Infantile Convulsions and Paroxysmal Choreoathetosis, Familial

The FDA is taking issue with a promotional website from Taiho Oncology, alleging it misleadingly describes the benefits of its bile duct cancer drug Lytgobi, which won accelerated approval in 2022. The untitled letter sent last week calls out the way the company’s website offers allegedly misleading interpretations of efficacy in the single-arm trial on which the accelerated approval was based. The agency said certain efficacy-related parts of the webpage misbrand Lytgobi “by misleadingly suggesting that FOENIX-CCA2,” a Phase 1/2 study in 103 patients , “provided interpretable results regarding the effects of Lytgobi on PFS [progression-free survival] and OS [overall survival] endpoints.” Because FOENIX-CCA2 was designed as a single-arm trial, with no comparator arm, the FDA added, “the reported PFS and OS results are uninterpretable; absent an appropriate comparator, it is not possible to determine if the observed effect is attributable to Lytgobi or to other factor(s), such as the natural history of the disease.” The FDA acknowledged that Taiho said the data presented on its site should be interpreted with caution, but noted that such statements do “not render the promotional communication nonmisleading in light of the issues with FOENIX-CCA2.” Presentations on the website are also “misleading by suggesting that Lytgobi improves DCR [disease control rate] in patients with locally advanced or metastatic iCCA [intrahepatic cholangiocarcinoma] based on a composite of complete response (CR), partial response (PR), and stable disease (SD) when the study from which the presentations were drawn could not demonstrate this result,” the agency said. In a statement to Endpoints News , Taiho said it worked to present data “in a manner that is truthful and not misleading and consistent with FDA guidelines” and was “reviewing their comments and will respond appropriately and in a timely manner.” This is the second untitled letter this year from the FDA’s Office of Prescription Drug Promotion to industry.

DOVER, Del. and EPALINGES, Switzerland, Dec. 10, 2024 /PRNewswire/ -- Pilatus Biosciences Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its leading molecule, PLT012, for treating liver and intrahepatic bile duct cancer (HCC/ICCA). Achieved in November 2024, this designation marks a crucial advancement in developing innovative therapies for patients facing these challenging cancers. Dr. Raven Lin, CEO, emphasized the company's commitment to addressing urgent medical needs, while Prof. Ping-Chih Ho highlighted PLT012's unique therapeutic approach targeting the tumor microenvironment. Pilatus is actively working with regulatory authorities to expedite PLT012's development and availability. Continue Reading Pilatus Biosciences, a preclinical-stage biopharmaceutical company spun out from the Ludwig Institute for Cancer Research (Lausanne), is leading the development of first-in-class biologics targeting metabolic checkpoints. Supported by the Cancer Research Institute (New York), the company employs a pioneering approach to immunometabolism, reprogramming the immune microenvironment to combat cancer effectively. "We are honored to receive Orphan Drug Designation for PLT012, a milestone that reflects our dedication to addressing the urgent need for innovative therapies in liver and intrahepatic bile duct cancer," said Dr. Raven Lin, CEO and Co-founder of Pilatus Biosciences. Prof. Ping-Chih Ho, Chair of the Scientific Advisory Board and Co-founder of Pilatus Biosciences, added, "PLT012 leverages metabolic checkpoint targeting to reprogram the tumor microenvironment (TME), offering a unique therapeutic approach. This designation highlights the promising scientific discoveries and results we have achieved in addressing the underserved area. It further motivates us to accelerate PLT012's development and collaborate globally to bring this promising treatment to patients with limited options." Currently, Pilatus Biosciences is advancing the development of PLT012, actively engaging with regulatory authorities and stakeholders to expedite the availability of this promising therapy. About PLT012 PLT012, is a humanized anti-CD36 antibody with a unique dual mechanism of action (MOA): it simultaneously disarms immunosuppressive cell populations and amplifies effector T cell functions. PLT012 has shown potential against multiple tumors with unmet medical needs. It is set to advance to its first U.S. IND submission and first patient dosing in 2025. As a monotherapy, PLT012 demonstrates remarkable anti-tumor efficacy in both immune 'hot' and 'cold' tumor models with a significant augmentation in GzmB-expressing CD8+ T cells and reductions in both intratumoral Tregs and pro-tumorigenic macrophage. Additionally, PLT012 treatment alters the exhaustion features of cytotoxic CD8+ T cells by increasing the populations of progenitor- (Texprog) and tumoricidal activities in terminal-exhausted T cells (Texterm), highlighting enhanced anti-tumor immunity when combined with immune checkpoint blockade therapies, such as PD-1 or PD-L1 inhibitors. About Orphan Drug Designation The FDA's Orphan Drug Designation (ODD) program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. ODD qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and 7 years marketing exclusivity upon FDA approval. About Liver and Intrahepatic Bile Duct Cancer (HCC/ICCA) Primary liver cancer first occurs in either the liver or the intrahepatic bile ducts. The two most common types of liver and intrahepatic bile duct cancer are hepatocellular carcinoma (HCC, 80-90% of cases), and intrahepatic cholangiocarcinoma (ICCA, 10-15% of cases). In HCC and ICCA, metabolic reprogramming plays a crucial role in promoting tumor progression by modifying the TME to support tumor growth and immune evasion. For HCC, the most common first-line systemic therapies include either a combination of a PD-L1 inhibitor and a VEGF inhibitor or a combination of a PD-L1 inhibitor and a CTLA-4 inhibitor. Most patients will require multiple lines of treatment, as the recurrence rate of HCC has been reported to be as high as 88%. Second-line treatments for HCC are primarily multiple tyrosine kinase receptor inhibitors, even the incidence of a second HCC recurrence is 50%-70%. Additional lines of treatment may be administered if the patient can tolerate a second-line therapy with a different MOA than those previously administered. PLT012 emerges as a promising candidate, demonstrating dual MOA that synergizes with existing treatments and provide immune-stimulating effects in the TME, potentially enhancing therapeutic outcomes. About Pilatus Biosciences Inc.: Pilatus Biosciences is pioneering in discovering and developing first-in-class antibodies and bifunctional proteins that target metabolic checkpoints, with the goal of driving immuno-microenvironment reprogramming to combat cancer. With deep expertise in immunometabolism research, Pilatus Biosciences partners with leading cancer research institutions and hospitals worldwide. To strengthen its R&D capabilities, the company established a lab in Taiwan in July 2024, focused on supporting early clinical development and biomarker discovery. Pilatus Biosciences operates globally, utilizing a cross-border functional team and fostering external collaborations to maintain an agile, cost-efficient development platform, driving its efforts to create groundbreaking therapies. For more information about Pilatus Biosciences and its work in the field of oncology, please visit . SOURCE Pilatus Biosciences WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Results show that percutaneous hepatic perfusion with CHEMOSAT® is an effective and safe treatment option for patients with advanced cholangiocarcinoma and has the potential to prolong life in patients with inoperable, treatment-refractory liver metastases. NEW YORK, Dec. 5, 2022 /PRNewswire/ -- Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, announced the publication of a retrospective analysis of patients who underwent a percutaneous hepatic perfusion procedure (PHP) with CHEMOSAT for the treatment of either inoperable intrahepatic cholangiocarinomas (iCCA) or extrahepatic cholangiocarinoma (eCCA) with liver metastases. The article, New perspectives in unresectable cholangiocarcinoma? Evaluation of chemosaturation with percutaneous hepatic perfusion as a palliative treatment option by Dr. Cornelia L. A. Dewald, et al, was a retrospective analysis of 17 patients who underwent a total of 42 PHP procedures between October 2014 and September 2020 at the Hannover Medical School in Germany. The aim of the retrospective, monocentric study was to analyze PHP as a palliative treatment for unresectable liver dominant CCA. 16 of the 17 patients were evaluable for response as one patient died without follow-up imaging 13 weeks after the first PHP with no identifiable relationship to the PHP treatment. After the first PHP, one patient (6%) presented with a complete response (CR). Three patients (18%) had a partial response (PR) in the first follow-up exam and seven patients (44%) presented with stable disease (SD). Five patients (31%) had progressive disease (PD), one of which was limited to extrahepatic progression only. In total, in 17 treated patients an overall response rate (ORR) of 25% and a disease control rate (DCR) of 75% was achieved. Two patients with PR, six patients with SD and the patient with PD limited to extrahepatic progression received further PHP treatments. In the subsequent follow-up exams, the overall best therapy response in these patients was PR in 78% and SD in 22%. One patient was treated in total with 8 PHP treatments within 30 months. The median progression free survival (PFS) was 3.5 (95% CI:2.2–7.4) months with a similar median hepatic PFS of 3.6 [95% CI: 2.6–9.5] months. Calculated from first diagnosis of iCCA (or CCA liver metastases), the median survival was 27.6 [95% CI: 16.5–37] months. From first PHP, a median survival of 9.9 [95% CI: 3.8–21] months was observed, with a 1-year survival rate of 41%. For context, the authors noted that for inoperable CCA, the treatment options are limited and a median survival of 2.5 – 6 months is to be expected, which can be extended to approximately 12 months under first-line chemotherapy with gemcitabine and cisplatin. In this study all patients were previously treated with at least systemic therapy and the authors note that the results of their analyses confirms the potential for survival extension by PHP treatment even after the exhaustion of systemic therapies. No significant complications occurred during the PHP treatments. Significant, but transient and clinically manageable, hemotoxicity was reported with grade 3/4 thrombocytopenia after 50%, anemia after 26% and leukopenia after 21% of the PHP treatments. There were no PHP-related deaths. The authors stated that the toxicity rates were consistent both with previously published PHP values and with first-line systemic therapy with gemcitabine/cisplatin in CCA. The authors highlighted the increasing importance of locoregional forms of therapy in the treatment of CCA and that the new edition of the German S3 cancer guideline "Diagnostics and Therapy of Hepatocellular Carcinoma and Biliary Carcinomas" now includes PHP with melphalan for the treatment of inoperable iCCA or eCCA liver metastases. Based on the results of this study the authors concluded that for patients with inoperable, treatment-refractory iCCA and CCA liver metastases PHP is an effective and safe treatment option that has the potential to prolong life in a palliative setting. CCA are the second most common primary liver tumors, with an incidence of approximately 1.6 per 100,000 in the US and Europe, the majority of which are either iCCA or eCCA which become liver dominant. Radical surgical resection to tumor-free margins is the only curative therapy for non-metastatic CCA. Particularly in the case of iCCA however, due to long asymptomatic phases which often lead to an advanced tumor stage at initial diagnosis, less than 30–40% of patients are operable and there is a high risk of recurrence at 40–80% after surgical tumor resection. About Delcath Systems, Inc. Delcath Systems, Inc. is an interventional oncology company focused on the treatment of primary and metastatic liver cancers. The company's proprietary percutaneous hepatic perfusion (PHP) system is designed to administer high-dose chemotherapy to the liver while controlling systemic exposure and associated side effects. In the United States, the PHP system is being developed under the tradename HEPZATO KIT (melphalan hydrochloride for injection/hepatic delivery system), or HEPZATO, for the treatment of patients with unresectable hepatic-dominant metastatic ocular melanoma (mOM), also known as metastatic uveal melanoma (mUM) and is considered a combination drug and device product regulated by the United States Food and Drug Administration (FDA). In Europe, the PHP system is regulated as a Class lll medical device and is approved for sale under the trade name CHEMOSAT Hepatic Delivery System for Melphalan, or CHEMOSAT, where it has been used at major medical centers to treat a wide range of cancers of the liver. Contact: Investor Relations Contact: Ben Shamsian Lytham Partners 646-829-9701 [email protected] SOURCE Delcath Systems, Inc.