DETAILED ACTION
Acknowledgment is made of the receipt and entry of the amendment filed on 1/26/2026.
Any rejection found in the previous Office Action and not repeated herein has been withdrawn based upon Applicant’s amendments to the claims.
The text of those sections of Title 35, U.S. Code not included in this action can be found in a prior Office action.
Claims 1-6, 8, 17-20 and 22-25 are currently pending and under examination.
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 .
Claim Rejections - 35 USC § 112
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 1-6, 8, 17-20 and 22-25 are rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, because the specification, while being enabling for a combination treatment method for treating cystic fibrosis (CF) in a CF subject having one or more CFTR missense, nonsense and deletion mutations, wherein the combination treatment method comprises administering therapeutically effective amounts of a Class I potentiator, wherein the Class I potentiator is VX-770:
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combined with a Class II potentiator of structure 2e or 2i:
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to a subject in need of treatment, does not reasonably provide enablement for a combination treatment method for treating cystic fibrosis (CF) in a CF subject having one or more CFTR missense, nonsense and deletion mutations, wherein the combination treatment method comprises administering a Class I potentiator combined with at least one Class II potentiator having structure (A)
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wherein:
R1a is benzyl or benzyl substituted with one or more substituents selected from the group consisting of halo, and C1-C6 alkoxy; R2a is H or C1-C6 alkyl; and (sic). The specification does not enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to use the invention commensurate in scope with these claims (partially newly reapplied as necessitated by amendment).
Enablement is considered in view of the Wands factors (MPEP 2164.01(A)). These include: nature of the invention, breadth of the claims, guidance of the specification, the existence of working examples, state of the art predictability of the art and the amount of experimentation necessary. All of the Wands factors have been considered with regard to the instant claims, with the most relevant factors discussed below.
Nature of the Invention: The claims are drawn to a combination treatment method for treating cystic fibrosis (CF) in a CF subject having one or more CFTR missense, nonsense and deletion mutations, wherein the combination treatment method comprises administering any and all Class I potentiators combined with at least one Class II potentiator having structure (A)
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wherein:
R1a is benzyl or benzyl substituted with one or more substituents selected from the group consisting of halo, and C1-C6 alkoxy; R2a is H or C1-C6 alkyl; and (sic).
The nature of the invention is complex in that claims are is drawn to using any Class I potentiator in combination with any of the defined Class II potentiators as a therapeutic method of treating cystic fibrosis.
Breadth of the Claims: The claims are broad in that the claims recite a combination treatment method for treating cystic fibrosis (CF) in a CF subject having one or more CFTR missense, nonsense and deletion mutations, wherein the combination treatment method comprises administering any Class I potentiator combined with at least one Class II potentiator having structure (A)
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wherein:
R1a is benzyl or benzyl substituted with one or more substituents selected from the group consisting of halo, and C1-C6 alkoxy; R2a is H or C1-C6 alkyl; and (sic). The complex nature of the subject matter of this invention is greatly exacerbated by the breadth of the claims.
Guidance of the Specification and Existence of Working Examples: The specification describes administering is VX-770:
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combined with a Class II potentiator of structure 2e or 2i:
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for treating cystic fibrosis.
The specification envisions that by administering any type of Class I potentiator with Class II potentiators as claimed that this combination will have efficacy for treating cystic fibrosis.
However, no working examples are provided with regard to a method of treating cystic fibrosis with a generic Class I potentiator in combination with each type of the generically claimed Class II potentiators or representative examples of each.
Predictability and State of the Art: The state of the art at the time the invention was made was unpredictable and underdeveloped.
Mayo Clinic: Cystic Fibrosis (U*) teaches that medical options for treatment of cystic fibrosis include antibiotics, medicines that target gene changes, anti-inflammatory medicines, mucus thinning medication and bronchodilators.
Thus, while the claim-designated method may be useful for providing such an effect, Applicant does not disclose a combination treatment method for treating cystic fibrosis (CF) in a CF subject having one or more CFTR missense, nonsense and deletion mutations, wherein the combination treatment method comprises administering a Class I potentiator combined with at least one Class II potentiator having structure (A)
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wherein:
R1a is benzyl or benzyl substituted with one or more substituents selected from the group consisting of halo, and C1-C6 alkoxy; R2a is H or C1-C6 alkyl; and (sic).
Amount of Experimentation Necessary: The quantity of experimentation necessary to carry out the claimed invention is high, as the skilled artisan could not rely on the prior art or instant specification to teach how to use a combination of a Class I potentiator combined with at least one Class II potentiator having structure (A)
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wherein:
R1a is benzyl or benzyl substituted with one or more substituents selected from the group consisting of halo, and C1-C6 alkoxy; R2a is H or C1-C6 alkyl; and (sic) that can be administered in a therapeutically effective dose with an acceptable level of side-effects.
In view of the breadth of the claims and the lack of guidance provided by the specification as well as the unpredictability of the art, the skilled artisan would have required an undue amount of experimentation to make and/or use the claimed invention. Therefore, claims 1-8, 17-20 and 22-25 are not considered to be fully enabled by the instant specification.
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 1-6, 8, 17-20 and 22-25 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 applications subject to pre-AIA 35 U.S.C. 112, the applicant), regards as the invention (newly applied as necessitated by amendment).
The metes and bounds of claim 1 are rendered uncertain by the phrase “at least one Class II potentiator having structure (A)
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wherein:
R1a is benzyl or benzyl substituted with one or more substituents selected from the group consisting of halo, and C1-C6 alkoxy; R2a is H or C1-C6 alkyl; and” because the claim doesn’t have an end and it is not clear what is intended to come after the “and”. The lack of clarity renders the claims indefinite since the resulting claims do not clearly set forth the metes and bounds of the patent protection desired.
Response to Arguments
Applicant's arguments filed 1/26/2026 have been fully considered but they are not persuasive. Applicant argues that amended claim 1 is enabled to its amended scope and that the present inventors had discovered a treatment of cystic fibrosis of certain mutations by a combination therapy, which further activates a Class I potentiator with a Class II potentiator, thus improving the efficacy of the Class I potentiator by several folds. The underlying mechanism for the claimed therapy is free of prior art. Applicant further argues that Class I potentiators were well known and their potentiating mechanism was well understood. VX-770 is thus representative of the Class I potentiators that are exemplified and claimed. Because compounds 2e and 2i were demonstrated to improve the activity of VX-770, and they are highly representative.
However, this is not found persuasive because Applicant’s claims are not commensurate in scope with what Applicant has shown by way of example in the originally filed specification. The claim is still a reach through claim, in that any Class I potentiator, including those not discovered yet, are claimed. Further, only showing efficacy with one Class I potentiator is not representative. Applicant has demonstrated efficacy with specific compound, not a whole class of compounds. This also applies to Class II potentiators. The claims are not limited to compound A and are not limited to only the Class II potentiators in the specification. Therefore, the claims are not fully commensurate in scope with what Applicant is enabled for and the rejection is maintained for the reasons of record and the reasons set forth above.
Conclusion
No claims are allowed.
Applicant's amendment necessitated the new ground(s) of rejection presented in this Office action. Accordingly, THIS ACTION IS MADE FINAL. See MPEP § 706.07(a). Applicant is reminded of the extension of time policy as set forth in 37 CFR 1.136(a).
A shortened statutory period for reply to this final action is set to expire THREE MONTHS from the mailing date of this action. In the event a first reply is filed within TWO MONTHS of the mailing date of this final action and the advisory action is not mailed until after the end of the THREE-MONTH shortened statutory period, then the shortened statutory period will expire on the date the advisory action is mailed, and any nonprovisional extension fee (37 CFR 1.17(a)) pursuant to 37 CFR 1.136(a) will be calculated from the mailing date of the advisory action. In no event, however, will the statutory period for reply expire later than SIX MONTHS from the mailing date of this final action.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to Amy L. Clark whose telephone number is (571)272-1310. The examiner can normally be reached M-F 8:00am-5:00pm.
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If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Jennifer Michener can be reached at 571-272-1424. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
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/AMY L CLARK/Supervisory Patent Examiner, Art Unit 1628
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