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 Replimune Group Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported status updates highlighting progress with the Company’s key programs (Press release, Replimune, JAN 3, 2019, View Source [SID1234532398]).

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"Replimune had a very productive 2018 with our successful initial public offering in July providing us with the funds to continue to advance our new generation of potentially best in class oncolytic immuno-gene therapies into and through clinical trials," said Robert Coffin, Ph.D., co-founder, President and CEO of Replimune. "We have made tremendous progress with all aspects of our business and are pleased that all programs continue to progress on track. We now look forward to 2019 when we expect to initiate enrollment of the Phase 2 portion of the Phase 1/2 clinical trial of RP1 in combination with nivolumab in four solid tumor types, initiate enrollment of our potentially pivotal randomized controlled Phase 2 clinical trial of RP1 in combination with cemiplimab in cutaneous squamous cell carcinoma (CSCC), and initiate clinical development of our RP2 product candidate expressing anti-CTLA-4."

Program Updates

RP1:RP1 is Replimune’s first product candidate to enter the clinic and is based on a proprietary new strain of herpes simplex virus armed with a gene encoding a potent fusogenic protein (GALV-GP-R-), intended to enhance tumor killing potency, immunogenic cell death and the activation of systemic anti-tumor immune responses, and with a gene encoding the cytokine GM-CSF. Replimune is currently testing RP1 in a two-part Phase 1/2 clinical trial in collaboration with Bristol Myers Squibb. In part one of the Phase 1/2 clinical trial, Replimune is assessing the safety and tolerability of RP1 administered alone in patients with advanced solid tumors followed by dosing in combination with nivolumab anti-PD1 therapy. In part two of the Phase 1/2 clinical trial Replimune intends to study the safety and efficacy of RP1 in combination with nivolumab in four cohorts of patients with different solid tumor types. Replimune also intends to initiate a registration-directed randomized controlled Phase 2 clinical trial of approximately 240 patients with CSCC comparing treatment with cemiplimab alone to treatment in combination with RP1, under the Company’s collaboration with Regeneron. Cemiplimab is Regeneron’s anti-PD1 drug which was approved by the U.S. Food and Drug Administration (FDA) for the treatment of locally recurrent and metastatic CSCC in 2018.
Recent RP1-specific program progress is summarized below:

Completed enrollment of RP1 alone in the Phase 1 portion of the Phase 1/2 study.
Opened enrollment of the second part of the Phase 1 portion of the study, in which RP1 is being combined with nivolumab, in the United States (U.S.) and United Kingdom (UK).
Data from the full Phase 1 part of the Phase 1/2 study (RP1 alone and RP1 combined with nivolumab) is expected to be presented at a medical conference in the second half of 2019.
On track to initiate the Phase 2 portion of the study in the first half of 2019 in four cohorts of approximately 30 patients each with melanoma, bladder cancer, microsatellite instability high cancers, and non-melanoma skin cancers.
On track to initiate the registration-directed randomized, controlled Phase 2 clinical trial of RP1 in combination with cemiplimab in CSCC in the first half of 2019.
Pipeline product candidates (RP2 & RP3)

Replimune’s pipeline product candidates are further armed versions of RP1 which focus on the delivery of immune activating genes to tumors which target clinically validated pathways that act as the immune response is initiated. In particular, these are pathways where Replimune believes systemic engagement may be sub-optimal.

RP2 is a version of RP1 that, in addition to expressing GALV-GP-R and GM-CSF, also expresses a genetically encoded anti-CTLA-4 antibody intended to block the inhibition of the initiation of immune response caused by CTLA-4. RP2 is intended to be used primarily in combination with anti-PD-1 or anti-PD-L1 therapy.
The Company remains on track to initiate the clinical development of RP2 in a Phase 1 clinical trial of RP2 alone and in combination with anti-PD1 therapy in the first half of 2019.
RP3 is a further armed oncolytic immuno-gene therapy which expresses two immune co-stimulatory activating ligands. Following the assessment of a number of co-stimulatory pathways, which like anti-CTLA-4 are expected to be primarily active at the site and time of anti-tumor immune response initiation, the selected RP3 product candidate to be moved forward to clinical development has now been finalized and will express CD40L and 4-1BBL, together with anti-CTLA-4 and GALV-GP-R-. CD40L activates CD40, resulting in the broad activation of both innate and adaptive immunity, and 4-1BBL activates 4-1BB (CD137) to promote the expansion of cellular and memory immune responses.
The Company remains on track to initiate the clinical development of RP3 in a Phase 1 clinical trial of RP3 alone and in combination with anti-PD1 therapy in the first half of 2020.
Cash Position: Based on its current operating plan, Replimune expects that its current cash, cash equivalents and short-term investments will enable it to fund its operating expenses and capital expenditure requirements into the second half of 2021.

JPMorgan Conference Presentation and Webcast

As previously announced, Replimune will be presenting at the 37th Annual JPMorgan Healthcare Conference on January 9 at 8:00am PT.

A simultaneous webcast will be available in the Investors section of Replimune’s website at www.replimune.com. A replay will be available for 30 days following the conference.

On January 3, 2019 Peloton Therapeutics, Inc., a clinical-stage pharmaceutical company advancing first-in-class oral medicines for cancer and other serious conditions, reported that it will be presenting at the 37th Annual J.P. Morgan Healthcare Conference (Press release, Peloton Therapeutics, JAN 3, 2019, View Source [SID1234532415]). John A. Josey, Ph.D., Chief Executive Officer, will present at 3:30 p.m. PDT on Tuesday, January 8, 2019 at the Westin St. Francis in San Francisco, California.

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On January 3, 2019 GT Medical Technologies, Inc., a company dedicated to improving the lives of patients with brain tumors, reported positive results from a clinical trial supporting the efficacy and safety profile of a new treatment, GammaTile Therapy, for patients with recurrent, previously treated brain tumors known as meningiomas (Press release, GT Medical Technologies, JAN 3, 2019, View Source [SID1234532431]). Meningiomas are the most common type of primary brain tumor. They are usually non-cancerous but can have a significant impact on patients’ lives, causing headaches, seizures, cognitive decline, and other life-threatening symptoms. Results were published in the Journal of Neurosurgery (JNS), the official journal of the American Association of Neurological Surgeons.

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In this single-arm, prospective study, 20 recurrent meningiomas were treated with adjuvant GammaTile Therapy immediately following surgery. The study found that in patients undergoing surgery followed by GammaTile, there was a statistically significant improvement in time-to-local disease progression (TTP) compared to the same patients’ prior rounds of treatment. At the time of the analysis, median TTP for tumors treated with GammaTile had not been reached because less than half of the patients had experienced tumor recurrence. Median TTP for tumors treated with GammaTile is projected to be at least 29 months (95 percent confidence interval) – nearly a year longer than results for prior rounds of treatment (18.3 months TTP, HR=0.17, p=0.02). At 18 months post-treatment, tumors had not recurred in 89 percent of patients treated with GammaTile, compared to 50 percent for prior rounds of treatment without GammaTile. Treatment with GammaTile was well tolerated.

GammaTile is an FDA-cleared, surgically targeted radiation therapy (STaRT) for patients with recurrent intracranial neoplasms (brain tumors) including primary (benign or malignant) and metastatic tumors. Placed directly at the site of the tumor cavity during the last few minutes of excision surgery, GammaTile Therapy is a new approach that immediately begins targeting residual tumor cells, before they can replicate. Designed to help protect healthy brain tissue and facilitate rapid, accurate placement during the procedure, the therapy features a bioresorbable, conformable, 3D-collagen tile and uniform radiation source.

GammaTile Therapy offers advantages over the most common treatment for patients undergoing surgery for recurrent brain tumors: a course of External Beam Radiation Therapy (EBRT), which requires daily treatments for up to six weeks. Some patients may not be candidates for EBRT. Once the disease has returned, many people with recurrent brain tumors have already received levels of radiation therapy that make the risk of additional external beam radiation outweigh the potential benefits. Additionally, those patients who are potentially candidates for EBRT typically have to wait two weeks or more for surgical wound healing before beginning treatment, giving any residual tumor cells a chance to replicate.

"In patients with recurrent meningiomas, treatment options are extremely limited. Repeat surgery may not be a good option without an effective adjuvant therapy. With GammaTile, we can now offer patients who otherwise would not have been able to receive treatment or who would likely be facing early recurrence another option – one proven to delay local meningioma tumor progression out to two years," said Peter Nakaji, M.D., co-author of the study and GT MedTech’s co-founder and director of the Neurosurgery Residency Program at Barrow Neurological Institute. "Because it is delivered directly to the tumor bed, GammaTile offers the benefits of radiation while minimizing damage to surrounding healthy tissue from EBRT, and reduces the need for patients to return for daily outpatient radiation treatments."

Because the therapy is implanted at surgery, patients treated with GammaTile Therapy require no additional trips to the hospital or clinic for radiation therapy. The therapy is targeted, so patients receive radiation only where it is needed and may receive a lower overall level of exposure of normal tissue to radiation. GammaTile Therapy can emit two-and-a-half times the radiation dose compared to the dose that can be achieved from EBRT. This dose is delivered to a localized area and is highly lethal to residual tumor cells.

Approximately 400,000 Americans are newly diagnosed with some type of brain tumor each year.1 Despite the efforts of the most skilled brain tumor specialists throughout the world, outcomes for patients with brain tumors have improved little over the past 30 years. Recurrence of brain tumors is common, and about half of all patients treated for brain tumors have their disease recur within a year.

"As a treating physician, I have seen first-hand the need for better options for our patients. We created GammaTile as a therapy designed to be immediate, safe, predictable, and effective," said GT MedTech’s co-founder and chief technology officer David G. Brachman, M.D., lead author of the study, who previously served as chairman and medical director of Radiation Oncology at St. Joseph’s Hospital and Barrow Neurological Institute in Phoenix, Ariz. "These first published clinical data on the technology demonstrate that GammaTile is an effective therapy option that significantly delays the progression of this common brain tumor type."

GammaTile Therapy received FDA 510(k) regulatory clearance for the treatment of all types of recurrent brain tumors in July 2018. The data published in JNS are the initial results of a larger basket-design study that looked at the use of GammaTile in 108 patients with several kinds of recurrent brain tumors, including gliomas and brain metastasis.

Dr. Brachman continued, "We are encouraged by these data in meningiomas and look forward to sharing data on the use of this treatment in other aggressive brain tumor types in the near future."

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.