This branch of our Sponsor Atlas series focuses on young pharmaceutical and biotechnology companies that are in early stages or stealth mode. In the next edition of Sponsor Atlas: Discovering Biotech Startups, we’re looking at Tiburio Therapeutics, exploring the current affairs and future orientation of this emerging Cambridge - based biotech company. We will give a business overview of their current operations, summarize their outsourcing needs, map out their development goals and decision-makers, and highlight their current strategies for capturing innovation. If you haven’t already read our other blogs on new biotech startups, be sure to check them out here.
Tiburio Therapeutics is a developer of drug compounds intended to treat tumors and rare endocrine diseases. The company's compounds aid in the treatment of non-functioning pituitary adenoma using a dopamine-somatostatin chimeric molecule that has the potential to shrink or halt tumor growth, providing patients with effective treatment for rare neuroendocrine tumors and endocrine diseases.
Located near the Massachusetts Institute of Technology in Cambridge, Tiburio Therapeutics was founded in 2018. The company was launched out of an orphan drug accelerator Cydan, with financing from life science investors NEA, Lundbeckfonden Ventures, Longitude Capital, and Alexandria Venture Investments. Tiburio’s core business is to develop transformative treatments for patients with rare endocrine diseases. They have a very detailed website with information about their current research, product pipeline, and executive team.
Tiburio will advance two compounds licensed from Ipsen, TBR-760 for the treatment of non-functioning pituitary adenoma (NFPA), and TBR-065 for the treatment of additional rare endocrine diseases. As part of their initial launch, Tiburio raised a $31M Series A financing that will fund the company’s lead compound, TBR-760, through human proof-of-concept studies for NFPA, and further clinical assessment of TBR-065 as a treatment for rare endocrine diseases. Tiburio is the third orphan drug company launched by Cydan specifically for creating new therapies for 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, CEO 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.” 
Considering Tiburio's innovative platform of dopamine-somatostatin chimeric compounds that are currently under development, we think they are in a great position to fund a ton of outsourcing work. They have a few lead candidates already in clinical trials (with more on that in the next section). Their prospective pipeline will consist of therapies for treating rare endocrine diseases, and any experience with drug development for orphan drugs would be of tremendous benefit to Tiburio Therapeutics at this point.
Some of the ways service providers can get their foot in the door would be to support Tiburio in the following general areas: CMOs for Phase I/II clinical trial supplies, bioanalytical CROs, and preclinical CROs with animal model development for rare endocrine diseases.
Tiburio’s main focus is on developing TBR-760, a clinical-stage dopamine-somatostatin chimeric compound, for the treatment of non-functioning pituitary adenomas (NFPAs). Tiburio’s development-stage pipeline also includes TBR-065, another dopamine-somatostatin chimeric compound for other rare endocrine diseases.
NFPAs are benign growths in the pituitary gland that do not produce any excessive hormone into the blood and are not cancerous. NPFAs account for 15% of all pituitary adenomas, with approximately 70-90 cases per one million people.  NFPAs are known to disrupt vital activities within the brain on account of mass impact. They can easily trigger imaginative and prescient loss, hormone deficiencies, cranial nerve palsies, or other intractable complications. Current remedies prescribed include invasive neurosurgery, damaging radiation, and lifelong monitoring. Additionally, current surgical procedures do not heal these tumors and are known to cause an excessive risk of tumor recurrence.
TBR-760 has the potential to be the primary drug remedy for NFPAs. TBR-760 is being developed to shrink or stabilize non-functioning pituitary adenomas and should cut back or remove the necessity for surgical procedures and radiation, each of which is related to vital morbidity. Key knowledge from their initial research concluded that TBR-760 fully arrested tumor progress all through the eight-week remedy interval in mice with longtime tumors. During the study, it had successful results with the common tumor quantity in TBR-760-treated mice being 8.5±1.3mm3 vs. 54.61±10.6mm3 in vehicle-treated mice (p<0.05). 
In May 2020, Tiburio Therapeutics published new data from a preclinical study in the Journal of the Endocrine Society demonstrating that its lead compound, TBR-760, successfully arrested NFPA growth in a mouse model. The pre-clinical data indicated that TBR-760 completely arrested tumor growth in the pro-opiomelanocortin (POMC) gene knock-out (KO) mouse model of NFPA. Additionally, notable tumor shrinkage was observed in 20% of the mice treated with TBR-760.
“TBR-760 completely arrested tumor growth in a mouse model that spontaneously develops aggressive non-functioning pituitary adenomas,” said Heather Halem, Ph.D., VP of Research. “The pituitary tumors that form in the POMC-KO mice express similar levels of dopamine and somatostatin receptors as human NFPA, making this study fundamental in supporting the potential for tumor shrinkage in our planned phase 2 trial in adult patients with NFPA,” she continued. 
We think that Tiburio Therapeutics is on track to create some breakthroughs in the biotech industry over the next few years. Be sure to keep them on your contact list and look out for our next edition in this series coming soon.
If you would like a simple solution for keeping an eye on drug sponsor companies, like Tiburio Therapeutics, without relying on a database and generic lists of leads each week, we at Zymewire are here to help. Reach out today, and stay tuned for the next installment of the Sponsor Atlas: Discovering Biotech Startups. If you enjoy these articles, please feel free to give them a share through the social links below!