Telormedix, a clinical stage biopharmaceutical company focused on TLR7 agonists in the treatment of cancer and infectious diseases, has obtained the orphan drug designation to Vesimune for the treatment of carcinoma in situ (CIS) in the bladder, by the U.S. Food and Drug Administration (FDA).

Vesimune (TMX-101), is a proprietary targeted small molecule for the treatment of superficial bladder cancer. The active ingredient is a known immunomodulatory molecule with a favourable safety profile and a demonstrated clinical efficacy in oncological and viral diseases. It is Telormedix’ lead product, a TLR-7 agonist, that has successfully completed a Phase II trial in CIS of the bladder. The product is a unique sterile liquid formulation of a marketed immune modulatory compound, designed on innovative technology principles to carrier drug delivery systems in order to increase solubility, bio-adhesiveness and stability. These properties mean that the product can be used in therapeutic settings that the original product could not.

The FDA grants orphan drug status to products for rare diseases and defines a rare disease as one with a prevalence of less than 200,000 cases in the USA. As there are approximately 60.000 cases of non-muscle invasive CIS of the bladder, it is clearly a rare disease meaning that the orphan designation was extended to Vesimune.

Johanna Holldack, CEO of Telormedix, commented: “This is fantastic news that vindicates our development strategy. With Vesimune having completed Phase II and with this orphan drug designation, we are now in a pivotal position for discussions with potential license partners.”

In addition, Telormedix is developing two additional TLR7-targeted molecules, TMX-201 and TMX-202 both of which would make good vaccine adjuvants. As these molecules have substantially improved pharmacokinetics and pharmacodynamics profiles they have the potential to fully realise the anticancer promise of TLR 7 agonists. One of these molecules, TMX-202 has recently been selected for preclinical study for the topical treatment of skin cancers and other indications. The candidate has already successfully completed a number of in vivo studies.

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