Athenex Announces Encouraging Results of the First Cohort of Phase I Clinical Trial of Oraxol and CYRAMZA® (Ramucirumab) Combination Treatment in Gastric Cancer

On January 22, 2018 Athenex, Inc. (Nasdaq:ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported the completion of the first cohort of patients in its Phase 1b clinical trial of Oraxol (oral paclitaxel) plus CYRAMZA (ramucirumab) in gastric cancer patients that failed previous chemotherapies. Oraxol, an innovative development in the treatment of cancer, is a novel oral formulation of paclitaxel, a very effective and commonly used chemotherapy treatment for many cancers, combined with HM30181A (a novel P-gp inhibitor) (Press release, Athenex, JAN 22, 2018, View Source;p=RssLanding&cat=news&id=2327621 [SID1234523406]).

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A previously completed Phase II clinical trial of Oraxol in the second-line treatment of gastric cancer patients showed an encouraging overall median survival of 10.7 months. The expected overall survival for advanced gastric cancer patients who have failed previous chemotherapy and do not receive second-line therapy is approximately 4.0 months.

Ramucirumab, as a single agent or in combination with paclitaxel, is FDA-approved for the treatment of patients with advanced or metastatic, gastric or gastroesophageal junction (GEJ) adenocarcinoma with disease progression on or after prior fluoropyrimidine or platinum-containing chemotherapy. Ramucirumab is manufactured and marketed by Eli Lilly and Company. Athenex is evaluating the effect of Oraxol plus ramucirumab in gastric cancer in collaboration with Lilly.

Of the six patients in the first cohort, the Oraxol and ramucirumab combination treatment was well tolerated. Grade 4 neutropenia occurred in one patient who fully recovered and there were no patient deaths. There was no neuropathy. Two patients had partial responses (tumor shrinkage of 34-42%) and three patients had stable diseases (with tumor shrinkage of 27% in one patient). Only one patient had progressive disease. Although early, these results are regarded as encouraging compared with previous IV paclitaxel and ramucirumab combination therapy Phase III clinical trial results. Athenex is advancing to the second cohort with Oraxol dose escalation.

Dr. Rudolf Kwan, Athenex’s Chief Medical Officer, commented, "We are heartened to see Oraxol and ramucirumab is well-tolerated with encouraging tumor response in our first cohort of patients with gastric cancer that failed previous chemotherapies in this Phase I clinical trial. We are proceeding to the second cohort of patients with an escalation dose of Oraxol to explore the possibility of even better clinical efficacy with a higher dose."

Oraxol was initially discovered by Hanmi Pharmaceuticals and licensed to Athenex.

PharmaEssentia Corp., licensed the Taiwan rights to Oraxol from Athenex and is a partner in the development of Oraxol in Taiwan.

Athenex Announces Encouraging Early Clinical Efficacy and Safety Data of Oraxol in Clinical Trial for the Treatment of Breast Cancer

On January 22, 2018 Athenex (Nasdaq:ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported encouraging preliminary efficacy and safety data of Oraxol in the treatment of breast cancer in a pharmacokinetics (PK) and Phase I/II clinical trial of 24 patients in Taiwan (Press release, Athenex, JAN 22, 2018, View Source;p=RssLanding&cat=news&id=2327623 [SID1234523407]). Oraxol, an innovative development in the treatment of cancer, is a novel oral formulation of paclitaxel, an effective and commonly used chemotherapy treatment for many cancers, combined with HM30181A (a novel P-gp inhibitor).

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Among the first 12 evaluable patients, 50% had a partial response (>30% tumor shrinkage) and 50% had stable disease (all patients had tumor reduction, some up to 27%). There was no progressive disease. The follow-up time was only 2.6 months and such early response results are most encouraging. Grade 4 neutropenia was observed in 3 patients, all recovered completely with no patient death. There was no neuropathy.

Dr. Rudolf Kwan, Athenex’s Chief Medical Officer, commented, "We have seen consistent encouraging results in different clinical studies, showing the robust performance of Oraxol. We are delighted by the recent recommendation and positive comments from the Drug Safety and Monitoring Board, as well as the United Kingdom Medicines and Healthcare products Regulatory Agency giving Oraxol a Promising Innovative Medicine designation that will facilitate the development of Oraxol in Europe. We are very excited with these encouraging results and we are moving full speed to develop Oraxol for cancer patients in need of an effective oral paclitaxel with very low incidence of neuropathy."

Athenex previously announced that the Drug Safety and Monitoring Board unanimously recommended the continuation of our Phase III clinical trial comparing Oraxol versus intravenous paclitaxel in the treatment of metastatic breast cancer after the interim analysis of the first 90 patients on October 5, 2017. Additionally, the Company announced the receipt of the Promising Innovative Medicine designation for Oraxol by the United Kingdom Medicines and Healthcare products Regulatory Agency on December 27, 2017, qualifying Athenex to apply for Step II of the Early Access to Medicines Scheme to provide patients early access to Oraxol prior to receiving marketing authorization. Athenex also recently announced that the Chinese FDA has allowed the Investigational New Drug application for Oraxol on January 8, 2018.

Oraxol was initially discovered by Hanmi Pharmaceuticals and licensed to Athenex.

PharmaEssentia Corp., licensed the Taiwan rights to Oraxol from Athenex and is a partner in the development of Oraxol in Taiwan.

BioCryst Pharmaceuticals and Idera Pharmaceuticals Announce Merger to Combine Capabilities to Serve More Patients With Rare Diseases

On January 22, 2018 BioCryst Pharmaceuticals, Inc.(NASDAQ:BCRX), and Idera Pharmaceuticals, Inc. (NASDAQ:IDRA), reported that they have signed a definitive merger agreement to form a new enterprise focused on the development and commercialization of medicines to serve more patients suffering from rare diseases (Press release, BioCryst Pharmaceuticalsa, JAN 22, 2018, View Source [SID1234523408]). The combined company will be renamed upon closing and will be led by Vincent Milano, CEO of Idera, who will also serve as a member of the Board. BioCryst Chairman, Robert Ingram, will be Chairman of the Board of the combined company and BioCryst CEO Jon P. Stonehouse will serve as a member of the Board of Directors.

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"Both of our companies have aspired to become successful providers of therapeutics for patients suffering from rare life-threatening diseases. Both share a culture that puts patients first and keeps their interests at the very core of what we do, and how we do it," stated Vincent Milano, Idera’s Chief Executive Officer. "By merging our unique talents, experiences and assets, we instantly strengthen our ability to become a significant force for patients suffering from a broad range of rare diseases. We will also gain operational synergies and strengthen our financial position."

"The new company will have a robust late-stage pipeline with two Phase 3 candidates and two Phase 2 candidates, with several important catalysts for these programs anticipated in 2018. We will also have a deep early-stage pipeline that will continue to expand via our combined drug discovery capabilities and clinical expertise. We are extremely excited about our combined rare disease portfolio. We believe we will be well positioned to bring that portfolio to market with our proven commercial leadership team and business development opportunities."

"Bringing these two companies together accelerates the strategic initiatives of both organizations and immediately forms a substantial and differentiated biotech company serving patients in the rare disease community," stated Jon P. Stonehouse, BioCryst’s President and Chief Executive Officer. "Combining our respective pipelines, infrastructures and financial resources should enable the new company to grow faster, deliver for patients more rapidly and ultimately create sustainable shareholder value well beyond what either would achieve separately."

The combination of two companies is expected to capitalize on the collective skills sets, internal expertise and combined assets to create a comprehensive, sustainable rare disease-focused biotechnology leader highlighted by:

A robust development pipeline, including 4 late stage programs that provide near-term commercial and partnering opportunities:
BCX7353 – Phase 3 program for the prophylactic treatment of Hereditary Angioedema (HAE) in a capsule formulation with FDA orphan drug designation
IMO-2125 – Phase 3 program for the treatment of PD1-refractory melanoma in combination with ipilimumab with FDA orphan drug designation
IMO-8400 – Phase 2 program for dermatomyositis
BCX7353 – Phase 2 program for the acute treatment of HAE in a liquid formulation
Proven leadership across commercial, development, scientific and clinical functions, providing a combination of management and scientific talent that marries the necessary ingredients for a successful, sustainable biotech company.
Synergistic discovery engines highlighted by two distinct research technologies and expertise which expands the number of rare disease therapeutic targets and candidates.
Financial strength with approximately $243 million net cash balance (unaudited proforma cash balance as of December 31, 2017), with opportunities to add further non-dilutive capital to fund internal clinical development efforts, commercial launch efforts, and continued business development activities.
Transaction Summary
Under the terms of the merger agreement, each share of BioCryst common stock will be exchanged for 0.50 shares of the new company stock and each share of Idera common stock will be exchanged for 0.20 shares of the new company stock. The exchange ratio reflects an "at market" combination based upon the approximate 30-day average volume weighted trading prices for each company. On a proforma, fully diluted basis, giving effect to all dilutive stock options, units and warrants, BioCryst stockholders will own 51.6 percent of the stock of the combined company and Idera stockholders will own 48.4 percent. The stock issuance in the merger is expected to be tax-free to stockholders.

The merger agreement has been unanimously approved by the boards of directors of both companies. The transaction is subject to approval by the stockholders of both companies, as well as regulatory approvals and satisfaction of other customary closing conditions. A significant stockholder of each company has agreed to enter into a voting and support agreement and has agreed to vote in favor of the transaction. This stockholder owns approximately 9% of Idera shares outstanding and approximately 14% of BioCryst shares outstanding. The transaction is expected to be completed by the end of the second quarter of 2018.

The combined company, which will be renamed post-closing, will be headquartered in Exton, PA, at the current Idera headquarters, with a consolidated
research center in Birmingham, AL. In addition to Mr. Milano’s role as CEO of the combined company, Dan Soland will join the combined company and will serve in the role of Chief Operating Officer.

J.P. Morgan Securities LLC acted as exclusive financial advisor to BioCryst and Skadden, Arps, Slate, Meagher & Flom, LLPacted as BioCryst’s legal counsel in connection with the transaction. Goldman Sachs & Co. LLC acted as exclusive financial advisor to Idera and Latham & Watkins, LLP acted as Idera’s legal counsel in connection with the transaction.

Investor Event and Webcast
BioCryst and Idera will host a conference call and live webcast on Monday, January 22, 2018 at 10:00 A.M. ET to discuss the proposed merger and to answer questions to investors and analysts. To participate in the conference call, please dial (844) 882-7837 (domestic) and (574) 990-9824 (international). The webcast can be accessed live or in archived form in the "Investors" section of the companies’ websites at www.biocryst.com and www.iderapharma.com. A related slide presentation has been posted to each companies’ corporate websites in the "Investors" section which will be referenced during the conference call.

Investigational New Drug (IND) Application for EP4 antagonist AAT-007 for Oncology Filed in China

On January 22 2018 AskAt reported Ningbo NewBay Medical Technology Co., Ltd. (Headquarters in Ningbo, China, CEO: Zhenhai Shen, NewBay), a subsidiary company of Ningbo Tai Kang Medical Technology Co., Ltd ("Ningbo Tai Kang"), which licensed AskAt Inc.’s EP4 antagonist AAT-007 (generic name: grapiprant) for oncology in China, has filed an IND application with the China Food and Drug Administration (Press release, AskAt, JAN 22, 2018, View Source [SID1234535057]).

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AskAt Inc. will receive a milestone payment according to the terms of the License Agreement executed between Ningbo Tai Kang and AskAt as a result of achieving this filing.

Laminar Pharma awarded a 6,15M€ grant by the H2020 Programme to conduct a PIIb trial with 2OHOA in patients with newly-diagnosed glioblastoma

On January 21, 2018 Laminar Pharma, a pioneering clinical stage biopharmaceutical company developing a new generation of products modulating metabolism of membrane lipids based on the groundbreaking MLT platform, reported that the European Commission has awarded a 6,15M€ grant to the CLINGLIO consortium, lead by Laminar Pharma, to execute the project entitled "A Clinical Phase IIB trial with 2OHOA in patients with newly-diagnosed malignant glioma" (Press release, Laminar Pharma, JAN 21, 2018, View Source [SID1234562095]).

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The CLINGLIO project was evaluated as a Research and Innovation Action (RIA) within the call H2020-SC1-2017-Two-Stage-RTD, Topic SC1-PM-08-2017 (New therapies for rare diseases), part of the Health, Demographic Change and Well-being Work Programme of the H2020. Total budget available for this H2020-SC1-2017-Two-Stage-RTD call was 173M€, of which 65M€ were assigned to topic SC1-PM-08-2017. Overall, 668 proposals were submitted to this call, of which 37 were pre-selected for funding across the four topics of the call (5,5% of the initial proposals). In the New Therapies for Rare Diseases topic, the EC will select for funding up to 11 proposals with an average budget per project of around 6M€.

The grant has been awarded to a multinational, well balanced consortium formed by 5 leading clinical research institutions in the UK (Royal Marsden Hospital and Northern Institute for Cancer Research, University ofNewcastle upon Tyne), France (Institut Gustave Roussy), Italy (Istituto Neurologico Carlo Besta) and Israel (Hadassah Medical Organization), two universities in Spain (Universitat de les Illes Balears, UIB) and Italy (Universita degli Studi di Salerno) and 4 specialized SMEs from The Netherlands (SMS Oncology, clinical CRO), Hungary (Lipodom Kft, lipidomic analysis), USA (LMBRI, pharmacoeconomics and market access) and Spain (Praxis Pharmaceutical, Drug product manufacturing and commercialization, and Laminar Pharma, coordinator and sponsor of the clinical trial).

The main objective of the CLINGLIO project is to execute a randomised, double-blind, placebo-controlled adjuvant trial in primary newly diagnosed glioblastoma patients to assess the efficacy and safety of 2OHOA in combination with radiotherapy and temozolomide. This study is a phase IIB adaptive trial with interim dose selection, sample size reassessment and biomarker threshold/omics signature determination. It is anticipated that around 15 clinical research hospitals across Europe and Israel will recruit 150 patients in the first part of the study, distributed in three arms: 1) control, with Standard of Care (SoC) plus placebo, 2) SoC plus 2OHOA "low dose" and 3) SoC plus 2OHOA "high dose". An interim analysis will take place after 75 events (Disease Progression) occurs and depending on the results of this interim analysis 60 to 120 additional patients will be enrolled in the second part of the study. The primary endpoint will be Progression Free Survival, according to RANO criteria, and Overall Survival will be a key secondary endpoint.

If the results of this clinical trial are positive, Laminar Pharma plans to apply for a conditional approval of 2OHOA in Europe for the treatment of newly diagnosed GBM patients, in combination with radiotherapy and temozolomide