Ascendis

Pictured: BioMarin headquarters in California/iSto iStock , Sundry Photography The biotech beat Wall Street’s third-quarter revenue forecast by 6%, driven by increased uptake of its achondroplasia drug Voxzogo. However, William Blair downgraded BioMarin’s shares to market perform due to a “lack of near-term catalysts” and uncertainty around Voxzogo’s potential revenue growth. BioMarin Pharmaceuticals on Tuesday announced $746 million in total revenue in the third quarter , 28% year-over-year growth driven by the continued strong uptake of its therapies for rare genetic diseases. The biotech beat consensus estimates, surpassing Wall Street’s expected Q3 revenue of $703 million by 6%. BMO Capital Markets analyst Kostas Biliouris in an investor note attributed BioMarin’s strong performance to the continued growth of its achondroplasia medicine Voxzogo, with sales of $190 million jumping 54%. There are now over 3,800 patients on Voxzogo, according to BioMarin, up from around 3,500 in the second quarter . Still, Wall Street anticipated Voxzogo generating $195 million for BioMarin in Q3, a bar the company missed by 3%. Roctavian, a gene therapy approved in June 2023 for hemophilia A , brought in $7 million in the quarter, in line with the analyst consensus. And while its relative contribution to BioMarin’s total Q3 revenue is low, Roctavian presents a high-growth opportunity for the biotech with 600% sales growth versus the same period the year prior. Biliouris said that this “continued uptake” of Roctavian reflects the company’s efforts to focus on the U.S., Italy and Germany markets, potentially paving the way for hitting around $60 million in revenue for the gene therapy by 2025. Truist Securities analyst Joon Lee also credited BioMarin’s Sanofi-partnered enzyme replacement therapy Aldurazyme for its Q3 beat. Sales of Aldurazyme, indicated for mucopolysaccharidosis I, jumped 407% in the quarter bringing in $71 million and beating the consensus expectation of $30 million by $137%. Encouraged by its strong Q3 showing—and by the continued growth of its products—BioMarin on Tuesday raised its full-year 2024 revenue guidance to between $2.79 billion and $2.825 billion. The biotech previously expected a range of $2.75 billion to $2.825 billion this year. Despite BioMarin’s promising outlook for 2024, Lee warned about impending competitive pressure on Voxzogo. Last month, Ascendis Pharma revealed strong pivotal data for its investigational drug TransCon CNP, also known as navepegritide, which showed significant improvements in annual growth velocity in children with achondroplasia. Ascendis is gearing up for a regulatory filing for TransCon CNP in the first quarter of 2025. “In light of better-than-expected data, we’re left to wonder what further impact competition from TransCon-CNP may have on BioMarin’s mid- to long-term guidance,” Lee said. Likewise, William Blair analyst Sami Corwin in a note to investors said that despite the Q3 beat, her firm downgraded shares of BioMarin to market perform. “We do not think its clinical pipeline will be a source of significant value creation in the near term,” according to Corwin. “Voxzogo remains the key top-line growth driver for the company, but the threat of competition could erode its revenue growth and market share.”

GPCR drug developer Septerna plans to raise about $157 million in net proceeds from its proposed initial public offering , according to a new securities filing Monday morning. The Nasdaq debut would push out Septerna’s runway into 2027 and give it an overall capital heap of about $294 million. The Bay Area startup is marketing 10.93 million shares $SEPN in the range of $15 to $17 apiece, according to the SEC filing. The biotech, which works on oral drugs targeting G protein-coupled receptors, initially unveiled its Times Square ambitions on Oct. 2. Septerna is likely to go public right before the US presidential election, and no other major biotech startups are in the IPO on-deck circle. The market has been “quiet” since the steady stream of floats last month and earlier in October from companies like Upstream Bio, CAMP4 Therapeutics and MBX Biosciences. Like MBX, Septerna is working in the rare endocrinology field of hypoparathyroidism. The condition has also seen a bubbling of activity lately with the approval of Ascendis’ Yorvipath this summer and AstraZeneca’s acquisition of Amolyt Pharma. Septerna believes it’s the first to take an oral hypoparathyroidism treatment candidate into human studies. Septerna entered Phase 1 in Australia last month. The healthy volunteer study will include up to 180 adults to test once-daily and twice-daily dosing of its lead asset SEP-786. The biotech anticipates data in mid-2025 and then plans to move into Phase 1 in the US and “select foreign jurisdictions,” including places in Europe and Canada. The biotech has another oral small molecule for the inflammatory skin disease known as chronic spontaneous urticaria, or CSU. The company has started IND-enabling studies for the experimental medicine. It also wants to get into the popular fields of obesity and type 2 diabetes, but those programs are likely far away. The startup launched with $100 million in 2022 based on work in the lab of Nobel laureate and GPCR pioneer Robert Lefkowitz. Septerna then assembled a $150 million Series B in mid-2023. Third Rock Ventures, Samsara BioCapital, RA Capital, Deep Track, Invus, BVF and Goldman Sachs’ West Street Life Sciences all own 5% or more of the 68-employee startup.

▎药明康德内容团队编辑 日前，Ascendis Pharma宣布已向美国FDA提交其药品Skytrofa（lonapegsomatropin，又名TransCon hGH）的补充生物制品许可申请（sBLA），用于治疗患有生长激素缺乏症（GHD）的成人患者。 生长激素缺乏症是一种严重的罕见病，以身材矮小和代谢并发症为特征。在成人GHD患者中，垂体不能产生足够的生长激素，其症状和发病率包括中心肥胖、代谢综合征、骨密度下降、血脂水平改变、疲劳、全身无力、肌肉张力缺乏以及心理症状，如认知障碍、社交孤立、缺乏动力和抑郁。 此次sBLA的提交主要基于foresiGHt试验的结果，这是一项随机、双盲、平行组、安慰剂和活性药物人生长激素（hGH）对照的开放标签3期试验。该试验比较了每周一次TransCon hGH与每周一次安慰剂和每日一次活性对照药物在GHD成人患者中的疗效和安全性。该试验招募了259名年龄在23岁至80岁之间的GHD成人患者，按1：1：1的比例随机分组，根据年龄和口服雌激素摄入量评估，接受固定剂量的TransCon hGH、安慰剂或活性对照药物的治疗。TransCon hGH组和活性对照药物组患者每周接受的hGH剂量大致相同。 图片来源：123RF 分析显示，TransCon hGH在第38周时达成试验主要和关键次要疗效终点的优越性。与安慰剂组相比，接受TransCon hGH治疗的患者在第38周时躯干脂肪较基线显著减少，全身瘦肉量增加。TransCon hGH总体上是安全且耐受性良好的，没有因研究药物而停药的情况，且其安全性和耐受性与活性对照药物相当。 TransCon hGH是一款基于TransCon技术研发的每周给药一次、注射用人生长激素，旨在持续释放活性、未修饰的生长激素。患者从每天注射转换到每周注射，可将每年注射天数减少高达86%。TransCon hGH已分别被美国FDA和欧盟批准以Skytrofa为商品名上市，用于治疗GHD儿童和青少年。此前，维昇药业（VISEN Pharmaceuticals）获得Ascendis公司授权，拥有TransCon hGH在大中华区的独家开发、生产和商业化权益。该疗法在中国开展的3期临床试验达到了主要终点——治疗52周后，患者的年化生长速率为10.66厘米/年，与每天注射的生长激素相比达成非劣效性。中国国家药监局（NMPA）已受理维昇药业所递交该疗法的上市许可申请。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Ascendis Pharma Announces Submission of Supplemental Biologics License Application to FDA for TransCon™ hGH for the Treatment of Adults with Growth Hormone Deficiency. Retrieved October 2, 2024 from https://www.globenewswire.com/news-release/2024/09/30/2955194/0/en/Ascendis-Pharma-Announces-Submission-of-Supplemental-Biologics-License-Application-to-FDA-for-TransCon-hGH-for-the-Treatment-of-Adults-with-Growth-Hormone-Deficiency.html [2] 维昇药业内分泌产品管线研发进展. Retrieved October 2, 2024 from https://www.visenpharma.com/cn/ScienceAndProducts/Pipeline 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新