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Ipsen presents new analyses utilizing modern data mining approaches at ISPOR Europe 2019

On October 31, 2019 Ipsen (Euronext: IPN; ADR: IPSEY) reported that results from a network meta-analysis (NMA) in advanced renal cell carcinoma (aRCC), and a UK-focused budget impact study assessing long-acting somatostatin analogues (LA-SSAs) for the treatment of acromegaly and gastroenteropancreatic neuroendocrine tumors (GEP NET) will be presented at the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) Europe 2019 Annual Conference (Press release, Ipsen, OCT 31, 2019, View Source [SID1234550095]). ISPOR takes place in Copenhagen, Denmark from 2 – 6 November 2019.

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Key studies to be presented at ISPOR Europe 2019:

An assessment of the budget impact of LA-SSAs in the treatment of acromegaly and GEP NET, considering attributes related to the drug delivery of LA-SSAs in the UK.
An NMA analyzing cabozantinib versus standard-of-care comparators in progression free survival (PFS) and overall survival (OS) in the first-line treatment of advanced renal cell carcinoma.

"We’re excited to be sharing two interesting sets of results – namely a comparison of first-line therapies and budget impact of a treatment approach for acromegaly and gastroenteropancreatic neuroendocrine tumors," said Ulf Staginnus, Senior Vice President, Head Global Market Access and Pricing, Ipsen. "With more than 5,000 global healthcare leaders, seeking robust health solutions and new insights, ISPOR Europe 2019 is the perfect stage to share these results."

The delivery attributes of both LA-SSAs were considered in the UK-focused study assessing the budget impact of the LA-SSAs, lanreotide versus octreotide in the treatment of acromegaly and GEP-NET. Model inputs (including drug acquisition and administration costs) were based on publicly available sources. The analysis compared the current and hypothetical market share scenarios from three perspectives in the UK: the National Health Service (NHS), a regional clinical commissioning group (CCG), and a local institution (hospital). Results suggested that increasing the use of lanreotide to a hypothetical 80% market share for lanreotide in the UK would reduce overall LA-SSAs patient treatment expenses by £2.9 million annually in the UK (a reduction of 3.6% from the current budget estimate of £80.6 million).

In the area of treatment provision in aRCC, Ipsen used an NMA to respond to the challenge presented to healthcare professionals by the introduction of targeted therapies in the last year with no way to objectively compare them. While randomized trials are the gold standard for comparative effectiveness research, they are not always available for clinically and economically important treatment comparisons. In this case, the NMA may offer some helpful insights as it suggests that cabozantinib significantly increases progression free survival (PFS) in intermediate and poor-risk patients when compared with standards-of-care and concludes that cabozantinib may be considered as an efficient treatment option in first-line aRCC.

"Modern quantitative data reviews of available agents offer additional insights into existing healthcare," said Bartek Bednarz, Senior Vice-President, Global Product and Portfolio Strategy, Ipsen. "The network meta-analysis (NMA) for cabozantinib and budget impact model for somatostatin analogues shared at ISPOR Europe 2019 are just part of Ipsen’s ongoing commitment to demonstrating benefit for payers and improving options for patients with high unmet needs."

Follow Ipsen on Twitter via @IpsenGroup and keep up to date with ISPOR Europe 2019 Conference news and updates by using the hashtag #ISPOREurope.

Overview of key Ipsen presentations at ISPOR Europe 2019:

Medicine Abstract title Abstract number/timing (CEST)
Cabometyx

(cabozantinib)

Cabozantinib versus standard-of-care comparators: a network meta-analysis of progression free survival and overall survival in the first-line treatment of advanced renal cell carcinoma
PCN42; Board D5

RESEARCH POSTER SESSION 2

CANCER

Monday, November 4, 2019

Display Hours: 15:30 – 19:00

Somatuline Autogel (lanreotide autogel/depot) Budget impact analysis of somatostatin analogues in the treatment of GEP-NET and acromegaly in the UK
PDG23; Board J3

RESEARCH POSTER SESSION 4

DRUGS & GENERICS

Tuesday, November 5, 2019

Display Hours: 15:45 – 19:00

N/A Retrospective Gesetzliche Krankenversicherung (statutory health insurance) (GKV) research study on the initial treatment of bladder carcinoma (BCA) by transurethral bladder resection (TURB) – a comparative analysis of costs and urological follow-up therapies using standard white light- (WL-) vs. Blue light- (WL-)TURB
PCN502; Board W12

RESEARCH POSTER SESSION 2

CANCER

Monday, November 4, 2019

Display Hours: 15:30 – 19:00

N/A Assessing the human and economic burden of short stature: a systematic literature review
PMU142; Board V19

RESEARCH POSTER SESSION 4

MULTIPLE DISEASES

Tuesday, November 5, 2019

Display Hours: 15:45 – 19:00

New England Journal of Medicine Publishes Results from Astellas’ Phase 3 ADMIRAL Trial of XOSPATA® (gilteritinib) in Adult Patients with FLT3 Mutation-Positive Relapsed/Refractory Acute Myeloid Leukemia1

On October 31, 2019 The New England Journal of Medicine published detailed results from the Phase 3 ADMIRAL trial, which found that Astellas Pharma Inc.’s (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D. "Astellas") XOSPATA (gilteritinib) demonstrated significantly longer Overall Survival (OS) than salvage chemotherapy in adult patients with relapsed (disease that has returned) or refractory (resistant to treatment) Acute Myeloid Leukemia (AML) with a FMS-like tyrosine kinase 3 (FLT3) mutation (Press release, Astellas, OCT 31, 2019, View Source [SID1234550086]). Common adverse events from the study of grade 3 or higher in the gilteritinib group were febrile neutropenia (45.9%), anemia (40.7%), and thrombocytopenia (22.8%).1 The results appear in the October 31 print edition of the Journal and are currently available online.1

"These data show that in a very high-risk leukemia population, single agent FLT3-targeted therapy leads to superior clinical outcomes compared to salvage chemotherapy," said Alexander Perl, MD, associate professor of Hematology-Oncology in the Abramson Cancer Center at the University of Pennsylvania, and the trial’s principal investigator. "This is an important result for patients who previously had quite limited treatment options."

"The detailed findings from our Phase 3 ADMIRAL trial further validate that XOSPATA is a significant addition to the treatments available for people with FLT3 mutation positive relapsed or refractory AML," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head at Astellas. "We are especially pleased to see the results published in the New England Journal of Medicine, which is widely regarded as one of the most prestigious scientific medical journals."

The U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) in May of 2019 to update the U.S. product labeling for XOSPATA to include final analysis OS data from the ADMIRAL trial.2

XOSPATA was discovered through a research collaboration with Kotobuki Pharmaceutical Co., Ltd., and Astellas has exclusive global rights to develop, manufacture and commercialize XOSPATA.

XOSPATA was approved by the Japan Ministry of Health, Labor and Welfare (MHLW) for relapsed or refractory AML with FLT3 mutations and launched as XOSPATA 40 mg Tablets in 2018.3 In October 2019, the European Commission (EC) approved XOSPATA as a monotherapy for the treatment of adult patients with relapsed or refractory AML with a FLT3 mutation.4 XOSPATA has been designated an orphan medicinal product and also received accelerated assessment from the European Medicines Agency (EMA) earlier this year, which reduced the timeframe for approval.5,6,7 Astellas is currently investigating gilteritinib in various FLT3 mutation-positive AML patient populations through several clinical trials. Visit View Source to learn more about ongoing gilteritinib clinical trials.

Takeda Reports Solid Second Quarter FY2019 Results and Raises Profit Guidance for the Full Year

On October 31, 2019 Takeda Pharmaceutical Company Limited (TOKYO:4502)(NYSE:TAK) (Press release, Takeda, OCT 31, 2019, View Source [SID1234550063]):

Underlying Revenue declined -0.2% vs FY2018 H1 pro-forma revenue2

Takeda’s 14 global brands with reported revenue of 547.0 billion yen in aggregate posted a strong year-over-year underlying growth of +21%, driven by ENTYVIO growing +33.9%, ALBUMIN/FLEXBUMIN growing +16.9%, and NINLARO growing +32.7%, offset by the negative impact of intensified competition and generic erosion.
Underlying growth in key business areas for FY2019 H1: GI (+9%), Oncology (+11%), Neuroscience (+6%), and Plasma Derived Therapy (PDT) Immunology (+4%), offset by Rare Disease (-11%) due to decline as expected in Rare Hematology.
Within Rare Disease, Rare Hematology continues to be impacted by competition and price pressure, and growth in Hereditary Angioedema (HAE) was negatively affected by stocking in the prior year as well as generic entry for FIRAZYR.
Immunoglobulin returned to growth as expected in the 3 months of FY2019 Q2 at an underlying growth rate of +7.6% and +3% for H1, over the same period of last year.
Underlying Core Operating Profit Margin of 32.2% for FY2019 H1 with cost synergies and OPEX efficiencies driving margins

Reported Operating Profit declined -70.7% to 50.3 billion yen, largely impacted by non-cash purchase accounting expenses including unwinding of inventory step-up, increased amortization of intangibles, as well as one-time integration costs.
Core Operating Profit increased +155.5% to 541.6 billion yen primarily due to the acquisition of Shire, solid performance of 14 global growth brands and improved operating efficiency partially offset by the negative impact of intensified competition and generic erosion.
Underlying Core Operating Profit Margin for FY2019 H1 was 32.2% reflecting continued OPEX discipline and cost synergies.
Underlying Core EPS for H1 was 249 yen.
Investing in future growth by adding 23 plasma collection centers since the closing of the Shire acquisition, acquiring license for first-in-class celiac disease therapy (TAK-101) from COUR Pharmaceuticals, and continued investment in our R&D engine.
1

Please refer to note iii in Reported Results for H1 (April – June) FY2019 table for Core Operating Profit definition.

Growth versus FY2018 H1 pro-forma revenue (6-month April-September 2018 combined revenue of Legacy Takeda and Legacy Shire, excludes its oncology business and US GAAP results were conformed to IFRS, without material differences). Please see the appendix for more details.

Achieved Several Important Pipeline Milestones

Data presented at World Sleep Congress demonstrated early evidence of efficacy for TAK-925 in Narcolepsy Type 1.
ENTYVIO head-to-head study and TAK-620 (Maribavir) Ph-2 data published in The New England Journal of Medicine.
TRINTELLIX approved in Japan for the treatment of depression and depressed state.
Submitted a Marketing Authorization Application (MAA) in Japan for a subcutaneous formulation of ENTYVIO for patients with moderately to severely active ulcerative colitis.
8 potential best-in-class or first-in-class New Molecular Entities (NMEs) in pivotal studies.
Divesting non-core assets to accelerate deleveraging and focus the business

Paid down 584.5 billion yen of debt and de-levered from 4.7x at end of FY2018 to 3.9x Net debt / adjusted EBITDA as of September 2019. This does not include the sale of OTC and prescription pharmaceutical assets in certain Near East, Middle East and Africa countries to Acino for more than $200 million.
Negotiations ongoing for further potential divestments.
Christophe Weber, Chief Executive Officer, commented:

"We are pleased with our recent financial results, which reflect strong underlying performance across our 14 global brands and OPEX improvements that has allowed us to raise our guidance. I am particularly encouraged by the rapid de-leveraging year-to-date.
We are also satisfied with the progress of our integration efforts. Takeda employees are excited and engaged around our next phase of growth and our undeniable progress towards becoming one integrated company.
Our excitement around the future of Takeda is also derived from the strength and breadth of our pipeline. We look forward to providing some additional insight and commentary around our portfolio, pipeline, and growth strategy at our upcoming R&D Day in November."

Reported Results for FY2019 H1 (April – June)

(billion yen)

REPORTED

CORE

UNDERLYING i.

FY2019 H1

VS. PRIOR YEAR

FY2019 H1

VS. PRIOR YEAR

Revenue

1,660.2

+88.5%

1,660.2

+88.5%

-0.2% y-o-y ii.

(pro-forma)

Operating Profit

50.3

-70.7%

541.6 iii.

+155.5%

Margin

3.0%

-16.5pp

32.6%

+8.6pp

32.2%

Net Profit iv.

33.2

-73.8%

380.4

+130.3%

EPS (JPY)

21 yen

-140 yen

244 yen

+33 yen

249 yen

Underlying Growth 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.

ii.

Growth versus FY2018 H1 pro-forma revenue. Pro-forma revenue is the 6-month April-September 2018 combined revenue of Legacy Takeda and Legacy Shire, US GAAP results conformed to IFRS, without material differences. The adjustments also include removal of impacts related to Shire’s oncology business which was divested in August 2018.

iii.

Core Operating Profit represents net profit adjusted to exclude income tax expenses, our 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 intangible assets associated with products and other items that management believes are unrelated to our core operations, such as purchase accounting effects and transaction related costs.

iv.

Attributable to the owners of the company.

FY2019 Management Guidance: Upgrading guidance to reflect positive business momentum

Previous Guidance
(July 31, 2019)

Revised Guidance
(October 31, 2019)

Underlying Revenue Growth i.

Flat to slightly increasing

Underlying Core Operating Profit Margin

Mid-to-high-twenties %

High-twenties %

Underlying Core EPS

360 – 380 yen

370 – 390 yen

Annual Dividend per Share

180 yen

i.
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 conformed from US GAAP to IFRS, without material differences.

FY2019 Reported Forecast: Revenue decreasing due to FX impact, but profit increasing

For more details on Takeda’s FY2019 Q2 results and other financial information, please visit View Source

Takeda to Host R&D Day on November 14 in New York, Plasma-Derived Therapies Day on November 15 in Covington, GA, and R&D and Plasma-Derived Therapies Day on November 21 in Tokyo;

At each event, Takeda executives are expected to discuss, among other things, the Company’s near-term and sustained growth strategies and next-generation platforms. Each event will be accessible via a live webcast on the Investors section of the Company’s website: View Sourcequarterly-announcements/quarterly-announcements-2019/.
A replay of each webcast will be archived on the website along with the presentation slides associated with each event.

Presenting data at the cancer immunotherapy SITC conference

On October 30, 2019 MonTa Biosciences reported that is attending the cancer immunotherapy SITC (Free SITC Whitepaper) conference in Washington DC to present data on our lead candidate MBS8 (Press release, MonTa Biosciences, OCT 30, 2019, View Source [SID1234618628]).

Athenex Announces European Commission Grants Orphan Designations for Paclitaxel and Encequidar for the Treatment of Soft Tissue Sarcoma

On October 30, 2019 Athenex, Inc. (Nasdaq: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported that it has received Orphan Designations from the European Commission (EC) for paclitaxel and encequidar ("Oral Paclitaxel") for the treatment of soft tissue sarcoma, following a positive opinion from the European Medicines Agency (EMA) (Press release, Athenex, OCT 30, 2019, View Source [SID1234573893]).

Dr. Rudolf Kwan, Chief Medical Officer of Athenex, commented, "We are very pleased to receive Orphan Designations from the European Commission, as this further recognizes the potential benefits of Oral Paclitaxel. Earlier this year, we shared encouraging early results from a clinical study in angiosarcoma, a form of soft tissue sarcoma, that showed rapid responses in a difficult to treat patient population. We will soon be extending this study to include sites in Europe."

The EC grants Orphan Designation status to support development of medicines for the treatment of diseases that affect fewer than 5 in 10,000 people in the European Union and provide a significant benefit to those affected by the condition. Orphan Designation may provide certain benefits, including protocol assistance, fee reductions, and a ten-year period of market exclusivity if the drug is approved.

The U.S. FDA has granted oral paclitaxel and encequidar an Orphan Drug Designation in the treatment of angiosarcoma.

The Orascovery platform was initially developed by Hanmi Pharmaceuticals and licensed exclusively to Athenex for all major worldwide territories except Korea, which is retained by Hanmi.