On November 28, 2016 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported advancements in the Company’s clinical programs evaluating tazemetostat, its first-in-class EZH2 inhibitor (Press release, Epizyme, NOV 28, 2016, View Source [SID1234516810]). The U.S. Food and Drug Administration (FDA) has granted tazemetostat Fast Track designation in patients with diffuse large B-cell lymphoma (DLBCL) with EZH2 activating mutations. Additionally, Epizyme is focusing its Phase 2 registration-enabling study in adult patients with genetically-defined solid tumors on those cancers marked by loss of INI1, and has expanded enrollment in the epithelioid sarcoma cohort of the study based on encouraging early activity in this patient population. Schedule your 30 min Free 1stOncology Demo! "We are very proud of the continued advancement of our tazemetostat clinical program in both non-Hodgkin lymphoma and genetically-defined solid tumors, important areas of unmet medical need for patients with cancer," said Robert Bazemore, president and chief executive officer, Epizyme. "These developments reflect the execution of our strategy to identify the patient groups who may benefit most from tazemetostat, and our efforts to bring this investigational medicine to patients as quickly as possible."
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NHL Program Update: Fast Track Designation for DLBCL Subtype
The FDA has granted Fast Track designation for the investigation of tazemetostat for the treatment of patients with relapsed or refractory DLBCL whose tumors carry an EZH2 activating mutation. Tazemetostat inhibits EZH2, a histone methyltransferase that is increasingly understood to play a role in the growth and proliferation of a number of cancers, including DLBCL, the most commonly diagnosed form of NHL.
The FDA Fast Track program is designed to facilitate the development of important new drugs and to provide patients access to those drugs more quickly. The designation enables early and frequent communication between FDA and a product sponsor throughout the drug development and review process. Through the Fast Track program, a product may be eligible for priority review at the time of a new drug application (NDA) filing and may also be eligible to submit completed sections of the NDA on a rolling basis before the complete application is submitted. These expedited processes can potentially reduce development time and cost associated with bringing a drug to market.
Genetically Defined Solid Tumor Program Update: Focus on INI1-Negative Tumors
Following review by the Independent Data Monitoring Committee, Epizyme has expanded the epithelioid sarcoma cohort of its ongoing Phase 2 trial in adult patients with INI1-negative solid tumors. This expansion is based on encouraging early activity seen, including confirmed objective responses, in the cohort. The company plans to enroll an additional 30 patients with epithelioid sarcoma, bringing the cohort to a total of 60 patients. This study represents the largest trial conducted to date in this rare tumor type. Epithelioid sarcoma is a soft tissue sarcoma characterized by loss of INI1. There are currently no approved systemic therapies for the treatment of patients with epithelioid sarcoma, and outcomes are extremely poor.
The synovial sarcoma arm of the Phase 2 trial has been fully enrolled. Although some patients remain on treatment, Epizyme has concluded that the activity of tazemetostat in this cohort is insufficient to continue further investigation of tazemetostat as a monotherapy. Unlike the cancers in the other four arms of the study, synovial sarcoma is characterized by a functional dysregulation of INI1, rather than by a complete loss of INI1.
Epizyme is now focusing its efforts on the four cohorts of INI1-negative tumors in its study, including the epithelioid sarcoma cohort. Enrollment continues in these cohorts and Epizyme plans to present data from the Phase 2 trial in the first half of 2017.
"The early clinical activity observed in the epithelioid sarcoma arm provides encouraging evidence of the effectiveness of EZH2 inhibition with tazemetostat in patients with INI1-negative tumors," added Peter Ho., M.D., Ph.D., chief medical officer, Epizyme. "The clinical experience observed so far in both the epithelioid sarcoma and synovial sarcoma cohorts is consistent with our Phase 1 experience for these cancers. We look forward to assessing clinical activity in the Phase 2 study and reporting data in the first half of 2017."
About the Tazemetostat Clinical Program
Tazemetostat, a first-in-class EZH2 inhibitor, is being evaluated as a monotherapy and in combination with other agents in multiple cancer indications. Phase 2 studies of tazemetostat as a monotherapy are currently ongoing in patients with non-Hodgkin lymphoma; in adults with INI1-negative tumors and children with certain genetically defined solid tumors, including INI1-negative tumors and synovial sarcoma; and in patients with mesothelioma characterized by BAP1 loss-of-function.
Additionally, tazemetostat is being evaluated in two combination studies in patients with DLBCL. A Phase 1b/2 trial of tazemetostat in combination with R-CHOP, an immuno-chemotherapy regimen consisting of rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone, is being conducted as a front-line treatment for patients with DLBCL as part of Epizyme’s collaboration with the Lymphoma Study Association. A Phase 1b study evaluating tazemetostat in combination with Tecentriq (atezolizumab), an anti-PD-L1 cancer immunotherapy approved by the U.S. Food and Drug Administration, is being conducted as part of a collaboration with Genentech, a member of the Roche Group, in relapsed and refractory patients with DLBCL. For more information on tazemetostat clinical trials, please visit www.epizymeclinicaltrials.com.
Chugai and Berlin-Chemie – the Menarini Group Announce Global License Agreement for an Anti-cancer Agent, PA799
On November 28, 2016 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) and Berlin-Chemie Menarini, a company of the Menarini Group, reported that both companies have entered into a global license agreement for PA799, class I PI3K inhibitor (Press release, Chugai, NOV 28, 2016, View Source [SID1234516809]). Schedule your 30 min Free 1stOncology Demo! PA799 is the PI3K inhibitor originated by Chugai, which conducted phase I study for solid tumors in Europe. With its high selectivity for class I enzyme, the drug candidate showed a good safety profile in the clinical trial.
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"We are pleased to sign a license agreement for PA799 with Menarini Group," said Chugai’s Representative Director, President and Chief Operating Officer, Tatsuro Kosaka. "PI3K is assumed to be one of the important kinases in the signaling pathway for the proliferation, the differentiation and the survival of tumor cells. We hope that the development of PA799 by Menarini Group will bring benefit to the patients as early as possible."
"We are excited about the opportunity to develop this anti-cancer agent, we believe in the mechanism of action of the PA799, and we are confident it will bring a great value to cancer patients in the world," said Reinhard Uppenkamp, Chief Executive Officer of Berlin-Chemie Menarini. "The scientific skills of Chugai and ability to conduct clinical trials and regulatory activities of Menarini Group will be in synergy for the future development of this product."
"We welcome the achievement of this strategic partnership with Chugai. This agreement allows us to add a key component in our portfolio, in fact, we make today a key step in the effort to expand the multimodal oncology pipeline of Menarini Group." said Lucia Aleotti, Chairman of Menarini Group.
Under the agreement, Chugai will grant Menarini Group an exclusive license for the manufacturing, development and marketing of PA799 worldwide. Under the terms of the agreement, Chugai will receive an upfront, milestone and royalty payments from Menarini Group.
AVEO Announces Submission of Responses to the EMA Day 120 List of Questions for Marketing Authorization Application for Tivozanib in Renal Cell Carcinoma
On November 28, 2016 AVEO Oncology (NASDAQ:AVEO) reported that its development partner, EUSA Pharma, a specialty pharmaceutical company with a focus on oncology and oncology supportive care, has submitted its responses to the European Medicines Agency (EMA) Day 120 List of Questions (Press release, AVEO, NOV 28, 2016, View Source [SID1234516807]). The Day 120 List of Questions were issued by the Committee for Medicinal Products for Human Use (CHMP) as part of the centralized review process of the Marketing Authorization Application (MAA) for tivozanib for the first-line treatment of advanced renal cell carcinoma (RCC). The next step in the filing process, the EMA Day 180 List of Outstanding Issues, is expected in the first quarter of 2017.
“This submission represents an important step forward in working toward an anticipated first half 2017 European approval decision, one of three potential key tivozanib-related milestones during this period,” said Michael Bailey, president and chief executive officer of AVEO. “Given tivozanib’s unique activity and safety profile, we believe the first line RCC market in Europe represents a meaningful commercial opportunity, and we look forward to working with EUSA Pharma to fully elucidate this potential. In addition to a potential European approval decision, we expect to have initial results from the Opdivo combination TiNivo study in RCC and may see milestones from Ophthotech for tivozanib in acute macular degeneration in the first half of 2017.”
Can-Fite Reports Financial Results for Nine Months Ended September 30, 2016 & Provides Clinical Update
On November 25, 2016 Can-Fite BioPharma Ltd. (NYSE MKT: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs being developed to treat inflammatory diseases, cancer and sexual dysfunction, reported financial results for the nine months ended September 30, 2016 and updates on its drug development programs (Filing, Q3, Can-Fite BioPharma, 2016, NOV 25, 2016, View Source [SID1234516799]).
Clinical Development Program and Corporate Highlights Include:
● Piclidenoson (CF101) – EMA Clearance Received for Phase III Trials in Rheumatoid Arthritis & Psoriasis to Commence in 2017
Rheumatoid Arthritis: Piclidenoson is being developed as a first line therapy and replacement for the current gold standard, Methotrexate (MTX), the most widely used drug for rheumatoid arthritis. The Company plans to submit its study protocol to Institutional Review Boards (IRBs) of clinical sites in the first quarter of 2017.
Rheumatoid arthritis is a treatment market forecast to reach $38.5 billion by 2017.
Psoriasis: Can-Fite reached an agreement with the European Medicines Agency (EMA) on the final design of a global pivotal Phase III trial for Piclidenoson in the treatment of psoriasis. The Phase III trial, expected to commence enrollment in the second half of 2017, will investigate the efficacy and safety of Piclidenoson compared to placebo as its primary endpoint and as compared to apremilast (Otezla) as its secondary endpoint in approximately 400 patients with moderate-to-severe plaque psoriasis.
During the third quarter, Can-Fite received a Notice of Allowance from the European Patent Office indicating the patent titled, "Pharmaceutical Composition Comprising A3 Adenosine Receptor Agonist (IB-MECA/CF-101) for Treatment of Psoriasis" will be granted.
The Journal of Drugs in Dermatology published data in August from Can-Fite’s Phase II/III trial of Piclidenoson in the treatment of moderate to severe psoriasis. The study is titled "Treatment of Plaque-Type Psoriasis With Oral CF101: Data from a Phase II/III Multicenter, Randomized, Controlled Trial."
The psoriasis market is forecast to be $8.9 billion in 2018.
● Namodenoson (CF102) – Distribution deal in South Korea; Ongoing Phase II in Liver Cancer & Phase II NAFLD/NASH Protocol Submitted to IRB
In October, Can-Fite signed a distribution agreement with Chong Kun Dang Pharmaceuticals (CKD) for the exclusive right to distribute Namodenoson for the treatment of liver cancer in South Korea. The deal includes up to $3,000,000 in upfront and milestone payments, plus a percentage of royalties on net sales in the low twenties. CKD also negotiated for a right of first refusal to distribute Namodenoson for other indications for which Can-Fite develops Namodenoson.
Liver Cancer: Can-Fite continues to enroll and dose patients in its global Phase II study of Namodenoson in the treatment of hepatocellular carcinoma, the most common form of liver cancer. A total of approximately 78 patients are expected to be enrolled in the U.S., Europe, and Israel.
Molecular Medicine Report published an article titled, "A3 adenosine receptor agonist, CF102, protects against hepatic ischemia/reperfusion injury following partial hepatectom" during the third quarter. The article reports the results of preclinical studies conducted by Can-Fite, showing Namodenoson protects the liver from ischemia/reperfusion injury and regenerates liver cells following partial hepatectomy.
Liver cancer drugs are expected to generate $1.4 billion in sales in 2019.
NAFLD/NASH: In conjunction with world renowned Key Opinion Leaders in the field of liver diseases, Can-Fite completed the study design of its upcoming Phase II trial of Namodenoson in the treatment of non-alcoholic fatty liver disease (NAFLD), the precursor to non-alcoholic steatohepatitis (NASH).
This Phase II clinical trial protocol was submitted to leading Institutional Review Boards (IRB) in Israel. Top medical centers in Israel, including Hadassah Medical Center and Rabin Medical Center are expected to participate in the planned study by enrolling and treating patients.
By 2025, the addressable pharmaceutical market for NASH is estimated to reach $35-40 billion.
● CF602 – Broad Patent Estate in Sexual Dysfunction & Other Indications
A Notice of Allowance was granted to Can-Fite by the U.S. Patent and Trademark Office in November for the Company’s patent application covering A3 adenosine receptor (A3AR) ligands for use in the treatment of erectile dysfunction. The patent addresses methods for treating erectile dysfunction with different A3AR ligands including Can-Fite’s erectile dysfunction drug candidate, CF602. With this new broader patent protection, Can-Fite has made a strategic decision to investigate additional compounds, owned by the Company, for the most effective and safest profile in this indication. As such, the Company will postpone its planned Investigational New Drug (IND) submission for this indication.
"Today Can-Fite stands poised to enter two pivotal Phase III trials in autoimmune disease where there is a clear need for an effective oral drug that can be taken safely, on a long-term basis. In the treatment of liver diseases, we look forward to completing our Phase II trial in liver cancer and commencing a Phase II trial in NAFLD/NASH. We believe our small molecule oral drug candidates offer very clear advantages over other drugs on the market that patients cannot tolerate longer term due to safety and IV administration issues. Along with benefits to patients, it is our strong belief that our drug candidates will improve healthcare economics and create growing value for our shareholders," stated Can-Fite CEO Dr. Pnina Fishman.
Revenues for the nine months ended September 30, 2016 were NIS 0.64 million (U.S. $0.17 million) compared to NIS 0.54 million (U.S. $0.14 million) in the first nine months of 2015. The increase in revenue was due to the recognition of a portion of the NIS 5.14 million (U.S. $1.36 million) upfront payment received in March 2015 under the distribution agreement with Cipher Pharmaceuticals.
Research and development expenses for the nine months ended September 30, 2016 were NIS 15.45 million (U.S. $4.11 million) compared with NIS 9.58 million (U.S. $2.55 million) for the same period in 2015. Research and development expenses for the first nine months of 2016 comprised primarily of expenses associated with the Phase II study for CF102, preclinical study for CF602, as well as expenses for ongoing studies of CF101. The increase is due to costs associated with preparations of the aforementioned studies.
General and administrative expenses were NIS 7.88 million (U.S. $2.1 million) for the nine months ended September 30, 2016 compared to NIS 6.79 million (U.S. $1.81 million) for the same period in 2015. The increase is primarily due to an increase in share based compensation expense.
Financial income, net for the nine months ended September 30, 2016 aggregated NIS 3.12 million (U.S. $0.83 million) compared to financial expenses, net of NIS 4.7 million (U.S. $1.25 million) for the same period in 2015. The increase in financial income, net in the first nine month of 2016 was mainly due to a decrease in the fair value of warrants that are accounted for as financial liability as compared to an increase in the fair value of these warrants in the same period in 2015. In addition, the increase in financial income, net in the first nine months of 2016 was offset by an increase in financial expenses due to exchange rate differences as compared to insignificant financial income from exchange rate differences and capital issuance expenses for the same period in 2015.
Can-Fite’s net loss for the nine months ended September 30, 2016 was NIS 19.56 million (U.S. $5.2 million) compared with a net loss of NIS 20.53 million (U.S. $5.46 million) for the same period in 2015. The decrease in net loss for the first nine month of 2016 was primarily attributable to an increase in financial income, net offset by an increase in research and development expenses.
As of September 30, 2016, Can-Fite had cash and cash equivalents of NIS 37.57 million (U.S. $10 million) as compared to NIS 66.03 million (U.S. $17.57 million) at December 31, 2015. The decrease in cash during the nine months ended September 30, 2016 is due to operating expenses.
For the convenience of the reader, the reported NIS amounts have been translated into U.S. dollars, at the representative rate of exchange on September 30, 2016 (U.S. $1 = NIS 3.758).
The Company’s consolidated financial results for the nine months ended September 30, 2016 are presented in accordance with International Financial Reporting Standards.
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Kymab secures US$100 million Series C funding
On November 24, 2016 Kymab Group Limited ("Kymab"), a leading monoclonal antibody biopharmaceutical group, reported that it has successfully secured a US$100 million (£81 million) Series C financing (Press release, Kymab, NOV 24, 2016, View Source [SID1234537010]). The financing was led by new investors ORI Healthcare Fund L.P. ("ORI Fund") with participation by Shenzhen Hepalink Pharmaceutical Co., Ltd ("Hepalink") as well as follow-on investments from existing shareholders: Wellcome Trust, Bill & Melinda Gates Foundation, Malin Corporation plc, CF Woodford Equity Income Fund and Woodford Patient Capital plc.
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The funds will enable Kymab to advance its proprietary pipeline of first-in-class therapeutic human monoclonal antibodies the first of which is commencing clinical development in 2017.
"We are delighted to welcome new investors ORI Fund and Hepalink and would like to thank our existing investors for their continued support in our goal of building Kymab into a sustainable global biopharmaceutical company with a pipeline of products in four main therapeutic areas: immuno-oncology, auto-immunity, haematology and infectious disease," says Dr Dave Chiswell, CEO of Kymab.
"ORI Fund and Hepalink bring deep experience of the pharmaceutical industry. Hepalink has a global reach for their products and have biologics manufacturing capability in the US. This investment will help us maximise the potential of the Kymab pipeline as we develop and commercialise monoclonal antibody medicines for patients world-wide."
"We are very fortunate to have the opportunity to invest in Kymab which is equipped with world class technologies, world class programs, a world class team and world class investors," says Ms Simone Song, Senior Partner of ORI Fund. "We look forward to working with Dave and his team to fully realise the potential of Kymab as it enters into the clinic with a global presence."
"We have had a biologics strategy for a number of years and believe Kymab has one of the most comprehensive humanised transgenic antibody platforms which is already delivering first-in-class antibodies," explains Mr Li Li, President and Chairman of Hepalink. "We are pleased to invest in a world leading antibody company and look forward to potential collaborative opportunities with Kymab"
Kymab is using the Kymouse transgenic human antibody platform to discover and develop fully human monoclonal antibody drugs. Data published in Nature Biotechnology demonstrate that the Kymouse technology yields an antibody library constituted from 100 trillion different antibodies. From this deep library rare high-quality antibodies can be selected and developed into therapeutics.
Five of the top ten best-selling drugs are antibodies. This is because antibodies are natural products with exquisite specificity and potency, and generally have superior safety profiles. The challenge has been to capture the full human antibody repertoire and to recapitulate all its attributes.
The Series C follows the US$120 million Series A and B financings.
Notes to Editors
Issued 24 November, 2016