On November 3, 2021 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that the European Medicines Agency (EMA) has validated the Type II Variation Application for trastuzumab deruxtecan, a HER2 directed antibody drug conjugate (ADC) being jointly developed by Daiichi Sankyo and AstraZeneca (LSE/STO/Nasdaq: AZN), for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior anti-HER2-based regimen (Press release, Daiichi Sankyo, NOV 3, 2021, View Source [SID1234594266]).

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!

Validation confirms that the application is complete and commences the scientific review process by the EMA’s Committee for Medicinal Products for Human Use (CHMP). This application is based on the positive results from the DESTINY-Gastric01 pivotal phase 2 trial published in The New England Journal of Medicine and the DESTINY-Gastric02 phase 2 trial recently presented at the 2021 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress.

"After progression on initial therapy, patients with HER2 positive advanced gastric cancer are faced with limited options in Europe, so there is a significant unmet need for new therapeutic options," said Gilles Gallant, BPharm, PhD, FOPQ, Senior Vice President, Global Head, Oncology Development, Oncology R&D, Daiichi Sankyo. "We look forward to working with the EMA on its review of this application and to potentially bring trastuzumab deruxtecan to physicians and patients in Europe, as it offers the potential to provide an important new treatment option to patients with locally advanced or metastatic HER2 positive gastric or GEJ adenocarcinoma who have received a prior anti-HER2-based regimen."

About HER2 Positive Gastric Cancer

Gastric (stomach) cancer is the fifth most common cancer worldwide and the fourth highest leading cause of cancer mortality, with a five-year global survival rate of 5% to 10% for advanced or metastatic disease.1,2,3 There were approximately one million new cases of gastric cancer and 768,000 deaths reported worldwide in 2020.4 In Europe, approximately 136,000 cases of gastric cancer are diagnosed annually, and Eastern Europe has the second highest incidence of gastric cancer worldwide after Eastern Asia.3,4 Gastric cancer is the sixth leading cause of cancer death in Europe, and is typically diagnosed in the advanced stage but even when diagnosed in earlier stages of the disease the survival rate remains modest.3,5,6,7

Approximately one in five gastric cancers are HER2 positive.8,9 HER2 is a tyrosine kinase receptor growth promoting protein expressed on the surface of many types of tumors including breast, gastric, lung and colorectal cancers.9 HER2 overexpression may be associated with a specific HER2 gene alteration known as HER2 amplification.9

Recommended first-line treatment for HER2 positive advanced or metastatic gastric cancer is combination chemotherapy plus trastuzumab, an anti-HER2 medicine, which has been shown to improve survival outcomes when added to chemotherapy.10 For patients with metastatic gastric cancer that progress following initial treatment with a trastuzumab-based regimen, treatment options are limited, and in many regions in the world there are no additional HER2 directed medicines available.1,5,11

About DESTINY-Gastric01

DESTINY-Gastric01 is a pivotal, randomized, open-label, multi-center phase 2 trial assessing the safety and efficacy of trastuzumab deruxtecan (6.4 mg/kg) in patients from Japan and South Korea with HER2 positive (defined as IHC3+ or IHC2+/ISH+) advanced gastric cancer or gastroesophageal junction adenocarcinoma who have progressed on two or more prior treatment regimens including fluoropyrimidine (5-FU), platinum chemotherapy and trastuzumab. Patients were randomized 2:1 to receive trastuzumab deruxtecan or physician’s choice of chemotherapy (paclitaxel or irinotecan monotherapy). The primary endpoint of DESTINY-Gastric01 is objective response rate (ORR). Secondary endpoints include overall survival (OS), progression-free survival (PFS), duration of response (DoR), disease control rate and time to treatment failure as well as pharmacokinetic and safety endpoints. DESTINY-Gastric01 enrolled 189 patients at multiple sites in Japan and South Korea. For more information about the trial, visit ClinicalTrials.gov.

About DESTINY-Gastric02

DESTINY-Gastric02 is an open-label, single-arm, phase 2 trial in Western patients evaluating the safety and efficacy of trastuzumab deruxtecan (6.4 mg/kg) in patients with HER2 positive metastatic and/or unresectable gastric or GEJ adenocarcinoma with disease progression on or after a trastuzumab-containing regimen. The primary endpoint of DESTINY-Gastric02 is confirmed ORR based on independent central review. Secondary endpoints include PFS, OS, DoR and safety. DESTINY-Gastric02 enrolled 79 patients at multiple sites in North America and Europe. For more information about the trial, visit ClinicalTrials.gov.

About Trastuzumab Deruxtecan

Trastuzumab deruxtecan (fam-trastuzumab deruxtecan-nxki in the U.S. only) is a HER2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, trastuzumab deruxtecan is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC scientific platform. Trastuzumab deruxtecan consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker.

Trastuzumab deruxtecan (5.4 mg/kg) is approved in more than 30 countries for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received two or more prior anti-HER2 based regimens based on the results from the DESTINY-Breast01 trial.

Trastuzumab deruxtecan (6.4 mg/kg) is approved in Israel, Japan, Singapore, and U.S. for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial.

About the Trastuzumab Deruxtecan Clinical Development Program

A comprehensive global development program is underway evaluating the efficacy and safety of trastuzumab deruxtecan monotherapy across multiple HER2 targetable cancers including breast, gastric, lung and colorectal cancers. Trials in combination with other anticancer treatments, such as immunotherapy, are also underway.

Trastuzumab deruxtecan was highlighted in the Clinical Cancer Advances 2021 report as one of two significant advancements in the "ASCO Clinical Advance of the Year: Molecular Profiling Driving Progress in GI Cancers," based on data from both the DESTINY-Gastric01 and DESTINY-CRC01 trials, as well as one of the targeted therapy advances of the year in non-small cell lung cancer (NSCLC) based on the interim results of the HER2 mutated cohort of the DESTINY-Lung01 trial.

Trastuzumab deruxtecan recently received its fourth Breakthrough Therapy Designation (BTD) in the U.S., which was for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received one or more prior anti-HER2-based regimens.

About the Daiichi Sankyo and AstraZeneca Collaboration

Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize trastuzumab deruxtecan in March 2019 and datopotamab deruxtecan (Dato-DXd) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of trastuzumab deruxtecan and datopotamab deruxtecan.

About Daiichi Sankyo in Oncology

The oncology portfolio of Daiichi Sankyo is powered by our team of world-class scientists that push beyond traditional thinking to create transformative medicines for people with cancer. Anchored by our DXd antibody drug conjugate (ADC) technology, our research engines include biologics, medicinal chemistry, modality and other research laboratories in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in the U.S. We also work alongside leading academic and business collaborators to further advance the understanding of cancer as Daiichi Sankyo builds towards our ambitious goal of becoming a global leader in oncology by 2025.

On November 3, 2021 SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company pioneering innovative medicines to overcome and prevent difficult-to-treat and drug-resistant infections, reported it will host a conference call and live webcast at 8:30 a.m. ET on Wednesday, November 10, 2021, to provide a corporate update and discuss the Company’s financial results for the third quarter ended September 30, 2021 (Press release, Scynexis, NOV 3, 2021, View Source [SID1234594265]).

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!

Conference call and webcast details:

A live audio webcast can be accessed by visiting the Investor Relations section of the Company’s website, www.scynexis.com. A replay of the webcast will be archived on the SCYNEXIS website for 90 days following the event.

On November 3, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported that it will release third quarter 2021 financial results before the market opens on Wednesday, November 10, 2021 (Press release, Hookipa Biotech, NOV 3, 2021, View Source [SID1234594264]).

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!

The Company will not be conducting a conference call in conjunction with this earnings release.

On November 3, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported the publication of new long-term clinical utility data showing that the company’s Afirma Genomic Sequencing Classifier (GSC) helped reduce unnecessary surgeries in patients with indeterminate thyroid nodule cytology, as compared to the use of no molecular testing. The peer-reviewed paper appears online in The Journal of the Endocrine Society (Press release, Veracyte, NOV 3, 2021, View Source [SID1234594263]).

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!

Veracyte estimates that each year approximately 565,000 people with thyroid nodules undergo fine-needle aspiration (FNA) biopsies to assess potentially cancerous nodules. Up to 30 percent of these patients receive indeterminate results – meaning their nodules are not clearly benign or malignant based on traditional cytopathology evaluation. Historically, most of these patients were directed to diagnostic surgery, even though 70 percent to 80 percent of the time the nodules proved to be benign.1

The Afirma GSC helps physicians identify patients with benign thyroid nodules from among those with indeterminate FNA results, so that they may avoid unnecessary thyroid surgery. Current American Thyroid Association guidelines include molecular testing as a recommended option to achieve definitive diagnosis for nodules classified as indeterminate following FNA biopsy.1

In the current study, researchers at the University of Nebraska Medical Center (UNMC) and the VA Nebraska-Western Iowa Health System retrospectively analyzed 468 cytologically indeterminate thyroid nodules from January 2013 to December 2019 to assess and compare how use of the Afirma GSC (n=124) and the original Afirma Gene Expression Classifier (GEC; n=71) impacted patient care. Use of these tests was also compared to nodules that did not undergo molecular testing (n=273). They found that the Afirma GSC identified 30 percent more nodules as benign, compared to the GEC (60 percent vs. 46 percent, respectively) and that use of the Afirma GSC resulted in 41 percent fewer surgeries, compared to patients with no molecular testing (40 percent vs. 68 percent, respectively). Additionally, when surgery was performed, patients deemed "suspicious for cancer" by the Afirma GSC were 95 percent more likely to have cancer compared to those who had no molecular testing (39 percent vs. 20 percent, respectively).

"Our analysis showed a significant improvement in the benign call rate with the Afirma GSC as compared to no molecular testing, as well as a significant increase in confirmed malignancies among those patients who did go to surgery when utilizing the test," said Whitney Goldner, M.D., professor, Department of Internal Medicine, Division of Diabetes, Endocrinology, and Metabolism, University of Nebraska Medical Center. "Additionally, the test demonstrated high sensitivity and NPV, and we saw enhanced specificity and PPV with the GSC test as compared to the Afirma GEC, which is all consistent with prior studies. Overall, these results demonstrate the value and accuracy of Afirma GSC testing in the diagnostic management of cytologically indeterminate thyroid nodules."

"The Afirma GSC continues to usher in a paradigm shift in the management of patients with indeterminate thyroid nodules," said Joshua Klopper, M.D., Veracyte’s medical director, Endocrinology. "More patients are avoiding unnecessary surgery and more patients have a confirmed indication for surgery when it is performed. These new findings underscore the value that Afirma testing delivers to patients, physicians and the overall healthcare system."

On November 3, 2021 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel small molecules to treat and improve the lives of patients with genetically defined cancers, reported financial results for the third quarter of 2021 (Press release, Biomea Fusion, NOV 3, 2021, View Source [SID1234594262]).

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!

"The Third Quarter was a historic one for the company as it advanced its first investigational drug into a clinical stage program. BMF-219, our internally discovered and developed small molecule, has shown broad anti-cancer activity pre-clinically and we now have the opportunity to explore this potential in patients in need." said Thomas Butler, Biomea’s CEO and Chairman of the Board. "In September, we received FDA clearance of our IND for BMF-219, and we are now working diligently to initiate a Phase I study for the treatment of relapsed or refractory acute leukemia. We look forward to providing clinical development updates as we deepen our understanding of menin’s role in various cancers and the potential impact of irreversible inhibition for these patient populations. Our vision for this company has always been to develop a suite of novel covalent inhibitors that can act as single agent therapies or in combination, together, to build best-in-class medicine for patients. We have executed on this promise by hiring world-class drug hunters, and developers, and by custom designing and opening a state-of-the-art Research Center. We now have our first molecule ready for the clinic, and a number of exciting programs advancing through pre-clinical development."

Mr. Butler continued, "As we move BMF-219 into the clinic, we intend to also focus on leveraging our novel chemistry across multiple indications. To that end, we anticipate announcing our next pipeline candidate in the first half of 2022."

Business Highlights

FDA clearance of IND for First-in-Human Phase I Clinical Trial of BMF-219. In September 2021, Biomea received FDA clearance of its IND for BMF-219, an irreversible menin inhibitor for the treatment of relapsed or refractory acute leukemias including those with an MLL/KMT2A gene rearrangement or NPM1 mutation.
Preclinical studies underway for DLBCL and multiple myeloma (MM) as potential indications for BMF-219. Biomea is conducting preclinical studies to demonstrate the potential for BMF-219 in genetically defined patient subsets of DLBCL and MM.
Pathway validation studies of irreversible menin inhibition in diabetes. Biomea is continuing preclinical studies to explore the potential of its irreversible menin inhibitor approach for the treatment of type 2 diabetes. The company plans to report findings from these studies in the first quarter of 2022.
Launch of Biomea Innovation Center; scale-up of R&D organization. Biomea recently opened the Biomea Innovation Center with laboratory space and a dedicated R&D team focused on leveraging the company’s FUSION platform to generate a broad portfolio of next generation covalent inhibitors.
"Our CEO’s vision for the company is to build a fully sustainable, world-class R&D organization, which mines the deep potential of our FUSION platform and irreversible binding small molecule approach. With our recently opened Biomea Innovation Research Center and our continued scale-up of an extremely talented, forward-and creative-thinking scientific team, we will aggressively interrogate our irreversible binding approach against known and validated biological targets with the goal of rapidly advancing a broad portfolio of therapies designed to deliver enhanced efficacy and safety for patients. Thanks to our successful IPO and considering our current programs’ quarterly expenses, we expect our cash balance to sustain our operations well into 2024," concluded Ramses Erdtmann, Biomea’s COO and President.

Financial Highlights

Third Quarter 2021 Year to Date Financial Results

Biomea reported a net loss attributable to common stockholders of $26.9 million for the first nine months of 2021, compared to a net loss of $1.8 million for the same period in 2020.
Research and development expenses were $16.9 million for the first nine months of 2021, compared to $1.3 million for the same period in 2020. The increase of $15.6 million was primarily due to an increase in personnel-related expenses, as well as an increase in pre-clinical development costs, including manufacturing and external consulting, related to the IND-enabling studies for BMF-219.
General and administrative expenses were $10.0 million for the first nine months of 2021, compared to $0.5 million for the same period in 2020. The increase of $9.5 million was primarily due to higher personnel-related expenses and other corporate costs to support the Company’s public company status.
As of September 30, 2021, the Company had cash, cash equivalents, restricted cash, and investments of $191.9 million.
About Acute Myeloid Leukemia (AML)

AML is the most common form of acute leukemia in adults and represents the largest number of annual leukemia deaths in the U.S. and Europe. AML originates within the white blood cells in the bone marrow and can rapidly move to the blood and other parts of the body, including the lymph nodes, spleen, and central nervous system. Approximately 30,000 people in the U.S. and Europe are diagnosed with AML each year, and the five-year overall survival rate in adults roughly 29%. Among patients with relapsed/refractory disease, the need is greatest, as the overall survival is approximately 3 to 9 months. It is estimated that upwards of 45% of AML patients have menin dependent genetic drivers (MML-r or NPM1).

About BMF-219

BMF-219 is an irreversibly binding inhibitor of menin, a protein that is known to play an essential role in oncogenic signaling in genetically defined leukemias. Preclinically, BMF-219 has demonstrated robust downregulation of key leukemogenic genes in addition to menin itself (via MEN1) in well-established MLLr AML cell lines. Additionally, BMF-219 has shown efficacy in multiple in vivo and in vitro models of acute leukemias. BMF-219 will be evaluated in a first-in-human trial in patients with relapsed or refractory acute leukemia with MLL/KMT2A gene rearrangement or NPM1 mutation.