Prevention of Preterm Birth – LPCN 1107
Product Candidate: LPCN 1107
Potential to be the new standard of care for the prevention of preterm birth.
Product Attributes:
LPCN 1107 is an oral therapy option comprising 17-alpha-hydroxyprogesterone caproate (HPC) for the prevention of recurrent preterm birth. Numerous Preclinical and clinical studies have demonstrated the first oral enablement of HPC via Lipocine’s proprietary oral delivery technology, which overcomes a significant decades-long technical challenge.
Poorly water-soluble 17-alpha-hydroxyprogesterone caproate (HPC) is an ester of endogenous hydroxyprogesterone, a progestogen with no androgenic/anti-androgenic or estrogenic activity. It is not prone to CNS side effects from GABA-modulating metabolites. HPC safety is well characterized with 2000+ patients in previous trials, decades of clinical use, and literature safety reports at higher doses.
The FDA has granted orphan drug designation to LPCN 1107 based on a major contribution to patient care. Orphan designation qualifies Lipocine for various development incentives, including tax credits for qualified clinical testing, and a waiver of the prescription drug user fee when we file our NDA.
Our success-focused clinical design is based on available HPC efficacy data directed to improving neonatal composite index (NCI), a medically relevant and the most responsive endpoint, in target population of higher risk PTB (gestational age of previous PTB of < 35 weeks that is most in need and likely to benefit.
About Indication:
PTB is when an infant is born 37 weeks before pregnancy, with early preterm being <35 weeks. PTB rates were estimated as ~ 11% of all livebirths worldwide, ranging from about 5% in several European countries to 18% in some African countries. More than 60% of PTB babies were born in south Asia and sub-Saharan Africa, where 52% of the global livebirths occur. PTB also affects rich countries, e.g., the USA has high rates and is one of the ten countries with the highest numbers of PTBs.
The history of a most recent spontaneous PTB increases the risk of PTB in the current, second or third, pregnancy by 4 times and a history of spontaneous PTBs in the first and second pregnancy increases the risk of PTB in the current, third, pregnancy by 8 times.
A developing infant goes through important growth throughout pregnancy, including in the final months and weeks; vital body organs and systems, e.g., brain, lungs and the immune system need the final weeks of pregnancy to fully develop
Infants born too early have higher rates of death and disability. PTB and low birth weight accounted for ca 16% of infant deaths (deaths before 1 year of age). Babies who survive PTB could have short- and long-term health complications. PTBs may also take an emotional toll and be a financial burden for families.
Manuck et al., AJOG, 215(1):103.e1-103.e14, 2016
The expense associated with PTB involves not only the immediate cost of the preterm baby being treated in the hospital ICU setting but includes the long-term treatment costs for disabilities for the life of the child. Preterm birth (“PTB”) is a health crisis in the US and globally and is estimated to cost the US healthcare system >$25B annually.
Source: National Vital Statistics Reports, Vol 67, No. 8, 2018; Centers for Disease Control and Prevention, 2017; World Health Organization, Born too soon: The global action report on preterm birth, 2012; World Health Organization, Preterm Birth, 2022
Globally, the rate of PTB ranges from 5% to 18% of babies born each year and PTB occurs in ~10% of all US births or 1 PTB every minute. Roughly, 1 million children younger than 5 years of age die each year due to PTB complications.
There is a significant unmet need for a reliable ‘patient friendly’ product for the prevention of PTB. In 2023 The FDA citing that Absent evidence of clinical benefit, using Makena to prevent recurrent PTB in pregnant women exposes them only to risk, so the risk-benefit balance is unfavorable, announced the final decision to withdraw approval of the injectable HPC Makena and its generics, which are now no longer commercially available.
As of 2025, the current ACOG recommendations for prevention of PTB include vaginal progesterone, which has not been proven effective in the absence of a shortened cervix, and Intramuscular 17-OHPC, which is not recommended as primary prevention for patients with a history of spontaneous PTB and only available by compounding.
Source: Prediction and Prevention of Spontaneous Preterm Birth: ACOG Practice Bulletin, Number 234. Obstet Gynecol. 2021 Aug 1;138(2):e65-e90. doi: 10.1097/AOG.0000000000004479. PMID: 34293771
https://www.fda.gov/news-events/press-announcements/fda-commissioner-and-chief-scientist-announce-decision-withdraw-approval-makena