Zanidatamab Phase 2 Clinical Trial Demonstrates Promising Response Rate and
Durability in First-Line HER2-Positive Gastroesophageal Adenocarcinoma (GEA)
at the European Society for Medical Oncology (ESMO) Annual Congress

On September 16, 2021 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported that new clinical data for zanidatamab, a HER2-targeted bispecific antibody, demonstrate promising response rates and durability in first-line HER2-positive GEA (Press release, Zymeworks, SEP 16, 2021, View Source [SID1234587812]). These data were presented today by lead study investigator, Geoffrey Ku, M.D., Medical Oncologist at Memorial Sloan Kettering Cancer Center (MSK), at the ESMO (Free ESMO Whitepaper) Annual Congress.

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Phase 2 Study Results

The data presented at ESMO (Free ESMO Whitepaper) are from a clinical study of 36 patients with HER2-expressing GEA who received zanidatamab in combination with either CAPOX (capecitabine/oxaliplatin; n=14), FP (5FU/cisplatin; n=2), or mFOLFOX6 (5FU/leucovorin/oxaliplatin; n=20). None of the patients had received prior HER2-targeted therapies.

In 28 response-evaluable patients with metastatic HER2-positive GEA, zanidatamab plus chemotherapy resulted in a cORR of 75% and disease control rate (DCR) of 89% overall, with a cORR of 93% and DCR of 100% in the proposed Phase 3 regimen of zanidatamab + CAPOX/FP. All patients except one experienced a decrease in their tumor size. The mDOR is 16.4 months and the mPFS is 12.0 months across all treatment regimens with 61% of patients still on study at the time of data cutoff.

In addition, the data demonstrate that zanidatamab plus chemotherapy is generally well tolerated, with the majority of treatment-related adverse events (TRAEs) considered mild to moderate in severity (Grade 1 or 2). The most common grade ³ 3 TRAE was diarrhea which was manageable in the outpatient setting; introduction of prophylactic loperamide reduced the incidence in cycle 1 from 44% to 18%. No severe (grade ³ 3) infusion-related reactions or cardiac events were observed.

"Despite recent advances, metastatic HER2-expressing GEA has high morbidity and mortality, and new treatment options are desperately needed," said the principal investigator on the trial, Geoffrey Ku, M.D., Medical Oncologist at MSK. "The data presented today at ESMO (Free ESMO Whitepaper) demonstrate the potential of zanidatamab in the first-line setting, highlighted by response rates and durability that compare favorably to the current standard of care as well as to emerging treatments. These data support further evaluation of zanidatamab plus chemotherapy in a randomized pivotal trial in first-line HER2-positive GEA."

HER2 is overexpressed in approximately 20% of GEA patients. For these patients, Herceptin (trastuzumab) is the only approved HER2-targeted therapy in 1L treatment, and therapeutic options are currently limited if disease progression occurs. Zanidatamab’s bispecific approach simultaneously binds two non-overlapping epitopes of HER2, ECD2 and ECD4, resulting in multiple mechanisms of action that provide potential therapeutic benefits beyond the combination of two monoclonal antibodies.

"Herceptin and chemotherapy have been the standard of care in first-line HER2-positive GEA for over 10 years. The data presented today give us the confidence that we have developed a next-generation HER2-targeted agent that has the potential to provide patients with an improved option in this setting," said Neil Josephson, M.D., Zymeworks’ Interim Chief Medical Officer. "Based on these data, we will pursue a randomized, Phase 3 trial with the aim of establishing zanidatamab as the foundational agent of a new standard of care in first-line HER2-positive GEA."

In addition to the Phase 2 study presented today, Zymeworks and its partner, BeiGene, Ltd., are conducting a parallel open-label Phase 2 clinical trial evaluating zanidatamab and chemotherapy in combination with the PD-1 inhibitor, tislelizumab, in first-line HER2-positive metastatic GEA. These results are expected to be presented at a future medical conference.

Zymeworks plans to launch, with BeiGene, a randomized, global Phase 3 study (HERIZON-GEA-01) in Q4 2021. The study will evaluate zanidatamab plus chemotherapy (CAPOX or FP) with or without tislelizumab, versus standard of care (trastuzumab plus chemotherapy), for first-line treatment of locally advanced, unresectable, or metastatic HER2-positive GEA.

"Our long-standing vision for zanidatamab has been for it to become a best-in-class HER2-targeted therapeutic that could address the needs of a broad spectrum of patients with HER2-expressing cancers," said Ali Tehrani, Ph.D., Zymeworks’ President & CEO. "Over the years we have shared data that have showcased the promising anti-tumor activity and safety profile of zanidatamab; that said, the data shared today stand out as they represent the first clinical validation of zanidatamab in a front-line setting. As we embark on our second pivotal trial and prepare for commercialization, these data represent a landmark moment for zanidatamab and for Zymeworks."

Dr. Ku has provided advisory services for Zymeworks.

ESMO Presentation

The presentation will be available to conference registrants on the ESMO (Free ESMO Whitepaper) conference website as well as to the general public on the Zymeworks website at View Source

Title: Phase (Ph) 2 Study of Zanidatamab + Chemotherapy (chemo) in First Line (1L)

HER2-expressing Gastroesophageal Adenocarcinoma (GEA)

Lead Author: Geoffrey Ku, M.D., Memorial Sloan Kettering Cancer Center, New York, NY, US

Abstract: 3678

E-poster: 1380P

Conference Call and Webcast

The company will host a conference call and webcast to discuss the updated data. The event will be led by Ali Tehrani, Ph.D., Zymeworks’ President and CEO and Neil Josephson, M.D., Zymeworks’ Interim Chief Medical Officer, and will include a presentation by medical oncologist and principal investigator, Geoffrey Ku, M.D., Memorial Sloan Kettering Cancer Center. Dr. Ku and members of Zymeworks’ executive team will be available to answer questions at the conclusion of the call.

Date: Thursday, September 16th

Time: 7:30 am ET

Interested parties can access the live webcast via the Zymeworks’ website at View Source A recorded replay will be accessible after the event through the Zymeworks website.

About Zanidatamab

Zanidatamab is a bispecific antibody, based on Zymeworks’ Azymetric platform, that can simultaneously bind two non-overlapping epitopes of HER2, known as biparatopic binding. Zanidatamab’s unique binding properties result in multiple mechanisms of action including HER2-receptor clustering, internalization, and downregulation; inhibition of growth factor-dependent and -independent tumor cell proliferation; antibody-dependent cellular cytotoxicity and phagocytosis; and complement-dependent cytotoxicity. Zymeworks is developing zanidatamab in multiple Phase 1, Phase 2, and pivotal clinical trials globally as a targeted treatment option for patients with solid tumors that express HER2. The FDA has granted Breakthrough Therapy designation for zanidatamab in patients with previously treated HER2 gene-amplified biliary tract cancer (BTC), and two Fast Track designations to zanidatamab, one as monotherapy for refractory BTC and one in combination with standard of care chemotherapy for first-line gastroesophageal adenocarcinoma (GEA). These designations mean zanidatamab is eligible for Accelerated Approval, Priority Review and Rolling Review, as well as intensive FDA guidance on an efficient drug development program. Zanidatamab has also received Orphan Drug designations for the treatment of biliary tract, gastric and ovarian cancers, as well as Orphan Drug designations from the European Medicines Agency for the treatments of biliary tract and gastric cancer.

Biomea Fusion Announces FDA Clearance of Investigational New Drug Application for Irreversible Menin Inhibitor BMF-219

On September 16, 2021 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a biopharmaceutical company focused on the discovery and development of irreversible small molecules to treat patients with genetically defined cancers, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug application to begin a Phase I trial of BMF-219, a selective irreversible menin inhibitor, in adult patients with relapsed or refractory acute leukemia including those with an MLL/KM2TA gene rearrangement or NPM1 mutation (Press release, Biomea Fusion, SEP 16, 2021, View Source [SID1234587811]).

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"First of all, I would like to take this opportunity to thank the FDA, the Contract Research Organizations, our consultants, our investors, and of course TEAM FUSION for the commitment, guidance, support, and tireless effort in getting BMF-219, an investigational new drug, in the hands of patients in need. It was a true community effort, and we are so blessed here at Biomea to be in position to provide an impactful therapy against aggressive cancers," said Thomas Butler, Biomea’s CEO and Chairman of the Board. "This is just the beginning for BMF-219 as we are planning to pursue multiple indications with our novel molecule. This is also just the beginning for the company, as we continue to make significant progress with our pipeline programs. We are in a strong position to continue to bring novel small molecules into the clinic and help the many patients with life threatening and life altering diseases."

"Over the past 6 months, we have brought together a first-class team of biotech professionals to tackle our next phase of growth, which will include clinical development of BMF-219 in not only liquid but also solid tumors," said Ramses Erdtmann, Biomea’s COO and President. "BMF-219 is a very special compound, with a unique effect on menin which we believe will lead to improved outcomes for patients with specific gene arrangement and mutations."

An irreversible small molecule, such as BMF-219, is a synthetic compound that forms a permanent bond to its target protein and offers a number of potential advantages over conventional reversible drugs, including greater target selectivity, lower drug exposure, and the ability to drive a deeper, more durable response.

The Phase 1, first-in-human, open-label, dose-escalation and dose-expansion clinical trial of BMF-219 will assess the safety, pharmacokinetic (PK) and pharmacodynamic (PD) profile of BMF-219 in adult patients with relapsed or refractory acute leukemia including those with an MLL/KM2TA gene rearrangement or NPM1 mutation.

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/KM2TA gene rearrangement or NPM1 mutation.

Vincerx Pharma Announces $50 Million Private Placement

On September 16, 2021 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported that it has entered into a definitive agreement on September 15, 2021 to sell an aggregate of 3.5 million shares of common stock at a purchase price of $14.50 per share (Press release, Vincerx Pharma, SEP 16, 2021, View Source [SID1234587807]). The private placement was led by new and existing investors, including Deerfield Management Company, Rock Springs Capital, Point72 Asset Management, and Sphera Healthcare, among others.

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The Company intends to use the net proceeds from the financing for working capital and general corporate purposes, including to support the clinical evaluation of VIP152, the Company’s potent and selective inhibitor of CDK9, in additional indications and combination regimens, as well as to advance its bioconjugation platform. Vincerx had $85.6 million in cash and cash equivalents as of June 30, 2021.

"The Vincerx team remains focused on our mission of developing innovative and urgently needed oncology therapies. We look forward to the planned initiation of our Phase 1 dose escalation study in CLL relapsed or refractory to venetoclax and BTK inhibitors in the second half of this year. In addition to our monotherapy approach, we are also excited to initiate our combination studies in the early part of next year, which would expand our addressable patient population and allow us to move to earlier lines of therapy," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx.

The financing is expected to close on September 20, 2021, subject to satisfaction of customary closing conditions. SVB Leerink acted as the lead placement agent. LifeSci Capital and Cantor Fitzgerald acted as co-placement agents.

The securities to be sold in the private placement have not been registered under the Securities Act of 1933 or applicable state securities laws and may not be offered or sold in the United States absent registration under the Securities Act or an applicable exemption from such registration requirements. The Company has agreed to file a resale registration statement with the U.S. Securities and Exchange Commission covering the resale of the shares of common stock sold in the private placement. This press release shall not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus.

Targovax ASA: Poster at European Society for Medical Oncology (ESMO)

On September 16, 2021 Targovax ASA (OSE: TRVX), a clinical stage immuno-oncology company developing immune activators to target hard-to-treat solid tumors, reported that the poster "A pilot study of Engineered Adenovirus ONCOS-102 in combination with pembrolizumab (pembro) in checkpoint inhibitor refractory advanced or unresectable melanoma" is now available at the ESMO (Free ESMO Whitepaper) congress website and on the Company’s website (Press release, Targovax, SEP 16, 2021, View Source [SID1234587806]). The poster will be presented as an e-poster at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) congress by Dr Alexander N. Shoushtari, Memorial Sloan Kettering Cancer Center.

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The poster presents the pilot study of ONCOS-102 in combination with pembrolizumab (pembro) in checkpoint inhibitor refractory advanced or unresectable melanoma. The poster concludes that co-administration of ONCOS-102 and pembrolizumab is safe and feasible for patients with melanomas progressing on PD-1 blockade. Rapid clinical objective responses were seen in patients treated both sequentially and in combination, and immune markers demonstrating induction of beneficial tumor microenvironment changes support the role of ONCOS-102 as a complementary treatment with aPD1 and other IO modalities.

E-poster title: A pilot study of Engineered Adenovirus ONCOS-102 in combination with pembrolizumab (pembro) in checkpoint inhibitor refractory advanced or unresectable melanoma
E-poster number: 1083P
Presenter: Dr Alexander N. Shoushtari, Memorial Sloan Kettering Cancer Center

Corporate Slide Presentation, dated September 16, 2021

On September 16, 2021, Silverback Therapeutics, Inc. (the "Company") presented its corporate slide presentation (Presentation, Silverback Therapeutics, SEP 16, 2021, View Source [SID1234587805]).

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