THE PRODUCT

Spx81-5

Medical Need

Diabetes is generally described as the largest epidemic of the 21st century; in 2015, 9.4% of the U.S. population had diabetes, totaling 30.3 million adults.

Diabetic retinopathy (DR) is a leading cause of blindness, with a prevalence of 35% of diabetics. Existing injectable products cannot be used early-stage, fail to elicit a response in ~30% of patients, and often fail to prevent long-term retinal thinning. The total cost of U.S. retinal vision loss was estimated at $8.7 billion in 2013.

The Product

PEDF is a 418-amino acid polypeptide in the serpin gene family secreted by the retinal cells.

The protein is widely recognized for its neuroprotective, anti-inflammatory, and anti-angiogenesis activities in the retina and other tissues and may be deficient in diabetic patients. Skyran has isolated a 17-mer minimum active region of PEDF and used a series of conservative peptide modification strategies to obtain several novel, more active PEDF mimetics.

Spx81-5, the lead PEDF mimetic, has increased biological activity over the native fragment, shows no evidence of toxicity in preliminary studies, and adopts a well-defined alpha helical structure making it a pharmaceutically attractive molecule. Skyran has formulated Spx81-5 for topical use as an eye drop that shows bioavailability in the Ins2Akita genetic mouse model of DR and in the non-human primate eye. Spx81-5 and several analogs are protected by U.S. patent 9,611,314, filed September 12, 2014 and issued April 4, 2017.

Target Market


 

Globally, there are ~93 million people with DR; over eight million of these patients are in the United States.

The National Eye Institute projects that by 2030, 11.3 million Americans will have DR. Many of these patients require early treatment and prefer an eye drop over regular intraocular injections.

Target Patient Segments

DR patients

  1. with early-stage disease,
  2. who prefer drops over intraocular injections, or
  3. who have failed anti-VEGF therapy.
  • 93 million people with DR
  • 93 million people with DR
  • 93 million people with DR

Competitive Advantages


 

  • Current treatments fail to address early-stage retinal damage. Skyran will test Spx81-5 in early disease.
  • Some patients respond transiently and become resistant to anti-VEGF therapies, while approximately 30% of patients do not respond at all. PEDF therapy may be useful in these patients.
  • Anti-VEGF treatment fails to prevent disease progression; retinal thinning is evident over 2-5 years
  • Anti-VEGF therapies require regular ocular injections (every 4-6 weeks) in a doctor’s office. Our data-to-date show the utility of Spx81-5 in a drop formulation.

Business Model


 

Skyran will use an efficient semi-virtual model. We will keep critical path activities in-house and use established third-party CRO’s with expertise and lower costs to run non-clinical projects (e.g., toxicology, manufacturing, PR&D) until we have submitted an IND.

Where possible, we will use consultants on a project basis to limit fixed cost. Our initial key consultants will include a preclinical development lead, as well as experts in PK/Toxicology, CMC, and Regulatory. To support our ongoing preclinical work and clinical planning, we will build a Scientific Advisory Board of experts in retinal disease.

STRONG INTELLECTUAL PROPERTY

  • Issued U.S. patent

  • Filed 9/12/2014, issued 4/4/2017

  • Composition of matter for lead compound and five analogs

  • Includes administration by eye drop, foam, gel, or injection

  • Covers use in diabetic retinopathy, retinopathy of prematurity, glaucoma, and more

  • Divisional patent filed 4/3/2017

  • Includes 16 additional analogs

Fund Strategy


 

Skyran has a three-part funding strategy:

1. Actively pursuing non-dilutive funding. Filed $225,000 Phase I STTR application in Sept. 2017. If successful, Skyran will apply for a Phase II STTR in 2018. Skyran has also been awarded several other small grants and is seeking other grant opportunities.

2. Through the University City Science Center’s Phase 1 Ventures program, Skyran received $25,000 in seed funding and is eligible for up to $450,000 in matching funds for additional non-dilutive funding.

3. Seeking angel/venture investment to provide balance of funds required to achieve IND. One individual life sciences investor already committed.

2018: $600,000-$750,000

  • Engage consultants for PK/Toxicology, CMC, Regulatory
  • Proof-of-concept in second animal model, including visual acuity data
  • Validation of eye drop PK in second animal model
  • Initiate IND-enabling studies

2019: $1.4-2.4 million

  • Formulation process development and scale-up
  • File IND

2020-2022

  • Phase I/IIa clinical trials
  • Expand capabilities: hire head of R&D and clinical operations lead