Aerogen Pharma, a developer of innovative inhaled treatments for patients in critical care, and Nuance Pharma ("Nuance"), a specialty care focused biopharma with late-stage clinical programs and existing commercial operations, today announce an exclusive agreement to join forces in Greater China to develop a superior, non-invasive approach to treatment of RDS, a life-threatening condition of preterm infants associated with long-term lung health issues in survivors.

The licensing agreement grants Nuance the exclusive rights to develop and commercialize AeroFact™, an innovate new therapy combining a well-established pulmonary surfactant drug with proprietary vibrating mesh nebulizer aerosolization technology, in Greater China (Mainland China, Taiwan, Hong Kong and Macau). The partners believe that AeroFact offers the prospect of an efficient, non-invasive, and easy-to-use product with benefits over conventional invasively-delivered lung surfactant that will improve clinical outcomes for the hundreds of thousands of premature babies around the world whose healthy development is threatened by RDS.

Under terms of agreement, Aerogen Pharma will receive upfront payment of $20 million and $10 million equity in Nuance, as well as potential development, regulatory, and commercial milestone payments totaling $182.5 million, plus escalating tiered low double-digit royalties on net sales within the territory.

John Power, Aerogen Founder & Chief Executive Officer, commented: "Pulmonary surfactant is a life-saving treatment for babies who develop RDS, but its conventional use involves sedation and intubation, an invasive process with significant potential complications. The neonatology community has long hoped for a non-invasive alternative that will enable the benefits of surfactant treatment without these drawbacks. Given our existing commercial distribution activities with Nuance and the significant investment they have made in establishing an experienced and highly qualified clinical and regulatory development team, we are confident that we have both the right product and the right partner for this critical market". Colin Telfer, Head of Business Operations at Aerogen Pharma, added: "Non-invasive delivery of surfactant to infants with RDS represents both a huge unmet medical need and a compelling commercial opportunity for Aerogen Pharma. We are delighted to be partnering with Nuance to develop and commercialize AeroFact in China, which today stands as the world's largest surfactant market."

Mark G. Lotter, Chief Executive Officer and Co-Founder of Nuance, commented: "RDS, as a breathing disorder, is rare disease in terms of absolute prevalence rate, but quite common in premature infants. We are thrilled to enter into this agreement with our long-time partner Aerogen on AeroFact, to set a new standard in treatment of RDS, and to address the critical unmet medical needs through provision of less invasive treatments for highly vulnerable infant patients. AeroFact represents an ideal fit for Nuance's portfolio, given our focus on developing and commercializing innovative products in respiratory space for the Greater China and AsiaPac."

About AeroFact

AeroFact is a nasally inhaled surfactant based on a combination of the long-established bovine lung surfactant Alveofact® (Lyomark Pharma GmbH, Germany) with a precise and highly efficient nasal delivery system featuring Aerogen's next generation "PDAP™" delivery technology and a touch-screen controller interface. Aerosol delivery of pulmonary surfactant to preterm infants through the nose to the lung is extremely demanding, due to both the technically challenging physical properties of the drug and the difficulties inherent in treatment of such small patients, but is achieved by PDAP, a technical breakthrough based on patented enhancements to the Aerogen group's market-leading aerosol generator technology (www.aerogen.com). AeroFact is anticipated to reduce the need for sedation, invasive intubation and mechanical ventilation, all features of current surfactant treatment methods associated with adverse side effects and the potential to exacerbate chronic lung disease in preterm infants.

AeroFact has received Fast Track and Orphan Drug designations from FDA and is currently under investigation in a Phase 2b multicenter, partially-blinded, randomized, controlled, parallel-group dose-ranging clinical trial. This trial, listed at www.clinicaltrials.gov as NCT03969992, is designed to assess the clinical activity, safety and tolerability of AeroFact at two dose levels, with the primary aim of determining an optimal dose of AeroFact to reduce the need for conventional invasive surfactant instillation in the first week of life. Secondary objectives include evaluation of safety and tolerability, comparison of time to invasive treatment across groups requiring intubation, and comparison of respiratory morbidity and resource utilization between groups. Approximately 42 clinical sites in the United States and Canada are involved.

The current trial follows encouraging results of an initial 30-patient trial in Australia, which demonstrated the potential for effective treatment of RDS symptoms by trans-nasal pulmonary aerosol delivery. Although not powered to reach statistical significance, this study also showed a trend towards a reduction in the need for invasive administration procedures and the incidence of bronchopulmonary dysplasia, which may support more routine use of surfactant therapy at an earlier stage in RDS progression.

About Aerogen Pharma

Aerogen Pharma is an Irish specialty pharmaceutical company developing inhaled products for quick, efficient, consistent and user-friendly treatment of critically ill patients in hospital acute care. Aerogen Pharma improves existing drugs by targeting them to the lung using a proprietary, high performance aerosol delivery system based on the market-leading nebulizer technology created by its parent organization, the Aerogen group. For more, see www.aerogenpharma.com.

About Nuance

Nuance is a Shanghai headquartered specialty care focused biopharma company focusing on respiratory, pain management and iron deficiency areas. With the mission to address critical unmet medical needs in Greater China and AsiaPac, since inception from 2014, Nuance has built a late clinical stage portfolio under development focusing on key therapeutic areas through global partnerships, while maintaining a self-sustainable commercial operation.

Media contact:

Aerogen Pharma: Colin Telfer, Head of Business Operations
Email: ctelfer@aerogenpharma.com 
Phone: +1 650 440 8963

Nuance: Charlie Chen, Chief Operating Officer
Email: charlie.chen@nuancepharma.cn
Phone: +86 21 3203 5318