Advent Therapeutics Awarded $3 Million NIH Grant for Novel Neonatal Lung Therapy

DOYLESTOWN, Pa., July 18, 2023 (GLOBE NEWSWIRE) -- Advent Therapeutics, Inc., a biotechnology company pioneering an optimized retinol palmitate (vitamin A) drug product as a breakthrough non-invasive inhaled neonatal lung therapy, has been awarded a $3 million National Institutes of Health (NIH) Small Business Innovation Research (SBIR) Phase IIB grant. This grant allows the Company to position its novel aerosolized vitamin A formulation for commercialization in 2025 to prevent Bronchopulmonary Dysplasia (BPD). BPD is a rare disease and the most common serious complication facing premature infants, resulting in significant morbidity and mortality. There are currently no approved therapies. With this grant, the Company has now received in excess of $6 million from NIH for its neonatal programs.

“This NIH award will allow us to greatly accelerate the development and advance towards approval of our optimized vitamin A drug product,” commented David L. Lopez, Chief Executive Officer, Advent Therapeutics. “Advent’s novel, precisely optimized products are intended to address the unmet medical need of BPD facing premature infants and reduce morbidity and mortality in this fragile patient population. Our primary focus is to accelerate the path to commercialization, first in the US, then EU and worldwide. US launch of Advent’s initial vitamin A drug product for injection is possible in early 2025 with potential peak annual revenues in excess of $100 million for the prevention of BPD. In addition, we intend to pursue broader applications for our novel retinol palmitate drug product that have significant market potential.”

This SBIR Phase IIB grant, awarded under the auspices of both the National Heart, Lung, and Blood Institute (NHLBI) and National Center for Advancing Translational Sciences (NCATS), will support development of Advent’s proprietary aerosolized formulation of vitamin A. The $3 million provided under the SBIR Phase IIB grant will be used to complete preclinical requirements and manufacturing scale-up leading to pivotal human clinical trials with Advent’s inhalable retinol palmitate product to potentially gain US and other worldwide marketing approvals.

Dr. Virender Rehan, Advent’s Principal Investigator at the Lundquist Institute, Professor of Pediatrics, David Geffen School of Medicine at UCLA, Chief of the Division of Neonatology, and Director of the Neonatal Intensive Care Unit, Harbor-UCLA Medical Center, said, “BPD is the most frequent serious complication that affects roughly 15,000 infants annually in the US, with an even greater number of premature infants at risk. With no approved drugs for BPD, these fragile, at-risk premature infants require prolonged mechanical ventilation or oxygen support and lengthy hospital stays often resulting in recurring hospitalizations and pulmonary infections. A baby with BPD adds significant emotional constraints for the family and financial burden to the healthcare system.”

Advent has longstanding collaborations with Dr. Rehan and the world-renowned Lundquist Institute for Biomedical Innovation. The Lundquist Institute is one of the foremost independent, non-profit research institutes in the US and is partnered with Harbor-UCLA Medical Center.

Dr. Virender continued, “I am extremely pleased to be involved in developing Advent’s novel form of inhalable vitamin A therapy. This product has the potential to be a “first-in-class” therapy capable of non-invasively delivering a safe and effective aerosolized vitamin A dose directly to the neonate’s lung. A lung-targeted inhalation therapy that could effectively reduce development of BPD would have significant clinical, financial, and societal implications by directly addressing co-morbidities suffered by these fragile patients.”

Advent has previously been awarded Orphan Drug Designation in both US and Europe for its proprietary vitamin A formulation. Orphan Drug Designation provides incentives including assistance with drug development process and exemption from user fees, as well as a seven-year window of exclusive US marketing rights (10 years in Europe) following marketing approval.

ABOUT BRONCHOPULMONARY DYSPLASIA (BPD)
BPD is a rare disease and the most common serious complication facing premature infants, resulting in significant morbidity and mortality. It is an extremely costly respiratory disorder afflicting premature infants for which there is currently no approved drugs worldwide. Annually, in both US and Europe, there are approximately 100,000 premature infants at-risk for BPD and many more in Asia-Pacific, Latin America and throughout the world. Hospital costs in the US for treating a BPD infant in the first year are approximately $442,000 with an average first year hospital stay of 103 days. US healthcare costs of treating babies with BPD have been estimated as $2.4 billion per year.

NOVEL VITAMIN A DRUG PRODUCT MANUFACTURING
Advent is completing manufacturing scale-up of its proprietary optimized retinol palmitate drug product at Pace^® Analytical Life Sciences. Commercial product for administration by injection is expected by 4Q 2024. Pace is a highly regarded contract development and manufacturing organization (CDMO) with facilities throughout the US, including San Diego, CA, where Advent’s manufacturing scale-up is being completed, and Salem, NH, where commercial batches will be produced. To support commercialization, Advent has also established a partnership with dsm-firmenich that provides Advent with a supply of highest quality active pharmaceutical ingredient (API) and access to dsm-firmenich’s Drug Master File for US and other worldwide regulatory authority marketing approvals. dsm-firmenich is one of the largest multinational companies that produces API and other products for the life sciences industry.

ABOUT ADVENT THERAPEUTICS
Advent Therapeutics, Inc. (Advent) was founded by senior industry executives with extensive expertise and proven track records in developing products in respiratory diseases for neonatal, pediatric and similar Orphan Drug “niche” markets and therapies. Advent is committed to developing life-changing medicines for the most-fragile preterm infants and is focusing on novel approaches to addressing serious unmet medical needs facing this significantly underserved Orphan Drug patient population. Advent has developed a proprietary formulation of optimized retinol palmitate (vitamin A) specifically designed for dosing to preterm infants to address vitamin A deficiency and associated serious complications of prematurity such as bronchopulmonary dysplasia (BPD), retinopathy of prematurity (ROP), and neonatal sepsis, all significantly impacting both short- and long-term clinical outcomes. The Company is also pursuing the potential of inhaled optimized retinol palmitate to mitigate acute lung injury (ALI) and address CBRN (chemical, biological, and radio-nuclear) threat agents, and pulmonary infections (bacterial, influenza, SARS-CoV-2), which often progress to life-threatening acute respiratory distress syndrome (ARDS). These represent large therapeutic opportunities for which Advent expects to partner with others for development and commercialization. For more information, please visit Advent’s website at http://www.advent-therapeutics.com.

KEY PARTNERS AND SPONSORS

dsm-firmenich – Advent has a supply arrangement with dsm-firmenich that provides the Company with supplies of vitamin A palmitate, the critical high-quality active pharmaceutical ingredient (API), necessary for development and commercialization. dsm-firmenich is a world leading supplier of vitamins, carotenoids and other nutritional solutions for the pharmaceutical industry. dsm-firmenich is a Swiss-Dutch company, listed on the Euronext Amsterdam, with operations in almost 60 countries and revenues of more than €12 billion. Through their collaboration since 2015, dsm-firmenich has provided invaluable technical support and maintains an active FDA Drug Master File (DMF) that will be incorporated into Advent’s US and other worldwide regulatory approvals.

BEN FRANKLIN TECHNOLOGY PARTNERS OF SOUTHEASTERN PA – Advent received seed capital from Ben Franklin and maintains a strong relationship that offers business and technical expertise and the potential for additional venture funding. Ben Franklin has been ranked as a top 10 venture capital firm in Pitchbook and has invested over $200 million in more than 2,000 regional enterprises, many of which have gone on to become industry leaders.

PENNSYLVANIA BIOTECHNOLOGY CENTER (BUCKS COUNTY) – Advent maintains lab and office space at the Pennsylvania Biotechnology Center located in Doylestown, Bucks County. The PABC is one of the nation’s leading biotech incubators and seeks to advance biotechnology in the region and foster the launch of new ideas and discoveries that will make a difference.

FORWARD LOOKING STATEMENTS
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.

Media and investor contact:
David L. Lopez
[email protected]