Zopapogene imadenovec

FDA granted priority review to Company's BLA for PRGN-2012 for the treatment of adults with recurrent respiratory papillomatosis and set PDUFA target action date for August 27, 2025 Results from pivotal clinical study of PRGN-2012 were published in The Lancet Respiratory Medicine PRGN-2012 treatment demonstrated durable complete responses with median duration of response of two years, with some complete responders surgery-free beyond three years as of August 28, 2024 data cutoff Company continues to rapidly advance commercial and manufacturing readiness campaign in anticipation of 2025 commercial launch Company ended 2024 with $97.9 million in cash, cash equivalents, and investments, extending its cash runway into 2026, beyond potential 2025 commercial launch of PRGN-2012 GERMANTOWN, Md., March 19, 2025 /PRNewswire/ -- Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, today announced full year 2024 financial results and business updates. "Last year, we achieved several milestones on our path to potentially bring our innovative PRGN-2012 therapy to RRP patients, including presentation of groundbreaking pivotal data and BLA submission to the FDA. Through prioritizing our portfolio and financing activities, we strengthened our financial position, enabling ongoing commercial and manufacturing readiness efforts for PRGN-2012 in anticipation of a potential 2025 launch. Our commercial organization has been working to scale up quickly, right-sizing the organization to rapidly capitalize on the immense demand from patients and treating physicians for a new treatment paradigm that may finally address the underlying cause of the disease," said Helen Sabzevari, PhD, President and CEO of Precigen. "Already this year, our BLA for PRGN-2012 received priority review from the FDA with an August 2025 PDUFA action date, bringing us a step closer to launching the first and only FDA-approved treatment to the approximately 27,000 adult RRP patients in the US. FDA approval for PRGN-2012 would fundamentally change Precigen, enabling the transition from clinical to commercial stage with the real and imminent potential to begin realizing product sales this year and providing financial tailwinds to enable potential expansion of PRGN-2012 to new indications and geographical markets." "As a result of the preferred stock offering and sale of intellectual property and related royalty rights for a non-core asset, we extended our cash runway into 2026, beyond the potential commercial launch of PRGN-2012 this year. We are preparing for the transition to a commercial stage company and the potential to add product-related revenue," said Harry Thomasian Jr., CFO of Precigen. Key Program Highlights PRGN-2012 (nonproprietary name: zopapogene imadenovec†) AdenoVerse® Gene Therapy in RRP PRGN-2012 is an investigational off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP). PRGN-2012 received Breakthrough Therapy Designation, Orphan Drug Designation, and an accelerated approval pathway from the FDA, and Orphan Drug Designation from the European Commission. In February 2025, the US Food and Drug Administration (FDA) accepted the company's biologics license application (BLA) for PRGN-2012, and granted priority review to the BLA with a Prescription Drug User Fee Act (PDUFA) target action date set for August 27, 2025. The FDA has indicated that they are not currently planning to hold an advisory committee meeting to discuss the BLA. Results from the pivotal clinical study of PRGN-2012 for the treatment of RRP were presented at the 2024 American Society of Clinical Oncology (ASCO) annual meeting in a late-breaking oral presentation titled, "PRGN-2012, a novel gorilla adenovirus-based immunotherapy, provides the first treatment that leads to complete and durable responses in recurrent respiratory papillomatosis patients" and published in The Lancet Respiratory Medicine. Pivotal study successfully met its primary safety and pre-specified primary efficacy endpoints. PRGN-2012 was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2. 51% (18 out of 35) of patients achieved Complete Response, requiring no surgeries after treatment with PRGN-2012. Complete Responses have been durable beyond 12 months with median duration of follow up of 24 months, with some complete responders surgery-free beyond three years as of the August 28, 2024 data cutoff. 86% of patients (30 out of 35) had a decrease in surgical interventions in the year after PRGN-2012 treatment compared to the year prior to treatment; RRP surgeries reduced from a median of 4 (range: 3-10) pre-treatment to 0 (range: 0-7) post-treatment. PRGN-2012 treatment induced HPV 6/11-specific T cell responses in RRP patients with a significantly greater expansion of peripheral HPV-specific T cells in responders compared with non-responders. PRGN-2012 significantly (p < 0.0001) improved anatomical Derkay scores and VHI-10 scores in complete responders. Patient enrollment continues to advance in the confirmatory clinical trial of PRGN-2012 in accordance with the guidance from the FDA to initiate the study prior to submission of the BLA. The Company continues to rapidly advance its commercial and manufacturing readiness campaign in anticipation of a potential launch in 2025. Based on recently updated internal analysis derived from review of claims data, the market opportunity for PRGN-2012 in RRP is estimated to be approximately 27,000 adult patients in the US and more than 125,000 patients outside of the US. PRGN-2009 AdenoVerse® Gene Therapy in HPV-associated cancers PRGN-2009 is an investigational off-the-shelf AdenoVerse gene therapy designed to activate the immune system to recognize and target HPV-associated cancers. PRGN-2009 Phase 2 clinical trials under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer are ongoing. PRGN-3006 UltraCAR-T® in AML and MDS PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T cell (CAR-T) therapy engineered to simultaneously express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a safety/kill switch. PRGN-3006 has been granted Orphan Drug Designation in patients with acute myeloid leukemia (AML) and Fast Track Designation in patients with relapsed/refractory (r/r) AML by the FDA. The Company has completed enrollment of the Phase 1b trial for PRGN-3006 in acute myeloid leukemia (AML), which received Fast Track designation from the FDA, and is preparing for an end of Phase 1b meeting with the FDA to discuss next steps. Financial Highlights In December 2024, the Company raised $87.5 million, of which $79.0 million was from a private placement offering of convertible preferred stock, and an additional $8.5 million was from the sale of certain intellectual property and royalty rights related to a non-core asset. By year's end 2024, cash, cash equivalents, and investments totaled $97.9 million. Full Year 2024 Financial Results Compared to Prior Year Period Total revenues decreased $2.3 million, or 37%, and cost of products and services decreased $1.8 million or 30% compared to the year ended December 31, 2023. These decreases were primarily related to reductions in product and service volumes at Exemplar. Research and development expenses increased $4.5 million, or 9%, compared to the year ended December 31, 2023. This increase was primarily the result of $3.1 million of increased costs associated with the initiation of the PRGN-2012 confirmatory clinical trial, increased drug manufacturing material costs related to PRGN-2012 for potential commercial use, and professional fees incurred in conjunction with the Company's completed BLA submission and commercial readiness planning as well as the design and implementation of our manufacturing facility. Additionally, employee-related expenses rose by $3.0 million primarily due to severance charges incurred as a result of the Precigen workforce reduction in 2024 and the suspension of ActoBio's operations. These increases were partially offset by a $1.9 million reduction in depreciation and amortization expense as a result of the impairment of noncurrent assets related to the suspension of ActoBio's operations during the second quarter of 2024 as well as a reduction in clinical study expenses associated with programs that were deprioritized during the third quarter of 2024. SG&A expenses increased $0.9 million, or 2%, compared to the year ended December 31, 2023. As a result of the Company's increased focus on PRGN-2012, commercial readiness costs increased in 2024 compared to the prior year. In addition, the second and third quarters of 2024 included higher severance costs associated with the suspension of ActoBio's operations and the Precigen workforce reduction. These increases were partially offset by a decrease in stock compensation and insurance rates in 2024 compared to the same period in 2023. The Company recorded a $5.8 million impairment charge in the fourth quarter of 2024 related to its Exemplar subsidiary as a result of the Company's annual goodwill impairment test. Also, in conjunction with the suspension of ActoBio's operations, the Company recorded $34.5 million of impairment charges related to goodwill ($1.6 million) and other noncurrent assets ($32.9 million) in the second quarter of 2024. Total other income, net, increased $3.6 million, or 106% compared to the year ended December 31, 2023. This increase was primarily derived from an $8.5 million gain on the sale of intellectual property rights and royalty rights related to a non-core asset in December 2024 as well as a $0.5 million reduction in interest expense due to the final retirement of the Company Convertible Notes in the second quarter of 2023. This increase was partially offset by a reclassification of cumulative translation losses of $2.9 million, which resulted from the final closing of the ActoBio facilities in the third quarter of 2024, as well as a reduction of $1.8 million in interest income compared to the same period in 2023. Net loss was $126.2 million, or $(0.47) per basic and diluted share, compared to net loss of $95.9 million, or $(0.39) per basic and diluted share, in year ended December 31, 2023. Precigen: Advancing Medicine with Precision ® Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit or follow us on LinkedIn or YouTube. Trademarks Precigen, UltraCAR-T, UltraPorator, AdenoVerse, UltraVector and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners. Cautionary Statement Regarding Forward-Looking Statements Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon the Company's current expectations and projections about future events and generally relate to plans, objectives, and expectations for the development of the Company's business, including the timing and progress of preclinical studies, clinical trials, discovery programs and related milestones, the promise of the Company's portfolio of therapies, and in particular its CAR-T and AdenoVerse therapies. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release. The Company has no obligation to provide any updates to these forward-looking statements even if its expectations change. All forward-looking statements are expressly qualified in their entirety by this cautionary statement. For further information on potential risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in the Company's most recent Annual Report on Form 10-K and subsequent reports filed with the Securities and Exchange Commission. Investor Contact: Steven M. Harasym Tel: +1 (301) 556-9850 [email protected] Media Contacts: Donelle M. Gregory [email protected] †zopapogene imadenovec is the nonproprietary name for the investigational therapeutic known as PRGN-2012. Zopapogene imadenovec has not been approved by any health authority in any country for any indication. SOURCE Precigen, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

BFC Group简介 BFC Group是一家立足于中国的精品投行，专注于医疗健康领域的并购、专利许可、融资。我们的业务板块涉及中国、美国和欧洲，曾与国内外众多领先的医疗健康公司有过合作并保持着良好的关系。随着在美国和中国一线运营经验的不断积累，我们对客户在本土之外建立公司或扩展业务所面临的挑战有着深刻的理解。 立于塔顶，高瞻之见。 使命愿景 紧跟医疗健康领域趋势，通过合作推动科学突破与创新。 依客户需求制定战略拓展计划，并在目标市场全方位落实与执行。 成为专注医疗健康行业最优秀的投资银行。 No.1 香港资本市场 香港股市总体概览 2月26日，香港特区政府财政司司长陈茂波公布了2025/2026财政年度特区政府财政预算案。此次发布的财政预算案中涉及多项港股改革方向，包括推进人民币柜台纳入港股通、筹备开通“科企专线”、优化双重主要上市及第二上市门槛涉及港股改革等。 特朗普关税影响及媒体称国际对冲基金获利了结港股，上周港股回调，恒生指数累计下跌2.3%。 2025年至今，恒生指数累计上涨14.4%。 上周，恒生医疗保健指数(HSHCI)累计下跌3.6%。 康宁杰瑞制药-B周涨33.6%，消息面上：2月27日康宁杰瑞生物制药与石药集团共同宣布，HER2双特异性抗体偶联药物(ADC)JSKN003对比恩美曲妥珠单抗(T-DM1)治疗HER2阳性晚期乳腺癌的Ⅲ期临床研究完成首例患者给药。公司指目前JSKN003治疗HER2低表达乳腺癌(JSKN003-302)、铂耐药卵巢癌(JSKN003-306)的Ⅲ期临床研究也在顺利进行中。另恒生指数公司近期公布了其定期的季度指数调整结果。调整将于3月10日起生效。其中，康宁杰瑞制药遭剔除出恒生综合指数。 科济药业-B周涨24.7%，消息面上：2月25日公司宣布与珠海软银欣创旗下管理基金计划达成协议，共同投资优恺泽生物医药（上海）有限公司（“优恺泽”）。根据协议条款，优恺泽获得科济药业治疗多发性骨髓瘤、浆细胞白血病的通用型BCMA CAR-T细胞产品以及治疗B细胞肿瘤的通用型CD19/CD20 CAR-T细胞产品在中国内地的研发、生产与商业化的独家权利。珠海软银欣创旗下的基金计划以人民币8000万元认购优恺泽新增注册资本，从而持有优恺泽交易完成后注册资本的8%，科济药业于优恺泽的权益将由100%摊薄至92%。 2025年至今，医药板块领先大盘，恒生医疗保健指数累计上涨16.6%，高于恒生指数2.2个百分点。 香港股市医疗健康板块概览 2025年2月恒生医疗保健指数上涨16.1%。上周市值前20个股大多下跌。 上周康方生物逆势上涨5.8%，2月24日，康方生物宣布，依沃西国际市场开发合作伙伴summit therapeutics已与辉瑞(Pfizer)达成临床试验合作，共同推进康方生物自主研发的PD-1/VEGF双抗依沃西与辉瑞多款抗体偶联药物(ADCs)在多种实体瘤中的联合治疗应用。根据协议条款，辉瑞将全面负责本次合作的系列临床试验的开展，Summit将提供依沃西用于该系列临床试验；双方将各自保留其产品的相关权利。2月27日，康方生物宣布其自主研发的差异化HER3 ADC新药AK138D1，治疗晚期恶性肿瘤的1期临床研究在澳洲完成首例受试者入组。根据新闻稿，AK138D1是康方生物首个进入临床阶段的ADC药物。 香港股市医疗健康板块证券发行 2024年共计有12家企业上市(其中3家为依照18A新规上市的未盈利生物医药公司)，共募集金额54.0亿港元。2025年截至目前，共有1家企业上市(其中1家为依照18A新规上市的未盈利生物医药公司)，数量落后去年同期50.0%，金额总数上落后去年同期27.2%。 上周无新增IPO。 2024年共计19家企业完成定增，共募资金额80.9亿港元。2025年至今，共有5家企业完成定增，目前数量与去年同期持平，金额总数上领先去年同期155.7%。 2月25日，晶泰控股-P公告称已完成配售3.42亿股，配售价为每股6.10港元，筹资总额为20.88亿港元。所得款拟用于(i)产品持续迭代升级，提升研发技术能力和解决方案能力，数据生成及模型建立；(ii)产品商业化及业务发展，加强与从事于与本集团类似及互惠互利行业的外部各方及合作伙伴的业务合作，扩大本公司的规模及市场份额；及(iii)潜在机会收购及投资、人才吸引与引进、营运资金补充和一般公司用途。 香港股市医疗健康板块IPO动态 港股医疗板块拟上市企业 No.2 沪深资本市场 沪深股市总体股市概览 本周，上证指数下跌1.7%，沪深300下跌2.2%。 3月2日，国家统计局发布2月份制造业采购经理指数，为50.2%，比上月上升1.1个百分点；非制造业商务活动指数为50.4%，比上月上升0.2个百分点；综合PMI产出指数为51.1%；三大指数均位于扩张区间，我国经济景气水平总体有所回升。 截至目前，2025年上证综指下跌0.9%，沪深300下跌1.1%。 上周，上证医药下跌3.2%，医药中信指数下跌2.8%。 国家医保局2月21日发函，向各地通报中心静脉给药价格综合治理成果，PORT整体费用将从6000元左右降至3300元左右；PICC整体费用将从3000元左右降至1300元左右；CVC整体费用将从800多元降至450元左右。涉及调价的21个省份均已发文调整相关医疗服务项目价格，并自2025年1月1日起实施。 2025年，A股医药板块回暖。上证医药上涨2.1%，领先上证综指3.0个百分点。医药中信指数上涨1.0%，领先沪深300指数2.1个百分点。 沪深股市医疗健康板块概览 2023年医药中信指数下跌6.77%。继2024年12月下跌5.5%后，2025年1月下跌3.8%。上周市值前20个股多数下跌。 艾力斯周内上涨4.83%。2月25日，艾力斯发布业绩公告：报告期内，艾力斯实现营业总收入35.58亿元，较上年同期的20.18亿元同比增长76.29%；归属于母公司所有者的净利润达14.24亿元，同比大幅增长121.08%，上年同期为6.44亿元；归属于母公司所有者的扣除非经常性损益的净利润为13.52亿元，同比增长122.96%。此次业绩的大幅增长主要得益于公司核心产品甲磺酸伏美替尼片（艾弗沙 ®）的出色市场表现。艾弗沙®于2021年3月获国家药品监督管理局批准上市，专门用于治疗第一代或第二代EGFR-TKI治疗后出现耐药的T790M突变NSCLC患者。报告期内，该产品实现销售收入约35.06亿元。 沪深股市医疗健康板块概览 上周，创新药指数下跌2.4%，医疗器械指数下跌4.1%。 百利天恒周内累计上涨11.25%。27日晚，公司发布2024年度业绩快报。公告显示，公司2024年实现营业总收入58.23亿元，同比增长936.31%；归属于母公司所有者的净利润达36.58亿元，同比实现扭亏为盈。业绩大幅增长的主要原因是公司核心产品BL-B01D1（EGFR×HER3双抗ADC）的海外合作伙伴BMS基于合作协议支付了8亿美元不可撤销、不可抵扣的首付款。这笔款项显著提升了公司的营业收入和净利润。 神州细胞周内累计上涨2.93%。24日晚，神州细胞发布业绩快报：报告期内，公司实现营业总收入25.13亿元，同比增长33.13%；营业利润达5.68亿元，同比激增8829.33%；较上年（亏损3.96亿元） 实现全面扭亏为盈。公司称业绩提升主要原因是公司核心产品安佳因®（重组VIII因子）销售稳定。 沪深股市医疗健康板块新股发行 2024年，共4家企业申报IPO。总体数量同比减少71.4%。共募集资金2.47亿元人民币，总体金额同比减少87.7%。 上周无新增IPO。 2024年，有7家企业发行定增，总体数量同比减少65%。累计定增金额4.95亿元人民币，总体金额同比减少67.6%。 上周无新增定增。 沪深股市医疗健康板块IPO动态 沪深股市医疗板块IPO排队企业情况 No.3 NASDAQ 美国股市总体股市概览 上周，美乌谈判牵动市场情绪，最终的谈判破裂加剧美股震荡。 上周周内美股三大指数涨跌不一，标普500指数上周周内累计下跌0.98%，道琼斯工业指数周内累涨0.95%，纳斯达克指数周内累跌3.47%。 2月份标普500指数累计下跌1.42%，道指2月累跌1.58%，纳指累跌3.95%。 2025年截至目前，纳指累计下跌2.4%。 上周纳斯达克生物技术指数 (NBI）逆势回落1.6%。 2月27日，Precigen宣布FDA受理其HPV治疗性疫苗PRGN-2012的上市申请并授予优先审评资格，用于治疗复发性呼吸道乳头状瘤病（RRP），PDUFA日期为2025年8月27日。PRGN-2012 是一种用于治疗成人复发性呼吸道乳RRP的 AdenoVerse® 基因疗法。如顺利获批，PRGN-2012将成为首款和唯一一款治疗RRP的药物。同时，PRGN-2012也将成为首款HPV治疗性疫苗。 2024年截至目前，NBI累涨4.8%，反超纳指7.2个百分点。 美国股市医疗健康板块概览 2025年开年至今，纳斯达克生物技术指数 (NBI)表现稳定，2月总体微调0.3%。上周NBI指数回落，指数成分公司股价涨跌互现。 近日，辉瑞宣布停止血友病B基因疗法Beqvez的全球开发和商业化，这标志着辉瑞进一步退出基因疗法领域。Beqvez是一种一次性治疗方案，去年4月获 FDA 批准，7月起在欧洲上市，每位患者的定价为350万美元。据称“由于患者和医生对血友病基因疗法的兴趣有限”，辉瑞决定停止这一项目。自获批以来，没有患者接受过商业化的Beqvez治疗。 美国股市医疗健康板块新股发行 2024年全年NGS&NGM医疗健康板块累计有30支新股发行，累计募资超55.13亿美金。 2025年截至目前，NGS&NGM医疗健康板块共有8支新股发行，累计募资7.75亿美元，相较去年同期数量上下降20%，金额下降74.5%。 上周2月25日，美股NASDAQ GM板块新增1个IPO。来自Basel Medical新加坡博思医疗集团，公司主营提供广泛的骨科、创伤和运动医学服务，此次IPO募资总额为882万美元。 2024年共有322家企业完成定增，累计募集资金约293.8亿美元。 2025年截至目前共有62家企业完成定增，总体数量上比去年同期下降45.6%，金额较去年同期下降84.1%。 上周共有2起新增定增。其中募资金额最高的是BiomX,一家专注于利用自然和工程化的噬菌体技术开发产品，旨在针对和杀死与慢性疾病相关的特定有害细菌，如囊性纤维化（CF）和糖尿病足骨髓炎（DFO）的临床阶段的公司。公司此次以0.93美元每股的价格募集了263万美元。 医疗健康板块IPO动态 No.4 近期融资事件