Nobias Therapeutics, a clinical-stage company developing novel small-molecule therapeutics for rare diseases, has announced its intent to focus its resources on its development programs following receipt of positive data from a multi-center, randomized, double blind, placebo-controlled Phase 2 clinical trial of its lead asset, NB-001.
PHILADELPHIA, Nov. 14, 2024 /PRNewswire-PRWeb/ -- Nobias Therapeutics, a clinical-stage company developing novel small-molecule therapeutics for rare diseases, has announced its intent to focus its resources on its development programs following receipt of positive data from a multi-center, randomized, double blind, placebo-controlled Phase 2 clinical trial of its lead asset, NB-001. NB-001 is a small-molecule metabotropic glutamate receptor (mGluR) modulator for the potential treatment of the neuropsychiatric symptoms associated with 22q11 deletion syndrome (22q11DS), the most common human microdeletion.
Children living with 22q11DS, also known as DiGeorge syndrome, typically experience a distinctive mix of neuropsychiatric conditions that include elements of ADHD, anxiety, and autism. Currently, there are no population-specific approved therapies for these conditions, leaving many patients with inadequate treatment options and resulting in a significant burden for their parents and other caretakers. As an mGluR activator, NB-001 is uniquely positioned to help compensate for the disruption to neuronal signaling caused by this deletion—which is responsible for the often severe neuropsychiatric symptoms experienced by 22q11DS patients.
"After reviewing the positive data from the Phase 2 clinical trial, which demonstrate the safety and tolerability of NB-001 – and a clear efficacy signal – we believe this compound has the ability to change patient lives," said Patrick Dougherty, CEO. "Following development of a disease-specific clinical global impression (CGI) assessment scale this summer, we are now focused on Q4 discussions with the FDA to clarify the regulatory path to advance NB-001 into a registrational study for 22q11DS."
As part of this sharpened focus, Patrick Dougherty has been named CEO at Nobias Therapeutics. Dougherty, who had been consulting for Nobias since 2021, is a life sciences executive with more than 20 years of experience at the intersection of science and business across a range of leadership roles, including a decade at GSK in R&D strategy and business development, as part of the management team at WindMIL Therapeutics, and as an engagement manager at L.E.K. Consulting. Prior to joining Nobias, Dougherty established Beech Street Advisors, LLC, a biotech advisory practice supporting early-stage therapeutic companies by providing hands-on, functional leadership in business strategy, business development, and executive operations.
"The entire Board is thrilled to have Patrick join Nobias in this capacity," said Brian Halak, Nobias co-founder, Chairman of the Nobias Board of Directors, and a Partner at Domain Associates and Medical Excellence Capital. "Following receipt of the impressive Phase 2 data, we immediately recognized Nobias' 22q program as a groundbreaking and critical asset deserving of our full commitment. NB-001 is going to bring better treatment options to children with 22q11DS, and holds significant expansion potential – for adults with 22q11DS, and more broadly, other patients living with comorbid psychiatric symptoms that may be driven by genetic disruptions to glutamatergic signaling."
As a result of these changes, the company has also relocated the base of its operations to its office in the University City section of Philadelphia. This location provides access to local medical and scientific expertise and to the robust pool of late-stage development talent present in the Philadelphia pharma/biotech ecosystem.
Visit the company's newly redeveloped website, http://www.nobiastx.com, to learn more.
About Nobias Therapeutics
Nobias Therapeutics is a clinical-stage company developing novel small molecule therapeutics for rare diseases. Our unique access to rich genomic and health datasets and strong relationships with pediatric academic medical centers across North America allow us to rapidly advance drug discovery for genetically-defined rare diseases, with an initial focus on pediatric indications, but expansion opportunities in adults and non-rare indications. To learn more, please visit http://www.nobiastx.com and follow Nobias Therapeutics on LinkedIn.
Media Contact
Nobias Therapeutics, Nobias Therapeutics, 315-463-2325, [email protected], www.nobiastx.com
SOURCE Nobias Therapeutics
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