Ponsegromab

恶病质也被称之为消耗综合征，在多种癌症患者中普遍存在，发生了恶病质会导致患者的体重减轻、肌肉萎缩、生活质量下降，此外也会影响患者的生存期。关于恶病质的判定，国际的标准为6个月内体重减轻超过5%，或者体重减轻超过2%且体质指数BMI小于20或患有肌肉减少症。美国和欧洲尚未批准用于治疗癌症恶病质的药物，所以出现了恶病质则治疗选择有限。 最近的一项指南支持使用低剂量的奥氮平来改善晚期癌症患者的食欲和体重，此外孕酮类似物或糖皮质激素也能带来短期的益处，但会产生不良反应。比如孕激素使用会导致血栓栓塞事件，其他的临床试验则未能给出足够信服力的数据，因此关于恶病质并无监管部门批准的药物。日本批准使用生长素释放肽受体激动剂阿拉莫林来治疗癌症恶病质，但该药物仅仅导致身体成分略有增加，但是没有改善患者的肌肉，所以这个药没有活得美国FDA的批准。 1、GDF-15的Ponsegromab和最新数据 生长分化因子15属于体重调节、厌食症的调节剂，与恶病质的发病机制相关，在动物学试验GDF-15诱导了恶病质的发生，如果抑制GDF-15则可以缓解症状。今天癌度给大家介绍的Ponsegromab是一种抑制GDF-15的药物，在一项早期的临床试验中，Ponsegromab与体重增加、食欲改善相关，且发生不良事件的概率较低。所以就有了下面的一项二期临床试验，入组的患者数量更多，研究结果刊登在国际学术期刊《新英格兰医学》。 研究的刊例结果 尽管属于二期临床试验，这个研究入组的患者还是比较多的，共计有187名患者进行随机分组，其中40%的患者是非小细胞肺癌患者，32%的患者患有胰腺癌，29%的患者患有结直肠癌。患者用药时间为12周，分别接受不同剂量的Ponsegromab或安慰剂治疗。 Ponsegromab可以改善恶病质 上面的图示表示了研究的对比结果，下面的图示表示是不同剂量Ponsegromab的情况，其中400毫克Ponsegromab的剂量效果最好，相比安慰剂来说，400毫克的Ponsegromab剂量可以改善患者的食欲、改善了患者体重。 2、什么时候可以用？ 一般来说刊登在国际学术期刊《新英格兰医学》都是比较重要的医学突破，而且这个临床试验入组的患者数量还是足够的，所以相信这款药应该在二期临床试验结果公布之后就会提交上市申请，但具体什么时候批准上市还是稍微需要一点时间。此外这款药还有一些研究需要进一步做，比如用的时间更长一些会不会效果更好，使用12周的时间是彻底解决了恶病质的问题还是暂时缓解？这些都是需要进一步研究的问题。 当然大家可以关注这款药在国内的临床试验情况，如果有机会能参加临床试验免费用药，那就是最好的情况了。因为这类药物暂时没有仿制药，也可能短期没有同类药物出现，而且属于抗体类药物，也不利于仿制，所以价格也会肯定比较高，参加临床试验免费使用是最理想的情况，欢迎大家关注癌度，了解全球肿瘤诊疗的最新进展。 癌度与吉因加启动的“基因检测人人可及”计划里，仅需要3000元就能检测36个基因，大家可以联系癌度或扫描上面的二维码咨询癌度医学顾问，了解基因检测的相关问题。  往期推荐  肿瘤的诊断和治疗：肿瘤专家谈胰腺癌的个性化治疗，希望在哪里？ 老药新用，之前批准用于骨髓瘤和淋巴瘤的塞利尼索，对KRAS基因突变肺癌有治疗效果！ 电影明星杜夫·龙格尔从诊断为晚期癌症，到通过治疗摆脱癌症的经历！ 新一代激素受体阳性乳腺癌芙仕得疗效数据公布，刊登新英格兰医学杂志！

hapabapa/Getty Images CEO Albert Bourla, who is under attack from activist investor Starboard Value, got a much-needed victory on Tuesday as Pfizer reported that third-quarter revenue and adjusted profit beat the analysts’ consensus. With a $1 billion stake in Pfizer, activist investor Starboard Value has recently criticized CEO Albert Bourla and senior management for “dramatically” underperforming peers and the market. However, the company on Tuesday reported its third consecutive quarter of positive results , handily beating Wall Street’s expectations. In Q3, Pfizer secured revenue of $17.7 billion compared to the consensus estimate of $14.92 billion, while reporting earnings per share of $1.06 that topped the analyst estimate of $0.62. COVID-10 antiviral medication Paxlovid brought in $2.7 billion in the quarter, up from the $202 million that Pfizer posted in Q3 2023. Guggenheim analyst Vamil Divan in a note to investors called Pfizer’s Q3 performance “very strong” and highlighted Paxlovid sales, “which beat our increased expectations by over $1B and consensus by more than $2B.” BMO Capital Markets analyst Evan Seigerman called Tuesday’s financial results a “solid” Q3 for Pfizer with a top- and bottom-line beat. The quarterly win comes at a critical time for Pfizer’s CEO, who is under pressure from Starboard Value. Analysts in recent weeks have rallied around Bourla in response to the Starboard challenge. However, Guggenheim’s Divan on Tuesday warned that “investors will continue to look for more visibility into how management hopes to navigate the various challenges they face in the coming years as many of their top products either lose patent protection or face increased competition.” Looking ahead, Pfizer on Tuesday raised its full-year 2024 revenue outlook to between $61 billion and $64 billion, up from a previous range of $59.5 billion to $62.5 billion. The company also raised its guidance on adjusted annual earnings per share to a range of $2.75 to $2.95 per share, an increase from a previous range of $2.45 to $2.65 per share. Leerink analyst David Risinger in a note to investors said Pfizer’s new guidance ranges imply a “conservative” Q4. Assuming the company achieves the high end of its new guidances, Risinger contends revenue for the final quarter of 2024 would be $17.3 billion, which is 4% below current consensus of $18.1 billion. Risinger in a separate note said that “likely in response to activist investor Starboard engagement,” Pfizer plans to showcase the company’s pipeline on Nov. 1, starting with ponsegromab—a monoclonal antibody directed against growth differentiation factor-15 (GDF-15), which showed positive results in a Phase II study in patients with cancer cachexia and elevated levels of GDF-15. The candidate is also being investigated in a Phase II study in patients with heart failure and elevated serum GDF-15 concentrations. “We learned from a clinicaltrials.gov update that Pfizer recently upsized and delayed ponsegromab’s Ph2 trial, including changing the estimated primary completion date from March 2025 to March 2026,” according to Risinger. While he awaits management comments on the rationale behind the heart failure trial changes, Risinger continues to view ponsegromab as having “multi-billion-dollar blockbuster potential in cancer cachexia due to lack of approved therapies plus potentially other indications.”