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Orchard Therapeutics Waiting To Bloom

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Shares of Orchard Therapeutics plc (ORTX) are down 11 percent year-to-date while the iShares Nasdaq Biotechnology ETF (IBB), a bellwether of investor sentiment, has gained 8 percent for the same period.

UK-based Orchard Therapeutics is a commercial-stage company focusing on ex vivo autologous hematopoietic stem cell (HSC) gene therapy for rare diseases.

The Company has a pipeline of six clinical-stage gene therapies, and over the next three years, 3 of its investigational gene therapies are expected to reach the FDA altar.

The most advanced investigational product is OTL-200 for metachromatic leukodystrophy, an inherited lysosomal storage disorder. Patients with metachromatic leukodystrophy experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see.

A registrational trial of OTL-200 exceeded the primary efficacy endpoint for motor function in the most severe forms of the disease, according to the Company.

Orchard Therapeutics plans to file for approval of OTL-200 for metachromatic leukodystrophy in Europe in the first half of 2020 and also seek approval in the U.S. one year after the Europe filing.

Next up in the pipeline are OTL-101 for adenosine deaminase severe combined immune deficiency (ADA-SCID), and OTL-103 for Wiskott-Aldrich syndrome (WAS).

OTL-101

A two-year follow-up data in 20 patients from the registrational trial evaluating OTL-101 in adenosine deaminase severe combined immune deficiency were presented in February of this year.

Severe combined immunodeficiency (SCID), also known as bubble boy disease is an extremely rare, life-threatening genetic disorder, caused by a severe defect in key immune cells - i.e., both T- & B-lymphocytes - resulting in a severely compromised immune system and death by one year of age if untreated. The two most common types of SCID are the classical X-linked SCID (XSCID) that occurs in males, and ADA-SCID caused due to adenosine deaminase (ADA) deficiency.

There was a 100% overall survival and 100% event-free survival in patients treated with OTL-101 compared to 88% overall survival and 56% event-free survival with historical hematopoietic stem cell transplantation (HSCT) overall at 24 months, according to the Company.

The Company is expected to present engraftment data in 10 patients from a clinical trial of OTL-101 for severe combined immune deficiency due to adenosine deaminase deficiency using a cryopreserved formulation this year.

Orchard Therapeutics is planning to seek FDA approval for OTL-101 in the U.S. next year.

OTL-103

Data from an interim analysis of a registrational trial of OTL-103 in patients with severe Wiskott-Aldrich syndrome with follow-up ranging from 0.5 to 5.6 years showed a decrease in the frequency of severe infections, elimination of severe bleeding episodes and a great reduction of moderate bleeding episodes, according to the Company.

A three-year follow-up data in eight patients from a registrational trial of OTL-103 for Wiskott-Aldrich syndrome is expected to be reported this year.

Wiskott-Aldrich Syndrome is a life-threatening inherited immune disorder characterized by autoimmunity, eczema, abnormal platelet number and function.

A cryopreservation formulation registrational clinical trial for OTL-103 in patients with Wiskott-Aldrich Syndrome was initiated in January of this year. The Company remains on track to file for regulatory approval of OTL-103 in the U.S. and Europe in 2021.

Some of the other early-stage programs of Orchard Therapeutics are OTL-203 for the treatment of mucopolysaccharidosis type I (MPS-I), OTL 300 for the treatment of transfusion-dependent beta-thalassemia and OTL-102 for the treatment of X-linked chronic granulomatous disease.

The clinical proof-of-concept data for OTL-102 for the treatment of X-linked chronic granulomatous disease were presented in February of this year and the clinical proof-of-concept data for OTL-300 for the treatment of transfusion-dependent beta-thalassemia were presented in April.

The Company is expected to present data from the ongoing proof-of-concept trial of OTL-203 for MPS-I next month.

Besides the above-mentioned gene therapy product candidates, the Company has one approved gene therapy - Strimvelis, which received European Commission approval in 2016, for adenosine deaminase severe combined immunodeficiency (ADA-SCID). The sales of Strimvelis have been very limited, and in 2018, this gene therapy registered only $2.1 million in net product sales.

Strimvelis, OTL-200, and OTL-103 were all handed out to Orchard Therapeutics by GlaxoSmithKline (GSK) under a strategic agreement executed by the two companies last April. Under that agreement, GSK received a 19.9% equity stake along with a seat on Orchard Therapeutics' board.

Key Numbers:

Cash on hand: $423 million
Long-term debt: $24.5 million

With a couple of readouts lined up for the remainder of this year, and important regulatory events expected over the coming year and beyond, it's worth keeping an eye on Orchard.

The American Depositary Shares of Orchard Therapeutics, which made their debut on the Nasdaq Global Select Market on October 31, 2018, have thus far hit a low of $8.65 and a high of $21.64. ORTX closed Wednesday's (Aug.14, 2019) trading at $13.92, up 6.58%.

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