CAMBRIDGE, Mass.--(BUSINESS WIRE)--Proclara Biosciences, a biotechnology company developing novel therapies for diseases caused by protein misfolding, today announced recent progress across its product pipeline. The company will continue its ongoing Phase 1b trial of NPT088 in patients with Alzheimer’s disease following the positive recommendation of the trial’s independent data monitoring committee. This recommendation is based on a review of interim safety data compiled from the trial to date, which show that NPT088 was safe and well-tolerated in patients treated at a dose of 0.6 mg/kg once monthly for six months. Proclara remains on track to report topline data from all trial participants in the summer of 2018.
In addition, Proclara announced that it will develop NPT189, its next generation drug candidate, for the treatment of orphan peripheral amyloidoses. NPT189 utilizes Proclara’s proprietary General Amyloid Interaction Motif (GAIM) technology to target multiple toxic protein aggregates, including amyloid-β (Aβ), tau, α-synuclein, antibody light chain (AL), and transthyretin (TTR). Proclara expects to file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) in the first half of 2018.
“We are encouraged by the initial interim data from our Phase 1b trial of NPT088 in patients with Alzheimer’s disease. These data demonstrate that NPT088 is generally safe and well tolerated given in repeat doses, and it may overcome hurdles that have historically limited progress in the space,” said Suzanne Bruhn, Ph.D., president and chief executive officer of Proclara. “We are also pleased to announce our development strategy for NPT189, the second compound to emerge from our GAIM-based technology platform. By simultaneously targeting multiple misfolded proteins, our therapies have great potential to improve the lives of patients with diseases driven by the toxic effects of misfolded protein deposits, including age-related neurodegenerative diseases and orphan amyloidoses.”
NPT088 Phase 1b Study Details
Proclara’s Phase 1b study of NPT088 was initiated in September 2016 and is designed to evaluate the safety and tolerability of multiple doses of NPT088, as well as its pharmacokinetic, immunogenicity, and pharmacodynamic characteristics, as measured by amyloid-β and tau PET imaging, in patients with mild-to-moderate Alzheimer’s disease and β-amyloidosis and tauopathy. The trial is a randomized, double-blind, placebo-controlled study that will enroll up to 66 patients in four dose cohorts. The company is currently enrolling the second patient cohort.
Proclara expects to release topline efficacy data in the summer of 2018. Successful completion of the Phase 1b study is expected to support Phase 2 and 3 development programs for Alzheimer's disease and Parkinson’s disease.
About Alzheimer’s disease
Alzheimer’s disease is the most common neurodegenerative disease and the leading cause of dementia. A progressive and irreversible brain disorder that impacts memory and thinking skills, Alzheimer’s often affects the ability to carry out basic daily activities. The disease is characterized by the buildup of misfolded protein aggregates in the brain, including plaques, made of aggregated β-amyloid, tangles, made of aggregated tau, and sometimes α-synuclein aggregates. These toxic aggregates cause brain dysfunction and eventually, nerve cell death. Alzheimer’s affects approximately 47 million victims worldwide, and that number is expected to grow to 132 million by 2050.1
About Peripheral Amyloidoses
Peripheral amyloidoses are a group of systemic diseases caused by the deposition of misfolded protein aggregates, called amyloid deposits, that can lead to organ dysfunction and potentially organ failure. Clinically well-defined amyloidoses include primary amyloidosis (also called AL or light chain), and transthyretin amyloidosis (also called ATTR), which includes inherited forms caused by mutations in the TTR gene. There are believed to be about 4,000 new cases of AL amyloidosis annually in the United States,2 while the disease-causing mutations in TTR occur at the rate of approximately 0.1% in Caucasians and approximately 4% in African Americans. Approximately 50,000 people worldwide suffer from TTR.3, 4
About Proclara Biosciences
Proclara Biosciences is a biotechnology company advancing product candidates developed based on its proprietary GAIM technology, which is capable of simultaneously targeting multiple toxic misfolded proteins. The broad applicability of the GAIM technology enables the company to target multiple protein misfolding diseases, including neurodegenerative diseases and orphan systemic amyloidoses. Lead GAIM drug candidate, NPT088, is in clinical development for the treatment of Alzheimer’s disease. For more information, please visit proclarabio.com.
1. Alzheimer’s Disease International, World Alzheimer report 2016, (www.alz.co.uk)
2. Amyloidosis Research Consortium, Guidance for Industry – AL Amyloidosis – Developing Drugs for Treatment, Dec 2016 (www.arci.org)
3. Ando, et al., Orphanet J Rare Dis. 2013
4. Ruberg, et al., Circulation. 2012