诺华再次尝试亨廷顿氏舞蹈症，斥资10亿美元合作开发PTC治疗药物-动脉网

Novartis Tries Again in Huntington’s, Putting Up $1B to Partner on a PTC Therapeutics Drug

MedCity News 等信源发布 2024-12-03 08:38

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Nearly six months ago, PTC Therapeutics reported positive interim data for a drug in mid-stage development for Huntington’s disease, an inherited neurological disorder with limited treatment options and a history of stymied drug research efforts. The PTC drug’s data readout prompted another company to make an unsolicited bid for the program..

近六个月前，PTC Therapeutics报告了亨廷顿舞蹈病中期开发药物的积极中期数据，亨廷顿舞蹈病是一种遗传性神经系统疾病，治疗选择有限，药物研究工作受阻。PTC药物的数据读数促使另一家公司主动投标该项目。。

Rather than jump at the offer, PTC decided to see if others might also be interested in the asset. Novartis has won this competitive process. On Monday, the companies announced the Swiss pharma giant is paying $1 billion up front to license global rights to the PTC Huntington’s drug, PTC518.

PTC决定看看其他人是否也对这项资产感兴趣，而不是直接接受这项提议。诺华赢得了这一竞争过程。周一，两家公司宣布，这家瑞士制药巨头将预付10亿美元，以获得PTC亨廷顿药物PTC518的全球许可。

PTC is still responsible for completing the ongoing placebo-controlled portion of the Phase 2 test of PTC518, and the Warren, New Jersey-based biotech will lead planned discussions with the FDA later this month to discuss the Phase 3 clinical trial. Novartis will take over the program for its pivotal study and is also responsible for the drug’s manufacturing and commercialization, though the deal calls for the companies to share profits from U.S.

PTC仍负责完成PTC518第二阶段测试中正在进行的安慰剂对照部分，总部位于新泽西州沃伦的生物技术公司将于本月晚些时候与FDA进行计划中的讨论，以讨论第三阶段临床试验。。

sales of an approved product — 60% to Novartis and 40% to PTC. The biotech is in line for up to $1.9 billion in milestone payments from Novartis plus royalties from sales of the drug in other markets. This deal is expected to close in the first quarter of 2025..

批准产品的销售额-60%给诺华，40%给PTC。这家生物技术公司将从诺华获得高达19亿美元的里程碑式付款，再加上该药物在其他市场销售的版税。该交易预计将于2025年第一季度完成。。

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Heard at HLTH 2024: Insights from Innovative Healthcare Executives

在HLTH 2024上听到：来自创新医疗保健高管的见解

Executives from Imagine360, Verily, BrightInsight, Lantern, and Rhapsody shared their approaches to reducing healthcare costs and facilitating digital transformation.

来自Imagine360、Verily、BrightInsight、Lantern和Rhapsody的高管分享了他们降低医疗成本和促进数字转型的方法。

By MedCity News

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Huntington’s stems from a genetic mutation that leads to defective versions of huntingtin, a protein key to neuronal function. Mutated huntingtin causes neuronal damage and cell death that affects movement, speech, and swallowing. The disease also leads to behavioral, cognitive, and motor symptoms. Drugs used to treat Huntington’s address certain disease symptoms.

亨廷顿舞蹈症源于一种基因突变，导致亨廷顿舞蹈症的缺陷版本，亨廷顿舞蹈症是神经元功能的关键蛋白质。突变的亨廷顿蛋白会导致神经元损伤和细胞死亡，从而影响运动，言语和吞咽。该疾病还导致行为，认知和运动症状。用于治疗亨廷顿舞蹈症的药物可以解决某些疾病症状。

There are no approved therapies that delay Huntington’s onset or slow its progression..

没有批准的疗法可以延缓亨廷顿舞蹈症的发作或减缓其进展。。

PTC518 is a small molecule that affects the splicing of messenger RNA in order to reduce levels of a target protein. The drug comes from the same PTC technology that yielded Evrysdi, an mRNA-splicing drug that won FDA approval in 2020 as a treatment for the rare muscle disease spinal muscular atrophy (Evyrsdi is marketed by Roche under a partnership).

PTC518是一种小分子，可影响信使RNA的剪接，以降低靶蛋白的水平。该药物来自产生Evrysdi的相同PTC技术，Evrysdi是一种mRNA剪接药物，于2020年获得FDA批准，用于治疗罕见的肌肉疾病脊髓性肌萎缩症（Evyrsdi由罗氏公司合作销售）。

The PTC518 data that prompted deal interest from other companies are interim results from a Phase 2 test showing the once-daily tablet led to dose-dependent lowering of mutant huntingtin protein in the blood and cerebrospinal fluid. The company also reported favorable trends on several clinical assessments of Huntington’s.

引起其他公司交易兴趣的PTC518数据是第二阶段测试的中期结果，该测试显示每日一次的片剂导致血液和脑脊液中突变亨廷顿蛋白的剂量依赖性降低。该公司还报告了几项亨廷顿舞蹈症临床评估的有利趋势。

PTC said the drug was safe and well tolerated by study participants. Additional Phase 2 data are expected in the second quarter of 2025..

PTC表示，研究参与者对该药物安全且耐受性良好。预计2025年第二季度将有更多的第二阶段数据。。

Huntington’s drug research has yielded many disappointments. In 2021, Roche halted a Phase 3 test of tominersen at the recommendation of the trial’s independent data monitoring committee. The drug candidate, an antisense oligonucleotide from Roche’s partnership with Ionis Pharmaceuticals, is designed to bind to mRNA in order to prevent production of mutant huntingtin.

亨廷顿的药物研究产生了许多失望。2021年，罗氏根据试验独立数据监测委员会的建议，停止了tominersen的3期试验。候选药物是罗氏与Ionis Pharmaceuticals合作的反义寡核苷酸，旨在与mRNA结合，以防止突变亨廷顿蛋白的产生。

Roche has not given up on this drug entirely. In 2022, the pharma giant revealed plans for a new Phase 2 trial in younger Huntington’s patients with less severe disease. More recently, Sage Therapeutics reported it would stop dosing of dalzanemdor, a small molecule that had reached Phase 2 testing in Huntington’s.

罗氏并没有完全放弃这种药物。2022年，这家制药巨头宣布计划对病情较轻的年轻亨廷顿舞蹈症患者进行新的2期临床试验。最近，Sage Therapeutics报道，它将停止服用达尔扎涅姆多（dalzanemdor），达尔扎涅姆多是一种小分子，已在亨廷顿舞蹈病（Huntington's）进行2期测试。

The November decision was based on the recommendation of independent observers of the clinical trial..

11月的决定是基于临床试验独立观察员的建议。。

Novartis has also experienced disappointment in Huntington’s. Its internally discovered drug candidate, branaplam, was initially developed as a treatment for spinal muscular atrophy before the company changed the small molecule’s focus to Huntington’s. In 2022, Novartis suspended dosing of branaplam after findings suggested the experimental drug may have caused peripheral neuropathy in some study participants.

诺华在亨廷顿也经历了失望。其内部发现的候选药物branaplam最初是作为治疗脊髓性肌萎缩症的药物开发的，后来该公司将小分子的重点转移到亨廷顿氏病。2022年，诺华停止服用布拉那普兰，因为研究结果表明该实验药物可能在一些研究参与者中引起周围神经病变。

The mid-stage study was subsequently discontinued..

中期研究随后停止。。

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Health Executives on Digital Transformation in Healthcare

卫生管理人员对医疗保健数字化转型的看法

Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare.

听取来自Quantum Health、Surescripts、EY、Clinical Architecture和Personify Health的高管分享他们对医疗保健数字转型的看法。

By MedCity News

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In a note sent to investors, Leerink Partners analyst Joseph Schwartz said Novartis seems like a logical partner for PTC518, as branaplam’s approach to Huntington’s was similar to the PTC drug. In a Monday conference call, PTC executives said besides Novartis’s competitive financial offer, the pharma giant was selected because of its experience developing neuroscience drugs.

Leerink Partners分析师约瑟夫·施瓦茨（JosephSchwartz）在发给投资者的一份说明中表示，诺华似乎是PTC518的合理合作伙伴，因为布拉纳普兰治疗亨廷顿舞蹈症的方法类似于PTC药物。在周一的一次电话会议上，PTC高管表示，除了诺华具有竞争力的财务报价外，这家制药巨头之所以被选中，是因为其开发神经科学药物的经验。

William Blair analyst Sami Corwin wrote in an investor note that the deal makes financial sense for PTC, which last week struck a $150 million deal to sell the priority review voucher (PRV) it received for the recent FDA approval of gene therapy Kebilidi for an inherited enzyme deficiency. She said PTC now has additional funds to support its drug portfolio without needing to also pay for a pivotal clinical trial..

。她说，PTC现在有额外的资金来支持其药物组合，而不需要支付关键的临床试验费用。。

“We see the $1 billion upfront payment, in addition to the $150 million PRV sale, as providing the company with sufficient financial funds to reach cash flow break even in the future without the need to raise additional funds,” Corwin said. “In addition, the deal provides PTC with a strategic partner that has experience developing drugs for CNS-based diseases, which could accelerate the clinical development and bolster the commercialization of PTC518, in our view.

科尔温说：“我们认为，除了1.5亿美元的PRV销售外，10亿美元的预付款为该公司提供了足够的财务资金，使其在未来实现现金流收支平衡，而无需筹集额外资金。”。“此外，该交易为PTC提供了一个战略合作伙伴，该合作伙伴具有开发中枢神经系统疾病药物的经验，我们认为这可以加速临床开发并促进PTC518的商业化。

We also see the deal as validating PTC518 and PTC’s splicing platform.”.

我们还认为该交易验证了PTC518和PTC的拼接平台。”。

In the hands of a big pharma company with neuroscience experience, William Blair gives PTC518 a 50% probability of success, modeling peak revenue of $5 billion, about $1.43 billion of which would go to PTC. In the nearer term, PTC is preparing to meet with the FDA later this month to discuss the design of the Phase 3 study.

在一家拥有神经科学经验的大型制药公司手中，威廉·布莱尔（WilliamBlair）为PTC518提供了50%的成功概率，模拟了50亿美元的峰值收入，其中约14.3亿美元将流向PTC。近期内，PTC准备在本月晚些时候与FDA会面，讨论第三阶段研究的设计。

Schwartz noted that PTC is seeking alignment with the regulator on using lowering of huntingtin levels as a potential surrogate endpoint to support accelerated approval. This endpoint could also serve as the primary endpoint of a Phase 3 study supporting a traditional approval..

施瓦茨指出，PTC正在寻求与监管机构达成一致，将降低亨廷顿蛋白水平作为支持加速批准的潜在替代终点。该终点也可以作为支持传统批准的第三阶段研究的主要终点。。

Photo: Jolygon, Getty Images

照片：Jolygon，Getty Images

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亨廷顿舞蹈病

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