Week Ahead In Pharma: Data Readouts, FDA Decision (AQST, BCRX, ETON...)

(RTTNews) - In the week that passed by, Cassava Sciences Inc. (SAVA) made the biggest weekly advance, with the shares surging over 200%, thanks to promising final results of a phase IIb study of its lead drug candidate, Sumifilam, in Alzheimer's disease, and news of insider purchase. Novus Therapeutics Inc. (NVUS), which made a weekly gain of 133%, and Immunomedics Inc. (IMMU), which returned 133% are the other top weekly gainers in the healthcare sector.

Now, let's take a look at some of the stocks that merit attention in the coming week.

1. Agios Pharmaceuticals Inc. (AGIO)

Agios is a revenue-generating company developing novel investigational medicines to treat malignant hematology, solid tumors, and rare genetic diseases.

The company markets TIBSOVO, which is approved for the treatment of acute myeloid leukemia in adults with an IDH1 mutation, and Idhifa, which is approved for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) with an IDH2 mutation.

TIBSOVO is also being tested as a potential treatment for cholangiocarcinoma patients with an isocitrate dehydrogenase 1 (IDH1) mutation. Cholangiocarcinoma is a group of cancers that begin in the bile ducts. (Source: NIH). The company reported positive results from a global phase III trial of TIBSOVO in previously treated cholangiocarcinoma patients with an isocitrate dehydrogenase 1 (IDH1) mutation, dubbed ClarIDHy, last May.


The mature overall survival data from the ClarIDHy phase III study due this quarter is awaited. If the data are supportive, the company plans to file a supplemental new drug application for TIBSOVO in previously treated IDH1-mutant cholangiocarcinoma in the first quarter of 2021.

AGIO closed Friday's trading at $38.46, down 0.54%.

2. Aquestive Therapeutics

Aquestive Therapeutics Inc.'s (AQST) lead drug candidate Libervant awaits the decision of the FDA on September 27, 2020.

Libervant is proposed for the rapid treatment of acute uncontrolled seizures in refractory patients with epilepsy on stable anti-epileptic drugs.

The current standard of care rescue therapy for patients with refractory epilepsy is Diastat, a diazepam based rectal gel that is invasive, inconvenient, and difficult to administer.

If approved by the FDA, Libervant will an alternative to Diastat and will be the first oral diazepam-based therapy approved for the management of seizure clusters in a population of 1.2 million refractory epilepsy patients.

AQST closed Friday's trading at $9.05, up 1.57%.

3. Atara Biotherapeutics Inc. (ATRA)

Atara Biotherapeutics is a phase III allogeneic T-cell immunotherapy company developing novel allogeneic transformative therapies for patients with severe diseases including solid tumors, hematologic cancers, and autoimmune diseases.

The company's lead drug candidate is Tab-cel, under phase III development for Epstein-Barr virus-driven post-transplant lymphoproliferative disease (EBV+ PTLD).

Epstein-Barr virus, or EBV, the first human oncovirus, has been implicated in the development of a wide range of diseases, including lymphomas and other cancers. Although this virus is kept under check in people with normal-functioning immune system, immunocompromised patients, such as those undergoing hematopoietic cell transplants, or HCT, or solid organ transplants, or SOT, have a reduced ability to control it. EBV transformed cells can, in some patients, proliferate and cause an aggressive, life-threatening cancer called EBV+ PTLD.


An interim analysis of the Tab-cel phase III study is due this quarter after which the company will discuss the totality of Tab-cel data with the FDA in a pre- biologics license application (BLA) meeting prior to initiating the BLA submission.

The company is on track to initiate the BLA submission of Tab-cel for patients with EBV+ PTLD by the end of this year.

ATRA closed Friday's trading at $15.87, up 0.44%.

4. BioCryst Pharmaceuticals Inc. (BCRX)

BioCryst Pharma is a clinical-stage company developing oral, small-molecule medicines that treat rare diseases.

The company's lead drug candidate is ORLADEYO, an oral treatment for hereditary angioedema, under FDA review, with a decision expected on December 3, 2020.

Also in the pipeline is BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases. On May 6, 2020, the company reported proof of concept data for BCX9930 in three treatment-naïve paroxysmal nocturnal hemoglobinuria (PNH) patients in the lowest dose cohort of 50 mg and 100 mg. An additional treatment-naïve PNH cohort, evaluating 200 mg BCX9930 followed by 400 mg twice-daily dose is also underway.


Data from the proof of concept trial of BCX9930 at 200/400mg dosage in treatment-naïve paroxysmal nocturnal hemoglobinuria patients due this quarter is awaited.

BCRX closed Friday's trading at $4.01, up 0.50%.

5. Eton Pharmaceuticals Inc. (ETON)

Eton Pharma is a revenue-generating specialty pharmaceutical company and it has six product candidates submitted to the FDA and two additional product candidates that are expected to be submitted before the end of this year.


The FDA decision on EM-100, an over-the-counter preservative-free formulation of ketotifen ophthalmic solution, proposed for the treatment of ocular itching associated with allergic conjunctivitis, due on September 15 is yet to be announced.

Eton Pharma out-licensed EM-100 to Bausch Health Companies Inc. (BHC) in 2019.

ETON closed Friday's trading at $9.06, up 11.51%.

6. Myovant Sciences (MYOV)

Myovant is a healthcare company developing innovative treatments for women's health and prostate cancer.

The company's lead drug candidate is Relugolix being explored as a treatment of uterine fibroids, endometriosis, and advanced prostate cancer.

The New Drug Application for Relugolix monotherapy as a treatment for advanced prostate cancer is under FDA review, with a decision expected on December 20, 2020. The FDA decision on the New Drug Application for a once-daily Relugolix combination tablet for the treatment of women with heavy menstrual bleeding associated with uterine fibroids is expected on June 1, 2021.


Data from an additional key secondary endpoint, castration resistance-free survival, in the phase III trial of Relugolix in advanced prostate cancer, dubbed HERO, due this quarter are awaited. The NDA submission of Relugolix in advanced prostate cancer was based on positive results from the phase III HERO study.

MYOV closed Friday's trading at $21.99, up 4.12%.

7. Ovid Therapeutics Inc. (OVID)

Ovid Therapeutics is a biopharmaceutical company developing medicines that transform the lives of people with rare neurological diseases.

The company's lead drug candidate is OV101, currently in a pivotal phase III trial for the treatment of Angelman syndrome and it successfully completed a phase II study in Fragile X syndrome in May this year.

OV935 is another drug candidate (soticlestat) in the pipeline and is being developed in collaboration with Takeda Pharma. This compound is under phase II trial for the treatment of CDKL5 deficiency disorder (CDD) and Dup15q syndrome, dubbed ARCADE, and a phase II trial in children with Dravet syndrome (DS) or Lennox-Gastaut syndrome, which was completed in August this year.


Results from the phase II ARCADE trial in CDKL5 deficiency disorder (CDD) and Dup15q syndrome, due this quarter, are awaited.

OVID closed Friday's trading at $6.69, unchanged from the previous day's close.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


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