Preliminary Data from PTI-428 + Orkambi 28 Day
Clinical Study on Track for Q4 2017 Initial Data from PTI-801
+ Orkambi 14 Day Clinical Trial Anticipated in Q4 2017 Initial
Data from PTI-808 SAD and MAD HV 7 day Study Anticipated in Q4
2017 Initial Data from PTI-808 + PTI-801 + PTI-428
coadministration HV 7 day Study Anticipated in Q4 2017 Dual
Combination CF Clinical Study of PTI-801 + PTI-808 Expected to
Initiate in Q4 2017 Preliminary Data from PTI-428 + Kalydeco
14 Day Clinical Study Anticipated in Q1 2018 Triple
Combination CF Study of PTI-428 + PTI-801 + PTI-808 Planned to
Initiate in 1H 2018
Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage
biopharmaceutical company developing small molecule therapeutics to
treat cystic fibrosis (CF) and other diseases caused by
dysfunctional protein processing, today announced third quarter
2017 results and provided an update on the Company’s product
candidates.
“Next generation CF therapies based on combined
use of CFTR modulators are fundamental to raising the bar of
disease targeting treatment. The team at Proteostasis has worked
determinedly to deliver three truly novel, differentiated CFTR
modulators to the clinic, generating data on safety, tolerability,
and PK of their combined use,” said Meenu Chhabra, President
and CEO of Proteostasis Therapeutics. “Our portfolio of CFTR
modulators includes PTI-801, a new generation corrector, PTI-428, a
novel class of CFTR modulator termed amplifier, and PTI-808, a
novel potentiator. Our CFTR modulators have shown synergy in
vitro with other known and investigational CFTR modulators,
creating a multiplicity of possible development pathways, including
add-on therapies to marketed CFTR modulators and proprietary double
and triple combinations. Notably, in vitro data in patient cells
showed that the combination of PTI-801 and PTI-808 restored CFTR
protein activity to levels beyond those observed in cells from
healthy carriers. Given the potential therapeutic benefit of
multiple combination options and our commitment to next generation
modulators, our combination development plan includes a PTI-801 and
PTI-808 doublet and a PTI-808, PTI-801, and PTI-428 triplet.
This effort enables us to both diversify our combination strategy
and understand the value potential of each component within our
portfolio. We look forward to initiating the dual combination
study in CF patients in the fourth quarter of 2017, and a triple
combination study in first half of 2018.”
PTI-428 - Cystic Fibrosis Transmembrane Conductance
Regulator (CFTR) Amplifier
Proteostasis is conducting a 28-day Phase 2
clinical trial for PTI-428, its CFTR amplifier, which is dosing CF
patients who receive PTI-428 or placebo for 28 days in addition to
Orkambi® as their background therapy. The Company has completed
enrollment and expects preliminary data from this cohort in the
fourth quarter of 2017. The Company is also conducting a 14 day
study of patients on Kalydeco® to receive PTI-428 or placebo for 14
days. The study has commenced dosing and Proteostasis currently
intends to report preliminary data in the first quarter of
2018.
PTI-801 – New Generation CFTR Corrector;
PTI-808 – Novel CFTR Potentiator
PTI-801 is a new generation CFTR corrector that
was internally discovered in a high-throughput screen based on its
demonstrated in vitro enhancement of CFTR function and synergy with
CFTR amplifiers. The Company is conducting a 14-day Phase 1 study
of PTI-801 and is currently enrolling and dosing CF patients on
Orkambi® background therapy. Proteostasis expects to report initial
data from this study in the fourth quarter of 2017.
Proteostasis is also conducting a Phase 1 study
of PTI-808, its CFTR potentiator. Proteostasis expects to report
initial safety, tolerability and PK data in healthy volunteers in
the fourth quarter of 2017.
PTI Combination studies in Healthy
Volunteer and CF Patients
Proteostasis is now enrolling healthy volunteers
in safety, tolerability and PK studies in dual and triple
combinations of its proprietary CFTR modulators, with preliminary
data expected in the fourth quarter of 2017. The first
combination study in CF patients will investigate the
coadministration of PTI-801 and PTI-808 and is planned to start in
the fourth quarter of 2017, to be followed by the initiation of a
triple combination study of PTI-808, PTI-801, and PTI-428 in the
first half of 2018.
Third Quarter 2017 Financial Results
Three Months Ended September 30, 2017
Revenue was $1.6 million for the three months
ended September 30, 2017, compared to $1.7 million for the three
months ended September 30, 2016. The decrease of $0.1 million is
the result of a reduction of $0.7 million of revenue recognized
under the Company’s collaboration with Biogen, which terminated in
the fourth quarter of 2016. This decrease was partially offset by
an increase of $0.6 million of revenue recognized under the
Company’s agreement with Astellas in the three months ended
September 30, 2017, which is primarily a result of payments under
the agreement being recognized as revenue over the research term,
with a cumulative catch-up for the elapsed portion of the research
term.
Research and development expenses were $12.9
million for the three months ended September 30, 2017, compared to
$9.2 million for the three months ended September 30, 2016. The
increase of $3.7 million was primarily due to an increase of $1.4
million in activities supporting our CF program as we continue to
advance our candidates in the clinic. During the three months ended
September 30, 2017, costs incurred on PTI-801 and PTI-808 accounted
for a combined increase of $5.4 million, or $2.7 million each,
largely related to increased manufacturing, preclinical and
clinical trial expenses as we transition to Phase 1 clinical
trials. The increase was partially offset by a $3.3 million
decrease in spending on PTI-428 as compared to the same period last
year. Additionally, there was an increase of $1.2 million in
personnel-related costs during the three months ended September 30,
2017, as compared to September 30, 2016, including $0.3 million in
employee stock-based compensation expense, primarily driven by an
overall increase in headcount to support the development of our CF
product pipeline.
General and administrative expenses were $2.7
million for the three months ended September 30, 2017, compared to
$3.3 million for the three months ended September 30, 2016. The
decrease of $0.6 million in general and administrative expenses was
primarily due to a decrease of $0.4 million in professional fees
and a $0.4 million decrease in facility costs, partially offset by
an increase of $0.3 million in personnel-related costs, including
$0.2 million of employee stock-based compensation.
Net loss was $14.0 million for the three months
ended September 30, 2017, compared to $10.8 million for the three
months ended September 30, 2016.
Nine Months Ended September 30, 2017
Revenue was $3.7 million for the nine months
ended September 30, 2017, compared to $4.3 million for the three
months ended September 30, 2016. The decrease of $0.6 million is
the result of a reduction of $2.1 million of revenue recognized
under the Biogen collaboration that terminated in the fourth
quarter of 2016. This decrease was partially offset by an increase
of $1.5 million of revenue recognized under the Astellas agreement.
Payments under the agreement are recognized as revenue over the
research term, with a cumulative catch-up for the elapsed research
term being recognized at the time any such payments are earned.
Research and development expenses were $41.4
million for the nine months ended September 30, 2017, compared to
$23.5 million for the nine months ended September 30, 2016. The
increase of $17.9 million was primarily due to an increase of $10.9
million in activities supporting our CF program, primarily related
to the continued advancement of our Phase 1 clinical trials for
PTI-801 and PTI-808 which accounted for a combined $11.8 million
increase due to manufacturing, preclinical and clinical trial
expenses as we prepared and advanced these candidates into their
Phase 1 clinical trials during the nine months ended September 30,
2017. Costs incurred on PTI-428 decreased by $1.9 million during
the nine months ended September 30, 2017. Additionally, there was
an increase of $4.1 million in personnel-related costs, including
$0.7 million in employee stock-based compensation expense,
primarily driven by an overall increase in headcount to support the
development of our CF product pipeline.
General and administrative expenses were $8.8
million for the nine months ended September 30, 2017, compared to
$8.7 million for the nine months ended September 30, 2016.
Net loss was $46.0 million for the nine months
ended September 30, 2017, compared to $27.8 million for the nine
months ended September 30, 2016. Cash, cash equivalents, and
short-term investments were $43.2 million as of September 30,
2017. Based on the Company’s current operating plan, the
Company expects its cash, cash equivalents, and short-term
investments will be sufficient to fund its operating expenses and
capital expenditures requirements through the second quarter of
2018.
About Proteostasis Therapeutics,
Inc.
Proteostasis Therapeutics, Inc. is a clinical
stage biopharmaceutical company developing small molecule
therapeutics to treat cystic fibrosis (CF) and other diseases
caused by dysfunctional protein processing. Headquartered in
Cambridge, MA, the Proteostasis Therapeutics team focuses on
identifying therapies that restore protein function. In addition to
its multiple programs in cystic fibrosis, Proteostasis Therapeutics
has formed a collaboration with Astellas Pharma, Inc. to research
and identify therapies targeting the Unfolded Protein Response
(UPR) pathway. For more information, visit
www.proteostasis.com.
Safe Harbor
To the extent that statements in this release
are not historical facts, they are forward-looking statements
reflecting the current beliefs and expectations of management made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995. Words such as “aim,” “may,”
“will,” “expect,” “anticipate,” “estimate,” “intend,” and similar
expressions (as well as other words or expressions referencing
future events, conditions or circumstances) are intended to
identify forward-looking statements. Examples of
forward-looking statements made in this release include, without
limitation, statements regarding the expected timing of the
initiation of, patient enrollment in, data from, and the completion
of, our clinical studies and cohorts for PTI-428, PTI-801, PTI-808
and our dual and triple combination therapy candidates, and the
expected timeline for our existing cash.
Forward-looking statements made in this release involve
substantial risks and uncertainties that could cause actual results
to differ materially from those expressed or implied by the
forward-looking statements, and we, therefore cannot assure you
that our plans, intentions, expectations or strategies will be
attained or achieved. Such risks and uncertainties include,
without limitation, the possibility final or future results from
our drug candidate trials (including, without limitation, longer
duration studies) do not achieve positive results or are materially
and negatively different from or not indicative of the preliminary
results reported by the Company (noting that these results are
based on a small number of patients and small data set),
uncertainties inherent in the execution and completion of clinical
trials (including, without limitation, the possibility FDA requires
us to run cohorts sequentially or conduct additional cohorts or
pre-clinical or clinical studies), in the enrollment of CF patients
in our clinical trials, in the timing of availability of trial
data, in the results of the clinical trials, in possible adverse
events from our trials, in the actions of regulatory agencies, in
endorsement, if any, by therapeutic development arms of CF patient
advocacy groups, and those set forth in our Annual Report on Form
10-K for the year ended December 31, 2016, our Quarterly Reports on
Form 10-Q and our other SEC filings. We assume no obligation
to update or revise any forward-looking statements, whether as a
result of new information, future events or otherwise.
CONTACTS:
Investors and Media:David
PittsArgot Partners212.600.1902david@argotpartners.com
PROTEOSTASIS THERAPEUTICS,
INC.
CONDENSED BALANCE
SHEETS(In thousands, except share and per share
amounts)(Unaudited)
|
|
September 30, |
|
|
December 31, |
|
|
|
2017 |
|
|
2016 |
|
Assets |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
Cash and
cash equivalents |
|
$ |
18,192 |
|
|
$ |
18,613 |
|
Short-term investments |
|
|
25,034 |
|
|
|
66,897 |
|
Restricted cash |
|
|
294 |
|
|
|
— |
|
Accounts
receivable |
|
|
1,079 |
|
|
|
668 |
|
Prepaids
and other current assets |
|
|
1,838 |
|
|
|
4,059 |
|
Total
current assets |
|
|
46,437 |
|
|
|
90,237 |
|
Property and equipment,
net |
|
|
12,582 |
|
|
|
541 |
|
Other assets |
|
|
41 |
|
|
|
68 |
|
Restricted cash, net of
current portion |
|
|
1,656 |
|
|
|
294 |
|
Total
assets |
|
$ |
60,716 |
|
|
$ |
91,140 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
|
|
|
Accounts
payable |
|
$ |
2,535 |
|
|
$ |
2,021 |
|
Accrued
expenses |
|
|
5,855 |
|
|
|
4,328 |
|
Deferred
revenue |
|
|
1,754 |
|
|
|
2,204 |
|
Deferred
rent |
|
|
139 |
|
|
|
201 |
|
Other
liabilities |
|
|
756 |
|
|
|
— |
|
Total
current liabilities |
|
|
11,039 |
|
|
|
8,754 |
|
Deferred revenue, net
of current portion |
|
|
— |
|
|
|
752 |
|
Deferred rent, net of
current portion |
|
|
— |
|
|
|
87 |
|
Derivative
liability |
|
|
3 |
|
|
|
91 |
|
Other liabilities, net
of current portion |
|
|
11,355 |
|
|
|
— |
|
Total
liabilities |
|
|
22,397 |
|
|
|
9,684 |
|
Commitments and
contingencies |
|
|
|
|
|
|
|
|
Stockholders’
equity: |
|
|
|
|
|
|
|
|
Preferred
stock, $0.001 par value; 5,000,000 shares authorized as of
September 30, 2017 and December 31, 2016, respectively; no shares
issued and outstanding as of September 30, 2017 and December 31,
2016 |
|
|
— |
|
|
|
— |
|
Common
stock, $0.001 par value; 125,000,000 shares authorized as of
September 30, 2017 and December 31, 2016, respectively; 25,112,724
and 25,000,734 shares issued and outstanding as of September 30,
2017 and December 31, 2016, respectively |
|
|
26 |
|
|
|
26 |
|
Additional paid-in capital |
|
|
241,758 |
|
|
|
238,902 |
|
Accumulated other comprehensive loss |
|
|
(7 |
) |
|
|
(22 |
) |
Accumulated deficit |
|
|
(203,458 |
) |
|
|
(157,450 |
) |
Total
stockholders’ equity |
|
|
38,319 |
|
|
|
81,456 |
|
Total
liabilities and stockholders’ equity |
|
$ |
60,716 |
|
|
$ |
91,140 |
|
|
CONDENSED STATEMENTS OF
OPERATIONS(In thousands, except share and per
share amounts)(Unaudited)
|
|
Three Months Ended
September 30, |
|
|
Nine Months Ended
September 30, |
|
|
|
2017 |
|
|
2016 |
|
|
2017 |
|
|
2016 |
|
Revenue |
|
$ |
1,551 |
|
|
$ |
1,715 |
|
|
$ |
3,719 |
|
|
$ |
4,324 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
12,894 |
|
|
|
9,218 |
|
|
|
41,372 |
|
|
|
23,498 |
|
General
and administrative |
|
|
2,741 |
|
|
|
3,266 |
|
|
|
8,813 |
|
|
|
8,682 |
|
Total
operating expenses |
|
|
15,635 |
|
|
|
12,484 |
|
|
|
50,185 |
|
|
|
32,180 |
|
Loss from
operations |
|
|
(14,084 |
) |
|
|
(10,769 |
) |
|
|
(46,466 |
) |
|
|
(27,856 |
) |
Interest income |
|
|
155 |
|
|
|
36 |
|
|
|
515 |
|
|
|
56 |
|
Other expense, net |
|
|
(25 |
) |
|
|
(38 |
) |
|
|
(57 |
) |
|
|
(15 |
) |
Net loss |
|
|
(13,954 |
) |
|
|
(10,771 |
) |
|
|
(46,008 |
) |
|
|
(27,815 |
) |
Accruing dividends on
preferred stock |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(1,378 |
) |
Net loss attributable
to common stockholders |
|
$ |
(13,954 |
) |
|
$ |
(10,771 |
) |
|
$ |
(46,008 |
) |
|
$ |
(29,193 |
) |
Net loss per share
attributable to common stockholders—basic and diluted |
|
$ |
(0.56 |
) |
|
$ |
(0.54 |
) |
|
$ |
(1.84 |
) |
|
$ |
(1.75 |
) |
Weighted average common
shares outstanding—basic and diluted |
|
|
25,093,344 |
|
|
|
20,073,685 |
|
|
|
25,051,536 |
|
|
|
16,672,368 |
|
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