Autolus Therapeutics Rises on FDA Orphan Drug Designation

Autolus Therapeutics (NASDAQ:AUTL) got a boost from the US Food and Drug Administration (FDA) after the agency gave orphan drug designation to the company’s AUTO1 treatment for acute lymphoblastic leukemia (ALL).

In a press release on Tuesday (November 5), the company said that in its ongoing studies the treatment has demonstrated high remission rates and positive CAR T-cell expansion and persistence without initiating high-grade cytokine release syndrome.

AUTO1 is a CD19 CAR T-cell investigational treatment that is intended to overcome safety limitations while still being as effective as existing CD19 CAR-T cell treatment options.

Dr. Christian Itin, chairman and CEO of Autolus, commented that the company will present AUTO1 data at the American Society of Hematology at the end of this year. AUTO1 is currently being tested in two Phase 1 studies — one in pediatric patients with ALL and the other in adult ALL patients.

According to the National Cancer Institute, ALL is cancer that is found in the blood and bone marrow and results in too many lymphocytes, which are a type of white blood cell. Symptoms of ALL include fever, exhaustion and easy bleeding and bruising.

In 2019 alone, it is estimated there will be roughly 5,900 ALL diagnoses in the US, with 1,500 of those cases resulting in death. ALL accounts for less than 1 percent of all cancer diagnoses in the US.

Looking ahead, a research report from Allied Market Research states that the global ALL market will reach US$3.52 billion in 2026, up from US$2.33 billion in 2018. That represents a compound annual growth rate of 5.3 percent during that time.

According to the report, an increase in incidents and bone marrow biopsies will fuel growth, while research and development investments for different types of leukemia will also be on the rise. Awareness for targeted therapies across the entire ALL therapeutics market is projected to grow exponentially as well.

Autolous also said Tuesday that it will present preclinical data on AUTO6NG, which is currently in a Phase 1 clinical trial for pediatric neuroblastoma, at the Annual Meeting of the Society for Immunotherapy of Cancer. The meeting will take place between November 6 and 10.

Shares of Autolus Therapeutics rose 10.69 percent on Tuesday to close at US$14.29. Year-to-date the company’s share price has dipped over 57 percent.

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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.