On January 4, 2019 BioNTech AG, a rapidly growing biotechnology company developing precise and individualized immunotherapies for the treatment of cancer and the prevention of infectious diseases, reported that it has extended its research collaboration with Sanofi initiated in late 2015 and Sanofi is investing* EUR 80 million (approximately USD 91.5 million) in equity in BioNTech (Press release, BioNTech, JAN 4, 2019, View Source [SID1234532446]).

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Simultaneously BioNTech has entered into an agreement with Sanofi to co-develop the first cancer immunotherapy candidate from the collaboration that is entering clinical testing in multiple solid tumors. This follows BioNTech’s decision from early 2018 to exercise one of its option rights under the 2015 research collaboration to co-develop and co-commercialize this product candidate with Sanofi.

The investigational therapy consists of an mRNA mixture encoding immunomodulatory cytokines that are injected directly into the tumor. Local administration of immunotherapies to the tumor microenvironment provides the opportunity to stimulate innate and adaptive immune responses against tumors, while potentially avoiding toxicities related to systemic administration of immuno-modulatory therapeutics.

"The extension of the research collaboration and equity investment demonstrates a deepening of our partnership with Sanofi as we seek to rapidly drive novel, disruptive programs through clinical testing to commercialization together," said Prof. Dr. Ugur Sahin, Co-Founder and CEO of BioNTech. "We are pleased that with this program we have moved from concept to clinical stage in well under three years."

"We are pleased to be initiating clinical testing of our first mRNA-based cancer immunotherapy in collaboration with our partners at BioNTech," commented Yong-Jun Liu, Global Head of Research, Senior Vice President R&D, Sanofi. "Our joint team, consisting of scientists and clinicians from both Sanofi and BioNTech, has worked tirelessly to expedite development of this new and innovative clinical candidate because targeted mRNA therapies may have the potential to be effective for cancer patients. We look forward to continuing a productive partnership with BioNTech in the years to come."

On January 4, 2019 Alder BioPharmaceuticals, Inc. reported that it has entered into a Distribution Agreement (the "Distribution Agreement") with J.P. Morgan Securities LLC, as our sales agent (the "Sales Agent"), pursuant to which we may offer and sell from time to time through the Sales Agent up to $100,000,000 maximum aggregate offering price of our common stock, par value $0.0001 per share ("Common Stock"), in such amounts as we may specify by notice to the Sales Agent, in accordance with the terms and conditions set forth in the Distribution Agreement (Filing, 8-K, Alder Biopharmaceuticals, JAN 4, 2019, View Source [SID1234532445]).

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Sales, if any, of the Common Stock pursuant to the Distribution Agreement may be made in negotiated transactions or transactions that are deemed to be "at-the-market" offerings as defined in Rule 415 under the Securities Act, including sales made directly on the Nasdaq Stock Market, or sales made to or through a market maker other than on an exchange. Under the Distribution Agreement, we will set the parameters for the sale of shares, including the number of shares to be issued, the time period during which sales are requested to be made, limitation on the number of shares that may be sold in any one trading day and any minimum price below which sales may not be made. We are not obligated to sell any Common Stock under the Distribution Agreement. The Common Stock will be offered and sold pursuant to our shelf registration statement on Form S-3 (File No. 333-216199) which was automatically effective upon filing with the Securities and Exchange Commission on February 23, 2017. We may terminate the Distribution Agreement upon written notice to the Sales Agent for any reason or by the Sales Agent upon written notice to us for any reason or at any time under certain circumstances, including but not limited to the occurrence of a material adverse change in our company.

The Distribution Agreement contains customary representations, warranties and agreements by us, and indemnification rights and obligations of the parties. The Distribution Agreement provides that the Sales Agent will be entitled to compensation for its services equal up to 3.0% of the gross sales price per share of all shares sold through the Sales Agent under the Distribution Agreement. Under the terms of the Distribution Agreement, we have agreed to indemnify the Sales Agent against certain specified types of liabilities, including liabilities under the Securities Act of 1933, as amended, to contribute to payments the Sales Agent may be required to make in respect of these liabilities, and to reimburse the Sales Agent for certain expenses. In the ordinary course of business, the Sales Agent or their respective affiliates from time to time have provided and may in the future provide various investment banking, commercial banking and financial advisory services to the company and/or its affiliates, for which they have received or may receive customary compensation.

We intend to use the net proceeds from the sale, if any, of the securities offered in the offering for the commercialization of eptinezumab up to and through launch and the manufacture of commercial supply for eptinezumab, and may also use net proceeds for future eptinezumab clinical trials, the development of ALD1910 and for working capital and general corporate purposes.

The above summary of the Distribution Agreement does not purport to be complete and is qualified in its entirety by reference to the Distribution Agreement, a copy which is attached as Exhibit 1.1 to this Current Report on Form 8-K and incorporated herein by reference. The legal opinion of Cooley LLP relating to the shares of Common Stock being offered pursuant to the Distribution Agreement is filed as Exhibit 5.1 to this Current Report on Form 8-K.

This Current Report shall not constitute an offer to sell or the solicitation of an offer to buy the securities discussed herein, nor shall there be any sale of such securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On January 4, 2019 Epizyme, Inc. (Nasdaq: EPZM), a clinical-stage company developing novel epigenetic therapies, reported a comprehensive set of pipeline updates, including that the company has identified a path to submission for accelerated approval of tazemetostat for patients with relapsed and/or refractory follicular lymphoma (FL), both with and without EZH2 activating mutations (Press release, Epizyme, JAN 4, 2019, View Source [SID1234532444]). The company recently conducted a productive meeting with the U.S. Food and Drug Administration (FDA) to discuss the FL registration strategy based on the current patient population in its ongoing Phase 2 clinical trial. Following the discussion, Epizyme has defined a registration strategy for tazemetostat in both EZH2 mutant and wild type FL patient populations, where patients’ disease has progressed following two or more lines of therapy. Based on this, the company anticipates submitting a New Drug Application (NDA) for this indication in the fourth quarter of 2019. In addition, the company provided an update on its clinical and preclinical pipeline and anticipated milestones for 2019.

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"Follicular lymphoma is an incurable cancer today, and in the third line and later settings, there are limited effective treatment options. Defining a clear path to a regulatory submission for tazemetostat for this patient population marks a huge step forward for patients and an opportunity to change the course of FL treatment," said Shefali Agarwal, M.D., chief medical officer of Epizyme. "This FL NDA submission would mark the second for tazemetostat in one year, following our first submission for epithelioid sarcoma, which is on track for the second quarter of 2019. If successful, tazemetostat is poised to be the first commercially available EZH2 inhibitor. We look forward to advancing our submission preparations and further engaging with FDA, as we work expeditiously to bring tazemetostat to the patients who need it."

Tazemetostat Registration Update for Follicular Lymphoma

Phase 2 Study Fully Enrolled: The ongoing Phase 2 study has been fully enrolled and based on discussions with FDA, is expected to provide the necessary relapsed and/or refractory FL patients needed for an NDA submission, with 45 patients with EZH2 activating mutations and 54 patients with wild-type EZH2.

Registration Path Identified for NDA Submission in Follicular Lymphoma: Epizyme recently met with FDA to review its planned registration strategy for tazemetostat for patients with FL who have been previously treated with two or more systemic therapies, which represents a population of unmet medical need. The company has identified a path to a submission for accelerated approval for patients with both mutant and wildtype EZH2, based on the ongoing Phase 2 study. Epizyme will further advance the Phase 2 study, with updated data to be reported at a medical meeting in mid-2019 and an NDA submission targeted for the fourth quarter of 2019.

Confirmatory Program Could Support Expansion into Earlier Treatment Lines of Follicular Lymphoma: As part of an accelerated approval strategy, Epizyme plans to conduct a confirmatory program to support full approval of tazemetostat in FL, while also supporting its potential expansion into the second-line treatment of FL. Under its Fast Track designation, the company intends to engage with FDA in the first half of 2019 to discuss the confirmatory program and will share details upon initiation.

Tazemetostat Registration Update for Epithelioid Sarcoma

NDA Submission for Epithelioid Sarcoma on Track for Second Quarter of 2019: Epizyme is advancing preparations for its first NDA submission for tazemetostat in the second quarter of 2019 using the accelerated approval pathway for the treatment of patients with epithelioid sarcoma (ES). ES is an ultra-rare and difficult-to-treat sarcoma with no specifically indicated FDA-approved therapies today. If approved, tazemetostat could be the first treatment specifically indicated for patients with ES. The company has begun pre-commercial activities, with plans to commercialize tazemetostat on its own in the U.S.

Tazemetostat Program Expansion Updates

Combination Study to Begin in Follicular Lymphoma in 2019: Based on the monotherapy efficacy and safety data generated to-date, Epizyme plans to explore the potential of tazemetostat in earlier lines of FL as combination therapy. The company is assessing the opportunity to conduct a combination study that would compare the chemo-free combination of rituximab and Revlimid (R2) with tazemetostat versus R2 with placebo in patients with relapsed or refractory FL. Epizyme plans to provide an update on its combination study plans once they have been finalized.

R-CHOP Combination Data Further Support Tazemetostat Combination Potential: Under its collaboration agreement with Epizyme, the Lymphoma Study Association (LYSA) reported data at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on the combination of tazemetostat with R-CHOP as a front-line treatment for patients with diffuse large B-cell lymphoma. The data showed that the combination of the two agents was generally well-tolerated, confirmed the recommended tazemetostat dose for the combination to be 800mg twice-daily and demonstrated clinical activity, with 87 percent of patients experiencing a metabolic complete response. Based on these data, Epizyme is considering opportunities to expand the evaluation of this combination into patients with FL.

Plans in Place to Expand Tazemetostat into New Indications and Combinations: Based on its mechanism of action, favorable safety reported and demonstrated activity in multiple tumor types and treatment settings, Epizyme plans to expand tazemetostat’s potential utility into additional combinations and indications. The company has identified the next set of clinical assessments for tazemetostat, including a combination study in castration-resistant prostate cancer that is slated to begin in mid-2019, and assessment in platinum-resistant tumors, such as small-cell lung cancer, triple-negative breast cancer and ovarian cancer, slated to begin in the second half of 2019.

Genentech and Epizyme to Close Tecentriq Combination Assessment in NSCLC: As part of a collaboration agreement, Genentech/Roche initiated assessment of tazemetostat in combination with Tecentriq for the treatment of non-small cell lung cancer (NSCLC) in an arm of its MORPHEUS NSCLC Trial. Before patients had been enrolled in the study, recruitment was halted due to the partial hold placed on tazemetostat studies. Epizyme has since reopened enrollment in the United States and Germany for studies for which it is the sponsor. Due to the hold and strategic reprioritizations, the companies have jointly opted not to move forward with the NSCLC combination study.

Preclinical and Discovery Pipeline Update

EZM8266 on Track to Begin Clinical Development: Throughout 2018, Epizyme conducted IND-enabling studies on its next development candidate, EZM8266, a novel G9a inhibitor for the treatment of sickle cell disease. The company is on track to begin clinical development of EZM8266 in the second half of 2019 with a dose-finding and safety study.

Two Research Programs to Be Advanced under Boehringer Ingelheim Collaboration: In November 2018, Epizyme entered a strategic collaboration with Boehringer Ingelheim focused on the research, development and commercialization of novel small molecule inhibitors directed toward two previously unaddressed epigenetic targets as potential therapies for people with cancer. Specifically, these targets are enzymes within the helicase and histone acetyltransferase (HAT) families that when dysregulated have been linked to the development of cancers that currently lack therapeutic options.

Updated Financial Guidance

Based on enhanced operating efficiencies, partner revenues and proceeds from the company’s underwritten public offering completed in October 2018, Epizyme has extended its expected capital runway into the second quarter of 2020 based on current operating plans.

"We are at a point in our company’s evolution where we are beginning to realize the true value of all of the hard work to which we have dedicated ourselves over the past several years. 2019 is set to be a year of pivotal milestones for Epizyme, providing validation of our expertise in drug development and bringing us closer to achieving our mission of helping patients," said Robert Bazemore, president and chief executive officer of Epizyme. "With defined registration paths for tazemetostat in two indications and plans to expand into other combinations and indications, tazemetostat has the potential to generate significant value for the patients and physicians who need new treatment options, and for Epizyme. Outside of tazemetostat, our research capabilities provide additional advantages to our company, and we are excited to be moving EZM8266 into the clinic and working with our partners to advance earlier programs. I am proud of what we have accomplished, and look forward to what is ahead as we transition to a commercial-stage company that can truly have an impact on patients."

Conference Call Information

Epizyme will host a conference call today, Jan. 4, at 8:30 a.m. ET to review this corporate update. To participate in the conference call, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 7289720. A live webcast and slides will be available in the investor section of the company’s website at www.epizyme.com. The webcast and slides will be archived for 60 days following the call and presentation.

On January 4, 2019 BerGenBio ASA (OSE: BGBIO), reported that the first patient has been dosed in an investigator-initiated phase II trial of bemcentinib, a selective, potent and orally bio-available AXL inhibitor, in patients with high-risk myelodysplastic syndrome (MDS) who have failed first-line treatment with hypomethylating agents (Press release, BerGenBio, JAN 4, 2019, View Source [SID1234532440]). The trial may also enrol a proportion of patients with acute myeloid leukaemia (AML). The study is being sponsored by GWT-TUD GmbH (a specialist cancer clinical research institution associated with the University of Dresden, Germany) with the support of BerGenBio.

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The trial (BGBIL009 / BERGAMO) aims to confirm the efficacy of bemcentinib monotherapy in patients with high-risk MDS and AML and will enrol up to 43 patients at 8 hospitals in Germany, France, the Netherlands and Italy. The study will allow for the evaluation of potential predictive and pharmacodynamic biomarkers for MDS in bone marrow and blood, including those associated with patient benefit from bemcentinib.

Prof. Uwe Platzbecker, lead investigator of the trial and director of the Medical Clinic and Policlinic 1, Hematology and Cell Therapy at the University Hospital in Leipzig, Germany, commented: "As treatment of MDS and AML has not changed significantly over the past decades, novel therapies are urgently needed. The survival is still dismal, especially in elderly patients who are not eligible for allogeneic stem cell transplantation and who have failed first line treatment with hypomethylating agents. AXL, a member of the Tyro3, AXL, Mer (TAM) receptor family, mediates proliferation and survival of leukemic cells and is upregulated upon cytostatic treatment. Pre-clinical studies with the inhibitor bemcentinib demonstrated in vitro and in mouse models that leukaemic proliferation was blocked by interference with AXL signalling. Hence, AXL represents a promising new target for the patient population investigated in the BERGAMO trial."

Richard Godfrey, Chief Executive Officer of BerGenBio, added: "We are very pleased that Prof. Platzbecker and GWT-TUD are initiating this study, which if positive will add valuable information on bemcentinib’s monotherapy use in a larger leukaemia population and provide support for our broader development plans for bemcentinib in these indications. We look forward to reporting results as the trial progresses."

END

About MDS
Myelodysplastic syndromes (MDS) are stem cell disorders characterised by a decreased ability of the bone marrow to produce normal blood cells and platelets. MDS is associated with increased risk of developing AML and immune dysfunctions are seen in patients both with lower and higher-risk MDS. Drugs that modify immunological responses can improve blood values and prolong survival in some patients. Thus far, however, the only curative treatment for MDS remains stem cell transplantation. Hence, there is an urgent need for novel therapies to treat MDS.

About AXL
AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms underlying aggressive and life-threatening diseases. In cancer, AXL drives tumour survival, treatment resistance and spread, as well as suppressing the body’s immune response to tumours. AXL expression has been established as a negative prognostic factor in many cancers. AXL inhibitors, therefore, have potential value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities.

About Investigator-sponsored trials
Investigator-sponsored clinical trials are clinical trials proposed by front-line patient-facing physicians who act as the regulatory sponsor and are supported by industry in bespoke clinical development partnerships. The industry partner does not assume the role of sponsor according to European or US regulatory guidelines but may offer support in a variety of different ways, such as providing investigational medicinal product at no cost

On January 4, 2019 Lamellar Biomedical Limited (Lamellar), an innovative biotechnology company, pioneering new approaches for the safe and effective transfer of functional nucleic acids, reported that Dr Alec McLean, CEO; Steven Porteous, Head of Clinical and Regulatory and Dr Lynsey Howard, Head of Pre-clinical Development will be attending Biotech Showcase in San Francisco on 7-9 January 2019 (Press release, Lamellar Biomedical, JAN 4, 2019, View Source [SID1234532438]).

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During the event the Lamellar team will conduct multiple business development meetings with potential partners and investors to highlight the potential of its cutting-edge LAMELLASOME technology for the delivery of nucleic acid therapeutics (NAT). These meetings will build on the very positive reaction to its LAMELLASOME platform, when it was introduced at BIO-Europe 2018 in Copenhagen in November 2018.

Alec McLean, CEO of Lamellar Biomedical, said, "We were very pleased with the reaction from potential partners and investors when we introduced our LAMELLASOME platform for the safe and effective delivery of nucleic acid therapeutics at BIO-Europe late last year. We are now looking forward to multiple new and follow up meetings in San Francisco reflecting the explosion in the number of novel nucleic-acid based therapeutics being developed for a broad range of diseases. Two key factors limiting their progress, from preclinical promise to clinical reality, are safe and effective delivery. Based on our work to-date, Lamellar is confident that it is in an unique position to unlock this enormous potential of nucleic acid based therapeutics using its LAMELLASOME technology."

Lamellar believes that its LAMELLASOME technology will play a key role in realising the potential of many of the nucleic acids in development, including mRNAs, siRNAs, miRNAs and plasmids, potentially revolutionising the treatment of numerous rare and intractable diseases.

In October 2018, Lamellar filed a patent detailing that it had demonstrated functional target downregulation by a transfected nucleic acid delivered via its LAMELLASOME technology. Lamellar has also shown that LAMELLASOME formulations have been effective in delivering functional nucleic acids to a range of cell types including macrophages, human pulmonary fibroblasts and human dendritic cells.

Crucially, all transfected cells types have exhibited very high cell viability and maintenance of cell phenotype/function. Lamellar’s LAMELLASOME technology is entirely novel and does not utilise viral vectors or cationic or divalent ion-associated liposomal systems.

Lamellar is also developing its own pipeline of nucleic-acid based therapeutics, the most advanced of which target two areas of unmet clinical need: Idiopathic Pulmonary Fibrosis and Cystic Fibrosis.

Lamellar is currently completing in-vivo models to demonstrate safe and effective pulmonary delivery and transfection of microRNA(s) prior to executing the final proof of concept efficacy study for its Idiopathic Pulmonary Fibrosis microRNA product in Q1 2019. These studies will be designed to show the performance of LAMELLASOME IPF-NA product in-vivo as well as to demonstrate the unique capabilities of LAMELLASOME nucleic acid transfer technology platform.

The Lamellar team will be available to present the proprietary LAMELLASOME technology to any companies attending Biotech Showcase who see enhanced delivery of their novel nucleic-acid based therapeutics as crucial to their future success.