Periodic Reporting for period 2 - ELGN-GI (A first-in-class therapy for intestinal malabsorption in premature newborns)
Okres sprawozdawczy: 2023-11-01 do 2024-10-31
10% of infants worldwide are born prematurely, and the number is rising. In developed countries, survival rates of preterm infants are high, however their outcomes are often suboptimal due to prematurity associated complications.
Underdeveloped gastrointestinal tract resulting in an inability to absorb nutrition is one of their main problems. Currently, the only solution is delivering nutrients directly to the bloodstream (parenteral nutrition), which comes with a risk of complications and is inadequate for supplying the nutrients needed for the baby's development. Transitioning to enteral nutrition autonomy as fast as possible is a treatment goal for improving the outcomes of these infants.
ELGN-GI is an oral medication based on enteral insulin as the active substance. Physiologically, insulin was demonstrated to be present in the amniotic fluid and breastmilk after birth for accelerating the ability to break down nutrition and absorb it. Data indicate that premature infants may benefit from a longer exposure to insulin. ELGN-GI was developed exactly for this purpose. The drug is acting locally in the GI tract, without systemic absorption, and is accelerating the GI development to allow a faster maturation and transition to full enteral nutrition. It was already studied in clinical trials, including a previous phase III study with more than 300 infants, and was demonstrated to be safe and effective.
The upcoming phase III trial is the last study before filing for marketing authorization.
Following approval, preterm infants born before 32 weeks of gestation will be treated with ELGN-GI
Underdeveloped gastrointestinal tract resulting in an inability to absorb nutrition is one of their main problems. Currently, the only solution is delivering nutrients directly to the bloodstream (parenteral nutrition), which comes with a risk of complications and is inadequate for supplying the nutrients needed for the baby's development. Transitioning to enteral nutrition autonomy as fast as possible is a treatment goal for improving the outcomes of these infants.
ELGN-GI is an oral medication based on enteral insulin as the active substance. Physiologically, insulin was demonstrated to be present in the amniotic fluid and breastmilk after birth for accelerating the ability to break down nutrition and absorb it. Data indicate that premature infants may benefit from a longer exposure to insulin. ELGN-GI was developed exactly for this purpose. The drug is acting locally in the GI tract, without systemic absorption, and is accelerating the GI development to allow a faster maturation and transition to full enteral nutrition. It was already studied in clinical trials, including a previous phase III study with more than 300 infants, and was demonstrated to be safe and effective.
The upcoming phase III trial is the last study before filing for marketing authorization.
Following approval, preterm infants born before 32 weeks of gestation will be treated with ELGN-GI
The work carried out was dedicated to preparations for the clinical study. The preformed activities included study documents and submission package development, CTA submissions to competent authorities and ethics committees, sites selection and contracting, electronic systems set up, vendors selection, GMP manufacturing, labelling, packaging, and shipment of the investigational medicinal product.
The drug product was successfully manufactured (active medication and placebo). Analytical laboratories (GMP certified) were qualified for release and stability testing of the finished product. Final batch release and certification was performed by the European QP. Clinical batches were packaged and labelled in accordance with GMP guidelines and specific clinical requirements.
CRO contracting was finalized. Elgan have put together the infrastructure to ensure proper oversight and guidance to all contracted vendors which are used for outsourced activities. Safety reporting processes were defined, with all relevant stakeholders contracted.
The electronic systems were designed, tested, and validated according to the relevant regulations and the study-specific documents. All trial infrastructure is in place for launch of the clinical trial which would start imminently.
The drug product was successfully manufactured (active medication and placebo). Analytical laboratories (GMP certified) were qualified for release and stability testing of the finished product. Final batch release and certification was performed by the European QP. Clinical batches were packaged and labelled in accordance with GMP guidelines and specific clinical requirements.
CRO contracting was finalized. Elgan have put together the infrastructure to ensure proper oversight and guidance to all contracted vendors which are used for outsourced activities. Safety reporting processes were defined, with all relevant stakeholders contracted.
The electronic systems were designed, tested, and validated according to the relevant regulations and the study-specific documents. All trial infrastructure is in place for launch of the clinical trial which would start imminently.
ELlGN-GI will create a huge impact on preterm infants’ care and improve their outcomes, as well as reduce complications and hospitalization – thus costs and staff burden. Elgan performed a market research study, incorporating data collected in the previous phase III clinical trial. The report included identifying and analysing the main value drivers of ELGN-GI from the payers' perspective in several key markets, considering the specificities in each of them- a tailored market access approach was developed. Potential Reimbursement schemes, purchasing methods and funding mechanisms for inpatient medications were discussed, as well as potential competitors.
Scientific findings identified microbiome changes following administration of the ELGN-GI drug, potentially explaining the underlying mechanism for the efficacy observed and reduction in the rate of necrotizing enterocolitis. These results are under review and publishing by the investigators. They might support pharmaco-economic and clinical benefits of the drug, promoting the adoption and sales post- launch.
Elgan is routinely attending and sponsoring local and international medical conferences. Several scientific presentations of Elgan's data were delivered during these meetings, with great interest from the participants. The European neonatology community is familiar and supportive of Elgan's clinical program. Elgan continues to collaborate with nonprofit patient parent organizations, that are also supportive of the project. This wide support from the relevant stakeholders will facilitate the adoption of the product once approved, towards becoming a worldwide standard of care.
Elgan's intellectual property is extensive, with many patents allowed over the last two years, substantiating the exclusivity of the drug in the market. Being an orphan drug as well as paediatric, it is also eligible for additional regulatory exclusivities.
Scientific findings identified microbiome changes following administration of the ELGN-GI drug, potentially explaining the underlying mechanism for the efficacy observed and reduction in the rate of necrotizing enterocolitis. These results are under review and publishing by the investigators. They might support pharmaco-economic and clinical benefits of the drug, promoting the adoption and sales post- launch.
Elgan is routinely attending and sponsoring local and international medical conferences. Several scientific presentations of Elgan's data were delivered during these meetings, with great interest from the participants. The European neonatology community is familiar and supportive of Elgan's clinical program. Elgan continues to collaborate with nonprofit patient parent organizations, that are also supportive of the project. This wide support from the relevant stakeholders will facilitate the adoption of the product once approved, towards becoming a worldwide standard of care.
Elgan's intellectual property is extensive, with many patents allowed over the last two years, substantiating the exclusivity of the drug in the market. Being an orphan drug as well as paediatric, it is also eligible for additional regulatory exclusivities.