On December 19, 2019 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the 38TH Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 12:00 pm PT in San Francisco (Press release, Ultragenyx Pharmaceutical, DEC 19, 2019, View Source [SID1234552511]).

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The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On December 19, 2019 NexImmune, a clinical-stage biopharmaceutical company developing novel immune-therapeutics based on a proprietary Artificial Immune Modulation (AIM) nanotechnology platform, reported that it has received IND clearance for the Company’s second cellular therapy product (Press release, NexImmune, DEC 19, 2019, View Source [SID1234554874]). NEXI-002 is being developed for the treatment of Multiple Myeloma patients that have failed at least three prior lines of therapy. Scott P. Carmer, NexImmune’s President and CEO, commented "FDA clearance of our second IND this quarter marks another significant milestone for Neximmune and demonstrates our team’s focus and commitment to bringing novel therapies to patients with significant unmet need. NEXI-002 is a T cell therapy that consists of T cell populations directed against multiple tumor-relevant antigen targets. In addition, the T cells expanded by our proprietary E+E system consist of T cell subtypes critical to both potent anti-tumor activity and generation of the long-term immunologic memory required for durable responses. Because of this, we are hopeful NEXI-002 will address key limitations observed with other cellular immunotherapies."

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The Phase 1/2 trial of NEXI-002 will begin enrolling patients at clinical sites across the United States. Key sites planning to enroll patients include The Dana Farber Cancer Institute, Memorial Sloan Kettering, MD Anderson, and The Karmanos Cancer Institute. The trial is a multi-center, open-label, single-arm study evaluating the safety, tolerability and initial efficacy of adoptively-transferred patient-derived T-cells as a treatment for Multiple Myeloma patients that have failed >3 previous lines of therapy. The trial will evaluate a single dose of NEXI-002, with three patients enrolled in an initial safety cohort, followed by an expansion phase which will enroll up to 20 additional patients. All patients will be followed for at least one year.

Paul Richardson, MD, the RJ Corman Professor of Medicine at Harvard Medical School, Clinical Program Leader and Director of Clinical Research at Dana Farber’s Jerome Lipper Multiple Myeloma Center and the clinical trial’s lead investigator, stated "although we have made significant progress in treating patients diagnosed with Mulitple Myeloma, we continue to need novel treatment strategies and, in particular, effective immune therapy. For that reason, a significant unmet need remains for the vast majority of our patients. Novel immune-based therapies like NEXI-002 may represent a promising and practical option for those patients that have either relapsed after, or are refractory to, currently available treatment. We are excited to be a lead site for this trial and look forward to dosing our first patients."

About Multiple Myeloma
According the the Multiple Myeloma Research Foundation, multiple myeloma is a type of blood cancer that affects plasma cells. In multiple myeloma, malignant plasma cells accumulate in the bone marrow, crowding out the normal plasma cells that help fight infection. These malignant plasma cells then produce an abnormal antibody called M protein, which offers no benefit to the body and may cause tumors, kidney damage, bone destruction, and impaired immune function. The hallmark characteristic of multiple myeloma is a high level of M protein in the blood. Multiple myeloma typically displays the most activity in bone marrow, which includes the marrow in the spine, pelvic bones, ribs, shoulders, and hips.

Though it is rarely curable, multiple myeloma is a manageable disease that has seen rapid medical advancement over the past decade. Unfortuntaley, despite the introduction of novel therapies that offer many Multiple Myeloma patients temporary remission from their cancer, all patients will ultimately experience disease relapse.

About NEXI-002
NEXI-002 is an endogenous (non-genetically engineered) cellular therapy and includes populations of primed antigen specific CD8+ cells directed at five specific multiple myeloma antigen targets. Generating T cells against multiple tumor targets minimizes the potential for tumor escape. In addition, NEXI-002 contains T cell subtypes that are predominantly memory phenotypes, with the majority being characterized as stem cell memory and central memory T cells. T cell products that contain high proportions of memory cells, particularly central memory and stem cell memory, have been associated with potent anti-tumor activity, long-term T cell persistence and durable anti-tumor response.

On December 19, 2019 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform technology, ImmTOR, reported that it has entered into definitive agreements to sell securities in a private placement with a group of institutional investors and certain members of the Company’s Board of Directors (Press release, Selecta Biosciences, DEC 19, 2019, View Source [SID1234552494]). The lead institutional investors in the private placement were Vivo Capital LLC, Mangrove Partners, EcoR1 Capital LLC, BVF Partners L.P., and Boxer Capital LLC. The transaction is expected to result in gross proceeds to the company of approximately $70 million, before deducting placement agent fees and other offering expenses.

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The Company plans to use the net proceeds from the financing primarily to fund the ongoing head-to-head Phase 2 COMPARE clinical trial of its lead product candidate, SEL-212, for the treatment of chronic refractory gout, its gene therapy programs, and for general corporate purposes.

Pursuant to the terms of the securities purchase agreement, the Company will issue 45,977,011 units at a price of approximately $1.5225 per unit. Each unit consists of approximately one share of common stock and a warrant to purchase 0.5 of a share of common stock at an exercise price of $1.46. Certain institutional investors have elected to receive pre-funded warrants to purchase common stock in lieu of a portion of their common stock. The closing of the offering is subject to certain conditions and is expected to occur on Monday, December 23, 2019.

Cantor Fitzgerald & Co. served as placement agent for the offering.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws, and will be sold in a private placement pursuant to Regulation D of the Securities Act. The securities being issued in the private placement may not be offered or sold in the United States absent registration or pursuant to an exemption from the registration requirements of the Securities Act and applicable state securities laws. The Company has agreed to file a registration statement covering the resale of the securities acquired by the investors in the private placement.

This press release does 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 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.

On December 19, 2019 Bridge Biotherapeutics Inc., a clinical stage biotech company headquartered in Seongnam, South Korea, reported that the company filed an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) and the Ministry of Food and Drug Safety (MFDS) in Korea to initiate phase I/II study of BBT-176, a clinical candidate of targeted lung cancer therapy (Press release, BridgeBio, DEC 19, 2019, View Source [SID1234552532]).

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BBT-176, a novel epidermal growth factor receptor – tyrosine kinase inhibitor (EGFR-TKI) is designed to inhibit EGFR with C797S mutations, which arise as Tagrisso (osimertinib) resistant mutations following Tagrisso treatment in non-small cell lung cancer (NSCLC). BBT-176 exhibited strong antitumor efficacy in xenograft models harboring C797S triple mutations including Del19/T790M/C797S and L858R/T790M/C797S. Furthermore, BBT-176 displayed markedly enhanced efficacy when combined with anti-EGFR antibodies.

Since the EGFR C797S mutation was reported 3 years ago, as the first evidence of Tagrisso resistance, no major breakthroughs have been achieved to target the clinically relevant mutant variant that impedes covalent bond formation with irreversible EGFR inhibitors.[i]

The company plans to initiate dose escalation studies in advanced NSCLC patients in Korea next year and to develop further clinical studies in both Korea and the US afterwards. In the Phase I/II study with NSCLC patients, the safety, tolerability and efficacy of the candidate will be observed.

"We are proud of the IND submission for BBT-176, which has shown a potential to be developed as a highly mutant-selective, fourth-generation EGFR-TKI for NSCLC treatment," stated James Lee, CEO of Bridge Biotherapeutics. "Our team will make our best effort to develop novel targeted lung cancer therapy inhibiting C797S EGFR mutation."

BBT-176 was discovered by Korea Research Institute of Chemical Technology (KRICT), a Korean government research institute, and was licensed to Bridge Biotherapeutics in December 2018 for the worldwide exclusive right for further development.

Lung cancer is the leading cause of cancer death, accounting for about one-fifth of all cancer deaths. It is split into NSCLC and small cell lung cancer (SCLC) and NSCLC accounts for approximately 85% of all lung cancers. Overall, across 8 major countries including the U.S., 5 EU countries, China and Japan, the total NSCLC population as of 2015 is assumed 2 million and the incidence of NSCLC is expected to increase at an annual growth rate of 3.1% from 2015 to 2025.[ii]

On December 19, 2019 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology (I-O) company with a pipeline of immune checkpoint antibodies, adoptive cell therapies1 and cancer vaccines, reported the first patient dosed with AGEN1181, an anti-CTLA-4 antibody, in combination with balstilimab, Agenus’ PD-1 inhibitor (Press release, Agenus, DEC 19, 2019, View Source [SID1234552496]). Agenus began this study as a monotherapy in patients with advanced solid tumors within a Phase 1 dose escalation study (NCT03860272) in April 2019.

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The first patients to be treated with AGEN1181 as a monotherapy as well as the combination trial with PD-1 were dosed by Dr. Steven O’Day, Executive Director of the John Wayne Cancer Institute & Cancer Clinic, and a pioneer in delivering immune therapies to patients with cancer.

"AGEN1181, with its potential for enhanced immune activation and tumor fighting abilities, may bring superior benefit to a broader group of patients compared to first generation anti-CTLA-4 antibodies," said Dr. O’Day. "The pre-clinical data suggest the superiority of this molecule as a monotherapy and in combination with anti-PD-1, like balstilimab. Furthermore, AGEN1181 was designed to bring benefit to a broad population of patients both who respond to first generation molecules and those who do not respond due to a genetic polymorphism. Expanding the important immune priming benefit of CTLA-4 to a broad group of patients would be outstanding. I am thrilled to be working with this compound."

Balstilimab, the company’s proprietary anti-PD-1 antibody, is currently being evaluated as a monotherapy and in combination with zalfrelimab (a first generation CTLA-4) in trials designed to support a planned BLA filing in 2020 for patients with relapsed/refractory cervical cancer.

This expanded trial is another example of Agenus’ ability to rapidly advance its novel pipeline. Next-Gen CTLA-4 is a novel antibody from Agenus’ discovery engine, and with its own PD-1 molecule, Agenus has been able to move this important combination into the clinic with a high sense of urgency.

About AGEN1181
AGEN1181, a novel ‘Fc engineered’ antibody with potential for enhanced anti-tumor functions, is specifically designed to boost cancer killing immune cells and deplete intratumoral regulatory T cells that promote immune evasion. By enhancing binding to a specific Fc receptor, FcγRIIIA, on antigen-presenting cells or natural killer cells, AGEN1181 significantly enhances the therapeutic potential of anti-CTLA-4 therapy leveraging novel mechanisms that are not captured by the first-generation anti-CTLA-4 therapies. AGEN1181’s Fc engineered backbone improves the cross-talk between antigen-presenting cells and T cells, to enable optimal T cell priming, activation and formation of durable memory responses. Moreover, AGEN1181 enhanced binding to FcγRIIIA, significantly increases the potential to deplete intratumoral regulatory T cells, a significant barrier to successful anti-cancer immune responses. Notably, AGEN1181 is engineered to strongly bind both the low affinity and high affinity FcγRIIIA polymorphisms, unlike first generation molecules which weakly bind the low affinity polymorphism. AGEN1181 is designed to expand the benefit to an additional ~40% of patients with the low affinity polymorphism and enhance the benefits of CTLA-4 in all patients.