U.S. Food and Drug Administration (FDA) rulings and the results from clinical trials have proven time and again that they can make or break companies. The biotech and pharmaceutical companies that subject themselves to this lengthy process are taking on a fair amount of risk of failure, but if a drug is approved or passes a clinical trial, there can be massive upside.

24/7 Wall St. has collected several big FDA decisions and mid- to late-stage trials that should be coming up in November and December, as well as added some color with the trading range and price target. Also check out October’s FDA decisions and trial results.

November and December signify the end of the year and a time when many biotech and pharmaceutical companies expect to produce results or receive approvals — this time of the year perhaps more than any other time.

As a side note about the Prescription Drug User Fee Act (PDUFA): a Priority Review designation is granted to medicines that the FDA determines have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease.

Medicines

Medicines Co. (NASDAQ: MDCO) has two Phase 2 studies expected to come out by November 15. First the company is conducting and presenting an interim analysis of its Orion-1 Phase 2 trial of  single or multiple subcutaneous injections of PCSK9si in patients with atherosclerotic cardiovascular disease (ASCVD) or ASCVD-risk equivalents (diabetes and familial hypercholesterolemia). Additionally, the company anticipates that top-line data from Day 180 follow-up for up to 200 patients will be presented at the Late-Breaking Clinical Trial Session and Day 180 follow-up in all 501 patients will be completed, analyzed and top-line results disclosed before the end of 2016.

As for the second Phase 2 study, the company expects to present to post results from its Milano-Pilot study for MDCO-216, which is a complex of dimeric recombinant apolipoprotein A-1 Milano (ApoA-1 Milano) and a phospholipid and is currently under development to improve cardiovascular outcomes by reducing plaque burden in patients with atherosclerotic disease. This also will take place at the Late-Breaking Clinical Trial Session at the AHA Scientific Sessions on November 15.

Shares of Medicines most recently closed at $38.51, with a consensus analyst price target of $50.80 and a 52-week trading range of $27.50 to $38.90.

Rigel Pharmaceuticals

At the end of August, Rigel Pharmaceuticals Inc. (NASDAQ: RIGL) announced that fostamatinib, its oral spleen tyrosine kinase inhibitor, met the primary endpoint in the first of two double-blind studies in the FIT Phase 3 clinical program for the treatment of adult chronic/persistent immune thrombocytopenia. The results from the second FIT Phase 3 study are expected in October/November 2016. November 30 is listed as the last possible date based on guidance.

Shares of Rigel last closed at $4.01. The consensus price target is $8.25, and the 52-week range is $1.88 to $4.38.

Galena Biopharma

Galena Biopharma Inc. (NASDAQ: GALE) currently has two investigator-sponsored trials ongoing with NeuVax in combination with trastuzumab (Herceptin). The combination of trastuzumab and NeuVax has been shown pre-clinically and in a pilot study to be synergistic. The Phase 2b clinical trial is an investigator-sponsored, 300 patient study enrolling HER2 1+ and 2+, HLA A2+, A3+, A24 and/or A26, node positive, and high-risk node negative patients, in the treatment of breast cancer. The endpoint is disease free survival and preliminary safety data will be presented in the fourth quarter of this year. The primary endpoint of the study is disease-free survival. Genentech/Roche are providing the trastuzumab and partial funding for this trial.

Shares of Galena closed at $0.33, with a consensus price target of $1.13 and a 52-week range of $0.28 to $2.49.

Ariad Pharmaceuticals

Ariad Pharmaceuticals Inc. (NASDAQ: ARIA) announced that the first patient in its Phase 2 Optic trial of Iclusig (ponatinib) for the treatment of patients with refractory, chronic-phase chronic myeloid leukemia. The trial is expected to inform the optimal use of Iclusig in these patients. Results from this trial are expected by the end of 2016. Frank G. Haluska, M.D., Ph.D., senior vice president of clinical research and development and chief medical officer at Ariad, added:

The Optic trial is expected to provide important data regarding the efficacy and safety of starting doses of ponatinib lower than the currently approved dose of 45 mg/day. The trial will give us the opportunity to prospectively evaluate strategies for dose reductions after patients have achieved a major cytogenetic response, with a goal of minimizing risk, while helping to maintain patients on a lower dose and to optimize therapy.

Shares of Ariad were last seen at $13.87, with a consensus price target of $11.63 and a 52-week range of $4.37 to $14.26.

Syngery Pharmaceuticals

Syngery Pharmaceuticals Inc. (NASDAQ: SGYP) has just announced that it will present new data on plecanatide, an investigational compound for the treatment of chronic idiopathic constipation and irritable bowel syndrome with constipation (IBS-C), at the American College of Gastroenterology (ACG) annual scientific meeting in Las Vegas in mid-October. Looking even farther ahead, it has a set PDUFA target action date for January 29, 2017. Also Synergy plans to file a New Drug Application Supplement with Clinical Data (sNDA) for plecanatide in IBS-C in the first quarter of 2017 and expects a 10-month review period from submission.

Shares of Synergy Pharma last closed at $5.63, with a consensus price target of $9.90 and a 52-week range of $2.50 to $7.15.

Horizon Pharma

Horizon Pharma PLC (NASDAQ: HZNP) completed its target enrollment of its Phase 3 study evaluating Actimmune (interferon gamma-1b) for the treatment of people with Friedreich’s ataxia, a degenerative neuro-muscular disorder. The study reached its target enrollment of 90 patients at four sites in the United States, and top-line results are expected by the end of 2016. Because enrollment was completed back in May, these results should be due relatively soon.

Shares of Horizon recently closed at $19.25, in a 52-week range of $12.86 to $23.70. The consensus price target is $31.33.

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CytRx

CytRx Corp. (NASDAQ: CYTR) is waiting for its second analysis for its Phase 3 trial of aldoxorubicin in second-line soft tissue sarcoma (STS) in the fourth quarter of 2016. Also its single-agent aldoxorubicin trials in glioblastoma and Kaposi’s sarcoma, along with its combination trials in STS and metastatic solid tumors are fully enrolled and either completed or near completion.  The Phase 2b second-line small cell lung cancer aldoxorubicin trial is enrolled, and the company currently anticipates reporting top-line results in this time as well.

Shares of CytRx were last trading at $0.58, with a consensus price target of $4.58 and a 52-week range of $0.55 to $3.66.

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