Intrexon's Blood Cancer Drug Gets Orphan Drug Status From FDA

Intrexon Corporation’s (NASDAQ:XON) subsidiary, Precigen, Inc., announced that the FDA has granted orphan drug designation (ODD) to PRGN-3006, a rare cancer candidate. PRGN-3006 is a first-in-class investigational therapy using Precigen's non-viral UltraCAR-T therapeutic platform for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML), a cancer that starts in the bone marrow but often moves to blood.Precigen announced the completion of enrollment in the first cohort of this study and expects an initial data readout in the second half of 2020.

Shares of the company have lost 20.4% in the past year compared with the industry’s decline of 6.7%.

Notably, ODD is granted to drugs, which are capable of treating rare diseases that affect less than 200,000 people in the United States. This status also makes the company entitled to certain other benefits, including tax credits, and funds clinical study expenses.

PRGN-3006 utilizes Precigen's transformative UltraCAR-T therapeutic platform, which eliminates ex vivo expansion, reduces manufacturing time and provides the ability to administer CAR-T therapy to patients only one day after non-viral gene transfer at the cancer center.

We remind investors that, a few days ago, Intrexon announced that it will refocus on healthcare, change its name to Precigen, Inc. It also appointed Helen Sabzevari, PhD, as president and CEO.

Moreover, Intrexon signed definitive agreements to sell certain other non-healthcare assets to Third Security, LLC, a venture capital firm that invests in high-growth, technology-driven businesses, for $53 million in cash plus the contingent right to receive certain additional amounts that the latter may earn from these assets after closing of the deal. The company also entered into an agreement to sell its interest in EnviroFlight, LLC, to Darling Ingredients, Inc. for $12.2 million in cash.

Zacks Rank & Key Picks

Intrexon currently carries a Zacks Rank #3 (Hold).

A few better-ranked stocks in the medical drugs sector are AcelRx Pharmaceuticals, Inc. (NASDAQ:ACRX) , Assertio Therapeutics, Inc. (NASDAQ:ASRT) and Corcept Therapeutics Inc. (NASDAQ:CORT) ,all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

AcelRx’s loss per share estimates have narrowed from 72 cents to 70 cents for 2019 and from 67 cents to 62 cents for 2020 in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters by 24.24%, on average.

Assertio delivered a positive earnings surprise in the trailing four quarters by 26.88%, on average.

Corcept’s earnings per share estimates have increased from 73 cents to 77 cents for 2019 and from 93 cents to 98 cents for 2020 in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters by 20.02%, on average.

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