Cenegermin-BKBJ

Phase 3 SELVA trial evaluating QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for microcystic lymphatic malformations completed enrollment, exceeding enrollment target by over 25%; top-line results on track for the first quarter of 2026 Top-line results for Phase 2 TOIVA trial evaluating QTORIN™ rapamycin for cutaneous venous malformations remain on track for the fourth quarter of 2025 QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for microcystic lymphatic malformations and cutaneous venous malformations Company plans to announce both a third clinical indication for QTORIN™ rapamycin and a second QTORIN™ platform candidate before year-end 2025 Cash and cash equivalents of $70.4 million as of June 30, 2025 are expected to fund operations into the second half of 2027 Company to host conference call at 8:30 a.m. ET today WAYNE, Pa., Aug. 14, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or “the Company”), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, reported financial results for the second quarter ending June 30, 2025 and provided a corporate update. “In the second quarter of 2025, we continued to advance Palvella’s mission of pioneering first-in-disease therapies for individuals with serious, rare genetic skin diseases,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “We remain on track for two near-term data readouts for QTORIN™ rapamycin, including our Phase 3 SELVA study in microcystic LMs which we believe will support a New Drug Application submission in 2026. In parallel, we are preparing to announce two new development programs in 2025, further expanding our rare disease pipeline. With strong momentum and a solid financial foundation, Palvella is well-positioned for continued growth and execution on our vision.” Recent Research and Development Highlights QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations (microcystic LMs) Completed enrollment in the Phase 3 SELVA trial, a single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin for the treatment of microcystic LMs; trial enrolled 51 subjects, exceeding its original target of 40 subjects by over 25%. The Company received initial proceeds from an FDA Orphan Products Grant which could total up to $2.6 million to support the Phase 3 SELVA trial. The FDA Orphan Products Grants Program provides non-dilutive funding to advance the development of promising therapies for rare diseases. Key presentations and posters featured at major scientific meetings included: Oral presentation highlighting QTORIN™ rapamycin for the treatment of microcystic LMs was presented by Amy Paller, M.S., M.D., Chair of Dermatology at Northwestern University’s Feinberg School of Medicine, at the 15th World Congress of Pediatric Dermatology; the presentation highlighted the Phase 3 SELVA trial design and reviewed Phase 2 results.Poster highlighting the estimated diagnosed U.S. prevalence and U.S. annual incidence of lymphatic malformations was presented at the 82nd Annual Meeting of the Society for Investigative Dermatology. The United States Patent and Trademark Office (USPTO) issued patent No. 12,268,673 with claims to QTORIN™ rapamycin for the treatment of microcystic LMs with anticipated patent life extending into 2038. Top-line results from SELVA are on track for the first quarter of 2026. QTORIN™ rapamycin for the treatment of cutaneous venous malformations (cutaneous VMs) Continued execution of ongoing Phase 2 TOIVA trial, a single-arm, open-label, baseline-controlled clinical trial evaluating QTORIN™ rapamycin for the treatment of cutaneous VMs. Enrollment is ongoing, targeting approximately 15 subjects. The Company, in collaboration with key opinion leaders in treating venous malformations, completed a nationally representative, blinded, real-world observational study estimating the U.S. annual prevalence of cutaneous VMs, with findings submitted to a peer reviewed journal. Top-line results from TOIVA are on track for the fourth quarter of 2025. QTORIN™ pipeline development The expansion of QTORIN™ rapamycin into a third serious, rare skin disease with no FDA-approved therapies is progressing, with clinical and regulatory planning ongoing, and announcement of the program is planned for the second half of 2025. The second product candidate from the QTORIN™ platform for a serious, rare skin disease with no FDA-approved therapies, has achieved key pre-clinical development milestones, and announcement of the program is planned for the second half of 2025. Recent Corporate Highlights Wes Kaupinen, Palvella’s Founder and CEO, participated in the FDA’s CEO Forum with Commissioner Marty A. Makary, M.D., M.P.H, and other senior FDA officials on June 5th. Strengthened executive leadership team with the appointment of Ashley Kline, a rare disease commercial veteran, as Chief Commercial Officer. Ashley previously led the successful launch of Oxervate®, a first-in-disease topical therapy for neurotrophic keratitis, a rare, serious, and progressive eye disease that previously had no FDA-approved therapies. The USPTO issued patent No. 12,329,748 encompassing a wide range of composition and method-of-use claims for QTORIN™ rapamycin and other mTOR inhibitors formulated in 0.1–20% anhydrous compositions for a broad spectrum of rare and common skin diseases, with patent term expected through at least 2038. The Company was added to the Russell 3000® and Russell 2000® indexes on June 27, 2025. Second Quarter 2025 Financial Results Cash and cash equivalents as of June 30, 2025 were $70.4 million. Palvella expects such resources will be sufficient to fund its operations into the second half of 2027, and sufficient to accomplish its current strategic agenda. Research and development expenses were $5.1 million for the three months ended June 30, 2025, as compared to $1.4 million for the three months ended June 30, 2024. The increase in research and development expenses was primarily due to increased spending on the clinical development of QTORIN™ rapamycin for the treatment of microcystic LMs and cutaneous VMs, including conducting the Phase 3 SELVA and Phase 2 TOIVA trials, which were initiated in 2H 2024. General and administrative expenses were $4.1 million for the three months ended June 30, 2025, as compared to $1.5 million for the three months ended June 30, 2024. The increase in general and administrative expenses was primarily driven by increased employee compensation expense due to headcount additions, as well as increases in expenses related to operating as a publicly-traded company. Net loss attributable to common stockholders was $9.5 million, or $0.86 per basic and diluted share, for the three months ended June 30, 2025, as compared to net loss attributable to common stockholders of $4.4 million, or $2.47 per basic and diluted share, for the three months ended June 30, 2024. Shares outstanding were 13,735,390 as of August 8, 2025, including 11,059,665 shares of common stock and 2,675,725 common share equivalents assuming conversion of outstanding preferred shares and prefunded warrants. Conference Call Details Palvella will host a conference call and live audiovisual webcast to discuss the Company's second quarter 2025 financial results and provide a corporate update at 8:30 a.m. ET today. To access the live webcast of the call with slides, please click here or visit the “Events & Presentations” section of Palvella’s website. To access the call by phone, please use this registration link, and you will be provided with dial in details. A replay of the webcast will be available approximately 2 hours after the conclusion of the call and archived for 90 days under the “Events & Presentations” section of the Company's website at www.palvellatx.com. About Palvella Therapeutics Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being evaluated in the Phase 3 SELVA clinical trial in microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial in cutaneous venous malformations. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter). QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication. Forward-Looking Statements This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)). These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella, as well as assumptions made by, and information currently available to, the management of Palvella. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from ongoing clinical trials, Palvella’s clinical development plans and related anticipated development milestones, Palvella’s cash and financial resources and expected cash runway, and the potential of, and expectations regarding, Palvella’s programs, including QTORIN™ rapamycin, and its research-stage opportunities and any additional indications or platform candidates, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin; the outcome of early clinical trials for Palvella’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella’s current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that Palvella may face. Except as required by applicable law, Palvella does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release. Contact Information Investors Wesley H. Kaupinen Founder and CEO, Palvella Therapeutics wes.kaupinen@palvellatx.com Media Marcy Nanus Managing Partner, Trilon Advisors LLC mnanus@trilonadvisors.com PALVELLA THERAPEUTICS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except share and per share amounts) Three Months EndedJune 30, Six Months EndedJune 30, 2025 2024 2025 2024 Operating expenses: Research and development$5,118 $1,442 $9,192 $2,426 General and administrative 4,132 1,466 7,929 2,241 Total operating expenses 9,250 2,908 17,121 4,667 Loss from operations (9,250) (2,908) (17,121) (4,667) Total other income (expense), net (221) (1,264) (535) (2,041) Net loss$(9,471) $(4,172) $(17,656) $(6,708) Less: Cumulative Series D preferreddividends — (194) — (388) Net loss attributable to commonstockholders$(9,471) $(4,366) $(17,656) $(7,096) Net loss per share — basic and diluted$(0.86) $(2.47) $(1.60) $(4.01) Weighted-average number of commonshares used in computing net loss per share— basic and diluted 11,052,741 1,770,167 11,033,327 1,770,167 PALVELLA THERAPEUTICS, INC. CONDENSED CONSOLIDATED BALANCE SHEET INFORMATION (in thousands) June 30,2025 December 31,2024 Assets Cash and cash equivalents$70,433 $83,602 Other current assets 3,314 4,632 Total current assets 73,747 88,234 Total assets$73,747 $88,234 Liabilities and Stockholders' Equity Current liabilities$9,616 $12,038 Non-current liabilities 16,351 13,589 Total liabilities 25,967 25,627 Total stockholders' equity 47,780 62,607 Total liabilities and stockholders’ equity$73,747 $88,234

这一轮创新药板块性行情，演绎的无比极致，无论是属于基本面投资者能力范围内的龙头Biotech，还是属于概念趋势投资者的新芽药企，都走出了属于各自的一波行情。 成熟投资者，要理解基本面驱动的逻辑，也要理解资金面驱动的逻辑。 正是有这么一家创业板的药企，在一个月的时间内涨了100%，短期涨幅冠绝创业板的绝大多数生物医药企业，并且该企业对外交流并不多，以至于很多投资人甚至不知道公司在涨什么，它就是海特生物。 海特生物2024年年报显示，公司核心业务为药品制造和医药研发服务，前者主要核心产品为注射用鼠神经生长因子“金路捷”，2024年收入约1.2亿人民币；后者则分为两块，一个是仿制药为主创新药为辅的CRO-CDMO一体化服务，一个则是提供API和原料药CDMO解决方案，2024年实现约4.11亿人民币收入。即便是如此，公司在2024年依旧录得了归母净利润-6934.87万，2025Q1则录得了归母净利润-1393.04万。 显然，仅仅看海特生物目前固有的业务是找不到公司市值暴涨的任何逻辑，事实上市场把公司视为一个”高价值的创新药资产包“，包括现有在销售的创新药长期市场潜力和参股公司进度靠前的创新药管线价值，这给予了市场较大的想象空间，本文借此进行梳理分析。 01 MM新药的新拓展 海特生物在近年来有一个全新的增长点，便是创新药注射用埃普奈明（以下统称“沙艾特”）在2023年11月获批上市，并且在2024年5月正式商业化销售（当年录得销售额2636.83万元），2025年1月1日正式纳入国家医保目录。 沙艾特全球首个且唯一上市的死亡受体4/死亡受体5（DR4/DR5）激动剂，沙艾特作用机制与TRAIL（肿瘤坏死因子相关凋亡诱导配体）相同，与DR4/DR5结合激活凋亡通路，通过“外源性凋亡通路”杀伤肿瘤细胞，且具有p53非依赖性。 目前沙艾特联合沙利度胺和地塞米松获批既往接受过至少2种系统性治疗方案的复发或难治性多发性骨髓瘤（RRMM）成人患者，由此可知其目标群体为后线/末线MM患者，与CD38抗体、TCE、BCMA CAR-T等新型治疗手段同台竞技。 同被纳入医保目录且被CSCO指南推荐治疗RRMM的卡非佐米，被视为沙艾特的合适参照物之一，卡非佐米2023年全球销售额达14.03亿美元，而在国内2023年销售额约合2.86亿元人民币（2022年初上市供应）。 沙艾特在RRMM治疗优势主要有几点： 1）安全性良好-卡非佐米治疗出现的心脏、肺、肾、胃肠、神经毒性，沙艾特主要不良反应为肝毒性，多为轻度，大部分患者无需干预即可恢复，患者具有更好的耐受性； 2）疗效-在同类RRMM人群中，沙艾特方案在OS和DOR上均比卡非佐米方案延长≥6 个月，且对p53缺失等高危亚组优势更大（非头对头）； 基于卡非佐米的商业化情况和沙艾特自身独特优势，沙艾特今年有机会冲击2-3亿元甚至以上的销售额，并且有望在国内冲击10亿元的销售额。 值得注意的是，沙艾特的叙事并未在RRMM领域止步。 临床前及早期临床显示，沙艾特可与蛋白酶体抑制剂、免疫调节剂、BCMA CAR-T等 多种机制药物协同，进一步增强肿瘤细胞凋亡并重塑免疫微环境，这意味着该药物可能跟其他新型药物并非竞争甚至是互补的关系。 据管理层介绍，沙艾特可能通过影响免疫微环境的方式，对接受CAR-T细胞回输的患者体内的CAR-T细胞扩增产生积极影响，国内部分一线血液病医院也与公司合作开展沙艾特用于CAR-T前桥接化疗及CAR-T后的维持治疗等方向的临床研究。 另外，沙艾特正在进行实体瘤的拓展，进度最快的为肉瘤（骨肉瘤/软组织肉瘤）。 02 眼科药物的突破性 在眼科领域，海特生物有两项重要的布局，一是公司自主研发了HT006.2.2滴眼液，二是战略投资了眼科遗传病基因治疗领域的中眸医疗（占比15.38%）。 HT006.2.2滴眼液活性成分是重组人神经生长因子（rhNGF），核心适应症为中、重度神经营养性角膜炎（NK），目前处于Ib期临床，据介绍其经处方优化后稳定性优于同类产品Cenegermin。 对标产品Cenegermin是全球首个用于治疗中重度NK局部生物制剂，作为一线或二线非手术方案，适用于传统保守治疗失败或手术高风险NK患者。 不过，NK的发病率低于万分之五，患者总体人数不算大且诊出率较低，相对而言潜在市场空间有限，若未来rhNGF能拓展至其他角膜神经损伤相关适应症，会有更大市场空间。 海特生物更令人注意的布局是战略投资中眸医疗。 中眸医疗的核心管线是ZM-02，这是一种光遗传基因疗法，是全球首款广谱视网膜色素变性（RP）基因治疗药。 ZM-02的核心机理是通过以AAV为载体将“人造光敏蛋白表达基因”下游不感光的视网膜细胞膜，从而恢复患者视网膜对光的信号和视力，据公司介绍该类疗法适用于光感受器凋亡的患者，包括晚期RP、晚期黄斑萎缩AMD等。 已有临床前及临床数据显示，在动物模型中，中眸医疗筛选出的光敏蛋白（PsCatCh2.0）将失明小鼠的视力恢复到相当于人类视力的0.3左右；人体临床中，两位已入组视网膜色素变性患者都出现了视觉功能的明显提升。值得注意的是，该疗法为基于AAV的基因治疗，能够做到一次注射长期有效。 （患者治疗两周后的视力改善对比  图源：生辉） 仅以RP适应症为例，美国潜在患者人数可能高达10万人，而中国患者规模可能超过百万，中重度患者占到其中的60-70%。目前全球范围缺乏有效的治疗手段，唯一上市的基因疗法Luxturna仅限RPE65突变相关RP，2023年销售5800万 美元，2024年受限于免疫性难题和产能不足。而ZM-02是广谱性的基因治疗，不受视网膜色素变性的特定基因突变类型限制，拥有更宽阔的市场空间。加上老年性黄斑变性中的晚期黄斑萎缩（GA）约占晚期AMD的20–25%，推算全球GA患者150–200万人，中国预计30-40万人，市场空间更大。 尽管中眸医疗对目前自家ZM-02管线的阶段性估值为96.55亿人民币确实有些乐观，但ZM-02的广阔市场空间和眼科重磅炸弹的潜力不可否认，需要进一步在临床验证有效性和安全性数据。 03 参股公司小分子PCKS9，进度靠前 早在2021年，海特生物对西威埃医药进行增资，增资完成后持有该公司约13.64%的股权（第三大股东），这项布局也使得海特生物一只脚踏入了心血管创新药领域。 西威埃医药是一家专注于心血管和肝脏代谢疾病创新药的Biotech，其核心管线是口服小分子PCSK9抑制剂CVI-LM001，另外还有进入临床的THR- β选择性激动剂CVI-2742，在治疗NASH和NAFLD患者方面具有同类最佳潜力。 尽管CVI-LM001暂未公布最新的II期临床数据，其在Ib期药效探索研究就已经看到明确的初步疗效证据（相比安慰剂组治疗组导致血液LDL-C水平显著降低）。 从进度上看，目前口服PCSK9全球第一梯队的管线为默克的Enlicitide（小分子化药）和阿斯利康的AZD0780（口服多肽），均处于临床三期（默克占得先机），第二梯队则是诺和诺德的NNC0385-0434、西威埃的CVI-LM001，均处于临床二期。 目前市场上获批的PCSK9药物均为注射剂型，尽管首个siRNA药物出现展现了一定的竞争格局的变化，但整体PCSK9药物市场仍然在2024年保持总体规模的强劲增长。 海外行业专家评论表明，如果疗效和耐受性相当，口服PCSK9药物有潜力完全取代注射剂型；并且，海外分析师预测默克的Enlicitide峰值销售额有望达到40亿美元。 CVI-LM001有望成为国内最快进入临床三期的口服PCSK9分子之一，未来有望实现授权出海，与合作伙伴分享全球百亿美元市场。 结语：在2024年的年报里，海特生物体内看起来“真的没什么东西”，但这不妨碍公司”全新商业化创新药+体外一众FIC的管线资产包“被市场投资者视为“香饽饽”，不说未来兑现如何，至少它们叙事的宏大性和故事性，是令人多巴胺拉满的。 识别微信二维码，添加抗体圈小编，符合条件者即可加入抗体圈微信群！ 请注明：姓名+研究方向！ 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。