Full Press Release Details
Insmed Highlights Progress Across Portfolio
and Outlines Growth Strategy at R&D Day
- Initiated Phase 1 study of treprostinil
palmitil inhalation powder (TPIP); Phase 2a study in pulmonary arterial hypertension planned for 2021 -
- Expects to initiate registrational
Phase 3 ASPEN trial of brensocatib in patients with non-cystic fibrosis bronchiectasis (NCFBE) in fourth quarter of 2020; expansion
into additional neutrophil-mediated diseases planned in 2021 -
- Expanding commercial opportunity
for ARIKAYCE with planned European launch by end of 2020 and potential Japanese launch in 2021, if approved, in refractory MAC
lung disease; expects to initiate registrational program in front-line NTM in 2020 -
- Cash runway of over three years
based on currently planned development programs -
BRIDGEWATER, N.J., September 30, 2020 /PRNewswire/ -
Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious
and rare diseases, today announced progress across its portfolio of therapies and pipeline candidates, which will be discussed
in further detail today at the Company's virtual Research & Development (R&D) Day. The event will feature presentations
from the Insmed management team and Ronald J. Oudiz, M.D., FACP, FACC, FCCP, Professor of Medicine at the David Geffen School of
Medicine at the University of California Los Angeles (UCLA), the Interim Chief, Division of Cardiology, and a Director at the Liu
Center for Pulmonary Hypertension at the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, and the lead
investigator in the Company's planned Phase 2a trial for treprostinil palmitil inhalation powder (TPIP).
"Our success in building a commercial company with a global
footprint and strong pipeline sets the stage for Insmed's next chapter of growth," said Will Lewis, Chair and Chief
Executive Officer of Insmed. "With ARIKAYCE, our global expansion efforts and upcoming study in front-line NTM patients has
the potential to establish this first-in-disease program as the standard of care in NTM lung disease worldwide. We believe brensocatib,
our DPP1 inhibitor, offers an entirely new approach to address a wide array of neutrophil-mediated diseases. We plan to build upon
our early leadership position in this space with our anticipated near-term registrational study in NCFBE, followed by a new development
program in cystic fibrosis and plans to explore other opportunities across the neutrophil-mediated disease landscape. We are also
pleased to announce the initiation of a Phase 1 trial of TPIP, which represents an exciting opportunity to more fully harness the
potential of the prostanoid pathway for the treatment of pulmonary arterial hypertension and may offer a disease-modifying effect.
We are well capitalized to execute against our strategic objectives during this next phase of growth for Insmed."
Select R&D Day Highlights
Initiated Phase 1 Trial for Treprostinil Palmitil Inhalation
The Company announced today that it has initiated dosing of
the first subjects in the Phase 1 healthy volunteer trial of TPIP in the United States. The objective of this first-in-human single
ascending dose and multiple ascending dose study is to assess the pharmacokinetics and tolerability profile of TPIP. Top-line data
are expected in the first quarter of 2021. The Company also announced plans to initiate a Phase 2a study in patients with pulmonary
arterial hypertension (PAH) in early 2021.
Today, Dr. Oudiz will provide a review of the PAH treatment
landscape and the remaining unmet medical need. Additionally, the Company will provide an update on TPIP and review key highlights
of preclinical data supporting the product candidate's potentially differentiated product profile that could prove to be
an advantageous addition to the therapeutic arsenal for the treatment of patients with PAH.
Treprostinil palmitil is a prodrug consisting of treprostinil
linked by an ester bond to a 16-carbon chain. The prodrug becomes active when esterases cleave off the 16-carbon chain, resulting
in the active molecule treprostinil. TPIP is formulated as a dry powder for administration in a capsule-based inhalation device.
Building a Leading Neutrophil-Mediated Portfolio with Brensocatib
Insmed will outline its plans to leverage the potential of brensocatib,
its small molecule, oral, reversible inhibitor of dipeptidyl peptidase I (DPP1), in an array of neutrophil-mediated diseases, beginning
with NCFBE. The Company will discuss the study design for its registrational Phase 3 trial of brensocatib for the treatment of
NCFBE. The trial, known as ASPEN, was developed with input from both the U.S. Food and Drug Administration (FDA) and the European
Medicines Agency (EMA). Insmed remains on track to launch the ASPEN trial in the fourth quarter of 2020.
The Company also announced plans to advance a clinical development
program for brensocatib in cystic fibrosis (CF) and to discuss this program with health authorities in the first half of 2021.
Global Expansion for ARIKAYCE (Amikacin Liposome
Inhalation Suspension)
ARIKAYCE is the first and
only FDA-approved therapy for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a
combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. Today the
Company will provide an update on its global expansion efforts in Europe and Japan. Insmed recently received a positive
opinion from the Committee for Medicinal Products for Human Use of the EMA for the treatment of nontuberculous mycobacterial
(NTM) lung infections caused by MAC in adults with limited treatment options who do not have CF. The Company anticipates
receiving Marketing Authorization in the European Union by the end of October, with a planned first European launch by end of
2020. In addition, Insmed filed a Japanese New Drug Application (JNDA) with the Japanese Ministry of Health, Labour and
Welfare in March of this year. Following an anticipated 12-month review, if ARIKAYCE is approved, the Company would
anticipate launching ARIKAYCE in Japan in the middle of 2021.
Additionally, the Company will provide details around its planned
registration program to pursue regulatory approval of ARIKAYCE as a front-line therapy designed to establish a new standard of
care for patients suffering from NTM lung disease. The clinical study design includes two separate but inter-related clinical trials
to be conducted in parallel. ENCORE is the pivotal study intended to fulfill the post-marketing requirement to allow full approval
of ARIKAYCE in the United States. The ARISE trial is an interventional study designed to validate the patient-reported outcome
(PRO) tool that will be used to measure efficacy in ENCORE. The Company plans to initiate both trials globally in the fourth quarter
As of June 30, 2020, Insmed had cash and cash equivalents
of $641.9M. As of today, the Company expects its cash runway to extend over three years based on currently planned development
The R&D Day is scheduled to take place today from 10:00
a.m. to 12:30 p.m. ET in a virtual format. The call and accompanying slides will be webcast live on the Company's
website at https://investor.insmed.com/events. To listen to the conference call live, please dial (888) 317-6003 (domestic) or
(412) 317-6061 (international) and reference conference ID number 0587949.
A replay of the conference call will be accessible approximately
on hour after its completion through October 14, 2020 by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international)
and referencing replay access code 10147009. A webcast of the call will also be archived for 90 days under the Investor Relations
section of the Company's website at https://investor.insmed.com/events.
Insmed Incorporated is a global
biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial
product, ARIKAYCE (amikacin liposome inhalation suspension), is the first and only therapy approved in the
United States for the treatment of refractory Mycobacterium avium complex (MAC) lung disease as part of
a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. MAC lung disease
is a chronic, debilitating condition that can cause severe and permanent lung damage. Insmed's earlier-stage clinical pipeline
includes brensocatib, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis
bronchiectasis and other inflammatory diseases, and treprostinil palmitil inhalation powder, a treprostinil prodrug formulated
as a dry powder for inhalation, which may offer a differentiated product profile for rare pulmonary disorders, including pulmonary
arterial hypertension. For more information, visit www.insmed.com.
Forward-looking Statements
This press release contains forward-looking statements that
involve substantial risks and uncertainties. "Forward-looking statements," as that term is defined in the Private Securities
Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties. Words
herein such as "may," "will," "should," "could," "would," "expects,"
"plans," "anticipates," "believes," "estimates," "projects," "predicts,"
"intends," "potential," "continues," and similar expressions (as well as other words or expressions
referencing future events, conditions or circumstances) may identify forward-looking statements.
The forward-looking statements in this press release are based
upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which