CINCINNATI - Airway Therapeutics has made a promising breakthrough in better treating premature babies with underdeveloped lungs.
The Cincinnati startup's protein treatment could help reduce the incidence of Bronchopulmonary Dysplasia, or BPD, a potentially lifelong, chronic lung disease associated with preemies.
Airway Therapeutics is one of CincyTech's portfolio companies, and its treatment could be in use by the end of the decade. Seed money has also come from Cincinnati Children's Hospital Medical Center, which has a pulmonary research program.
9 Questions for Dr. Marc Salzberg, Airway Therapeutics CEO
1. How widespread is the problem of BPD among infants, is it more common in the cases of multiple births?
Several factors contribute to BDP. Ten percent of low birth weight babies (less than 2.2 pounds) and 40 percent of very-low birth weight babies (less than 1.76 lbs) develop BPD. Preterm very-low birth weight babies are at higher risk for BPD. Intrauterine infection is considered a risk factor as well. There is a strong genetic component linked to the incidence of BPD. Twin studies revealed a 50 percent heritability of BPD. Also, BPD is more common in babies from mothers who smoked or took steroids during pregnancy.
2. Could you explain to a lay person how this protein treatment works?
Preterm babies, and especially very low birth-weight babies, have under-developed lungs that are not capable of breathing on their own. The lungs of these preterm infants are deficient in lung surfactant and the associated surfactant proteins. Without the combination of surfactant and its proteins the under-developed lungs cannot sufficiently inflate or exhale without collapsing.
This is why the preterm baby requires both ventilation to administer oxygen and surfactant therapy. BPD develops as a result of the lung injury, inflammation and infection caused by the mechanical ventilation and administration of inhaled oxygen.
Today’s commercially available surfactant treatment is considered standard and critical therapy for preterm babies. However, commercially available surfactant lacks a protein, Surfactant Protein-D (SP-D). This not only has strong anti-inflammation and anti-infection properties, but also stabilizing the lung surfactant and ensure it is delivered to the right place in the lung at the right time.
Airway Therapeutics has developed recombinant human SP-D. It's been demonstrated that its product, known as AT-100, has strong anti-inflammation and anti-infection properties and can protect commercially available surfactant from degrading. This should lead to the decrease of time on ventilation for the babies and hence decrease a major risk factor for the development of BPD.
3. Where is the company in the process of bringing a product to market?
Pre-clinical studies in a clinically relevant model of BPD proved the concept that rhSP-D reduces the inflammation associated with ventilator- induced damage. Those studies also showed that rhSP-D stabilized the commercially available Surfactant against degradation and made it more structurally similar to naturally occurring surfactant so it would be better recycled within the lung. Hence, it is expected that rhSP-D will prevent or significantly reduce the incidence of BPD.
Airway Therapeutics has managed to also manufacture the protein, which is a major step in the developed of a biotechnologically produced protein. The next steps are to manufacture a larger batch of rhSP-D ready for use in humans. That material will be used to conduct toxicology studies in animals needed prior to use the product in babies. Then we plan to run the clinical studies required for obtaining marketing authorization. BPD is a rare disease (orphan indication). This will allow us to run a smaller and shorter than normal clinical development program.
4. What are the biggest challenges for bringing a treatment like this to market?
One challenge is the manufacturing of stable and pure product. The other is to define the adequate patient population and endpoints in the clinical studies.
5. How is the protein administered?
rhSP-D is added to the mechanical inhalation at the same time that the commercially available surfactant is added.
6. When will clinical trails start? When could a medicine be ready for use in hospitals?
We expect to start clinical trials in 18-24 months from now. It's our hope to obtain marketing authorization in 2019.
Next page: The role of CincyTech
7. What role has CincyTech played in the growth of Airway Therapeutics?
CincyTech has played and continues to play various important roles for Airway Therapeutics. CincyTech and Cincinnati Children's Hospital Medical Center (CCHMC) were the founding investors in the company, and have since re-invested. CincyTech identified the current management team, is helping us to seek additional funds through its large investor network and supports our management with guidance.
8. Does the company plan further partnerships on developing new treatment with Cincinnati Children's Hospital?
We are working closely with the CCHMC and reviewing other licensing opportunities originating from their research.
9. What's next for Airway Therapeutics?
We're fundraising for the scale-up of protein production to conduct animal studies. That will be followed by the clinical development program.
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