Updated Preliminary Data from SMA FIREFISH Program in Type 1 Babies with Risdiplam

risdiplam- RG7916 is well tolerated at all dose levels with no drug-related safety findings –

SOUTH PLAINFIELD, N.J., June 16, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy (SMA), at the 22nd Annual SMA Researcher Meeting. The data presented by Dr. Baranello demonstrated that at Day 182, over 90% of the babies achieved a greater than 4-point increase in CHOP-INTEND score compared to baseline. The CHOP-INTEND data were further supported by video footage presented by Dr Baranello demonstrating antigravity movements, the ability to control their head, roll, or sit in babies participating in FIREFISH. Part 2 of the pivotal FIREFISH study is ongoing. The SMA program is a collaboration between PTC, Roche, and the SMA Foundation.

”We are delighted that up to 6.5-fold increase of protein production has translated into clinical impact for these babies in the FIREFISH study,” said Stuart W. Peltz, Ph.D. Chief Executive Officer of PTC Therapeutics. ”The survival data and CHOP-INTEND scores are very promising, since babies with Type 1 SMA typically do not experience functional motor milestone improvement based on natural history. We look forward to sharing updates for the programs as the data further develop at upcoming medical meetings.”

The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) is a test designed to measure motor milestone development of patients with SMA Type 1. In the FIREFISH study the median increases in CHOP-INTEND scores were 5.5 points (n=20) at Day 56, 12.5 points (n=16) at Day 119, and 14 points (n=11) at Day 182 of treatment. The proportion of patients achieving greater than a four-point increase from baseline in CHOP-INTEND scores was 75% at Day 56 (n=20), 94% at Day 119 (n=16), and 91% (n=11) at Day 182. In addition, risdiplam has been well tolerated at all dose levels and to date there have been no drug-related safety findings leading to withdrawal. Also, no babies have required a tracheostomy or permanent ventilation since study initiation and no baby has lost the ability to swallow. Median age of patients at first dose in the FIREFISH trial is 6.7 months; oldest patient in the trial is currently 23.8 months old.

”I am impressed by the clinical data and the changes reported by the patients’ families,” stated Dr. Giovanni Baranello, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy. ”Data on motor function seem more encouraging when we consider that we are seeing motor function improvements and milestones achievement at this early stage of the study, which was essentially a dose-finding study and most of the infants included have received their first dose after the age of 5 months. It is exciting to see evidence of clinical benefit from a systemic oral treatment for SMA.”

Risdiplam is an investigational splicing modifier targeting the survival motor neuron 2 (SMN2) RNA, restoring a functional transcript. Risdiplam is taken orally, crosses the blood brain barrier, and shows systemic distribution to the organs that are affected by low levels of SMN protein.

Other presentations included an analysis of data from Part 1 of the SUNFISH study which demonstrated that risdiplam administration resulted in a dose-dependent increase in SMN protein levels up to 3.5-fold; pharmacodynamic data from the JEWELFISH trial; and preclinical data demonstrating SMN protein production and distribution, as well as the development of the SMA Independence Scale (SMAIS).

SUNFISHA 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 risdiplam dose levels. The pivotal SUNFISH Part 2, in non‐ambulant patients with Type 2 or 3 SMA, is evaluating safety and efficacy of the risdiplam dose level selected from Part 1 for 24 months, followed by an open label extension. This study is recruiting globally.

JEWELFISHAn ongoing, exploratory, open-label study to establish the safety and tolerability of risdiplam in people who have previously participated in a study with another therapy targeting SMN2 splicing.

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Mia

Mia

Mother of three childrens. One of the twins my eldest son had SMA Typ II -III. He passad away and spread his wings into the sun . He lived 3 years and seven months. Fly my sweet little honeybee🧚‍♂️🦋 fly my little Honeybee 🐝

5 reaktioner på ”Updated Preliminary Data from SMA FIREFISH Program in Type 1 Babies with Risdiplam”

  1. Vielversprechende Resultate aus der Firefish Study mit Risdiplam (RG7916):

    Die neusten Resultaten von Risdiplam (RG7916) wurden auf der CureSMA 2018 Konferenz veröffentlicht. Die Studie untersucht die Wirksamkeit und Sicherheitsprofil von Risdiplam (RG7916) Babys mit SMA Typ 1.

    Zusammenfassung aus der Studie Teil 1#
    90% der Babies mit SMA Typ 1 zeigten auf der CHOP-Intend Skala das sie 4 bis mehrere Punkte dazu gewonnen haben.
    Babies zeigten auf Videos Bewegungen gegen die Schwerkraft, Kopfkontrolle, Babies konnten sich rollen und sogar sitzen was normalerweise per Definition nie können.

    Risdiplam wird gut toleriert es traten keine Sicherheitsprobleme auf

    Kein Baby brauchte in der Studie eine permanente Beatmung und keines hat die Fähigkeit zu schlucken verloren. Die Kinder waren im 6 und 7 Monate bei der ersten Dosis, das älteste Kind ist zurzeit 23.8 Monate alt.

    Weitere Bemerkenswerte Resultaten ist der bis zu 6.5-fache Anstieg von der SMN-Protein Produktion. Spinraza bekommt damit einen harten Konkurrenten

  2. Lovande kliniska resultat från Firefish-studien av Risdiplam (RG7916):

    De senaste resultaten från Risdiplam (RG7916) publicerades vid CureSMA 2018-konferensen. Studien undersöker effekten och säkerhetsprofilen vid Risdiplam (RG7916) barn med SMA typ I.

    Sammanfattning från studie fas 1 #
    90% av barnen med SMA typ I visade på CHOP-Intend-skalan att de fick 4 eller fler poäng.
    Babys visar via videoen: att hon kunde flyttar sig mot gravitationen, huvudkontroll, spädbarn kunde rulla till och med sitta, som normalt enligt definition aldrig kan.

    Risdiplam tolereras väl, det fanns inga säkerhetsfrågor

    Ingen barn behövde permanent ventilation i studien och ingen förlorade förmågan att svälja. Barnen var 6 och 7 månader vid första dosen, det äldsta barnet är för närvarande 23,8 månader gammalt.

    Andra anmärkningsvärda resultat inkluderar upp till 6,5 ökade SMN proteinnivåer. Spinraza får med Risdiplam en stora konkurrent på marknaden

  3. Lovende kliniske resultater fra Firefish Study af Risdiplam (RG7916)

    De seneste Risdiplam-resultater (RG7916) blev offentliggjort på CureSMA 2018-konferencen. Undersøgelsen viser effekten og sikkerhedsprofilen for Risdiplam (RG7916) børn med SMA type I.

    Resumé fra studiefase #1
    90% af børnene med SMA type I viste på CHOP-Intend-skalaen, at de scorede 4 eller flere punkter
    Babys viser i videoen: at hun kunne bevæge sig mod tyngdekraften, hovedkontrol, spædbarn kan rulle selv sidde, som normalt – hvilket aldrig er muligt.

    Risdiplam er godt tolereret, der var ingen sikkerhedsproblemer

    Ingen børn havde brug for permanent ventilation i studiet, og ingen mistede evnen til at sluge. Børnene var 6 og 7 måneder ved den første dosis, og det ældste barn er for tiden 23,8 måneder gammel.

    Andre bemærkelsesværdige resultater omfatter op til 6,5 5 gange stigning i SMN protein niveauer. Spinraza får en stor konkurrent på markedet med Risdiplam

  4. I see that the future drug for SMA finally got a product name. Promising results 👍

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