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>> TOP ONCOLOGY UPDATES FOR SEPTEMBER 06, 2024
Abdera's FDA Orphan Drug, Akeso & Gilead at ESMO 2024
Abdera Therapeutics
receives FDA orphan drug designation for ABD-147
- ABD-147 is a precision radiopharmaceutical biologic therapy targeting DLL3 on neuroendocrine tumors.
- The FDA's Orphan Drug Designation provides incentives like tax credits, user fee exemptions, and potential market exclusivity.
- Abdera plans to initiate a Phase 1 clinical trial in 2024 for patients with SCLC or LCNEC who previously received platinum-based therapy.
- ABD-147 also received Fast Track designation for treating extensive stage small cell lung cancer (ES-SCLC).
Akeso Biopharma
to present data from 13 clinical studies at ESMO 2024
- Akeso will showcase results from 13 clinical studies on its internally developed antibodies at ESMO Congress 2024.
- Featured antibodies include cadonilimab, ivonescimab, ligufalimab, and penpulimab.
- Studies cover various cancers such as colorectal, breast, head and neck, liver, and more.
- Ivonescimab's combination with ligufalimab will be presented for the first time.
Ribometrix Inc.
to present data supporting potential of eIF4E program in KRAS mutant non-small cell lung cancer at ESMO 2024
- Ribometrix will present data on its eIF4E program at the ESMO Congress in Barcelona, Spain, from September 13-17, 2024.
- The presentation will cover in vitro and in vivo studies of RBX-6610, a small molecule eIF4E inhibitor, for treating KRASG12C mutant NSCLC.
- RBX-6610 monotherapy showed anti-proliferative effects in KRASG12C mutant tumor cell lines, including those with acquired resistance.
- RBX-6610 combined with KRAS inhibitors resulted in significant tumor regression and re-sensitized resistant tumor cells to KRAS inhibitors.
Gilead Sciences
to present new Trodelvy data at the IASLC 2024 World Conference on Lung Cancer
- Gilead will present new data from its lung cancer clinical development program at the IASLC 2024 World Conference on Lung Cancer.
- Key presentations include initial results from the EVOKE-02 study in previously untreated advanced or metastatic non-small cell lung cancer (mNSCLC).
- Subgroup analysis from the EVOKE-01 study shows overall survival improvement in second-line mNSCLC patients.
- Updated data from the TROPiCS-03 study demonstrate promising activity in extensive stage small cell lung cancer (ES-SCLC).
拜耳医药保健
to present new prostate cancer data and continued oncology portfolio research at ESMO 2024
- Bayer will present new oncology data at ESMO 2024, including results from the Phase III ARANOTE trial on NUBEQA (darolutamide) plus ADT in mHSPC patients.
- Late-breaking results from the EORTC's Phase III PEACE-III study on XOFIGO (radium-223 dichloride) in combination with enzalutamide for mCRPC will be presented.
- Updated analysis from the SCOUT and NAVIGATE studies on VITRAKVI (larotrectinib) in TRK fusion cancer patients will be highlighted.
- Trial in Progress (TiP) data from the Phase III SOHO-2 trial on BAY 2927088 for NSCLC with HER2-activating mutations will be presented.
NextPoint Therapeutics, Inc.
announces first-in-class T cell engager NPX372 as new drug candidate targeting B7-H7 in solid tumors
- NextPoint Therapeutics unveils NPX372, a novel T cell engager targeting the B7-H7 axis in solid tumors.
- B7-H7 is an immunomodulatory receptor upregulated in various solid tumors, making it a unique target for tumor-directed therapies.
- NPX372 is a CD3 bispecific antibody that redirects T cell-mediated cytotoxicity toward B7-H7-positive tumors.
- Preclinical data show NPX372's potent anti-tumor responses and favorable safety profile, with no indication of cytokine release syndrome.
- NextPoint is advancing the Investigational New Drug (IND) application for NPX372.
Ocuphire Pharma
initiates VEGA-3 phase 3 trial evaluating phentolamine ophthalmic solution 0.75% for presbyopia
- Ocuphire Pharma has started the VEGA-3 Phase 3 trial for Phentolamine Ophthalmic Solution 0.75% to treat presbyopia.
- The trial is a randomized, double-masked, placebo-controlled study involving 545 participants.
- Participants will receive one drop of the solution or placebo each evening, with the primary endpoint being a 15-letter improvement in near visual acuity.
- Top-line data from the VEGA-3 trial is expected in the first half of 2025.
Aurinia Pharmaceuticals Inc.
announces first participant dosed in AUR200 single ascending dose trial
- Aurinia Pharmaceuticals has dosed the first participant in a Phase 1a single ascending dose (SAD) study of AUR200.
- The study will assess safety, tolerability, pharmacokinetics, and biomarker changes in healthy volunteers.
- Data from the study is expected in the first half of 2025.
- AUR200 targets BAFF and APRIL, cytokines involved in B-cell survival and differentiation, and is intended for autoimmune diseases with high unmet needs.
Crestone Pharma
announces positive data from phase 2 clinical trial of CRS3123 for C. difficile infections
- Crestone reported positive topline results from the Phase 2 trial of CRS3123 in patients with Clostridioides difficile infection (CDI).
- Clinical cure rates at day 12 were 97% for CRS3123 and 93% for vancomycin, with no clinical failures.
- CDI recurrence rates at day 40 were significantly lower for CRS3123 (4%) compared to vancomycin (23%).
- The National Institute of Allergy and Infectious Diseases (NIAID) has provided $4.5 million in new funding for further studies based on these results.
Cyclo Therapeutics, Inc.
presents encouraging preliminary safety data from ongoing pivotal phase 3 study and substudy for the treatment of Niemann-Pick disease type C1
- Cyclo Therapeutics presented positive preliminary data from its ongoing pivotal Phase 3 study (TransportNPC?) and substudy evaluating Trappsol? Cyclo? for Niemann-Pick Disease Type C1 (NPC1).
- The TransportNPC? study, which completed enrollment in May 2024, is the most comprehensive controlled pivotal study for NPC1, involving 104 patients.
- The substudy, focusing on newborns to 3-year-olds, aims to evaluate Trappsol? Cyclo?'s ability to target visceral aspects of NPC1.
- Safety data showed 625 adverse events, with 80% being mild, and no patients withdrawing due to safety concerns.
- Topline data from the 48-week interim analysis is anticipated in H1 2025, with potential marketing applications to follow if data meets statistical significance.
>> TOP NEUROLOGY UPDATES FOR SEPTEMBER 06, 2024
Abdera's FDA Orphan Drug, Ocuphire's Phase 3 Trial, LUCA's Groundbreaking Research
Abdera Therapeutics
receives FDA orphan drug designation for ABD-147
- ABD-147 is a precision radiopharmaceutical biologic therapy targeting DLL3 on neuroendocrine tumors.
- The FDA's Orphan Drug Designation provides incentives like tax credits, user fee exemptions, and potential seven-year market exclusivity.
- Abdera plans to initiate a Phase 1 clinical trial in 2024 for patients with SCLC or LCNEC who previously received platinum-based therapy.
- ABD-147 also received Fast Track designation for treating extensive stage small cell lung cancer (ES-SCLC).
Ocuphire Pharma
initiates VEGA-3 phase 3 trial evaluating phentolamine ophthalmic solution 0.75% for presbyopia
- Ocuphire Pharma has started the VEGA-3 Phase 3 trial for Phentolamine Ophthalmic Solution 0.75% to treat presbyopia.
- The trial is a randomized, double-masked, placebo-controlled study involving 545 participants.
- Participants will receive one drop of the solution or placebo each evening, with the primary endpoint being a 15-letter improvement in near visual acuity.
- Top-line data from the VEGA-3 trial is expected in the first half of 2025.
Cyclo Therapeutics, Inc.
presents encouraging preliminary safety data from ongoing pivotal phase 3 study and substudy for the treatment of Niemann-Pick disease type C1
- Cyclo Therapeutics presented positive preliminary data from its ongoing pivotal Phase 3 study (TransportNPC?) and substudy evaluating Trappsol? Cyclo? for Niemann-Pick Disease Type C1 (NPC1).
- The TransportNPC? study, the most comprehensive controlled pivotal study for NPC1, completed enrollment in May 2024 with 104 patients.
- The substudy, focusing on newborns to 3-year-olds, completed enrollment with 10 patients and aims to evaluate the safety and efficacy of early intervention.
- Topline data from the 48-week interim analysis is anticipated in H1 2025, with potential marketing applications to follow if data meets statistical significance.
LUCA Science publishes groundbreaking research in Nature Metabolism: mitochondria transfer-based therapies reduce the morbidity and mortality of Leigh syndrome
- LUCA Science Inc., in collaboration with Osaka University and Washington University School of Medicine, published a study in Nature Metabolism.
- The study demonstrates that mitochondria transfer-based therapies can alleviate symptoms and reduce morbidity and mortality in a Leigh Syndrome (LS) mouse model.
- Key findings include significant improvements in LS-affected mice through wild-type bone marrow transplantation and administration of isolated mitochondria.
- LUCA Science is advancing towards GMP development activities to bring Mitochondria oRganelle Complex Q (MRC-Q) to clinical development.
Medincell
’s partner
Teva Pharmaceuticals
provides new update on pivotal clinical phase 3 of investigational olanzapine long-acting injectable (LAI)
- Teva Pharmaceuticals announced that approximately 99% of the targeted injections for submission have been performed.
- Full submission safety results for Olanzapine LAI are expected to be available in H2 2024.
- Olanzapine LAI is a once-monthly subcutaneous injection with a potentially favorable safety profile.
- Medincell may receive up to $117 million in development and commercial milestones, plus royalties on net sales.
Evotec
and
X-Chem, Inc.
collaborate to accelerate early-stage drug discovery
- Evotec SE and X-Chem announced a collaboration to accelerate early-stage drug discovery.
- X-Chem’s DNA-encoded library (DEL) technology will be integrated with Evotec’s screening platforms.
- The collaboration includes access to X-Chem’s DELflex and HITMiner solutions.
- The partnership aims to streamline hit finding and deliver optimal drug intervention starting points.
NImmune Biopharma, Inc
announces R&D collaboration with BioTherapeutics to advance inflammation and immunology precision medicines
- NImmune Biopharma partners with BioTherapeutics to enhance precision immunology capabilities.
- Collaboration includes access to BioTherapeutics' preclinical services, regulatory capabilities, and proprietary animal models.
- NImmune aims to accelerate the development of immunoregulatory therapeutics for inflammatory and autoimmune diseases.
- The partnership complements NImmune's existing R&D collaboration with NIMML Institute.
Vor Bio
announces new clinical data validating approach of using shielded transplants to deliver targeted therapies
- New clinical data from Phase 1/2 VBP101 study of relapsed/refractory AML patients receiving trem-cel followed by Mylotarg.
- Data showed reliable engraftment, shielding from Mylotarg on-target toxicity, broadened therapeutic window, and early evidence of patient benefit.
- 18 patients treated with trem-cel; 10 received Mylotarg as of July 19, 2024.
- Vor Bio plans to discuss a pivotal trial design for trem-cel + Mylotarg with the FDA by year-end.
Aurinia Pharmaceuticals Inc.
announces first participant dosed in AUR200 single ascending dose trial
- Aurinia Pharmaceuticals has dosed the first participant in a Phase 1a single ascending dose (SAD) study of AUR200.
- The study will assess safety, tolerability, pharmacokinetics, and biomarker changes in healthy volunteers.
- Data from the study is expected in the first half of 2025.
- AUR200 targets both BAFF and APRIL, showing potential as a best-in-class therapy for autoimmune diseases.
Crestone Pharma
announces positive data from phase 2 clinical trial of CRS3123 for C. difficile infections
- Crestone reported positive topline results from the Phase 2 trial of CRS3123 in patients with Clostridioides difficile infection (CDI).
- Clinical cure rates at day 12 were 97% for CRS3123 and 93% for vancomycin, with no clinical failures.
- CDI recurrence rates at day 40 were significantly lower for CRS3123 (4%) compared to vancomycin (23%).
- The National Institute of Allergy and Infectious Diseases (NIAID) has provided $4.5 million in new funding for further studies.
