On August 19, 2024 OS Therapies (NYSE American: OSTX) ("OS Therapies" or "the Company"), an ADC and Immunotherapy research and clinical-stage biopharmaceutical company, reported that it has been accepted into Johnson & Johnson Innovation – JLABS (Press release, OS Therapies, AUG 19, 2024, View Source;JLABS [SID1234645998]). Through membership of JLABS, OS Therapies aims to further develop and enter the clinic with its tunable Antibody Drug Conjugate (ADC) linker-based platform that relies on pH sensitive linkers & coating technology to reduce off-target effects to improve safety and increase the number & diversity of therapeutic payloads delivered. Through these developments, OS Therapies hopes to offer improved efficacy versus other ADCs in the market or in development. Drug candidates developed using OS Therapies’ tunable ADC platform leverage this unique linker platform to create new intellectual property for both novel and off-patent targeting antibodies and drug payloads.

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On August 19, 2024 MOMA Therapeutics, a clinical-stage biopharmaceutical company discovering and developing a new generation of precision therapeutics, reported that the first patient has been dosed in its Phase 1 clinical trial to assess the safety and tolerability of MOMA-313, a novel, highly potent and selective oral polymerase theta helicase inhibitor (Press release, MOMA Therapeutics, AUG 19, 2024, View Source [SID1234645997]).

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MOMA-313 is initially being developed in combination with a PARP inhibitor for patients with solid tumors harboring alterations in certain DNA repair genes, including subgroups of prostate cancer, pancreatic cancer and breast cancer.

In parallel, MOMA announced the selection of a development candidate for its second lead program, MOMA-341, an oral potent and selective covalent Werner helicase inhibitor for treatment of cancers with microsatellite instability. An IND application filing with the FDA is anticipated in the first quarter of 2025.

"We are excited to be progressing two molecules with best-in-class potential toward the benefit of cancer patients in need in such close proximity," said Asit Parikh, M.D., Ph.D., chief executive officer of MOMA. "The advance into clinical development for a company founded as an idea on paper a few years back is humbling. This progress serves as a testament to the excellence and unwavering commitment of the entire MOMA team."

"Advancing two highly potent and selective drug candidates derived from our proprietary KNOMATIC platform toward the clinic is a unique privilege," added Peter Hammerman, M.D., Ph.D., chief scientific officer of MOMA. "The goal with each candidate is to translate groundbreaking science into life-altering medicine."

About MOMA-313 and the Phase 1 Trial

MOMA-313 is a potent and selective oral polymerase theta (Polθ) helicase inhibitor. Polθ is involved in the repair of DNA double-strand breaks that arise during DNA replication. The Phase 1 trial (NCT06545942) is a multi-center, open-label study designed to evaluate the safety and tolerability of MOMA-313 as monotherapy and in combination with the PARP inhibitor olaparib for patients where a PARP inhibitor would be expected to provide benefit. Response to PARP inhibitors can be short-lived due to primary and acquired resistance. Pre-clinical studies suggest that combining a PARP inhibitor with a Polθ inhibitor could afford unique mechanistic potential to deepen and prolong that response.

About MOMA-341

MOMA is developing MOMA-341 as an oral, potent and selective covalent inhibitor of Werner helicase with a novel chemical scaffold as monotherapy and in combination with chemotherapy and immunotherapy in tumors with microsatellite instability (e.g., colorectal, gastric, endometrial cancers).

About the KNOMATIC platform

The KNOMATIC platform integrates deep structural insights, advanced hit-finding technologies, and computation-enabled lead optimization to accelerate discovery of novel therapeutics targeting families of highly dynamic proteins, such as ATPases and GTPases. MOMA-313 and MOMA-341 were discovered and developed through the application of the KNOMATIC platform.

On August 19, 2024 Scorpion Therapeutics, Inc. ("Scorpion"), a pioneering clinical-stage oncology company dedicated to transforming the lives of cancer patients by redefining the frontier of precision oncology, reported that it was selected to present initial, first-in-human clinical results from the Phase 1/2 study of STX-478, a mutant-selective PI3Kα inhibitor, in advanced solid tumor patients in a Proffered Paper late-breaking session at the European Society for Medical Oncology ("ESMO") Congress 2024, taking place September 13 – 17, 2024 in Barcelona, Spain (Press release, Scorpion Therapeutics, AUG 19, 2024, View Source [SID1234645996]).

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"The presentation of first-in-human clinical data for our lead candidate, STX-478, marks an important milestone for patients living with advanced solid tumors driven by PI3Kα mutations, especially HR+/HER2- breast cancer, and for Scorpion as we work to broaden the reach and impact of precision oncology for patients with high unmet medical need," said Mark Chao, M.D., Ph.D., Chief Medical Officer of Scorpion. "While PI3Kα has been one of the highest profile targets in cancer for years, current treatment options suffer from toxicity limitations and subpar efficacy due to a lack of mutant selectivity. These initial data will provide early insight into how STX-478’s differentiated profile translates to potentially superior outcomes in PI3Kα-mutated cancers, and we look forward to presenting initial assessments of safety, pharmacokinetic and pharmacodynamic data and preliminary efficacy data at the ESMO (Free ESMO Whitepaper) 2024 Congress."

Details of the Proffered Paper presentation are as follows:

Title: First-in-human Results of STX-478, a Mutant-selective PI3Kα Inhibitor, in Advanced Solid Tumor Patients
Presentation Number: LBA27
Session Name: Proffered paper session 2: Developmental therapeutics (ID 156)
Session Date & Time: Sunday, September 15, 2024, at 14:45 CEST – 16:15 CEST (8:45 a.m. – 10:15 a.m. ET)
Presentation Time: 14:55 CEST – 15:05 CEST (8:55 a.m. – 9:05 a.m. ET)
Location: Salamanca Auditorium – Hall 5
Presenter: Alberto J. Montero, M.D. (University Hospitals Cleveland, OH, USA)

According to ESMO (Free ESMO Whitepaper), the late-breaking abstract will be published via the ESMO (Free ESMO Whitepaper) website at 00:05 CEST on September 15 (6:05 p.m. ET on September 14). Data presented at the conference will be available to view in the "Publications & Presentations" section of the Scorpion website (View Source) following the ESMO (Free ESMO Whitepaper) 2024 Congress.

About STX-478

STX-478 was designed to improve outcomes in patients harboring PI3Kα mutations, one of the most prevalent drivers of cancer, occurring in over 166,000 patients per year with breast, gynecological and other solid tumors in the United States alone. In preclinical models, STX-478 demonstrated robust activity across a range of PI3Kα mutations while sparing wild-type PI3Kα inhibition in normal tissues; previous generations of non-selective PI3Kα inhibitors have limited patient benefit due to these on-target toxicities. Scorpion’s Phase 1/2 clinical trial is a multi-center, global, open-label study designed to evaluate the safety and tolerability of STX-478 in multiple ascending doses for patients with locally advanced or metastatic HR+/HER2- breast cancer and other solid tumors driven by PI3Kα mutations. The program entered the clinic in 2023 and has rapidly advanced, now in multiple expansion cohorts across a range of solid tumors and in breast cancer as monotherapy and in combinations with fulvestrant and CDK4/6 inhibitors. To learn more about the first-in-human trial of STX-478, please visit this page.

On August 19, 2024 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, reported that the completion of enrollment in the pivotal Phase II clinical trial of satricabtagene autoleucel ("satri-cel", R&D code: CT041, an autologous CAR-T product candidate against Claudin18.2) in accordance with the clinical protocol, for advanced gastric/gastroesophageal junction cancers (GC/GEJ) in China (Press release, Carsgen Therapeutics, AUG 19, 2024, https://www.prnewswire.com/news-releases/carsgen-announces-enrollment-completion-in-china-gcgej-pivotal-phase-ii-clinical-trial-of-the-first-in-class-claudin18-2-car-t-satri-cel-302225000.html [SID1234645994]).

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This study is an open-label, multicenter, randomized clinical trial in China evaluating the efficacy and safety of satri-cel in subjects with Claudin18.2-positive advanced GC/GEJ patients who have failed at least two prior lines of therapy. Treatment for the control group is physician’s choice (paclitaxel, docetaxel, irinotecan, apatinib, or nivolumab).

Gastric cancer is one of the most common malignant tumors in China, with an incidence of approximately 359,000 incident cases and a mortality of approximately 260,000 deaths in 2022[1]. Patients with advanced gastric cancer often face limited treatment options and poor prognosis. There is a strong need for more innovative and efficacious therapies. Satri-cel is an autologous CAR T-cell product candidate against Claudin18.2 that has the potential to be first-in-class globally. It primarily targets GC/GEJ and pancreatic cancer (PC). The published clinical data on satri-cel have shown promising safety and efficacy.

The final results of the investigator-initiated Phase I clinical trial CT041-CG4006 (NCT03874897) were published in Nature Medicine and orally presented at the 2024 ASCO (Free ASCO Whitepaper) annual meeting. There were no dose-limiting toxicities (DLTs), no Grade 3 or higher cytokine release syndrome (CRS), or immune effector cell-associated neurotoxicity syndrome (ICANS) observed. In 51 patients with GC/GEJ with target lesions at baseline who received satri-cel monotherapy, the overall response rate (ORR) was 54.9% (28/51), the disease control rate (DCR) was 96.1% (49/51), and the median duration of response (mDOR) was 6.4 months.[2][3] The phase Ib results from CT041-ST-01 (NCT04581473) clinical trial presented at the 2022 ASCO (Free ASCO Whitepaper) annual meeting, further showed the manageable safety and promising efficacy of satri-cel. Among 14 GC/GEJ patients who had received at least 2 prior lines of treatment and received satri-cel monotherapy, there were no DLTs or AEs leading to death. No ICANS or gastrointestinal mucosal injury were reported. The ORR and DCR were 57.1% and 78.6% respectively.[4]

Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "We are very pleased to announce the successful completion of patient enrollment in the pivotal Phase II clinical trial of satri-cel in China. This significant milestone marks another solid step forward in our development of CAR T-cell therapies for solid tumors. We extend our gratitude to all the investigators, the patients and their families for their trust and support. We look forward to the submission of New Drug Application and the approval in China, to benefit patients with gastric cancer. As one of the leading companies in the field of CAR T-cell therapies, we remain committed to addressing the major challenges faced by existing CAR T-cell therapies and will develop more innovative CAR-T products for cancer patients."

About Satri-cel

Satri-cel is an autologous CAR T-cell product candidate against the protein Claudin18.2 that has the potential to be first-in-class globally. Satri-cel targets Claudin18.2 positive solid tumors with a primary focus on gastric cancer/gastroesophageal junction cancer (GC/GEJ) and pancreatic cancer (PC). Ongoing trials include investigator-initiated trials (CT041-CG4006, NCT03874897), a confirmatory Phase II clinical trial for advanced GC/GEJ in China (CT041-ST-01, NCT04581473), a Phase I clinical trial for PC adjuvant therapy in China (CT041-ST-05, NCT05911217), and a Phase 1b/2 clinical trial for advanced gastric or pancreatic adenocarcinoma in North America (CT041-ST-02, NCT04404595). Satri-cel was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. FDA for the treatment of Claudin18.2-positive advanced GC/GEJ cancers in January 2022 and PRIME eligibility by the EMA for the treatment of advanced gastric cancer in November 2021. Satri-cel received an Orphan Drug designation from the U.S. FDA in 2020 for the treatment of GC/GEJ and an Orphan Medicinal Product designation from the EMA in 2021 for the treatment of advanced gastric cancer.

On August 19, 2024 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells, reported a business and financial update for the second quarter 2024 (Press release, Cue Biopharma, AUG 19, 2024, View Source [SID1234645993]).

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Recent Business Highlights

Delivered oral presentation on updated data from ongoing Phase 1(b) trial of CUE-101 as a first line (1L) therapy in human papillomavirus positive (HPV+) recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC), with standard of care (SOC) checkpoint inhibitor (CPI) KEYTRUDA (pembrolizumab) and as a monotherapy in second line and beyond (2L+) HPV+ R/M HNSCC, as well as a poster presentation on CUE-102 in Wilms’ Tumor 1 (WT1) positive cancers at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting held in June.
Further advanced autoimmune program CUE-401, partnered with Ono Pharmaceutical, Ltd., and CUE-501, our lead CUE-500 series asset.
Programs are designed to address significant unmet medical need in large patient populations across numerous autoimmune and inflammatory diseases
Preclinical data has progressed well with consistent positive activity in multiple disease models
Initiated strategic prioritization of autoimmune programs to focus upon near-term and intermediate value creation potential, to enable optimization and reduction of capital requirements, while retaining oncology programs, CUE-101 and CUE-102, as promising clinical data further matures.
Positive observations as updated data continues to mature from ongoing oncology clinical trials, CUE-101 and CUE-102.
CUE-101 in combination with pembrolizumab in 1L R/M HNSCC, as of August 4, 2024, demonstrated an objective response rate (ORR) of 46% in all patients with combined positive score (CPS) ≥1 and 50% ORR with CPS <20, median progression free survival (mPFS) of 5.8 months and median overall survival (mOS) of 21.8 months
CUE-101 monotherapy in 2L+ HPV+ R/M HNSCC demonstrated mOS of 20.8 months vs. mOS of 7.5 and 8.4 months observed in trials in 2L patients with two different CPIs: OPDIVO (nivolumab) and pembrolizumab, respectively
CUE-102 monotherapy in late-stage refractory metastatic cancers has been well tolerated and to date, has demonstrated dose-dependent increases in exposure and activation and expansion of WT1-specific T cells with observed anti-tumor activity in two gastric and ovarian patients, as well as disease control in several tumor types in multiple patients in the dose escalation Phase 1 trial
"We had a highly productive second quarter with promising clinical data continuing to mature from our ongoing oncology trials further supporting our belief that CUE-101, as our lead representative CUE-100 program, has the potential to establish a new standard of care for HPV+ HNSCC patients," said Daniel Passeri, chief executive officer of Cue Biopharma. "These observations, combined with the ongoing advancements of our prioritized autoimmune programs and the recent implementation of a highly focused, strategic business model underscoring our objectives to proactively manage and mitigate capital access risk, support the transformational potential of our Immuno-STAT platform to accomplish our mission of developing breakthrough immunotherapies to establish a new standard of care in the treatment of cancer and autoimmune disease."

Second Quarter 2024 Financial Results
The Company reported collaboration revenue of $2.7 million and $1.4 million for the three months ended June 30, 2024 and 2023, respectively. The increase was due to the timing of revenue earned from the collaboration and option agreement with Ono Pharmaceutical Co., Ltd.

Research and development expenses were $9.5 million and $10.7 million for the three months ended June 30, 2024 and 2023, respectively. The decrease was primarily due to a decrease in research and laboratory costs and compensation expense.

General and administrative expenses were $3.5 million and $4.2 million for the three months ended June 30, 2024 and 2023, respectively. The decrease was primarily due to decreases in professional fees, employee compensation, overhead, and stock-based compensation expense.

The Company reported collaboration revenue of $4.4 million and $1.6 million for the six months ended June 30, 2024 and 2023, respectively. The increase was due to the timing of revenue earned from the collaboration and option agreement with Ono Pharmaceutical Co., Ltd.

Research and development expenses were $19.7 million and $20.0 million for the six months ended June 30, 2024 and 2023, respectively. The decrease was primarily due to a decrease in research and laboratory costs and stock-based compensation expense, partially offset by an increase in clinical expenses.

General and administrative expenses were $7.7 million and $8.4 million for the six months ended June 30, 2024 and 2023, respectively. The decrease was primarily due to decreases in professional fees and employee compensation.

As of June 30, 2024, the Company had $30.0 million in cash and cash equivalents compared with $48.5 million as of June 30, 2023. We expect our current cash, cash equivalents, and marketable securities to fund operations through the second quarter of 2025.