On January 3, 2019 Veracyte, Inc. ("Veracyte") (Nasdaq: VCYT) reported that it has entered into a long-term strategic collaboration with Johnson & Johnson Innovation LLC* and the Lung Cancer Initiative at Johnson & Johnson to advance the development and commercialization of novel diagnostic tests to detect lung cancer at its earliest stages, when the disease is most treatable (Press release, Veracyte, JAN 3, 2019, View Source [SID1234532382]). The collaboration will build upon foundational "field of injury" science — where genomic changes associated with lung cancer can be identified with a simple brushing of a person’s airway — to develop new interventions that can save lives.

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Under terms of the agreement, Veracyte and the Lung Cancer Initiative at Johnson & Johnson, whose mission is to prevent, intercept and cure lung cancer, will combine clinical study cohorts involving more than 5,000 patients with multiple years of clinical outcome data. Veracyte will contribute bronchial and nasal samples from its clinical trials, which are part of the company’s extensive lung cancer-focused biorepository. Veracyte will deploy its RNA whole-transcriptome sequencing platform, utilizing high-dimensional data and machine learning pipelines on the combined cohort, providing the most comprehensive genomic content available which the Lung Cancer Initiative at Johnson & Johnson will have access to for therapeutic purposes.

The collaboration is expected to accelerate two key lung cancer programs for Veracyte. First, the development of the first non-invasive nasal swab test for early lung cancer detection and, secondly, the commercialization of its Percepta classifier on the company’s RNA whole-transcriptome sequencing platform, which, as a result of this collaboration, is expected to launch in the first half of 2019.

"We are thrilled to team up with Johnson & Johnson and their Lung Cancer Initiative in the fight against lung cancer," said Bonnie Anderson, chairman and chief executive officer of Veracyte. "This strategic collaboration further advances Veracyte’s pioneering position in lung cancer diagnosis and underscores the promise of our field of injury science and approach. With the acceleration of our product pipeline, we believe this collaboration expands our addressable lung cancer diagnostic market to a $30 billion to $40 billion global opportunity."

Anderson continued, "We estimate the combined monetary and non-monetary value of the collaboration to Veracyte at more than $50 million. The monetary component consists of a $5 million upfront payment and the potential to earn up to a total of $15 million in future development and reimbursement milestone payments. The non-monetary value reflects the significant value of clinical cohorts accessible to Veracyte through the collaboration. Also as result of this agreement, we expect our biopharmaceutical service revenue for 2019 to increase by an estimated $5 million."

Lung cancer is the leading cause of cancer deaths worldwide. In the United States, lung cancer causes more than 154,000 lung cancer-related deaths each year – more than the next three most prevalent cancers combined. Because lung cancer is difficult to diagnose before it has metastasized, only 16 percent of cases are detected at an early stage, when the disease is more treatable. Lung cancer’s five-year survival rate is only 18 percent, much lower than that of other common cancers. Approximately 80 percent of lung cancer deaths are caused by smoking.

*Johnson & Johnson Services, Inc. is the legal entity party to the agreement.

Conference Call and Webcast Information

Veracyte will host a conference call and simultaneous webcast today at 8:30 a.m. ET to discuss its collaboration with Johnson & Johnson Innovation and the Lung Cancer Initiative at Johnson & Johnson. The call and webcast may be accessed as follows:

Webcast:
View Source

Dial-in number (U.S.): (855) 541-0980
International Number (Canada only): (970) 315-0440
Conference ID: 5295859
The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source.

On January 3, 2019 Tarveda Therapeutics, Inc., a clinical stage biopharmaceutical company discovering and developing a new class of potent and selective miniature drug conjugates (Pentarins) for the treatment of patients with a wide range of solid tumors reported that Drew Fromkin, President and Chief Executive Officer, reported that it will present at the 11th Annual Biotech Showcase, occurring January 7-9, 2019 at the Hilton Union Square in San Francisco (Press release, Tarveda Therapeutics, JAN 3, 2019, View Source [SID1234532416]).

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The presentation will take place at 2:30pm Pacific Time on Monday, January 7 in Franciscan – D.

In the presentation, Mr. Fromkin will provide an overview of the Company’s two clinical programs including PEN-221, which is currently in clinical evaluation for the treatment of patients with somatostatin receptor 2 (SSTR2) positive neuroendocrine tumors and PEN-866, the first miniature drug conjugate from Tarveda’s HSP90 binding conjugate platform, which is being developed for the treatment of patients with solid tumors including but not limited to small cell lung cancer, pancreatic cancer and sarcomas.

On January 3, 2019 1ST Biotherapeutics, Inc., a preclinical-stage biotechnology company focused on neurodegenerative diseases, immuno-oncology, and orphan diseases, and twoXAR, Inc., an artificial intelligence (AI)-driven biopharmaceutical company, reported an agreement to jointly discover and develop novel, efficacious treatments to address unmet medical needs in glioblastoma multiforme ("glioblastoma") (Press release, 1ST Bio Therapeutics, JAN 3, 2019, View Source [SID1234532432]).

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Under the agreement, twoXAR will use its proprietary AI technology to identify a set of drug candidates with the potential to slow, stop, or reverse the progression of glioblastoma. twoXAR and 1ST Biotherapeutics will select candidates from this set to test in preclinical efficacy models of glioblastoma. Following identification of one or more candidates based on those evaluated, 1ST Biotherapeutics will use its team’s expertise in drug development to optimize candidates and finalize the creation of novel, efficacious treatments. Further details of the agreement were not disclosed.

Glioblastoma is the most common and lethal primary malignant brain tumor with a median survival of approximately 15 months1. It is characterized by an aggressive tumor that infiltrates various portions of the brain and is associated with rapid onset of neurological symptoms, including nausea, vomiting, severe headaches, cognitive issues, and seizures2. Current treatment options consist of surgical resection, radiation, chemotherapy, and angiogenesis inhibitors2. However, most glioblastoma patients experience disease relapse despite these aggressive therapies. It is estimated that 25,000 people will be diagnosed with glioblastoma in 20181.

"1ST Biotherapeutics is focused on efficiently building a pipeline of first-in-class therapeutic candidates with high likelihood of clinical success," said Jamie Jae Eun Kim, CEO of 1ST Biotherapeutics. "The twoXAR team has a track record of rapidly identifying testable novel treatments that can lead to first-in-class therapeutics. This collaboration is an opportunity to combine twoXAR’s AI-driven drug discovery approach and the 1ST Biotherapeutics team’s expertise in chemistry and pharmacology to discover and develop effective molecular therapeutics for glioblastoma patients."

"We are pleased to collaborate with 1ST Biotherapeutics, because we share common goals of efficiently discovering and developing novel therapeutics for diseases with high unmet medical need, such as glioblastoma," said Andrew A. Radin, Co-Founder and CEO of twoXAR. "The 1ST Biotherapeutics team’s deep medicinal chemistry and drug development experience in CNS and oncology diseases provides a strong complement to twoXAR’s data-driven discovery approach."

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 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of novel cellular immunotherapies, reported its anticipated milestones for 2019 (Press release, Unum Therapeutics, JAN 3, 2019, View Source [SID1234532383]).

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"Unum made substantial progress in 2018 as we reported early data from our programs in non-Hodgkin lymphoma and multiple myeloma, while simultaneously expanding applications of our ACTR platform into solid tumors and introducing a second novel technology platform, BOXR, designed to improve the functionality of engineered T cells," said Chuck Wilson, CEO of Unum. "We expect 2019 also to be a year of significant momentum, with data expected from all four of our ongoing clinical programs, including readouts from the ATTCK-20-03 and ATTCK-17-01 trials, as well as an initial data readout from our first study in solid tumors. Additionally, we continue to innovate in the field of cell therapy and have recently nominated our first development candidate from our BOXR technology platform to advance toward clinical development."

Anticipated 2019 Milestones

ACTR707 + rituximab in r/r NHL

Complete the dose escalation phase of ATTCK-20-03, the ongoing, multicenter Phase 1 study testing ACTR707 in combination with rituximab to treat patients with relapsed/refractory B cell non-Hodgkin lymphoma. Advance into cohort expansion to confirm the preliminary recommended Phase 2 dose.
Report results from the dose escalation phase and preliminary data from the cohort expansion phase of the ATTCK-20-03 study.
ACTR087 + SEA-BCMA in r/r multiple myeloma

Progress dose escalation of ACTR087 and SEA-BCMA in ATTCK-17-01, a Phase 1, multi-center, open-label trial designed to test the safety, tolerability and anti-myeloma activity of the combination in patients with r/r multiple myeloma.
Report clinical data from multiple cohorts of the dose escalation phase.
ACTR707 + trastuzumab in HER2+ advanced cancers

Enroll and dose patients in Unum’s first ACTR T cell study in solid tumors, ATTCK-34-01, a multicenter, single-arm, open-label dose escalation study evaluating ACTR T cells in combination with trastuzumab in patients with HER2+ advanced cancers.
Report initial clinical data from ongoing dose escalation.
ACTR087 + rituximab in r/r NHL

Complete enrollment in ATTCK-20-2, a Phase I clinical trial evaluating safety and anti-lymphoma activity of ACTR087 in combination with rituximab in patients with relapsed or refractory B cell NHL, and report data from the trial.
BOXR Platform

Continued progress with new Bolt-On Chimeric Receptor technology platform ("BOXR") that improves T cell functionality by countering immunosuppression in solid tumor cancers. Initiate clinical development of BOXR1030, which has been nominated as the first product candidate from this platform.