Lipigon's second project, which is out-licensed to CombiGene AB (publ), aims to help patients affected by partial lipodystrophy, a rare disease characterized by dysfunctional fat tissue. CombiGene estimates that the disease affects roughly 800 individuals across North America and Europe. The anticipated market value for this indication is several billion SEK (Swedish Kronor).
At present, no effective treatments exist for these patients. When gauging the potential market for Lipigon's medication, it's conceivable that the drug might benefit 25 to 50 percent of these patients. Lipigon currently projects the treatment cost per patient to be USD 1.5 million in the USA and USD 1.3 million in Europe, yielding a total potential sales range of USD 700 million to 14.5 billion.
Project 2 has been outlicensed to CombiGene since 2019. It is now part of CombiGene's development portfolio. Lipigon is entitled to revenue sharing and royalties on any sales.
Under the agreement, Lipigon receives early milestone payments for their collaborative lipodystrophy project, revenue sharing from potential third-party sales, and royalties upon market launch. Additionally, Lipigon acquired 1,322,751 shares in CombiGene, which, at the time of the agreement, held a market value of SEK 1.5 million. In August 2020, the company received another milestone payment in the form of shares, valued at roughly SEK 1.5 million, related to a patent application. More details can be found on CombiGene's website.
Existing Treatments on the Market
Metreleptin is an approved medication for lipodystrophy; however, it's effective only for a subset of patients.
For immediate treatment, either fasting or restricted food intake is advised to alleviate strain on the liver.
Liver transplants are frequently required for elderly patients or those who are severely affected.
To the best of Lipigon's knowledge, no drug development endeavors are specifically addressing the primary concern for these patients: excessive liver fat. By reducing the storage of fat, the liver may restore crucial metabolic functions, thus diminishing the likelihood of metabolic complications. In collaboration with CombiGene, a significant milestone was achieved with the presentation of substantial supporting data for a patent application in August 2020.
Relevant Structural Deals
Drug projects targeting established markers, such as low-density lipoprotein (LDL) cholesterol and its regulators, grounded in robust clinical, genetic, and fundamental research, are well-positioned for early partnerships. This potential is amplified by the significant medical necessity and commercial prospects inherent in the disease area.
In 2017, Regenxbio acquired Dimension Therapeutics with a transaction value of USD 85 million for two projects that had not reached the clinic.
In 2019, Silence and Mallinckrodt started a collaboration on a preclinical orphan drug project with USD 20 million in advance payment and USD 693 million in milestone payment.