On January 25, 2022 Lineberger Comprehensive Cancer Center reported An anonymous donor has made a $25 million gift to establish the UNC Lineberger Center for Triple Negative Breast Cancer and to support other key UNC Lineberger initiatives (Press release, Lineberger Comprehensive Cancer Center, JAN 25, 2022, View Source [SID1234606783]). This is the largest donation in UNC Lineberger’s history, and it enables the cancer center to advance its groundbreaking research on diagnosing and treating a highly aggressive breast cancer that disproportionately affects Black, Latina and young women and historically has limited research funding.

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The gift was made in gratitude for the care a family member received while being treated for cancer at UNC, and to help expand and expedite the cutting-edge cancer research being conducted at UNC Lineberger. Specifically, the donor designated their investment to help women and men with all types of breast cancer, especially triple negative breast cancer because of its poor prognosis. In addition, the gift will support research directed toward developing more effective treatments for metastatic disease, improving pediatric cancer care, and eliminating racial disparities in cancer treatment outcomes.

Lisa A. Carey, MD, MSc, FASCO, will serve as the inaugural director of the UNC Triple Negative Breast Cancer Center. Carey, the Richardson and Marilyn Jacobs Preyer Distinguished Professor in Breast Cancer Research and a medical oncologist who specializes in treating breast cancer patients, said it is hard to overestimate the gift’s potential impact on advancing triple negative breast cancer research and care.

"While research advances the past 30 years have led to new and more effective treatments for many types of breast cancer, this isn’t the case with triple negative breast cancer," said Carey, who, in addition to her clinical responsibilities, is the deputy director of clinical sciences and co-leader of the breast cancer research program at UNC Lineberger. "The good news is this gift will be a game changer. It provides the cancer center with the resources to expand and speed the pace of our research focused on generating insights that lead to better treatments and outcomes for women with triple negative breast cancer."

Accounting for roughly 10-20% of breast cancer cases in the United States, triple negative breast cancer is so named because it lacks the estrogen, progesterone and HER2 protein receptors commonly associated with other breast cancers. It is an aggressive, fast-growing cancer that has a high risk of spreading beyond the breast and of recurring despite treatment. It has significantly poorer outcomes than other breast cancers, and it disproportionally affects Black, Latina and young women. The only current standard of care involves chemotherapy.

In addition to establishing this new research center, the money will create multiple professorships and accelerate three strategic research initiatives that build on existing UNC Lineberger strengths:

Developing new treatments particularly those that harness a patient’s immune system, including chimeric antigen receptor t-cell (CAR-T) immunotherapy. The objective is more personalized, more effective and less toxic treatment than currently available;
Expanding the genetic understanding and classification of cancer types to improve diagnostics and uncover new targets and modes of therapy;
And creating greater knowledge of nutrition and metabolism and their impact on disease prevention and more holistic treatment options.
"Our world-class researchers at the UNC Lineberger Comprehensive Cancer Center are applying innovative approaches to solving some of the grand challenges of our time," said UNC-Chapel Hill Chancellor Kevin M. Guskiewicz. "Our experts have done foundational work, especially in the space of triple negative breast cancer. Under Lisa Carey’s leadership, they are uniquely positioned to make the most of this generous gift and find life-saving treatments that improve health outcomes for patients in North Carolina and beyond."

Foundational research at UNC Lineberger

UNC Lineberger is internationally recognized for its foundational discoveries that have advanced the field of triple negative breast cancer research.

In 2000, Charles Perou, PhD, the May Goldman Shaw Distinguished Professor of Molecular Oncology and co-leader of the UNC Lineberger breast cancer research program, published a groundbreaking paper identifying the molecular subtypes of breast cancer and demonstrating that breast cancer was not one disease but a collection of diseases with different prognoses. Specifically, the findings defined and characterized the dominant biology underlying triple negative breast cancer.

In 2001, Carey and her UNC Lineberger colleagues launched the first clinical trial in the U.S. that targeted triple negative breast cancers. Through its longstanding population-based study, the Carolina Breast Cancer Study, UNC Lineberger researchers also were the first to demonstrate that triple negative breast cancer disproportionately affected Black women, particularly young black women.

"UNC Lineberger has a world-leading record of advancing understanding of triple negative breast cancer and its therapy," said H. Shelton Earp, MD, UNC Lineberger director. "This remarkable gift will enable us to create an unmatched national hub of research excellence combining and enhancing our expertise in genomics, immunotherapy, and cancer nutrition towards more effective and less toxic therapy for advanced triple negative breast cancer, and with the knowledge gained and our world class community engagement team, better prevention, early detection, and timely therapy for all North Carolina’s rural and urban populations."

"This magnanimous gift is both inspiring and transformative, and it will be life-saving," said A. Wesley Burks, MD, dean of the UNC School of Medicine, vice chancellor for medical affairs, and CEO of UNC Health. "In addition to building on the depth of our expertise in cancer research and care, this gift enables us to focus on uncovering what makes some cancers so difficult to treat and identifying the drivers of racial disparities in cancer treatment outcomes. This is critically important work."

On January 25, 2022 Sierra Oncology, Inc. (NASDAQ: SRRA), a late-stage biopharmaceutical company dedicated to delivering targeted therapies for rare cancers, reported it has entered into a term loan agreement with Oxford Finance, LLC that is designed to primarily support the commercial preparation and potential launch of momelotinib, an investigational agent for the treatment of myelofibrosis, a rare form of blood cancer (Press release, Sierra Oncology, JAN 25, 2022, View Source [SID1234606782]). The Company estimates it had cash and cash equivalents of $104.7 million as of December 31, 2021.

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Stephen Dilly, MBBS, PhD, President and Chief Executive Officer of Sierra Oncology, said, "Our strong cash position together with the term loan facility with Oxford create great financial optionality for us as we move towards an NDA filing and potential approval of momelotinib."

Under the terms of the loan agreement with Oxford Finance, LLC, Sierra drew an initial $5 million term loan at closing. The company has the ability to access up to an additional $120 million in a series of tranches, $70 million of which are based on certain pre-determined milestones, including US regulatory approval and financing, and $50 million is at the lender’s discretion. In addition, the Company’s Series B Warrants expire 75 days from today, and if fully exercised, would provide a total of $33.3 million in proceeds to the company.

About Momelotinib

Momelotinib is a potent, selective and orally bioavailable JAK1, JAK2 and ACVR1 / ALK2 inhibitor currently under investigation for the treatment of myelofibrosis. Myelofibrosis results from dysregulated JAK-STAT signaling and is characterized by constitutional symptoms, splenomegaly (enlarged spleen) and progressive anemia.

Sierra Oncology announced topline results of the Pivotal Phase 3 MOMENTUM clinical trial, a global, randomized, double-blind study evaluating momelotinib for the treatment of symptomatic and anemic myelofibrosis patients, on January 25, 2022. The company plans to submit a New Drug Application with the US Food & Drug Administration (FDA) in the second quarter of 2022. The FDA has granted Fast Track designation for momelotinib.

On January 25, 2022 ITM Isotope Technologies Munich SE (ITM), a leading radiopharmaceutical biotech company, reported that the first patient has been treated in its second pivotal phase III clinical trial, COMPOSE (NCT04919226), evaluating the company’s lead radiopharmaceutical candidate, ITM-11 (n.c.a. 177Lu-edotreotide), for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs) (Press release, ITM Isotopen Technologien Munchen, JAN 25, 2022, View Source [SID1234606781]). ITM-11 is a Targeted Radionuclide Therapeutic consisting of the high-quality radioisotope no-carrier-added lutetium-177 (n.c.a. 177Lu) fused with a somatostatin analogue to specifically target somatostatin receptor-positive (SSTR+) GEP-NETs. While COMPOSE is evaluating ITM-11 for the treatment of well-differentiated high grade 2 and grade 3 GEP-NETs, the radiopharmaceutical is also being investigated in ITM’s ongoing pivotal phase III trial, COMPETE (NCT03049189), in patients with grade 1 and 2 GEP-NETs. GEP-NETs are rare types of tumors that can occur in the pancreas or in other parts of the gastrointestinal tract. Due to their frequent asymptomatic and progressive nature, GEP‑NETs often present late with advanced disease requiring innovative therapeutic measures. The trial design of COMPOSE was recently presented at the 2022 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO-GI) and the 2021 North American Neuroendocrine Tumor Society (NANETS) annual symposium.

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"We are committed to providing urgently needed solutions for the treatment of GEP-NETs which are often diagnosed with advanced disease," commented Steffen Schuster, Chief Executive Officer of ITM. "As such, we hope to build upon previous promising data to demonstrate in COMPETE and COMPOSE that Targeted Radionuclide Therapy with ITM-11 has the potential to improve treatment outcomes and quality of life for a broad patient population."

"Targeted Radionuclide Therapy is a promising therapeutic concept that enables a precise intervention both for the primary tumor as well as for metastases. N.c.a. 177Lu-edotreotide has demonstrated potential in earlier stage GEP-NET patients, and I look forward to evaluating it in a more advanced late-stage population with higher tumor grade in high need of better therapeutic options," added Prof. Walter, Principal Investigator of COMPOSE at Hospices civils de Lyon, France.

COMPOSE (NCT04919226) is an international, prospective, randomized, controlled, open-label, multi-center phase III clinical trial to evaluate the efficacy, safety, and patient-reported outcomes of first- or second-line treatment with ITM-11 (n.c.a. 177Lu-edotreotide) compared to best standard of care in patients with well-differentiated high grade 2 and grade 3 (Ki-67 index 15-55), SSTR+, GEP-NETs. The study aims to randomize 202 patients 1:1 to ITM-11 or to best standard of care — either chemotherapy (CAPTEM or FOLFOX) or everolimus — according to the investigator’s choice. The primary endpoint of the study is progression-free survival, which will be assessed every 12 weeks from randomization onwards. Secondary outcome measures include overall survival up to two years after disease progression. Sponsor of the COMPOSE trial is ITM Solucin GmbH, a subsidiary of ITM Isotope Technologies Munich SE.

– End –

About Targeted Radionuclide Therapy

Targeted Radionuclide Therapy is an emerging class of cancer therapeutics, which seeks to deliver radiation directly to the tumor while minimizing radiation exposure to normal tissue. Targeted radiopharmaceuticals are created by linking a therapeutic radioisotope to a targeting molecule (e.g., peptide, antibody, small molecule) that can precisely recognize tumor cells and bind to tumor-specific characteristics, such as receptors on the tumor cell surface. As a result, the radioisotope accumulates at the tumor site and decays, releasing a small amount of ionizing radiation, thereby destroying tumor tissue. The highly precise localization enables targeted treatment with minimal impact to healthy surrounding tissue.

About ITM-11 (n.c.a. 177Lu-edotreotide)

ITM-11, ITM’s therapeutic radiopharmaceutical candidate being investigated in the phase III clinical studies COMPETE and COMPOSE, consists of two components: the medical radioisotope no-carrier-added lutetium-177 (n.c.a. 177Lu) and the targeting molecule edotreotide, a synthetic form of the peptide hormone somatostatin that targets neuroendocrine tumor-specific receptors. Edotreotide binds to these receptors and places the medical radioisotope n.c.a. lutetium-177 directly onto the diseased neuroendocrine cells so that it accumulates at the tumor site. N.c.a. lutetium-177 is internalized into the tumor cells and decays, releasing medical radiation (ionizing β-radiation) with a maximum radius of 1.7 mm and destroying tumor tissue. The highly precise localization can result in the healthy tissue surrounding the targeted tumor being minimally affected.

On January 25, 2022 MiNA Therapeutics Limited ("MiNA" or the "Company"), the pioneer in small activating RNA (RNAa) therapeutics, reported that it has dosed the first patient in a global Phase 2 clinical trial (OUTREACH-2) of MTL-CEBPA in combination with second line standard of care sorafenib (a tyrosine kinase inhibitor (TKI)) in advanced hepatocellular carcinoma (HCC or liver cancer) (Press release, MiNA Therapeutics, JAN 25, 2022, View Source [SID1234606780]).

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OUTREACH-2 is a multi-centre, open-label, randomised study of MTL-CEBPA in combination with sorafenib, compared to sorafenib alone, in TKI-naïve advanced pre-treated HCC patients with viral hepatitis etiology. The study will recruit up to 150 patients globally from centres in the US, Europe, and Asia. OUTREACH-2 is designed to assess further to what extent the MTL-CEBPA and sorafenib combination offers therapeutic advantage compared to sorafenib alone for the treatment of advanced HCC. The study’s primary endpoint is progression-free survival by blinded radiological assessment and the study is expected to complete in the first quarter of 2024. If the data from this Phase 2 OUTREACH-2 study are satisfactory to the US Food and Drug Administration (FDA), this could enable an Accelerated Approval of MTL-CEBPA in combination with sorafenib soon thereafter.

Professor Tim Meyer, Professor of Experimental Cancer Medicine at University College London, and Chief Investigator of the study, commented:
"Advanced liver cancer remains a significant unmet medical need, in particular for those patients who are resistant to front line systematic therapy. This combination treatment demonstrated intriguing signals of activity in a Phase 1b trial, including durable and complete tumour responses. We believe that MTL-CEBPA’s immunological activity in the tumour microenvironment enables a greater effectiveness of sorafenib and we are excited to seek to validate those early findings in this Phase 2 clinical trial."

Robert Habib, CEO of MiNA Therapeutics, commented:
"We are very excited to dose our first patient in the OUTREACH-2 study, which is the first Phase 2 clinical trial of a RNAa therapeutic. MTL-CEBPA has demonstrated its potential to make tumours more susceptible to established anti-cancer therapies, which can significantly improve treatment outcomes for patients. We look forward to building on data from our successful Phase 1b study of the sorafenib combination and to developing MTL-CEBPA more broadly for the benefit of patients."

The study builds on the successful proof-of-concept data from MiNA’s first-in-human, Phase 1b clinical trial (OUTREACH) evaluating the safety and tolerability of the therapeutic combination in patients with advanced HCC. The clinical activity observed in OUTREACH, which included durable and complete tumour responses not common with sorafenib alone, suggested that MTL-CEBPA may increase the effectiveness of sorafenib as a second line standard of care for HCC. The OUTREACH study showed that MTL-CEBPA plus sorafenib achieved a complete response rate (CRR) of 13% and an overall response rate (ORR) of 27% in the target population. By comparison sorafenib alone achieved a CRR of <1% and an ORR of 5-11% in recent published studies. The trial protocol has been accepted in an investigational new drug (IND) application by the FDA. The FDA has also granted Orphan Drug Designation for MTL-CEBPA in combination with sorafenib for the treatment of HCC of viral hepatitis etiology who have progressed following prior therapy. As a monotherapy, MTL-CEBPA achieved a CRR of 2% and an ORR of 6%, despite not being intended to target the tumours directly.

As the first ever RNAa therapy to enter the clinic, MTL-CEBPA is being studied as a combination therapy in cancer. The mechanism of action of MTL-CEBPA is to reduce or remove one of the main defence mechanisms by which tumours can resist the immune system, thereby opening the tumour to attack by the immune system and tumour-targeting drugs. In combination, data suggests that the drug can significantly improve the effectiveness of established cancer treatments by altering the tumour microenvironment in favour of those treatments. It achieves this by using the RNA activation mechanism to boost and restore expression of the C/EBP-α protein to normal levels which, in turn, reduces immune suppression by myeloid cells in which this protein has been down-regulated by the tumour. The drug candidate is also being investigated in an investigator-sponsored Phase 1a/1b study in first-line HCC in combination with first-line standard of care combination atezolizumab and bevacizumab in collaboration with F Hoffman-La Roche Ltd, as well as in an ongoing multi-centre Phase 1b clinical trial in patients with a variety of advanced solid tumours in combination with pembrolizumab, a PD-L1 inhibitor.

About MTL-CEBPA
MTL-CEBPA is the first therapy that specifically up-regulates CCAAT/enhancer binding protein alpha (C/EBP-α), a transcription factor that acts as a master regulator of myeloid cell lineage determination and differentiation. Dysregulated myeloid cells have been implicated in several diseases and in solid tumour cancers these cells have been identified as a critical barrier to induction of clinical response for many therapies. In pre-clinical studies MTL-CEBPA has been shown to improve the anti-tumour activity of cancer therapies by targeting dysregulated myeloid cells and reducing or eliminating their suppressive effect on immune response and therapies in the tumour micro-environment. MTL-CEBPA is currently in clinical development in three different studies as a combination therapy for the treatment of both first- and second-line advanced liver cancer and for a variety of other advanced solid tumour malignancies.

On January 25, 2022 West Pharmaceutical Services, Inc. (NYSE: WST), a global leader in innovative solutions for injectable drug administration, reported an exclusive supply and technology agreement with Corning Incorporated (NYSE: GLW) (Press release, West Pharmaceutical Services, JAN 25, 2022, View Source [SID1234606779]). The new collaboration includes a multimillion-dollar investment to expand Corning’s Valor Glass technology to enable advanced injectable drug packaging and delivery systems for the pharmaceutical industry with the goal of advancing patient safety and expanding access to life-saving treatments.

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By combining West’s industry-leading NovaPure components, with Daikyo Flurotec coating technology, and Corning’s Valor Glass and Velocity Vials, the strategic collaboration will enable new, advanced pharmaceutical packaging solutions. The collaboration optimizes the materials science and manufacturing expertise of both companies to help bio-pharma producers navigate the complex regulatory environment and mitigate risk in bringing drugs to market.

"West and Corning have developed this exceptional collaboration to offer leading elastomer-glass system solutions for the containment and delivery of injectable medicines," said Eric M. Green, President and Chief Executive Officer, West. "At West, we value the state-of-the-art products offered by Corning and are excited to build together the next generation of integrated packaging and delivery innovations."

This portfolio of new elastomer-glass system offerings and supportive data services will utilize West’s NovaPure components along with Corning’s Valor Glass technology. West’s NovaPure components adhere to Quality by Design principles and processing, representing the highest standards in pharmaceutical components. For customers, it provides the assurance of components with the tightest specifications applicable within today’s formulation and manufacturing process capabilities at West. Corning’s Valor Glass is intended to enhance the storage and delivery of drugs, providing more reliable access to state-of-the-art medicines essential to public health. Valor Glass packaging can enable increased throughput and offers high chemical durability, strength, and damage resistance.

"This partnership and investment enable the development and industry-leading solutions that enhance patient safety, increase quality and reliability in highly regulated markets, and ensure greater capacity for life-saving drugs. West is a great partner. The combination of Corning’s Valor Glass, along with West’s NovaPure components, is a win-win for customers and patients all over the world," said Wendell P. Weeks, Chairman and Chief Executive Officer, Corning.