Efgartigimod Alfa

中国医药创新的内生动力还在不在？近期陆续出炉的biotech三季报给出了答案。 11月12日晚间，再鼎医药2024年的三季报发布，“降本增效”在这份季报中得到了生动的体现：第三季度产品收入达到1.02亿美元，同比增长47%；强劲的营收增长使得再鼎的亏损大幅收窄，已能看到经营回血的希望。 业绩会上，再鼎医药首席运营官Josh Smiley表示：“我们有望在2025年底实现盈利。” 第三季度，再鼎的FcRn拮抗剂艾加莫德、PARP抑制剂尼拉帕利两个品种季度销售额都来到了新高：尼拉帕利第三季度达到4820万美元，同比增长16%；艾加莫德也销售了2730万美元，环比增长18%。 成立十年来，再鼎医药孵化出了五个上市产品，商业化成果正在逐步兑现当初的预期。如果一切顺利，今年再鼎的营收应当比去年有不小的增长。 第二增长曲线 艾加莫德已经逐渐成为再鼎医药的第二增长曲线，标志着再鼎管线梯队正在形成。 截至三季度末，艾加莫德实现收入6365万美金，这款治疗全身型重症肌无力的罕见病药物，已经在这一治疗领域中形成领先优势。 去年9月份，艾加莫德静脉注射剂型“卫伟迦”获批上市，今年1月顺利纳入医保之后，放量更加明显。因此今年上半年，再鼎医药明确提高了艾加莫德的销售预期：预计全年将达到8000万美元的销售额。 如今看来，再鼎对艾加莫德的销售预期应当还可以再提高一些。 而且，艾加莫德的销售峰值还远未达到。再鼎医药的美股业绩会上，公司披露：艾加莫德已经覆盖了大约国内65%全身型重症肌无力的潜在市场，已经提前完成了全年的进院目标。全国共有17万名全身型重症肌无力患者，目前有约10000名患者接受过艾加莫德治疗。 如果艾加莫德的渗透率能再提高一倍，这一品种将会成为再鼎医药下一个10亿级单品。 这一目标并不难实现。在业绩会上，再鼎医药对投资人表示：在进医保的初期，艾加莫德主要在针对急性期患者的使用上取得了很好的成绩，这部分患者大约只占gMG患者总数的20%。而在第三季度，我们观察到更多的维持期患者接受卫伟迦的治疗。这意味着除了有稳定的新患增加，更多维持期的患者会使用艾加莫德。 而且艾加莫德还在开发新的剂型。今年7月，艾加莫德皮下注射剂“卫力迦”获批，用于治疗乙酰胆碱受体抗体阳性的成人全身型重症肌无力（gMG）患者，11月11日，“卫力迦”用于治疗慢性炎性脱髓鞘性多发性神经根神经病（CIDP）的新适应症获批，这一适应症国内没有其他创新药物获批，艾加莫德将独享这一市场。 根据临床管线，再鼎医药还在推进艾加莫德多个适应症的临床，如大疱性类天疱疮、甲状腺眼病、肌炎、血清阴性重症肌无力等，这意味着艾加莫德还可能有更可观的增长空间。 差异化布局成果显现 再鼎医药给外界的印象是差异化管线布局的思路始终非常清晰，能不断拿出行业领先的品种。过去几年，再鼎推出的“则乐”、“卫伟迦”等管线都是引领治疗领域突破的品种。 未来几年，再鼎还会给市场带来怎样的惊喜？ 从公司内部口径来看，如今的再鼎只是刚刚站在商业价值加速转化的临界点上。 再鼎医药董事长杜莹表示：今年在后期管线取得了很大成果。未来一到两年内，至少会有5个产品商业化落地，包括精神疾病药物KarXT，以及靶向FGFR2b药物Bemarituzumab。 KarXT是一种口服M1/M4型毒蕈碱乙酰胆碱受体激动剂和毒蕈碱受体拮抗剂的组合，用于精神分裂症、阿尔茨海默症精神症状等精神疾病的治疗。杜莹披露，KarXT预计在2025年年初递交上市申请。 国内精神疾病创新药物并不多见，阿尔茨海默病更是全球医学难题。再鼎如果在这一领域有所突破，可能会引发新一轮估值浪潮。 杜莹提到的另一款药物Bemarituzumab用于胃癌的一线治疗，今年10月完成了全球三期临床入组。临床管线里还酝酿着更多潜力新品，如编号为ZL-1310的DLL3 ADC，目前已经在在1期临床中显现出治疗广泛期小细胞肺癌同类最佳的潜力。 如果这些重磅管线能顺利转化，再鼎产品的治疗领域覆盖广度和深度将进一步提升，在肿瘤、罕见病、抗感染、精神等多个创新关键领域都有布局，届时将会非常考验再鼎的研发投入力度和销售管理水平。 再鼎已经开始重视这一点，三季报显示，公司的研发费用率和销售费用率较去年同期均有所缩减，刚刚过去的第三季度成为再鼎医药有记录以来亏损净额最低的一个季度。 再鼎的产品销售的持续向好，自然相关费率降低。更重要的是再鼎在同时支持近10个产品同步推进研发和销售的基础上，管线推进与商业化效率在明显提升，这是支持后续研发和销售拓展的关键指标。 截至第三季度末，再鼎医药账面上还有7.16亿美元的现金储备。资金是当下创新药企最宝贵的资源，在直接融资渠道接近枯竭的背景下，再鼎足以安然度过周期，迎接下一波热潮。未来的再鼎，很快会有拨开云雾的那天。 撰稿丨杨曦霞 编辑丨江芸 贾亭 运营｜廿十三 插图｜视觉中国 声明：健识局原创内容，未经许可请勿转载 医保局这一重大政策，点燃医药板块热度 单药年销售有望突破10亿！诺诚健华又行了 新事丨以岭药业被计为“一般失信”

WALTHAM, Mass.--( BUSINESS WIRE )--Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology company focused on discovering and developing potential best-in-class medicines for serious and rare diseases, today reported recent business highlights and financial results for the third quarter ending September 30, 2024. “ We had another exceedingly strong quarter of execution across our TED and FcRn portfolios,” said Steve Mahoney, Viridian’s President and Chief Executive Officer. “ In TED, we reported highly-compelling and positive phase 3 data for veligrotug and eagerly anticipate the readout of THRIVE-2 in December. We also initiated both pivotal phase 3 clinical trials for VRDN-003, our next-generation subcutaneous IGF-1R inhibitor. For our FcRn inhibitor portfolio, we are thrilled today to share VRDN-008 NHP data for the first time, demonstrating a potential best-in-class pharmacokinetic and pharmacodynamic profile for this half-life extended bispecific FcRn inhibitor. Together with our Fc fragment approach with VRDN-006, we believe these programs in our FcRn portfolio will bring differentiated options for patients and each contribute to an exciting pipeline beyond TED. This quarter, we also further strengthened our cash position with a financing that allows us to accelerate our R&D pipeline and extend our cash runway into the second half of 2027.” RECENT TED PORTFOLIO PROGRESS Veligrotug is an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody in phase 3 development for thyroid eye disease. Achieved All Primary and Secondary Endpoints in THRIVE: In September, Viridian announced positive topline results for veligrotug in THRIVE, a global phase 3 clinical trial in patients with active TED. Veligrotug met all of its primary and secondary endpoints after five infusions with high statistical significance (p < 0.0001) and was generally well-tolerated. THRIVE-2 Topline Data Readout on Track for December 2024: THRIVE-2 is a global phase 3 clinical trial assessing the efficacy and safety of veligrotug after five infusions in patients with chronic TED. THRIVE-2 completed enrollment in July 2024 with a total of 188 patients, exceeding the enrollment target of 159 patients due to patient demand, and is on track for topline readout of the 15-week primary efficacy analysis in December. Veligrotug BLA on Track for Second Half 2025: Viridian anticipates submitting a Biologics License Application (BLA) in the second half of 2025, pending data, and expects that its data package will support a marketing authorization application in Europe. VRDN-003 is a potential best-in-class, subcutaneous, half-life extended anti-IGF-1R antibody with the same binding domain as veligrotug. REVEAL-1 and REVEAL-2 Initiated in August 2024: REVEAL-1 and REVEAL-2 are designed to evaluate VRDN-003 in patients with active and chronic TED, respectively, via a subcutaneous administration every four weeks or every eight weeks. Viridian believes the THRIVE topline results provide strong evidence to support the profile of VRDN-003 as a potential best-in-class subcutaneous anti-IGF-1R antibody in a low-volume, infrequent, self-administered, subcutaneous injection designed for patients to use at home. Topline Data and BLA in 2026: Viridian anticipates topline data from both REVEAL-1 and REVEAL-2 in the first half of 2026, with a BLA submission planned by year-end 2026. The company plans to launch VRDN-003 with a commercially available low-volume autoinjector for at-home administration. FCRN PORTFOLIO UPDATE WITH NEW VRDN-008 NHP DATA VRDN-006 is a highly selective Fc fragment which inhibits FcRn, and is designed to be a convenient subcutaneous and self-administered option for patients. IND on Track for Year-End 2024: Viridian is on track to submit an IND application for VRDN-006 by year-end 2024. Viridian anticipates starting a phase 1 clinical trial for VRDN-006 in healthy volunteers in early 2025 and proof-of-concept IgG reductions from that study in the second half of 2025. VRDN-008 is a half-life extended bispecific FcRn inhibitor comprising an Fc fragment and an albumin-binding domain designed to prolong IgG suppression and provide a potentially best-in-class subcutaneous option for patients. Positive New NHP Data Confirms Half-Life Extension: In a head-to-head study, single doses of VRDN-008 demonstrated 3x the half-life of efgartigimod and showed a deeper and more sustained IgG reduction in NHPs with peak IgG reductions that were 20% deeper than efgartigimod. IgG levels returned to baseline 35 days after VRDN-008 dosing, more than twice as long as efgartigimod, which returned to baseline 14 days after dosing. VRDN-008 spared albumin and low-density lipoprotein (LDL), consistent with efgartigimod. Given that NHP data for FcRn inhibitors have been highly translatable to humans, Viridian believes this data shows the potential for VRDN-008 to be a best-in-class, extended half-life FcRn inhibitor for patients. IND Submission Expected Year-End 2025: With these positive proof-of-concept data for VRDN-008, Viridian plans to further advance IND-enabling activities and submit an IND by year-end 2025. Viridian anticipates proof-of-concept IgG reduction data in healthy volunteers in the second half of 2026. FINANCIAL RESULTS Cash Position : Cash, cash equivalents, and short-term investments were $753.2 million as of September 30, 2024, compared with $571.4 million as of June 30, 2024. The company completed a public offering in September 2024 with net proceeds of $243.2 million and believes that its current cash, cash equivalents, and short-term investments will be sufficient to fund its operations into the second half of 2027. R&D Expenses : Research and development expenses were $69.2 million during the quarter ended September 30, 2024, compared to $30.4 million during the quarter ended September 30, 2023. The increase in research and development expenses was driven by increased clinical trials costs associated with our ongoing THRIVE and THRIVE-2 pivotal clinical trials, as well as increased personnel costs to support our pipeline development. G&A Expenses : General and administrative expenses were $14.4 million during the quarter ended September 30, 2024, compared to $20.9 million during the quarter ended September 30, 2023. The decrease in general and administrative expenses was driven by a decrease in personnel-related costs, including share-based compensation expenses. Net Loss : The company’s net loss was $76.7 million for the third quarter ended September 30, 2024, compared with $47.7 million for the same period last year. Shares Outstanding: As of September 30, 2024, Viridian had 97,850,645 shares of common stock outstanding on an as-converted basis, which included 79,181,445 shares of common stock and an aggregate 18,669,200 shares of common stock issuable upon the conversion of 134,864 and 145,160 shares of Series A and Series B preferred stock, respectively. About Viridian Therapeutics Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas. Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting a pivotal program for veligrotug (VRDN-001), including two global phase 3 clinical trials (THRIVE and THRIVE-2), to evaluate its efficacy and safety in patients with active and chronic TED. Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED, including two ongoing global phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of VRDN-003 in patients with active and chronic TED. In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases. Viridian is based in Waltham, Massachusetts. For more information, please visit www.viridiantherapeutics.com . Follow Viridian on LinkedIn and X . Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “on track,” “plan,” “potential,” “predict,” “project,” “design,” “should,” “target,” “will,” or “would” or other similar terms or expressions that concern our expectations, plans and intentions. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. Forward-looking statements include, without limitation, statements regarding: preclinical and clinical development of Viridian’s product candidates veligrotug (formerly VRDN-001), VRDN-003, VRDN-006 and VRDN-008; anticipated start dates of studies, including the initiation date of the phase 1 clinical trial for VRDN-006; milestones; timelines; anticipated data results and timing of their disclosure, including topline results; regulatory interactions and anticipated timing of regulatory submissions, including the anticipated IND submissions for VRDN-006 and VRDN-008 and the anticipated BLA submissions for veligrotug and VRDN-003; Viridian’s expectation that its data package will support a BLA submission for veligrotug in the second half of 2025, pending data; Viridian’s expectation that its data package will support a marketing authorization application in Europe for veligrotug; clinical trial designs, including the REVEAL-1 and REVEAL-2, global phase 3 clinical trials for VRDN-003; Viridian’s plans to launch VRDN-003 with a commercially available auto-injector pen, if approved; the potential utility, efficacy, potency, safety, clinical benefits, clinical response, convenience and number of indications of veligrotug, VRDN-003, VRDN-006 and VRDN-008; potential dosing regimen for VRDN-008; Viridian’s product candidates potentially being best-in-class; and that Viridian’s cash, cash equivalents and short-term investments will be sufficient to fund its operations into the second half of 2027. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to: potential utility, efficacy, potency, safety, clinical benefits, clinical response and convenience of Viridian’s product candidates; that results or data from completed or ongoing clinical trials may not be representative of the results of ongoing or future clinical trials; that preliminary data may not be representative of final data; the timing, progress and plans for our ongoing or future research, preclinical and clinical development programs; changes to trial protocols for ongoing or new clinical trials; expectations and changes regarding the timing for regulatory filings; regulatory interactions expectations and changes regarding the timing for enrollment and data; uncertainty and potential delays related to clinical drug development; the duration and impact of regulatory delays in our clinical programs; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; manufacturing risks; competition from other therapies or products; estimates of market size; other matters that could affect the sufficiency of existing cash, cash equivalents and short-term investments to fund operations; our financial position and projected cash runway; our future operating results and financial performance; Viridian’s intellectual property position; the timing of preclinical and clinical trial activities and reporting results from same; and those risks set forth under the caption “Risk Factors” in our most recent quarterly report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 8, 2024 and other subsequent disclosure documents filed with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither the company, nor its affiliates, advisors, or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (amounts in thousands, except share and per share data) (unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Revenue: Collaboration Revenue - related party $ 86 $ 72 $ 230 $ 242 Total revenue 86 72 230 242 Operating Expenses: Research and development 69,158 30,385 166,294 121,208 General and administrative 14,408 20,911 45,499 62,006 Total operating expenses 83,566 51,296 211,793 183,214 Loss from operations (83,480 ) (51,224 ) (211,563 ) (182,972 ) Other income (expense) Interest and other income 7,795 4,164 23,527 13,029 Interest and other expense (1,004 ) (600 ) (2,188 ) (931 ) Net loss (76,689 ) (47,660 ) (190,224 ) (170,874 ) Change in unrealized gain on investments 1,475 109 594 326 Comprehensive loss $ (75,214 ) $ (47,551 ) $ (189,630 ) $ (170,548 ) Net loss $ (76,689 ) $ (47,660 ) $ (190,224 ) $ (170,874 ) Net loss per share, basic and diluted $ (1.15 ) $ (1.09 ) $ (2.98 ) $ (3.97 ) Weighted-average shares used to compute basic and diluted loss per share 66,420,063 43,654,577 63,800,798 43,057,658 Viridian Therapeutics, Inc. Selected Financial Information Condensed Consolidated Balance Sheets (amounts in thousands) (unaudited) September 30, December 31, 2024 2023 Cash, cash equivalents and short-term investments $ 753,240 $ 477,370 Other assets 18,660 13,054 Total assets $ 771,900 $ 490,424 Total liabilities 64,404 48,402 Total stockholders’ equity 707,496 442,022 Total liabilities and stockholders’ equity $ 771,900 $ 490,424