PTC Therapeutics Announces Positive Preliminary Clinical Data from the FIREFISH Study in Type 1 SMA Patients

The clinical data were presented at the annual meeting of the American Academy of Neurology.

The presentation from Part 1, the Dose Finding section, of the ongoing FIREFISH study. The open-label study investigated RG7916, an oral survival motoneuron 2 (SMN2) splice modifier, in type 1 SMA patients.

The data from Part 1 were presented at the American Academy of Neurology 2018 Annual Meeting and showed that RG7916 was well tolerated at all doses and to date has no drug-related safety findings leading to withdrawal

In addition, no babies have required a tracheostomy or permanent ventilation since study initiation and no baby has lost the ability to swallow. The median age of first dose was 6.7 months and babies have received RG7916 for a duration of up to 14.8 months. Recruitment is ongoing globally for the pivotal second part of the FIREFISH study.

”RG7916 continues to demonstrate compelling results in SMA patients and we look forward to making further gains to combat this devastating, fatal disease,” said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. ”We are encouraged by this promising data as we continue our ongoing global recruitment efforts for the SMA programs.”

Roche släpper stor uppdatering på de två molekylerna RG7916 och OLESOXIME – pågående studier

FIREFISH is an ongoing, global multi-center, open-label, seamless Phase 2 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. Data from 21 patients from the completed Part 1 portion of this study suggested that RG7916 produced a dose-dependent increase in SMN protein levels in babies with Type 1 SMA.

SMA Type 1 is a devastating disease in which babies rarely live past the second year of life without permanent nutritional and/or ventilatory support,” stated Dr. Giovanni Baranello, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy. ”This early data on the infants’ ability to swallow and lack of need of permanent ventilatory support is very encouraging.”

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.

FIREFISH: An open-label, two-part clinical trial. Part 1 was a dose escalation study in 21 infants for a minimum of 4 weeks. The primary objective of Part 1 was to assess the safety profile of RG7916 in infants and determine the dose for Part 2. Part 2 is a single-arm study with the dose selected in Part 1 in approximately 40 infants with Type 1 SMA for 24 months, followed by an open-label extension. This study is recruiting globally.

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, is evaluating safety and efficacy of the RG7916 dose level selected from Part 1. This study is recruiting globally.

JEWELFISH: An ongoing, 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.

SMA TYPE I , II, III – Clinical Trials with RG7916

Nya säkerhetsdata från SUNFISH del 1 och molekyl RG7916 vid patienter med typ 2 eller 3 spinal muskulatrofi

Roche meddelar positiva säkerhetsdata från den kliniska fas 2/3-studie med läkemedelskandidaten och molekyle RG7916 på AAN2018 -kongressen

We have previously presented an early analysis of SUNFISH Part 1, which showed that RG7916 administration results in a dose-dependent increase in full-length SMN2 mRNA and a concomitant decrease in SMN2Δ7 mRNA. Recent analysis of SMN protein levels in whole blood showed that in patients with SMA, SMN protein increased in a dose-dependent manner up to median 2.5-fold. The safety, tolerability and PK/PD data from Part 1 informed the selection of a RG7916 dose level for SUNFISH Part 2 predicted to lead to clinically efficacious increases in SMN protein. To date, no drug-related adverse events leading to withdrawal have been observed.

SUNFISH (NCT02908685) — type 2 och 3 SMA

Roche släpper stor uppdatering på de två molekylerna RG7916 och OLESOXIME – pågående studier

SMA TYPE I , II, III – Clinical Trials with RG7916

Roche veröffendlicht neue Informationen über laufende Studien von RG7916 bei SMA Typ I, II , III

Liz schrieb bereits in Schwedisch darüber. Roche hat ein aktuelles Update über die laufende Studien über das Molekül RG7916 veröffendlicht

Die Medizin wird oral verabreicht und in Roche Studie gibt es auch Erwachsene Patienten mit SMA Typ 3, (25 Jahre) die daran teilnehmen.

RG7916 ist ein SMN2-Spleißmodifikator und wird in drei laufenden klinischen Studien, FIREFISH, SUNFISH und JEWELFISH, untersucht.

FIREFISH für Babys mit Typ 1 SMA – Beginn Teil 2

FIREFISH besteht aus zwei Teilen: Teil 1 bewertete das Sicherheitsprofil von RG7916 bei verschiedenen Dosierungen und zeigte, dass RG7916 bei allen Dosierungen sicher und gut verträglich war. In Teil 1 hat Roche die Konzentration von RG7916 und den SMN-Proteinspiegel im Blut gemessen. Dies hat Roche nun bewegt, die in Teil 2 zu untersuchende Dosis von RG7916 zu bestätigen. Teilnehmer in Teil 1 erhalten weiterhin RG7916 in einer offenen Verlängerungsphase.

Teil 2 von FIREFISH ist nun in Arbeit und wird die Wirksamkeit und Sicherheit von RG7916 bei ungefähr 40 Babys untersuchen. Teil 2 der Studie hat eine Laufzeit von zwei Jahren, nach der alle Babys weiterhin RG7916 erhalten, bis es in den Ländern verfügbar ist. Die primäre Analyse der Wirksamkeit und Sicherheit von RG7916 findet statt, nachdem alle Patienten ein Jahr lang behandelt wurden.

Die Rekrutierung für FIREFISH Teil 2 hat in Belgien, Frankreich und Italien begonnen. Weitere Länder und Standorte werden nach Zustimmung des Gesundheitsamts und der Ethikkommission schrittweise eröffnet.

SMA Clinical Trials with RG7916/
Roche bringing patient support for spinal muscular atrophy
Roche släpper stor uppdatering på de två molekylerna RG7916 och OLESOXIME – pågående studier

Roche släpper stor uppdatering på de två molekylerna RG7916 och OLESOXIME – pågående studier

Roche släpper ut en stor uppdatering på Firefish för typ 1 SMA- Fas 2 ska till att börja. Rekrytering för Firefish Del 2 har börjat i Belgien, Frankrike och Italien.

JEWELFISH/strong> är en studie som undersöker säkerhet och tolerans hos RG7916 hos personer med SMA typ 2 och 3 som tidigare deltagit i en studie med en annan SMN2-specifik molekyl. Rekryteringen fortsätter i studiecentra i Europa och USA.

RG7916 utvecklades i samarbete med PTC Therapeutics och SMA Foundation

OLEOS-studier är en pågående öppna förlängningsstudier, icke-ambulerande typ 2 och 3 SMA-patienter som har deltagit i tidigare studier med Olesoxime. Patienter övervakas i flera år.

Båda molekylerna ges via mun (eller g-tub) och fungerar på olika sätt.   Som ett resultat kan det sprida effekterna i varje cell i kroppen och inte bara i centrala nervsystemet ensam

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Clincial Trias with RG7916

Roche bringing patient support for spinal muscular atrophy

Roche -veröffentlicht neue Informationen über laufende Studien von RG7916 bei Smatyp 1-3/