On July 18, 2016 Delcath Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, reported that abstracts from two studies conducted in Germany of use of the Delcath Hepatic CHEMOSAT Delivery System to treat patients with liver metastases, have been accepted for presentation as posters at the Cardiovascular and Interventional Radiology Society of Europe (CIRSE) annual meeting (Press release, Delcath Systems, JUL 18, 2016, View Source;p=RssLanding&cat=news&id=2186055 [SID:1234513927]). The abstracts are:

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Percutaneous Isolated Hepatic Perfusion (Chemosaturation) In Patients With Primary Or Secondary Liver Tumours: Experience In 20 Patients, by S. Marquardt, et al, of Hanover Medical School in Hanover, Germany.

Secondary Resectability Of Ocular Melanoma Liver Metastases Following Percutaneous Hepatic Perfusion by M. Zeile, and A. Stang, et al of the Asklepios Barmbek Clinic in Hamburg, Germany.

On July 18, 2016 ArQule, Inc. (Nasdaq:ARQL) reported the first public presentation of data on its novel BTK inhibitor, ARQ 531, at the 2016 Pan Pacific Lymphoma Conference in Koloa, Hawaii (Press release, ArQule, JUL 18, 2016, View Source [SID:1234513933]). ARQ 531 is an investigational, orally bioavailable, potent and reversible Bruton’s tyrosine kinase (BTK) inhibitor.

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In preclinical testing, ARQ 531 demonstrated biochemical inhibition of both wild type and C481S-mutant BTK at sub-nanomolar levels and potent cellular inhibition in C481S-mutant BTK cells that are resistant to ibrutinib. This molecule also exhibits a distinct kinase selectivity profile with inhibitory activity against several key oncogenic drivers related to ibrutinib resistance. It is currently estimated that about 10% of patients treated with ibrutinib develop resistance with over 80% presenting the C481S mutation. This incidence rate is expected to increase.

Additionally, the compound potently suppresses cell proliferation of hematological malignancies in vitro, with B-cell receptor signaling inhibition. ARQ 531 also demonstrates strong in vivo target and pathway inhibition of phospho-BTK with potent and durable growth suppression.

"We are beginning to see increasing resistance to ibrutinib which is creating the need for a BTK inhibitor, like ARQ 531, that targets the C481S mutation," said Dr. Brian Schwartz, Head of Research and Development and Chief Medical Officer at ArQule. "The preclinical profile of ARQ 531 as a potent and reversible inhibitor of wild type and mutant BTK presents the potential for a first-in-class and best-in-class molecule. We are working toward completing GLP toxicology studies and filing an IND in early 2017."

The poster titled "ARQ 531, a Novel, Oral, Non-Covalent Inhibitor of Wild Type and C481S Mutant BTK with Potent Anti-Tumor Activity" can be viewed at View Source

About BTK and ARQ 531

ARQ 531 is an investigational, orally bioavailable, potent and reversible Bruton’s tyrosine kinase (BTK) inhibitor. Biochemical and cellular studies have shown that ARQ 531 inhibits both the wild type and C481S-mutant forms of BTK. The C481S mutation is a known emerging resistance mechanism for first generation irreversible BTK inhibitors. ARQ 531 has high oral bioavailability as well as good ADME, pharmacokinetic and metabolic properties. The company plans to file an IND for ARQ 531 in early 2017. BTK is a therapeutic target that has been clinically proven to inhibit B-cell receptor signaling in blood cancers.

On July 18, 2016 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the phase III GOYA study evaluating Gazyva/Gazyvaro (obinutuzumab) plus CHOP chemotherapy (G-CHOP) in people with previously untreated diffuse large B-cell lymphoma (DLBCL) did not meet its primary endpoint of significantly reducing the risk of disease worsening or death (progression-free survival; PFS) compared to MabThera/Rituxan (rituximab) plus CHOP chemotherapy (R-CHOP) (Press release, Hoffmann-La Roche , JUL 18, 2016,
View Source [SID:1234513926]).

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Adverse events with Gazyva/Gazyvaro and MabThera/Rituxan were consistent with those seen in previous clinical trials when each was combined with various chemotherapies. Data from the GOYA study will be presented at an upcoming medical meeting.

"Two previous studies showed Gazyva/Gazyvaro helped people with previously untreated follicular lymphoma or chronic lymphocytic leukaemia live longer without their disease worsening compared to MabThera/Rituxan, when each was combined with chemotherapy. We were hopeful we could show a similar result for people with diffuse large B-cell lymphoma and once again improve on the standard of care," said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development. "We will continue to analyse the GOYA data to better understand the results, and to study other investigational treatments in this disease with the goal of further helping these patients."

About the GOYA study
GOYA (NCT01287741) is a global phase III open-label, multi-centre, randomised two-arm study examining the efficacy and safety of the combination of Gazyva/Gazyvaro plus CHOP chemotherapy (G-CHOP) compared to MabThera/Rituxan plus CHOP chemotherapy (R-CHOP). GOYA included 1,418 previously untreated patients with CD20-positive DLBCL. The primary endpoint of the study is investigator-assessed PFS, with secondary endpoints including PFS assessed by independent review committee (IRC), response rate (overall response, ORR; and complete response, CR), overall survival (OS), disease free survival (DFS) and safety profile. The GOYA study is being conducted in cooperation with the Fondazione Italiana Linfomi (FIL, Italy).

About Gazyva/Gazyvaro (obinutuzumab)
Gazyva/Gazyvaro is an engineered monoclonal antibody designed to attach to CD20, a protein expressed on certain B cells, but not on stem cells or plasma cells. Gazyva/Gazyvaro is designed to attack and destroy targeted B-cells both directly and together with the body’s immune system.
Gazyva/Gazyvaro is currently approved in more than 70 countries in combination with chlorambucil, for people with previously untreated chronic lymphocytic leukaemia. The approvals were based on the CLL11 study, showing significant improvements with Gazyva/Gazyvaro plus chlorambucil across multiple clinical endpoints, including PFS, overall response rate (ORR), complete response rate (CR), and minimal residual disease (MRD) when compared head-to-head with MabThera/Rituxan plus chlorambucil.
In February 2016, Gazyva was approved by the US Food and Drug Administration in combination with bendamustine followed by Gazyva alone for people with follicular lymphoma who did not respond to a Rituxan-containing regimen, or whose follicular lymphoma returned after such treatment. In June 2016, Gazyvaro was approved by the European Commission in combination with bendamustine followed by Gazyvaro maintenance in people with follicular lymphoma who did not respond or who progressed during or up to six months after treatment with MabThera or a MabThera-containing regimen. Both approvals were based on the phase III GADOLIN study, showing a significant improvement in progression-free survival with Gazyva/Gazyvaro-based therapy compared to bendamustine alone. Gazyva is marketed as Gazyvaro in the EU and Switzerland.

In May 2016, the phase III GALLIUM study in people with previously untreated follicular lymphoma met its primary endpoint early. GALLIUM compared the efficacy and safety of Gazyva/Gazyvaro plus chemotherapy (CHOP, CVP or bendamustine) followed by Gazyva/Gazyvaro alone, head-to-head with MabThera/Rituxan plus chemotherapy followed by MabThera/Rituxan alone. Results from a pre-planned interim analysis showed that Gazyva/Gazyvaro-based treatment resulted in superior progression-free survival compared to MabThera/Rituxan-based treatment. Adverse events with either Gazyva/Gazyvaro or MabThera/Rituxan were consistent with those seen in previous clinical trials when each was combined with various chemotherapies. Data from the GALLIUM study will be presented at an upcoming medical meeting and submitted to health authorities for approval consideration.
Additional combination studies investigating Gazyva/Gazyvaro with other approved or investigational medicines, including cancer immunotherapies and small molecule inhibitors, are underway across a range of blood cancers.

About DLBCL
Diffuse large B-cell lymphoma (DLBCL) is the most common subtype of non-Hodgkin lymphoma (NHL), accounting for about one in three cases of NHL1. DLBCL is an aggressive (fast-growing) type of NHL, which is generally responsive to treatment in the frontline2. However, as many as 40% of patients will relapse, at which time salvage therapy options are limited and survival is short2. Approximately 123,000 people worldwide are estimated to be diagnosed with DLBCL each year3.

On July 19, 2016 Apogenix, a biopharmaceutical company developing next-generation immuno-oncology therapeutics, reported that the European Patent Office has granted the Company a patent for the use of CD95 ligand inhibitors including APG101 in the treatment of myelodysplastic syndromes (MDS) (Press release, Apogenix, JUL 16, 2016, View Source [SID1234524578]). The patent covers the use of such inhibitors in low to intermediate-1 risk transfusion-dependent MDS patients and is valid until July 2033.

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While Apogenix already holds a broad patent portfolio covering APG101, including composition of matter as well as manufacturing and method of treatment of glioblastoma, this is the first patent to cover its clinical use specifically in MDS patients.

"The granting of this additional patent for APG101 further validates our innovative drug development approach and strengthens the protection of our most advanced asset," said Thomas Hoeger, Ph.D., CEO of Apogenix. "With the recently announced positive results from the Phase I trial in MDS patients, we now intend to further develop APG101 in this indication, for which today there are no sufficient treatment options available.

The recently completed Phase I trial evaluated APG101 in low to intermediate-1 risk transfusion-dependent MDS patients and demonstrated the tolerability as well as activity of the drug candidate: APG101 treatment stimulated erythropoiesis and led to a significant decrease in transfusion frequency in this patient population. To further evaluate the efficacy and safety of APG101 in the treatment of MDS patients, Apogenix is currently in preparation of Phase II proof-of-concept trials.
About Myelodysplastic Syndromes (MDS) MDS is a bone marrow disorder that is characterized by ineffective hematopoiesis and can lead to severe anemia. In most cases, the anemia is treated with blood transfusions that eventually result in an iron overload, which can damage the liver and other organs. At the same time, the number of thrombocytesthat are responsible for coagulation and the number of leucocytes that are responsible for immune defense significantly decrease in patients with this disorder. As a result, MDS patients frequently suffer from sudden bleeding and lifethreatening infections. In addition, they are at risk of developing acute myeloid leukemia, a type of blood cancer. About APG101 Apogenix’s lead immuno-oncology candidate APG101 is a fully human fusion protein that consists of the extracellular domain of the CD95 receptor and the Fc domain of an IgG antibody. APG101 is being developed for the treatment of solid tumors and malignant hematological diseases. By blocking the CD95 ligand, which negatively regulates erythrocyte production in MDS patients, APG101 directly addresses the cause of the disorder and could thus potentially provide a cure for MDS.

On July 15, 2016 (Sobi) reported its results for the second quarter 2016 (Press release, Swedish Orphan Biovitrum, JUL 15, 2016, View Source;Media/News/RSS/?RSS=View Source [SID:1234513892]). Revenue for the quarter totalled SEK 1,469 M (764), an increase of 92 per cent compared to previous year. All parts of the business contributed to the result with Haemophilia, Inflammation and Partner Products delivering strong performance.

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Business Summary Q2 2016

Alprolix approved in the EU with first sales reported in Germany in early June
Elocta approved in Switzerland
European patent granted on new formulation of Kineret
Signed licensing agreement with Affibody for IL-1
Signed two manufacturing agreements with Pfizer
Orfadin Oral Suspension and 20 mg capsule approved in the US
Entered into new credit facility and redeemed SEK 800 M bond loan 2012/2017 prior to final maturity
Financial Summary Q2 2016 (Q2 2015)

Total revenue was SEK 1,469 M (764), an increase of 92 per cent (95 per cent at CER)
Product revenue was SEK 1,288 M (593), an increase of >100 per cent (>100 per cent at CER)
Revenues include a SEK 386 M one-time credit from Biogen triggered by first commercial sales of Alprolix
Gross margin was 72 per cent (63)
EBITA was SEK 550 M (74)
Earning per share 0.99 SEK (-0.01)
"The highlight of the first half of 2016 has been the launch of our long-acting Haemophilia franchise in Europe, with supporting achievements seen in the solid growth across the commercial portfolio, and in several milestones including two significant new manufacturing agreements with Pfizer, two Orfadin approvals and the granting of a European patent for a new citrate free formulation of Kineret", said Geoffrey McDonough, CEO and Pesident at Sobi.

"During the quarter, we have continued to build momentum for the launch of Elocta and we now have patients using the product in Germany, UK, Ireland, Sweden and in the Middle East. It has also been an exciting quarter for Alprolix with the approval by the European Commission in May. We are very pleased that Alprolix is now commercially available in Germany, and continue to work to make this innovative treatment available as quickly as possible in more countries in Europe in a similar launch sequence as for Elocta."

Financial Summary
Q2 Q2 H1 H1 Full year
Amounts in SEK M 2016 2015 Change 2016 2015 Change 2015
Total revenues1 1,469 764 92% 2,742 1,629 68% 3,228
Gross profit 1,065 482 >100% 2,009 1,001 >100% 2,007
Gross margin 72% 63% 73% 61% 62%
EBITA 550 74 >100% 1,052 247 >100% 433
EBIT (Operating profit/loss) 453 3 >100% 862 105 >100% 146
Profit/loss for the period 265 -2 >100% 567 73 >100% 68
1 Q2 2016 revenues include a one time credit of SEK 386 M relating to first commercial sales of Alprolix. H1 2016 revenues also include the one time credit received in Q1 of SEK 322 M relating to first commercial sales of Elocta.
Outlook 2016 – unchanged

Sobi continues to expect total revenue for the full year to be in the range of SEK 4,800 to 5,000 M. Revenues include one time credits for Elocta of SEK 322 M and for Alprolix SEK 386 M, which do not impact cash. Gross margin is expected be in the range of 68 to 70 per cent.

Sobi will continue to invest in the launches of Elocta and Alprolix, and will also take on incremental costs of SEK 250 – 300 M reflecting its 50 per cent share of Biogen’s ongoing development costs for the products. Sobi will assume these costs when it becomes marketing authorisation holder for Elocta, which occurred 24 March 2016; and for Alprolix expected in the second half of the year. These incremental costs are included in this outlook.

Sobi expects EBITA for the full year to be in the range of SEK 1,200 to 1,300 M.