DETAILED ACTION
Claims 1-14 are pending in the instant application.
Applicant's election read on species election as follow:
1)-b)-i) serine palmitoyltransferase (SPL/SPTLC)
2)-b)-i) small molecule from Table A= compound 4 in the reply filed on 12/18/2025 is acknowledged.
The requirement is still deemed proper and is therefore made FINAL.
Claims 1-9, 11-14 will be examining since read on species election without traverse as follow:
1)-b)-i) serine palmitoyltransferase (SPL/SPTLC)
2)-b)-i) small molecule from Table A= compound 4.
Claim10 and the remaining subject matter of claims 1-9, 11-14 are withdrawn per 37 CFR 1.142(b).
2. Claim Rejections - 35 USC § 112
The following is a quotation of the second paragraph of 35 U.S.C. 112:
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 1-9, 11-13 are rejected under 35 U.S.C. 112, second paragraph, as being indefinite for failing to particularly point out and distinctly claim the subject matter which applicant regards as the invention.
Claims 1-13 are rejected because the term “an inhibitor of sphingolipid adapted” is confusing. Are these claims being “method” or “compound” claims? Correction is required.
3. 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.
Claims 1-9, 11-13 are 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 USPQ 475 (D.D.C. 1966).
4. Claim Rejections - 35 USC § 112
The following is a quotation of the first paragraph of 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 1-9, 11-14 are rejected under 35 U.S.C. 112, first paragraph, because the specification, while being enabling for inhibition of sphingolipids, does not reasonably provide enablement for treating muscle disease. 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 and/or use the invention commensurate in scope with these claims.
There are many factors to be considered when determining whether there is sufficient evidence to support a determination that a disclosure does not satisfy the enablement requirement and whether any necessary experimentation is “undue”. These factors include 1) the breadth of the claims, 2) the nature of the invention, 3) the state of the prior art, 4) the level of one of ordinary skill, 5) the level of predictability in the art, 6) the amount of direction provided by the inventor, 7) the existence of working examples, and 8) the quantity of experimentation needed to make or use the invention based on the content of the disclosure. In re Wands, 858 F.2d 731, 737, 8 USPQ2d 1400, 1404 (Fed. Cir. 1988).
1) The breadth of the claims.
2) The nature of the invention,
3) The state of the prior art,
4) The level of one of ordinary skill,
5) The level of predictability in the art,
6) The amount of direction provided by the inventor,
7) The existence of working examples,
8) The quantity of experimentation needed to make or use the invention based on the content of the disclosure.
The nature of the invention: The instant invention is drawn to method for treating and/or preventing muscle disease.
The state of the prior art: As indicated by the prior art, there is not any association between sphingolipids inhibitors and treating muscle disease. There is no guidance in the prior art that sphingolipids inhibitor could be useful to treat muscle disease.
The predictability in the art: At present using sphingolipids to treat muscle disease is purely speculative. It is noted that the pharmaceutical art is unpredictable, requiring each embodiment to be individually assessed for physiological activity. In re Fisher, 427 F. 2d 833, 166 USPQ 18 (CCPA 1970) indicates that the more unpredictable an area is, the more specific enablement is necessary in order to satisfy the statute. They have not shown a nexus to treat diseases but have provided only reports in vitro which, absent a correlation to treat of diseases, are not sufficient to enable the invention.
The level of the skill in the art: The level of skill in the art is high.
Amount of guidance/working examples: Applicant provides examples of the test compounds to inhibit sphingolipids on pages 41-45. However, there is no guidance for using a therapeutically effective amount of a compound of Formula (I) to treat muscle disease.
The breadth of the claims: The claims encompass a method for treating muscle disease.
The quantity of experimentation needed: Since the guidance and teaching provided by the specification is insufficient for treating diseases associated with therapeutically effective amount of a compound of formula (I) is efficacious, one of ordinary skill in the art, even with high level of skill, is unable to use the instant compounds as claimed without undue experimentation.
Taking all of the above into consideration, it is not seen where the instant claims 1-9, 11-14, for treating diseases associated with therapeutically effective amount of a compound of Formula (I) is efficacious, have been enabled by the instant specification.
5. Claims 1-9, 11-14 are rejected under 35 U.S.C. 112, first paragraph, because the specification, does not reasonably provide enablement for treating and preventing any and all diseases. The specification does not enable any person skilled in the art to which, or with which it is most nearly connected, to make and/or use the invention commensurate in scope with these claims. Applicants are not enabled for preventing any of these diseases. The only established prophylactics are vaccines not the compounds such as present here. In addition, it is presumed that “prevention” of the claimed diseases would require a method of identifying those individuals who will develop the claimed diseases before they exhibit symptoms. There is no evidence of record that would guide the skilled clinician to identify those who have the potential of becoming afflicted.
“The factors to be considered [in making an enablement rejection] have been summarized as the quantity of experimentation necessary, the amount of direction or guidance presented, the presence or absence of working examples, the nature of the invention, the state of the prior art, the relative skill of those in that art, the predictability or unpredictability of the art, and the breadth of the claims”, In re Rainer, 146 USPQ 218 (1965); In re Colianni, 195 USPQ 150, Ex parte Formal, 230 USPQ 546. 1) As discussed above, preventing diseases requires identifying those patients who will acquire the disease before occurs. This would require extensive and potentially opened ended clinical research on healthy subjects. 2) There is no working example of such a preventive procedure in man or animal in the specification. 3) The claims rejected are drawn to medical treatment and are therefore physiological in nature. 4) The state of the art is that no general procedure is art-recognized for determining which patients generally will become afflicted before the fact. 5) The artisan using Applicants invention would be a Board Certified physician who specialized to treat diseases with an MD degree and several years of experience. Despite intensive efforts, pharmaceutical science has been unable to find a way of getting a compound to be effective for the prevention of disorder diseases generally. Under such circumstances, it is proper for the PTO to require evidence that such an unprecedented feat has actually been accomplished, In re Ferens, 163 USPQ 609. No such evidence has been presented in this case. The failure of skilled scientists to achieve a goal is substantial evidence that achieving such a goal is beyond the skill of practitioners in that art, Genentech vs. Novo Nordisk, 42 USPQ2nd 1001, 1006. This establishes that it is not reasonable to any agent to be able to prevent disorders generally. That is, the skill is so low that no compound effective generally against disorders has ever been found let alone one that can prevent such conditions. 7) 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). 8) The claims broadly read on all patients, not just those undergoing therapy for the claimed diseases and on the multitude of compounds embraced by Formula (I).
The Examiner suggests deletion of the word “prevention”.
6. 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.
Claims 1-9, 11-14 are rejected under 35 U.S.C. 102(a)(2) as being anticipated by Asano et al., US 10519110. Asano et al. discloses the instant claimed compound on columns 213-214 as follow:
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. On columns 527-528 disclose the above compound can be used for SPT enzyme inhibition. On columns 531-532 disclose the above compound can be used to treat Niemann-pick disease, cancer, congenital disease associated with sphingolipid accumulation. The above compound can be serine palmitoyltransferase (SPT) inhibitors (abstract). Cancer, congenital disease can cause muscle disease too. The scope of claims 8-9 is inherently there since Asano inhibit (SPT). Therefore, the instant claims are anticipated by Asano et al.
7. Claims 1-9, 11-14 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Adachi et al., Biochemical and Biophysical Research Communications 497 (2018), 1171-1176. Adachi et al. discloses the instant claimed compound on page 1173 as follow:
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,
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. On abstract and Table 1 disclose the above compound can be used for SPT enzyme inhibition. On columns 531-532 disclose the above compound can be used to treat cancer. The above compound can be serine palmitoyltransferase (SPT) inhibitors (abstract). Cancer can cause muscle disease too. The scope of claims 8-9 is inherently there since Adachi inhibit (SPT). Adachi disclose potent SPT inhibitors with enzymatic, binding, kinetic, and cellular analyses on page 1176. Therefore, the instant claims are anticipated by Adachi et al.
8. Claims 1-9, 11-14 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Kojima et al., Bioorganic & Medicinal Chemistry 26 (2018), 2452-2465. Kojima et al. discloses the instant claimed compound on page 2453, figure 2 as follow:
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. On abstract and Figure 3 on page 2457 disclose the above compound can be used for SPT enzyme inhibition. On page 2458, Table 1 shows the above compound has SPT enzyme inhibition activity. On page 2459 disclose the above compound suppressed growth of the cancer cell in conclusion). The above compound can be serine palmitoyltransferase (SPT) inhibitors (abstract). Cancer can cause muscle disease too. The scope of claims 8-9 is inherently there since Kojima inhibit (SPT). Therefore, the instant claims are anticipated by Kojima et al.
9. Any inquiry concerning this communication or earlier communications from the examiner should be directed to Niloofar Rahmani whose telephone number is
571-272-4329. The examiner can normally be reached on Monday through Friday from 8:30 am to 5:00 pm.
If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Renee Claytor, can be reached on 571-272-8394. The fax phone number for the organization where this application or proceeding is assigned is 703-872-9306.
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/NILOOFAR RAHMANI/
03/02/2026