FDA grants priority review to SpringWorks Therapeutics’ new drug application for mirdametinib for the treatment of adults and children with NF1-PN
- The FDA has accepted SpringWorks Therapeutics' NDA for mirdametinib, granting it Priority Review with a PDUFA action date of February 28, 2025.
- The European Medicines Agency has also validated the Marketing Authorization Application for mirdametinib.
- Mirdametinib could become the first approved therapy for adults and a best-in-class therapy for children with NF1-PN.
- The submissions include data from the Phase 2b ReNeu trial, which showed robust objective response rates and a manageable safety profile.
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European Commission approves Astellas' Padcev in combination with Keytruda for first-line treatment of advanced urothelial cancer
- The European Commission has granted Marketing Authorization for Padcev (enfortumab vedotin) in combination with Keytruda (pembrolizumab) for first-line treatment of advanced urothelial cancer.
- Approval is based on positive overall survival and progression-free survival results from the Phase 3 EV-302 trial.
- The combination nearly doubled median overall survival and significantly extended progression-free survival compared to platinum-containing chemotherapy.
- Astellas is working with local regulatory authorities to ensure patient access across the EU.
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Yunovia announces IND approval by the MFDS to initiate phase 1 MAD study for the small molecule GLP-1 agonist
- Yunovia received IND approval from the MFDS of Korea for a Phase 1 Multiple Ascending Dose (MAD) study of ID110521156.
- The study will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ID110521156.
- ID110521156 is an orally available small molecule GLP-1 agonist aimed at treating obesity and diabetes.
- The company plans to collect PD data, including continuous glucose monitoring and body weight changes, from a 4-week MAD study.
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Ocugen completes dosing in high dose cohort of phase 1/2 GARDian clinical trial for Stargardt disease
- Ocugen has completed dosing in the third cohort of its Phase 1/2 GARDian clinical trial for OCU410ST, targeting Stargardt disease.
- Three subjects received a single subretinal injection of the highest dose (2.25Ă—10^11 vg/mL) being tested.
- The trial is being conducted at six leading retinal surgery centers across the U.S.
- The GARDian trial will assess the safety and efficacy of OCU410ST, with Phase 1 being a multicenter, open-label, dose-ranging study.
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Telix submits NDA for TLX101-CDx brain cancer imaging agent
- Telix Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for TLX101-CDx (Pixclara), a PET imaging agent for glioma.
- Pixclara has been granted Orphan Drug and Fast Track designations by the FDA, facilitating expedited review.
- The agent aims to improve diagnosis and management of glioma, particularly in the post-treatment setting.
- Pixclara is also being developed as a companion diagnostic agent for TLX101, Telix’s investigational neuro-oncology drug candidate.
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Foundation Medicine collaborates with Merus to advance treatment options in NRG1 fusion-driven tumors
- Foundation Medicine partners with Merus to develop an RNA platform as a companion diagnostic for Merus' bispecific antibody zenocutuzumab (Zeno).
- Zeno targets the neuregulin/HER3 tumor-signaling pathway in solid tumors with NRG1 fusions, showing potential effectiveness in preclinical studies.
- The FDA has accepted a Biologics License Application for Zeno under priority review for NRG1+ non-small cell lung cancer (NSCLC) and pancreatic cancer (PDAC).
- Foundation Medicine’s RNA platform can detect fusions in 318 genes and supports biomarker discovery with gene expression reporting of over 1,500 genes.
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Bayer and NextRNA Therapeutics enter strategic collaboration to develop small molecules targeting long non-coding RNAs in oncology
- Bayer and NextRNA Therapeutics have entered a collaboration and license agreement to develop small molecule therapeutics targeting lncRNAs in oncology.
- The collaboration will focus on two oncology programs, with one currently in early preclinical development and the other based on targets identified by NextRNA's platform.
- NextRNA will receive up to $547 million, including upfront and milestone payments, research funding, and tiered royalties on net sales.
- Bayer gains access to NextRNA’s approach to inhibit lncRNAs by disrupting their interaction with RNA-binding proteins using small molecules.
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Everest Medicines announces interim results for first half of 2024
- Total revenue for H1 2024 reached RMB 301.5 million, a 158% increase from H2 2023.
- NEFECON® was commercially launched in mainland China and Singapore, and approved in Hong Kong.
- XERAVA® continues to show robust sales growth in China.
- Etrasimod was approved in Macau and is expected to launch in the Greater Bay Area of China.
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Harbour BioMed announces 2024 interim results
- Reported revenues of US$23.7 million and an overall profit of approximately US$1.4 million for the first half of 2024.
- Batoclimab (HBM9161) BLA re-submitted to NMPA in June 2024, accepted in July 2024, with consistent efficacy and safety data.
- Porustobart (HBM4003) shows positive data in monotherapy and combination trials for various solid tumors.
- Nona Biosciences entered into significant collaborations with Boostimmune, Astra Zeneca, and Alaya.bio in 2024.
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Keymed Biosciences announces interim results for first half of 2024
- Presented long-term efficacy and safety data from Phase III trial of Stapokibart for moderate-to-severe AD at EAACI 2024.
- NDA for Stapokibart for CRSwNP and SAR accepted by NMPA and granted priority review.
- AstraZeneca presented Phase I study data of CMG901 (AZD0901) for advanced G/GEJ cancer at ASCO 2024.
- Submitted IND application for CM313 to assess in patients with primary immune thrombocytopenia.
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