1stOncology™: Cancer Intelligence Service – Page 8459 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Vaccinex Reports Third Quarter 2021 Financial Results and Provides Corporate Update

On November 8, 2021 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, reported financial results for the third quarter ended September 30, 2021 and provided a corporate update (Press release, Vaccinex, NOV 8, 2021, View Source [SID1234594781]).

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!

"We made steady progress on our pepinemab clinical programs since our last quarterly update. Enrollment is now underway in the Phase 1b/2 head and neck cancer trial and the Phase 1/2a Alzheimer’s disease trial, two of the serious indications in which SEMA4D is overexpressed and is believed to contribute to disease pathology," stated Maurice Zauderer, Ph.D., President and Chief Executive Officer.

"As presented at the September European Huntington’s Disease Network meeting, we believe post-hoc analysis of the Phase 2, SIGNAL trial of pepinemab in patients with early manifest Huntington’s disease (HD) supports the potential cognitive benefit of treatment with pepinemab in HD patients, particularly those with mild advanced disease. We are engaged in discussions with potential partners for the advancement of this important program."

Pepinemab Clinical Updates:

Head and Neck Cancer. Enrollment is underway in the Phase 1b/2 clinical trial evaluating pepinemab in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in advanced recurrent or metastatic head and neck cancer.

The study will enroll up to 65 subjects across 18 U.S. trial sites and will assess whether combination therapy can improve responses in this population. Key endpoints of the study will include objective response, progression free survival and overall survival. Vaccinex anticipates data from this study in the second half of 2022.

Multiple prior studies suggest that inhibition of SEMA4D increases immune infiltration and alters the balance of cytotoxic and immunosuppressive cells in the tumor microenvironment. As SEMA4D is highly expressed in head and neck cancer, there is strong rationale for development in this indication.

Alzheimer’s Disease. Patient screening and enrollment have been initiated in the Phase 1/2a clinical trial of pepinemab in Alzheimer’s disease. The Alzheimer’s trial is being funded in part by the Alzheimer’s Drug Discovery Foundation by the Alzheimer’s Association under the 2020 Part the Cloud Program.

The randomized, double-blind, placebo-controlled, multi-center safety and biomarker study of pepinemab in early AD is planned to enroll 40 subjects across 14 U.S. trial sites. Vaccinex anticipates topline data from this study in late 2022 or early 2023.
Huntington’s disease. We believe that post-hoc analysis of the Phase 2, double-blind, placebo-controlled SIGNAL trial of pepinemab in patients with early manifest Huntington’s disease (HD) supports the potential cognitive benefit of treatment with pepinemab in HD patients, particularly those with mild cognitive deficits.
Highly significant improvement (p=0.007) in the (Huntington’s Disease Cognitive Assessment Battery (HD-CAB) Composite score, a widely employed measure comprised of 6 different cognitive assessments.
Significant benefit in reducing apathy severity (p=0.017, 1-sided), a problem behavior that has previously been correlated with cognition in both HD and AD.
Striking increase in brain metabolic activity as measured by FDG-PET in most brain regions. Decline in FDG-PET signal has been reported to correlate with cognitive decline in several studies of AD.
The company continues to actively explore advancing pepinemab into a Phase 3 HD trial in collaboration with a biopharmaceutical partner; discussions are ongoing.

Other Trials. Pepinemab is also being evaluated in multiple investigator-sponsored trials (ISTs) being conducted by the Winship Cancer Institute of Emory University to evaluate pepinemab in combination with checkpoint inhibitors in "Window of Opportunity" studies in head and neck cancer and melanoma.
Upcoming Anticipated Milestones:

H2:2022– Meaningful data from open label head and neck cancer trial
Late 2022/Early 2023 – Topline data from randomized Alzheimer’s trial
ActivMAb Updates:

As previously announced, we have entered into several collaborations with pharmaceutical and biotechnology companies employing the unique capabilities of our ActivMAb antibody discovery platform to address difficult to drug G-protein Coupled Receptors (GPCRs) known to be strongly associated with diseases. "We believe this enabling technology will allow us and our collaborators to address significant market opportunities," said Ernest S. Smith, Ph.D., Chief Scientific Officer.

Financial Results for the Three Months Ended September 30, 2021:

Research and Development Expenses. Research and development expenses for the three months ended September 30, 2021 were $3.6 million as compared to $7.3 million for the comparable period in 2020.

General and Administrative Expenses. General and administrative expenses for the three months ended September 30, 2021 were $1.5 million as compared to $1.9 million for the comparable period in 2020.

Cash and Cash Equivalents and Marketable Securities. Cash and cash equivalents and marketable securities on September 30, 2021 were $13.8 million, as compared to $10.6 million as of December 31, 2020.

RAPT Therapeutics to Participate in Upcoming Investor Conferences

On November 8, 2021 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology, reported that Brian Wong, M.D., Ph.D., President and CEO, will present at the following investor conferences in November (Press release, RAPT Therapeutics, NOV 8, 2021, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-participate-upcoming-investor-conferences [SID1234594780]):

Stifel 2021 Virtual Healthcare Conference – Company presentation on Monday, November 15, 2021, at 2:00 p.m. ET
Piper Sandler 33rd Annual Virtual Healthcare Conference – Pre-recorded fireside chat available on-demand Monday, November 22, 2021, at 10:00 a.m. ET
To access the live webcast or subsequent archived recordings of the company presentations, please visit the RAPT Therapeutics website at https://investors.rapt.com/events-and-presentations.

Tempest Further Bolsters Patent Portfolio with Issuance of Multiple Patents for TPST-1495

On November 8, 2021 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage oncology company developing potentially first-in-class therapeutics that combine both targeted and immune-mediated mechanisms, reported that the U.S. Patent and Trademark Office has issued multiple patents covering composition of matter for the company’s therapeutic product candidate TPST-1495, an orally available small molecule designed to selectively block the EP2 and EP4 receptors in the prostaglandin (PGE2) pathway (Press release, Tempest Therapeutics, NOV 8, 2021, View Source [SID1234594779]).

"We have shown in pre-clinical studies that our approach to inhibit signaling through both EP2 and EP4 receptors with our TPST-1495 dual antagonist confers significantly increased potency compared to single EP4 inhibitors, as well as improvement over other prostaglandin-inhibition approaches including NSAIDs, and could represent an important evolution in the targeting of this pathway in oncology," said Tom Dubensky, president of Tempest. "Prostaglandin signaling drives a wide range of cancers as well as resistance to immune checkpoint inhibitor therapies, so we are excited about the potential of our ongoing clinical evaluation of TPST-1495. The intellectual property portfolio covering TPST-1495 and its uses is important to protect the differentiated properties of this molecule."

The patent portfolio now includes U.S. Patent Numbers 10,968,201 and 11,066,405, as well as a number of pending applications in the United States and other markets outside of the United States. The pending applications cover compositions of matter and methods of use, which further strengthen Tempest’s intellectual property for the combined inhibition of EP2 and EP4 prostaglandin PGE2 receptor signaling.

About TPST-1495

TPST-1495 is an orally-available small molecule designed to block the EP2 and EP4 receptors in the prostaglandin (PGE2) pathway, while sparing the homologous but differentially active EP1 and EP3 receptors. PGE2 signaling through EP2 and EP4 has been observed both to enhance tumor progression and promote immune suppression. Tempest has conducted head-to-head preclinical studies comparing TPST-1495 to single antagonists of EP2 and EP4 and observed significantly enhanced activity of TPST-1495 in both overcoming PGE2-mediated suppression of human immune cells in vitro, as well as significantly increased anti-tumor activity in mouse models of human colorectal cancer. Tempest is currently evaluating the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and possible anti-tumor activity of TPST-1495 in a multicenter Phase 1a/1b dose and schedule optimization study in subjects with advanced solid tumors, with the potential to expand in indications known to be prostaglandin-driven, including colorectal cancer, or CRC, and in a tumor indication-agnostic, biomarker-selected cohort.

Bayer Pharmaceuticals and The Life Raft Group, a Non-Profit, Announce Innovative Collaboration to Accelerate Cancer Research for GIST Patients

On November 8, 2021 Bayer Pharmaceuticals, working with The Life Raft Group (LRG), reported that they have extended a research collaboration to broaden access to comprehensive genomic testing for Gastrointestinal Stromal Tumor (GIST) patients (Press release, Bayer, NOV 8, 2021, View Source [SID1234594768]). This collaboration aims to advance precision medicine in oncology and use comprehensive genomic testing to identify patients in the United States (U.S.) who may benefit from personalized care.

Comprehensive genomic testing, also known as biomarker testing or molecular testing, is a crucial step in understanding the genomic factors that play a role in establishing a GIST diagnosis. There are different types of comprehensive genomic testing: the most common tests are basic mutational testing and advanced next-generation sequencing. These tests may help identify alterations, or changes, within DNA and/or RNA of cancer cells that determine how a tumor behaves or why it grows. Basic mutational testing focuses on testing specific genes like c-KIT, PDGFRA, and BRAF. Advanced next-generation sequencing may test a broader range of genes, usually between 5 – 500+ genes, including neurotrophic receptor tyrosine kinase (NTRK) gene fusions. The results can help healthcare providers match patients with available treatment options or clinical trials. Finding the right therapeutic can be a turning point in the treatment journey of a GIST patient.

GISTs are a type of soft tissue sarcoma – cancers that develop in connective or supporting tissues – that affect an estimated 3,000 to 5,000 people in the U.S. each year.1 One genomic alteration that can drive the growth and spread of GIST is called an NTRK gene fusion. There are currently treatments for GIST driven by NTRK gene fusions approved in the U.S.

Criteria for patients to participate in this program include: be a U.S. resident, be or become a part of The Life Raft Group GIST Patient Registry, have a treating oncologist, must not have had any type of prior comprehensive genomic testing (basic mutational testing or next-generation sequencing), or have previously been identified with wildtype c-KIT or PDGFRA results from basic mutational testing.

The Life Raft Group, based in Wayne, New Jersey, is a global leader in GIST, focused on educating patients, providing resources, and launching initiatives for the community for better patient outcomes. This collaboration is an example of the vital role patient advocacy groups play in bridging the gap between researchers and motivated patient populations willing to be part of innovative studies.

"As part of our commitment to precision oncology, we believe identifying NTRK patients early through comprehensive genomic testing is a critical step in the cancer diagnosis of oncology patients experiencing metastatic disease, as it helps physicians understand the underlying drivers of tumor growth and can inform the treatment approach," said Iain Webb, M.D., Vice President, U.S. Medical Affairs, Oncology at Bayer. "That is why we are pleased to collaborate with The Life Raft Group to increase the availability of comprehensive genomic testing for GIST patients to potentially uncover NTRK gene fusions and provide options for those who may benefit from precision oncology treatments."

This collaboration builds on Bayer’s existing commitment to advance precision oncology, which includes research to find targets and pathways that drive cancer growth, and how these findings can impact the way cancer is treated.

"We are excited to be a part of this groundbreaking opportunity to work with great organizations to pave the road towards precision medicine and to help us to reach our goal of increasing the testing rate among GIST patients," said Denisse Montoya, Patient Registry Director at The Life Raft Group. "By offering comprehensive genomic testing with no charge, many patients will have the opportunity to know what genes are driving their GIST and benefit from genetic-informed care. Results from this testing collaboration will help us understand more traits and patterns within GIST that will help us with our aim of accelerating GIST cancer research."

Healthcare professionals with eligible patients can find additional information here.

Merck Announces Receipt of Antitrust Clearance in Germany and Austria Relating to Tender Offer to Acquire Acceleron Pharma Inc.

On November 8, 2021 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported that its pending acquisition of Acceleron Pharma Inc. (Nasdaq: XLRN) has been cleared by the competition authorities in Germany and Austria (Press release, Merck & Co, NOV 8, 2021, View Source [SID1234594755]).

As previously announced on October 12, 2021, Merck commenced, through a subsidiary, a cash tender offer to purchase all outstanding shares of common stock of Acceleron, for $180 in cash, without interest and less any required tax withholding. The receipt of antitrust clearance from the competition authorities in Germany and Austria satisfies one of the conditions necessary for the consummation of the tender offer. Consummation of the tender offer remains subject to other conditions described in the tender offer statement on Schedule TO filed with the U.S. Securities and Exchange Commission (the "SEC") on October 12, 2021, including the termination or expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended ("HSR") and the tender of shares representing at least a majority of the total number of Acceleron’s outstanding shares. As previously announced on October 29, 2021, the tender offer will expire at 5:00 p.m., Eastern Time, on November 18, 2021, unless further extended in accordance with the merger agreement and the applicable rules and regulations of the SEC. The acquisition is expected to close in the fourth quarter of 2021.

The Depositary for the tender offer is Computershare Trust Company, N.A., c/o Voluntary Corporate Actions, P.O. Box 43011, Providence, RI 02940-3011. The Information Agent for the tender offer is Innisfree M&A Incorporated, 501 Madison Avenue, 20th floor, New York, NY 10022. The tender offer materials may be obtained at no charge by directing a request by mail to Innisfree M&A Incorporated or by calling toll free at (877) 800-5195, and may also be obtained at no charge at the website maintained by the SEC at www.sec.gov.