Lutetium Dotatate LU-177

FDA approves first Menkes disease treatmentThe FDA cleared Zycubo (copper histidinate) from Zydus Lifesciences' US subsidiary Sentynl Therapeutics as the first treatment for paediatric patients with Menkes disease, a rare and often fatal inherited disorder of copper deficiency, offering a subcutaneously-administered copper replacement therapy."For the first time, patients have access to an approved therapy, offering hope where no options existed," said Sharvil Patel, managing director of Zydus.The approval was backed by two single-arm trials comparing 66 Zycubo recipients with 17 untreated controls. Results showed that initiating treatment within four weeks of birth cut mortality by 78%, with nearly half surviving beyond six years — some over 12 years — while no untreated patients lived past six; commencing treatment later also delivered meaningful survival gains.The drug, rights of which were gained from Fortress Biotech in 2023, was issued a complete response letter by the US regulator last year, owing to manufacturing site deficiencies.Novartis goes shopping in China for next RLTNovartis signed a worldwide licensing deal with China-based Zonsen PepLib Biotech for a peptide-based radioligand therapy (RLT) asset, agreeing to pay $50 million upfront and unspecified potential future development, regulatory and sales milestones, plus tiered royalties.Novartis will take over global development and commercialisation of the undisclosed programme, which PepLib has advanced internally. "This asset complements our industry-leading efforts to advance promising next-generation RLTs," said Shiva Malek, global head of oncology, Biomedical Research, Novartis, which is anchored by Pluvicto (lutetium Lu 177 vipivotide tetraxetan) for PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) and Lutathera (lutetium Lu 177 dotatate) for certain neuroendocrine tumours.Last week, the company revealed plans to build a new RLT manufacturing facility in Winter Park, Florida — its fourth in the US, with a fifth planned as well. The Florida site, expected to come online in 2029, is designed to improve the delivery of highly time-sensitive RLT doses, helping Novartis maintain its target of administering more than 99% of treatments on the planned day.PepLib, which was founded in 2017 and employs more than 200 people across offices in China and New Jersey, said it has built proprietary peptide libraries supporting multiple modalities, including peptide radionuclide conjugates, peptide oligonucleotide conjugates and peptide drug conjugates.Oricell's CAR-T ambitions buoyed by $70M raiseOricell Therapeutics closed a $70 million series C1 financing as it looks to advance its pipeline of CAR-T cell therapies for solid tumours. The funding round was co-led by Beijing Medical and Health Care Industry Investment Fund and Qiming Venture Partners, with other participants including NGS Super, E-Town Capital, Elikon Venture and Talon Capital. The biotech's lead programme, Ori-C101, is an autologous GPC3-targeted CAR-T therapy for advanced hepatocellular carcinoma, which is entering a registrational pivotal trial. Beyond this, Oricell's secreting CAR-T, OriC902, has shown efficacy and durability in ultra-late-line and difficult-to-treat solid tumours, while it is also working on dual-targeted in vivo CAR-T.Oricell — which raised $45 million in 2023 and over $120 million a year earlier — will use the latest proceeds to accelerate its global expansion and clinical development, while strengthening its technological capabilities and preparing for commercialisation.Marea's acromegaly therapy moves to Phase IIMarea Therapeutics reported positive top-line results from its Phase I study of MAR002, a first-in-class monoclonal antibody targeting the growth hormone receptor (GHR) for acromegaly. Data showed "robust and durable" suppression of insulin-like growth factor-1 (IGF-1) in healthy adult male volunteers, with peak reductions of 52% at the highest tested dose and more than 45% suppression out to 43 days, potentially allowing for biweekly to monthly dosing. The company plans to begin a Phase II/III study of MAR002 in mid-2026."This is compared to a maximum suppression of 48%...and greater than 45% suppression lasting to only five days with the current commercially available GHR antagonist," Marea said. MAR002 was well tolerated in healthy volunteers, with no drug-related safety signals observed. Marea also highlighted progress on its lead cardiometabolic programme, MAR001, a monoclonal antibody targeting ANGPTL4 to reduce remnant cholesterol and atherosclerotic cardiovascular disease risk. Enrolment in the Phase IIb TYDAL-TIMI 78 study is expected to finish by the end of the month, with several data readouts due in 2026. A half-life-extended version, MAR001-HLE, is advancing toward Phase III readiness with a streamlined Phase I pharmacokinetics bridging study planned for later this year.RFK Jr. adds two more members to CDC vaccine panelHealth and Human Services (HHS) Secretary Robert F. Kennedy Jr. appointed two obstetrician-gynaecologists, Kimberly Biss and Adam Urato, to the Centers for Disease Control and Prevention's Advisory Committee on Immunization Practices (ACIP). Kennedy said the appointments "bring the scientific credentials, clinical experience, and integrity this committee requires" and emphasised his commitment to "transparency, gold standard science, and diverse expertise in guiding the nation’s immunization policies." The two join 11 other members selected after Kennedy fired the previous panel of experts and reconstituted ACIP last June.Urato, a maternal-foetal medicine specialist with prior academic appointments at Harvard, Tufts, and the University of South Florida, has participated in FDA advisory panels. Biss has held leadership roles at Bayfront Health/Orlando Health Bayfront Hospital and her experience includes clinical education roles and publication on COVID-19 vaccine safety for pregnant women, according to an HHS release Tuesday.In 2023, Biss told the GOP-led House subcommittee that after receiving COVID-19 vaccines, some patients in her practice had faced irregularities with their menstrual cycles that were so severe she had to perform surgeries and hysterectomies. She also testified that "my miscarriage rate from year to year went up 100% in 2021" and suggested the vaccines might have been responsible for some women entering early menopause.Meanwhile, Urato has also questioned the safety of vaccines given during pregnancy, including COVID-19, influenza, Tdap and respiratory syncytial virus. He raised concerns about the use of antidepressants in pregnancy and petitioned the FDA last year to add a boxed warning to SSRIs regarding risks of pregnancy complications and alteration of foetal brain development.

Novartis is paying $50 million upfront to license a peptide-based radiopharmaceutical asset from China-headquartered Zonsen PepLib Biotech. The Monday release did not name the candidate, its target or what stage of development it is in. What is known is that PepLib, founded in 2017, is focused on developing peptide-based therapies, including peptide-radionuclide conjugates. The biotech’s preclinical pipeline includes a “radiopharmaceutical carrier” program at the lead-optimization stage. Novartis will be responsible for developing and commercializing the licensed candidate. PepLib could get an undisclosed amount in milestones as well as tiered royalties on future sales. The deal should “complement” Novartis’ existing portfolio of radioligand therapies (RLTs), according to the release. The Swiss drugmaker is currently a market leader in RLTs due to Pluvicto for metastatic castration-resistant prostate cancer and Lutathera for gastroenteropancreatic neuroendocrine tumors. The two assets together pulled almost $800 million in third-quarter sales last year. Error: Survey not found. In its clinical RLT pipeline, Novartis is advancing a lutetium-based candidate targeting GRPR and a separate program targeting FAP, among others. The company has been ramping up its capabilities in the space, opening its third US-based RLT manufacturing site in November. Last week, it unveiled plans to build a fourth factory in Florida. In November, Novartis signed a licensing pact with Ratio Therapeutics for its SSTR2-targeting radiopharmaceutical program. In 2024, it acquired radiopharmaceutical startup Mariana Oncology for $1 billion upfront . In a separate announcement on Monday, Novartis licensed a preclinical candidate for Alzheimer’s disease from SciNeuro for $165 million upfront. In December, it teamed up with London-based biotech Relation to discover new drugs for immune diseases with the help of AI.