On April 12, 2021 XNK Therapeutics AB ("XNK" or the "Company"), a Swedish clinical stage immunotherapy company treating cancer by developing novel NK cell-based therapies, reported the completion of a private placement of SEK 64 million (Press release, CellProtect Nordic Pharmaceuticals, APR 12, 2021, View Source [SID1234577885]). The purpose of the issue is primarily to finance the Company’s research and development efforts.

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"XNK has made significant progress with its technology platform and this new funding will be key to continue the clinical development of our leading investigational drug candidate," said Johan Liwing, CEO of XNK.

On April 11, 2021 StoneWise, a medical technology company that accelerates novel drug discovery with artificial intelligence, reported that it has completed its series B and B+ financing to raise a total of US$100 million (Press release, Stonewise, APR 11, 2021, View Source [SID1234654561]).

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The series B financing was led by Legend Capital, while the series B+ funding was co-led by Greater Bay Area Community Development Fund and Lightspeed China. New investors include CDH VGC Fund and Eastern Bell Capital. Existing shareholders HillHouse Ventures, SIG, Long Hill Capital, and Linear Capital made additional investments.

The proceeds will be used to recruit and retain world-leading talents and upgrade the AI-enabled drug discovery platform so as to advance innovation and improve R&D efficiency.

"We will use the proceeds from these two rounds of financing to strengthenour talent team, accelerate technology iteration, and support project execution," says Zhou Jielong, founder of StoneWise. "We expect more eminent professionals to join us in the early stage of pharmaceutical development. Together, we will identify promising drug candidates faster, advance the use of AI in drug innovation, and improve life quality," he continues.

StoneWise completed four rounds of financing in 2020. Its investors include high impact players in both the pharmaceutical and the AI sectors. The latest financing will empower StoneWise to leverage its molecular design platform and knowledge graph to gear up AI-driven discovery of novel drugs.

It takes up to 15 years and around US$2.6 billion on average to develop a new drug. As one of the global pioneers to apply AI to small molecule drug R&D, StoneWise makes drug development more time-efficient and cost-effective with its drug discovery platform.

The platform provides pharmaceutical companies an integrated solution, featuring a vast range of tools such as a wide range of AI models, interactive medical knowledge graph, deep learning-driven scaffold hopping/derivatization, and generative molecular creation and design.

Richard Li, President of Legend Capital says, "Drug innovation is at the core of pharmaceutical development, and it is also a major entry point for AI application. StoneWise is an industry leader in terms of new technology development, strategic planning and project execution. We are confident that via integrating AI with computational chemistry, pharmacology, molecule dynamics, quantum mechanics, and other cutting-edge technologies, StoneWise will strive to lead AI-driven drug innovation."

"Drug discovery industry faces challenges such as long R&D cycles, low success rates, and sky-rocketing costs, which can be more effectively addressed by AI technologies. Up to now, multiple drugs developed with the support of AI entered into clinical development stages," says Mingyu Wang, senior partner of CDH VGC. "StoneWise’s AI platform has been well received by many pharmaceutical companies. We are confident on its growth potential and expect a continuous output of high-quality drug candidates., and a significantly improved drug discovery R&D system."

On April 11, 2021 Checkmate Pharmaceuticals, Inc. (NASDAQ: CMPI) ("Checkmate"), a clinical stage biopharmaceutical company focused on developing its proprietary technology to harness the power of the immune system to combat cancer, reported the presentation of new translational data from Checkmate’s Phase 1b trial of vidutolimod (CMP-001) in combination with pembrolizumab in patients with advanced anti-PD-1 refractory melanoma (Press release, Checkmate Pharmaceuticals, APR 11, 2021, View Source [SID1234577914]).

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Vidutolimod (CMP-001) demonstrates improved response in noninflamed anti–PD-1 refractory melanoma and response is associated with serum CXCL10 (Abstract #: 5231: NCT02680184)

During the 2021 AACR (Free AACR Whitepaper) Virtual Clinical Trials with Novel Immuno-oncology Strategies Session on April 11 from 4:00 – 5:45pm ET, Jason Luke, MD, Director, Cancer Immunotherapeutics Center, UPMC Hillman Cancer Center, and Associate Professor of Medicine, University of Pittsburgh School of Medicine, presents new translational data from an ongoing Phase 1b study of vidutolimod in combination with pembrolizumab in patients with PD-1 blockade refractory advanced melanoma.

Key highlights from these clinical data include:

93% of patients had progressive disease as their last response assessment on prior PD-1 blockade therapy, 42% had an elevated LDH, and the median sum of the target lesions longest diameter was 6.7 cm, indicating advanced disease
Response rates to vidutolimod in combination with pembrolizumab were similar across baseline patient characteristics including BRAF mutation, LDH level, number of prior systemic cancer treatments, best response to prior PD-1 blockade therapy, and prior ipilimumab
Responders and non-responders were not distinguished by baseline tumor characteristics including PD-L1 CPS, IFNg transcriptional signature, CD8+ T cells, or nonsynonymous mutations
All patients showed the expected rapid induction of anti-Qb antibodies to the virus-like particle (VLP), which facilitate the pharmacodynamic response to vidutolimod, and the antibody titers were not associated with clinical response
Clinical activity of vidutolimod in combination with pembrolizumab was associated with serum CXCL10 induction magnitude, induction of an inflammatory gene expression profile, and CD8+ T cells in injected and noninjected tumors
"These translational data provide new insights into the rapid pharmacodynamic responses to vidutolimod and support the conclusion that clinical responses to treatment are not associated with the previously-described predictive markers for response to PD-1 blockade such as inflamed tumors," said Dr. Jason Luke.

On April 11, 2021 Taiho Oncology, Inc. reported efficacy and safety results from the Phase 2 FOENIX-CCA2 trial, a single-arm multicenter Phase 2 study evaluating futibatinib (TAS-120) in patients with intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 gene rearrangements including gene fusions who have failed at least one line of therapy (Press release, Taiho, APR 11, 2021, View Source [SID1234577871]). The data were presented online as an oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 Week 1 Clinical Trials Plenary from 2:00 – 3:45 PM ET on April 11, 2021.

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In the FOENIX-CCA2 trial, 103 patients with locally advanced or metastatic unresectable iCCA harboring FGFR2 gene rearrangements including fusions who had received one or more prior lines of systemic therapy received futibatinib 20 mg once daily until disease progression or unacceptable toxicity. The study met its primary endpoint of a greater than 20% objective response rate (ORR) assessed by independent central review with an ORR of 41.7%. Secondary endpoints of duration of response (DOR) and disease control rate (DCR) were also reported; responses were durable, with a median DOR of 9.7 months and 72% of responses ≥6 months, and a DCR of 82.5%. Median progression-free survival (PFS) was 9.0 months and median overall survival (OS) was 21.7 months, with 72% of patients alive at 12 months.

Common treatment-related adverse events (TRAEs) were hyperphosphatemia (85%), alopecia (33%) and dry mouth (30%). The most frequent grade 3 TRAE was hyperphosphatemia (30%), which resolved in patients with adequate management. One grade 4 TRAE of increased ALT was reported and there were no treatment related deaths.

"The results of FOENIX-CCA2 are significant for patients living with refractory intrahepatic cholangiocarcinoma with FGFR2 gene fusions or other rearrangements as futibatinib showed a meaningful ORR of 41.7% and good durability of responses," said medical oncologist Lipika Goyal, MD, MPhil, Massachusetts General Hospital Cancer Center, and lead investigator on the study. "These results represent another example of the promise of precision medicine in cholangiocarcinoma and indicate that futibatinib could be an option for patients with refractory CCA if approved."

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for futibatinib for the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions in February 2021 based on efficacy and safety results from the FOENIX-CCA2 study. The FDA Office of Orphan Drug Development granted futibatinib orphan drug status for the treatment of cholangiocarcinoma in May 2018.

Additional Data on Futibatinib (TAS-120) in iCCA Presented

Taiho Oncology also presented preclinical and Phase 1 clinical data for futibatinib at AACR (Free AACR Whitepaper) as poster presentations. Presentations include:

Acquired resistance to ATP-competitive and irreversible FGFR inhibitors (FGFRi’s): A library-based approach: Hiroshi Sootome, MS, Manager, Translational Research Planning & Management group, Taiho Pharmaceutical Co., Ltd. (1117). Results were shared online as a poster presentation from 8:30 AM – 11:59 PM ET on April 10, 2021.
Effect of futibatinib on QT/QTc interval: a randomized, controlled, double-blind study: Ikuo Yamamiya, PhD, Associate Director, Bioanalytics & DMPK, Taiho Oncology, Inc. (CT128). Results were shared online as a poster presentation from 8:30 AM – 11:59 PM ET on April 10, 2021.
Evaluation of potential drug-drug interactions (DDIs) between futibatinib and CYP3A inhibitors/inducers, CYP3A substrates, or proton pump inhibitors (PPIs): Ikuo Yamamiya, PhD, Associate Director, Bioanalytics & DMPK, Taiho Oncology, Inc. (CT125). Results were shared online as a poster presentation from 8:30 AM – 11:59 PM ET on April 10, 2021.
Please visit Taiho Oncology’s virtual Medical Booth for more information.

"With the low survival rates typically seen in patients with intrahepatic cholangiocarcinoma, the possibility of a new treatment option with demonstrated efficacy and safety is an important development for the oncology community," said Martin J. Birkhofer, MD, Senior Vice President and Chief Medical Officer, Taiho Oncology, Inc. "Taiho Oncology looks forward to sharing these data with regulatory authorities, with the hope of supporting approval for this important investigational therapy."

About Futibatinib (TAS-120)
Futibatinib (TAS-120) is an investigational, oral, potent, selective, and irreversible small-molecule inhibitor of FGFR1, 2, 3 and 4 being studied as a potential treatment for patients with advanced solid tumors with FGFR1-4 genetic aberrations, including cholangiocarcinoma, who were previously treated with chemotherapy or other therapies. Futibatinib selectively and irreversibly binds to the ATP binding pocket of FGFR1-4 resulting in the inhibition of FGFR-mediated signal transduction pathways, reduced tumor cell proliferation and increased tumor cell death in tumors with FGFR1-4 genetic aberrations.

About Cholangiocarcinoma
Cholangiocarcinoma (CCA), also known as bile duct cancer, is not common. About 8,000 people in the U.S. are diagnosed with CCA each year.1 This includes both intrahepatic (inside the liver) and extrahepatic (outside the liver) cancers. CCA can occur at younger ages, but it is seen mainly in older people. The average age of people in the U.S. diagnosed with cancer of the intrahepatic bile ducts is 70, and for cancer of the extrahepatic bile ducts it is 72.1 The five-year survival rate of intrahepatic CCA (all SEER stages combined) is 9%.2

The main treatment for CCA is surgery. Radiation therapy and chemotherapy may be used if the cancer cannot be entirely removed with surgery and in cases where the edges of the tissues removed at the operation show cancer cells (also called a positive margin). Both stage III and stage IV cancers cannot be completely removed surgically. Currently, standard treatment options are limited to radiation, palliative therapy, liver transplantation, surgery, chemotherapy and interventional radiology.3

On April 11, 2021 Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, reported that the first patient has been dosed in a phase II trial (BUNCH) in China of ATG-008 (onatasertib), a second-generation mTORC1/2 inhibitor, for the treatment of advanced solid tumors harboring NFE2L2, STK11, RICTOR or other specific genetic alterations (Press release, Antengene, APR 11, 2021, View Source [SID1234577870]). The purpose of the single-arm, open-label trial is to evaluate the safety and efficacy of ATG-008 in patients with such advanced solid tumors that may be predictably sensitive to inhibition of mTORC1/2.

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According to the latest report by the World Health Organization, there were an estimated 19.3 million newly diagnosed cancer cases and 10 million cancer-related mortalities in 2020 globally. Furthermore, the report also projected a 47% increase in cancer-related disease burden in the coming 20 years. China now ranks number one in both cancer incidence and mortality rates[i]. This reminds us of the daunting challenges in cancer treatment and the ever more urgent need for effective novel anti-cancer therapies in China.

There is a close association between the mTOR signaling pathway and genetic mutations in NFE2L2, STK11 and RICTOR. ATG-008, a potent selective inhibitor of mTOR kinase that is currently under clinical development, may induce the cell death of multiple tumor types through the dual-inhibition of mTORC1 and mTORC2. Therefore, mTOR inhibitor ATG-008 has the potential to offer a new treatment option for patients with advanced solid tumors with such genetic alterations. Moreover, preclinical and clinical data also demonstrated the potent antitumor activity of ATG-008 in multiple tumor types.

Dr. Jay Mei, Founder, Chairman and CEO of Antengene, commented: "There are urgent needs for more effective treatment options for patients with various advanced solid tumors. We believe ATG-008 has strong potential to address a serious unmet medical need in a hard-to-treat group of cancers. This trial also marks an important step in the development of our innovative pipeline for the treatment of patients with solid tumors in China. We look forward to producing important clinical data to guide the further development and continue to demonstrate Antengene’s leadership in drug development for mTORC1/2 inhibitors."

Antengene has initiated several clinical trials in China and other Asia Pacific countries and regions with ATG-008 in the treatment of advanced hepatocellular carcinoma (HCC), advanced non-small-cell lung cancer (NSCLC) and in combination with an anti-PD-1 antibody in advanced solid tumors including hepatocellular carcinoma (HCC).

About ATG-008 (onatasertib)

ATG-008 (onatasertib) is a second-generation mTORC1/2 inhibitor, for which development and commercialization rights in Asia Pacific were licensed from Celgene (now Bristol Myers Squibb). ATG-008 is currently being studied in multi-regional clinical trials for the treatment of advanced hepatocellular carcinoma (HCC), as well as non-small-cell lung cancer (NSCLC), advanced solid tumors harboring NFE2L2, STK11, RICTOR or other specific genetic alterations and other cancers as a single agent or in combination with an anti-PD-1 antibody.