Relief Provides Update on Progress and Plans with ACER-001 for the Treatment of Urea Cycle Disorders
Geneva, Switzerland, May 18, 2021 – RELIEF THERAPEUTICS Holding AG (SIX: RLF, OTCQB: RLFTF)(“Relief”), a biopharmaceutical company with its lead compound RLF-100TM (aviptadil) in advanced clinical development to treat severe COVID-19 patients, today provided an update on the development of ACER-001, a proprietary powder formulation of sodium phenylbutyrate (NaPB) designed to be both taste-masked and immediate release, in the lead indication, urea cycle disorders (UCDs). UCDs are a group of rare genetic metabolic disorders which can lead to an excess accumulation of ammonia in the bloodstream, causing different symptoms such as somnolence, coma, and, in the worst case, may lead to multi-organ failure. Better, more affordable treatment options for UCDs are urgently needed.
Regulatory update U.S.: Relief’s partner, Acer Therapeutics, recently announced that it had held a Type B pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA). Acer remains on track to submit an NDA for ACER-001 for the treatment of patients with UCDs in mid-2021, provided that no additional data are requested by the FDA and ongoing development activities are successfully completed (including evaluation of product stability data and reaching agreement on the Pediatric Study Plan (PSP)).
Regulatory update Europe: Relief is responsible for the development and commercialization of ACER-001 in Europe and expects to discuss its plans with the European Medicines Agency this summer. Pending the outcome of these discussions and provided that Acer submits an NDA in the U.S. mid this year as planned, Relief then anticipates submitting a Marketing Authorization Application (MAA) for approval of ACER-001 for the treatment of UCDs in the European Union before the end of 2021.
Based on the timelines outlined above and pending a positive decision by regulators, ACER-001 could be launched in both the U.S. and Europe during 2022.
ACER-001 is also being developed for the treatment of Maple Syrup Urine Disease (MSUD).
Jack Weinstein, Chief Financial Officer and Treasurer of Relief, said, “It is great to see the progress being made with ACER-001. We are excited to continue moving forward with the Acer team to develop and commercialize this product candidate around the globe to address important unmet needs for patients with UCDs, a rare and debilitating set of diseases. Following on the important steps being made by our partner in the U.S., we expect to be able to move forward rapidly in Europe.”
Mal wieder eine eher gute News, jedoch mit viel "wenn und wenn und unter Vorbhalt dass..."
BTW: Über Zeysami wird kein einziges Wort verloren (schon bald 1 Monat her seit der letzten News?). Ich interpretiere diese Meldung weit hergeholt schon fast so, als ob Relief selbst nicht mehr an "ihr" Zeysami" Medikament glaubt.
BTW2: Was mir auch noch aufgefallen ist, RLF schreibt in ihrer Meldung vom 22. April 2021 wieder von der RLF-100 Formulierung und nennt das Ganze nicht mehr Zeysami. Haben die mit NeuroRx bereits komplett abgeschlossen?
Allen Investierten weiterhin viel Durchhaltewillen.