Naia Rare Diseases granted orphan drug designation for NB1001

Naia Limited, an international drug development company today announced that its subsidiary, Naia Rare Diseases, has been granted by the U.S. Food and Drug Administration (FDA) orphan drug designation for NB1001, its long-acting GLP-1 receptor agonist, for the treatment of Short Bowel Syndrome (SBS).

“The receipt of orphan drug designation for NB1001 is further indication that there is a great unmet medical need for the treatment of Short Bowel Syndrome, and our drug candidate may represent a promising new approach for the treatment of this disease,” said H. Daniel Perez, M.D., co-founder, chairman and chief executive officer of Naia Limited.

Mark Bagnall, co-founder and chief financial officer of Naia Limited, added, “We are committed in advancing NB1001 through clinical trials with the initiation of a Phase 1b dose de-escalating in SBS patients by the second quarter of 2016.”

NB1001 (XTEN™-GLP-1) is a long-acting Glucagon-like peptide-1 (GLP-1) receptor agonist that combines exenatide with a proprietary extended half-life technology. NB1001 will be administered as a replacement therapy (replacing endogenous GLP-1 lost by bowel resection), in contrast to GLP-1 agonists used to treat Type 2 diabetes, which are administered at pharmacologic levels in order to lower blood sugar. Lower doses of NB1001 combined with a longer half-life will provide a differentiated, safe, effective and convenient therapeutic approach for these patients.

GLP-1 is produced by the L cells of the ileum. Upon food ingestion, low concentrations of endogenous GLP-1 act as a negative modulator of gastric and duodenal contractility, slowing the transit of food from the stomach. Slow transit results in better digestion of food and better absorption of nutrients in the small bowel.  After extensive intestinal resection, SBS patients lack endogenous GLP-1. Deficiency of GLP-1 leads to fast transit of food through a shortened bowel and diminished absorption of nutrients leading to severe nutritional deficiency and fluid imbalance.

FDA orphan drug designation is granted to investigational therapies addressing rare medical diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

NB1001 was previously being developed for Type 2 diabetes, and in a 70-patient Phase 1b study, NB1001 was shown to be safe, well-tolerated and lowered HbA1c in a dose-dependent manner.

About Naia Limited
Naia Limited builds and funds new biotech companies using de-risked clinical stage assets. Naia’s structure spreads overhead costs and resources during early development of assets thus reducing costs and time.  The company has an international base of operations to develop therapeutics better, faster and more inexpensively – in proximity to target markets. Naia’s operating structure includes a Cayman Island-based management company and several holding companies that focus on development activities of like assets. The company is developing a diverse portfolio of therapeutics that address regional and global unmet medical needs in various markets with an initial focus on rare and metabolic diseases.

Naia Limited Successfully Completes Pre-IND Meeting with US FDA for NB1001

Naia Limited Successfully Completes Pre-IND Meeting with US FDA for NB1001, Long-Acting GLP-1 (XTEN™-GLP-1) Receptor Agonist for Treatment of Short Bowel Syndrome

 –Company Plans to Initiate Phase 1b Study in Second Quarter of 2016–

 –Submits Orphan Drug Application for NB1001 in U.S. and Europe–

RICHMOND, Calif. Oct 12, 2015 – Naia Limited, an international drug development company with an initial portfolio of innovative, clinical-stage compounds for short bowel syndrome, NASH and Type 2 diabetes, today announced that its subsidiary, Naia Rare Diseases, Inc.  successfully completed a pre-IND (Investigational New Drug) meeting with the U.S. Food and Drug Administration (FDA). The FDA has agreed to Naia’s proposed plans for advancing the clinical development of NB1001, its long-acting GLP-1 (XTEN™-GLP-1) receptor agonist, including a Phase 1 dose de-escalating clinical trial in patients with Short Bowel Syndrome. Naia expects to initiate the Phase 1b study in the second quarter of 2016, and based on its current forecast clinical timelines, to be able to file a new drug application (NDA) for NB1001 by the fourth quarter of 2018.

“With the positive feedback we received from the FDA, we are assured that we are on the right path with the development of NB1001 and our rare disease pipeline,” said H. Daniel Perez, M.D., co-founder, chairman and chief executive officer of Naia Limited. “Based on our discussions with the Agency, we plan to initiate a Phase 1 dose de-escalating clinical trial with NB1001 in the second quarter of 2016. This study design will allow us to assess both drug safety, preliminary efficacy and the doses we will use in our planned phase 2/3 pivotal study.”

Naia also announced today that it has filed for orphan drug status with the FDA and the European Medicines Agency (EMA) for NB1001 for the treatment of short bowel syndrome.

“Short bowel syndrome has been designated a rare disease by both the FDA and the EMA, and we believe that developing NB1001 as an orphan drug  in the U.S. and Europe is the most expedient and cost-effective way to  bring it to market,” said Mark Pimentel, M.D., chairman of Naia’s scientific & clinical advisory board. “NB1001 is the only drug in development that I am aware of that directly addresses bowel motility. As a result it should allow many patients to eliminate the need for parenteral nutrition, thus considerably improving their quality of life.”

About NB1001

GLP-1 is produced by the L cells of the ileum. Upon food ingestion, low concentrations of endogenous GLP-1 act as a negative modulator of gastric and duodenal contractility, slowing the transit of food from the stomach. Slow transit results in better digestion of food and better absorption of nutrients in the small bowel.

NB1001 (XTEN™-GLP-1) is a long-acting GLP-1 (Glucagon-like peptide-1) receptor agonist that combines exenatide with a proprietary extended half-life technology.  NB1001 was previously being developed for Type 2 diabetes, and in a 70-patient Phase 1b study, NB1001 was shown to be safe, well-tolerated and lowered HbA1c in a dose-dependent manner.  NB 1001 will be administered as a replacement therapy (replacing endogenous GLP-1 lost by bowel resection), in contrast to GLP-1 agonists used to treat of Type 2 diabetes, which are administered at pharmacologic levels in order to lower blood sugar.

About GLP-1 Receptor Agonists.  GLP-1 receptor agonists are injectable drugs that mimic the action of human GLP-1 and have been used to treat Type 2 diabetes since 2005.  In the treatment of diabetes, pharmacologic concentrations of GLP-1 agonists reduce blood sugar by increasing insulin production in the pancreas, slowing absorption of glucose from the gut and reducing the activity of glucagon.

 About Short Bowel Syndrome
Short bowel syndrome is a malabsorption disorder caused by the surgical removal of the small intestine. In rare cases it is caused by the complete dysfunction of a large segment of bowel. Short bowel syndrome affects between 7,000 and 12,000 individuals in the United States and 5,000 to 15,000 in Europe. It has been designated a rare disease by the FDA and EMA and is eligible for orphan drug designation by both regulatory agencies.

Symptoms include abdominal pain, diarrhea, fluid depletion, weight loss and malnutrition, and fatigue. Patients with short bowel syndrome may have complications caused by malabsorption of vitamins and minerals, including anemia, hyperkeratosis (scaling of the skin), bruising, muscle spasms, poor blood clotting and bone pain.

There is no cure for short bowel syndrome. Treatments include surgical procedures and medications, including anti-diarrheal medicine, vitamin and mineral supplements, H2blocker and proton pump inhibitors to reduce stomach acid, and lactose supplements. In the United States and Europe, the injectable agent teduglutide is approved to treat short bowel syndrome.

 About Naia Limited

Naia Limited is employing a creative business model for global drug development that exploits untapped opportunities on a worldwide basis. This business model is designed to meet the needs of a complex healthcare ecosystem currently straining to contain costs while improving access to therapies. The company has an international base of operations to develop therapeutics better, faster and more inexpensively – in proximity to target markets. Naia’s operating structure includes a Cayman Island-based management company and several holding companies that focus on development activities of like assets. The company is developing a diverse portfolio of therapeutics that address regional and global unmet medical needs in various markets with an initial focus on rare and metabolic diseases. For more information, please visit www.naiapharma.com.

 

Investor Contact:
Mark Bagnall, Naia Limited
510-926-8237
mbagnall@naiapharma.com

Media Contact:
Andreas Marathovouniotis
917-957-1174
amarathis@marathisllc.com

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