PTI-801 Receives FDA Fast Track Designation;
Phase 1 Study Underway
Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a biopharmaceutical
company developing small molecule therapeutics to treat diseases
caused by dysfunctional protein processing such as cystic fibrosis
(CF), today announced financial results for the fourth quarter and
year ended December 31, 2016 and provided a corporate update.
“Today, CFTR modulator triple combinations
represent the greatest promise for delivering maximum efficacy for
the vast majority of CF patients and have become the central focus
in a rapidly evolving clinical landscape,” said Meenu Chhabra,
president and chief executive officer of Proteostasis Therapeutics.
“Preclinical data suggest that Proteostasis’ proprietary
triple combination solution of an amplifier (PTI-428), a corrector
(PTI-801) and a potentiator (PTI-808) has the potential to
demonstrate best-in-class efficacy because it is the only triple
combination that addresses separate aspects of CFTR dysfunction in
an entirely orthogonal manner from protein synthesis to processing
to function. To realize this potential in an increasingly
crowded clinical environment, we are pivoting our PTI-428 program
to ex-U.S. clinical sites, initiating the clinical development of
PTI-801 in regions where Orkambi is approved and expect to file an
IND for PTI-808 in the second quarter. Our goal is to
establish efficacy for PTI-428 and PTI-801 in an Orkambi
population, and thus start the proof of concept study for our
triple combination by the end of the year.”
Corporate
Updates
PTI-428 – Fast Track-Designated CFTR
Amplifier
To date, Proteostasis has reported preliminary
safety, pharmacokinetic and exploratory biomarker data from both
single and multiple ascending dose cohorts in its healthy volunteer
trial of PTI-428. The drug was well tolerated and no drug-related
SAEs were identified. Subjects who achieved a threshold
concentration approximating EC70 had a two-fold increase in cystic
fibrosis transmembrane conductance regulator (CFTR) mRNA and
protein which returned to baseline in the follow-up period.
Based on this nasal biomarker data, preclinical
studies, and study precedent for other CFTR modulator programs, the
Company now aims to report preliminary data in the second quarter
from at least 8 CF subjects from a cohort of stable Orkambi
patients dosed with PTI-428 for 7 days followed by a 7-day
follow-up period. After wash-out, these subjects will then be
eligible to enroll in a 28-day dosing cohort. Initial data will
include safety and pharmacokinetics (PK) as well as changes in CFTR
mRNA, protein, sweat chloride and lung function as measured by
forced expiratory volume in 1 second, or FEV1. Data from the 28-day
dosing cohort is expected in the second half of 2017.
The delay in study timeline from first quarter
to second quarter stems from a highly competitive clinical
development environment in the U.S., including several recent CF
study starts for investigational agents that, unlike PTI-428, have
received Therapeutics Development Network (TDN) endorsement and
ranking. The Company believes the TDN, the development arm of
the Cystic Fibrosis Foundation Therapeutics, Inc., can play a key
role in helping access U.S. study participants. The Company has an
active IND with the U.S. Food and Drug Administration (FDA) for
PTI-428 and, following deferred endorsement, continues discussions
with the TDN on its review of PTI-428.
To ensure the continued, rapid development of
PTI-428, the Company expects to focus substantial resources toward
European clinical development. To this end, the Company has
sought and received endorsement and prioritization in Europe by the
Clinical Trial Network (CTN), which is the development network of
the European Cystic Fibrosis Society. In addition, PTI-428
has been reviewed and approved for clinical study by the Medicines
and Healthcare products Regulatory Agency (UK) and Health
Canada.
PTI-801, PTI-808 and PTI-NC-733 – Second
Generation Fast Track-Designated CFTR Corrector, Potentiator and
Triple Combination Therapy
In the first quarter of 2017, Proteostasis filed
an IND for its proprietary corrector, PTI-801, and is currently
initiating a Phase 1 healthy volunteer study to assess its safety
and PK. The Company also announced today that PTI-801 has
received fast track designation from the FDA for the treatment of
CF. Fast Track designation is designed to facilitate development
and expedite review of new therapies that address unmet medical
needs of patients with serious conditions. The designation
offers various benefits, including more frequent interaction with
the FDA, eligibility for Accelerated Approval or Priority Review,
if relevant criteria are met, and the opportunity to submit a new
drug application (NDA) on a rolling basis.
The Company plans for the Phase 1 trial, which
will also investigate PTI-801 in CF patients, to run in U.S. and
European sites and is working with both the TDN and CTN to review
the study protocol for sanctioning. Subject to favorable
results from the healthy volunteer arm, the Company expects data
from patients stable on Orkambi who have been dosed with PTI-801
for 14 days in the second half of 2017.
Subject to filing an IND for PTI-808 in the
second quarter of 2017, and positive results from its PTI-428 and
PTI-801 programs, the Company aims to initiate a trial for our
third CFTR modulator by the end of 2017. The PTI-808 Phase 1 study
is expected to include SAD and MAD cohorts in healthy volunteers as
well as co-administration of PTI-428 and PTI-801 and ascending
doses of PTI-808, for the proprietary triple combination therapy
PTI-NC-733 in CF patients homozygous for F508del who are not
receiving Orkambi. The Company believes that PTI-NC-733 could
represent a new standalone therapy for CF.
Personnel Updates
On March 8, 2017, the Board elected Brett
Hagen, the Company’s Vice President of Finance, Controller,
Assistant Treasurer and Principal Accounting Officer, to the
additional position of Principal Financial Officer. Mr.
Hagen, age 43, has been serving as our Controller since May 2016,
Principal Accounting Officer since August 2016, Assistant Treasurer
since October 2016, Vice President of Finance since February 2017
and Principal Financial Officer since March 2017. Previously,
Mr. Hagen served as the controller for BIND Therapeutics, Inc.,
Vice President, Finance/Segment Controller of Boston Private
Financial Holdings, Manager of SEC Financial Reporting and
Technical Account of The Princeton Review, Controller of
BioProcessors Corporation and held a number of positions at
PricewaterhouseCoopers LLP. He received a B.A. from University of
Minnesota, a Master in Accountancy from Wright State University and
a M.S. in Finance from Suffolk University.
Fourth Quarter & Year End
2016 Financial Results
Proteostasis reported a net loss of
approximately $37.2 million for the full year ended December 31,
2016, as compared to a net loss of $25.0 million for the prior year
ended December 31, 2015. The Company reported a net loss of $9.4
million for the three months ended December 31, 2016, as compared
to a net loss of $5.6 million for the three months ended December
31, 2015.
Research and development expenses were $34.0
million for the full year ended December 31, 2016, as compared to
$22.5 million for the prior year ended December 31, 2015. Research
and development expenses for the three months ended December 31,
2016 were $10.5 million, as compared to $5.8 million for the same
period in the prior year. The increase was due to costs incurred in
advancing the preclinical development and clinical trials of our CF
program, including support for our Phase 1 clinical trials of
PTI-428, which commenced during the first quarter of 2016.
General and administrative expenses for the full
year ended December 31, 2016 were $11.9 million, as compared to
$6.3 million for the prior year. General and administrative
expenses for the three months ended December 31, 2016 were $3.2
million, as compared to $1.8 million for the same period in the
prior year. The increase in G&A expense in these periods was
due primarily to an increase in professional fees and personnel
related costs.
Proteostasis ended the fourth quarter of 2016
with $85.5 million in cash, cash equivalents and
investments.
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
biopharmaceutical company dedicated to the discovery of
groundbreaking therapies to treat diseases caused by dysfunctional
protein processing, such as cystic fibrosis (CF). Headquartered in
Cambridge, MA, the Proteostasis Therapeutics team focuses on
identifying therapies that modulate the proteostasis imbalance in
cells and restore protein function. Proteostasis Therapeutics is
currently enrolling eligible adults with CF to participate in its
Phase 1 clinical trials of PTI-428. 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 our completion
of, our clinical studies and cohorts for PTI-428, PTI-801, PTI-808
and our triple combination therapy candidate, PTI-NC-733, the
timing of our expected IND filing for PTI-808, our cash forecast,
and discussions with and endorsement by therapeutic development
arms of CF patient advocacy groups. 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, uncertainties
inherent in the execution and completion of clinical trials
(including, without limitation, the possibility FDA requires us to
run additional cohorts sequentially), in the enrollment of CF
patients in our clinical trials, in the timing of availability of
trial data, 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, 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.
|
PROTEOSTASIS THERAPEUTICS, INC. |
BALANCE SHEETS |
(In thousands, except share and per share
amounts) |
|
|
|
December 31, |
|
|
|
2016 |
|
|
2015 |
|
Assets |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
Cash and
cash equivalents |
|
$ |
18,613 |
|
|
$ |
13,844 |
|
Short-term investments |
|
|
66,897 |
|
|
|
— |
|
Accounts
receivable |
|
|
668 |
|
|
|
918 |
|
Prepaids
and other current assets |
|
|
4,059 |
|
|
|
180 |
|
Total
current assets |
|
|
90,237 |
|
|
|
14,942 |
|
Property and equipment,
net |
|
|
541 |
|
|
|
566 |
|
Deferred offering
costs |
|
|
— |
|
|
|
2,744 |
|
Other assets |
|
|
68 |
|
|
|
144 |
|
Restricted cash |
|
|
294 |
|
|
|
294 |
|
Total
assets |
|
$ |
91,140 |
|
|
$ |
18,690 |
|
Liabilities, Convertible Preferred Stock and
Stockholders’ Equity |
|
|
|
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
|
|
|
Accounts
payable |
|
$ |
2,021 |
|
|
$ |
3,330 |
|
Accrued
expenses |
|
|
4,328 |
|
|
|
2,248 |
|
Deferred
revenue |
|
|
2,204 |
|
|
|
4,076 |
|
Deferred
rent |
|
|
201 |
|
|
|
182 |
|
Total
current liabilities |
|
|
8,754 |
|
|
|
9,836 |
|
Deferred revenue, net
of current portion |
|
|
752 |
|
|
|
4,265 |
|
Deferred rent, net of
current portion |
|
|
87 |
|
|
|
287 |
|
Preferred stock warrant
liability |
|
|
— |
|
|
|
110 |
|
Derivative
liability |
|
|
91 |
|
|
|
2 |
|
Total
liabilities |
|
|
9,684 |
|
|
|
14,500 |
|
Commitments and
contingencies (Note 16) |
|
|
|
|
|
|
|
|
Convertible preferred
stock (Series A and B), $0.001 par value; 0 and 110,057,398 shares
authorized as of December 31, 2016 and 2015, respectively; 0
and 104,854,769 shares issued and outstanding as of December
31, 2016 and 2015, respectively; aggregate liquidation
preference of $0 and $149,392, respectively, as of December
31, 2016 and 2015 |
|
|
— |
|
|
|
112,292 |
|
Total
stockholders’ equity (deficit) |
|
|
81,456 |
|
|
|
(108,102 |
) |
Total
liabilities, convertible preferred stock and stockholders’ equity
(deficit) |
|
$ |
91,140 |
|
|
$ |
18,690 |
|
PROTEOSTASIS THERAPEUTICS, INC. |
STATEMENTS OF OPERATIONS |
(In thousands, except share and per share
amounts) |
|
|
|
Three Months
Ended |
|
|
Year Ended |
|
|
|
December 31, |
|
|
December 31, |
|
|
|
2016 |
|
|
2015 |
|
|
2016 |
|
|
2015 |
|
Revenue |
|
$ |
4,060 |
|
|
$ |
1,234 |
|
|
$ |
8,384 |
|
|
$ |
4,312 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
10,461 |
|
|
|
5,787 |
|
|
|
33,959 |
|
|
|
22,524 |
|
General
and administrative |
|
|
3,198 |
|
|
|
1,769 |
|
|
|
11,880 |
|
|
|
6,322 |
|
Total
operating expenses |
|
|
13,659 |
|
|
|
7,556 |
|
|
|
45,839 |
|
|
|
28,846 |
|
Loss from
operations |
|
|
(9,599 |
) |
|
|
(6,322 |
) |
|
|
(37,455 |
) |
|
|
(24,534 |
) |
Interest income |
|
|
190 |
|
|
|
— |
|
|
|
246 |
|
|
|
— |
|
Interest expense |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(599 |
) |
Other income (expense),
net |
|
|
(8 |
) |
|
|
691 |
|
|
|
(23 |
) |
|
|
93 |
|
Net loss |
|
|
(9,417 |
) |
|
|
(5,631 |
) |
|
|
(37,232 |
) |
|
|
(25,040 |
) |
Modification of Series
A preferred stock |
|
|
— |
|
|
|
743 |
|
|
|
— |
|
|
|
11,481 |
|
Modification of Series
B preferred stock |
|
|
— |
|
|
|
(26 |
) |
|
|
— |
|
|
|
(26 |
) |
Accruing dividends on
Series A preferred stock |
|
|
— |
|
|
|
(3,026 |
) |
|
|
(1,378 |
) |
|
|
(9,724 |
) |
Net loss attributable
to common stockholders |
|
$ |
(9,417 |
) |
|
$ |
(7,940 |
) |
|
$ |
(38,610 |
) |
|
$ |
(23,309 |
) |
Net loss per share
attributable to common stockholders—basic and diluted |
|
$ |
(0.38 |
) |
|
$ |
(13.95 |
) |
|
$ |
(2.06 |
) |
|
$ |
(42.14 |
) |
Weighted average common
shares outstanding—basic and diluted |
|
|
24,975,010 |
|
|
|
569,026 |
|
|
|
18,759,369 |
|
|
|
553,162 |
|
CONTACTS:
Investors:
David Pitts
Argot Partners
212.600.1902
david@argotpartners.com
Media:
Eliza Schleifstein
Argot Partners
973.361.1546
eliza@argotpartners.com
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