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ATB-346 Shows Promising Results in Experimental Melanoma

On September 22, 2016 Antibe Therapeutics Inc. ("Antibe") (TSXV: ATE, OTCQX: ATBPF), a commercial-stage pharmaceutical growth company, reported anti-cancer potential of its lead drug, ATB-346 (Press release, Antibe Therapeutics, SEP 22, 2016, View Source [SID:SID1234515305]). In a separate study earlier this year, ATB-346 was shown to be very effective in reversing colon and intestinal tumour growth in mice. Further to this study, a new publication has shown promising results in the chemoprevention and treatment of melanoma in mice. Melanoma is one of the most drug-resistant and invasive types of cancer, and both the incidence and mortality rates are increasing.

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"Although we remain focused on our ongoing human studies to advance ATB-346 as a treatment for pain and inflammation, we have compelling data from studies of colon cancer and now melanoma," commented John Wallace, Chief Scientific Officer of Antibe. "We are encouraged by these preliminary data and plan to continue this research in support of the potential advancement of ATB-346 into selected cancer indications."

In the new publication, Dr. Angela Ianaro’s laboratory at the University of Naples (Italy), in collaboration with Dr. Wallace, examined the possibility that ATB-346 may have greater beneficial effects in terms of reducing melanoma than naproxen, the most-prescribed nonsteroidal anti-inflammatory drug ("NSAID") in the USA. NSAIDs have been shown to reduce tumour growth, but they also cause significant gastrointestinal bleeding. In contrast, ATB-346 has been shown to be GI-safe in animal studies.

The effects of ATB-346 were first compared to naproxen in an in vitro melanoma model, and was found to be much more effective. The investigators then implanted melanoma cells into mice, and monitored the growth of tumours. Treatment with naproxen had a modest beneficial effect, reducing tumour volume by 23% and tumour weight by 20%. In contrast, treatment with an equivalent dose of ATB-346 was 3-times more effective, reducing tumour volume by 69% and tumour weight by 61%. Mechanistic studies confirmed that ATB-346 was more effective in promoting death (apoptosis) of cancer cells.

The article was published online in the September issue of the journal "Pharmacological Research" and can be found at: www.sciencedirect.com/science/article/pii/S1043661816304467

Syros Announces First Patient Enrolled in Phase 2 Clinical Trial of SY-1425 in Genomically Defined Patients with Acute Myeloid Leukemia or Myelodysplastic Syndrome

On September 22, 2016 Syros Pharmaceuticals (NASDAQ: SYRS) reported that the first patient has been dosed in the Phase 2 clinical trial of its lead drug candidate, SY-1425, a first-in-class selective retinoic acid receptor alpha (RARα) agonist, in genomically defined subsets of patients with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) identified using a novel biomarker discovered by its gene control platform (Press release, Syros Pharmaceuticals, SEP 22, 2016, View Source [SID:SID1234515304]).

"This is an important milestone for Syros and for patients," said David A. Roth, M.D., Chief Medical Officer of Syros. "There has been little improvement in the treatment of AML and MDS for the past 20 years, and survival rates for these patients lag behind many other blood cancers. Treatment with SY-1425 represents a promising therapeutic strategy for subsets of AML and MDS patients with a novel biomarker that we discovered using our gene control platform. Our pioneering approach is designed to advance a new wave of medicines to control the expression of disease-driving genes in genomically defined subsets of patients to provide them with a profound and durable clinical benefit. Our goal is to rapidly advance this first-in-class therapy for these currently underserved patients."

Using its gene control platform, Syros discovered subsets of AML and MDS patients whose tumors have a highly specialized regulatory region of non-coding DNA, known as a super-enhancer, that is associated with the RARA gene, which codes for the RARα transcription factor. The super-enhancer is believed to lead to over-production of the RARα transcription factor, locking cells in an immature, undifferentiated and proliferative state. Syros further investigated this unique biology directly in patient tissues and conducted preclinical studies showing that the RARA super-enhancer is predictive of response to treatment with SY-1425 in preclinical models of AML. Based on that data, Syros is implementing a biomarker strategy for its Phase 2 trial that selects a subset of approximately 25 percent of AML and MDS patients who may respond to treatment with SY-1425.

"The prognosis for these patients is poor, and targeted approaches like SY-1425 offer hope for much-needed new therapies that attack the underlying biology of the disease and hopefully allow patients to live longer without the toxicities of traditional chemotherapy," said Rachel J. Cook, M.D., M.S., assistant professor of medicine at Oregon Health & Science University and an investigator in the trial. "We’re pleased to have enrolled the first patient in this clinical trial and look forward to further investigating SY-1425 for this newly identified subset of AML and MDS patients."

The Phase 2 clinical trial is a multi-center, open-label trial exploring safety and efficacy in relapsed or refractory AML or high-risk MDS patients who have been prospectively selected using the Company’s biomarker strategy. The trial is expected to enroll approximately 40 patients, and the primary endpoint of the trial will be overall response rate. The trial will also assess pharmacodynamic biomarkers, duration of response, safety and tolerability, and survival. Additional details about the trial can be found using the identifier NCT02807558 at www.clinicaltrials.gov.

SY-1425 is approved in Japan as Amnolake (tamibarotene) to treat a different form of AML known as acute promyelocytic leukemia (APL), in which it has a well-established efficacy and safety profile. Syros in-licensed SY-1425 to develop and commercialize it in North America and Europe for all cancers.

argenx provides update on lead programs in auto-immune disease and oncology at R&D day in New York

On September 22, 2016 argenx (Euronext Brussels: ARGX), a clinical-stage biopharmaceutical company focused on creating and developing differentiated therapeutic antibodies for the treatment of cancer and severe autoimmune diseases, today provided highlights from its lead pipeline programs, ARGX-113 and ARGX-110, during its R&D in New York City (Press release, arGEN-X, SEP 22, 2016, View Source [SID:SID1234515303]).

"We are very pleased to announce today our strategic plan for driving forward our antibody pipeline through 2017; it includes several important value creating events including two Phase 2 studies in important autoimmune indications for ARGX-113 and two combination studies in serious hematological malignancies for ARGX-110," said Tim Van Hauwermeiren, CEO of argenx. "We’re particularly proud of the speed of our development plan and look forward to continuing to advance our entire pipeline rapidly towards becoming an integrated drug development company."

Members of argenx management as well as guest speakers Dr. James Howard from the University of North Carolina, Dr. Adrian Newland from The Royal London Hospital, and Dr. Owen O’Connor from Columbia University presented the following program highlights:

ARGX-113

ARGX-113 is a potential breakthrough therapy for the treatment of IgG-mediated autoimmune diseases. It is the Fc-portion of an antibody enhanced by argenx proprietary ABDEG technology to block the recycling receptor FcRn leading to fast depletion of IgGs, including autoimmune disease-causing IgG’s. In June 2016, argenx announced favorable safety and tolerability, and efficacy data from its Phase 1 multiple ascending dose study in healthy volunteers. Early pharmacodynamic results showed selective and potent IgG reduction of up to 85%.

Today, argenx announces further results from the Phase 1 study of ARGX-113, across multiple doses and dosing regimens, and its plan to advance ARGX-113 into Phase 2 studies in myasthenia gravis (MG) and immune thrombocytopenic purpura (ITP). argenx plans to open the MG study before the end of the year and the ITP study shortly thereafter.

ARGX-110

ARGX-110 is a SIMPLE Antibody that potently blocks CD70-induced tumor cell proliferation and tumor escape from immune surveillance. It is currently being studied in an ongoing Phase 1b expansion cohort in patients with relapsed/refractory T-cell lymphoma (TCL).

Today, argenx announced new patient anecdotes from its Phase 1b safety expansion cohort showing a favorable safety profile and biological activity in cutaneous T-cell lymphoma (CTCL) patients, evidenced by biomarker data and clinical disease score. The results established the Phase 2 dose and also refined the biomarkers to be used to assess the ongoing effects of ARGX-110 in TCL patients. The Company presented its plan to study ARGX-110 in two combination studies in TCL and AML, both with the respective standard of care. argenx aims to initiate at least one combination study before the end of the year.

Evotec enters into a research collaboration with Inserm in oncology

On September 22, 2016 Evotec AG (Frankfurt Stock Exchange: EVT, TecDAX, ISIN: DE0005664809) reported that the Company has entered into a research collaboration with Inserm Transfert, the private subsidiary of the French National Institute of Health and Medical Research ("Inserm") (Press release, Evotec, SEP 22, 2016, View Source [SID:SID1234515281]).

Evotec has entered into multiple collaborations with top academic institutions in the USA, Germany and the UK. This collaboration with Inserm marks the initiation of the first project under Evotec’s and Sanofi’s Academic Bridge, which was established as a result of the multi-component strategic alliance between the companies effective 01 April 2015. This Academic Bridge aims to accelerate the translation of promising cutting-edge science from French academic institutions into pharmaceutical product candidates. In this joint effort, Evotec scouts and incubates projects generated in France under its EVT Innovate strategy.

A year after initiating the French Academic Bridge, the first collaboration between Evotec and Prof. Gilles Favre’s team from Inserm/the Université Toulouse III – Paul Sabatier/Oncopole de Toulouse has started. The research collaboration’s goal is to characterise and develop new selective modulators of RhoB functions as a promising approach to increase therapeutic options in many cancers with high unmet medical need. RhoB is an exciting oncological target implicated in the control of cellular stress response, migration, tumour neovascularisation and progression.

Dr Pascale Augé, Chairman of the executive management board of Inserm Transfert, commented: "We happily welcome the signing of this partnership with Evotec. It will allow Prof. Favre and his team to further develop their research in the cancer field and move to the industrial development."

Dr Cord Dohrmann, Chief Scientific Officer of Evotec, added: "We are excited to be part of this great research initiative that combines the best-in-class ingredients of what is needed for highly innovative and differentiated drug candidates in oncology. This is Evotec’s first of hopefully many more academic collaborations in France. We are very proud to add Inserm to our academic partners, thereby further broadening our efforts to bridge leading academic science into industry standard drug discovery programmes."

ABOUT THE FRENCH NATIONAL INSTITUTE OF HEALTH AND MEDICAL RESEARCH (INSERM) Founded in 1964, the French National Institute of Health and Medical Research (Inserm) is a public science and technology institute, jointly supervised by the French Ministry of Education, higher Education and Research and the Ministry of Social Affairs, health and women’s rights. The mission of its scientists is to study all diseases, from the most common to the rarest, through their work in biological, medical and public health research. Inserm supports more than 300 laboratories across France. In total, the teams include nearly 15,000 researchers, engineers, technicians and administrative staff, etc. Inserm is a member of the National Alliance for Life and Health Sciences, founded in April 2009 with CNRS, Inserm, the CEA, Inra, INRIA, the IRD, the Pasteur Institute, the Conference of University Presidents (CPU) and the Conference of Chairmen of The Regional and University Hospital Centres. For more information, please go to www.inserm.fr.

ABOUT INSERM TRANSFERT
Founded in 2000, Inserm Transfert SA is the private subsidiary of the French National Institute of Health and Medical Research (Inserm), dedicated to technology transfer (from invention disclosure to industrial partnership). Inserm Transfert also manages European and International research projects, supports large scale projects in epidemiology and public health. Inserm Transfert has run a proof of concept fund since 2009. The company also supports entrepreneurs in the biotech sector, in partnership with Inserm Transfert Initiative. For more information, please go to: www.inserm-transfert.fr.

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]).

"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.