智擎- Nanobiotix 新藥NBTXR3 phase I trial !!!!

智擎法夥伴NBTXR3一期臨床初步安全性獲確認 精實新聞 2012-11-14 09:50:31 記者 蕭燕翔 報導 智擎(4162)宣布，法國授權合作夥伴Nanobiotix S.A的癌症放射治療NBTXR3(PEP503)，第一期臨床試驗初步安全性獲確認，預計明年第一季底完成一期臨床，目標2015年上半年完成二期臨床試驗後申請上市，未來將不排除隨時在台灣及亞太地區以醫材方式，申請進行臨床試驗。市場看好，該技術有助於補全放射治療的不足，商機可期。智擎是在今年8月初與法國Nanobiotix S.A.公司簽署合作協議，雙方同意由智擎支付100萬美元簽約金，取得該技術在台灣、中國大陸、日本、韓國、印度、澳洲及其他亞太地區國家的開發與商品化獨家授權，未來至成功上市及銷售後，將總計支付5,600萬美元的階段性授權金(milestone payment)，並依照其在授權區域內的淨銷售額分級，支付Nanobiotix最高2位數百分比的權利金(royalties)。 而智擎最新宣布，法國夥伴第一期人體臨床試驗於軟組織肉瘤(Soft Tissue Sarcoma)的第一群組6位患者治療安全性(safety)數據，獲獨立監測委員會（Independent Data Monitoring Committee，IDMC）確認，並同意根據其臨床試驗計劃書(protocol)內容繼續招收第二群組新病人。智擎表示，考量爭取上市的最快時機，該技術在歐盟是以醫材的方式，申請進入人體臨床試驗，而第一期最重要是確認安全性，根據法國夥伴規劃，第一期臨床總計將收案約20組病患，並分三組進行，目前第一組病患安全性數據已獲確認，第二、三組也將陸續跟進，預計明年第一季底完成第一期人體臨床試驗，2015年上半年完成二期臨床後申請上市。 智擎也指出，除了歐洲地區外，也隨時不排除比照歐盟方式，以醫材申請台灣及亞太地區的臨床試驗。業界評估，該技術主要是補足現行放射治療的不足，可擔任類似現有放射治療先驅標靶的角色，提高放射治療對腫瘤細胞的針對性，降低對周圍健康組織的傷害，以目前全球五成病人接受放射治療的比例估算，商機可期。 法人也預期，隨雙方合作一期臨床明年第一季完成，智擎將依照協議支付一部份的階段里程碑金，惟只要該醫材順利上市，有機會複製PEP02外的第二個成功案例，成為智擎新的金雞母。

IDMC reviews data from Nanobiotix's NBTXR3 Phase I study on advanced soft tissue sarcoma Published on November 13, 2012 at 11:59 PM · Nanobiotix (Euronext: NANO / ISIN: FR0011341205), announced today that the Independent Data Monitoring Committee (IDMC) has reviewed the data of the first 6 patients treated in the phase I Study with the lead product NBTXR3 for the treatment of advanced Soft Tissue Sarcoma of the extremity. Based on the review of the safety data, the IDMC has unanimously recommended continuing the enrollment in the study according to the protocol. This trial is realized in the Institut Gustave Roussy, France."This milestone is crucial for NANOBIOTIX" says Laurent Levy, CEO and co-founder of Nanobiotix. "It is in accordance with our expectations concerning our NanoXray products' risk profile. After our successful IPO, we are happy to deliver this first milestone to all our investors and subscribers who have had confidence in the company. We intend to continue to deliver with our breakthrough technology to bring those products to patients rapidly and efficiently. This initial step is "derisking" our approach and gives us confidence to expend the clinical development to different indications."The IDMC consists of 3 independent members and has the responsibility of ensuring the safety of all patients enrolled in the study, the quality of the data collected and the continued scientific validity of the study design. The IDMC has reviewed the data of the first group of patients, focusing on patient's safety and tumor surgery feasibility.

The clinical trial evaluates the following endpoints:

Primary endpoints Feasibility of the injection of NBTXR3 and its activation by radiotherapy in small and big tumors

Safety profile of NBTXR3 and its activation by radiotherapy

Secondary endpoints

Tumor response to NBTXR3 activated by radiotherapy, in terms of cancer cell killing

Tumor downsizing and carcinologic surgery feasibility with NBTXR3 activated by radiotherapy (medical imaging)

Kinetic profile of NBTXR3 in the body

In this first group of patients, all the primary endpoints were reached, NBTXR3 demonstrated good safety and surgery was feasible with limb preservation in all patients.The IDMC interim safety analysis found no evidence of local or general toxicity and accordingly the study is continuing. Based on their analysis, the committee has recommended starting the second group of patients without protocol amendments.