On January 3, 2019 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, reported that it has exclusively licensed worldwide rights to a mesothelin-targeted chimeric antigen receptor T-cell (CAR T) immunotherapy for solid tumors from Memorial Sloan Kettering Cancer Center (MSK) (Press release, Atara Biotherapeutics, JAN 3, 2019, View Source [SID1234532388]). The most advanced program in Atara’s CAR T collaboration with MSK will focus on development of a next-generation, mesothelin-targeted CAR T using novel 1XX CAR signaling domain and PD-1 dominant negative receptor (DNR) checkpoint inhibition technologies for patients with mesothelin-associated solid tumors.

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"We are excited by the promising initial clinical results of the mesothelin-directed CAR T approach and believe mesothelin, an antigen well recognized as associated with aggressive solid tumors, is a promising target for developing a next-generation CAR T incorporating our PD-1 DNR technology," said Prasad S. Adusumilli, M.D., FACS, Deputy Chief of Thoracic Service, Director of Mesothelioma Program and Head of Solid Tumors Cell Therapy at the Cellular Therapeutics Center at MSK.

PD-1 DNRs are modified PD-1 receptors that lack inhibitory function and are designed to enhance CAR T efficacy in solid tumors without the need for development of a CAR T in combination with a PD-1 checkpoint inhibitor. Animal models have demonstrated that CAR T immunotherapies with a PD-1 DNR result in enhanced activity against solid tumors1.

"We look forward to collaborating with Atara to develop a next-generation mesothelin-targeted CAR T immunotherapy," said Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering, and Head, Gene Expression and Gene Transfer Laboratory at MSK. "Our novel mesothelin 1XX CAR is designed to extend functional CAR T cell persistence by sustaining T cell effector functions without precipitating exhaustion, provides a complementary technology to tackle challenging tumor microenvironments."

Mesothelin is a solid tumor-associated antigen that is expressed at high levels on the surface of cells in aggressive solid tumors including mesothelioma, triple-negative breast cancer, esophageal cancer, pancreatic cancer and non-small cell lung cancer. Initial results from an ongoing MSK investigator-sponsored Phase 1 study (NCT02414269) of a mesothelin-targeted CAR T immunotherapy for patients with malignant pleural cancers were presented at the 2018 American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting and support activity and safety in patients with advanced mesothelioma2,3. This ongoing Phase 1 dose-escalation study continues to accrue patients and enhanced response rates were observed when patients were subsequently treated with pembrolizumab, a PD‑1 checkpoint inhibitor. MSK is also investigating mesothelin-targeted CAR T cells for patients with advanced breast and lung cancer (NCT02792114). Additional results from these ongoing studies are expected to be presented at upcoming scientific congresses.

"Our CAR T strategy includes collaboration with investigators on cutting-edge technologies to advance programs with near-term clinical potential," said Dietmar Berger, M.D., Ph.D., Global Head of Research and Development of Atara Biotherapeutics. "Based on the encouraging MSK Phase 1 clinical experience, we are progressing a next‑generation, mesothelin‑targeted CAR T that leverages MSK technologies designed to further enhance responses in patients with mesothelioma and other advanced solid tumors. We are also developing three additional next-generation and off-the-shelf, allogeneic CAR T immunotherapies in oncology for patients with acute myelogenous leukemia (AML) and B-cell malignancies, leveraging Atara’s world-class T cell manufacturing capabilities and research expertise."

In the MSK investigator-sponsored Phase 1 study presented at the 2018 ASGCT (Free ASGCT Whitepaper) Annual Meeting, patients with malignant pleural cancers were administered a mesothelin-targeted autologous CAR T immunotherapy regionally into the chest cavity following cyclophosphamide pre-conditioning (NCT02414269). Of the six patients treated with CAR T cells following preconditioning cyclophosphamide who subsequently received checkpoint inhibitors, one showed a complete metabolic response (CMR), two had a partial response (PR) and one exhibited stable disease (SD). Mesothelin-targeted CAR T immunotherapy and PD-1 checkpoint inhibition was well-tolerated with no adverse events greater than Grade 2 and no on-target, off-tumor toxicity.

Financial terms of the agreement were not disclosed.

References
1Cherkassky L, et al. Human CAR T cells with cell-intrinsic PD-1 checkpoint blockade resist tumor-mediated inhibition. J Clin Invest. 2016 Aug 1;126(8):3130-44. doi: 10.1172/JCI83092. Epub 2016 Jul 25.
2Adusumilli PS, et al. A phase I clinical trial of malignant pleural disease treated with regionally delivered autologous mesothelin-targeted CART cells: safety and efficacy – a preliminary report. Abstract 342; 2018 ASGCT (Free ASGCT Whitepaper) Annual Meeting; Chicago, IL; May 16-19, 2018.
3Adusumilli PS, et al. Regional delivery of mesothelin-targeted CAR T cell therapy generates potent and long-lasting CD4-dependent tumor immunity. Sci Transl Med. 2014 Nov 5;6(261):261ra151. doi: 10.1126/scitranslmed.3010162.

On January 3, 2019 Allergan plc (NYSE: AGN) reported it intends to release fourth quarter and full year 2018 financial results on Tuesday, January 29, 2019, prior to the open of U.S. Financial Markets (Press release, Allergan, JAN 3, 2019, View Source(3) [SID1234532406]).

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Allergan will host a conference call and webcast at 8:30 a.m. Eastern Time on Tuesday, January 29, 2019 to discuss its financial results. The dial-in number to access the call is U.S./Canada (877) 251-7980, International (706) 643-1573, and the conference ID is 8599848.

A replay of the conference call will also be available beginning approximately two hours after the call’s conclusion and will remain available through 11:30 p.m. Eastern Time on February 28, 2019. The replay may be accessed by dialing (855) 859-2056 or (404) 537-3406 and entering the conference ID 8599848.

To access the webcast, please visit Allergan’s Investor Relations website at View Source;. A replay of the webcast will also be available on Allergan’s Investor Relations website.

On January 3, 2019 GeneCentric Therapeutics, Inc. reported the appointment of Michael V. Milburn, PhD, as Chief Executive Officer and President, and he will join GeneCentric’s Board of Directors (Press release, GeneCentric Therapeutics, JAN 3, 2019, View Source [SID1234532422]). Dr. Myla Lai-Goldman, founder, current CEO and President, will assume the newly-created role of Executive Chairperson of the Board of Directors, succeeding Clay Thorp, Founder and General Partner at Hatteras Venture Partners, as Board Chair.

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"We founded GeneCentric, with UNC researchers Chuck Perou and Neil Hayes, with a vision to develop cancer tests to match a drug to an individual patient in order to maximize the efficacy of cancer treatments and reduce adverse events," said Dr. Lai-Goldman. "We have made great progress in establishing a subtyping platform built around an expanding array of pan-cancer tests, applying the platform to lung, head and neck, bladder, and pancreatic cancer, and entering initial partnerships with pharmaceutical companies to enable precision drug development in oncology. Michael is well positioned to build on the scientific base we have established and drive our commercial efforts with pharmaceutical companies."

In addition to GeneCentric, Dr. Lai Goldman serves on the Board of Directors at West Pharmaceutical Services and Qvella Corporation as well as a Venture Partner with Hatteras Venture Partners.

"GeneCentric is at the forefront of developing RNA-based informatics and data science which will be key to developing the next generation cancer immunotherapies and chemotherapies," said Milburn. "Myla and the scientific founders and team have been instrumental in building a technology that is highly valued by our pharmaceutical partners to get the right cancer treatment to the right patient. This is a very exciting time for GeneCentric as we begin to deploy our cancer tests alongside cancer drugs."

Dr. Milburn joined GeneCentric in September of 2018 as Chief Scientific Officer and leads the development of its RNA-based technology platform for developing new cancer tests focused on pharmaceutical drug development. Previously, Dr. Milburn was CSO at Metabolon and led the R&D efforts that established the company as a premier metabolomics technology. Prior to Metabolon, he served as Senior Vice President of Research and Corporate Development at Sirtris Pharmaceuticals and Senior Vice President of Research at Plexxikon. He also held senior research positions at Structural Genomix and GlaxoWellcome.

Dr. Milburn received his PhD in Biophysical Chemistry from the University of California at Berkeley and was a post-doctoral research fellow at Harvard Medical School. Dr. Milburn has published over 100 peer-reviewed scientific articles and is an Adjunct Professor in the Department of Molecular and Structural Biochemistry at NCSU.

"Michael brings a broad range of expertise that spans drug development, biomarker discovery and diagnostic development and commercialization, along with a strong track record commercial execution that built significant value," said Clay Thorp, Founder and General Partner at Hatteras Venture Partners. "We look forward to his new leadership role at GeneCentric as they expand their cancer technology and expertise with pharmaceutical partners."

On January 3, 2019 Cydan reported the launch of Tiburio Therapeutics Inc. (Tiburio), a private biopharmaceutical company focused on developing treatments for rare neuroendocrine tumors and rare endocrine diseases (Press release, Cydan Development, JAN 3, 2019, View Source [SID1234553930]). Tiburio will advance two compounds licensed from Ipsen for the treatment of non-functioning pituitary adenoma (NFPA) (TBR-760) and for additional rare endocrine diseases (TBR-065). As part of the launch, Tiburio raised a $31M Series A financing that will fund the company’s lead compound, TBR-760, through human proof-of-concept for the treatment of NFPA and further clinical assessment of TBR-065 as a treatment for rare endocrine diseases. In conjunction with the Tiburio financing, Abraham N. Ceesay has been appointed chief executive officer of the company. Tiburio is the third orphan drug company launched by Cydan, an orphan drug accelerator dedicated to creating therapies to improve the lives of patients living with orphan diseases.

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"Patients suffering from rare neuroendocrine tumors and endocrine diseases represent a significantly underserved patient population due to the lack of effective treatment options," said Abraham N. Ceesay, chief executive officer of Tiburio. "TBR-760 and TBR-065 represent potential breakthroughs in the treatment of debilitating neuroendocrine diseases and we, at Tiburio, are intently focused on advancing these compounds for the benefit of patients. We will immediately begin Phase 2 enabling studies with TBR-760 and anticipate enrolling patients with NFPA in a Phase 2 study in the 2nd half of 2019."

The development of NFPA, which is a non-metastatic tumor in the pituitary gland in the brain, can result in life-altering and potentially life-threatening consequences for patients. Currently there are no approved therapies to treat these tumors and patients must undergo transsphenoidal surgery and/or radiation to remove or shrink the tumor. TBR-760 is a dopamine-somatostatin chimeric molecule that inhibits NFPA cell proliferation and has the potential to shrink or halt tumor growth. In prior clinical trials, TBR-760 has been shown to have a favorable safety profile.

"The agreement with Cydan to license these two innovative compounds to Tiburio expands upon the work we’ve done to advance them through Phase 2 trials, providing a foundation for their safety profile and mechanism of action," said Alexandre LeBeaut, MD, executive vice president, research & development and chief scientific officer, Ipsen. "The Cydan team’s expertise makes them a natural choice to partner with to ensure these programs can have impact for patients in need."

"Nonfunctioning pituitary adenoma can have a significant and lasting impact on patients, often requiring multiple surgeries to control tumor size, as well as radiation therapy. These invasive neurosurgeries are associated with high-risk complications," said Shi Yin Foo, MD, PhD, MMSc, chief medical officer of Cydan and acting chief medical officer of Tiburio. "There are no approved therapies and the current treatment options for these patients are invasive, risky, and often ineffective in the longer term. We can do better for patients and believe TBR-760 represents a potentially game-changing therapy for patients."

"TBR-760’s unique mechanism of action offers an exciting potential treatment for patients living with NFPA," said Chris Adams, co-founder and chief executive officer of Cydan. "Following an extensive review of the prior TBR-760 preclinical and clinical data, the Cydan team launched our third company, Tiburio, to execute a rapid clinical and regulatory program to bring this drug to patients in need."

Tiburio was granted an exclusive world-wide license to both TBR-760 and TBR-065. Ipsen will receive a minority ownership in the company as well as development and commercial milestone payments and royalties on sales. Tiburio is responsible for all future development and commercialization costs for both compounds. Additional financial terms were not disclosed.

Investment in the $31M Series A came from Cydan’s syndicate of leading life sciences investors, New Enterprise Associates, Longitude Capital, Lundbeckfonden Ventures, and Alexandria Venture Investments.

About NFPA

The pituitary gland, a master regulator of endocrine function, is located below the base of the brain. A nonfunctioning pituitary adenoma (NFPA) is a non-metastatic pituitary tumor that arise from gonadotroph cells in the pituitary. Patients often present with symptoms related to the size of the NFPA tumor, including headaches, vision problems, changes in milk production, weight change, menstrual changes, fatigue, sleep disruption, and depression or cognitive/behavioral changes.

Current treatment is limited to transsphenoidal surgery (TSS), which is performed through the nose and sinus passage, and/or radiation to halt the growth of these tumors. However, these two treatment options come with serious and high-risk consequences. In addition to these interventions designed to remove or reduce the size of the tumors, patients often require a complex set of medications to manage the endocrine disruption and other symptoms caused by the tumors and side effects of TSS and radiation.

In the U.S. there are approximately 5,000 new cases of NFPA that require TSS each year and 40-50% of these tumors will regrow within the first 5 years after surgery. Additionally, there are approximately 50,000 patients living with NFPA who may benefit from therapy to control tumor growth.

About Abraham Ceesay, CEO Tiburio

Mr. Ceesay has over 17 years of experience in leading biopharmaceutical companies and commercializing innovative therapeutic products. Prior to joining Tiburio, he served as chief operating officer at scPharmaceuticals where he developed and led all operational and commercial aspects of the company. Mr. Ceesay was integral in raising more than $140 million in private and public capital as well as closing the company’s initial public offering. Prior to joining scPharmaceuticals, he served as vice president, Sales, Marketing, and Commercial Operations at Keryx Biopharmaceuticals and spent four years at Ironwood Pharmaceuticals as Vice President of Marketing, and held responsibility for the management of the U.S. P&L, leadership of the Linzess brand team and co-promotion collaboration with Forest Laboratories/Allergan. Previously he was at Genzyme/Sanofi, initially as a field sales specialist and ultimately as the director, Renal Global Marketing, in which capacity he led the global launch of Renvela and held global marketing responsibility for the company’s renal franchise (Renagel, Renvela, Hectorol). Mr. Ceesay serves on the Board of Advisors for Life Science Cares and the Board of Directors for Food for Free. He holds a bachelor’s degree from Ithaca College, and a Master of Business Administration from Suffolk University’s Sawyer School of Management.

On January 3, 2019 Ophthotech Corporation (NASDAQ: OPHT) reported that Glenn P. Sblendorio, Chief Executive Officer and President, will present an overview of the Company at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Thursday, January 10, 2019 at 10:00 a.m. Pacific Time (1:00 p.m. Eastern Time) (Press release, Ophthotech, JAN 3, 2019, View Source [SID1234532407]).

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Investors and the general public are invited to listen to a live webcast of the presentation at www.ophthotech.com. Please connect to Ophthotech’s website several minutes prior to the start of the presentation to ensure adequate time for any software download that may be necessary. A replay will be available for 14 days following the presentation.