On January 7, 2019 bluebird bio, Inc. (Nasdaq: BLUE) and Inhibrx, Inc. (Inhibrx) reported that they have entered into an exclusive license agreement to research, develop and commercialize chimeric antigen receptor (CAR) T cell therapies using Inhibrx’s proprietary single domain antibody (sdAb) platform to multiple cancer targets (Press release, bluebird bio, JAN 7, 2019, View Source [SID1234532541]). The small size of sdAbs may enable the generation of more complex CAR T cell products such as those designed to combine additional functions into a single CAR molecule or recognize multiple tumor antigens simultaneously.

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"Access to the Inhibrx sdAb binder technology will allow us to combine the advancements we’ve made with our T cell therapy platform with their sdAb binder technology to generate novel cellular therapies with the potential to help patients in their fight against cancer," said Philip Gregory, D. Phil., chief scientific officer, bluebird bio. "The technology from Inhibrx adds to our growing portfolio of tools and technologies that we can combine with our internal lentiviral vector, CAR and T cell expertise to discover potential new product candidates designed to recognize tumor-specific proteins expressed by cancer cells and kill them upon engagement."

"We are pleased to have formalized our relationship with bluebird bio, allowing us to couple our proprietary sdAb platform with a leading cell therapy platform," said Brendan Eckelman, Chief Scientific Officer and Executive Vice President of Corporate Strategy of Inhibrx. "Together with bluebird bio, we have generated compelling proof of concept preclinical data on the utility of incorporating our sdAbs into bluebird bio’s constructs for CAR-T cell generation."

Under the terms of the license agreement, Inhibrx will provide bluebird bio the exclusive worldwide rights to develop, manufacture and commercialize certain cell therapy products containing sdAbs directed to various cancer targets. bluebird bio will be responsible for the clinical development and commercialization of the cancer-targeting CAR-T products. Inhibrx received a $7.0 million upfront payment and is also entitled to receive specified developmental milestone payments as well as percentage tiered royalties on future product sales.

On January 7, 2019 Generex Biotechnology Corporation (OTCQB:GNBT) is reported that Joe Moscato, the company’s CEO is attending the annual J.P. Morgan Healthcare Conference from January 7 to 9, 2019 (Press release, Generex, JAN 7, 2019, View Source [SID1234532540]). Mr. Moscato is meeting with leading healthcare investors to present the corporate vision for Generex Biotechnology as an integrated healthcare holding company with a unique strategy to provide end-to-end, patient centric solutions to enhance the doctor patient relationship.

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With 9,000 attendees and representation from over 450 companies, the annual J.P. Morgan Healthcare Conference is the largest and most informative healthcare investment symposium in the industry, bringing together industry leaders, emerging fast-growth companies, innovative technology creators, and members of the investment community.

"We are pleased to unveil the "New" Generex to the biotech and healthcare investment community," stated Joe Moscato, CEO of Generex. "After two-plus years of reorganizing the operations, capital structure, and strategic vision for the company, Generex is poised to deliver on our corporate mission to provide physicians, hospitals, and healthcare providers an end-to-end solution for patient centric care from rapid diagnosis through delivery of personalized therapies, streamlining care processes, minimizing expenses, and delivering transparency for payers. Entering 2019, we are building value in Generex by executing on our strategic plan with our newly acquired MSO and pharmacy network, the advancement of our clinical stage assets in immune-oncology, and through acquisitions in cutting-edge areas like regenerative medicine."

On January 7, 2019 Rainier Therapeutics, Inc., a privately-held clinical stage drug development company, reported that its targeted antibody vofatamab has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for treatment of patients with advanced or metastatic urothelial cell carcinoma (bladder cancer) that is positive for FGFR3 mutation and/or fusion (Press release, Rainier Therapeutics, JAN 7, 2019, View Source [SID1234532539]).

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"This Fast Track designation underscores the great unmet medical need that exists for the treatment of bladder cancer," said Scott Myers, Chairman and CEO of Rainier Therapeutics. "As the only antibody specifically targeted to FGFR3 we know to be in clinical development, we believe vofatamab offers a promising therapeutic option. We look forward to further data from our ongoing trials and working to advance our development efforts."

The FDA’s Fast Track designation is a process to facilitate development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Through the Fast Track program, more frequent meetings may be scheduled with the FDA to discuss the drug’s development plan and to ensure the collection of appropriate data needed to support approval. Additionally, the drug may qualify for accelerated approval and priority review and, at the time of a biologics license application (BLA) filing, the drug candidate’s sponsor may be eligible to submit completed sections of the BLA on a rolling basis before the complete application is submitted.

"Fast Track designation offers the potential to reduce development time and cost associated with bringing a drug to patients," said Valerie Fauvelle, Vice President, Regulatory Affairs at Rainier Therapeutics. "We look forward to working with the FDA to rapidly advance vofatamab through the clinical development and regulatory processes."

Vofatamab (B-701) is an antibody specifically targeted against the fibroblast growth factor receptor 3 (FGFR3), a known driver of bladder and other cancers. Vofatamab is the most advanced targeted biologic specific for FGFR3 known by Rainier to be in clinical development.

Rainier Therapeutics has ongoing Phase 1b and Phase 2 clinical studies of vofatamab in metastatic bladder cancer – the Fierce 21 and Fierce 22 studies. In addition, Rainier Therapeutics plans to study vofatamab in early stage bladder cancer – the Fierce 23 trial.

The Fierce 21 trial is evaluating vofatamab alone and in combination with docetaxel versus docetaxel alone to determine safety and efficacy in the treatment of patients with locally advanced or metastatic bladder cancer with FGFR3 mutant/fusion who have relapsed after, or are refractory to, at least one prior line of chemotherapy. For more on this trial, visit www.clinicaltrials.gov (NCT0240542).

The Fierce 22 trial is evaluating vofatamab in combination with pembrolizumab, an immune checkpoint inhibitor, to determine safety, tolerability and efficacy in the treatment of patients with locally advanced or metastatic bladder cancer, who have progressed following platinum-based chemotherapy and who have not received prior immune checkpoint inhibitor therapy. For more on this trial, visit www.clinicaltrials.gov (NCT03123055).

The Fierce 23 trial will evaluate vofatamab monotherapy in non-muscle invasive bladder cancer (NMIBC). This trial is planned to start in 2019.

On January 7, 2019 OncoSec Medical Incorporated (OncoSec) (NASDAQ:ONCS), a company developing novel cancer immunotherapies, reported it has established a collaboration with the GOG Foundation, Inc., (GOG Foundation), to conduct a registration-enabled study of TAVO (tavokinogene telseplasmid) in women with recurrent/persistent cervical cancer (OMS-150) (Press release, OncoSec Medical, JAN 7, 2019, View Source [SID1234532538]).

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In June, 2018, KEYTRUDA (pembrolizumab) received accelerated approval from the FDA for the treatment of advanced cervical cancer with disease progression during or after chemotherapy based on data from a single-arm 98 patient study that showed a 14% overall response rate (ORR). Previous data in other advanced solid tumors demonstrate that TAVO combined with KEYTRUDA can induce objective responses in patients who do not respond to anti-PD-1 antibody monotherapy.

In this registration-directed clinical trial, OncoSec and GOG will evaluate the combination of TAVO and commercially available KEYTRUDA with the goal of achieving a clinically meaningful response rate greater than what has already been demonstrated with KEYTRUDA alone (14%). OncoSec and the GOG Foundation plan to enroll approximately 80 to 100 patients, who qualify for standard of care treatment with KEYTRUDA, in this single-arm study with TAVO. The trial will be open to patients with surface or subcutaneous lesions that are accessible via TAVO’s current delivery system. Patient enrollment is expected to begin in the first half of 2019. Importantly, should a clinically meaningful increase be observed in patients receiving the TAVO beyond that which they receive from KEYTRUDA alone, OncoSec plans to seek accelerated approval of TAVO in this patient population.

"KEYTRUDA is only the second drug in 30 years to be approved for the treatment of cervical cancer and, though it represents significant progress, the number of patients who can benefit is limited. Our goal is to improve upon the 14% KEYTRUDA response rate with the addition of TAVO," said Daniel J. O’Connor, President and Chief Executive Officer of OncoSec. "We believe that TAVO, our proprietary intratumoral plasmid-based IL-12, is an excellent complement for expanding the clinical benefit of anti-PD-1 therapies, especially for those patients that are resistant to anti-PD-1 therapies. Given that KEYTRUDA is already approved and reimbursed for this indication, this study fits perfectly with our strategy of identifying opportunities to conduct small, relatively low-cost single-arm clinical studies that have the potential to offer a rapid path to drug approval and commercialization."

The study will be conducted within GOG Foundation’s network under OncoSec’s investigational new drug (IND) application for TAVO. The GOG Foundation is a world-renowned non-profit organization with the purpose of conducting clinical research for the prevention and treatment of all gynecologic cancers, such as ovarian cancer, cervical cancer, endometrial cancer, vulvar cancer, and vaginal cancer. Its members make up a multi-disciplinary group, consisting of gynecologic oncologists, medical oncologists, pathologists, radiation oncologists, nurses, statisticians, and basic scientists.

"Conducting research that can lead to promising new therapies for women facing cervical cancer and other gynecological malignancies is central to our mission, and this collaboration is an exciting opportunity to bring our esteemed network and expertise in quality scientific research to the table," said Larry J. Copeland, MD, GOG Foundation President. "We’re grateful to play a role in this trial and look forward to advancing this therapy through the clinic."

On January 7, 2019 Stemline Therapeutics, Inc. (NASDAQ:STML), a biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that it has submitted the marketing authorization application (MAA) for ELZONRIS (tagraxofusp) to the European Medicines Agency (EMA) (Press release, Stemline Therapeutics, JAN 7, 2019, View Source [SID1234532537]). The MAA seeks approval for treating patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In November 2018, the EMA granted the ELZONRIS MAA accelerated assessment.

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On December 21, 2018, ELZONRIS was approved by the U.S. Food and Drug Administration (FDA) for the treatment of BPDCN in adult and pediatric patients, two years and older, in both treatment-naïve and previously-treated populations. ELZONRIS is the first treatment approved for BPDCN and the first approved CD123-targeted therapy.

"The submission of the ELZONRIS MAA is another major step forward for providing this important targeted treatment to patients, globally," said Ivan Bergstein, M.D., CEO of Stemline Therapeutics. "We look forward to working closely with the EMA to ensure this treatment is available to patients as quickly as possible. In parallel, our commercial team is continuing its ongoing effort to raise awareness of both CD123 testing and BPDCN worldwide. Potential European approval offers us an opportunity to significantly increase the number of patients who may benefit from ELZONRIS."

About ELZONRIS
ELZONRIS (tagraxofusp), a CD123-directed cytotoxin, was approved by the Food and Drug Administration (FDA) on December 21, 2018 for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In November 2018, the European Medicines Agency (EMA) granted ELZONRIS accelerated assessment to the marketing authorization application (MAA), which was submitted to the EMA in January 2019. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and other CD123 positive diseases.

About BPDCN
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.