On Jaunuary 11, 2018 Forty Seven Inc. a clinical-stage company focused on developing the next generation of transformational immuno-oncology treatments to enable a patient’s immune system to defeat their cancer, reported an agreement with Merck KGaA, Darmstadt, Germany to conduct a Phase 1b clinical trial combining Hu5F9-G4 with avelumab in patients with ovarian cancer (Press release, Forty Seven, JAN 11, 2018, View Source [SID1234527432]).

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PD-L1 and CD47 are immunosuppressant molecules overexpressed on cancer cells that send inhibitory signals to T cells and macrophages, respectively. Binding of avelumab to PD-L1 takes the brakes off T cells and, in a similar way, binding of Hu5F9-G4 to CD47 takes the brakes off macrophages.

"PD-L1 inhibitors, like avelumab, belong to a class of new immunological therapies for cancer known as checkpoint inhibitors that offer the opportunity for long-term remissions in some cancer patients," said Forty Seven Inc. CMO Chris Takimoto. "Not all patients however, respond to checkpoint inhibitors, so additional scientifically driven combination approaches are required."

"Ovarian cancer patients have limited treatment options, especially as they are often diagnosed at a late stage in their disease," says Dr. Alise Reicin, Head of Global Clinical Development at the Biopharma business of Merck KGaA, Darmstadt, Germany, which in the US and Canada operates as EMD Serono. "We have two ongoing registrational studies exploring the role that avelumab could play both as a monotherapy and in combinations in ovarian cancer. This collaboration enhances our strategic approach to novel I-O combinations in this disease setting. We are hopeful that through these efforts we will discover viable options to help patients with this hard-to-treat cancer."

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 Puma Biotechnology, Inc. (the "Company") reported it met today with the Committee for Medicinal Products for Human Use ("CHMP") Scientific Advisory Group on Oncology ("SAG") (Press release, Puma Biotechnology, JAN 11, 2018, View Source [SID1234523065]). SAG was asked to provide an opinion on the clinical relevance of the 5-year absolute treatment difference in invasive disease free survival seen in the Phase III ExteNET trial and on the risk of gastrointestinal toxicity with neratinib and its acceptability in the proposed patient population in the Company’s Marketing Authorization Application ("MAA") for neratinib. Based on the feedback from SAG and the rapporteurs, the Company intends to modify the summary of product characteristics ("SmPC"), sometimes referred to as the European product label, in its MAA for neratinib to further refine the intended population to patients at a high risk of disease recurrence.

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CHMP will be conducting an oral hearing to discuss the MAA for neratinib on January 23, 2018, and the Company has been invited to present the risk benefit profile of neratinib in the identified population at this meeting

On January 11, 2018 Novartis reported that Elizabeth (Liz) Barrett, currently Global President Oncology at Pfizer, Inc., has been appointed CEO Novartis Oncology and a member of the Executive Committee of Novartis, effective February 1, 2018 (Press release, Novartis, JAN 11, 2018, View Source [SID1234523064]). She will be based in Basel. Mrs. Barrett succeeds Bruno Strigini who decided to retire from Novartis for personal reasons.

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Vasant (Vas) Narasimhan, designated CEO of Novartis, said: "Liz is a highly accomplished and recognized oncology and people leader with an impressive record of building successful business organizations in the US, Europe and globally. She has been instrumental in creating new commercial models, driving innovation in close partnership with research and development and leveraging business development opportunities. Her long-time commercial pharma industry experience, marketing skills and perspectives make Liz a great fit to further develop our oncology business."

Mrs. Barrett said: "I feel honored to join Novartis, a recognized pioneer in the oncology area. It is key to me to contribute to transformative advancements in oncology, and to serve in a highly impactful leadership role to this end."

Mrs. Barrett has held numerous leadership positions in the pharmaceutical industry as well as in the consumer sector. In her most recent role at Pfizer, she led the oncology business through a significant period of growth achieved in less than three years. Before joining Pfizer in 2009, she worked at Cephalon, Inc. and Johnson & Johnson. She started her career at Kraft Foods Group, Inc. in 1984.

Mrs. Barrett is a US citizen and has lived and worked in the US and Europe. She obtained an MBA in Marketing from the Saint Joseph’s University in Philadelphia and a BSc in Business Administration from the University of Louisiana.

Novartis also announced today that Robert Kowalski, Pharm.D., Head of Global Regulatory Affairs, will assume ad interim leadership of the Drug Development Organization, effective February 1, 2018. Dr. Kowalski has been Head of Global Regulatory Affairs for Novartis since February 2016 and has played an important leadership role in securing approvals for several breakthrough medicines including our revolutionary CAR-T therapy Kymriah. The definitive Head of Global Drug Development will be announced in due course.

Disclaimer

This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, . Forward-looking statements can generally be identified by words such as "effective," "will," "to further develop," or similar expressions, or by express or implied discussions regarding potential future sales or earnings of the Novartis Group. You should not place undue reliance on these statements. Such

forward looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward looking statements. There can be no guarantee that Novartis will be commercially successful in the future, or achieve any particular financial results. In particular, our expectations could be affected by, among other things: regulatory actions or delays or government regulation generally; the potential that the strategic benefits, synergies or opportunities expected from the significant reorganizations of recent years may not be realized or may take longer to realize than expected; the uncertainties inherent in the research and development of new healthcare products; our ability to obtain or maintain proprietary intellectual property protection on key products; safety, quality or manufacturing issues; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; uncertainties regarding actual or potential legal proceedings; and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

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.