Global Pharyngeal Cancer Therapeutics Market 2019-2023 | Emergence of Pipeline Molecules to Boost Growth | Technavio

LONDON–(BUSINESS WIRE)–#Biotechnology–The global pharyngeal cancer therapeutics market is expected to post a CAGR of over 6% during the period 2019-2023, according to the latest market research report by Technavio. Request a free sample report


Pharyngeal cancer is one of the most common types of cancer as it largely attributed to the prevalence of high-risk factors such as increase in the use of tobacco, excessive alcohol consumption, increase in viral infections, and occupational exposure. Viral infections associated with the throat can lead to the development of throat cancer, including pharyngeal cancer. Moreover, individuals working in high-dose radiation environments and wood, metal, leather, textile, and petroleum industries can also develop pharyngeal cancer as they are exposed to certain chemicals, dust, or fumes. Thus, such high-risk factors are expected to fuel the growth of the market during the forecast period.

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As per Technavio, the emergence of pipeline molecules, will have a positive impact on the market and contribute to its growth significantly over the forecast period. This research report also analyzes other important trends and market drivers that will affect market growth over 2019-2023.

Global Pharyngeal Cancer Therapeutics Market: Emergence of pipeline molecules

Despite the high prevalence of pharyngeal cancer, there is a lack of approved targeted therapy drugs for treatment. This is encouraging vendors to invest significantly in the development of novel therapeutics with limited side effects. For instance, Roche is developing TECENTRIQ, which is a novel PD-L1 monoclonal antibody to prevent cancer immune evasion. This molecule is being investigated in Phase lll trials in patients with adjuvant SCCHN. The filing of this molecule is expected in 2022, and it is expected to receive marketing approval by the end of the forecast period. Thus, the expected approvals of the late-stage pipeline molecules are anticipated to propel the growth of the market during the forecast period.

“Apart from the high-risk factors for pharyngeal cancer, other factors such as the presence of reimbursement and patient assistance programs, along with the advent of novel therapies are expected to boost the market growth during the forecast period,” says a senior analyst at Technavio.

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Global Pharyngeal Cancer Therapeutics Market: Segmentation Analysis

This market research report segments the global pharyngeal cancer therapeutics market by product (targeted therapy and chemotherapy) and geographical regions (North America, Europe, Asia, and ROW).

The North American region led the market in 2018, followed by Europe, Asia, and ROW respectively. During the forecast period, the North American region is expected to maintain its dominance and register the highest incremental growth due to the high prevalence of the disease and recent product approvals in the region.

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Some of the key topics covered in the report include:

Market Landscape

  • Market ecosystem
  • Market characteristics
  • Market segmentation analysis

Market Sizing

  • Market definition
  • Market size and forecast

Five Forces Analysis

Market Segmentation

Geographical Segmentation

  • Regional comparison
  • Key leading countries

Market Drivers

Market Challenges

Market Trends

Vendor Landscape

  • Vendors covered
  • Vendor classification
  • Market positioning of vendors
  • Competitive scenario

About Technavio

Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions.

With over 500 specialized analysts, Technavio’s report library consists of more than 10,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio’s comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

If you are interested in more information, please contact our media team at media@technavio.com.

Contacts

Technavio Research

Jesse Maida

Media & Marketing Executive

US: +1 844 364 1100

UK: +44 203 893 3200

www.technavio.com

Stanford Medicine Recognized by Vizient as a Top Performer in Quality and Safety for 2019

STANFORD, Calif.–(BUSINESS WIRE)–Stanford Medicine has been ranked in the top ten percent among peer academic medical centers (AMCs) for both inpatient and outpatient care by Vizient, Inc., the nation’s leading health care performance improvement company that measures overall safety and quality in patient care.

In recognition of these achievements in care quality and safety, Vizient presented Stanford Medicine with two awards that were jointly accepted by David Entwistle, president and CEO of Stanford Health Care and Lloyd Minor, M.D., dean of the Stanford University School of Medicine, on September 19 during the 2019 Vizient Connections Education Summit.

“This tremendous achievement is a tribute to the hard work and dedication of our staff and faculty at Stanford Health Care and the School of Medicine,” said Entwistle. “I want to thank each and every one of our colleagues who have prioritized our commitment to continuously improving patient care. We could not have done this without you. I look forward to continuing our important work to further enhance safety and quality as we move into our new, state-of-the-art hospital where we will be able to bring the latest biomedical advances to patients.”

“Our rankings in the top ten percent for both inpatient and ambulatory care are a direct result of the collaboration between staff and faculty at Stanford Health Care and the School of Medicine that began years ago when we aligned our priorities and focused on strengthening the safety and quality of care,” said Minor. “Working together, we have been able to improve faster than we ever imagined possible.”

As a top performer in the 2019 Bernard A. Birnbaum, MD, Quality Leadership Award category, Stanford Medicine was one of 11 Vizient members in the AMC cohort that was recognized for demonstrating excellence in delivering high-quality care based on the Vizient Quality and Accountability Ranking. Stanford Medicine also received the 2019 Ambulatory Care Quality and Accountability Award, which recognized Stanford Medicine as a faculty practice that demonstrates excellence in delivering high-quality outpatient care.

“As a world-class hospital and research center, Stanford Medicine uniquely understands the science of safety and quality as well as the importance of culture,” said Karen Frush, chief quality officer at Stanford Health Care. “The recognition by Vizient is additional proof that the systems we use to continually assess and improve the care we provide actually work.”

More about the 2019 Bernard A. Birnbaum, MD, Quality Leadership Award

The Bernard A. Birnbaum, MD, Quality Leadership Award recognizes Stanford Medicine’s performance-driven culture for achieving the highest quality and patient-centered outcomes. This year, 349 participating hospitals were segmented into four cohorts for the Vizient Quality and Accountability Ranking. The ranking measured performance based on safety, mortality, clinical effectiveness, efficiency and patient centeredness. The ranking’s composite scoring system uses patient-level performance data from a variety of sources including the Vizient Clinical Data Base, core measures data, the Hospital Consumer Assessment of Healthcare Providers and Systems (HCAHPS) survey and the Centers for Disease Control and Prevention’s National Healthcare Safety Network (NHSN).

The Vizient Quality and Accountability Ranking helps participating hospitals and health systems understand their performance against their peers, and identifies structures and processes associated with high performance in quality and safety across a broad spectrum of patient care activity. The recognition period is for work spanning July 2018 through June 2019.

More about the 2019 Ambulatory Care Quality and Accountability Award

Ambulatory Care Quality and Accountability Award recipients are determined through the Vizient Ambulatory Care Quality and Accountability Study, which assesses data from participating academic faculty practices to measure performance in five domains: access to care, quality, efficiency, continuum of care and equity. The composite scoring system utilizes practice and patient-level data across several databases including the Vizient-AAMC Clinical Practice Solutions Center. The recognition period is for work spanning July 2018 through June 2019.

“As the industry continues to evolve, payment and care delivery models will also change, making it ever more important for faculty practices to leverage data to create a quality-driven organization,” said David Levine, group senior vice president, advanced analytics and product management for Vizient. “We celebrate Stanford Medicine for its ability to deliver ambulatory care that focuses on patient quality, while at the same time improving cost and accessibility to benefit not only the patients, but also the community it serves.”

About Stanford Health Care

Stanford Health Care seeks to heal humanity through science and compassion, one patient at a time, through its commitment to care, education, and discovery. Stanford Health Care delivers clinical innovation across its inpatient services, specialty health centers, physician offices, virtual care offerings and health plan programs.

Stanford Health Care is part of Stanford Medicine, a leading academic health system that includes Lucile Packard Children’s Hospital Stanford. Stanford Medicine is renowned for breakthroughs in treating cancer, heart disease, brain disorders, and surgical and medical conditions.

Contacts

Julie Greicius

650-723-4598

jgreicius@stanford.edu

Verastem Oncology Announces COPIKTRA™ (Duvelisib) Presentations at the 18th Annual International Workshop on Chronic Lymphocytic Leukemia

BOSTON–(BUSINESS WIRE)–Verastem, Inc. (Nasdaq:VSTM) (Verastem Oncology or the Company), a biopharmaceutical company focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients, today announced that three posters highlighting clinical data for COPIKTRA™ (duvelisib) will be presented at the 18th Annual International Workshop on Chronic Lymphocytic Leukemia taking place September 20-23, 2019, in Edinburgh, UK. The presented abstracts focus on clinical data from the Phase 3 DUO study including evaluation of COPIKTRA efficacy and safety in heavily pre-treated patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), dose modification data and results from a post-hoc analysis evaluating the effect of COPIKTRA on lymphocytosis in patients, including those with high-risk factors.

Many patients with CLL or SLL eventually relapse and are in need of additional therapy options to help control their disease,” said Marco Montillo, M.D., Head, Department of Hematology and Oncology, Niguarda Cancer Center, Niguarda Hospital, Milan, Italy. “In the DUO study, duvelisib monotherapy demonstrated significantly improved clinical outcomes in patients with relapsed refractory disease who had received two or more prior therapies. In the study, the safety profile of duvelisib was generally manageable through dose modifications or interruptions.”

The data presented at iwCLL this year highlight findings from the Phase 3 DUO study in the labeled indication of relapsed or refractory CLL/SLL after at least 2 prior therapies,” commented Hagop Youssoufian, MSc, M.D., Head of Medical Strategy at Verastem Oncology. “Of note, response rates appeared to be preserved in COPIKTRA-treated patients whose adverse events, occurring on therapy, were effectively managed through dose interruptions or dose reductions. These findings from the study are an important factor in helping physicians navigate the management of adverse events in the clinical setting with the goal of not compromising efficacy.”

COPIKTRA, a targeted oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, received approval as monotherapy from the U.S. Food and Drug Administration (FDA) in September 2018 for the treatment of patients with relapsed or refractory CLL/SLL after at least two prior therapies. COPIKTRA also received accelerated approval for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. Continued approval in FL may be contingent upon verification and description of clinical benefit in confirmatory trials.

Details for the iwCLL 2019 poster presentations are as follows:

 

Title: An improved benefit-risk profile of duvelisib in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma who received ≥2 prior therapies

Lead author: Marco Montillo, Niguarda Cancer Center

Presentation ID: 1914

Date and time: Sunday, September 22, 2019; all day

 

Title: Effect of dose modifications on response to duvelisib in patients with relapsed or refractory CLL/SLL in the DUO trial

Lead author: Paolo Ghia, Università Vita-Salute San Raffaele and IRCCS Ospedale San Raffaele

Presentation ID: 2034

Date and time: Sunday, September 22, 2019; all day

 

Title: Patterns of duvelisib-induced lymphocytosis in patients with relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma, including those with high-risk factors treated in the DUO trial

Lead author: Jacqueline Barrientos, Zucker School of Medicine at Hofstra/Northwell

Presentation ID: 2033

Date and time: Sunday, September 22, 2019; all day

PDF copies of these poster presentations will be available here after the meeting.

SELECT IMPORTANT SAFETY INFORMATION

This does not include all information needed to use COPIKTRA™ (duvelisib) safely and effectively. See full Prescribing Information.

WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR COLITIS, CUTANEOUS REACTIONS, and PNEUMONITIS

See full Prescribing Information for complete boxed warning

  • Fatal and/or serious infections occurred in 31% of COPIKTRA-treated patients. Monitor for signs and symptoms of infection. Withhold COPIKTRA if infection is suspected.
  • Fatal and/or serious diarrhea or colitis occurred in 18% of COPIKTRA-treated patients. Monitor for the development of severe diarrhea or colitis. Withhold COPIKTRA.
  • Fatal and/or serious cutaneous reactions occurred in 5% of COPIKTRA-treated patients. Withhold COPIKTRA.
  • Fatal and/or serious pneumonitis occurred in 5% of COPIKTRA-treated patients. Monitor for pulmonary symptoms and interstitial infiltrates. Withhold COPIKTRA.

WARNINGS AND PRECAUTIONS

  • Hepatotoxicity: Monitor hepatic function.
  • Neutropenia: Monitor blood counts.
  • Embryo-Fetal toxicity: COPIKTRA can cause fetal harm. Advise patients of potential risk to a fetus and to use effective contraception.

ADVERSE REACTIONS

The most common adverse reactions (≥20%) are diarrhea or colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory infection, pneumonia, musculoskeletal pain, and anemia.

To report Adverse Reactions, contact FDA at 1-800-FDA-1088 (1-800-332-1088) or www.fda.gov/medwatch and Verastem Oncology at 1-877-7RXVSTM (1-877-779-8786).

DRUG INTERACTIONS

  • CYP3A inducers: Avoid co-administration with strong CYP3A inducers.
  • CYP3A inhibitors: Monitor for COPIKTRA toxicities when co-administered with strong or moderate CYP3A inhibitors. Reduce COPIKTRA dose to 15 mg twice daily when co-administered with strong CYP3A4 inhibitors.
  • CYP3A substrates: Monitor for signs of toxicities when co-administering COPIKTRA with sensitive CYP3A substrates.

USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed.

Please see accompanying full Prescribing Information, including Boxed Warning.

About Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are cancers that affect lymphocytes and are essentially the same disease, with the only difference being the location where the cancer primarily occurs. When most of the cancer cells are located in the bloodstream and the bone marrow, the disease is referred to as CLL, although the lymph nodes and spleen are often involved. When the cancer cells are located mostly in the lymph nodes, the disease is called SLL. The symptoms of CLL/SLL include a tender, swollen abdomen and feeling full even after eating only a small amount. Other symptoms can include fatigue, shortness of breath, anemia, bruising easily, night sweats, weight loss, and frequent infections. However, many patients with CLL/SLL will live for years without symptoms. There are approximately 200,000 patients in the US affected by CLL/SLL with nearly 20,000 new diagnoses this year alone. While there are therapies currently available, real-world data reveals that a significant number of patients either relapse following treatment, become refractory to current agents, or are unable to tolerate treatment, representing a significant medical need. The potential of additional oral agents, particularly as a monotherapy that can be used in the general community physician’s armamentarium, may hold significant value in the treatment of patients with CLL/SLL.

About COPIKTRA™ (duvelisib)

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status, and is being investigated in combination with other agents through investigator-sponsored studies.4 For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

About Verastem Oncology

Verastem Oncology (Nasdaq: VSTM) is a commercial biopharmaceutical company committed to the development and commercialization of medicines to improve the lives of patients diagnosed with cancer. We are driven by the strength, tenacity and courage of those battling cancer – single-minded in our resolve to deliver new therapies that not only keep cancer at bay, but improve the lives of patients diagnosed with cancer. Because for us, it’s personal.

Our first FDA approved product is now available for the treatment of patients with certain types of indolent non-Hodgkin’s lymphoma (iNHL). Our pipeline comprises product candidates that seek to treat cancer by modulating the local tumor microenvironment. For more information, please visit www.verastem.com.

Forward looking statements notice

This press release includes forward-looking statements about Verastem Oncology’s strategy, future plans and prospects, and financial results. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement.

Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the commercial success of COPIKTRA™ in the United States; physician and patient adoption of COPIKTRA, including those related to the safety and efficacy of COPIKTRA; the uncertainties inherent in research and development of COPIKTRA, such as negative or unexpected results of clinical trials; whether and when any applications for COPIKTRA may be filed with regulatory authorities in any other jurisdictions; whether and when regulatory authorities in any other jurisdictions may approve any such other applications that may be filed for COPIKTRA, which will depend on the assessment by such regulatory authorities of the benefit-risk profile suggested by the totality of the efficacy and safety information submitted and, if approved, whether COPIKTRA will be commercially successful in such jurisdictions; our ability to obtain, maintain and enforce patent and other intellectual property protection for COPIKTRA and our other product candidates; the scope, timing, and outcome of any legal proceedings; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of COPIKTRA; the fact that regulatory authorities in the U.S. or other jurisdictions, if approved, could withdraw approval; whether preclinical testing of our product candidates and preliminary or interim data from clinical trials will be predictive of the results or success of ongoing or later clinical trials; that the timing, scope and rate of reimbursement for our product candidates is uncertain; that third-party payors (including government agencies) may not reimburse for COPIKTRA; that there may be competitive developments affecting our product candidates; that data may not be available when expected; that enrollment of clinical trials may take longer than expected; that COPIKTRA or our other product candidates will cause unexpected safety events, experience manufacturing or supply interruptions or failures, or result in unmanageable safety profiles as compared to their levels of efficacy; that COPIKTRA will be ineffective at treating patients with lymphoid malignancies; that we will be unable to successfully initiate or complete the clinical development and eventual commercialization of our product candidates; that the development and commercialization of our product candidates will take longer or cost more than planned; that we may not have sufficient cash to fund our contemplated operations; that we, CSPC Pharmaceutical Group, Yakult Honsha Co., Ltd., Sanofi or Infinity Pharmaceuticals, Inc. will fail to fully perform under the duvelisib license agreements; that we may be unable to make additional draws under our debt facility or obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity, debt financing or otherwise; that we will not pursue or submit regulatory filings for our product candidates, including for duvelisib in patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or indolent non-Hodgkin lymphoma (iNHL) in other jurisdictions; and that our product candidates will not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients.

Other risks and uncertainties include those identified under the heading “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2019, as filed with the Securities and Exchange Commission (SEC) on August 1, 2019, its Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the SEC on March 12, 2019 and in any subsequent filings with the SEC. The forward-looking statements contained in this press release reflect Verastem Oncology’s views as of the date hereof, and the Company does not assume and specifically disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.

References

1

Winkler D.G., Faia K.L., DiNitto J.P. et al. PI3K-delta and PI3K-gamma inhibition by IPI-145 abrogates immune responses and suppresses activity in autoimmune and inflammatory disease models. Chem Biol 2013; 20:1-11.

2

Reif K et al. Cutting Edge: Differential Roles for Phosphoinositide 3 kinases, p110-gamma and p110-delta, in lymphocyte chemotaxis and homing. J Immunol 2004:173:2236-2240.

3

Schmid M et al. Receptor Tyrosine Kinases and TLR/IL1Rs Unexpectedly activate myeloid cell PI3K, a single convergent point promoting tumor inflammation and progression. Cancer Cell 2011;19:715-727.

4

www.clinicaltrials.gov, NCT03372057.

 

Contacts

Investors:
John Doyle

Vice President, Investor Relations & Finance

+1 781-469-1546

jdoyle@verastem.com

Media:
Lisa Buffington

Corporate Communications

+1 781-292-4205

lbuffington@verastem.com

UnitedHealthcare and Optum Take Action to Support People in Texas Affected by Floods

  • Assistance for UnitedHealthcare plan participants whose access to care or prescriptions needs may have been affected
  • Free emotional-support help line from Optum open to anyone

 

HOUSTON–(BUSINESS WIRE)–UnitedHealthcare and Optum, the health benefits and services companies of UnitedHealth Group (NYSE: UNH), are taking action to help people who may be affected by severe weather and flooding across parts of Texas.

Support includes assisting health plan participants who may need to make alternate arrangements to access care and early prescription refills, as well as offering a free emotional-support line to help people who might be affected.

  • Help Finding a Network Care Provider, Early Refills: Plan participants who need help finding a care provider in the UnitedHealthcare network or obtaining early prescription refills can call customer care at the number located on the back of their medical ID cards.

     
  • For plan participants who may have misplaced their medical ID cards, call 866-633-2446, 8 a.m.-8 p.m. (in the local time zone), Monday through Friday. People enrolled in Medicaid, employer-sponsored and individual health plans (except Medicare) who have a smartphone can download the free Health4Me app, which provides instant access to their ID card, network care providers, their personal health benefits and more. The Health4Me app is available as a free download at the Apple iTunes App Store and the Android Market on Google Play.

     
  • Free Help Line: Optum, a leading health and behavioral health services company, is offering a free emotional-support help line.

     

    The toll-free number, 866-342-6892, will be open 24 hours a day, seven days a week, for as long as necessary. The service is free of charge and open to anyone. Specially trained Optum mental health specialists help people manage their stress and anxiety so they can continue to address their everyday needs. Callers may also receive referrals to community resources to help them with specific concerns, including financial and legal matters.

     

    Along with the toll-free help line, emotional-support resources and information are available online at www.liveandworkwell.com.

About UnitedHealth Group

UnitedHealth Group (NYSE: UNH) is a diversified health care company dedicated to helping people live healthier lives and helping make the health system work better for everyone. UnitedHealth Group offers a broad spectrum of products and services through two distinct platforms: UnitedHealthcare, which provides health care coverage and benefits services; and Optum, which provides information and technology-enabled health services. For more information, visit UnitedHealth Group at www.unitedhealthgroup.com or follow @UnitedHealthGrp on Twitter.

Click here to subscribe to Mobile Alerts for UnitedHealth Group.

Contacts

Dimitri Senaratna

UnitedHealth Group

952-936-3275

dimitri_senaratna@uhg.com

 Ipsen to Present New Data at 13th Annual Conference of the International Liver Cancer Association (ILCA 2019)

Results from a matching-adjusted indirect comparison (MAIC) suggest that cabozantinib provides two additional months of progression-free survival versus regorafenib in the second-line treatment of advanced hepatocellular carcinoma1

PARIS–(BUSINESS WIRE)–Regulatory News:

Ipsen (Euronext: IPN; ADR: IPSEY) today presents results from the matching-adjusted indirect comparison (MAIC) of cabozantinib (Cabometyx®) versus regorafenib (Stivarga®) for the second-line treatment (2L) of patients with advanced hepatocellular carcinoma (aHCC) who received sorafenib as the only prior systemic therapy. Using data from the Phase III CELESTIAL and RESORCE trials, the MAIC showed that cabozantinib offers greater efficacy versus regorafenib.

Using data from the Phase III CELESTIAL and RESORCE trials, the MAIC showed that in the 2L CELESTIAL sub-population who had received sorafenib as the only prior systemic therapy, cabozantinib significantly improved progression-free survival (PFS), with an additional 2.4 months provided vs. regorafenib (5.6 months vs. 3.2 months [95% confidence interval (CI): 4.90-7.26], p<0.05). Median overall survival (OS) was also favorable with cabozantinib (11.4 months vs. 10.8 months), though statistical significance was not met.1

Results from MAIC will be presented by Dr. Katie Kelley, oncologist at the University of California, San Francisco and lead investigator, at the 13th Annual Conference of the International Liver Cancer Association (ILCA 2019) taking place on 20-22 September 2019 in Chicago, USA (poster/abstract #P-021).

In the previously presented randomized, double-blind, Phase III CELESTIAL trial evaluating cabozantinib compared with placebo in previously treated patients with aHCC, in the overall CELESTIAL intent-to-treat population, cabozantinib significantly improved median PFS, with an additional 3.3 months provided vs placebo (5.2 months vs. 1.9 months [95% CI, 4.0 to 5.5], p<0.001) and median OS, with an additional 2.2 months vs placebo (10.2 months vs. 8.0 months [95% confidence interval (CI): 9.1 to 12.0), p=0.005).1

Hepatocellular carcinoma is a devastating disease with only a few treatment options demonstrating survival benefits and many investigational drugs have failed to meet overall survival endpoints in clinical trials,” said Dr. Kelley. “The MAIC analysis brings further insight into the comparative effectiveness of the key second-line treatments for advanced hepatocellular carcinoma, particularly in relation to important endpoints like progression-free survival. These results may support clinicians in making informed treatment decisions in order to deliver optimal care for their patients.”

Grade 3/4 adverse events affecting more than 5% of patients were comparable for the two studies, except for diarrhea which was lower with regorafenib.1

MAICs are a way of providing a timely comparison of the effectiveness of different medical interventions in the absence of head-to-head randomized trials.2 While indirect comparisons of treatments across separate studies can be performed, these analyses may be biased by cross-trial differences in patient populations, sensitivity to modeling assumptions, and differences in the definitions of outcome measures. MAICs use individual patient data (IPD), also referred to as individual-level data, from trials of one treatment to match baseline summary statistics reported from trials of another treatment and reduce observed cross-trial differences.2 After matching, treatment outcomes are compared across balanced trial populations. It should be noted that, even after matching, bias may still occur in MAIC due to imbalance in unobserved factors, and it cannot completely replace a head-to-head randomized and controlled trial.1

At Ipsen, our mission is to prolong and improve patients’ lives and health outcomes, and we acknowledge the importance of providing healthcare professionals with the best available evidence to achieve these goals for patients,” said Dr. Yan Moore, Ipsen’s Senior Vice President, Head of Oncology Therapeutic Area. “The recent rapid development of new second-line treatments for patients with advanced HCC has led to the generation of information mainly based on placebo-controlled trials. While alternative methodological approaches such as MAIC are not substitutes to evidence-based prospective clinical trials, it is important to recognize the need for further insights into the comparative effectiveness of current treatment approaches.”

About the matching-adjusted indirect comparison of cabozantinib and regorafenib

The aim of this MAIC was to compare the safety and efficacy of cabozantinib and regorafenib for patients with aHCC who have received sorafenib as the only prior systemic therapy. Through the MAIC, IPD from patients enrolled in the CELESTIAL3 who had received cabozantinib as second-line therapy following sorafenib as the sole prior therapy (N=495) were adjusted to match the average baseline (BL) characteristics of the 573 patients enrolled in the regorafenib study RESORCE,4 for which individual-level data (ILD) are not available.

After matching, the selected BL characteristics were balanced across trials. The BL characteristics available for matching for both trials and deemed potential effect modifiers by key opinion leaders were:1

  • age group
  • race
  • geographical region
  • ECOG (Eastern Cooperative Oncology Group) performance status
  • Child-Pugh class
  • duration of prior sorafenib treatment
  • extrahepatic disease
  • macrovascular invasion
  • etiology of HCC (hepatitis B, alcohol use and hepatitis C)
  • AFP (alpha-fetoprotein tumor marker) level

In the first indirect comparison of cabozantinib and regorafenib in 2L HCC (post-sorafenib):1

  • Cabozantinib significantly improved median PFS, with an additional 2.4 months provided versus regorafenib (5.59 months vs. 3.19 months [95% CI: 4.90-7.26], p<0.05)
  • OS also favored cabozantinib, with a median OS of almost 1 year (11.37 months vs. 10.79 months), though statistical significance was not met

Grade 3/4 adverse events (AEs) affecting more than 5% of patients were comparable for the two studies, except for diarrhea which was significantly lower with regorafenib.

It should be noted that, even after matching, bias may still occur in MAIC due to imbalance in unobserved factors, and it cannot replace a head-to-head randomized control trial.

About CELESTIAL

CELESTIAL is a randomized, double-blind, placebo-controlled global Phase III study of cabozantinib versus placebo in patients with advanced hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. The study was conducted at more than 100 sites globally in 19 countries. The trial was designed to enroll 760 patients with advanced hepatocellular carcinoma (HCC) who previously received sorafenib and may have received up to two prior systemic cancer therapies for hepatocellular carcinoma (HCC) and had adequate liver function. Enrollment of the trial was completed in September 2017, and 773 patients were ultimately randomized. Patients were randomized 2:1 to receive 60 mg of cabozantinib once daily or placebo and were stratified based on etiology of the disease (hepatitis C, hepatitis B or other), geographic region (Asia versus other regions) and presence of extrahepatic spread and/or macrovascular invasion (yes or no). No cross-over was allowed between the study arms.

The primary endpoint for the trial is OS, and secondary endpoints include objective response rate and progression-free survival. Exploratory endpoints included patient-reported outcomes, biomarkers and safety.

Based on available clinical trial data from various published trials conducted in the second-line setting of advanced hepatocellular carcinoma (HCC), the CELESTIAL trial statistics for the primary endpoint of OS assumed a median OS of 8.2 months for the placebo arm. A total of 621 events provide the study with 90 percent power to detect a 32 percent increase in median OS (HR = 0.76) at the final analysis. Two interim analyses were planned and conducted at 50 percent and 75 percent of the planned 621 events.

CELESTIAL trial met its primary endpoint of overall survival (OS), with cabozantinib providing a statistically significant and clinically meaningful improvement in median OS compared to placebo in patients with advanced HCC. The independent data monitoring committee for the study recommended that the trial should be stopped for efficacy following review of the second planned interim analysis. The safety data in the study were consistent with the established profile of cabozantinib.

About hepatocellular carcinoma (HCC)

HCC is an aggressive and lethal disease with the number of deaths per year close to its incidence worldwide.5 It accounts for about 90% of all liver cancers and there were over 840,000 new cases of liver cancer in worldwide in 2018.5,6 It is the fifth most common cancer and the second most frequent cause of cancer-related death globally.7

About Ipsen products

This press release mentions investigational uses of Ipsen products. Product indications and approvals for use vary by jurisdiction; please see SmPC/PI for full indications and safety information.

About CABOMETYX® (cabozantinib)

CABOMETYX® is not marketed by Ipsen in the U.S.

CABOMETYX® 20mg, 40mg and 60mg film-coated unscored tablets

Active ingredient: Cabozantinib (S)-malate 20mg, 40mg and 60mg

Other components: Lactose

Indications: In the U.S., CABOMETYX® tablets are approved for the treatment of patients with advanced RCC and for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.

CABOMETYX® tablets are also approved in: the European Union, Norway, Iceland, Australia, Switzerland, South Korea, Canada, Brazil and Taiwan for the treatment of advanced RCC in adults who have received prior VEGF-targeted therapy; in the European Union for previously untreated intermediate- or poor-risk advanced RCC; in Canada for adult patients with advanced RCC who have received prior VEGF targeted therapy; and in the European Union, Norway and Iceland for HCC in adults who have previously been treated with sorafenib.

CABOMETYX® is not indicated for previously untreated advanced HCC.

Dosage and administration: The recommended dose of CABOMETYX® is 60 mg once daily. Treatment should continue until the patient is no longer clinically benefiting from therapy or until unacceptable toxicity occurs. Management of suspected adverse drug reactions may require temporary interruption and/or dose reduction of CABOMETYX therapy. For dose modification, please refer to full SmPC. CABOMETYX® is for oral use. The tablets should be swallowed whole and not crushed. Patients should be instructed to not eat anything for at least 2 hours before through 1 hour after taking CABOMETYX®.

Contraindications: Hypersensitivity to the active substance or to any of the excipients listed in the SmPC.

Special warnings and precautions for use:

Monitor closely for toxicity during first 8 weeks of therapy. Events that generally have early onset include hypocalcemia, hypokalemia, thrombocytopenia, hypertension, palmar-plantar erythrodysaesthesia syndrome (PPES), proteinuria, and gastrointestinal (GI) events.

Perforations and fistulas: serious gastrointestinal perforations and fistulas, sometimes fatal, have been observed with cabozantinib. Patients with inflammatory bowel disease, GI tumor infiltration or complications from prior GI surgery should be evaluated prior to therapy and monitored; if perforation and unmanageable fistula occur, discontinue cabozantinib.

Thromboembolic events: use with caution in patients with a history of or risk factors for thromboembolism; discontinue if acute myocardial infarction (MI) or other significant arterial thromboembolic complication occurs.

Hemorrhage: not recommended for patients that have or are at risk of severe hemorrhage.

Wound complications: treatment should be stopped at least 28 days prior to scheduled surgery (including dental).

Hypertension: monitor blood pressure (BP); reduce with persistent hypertension and discontinue should uncontrolled hypertension or hypertensive crisis occur.

Palmar-plantar erythrodysesthesia (PPES): interrupt treatment if severe PPES occurs.

Proteinuria: discontinue in patients with nephrotic syndrome.

Reversible posterior leukoencephalopathy syndrome (RPLS): discontinue in patients with RPLS.

QT interval prolongation: use with caution in patients with a history of QT prolongation, those on antiarrhythmics or with pre-existing cardiac disease.

Excipients: do not use in patients with hereditary problems of galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption.

Drug interactions: Cabozantinib is a CYP3A4 substrate. Potent CYP3A4 inhibitors may result in an increase in cabozantinib plasma exposure (e.g. ritonavir, itraconazole, erythromycin, clarithromycin, grapefruit juice). Coadministration with CYP3A4 inducers may result in decreased cabozantinib plasma exposure (e.g. rifampicin, phenytoin, carbamazepine, phenobarbital, St John’s Wort). Cabozantinib may increase the plasma concentration of P-glycoprotein substrates (e.g. fexofenadine, aliskiren, ambrisentan, dabigatran etexilate, digoxin, colchicine, maraviroc, posaconazole, ranolazine, saxagliptin, sitagliptin, talinolol, tolvaptan). MRP2 inhibitors may increase cabozantinib plasma concentrations (e.g. cyclosporine, efavirenz, emtricitabine). Bile salt sequestering agents may impact absorption or reabsorption resulting in potentially decreased cabozantinib exposure. No dose adjustment when co-administered with gastric pH modifying agents. A plasma protein displacement interaction may be possible with warfarin. INR values should be monitored in such a combination.

Women of childbearing potential/contraception in males and females: Ensure effective measures of contraception (oral contraceptive plus a barrier method) in male and female patients and their partners during therapy and for at least 4 months after treatment.

Pregnancy and lactation: CABOMETYX® should not be used during pregnancy unless the clinical condition of the woman requires treatment. Lactation – discontinue breast-feeding during and for at least 4 months after completing treatment. Drive and use machines: Caution is recommended

Adverse reactions:

The most common serious adverse reactions are hypertension, diarrhea, PPES, pulmonary embolism, fatigue and hypomagnesaemia. Very common (>1/10): anemia, lymphopenia neutropenia, thrombocytopenia, hypothyroidism, dehydration, decreased appetite, hyperglycemia, hypoglycemia, hypophosphatasemia, hypoalbuminemia, hypomagnesaemia, hyponatremia, hypokalemia, hyperkalemia, hypocalcemia, hyperbilirubinemia, peripheral sensory neuropathy, dysgeusia, headache, dizziness, hypertension, dysphonia, dyspnea, cough, diarrhea, nausea, vomiting, stomatitis, constipation, abdominal pain, dyspepsia, oral pain, dry mouth, PPES, dermatitis acneiform, rash, rash maculopapular, dry skin, alopecia, hair color change, pain in extremity, muscle spasms, arthralgia, proteinuria, fatigue, mucosal inflammation, asthenia, weight decreased, serum ALT, AST, and ALP increased, blood bilirubin increased, creatinine increased, triglycerides increased, white blood cell decreased, GGT increased, amylase increased, blood cholesterol increased, lipase increased. Common (>1/100 to <1/10): abscess, tinnitus, pulmonary embolism, pancreatitis, abdominal pain upper, gastro-esophageal reflux disease, hemorrhoids, pruritus, peripheral edema, wound complications. Uncommon (>1/1000 to <1/100): convulsion, anal fistula, hepatitis cholestatic, osteonecrosis of the jaw. Selected adverse events: GI perforation, fistulas, hemorrhage, RPLS.

Prescribers should consult the SPC in relation to other adverse reactions.

For more information, see the regularly updated registered product information on the European Medicine Agency www.ema.europa.eu

CABOMETYX® is marketed by Exelixis, Inc. in the United States. Ipsen has exclusive rights for the commercialization and further clinical development of CABOMETYX® outside of the United States and Japan.

About Ipsen

Ipsen is a global specialty-driven biopharmaceutical group focused on innovation and specialty care. The group develops and commercializes innovative medicines in three key therapeutic areas – Oncology, Neuroscience and Rare Diseases. Its commitment to Oncology is exemplified through its growing portfolio of key therapies for prostate cancer, neuroendocrine tumors, renal cell carcinoma and pancreatic cancer. Ipsen also has a well-established Consumer Healthcare business. With total sales over €2.2 billion in 2018, Ipsen sells more than 20 drugs in over 115 countries, with a direct commercial presence in more than 30 countries. Ipsen’s R&D is focused on its innovative and differentiated technological platforms located in the heart of the leading biotechnological and life sciences hubs (Paris-Saclay, France; Oxford, UK; Cambridge, US). The Group has about 5,700 employees worldwide. Ipsen is listed in Paris (Euronext: IPN) and in the United States through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information on Ipsen, visit www.ipsen.com.

Forward Looking Statement

The forward-looking statements, objectives and targets contained herein are based on the Group’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect the Group’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words “believes”, “anticipates” and “expects” and similar expressions are intended to identify forward-looking statements, including the Group’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by the Group. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising product in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. The Group must face or might face competition from generic products that might translate into a loss of market share. Furthermore, the Research and Development process involves several stages each of which involves the substantial risk that the Group may fail to achieve its objectives and be forced to abandon its efforts with regards to a product in which it has invested significant sums. Therefore, the Group cannot be certain that favorable results obtained during pre-clinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the product concerned. There can be no guarantees a product will receive the necessary regulatory approvals or that the product will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the Group’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the Group’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The Group also depends on third parties to develop and market some of its products which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to the Group’s activities and financial results. The Group cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of the Group’s partners could generate lower revenues than expected. Such situations could have a negative impact on the Group’s business, financial position or performance. The Group expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. The Group’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to the Group’s 2018 Registration Document available on its website (www.ipsen.com).

1 Abou-Alfa, G.K., et al. Cabozantinib in patients with advanced and progressing hepatocellular carcinoma. NEJM. 2018;379:54-63. Available at: https://www.nejm.org/doi/full/10.1056/NEJMoa1717002. Accessed August 2019.

2 Signorovitch, J.E., et al. Matching-adjusted indirect comparisons: a new tool for timely comparative effectiveness research. Value Health. 2012;15(6):940-7. Available at https://www.ncbi.nlm.nih.gov/pubmed/22999145. Accessed August 2019

3 Bruix, J., et al. Regorafenib for patients with hepatocellular carcinoma who progressed on sorafenib treatment (RESORCE): a randomized, double-blind, placebo-controlled, phase 3 trial. Lancet. 2017;389(10064):56-66. Available at: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(16)32453-9/fulltext. Accessed August 2019
4Aggarwal, M., et al. Systemic treatment for hepatocellular carcinoma. Chronic Dis Transl Med. 2018;4(3):148–155. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6160617/. Accessed August 2019.

5 American Institute of Cancer Research. Liver cancer statistics. Available at: https://www.wcrf.org/dietandcancer/cancer-trends/liver-cancer-statistics. Accessed August 2019.

6 European Association for the Study of the Liver. EASL Clinical Practice Guidelines: Management of hepatocellular carcinoma. J.

Contacts

Christian Marcoux

Corporate Communications

+33 (0) 1 58 33 67 94

christian.marcoux@ipsen.com

Kelly Blaney

Corporate Communications

+44 (0) 7903 402275

kelly.blaney@ipsen.com

Financial Community

Eugenia Litz

Vice President, Investor Relations

+44 (0) 1753 627721

eugenia.litz@ipsen.com

Myriam Koutchinsky

Investor Relations Manager

+33 (0)1 58 33 51 04

myriam.koutchinsky@ipsen.com

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Newron Pharmaceuticals: Commercialization Partner Meiji Seika Together with Eisai Announce the Approval of Safinamide for Parkinson’s Disease in Japan

MILAN–(BUSINESS WIRE)–$NWRN–Newron Pharmaceuticals S.pA. (“Newron”) (SIX: NWRN), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system, is pleased to note its partner Meiji Seika Pharma Co., Ltd., together with Eisai Co., Ltd., have announced the manufacturing and marketing approval in Japan for Equfina® TABLETS (safinamide mesilate, “safinamide”) for the indication of improvement of wearing-off phenomenon in patients with Parkinson’s disease under treatment with a drug containing levodopa.

The full text of the announcement from Meiji Seika and Eisai is as follows:

Eisai and Meiji Announce Parkinson’s Disease Treatment Equfina® TABLETS (Safinamide Mesilate) Approved in Japan

Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, “Eisai”) and Meiji Seika Pharma Co., Ltd. (Headqurters: Tokyo, CEO: Daikichiro Kobayashi, “Meiji”) today announced the manufacturing and marketing approval in Japan for the indication of improvement of wearing-off phenomenon in patients with Parkinson’s disease under treatment with a drug containing levodopa for Equfina® TABLETS (safinamide mesilate, “safinamide”), which was developed for use in the treatment of patients with Parkinson’s disease, was obtained. In Japan, Meiji holds the manufacturing and marketing approval for safinamide, and Eisai exclusively sells safinamide.

Parkinson’s disease is a neurodegenerative disease which causes motor impairment, with symptoms including tremors in the limbs, muscular rigidity and shuffling gait. It is caused by degeneration of the dopamine nervous system, which leads to a shortage of dopamine, a neurotransmitter in the brain. There are approximately 200,000 patients suffering from Parkinson’s disease in Japan1, and the number of patients is increasing due to the aging of the population.1, 2 Drugs containing levodopa are widely used to treat Parkinson’s disease by replenishing the brain’s supply of dopamine. However, as the disease progresses, levodopa’s duration of effect (“on” time) decreases, and there are cases where patients may experience wearing-off phenomena, a return of Parkinson’s disease symptoms before the next dose. Safinamide through its main mechanism of action as a selective monoamine oxidase B (MAO-B) inhibitor, increases the density of endogenous dopamine and exogenous dopamine from levodopa-containing drugs in the brain.

This manufacturing and marketing approval is based on a double-blind, placebo-controlled Phase II/III study (study ME2125-3) to evaluate the efficacy and safety of safinamide as add-on therapy and an open label Phase III study (study ME2125-4) to evaluate the safety and efficacy of long-term administration of safinamide in Japanese patients with Parkinson’s disease with wearing-off phenomena who are currently receiving levodopa, as well as global clinical trials.

In study ME2125-3, the change in mean daily “on” time from baseline to 24 weeks of the treatment phase, which is the primary endpoint, of treatment with safinamide 50 mg and 100 mg were statistically significant compared to placebo-controlled treatment. The most common adverse drug reactions (ADRs) (incidence 3% and higher) observed with patients with safinamide 50 mg and 100 mg were dyskinesia and visual hallucination. Also, in study ME2125-4, with regard to the change in mean daily “on” time from baseline to 52 weeks of the treatment phase, the “on” time with long-term administration of safinamide was extended, and showed the continued effectiveness. The most common ADRs (incidence 3% and higher) observed with patients were dyskinesia, falls, and constipation.

By providing Equifina TABLETS as a new option for Parkinson’s disease treatment, Eisai and Meiji will make further contributions to address the diverse needs of, and increase the benefits provided to, Parkinson’s disease patients and their families.

1. About Equfina® TABLETS (generic name: safinamide mesilate, “safinamide”)

Safinamide is a selective monoamine oxidase B (MAO-B) inhibitor, which reduces the degradation of excreted dopamine, helping to maintain the density of dopamine in the brain. Additionally, safinamide blocks sodium ion channels and inhibits glutamate release, and possesses both dopaminergic and non-dopaminergic mechanisms.

Global and domestic clinical trials of safinamide in combination with levodopa for the treatment of mid- to late-stage Parkinson’s disease showed extended “on” time and an improvement in motor function.3,4

2. About Licensing Agreement between Eisai and Meiji for Safinamide

Safinamide was discovered and developed by Newron Pharmaceuticals S.p.A. (Headquarters: Milan, Italy, “Newron”). In 2011, Newron entered into a licensing agreement with Meiji, granting Meiji exclusive rights to develop, manufacture and commercialize the drug in Japan and Asia. Under the license agreement signed between Eisai and Meiji in March 2017, Eisai has the exclusive rights to market safinamide in Japan, as well as to develop and market safinamide in Asia*. Safinamide is marketed under the name “Xadago” in 17 countries in Europe and the United States.

* South Korea, Taiwan, Brunei, Cambodia, Laos, Malaysia, the Philippines, Indonesia, Thailand, Vietnam, Myanmar, Singapore, Hong Kong, and Macau

3. About study ME2125-3 (Phase II/III Clinical Study)

Study ME2125-3 was a multicenter, double-blind, placebo-controlled, randomized, parallel group study to evaluate the efficacy and safety of two doses of safinamide (50 and 100 mg, once a day for 24 weeks) administered orally as add-on therapy in Japanese patients with Parkinson’s disease with wearing-off phenomenon who are currently receiving a drug containing levodopa. In this study, the primary endpoint was the change in mean daily “on” time from baseline to 24 weeks of the treatment phase, and verified the superiority of each dose of safinamide over placebo. Regarding the changes from baseline of mean daily “on” time of 24 weeks of treatment, the increases of 1.39 hours (95%CI: 0.67,2.11) with safinamide 50 mg and 1.66 hours (95%CI: 0.93,2.39) with safinamide 100 mg were shown compared to placebo, and statistically significant “on” time extension were indicated in patients with both safinamide 50 mg and 100 mg. The most common ADRs (incidence 3% and higher) observed in patients taking safinamide 50 mg and 100 mg were dyskinesia and visual hallucination.

4. About study ME2125-4 (Phase III Clinical Study)

Study ME2125-4 was an open-label, multicenter study to evaluate the long-term efficacy and safety of two doses of safinamide (50 and 100 mg, once a day for 52 weeks) administered orally as add-on therapy in Japanese patients with Parkinson’s disease with wearing-off phenomenon who are currently receiving a drug containing levodopa. In this study, in addition to evaluating the safety of long-term administration of safinamide, the study evaluated the change in mean daily “on” time from baseline to 52 weeks of the treatment phase as the primary efficacy endpoint. Regarding the changes (Least Square Mean (LSM) ± Standard Deviation of Lateral Position (SDLP)) from baseline of mean daily “on” time of 52 weeks of treatment, it was 1.42±2.72 hours, and the continuous efficacy of long-term administration was shown. The most common ADRs (incidence 3% and higher) observed were dyskinesia, falls, and constipation.

5. About Parkinson’s Disease

Parkinson’s disease is a neurodegenerative disease which causes motor impairment, including shaking in the limbs, muscular rigidity and shuffling gait. It is caused by degeneration of the dopamine nervous system, which leads to a shortage of dopamine, a neurotransmitter in the brain. According to the estimation of Japanese Society of Neurology, there are approximately 200,000 patients suffering from Parkinson’s disease in Japan.1 Also, the approximate 3 million patients suffer from Parkinson’s disease in Asia.5 The number of patients increasing due to the aging of the population.2 Levodopa is widely used to treat Parkinson’s disease by replenishing the brain’s supply of dopamine. However, as the disease progresses, the duration of a drug containing levodopa of effect (“on” time) decreases, and there are cases of Parkinson’s disease symptoms returning before the next dose (“wearing-off” phenomenon). To prevent the “wearing-off” phenomenon, combination therapy with a drug that has a different mechanism of action to a drug containing levodopa is administered.

6. About Eisai Co., Ltd.

Eisai Co., Ltd. is a leading global research and development-based pharmaceutical company headquartered in Japan. We define our corporate mission as “giving first thought to patients and their families and to increasing the benefits health care provides,” which we call our human health care (hhc) philosophy. With approximately 10,000 employees working across our global network of R&D facilities, manufacturing sites and marketing subsidiaries, we strive to realize our hhc philosophy by delivering innovative products to address unmet medical needs, with a particular focus in our strategic areas of Oncology and Neurology. As a global pharmaceutical company, our mission extends to patients around the world through our investment and participation in partnership-based initiatives to improve access to medicines in developing and emerging countries.

For more information about Eisai Co., Ltd., please visit https://www.eisai.com/.

7. About Meiji Seika Pharma Co., Ltd.

In order to protect and improve people’s health and lives, Meiji Seika Pharma, as a “Speciality and Generic Pharmaceuticals Company,” runs its pharmaceutical business in the two main fields, infectious disease and central nervous system disorders, as well as generic drugs. Meiji Seika Pharma strives to respond to diversified medical needs and contributes to the well-being of people worldwide.

For details, please visit its corporate website: https://www.meiji.com/global/about-us/corporate-profile/meiji-seika-pharma/

1 Japanese Society of Neurology. Treatment and Management Guideline 2018 for Parkinson’s Disease

2 Japan Intractable Diseases Information Center http://www.nanbyou.or.jp/
3 Borgohain R et al. Randomized Trial of Safinamide Add-On to Levodopa in Parkinson’s Disease With Motor Fluctuations. Mov Disord. 2014 Feb;29(2):229-37

4 Schapira AH et al. Assessment of Safety and Efficacy of Safinamide as a Levodopa Adjunct in Patients With Parkinson Disease and Motor Fluctuations: A Randomized Clinical Trial. JAMA Neurol. 2017 Feb 1;74(2):216-224

5 E Ray Dorsey et al. Global, regional, and national burden of Parkinson’s disease, 1990–2016: a systematic analysis for the Global Burden of Disease Study 2016 Lancet Neurol. 2018;17:939–53

About Newron Pharmaceuticals

Newron (SIX: NWRN, XETRA: NP5) is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system. The Company is headquartered in Bresso near Milan, Italy. Xadago®/safinamide has received marketing authorization for the treatment of Parkinson’s disease in the European Union, Switzerland, the USA, Australia, Canada, Brazil, the United Arab Emirates and Japan, and is commercialized by Newron’s Partner Zambon. US WorldMeds holds the commercialization rights in the USA. Meiji Seika has the rights to develop and commercialize the compound in Japan and other key Asian territories. In addition to Xadago®/safinamide for Parkinson’s disease, Newron has a strong pipeline of promising treatments for rare disease patients at various stages of clinical development, including sarizotan for patients with Rett syndrome and ralfinamide for patients with specific rare pain indications. Newron is also developing Evenamide as the potential first add-on therapy for the treatment of patients with positive symptoms of schizophrenia. For more information, please visit: www.newron.com

Important Notices

This document contains forward-looking statements, including (without limitation) about (1) Newron’s ability to develop and expand its business, successfully complete development of its current product candidates and current and future collaborations for the development and commercialisation of its product candidates and reduce costs (including staff costs), (2) the market for drugs to treat CNS diseases and pain conditions, (3) Newron’s anticipated future revenues, capital expenditures and financial resources, and (4) assumptions underlying any such statements. In some cases, these statements and assumptions can be identified by the fact that they use words such as “will,” “anticipate,” “estimate,” “expect,” “project,” “intend,” “plan,” “believe,” “target,” and other words and terms of similar meaning. All statements, other than historical facts, contained herein regarding Newron’s strategy, goals, plans, future financial position, projected revenues and costs and prospects are forward-looking statements. By their very nature, such statements and assumptions involve inherent risks and uncertainties, both general and specific, and risks exist that predictions, forecasts, projections and other outcomes described, assumed or implied therein will not be achieved. Future events and actual results could differ materially from those set out in, contemplated by or underlying the forward-looking statements due to a number of important factors. These factors include (without limitation) (1) uncertainties in the discovery, development or marketing of products, including without limitation negative results of clinical trials or research projects or unexpected side effects, (2) delay or inability in obtaining regulatory approvals or bringing products to market, (3) future market acceptance of products, (4) loss of or inability to obtain adequate protection for intellectual property rights, (5) inability to raise additional funds, (6) success of existing and entry into future collaborations and licensing agreements, (7) litigation, (8) loss of key executive or other employees, (9) adverse publicity and news coverage, and (10) competition, regulatory, legislative and judicial developments or changes in market and/or overall economic conditions. Newron may not actually achieve the plans, intentions or expectations disclosed in forward-looking statements, and assumptions underlying any such statements may prove wrong. Investors should therefore not place undue reliance on them. There can be no assurance that actual results of Newron’s research programmes, development activities, commercialisation plans, collaborations and operations will not differ materially from the expectations set out in such forward-looking statements or underlying assumptions. Newron does not undertake any obligation to publicly update or revise forward-looking statements except as may be required by applicable regulations of the SIX Swiss Exchange, where the shares of Newron are listed. This announcement is not an offer for sale of securities in the United States, Canada, Australia or Japan or any other jurisdiction where such an offer or solicitation would otherwise be unlawful. The securities referred to herein may not be sold in the United States absent registration or an exemption from registration under the U.S. Securities Act of 1933, as amended. Newron does not intend to register any of its securities in the United States or to conduct a public offering of its securities in the United States. This document does not contain or constitute an offer or invitation to purchase or subscribe for any securities of Newron and no part of this document shall form the basis of or be relied upon in connection with any contract or commitment whatsoever.

Contacts

Newron
Stefan Weber – CEO

+39 02 6103 46 26

pr@newron.com

UK/Europe
Julia Phillips / Natalie Garland-Collins, FTI Consulting

+44 20 3727 1000

SCnewron@fticonsulting.com

Switzerland
Martin Meier-Pfister, IRF

+41 43 244 81 40

meier-pfister@irf-reputation.ch

Germany/Europe
Anne Hennecke, MC Services

+49 211 52925222

anne.hennecke@mc-services.eu

USA
Paul Sagan, LaVoieHealthScience

+1 617 374 8800, Ext. 112

psagan@lavoiehealthscience.com