On December 20, 2019 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration ("NMPA") has granted Priority Review status to the New Drug Application ("NDA") for surufatinib for the treatment of patients with advanced non-pancreatic neuroendocrine tumors ("NET") (Press release, Hutchison China MediTech, DEC 20, 2019, View Source [SID1234552546]).

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"Surufatinib is our second drug to be granted priority review by the NMPA," says Christian Hogg, Chief Executive Officer of Chi-Med. "We are working closely with the NMPA as they review our NDA for the treatment of non-pancreatic NET. Surufatinib being granted priority review is a positive step forward in potentially bringing this innovative drug to patients, who currently have very limited treatment options."

In November 2019, the NDA for surufatinib for the treatment of non-pancreatic NET was accepted for review by the NMPA, and the U.S. Food and Drug Administration granted Orphan Drug designation to surufatinib for the treatment of pancreatic NET.

About Surufatinib

Surufatinib (previously known as HMPL-012 or sulfatinib) is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor ("VEGFR") and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies. Surufatinib is in several late-stage and proof-of-concept clinical trials in China and proof-of-concept clinical trials in the U.S.

According to Frost & Sullivan, the market for anti-angiogenesis VEGF/VEGFR inhibitors in China has grown from US$500 million in 2015 to over US$1.5 billion in 2019 and is expected to reach US$5 billion by 2026.

Chi-Med currently retains all rights to surufatinib worldwide.

Non-Pancreatic neuroendocrine tumors in China: In 2015, we initiated the SANET-ep study, a Phase III study of surufatinib in advanced neuroendocrine tumors – extra-pancreatic patients in China for whom there is no effective therapy. In June 2019, a 198 patient interim analysis was conducted, leading the independent data monitoring committee to determine that the study met the pre-defined primary endpoint of progression-free survival ("PFS") and should be stopped early. In November 2019, the NDA was accepted for review by the NMPA.

Pancreatic neuroendocrine tumors in China: In 2016, we initiated the SANET-p study, which is a pivotal Phase III study in patients with low- or intermediate-grade, advanced pancreatic neuroendocrine tumors in China. The primary endpoint is PFS. We expect an interim analysis in the first half of 2020 and enrollment to complete in 2020 (clinicaltrials.gov identifier: NCT02589821).

Neuroendocrine tumors in the U.S. and Europe: We are planning a U.S. registration study in neuroendocrine tumors patients based on the encouraging data from the Phase II and Phase III studies of surufatinib in neuroendocrine tumors in China (clinicaltrials.gov identifier: NCT02267967), and the ongoing Phase Ib study in the U.S. (clinicaltrials.gov identifier: NCT02549937).

Biliary tract cancer in China: In March 2019, we initiated a Phase IIb/III study comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy. The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier NCT03873532).

Immunotherapy combinations: In November 2018 and September 2019, we entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-programmed cell death protein 1 (PD-1) monoclonal antibodies. This included global collaborations to evaluate the combination of surufatinib with Tuoyi, approved in China by Shanghai Junshi Biosciences Co. Ltd, and with Tyvyt, approved in China by Innovent Biologics, Inc.

About Neuroendocrine Tumors (NET)

Neuroendocrine tumors form in cells that interact with the nervous system or in glands that produce hormones. They can originate in various parts of the body, most often in the gut or the lungs and can be benign or malignant. Neuroendocrine tumors are typically classified as pancreatic neuroendocrine tumors or non-pancreatic neuroendocrine tumors. Approved targeted therapies include Sutent and Afinitor for pancreatic neuroendocrine tumors, or well-differentiated, non-functional gastrointestinal or lung neuroendocrine tumors.

According to Frost and Sullivan, there were 19,000 newly diagnosed cases of neuroendocrine tumors in the U.S. in 2018. Importantly, neuroendocrine tumors are associated with a relatively long duration of survival compared to other tumors. As a result, there were approximately 141,000 estimated patients living with neuroendocrine tumors in the U.S. in 2018 of which over 90%, or approximately 132,000, were non-pancreatic neuroendocrine tumor patients.

In China, there were approximately 67,600 newly diagnosed neuroendocrine tumor patients in 2018 and, considering the current incidence to prevalence ratio in China, potentially as many as 300,000 patients living with the disease in the country1. It is estimated that approximately 80% of the patients living with neuroendocrine tumors in China are non-pancreatic neuroendocrine tumor patients.

1 According to Frost & Sullivan, in 2018, there were 19,000 newly diagnosed cases of NETs in the U.S and an estimated 141,000 patients living with NETs. The current incidence to prevalence ratio in China is estimated at 4.4, lower than the 7.4 ratio in the U.S. due to lower access to treatment options.

On December 20, 2019 Kyowa Kirin Co., Ltd. (TSE: 4151, President and CEO: Masashi Miyamoto, "Kyowa Kirin") reported that it has received the partial change approval for ORKEDIA (code name: KHK7580, generic name: evocalcet)*1 for the treatment of hypercalcemia in patients with parathyroid carcinoma or primary hyperparathyroidism who are unable to undergo parathyroidectomy or relapse after parathyroidectomy*2 from the Ministry of Health, Labor and Welfare (MHLW) in Japan (Press release, Kyowa Hakko Kirin, DEC 20, 2019, View Source [SID1234552543]).

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The approval is based on the result of the Phase 3 clinical trial that evaluates the efficacy of ORKEDIA for hypercalcemia in patients with parathyroid carcinoma or primary hyperparathyroidism who are unable to undergo parathyroidectomy or relapse after parathyroidectomy.

ORKEDIA was also granted Orphan Drug Designation for the treatment of hypercalcemia in patients with parathyroid carcinoma or primary hyperparathyroidism who are unable to undergo parathyroidectomy or relapse after parathyroidectomy by the MHLW on March 4, 2019.

The Kyowa Kirin Group companies strive to contribute to the health and well-being of people around the world by creating new value through the pursuit of advances in life sciences and technologies. *1.

About ORKEDIA (evocalcet)
ORKEDIA is a small molecular compound and a novel type of calcimimetics discovered by Mitsubishi Tanabe Pharma Corporation (President & Representative Director, CEO: Masayuki Mitsuka, "Mitsubishi Tanabe Pharma"). Kyowa Kirin signed a license agreement of ORKEDIA with Mitsubishi Tanabe Pharma for the rights of cooperative research, develop, market and manufacture of the product in Japan and some parts of Asia in March 2008. In March 2018, Kyowa Kirin received approval of ORKEDIA for secondary hyperparathyroidism in maintenance dialysis patients and later in May 2018, the product was launched in Japan market, named ORKEDIA Tablets. *

2. About Hypercalcemia in patients with parathyroid carcinoma or primary hyperparathyroidism who are unable to undergo parathyroidectomy or relapse after parathyroidectomy

Parathyroid carcinoma and primary hyperparathyroidism (PHPT) are diseases in which serum calcium levels are elevated due to over autonomous secretion of parathyroid hormone (PTH) from tumors of the parathyroid gland. Parathyroidectomy (PTx) is the only reliable method to treat parathyroid carcinoma and PHPT. Control of hypercalcemia can prove challenging in cases of PHPT where PTx cannot be considered because of concomitant diseases or where PHPT recur after PTx and parathyroid carcinoma, although patients with such hypercalcemia are very rare. Hypercalcemia causes symptoms of fatigue, polyuria, thirst, and renal impairment and severe hypercalcemia can result in death due to a hypercalcemic crisis

On December 20, 2019 AstraZeneca reported that it has agreed to sell the commercial rights to Arimidex (anastrozole) and Casodex (bicalutamide) in a number of European, African and other countries1 to Juvisé Pharmaceuticals (Press release, AstraZeneca, DEC 20, 2019, View Source [SID1234552541]).

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The medicines, used primarily to treat breast and prostate cancers, have lost their compound patent protection in these countries. AstraZeneca already divested the rights to both Arimidex and Casodex in the US in 2017.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: "Arimidex and Casodex are important established medicines and we are pleased that Juvisé Pharmaceuticals will now take on the work of making sure patients continue to have access to them. Today’s agreement is part of a broader strategy of reducing our portfolio of mature medicines to reallocate resources towards developing our pipeline of new medicines."

Financial considerations

As there were no closing conditions to the divestment, the agreement became effective upon signing. Juvisé Pharmaceuticals has made an upfront payment of $181m to AstraZeneca and may also make future sales-contingent payments of up to $17m. Income arising from the upfront payment will be reported in AstraZeneca’s financial statements in the fourth quarter of 2019. Income from the upfront and any future payments will be reported within Other Operating Income & Expense. In 2018, Arimidex had sales of $37m in the countries covered by this agreement, while Casodex had sales of $24m. The divestment does not change the Company’s financial guidance for 2019.

About Arimidex

Arimidex (anastrozole) is an aromatase inhibitor, indicated primarily for the adjuvant treatment of postmenopausal women with hormone receptor-positive early breast cancer, the first-line treatment of postmenopausal women with hormone receptor-positive or hormone receptor-unknown locally advanced or metastatic breast cancer and the treatment of advanced breast cancer in postmenopausal women with disease progression, following tamoxifen therapy.

About Casodex

Casodex (bicalutamide) is an androgen-receptor inhibitor, indicated for use in combination therapy with a luteinising hormone-releasing hormone analogue for the treatment of Stage D2 metastatic carcinoma of the prostate.

On December 19, 2019 BridgeBio Pharma, Inc. (Nasdaq: BBIO) reported that it will present at the 38th Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2020 at 3 PM (PST) in San Francisco (Press release, BridgeBio, DEC 19, 2019, View Source [SID1234576261]).

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The presentation will be webcast live and can be accessed at www.bridgebio.com on the For Investors page under News & Events. The webcast will be available for replay for 30 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.