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 PharmaCyte Biotech (OTCQB: PMCB) is reported to embark upon a planned U.S. FDA Phase 2b clinical trial to treat locally advanced, inoperable pancreatic cancer (LAPC) at trial sites all over the United States, and with that journey comes the need to keep its signature technology, Cell-in-a-Box, protected should it one day receive marketing approval from drug regulatory agencies in the U.S. and Europe (Press release, PharmaCyte Biotech, FEB 4, 2020, View Source [SID1234553841]). PharmaCyte’s Cell-in-a-Box technology for the treatment of pancreatic cancer is certainly protected well into the future if/when it does receive the coveted marketing approval it’s striving for upon the completion of clinical trials.

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Currently, PharmaCyte is revamping its provisional patent application and its strategy related to full patent protection. The idea is to present the United States Patent and Trademark Office (USPTO) with an acceptable provisional patent application that protects its therapy to treat cancerous tumors, including the therapy that will be used in its upcoming clinical trial in LAPC.

Provisional patent applications are a way to establish and protect a "date of invention" or "priority filing date" for one year. The provisional patent application was created to provide inventors with a way to begin protecting their inventions, and an approved provisional patent application will provide PharmaCyte 12 months to prepare a full patent application. This approach will offer the company an opportunity to establish an early effective filing date for a patent.

A provisional patent—and eventually a full patent—would give PharmaCyte a fresh 20 years of patent protection; however, even without it, PharmaCyte has protections in place to protect its technology that are in line with any biotechnology/pharmaceutical company developing treatments after many years. In most cases, the 20-year patent protection that biotechnology/pharmaceutical companies have in place while developing drugs/treatments, have their 20-year time frame cut in half to 10 years by the time the drug hits the marketplace.

Patents are typically awarded within a few years after the patent application submission, but a common misconception is that the patent begins only after the drug hits the market, so with this in mind, PharmaCyte will actually have longer protections in place than most biotechnology/pharmaceutical companies have upon marketing approval after years of development.

How is this possible? Well, actually PharmaCyte has two protections in place that will keep its technology protected—and unlike 20-year patent protection where the clock is ticking throughout development, these protections don’t begin until the therapy is approved. First, PharmaCyte is developing a "biologic" therapy, which offers protections under the Affordable Care Act, and second, PharmaCyte’s therapy for pancreatic cancer was granted Orphan Drug Designation from both the U.S. FDA and in the European Union by the European Medicines Agency (EMA).

PharmaCyte’s pancreatic cancer therapy was designated an orphan drug and listed in the official registry of medicinal products for rare diseases by the U.S. FDA on December 17, 2014. This orphan drug status assures marketing exclusivity for PharmaCyte’s pancreatic cancer therapy in the U.S. for 7 years after market approval by the FDA. Also, PharmaCyte has orphan drug status in the European Union (EU) for its pancreatic cancer therapy. This designation provides 10 years of marketing exclusivity in all countries in the EU following approval by the European Medicines Agency (EMA).

In addition, the Biologics Price Competition and Innovation Act (BPCIA), which was enacted as part of the Affordable Care Act in 2010, establishes a period of 12 years of "data exclusivity" for reference products to preserve incentives for future innovation. Under this framework, data exclusivity protects the data in the innovator’s regulatory application by prohibiting others, for a period of 12 years, from gaining FDA approval based in part on reliance on or reference to the innovator’s data in a biosimilar application. PharmaCyte’s 12-year exclusivity doesn’t begin until the FDA approves the company’s pancreatic cancer therapy.

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said of the protections for the company’s pancreatic cancer therapy, "If our pancreatic cancer therapy receives FDA approval, the orphan drug designation in the U.S. and the EU, together with the BPCIA data exclusivity, will give us substantial marketing exclusivity for our pancreatic cancer therapy. Any new patent application, while it does include our pancreatic cancer therapy, should really be viewed as an opportunity to dramatically broaden PharmaCyte’s ability to protect our therapy for all malignant tumors for the next 20 years."

On February 4, 2020 Celsion Corporation (NASDAQ: CLSN), an oncology drug-development company, reported Prof. Riccardo Lencioni, M.D., FSIR, EBIR delivered a presentation titled "Thermally-Sensitive Ablation Enhancers: Where Do We Stand?" at the SPECTRUM 2020 Interventional Oncology Conference held in Miami , FLA last month (Press release, Celsion, FEB 4, 2020, View Source [SID1234553840]). Dr. Lencioni is a professor in the Department of Radiology at the University of Pisa School of Medicine in Italy and is an Honorary Research Professor of Interventional Oncology at the Miami Cancer Institute. He was the lead principal investigator in Europe for the Company’s completed Phase III HEAT Study in hepatocellular carcinoma (HCC), or primary liver cancer, using radiofrequency ablation (RFA) plus ThermoDox, the Company’s lead product.

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The SPECTRUM Conference is the only interventional oncology conference endorsed by The American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). In his talk, Prof. Lencioni focused on the HEAT Study subgroup analysis showing the duration of RFA heating time per tumor volume of 45 minutes or longer plus ThermoDox was key to overall survival benefit in this patient population. He noted that in early-stage HCC, nearly 50% of patients with a solitary lesion of less than 5 cm on imaging have microsatellites on histology. While RFA and other energy sources are not able to treat these microsatellites, a thermosensitive drug carrier such as ThermoDox would deposit increased amounts of doxorubicin in the margins of a tumor given increased ablation time.

"The significant attention ThermoDox is receiving among key opinion leaders at important medical conferences has helped build awareness of our drug in combination with RFA for treating HCC," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "We are grateful to Prof. Lencioni for helping Celsion create the training video on RFA heating time for lesion sizes from 3 cm to 7 cm that is being used by all investigators in our current Phase III OPTIMA Study."

The Company’s OPTIMA Study was based on the prospective analysis of the HEAT Study subgroup of patients who received 45 minutes or more of RFA energy. The OPTIMA Study was fully enrolled in August 2018 with 556 subjects from 65 clinical sites in 14 countries. At its first interim analysis in November 2019 following 128 patient events, or deaths, the independent Data Monitoring Committee (iDMC) unanimously recommended the OPTIMA Study continue according to protocol based on safety and data integrity.

The Company re-affirms its projection that its second pre-planned interim efficacy analysis will occur during the second quarter of 2020, following 158 patient events, or deaths. The hazard ratio for success at 158 events is 0.70, which is below the hazard ratio of 0.65 observed for the 285 patients in the HEAT Study subgroup of patients treated with RFA of 45 minutes or longer.

Mr. Tardugno added, "We were pleased that the HCC part of the SPECTRUM program featured invited faculty with ties to Celsion, such as Dr. Josep Llovet, Founder and Director of the Liver Cancer Program and Full Professor of Medicine at the Mount Sinai School of Medicine in New York, who is chair of the OPTIMA iDMC, and Dr. Ghassan Abou-Alfa of Memorial Sloan Kettering Cancer Center and Professor, Weill Medical College at Cornell University, both in New York City, who was a member of the HEAT Study DMC. The medical community’s independent assessment, along with the National Institutes of Health’s published confirmation of the hypothesis supporting the potential for ThermoDox as a curative treatment for the largest unmet need in oncology is gratifying indeed."

On February 4, 2020 Horizon Therapeutics plc (Nasdaq: HZNP) reported that its fourth-quarter and full-year 2019 financial results will be released on Wednesday, Feb. 26, 2020 (Press release, Horizon Therapeutics, FEB 4, 2020, Horizon Therapeutics plc to Release Fourth-Quarter and Full-Year 2019 Financial Results and Host Webcast on Feb. 26, 2020 [SID1234553838]). Following the announcement, Horizon’s management will host a live webcast at 8 a.m. Eastern Time to review the Company’s financial and operating results.

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The live webcast and a replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay of the webcast will be available approximately two hours after the live webcast.

On February 4, 2020 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need, reported that Steven A. Shallcross, Chief Executive and Financial Officer, is scheduled to present at the BIO CEO & Investor Conference on Monday, February 10, 2020, at 11:00 a.m. (ET) at the New York Marriott Marquis (Wilder room) (Press release, Synthetic Biologics, FEB 4, 2020, View Source [SID1234553837]).

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A live webcast of Synthetic Biologics’ presentation may be accessed by logging onto the internet at http://www.veracast.com/webcasts/bio/ceoinvestor2020/17107426667.cfm. A replay will be archived and accessible for 90 days at the same website and at www.syntheticbiologics.com.