Alex Philippidis Senior News Editor Genetic Engineering & Biotechnology News

Is greed good? It is for these biotech and biopharma firms.

Following is a list of the top 10 best stock performers of 2013—the 10 biotech, pharma, tools/tech, and services (CROs) company stocks that gained the most in value during 2013 (as of December 13). Companies are listed by name; their stock exchange and trading symbol; the closing price on December 13, 2013, and December 13, 2012; 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. One of the 10 (Gentium) will likely disappear from the next update of this GEN List, since it has entered into an agreement to be acquired by Jazz Pharmaceuticals for about $1 billion.

Not listed are companies that appeared to show even larger price gains, but only as a result of reverse stock splits, since the adjusted closing prices resulting from the splits showed much smaller year-to-year gains for investors.

#10. Prothena


Closing price on December 13, 2013: $28.16

Closing price on December 21, 2012: $7.201

% Change: 291.1%

2013 highlights: The company capped a near-year as a drug discoverer spinout of Elan (recently acquired by Perrigo for $8.6 billion) on December 12, by inking an up-to-$600 million deal with Roche to co-develop and co-commercialize antibodies that target alpha-synuclein—including PRX002, Prothena’s preclinical monoclonal antibody for Parkinson’s disease. The company in April launched its Phase I trial assessing safety and tolerability of NEOD001 in AL amyloidosis, with data to come in 2014—by which time Prothena expects to move forward with an IND submission and start of clinical trials for PRX002.

#9. BioCryst Pharmaceuticals


Closing price on December 13, 2013: $6.05

Closing price on December 13, 2012: $1.51

% Change: 300.7%

2013 highlights: Like Lazarus and the 1978 New York Yankees, BioCryst’s peramivir seemingly rose from the dead in April, after the company announced FDA’s finding that the intravenous flu drug candidate’s NDA “supports a reviewable NDA submission” for acute uncomplicated influenza. Five months earlier, the company all but buried peramivir’s development effort, leading to half its staff being downsized. In September, BioCryst won a five-year, up-to-$22 million contract ($5 million upfront) with NIH’s National Institute of Allergy and Infectious Diseases to develop BCX4430, the lead compound in the company’s broad-spectrum antiviral research program. In November, the company launches the Phase IIa OPuS-1 proof-of-concept trial for oral BCX4161 in patients with hereditary angioedema (HAE). And the stock reached $6.91 on December 18 after two optimized plasma kallikrein inhibitors were advanced into preclinical development as potential once-daily, oral treatments for prevention of HAE attacks.

#8. Catalyst Pharmaceutical Partners


Closing price on December 13, 2013: $1.92

Closing price on December 13, 2012: $0.46

% Change: 317.4%

2013 highlights: The company, and its stock, enjoyed a comeback year following a disappointing 2012, when a Phase IIb candidate for cocaine addiction missed its endpoint. Catalyst reported progress in the clinical development of its lead product, Firdapse for Lambert-Eaton Myasthenic Syndrome (LEMS), now in a Phase III trial expected to report top-line data during the second quarter of 2014. Catalyst won “Breakthrough Therapy Designation” from FDA for Firdapse, which is the first and only European-approved drug for adults with LEMS, and is licensed from BioMarin Pharmaceutical.

#7. Idera Pharmaceuticals


Closing price on December 13, 2013: $3.09

Closing price on December 13, 2012: $0.72

% Change: 329.2%

2013 highlights: Company opened patient enrollment in December in a Phase I/II trial of IMO-8400 in patients with Waldenström’s macroglobulinemia, following acceptance of its NDA by FDA. In July, Idera reported positive results from a Phase I trial, namely inhibition of toll-like receptor (TLR) 7-, 8-, and 9-mediated cytokines, including tumor necrosis factor-alpha (TNF-α), interleukin-1 beta (IL-1β), interleukin-6 (IL-6), interferon-alpha (IFN-α), and other pro-inflammatory cytokines. A month later, the National Cancer Institute agreed to evaluate Idera’s TLR antagonists as a potential approach to the treatment of certain genetically defined B-cell lymphomas.

#6. Keryx Biopharmaceuticals


Closing price on December 13, 2013: $12.17

Closing price on December 13, 2012: $2.83

% Change: 330.0%

2013 highlights: FDA in October accepts for filing the company’s NDA for Zerenex (ferric citrate coordination complex) for hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis. Zerenex’ PDUFA goal date is June 7, 2014, with a product launch expected in mid-2014. In advance of that launch, the company said it hired professionals with backgrounds in dialysis and CKD. In November, Keryx trumpets positive Phase II top-line results, meeting its co-primary endpoints of statistically significant changes in both serum phosphorus and transferrin saturation versus placebo after 12 weeks of treatment.

#5. Puma Biotechnology


Closing price on December 13, 2013: $86.92

Closing price on December 13, 2012: $19.49

% Change: 346.0%

2013 highlights: Launches Phase III trial in June of its lead drug candidate PB272 (neratinib) in patients with HER-2 positive metastatic breast cancer who have failed two or more prior treatments. Neratinib “graduates” from the Phase II I-SPY 2 trial in December toward a future I SPY 3 Phase III trial—but based on showing Bayesian predictive probability that a regimen will show statistical superiority in a 300-patient confirmatory trial. The nontraditional methodology sparks controversy—but may explain why shares performed even better after December 13, closing December 20 at $101.39.

#4. Acadia Pharmaceuticals


Closing price on December 13, 2013: $23.03

Closing price on December 13, 2012: $4.78

% Change: 381.8%

2013 highlights: FDA shortened the company’s timeline to a marketing decision on pimavanserin for Parkinson’s disease psychosis in April by agreeing to accept for NDA purposes data from the Phase III -020 study, together with supportive data from other studies, rather than insist the company launch another Phase III trial, “-021”, that was panned as a confirmatory trial. A month earlier, share prices jumped 28% after the company reported positive Phase III data from the -020 trial.

#3. Gentium


Closing price on December 13, 2013: $56.90

Closing price on December 13, 2012: $11.46

% Change: 396.5%

2013 highlights: Gentium’s biggest highlight came six days after December 13, when Jazz Pharmaceuticals announced it was acquiring the company for about $1 billion; the deal is expected to close in 2014. In October, Gentium won European marketing approval for its lead product Defitelio (defibrotide) for the rare disorder of severe hepatic veno-occlusive disease (VOD) in adults and children undergoing hematopoietic stem cell transplantation.

#2. Galectin Therapeutics


Closing price on December 13, 2013: $8.27

Closing price on December 13, 2012: $1.60

% Change: 416.9%

2013 highlights: The company released positive preclinical data in August showing that treatment with anti-galectin therapy GR-MD-02 showed a robust effect in reducing lung fibrosis in a model, with somewhat lesser effect of GM-CT-01. On April 29, researchers from Galectin joined their collaboration partners at the Icahn School of Medicine at Mount Sinai at the International Liver Congress in Amsterdam, where they presented data showing the two drugs reversed cirrhosis of the liver in experimental animals (April 29).

#1. Insys Therapeutics


Closing price on December 13, 2013: $38.06

Closing price on December 13, 2012: $7.06

% Change: 439.1%

2013 highlights: Shares traded even higher ($48.82 close on December 9) until the company said December 12 it received a subpoena from the Office of Inspector General of the Department of Health and Human Services requesting documents regarding Subsys, including Insys’ sales and marketing practices relating to the cancer pain drug. Subsys’ sales success helped company shares climb earlier, as did the company reporting its first profitable quarters, and the closing of an IPO that raised gross proceeds of $36.8 million.

1 Prothena began trading shares on December 21, 2012, following Elan’s spinoff of what it termed a substantial portion of its drug discovery business into a new, publicly traded independent company.

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