Pulmatrix Announces Positive Top-Line Results from Parts 1 and 2 of the 3-Part Phase 1/1b Clinical Trial of Pulmazole - an Inhaled Dry-Powder iSPERSE™ Formulation of Itraconazole

LEXINGTON, Mass., June 25, 2018 /PRNewswire/ -- Pulmatrix, Inc. (NASDAQ: PULM), a clinical stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary diseases announced today top-line results for the ongoing first-in-human study for Pulmazole (PUR1900) – an inhaled iSPERSE^TM formulation of the anti-fungal drug itraconazole for the treatment of allergic bronchopulmonary aspergillosis (ABPA) in patients with asthma.  Pulmazole was well tolerated across all doses tested in all cohorts in Parts 1 and 2.  Additionally, summary data for systemic pharmacokinetics from Part 2 following 14 days of daily inhalation of Pulmazole at doses ranging between 10 and 35 mg inhaled itraconazole suggest that total systemic exposure over 24 hours (AUC_0-24h) is approximately 100- to 400-fold lower that what would be expected following administration of 200 mg of oral Sporanox.  These results support the potential of Pulmazole to improve the safety profile of oral Sporanox, as there is a progressive increase in the probability of toxicity occurring with increasing concentrations of itraconazole after oral administration.

"The trial results suggest that inhalation of itraconazole can be safe and well tolerated and may be a better approach to treating ABPA, a disease for which there are no currently approved therapies," said Robert W. Clarke, Ph.D., Chief Executive Officer of Pulmatrix. "The reduced systemic exposure combined with the safety and tolerability profile observed supports the feasibility and potential differentiated benefits associated with this route of administration to treat aspergillus-related diseases in the lungs."

The Phase 1/1b study included 58 patients in a 3-part open-label study in healthy adults (Parts 1 and 2) and in adults with mild to moderate, stable asthma (Part 3). The main objectives of Part 1 single ascending dose and Part 2 multiple ascending dose was to evaluate the safety, tolerability, and pharmacokinetics (PK) of Pulmazole administered as either single- or multiple- doses administered daily over 14 days in normal healthy volunteers. The maximum dose of Pulmazole planned to be administered in this study was 35 mg itraconazole (70 mg dry powder including excipients).  A total of 23 subjects were dosed across 4 cohorts (5 mg, 10 mg, 25 mg, and 35 mg itraconazole) in Part 1, and a total of 18 subjects were dosed across 3 cohorts (10 mg, 20 mg, and 35 mg itraconazole) in Part 2. 

Results from Part 1 and Part 2 of the study demonstrated that Pulmazole was safe and well tolerated.  All study drug-related adverse events (AEs) were characterized as mild, and no moderate, severe or serious study drug-related AEs were reported.  The most common adverse event was the infrequent occurrence of a mild cough. Pharmacokinetic analysis of plasma showed that following dosing with Pulmazole, systemic drug levels were 100- to 400-fold lower than historically reported levels following dosing of oral Sporanox at the currently prescribed dose.

All subjects in Part 3 have completed their study visits and analysis of the preliminary data is underway and expected to be complete in mid-July 2018.

Jim Roach, MD, Chief Medical Officer of Pulmatrix remarked, "We are extremely pleased that upon review of the preliminary results from the normal healthy volunteer component of the study, Pulmazole was well tolerated up to the maximum dose tested.  Additionally, the substantially lower systemic exposure for itraconazole following inhaled administration speaks to the potential for Pulmazole to improve upon the known safety concerns associated with oral Sporanox.  We look forward to further reviewing and reporting the data from Part 3 of the study in asthmatic subjects, which included measuring itraconazole in sputum to assess relative lung exposure following administration of either oral Sporanox or Pulmazole, in the next few weeks."

About ABPA
ABPA is a disease that occurs most often in patients with underlying asthma or cystic fibrosis, and it is characterized by an exaggerated allergic hypersensitivity response of the immune system to the fungus Aspergillus colonizing and growing in the airways. Oral itraconazole (Sporanox®) is currently used as an adjunctive treatment to corticosteroids in ABPA patients. However, its use is limited by poor bioavailability, variable pharmacokinetics, and toxicity concerns related primarily to the risk of gastrointestinal and cardiac side effects, as well as extensive drug-drug interactions.  The Pulmatrix Pulmazole program is the first inhaled dry powder version of itraconazole known to the company to be advanced into clinical development, with the goal of improving upon the known safety and efficacy profile associated with oral Sporanox by delivering the drug directly to the lung. 

About Pulmatrix          
Pulmatrix is a clinical stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary disease using its patented iSPERSE™ technology. The Company's proprietary product pipeline is focused on advancing treatments for serious lung diseases, including Pulmazole, an inhaled anti-fungal for patients with allergic bronchopulmonary aspergillosis ("ABPA"), and PUR1800, a narrow spectrum kinase inhibitor for patients with obstructive lung diseases including asthma and chronic obstructive pulmonary disease ("COPD"). In addition, Pulmatrix has partnered with Vectura Group plc to develop Pulmatrix's long-acting muscarinic antagonist drug candidate, PUR0200, for COPD for the U.S. market. Pulmatrix's product candidates are based on iSPERSE™, its proprietary engineered dry powder delivery platform, which seeks to improve therapeutic delivery to the lungs by maximizing local concentrations and reducing systemic side effects to improve patient outcomes.

FORWARD-LOOKING STATEMENTS
Certain statements in this press release that are forward-looking and not statements of historical fact are forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The Company cautions that such statements involve risks and uncertainties that may materially affect the Company's results of operations. Such forward-looking statements are based on the beliefs of management as well as assumptions made by and information currently available to management. Actual results could differ materially from those contemplated by the forward-looking statements as a result of certain factors, including but not limited to the ability to establish that potential products are efficacious or safe in preclinical or clinical trials; the ability to establish or maintain collaborations on the development of therapeutic candidates; the ability to obtain appropriate or necessary governmental approvals to market potential products; the ability to obtain future funding for developmental products and working capital and to obtain such funding on commercially reasonable terms; the Company's ability to manufacture product candidates on a commercial scale or in collaborations with third parties; changes in the size and nature of competitors; the ability to retain key executives and scientists; and the ability to secure and enforce legal rights related to the Company's products, including patent protection. A discussion of these and other factors, including risks and uncertainties with respect to the Company, is set forth in the Company's filings with the Securities and Exchange Commission, including its annual report on Form 10-K filed by the Company with the Securities and Exchange Commission on March 13, 2018, as may be supplemented or amended by the Company's Quarterly Reports on Form 10-Q. The Company disclaims any intention or obligation to revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Investor Contact                                              
Robert Clarke, CEO                                           William Duke, CFO       
(781) 357-2333                                                   (781) 357-2333    
[email protected]                                      [email protected]

SOURCE Pulmatrix, Inc.

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