Jae Gyun. Jeong

Patent Trials and Appeals Board

Oct 4, 2019

15030999 - (D) (P.T.A.B. Oct. 4, 2019)

UNITED STATES PATENT AND TRADEMARK OFFICE
UNITED STATES DEPARTMENT OF COMMERCE
United States Patent and Trademark Office
Address: COMMISSIONER FOR PATENTS
P.O. Box 1450
Alexandria, Virginia 22313-1450
www.uspto.gov
APPLICATION NO. FILING DATE FIRST NAMED INVENTOR ATTORNEY DOCKET NO. CONFIRMATION NO.
15/030,999 04/21/2016 Jae Gyun JEONG 33730-36745/US 6457
758 7590 10/04/2019
FENWICK & WEST LLP
SILICON VALLEY CENTER
801 CALIFORNIA STREET
MOUNTAIN VIEW, CA 94041
EXAMINER
ULM, JOHN D
ART UNIT PAPER NUMBER
1649
NOTIFICATION DATE DELIVERY MODE
10/04/2019 ELECTRONIC
Please find below and/or attached an Office communication concerning this application or proceeding.
The time period for reply, if any, is set in the attached communication.
Notice of the Office communication was sent electronically on above-indicated "Notification Date" to the
following e-mail address(es):
PTOC@Fenwick.com
PTOL-90A (Rev. 04/07)
UNITED STATES PATENT AND TRADEMARK OFFICE
__________

BEFORE THE PATENT TRIAL AND APPEAL BOARD
__________

Ex parte JAE GYUN JEONG
__________

Appeal 2018-007863
Application 15/030,999
Technology Center 1600
__________

Before FRANCISCO C. PRATS, TAWEN CHANG, and DAVID COTTA,
Administrative Patent Judges.

PRATS, Administrative Patent Judge.

DECISION ON APPEAL

Pursuant to 35 U.S.C. § 134(a), Appellant1 appeals from the
Examiner’s decision to finally reject claims 10 and 15–24. We have
jurisdiction under 35 U.S.C. § 6(b).
We REVERSE.

STATEMENT OF THE CASE
Appellant’s invention is directed to the discovery that amyotrophic
lateral sclerosis (ALS, also known as Lou Gehrig’s disease), a disorder

1 We use the word “Appellant” to refer to “applicant” as defined in 37
C.F.R. § 1.42. Appellant identifies the real party in interest as ViroMed Co.,
Ltd. Appeal Br. 3.
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involving the destruction of motor neurons, “can be treated by using a
composition containing, as an active ingredient, two or more isoforms of
hepatocyte growth factor (HGF) or a polynucleotide encoding the isoforms.”
Spec. 3.
The Specification discloses that, in one embodiment, “the two or more
isoforms of HGF include full-length HGF (flHGF) and deleted variant HGF
(dHGF). The use of a composition containing both the full-length HGF and
the deleted variant HGF can prevent or treat ALS very effectively.” Id. at
4–5.
The Specification discloses that “[t]he term ‘treatment’ used herein
refers to (a) the suppression of the progress of amyotrophic lateral sclerosis,
(b) the relief of amyotrophic lateral sclerosis, or (c) the removal of
amyotrophic lateral sclerosis.” Id. at 9.
The Specification discloses that, most preferably, “the pharmaceutical
composition of this invention may be administered intramuscularly, spinally,
intrathecally, intraventricularly, parenchymally, or intracranially.” Id. at 17.
The Specification discloses that a preferred example of a single
polynucleotide expressing the two or more distinct isomers of HGF useful
for treating ALS is the recombinant plasmid vector “pCK-HGFX7, the
contents of which are described in detail in PCT/KR1999/000855 and
PCT/KR2003/000548, as described above.” See id. at 16; see also id. at 2
(incorporating PCT/KR1999/000855 and PCT/KR2003/000548 by
reference).
Examples 1–5 in the Specification are in vitro experiments
investigating the effects of administering pCK-HGFX7 to a variety of
cultured neuronal cells. See id. at 21–30.
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Examples 1–3 show that, in vitro, pCK-HGFX7 can enhance neural
cell growth. See id. at 21–24. Example 3 also shows that, in vitro, pCK-
HGFX7 can inhibit neural cell apoptosis (cell death). See id. at 24–25.
Example 4 of the Specification shows that, when administered to
cultured cells subjected to hydrogen peroxide-induced oxidative stress
conditions, pCK-HGFX7 can inhibit motor neuron apoptosis, measured by
counting surviving cells and by assaying expression of relevant genes. See
id. at 25–29.
In Example 5, the Specification explains that the “in vitro assay using
the G93A mutant form of superoxide dismutase 1 (SOD1), which is one of
the ALS causes, has been developed by many researchers.” Id. at 29. The
Specification discloses that the results for Example 5 “indicat[e] the effect of
[pCK-HGFX7] inhibiting the deterioration in cell growth, which is caused
by the delivery of G93A mutant hSOD1 [human SOD1] (see FIG. 7).” Id. at
30.
In Example 6, the Specification explains that, because “superoxide
dismutase 1 (SOD1) mutation has been found to be one of the ALS causes,
the ALS mouse model using this gene was developed, and currently, ALS
researchers throughout the world have conducted various researches using
this animal model.” Id.
Example 6 describes an experiment in which pCK-HGFX7, as well as
control plasmids, were administered intramuscularly to mice having the
ALS-modeling SOD1 G93A mutation. See id. at 30–31 (“[The] B65JL-Tg
(SOD1*G93A)1Gur/J (002726) [mouse], which has been widely used, was
selected, and used for tests.”). The mice were tested for grip strength in
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“one of the representative methods for assessing muscular strength of the
mouse.” Id. at 31 (citation omitted).
The Specification discloses that the mice that received pCK-HGFX7
exhibited “significantly improved” grip strength compared to the mice that
received control plasmids. Id. at 32; see also Fig. 8 (showing results of grip
strength test).
Claims 10 and 24 illustrate the appealed subject matter, and read as
follows:
10. A method for treating amyotrophic lateral
sclerosis, the method comprising administering, to a human
patient with amyotrophic lateral sclerosis, a composition
containing, as an active ingredient, a polynucleotide encoding
two or more isoforms of hepatocyte growth factor (HGF),
wherein:
the two or more isoforms of HGF comprise a full-length
HGF (flHGF) and a deleted variant HGF (dHGF),
the full-length HGF (flHGF) comprises an amino acid
sequence of SEQ ID NO: 1, and the deleted variant
HGF (dHGF) comprises the amino acid sequence
of SEQ ID NO: 2.

24. A method for treating amyotrophic lateral sclerosis
comprising the step of: intramuscular administration of pCK-
HGFX7 to a human patient with amyotrophic lateral sclerosis.
Appeal Br. 35–36.
The following rejections are before us for review:
(1) Claims 10 and 15–24, under 35 U.S.C. § 112(a),2 for failure to
comply with the written description requirement (Ans. 3–5); and

2 This application was filed on April, 21, 2016, as the national stage of a
Korean PCT application filed October 22, 2014. See Application Data Sheet
entered April 21, 2016. Accordingly, the version of § 112 enacted by the
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(2) Claims 10 and 15–24, under 35 U.S.C. § 112(a), for failure to
comply with the enablement requirement (Ans. 5–9).

STANDARD OF REVIEW
As stated in In re Oetiker, 977 F.2d 1443, 1445 (Fed. Cir. 1992):
[T]he examiner bears the initial burden . . . of presenting a
prima facie case of unpatentability. . . .
After evidence or argument is submitted by the applicant in
response, patentability is determined on the totality of the
record, by a preponderance of evidence with due consideration
to persuasiveness of argument.

WRITTEN DESCRIPTION
The Examiner’s Prima Facie Case
The Examiner’s position, essentially, is that Appellant’s claims do not
comply with the written description requirement because the Specification
fails to establish that Appellant was in possession of the claimed methods of
treating ALS in humans. See Ans. 3–5.
In particular, the Examiner contends that the Specification does not
include a working example of treating ALS in a human, and except for the
known drug riluzole, “neither the instant specification nor the prior art of
record describes any method of treating ALS in humans that has proven to
provide even minimal benefit, much less one that involves the administration
of therapeutic polynucleotides.” Id. at 3.

Leahy-Smith America Invents Act (“AIA”) applies to the claims on appeal.
See AIA, Public Law 112-29, § 4(e), 125 Stat. 297.
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The Examiner contends that ALS “has, thus far, proven essentially
untreatable,” and therefore, possession of the claimed methods of treatment
“requires substantially more than a sound scientific theory in combination
with in vitro data obtained from an experimental system for which there is
no established correlation between positive results obtained for a therapeutic
protocol in that system and efficacy of that protocol in the treatment of
ALS.” Id. at 4.
The Examiner summarizes the rejection’s rationale as follows:
To demonstrate the reduction to practice of a therapeutic
protocol for the treatment of ALS in a human subject afflicted
therewith requires either a working embodiment, a
demonstration of operability of that protocol in the treatment of
an art accepted animal model of the condition to be treated
wherein that animal model has been shown to be reliably
predictive of the efficacy of a protocol in the treatment of the
condition, or a demonstration that the therapeutic agent
employed in that protocol possesses an in vitro activity in
which the majority of compounds possessing that activity have
been shown to be effective in the treatment of that condition. In
the instant case, Appellant has provided none of these.
Consequently, Appellant has failed to demonstrate possession
of the claimed method as of the earliest effective filing date of
the instant application.
Id. at 4–5.
Analysis
Having carefully considered the evidence and arguments presented by
Appellant and the Examiner, we find that the preponderance of the evidence
does not support the Examiner’s determination that Appellant’s claims fail
to comply with the written description requirement.
The written description requirement “is about whether the skilled
reader of the patent disclosure can recognize that what was claimed
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corresponds to what was described; it is not about whether the patentee has
proven to the skilled reader that the invention works, or how to make it
work, which is an enablement issue.” Alcon Research Ltd. v. Barr Labs.,
Inc., 745 F.3d 1180, 1191 (Fed. Cir. 2014) (emphasis added).
Thus, contrary to the Examiner’s contention that it was necessary for
the Specification to provide a working example or evidence of an actual
reduction to practice (see Ans. 4–5), our reviewing court has stated expressly
that “[t]here is no requirement that the disclosure contain either examples or
an actual reduction to practice.” Id. at 1190 (internal citations and
quotations omitted).
“[R]ather, the critical inquiry is whether the patentee has provided a
description that in a definite way identifies the claimed invention in
sufficient detail that a person of ordinary skill would understand that the
inventor was in possession of it at the time of filing.” Id. at 1190–91
(internal citations and quotations omitted).
In the present case, each of Appellant’s independent claims 10, 23,
and 24 recites a process of treating ALS by administering a polynucleotide
to a human patient. See Appeal Br. 35–36. Claim 24 expressly recites
administering the plasmid pCK-HGFX7 to the patient, and it is undisputed
that the administered polynucleotide recited in claims 10 and 23
encompasses pCK-HGFX7. See id.
As noted in the introductory summary above, the Specification
expressly discloses that Appellant’s invention is treating ALS, that treating
ALS encompasses merely suppressing the progress of the disease, that the
therapeutic agent may be a polynucleotide encoding a full length HGF in
combination with a deleted variant HGF, and that the therapeutic
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composition is preferably administered intramuscularly, spinally,
intrathecally, intraventricularly, parenchymally, or intracranially. See Spec.
3–5, 9, and 17.
As also noted above, the Specification states expressly that pCK-
HGFX7 is the preferred polynucleotide for treating ALS, that pCK-HGFX7
exhibits neuron-protecting and growth-promoting properties in vitro
including in cells subjected to oxidative stress, as well as in cells engineered
to express the G93A SOD1 mutation, a mutation known to be one cause of
ALS. See id. at 24–30. And, the Specification discloses that intramuscular
pCK-HGFX7 administration to a widely used mouse model of ALS having
the G93A SOD1 mutation results in a significant improvement in grip
strength, as compared to control plasmids. See id. at 30–32; Fig. 8.
Thus, to summarize, the Specification states expressly that
Appellant’s invention is treating ALS, the Specification describes a specific
polynucleotide to be used in that treatment as well as specific administration
modes, and the Specification substantiates the inventive treatment method
through relevant in vitro tests as well as in an animal model undisputedly in
wide usage. Given these disclosures, we find that the Specification
identifies the claimed ALS treatment methods in a definite way, and in
sufficient detail, such that a skilled artisan would understand that the
inventor possessed the claimed methods at the time of filing. See Alcon v.
Barr, 745 F.3d at 1190–91. We are not persuaded, therefore, that the
Specification fails to provide adequate descriptive support for the claimed
ALS treatments.
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The Examiner contends that, in relation to diseases like ALS with few
art-recognized treatments, the written description requirement mandates a
higher level of disclosure:
In a situation in which Appellant is claiming a method of
treating a disease for which essentially no treatment has thus far
proven effective, the courts have held that “[A] reduction to
practice does not occur until the inventor has determined that
the invention will work for its intended purpose” (Estee Lauder
Inc. v. L’Oreal, S.A., 129 F.3d 588, 593, 44 USPQ2d 1610,
1614 (Fed. Cir. 1997), and that “determining that the invention
will work for its intended purpose may require testing
depending on the character of the invention and the problem it
solves” (Mahurkar[] v. C.R. Bard, Inc., 79 F.3d 1572, 1578, 38
USPQ2d 1288, 1291 (Fed. Cir. 1996).
Ans. 4.
We are not persuaded. Contrary to the Examiner’s contention, neither
Estee Lauder nor Markuhar involved claims directed to treatments of
diseases for which previous therapies had proven ineffective. Rather, Estee
Lauder was an appeal from an interference proceeding, and involved claims
to a composition for protecting skin from UV damage. See 129 F.3d at
589–90. And in Markuhar, the claim at issue was directed to a double
lumen catheter. See 79 F.3d at 1575.
In addition, neither Estee Lauder nor Mahurkar involved the issue of
whether, under § 112, the claims at issue had adequate descriptive support in
their respective specifications. Rather, both cases involved the issue of
whether the inventors had shown evidence of reduction to practice sufficient
to establish the priority of the claims at issue. See Estee Lauder, 129 F.3d at
592 (“The primary question before us is whether Estee Lauder established
by a preponderance of the evidence that it successfully reduced its invention
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to practice before the April 13, 1987, critical date. If it did, then it is entitled
to priority.); Markuhar, 79 F.3d at 1578 (“To show actual reduction to
practice, an inventor must demonstrate that the invention is suitable for its
intended purpose.”).
As noted above, when evaluating claims for compliance with the
written description requirement “[t]here is no requirement that the disclosure
contain either examples or an actual reduction to practice.” Alcon v. Barr,
745 F.3d at 1190 (internal and citations quotations omitted). We are not
persuaded, therefore, that the present case is governed by Estee Lauder or
Markuhar, or the language in those cases cited by the Examiner.
We acknowledge our reviewing court’s recent cautionary statement
that, “[b]ased on the specific facts of certain cases, it is unnecessary to prove
that a claimed pharmaceutical compound actually achieves a certain result.
But when the inventor expressly claims that result, our case law provides
that that result must be supported by adequate disclosure in the
specification.” Nuvo Pharms. (Ireland) Designated Activity Co. v. Dr.
Reddy’s Labs. Inc., 923 F.3d 1368, 1385 (Fed. Cir. 2019) (emphasis added).
We find the present situation to be distinguishable from Nuvo. In
Nuvo, the specification at issue failed to provide any disclosure supporting a
rationale that explained why the claimed combination of therapeutic
ingredients would be beneficial to patients. See id. at 1381. Here, in
contrast, the Specification states expressly that Appellant’s invention is
treating ALS, the Specification describes a specific polynucleotide to be
used in that treatment as well as specific administration modes, and the
Specification substantiates the inventive treatment method through relevant
in vitro tests as well as in an animal model undisputedly in wide usage.
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In sum, for the reasons discussed, we find that, on balance, the
preponderance of the evidence does not support the Examiner’s finding that
Appellant’s claims lack sufficient descriptive support in the Specification.
We therefore reverse the Examiner’s rejection of claims 10 and 15–24 for
failure to comply with the written description requirement.

ENABLEMENT
The Examiner’s Prima Facie Case
In rejecting claims 10 and 15–24 for failure to comply with the
enablement requirement, the Examiner finds that, although a person skilled
in neurobiology “could readily administer a polynucleotide encoding two or
more isoforms of HGF to a human afflicted with ALS, the evidence of
record supports the conclusion that the artisan has no expectation that such
administration would be beneficial in the least to the subject being treated.”
Ans. 5.
In other words, the Examiner contends, “one of skill has no
reasonable expectation that the claimed method is operable.” Id.; see also
id. at 6 (“The text on pages 9 to 18 of the instant [S]pecification clearly fails
to supply the guidance that would be needed by a routine practitioner to
reasonably predict the operability of a particular embodiment and constitute
nothing more than an invitation to experiment.”).
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The Examiner cited DiBernardo3 and Benatar4 as evidence that a
skilled artisan would not have considered the experimental results using the
SOD1 mouse model in Example 6 of the Specification, discussed above, to
be predictive of whether ALS could be treated by administering a
polynucleotide, such as pCK-HGFX7, encoding two isoforms of HGF. Id.
at 7–8.
In addition to finding that a skilled artisan would have considered the
Specification insufficient to establish the operability of the claimed ALS
treatments, the Examiner contends that the Specification fails to disclose “an
effective route, duration and quantity of administration of the therapeutic
polynucleotides employed therein to a subject.” Id. at 6. Therefore, the
Examiner determines, a skilled artisan would have to undertake “a
substantial amount of undue experimentation involving the variation in the
amount and duration of administration of a polynucleotide of the instant
invention and in determining a suitable route of administration for the
treatment of ALS in a human subject, with no actual expectation of success.”
Id. (citing In re Colianni, 561 F.2d 220 (CCPA 1977)).
Summarizing the rejection, the Examiner contends that “[t]he instant
specification is not enabling for a method of treating ALS in a human
subject because one can not follow the guidance presented therein and
practice the claimed method with any reasonable expectation of success

3 Allitia B. DiBernardo & Merit E. Cudkowicz, Translating preclinical
insights into effective human trials in ALS, 1762 BIOCHIMICA ET BIOPHYSICA
ACTA 1139–49 (2006).
4 Michael Benatar, Lost in translation: Treatment trials in the SOD1 mouse
and in human ALS, 26 NEUROBIOLOGY OF DISEASE 1–13 (2007).
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without the need for further substantial inventive contribution, if at all.” Id.
at 9.
Analysis
Having carefully considered the evidence and arguments presented by
Appellant and the Examiner, we conclude that, on balance, the
preponderance of the evidence does not support the Examiner’s
determination that Appellant’s claims fail to comply with the enablement
requirement.
We acknowledge that a mere plan or “germ of an idea” is insufficient
to enable claims directed to methods of treating specific disorders. In re
’318 Patent Infringement Litig., 583 F.3d 1317, 1324 (Fed. Cir. 2009)
(affirming judgment of non-enablement of claims to treating Alzheimer’s
disease where disclosure lacked in vitro test results and only proposed, but
did not provide animal test results).
In the present case, however, the Specification, as discussed above,
states expressly that Appellant’s invention is treating ALS, the Specification
describes a specific polynucleotide to be used in that treatment as well as
specific administration modes, and the Specification substantiates the
inventive treatment method through relevant in vitro tests as well as in an
animal model undisputedly in wide usage.
It is well settled that when making an enablement rejection, “it is
incumbent upon the Patent Office . . . to explain why it doubts the truth or
accuracy of any statement in a supporting disclosure and to back up
assertions of its own with acceptable evidence or reasoning which is
inconsistent with the contested statement.” In re Marzocchi, 439 F.2d 220,
224 (CCPA 1971). In that regard, both the DiBernardo and Benatar
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publications cited by the Examiner include disclosures suggesting that a
skilled artisan would have understood the G93A mutant SOD1 mouse model
used in Example 6 of the Specification not to be fully reliably in predicting
whether a tested agent would be useful for treating ALS.
Specifically, DiBernardo discloses that the drug riluzole, “first
demonstrated to be effective in humans was later shown to be effective in
the SOD1G93A mouse model,” whereas in contrast “a number of
compounds found to be effective in this model failed in humans, including
vitamin E, gabapentin, topiramate, celecoxib, and creatine.” DiBernardo
1142 (citations omitted).
Benatar discloses:
The most popular animal model of amyotrophic lateral sclerosis
(ALS) is the mouse that has been genetically engineered to
express a mutant form of the human superoxide dismutase
(SOD1) gene. The most commonly used SOD1 mouse harbors
the glycine to alanine mutation at amino acid 93 (G93A). Since
the identification of mutations in the SOD1 gene as an
important cause of familial ALS . . . and the generation of the
SOD1 mouse model of ALS several years later . . . , there have
been dozens of studies of a panoply of therapeutic agents in
SOD1 mice. The choice of therapeutic agents in many clinical
trials of human ALS . . . has been predicated, at least in part, on
the efficacy of these drugs when studied in the SOD1 mouse
. . . . Nevertheless, success in human clinical trials has been
extremely limited, calling into question the utility of such
preclinical data for identifying therapeutic agents that are
worthy of further study in humans.
Benatar 1 (citations omitted).
Our reviewing court has explained, however, that when considering
enablement in relation to methods of treating human disorders, “a rigorous
correlation is not necessary where the disclosure of pharmacological activity
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is reasonable based upon the probative evidence.” ’318 Patent Infringement
Litig., 583 F.3d at 1325 (citing Cross v. Iizuka, 753 F.2d 1040, 1050 (Fed.
Cir. 1985)).
In the present case, although DiBernardo teaches that at least five
pharmaceutical agents effective in the SOD1 mutant mouse model were not
successful in treating ALS in humans, DiBernardo nonetheless also discloses
that the drug riluzole was found to be efficacious in both humans and the
SOD1 G93A mutant mouse model. See DiBernardo 1142. Moreover,
DiBernardo describes that animal model as “a powerful tool for dissecting
the biology of motor neuron disease” despite the fact that a number of agents
effective in the SOD1 mutant mouse were not useful for treating ALS. Id.;
see also id. at 1143 (“Despite its track record of poor predictive ability, the
transgenic mouse model remains a critically important tool both to unravel
the complex stages of motor neuron disease, and to pursue new therapeutic
approaches.”) (emphasis added).
Benatar, despite its teachings questioning the predictive value of the
SOD1 mutant mouse, nonetheless describes the SOD1 mutant mouse as a
widely used and important benchmark for determining whether to undertake
clinical trials in humans. See Benatar 1 (describing the SOD1 mutant mouse
as the “most popular animal model of amyotrophic lateral sclerosis (ALS)”
and the G93A SOD1 mutant mouse as the “most commonly used SOD1
mouse”); see id. (“The choice of therapeutic agents in many clinical trials of
human ALS . . . has been predicated, at least in part, on the efficacy of these
drugs when studied in the SOD1 mouse.”) (citations omitted).
Thus, while DiBernardo and Benatar teach that efficacy in the SOD1
mutant mouse might not be rigorously correlated to effective ALS
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treatments in humans, DiBernardo and Benatar nonetheless also describe the
SOD1 mutant mouse as being in widespread use among skilled artisans, as
being an important tool for researching ALS treatments, and as being an
animal model upon which many clinical trials (i.e., administration of ALS
treatments to humans) were predicated. In other words, while DiBernardo
and Benatar provide evidence questioning the ability of the SOD1 mutant
mouse to absolutely predict efficacy against ALS in humans, those
publications nonetheless also provide countervailing evidence supporting the
premise argued by Appellant—that, overall, the state of the art was that
skilled artisans considered the SOD1 mutant mouse model reasonably
predictive of successful ALS treatments in humans.
The Kessler Declaration5 provides additional evidence that skilled
artisans considered the SOD1 mutant mouse model reasonably predictive of
successful ALS treatments in humans. Consistent with DiBernardo and
Benatar, Dr. Kessler testifies that the “transgenic SOD mouse model is the
animal model that has been most commonly used and is most widely
accepted in the field of ALS drug testing and development” and that the
SOD mouse model “was used as a model for testing therapeutic effects of
both ALS drugs now approved by FDA, Rilutek (riluzole) and Radicava
(edaravone).” Kessler Decl. ¶ 14 (emphasis added).
Dr. Kessler testifies, moreover, that no animal model is absolutely
predictive of successful human treatments. See id. ¶ 13 (“In no disease with
which I am familiar is there a perfect in vitro or animal model, one in which

5 Declaration of John A. Kessler, MD, under 37 C.F.R. § 1.132 (signed July
26, 2017; “Kessler Decl.”).
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therapeutic success in the in vitro or animal model invariably translates into
an effective therapy for human patients.”).
In sum, we agree with the Examiner that DiBernardo and Benatar
provide evidence calling into question the predictive value of the animal
model used in Appellant’s Specification. Nonetheless, DiBernardo and
Benatar also provide evidence that, overall, the state of the art was that
skilled artisans considered the SOD1 mutant mouse model reasonably
predictive of successful ALS treatments in humans, a premise squarely
supported by the Kessler Declaration. And, as noted above, both FDA-
approved drugs for treating ALS were active in the SOD1 mutant mouse.
See id. ¶ 14. And importantly, as also noted above, “a rigorous correlation is
not necessary where the disclosure of pharmacological activity is reasonable
based upon the probative evidence.” ’318 Patent Infringement Litig., 583
F.3d at 1325 (citing Cross v. Iizuka, 753 F.2d at 1050).
Accordingly, on balance, we are not persuaded that the preponderance
of the evidence supports the Examiner’s conclusion that the Specification
fails to enable the claimed methods of treating ALS, despite the absence in
the Specification of a working example in which a polynucleotide
encompassed by the claims is administered to humans with ALS. See ’318
Patent Infringement Litig., 583 F.3d at 1324 (“[H]uman trials are not
required for a therapeutic invention to be patentable.”); see also In re Brana,
51 F.3d 1560, 1567 (Fed. Cir. 1995) (finding human trials unnecessary in
reversing how-to-use enablement rejection). We, therefore, reverse the
Examiner’s rejection of claims 10 and 15–24 for lack of enablement.

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CONCLUSION
In summary:
Claims
Rejected
Basis Affirmed Reversed
10, 15–24 § 112(a), written
description

10, 15–24
10, 15–24 § 112(a), enablement 10, 15–24
Overall Outcome 10, 15–24

REVERSED