Benzinga | Sep 30, 2020 08:07AM EDT

Insmed Outlines Growth Strategy At R&D Day

BRIDGEWATER, N.J., Sept. 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 Powder

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(r) (Amikacin Liposome Inhalation Suspension)

ARIKAYCE is the first and only FDA-approved therapy for the treatment ofMycobacterium aviumcomplex (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 theEMA 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 of 2020.

Financial Update

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 programs.

Conference Call

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.