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Repros Therapeutics (RPRX) surged in after-hours trading on Wednesday after the company announced positive results from a Phase 2 study evaluating Proellex (telapristone acetate) for the treatment of premenopausal women with pelvic pain associated with endometriosis. The primary endpoint was the change from baseline in pelvic pain as measured by a scale called Biberoglu and Behrman Symptom Score (BBSS) over the treatment period. 70% of the patients treated with Proellex became amenorrheic, which was associated with a substantial reduction in pain and analgesic use. Responses were similar between the two doses of Proellex so the reported results are pooled. The median percent change from baseline in BBSS score related to menstrual pain was a reduction of 85.4% compared to -37.5% for placebo (p=0.0008). Reductions in non-menstrual pain were also observed but they were similar between the treatment groups.

BioMarin Submits Long-Term Efficacy Data for Cerliponase Alfa- BioMarin Pharmaceuticals (BMRN) announced that it has submitted ong-term efficacy data supporting the use of cerliponase alfa in children with CLN2 disease, a form of Batten disease, a rare inherited disorder in which the body's cells lose their ability to dispose of wastes. The majority of affected children are no longer able to walk and talk by age six. The submission was made as per a request from the FDA. he agency designated the submission as a major amendment so its action date (PDUFA) has been extended three months to April 27, 2017. An advisory committee meeting will also be held, although the specific date has not been set.

Retrophin Announces Positive Results from DUET Study- Retrophin (RTRX) shares closed more than 27% higher on Wednesday after the company announced positive results in a Phase 2 clinical trial, DUET, assessing its sparsentan (RE-021) for the treatment of focal segmental glomerulosclerosis, a rare kidney disorder that leads to end-stage renal disease. The study met its primary endpoint of a statistically valid reduction (more than two-fold) in proteinuria (protein in urine) compared to irbesartan [Sanofi's (NYSE:SNY) Avapro] after an eight-week treatment period. Specifically, patients treated with 200, 400 and 800 mg/day of sparsentan (n=64) showed a mean reduction of proteinuria from baseline of 44.8% after eight weeks of treatment compared to 18% for those (n=32) receiving 300 mg/day of irbesartan (p=0.006). The 400 mg and 800 mg sparsentan cohorts also showed superiority to 300 mg of irbesartan, 47.4% vs. 19.0% (p=0.011).

Agios Rallies as Celgene Files NDA for AG-221- Agios Pharmaceuticals (AGIO) shares rose more than 25% on Wednesday after the company announced that its collaboration partner Celgene (CELG) plans to file a New Drug Application (NDA) with the FDA by year end seeking approval of Agios' AG-221 for the treatment of patients with advanced blood cancers who have a mutation in a protein called isocitrate dehydrogenase-2 (IDH2). Celgene will be discussing its plans for the NDA today at the Citi 11th Annual Biotech Conference in Boston. The data supporting the NDA was generated in a Phase 1/2 study in patients with IDH2 mutation-positive acute myeloid leukemia (AML). AG-221 (enasidenib) inhibits IDH2.

Evoke Pharma Provides Update on EVK-001- Evoke Pharma (EVOK) shares surged on Wednesday after the company announced results from its pre-NDA meeting with the FDA regarding lead product candidate Gimoti (EVK-001)(metoclopramide nasal spray) for the relief of symptoms related to acute/recurrent diabetic gastroparesis in adult women. Based on the agency's response to the information package and what was discussed during the meeting, the company believes it has the information it needs to complete the sections in a manner acceptable to the FDA.

CytRx Completes Enrollment in Mid-Stage Study of Lead Product Candidate- CytRx (CYTR) announced that it has completed enrollment of 132 patients in its Phase 2b clinical trial assessing lead product candidate aldoxorubicin in treatment-experienced patients with small cell lung cancer (SCLC). The primary endpoint is progression-free survival (PFS) at Month 24. Overall survival is a secondary endpoint. According to ClinicalTrials.gov, the estimated study completion date is July 2017, but this needs revising since the completion should be September 2018 based on two-year PFS data.

Raptor Pharmaceuticals Announces Data for Inhaled Levofloxacin- Raptor Pharmaceuticals (RPTP) announced that a meta-analysis of all randomized clinical trials of inhaled antibiotics in cystic fibrosis (CF) patients showed QUINSAIR (aerosolized form of levofloxacin) had similar efficacy to three other inhalable antibiotics in treating lung infections involving the bacterium Pseudomonas aeruginosa, the primary cause of progressive lung function decline in CF sufferers. The data were presented today at the European Respiratory Society International Congress in London. QUINSAR, acquired from Tripex Pharmaceuticals in August 2015, was approved in Europe in March 2015 and in Canada three months later. It is not yet available in the U.S.

Capricor Completes Enrollment in Early Stage Study of Lead Product Candidate- Capricor Therapeutics (CAPR) announced that it has completed enrollment target of 25 patients n its Phase 1/2 study, HOPE-Duchenne, assessing lead product candidate CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). The primary endpoint of the trial is the safety and tolerability of CAP-1002 72 hours post infusion. Secondary endpoints include a cardiac structural composite measured by MRI, a functional composite of mobility measurements and a quality-of-life composite. According to ClinicalTrials.gov, the estimated final data collection date for the primary endpoint is August.

Merck & Co. (MRK) announced that the FDA has accepted for review its supplemental Biologics License Application (sBLA) seeking approval of the use of KEYTRUDA (pembrolizumab) for the first-line treatment of patients with advanced non-small cell lung cancer (NSCLC) whose tumors express PD-L1, a Breakthrough Therapy designation. The agency's action date (PDUFA) is December 24.

Matinas BioPharma (MTNB) announced that the FDA has granted a Qualified Infectious Disease Product (QIDP) and Fast Track status to its Phase 2-stage MAT2203 for prophylactic treatment of invasive fungal infections due to immunosuppressive therapy.

La Jolla Pharmaceuticals (LJPC) announced that it has reached an agreement with the European Medicines Agency on the design of a pivotal study assessing LJPC-401, a novel formulation of the iron regulator hepcidin, for the treatment of beta thalassemia (BT) patients suffering from iron overload. BT is an inherited disorder that reduces the production of hemoglobin. The primary endpoint of the study, expected to commence in mid-2017, will be a clinically relevant measurement directly related to iron overload.

No patents to report.

Immune Pharmaceuticals (IMNP) announced that it has signed a stock purchase agreement with an existing shareholder for the sale of 4M shares of common stock at $0.50 a share.

Aralez Pharma (ARLZ) announced that it has acquired the rights to Merck’s (MRK) antiplatelet drug ZONTIVITY (vorapaxar) in the U.S. and Canada. According to the terms of the agreement, Merck will receive an upfront cash payment of $25M plus sales-based milestones and royalties. Merck will continue to distribute the product for up to 12 months on Aralez's behalf while the product rights, packaging, labeling and other items are transferred to Aralez's Ireland-based subsidiary Aralez Pharmaceuticals Trading DAC.

BioLineRx (BLRX) announced that it will collaborate with Roche’s (RHHBY) Genentech unit in several Phase 1b clinical trials to evaluate the combination of BioLineRx's lead product candidate BL-8040 and TECENTRIQ (atezolizumab) in multiple cancers. According to the terms of the agreement, Genentech will sponsor and conduct several Phase 1b studies in multiple solid cancers. BioLineRx will sponsor and conduct one Phase 1b study in acute myeloid leukemia (AML). All studies will be open-label and single-arm. After the trials are completed, both parties will have the option to expand the collaboration to include a pivotal registration study. Additional details are not disclosed.

Sage Therapeutics (SAGE) announced that it has commenced a $150 million public offering of common stock. Prices, volume and terms of the deal have not yet been disclosed.

Xenon Pharmaceuticals (XENE) announced that it has commenced a public offering of common stock o be sold by the company and certain shareholders. Price, volume and terms of the offering have not yet been disclosed.

No IPOs to report.

No earnings to report.

Company (Ticker) Brokerage Action Recommendation Price Target My Price Target
Abeona Therapeutics (ABEO) Cantor Fitzgerald Initiation Buy N/A N/A
Aerie Pharmaceuticals (AERI) Brean Capital Reiterate Buy

$48

N/A
Amphastar Pharmaceuticals (AMPH) Piper Jaffray Cos. Price Target Raised Overweight From $22 to $23 N/A
BioLineRx (BLRX) Maxim Group Reiterate Hold

$1

N/A
BioMarin Pharmaceuticals (BMRN) JPMorgan Chase & Co. Reiterate Buy N/A N/A
Bristol-Myers Squibb (BMY) Leerink Swann Reiterate Buy

$75

N/A
Celgene (CELG) Citigroup Reiterate Buy

$155

$140

Coherus Biosciences (CHRS) Maxim Group Initiation Buy

$43

N/A
Caladrius Biosciences (CLBS) HC Wainwright Reiterate Buy N/A N/A
Dynavax Technologies (DVAX) RBC Capital Markets Reiterate Sector Perform

$16

$50

Histogenics (HSGX) William Blair Downgrade From Outperform to Market Perform N/A N/A
Jazz Pharmaceuticals (JAZZ) Leerink Swann Reiterate Outperform

$202

N/A
Johnson & Johnson (JNJ) BTIG Research Reiterate Neutral N/A N/A
Ligand Pharmaceuticals (LGND) Deutsche Bank Upgrade/Price Target Cut  From Sell to Hold From $110 to $105 N/A
Ligand Pharmaceuticals (LGND) Roth Capital Reiterate Buy

$149

N/A
MacroGenics (MGNX) Cowen and Company Initiation Outperform N/A N/A
Mallinckrodt (MNK) Piper Jaffray Cos. Price Target Cut  Overweight From $112 to $105 N/A
Momenta Pharmaceuticals (MNTA) Maxim Group Downgrade From Hold to Sell

$6

N/A
Pacira Pharmaceuticals (PCRX) Cowen and Company Initiation Market Perform N/A N/A
Rosetta Genomics (ROSG) S&P Equity Research Price Target Cut  N/A From $0.94 to $0.82 N/A
Retrophin (RTRX) Leerink Swann Price Target Raised N/A

$32

N/A
Sucampo Pharmaceuticals (SCMP) Northland Securities Initiation Outperform

$15

N/A
Seattle Genetics (SGEN) Morgan Stanley Initiation Overweight

$60

N/A
Tonix Pharmaceuticals Holding (TNXP) Cantor Fitzgerald Downgrade From Buy to Hold N/A N/A
Tonix Pharmaceuticals Holding (TNXP) Oppenheimer Holdings Downgrade From Outperform to Market Perform N/A N/A
Tonix Pharmaceuticals Holding (TNXP) Roth Capital Downgrade From Buy to Neutral N/A N/A
Valeant Pharmaceuticals International  (VRX) Morgan Stanley Reiterate Overweight

$42

N/A
Valeant Pharmaceuticals International  (VRX) RBC Capital Markets Reiterate Sector Perform

$36

N/A
Zynerba Pharmaceuticals (ZYNE) Canaccord Genuity Reiterate Buy

$35

N/A

Adamas Pharmaceuticals (ADMS)- William W. Ericson, Director, bought 51,919 shares in two separate transactions. Ericson bought 43,719 shares at $14.75; and 7,200 shares at $15. Ericson now owns 3,823,605 shares of ADMS. MDV IX LP, a 10% owner, bought 51,919 shares in two separate transactions. MDV IX LP bought 43,719 shares at $14.75; and 7,200 shares at $15. MDV IX LP now owns 3,823,605 shares of ADMS.

Irownwood Pharmaceuticals (IRWD)- Lawrence S. Olanoff, Director, sold 3,200 shares at $13.72. The total value of the transaction was $43,904. Olanoff still owns 29,305 shares of IRWD.

Alkeremes (ALKS)- Elliott Ehrich, EVP, R&D & CMO, Alkermes Inc., sold 10,000 shares at $46.02. The total value of the transaction was $460,235. Ehrich still owns 45,311 shares of ALKS. Iain Michael Brown, SVP Finance & CAO, Alks Inc., sold 29,976 shares at $45.92. The total value of the transaction was approximately $1.38 million. Brown still owns 22,227 shares of ALKS. David Joseph Gaffin, SVP, CLO, Alkermes Inc., sold 2,382 shares at $45.92. The total value of the transaction was $109,378. Gaffin still owns 20,434 shares of ALKS. Paul J. Mitchell, Director, sold 2,000 shares at $45.92. The total value of the transaction was $91,834. Mitchell still owns 8,000 shares of ALKS.

No management changes and additions to report.

NYSE- Intrexon (XON) shares were among the major movers on the NYSE. The stock closed 4.46% higher.

NASDAQ- Retrophin (RTRX) shares were among the major gainers on the NASDAQ. The stock closed 27.51% higher. Agios Pharmaceuticals (AGIO) ended the day 25.88% higher. GW Pharmaceuticals (GWPH) ended the day 23.55% higher. CoLucid Pharmaceuticals (CLCD) shares were among the major losers on the NASDAQ. The stock closed 14.27% lower. Catabasis Pharmaceuticals (CATB) ended the day 11.70% lower. Proteostasis Therapeutics (PTI) ended the day 9.78% lower.

NYSEMKT- Asterias Biotherapeutics (AST) shares were among the major movers on the NYSEMKT. The stock closed 6.10% higher.

OTC- Amarantus Bioscience Holdings (AMBS) shares were among the major movers on the OTC market. The stock closed 3.32% higher.

Company (Ticker) Short Interest as a % of Float % Change Days to Cover
Alcobra (ADHD)

11.1%

3.2%

29

Alder BioPharmaceuticals (ALDR)

20.2%

-2.5%

18

Aldeyra Therapeutics (ALDX)

0.9%

6.1%

1

Alexion Pharmaceuticals (ALXN)

2.7%

5.2%

4

Alimera Sciences (ALIM)

6.4%

13.7%

2

Alliqua BioMedical (ALQA)

1.6%

4.8%

5

Alnylam Pharmaceuticals (ALNY)

10.5%

-9.1%

12

AMAG Pharmaceuticals (AMAG)

21.9%

3.7%

5

Amgen (AMGN)

1.1%

6.5%

3

Amicus Therapeutics (FOLD)

16.2%

3.6%

10