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催化剂制药宣布Firdapse治疗MuSK抗体阳性重症肌无力的利好数据(英文)

2017-03-16 00:36:34 来源:
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Catalyst intends to proceed to U.S. multi-center pivotal trial



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Expects additional data to be presented at upcoming medical congresses in 2017


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CORAL GABLES, Fla., March  15, 2017  (GLOBE NEWSWIRE) --  Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare neuromuscular and neurological diseases, today announced positive top-line results from the investigator-sponsored trial evaluating Firdapse (Amifampridine Phosphate) as a treatment for myasthenia gravis patients with anti-MuSK antibodies (MuSK-MG). MuSK-MG, is an ultra-rare sub-population of myasthenia Gravis (MG) patients which is a debilitating neuromuscular disease, and there are currently no FDA approved therapies for this specific form of MG.  Both of the co-primary efficacy endpoints of change from baseline (CFB) in total Quantitative Myasthenia Gravis (QMG) score (p=0.0003) and CFB in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score (p=0.0006) were statistically and clinically significant in this seven patient trial.  Several secondary efficacy measures also achieved statistical significance.  Amifampridine phosphate was well tolerated in this population of patients.

  The study was conducted by a team of researchers led by Renato Mantegazza, MD, Director, Department of Neuroimmunology and Neuromuscular Diseases, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy, a major referral center for MuSK-MG patients.  The study was designed as a randomized (1:1), double-blind, placebo-controlled, crossover, outpatient study to evaluate the safety, tolerability and potential efficacy of amifampridine phosphate in patients diagnosed with MuSK-MG.  Catalyst provided funding, study drug, and placebo for this trial.

  Dr. Mantegazza, the principal investigator of this trial, stated, “Our prospective study evaluating amifampridine phosphate for the symptomatic relief of antibody positive MuSK-MG was statistically significant in demonstrating that it can be an important treatment option.  Not only are the results statistically significant, but more importantly, there was a large clinical benefit to the patients.  Current treatments for MuSK-MG patients are often inadequate and these patients often face a lifetime of severe complications, including difficulty walking, talking, swallowing, and breathing normally, and in some cases their disease may be life-threatening and require hospitalization and intensive care.  Amifampridine phosphate may offer us an effective treatment option.  I look forward to the day when I can use this drug in routine clinical practice of treating MuSK-MG patients.”

  Dr. Silvia Bonanno, one of the investigators from the Istituto Neurologico Carlo Besta, is planning to present these results at the 13th International Conference on Myasthenia Gravis and Related Disorders in May, 2017 in New York City, provided her abstract is accepted as a Hot Topic Short Talk.  This conference is organized by the Myasthenia Gravis Foundation of America and the New York Academy of Sciences.

  “These data announced today should allow us to accelerate our MuSK-MG program over the coming months, as we expect to consult with our external experts and regulatory agencies on a pivotal clinical development plan,” said Patrick J. McEnany, Catalyst’s Chief Executive Officer. “I would like to thank Dr. Mantegazza, his associates at Carlo Besta Neurological Institute, and the patients that participated in this important clinical trial.”

  "While several effective treatment options exist for the anti-acetylcholine receptor form of myasthenia gravis (AcHR-MG), MuSK-MG has been particularly refractory to current MG treatment options and represents an unmet medical need in the MG community of patients," stated Gary Ingenito MD, Ph.D., Catalyst's Chief Medical Officer.  Dr. Ingenito continued: "If the significant clinical effect observed in this trial is reproduced in a multicenter trial, amifampridine phosphate would, upon approval, likely become the first line standard of care for MuSK-MG.  Based on these results we intend to discuss with FDA conducting a registration trial in the United States evaluating amifampridine phosphate for the symptomatic treatment of patients with MuSK-MG."

  About the Clinical Trial

  The MuSK-MG “proof-of-concept” trial was a randomized, double blind, placebo-controlled, single site, outpatient, investigator-sponsored, clinical trial to evaluate the safety and efficacy of amifampridine phosphate in myasthenia gravis patients with a positive serological test for the anti-MuSK antibodies (MuSK-MG).  Catalyst provided the investigational drug, placebo, and funding for this clinical trial.

  Patients were enrolled into the trial and were titrated to an effective dose of amifampridine phosphate for a period of at least 4 weeks in a "run-in" phase of the trial.  Following achievement of a dosage that effectively managed the patient's symptoms, the patients were randomized 1:1 into one of two crossover treatment groups.  There were three treatment periods in this crossover design, referred to as a "switch-back" crossover design, that enables both the determination of the effect of the treatment as well as the correction of any "subjective carryover" from earlier treatment periods into later treatment periods.  Carryover in crossover designs is a common concern of regulatory agencies, and this design effectively corrects for and eliminates the effects of carryover from the efficacy assessments.  Each treatment period lasted 1 week for a total of treatment duration of 3 weeks alternating with either amifampridine phosphate or placebo.  The co-primary efficacy endpoints of change from base (CFB) in total Quantitative Myasthenia Gravis (QMG) score and CFB in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score at the 7th day of each period were assessed using a mixed carryover effects statistical model of Kunert and Stufken.  Secondary endpoints consisted of, in order, CFB in Myasthenia Gravis Composite (MGC) Score, CFB in Neurological Institute Carlo Besta-Myasthenia Gravis (NICB-MG) score, proportion of patients with a 2, or larger, point reduction in MG-ADL, proportion of patients with a 3, or larger, reduction in MGC, CFB in Myasthenia Gravis Quality of Life, 15 domain, (MG-QoL 15) score, and CFB in Fatigue Severity scale (FSS).  Catalyst's funding of this study included the use of a CRO, and this study was run as a well-controlled trial suitable for regulatory submission.

  About MuSK Myasthenia Gravis

  About 15% of MG patients test negative for the acetylcholine receptor antibody. These patients have seronegative (SN) MG. Approximately 40-50% of these patients with SNMG (equating to an estimate of approximately 4,500 patients in the United States) test positive for the anti-MuSK antibody. MuSK is a protein that is required for the maintenance of the neuromuscular junction and patients with the anti-MuSK antibody are identified as having MuSK-MG. MuSK-MG is a clinically distinguishable, more severe form of MG. The disease is characterized by a predominance in females, a prevalent involvement of cranial and bulbar muscles, high incidence of respiratory crises and a resistance to treatment. Although many patients with MuSK MG are presently treated with anticholinesterase inhibitors or immunosuppressants, such patients do not generally respond adequately to these treatments.

  About Catalyst Pharmaceuticals

  Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, including Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic syndromes (CMS), MuSK myasthenia gravis and infantile spasms. Firdapse has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of LEMS and Orphan Drug Designation for LEMS, CMS and myasthenia gravis. Firdapse is the first and only approved drug in Europe for symptomatic treatment in adults with LEMS.

  Catalyst is also developing CPP-115 to treat refractory infantile spasms and possibly refractory Tourette's Disorder. CPP-115 has been granted U.S. Orphan Drug Designation for the treatment of infantile spasms by the FDA and has been granted E.U. Orphan Medicinal Product Designation for the treatment of West syndrome by the European Commission.  In addition, Catalyst is developing a generic version of Sabril (vigabatrin).

  Forward-Looking Statements

  This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including whether the receipt of breakthrough therapy designation for Firdapse will expedite the development and review of Firdapse by the FDA or the likelihood that the product will be found to be safe and effective, the timing of Catalyst's second trial evaluating Firdapse for the treatment of LEMS and whether the trial will be successful, whether Catalyst's assumptions in its updated business plan will be accurate and the impact of unanticipated events or delays in projected activities on Catalyst's cash requirements and on Catalyst's ability to get to an accepted NDA submission for Firdapse without the need for additional funding, what clinical trials and studies will be required before Catalyst can resubmit an NDA for Firdapse for the treatment of CMS and whether any such required clinical trials and studies will be successful, whether any NDA for Firdapse resubmitted to the FDA will ever be accepted for filing,  the timing of any such NDA filing or acceptance, whether, if an NDA for Firdapse is accepted for filing, such NDA will be given a priority review by the FDA, whether any future trial evaluating Firdapse for the treatment of MuSK-MG will be successful and whether Catalyst can obtain the funding required to conduct such trial, whether Firdapse will ever be approved for commercialization, whether Catalyst will be the first company to receive approval for amifampridine (3,4-DAP), giving it 5-year marketing exclusivity for its product, whether CPP-115 will be determined to be safe for humans, what additional testing will be required before CPP-115 is "Phase 2 ready", whether CPP-115 will be determined to be effective for the treatment of refractory infantile spasms or possibly Tourette's Disorder, or for any other indications, whether Catalyst can successfully design and complete a bioequivalence study of its version of vigabatrin compared to Sabril that is acceptable to the FDA, whether any such bioequivalence study the design of which is acceptable to the FDA will be successful, whether any ANDA that Catalyst submits for a generic version of Sabril will be accepted for filing, whether any ANDA for Sabril accepted for filing by the FDA will be approved (and the timing of any such approval), whether any of Catalyst's product candidates will ever be approved for commercialization or successfully commercialized, and those other factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2015 and its other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.

  CONTACT: Investor Contact

  rian Korb

  he Trout Group LLC

  646) 378-2923

  korb@troutgroup.com

  Media Contacts

  avid Schull

  usso Partners

  212) 845-4271

  avid.schull@russopartnersllc.com

  Company Contact

  atrick J. McEnany

  atalyst Pharmaceuticals

  hief Executive Officer

  305) 529-2522

  mcenany@catalystpharma.com


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转播到腾讯微博 发表时间:2017-03-17 15:16:16  IP:已记录
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好消息!催化剂制药罕见重症肌无力试验性药物Firdapse临床试验取得成功!

来源:生物谷 2017-03-21 15:27“试”点好的,给实验一个负责的态度!

大国崛起!“智”造中国:“智”造中国:医药工业4.0与绿色制药高峰论坛


2017年3月21日讯 /生物谷BIOON /——催化剂制药(Catalyst Pharma)近日宣布其治疗一种严重重症肌无力(MG,一种使人衰弱的神经肌肉疾病)的实验性药物达到试验主要目标。

这个药物叫做Firdapse,该公司在该试验中使用该药治疗7名患有Musk阳性的MG(Musk-MG)患者,这类患者属于比较罕见的MG病人。

这个包含7个病人的临床试验评估了Firdapse(二胺吡啶磷酸盐)的安全性、病人对该药的耐受性及有效性,结果发现该药物满足了两个主要疗效终点。

根据总的定量重症肌无力得分及重症肌无力的日常生活得分,各参数与基线的差值在统计学和临床学上都具有显著性。

该公司声称各种次要疗效检测也具有统计学上的显著性,这群病人对二胺吡啶磷酸盐具有较好的耐受性。

催化剂制药首席医疗官Gary Ingenito说:“尽管抗乙酰胆碱受体的重症肌无力患者(AcHR-MG)有几种有效的治疗备选方法,但是MuSK-MG对目前的MG疗法具有很强的耐受性,因此是MG病人未得到满足的一个医学需求。”

“如果这项临床试验观察到的显著性临床效应能够在多中心试验中得到重复,那么一旦二胺吡啶磷酸盐获得批准,它将可能成为MuSK-MG病人的一线标准疗法。”

该公司为这项MuSK-MGA概念验证性试验提供了资金、研究药物及安慰剂。

基于这些结果,该公司计划与FDA讨论在美国进行注册试验,评估该药物对症治疗MuSK-MG病人的疗效。

催化剂制药注意到尽管许多MuSK-MG病人目前采用抗胆碱酯酶抑制剂或者免疫抑制剂进行治疗,但是这些病人不会对治疗产生足够强的反应。(生物谷Bioon.com)


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转播到腾讯微博 发表时间:2017-04-29 21:44:27  IP:已记录
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