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 .
Non-Final Rejection
The Status of Claims:
Claims 1-16 are pending.
Claims 1-16 are rejected.
Claims 1-5 and 15-16 are objected.
DETAILED ACTION
1. Claims 1-16 are under consideration in this Office Action.
Priority
2. It is noted that this application is a 371 of PCT/IB2022/055639 06/17/2022 ,which has priority date of 63212465 06/18/2021.
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Drawings
3. The drawings filed on 12/12/2023 are accepted by the examiner.
IDS
4. The IDS filed on 12/12/23, 4/11/25, 11/5/25, 2/2/26 are reviewed by the
examiner.
Claim Objections
Claims 1-5 and 15-16 are objected to because of the following informalities:
In the claims,1-4, and 15-16 the parentheses in the phrase “(e.g., iptacopan hydrochloride)”, “(wherein the dosing amount refers to the anhydrous free base of iptacopan hydrochloride)”, or “(e.g., iptacopan hydrochloride, e.g., iptacopan hydrochloride monohydrate, e.g., iptacopan hydrochloride monohydrate Form HB)” are recited. The use of the parentheses can be improper. The examiner recommends to remove the parentheses in the phrases from the claims.
In claims 3 and 5, the phrases “once daily (q.d.)” and/or “twice daily (b.i.d.)” are recited. These expressions can be redundant. The examiner recommends to remove “(q.d.)” and/or “(b.i.d.)” from the claims.
Claim Rejections - 35 USC § 101
35 U.S.C. 101 reads as follows:
Whoever invents or discovers any new and useful process, machine, manufacture, or composition of matter, or any new and useful improvement thereof, may obtain a patent therefor, subject to the conditions and requirements of this title.
Claim 16 is rejected under 35 U.S.C. 101 because the claimed recitation of a use, without setting forth any steps involved in the process, results in an
improper definition of a process, i.e., results in a claim which is not a proper
process claim under 35 U.S.C. 101. See for example Ex parte Dunki, 153 USPQ
678 (Bd.App. 1967) and Clinical Products, Ltd. v. Brenner, 255 F. Supp. 131, 149
USPO 475 (D.D.C. 1966).
Claim Rejections - 35 USC § 112
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.
Claims 1-16 are 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 pre-AIA the applicant regards as the invention.
Regarding claims 1-9, 11, 14-15-16, the abbreviated expression “ e.g. “ or the phrase "for example" renders the claim indefinite because it is unclear whether the limitation(s) following the phrase are part of the claimed invention. See MPEP § 2173.05(d).
A broad range or limitation together with a narrow range or limitation that falls within the broad range or limitation (in the same claim) may be considered indefinite if the resulting claim does not clearly set forth the metes and bounds of the patent protection desired. See MPEP § 2173.05(c). In the present instance, claim 2 recites the broad recitation” about 50 mg to about 200 mg” , and the claim also recites “about 50 mg, about 100 mg, or about 200 mg” which is the narrower statement of the range/limitation. The claim(s) are considered indefinite because there is a question or doubt as to whether the feature introduced by such narrower language is (a) merely exemplary of the remainder of the claim, and therefore not required, or (b) a required feature of the claims. The examiner recommends to put the narrowed limitation to its dependent claims.
In claim 9, the limitations “ as defined by Kaufman et al. (Kaufman RM, Djulbegovic B, Gernsheimer T, et al (2015) Platelet transfusion: a clinical practice guideline from the AABB. Ann Intern Med; 162(3):205-13)” are recited. These limitations can be improper and inappropriate because the claim cannot refer back to those limitations in the specification; The examiner recommends to remove them from the claim. .
In claims 10 and 13, the term" about ” of the phrase” at least about “ is recited. This expression is vague and indefinite because of the following reason:
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The examiner recommends to remove the term" about”. from the claims. Appropriate correction is required.
In claim 15, the phrase " for use in the treatment of an autoimmune benign hematological disorder..." is recited. This claim can be confusing and considered as a hybrid claim, which contains a compound and method claim together. The examiner recommends to separate the claim into two different claims.
Claim 16 provides for the use of iptacopan or a pharmaceutically acceptable salt thereof, but, since the claim does not set forth any steps involved in the method/process, it is unclear what method/process applicant is intending to encompass. A claim is indefinite where it merely recites a use without any active, positive steps delimiting how this use is actually practiced.
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) 15 is rejected under 35 U.S.C. 102(a)(2) as being anticipated clearly by Adams et al (US 2016/0152605 A1).
Adams et al discloses the following compound:
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, which is known as iptacopan hydrochloride (see page 115, example 26d).
Regardless of the intended use, this is identical with the claim.
Claim Rejections - 35 USC § 103
The following is a quotation of 35 U.S.C. 103 which forms the basis for all obviousness rejections set forth in this Office action:
A patent for a claimed invention may not be obtained, notwithstanding that the claimed invention is not identically disclosed as set forth in section 102, if the differences between the claimed invention and the prior art are such that the claimed invention as a whole would have been obvious before the effective filing date of the claimed invention to a person having ordinary skill in the art to which the claimed invention pertains. Patentability shall not be negated by the manner in which the invention was made.
The factual inquiries set forth in Graham v. John Deere Co., 383 U.S. 1, 148 USPQ 459 (1966), that are applied for establishing a background for determining obviousness under 35 U.S.C. 103 are summarized as follows:
1. Determining the scope and contents of the prior art.
2. Ascertaining the differences between the prior art and the claims at issue.
3. Resolving the level of ordinary skill in the pertinent art.
4. Considering objective evidence present in the application indicating obviousness or nonobviousness.
5. Claim 1-5 and 15 are rejected under 35 U.S.C. 103 as being unpatentable over Adams et al (US 2016/0152605 A1) in view of Wikipedia (List of autoimmune diseases, Jan. 9, 2019, p. 1-20).
Applicants claim the followings:
1. A method of treating an autoimmune hematological disorder in a subject, e.g., a patient, in need thereof, the method comprising administering to the subject, e.g., the patient, a therapeutically effective amount of iptacopan or a pharmaceutically acceptable salt thereof (e.g., iptacopan hydrochloride) to thereby treat the subject, e.g. patient, wherein the autoimmune hematological disorder is selected from the group consisting of immune thrombocytopenia (ITP), cold agglutinin disease (CAD), warm autoimmune hemolytic anemia (wAIHA), and thrombic thrombocytopenic purpura (TTP).
2. The method of claim 1, wherein the therapeutically effective amount of iptacopan or a pharmaceutically acceptable salt thereof (e.g., iptacopan hydrochloride) comprises a dose of about 50 mg to about 200 mg, about 50 mg, about 100 mg, or about 200 mg (wherein the dosing amount refers to the anhydrous free base of iptacopan hydrochloride).
3. The method of claim 1, wherein the therapeutically effective amount of iptacopan or a pharmaceutically acceptable salt thereof (e.g., iptacopan hydrochloride) is administered once daily (q.d.) or twice daily (b.i.d.) to the subject, e.g., the patient.
4. The method of claim 1, wherein the method comprises orally administering iptacopan or a pharmaceutically acceptable salt thereof (e.g., iptacopan hydrochloride, e.g., iptacopan hydrochloride monohydrate, e.g., iptacopan hydrochloride monohydrate Form HB)to the subject, e.g., the patient.
5. The method of claim 1, wherein the method comprises orally administering iptacopan or a pharmaceutically acceptable salt thereof, at a dose of about 200 mg twice daily (b.i.d.) to the subject, e.g., the patient, wherein the autoimmune hematological disorder is selected from the group consisting of immune thrombocytopenia (ITP), or cold agglutinin disease (CAD).
Determination of the scope and content of the prior art
Adams et al discloses a method of treating a variety of diseases and conditions including an autoimmune disease (see page 131, claims 19-20) by using a compound selected from 4-((4-ethoxy-1-((5-methoxy-7-methyl-1 H-indol-4-yl)methyl)piperidin-2-yl)benzoic acid (see page 130, claim 15) or
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, which is known as iptacopan hydrochloride as in claims 1-2, 4 15 (see page 115, example 26d).
Furthermore, a pharmaceutical composition or combination can be in unit dosage of about 1-1000 mg of active ingredient(s) for a subject of about 50-70 kg, or about 1-500 mg or about 1-250 mg as in claim 2 or about 1-150 mg or about 0.5-100 mg, or about 1-50 mg of active ingredients. The therapeutically effective dosage of a compound, the pharmaceutical composition, or the combinations thereof, is dependent on the species of the subject, the body weight, age and individual condition, the disorder or disease or the severity thereof being treated. A physician, clinician or veterinarian of ordinary skill can readily determine the effective amount of each of the active ingredients necessary to prevent, treat or inhibit the progress of the disorder or disease(see page 25 , a paragraph#0447).
In addition, the kit may be used for administering different dosage forms, for example, oral and parenteral, for administering the separate compositions at different dosage intervals, as in claims 3 and 5 (partially) or for titrating the separate compositions against one another. To assist compliance, the kit of the invention typically comprises directions for administration. see page 25 , a paragraph#0454)
The current invention, however, differs from the prior art in that the specific names of autoimmune diseases such as immune thrombocytopenia (ITP), cold agglutinin disease (CAD), and/or thrombic thrombocytopenic purpura (TTP) and a dose of about 200 mg twice daily are unspecified in the prior art.
Wikipedia teaches the list of autoimmune diseases which include autoimmune hemolvtic anemia , autoimmune neutropenia , autoimmune thrombocytooenic purpura , cold agalutinin disease as in claim 1and others (see page 6 , a section of blood).
Ascertainment of the difference between the prior art and the claims
The difference between the instant application and the applied Adams art is that the Adams et al does not expressly teach the specific names of autoimmune diseases such as immune thrombocytopenia (ITP), cold agglutinin disease (CAD), and/or thrombic thrombocytopenic purpura (TTP) and a dose of iptacopan about 200 mg twice daily. The deficiencies of the Adams et al are partially cured by the Wikipedia .
The difference between the instant application and the applied Wikipedia art is that the Wikipedia does not expressly teach the use of the therapeutically effective amount of iptacopan or a pharmaceutically acceptable salt for the treatment and its dose of about 200 mg twice daily. The deficiency of the Wikipedia are partially cured by the Adams et al.
Resolving the level of ordinary skill in the pertinent art.
Regarding the Claim 5, with respect to the lack of disclosing the claimed dose of iptacopan about 200 mg twice daily, Adams et al does teach that the compound of iptacopan hydrochloride can be used for treating an autoimmune disease such as cold agglutinin disease as shown in Wikipedia ; Adams et al also gives a general guidance that an active ingredient can be in the range of about 1-250 mg (see page 25 , a paragraph#0447). Furthermore, Adams et al describes that a physician, clinician or veterinarian of ordinary skill can readily determine the effective amount of each of the active ingredients necessary to treat the disorder or disease(see page 25 , a paragraph#0447). Therefore, if the skilled artisan in the art had desired to control a patient with the condition of cold agglutinin disease as shown in Wikipedia , it would have been obvious to be motivated to adjust the dosage of Adams’ iptacopan about 200 mg twice daily by a routine medical practice. This is because it is within the purview of the skilled artisan in the art to do so.
Considering objective evidence present in the application indicating obviousness or nonobviousness.
Adams et al expressly discloses the method of treating an autoimmune disease (see page 131, claims 19-20) by using the compound selected from 4-((4-ethoxy-1-((5-methoxy-7-methyl-1 H-indol-4-yl)methyl)piperidin-2-yl)benzoic acid (see page 130, claim 15) or iptacopan hydrochloride (see page 115, example 26d).
Whereas Wikipedia does teach the list of autoimmune diseases which include autoimmune hemolvtic anemia , autoimmune neutropenia , autoimmune thrombocytooenic purpura , cold agalutinin disease and others (see page 7 , a section of blood).
Adams et al and Wikipedia are closely related to each other with respect to the broad spectrum of treating any autoimmune diseases vs. the specific names of autoimmune hematological diseases. Furthermore, Adams et al does teach that any autoimmune disease including cold agalutinin disease can be treatable by using iptacopan (see page 130, claim 15) and (see page 131, claims 19-20).
So, if the skilled artisan in the art had desired to treat cold agalutinin disease by using iptacopan, it would have been obvious to the skilled artisan in the art before the effective filing date of the claimed invention to be motivated to combine the teaching of Wikipedia‘s cold agalutinin disease with Adams’ method of treating an autoimmune disease. This is because the skilled artisan in the art would expect such a combined prior art to be successful and feasible as guidance shown in the prior art.
Conclusion
Claims 1-16 are rejected.
Claims 1-5 and 15-16 are objected
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/TAYLOR V OH/Primary Examiner, Art Unit 1625 2/4/2026