Denali Therapeutics’ Post

𝐀𝐮𝐭𝐨𝐥𝐮𝐬 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬 𝐀𝐝𝐯𝐚𝐧𝐜𝐞𝐬 𝐁𝐫𝐞𝐚𝐤𝐭𝐡𝐫𝐨𝐮𝐠𝐡 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐟𝐨𝐫 𝐀𝐝𝐮𝐥𝐭 𝐁-𝐂𝐞𝐥𝐥 𝐀𝐜𝐮𝐭𝐞 𝐋𝐲𝐦𝐩𝐡𝐨𝐛𝐥𝐚𝐬𝐭𝐢𝐜 𝐋𝐞𝐮𝐤𝐞𝐦𝐢𝐚 Autolus Therapeutics plc (Nasdaq: AUTL) Autolus Therapeutics, a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announces that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for obecabtagene autoleucel (obe-cel). Obe-cel is Autolus’ lead investigational chimeric antigen receptor (CAR) T cell therapy, for the treatment of patients with relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL). The BLA submission is based on data from the Pivotal Phase 2 FELIX study of obe-cel in adult r/r B-ALL. The data which were presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting in June 2023 and will be updated at the upcoming Annual Meeting of the American Society for Hematology Meeting in December in San Diego. “We are looking forward to continuing working with the FDA through the regulatory approval process,” commented Dr. Christian Itin Christian Itin, Chief Executive Officer of Autolus. “I would like to thank the treating physicians, patients, caregivers, and the dedicated team at Autolus for their support, trust and commitment for the program to reach this important milestone.” Autolus plans to submit a Marketing Authorization Application for obe-cel in relapsed/refractory ALL to the European Medicines Agency (EMA) in the first half of 2024. Obe-cel has been granted Orphan Drug Designation by the FDA, Orphan Medical Product Designation by the EMA, Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA and PRIority MEdicines (PRIME) designation by the EMA for adult r/r B-ALL. #medicine #fda #healthcare #submission #celltherapy #treatment #therapeutics #patient

Rise Therapeutics, a clinical-stage biotechnology company developing novel oral immunotherapeutic medicines, today announced that it has enrolled its first patient in its R-2487 Phase 1 rheumatoid arthritis clinical trial. "This is an important milestone for the company and patients suffering from rheumatoid arthritis" states Gary Fanger President and Chief Executive Officer of Rise Therapeutics. "This achievement reinforces our commitment to deliver innovative new medicines to the clinical community." The Phase I rheumatoid arthritis clinical trial (NCT05961592) is a first-in-human clinical trial designed to understand safety, pharmacodynamics, and clinical activity of R-2487 in patients suffering from rheumatoid arthritis. The clinical study is being conducted in the United States at multiple sites around the country. This single and repeat dose study in patients is positioning R-2487 to intervene in rheumatoid arthritis progression prior to traditional infused biologics therapy. The study will enroll up to 36 participants where clinical activity will be evaluated by improvement in disease severity and using a variety of key biomarker and pharmacodynamic assessments.

"Pliant Therapeutics: Targeting Idiopathic Pulmonary Fibrosis Bexotegrast has received Fast Track Designation and Orphan Drug Designation from the FDA for IPF and PSC. Phase 2a study evaluating bexotegrast against IPF in a study called INTEGRIS-IPF. That trial met its main and secondary goals in early May but did not impress the investment community. The goals of the study were around safety and tolerability, which were met, and the trial produced no drug-related serious adverse events. Phase 3 studies probably don't initiate until 2025 at the earliest." https://lnkd.in/gZNJMHsB

PATRITUMAB DERUXTECAN BLA RECEIVES CR LETTER FROM FDA DUE TO INSPECTION FINDINGS AT THIRD-PARTY MANUFACTURER Daiichi Sankyo and Merck partnered to bring this first in class HER3 directed ADC to patients, and FDA had a PDUFA goal date of earlier this week.   Instead of an approval they got a CR letter, Complete Response (or non approval) citing findings pertaining to an inspection of a third party manufacturing facility. No issues with safety or efficacy data.   Daiichi/Merck filed for Accelerated Approval based on a single arm trial (n=225) in third line EGFR mutated patients – very refractory patients – with a response rate of about 30% and good durability. If approved, patritumab would have met a high unmet need. Presumably this issue at the manufacturing facility can be corrected but it will likely take some time. Highlights how all areas must be inspection ready, manufacturing sites are an integral and often underappreciated piece in drug approvals and subsequent delivery to patients with cancer.   https://lnkd.in/eigVnDKJ  

Kyverna Therapeutics Announces FDA Clearance of Phase 2 IND for KYV-101, a Fully Human CD19 CAR T-Cell Therapy to Treat Myasthenia Gravis EMERYVILLE, Calif., November 13, 2023 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Kyverna), a patient-centered clinical-stage biopharmaceutical company focused on developing cell therapies for patients suffering from autoimmune diseases, today announced the clearance of its fifth Investigational New Drug (IND) application for KYV-101 by the U.S. Food and Drug Administration (FDA). This will allow Kyverna to initiate a Phase 2 open-label, multicenter study of KYV-101, an autologous, fully human CD19 chimeric antigen receptor (CAR) T cell product candidate for the treatment of myasthenia gravis (MG). The trial, named KYSA-6, expands Kyverna's current pipeline, which includes the ongoing Phase 1 KYSA-1 trial in the U.S. and the ongoing Phase 1/2 KYSA-3 trial in Germany, where KYV-101 is currently investigated in adults with active lupus nephritis, and the ongoing Phase 1/2 KYSA-5 https://lnkd.in/gzvVP8nw

"The [Allogene Therapeutics] ALPHA3 trial will screen patients who are likely to relapse after 1L treatment for enrollment in the trial by using the Foresight Diagnostics CLARITY™ Investigational Use Only (IUO) #MRD test, powered by PhasED-Seq™, which recently received Investigational Device Exemption (IDE) approval from the U.S. Food and Drug Administration (FDA). Leveraging CLARITY’s ultra-sensitive MRD technology, cema-cel will be administered as a one-time infusion immediately upon detection of MRD at the completion of six cycles of R-CHOP or other standard 1L chemoimmunotherapy regimen. When given as a “7th cycle” of frontline treatment to eligible patients with MRD, consolidation treatment with cema-cel has the potential to meaningfully improve 1L cure rates in #LBCL. 'Following the FDA Advisory Committee’s recent recommendation to include MRD as an endpoint to accelerate clinical trials in multiple myeloma, the ALPHA3 trial is yet another step forward towards broader implementation of MRD detection in drug development and clinical decision making,” said Dr. Sandra Close, Chief Operating and Compliance Officer at Foresight Diagnostics. “We believe the Foresight Diagnostics CLARITY MRD platform has the performance to enable actionable treatment decisions at end of therapy when residual disease levels are challenging to detect using conventional methods.'” #oncology #cancer #medicine #precisionmedicine #personalizedmedicine #ctDNA #diagnostics #healthcare

US FDA approved #BMS’s #Augtyro™ (#repotrectinib) to treat NTRK-positive locally advanced or metastatic solid tumors in patients 12 years and older. The approval was based on clinical efficacy demonstrated in TRIDENT-1 trial, with significant response rates in both tyrosine kinase inhibitor (TKI)-naïve and TKI-pretreated patients. This approval marks the second US indication for Augtyro, adding to its previous approval for ROS1-positive NSCLC. Follow our page for more industry updates. #raslss #drugapproval #FDA #FDAapproval #cancer #oncology #cancerresearch #NSCLC #lungcancer #nonsmallcell

Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company pioneering a novel class of small molecule medications designed to combat inflammation, today announced positive results following the successful completion of a Phase 1 clinical trial for its leading candidate, HT-6184, a highly selective and orally bioavailable inhibitor of NLRP3/NEK7, a main driver of immune-inflammatory diseases, hematologic malignancies, and even cancer. David Bearss Ph.D., CEO and President of Halia Therapeutics. "Chronic inflammation caused by aberrant inflammasome activation has been linked to various illnesses, and the results of this study show promise in inhibiting chronic inflammation by disrupting the inflammasome's assembly process. The outcome of this study validates our approach to combat inflammation and paves the way for further clinical studies." The Phase 1 trial, registered under NCT05447546, was a randomized, placebo-controlled, single-center study conducted with healthy volunteers. It consisted of both single ascending dose (SAD) and multiple ascending dose (MAD) escalation phases, with a focus on assessing the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of HT-6184.

#USFDA approved #BMS’s #Augtyro™ (#repotrectinib) to treat NTRK-positive locally advanced or metastatic solid tumors in patients 12 years and older. The approval was based on clinical efficacy demonstrated in TRIDENT-1 trial, with significant response rates in both tyrosine kinase inhibitor (TKI)-naïve and TKI-pretreated patients. This approval marks the second US indication for Augtyro, adding to its previous approval for ROS1-positive NSCLC. BMS got Augtyro through its $4.1 B acquisition of Turning Point Therapeutics in 2023. At the time of the deal, Leerink Partners analysts predicted that Augtyro could reach $1.4 B sales at peak in the first-line setting and $455 M in the second line. Detailed news here: https://lnkd.in/gmuv-Ysk Stay updated on the latest trends and news in the pharmaceutical industry. Follow #RASLSS: https://lnkd.in/de5zNWmK #Pharmaceuticals #FDAappoval #TKI #TyrosineKinaseInhibitor #Oncology #NTRK #SolidTumors #BMS #CancerResearch #PrecisionMedicine #MedicalInnovation #Biotechnology #OncologyResearch #ClinicalTrials #Healthcare #LifeSciences #PharmaInnovation #CancerAwareness #NextGenTherapies