On September 21, 2016 Nicox S.A. (Euronext Paris: FR0013018124, COX), the international ophthalmic R&D company, reported its financial results for the six months ended June 30, 2016, and provided an update on its activities (Press release, NicOx, SEP 21, 2016, View Source [SID:SID1234515282]).

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"Following the recently completed transaction with GHO Capital involving our European and International commercial assets, we are now well positioned to refocus our resources on our promising R&D pipeline as we await FDA approval decisions for latanoprostene bunod and AC-170" commented Michele Garufi, Chairman and Chief Executive Officer of Nicox. "This transaction allows us to reduce our infrastructure costs by €6 million (approximately 66%) and, together with the recent financing of €18 million, to accelerate the development of our proprietary pipeline assets, including obtaining clinical proof-of-concept data for NCX 4251 and NCX 470 by the end of 2018."

Operational highlights for the six months to June 30, 2016 and post-reporting period

In August 2016, Nicox completed the transfer of its European and International commercial operations to a new pan-European ophthalmic specialty pharmaceutical company led by GHO Capital. The transaction was valued at up to €26 million and the Company received a €9 million upfront cash payment upon closing of the transaction and a minority stake in the new company.
In July 2016, Nicox completed a financing through a reserved capital increase of ordinary shares of the Company with a specific category of investors. Gross proceeds from the financing were €18 million.
On July 21, 2016, latanoprostene bunod licensee Bausch + Lomb (a wholly-owned subsidiary of Valeant Pharmaceuticals International, Inc.) announced its receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for the use of latanoprostene bunod for the treatment of glaucoma. The CRL cited concerns pertaining to a Current Good Manufacturing Practice (CGMP) inspection at Bausch + Lomb’s manufacturing facility in Tampa, Florida. The FDA’s letter did not identify any efficacy or safety concerns with respect to the latanoprostene bunod NDA or additional clinical trials needed for the approval of the NDA. Bausch + Lomb is currently working with the FDA to resolve and address the CGMP concerns. Latanoprostene bunod has the potential to generate significant revenue for Nicox through milestones (up to $132.5 million net, mainly on commercial sales targets) and royalties (potential net tiered royalties on sales from 6% to 11%).
In June, Nicox announced that the U.S. FDA had accepted the Company’s NDA for AC-170, a novel, proprietary eye drop formulation of cetirizine, targeting the treatment of ocular itching associated with allergic conjunctivitis. The FDA also granted Priority Review and assigned a Prescription Drug User Fee (PDUFA) goal date of October 18, 2016 (contingent upon the information and data provided by Nicox during the review period). Approval of the AC-170 NDA prior to December 1st, 2016 will trigger a milestone payment of $35 million in Nicox shares to former Aciex shareholders or $10 million in Nicox shares if approval of the NDA is received after this date. Nicox is currently in partnering discussions in the United States for this program.
Other Updates

• Philippe Masquida, EVP, Managing Director European & International Operations, will be leaving the company at the end of September 2016, following the successful completion of the transfer of the European and International commercial operations. Nicox sincerely thanks Philippe for his years of service and his significant contributions to the commercial business.

First-half financial summary2

The Group’s revenues have been retreated following reclassification of the European commercial business as Discontinued Operations. For information, the European and International product sales of the operations transferred to VISUfarma BV, the new Group led by GHO Capital, were €7.1 million over the first half of 2016, an increase of 50% compared to the same period in 2015.

Excluding Discontinued Operations the operating expenses for the period were €12.0 million compared to €8.8 million for the six months to June 2015. The increase in operating expenses over the period is mainly explained by costs related to the submission of the AC-170 NDA.

Excluding the Discontinued Operations, the Group recorded a net loss of €12.3 million as of June 2016, compared to a net loss of €10.1 million3 at the same date in 2015.

The Group had cash, cash equivalents and financial instruments of €12.3 million as of June 30, 2016, compared to €29 million on December 31, 2015; however, considering the financing in July of this year of €18 million gross and the €8.9 million cash payment following the transfer of the European commercial operations to VISUfarma BV, our unaudited cash, cash equivalents and financial instruments at August 31, 2016 are estimated at €34.1 million.

The half-yearly financial report will be available in French by the end of September 2016 on Nicox’s website www.nicox.com in the section Investor Information > Regulated information > Financial Information.

The procedures relating to the limited review of the interim financial statements have been carried out. The limited review report will be issued after the finalisation of the procedures required for the publication of the-first half financial report.

On September 21, 2016 Peregrine Pharmaceuticals, Inc. (NASDAQ:PPHM) (NASDAQ:PPHMP), a biopharmaceutical company committed to improving patient lives by manufacturing high quality products for biotechnology and pharmaceutical companies and advancing its proprietary R&D pipeline, reported that top-line data from the Phase III SUNRISE trial of bavituximab in patients with previously treated locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) will be presented as a late-breaking oral presentation at the upcoming European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Congress (Press release, Peregrine Pharmaceuticals, SEP 21, 2016, View Source [SID:SID1234515262]). Presented data will include a biomarker in the SUNRISE trial that correlated with a statistically significant improvement in overall survival for patients treated with bavituximab in combination with docetaxel compared to patients treated with docetaxel alone. Peregrine will file a new patent application directed to the use of the biomarker prior to the presentation of results at the ESMO (Free ESMO Whitepaper) 2016 Congress, which is being held October 7-11, 2016 in Copenhagen, Denmark.

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Details of the Phase III SUNRISE trial data presentation are as follows:
Presentation Number: LBA45
Presentation Title: Top-line results from SUNRISE: A Phase III Randomized Double-Blind, Placebo-Controlled Multicenter Trial of Bavituximab Plus Docetaxel in Patients with Previously Treated Stage IIIb/IV Non-Squamous Non-Small Cell Lung Cancer
Date: Monday, October 10, 2016
Time: 9:15 a.m. (local time in Copenhagen)

"I want to start by thanking the patients, investigators and scientists involved in the SUNRISE trial that have made possible the continuing collection and analysis of important data from the study. While the current interim analysis is still ongoing, it is exciting to already see that a biomarker associated with positive outcomes for patients receiving the combination of bavituximab with docetaxel has been identified. In the evolving cancer therapeutic space, biomarker identification is playing an increasingly critical role in guiding clinical development strategies and trial designs," said Steven W. King, president and chief executive officer of Peregrine. "It is important to note that we are undertaking a broad biomarker analysis effort as part of the SUNRISE trial and this initial set of data is just the first of what we hope will be several findings that will help guide the bavituximab clinical program going forward. We look forward to presenting results from this ongoing analysis effort at the ESMO (Free ESMO Whitepaper) 2016 Congress, as well as other medical conferences as the additional data becomes available."

The primary goal of the biomarker analysis is to identify a biomarker profile for patients that receive the most benefit from a bavituximab-containing therapeutic regimen. As specified in the study protocol, thousands of patient samples were collected to potentially identify biomarkers associated with improved outcome for patients receiving bavituximab. Peregrine is in the process of filing a new patent application directed to the use of the initial biomarker discovery which will be presented at the ESMO (Free ESMO Whitepaper) 2016 Congress. Additional patient sample testing and analysis is ongoing and may result in other biomarkers of importance.

Bavituximab is an investigational chimeric monoclonal antibody that targets phosphatidylserine (PS). Signals from PS inhibit the ability of immune cells to recognize and fight tumors. Bavituximab is believed to override PS mediated immunosuppressive signaling by blocking the engagement of PS with its receptors as well as by sending an alternate immune activating signal. PS targeting antibodies have been shown to shift the functions of immune cells in tumors, resulting in multiple signs of immune activation and anti-tumor immune responses.

Peregrine’s clinical development strategy for bavituximab is currently focused on small, early-stage proof-of-concept trials evaluating the drug in combination with other cancer treatments. The intent behind this strategy is to control research and development costs, while continuing to generate clinical data to further validate bavituximab’s combination potential that will be critical to bringing onboard a partner to help advance the program.

On September 21, 2016 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapeutics, reported that the Company has entered into a collaboration agreement with Foundation Medicine, Inc. to support patient identification and enrollment for Epizyme’s ongoing Phase 2 clinical trial of tazemetostat in patients with non-Hodgkin lymphoma (NHL) (Press release, Epizyme, SEP 21, 2016, View Source [SID:SID1234515264]). Foundation Medicine’s SmartTrials Precision Enrollment Program and FoundationOne Heme panel will assist in identifying a population of individuals with NHL who harbor EZH2 mutations and constitute specific cohorts in the Epizyme trial.

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Tazemetostat is Epizyme’s oral, first-in-class EZH2 inhibitor being investigated in multiple ongoing clinical trials. Early clinical data suggest tazemetostat has encouraging clinical activity and a favorable safety profile in patients with relapsed and refractory NHL.

"This agreement exemplifies our commitment to execute enrollment in our ongoing Phase 2 NHL study, particularly patients with EZH2 mutations," said Robert Bazemore, president and chief executive officer of Epizyme. "We are pleased to partner with Foundation Medicine, an industry leader and innovator in molecular information and comprehensive genomic profiling, to advance our tazemetostat clinical program and accelerate identification of patients who may benefit from this therapy."

FoundationOne Heme is Foundation Medicine’s validated, comprehensive genomic profiling assay, specifically designed for hematological malignancies and sarcomas, which identifies the unique genomic alterations in an individual’s cancer and matches the findings with relevant targeted therapy and clinical trial treatment options for patients. Through this collaboration, Foundation Medicine’s SmartTrials Precision Enrollment program will identify individuals across the U.S. living with NHL who harbor EZH2 mutations as detected by FoundationOne Heme in the course of routine clinical care. The treating physicians of these identified patients will be contacted and informed of Epizyme’s ongoing Phase 2 study of tazemetostat, including relevant details about the trial, to assist the physician in evaluating tazemetostat as a potential treatment option.

About the Tazemetostat Clinical Trial Program
Tazemetostat, a first-in-class EZH2 inhibitor, is currently being studied in ongoing Phase 2 programs in patients with NHL. It is also being studied in adult and pediatric patients with certain genetically defined solid tumors, including INI1-negative and SMARCA4-negative tumors and synovial sarcoma, and in patients with mesothelioma.

The company plans to initiate additional clinical trials of tazemetostat in 2016, including a combination with R-CHOP in collaboration with the Lymphoma Study Association and a combination with Tecentriq (atezolizumab) in collaboration with Genentech, a member of the Roche Group.

About SmartTrials Precision Enrollment Program and FoundationOne Heme
Foundation Medicine’s SmartTrials Precision Enrollment program leverages the company’s unique combination of broad collaborations with biopharma, industry-leading clinical assays and the FoundationCORE genomic knowledgebase with more than 90,000 real-world genomic profiles to overcome the major roadblocks to today’s clinical trial system, namely patient identification and access to trials.

FoundationOne Heme, an integrated DNA/RNA platform using targeted hybrid-capture next-generation sequencing, is a comprehensive genomic profile developed to detect genomic alterations with therapeutic relevance, including single-nucleotide substitutions, insertions and deletions, copy number alterations and rearrangements, which are not fully evaluated using conventional diagnostic assays. FoundationOne Heme simultaneously detects all classes of genomic alterations in the DNA of 405 cancer-related genes and employs RNA sequencing across 265 genes to capture a broad range of gene fusions, a type of alteration that is a common driver of hematologic cancers. It is designed to provide physicians with clinically actionable information to guide treatment options for patients based on the genomic profile of their cancer.

On September 20, 2016 Bristol-Myers Squibb Company (NYSE:BMY) and LabCentral, an innovative, shared laboratory space designed as a launch pad for life-sciences and biotech startups, reported that PanTher and Suono Bio are the winners of Bristol-Myers Squibb’s Golden Tickets for LabCentral (Press release, Bristol-Myers Squibb, SEP 20, 2016, View Source [SID:SID1234515233]). As a platinum sponsor of LabCentral, Bristol-Myers Squibb can select up to two innovative life-sciences and biotech startup companies per year of active sponsorship for "Golden Tickets," which underwrite the cost of one lab bench for one year in LabCentral’s Kendall Square facility.

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"PanTher and Suono Bio are working to deliver innovative technologies that have the potential to impact patients with serious diseases, and we’re pleased that the Golden Tickets will enable them to advance their research," said Carl Decicco, Ph.D., Head of Discovery at Bristol-Myers Squibb. "Our sponsorship of LabCentral, and the awarding of Golden Tickets to these two companies, align with our strategy to encourage scientific innovations in our disease areas of focus, from academia through early development."

"Bristol-Myers Squibb has made an excellent selection of its first two Golden Ticket winners – we know them both well," commented LabCentral Co-Founder and President Johannes Fruehauf, M.D., Ph.D. "Both have the potential to make major inroads against devastating diseases using novel approaches to drug delivery. We are excited to have them join in the vibrant ecosystem of the LabCentral community and look forward to watching as these companies move down the path toward success."

A pre-clinical-stage company, PanTher Therapeutics is working to revolutionize the treatment of inoperable, locally advanced solid tumors − studying the direct delivery of existing, already proven chemotherapy agents directly onto the tumor for consistent, slow release over time. The company designed its novel delivery method to potentially eliminate the toxicity and debilitating side effects that chemo agents can produce when delivered systemically through traditional IV or oral administration. Its first potential indication is pancreatic cancer, a particularly lethal disease that affects more than 53,000 Americans annually, where excruciating symptoms arise from the primary mass invading nearby vital organs. By changing the route of administration to target just the tumor, PanTher is designed to increase the amount of drug reaching the intended destination with the aim to enhance therapeutic efficacy. Eliminating adverse outcomes may also help to lower healthcare costs. Pancreatic cancer accounts for about three percent of all cancers in the U.S. and about seven percent of cancer deaths. A privately held company, PanTher is completing pre-clinical studies prior to initiating human trials and exploring opportunities for partnerships to expand its product pipeline.

Suono Bio, a preclinical stage company, has developed breakthrough technology − the "SuonoCalmTM" system − designed to potentially enable ultra-rapid delivery of therapeutics across tissues, including the gastrointestinal (GI) tract. Preclinical studies have demonstrated that the ultrasound-based technology can deliver small molecules, proteins, and nucleic acids locally and systemically, validating further study of the SuonoCalm system. Designed as an easy-to-use device to enable patients to self-administer medication at home, the SuonoCalm technology may also be applicable to a broad set of conditions outside of the GI tract.

On September 20, 2016 Oncology Venture Sweden AB (OV:ST) and Cadila Pharmaceuticals Ltd., Ahmedabad, State of Gujarat, India reported the entering of a co-development agreement to develop the anticancer product LiPlaCis in combination with its Drug Response Predictor – DRP (Press release, Oncology Venture, SEP 20, 2016, View Source;and-its-drug-respo,c2082912 [SID1234561591]). The aim is to evaluate the LiPlaCis efficacy in several different indications and perform a randomized phase 3 trial as corner stone and part of the data package study for marketing approval by the FDA, EMA, CDSCO (Central Drugs Standard Control Organization of India). The mutual goal is to sell or out license the product in combination with its Companion Diagnostic (DRP) when the clinical benefit has been documented. Cadila will perform four (4) phase 2 and one pivotal, randomized phase 3 trial over a period of three years. Initiation of individual studies will be announced separately. Cadila will invest in kind in research and drug development activities in 310 cancer patients and DRP screening of more than 1400 patients. In the consortium of owners now including Cadila Pharmaceuticals, LiPlasome, MPI and Oncology Venture – Oncology Venture owns 29% of the total value of the LiPlaCis project after Phase 3. Cadila has commercialization rights in India, Russia, Africa and South East Asia (ASEAN countries only). Oncology Venture has the commercialization rights in America, Europe and China and RoW. Oncology Venture is responsible for the manufacturing and will provide the product. Estimated costs for product in 2017-2018 is 0,6 MUSD. Cadila will in collaboration with an expert team in Oncology Venture set up a laboratory for the tissue handling in India. The DRP analysis will be paid by Cadila and Oncology Venture will provide the DRP evaluations. When developed, the parties may choose to market themselves in their own territories or out-license or sell to a third party. The potential of LiPlaCis sales in the two major indications alone in breast cancer in USA and EU is in excess of 700 MUSD annually and if successful LiPlaCis would compete in a market which currently has a value in excess of 5 billion USD in lung cancer in Europe and USA.

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Around 310 cancer patients will according to the deal partake in clinical trials at a FDA and EMA quality level.
The agreement is entered between Cadila Pharmaceuticals Ltd. and Oncology Venture ApS which is 100% owned by Oncology Venture Sweden AB. Cadila Pharmaceuticals Ltd. is one of the largest privately held pharmaceutical companies in India.

"Cadila is one of the largest privately held pharma companies in India. The transformational partnership with Cadila places Oncology Venture in another league. Together with Cadila we aim to take LiPlaCis and its Drug Response Predictor – DRP – through a strong and focused development program with the goal of receiving marketing approval. We will together with Cadila who invests heavily as an ‘in kind’ investment test LiPlaCis in four promising indications: breast, head & neck, skin and esophageal cancer which gives the drug a really good chance to benefit patients who are likely to respond," says Peter Buhl Jensen, M.D., CEO of Oncology Venture. "In this partnership OV has the opportunity to build a much larger value instead of selling it outright. Cisplatin is one of the most used drugs in cancer treatment and the potential sales with the improved LiPlaCis formulation is huge," Buhl Jensen further comments.

On this occasion, Dr. Rajiv Modi, Chairman and Managing Director, Cadila Pharmaceuticals Ltd., said, "Cadila Pharmaceuticals believes in providing world-class healthcare products to improve quality of life of patients. This agreement with Oncology Venture is an affirmation of our commitment to develop novel treatment for those diseases in the realm of unmet medical needs. Cancer affects millions of people around the globe. We look forward to developing the product to benefit millions of patients who are affected by the deadly disease."
 
The Partnership deal
The deal with Cadila Pharmaceuticals will finance and perform studies in a total of 310 cancer patients with highest likelihood of sensitivity to LiPlaCis. The deal covers the following:
1. Screening by the use of the LiPlaCis-DRP of 1 250 metastatic breast cancer patients to identify 250 patients with high likelihood to respond to LiPlaCis treatment and perform a randomized phase 3 trial in these 250 patients comparing standard therapy with LiPlaCis.
2. Through Cadilas strong network and Cadilas CRO run four (4) clinical Phase 2 trials in 20 patients (out of 100 screened) with Head & Neck cancer, 20 prostate cancer patients (out of 100 screened), and 10 skin cancer patients and 10 esophagus cancer patients – the two latter indications are in a high frequency sensitive to cisplatin – of which LiPlaCis is an improved formulation – why the patients will not be screened.

Cadila has commercialization rights in India, Russia, Africa and South East Asia (ASEAN countries only), Oncology Venture has the commercialization rights to America, Europe and China and RoW. Oncology Venture will be responsible for the manufacturing and pay for manufacturing of the product. Estimated costs for product in 2017-2018 is 0,6 MUSD. In the consortium of owners now including Cadila Pharmaceuticals, LiPlasome, MPI and Oncology Venture – Oncology Venture owns 29% of the total value of the LiPlaCis project after Phase 3. When developed parties may choose to market themselves in their own territories or out license or sell to a third party.

About the LiPlaCis license from LiPlasome Pharma and the DRP license from MPI
Oncology Venture has in-licensed LiPlaCis from LiPlasome Pharma and the LiPlaCis DRP from MPI and has now entered a development partnership with Cadila Pharma. The financial impact is as follows: In the consortium of owners now including Cadila Pharmaceuticals, LiPlasome, MPI and Oncology Venture – Oncology Venture owns 29% of the total value of the LiPlaCis project after Phase 3.OV pays for the manufacturing of the product. Oncology Venture did not pay any upfront payment to LiPlasome but pays 2×9 MUSD in sales milestones to LiPlasome once LiPlaCis is commercialized either via a partner or sold on the market. (please see latest company prospectus).Cost of clinical trials in oncology all phases per patient 59.500 USD (PhRMA 2013).
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