Companies in the pharmaceutical industry generally are involved in the lengthy process of getting their drug candidates to market through clinical trials. There can be a fair amount of risk or massive risk involved, as many emerging biotech companies have their entire future based on one drug candidate. On the other hand, if a drug is approved or passes a clinical trial, then there can be incredible upside, depending on a few factors. The reality is that U.S. Food and Drug Administration (FDA) rulings can make or break some companies — with the future in question or with double-digit or even triple-digit gains.

24/7 Wall St. has collected eight key FDA decisions coming up in 2016 or 2017. We have added some color, along with the trading range and price target. Note that, due to many outside and internal factors, there are no assurances that the dates will not change (as Puma just witnessed).

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.

Vertex Pharmaceuticals

By the end of 2016, Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) is expected to report its Phase 3 interim analysis of VX-661 for the treatment of cystic fibrosis (CF). However, the company currently has a broad Phase 3 program, meaning multiple late-stage trials for different groups of patients with CF who have at least one copy of the F508del mutation.

Two of these studies in people with gating or residual function mutations are expected to complete their enrollment by the end of 2016, and data from these studies are expected in the first half of 2017. Earlier this year Jefferies and Credit Suisse, among other analysts, issued positive calls on this company, each calling for an impressive upside of close to doubling.

Shares of Vertex closed most recently at $78.70, with a consensus analyst price target of $130.57 and a 52-week trading range of $75.90 to $143.45. Consensus estimates predict that the company will have over $2 billion in revenues for the 2016 full year.

Incyte

Recruitment was completed in January for two Incyte Corp. (NASDAQ: INCY) Phase 2 trials of ruxolitinib in the treatment of colorectal and breast cancer. The overall survival data from both studies is expected by the end of 2016. This data will make or break these mid-stage trials. Considering the broad application to both breast cancer and colorectal cancer, and potentially more cancers in future indications, a successful trial could prove to be electric for this stock. Credit Suisse weighed in on Incyte, seeing some solid upside with a $110 price target, with emphasis on the pipeline.

Incyte shares closed most recently at $68.45. The consensus price target is $91.29, and the 52-week range is $55.00 to $133.62. Consensus estimates call for about $1 billion in revenues for the 2016 full year.
Ultragenyx Pharmaceutical

Of the multiple clinical trial results Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has expected in the near future, all are viable according to analysts. First the company expects Phase 2 interim data of KRN23 for tumor-induced osteomalacia to be reported by mid-2016, as well as Phase 3 data of rhGUS for mucopolysaccharidosis 7 (MPS7).

Phase 2 seizure study data in glucose transporter 1 deficiency syndrome (Glut1 DS) patients are expected in the second half of 2016. Jefferies sees a favorable risk/reward profile for this company concerning its KRN23 study, and that there is likely meaningful reduction in seizures from triheptanoin Phase 2 data in Glut1 DS based on optimized protocol and data from investigator sponsored trials.

Ultragenyx last closed at $56.81, with a consensus price target of $99.07 and a 52-week range of $49.00 to $137.05. Currently the company does not have any revenues.

GW Pharmaceuticals

After being on a roller-coaster of late, the rest of the year could be incredible for GW Pharmaceuticals PLC (NASDAQ: GWPH), based on its results. Previously Epidiolex achieved its primary endpoint of median reduction in seizure frequency in patients with Dravet syndrome, setting up for potentially positive top-line results before the end of 2016.

Other results for Epidiolex are expected for Lennox-Gastaut syndrome by mid-2016. Analysts are considering the reaction to previous results as a positive catalyst for this stock for the rest of 2016, with Merrill Lynch seeing this as potentially doubling. Also GW Pharma is reporting its Phase 2 data of GWP42004 in the first half of 2016 for the treatment of type 2 diabetes. The Phase 2 results from GWP42004 in the treatment of epilepsy are expected in the fourth quarter of 2016.

Shares last traded at $76.99, with a consensus price target of $148.83 and a 52-week range of $35.83 to $133.98. Analysts see nearly $22 million in revenues for 2016.

Puma Biotechnology

Puma Biotechnology Inc. (NYSE: PBYI) expects to continue to return value to shareholders in 2016 with neratinib. It is submitting a New Drug Application (NDA) to the FDA by mid-2016, which was delayed from what was expected to be a first quarter of 2016 filing. It is also reporting additional data from the Phase 2 trial of neratinib as an extended adjuvant treatment in HER2-positive early stage breast cancer using loperamide prophylaxis during the first half of 2016.

Expect even more data when the company reports its Phase 2 data from an investigator sponsored trial of neratinib in patients with HER2-negative breast cancer who have a HER2 mutation in mid-2016. By the end of 2016, we can expect Phase 2 data from PB272 Summit trial and for HER2 non-amplified breast cancer. In either the fourth quarter of 2016 of the first quarter of 2017, the company expects to present Phase 3 data of PB272 for third-line HER2-positive metastatic breast cancer.

Shares of Puma were last seen at $35.37, with a consensus price target of $112.83 and a 52-week range of $34.71 to $244.90. This company currently does not have any revenue.
Celator Pharmaceuticals

Similar to GW Pharmaceuticals, Celator Pharmaceuticals Inc. (NASDAQ: CPXX) is coming off positive results from recent trials and seeing shares soar (from $2 to over $10). Investors are expecting a sizable reaction from its clinical trial results. Earlier in March, Celator announced positive results  from its Phase 3 trial of Vyxeos piposome for injection (also known as CPX-351) in patients with high-risk (secondary) acute myeloid leukemia. Based on these results the company expects to submit a NDA for Vyxeos with the FDA later this year, and submit a Marketing Authorization Application with the European Medicines Agency in the first quarter of 2017.

Celator close most recently at $11.05. The consensus price target is $18.33, and the 52-week range is $1.12 to $12.73. This company currently does not have any revenue, and analysts do not expect sales of more than $100 million until 2019.

BioCryst Pharmaceuticals

After it announced results from OPuS-2 for the treatment of hereditary angioedema (HAE) attacks earlier in the first quarter of 2016, BioCryst Pharmaceuticals Inc. (NASDAQ: BCRX) got crushed. Unfortunately, the company said that this treatment failed to demonstrate a statistically significantly lower mean attack rate than a placebo. This was a liquid-filled soft gel formulation for the prophylactic treatment of HAE.

The company did say that oral administration of avoralstat in OPuS-2 was generally safe and well tolerated. It also noted that the adverse event profile was similar to that for the placebo, and no safety signals were observed. BioCryst plans to release its Phase 2 data for BCX7353 in the treatment of HAE by the end of 2016, which could either be incredibly positive or another let down. It seems that not all investors are writing off BioCryst yet.

BioCryst closed at $2.76, within a 52-week range of $1.63 to $16.83. The consensus price target is $6.56. Consensus estimates call for revenues of $21.5 million for the full year.

Galena Biopharma

In the fourth quarter of 2016, Galena Biopharma Inc. (NASDAQ: GALE) expects to report data from its Phase 2b randomized, combination trial with NeuVax and trastuzumab. At that time, this company expects to present interim safety data as well as patient immunology data on the A24 and A25 human leukocyte antigen status in the fourth quarter. What stands out with this trial is that trastuzumab is one of the highest revenue drugs in the world and it better known as Herceptin. Positive results in conjunction with a best-selling drug could skyrocket the stock — and of course a negative outcome could prove costly.

Shares of Galena closed at $1.06. The stock has a consensus price target of $3.90 and a 52-week range of $0.59 to $2.39. In 2015, this company had annual revenues totaling $9.73 million, but revenue is not expected to ramp up until 2019.

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