Tofacitinib Citrate

Drug-device combination leverages Biora’s NaviCap™ platform to deliver tofacitinib directly to colonic tissue as a potential treatment for ulcerative colitis BT-600 was well tolerated and met all trial objectives, demonstrating the NaviCapplatform’s ability to deliver therapeutics directly to the colon Drug absorption in colonic tissue extended to distal colon, suggesting pan-colonic delivery Company to host virtual event with key opinion leaders on July 17 SAN DIEGO, July 01, 2024 (GLOBE NEWSWIRE) -- Biora Therapeutics, Inc. (Nasdaq: BIOR), the biotech company reimagining therapeutic delivery, today shared positive topline results from its clinical trial of BT-600, an orally administered drug-device combination in development for the potential treatment of patients with ulcerative colitis (UC). BT-600 leverages Biora’s ingestible NaviCap™ device to deliver a proprietary liquid formulation of tofacitinib directly to the colon. Results from this Phase 1 clinical trial involving 48 healthy volunteers met all trial objectives, with demonstrated drug absorption in colonic tissue that extended to the distal colon, suggesting pan-colonic delivery. Daily dosing with BT-600 was well tolerated by all participants. “Successful completion of our Phase 1 clinical trial is an important milestone for Biora,” said Ariella Kelman, MD, Chief Medical Officer of Biora Therapeutics. “All study objectives were met, and we confirmed that the NaviCap platform can deliver tofacitinib topically to the colon, with lower peak systemic exposure than with conventional oral delivery. These results support our plan to advance BT-600 into our Phase 1b clinical trial in patients with UC.” “We are extremely encouraged by the results from this trial, which demonstrate the NaviCap platform’s ability to deliver drug to the location of disease, where it’s needed,” said Adi Mohanty, Chief Executive Officer of Biora Therapeutics. “Our anatomically targeted approach has the potential to improve the efficacy of JAK inhibitors and other drug classes. We envision a portfolio of NaviCap-delivered therapeutics unlocking new treatment potential for patients with GI diseases.” “I would like to thank the study participants, clinicians, and our Biora team for conducting such a well-executed trial,” continued Mr. Mohanty. “Our team continues to execute at a high level as we meet our NaviCap platform milestones, while the BioJet™ platform is also progressing well and is on track to meet our previously stated goals.” Summary of Key BT-600 Phase 1 Trial Results Results from the Phase 1 clinical trial demonstrate a pharmacokinetic (PK) pro with drug delivery and absorption in the colon for both single and multiple ascending dose (SAD/MAD) cohorts. First evidence of systemic absorption of tofacitinib was at six hours, consistent with colonic (vs. upper gastrointestinal) delivery. Maximal levels in the trial occurred at eight to ten hours vs. 30 minutes for conventional oral tofacitinib in other trials. Maximal systemic drug exposure was three to four times lower than that seen with conventional oral tofacitinib in other trials, demonstrating the NaviCap platform’s ability to deliver locally to the colon and limit systemic drug exposure. The distribution of colon tissue exposure suggests that pan-colonic delivery of tofacitinib was achieved. Sites in the distal colon were biopsied, following delivery of tofacitinib in the proximal colon, for evidence of tissue drug exposure Biopsy results provided evidence of drug exposure extending to the distal colon, at common sites of disease. Post-retrieval device analysis further confirmed that NaviCap devices accurately delivered drug in the colon, with 100% of devices (SAD) and 98% of devices (MAD) detecting colon entry. NaviCap devices were well tolerated by participants in both the SAD and MAD cohorts. Virtual Event Details The company will host a KOL event with members of management and its Clinical Advisory Board to provide additional details regarding the Phase 1 trial and plans for the next phase of clinical development. Date: Wednesday, July 17, 2024 Time: 2:00 PM Eastern / 11:00 AM Pacific time Live Webcast: Attendees may register in advance using the webcast link above. A replay will be available online following the event. Phase 1 Clinical Trial Design The objectives of this Phase 1 randomized, double-blind, placebo-controlled, single and multiple ascending dose (SAD/MAD) clinical trial were to evaluate the safety and pharmacokinetics of BT-600 when administered orally in healthy adult participants. The trial, which was conducted in the United States, consisted of two parts: The first part was comprised of 24 participants receiving a single ascending dose of BT-600 with tofacitinib at 5 mg or 10 mg doses or placebo. The second part was comprised of 24 participants receiving multiple ascending-doses of BT-600 with tofacitinib at 5 mg or 10 mg doses or placebo daily for 7 days. The trial is listed at clinicaltrials.gov (NCT06275464). The “other trials” referred to in the summary of the Phase 1 clinical trial results above were conducted at different times, with differences in trial design and patient populations. As a result, cross-trial comparisons cannot be made and no head-to-head clinical trials have been conducted. About BT-600 BT-600 is a drug/device combination of Biora’s NaviCap™ ingestible drug delivery device with a proprietary liquid formulation of tofacitinib, for the potential treatment of moderate to severe ulcerative colitis. The NaviCap device is orally administered and has been designed for anatomically targeted therapeutic delivery directly to the colon in this application. About the NaviCap™ Targeted Oral Delivery Platform Biora's NaviCap targeted oral therapeutics platform utilizes a novel approach that could improve patient outcomes by enabling delivery of therapeutics directly to the site of disease, increasing therapeutic activity in tissue while reducing systemic uptake. For the 1.8 million patients in the United States who suffer from inflammatory bowel disease (IBD), existing therapeutics offer less than ideal efficacy, likely because of the challenges with safely achieving sufficient drug activity in the affected tissues. Research has shown that targeted delivery of therapeutics has the potential to improve patient outcomes in IBD. The NaviCap platform uses an ingestible device designed for targeted delivery of therapeutics to improve treatment of ulcerative colitis. Once swallowed, Biora’s GItrac™ autolocation technology enables the device to autonomously identify targeted locations in the GI tract and release a therapeutic dose of up to 500µl. Studies of the NaviCap device in healthy volunteers and patients with ulcerative colitis demonstrated successful delivery to the colon regardless of variable GI conditions, in both fasted and fed states. About Ulcerative Colitis Ulcerative colitis (UC) is a type of IBD that causes chronic inflammation and damage to the colon. Common symptoms include abdominal pain, increased bowel movements, stool urgency, and rectal bleeding. Despite the availability of advanced treatments for UC, including biologics, immunomodulators, and targeted synthetic small molecules, only about 40% of patients achieve clinical remission in induction trials. Surgical intervention is needed in approximately 20% of UC patients, with up to 10% of patients requiring surgical removal of the colon. About 1.5 million people are affected with UC in the United States alone, and ~40,000 new cases are diagnosed each year. About Biora Therapeutics Biora Therapeutics is reimagining therapeutic delivery. By creating innovative smart pills designed for targeted drug delivery to the GI tract, and systemic, needle-free delivery of biotherapeutics, the company is developing therapies to improve patients’ lives. Biora is focused on development of two therapeutics platforms: the NaviCap™ targeted oral delivery platform, designed to improve outcomes for patients with inflammatory bowel disease through treatment at the site of disease in the gastrointestinal tract, and the BioJet™ systemic oral delivery platform, designed to replace injection for better management of chronic diseases through needle-free, oral delivery of large molecules. For more information, visit bioratherapeutics.com or follow the company on LinkedIn or X. Safe Harbor Statement or Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, which statements are subject to substantial risks and uncertainties and are based on estimates and assumptions. All statements, other than statements of historical facts included in this press release, including statements concerning the progress and future expectations and goals of our research and development, preclinical and clinical trial activities, including those involving BT-600 and our NaviCap platform, and partnering and collaboration efforts with third parties, are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “envision,” “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “anticipate,” “forward,” “believe,” “design,” “estimate,” “predict,” “potential,” “plan,” “goal(s)” “target,” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These statements reflect our plans, estimates, and expectations, as of the date of this press release. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the forward-looking statements expressed or implied in this press release. Such risks, uncertainties, and other factors include, among others, our ability to innovate in the field of therapeutics, our ability to make future FDA filings and initiate and execute clinical trials on expected timelines or at all, our ability to obtain and maintain regulatory approval or clearance of our products on expected timelines or at all, our plans to research, develop, and commercialize new products, the unpredictable relationship between preclinical study results and clinical study results, our expectations regarding allowed patents or intended grants to result in issued or granted patents, our expectations regarding opportunities with current or future pharmaceutical collaborators or partners, our ability to raise sufficient capital to achieve our business objectives, our ability to maintain our listing on the Nasdaq Global Market, and those risks described in “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Annual Report on Form 10-K for the year ended December 31, 2023 filed with the Securities and Exchange Commission (SEC) and other subsequent documents, including Quarterly Reports on Form 10-Q, that we the SEC. Biora Therapeutics expressly disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law. Investor Contact Chuck Padala Managing Director, LifeSci Advisors IR@bioratherapeutics.com (646) 627-8390 Media Contact Liz Robinson CG Life lrobinson@cglife.com

跑车百米加速拼的是发动机的性能，一家Biotech一年内上市两款源头创新药，考验的什么？ 一款中国创新药从拿到上市批文到首方落地需要多久？在Biotech商业化能力越来越受到瞩目的时代，答案一定是越快越好。 迪哲医药再一次成为这场考试中最快交卷的，仅用了2天，戈利昔替尼就实现了从拿到药品注册证书到首方落地。 巧合的是，上一个保持非自有工厂发货最快行业纪录的，同样是迪哲，其首款创新产品舒沃替尼在2023年实现了“4天首方落地”。 或许是“一回生二回熟”，有了上一次的经验，迪哲在戈利昔替尼的商业化运营上，让外界读出了一种“志在必得”：将这款近十年来获批的首个针对复发难治性PTCL创新药以最快的速度落地。 成立六年实现两款全球首创/潜在同类最佳新药上市，迪哲开挂了？ 两次从0到1 目前全球已上市一代、二代JAK抑制剂大多作用于自免领域，如诺华的芦可替尼，辉瑞的托法替布、阿布昔替尼，艾伯维的乌帕替尼，在特应性皮炎、类风湿性关节炎等领域均有所斩获。 迪哲的戈利昔替尼作为第二代JAK抑制剂，却另辟蹊径，与前辈们最显著的特点在于差异化的适应证布局，并未选择自免赛道，而是首创了通过靶向JAK/STAT通路来治疗外周T细胞淋巴瘤（PTCL），这也使得戈利昔替尼成为该治疗领域全球首个且唯一的全新作用机制药物。 戈利昔替尼的“新”体现在两个层面：新机制与新结构。 迪哲医药CEO张小林表示，迪哲研发团队最早发现并通过国际多中心临床验证了JAK/STAT通路是治疗外周T细胞淋巴瘤的高潜力靶点。同时，戈利昔替尼独特的创新分子结构设计，实现了对JAK1的极高选择性和长半衰期，可对肿瘤持续稳定强抑制的同时大幅提升安全耐受。 在张小林看来，正是基于创新的作用机制和分子设计，临床上才取得了突破性疗效。发表于《柳叶刀∙肿瘤学》杂志的全球关键性注册临床研究“JACKPOT8 B部分”数据表明，戈利昔替尼单药治疗复发/难治性（r/r） PTCL的客观缓解率（ORR）为44.3%，完全缓解（CR）率为23.9%，中位缓解持续时间（DoR）长达20.7个月，均是既往靶向治疗方案的近2倍。 现阶段，PTCL存在较大的治疗缺口，戈利昔替尼的顺利获批将很好地填补临床上的空白。张小林告诉E药经理人，戈利昔替尼上市后有望冲破PTCL治疗瓶颈，改变当前PTCL的诊疗格局。PTCL恶性程度高，且异质性强、发病机制复杂，在所有非霍奇金淋巴瘤中生存率最低，一线治疗主要依赖CHOP为基础的四联化疗，治疗效果不理想，且患者复发风险极高，而r/r PTCL的生存预后更差：3年生存率仅为23%，中位总生存期（OS）仅5.8个月。 近十年来未有针对PTCL的创新药上市。值得注意的是，在今年4月的CSCO指南大会上，戈利昔替尼凭借优异的临床数据，已提前获《CSCO淋巴瘤诊疗指南（2024版）》推荐。 随着产品上市，JAK抑制剂在T细胞淋巴瘤的最终表现，也将通过商业化数据给出答案。此外，张小林表示，戈利昔替尼正在推进海外注册的工作，争取以最快的速度让全球患者享受到这一创新成果，为全球患者赢得全新的治疗希望。 实现从0到1突破的戈利昔替尼，是迪哲在差异化创新战疫中打出的第二枚“子弹”，打头炮的舒沃替尼，突破肺癌难治靶点在国内首发上市后，同样走在冲击海外市场的路上。 不久前结束的ASCO大会上，迪哲医药以口头报告形式公布了舒沃替尼针对经EGFR 20号外显子插入突变（Exon20ins）非小细胞肺癌（NSCLC）首个国际多中心注册临床研究“悟空1B”的初步分析结果，研究达到主要终点，舒沃哲高效低毒，展现全球同类最佳潜力。 2024年4月，该药物一线治疗EGFR Exon20ins NSCLC获美国FDA突破性疗法认定，是该领域全球唯一全线获FDA突破性疗法认定的药物。 作为迪哲成立6年后结出的第一颗果实，舒沃替尼于2023年8月在国内获批上市，是首款针对这一适应证的中国创新药。 早在2022年，舒沃替尼就已成为肺癌领域首个获中美两地监管机构双“突破性疗法认定”的中国创新药。目前舒沃替尼已被作为I级推荐用药纳入《CSCO非小细胞肺癌诊疗指南（2024版）》，是该指南中针对EGFR exon20ins NSCLC的唯一I级推荐方案。 值得注意的是，从国内首例临床研究受试者入组，到正式获批上市，舒沃替尼仅用时不到4年，甚至超越了第三代EGFR-TKI奥希替尼在国内的进展速度（4.5年）。 张小林直言，自迪哲成立以来，始终聚焦在自身有竞争优势的领域。“我们用自己优势去跟别人竞争，而不是今天流行什么去做什么，盲目追逐热点本质就变成已失去自身优势。”也正是基于这样的核心战略，迪哲立足于推进在科学上有优势、有把握的产品进行研发，并把它们尽早推向市场，加速实现自我造血。在张小林看来，目前迪哲医药的每一条研发管线都极具BIC/FIC潜力。 商业化加速 摘“U”还远吗 自国家药监局下发戈利昔替尼的上市许可那一刻起，迪哲就全面开启了“24小时不间断”商业化上市推进。从定稿说明书、包材印刷、生产、包装、检验、质量放行、全国配送、上架、开单，公司和外部合作伙伴日夜兼程无缝接力，才能在短短2天时间里把药物送达全国各地。此前，同行非自由工厂发货的最快速度也要20天。 对于尚未盈利的创新药企来说，何时能够通过商业化扭亏为盈是如今资本寒冬之下，愈发被关注的话题。2021年登陆科创板的迪哲，目前尚未实现盈利。 对于尚未实现盈利的创新药企来说，通过高效的商业化运营实现亏损收窄乃至扭亏为盈是市场所关心的。 2023年可以作为分水岭，国内数家biotech公司宣布扭亏为盈，如复宏汉霖、康方生物、和黄医药等。对创新药企来说，想在未来的竞争中取得领先，既要在研发端具备以临床价值为导向的高质量创新实力，开发具有差异化竞争优势的产品，又要在市场端具备高效的商业化能力。 如今迎来第二款商业化产品的迪哲何时能实现由亏转盈？张小林此前曾表示，“在快速形成自我造血机制后，逐步向更多适应证和管线拓展，以实现更大的商业化成功。”张小林这样说道。 上市即将满一年的舒沃替尼在迪哲医药2023年年报与2024年一季报中，都交出了颇为出色的成绩单。 具体来看，2023年，舒沃替尼8月上市后，商业化首年实现销售收入约9129万元。到了2024年一季度，销售收入达到8132万元，环比增长近60%，商业化成绩单可圈可点。 据迪哲医药首席商务官吴清漪透露，舒沃替尼上市首月销售额就突破了4000万元，这也是罕见靶点新药上市首月销售额的新纪录。自2023年8月到2024年3月，7个月的时间销售额突破1.8亿，在市场看来迪哲医药的商业化能力颇有潜力。 戈利昔替尼拿到新药批文后2天首方落地的速度也刷新了一年前舒沃替尼创下的记录。吴清漪告诉E药经理人，去年舒沃替尼获批上市后的第4天实现首方落地，刷新了国内非自有工厂发货最快首方落地的行业纪录。如今戈利昔替尼再这一基础上将时间又缩短了两天，其自身高效的商业化运营正在得到验证。 创新药商业化是一项很复杂的系统工程，需要整体布局，每个核心环节都需要反复打磨。在这之中，最重要的是建立一只有战斗力的团队，才能高效地实现目标。在吴清漪看来，迪哲拥有一只整建制的超强战斗力的商业化团队。 一款源头创新产品的技术壁垒，避免了同质化产品扎堆的情况，能够为产品建立良好的市场竞争格局，并奠定商业化成功的基础。随着戈利昔替尼的上市，再一次验证迪哲商业化实力的时候到了。 与此同时，创新药进医保是如今国内医药市场中，任何一家创新药企业不能回避的问题。对于创新药来说，最大程度提高患者可及性的关键是医保准入。舒沃替尼的上市错过了2023年医保目录6月30日的“关门”时间。 而现在来看，舒沃替尼与戈利昔替尼都满足了6月13日新版医保目录征求意见稿的门槛。迪哲医药表示，积极争取将两款药物纳入国家医保目录是公司下一阶段的目标。 对于迪哲而言，已上市的两款产品都具有BIC/FIC的创新性，而源头创新正是商业化成功的护城河，能够有效避免同质化扎堆的局面，创造出良好的市场竞争格局。或许迪哲的经验也给正在同质化靶点里“内卷”的biotech公司指了另一条路：与其在同质内卷中艰难喘息，不如放手大胆去差异化创新里厮杀。  编者按  【做难而正确的事】 大多数人都会做简单的事、获取轻易可摘得的果实；而总有些人明知面对的是一条布满荆棘的路，却依然走下去，选择去做那些不坚持很容易，坚持会很难的事。正如稻盛和夫所言，真正的强大，就是做难而正确的事。对于如今产业内的生物医药公司而言，坚持创新是一件难而正确的事。 寒冬之下，如何让公司长久持续的发展；如何把最优势的资源最合理、有效地聚焦；如何建立一支高效、敏捷的创新团队来顺应未来的变化……都是摆在企业管理者面前的挑战和难题。 面对复杂多变的内外部环境，或加速转型、或稳步推进研发、或走好国际化出海……国内的医药企业该如何保持定力，穿越低谷，进入全新成长周期？ E药经理人推出【做难而正确的事】专题，希望通过讲述医疗健康领域企业在正确的战略指引下，克服重重磨难取得成功的故事，与中国的医药企业家们共同探讨如何在产业动荡期突破制约我国医药产业的快速发展的“屏障”，直面机遇和挑战，坚持走“难而正确”之路，推动公司的创新、转型和高速发展。 精彩推荐 CM10 | 集采 | 国谈 | 医保动态 | 药审 | 人才 | 薪资 | 榜单 | CAR-T | PD-1 | mRNA | 单抗 | 商业化 | 国际化 ｜ 猎药人系列专题 | 出海 启思会 | 声音·责任 | 创百汇 | E药经理人理事会 | 微解药直播 | 大国新药 | 营销硬观点 | 投资人去哪儿 | 分析师看赛道 | 药事每周谈 | 医药界·E药经理人 | 中国医药手册 创新100强榜单 | 恒瑞 | 中国生物制药 | 百济 | 石药 | 信达 | 君实 | 复宏汉霖 ｜翰森 | 康方生物 | 上海医药 | 和黄医药 | 东阳光药 |  荣昌 | 亚盛医药 | 齐鲁制药 | 康宁杰瑞 | 贝达药业 | 微芯生物 | 复星医药 ｜再鼎医药｜亚虹医药 跨国药企50强榜单 | 辉瑞 | 艾伯维 | 诺华 | 强生 | 罗氏 | BMS | 默克 | 赛诺菲 | AZ | GSK | 武田 | 吉利德科学 | 礼来 | 安进 | 诺和诺德 | 拜耳 | 莫德纳 | BI | 晖致 | 再生元