IDRA: Idera Pharmaceuticals ($4.64)

Idera Pharmaceuticals became a new holding in December when we started buying the stock here IDRA: $3.04, and traded in /out, then back in using volatility to our advantage to buy shares back. Since then, the stock has advanced nicely to $4.64 with the addition of new key employees.  The special thing about the new personnel is that they have prior ties while serving at Genzyme, an extremely successful company that was purchased by Sanofi for $20 billion dollars.  On January 9th, 2014, two new key people were hired by IDRA.  Lou Brenner MD, has joined as senior vice president and chief medical officer, and has experience with advancing clinical trials with previous companies.  Also, Dr. Mark Goldberg has been appointed as a new board member. Previously, while at Genzyme he played a key role in the development of four successful orphan therapies. Dr. Goldberg is a board-certified medical oncologist and hematologist and has published more than 50 papers.  So the list of employees that are currently at IDRA with prior experience working together at some point at Genzyme consist of the following:

CFO: Louise Arcudi
Chairman of Board:  James Geraghty
Board Member:  Mark Goldberg
Chief Medical Officer:  Lou Brenner

Near Term Catalyst:

• 16th annual BIO CEO & Investor Conference 2-10-14.
• Phase 2 top line results IMO-8400 plaque psoriasis, first quarter.
• Phase 2 top line results IMO-8400 plaque psoriasis dose escalation .60 mg. second quarter.

Bottom Line:  Idera Pharmaceuticals is making progress behind the scenes with the addition of key personnel that have prior experience growing an emerging biotechnology company while at Genzyme.  Near term catalyst could move the stock higher in the coming weeks.  Thank you for reading.

Contact: portfoliomgt1@gmail.com

Perspective On The Recent Selloff

The current correction is -3.14% from the all time highs.  So, is this the beginning of a bear market or just the typical (-4% to -7%) pullback that we have seen a few times throughout 2013 that has led to new highs shortly after?

Above is a weekly chart which shows all the pullbacks in 2013 and the support that the 25 EMA (exponential moving average) has provided.  So, at the very least, I am expecting the market to fall to this area before reversing back higher.  A clean break and close lower of this indicator, then I think we are looking at a correction of up to -10% potentially.  

Bottom Line:  Next week will be a very important week for market direction. We have a two day FOMC meeting mid week to see if there will be further liquidity taper, as well as more earnings reports to ponder. Thank you for reading.

Contact: portfoliomgt1@gmail.com 
                 586-431-8000
 

IDRA: Idera Pharmaceuticals ($3.04)

Idera Pharmaceuticals is a new holding.  Idera is a clinical stage biotechnology company developing a novel approach to the treatment of autoimmune diseases and certain genetically defined forms of B-cell lymphoma.  Their technology platform is based on nucleic acid therapies to inhibit over-reaction of Toll-like receptors.

Quick Facts:
135 million shares outstanding including warrants
84 million in cash
5M / Q burn rate
810 Million Market Cap
2031 for IMO-8400, 2034 IMO-9200 Patent Expiration

Clinical Trials:
Phase 2 Moderate to Severe Plaque Psoriasis.
Phase 1/2 B-Cell Lymphoma with MYD 88 L265P.

Catalyst:
Phase 2 for Psoriasis top line results in January.
Complete enrollment Phase 1/2 B-Cell Lymphoma?

Bottom Line:  Idera is unique with their technology based around Toll like receptors.  The company is currently working on the dosing for their IMO-8400 and MO-9200 technologies.  They have cash for now that should last until third quarter of 2015, and also patent protection until 2031 and 2034.

ACAD: Acadia Pharmaceuticals ($23.03)

Two companies that we currently hold shares in, Acadia Pharmaceuticals and Omeros, both presented at the 24th annual Oppenheimer Healthcare Conference in New York this week.  From their presentations, I wanted to add any new relevant information for future reference.

ACAD
CEO Uli Hacksell presented on Tuesday.  Some new takeaway's from his presentation are below.
A)  He mentioned that the patent for Pimavanserin expires in 2028, but also mentioned the potential for filing a patent extension after that time period was a possibility.
B)  The other significant new information had to do with side effects and the box labeling for PDP.  He mentioned that under very high dosing (up to 300 mg) nausea was noticed.  Pima will only be prescribed at 40 mg for PDP.  No other side effects were mentioned as he said this is a very unique safe molecule.
C)  All timelines remained the same as far as filing the new drug application for PDP (by end of 2104) and phase 2 ADP data potentially taking up to two years for data.
D)  CEO expects at the very minimum equal pricing to other current atypical anti-psychotic drugs.
Another positive conference for ACAD. The CEO keeps hammering home the safety and efficacy of Pima compared to other Atypical anti-psychotics currently being used, and the potential for premium pricing for the unmet need of PDP and ADP.

OMER
Omeros CEO Greg Demopulos presented on Wednesday, some key information below.
A)  There was much mention regarding partnering for some of their indications, but no concrete details.
B)  The phase 2 Schizo data which was previously thought to be made public in December, now has been pushed back to January.
C)  They have been pursuing hard, getting medical reimbursement for OMS302 or Omidria, which is expected to be approved mid 2014 and launched there after.
D)  Nothing mentioned regarding their cash position.
The phase 2 announcement of OMS824 top line results in January, and the medical reimbursement for Omidria which is expected to launch after approval in mid 2014 are two important events.
Thank you for reading.

TRGT: Targacept ($5.75)

(TRGT) Targacept has developed what they trademark as NNR Therapeutics or neuronal nicotinic receptors. The company is dedicated to the discovery and early development of compounds that interact with nicotine receptors in the human nervous system to treat such diseases as Alzheimer's Disease, Parkinson's Disease, Tourette's Syndrome, schizophrenia and others.

Therapeutic Applications of Nicotine
Nicotine's beneficial effects on learning, memory and other physiological and behavioral endpoints have been well documented in scientific literature.  Some studies reported that smokers had a lower incidence rate of Alzheimer's and Parkinson's Disease.  It was also thought that nicotine could have a beneficial effect on schizophrenia.

Dvelopment of Novel Nicotinic Therapeutics
Companies like Targacept and Forum Pharmaceuticals (previously EnVivo), have worked to discover and develop nicotine-like therapeutic compounds that could be suitable for humans without the side effects of increase in heart rate, blood pressure and nausea.

Current Trials
Targacept  Alzheimer's Phase 2 Trial
- Ongoing with top line results mid 2014.
Targacept  Schizophrenia Phase 2 Trial
- Ongoing with top line results Dec. 13 or Jan. 2014.
Forum Alzheimer's Phase 3 Trial
- Still recruiting

The primary outcome measure in the Alzheimer trials above is the change from baseline in the Alzheimer's Disease Assessment Scale-Cognitive Subscale 13-item, or (ADAS-Cog-13).  ADAS-Cog-13 helps evaluate cognition and differentiates between normal cognitive functioning and impaired cognitive functioning.

Note that neither company or trial above is seeking a cure for Alzheimer's or Schizophrenia, but trying to improve cognition and dementia with their treatment for a given period of time, above the current standard of care.

MRTX: Mirata Therapeutics ($17.37)

Mirata Therapeutics (MRTX), is a microcap oncology company with their lead inhibitor mocetinostat (MGCD0103), an oral spectrum-selective HDAC inhibitor for the treatment of myelodysplastic syndrome or lymphoma.  The company has two other inhibitor products in early stages of testing but mocetinostat is the focus of this post because it is furthest along in the FDA approval process, with the initiation of a phase 3 trial scheduled for the second half of 2014.  The company believes they could be the first HDAC inhibtor to the market for first line myelodyspaltic patients, a disease that affects 10,000 to 30,000 people in the US and that may have a global MDS market of $1.5 billion by 2017, based on research here Global MDS Market.
Quick facts:
13 million shares outstanding
220 million market cap
68 million in cash
Patent Exp: 2022 for (mocetinostat)
13 clinical trials and 437 patients have used (MGCD0103)
Competition:  MEI Pharma (pracinostat) Patent Exp. 2028 for composition of matter.
Phase II Results  (Presented at ASCO June 2013 Abstract 7116)
The subset analysis involved 28 MDS patients.
54% had bone marrow blast count >10%.
64% categorized as intermediate or high risk.
Median age was 72.
Pre-treated with one or more prior therapy:  (50%) of patients.
Objective Response Rate:
(CR + CRi + Hl) 61%.
Disease Control Rate:  
(CR + CRi + Hl + SD) 93%.
Grade 3 drug related adverse events:
Fatigue (23%), Nausea (22%), Diahrea (17%), and vomiting (9%).
The results above compare favorably to current standard of care and is leading to a phase 3 trial for MGDC0103 in first line MDS patients second half of 2014.

Bottom Line:  The company plans to finalize discussions and obtain a special protocol assessment from the FDA for the design of a phase 3 trial for mocetinostat in patients with mylodysplastic syndrome in the first half of 2014.  There is competition from other companies that use HDAC therapy for myelodysplatic syndrom (MDS), but Mirati's data looks better than the current standard of care. The company is about a year ahead of Mei Pharma's trials where the data looks equal in efficacy so far.  The company can be first to market with an oral HDAC inhibitor in combination with AZA for patients in first line MDS.

Disclosure:  We currently do not hold shares of MRTX.

ACAD: Acadia Pharmaceuticals ($22.94)

Just released today, Acadia Pharmaceuticals has started a Phase 2 trial for ADP (Alzheimer's Disease Psychosis).  The press release with the fine details is below.  This trial will be conducted in London England.

The Phase II feasibility trial, referred to as the -019 Study, is a randomized, double-blind, placebo-controlled study designed to examine the efficacy and safety of pimavanserin in about 200 patients with ADP. The study is being conducted through a large network of research care homes established as part of the National Institute for Health Research (NIHR) Maudsley Biomedical Research Unit. Following a screening period that includes brief psycho-social therapy, patients will be randomized on a one-to-one basis to receive either 40 mg of pimavanserin or placebo once-daily for 12 weeks. The -019 Study will assess several key efficacy endpoints, including use of the Neuropsychiatric Inventory - Nursing Home (NPI-NH) scale to measure psychosis (hallucinations and delusions), agitation/aggression, and sleep/nighttime behavior, as well as use of the Cohen-Mansfield Agitation Inventory - Short Form (CMAI-SF) scale and the Alzheimer’s Disease Cooperative Study - Clinical Global Impression of Change (ADCS-CGIC) scale. Key efficacy endpoints will be based on the change at week six from baseline. The study will also assess additional exploratory endpoints, including the cognitive status of patients using the Mini-Mental State Examination (MMSE) scale, and the durability of response to pimavanserin through twelve weeks of therapy.

About Alzheimer’s Disease Psychosis

According to the Alzheimer’s Association, 5.4 million people in the United States are living with Alzheimer’s disease. While the criteria for diagnosing Alzheimer’s disease are mostly focused on cognitive deficits, it is often the psychiatric and related behavioral symptoms that are most troublesome for caregivers and lead to poor quality of life for patients. An estimated 25 to 50 percent of Alzheimer’s patients may develop Alzheimer’s disease psychosis (ADP), which is commonly characterized by disturbing visual hallucinations and delusions. The diagnosis of ADP is associated with more rapid cognitive and functional decline and institutionalization. There currently is no therapy approved for the treatment of ADP in the United States.

Study Collaboration   “We are delighted to pursue this clinical study in collaboration with Professor Ballard and King’s College London,” said Roger G. Mills, M.D., ACADIA’s Executive Vice President of Development and Chief Medical Officer. “We believe that their unique clinical research infrastructure and expertise will provide access to a pool of well-characterized ADP patients and enable the use of a small and geographically-focused group of highly trained raters, which we expect to enhance study precision.”

Bottom Line:  ACAD has given this phase 2 trial for ADP every chance of success by working with Clive Ballard and the King's College London.  The study will be a small geographically focused group of highly trained raters which should enhance the study precision.  Thank you for reading.

OMER: Third Quarter Financial Summary ($8.63)

Omeros released their third quarter earnings results on Thursday November 10th.  The summary of their financial condition is listed below.

* $26 million in cash, as of their earnings release on Thursday.
* The burn rate is approximately $4.5 million per month.
* $20 million in debt as of the third quarter with a fixed annual rate of 9.25%.  All interest and principle is due by December 1, 2016.
* MLV - The company has an open agreement with MLV to sell an aggregate of up to $49.3 million.  They sold 373,700 shares of common stock in October at the price of $13.29 per share raising $4.9 million leaving $44.4 million available for future funding.
* Omer has approximately 38 million shares including stock options and warrants.
* The company believes that with the cash on hand, approximately $26 million and the avenues to other financing such as the MLV agreement, the company has enough for at least the next 12 months.

Commercialization:
The FDA has accepted their application for OMS302 and the company is planning the commercial launch for the second half of 2014, if approved mid 2014.

Bottom Line:  Similar to other emerging biotechnology companies, financing is always a top priority with potential dilution of the shares.  We continue to like the quality, the potential of the companies pipeline, and the management team.  The stock will remain volatile in the near term, and we will use that volatility to add shares on weakness.  Thank you for reading.

ACAD: Third Quarter Conference Call ($21.00)

It was a positive 3rd quarter conference call from ACAD after hours today. The main points to consider are listed below.

A)  The company will start a phase 2 Alzheimer's trial in November with Pimavanserin for patients with (ADP) Alzheimer's Disease Psychosis.
B)  Three month stability testing has been completed in October.  Typically the FDA would like to see 12 month stability testing when submitting a new drug application.  Perhaps they can submit while continuing additional stability testing in progress.
C)  They will have 75 sales representatives in the US to sell Pimavanserin for PDP, and more than likely partner outside of U.S.
D)  Regarding the EU approval process for PDP.  ACAD will talk with the EU in the fall of 2013.
E)  Currently 200 patients have been taking Pima for over one year, and 100 patients over two years.  The longest patient has been taking Pima for eight years.  More proof of solid safety data that Pimavanserin offers that other atypical drugs do not.

Bottom Line:  ACAD is doing everything right by preparing to start the Alzheimer's phase 2 trial in November, and at the same time is readying data for FDA approval for PDP by the end of 2014.  The stock has come down considerably making the price today a good area to start a position. Thank you for reading.

GILD: Gilead Sciences Hepatitis C Pipeline ($70.97)

Gilead Sciences hepatitis C (HCV) drug sofosbuvir with ribavarin is currently under review with the FDA for stage 2 and 3 genotype infection.  Also under consideration, is sofosbuvir in combination with ribavarin and peglated interferon for the treatment of chronic hepatitis C in treatment naive adult patients with genotype 1 and 4 infection.  Approximately 75% of patients with HCV fall into the genotype 1 catagory.
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GILD is currently conducting a phase 3 trial called ION-3 for treatment naive genotype 1 infected patients. This phase 3 study was initiated from the positive results of the phase 2 study called LONESTAR.

LONESTAR Phase 2 Results

Treatment                      Duration         Population                  Results

Sofosbuvir + ledipasvir  8 weeks  GT-1 treatment naive  95%  (19/20) SVR 8

Sofosbuvir + ledipasvir  8 weeks  GT-1 treatment naive 100% (21/21) SVR 8

+ RBV            

Sofosbuvir + ledipasvir 12 weeks GT-1 treatment naive 100% (19/19) SVR 4
Sofosbuvir + ledipasvir 12 weeks GT-1 treatment exp.   95%  (18/19) SVR 4
Sofosbuvir + ledipasvir 12 weeks GT-1 treatment exp.   95%  (21/21) SVR 4
+ RBV

The phase 2 Lonestar trial results above suggest that a once daily all oral therapy of sofosbuvir + ledipasvir may have the potential to cure most genotype 1 HCV infected patients 100% at 12 weeks naive.

ION-3  Phase 3 Current Study Design

Sofosbuvir + ledipasvir 8 weeks GT-1 treatment naive
Achieved 95% from LONESTAR.
Sofosbuvir + ledipasvir + RBV 8 weeks GT-1 treatment naive
Achieved 100% from LONESTAR.
Sofosbuvir + ledipasvir 12 weeks GT-1 treatment naive
Achieved 100% from LONESTAR.

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GILD has just posted results of their PHOTON-1 phase 3 trial for patients with HCV and HIV.

76% SVR 12 for genotype 1 Hpc/HIV patients after 24 wks of sofosbuvil + ribavirin (no interferon)

88% SVR 12 for genotype 2 Hpc/HIV patients after 12 wks of sofosbuvil + ribavirin

67% SVR 12 for genotype 3 Hpc/HIV patients after 12 wks of sofosbuvil + ribavirin

The phase 3 PHOTON-1 trial results above are impressive given the fact that patient cohorts (Hep C + HIV) were the most refractory among drug therapy, most of them have failed prior treatment. 

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Bottom Line:  The $20 billion hepatitis C market has grabbed the attention of several companies attempting to enter with new and improved products.  From data researched, I believe GILD has the best product and safety profile coming to the market and the best pipeline for HCV.  Thank you for reading.

GILD: Reports 3rd Quarter Earnings

GILD - Gilead Sciences is a top selection for 2014. Today after the close, the company comfortably beat top and bottom line analyst 3rd quarter estimates and guided higher for the remainder of 2013.  Here are some highlights and future potential catalyst.
*  Today's earnings beat on the revenue side of 2.78 versus a 2.72 estimate and earnings per share was .52 cents versus an estimate of .48 cents. GILD also raised year end guidance numbers.
*  Stock buybacks will continue into next year.
*  On Friday October 25th, the FDA advisory panel voted unanimously in favor to approve Sofosbuvir to treat several forms of Hepatitis C.  The total HCV market is 10 billion dollars annually.
*  The final FDA approval could come at anytime from here through December.
*  Analyst are forecasting $1.85 billion in sales alone for Sofosbuvir in 2014, if it prices around $85,000 per patient.  I think that is rather conservative and I expect the company to beat the numbers throughout 2014.
The weekly chart below is near all time highs and appears to be overbought in the short term.  I expected to see some profit taking post report, but we will have to see how that plays our tomorrow.

Bottom Line:  GILD looks ready to have a strong 2014 with the launch of HCV drug Sofosbuvir. The key to the approval expected in December is how the FDA allows for the labeling on approval. Thank you for reading. 

OMER: ($11.41) More Positive Data Points

A PR from Omeros released on Thursday provided even more detail on a previously run FDA phase 1 trial with OMS824 (PDE10) program for Schizophrenia.  The company has been gathering some nice safety, tolerability, pharmakonitec, dosing levels, and PET scan data from just this phase 1 trial.  Below is a rundown of that data.

Phase I  OMS824 trial for Schizophrenia patients:

A)  OMS824 has achieved 63% engagement (PET scans) at a higher dose than previously reported at PDE10 and did not trigger the dose limiting side effects seen with other PDE10 inhibitors.
B)  Healthy male subjects received OMS824 once daily for seven days at a dose higher than previously evaluated in the target-engagement clinical trial.
C)  The drug was well tolerated, with mild somnolence as the only apparent side effect.
D)  In May, Omeros reported that a lower dose evaluated in an ongoing PET clinical trial demonstrated target engagement greater than had been previously reported for any PDE10 inhibitors.  The results showed a 50% occupancy at the selected dose without triggering extrapyramidal symptoms.

The company is currently in a phase II trial with OMS824 for stable Schizophrenia subjects, evaluating safety as the primary outcome, and pharmakonitecs and cognition as secondary outcome. The results will be released by the end of 2013.

Bottom Line:  Effective inhibition of the PDE10 is a promising mechanism for the treatment of Huntington's or Schizophrenia disease.  Omeros has been gathering some nice data on this previously run phase I trial, and is currently in a phase II trial which will gather even more data on the safety, tolerability, and some cognition reports may be possible.  The company has said that the cognition secondary outcome may not be substantial with a small size of just 32 patients.  Thank you for reading.