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.

On July 15, 2016 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP) reported the following statement regarding the Company’s ongoing multicenter Phase 1 study of Ad-RTS-hIL-12 + orally administered veledimex in recurrent or progressive glioblastoma (GBM) or grade III malignant glioma:

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"This Phase I study is being conducted in late-stage, recurrent GBM, so these patients are all, unfortunately, medically fragile. The first two patient deaths, which occurred 6.7 months and 3.9 months after treatment, were unrelated to study drug (Press release, Ziopharm, JUL 15, 2016, View Source [SID:1234513900]). A third death has just been reported to us and we are collecting and analyzing information in order to properly and timely report it to the FDA. The cause of death is intracranial hemorrhage, which occurred some time after the patient had been discharged from the treating center. This is an isolated case, and there have been no reported related instances of brain hemorrhage in any pervious cohort or prior studies with Ad-RTS-hIL-12 + veledimex. Enrollment remains open in the study, and we will be discussing with our Safety Review Committee the appropriate course of action. For patients who have experienced multiple recurrences, as these patients have, prognoses are particularly poor. Median follow up in the first dose cohort from our study is now 8 months, in a population with an expected overall survival of 3 to 5 months for patients that have failed temozolomide and bevacizumab, or equivalent salvage chemotherapy. For the patients that remain in follow up in this Phase I study, we believe that preliminary overall survival remains encouraging. The Company expects to provide an update once a course of action has been determined."

About Glioblastoma

Glioblastoma is an aggressive primary brain tumor affecting approximately 74,000 people worldwide each year.i, ii Recurrent glioblastoma is an aggressive cancer with one of the lowest 3-year survival rates, at 3%, among all cancers.iii For patients who have experienced multiple recurrences the prognosis is particularly poor, with a median overall survival (OS) of 6-7 months, while OS in patients that have failed temozolomide and bevacizumab, or equivalent salvage chemotherapy, is approximately 3-5 months.iv, v

On July 14, 2016 Panther Biotechnology, Inc. (OTC PINK: PBYA) (Panther), a biotechnology company specializing in the development of enhanced therapeutics for the treatment of neoplastic and autoimmune disorders reported that it has initiated the formal drug development process for its lead compound, Transferrin Doxorubicin (TRF-DOX) (Press release, Panther Biotechnology, JUL 14, 2016, View Source [SID1234517416]). In April 2015, Panther entered into a definitive agreement with privately held Faulk Pharmaceuticals, Inc. to acquire Faulk’s pharmaceutical technology assets which included TRF-DOX. The transaction provided Panther with a proprietary, multi-nationally patent protected, ligand-drug conjugate technology platform as well as a pipeline of drug product candidates that address unmet medical needs in oncology, autoimmune, antiviral and other disease indications. Panther was particularly interested in the TRF-DOX conjugate as it has shown promising safety and preliminary efficacy in a randomized controlled study of ovarian cancer outside the US.

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Panther has executed an agreement with Dr. Patricia D. Williams of IND Directions, LLC in Washington DC to lead all aspects of the drug development process for TRF-DOX including regulatory, preclinical, manufacturing and clinical development activities. Dr. Williams has over 30 years of experience designing and implementing high quality drug development programs for small molecules, biologics, and gene therapies. Dr. Williams has held various positions of increasing responsibilities at major pharmaceutical and biotechnology companies (Bristol-Myers, Eli Lilly, American Cyanamid, Ligand, Biochem Pharma) as well as contract research and consulting organizations (SRA Life Sciences, TherImmune, Gene Logic and Summit Drug Development Services). In addition, Dr. Williams has already worked on TRF-DOX with Faulk Pharmaceuticals in the past. Teams under Dr. Williams’ leadership have advanced over 50 small molecules and biologics into clinical development. Under Dr. Williams’ leadership, Panther has initiated the preparation of a formal Pre-Investigational New Drug (IND) submission to the US Food and Drug Administration (FDA). This step will result in FDA’s input into Panther’s Phase II clinical plans with TRF-DOX as well as the preclinical and manufacturing data that will support clinical trials with TRF-DOX.

"We are very excited to work with Dr. Williams to initiate clinical development of the Faulk Pharmaceutical technology, which we see as complementary to our efforts to develop and commercialize innovative pharmaceutical approaches for the treatment of cancer. Not only does Dr. Williams provide enormous experience, her previous work on the TRF-DOX will be invaluable to our efforts," stated Evan Levine, Chief Executive Officer of Panther. "The goal of this next phase is to present a clinical plan to the FDA that has the opportunity to generate a statistical signal over the reference drug and progress Transferrin Doxorubicin towards commercialization. We will continue to inform the public as critical milestones are achieved."

TRF-DOX is a combination of the iron-binding glycoprotein, transferrin, and Doxorubicin resulting in targeted delivery to tumors with the reduction of serious side effects. TRF-DOX leverages the targeting ability of the plasma glycoprotein transferrin to deliver a powerful chemotherapeutic payload to cancerous cells which have elevated levels of transferrin receptors. In vitro assays demonstrate growth inhibition of cancer cells that are resistant to other chemotherapies including Doxorubicin itself. Cytotoxicity studies demonstrate that a doxorubicin dose reduction of ten to one hundred-fold kills all cancer cells in multiple cancer types. In vivo studies demonstrate that TRF-DOX selectively binds tumors, inhibits tumor growth better than unmodified Doxorubicin, and increases survival. This improved therapeutic index suggests that further improvements in efficacy without added toxicity can be achieved.

On July 14, 2016 Peregrine Pharmaceuticals, Inc. (NASDAQ:PPHM) (NASDAQ:PPHMP), a biopharmaceutical company committed to improving patient lives by delivering high quality biological products through its contract development and manufacturing organization (CDMO) services and by advancing its novel R&D pipeline, reported that the company has entered into an exclusive licensing agreement with University of Texas (UT) Southwestern Medical Center for a novel exosome technology that has potential application as a simple blood test to detect or monitor cancer (Press release, Peregrine Pharmaceuticals, JUL 14, 2016, View Source [SID:1234513872]). The company intends to develop a novel cancer test utilizing internal expertise and then pursue revenue-generating partnering opportunities at an early stage of development.

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Tumor exosomes represent small pieces of tumor cells that are released into the blood as tumors grow. Tumor derived exosomes have phosphatidylserine (PS) on their surface as a detectable marker. It is believed that even small tumors begin to release PS-positive exosomes and thus the ability to detect these exosomes in the blood may be an indicator of the presence of a tumor.

The licensing agreement is the result of the long-standing sponsored research agreement between Peregrine and UT Southwestern focused on PS, a highly immunosuppressive signaling molecule. The new technology licensed by Peregrine relates to assays that are able to detect small amounts of PS-exosomes in a patient blood sample as a way to potentially detect cancer at a very early stage of development. Preliminary studies have demonstrated that the levels of PS-positive exosomes present in the blood of cancer patients are higher than levels found in the blood of healthy volunteers. Furthermore, study findings also suggest that there is a correlation between the level of PS-positive exosomes detected in the blood of cancer patients and disease burden.

"We are excited to enter into this licensing agreement with our long-term collaborators at UT Southwestern. This technology offers a promising product development opportunity and aligns directly with the company’s expertise with our proprietary PS-targeting platform and our longstanding CDMO capabilities around the development, qualification, and validation of in vitro analytical assays. As such, there are significant opportunities to use this technology as both a complementary tool in bavituximab’s ongoing development, as well as more broadly as the basis for novel cancer detection and monitoring tests that can be the focus of partnering efforts," said Jeff T. Hutchins, Ph.D., Peregrine’s vice president, preclinical research. "It is important to note that this development program will require minimal capital investment and has the potential to create significant value over the next 18 months, including potential partnering opportunities. As a result, we feel that today’s licensing deal provides yet another important driver in our ongoing efforts to achieve profitability."

Together, the Peregrine and Avid Bioservices teams have the existing infrastructure, staff and expertise to develop, optimize and validate a functional assay capable of detecting PS-positive exosomes from a blood sample. Given the company’s extensive experience in developing assays of this type, Peregrine does not anticipate the need to add personnel or any specialized equipment for this project. The company intends to establish clinical proof-of-concept for the test and expects to initiate partnering discussions for the program in 2017.

"One of the most exciting aspects of this technology is the potential synergy that it offers with our ongoing bavituximab clinical development program. Through our ongoing work with bavituximab, we have gained significant understanding of PS-mediated immunosuppression in cancer," said Joseph Shan, MPH, vice president, clinical and regulatory affairs of Peregrine. "The availability of a PS-specific biomarker which can be implemented in our planned future bavituximab clinical trials aligns nicely with our refocused bavituximab development strategy aimed at generating the most meaningful data possible from small, early stage clinical trials to support partnering efforts."

On July 14, 2016 Provectus Biopharmaceuticals, Inc. (NYSE MKT: PVCT, www.provectusbio.com), a clinical-stage oncology and dermatology biopharmaceutical company ("Provectus" or "The Company"), reported that it has formed an Australian subsidiary, Provectus Biopharmaceuticals Australia Pty Ltd. In addition, the Company is opening a Sydney office in New South Wales (Press release, Provectus Pharmaceuticals, JUL 14, 2016, View Source [SID:1234513875]).

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Peter Culpepper, Interim CEO, stated, "The creation of an Australian entity is a fundamental part of our plans for extended global reach in conjunction with planned partnering for commercialization of PV-10."

He noted, "Provectus has already been very active in Australia for years because of our research into PV-10 as an investigational treatment for melanoma. In fact, we began our phase 1 study of PV-10 in 2005 at the Sydney Melanoma Unit in North Sydney and the Newcastle Melanoma Unit in Waratah, both in New South Wales. Since then, we have also worked with the Princess Alexandra Hospital in Brisbane, Queensland, the Royal Adelaide Hospital in Adelaide, South Australia, and the Peter MacCallum Cancer Centre in Melbourne, Victoria."

Culpepper concluded, "With a subsidiary in Australia, we are bringing our corporate structure in line with our scientific work. Our research and development program has been international from the very beginning, and now, Provectus is an international company. The new unit should make it easier to work with the Australian regulatory authorities, and having an office in the region may facilitate our work in Asian markets as Sydney is just two hours ahead of Beijing, Hong Kong and Singapore. If and when PV-10 receives approval in Australia and other nations in the region, we will have pre-positioned ourselves to develop a sales and marketing force."