On August 19, 2024 Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) ("Tiziana" or the "Company"), a biotechnology company developing breakthrough immunomodulation therapies with its lead development candidate, intranasal foralumab, a fully human, anti-CD3 monoclonal antibody, reported the appointment of Ivor Elrifi as its Chief Executive Officer (CEO), effective immediately (Press release, Tiziana Life Sciences, AUG 19, 2024, View Source [SID1234645990]).

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"We are delighted to welcome Ivor Elrifi as the new CEO of Tiziana Life Sciences," said Gabriele Cerrone, Founder and Executive Chairman of the Board. " Ivor has an invaluable track record in creating substantial value for biotech and pharma companies with his strategic vision in building intellectual property portfolios and transforming the IP into transformative business development deals and M&A transactions. Tiziana looks forward to creating further value with Ivor as we proceed with our human clinical trials."

Tiziana Life Sciences is currently focused on advancing intranasal foralumab through clinical trials for non-active Secondary Progressive Multiple Sclerosis (na-SPMS), Alzheimer’s Disease and ALS. Under Ivor’s leadership, the company aims to accelerate these programs and capitalize on opportunities to address significant unmet medical needs.

Ivor was formerly the global head of the Patent Group at Cooley since 2014 and before that the global head of Patents at Mintz Levin from 1999 – 2014. He has counseled companies in various key industries, including pharmaceutical, biotechnology, life sciences and medical device companies, research institutions, universities, hospitals and governments throughout the world, particularly in the US and Europe. Ivor has guided clients in developing and implementing intellectual property strategies and in the prosecution, licensing and enforcement of patents. He has extensive experience in advising clients on strategic transactional work and regularly counsels’ clients with respect to investments, business development and mergers and acquisitions, including acquisition transactions involving Novartis, Eli Lilly, Biogen and Astellas.

He has received various awards throughout his career, including being named an "LMG Life Sciences: Life Science Star," and ranked nationally in Chambers USA since 2007. Elrifi earned his B.S. and Ph.D. in Biology from Queen’s University and his J.D. from Osgoode Hall Law School.

"I am honored to lead Tiziana Life Sciences at this pivotal time in its growth trajectory," said Ivor Elrifi. "The company’s commitment to developing transformative therapies holds great promise, and I look forward to working with the talented team to advance our clinical programs and deliver value to patients and shareholders."

Mr. Elrifi succeeds Gabriele Cerrone, who was acting CEO and will remain in his role as Executive Chairman of the board and will continue to play a key role in advancing Tiziana’s scientific agenda.

About Foralumab

Foralumab, a fully human anti-CD3 monoclonal antibody, is a biological drug candidate that has been shown to stimulate T regulatory cells when dosed intranasally. At present, 10 patients with Non-Active Secondary Progressive Multiple Sclerosis (na-SPMS) have been dosed in an open-label intermediate sized Expanded Access (EA) Program with either an improvement or stability of disease seen within 6 months in all patients. The FDA has recently allowed an additional 20 patients to be enrolled in this EA program. In addition, intranasal foralumab is currently being studied in a Phase 2a, randomized, double-blind, placebo-controlled, multicenter, dose-ranging trial in patients with non-active secondary progressive multiple sclerosis (NCT06292923).

Activated T cells play an important role in the inflammatory process. Foralumab, the only fully human anti-CD3 monoclonal antibody (mAb), binds to the T cell receptor and dampens inflammation by modulating T cell function, thereby suppressing effector features in multiple immune cell subsets. This effect has been demonstrated in patients with COVID and with multiple sclerosis, as well as in healthy normal subjects. The non-active SPMS intranasal foralumab Phase 2 trial (NCT06292923) began screening patients in November of 2023. Immunomodulation by nasal anti-CD3 mAb represents a novel avenue for treatment of neuroinflammatory and neurodegenerative human diseases.

On August 19, 2024 Daiichi Sankyo and Astrazeneca reported that ENHERTU (fam-trastuzumab deruxtecan-nxki) has been granted Breakthrough Therapy Designation (BTD) in the U.S. for the treatment of unresectable or metastatic hormone receptor positive HER2 low (IHC 1+ or IHC 2+/ISH-) or HER2 ultralow (IHC >0 <1+) breast cancer patients who have received either two lines of endocrine therapy in the metastatic setting, or one line of endocrine therapy if they had demonstrated disease progression within six months of starting first-line treatment with endocrine therapy in combination with a CDK4/6 inhibitor or within 24 months of the start of adjuvant endocrine therapy (Press release, Daiichi Sankyo, AUG 19, 2024, View Source [SID1234645992]).

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ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (LSE/STO/Nasdaq: AZN). The U.S. Food and Drug Administration (FDA) BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines.

The FDA granted this BTD based on data from the DESTINY-Breast06 phase 3 trial presented as a late-breaking oral session at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (#ASCO24) Annual Meeting.

"If approved, ENHERTU could once again change the treatment paradigm for certain patients with breast cancer, pushing past old boundaries and broadening the number of people who may be eligible for a HER2 directed therapy," said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. "The designation also showcases Daiichi Sankyo’s commitment to pioneering cutting-edge science to deliver medicines like ENHERTU that create new standards of care for patients with cancer."

ENHERTU has received eight BTDs, including four in metastatic breast cancer. In addition to the new BTD, the three previous BTDs for breast cancer were for later-line HER2 low metastatic breast cancer, second-line HER2 positive metastatic breast cancer and later-line HER2 positive metastatic breast cancer. ENHERTU also received four additional BTDs, including for HER2 positive (IHC 3+) metastatic solid tumors, HER2 positive metastatic colorectal cancer, HER2 (ERBB2) mutant metastatic non-small cell lung cancer (NSCLC) and HER2 positive metastatic gastric cancer. The new BTD for ENHERTU also represents the eleventh BTD across Daiichi Sankyo’s oncology pipeline.

About DESTINY-Breast06

DESTINY-Breast06 is a global, randomized, open-label, phase 3 trial evaluating the efficacy and safety of ENHERTU (5.4 mg/kg) versus investigator’s choice of chemotherapy (capecitabine, paclitaxel or nab paclitaxel) in patients with HR positive, HER2 low (IHC 1+ or IHC 2+/ISH-) or HER2 ultralow (defined as IHC 0 with membrane staining [IHC >0 <1+]) advanced or metastatic breast cancer. Patients in the trial had no prior chemotherapy for advanced or metastatic disease and received at least two lines of prior endocrine therapy in the metastatic setting. Patients also were eligible if they had received one prior line of endocrine therapy combined with a CDK4/6 inhibitor in the metastatic setting and experienced disease progression within six months of starting first-line treatment or received endocrine therapy as an adjuvant treatment and experienced disease recurrence within 24 months.

The primary endpoint of DESTINY-Breast06 is progression-free survival (PFS) in the HR positive, HER2 low patient population as measured by blinded independent central review (BICR). Key secondary endpoints include PFS by BICR in the overall trial population (HER2 low and HER2 ultralow), overall survival (OS) in patients in the HER2 low patient population and OS in the overall trial population. Other secondary endpoints include objective response rate, duration of response, time to first subsequent treatment or death, time to second subsequent treatment or death and safety. Analysis of the HER2 ultralow subgroup was not powered to demonstrate statistical significance.

DESTINY-Breast06 enrolled 866 patients (n=713 for HER2 low and n=153 for HER2 ultralow) at multiple sites in Asia, Europe, North America, Oceania and South America. For more information about the trial, visit ClinicalTrials.gov.

About Breast Cancer and HER2 Expression

Breast cancer is the second most common cancer and one of the leading causes of cancer-related deaths worldwide.1 More than two million breast cancer cases were diagnosed in 2022 with more than 665,000 deaths globally.1 In the U.S., more than 300,000 cases of breast cancer are diagnosed annually.2 While 3 survival rates are high for those diagnosed with early breast cancer, only about 30% of patients diagnosed with or who progress to metastatic disease are expected to live five years following diagnosis.3 HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumors, including breast cancer.4 Patients with high levels of HER2 expression (IHC 3+ or IHC2+/ISH+) are classified as HER2 positive and treated with HER2 targeted therapies, representing approximately 15 to 20% percent of all breast cancers.5 Historically, tumors that were not classified as HER2 positive were classified as HER2 negative, despite the fact that many of these tumors still carry some level of HER2 expression.6 It is estimated that approximately 60% to 65% of HR positive, HER2 negative breast cancers are HER2 low and potentially an additional 25% may be HER2 ultralow.7,8 Endocrine therapies are widely given consecutively in the early lines of treatment for HR positive metastatic breast cancer. However, following two lines of endocrine therapy, further efficacy with additional endocrine treatment is often limited.

The current standard of care following endocrine therapy is chemotherapy, which is associated with poor response rates and outcomes.9,10,11,12 Prior to the approval of ENHERTU following chemotherapy in HER2 low metastatic breast cancer based on the DESTINY-Breast04 trial, there were no targeted therapies approved specifically for patients with HER2 low expression.13 There are no targeted therapies specifically approved for patients with HER2 ultralow expression.14 About ENHERTU ENHERTU (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in the U.S. only) is a HER2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, ENHERTU is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC scientific platform.

ENHERTU consists of a HER2 monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. ENHERTU (5.4 mg/kg) is approved in more than 65 countries worldwide for the treatment of adult patients with unresectable or metastatic HER2 positive (IHC 3+ or in-situ hybridization (ISH)+) breast cancer who have received a prior anti-HER2-based regimen, either in the metastatic setting or in the neoadjuvant or adjuvant setting, and have developed disease recurrence during or within six months of completing therapy based on the results from the DESTINY-Breast03 trial.

ENHERTU (5.4 mg/kg) is approved in more than 65 countries worldwide for the treatment of adult patients with unresectable or metastatic HER2 low (IHC 1+ or IHC 2+/ISH-) breast cancer who have received a prior 4 systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy based on the results from the DESTINY-Breast04 trial.

ENHERTU (5.4 mg/kg) is approved in more than 35 countries worldwide for the treatment of adult patients with unresectable or metastatic NSCLC whose tumors have activating HER2 (ERBB2) mutations, as detected by a locally or regionally approved test, and who have received a prior systemic therapy based on the results from the DESTINY-Lung02 trial. Continued approval in the U.S. for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ENHERTU (6.4 mg/kg) is approved in more than 45 countries worldwide for the treatment of adult patients with locally advanced or metastatic HER2 positive (IHC 3+ or IHC 2+/ISH+) gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01, DESTINY-Gastric02 and/or DESTINY-Gastric06 trials. Full approval in China for this indication will depend on whether a randomized controlled confirmatory clinical trial can demonstrate clinical benefit in this population.

ENHERTU (5.4 mg/kg) is approved in the U.S. for the treatment of adult patients with unresectable or metastatic HER2 positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options based on efficacy results from the DESTINY-PanTumor02, DESTINY-Lung01 and DESTINY-CRC02 trials. Continued approval for this indication in the U.S. may be contingent upon verification and description of clinical benefit in a confirmatory trial. About the ENHERTU Clinical Development Program A comprehensive global clinical development program is underway evaluating the efficacy and safety of ENHERTU monotherapy across multiple HER2 targetable cancers. Trials in combination with other anticancer treatments, such as immunotherapy, also are underway

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.

On August 19, 2024 Akari Therapeutics, Plc (Nasdaq: AKTX), an innovative biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, reported financial results for the second quarter ended June 30, 2024 as well as recent company highlights (Press release, Akari Therapeutics, AUG 19, 2024, View Source [SID1234645976]).

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"Moving into my fourth month as Interim CEO of Akari, we continue to make progress on multiple fronts. We prioritized our PAS-nomacopan geographic atrophy development program, and I am pleased to report we are making steady progress. In July we received positive and constructive Pre-IND (PIND) feedback from the US FDA and based on the feedback, we plan on filing an IND application in 2025 for our Phase 1 clinical studies of PAS-nomacopan in geographic atrophy. In addition, in support of this IND application, I am happy to share that we have released our first full-scale batch of drug substance under applicable Good Manufacturing Practice (GMP) conditions, which will be suitable for clinical use. This manufacturing has been supported by our manufacturing partner, Wacker Biotech GmbH. Sufficient clinical material has been produced to support both the final IND-enabling studies and initial clinical development in geographic atrophy (GA). I would like to thank the FDA as well as Dr. Miles Nunn and his development team at Akari, who have worked tirelessly to advance the development of PAS-nomacopan into the clinic," stated Dr. Samir R. Patel, Interim CEO of Akari.

"Our merger with Peak Bio continues to progress and we remain on track for a fourth quarter closing," continued Dr. Patel. "We continue to explore licensing and partnership opportunities for both nomacopan and PAS-nomacopan."

Recent Company Highlights

Announced portfolio prioritization plan for combined go-forward company which will focus on Peak’s antibody drug conjugate (ADC) platform technology and Akari’s PAS-nomacopan GA program. As a result of this prioritization, the Company’s HSCT-TMA program was suspended.
Announced key leadership changes, including the appointment of experienced life sciences entrepreneur Samir R. Patel, M.D. as interim CEO. Interim CEO employment contract consists solely of equity compensation.
Implemented reduction-in-force as part of an operational restructuring plan, which included the elimination of certain senior management positions, to reduce operating costs while supporting the Company’s long-term strategic plan.
Issued unsecured convertible, short-term promissory notes to Dr. Patel and Ray Prudo, M.D., the Company’s Chairman of the Board, each in the amount of $500,000 to provide operating capital.
Raised a total of $7.6 million in gross proceeds from a private placement of ADSs and warrants with certain investors, including Dr. Patel and Dr. Prudo.
Received positive and constructive regulatory feedback from the US FDA for PAS-nomacopan in the treatment of GA which will provide alignment and clarity on Akari’s IND enabling preclinical plans and clinical strategy prior to advancement into Phase 1 clinical studies. Based on the FDA’s feedback, the company plans to file an IND application for the use of PAS-nomacopan in GA in the second half of 2025.
Full-scale drug substance GMP batch manufactured by Wacker Biotech GmbH has been released and is suitable for use in the clinic, providing enough clinical material to support both the final IND-enabling studies and initial clinical development in GA.
Second Quarter 2024 Financial Results

As of June 30, 2024, the Company had cash of $4.2 million, compared to $3.8 million as of December 31, 2023.

Research and development expenses were $3.3 million and $5.6 million for the three and six months ended June 30, 2024, respectively, as compared to $1.5 million and $3.3 million, respectively, for the same periods in 2023. The increases on a year-to-date basis were due primarily to increases in manufacturing costs associated with the development of PAS-nomacopan.

General and administrative expenses were $2.2 million and $4.9 million for the three and six months ended June 30, 2024, respectively, as compared to $3.1 million and $6.0 million, respectively, for the same periods in 2023. The decreases on a year-to-date basis were due primarily to decreased headcount as part of the implementation of a reduction-in-force as part of our operational restructuring plan that was announced in May 2024.

Merger-related costs were $0.3 million and $1.3 million for the three and six months ended June 30, 2024, respectively, and represent costs incurred directly related to the proposed merger with Peak Bio, which was announced in March 2024. No such costs were incurred during the same periods in 2023.

Restructuring and other costs were $1.6 million for each of the three and six months ended June 30, 2024 and relate to costs incurred directly related to the reduction-in-force as part of our operational restructuring plan, which was announced in May 2024. No such costs were incurred during the same periods in 2023.

Net loss was $7.6 million and $13.1 million for the three and six months ended June 30, 2024, respectively, as compared to $4.0 million and $3.0 million, respectively, for the same periods in 2023.

Readers are referred to, and encouraged to read in its entirety, the company’s Quarterly Report on Form 10-Q for the three and six months ended June 30, 2024, as filed with the Securities and Exchange Commission on August 19, 2024, which includes further detail on the Company’s business plans, operations, financial condition, and results of operations.

About the Merger

On March 5, 2024, Akari and Peak Bio announced a definitive agreement to merge as equals in an all-stock transaction. The combined entity will operate as Akari Therapeutics, Plc, which is expected to continue to be listed and trade on the Nasdaq Capital Market as AKTX, under the Chairmanship of Hoyoung Huh, MD, PhD. Under the terms of the agreement, Peak stockholders will receive a number of Akari ordinary shares (represented by American Depositary Shares) for each share of Peak stock they own, as determined on the basis of the exchange ratio described in the agreement. The exchange is expected to result in implied equity ownership in the combined company of approximately 50% for Akari shareholders and approximately 50% for Peak stockholders on a fully diluted basis, subject to adjustment under certain circumstances, including based on each party’s relative level of net cash at the closing of the proposed transaction. The transaction is expected to close in the fourth quarter of this year subject to the satisfaction of customary closing conditions, including approval by the shareholders of both companies.

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.