NLM-001
A Potential Best-in-Class Hedgehog Inhibitor
At Nelum Pharma, we are focused on leveraging our expertise to target fibrosis and various forms of cancer. Our innovative approach and research drive our pursuit of impactful therapies for these challenging conditions.
Hedgehog Pathway Inhibitor
NLM-001 is an inhibitor of the hedgehog pathway initially developed by Takeda and known as Tak-441. Nelum unlicensed the molecule and renamed it as NLM-001.
Safety and
Efficacy
NLM-001 has been administered to 60 patients in two clinical trials, demonstrating a good safety profile as well as efficacy.
PK Profile as Differentiator
NLM-001 PK profile is linear and dose proportional with rapid absorption and fast elimination while most hedgehog inhibitors have a non-linear AUC and a slow systemic elimination.
About Pulmonary Fibrosis
Pulmonary Fibrosis is a progressive, chronic fibrosing disease. With the only curative option being a lung transplant which is limited to a low number of patients.
1. An Unmet Clinical Need
Approved therapies slow down the disease progression but don't reduce the fibrosis or increase lung capacity.
Patients diagnosed with IPF have a median survival of 3 to 5 years.
2. An Orphan Disease
In the United States, approximately 50,000 cases are newly diagnosed and 40,000 patients die each year.
The prevalence is estimated at 2.4 cases per 10,000 people and the incidence is 0.75 new cases per 10,000 people.
The Role of the Hedgehog Pathway in Fibrosis
In the context of pulmonary fibrosis, the Hedgehog pathway has been implicated in the pathogenesis and progression of the disease, influencing key aspects such as fibroblast activation, myofibroblast differentiation, and extracellular matrix (ECM) deposition.
Step 1:
Chronic Lung Injury
Step 2:
Activation of Hedgehog Pathway
Step 3:
Key Effects
Fibroblast activation and myofibroblast differentiation:
Produce excessive ECM proteins like collagen. This contributes to the fibrotic scar formation that characterizes pulmonary fibrosis.
Fibroblast proliferation:
Hedgehog signaling enhances fibroblast proliferation, which exacerbates fibrosis by increasing the number of fibroblasts in the lung.
Inflammation and epithelial-to-mesenchymal transition (EMT):
They lose their epithelial characteristics and gain mesenchymal properties, further fueling fibrosis.
Meet The Team
- Founders -
Manuel Hidalgo, MD, PhD
Chairman of the Scientific Advisory Board
Chief, Division of Hematology and Medical Oncology at Weill Cornell Medicine
Member of the Board at BMS
Member of the Board at Guardant Health
Evelio Perea, MSc
CEO and Chairman of the Board
Serial entrepreneur
Founder of PH Clinical Research, Personalized Oncology Services, PX Source and Science Tools
Laureano Simon, PhD
Serial entrepreneur
Founder of Oncomatryx, Sunrock, and other biotech companies, including Patia and Progenika
- Scientific Advisory Board -
Dr. David Wilkes
Dean Emeritus of the University of Virginia Medical, VA
Dr. Anna Podolanczuk
Associate Professor of Medicine. Weill Cornell Medicine, NY
Dr. Claudia Valenzuela
Pulmonologist. Chair of ILD Unit.
Hospital La Princesa, Madrid, Spain