Dordaviprone

一、新药快讯 1、自噬抑制剂联合高剂量CDK4/6抑制剂治疗晚期乳腺癌 对于激素受体阳性乳腺癌，内分泌治疗联合CDK4/6抑制剂是一线标准治疗，但是20%患者在6个月内耐药，2024年12月27日，中山大学宋尔卫团队在Med杂志发布了一项研究，使用羟氯喹联合高剂量CDK4/6抑制剂，剂量扩大的患者群体，联合疗法6个月的临床获益率达到了90%，明显高于以往临床研究的获益率，这个组合疗法估计将会进一步扩大入组患者。 2、奥拉帕利新适应症获批，用于BRCA基因突变早期乳腺癌辅助治疗 2025年1月2日，PARP抑制剂奥拉帕利在国内获批新的适应症，用于接受过新辅助或辅助化疗的携带有害或疑似有害胚系 BRCA 突变、人表皮生长因子受体 2阴性早期高风险乳腺癌成人患者的辅助治疗。这个适应症是基于一项三期临床试验结果，相比安慰剂，奥拉帕利将浸润性乳腺癌复发、新发癌症或死亡的风险降低了42%，死亡风险降低了32%。 3、胶质瘤抗癌新药ONC201，30%的患者肿瘤病灶显著缩小或消失 近日，一款代号为ONC201的药物向美国食品药品监督管理局FDA发起上市申请，用于治疗复发性H3K27M突变型弥漫性胶质瘤。基于相应的临床试验结果，这款药对于高级别胶质瘤患者的治疗应答率达到了20%，疾病控制率为40%。对于低级别胶质瘤患者，ONC201的治疗应答率达到了30%，疾病控制率达到了44%，当然需要注意这款药需要患者明确特定的基因突变，否则是没有上述治疗效果的。 二、新药快讯 1、2024年乳腺癌热词公布，抗体偶联药开创新时代 2024年乳腺癌年终盘点暨临床热词发布会上，根据临床领域重要期刊的重磅研究、药物、靶点出现频次和学术权重，评选出2024年乳腺癌领域的10大热词，德曲妥珠单抗荣获榜首，TROP-2位于第二位，帕博利珠单抗位于第三位，此外 ，DESTINY-Breast06、Dato-DXd 等均进入年度热词榜单。这个榜单体现了抗体偶联药的治疗趋势，即将成为乳腺癌治疗的主要治疗策略。

Potential Approval in Q3 2025 in Recurrent H3 K27M-Mutant Diffuse Glioma Submission Plan Follows Productive and Collaborative Pre-NDA Interactions with FDA Company to Host Conference Call on Tuesday, December 10 at 8:30 AM ET DURHAM, N.C., Dec. 09, 2024 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ: CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today announced that, following extensive dialogue with the U.S. Food and Drug Administration (FDA), the Company plans to submit a complete New Drug Application (NDA) seeking accelerated approval for dordaviprone as a treatment for recurrent H3 K27M-mutant diffuse glioma in the United States before year-end. “We expect that, if approved, dordaviprone will fundamentally change the treatment landscape for patients suffering from this lethal form of brain cancer who have extremely limited treatment options. We have worked collaboratively with the U.S. FDA, disease experts and patient advocates throughout the year to potentially accelerate access to dordaviprone for this patient community,” said Mike Andriole, Chief Executive Officer of Chimerix. “In anticipation of a potential approval, we have bolstered our commercial leadership team and will be ready for a U.S. launch as early as the third quarter of 2025, pending application acceptance and Priority Review, if granted.” “As a pediatric oncologist, this program is particularly meaningful given the impact a potential approval would have on children and young adults devastated by this disease. We are confident that the data generated to date could support an accelerated approval for this urgent unmet medical need,” said Allen Melemed M.D., Chief Medical Officer of Chimerix. “H3 K27M mutant gliomas are extremely aggressive and affect over 2,000 patients annually in the United States. If successful, dordaviprone would be the first FDA-approved therapy for this lethal disease, as well as one of the first molecularly defined approvals for any high-grade glioma.” The following recent program milestones and additional supportive data were extensively discussed with the FDA and will be included in the NDA: Substantial enrollment of the Phase 3 ACTION studyPhase 2 objective response rate of the 50-patient primary efficacy analysis assessed by blinded independent central review (BICR) as the primary basis of efficacy in the NDASeveral response assessments, including the most contemporary response assessment criteria for gliomas, Response Assessment in Neuro-Oncology 2.0 (RANO 2.0), under which dordaviprone demonstrated an objective response rate of 28%, a median duration of response of 10.4 months and a median time to response of 4.6 months Additional clinical data sets and patient narratives supportive of the primary efficacy analysisClinical and nonclinical demonstration of dordaviprone-driven reversal of the central hallmark of H3 K27M-mutant glioma, H3K27 trimethyl lossComprehensive safety database of glioma patients and healthy volunteers that supports a favorable benefit/risk profileComprehensive clinical pharmacology and chemistry, manufacturing, and controls (CMC) studies Chimerix will request Priority Review for the NDA. If granted, the resulting six-month FDA review period is expected to result in a potential initial Prescription Drug User Fee Act (PDUFA) action date in the third quarter of 2025. Dordaviprone has received Rare Pediatric Disease Designation for H3 K27M-mutant glioma and is eligible to apply for a Rare Pediatric Disease Priority Review Voucher (PRV). Chimerix intends to apply for a Rare Pediatric Disease PRV in the upcoming NDA submission. Conference Call and WebcastChimerix will host a conference call and live audio webcast on Tuesday, December 10 at 8:30 a.m. ET. To access the live conference call, please dial 646-307-1963 (domestic) or 800-715-9871 (international) at least five minutes prior to the start time and refer to conference ID 8870160. A live audio webcast of the call will also be available on the Investors section of Chimerix’s website, www.chimerix.com. An archived webcast will be available on the Chimerix website approximately two hours after the event. About Chimerix Chimerix is a biopharmaceutical company with a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The Company’s most advanced clinical-stage development program, dordaviprone, is in development for H3 K27M-mutant glioma. The Company is conducting Phase 1 dose escalation studies of ONC206 to evaluate safety and PK data. About DordaviproneDordaviprone (ONC201) is a novel first-in-class small molecule imipridone that selectively targets the mitochondrial protease ClpP and dopamine receptor D2 (DRD2). Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include those relating to, among other things: the possible regulatory path forward for dordaviprone, including the potential to seek accelerated approval, Priority Review, rare pediatric disease Priority Review vouchers and approval for marketing authorization; timing and consequences of an NDA submission to FDA; FDA’s acceptance for filings; the timeline of related discussions with the FDA; the initial potential PDUFA timing; the potential commercial opportunity; the ability of dordaviprone to attain significant market acceptance among disease experts, patient advocates and their patients; and the expected impact of dordaviprone on patients. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are: risks related to the ability to obtain and maintain accelerated approval, Priority Review, rare pediatric disease Priority Review vouchers, and approval for marketing authorization; uncertainty on the response of regulators to including additional supportive data to be submitted in the NDA filing, including RANO 2.0 assessments, and uncertainty with respect to the initial potential PDUFA timing; risks related to the timing, completion and outcome of the Phase 3 ACTION study of dordaviprone; risks associated with market acceptance; risks associated with repeating positive results obtained in prior preclinical or clinical studies in future studies; risks related to the clinical development of our clinical candidates; and additional risks set forth in the Company’s filings with the Securities and Exchange Commission. These forward-looking statements represent the Company's judgment as of the date of this release. The Company disclaims, however, any intent or obligation to update these forward-looking statements. CONTACT: Will O’ConnorStern Investor Relations212-362-1200 Dana DavisSteelwire Public Relationsdana@steelwire.co