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Astellas to Acquire Ganymed Pharmaceuticals -Acquisition Would Expand Astellas’ Oncology Pipeline with Antibody in Late-Stage –

On October 28, 2016 Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, "Astellas") and Ganymed Pharmaceuticals AG (CEO; Özlem Türeci, "Ganymed"), a biopharmaceutical company located in Mainz, Germany which focuses on the development of antibodies against cancer, reported that Astellas and Ganymed’s shareholders have entered into an agreement for Astellas to acquire Ganymed (Press release, Astellas, OCT 27, 2016, View Source [SID1234516055]). The transaction would enable Astellas to continue to build upon its leading oncology franchise as a platform for sustainable growth.

Under the agreement, Astellas will pay EUR 422 million to acquire 100% of the equity in Ganymed. In addition, Ganymed’s shareholders will become eligible to receive up to EUR 860 million in further contingent payments based on progress in the development of IMAB362, Ganymed’s most advanced clinical program. Upon completion of the transaction, Ganymed would become a wholly owned subsidiary of Astellas. The closing of the transaction is subject to customary regulatory approvals, and is expected to be finalized in the next several weeks.

"Oncology is one of our focus therapeutic areas and key drivers for our growth. The acquisition of Ganymed will enable Astellas to further expand our oncology presence by adding a late-stage antibody asset with the potential to establish a new pillar following XTANDI," commented Yoshihiko Hatanaka, President and CEO, Astellas. "We aim to deliver a potential new therapeutic option to cancer patients who currently have limited treatment options available to them."

Dr. Özlem Türeci, CEO and co-founder of Ganymed said, "I am impressed by the competence, commitment and vision of Astellas. Recognizing their global resources, I am also confident that Astellas will be able to turn innovative science and promising clinical results into near-term value for patients". And Helmut Jeggle, Ganymed’s Supervisory Board member and General Manager of the family office of the Strüngmann brothers (Athos Beteiligungsverwaltung GmbH) added, "We are pleased to hand over the Ganymed portfolio with its important novel treatment approaches for unmet needs in solid tumors to a strong and dedicated player in healthcare".

Ganymed is a privately-held biopharmaceutical company founded in 2001 and focuses on the development of a new class of cancer drugs. Ganymed has several oncology pipeline assets in pre-clinical and clinical stages including IMAB362. Recent results of a Phase 2b study (FAST) of IMAB362 in gastroesophageal cancer patients positive for Claudin18.2 showed that IMAB362 extended the median progression-free survival (7.9 months vs. 4.8 months, HR 0.47, p=0.0001) and the median overall survival (13.2 months vs. 8.4 months, HR 0.51, p=0.0001) when added to standard chemotherapy. In the subgroup of patients with the highest levels of Claudin18.2, IMAB362 resulted in near-doubling of overall survival (16.7 months vs. 9.0 months, HR 0.45, p<0.0005). The most frequent adverse effects observed during the study were vomiting, nausea and neutropenia.

Astellas is still reviewing the impact of this transaction on its financial forecasts for the fiscal year ending March 31, 2017.

Acquisition Summary
Acquiring company: Astellas Pharma Inc.
Major shareholders of Ganymed: ATS Beteiligungsverwaltung GmbH, MIG Fonds, FCP Gany GmbH and Future Capital AG
Payment: Cash on hand
Amount:
EUR 422 million upon the acquisition of 100% equity in Ganymed
Up to EUR 860 million in further contingent payments based on progress in the development of IMAB362, Ganymed’s most advanced clinical program
Expected timing of closing: Next several weeks, subject to customary regulatory approvals
Finance Advisor to Astellas: None
Legal Advisor to Astellas: Latham & Watkins LLP
Finance Advisor to Ganymed’ shareholders: klugeconcepts, JP Morgan
Legal Advisor to Ganymed`shareholders: Freshfields Bruckhaus Deringer, honert + partner

Overview of Acquired Company
Corporate Name: Ganymed Pharmaceuticals AG
Location: Mainz, Germany
Representative: Özlem Türeci, CEO
Founded year: 2001
Capital Stock: EUR 1,416,308 (as of end of May 2016)
Number of employees: 85
Relationship with Astellas: There is no relationship between Astellas and Ganymed required to be disclosed

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About IMAB362
IMAB362 is a new investigational antibody drug that is specific for the tight junction protein Claudin18.2. This unique target is restricted to stomach cells only and is absent from all other healthy tissues. Claudin 18.2 is expressed in various high medical need cancers including in about 80% of gastrointestinal adenocarcinomas, 60% pancreatic tumors as well as in biliary, ovarian and lung cancer. Its mechanism of action includes activation of antibody-dependent cell-mediated cytotoxicity (ADCC), complement-dependent cytotoxicity (CDC) and – in combination with chemotherapy – T-cell infiltration and modulation of the tumor microenvironment. IMAB362 has received orphan drug designation in the US and Europe for gastric and pancreatic cancer.

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The Myriad myPath® Melanoma Test Effectively Diagnosed Patients in the Largest Outcomes-Based Study for a Melanoma Diagnostic

On October 27, 2016 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported it will present two new studies at the American Society of Dermatopathology (ASDP) annual meeting being held Oct. 27-30, 2016 in Chicago, Ill (Press release, Myriad Genetics, OCT 27, 2016, View Source [SID1234516051]). The research being presented validates the accuracy of Myriad myPath Melanoma in differentiating benign skin nevi from malignant melanoma.

"We are presenting landmark data from the largest outcomes-based study ever performed with a melanoma diagnostic," said Loren Clarke, M.D., medical director, Dermatology, Myriad Genetic Laboratories. "The myPath Melanoma test performed very well and identified patients with melanoma versus benign skin lesions with greater than 95 percent diagnostic accuracy, which is exceptional in molecular diagnostics for cancer, particularly given the extreme heterogeneity of melanoma."

"Pigmented or suspect skin lesions are difficult to diagnose in approximately 15 percent of cases," said Sancy Leachman, M.D., Ph.D., chair of the Department of Dermatology in the Oregon Health & Science University School of Medicine and director of the Melanoma Research Program at the Knight Cancer Institute. "A highly accurate biomarker like the myPath Melanoma test should help dermatologists augment their diagnosis of melanoma, improve patient care and lower healthcare costs."

Below are the featured presentations at ASDP (#ASDP2016).

Poster Presentation
Title: Diagnostic Distinction of Malignant Melanoma and Benign Nevi by a Gene Expression Signature and Correlation to Clinical Outcome.
Presenter: Jennifer Ko.
Date: Friday, Oct. 28, 2016: 4:15 — 5:00 p.m. and Saturday, Oct. 29, 2016 10:00 — 10:45 a.m. CT.

In this study, research collaborators from the Cleveland Clinic, Stanford University and Nottingham University assessed the clinical accuracy (sensitivity and specificity) of the myPath Melanoma test against clinical outcomes in 182 patients with skin lesions (99 melanomas and 83 nevi) with more than 5 years of follow up. The results show that the myPath Melanoma test accurately differentiated benign lesions from melanoma with a sensitivity of 93.8 percent and a specificity of 96.2 percent when compared to known clinical outcomes. The diagnostic accuracy of the myPath Melanoma test was high even in a subset of difficult-to-diagnose cases and, in combination with two previous validation studies, the findings support its use as an adjunct method for the early and accurate diagnosis of melanoma.

Podium Presentation
Title: Gene Expression Signature as an Ancillary Method in the Diagnosis of Desmoplastic Melanoma.
Presenter: Loren Clarke.
Date: Sunday, Oct. 30, 2016: 8:20 — 8:30 a.m. CT.

The objective of this study was to assess the accuracy of the myPath Melanoma test in the differentiation of desmoplastic melanoma (DM) from benign skin lesions. These lesions represent approximately one percent of melanomas, but are known to be very difficult to diagnose. The analysis included samples from 20 patients with DM and 27 from patients with benign moles (nevi). The results showed that the myPath Melanoma test was positive in 15 of the 20 known melanomas, negative in four and indeterminate in one. The myPath score was negative in 24 of the benign nevi and indeterminate in three. Based on these findings, the myPath Melanoma test demonstrated approximately 80 percent diagnostic accuracy in this very difficult-to-diagnose subtype.

For more information about the meeting, please visit the ASDP website at View Source Follow Myriad on Twitter via @MyriadGenetics to stay informed about news and updates from the Company.

About Melanoma
Melanoma is one of the fastest growing cancers in the United States and can strike people of all ages, races and skin types. With a one-in-50 lifetime risk of developing melanoma, nearly 76,000 Americans are expected to be diagnosed with Stage I-IV melanoma and another 68,000 will be diagnosed with melanoma in situ — totaling approximately 144,000 total diagnoses. Early and accurate diagnosis of melanoma is critical for long-term survival. For more information visit: www.mypathmelanoma.com/ and www.myriadpro.com/melanoma.

About Myriad myPath Melanoma
Myriad myPath Melanoma is a clinically validated test to be used as an adjunct to histopathology when the distinction between a benign nevus and a malignant melanoma cannot be made confidently by histopathology alone. The test measures the expression of 23 genes and accurately distinguishes melanoma from benign nevi.

AstraZeneca head and neck cancer trials

On October 27, 2016 following the recent update on clinicaltrials.gov, AstraZeneca reported that the US FDA has placed a partial clinical hold on the enrolment of new patients with head and neck squamous cell carcinoma (HNSCC) in clinical trials of durvalumab as monotherapy and in combination with tremelimumab or other potential medicines (Press release, AstraZeneca, OCT 27, 2016, View Source [SID1234516048]). All trials are continuing with existing patients.

The partial clinical hold on new patient enrolment relates only to head and neck cancer. Trials for durvalumab in different cancer types, as monotherapy or in combination with tremelimumab or other potential medicines, are progressing as planned, with pivotal data in lung cancer anticipated in the first half of 2017.

The FDA’s decision follows voluntary action by AstraZeneca to pause enrolment of new HNSCC patients while a detailed analysis is conducted of adverse events related to bleeding that were observed as part of routine safety monitoring of the Phase III KESTREL and EAGLE trials. Bleeding is a known complication in treatments of head and neck cancers primarily due to the nature of the underlying disease, the proximity of tumours to major blood vessels and use of prior cancer therapies, which may involve surgery and radiation.

AstraZeneca has submitted its analysis of the observed bleeding events to the FDA for review and is working closely with the Agency, providing the required information to resume new patient enrolment as soon as possible.

TG Therapeutics, Inc. Announces Publication in Blood Describing a Novel Complimentary Mechanism of the PI3K-delta Inhibitor, TGR-1202

On October 27, 2016 TG Therapeutics, Inc. (NASDAQ:TGTX) reported the publication of preclinical data describing the synergy of the Company’s next generation, once daily, PI3K-delta inhibitor, TGR-1202, with the proteasome inhibitor carfilzomib and the unique effects of the combination to silence c-Myc in various preclinical lymphoma and myeloma models (Press release, TG Therapeutics, OCT 27, 2016, View Source [SID1234516047]). In addition, the manuscript also, for the first time, reports on TGR-1202’s unique complimentary mechanism of inhibiting the protein kinase casein kinase-1 (CK1) epsilon, which may contribute to the silencing of c-Myc and explain TGR-1202’s clinical activity in aggressive lymphoma, including Diffuse Large B-cell Lymphoma (DLBCL). The preclinical data are described further in the manuscript titled, "Silencing c-Myc Translation as a Therapeutic Strategy through Targeting PI3K Delta and CK1 Epsilon in Hematological Malignancies," which was published online yesterday in the First Edition section of Blood, the Journal of the American Society of Hematology (ASH) (Free ASH Whitepaper). The online version of the article can be accessed at www.bloodjournal.org.

"We want to thank Dr. Deng, Dr. O’Connor, and the team from Columbia Presbyterian Medical Center and the Center for Lymphoid Malignancies for their exhaustive and comprehensive interrogation of TGR-1202 and the elucidation of this novel complimentary mechanism. For quite some time we have been presenting the differentiated safety profile observed with TGR-1202 and the differentiated chemical structure compared to other PI3K-delta inhibitors. This preclinical work demonstrates that TGR-1202 not only potently targets PI3K-delta but in addition uniquely targets a relatively novel kinase, CK1-epsilon, which perhaps offers another rationale for the differentiated activity and safety effects we have seen in patients. We look forward to exploring this exciting concept further in the recently launched clinical trial," stated Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer.

"The data in this paper clearly demonstrates that TGR-1202 and carfilzomib in combination is markedly synergistic and selectively silenced c-Myc compared to combinations with idelalisib and bortezomib. In addition, we were excited to identify and elucidate the previously unknown mechanism of TGR-1202 and its effect on CK1 epsilon which was not exhibited by either idelalisib or duvelisib based on a kinome profiling platform analyzed. We believe this research may help explain in part the preliminary activity demonstrated by TGR-1202 in DLBCL. Given TGR-1202’s distinct safety profile as a single agent and its uniquely demonstrated ability to be used in combination with other agents, we look forward to bringing this novel combination to the clinic in our recently announced Phase 1 study of TGR-1202 and carfilzomib in patients with lymphoma," stated Dr. Owen A. O’Connor, Professor of Medicine and Experimental Therapeutics, Director Lymphoid Malignancies at Columbia Presbyterian Medical Center.

Based on this extensive preclinical work, the Company recently announced the launch of a Phase 1/2 study to evaluate the safety and efficacy of TGR-1202 in combination with carfilzomib, in patients with relapsed or refractory lymphoma, particularly c-Myc driven lymphomas which are aggressive in nature. This study is currently open to enrollment at the Center for Lymphoid Malignancies, Columbia Presbyterian Medical Center, New York, NY. More information on this clinical study can be found at www.clinicaltrials.gov.

Sobi™ publishes its report for the third quarter 2016, raises guidance

On October 27, 2016 Swedish Orphan Biovitrum AB (publ) (Sobi) reported its results for the third quarter 2016 (Press release, Swedish Orphan Biovitrum, OCT 27, 2016, View Source;Media/News/RSS/?RSS=View Source [SID1234516046]). Revenue for the quarter totalled SEK 1,171 M (786), an increase of 49 per cent compared to previous year. Based on strong performance across the portfolio and an earlier launch for Alprolix, the company has raised guidance for the full year.

Business highlights Q3 2016

Elocta reimbursed in the UK, Italy, France and Spain
Alprolix reimbursed in the UK
Long term Elocta and Alprolix data presented at WHF 2016 World Congress
Orfadin capsule filing validated by Health Canada
Milan Zdravkovic appointed as SVP, Head of R&D
Financial highlights Q3 2016 (Q3 2015)

Total revenue of SEK 1,171 M (786), an increase of 49 per cent
Product revenue of SEK 1,009 M (645), an increase of 56 per cent (57 per cent at CER)
Gross margin of 67 per cent (62)
EBITA of SEK 282 M (97)
Ended the quarter with a cash position of SEK 824 M
Earnings per share 0.53 SEK (0.02)
"Results in the third quarter had a positive contribution from the ongoing launch of Elocta, from an earlier than anticipated launch for Alprolix and from Kineret. We continue to lay the foundation for a sustainable haemophilia business in Europe with both Elocta and Alprolix gaining several important reimbursement approvals in major markets", said Geoffrey McDonough, CEO and President at Sobi.

"A significant milestone after the quarter was the orphan designation approval by the European Commission for our development candidate SOBI003 – a chemically modified human recombinant sulfamidase for the treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome). MPS IIIA is a severe and debilitating disease with devastating consequences for patients, and there is presently no treatment available."

Financial Summary
Q3 Q3 Jan-Sep Jan-Sep Full year
Amounts in SEK M 2016 2015 Change 2016 2015 Change 2015
Total revenues1 1 171 786 49% 3 913 2 414 62% 3 228
Gross profit 782 486 61% 2 791 1 486 88% 2 007
Gross margin 67% 62% 71% 62% 62%
EBITA 282 97 >100% 1 334 343 >100% 433
EBIT (Operating profit/loss) 171 25 >100% 1 034 129 >100% 146
Profit/loss for the period 143 5 >100% 710 77 >100% 68
1Jan-Sep 2016 revenues include a one-time credit in Q1 of SEK 322 M relating to the first commercial sales of Elocta, and a one-time credit in Q2 of SEK 386 M relating to first commercial sales of Alprolix.
Outlook 2016 – guidance raised

For the full-year 2016, Sobi now expects revenues of SEK 5,125—5,200 M (4,800-5,000). Revenues include one-time credits for Elocta of SEK 322 M and for Alprolix of SEK 386 M which do not impact cash. Gross margin is now expected to be 70 per cent (68-70) and EBITA for the full-year in the range of SEK 1,475–1,525 M (1,200-1,300).

The original outlook was published 29 February 2016.

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Sobi’s report for the third quarter 2016 can be found on View Source;Media/Financial-Reports/