Dragonfly Therapeutics, Inc.’s Post

Have you reviewed the latest FDA guidance that addresses the use of PROs in cancer clinical trials? It provides technical guidance about incorporating the right PROs into trials that facilitate regulatory approval. The FDA guidance provides a more encompassing and detailed, designed to help trialists, regulators and drug developers understand best how to use aspects of PRO data including the structure of data, handling missing PRO data, and its statistical analysis. Learn how Clinical ink is uniquely positioned to understand the use of these aspects to support a drug's approval, and how our innovative solutions collect, store, and analyze PRO data for trial success. #PatientReportedOutcomes #CancerResearch #FDAGuidance

Today we filed our fiscal year end financials with SEDAR and shared a summary of key 2023 accomplishments, including positive T1D clinical results, along with select 2024 milestones (it's going to be an exciting year for Sernova!): 📈 Additional data from Cohort 2 of the ongoing U.S. Phase 1/2 clinical trial, which is evaluating its enhanced 10-channel Cell Pouch in patients with T1D, are expected beginning in Q1. ⚕️Additional preclinical data to support an IND application to evaluate the Company’s autograft thyroid cells in patients suffering from thyroid disease, with no immunosuppressive regimen required. 🤝 Potential engagement with additional life sciences focused companies, academic institutions and external parties through partnership and collaboration opportunities, which could be announced over the course of 2024. 💰 Additional funding to support growth through strategic alliances, credit facilities and/or institutional equity financings with the goal of maximizing shareholder value. #T1D #IND #CellTherapy #Biotechnology

Promising #phase1 #clinical trial. RBS2418 is a nucleoside-derived small molecule that selective binding to the active site of ENPP.

One of the most important aspects of this is the up-and-coming platform to deal with patients with thyroid disease. As Cynthia and I discussed in our recent interview (see https://lnkd.in/ezdTkQAW) it could actually supplant T1D near-term as Sernova's most important and monetarily lucrative application.

In conversations about #CRISPR, we often focus on the science, rather than the patient perspective. So it was a pleasure to speak with Jimi Olaghere about how #Casgevy has changed his life! 🧬✂️ Jimi was one of the first patients to receive Casgevy to treat his #sicklecelldisease. Hear him discuss the incredible impact of this treatment, how he got involved in the clinical trial, his thoughts on making genomic medicines equitable, and his advice for other patients who are considering taking Casgevy. 🩺💉 You can watch the full video at the link below. Thanks as always to my interview co-pilot, Kevin Holden! 👨🔬 Stay tuned for the next video in this series for Synthego Corporation - more expert perspectives are on the way.

While we are disappointed with the topline results of the Phase 3 soticlestat trials announced by Takeda, medical research is not without risk. It is important to focus on the key question – Can one benefit patients? As Takeda states they are going to engage with the health authorities based on the totality of the data we have accumulated over 8 years. The results of the clinical trials will be presented at an upcoming scientific congress. We wish great success to the team at Takeda. We sold soticlestat back to Takeda in 2021 and have not been involved in these trials following the very successful Phase 2 readouts from the trials we and Takeda ran together. We used the capital from the sale of soticlestat to Takeda to build an exciting and differentiated pipeline that holds tremendous promise for people living with epilepsies and brain conditions. Our clinical and preclinical pipeline of small molecules is rich, promising and novel. We anticipate several near-term clinical milestones, and we will continue to move forward to advance our mission of bringing new treatment options to patients.

Q3 #FDA and #EMA #DrugApprovals reflect a research focus on novel therapeutic targets and companion diagnostics for several malignancies: https://lnkd.in/eeVbnS3a

Antibody-Drug Conjugate (ADC) is an exciting and growing field. 11 FDA approved ADCs., ten times more in clinical trials and yet so many aspects to investigate. If you are not familiar with the topic here is a didactic review on the technology and the latest trends. https://lnkd.in/eFe-ABdi Having worked on ADCs I highly recommend that read.

Very interesting phase II basket trial looking at using cell-free DNA to test for HER2 amplification. In this trial, patients who were HER2 amplified according to cell-free DNA had a median progression-free survival of 7.0 months when given trastuzumab deruxtecan. This does appear to be a proof of concept that shows that we might be able to start testing for HER2 via liquid biopsy, avoiding issues with tissue sampling. One issue is that 4,734 patients underwent testing, 252 were considered HER2 amplified, and 62 patients were included in the study. So overall, very few patients were considered HER2 amplified, and it's hard to conclude anything definitively from 62 patients. Trastuzumab deruxtecan does have a cytotoxic effect independent of its binding to HER2, so the question is, would this have been better than salvage chemotherapy? #enhertu #her2 https://lnkd.in/e2UBujBm

Tiziana Life Sciences Ltd (NASDAQ:TLSA) has announced six-month data showing positive #clinical improvements related to Modified Fatigue Impact Scale (MFIS) scores and similar important clinical measures of physical function in foralumab-treated, non-active Secondary Progressive Multiple #Sclerosis (na-SPMS) patients participating in an Expanded Access (EA) Program. The clinical-stage #biopharmaceutical company said numeric improvements were seen across key multiple sclerosis measures, including the Modified Fatigue Impact Scale, Expanded Disability Status Scale, Timed 25-Foot Walk test, Pyramidal Scores and NeuroQoL Fatigue scores.  "Intranasal #foralumab is continuing to show clinical improvements over time in patients with na-SPMS where foralumab targets inflammation in the brain," #Tiziana Life Sciences acting CEO Gabriele Cerrone said in a statement.   More at #Proactive #ProactiveInvestors http://ow.ly/wA6g104XVCg #NASDAQ #TLSA