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Rubius Therapeutics (RUBY) Aiming To Make 2020 The Year Of Execution

pharma 050517 19feb20 lt

Shares of Rubius Therapeutics Inc. (RUBY) have lost over 57 percent of their value from the yearly high of $20.04, recorded on April 5, 2019, and trade around $8.

Rubius Therapeutics is a clinical-stage biopharmaceutical company developing a new class of medicines called Red Cell Therapeutics that are genetically engineered red blood stem cells for the treatment of rare enzyme deficiencies, cancer, and autoimmune diseases.

The Company's first investigational product to enter clinical trials is RTX-134, designed to treat phenylketonuria (PKU).

A phase Ib trial of RTX-134 for the potential treatment of patients with phenylketonuria was initiated in January of this year. This trial is designed to enroll 12 patients.

Phenylketonuria, or PKU, is a genetic dietary condition in which patients are deficient in the enzyme needed to break down dietary phenylalanine (Phe). This condition may lead to intellectual disability, delayed development, impaired cognitive function, and irreversible brain damage.

There are two approved medications to treat PKU - BioMarin's Palynziq and Kuvan.

According to a report by market research and consulting company Grand View Research, the global phenylketonuria treatment market size was valued at $445.8 million in 2018, and is expected to register a CAGR of 11.0% from 2019 to 2026.

Initial clinical data from the first patients enrolled in the phase Ib trial of RTX-134, including preliminary safety, the longevity of RTX-134 in circulation and proof-of-mechanism as measured by the production of trans-cinnamic acid (TCA), a metabolite of phenylalanine when degraded by phenylalanine ammonia lyase (PAL), are expected this year.

The other products in the pipeline are:

-- RTX-240 for the treatment of solid tumors, for which an IND has been filed with the FDA. The Company expects to get clearance for the RTX-240 IND this year.

-- RTX-321 for the treatment of HPV-positive tumors. An IND for RTX-321 is expected to be filed by year-end.

Rubius ended September 30, 2019, with cash, cash equivalents and investments of $324.7 million.

RUBY has traded in a range of $6.96 to $20.04 in the last 1 year.

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