Week Ahead In Pharma: Data Readouts, Results From Interim Analysis, ERS Congress Presentation

biotech sept04 lt

The week that passed by saw shares of Aimmune Therapeutics (AIMT) make the biggest weekly advance, gaining over 170%, as the peanut allergy drug maker is all set to be acquired by Nestle Health Science for $34.50 per share in cash. TRACON Pharmaceuticals Inc. (TCON), Akcea Therapeutics Inc. (AKCA), and Corcept Therapeutics Incorporated (CORT) are the other pharma stocks that ended with a weekly gain of more than 50%.

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

1. Applied Genetic Technologies Corp. (AGTC)

Applied Genetic Technologies is a clinical-stage biotechnology company developing gene therapies for rare diseases. The most advanced clinical program is AGTC-501 for the treatment of X-linked retinitis pigmentosa, an inherited retinal disease.

AGTC-501 is under a phase I/II trial and encouraging interim six-month data from this trial was reported in January of this year. The data suggest durable and meaningful improvements in central visual sensitivity, according to the company. The trial involves 6 dose groups and 28 patients.

X-linked retinitis pigmentosa, which is associated with degeneration of rods and cones, leads to early night blindness and progressive constriction of visual fields. This rare disease affects an estimated 20,000 patients in the U.S. and the EU.


Applied Genetic Technologies is scheduled to provide a re-analysis of dose Groups 2 and 4 data, and new preliminary visual sensitivity data from Group 5 of the phase I/II trial of AGTC-501 on September 9.

The company will also provide a management update on its planned phase II/III X-Linked Retinitis Pigmentosa (XLRP) clinical trial design.

Cash position:

The company's cash, cash equivalents, and investments as on March 31, 2020, totaled $84.5 million.

AGTC closed Friday's trading at $5.86, up 0.69%.

2. Corbus Pharmaceuticals Holdings Inc. (CRBP)

Corbus Pharmaceuticals is a clinical-stage drug development company focusing on inflammatory and fibrotic diseases.

The company's lead product candidate is Lenabasum that is currently being evaluated in systemic sclerosis, cystic fibrosis, dermatomyositis, and systemic lupus erythematosus.

Lenabasum is under a phase III trial in systemic sclerosis, dubbed RESOLVE-1, in a phase III study in dermatomyositis, dubbed DETERMINE, and in phase IIb studies in cystic fibrosis and systemic lupus erythematosus.


The topline data from the RESOLVE-1 phase III study of Lenabasum for treatment of systemic sclerosis and phase IIb study cystic fibrosis are expected this month.

Cash position:

The company's cash and cash equivalents on hand on July 28, 2020 were about $101 million.

CRBP closed Friday's trading at $9.25, down 1.39%.

3. Sutro Biopharma Inc. (STRO)

Sutro Biopharma is a clinical-stage company developing next-generation cancer and autoimmune therapeutics.

Two of the company's internally-developed compounds - STRO-001 and STRO-002 - are in phase I development. STRO-001 is a CD74-targeting antibody-drug conjugate (ADC) currently being investigated in patients with advanced B-cell malignancies, including multiple myeloma and non-Hodgkin lymphoma. STRO-002 is a folate receptor alpha (FolRa)-targeting ADC being tested in patients with ovarian and endometrial cancers.


The company is slated to discuss updated data from its ongoing phase I dose-escalation study of STRO-002 in ovarian and endometrial cancer on September 9.

The interim data regarding safety and anti-tumor activity results in heavily pre-treated patients with ovarian cancer from the trial were reported in April of this year. The results demonstrated preliminary evidence of antitumor activity and clinical benefit, according to the company.

Cash position:

Cash, cash equivalents, and marketable securities as of June 30, 2020, were $207 million.

STRO closed Friday's trading at $12.54, down 3.54%.

4. Tonix Pharmaceuticals Holding Corp. (TNXP)

Tonix Pharma is a clinical-stage biopharmaceutical company focusing on immunology and central nervous system diseases.

The company's lead drug candidate is TNX-102 SL for the management of fibromyalgia, which is under a phase III trial, dubbed RELIEF.

Fibromyalgia is a multi-symptom disorder that originates in the central nervous system and is characterized by widespread pain, non-restorative sleep, fatigue, and disability.

In an earlier phase III trial of TNX-102 SL in fibromyalgia, dubbed AFFIRM, the primary efficacy endpoint did not achieve statistical significance.


The results from an interim analysis of the RELIEF study are expected this month. The interim analysis conducted by an Independent Data Monitoring Committee will make one of four recommendations: (1) stop the study for success; (2) continue the study as planned; (3) continue to enroll with a specified increase in the total number of participants in the full study; or (4) stop the study for futility.

Cash position:

The company ended June 30, 2020, with cash and cash equivalents of $55.0 million.

TNXP closed Friday's trading at $0.82, down 5.60%.

5. Verona Pharma plc (VRNA)

Verona Pharma is a clinical-stage biopharmaceutical company developing therapies for the treatment of respiratory diseases with significant unmet medical needs.

The company's lead investigational drug is Ensifentrine which is being explored in the indications of COPD, COVID-19, and other respiratory diseases, including asthma and cystic fibrosis.

Ensifentrine, which is in phase IIb clinical development for the maintenance treatment of chronic obstructive pulmonary disease (COPD), is being tested in three formulations - nebulized, dry powder inhaler (DPI), and pressurized metered-dose inhaler (pMDI).


The company is scheduled to present new subgroup analysis from phase IIb trials with nebulized Eensifentrine in chronic obstructive pulmonary disease at the European Respiratory Society ("ERS") International Congress on Tuesday, September 8.

The company first reported positive data from the phase IIb COPD study with nebulized Ensifentrine in March 2018 and then in January 2020.

Cash position:

The company's cash on hand on June 30, 2020, amounted to $22.4 million.

VRNA closed Friday's trading at $6.55, down 2.09%.

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