Celltelligence’s Post

#Clinicalfindings Andexanet for Factor Xa Inhibitor–Associated Acute Intracerebral Hemorrhage Patients on factor Xa inhibitors within 15 hours of hemorrhage were randomly assigned to receive #andexanet or usual care. Although andexanet showed higher hemostatic efficacy (67.0% vs. 53.1%; P=0.003) and greater reduction in anti-factor Xa activity (94.5% vs. 26.9%; P<0.001), more #thrombotic events (10.3% vs. 5.6%; P=0.048), including ischemic stroke (6.5% vs. 1.5%) were observed. #HCPs, how best can the thrombotic events be managed in the clinical setting? Funded by Alexion Pharmaceuticals, Inc. Rare Disease; ANNEXA-I ClinicalTrials.gov number, NCT03661528.). https://lnkd.in/gNsn8ZuK

Kashiv BioSciences Announces Completion of Enrollment for Phase III Clinical Trial of ADL018, a Biosimilar Candidate to XOLAIR® (omalizumab) The completion of patient enrollment for global Phase III trial of ADL018, most advanced biosimilar candidate following the FDA approval of Releuko® and Fylnetra® in 2022. This milestone underscores their commitment to advancing our biosimilar pipeline and providing cost-effective therapeutic options. This pivotal study aims to compare the efficacy, safety, tolerability, and immunogenicity of ADL018 with XOLAIR® in patients with chronic idiopathic/spontaneous urticaria (CSU).

The FDA has approved Anktiva (nogapendekin alfa inbakicept-pmln) intravesical solution, in combination with Bacillus Calmette-Guérin (BCG), for the treatment of adult patients with BCG-unresponsive non–muscle invasive #bladdercancer with carcinoma in situ with or without papillary tumors. For all the latest drug news, check out our drug referential resource: https://ow.ly/jfz450Rnclg #DrugNews #Pharmacy #Medication

Today Atara announced that it submitted a Biologics License Application (BLA) to the U.S. FDA for tabelecleucel (tab-cel®) indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with Epstein-Barr virus (#EBV) positive post-transplant lymphoproliferative disease (#PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate. This represents the first-ever #allogeneic #Tcell therapy BLA submission to the FDA, and if approved, tab-cel would be the first approved therapy in the U.S. for EBV+ PTLD. Read more about this significant milestone for Atara: https://bit.ly/4dL7elE

Redx Pharma PLC (AIM:REDX) said it has kicked off a phase I clinical trial for RXC008, a 'first-in-class' ROCK inhibitor treatment targeting fibrostenotic Crohn's disease. This study aims to assess the safety and pharmacokinetic profile of the drug - in other words, its activity in the body over some time, including the processes by which RXC008 is absorbed, distributed in the body, localised in the tissues, and excreted. It will be assessed in healthy volunteers with the results from the study expected by the end of this year. Chief executive Lisa Anson told investors: "We are delighted to confirm that the first participant has been dosed in the RXC008 phase I clinical study. "RXC008 is a potential first-in-class treatment for patients with fibrostenotic Crohn's disease, a debilitating condition where successive surgeries are the only treatment option available today. More at #Proactive #ProactiveInvestors #AIM #REDX #Crohnsdisease #treatment http://ow.ly/SBp8105k03L

Entrada Therapeutics, which received early funding from CureDuchenne, reported positive data from a Phase 1 ENTR-601-44-101 trial in healthy volunteers. ENTR-601-44, which is designed to skip exon 44, was well-tolerated, and the six individuals in the highest dose cohort (6 mg/kg) demonstrated an average of 0.44% exon skipping after a single dose. This was statistically significant compared to placebo control. Entrada plans to submit regulatory applications in Q4 2024 to initiate separate global Phase 2 clinical trials of ENTR-601-44 for Duchenne amenable to skipping exon 44, and for ENTR-601-45 for Duchenne amenable to skipping exon 45. They also plan to submit regulatory applications in 2025 for a global Phase 2 for Duchenne amenable to skipping exon 50. Link to press release: https://lnkd.in/gnd7kvUN

Outlook Therapeutics, Inc. has submitted a Special Protocol Assessment (SPA) request for the required additional adequate and well-controlled study of ONS-5010. The revised protocol is the subject of the SPA request, in which Outlook Therapeutics is seeking further confirmation from the FDA that NORSE EIGHT, if successful, addresses the #FDA’s requirement for a second adequate and well-controlled clinical trial to support the resubmission of the ONS-5010 BLA for wet AMD. The FDA is expected to respond to the SPA in early February 2024. NORSE EIGHT will be a randomized, controlled, parallel-group, masked study of neovascular age-related macular degeneration subjects randomized in a 1:1 ratio to receive 1.25 mg ONS-5010 or 0.5 mg #ranibizumab intravitreal injections. Subjects will receive injections at Day 0 (randomization), Week 4, and Week 8 visits. Approximately 400 patients are expected to be enrolled in the study. The company expects to resubmit the ONS-5010 BLA by the end of calendar year 2024 to include the results of NORSE EIGHT and the additional CMC work to address the issues identified by FDA in the Complete Response Letter issued in August 2023 to support approval. #AMD #MacularDegeneration #biosimilar #retina #ophthalmology https://shorturl.at/tB134

Join us at the 18th Annual Pain Therapeutics Summit (https://lnkd.in/evDCNbu) on October 28-29, 2024 in Boston, MA! On the second day, our CEO Prof. Geert Jan Groeneveld will give a presentation on proof-of-concept in early phase clinical studies of new analgesics. CHDR has created a comprehensive nociceptive test battery, PainCart, to screen new analgesic compounds for their potential to influence various pain mechanisms, both nociceptive and neuropathic. This allows us to determine the analgesic potency and profile of new drugs in healthy volunteers, establishing the active concentration range to aid in dose selection for initial patient studies. PainCart has been used to generate proof-of-concept for Nav1.8 blockers, advancing them to phase 2 and phase 3 trials, and to make early no-go decisions for clinical drug programs. In his presentation, Prof. Groeneveld will introduce PainCart and he will provide examples of both types of studies. #clinicalresearch #pain #painresearch

Exciting News in Cardiovascular Research! Today marks a significant milestone as a groundbreaking small molecule drug, CardiolRx™ currently in Phase II trials for recurrent pericarditis, receives Orphan Drug Designation from the FDA. This achievement underscores its potential to transform the lives of patients. Kudos to the team at Cardiol Therapeutics Inc. for their dedication. 👇Learn more about this groundbreaking development and the ongoing MAvERIC-Pilot study in the comments below. #MedicalInnovation #healthcareadvancements #cardiovascularresearch

Projecting ourselves, hopefully, to an approval: an interesting nuance on cell therapy (allogeneic but not universal, thus requiring the maintenance of an HLA-typed inventory). And thus, another one with a, hem, *interesting* business model. But given the almost-complete lack of *business* in CGT these days (outside of CAR-Ts), we'll watch anything! #cellandgenetherapy