On October 19, 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 three poster presentations at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 35th Anniversary Annual Meeting (SITC 2020) (Press release, Cue Biopharma, OCT 19, 2020, View Source [SID1234608294]).

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Presentation Details:
Title: Immuno-STATs: Leveraging protein engineering to expand and track antigen-specific T cells in vivo
Poster #: 623
Presenter: Steven Almo, Ph.D., ‎co-founder
Date: Wednesday, November 11, 2020 from 5:15–5:45 p.m. EST and Friday, November 13, 2020 from 4:40–5:10 p.m. EST

Title: CUE-100 series Immuno-STATs from concept to the clinic: Leveraging protein engineering to stimulate and selectively deliver affinity-attenuated IL-2 to antigen-specific T cells
Poster #: 553
Presenter: Saso Cemerski, Ph.D., ‎vice president and head, discovery and translational immunology
Date: Wednesday, November 11, 2020 from 5:15–5:45 p.m. EST and Friday, November 13, 2020 from 4:40–5:10 p.m. EST

Title: A phase 1 trial of CUE-101 a novel HPV16 E7-pHLA-IL2-Fc fusion protein in patients with recurrent/metastatic HPV16+ head and neck cancer
Poster #: 354
Presenter: Sara I. Pai, M.D., Ph.D., associate professor, Massachusetts General Hospital and Harvard Medical School, Boston
Date: Thursday, November 12, 2020 from 4:50–5:20 p.m. EST and Saturday, November 14, 2020 from 1–1:30 p.m. EST

"Data presented from these posters further demonstrate the therapeutic potential of our Immuno-STAT platform," said Anish Suri, Ph.D., president and chief scientific officer of Cue Biopharma. "We look forward to presenting preclinical data highlighting the IL-2 based CUE-100 series Immuno-STATs, as well as results from our ongoing open-label, dose escalation Phase 1 monotherapy trial with CUE-101 in patients with HPV16+ head and neck cancer."

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-regulatory 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, modified outside the body (ex vivo), and reinfused.

On October 19, 2020 Betta Pharmaceutical Co., Ltd. reported that the first subject of the Phase I clinical study of the new drug BPI-27336 was successfully enrolled (Press release, Betta Pharmaceuticals, OCT 19, 2020, View Source [SID1234574036]). On October 20, the patient’s first administration was completed. The study is called "Phase I clinical study of BPI-27336 tablets in patients with advanced solid tumors", and the main investigator is Professor Shen Lin from Beijing Cancer Hospital. The study includes two parts: dose escalation and enrollment expansion. The main goal is to evaluate the safety and tolerability of BPI-27336 as a selective ERK1/2 small molecule inhibitor for patients with advanced solid tumors, explore the maximum tolerated dose (MTD) and Dose-limiting toxicities (DLTs), as well as determining the recommended dose for later clinical studies, and evaluating the initial efficacy.

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Mechanism of action of BPI-27336

ERK1/2 is an important member of the mitogen-activated protein kinases (MAPK) family, located downstream of the RAS/RAF/MEK/ERK (RAS-MAPK) pathway. The RAS-MAPK signaling pathway is involved in a series of cell physiological activities such as cell growth, development, differentiation, and apoptosis, and is the hardest hit area that induces tumors. Domestic and foreign literature reports that MAPK is obviously related to the occurrence and development of lung cancer, breast cancer, ovarian cancer, esophageal cancer, colon cancer, stomach cancer, liver cancer and other tumors. As the "final manager" downstream of the RAS-MAPK pathway, targeted inhibition of ERK1/2 is expected to be used to treat cancer caused by abnormal activation of the RAS-MAPK pathway, and may also be resistant to RAF or MEK inhibitors due to the reactivation of ERK1/2 The medicine is effective for patients.

Up to now, there is no drug marketed for ERK target in the world. As a new type of potent and selective ERK1/2 small molecule oral inhibitor, BPI-27336 has shown excellent anti-tumor effects in a variety of preclinical disease models, especially KRAS mutant tumor models, and has good safety. Sex, tolerability and pharmacokinetic characteristics. BPI-27336 is expected to improve the drug resistance of the RAS-MAPK pathway and enhance the efficacy, and it is expected to bring a breakthrough in the drugless global problem of KRAS mutant tumors. We look forward to accelerating the progress of clinical research with the help of the cooperation center and experts, providing patients with new treatment plans and meeting urgent clinical needs.

According to the announcement of the work plan for the registration and classification of chemical drugs, BPI-27336 is an "innovative drug that has not been marketed at home or abroad", and its registration is classified as category 1 chemical drugs. In February 2020, Betta Pharmaceuticals received the "Clinical Trial Notice" issued by the National Medical Products Administration (NMPA). BPI-27336 tablets meet the requirements of relevant drug registration regulations and agreed to carry out clinical research on solid tumors in accordance with the submitted plan (Acceptance number CXHL1900397, CXHL1900398).

On October 19, 2020 ONK Therapeutics reported for the second time this year alongside American biotech investor Acorn Bioventures and other shareholders in an $8 million (about €6.8 million) fundraising (Press release, ONK Therapeutics, OCT 19, 2020, View Source [SID1234570585]).

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ONK, which is developing enhanced natural killer cells in the immune system to target cancers, has now raised $14.6 million over the past six months. In May, Mr Mulligan led an investment that saw his cell engineering business, Avectas, take a 20 per cent stake in ONK.

ONK, an NUI Galway spinout founded by Prof Michael O’Dwyer in 2015, has also appointed industry veteran Chris Nowers as chief executive. Mr Nowers is a cell therapy expert who has worked in management roles with both Amgen and Bristol Myers Squibb over a 25-year career.

Prof O’Dwyer will continue in his role as chief scientific officer of the group. The latest funding will allow the company expand its team and its preclinical R&D and manufacturing capabilities.

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ONK has developed a process that genetically engineers natural killer (NK) cells – which are part of the body’s immune system – to allow them to better pursue cancer cells that have made themselves invisible to normal NK cells.

It licensed one of its prospective therapies – targeting blood cell cancers such as non-Hodgkin’s lymphoma and certain forms of leukaemia – to Avectas, whose Solupure process allows the gene editing to be done rapidly and safely, without upsetting the cells and in a way that also keeps the costs of the process down.

ONK’s second programme is hoping to help certain multiple myeloma patients.

Different patients
Unlike personalised medicine, where a patient’s own cells are engineered and reintroduced to the body to fight disease, ONK and Avectas are looking to engineer cells that can be used in many different patients suffering from these cancers.

Learn more
"This exciting off-the-shelf platform has the potential to produce a next generation of cell therapies, which could improve performance and overcome some of the shortcomings seen with earlier approaches," Mr Nowers said.

Acorn Bioventures partner Isaac Manke, who will join the board of ONK, said it was "a highly innovative next-generation NK cell therapy company with an exciting portfolio of research stage assets". He said the new funding should help bring the company’s pipeline drugs towards clinical trial stage.

This is understood to be Acorn’s first investment in an Irish business. The group, founded in 2017, specialises in investing in small-capitalisation public and private biotechnology companies.

On October 19, 2020 Diffusion Pharmaceuticals Inc. (Nasdaq: DFFN) ("Diffusion" or "the Company"), reported the appointment of Christopher D. Galloway, M.D. to Chief Medical Officer, effective immediately (Press release, Diffusion Pharmaceuticals, OCT 19, 2020, View Source [SID1234568923]). Dr. Galloway will report to Robert Cobuzzi, Ph.D., Diffusion’s Chief Executive Officer.

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Dr. Galloway is board certified in Emergency Medicine and will assume responsibility of leading the Company’s product development efforts, including clinical trials with Trans Sodium Crocetinate ("TSC"), which is being evaluated in an ongoing Phase 1b clinical study in Romania in patients with COVID-19. In addition, Dr. Galloway will lead the expansion of the Company’s work with TSC into other hypoxia-related conditions. Dr. Galloway brings to Diffusion more than 20 years of experience both in industry leadership roles in clinical development and medical affairs across multiple therapeutic areas and stages of development, as well as the perspective of a practicing physician.

"We are delighted to welcome Chris as a member of the Diffusion team. He brings a breadth of therapeutic area experiences both as a practicing physician and from his time in the biopharmaceutical industry. His leadership will be of significant benefit for Diffusion as we continue and expand the development of TSC for conditions and diseases where low oxygen levels are an issue, including respiratory diseases, such as COVID-19, oncology, and other hypoxia-related conditions," said Dr. Cobuzzi.

Dr. Galloway joins Diffusion from La Jolla Pharmaceuticals, where he has served as Senior Medical Director in critical care since August 2018. Among his duties at La Jolla, he chaired and oversaw the investigator-initiated and collaborative research programs, as well as supported the commercial and medical teams for the launch of GIAPREZA (angiotensin II). Prior to La Jolla, he was Medical Director for global clinical development at the immuno-oncology company, Rakuten Medical. At Rakuten, he was involved in clinical development, operations, and trial execution from Phase 1 through Phase 3 in multiple indications. Previously he has served as Medical Affairs Director within Merck’s immunotherapy division, and also led the team at DaVita Clinical Research as Medical Director and Principal Investigator specializing in early phase studies.

"I am thrilled to be joining the talented team at Diffusion and overseeing the development of our lead product, TSC," said Dr. Galloway. " Hypoxia-related pathology is prevalent across a multitude of medical conditions and therapeutic disciplines, and I believe TSC has the potential for meaningful improvement in patient care and outcomes."

Dr. Galloway received his M.D. degree from the University of Texas Medical Branch at Galveston, and completed his residency in Emergency Medicine at Carolinas Medical Center in Charlotte, N.C. He received a BA in biology from the University of Texas at Austin, and is licensed to practice medicine in Colorado. Dr. Galloway is a Diplomate of the American Board of Emergency Medicine.

Inducement Grant Under NASDAQ Listing Rule 5635(c)(4

In connection with Dr. Galloway’s new employment, the Compensation Committee of Diffusion’s Board of Directors has approved the grant of non-qualified stock options to Dr. Galloway, who will receive options to purchase 200,000 shares of Diffusion’s common stock. The grant date for the options will be October 19, 2020, and the exercise price per share for such stock options will be the closing price of Diffusion’s common stock on such date, as reported by NASDAQ. The grant was approved and will be made as an inducement material to Dr. Galloway’s acceptance of employment with Diffusion, in accordance with NASDAQ Listing Rule 5635(c)(4).

The options will have a 10-year term and will vest on a monthly basis over the 36 months after the date of grant, subject to Dr. Galloway’s continuous employment with Diffusion through each applicable vesting date. In addition, the options will be subject to acceleration or forfeiture upon the occurrence of certain events as set forth in Dr. Galloway’s option and employment agreements.

On October 19, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that it will report its third-quarter 2020 financial results on Thursday, October 29, 2020 after the financial markets close (Press release, Vertex Pharmaceuticals, OCT 19, 2020, View Source [SID1234568691]). The company will host a conference call and webcast at 4:30 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International).

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The conference call will be webcast live and a link to the webcast can be accessed through Vertex’s website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company’s website.