On November 18, 2024 OncoSpherix, Inc., a preclinical-stage cancer drug development company advancing HIF inhibitors for a variety of cancer indications, reported participation in the 2024 Georgia Bio Life Sciences Summit last month from October 21 to October 22, 2024, where the company was featured in the prestigious Company Showcase (Press release, OncoSpherix, NOV 18, 2024, View Source [SID1234648473]). This event provided a valuable platform for OncoSpherix to present its pioneering developments in cancer therapies and underscore its commitment to advancing cancer treatment.

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The Georgia Bio Life Sciences Summit, known for bringing together key players in the biotechnology and life sciences sectors, offered OncoSpherix an opportunity to engage with industry leaders, investors, and researchers. The Company Showcase spotlighted OncoSpherix’s unique approach to precision oncology, focusing on therapies designed to block activation of HIF and combat hard-to-treat cancers more effectively.

"We are honored to have been part of the Company Showcase at the 2024 Georgia Bio Life Sciences Summit," said OncoSpherix CEO, Margaret Offermann, MD, PhD. "This platform allowed us to share our breakthroughs in developing new drugs for oncology and our mission to bring innovative, effective therapies to patients in need. The positive response from the community reinforces our commitment to developing solutions that will make a significant impact on cancer treatment."

On November 18, 2024 Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, reported the opening of enrollment for the Phase 1 clinical trial evaluating ADI-270 in patients with metastatic/advanced clear cell renal cell carcinoma (ccRCC) (Press release, Adicet Bio, NOV 18, 2024, View Source [SID1234648488]).

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"Solid tumors represent one of the highest unmet medical needs in oncology and have yet to benefit from the breakthroughs observed with CAR T cell therapies in hematologic malignancies. Emerging data from ADI-270, our armored allogeneic ‘off the shelf’ gamma delta 1 CAR T cell therapy targeting CD70 positive cancers, have shown potential in addressing this gap," said Chen Schor, President and Chief Executive Officer. "At the recent ASGCT (Free ASGCT Whitepaper) conference, we presented preclinical data in which ADI-270 demonstrated significant tumor infiltration, resistance to the immunosuppressive tumor microenvironment, and potent activity via CAR and innate-mediated targeting, highlighting its potential for treating solid tumors. We look forward to enrolling patients and anticipate sharing preliminary clinical data from the trial in the first half of 2025."

About the Phase 1 Trial

The Phase 1 multicenter, open-label clinical trial is designed to investigate ADI-270 as monotherapy in adults with relapsed or refractory ccRCC. Following lymphodepletion, patients will be eligible to receive a single dose of ADI-270 with a starting dose level of 3E8 CAR+ cells. Subject to meeting protocol defined criteria, patients enrolled in the trial may be eligible to receive a second dose of ADI-270. The dose escalation and dose expansion portions of the trial will evaluate safety, tolerability, and pharmacokinetics as well as anti-tumor activity as assessed by overall response rate, duration of response and disease control rate.

About ADI-270

ADI-270 is an armored allogeneic "off-the-shelf" gamma delta CAR T cell therapy candidate targeting CD70-positive cancers. CD70 is a compelling target due to its high expression in both solid and hematological malignancies. ADI-270 is engineered with a third-generation CAR designed to target CD70 using its natural receptor, CD27, as the binding moiety and is further armored with a dominant negative form of the transforming growth factor-β receptor II (dnTGFβRII) to provide functional resilience to the immunosuppressive tumor microenvironment. ADI-270 is also designed to increase exposure and persistence by reducing susceptibility to host vs. graft elimination. These properties of ADI-270 combined with the potent tumor infiltration demonstrated with gamma delta 1 T cells aim to improve clinical responses of RCC patients and other patients with CD70+ tumors.

About Renal Cell Carcinoma

Renal cell carcinoma (RCC) is the most common tumor of the kidney, constituting 80% to 85% of primary renal neoplasms. Clear cell RCCs (ccRCC) are the most common subtype, accounting for 80% of all RCCs. ccRCC is an aggressive subtype arising from renal stem cells commonly arising in the proximal nephron and tubular epithelium, and often metastasizes to the lungs, liver, and bones. Approximately 20% of newly diagnosed cases of RCC are locally advanced or metastatic and up to 30% of patients will develop metastatic disease following nephrectomy. While the 5-year survival rate for localized RCC is 93%, the 5-year survival rate for advanced disease is 15%.

On November 18, 2024 Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the Marketing Authorization Application for KIZFIZO (temozolomide oral suspension) for the treatment of patients with relapsed or refractory high-risk neuroblastoma, because the CHMP considered that a positive benefit-risk balance has not been established at this stage (Press release, ORPHELIA Pharma, NOV 18, 2024, View Source [SID1234648474]). Orphelia will seek a re-examination of the opinion by the CHMP.

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"We are extremely disappointed by the CHMP’s negative opinion and understand that this may also be disappointing for the pediatric oncology community as well as for parents of children suffering from cancer." said Jérémy Bastid, Chief Medical Officer at Orphelia Pharma. "There is a significant demand for developing children-adapted formulations. Orphelia Pharma has been developing KIZFIZO in collaboration with Gustave Roussy, the leading European cancer center, for the last seven years specifically to address this unmet medical need for a drinkable temozolomide medication in the treatment of relapsed or refractory neuroblastoma, which affects very young children".

"As part of the re-examination process, we will seek to address the CHMP’s grounds for refusal and we will work diligently to substantiate the clinical benefit of the treatment so that KIZFIZO can be available to pediatric patients in the EU without further delay" commented Laurent Martin, Chief Pharmaceutical Affairs Officer at Orphelia Pharma.

In the meantime, Orphelia Pharma will, subject to agreement by the relevant authorities, continue to make KIZFIZO available to pediatric patients suffering from relapsed or refractory neuroblastoma under compassionate use or early access programs as well as to patients enrolled into ongoing clinical trials.

About KIZFIZO 40 mg/ml

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed/designed specifically for use in the treatment of children with relapsed or refractory high-risk neuroblastoma, which carries a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma.

In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed neuroblastoma as monotherapy or in combination with irinotecan or topotecan.

KIZFIZO has received Orphan Drug Designation (ODD) from the EMA and the FDA, the formulation is covered by granted patents and pending applications in Europe and the US.

The pharmacokinetics of KIZFIZO in children have been evaluated in TEMOkids, a European multicenter population pharmacokinetic acceptability and safety study in pediatric patients in need of temozolomide (NCT04610736).

Efficacy and safety data for temozolomide in relapsed or refractory neuroblastoma submitted in the application includes in particular:

BEACON-Chemo, a sub-analysis of the chemotherapy arms of the BEACON study, a prospective randomized phase II study in refractory or relapsed neuroblastoma. This study was sponsored by Birmingham University (UK).
Retro-TMZ, a multicenter descriptive, retrospective study, assessing the efficacy and tolerability of temozolomide in children with refractory or relapsed neuroblastoma. This study was conducted by Gustave Roussy (France).
About Neuroblastoma

Neuroblastoma is the most common extracranial cancer in early childhood, with approximately 900 new cases diagnosed per year in the European Union. It almost exclusively affects children under five, with a median age at diagnosis of 18 months. Neuroblastoma has a wide diversity of clinical outcomes, which is reflected in the risk stratification. Approximately 40% of patients have the high-risk disease and often face a poor response to first line induction therapy or later relapse. There remains a high unmet need for relapsed or refractory neuroblastoma patients and the best therapeutic strategy is still an intensive area of research. Temozolomide is the standard chemotherapy and is therefore an essential part of the treatment armamentarium for these patients.

On November 18, 2024 Foresight Diagnostics, a leader in ultrasensitive minimal residual disease (MRD) testing, reported the presentation of multiple studies showcasing Foresight CLARITY MRD at the upcoming 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place December 7-10 in San Diego, California (Press release, Foresight Diagnostics, NOV 18, 2024, View Source [SID1234648489]).

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The presentations span various lymphoma types and treatment settings, demonstrating the broad applicability of the Foresight CLARITY MRD detection technology for monitoring treatment response and predicting patient outcomes. The studies include collaborations with multiple pharmaceutical companies and research institutions, including AstraZeneca, Bristol Myers Squibb (BMS), Daiichi Sankyo (DSI), Stanford University, Washington University, and others.

"These studies add to the growing body of evidence supporting ultra-sensitive MRD testing as a critical tool in lymphoma care and drug development," said David Kurtz, MD, PhD, Chief Medical Officer and Head of Research at Foresight Diagnostics. "We see in these studies that MRD measurement with Foresight CLARITY consistently enables earlier and more accurate treatment response assessment than conventional imaging-based methods. The ability to monitor disease status in real-time with ultra-sensitive MRD assays like Foresight CLARITY may help accelerate clinical trials and lead to more personalized decision-making for patient care."

Results utilizing Foresight CLARITY MRD technology are featured in six abstracts, including three that are co-authored by Foresight.

Presentation highlights include:

LBCL/DLBCL:

ctDNA-MRD as a surrogate endpoint for 2L CAR T – Analysis of the Phase 3 TRANSFORM trial in second-line treatment for large B-cell lymphoma (LBCL) reinforced the potential of circulating tumor DNA (ctDNA) as an earlier endpoint for response assessment and assessment of clinical trial results. In the TRANSFORM study, patients receiving lisocabtagene maraleucel (liso-cel) had significantly improved event-free survival compared to standard of care. Consistent with this result, patients receiving liso-cel also demonstrated higher rates of MRD clearance, demonstrating CLARITY’s ability to measure differential treatment responses.
MRD Response with novel first-line therapy in high-risk DLBCL – Analysis of a Phase 1b trial CC-220-DLBCL-001 combining golcadomide with R-CHOP in previously untreated aggressive B-cell lymphoma showed promising MRD clearance rates. The data demonstrated that 90% of patients achieved MRD negativity at end of treatment with golcadomide 0.4mg + R-CHOP, with strong responses observed even in high-risk patients. These data further support the ongoing Phase 3 GOLSEEK-1 study investigating this novel combination therapy in high-risk 1L LBCL.
Enhanced risk stratification by MRD vs. traditional methods – New research investigates the predictive value of the International Prognostic Index (IPI), currently the primary pre-treatment risk assessment tool for DLBCL. The study revealed that pre-treatment IPI scores did not significantly correlate with end-of-treatment MRD status, with MRD testing being a superior predictor of progression-free survival.
Follicular Lymphoma:

MRD-based response assessment of novel FL therapies – Two studies demonstrated the value of MRD assessment in follicular lymphoma trials, with new treatment approaches showing early clearance. The CD19xCD3 bispecific antibody AZD0486 achieved 89% MRD negativity in relapsed or refractory FL patients by 12 weeks in complete responders. In the 1L setting, the chemotherapy-free combination of mosunetuzumab and polatuzumab vedotin showed similar early MRD clearance, with 7 of 8 complete responders achieving MRD negativity before cycle 3. These results highlight the potential for MRD as an early indicator of therapeutic response in FL clinical trials.
PTCL:

ctDNA as a prognostic biomarker for T-cell lymphoma – Analysis from the VALENTINE-PTCL01 Phase 2 trial in relapsed/refractory peripheral T-cell lymphoma (PTCL) revealed that patients achieving early, deep ctDNA reduction (>5-fold decrease by Cycle 2 Day 1) experienced significantly longer progression-free survival, establishing ctDNA monitoring as a powerful tool for early response assessment.
Information on abstract presentation time and dates can be found below. To meet with Foresight Diagnostics, visit booth #3423 or contact us at [email protected].

Oral Presentations:

Circulating Tumor DNA (ctDNA) as an Early Outcome Predictor in Patients with Second-Line Large B-Cell Lymphoma After Lisocabtagene Maraleucel Versus Standard of Care Treatment from the Phase 3, Randomized TRANSFORM Study

Presenter: Ash Alizadeh, MD, PhD (Stanford University)
Session 628
Date/Time: Saturday, December 7, 2024, 10:45 a.m.
Sponsor: Bristol Myers Squibb
Co-authored by Foresight Diagnostics
Escalating Doses of AZD0486, a Novel CD19xCD3 T-Cell Engager, Result in High Complete Remissions with Rapid Clearance of Minimal Residual Disease in Patients with Relapsed/Refractory Follicular Lymphoma

Presenter: Jing-Zhou Hou, MD, PhD (UPMC Hillman Cancer Center)
Session 623
Date/Time: Saturday, December 7, 2024, 5 p.m.
Sponsor: AstraZeneca
Golcadomide (GOLCA) Plus R-CHOP Has High Minimal Residual Disease (MRD) Negativity across High-Risk, Untreated Aggressive B-Cell Lymphoma (a-BCL)

Presenter: Jason R. Westin, MD (MD Anderson)
Session 627
Date/Time: Sunday, December 8, 2024, 12:30 p.m.
Sponsor: Bristol Myers Squibb
International prognostic index poorly predicts EOT ctDNA MRD status in DLBCL and has limited impact on its predictive value for outcomes

Presenter: Jordan S. Goldstein, MD, MS (Stanford University)
Session 626
Date/Time: Sunday, December 8, 2024, 5 p.m.
Co-authored by Foresight Diagnostics
Poster Presentations:

Prediction of Clinical Response by Phased Variants in Circulating Tumor DNA (ctDNA) in the VALENTINE-PTCL01 trial of Patients with Relapsed or Refractory Peripheral T-Cell Lymphoma

Presenter: Neha Mehta-Shah, MD, MSCI (Washington University)
Abstract #4342, Session 621
Date/Time: Monday, December 9, 2024, 6-8 p.m.
Sponsor: Daiichi Sankyo
Co-authored by Foresight Diagnostics
Mosunetuzumab Plus Polatuzumab Vedotin Induces Early Complete Responses in Previously Untreated High Tumor Burden Follicular Lymphoma

Presenter: David A Russler-Germain, MD, PhD (Washington University)
Abstract #4414, Session 623
Date/Time: Monday, December 9, 2024, 6-8 p.m.

On November 18, 2024 Ratio Therapeutics Inc. (Ratio), a pharmaceutical company employing innovative technologies to develop best-in-class radiopharmaceuticals for cancer treatment and monitoring, reported to have entered into an exclusive worldwide license and collaboration agreement with Novartis Pharma AG, a subsidiary of Novartis AG (NYSE: NVS) (Press release, Ratio Therapeutics, NOV 18, 2024, View Source [SID1234648475]). The collaboration leverages Ratio’s radioligand therapy discovery and development expertise as well as its technology platforms for the development of a Somatostatin Receptor 2 (SSTR2) radiotherapeutic candidate for cancer.

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"The team at Ratio is honored and excited to partner with Novartis on the development of a next-generation SSTR2-targeting therapeutic," said Jack Hoppin, Ph.D., Chief Executive Officer of Ratio. "Together, we aim to develop a best-in-class therapy in the fight against SSTR2-expressing tumors."

"Radioligand therapies hold transformative potential for certain forms of cancer, and Novartis is committed to maximizing their impact by continually improving the benefit for patients," said Fiona Marshall, President of Biomedical Research at Novartis. "We are delighted to collaborate with Ratio to advance this RLT candidate and work together to bring forward additional therapeutic options for patients with difficult-to-treat cancer."

Under the terms of the agreement, Ratio will receive combined upfront and potential milestone payments up to $745m, and is eligible to receive tiered royalty payments. Ratio will collaborate with Novartis to drive preclinical activities to research and select an SSTR2-targeting development candidate. Novartis will assume responsibility for all remaining development, manufacturing, and commercialization activities.

The collaboration combines the expertise and strengths of Ratio and Novartis to further elevate the safety and efficacy of radiopharmaceuticals for patient benefit.

Chestnut Partners served as exclusive financial advisor to Ratio for this transaction.