Royalty Report: Drugs, Disease, Medical – Collection: 256146


Curated Royalty Rate Report
Created On: 2020-07-15, Record Count: 3


This collection of transactions and supporting information was developed using our AI algorithm to curate similar royalty reports into a cohesive collection to support your licensing, transfer pricing or other transaction scenarios where documented royalty rates and/or deal terms are important.
Created On: 2020-07-15
Record Count: 3

Primary Industries

  • Drugs
  • Disease
  • Medical
  • Therapeutic
  • Pharmaceuticals
  • Receptors
  • Central Nervous System

IPSCIO Report Record List

Below you will find the records curated into this collection.  This summary includes the complete licensed property description so that you can review and determine if this collection covers the topics, technology or transaction type that is relevant for your needs.  The full report will include all relevant deal data such as the royalty base, agreement date, term description, royalty rates and other deal terms.  For reference, here is a sample of a full IPSCIO curated royalty rate report: Sample Report

IPSCIO Record ID: 256146

License Grant
The parties agree to enter into this agreement for specified milestones, as well as royalties on any future worldwide net sales of PTX-022.
License Property
PTX-022 is a product candidate being developed to treat pachyonychia congenita (“PC”).  PC is a serious, chronically debilitating genetic disorder that results in malformation of the skin and severely limits the mobility and quality-of-life of those affected. Palvella’s PTX-022 program has received Fast Track and Orphan Drug designations for treatment of PC from the U.S. Food and Drug Administration (“FDA”). PC affects up to 10,000 people in the U.S. and no FDA-approved therapies exist to treat the disorder.

Pachyonychia congenita is a rare genodermatosis due to mutations in one of four keratin genes. It is characterized by dystrophic, thickened nails and painful palmoplantar keratoderma.

Field of Use
This agreement pertains to the drug industry.

IPSCIO Record ID: 258420

License Grant
The parties entered into a collaboration to develop and commercialize gene therapy treatments for Inherited Retinal diseases (IRDs).  The Licensee will receive worldwide exclusive rights to commercialize product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, X-linked retinitis pigmentosa (XLRP) and options to additional IRD programs.
License Property
Under the agreement, the two companies will collaborate in the clinical development of Licensors leading IRD pipeline, including product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3 and X-linked retinitis pigmentosa (XLRP). In addition, the parties are entering into a research collaboration covering Licensor’s pipeline of pre-clinical programs for IRDs. The two companies are also entering into a research collaboration to further develop AAV manufacturing technology as well as clinical and commercial manufacturing supply agreements for the clinical and research programs.

X-linked retinitis pigmentosa (XLRP) is and inherited condition caused by mutations in the RPGR gene. XLRP causes progressive vision loss in boys and young men. The condition begins with night blindness and is followed by progressive constriction of the field of vision. XLRP often results in total blindness and there is no specific treatment for this condition.

Field of Use
Achromatopsia (ACHM) is an inherited retinal disease that prevents cone photoreceptors from functioning. Patients are legally blind from birth and usually suffer from severely reduced visual acuity of 20/200 or worse; a disabling sensitivity to light, or photophobia; total color blindness; and involuntary back and forth eye movements, or nystagmus. There are currently no approved treatments for achromatopsia.

AAV-CNGB3 and AAV-CNGA3, gene therapy candidates designed to restore cone function, are delivered via subretinal injection to the area of the eye where most of the cones in the retina are located. AAV-CNGB3 was granted orphan drug designation (ODD), rare pediatric disease and Fast Track designations by the U.S. Food and Drug Administration (FDA), and orphan medicinal product and PRIME designations by the European Medicines Agency (EMA) for the treatment of achromatopsia caused by mutations in the CNGB3 gene. A Phase 1/2 clinical trial of AAV-CNGB3 in both adult and pediatric patients is underway.

X-linked retinitis pigmentosa (XLRP) represent some of the most severe forms of RP, resulting in early onset in childhood and rapid progression to blindness by the time patients reach 20 to 30 years old. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. There are currently no approved treatments for XLRP.

AAV-RGPR is designed to treat the most common form of XLRP caused by mutations in the RPGR gene. Both rod and cone photoreceptors require RPGR to function. AAV-RPGR has received Fast Track designation and ODD from the FDA and orphan medicinal product designation from the EMA. A Phase 1/2 clinical trial of AAV-RPGR in adult and pediatric patients is underway.

This strategic collaboration agreement is related to development and commercialization of gene therapies for the treatment of inherited retinal diseases (IRDs).  IRDs are a group of rare eye conditions caused by an inherited gene mutation that are often characterized by progressive retinal degeneration which leads to severe vision impairment, loss or blindness.

IPSCIO Record ID: 60411

License Grant
The parties have an agreement regarding the development of Captisol-enabled therapeutics.
License Property
Captisol-enabled SAGE-547 is an allosteric modulator of both synaptic and extra-synaptic GABAA receptors that is in clinical development as an adjunctive therapy, a therapy combined with current therapeutic approaches, for the treatment of Super-refractory status epilepticus (SRSE).   SAGE-547 was granted Fast Track designation, which is intended to facilitate the development and expedite the review of drug candidates that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs, and orphan drug designation, which is intended to facilitate drug development for rare diseases, by the FDA for SRSE.
Field of Use
Licensee is conducting a Phase 3 clinical trial for the development of Captisol-enabled therapeutics for a broad range of debilitating central nervous system conditions.
Disclaimer: The information gathered from RoyaltySource® database was sourced from the U.S. Securities and Exchange Commission EDGAR Filings and other public records. While we believe the sources to be reliable, this does not guarantee the accuracy or completeness of the information provided. Further, the information is supplied as general guidance and is not intended to represent or be a substitute for a detailed analysis or professional judgment. This information is for private use only and may not be resold or reproduced without permission.