Celltelligence’s Post

Dear LinkedIn Family, I would like to discuss the following update. FDA decides to withdraw Pepaxto (melphalan flufenamide) On 23rd February 2024, the FDA made a significant announcement regarding the withdrawal of Pepaxto (melphalan flufenamide), a formulation developed by Oncopeptides AB. Pepaxto had previously received accelerated approval for its combined use with dexamethasone in treating certain individuals afflicted with multiple myeloma. The FDA's Center for Biologics Evaluation and Research (CBER) division outlined the reasons behind the withdrawal of Pepaxto: 1. Following a comprehensive review of data from the OCEAN confirmatory studies and extensive deliberation by the Oncologic Drugs Advisory Committee (ODAC), it was determined that Pepaxto did not demonstrate clinical benefit. The ODAC voted 14 to 2 against Pepaxto's favorable benefit-risk profile. 2. The available data did not support Pepaxto's clinical safety and efficacy for its indicated use. The FDA intends to publish this decision as a notice in the Federal Register and remove Pepaxto from the Orange Book. #fda #FDAupdates #oncopeptides #pepaxto #CBER

Check out and subscribe to our BioPharmCatalyst Weekly Watchlist! Stay on top of which companies have key upcoming data readouts and regulatory decisions for the quarter. Weekly Watchlist Recap for June 15th, 2024: -The FDA has extended the Priority Review period for Rocket Pharmaceuticals' (RCKT) BLA filing for KRESLADI, targeting LAD-I, to June 30th, 2024. -Verona Pharma (VRNA) has a PDUFA target action date for ensifentrine, targeting chronic obstructive pulmonary disease, set for June 26, 2024. -Jazz Pharmaceutical (JAZZ) will have a top-line data readout for its Phase 2b trial of suvecaltamide, targeting essential tremor, in late first half of 2024. https://lnkd.in/epJ_t-Qc #watchlist #biotechinvesting #catalysts #fasterscience #clinicaltrials #datareadout #pharma #biotech #biotechstocks

Despite the summer months being slower for most industries, there is still plenty to be excited about in the way of biotech investing in the coming months. Check out our key upcoming catalysts for the quarter to see which companies have important data readouts and regulatory decisions coming up!

Tonix Pharmaceuticals 𝐒𝐮𝐛𝐦𝐢𝐭𝐬 𝐍𝐞𝐰 𝐃𝐫𝐮𝐠 𝐀𝐩𝐩𝐥𝐢𝐜𝐚𝐭𝐢𝐨𝐧 𝐟𝐨𝐫 𝐓𝐍𝐗-𝟏𝟎𝟐 𝐒𝐋 𝐭𝐨 𝐅𝐃𝐀! We're excited to announce the submission of our 𝐍𝐞𝐰 𝐃𝐫𝐮𝐠 𝐀𝐩𝐩𝐥𝐢𝐜𝐚𝐭𝐢𝐨𝐧 𝐟𝐨𝐫 𝐓𝐍𝐗-𝟏𝟎𝟐 𝐒𝐋, a non-opioid treatment for fibromyalgia. The NDA is backed by two successful Phase 3 trials showing significant reduction in pain. If approved, TNX-102 SL will be the first new drug for fibromyalgia in over 15 years! 𝐋𝐞𝐚𝐫𝐧 𝐦𝐨𝐫𝐞 𝐚𝐛𝐨𝐮𝐭 𝐨𝐮𝐫 𝐣𝐨𝐮𝐫𝐧𝐞𝐲 𝐭𝐨 𝐛𝐫𝐢𝐧𝐠 𝐫𝐞𝐥𝐢𝐞𝐟 𝐭𝐨 𝐭𝐡𝐨𝐬𝐞 𝐬𝐮𝐟𝐟𝐞𝐫𝐢𝐧𝐠 𝐟𝐫𝐨𝐦 𝐜𝐡𝐫𝐨𝐧𝐢𝐜 𝐟𝐢𝐛𝐫𝐨𝐦𝐲𝐚𝐥𝐠𝐢𝐚 𝐩𝐚𝐢𝐧. 🔗https://lnkd.in/gWT9fbPn #pharmanews #fibromyalgiatreatment #fdaapproval #drugdevelopment #healthcareinnovation #painrelief #fibrowarriors #fibromyalgiaawareness #chronicpain #medicalbreakthrough #nonopioid #fasttrack #clinicaltrials #newdrug #tonixpharma

We're kicking off day one of #DPHARM2024 with words from Patient Keynote, Sarah Zenner-Dolan, moderated by Peyton Howell, MHA, Parexel. A former pharmaceutical executive, Ms Zenner-Dolan's view of our industry was transformed after being diagnosed with Parkinson's disease and participating in a clinical trial as a patient. Learn more about the 14th annual program here: https://lnkd.in/ejMS4E7a #DPHARM2024 #pharma #patientadvocate

Biosimilars for Regeneron’s aflibercept 2mg are coming to the US. The FDA has given two drugs, Yesafili (Biocon Biologics) and Opuviz (Biogen and Samsung Bioepis), the thumbs up with an interchangeability designation. The FDA has indicated that the drugs’ performance and safety profile were similar according to a battery of comparative measures, including clinical trials. The announcement and its impacts are now reverberating through the American anti-VEGF market for retinal diseases. What will the implications be for both patients and caregivers? https://lnkd.in/gg5sFUhW #antiVEGF #Biosimilars #MediaMICE

Merck Advances Bomedemstat in Phase III Trial for Essential Thrombocythemia Pharmaceutical giant Merck has announced the start of the Phase III Shorespan-007 trial to evaluate bomedemstat (MK-3543) in approximately 300 essential thrombocythemia (ET) patients globally. The study is set to compare bomedemstat to hydroxyurea, the current standard-of-care; and the FDA has granted bomedemstat both the 'orphan drug' and 'fast track' designations for ET and myelofibrosis treatment. This Phase III trial, along with the ongoing Shorespan-006 study, aims to advance bomedemstat as a novel treatment option for patients living with this challenging myeloproliferative neoplasm. At OxyDial, we're encouraged by Merck's progress in developing bomedemstat as a potential new treatment option for patients living with this rare blood disorder. The initiation of this Phase III trial represents an important milestone in advancing innovative therapies that could improve disease control and quality of life for those impacted by essential thrombocythemia. Read more: https://lnkd.in/eiHZwezF #Merck #Bomedemstat #EssentialThrombocythemia #ClinicalTrial #HematologyResearch

Biosimilars for Regeneron’s aflibercept 2mg are coming to the US. The FDA has given two drugs, Yesafili (Biocon Biologics) and Opuviz (Biogen and Samsung Bioepis), the thumbs up with an interchangeability designation. The FDA has indicated that the drugs’ performance and safety profile were similar according to a battery of comparative measures, including clinical trials. The announcement and its impacts are now reverberating through the American anti-VEGF market for retinal diseases. What will the implications be for both patients and caregivers? https://lnkd.in/g5CvXYND #antiVEGF #Biosimilars #MediaMICE

Importance of life cycle management - Regeneron has been taking steps to minimize the hit from incoming biosimilars of its drug Eylea, by pivoting to Eylea HD. The higher-dose version of the drug was approved by the US FDA in August of last year. The new version of the drug has the same indications as the previous version, but a longer dosing gap brings in new IP around the higher-dose version. Regeneron could switch over patients taking the standard dose to the HD version, which would be protected by IP and potential regulatory exclusivity, and cushion the impact from biosimilar drugs expected for the standard dose in 2024. The new dosing also allows Regeneron to take on competition with Roche’s Vabysmo

Final FDA guidance for Oligonucleotide drug development recommends QT prolongation risk assessment, immunogenicity assay development and evaluation of hepatic & renal impaired patients. FDA guidance: https://lnkd.in/g2EmpR8p Read our blog: https://lnkd.in/egviY3vA #RNA #OligonucleotideTherapies #ClinicalDrugDevelopment