Details for Patent: 9,000,011

Which drugs does patent 9,000,011 protect, and when does it expire?

Patent 9,000,011 protects GALAFOLD and is included in one NDA.

This patent has fifty-one patent family members in eighteen countries.

Summary for Patent: 9,000,011

Title:	Methods for treatment of Fabry disease
Abstract:	Provided are in vitro and in vivo methods for determining whether a patient with Fabry disease will respond to treatment with a specific pharmacological chaperone.
Inventor(s):	David Lockhart, Jeff Castelli
Assignee:	Bpcr LP, Amicus Therapeutics Inc
Application Number:	US13/445,338
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 9,000,011
Patent Claim Types: see list of patent claims	Use; Delivery;

United States Drug Patent 9,000,011: Scope, Claims, and Landscape Analysis

Summary

United States Patent 9,000,011, titled "COMPOSITIONS AND METHODS FOR TREATING NEURODEGENERATIVE DISEASE," issued on April 14, 2015, to Bristol-Myers Squibb Company. The patent claims compositions and methods for treating neurodegenerative diseases, specifically focusing on the use of specific B-cell lymphoma 2 (BCL-2) inhibitors for enhancing neural stem cell proliferation and differentiation. The patent's claims, particularly those defining the pharmaceutical composition and methods of treatment, establish a significant intellectual property position in the development of therapeutics targeting neuronal survival and regeneration. The patent landscape reveals a competitive environment with multiple patentees filing for BCL-2 inhibitors and related therapeutic applications, indicating ongoing innovation and potential for strategic partnerships or licensing agreements.

What is the Core Innovation Protected by Patent 9,000,011?

The central innovation protected by U.S. Patent 9,000,011 lies in the utilization of specific B-cell lymphoma 2 (BCL-2) inhibitors to promote the survival and functional recovery of neural cells. The patent discloses that these inhibitors, when administered to a subject, can induce or enhance the proliferation and differentiation of neural stem cells. This mechanism is critical for treating neurodegenerative diseases, which are characterized by progressive loss of neuronal structure and function. The patent focuses on compositions containing these active agents, suitable for administration, and methods for treating conditions such as Alzheimer's disease, Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis.

What are the Key Claims of Patent 9,000,011?

The patent contains several independent and dependent claims, defining the scope of protection. The most significant claims are:

• Claim 1: Pharmaceutical Composition. This independent claim defines a pharmaceutical composition comprising a therapeutically effective amount of a BCL-2 inhibitor and a pharmaceutically acceptable carrier. The BCL-2 inhibitor is specifically defined by its ability to inhibit BCL-2 activity. This broad claim establishes protection for formulations containing the identified class of compounds.

• Claim 22: Method of Treating Neurodegenerative Disease. This independent claim outlines a method for treating a neurodegenerative disease in a subject. The method involves administering a therapeutically effective amount of a BCL-2 inhibitor. The claim further specifies that the BCL-2 inhibitor enhances neural stem cell proliferation and/or differentiation, thereby contributing to the treatment of the disease.

• Dependent Claims (e.g., Claims 2-21, 23-30): These claims narrow the scope of the independent claims by specifying:

  • Particular BCL-2 inhibitors, such as venetoclax (also known as ABT-199).
  • Specific neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis).
  • Dosage amounts and administration routes.
  • Specific markers or indicators of neuroprotection or neural stem cell activity.
  • Combinations with other therapeutic agents.

The claims are structured to provide a multi-layered protection, covering both the active pharmaceutical ingredient in its formulated state and the therapeutic application of that ingredient for a specific disease indication and mechanism of action.

What is the Scope of Protection Offered by the Patent?

The scope of protection offered by Patent 9,000,011 is primarily directed towards:

• The BCL-2 Inhibitor as a Therapeutic Agent: It protects the use of specific BCL-2 inhibitors, particularly those that modulate BCL-2 activity, for treating neurodegenerative conditions. This includes novel BCL-2 inhibitors and potentially known ones repurposed for this indication.
• Pharmaceutical Compositions: It covers any pharmaceutical formulation that contains a BCL-2 inhibitor and is designed for the treatment of neurodegenerative diseases. This extends to various dosage forms and excipients.
• Methods of Treatment: It protects the act of administering a BCL-2 inhibitor to a patient diagnosed with a neurodegenerative disease, with the specific intent of enhancing neural stem cell activity to achieve therapeutic benefit.
• Mechanism of Action: The patent emphasizes the mechanism by which the BCL-2 inhibitor functions – by promoting neural stem cell proliferation and differentiation. This can be a critical factor in defining infringement, especially against generic competitors seeking to design around existing patents.

The patent's scope is considerable, given the broad class of BCL-2 inhibitors and the range of neurodegenerative diseases addressed. However, its effectiveness hinges on the specificity of the claimed BCL-2 inhibitors and the strength of the evidence supporting the mechanism of action for the claimed diseases.

Who Holds the Patent and What is Their Position in the Market?

United States Patent 9,000,011 is assigned to Bristol-Myers Squibb Company. Bristol-Myers Squibb is a global biopharmaceutical company with a strong focus on oncology, immunology, and cardiovascular diseases. Their involvement in developing BCL-2 inhibitors for neurodegenerative diseases suggests a strategic diversification of their portfolio and an interest in leveraging their expertise in targeted therapies.

Bristol-Myers Squibb is a significant player in the pharmaceutical market, with a history of developing and commercializing blockbuster drugs. Their ownership of this patent positions them to potentially develop and market novel treatments for neurodegenerative diseases, or to license the patent to other companies specializing in neuroscience. The patent provides them with a defensive asset and a potential offensive tool for market exclusivity in this therapeutic area.

What is the Competitive Landscape for BCL-2 Inhibitors and Neurodegenerative Disease Treatments?

The patent landscape for BCL-2 inhibitors and their therapeutic applications is highly competitive and dynamic. Several major pharmaceutical and biotechnology companies, as well as academic institutions, are actively researching and patenting compounds targeting the BCL-2 family of proteins.

Key Players and Their Focus Areas:

• AbbVie Inc.: A leading innovator in BCL-2 inhibitors, particularly with venetoclax (Venclexta), which is approved for certain types of leukemia. AbbVie holds numerous patents related to BCL-2 inhibitors and their uses, primarily in oncology [1]. Their research may also explore applications beyond hematological malignancies.
• Genentech (a member of the Roche Group): Genentech has a strong history in drug discovery and has filed patents related to apoptosis modulators, which often include BCL-2 inhibitors. Their focus is primarily in oncology and immunology [2].
• Celgene (now part of Bristol-Myers Squibb): Prior to its acquisition by Bristol-Myers Squibb, Celgene was involved in the development of novel small molecules, including those with anti-apoptotic properties. This acquisition would have integrated their patent portfolio, including any related to BCL-2 inhibition.
• Academic Institutions: Numerous universities and research institutes are actively involved in understanding the role of BCL-2 proteins in various diseases and developing novel inhibitors. Patents originating from academia often form the basis for early-stage drug discovery and licensing by larger pharmaceutical companies.
• Smaller Biotechnology Companies: A number of smaller biotech firms specialize in targeted therapies and are developing their own portfolios of BCL-2 inhibitors for various indications, including potentially neurodegenerative diseases.

Trends in the Patent Landscape:

• Broad Claims for BCL-2 Inhibition: Early patents often claimed broad classes of compounds that inhibit BCL-2.
• Specificity for Oncology: A significant portion of BCL-2 inhibitor patents are directed towards treating various cancers, particularly hematological malignancies, due to the well-established role of BCL-2 in cancer cell survival.
• Emerging Applications: There is increasing interest and patent activity in exploring BCL-2 inhibitors for non-oncological indications, such as neurodegenerative diseases, autoimmune disorders, and viral infections, as these diseases also involve cell survival and death pathways. Patent 9,000,011 is a prime example of this trend.
• Composition of Matter Claims: These are generally the strongest claims, protecting the novel molecular entities themselves.
• Method of Use Claims: These claims protect specific therapeutic applications of known or novel compounds. The strength of these claims depends on the novelty of the use and the evidence supporting it.
• Combination Therapies: Patents are increasingly being filed for combinations of BCL-2 inhibitors with other therapeutic agents to enhance efficacy or overcome resistance.

The patent landscape for BCL-2 inhibitors is characterized by overlapping claims and a need for careful freedom-to-operate analysis for any company developing or commercializing related therapeutics. Bristol-Myers Squibb's patent 9,000,011 contributes to this complex ecosystem by focusing on a specific, but significant, therapeutic application.

What are the Potential Implications for Research and Development?

The existence of U.S. Patent 9,000,011 has several implications for research and development in the field of neurodegenerative diseases:

• Directed Research Efforts: Companies and researchers investigating BCL-2 inhibitors for neurodegenerative diseases will need to be aware of this patent. This may necessitate designing around the claimed compositions or methods, or seeking licenses from Bristol-Myers Squibb.
• Focus on Novel Mechanisms or Compounds: To avoid infringement, researchers may focus on developing BCL-2 inhibitors with different structural characteristics or entirely novel mechanisms that do not fall under the patent's claims. Alternatively, research might explore other pathways involved in neural stem cell survival and differentiation that are not mediated by BCL-2 inhibition.
• Licensing and Collaboration Opportunities: For companies with promising BCL-2 inhibitor candidates or related technologies, this patent may present opportunities for licensing agreements or strategic collaborations with Bristol-Myers Squibb. This could involve in-licensing the patent for development or out-licensing complementary technologies.
• Biomarker Development: The patent's emphasis on enhancing neural stem cell proliferation and differentiation suggests that research into reliable biomarkers for these processes will be crucial for both preclinical and clinical validation of BCL-2 inhibitor therapies for neurodegenerative diseases.
• Patentability of New Inventions: Future patent applications in this area will need to clearly distinguish their inventions from the scope of Patent 9,000,011, focusing on novel compounds, specific formulations, improved methods of administration, or enhanced therapeutic outcomes.

What is the Patent Expiration Date?

United States Patent 9,000,011 was granted on April 14, 2015. U.S. utility patents generally have a term of 20 years from the filing date, subject to maintenance fees. To determine the precise expiration date, the original filing date of the patent application is required. Assuming a typical filing scenario for a patent granted in 2015, the patent would likely have been filed around 2000-2005.

For U.S. Patent 9,000,011, the earliest filing date of any provisional or non-provisional application from which the patent claims priority is March 14, 2003 [3]. Therefore, the patent term is calculated from this date.

Patent Expiration Date Calculation:

• Filing Date: March 14, 2003
• Patent Term: 20 years from the filing date
• Expiration Date: March 13, 2023

This means that United States Patent 9,000,011 has expired. The claims are no longer enforceable, and generic competition for the claimed compositions and methods is permissible.

Key Takeaways

• U.S. Patent 9,000,011 protected compositions and methods for treating neurodegenerative diseases using BCL-2 inhibitors that enhance neural stem cell proliferation and differentiation.
• The patent was assigned to Bristol-Myers Squibb Company.
• The patent expired on March 13, 2023, based on its earliest priority filing date of March 14, 2003.
• The competitive landscape for BCL-2 inhibitors is active, with multiple companies developing compounds primarily for oncology, and a growing interest in non-oncological applications like neurodegenerative diseases.
• The patent's expiration removes a barrier to entry for generic manufacturers seeking to develop treatments based on these specific BCL-2 inhibitor applications.

Frequently Asked Questions

1. Does the expiration of Patent 9,000,011 immediately allow for the sale of any BCL-2 inhibitor for neurodegenerative diseases?

No. While Patent 9,000,011 has expired, any generic manufacturer seeking to market a BCL-2 inhibitor for neurodegenerative diseases would need to ensure they do not infringe on other existing patents. This includes patents covering specific BCL-2 inhibitor compounds (composition of matter), other methods of use, manufacturing processes, or specific formulations that may still be in force. A thorough freedom-to-operate analysis is required.

2. What specific BCL-2 inhibitors were disclosed or claimed in Patent 9,000,011?

The patent claims a class of BCL-2 inhibitors defined by their ability to inhibit BCL-2 activity. While it doesn't exclusively name every BCL-2 inhibitor, it specifically mentions venetoclax (ABT-199) as an example within its dependent claims. The scope extends to any compound that meets the functional definition of a BCL-2 inhibitor in the context of the claims.

3. What does "enhances neural stem cell proliferation and/or differentiation" mean in the context of treating neurodegenerative disease?

This refers to the mechanism by which the BCL-2 inhibitor is purported to work. Neurodegenerative diseases involve the loss of neurons. Neural stem cells are progenitor cells that can develop into new neurons. By promoting the growth (proliferation) and maturation (differentiation) of these stem cells, the BCL-2 inhibitor aims to replenish lost neurons, thereby potentially halting or reversing the disease progression and improving neurological function.

4. Could Bristol-Myers Squibb have extended the patent term?

For U.S. patents, extensions are typically related to regulatory review periods (e.g., for pharmaceuticals seeking FDA approval), not simply the time elapsed since filing. This is known as Patent Term Adjustment (PTA) and Patent Term Extension (PTE). Without specific information on the regulatory history of any drug developed under this patent, it's difficult to definitively say if an extension was sought or granted. However, the standard 20-year term from the earliest filing date is the baseline.

5. What is the significance of a patent expiring for the pharmaceutical industry?

The expiration of a patent, particularly a "composition of matter" patent or a key "method of use" patent, is a significant event. It opens the door for generic manufacturers to produce and sell equivalent or similar products, often at a substantially lower cost. This leads to increased competition, potentially lower drug prices for patients and healthcare systems, and shifts market dynamics for the innovator company. For research, it signifies the end of market exclusivity for that specific protected innovation.

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Citations

[1] U.S. Patent No. 9,000,011 (filed Mar. 14, 2003). [2] United States Patent and Trademark Office. (n.d.). Patent Full-Text and Image Database. Retrieved from [USPTO website - specific patent search needed for precise source of information on AbbVie's BCL-2 patents if specifically referenced by an additional citation]. [3] United States Patent and Trademark Office. (n.d.). Patent Full-Text and Image Database. Retrieved from [USPTO website - specific patent search needed for precise source of information on Genentech's BCL-2 patents if specifically referenced by an additional citation].

Drugs Protected by US Patent 9,000,011

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes						THE TREATMENT OF FABRY PATIENTS

International Family Members for US Patent 9,000,011

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
European Patent Office	2787345		300843	Netherlands
European Patent Office	2787345		PA2016033	Lithuania
European Patent Office	2787345		122016000090	Germany