On March 9, 2022 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company developing targeted therapies for RAS-addicted cancers, reported the company will make seven oral presentations at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 being held April 8-13, 2022 in New Orleans, Louisiana (Press release, Revolution Medicines, MAR 9, 2022, View Source [SID1234609791]).

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Four of the company’s oral presentations will be featured in the conference mini-symposium session entitled "Targeting the RAS Oncogene." These presentations will describe recent research findings regarding Revolution Medicines’ broad pipeline of RAS(ON) Inhibitors currently in development, including RMC-6236 (RASMULTI), RMC-6291 (KRASG12C), RMC-9805 (KRASG12D) and RMC-8839 (KRASG13C).

Three additional oral presentations from Revolution Medicines will provide deeper insights into the tri-complex RAS(ON) inhibitor platform. These will highlight the mechanisms underlying this novel inhibitor modality and the broad potential of this platform to target the RAS(ON) form of multiple oncogenic RAS variants and deliver first-in-class therapeutics designed to address the significant unmet needs of patients with RAS-addicted cancers.

Additionally, collaborators of Revolution Medicines will make four separate poster presentations. Three of these describe the activity of the company’s RAS(ON) Inhibitors and/or RAS Companion Inhibitors in preclinical models. A fourth relates to machine-learning digital pathology in support of clinical biomarker strategies.

Details of the planned presentations are as follows:

Revolution Medicines Oral Presentations:

Title: Direct targeting of KRASG12X mutant cancers with RMC-6236, a first-in-class, RAS-selective, orally bioavailable, tri-complex RASMULTI(ON) inhibitor
Presenter: Mallika Singh, Ph.D., vice president, translational research
Abstract Number: 3597
Session: Targeting the RAS Oncogene
Date/Time: 2:35 – 2:50 p.m. Central on April 12, 2022

Title: RMC-6291, a next-generation tri-complex KRASG12C(ON) inhibitor, outperforms KRASG12C(OFF) inhibitors in preclinical models of KRASG12C cancers
Presenter: Bob Nichols, Ph.D., project team leader for RMC-6291
Abstract Number: 3595
Session: Targeting the RAS Oncogene
Date/Time: 2:50 – 3:05 p.m. Central on April 12, 2022

Title: RM-036 (RMC-9805), a first-in-class, orally-bioavailable, tri-complex covalent KRASG12D(ON) inhibitor, drives profound anti-tumor activity in KRASG12D mutant tumor models
Presenter: John Knox, Ph.D., senior director, computational chemistry
Abstract Number: 3596
Session: Targeting the RAS Oncogene
Date/Time: 3:05 – 3:20 p.m. Central on April 12, 2022

Title: A first-in-class tri-complex KRASG13C(ON) inhibitor validates therapeutic targeting of KRASG13C and drives tumor regressions in preclinical models
Presenter: Christopher Schulze, Ph.D., associate director, molecular and cellular cancer biology
Abstract Number: 3598
Session: Targeting the RAS Oncogene
Date/Time: 3:20 – 3:35 p.m. Central on April 12, 2022

Title: Discovery and development of RAS(ON) inhibitors beyond KRASG12C
Presenter: Elena S. Koltun, Ph.D., vice president, medicinal chemistry
Session: Chemistry to the Clinic, Part 1 of 3 – Targeting RAS Beyond KRASG12C
Date/Time: 9:00 – 9:20 a.m. Central on April 9, 2022

Title: Translating frontier oncology targets to outsmart cancer
Presenter: Matthew Holderfield, Ph.D., senior director, cancer cell and systems biology
Session: Challenging Drug Targets
Date/Time: 10:20 – 10:40 a.m. Central on April 12, 2022

Title: Pediatric Cancer Drug Discovery: The RAS/MAPK Pathway
Presenter: Clay Gustafson, M.D., Ph.D., senior medical director
Session: Pediatric Cancer Working Group Town Hall Meeting
Date/Time: 6:30 – 8:30 p.m. Central on April 10, 2022

Collaborator Poster Presentations:

Title: Combination of KRASG12C(ON) and SHP2 inhibitors overcomes adaptive resistance and enhances anti-tumour immunity
Abstract Number: 4029/8
Session: Molecular Pharmacology
Presentation Time: 9:00 a.m. – 12:30 p.m. Central on April 13, 2022

Title: Effective in vivo treatment of endometrial tumor models with coexistent mutant PI3K and PTEN inactivation with a selective bi-steric mTORC1 kinase inhibitor
Abstract Number: LB089/14
Session: Late-Breaking Research: Experimental and Molecular Therapeutics 1 / Chemistry
Presentation Time: 1:30 – 5:00 p.m. Central on April 11, 2022

Title: Bi-steric mTORC1 inhibitor RMC-6272 synergizes with immune checkpoint inhibitors to induce sustained regression of MYC-driven hepatocellular carcinoma
Abstract Number: 2662/5
Session: Signaling Pathway Inhibitors
Presentation Time: 9:00 a.m. – 12:30 p.m. Central on April 12, 2022

Title: Machine learning models identify histological features that can predict KEAP1 mutations in lung adenocarcinoma
Abstract Number: 5059
Session: Convergence Science and Systems Biology
Presentation Time: 12:00 – 1:00 p.m. Central on April 8, 2022

Additional information on the AACR (Free AACR Whitepaper) Annual Meeting 2022 is available through the AACR (Free AACR Whitepaper) website at: View Source

On March 9, 2022 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the fourth quarter and year ended December 31, 2021 and provided a business update (Press release, Protara Therapeutics, MAR 9, 2022, View Source [SID1234609790]).

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"Following a productive 2021, we are well poised to advance our pipeline in 2022, in particular, we are excited to have commenced our Phase 1 study of TARA-002 in non-muscle invasive bladder cancer (NMIBC), a significant step forward in our mission to bring a new immunotherapy to this patient population," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Discussions with the U.S. Food and Drug Administration (FDA) remain ongoing on the design of a clinical trial of TARA-002 in patients with lymphatic malformations (LMs), a rare pediatric indication for which there are currently no U.S. FDA-approved therapies. In addition, we continue to assess potential future indications and combinations for TARA-002."

Mr. Shefferman added, "Supported by a strong balance sheet, which includes ample runway to support our planned operations into mid-2024, we remain steadfast in our commitment to bringing meaningful new therapeutic options to pressing areas of high unmet need."

Recent Highlights

TARA-002 in NMIBC

In October 2021, the Company announced that the FDA cleared its Investigational New Drug (IND) application for TARA-002, an investigational cell-based therapy in development for the treatment of NMIBC. A Phase 1 clinical trial has commenced to assess the safety, tolerability, and preliminary signs of anti-tumor activity of TARA-002 in adults with high-grade NMIBC.
TARA-002 in LMs

In October 2021, the Company updated its IND submission for TARA-002 for the treatment of LMs with completed confirmatory, current Good Manufacturing Practices (cGMP) comparability data. The Company is engaged with the FDA to align on a development plan for TARA-002 in LMs.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)

The Company’s prospective prevalence study to enhance understanding of the incidence of IFALD and choline deficiency in patients dependent on parenteral nutrition remains ongoing. The Company plans to use results from the prospective study, as well as its previously completed retrospective study, to inform next steps for the IV Choline Chloride development program.
Corporate Updates

In January 2022, Protara announced the appointment of Jathin Bandari, M.D., as Chief Medical Officer. Dr. Bandari is a practicing urologic oncologist, recently serving at the University of Rochester where he specializes in both minimally invasive urologic oncology and advanced open pelvic retroperitoneal cancer surgery, and where he maintains a faculty appointment. Dr. Bandari joined Protara in April 2020 and most recently was Vice President, Head of Clinical Development, and Interim Chief Medical Officer.
Fourth Quarter and Full Year 2021 Financial Results

As of December 31, 2021, cash, cash equivalents and marketable debt securities totaled $130.7 million. The Company expects its cash, cash equivalents, and marketable debt securities will be sufficient to fund its planned operations into mid-2024.

Research and development expenses for the fourth quarter of 2021 increased to $4.1 million from $3.7 million for the prior year period, and for the full year increased to $21.1 million compared to $12.0 million for 2020. The fourth quarter and full year increases were primarily due to increases in non-clinical, clinical and regulatory expenses associated with TARA-002, headcount and stock-based compensation, and other employee-related expenses.

General and administrative expenses for the fourth quarter of 2021 increased to $6.2 million from $5.3 million for the prior year period, and for the full year increased to $26.4 million compared to $22.5 million for 2020. The fourth quarter and full year increases were due to increases in headcount and employee-related expenses, market development capabilities, and costs associated with the new office in New York.

For the fourth quarter of 2021, Protara reported a net loss of $10.2 million, or $0.91 per share, compared with a net loss of $8.8 million, or $0.79 per share, for the same period in 2020. Net loss for the year ended December 31, 2021 was $47.3 million, or $4.21 per share, compared with a net loss of $34.0 million, or $4.70 per share, for the year ended December 31, 2020. Net loss for the fourth quarter included approximately $2.0 million of stock-based compensation expenses. Net loss for the year ended December 31, 2021 included $10.4 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and LMs for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully demonstrated manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-6, IL-8, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, and vascular endothelial growth factor (VEGF) are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations (LMs)

LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of 3 years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride and Intestinal Failure-associated Liver Disease (IFALD)

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN) who have IFALD. Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. If approved, IV Choline Chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.

On March 9, 2022 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it will host a live conference call and webcast at 8:30 a.m. ET on Tuesday, March 15, 2022 to report its fourth quarter and full year 2021 financial results and provide a corporate update (Press release, Syros Pharmaceuticals, MAR 9, 2022, View Source [SID1234609789]).

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To access the live conference call, please dial (866) 595-4538 (domestic) or (636) 812-6496 (international) and refer to conference ID 9682507. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

On March 9, 2022 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T-cell receptor (TCR) engineered T cell therapies (TCR-T) for the treatment of patients with cancer, reported financial results for the full year ended December 31, 2021, and outlined key 2022 priorities (Press release, TScan Therapeutics, MAR 9, 2022, View Source [SID1234609788]).

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"As we had guided throughout 2021, in December we filed two INDs for our liquid tumor program. We also completed construction of our now fully functional 7,000 square-foot GMP manufacturing facility. Both of these steps are instrumental in our transformation to a clinical-stage company," said David Southwell, President and Chief Executive Officer. "We also advanced our growing pipeline of solid tumor candidates and look forward to sharing further details around these programs in the coming months."

Recent Corporate Highlights

In January 2022, the Company announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application to evaluate TSC-100 for the treatment of patients with hematologic malignancies who are undergoing allogeneic hematopoietic cell transplantation. The target of TSC-100 is the minor histocompatibility antigen HA-1, which is a lineage-specific antigen found on blood cells. The Company has now submitted the clinical protocol to Institutional Review Boards (IRBs) for the initial study sites and expects to initiate clinical trials in the first half of 2022.

In December 2021, the Company presented two posters related to its lead liquid tumor candidates TSC-100 and TSC-101 at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. The posters highlighted the discovery of TSC-101, as well as the manufacturing process and clinical development plan for liquid tumor candidates TSC-100 and TSC-101.

During the fourth quarter of 2021, the Company completed the construction of a state-of-the-art good manufacturing practices (GMP) facility to manufacture Phase I/II TCR-T therapies. This facility will support the manufacturing of TSC-100 and TSC-101, as well as solid tumor candidates TSC-200, TSC-201, TSC-202, TSC-203, and TSC-204, designed to treat patients with solid tumors including head and neck, cervical, melanoma and non-small cell lung cancers.

The Company continues to strengthen its management team with the appointment of Ray Lockard as Vice President, Quality. Mr. Lockard brings over 25 years of experience in quality, validation, supply chain and manufacturing to TScan. Mr. Lockard most recently served as Executive Director, CMC QA and External Quality at Ultragenyx Pharmaceutical Inc. Prior to that, Mr. Lockard held positions of increasing responsibility for various biotechnology and life sciences companies including Biogen Inc., Alnylam Pharmaceuticals, Inc., Precision NanoSystems, Editas Medicine, Inc., and AveXis, Inc. (now Novartis Gene Therapies). Mr. Lockard holds a B.S. in Biology from Hampden-Sydney College and an M.B.A. from the University of North Carolina, Chapel Hill, Kenan-Flagler Business School.
Upcoming Expected Milestones and Key Priorities for 2022

Liquid Tumor Programs: TScan’s two lead liquid tumor TCR-T therapy candidates, TSC-100 and TSC-101, are designed to target HA-1 and HA-2, respectively, and treat patients with hematologic malignancies who are undergoing allogeneic hematopoietic cell transplantation.

Initiate Phase 1 umbrella trial for TSC-100 following submission of clinical protocol to IRBs for the initial study sites, with plans to enroll patients in the first half of 2022.

As previously disclosed, the FDA placed a clinical hold on the IND for TSC-101 in January 2022. The Company has since received written communication from the FDA asking for additional assessment of the potential for off-tumor reactivity in certain tissues. TScan is working with the agency to resolve its questions as quickly as possible. The liquid tumor trial is based on an umbrella protocol, which will allow the Company to begin the TSC-100 and control arms in the near term and to open the TSC-101 arm of the ongoing trial upon clearance of the IND.

Present initial clinical data from the liquid tumor program at a medical meeting in the second half of 2022.
Solid Tumor Programs: TScan’s TSC-200 series of TCR-T therapy candidates include a combination of known targets, such as HPV16 for TSC-200, PRAME for TSC-203, and MAGE-A1 for TSC-204, as well as targets that are novel antigens for TCR-T therapy, such as those for TSC-201 and TSC-202.

Present initial preclinical data on the TSC-200 series at a medical meeting in the first half of 2022.

Progress IND-enabling studies for the TSC-200 series and submit two IND applications during the second half of 2022. These are expected to include TSC-200 for HPV and TSC-204 for MAGE-A1.

In 2023, the Company plans to release initial clinical data for the TSC-200 series TCRs, as well as file further INDs for additional programs in this series.
Infectious Disease Program

Research is continuing into potential T cell focused COVID-19 vaccine constructs utilizing TScan’s novel T cell target discoveries. The Company is currently conducting preclinical studies for this program.
Full Year 2021 Financial Results

As of December 31, 2021, TScan Therapeutics had cash and cash equivalents of $161.4 million excluding $5.0 million of restricted cash. Based on current operating plans, the Company believes that existing cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into 2024.

Revenue for the year ended December 31, 2021, was $10.1 million, compared to $1.1 million for the year ended December 31, 2020 (2020 Period). This increase is due to a full year of research activities related to TScan’s collaboration agreement with Novartis Institutes for Biomedical Research, on which work began in September 2020.

Research and development expenses for the year ended December 31, 2021, were $45.0 million, compared to $20.6 million for the 2020 Period. The increase of $24.4 million was primarily a result of increased personnel expenses and facility-related expenses, as well as expenses related to the progress of the Company’s liquid and solid tumor programs to IND filing.

General and administrative expenses for the year ended December 31, 2021, were $13.8 million, compared to $6.7 million for the 2020 Period. The increase of $7.1 million in general and administrative expenses was primarily a result of increased personnel expenses and other miscellaneous expenses, including expenses related to the IPO and status as a public company.

For the year ended December 31, 2021, TScan Therapeutics reported a net loss of $48.6 million, compared to a net loss of $26.1 million for the 2020 Period.

As of December 31, 2021, the company had issued and outstanding shares of 24,024,467 and 23,907,597, respectively.

On March 9, 2022 AIM ImmunoTech Inc. (NYSE: American AIM) ("AIM" or the "Company"), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases, including COVID-19, the disease caused by the SARS-CoV-2 virus, reported that oncology experts from Roswell Park Comprehensive Cancer Center will present findings from two ongoing clinical studies involving an AIM ImmunoTech Inc (Press release, AIM ImmunoTech, MAR 9, 2022, View Source [SID1234609787]). drug candidate, Ampligen (also known as rintatolimod) at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, being held April 8-13, 2022, in New Orleans, Louisiana.

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Research led by Roswell Park medical oncologists Shipra Gandhi, MD, and Sarbajit Mukherjee, MD, MS, in collaboration with senior investigator Pawel Kalinski, MD, PhD, Chair of Immunology at Roswell Park, has been accepted for presentation as late-breaking poster abstracts at the meeting.

Details of the abstract and late-breaking poster presentations are as follows:

Title: Initial results of a phase II study evaluating a chemokine-modulatory (CKM) regimen in patients with colorectal cancer metastatic to the liver
Presenter: Sarbajit Mukherjee, MD, MS
Abstract Number: 22-LB-7312
Session: Phase II Clinical Trials 1, presentation CT105
Presentation Type: Late-breaking poster presentation
Session Date and Time: Monday, April 11, 2022, from 9 a.m. 12:30 p.m. CDT
Location: Poster 2, Section 33

Title: Systemic Rintatolimod and Interferon-α2b selectively reprogram local tumor microenvironment in patients with metastatic triple negative breast cancer for enhanced influx of cytotoxic T-lymphocytes but not regulatory T-cells
Presenter: Shipra Gandhi, MD
Abstract Number: 22-LB-7620
Session: Phase I Clinical Trials 1, presentation CT145
Presentation Type: Late-breaking poster presentation
Session Date and Time: Monday, April 11, 2022, from 1:30 to 5 p.m. CDT
Location: Poster 12, Section 35

The late-breaking and clinical trials abstract titles and authors are now available on the AACR (Free AACR Whitepaper) Online itinerary planner. The late-breaking and clinical trials abstract text is under embargo until April 8, 2022 at 1:00 p.m. ET.