Notice of Pre-AIA or AIA Status
The present application, filed on or after March 16, 2013, is being examined under the first inventor to file provisions of the AIA .
Status of the Claims
Currently, claims 1-2, 4, 10-11, 13-15, 17, 19, 23, 26, 28-30, 32, 34, 36 and 38-39 are pending in the instant application.
Information Disclosure Statement
The information disclosure statement (IDS) submitted on 08/13/2024, 09/11/2024, 10/06/2025, and 12/19/2025 are in compliance with the provisions of 37 CFR 1.97. Accordingly, the information disclosure statement is being considered by the examiner.
Election/Restrictions
In response to the Restriction requirement submitted on 08/06/2025, Applicant has elected the following invention:
Group I, claims 1, 10-11, 13-15, 17, 19, 23, 26, 28-30, 32, 34, 36, and 38-39, drawn to a method of treating a neurological disorder in a human patient, comprising administering a PIKfyve inhibitor, wherein the patient does not express a mutant form of c9orf72 comprising an expanded GGGGCC hexanucleotide repeat.
In response to the Election requirement submitted on 08/06/2025, Applicant has elected the following species:
A single compound :
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A single neurological disorder: Amyotrophic Lateral Sclerosis
Claims 1, 10-11, 14, and 34 read on the elected species. Accordingly, claims 1, 10-11, 14, and 34 are pending examination, and all other pending claims are hereby withdrawn from consideration.
Claim Rejections - 35 USC § 112 – Second paragraph
The following is a quotation of 35 U.S.C. 112(b):
(b) CONCLUSION.—The specification shall conclude with one or more claims particularly pointing out and distinctly claiming the subject matter which the inventor or a joint inventor regards as the invention.
The following is a quotation of 35 U.S.C. 112 (pre-AIA ), second paragraph:
The specification shall conclude with one or more claims particularly pointing out and distinctly claiming the subject matter which the applicant regards as his invention.
Claim 14 is rejected under 35 U.S.C. 112(b) or 35 U.S.C. 112 (pre-AIA ), second paragraph, as being indefinite for failing to particularly point out and distinctly claim the subject matter which the inventor or a joint inventor (or for applications subject to pre-AIA 35 U.S.C. 112, the applicant), regards as the invention.
Claim 14 is rejected for reciting the phrase “binds to and/or inhibits PIKfyve enzymatic activity” because a person of ordinary skill in the art would note reasonably be able to understand the metes and bounds of the claim. From the recitation it is unclear what the recited small molecule would be binding to. This rejection may be overcome by a re-wording of the claim to clarify the binding target (i.e., “a small molecule that binds PIKfyve enzyme and/or inhibits PIKfyve enzymatic activity”).
Claim Rejections - 35 USC § 112 – First Paragraph
The following is a quotation of the first paragraph of 35 U.S.C. 112(a):
(a) IN GENERAL.—The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor or joint inventor of carrying out the invention.
The following is a quotation of the first paragraph of pre-AIA 35 U.S.C. 112:
The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor of carrying out his invention.
Claims 1, 10-11, 14, and 34 rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, as failing to comply with the written description requirement. The claim(s) contains subject matter which was not described in the specification in such a way as to reasonably convey to one skilled in the relevant art that the inventor or a joint inventor, or for applications subject to pre-AIA 35 U.S.C. 112, the inventor(s), at the time the application was filed, had possession of the claimed invention.
Claim 1 recites a method of treating “a neurological disorder” by administration of an “effective amount of a PIKfyve inhibitor”. Applicant’s disclosure is not sufficient to meet the written description requirement for the instant claim because the recitation would encompass any and all compounds having PIKfyve inhibiting activity for use in treating any and all neurological diseases in patients who do not express the recited c9orf72 mutant. This would include an innumerable amount of conditions and compounds not fully described by the specification.
Claim 10 further limits the method of claim 1 wherein the neurological disease is a neuromuscular disease. The instant claim does not mee the written description requirement for largely the same reasons as claim 1. The reason being that the instant claim would encompass any and all compounds having PIKfyve inhibition ability being used to treat any neuromuscular disorders. The provided disclosure does not meet the written description requirement for the innumerable amount of compounds and conditions encompassed by the claim.
Claim 11 further limits the method of claim 10 wherein the neuromuscular disorder is selected from the group consisting of the recited Markush group. The instant claim does not meet the written description requirement for much the same reasons as claims 1 and 10 in that the disclosure lacks sufficient support to claim that any and all compounds with PIKfyve inhibiting activity would be effective for the treatment of each of the recited neuromuscular disorders.
Claim 14 further limits the method of claim 1 wherein the PIKfyve inhibitor is a small molecule compound that binds to and /or inhibits PIKfyve enzymatic activity. The instant claim fails to meet the written description requirement because the instant claim would encompass the use of small molecule PIKfyve inhibitory compounds for effective treatment of any and all neurological disorders. As this would encompass a vast array of disorders with potentially differing etiologies not disclosed in the specification, the written description requirement cannot be considered to be met.
Claim 34 further limits the method of claim 14 wherein the PIKfyve inhibitor is a compound of formula (IX). The instant claim fails to meet the written description requirement for much the same reason as claim 14. More specifically, that Applicant’s specification does not provide sufficient support for the use of a compound of formula (IX) to treat any and all neurological disorders.
Claim Rejections - 35 USC § 102
The following is a quotation of the appropriate paragraphs of 35 U.S.C. 102 that form the basis for the rejections under this section made in this Office action:
A person shall be entitled to a patent unless –
(a)(1) the claimed invention was patented, described in a printed publication, or in public use, on sale, or otherwise available to the public before the effective filing date of the claimed invention.
(a)(2) the claimed invention was described in a patent issued under section 151, or in an application for patent published or deemed published under section 122(b), in which the patent or application, as the case may be, names another inventor and was effectively filed before the effective filing date of the claimed invention.
Claim(s) 1 and 10-11 is/are rejected under 35 U.S.C. 102(a)(2) as being anticipated by Rosen (US 20230038929 A1).
Claim 1 recites a method of treating a neurological disorder in a human patient, the method 1. comprising administering to the patient a therapeutically effective amount of a PIKfyve inhibitor, wherein the patient does not express a mutant form of c9orf72 comprising an expanded GGGGCC hexanucleotide repeat.
Rosen teaches a method of treating neurological disorders by administration of compounds as PIKfyve inhibitors. Among the neurological disorders disclosed by Rosen is Amyotrophic Lateral Sclerosis (ALS) (specification [0140])1. Rosen further carries out an IPSC MN survival assay on their compounds, wherein the IPSC cells are derived from human motor cells having the SOD1 genetic variant of ALS. The results of said assay are provided in the table below (specification [0223], Table 7):
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As can be seen in the table above, at least compound 9 of Rosen was effective in increasing the survival rate of cells derived from individuals having the C9 (c9orf72) genetic variant and the SOD1 genetic variant, respectively. The increased survival rate of each cell line indicates that at least compound 9 would be effective to treat either variant of ALS. Essentially, Rosen teaches the treatment of ALS in a subject not having a mutant form of c9orf72 comprising an expanded GGGGCC hexanucleotide repeat, by administration of a PIKfyve inhibitor.
Claim 10 further limits the method of claim 1 wherein the neurological disorder is a neuromuscular disorder.
Rosen teaches the use of PIKfyve inhibitors to treat neurological disorders, including Amyotrophic Lateral Sclerosis (ALS), which is also classed as a neuromuscular disease.
Claim 11 further limits the method of claim 10 wherein the neuromuscular disease is selected from amyotrophic lateral sclerosis, congenital myasthenic syndrome, congenital myopathy, cramp fasciculation syndrome, Duchenne muscular dystrophy, glycogen storage disease type II, hereditary spastic paraplegia, inclusion body myositis, Isaac's Syndrome, Kearns-Sayre syndrome, Lambert-Eaton myasthenic syndrome, mitochondrial myopathy, muscular dystrophy, myasthenia gravis, myotonic dystrophy, peripheral neuropathy, spinal and bulbar muscular atrophy, spinal muscular atrophy, Stiff person syndrome, Troyer syndrome, and Guillain-Barré syndrome.
As discussed above in the claim 10 rejection, Rosen teaches the treatment of ALS by administering PIKfyve inhibitors.
Claim(s) 1, 14, and 34 is/are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Bhagwat (WO 2017/040971 A1).
Claim 1 recites a method of treating a neurological disorder in a human patient, the method 1. comprising administering to the patient a therapeutically effective amount of a PIKfyve inhibitor, wherein the patient does not express a mutant form of c9orf72 comprising an expanded GGGGCC hexanucleotide repeat.
Bhagwat teaches the treatment of neurodegenerative disorders by administration of an effective amount of a PIKfyve inhibitor (page 1, lines 9-14)2. Bhagwat makes no mention of any subjects being treated as having or expressing the recited mutant form of c9orf72.
Claim 14 further limits the method of claim 1 wherein the PIKfyve inhibitor is a small molecule that binds to and/or inhibits PIKfyve enzymatic activity.
As discussed above in the claim 1 rejection, Bhagwat teaches the use of PIKfyve inhibitor compounds for the treatment of neurological disorders. Of the compounds taught by Bhagwat the following exemplary compound D-1 is provided (page 19, Table 4):
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The above compound is identical to the compound elected by Applicant, and under broadest reasonable interpretation is considered as a small molecule compound.
Claim 34 further limits the method of claim 14 wherein the PIKfyve inhibitor is a compound represented by formula (IX):
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As discussed previously, Bhagwat teaches the following compound D-1:
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Compound D-1 anticipates a compound of formula (IX) wherein:
Ar is pyridyl
R1 is alkyl
R2 is hydrogen
Conclusion
Claims 1, 10-11, 14, and 34 are rejected.
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If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Jeffrey S Lundgren can be reached at (571) 272-5541. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
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/ERIC TRAN/Examiner, Art Unit 1629
/JEFFREY S LUNDGREN/Supervisory Patent Examiner, Art Unit 1629
1 “In some embodiments, the neurological disease is ALS, FTD, Alzheimer's disease, Parkinson's disease, Huntington's disease, or CMT. In some embodiments, the neurological disease is ALS.”
2 “This disclosure features methods for treating lysosomal storage disorders (e.g. Gangliosidosis, MPS, and other deficiencies in lysosomal enzymes) and/or neurodegenerative diseases (e.g., Alzheimer’s disease), which include administering to a subject (e.g., a human patient) in need of such treatment (e.g., identified as being in need of such treatment) an effective amount of one or more PIKfyve inhibitors, or a pharmaceutically acceptable salt thereof.”