Boston Pharmaceuticals, Inc.

Palvella Therapeutics to debut on Nasdaq under the ticker symbol “PVLA” as a publicly traded rare disease biopharmaceutical company advancing a late clinical-stage pipeline and a platform for treating serious, rare genetic diseases Strong balance sheet with approximately $80.0 million of cash and cash equivalents, including proceeds from a PIPE financing co-led by BVF Partners, L.P. and Frazier Life Sciences Cash expected to fund operations into the second half of 2027, including through Phase 3 SELVA clinical trial of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs) and Phase 2 clinical trial in cutaneous venous malformations (cutaneous VMs) Microcystic LMs is a chronically debilitating and lifelong genetic disease affecting an estimated more than 30,000 diagnosed patients in the U.S. QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for microcystic LMs and cutaneous VMs WAYNE, Pa., Dec. 13, 2024 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. (Palvella), 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, today announced the completion of its previously announced merger with Pieris Pharmaceuticals, Inc. (Pieris). The combined company will operate under the name Palvella Therapeutics, Inc., and its shares are expected to begin trading on the Nasdaq Capital Market on December 16, 2024, under the ticker symbol "PVLA". Palvella will continue to be led by Wes Kaupinen, its Founder and Chief Executive Officer, and other members of the Palvella management team. The transaction was approved by Pieris stockholders at a special meeting held on December 11, 2024, and the transaction had been previously approved by Palvella stockholders. "With strong support from leading healthcare-dedicated investors, Palvella is well positioned to enter the public markets and pursue our vision of becoming the leading rare disease company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases," said Mr. Kaupinen. “This transaction will enable us to accelerate late-stage development of QTORIN™ rapamycin, our lead product candidate, for microcystic LMs and cutaneous VMs while also further advancing additional novel product candidates from our QTORIN™ platform." Concurrent with the merger, Palvella completed a previously announced oversubscribed $78.9 million private placement co-led by BVF Partners, L.P., an existing investor, and Frazier Life Sciences, a new investor, and with participation from a syndicate of leading healthcare-dedicated investors. Additional new investors include Blue Owl Healthcare Opportunities, Nantahala Capital, DAFNA Capital Management, ADAR1 Capital Management, and a healthcare dedicated fund. Existing investors Samsara BioCapital, Petrichor, CAM Capital, Ligand Pharmaceuticals, Integrated Finance Group (an AscellaHealth partner company), BioAdvance, and Gore Range Capital also participated in the financing. Palvella's cash and cash equivalents of approximately $80.0 million is expected to fund operations into the second half of 2027, including through results from the SELVA Phase 3 clinical trial of QTORIN™ rapamycin for the treatment of microcystic LMs and Phase 2 clinical trial of QTORIN™ rapamycin in cutaneous VMs. Palvella’s research team developed QTORIN™, a patented and versatile platform designed to generate novel topical therapies that penetrate the deep layers of the skin to locally treat a broad spectrum of serious, rare genetic skin diseases. Well-accepted mechanisms of action of rapamycin and other therapeutic agents represent potential therapies for rare genetic skin diseases. However, the adverse event profile of those agents through systemic exposure poses significant barriers to patient adoption. Palvella’s QTORIN™ product candidates are designed for targeted, localized delivery of therapeutic agents to pathogenic tissue of interest while minimizing systemic absorption and thereby reducing the risk of unwanted adverse events associated with systemic therapy. Palvella's lead product candidate QTORIN™ rapamycin is a novel, patented 3.9% rapamycin anhydrous gel currently under development for the treatment of microcystic LMs, cutaneous VMs, and other serious, functionally debilitating skin diseases driven by the overactivation of the mammalian target of rapamycin (mTOR) pathway. QTORIN™ rapamycin has received FDA Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation for microcystic LMs and is the recent recipient of up to a $2.6 million FDA Orphan Products Grant. QTORIN™ rapamycin has also received Fast Track Designation for venous malformations. QTORIN™ rapamycin is protected by issued composition patents covering anhydrous gel formulations of rapamycin, as well as methods of use, in the U.S., Japan, Australia, China and Israel and pending patent applications broadly covering anhydrous gel formulations of rapamycin, as well as methods of use, in the U.S. and other countries. In the third quarter of 2024, Palvella initiated SELVA, a 24-week, Phase 3, single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin administered once daily for the treatment of microcystic LMs. The primary efficacy endpoint is the change from baseline in the overall microcystic LM Investigator Global Assessment (mLM-IGA) at week 24. The Phase 3 study is enrolling approximately 40 subjects, age six or older, at leading vascular anomaly centers across the U.S. Transaction Details Based on the final exchange ratio of approximately 0.30946 shares of Pieris common stock for each share of Palvella common stock, at the closing of the merger, there are approximately 13.95 million shares of the combined company's common stock outstanding on a diluted basis, with prior Pieris stockholders owning approximately 11% on a diluted basis and prior Palvella stockholders (including investors in the private placement) holding approximately 89% of the combined company's outstanding common stock on a diluted basis. In connection with the closing of the merger, Pieris issued a non-transferable contingent value right (CVR) to Pieris shareholders of record immediately prior to the closing, which does not include the former holders of shares of Palvella or the private financing investors. Holders of the CVR will be entitled to receive payments from proceeds received by the combined company, if any, under Pieris' existing partnership agreements with Pfizer and Boston Pharmaceuticals, in addition to other potential licensing agreements involving certain of Pieris' legacy assets, as well as certain potential payments related to historical research and development tax credits, which may or may not be realized. TD Cowen served as lead placement agent and Cantor served as a placement agent for Palvella's concurrent financing. Troutman Pepper Hamilton Sanders LLP served as legal counsel to Palvella. Cooley LLP served as legal counsel to the placement agents. Stifel served as the exclusive financial advisor to Pieris and Mintz, Levin, Cohn, Ferris, Glovsky, and Popeo, P.C. served as legal counsel to Pieris. About Microcystic Lymphatic Malformations Microcystic LMs are a rare, chronically debilitating genetic disease caused by dysregulation of the phosphatidylinositol 3-kinase (PI3K)/mTOR pathway. The disease is characterized by malformed lymphatic vessels that protrude through the skin and persistently leak lymph fluid (lymphorrhea) and bleed, often leading to recurrent serious infections and cellulitis that can cause hospitalization. The natural history of microcystic LMs are persistent and progressive without spontaneous resolution, with symptoms generally worsening during life, including increases in the number and size of malformed vessels that lead to complications and lifetime morbidity. There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States. About Palvella Therapeutics Founded and led by rare drug disease drug development veterans, Palvella Therapeutics (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 in the Phase 3 SELVA clinical trial in microcystic lymphatic malformations (microcystic LMs) and a Phase 2 trial in cutaneous venous malformations. For more information, please visit www.palvellatx.com or follow the Company on LinkedIn. QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency. 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 and Pieris, as well as assumptions made by, and information currently available to, management of Palvella and Pieris. 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, the sufficiency of the combined company’s capital resources; the combined company’s cash runway; the expected timing of the closing of the proposed transactions; statements regarding the potential of, and expectations regarding, Palvella’s programs, including QTORIN™ rapamycin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity; the expected timing of initiating, as well as the design of Palvella’s Phase 2 clinical trial of QTORIN™ rapamycin in cutaneous venous malformations. 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 limited operating history of each company; the significant net losses incurred since inception; 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 the 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 and Pieris to protect their respective intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in Pieris’ most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K filed with the SEC, as well as the registration statement on Form S-4 filed with the SEC by Pieris in connection with the merger. Palvella and Pieris can give no assurance that the conditions to the proposed transactions will be satisfied. Except as required by applicable law, Palvella and Pieris undertake no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release. Palvella Therapeutics Contact Information Investors Wesley H. Kaupinen Founder and CEO, Palvella Therapeutics wes.kaupinen@palvellatx.com Media Stephanie Jacobson Managing Director, Argot Partners palvella@argotpartners.com

The lupus nephritis market is poised for growth, driven by rising disease prevalence, advancements in therapeutics such as biologics and targeted therapies, and increased investment in R&D. Heightened awareness and robust support from governments and key industry players further create a dynamic environment for innovation and better patient outcomes. LAS VEGAS, Dec. 3, 2024 /PRNewswire/ -- DelveInsight's ' Lupus Nephritis Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline lupus nephritis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the lupus nephritis pipeline domain. Key Takeaways from the Lupus Nephritis Pipeline Report DelveInsight's lupus nephritis pipeline report depicts a robust space with 30+ active players working to develop 35+ pipeline therapies for lupus nephritis treatment. Key lupus nephritis companies such as AstraZeneca, Hoffmann-La Roche, Novartis, Qilu Pharmaceutical, Vera Therapeutics, Conduit Pharmaceuticals, Amgen, Johnson & Johnson, BeiGene, RemeGen, Argenx, Kyverna Therapeutics, Cabaletta Bio, ImmPACT Bio, Alpine Immune Sciences, AlloSite Therapeutics, iCell Gene Therapeutics, Caribou Biosciences, Adicet Bio, Shenzhen Pregene Biopharma Co., Ltd., Resolve Therapeutics, BioAegis Therapeutics, Lepton Pharmaceuticals, Transcenta Holding, and others are evaluating new lupus nephritis drugs to improve the treatment landscape. Promising lupus nephritis pipeline therapies such as Anifrolumab, Obinutuzumab, Ianalumab, QLG1074, Atacicept, AZD1656, Daxdilimab, Nipocalimab, Zanubrutinib, Telitacicept, Efgartigimod alfa, KYV-101, CABA-201, IMPT-514, Povetacicept, ONT01, BCMA-CD19 cCAR T cells, CB-010, ADI-001, PRG-2311, RSLV-621, Recombinant human plasma gelsolin, LN-008, TST 008, and others are under different phases of lupus nephritis clinical trials. In November 2024, Adicet Bio announced that the first LN patient had been dosed in the Phase I clinical trial evaluating ADI-001 in autoimmune diseases. In November 2024, Kyverna Therapeutics announced that it would present updated clinical data from LN patients treated with KYV-101 in ongoing Kyverna-sponsored KYSA-1 and KYSA-3 Phase I/II studies and named patient treatments. In October 2024, Kezar Life Sciences suspended subject enrolment and patient dosing in the Phase II PALIZADE trial of zetomipzomib for active lupus nephritis. In June 2024, Nkarta announced the initiation of Ntrust-I, a multi-center clinical trial of NKX019 in lupus nephritis, with the first patient in screening. The company also announced the clearance by the U.S. Food and Drug Administration (FDA) of its second Investigational New Drug (IND) application for NKX019 in autoimmune disease. In June 2024, Adicet Bio announced that the US Food and Drug Administration (FDA) had granted Fast Track Designation to ADI-001 for the potential treatment of relapsed/refractory class III or class IV lupus nephritis. In April 2024, Caribou Biosciences announced that it had received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for CB-010, an allogeneic anti-CD19 CAR-T cell therapy with a PD-1 knockout (KO), for the treatment of lupus nephritis (LN) and extrarenal lupus (ERL). The Phase 1, multicenter, open label GALLOP clinical trial of CB-010 in patients with LN and ERL is expected to initiate by year-end 2024. In March 2024, Artiva Therapeutics is looking to dose the first patient in a Phase I/II trial assessing its cell therapy AlloNK in lupus nephritis (LN). Request a sample and discover the recent advances in lupus nephritis treatment drugs @ Lupus Nephritis Pipeline Report The lupus nephritis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage lupus nephritis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the lupus nephritis clinical trial landscape. Lupus Nephritis Overview Lupus nephritis is a serious complication of systemic lupus erythematosus (SLE), an autoimmune disease that can affect multiple organ systems. In lupus nephritis, the immune system mistakenly attacks the kidneys, leading to inflammation and damage. The exact cause is not fully understood, but it is believed to involve a combination of genetic, hormonal, and environmental factors, including infections and exposure to certain drugs or UV light. Symptoms of lupus nephritis can vary but often include swelling in the legs and feet, high blood pressure, foamy urine, and in severe cases, hematuria. Patients may also experience fatigue, joint pain, and skin rashes, typical of lupus. Diagnosis usually involves a combination of blood tests, urinalysis, and imaging studies. A kidney biopsy may be necessary to evaluate the extent of kidney damage and guide treatment. Treatment for lupus nephritis aims to reduce inflammation and prevent kidney damage. It often involves corticosteroids and immunosuppressive medications such as mycophenolate mofetil or azathioprine. In severe cases, more aggressive treatments, including cyclophosphamide or biologic therapies like belimumab, may be utilized. Regular monitoring of kidney function and overall health is crucial for managing the disease effectively. Find out more about lupus nephritis treatment drugs @ Drugs for Lupus Nephritis Treatment A snapshot of the Lupus Nephritis Pipeline Drugs mentioned in the report: Learn more about the emerging lupus nephritis pipeline therapies @ Lupus Nephritis Clinical Trials Lupus Nephritis Therapeutics Assessment The lupus nephritis pipeline report proffers an integral view of the lupus nephritis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Lupus Nephritis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Interferon alpha-beta receptor antagonists, Cell death inhibitors, Cell death stimulants, Antibody-dependent cell cytotoxicity, B-cell activation factor receptor antagonists, Glucokinase stimulants, Dendritic cell inhibitors, Complement factor D inhibitors, Complement C3 inhibitors, Proteasome inhibitors, Natural killer cell replacements, Immunologic cytotoxicity; T lymphocyte replacements Key Lupus Nephritis Companies: AstraZeneca, Hoffmann-La Roche, Novartis, Qilu Pharmaceutical, Vera Therapeutics, Conduit Pharmaceuticals, Amgen, Johnson & Johnson, BeiGene, RemeGen, Argenx, Kyverna Therapeutics, Cabaletta Bio, ImmPACT Bio, Alpine Immune Sciences, AlloSite Therapeutics, iCell Gene Therapeutics, Caribou Biosciences, Adicet Bio, Shenzhen Pregene Biopharma Co., Ltd., Resolve Therapeutics, BioAegis Therapeutics, Lepton Pharmaceuticals, Transcenta Holding, and others. Key Lupus Nephritis Pipeline Therapies: Anifrolumab, Obinutuzumab, Ianalumab, QLG1074, Atacicept, AZD1656, Daxdilimab, Nipocalimab, Zanubrutinib, Telitacicept, Efgartigimod alfa, KYV-101, CABA-201, IMPT-514, Povetacicept, ONT01, BCMA-CD19 cCAR T cells, CB-010, ADI-001, PRG-2311, RSLV-621, Recombinant human plasma gelsolin, LN-008, TST 008, and others. Dive deep into rich insights for new drugs for lupus nephritis treatment, visit @ Lupus Nephritis Drugs Table of Contents For further information on the lupus nephritis pipeline therapeutics, reach out @ Lupus Nephritis Treatment Drugs Related Reports Lupus Nephritis Epidemiology Lupus Nephritis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical, and forecasted lupus nephritis epidemiology in the 7MM. Lupus Nephritis Market Lupus Nephritis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key lupus nephritis companies, including NOVARTIS, MORPHOSYS, ASTRAZENECA, ROCHE, KEZAR LIFE SCIENCES, ALEXION PHARMACEUTICALS, CABALETTA BIO, among others. Systemic Lupus Erythematosus Market Systemic Lupus Erythematosus Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key SLE companies, including Biogen, Novartis, MorphoSys, Idorsia Pharmaceuticals, Viatris, RemeGen, UCB Pharma, Genentech, Bristol Myers Squibb, AbbVie, among others. Systemic Lupus Erythematosus Pipeline Systemic Lupus Erythematosus Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myasthenia gravis companies, including Yake Biotechnology, UCB, Sorrento Therapeutics, SinoMab Bioscience Ltd, Shanghai Junshi Biosciences, Sareum, Sanofi, Roche, Rheos Medicine, Resolve, Provention Bio, Pfizer, Novartis, Neovacs, Merck, Medsenic, Landos Biopharma, Kezar Life Sciences, Kangpu Biopharmaceuticals, Janssen Research & Development, Janssen, InnoCare, ImmuPharma, I-MAB Biopharma, ILTOO, Idorsia Pharmaceuticals, Horizon Therapeutics, Genovax, Exinda Thearapeutics, Equillium, Eli Lilly and Company, Eisai, Daiichi Sankyo Company, Corestem, Corbus Pharmaceuticals, Citryll BV, Chipscreen Biosciences, Carna Bioscience, Bristol-Myers Squibb, Brickell Biotech, Boston Pharmaceuticals, Biogen, Athos Therapeutics, Asahi Kasei Pharma, Aria Pharmaceuticals, Antengene Therapeutics, Amgen, Alpine Immune Sciences, Akeso Biopharma, AbbVie, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

全球药物批准/研发动态 01 全球新药批准情况 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）全球（不含中国）共有5个新药获批上市。其中，NDA批准3个，BLA批准1个，新制剂批准1个。与上个统计周期相比，本次增加2个批准新药。 11月14日，PTC Therapeutics宣布FDA加速批准基因疗法Kebilidi（Eladocagene exuparvovec）用于治疗芳香族L-氨基酸脱羧酶缺乏症（AADCD）的儿童和成人患者。Kebilidi是一种基于腺相关病毒2（AAV2）载体的基因疗法，旨在通过一次性治疗，将功能正常的DDC基因直接递送到大脑的壳核中，提高AADC酶的水平并恢复多巴胺的生产。在临床研究中，无法达到预期运动发育里程碑的患者早在接受治疗3个月后就能观察到具有临床意义的运动功能改善，这一转变持续到接受治疗后5年以上。 11月16日，Syndax Pharmaceuticals宣布FDA批准“first-in-class”小分子抑制剂Revuforj（Revumenib）用于治疗携带KMT2A易位的复发或难治性（R/R）急性白血病成人和1岁以上儿童患者。在疗效评估人群中，CR/CRh的比例为21%。CR/CRh患者的中位缓解持续时间为6.4个月，达到CR或CRh的中位时间为1.9个月，23%的患者在接受Revuforj治疗后接受了造血干细胞移植（HSCT）。 全球（不含中国）新药批准情况（部分） 02 全球新药申报进展 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）全球（不含中国）共有8个新药申报上市。其中，NDA申报4个，BLA申报4个。与上次统计周期相比，本次增加3个NDA/BLA申报。 11月13日，AstraZeneca和Daiichi Sankyo宣布已为Trop2靶向抗体偶联药物Datopotamab Deruxtecan（Dato-DXd）向FDA递交BLA，用以治疗之前曾接受过全身性治疗（包括EGFR靶向治疗）的携带表皮生长因子受体（EGFR）突变肿瘤的局部晚期或转移性非小细胞肺癌（NSCLC）成年患者。 NDA/BLA申报（部分） 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）全球（不含中国）共有4个药物获监管机构特殊资格认定。其中，化学药2个，生物药2个。与上次统计周期相比，本次增加1个获监管机构特殊资格认定的药物。 11月13日，NurExone Biologic Inc.宣布EMA已授予ExoPTEN疗法孤儿药产品资格认定。ExoPTEN旨在提供脊髓损伤后的神经再生和功能恢复，它使用间充质干细胞衍生的细胞外囊泡，这些囊泡加载了靶向PTEN（神经再生中的关键蛋白）的siRNA。该认定支持了ExoPTEN的开发，并为更快地进入欧洲市场开辟了一条途径。急性脊髓损伤市场面临相当大的挑战，患者通常需要终身护理，并且有效的治疗选择有限。ExoPTEN促进脊髓恢复的创新方法直接解决了这一差距。 11月15日，AlveoGene的AVG-002获得FDA罕见儿科疾病资格认定（RPDD），AVG-002是其针对致命性新生儿表面活性剂蛋白B（SP-B）缺乏症的新型吸入基因疗法。AVG-002通过呼吸滴注将功能性SP-B基因高效、高效地直接递送到新生儿深肺泡区域。单剂量AVG-002可显著延长生存期，从而有可能通过单次给药进行终身治疗。AVG-002治疗后疾病诱导的肺组织中恢复正常肺组织学和功能的研究结果进一步强化了这些数据。 监管机构特殊资格认定药物 03 全球新药研发进展 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）全球（不含中国）新药临床研发状态更新共计67条，涉及肿瘤、血液和淋巴疾病、心血管疾病、精神疾病以及眼部疾病等共计14个领域。 其中，肿瘤领域临床进展更新居各领域之首，为23条：涉及化学药12条，生物药8条，细胞疗法2条，疫苗1条。 11月12日，SpringWorks Therapeutics发表了在研MEK抑制剂Mirdametinib用于治疗1型神经纤维瘤病相关丛状神经纤维瘤（NF1-PN）成人和儿童患者的关键IIb期ReNeu试验的数据。截至2023年9月20日，该试验达成主要终点。在24个治疗周期（约22个月）内，成人患者的ORR为41%，儿童患者的ORR为52%。在获得确认客观缓解的患者中，62%的成人和52%的儿童实现了目标肿瘤体积与基线相比最高缩小幅度超过50%。 11月16日，Boston Pharmaceuticals宣布了一项II期试验的积极结果，评估了每月一次的长效人类成纤维细胞生长因子21类似物Efimosfermin alfa（BOS-580）对具有F2/F3期肝纤维化的代谢功能障碍相关脂肪性肝炎患者的疗效与安全性。BOS-580的治疗可显著改善肝纤维化≥1级，且MASH不会恶化，并且在24周内MASH缓解且肝纤维化不会恶化。 全球新药研发进展详情（部分） 04 全球医药交易事件 本次统计周期（2024.11.09-11.15）全球（含中国）医药交易时间共计34起，涉及药物权益转让、公司并购等多起交易事件。 全球医药交易时间汇总表（部分） 国内药物批准/研发动态 01 国内新药批准情况 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）国内共有2个新药获NMPA批准上市。其中，新适应症批准1个，新剂型批准1个。与上次统计周期相比，本次减少8个NMPA批准新药。 11月11日，argenx SE和Zai Lab Limited宣布NMPA批准VYVGART Hytrulo 1,000毫克（5.6毫升）/瓶[Efgartigimod alfa注射液（皮下注射）]用于治疗慢性炎症性脱髓鞘性多发性神经病（CIDP）成人患者的补充生物制品许可申请（sBLA）。与安慰剂相比，VYVGART Hytrulo的复发风险降低了69%，在研究的开放标签期间接受治疗的中国参与者中有78%表现出临床改善。 11月11日，NMPA官网公示，科笛集团申报的盐酸米诺环素泡沫剂已在中国获批上市，这是一款外用4%米诺环素泡沫剂（CU-10201），获批用于9岁及以上儿童和成人患者的非结节性中度至重度寻常痤疮炎症性病变的局部治疗。临床试验结果显示，受试者在治疗12周后，CU-10201组炎性皮损的改善显著优于安慰剂组，达到主要终点指标。基于IGA评分的成功率，12周治疗后CU-10201组非炎性皮损的改善均显著优于安慰剂组，达到次要终点指标，总体安全性方面和给药部位局部耐受性良好。 国内新药批准情况 02 国内新药临床默示许可进展 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）国内共有44个新药获临床默示许可，涉及65个受理号。其中，化学药21个，治疗用生物制品22个，预防用生物制品1个。与上次统计周期相比，本次增加40个临床默示许可获批受理号。 本周国内新药临床默示许可进展（部分） 03 国内新药申报进展 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）国内共有7个新药申报上市，涉及9个受理号。其中，化药4个，治疗用生物制品1个，预防用生物制品2个。本次新药申报上市受理号数量与上次统计周期持平。 国内新药申报上市情况（部分） 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）国内共有44个新药申报临床，涉及66个受理号。其中，化学药22个，治疗用生物制品19个，预防用生物制品3个。与上次统计周期相比，本次减少3个临床申报受理号。 国内新药临床申报情况（部分） 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）国内共有7个药物获NMPA特殊资格认定。其中，化学药3个，生物药4个。与上次统计周期相比，本次增加4个获监管机构特殊资格认定的药物。 11月12日，CDE官网最新公示，荣昌生物自主研发的全球首款注射用重组B淋巴细胞刺激因子（BLyS）/增殖诱导配体（APRIL）双靶点的新型融合蛋白泰它西普（RC18，泰爱®），新适应症成人全身型重症肌无力（gMG）拟纳入优先审评。临床研究结果显示，泰它西普240mg组QMG评分平均降低9.6分，第12周QMG评分降低9.5分，MG-CAS自基线至第4周下降且在整个研究中持续。在第24周，患者获得QMG临床意义应答（下降3分）的累积百分比为100%，获得QMG显著应答（下降5分）的累积百分比为86.7% 11月15日，CDE官网最新公示，信达生物1类新药IBI354拟纳入突破性治疗品种，针对适应症为铂耐药卵巢癌。IBI354是一款抗HER2单克隆抗体-喜树碱衍生物偶联物，药物抗体比（DAR）值为8，能够携带更多的毒素载荷到达肿瘤细胞。针对铂耐药卵巢癌患者的临床数据显示，其总体ORR为40.2%，DCR为81.6%，40例接受高剂量（12mg/kg）IBI354治疗的卵巢癌受试者中ORR达到52.5%，DCR达90.0%，安全性和有效性良好。 国内监管机构特殊资格认定药物 04 国内新药研发进展 根据药渡数据统计分析，本次统计周期（2024.11.09-11.15）国内新药临床研发状态更新共计12条，涉及肿瘤、皮肤病、呼吸系统疾病和感染共计4个领域。其中，化学药9条，生物药3条。与上次统计周期相比，本次增加1条国内新药临床研发状态更新。 11月11日，翰森制药宣布，其自主研发的每日一次口服P2X3拮抗剂HS-10383治疗慢性咳嗽的I期临床试验取得积极成果。I期临床试验显示，HS-10383整体耐受性良好，HS-10383具有35~48小时的终末半衰期，支持每日一次口服给药。试验结果表明，HS-10383整体耐受性良好，所有不良事件均为轻度至中度，没有味觉障碍相关的不良事件（AEs）被观察到，没有治疗期间不良事件（TEAEs）导致终止治疗的情况。 11月14日，由吉林省肿瘤医院程颖教授牵头开展的特瑞普利单抗联合化疗一线治疗广泛期小细胞肺癌 (ES-SCLC) 的III期EXTENTORCH研究结果发表在《美国医学会杂志·肿瘤学》。Ⅲ期临床研究试验组1年PFS率提升近4倍。与单纯化疗相比，特瑞普利单抗联合化疗显著改善患者PFS和OS，将疾病进展或死亡风险降低33%，死亡风险降低了20%。亚组分析显示，所有关键亚组均显示出一致的PFS和OS获益。 国内新药研发进展（部分） 05 国内新药研发领域政策法规动态 国家药监局药审中心关于发布《基于疾病登记的真实世界数据应用指导原则（试行）》的通告（2024年第48号） 为指导申办者在药品临床研发中规范合理的应用基于疾病登记的真实世界数据，药审中心研究起草了《基于疾病登记的真实世界数据应用指导原则（试行）》（见附件）。根据《国家药监局综合司关于印发药品技术指导原则发布程序的通知》（药监综药管〔2020〕9号）要求，经国家药品监督管理局审查同意，现予发布，自发布之日起施行。特此通告。 06 国内新药研发领域热点新闻 康宁杰瑞开始底部反转 康宁杰瑞过去多个项目的失败，让整个市场都见识到了创新药研发残酷的一面。在2024年以前，中国的Biotech甚少传出研发失败的声音，主要原因在于此前整个研发领域充斥着各种me-too。而如今，低垂的果实已经被采摘完毕，创新药研发开始进入深水区。而康宁杰瑞的数次失败也说明了这些项目是真正的First-in-class项目，没办法耍小聪明，前路尽是未知。 往往在这种时刻，更能体现一家具有真正创新精神Biotech的韧性。在资本市场逐渐火热的氛围下，康宁杰瑞已经悄悄地从低谷爬起，短时间内实现了市值翻倍。更多资讯，请阅读原文 License in的“优等生” Biotech正在上演盈利大戏。 已经实现扭亏的Biotech，或是得益于自研产品实现商业化销售，或是将在研管线对外授权提前斩获现金流，其中尤以第二种商业模式最为普遍。 然而更具看点的是，曾经充满争议的License-in模式似乎也正在走通。云顶新耀、再鼎医药这两家“优等生”，正在用实际成果验证这一模式的可行性。更多资讯，请阅读原文 咨询、合作请联系作者 小D有话说 为方便读者阅读及保存，我们将周报原文整理成了PDF版本，如需获取全文，可点击最上方蓝字，在药渡Daily公众号后台回复“1121周报”，即可下载。