On September 20, 2022 Shanghai Junshi Biosciences Co., Ltd ("Junshi Biosciences", HKEX: 1877; SSE: 688180), a leading innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies, reported that the China National Medical Products Administration ("NMPA") has approved the supplemental new drug application ("sNDA") for toripalimab in combination with pemetrexed and platinum as the first-line treatment in EGFR mutation-negative and ALK mutation-negative, unresectable, locally advanced or metastatic non-squamous non-small cell lung cancer ("NSCLC"). This is the sixth indication approved for toripalimab in China and will bring more treatment options to Chinese patients with advanced NSCLC.

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"We are very pleased that toripalimab’s first indication for the treatment of lung cancer has been approved, which means we will be able to help more patients fight against malignant tumors with the highest incidence and mortality rates in China," said Dr. Jianjun Zou, Global Research and Development President at Junshi Biosciences. "We are particularly grateful to our patients, researchers, and R&D teams that participated in the clinical trials, whose efforts and contributions allowed our study to progress during the COVID-19 pandemic. We will continue to advocate for the use of toripalimab in the treatment of lung cancer as well as other cancers, and we hope to provide many more patients with better treatment options and greater survival benefits as soon as possible!"

Professor Jie Wang from the Cancer Hospital, Chinese Academy of Medical Sciences elaborated on toripalimab’s performance in clinical trials. "In China, the number of lung cancer patients is massive and so is the demand for treatment. The CHOICE-01 study provided us with reliable and substantiating data, confirming that the addition of toripalimab to standard first-line chemotherapy can bring longer progression-free survival ("PFS") and overall survival ("OS") to patients with advanced NSCLC, regardless of PD-L1 expression and with a manageable safety profile. For patients with advanced non-squamous NSCLC, published data reveal that the 2-year OS rate is around 50%, and according to the CHOICE-01 study results, toripalimab in combination with chemotherapy is the only PD-(L)1 inhibitor combination in this field that can achieve a 2-year OS rate of more than 60%, not to mention a 50% decrease in risk of death, indicating that toripalimab plus standard chemotherapy can significantly increase survival benefits for patients. We are excited for toripalimab’s continuous contribution to the battle against lung cancer!"

Between April 2, 2019 to August 5, 2020, the CHOICE-01 study enrolled a total of 465 NSCLC patients in 63 centers in China, among which 245 non-squamous NSCLC patients were randomly allocated in the ratio of 2:1 to receive toripalimab/placebo in combination with pemetrexed and cisplatin/carboplatin. After disease progression, eligible control subjects could receive crossover treatment with toripalimab monotherapy. Previously, the latest research results of the CHOICE-01 study were announced at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Plenary Series March Program and the ASCO (Free ASCO Whitepaper) annual meeting. The study data showed that compared to chemotherapy alone, toripalimab in combination with chemotherapy in the first-line treatment of patients with advanced NSCLC without EGFR/ALK mutations can significantly improve the PFS and the OS of patients with a manageable safety profile regardless of PD-L1 expression status.

As of October 31, 2021, in 245 non-squamous NSCLC patients, the median PFS of toripalimab in combination with chemotherapy was 9.7 months, which was 4.2 months longer than placebo in combination with chemotherapy (HR = 0.48 [95% CI: 0.35-0.66], p < 0.0001); the median OS of toripalimab in combination with the chemotherapy group has yet to be reached, while OS benefits had already been observed, reducing the risk of death by 52% (HR=0.48 [95%CI: 0.32-0.71]).

About NSCLC
Lung cancer is currently the second most prevalent malignant tumor with the highest mortality rate in the world[1], and the most prevalent with the highest mortality rate in China[2]. According to data released by the World Health Organization, in 2020, the number of new lung cancer cases in China amounted to 816,000 and accounted for 17.9% of all new cancer cases in China[2]. In the same year, the number of lung cancer deaths in China amounted to 715,000 and accounted for 23.8% of all cancer deaths in China[2]. NSCLC is a major subtype of lung cancer, accounting for approximately 85% of all cases[3]. Non-squamous NSCLC patients account for approximately 70% of all NSCLC patients[4]. Existing domestic and overseas studies have shown that the use of anti-PD-(L)1 monoclonal antibodies as monotherapy or in combination with chemotherapy has already become new standard for the first-line treatment of advanced driver gene-negative NSCLC.

[1] View Source
[2] View Source
[3] Rosell R, Karachaliou N. Large-scale screening for somatic mutations in lung cancer. Lancet, 2016, 387(10026): 1354-1356. doi: 10.1016/S0140-6736(15)01125-3
[4] Gridelli C, et al. Nat Rev Dis Primers. 2015;1:15009

About Toripalimab
Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and for enhanced receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 promotes the immune system’s ability to attack and kill tumor cells. In China, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing in 2018 (approved in China as TUOYI). Currently, there are six approved indications for toripalimab in China.

(Press release, Shanghai Junshi Bioscience, SEP 20, 2022, View Source [SID1234663439])

On September 20, 2022 Affirma Biotech reported the closing of its seed capital round with a total of 600,000 Euros that the company will invest in the optimization of its preclinical candidates. During the past year a total amount of 960.000 Euros in funding has been raised by the company from several business angels with extensive experience in the biotech industry. The lead investors of this round are Alfons Hidalgo (founder of Infinitec), Luis Ruiz Avila and Reig Jofre Investments among others. Affirma Biotech also has Start-up Capital grant from Acció.

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The company discovered a new family of orally administered drugs that are currently being optimized and that will be developed as new antivirals for the treatment of chronic hepatitis B virus infection, as well as other unresolved infections. Chronic hepatitis B is currently the most prevalent viral infection worldwide. More than 290 million patients are chronically infected with this virus that is currently the main cause of liver cancer, one of those with the worst prognosis. The burden of the disease is estimated at approximately 1 million deaths each year.

Maribel Berges, founder and CEO of the company, emphasizes the importance of developing new therapeutic tools against viral infections «that will contribute to solve these increasingly urgent global sanitary challenges. We think that our innovative approach will be key to improve the treatment of diseases that today do not have a complete medical solution». The company’s goal is to start clinical trials within the next two years.

(Press release, Affirma Biotech, SEP 20, 2022, View Source [SID1234662179])

On September 20, 2022 Integra Therapeutics reported the company has been awarded €1-million grant from Eurostars, the largest funding programme of the European Commission that supports innovative SMEs in developing collaborative transnational projects geared towards the market (Press release, Integra Therapeutics, SEP 20, 2022, View Source [SID1234654531]). The company applied for the call as part of a consortium with Caszyme, a Lithuanian company that specialises in CRISPR technology and molecular tools development and application.

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The funding of the project submitted by Integra Therapeutics and Caszyme will go towards discovery, characterisation and execution of in vivo studies on the 2.0 version of the Integra Therapeutics FiCAT gene-writing platform.

The key difference between FiCAT 1.0 and FiCAT 2.0 will be the addition of novel nucleases discovered by Caszyme, which have different characteristics from the Cas9 nucleases and expand their potential uses in advanced therapies. During the execution of the project, Integra and Caszyme will enhance the functionality of novel nucleases in human cells.

The partnership between Integra Therapeutics and Caszyme will unite scientific know-how and technology from top academic and industrial biomedical research. The founders of Caszyme are Prof. Virginijus Šikšnys, Dr. Giedrius Gasiūnas and Dr. Monika Paule, the first scientists to prove that CRISPR-Cas9 can be used to program DNA double-strand breaks in a genome of interest, opening a new era in gene editing.

The project is scheduled to kick off on the 1st of November 2022 and finish at the end of 2025.

The Eurostar programme is part of Horizon Europe and is managed by Eureka with a network of national coordinators from the 37 participating countries, which from Spain is the Centre for the Development of Industrial Technology (CDTI).

On September 20, 2022 GenScript USA Inc., the world’s leading life-science research tools and services provider and Avectas, reported a cell engineering technology leader, are partnering to develop an improved non-viral cell therapy manufacturing process (Press release, Avectas, SEP 20, 2022, View Source [SID1234626218]). The two companies share a goal of providing their customers with potent, new methods for developing cell therapies that offer an improved safety profile over viral and non-viral vector techniques.

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By combining Avectas’ cell engineering technology and know-how with GenScript’s expertise in synthetic long oligo production, the partnership aims to demonstrate a novel and efficient solution for cell therapy manufacturing and to improve editing efficiency and cell viability over traditional delivery methods.

CRISPR-based non-viral gene editing methods have gained popularity among research teams following concerns about the FDA’s recent draft guidance on the use of viruses for gene and cell therapy. GenScript is collaborating with both academic and industry partners in the development of CRISPR non-viral gene editing to enable next-generation gene- and cell-therapy R&D projects.

Intracellular delivery is integral to the generation of engineered ex vivo cell-based therapies, including genome editing approaches. But the limitations of current delivery modalities, both viral and non-viral, led Avectas to develop the non-viral solupore cell engineering technology, which enables efficient delivery of cargoes into cells for the development of next-generation therapies.

The research teams will apply solupore technology to permeabilize the target cell membrane so that engineered cargoes can be delivered while retaining very high levels of cell viability and functionality. GenCRISPR synthetic sgRNA and Cas9 protein are then complexed into a ribonucleic protein that is co-delivered with GenExact ssDNA HDR templates into the cell nucleus.

"GenScript is excited to partner with Avectas as part of our program to develop novel RNP and oligo delivery systems for non-viral cell engineering," said Ray Chen, PhD, president of GenScript USA Life Science Group. "We expect this method will provide our customers with more complete solutions for efficient gene editing using our GenCRISPR sgRNA and ss/dsDNA HDR templates."

"Avectas is delighted to partner with GenScript to combine its innovative editing tools together with our solupore delivery platform," said Michael Maguire, PhD, CEO of Avectas. "This will enable the development of next-generation cell therapies differentiated by the quality of the modified cells, which retain high viability, functionality, and post-process proliferation."

On September 20, 2022 Basilea Pharmaceutica Ltd, a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, reported that it has entered into an asset purchase agreement and a sub-license agreement with SillaJen, Inc. for Basilea’s novel kinase inhibitor, BAL0891, a potential first-in-class mitotic checkpoint inhibitor, that drives aberrant tumor cell division leading to tumor cell death (Press release, Basilea Pharmaceutica, SEP 20, 2022, View Source [SID1234625345]).

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Basilea in-licensed BAL0891 in 2018 from the Dutch precision medicine company NTRC. Under the asset purchase agreement Basilea is selling its intellectual property rights generated under the license and collaboration agreement with NTRC. In addition, Basilea is sub-licensing its rights and obligations under the license and collaboration agreement with NTRC to SillaJen.

Adesh Kaul, Chief Financial Officer of Basilea, said: "We are pleased to have found in SillaJen an experienced oncology partner for the further development of BAL0891. This transaction marks an important step on our path to be a focused anti-infectives company. We are proud to have advanced BAL0891 through preclinical development to the start of clinical studies. The unique inhibition profile of BAL0891, targeting both TTK and PLK1 kinases, differentiates the compound and offers the potential for development in multiple cancers. Through the structure of the transaction we continue to participate in the long-term value creation potential of this promising program."

Jaegyeong Kim, MD, CEO at SillaJen, Inc. stated: "We are excited to acquire the rights to BAL0891. This agreement for a first-in-class anti-cancer drug, demonstrates our commitment to enhance lives of patients suffering from advanced solid tumors. In addition to our leading pipeline of Pexa-Vec, and the next generation SJ-600 series of oncolytic virus modalities, we are committed to advance and further develop medicines that will address the greatest unmet needs in cancer treatment. We hope to successfully deliver these products and help alleviate the burden of cancer patients worldwide."

Under the terms of the agreement, Basilea will receive upfront and near-term milestone payments of USD 14 million. Basilea is also eligible to receive further payments of up to approximately USD 320 million upon the achievement of predefined development, regulatory and sales milestones and tiered royalties on net sales starting in the single digit range going up to double-digits. Basilea remains responsible for making milestone and royalty payments to NTRC according to the license and collaboration agreement with NTRC.

In February this year Basilea announced its intention to focus on becoming a leading anti-infectives company and therefore to separate its oncology assets. Basilea has already made significant progress in the implementation of the new strategy and expects no material expenses related to oncology activities beyond 2022. Basilea is on track to achieve sustainable profitability from 2023.

Basilea updates its full-year 2022 financial guidance. It expects higher total revenues of CHF 116 to 122 million (previously CHF 106 to 112 million), an improved operating result of CHF -10 to -15 million (previously CHF -20 to -25 million) and improved cash flow from operating activities of CHF 0 to -5 million (previously CHF -10 to -15 million).

About SillaJen, Inc.

SillaJen, Inc. (KOSDAQ:215600) is one of the leading biotech companies in the development of oncolytic vaccinia immunotherapy. It is headquartered in Seoul, South Korea, with research center and satellite offices in Busan, South Korea and San Francisco, CA. SillaJen, Inc. is conducting Phase 2 trials for the treatment of renal cell carcinoma with the company’s lead pipeline, Pexa-Vec. The company is also developing intravenous injectable oncolytic vaccinia virus pipelines (SJ-600 series) to be more applicable and efficacious against various tumors. Additional information about SillaJen, Inc. is available at www.sillajen.com.

About BAL0891

BAL0891 is a first-in-class mitotic checkpoint inhibitor that pushes cells through mitosis without adequate time for correct chromosome segregation. This results in aberrant tumor cell division leading to tumor cell death. The compound is a unique dual inhibitor of threonine tyrosine kinase (TTK) and polo-like kinase 1 (PLK1). Both kinases collaborate in activating the mitotic spindle assembly checkpoint (SAC), a cell division mechanism regulating correct chromosome alignment and segregation. The dual action of BAL0891 leads to a rapid disruption of the SAC driving cells through mitosis before the chromosomes are properly aligned, leading to premature cell division and tumor cell death. BAL0891 has shown anti-proliferative activity across diverse tumor cell lines in vitro and single agent efficacy in in-vivo models of solid human cancers. BAL0891 was in-licensed from NTRC in 2018.