FIREFISH is a two-part seamless, open-label, multi-center study to investigate the safety and efficacy of RG7916 in infants and babies with Type 1 SMA.
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[27-January-2018] |
SOUTH PLAINFIELD, N.J., Jan. 27, 2018 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the presentation of early interim data from Part 1, the dose-finding portion of the FIREFISH study. FIREFISH is a two-part seamless, open-label, multi-center study to investigate the safety and efficacy of RG7916 in infants and babies with Type 1 SMA. RG7916 has been safe and well tolerated at all doses and there have been no drug-related safety findings leading to withdrawal. In addition, data on the ability to swallow and requirements for tracheostomy or permanent ventilation, together with overall survival were also presented. Previously published natural history data indicate that in a comparable historic cohort the median age of event-free survival for SMA Type 1 infants to be between 8 and 10.5 months1,2. The presentation was given by Dr. Giovanni Baranello, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy, at the International Scientific Congress on spinal muscular atrophy in Kraków, Poland, and will be made available via a link under the investor relations section of the PTC website. (www.ptcbio.com/smaeurope) “It is exciting to show for the first time that an oral small molecule demonstrates early signs of clinical benefit,” stated Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. “We believe a drug that distributes to both the CNS and peripheral tissues can provide an important benefit to children suffering from this devastating, fatal disease. We anticipate that the trial will transition to the pivotal portion in the coming months.” FIREFISH (NCT02913482) is a multi-center, open-label, seamless pivotal study evaluating the safety and efficacy of RG7916 in babies aged 1-7 months at enrollment with Type 1 SMA and two SMN2 gene copies. The exploratory Part 1 (n=8-24) is assessing the safety, tolerability, pharmacokinetics and pharmacodynamics of RG7916 at different dose levels. In Part 1, patients receive RG7916 for at least 4 weeks (or 2 weeks after steady-state is achieved) of daily administration; patients then enter an extension phase with RG7916. The confirmatory Part 2 (n=40) will assess the safety and efficacy of RG7916 at the dose level selected from Part 1 over 24 months. The primary endpoint for Part 2 is the proportion of infants sitting without support for 5 seconds, assessed by the Gross Motor Scale of the BSID-III, after 12 months of treatment. “This early interim analysis of survival and the delay to milestone events that are hallmarks of the progression of SMA in infants are promising,” stated Dr. Giovanni Baranello, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy. “These data, in conjunction with earlier poster presentations at the Congress from the trials in patients with type 2/3 SMA, are supportive of safety and sustained increase in SMN protein levels. Clinical trials in SMA patients require a globalization of clinical research; particularly, it is essential for a coordinated multidisciplinary team to support the babies and their families and to ensure the best standard of care to promote the higher benefit to affected children from potential new treatments.” In addition to the oral presentation, three posters were on display during the Congress: updated pharmacodynamic and safety data from SUNFISH Part 1, preliminary evidence for pharmacodynamic effects of RG7916 in JEWELFISH, and preclinical data demonstrating the relationship between central and peripheral SMN protein increase upon treatment with RG7916. The data presented demonstrate systemic and dose-dependent increase of SMN protein levels. The data from mice and other species suggest that SMN protein level increases seen in the blood of patients following RG7916 treatment reflect SMN protein level increases in the CNS, muscle and other key issues affected in SMA. In addition, RG7916 has been safe and well tolerated at all doses and there have been no drug-related safety findings leading to withdrawal. The U.S. Food and Drug Administration granted orphan drug designation to RG7916 for the treatment of patients with SMA. RG7916 directly targets the underlying molecular deficiency of SMA by modulating SMN2 splicing to increase expression of full-length SMN2 mRNA from the SMN2 gene. An interim analysis from the first part of SUNFISH of the five cohorts treated with RG7916 for 28 days or longer demonstrated an exposure-dependent increase in SMN protein. SMA is characterized by reduced levels of SMN protein, motor neuron loss, and muscle atrophy. To date, RG7916 remains well-tolerated in patients at all doses and there have been no drug-related safety findings leading to withdrawal. The SMA program was initially developed by PTC Therapeutics in partnership with the SMA Foundation in 2006 to accelerate the development of a treatment for SMA. In November 2011, Roche gained an exclusive worldwide license to the PTC/SMA Foundation SMN2 alternative splicing program. The development of these compounds is being executed by Roche and overseen by a joint steering committee with members from PTC, Roche, and the SMA Foundation. 1 Kolb S, et al. Amer Neuro Assoc 2017; 883-891 About Spinal Muscular Atrophy (SMA) About the SMA Clinical Trials SUNFISH: A double?blind, two?part, placebo?controlled trial. Part 1 enrolled patients with Type 2 or 3 SMA to evaluate safety, tolerability, and PK/PD of several RG7916 dose levels. The pivotal SUNFISH Part 2, in non?ambulant patients with Type 2 or 3 SMA, will evaluate safety and efficacy of the RG7916 dose level selected from Part 1. JEWELFISH: An exploratory, open-label study to establish the safety and tolerability of RG7916 in people who have previously participated in a study with another therapy targeting SMN2 splicing. About PTC Therapeutics For More Information: Investors: Media: Forward Looking Statements: PTC’s actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the initiation, enrollment, conduct and availability of data from either the SUNFISH or FIREFISH studies and the outcome of such studies; events during, or as a result of, these studies that could delay or prevent further development of RG7916, including future actions or activities under the SMA joint development program; our expectations for regulatory approvals; PTC’s scientific approach and general development progress; and the factors discussed in the “Risk Factors” section of PTC’s most recent Quarterly Report on Form 10-Q as well as any updates to these risk factors filed from time to time in PTC’s other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products, including with respect to PTC’s joint development program in SMA with Roche and the SMAF. There are no guarantees that any product candidate under the joint development program will receive regulatory approval in any territory or prove to be commercially successful. The forward-looking statements contained herein represent PTC’s views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. View original content:http://www.prnewswire.com/news-releases/preliminary-data-from-firefish-trial-in-type-1-sma-infants-presented-at-the-international-scientific-congress-on-spinal-muscular-atrophy-300589231.html SOURCE PTC Therapeutics, Inc. | ||
Company Codes: NASDAQ-NMS:PTCT |