Vascarta seeks strategic partners to help develop and commercialize its unique technologies. We are dedicated to bringing to market New Molecular Entities (NMEs) that treat pain and inflammation. We have three foundational assets:
Asset #1 – A patented topical, transdermal delivery technology (Vasporta™) that facilitates superior clinical translation of small molecules compared to oral administration.
- The Vasporta™ technology has directly led to our lead product candidate Vasceptor® (VAS-101) which effectively delivers curcumin transdermally. Moreover, Vascarta already has successfully applied the Vasporta™ technology to other compounds beyond the curcumin used in Vasceptor®.
Asset #2 – Our Lead Product Candidate Vasceptor® (VAS-101) harnesses the proven anti-inflammatory efficacy of curcumin through patented transdermal delivery which overcomes the limited bioavailability and suboptimal effectiveness of oral dosing.
- Vasceptor® (VAS-101) is being clinically studied for its potential to enable significant advances in the treatment of Sickle Cell Disease and Osteoarthritis. Both of these are indications with large sales potential and substantial unmet medical needs. Phase 1 clinical trials for Sickle Cell Disease and Osteoarthritis were initiated 1Q2025 and expected to complete by 4Q2025.
- Moreover, Vasceptor® has demonstrated promising pre-clinical efficacy in several additional health conditions including Red Blood Cell Preservation & Storage, Hypertension/High blood pressure, and Inflammaging.
Asset #3 – Exclusive license rights to a novel method for making existing cancer therapies safer and more effective through unique and complementary mechanisms of action via new and unique linker chemistry.
- This “STO” technology already has yielded promising preclinical results against Glioblastoma and Pancreatic Cancer and also can be applied to other solid forms of cancer.
- In addition, a pharmaceutical partner may use Vascarta’s Vasporta™ and anti-cancer linking technologies to simplify drug delivery, increase bioavailability of oral administered assets, improve drug efficacy, improve safety profile, and/or strengthen patent protection of its own molecules.
Market Research For Selected Indications
- Sickle Cell Disease
- Osteoarthritis
- Glioblastoma
- Pancreatic Cancer
Sickle Cell Disease Overview
As described below, Sickle Cell Disease has suboptimal approved therapies with unmet needs that may be addressed by Vasceptor®. We anticipate that addressing these unmet needs could generate annual Vasceptor® (VAS-101) sales of $0.5 billion (USD) in the USA and Europe:
Suboptimal Approved Sickle Cell Disease (SCD) Therapies
- SCD is lacking safe and effective therapies for patients of all ages
- Poor patient compliance with existing therapies
- Current approved therapies do not fully address the consequences of SCD
- Existing treatments do not adequately reduce blood transfusions
- Patients continue to experience frequent pain and rely on opioids
- Physicians are discouraged from prescribing opioids
- Recently approved gene therapies are cost prohibitive ($2+ million)
- Oxbryta and Adakveo were recently withdrawn from the market
FDA-Approved SCD Therapies
| Product | Company | Dosage Form | Mechanism of Action | Limitations | List Price | Commercial Comment |
|---|---|---|---|---|---|---|
| Hydroxyurea | Generic | Oral Capsule | Raises Fetal Hemoglobin | Poor compliance | $500–$1,000/year | Generic |
| Endari | Emmaus Life Sciences | Oral Powder | Regulates and prevents oxidative damage in the RBC | Poor compliance, difficult to take | >$40K/year | Generic available as of July 2024 |
| Adakveo | Novartis | IV Infusion | P-selectin blocker, prevents RBC from sticking | Serious adverse events and side effects | >$100K/year | Future uncertain, withdrawn from EU in Aug 2023 |
| Voxelotor | Pfizer | Oral Tablets | Inhibits HbS polymerization | Serious side effects | >$100K/year | Withdrawn globally in Sept 2024 |
| Casgevy | Vertex | IV Infusion | Gene Editing | Cost prohibitive, very limited use | $2.2 million (one time) | One-time use |
| Lyfgenia | Bluebird Bio | IV Infusion | Gene Editing | Cost prohibitive, very limited use | $3.1 million (one time) | One-time use |
Vasceptor® Represents A Potential Solution for SCD Patients
Vasceptor® addresses limitations of FDA-approved SCD therapies:
- Relieves pain
- Reduces systemic inflammation
- Stabilizes red blood cells
- Limits organ damage
- Prophylactically prevents vaso-occlusive crises
- Limits oxidative damage
- Prevents mitochondrial dysfunction
- Prevents and reverses endothelial dysfunction
- Enhances Nitric Oxide production in the vasculature
- Does not alter hemoglobin oxygen binding properties
- Curcumin is FDA recognized as a GRAS ingredient
- Safe and easy to use for patients of all ages
Market Opportunity – Sickle Cell Disease
- Global SCD Market: Estimated to garner around USD $50.45 billion by 2032 and is expanding at a healthy CAGR of 35.1% from 2023 to 2032
- BioPharma is heavily invested in SCD R&D
- High reimbursement for sickle cell therapies has been established in the USA & EU
- *Net price target per patient for Vasceptor®
- $36,000 annually in the U.S.
- $23,400 annually in the E.U.
- *10% market share US & EU = $500,400,000 in net sales
- EX-US (other than EU) not included in Vascarta® financial model
- Global revenue opportunity in the billions
Osteoarthritis Overview
As described in the slides below, there are many Osteoarthritis sufferers, but current medications have substantial limitations that may be addressed by VAS-101 (Vasceptor®). Addressing these needs could generate annual sales of $1 billion (USD) in the USA alone:
Large Number of Osteoarthritis (OA) Sufferers
- Approximately 32.5 million U.S. adults have OA
- Around 500 million patients globally
- 71% of OA patients feel fatigued
- 51% of OA patients have trouble walking for 15 minutes
- Incidence of OA is on the rise
- Almost half of adults with heart disease or diabetes have arthritis
- Arthritis is the leading cause of disability among U.S. adults
- Huge economic impact and cost to the healthcare system:
- Lost earnings: $71.3 billion
- Medical cost: $65.5 billion
- In 2006, OA led to an estimated 2 million fractures in the U.S.
Limitations of Current Osteoarthritis (OA) Therapies
- Opioids: Highly addictive pain medication – side effects include:
- Respiratory depression
- Apnea
- Respiratory arrest
- Circulatory depression
- Hypotension and shock
- Constipation
- Due to addiction potential, prescribing restrictions and scrutiny exist for healthcare providers who use opioids
- NSAIDs: Long-term use not recommended – serious side effects include:
- Stomach bleeding, ulcers, gastrointestinal issues
- Increased risk of strokes, heart attacks, heart-related deaths
- Kidney problems and allergic reactions
- Dangerous drugs for elderly patients
Vasceptor® Represents A Potential Solution for OA Patients
- Highly effective anti-inflammatory & anti-pain effects
- Fast acting
- Convenient dosing
- Once daily or LESS frequently
- Strong safety
- NO addiction
- NO cardiovascular, kidney, liver, or GI side effects
- Only known “side effect” is yellow color where product is applied
Market Opportunity - Osteoarthritis
| Current Drugs for OA | MOA | Availability | Generic or RX |
|---|---|---|---|
| NSAIDs | Anti-inflammatory | OTC and Prescription | Generic Market |
| Prednisone | Immune Suppressant | Prescription Only | Generic Market |
| Gabapentin | Alters Brain Activity–Neurotransmitters | Prescription Only | Generic Market |
| Cymbalta | Alters Serotonin and Norepinephrine | Prescription Only | Generic Market |
| Muscle Relaxants | CNS Depressants | Prescription Only | Generic Market |
| OPIOIDS | Block Pain Signals in the Brain & Body | Prescription Only | Generic Market |
| Vasceptor®* | Multi Modal – Activator of SIRT1 & Nrf2 | Prescription Only | Branded |
*Currently in clinical development
Glioblastoma and Pancreatic Cancer Financial Opportunity Overview
There is a substantial unmet need given the relative 5-year survivability of Glioblastoma is only around 5% and the 5-year survivability of Pancreatic Cancer is only 13%. As an example of the value from anti-cancer technology using novel means to overcome tumor defenses which is offered via the Vascarta-CUNY anti-cancer linker technology, Merck acquired a Yale-developed anti-cancer technology platform for $30 million upfront, plus the potential for up to $1.3 billion in milestone payments.
Conservative annual net sales projections for the Vascarta anti-cancer linking technology are $300 million for Glioblastoma and over $400 million for Pancreatic Cancer.
We invite prospective partners to contact:
- Vascarta Chairman, CEO & President, Dr. Richard Prince, rprince@vascarta.com
- Vascarta Chief Business Officer, Mark Diamond, mdiamond@vascarta.com