On March 13, 2017 SBI Pharmaceuticals Co., Ltd. (Head Office: Minato-ku, Tokyo; Representative Director and President: Yoshitaka Kitao; "SBI Pharma"), a subsidiary of SBI Holdings, Inc., engaged in the research and development of medicines using 5-ALA* (5-Aminolevulinic acid), reported that it has entered a licensing agreement with Chugai Pharmaceutical Co., Ltd. (Head Office: Chuo-ku, Tokyo; Chairman & CEO: Osamu Nagayama; "Chugai") to grant Chugai the exclusive marketing rights of "ALAGLIO Granule 1.5g ("ALAGLIO")" in Japan for visualizing tumor tissues during resection of bladder cancer (Press release, Chugai, MAR 13, 2017, View Source [SID1234518088]). The approval for manufacture and marketing of ALAGLIO is under application.

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In a surgical procedure to remove bladder cancer called TURBT** (TransUrethral Resection of Bladder Tumor), the key is discerning cancer tissues from normal tissues and removing all the cancerous tumors only. By orally administering ALAGLIO to a patient three hours before surgery and shedding a blue excitation light on the lesion during the surgery, the cancerous portion emits red fluorescence, thereby making it easier to distinguish between cancer and normal tissues.

Through the exclusive marketing agreement, SBI Pharma and Chugai join and further strengthen the effort to speed the availability of ALAGLIO, a new therapeutic option, for patients fighting against bladder cancer and healthcare professionals.

* 5-aminolevulinic acid (5-ALA)
An amino acid produced in mitochondria. It is an important substance that serves as a functional molecule related to energy production in the form of heme and cytochromes, and its productivity is known to decrease with age. 5-ALA is contained in food such as shochu lees, red wine and Asian ginseng. It is also known as a material forming chloroplasts in plants.

** Transurethral resection of bladder tumor (TURBT)
TURBT is a method to insert a surgical endoscope from the urethra without laparotomy, and resect the tumor while preserving the bladder function.

On March 13, 2017 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the fourth quarter and full year ended December 31, 2016 (Press release, Bellicum Pharmaceuticals, MAR 13, 2017, View Source [SID1234518086]).

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"Bellicum had a productive 2016 marked by significant clinical and regulatory progress with our diverse pipeline of controllable cell therapies," said Rick Fair, Bellicum’s President and Chief Executive Officer. "On the regulatory front, we clarified our path to approval with BPX-501 and rimiducid in Europe, and made substantial progress in dialogue with the FDA on the design of U.S. registration trials. In the clinic, we initiated two new programs late in 2016 with the first controllable CAR T and TCR candidates that incorporate our novel molecular switch technologies. These accomplishments, and continued enhancement of our technology platform, reinforce our belief that Bellicum is uniquely positioned to deliver important new cell therapies to patients."

2016 PROGRAM HIGHLIGHTS AND CURRENT UPDATES

BPX-501:
Adjunct T-cell therapy, administered after allogeneic hematopoietic stem cell transplantation (HSCT) to support faster immune recovery, improved infection control, and reduced mortality and GvHD, being evaluated in blood cancers and orphan inherited blood disorders

The Company advanced discussions with the U.S. FDA on BPX-501’s path for product registration in the U.S. Bellicum expects to conduct separate clinical trials for nonmalignant and malignant pediatric patients in the haploidentical stem cell transplant setting, including a non-randomized clinical trial in patients with orphan inherited blood disorders and a controlled trial in patients with blood cancers. Further details remain under discussion, and the Company expects to finalize discussions with the FDA on both protocols in the second quarter of 2017.

In the fourth quarter, Bellicum completed protocol assistance with the European Medicines Agency (EMA), and continues to enroll patients in the BP-004 clinical trial to support E.U. product registration of BPX-501 and rimiducid. The Company will also conduct a comparator trial to evaluate outcomes of pediatric patients with malignant and nonmalignant diseases receiving a matched unrelated donor (MUD) HSCT. The primary endpoint is event-free survival at six months (with events defined as transplant-related or non-relapse mortality, severe GvHD, and serious infection). The Company expects both trials to be fully enrolled later this year and to submit Marketing Authorization Applications (MAAs) to the European Medicines Agency (EMA) by mid-2018.

Bellicum reported updated clinical data from the BP-004 clinical trial that are supportive of E.U. regulatory submission. Data presented at the BMT (Bone Marrow Transplant) Tandem Meetings last month showed that cumulative incidence of treatment-related mortality remained very low, with six-month and one-year survival rates of 98.4 percent and 97.2 percent, respectively. There were no serious adverse events associated with use of BPX-501 or rimiducid. For the subset of 73 patients with six months of follow-up, the patients had rapid immune reconstitution, including full recovery and normalization of T cells, B cells and immunoglobulins.

Bellicum was awarded $16.9 million to help fund a global clinical program with BPX-501 in adults with high- and intermediate-risk AML (acute myeloid leukemia) from the Cancer Prevention and Research Institute of Texas (CPRIT). The Company expects to initiate a clinical trial later this year that evaluates outcomes of patients who receive a haploidentical HSCT with the post-transplantation cyclophosphamide (PT/Cy) regimen with or without BPX-501.

BPX-501 and rimiducid received Orphan Drug Designations in the E.U. and U.S.
BPX-601:
Novel GoCAR-T product candidate designed with the proprietary iMC activation switch to improve efficacy

Bellicum has begun patient dosing in an initial Phase 1 clinical trial with BPX-601 in patients with nonresectable pancreatic cancer who test positive for prostate stem cell antigen (PSCA). BPX-601 is believed to be the first CAR T-cell product candidate to enter the clinic that is designed to enable control over the expansion and stimulation of the cells.
BPX-701:
High affinity T-cell receptor (TCR) product candidate designed with the CaspaCIDe switch to improve safety

The Company initiated a Phase 1 clinical trial with BPX-701 in the U.S., initially in HLA-A2 positive patients with refractory or relapsed acute myeloid lymphoma (AML) and myelodysplastic syndromes (MDS) who test positive for preferentially-expressed antigen in melanoma (PRAME).
Preclinical Research:

Bellicum reported preclinical results with its GoCAR-T and GoTCR technologies at ASH (Free ASH Whitepaper) 2016, demonstrating unique control of cell proliferation and persistence, which may improve outcomes in solid tumors.

The Company continues to advance a novel dual-switch technology, with both activation and safety switches, designed to manage the persistence and safety of tumor antigen-specific CAR T cells, with new preclinical data planned to be reported at the American Association of Clinical Research (AACR) (Free AACR Whitepaper) annual meeting in April.
2016 AND RECENT CORPORATE UPDATES

Bellicum welcomed new Chief Executive Officer Rick Fair in preparation for the next phase of the Company’s growth. Mr. Fair joined Bellicum from Genentech/Roche, where he most recently served as Senior Vice President, Head of Oncology Global Product Strategy, and was responsible for the successful development and commercialization of multiple novel cancer therapies.

Bellicum expanded its research collaboration with Ospedale Pediatrico Bambino Gesù (OPBG) with its first partnered product candidate, an OPBG-designed, CaspaCIDe-enabled CD19 CAR, expected to enter the clinic in the second half of 2017 in patients with B-cell malignancies.

Bellicum and Adaptimmune entered a staged collaboration to develop next-generation T-cell therapies. The companies will evaluate the potential of Bellicum’s GoTCR technology and Adaptimmune’s affinity-optimized SPEAR T cells to create enhanced TCR product candidates, with the option of progressing to a two-target co-development and co-commercialization phase.

The Company expanded its research collaboration with Leiden University Medical Center for discovery of natural high-affinity TCRs for several cancers. Bellicum will provide financial support to LUMC over a three-year term in exchange for the right to exclusively license any high-affinity TCRs discovered under the new agreement.

Bellicum made substantial progress toward completion of its U.S. cGMP cellular therapy and viral vector manufacturing facility in Houston, and has recently begun manufacturing BPX-501 clinical trial drug for U.S. centers. Bellicum expects to complete the remaining work in mid-2017.
ANTICIPATED 2017 MILESTONES

Bellicum expects to:

Finalize FDA discussions on the U.S. regulatory path for BPX-501 and rimiducid in the second quarter, and begin enrollment of U.S. registration trials during the second half of 2017
Complete enrollment into the European BP-004 and MUD comparator clinical trials this year, and prepare for the anticipated MAA filings for BPX-501 and rimiducid to the EMA by mid-2018
Update BP-004 clinical data at medical conferences in 2017, including the annual meetings of the European Hematology Association (EHA) (Free EHA Whitepaper) and the American Society of Hematology (ASH) (Free ASH Whitepaper)
Initiate the CPRIT-supported clinical trial of BPX-501 in adults with AML by year-end
Present preclinical data on Bellicum’s novel dual-switch technology at AACR (Free AACR Whitepaper) in April
In addition, as part of its Bellicum partnership, OPBG is expected to initiate a Phase 1 clinical trial with a CaspaCIDe-enabled CD19 CAR in the second half of 2017 and report initial data in 2018.

Fourth Quarter and Full Year 2016 Financial Results

Cash Position and Guidance: Bellicum ended the year on December 31, 2016 with cash, restricted cash and investments totaling $113.4 million, compared to $150.4 million at December 31, 2015. In March 2017, Bellicum borrowed the final $10.0 million tranche under its agreement with Hercules Capital. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements through at least the first quarter of 2018. Projected cash outlays in 2017 include approximately $15 million for capital projects, primarily the completion of the buildout of in-house U.S. manufacturing facilities.

R&D Expenses: Research and development expenses were $15.1 million and $51.3 million for the fourth quarter and year ended December 31, 2016, respectively, compared to $10.2 million and $33.6 million during the comparable periods in 2015. The higher expenses in the 2016 periods were primarily due to an increase in BPX-501 clinical and manufacturing costs as a result of increased patient enrollment in clinical trials, an increase in costs related to BPX-601 and BPX-701 which entered the clinic in late 2016, and an increase in general research and development costs including personnel costs, and allocated overhead costs.

License fees were $0.3 million and $0.6 million for the fourth quarter and year ended December 31, 2016, respectively, compared to $3.0 million and $3.2 million for the comparable periods in 2015. The 2015 license fees were primarily due to a new license agreement with Agensys, an affiliate of Astellas, as consideration for rights granted to Bellicum under an agreement related to the BPX-601 product candidate, whereby Agensys was paid a non-refundable upfront fee of $3.0 million.

G&A Expenses: General and administrative expenses were $4.2 million and $16.9 million for the fourth quarter and year ended December 31, 2016, respectively, compared to $3.8 million and $12.7 million during the comparable periods in 2015. The increased G&A expenses in 2016 were primarily due to overall growth and public company related costs, including an increase in personnel, legal and accounting expenses and costs related to facilities, insurance and travel.

Net Loss: Bellicum reported a net loss of $19.9 million for the fourth quarter of 2016 and $69.2 million for the year ended December 31, 2016, compared to a net loss of $16.8 million and $48.5 million for the comparable periods in 2015. The results included non-cash, share-based compensation charges of $3.1 million and $12.3 million for the fourth quarter and year ended December 31, 2016, respectively, and $2.5 million and $8.4 million for the comparable periods in 2015.

Shares Outstanding:
At December 31, 2016, Bellicum had 27,155,565 shares of common stock outstanding.

On March 13, 2017 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) reported that Celgene Corporation has designated the development candidate focused on MTAP (methylthioadenosine phosphorylase) deleted cancers as a development candidate under the master research and collaboration agreement (the "Agreement") dated May 17, 2016 (Press release, Agios Pharmaceuticals, MAR 13, 2017, View Source [SID1234518084]).

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Under the terms of the Agreement, Celgene will pay Agios an $8 million designation fee for the MTAP pathway program. Exploratory research, drug discovery and early development on the MTAP pathway program is led by Agios, and Celgene will have an opt-in right on the program up through Phase 1 dose escalation for at least a $30 million fee. Upon opt-in, Celgene and Agios will have global co-development and co-commercialization rights with a worldwide 50/50 cost and profit share on the MTAP pathway program, and Agios will be eligible for up to $169 million in clinical and regulatory milestone payments.

"We are pleased that Celgene has designated this fourth development candidate discovered and developed at Agios since the beginning of our research collaboration with them in 2010," said Scott Biller, Ph.D., chief scientific officer at Agios. "We have clearly demonstrated our ability to translate novel Agios discoveries into important precision medicines in areas of high unmet need with our IDH portfolio. We look forward to exploring the potential of our MTAP program in patients following our expected IND submission by the end of this year."

MTAP-deletions are present in approximately 15 percent of all cancers. As described in a 2016 Cell Reports publication, Agios discovered a novel pathway in MTAP-deleted tumors which, when inhibited, results in robust anti-tumor activity in animal models. This pathway can be modulated by small molecule inhibitors, as demonstrated in a preclinical data presentation at the Keystone Tumor Metabolism meeting in Whistler, British Columbia on March 9, 2016. The presentation can be found under Publications in the Research section of the Agios website (www.agios.com).

The $8 million designation fee is expected to be received in the second quarter of 2017.