June 7, 2024 - 🧬 [nGram] Today’s Oncology Scoop: FDA Approves Geron's RYTELO™, AbbVie's Positive Phase 2 Results, and More

  1. Geron announces FDA approval of Rytelo for treatment of lower-risk MDS with transfusion-dependent anemia
    • Geron Corporation announced FDA approval of Rytelo (imetelstat) for adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia.
    • Rytelo is the first telomerase inhibitor approved for this indication, offering potential for over 24 weeks of freedom from red blood cell transfusions.
    • Approval is based on results from the IMerge Phase 3 clinical trial, which showed significantly higher rates of transfusion independence compared to placebo.
    • The trial demonstrated durable and sustained transfusion independence, with median durations of approximately 1 year for 8-week responders and 1.5 years for 24-week responders.
    • Common adverse reactions included neutropenia and thrombocytopenia, which were generally manageable with dose modifications.
    • Geron aims to ensure broad access to Rytelo through its REACH4RYTELO Patient Support Program.
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  2. EASL wrap-up session highlighted late-breaking clinical data of first-in-class HBV-specific TCR T cell therapy (SCG101) showing improved overall survival in HBV-related hepatocellular carcinoma
    • SCG Cell Therapy presented clinical data on SCG101 at the EASL Congress 2024 in Milan, Italy.
    • SCG101 showed promising antiviral and antitumor activities in patients with advanced HBV-related hepatocellular carcinoma (HCC).
    • In a first-in-human trial, SCG101 achieved a 33% objective response rate (ORR) with partial and complete remissions.
    • 67% of patients experienced significant serum HBsAg reduction, correlating with tumor reduction and prolonged survival.
    • SCG101 was well tolerated with no serious adverse events; common side effects included transient liver enzyme elevation, cytokine release syndrome, and fever.
    • SCG101 is currently being evaluated in a Phase 1/2 clinical trial (NCT05417932) with approvals from FDA, NMPA, HSA, and DOH.
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  3. AbbVie announces positive topline results from phase 2 PICCOLO trial evaluating mirvetuximab soravtansine for high folate receptor-alpha expressing platinum-sensitive ovarian cancer
    • PICCOLO trial met its primary endpoint with an objective response rate (ORR) of 51.9%.
    • The median duration of response (DOR) was 8.25 months.
    • The safety profile was consistent with previous studies, with no new safety concerns identified.
    • PICCOLO is a single-arm Phase 2 trial for patients with FR-alpha high platinum-sensitive ovarian cancer who have received at least two prior lines of platinum-containing therapy.
    • Full data from the PICCOLO study will be presented at a future medical meeting.
    • Mirvetuximab soravtansine is also being studied in the Phase 3 GLORIOSA trial in combination with bevacizumab.
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  4. CG Oncology announces Nature Medicine publication of final results from CORE-001 study of cretostimogene grenadenorepvec in combination with pembrolizumab in BCG-unresponsive NMIBC
    • Final positive CORE-001 safety and efficacy findings published in Nature Medicine and featured at ASCO 2024.
    • 54% complete response (CR) rate in the intention-to-treat population at 24 months, meeting the primary endpoint of the phase 2 study.
    • CR rate at 12 months was 57%, with 95% of patients maintaining CR for another 12 months.
    • Progression-free survival (PFS) at 24 months is 100%, with no progression to muscle invasive cancer or metastatic disease.
    • Cystectomy-free survival (CFS) at 24 months was 80%; for patients in CR, CFS at 24 months was 100%.
    • The combination of cretostimogene and pembrolizumab received FDA Breakthrough Therapy Designation in May 2023.
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  5. Qu Biologics and Foundation Medicine collaborate to impact the care of patients with late-stage colorectal cancer
    • Qu Biologics and Foundation Medicine announce a partnership to improve care for late-stage colorectal cancer patients.
    • The collaboration focuses on a Phase II multicenter trial (PERIOP-06) assessing QBECO SSI's effectiveness in reducing post-operative immune suppression.
    • Foundation Medicine will provide genomic and transcriptomic profiling to predict patient response and assess minimal residual disease.
    • The trial aims to correlate genomic profiling results with changes in immune function and patient survival outcomes.
    • The study is led by GI Oncology surgeons Dr. Rebecca Auer and Dr. Paul Karanicolas and is currently enrolling patients in Ontario.
    • The partnership may extend to future studies to monitor disease progression and expedite QBECO SSI's approval and accessibility.
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  6. Scientific journal reports findings that show LIXTE’s lead clinical compound, LB-100, increases recognition of colon cancer cells by the immune system
    • LIXTE Biotechnology's LB-100 can turn immunologically 'cold' tumors 'hot,' enhancing immunotherapy benefits.
    • The study, published in EMBO Reports, shows LB-100 disrupts mRNA splicing, generating neo-antigens presented to the immune system.
    • This mechanism supports ongoing clinical trials in lung and ovarian cancer combining LB-100 with checkpoint immunotherapy.
    • The research indicates potential for expanding the number of colon cancer patients who respond to immunotherapy.
    • The study provides direct evidence that LB-100 generates neo-antigens presented in the context of MHC class I antigens.
    • Proof-of-concept clinical trials are in progress for colon, small cell lung, and sarcoma cancers.
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  7. HUTCHMED initiates phase I trial of menin inhibitor HMPL-506 in patients with hematological malignancies in China
    • HUTCHMED has started a Phase I clinical trial of HMPL-506, a menin inhibitor, in China.
    • The trial is a multicenter, open-label study focusing on safety, pharmacokinetics, and efficacy.
    • The study includes a dose escalation phase and a dose expansion phase, aiming to enroll at least 60 patients.
    • The first patient received their dose on May 31, 2024.
    • HMPL-506 targets the menin protein, which is involved in gene expression and cell signaling, particularly in AML.
    • HUTCHMED retains all global rights to HMPL-506.
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  8. Syndax advances revumenib trial to phase 1b for metastatic MSS CRC
    • Syndax Pharmaceuticals is advancing to the Phase 1b portion of its trial for revumenib in relapsed or refractory metastatic MSS colorectal cancer (CRC).
    • The decision follows a favorable safety profile observed in the Phase 1a portion, as recommended by the Independent Data Monitoring Committee (IDMC).
    • The Phase 1/2 trial aims to assess the safety, tolerability, and anti-tumor activity of revumenib.
    • In Phase 1a, 19 patients were enrolled across three dose cohorts, with no Grade 3 or greater treatment-related adverse events observed.
    • Initial efficacy results showed a 33% stable disease rate at 16 weeks, supporting the advancement to Phase 1b.
    • The selected dose for Phase 1b is 276 mg three times a day (TID).
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  9. MAIA Biotechnology announces year-to-date achievements and highlights recent clinical progress for novel anticancer agent
    • MAIA Biotechnology reported significant progress for its lead candidate, THIO, a telomere-targeting agent for non-small cell lung cancer (NSCLC).
    • The Phase 2 THIO-101 trial evaluates THIO sequenced with cemiplimab in patients with advanced NSCLC who failed two or more standard therapies.
    • Key trial results include an overall response rate (ORR) of 38%, disease control rate (DCR) of 85%, and median progression-free survival (PFS) of 5.5 months.
    • THIO demonstrated a favorable safety profile, with 180mg per cycle showing the greatest efficacy.
    • MAIA's cash position improved significantly, with $12.4 million raised since February 2024 to fund ongoing clinical development.
    • The Phase 2 THIO-101 trial is expected to near completion in 2024, with ongoing research for second-generation THIO-like compounds.
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  10. Kiromic BioPharma reports six-month results from first patient enrolled in Deltacel-01 clinical trial
    • Kiromic BioPharma reports favorable six-month follow-up results from the first patient in the Deltacel-01 Phase 1 clinical trial.
    • The trial evaluates Deltacel (KB-GDT-01), an allogeneic, off-the-shelf Gamma Delta T-cell therapy, in patients with stage 4 metastatic non-small cell lung cancer (NSCLC).
    • Scans showed a 13% decrease in tumor size and no new tumor lesions, translating to six-month progression-free survival.
    • No dose-limiting toxicities were reported, reinforcing the safety profile of Deltacel.
    • The fifth patient began treatment in May and is expected to have their first efficacy assessment in July, while the sixth patient is undergoing pre-enrollment screening.
    • Long-term follow-up results for other subjects in the first and second cohorts are expected by the end of June.
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