CASI Pharmaceuticals Inc. (NASDAQ:CASI)

Cellectar (NASDAQ: CLRB) Jumps 35% on Expansion of Phase 2 Trial

Cellectar Biosciences (NASDAQ: CLRB), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, today announces that the company will increase the targeted patient enrollment in the relapsed/refractory (R/R) multiple myeloma (MM) cohort of its currently enrolling Phase 2 clinical trial of CLR 131. Data from the MM cohort of the study demonstrated that the treatment exceeded pre-specified criteria for clinically meaningful benefit. As a result, the cohort will be expanded up to as many as 40 patients.

About the Phase 2 Study of CLR 131
The Phase 2 study is being conducted in approximately 10 leading cancer centers in the United States for patients with relapsed or refractory B-cell hematologic cancers.

About CLR 131
Cellectar (CLRB) developed CLR 131 as an investigational compound under development for a range of orphan designated cancers. It is currently being evaluated as a single-dose treatment in a Phase I clinical trial in patients with R/R MM as well as in a Phase II clinical trial for R/R MM and select R/R lymphomas with either a one- or two-dose treatment. Based upon preclinical and interim Phase I study data, treatment with CLR 131 provides a novel approach to treating solid and hematological tumors and may provide patients with therapeutic benefits, including overall survival, an improvement in progression-free survival, surrogate efficacy marker response rate, and overall quality of life.

I have no positions in any of the stocks mentioned, and have no plans to initiate any positions within the next 72 hours. All information, including any data, is provided without any guarantees of accuracy.

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About the author: Monica has an undergraduate degree in Accounting and an MBA she earned – with Honors. She has six years of experience in the financial markets and has been an analyst for the past two years.

Xenon Pharmaceuticals Inc. (NASDAQ:XENE) Reports Failed Phase 2 Study

Xenon Pharmaceuticals Inc. (NASDAQ:XENE)

Xenon Pharmaceuticals Inc. (NASDAQ:XENE) is based in Burnaby, British Columbia, Canada. XENE shares are down over 50% on massive volumes. The heavy price action reflects the markets view of the announcement by the company that its double-blind, multi-center, vehicle-controlled, parallel group study failed its Phase 2 clinical study. The announcement came in the form of a mid-stage assessment on the efficacy, safety, and tolerability of Xenon’s lead dermatological drug candidate XEN801.

History

XEN801 is a small molecule inhibitor of SCD1 that was developed to treat moderate to severe acne. Acne forms as a result of hair follicles being blocked – typically caused by the sebaceous glands becoming clogged with excess sebum and dead skin cells. Xenon Pharmaceuticals Inc. (NASDAQ:XENE) believed that SCD1 inhibitors, topically applied, would treat acne at the root cause by reducing sebaceous gland enlargement and reducing sebum production. Estimates put the number of Americans that suffer from the condition between 40 50 million.

XEN801 entered into a Phase 1 clinical trial in 2015. That study was completed in the same year. At that time XEN801 was found to be well-tolerated and safe to use as a result of the data gathered from 48 volunteers. The volunteers were dosed with a 14 or 21 day treatment. Different dose applications were evaluated of the 1% XEN801 drug. At this point, no serious side-effects were observed.

Xenon Pharmaceuticals Inc. (NASDAQ:XENE) then initiated a Phase 2 clinical study on 165 patients. Subjects applied a gel version of XEN801 or a placebo topically to the facial area for 12 weeks. The primary endpoint was the percent change in total lesion count from week 1 to week 12. The secondary endpoint included efficacy assessments of inflammatory and/or non-inflammatory lesion counts.

Phase 2 Mid-Stage Announcement

Results assessed by Xenon Pharmaceuticals Inc. (NASDAQ:XENE) showed no statistically significant difference between the placebo and XEN801 in achieving the primary endpoint. Additionally, results showed no statistical difference between XEN801 and the placebo in achieving its secondary endpoints. XEN801 was well tolerated and exhibited favorable safety characteristics. There were no serious drug-related side effects.

The Numbers

Shares of Xenon Pharmaceuticals Inc. (NASDAQ:XENE) fell over 50% on the news. XENE shares closed Thursday at $9.85. After the news hit the markets on Friday, XENE hit a morning low of $4.30. XENE shares have a 52-week high of $9.95, achieved yesterday, and a 52-week low of $5.65, achieved prior to today’s price action. In the past week, the trading range has been over $5.60. Volumes are exceptionally heavy. XENE shares are normally thinly traded – they average less than 87,000 per day. However today, less than an hour into trading, over 1.5 million XENE shares have traded hands.

3/24/2017
Ticker Symbol XENE
Last Price a/o 10:11 AM EST  $                      4.59
Average Volume                      86,350
Market Cap (mlns)  $                  176.71
Sales (mlns) $1.80
Shares Outstanding (mlns) 17.94
Share Float (mlns) 15.34
Shortable Yes
Optionable No
Inside Ownership 1.10%
Short Float 1.02%
Short Interest Ratio 1.8
Quarterly Return 21.60%
YTD Return 27.92%
Year Return 49.58%

I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 96 hours. All information, or data, is provided with no guarantees of accuracy.

About the author: Marc has a degree in economics and a MSc. in Finance. Over his 20-year career, Marc has worked for global investment firms in Europe and the United States as an analyst, fund manager, and consultant.

Phase 2 Data Boosts Shares of Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH)

Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH)

Shares of Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH) have hit $6.35 (up over 70%) in pre-market trading on heavy volumes. The Canadian-based company released top-line results from its Phase 2b AURA-LV (AURA) study in lupus nephritis with the firm’s product candidate voclosporin.

Lupus nephritis is a type of kidney disease. The Lupus Foundation of America estimates that 1.5 million Americans, and at least five million people worldwide, have a form of lupus. Lupus is much more common in women than in men and most often strikes during the child-bearing years. Nine out of ten people who have lupus are women and is found more frequently in people of African or Asian background. African Americans and Asian Americans are about 2 to 3 times more likely to develop lupus than Caucasians.

At 48 weeks, the trial met the complete and partial remission endpoints, demonstrating statistically significantly greater complete and partial endpoints in patients in both low dose and high dose cohorts versus the control group. No unexpected safety signals were observed and there were no additional deaths in the voclosporin treated patients; however, there were three deaths and one malignancy reported in the control arm after completion of the study treatment period. Three deaths occurred during the trail but, according to reports, all were in the placebo group of the trial.

Dr. Brad Rovin, FASN, Director of Nephrology and Vice Chairman of Research for the Department of Internal Medicine at the Ohio State University Wexner Medical Center stated in a press release “The AURA trial’s long-term results convincingly demonstrate that the addition of voclosporin to standard of care treatment is superior to standard of care alone. These data are not only statistically significant, but clinically important. Twice as many patients given 23.7mg voclosporin twice daily achieved a complete renal response compared to those treated with placebo.”

Prior to today’s market activity, Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH) had impressive performance. AUPHG shares were up 77.5% for the year, 76.7% YTD, and 15.94% for the month. Should AUPH hold these price levels, it will close the day with new 52-week closing highs. Five firms follow Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH). Four rate AUPH shares as a “Strong Buy” and one rates the shares as a “Hold”.

3/2/2017
Ticker Symbol AUPH
Last Price a/o 9:00 AM EST  $                      6.18
Average Volume                1,750,000
Market Cap (mlns)  $                  143.93
Sales (mlns) $0.20
Shares Outstanding (mlns) 38.79
Share Float (mlns) 22.43
Shortable Yes
Optionable Yes
Inside Ownership 0.00%
Short Float 11.73%
Short Interest Ratio 1.5
Quarterly Return 23.67%
YTD Return 76.67%
Year Return 77.51%

I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 96 hours. All information, or data, is provided with no guarantees of accuracy. 

About the author: Steve Clark is a 23-year Wall St professional with stints in M&A, risk management, and algorithm trading.

Why Investors Were Disappointed with BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX)

BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX)

BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) gapped down and opened 23% below Friday’s close of $5.49. However by early afternoon trading BCRX have climbed back to over $5.00 and a lower loss of over 7%. Volumes have been heavy – over 15 times their daily average.

BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported Q4 and full year earnings today. The street was expecting a Q4 net loss of $0.25 on revenues of $4.7 million. However the company surprised to the upside with revenues of $9 million and only a net loss of $0.06. For the year, the company reported that its loss widened to $55.1 million, or 75 cents per share. Revenue was reported as $26.4 million.

Investors were less positive over the reports that came from BioCryst’s interim Phase 2 study results. In the phase II study of 28 patients, treatment with BioCryst’s BCX7353 reduced the rate of hereditary angioedema attacks by 52% compared to a placebo. The disease stems from a protein imbalance in the patient’s blood which causes swelling under the skin. Hereditary angioedema is rare and afflicts approximately one in 30,000 people. Current treatments are delivered by injection. BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) is developing an oral drug to hopefully replace the injections. Analysts were expecting a rate reduction in attacks above 70% – so while the drug proved successful against a placebo, the results were short of investor expectations. A conmpeting, iunjectionable drug being developed by Shire (SHPG) reportedly is showing attack reductions around 90%.

BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) core development programs include BCX7353 and additional 2nd generation oral inhibitors of plasma kallikrein for hereditary angioedema; and BCX4430 (GALIDESIVIR), a broad spectrum antiviral for hemorrhagic fevers.

Prior to today, BioCrust shares had been performing well – over 175% for the year to the upside. BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) posted their best sales year in 2015 ($48.3 million). It had also narrowed its EPS loss of $0.68 in 2014 to a smaller loss of $0.59 in 2015. Ten firms follow BioCryst and six rate BCRX as a “Strong Buy”, three rate the shares as a “Hold”, and one rates the shares as an “Underperform”.

2/27/2017
Ticker Symbol BCRX
Last Price a/o 2:39 PM EST  $                      5.10
Average Volume                    982,900
Market Cap (mlns)  $                  413.01
Sales (mlns) $22.00
Shares Outstanding (mlns) 75.23
Share Float (mlns) 72.75
Shortable Yes
Optionable Yes
Inside Ownership 1.60%
Short Float 10.84%
Short Interest Ratio 80.2
Quarterly Return -6.95%
YTD Return -13.27%
Year Return 177.27%

I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 96 hours. All information, or data, is provided with no guarantees of accuracy.

About the author: Marc has a degree in economics and a MSc. in Finance. Over his 20 year career, Marc has worked for global investment firms in Europe and the United States as an analyst, fund manager, and consultant.

Ocera Therapeutics (Nasdaq: OCRX) Phase 2b Results Disappoint

Ocera Therapeutics, Inc. – Nasdaq: OCRX

Shares of Ocera Therapeutics are down over 70% after it announced top-line results from its phase 2b study that suggested no statistical improvements from its product candidate versus the placebo. Ocera shares are traded on the Nasdaq under ticker OCRX. Ocera still intends to pursue development of the drug.

“Initial analysis shows that a higher percentage of patients respond as the dose of OCR-002 increases, notwithstanding that the clinical endpoints did not reach statistical significance,” said Stan Bukofzer, M.D., Chief Medical Officer of Ocera. “This dose-response direction was also observed for the highly statistically significant ammonia reduction. Moreover, the safety profile shows no safety signal for OCR-002 compared to placebo. At the highest drug doses, there were favorable differences compared to placebo in the frequency of deaths and serious life threatening safety events. We expect further exploratory analyses will provide additional clarity for our goal of advancing intravenous OCR-002 into Phase 3 development.”

Hepatic encephalopathy is a debilitating and progressive complication of liver cirrhosis or liver failure, marked by increasing ammonia levels, mental changes including confusion, impaired motor skills, disorientation, and in its more severe form, stupor, coma and even death. It is estimated that HE-related hospitalization costs exceed $7 billion1 annually in the U.S. alone.

OCRX shares have experienced narrowing EPS losses over the last five years and in 2015 a loss of $1.33 was posted. OCRX sales have been weak and in 2015 posted $100k. Five firms follow Ocera and all rate OCRX shares as a “Strong Buy” with a consensus price target of $10.

I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 96 hours. All information, or data, is provided with no guarantees of accuracy.

1/30/2017
Ticker Symbol OCRX
Last Price a/o 12:06 PM EST  $                      0.60
Average Volume 312,000
Market Cap (mlns)  $                    47.40
Sales (mlns) $0.10
Shares Outstanding (mlns) 23.12
Share Float (mlns) 22.68
Shortable Yes
Optionable Yes
Inside Ownership 0.30%
Short Float 1.08%
Short Interest Ratio 0.79
Quarterly Return -15.46%
YTD Return -2.38%
Year Return -24.91%

Marinus Pharmaceuticals (Nasdaq: MRNS) Up on Massive Volume

Marinus Pharmaceuticals, Inc. – Nasdaq: MRNS

Massive volumes are being traded in nano-cap Marinus Pharmaceuticals. Traded on the Nasdaq under ticker MRNS, volumes are over nine times their daily average. The increased volumes appear to be in response to news of positive preliminary data from the initial CDKL5 patients enrolled in its ongoing Phase 2 open-label study evaluating its CNS-selective GABAA modulator, ganaxolone, as a treatment for orphan, genetic disorders. Marinus is currently evaluating ganaxolone in orphan pediatric indications for the treatment of genetic seizure and behavior disorders, and preparing to initiate Phase 2 studies in status epilepticus, an orphan indication, and postpartum depression.

CDKL5 is a serious and rare genetic disorder that is caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. It predominantly affects girls and is characterized by early-onset, difficult-to-control seizures and severe neuro‑developmental impairment.

Two firms follow Marinus Pharmaceuticals. One rates MRNS as a “Strong Buy” and the other rates the shares as a “Hold”. The consensus price target for MRNS is $2.50. Shareholders of MRNS have never experienced a positive EPS and last year MRNS had an EPS loss of $1.67 on zero reported sales.

I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 96 hours. All information, or data, is provided with no guarantees of accuracy.

Merrimack Pharmaceuticals Inc. (Nasdaq: MACK) Halts Phase 2 Study – Shares Plummet

Merrimack Pharmaceuticals Inc.; Nasdaq: MACK

Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) shares have dropped over 18% in pre-market trading following today’s announcement that, following a recent independent Data and Safety Monitoring Board (DSMB) recommendation and subsequent futility analysis, it has decided to stop the Phase 2 HERMIONE study of MM-302 (HER2 antibody-targeted liposomal doxorubicin) in HER2-positive metastatic breast cancer patients who had previously been treated with trastuzumab (Herceptin®), pertuzumab (Perjeta®) and ado-trastuzumab emtansine (T-DM1, Kadcyla®).

The decision to stop the trial was made following the DSMB’s opinion that continuing would be unlikely to demonstrate benefit over the comparator treatments. Subsequent to this recommendation, a futility assessment was performed that confirmed the DSMB’s opinion. Both the treatment and control arms were found to have shorter than expected median progression free survival.

From Merrimack Phamaceutical’s press release:

“Late line HER2-positive breast cancer is very difficult to treat, especially in this new and previously unstudied group of patients who appear to experience rapid cancer progression following treatment with trastuzumab, pertuzumab and ado-trastuzumab emtansine,” said. “While we are disappointed with this outcome, we would like to thank the study Steering Committee, the investigators and, most importantly, the patients who participated in the HERMIONE trial. We will report our learnings from this study at a later date.”

Three analysts cover Merrimack Pharmaceuticals Inc. All have given MACK a rating of “Hold” but it is important to note that none of the ratings have been reviewed since today’s announcement. MACK shares have never had a positive EPS – in 2015 Mack had a loss of $1.33 EPS. Sales have performed better. In 2012, sales were reported at $34.2 million and in 2015 sales were reported at $89.3 million.

I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 96 hours. All information, or data, is provided with no guarantees of accuracy.

Ticker Symbol MACK
Last Price a/o 8:10 AM EST  $                    4.60
Average Volume 3.4 million
Market Cap $678.7 million
Sales $104.5 million
Shares Outstanding 126.4 million
Share Float 119.32 million
Shortable Yes
Optionable Yes
Inside Ownership 0.10%
Short Float 32.17%
Short Interest Ratio 11.29
Quarterly Return 5.71%
YTD Return -32.03%
Year Return -30.71%