On January 12, 2018 Ipsen (Euronext: IPN; ADR: IPSEY) reported the appointment of Richard Paulson as Executive Vice-President and Chief Executive Officer of Ipsen North America, responsible for all commercial operations throughout the region. Mr Paulson will assume oversight of all areas supporting the North American business effective from February 5th (Press release, Ipsen, JAN 12, 2018, View Source [SID1234523076]). The role reports directly to David Meek, CEO of Ipsen, and Mr Paulson will be a member of the Ipsen Executive Leadership Team. He joins Ipsen from Amgen where he most recently served as Vice-President and General Manager of the Oncology Business Unit.

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David Meek, Ipsen’s CEO, commented, "We are extremely delighted to welcome Richard to Ipsen, to drive the continued growth of our North American business and further our development as a leading biopharma company focused on innovation and specialty care. Richard brings a wealth of great leadership experience, vision, and a track record of exceptional execution in leading and growing businesses, both in the US and internationally. He will bring great passion and energy to our work with patients."

During a 10-year career at Amgen, Richard Paulson held a number of positions in the company, including General Manager, Central and Eastern Europe, and subsequently General Manager Germany, before assuming leadership positions in Amgen’s Oncology Business Unit. Prior to joining Amgen, he held international positions in general management, marketing and market access with Pfizer. He also served in a number of sales and marketing roles with increasing seniority for GlaxoWellcome in Canada. He holds an MBA from the University of Toronto.

Richard Paulson added: "I am honored to join Ipsen at this time of rapid transformation and growth, as the company progresses in its evolution as a leading global biopharma company. Throughout my career, in markets around the world, I have been passionate about building and leading diverse, high-performing teams, and driving innovation for patients. I am extremely excited to join this dynamic company, with its clear mission and commitment to developing patient-focused solutions that put the patients’ needs first."

With the former President North America Cynthia Schwalm leaving Ipsen to pursue other interests, Ipsen would like to thank Cynthia for her many contributions to the growth of Ipsen’s North American business, and wishes her well in her future endeavors

On Jan. 11, 2018 /Forty Seven Inc., a clinical-stage company focused on developing the next generation of transformational immuno-oncology treatments to enable patient’s immune systems to defeat their cancer, reported an agreement with Genentech Inc., a member of the Roche Group., for Genentech to sponsor two clinical trials combining Forty Seven’s CD47 antibody, Hu5F9-G4, with Genentech’s PD-L1 antibody, atezolizumab (TECENTRIQ), in patients with acute myeloid leukemia (AML) and with urothelial (bladder) cancer (Press release, Hoffmann-La Roche, JAN 11, 2018, View Source [SID1234531337]).

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CD47 is an immune modulator molecule overexpressed on cancer cells that sends inhibitory signals to macrophages. Binding of Hu5F9-G4 to CD47 takes the brakes off macrophages enabling them to phagocytose, or swallow, tumor cells.

Craig Gibbs, Ph.D., M.B.A., Chief Business Officer at Forty Seven Inc. said, "There is a large unmet medical need for new therapies for AML and bladder cancer patients, particularly those who are elderly or have compromised organ function and are not able to withstand the side-effects of chemotherapy. We are excited to evaluate these novel combinations in collaboration with a global leader in oncology."

References
CD47 blockade as another immune checkpoint therapy for cancer. Robert H. Vonderheide, Nature Medicine 21, 1122, 2015.

TECENTRIQ (atezolizumab) is a registered trademark of Genentech, a member of the Roche Group.

On January 11, 2018 Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, reported the publication of clinical and pre-clinical results by research collaborators at Rgenix and The Rockefeller University (Press release, Rgenix, JAN 11, 2018, View Source [SID1234523092]). The results of the collaboration, published in the January 11 online issue of Cell, reveal that the Liver-X Receptor/Apolipoprotein E (LXR/ApoE) pathway regulates anti-tumor immunity via effects on myeloid-derived suppressor cells (MDSCs).

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Rgenix Announces Publication In Cell Demonstrating Activation of LXR/ApoE with RGX-104 Enhances Antitumor Immunity

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MDSCs are an immunosuppressive cell population that have been found to be circulating at high levels in cancer patients who are also commonly found to be non-responders to immunotherapy. RGX-104 was able to deplete MDSCs both in cancer patients participating in the dose escalation portion of the Phase 1a/b trial and in mice. This resulted in robust activation of relevant T cell populations in both settings.

"We are pleased to have this opportunity to share our latest research results in Cell that illustrate the effects our first-in-class RGX-104 compound has on one of the key cells that are responsible for suppressing the immune response in cancer patients," said Masoud Tavazoie, M.D., Ph.D., and Chief Executive Officer and co-founder of Rgenix. "We believe RGX-104 represents a novel strategy for stimulating anti-tumor immunity, and this data cements this belief and furthers our resolve to continue developing this compound both as monotherapy as well as in combination with checkpoint inhibitor therapy."

Sohail Tavazoie, M.D., Ph.D., Leon Hess Associate Professor and Head of Elizabeth and Vincent Meyer Laboratory of Systems Cancer Biology at the Rockefeller University who was the senior-author of the study as well as co-founder of Rgenix, added: "The data our research has generated at this stage is exciting and supports our perspective that RGX-104 has the potential to modulate the immune response in a broad array of cancers. We will continue to progress in our research and our pursuit of unique treatments for patients affected by cancers with a high unmet medical need."

The paper, titled "LXR/ApoE Activation Restricts Innate Immune Suppression in Cancer", was published today and is available online.

On January 11, 2018 Helsinn, a Swiss pharmaceutical group focused on building quality cancer care products, and MEI Pharma, Inc. (Nasdaq: MEIP), an oncology company focused on the clinical development of novel therapies for cancer, reported that the European Medicines Agency (EMA) has granted Orphan Drug Designation to pracinostat, an investigational drug candidate currently in a Phase 3 study in combination with azacitidine for the treatment of acute myeloid leukemia (AML) in adult patients unfit to induction chemotherapy (Press release, MEI Pharma, JAN 11, 2018, View Source [SID1234523054]).

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The orphan drug designation is based on the scarcity of treatments for patients suffering from AML and on positive Phase 2 study results (see below) that were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, in December 2016.

The EMA orphan drug designation is a status assigned to a medicine intended for use against a rare condition in the European Union (prevalence of the condition in the European Union must not be more than 5 in 10,000). The designation allows pharmaceutical companies to benefit from incentives offered by the EU to develop a medicine for the treatment, prevention or diagnosis of a disease that is life threatening or a chronically debilitating rare disease. These include 10 years of market exclusivity once approved, alongside a range of other regulatory advantages.

Riccardo Braglia, Helsinn Group Vice Chairman and CEO, commented: "Helsinn is pleased with the decision of the EMA to grant orphan drug designation to pracinostat. This decision encourages us to continuously dedicate significant resources to accelerate our clinical trial program, with a goal of helping patients who are fighting rare and difficult-to-treat diseases, such as AML and, at present, have very few treatment options. Following the positive Phase 2 clinical trials of pracinostat for patients with AML, Helsinn has recently initiated the Phase 3 program."

"This designation from the EMA recognizes the potential that pracinostat holds in addressing a significant unmet need for those suffering with AML," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "This represents another important milestone in the global development strategy for pracinostat."

1st patient randomized in Phase 3 trial:

In August of 2017, Helsinn and MEI Pharma announced that the first patient has been dosed in the pivotal Phase 3 study of the investigational agent pracinostat in combination with Azacitidine in adults with newly diagnosed acute myeloid leukemia (AML) who are unfit to receive intensive induction chemotherapy. The primary endpoint of the study is overall survival. Secondary endpoints include, among others, morphologic complete remission (CR) rate, cytogenetic complete remission and complete remission without minimal residual disease.

Phase 2 study results for pracinostat for the treatment of AML:

Results from a Phase 2 open-label, single-arm, multicenter study of pracinostat and azacitidine in 50 patients aged ≥ 65 years with newly diagnosed AML not eligible for induction chemotherapy showed a median overall survival of 19.1 (95%CI: 10.0-26.5) months, one-year survival of 62% and a CR rate of 42%. These results were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2016.

About AML
AML is the most common acute leukemia affecting adults, and its incidence is expected to continue to increase as the population ages. The American Cancer Society estimates about 21,380 new cases and 10,590 deaths from AML in the U.S. for 2017; the average age of a patient with AML is about 67 years. According to the Surveillance of Rare Cancers in Europe project, the incidence of AML in Europe is 3.7 per 100,000. There are currently no drugs approved in the U.S. to treat AML in patients who are unfit for intensive induction chemotherapy, though hypomethylating agents are recommended by the National Comprehensive Cancer Network (NCCN) guidelines. In the EU, Azacitidine is approved for the treatment of adult patients who are not eligible for hematopoietic stem cell transplant (HSCT) with AML with >30% marrow blasts according to the World Health Organization (WHO) classification, and decitabine is approved the treatment of adult patients with newly diagnosed de novo or secondary AML, according to the World Health Organisation (WHO) classification, who are not candidates for standard induction chemotherapy.

About Pracinostat
Pracinostat is an oral histone deacetylase (HDAC) inhibitor that is in late-stage clinical development. The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation for pracinostat in combination with Azacitidine for the treatment of patients with newly diagnosed AML who are ≥75 years of age or unfit for intensive chemotherapy. In August 2016, Helsinn and MEI Pharma entered into an exclusive license, development and commercialization agreement for pracinostat in AML and other potential indications. Under the terms of the agreement, Helsinn is granted a worldwide exclusive license to develop, manufacture and commercialize pracinostat, and is primarily responsible for funding its global development and commercialization. Pracinostat is under clinical investigation and has not been approved by any health authority worldwide.

On January 11, 2018 HedgePath Pharmaceuticals, Inc. (OTCQX:HPPI), a clinical stage biopharmaceutical company that discovers, develops and plans to commercialize innovative therapeutics for patients with cancer, reported that it has entered into a definitive preferred stock and warrant Securities Purchase Agreement with its majority stockholder Mayne Pharma under which Mayne Pharma has agreed to invest up to $5 million in HPPI, the first $2.4 million of which was received by HPPI on January 10, 2018, with a second tranche of $1.6 million to be funded by mid-2018, and a third tranche to be funded by year end if HPPI’s pending New Drug Application (NDA) for the SUBA-Itraconazole treatment of Basal Cell Carcinoma Nevus Syndrome (BCCNS) is accepted for filing by the U.S. Food and Drug Administration (FDA) (Press release, HedgePath Pharmaceuticals, JAN 11, 2018, View Source [SID1234523061]).

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This latest investment by Mayne Pharma will support HPPI’s clinical and regulatory efforts during 2018 toward the anticipated filing of the SUBA-Itraconazole BCCNS NDA with FDA during 2018. The price paid by Mayne Pharma for its shares of preferred stock and warrants (which represent 50% warrant coverage) is effectively $0.23 per share of HPPI’s common stock.

As reported in October 2017, based on the completion of enrollment for, and interim results observed, in its open label Phase 2(b) SCORING trial in the U.S., HPPI is preparing a pre-NDA meeting request for submission to FDA, with the goal of meeting with representatives of FDA during the second quarter of 2018 to discuss the NDA filing.

In connection with this financing, HPPI and Mayne Pharma have also entered into an important amendment to their key Supply and License Agreement (SLA) (under which HPPI has the rights from Mayne Pharma to develop and commercialize SUBA-Itraconazole products for cancer in the U.S.) to eliminate the provision that would have allowed Mayne Pharma to terminate the SLA in the event that HPPI had not received NDA approval for a product covered by the SLA by October 31, 2018.

Instead, the amended SLA allows HPPI and Mayne Pharma to continue the development and marketing of SUBA-Itraconazole-based cancer products in the United States under the SLA and, instead of a termination right, grants Mayne Pharma the option (but not the obligation) to assume control of the economic benefits of the SUBA-Itraconazole BCCNS program only in the event that a SUBA-Itraconazole NDA filing under the SLA has not been accepted for filing by FDA (as opposed to approved by FDA) by December 31, 2018 or if HPPI fails to launch SUBA-Itraconazole for treatment of BCCNS by June 30, 2020. Only in the event this option is exercised

by Mayne Pharma, HPPI will license only its BCCNS-related clinical data and intellectual property to Mayne Pharma and would receive back from Mayne Pharma a cash royalty on net sales of SUBA-Itraconazole for the treatment of BCCNS in the U.S.

HPPI separately announces today that, based upon clinical data from the BCCNS Phase 2(b) trial, Mayne Pharma filed for Orphan Designation in Europe with the EMA (European Medicines Agency) in 2017 and orphan designation was recently granted for the SUBA-Itraconazole treatment of BCCNS. If the SUBA-Itraconazole treatment of BCCNS is approved, it would carry a 10 year period of marketing exclusivity in Europe. The BCCNS program already carries an Orphan Drug Designation in the U.S., which upon approval by the FDA would provide 7 years of marketing exclusivity in the United States.

Nicholas J. Virca, HPPI’s President and Chief Executive Officer, stated that, "We are very pleased and view it as an important validation that Mayne Pharma is continuing its support of our efforts with this additional investment, which brings Mayne Pharma’s total investment in HPPI to more than $13 million. Importantly, Mayne Pharma and HPPI have also removed a significant risk and investor concern that our SLA could have been terminated outright in 2018 and replaced this risk with what we believe is a fair compromise that protects HPPI’s business plan and interests and assets generated under the SLA for the benefit of all of HPPI’s stockholders."

Readers are advised that further details regarding the terms of the preferred stock and warrant financing and the SLA amendment described herein will be provided in a Current Report on Form 8-K to be filed by HPPI with the Securities and Exchange Commission.