P2 Lipodystrophy

P2 Lipodystrophy
Phase I
Phase II
Phase III

Lipigon's second project aims to help patients affected by lipodystrophy, a rare disease characterized by partial or complete absence of fat in the body.

The project has been licensed to Combigene AB. Learn more on Combigenes' website.


In the absence of normal body fat, various organs in the body, particularly in the liver, start to accumulate fat. This can lead to severe metabolic complications, including extreme insulin resistance, hypertriglyceridemia (elevated levels of the blood fat triglycerides), and cardiovascular disease. These conditions not only reduce the patient's lifespan but also significantly reduce their overall quality of life.

Currently, there are a limited number of treatments available that alleviate some of the symptoms associated with lipodystrophy. However, none of these treatments address the root cause of the disease. For this patient group, physicians primarily focus on providing preventive care for cardiovascular disease and diabetes. Additionally, hormone supplementation (such as Metreleptin) is administered to patients with generalized lipodystrophy to improve their well-being to some extent. Nevertheless, a specific treatment for the largest group of patients with partial lipodystrophy remains elusive.


Lipigon's target market for the drug in P2 Lipodystrophy, aimed at addressing lipodystrophy, is focused on patients with partial lipodystrophy. According to Lipigon's collaborative partner, CombiGene, their market analysis suggests that there are currently around 500 patients in the USA and 300 patients in the EU with this condition. The estimated market value for this indication is in the range of several billion SEK (Swedish Kronor).


Mechanism of Action

In P2 Lipodystrophy, gene therapy is utilized as the method of treatment. By encouraging the expression of a specific gene in the liver, the metabolism of liver fats is accelerated, resulting in a reduction of fat levels in the liver to normal ranges. This approach enables the liver to regain essential functions in the body's metabolism.

The Lipodystrophy project focuses on, in combination with conventional treatment, reducing the risk of life-threatening metabolic disease caused by fat accumulation in the liver, rather than directly addressing the deficiency of body fat tissue. To the best of Lipigon's knowledge, there are currently no other known drug development programs targeting the root issue of the disease, which is the elevated levels of fats in the liver.


In recent years, gene therapy has seen significant advancements. Unlike conventional drug treatments for chronic diseases that necessitate ongoing medication, gene therapy can, in its most promising applications, deliver lifelong effects with just a single treatment.

Out-licensing Agreement

Since 2019, Project 2 has been under an out-licensing agreement with CombiGene and is now an integral part of CombiGene's development portfolio. In this arrangement, Lipigon is entitled to receive early milestone payments within the collaborative lipodystrophy project, share in the revenue generated from potential third-party sales, and earn royalties upon market launch.

As part of the agreement, Lipigon was also granted 1,322,751 shares in CombiGene, which had an approximate market value of 1.5 million SEK at the time of the agreement's inception. Furthermore, in August 2020, the company received an additional milestone payment in the form of shares valued at around 1.5 million SEK, associated with a patent application.

Project Status Update

The pharmaceutical project targeting lipodystrophy has demonstrated positive outcomes in preclinical models. Presently, the project is undergoing optimization and adaptation for human applications, including the implementation of long-term studies in disease models as outlined in the collaboration agreement with CombiGene.

A noteworthy achievement occurred in August 2020 when substantial supporting data were presented, paving the way for a patent application. This marked a significant milestone in Lipigon's collaboration with CombiGene.