SLEIGHT OF

HAND TWIST OF
FATE*
Multiple questions arise upon review of
APLT’s Galactosemia Foundation (Jul 17-19,
2020) data presentation

Footnote: Random title. Lyrics to an all-time classic the author was humming at the time of penning this report. No relation causation implication or pun intended.

Fun game: try counting the black dots in the matrix on this cover. Now imagine if clinical data were this hard to grasp…
Legal Disclaimer
As of the date of this presentation, the Authors may have positions in the stock of the
Company/companies mentioned and stand to realize gains in the event that the stock price
declines. Following the publication, the Authors may transact in the securities of the
Company. All information contained in this report is believed to be accurate and reliable and
has been obtained from public sources. All expressions of opinion are subject to change
without notice, and the Authors are not obligated to update this report or any information
contained herein. Research reports and presentations are not investment advice or
recommendations to buy or sell any security. All investors should perform their own diligence
and reach their own conclusions. Figures sourced from publicly disclosed company material,
sellside notes and other public sources. Annotations may have been added for emphasis. This
factual analysis is meant to serve as a launchpad for critical discussion and dissection of
publicly presented data, and to attempt to uncover the truth behind the same

2
 When one is one’s own worst enemy
In what turned out to be a colossal unforced error, certain
Austrian soldiers in the field of battle attacked their fellow
men believing them to be the enemy (well before the enemy
had even arrived).
Bad decision making snowballed the issue, and mistake after
communication mistake in reacting to the available data led to
a resounding defeat when the enemy (the Ottomans)
eventually showed up 2 days later.

Whenever I mentioned this story to my

grandfather, he’d simply say “foitrottl!”

Speaking of unforced errors and mistakes and general chaos around data…

PS: IF YOU KNOW THE MULTIPLE EXISTING ISSUES WITH APLT ALREADY, JUMP TO SLIDE3 8…
 NOT our words, these are excerpts from notes from the analysts that cover
APLT!! Pretty much every time this company presents data or updates deck…
THIS IS WHAT “CONFUSION” AND “SLOPPINESS” LOOK LIKE

“Confusing”. “Sloppiness in data presentations”. “concerns

about QA/QC of a regulatory submission”.

“restated figures”. “updated baseline levels”

“unforced errors”. “QC issues”

“was supposed to be fixed?”. “far from pristine”

Citations:Cowen, “Applied Presents Incremental Updates At The Galactosemia Meeting,” dated 7/19/20.UBS, “Applied Therapeutics Inc., "Short report" – walking 4
through the issues,” dated 7/6/20.UBS, “Applied Therapeutics Inc., Another slide deck? Clarity but also some Qs,” dated 7/9/20.Barclays, “Applied Therapeutics, Inc.,
Galactosemia Foundation Presentations Offer a Reset Point into 2H Events,” dated 7/19/20
Experienced biotech investors- what do you typically think when you see a small
cap biotech having to RESTATE and CORRECT CLINICAL data
multiple times, especially a dataset this small (n<20)?

More importantly, what will the FDA think?

5
How did we go from an innocent “update” to “another slide deck?”??

From 4/22
to 7/9

Citations: UBS, “Applied Therapeutics Inc., Update to ACTION-Galactosemia presentation,” dated

4/22/20; UBS, “Applied Therapeutics Inc., Another slide deck? Clarity but also some Qs,” dated 7/9/20.
From “stands behind its data” to “corrected changes”,
“updated”, “initially calculated incorrectly”…
From 7/3 to
7/19

Citations: Applied Therapeutics Press Release “Applied Therapeutics Takes Action Against
Fraudulent “Short Report”” dated 7/3/20; Barclays, “Applied Therapeutics, Inc., Galactosemia
Foundation Presentations Offer a Reset Point into 2H Events,” dated 7/19/20.
Wait… were you under the
impression that these
“mistakes” have really been
“fixed”?
8
As of July 21, 2020… NO, they have
not.
• IN FACT, UPON CRITICAL ANALYSIS, THERE ARE MORE QUESTIONS THAN ANSWERS

• THE LOGICAL QUESTION IS, IS THERE SOMETHING SYSTEMICALLY WRONG WITH THIS
DATASET?

• We urge investors to ask these questions and to use them as a launchpad to debate why
this company has, to date, not been able to assemble a single, coherent, unblemished
investor deck without “mistakes”
• To question, at a minimum, how shareholders can have faith in their ability to
comprehend the FDA’s demands which are generally orders more complex, and at the
very least, involve presenting a much more comprehensive dataset to the agency. An
agency which by the way, is cramped for time and handing out standard review and
CRLs to even Breakthrough Therapy Designated drugs (see ACAD 7/20, HRTX ICPT 6/29)
9
QUESTION 1- OK, YOU CORRECTED + RE-
CORRECTED BASELINE. BUT LOOKS LIKE YOU
DIDN’T
The company CORRECT THE
claimed (according to their REST??!
sellside notes) that
the baseline was corrected to measure the mean of levels on
7/19/20

day -1 and day 1. Problem is, they said that on 4/22

AND THEN AGAIN on 7/19 to justify the “correction”.
Even accounting for the pbo patient who “wasn’t QCed”(UBS
note, not an 8k) (!!), it doesn’t explain why the changes in
baseline for the treatment group had to be RE-CORRECTED!!

4/22/20

Citations: UBS, “Applied Therapeutics Inc., Update to ACTION-Galactosemia presentation,” dated 4/22/20; Barclays, “Applied Therapeutics, Inc.,
Galactosemia Foundation Presentations Offer a Reset Point into 2H Events,” dated 7/19/20.
QUESTION 1- OK, YOU CORRECTED + RE-
CORRECTED BASELINE. BUT LOOKS LIKE YOU
DIDN’T CORRECT THE REST??
SO THE STARTING POINT HAS CHANGED FOR EACH INDIVIDUAL PATIENT
4/28/20

By the way, same values for 2 cohorts?!! (Yes, we know they

“fixed” these. But wait till you see what’s on the next slide)

7/18/20

So 20mg/kg COHORT baseline (we’re focusing on this dose since it has shown the “best” data in totality of plasma and urine galactitol reduction) shifted by ~100
ng/mL, and individual baselines shifted as well
Citations: Applied Therapeutics, “Galactosemia Educational Forum” deck, dated
4/28/20; Applied Therapeutics Galactosemia Foundation presentation dated
7/18/20 BUT……
QUESTION 1- OK, YOU CORRECTED + RE-
CORRECTED BASELINE. BUT LOOKS LIKE YOU
DIDN’T CORRECT
4/28/20 THE REST?? 7/18/20

BUT…… THE ABSOLUTE GALACTITOL REDUCTIONS ARE EXACTLY THE SAME

FOR 5mg/kg and 20 mg/kg TREATMENT GROUPS… SO…
(pbo changes because of addition of 2 more patients along with 40mg/kg cohort)

Citations: Applied Therapeutics, “Galactosemia Educational Symposium” deck dated 4/28/20; Applied Therapeutics, Galactosemia Foundation presentation
dated 7/18/20.
QUESTION 1- OK, YOU CORRECTED + RE-CORRECTED
BASELINE. BUT LOOKS LIKE YOU DIDN’T CORRECT
THE REST??

4/28/20 7/18/20

SO HOW CAN THE PERCENTAGE REDUCTIONS BE THE SAME????

One more time, baseline changed, cohort reduction for 5 and 20mg/kg stay the same, YET THE PERCENTAGE
REDUCTION FOR INDIVIDUAL PATIENTS REMAINS EXACTLY THE SAME! MATHEMATICALLY IMPOSSIBLE!!
PLEASE EXPLAIN??! Surely it isn’t the day 32 to day 32-34 change (why was this done?). Read on…
Citations: Applied Therapeutics, “Galactosemia Educational Symposium” deck dated 4/28/20; Applied Therapeutics, Galactosemia Foundation presentation
dated 7/18/20.
QUESTION 2- HOW CAN YOU CLAIM GALACTITOL LEVELS ARE
CONSISTENT??
So, we learned quite a bit and it's actually very encouraging that on patient “So really comprehensive understanding of what these patients
levels of galactitol are very stable and consistent at baseline. And so when look like in terms of clinical presentation of disease, what their
we looked at screening versus baseline levels of these patients in numbers issues are, how those worsen over time, as well as biochemical
are very close.” analysis of galactitol levels, potential variability between patients
– Shendelman, CEO 1/8/20 and galactitol and galactose levels. It turns out and this is
something that we had discussed in our top line data. Levels of
galactose and galactitol in these patients are actually very
consistent over time and across the population”
– Shendelman, CEO 3/11/20

PLEASE EXPLAIN HOW THIS CAN BE TERMED CONSISTENT??

HOW CAN CHANGES APPROACHING YOUR BEST DOSE’S
TREATMENT EFFECT BE DEEMED CONSISTENT??
THIS ALSO REMOVES ANY DOUBT FROM THE PREVIOUS SLIDE THAT
CHANGING THE TIMEPOINT TO DAY 32-34 VS. DAY 32 MAGICALLY
RESULTED IN EXACTLY THE SAME VALUES

Citations: Applied Therapeutics, ACTION-GALACTOSEMIA Topline Data Call, 1/8/20, transcript from Bloomberg; Applied Therapeutics, Barclays Global Healthcare
Conference, 3/11/20, transcript from Bloomberg.
QUESTION 3- IF THEY ARE NOT
CONSISTENT, WHY HAVEN’T YOU SHOWN
INDIVIDUAL PBO CURVES?
If you believe in your drug’s treatment effect, wouldn’t showing individual patient curves
only go further to fortify the strength of your data? We have seen that galactitol can vary
significantly from day to day based on your own baseline data, so why not show investors
the true placebo adjusted effect?

WHY NOT SHOW INDIVIDUAL PLACEBO CURVES WHEN WE KNOW YOU HAVE THAT DATA?
Citations: Applied Therapeutics, Galactosemia Foundation presentation dated 7/18/20.
QUESTION 4- ON THAT NOTE PLEASE EXPLAIN YOUR
COMMENTARY FROM JANUARY AROUND PBO ADJUSTED
EFFECT?
• From your topline PR (below), please explain how the plasma galactitol reduction is 45-
54% FROM BASELINE VERSUS PLACEBO? By generally accepted biotech verbiage, That
implies a placebo ADJUSTED number. Why did the PR not simply say “from baseline”?
• From the previous few slides, it appears the placebo effect is indeed non-insignificant.
After all the 5mg/kg is approximately the same as placebo. Again, why not show the
individual placebo curves?

APLT announced an offering with gross proceeds ~$143.4MM a few days after this PR
Citations: Applied Therapeutics Press Release, “Applied Therapeutics Announces Positive Topline Results of Pivotal Phase 2 ACTION-Galactosemia Study
of AT-007 in Galactosemia Patients” dated 1/8/20. Annotations added.
QUESTION 4- ON THAT NOTE PLEASE EXPLAIN YOUR
COMMENTARY FROM JANUARY AROUND PBO ADJUSTED
EFFECT?
• For reference, here is a press release and presentation excerpt from
Myokardia, which announced positive Phase 3 results. Note the use of the
word “VERSUS”.

WHAT EXACTLY IS THE TRUE PLACEBO-ADJUSTED REDUCTION FOR AT-007? WHY MUST
INVESTORS HAVE TO CALCULATE THEMSELVES USING ASSUMPTIONS WHEN YOU HAVE THE DATA IN HAND?
Citations: Myokardia, “EXPLORER-HCM Topline Supplemental Materials vF” presentation, dated 5/11/20.
QUESTION 5- WHAT IS THE RELEVANCE OF MAX
REDUCTION ?
From the time curves, it is apparent that the 20mg/kg patients on average (MAKING A BIG ASSUMPTION THAT
THE BASELINE IS CORRECTLY GRAPHED!!) start at 2500 ng/mL and at day 32 start the day at between 1800-
1900 ng/mL. Even the most generous assumption only implies a ~30% effect. And then you have to adjust for
placebo…

IS THIS CALCULATION FOR THE PLACEBO ADJUSTED REDUCTION IN THE RIGHT BALLPARK? WHY DO YOU NOT
TALK ABOUT IT IF YOU TRULY BELIEVE IT’S MEANINGFUL? IS THE FDA OK WITH THE PBO-ADJUSTED NUMBER?
Citations: Applied Therapeutics, Galactosemia Foundation presentation dated 7/18/20.
QUESTION 5b- WHAT IS THE RELEVANCE OF MAX
REDUCTION ?
According to sellside we were supposed to have seen the mean reduction in plasma galactitol. Isn’t
this a figure APLT should have handy? Why have we still not seen it after multiple iterations of
presentations?

Again, given the INTRADAY

VARIABILITY (CLEARLY EVIDENT
IN APLT’S OWN GRAPHS) ISN’T
THE MEAN REDUCTION THAT
MUCH MORE RELEVANT?

DOES THE FDA NOT CARE ABOUT MEAN PLACEBO ADJUSTED REDUCTION?

Citations: UBS, “Applied Therapeutics, Inc. “Short Report” – walking through the issues,” dated 7/6/20.
QUESTION 6- IS THERE SOMETHING WRONG WITH
YOUR ASSAY?
5 mg/kg dose on average has similar max galactitol reduction as pbo as a cohort, but the
respective error bars are SO different- tiny for 5mg/kg, WIDE for placebo

PLEASE EXPLAIN WHAT IS WRONG WITH YOUR ASSAY? HOW CAN THESE DATA BE
ACCURATE GIVEN THESE ERROR BARS? WE ARE HAVING SEASONED
BIOSTATISTICANS GO THROUGH THE “DATA” TO TRY AND ANSWER THIS OURSELVES
AND SPEAKING OF WRONG ASSAYS…
Citations: Applied Therapeutics, Galactosemia Foundation presentation dated 7/18/20.
 QUESTION 7- YOUR MOST IMPORTANT BIOMARKER IS
URINE GALACTITOL SO WHY IS IT ALL OVER THE PLACE??

IS THIS PERCENT CHANGE OR ABSOLUTE CHANGE?? THE CHART TITLE IMPLIES ONE THING, THE Y AXIS IS SOMETHING ELSE
ENTIRELY. WHY IS THIS DATA STILL PRELIMINARY?? WHAT IS GOING ON WITH PLACEBO ERROR BARS? DOES
IT HAVE SOMETHING TO DO WITH YOUR ASSAY? WHY DOES 40MG/KG LOOK SO BAD?

Citations: Applied Therapeutics, Galactosemia Foundation presentation dated 7/18/20.

QUESTION 7- YOUR MOST IMPORTANT BIOMARKER IS URINE
GALACTITOL SO WHY IS IT ALL OVER THE PLACE??
Is Plasma Enough? Galactosemia KOL Weighs In
Dr. Eva Morava-Kozicz
Q: “… Would you consider a 40% reduction in
galactitol (plasma), still clinically
meaningful?..”
A: “… Now you are asking me hard question
again because I am used to monitoring
urine excretion of galactitol so I do not
know, I cannot translate that right away ….
Probably 50% reduction or 40% reduction has
therapeutic effect … But it is hard to say
without measuring it in different tissues,
right?...”

APLT’s own KOL relies on urine

measurements and doubts the reliability
of using a single method such as plasma.

Citations: UBS call with Eva Morava-Kozicz (MD, PhD) on May, 15 2020. Dr. Morava-Kozicz presented for Applied Therapeutics at Galactosemia Educational
Symposium April 28, 2020; Applied Therapeutics, “Galactosemia Educational Symposium” deck, dated 4/28/20.
QUESTION 7- YOUR MOST IMPORTANT BIOMARKER IS URINE
GALACTITOL SO WHY IS IT ALL OVER THE PLACE??
Dr. Eva Morava-Kozicz (MD, PhD)
“…We are not measuring galactitol in clinical practice in other tissues. So, my experience in patients is urine. I cannot translate it to
brain levels ... because this is not what we are doing in clinical practice…”
“… If I can reduce urine galactitol to the half of it I am already proud of myself that I am doing a good treatment strategy…”

In a recent presentation at the UBS conference (May 18, 2020), the CEO clearly stated that levels in blood and
urine do correlate. So why does the previous slide not back this up at all?
“… Urine levels is what previously was done in the field… this is what happened more often in the literature
and so we felt that it would be important to measure that in our clinical study. It’s much more accurate to look
at galactitol levels in the blood but we did not know that before we did the experiments and saw that this was
the case. We did look at galactitol levels in blood and in urine. Levels of galactitol both in blood and in urine do
correlate, it’s just little bit technically more difficult to look in urine… it’s more annoying than taking the blood
sample, so we did it, we thought it would be important to have it … they do correlate.. but I think from this
moment on we should focus on galactitol in the blood because we saw that the accuracy was just so much
greater...”
- Shendelman, CEO 5/18/20

Citations: UBS call with Eva Morava-Kozicz (MD, PhD) on May 15 2020. Dr. Morava-Kozicz presented for Applied Therapeutics at Galactosemia Educational
Symposium April 28, 2020; Applied Therapeutics, UBS Global Virtual Healthcare Conference, 5/18/20, transcript from FactSet.
QUESTION 8- WE FINALLY GOT URINE DATA THAT YOU CAVEATED AS
“EXPLORATORY”. WHAT CAN WE EXPECT FROM THE BRAIN DATA?
According to sellside AND management commentary we were supposed to have seen
the brain, EKG, and potentially even the seizure frequency data by SIMD in April
“So, you are correct that we measured galactitol not only in the blood, which is what
we released in January. But we also are able to look at galactitol levels in the brain
by MR spectroscopy and so we took -- we did MR spectroscopy at baseline and end
of treatment on all of our patients and that's underway, that will be released in our
April presentation. We also from the safety perspective had done EKGs at baseline
in this treatment. We didn't know there would be an EKG abnormality, but we did
collect that data. So that's also under evaluation at baseline and end of treatment.”
- Shendelman, CEO 3/3/20

Source: Applied Therapeutics, Cowen conference 3/3/20, Transcript via Bloomberg

Source: Cowen, Applied Therapeutics “REPORTS Q4; AT-007 NDA FILING ON TRACK FOR H2” dated 3/13/20;
Source: Cowen, Applied Therapeutics “HIGHLIGHTS FROM DINNER WITH MANAGEMENT AT COWEN'S 40TH HEALTH CARE CONFERENCE” dated 3/3/20
Source: UBS, Applied Therapeutics “Another slide deck? Clarity but also some Qs” dated 7/9/20
QUESTION 8- WE FINALLY GOT URINE DATA THAT YOU CAVEATED AS
“EXPLORATORY”. WHAT CAN WE EXPECT FROM THE BRAIN DATA?

THIS SHOULD BE A LAYUP- IS THIS LABELED CORRECTLY? OR

IS IT ANOTHER “MISTAKE”? IS THIS A SIGN OF THINGS TO
COME WHEN (IF) WE DO GET THE BRAIN DATA?

BY THE WAY, THE FDA GUIDANCE SPECIFIES EFFECT “IN

THE TISSUE OF INTEREST”. WHY DOES APLT ITSELF
HIGHLIGHT THE CNS MANIFESTATIONS AND THEN NOT
SHOW THE BRAIN DATA? ARE WE TO SERIOUSLY BELIEVE
THE FDA DOESN’T CARE ABOUT ITS OWN GUIDANCE?

Citations: Applied Therapeutics, Galactosemia Foundation presentation dated 7/18/20.

QUESTION 8b- WE FINALLY GOT URINE DATA THAT YOU CAVEATED AS
“EXPLORATORY”. WHY DID YOU DELETE THE GALACTOSE AND GAL-1-P
DATA FROM YOUR DECK?
Yet another instance of impossibly identical placebo curves. However, if this was an “innocent mistake,” why was
this data deleted rather than corrected? This question has been left unanswered for nearly 3 months.

. . . Not to mention the missing Gal-1p

data, which had no obvious errors (at
least that we know of). Why not
present data that is clearly relevant to
investors?

Citations: Applied Therapeutics, “ACTION-GALACTOSEMIA: April 2020 Trial Results” deck dated 4/21/20.Highlighted slide has since been
deleted and not “corrected” as of the time of this writing. Annotations added.
QUESTION 9- WHAT EXACTLY HAPPENED DURING YOUR MEETING WITH
THE FDA THAT LED TO THE CHANGES IN YOUR FILING TIMELINE AND PED
TRIAL DESIGN?
The Company USED TO consistently guide to submit an NDA for AT-007 in 2H20:

March Presentation:

Barclays HC conf. 3/11/20

“And we read out positive top-line data in our Galactosemia program in January as you referenced, and we're planning
to submit an NDA in the Fall of this year.”
– Shendelman, CEO

Company PR, 4/21/20

Citations: Applied Therapeutics, Investor Presentation for March 2020; Barclays Global Healthcare Conference. Applied
Therapeutics. 03/11/2020; Applied Therapeutics Press Release from April 21, 2020
QUESTION 9- WHAT EXACTLY HAPPENED DURING YOUR MEETING WITH
THE FDA THAT LED TO THE CHANGES IN YOUR FILING TIMELINE AND PED
TRIAL DESIGN?
May Presentation – timing of NDA submission slips to “~YE 2020.”

This change seems innocent enough, until you find out that The Company met with the FDA just days
earlier.

YOU CAN’T BLAME THE CORONAVIRUS FOR EVERY DELAY, ESPECIALLY BECAUSE THERE APPEARS TO
BE MORE TO THIS PARTICULAR TIMELINE PUSH. READ ON…

Citations: Applied Therapeutics, Investor Presentation for May 2020; Applied Therapeutics “ACTION Galactosemia Phase 2 Top Topline
Results Call” 1/8/20, transcript from Bloomberg; Applied Therapeutics, UBS Global Healthcare Conference 5/18/20, transcript from FactSet.
QUESTION 9- WHAT EXACTLY HAPPENED DURING YOUR MEETING WITH
THE FDA THAT LED TO THE CHANGES IN YOUR FILING TIMELINE AND PED
TRIAL DESIGN?
Topline Results Call (1/8/20) Cowen conf. (3/3/20)
“Given the magnitude of the effect and the definitive “And so, we've had a really ongoing dialogue with the
NIS we're in a really great place. And I also just want FDA about this biomarker based guidance that we're
to highlight that in our animal model as disease, a using. We've had a very constructive dialogue with
50% reduction in galactitol produced a meaningful them all along. And so we do believe we're in a very
impact on clinical outcomes. So that's really what we strong position. We're adding some additional
were targeting and it's 50% reduction. So with this data in. So, we have a long-term extension study
data, we can immediately file for approval that's ongoing. I think that will be very helpful
and importantly begin our pediatric study” supportive data, that we'd like to include and we'd
– Shoshana Shendelman, CEO
also like to do our pediatric study prior to the
submission if at all possible and we think that will be
possible.”
– Shoshana Shendelman, CEO

Citations: Applied Therapeutics, “ACTION-Galactosemia Phase 2 Topline Results Conference Call” 1/8/20, transcript from
Bloomberg; Applied Therapeutics, Cowen Annual Health Care Conference, 3/3/20. Transcript from Bloomberg.
QUESTION 9- WHAT EXACTLY HAPPENED DURING YOUR MEETING WITH
THE FDA THAT LED TO THE CHANGES IN YOUR FILING TIMELINE AND PED
TRIAL DESIGN?
Now obviously, we've had ongoing conversations with the FDA about the pediatric study and what
that looks like, we're in a really great place in terms of design”
– Shoshana Shendelman, CEO 1/8/20
If the FDA walked The Company through the design of the Pediatric study in January, then why did it
require last minute changes to the protocol in MAY??

SO, FROM “GREAT PLACE IN TERMS OF DESIGN” TO “FEEDBACK WAS INCORPORATED” TO “MOSTLY MIRROR” THE
ADULT STUDY
Citations: Applied Therapeutics, “ACTION-Galactosemia Phase 2 Topline Results Conference Call” 1/8/20, Transcript from Bloomberg; Applied Therapeutics, Form 10Q filed
5/11/20 ; Goldman Sachs note: Applied Therapeutics (APLT): GS 41st Annual Global Healthcare Conference 2020 - Key Takeaways
QUESTION 9- WHAT EXACTLY HAPPENED DURING YOUR MEETING WITH
THE FDA THAT LED TO THE CHANGES IN YOUR FILING TIMELINE AND PED
TRIAL DESIGN?

IN JUNE YOU SAID THE PEDIATRIC STUDY WOULD LARGELY MIRROR THE ADULT STUDY. THE ABOVE IS NOT WHAT A
MIRROR LOOKS LIKE. PLEASE HELP SQUARE THIS CIRCLE. ALSO, WILL YOU PR THE NDA FILING SINCE THAT IS
STANDARD FOR ALL SMALL CAP BIOTECHS?
31
Citations: Applied Therapeutics, Galactosemia Foundation presentation dated 7/18/20 .
QUESTION 10- WHAT’S WITH ALL THE STOCK SALES?

THIS IS PRETTY SELF EXPLANATORY. (RED MARK STOCK SALES). PLEASE EXPLAIN HOW YOU CAN SAY YOU STAND
BEHIND YOUR DATA, CHANGE SAID DATA, CHALK IT UP TO “MISTAKES”, AND BE SELLING STOCK AT THE SAME TIME?

Citations: Bloomberg
SUMMARY AND CLOSING THOUGHTS
• As others before us have noted, data from this company have been riddled with errors and detached,
unexplainable conclusions that fall apart once one opens up the hood.
• We review the latest data (Galactosemia Foundation, July 17-19, 2020) that sellsiders are clamoring
to call “fixed” and “finally corrected” and “overhang removed” and find that far from being pristine, the
data call into question almost every single aspect of consequence, and question what is going on
behind the scenes as far as QA/QC is concerned, particularly with a promised NDA submission
deadline approaching. [Note the company has already publicly admitted to a sellsider, though not in
8Ks as would be appropriate, of at least one major instance of QC failure]
• We provide herein a framework for investors to better critically evaluate the prospects of this
company by highlighting the clear issues and shortfalls that have simply not been stamped out
despite multiple attempts to “correct” the data. We urge the company to provide fulsome responses
for what we believe are standard biotech investor questions if they believe in their drug and its
therapeutic effect.
• Net-net, all stakeholders will be better off separating the signal from this self-inflicted noise rather
than go the route of the Austrian army that failed to remove itself from several snowballing unforced
errors much like the current setup in this name. Above all, avoid looking like a Foitrottl!
• We look forward to this company’s next data release.

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