June 5, 2024 - 🧬 [nGram] Today’s Oncology Scoop: Pierre Fabre's IND Filing, Cellectis' Orphan Drug, ASCO 2024 Highlights

  1. Pierre Fabre Laboratories announce IND filing for PFL-002/VERT-002, a potential treatment for patients with solid tumors, including non-small cell lung cancer with MET alterations
    • Pierre Fabre Laboratories filed an IND application with the FDA for PFL-002/VERT-002.
    • The Phase I/II trial will assess safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy.
    • The trial targets NSCLC patients with MET alterations, including those with resistance to other treatments.
    • PFL-002/VERT-002 is a monoclonal antibody developed by Vertical Bio, acting as a degrader of c-MET.
    • The first patient enrollment is expected by the end of 2024.
    • Pierre Fabre Laboratories is expanding its precision oncology portfolio with several new assets.
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  2. Cellectis receives orphan drug designation for UCART22 for acute lymphoblastic leukemia
    • Cellectis announced that the European Commission (EC) granted Orphan Drug Designation (ODD) to UCART22 for treating Acute Lymphoblastic Leukemia (ALL).
    • UCART22 is an allogeneic CAR T-cell product targeting CD22, evaluated in the BALLI-01 Phase 1/2 study.
    • ALL accounts for 12% of all leukemia cases and progresses rapidly, often fatal within weeks or months if untreated.
    • Preliminary clinical data showed a 67% response rate at Dose Level 2 for UCART22-P2, compared to 50% at Dose Level 3 for UCART22-P1.
    • The ODD provides regulatory, financial, and commercial incentives for developing treatments for rare diseases with no satisfactory options.
    • Cellectis plans to update on the progress of BALLI-01 by the end of 2024.
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  3. FDA approved phase II/III clinical study of KPG-121 in combination with abiraterone as a first line treatment for mCRPC
    • The FDA has approved a Phase II/III clinical trial of KPG-121 in combination with abiraterone for first-line treatment of metastatic castration-resistant prostate cancer (mCRPC).
    • KPG-121, discovered by Kangpu Biopharmaceuticals, targets the Cereblon (CRBN) E3 ubiquitin ligase complex, promoting anti-proliferation and anti-angiogenesis activities.
    • In xenograft models, KPG-121 significantly improves anti-tumor efficacy when combined with androgen-receptor antagonists compared to androgen-receptor antagonist therapy alone.
    • A Phase I study in the US (NCT03569280) demonstrated that KPG-121 was well tolerated and showed a favorable pharmacokinetic profile and promising efficacy.
    • Kangpu Biopharmaceuticals focuses on developing innovative small molecules for treating solid tumors, hematologic malignancies, autoimmune diseases, and inflammatory disorders.
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  4. Cyclacel’s fadraciclib demonstrates efficacy in patient-derived colorectal cancer models at the 2024 ASCO annual meeting
    • Fadraciclib, a novel CDK2/9 inhibitor, shows potential as a treatment for metastatic colorectal cancer (CRC).
    • Preclinical data presented at the ASCO Annual Meeting demonstrated significant tumor growth inhibition in CRC patient-derived organoids (PDOs) and xenografts (PDX).
    • Fadraciclib induced anaphase catastrophe, a novel cancer-specific mechanism, and triggered apoptosis in CRC models.
    • The study involved 18 CRC PDOs treated with standard chemotherapy, palbociclib, or fadraciclib, with fadraciclib showing superior efficacy.
    • Fadraciclib's ability to inhibit CDK2/9, downregulate MYC protein levels, and induce apoptosis was validated through various assays.
    • Cyclacel is currently enrolling patients in the proof of concept part of the 065-101 study, with initial data expected in the second half of 2024.
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  5. Live from ASCO 2024: oral report released latest data of olverembatinib in SDH-deficient GIST, including a CBR of 92.3%
    • Ascentage Pharma presented updated clinical data of olverembatinib at the 60th ASCO Annual Meeting.
    • The Phase I study enrolled 26 patients with SDH-deficient GIST, showing a clinical benefit rate (CBR) of 92.3%.
    • 6 patients achieved partial responses (PR) and 18 achieved stable diseases (SD) lasting longer than four treatment cycles.
    • The median progression-free survival (PFS) was 25.7 months.
    • Olverembatinib has been granted Breakthrough Therapy Designation by China's NMPA for SDH-deficient GIST.
    • The drug is also being evaluated for other indications, including hematologic malignancies.
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  6. CARsgen presents updated results on satri-cel in Nature Medicine and at 2024 ASCO
    • CARsgen Therapeutics announced final follow-up results of the CT041-CG4006 trial of satri-cel, published in Nature Medicine on June 3, 2024.
    • Data were also presented at the 2024 ASCO Annual Meeting on June 3, 2024.
    • Satri-cel is an autologous CAR T-cell product candidate targeting Claudin18.2, aimed at treating gastrointestinal cancers.
    • The study showed promising efficacy and manageable safety profiles in patients with Claudin18.2-positive advanced gastrointestinal cancers.
    • Ongoing trials include Phase II and Phase I trials in China and North America for gastric and pancreatic cancers.
    • Satri-cel has received multiple designations from the U.S. FDA and EMA, including RMAT, PRIME eligibility, and Orphan Drug designations.
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  7. MediciNova receives notice of allowance for new patent covering MN-166 (ibudilast) for the prevention of metastasis of various solid cancers
    • MediciNova has received a Notice of Allowance from the U.S. Patent and Trademark Office for a patent covering MN-166 (ibudilast).
    • The patent covers the prevention of metastasis in various cancers, including pancreatic, lung, breast, colorectal, melanoma, and ovarian cancers.
    • The allowed claims include the use of MN-166 in combination with therapies such as chemotherapy, immunotherapy, radiotherapy, and others.
    • The patent covers oral administration, a wide range of doses, different dosing frequencies, and treatment periods.
    • Once issued, the patent is expected to expire no earlier than July 2042.
    • MN-166 (ibudilast) is in late-stage clinical development for neurodegenerative diseases and is also being evaluated for glioblastoma, Long COVID, and other conditions.
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  8. Quantro Therapeutics reaches a milestone in the collaboration with Boehringer Ingelheim to develop first-in-class cancer treatments
    • QUANTRO Therapeutics achieved a key milestone in its R&D collaboration with Boehringer Ingelheim.
    • The milestone involved successful application of QUANTRO’s QUANTROseq Transcriptional Fingerprint technology.
    • All pre-set goals for technical proof-of-concept, cross-validation of HTS screening technology, and identification of high-quality hits were accomplished.
    • The joint R&D program, initiated in 2022, aims to develop first-in-class cancer treatments targeting previously undruggable transcription factors.
    • QUANTRO will receive an undisclosed milestone payment, with the total potential transaction value exceeding EUR 500 million.
    • Next steps include advancing the identified hits to the discovery and development stages.
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  9. Cue Biopharma presents updated data from phase 1 trial of CUE-101 in recurrent/metastatic HPV+ head and neck cancer at the 2024 ASCO annual meeting
    • Overall response rate (ORR) of 46% and 12-month overall survival (OS) of 96% in first line (1L) recurrent/metastatic (R/M) HPV+ head and neck squamous cell carcinoma (HNSCC) treated with CUE-101 and pembrolizumab.
    • Median overall survival (mOS) of 20.8 months in second line (2L) and beyond HPV+ HNSCC patients treated with CUE-101 monotherapy, compared to historical mOS of 7.5 and 8.4 months in third-party checkpoint inhibitor trials.
    • ORR of 46% and Disease Control Rate (DCR) of 79% in patients with combined positive score (CPS) ≥1, compared to an ORR of 19% with pembrolizumab alone in the historical KEYNOTE-048 trial.
    • 12-month OS of 96% and median progression free survival (PFS) of 5.8 months compared to 3.2 months with pembrolizumab alone in the KEYNOTE-048 trial.
    • No unanticipated, significant safety concerns have emerged in either the combination or monotherapy trials, and adverse events have been readily managed.
    • CUE-101 data will be presented in an oral presentation at the 2024 ASCO Annual Meeting.
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  10. Ascentage Pharma releases updated data on lisaftoclax at ASCO 2024
    • Ascentage Pharma presented updated results from a global, multicenter Phase Ib/II study of lisaftoclax at the ASCO 2024 Annual Meeting.
    • The study evaluated lisaftoclax alone or in combination with ibrutinib or rituximab for treating Waldenström macroglobulinemia (WM).
    • Lisaftoclax combined with ibrutinib showed a 90.9% objective response rate (ORR) in treatment-naïve WM patients, with manageable adverse events and no drug-drug interactions.
    • The study enrolled 46 patients across three arms, with varying doses of lisaftoclax administered in 28-day cycles.
    • Efficacy results showed ORRs of 41.7%, 90.9%, and 37.5% for Arms A, B, and C, respectively, with no significant differences based on CXCR4 mutation status in Arms B and C.
    • Safety results indicated that lisaftoclax was well tolerated, with no maximum-tolerated dose reached and manageable adverse events, including neutropenia and tumor lysis syndrome.
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