Naia Rare Diseases Announces UK Clinical Trial Authorisation for NB 1001 for Short Bowel Syndrome – Nov 15 2017

Naia Rare Diseases Announces Clinical Trial Authorisation (CTA) to Initiate Clinical Trials of NB 1001 for Short Bowel Syndrome in the United Kingdom

RICHMOND, CA –(Marketwired – November 15, 2017) – Naia Rare Diseases, a biopharmaceutical company developing drugs for Short Bowel Syndrome (SBS) and other rare gastrointestinal diseases today announced that it has received Clinical Trial Authorisation (“CTA”) from the Medicine and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom to initiate a clinical trial of its long-acting GLP-1 agonist, NB 1001, in adult patients with SBS.

“We are pleased to have been granted the CTA and look forward to starting trials in Europe,” said H. Daniel Perez, MD, President and CEO of Naia Rare Diseases and look forward to providing further details on its progress in the near future.”

We believe NB 1001 has the potential to make a significant impact on the lives of patients with SBS. NB1001 is the only drug in development that directly addresses increased bowel motility in SBS. By slowing gut transit, NB 1001 will allow for better absorption of fluids and nutrients. As a result it should allow patients to eliminate the need for parenteral nutrition, thus considerably improving their quality of life.

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.

About NB 1001
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. Licensed from Amunix Operating Inc., NB 1001 uses Amunix’s proprietary “XTEN” technology to extend the half-life of the GLP-1 peptide and allows for up to once-per-month dosing, thus considerably increasing convenience for patients and caregivers. Because of lower overall exposure and dose required (as a replacement therapy) NB 1001 will have an increased safety window compared to other GLP-1 agonists, important as SBS patients are a very fragile population. NB 1001 was previously developed to treat type 2 diabetes and in a 70 patient clinical study demonstrated efficacy and an extended half-life up to 30 days.

NB1001 has received orphan drug designation and has been granted an IND by the FDA.

About Naia Rare Diseases
Naia Rare Diseases is a development stage biopharmaceutical company developing novel drugs for rare gastrointestinal disorders. The company is pursuing three development programs including NB 1001 for Adult SBS, NB 1001 for pediatric SBS and NB 1002, a GLP-2 agonist, for an undisclosed orphan gastrointestinal indication. Naia Rare Diseases has been funded primarily by its parent company, Naia Limited, a company focused on building and funding new biotech companies using de-risked clinical stage assets. For more information, please visit www.naiapharma.com.

 

Contact: 
Mark Bagnall
Naia Pharmaceuticals
510-926-8237
mbagnall@naiapharma.com

Naia Rare Diseases Secures Investment from Cedars-Sinai Health System

Naia Rare Diseases

Naia Rare Diseases Secures Investment from Cedars-Sinai Health System

 – Lead Program Based on Clinical Research at Cedars-Sinai –

RICHMOND, Calif, April 3, 2017 –Naia Rare Diseases, a biopharmaceutical company developing drugs for Short Bowel Syndrome (SBS) and other rare gastrointestinal diseases today announced that Cedars-Sinai will participate on its ongoing Series B financing round.

“I am very happy to announce that Cedars-Sinai has made this investment,” said H. Daniel Perez, MD, Chairman, President and CEO of Naia Rare Diseases.  “The team at Cedars-Sinai has been a great partner for Naia over the past few years.  Their investment reflects confidence in Naia’s clinical program and allows us to continue progress towards our first human trial in patients with SBS .”

Naia’s lead program, NB 1001, is based on clinical research licensed from Cedars-Sinai and based on work published by Mark Pimentel, MD, FRCPC demonstrating that patients with SBS can benefit from treatment with the GLP-1 agonist, exenatide. Dr. Pimentel is chairman of Naia’s board of scientific and clinical advisors and professor of medicine and director of the gastrointestinal motility program and laboratory at Cedars-Sinai Medical Center.

Dr. Pimentel will be principal investigator of the upcoming Phase 1b clinical trial of NB 1001.  In preparation for the trial, supplies of NB 1001 have been fully manufactured and study centers in Europe and the US have been identified

 About NB 1001

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.  Licensed from Amunix Operating Inc., NB 1001 uses Amunix’s proprietary XTEN® technology to extend the half-life of the GLP-1 peptide, allowing for up to once-per-month dosing, thus considerably increasing convenience for patients and caregivers. Because of lower overall exposure and dose required (as a replacement therapy) NB 1001 will have an increased safety window compared to other GLP-1 agonists, important as SBS patients are a very fragile population. NB 1001 was previously developed to treat type 2 diabetes and in a 70 patient clinical study demonstrated efficacy and an extended half-life up to 30 days.

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.

NB1001 has received orphan drug designation by the FDA.

 About Naia Rare Diseases

Naia Rare Diseases is a development stage biopharmaceutical company developing novel drugs for rare gastrointestinal disorders.  The company is pursuing three development programs including NB 1001 for Adult SBS, NB 1001 for pediatric SBS and NB 1002, a GLP-2 agonist, an undisclosed orphan gastrointestinal indication.  Naia Rare Diseases has been funded primarily by its parent company, Naia Limited, a company focused on building and funding new biotech companies using de-risked clinical stage assets. For more information, please visit www.naiapharma.com.

 

Contact:

Mark Bagnall, Naia Pharmaceuticals

510-926-8237

mbagnall@naiapharma.com

Naia Rare Diseases Announces Investigational New Drug Application Submission to Initiate Clinical Trials of NB 1001 for Short Bowel Syndrome

Naia Rare Diseases Announces Investigational New Drug Application Submission to Initiate Clinical Trials of NB 1001 for Short Bowel Syndrome

 

— Mark Pimentel, MD, of Cedars-Sinai to be Principal Investigator–

 

RICHMOND, Calif, November 29, 2016 –Naia Rare Diseases, a biopharmaceutical company developing drugs for Short Bowel Syndrome (SBS) and other rare gastrointestinal diseases today announced that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a Phase 1b clinical trial for its long-acting GLP-1 agonist, NB 1001, in adult patients with SBS.

Mark Pimentel MD FRCPC, chairman of Naia’s board of scientific and clinical advisors and professor of medicine and director of the gastrointestinal motility program and laboratory at Cedars-Sinai Medical Center, will be the principal investigator (PI) of the study.

“We are pleased to have this IND under review by the FDA,” said H. Daniel Perez, MD, President and CEO of Naia Rare Diseases. “We very much appreciate that Dr Pimentel has agreed to lead the clinical trial as PI.  We look forward to providing further details on the progress of the trial in the near future.”

“I am excited to participate in this study,” said Dr Pimentel.  “I believe NB 1001 has the potential to make a significant impact on the lives of patients with SBS, and look forward to working with the team at Naia as they develop NB 1001 further. 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.”

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.

About NB 1001

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.  Licensed from Amunix Operating Inc, NB 1001 uses Amunix’s proprietary “XTEN” technology to extend the half-life of the GLP-1 peptide and allows for up to once-per-month dosing, thus considerably increasing convenience for patients and caregivers. Because of lower overall exposure and dose required (as a replacement therapy) NB 1001 will have an increased safety window compared to other GLP-1 agonists, important as SBS patients are a very fragile population. NB 1001 was previously developed to treat type 2 diabetes and in a 70 patient clinical study demonstrated efficacy and an extended half-life up to 30 days.

 

NB1001 has received orphan drug designation by the FDA.

 

About Naia Rare Diseases

Naia Rare Diseases is a development stage biopharmaceutical company developing novel drugs for rare gastrointestinal disorders.  The company is pursuing three development programs including NB 1001 for Adult SBS, NB 1001 for pediatric SBS and NB 1002, a GLP-2 agonist, an undisclosed orphan gastrointestinal indication.  Naia Rare Diseases has been funded primarily by its parent company, Naia Limited, a company focused on building and funding new biotech companies using de-risked clinical stage assets. For more information, please visit www.naiapharma.com.

 

 

Contact:

Mark Bagnall, Naia Pharmaceuticals

510-926-8237

mbagnall@naiapharma.com

Naia Rare Diseases to Present at 28th Annual ROTH Capital Conference

Naia Rare Diseases Inc to Present at 28th Annual ROTH Capital Conference on Tuesday, March 15, 2016

Richmond, CA–(Mar 11, 2016) – Naia Rare Diseases, Inc., a biotechnology company developing novel treatments for rare gastrointestinal diseases, today announced that it will be presenting at the 28th Annual ROTH Capital Conference on Tuesday, March 15, 2016. The Conference will be held at the Ritz Carlton in Dana Point, California.

The Company’s presentation by H. Daniel Perez, MD, Chief Executive Officer, is scheduled for March 15 at 4:00 p.m. PST, in the private company section.   Dr. Perez will provide an update on the development of NB 1001, the Company’s novel drug candidate for the treatment of Short Bowel Syndrome (SBS), and the other programs in the Company’s pipeline.

Dr. Perez and Mark Bagnall, the Company’s Chief Financial Officer, will be available for meetings during the Conference.

About NB 1001

NB 1001 is a proprietary long-acting version of the GLP-1 agonist, exenatide, developed specifically to treat SBS.   Unlike other GLP-1 agonists, NB 1001 will be dosed as a replacement therapy.  Because of the lower overall exposure and favorable PK profile, NB 1001 will have a superior safety window, an important consideration in the care of SBS patients. NB 1001 works by slowing gastric contractions, increasing the transit time for food in the small bowel, and thus improving the absorption of nutrients. NB 1001 was previously developed to treat type 2 diabetes and in a 70 patient clinical study demonstrated efficacy with no observed side effects.

Naia Rare Diseases intends to file an IND for NB 1001 in the second quarter of 2016, and will conduct a Phase 1b open label, single dose, dose de-escalation study in adult SBS patients in the second half of 2016.  The company believes that a single Phase 2/3 study will be required for product approval.

About Naia Rare Diseases

Naia Rare Diseases develops novel treatments for rare gastrointestinal diseases.  The Company has programs focused on adult and pediatric SBS and other rare gastrointestinal disorders.  Naia Rare Diseases is affiliated with Naia Pharmaceuticals which provides operating infrastructure and support in the development of new companies. For more information, please visit www.naiapharma.com.

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

Naia Rare Diseases appoints Professor Palle Bekker Jeppesen to its Scientific Advisory Board

Naia Rare Diseases, Inc. appoints Professor Palle Bekker Jeppesen to its Scientific Advisory Board

— Appointment Completes Company’s Scientific Advisory Board —

RICHMOND, Calif., January 18, 2016 – Naia Limited, an international drug development company today announced that its subsidiary, Naia Rare Diseases, has appointed Professor Palle Bekker Jeppesen, MD, Head of Research at the Department of Gastroenterology, Rigshospitalet, Copenhagen, to its Scientific Advisory Board.  Professor Jeppesen is a recognized expert in the treatment of Short Bowel Syndrome (SBS) and has been a leading researcher developing new therapeutic approaches for the disease.  He was the principal investigator in the clinical studies that led to the approval of teduglutide (Gattex ®), to date the only FDA-approved product for SBS.

Professor Jeppesen joins current Scientific Advisory Board members, Mark Pimentel, MD, FRCPC, professor of medicine and director of the gastrointestinal motility program and laboratory at Cedars-Sinai, Mark DeLegge, MD, professor of medicine at the Medical University of South Carolina, John DiBaise, MD, professor of medicine at Mayo Clinic, Scottsdale, Arizona, Stuart Kaufman, MD, professor of pediatrics and medical director, pediatric transplantation, Georgetown Medical Center and Farooq Rahman, BSc, Ph.D., FRCP, consultant gastroenterologist & lead physician for intestinal failure, University College Hospital, London.

“We are excited that Professor Jeppesen has agreed to join Naia Rare Diseases Scientific Advisory Board,” said H. Daniel Perez, M.D., co-founder, chairman and chief executive officer of Naia Limited. “He is an acknowledged expert in the treatment of SBS and his addition to our already distinguished board is a testament to the importance of what we are doing.”

Naia Rare Diseases is developing therapeutics for SBS and other rare gastrointestinal diseases.  The company intends to file an IND for its lead program, NB 1001 in the second quarter of 2016, and will conduct a Phase 1b open label, single dose, dose de-escalation study in adult SBS patients in the second half of 2016.  The company believes that a single Phase 2/3 study will be required for product approval.

“I am very happy to join such a group of distinguished colleagues on the Naia Scientific Advisory Board” said Professor Jeppesen.  “I believe Naia is working on potentially significant treatments for SBS and I look forward to working with Naia to bring these therapies to SBS patients in need.”

Professor Palle Bekker Jeppesen is the Head of Research at the Department of Gastroenterology at Rigshospitalet in Copenhagen, Denmark. He is also an Affiliate Professor in the Department of Nutrition, Exercise and Sports at the unit for Clinical and Experimental Nutrition, University of Copenhagen.

His major research interests include patients with short bowel syndrome and intestinal failure with a recent focus on novel therapies for their management, rehabilitation and care. He was principal investigator in the international, multicenter, phase 2 and 3 program, which led to approval of the drug teduglutide by the FDA and EMA to treat SBS.  Professor Jeppesen graduated in medicine from the University of Copenhagen, Denmark, in 1989 and completed his residency in medical gastroenterology at the Rigshospitalet in Copenhagen. He completed his PhD thesis, entitled The significance of the fatty acid chain-length for the clinical effect in the enteral and parenteral nutrition in patients with malabsorption in 1998 and his Doctor’s Degree, entitled Intestinal insufficiency and failure, in 2003.

About NB 1001

NB 1001 is a proprietary long-acting version of the GLP-1 agonist, exenatide, developed specifically to treat SBS.   Licensed from Amunix Operating Inc., NB 1001 uses Amunix’s proprietary “XTEN” technology to extend the half-life of the GLP-1 peptide and allows for up to once-per-month dosing, thus considerably increasing convenience for patients and caregivers. Because of lower overall exposure and dose required (as a replacement therapy) NB 1001 will have an increased safety window compared to other GLP-1 agonists, important as SBS patients are a very fragile population. Exenatide, has been studied in patients with SBS by Dr. Pimentel and demonstrated efficacy by slowing gastric contractions as well as bowel movements and improving nutritional status leading to discontinuation of parenteral nutrition. NB 1001 was previously developed to treat type 2 diabetes and in a 70 patient clinical study demonstrated efficacy with no observed side effects.

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. For more information, please visit www.naiapharma.com.

 

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

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