Innate Pharma announces clinical data presentations on IPH5401 and monalizumab at ESMO 2019

On September 23, 2019 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH) reported that it will provide an update on its clinical trial pipeline at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress in Barcelona, Spain, September 27-October 1, 2019 (Press release, Innate Pharma, SEP 23, 2019, View Source [SID1234539684]). Highlights will include preliminary results of STELLAR-001, a Phase I dose escalation study of the anti-C5aR antibody, IPH5401, in combination with durvalumab in advanced solid tumors. In addition, updated data from ongoing clinical trials evaluating lead asset, monalizumab, partnered with AstraZeneca, will be presented.

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"We are very pleased to present new data on two of our clinical assets at ESMO (Free ESMO Whitepaper) Congress 2019. Our poster on IPH5401 will be the first clinical data presentation for this agent in combination with an anti-PD-L1 antibody in oncology," commented Pierre Dodion, Chief Medical Officer of Innate Pharma. "We will also present late follow-up clinical data on the combination of monalizumab and cetuximab in head and neck cancer. We look forward to sharing this new, promising clinical data to the medical community."

Poster presentations September 30, 2019, 12:00-13:00 – Poster Area Hall 4

Preliminary results of STELLAR-001, a dose escalation phase I study of the anti-C5aR, IPH5401, in combination with durvalumab in advanced solid tumors. (ID 1285)
Poster #1203P | Presenter: Christophe Massard (Villejuif, France)
Monalizumab in combination with cetuximab in patients (pts) with recurrent or metastatic (R/M) head and neck cancer (SCCHN) previously treated or not with PD-(L)1 inhibitors (IO): 1-year survival data. (ID 1242)
Poster #1134P | Presenter: Roger B. Cohen (Philadelphia, PA, USA)
Durvalumab + monalizumab, mFOLFOX6, and bevacizumab in patients (pts) with metastatic microsatellite-stable colorectal cancer (MSS-CRC) (ID 3681)
Poster #1201P | Presenter: May Cho (Sacramento, CA, USA)

Roche to present new data from its broad oncology portfolio at the European Society for Medical Oncology 2019 Congress

On September 23, 2019 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that results from a number of studies across its comprehensive oncology portfolio, covering a broad range of cancers including bladder, lung and breast will be presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress, taking place from 27 September – 1 October in Barcelona, Spain (Press release, Hoffmann-La Roche, SEP 23, 2019, View Source [SID1234539683]). A total of 100 abstracts and 15 late-breaking abstracts that include a Roche medicine will be presented at this year’s congress.

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"We are excited to present a number of key datasets across a broad range of diseases at ESMO (Free ESMO Whitepaper) this year, including results from our Tecentriq combination study in people with previously untreated advanced bladder cancer, the first positive Phase III cancer immunotherapy trial in this setting. In addition, we will present data from our Phase III Tecentriq monotherapy study in previously untreated advanced lung cancer," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "We will also share data from a study using Foundation Medicine’s liquid (blood) biopsy, a more patient-friendly approach to diagnosing and treating cancer. The results of the first cohort of this study involving ALK+ non-small cell lung cancer patients who received Alecensa will be presented at the congress."

Follow Roche on Twitter via @Roche and keep up to date with ESMO (Free ESMO Whitepaper) 2019 Congress news and updates by using the hashtag #ESMO19.

Key presentations
Bladder cancer:
First results from the Phase III IMvigor130 study will be presented as an oral presentation at the Presidential Symposium on 30 September at 17:53-18:05 CEST (abstract LBA14) and will be featured in the official ESMO (Free ESMO Whitepaper) press programme at 07:30 CEST on the same day. This study evaluates the efficacy and safety of Tecentriq (atezolizumab) in combination with platinum-based chemotherapy or alone versus platinum-based chemotherapy in patients with previously untreated locally advanced or mUC, and supports Roche’s broad clinical development programme for Tecentriq in bladder cancer.

IMvigor130 met its co-primary endpoint of progression-free survival (PFS). The combination showed a statistically significant reduction in the risk of disease worsening or death in people with previously untreated locally advanced or metastatic urothelial carcinoma (mUC). Encouraging overall survival (OS) results were observed at this interim analysis, however these results are not yet mature. The study represents the first positive Phase III immunotherapy combination study in patients with previously untreated bladder cancer. Tecentriq monotherapy was the first cancer immunotherapy approved in bladder cancer.

Lung cancer:
Alecensa (alectinib)
Results from the first cohort of the Phase II/III Blood First Assay Screening Trial (BFAST) will be presented at ESMO (Free ESMO Whitepaper) 2019. BFAST is the first prospective trial to use blood-based next generation sequencing (NGS) as the sole method of identifying and assigning non-small cell lung cancer (NSCLC) patients to targeted therapy based on actionable genetic alterations without the need for tissue biopsy. The study utilised FoundationOne Liquid, Foundation Medicine’s liquid (blood) biopsy assay to detect fusions in circulating tumour DNA (ctDNA) from a simple blood draw to identify anaplastic lymphoma kinase (ALK) status and consequently eligibility for Alecensa.

In addition to BFAST, updated data from the pivotal Phase III ALEX study investigating the efficacy and tolerability of Alecensa in comparison to crizotinib in patients with untreated ALK-positive NSCLC will also be presented at the congress, along with real world data on the use of Alecensa in clinical practice from the Flatiron Health database.

Tecentriq (atezolizumab)
Results from the Phase III IMpower110 study evaluating Tecentriq as first-line monotherapy compared with cisplatin or carboplatin and pemetrexed or gemcitabine (chemotherapy) as first-line treatment in patients with advanced squamous and non-squamous NSCLC will be presented. The study met its primary endpoint of OS, demonstrating a statistically significant OS benefit for Tecentriq monotherapy in people with high levels of PD-L1 expression, compared with chemotherapy alone.

In addition, updated OS results from the IMpower133 study will also be presented at ESMO (Free ESMO Whitepaper). IMpower133 showed that first-line Tecentriq in combination with chemotherapy helped people with extensive-stage small cell lung cancer (ES-SCLC) live significantly longer compared with chemotherapy alone. This updated data presentation will also feature analysis by PD-L1 subgroup.

Rozlytrek(entrectinib)
Additionally, results from studies in partnership with Flatiron Health will be presented, including validation of the use of NGS data on a broad scale to improve the understanding of clinical outcomes in people with metastatic lung cancer, and results that illustrate how real-world data can be used to supplement evidence from clinical trials comparing Rozlytrek clinical data with a matched cohort treated with current standard of care from the Flatiron database in ROS1-positive lung cancer.

Liver cancer (HCC):
Tecentriq + Avastin (bevacizumab)
Results will be presented from a Phase Ib study evaluating the efficacy and safety of Tecentriq plus Avastin as a treatment for people with unresectable hepatocellular carcinoma (HCC), the most common form of liver cancer, who have not received prior systemic therapy. Data will be presented underscoring the potential benefit of Tecentriq plus Avastin for HCC patients, as well as randomised data testing the combination versus Tecentriq monotherapy.

Breast cancer:
Tecentriq
During ESMO (Free ESMO Whitepaper), data from a post-hoc (exploratory) analysis of the IMpassion130 study will be presented, evaluating the performance of VENTANA PD-L1 (SP142) and two other PD-L1 immunohistochemistry (IHC) assays in unresectable locally advanced or metastatic triple-negative breast cancer (mTNBC) in predicting clinical activity of Tecentriq plus nab-paclitaxel.

Primary results of IMpassion130 showed that Tecentriq plus nab-paclitaxel reduced the risk of disease worsening or death, and an encouraging OS was observed at interim analysis in people with mTNBC who were tested positive for PD-L1 expression on tumour-infiltrating immune cells compared to nab-paclitaxel alone.

The assessment of PD-L1 on tumour-infiltrating immune cells is essential for identifying the patients with mTNBC benefiting from this Tecentriq combination. PD-L1 expression status in the IMpassion130 study was assessed by the VENTANA PD-L1 (SP142) assay.

HER2 portfolio/medicines
Exploratory analysis of the international Phase III, KATHERINE trial – evaluating the efficacy and safety of Kadcyla (trastuzumab emtansine) versus Herceptin (trastuzumab) as an adjuvant therapy in people with HER2-positive early breast cancer (eBC) who have residual disease after neoadjuvant taxane and Herceptin-based treatment, have been accepted for oral presentation at this year’s congress. This updated analysis examined the association of prior neoadjuvant treatment with characteristics of peripheral neuropathy and thrombocytopenia amongst the study population observed in the KATHERINE trial as well as further data on central nervous system (CNS) recurrences.

Primary results from the Phase III KATHERINE study showed that Kadcyla significantly reduced the risk of invasive breast cancer recurrence or death from any cause (invasive disease-free survival; iDFS) by 50% (Hazard Ratio [HR]=0.50, 95% CI: 0.39-0.64, p<0.0001) compared to Herceptin as an adjuvant treatment in people with HER2-positive eBC who have residual invasive disease after neoadjuvant taxane and Herceptin-based treatment.

Also being presented at ESMO (Free ESMO Whitepaper) 2019, are data from an epidemiological study looking at the impact of adjuvant Kadcyla on the incidence of metastatic breast cancer (mBC), as well as results from an analysis of data from the Flatiron database, which assessed challenges in determining the effectiveness of HER2-targeted treatment sequencing in the real world.

OS results from the Phase II KATE2 study, evaluating the efficacy and safety of Kadcyla in combination with Tecentriq versus Kadcyla and placebo in people with HER2-positive locally advanced or mBC who have received prior Herceptin and taxane-based therapy, will also be shared in an oral presentation.

Cancer of Unknown Primary Site (CUP):
Featured in the official ESMO (Free ESMO Whitepaper) press programme at 08:30 on 28 September will be a retrospective analysis using comprehensive genomic profiling (CGP) of carcinoma of unknown primary origin (CUP) patients that investigated eligibility for molecularly guided targeted or immunotherapy treatment options in the CUPISCO study design. To make that determination, archival tissue from centrally reviewed CUP cases was subjected to CGP using the FoundationOne CDx test. CUPISCO is a Phase II, randomised, active-controlled, multi-centre study comparing the efficacy and safety of targeted therapy or cancer immunotherapy guided by genomic profiling versus platinum-based chemotherapy in patients with a CUP site.

Overview of key presentations featuring Roche medicines at ESMO (Free ESMO Whitepaper) 2019

About Roche in Oncology
Roche has been working to transform cancer care for more than 50 years, bringing the first specifically designed anti-cancer chemotherapy drug, fluorouracil, to patients in 1962. Roche’s commitment to developing innovative medicines and diagnostics for cancers remains steadfast.

The Roche Group’s portfolio of innovative cancer medicines includes: Alecensa (alectinib); Avastin (bevacizumab); Cotellic (cobimetinib); Erivedge (vismodegib); Gazyva/Gazyvaro (obinutuzumab); Herceptin (trastuzumab); Kadcyla (trastuzumab emtansine); MabThera/Rituxan (rituximab); Perjeta (pertuzumab); Polivy (polatuzumab vedotin-piiq); Tarceva (erlotinib); RozlytrekTM (entrectinib); Tecentriq (atezolizumab); Venclexta/Venclyxto (venetoclax); Xeloda (capecitabine); Zelboraf (vemurafenib). Furthermore, the Roche Group has a robust investigational oncology pipeline focusing on new therapeutic targets and novel combination strategies. For more information on Roche’s approach to cancer, visit www.roche.com.

Clovis Oncology Acquires Rights to FAP-Targeted Radiopharmaceutical Program from 3B Pharmaceuticals

On September 23, 2019 Clovis Oncology, Inc. (NASDAQ: CLVS) reported that it has entered into a global licensing and collaboration agreement with 3B Pharmaceuticals GmbH (3BP), a private German biotechnology company developing targeted radiopharmaceutical drugs and diagnostics for oncology indications with a high unmet medical need (Press release, Clovis Oncology, SEP 23, 2019, View Source [SID1234539682]). The initial focus is on developing a peptide-targeted radionuclide therapy (PTRT) and imaging agent targeting fibroblast activation protein alpha (FAP). FAP is highly expressed in many epithelial cancers, including more than 90 percent of breast, lung, colorectal and pancreatic carcinomas.1 Clovis will conduct global clinical trials and has obtained U.S. and global rights, excluding Europe (inclusive of Russia, Turkey and Israel), where 3BP retains rights. The parties have also agreed to collaborate on a discovery program directed at three additional targets for radionuclide therapy, to which Clovis will have global rights.

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This press release features multimedia. View the full release here: View Source

Terms of the transactions include approximately $12 million in upfront payments to 3BP. Upon achievement of certain development and regulatory milestones, additional potential milestone payments and single- to low-double-digit commercial royalties would be paid to 3BP by Clovis. Clovis will be responsible for a limited number of 3BP full-time employees (FTEs) and external costs during the pre-clinical development. Research and development expense guidance provided by Clovis on its August 1 financial results call does not change as a result of today’s announcement.

"We are extremely enthusiastic about the opportunity to develop this novel class of targeted radiopharmaceutical therapies, with an initial focus on fibroblast activation protein alpha. Targeted radiopharmaceutical therapy represents a next frontier in oncology drug development, with potential application across multiple tumor types. In particular, FAP represents a very compelling target given its overexpression across numerous tumor types and limited expression in healthy tissue," said Patrick J. Mahaffy, President and CEO of Clovis Oncology. "Additionally, as a result of our discovery collaboration, Clovis intends to further expand its pipeline with additional targeted radiopharmaceutical candidates that result from the discovery program using 3BP’s technology platform. We are delighted to work with 3BP given their leadership in the discovery and development of peptide-targeted radionuclide therapies."

The collaboration is initially focused on the development of an FAP-targeted preclinical candidate identified by 3BP’s technology platform. FAP is highly expressed in cancer-associated fibroblasts (CAFs) which are found in the majority of cancer types and play an intricate role in driving tumor growth. Targeting CAFs with an FAP radiopharmaceutical is believed to have multiple modes of anti-tumor action, but principally relies on the induction of DNA damage in tumor cells by ionizing radiation emitted locally from neighboring CAFs targeted by the therapy.

Clovis and 3BP also announced their intention to enter into a collaboration for the discovery and development of radiopharmaceuticals for three additional targets using 3BP’s technology platform. 3BP will be responsible for discovery activities for the three targets. Once lead molecules have been identified, responsibilities will transition to Clovis for Investigational New Drug (IND)-enabling studies.

"We have focused for many years on developing a peptide technology platform for the discovery and development of innovative radiopharmaceuticals, which we believe represents the best means of selectively delivering potent radiation to tumors," said Dr. Ulrich Reineke, Managing Director of 3BP. "As we are approaching clinical development, we are very enthusiastic about partnering with Clovis Oncology to move our FAP-targeted product forward and to collaborate further on building a portfolio of targeted radiopharmaceutical therapeutics. We believe this is an ideal partnership for the rapid clinical development of our radiopharmaceuticals for the benefit of patients with many different types of cancer."

About Fibroblast Activation Protein Alpha (FAP)

Fibroblast activation protein alpha, or FAP, is highly expressed in cancer-associated fibroblasts (CAFs) which are found in the majority of cancer types, potentially making it a suitable target across a wide array of solid tumors. FAP is highly expressed in many epithelial cancers, including more than 90 percent of breast, lung, colorectal and pancreatic carcinomas.1 CAFs are highly prevalent in the tumor microenvironment of many cancers and persist through all malignant stages of a tumor, from primary tumor to metastasis. FAP has limited expression on normal fibroblasts, reducing the potential for effects in normal tissue.

About Peptide-Targeted Radionuclide Therapy (PTRT)

Peptide-targeted radionuclide therapy involves a small amount of radioactive material (radionuclide) that is combined with a cell-targeting moiety peptide for the treatment of cancer; PTRT is considered a form of radiopharmaceuticals. The targeting peptide is able to recognize and bind to specific features of tumors, such as antigens and cell receptors. When injected into the patient’s bloodstream, the peptide attaches to cancer cells or cancer-associated stromal cells, delivering a high dose of radiation to the tumor while sparing normal tissues.

About FAP-Targeted Radiopharmaceuticals

Clinical studies of small molecule imaging agents targeting FAP have validated this target in a diverse number of cancer indications and support the further evaluation of peptide-targeted radionuclide therapy. FAP-targeted radiopharmaceuticals have at least two potential modes of anti-tumor activity: radiation crossfire, in which tumor cells are irradiated due to their close proximity to CAFs; and depletion of CAFs, disrupting the communication between the tumor cells and the tumor stroma. In addition, in certain tumor types, such as sarcoma and mesothelioma, FAP is expressed on the tumor cells themselves, and in those tumors, FAP-targeted radiopharmaceuticals may have a direct antitumor effect.

BeiGene Announces Clinical Results on Tislelizumab Presented at the 22nd Annual Meeting of the Chinese Society of Clinical Oncology (CSCO)

On September 22, 2019 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported clinical results on its investigational anti-PD-1 antibody tislelizumab from three ongoing clinical trials in China (Press release, BeiGene, SEP 22, 2019, View Source [SID1234539708]). These new or updated data were presented in five of the seven oral presentations on tislelizumab at the 22nd Annual Meeting of the Chinese Society of Clinical Oncology (CSCO), taking place September 18-22, 2019 in Xiamen, China. Additional BeiGene clinical data being presented at CSCO include four poster presentations on tislelizumab, zanubrutinib, and pamiparib.

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"Taken together these data show the potential for tislelizumab to benefit patients across a number of indications where we see unmet need in China and around the world," said Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene. "In anticipation of our first regulatory approvals in China for tislelizumab in classical Hodgkin’s lymphoma and urothelial carcinoma, we are nearing the first stage of completion on our state-of-the art biologics manufacturing facility, which we expect will be a model for quality biologics manufacturing operations at a global scale."

Clinical Results from a Phase 2 Trial of Tislelizumab Plus Chemotherapy as First-Line Treatment for Patients with Lung Cancer

This open-label, multi-cohort, Phase 2 trial of tislelizumab in combination with chemotherapy as first-line treatment for patients with advanced lung cancer (clinicaltrials.gov identifier: NCT03432598) is being conducted in China.

Patients with non-squamous non-small cell lung cancer (NSCLC) were treated with tislelizumab at a dose of 200mg and doublet chemotherapy on day one of each three-week cycle; chemotherapy was given for up to four cycles, with pemetrexed and tislelizumab continued as scheduled if clinically appropriate. Patients with squamous NSCLC (two cohorts) and small cell lung cancer (SCLC) were treated with tislelizumab at a dose of 200mg and doublet chemotherapy every three weeks, for four-six cycles, with tislelizumab continued as scheduled if clinically appropriate.

As of February 25, 2019, 54 patients had received tislelizumab, with a median duration of treatment of 38.4 weeks (3-79). Fourteen patients remained on treatment as of the data cutoff. Results included:

The confirmed overall response rate (ORR) across all cohorts was 66.7% (n=36), with ORRs of 43.8% (7/16) in patients with non-squamous NSCLC; 80.0% (12/15) in patients with squamous NSCLC (cohort A); 66.7% (4/6) in patients with squamous NSCLC (cohort B); and 76.5% (13/17) in patients with SCLC;

Median progression-free survival (PFS) was measured at a later data cutoff on June 30, 2019, and was 9.0 months in patients with non-squamous NSCLC, 7.0 months in squamous NSCLC (cohort A), 6.9 months in SCLC, and in squamous NSCLC (cohort B) the median PFS had not yet been reached;

At the median follow-up of 15.3 months, overall survival (OS) in patients with SCLC was 15.6 months; OS in other cohorts had not yet been reached;

Treatment-emergent adverse events (TEAEs) occurred in all 54 patients; adverse events (AEs) reported as related to tislelizumab occurred in 46 patients (85.2%), and seven patients (13%) discontinued tislelizumab treatment due to AEs;

Grade ≥3 TEAEs occurred in 43 patients, with the most common being decreased neutrophil count (48.1%), anemia (18.5%), decreased white blood cell count (13%), decreased platelet count (13%), thrombocytopenia (11.1%), neutropenia (7.4%), and increased alanine aminotransferase (ALT; 5.6%).

A total of 14 patients (25.9%) experienced at least one immune-related adverse event (irAE), with the most common being thyroid disorders (16.7%), immune-mediated pneumonitis (7.4%), and immune-mediated hepatitis (3.7%);

The most common TEAEs of any grade reported to be related to tislelizumab included asthenia (18.5%); hypothyroidism (13%); decreased appetite (11.1%); increased ALT (11.1%); and increased aspartate aminotransferase (AST; 11.1%); and

Fourteen patients (25.9%) experienced at least one serious TEAE; one patient with squamous NSCLC (cohort A) had a fatal AE of immune-mediated myositis/rhabdomyolysis/cardiomyopathy after one dose of tislelizumab.

Updated Clinical Results from a Phase 2 Trial of Tislelizumab in Combination with Chemotherapy in Patients with ESCC

This open-label, multi-cohort, Phase 2 trial of tislelizumab in combination with chemotherapy as first-line treatment for patients with advanced esophageal, gastric or gastroesophageal junction carcinoma (clinicaltrials.gov identifier: NCT03469557) is being conducted in China. Updated results from the ESCC cohort were reported in an oral presentation at CSCO.

Patients were treated with tislelizumab at a dose of 200mg and cisplatin on day one, and fluorouracil (5-FU) on days one through five during each 21-day cycle. At the time of data cutoff on March 31, 2019, 15 patients with ESCC had received treatment with tislelizumab, and four remained on treatment. Results included:

As of the data cutoff, seven patients achieved a confirmed partial response (PR) and the ORR was 46.7%;

Median duration of response (DoR) was 12.8 months; median PFS was 10.4 months (5.6-15.1);

Despite a median follow-up of 13.0 months, median OS had not been reached;

AEs reported in this cohort were consistent with the safety profile of tislelizumab observed in previous studies with other tumor types and were generally of low severity; AEs reported in this cohort were consistent with the known tolerability profile of PD-1 inhibitors in combination with chemotherapy;

The most common TEAEs were anemia (n=12) and decreased appetite (n=11);

Five patients discontinued tislelizumab treatment due to TEAEs, including pneumonitis, tracheal fistula, increased AST, lung infection, and autoimmune dermatitis (n=1, each);

Twelve patients (80%) experienced 23 irAEs, with the most common being rash (20%), pruritis (20%), increased AST (13.3%), increased ALT (13.3%), lung infection (13.3%), and autoimmune dermatitis (13.3%). The majority of irAEs were mild to moderate in severity; five grade ≥3 irAEs were reported in four patients (26.7%) and lung infection was the only irAE of grade ≥3 occurring in two patients (13.3%);

The most common AEs of any grade reported to be related to tislelizumab included decreased appetite (66.7%), anemia (60%), nausea (40%), leukopenia (40%), decreased neutrophil count (40%), vomiting (33.3%), decreased weight (33.3%), decreased white blood cell count (33.3%), asthenia (33.3%), hypoalbuminemia (33.3%), and hyponatremia (33.3%);

The most common grade > 3 AEs considered related to treatment with tislelizumab included vomiting (20%), hyponatremia (20%); anemia (13.3%); and leukopenia (13.3%);

The only serious TEAE of any attribution occurring in more than one patient were dysphagia (n=3) and fatigue (n=2); one case of each was considered possibly related to tislelizumab; and

One patient experienced a fatal AE of hepatic dysfunction, which was considered mainly related to progressive disease and also possibly related to study treatment or underlying type B hepatitis.

Clinical Results of Tislelizumab from a Phase 1/2 Trial in Patients with Advanced Solid Tumors

This multi-center, open-label Phase 1/2 trial of tislelizumab as monotherapy in patients with advanced solid tumors (chinadrugtrials.org registration number: CTR20160872) is being conducted in China and consists of a Phase 1 dose verification and pharmacokinetics component, and a Phase 2 indication expansion in disease-specific cohorts, including patients with non-small cell lung cancer (NSCLC), melanoma, urothelial carcinoma (UC), renal cell carcinoma (RCC), esophageal squamous cell carcinoma (ESCC), gastric cancer (GC), hepatocellular carcinoma (HCC), and microsatellite instability-high/mismatch repair deficient (MSI-H/dMMR) solid tumors.

As of December 1, 2018, 300 patients across all indications had been treated in this study with tislelizumab at a dose of 200mg every three weeks.

Safety Profile in All Indications (N=300):

Tislelizumab was generally well tolerated among patients with advanced solid tumors;

Most treatment-related adverse events (TRAEs) were grade ≤2 in severity, with the most common being anemia (23%), increased AST (22%), increased ALT (20%), proteinuria (14%), increased blood bilirubin (13%), hypothyroidism (11%), decreased white blood cell count (11%), increased conjugated bilirubin (11%) and pyrexia (10%);

The most common grade ≥3 TRAEs were increased gammaglutamyl transferase (GGT) (4%), anemia (3%), and increased AST (3%);

One patient with GC experienced a fatal brain edema, which was considered possibly related to tislelizumab treatment by the investigator;

Most irAEs were grade ≤2 in severity, with the most common being increased AST/ALT (24%) and hyperbilirubinemia (15%); and

The most common grade ≥3 irAEs were increased GGT (4%) and increased AST/ALT (3%).

Efficacy Profile:

Cohort Patient Number
(all evaluable for response) Median Number of Prior Lines of Systemic Anti-Cancer Therapy Median Follow-up
(months) Responses Median OS
(months)
NSCLC 56 2 9 (0-19) ORR 18%

10 confirmed PRs

21 SDs

Not reached;

Median PFS was 4.0 (2.1-8.1)
Melanoma 34 2 8 (1-18) ORR 15%

5 confirmed PRs

8 SDs

11.3
UC 22 1 4.2 (1-22) ORR 14%

3 confirmed PRs

6 SDs

4.3
RCC 21 2 16 (3-18) ORR 10%

2 confirmed PRs

9 SDs

Not reached
ESCC 26 2 5 (2-19) ORR 8%

2 confirmed PRs

7 SDs

4.8
GC 24 2 6 (1-18) ORR 17%

4 confirmed PRs

3 SDs

4.7
HCC 18

All patients had Child-Pugh A liver status. Sixteen patients had hepatitis B virus infection, one had hepatitis C virus infection, and one patient was uninfected 1.5 8 (3-17) ORR 17%

3 confirmed PRs

7 SDs

Not reached
MSI-H/dMMR Solid Tumor 16 (all evaluable for response)

Among the 16 patients, 14 patients had colorectal carcinoma, one had GC and one had unknown primary tumor site 2 11 (2-17) ORR 19%

3 confirmed PRs

5 SDs

Not reached

About Tislelizumab

Tislelizumab (BGB-A317) is an investigational humanized IgG4 anti–PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug candidate produced from BeiGene’s immuno-oncology biologics program and is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.

Ongoing clinical trials of tislelizumab include a Phase 3 clinical trial in patients with second-line or third-line non-small cell lung cancer (NSCLC); a Phase 3 clinical trial in first-line patients with hepatocellular carcinoma (HCC); a Phase 3 clinical trial in second-line patients with esophageal squamous carcinoma (ESCC); a Phase 3 clinical trial in first-line patients with gastric cancer (GC); a Phase 3 clinical trial in first-line patients with ESCC; and a Phase 2 clinical trial in second- or third-line patients with HCC. The aforementioned trials are enrolling patients in multiple countries, including the United States, Europe, and China.

In addition to a pivotal Phase 2 clinical trial in patients with relapsed or refractory (R/R) classical Hodgkin’s lymphoma (cHL) and a pivotal Phase 2 clinical trial in patients with locally advanced or metastatic urothelial cancer, BeiGene is conducting a Phase 3 clinical trial in first-line patients with non-squamous NSCLC; a Phase 3 clinical trial in first-line patients with squamous NSCLC; a Phase 3 clinical trial in patients with first-line nasopharyngeal cancer (NPC); a Phase 3 clinical trial in first-line patients with urothelial carcinoma (UC); a Phase 3 clinical trial in patients with localized ESCC; and a Phase 2 trial in patients with MSI-H or dMMR solid tumors. These studies have been enrolling patients primarily in China.

New drug applications (NDA) for tislelizumab in patients with R/R cHL and in patients with previously treated locally advanced or metastatic UC have been accepted and granted priority review by the China National Medical Products Administration (NMPA, formerly known as CFDA). BeiGene has full development and commercial rights to tislelizumab worldwide.

2019 CSCO| CStone announces promising ORR, anti-tumor activity and safety data with its anti-PD-L1 antibody CS1001 in MSI-H/dMMR solid tumors

On September 21, 2019 CStone Pharmaceuticals ("CStone" or the "Company", HKEX: 2616) reported results from the microsatellite instability high/deficient mismatch repair (MSI-H/dMMR) solid tumor cohort in the GEMSTONE-101 Phase Ib study of the Company’s investigational anti-PD-L1 antibody CS1001 for the first time in an oral presentation at the 22nd Annual Meeting of the Chinese Society of Clinical Oncology (CSCO, 2019 CSCO Annual Meeting) (Press release, CStone Pharmaceauticals, SEP 21, 2019, View Source [SID1234539681]).

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The objectives of the MSI-H/dMMR cohort study were to assess CS1001’s preliminary anti-tumor efficacy as a second-line or later monotherapy, and to further evaluate the safety and tolerability of CS1001.

Microsatellites (MS) are repetitive genetic sequences consisting of repeated DNA motifs. Defective DNA mismatch repair pathway causes instability in the length of DNA motifs, resulting in a phenomenon that is called microsatellite instability (MSI). MSI‐H indicates a high level of MSI, which is predictive of the loss of mismatch repair function and the possible presence of a large amount of mutation-derived tumor antigens that produce good responses to immunotherapies. Published data suggests MSI-H/dMMR is most common in patients with endometrial cancer, gastric adenocarcinoma, malignant small intestine tumor, and colorectal adenocarcinoma.

Professor Lin Shen, Vice President of Beijing Cancer Hospital, and the presenter of the results, commented: "Preliminary efficacy data from this study cohort indicates CS1001’s promising anti-tumor activity in patients with MSI-H/dMMR solid tumors. Also shown in the results are CS1001’s good safety and tolerability."

Dr. Frank Jiang, Chairman and CEO of CStone, commented: "I am pleased that CS1001, our core PD-L1 drug candidate, has achieved favorable preliminary results in this MSI-H/dMMR study cohort. At present, no PD-L1 inhibitor has been approved worldwide for the treatment of this type of solid tumors. We hope in future studies CS1001 will demonstrate its therapeutic potential in more tumor types and become the most rapidly developed immuno-oncology drug for MSI-H/dMMR solid tumors."

CStone’s Chief Medical Officer, Dr. Jason Yang, noted: "In view of the good responses to immunotherapies in MSI-H/dMMR solid tumors, effective immuno-oncology treatments will hopefully bring survival benefits to such patient population. The results from this study has shown CS1001’s therapeutic potential, and its 38% overall response rate (ORR), with 28.6% confirmed, in these heavily pretreated patients is remarkable compared to some of the approved PD-1 drugs. CS1001 is the monoclonal antibody that most closely mirrors natural G-type immune globulin 4 (IgG4) human antibody, which hopefully can lead to the demonstration of its unique safety advantage in subsequent clinical studies."

Overview of the MSI‐H/dMMR cohort of the GEMSTONE-101 Phase Ib ≥2L study
This study cohort included unresectable or metastatic MSI‐H/dMMR solid tumor patients who failed to achieve satisfactory outcomes from prior treatments and lacked alternative treatment options. A total of 21 patients were enrolled in this cohort, 18 of whom had colorectal cancer, 2 with pancreatic cancer, and 1 with small intestine cancer. 13 of the enrolled patients had previously been treated with second line or later therapies. During the study, patients were administered with 1,200 mg CS1001 once every three weeks, until disease progression or intolerance.

Demographics and baseline characteristics

Of the 21 enrolled patients, 9 remained on treatment, 12 discontinued treatment.
Main reasons for discontinuation were disease progression (n=8), patient’s decision (n=2), death due to disease progression (n=1), and other reasons (n=1).
No patient discontinued due to AEs.
Preliminary efficacy data

CS1001 has shown promising anti-tumor activity in patients with MSI-H/dMMR solid tumors. 21 patients who received the treatment were included in efficacy analysis, and 8 (38.1%) of them achieved partial response (PR, per RECIST V1.1), 6 (28.6%) were confirmed PRs.
The disease control rate was 57.1% (12/21).
The duration of response (DOR) ranged from 0.03+ to 8.6+ months, and the median DOR was not reached.
Safety data

CS1001 has shown a good safety profile

The median treatment duration of the 21 treated patients was 137 days (21-377).
During treatment, 20 patients (95.9%) developed adverse events (AEs), and close to 1/4 of those patients had Grade 3 or higher AEs.
18 patients (85.7%) developed AEs that were related to the CS1001 treatment, and only 1 patient (4.8%) developed an AE that was Grade 3 or higher in severity.
2 patients (9.5%) developed serious adverse events (SAEs) and neither was related to the CS1001 treatment.
9 patients (42.9%) developed immune-related adverse events (irAEs) that were Grade 1-2 in severity.
No discontinuation or death occurred due to AEs.
About CS1001
CS1001 is an investigational monoclonal antibody directed against PD-L1 being developed by CStone. Authorized by the U.S. based Ligand Corporation, CS1001 is developed by the OMT transgenic animal platform, which can generate fully human antibodies in one step. As a fully human, full-length anti-PD-L1 monoclonal antibody, CS1001 mirrors natural G-type immune globulin 4 (IgG4) human antibody, which can reduce the risk of immunogenicity and potential toxicities in patients, potentially representing a unique advantage over similar drugs.

CS1001 has completed a Phase I dose-escalation study in China, in which CS1001 showed good tolerability and produced sustained clinical benefits during the Phase Ia stage of the study.

CS1001 is being investigated in a number of ongoing clinical trials, including one Phase I bridging study in the U.S. In China, its clinical program includes one multi-arm Phase Ib study, two pivotal Phase II studies, and three Phase III studies for several tumor types.