These public companies climbed highest in a year when biotech stocks scaled new heights.

In a year that saw biotech stocks rise to uncharted heights, it shouldn’t be surprising that this year’s top 10 Wall Street Winners—the 10 biotech, pharma, tools/tech, and services (CROs) company stocks that gained the most in value during 2014 (measured over a one-year period ending December 19)—mostly rose higher than last year’s Top 10 Wall Street Winners of 2013.

This year’s top gainer, for example, finished 487% above its year-ago price, compared with 439% for last year’s #1 Wall Street winner, Insys Therapeutics. And while this year’s #10 finished slightly lower than last year’s (277.1% vs. 291.1% for Prothena on the 2013 list), it is still true that the lowest-ranked among the top 10 much more than tripled their share price over a 12-month period—indeed, nearly quadrupled it.

Following is GEN’s list of the top 10 best stock performers of 2014. Companies are listed by name; their stock exchange and trading symbol; the closing price on December 19, 2014, and December 19, 2013; the percentage of change between the closing prices; and a brief explanation for the companies’ good fortune.

The top performers are up-and-coming companies, small- to mid-capitalization (“small cap” to “mid cap”) stocks that jumped in value as a result of some sort of good news—from marketing approvals, to encouraging clinical trials results, to rumors of an acquisition by a much larger company. This year’s #1 and #10 companies will likely disappear from next year’s List of Wall Street Winners, since both are being acquired by larger biopharmas. Not included on this list are companies that went public during 2014, as a result generating less than a year’s share-price data.


#10. Prosensa Holding

NASDAQ: RNA

December 19, 2014: $18.78

December 19, 2013: $4.98

% Change: 277.1%

Like the proverbial phoenix, the company’s Duchenne muscular dystrophy (DMD) candidate drisapersen rose from the ashes of a 2013 Phase III failure that led to the end of its collaboration with GlaxoSmithKline (and caused its share price to free-fall 70% on a single day). And while the comeback wasn’t immediate, the company’s shares started rising in earnest May 27-30, when the price zoomed almost 39% to $11.03; the company basked in investor bullishness on DMD drugs after Translarna (PTC Therapeutics) was approved by the European Commission. Not surprisingly, the company enjoyed a single-day nearly 63% gain, to $18.60, on November 24, the day it announced it was being acquired by BioMarin Pharmaceutical for up to $840 million–$680 million upfront, $160 in milestone payments tied to approvals of drisapersen in the U.S. ($80 million) and Europe ($80 million).


#9. Amicus Therapeutics

NASDAQ: FOLD

December 19, 2014: $8.03

December 19, 2013: $2.12

% Change: 278.8%

While “FOLD” may be the company’s stock symbol, shares did anything but that in 2014, as prices increased more or less steadily. The biggest leap occurred over 21 days ending August 28, when shares climbed about 81% to $7.27. The period was bookended by strong second-quarter results August 7, and especially positive Phase III data August 20 from its second study of its migalastat in Fabry patients with amenable mutations; migalastat met its co-primary endpoints of comparability to enzyme replacement therapy on two measures of kidney function after 18 months. After backsliding in September and October, shares jumped 18%, to $6.70 on November 17, the first trading day after release of more positive migalastat data concerning secondary cardiac and composite endpoints. Over 12 days in November (Nov. 14-26), shares rose 43% to $8.13.


#8. Pernix Therapeutics

NASDAQ: PTX

December 19, 2014: $9.65

December 19, 2013: $2.46

% Change: 292.3%

Several announcements had the effect of raising the company’s share price, starting Feruary. 5 with its naming Doug Drysdale as CEO and disclosing a $65 million institutional investment toward working capital and expansion capital for future acquisitions of specialty products. Investors responded by driving up shares 69% to $3.53. From March 17, when it reported finishing 2013 with a 38% jump in revenues, to March 21, the stock climbed 58% to $5.93. Two months later, on May 15, the stock climbed 15% to $6.43, a day after the company bolstered its pipeline by acquiring Treximet® tablets for migraine from GlaxoSmithKline for $250 million upfront. The company credited its Treximet launch with a Q3 net revenue increase of 72% year-over-year on November 10, prompting more investor buys as shares rose 14.5% to $11.35.


#7. bluebird bio

NASDAQ: BLUE

December 19, 2014: $92.00

December 19, 2013: $20.51

% Change: 348.6%

Shares spiked upward twice in 2014 starting in June. The stock price rose 32%, to $34.46 on June 16, the first trading day after the company presented initial positive clinical data from its HGB-205 clinical study of its lentiviral gene therapy product candidate LentiGlobin BB305 in patients with beta-thalassemia major. The best was yet to come, as shares ballooned 83% to $84.28 on December 9, the next trading day after the company said the first four subjects treated with BB305 produced enough hemoglobin to reduce or eliminate the need for transfusion support after at least three months of follow up.


#6. Achillion Pharmaceuticals

NASDAQ: ACHN

December 19, 2014: $14.21

December 19, 2013: $3.13

% Change: 354.0

After an up-and-mostly-down first five months, shares rocketed 83%, to $7.79 on June 10, after the company announced a double-dose of good news: It began dosing the first patients with its ACH-3422 in an ongoing Phase I trial of the candidate for genotype 1 chronic hepatitis C viral infection (HCV), while the FDA lifted its clinical hold on sovaprevir, allowing its use on HCV patients in trials. Shares also climbed 27% on November 10, the first trading day after presenting encouraging data showing Its ACH-3102 with sofosbuvir met its primary endpoint for its ongoing Phase I trial evaluating the combination for genotype 1 HCV. All 12 patients achieved sustained viral response 12 weeks after completion of therapy


#5. TG Therapeutics

NASDAQ: TGTX

December 19, 2014: $16.45

December 19, 2013: $3.59

% Change: 358.2%

A series of positive announcements drew investors to the company’s stock in 2014. Shares jumped almost 32%, to $9.61, from June 11 to June 13, when the company announced a 90% overall response rate at an ongoing Phase II trial’s first efficacy assessment for the combination of its TG-1101 (Ublituximab) and the Pharmacyclics/Janssen drug Ibrutinib—six of seven Chronic Lymphocytic Leukemia (CLL) and all three Mantle Cell Lymphoma (MCL) patients. Shares also rose 16%, to $11.60, on Sept. 17, two days after news of a sooner-than-expected agreement with the FDA on a Phase III trial protocol for TG-1101. And after upbeat Phase I/II results announced December 9 from a combination of TG-1101 and another company drug candidate, TGR-1202, in patients with B-cell malignancies, shares leaped 30.5% over three days, to $18.25 on December 12.


#4. Receptos

NASDAQ: RCPT

December 19, 2014: $133.61

December 19, 2013: $25.36

% Change: 426.9%

After an up-and-down first five months, shares began climbing steadily in June. Shares jumped 41% to $95.76 on Oct. 28, a day after the company reported positive results from the Phase II TOUCHSTONE trial for its ulcerative colitis drug candidate RPC1063. The trial met its primary endpoint and all secondary endpoints with statistical significance in patients on the 1 mg dose of RPC1063 in the 8-week induction period, the company announced. Following the clinical trial update, Credit Suisse raised its target price on the company’s stock from $75 to $125, while maintaining its “Outperform” rating.


#3. OvaScience

NASDAQ: OVAS

December 19, 2014: $47.85

December 19, 2013: $8.89

% Change: 438.2%

After largely stable prices during the first half of 2014, shares began climbing consistently in August following upbeat second-quarter results and progress toward the international rollout of its fertility treatments. In December, the company rang out the year with a one-two punch that doubtless pleased investors: A 19% jump, to $34.96, on December 17 following the company announcing it was able to advance all three of its fertility treatment options—AUGMENTSM, OvaPrimeSM and OvaTureSM. A day later, shares zoomed about 24%, to $43.22, after Wedbush Securities upgraded its rating of the company’s shares from Neutral to Outperform and predicted a price target of $47. Two other investment firms more than doubled their price targets for the company—Leerink (from $23 to $56), and H.C. Wainwright (from $40 to $100).


#2. Agios Pharmaceuticals

NASDAQ: AGIO

December 19, 2014: $119.62

December 19, 2013: $21.79

% Change: 449.0%

Agios saw less of a jolt than a series of increases in its stock price that added up. On April 7, shares closed up 28% at $45.35 following announcement the previous day of positive results from preliminary Phase I data for AG-221, developed in collaboration with Celgene. Of seven evaluable patients, three experienced complete remission, while another two reported complete remissions with incomplete platelet recovery. AG-221 is a first-in-class inhibitor of isocitrate dehydrogenase-2 (IDH2) mutations in patients with advanced blood cancers. Investors speculated on more good news September 26, when the share price rose 19%, to $63.76, after the company said it would release initial clinical data from a Phase I study of AG-120, in November. The November 18 announcement was indeed good news: Four complete remissions among responses in seven of 14 patients studied. Investors responded the next day by sending shares 14.5% higher, to $95.97.


#1. Avanir Pharmaceuticals

NASDAQ: AVNR

December 19, 2014: $16.85

December 19, 2013: $2.87

% Change: 487.1%

Investors no doubt welcomed the nearly 13% jump (to $16.92) in share price when the company announced its planned acquisition for $3.5 billion by Otsuka Pharmaceutical on December2. Yet that wasn’t the biggest increase Avanir shares would enjoy in 2014. That distinction occurred almost two months earlier on September 15, when shares jumped 85%, to $12.49 after the company disclosed positive results from its Phase II clinical trial evaluating the safety and efficacy of AVP-923 for the treatment of agitation in patients with Alzheimer's disease: AVP-923 use correlated with significantly reduced agitation as measured by the primary endpoint, the agitation/aggression domain score of the neuropsychiatric inventory (NPI) compared to placebo. Alzheimer’s is a notoriously difficult indication for developing new drugs: In July, a Cleveland Clinic study found a 99.6% failure rate of clinical trials for Alzheimer's drug candidates between 2002 and 2012.
































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