On November 30, 2020 NuCana plc (NASDAQ: NCNA) reported that the final results of the Phase Ib study of Acelarin plus cisplatin for patients with advanced biliary tract cancer (ABC-08) have been published online ahead of print in The Oncologist (Press release, Nucana BioPharmaceuticals, NOV 30, 2020, View Source [SID1234571982]). Encouraging interim data had previously been reported and the final data confirm the high objective response rate and favorable safety profile of Acelarin plus cisplatin in this patient population.

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In the efficacy-evaluable patient population, the Objective Response Rate was 44%. By comparison, in the ABC-02 study, which led to gemcitabine plus cisplatin becoming the current standard of care for the first-line treatment of patients with advanced biliary tract cancer, an Objective Response Rate of 26% was achieved in the efficacy-evaluable population.

Dr. Mairéad McNamara, Co-Chief Investigator of the ABC-08 study and Senior Lecturer and Honorary Consultant in Medical Oncology at the University of Manchester and The Christie NHS Foundation Trust remarked: "Acelarin combined with cisplatin demonstrated a favorable safety profile and achieved good tumor control. Responses were seen across all five biliary tract cancer sub-types, including a Complete Response in one patient, a very rare occurrence in this patient population. In ABC-02, only one out of 161 efficacy-evaluable patients who received gemcitabine plus cisplatin achieved a Complete Response. Additionally, one patient with Stable Disease, whose tumor had initially been considered unsuitable for surgical resection, was able to have complete surgical removal of the tumor following treatment with Acelarin plus cisplatin. Four patients were still alive at the end of follow-up, having survived between 16 and 36 months."

"We are very encouraged by the final efficacy and safety data from ABC-08", said Hugh S. Griffith, NuCana’s founder and CEO. "We are committed to developing Acelarin plus cisplatin as the first approved front-line treatment for patients with advanced biliary tract cancer. We continue to drive recruitment in the ongoing Phase III NuTide:121 Study where we believe we have the potential opportunity to apply for accelerated approval based on Objective Response Rate. We remain on track to enroll the required number of patients in NuTide:121 by the end of 2021 in order to conduct the first interim analysis in 2022."

Professor Juan Valle, Co-Chief Investigator of the ABC-02, ABC-08 and NuTide:121 studies and Professor and Honorary Consultant in Medical Oncology at the University of Manchester and The Christie NHS Foundation Trust said: "Biliary tract cancer is a devastating disease for which more effective treatments are desperately needed. While targeted therapies and immunotherapies may play a role in the treatment of biliary tract cancer in the future, chemotherapy will remain the backbone of treatment. Medicines like Acelarin, with the potential for improved efficacy and safety, would be welcome additions to the treatment options for patients with biliary tract cancer."

About NuTide:121

NuTide:121 is a global, multi-center, randomized Phase III study that is enrolling up to 828 patients in approximately 130 sites across North America, Europe, Asia and Australia. Patients are being randomized 1:1 and treated with either a combination of Acelarin (725 mg/m2) plus cisplatin (25 mg/m2) or the current standard of care regimen, gemcitabine (1,000 mg/m2) plus cisplatin (25 mg/m2). The primary objectives of NuTide:121 are Overall Survival (OS) and Objective Response Rate (ORR). Three interim analyses, including two designed to support accelerated approval, are planned as part of the Phase III study protocol, in addition to the final analysis. Based on discussions with the FDA and subject to any further regulatory guidance, the Company believes that a statistically significant improvement in ORR at either of the first two interim analyses, supported by positive trends in other endpoints, could potentially allow for an accelerated approval of a new drug application (NDA) for Acelarin. Accelerated approval requires a confirmatory clinical study to verify the drug’s clinical benefit. If accelerated approval were to occur, NuTide:121 would continue and the Company anticipates that data from subsequent analyses could provide the confirmatory data to support full (regular) approval.

About Biliary Tract Cancer

Biliary tract cancer, including cholangiocarcinoma, gallbladder and ampullary carcinoma, is cancer originating in the bile duct, a vessel that transports bile from the liver to the gallbladder and small intestine. Approximately 178,000 new cases of biliary tract cancer are diagnosed each year worldwide, with more than 18,000 of those diagnoses in the United States. There are currently no agents approved for the treatment of biliary tract cancer; however, the worldwide standard of care in the first-line setting for patients with advanced biliary tract cancer is the combination of gemcitabine and cisplatin.

On November 30, 2020 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for zanidatamab in patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC) (Press release, Zymeworks, NOV 30, 2020, View Source [SID1234571981]).

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The FDA grants Breakthrough Therapy designation to new medicines that are intended to treat a serious condition and where clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint. Zanidatamab will now be eligible for Accelerated Approval, Priority Review and Rolling Review, as well as intensive FDA guidance on an efficient drug development program.

"This Breakthrough Therapy designation from the FDA, based on data generated in BTC patients treated in the initial Phase 1 trial, is recognition of the potential of zanidatamab to provide a new approach to cancer treatment," said Diana Hausman, M.D., Chief Medical Officer at Zymeworks. "This milestone supports our strategy for accelerated approval and will help make zanidatamab available for patients as quickly as possible."

"BTC is a rare and aggressive cancer," said James Priour, Senior Vice President, Commercial, at Zymeworks. "Receiving this designation from the FDA is testament to the potential of zanidatamab to be the first HER2-targeting therapy approved for metastatic BTC patients."

Earlier this year, Zymeworks initiated a global Phase 2b registration-enabling study of single agent zanidatamab in patients with previously treated HER2 gene-amplified BTC. This study, which is currently enrolling patients, is designed to support accelerated approval based on a primary endpoint of objective response rate, and secondary endpoints of duration of response and safety and may enable submission of a Biologics License Application (BLA) as early as 2022.

This Breakthrough Therapy designation was based on an ongoing clinical trial of zanidatamab in patients with locally advanced (unresectable) and/or metastatic HER2-expressing tumors including BTC. Updated clinical data for single agent zanidatamab patients with BTC has been accepted for presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Virtual Gastrointestinal Cancers Symposium (ASCO GI) January 15-17, 2021.

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. This unique design results in multiple mechanisms of action including dual HER2 signal blockade, increased binding, and removal of HER2 protein from the cell surface, and potent effector function leading to encouraging antitumor activity in patients. Zymeworks is developing zanidatamab in multiple Phase 1, Phase 2, and registration-enabling clinical trials globally as a targeted treatment option for patients with solid tumors that express HER2. In addition to Breakthrough Therapy designation for zanidatamab in BTC, the US FDA has granted two Fast Track designations to zanidatamab, one as a single agent for refractory BTC and one in combination with standard of care chemotherapy, for first-line gastroesophageal adenocarcinoma (GEA). Zanidatamab has also received Orphan Drug designations for the treatment of biliary tract, gastric and ovarian cancers from the US FDA, as well as Orphan Drug designation for the treatment of gastric cancer from the European Medicines Agency.

About Biliary Tract Cancer

Biliary tract cancer (BTC), including gallbladder cancer and cholangiocarcinoma (bile duct cancer), accounts for approximately 3% of all adult cancers and is associated with a poor prognosis. Globally, more than 210,000 people are diagnosed with BTC every year. Most patients (> 65%) with BTC are diagnosed with tumors that cannot be removed surgically, and even those patients who undergo potentially curative surgery have a high recurrence rate. Treatment options are limited for patients with advanced BTC who experience disease progression after front-line chemotherapy.

The human epidermal growth factor receptor 2 (HER2) is a well-described target for anti-cancer therapy. Tumor cells that produce a higher than normal level of HER2 tend to grow more quickly and spread to other parts of the body. About 5% to 19% of patients with BTC have tumors that express HER2, suggesting that these patients may potentially benefit from HER2-targeted therapy. Currently no HER2-targeted therapy has been approved for the treatment of BTC.

On November 30, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported that it has commenced an underwritten public offering of $150 million of shares of its common stock (Press release, Intellia Therapeutics, NOV 30, 2020, View Source [SID1234571979]). Intellia also intends to grant the underwriters a 30-day option to purchase up to an additional fifteen percent (15%) of the shares of common stock offered in the public offering. All of the shares in the proposed offering are to be sold by Intellia.

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Goldman Sachs & Co. LLC, Jefferies and SVB Leerink are acting as joint book-running managers for the proposed offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

The shares of common stock are being offered by Intellia pursuant to a shelf registration statement that was previously filed with, and subsequently declared effective by, the U.S. Securities and Exchange Commission (SEC). A preliminary prospectus supplement and accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and may be obtained, when available, from: Goldman Sachs & Co. LLC, by mail at 200 West Street, New York, NY 10282, Attention: Prospectus Department, by telephone at (866) 471-2526, or by email at [email protected]; Jefferies LLC, by mail at 520 Madison Avenue, 2nd Floor, New York, NY 10022, Attention: Equity Syndicate Prospectus Department, by telephone at (877) 547-6340, or by email at [email protected]; SVB Leerink LLC, by mail at One Federal Street, 37th Floor, Boston, MA 02110, Attention: Syndicate Department, by telephone at (800) 808-7525, ext. 6132, or by email at [email protected]; or by accessing the SEC’s website at www.sec.gov.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On November 30, 2020 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported that it has commenced an underwritten public offering of $60,000,000 of shares of its common stock (Press release, Cogent Biosciences, NOV 30, 2020, View Source [SID1234571978]). In addition, Cogent Biosciences has granted the underwriters a 30-day option to purchase up to an additional $9,000,000 of shares of its common stock. All of the shares of common stock to be sold in the underwritten public offering are being offered by Cogent Biosciences.

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Cogent Biosciences intends to use the net proceeds from the offering for development, regulatory and commercial preparation activities relating to PLX9486 and other product candidates, as well as for working capital and general corporate purposes.

Jefferies and Piper Sandler & Co. are acting as joint book-running managers for the offering. Wedbush PacGrow, LifeSci Capital and Ladenburg Thalmann are also acting as co-managers for the offering.

A registration statement relating to these securities has been filed with the Securities and Exchange Commission (SEC) and became effective on May 1, 2019.

A preliminary prospectus supplement and accompanying base prospectus relating to and describing the terms of the offering have been filed with the SEC. The securities described above have not been qualified under any state blue sky laws. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. The offering can be made only by means of a prospectus, copies of which may be obtained at the SEC’s website at www.sec.gov, or by request to Jefferies LLC (Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, New York 10022; telephone: 877-821-7388; email: [email protected]); or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, or by telephone at (800) 747-3924, or by email at [email protected].

On November 30, 2020 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that it has commenced clinical trial site recruitment for its upcoming Acclaim-1 clinical trial for the treatment of non-small cell lung cancer (NSCLC) (Press release, Genprex, NOV 30, 2020, View Source [SID1234571977]). As planned, the timing of the patient recruitment and enrollment puts the Company on track for its Acclaim-1 clinical trial to commence in the first-half of 2021.

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Acclaim-1 is an open-label, multi-center Phase 1/2 clinical trial that combines Genprex’s lead drug candidate, REQORSA immunogene therapy with AstraZeneca PLC’s Tagrisso in patients with late stage NSCLC with mutated epidermal growth factor receptors (EGFRs), whose disease progressed after treatment with Tagrisso. Genprex received U.S. Food and Drug Administration (FDA) Fast Track Designation for its Acclaim-1 patient population in January of 2020.

"Our clinical team is engaging with prestigious cancer centers and research institutions across the U.S. to ensure we select optimal study sites, which play an important role in the success of a clinical trial," said Rodney Varner, President and Chief Executive Officer of Genprex. "We look forward to working with leading clinical investigators who will help drive our mission to bring forth a treatment for advanced lung cancer patients."

The Company plans to conduct the Acclaim-1 clinical trial in approximately 10 U.S. sites with about 100 patients (9-18 patients in the Phase 1 component and 82 patients in the Phase 2 component). An interim analysis will be performed after 53 events (i.e., progression of disease or death).

Additional information on the Acclaim-1 clinical trial can be found by visiting ClinicalTrials.gov.