TREND: Smallcap biotechs win in stock markets on positive drug trials

pharmafile | March 21, 2016 | Feature | Research and Development, Sales and Marketing |   

Troubled Canadian drug firm Valeant Pharma (NYSE: VRX) saw its share price halved in a single day after the company announced disappointing outlook for 2016. However, it is nothing short of sunny for the small cap biotech companies, with stocks rallying as much as 400% following positive drug trial updates. Niche research in focussed areas yielding positive outcomes in mid to late stages, has meant the shareholders can now laugh all the way to the bank as sentiment drives the stock price up.

On March 17, shares in trial-stage biotechnology company Vitae Pharmaceuticals (Nasdaq: VTAE) jumped to double following news of success in mid-stage study for its psoriasis drug. 

Small cap firms with promising drug candidates have seen fortunes multiply with more than a handful witnessing a surge in stock price in March alone. With research showing positive outcomes for conditions ranging from psoriasis to leukaemia, stakeholders are raking in the spoils as the companies prepare for new drug launches.

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Here’s a quick look at the companies that saw their stocks move significantly, in some cases soaring up to four times in value, following drug-trial results.

The Big Gainers

Shares in Vitae Pharmaceuticals (Nasdaq: VTAE) shot up to nearly double after the company reported mid-stage trials of its psoriasis drug significantly reduced the skin condition.

The company said, patients taking 350 mg dose of drug candidate VTP-43742, demonstrated a 24% reduction in the psoriasis area severity index than the placebo, while for patients taking the 700 mg dose witnessed a 30% decrease.

US-based biotech firm Vericel (Nasdaq: VCEL) stock price nearly doubled following results of mid-stage study for its trial drug to treat heart condition. The company said the heart failure drug met the primary end-point in trials.

The maker of cell therapy products announced top-line results for its trial-drug ixmyelocel-T showed a reduction in the total number of deaths, cardiovascular hospitalizations or unplanned outpatient and emergency department visits to treat acute decompensated heart failure during the 12 months following treatment with ixmyelocel-T compared to placebo.

Vericel said the therapy was granted orphan drug status to treat dilated cardiomyopathy by the US Food and Drug Administration (FDA).

 Source: Pharmafile.com

Shares in UK-based GW Pharmaceuticals (Nasdaq: GWPH) more than doubled after the company announced positive results for late-stage trials for its investigational drug Epidiolex (cannabidiol or CBD) to treat Dravet syndrome, a rare and severe form of epilepsy in children.

The company said Phase III trials for its trial drug Epidiolex met the primary endpoint of a significant reduction in convulsive seizures assessed.

Epidiolex has both Orphan Drug Designation and Fast Track Designation from the US Food and Drug Administration (FDA) in the treatment of Dravet syndrome, a rare and debilitating type of epilepsy for which there are currently no treatments approved in the US.

In addition to this first Phase III trial, GW is also conducting a second Phase III trial in Dravet syndrome.

Stock price for trial-stage Celator Pharmaceuticals (Nasdaq: CPXX) soared gaining over four times in value as the company announced late-stage trials for its leukemia drug met its primary endpoint.

The company said following the trial results it will file a New Drug Application (NDA) for its drug compound Vyxeos (cytarabine/daunorubicin) with the US Food and Drug Administration (FDA) later in the year and submit a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in the first quarter of 2017.    

Sarepta Therapeutics (Nasdaq: SRPT) stock jumped nearly 15% as the US Food and Drug Administration (FDA) set April 25 as the date to  review the company’s trial drug to treat muscle disease.

Sarepta’s drug candidate Eteplirsen is in clinical trials for the treatment of Duchenne muscular dystrophy (DMD). The meeting was originally scheduled for January 22.

The Losers

Shares in Celldex Therapeutics (Nasdaq: CLDX) more than halved in value following news independent regulators have recommended the discontinuation of late-stage trial of its brain cancer drug on the back of disappointing study results.

The company said it received the data for its Rintega (rindopepimut) on March 4, and is in the process of reviewing the results.

PTC Therapeutics (Nasdaq: PTCT) said it does not expect the Canadian regulators to review its trial drug to treat a genetic muscle-wasting condition in the first half of 2016 as it plans to submit results from a new trial. Shares in the company dropped over 10% on the news.

The company said it plans a New Drug Submission (NDS) following the results of the late-stage trials for its Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD).

Anjali Shukla

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