On August 4, 2022 Bio-Teche Corporation (NASDAQ: TECH) reported Chuck Kummeth’s planned two-year transition from his position as Bio-Techne’s Chief Executive Officer (CEO) effective June 30, 2024 (Press release, Bio-Techne, AUG 4, 2022, View Source [SID1234617452]). The Company’s Board of Directors has begun its search for Mr. Kummeth’s replacement, evaluating both internal and external candidates to assume the CEO position upon Kummeth’s retirement at the end of its fiscal 2024. It is anticipated that Mr. Kummeth will continue to serve on Bio-Techne’s Board of Directors upon his retirement.

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!

Mr. Kummeth has served as Chief Executive Officer and as a member of the Company’s Board of Directors since April 2013. Under Mr. Kummeth’s leadership, the Company delivered on key strategic initiatives to accelerate its organic growth rate, expand its total addressable market and fortify its position as a leader in the life science tools and diagnostics markets. During Kummeth’s tenure as CEO, Bio-Techne grew its revenue from approximately $311 million in fiscal 2013 to $1.1 billion in fiscal 2022, representing a compounded annual growth rate (CAGR) of over 15%. Additionally, the Company grew from approximately 800 to over 3,000 employees, expanded its product portfolio and total addressable market through the completion of 17 acquisitions, established a best-in-class executive leadership team, and implemented processes and procedures to drive future growth.

"During the last nine years, Chuck has done an extraordinary job accelerating growth, building a strong leadership team and positioning Bio-Techne for the future," said Robert Baumgartner, Bio-Techne’s Chairman of the Board of Directors. "Throughout his tenure as CEO, Chuck and the Board have been focused on building management bench strength. Chuck has successfully built an extremely strong leadership team, including a deep bench of experienced and results-driven executives. Additionally, the Company’s enviable position as a rapidly growing, leading life science tools and diagnostics company positions Bio-Techne to attract top-caliber external candidates. With Chuck at the helm for the next two years, we have time for a thorough evaluation of potential candidates and a seamless transition to his eventual successor."

"I am looking forward to continuing to lead the Company and execute our strategic plan over the next two years. As a Board member, I will play an instrumental role in choosing my successor," commented Chuck Kummeth, President and Chief Executive Officer of Bio-Techne. "I am extremely proud of the Company and team we have built over the last nine years. Bio-Techne is in an ideal position to find the leader to drive the business forward starting in fiscal 2025."

On August 4, 2022 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, reported business highlights and financial results for the second quarter ended June 30, 2022 (Press release, Agios Pharmaceuticals, AUG 4, 2022, View Source [SID1234617451]).

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!

"As we look ahead to the next chapter for the company, Agios is operating from a position of strength. We are executing our commercial launch of PYRUKYND, the first therapy for a rare, debilitating, lifelong blood disorder; we also have five pivotal trials underway, multiple early-stage studies planned or ongoing, a promising preclinical pipeline and a strong balance sheet providing optionality for the future growth of the business," said Jackie Fouse, Ph.D., chief executive officer at Agios. "As I reflect on my legacy as CEO of Agios, I am proud of the bold and strategic decisions we have made to maximize and accelerate our impact for people with genetically defined diseases. I am tremendously grateful to the Agios team for their unwavering resiliency in the face of unprecedented challenges and heartfelt dedication to patients and each other. I look forward to my new role at Agios as board chair and to supporting Brian as he leverages his expertise to expand Agios’ genetically defined disease capabilities, foster the company’s differentiated culture and drive our next phase of impact."

Second Quarter 2022 & Recent Highlights

Continued to execute U.S. launch of PYRUKYND, generating $3.1 million in U.S. net revenue for the second quarter of 2022, the first full quarter following FDA approval.
Initiated Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND in pediatric patients with PK deficiency who are not regularly transfused and who are regularly transfused, respectively.
Announced that effective August 8, 2022, Dr. Fouse will transition to the role of chair of Agios’ board of directors and Brian Goff will assume the role of Agios’ chief executive officer and member of the board of directors.
Evolved Agios’ research approach to focus on advancing the company’s existing validated preclinical programs and in-licensing or acquiring well-characterized, high-potential assets.
Expanded role of Sarah Gheuens, M.D., Ph.D., to chief medical officer and head of research and development, incorporating research and discovery sciences in addition to her existing chief medical officer responsibilities.
Presented clinical and translational data at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Congress, including new data supporting the potential benefits of PYRUKYND treatment in adults with PK deficiency.
Key Upcoming Milestones & Priorities

Agios expects to execute on the following key milestones and priorities in 2022:

Adult PK Deficiency: Receive European Medicines Agency (EMA) regulatory decision for PYRUKYND in adults with PK deficiency by year-end.
Thalassemia: Enroll a meaningful portion of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively, by year-end.
Sickle Cell Disease: Complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND in sickle cell disease by year-end.
Myelodysplastic Syndrome: Initiate Phase 2a study of AG-946 in adults with low- to intermediate-risk MDS by year-end.
Data Presentations: Continue to publish clinical and translational data supporting the utility of PK activators across key disease areas and elucidating the burden of disease for PK deficiency, thalassemia and sickle cell disease.
Second Quarter 2022 Financial Results

The financial results discussion compares Agios’ continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.

Revenue: Net U.S. product revenue from sales of PYRUKYND for the second quarter of 2022 was $3.1 million. This revenue reflects the first full quarter of PYRUKYND launch, following FDA approval on February 17, 2022. In addition, Agios recognized revenue of $2.5 million dollars in the second quarter of 2022 as an up-front payment associated with the licensing of intellectual property for the company’s Friedreich’s Ataxia preclinical program.

Cost of Sales: Cost of sales for the second quarter of 2022 was $0.4 million.

Non-Operating Income: Non-operating income included approximately $2.7 million from TIBSOVO (ivosidenib) royalties for the second quarter of 2022.

Research and Development (R&D) Expenses: R&D expenses were $74.5 million for the second quarter of 2022 compared to $62.0 million for the second quarter of 2021. The year-over-year increase in R&D was driven primarily by increased headcount and workforce-related expenses, planned increased activity associated with the PAH preclinical program, start-up costs for the AG-946 Phase 2a MDS study, increased spend for the AG-946 Phase 1 trial, and start-up costs for the PYRUKYND pivotal studies in sickle cell disease and pediatric PK deficiency.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $28.3 million for the second quarter of 2022 compared to $29.2 million for the second quarter of 2021. The year-over-year decrease in SG&A expenses was primarily attributable to the completion of the reimbursable transition services Agios provided to Servier, which concluded in the first quarter of 2022, related to the sale of the oncology business.

Net Loss from Continuing Operations: Net loss from continuing operations was $91.8 million for the second quarter of 2022 compared to a net loss of $82.8 million for the second quarter of 2021.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2022, were $1.1 billion compared to $1.7 billion as of June 30, 2021. The year-over-year decrease is attributable to operating expenses and 5.7 million shares of common stock that the company repurchased for $273.4 million during the third and fourth quarters of 2021. Agios expects that its cash, cash equivalents and marketable securities will enable the company to execute its operating plan through major catalysts and to cash-flow positivity without the need to raise additional equity.

Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2022 financial results and recent business activities. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On August 4, 2022 Sysmex reported that Summary of Consolidated Financial Results [ IFRS ] for the First Three Months of the Fiscal Year Ending March 31, 2023 (Press release, Sysmex, AUG 4, 2022, View Source [SID1234617445])

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. Results for the First Three Months of the Fiscal Year Ending March 31, 2023
(1) Operating results
(2) Financial condition

2. Dividend

3. Financial Forecast for the Year Ending March 31, 2023

4. Other Information
(1) Changes in significant consolidated subsidiaries (which resulted in changes in scope of consolidation): Yes Excluded: One company (Sysmex International Reagents Co., Ltd.)
(2) Changes in accounting policies and accounting estimates 1) Changes in accounting policies required by IFRS: No 2) Other changes in accounting policies:

No 3) Changes in accounting estimates: No
(3) Number of outstanding stock (common stock)
1) Number of outstanding stock at the end of each fiscal period (including treasury stock): 209,665,482 shares as of Jun. 30, 2022; 209,657,362 shares as of Mar. 31, 2022 2) Number of treasury stock at the end of each fiscal period: 447,255 shares as of Jun. 30, 2022; 447,255 shares as of Mar. 31, 2022 3) Average number of outstanding stock for each period (cumulative): 209,214,577 shares for the three months ended Jun. 30, 2022 209,003,218 shares for the three months ended Jun. 30, 2021

1. Qualitative information on quarterly financial results
1) Operating performance analysis Future-related information contained in the text below is based on the judgement as of the end of the fiscal period under review. In the past, the Group booked configuration and customization costs in cloud computing contracts as other non-current assets. However, in the fiscal year ended March 31, 2022 we began recognizing these costs as expenses when the services are received, in accordance with a decision announced in April 2021 by the IFRS Interpretation Guidelines Committee. These changes in accounting policy have been applied retroactively, and we are comparing and analyzing the figures for the first quarter of the previous fiscal year to reflect this retroactive treatment. In the first quarter of the fiscal year ending March 31, 2023, the Japanese economy was characterized by a gradual easing of the severe situation brought about by COVID-19. Economic activity resumed, and corporate earnings, capital investment and personal consumption showed some signs of recovery. Even so, the outlook remained clouded by such factors as high resource prices and rising import costs, as yen depreciation continued. Overseas, the overall trend is toward recovery, although the situation varies by country and region.

There are concerns about an economic slowdown due to soaring energy and raw material prices stemming from increasing geopolitical risks, as well as a shift toward tighter U.S. monetary policy. On the healthcare front, aging populations in developed countries are driving demand for more efficient healthcare. Economic growth in emerging markets is causing healthcare demand to increase and prompting higher levels of healthcare quality and service enhancements. At the same time, rapid advances in the application of artificial intelligence, information and communications technology, and other leading-edge technologies to the healthcare sector are expected to continue providing opportunities for growth.

The global COVID-19 pandemic has also prompted considerations about healthcare systems and the potential for major changes in the healthcare environment itself. We anticipate further opportunities for growth, such as improving access to healthcare and a focus on self-medication. Under these circumstances, in an effort to expand its product portfolio in the hematology field Sysmex launched sample transportation system modules that can be connected to the XR-Series Automated Hematology Analyzer, its next-generation flagship model. The modules are equipped with the world’s first automated measurement function for quality control material

*1. They also enable sample sorting, sample archiving, and automated transportation of cleaning materials, will facilitate greater automation and efficiency in clinical testing operations. We will contribute toward the optimization of laboratory operations according to regional characteristics and facilities’ needs. In addition, we will continue advancing our product portfolio in the hematology field to this end. Sysmex acquired additional shares in Astrego Diagnostics AB, changing the company from an equity-method affiliate to a wholly owned subsidiary. Through this acquisition, Sysmex aims to accelerate the clinical application of a rapid antimicrobial susceptibility test

*2 that Astrego is developing for urinary tract infections

*3. Going forward, the companies aim to address medical issues through initiatives to counter antimicrobial resistance

*4. As an initiative toward the realization of personalized medicine, Sysmex submitted an application for manufacturing and marketing approval to the Pharmaceuticals and Medical Devices Agency (PMDA) for a genetic panel testing system for inherited retinal dystrophy (IRD)

*5. The system uses a next-generation sequencer

*6 to detect and analyze information on multiple genetic mutations in target genes taken from the blood of patients with IRD. This testing system is designed ultimately to assist in the planning of treatment and low vision care

*7 tailored to the causative genes as well as molecular evidence-based genetic counseling. As a result, we hope to contribute significantly to the quality of life of patients by clarifying the risk of developing IRD and prediction of symptom progression. As the global general distributor, Sysmex continued to market hinotori to medical institutions in Japan. (The hinotori Surgical Robot System is the first made-in-Japan robotic-assisted surgery system.) Based on our launch in the Japanese market, we are putting together regulatory affairs and sales systems in preparation to enter overseas markets.

*1 According to Sysmex’s research

*2 Antimicrobial susceptibility test： A test to determine the efficacy of various antimicrobial drugs against pathogenic bacteria detected in a sample.

*3 Urinary tract infections: The urinary tract runs between the kidneys and the urethral opening. Inflammations due to － 3 － the incursion of bacteria into the urinary tract are known as urinary tract infections. Such infections can lead to bladder inflammation and pyelonephritis (inflammation of the kidneys).

*4 Antimicrobial resistance: This phenomenon occurs when living organisms develop a resistance to a drug, whose efficacy is reduced or nullified as a result. Bacteria that have developed microbial resistance are known as antimicrobial-resistant bacteria.

*5 Inherited retinal dystrophy: A hereditary progressive disease presumably caused by a gene mutation. Several diseases that present similar symptoms are collectively referred to as IRD. Its main symptoms include night blindness (difficulty seeing in dim light), tunnel vision (a narrowing field of vision), and progressive loss of vision, which can lead to a complete loss of vision in severe cases. It is estimated that one out of every 4,000 to 8,000 people develops an IRD. The most common IRD subtype is Retinitis Pigmentosa (a designated intractable disease, Notification No: 90).

*6 Next-generation sequencer: An analyzer for reading large volumes of DNA base sequences.

*7 Low vision care: To those who experience any difficulty in their daily lives due to vision problems, a wide variety of support is offered, including the provision of advice or medical devices to improve quality of life, provision of various counseling and information services on careers and employment, and use of welfare programs.

On August 4, 2022 AstraZeneca and MSD reported it’s Lynparza (olaparib) has been approved in the European Union (EU) as monotherapy or in combination with endocrine therapy for the adjuvant treatment of adult patients with germline BRCA1/2 mutations (gBRCAm), who have human epidermal growth factor receptor 2 (HER2)-negative high-risk early breast cancer previously treated with neoadjuvant or adjuvant chemotherapy (Press release, AstraZeneca, AUG 4, 2022, View Source [SID1234617441]).

This approval by the European Commission was based on results from the OlympiA Phase III trial published in The New England Journal of Medicine in June 2021 and follows the recommendation for approval in the EU by the Committee for Medicinal Products for Human Use of Lynparza in this setting.1 In the trial, Lynparza demonstrated a statistically significant and clinically meaningful improvement in invasive disease-free survival (iDFS), reducing the risk of invasive breast cancer recurrences, new cancers, or death by 42% versus placebo (based on a hazard ratio [HR] of 0.58; 99.5% confidence interval [CI] 0.41-0.82; p<0.0001).

Lynparza also demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS), reducing the risk of death by 32% versus placebo (based on a HR of 0.68; 98.5% CI 0.47-0.97; p=0.009). The safety and tolerability profile of Lynparza in this trial was in line with that observed in prior clinical trials.

Breast cancer is the most diagnosed cancer worldwide with an estimated 2.3 million patients diagnosed in 2020.2 Approximately 90% of all breast cancer patients worldwide are diagnosed with early breast cancer and BRCA mutations are found in approximately 10% of HER2-negative patients in Europe.3-5

Professor Andrew Tutt, Global Chair of the OlympiA Phase III trial and Professor of Oncology at The Institute of Cancer Research, London and King’s College London, said: "Today’s approval marks a new era of care in Europe for patients with an inherited form of breast cancer. For patients with high-risk early-stage breast cancer, including those with germline BRCA mutations, recurrence rates remain unacceptably high, with more than one in four of these patients seeing their cancer return following surgery and systemic treatment. Olaparib is the first PARP inhibitor to demonstrate improved overall survival for high-risk early-stage breast cancer patients with germline BRCA mutations and I am hopeful it will become a new standard of care."

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said:
"With this approval, Lynparza is now the first and only PARP inhibitor available for patients with germline BRCA-mutated HER2-negative early breast cancer in Europe. We can now bring the benefits of Lynparza to this earlier setting to help reduce the risk of life-threatening recurrence."

Dr Eliav Barr, Senior Vice President, Head of Global Clinical Development and Chief Medical Officer, MSD Research Laboratories, said: "Today’s approval offers patients with germline BRCA-mutated HER2-negative early-stage breast cancer a new, much-needed treatment option. Lynparza as adjuvant treatment can significantly reduce the risk of disease recurrence and death, reinforcing the importance of conducting germline BRCA testing as soon as possible after diagnosis."

In March 2022, Lynparza was approved in the US for the treatment of gBRCAm, HER2-negative high-risk early breast cancer. Lynparza is also approved in the US, EU, Japan, and many other countries for the treatment of patients with gBRCAm, HER2-negative, metastatic breast cancer previously treated with chemotherapy based on results from the OlympiAD Phase III trial. In the EU, this indication also includes patients with locally advanced breast cancer.

Notes

Financial considerations
Following this approval for Lynparza in the EU, AstraZeneca will receive a regulatory milestone payment from MSD of $75m, anticipated to be booked as Collaboration Revenue during the third quarter of 2022.

Early breast cancer
Early breast cancer is defined as cancer confined to the breast with or without regional lymph node involvement, and the absence of distant metastatic disease.6,7 In the EU, breast cancer alone accounts for approximately 29% of all cancers in women with 1 in 7 women developing the disease in their lifetime. In 2020, breast cancer accounted for an estimated 350,000 new cases and over 90,000 deaths.8 Despite advancements in the treatment of early breast cancer, up to 30% of patients with high-risk clinical and/or pathologic features recur within the first few years and patients with gBRCA mutations are more likely to be diagnosed at a younger age than those without these mutations.5,9

Breast cancer is one of the most biologically diverse tumour types with various factors fuelling its development and progression.10 The discovery of biomarkers in the development of breast cancer has greatly impacted scientific understanding of the disease.11

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!

OlympiA
OlympiA is a Phase III, double-blind, parallel group, placebo-controlled, multicentre trial testing the efficacy and safety of Lynparza tablets versus placebo as adjuvant treatment in patients with gBRCAm high-risk HER2-negative early breast cancer, who have completed definitive local treatment and neoadjuvant or adjuvant chemotherapy.12

The primary endpoint of the trial was iDFS defined as time from randomisation to date of first locoregional or distant recurrence or new cancer or death from any cause.1

The OlympiA Phase III trial is led by the Breast International Group in partnership with the Frontier Science & Technology Research Foundation, NRG Oncology, the US National Cancer Institute, AstraZeneca and MSD. The trial is sponsored by NRG Oncology in the US and by AstraZeneca outside the US.

BRCA
BRCA1 and BRCA2 are human genes that produce proteins responsible for repairing damaged DNA and play an important role maintaining the genetic stability of cells.9 When either of these genes is mutated or altered such that its protein product either is not made or does not function correctly, DNA damage may not be repaired properly, and cells become unstable. As a result, cells are more likely to develop additional alterations that can lead to cancer. Cancers with BRCA mutations are more likely to be sensitive to PARP inhibitors including Lynparza.13-16

Lynparza
Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination repair (HRR), such as those with mutations in BRCA1 and/or BRCA2, or those where deficiency is induced by other agents (such as new hormonal agents – NHAs).

Inhibition of PARP proteins with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death.

Lynparza is currently approved in a number of countries across PARP-dependent tumour types with defects and dependencies in the DDR pathway including maintenance treatment of platinum-sensitive relapsed ovarian cancer and as both monotherapy and in combination with bevacizumab for the 1st-line maintenance treatment of BRCA-mutated (BRCAm) and homologous recombination repair deficient (HRD)-positive advanced ovarian cancer, respectively; for gBRCAm, HER2-negative metastatic breast cancer (in the EU and Japan this includes locally advanced breast cancer); for gBRCAm, HER2-negative high-risk early breast cancer (in the EU and US); for gBRCAm metastatic pancreatic cancer; and HRR gene-mutated metastatic castration-resistant prostate cancer (BRCAm only in the EU and Japan).

Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

The AstraZeneca and MSD strategic oncology collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and Koselugo (selumetinib), a mitogen-activated protein kinase (MEK) inhibitor, for multiple cancer types.

Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies. The companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines independently.

AstraZeneca in breast cancer
Driven by a growing understanding of breast cancer biology, AstraZeneca is starting to challenge and redefine the current clinical paradigm for how breast cancer is classified and treated, to deliver even more effective treatments to patients in need – with the bold ambition to one day eliminate breast cancer as a cause of death.

AstraZeneca has a comprehensive portfolio of approved and promising compounds in development that leverage different mechanisms of action to address the biologically diverse breast cancer tumour environment.

AstraZeneca aims to continue to transform outcomes for HR-positive breast cancer with foundational medicines Faslodex and Zoladex and the next-generation oral selective oestrogen receptor degrader (SERD) and potential new medicine camizestrant.

The PARP inhibitor, Lynparza, is an approved targeted treatment option for early and metastatic breast cancer patients with an inherited BRCA mutation. AstraZeneca with MSD continue to research Lynparza in breast cancer patients with an inherited BRCA mutation.

Building on the initial approvals of Enhertu, a HER2-directed antibody drug conjugate (ADC), in previously treated HER2-positive metastatic breast cancer, AstraZeneca and Daiichi Sankyo are exploring its potential in earlier lines of treatment and in new breast cancer settings.

To bring much needed treatment options to patients with triple-negative breast cancer, an aggressive form of breast cancer, AstraZeneca is testing immunotherapy Imfinzi in combination with other oncology medicines, including Lynparza and Enhertu, evaluating the potential of AKT kinase inhibitor, capivasertib, in combination with chemotherapy, and collaborating with Daiichi Sankyo to explore the potential of TROP2-directed ADC, datopotamab deruxtecan.

AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.

The Company’s focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.

AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

On August 4, 2022 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, reported financial results for the second quarter ended June 30, 2022 and provided program updates (Press release, Aeglea BioTherapeutics, AUG 4, 2022, View Source [SID1234617440]).

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 continue to make progress with our Homocystinuria program and are encouraged by the physician and patient interest in advancing our Phase 1/2 trial. There remains a great need for better treatment options for Homocystinuria patients and we look forward to sharing data from this trial later this year," said Anthony Quinn, M.B., Ch.B., Ph. D., president and chief executive officer of Aeglea.

"Given the current state of the financial markets and the implications for Aeglea and the biotechnology industry broadly, we are taking measures to reduce our cash burn and strengthen our financial position," said Jonathan Alspaugh, chief financial officer of Aeglea. "This includes reevaluating the allocation of resources to the pegzilarginase program as all patients have now transitioned to open label studies from which we’ve collected significant data. While we work with the FDA to identify a viable path forward, we will ensure that patients currently on pegzilarginase continue to receive treatment. We have also made the decision to postpone certain activities in our preclinical Cystinuria program due to strategic realignment and manufacturing constraints."

Dr. Quinn continued, "We continue to believe in the power of enzyme engineering to address unmet needs in rare metabolic diseases and that with our platform we have the potential to develop life-changing enzyme therapies for these patients. We remain dedicated to advancing our programs, supporting the patient communities we serve, and building a successful company."

Program and Corporate Updates

AGLE-177 in Homocystinuria

Completed dosing of patients in cohort 2 in Phase 1/2 clinical trial; cohort 3 currently being enrolled.
Opened first U.S. site to support enrollment of clinical trial.
Expect to announce Phase 1/2 clinical data in the fourth quarter of 2022.
Pegzilarginase in Arginase 1 Deficiency

Actively engaged with U.S. Food and Drug Administration (FDA) to discuss items cited in the Refusal to File letter and to identify a viable regulatory approach and path to the resubmission of the Biologics License Application (BLA) for pegzilarginase for the treatment of Arginase 1 Deficiency.
Cystinuria

Continuing certain Investigational New Drug (IND)-enabling activities but have postponed some manufacturing and other activities due to strategic prioritization and limited availability of raw materials.
Corporate

Strengthened the company’s financial position with a registered direct offering resulting in gross proceeds of $45.0 million, which extends the company’s cash runway through the second quarter of 2023.
Second Quarter 2022 Financial Results

As of June 30, 2022, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $90.4 million, including the $45.0 million in gross proceeds from the company’s registered direct offering in May 2022. The company expects its cash, cash equivalents and marketable securities will enable it to fund its operating expenses and capital expenditure requirements through the second quarter of 2023.

Aeglea recognized development fee revenues of $0.6 million in the second quarter of 2022 as a result of its license and supply agreement with Immedica for the commercial rights to pegzilarginase in certain territories outside the U.S. The revenue was related to the PEACE Phase 3 trial and BLA package. In the second quarter of 2021, Aeglea recognized $13.7 million of license and development revenue in connection with the Immedica license and supply agreement.

Research and development expenses totaled $15.4 million for the second quarter of 2022 and $13.6 million for the second quarter of 2021. The increase was primarily associated with expenses related to IND-enabling activities for AGLE-325 for the treatment of Cystinuria and pegzilarginase manufacturing to support ex-U.S. supply. The increase was partially offset by a decrease in expenses related to the PEACE Phase 3 trial of pegzilarginase and reductions in preclinical lab work.

General and administrative expenses totaled $7.7 million for the second quarter of 2022 and $6.8 million for the second quarter of 2021. This increase was primarily due to increased compensation and other personnel expenses as well as commercial capabilities and infrastructure expenses.

Net loss totaled $22.3 million and $6.8 million for the second quarter of 2022 and 2021, respectively, with non-cash stock compensation expense of $2.0 million and $2.1 million for the second quarter of 2022 and 2021, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is a novel recombinant human enzyme engineered to degrade the amino acid arginine and has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of people with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality.

The PEACE Phase 3 clinical trial met its primary endpoint with a 76.7% reduction in mean plasma arginine compared to placebo. Additionally, 90.5% of pegzilarginase treated patients achieved normal plasma arginine levels. The arginine lowering was accompanied by a positive trend in Gross Motor Function Measure Part E, a measure of patient mobility. Aeglea’s Phase 1/2 and Phase 2 Open-Label Extension (OLE) data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough Therapy, Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration as well as Orphan Drug Designation from the European Medicines Agency.

About AGLE-177 in Homocystinuria

AGLE-177 is a novel recombinant human enzyme, which is engineered to degrade the amino acid homocysteine and its dimer. AGLE-177 is currently being studied in a Phase 1/2 clinical trial for the treatment of patients with Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of total homocysteine. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis), developmental delay, intellectual disability, lens dislocation and severe near sightedness. Preclinical data demonstrated that AGLE-177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received both U.S. and EU Orphan Drug Designation as well as U.S. Rare Pediatric Disease Designation.