Positive opinions based on significant survival benefit in HIMALAYA and POSEIDON Phase III trials

On December 19, 2022 AstraZeneca reported that Imfinzi (durvalumab) and Imjudo (tremelimumab) combinations have been recommended for marketing authorisation in the European Union (EU) for advanced liver and lung cancers (Press release, AstraZeneca, DEC 19, 2022, View Source [SID1234625379]).

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The concurrent positive opinions recommend authorising Imfinzi in combination with Imjudo for 1st-line treatment of adult patients with advanced or unresectable hepatocellular carcinoma (HCC); and Imfinzi in combination with Imjudo and platinum-based chemotherapy for the treatment of adult patients with Stage IV (metastatic) non-small cell lung cancer (NSCLC).

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinions on results from the HIMALAYA Phase III trial, which was published in the New England Journal of Medicine Evidence, and results from the POSEIDON Phase III trial, which was published in the Journal of Clinical Oncology.

Bruno Sangro, MD, PhD, Director of the Liver Unit at Clínica Universidad de Navarra, Professor of Internal Medicine at the University of Navarra School of Medicine and a lead investigator in the HIMALAYA trial, said: "Liver cancer is a leading cause of cancer death in Europe, and patients with advanced disease face an especially grim prognosis with limited treatment options in the 1st-line setting. The combination of Imjudo and Imfinzi demonstrated a meaningful improvement in overall survival with no increase in severe liver toxicity or bleeding risk, which are important considerations for these patients who often have advanced disease."

Solange Peters, MD, PhD, President of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper), head of the medical oncology service and chair of thoracic oncology at Hospitalier Universitaire Vaudois, Lausanne, Switzerland, and principal investigator in the POSEIDON trial, said: "Metastatic non-small cell lung cancer remains a complex and devastating diagnosis and there is still an urgent need for new life-extending treatment options. The latest data from the POSEIDON trial demonstrate the long-term survival benefit of Imjudo added to Imfinzi and chemotherapy and support the important role this novel combination could have for patients with metastatic non-small cell lung cancer in Europe."

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: "Patients in Europe diagnosed with these advanced cancers urgently need treatment combinations that can help them live longer. If approved, these Imjudo and Imfinzi combinations will provide patients with novel options that harness the potential long-term survival benefits seen with CTLA-4 inhibition."

Imjudo and Imfinzi in liver cancer

The CHMP positive opinion for the treatment of HCC is based on results from the HIMALAYA Phase III trial, in which a single dose of the anti-CTLA-4 antibody Imjudo 300mg added to the anti-PD-L1 antibody Imfinzi 1500mg followed by Imfinzi every four weeks (STRIDE regimen) reduced the risk of death by 22% versus sorafenib (hazard ratio [HR] 0.78; 95% confidence interval [CI], 0.66-0.92; p= 0.0035). An estimated 31% of patients treated with the combination were still alive after three years, with 20% of patients treated with sorafenib still alive at the same duration of follow-up.

The safety profiles of the combination of Imjudo added to Imfinzi and for Imfinzi alone were consistent with the known profiles of each medicine, and no new safety signals were identified.

Liver cancer is the third-leading cause of cancer death and the sixth most commonly diagnosed cancer worldwide.1,2 Approximately 87,000 Europeans were diagnosed with liver cancer in 2020, with 51% of patients at an advanced cancer stage at time of diagnosis. Rates of liver cancer continue to rise rapidly, with a 70% increase of liver cancer-related mortality in the EU from 1990-2019.3

Imjudo and Imfinzi in lung cancer

The CHMP positive opinion for the treatment of metastatic NSCLC is based on results from the POSEIDON Phase III trial, which showed patients treated with a limited course of five cycles of the anti-CTLA-4 antibody Imjudo added to Imfinzi plus four cycles of platinum-based chemotherapy experienced a 23% reduction in the risk of death versus a range of chemotherapy options (HR 0.77; 95% CI, 0.65-0.92; p=0.00304). An estimated 33% of patients were alive at two years versus 22% for chemotherapy. This treatment combination also reduced the risk of disease progression or death by 28% compared to chemotherapy alone (HR 0.72; 95% CI, 0.60-0.86; p=0.00031).

Updated results from the POSEIDON Phase III trial after approximately four years of follow-up presented at the European Society for Medical Oncology Congress 2022 demonstrated sustained survival benefit, improving overall survival (OS) by 25% compared to chemotherapy alone (HR 0.75; 95% CI, 0.63-0.88). An estimated 25% of patients treated with the combination were alive at three years versus 13.6% for those treated with chemotherapy alone.

The safety profile for Imjudo plus Imfinzi and chemotherapy was consistent with the known profiles of each medicine, and no new safety signals were identified.

Stage IV is the most advanced form of lung cancer and is often referred to as metastatic disease.4,5 Approximately 40% of people with NSCLC have Stage IV disease at the time of diagnosis.6 Almost 5% of patients with metastatic NSCLC in England will survive five years after diagnosis, based on data between 2013-2017.7

In October 2022, Imjudo plus Imfinzi was approved in the US for the treatment of adults with unresectable HCC. Imjudo plus Imfinzi in combination with platinum-based chemotherapy was approved in the US in November 2022 for the treatment of adults with metastatic NSCLC. Regulatory applications for both indications are also currently under review in several other countries based on the HIMALAYA and POSEIDON results, respectively.

Notes

Liver cancer
About 75% of all primary liver cancers in adults are HCC.1 Between 80-90% of all patients with HCC also have cirrhosis.8 Chronic liver diseases are associated with inflammation that over time can lead to the development of HCC.8

More than half of patients are diagnosed at advanced stages of the disease, often when symptoms first appear.9 A critical unmet need exists for patients with HCC who face limited treatment options.9 The unique immune environment of liver cancer provides clear rationale for investigating medications that harness the power of the immune system to treat HCC.9

Stage IV NSCLC
Lung cancer is the second most common form of cancer globally, with more than two million patients diagnosed in 2020.10 Lung cancer is broadly split into NSCLC and small-cell lung cancer (SCLC), with 80-85% classified as NSCLC. Within NSCLC, patients are classified as squamous, representing 25-30% of patients, or non-squamous, representing approximately 70-75% of patients.4, 11-12

HIMALAYA
HIMALAYA was a randomised, open-label, multicentre, global Phase III trial of Imfinzi monotherapy and a regimen comprising a single priming dose of Imjudo 300mg added to Imfinzi 1500mg followed by Imfinzi every four weeks versus sorafenib, a standard-of-care multi-kinase inhibitor.

The trial included a total of 1,324 patients with unresectable, advanced HCC who had not been treated with prior systemic therapy and were not eligible for locoregional therapy (treatment localised to the liver and surrounding tissue).

The trial was conducted in 181 centres across 16 countries, including in the US, Canada, Europe, South America and Asia. The primary endpoint was OS for the combination versus sorafenib and key secondary endpoints included OS for Imfinzi versus sorafenib, objective response rate and progression-free survival (PFS) for the combination and for Imfinzi alone.

POSEIDON
The POSEIDON trial was a randomised, open-label, multi-centre, global, Phase III trial of Imfinzi plus platinum-based chemotherapy or Imfinzi, tremelimumab and chemotherapy versus chemotherapy alone in the 1st-line treatment of 1,013 patients with metastatic NSCLC. The trial population included patients with either non-squamous or squamous disease and the full range of PD-L1 expression levels. POSEIDON excluded patients with certain epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) fusions.

In the experimental arms, patients were treated with a flat dose of either Imfinzi (1,500mg) or Imfinzi plus Imjudo (75mg) with up to four cycles of chemotherapy every three weeks before either Imfinzi maintenance once every four weeks or Imfinzi and a fifth dose of Imjudo given at week 16. In comparison, the control arm allowed up to six cycles of chemotherapy. Pemetrexed maintenance treatment was allowed in all arms in patients with non-squamous disease if given during the induction phase. Nearly all patients with non-squamous disease (95.5%) had pemetrexed and platinum, while the majority of patients with squamous disease receiving chemotherapy (88.3%) received gemcitabine and platinum.

Primary endpoints included PFS and OS for the Imfinzi plus chemotherapy arm. Key secondary endpoints included PFS and OS in the Imfinzi plus Imjudo and chemotherapy arm. As PFS endpoints were met for both experimental arms, the prespecified statistical analysis plan allowed for testing OS in the Imfinzi plus Imjudo and chemotherapy arm. The OS trend observed in the Imfinzi plus chemotherapy arm did not achieve statistical significance. The trial was conducted in more than 150 centres across 18 countries, including the US, Europe, South America, Asia and South Africa.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

Imfinzi is the only approved immunotherapy in the curative-intent setting of unresectable, Stage III NSCLC in patients whose disease has not progressed after chemoradiotherapy and is the global standard of care in this setting based on the PACIFIC Phase III trial.

Imfinzi is also approved in the US, EU, Japan, China and many other countries around the world for the treatment of extensive-stage SCLC based on the CASPIAN Phase III trial. In an exploratory analysis in 2021, updated results from the CASPIAN trial showed Imfinzi plus chemotherapy tripled patient survival at three years versus chemotherapy alone.

Imfinzi is also approved in combination with Imjudo and chemotherapy in metastatic NSCLC in the US; in combination with chemotherapy in locally advanced or metastatic biliary tract cancer (BTC) in the US and several other countries, in combination with Imjudo in unresectable HCC in the US and in previously treated patients with advanced bladder cancer in several countries.

Since the first approval in May 2017, more than 100,000 patients have been treated with Imfinzi.

As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, bladder cancer, several gastrointestinal (GI) cancers, ovarian cancer, endometrial cancer and other solid tumours. 

Imjudo
Imjudo (tremelimumab) is a human monoclonal antibody that targets the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4). Imjudo blocks the activity of CTLA-4, contributing to T-cell activation, priming the immune response to cancer and fostering cancer cell death.

In addition to its approved indications in liver and lung cancers, Imjudo is being tested in combination with Imfinzi across multiple tumour types including locoregional HCC (EMERALD-3), SCLC (ADRIATIC) and bladder cancer (VOLGA and NILE).

Update on PEARL Phase III trial of Imfinzi monotherapy in Stage IV non-small cell lung cancer

On December 19, 2022 Astrazeneca reported that The PEARL Phase III trial for Imfinzi (durvalumab) did not achieve statistical significance for the primary endpoints of improving overall survival (OS) versus platinum-based chemotherapy as a monotherapy treatment of patients with Stage IV (metastatic) non-small cell lung cancer (NSCLC) whose tumour cells express high levels (25% or more) of PD-L1, or in a subgroup of patients at low risk of early mortality (Press release, AstraZeneca, DEC 19, 2022, View Source [SID1234625376]). There was an improvement in OS with Imfinzi monotherapy, which was clinically meaningful in the subset of patients with PD-L1 tumour expression greater than 50%, a secondary endpoint. The trial was conducted primarily in Asia.

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Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: "With PEARL, we set out to answer important scientific questions in the treatment of metastatic non-small cell lung cancer at a time when patient selection for immune checkpoint inhibitors was still evolving. We are encouraged to see patients in the metastatic setting at a higher level of PD-L1 tumour expression demonstrate the most benefit with Imfinzi monotherapy treatment, as is commonly seen in this class. We remain steadfast in our dedication to developing new and improved medicines and regimens for patients with lung cancer across our diverse portfolio."

The safety and tolerability profile for Imfinzi was broadly consistent with the known profile of the medicine, and no new safety signals were identified. The data will be shared in due course.

Notes

Stage IV NSCLC
Lung cancer is the second most common form of cancer globally, with more than two million patients diagnosed in 2020.1 Lung cancer is broadly split into NSCLC and small-cell lung cancer (SCLC), with 80-85% classified as NSCLC.2,3 Within NSCLC, patients are classified as squamous, representing 25-30% of patients, or non-squamous, representing approximately 70-75% of patients.3-5

PEARL
PEARL was a randomised, open-label, multicentre, global Phase III trial of Imfinzi monotherapy versus platinum-based chemotherapy (investigator’s choice) as a 1st-line treatment in patients with metastatic NSCLC.

Eligible patients had tumours expressing high levels of PD-L1, defined as ≥25% of tumour cells (TC). The predominantly Asian trial population included both smokers and non-smokers and patients of squamous and non-squamous histology. PEARL excluded patients with certain epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) fusions.

The two primary endpoints were OS in patients whose tumours expressed high levels of PD-L1 (TC≥25%) and OS in a subgroup of patients identified as being at low risk of early mortality using a model developed by AstraZeneca that evaluates various clinical parameters prior to treatment. The trial included centres in Asia, Europe and Australia.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

Imfinzi in combination with Imjudo (tremelimumab) plus platinum-based chemotherapy is approved in the US for the treatment of Stage IV (metastatic) NSCLC based on the POSEIDON Phase III trial. Additionally, Imfinzi is the only approved immunotherapy and the global standard of care in the curative-intent setting of unresectable, Stage III NSCLC in patients whose disease has not progressed after chemoradiation therapy based on the PACIFIC Phase III trial. Imfinzi is also approved in the US, the EU, Japan, China and many other countries around the world for the treatment of extensive-stage small cell lung cancer based on the CASPIAN Phase III trial.

In addition to its approved indications in lung cancer, Imfinzi is the only approved immunotherapy in unresectable or metastatic biliary tract cancer and is also approved in unresectable hepatocellular carcinoma in combination with Imjudo. It is also approved for previously treated patients with advanced bladder cancer in several countries.

As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, bladder cancer, several gastrointestinal cancers, ovarian cancer, endometrial cancer, and other solid tumours.

Press Release: Sanofi and Innate Pharma expand collaboration for natural killer cell therapeutics in oncology

On December 19, 2022 Sanofi (NASDAQ: SNY) and Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) reported an expansion of their collaboration, with Sanofi licensing a natural killer (NK) cell engager program targeting B7H3 from Innate’s ANKETTM (Antibody-based NK Cell Engager Therapeutics) platform (Press release, Sanofi, DEC 19, 2022, View Source [SID1234625375]). Sanofi will also have the option to add up to two additional ANKETTM targets. Upon candidate selection, Sanofi will be responsible for all development, manufacturing and commercialization. Innate and Sanofi signed a first NK cell engagers collaboration in 2016 for the generation and evaluation of up to two bispecific NK cell engagers, which are currently being evaluated by Sanofi’s R&D team, with one of these molecules already in clinical studies.
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Valeria Fantin, Ph.D.
Global Head of Oncology Research at Sanofi
"At Sanofi, we are exploring the potential of NK cells for cancer immunotherapy, a key pillar for our oncology strategy. Our relationship with Innate aligns with our commitment to work with promising French companies and supports our ambition to develop a diverse portfolio of next-generation NK cell engagers, highly synergistic with Sanofi’s allogeneic NK cell platform, engineered lymphokines that stimulate NK cells, and growing Immuno-oncology pipeline. As a leading global company with roots in France, we are proud to collaborate to support the French healthcare ecosystem."

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Yannis Morel, Ph.D.
Executive Vice President, Product portfolio strategy & Business development at Innate Pharma
"Building on the success of our existing collaboration on hematologic targets, we are pleased to expand and strengthen our partnership with Sanofi on NK Cell Engagers with the addition of up to three new programs, including in solid tumors. Sanofi’s investment in Innate further validate the value of our ANKETTM platform and its potential to address multiple tumor types. By incorporating various tumor antigen binders, NK Cell Engagers are a versatile technology that may provide new options for patients and offer clinical benefit across multiple cancers, whilst also maintaining a good safety profile. This agreement also highlights Innate’s strategy to build a broad portfolio of ANKET programs addressing different types of cancer."

Under the terms of the new license agreement, Innate will receive €25m upfront payment and up to €1.35bn total in preclinical, clinical, regulatory and commercial milestones plus royalties on potential net sales. Closing of the transaction is subject to HSR approval.

About 2016 Sanofi/Innate research collaboration and licensing agreement
In 2016, Sanofi and Innate entered into a research collaboration and licensing agreement for the generation and evaluation of up to two bispecific NK cell engagers, using technology from Innate Pharma and Sanofi’s proprietary bispecific antibody format as well as tumor targets.

A Phase 1/2 clinical trial by Sanofi is ongoing, evaluating IPH6101/SAR’579 (SAR443579), the first NKp46/CD16-based CD123-targeted ANKETTM NK cell engager, in patients with relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL) or high-risk myelodysplastic syndrome (HR-MDS).

In the summer 2022, Sanofi had made the decision to progress IPH6401/SAR’514 (SAR445514) into investigational new drug (IND)-enabling studies. IPH6401/SAR’514 is a BCMA-targeting NK cell engager using Sanofi’s proprietary CROSSODILE multi-functional platform, which comprises the Cross-Over-Dual-Variable-Domain (CODV) format. It induces a dual targeting of the NK activating receptors, NKp46 and CD16, for an optimized NK cell activation, based on Innate’s ANKETTM proprietary platform.

Under the terms of the original license agreement, Sanofi is responsible for the development, manufacturing and commercialization of products resulting from the research collaboration. Innate Pharma will be eligible to up to €400m in development and commercial milestone payments as well as royalties on net sales. To date, €13m milestone payments to Innate have been announced.

Nykode Therapeutics and Richter-Helm BioLogics Announce Strategic Manufacturing Partnership

On December 19, 2022 Nykode Therapeutics ASA (OSE: NYKD), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel immunotherapies, reported that it has entered into a strategic manufacturing partnership with Richter-Helm BioLogics GmbH & Co KG to supply plasmid DNA for Nykode’s wholly owned and partnered product portfolio (Press release, Nykode Therapeutics, DEC 19, 2022, View Source [SID1234625374]).

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"We are excited about the strategic manufacturing partnership with Richter-Helm BioLogics. As a leading manufacturer of DNA vaccines, they will provide the long-term expertise and capacity needed to support our ambitious growth and pipeline development," stated Mette Husbyn, Chief Technology Officer of Nykode Therapeutics.

"We are thrilled to collaborate with Nykode Therapeutics and to support the development of their leading DNA vaccine platform. After 30 years of manufacturing plasmid DNA, we have gained an unparalleled amount of first-hand plasmid DNA process knowledge and experience working with regulatory agencies to ensure cGMP compliance for clinical trials and late-stage product candidates," said Dr. Kai Pohlmeyer, Managing Director of Richter-Helm BioLogics.

Manufacturing is a strategic focus area for Nykode and its license partners. Under the partnership agreement with Richter-Helm BioLogics, Nykode will have the option to tech transfer the proprietary manufacturing processes to its partners, if requested.

Biocytogen Announces FDA Clearance of IND Application for Bispecific Antibody YH008

On December 18, 2022 Biocytogen Pharmaceuticals (Beijing) Co., Ltd. ("Biocytogen", HKEX: 02315) reported that the US FDA has approved the Investigational New Drug (IND) application for a phase I study of YH008, an internally developed first-in-class PD-1 x CD40 bispecific antibody (bsAb) (Press release, Biocytogen, DEC 18, 2022, View Source [SID1234625367]). The IND application was completed by Biocytogen’s wholly owned subsidiary Eucure Biopharma.

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The trial is an open-label, dose-escalation study (No. YH008101) that will evaluate the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of YH008 monotherapy in patients with PD-(L)1-resistant advanced solid tumors or hematological malignancies.

YH008 exerts antagonistic and agonistic activities on PD-1 and CD40, respectively. In vitro and in vivo studies indicate that activation of the CD40 signaling pathway is dependent on PD-1 expression. Therefore, YH008 can conditionally activate CD40 pathway in the tumor microenvironment where tumor specific PD-1+ T cells are enriched, without systemic CD40 non-specific activation. Additionally, YH008 was engineered with an Fc-silent IgG1 isotype to avoid Fc-receptor-mediated non-specific immune activation. YH008 demonstrated superior anti-tumor activity than parental monoclonal antibodies (mAbs) or combination therapy in vivo. Moreover, the anti-tumor activity of YH008 was also superior to benchmark PD-1 mAbs and PD-L1 x CD40 bsAbs in syngeneic models. In vivo pharmacodynamic studies indicate that YH008 can activate tumor-infiltrating DCs and T cells. In addition, both in vivo studies and GLP toxicology studies indicate improved safety of YH008 compared with benchmark CD40 mAbs.

"The CD40 agonistic activity of YH008 is PD-1-dependent, which allows for more targeted immune cell activation and synergies," said Dr. Yi Yang, Chief Scientific Officer (CSO) of Biocytogen. "These characteristics prevent occurrence of liver toxicity, even at high doses, while enhancing anti-tumor activity, giving YH008 high clinical potential."

"YH008 is a first-in-class bispecific antibody discovered through large-scale in vivo efficacy screening, where it demonstrated excellent anti-tumor activity, even in cold tumors," said Dr. Rong Chen, CEO and CMO of Eucure Biopharma, VP of Biocytogen. "With this IND clearance for YH008, the company will have more products with diversified modalities entering the clinic to benefit patients."