and endocrine disorders, with three compounds currently in clinical trials. Viking's research and development activities leverage its expertise in metabolism to develop innovative therapeutics designed to improve patients' lives. Viking's clinical programs include VK2735, a novel dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors for the potential treatment of various metabolic disorders. Data from a Phase 1 and a Phase 2 trial evaluating VK2735 (dosed subcutaneously) for metabolic disorders demonstrated an encouraging safety and tolerability profile as well as positive signs of clinical benefit. Concurrently, the company is evaluating an oral formulation of VK2735 in a Phase 1 trial. Viking is also developing VK2809, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the treatment of lipid and metabolic disorders. The compound successfully achieved both the primary and secondary endpoints in a recently completed Phase 2b study for the treatment of biopsy-confirmed non-alcoholic steatohepatitis (NASH)/metabolic associated steatohepatitis (MASH) and fibrosis. In a Phase 2a trial for the treatment of non-alcoholic fatty liver disease (NAFLD) and elevated LDL-C, patients who received VK2809 demonstrated statistically significant reductions in LDL-C and liver fat content compared with patients who received placebo. The company's newest program is evaluating a series of internally developed dual amylin and calcitonin receptor agonists (or DACRAs) for the treatment of obesity and other metabolic disorders. In the rare disease space, Viking is developing VK0214, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the potential treatment of X-linked adrenoleukodystrophy (X-ALD). In a Phase 1b clinical trial in patients with the adrenomyeloneuropathy (AMN) form of X-ALD, VK0214 was shown to be safe and well-tolerated, while driving significant reductions in plasma levels of very long-chain fatty acids (VLCFAs) and other lipids, as compared to placebo. For more information about Viking Therapeutics, please visit . Forward-Looking Statements This press release contains forward-looking statements regarding Viking Therapeutics, Inc., under the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995, including statements about Viking's expectations regarding its clinical and preclinical development programs, anticipated timing for reporting clinical data and cash resources. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and adversely and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the success, cost and timing of Viking's product candidate development activities and clinical trials, including those for VK2735, VK0214, VK2809, and the company's other incretin receptor agonists; risks that prior clinical and preclinical results may not be replicated; risks regarding regulatory requirements; and other risks that are described in Viking's most recent periodic reports filed with the Securities and Exchange Commission, including Viking's Annual Report on Form 10-K for the year ended December 31, 2023, and subsequent Quarterly Reports on Form 10-Q, including the risk factors set forth in those filings. These forward-looking statements speak only as of the date hereof. Viking disclaims any obligation to update these forward-looking statements except as required by law. SOURCE Viking Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Metsera, the well-funded obesity biotech from Population Health Partners and ARCH Venture Partners, has unveiled its first clinical data just a few months after emerging with $290 million and a broad pipeline of weight loss drug candidates. The New York City startup said its GLP-1 receptor agonist, a long-acting injectable known as MET-097, was able to reduce some patients’ weight from baseline by 7.5% at day 36 in a Phase 1 study. That measurement came one week after a cohort of patients received their fifth and final dose of 1.2 mg. Looking farther out, four weeks after the final dose was given, Metsera said the cumulative weight loss grew to 8.1% for that group in the 125-person placebo-controlled trial. “We have a feeling that we can be really quite competitive with this,” CEO Clive Meanwell told Endpoints News in an interview Monday night. Doses ranging from 0.16 mg to 1.6 mg were tested in the single-ascending and multiple-ascending dose trials. At 1.6 mg, there was “quite a lot of nausea, vomiting, and would not be suitable for standard of care,” Meanwell said. At the 1.2 mg dose, the CEO said side effects were “very, very similar to the class.” Almost all of the adverse events were mild, with a “few hours at most” of nausea. Less than a handful of patients had diarrhea, he said. The study was conducted in the Kansas City area by the CRO Altasciences. Novo Holdings — the controlling shareholder of one of the market leaders in obesity, Novo Nordisk — owns the contract research company and also runs some early-stage testing for the Danish drug giant, Meanwell said. Metsera’s results are early and will need to be replicated in a larger and longer Phase 2b study, which Meanwell expects to be 13 weeks in duration and could potentially test Metsera’s drug candidate head-to-head against leading obesity medications. The startup is still internally debating which dose to test in the upcoming trial, whether that be 0.8 mg or 1.2 mg, Meanwell said. Other companies have recently reported higher levels of weight loss, albeit with different types of drugs and longer time frames. Zealand Pharma’s GLP-1/GLP-2 receptor dual agonist notched up to 8.3% placebo-adjusted mean weight reduction at 13 weeks, and Roche’s once-daily pill saw up to 7.3% weight loss within one month. In long-term studies for GLP-1 receptor agonists, Meanwell said that “you’ve got to get into high-teens, low-20s.” The company also has an earlier-stage amylin-calcitonin receptor agonist being combined with its GLP-1 that would be “very competitive” if it induces 25% to 30% weight loss, Meanwell said. While Metsera’s far behind the well-entrenched first-movers of Novo Nordisk and Eli Lilly, the company thinks it can carve a lane in the obesity market with a once-monthly option that has a much longer half-life. It said MET-097, which comes from its acquisition of UK startup Zihipp, had a half-life of 380 hours. Novo cut a Phase 1 trial studying a once-monthly GLP-1/GIP agonist due to “portfolio prioritizations,” it said last month. The market leaders, Novo’s Wegovy and Lilly’s Zepbound, are indicated for once-weekly injections. Those behemoths are also testing longer-duration treatment options and have bought up a few obesity startups to get access to muscle-preserving medicines or other mechanisms of action. Novo’s bet on a second iteration of a previously shelved class, CB1, hit a speed bump last week with neuropsychiatric effects that overshadowed efficacy. Meanwell said the goal is also to get rid of the need for a titration schedule that gets patients acclimated to the higher doses. Metsera is still finalizing its Phase 2b trial, which will begin next quarter and will be conducted in the US. The trial is slated to have data by next summer, meaning the startup could quickly start a Phase 3 as soon as the second half of next year, Meanwell said.

全球投资和增长环境充满挑战，但 30 家最大的上市生物制药公司的整体研发效率却有所提高。未来几年，炒作周期会影响这一情况吗？ 2023年初新冠疫情的影响开始减弱，但全球范围内其他危机的持续存在，例如俄乌冲突，以及新出现的紧张局势，如以色列与哈马斯之间的冲突，都表明国际社会经济环境依然充满不确定性和动荡。这些事件不仅对当地国家和地区的稳定构成挑战，也对全球市场、供应链、能源价格以及投资者信心产生深远影响。 在这样的背景下，各国政府和国际组织需要采取有效措施来应对这些危机，包括但不限于促进外交解决方案、加强国际合作、支持受影响地区的经济恢复和人道主义援助。同时，企业和个人也需要灵活应对，通过多元化投资、风险管理和持续关注地缘政治动态来减轻潜在的负面影响。 此外，社会经济环境的不稳定性也凸显了加强全球治理和国际合作的重要性，以构建更加稳定和有韧性的国际体系，应对未来可能出现的类似挑战。 生物科技领域的投资和增长面临高利率等挑战，但2023年XBI指数的下跌6%并未阻止该行业的整体研发效率提升。进入2024年上半年，随着市场预期的普遍复苏和对全年利率下降的预期，XBI指数实现了10%的增长。这表明，尽管短期内市场会受到炒作周期的影响，但长期来看，行业的持续创新和市场的基本面对研发效率的提升起到了关键作用。 全球商业环境面临诸多挑战，制药行业显示出相对较强的抗跌性。2023年，标准普尔500制药业指数保持稳定，并未出现明显波动。进入2024年上半年，该指数录得14%的涨幅，反映出市场对制药行业的信心正在增强。 美国食品药品监督管理局（FDA）对新药的审批活动也显示出行业的活力。尽管2024年迄今为止批准的产品数量为21个，略低于去年同期的23个，但考虑到2023年全年共批准了55个产品，这表明制药行业的研发管线依然活跃，且监管机构对创新药品的审批保持着积极的态度。 这些数据表明，尽管市场环境存在波动，制药行业依靠其持续的创新能力和满足未竟医疗需求的使命，继续展现出增长潜力和市场吸引力。此外，制药行业的稳健表现也可能受益于其在研发上的持续投入、市场需求的稳定性以及监管环境的支持。 前 30 名企业研发生产力有所提高 – 前 10 名与其他企业之间的差距正在扩大 在生物制药领域，30家最大的上市生物制药公司整体研发效率的显著提升，同比增长约35%，这一趋势值得关注。然而，这一增长并非均匀分布在所有公司中，而是主要由前10家公司所推动。这些公司的研发效率与其他公司相比，呈现出明显的领先优势。 具体来说，前10家公司的研发效率与其他公司相比，差距已经扩大到了2.5倍，而在过去的几年中，这一差距通常维持在1.5至2倍之间。这种扩大的差距可能由多种因素驱动： 资源集中：前10家公司可能拥有更多的资源来投入研发，包括资金、人才和设备，这使得它们能够更快速地推进研发项目。 研发策略：这些公司可能采取了更有效的研发策略，例如专注于高潜力的化合物、利用人工智能和机器学习技术来优化药物发现过程。 合作与伙伴关系：前10家公司可能与其他生物制药公司、学术机构或技术公司建立了更紧密的合作关系，加速了研发进程。 风险管理：在研发过程中，这些公司可能更有效地管理风险，通过精准的临床试验设计和数据分析，提高了研发成功率。 市场定位：前10家公司可能在市场定位上更为精准，专注于那些需求迫切且具有较大市场潜力的治疗领域。 政策和法规：这些公司可能更好地适应了政策和法规的变化，例如通过与监管机构的密切沟通，加快了审批流程。 这种差距的扩大对整个行业来说是一个重要的信号，表明研发效率的提升并不总是普遍的，而是可能集中在行业领先者手中。这可能对市场竞争格局、创新药物的供给以及整个行业的健康发展产生深远的影响。对于其他公司而言，了解并学习前10家公司的成功经验，以缩小这一差距，将是提升自身竞争力的关键。 2023年初新冠疫情的影响开始减弱，但全球范围内其他危机的持续存在，例如俄乌冲突，以及新出现的紧张局势，如以色列与哈马斯之间的冲突，都表明国际社会经济环境依然充满不确定性和动荡。这些事件不仅对当地国家和地区的稳定构成挑战，也对全球市场、供应链、能源价格以及投资者信心产生深远影响。 推动这一增长的宏观趋势包括但不限于： 新冠疫情期间疫苗和抗病毒药物的研发经验，为行业提供了宝贵的快速响应和创新模式。 肥胖症治疗领域成为新的增长点，特别是在新药价值（NPV）方面表现突出。 肿瘤治疗领域继续是研发的热点，其中抗体偶联药物、双特异性T细胞衔接器、基于新抗原的mRNA疫苗以及CDK4/6类药物的开发进展，都是推动估值增长的关键因素。 这些趋势表明，尽管短期内市场会受到炒作周期的影响，但长期来看，行业的持续创新和市场的基本面对研发效率的提升起到了关键作用。 在医疗和商业领域，一旦某些产品获得成功，便可能引发行业模仿的潮流。这种趋势背后的群体心理导致众多企业纷纷投入资源，追求相似的目标。以肿瘤免疫检查点抑制剂为例，尽管许多新抗体的商业前景并不清晰，但资金仍在不断涌入。这些企业为了证明其巨额投资的合理性，常常假设其他竞争者会在研发过程中遭遇失败或无法实现市场差异化，而忽视了这种情况实际发生的概率。为了实现正的经济净现值（eNPV），企业需要深入分析市场，评估自身项目的成功率和差异化潜力。 诺和诺德再次位居榜首 在Catenion进行的纵向研究中，诺和诺德公司在2023至2024年间的研发效率和企业表现方面荣获最高荣誉，这一成就并不出人意料。公司在肥胖症治疗领域的深厚积累，正使其成为医药行业焦点。Ozempic、Wegovy和Rybelsus这三款GLP-1激动剂药物的总净现值达到了1810亿美元。与此同时，与司美格鲁肽联用的III期胰淀素受体激动剂CagriSema的组合，其市场价值在一年内增长了一倍多，达到了740亿美元，显示出其在减肥治疗领域的巨大潜力。 诺和诺德的市值已经达到了4500亿美元，这一数值几乎与丹麦的国内生产总值相当。鉴于其庞大的市值，公司在未来几年中必须展现出卓越的业绩增长，以回应投资者对其持续增长的期待。 Vertex 在研发效率方面稳居第二 Vertex Pharmaceuticals Incorporated 在囊性纤维化（CF）治疗领域的领导地位仍然牢固，其75%的估值依赖于该领域的药物销售。公司的主打药物 Trikafta 预计将在2037年前保持市场独占权，但市场焦点可能会逐渐转移到正在接受 FDA 审查的新三联疗法药物 Vanzacaftor 上。与此同时，Vertex 也在积极扩展其产品线，例如 Casgevy，这是首个获批用于治疗镰状细胞病的 CRISPR/Cas9 基因编辑疗法，其开发得益于与 CRISPR Therapeutics 的合作，涉及高达9亿美元的预付款项，目前市场估值约为40亿美元。 Vertex Pharmaceuticals Incorporated 在制药行业的发展势头排名中独占鳌头，其产品线充分展现了公司的研发实力。特别值得注意的是，Vertex 针对 NaV1.8 离子通道的 III 期临床试验的非阿片类止痛药项目，市场估值高达110亿美元，这一药物的研发进展备受业界关注。 在2024年4月，Vertex Pharmaceuticals 宣布了一项重大战略举措，以49亿美元收购 Alpine Immune Sciences Inc.。此次收购的核心目标是获得 povetacicept，一种处于II期临床试验阶段的 BAFF/APRIL 抑制剂，该药物在治疗 IgA 肾病方面展现出了巨大的潜力，被认为是同类中的佼佼者。 Vertex的成功并非一蹴而就，而是通过一系列稳健的增长策略和高盈利能力实现的。公司没有依赖大规模的交易或媒体炒作，而是通过一系列补充性和以产品为中心的交易，逐渐在全球生物制药行业中占据了一席之地。如今，Vertex的市值已经达到了约1250亿美元，其产品组合极为丰富多样，覆盖了从罕见病到大众市场的各种疾病领域，包括神经性疼痛等。这种多样化的产品线不仅为Vertex带来了稳定的增长，也为公司在全球医药市场中的领导地位提供了坚实的基础。 减肥药的成功能否推动礼来达到 10000 亿美元的市值？ 礼来公司，以其在研发生产力和企业表现上的卓越表现位列第三，目前市值高达8170亿美元，有望成为首家市值突破万亿美元大关的制药企业。其主要推动力来自于其创新的肠促胰岛素药物tirzepatide，该药物在治疗糖尿病、肥胖症以及未来可能的适应症方面，预计市场价值超过2000亿美元。此外，礼来的产品组合中，以新型肠促胰岛素为主导的产品线，整体市场估值达到560亿美元。 礼来公司在2023年通过一系列战略性收购，进一步巩固了其在制药行业的领先地位。其中，最引人注目的两笔交易分别是对DICE公司的收购，价值24亿美元，以及对Versanis公司的收购，价值19亿美元。DICE公司的收购，主要是看中了其口服IL17抑制剂以及口服蛋白质-蛋白质相互作用抑制剂的技术平台。而Versanis公司的收购，则完全符合礼来在肥胖症治疗领域的战略布局，旨在解决减肥过程中肌肉质量减少这一尚未得到充分解决的问题。 Bimagrumab是礼来公司开发的一种II期激活素II型受体阻滞剂，其作用机制是通过减少脂肪组织并增加肌肉质量，从而为肥胖症患者提供了一种新的治疗选择。这种药物的开发和应用，进一步证明了礼来在创新药物研发方面的深厚实力和对未来市场需求的精准把握。 尽管肿瘤领域的资产在礼来公司的总投资组合中仅占14%的比重，但其在公司整体战略中依然扮演着关键角色。Verzenio，作为礼来的主要肿瘤资产之一，是一款CDK4/6抑制剂，预计到2030年的销售额将超过90亿美元，显示出其强劲的市场潜力。 在中枢神经系统领域，礼来通过获得用于治疗阿尔茨海默病的淀粉样蛋白抗体donanemab的批准，实现了在该领域的重大突破。尽管donanemab的净现值（NPV）高达80亿美元，但由于公司整体战略的调整和市场的变化，中枢神经系统领域的资产在礼来总价值中的占比已经降至4%。这一变化反映了礼来在不断优化其产品组合，以适应市场和患者需求的动态变化。 Moderna 和 BioNTech – 两种不同的战略 尽管COVID-19疫苗的销售额逐渐减少，Moderna公司和BioNTech公司依然稳居研发效率排行榜的前列，分别位列第四和第六。这两家公司虽然都拥有类似的mRNA技术平台，并成功研发出COVID-19疫苗，但在战略上却有着显著的差异。Moderna公司正在加强mRNA技术在治疗领域的应用，并持续聚焦于传染病领域。通过其针对RSV的mRNA疫苗mRESVIA，Moderna已在该领域获得了第二个获批的mRNA产品。此外，Moderna正在将其mRNA平台扩展至心血管和肿瘤治疗领域，最近公布的数据显示，其与Keytruda联合使用的新型抗原疫苗在治疗转移性黑色素瘤方面取得了积极的效果。 与Moderna的战略不同，BioNTech公司正致力于回归其在肿瘤治疗领域的初心，并利用其在COVID-19疫苗上获得的利润，通过与Genmab A/S、BioTheus和Duality Biologics等公司的合作，将其业务拓展到单克隆抗体(mAb)、双特异性抗体和抗体药物偶联物(ADC)领域。BioNTech公司目前处于III期临床试验的下一代CTLA-4单克隆抗体，有望为PD-(L)1治疗后的患者提供新的治疗方案。此外，VEGFxPD-L1双特异性抗体的研发进展，特别是在Summit和Akeso公司的相关药物在头对头试验中首次超越Keytruda后，可能会成为推动公司中期价值增长的强劲动力。 尽管BioNTech公司在肿瘤治疗领域投入了大量精力，但其最具价值的研发管线资产仍然是与辉瑞公司合作开发的III期流感mRNA疫苗，价值高达17亿美元。然而，两家公司面临的一个关键不确定性是其COVID-19 mRNA疫苗的未来发展：它们当前的市场价值究竟如何？不同分析师对此持有不同看法，这可能成为影响两家公司估值的主要变量，并可能决定它们在未来几年是否能够继续保持在行业前30强的地位。 Moderna 在构建价值链并向投资者展示疫情后的未来展望方面取得了一定的进展，这使得它在本年度的排名中相对 BioNTech 显得更为突出。 Enhertu 和第一三共的 ADC 平台继续获胜 在全球投资和增长环境面临挑战的背景下，全球30家顶尖上市生物制药公司的研发效率却意外地实现了提升。未来几年内，市场的炒作周期可能会对这种趋势产生一定的影响。在这一领域，第一三共和阿斯利康通过合作，借助第一三共的ADC平台和Enhertu的成功，以及双方的互利共赢交易，有望催生出新的超级重磅炸弹级药物——目前正在申请中的针对TROP2的ADC药物，其市场潜力估值超过170亿美元。 第一三共的先进技术平台不仅巩固了其在全球生物制药领域的领先地位，还为与默沙东的另一项潜在高收益的ADC药物合作铺平了道路。尽管遭遇了挫折，其主要产品之一，针对HER3的ADC药物patritumab deruxtecan，因生产问题近期未能获得FDA的批准。面对这一挑战，第一三共已将生产任务委托给外部合作伙伴。尽管如此，FDA的拒绝无疑为公司在日本和德国本土增加ADC生产能力提供了额外的动力。 ADC技术的发展不仅为第一三共和阿斯利康带来了转机，也在肿瘤治疗领域引发了一场变革，将治疗的焦点从免疫肿瘤转移到了靶向肿瘤治疗。近期，强生对Ambrx的收购以及Genmab对普方生物的收购，标志着这一领域并购活动的活跃态势。 这些并购案例反映出ADC药物因其精准的靶向能力和强大的治疗效果，正成为肿瘤治疗领域的研究热点。随着这一领域的不断发展，预计未来将有更多的创新药物和治疗方法涌现，为患者提供更多的治疗选择，同时也为相关企业带来新的增长机会。 阿斯利康正积极投资于具有重大市场潜力的多种创新疗法，包括处于晚期开发阶段的双特异性CTLA4xPD1疗法、针对B7-H4的单克隆抗体（mAb），以及小分子选择性雌激素受体降解剂camizestrant。这些项目均由公司内部研发团队独立开发。 安进和再生元 - 两家重回前十的知名公司 自2019年起，再生元制药公司（Regeneron Pharmaceuticals, Inc.）与安进公司（Amgen, Inc.）虽未位列研发生产力排行榜前十，但它们近期均以显著的成就和业绩强势回归，分别在研发生产力方面位列第8和第9位，在公司绩效方面分别位列第4和第16位。再生元的市值已突破1000亿美元大关，其研发的新型双特异性抗体在肿瘤和免疫治疗领域展现出广阔的应用前景。此外，该公司还成功管理了其超级重磅药物Dupixent和Eylea的产品生命周期，推出了高剂量版本，旨在降低给药频率，为视网膜疾病患者提供潜在的新标准治疗方法，并计划延长Eylea的专利保护期至2027年。 与再生元相似，安进公司的发展势头部分得益于其在双特异性抗体领域的深入研究和投资。其最近获得加速批准的小细胞肺癌治疗药物BiTE Immeltira，便是其研发策略成功的一个例证。此外，安进通过收购Horizon Therapeutics plc（在2022年的排名中位列第五），进一步扩大了其产品线，特别是将重磅药物Tepezza纳入其产品组合中，为其增长提供了新的动力。然而，安进最显著的价值增长点来自于与阿斯利康共同开发的抗TSLP单克隆抗体Tezspire，该药物已经获批用于治疗哮喘，成为公司产品管线中的重要组成部分。 在当前肥胖治疗领域的热潮中，安进和再生元均展现出了强烈的参与意愿，并寻求解决现有重磅药物所未能覆盖的治疗需求。安进的II期研究项目MariTide展现出成为超级重磅药物的潜力，它旨在通过延长给药间隔并优化停药后的减重效果来提升治疗效果。与此同时，再生元在其早期临床研发管线中布局了两种靶向肌肉保护激活素通路的抗体，这可能为肥胖治疗提供新的策略。 随着诺和诺德和礼来在肥胖治疗领域的临床数据不断积累和成熟，安进和再生元作为后来者，他们的差异化竞争策略和潜在的市场表现将引起业界的广泛关注。这两家公司的积极布局和创新方法可能会为肥胖治疗领域带来新的变革，并为患者提供更多的治疗选择。 Jazz Pharmaceuticals – Top 10中的新秀 在最近两个年度的评估中，Jazz Pharmaceuticals plc在中型制药公司的排名中表现卓越，现在它不仅首次跻身全球销售额最高的30家生物制药公司之列，而且还迅速占据了前十名的位置（在研发生产力方面排名第10，在公司绩效方面排名第7）。这一成就标志着Jazz Pharmaceuticals plc在生物制药行业中的地位和影响力有了显著提升。 Jazz Pharmaceuticals plc之所以能够成为最大的生物制药公司之一，并在研发生产力和公司绩效方面获得前十名的排名，主要得益于其治疗嗜睡症的药物Xywav和治疗罕见癫痫的Epidolex在市场上取得的巨大成功。Jazz的战略重点包括通过公司收购和资产交易来实现业务扩张，特别是其对肿瘤治疗领域的长期多元化发展。 Jazz能否保持其在30家最大生物制药公司中的地位，并维持其在前10名的排名，将取决于其持续的研发成果和业务多元化能力。目前，Jazz的产品管线中排名第15位的是zanidatamab，这是一种由Zymeworks, Inc.开发的针对HER2表达肿瘤的双特异性HER2抗体。这表明Jazz需要继续加强其研发力度和产品线，以确保长期保持在行业的领先地位。 中型规模公司排名 - 前五名以抗体和中枢神经系统为主 在我们的第三次年度中型公司（销售额排名31-50位）排名中，榜单发生了一些变化。Jazz Pharmaceuticals plc的上升使其进入了前30名的行列，而之前位于前30名的江苏恒瑞医药则下滑至中型公司排名中。此外，还有几家新公司加入了排名。在排名前5的公司中，有两家得益于其在中枢神经系统（CNS）领域的强势地位（Alnylam Pharmaceuticals Inc.和Neurocrine Biosciences, Inc.），而其他三家公司则通过结合抗体技术与在肿瘤治疗领域的重点来实现其排名（Genmab、百济神州、江苏恒瑞医药）。这些变化反映了生物制药行业中的动态竞争和不断变化的市场地位。 新晋公司Alnylam Pharmaceuticals Inc.在研发生产力排名中一跃成为榜首。作为一家RNA干扰（RNAi）技术平台公司，Alnylam与多个治疗领域的大型制药企业建立了合作关系，并成功推出了数款商业化的RNAi治疗药物，其市场估值持续攀升。例如，与诺华合作开发的前蛋白转化酶枯草杆菌蛋白酶（PCSK9）siRNA药物Leqvio，自去年以来其净现值从17亿美元增长到了49亿美元。此外，Alnylam的Amvuttra，一款用于治疗遗传性转甲状腺素蛋白淀粉样多发性神经病（hATTR-PN）的药物，已经获得批准，并且与赛诺菲合作，在ATTR-CM的III期临床试验中取得了积极成果，目前该药物仍在进一步研发之中。 在中型公司的排名中，百济神州和江苏恒瑞这两家中国企业分别稳居第二和第三名的位置。它们专注于肿瘤治疗领域，并在全球市场上展现出了显著的竞争力。尽管许多中国生物制药公司主要集中于国内市场，但百济神州和江苏恒瑞却以其全球化战略和国际视野，成为了中国企业中的佼佼者。这两家公司通过积极参与国际合作和推动创新药物的全球开发与注册，引领着中国生物制药行业的国际化进程。 百济神州作为新进入者在我们的中型公司研发生产力排名中引起了关注。其PD-1抑制剂Tevimbra的市场潜力估值超过73亿美元，在美国获得批准上市，而其BTK抑制剂Brukinsa的市场潜力估值更是翻倍增至约160亿美元，这两项成果显著推动了百济神州在商业化和研发方面的成功。Brukinsa在针对复发或难治性慢性淋巴细胞白血病的头对头试验中，展现了在无进展生存期和安全性方面优于艾伯维和强生的Imbruvica。 尽管罗氏和吉利德科学/Arcus的TIGIT类药物在临床试验中的失败对百济神州的产品线造成了一定影响，导致诺华去年决定终止与百济神州的共同开发协议，但百济神州依然专注于通过合作来扩大其全球足迹。此外，百济神州在美国建立的新生产基地也进一步扩展了其在该地区的业务。 与所有致力于肿瘤治疗的公司一样，百济神州也在加强自身的ADC连接子平台，并通过与上海映恩生物（DualityBio）达成的协议，扩大了其在ADC领域的开发力度。这些举措体现了百济神州在全球生物制药行业中的竞争力和对创新药物研发的持续承诺。 江苏恒瑞医药股份有限公司在医药行业的排名历史上一直较为靠前，长期位列前30强，并且在多数排名中都稳居前十。不过，本年度由于销售预期的下调，其排名有所下降至中等水平。然而，公司与德国默克公司达成了一项价值1.69亿美元的预付款交易，涉及其研发的PARP1抑制剂和Claudin-18.2 ADC。虽然江苏恒瑞在行业发展趋势上占据领先地位，但其产品组合中尚缺乏高价值的重磅药物。这种情况与其较低的研发投入相辅相成。 Genmab公司在晚期抗体领域的业务虽然从先前的榜首位置下滑至第四位，但其投资组合价值保持稳定，主要得益于其与强生合作的Darzalex（一种针对CD38的抗体药物）的强劲表现，该药物的市场价值预期超过110亿美元。尽管其研发管线中CD3xCD20双特异性抗体epcoritamab（商品名Epkinly）的获批导致其研发动力排名有所下降，但公司通过首次战略性并购，以18亿美元收购了普方生物，这不仅加强了其在抗体药物偶联物（ADC）领域的能力，也扩大了其在妇科和实体瘤治疗领域的市场影响力。这次并购标志着Genmab在拓展其产品线和治疗领域方面迈出了重要一步。 Neurocrine Biosciences, Inc. 作为新晋企业，在排名中位列第五。公司专注于治疗中枢神经系统疾病，其主要的价值来源是Ingrezza，这是一种VMAT2抑制剂，已经获得批准用于治疗迟发性运动障碍和亨廷顿舞蹈症，市场估值高达70亿美元。Neurocrine在发展势头上排名第二，其主要推动力是Crinecerfont，这是一种正在接受美国食品药品监督管理局（FDA）审查的CRF1拮抗剂。 然而，Neurocrine能否持续保持其发展势头尚存疑问，因为该公司与武田制药合作开发的授权产品在II期临床试验中多次遭遇失败。此外，公司的未来战略可能会有所调整，因为长期担任首席执行官的Kevin Gorman在服务32年后即将退休。这一变化可能会对公司的发展方向和战略决策产生重要影响。 展望 – 炒作周期动态？ 生物制药行业的一大特点是，投资者和决策者在面对新兴科学和技术时，往往表现出强烈的群体心理。这种现象可以通过行业炒作周期来形象地描述。一般来说，一项新技术在学术界经过多年的孵化，才会催生出第一批公司，并开始进行商业化交易。在这一过程中，先驱公司往往经历长期的起伏，直到某个关键事件的发生——可能是一笔巨额交易，或者是具有突破性的临床试验结果——这会触发市场的热情，推高交易量和公司估值，同时也加剧了对错失良机的恐惧。 这一阶段通常以少数真正的成功者为特征，他们有时能够获得巨大的回报。然而，与此同时，许多边缘或缺乏差异化的追随者可能只能获得有限的回报，或者在某些情况下，他们的投资可能完全失败。目前，受到这种炒作周期影响的一些领域包括： 肥胖症 -随着时代的变迁，肥胖症治疗领域的投资热情已经发生了翻天覆地的变化。回想过去，诺和诺德和礼来通过肠促胰岛素类药物的创新，为这一领域注入了新的活力。就在五年前，肥胖症在投资者的关注列表中还处于末端。然而，现在的情况已经大不相同。 人工智能 -目前还没有AI直接参与研发的药物对行业产生显著影响，但大型语言模型已经在药物研发的多个环节展现出其潜力，引发了业界对于AI在药物研发中所能扮演角色的巨大期待。值得注意的是，这一技术趋势的触发并非源自生物制药行业内部，而是像ChatGPT这样的技术进步，激发了高管和投资者的强烈兴趣和对错失机会的担忧。 ADC –第一三共的Enhertu的成功为这一技术带来了突破，结束了此前多年的不确定性。然而，ADC技术的真正潜力还有待进一步探索。 寻找下一个大型产品管线免疫药物：TL1A抗体的发现促成了一些历史上规模巨大的药物许可交易，例如罗氏预付71亿美元收购RVT3101，以及与Roivant Sciences和辉瑞的三方合作；默沙东以103亿美元收购Prometheus；武田预付40亿美元收购Nimbus Therapeutics, Inc.的Tyk2抑制剂。这些交易展示了市场对于创新免疫疗法的极高期待。 这些案例仅仅是众多可能的炒作周期中的几个，它们的发展轨迹以及对研发效率的潜在影响值得密切关注。肥胖症治疗和ADC技术的成功案例表明，某些领域可能会经历从被低估到被高度追捧的转变。然而，并非所有领域都能实现这种转变，关键在于能否实现真正的差异化。 在这些炒作周期中，许多后来者已经将过高的期望体现在其估值和交易条款上。这可能导致市场对这些领域的过度乐观，从而忽视了实际的研发风险和挑战。因此，对于这些公司和投资者来说，识别并实现有意义的差异化是确保其可持续性的关键。 差异化可能体现在多个方面，包括但不限于： 技术创新：开发具有独特机制或作用方式的新药，以解决现有治疗方法无法解决的问题。 市场定位：针对特定的患者群体或疾病亚型，提供更精准的治疗选择。 研发效率：通过优化研发流程、利用先进技术（如AI）提高研发效率和成功率。 合作与伙伴关系：与其他公司或研究机构建立战略伙伴关系，共享资源和知识，加速研发进程。 最终，只有那些能够持续提供创新解决方案并实现差异化的公司，才能在竞争激烈的市场中获得成功，并为投资者带来长期的回报。 本文节选自来Catenion的调研报告“2023-2024 Global Biopharma R&D Productivity And Growth Ranking”，由于水平有限，详细内容，请参考原文。 识别微信二维码，添加生物制品圈小编，符合条件者即可加入 生物制品微信群！ 请注明：姓名+研究方向！ 版 权 声 明 本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。