On January 30, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported that the Company has received notice from the U.S. Food and Drug Administration (FDA) that U.S. studies of BPX-501 have been placed on a clinical hold following three cases of encephalopathy deemed as possibly related to BPX-501 (Press release, Bellicum Pharmaceuticals, JAN 30, 2018, View Source [SID1234523650]).

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Bellicum is awaiting formal communications from the FDA to determine the requirements for resuming studies, and will be working closely with the FDA to address their questions. The FDA clinical hold does not affect the ongoing BP-004 registration trial in Europe.

Encephalopathy has been reported in the allogeneic stem cell transplant literature. Risk factors for encephalitis/encephalopathy after allogeneic stem cell transplants include prolonged immunodeficiency, selected medications, infections, and inflammatory processes such as graft versus host disease. Bellicum has treated more than 240 patients with BPX-501 cells on three allogeneic haploidentical stem cell transplantation protocols. These three cases are complex, with a number of potential confounding factors—including, in certain of the cases, prior failed transplants, prior history of immunodeficiency, concurrent infection, and administration of rimiducid in combination with other medications. Bellicum is working with FDA to evaluate the risk of encephalopathy in patients receiving BPX-501.

On January 31, 2018 Astellas reported that it has Appointed Kenji Yasukawa as President and CEO (Press release, Astellas, JAN 30, 2018, View Source [SID1234523647]).

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On January 31, 2018 Astellas reported that it has appointed Kenji Yasukawa as President and CEO (Press release, Astellas, JAN 30, 2018, View Source [SID1234523647]).

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On January 31, 2018 Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, "Astellas") reported the financial results for the first nine months of fiscal year 2017 ending March 31, 2018 ("FY2017") (Press release, Astellas, JAN 30, 2018, View Source [SID1234523646]).

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"Key global products including XTANDI continued to demonstrate steady growth in the first nine months of FY2017. We achieved many milestones including the acquisition of Mitobridge, Inc. in January 2018 by exercising the option right to acquire Mitobridge. The transaction accelerates Astellas’ research and development in diseases associated with mitochondrial dysfunctions," said Yoshihiko Hatanaka, president and CEO, Astellas. "We remain committed to creating innovative medical solutions and delivering value for patients and all stakeholders, as we continue to advance our strategic plan by maximizing the value of our products, creating innovation and pursuing operational excellence."

Quarterly Revenue Highlights

Sales in the first nine months of FY2017 decreased 0.6% compared to those in the corresponding period of the previous fiscal year ("year-on-year") to ¥999.4 billion due to the impact of certain items such as the transfer of the global dermatology business in April 2016 and the transfer of long-listed products in Japan in April 2017.

Oncology franchise
Sales of XTANDI increased 16.2% year-on-year to ¥219.9 billion. Sales grew steadily in all regions including Japan, the Americas, EMEA1 and the Asia and Oceania region.

Urology OAB franchise
Sales of Betanis / Myrbetriq / BETMIGA increased 30.0% year-on-year to ¥93.1 billion. Sales increased in all regions of the world. Sales of Vesicare, however, decreased 12.1% year-on-year to ¥78.5 billion.

Transplantation franchise
Sales of Prograf increased 5.6% year-on-year to ¥150.2 billion, and continued to grow in Japan, EMEA and the Asia and Oceania regions.

Other new and key products
In the Japanese market, continued growth was achieved for products such as Celecox for the treatment of inflammation and pain, Symbicort for the treatment of bronchial asthma, Suglat for the treatment of type 2 diabetes, and Cimzia for the treatment of adult patients with rheumatoid arthritis. Meanwhile, we are working to further penetrate the hypercholesterolemia market following our launch of Repatha, which occurred in April 2016, and of LINZESS for the treatment of irritable bowel syndrome with constipation, which launched in March 2017. In the Americas, sales of azole antifungal CRESEMBA grew.

Sales by Region2

Sales in Japan and EMEA decreased, while sales in the Americas and the Asia and Oceania region increased. As for the Japanese market, sales decreased 13.7% year on year to ¥309.0 billion largely due to the impact of transferring 16 long-listed products in April 2017, and the introduction of generics for Micardis for the treatment of hypertension in June 2017. In EMEA, sales decreased due to the impact of transferring the dermatology business in April 2016, yet sales showed an increase when calculated excluding this impact.

FY2017 Guidance

The company’s business forecasts for FY2017 remain unchanged from the consolidated full-year business forecasts announced in October 2017.

Strategic Quarterly Highlights

Astellas continues to create sustainable growth over the mid-to-long term through the pursuit of three main strategies: "Maximizing the Product Value," "Creating Innovation" and "Pursuing Operational Excellence." The company achieved multiple accomplishments against these strategies as outlined below:

Maximizing the Product Value

Continued to maximize the growth of the Oncology franchise centered on XTANDI and the Urology OAB franchise including Vesicare and Betanis / Myrbetriq / BETMIGA with new launches across various countries and a growth in franchise sales globally.
Extended the agreement pertaining to the sale of Telmisartan (Micardis family) in October 2017.
Concluded a co-promotion agreement with MSD K.K. for a combination drug of the DPP-4 inhibitor sitagliptin phosphate hydrate and the SGLT2 inhibitor ipragliflozin L-Proline for the treatment of type-2 diabetes in Japan in November 2017.
Creating Innovation

Launched "JOINUS," a new drug discovery program using a drug-repositioning compound library jointly conducted by Astellas, Mitsubishi Tanabe Pharma, and Daiichi Sankyo, in October 2017.
Announced a collaboration utilizing Universal Donor Cell Technology to create a cell therapy product that can be administered to any recipient without the need for human leukocyte antigen matching in October 2017.
Announced – and completed – execution of Astellas’ exclusive option right to acquire Mitobridge in December 2017 and January 2018, respectively.
The following lists the main development advances achieved during this quarter of FY2017:

Announced that the first Phase 3 trial in Japan evaluating roxadustat for anemia in chronic kidney disease patients demonstrated positive efficacy results in October 2017.
Submitted a new drug application for Gonax 12-week extended-release formulation for treatment of prostate cancer in Japan in November 2017.

(1) EMEA: Europe, the Middle East and Africa
(2) Sales by Region: based on location of sellers

On January 30, 2018 BridgeBio Pharma reported that it is has licensed infigratinib (BGJ398), a highly potent and selective inhibitor of the tyrosine kinase receptor FGFR, from Novartis (Press release, BridgeBio, JAN 30, 2018, View Source [SID1234523638]). In addition, BridgeBio announced that it was launching new subsidiary QED Therapeutics to drive development of infigratinib with an initial financial commitment of $65 million.

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FGFR has been implicated as a driver mutation across multiple oncologies – including roughly one out of every five cases of cholangiocarcinoma and urothelial carcinoma – and in multiple forms of pediatric skeletal dysplasias, namely achondroplasia, which affects one out of every 20,000 live births.

Infigratinib is currently in a Phase 2 clinical trial for patients with chemotherapy-refractory bile duct cancer (cholangiocarcinoma) containing FGFR2 fusions. Early clinical results, recently published in the Journal of Clinical Oncology, demonstrated that the compound showed meaningful activity in this population.

"We are committed to moving this compound forward in late-stage development and further proving the strong efficacy in cancer that has already been demonstrated across multiple trials," said Daniel Hoth, M.D., QED’s chief medical officer, who has devoted over three decades to drug development, including time as chief of the Investigational Drug Branch of the National Cancer Institute (NCI).

Cholangiocarcinoma affects approximately 6,000 to 8,000 patients a year in the United States. Treatment options are limited, and survival rates vary depending on whether cholangiocarcinoma is found on the bile duct branches within the liver versus those outside of the liver.

"Despite immense strides in studying potential drugs in cholangiocarcinoma, there remains significant need to provide options to these patients," said Stacie Lindsey, president of the Cholangiocarcinoma Foundation. "The patients and caregivers we work with are very hopeful given data already generated with infigratinib, and we are excited that the passionate team at BridgeBio and QED are working to advance this drug."

BridgeBio co-founder and investor Frank McCormick, Ph.D., head of the NCI’s Ras initiative and former CSO and co-founder of Onyx Pharmaceuticals, remarked "Infigratinib embodies the crux of what we set out to do at BridgeBio: develop targeted therapies for genetically-driven tumors and monogenic disorders."

In addition to its clinical data in FGFR-driven cancer, infigratinib has demonstrated potential in skeletal dysplasias, including achondroplasia. In the early work published in the Journal of Clinical Investigation, researchers demonstrated that low doses of infigratinib corrected pathological hallmarks of achondroplasia in mouse models.

Neil Kumar, Ph.D., chief executive officer of BridgeBio, noted that with infigratinib, "We have a late-stage, targeted oncology compound that has demonstrated clear efficacy in the clinic. With the same molecule, we have a potential best-in-class therapy to treat achondroplasia at its source."

While specific terms of the deal have not been disclosed, BridgeBio has committed $65 million in financing to QED, which is inclusive of a substantial upfront payment to Novartis as well as equity in QED. Novartis will also receive additional payments upon the realization of development and sales milestones as well as royalties.