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.

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.

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.

On January 22, 2018 Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, "Astellas") reported that the European Commission (EC) has issued Orphan Designation to gilteritinib for the treatment of patients with acute myeloid leukaemia (AML) (Press release, Astellas, JAN 22, 2018, View Source [SID1234523405]). The decision follows a positive recommendation for Orphan Designation from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP). In Europe, an Orphan Designation is granted to a medicine that may be of significant benefit to patients with a rare condition, affecting no more than five in 10,000 people.1

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The announcement follows the recent Orphan Drug Designation in the United States granted by the US Food and Drug Administration (FDA) to gilteritinib on July 13, 2017.2

"Around 13,000 people will be diagnosed with AML in Western Europe and, while AML patients constitute a small proportion of the overall population, they are faced with a life-threatening condition," said Steven Benner, M.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development, Astellas. "We are grateful to the EMA for acknowledging the unique needs of patients with rare diseases, and for providing a potential path forward for gilteritinib in supporting these patients."

AML is a cancer that impacts the blood and bone marrow, and its incidence increases with age.3 In Western Europe, there are around 13,000 new cases of AML every year.4 In Japan, approximately 4,500 patients are diagnosed with AML each year.5,6 The American Cancer Society estimates that in 2017 approximately 21,000 new patients will be diagnosed with AML in the United States and about 10,000 cases will result in death.7

About Gilteritinib
Gilteritinib is an investigational compound that has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) as well as FLT3 tyrosine kinase domain (TKD),8 two common types of FLT3 mutations that are seen in approximately one-third of patients with AML.9 Further, gilteritinib has also demonstrated inhibition of the AXL receptor in AML cell lines.8 Astellas is currently investigating gilteritinib in various FLT3 mutation-positive AML patient populations through several Phase 3 trials.10,11 Visit www.clinicaltrials.gov to learn more about ongoing gilteritinib clinical trials.

Gilteritinib was discovered through a research collaboration with Kotobuki Pharmaceutical Co., Ltd., and Astellas has exclusive global rights to develop, manufacture and potentially commercialise gilteritinib. Gilteritinib has been granted

ONC/17/0046/APEL
Date of preparation: January 2018

Orphan Drug designation2 and Fast Track Designation12 by the U.S. FDA, and SAKIGAKE Designation by the Japan Ministry of Health, Labour and Welfare.13

The safety and efficacy of the agent discussed herein are under investigation and have not been established. There is no guarantee that the agent will receive regulatory approval and become commercially available for the uses being investigated.

On January 22, 2018 Genmab A/S (Nasdaq Copenhagen: GEN) reported that Novartis (SIX Swiss Exchange: NOVN) intends to transition Arzerra (ofatumumab) for the treatment of certain chronic lymphocytic leukemia (CLL) indications from commercial availability to limited availability via compassionate use programs in all markets around the world except the United States (Press release, Genmab, JAN 22, 2018, View Source [SID1234523372]). Novartis will work with regulatory authorities to establish compassionate use programs for patients outside the US currently being treated with Arzerra as an ongoing treatment, to ensure continuity of treatment for all patients who benefit from Arzerra. As a consequence, Novartis will pay Genmab a lump sum of USD 50 million as payment for lost potential milestones and royalties. This amount will be included in Genmab’s 2018 guidance which will be issued on February 21, 2018. Royalties will continue to be earned on net sales of Arzerra. Arzerra is marketed under a collaboration agreement between Genmab and Novartis.

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"Novartis’ intention to transition Arzerra to compassionate use programs in the non-US markets reflects the fact that many more drugs have become available for CLL over the last five years and that there is a low number of patients using Arzerra outside of the US market. The compassionate use programs will ensure that patients who benefit from Arzerra can remain on treatment. We welcome the continued commercial availability of Arzerra for US patients," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Patients accessing the compassionate use program will be offered continued treatment with Arzerra for as long as they benefit from it, free of charge.

Novartis intends to start the transition process as soon as a carefully structured plan has been agreed with the various regulatory authorities.

The two Phase III studies of ofatumumab currently ongoing in Relapsing Multiple Sclerosis and the study of Arzerra in indolent non-Hodgkin lymphoma will continue.

About Ofatumumab (Arzerra)
Ofatumumab is a human monoclonal antibody that is designed to target the CD20 molecule found on the surface of chronic lymphocytic leukemia (CLL) cells and normal B lymphocytes.

In the United States, Arzerra is approved for use in combination with chlorambucil for the treatment of previously untreated patients with CLL for whom fludarabine-based therapy is considered inappropriate, for use in combination with fludarabine and cyclophosphamide for the treatment of patients with relapsed CLL, and for extended treatment of patients who are in complete or partial response after at least two lines of therapy for recurrent or progressive CLL. In the European Union, Arzerra is currently approved for use in combination with chlorambucil or bendamustine for the treatment of patients with CLL who have not received prior therapy and who are not eligible for fludarabine-based therapy and in combination with fludarabine and cyclophosphamide for adult patients with relapsed CLL. In more than 60 countries worldwide, including the United States and EU member countries, Arzerra is also currently indicated as monotherapy for the treatment of patients with CLL who are refractory after prior treatment with fludarabine and alemtuzumab.

Please consult the full European Summary of Product Characteristics and full US Prescribing information, including Boxed Warning, for all the labeled safety information for Arzerra.

Under the collaboration with Novartis, a subcutaneous formulation of ofatumumab is also being investigated in two Phase III clinical studies in relapsing multiple sclerosis.

Novartis has rights to develop ofatumumab in autoimmune indications, including multiple sclerosis.