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MacroGenics’ MGD006 Granted Orphan Drug Status for AML by FDA

On January 5, 2016 MacroGenics, Inc. (Nasdaq: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MGD006 (also known as S80880), a DART molecule that recognizes both CD123 and CD3, for the investigational treatment of acute myeloid leukemia (AML) (Press release, MacroGenics, JAN 5, 2017, View Source [SID1234517281]).

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MGD006 is currently being evaluated in the U.S. and Europe in a Phase 1 dose-escalation study designed to assess the safety and tolerability of the molecule in patients with relapsed/refractory AML or myelodysplastic syndrome (MDS). MacroGenics retains full development and commercialization rights to MGD006 in the U.S., Canada, Mexico, Japan, South Korea and India. Servier participates in the development and has rights to MGD006 in all other countries.

The FDA orphan drug designation provides certain incentives for medications intended for the treatment, diagnosis or prevention of rare diseases. At present, these incentives include seven years of marketing exclusivity for the orphan indication, certain federal grants, tax credits and waiver of certain FDA fees.

"The FDA’s decision to grant orphan drug designation for MGD006 in AML is an important regulatory milestone for MacroGenics as we continue to develop this bispecific DART molecule in this difficult-to-treat disease," said Scott Koenig, M.D., Ph.D., President and Chief Executive Officer of MacroGenics. "We believe MGD006 has the potential to be a significant advancement in the treatment of AML, and are pleased that the FDA has recognized the potential of MGD006 to benefit patients in need. We expect to select a dose this year to advance MGD006 into its next phase of clinical development."

About MGD006

MGD006 is a clinical-stage molecule that recognizes both CD123 and CD3. CD123, the Interleukin-3 receptor alpha chain, has been reported to be over-expressed on cancer cells in a wide range of hematological malignancies including AML and MDS.

The primary mechanism of action of MGD006 is believed to be its ability to redirect T lymphocytes to kill CD123-expressing cells. To achieve this, the DART molecule combines a portion of an antibody recognizing CD3, an activating molecule expressed by T cells, with an arm that recognizes CD123 on the target cancer cells.

In December 2016, MacroGenics presented initial clinical experience from the ongoing Phase 1 study of MGD006. Dosing schema and supportive care regimens have been refined to enable therapeutic goals and decrease the effects of cytokine induction by MGD006, an anticipated event resulting from the engagement and activation of T lymphocytes. In addition, the Company continues to characterize the pharmacokinetic properties and clinical activity of MGD006 in AML patients.

RedHill Biopharma Announces New Research Collaboration with Aarhus University for Oncology Drug MESUPRON

On January 5, 2017 RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on the development and commercialization of late clinical-stage, proprietary, orally administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, reported the signing of a new collaboration agreement with the Department of Molecular Biology and Genetics of Denmark-based Aarhus University ("AU") for the evaluation of RedHill’s Phase II-stage oncology drug candidate, MESUPRON (upamostat) (Press release, RedHill Biopharma, JAN 5, 2017, View Source [SID1234517280]).

MESUPRON, a proprietary, first-in-class, orally administered protease inhibitor with several potential mechanisms of action to inhibit tumor invasion and metastasis, presents a new non-cytotoxic approach to cancer therapy. In 2014 RedHill acquired the exclusive worldwide development and commercialization rights to MESUPRON, excluding China, Hong Kong, Taiwan and Macao, from Germany’s WILEX AG for all indications. Wilex AG completed several clinical studies with MESUPRON for different indications, including two Phase II proof-of-concept studies, one for pancreatic cancer and one for metastatic breast cancer.

Terry Plasse, MD, RedHill’s Medical Director said: "To date, the collaboration with Aarhus University has led to findings with MESUPRON on the structure activity relationships between its active metabolite UK-1 and proteases, an established family of molecular targets with therapeutic potential in oncology indications. Previous non-clinical trials conducted in Denmark with Aarhus University have identified multiple proteases, which may be more sensitive to MESUPRON than the originally proposed target, uPA. We hope that further evaluation of MESUPRON, together with Aarhus University, will enable the optimal selection of appropriate patients toward demonstrating the activity of MESUPRON in forthcoming planned clinical trials."

The Non-clinical studies with MESUPRON are intended to support the clinical data from previous Phase I and Phase II studies, and may allow RedHill to take a precision medicine approach going forward.

About MESUPRON:
MESUPRON is a proprietary, first-in-class, orally administered protease inhibitor. Protease inhibitors, including urokinase-type plasminogen activators (uPa), have been shown to play key roles in tumor invasion and the metastasis process. High levels of certain proteases, including uPA, are associated with poor prognosis in various solid tumor cancers, such as pancreatic, gastric, breast and prostate cancers. MESUPRON presents a promising new non-cytotoxic approach to cancer therapy with several potential mechanisms of action to inhibit both tumor metastasis and growth. MESUPRON has undergone several Phase I studies and two Phase II proof-of-concept studies. The first Phase II study was in locally advanced, unresectable pancreatic cancer and the second study in metastatic breast cancer in combination with first-line chemotherapeutic agents.

Pieris Pharmaceuticals and Servier Forge Strategic Immuno-oncology Co-development Alliance

On January 5, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform, and Servier, an independent international pharmaceutical company, reported a broad collaboration in immuno-oncology (IO) (Press release, Pieris Pharmaceuticals, JAN 5, 2017, View Source [SID1234517279]). Despite the impressive clinical efficacy of checkpoint inhibitors to date, a majority of patients fail to respond to approved therapies. The collaboration seeks to address this significant unmet clinical need by advancing a series of novel molecules, including multiple dual immune checkpoint blockade approaches.

Under the collaboration, Pieris and Servier will initially pursue five bispecific therapeutic programs, led by Pieris’ PRS-332 program, a potentially best-in-class PD-1-targeting bispecific checkpoint inhibitor. Pieris and Servier will jointly develop PRS-332 and split commercial rights geographically, with Pieris retaining all commercial rights in the United States and Servier having commercial rights in the rest of the world. The four additional committed programs have been defined, which may combine antibodies from the Servier portfolio with one or more Anticalin proteins based on Pieris’ proprietary platform to generate innovative immuno-oncology bispecific drug candidates. The collaboration may be expanded by up to three additional therapeutic programs. Pieris has the option, at a predefined time point, to co-develop and retain commercial rights in the United States for up to three programs beyond PRS-332, while Servier will be responsible for development and commercialization of the 4 other programs worldwide.

The financial terms of the collaboration include an upfront payment to Pieris of EUR30 million (approximately $31.3 million USD). Pieris may also receive FTE funding for specific projects, an option fee upon potential expansion of the collaboration as well as development-dependent and commercial milestone payments for PRS-332 and each additional program. The total development, regulatory and sales-based milestone payments to Pieris could reach EUR324 million (approximately $338 million USD) for PRS-332, and up to EUR193 million (approximately $201 million USD) for each of the other programs.Pieris and Servier will share preclinical and clinical development costs for each co-developed program. In addition, Pieris will be entitled to receive tiered royalties up to low double digits on the sales of commercialized products in the Servier territories.

Pieris’ multispecific technology allows simultaneous checkpoint inhibition on the same cell, which could have a clear advantage over monoclonal antibody cocktails against different checkpoint targets. PRS-332 is a novel PD-1-based bispecific, comprising an anti-PD-1 antibody genetically linked to an Anticalin protein targeting an undisclosed checkpoint target. Pieris has developed PRS-332, which is currently in preclinical development, with the intent to simultaneously block two immune checkpoints co-expressed on exhausted T cells to further improve on existing PD-1 therapies.

"Servier is a highly complementary partner for Pieris, with a very clear commitment to oncology and outstanding development capabilities," stated Dr. Louis Matis, Senior Vice President and Chief Development Officer of Pieris. "The synergies of building unique bispecifics from Servier’s antibodies and Pieris’ Anticalin proteins are multifold, as the versatility of our platform allows for extensive combinatorial target opportunities with the numerous IO ‘building blocks’ our team has discovered to date."

"This alliance will significantly enhance Servier’s portfolio in immuno-oncology, which already comprises 5 products in late preclinical or early development. Servier’s recognized expertise in drug development will efficiently complement Pieris’ innovative technology, allowing both companies to bring innovative solutions to cancer patients," stated Jean-Pierre Abastado, PhD, Director of Oncology R&D at Servier.

"Servier has built a diversified and innovative portfolio in oncology that includes small molecules, engineered antibodies, and cell therapies for the treatment of both hematological malignancies and solid tumors. Today’s alliance with Pieris adds another dimension to our strategy of becoming a key player in oncology, providing several next-generation bispecific IO drugs to our pipeline," added Emmanuel Canet, M.D., Ph.D., President of Servier R&D.

"Our alliance with Servier is clearly a transformative one for Pieris and is the type of partnership we deliberately set out to achieve to create significant long-term value. This collaboration provides not only an opportunity to advance multiple programs with retained rights in the number one oncology market, but also provides significant funding and flexibility for Pieris to balance financial and operational resources as we enter the next stage of corporate development," stated Stephen Yoder, President and Chief Executive Officer of Pieris. "The Servier alliance will act as a significant building block of our pipeline expansion in immuno-oncology and demonstrates the value of our proprietary Anticalin drug class."

Neon Therapeutics Secures $70 Million in Series B Financing

On January 5, 2017 Neon Therapeutics, an immuno-oncology company developing neoantigen-based therapeutic vaccines and T cell therapies to treat cancer, reported the successful completion of a $70 million Series B financing (Press release, Neon Therapeutics, JAN 5, 2017, View Source [SID1234517277]). Proceeds from the financing will be used to advance Neon Therapeutics’ lead program NEO-PV-01, a fully personalized neoantigen vaccine, through an ongoing Phase 1b clinical trial. In addition, this investment will support preclinical development of NEOPTC-01, a personalized adoptive T cell program, and the Shared Neoantigen Program.

The Series B financing was led by Partner Fund Management. Joined by existing investors Third Rock Ventures and Access Industries, additional new investors included Fidelity Management & Research Company, Wellington Management Company, Inbio Ventures and Nextech Invest.

"The investor quality in this financing reflects the promise of the neoantigen biology platform, as well as our leading position," said Hugh O’Dowd, chief executive officer of Neon Therapeutics. "This capital is a testament to our strong team and our approach to neoantigen-based therapeutics, and will provide important resources to bring potentially life-changing medicines to cancer patients in need."

Seattle Genetics Initiates Phase 1 Trial of SGN-CD352A for Patients with Relapsed or Refractory Multiple Myeloma

On January 5, 2017 Seattle Genetics, Inc. (NASDAQ: SGEN), a global biotechnology company, reported enrollment of the first patient in a multicenter phase 1 clinical trial of SGN-CD352A for patients with relapsed or refractory multiple myeloma (MM) (Press release, Seattle Genetics, JAN 5, 2017, View Source [SID1234517276]).

SGN-CD352A is an investigational CD352-targeted antibody-drug conjugate (ADC) utilizing Seattle Genetics’ proprietary ADC technology, an engineered cysteine antibody (EC-mAb) stably linked to a highly potent cytotoxic agent called a pyrrolobenzodiazepine (PBD) dimer. CD352 is broadly expressed on B-cell cancers including multiple myeloma, chronic lymphocytic leukemia and non-Hodgkin lymphoma, while exhibiting low expression on normal white blood cells. The trial is designed to assess the safety and antitumor activity of SGN-CD352A. This study represents Seattle Genetics’ first clinical-stage ADC program in development for MM, demonstrating the breadth of potential therapeutic applications for its industry-leading ADC technology platform.

"More than 124,000 people worldwide are diagnosed annually with multiple myeloma, most relapsing or becoming resistant to current therapies," said Robert Lechleider, M.D., Senior Vice President, Clinical Development at Seattle Genetics. "SGN-CD352A is a novel targeted investigational compound for multiple myeloma, and it is our latest antibody-drug conjugate, or ADC, in an expanding and robust pipeline of clinical stage empowered antibody therapies to address blood cancers and solid tumors. As we begin clinical development of our first compound for multiple myeloma, we continue to explore the broad potential of our ADC technology platform for people with cancer."

The phase 1, open-label multicenter clinical study is designed to evaluate the safety and preliminary antitumor activity of SGN-CD352A as a single agent in adults with relapsed or refractory MM. The trial will be conducted in two parts, with a dose escalation part to identify the maximum tolerated dose of SGN-CD352A followed by an expansion part to further define safety and antitumor activity. SGN-CD352A will be administered every four weeks, and the study will enroll approximately 75 relapsed or refractory patients at multiple centers in the United States.

Preclinical SGN-CD352A data presented at the 2016 American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting demonstrated that SGN-CD352A specifically binds to target cells and induces potent antitumor activity in both MM and non-Hodgkin lymphoma disease models. In addition to being a potential new monotherapy for MM, the tolerability profile from preclinical results suggests that SGN-CD352A may be combined with current standard of care treatments for MM. ADCs are designed to selectively deliver cell-killing agents to tumor cells, and thus may reduce many of the toxic effects of traditional chemotherapy while enhancing antitumor activity. With more than 15 years of experience and innovation, Seattle Genetics is the leader in ADC development.

For more information about the trial, including enrolling centers, please visit www.clinicaltrials.gov.

About Multiple Myeloma

Multiple myeloma (MM) is a rare and aggressive cancer that forms in white blood cells called plasma cells. Cancerous plasma cells can crowd out healthy blood cells, impair bone strength and weaken the immune system. Despite recent medical advances, MM still remains an incurable disease in which patients eventually progress and die. Within one year of first-line therapy, 32 percent of transplant patients and 44 percent of non-transplant patients relapse. Remission periods are typically shorter for each subsequent line of therapy, with some patients receiving more than four lines of treatment over the course of their disease. After lymphoma and leukemia, MM is the third most common blood cancer in the US. According to the World Health Organization, in 2015 more than 124,000 new cases of MM were diagnosed worldwide and more than 87,000 people died from the disease.

About SGN-CD352A

SGN-CD352A is a novel investigational ADC targeted to CD352 utilizing Seattle Genetics’ proprietary ADC technology. CD352 is broadly expressed on B-cell cancers including multiple myeloma, chronic lymphocytic leukemia, and non-Hodgkin lymphoma, while exhibiting low expression on normal white blood cells. The CD352 engineered cysteine antibody is stably linked to a highly potent DNA binding agent called a pyrrolobenzodiazepine (PBD) dimer via site-specific conjugation technology (EC-mAb). PBD dimers are significantly more potent than systemic chemotherapeutic drugs and the EC-mAb technology allows uniform drug-loading onto an ADC. The ADC is designed to be stable in the bloodstream and to release its potent cell-killing PBD agent upon internalization into CD352-expressing cells.