Nicox first half 2016 business and financial update

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

CRT and SV life sciences form Artios Pharma to focus on DNA damage response

On September 21, 2016 CANCER RESEARCH TECHNOLOGY (CRT) – the development and commercialisation arm of Cancer Research UK – and SV Life Sciences have reported the launch of Artios Pharma, a new company formed to develop drugs targeting the DNA damage response in cancer (Press release, Cancer Research Technology, 21 21, 2016, View Source [SID1234523182]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Artios has licensed its two lead DNA damage response programmes from CRT and has signed a non-exclusive research collaboration agreement through which Artios will work with CRT Discovery Laboratories to progress the lead programmes, and discover and develop additional promising drug targets selected from Cancer Research UK’s portfolio of DNA damage repair research.

DNA damage response therapies target the way cancer cells repair damaged DNA. Faults in DNA repair lead to an increased risk of cancer and drive the growth of tumours. Blocking the repair mechanisms on which cancer cells rely has been shown to selectively kill them.

These DNA damage response targeted therapies have the potential to work alone or in combination with chemotherapy, radiotherapy or immunotherapy drugs.

The company’s lead programme targets the pol-theta molecule and builds on cutting-edge cell biology research from Professor Gillies McKenna’s and Dr Geoff Higgins’ laboratories at the Cancer Research UK/MRC Oxford Institute for Radiation Biology.

The pol-theta molecule is thought to control DNA repair processes in certain tumours. Knocking out the target could remove a vital path relied on by the cancer cell, causing it to die. A second highly promising programme against an undisclosed target has also been licensed to Artios.

Artios is actively building a pipeline of promising first-in-class DNA damage response therapies from leading researchers in the field. Cancer Research UK’s extensive research base will provide the foundation for CRT to provide Artios with additional new molecular targets and establish collaborative partnerships with world-class scientists in the DNA damage response field.

Under the terms of the agreements, CRT will receive research funding into its Discovery Laboratories, equity in the company, and be eligible to receive milestone payments and royalties on projects advancing through Artios’ drug pipeline.

Led by SV Life Sciences, Artios has raised series A financing of £25million ($33.2million) from Merck Ventures, Imperial Innovations, Arix Bioscience PLC, CRT Pioneer Fund (managed by Sixth Element) and AbbVie Ventures.

Dr Keith Blundy, CRT’s chief executive officer, said: "We are pleased to have worked with SV Life Sciences to bring together the Cancer Research UK academic network, a portfolio of leading DNA damage response opportunities and the CRT Discovery Laboratories’ drug discovery platform to help build a strong development pipeline for Artios. This exciting development has enabled us to leverage the expertise of the Artios management team and financing from leading venture companies to help establish a company that has the potential to bring real impact to cancer patients."

Kate Bingham, managing partner at SV Life Sciences, said: "Artios represents a unique opportunity to build a world-class DNA damage response pipeline through partnerships with leading DNA repair researchers in the UK and worldwide. We are delighted to have worked together with CRT to form this company and are pleased with the strong investor interest in Artios, reflecting the potential of DNA damage response. With the strength of their management team, Artios has the potential to disrupt the DNA damage response space and provide significant new therapies for cancer patients."

Dr Niall Martin, CEO of Artios Pharma, said: "Targeting the DNA damage response is an exciting and promising field of biology with growing interest following the recent success of PARP inhibitors. DNA damage response drug products have the potential to become established as first-line treatments, either as single agents or for use in combination with many approved therapies. It is an ideal time for Artios to be entering the field as a DNA damage response-focused, independent biotech company. We’re delighted to welcome our world-class investors to the company and to announce our first partnership with Cancer Research Technology."

Exelixis Provides Update on Timing of Key Cabozantinib Clinical Data Presentation at the ESMO 2016 Congress

On September 20, 2016 Exelixis, Inc. (NASDAQ:EXEL) reported an update on the timing of a key data presentation for cabozantinib at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Congress, which is being held October 7-11, 2016 in Copenhagen, Denmark (Press release, Exelixis, SEP 20, 2016, View Source;p=RssLanding&cat=news&id=2204894 [SID:SID1234515256]). Detailed results from CABOSUN, the randomized phase 2 clinical trial of cabozantinib compared with sunitinib in patients with previously untreated advanced renal cell carcinoma (RCC), has been selected for the Presidential Symposium 3 session on Monday, October 10, 2016, starting at 16:30 CEST (local Copenhagen time) / 10:30 a.m. EDT / 7:30 a.m. PDT.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The full logistical details for the CABOSUN data presentation are as follows:

Oral Presentation

[LBA30_PR] "CABOzantinib versus SUNitinib (CABOSUN) as initial targeted therapy for patients with metastatic renal cell carcinoma (mRCC) of poor and intermediate risk groups: Results from ALLIANCE A031203 Trial."

Dr. Toni Choueiri, Director, Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA

Session: Presidential Symposium 3, Monday, October 10, 2016 – 16:30-18:10 CEST, Copenhagen room

Note: This is a National Cancer Institute Cancer Therapy Evaluation Program (NCI-CTEP) study.

The late-breaking CABOSUN abstract was initially slated for an oral presentation at a Proffered Paper session on Saturday, October 8, 2016. Exelixis previously announced that data from the Exelixis-discovered compounds cabozantinib and cobimetinib would be the subject of fifteen presentations at the ESMO (Free ESMO Whitepaper) 2016 Congress. For full details on Exelixis’ presence at the conference, please see the company’s press release issued on August 31, 2016.

Puma Biotechnology Announces U.S. FDA Acceptance of New Drug Application for PB272 (Neratinib) for Extended Adjuvant Treatment of HER2-Positive Early Stage Breast Cancer
Read

On September 20, 2016 Puma Biotechnology, Inc. (NYSE: PBYI), a biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for its lead product candidate PB272 (neratinib) for the extended adjuvant treatment of patients with early stage HER2-overexpressed/amplified breast cancer who have received prior adjuvant trastuzumab (Herceptin)-based therapy (Press release, Puma Biotechnology, SEP 20, 2016, View Source [SID:SID1234515515]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The FDA acceptance of our NDA is an important regulatory milestone," said Alan H. Auerbach, Chief Executive Officer and President of Puma. "Although the use of trastuzumab in the adjuvant setting has led to a reduction in disease recurrence in patients with early stage HER2-positive breast cancer, there remains an unmet clinical need to further reduce the risk of recurrence and improve outcome following trastuzumab therapy. We believe that neratinib may be able to provide this type of improvement to further help patients with this disease. We look forward to working with the FDA during their review of this submission."

The submission is supported by the results of the ExteNET Phase III study, in which treatment with neratinib resulted in a 33% reduction of risk of invasive disease recurrence or death versus placebo (hazard ratio = 0.67, p = 0.009). The 2-year invasive disease free survival (DFS) rate for the neratinib arm was 93.9% and the 2-year invasive DFS rate for the placebo arm was 91.6%. For the pre-defined subgroup of patients with hormone receptor positive disease, the results of the trial demonstrated that treatment with neratinib resulted in a 49% reduction of risk of invasive disease recurrence or death versus placebo (hazard ratio = 0.51, p = 0.001). For the patients with hormone receptor positive disease, the 2-year invasive DFS rate for the neratinib arm was 95.4% and the 2-year invasive DFS rate for the placebo arm was 91.2%.

Results of the study were published online in The Lancet Oncology on February 10, 2016.

The most frequently observed adverse event for the neratinib-treated patients was diarrhea, with approximately 39.9% of the neratinib-treated patients experiencing grade 3 or higher diarrhea (1 patient (0.1%) had grade 4 diarrhea). Patients who received neratinib in the ExteNET trial did not receive any prophylaxis with antidiarrheal agents to prevent the neratinib-related diarrhea. In patients with HER2-positive early stage breast cancer who have previously been treated with adjuvant trastuzumab and received anti-diarrheal prophylaxis with loperamide, interim results of a Phase II study of neratinib monotherapy demonstrated that treatment with prophylactic loperamide reduced the rate of grade 3 or higher diarrhea to between 13.0% and 18.5%.

About ExteNET

The ExteNET trial is a double-blind, placebo-controlled, Phase III trial of neratinib versus placebo after adjuvant treatment with trastuzumab (Herceptin) in women with early stage HER2-positive breast cancer. The trial randomized 2,840 patients in 41 countries with early-stage HER2-positive breast cancer who had undergone surgery and adjuvant treatment with trastuzumab. After completion of adjuvant treatment with trastuzumab, patients were randomized to receive extended adjuvant treatment with either neratinib or placebo for a period of one year. Patients were then followed for recurrent disease, ductal carcinoma in situ (DCIS), or death for a period of two years after randomization in the trial. The primary endpoint of the trial was invasive DFS.

Mirna Therapeutics Halts Phase 1 Clinical Study of MRX34

On September 20, 2016 Mirna Therapeutics, Inc. (Nasdaq:MIRN), a clinical stage biopharmaceutical company, reported its decision to close the ongoing Phase 1 study of MRX34, its investigational microRNA therapy for multiple cancers (Press release, Mirna Therapeutics, SEP 20, 2016, View Source [SID:SID1234515258]). The Company voluntarily halted enrollment and dosing in the clinical study following multiple immune-related severe adverse events (SAE) observed in patients dosed with MRX34 over the course of the trial.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Patient safety is the primary objective of our MRX34 Phase 1 clinical trial," said President and CEO Paul Lammers, M.D., M.Sc. "We made the difficult decision to close the study after a fifth, immune-related serious adverse event was recently reported by one of our clinical sites. This patient experienced severe (Grade 4) cytokine release syndrome and is undergoing treatment. We have notified the U.S. FDA and the Korean FDA of our decision and are in the process of closing the trial."

Mirna also announced that it will not be initiating a translational medicine study of MRX34 in melanoma patients, planned to begin later this year. The Company will be further analyzing its full preclinical and clinical data set, and will discuss with its advisors, as well as the FDA, possible future development of MRX34 and will provide updates when appropriate.