Saturday, November 16, 2013

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.

Thursday, November 14, 2013

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.

Sunday, November 10, 2013

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.

Wednesday, November 6, 2013

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.

Saturday, November 2, 2013

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.