On December 18, 2020 Bavarian Nordic A/S (OMX: BAVA, OTC: BVNRY) reported top-line results from a Phase 2 clinical study evaluating the targeted immunotherapy candidate, BN-Brachyury in the treatment of advanced chordoma, a rare cancer occurring in the base of the skull and spine (Press release, Bavarian Nordic, DEC 18, 2020, View Source,of%20the%20skull%20and%20spine. [SID1234573079]). While the study failed to meet its primary endpoint, it provided evidence of clinical activity.

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The Phase 2 study enrolled 29 patients, which were treated with a combination of the prime-boost vaccine candidate, BN-Brachyury and the current standard of care, radiation therapy. Whereas radiation has been shown to inflame the tumor, thereby releasing cancer antigens, BN-Brachyury has been designed to teach T cells to attack and kill brachyury-expressing tumor cells. Patients were monitored over a period of 12 months after radiation therapy, a timeframe during which historical controls show an objective response rate (ORR) of less than 5% with radiation alone. The overall goal of the study was to achieve four patients with objective responses, corresponding to an ORR of approximately 14% for all patients enrolled.

Two patients achieving objective responses were observed in the study, as measured by the predefined conventional assessment criteria (RECIST) according to the study protocol. In addition, 19 patients have stable disease, while the rest progressed, or left the study before the assessment was performed. The RECIST criteria assumes tumors are spherical and assesses their size by measuring the tumor diameter. This method may, however, underestimate the chordoma tumor shrinkage, as a retrospective analysis of six of the patients enrolled into the study revealed four of these patients had an objective response when tumor shrinkage was evaluated by a volumetric assessment. These intriguing signs of clinical efficacy are also supported by two chordoma patients that have recorded either a volumetric objective response, or a symptomatic response following the intravenous administration of BN-Brachyury in a separate on-going trial.

While no further studies of BN-Brachyury administered subcutaneously are planned, the signs of clinical efficacy that have been observed support brachyury as a potentially important immunotherapy target, particularly for chordoma. The Company is now looking to adapt ongoing trials to investigate MVA-based vaccines encoding brachyury administered intravenously – a promising approach designed to utilize broader aspects of the immune response while improving T cell activation and function.

"Chordoma is known for its refractoriness to all treatments investigated so far. The observation of some hints of efficacy with our immunotherapy approach is encouraging enough for us to continue with our efforts to develop a new treatment for this highly unmet medical need," said Paul Chaplin, President and Chief Executive Officer of Bavarian Nordic.

On December 18, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer reported that it has entered into a license agreement with SciClone Pharmaceuticals International Ltd ("SciClone") to be the exclusive co-development and commercialization partner of the Company’s antibodies, DANYELZA (naxitamab-gqgk) for the treatment of patients with relapsed/refractory high-risk neuroblastoma and omburtamab, if approved, for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma in China (Press release, Y-mAbs Therapeutics, DEC 18, 2020, View Source [SID1234573080]). DANYELZA (naxitamab-gqgk) 40mg/10mL was approved by the U.S. Food and Drug Administration ("FDA") on November 25, 2020 and is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. The Company plans to resubmit its Biologics License Application ("BLA") to the FDA for omburtamab by the end of 2020 or in early 2021.

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The license agreement includes Greater China, including Mainland China, Taiwan, Hong Kong and Macau. Under the terms of the agreement, SciClone will employ its development, sales, marketing and regulatory expertise to commercialize DANYELZA and omburtamab, if approved, in the territory. All other unpartnered geographies worldwide remain with the Company. Under the terms of the agreement, SciClone will pay Y-mAbs a $20 million upfront payment with the potential for Y-mAbs to receive up to $100 million in additional development, regulatory and sales milestone payments for both programs, as well as double-digit royalties on net sales for DANYELZA and omburtamab in the territory.

"We are very pleased to enter this license agreement with SciClone, and hope to see a DANYELZA and omburtamab, if approved, being made available to appropriate children with unmet medical needs in China. This partnership marks an important milestone in our aim to make our lead product and product candidate globally available," said Thomas Gad, founder, Chairman and President at Y-mAbs.

"DANYELZA and omburtamab were initially identified and sourced from Memorial Sloan Kettering Cancer Center in New York, and have been developed by Y-mAbs as novel therapies. We believe these antibodies show great promise," said Hong Zhao, President and Chief Executive Officer of SciClone. "We are excited to partner with Y-mAbs team to commercialize DANYELZA for the treatment of relapsed/refractory high-risk neuroblastoma and omburtamab for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma, if approved. This strategic partnership recognizes SciClone’s capability as a leading biotech company with integrated platform of development and commercialization."

Researchers at MSK developed DANYELZA and omburtamab, which are exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the compounds and in Y-mAbs.

About DANYELZA (naxitamab-gqgk)

DANYELZA (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information for complete Boxed Warning and other important safety information.

On December 18, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer reported that it has entered into a distribution agreement with Swixx BioPharma AG ("Swixx") to be the exclusive distributor of the Company’s antibodies, DANYELZA (naxitamab-gqgk) for the treatment of patients with relapsed/refractory high-risk neuroblastoma and omburtamab, if approved, for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma in Eastern Europe, including Russia (Press release, Y-mAbs Therapeutics, DEC 18, 2020, View Source [SID1234573081]). DANYELZA (naxitamab-gqgk) 40mg/10mL was approved by the U.S. Food and Drug Administration ("FDA") on November 25, 2020 and is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. The Company plans to resubmit its ("BLA") to the FDA for omburtamab by the end of 2020 or in early 2021.

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The distribution agreement includes the European territories of Bosnia & Herzegovina, Bulgaria, Croatia, Czech Republic, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, Russia, Serbia, Slovakia and Slovenia. Under the terms of the agreement, Swixx will employ its sales and marketing expertise to distribute DANYELZA and omburtamab, if approved, in the territory. In addition, Swixx will submit registration files on behalf of Y-mAbs in certain parts of the territory. All other unpartnered geographies worldwide remain with the Company. Financial details were not disclosed.

"We are very pleased to enter this distribution agreement with Swixx, and hope to see a DANYELZA and omburtamab, if approved, being made available to appropriate children with unmet medical needs in Eastern Europe and Russia," said Thomas Gad, founder, Chairman and President at Y-mAbs.

Researchers at Memorial Sloan Kettering Cancer Center ("MSK") developed DANYELZA and omburtamab, which are exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the compounds and in Y-mAbs.

About DANYELZA (naxitamab-gqgk)

DANYELZA (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information for complete Boxed Warning and other important safety information.

On December 18, 2020 A second discovery out of Cancer Therapeutics CRC reported that it has entered into clinical trials (Press release, Cancer Therapeutics CRC, DEC 18, 2020, View Source;utm_medium=rss&utm_campaign=second-cancer-therapeutics-crc-discovery-enters-clinical-trials [SID1234573114]). If successful the discovery could be used to treat breast, prostate and lung cancers.

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The discovery centres around the KAT6A protein, which is commonly implicated in cancer types with solid tumours. Associate Professor Tim Thomas brought this protein to the attention of Cancer Therapeutics CRC researchers who then developed potential therapies that have subsequently been licensed to Pfizer through the CRC’s commercial partner, CTxONE.

"The KAT6A project was brought into Cancer Therapeutics as a novel target idea, and through collaboration between our experienced drug development researchers and the original investigators, we were able to develop potential therapies that were subsequently licensed to Pfizer. It is exciting to see this project enter clinical trials and take one step closer to becoming a treatment," said Brendon Monahan, Chief Scientific Officer at Cancer Therapeutics CRC.

This is the second of Cancer Therapeutics CRC’s discoveries to enter the clinic in 2 months. This highlights the value of collaborative research models such as Cancer Therapeutics CRC to the medical research ecosystem.

"The collaboration has resulted in such a successful team of high calibre researchers who have repeatedly shown they produce world class results. In addition to improving cancer treatment outcomes, this will potentially bring returns greater than $400 Million that can be used to grow Australia’s drug discovery capabilities creating jobs and opportunities" said Lisa Dube, CEO of Cancer Therapeutics CRC.

On December 18, 2020 Janux Therapeutics, reported a strategic collaboration and license agreement with Merck, known as MSD outside the United States and Canada, to discover, develop and commercialize innovative, next generation T cell engager immunotherapies for the treatment of cancer (Press release, Janux Therapeutics, DEC 18, 2020, View Source [SID1234573083]). The goal of the collaboration is to use Janux’s proprietary Tumor Activated T Cell Engager (TRACTrTM) technology to engineer novel, T cell engager drug candidates directed against two cancer targets selected by Merck. Under the terms of the agreement, Merck has received an exclusive worldwide license to products and intellectual property developed from this collaboration. In exchange, Janux will be eligible to earn up to $500.5 million per target in upfront and milestone payments plus royalties on sales of any product derived from the collaboration. Merck will fund research and development performed under the collaboration.

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T cell engagers are an emerging class of immunotherapies that bind to a tumor cell and recruit a patient’s T cells to eradicate tumor cells, but previous technologies have been constrained by dose-limiting toxicities, poor pharmacokinetic profiles, and attenuated efficacy. Janux’s proprietary TRACTr technology is designed to overcome these limitations by integrating tumor-specific activation with crossover pharmacokinetics to produce best-in-class T cell engager therapeutics. In preclinical studies, Janux TRACTr drug candidates have demonstrated comparable anti-tumor efficacy relative to standard T cell engagers but lack the associated liabilities related to cytokine release, healthy tissue toxicities, or systemic immune activation.

"At Janux, we have developed a technology to engineer best-in-class T cell engagers that are potent and highly tumor specific, which is essential for an immune response that kills tumor cells but spares healthy tissue," said David Campbell, Ph.D., President and CEO of Janux Therapeutics. "Partnering with Merck, a world leader in immuno-oncology, provides us with important expertise and resources in developing next generation T cell engager therapies that will make immunotherapy work for more cancer patients."