Elixir Medical Corporation’s Post

Is it prime time for #menin inhibitors in #AML and #ALL? With multiple inhibitors in the clinic, we're about to find out the fate of Syndax's first-in-class Menin inhibitor as the FDA ponders the submission. A decision is expected in September. See the review on the current state of the filed. Fingers crossed to give #patients a new option https://lnkd.in/gcvV7FK3

In a Phase III trial, the novel drug achieved near-complete stabilisation of TTR proteins in ATTR-CM patients #drugdevelopment #clinicaltrials #EPRTalks

In a Phase III trial, the novel drug achieved near-complete stabilisation of TTR proteins in ATTR-CM patients #drugdevelopment #clinicaltrials #EPRTalks

Reduced antiarrhythmic drug use, superior freedom from Atrial Arrhythmia recurrence* and fewer repeat ablations are just a few of the positive outcomes we’re seeing two years out with the randomized-controlled CEASE-AF Trial using Hybrid ablation and LAAE compared to endocardial ablation alone. To learn more about the CEASE-AF Trial, visit https://okt.to/mbQeau *Freedom from atrial arrhythmias were defined as atrial fibrillation, atrial flutter, atrial tachycardia in patients off anti-arrhythmic drugs (AADs) except previously failed AADs at doses not exceeding those previously failed. Patients had an enlarged left atrium >4 cm ≤ 6 cm. Hybrid data were directly compared to endocardial ablation alone.

STAT published this article highlighting the overwhelmingly positive data Dr. Christian Ruff from the TIMI Study Group presented this past weekend at #AHA23.   “Bleeding or the fear of bleeding drives substantial under-treatment of patients, which results in devastating strokes,” said Christian Ruff, lead investigator of the trial and director of general cardiology at Brigham and Women’s Hospital. “The hope is if we had an even safer anticoagulant, that that would finally allow us to prescribe it to almost all patients and that they would actually take it, and that would be a substantial advance."    #DYK, novel, dual-acting #FactorXI / XIa inhibitor, abelacimab, met its primary endpoint with a 67% reduction of major or clinically relevant non-major bleeding compared with rivaroxaban. The treatment, injected monthly, also reduced the risk of major bleeding events by 74% and gastrointestinal bleeding by 93%. Ruff also noted that "since abelacimab is a monoclonal antibody, it doesn’t have contrainteractions as many oral treatments do.”   Read more from this informative interview with Elaine Chen. 👇

In the past week, the Celltelligence team detected relevant news that was not discussed in individual blasts but worth highlighting. In this blast, Celltelligence will cover the following topics: • On Friday, March 15, a new Ph3 TRANSFORM FL (https://bit.ly/43qZAYZ) trial evaluating #Breyanzi (#CD19 CAR-T) compared to standard of care (SoC) therapies in r/r FL patients was observed on CT.gov • On Wednesday, March 20, Bristol Myers Squibb announced (press release: https://bit.ly/3VulPek) that the European Commission (EC) has granted approval to #Abecma (Bristol Myers Squibb / 2seventy bio’s #BCMA CAR-T) for the treatment of adult patients with r/r MM who have relapsed after at least two LoTs, including an immunomodulatory agent (#IMiD), a proteasome inhibitor (PI), and an anti-CD38 antibody Celltelligence provides insights: https://bit.ly/CT-865286

AbbVie, a leader in the #migraine space, reported data from the #phase3 PRODROME study showing its migraine drug Ubrelvy (ubrogepant), when given ahead of an expected episode, can safely and effectively reduces the likelihood of developing moderate or severe #headache. Ubrelvy is an oral small molecule drug that binds and inhibits the CGRP receptor typically elevated during migraine attacks. The drug won the FDA’s approval in 2019 for the acute treatment of migraine with or without aura in adults. In the U.S., Ubrelvy is the first oral pill designed to block CGRP. #migrainerelief #migraines

In many respects, 2023 was a very busy year. For FDA, this included 72 approvals of novel therapeutic agents (55 at CDER and 17 at CBER) plus 21 Complete Response Letters (CRLs) for novel agents. More than 35 of these approvals were for #rarediseases including rare cancers, various blood disorders, metabolic, CNS, dermatological, immunological, and infectious diseases, and many more. Behind each #orphandrug approval: new hope for countless patients and families affected by rare diseases that finally have life-altering treatments and cures. https://lnkd.in/e87KRNPi

Endeavor's audacious endeavor to reverse IPF is producing exciting results. The totality of the evidence seems to indicate the drug, ENV-101, might be improving the lung function. This is a giant step forward compared to the current standard of care drugs Ofev and Esbriet, which can only slow down disease worsening with significant tolerability issues to go with it. With that said, it's only 12-week data in a small Ph2a trial - we've seen plenty of spectacular Ph3 failures in IPF in the past, so the jury is still out on whether the efficacy will hold up in a larger and longer trial. In addition, for this novel MoA, will there be any long-term safety issues?

In my new article, I talk about the incredible potential of a unique new drug, SPG302, developed by Spinogenix, Inc., for treating amyotrophic lateral sclerosis (ALS) and how the FDA has approved it for ongoing clinical trials. The drug regenerates the synapses damaged by the fatal disease. In Phase 1 trials conducted in Australia, the drug has proven safe and is now being trialled in people with ALS. It could be a watershed moment in ALS treatment. #ALS #synapse #smallmolecule https://lnkd.in/gbJhSmbx