7月 30, 2023
  • DTx Pharma 是一家处于临床前阶段的生物技术公司,利用其专有的脂肪酸配体偶联寡核苷酸 (FALCON) 平台,专注于开发用于神经科学领域适应症的小干扰核酸(siRNA)疗法。
  • 此项交易包括 DTx-1252(一种治疗神经肌肉疾病腓骨肌萎缩症1A型(CMT1A)的潜在疗法)和另外两个针对其他神经科学领域适应症的临床前项目
  • 本次收购将 DTx 的 FALCON 平台纳入诺华 siRNA技术平台,从而增强诺华在基于 RNA 的疗法方面的研发能力

诺华日前宣布收购 DTx Pharma,这是一家位于美国圣地亚哥、处于临床前阶段的生物技术公司,致力于利用其专有的脂肪酸配体偶联寡核苷酸( FALCON )平台开发用于神经科学领域适应症的小干扰核酸(siRNA)疗法。

DTx 的先导项目DTx-1252靶向作用于导致 CMT1A 的根源--PMP22蛋白过度表达,这种蛋白会导致支撑和隔离周围神经系统神经的髓鞘功能异常。DTx-1252 最近获得美国食品药品监督管理局(FDA)孤儿药资格认定,1 它能减少雪旺细胞中PMP22蛋白的表达,被靶向的雪旺细胞能够参与周围神经发育、维护和功能调节。FALCON 平台通过将 siRNA 与天然脂肪酸偶联,改善其生物分布,使得特定组织和细胞能够胞内摄入(偶联体),从而提升药物靶向能力。2

诺华生物医学研究中心(NIBR)总裁Fiona Marshall说:“多年以来针对神经肌肉疾病或者其他神经系统疾病的治疗选择一直很有限,我们期待继续开发DTx的治疗项目,为患者带来新的希望。我们也很高兴能将 DTx 的 FALCON 技术引入诺华,并探索其向肝外组织递送药物的潜力。”

腓骨肌萎缩症(CMT)是一组影响神经系统的遗传性疾病,其中CMT1A是最常见的亚型,在美国和欧洲约有 15 万名患者,3属于慢性进展的周围神经系统退行性疾病,会导致进行性肌肉萎缩、神经性疼痛和行走困难,并严重影响生活质量。由于目前还没有针对 CMT1A 潜在遗传病因的获批疗法,DTx-1252 有可能成为治疗该疾病的同类首创药物。4

除 DTx-1252 外,此项交易还为诺华带来了另外两个针对其他神经科学领域适应症的临床前项目,在诺华现有的xRNA能力基础上扩展了 siRNA技术平台,这也是诺华的技术平台之一。该协议彰显了诺华聚焦五大核心治疗领域之一—神经科学的决心,以期为神经肌肉疾病患者寻求创新疗法。

诺华生物医学研究中心神经科学领域全球负责人Robert Baloh表示:“越来越多可用的治疗平台库让我们得以继续研发新型、高价值的药物,特别是在疾病的生物学机制已经明确的情况下。我们期待继续开发 DTx 的针对 CMT1A 的潜在同类首创药物,并且希望能为罹患这种使人衰弱的神经肌肉疾病的患者带来新的治疗选择。”

根据此项协议条款,诺华将支付 5 亿美元的预付款,并在交易达到预先规定的里程碑后支付额外款项。


关于诺华中国
诺华公司中文名取意“承诺中华”,即通过不断创新的产品和服务,提高中国人民的健康水平和生活质量。自1987年以来,诺华已有近100款创新药及新适应症在华获批。诺华在中国的业务包括诺华创新药物(中国)与山德士(中国),并在北京、上海和江苏设立了研发机构。诺华在中国拥有超过8000名员工。如需更多信息,敬请访问诺华中文网站:www.novartis.com.cn

References

[1]U.S. Food & Drug Administration. "Orphan Drug Designations and Approvals: Double-stranded small-interfering ribonucleic acid (siRNA) comprised of an antisense strand complementary to a targeted sequence within human PMP22 messenger RNA and a nucleotide sense strand linked to a fatty acid motif." Available at: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cf…. Accessed July 7, 2023.

[2]DTx Pharma. Available at: https://dtxpharma.com/platform/. Accessed April 19, 2023.

[3]Data on file.

[4]“Charcot-Marie-Tooth disease type 1A.” NIH Genetic and Rare Diseases (GARD) Information Center. Available at: https://rarediseases.info.nih.gov/diseases/1245/charcot-marie-tooth-dis…. Accessed April 19, 2023.

This media update contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for any investigational or approved products resulting from the DTx-1252 program or the FALCON platform, the acquisition of DTx Pharma, or regarding potential future revenues from DTx-1252. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that any investigational or approved products resulting from the DTx-1252 program or the FALCON platform will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Neither can there be any guarantee expected benefits or synergies from this transaction will be achieved in the expected timeframe, or at all, nor can there be any guarantee that the DTx-1252 program will be commercially successful in the future. In particular, our expectations regarding DTx-1252 or the transaction described in this media update could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update as a result of new information, future events or otherwise.