On January 3, 2019 Exicure, Inc. (OTCQB: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional spherical nucleic acid (SNA) constructs, reported that its CEO, Dr. David Giljohann, will present a corporate update on Tuesday, January 8, 2019 from 2:30 pm to 3:00 pm PT at the Biotech Showcase 2019 (Press release, Exicure, JAN 3, 2019, View Source [SID1234532412]). The presentation will be in the Franciscan A room on the Ballroom level of the Hilton San Francisco Union Square.

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A live audio webcast will be available on the Investors section of Exicure’s website: www.exicuretx.com. The webcast will be archived for approximately 30 days following the event.

On January 3, 2019 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Codiak BioSciences, Inc. reported that the companies have entered into a strategic collaboration agreement focused on the research, development and commercialization of exosome therapeutics to treat cancer (Press release, Jazz Pharmaceuticals, JAN 3, 2019, View Source [SID1234532428]). Codiak granted Jazz an exclusive, worldwide, royalty-bearing license to develop, manufacture and commercialize therapeutic candidates directed at five targets to be developed using Codiak’s engEx precision engineering platform for exosome therapeutics. The targets focus on oncogenes that have been well validated in hematological malignancies and solid tumors but have been undruggable with current modalities, including NRAS and STAT3.

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Under the terms of the agreement, Codiak is responsible for the execution of pre-clinical and early clinical development of therapeutic candidates directed at all five targets through Phase 1/2 proof of concept studies. Following the conclusion of the applicable Phase 1/2 study, Jazz will be responsible for future development, potential regulatory submissions and commercialization for each product. Codiak has the option to participate in co-commercialization and cost/profit-sharing in the U.S. and Canada on up to two products.

As part of the agreement, Jazz will pay Codiak an upfront payment of $56 million. Codiak is eligible to receive up to $20 million in preclinical development milestone payments across all five programs. Codiak is also eligible to receive milestone payments totaling up to $200 million per target based on Investigational New Drug application acceptance, clinical and regulatory milestones, including approvals in the U.S., European Union and Japan, and sales milestones. Codiak is also eligible to receive tiered royalties on net sales of each approved product, with percentages ranging from mid-single digits in the lowest tier to high teens in the highest tier.

"We are looking forward to partnering with Codiak and their world-class management and R&D team. Their innovative exosome platform offers the opportunity to address targets that have been frequently implicated in hematological malignancies and solid tumors, including targets recognized as undruggable with current approaches," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "The exosome approach is differentiated and represents the ideal complement to our CombiPlex platform and other collaboration programs. The ability to develop multiple new therapies through these technologies is an important advance as we seek to add long-term value for patients and shareholders."

"We are excited to be working with the team at Jazz to bring a new class of medicines into clinical testing to treat patients with cancer," said Douglas E. Williams, PhD, president and chief executive officer of Codiak. "We believe that the Codiak engEx platform, along with Jazz’s insights into relevant but intractable targets, creates a unique opportunity to improve patient outcomes in various cancers. Codiak’s proprietary engEx platform allows us to deliver potent therapeutics in a precise, targeted and tunable way. This collaboration allows Codiak to accelerate the potential of our engEx platform as we advance our lead candidates and core pipeline."

Exosomes are naturally occurring, nanometer-sized vesicles that are released and received by nearly all cells in the body. They serve as an efficient system of intercellular communication. In the tumor microenvironment, exosomes have been shown to carry molecular messages between tumor and immune cells. Codiak has co-opted this process and, through its engEx platform, engineers potent drug molecules into the exosome to create bespoke therapeutic candidates which have shown potent preclinical antitumor activity.

With the engEx platform, Codiak has the ability to incorporate a range of therapeutic drug classes—including small molecules, proteins, peptides, cytokines and nucleic acids—onto the surface or in the lumen of its therapeutic exosomes. engEx also enables precise modifications of the engineered exosomes to optimize potency and to alter or enhance tropism for directed delivery to desired cell types. These flexible design features allow Codiak to create an optimized exosome therapeutic for the drug target and desired site of action.

Jazz Pharmaceuticals Conference Call Details
Jazz Pharmaceuticals and Codiak will host an investor conference call and live audio webcast on Friday, January 4, 2019 at 8:30 a.m. EST (1:30 p.m. GMT) to discuss the collaboration. The live webcast may be accessed from the Investors section of Jazz Pharmaceuticals’ website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 9698918.

A replay of the conference call will be available through January 11, 2019 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 9698918. An archived version of the webcast will be available for at least one week in the Investors section of Jazz Pharmaceuticals’ website at www.jazzpharmaceuticals.com

On January 3, 2018 Bristol-Myers Squibb Company (NYSE:BMY) and Celgene Corporation (NASDAQ:CELG) reported that they have entered into a definitive merger agreement under which Bristol-Myers Squibb will acquire Celgene in a cash and stock transaction with an equity value of approximately $74 billion (Press release, Bristol-Myers Squibb, JAN 3, 2019, View Source [SID1234532379]). Under the terms of the agreement, Celgene shareholders will receive 1.0 Bristol-Myers Squibb share and $50.00 in cash for each share of Celgene. Celgene shareholders will also receive one tradeable Contingent Value Right (CVR) for each share of Celgene, which will entitle the holder to receive a payment for the achievement of future regulatory milestones. The Boards of Directors of both companies have approved the combination.

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The transaction will create a leading focused specialty biopharma company well positioned to address the needs of patients with cancer, inflammatory and immunologic disease and cardiovascular disease through high-value innovative medicines and leading scientific capabilities. With complementary areas of focus, the combined company will operate with global reach and scale, maintaining the speed and agility that is core to each company’s strategic approach.

Based on the closing price of Bristol-Myers Squibb stock of $52.43 on January 2, 2019, the cash and stock consideration to be received by Celgene shareholders at closing is valued at $102.43 per Celgene share and one CVR (as described below). When completed, Bristol-Myers Squibb shareholders are expected to own approximately 69 percent of the company, and Celgene shareholders are expected to own approximately 31 percent.

"Together with Celgene, we are creating an innovative biopharma leader, with leading franchises and a deep and broad pipeline that will drive sustainable growth and deliver new options for patients across a range of serious diseases," said Giovanni Caforio, M.D., Chairman and Chief Executive Officer of Bristol-Myers Squibb. "As a combined entity, we will enhance our leadership positions across our portfolio, including in cancer and immunology and inflammation. We will also benefit from an expanded early- and late-stage pipeline that includes six expected near-term product launches. Together, our pipeline holds significant promise for patients, allowing us to accelerate new options through a broader range of cutting-edge technologies and discovery platforms."

Dr. Caforio continued, "We are impressed by what Celgene has accomplished for patients, and we look forward to welcoming Celgene employees to Bristol-Myers Squibb. Our new company will continue the strong patient focus that is core to both companies’ missions, creating a shared organization with a goal of discovering, developing and delivering innovative medicines for patients with serious diseases. We are confident we will drive value for shareholders and create opportunities for employees."

"For more than 30 years, Celgene’s commitment to leading innovation has allowed us to deliver life-changing treatments to patients in areas of high unmet need. Combining with Bristol-Myers Squibb, we are delivering immediate and substantial value to Celgene shareholders and providing them meaningful participation in the long-term growth opportunities created by the combined company," said Mark Alles, Chairman and Chief Executive Officer of Celgene. "Our employees should be incredibly proud of what we have accomplished together and excited for the opportunities ahead of us as we join with Bristol-Myers Squibb, where we can further advance our mission for patients. We look forward to working with the Bristol-Myers Squibb team as we bring our two companies together."

Compelling Strategic Benefits

Leading franchises with complementary product portfolios provide enhanced scale and balance. The combination creates:
Leading oncology franchises in both solid tumors and hematologic malignancies led by Opdivo and Yervoy as well as Revlimid and Pomalyst;
A top five immunology and inflammation franchise led by Orencia and Otezla; and
The #1 cardiovascular franchise led by Eliquis.

The combined company will have nine products with more than $1 billion in annual sales and significant potential for growth in the core disease areas of oncology, immunology and inflammation and cardiovascular disease.

Near-term launch opportunities representing greater than $15 billion in revenue potential. The combined company will have six expected near-term product launches:
Two in immunology and inflammation, TYK2 and ozanimod; and
Four in hematology, luspatercept, liso-cel (JCAR017), bb2121 and fedratinib.

These launches leverage the combined commercial capabilities of the two companies and will broaden and enhance Bristol-Myers Squibb’s market position with innovative and differentiated products. This is in addition to a significant number of lifecycle management registrational readouts expected in Immuno-Oncology (IO).

Early-stage pipeline builds sustainable platform for growth. The combined company will have a deep and diverse early-stage pipeline across solid tumors and hematologic malignancies, immunology and inflammation, cardiovascular disease and fibrotic disease leveraging combined strengths in innovation. The early-stage pipeline includes 50 high potential assets, many with important data readouts in the near-term. With a significantly enhanced early-stage pipeline, Bristol-Myers Squibb will be well positioned for long-term growth and significant value creation.

Powerful combined discovery capabilities with world-class expertise in a broad range of modalities. Together, the Company will have expanded innovation capabilities in small molecule design, biologics/synthetic biologics, protein homeostasis, antibody engineering and cell therapy. Furthermore, strong external partnerships provide access to additional modalities.

Compelling Financial Benefits

Strong returns and significant immediate EPS accretion. The transaction’s internal rate of return is expected to be well in excess of Celgene’s and Bristol-Myers Squibb’s cost of capital. The combination is expected to be more than 40 percent accretive to Bristol-Myers Squibb’s EPS on a standalone basis in the first full year following close of the transaction.
Strong balance sheet and cash flow generation to enable significant investment in innovation. With more than $45 billion of expected free cash flow generation over the first three full years post-closing, the Company is committed to maintaining strong investment grade credit ratings while continuing its dividend policy for the benefit of Bristol-Myers Squibb and Celgene shareholders. Bristol-Myers Squibb will also have significant financial flexibility to realize the full potential of the enhanced late- and early-stage pipeline.
Meaningful cost synergies. Bristol-Myers Squibb expects to realize run-rate cost synergies of approximately $2.5 billion by 2022. Bristol-Myers Squibb is confident it will achieve efficiencies across the organization while maintaining a strong, core commitment to innovation and delivering the value of the portfolio.

Terms and Financing

Based on the closing price of Bristol-Myers Squibb stock on January 2, 2019, the cash and stock consideration to be received by Celgene shareholders is valued at $102.43 per share. The cash and stock consideration represents an approximately 51 percent premium to Celgene shareholders based on the 30-day volume weighted average closing stock price of Celgene prior to signing and an approximately 54 percent premium to Celgene shareholders based on the closing stock price of Celgene on January 2, 2019. Each share also will receive one tradeable CVR, which will entitle its holder to receive a one-time potential payment of $9.00 in cash upon FDA approval of all three of ozanimod (by December 31, 2020), liso-cel (JCAR017) (by December 31, 2020) and bb2121 (by March 31, 2021), in each case for a specified indication.

The transaction is not subject to a financing condition. The cash portion will be funded through a combination of cash on hand and debt financing. Bristol-Myers Squibb has obtained fully committed debt financing from Morgan Stanley Senior Funding, Inc. and MUFG Bank, Ltd. Following the close of the transaction, Bristol-Myers Squibb expects that substantially all of the debt of the combined company will be pari passu.

Accelerated Share Repurchase Program

Bristol-Myers Squibb expects to execute an accelerated share repurchase program of up to approximately $5 billion, subject to the closing of the transaction, market conditions and Board approval.

Corporate Governance

Following the close of the transaction, Dr. Caforio will continue to serve as Chairman of the Board and Chief Executive Officer of the company. Two members from Celgene’s Board will be added to the Board of Directors of Bristol-Myers Squibb. The combined company will continue to have a strong presence throughout New Jersey.

Approvals and Timing to Close

The transaction is subject to approval by Bristol-Myers Squibb and Celgene shareholders and the satisfaction of customary closing conditions and regulatory approvals. Bristol-Myers Squibb and Celgene expect to complete the transaction in the third quarter of 2019.

Advisors

Morgan Stanley & Co. LLC is serving as lead financial advisor to Bristol-Myers Squibb, and Evercore and Dyal Co. LLC are serving as financial advisors to Bristol-Myers Squibb. Kirkland & Ellis LLP is serving as Bristol-Myers Squibb’s legal counsel. J.P. Morgan Securities LLC is serving as lead financial advisor and Citi is acting as financial advisor to Celgene. Wachtell, Lipton, Rosen & Katz is serving as legal counsel to Celgene.

Bristol-Myers Squibb 2019 EPS Guidance

In a separate press release issued today, Bristol-Myers Squibb announced its 2019 EPS guidance for full-year 2019, which is available on the "Investor Relations" section of the Bristol-Myers Squibb website at View Source." target="_blank" title="View Source." rel="nofollow">View Source

Conference Call

Bristol-Myers Squibb and Celgene will host a conference call today, at 8:00 a.m. ET to discuss the transaction. The conference call can be accessed by dialing (800) 347-6311 (U.S. / Canada) or (786) 460-7199 (International) and giving the passcode 4935567. A replay of the call will be available from January 3, 2019 until January 17, 2019 by dialing (888) 203-1112 (U.S. / Canada) or (719) 457-0820 (International) and giving the passcode 4935567.

A live webcast of the conference call will be available on the investor relations section of each company’s website at Bristol-Myers Squibb View Source and Celgene View Source." target="_blank" title="View Source." rel="nofollow">View Source

Presentation and Infographic

Associated presentation materials and an infographic regarding the transaction will be available on the investor relations section of each company’s website at Bristol-Myers Squibb View Source and Celgene View Source as well as a joint transaction website at www.bestofbiopharma.com.

On January 3, 2019 Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage drug discovery and development company applying synthetic biology to beneficial microbes to develop novel living medicines, reported an overview of recent progress and outlined key objectives and anticipated milestones for 2019 (Press release, Synlogic, JAN 3, 2019, View Source [SID1234532396]).

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"2018 was a significant year for Synlogic as we advanced our two lead clinical programs and platform. We have demonstrated proof of mechanism in both programs in healthy volunteers and are currently evaluating safety and activity in patients with disease. In addition, we broadened our pipeline with the addition of our first immuno-oncology development candidate, SYNB1891," said Aoife Brennan, M.B. Ch.B., Synlogic’s president and chief executive officer. "In 2019 we look forward to presenting data from our two ongoing clinical programs that will inform the development of our Synthetic Biotic platform. With the recent expansion of our internal GMP-manufacturing capabilities, we are well positioned to maintain the momentum of the past year and continue to advance programs through clinical development as expeditiously as possible."

2019 Goals and Catalysts

Pipeline

SYNB1020: An orally delivered, first-in-class, Synthetic Biotic medicine designed for treatment of elevated blood ammonia levels (hyperammonemia) in chronic liver disease or genetic urea cycle disorders (UCDs).
In mid-2019, Synlogic expects to present top-line data from its randomized, double-blind, placebo -controlled Phase 1b/2a clinical trial evaluating SYNB1020 in patients with cirrhosis and elevated ammonia. The main endpoints of the study are safety and tolerability, as well as evidence of ammonia lowering in patients.
SYNB1618: An orally delivered, Synthetic Biotic medicine designed for the treatment of phenylketonuria (PKU).
In mid-2019, Synlogic expects to present top-line data from its randomized, double-blind, placebo-controlled Phase 1/2a clinical trial evaluating SYNB1618 in patients with PKU. The study is designed to evaluate safety and tolerability in this population as well as pharmacokinetics and pharmacodynamics as determined by the production of biomarkers specifically associated with SYNB1618 activity.
SYNB1891: Synlogic’s first immuno-oncology (IO) development candidate, a STING agonist-producing Synthetic Biotic medicine, designed to act as a dual innate immune activator, for the treatment of non-immunologically responsive solid tumors.
In the second half of 2019 the Company expects to file an IND application to enable advancement of SYNB1891 into a Phase 1 clinical study.
Pre-clinical data and early pipeline programs:
The company expects to publish and present data at major scientific and medical meetings throughout the year demonstrating the breadth and potential of its Synthetic Biotic platform.
Synlogic and AbbVie will continue to advance their ongoing collaboration to develop a Synthetic Biotic medicine for the treatment of inflammatory bowel disease (IBD).
Corporate

Synlogic ended the third quarter of 2018 with $133 million in cash and cash equivalents and expects that this will fund Company operations through 2020 under its current plan.
The Company will continue to explore additional strategic opportunities to expand the reach of its Synthetic Biotic platform.
2018 Accomplishments and Highlights:

Pipeline

SYNB1020:
Dosed first patient in Phase 1b/2a trial of SYNB1020 for the treatment of hyperammonemia in patients with cirrhosis and elevated ammonia. The clinical trial is a single and multiple dose-escalation, randomized, double-blind, placebo-controlled study of orally administered SYNB1020 in patients with cirrhosis and elevated blood ammonia, designed to evaluate safety, tolerability, kinetics, and pharmacodynamics as well as the ability of SYNB1020 to lower blood ammonia. Synlogic enrolled and treated an initial open-label sentinel cohort of six subjects with mild disease to ensure that SYNB1020 was safe in patients with liver disease who often have compromised barrier function and might be susceptible to infection. This part of the study is complete and Synlogic is enrolling patients with more advanced disease with elevated blood ammonia at baseline.
Presented data supporting continued development of SYNB1020 for the treatment of liver disease at the annual meeting of the American Association for the Study of Liver Diseases (AASLD). Synlogic presented data from a cross-sectional study designed to establish ammonia measurement parameters and ammonia levels in healthy volunteers at clinical sites that are participating in Synlogic’s ongoing Phase 1b/2a clinical trial of SYNB1020 in patients with cirrhosis and elevated ammonia. Preclinical data from a rat model were also presented by Synlogic’s collaborators that demonstrated dose-dependent lowering of blood ammonia by Synthetic Biotic strains designed to consume ammonia, confirming earlier preclinical observations in mouse models of liver disease.
SYNB1618:
Announced positive interim data from the healthy volunteer arm of its ongoing Phase 1/2a clinical trial evaluating SYNB1618 for the treatment of PKU. The data demonstrated a statistically significant, dose-dependent effect on treatment-associated biomarkers, indicating proof-of-mechanism, and also established a go-forward dose for the treatment arm in patients with PKU.
Published preclinical data in Nature Biotechnology identifying key biomarkers of SYNB1618 activity in healthy animal and disease models. The data demonstrated that oral administration of SYNB1618 significantly reduced blood phenylalanine (Phe) levels, the key metabolite associated with PKU, in mouse models of PKU and resulted in dose-dependent pharmacodynamics in healthy non-human primates (NHPs).
Fast Track designation granted to SYNB1618 for PKU by the U.S. Food and Drug Administration (FDA).
SYNB1891:
Presented preclinical data highlighting the potential of Synthetic Biotic medicines in IO and announced first IO clinical candidate at the annual meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper). A webcast of the presentation is available on the Synlogic website. Data presented at the meeting demonstrate the platform’s potential for the treatment of cancer and inflammation and specifically highlight the unique advantages of Synlogic’s approach to stimulate the innate immune system.
Corporate

Successfully completed two public offerings of common stock in January and April, resulting in approximately $83 million in total net proceeds.
The Company announced executive leadership changes including the appointment of Aoife Brennan, M.B., B.Ch., as president and chief executive officer, and the appointment of Antoine Awad as Head of Technical Operations.
Synlogic expanded its manufacturing capabilities to enable production of clinical trial material for its oral and immuno-oncology programs via entry into an agreement to lease GMP clean-room space from the Azzur Group, LLC. The agreement provides the Company with infrastructure that enables advancement of clinical programs through early and mid-phase studies and supports the production of both solid and oral formulations.
Advanced collaboration with AbbVie to develop Synthetic Biotic-based treatments for IBD resulting in payment to Synlogic of a $2.0 million milestone.
Synlogic was added to both the Russell 3000Index and the NASDAQ Biotechnology Index (Nasdaq: NBI).

On January 3, 2019 Adaptive Biotechnologies reported that Chad Robins, chief executive officer and co-founder, will present at the 37th Annual J.P. Morgan Healthcare Conference on January 8, 2019 at 11:30 am PT (2:30 pm ET) in San Francisco (Press release, Adaptive Biotechnologies, JAN 3, 2019, View Source [SID1234532413]). Robins will also participate in a panel presentation, Reinventing the Disease Continuum Through Precision Health, on January 7, 2019 at 5:00 pm PT (8:00 pm ET) to discuss the impact of immune profiling on the future of immune-driven medicine.

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A live webcast of the presentation and Q&A session can be accessed from the Newsroom under Events on the Adaptive website at adaptivebiotech.com. Following the live presentation, a replay of the webcast will be available on the Company’s website for 90 days.