HemoSavin Pharma (USA) Announces The First Oral Small Molecule Non-Factor Therapy for Hemophilia. ISTH 2024 Abstract PB0249

Hemophilia, a genetic bleeding disorder, affects over 400,000 people worldwide, with a majority receiving inadequate treatment. Traditional therapies, which often involve frequent and costly injections, pose significant burdens on patients, especially in developing regions. HemoSavin's new therapy aims to revolutionize this landscape by providing a cost-effective, orally administered alternative.

In its peer-reviewed poster presentation, HemoSavin presented that the lead candidate small molecule therapy significantly reduced activated partial thromboplastin time (aPTT) across various plasma samples. At a 5 mM concentration, its therapy reduced coagulation time by 41% in FVIII-deficient human plasma, matching 10% of normal FVIII levels. For FVIII-deficient canine plasma, a 4.16 mM concentration reduced coagulation time by 87%. In FIX-deficient and vW3 human plasmas, the active ingredient(s) in its therapy decreased coagulation times by 51% and 42%, respectively, at 5 mM and 5.82 mM concentrations. Soon-to-be-published studies performed in Professor Steven Pipe’s lab at the University of Michigan showed oral administration of HemoSavin’s therapy reduced blood loss by 70% in FVIII-deficient mice, significantly decreasing bleeding and enhancing clot formation. Our therapy is orally active in mice at doses as low as 6 mg/mouse, confers a high survival rate, and exhibits good shelf life, stability, ADME properties, and safety profiles in preliminary studies. “Since the mouse hemophilia model is an established and well-correlated model to the human disorder, we have high confidence to move forward to the next stage of pre-clinical/pre-IND studies,” said Dr.
Makarand Gore, CTO of HemoSavin. "Such oral or subcutaneous medicine, if proven in human studies will be a 'fantastic' development for doctors and patients alike," explained Dr. Steven J. Pipe.

Advantages Over Existing Treatments:
Current hemophilia treatments, including biologics and gene therapies, are often prohibitively expensive and logistically challenging, placing high burden on the medical system. HemoSavin's small molecule therapy, to be provided as a once-a-day pill, offers a practical and affordable solution featuring:
 
➔ Shelf Stability and Non-immunogenicity: Unlike biologics, our therapy is shelf-stable and non-immunogenic, simplifying storage and distribution.
➔ Reduced Medical System Burden: Does not require clinic visit or clinical skills or supervision to administer.
➔ Cost-effective Manufacturing: Production is cost-efficient, making it accessible for patients globally, particularly in low-resource settings.
➔ World-Wide Distribution: Addresses heretofore unmet needs for hemophilia treatment, especially in the less-developed parts of the world.

Clinical and Market Potential:
The active ingredient(s) in HemoSavin’s therapy has demonstrated robust in vitro and in vivo efficacy, suggesting its potential as an effective therapy for hemophilia A, hemophilia B, and von Willebrand disease. With hemophilia A treatments currently valued at $12 billion annually, this new therapy holds significant market potential.

“We have a pipeline of active ingredient(s) to address various bleeding disorders -- we are not a one trick pony. We are committed to pioneering next-generation therapies that can transform patients' lives. Our innovative approach, corroborated by the studies of esteemed researchers, could significantly impact hemophilia treatment,” stated Ashay Gore, Science officer of HemoSavin.

Acknowledgments:
This breakthrough was supported by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) under award number 1R43HL16233601. We are grateful to Dr. Steven W. Pipe and Dr. Timothy Nichols for their support and encouragement.

About HemoSavin Pharmaceuticals Inc.:
HemoSavin Pharmaceuticals, Inc. is a pre-clinical/Pre-IND stage company dedicated to commercializing the first oral small-molecule non-factor therapy for treatment of Hemophilia A. Our mission is to bridge the treatment gap and enhance the quality of life for patients with hemophilia and other coagulopathies globally.

For more information, please visit HemoSavin Pharmaceuticals.
Press Contact:
Ashay Gore, Science Officer
HemoSavin Pharmaceuticals Inc.
www.hemosavin.com
mgore@yewsavin.com