Mavupharma Selects First Immuno-Oncology Clinical Candidate MAVU-104, an Oral STING Pathway Enhancer

On January 3, 2019 Mavupharma (Mavu) reported that it has selected its first immuno-oncology clinical development candidate, MAVU-104, a novel innate immune modulator (Press release, Mavupharma, JAN 3, 2019, View Source [SID1234537639]).

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MAVU-104 is a first-in-class, orally active, small molecule inhibitor of ENPP1, a phosphodiesterase that negatively regulates the STING (Stimulator of Interferon Genes) pathway. STING signaling plays a crucial role in the generation of an immune response directed at the tumor, and enhancing STING signaling has been shown to induce cures in multiple preclinical cancer models. Inhibiting ENPP1 activity with MAVU-104 allows for highly-controlled enhancement of STING signaling in all tumors and lymph nodes without any injections.

"With our approach, which focuses on blocking a key negative regulator of the STING pathway, as opposed to directly stimulating STING itself, the degree and duration of innate immune activation are tunable, which avoids both overstimulation of the pathway and high levels of cytokine release," stated Mike Gallatin, Ph.D., Mavu’s president and co-founder. "Having selected our first immuno-oncology drug candidate, we are now focused on the path to IND for MAVU-104, which we expect to file in the second half of 2019."

Mavu’s approach may have significant advantages over other developmental therapies that stimulate the STING pathway. Published studies on the direct STING agonists in clinical development show that these therapies are administered via direct injection into the tumor(s) and may induce release of pro-inflammatory cytokines into systemic circulation, which has been associated with side effects.

Previously, Mavu presented data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2018 meeting showing that ENPP1 inhibition leads to tumor shrinkage and can be combined with other immunotherapies to induce long-term cures and durable immunologic memory in mouse models of cancer.

Pacira Reports Preliminary 2018 Net EXPAREL® Sales of $331 Million

On January 3, 2019 Pacira Pharmaceuticals, Inc. (NASDAQ:PCRX) reported preliminary unaudited EXPAREL (bupivacaine liposome injectable suspension) net product sales of $94 million for the fourth quarter of 2018 and $331 million for the full-year, as compared to $79 million and $283 million reported for the fourth quarter and full-year 2017, respectively (Press release, Pacira Pharmaceuticals, JAN 3, 2019, View Source [SID1234532376]). The company previously guided to full-year EXPAREL net product sales of $325 to $330 million.

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"We are very pleased with our 2018 results, with fourth quarter EXPAREL sales exceeding our expectations and marking the highest quarterly revenues ever reported by Pacira," said Dave Stack, chairman and chief executive officer of Pacira. "With an expanding number of payers, providers and patients recognizing the value of EXPAREL as a critical component within opioid-sparing pain management strategies, we are entering 2019 with strong momentum. Our robust partnership network also remains a core driver as we continue to focus on maximizing the value of EXPAREL, including its role in transitioning certain inpatient procedures to the ambulatory setting. We are particularly excited by new reimbursement codes, which went into effect on January 1, 2019, that we believe will meaningfully expand patient access through a product-specific C-code for ambulatory surgery centers and a procedure-based D-code for the oral surgery setting.

Looking ahead, we have put in place a number of growth initiatives that include building a pipeline of innovative non-opioid pain management and regenerative health solutions. Our goal is that these, coupled with our unique experience and position in the marketplace as a leader in non-opioid postsurgical pain management, will make a key contribution in confronting the current U.S. pain and opioid crises."

The financial information included in this press release is preliminary, unaudited and subject to adjustment. It does not present all information necessary for an understanding of the company’s fourth quarter and full-year financial results for 2018. Pacira expects to report its complete financial results for 2018, along with financial guidance for 2019, during the company’s fourth quarter earnings call, which will take place in the first quarter of 2019.

Exicure to Present at Biotech Showcase 2019 on January 8th at 2:30 pm PT

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.

Jazz Pharmaceuticals and Codiak BioSciences Announce Strategic Collaboration to Research, Develop and Commercialize Engineered Exosomes to Create Therapies for Hard-to-Treat Cancers

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

Tragara Pharmaceuticals Appoints Scott Megaffin as Chief Executive Officer and Changes Name to Adastra Pharmaceuticals

On January 3, 2019 Tragara Pharmaceuticals Inc. reported the appointment of Scott Megaffin as Chief Executive Officer and a member of the Board of Directors effective immediately (Press release, Tragara Pharmaceuticals, JAN 3, 2019, View Source [SID1234539282]). Megaffin brings extensive leadership experience in clinical stage companies in oncology, specialty and critical care. He replaces Tom Estok, who will remain active with the company as an advisor and continue in his role on the Board of Directors.

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The addition of Megaffin comes at the time of a strategic pivot for the company that includes a change of name to Adastra Pharmaceuticals Inc. As part of this strategy, Adastra has expanded its operations beyond San Diego with the opening of an office in Princeton, N.J. This is consistent with the Adastra strategy to grow and attract new pharmaceutical talent in the New Jersey area. Adastra, which is derived from the Latin phrase "to the stars," is a name that reflects the boundless motivation and dedication of each team member to the development of best-in-class therapeutics for patients with cancer.

"Scott has the right experience to drive forward all of the Adastra strategic imperatives and continued program advancement of TG02," said Dennis Podlesak, Adastra Chairman and Partner of Domain Associates. "The Board would like to acknowledge and thank Tom for his vision and dedication in getting us to this point. We have advanced TG02 in clinical studies in collaboration with healthcare providers in the United States and Europe for adult patients diagnosed with glioblastoma multiforme (GBM). Furthermore, we have prepared for the start of an exploratory study in pediatric patients with diffuse intrinsic pontine glioma (DIPG). Between now and the first quarter of next year, Adastra will have a series of clinical milestones and data read-outs that we expect will enable the company to explore multiple development and business paths forward."

Prior to joining the company, Megaffin was President of Churchill Pharmaceuticals LLC, where he successfully marshalled the company through a transaction that resulted in the sale of company assets before an FDA approval, while simultaneously preparing the company for its own possible commercialization activities. Before this, he held numerous global strategic and operational positions of increasing responsibility with Onconova Therapeutics Inc., Cephalon Inc., Adolor Corp., Yamounchi and Bristol-Myers Squibb Co. He has led six global product launches and clinical development programs in a variety of therapeutic categories, including oncology, hematology, virology, critical care, anti-infectives, pain and inflammation. Megaffin earned a B.S. in Biology from Pittsburg State University.

TG02 is a highly differentiated orally delivered inhibitor of cyclin-dependent kinase 9 (CDK9) with high penetration of the blood brain barrier, overcoming a major challenge in GBM drug development. TG02 exerts inhibitory effects on RNA Polymerase II phosphorylation, leading to depletion of the key cancer cell survival protein, Myc, thus committing cancer cells to apoptosis. TG02 has demonstrated broad anti-tumor activity across cell lines and shows a positive synergistic effect when added to standard of care treatments for GBM. Currently, the National Cancer Institute (NCI) and European Organisation for Research and Treatment of Cancer (EORTC) are enrolling patients in trials of TG02 in GBM. In addition, Adastra will initiate a study of TG02 in DIPG in the second quarter of this year.

Megaffin added, "I am very happy to take on this role at Adastra during this transformational year for the company. The TG02 trials in GBM are progressing nicely, and we are entering into the start of the DIPG study in the pediatric patient population. When evaluating the opportunity to join the company, I reviewed the data in hand for TG02 and became excited by its potential for the benefit of patients. GBM and DIPG are both devastating brain cancers and represent high unmet treatment needs. TG02 presents Adastra with regulatory opportunities of rapid advancement into registrational studies in the near future. We look forward to reporting our progress in 2019 as we focus sharply on clinical programs as well as exploring possible pipeline expansion opportunities."