Chiasma plans to initiate enrollment in the new
Phase 3 clinical trial – OPTIMAL – during second half of
2017Anticipates release of top-line data from new OPTIMAL trial by
end of 2019Conference call and webcast scheduled for 5 p.m. ET
today
Chiasma, Inc. (Nasdaq:CHMA), a clinical-stage biopharmaceutical
company focused on improving the lives of patients with rare and
serious chronic diseases, today announced it has reached agreement
with the U.S. Food and Drug Administration (FDA) on the design of a
new Phase 3 clinical trial for its octreotide capsules product
candidate, conditionally trade-named Mycapssa®, for the maintenance
therapy of adult patients with acromegaly. The agreed-upon study is
designed to address the concerns previously raised in the FDA’s
Complete Response Letter (CRL) and was reached through Special
Protocol Assessment (SPA) with the FDA’s Division of Metabolism and
Endocrinology Products. The trial, referred to as “OPTIMAL”
(Octreotide capsules vs. Placebo Treatment In MultinationAL
centers), is a randomized, double-blind, placebo-controlled,
nine-month trial in 50 adult acromegaly patients (at least 20% of
whom must be recruited from the United States). OPTIMAL utilizes
levels of insulin-like growth factor, IGF-1, a byproduct of
increased growth hormone (GH) levels caused by acromegaly, as the
sole primary endpoint measure.
The Company believes the trial is adequately powered to assess
maintenance of biochemical control with octreotide capsules
compared to placebo in adult acromegaly patients who previously
demonstrated biochemical control on somatostatin receptor ligand
injections. The Company anticipates release of top-line data from
this new Phase 3 clinical trial by the end of 2019.
A Special Protocol Assessment (SPA) is a process by which an
applicant and the FDA reach an agreement on the protocol design,
endpoints and analysis of a Phase 3 clinical study prior to
initiation, in order to determine if the study adequately addresses
scientific and regulatory requirements for FDA approval.
Octreotide capsules are an investigational new oral drug
proposed for the maintenance therapy of adult patients with
acromegaly. Acromegaly is most commonly caused by a benign tumor of
the pituitary gland that produces excess GH, ultimately leading to
significant health problems and early death if untreated. GH
regulates multiple metabolic processes and stimulates the
production of IGF-1 in the liver, which stimulates the growth of
bones and other tissues. If approved, octreotide capsules may be
the first oral somatostatin analog treatment option available for
acromegaly patients, where the current standard of care is
somatostatin analog injections.
“The agreement with the FDA on a clinical path forward is an
important step toward advancing octreotide capsules as a
maintenance treatment for adult acromegaly patients treated with
the approved injections,” said Mark Fitzpatrick, president and CEO
of Chiasma. “The Special Protocol Assessment indicates the agency’s
agreement that our planned new Phase 3 clinical trial is
appropriately designed to form the primary basis of an efficacy
claim. It is a critical milestone in the continued development of
octreotide capsules and provides regulatory clarity to enable us to
resubmit our New Drug Application (NDA) if the trial’s primary
endpoint is achieved. We appreciate the FDA’s guidance throughout
this past year as we worked through multiple strategies to
determine a viable development and regulatory path forward for
octreotide capsules.
“Our cash and investment balance was $80.1 million at June 30,
2017. Based on our current plans, we expect to have a cash and
investment balance of at least $60 million at the end of 2017. In
addition, we expect that our existing cash and investments will be
sufficient to fund our operations through our anticipated release
of top-line data from this new Phase 3 clinical trial by the end of
2019 and to support in parallel our MPOWERED trial,” Fitzpatrick
continued.
The Company continues to enroll patients in its international
Phase 3 MPOWERED clinical trial of octreotide capsules for the
maintenance treatment of adult patients with acromegaly to
potentially support regulatory approval in Europe. However, in
order to support enrollment rates for the OPTIMAL Phase 3 clinical
trial, Chiasma has significantly reduced the number of sites
enrolling patients in the MPOWERED trial. Chiasma has preserved
more than two-thirds of the clinical sites previously designated as
MPOWERED investigative sites for exclusive enrollment in the
OPTIMAL trial. Chiasma now expects to have top-line data from the
MPOWERED Phase 3 clinical trial in 2020.
About the OPTIMAL Phase 3 Trial
Chiasma plans to conduct a randomized, double-blind,
placebo-controlled, nine-month clinical trial in 50 adult
acromegaly patients (at least 20% of whom must be recruited from
the United States) whose disease is biochemically controlled, based
upon levels of IGF-1, a byproduct of increased GH levels caused by
acromegaly, on injectable somatostatin analogs at baseline (average
IGF-1 ≤1.0 x upper limit of normal (ULN)). The patients must also
have confirmed active acromegaly following their last surgical
intervention based upon an elevated IGF-1 at that time of >1.3 x
ULN. The trial will be randomized on a 1:1 basis to octreotide
capsules or placebo. Patients will be dose titrated from 40mg per
day to up to a maximum of 80mg per day, equaling two capsules in
the morning and two capsules in the evening. Patients meeting
predefined biochemical failure criteria during the course of the
trial will revert to their original treatment of injections and
will be monitored for the remainder of the trial. The primary
endpoint of the study is the proportion of patients who maintain
their biochemical response compared to placebo at the end of the
nine-month, double-blind, placebo-controlled period as measured
using the average of the last two IGF-1 levels ≤ 1.0 x ULN.
Hierarchical secondary endpoints that will be considered by the FDA
in evaluating the totality of evidence for octreotide capsules
treatment effect include:
- Proportion of patients who maintain GH response at week 36,
compared to screening;
- Time to loss of response of IGF-1 > 1×ULN;
- Time to loss of response of IGF-1 > 1.3×ULN;
- Change from screening to end of treatment in mean GH; and
- Change in IGF-1, from baseline to end of treatment.
The FDA required that the last two secondary endpoints be
analyzed by comparing the octreotide capsules treatment arm to the
placebo treatment arm at the end of the nine-month, double-blind,
placebo-controlled phase, including those patients that have been
rescued by injectable somatostatin analogs. We estimate that as
many as 90% or more of the placebo-treated patients may require
injectable somatostatin analog rescue therapy. The trial design
also includes as exploratory endpoints the last observed
biochemical values (measuring both GH and IGF-1) prior to rescue by
injectable somatostatin analogs.
Chiasma has completed the manufacturing of the necessary
clinical trial material, including placebo capsules, to initiate
the OPTIMAL trial. The Company expects to begin enrolling patients
in the new Phase 3 trial during the second half of 2017.
Conference Call InformationChiasma will conduct
an investor conference call to discuss the Special Protocol
Assessment, the OPTIMAL clinical trial and the development and
regulatory paths forward for octreotide capsules at 5:00 p.m. ET
today. A live webcast of the call will be available in the "News
& Investors" section of www.chiasma.com. The call also may be
accessed by dialing (877) 604-1612 or (201) 389-0883. A webcast
replay will be available following the call.
About Chiasma Chiasma is focused on improving
the lives of patients who face challenges associated with their
existing treatments for rare and serious chronic diseases.
Employing its Transient Permeability Enhancer (TPE®) technology
platform, Chiasma seeks to develop oral medications that are
currently available only as injections. The Company has reached
agreement with the U.S. Food and Drug Administration (FDA) on the
design of a new Phase 3 clinical trial for its octreotide capsules
product candidate, conditionally trade-named Mycapssa®, for the
maintenance therapy of adult patients with acromegaly. Chiasma is
headquartered in the United States with a wholly owned subsidiary
in Israel. Mycapssa and TPE are registered trademarks of
Chiasma.
Forward-Looking Statements This release
contains “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995, including, but
not limited to, statements regarding the Company’s commitment to
develop new treatment options for patients with rare and serious
chronic diseases, specifically acromegaly, the Company’s efforts to
potentially obtain regulatory approval in the United States by
conducting the new Phase 3 OPTIMAL clinical trial under a Special
Protocol Assessment, the Company’s efforts to potentially obtain
regulatory approval in Europe by conducting the ongoing MPOWERED
Phase 3 clinical trial, the Company’s ability to successfully
manufacture clinical trial material to enable the enrollment of
patients in the OPTIMAL trial in the second half of 2017, the
timing of receipt of top-line data and submission of regulatory
filings, including the Company’s ability to obtain top-line data
from the OPTIMAL trial by the end of 2019 and the Company’s ability
to obtain top-line data from the MPOWERED trial in 2020, and the
Company’s cash forecasts, including its expected cash and
investment balances as of the end of 2017 and the expectation that
it has sufficient existing cash and investments on hand to fund its
operations through its anticipated release of top-line data from
the new Phase 3 OPTIMAL clinical trial by the end of 2019 and to
support the MPOWERED trial in parallel. Any forward-looking
statements in this press release are based on management’s current
expectations of future events and are subject to a number of risks
and uncertainties that could cause actual results to differ
materially and adversely from those set forth in or implied by such
forward-looking statements. These risks and uncertainties
include, but are not limited to, risks associated with the
regulatory review and approval process generally; risks associated
with Chiasma’s Phase 3 clinical trial to support regulatory
approval of octreotide capsules in the E.U.; risks associated with
Chiasma conducting an additional randomized, double-blind and
controlled Phase 3 clinical trial to support regulatory approval of
octreotide capsules in the United States, including risks
related to the enrollment, timing and associated expenses; risks
associated with Special Protocol Assessment agreements, including
the risk that Special Protocol Assessment agreements are not a
guarantee of approval and the FDA may not approve octreotide
capsules even if the Phase 3 trial is successful; risks associated
with the ability of the Company’s suppliers to pass future
regulatory inspections; risks associated with obtaining,
maintaining and protecting intellectual property; risks associated
with Chiasma’s ability to enforce its patents against infringers
and defend its patent portfolio against challenges from third
parties; the risk that octreotide capsules, if approved, will not
be successfully commercialized; the risk of competition from
currently approved therapies and from other companies developing
products for similar uses; risks associated with Chiasma’s
financial position, including its ability to manage operating
expenses and/or obtain additional funding to support its business
activities; risks associated with Chiasma’s dependence on third
parties; and risks associated with defending any litigation,
including the risk that we incur more costs than we expect and
uncertainty involving the outcome. For a discussion of these
and other risks and uncertainties, and other important factors, any
of which could cause our actual results to differ from those
contained in the forward-looking statements, see the section
entitled “Risk Factors” in Chiasma’s Quarterly Report on Form 10-Q
for the quarter ended June 30, 2017 filed with the Securities and
Exchange Commission (SEC) on August 10, 2017, and in subsequent
filings with the Securities and Exchange Commission. All
information in this press release is as of the date of the release,
and Chiasma undertakes no duty to update this information unless
required by law.
Contact:
Andrew Blazier
Sharon Merrill Associates
(617) 542-5300
chma@investorrelations.com
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