Awny Farajallah, MD, FACP’s Post

Bridge Biotherapeutics, Inc. has completed patient enrollment in the Phase 2 clinical study evaluating the efficacy, safety, and tolerability of BBT-877 in patients with idiopathic pulmonary fibrosis (IPF). BBT-877, an experimental Autotaxin (ATX) inhibitor, demonstrated its ability to inhibit lysophosphatidic acid (LPA) production by up to 90 percent in the first-in-human study. We look forward to sharing topline data from this study in the first half of 2025. James Lee, founder and CEO of Bridge Biotherapeutics, said: "The completion of enrollment in the phase 2a clinical study of BBT-877 marks an important milestone in our efforts to develop innovative treatments for patients suffering from IPF. Closely looking for business alliance opportunities in the global pharmaceutical industry, we will remain dedicated to advancing this novel drug candidate, which we believe has the potential to make clinically meaningful outcomes in IPF patients." Learn more: https://prn.to/3WGCpbi #Bridging_Science_and_Patients #IPF #Autotaxin #fibrosis

Recently, at the 2024 European Hematology Association (EHA) meeting, Gilead announced that data from the Phase III ENHANCE study indicated that the efficacy of Magrolimab was not only unproven but may also have adverse effects on untreated high-risk myelodysplastic syndromes (MDS) patients. This suggests that the development of Magrolimab may be headed towards failure. The story of Gilead and Magrolimab dates back to March 2020. At that time, Gilead announced the acquisition of FortySeven for $4.9 billion in cash, with a focus on incorporating FortySeven's leading candidate, Magrolimab, to strengthen its own immuno-oncology research portfolio. Magrolimab initially showed remarkable performance. In September of the same year, the FDA granted Magrolimab Breakthrough Therapy designation based on positive results from an ongoing Phase Ib study. Data presented at the 2020 EHA meeting showed that 91% (n=33) of evaluable patients receiving Magrolimab in combination with azacitidine achieved an objective response, with 42% achieving a complete response (CR). However, dramatic reversals often occur unexpectedly, and Gilead found itself at the center of a storm. This is not the first time Gilead has taken a big gamble and failed, experiencing a series of setbacks. Over the past five years, it has spent over $40 billion with little to show for it so far. Gilead's substantial investments seem to demonstrate that money, indeed, cannot solve everything. #API #Pharmaceutical #Medical #Pharma

It’s exciting to see the results of this impressive cross-institutional collaboration to bring the first fully approved therapeutic for all DMD patients, valmorolone (Agamree), to the UK! ✅This impressive cross-institutional feat is a big win for translational research and involved many important key players from non-profit, academic, and government sectors. ⭐️The focus on de-risking in preclinical data generation and relying on validation across platforms and institutions with reliable and robust efficacy metrics was critical to the success of valmorolone’s approval and fulfills an important stated goal of one partner in this effort, TREAT-NMD, to “address the fragmentation currently hindering translational research for cutting edge therapies in rare neuromuscular diseases” and “accelerate the clinical application of promising treatments” (https://lnkd.in/gmEqZPX6).

First phase 3 results from CARTITUDE-4: Cilta-cel versus standard of care (PVd or DPd) in lenalidomide-refractory multiple myeloma. Ide-cel or Standard Regimens in Relapsed and Refractory Multiple Myeloma

Determined scientific endeavor and new drug ambitions push back innovation boundaries as leading Oncology R&D now firmly establishes itself in China

#TransThera Announces First Patient Dosed in Pivotal Clinical Trial in China Evaluating #Tinengotinib in Patients with Advanced #Cholangiocarcinoma and Disease Progression Following #FGFR inhibitor therapy Nanjing, China | November 21, 2023, TransThera Sciences (Nanjing) Inc., a clinical-stage biopharmaceutical company dedicated to innovating differentiated drugs globally, today announces the first patient has been dosed for the pivotal trial FIRST-CN08 of tinengotinib (TT-00420), an investigational next generation FGFR inhibitor for advanced cholangiocarcinoma（CCA）patients who have been refractory/relapsed to FGFR inhibitors.   The pivotal trial is a multi-center, single-arm, open-label study to be conducted in approximately 30 sites across China. The trial will evaluate the efficacy and safety of tinengotinib orally as monotherapy once a day in patients with advanced FGFR2 altered CCA who were relapsed or refractory on prior systemic chemotherapy and an FGFR inhibitor. Details on this trial are available on ClinicalTrials.gov, under identification number NCT06057571. “Dosing the first patient in (FPI) the pivotal FIRST-CN08 trial of tinengotinib for this disease setting is an important milestone and first step to what we hope will eventually allow us to bring a novel next generation FGFR inhibitor to these patients with high unmet medical needs,”  said Jean Fan, M.D., Chief Medical Officer of TransThera Sciences. “We are extremely appreciative of all the efforts and support by those who have worked on the study to achieve this FPI milestone. Tinengotinib has been granted breakthrough therapy designation (BTD) by the CDE (Center for Drug Evaluation) of the National Medical Products Administration (NMPA) in China for the treatment of CCA. We underscore our commitment to advancing the development of tinengotinib that has potential to demonstrate clinical benefit for CCA patients who have been refractory/replapsed to FGFR inhibitor. We look forward to the progress in the pivotal FIRST-CN08 study and the opportunity to bring the novel therapy to CCA patients.” For more information, please visit www.transthera.com

FDA Rejects Gastric Cancer Drug Zolbetuximab by Astella Due to Manufacturing Concerns The FDA has denied approval for Astellas Pharma's gastric cancer drug, zolbetuximab, due to concerns about the manufacturing process at a third-party facility. While the FDA's concerns are not related to the drug's clinical safety or efficacy, Astellas Pharma is collaborating with the FDA and the manufacturer to address the manufacturing issues. Zolbetuximab had previously received priority review status and positive results from Phase III clinical trials. Regulatory authorities in China and Japan are also evaluating the drug. Astellas Pharma remains committed to bringing zolbetuximab to patients despite this setback. For more details please click the link! https://lnkd.in/dGt-xjap #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

#Antibody-drug conjugates (#ADCs) dominated 2023 dealmaking, with some of the largest drugmakers – Pfizer, Merck & Co., AbbVie, and Bristol Myers Squibb – inking pacts worth billions. Now Roche, which markets two of the earliest approved ADCs, is investing in the space once again through a partnership with MediLink announced Tuesday that could be worth close to $1 billion. Roche is gaining exclusive global rights to YL211, an ADC targeting c-Met to treat solid tumours. The firm’s China Innovation Center will partner with MediLink Therapeutics to start a Phase I trial of YL211. MediLink Therapeutics will receive $50 million, comprising an upfront payment and near-term milestones, and is eligible for additional development, regulatory, and commercial milestones for a total deal value near $1 billion, plus tiered royalties." https://lnkd.in/dhMRePMC. #carletto #linkedin #vita #salute #sanita #donna #uomo #pharma

Organon Expects 2028 'Tidal Wave' For Biosimilars 👉 Who should read this? US biosimilar players. 👉 What is it? A breakdown of how Organon perceives near-term and slightly longer-term opportunities in the US biosimilars market. 👉 Why is it important? Organon's CEO breaks down several aspects of interest to US biosimilar players: current competition trends on Humira (adalimumab); future launches such as biosimilars to Perjeta (pertuzumab) and Prolia/Xgeva (denosumab); and further ahead, planned rivals to Opdivo (nivolumab) and Keytruda (pembrolizumab). Citeline Commercial Citeline #pharmaceuticals #biopharmaceuticals #genericdrugs #genericmedicines

Looking back on some ⚡highlights⚡ I'm celebrating from 2023: ➡ I co-authored a chapter on “Regional-Specific Studies” for the Regulatory Affairs Professionals Society (RAPS) textbook "Fundamentals of Pharmaceutical and Biologics Regulations: A Global Perspective" https://lnkd.in/e5jbpmeF and authored a chapter on “Dose Optimization of Oncology Drugs: A Clinical and Regulatory Perspective” for the new book, “Emerging Topics in Dose-Finding and Beyond”, to be published by Springer Nature Group in 2024 as a volume in the ICSA Book Series in Statistics https://lnkd.in/e2BsU24i ➡ I also wrote several scientific papers, including one on a decade of #FDA approvals for #pediatriconcology drugs in Elsevier EJC Paediatric Oncology https://lnkd.in/eNu9vfvs and one on FDA approvals of #oncology drugs for #tissueagnostic indications in Springer Nature Group Targeted Oncology https://lnkd.in/gAsG9PYG Another paper on #doseoptimization of targeted therapies will be published in a Cancers MDPI Special Issue in 2024. https://lnkd.in/g-ii5VND ➡ I wrote several pieces on #regulatory developments for the industry publications MedCity News https://lnkd.in/e5qVWVjg and Clinical Leader https://lnkd.in/e6QExRjA https://lnkd.in/guuAtzuE (one of which was recognized as a #Top10 contributed article for 2023!) https://lnkd.in/eTE3sfyb ➡ I spoke at several conferences, including Cambridge Healthtech Institute 14th Annual Summit for Clinical Ops Executives (SCOPE) #SCOPE2023 in Orlando in February, Terrapinn Festival of Biologics #FestOfBiologics conference in San Diego in March, and Hanson Wade Group 13th World Clinical Biomarkers & CDx Summit in Boston in September. More talks and panels lined up in the coming year, beginning with the marcus evans Group 5th Edition Excellence in #RealWorldEvidence and Data Utilization conference in Philadelphia in January. ➡ Finally, I received recognition from The Healthcare Technology Report (as a Top 25 Healthcare Technology Leader of Virginia) https://lnkd.in/ekJ6SPJj and from TOPRA - The Organisation for Professionals in Regulatory Affairs (with an Award for Regulatory Excellence in the category of Education). https://lnkd.in/eg4ufD7z I want to sincerely thank each of you for the support you’ve given me over the past year. Looking forward to an incredible 2024 - Happy New Year! 🥂