The right stuff made Plakous Therapeutics’ technology worthy of a winning ‘biomed’ PitchRounds position at AdvanSE 2022.


Baby Laurel was born at 24 weeks, 5 days, more than 15 weeks premature weighing just 1.5 pounds. Laurel is one of the roughly 3,000 very premature babies born in the United States every year who develop Necrotizing Enterocolitis (NEC), a rare pediatric disease without an approved diagnostic test or treatment. Nearly one-third of those cases result in the death of the infant due in part to inadequate length of exposure to amniotic fluid in the womb.


In addition to the high mortality rate for NEC, the current, extensive course of treating extremely premature babies with the rare disease brings an estimated $1.75 billion in NICU costs alone every year. 


Common complications of NEC include neurodevelopmental delay, failure to thrive,  gastrointestinal problems including strictures and adhesions, cholestasis, short bowel syndrome with or without intestinal failure that can be difficult to manage.  


Scott Washburn, MD, OB/GYN, the founder and Chief Medical Officer of Plakous Therapeutics set out to develop a placenta based biotherapeutic, a cell free mixture of cytokines and growth factors that mimics second trimester amniotic fluid in 2016.


Fast forward to the present day and, according to Plakous, “necrotizing enterocolitis remains the most threatening gastrointestinal complication of prematurity leading to high mortality, morbidity and cost.


Plakous Therapeutics’ novel treatment for NEC has shown a significant reduction in the severity of the disease. In preclinical studies of animals treated with Plakous’ biotherapeutic the severity of the disease was reduced by greater than 33%. The team and technology has further achieved:

  • Orphan Drug and Rare Pediatric Disease Designations from the FDA which includes access to a priority review voucher upon BLA approval
  • Accelerated FDA review (6 months)
  • Streamlined clinical trials
  • Market exclusivity
  • NIH SBIR Fast Track Grant of $1.72M 
  • Mayo Clinic agreement to develop Natural History Study


An Innovation Worthy of Ecosystem Validation

“Plakous Therapeutics has a unique offering that will be reimbursed; a platform biotherapeutic to prevent the rare pediatric disease, Necrotizing Enterocolitis, a true unmet medical need with no other product available,” said Life Science Management Consultants CEO Valerie Darling. “NEC afflicts 6% of the 50,000 babies born annually in the U.S weighing less than 3 pounds and carries a 30% mortality rate, represents $8.0B in overall healthcare costs, and 20% of annual NICU expenses. The therapeutic has received rare pediatric disease and orphan drug designations from the FDA, guaranteeing reimbursement.


The PitchRounds at AdvanSE 2022 judges deemed Plakous’ technology and opportunity worthy of the winning position in the biotechnology category.


“It was difficult to choose a winner at this year’s Pitch Round Competition due to a plethora of excellent companies and game-changing innovations,” said Darling. “In the end, it came down to ‘investability’ – the company that yielded high likelihood for an expedient return on investment with a first-to-market, unique product likely to receive reimbursement for a disease state or medical condition with no other options that represented significant healthcare cost and market potential.”


What can other innovators learn from Plakous’ experience at AdvanSE?

“Initially, innovators tend to focus too intently on their science and not its benefits during ‘introductory’ funding pitches to prospective investors,” Plakous CEO Robert Boyce told us. “In our prep sessions with our mentor colleagues we learned that we needed to focus more highly on our product’s benefit and how that benefit, the data, gets created more intently than on the chemistry or science itself.


“Almost every pitch scenario will involve unique audiences with unique levels of experience, interest and professional proficiencies. Some are more interested in the science and will not go forward until they believe the science works for them. Others will accept that your science is and does what you say it is and does at the outset, but will demand that you understand your marketplace. 


“It is also important to understand what you don’t know, especially related to health economics, manufacturing, logistics and therapeutic areas from which you might be taking market share, which represents a threat to some in the industry.


“On top of several years of refining our story and pitch decks, our colleagues at Keiretsu, and mentors introduced to us through Southeast Life Sciences have helped us crystallize audience appropriate stories and our value proposition. While the patient is the ultimate consumer of the technology, the hospital is the buyer and the doctor is the distributor. Our story, like many other innovators,’ must be compelling in all three scenarios to all three audiences.”


Funding the home stretch to FDA IND application and first-in-human trials

As of this story’s publication date, Plakous is narrowly focused on completing its pre-IND (Investigational New Drug) submission to the U.S. FDA by the end of the calendar year. Manufacturing and release testing over the next four or five months will help ensure the package demonstrates the consistency and potency of the product. Safety and toxicity studies are planned for early 2024.


“The activities of the next twelve to 24 months are critical to our commercialization pathway success and exit potential,” Rob said. “We are currently seeking $10.0M to get into human studies and an additional $25.0M round to get through clinical studies and to commercialization. Simultaneously, we are onboarding two new scientists and filling gaps in quality, regulatory and clinical operations to ensure we are fully prepared for the transition from preclinical to in-human clinical phases.


“We have a clear pathway to success for NEC and have plans–and early data–to support additional diseases of inflammation for indications including osteoarthritis and osteoporosis.


The team at Southeast Life Sciences congratulates Plakous Therapeutics on their PitchRounds success at AdvanSE 2022 and will be intently watching their high potential progress.