Agios Receives FDA Priority Review for New Drug Application: How are Gene Therapy Drug Approvals Impacting Stocks?

- February 15th, 2018

INN is taking a look at what other companies have received FDA approval for gene therapy drugs and how the approvals have shifted their stock prices—not only by increasing.

Genetic therapy could be one of the strongest methods to target disease and cancer treatments. Investors may be interested in following these genetic focused companies as they develop new therapies.

Agios Pharmaceuticals (NASDAQ:AGIO) a big pharma company focused on cancer metabolism, rare genetic diseases, and metabolic immuno-oncology announced on February 14 the FDA granted priority review for their NDA. This application was for Ivosidenib, a drug targeting the IDH1 mutation in patients with relapsed or refractory acute myeloid leukemia (AML). This type of leukemia is a blood and bone marrow cancer which spreads rapidly.

“IDH1 mutations occur in about 6 to 10 percent of AML patients. Recent publications have highlighted the advances in the understanding of the genetics underlying AML and the need for routine mutational analysis at diagnosis and relapse,” Agio said in the press release

2018 Genetics Market Report


Find out what to watch for in the world of genetics investing.


The company expects it will hear back about this NDA by August 21, 2018 with the priority review, which cuts the approval time by four months.

The drug is also being investigated other clinical studies such as patients with newly diagnosed AML, and those ineligible for intensive chemotherapy. The IDH1 mutation occurs in different cancers including cholangiocarcinoma, chondrosarcoma and glioma—which is a type of brain cancer, a skeletal system cancer, and a malignant brain tumour, respectively.

With Agios’ priority approval from the FDA, a number of other companies have also received FDA approval for gene therapy drugs, making the genetics industry an exciting sector for investors to keep an eye on in 2018.

Other companies with gene therapy drugs approved from the FDA

Another company revolutionizing the way medications can be personalized is Abeona Therapeutics (NASDAQ:ABEO). The company received an FDA designation for regenerative medicine advanced therapy for their gene therapy drug candidate EB-101 treating epidermolysis bullosa (EB) on January 29, 2018. This tissue disease mainly affects the skin by creating blisters, but internal organs and bodily systems can also be affected by EB—about 200 children a year are born with it in the US, according to the Dystrophic Epidermolysis Bullosa Research Association of America

“EB-101 is an autologous gene-corrected cell therapy approach that utilizes a patient’s own cells and genetically re-engineers them to produce the missing collagen protein, which helps hold skin on to the body.” said Timothy J. Miller, Ph.D., President and CEO of Abeona in the press release.

The company will continue with their clinical trials by moving on to Phase 3 of the drug with through the collaboration with the FDA.

2018 Genetics Market Report


Find out what to watch for in the world of genetics investing.


Spark Therapeutics (NASDAQ:ONCE) announced the news of the approval for Luxturna as a gene therapy for patients with RPE65 mutation-associated inherited retinal disease (IRD) on December 19, 2017 . This was pivotal for the FDA as it was the first gene therapy for a genetic disease approved; it’s also the first treatment for the RPE65 mutation IRD. IRD is “a group of rare blinding conditions caused by one of more than 220 different genes, often disproportionately affecting children and young adults,” the press release wrote.

Finally, the FDA additionally approved the first biosimilar—drugs that are biologically similar to drugs with expired patents—in December 2017, for the treatment of certain breast and stomach cancers. The drug Ogivri, created by Mylan (NASDAQ:MYL) and Biocon (NSE:BIOCON) is a biosimilar to Herceptin created by Roche’s (VTX:ROG) Genentech. This drug focuses on patients’ tumours which express the HER2 gene in each said type of cancer.

Investor Takeaway

Since Thursday’s  (February 15) announcement, shares of Agio increased 2.95 percent over a one-day period to close at $78.79. According to TipRanks, five analysts recommend this stock as a strong buy and one recommends to hold, with a 12-month prediction forecast of a high of $90.00.

For Abeona, when the news about the FDA designation was released in late January, its share price has dropped 25.85 percent to close at $12.05 on Thursday.

An article from Value Walk claims the drop may be due to manipulated data which analysts noticed on February 8, when the Phase 1/2 gene therapy trial of MPS IIIA was released. “Cohort 2 data was visibly mixed, with the strongest results coming from a disputed test method and with very negative indications coming from the industry standard test,” the article said.

On the other hand, Spark Therapeutics’ share price has increased  by 12.11 percent to $54.99 from $48.94 on December 19 , 2017 when its FDA approval was announced. TipRanks suggests a target price of $113 over the next year and shows 13 analyst recommend to buy, five to hold and one to sell.

Shares of Mylan and Biocon increased 14.02 percent to $41.65 and 38.41 percent to $9.42, respectively since the news was released about the FDA approval on December 1,  2017.

Roche’s price, however, has dropped nearly 10 percent from the same date, which may have been influenced by Mylan and Biocon’s biosimilar drug was approved by the FDA.

Investors may be interested in watching these companies mentioned along with others on our list of Gene Therapy Stocks to Watch to see where stock prices are going as the gene therapy market gets bigger.

Don’t forget to follow us @INN_LifeScience for real-time news updates!

All stock prices and quotes were taken from Google Finance as of February 15, 2018 after market close, unless otherwise noted.

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

2018 Genetics Market Report


Find out what to watch for in the world of genetics investing.


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