LNS8801 for Uveal Melanoma Receives Orphan Drug Status

First formed and launched within the UPstart incubator at the University of Pennsylvania’s Penn Center for Innovation (PCI), biopharmaceutical company Linnaeus Therapeutics, Inc. (“Linnaeus”) is now making strides in the oncology field. In a recent press release, Linnaeus shared that its drug candidate LNS8801 received Orphan Drug designation. Specifically, the drug is designed for patients with metastatic uveal melanoma, which affects around 5 million people yearly around the globe.

LNS8801 for Uveal Melanoma

Developed by Linnaeus, LNS8801 is an orally administered GPER-agonist. In a 2019 article published in BMB Reports, the authors explain that:

G protein-coupled estrogen receptor (GPER) is known to play an important role in hormone-associated cancers.

While researchers are not sure of the exact cause of melanoma, or some of its subtypes, some do believe there could be an underlying genetic factor. In this case, LNS8801 activates GPER and reduces c-Myc proteins. Altogether, it has anti-tumor properties and is able to harness the immune system against tumors.

Currently, Linnaeus is running an ongoing Phase 1/2 clinical trial. During the trial, researchers are evaluating the drug’s safety, efficacy, and tolerability as a singular treatment or in combination with KEYTRUDA. So far, in addition to being both safe and well-tolerated, LNS8801 has shown benefits for patients.

LNS8801 received Orphan Drug designation for its treatment of patients with uveal melanoma. Orphan Drug status is given to drugs or biologics designed to serve patients with rare diseases or conditions. In the United States, this is defined as a condition which affects less than 200,000 individuals. Once a drug developer receives this status, they also receive tax credits, fee waivers, and up to 7 years of market exclusivity. 


Typically, melanoma is a severe form of skin cancer which forms in melanin-producing cells called melanocytes. This cancer generally affects adults, although it can impact children as well. Melanoma is often found on the arms and legs of females, or the head or neck of males. Additionally, melanoma often affects those with lighter complexions.

Normally, our bodies have anywhere from 10-45 moles. These moles are typically one color (brown, black, tan) with a distinct border. However, melanoma symptoms include:

  • Changes in an existing mole or in skin color
  • New moles growing near existing moles
  • Changing size, shape, or color of any new skin spots

Your mole may be suspect if it is asymmetrical, has an irregular or ragged borderchanges colors, is larger in diameter than 1/4 inch, or is evolving over time, whether that means bleeding or changing in shape. Melanoma can spread to other areas of the body, including the eyes.

Learn more about melanoma.

Uveal Melanoma

In a 2016 article published in Eye, the authors describe uveal melanoma as:

a relatively rare disease, primarily found in the Caucasian population, [but] the most common primary intraocular tumor in adults with a mean age-adjusted incidence of 5.1 cases per million per year. Tumors are located either in iris (4%), ciliary body (6%), or choroid (90%).

The Kellogg Eye Center continues by explaining that uveal melanoma is considered small, medium, or large based on the size of the tumor. Those with fair skin, blue or green eyes, or blonde or red hair are at higher risk of developing uveal melanoma. In early stages, symptoms may not be present. However, if symptoms do appear, they include:

  • Vision loss
  • Blurry vision
  • Pupil distortion
  • A dark spot in the colored area of the eye

Generally, uveal melanoma is discovered during routine eye examinations. Once diagnosed, uveal melanoma can be treated using radioactive plaque therapy, surgery, or enucleation.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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