On February 4, 2020 Takeda Pharmaceutical Company Limited reported the (Press release, Takeda, FEB 4, 2020, View Source [SID1234553842]).

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Underlying Revenue declined -1.2% vs FY2018 Q3 YTD pro-forma2, expected to recover in Q4 resulting in flat to slightly increasing for the full year

Takeda’s 14 global brands, with reported revenue of 836.4 billion yen in aggregate, posted a strong year- over-year underlying revenue growth of +20%, driven by ENTYVIO (+35.4%), TAKHZYRO (+622.2%), and NINLARO (+28.9%).
Underlying revenue growth year-to-date was solid in the key business areas of GI (+10%), Plasma Derived Therapy (PDT) Immunology (+5%), Oncology (+7%), and Neuroscience (+5%), while Rare Diseases declined (-11%) for the following reasons:
Rare Hematology (-14%) continues to be impacted as expected by intensified competition and increasing price pressure.
Hereditary Angioedema (HAE) (-11%) continues to be negatively affected by stocking in the prior fiscal year, as well as generic entry for FIRAZYR.
Rare Metabolic (-4%) continue to be impacted by NATPARA which was recalled in the U.S. in September 2019.
Please refer to note iii to the table entitled "Reported Results for FY2019 Q3 YTD (April – December)" below for Core Operating Profit definition.
FY2018 Q3 YTD pro-forma baseline represents the sum of Takeda revenue for FY2018 Q3 YTD (Apr-Dec) plus Shire revenue for the same period, where Shire revenue was converted to JPY at the rate of $1 = 111 JPY (average FX rate for FY2018) and converted from US GAAP to IFRS with no material difference; Takeda revenue and Shire revenue was adjusted to remove the revenue from divested assets. Please see the appendix for more details.

Underlying Core Operating Profit Margin of 30.9% for FY2019 Q3 YTD driven by cost synergies and OPEX efficiencies

Reported Operating Profit declined year-over-year -42.9% to 162.5 billion yen, largely impacted by non-cash purchase accounting expenses including the unwinding of inventory step-up and amortization of intangible assets. Reported Operating Profit was also impacted by significant one-time costs related to the Shire integration.
Core Operating Profit increased year-over-year +129.9% to 792.2 billion yen, primarily due to the consolidation of Shire, while also benefitting from the strong performance of Takeda 14 global brands, cost synergies and improved OPEX efficiency.
Underlying Core Operating Profit Margin year-to-date was 30.9% reflecting continued OPEX discipline and cost synergies.
Underlying Core EPS year-to-date was 359 yen.
R&D Engine Delivered Several Important Pipeline Milestones in Q3

Wave 1 pipeline assets achieving important milestones:
Phase 3 study start for TAK-788 in treatment naïve Non-Small-Cell Lung Cancer (NSCLC) with exon 20 insertion mutations, and pevonedistat (TAK-924) in unfit Acute Myeloid Leukemia.
Updated Dengue vaccine candidate TAK-003 results from the phase 3 study were presented at the American Society of Tropical Medicine and Hygiene (ASTMH) Annual Meeting.
Announced partnership with MD Anderson Cancer Center which includes the development of ongoing Phase 1/2a Study of TAK-007, a CD19 CAR-NK.
Global Brands generating additional data in new indications:
Phase 3 trial of NINLARO (TOURMALINE-MM4) as first line maintenance therapy met primary endpoint (PFS) in multiple myeloma patients not treated with stem cell transplantation.
ALUNBRIG ongoing phase 3 data continued to show reduction in disease progression after two years as a first line treatment in adults with advanced anaplastic lymphoma kinase-positive (ALK+) NSCLC who had not received a prior ALK inhibitor.
Continued emphasis on divesting non-core assets and deleveraging to focus the business

Net debt / adjusted EBITDA at 4.1x having paid full-year dividend and tax on XIIDRA proceeds.
Negotiations ongoing for further potential non-core asset divestments.

Costa Saroukos, Chief Financial Officer, commented:
"Takeda’s third quarter results demonstrated a continuation of our solid year-to-date financial and business performance, driven by our 14 global brands, powerful R&D engine, and OPEX improvements that will help to ensure our sustainable growth. We are again increasing our full year guidance to reflect strong business momentum and faster than anticipated realization of synergies.

Now operating as One Takeda, we continue to execute as anticipated on our business priorities to drive long-term value, including the realization of $2 billion in cost synergies by the end of FY 2021, divest $10 billion in non-core assets to optimize our portfolio, and rapidly de-leverage our net debt / adjusted EBITDA towards our goal of 2x within fiscal years ending March 2022 – March 2024. In parallel, our R&D engine continues to advance highly innovative medicines that make a critical difference to patients through both Takeda’s global brands and new molecular entities, with multiple important data readouts on the horizon. Executing on these priorities will maximize value creation for all of our stakeholders and position Takeda for continued success."

Reported Results for FY2019 Q3 YTD (April – December)

Underlying results compares two periods (quarters or years) of financial results under a common basis and is used by management to assess the business. These financial results are calculated on a constant currency basis and excluding the impact of divestitures and other amounts that are unusual, non-recurring items or unrelated to our ongoing operations.
Growth versus FY2018 Q3 YTD pro-forma. FY2018 Q3 YTD pro-forma baseline represents the sum of Takeda revenue for FY2018 Q3 YTD (Apr-Dec) plus Shire revenue for the same period, both adjusted to remove the revenue from divested assets, converted to JPY at the rate of $1 = 111 JPY (average FX rate for FY2018), and converted from US GAAP to IFRS with no material differences. Please see the appendix for more details.
Core Operating Profit represents net profit adjusted to exclude income tax expenses, the share of profit or loss of investments accounted for using the equity method, finance expenses and income, other operating expenses and income, amortization and impairment losses on acquired intangible assets and other items unrelated to Takeda’s core operations, such as purchase accounting effects and transaction related costs.
Attributable to the owners of the company.
FY2019 Management Guidance: Upgrading guidance to reflect positive business momentum

Constant Exchange Rate growth (applying FY2018 full year average foreign exchange rate of 111 JPY/USD) compared to baseline of JPY 3,300 billion (Rounded pro-forma April 2018-March 2019 combined revenue of Legacy Takeda and Legacy Shire, converted at April 2018-March 2019 average exchange rate of 111 JPY/USD; also adjusted to remove the revenue from divested assets such as Techpool, Multilab, and TACHOSIL from Legacy Takeda, and the oncology portfolio and XIIDRA from Legacy Shire) and converted from US GAAP to IFRS, without material differences.

On February 4, 2020 Nanospectra Biosciences, Inc., a medical device company pioneering a novel use of nanomedicine for selective thermal ablation, reported the start of a pivotal study to determine the efficacy of using MRI/US fusion imaging technology to direct focal ablation of prostate tissue using nanoparticle-directed laser ablation (Press release, Nanospectra Biosciences, FEB 4, 2020, View Source [SID1234553858]). The initial two patient procedures in the pivotal trial have been completed at the University of Michigan, which also participated in the pilot study. The AuroLase pivotal study follows the successful first-in-human pilot study that enrolled 46 subjects at three U.S. sites.

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Clinical and functional outcomes of a subset of subjects at the lead site in the feasibility study were published in the prestigious Proceedings of the National Academy of Sciences (PNAS) last August in a paper titled, ‘Gold Nanoshell-Localized Photothermal Ablation of Prostate Tumors in a Clinical Pilot Device Study’. Thirteen of the first 15 prostate cancer patients treated in the clinical trial of the nanoparticle-based, focal therapy showed no detectable signs of cancer upon biopsy in the target ablation zone a year after treatment. The final results of the feasibility study are expected later this year.

The pivotal study is an open-label, multi-center, single-treatment study of AuroLase Therapy for the focal ablation of prostate tissue via nanoparticle directed near infrared irradiation and approved by the FDA under the original IDE. Up to sixty patients will be enrolled at up to eight clinical trial sites throughout the U.S.

Efficacy of focal ablation of prostate tissue will be assessed by MRI/Ultrasound guided target biopsy six months after laser treatment and at one year via targeted biopsy and standard systematic biopsy. Per standard of care, patient follow up will continue beyond the one-year study visit but will be outside the scope of the study. Patients will be consented for up to five years in order to track their disease status and progression or recurrence, if any.

"With AuroLase Therapy, we aspire to deliver a new standard of care for primarily intermediate risk localized prostate cancer patients and clinicians with the potential to significantly reduce side effects, retain all downstream clinical options for future treatment, and enable a more rapid return to a normal lifestyle than surgery, radiation or traditional focal therapies," said David Jorden, CEO of Nanospectra. "We appreciate the high level of interest and collaboration that we have received from our expert investigators, regulatory authorities and partners that has enabled us to move forward expeditiously with this pivotal study while the final study visits for subjects in the feasibility study continue over the first half of this year."

Dr. Ardeshir Rastinehad, Vice Chair of Lenox Hill Urology and System Director of Prostate Cancer/Northwell Health System and the initial study’s lead principal investigator, will present preliminary feasibility study data at the 12th International Symposium on Focal Therapy and Imaging in Prostate and Kidney Cancer. The conference will be held at the Westin Washington DC City Center on February 9 – 11, 2020.

For additional information on the pivotal study titled "An Extension Study of MRI/US Fusion Imaging and Biopsy in Combination with Nanoparticle Directed Focal Therapy for Ablation of Prostate Tissue" visit www.clinicaltrials.gov referencing NCT04240639.

About AuroLase Therapy
Nanospectra’s AuroLase Therapy utilizes the unique ‘optical tunability’ of a new class of nanoparticles, called AuroShells. The particles are delivered intravenously and accumulate in the tumor. The tumor is illuminated with a near-infrared laser. The particles selectively absorb the photonic laser energy, converting the light into heat, which in turn, destroys the tumor and the blood vessels supplying it; sparing adjacent tissue. AuroLase Therapy is used with an FDA-cleared laser that emits near-infrared energy and an FDA-cleared fiber optic probe for energy delivery percutaneously. AuroShell particles (also known as "nanoshells") consist of a gold metal shell and a non-conducting silica core and serve as the exogenous absorber of the near-infrared laser energy delivered by the probe. Nanospectra’s proprietary technology platform is demonstrated to be safe and effective in initial clinical trials and viable for multiple applications including solid tumors, tissue and drug delivery.

On February 4, 2020 Bio-Techne Corporation (NASDAQ: TECH) reported that its Board of Directors has decided to pay a dividend of $0.32 per share for the quarter ended December 31, 2019 (Press release, Bio-Techne, FEB 4, 2020, View Source [SID1234553805]). The quarterly dividend will be payable February 28, 2020 to all common shareholders of record on February 14, 2020. Future cash dividends will be considered by the Board of Directors on a quarterly basis.

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On February 4, 2020 GlycoMimetics, Inc. (Nasdaq: GLYC) reported several achievements for its GMI-1359 development program, including the issuance of a new patent and key designations granted by the U.S. Food and Drug Administration (FDA) that may provide future development support and marketing protections (Press release, GlycoMimetics, FEB 4, 2020, View Source [SID1234553827]). GMI-1359 is the Company’s novel drug candidate designed to simultaneously inhibit both E-selectin and CXCR4, two adhesion molecules involved in tumor trafficking and metastatic spread. Duke University investigators recently dosed the first patient in a proof-of-concept Phase 1b study to evaluate GMI-1359 drug candidate in patients with advanced breast cancer with bone metastases.

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New Patent

GlycoMimetics today said that the United States Patent and Trademark Office has issued a patent for GMI-1359, covering composition of matter as well as pharmaceutical formulations, and will provide protection through 2035, excluding any patent term adjustments or extensions.

"The new patent for GMI-1359 will help protect the composition of matter of this innovative approach in oncology. GMI-1359 may have a role in treating rare pediatric cancers, such as osteosarcoma, as well as breast cancer and other solid tumors that metastasize to bone," stated Rachel King, Chief Executive Officer of GlycoMimetics. "This intellectual property, as well as that previously granted in Europe, will play a key role as the company advances the drug candidate, especially with the new orphan and rare pediatric disease designations granted by the FDA."

New FDA Designations

In parallel, GlycoMimetics reported that the FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GMI-1359 for the treatment of osteosarcoma, a rare cancer affecting about 900 adolescents a year in the United States. These designations will aid in the development of this drug candidate, including making it eligible for the FDA’s Pediatric Priority Review Voucher.

"It’s encouraging for us as well as for patients and providers that the FDA recognizes the urgent need for new, more effective treatments for this devastating pediatric disease," stated Ms. King.

In addition to its clinical work in breast cancer, GlycoMimetics has conducted preclinical studies that have demonstrated strong support for the potential use of GMI-1359 in osteosarcoma. At the 2018 American Association of Cancer Research Annual Meeting, GlycoMimetics presented data establishing the biologic rationale for the use of a dual e-selectin/CXCR-4 inhibitor in pediatric and young adult patients with osteosarcoma. In that study, GMI-1359 was shown to inhibit tumor progression in an orthopedic model of osteosarcoma as well as inhibit the development of pulmonary metastases from primary osteosarcoma lesions. (View Source)

About Orphan Drug Designation

The FDA Orphan Drug Designation program provides orphan status to drugs and biologics that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Among the benefits of orphan designation in the U.S. are seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received.

About Rare Pediatric Disease Designation

The FDA defines a "rare pediatric disease" as a serious or life-threatening rare disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Under the FDA’sRare Pediatric Disease Priority Review Voucher program, a sponsor who receives an initial approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

About Osteosarcoma

Osteosarcoma is a rare cancer of the bone that usually affects the large bones of the arm or leg, often growing quickly and spreading to other parts of the body. It occurs most often in children and young adults between the ages of 10 and 30. Each year, about 800 to 900 new cases of osteosarcoma are diagnosed in the United States. For more information, please see the osteosarcoma fact sheets at the National Cancer Institute and the American Cancer Society.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as acute myeloid leukemia and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer, as well as in osteosarcoma, a rare pediatric tumor. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers. The newly initiated Phase 1b clinical study in breast cancer patients is designed to enable investigators to identify an effective dose of the drug candidate and to generate initial biomarker data around the drug’s activity.

On February 4, 2020 The French specialty pharmaceutical company, Juvisé Pharmaceuticals is reported its successful syndication of its EUR 213 million debt package supporting the acquisition of Arimidex and Casodex from AstraZeneca, with the collaboration of Société Générale and HSBC, acting as Physical Bookrunners and Mandated Lead Arrangers, BNP Paribas, acting as Mandated Lead Arranger and Lazard acting as financial advisor to Juvisé Pharmaceuticals (Press release, Juvise Pharmaceuticals, FEB 4, 2020, View Source [SID1234553843]).

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On the back of a strong support from both relationship lenders and institutional investors, syndication was wrapped following a successful and oversubscribed early-bird phase. The covenanted financing is comprised of a EUR 128 million TLB alongside a EUR 85 million TLA. This syndication was made possible through the collaboration with Juvisé Pharmaceuticals financial partners, with whom the company relies on long-term partnership characterized by openness and trust.

This successful syndication is another important step in Juvisé Pharmaceuticals development. It reasserts the company ability to leverage all debt capital instruments to fulfil its growth ambition, while keeping the flexibility and independence in decision making allowed by its privately held structure.

Frédéric Mascha, Founder and President of Juvisé Pharmaceuticals, declared, "We are very pleased that our acquisition debt package was successfully syndicated in this oversubscribed early-bird phase and we would like to thank our financial partners for the trust they have shown in our project. We are proud to see that our commitment towards physicians, patients, and our Pharma partners convinced renowned financial institutions to support our ambitious development plan."

About Arimidex and Casodex
Arimidex (anastrozole) and Casodex (bicalutamide) are hormone treatments for breast and prostate cancer. The two products are recognized as pillar in those cancer treatments and essential for both patients and physicians.

On 19th December 2019, Juvisé Pharmaceuticals acquired the rights of Arimidex and Casodex in 45 countries in Europe and a number of Middle Eastern and African markets.