OrphAI Therapeutics has been granted Orphan Drug Designation by the EU for AIT-101, aimed at treating amyotrophic lateral sclerosis (ALS). Following its US FDA approval, this marks a significant step toward battling this neurodegenerative disease. https://lnkd.in/e3rTq-K6
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The final months of 2024 may include an onslaught of highly-anticipated drug approvals. Medicines among the list include those that target cystic fibrosis, lung cancer and the rare genetic nervous system disorder AADC deficiency and if approved, patients suffering from these conditions will enter the New Year with a number of new treatment options. Learn more on these 5 FDA decisions to watch, below. #FDA #marketaccess #drugdevelopment
5 FDA decisions to watch in the fourth quarter
biopharmadive.com
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Enterprise Therapeutics has dosed the first patient in its pivotal Phase IIa clinical trial, which is investigating the efficacy and safety of ETD001 — an epithelial sodium channel (#ENaC) inhibitor designed to enhance hydration and mucus clearance in patients with cystic fibrosis. The study is also focusing on the suitability of such a treatment for the 10% of cystic fibrosis patients who are ineligible for CFTR modulators. #cysticfibrosis #clinicaltrials #CFTRmodulators #ENaCinhibitors https://lnkd.in/env3Jv_N
Enterprise Therapeutics' ETD001 Phase II trial doses first cystic fibrosis patient
manufacturingchemist.com
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Don't miss the #VT1021 update from Vigeo Therapeutics COO Dr. Jing Watnick at the Fifth Annual Glioblastoma Drug Development Summit in Boston MA this week. Dr. Watnick will be speaking during the afternoon session on 28 March on The #VT1021 Ph1/2 clinical program in #glioblastoma as well the potential future use of #VT1021 in Neuro-inflammatory diseases #GBM #Glioblastoma #NeuroinflammatoryDiseases #VT1021
Vigeo Therapeutics to Present at the 2024 Glioblastoma Drug Development Summit in Boston Massachusetts - Vigeo
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Recognizing the imprecision of initially establishing a drug dose, the FDA initiated Project Optimus in 2021, the goal of which is to optimize the treatment dose in relation to tolerability. The author and colleagues conducted a clinical trial of the "less is more" approach to drug dosing. Azacitidine (Vidaza) is approved by the FDA to treat myelodysplastic syndromes (MDS) with 7-day dosing on a 28-day chemotherapy cycle. It prolongs survival in patients with higher-risk MDS, but the 7-day dosing schedule is probably too toxic for patients with lower-risk MDS. Decitabine (Dacogen) is also approved to treat MDS, with 5-day dosing on a 28-day chemotherapy cycle. Bottom line: 3 day dosing was just as effective. More such studies are needed for treatments with high real life levels of discontinuation due to intolerability. But where will funding come from? Big Pharma is unlikely to promote research that may negatively affect it’s bottom line. #chemotherapydosing #myelodysplasticsyndrome #mds
Opinion | Ending Oncology's Toxic Relationship With High Chemotherapy Doses
medpagetoday.com
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TiNivo-2 Trial Does Not Meet Progression-Free Survival For Patients With Renal Cell Carcinoma: There were positive efficacy and safety results for tivozanib monotherapy consistent with previous analyses. #finance #pharmacy #lifesciences
TiNivo-2 Trial Does Not Meet Progression-Free Survival For Patients With Renal Cell Carcinoma
pharmacytimes.com
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CymaBay to be acquired by Gilead Sciences for USD 4.3 billion An investment in CymaBay Therapeutics by both Arctic Aurora LifeScience and Biotech Select is bearing fruit today, as the liver disease specialist has agreed to be acquired by Gilead Sciences for USD 4.3 billion, or USD 32.5 per share, representing a 26.5% premium to last Friday's close, while the stock has returned 35.4% this year. CymaBay's main asset is seladelpar, a potentially best-in-class molecule for the treatment of primary biliary cholangitis (PBC), an autoimmune disease that causes excruciating itching, among other symptoms. In PBC, the patient's immune system attacks the bile duct, causing it to become inflamed and slowly destroyed, which can ultimately lead to liver failure. In a pivotal Phase 3 trial, seladelpar significantly improved patients' condition, with a particularly strong effect on pruritus, which has a positive impact on patients' quality of life. Based on these data, the company is awaiting the FDA's decision on the drug's approval, which is expected by 14 August this year. Gilead has a historic presence in hepatitis and has been active in other fibrotic and inflammatory liver diseases, so the acquisition strengthens its presence in this area and has been well received by the markets.
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RAPT Therapeutics recently faced a significant setback as the FDA placed a clinical hold on two critical trials of zelnecirnon, targeting atopic dermatitis and asthma, due to a severe liver failure case in one patient. This incident led to a dramatic 67% drop in RAPT's stock price. Despite this challenge, it's important to note that the clinical hold does not affect RAPT’s oncology trial with tivumecirnon. RAPT emphasizes the isolated nature of this liver failure incident and is conducting a thorough investigation. Key Takeaways - 🔷 The complexity of drug development and the paramount importance of patient safety. 🔷 RAPT is committed to understanding the incident and working closely with the FDA to resolve the hold swiftly. This development serves as a crucial reminder of the delicate balance between innovation and safety in the pursuit of new therapies. #HealthcareInnovation #RAPTTherapeutics #PatientSafety #FDA #BiotechNews
FDA Puts Clinical Hold on RAPT Therapeutics’ Lead Candidate in Two Trials | BioSpace
biospace.com
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Another day, another life-changing scientific break through. Parkinson's particularly hits a raw nerve. Anyone who's seen a senior citizen suffering from this terrible affliction has got to feel some joy today. The bizarre paradox of America: The world's best medical innovation ecosystem coupled with the world's worst care delivery system. And "system" is being generous. The better word is probably "situation." #Biotech #Biotechnology #Bigpharma #Pharmaceutical #Parkinsons #Neurology
Cerevel Therapeutics on Thursday reported positive data from its Phase III TEMPO-3 trial, showing that tavapadon can significantly improve symptom control in patients with Parkinson’s disease. #pharma #biospace https://hubs.li/Q02tk5FN0
Cerevel Gets Phase III Parkinson’s Win on Heels of $8.7B AbbVie Acquisition | BioSpace
biospace.com
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💊 Breaking news: Drugmaker Eli Lilly and Company’s new disease-modifying #Alzheimers drug #donanemab — brand name Kisunla™ — has been approved by the FDA. Here's what you need to know: ✔️ This is the third monoclonal antibody drug for Alzheimer's approved by the #FDA. ✔️ Monoclonal antibodies are used in diseases like cancer treatments but in the past couple of years, have been approved for Alzheimer's. ✔️ MAB drugs work by mimicking our immune system to rid the brain of amyloid plaque, a early biomarker of Alzheimer's. ✔️ These drugs are only meant for very early stage patients diagnosed with MCI or early stage Alzheimer's with confirmed beta amyloid plaque. ✔️ Clinical studies have showed a very small slowdown in the disease. ✔️ This is only one piece of the puzzle for preventing Alzheimer's disease from advancing. 🔗 Read the full article:
FDA Approves New Alzheimer’s Drug Kisunla (Donanemab)
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Faron Pharmaceuticals Limited (AIM:FARN, OTC:FPHAF) has marked a significant step in the #BEXMAB trial by administering the first dose of #bexmarilimab to a patient with #MyelodysplasticSyndrome (MDS) who did not respond to standard hypomethylating agents (HMAs). #MDS is a type of #BloodCancer where the bone marrow produces poorly functioning blood cells. HMAs, commonly used in treating MDS, work by modifying DNA to promote healthy cell function. However, some patients do not respond to these treatments, presenting a critical need for new therapies. In its phase II trial, Faron is focusing on patients with HMA-refractory MDS, an aggressive form of #MyeloidLeukemia with limited treatment options. Researchers are conducting a randomised study with parallel assignment, testing bexmarilimab at two dose levels (3 mg/kg and 6 mg/kg). More at #Proactive #ProactiveInvestors #AIM #OTC #FARN #FPHAF #Faron #FaronPharmaceuticals http://ow.ly/fpsC1058gcV
Faron Pharma trains sights on hard-to-treat leukemia sufferers with latest leg of trial
proactiveinvestors.co.uk
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