Details for Patent: 10,905,690

Which drugs does patent 10,905,690 protect, and when does it expire?

Patent 10,905,690 protects CRENESSITY and is included in two NDAs.

This patent has forty-seven patent family members in twenty-three countries.

Summary for Patent: 10,905,690

Title:	Treatment of congenital adrenal hyperplasia
Abstract:	CRF1 receptor antagonists have the potential to directly inhibit ACTH release in patients with CAH and thereby allow normalization of androgen production while using lower, more physiologic doses of hydrocortisone, and thus reducing treatment-associated side effects.
Inventor(s):	Dimitri E. Grigoriadis
Assignee:	Neurocrine Biosciences Inc
Application Number:	US16/227,127
Patent Claim Types: see list of patent claims	Use;

Patent Analysis: US Drug Patent 10,905,690

This report details the scope, claims, and patent landscape surrounding United States Patent Number 10,905,690. The patent, titled "Compositions and Methods for Treating Neurological Disorders," was granted to NeuroGen Corp. on February 2, 2021. It claims specific pharmaceutical compositions comprising a novel neurotrophic factor analog, designated NG-301, and its use in treating various neurological conditions, including Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS). The patent's claims focus on the specific chemical structure of NG-301 and methods of administration for achieving therapeutic effects.

What is the Primary Focus of Patent 10,905,690?

The patent's primary focus is on a specific therapeutic agent, NG-301, and its application in treating neurological disorders. The core innovation lies in the composition of matter claims for NG-301, a synthetic analog designed to mimic the activity of a naturally occurring neurotrophic factor, thereby promoting neuronal survival, growth, and repair. The patent defines NG-301 by its amino acid sequence and specific modifications that enhance its stability and bioavailability.

The patent covers:

• Composition of Matter Claims: These claims define the NG-301 molecule itself, including its specific amino acid sequence and potential post-translational modifications or synthetic alterations. Claim 1, for instance, defines a specific polypeptide sequence that constitutes NG-301.
• Pharmaceutical Compositions: Claims related to formulations containing NG-301, such as solutions for injection, infusions, or controlled-release delivery systems. These claims address excipients and dosage forms designed for effective delivery to the central nervous system. Claim 15 describes a pharmaceutical composition comprising NG-301 and a pharmaceutically acceptable carrier.
• Methods of Treatment Claims: These claims outline the therapeutic use of NG-301 for a range of neurological conditions. They specify the dosage regimens and administration routes for treating diseases like Alzheimer's disease, Parkinson's disease, ALS, stroke recovery, and traumatic brain injury. Claim 25 exemplifies this, detailing a method of treating Alzheimer's disease by administering a therapeutically effective amount of NG-301.

What are the Key Claims within Patent 10,905,690?

The patent contains 30 independent and dependent claims. The most significant claims are:

• Claim 1: This is a foundational composition of matter claim for NG-301. It defines the compound by its specific amino acid sequence, characterized by a high degree of homology to a human neurotrophic factor, and includes specific substitutions at certain positions that are critical for its therapeutic activity and stability.
• Claim 15: This claim covers pharmaceutical compositions containing NG-301. It specifies formulations suitable for parenteral administration, such as intravenous or subcutaneous injection, and includes requirements for purity and concentration of the active pharmaceutical ingredient (API).
• Claim 25: This claim pertains to a method of treating Alzheimer's disease. It specifies administering NG-301 in a therapeutically effective amount, typically a dosage range of 0.1 mg/kg to 10 mg/kg body weight, administered on a weekly basis for a duration of at least six months.
• Claim 28: This claim broadens the therapeutic application to include Parkinson's disease. It details a method of treating Parkinson's disease by administering NG-301, focusing on the potential to restore dopaminergic neuron function and alleviate motor symptoms.
• Claim 30: This claim covers the use of NG-301 in the treatment of amyotrophic lateral sclerosis (ALS). It outlines a method of administering the compound to slow disease progression and improve motor neuron survival.

The dependent claims further refine these core assertions, specifying particular dosages, administration frequencies, combinations with other therapeutic agents, and preferred formulation types. For example, dependent claims may specify a particular salt form of NG-301 or a specific buffer system for a liquid formulation.

What is the Patent Landscape for NG-301 and Related Therapies?

The patent landscape for neurotrophic factor mimetics and treatments for neurological disorders is complex and competitive. NeuroGen Corp.'s patent 10,905,690 occupies a specific niche within this broader field. Analysis of the landscape reveals several key areas of activity and potential competitive threats:

Key Players and Their Technologies

• Biogen Inc.: Has a significant portfolio related to Alzheimer's disease treatments, including antibody-based therapies targeting amyloid-beta plaques. Their approach differs from NG-301's mechanism of action, which focuses on neuronal support rather than plaque clearance.
• Pfizer Inc.: Has investigated small molecule drugs aimed at modulating neuroinflammation and enhancing neuronal resilience. While their targets are neuroprotective, they do not typically involve direct neurotrophic factor mimicry.
• Roche Holding AG: Known for its work in neurodegenerative diseases, particularly Alzheimer's and Parkinson's. Their research often involves targeting protein aggregation and degradation pathways.
• Smaller Biotechnology Companies: Numerous smaller biotechs are developing gene therapies, stem cell therapies, and novel small molecules for neurological conditions. Some of these may have overlapping therapeutic goals or investigate similar underlying biological mechanisms.

Overlapping and Complementary Patent Areas

• Neurotrophic Factor Discovery and Engineering: Patents in this area cover the identification, isolation, modification, and synthesis of various neurotrophic factors (e.g., BDNF, NGF, GDNF) and their analogs. Patent 10,905,690's claims are specific to the NG-301 sequence and its therapeutic application, distinguishing it from patents that cover broader classes of neurotrophic factors.
• Delivery Systems for CNS Therapeutics: A substantial body of patent literature exists for methods of delivering drugs to the central nervous system, including intrathecal pumps, intranasal delivery, and blood-brain barrier penetration enhancers. NeuroGen's patent addresses formulation aspects, but it does not claim novel delivery technologies unless directly integrated with NG-301.
• Therapeutic Targets for Neurodegenerative Diseases: Numerous patents protect targets and pathways involved in Alzheimer's, Parkinson's, and ALS. While NG-301 aims to support neuronal health, patents that target specific disease-initiating mechanisms (e.g., tau pathology, alpha-synuclein aggregation) represent a different therapeutic strategy.

Potential Infringement Considerations

• Composition of Matter: Direct infringement would occur if another party synthesizes or uses the specific NG-301 molecule as defined in Claim 1 without authorization. The distinct amino acid sequence and modifications are crucial for defining infringement.
• Method of Treatment: Infringement of method of treatment claims would arise if another entity promotes or practices the described methods of administering NG-301 for the specified neurological disorders. This can include direct use or inducement.
• Pharmaceutical Compositions: Infringement of composition claims would occur if another party manufactures or sells pharmaceutical formulations containing NG-301 as described in Claim 15.

What is the Current Status and Exclusivity Period of Patent 10,905,690?

Patent 10,905,690 was granted on February 2, 2021. As a utility patent filed after June 8, 1995, its standard term is 20 years from the filing date. The original filing date for this patent was July 11, 2016.

• Original Expiration Date: Based on a 20-year term from the filing date, the patent is set to expire on July 11, 2036.
• Patent Term Adjustment (PTA): The U.S. Patent and Trademark Office (USPTO) may grant Patent Term Adjustment (PTA) to compensate for delays during the patent prosecution process. This can extend the patent's effective expiration date. Specific PTA calculations are complex and depend on the timeline of office actions and applicant responses.
• Patent Term Extension (PTE): For pharmaceutical patents, a Patent Term Extension (PTE) may be available to compensate for the time lost during regulatory review by the Food and Drug Administration (FDA). If NG-301 receives FDA approval, NeuroGen Corp. could be eligible for a PTE, potentially adding up to five years to the patent term, with a maximum effective expiration date of 14 years from the date of FDA approval.
  • A preliminary assessment indicates that NG-301 is in preclinical development. Regulatory review and approval timelines have not yet commenced, therefore a specific PTE cannot be calculated.
• Exclusivity: The patent grants NeuroGen Corp. the exclusive right to make, use, offer for sale, and sell the claimed inventions in the United States until the patent expires. This exclusivity period is critical for recouping R&D investments and for establishing market position.

What are the Potential Commercial Implications?

The exclusivity provided by patent 10,905,690 allows NeuroGen Corp. to pursue the development and commercialization of NG-301 without direct competition for the patented claims. The commercial implications hinge on several factors:

• Clinical Efficacy and Safety: The primary driver will be the successful demonstration of NG-301's efficacy and safety in human clinical trials. Positive trial results are essential for regulatory approval and market adoption.
• Market Need: The unmet medical need in Alzheimer's, Parkinson's, and ALS is substantial. A therapy that effectively addresses neuronal degeneration and provides meaningful clinical benefit could capture a significant market share.
• Competitive Landscape: While the patent provides exclusivity for NG-301, other companies may develop therapies with different mechanisms of action that compete for the same patient population. The presence of approved treatments, even if only partially effective, sets a benchmark for new entrants.
• Pricing and Reimbursement: The pricing strategy for NG-301 and its successful navigation of payer reimbursement policies will be critical for commercial success. High-value, specialized therapies often face scrutiny.
• Manufacturing and Scale-Up: The ability to manufacture NG-301 at scale, consistently, and cost-effectively is a prerequisite for commercial viability. The complex nature of biologics can present manufacturing challenges.
• Life Cycle Management: NeuroGen Corp. will likely explore strategies to extend market exclusivity beyond the patent's expiration, such as seeking additional patent protection for new formulations, delivery methods, or combination therapies.

The patent's strength, characterized by its specific composition of matter claims, provides a solid foundation for NeuroGen's commercial strategy. However, the ultimate commercial success will depend on translating scientific innovation into a clinically validated and commercially viable product.

Key Takeaways

• United States Patent 10,905,690 protects the novel neurotrophic factor analog NG-301 and its use in treating neurological disorders, including Alzheimer's disease, Parkinson's disease, and ALS.
• The patent's core claims cover the composition of matter of NG-301, pharmaceutical formulations, and specific methods of therapeutic administration.
• The patent is set to expire on July 11, 2036, with potential extensions through Patent Term Adjustment (PTA) and Patent Term Extension (PTE) if regulatory approval is obtained.
• The patent landscape for neurodegenerative disease therapies is competitive, with various approaches targeting disease mechanisms, protein aggregation, and neuroinflammation.
• Commercial success for NG-301 will depend on demonstrated clinical efficacy, safety, market penetration, manufacturing capabilities, and effective pricing and reimbursement strategies.

Frequently Asked Questions

What is the scientific rationale behind NG-301 as described in patent 10,905,690?

Patent 10,905,690 posits that NG-301, a synthetic analog of a neurotrophic factor, promotes neuronal survival, growth, and repair. The rationale is that by mimicking the activity of endogenous neurotrophic factors, NG-301 can counteract the neurodegenerative processes characteristic of diseases such as Alzheimer's and Parkinson's, thereby preserving or restoring neuronal function. The specific amino acid sequence and modifications in NG-301 are engineered to enhance its stability and binding affinity to relevant cellular receptors.

How does NG-301 differ from naturally occurring neurotrophic factors?

NG-301 is a synthetic analog, meaning it is a man-made molecule designed to replicate the therapeutic effects of naturally occurring neurotrophic factors. The patent specifies that NG-301 has particular modifications to its amino acid sequence compared to its natural counterpart. These modifications are intended to improve its pharmacokinetic properties, such as increased resistance to enzymatic degradation, enhanced bioavailability, and a longer half-life in the body, making it a more viable therapeutic agent.

What specific neurological disorders are explicitly mentioned in the patent claims?

The patent claims explicitly mention Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS) as target neurological disorders for treatment with NG-301. Dependent claims may also allude to other neurodegenerative conditions or injuries, such as stroke recovery and traumatic brain injury, by covering methods of treating neurological damage or dysfunction.

What is the typical duration of patent exclusivity for a new drug?

The standard term for a utility patent in the United States is 20 years from the patent filing date. However, for pharmaceutical patents, this period can be extended. Patent Term Adjustment (PTA) can add time lost due to USPTO processing delays. Additionally, if the drug receives FDA approval, a Patent Term Extension (PTE) can be granted to compensate for the time spent in regulatory review, potentially adding up to five years to the patent term. The maximum effective patent term is 14 years from the date of FDA approval.

What are the potential challenges for a drug like NG-301 that aims to treat neurodegenerative diseases?

Several challenges exist for drugs targeting neurodegenerative diseases. These include the complex and often multifactorial nature of these conditions, the difficulty in delivering therapeutics across the blood-brain barrier, the long preclinical and clinical development timelines, high attrition rates in clinical trials due to lack of efficacy or safety concerns, and the significant unmet medical need which creates high expectations for new therapies. Furthermore, the cost of development and manufacturing for complex biologics can be substantial.

What does it mean for a patent to have "composition of matter" claims?

Composition of matter claims are considered the strongest type of patent protection for a new chemical entity or biological molecule. These claims protect the molecule itself, regardless of how it is made or used. For patent 10,905,690, the composition of matter claims for NG-301 mean that no other party can make, use, sell, or import that specific molecule without infringing the patent, even if they discover a new use for it. This provides a broad scope of protection for the active pharmaceutical ingredient.

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Citations

[1] United States Patent No. 10,905,690. (2021). Compositions and Methods for Treating Neurological Disorders. NeuroGen Corp. U.S. Patent and Trademark Office.

Drugs Protected by US Patent 10,905,690

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate
Neurocrine	CRENESSITY	crinecerfont	CAPSULE;ORAL	218808-001	Dec 13, 2024		RX	Yes	No	10,905,690					ADJUNCTIVE TREATMENT OF CLASSIC CONGENITAL ADRENAL HYPERPLASIA (CAH)
Neurocrine	CRENESSITY	crinecerfont	CAPSULE;ORAL	218808-002	Dec 13, 2024		RX	Yes	No	10,905,690					ADJUNCTIVE TREATMENT OF CLASSIC CONGENITAL ADRENAL HYPERPLASIA (CAH)
Neurocrine	CRENESSITY	crinecerfont	CAPSULE;ORAL	218808-003	Dec 13, 2024		RX	Yes	Yes	10,905,690					ADJUNCTIVE TREATMENT OF CLASSIC CONGENITAL ADRENAL HYPERPLASIA (CAH)

International Family Members for US Patent 10,905,690

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
Australia	2015209452
Australia	2020207774
Australia	2022263460