On January 22, 2020 PDC*line Pharma, a clinical stage biotech company developing a new class of potent and scalable active immunotherapies for cancers, reported the completion of its Series B round of financing (Press release, PDC Line Pharma, JAN 22, 2020, View Source [SID1234553381]). The company has raised a total of €20 million ($22.2M).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Two and a half years after its last round of fundraising in July 2017, PDC*line Pharma has attracted new investors, securing a further capital increase of €13.9M ($15.5M). In addition, the company will receive €6.1M ($6.8M) in loans and subsidies from the Walloon Region of Belgium, which has backed the company since it expanded its operations there in 2016.

PDC*line Pharma has attracted five new investors: Korean Investment Partners, the leading multi-billion dollar South-Korean fund, two further South-Korean funds, Shinhan-Cognitive Start-up Fund and UTC 2019 BIOVENTUREFUND, as well as two Belgian funds, SRIW (The Regional Investment Company of Wallonia) and Sambrinvest (the investment fund of Charleroi). They join the company’s historical investors: SFPI-FPIM, the Belgian Federal Holding and Investment Company, Noshaq Group (ex-Meusinvest), the Financière Spin-off Luxembourgeoise/INVESTSUD Group and a group of international business angels and well-known entrepreneurs.

"We are delighted by the trust our investors have placed in us and the opportunity to successfully complete this fund raising," said Eric Halioua, president & CEO of PDC*line Pharma. "The competitive advantage of our vaccinal platform in cancer immunotherapy and the significant achievements we have made over the last three years, including the signing of an important licensing deal with LG-Chem in Asia and the start of our phase I/II trials in France and Belgium in non-small-cell lung cancer (NSCLC), have been key factors in our investors’ decisions."

The objectives of the phase I/II study (PDC-LUNG-101) are to assess the safety, tolerability, immunogenicity and preliminary clinical activity of the drug candidate, PDC*lung01, associated or not with anti-PD-1 treatment in NSCLC. A total of 66 evaluable HLA-A*02:01 positive NSCLC patients are expected in three clinical centers in Belgium and six sites in France. PDC*lung01 comprises PDC*line professional antigen-presenting cells loaded with HLA-A2 restricted peptides derived from six shared tumor antigens.

According to François Fontaine, general advisor to PDC*line Pharma at SFPI-FPIM, "PDC*line Pharma’s cancer vaccine platform is both highly innovative and meets an important medical need. It also has a clear societal impact for the Liège region, for Belgium and beyond. We welcome the continued strong support of the Walloon region and the new funding by existing and new investors."

"In line with our strategy to support transformative technologies, we are thrilled to join PDC*line Pharma in advancing its Smission to treat cancer patients," said Sangwoo Lee, managing director at Korea Investment Partners. "PDC*line Pharma’s scientific expertise is matched by the management it has assembled to advance its clinical and research programs."

On January 21, 2020 Gene Techno Science (Kidswell Bio) reported a joint development agreement with MabGenesis to obtain and apply patent for new antibodies with the effect of killing cancer cells (Press release, Kidswell Bio, JAN 21, 2020, View Source [SID1234625473]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

1. Purpose and background for concluding this agreement
This agreement aims to expand the new pipeline in the new biologic business, which is a future growth driver for our company. Recently, efficiency in cancer treatments has been improved with the emergence of antibody drugs and antibody drug conjugates in addition to conventional small molecule drugs. Furthermore, the realization of antibody drugs with new molecular mechanisms of action, such as Opdivo, has become a long-awaited solution for patients previously considered as untreatable. However, there are still many patients who cannot be cured with those drugs. In fact, R&D for rare and intractable cancers with small number of patients is inactive for financial reasons at present, and therefore development of new cancer treatment is essential.

MabGenesis is a bio venture company aiming to obtain a first-in-class, best-in-class monoclonal antibody using innovative and ingenious antibody isolation technology owned by Professor Kazuhiro Morishita, University of Miyazaki and Lecturer Gene Kurosawa, Fujita Health University. Focusing on this technology, GTS signed a joint research agreement with the aim of executing the development of cancer treatments with a new mechanism of action for rare and intractable cancers by acquiring monoclonal antibodies that bind specifically and firmly to specific antigens expressed on the surface of cancer cells.

2. Contents of this agreement Joint research agreement for acquisition of monoclonal antibodies to specific antigens expressed on the surface of cancer cells

3. Outline of counterparties to this agreement
1. Company Name MabGenesis Inc.
2. Name and title of
representatives
Chief Executive Officer Katsuhiro Shinjo
3. Head Office 1-4, Honcho, Nihonbashi, Chuo-ku, Tokyo
4. Established June 3rd, 2019
5. Main Business Research and development of pharmaceutical drugs
6. Capital 21,750 thousand Japanese Yen
4. Future outlook
The impact on the business results for the fiscal year ending March 2020 is expected
to be minimal.

On January 21, 2020 Cue Biopharma, Inc. (NASDAQ: CUE), a clinical-stage biopharmaceutical company engineering a novel class of injectable biologics to selectively engage and modulate targeted T cells within the body, reported the publication of research demonstrating the ability of its lead biologic candidate CUE-101 to activate tumor antigen specific antitumor immunity in the peer-reviewed medical journal Clinical Cancer Research, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Press release, Cue Biopharma, JAN 21, 2020, View Source [SID1234608306]). The manuscript by Steven Quayle et al. is titled "CUE-101, a Novel HPV16 E7 pHLA-IL-2-Fc Fusion Protein, Enhances Tumor Antigen Specific T Cell Activation for the Treatment of HPV16-Driven Malignancies." (http://bit.ly/2twfkxd)

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The research highlights the ability of the company’s proprietary Immuno-STAT (Selective Targeting and Alternation of T cells) platform to selectively engage and modulate targeted T cells within the body. CUE-101 is the company’s lead drug candidate from the IL-2 based CUE-100 series designed to directly engage and activate T cells to target HPV16-driven recurrent/metastatic head and neck squamous cell carcinoma (HNSCC).

The published results show that CUE-101 demonstrated selective binding, activation and expansion of the disease-relevant human T cell population in vitro, as well as predict a favorable safety profile. A murine surrogate molecule (mCUE-101) administered to HPV16 E7 tumor bearing mice resulted in selective expansion of disease-relevant T cells, anti-cancer efficacy and immunologic memory. In addition, mCUE-101 administered as a combination therapy with anti-PD-1 checkpoint inhibition further enhanced anti-tumor efficacy. These data support the potential for CUE-101 to enhance anti-tumor immunity in HPV16-driven malignancies. CUE-101 is currently being studied in a Phase 1 clinical trial (NCT03978689) for HPV16-driven HNSCC. The Phase 1 trial is evaluating the safety and tolerability, anti-tumor response, pharmacokinetics and immunogenicity of CUE-101 as a monotherapy in patients with confirmed HPV16-driven recurrent/metastatic HNSCC and HLA-A*02:01 serotype. Based on results from this trial, including translational pharmacodynamic immunoprofiling data, the company may expand the study to test CUE-101 in the neoadjuvant setting and in combination with checkpoint inhibitors in patients with HPV16-driven HNSCC.

"This seminal publication from Cue Biopharma exemplifies the core-strength of our Immuno-STAT biologics platform, in particular the CUE-100 series that incorporates rationally engineered IL-2 molecules for selective activation of tumor-specific T cells. The preclinical and ex vivo human translational data presented in this paper underscore the promising potential for the current, ongoing clinical study evaluating CUE-101 monotherapy in HPV-driven head and neck cancer," stated Anish Suri, Ph.D., president and chief scientific officer of Cue Biopharma.

HPV-driven cancers account for more than 20,000 deaths each year in the U.S. and Europe. The majority of these cancers are driven by HPV16 which carries the E7 protein targeted by CUE-101. Despite treatment with current standards of care, approximately 50% of patients with advanced disease will experience recurrence and significant quality of life impact. Patients with HPV-driven cancers represent an important unmet clinical need and underscore the opportunity for promising new therapeutics.

Clinical Cancer Researchis a peer-reviewed medical journal published by the American Association for Cancer Research (AACR) (Free AACR Whitepaper). The journal publishes innovative clinical and translational cancer research studies that bridge the laboratory and the clinic.

About the CUE-100 Series
The CUE-100 series consists of Fc-fusion biologics that incorporate peptide-MHC (pMHC) molecules along with rationally engineered IL-2 molecules. This singular biologic is anticipated to selectively target, activate and expand a robust repertoire of tumor-specific T cells directly in the patient. The binding affinity of IL-2 for its receptor has been deliberately attenuated to achieve preferential selective activation of tumor-specific effector T cells while reducing potential for effects on regulatory T cells (Tregs) or broad systemic activation, potentially mitigating the dose-limiting toxicities associated with current IL-2-based therapies.

About Immuno-STAT
Immuno-STAT biologics are designed for targeted modulation of disease-associated T cells in the areas of immuno-oncology and autoimmune disease. Each of our biologic drugs is designed using our proprietary scaffold comprising: 1) a peptide-MHC complex (pMHC) to provide selectivity through interaction with the T cell receptor (TCR), and 2) a unique co-stimulatory signaling molecule to modulate the activity of the target T cells.

The simultaneous engagement of co-stimulatory molecules and pMHC binding mimics the signals delivered by antigen presenting cells (APCs) to T cells during a natural immune response. This design enables Immuno-STAT biologics to engage with the T cell population of interest, resulting in highly targeted T cell modulation. Because our drugs are delivered directly in the patient’s body (in vivo), they are fundamentally different from other T cell therapeutic approaches that require the patients’ T cells to be extracted, stimulated and expanded outside the body (ex vivo), and reinfused in an activated state.

On January 21, 2020 Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a leader in the growing field of cancer metabolism-based therapeutics, reported its collaboration with Michigan Medicine to proceed with a Phase 1b/2 clinical trial of CPI-613 (devimistat) in combination with gemcitabine and cisplatin for patients with biliary tract cancer (Press release, Rafael Pharmaceuticals, JAN 21, 2020, View Source [SID1234553562]). The study will be a multicenter randomized trial of gemcitabine and cisplatin with or without devimistat as a first-line therapy for patients with locally advanced, unresectable or metastatic biliary tract cancer who have had no prior treatment.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The goal of the Phase 1b part of the trial will be to identify the maximum tolerated dose (MTD), or the highest dose of devimistat that does not cause side effects, when given in combination with gemcitabine and cisplatin. This will determine the recommended dose for the randomized Phase 2 part of the trial. The goal of Phase 2 will be to then determine the efficacy of devimistat in combination with gemcitabine and cisplatin as compared with the combination chemotherapy alone. An estimated 68 to 78 patients will be enrolled in the study.

"Biliary tract cancer is a rare and aggressive cancer that affects approximately 15,000 people in the United States each year," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "Launching this trial aligns with our mission to help patients with significant unmet medical needs."

Vaibhav Sahai, MBBS, MS., Associate Professor of Medical Oncology at Michigan Medicine, is the Principal Investigator on the study.

"Devimistat has demonstrated promising results in pancreatic cancer," said Dr. Sahai. "Rare cancers require dedicated and innovative research, and I am hopeful that we will advance treatment options for patients with biliary tract cancer."

About CPI-613 (devimistat)

CPI-613 (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient’s side effects. Combination with devimistat represent a diverse range of opportunities to substantially improve patient’s benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma and Burkitt’s lymphoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.

On January 21, 2020 REVOLUTION Medicines, Inc., a clinical-stage leader in the discovery and development of novel small molecule inhibitors of frontier oncology targets within notorious pathways, reported that it had filed to raise $100 million in IPO on Nasdaq. (Press release, Revolution Medicines, JAN 21, 2020, View Source [SID1234553553])

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Company Description

We are a clinical-stage precision oncology company focused on developing novel targeted therapies to inhibit elusive, high-value frontier targets within notorious growth and survival pathways, with particular emphasis on the RAS and mTOR signaling pathways. We define frontier targets as proteins that play an important role in cancer and for which there is either: no approved drug that directly inhibits it, or one or more approved drugs that directly inhibit it but through a mechanism of action that may not enable suppression of the full range of its biologic contributions to cancer. Our understanding of genetic drivers and adaptive resistance mechanisms in cancer, coupled with robust drug discovery and medicinal chemistry capabilities, has guided us to establish a deep pipeline targeting critical signaling nodes within these pathways. This cohesive approach underpins our clinical strategy of exploring mechanism-based dosing paradigms and in-pathway combinations to optimize treatment for cancer patients.

Our most advanced product candidate, RMC-4630, is a potent and selective inhibitor of SHP2, based on preclinical evidence described in this prospectus. SHP2 is a central node in the RAS signaling pathway. In collaboration with Sanofi, we are evaluating RMC-4630 in a multi-cohort Phase 1/2 clinical program. This RMC-4630 Phase 1/2 program currently consists of two active clinical trials: RMC-4630-01, a Phase 1 study of RMC-4630 as a single agent, and RMC-4630-02, a Phase 1b/2 study of RMC-4630 in combination with the MEK inhibitor cobimetinib (Cotellic). In this prospectus, we report preliminary data from 63 patients who had enrolled in our Phase 1 study and received RMC-4630 as monotherapy as of November 6, 2019 and from eight patients who had enrolled in our Phase 1b/2 combination study and received RMC-4630 as of November 14, 2019. Leveraging our proprietary tri-complex technology platform, we are also developing a portfolio of mutant-selective RAS inhibitors that we believe are the first potent, selective, cell-active inhibitors of the active, GTP-bound form of RAS, or RAS(ON). These inhibitors also have exhibited anti-tumor activity in vivo in preclinical models.

Initially, we will prioritize four mutant RAS(ON) targets—KRASG12C, KRASG13C, KRASG12D and NRASG12C—and expect to nominate our first development candidate in 2020. Our pipeline also includes inhibitors of other key nodes within the RAS and mTOR signaling pathways, such as SOS1 and mTORC1. Our pipeline includes one product candidate that is in clinical development and all of our other programs are in the preclinical stage. We believe our deep, differentiated pipeline and development strategies provide us with the opportunity to pioneer novel treatment regimens to maximize the depth and durability of clinical benefit and circumvent adaptive resistance mechanisms for patients with cancers dependent on these critical pathways.

— Under our collaboration on our SHP2 program with Sanofi, we have a 50-50 profit share and a co-promote right in the United States and are eligible to receive royalties on net sales outside of the United States. Sanofi is responsible for reimbursing substantially all of our research costs and all of our development costs for the SHP2 program. For all other programs, we retain worldwide commercial rights. —