RXi Pharmaceuticals Receives Orphan Drug Designation for Samcyprone™ from the U.S. FDA for the Treatment of Malignant Melanoma Stage IIb to IV

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fda_logoRxi se stále potácí před “bankrotem” po spekulativním obchodu hlavních věřitelů, jimž se společnost stala pouze výtahem pro vlastní firmy ve zcela jiném odvětví medicíny. Tato nová prohlášení Rxi jsou tak pouze vějičkami pro případné nové public investory. Předpokládám delisting z NASDAQ a návrat firmy na OTC trh. Věda není vše a ani objevitel RNAi dr. Mello není zárukou, že se výzkum neocitne ve slepé uličce. Informace, které společnost předkládá, jsou tak nyní velmi spekulativní a institucionální investory bezpochyby odradí od jakékoliv větší investice. Společnost by musela přijít se zásadním pokrokem v léčbě, který se ale zatím vůbec nekoná. Ačkoli se RNAi zdá jako velmi perspektivní cesta, pokud se nic nezmění, očekávám zásadní pád celého segmentu RNAi v tomto a příštích letech. Toto odvětví se zdá být zatím příliš náročné a vysoce nákladné a v dlouhodobém horizontu neočekávám zisky pro jakékoliv společnosti RNAi.

RXi Pharmaceuticals Corporation (NASDAQ: RXII), a biotechnology company focused on discovering and developing innovative therapies primarily in the areas of dermatology and ophthalmology that address high-unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation of its second clinical candidate, Samcyprone™, for the treatment of Malignant Melanoma Stage IIb to IV. A number of patients with Stage IIb to IV malignant melanoma develop cutaneous metastases. Samcyprone™ is being developed for treatment of these metastases.

Samcyprone is a topical formulation of Diphenylcylcopropenone () in clinical development for the treatment of warts, alopecia areata and cutaneous metastases of melanoma. The mechanism of action for treatment cutaneous metastases of melanoma involves elicitation of an immune response in the skin that subsequently causes destruction of the cutaneous tumor.

Melanomas, cancers that arise from melanocytes, are the most aggressive form of skin cancer.  Once melanoma has spread beyond the localized area of the primary lesion, the survival rate decreases and melanoma becomes increasingly more difficult to treat successfully.  Management of metastatic melanoma, including cutaneous metastases, is challenging and represents an area of great unmet need.

“This is an important step for patients suffering from this devastating disease,” said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He further added, “This orphan designation underscores the value of expanding our clinical pipeline through the acquisition of Samcyprone. It provides the Company with access to cost-saving benefits and incentives to aid in the development of this drug, and establishes a link to one of our other preclinical research programs with our sd-rxRNA platform focused on tyrosinase, a key enzyme in the synthesis of melanin.”

About Orphan Drug Designation

Orphan drug status is granted for novel drugs or biologics to treat rare medical diseases or conditions that affect less than 200,000 people in the United States. The designation qualifies the sponsor for numerous incentives including seven years of market exclusivity after the drug’s approval, tax credits for clinical research costs and application fee reductions.

About Samcyprone™

Samcyprone™ is a proprietary topical formulation of diphenylcyclopropenone (DPCP).  Samcyprone™ is being evaluated for the treatment of warts, alopecia areata and cutaneous metastases of malignant melanoma. DPCP is an immunomodulating agent that works by eliciting a T-cell response.  While the drug DPCP has been used for decades, it is expected that Samcyprone™ will result in a better safety profile, a more consistent drug product and equivalent efficacy at lower doses. It is also expected to achieve market exclusivity post approval. Currently, an investigator sponsored clinical trial in cutaneous metastases of melanoma, as well as a new investigator sponsored clinical trial in alopecia areata are ongoing. In addition, RXi plans to initiate a Phase 2a clinical trial in warts for Samcyprone by the end of this year.

The mechanism of action of Samcyprone™ is linked to DPCP’s ability to alter the expression of multiple genes and miRNAs involved in the immune response. RXi’s unique sd-rxRNA® platform allows for the rapid identification of lead compounds for multiple targets in different therapeutic areas. Our work with Samcyprone™ may also allow us to discover specific targets and develop new sd-rxRNAs for the potential treatment of immunological disorders that are relevant to the skin as well as various systemic diseases.

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