What Did Regulus Therapeutics Inc (NASDAQ:RGLS) Achieve in Q4?

Regulus Therapeutics Inc (NASDAQ:RGLS)

Regulus Therapeutics Inc (NASDAQ:RGLS) reported Q4 2016 earnings after market close on March 2. It also offered an update on its business, especially pipeline programs.

Regulus’ Q4 earnings at a glance

The company extended its mixed earnings track record in Q4. EPS loss of $0.38 widened from $0.14 a year earlier and was also worse than EPS loss of $0.37 that analysts were expecting. Looking back, Regulus’ has topped earnings expectations twice and missed twice over the last four quarters.

Revenue of less than $0.1 million in the latest quarter was in sharp contrast to revenue of $10.9 million in the same period a year earlier. Furthermore, the latest revenue figure came short of consensus estimate of $10.9 million.

Higher R&D expenses drag earnings

Regulus Therapeutics Inc (NASDAQ:RGLS) booked higher expenses related to research and development than it did a year earlier. R&D costs of $15 million were more than $12.8 million a year ago. Spending on clinical and pre-clinical programs drove Regulus’ R&D spending up and exerted downward pressure on earnings.

However, the company still management to maintain prudent financial controls as G&A expenses of $4.8 million in the latest quarter was down from $5.4 million a year earlier. Regulus said the decrease in S&G expenses was supported by lower staff expenses and stock-based compensations.

Cash down significantly

Regulus concluded 2016 with cash and equivalents totaling $76.1 million, significantly down from $115.3 million at the end of 2015.

Business condition update

Regulus Therapeutics Inc (NASDAQ:RGLS) shared several updates on its business condition. One of them was the unveiling of two new clinical candidates namely RGLS5040 (anti-miR-17) and RGLS4326 (anti-miR-27). It said RGLS5040 is being developed as a treatment of cholestatic diseases, while RGLS4326 is aimed at the kidney treatment market. The company intends to seek Investigational New Drug approvals for the candidates in the back half of 2017.

Regulus also updated on the Phase 1 MAD and Phase 2 HERA studies of its candidate RG-012, which is aimed at patients with various conditions of Alport syndrome. It initiated the MAD study in Q4 2016 and expects to release topline safety data from the study in Q2 2017. Regulus said HERA study of RG-012 will resume in the back half of 2017 after concluding MAD trial, with interim data from HERA study expected in mid-2018.

Stock movements

The market reacted positively to Regulus Therapeutics Inc (NASDAQ:RGLS)’s earnings and business updates, closed flat at $1.10 on Friday. However, the stock has declined more than 50% so far in 2017 and remains down more than 80% over the past one year.

3/3/2017
Ticker Symbol RGLS
Last Price a/o, 4:02PM EST $1.10
Average Volume 1.02Million
Market Cap (mlns) $58.22
Sales (mlns) $12.00
Shares Outstanding (mlns) 50.55
Share Float (mlns) 36.93
Shortable Yes
Optionable Yes
Inside Ownership 42.92%
Short Float 10.22%
Short Interest Ratio 4.43
Quarterly Return -56.86%
YTD Return -51.11%
Year Return -84.79%

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

Can Synthetic Biologics Inc (NYSEMKT:SYN) Keep Hope Alive?

Synthetic Biologics Inc (NYSEMKT:SYN)

Synthetic Biologics Inc (NYSEMKT:SYN)’s annual financial results released on Thursday showed the company swung to a loss in 2016. But the management still sought to keep hope alive, citing the potential in the company’s pipeline candidates to drive future growth and profitability.

Anxious investors sold the stock ahead of the earnings release, causing the stock to fall nearly 2% in regular session. But the stock bounced back in extended trading Thursday after Synthetic released the earnings and the management seemed to maintain an upbeat tone for the future.

Synthetic Biologics Inc (NYSEMKT:SYN) plunged into EPS loss of $0.29 in 2016, worse than EPS loss of $0.08 that analysts were expecting for the year.

What caused the losses?

Synthetic’s losses in 2016 stemmed from a spike in expenses, especially the G&A tab. G&A costs rose to $10.1 million from $8.1 million in the previous year. The company blamed higher bank and legal fees for the increase in G&A costs in the latest year. Synthetic’s bottom line also suffered the impact of costs associated with the relocation of administrative and finance offices to its headquarters in Maryland. Higher staff costs and increase in stock-based compensation also weighed on the bottom line in 2016.

However, the company still managed to reign on costs in its R&D department. R&D costs fell to $29.1 million from $32.9 million in 2015 lower costs associated with research activities, especially clinical development programs, helped reduce R&D costs.

Synthetic Biologics Inc (NYSEMKT:SYN) booked Other Income of $11.4 million in 2016, a dramatic improvement from Other Expenses of $3.8 million in 2015. But it ended the year with a small cash of just $19.1 million, down $1.8 million from the previous year.

Pipeline update

Synthetic Biologics Inc (NYSEMKT:SYN) provided an update on the development progress of its candidate called SYN-010, which is being developed as a treatment of irritable bowel syndrome with constipation (IBS-C).

It said Phase 2 proof-of-concept blind study of the compound yielded positive results compared to placebo, just as it expected.  The trial was carried out on 412 patients. The outcome showed that SYN-010 met, based on CDI rates, the primary endpoint that was demonstrating statistically significant risk reduction of 71.4% compared to the placebo.

Synthetic plans to request end of Phase 2 meeting with the FDA in the back half of 2017. It also expects to initiate Phase 3 study of the compound in the first half of 2018.

Stock performance

Shares of Synthetic Biologics Inc (NYSEMKT:SYN) fell 2.61% to $0.785 and closed at $0.765. The stock is up nearly 3% year-to-date and down more than 54% over the last 12 months.

03/03/2017
Ticker Symbol SYN
Last Price a/o, 4:02PM EST  $0.765
Average Volume 489,032
Market Cap (mlns) $89.90
Sales (mlns)
Shares Outstanding (mlns) 115.18
Share Float (mlns) 115.18
Shortable Yes
Optionable No
Inside Ownership 0.30%
Short Float 7.23%
Short Interest Ratio 10.92
Quarterly Return -6.78%
YTD Return 2.99%
Year Return -59.72%

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

NewLink Genetics Corporation (NASDAQ:NLNK) Has a Big Day

NewLink Genetics Corporation (NASDAQ:NLNK)

NewLink Genetics Corporation (NASDAQ:NLNK) gained 17.5% today on over five times the average daily volume. NewLink Genetics announced that it will be presenting at the 2017 American Association of Cancer Research annual meeting. Experts claim that when a firm agrees to participate in such an event, there is a high likelihood that they will be presenting positive news on one of their drug or treatment candidates.

NewLink Genetics (NASDAQ:NLNK) is a biopharmaceutical company at the forefront of discovering, developing and commercializing novel immuno-oncology product candidates to improve the lives of cancer patients. NewLink Genetics’ product candidates are designed to harness multiple components of the immune system to combat cancer.

Tuesday, February 28, 2017, NewLink Genetics Corp. (NASDAQ:NLNK) reported Q4 and full year earnings. NewLinks lost $13.5 million in its fourth quarter. The Ames, IA-based company said it had a loss of $0.46/share. The results exceeded Wall Street expectations. The average of analysts estimates of four analysts was reported to be a loss of $0.66/share. NewLink Genetics posted revenue of $12.7 million in the period – beating analyst estimates of $4.3 million. NewLink posted a yearly loss of $85.2 million, or $2.94 per share. Revenue was reported as $35.8 million.

NewLink Genetics Corp. (NASDAQ:NLNK) shares have risen 38.2% this week and 92% YTD. NLNK shares started the year around $10 and have risen in value each month for the past three months but are still over 4% from their 52-week highs. Of the six firms that follow NewLink Genetics Corp. (NASDAQ:NLNK), four rate the shares as a “Strong Buy” and two rate NLNK shares as a “Hold”.

3/2/2017
Ticker Symbol NLNK
Last Price a/o 5:08 PM EST  $                    19.73
Average Volume                    281,750
Market Cap (mlns)  $                  578.88
Sales (mlns) $30.70
Shares Outstanding (mlns) 29.34
Share Float (mlns) 21.62
Shortable Yes
Optionable Yes
Inside Ownership 1.50%
Short Float 10.61%
Short Interest Ratio 8.14
Quarterly Return 72.16%
YTD Return 91.93%
Year Return -0.85%

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 Karyopharm Therapeutics Inc. (NASDAQ:KPTI) Popped

Karyopharm Therapeutics Inc. (NASDAQ:KPTI)

Karyopharm Therapeutics Inc. (NASDAQ:KPTI) has announced it will be presenting at the 2017 American Association for Cancer Research annual meeting. KPTI shares were up over 32%, to a high of $14.63, on the news. Volumes are heavy – over ten times the pro-rata daily average. Traders are expecting that the presentation will reveal positive news concerning Karyopharm’s drug candidates selinexor (KPT-330), KPT-8602, and KPT-9274.

Selinexor is being evaluated in multiple later stage clinical trials in patients with relapsed and/or refractory hematological and solid tumor malignancies. To date, selinexor has been administered to more than 1,900 patients across company-sponsored and investigator-sponsored clinical trials. Karyopharm Therapeutics Inc. (NASDAQ:KPTI) will initiate a pivotal randomized Phase 3 study, known as the BOSTON (Bortezomib, Selinexor and dexamethasone) study in early 2017 which will evaluate selinexor in combination with bortezomib (Velcade®) and low-dose dexamethasone (SVd) compared to bortezomib and low-dose dexamethasone (Vd) in patients with multiple myeloma who have had one to three prior lines of therapy.

In preclinical models, KPT-8602 has a broad therapeutic window with minimal penetration of the blood brain barrier and, therefore, has the potential to serve as a second generation SINE™ compound for cancer indications. The first-in-humans clinical trial for KPT-8602 is a Phase 1/2 study in patients with relapsed/refractory multiple myeloma. The first patient in the Phase 1 dose escalation portion of the study was dosed in January 2016.

KPT-9274 is a first-in-class orally bioavailable small molecule that is a non-competitive dual modulator of PAK4 and NAMPT. Co-inhibition of these targets leads to synergistic anti-tumor effects through energy depletion, inhibition of DNA repair, cell cycle arrest, inhibition of proliferation, and ultimately apoptosis. The first patient was dosed with KPT-9274 in June 2016.

Of the eight firms that follow Karyopharm Therapeutics Inc. (NASDAQ:KPTI), six rate KPTI shares as a “Strong Buy”, one rates the shares as a “Buy” and one rates the shares as an “Underperform”. The MA-based biotech firm has reported negative EPS every year since 2011 and in 2015 reported an EPS loss of $3.32. Sales increased 50% in 2015 to $300,000 but were down from 2012 when the company posted $600,000 in sales.

3/2/2017
Ticker Symbol KPTI
Last Price a/o 11:02 AM EST  $                    13.38
Average Volume                    184,750
Market Cap (mlns)  $                  462.60
Sales (mlns) $0.10
Shares Outstanding (mlns) 41.94
Share Float (mlns) 29.67
Shortable Yes
Optionable Yes
Inside Ownership 0.10%
Short Float 4.97%
Short Interest Ratio 7.98
Quarterly Return 18.73%
YTD Return 17.34%
Year Return 73.16%

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.

Why did Infinity Pharmaceuticals, Inc. (NASDAQ:INFI) Gain +20%?

Infinity Pharmaceuticals, Inc. (NASDAQ:INFI)

Infinity Pharmaceuticals, Inc. (NASDAQ:INFI) shares have reached $3.84 (up 28%) on the company’s announcement that new Phase 1 data for IPI-549, an orally administered immuno-oncology development candidate, will be presented in a clinical trial poster session at the 2017 American Association for Cancer Research (AACR) Annual Meeting. Experts generally agree that it would be unusual for Infinity to agree to discuss trail data if they were anything but positive.

The Phase 1 study is ongoing and exploring the safety and activity of IPI-549 both as a monotherapy and in combination with Opdivo® (nivolumab), a PD-1 immune checkpoint inhibitor which is marketed by NJ-based Bristol-Myers Squibb (NYSE:BMY), in patients with advanced solid tumors.

More than 17% of all new cancer cases involve solid tumors in the lungs, head, or neck areas. The expansion cohorts evaluating IPI-549 plus Opdivo will include patients with non-small cell lung cancer, melanoma and squamous cell carcinoma of the head and neck. Cancer patients enrolled in the combination expansion cohorts represent a difficult-to-treat population, as they must have demonstrated initial resistance or subsequently develop resistance to a PD-1 or PD-L1 therapy immediately prior to enrolling in the study.

In 2016 INFI was removed from being a constituent stock in the NASDAQ Biotechnology index. But Infinity Pharmaceuticals, Inc. (NASDAQ:INFI) also had a rough 2016 with analysts. Of the ten reviews, seven resulted in downgrades and three reiterated their existing ratings. There is no doubt that sales from Infinity Pharmaceuticals, Inc. (NASDAQ:INFI) have been woefully inconsistent. In 2011 the company posted sales of $92.8 million followed by annual sales of $47.1 million, $0.00, $165 million, and $109.1 million in 2015. EPS has been more consistent but always negative. EPS losses in 2011 were -$1.50 followed by annual losses of -$1.70, -$2.65, -$0.36, and -$2.62 in 2015.

3/2/2017
Ticker Symbol INFI
Last Price a/o 10:04 AM EST  $                      3.60
Average Volume                1,110,000
Market Cap (mlns)  $                  147.27
Sales (mlns) $27.80
Shares Outstanding (mlns) 49.09
Share Float (mlns) 48.64
Shortable Yes
Optionable Yes
Inside Ownership 1.10%
Short Float 5.65%
Short Interest Ratio 2.48
Quarterly Return 165.49%
YTD Return 122.22%
Year Return -49.66%

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.

Here’s Why Stemline Therapeutics Inc (NASDAQ:STML)’s Shares Soared Despite Legal Threats

Stemline Therapeutics Inc (NASDAQ:STML)

Stemline Therapeutics Inc (NASDAQ:STML)’s shares rallied more than 14% on Wednesday despite legal threats facing the company following the death of a patient who received a treatment of the company’s lead drug candidate SL-401. The patient died of capillary leak syndrome (CLS), a well-known side effect of SL-401.

The reason shares of Stemline soared was that HC Wainwright analysts, in their updated note on the stock, downplayed the threat of the patient death to the future of SL-401. According to the analysts, the FDA is unlikely to demand that Stemline put the ongoing Phase 2 clinical trial of the candidate on hold because of the patient’s death. They reason that the cause of the death isn’t something secretive but a known side effect of the candidate. Furthermore, the analysts said that an antineoplastic agent called Ontak, which is in the same class as SL-401 and has received regulatory approval, also carries the same CLS side effect.

Stemline Therapeutics Inc (NASDAQ:STML) is developing SL-401 as a treatment for a rare blood cancer.

As such, the analysts have reiterated their BUY rating on Stemline and maintained a price target of $34 on the stock.

Legal threats

The report of the patient death has been widely covered by the media and has also led to several law firms filing class-action claims against Stemline Therapeutics Inc (NASDAQ:STML). Shareholder rights attorneys at Bronstein, Gewirtz & Grossman, LLC, for instance, have accused the company of withholding crucial information from investors, leading them to incur losses from their investment in the company’s stock.

They say that Stemline knew about the death of the patient on their clinical trial, yet the management sat on the information until the company announced a secondary equity offering of 4.5 million shares priced at $10 apiece. The attorneys say investors went on to purchase Stemline’s shares in the secondary offering oblivious lurking danger in the patient’s death report. When the report went public, shares of Stemline crashed, causing investors massive losses.

Bronstein, Gewirtz & Grossman’s class action suit is on behalf of investors who owned or acquired shares of Stemline between January 20, 2017 and February 1, 2017.

Analysts’ action on Stemline

Besides HC Wainwright, several other analysts have also recently commented on the stock of Stemline. Cowen analysts maintained their BUY rating on the stock in an updated note to investors on February 2. Jefferies analysts set a price target of $16 and gave the stock a BUY rating in their February 3 note.

Stock movements

Shares of Stemline Therapeutics Inc (NASDAQ:STML) jumped 14.3% to $8.00 on Wednesday. The stock is down more than 25% year-to-date, up has risen more than 73% over the last one year.

3/1/2017
Ticker Symbol STML
Last Price a/o, 4:02PM EST  $                          8.00
Average Volume 353,760.00
Market Cap (mlns)  $                  178.24
Sales (mlns) $0.90
Shares Outstanding (mlns) 22.28
Share Float (mlns) 21.29
Shortable Yes
Optionable Yes
Inside Ownership 9.40%
Short Float 3.50%
Short Interest Ratio 2.11
Quarterly Return -37.98%
YTD Return -25.23%
Year Return 72.04%

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

Why Calithera Biosciences Inc (NASDAQ:CALA)’s Shares Soared 29%

Calithera Biosciences Inc (NASDAQ:CALA)

Shares of Calithera Biosciences Inc (NASDAQ:CALA) have been on a tear this year amid positive developments around the company such a deal with o develop and commercialize a cancer drug and disclosure of a passive stake in the company by Viking Global Investors.

The stock got a boost on Wednesday, soaring nearly 29% following an announcement that Calithera’s founder and CEO, Susan M. Molineaux, will present at the upcoming healthcare conference organized by Cowen. It seems investors are anticipating positive announcements when Molineaux presents at the conference in Boston on March 8.

Viking’s 7.8% stake in Calithera

Viking Global Investors, a hedge fund that is also invested in Facebook Inc (NASDAQ:FB), Alphabet Inc (NASDAQ:GOOGL), and Microsoft Corporation (NASDAQ:MSFT), recently disclosed owning a passive stake in Calithera. The fund owns 7.8% of Calithera, according to regulatory filings.

$53 million global licensing deal for cancer drug

In January, Calithera Biosciences Inc (NASDAQ:CALA) announced entering into a global licensing agreement with Incyte involving its immuno-oncology dug candidate CB-1158. Incyte is injecting $53 million into Calithera in exchange for rights to sell the drug as a treatment for various blood disorders in markets outside the US. Calithera will remain certain global rights to CB-1158.

Calithera and Incyte are jointly fund the development of CB-1158. Incyte is bearing 70% of the drug’s development costs, while Calithera is shouldering the remaining 30%. The companies have also agreed to share proceeds from the drug, with Calithera taking 40% of profits from the sale of CB-1158 in the US and Incyte keeping the rest. Calithera also qualifies for milestone payments that could run up to $430 million. Additionally, Calithera is also eligible for royalties from non-US sales of CB-1158.

If Calithera Biosciences Inc (NASDAQ:CALA) ditches the co-funding arrangement with Incyte for the development of CB-1158, it would forgo US profits from the sale of the drug. However, its milestone eligibility would rise to $750 million.

Targeted indications

Calithera’s CB-1158 is being developed as a treatment for various cancers such as acute myeloid leukemia, non-small-cell lung cancer, renal cell cancer and breast cancer. It can work as a monotherapy or in combination with other drugs. For instance, it has been tried as a combination treatment with Bristol-Myers Squibb Co (NYSE:BMY)’s Opdivo and showed positive results in combating cancers with arginase-secreting cells.

Stock movements

Shares of Calithera Biosciences Inc (NASDAQ:CALA) soared 28.9% to close at $11.60 on Wednesday, setting a new 52-week high mark. The stock is up more than 256% year-to-date, and has risen nearly 90% over the last one year.

3/1/2017
Ticker Symbol CALA
Last Price a/o, 4:02PM EST  $                          11.60
Average Volume 661,390.00
Market Cap (mlns)  $                   243.25
Sales (mlns)
Shares Outstanding (mlns) 20.97
Share Float (mlns) 19.66
Shortable Yes
Optionable Yes
Inside Ownership 0.30%
Short Float 4.12%
Short Interest Ratio 1.22
Quarterly Return 241.18%
YTD Return 256.92%
Year Return 87.70%

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

Amicus Therapeutics, Inc. (NASDAQ:FOLD) Receives Prestigious SOTU Attention

Amicus Therapeutics, Inc. (NASDAQ:FOLD)

Amicus Therapeutics, Inc. (NASDAQ:FOLD) a Cranbury, NJ-based biotechnology company announced financial results for the full year ended December 31, 2016 today. The Company also provided program updates and reiterated full-year 2017 financial guidance. But that was nothing compared to the coverage it received last night from the President of the United Sates in his State of the Union Address to a joint session of Congress and the American people.

Total product revenue in the full year 2016 was approximately $5.0 million, which represents commercial sales of Galafold (migalastat) in Germany as well as reimbursed Expanded Access Programs (EAPs) in two countries during the third and fourth quarter of 2016. Total operating expenses increased to $186.0 million compared to $130.4 million for the full year 2015 primarily due to increases in commercial costs of the Fabry monotherapy program and manufacturing scale-up on the Pompe program. Amicus Therapeutics, Inc. (NASDAQ:FOLD) expects full-year 2017 net operating cash spend of between $175 million to $200 million and expects full-year 2017 total net cash spend (including third-party milestone payments and capital expenditures) of between $200 million and $225 million.

Dwarfing all of Amicus’ financial reporting was President Trump’s recounting the story of Megan Crowley, daughter of John Crowley, founder, CEO, and Chairman of Amicus Therapeutics, Inc. (NASD:FOLD). Here are the President’s words:

An incredible young woman is with us this evening who should serve as an inspiration to us all. Today is Rare Disease day, and joining us in the gallery is a Rare Disease Survivor, Megan Crowley. Megan was diagnosed with Pompe Disease, a rare and serious illness, when she was 15 months old. She was not expected to live past 5. On receiving this news, Megan’s dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure, and helped develop the drug that saved Megan’s life. Today she is 20 years old — and a sophomore at Notre Dame.

Megan’s story is about the unbounded power of a father’s love for a daughter.

But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need. If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan.”

The President’s words should comfort not only people that are hoping for a better quality of life that may be found through the use of treatments and drugs that are under development, but it will also mean that, for now, there may be a quicker path to revenues for companies that are finding themselves in a cash crunch due to the current regulatory process. If the President can deliver, it will be of great benefit to a great many people.

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.

More Positive News from NeuroDerm Ltd. (NASDAQ:NDRM)

NeuroDerm Ltd.(NASDAQ:NDRM)

NeuroDerm Ltd. (NASDAQ:NDRM) shares gapped up to open Wednesday trading. NDRM ended Tuesday at $23.15 then gapped up to open at $26.50 before hitting their inter-day high of $29.75. Trading has been over ten times the average daily volumes. The spike centered on news that the company’s lead product candidate, ND0612, has met it primary and key endpoints.

NeuroDerm Ltd. (NASDAQ:NDRM) is developing a drug for Parkinson’s Disease (PD). Parkinson’s Disease is a chronic and progressive disease of the central nervous system. It affects more than 10 million individuals worldwide. NeuroDerm Ltd. (NASDAQ:NDRM)’s ND0612H is designed to deliver steady subcutaneous doses of levodopa/carbidopa via a belt pump to PD patients with inadequately controlled motor fluctuations.

When a PD patient has an “OFF” episode, the PD patients is unable to perform routine activities like eating, bathing or dressing, is believed to happen when dopamine levels in the brain fall below a certain threshold. The subcutaneous doses of levodopa/carbidopa are thought to keep dopamine at levels which keeps the patient ON, or able to function normal routine activities. Levodopa, which is converted to dopamine in the brain, is a commonly prescribed drug for the treatment of Parkinson’s disease.

The patients in the group study receiving ND0612H experienced a statistically significant reduction in daily “OFF” time from baseline to day 28. Specifically, daily “OFF”” time was reduced by almost 51% from 5.5 hours to 2.7 hours (p=0.004) in the regimen 1 cohort (24-hour administration of ND0612H at a high day rate for 18 hours and low night rate for six hours). The 38 enrolled subjects had typical characteristics for patients with advanced Parkinson’s disease including: an average age of 63.5 years, 11.5 years since diagnosis and an average baseline OFF-time of 5.3 hours per day.

NeuroDerm Ltd. (NASDAQ:NDRM)  is the first to develop liquid levodopa (LD), the gold standard treatment for Parkinson’s disease, thus enabling for the first time continuous sub-cutaneous administration of this drug.

3/1/2017
Ticker Symbol NDRM
Last Price a/o 2:33 PM EST  $                    27.50
Average Volume                    187,400
Market Cap (mlns)  $                  607.22
Sales (mlns) $0.00
Shares Outstanding (mlns) 26.23
Share Float (mlns) 15.75
Shortable Yes
Optionable Yes
Inside Ownership 52.73%
Short Float 3.54%
Short Interest Ratio 2.98
Quarterly Return 31.91%
YTD Return 4.28%
Year Return 90.07%

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.