Cydan today announced the launch of Tiburio Therapeutics Inc. (Tiburio), a private biopharmaceutical company focused on developing treatments for rare neuroendocrine tumors and rare endocrine diseases. 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.

"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.

About Tiburio

Tiburio is a clinical stage biopharmaceutical company dedicated to advancing novel treatments for rare neuroendocrine tumors and rare endocrine diseases where there is significant patient need. Our pipeline consists of two clinical stage compounds, TBR-760 for the treatment of non-functioning pituitary adenoma (NFPA) and TBR-065 for additional rare endocrine diseases. The company was launched out of orphan drug accelerator Cydan with financing from leading life sciences investors, New Enterprise Associates, Longitude Capital, Lundbeckfonden Ventures, and Alexandria Venture Investments. Tiburio is located in Technology Square in Cambridge, Mass. For more information, please visit tiburio.com.

About Cydan

Cydan is an orphan drug accelerator dedicated to delivering therapies that will significantly improve the lives of people living with rare genetic diseases. The company evaluates new experimental therapies and advances those with the highest potential to be disease modifying treatments. Cydan's goal is to launch new companies focused on developing promising therapies for rare genetic diseases with high unmet medical need. Cydan was founded in 2013 by a management team with extensive drug discovery, clinical development, and business development experience and is currently financed by leading life sciences investors including New Enterprise Associates (NEA), Pfizer Venture Investments, Lundbeckfonden Ventures, Longitude Capital, and Alexandria Venture Investments. Cydan is based in Technology Square in Cambridge, Mass.