Geron announces FDA approval of Rytelo for treatment of lower-risk MDS with transfusion-dependent anemia
- The FDA has approved 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 red blood cell 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 include thrombocytopenia, neutropenia, and infusion-related reactions, which are generally manageable with dose modifications.
- Geron aims to ensure broad access to Rytelo through its REACH4RYTELO Patient Support Program.
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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 observed.
- 67% of patients experienced significant serum HBsAg reduction, correlating with tumor reduction and prolonged progression-free survival (PFS) and overall survival (OS).
- SCG101 was well tolerated with no serious adverse events; common side effects included transient liver enzyme elevation, cytokine release syndrome (CRS), and fever.
- SCG101 is currently being evaluated in an ongoing Phase 1/2 clinical trial (NCT05417932).
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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%.
- Median duration of response (DOR) was 8.25 months, a key secondary endpoint.
- Safety profile consistent with previous studies; no new safety concerns identified.
- PICCOLO is a single-arm Phase 2 trial for patients with FR-alpha high platinum-sensitive ovarian cancer.
- Full data to be presented at a future medical meeting.
- Mirvetuximab soravtansine is also being studied in the Phase 3 GLORIOSA trial.
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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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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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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.
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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 for its menin inhibitor HMPL-506 in China.
- The first patient received their dose on May 31, 2024.
- The trial is a multicenter, open-label study to assess safety, pharmacokinetics, and efficacy.
- The study includes a dose escalation phase and a dose expansion phase, with at least 60 patients expected to enroll.
- HMPL-506 is a selective small molecule inhibitor targeting the menin protein, relevant in MLL-rearranged and NPM1-mutant AML.
- No menin inhibitors are currently approved worldwide; HUTCHMED retains all rights to HMPL-506 globally.
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Syndax advances revumenib trial to phase 1b for metastatic MSS CRC
- Syndax Pharmaceuticals is advancing to the Phase 1b portion of its Phase 1/2 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 reviewed by the Independent Data Monitoring Committee (IDMC).
- The Phase 1a trial enrolled 19 patients across three dose cohorts (163 mg, 226 mg, and 276 mg TID) and showed no Grade 3 or greater treatment-related adverse events.
- Initial efficacy results indicated a 33% stable disease rate at 16 weeks, supporting the advancement to Phase 1b.
- The selected dose for Phase 1b is 276 mg TID, based on achieving full target saturation.
- Next steps include further exploration of revumenib's role in treating R/R metastatic MSS CRC.
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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.
- MAIA secured a high-value clinical supply agreement for cemiplimab used in the THIO-101 trial.
- The company raised approximately $12.4 million 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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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 doing well; 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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