September 19, 2024 - 🧬 [nGram] Today’s Neurology Scoop: FDA Approves Merck’s KEYTRUDA, argenx in The Lancet, Anokion's MS Data

1. FDA approves Merck’s Keytruda plus chemotherapy for advanced malignant pleural mesothelioma

   • The FDA has approved Keytruda (pembrolizumab) in combination with pemetrexed and platinum chemotherapy for first-line treatment of unresectable advanced or metastatic malignant pleural mesothelioma (MPM).
   • Approval is based on the Phase 2/3 IND.227/KEYNOTE-483 trial, which showed a 21% reduction in the risk of death compared to chemotherapy alone.
   • Keytruda plus chemotherapy also improved progression-free survival (PFS) and overall response rate (ORR) compared to chemotherapy alone.
   • The trial enrolled 440 patients and demonstrated significant improvements in overall survival (OS), PFS, and ORR.
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2. argenx announces publication in The Lancet Neurology of pivotal ADHERE study data in chronic inflammatory demyelinating polyneuropathy

   • ADHERE was the largest and most innovative clinical trial of CIDP patients to date.
   • VYVGART Hytrulo demonstrated a 61% reduction in disease progression and reduced risk of relapse.
   • The study showed rapid onset of action, with significant improvements in mobility, function, and strength.
   • 99% of trial participants elected to participate in the ADHERE open-label extension.
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3. Anokion announces new data from the phase 1 MoveS-it study supporting ANK-700 for relapsing-remitting multiple sclerosis

   • Anokion presented new data at the 40th Congress of ECTRIMS in Copenhagen, Denmark.
   • The Phase 1 MoveS-it study evaluated the safety and tolerability of ANK-700 in 34 patients with RRMS.
   • ANK-700 showed no Serious Adverse Events (SAEs) and no evidence of disease exacerbation.
   • Pre-clinical data demonstrated the potential of ANK-700 in expanding antigen-specific regulatory T-cells.
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4. Alzprotect announces supportive results from open-label extension of phase 2a study for progressive supranuclear palsy

   • Alzprotect completed the open-label extension (OLE) phase following the Phase 2a trial of AZP2006 for PSP.
   • Patients on AZP2006 showed delayed disease progression and stabilization, especially when treated early.
   • Even late-stage PSP patients who started AZP2006 in the OLE phase experienced stabilization.
   • No significant safety concerns were observed during the OLE phase.
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5. New Alzheon peer-reviewed publication on APOLLOE4 phase 3 trial design and baseline characteristics

   • Alzheon published a new scientific paper on the APOLLOE4 Phase 3 trial, focusing on APOE4/4 homozygotes with early Alzheimer's disease.
   • The trial is a 78-week, randomized, double-blind, placebo-controlled study involving 325 subjects aged 50-80 years.
   • ALZ-801/valiltramiprosate, the investigational oral therapy, aims to block neurotoxic beta amyloid oligomers and has shown favorable safety results.
   • Topline data from the trial is expected in late 2024, with a long-term extension study ongoing in the US, UK, and Canada.
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6. IGC Pharma announces additional data supporting IGC-AD1 as an Alzheimer's treatment

   • IGC Pharma's IGC-AD1 shows promising effects on tau tangles and spatial memory.
   • Preclinical studies reveal IGC-AD1 reduces hyperphosphorylation of tau protein by approximately 10%.
   • Morris Water Maze tests indicate a 50% faster reduction in escape latency times in treated mice.
   • IGC-AD1 is in a Phase 2 clinical trial for treating agitation in dementia due to Alzheimer's.
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7. Xspray Pharma announces positive FDA meeting and plans for Dasynoc NDA resubmission

   • Xspray Pharma plans to resubmit its NDA for Dasynoc in Q4 2024, following a productive meeting with the FDA.
   • The FDA recommended adjustments to Dasynoc's tablet strengths to reduce medication errors, requiring new batches to be produced.
   • Xspray Pharma has initiated production of these new batches and will provide further clarification on the manufacturing process.
   • A new PDUFA date will be assigned upon resubmission, with a final decision expected within two to six months.
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8. InnoCare announces approval of clinical trial of BCL2 inhibitor ICP-248 for acute myeloid leukemia in China

   • InnoCare Pharma received approval for an Investigational New Drug (IND) to conduct a clinical trial of BCL2 inhibitor ICP-248 in combination with azacitidine for acute myeloid leukemia (AML) in China.
   • AML is a malignant hematological disease originating from hematopoietic stem/progenitor cells, accounting for about 80% of acute leukemia in adults.
   • ICP-248 is a novel, orally bioavailable BCL2-selective inhibitor that selectively inhibits BCL2 and restores the mechanism of programmed cell death.
   • InnoCare aims to accelerate the clinical development of ICP-248 to benefit patients with hematological malignancies.
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9. Azitra receives fast track designation for ATR-04 for skin rash from EGFR inhibitors

   • Azitra, Inc. has been granted Fast Track Designation by the FDA for ATR-04, a treatment for moderate to severe EGFR inhibitor-associated dermal toxicity.
   • ATR-04 is a live biotherapeutic product containing a genetically engineered strain of Staphylococcus epidermidis.
   • The Fast Track Designation aims to expedite the development and review process, potentially allowing for accelerated approval or rolling review.
   • Azitra plans to initiate a multicenter, randomized, controlled Phase 1/2 clinical trial of ATR-04 by the end of 2024.
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