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Press Release: Seelos Therapeutics Completes Acquisition of Trehalose from Bioblast Pharma

Press Release: Seelos Therapeutics Completes Acquisition of Trehalose from Bioblast Pharma

新聞稿:塞洛斯治療完成收購 Bioblast 製藥的海藻糖
道琼斯 ·  2019/02/19 21:00

Press Release: Seelos Therapeutics Completes Acquisition of Trehalose from Bioblast Pharma

新聞稿:Seelos Treateutics完成從Bioblast Pharma收購海藻糖

Seelos Therapeutics Completes Acquisition of Trehalose from Bioblast Pharma

Seelos治療公司完成對Bioblast Pharma公司海藻糖的收購

Diversifies Seelos' pipeline in rare diseases

Seelos在罕見疾病方面的流水線多樣化

Collaborates with Team Sanfilippo Foundation in development of Trehalose in Sanfilippo syndrome

與Sanfilippo基金會團隊合作開發治療Sanfilippo綜合徵的海藻糖

NEW YORK, Feb. 19, 2019 (GLOBE NEWSWIRE) -- Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company, announced today a definitive agreement with Bioblast Pharma Ltd., (Nasdaq: ORPN), whereby Seelos acquired all development and commercial rights to Bioblast's proprietary trehalose 90 mg/mL IV (Trehalose) solution as well as the existing inventory of the drug which should be sufficient to fulfill its current research needs. Trehalose, which is currently an investigational molecule, has been studied in prior phase 2 clinical studies in over 70 patients with two rare diseases and demonstrated a favorable safety profile and promising efficacy signal.

紐約,2019年2月19日(環球網)--臨牀階段生物製藥公司Seelos Treateutics,Inc.(納斯達克股票代碼:SEEL)今天宣佈與Bioblast製藥有限公司(納斯達克:ORPN)達成一項最終協議,根據該協議,Seelos將收購Bioblast的專有海藻糖90毫克/毫升IV(海藻糖)溶液以及該藥物的現有庫存的所有開發權和商業權,該藥物應足以滿足其目前的研究需求。海藻糖目前是一種研究分子,已在先前的第二階段臨牀研究中對兩種罕見疾病的70多名患者進行了研究,並顯示出良好的安全性和良好的療效信號。

Under the terms of the acquisition, Seelos assumed a collaborative agreement with Team Sanfilippo Foundation (TSF), a nonprofit medical research foundation founded by parents of children with Sanfilippo syndrome. TSF, upon approval by the FDA, plans to begin an open label, Phase 2(b) clinical trial in up to 20 patients with Sanfilippo syndrome and Seelos will provide the clinical supply of Trehalose. The terms of the agreement entitle Seelos access to all clinical data from this trial. Based on the pre-clinical and in-vitro studies, there is a sound scientific rationale for developing Trehalose for the treatment of Sanfilippo syndrome.

根據收購條款,Seelos承擔了與Team Sanfilippo Foundation(TSF)的合作協議,Team Sanfilippo Foundation是一個非營利性醫學研究基金會,由患有Sanfilippo綜合徵的兒童的父母創立。在FDA批准後,TSF計劃在多達20名Sanfilippo綜合徵患者中開始開放標籤的2(B)期臨牀試驗,Seelos將提供海藻糖的臨牀供應。根據協議條款,Seelos有權訪問該試驗的所有臨牀數據。根據臨牀前和體外研究,開發海藻糖治療Sanfilippo綜合徵有充分的科學依據。

"The acquisition of Trehalose, along with our collaboration with TSF, are each important to understand in terms of their significance," said Raj Mehra, PhD, Chairman, Founder, and Chief Executive Officer, Seelos Therapeutics. "Taken together, these are validating achievements for Seelos' collaborative approach and speaks to the clinical and moral imperative to address the needs of patients with Sanfilippo syndrome, the need to rapidly advance novel therapeutics like Trehalose, and the need to continue to evaluate Trehalose in a range of such rare and devastating diseases including continuing the prior work in oculopharyngeal muscular dystrophy and spinocerebellar ataxia type 3."

Seelos治療公司董事長、創始人兼首席執行官Raj Mehra博士説:“收購海藻糖,以及我們與TSF的合作,都是瞭解它們的重要性的重要方面。綜上所述,這些都是Seelos合作方法的驗證成就,説明瞭滿足Sanfilippo綜合徵患者需求的臨牀和道德必要性,迅速推進海藻糖等新療法的需要,以及繼續評估海藻糖在一系列此類罕見和破壞性疾病中的作用,包括繼續先前在眼嚥肌營養不良和脊髓小腦性共濟失調3型方面的工作。“

"Our hope through this study is to evaluate the safety and efficacy of Trehalose in all forms of Sanfilippo, which is a devastating terminal disease that took the lives of 16 children last year," said Kathleen Buckley, President of the New York-based TSF. "Our team has tirelessly worked to raise proceeds to fund this study. If successful, we will work with the regulatory agencies in the US and EU to get this drug approved and into the hands of our families."

“我們希望通過這項研究來評估海藻糖對所有形式的桑菲裏波的安全性和有效性,這是一種毀滅性的絕症,去年奪走了16名兒童的生命,”紐約三菱婦科基金會的總裁凱瑟琳·巴克利説。我們的團隊不知疲倦地為這項研究籌集資金。如果成功,我們將與美國和歐盟的監管機構合作,使這種藥物獲得批准,並送到我們的家人手中。

Under the terms of the agreement, Seelos made an upfront payment to Bioblast and will make certain additional milestone payments upon the achievement of clinical and regulatory milestones, plus a royalty upon clinical success, and approval for commercialization. Full terms of this agreement are disclosed in the Current Report on Form 8-K, filed by Seelos with the Securities and Exchange Commission today.

根據協議條款,Seelos向Bioblast支付了一筆預付款,並將在實現臨牀和監管里程碑時支付某些額外的里程碑付款,以及在臨牀成功時支付特許權使用費,並批准商業化。這項協議的全部條款在Seelos今天提交給證券交易委員會的8-K表格的當前報告中披露。

About Trehalose

關於海藻糖

Trehalose is a protein stabilizer that also activates autophagy and crosses the blood-brain-barrier. Trehalose is a low molecular weight disaccharide (.342 kDa) that protects against pathological processes in cells. It has been shown to penetrate muscle and cross the blood brain barrier. In animal models of several diseases associated with abnormal cellular-protein aggregation, it has been shown to reduce pathological aggregation of misfolded proteins as well as to activate autophagy pathways through the activation of Transcription Factor EB ("TFEB"), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.

海藻糖是一種蛋白質穩定劑,也可以激活自噬並跨越血腦屏障。海藻糖是一種低分子二糖(0.342 kDa),可防止細胞內的病理過程。它已被證明可以穿透肌肉並穿過血腦屏障。在與細胞-蛋白質異常聚集相關的幾種疾病的動物模型中,它被證明可以減少錯誤摺疊的蛋白質的病理性聚集,並通過激活轉錄因子EB(TFEB)激活自噬途徑,轉錄因子EB是溶酶體和自噬基因表達的關鍵因子。TFEB的激活是一種新興的治療靶點,用於治療許多儲存物質病理性堆積的疾病。

Trehalose 90 mg/mL IV solution has demonstrated promising clinical potential in prior phase 2 clinical development for oculopharyngeal muscular dystrophy (OPMD) and spinocerebellar ataxia type 3 (SCA3, also called Machado Joseph disease), with encouraging safety and efficacy results thus far. These pathological proteins aggregate within cells, eventually leading to cell death. Prior preclinical studies indicate that this platform has the potential to prevent mutant protein aggregation in other devastating PolyA/PolyQ diseases.

90 mg/mL海藻糖靜脈溶液在治療眼嚥肌營養不良(OPMD)和脊髓小腦性共濟失調3型(SCA3,也稱為馬查多·約瑟夫病)的早期2期臨牀開發中顯示出良好的臨牀潛力,迄今取得了令人鼓舞的安全性和有效性結果。這些病理性蛋白質聚集在細胞內,最終導致細胞死亡。先前的臨牀前研究表明,該平臺有可能防止其他破壞性Polya/PolyQ疾病的突變蛋白聚集。

Two U.S. patents for parental administration of trehalose exist for patients with OPMD and SCA3; both are expected to expire in 2033. In addition, Orphan Drug Designation for OPMD and SCA3 has been secured in the U.S. and in the EU.

OPMD和SCA3患者目前有兩項父母給藥海藻糖的美國專利;這兩項專利預計都將於2033年到期。此外,OPMD和SCA3的孤兒藥物指定已在美國和歐盟獲得。

About Seelos Therapeutics, Inc.

Seelos治療公司簡介

Seelos Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development and advancement of novel therapeutics to address unmet medical needs for the benefit of patients with central nervous system (CNS) disorders and in rare diseases. The Company's portfolio includes several late-stage clinical assets targeting psychiatric and movement disorders, including orphan diseases. Seelos is based in New York, New York. Seelos' common stock is traded on The Nasdaq Capital Market under the symbol "SEEL". For more information, please visit our website: www.seelostherapeutics.com, the content of which is not incorporated herein by reference.

Seelos Treateutics,Inc.是一家臨牀階段的生物製藥公司,專注於新療法的開發和進步,以滿足中樞神經系統(CNS)障礙患者和罕見疾病患者的未滿足醫療需求。該公司的投資組合包括幾項針對精神障礙和運動障礙的晚期臨牀資產,包括孤兒疾病。Seelos的總部設在紐約,紐約州。Seelos的普通股在納斯達克資本市場上交易,代碼為“SEEL”。欲瞭解更多信息,請訪問我們的網站:www.seelosTreateutics.com,其內容未通過引用併入本文。

About Bioblast Pharma Ltd.

關於Bioblast Pharma Ltd.

Bioblast Pharma Ltd. (Nasdaq: ORPN) is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. For more information, please visit: www.bioblastpharma.com, the content of which is not incorporated herein by reference.

生物製藥有限公司(納斯達克代碼:ORPN)是一家臨牀階段的生物技術公司,致力於為罕見和超罕見遺傳病的患者開發具有臨牀意義的療法。欲瞭解更多信息,請訪問:www.bioblastpharma.com,其內容未通過引用併入本文。

About Team Sanfilippo Foundation

關於Sanfilippo團隊基金會

Team Sanfilippo Foundation (TSF) is a nonprofit medical research foundation founded in 2008 by parents of children with Sanfilippo Syndrome. TSF's mission is to fund potential therapies that can be in clinical trials in the near future. Team Sanfilippo is dedicated to providing assistance to families to gain access to clinical trials, treatments and compassionate use. Team Sanfilippo remains dedicated to getting children of all ages access to clinical trials and treatments and assistance to families enrolled in clinical trials. For more information, please visit teamsanfilippo.org.

Sanfilippo團隊基金會(TSF)是一個非營利性醫學研究基金會,由患有Sanfilippo綜合徵的兒童的父母於2008年創立。TSF的使命是為可能在不久的將來進入臨牀試驗的潛在療法提供資金。Sanfilippo團隊致力於為家庭提供幫助,使他們能夠獲得臨牀試驗、治療和同情使用。Sanfilippo團隊仍然致力於讓所有年齡段的兒童獲得臨牀試驗和治療,並向登記參加臨牀試驗的家庭提供援助。欲瞭解更多信息,請訪問Teamsanfilippo.org。

Forward-looking Statements

前瞻性陳述

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. For example, we are using forward-looking statements when we discuss Seelos' future operations and its ability to successfully initiate and complete clinical trials and achieve regulatory milestones and related timing; the nature, strategy and focus of Seelos' business; the development and commercial potential and potential benefits of any of Seelos' product candidates including Trehalose; and that Seelos' product candidates have the potential to address critical unmet needs of patients with serious diseases and conditions. Seelos may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on Seelos' current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Seelos could differ materially from those described in or implied by the statements in this press release, including: the uncertainties associated with the clinical development and regulatory approval of Seelos' product candidates, including potential delays in the commencement, enrollment and completion of clinical trials; the potential that earlier clinical trials and studies of Seelos' product candidates may not be predictive of future results; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the those risks discussed in Seelos' filings with the Securities and Exchange Commission. Except as otherwise required by law, Seelos disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

本新聞稿包含符合1995年“私人證券訴訟改革法”和其他聯邦證券法中“安全港”條款的前瞻性陳述。例如,當我們討論Seelos的未來運營及其成功啟動和完成臨牀試驗並實現監管里程碑及相關時機的能力;Seelos業務的性質、戰略和重點;Seelos的任何候選產品(包括海藻糖)的開發和商業潛力及潛在好處;以及Seelos的候選產品具有滿足嚴重疾病和病症患者未滿足的關鍵需求的潛力時,我們使用的是前瞻性聲明。Seelos可能無法實際實現前瞻性陳述中披露的計劃、實現意圖或達到預期或預測,您不應過度依賴這些前瞻性陳述。由於此類陳述涉及未來事件,並基於Seelos公司當前的預期,它們會受到各種風險和不確定性的影響,而Seelos的實際結果、表現或成就可能與本新聞稿中描述或暗示的情況大不相同,其中包括:與Seelos候選產品的臨牀開發和監管批准相關的不確定性,包括臨牀試驗的開始、登記和完成可能出現延遲;Seelos候選產品的早期臨牀試驗和研究可能無法預測未來結果;以及需要額外資本繼續推進這些候選產品。, 這可能不是以優惠的條件提供的,或者根本沒有。上述對可能導致實際事件與預期不同的重要因素的審查不應被解釋為詳盡無遺,應與本文中和其他地方包括的陳述一起閲讀,包括Seelos提交給美國證券交易委員會的文件中討論的那些風險。除非法律另有要求,否則Seelos沒有任何更新或修改任何前瞻性陳述的意圖或義務,這些前瞻性陳述僅説明截至本新聞稿發佈之日的情況,無論是由於新信息、未來事件或情況或其他原因。

Contact information:

聯繫方式:

Anthony Marciano

安東尼·馬西亞諾

Head of Corporate Communications

企業公關主管

Seelos Therapeutics, Inc.

Seelos治療公司

anthony.marciano@seelostx.com

郵箱:anthony.marciano@seelostx.com

www.seelostherapeutics.com

Www.seelostherapeutics.com

(END) Dow Jones Newswires

(完)道瓊斯通訊社

February 19, 2019 08:00 ET (13:00 GMT)

2019年2月19日東部時間08:00(格林尼治標準時間13:00)

譯文內容由第三人軟體翻譯。


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