Presentation of December 2017

On December 14, 2017 Fortress Biotech presented Corporate presentation of December 2017 (Presentation, Fortress Biotech, DEC 14, 2017, View Source [SID1234522645]).

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AIFA set the reimbursement price for Zalmoxis® at EUR 149.000 per infusion, gross of discounts foreseen by law, and also established a flat fee per patient

On December 13, 2017 MolMed S.p.A. (MLM.MI) reported that it obtained its first national marketing authorization for its proprietary product, Zalmoxis: the Board of Directors of AIFA (Agenzia Italiana del Farmaco) approved the agreement negotiated between AIFA’s Prices and Reimbursement Committee (CPR) and MolMed, in which the price and reimbursement for Zalmoxis medicinal product were defined (Press release, MolMed, DEC 13, 2017, View Source [SID1234595072]).

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Zalmoxis is MolMed’s first patient specific cell therapy product, based on genetically engineering of the immune system, administered following haploidentical haematopoietic stem-cell transplantation (HSCT) from partially compatible donors to adult patients with leukaemia and other high-risk haematologic malignancies. Zalmoxis is administered from the 21st day post-transplantation and foresees up to a maximum of 4 infusions per patient based on the achievement of immune-reconstitution.

The terms of the agreement provide for an ex-factory price, excluding VAT, of 149,000 EUR per infusion, gross of discounts foreseen by law and of selective reductions foreseen by AIFA Resolutions ("Determinazioni") of 3 July 2006 and 27 September 2006. Furthermore the agreement also set a flat fee per patient and a safeguard clause on sales for the first 24 months. Given the nature of the product, it will be a hospital-only dispensed therapy.

The agreement signed with AIFA will be effective from the fifteenth day subsequent to its publication in the Gazzetta Ufficiale of the Italian Republic.

Riccardo Palmisano, MolMed’s CEO, commented: "This is truly a turning point for a research and development company like MolMed: both Zalmoxis eligibility for reimbursement and the "Aifa granted price" acknowledge the value of our therapy and, at the same time, the successful completion of a top level Italian research, development and manufacturing journey. In addition, this result paves the way to the marketing of this sophisticated genetically engineered cell therapy. This first national authorization allows us to look with increasing confidence to the introduction of Zalmoxis in the other European countries covered by the Dompé agreement, from which we expect relevant results, considering our partner’s value, expertise and successful track record. Zalmoxis commercialization will allow MolMed to increase its revenues from proprietary products on top of those deriving from GMP development and production for third party."

Professor Claudio Bordignon, Founder and Chairman of MolMed, commented: "The inclusion of Zalmoxis among the medicinal products reimbursable by the National Health Service makes this revolutionary therapy available to those patients for whom the allogeneic stem cell transplantation is the best treatment option for high-risk leukaemia and other blood malignancies. In absence of a fully compatible donor, Zalmoxis makes the treatment from a haploidentical family donor safer, by eliminating post-transplant immunosuppression and, at the same time, resolving the potential occurrence of Graft versus Host Disease. Furthermore, Zalmoxis accelerates immune-reconstitution by protecting the patient against infections and leukaemia relapse. Zalmoxis has proven to significantly increase the one-year survival rate in the treated population."

"We are very pleased with the outcome of MolMed’s negotiation with AIFA. By stipulating this agreement, it is stated that Zalmoxis, the first ex-cell cellular therapy based on the immune system engineering for the PRESS RELEASE 2 treatment of adult patients with high risk blood malignancies, responds to a major clinical need", commented Eugenio Aringhieri, Chief Executive Officer of Dompé. "The synergy of our distinctive competences and shared value priorities, on which this alliance with MolMed is founded, will lead us in the interaction with the scientific community and the regulatory authorities aimed at bringing Zalmoxis to all Patients eligible for this therapy."

About Zalmoxis
Zalmoxis is an innovative therapy based on genetically engineering donor immune system T cells to carry an inducible "suicide gene". Administered to patients following HSCT from partially compatible donors (haploidentical HSCT), these cells foster an anti-leukaemia effect by eliminating post-transplant immunosuppression prophylaxis and inducing a rapid immune reconstitution. The suicide gene allows to readily control Graft versus Host Disease (GvHD), the most significant and serious adverse event in haploidentical transplantation, caused by the genetic disparity between patient and donor. Zalmoxis significantly increases long-term survival, regardless of disease status at transplant, thus making HSCT from partially compatible donors safer and more effective. On August 18th, 2016 the European Commission granted a Conditional Marketing Authorisation (CMA) for Zalmoxis, the first immunogene therapy, as patient-specific adjunctive treatment in haplo-identical haematopoietic stem-cell transplantation for adult patients with leukaemia and high-risk haematological malignancies. Following this authorization, which allows MolMed to market Zalmoxis in the 28 EU Member States and in the European Economic Area, activities aimed at its introduction on the European markets have increased. The marketing authorization issued by AIFA is therefore only the first out of the ones on which MolMed has been working since the date of the European CMA.

With the aim of successfully marketing Zalmoxis, on July 26th, 2017 MolMed signed an exclusive license and distribution agreement with Dompé Farmaceutici S.p.A., one of the leading Italian biopharmaceutical companies, granting Dompé the exclusive right and obligation to conduct all activities aimed at promoting, marketing, exploiting, distributing and selling Zalmoxis in all member countries of the current European Economic Area (EEA) and an option right for Switzerland, Turkey and Australia.

In parallel, preparation of the dossier to obtain price and reimbursement of Zalmoxis from the German and French authorities continued through direct interactions. Furthermore activities aimed at starting negotiations with authorities of other countries have been speeded up. In regard to distribution and marketing beyond the European borders, contracts have already been signed with Megapharm Ltd for Israel and with TTY Biopharm Company Ltd for Taiwan and a number of countries in South-East Asia.

Puma Biotechnology Announces Meeting of Scientific Advisory Group on Oncology in Europe to Review Neratinib for Extended Adjuvant Treatment of HER2-Positive Early Stage Breast Cancer

On December 13, 2017 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that the European Medicines Agency (EMA) has requested that the Scientific Advisory Group on Oncology provide an opinion on the clinical aspects of the Marketing Authorization Application (MAA) for neratinib at a meeting to be held on January 11, 2018 (Press release, Puma Biotechnology, DEC 13, 2017, View Source [SID1234522635]). In Europe, neratinib is an investigational therapy for the extended adjuvant treatment of early stage HER2-positive breast cancer that has previously been treated with a trastuzumab containing regimen.

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The Scientific Advisory Group on Oncology is convened at the request of the EMA to provide independent recommendations on scientific or technical matters related to pediatric and adult clinical oncology and hematology, or on any other scientific issue relevant to the work of the EMA that relates to this area.

Puma Biotechnology announced on August 22, 2016 that the MAA for neratinib had been validated by the EMA. The MAA for neratinib is based on results from both the Phase III ExteNET trial in extended adjuvant early stage HER2-positive breast cancer and the Phase II CONTROL trial in extended adjuvant early stage HER2-positive breast cancer. On August 2, 2017, Puma announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the EMA, had issued its Day-180 List of Outstanding Issues in the process of their ongoing regulatory review of Puma’s MAA. The Company expects to respond to the Day-180 List of Outstanding Issues by the deadline of December 22, 2017.

Pionyr Immunotherapeutics Completes $62 million Series B Financing to Advance Antibody-Based Therapeutics Targeting the Tumor Microenvironment

On December 13, 2017 Pionyr Immunotherapeutics, Inc., a company developing antibody therapeutics that increase the body’s antitumor immunity by rebalancing the myeloid infiltrate in the tumor microenvironment, reported the closing of a $62 million Series B investment round. This brings total funding since the company’s founding in 2015 to $72 million. The Series B financing was led by New Enterprise Associates and included Sofinnova Ventures and Vida Ventures, along with Pionyr’s existing investors, OrbiMed, SV Health Investors, Osage University Partners, and Mission Bay Ventures.

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Research conducted at Pionyr over the past year shows that its most advanced technology, Myeloid Tuning, is capable of rebalancing the tumor microenvironment to favor immune-activating myeloid cells over immune-suppressing myeloid cells. This activity is believed to enhance antitumor efficacy, particularly in combination with checkpoint inhibition. The proportions of specific subpopulations of myeloid cells in the tumor microenvironment have been shown to correlate with clinical outcome and predict checkpoint inhibitor responsiveness.

"Our technology has led to a promising pipeline of potential new therapeutics against novel and highly specific targets in the tumor microenvironment," Max Krummel, Ph.D., UC San Francisco professor, Pionyr co-founder and member of the board of directors said. "We believe these drug candidates hold the keys to an untapped area of immuno-oncology and may have a significant impact on multiple cancers."

Dr. Krummel has been a pioneer in immuno-oncology since the mid-1990s and is the co-inventor of YERVOY (ipilimumab), the first marketed checkpoint inhibitor, which was approved in 2011 to treat melanoma.

Pionyr was co-founded by Dr. Krummel and Sachdev Sidhu, Ph.D., a protein engineer and key player in early antibody phage-display technology platforms, formerly at Genentech, who is now head of protein engineering and antibody discovery at the University of Toronto.

Pionyr is led by industry veterans with deep biotech management experience and immuno-oncology expertise. Steven James, former CEO of Labrys Biologics (acquired by TEVA) and KAI Pharmaceuticals (acquired by Amgen), is the president and CEO of the company. The company’s senior vice president of research is Michel Streuli, Ph.D., who has over 15 years of pharma and biotech experience, and previously led immuno-oncology efforts at Gilead, Merck (as early development head for KEYTRUDA), Schering-Plough, and Organon.

"Pionyr has made great progress over the past year and we are extremely gratified that this has led to a financing sufficient in size to take two of our antibodies into human cancer trials," James said. "NEA, Sofinnova Ventures, and Vida Ventures are superb additions to our existing syndicate of top flight investors, and have been tremendously supportive of our efforts to push forward with multiple programs emerging from our pipeline."

Joining Prof. Krummel, Mr. James, Leon Chen, Ph.D. (OrbiMed Advisors), and Joshua Resnick, M.D. (SV Health Investors) on the Pionyr board of directors are Carol Gallagher, Pharm.D. (NEA), Mike Powell, Ph.D. (Sofinnova) and Arjun Goyal, M.D. (Vida Ventures) as an observer.

Dr. Carol Gallagher commented, "We are impressed with the rapid translation work the Pionyr team has conducted and look forward to working with them to enable these novel drug candidates to advance toward the clinic to evaluate the promise of this novel approach in immuno-oncology."

Bayer receives approval in China for Stivarga® (regorafenib) for the second-line systemic treatment of liver cancer (for specialized target groups only)

On December 13, 2017 Bayer reported that the Chinese Food and Drug Administration (CFDA) approved Stivarga (regorafenib) tablets for the second-line treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with Nexavar (sorafenib) (Press release, Bayer, DEC 13, 2017, View Source [SID1234523072]). The data from the pivotal Phase III RESORCE study showed that Stivarga (regorafenib) provided a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo; the median OS was 10.6 vs 7.8 months, (HR 0.62, 95% CI 0.50-0.78; p<0.0001). Exploratory analyses of the RESORCE trial showed that the median time from the start of prior sorafenib treatment to death was 26 months in patients receiving regorafenib versus 19.2 months in those receiving placebo. Regorafenib is the first drug approved for the second-line treatment of patients with HCC in China. The CFDA approval expands Bayer’s leadership in liver cancer with a treatment plan in HCC involving use of Stivarga directly after progression on Nexavar.

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"Following the approval of Stivarga for the treatment of metastatic colorectal cancer and gastrointestinal stromal tumors earlier this year in China, the approval in HCC brings new hope to Chinese patients with HCC who previously had no effective treatment options after being treated with Nexavar", said Robert LaCaze, Member of the Executive Committee of Bayer AG’s Pharmaceuticals Division and Head of the Oncology Strategic Business Unit. "The product is already approved for the treatment of HCC in many countries around the world, including US, Japan and in the EU, and this milestone expands Bayer’s global leadership in liver cancer."

Liver cancer is often more difficult to treat than other cancers with 466,000 new cases diagnosed and 422,000 deaths in China per year. Globally, it is the second leading cause of cancer-related deaths.

About Regorafenib (Stivarga)
Regorafenib is an oral multi-kinase inhibitor that potently blocks multiple protein kinases involved in tumor angiogenesis (VEGFR1, -2, -3, TIE2), oncogenesis (KIT, RET, RAF-1, BRAF), metastasis (VEGFR3, PDGFR, FGFR) and tumor immunity (CSF1R).

Regorafenib is approved under the brand name Stivarga in more than 90 countries worldwide, including the U.S., countries of the EU, China and Japan for the treatment of metastatic colorectal cancer (mCRC). The product is also approved in over 80 countries, including the U.S., countries of the EU, China and Japan, for the treatment of metastatic gastrointestinal stromal tumors (GIST). This year, it was also approved in the U.S., Japan and countries of the EU for second-line treatment of HCC.

In the EU, Stivarga is indicated as monotherapy for the treatment of adult patients with mCRC who have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidine-based chemotherapy, an anti-VEGF therapy and an anti-EGFR therapy, as well as for the treatment of adult patients with unresectable or metastatic GIST who progressed on or are intolerant to prior treatment with imatinib and sunitinib, and for the treatment of adult patients with HCC who have been previously treated with sorafenib.

Regorafenib is a compound developed by Bayer. In 2011, Bayer entered into an agreement with Onyx, now an Amgen subsidiary, under which Onyx receives a royalty on all global net sales of regorafenib in oncology.

About Oncology at Bayer
Bayer is committed to delivering science for a better life by advancing a portfolio of innovative cancer treatments. The oncology franchise at Bayer currently includes three oncology products and several other compounds in various stages of clinical development. Together, these products reflect the company’s approach to research, which prioritizes targets and pathways, with the potential to impact the way that cancer is treated.