DETAILED ACTION
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
Claims 1, 3-6, and 9-15 are pending. Acknowledgment is made of the amendment of claims 1, 9, and 11, and the cancellation of claims 2, 7, and 8, in the reply filed 08/18/2026.
Withdrawn Objections/Rejections
Applicant’s amendment to the drawings, filed 08/18/2026, overcomes the objection to the drawings for minor informalities. The objection to the drawings has been withdrawn.
Applicant’s amendment to the claims, filed 08/18/2026, overcomes the objection to claims 1, 8, 9, and 11 for minor informalities. The objection to claims 1, 8, 9, and 11 has been withdrawn.
Applicant’s amendment to the claims, filed 08/18/2026, overcomes the rejection of claims 1-10 and 13-15 under 35 U.S.C. 112(b) for indefiniteness. The rejection of claims 1-10 and 13-15 has been withdrawn.
Maintained Rejections
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, 3-6, and 13-15 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 method of treating LGMD comprising administering compounds of formula (I) where R2 to R6 are H, does not reasonably provide enablement for a method of treating LGMD comprising administering compounds of formula (I). The specification does not enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make the invention commensurate in scope with these claims.
To be enabling, the specification of the patent application must teach those skilled in the art how to make and use the full scope of the claimed invention without undue experimentation. In re Wright, 999 F.2d 1557, 1561 (Fd. Cir. 1993). Explaining what is meant by "undue experimentation," the Federal Circuit has stated that:
The test is not merely quantitative, since a considerable amount of experimentation is permissible, if it is merely routine, or if the specification in question provides a reasonable amount of guidance with respect to the direction in which experimentation should proceed to enable the determination of how to practice a desired embodiment of the claimed invention. PPG v. Guardian, 75 F.3d 1558, 1564 (Fed. Cir. 1996). As pointed out by the court in In re Angstadt, 537 F.2d 498 at 504 (CCPA 1976), the key word is "undue", not "experimentation".
The factors that may be considered in determining whether a disclosure would require undue experimentation are set forth In re Wands, 8 USPQ2d 1400 (CAFC 1988) at 1404 wherein, citing Ex parte Forman, 230 USPQ 546 (Bd. Apls. 1986) at 547 the court recited eight factors:
1- the quantity of experimentation necessary,
2- the amount of direction or guidance provided,
3- the presence or absence of working examples,
4- the nature of the invention,
5- the state of the prior art,
6- the relative skill of those in the art,
7- the predictability of the art, and
8- the breadth of the claims
These factors are always applied against the background understanding that scope of enablement varies inversely with the degree of unpredictability involved. In re Fisher, 57 CCPA 1099, 1108, 427 F.2d 833, 839, 166 USPQ 18, 24 (1970). Keeping that in mind, the Wands factors are relevant to the instant fact situation for the following reasons:
The nature of the invention
The nature of the invention relates to methods of treating LGMD comprising administering compounds of formula (I) in claim 1.
Predictability of the art
The hypothetical compounds in claim 1 would be unpredictable in terms of one skilled in the art being able to synthesize every possible compound claimed in instant claim 1. It is well established that “the scope of enablement varies inversely with the degree of unpredictability of the factors involved,” and physiological activity is generally considered to be an unpredictable factor. See In re Fisher, 427 F.2d 833, 839, 166 USPQ 18, 24 (CCPA 1970).
In terms of the law, MPEP 2107.03 states “evidence of pharmacological or other biological activity of a compound will be relevant to an asserted therapeutic use if there is a reasonable correlation between the activity in question and the asserted utility. Cross v. Iizuka, 753 F.2d 1040, 224 USPQ 739 (Fed. Cir. 1985); In re Jolles, 628 F.2d 1322, 206 USPQ 885 (CCPA 1980); Nelson v. Bowler, 626 F.2d 853, 206 USPQ 881 (CCPA 1980).” If correlation is lacking, it cannot be relied upon, Ex parte Powers, 220 USPQ 924; Rey-Bellet and Spiegelberg v. Engelhardt v. Schindler, 181 USPQ 453; Knapp v. Anderson, 177 USPQ 688. Indeed, the correlation must have been established “at the time the tests were performed”, Hoffman v. Klaus, 9 USPQ2d 1657.
Level of skill in the art
An ordinary artisan in the area of drug development would have experience in synthesizing and screening chemical compounds for particular activities, such as a medical doctor or chemist. Screening of new drug candidates, while complex, is routine in the art. The process of finding new drugs that have in vitro activity against a particular biological target, (i.e., receptor, enzyme, etc.) is well known. Additionally, while high throughput screening assays can often be employed, developing a therapeutic method, as claimed, is generally not well-known or routine, given the complexity of certain biological systems.
4. The breadth of the claims
The scope of the claims involves compounds of formula (I), shown below.
PNG
media_image1.png
211
156
media_image1.png
Greyscale
Claim 1 is very broad in the number of variables and the options of substituents for each variable. There is a large amount of hypothetical compounds included in claim 1, because each substituent may optionally be substituted with more substituents.
5. The amount of direction provided, the presence or absence of working examples, and the quantity of experimentation necessary
The specification only provides the synthesis of 9 compounds. In all of the compounds synthesized, R2 to R6 are H.
Synthesis methods are not taught in the specification to provide for the aforementioned variables to include all of the possible substituents listed in the claims. It would be expected that the variations of possible substituents (e.g., R2 to R6 being any alkyl, -COR7, -C(O)OR7, -OR7, -NR7R8, or halogen, which may then be further substituted) would change the reactivity of the compounds, and therefore would require alternate synthesis methods. In addition to requiring alternate synthesis methods, there is no guarantee that such compounds would be useful in the treatment of limb girdle muscular dystrophy, since the physiological properties of the compounds would be different. It would require one skilled in the art, such as a chemist, to perform hundreds of reactions to determine which compounds of formula (I) can be prepared and would require synthesis methods other than those provided in the specification. One would then have to determine which of the compounds is capable of treating LGMD through clinical trials or in vitro or in vivo studies. This is undue experimentation given the limited guidance and direction provided by Applicants.
Accordingly, the instant claims do not comply with the enablement requirement of 35 U.S.C. 112(a), since to practice the claimed invention a person of ordinary skill in the art would have to engage in undue experimentation, with no assurance of success.
Applicant Argues:
Applicant stated in the Remarks filed 08/18/2026 that Applicant has amended the claims so that the claimed compounds are “more closely aligned with those exemplified in the specification”.
Examiner Responds:
Applicant’s arguments filed 08/18/2026 have been fully considered but they are not persuasive. The scope of the R2 to R6 variables in the claims is significantly broader than what is exemplified in the specification, stating that R2 to R6 may be alkyl, -COR7, -C(O)OR7, -OR7, -NR7R8, or halogen, which may then be further substituted by a list of groups with different chemical and physiological properties. The specification only contains exemplified compounds where R2 to R6 are H. As stated in the above rejection, in addition to requiring alternate synthesis methods, there is no guarantee that such compounds would be useful in the treatment of limb girdle muscular dystrophy, since the physiological properties of the compounds would be different.
Claim Rejections - 35 USC § 103
The text of those sections of Title 35, U.S. Code not included in this action can be found in a prior Office action.
The factual inquiries 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.
This application currently names joint inventors. In considering patentability of the claims the examiner presumes that the subject matter of the various claims was commonly owned as of the effective filing date of the claimed invention(s) absent any evidence to the contrary. Applicant is advised of the obligation under 37 CFR 1.56 to point out the inventor and effective filing dates of each claim that was not commonly owned as of the effective filing date of the later invention in order for the examiner to consider the applicability of 35 U.S.C. 102(b)(2)(C) for any potential 35 U.S.C. 102(a)(2) prior art against the later invention.
Claims 1, 3-6, and 9-15 are rejected under 35 U.S.C. 103 as being unpatentable over Snape (WO 2022243973 A1), with an effective filing date of 21 May 2021, further in view of Magdinier et al. (WO 2021233810 A1), with an effectively filed date of 19 May 2020.
Snape teaches, in claim 16, a method of treating muscular dystrophy type 1 in a subject comprising administering 4-benzyl-2-naphthalen-1-yl-[1,2,4]thiadiazolidine-3,5-dione, shown below, as in instant claims 1, 3-6, and 9-12. It is taught, in claim 14, that the method comprises an additional adjuvant, as in instant claim 15.
PNG
media_image2.png
120
90
media_image2.png
Greyscale
Snape teaches, on page 10, that the disease being treated “includes, but is not limited, to myotonic dystrophy type 1 (DM1), also known as Steinherfs Disease, whether of Congenital, Childhood or Adult Onset sub-type DM1, as well as Fuchs endothelial corneal dystrophy”.
Snape fails to teach a specific embodiment where the muscular dystrophy being treated is limb girdle muscular dystrophy R1 calpain 3-related, as in instant claims 13 and 14.
However, Magdinier et al. teaches, on page 14 lines 31-35, a method comprising “the treatment of the muscle fibers innervated by motor neurons according to the invention with a test compound”. The method of treating the skeletal muscle fibers is taught, in claim 2, to comprise administering TDZD-8, shown below.
PNG
media_image3.png
183
282
media_image3.png
Greyscale
It is taught, in claim 13, that the neuromuscular disease is type 2A Limb-Girdle Muscular Dystrophy (LGMD2A).
Therefore, it would be prima facie obvious to one of ordinary skill in the art to extend the method of treating muscular dystrophy taught by Snape to include the treatment of LGMD, and specific types of LGMD, since a similar compound is taught by Magdinier et al. to be used in the treatment of LGMD, and since Snape teaches that the compounds may be used in the treatment of other endothelial muscular dystrophies.
One of ordinary skill in the art would have a reasonable expectation of success using compounds known to treat myotonic and endothelial muscular dystrophies for the treatment of the specific muscular dystrophies listed in instant claims 13 and 14, since the same compounds are taught to treat similar muscular dystrophies in the prior art. Even though the specific type of LGMD of instant claims 13 and 14 is not taught by the prior art, it would be routine procedure for one of ordinary skill in the art to use a known method to treat these types of LGMD, and one would have a reasonable expectation of success in doing so.
Applicant Argues:
Applicant states that the Formula (I) taught by Snape et al. is not present in the priority document. Applicant argues that the compound and method of treating LGMD2A taught by Magdinier et al. would not motivate one to use the compound taught by Snape et al. to treat LGMD2A. Additionally, Applicant states that Snape et al. does not teach the mechanism of action of Tideglusib on Wnt, mTOR, and GSK3β.
Examiner Responds:
Applicant’s arguments filed 08/18/2026 have been fully considered but they are not persuasive. The priority document of Snape et al. teaches, as stated in the rejection above, a method of treating DM1 myotonic dystrophy with Tideglusib (4-benzyl-2-naphthalen-1-yl-[1,2,4]thiadiazolidine-3,5-dione). Compounds with a close structural similarity are used by Magdinier et al. to treat a different form of dystrophy, LGMD2A. Therefore, one would be motivated to use Tideglusib to also treat other forms of dystrophy, including LGMD2A, since the compounds of close structural similarity, which are used to treat similar disorders, would be expected by one of ordinary skill in the art to have the same or similar effects.
Regarding Applicant’s statement that the mechanism of action on specific pathways is not mentioned by Snape et al., MPEP 2112 I states: “[T]he discovery of a previously unappreciated property of a prior art composition, or of a scientific explanation for the prior art’s functioning, does not render the old composition patentably new to the discoverer.” Atlas Powder Co. v. Ireco Inc., 190 F.3d 1342, 1347, 51 USPQ2d 1943, 1947 (Fed. Cir. 1999). The mechanism of action is not recited by Applicant in the claims, nor is it relevant to the arguments in the rejection above, since the discovery of a mechanism of action by an existing drug is not a patentably new discovery.
Conclusion
Claims 1, 3-6, and 9-15 are rejected.
THIS ACTION IS MADE FINAL. 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 RILLA M SAMSELL whose telephone number is (703)756-5841. The examiner can normally be reached Monday-Friday, 7-3.
Examiner interviews are available via telephone, in-person, and video conferencing using a USPTO supplied web-based collaboration tool. To schedule an interview, applicant is encouraged to use the USPTO Automated Interview Request (AIR) at http://www.uspto.gov/interviewpractice.
If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Jeffrey Murray can be reached at (571) 272-9023. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
Information regarding the status of published or unpublished applications may be obtained from Patent Center. Unpublished application information in Patent Center is available to registered users. To file and manage patent submissions in Patent Center, visit: https://patentcenter.uspto.gov. Visit https://www.uspto.gov/patents/apply/patent-center for more information about Patent Center and https://www.uspto.gov/patents/docx for information about filing in DOCX format. For additional questions, contact the Electronic Business Center (EBC) at 866-217-9197 (toll-free). If you would like assistance from a USPTO Customer Service Representative, call 800-786-9199 (IN USA OR CANADA) or 571-272-1000.
/R.M.S./Examiner, Art Unit 1624
/JEFFREY H MURRAY/Supervisory Patent Examiner, Art Unit 1624
Prosecution Insights