Royalty Report: Drugs, Drug Discovery – Collection: 314338

License Grant

Institute, not-for-profit healthcare system, hereby grants to Licensee (I) an exclusive license, with the right to grant sublicenses in multiple tiers, under the Institute Technology to research, develop, make, have made, use, offer to sell, sell, have sold, import and export Compounds and Products in the Field in the Licensed Territory; and (ii) a non-exclusive license, with the right to grant sublicenses in multiple tiers, to use Institute Know-How relating to Products for research purposes, and for commercial purposes for exploitation of any Institute Patents not otherwise exclusively committed to a Third Party outside of the Licensed Territory. For clarity, the license granted to Licensee under this Section 2.1(a) does not include the right for Licensee to practice any Institute Patent or use any Institute Know-How to develop, make, use or sell compounds or products that are proprietary to Institute other than Compounds or Products.

License Property

Compound means any of the following (a) Triheptanoin; (b) any odd  number carbon chains consisting of 5-15 carbon atoms attached to a carbohydrate glycerol backbone, and in each case of (a) and (b), including any conjugate, prodrug or other physical form thereof. Triheptanoin treatment is for patients with adult polyglucosan body disease.

Institute Patents means any and all Patents Controlled by Institute or its Affiliate(s) as of the Effective Date or thereafter during the Term that (i) claim the composition of matter of, or the method of manufacturing or using, any Compound or Product; or (ii) that otherwise relate to, or are reasonably necessary for, the use, development, manufacture or commercialization of any Compound or Product, including the Patents set forth.

7,754,764 – Fatty acid nutritional supplement
7,705,048 – Method of suppressing the effects of the translocase deficiency of a human infant comprising administration of a seven-carbon fatty acid
7,592,370 – Fatty acid treatment for cardiac patients

Product means any nutritional or pharmaceutical product, including all dosage forms and formulations, containing one or more Compound(s) as an Active Ingredient(s) (alone or as part of a Combination Product). Except when referred to in the Net Sales definition in describing how to calculate the Net Sales of Combination Products, all references to Product in this Agreement shall be deemed to include Combination Products. Two Products shall be deemed the same Product if they contain the same Compound as the Active Ingredient.

Patents means all patents and patent applications and any patents issuing therefrom (which for the purpose of this Agreement shall be deemed to include certificates of invention and applications for certificates of invention), including all divisionals, continuations, substitutions, continuations-in-part, converted provisionals, continued prosecution applications, adjustments, re-examinations, reissues, additions, renewals, revalidations, extensions (including patent term extensions, and supplemental certificates and the like), registrations, pediatric exclusivity periods of any such patents and patent applications, and any and all foreign equivalents of the foregoing.

Institute is a research center focused on finding prevention therapies and treatments for diseases and illnesses.

Field of Use

Field means any and all human and animal prophylactic, therapeutic and diagnostic uses for the treatment adult polyglucosan body disease.

Adult polyglucosan body disease (APBD) is a rare, genetic disorder characterized by a deficiency of glycogen-branching enzyme, resulting in the accumulation of polyglucosan bodies in muscle, nerve and various other tissues of the body. Polyglucosan bodies are composed of large, complex, sugar-based molecules.

Adult polyglucosan body disease (APBD) is an orphan disease and a glycogen storage disorder that is caused by an inborn error of metabolism. Symptoms can emerge any time after the age of 30; early symptoms include trouble controlling urination, trouble walking, and lack of sensation in the legs. People eventually develop dementia.

Licensee is a biotechnology company focused on the discovery and development of innovative therapeutics for patients with rare and ultra-rare genetic diseases.

License Grant

License to Commercialize Collaboration Compounds –  Licensor grants to Licensee a worldwide, exclusive license, with the right to sublicense, under the Licensor IP and Licensors interest in the Collaboration IP, to make, have made, use, sell, offer to sell and import Collaboration Compounds, and/or Collaboration Products based on such Collaboration Compounds, for any and all uses and indications in the Licensee Field.

License to Compounds (Other than Collaboration Compounds)  Derived from Licensee Compounds – Licensor grants to Licensee a worldwide, exclusive license, with the right to sublicense, under the Licensor IP and Licensors interest in the Collaboration IP, to make, have made, use, sell, offer to sell and import Collaboration Compounds, and/or Collaboration Products based on such Collaboration Compounds, for any and all uses and indications in the Licensee Field.

License Property

Collaboration Product shall mean a pharmaceutical product containing a Collaboration Compound that is sold by Licensee or Licensor, or their respective Sublicensees or Affiliates.  It is understood that, in the event any Collaboration Compound is sold for therapeutic purposes, such Collaboration Compound shall be deemed a Collaboration Product and shall be subject to milestones and royalties as set forth in this Agreement.

Collaboration Compound shall mean a Licensor Compound or a Non-Exclusive Compound or a compound that is derived, directly or indirectly, from a Licensee Compound, Licensor Compound, or Non-Exclusive Compound; in each case, that modulates the biological activity of a Collaboration Target at a level of potency to be established by the JRC.  A compound shall be deemed to have been derived directly from another compound if it (i) is the result of a chemical modification made to such a compound, (ii) is otherwise obtained from a chemical synthesis program based on one or more such compounds, (iii) is based on proprietary structure-activity data obtained from the testing of one or more such compounds, or (iv) is specifically or generically within the scope of one or more claims of any patent application or patent filed by Licensor or Licensee or their Affiliates to protect any compound in category (i), (ii) or (iii) above.  A compound shall be deemed to have been derived indirectly from another compound if it is the result of a series of iterations of any or all of (i) through (iv) above.  For purposes of clarity, any compound synthesized in the course of the Collaboration and based on a Licensee Compound, Licensor Compound, or Non-Exclusive Compound which is active against a Collaboration Target at the level of potency established by the JRC shall be deemed a Collaboration Compound.  Joint Research Committee or “JRC” shall mean the entity organized to manage the scientific implementation of the Collaboration.

Licensee IP shall mean Licensee Patents and Licensee Know-how.

Collaboration IP shall mean Collaboration Patents and Collaboration Know-how.  Collaboration IP shall not include Licensor IP or Licensee IP.

Licensor IP shall mean Licensor Patents and Licensor Know-how.  Licensor IP shall not include any intellectual property owned or licensed by Licensor or its Affiliates relating to the ECLiPSâ„¢ technology (i.e., the creation or use of encoded combinatorial libraries or any tag and/or marker compounds).

Licensee Patents shall mean all Patents in the Territory Controlled by Licensee or its Affiliates as the effective date, or any other Patent Controlled by Licensee during the Term; in each case, necessary for the discovery, development, manufacture, importation or use of one or more Collaboration Compounds and/or the development, manufacture, use, sale, importation or commercialization of corresponding Collaboration Products.

Licensor Patents shall mean all Patents in the Territory Controlled by Licensor or its Affiliates as the Effective Date, or any other Patent Controlled by Licensor during the Term; in each case, necessary for the discovery, development, manufacture, importation or use of one or more Collaboration Compounds and/or the development, manufacture, use, sale, importation or commercialization of corresponding Collaboration Products.

Collaboration Patents shall mean (i) all patents and patent applications claiming any invention or discovery which was conceived or reduced to practice during the Collaboration Term and in the course of the Collaboration, by employees or agents of Licensee, Licensor or any of their respective Affiliates, either alone or jointly (including, without limitation, the synthesis and composition of matter of any Collaboration Compound, or method of use thereof; and (ii) any divisions, continuations, continuations-in-part, reissues, reexaminations, extensions or other governmental actions which extend any of the subject matter of the patent applications or patents in (i) above, and any substitutions, confirmations, registrations, revalidations, or additions of any of the foregoing, in each case, which is owned or Controlled, in whole or part, by license, assignment or otherwise by Licensor or Licensee during the term of this Agreement; provided, however, that Collaboration Patents shall not include any Licensee Patents or Licensor Patents.

Licensor Compound shall mean any compound brought to the Collaboration by Licensor, which is Controlled by Licensor at or after the Effective Date, and which is proprietary to Licensor by virtue of being within the Licensor IP.

Field of Use

The primary objective of the alliance is to identify active molecules and bring them forward to clinical proof of concept, yielding novel candidates for drug development in various therapeutic areas.

Licensor Field shall mean, with respect to each Collaboration Compound for which Licensor has received approval to develop pursuant, all human and animal disorders and diseases treated by modulation of a Collaboration Target by such Collaboration Compound.

Licensee Field shall mean, with respect to each Collaboration Compound, all human and animal disorders and diseases treated by modulation of a Collaboration Target by such Collaboration Compound.

License Grant

Licensor grants to the Licensee of Switzerland an exclusive right, exercisable at any time during the Option Period, for Licensee as part of a mutually executed Collaboration and License Agreement to enter into an exclusive license under the Licensor Patents and Know-How and a sublicense of the Licensor controlled Patents to develop, make, have made, use, sell, have sold, offer to sell, commercialize and import ACER-001 in the Field in the Relief Territory, on the terms described in this Agreement.

License Property

Axxx-001 means Licensors taste-masked, immediate release powder formulation of sodium phenylbutyrate (NaPB) for the potential treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and maple syrup urine disease (MSUD).

Licensor Know-How means all unpatented inventions, technology, methods, materials, including biological and pharmaceutical materials, know-how, studies, pre-clinical and clinical data, including toxicology, safety data, and bioequivalence studies, tests and assays, reports, manufacturing processes, including manufacturing batch records to support regulatory approvals, regulatory filings and approvals and other information owned or licensed by Licensor as of the Effective Date with the right to license or sublicense without violating the terms of any agreement to which Licensor is party, in each case that relates to Axxx-001 or the subject matter claimed by Licensor Patents or Licensor controlled Patents.

Licensor patents are
Palatable comositions including Sodium Phenylbutyrate and uses thereof, and,
Administration of Sodium Phenylbutyrate in a fasted state to treat urea cycle disorders.

Licensor controlled patent is Methods of modulation of branched chain acids and uses thereof.

Field of Use

The Field means all indications or treatments of humans using Axxx-001,  for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD).

Urea cycle disorder (UCD) is an inherited disease that affects how the body removes the waste that is made from breaking down protein. Everyone needs protein, which is found in foods like dairy products, meat and fish.
Infants with a urea cycle disorder appear normal at birth but rapidly develop cerebral edema and the related signs of lethargy, anorexia, hyper- or hypoventilation, hypothermia, seizures, neurologic posturing, and coma.

Classical Maple Syrup Urine Disease (MSUD) is a disease of infancy which is an inherited disorder of metabolism of branched-chain amino acids (BCAA).
Maple syrup urine disease (MSUD) is an autosomal recessive metabolic disorder affecting branched-chain amino acids. It is one type of organic acidemia. The condition gets its name from the distinctive sweet odor of affected infants' urine, particularly prior to diagnosis and during times of acute illness.

License Grant

Licensor of Spain grants to Licensee of Switzerland, license rights over the Technology in the Field  An exclusive, even as to Licensor, worldwide, assignable, transferable license, including the right to sublicense through multiple tiers of sublicense, to Licensors IP to make, have made, use, import, export offer to sell, sell and have sold, copy, modify, perform, display, create derivative versions of Products in the Field or otherwise to exploit Licensor’s IP in the Field, for any lawful purpose; and,

An exclusive, even as to Licensor, worldwide, assignable, transferable sublicense, including the right to sublicense through multiple tiers of sublicense, to Institutions’ IP to make, have made, use, import, offer to sell, sell and have sold, copy, modify, perform, display, create derivative versions of Products in the Field or otherwise to exploit the Institutions’ IP in the Field to any lawful purpose.

License Property

The patents are
EP 16382660.5 – Isoquinoline compounds, methods for their preparation, and therapeutic uses thereof;
EP 16382672.0 – Heteroaryl compounds and their use in the treatment of gaucher’s disease;
PCT/EP2014/066392 – Di(hetero)arylamides and sulfonamides, methods for their preparation and therapeutic uses thereof; and,

EP15382019.6 – Heteroarylaminoisoquinolines, methods for their preparation and therapeutic uses thereof

The Institutions’ IP and Licensor’s IP are hereinafter jointly referred to as the Technology.
—  Institutions’ IP shall mean

(i) the patent no. EP 11380102 titled Method Of Binding Site And Binding Energy Determination By Mixed Explicit Solvent Simulations; (ii) any divisionals, substitutions, continuations and continuations-in part of the foregoing patent (excluding claims of the foregoing that is not entitled to the priority date of patent); (iii) all reissues, reexaminations, extensions, registrations, patent term extensions and restorations, supplementary protection certificates and renewals of any of the foregoing patent; and (iv) the Intellectual Property rights in connection thereto, including the corresponding know-how and software; in each case, anywhere in the world.

—  Licensor’s IP shall mean (i) the Site-Directed Enzyme Enhancement Therapy (SEE-TX®) discovery platform for identification of non-competitive pharmacological chaperones, (ii) Licensor’s Molecules acting as pharmacological chaperones, (iii) Licensor’s Know-How and (iv) Licensor’s Trademark named SEE-Tx including all methods, data sets, technical documents pertaining to the screening technology including specification, diagrams, lab results, any and all notes, all inventor notebook pages specific to the program, all patents, pending, issued, or filed to the screening technology.

Field of Use

Site-Directed Enzyme Enhancement Therapy (“SEE-Tx”) is used to discover novel allosteric sites on misfolded proteins and identify proprietary small molecules that bind these sites and restore protein folding, potentially treating the underlying disease. These small molecule binding sites, away from the protein’s active areas, are called allosteric sites. Licensee believes targeting the allosteric binding site instead of the active binding site can provide a number of advantages superior regulation of misfolded proteins implicated in disease, enhanced specificity by being non-competitive with the natural substrate and the potential for molecules with favorable drug-like properties. While the SEE-Tx platform is novel and has not been used to develop approved drugs yet, these advantages have the potential to ultimately enhance both tolerability and response.

License Grant

Australian Institute, a paediatric medical research institute, grants an exclusive worldwide license to the Licensee to use the know-how for developing, manufacturing and commercializing the product for proposed treatment for Friedreich’s ataxia using pharmaceutical grade Resveratrol. The License Agreement is for both parties to work jointly to develop an appropriate delivery system and conduct clinical trials for the purpose of product approval in the treatment of Friedreich’s ataxia and worldwide commercialization by the Licensee.

License Property

Resveratrol product delivers a therapeutically effective dose of resveratrol in the blood stream without causing gastrointestinal (GI) side effects.  Resveratrol is delivered into the blood plasma with significantly greater bioavailability than conventional resveratrol and has the ability to cross the blood-brain barrier, where it has, in studies conducted in Friedreich’s ataxia (FA) and Alzheimer’s disease (AD) patients, positive effects on oxidative stress, inflammation and mitochondrial function.

Field of Use

Field of use is for the treatment of Freidreich’s ataxia.

Friedreich's ataxia is an inherited disorder that affects some of the body's nerves. It is caused by a gene defect that is inherited from both parents. Symptoms often begin in late childhood and can include trouble walking, fatigue, changes in sensation, and slowed speech. These tend to get worse over time.

License Grant

For the Exclusive License Outside the Territory, Licensor of Japan grants an exclusive, except as to Licensor, for limited purpose, license, with the right to sublicense, under the Licensor Patents that cover outside of the Territory and Licensor Know-How and Licensors rights under the Joint Inventions and the Joint Patents to research, develop, make, have made, use, import, market, offer for sale and sell the Licensed Molecules and Products in the Field outside the Territory.

For the Research and Manufacturing License in the Territory, Licensor grants a non-exclusive license, with the right to sublicense, under the Licensor Patents that cover the Territory and Licensor Know-How and Licensors rights under the Joint Inventions and Joint Patents, to research, develop and manufacture the Licensed Molecules and Products in the Territory solely for the purpose of advancing the research, development and commercialization of the Licensed Molecules and Products outside the Territory.

License Property

Licensed Molecules shall mean any and all compounds that arc claimed or covered by an Licensor Patent, including, but not limited to PCAP-872, PCAP-831, PCAP-924 and PCAP-130, and the salts, metabolites and pro-drugs of such compounds.

Product shall mean a pharmaceutical product containing at least one Licensed Molecule, including all dosage forms, formulations, line extensions and modes of administration thereof.

The related patents include
–  Amino substituted diary [a,d.] Cycloheptene analogs as muscarinic agonists and methods of treatment of Neuropsychiatric disorders,
–  Use of N-Desmethylclozapine and related compounds as dopamine stabilizing agents, and,
–  Prodrugs of muscarinic agonists and methods of treatment of Neuropsychiatric disorders.

Field of Use

This collaboration agreement is to develop and commercialize a novel class of pro-cognitive drugs to treat patients with schizophrenia and related disorders in Japan and several other Asian countries.