Imugene receives Orphan Drug Designation for treatment of Bile Tract Cancer

On September 18, 2024 Imugene Limited (ASX:IMU), a clinical-stage immuno‐ oncology company, reported that it has received Orphan Drug Designation (ODD) from the United States Food and Drug Administration (FDA) for its novel oncolytic virotherapy CF33-hNIS (VAXINIA), for the treatment of cholangiocarcinoma, a rare and aggressive form of bile tract cancer (Press release, Imugene, SEP 18, 2024, https://mcusercontent.com/e38c43331936a9627acb6427c/files/d4c82d92-18f3-59f9-598e-a451d46a7ffe/Imugene_Receives_Orphan_Drug_Designation_for_VAXINIA.pdf [SID1234646708]).

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The FDA grants Orphan Drug status to promote development of drugs for rare diseases that affect fewer than 200,000 people in the United States. This designation provides Imugene with a range of incentives, including tax credits, potential grant funding, waiver of certain administrative fees, and seven years of market exclusivity upon FDA approval.

Imugene’s application was supported by preclinical and clinical data from its ongoing Phase 1 MAST (Metastatic Advanced Solid Tumours) trial, which is investigating the safety and efficacy of VAXINIA. Promising results have already been observed, including one patient with cholangiocarcinoma achieving a complete response and another achieving stable disease after treatment with VAXINIA.

The MAST trial, which involves delivering the oncolytic virus either as a monotherapy or in combination with checkpoint inhibitors, is currently in the dose-escalation phase. This has recently been expanded into a further cohort of 10 patients with bile tract cancers, including cholangiocarcinoma.

Imugene’s Managing Director & CEO, Leslie Chong, commented: "Receiving Orphan Drug Designation from the FDA is a major milestone for us. It reflects the potential of VAXINIA to address the urgent need for new treatments for cholangiocarcinoma, a disease with limited therapeutic options. We are excited to continue advancing this program, which has already shown meaningful clinical responses in patients."

Cholangiocarcinoma is a rare malignancy arising from the bile ducts, which has a poor prognosis and is often resistant to conventional treatments, including chemotherapy and immunotherapy. Imugene’s innovative oncolytic virotherapy, VAXINIA, is designed to selectively target and destroy cancer cells, while also stimulating an immune response against the tumour.

BostonGene and Leading Global Cancer Institutions Announce Nature Publication

On September 17, 2024 BostonGene, a leading provider of AI-based molecular and immune profiling solutions, and researchers from multiple cancer centers including, Memorial Sloan Kettering Cancer Center, Dana-Farber Cancer Institute, Weill Cornell Medicine and Washington University School of Medicine in St. Louis and others, reported the online publication of the manuscript "Deep Clinical Responses and Limited Inflammatory Toxicity in Patients with Relapsed/Refractory T-cell Lymphomas Receiving Duvelisib and Romidepsin"* in Nature Medicine (Press release, BostonGene, SEP 17, 2024, View Source [SID1234646714]). The study highlights significant clinical responses and reduced inflammatory toxicity in patients with relapsed and refractory T-cell lymphomas treated with a combination of duvelisib and romidepsin.

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Historically, PI3K inhibitors have been associated with autoimmune and infectious toxicities, often leading to market withdrawals. Researchers from multiple leading cancer centers had previously demonstrated single-agent activity of the PI3K-γδ inhibitor duvelisib in T-cell lymphomas, although inflammatory adverse events were noted. Based on this preliminary work, a new phase 1b/2a clinical trial was launched to investigate the safety and efficacy of duvelisib in combination with either romidepsin or bortezomib in patients with relapsed/refractory T-cell lymphomas. Combining duvelisib and romidepsin significantly improved treatment outcomes in patients with peripheral T-cell lymphomas (PTCL).

To explore tumor molecular features that may be linked to therapeutic response to this novel therapeutic combination, BostonGene performed DNA whole exome (WES) and RNA transcriptome (RNAseq) sequencing on both pre-treatment and on-treatment biopsies. BostonGene then performed integrated multi-omic molecular analyses, including cellular deconvolution, mutational landscape analyses, longitudinal genetic reconstruction to study tumor evolution, and deciphering immune microenvironment dynamic changes. BostonGene uncovered that PTCL patients with a follicular helper T-cell subtype showed increased response rates to the combination and identified gene expression patterns potentially linked to therapeutic resistance.

"Our findings offer a promising new approach for treating patients with relapsed and refractory T-cell lymphomas, specifically those with the follicular helper T-cell subtype. We are excited about the potential of this combination therapy to improve patient outcomes and expand treatment options in this challenging disease," said Nathan Fowler, MD, Chief Medical Officer at BostonGene.

*Research conducted in collaboration with Memorial Sloan Kettering Cancer Center, Weill Cornell Medicine, Dana-Farber Cancer Institute, Washington University School of Medicine in St. Louis, Stanford University, State Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center Collaborative Innovation Center for Cancer Medicine, Merck and Co.

ImmPACT Bio to Participate at Cell & Gene Therapy International 2024

On September 17, 2024 ImmPACT Bio USA, Inc. ("ImmPACT Bio"), a clinical-stage company developing transformative logic-gate-based chimeric antigen receptor (CAR) T-cell therapies for treating cancer and autoimmune diseases, reported that it will participate at the Cell & Gene Therapy International 2024 meeting, to be held September 23-26, 2024 in Boston, MA (Press release, ImmPACT-Bio, SEP 17, 2024, View Source;gene-therapy-international-2024-302248187.html [SID1234646713]). Sylvain Roy, ImmPACT Bio’s chief technology officer, will provide a presentation about cell therapy manufacturing entitled, "Transitioning from Manual to Automated Processing in Autologous CAR T Manufacturing: A Case Study," on Wednesday, September 25, 2024 at 12:00 PM ET.

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"While the efficacy and durability of autologous CAR T therapies remain unparalleled, a key challenge for scalability and continued adoption is the complexity and cost of manufacturing," said Mr. Roy. "At this meeting, ImmPACT Bio will present a case study of transition from an established GMP manual process to an automated, closed-processing manufacturing platform, and address key aspects on the optimization of CAR T-cell therapy manufacturing."

Subgroup Analysis from Pivotal WU-KONG1B Study Exhibits Robust Efficacy of Sunvozertinib in Non-Small Cell Lung Cancer Patients with EGFR Exon 20 Insertion Mutations Across Different Baseline Characteristics

On September 17, 2024 Dizal (SSE:688192), a biopharmaceutical company committed to developing novel medicines for the treatment of cancer and immunological diseases, reported subgroup analysis findings of its WU-KONG1 Part B (WU-KONG1B) study at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress (Press release, Dizal Pharma, SEP 17, 2024, View Source [SID1234646712]). The results showed promising anti-tumor efficacy of sunvozertinib in relapsed or refractory non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations (exon20ins) across different baseline characteristics, underpinning its significant clinical value for this patient population around the globe.

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WU-KONG1B is an open-label, multinational pivotal study to investigate the efficacy and safety of sunvozertinib in relapsed or refractory NSCLC with EGFR exon20ins. The study is currently being conducted across 10 countries and regions in Asia, Europe, North America, and South America. WU-KONG1B met its primary endpoint, with the preliminary results featured as an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, demonstrating the transformative potential of sunvozertinib as a single, oral agent to treat EGFR exon20ins NSCLC. Results of the subgroup analysis were presented on September 14 at the 2024 ESMO (Free ESMO Whitepaper) Congress in Barcelona, Spain.

As of March 22, 2024, a total of 107 patients with at least 33 EGFR exon20ins subtypes were included in the efficacy analysis set. The key findings were as follows:

Per independent review committee (IRC) assessment, target lesions shrinkage was observed in 92.4% (98/106) of patients.
Per IRC assessment, the best objective response rate (ORR) was 53.3%, including 3 complete response (CR).
By EGFR exon20ins region classification, the best ORR in near loop, far loop, C-helix and unknown were 51.9%, 59.1%, 66.7% and 40%, respectively.
IRC assessed ORR was comparable between different subgroups regardless of race, region, baseline disease characteristics and prior anti-cancer treatment history.

Race

Region

Baseline BM

Best Response, n (%)

Asian

(n = 62)

Non-Asian

(n = 45)

Asia

(n = 58)

Non-Asia

(n = 49)

With

(n = 27)

Without

(n = 80)

CR

3 (4.8)

0 (0.0)

3 (5.2)

0 (0.0)

0 (0.0)

3 (3.8)

PR

32 (51.6)

22 (48.9)

29 (50.0)

25 (51.0)

18 (66.7)

36 (45.0)

Prior Amivantamab treatment

Prior IO treatment

Best Response, n (%)

With

(n = 14)

Without

(n = 93)

With

(n = 52)

Without

(n = 55)

CR

0 (0.0)

3 (3.2)

2 (3.8)

1 (1.8)

PR

7 (50.0)

47 (50.5)

26 (50.0)

28 (50.9)

With median follow-up of 7 months, duration of response (DoR) was not reached, and 66.7% of responders were still responding.
The safety profile was similar to previously reported results, and clinically manageable.
"WU-KONG1B study enrolled more than 40% of non-Asian patients. The subgroup analysis suggested superior anti-tumor efficacies and well-tolerated safety profiles of sunvozertinib across EGFR exon20ins NSCLC patients with different baseline demographics and clinical characteristics on a global scale. We are intensifying our efforts to advance ongoing global pivotal studies and regulatory submissions of this FDA Breakthrough Therapy Designated asset, making available an effective and safe oral option to more patients around the world." said Xiaolin Zhang, PhD, CEO of Dizal.

WU-KONG28, a phase Ⅲ multinational randomized study, is ongoing to assess sunvozertinib versus platinum-based doublet chemotherapy as a first-line treatment in patients from 16 countries and regions in Asia, Europe, North America, and South America. The anticipated data of this study is expected to further improve outcomes of patients in this realm.

About sunvozertinib (DZD9008)

Sunvozertinib is an irreversible EGFR inhibitor discovered by Dizal scientists targeting a wide spectrum of EGFR mutations with wild-type EGFR selectivity. In August 2023, sunvozertinib received approval from NMPA to treat advanced NSCLC with EGFR exon20ins after platinum-based chemotherapies. The approval is based on the results of WU-KONG6 study, the pivotal study of sunvozertinib in platinum-based chemotherapy pretreated NSCLC with EGFR exon20ins. The primary endpoint of the study was the confirmed overall response rate (cORR) as assessed by the Independent Review Committee (IRC) reached 60.8%. Anti-tumor efficacy was observed across a broad range of EGFR exon20ins subtypes, and in patients with pretreated and stable brain metastasis. In addition, sunvozertinib also demonstrated encouraging anti-tumor activity in NSCLC patients with EGFR sensitizing, T790M, and uncommon mutations (such as G719X, L861Q, etc.), as well as HER2 exon20ins.

Sunvozertinib showed a well-tolerated and manageable safety profile in the clinic. The most common drug-related TEAEs (treatment-emergent adverse event) were Grade 1/2 in nature and clinically manageable.

Two global pivotal studies are ongoing in ≥ 2nd line (WU-KONG1 Part B) and 1st line setting (WU-KONG28), respectively, in NSCLC patients with EGFR exon20ins.

Pre-clinical and clinical results of sunvozertinib were published in peer-reviewed journals Cancer Discovery (IF:39.397) and The Lancet Respiratory Medicine (IF: 76.2).

Innovent Announces Clinical Data of IBI363 (First-in-class PD-1/IL-2α-bias Bispecific Antibody Fusion Protein) Combined with Bevacizumab in Advanced Colorectal Cancer at the 2024 ESMO Congress

On September 17, 2024 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, autoimmune, cardiovascular and metabolic, ophthalmology and other major diseases, reported that clinical data of IBI363 (first-in-class PD-1/IL-2α-bias bispecific antibody fusion protein) combined with bevacizumab in advanced colorectal cancer is presented at the 2024 ESMO (Free ESMO Whitepaper) Congress (Press release, Innovent Biologics, SEP 17, 2024, View Source [SID1234646711]). Currently, Innovent is conducting Phase 1/2 clinical trials in China, the United States, and Australia to evaluate the safety, tolerability and efficacy of IBI363 in subjects with advanced solid tumors.

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First-in-class PD-1/IL-2α-bias bispecific antibody fusion protein IBI363 combined with bevacizumab in patients with advanced colorectal cancer: Results from Phase 1 study

This Phase 1 study was conducted to evaluate the safety, tolerability and preliminary efficacy of IBI363 combined with bevacizumab in subjects with advanced colorectal cancer.

A total of 35 subjects received treatment of IBI363 combined with bevacizumab, demonstrating promising anti-tumor efficacy with good tolerability and safety

As of the data cutoff date (Aug 30, 2024), a total of 35 subjects with advanced colorectal cancer received combination treatment at 3 different dose levels (0.6 mg/kg IBI363 combined with 5 mg/kg bevacizumab Q2W, 1 mg/kg IBI363 combined with 5 mg/kg bevacizumab Q2W, and 1.5 mg/kg IBI363 combined with 7.5 mg/kg bevacizumab Q3W). Among them, 91.4% of the subjects had advanced colorectal cancer with microsatellite stable (MSS) or proficient mismatch repair (pMMR), and the MSI/MMR status was unknown in 8.6% subjects. 91.4% of the subjects had previously received 2 or more lines of systemic anti-tumor treatment. 51.4% of the subjects had liver metastases. 25.7% of the subjects had received prior immunotherapy. 40% of the subjects had KRAS/NRAS exon 2/3/4 mutations.
The most common treatment related adverse events (TRAEs) were arthralgia, thyroid disorders, and rash. The total incidence of TRAEs ≥ grade 3 was 22.9%. Immune related adverse events (irAEs) ≥ grade 3 occurred in 5.7% of subjects. The safety profile of the combination regimen was similar to that of IBI363 monotherapy, and no new safety signals were identified.
Promising anti-tumor activity in subjects with MSS/pMMR colorectal cancer; durable responses with a trend towards long-term benefit

As of the cutoff date, 32 subjects were efficacy evaluable having underwent at least one post-baseline tumor assessment. The ORR was 21.9% (confirmed ORR was 15.6%), and DCR was 65.6%. The median DoR was 8.1 months (95% CI: 1.5~8.2). The median PFS follow-up time was 7.6 months (95% CI: 4.0~9.4), and the median PFS was 4.1 months (95% CI: 1.7~8.1). The median OS was not reached.
Promising efficacy signals in colorectal cancer with and without baseline liver metastases

Among the 17 subjects with baseline liver metastases who underwent at least one post-baseline tumor assessment, ORR was 11.8% and DCR was 58.8%.
Among the 15 subjects without baseline liver metastases who underwent at least one post-baseline tumor assessment, ORR was 33.3% and DCR was 73.3%.
Promising efficacy signals in both IO-treated and IO-naïve colorectal cancer

Among the 8 subjects who had received prior immunotherapy and underwent at least one post-baseline tumor assessment, ORR was 25.0% and DCR was 62.5%.
Among the 24 IO-naïve subjects who underwent at least one post-baseline tumor assessment, ORR was 20.8% and DCR was 66.7%.
Promising efficacy signals in colorectal cancer with and without KRAS/NRAS exon 2/3/4 mutations

Among the 14 subjects with RAS exon 2/3/4 mutations and who had undergone at least one post-baseline tumor assessment, ORR was 21.4% and DCR was 57.1%.
Among the 10 subjects without RAS exon 2/3/4 mutations who had undergone at least one post-baseline tumor assessment, ORR was 30.0% and DCR was 90.0%.
In addition, data from a Phase 1 clinical study of IBI363 monotherapy in a colorectal cancer cohort, presented at ASCO (Free ASCO Whitepaper) 2024, showed promising efficacy and good tolerability. Ongoing studies are now exploring IBI363 in other malignancies, including NSCLC, melanoma and other solid tumors, as well as in combination regimens for MSS/pMMR advanced colorectal cancer. Updates on relevant data and analysis will be shared at upcoming academic conferences and in journals.

Professor Tao Zhang, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, stated: "Colorectal cancer is the third most common cancer type globally and ranks fourth in cancer-related mortality[1]. Despite recent advancements in colorectal cancer treatment, challenges such as chemotherapy toxicity and resistance continue to affect patients and clinicians. While immunotherapy offers new hope for advanced colorectal cancer patients, it is currently only approved for those with MSI-H/dMMR tumors. Research indicates that immunotherapy has limited efficacy in non-MSI-H/dMMR advanced colorectal cancer[2]. As an important cytokine activating tumor-specific CD8+T cells, IL-2 is complementary to immune checkpoint inhibitors in MOA. The combination of PD-1 and IL-2 may reverse the exhaustion of tumor-specific CD8+ T cells thereby overcoming immune resistance. The PD-1/IL-2α-bias bispecific molecule IBI363, when combined with bevacizumab, has shown promising anti-tumor activity in patients with non-MSI-H/dMMR advanced colorectal cancer. This combination has shown clinical benefits in both ORR and PFS, and maintains a manageable safety profile. Additionally, the combination regimen has proven effective in colorectal cancer patients with or without liver metastasis, prior immunotherapy, and RAS exon 2/3/4 mutations. IBI363 combined with bevacizumab elicited encouraging objective response rates and disease control rates, with durable responses and a trend towards long-term benefits, without introducing new safety risks in a colorectal cancer population that has previously shown very little response to immunotherapy. Overall, current clinical data suggest that IBI363 combined with bevacizumab holds significant promise for colorectal cancer and deserves further exploration."

Dr. Hui Zhou, Senior Vice President of Innovent, stated: "On top of the preliminary data reported at ASCO (Free ASCO Whitepaper), we are presenting more updated data at the ESMO (Free ESMO Whitepaper) Congress. In non-MSI-H/dMMR advanced colorectal cancer, the combination of IBI363 with bevacizumab has demonstrated strong anti-tumor effects, with durable responses and a trend towards long-term benefits. These promising results in a relatively ‘cold’ tumor suggest significant potential for IBI363 in this disease area. We are confident in the broad development prospects of IBI363 and look forward to seeing more mature data from higher doses and extended follow-up, which will help us advance to the next stage of clinical development."

About IBI363 (First-in-class PD-1/IL-2α-bias bispecific antibody fusion protein)

IBI363 is a first-in-class PD-1/IL-2α-bias bispecific antibody fusion protein independently developed by Innovent Biologics. It functions by both blocking the PD-1/PD-L1 pathway and activating the IL-2 pathway. The IL-2 arm of IBI363 is designed to maintain its affinity for IL-2Rα while reducing binding to IL-2Rβ and IL-2Rγ, thereby minimizing toxicity. The PD-1 binding arm not only blocks PD-1 but also selectively delivers IL-2. This approach targets and activates tumor-specific T cells that express both PD-1 and IL-2α, leading to more precise and effective activation of this T cell subpopulation. IBI363 has demonstrated robust antitumor activity in various tumor-bearing pharmacological models, but also showed outstanding efficacy in PD-1 resistance and metastasis models. In response to urgent clinical needs, Innovent is conducting clinical studies in China, the United States and Australia to further explore the efficacy and safety of IBI363 in advanced tumors.