Protalix Biotherapeutics – Using Plants to Treat Human Disease
by Pharmboy - August 17th, 2011 8:35 pm
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Protalix (AMEX: PLX) is revolutionizing the development and manufacturing of recombinant therapeutic proteins through its ProCellEx™ plant cell-based protein expression system. The company is focused on the development and commercialization of a proprietary pipeline (Figure 1) of novel and biosimilar proteins that target large, established pharmaceutical markets and that rely upon known biological mechanisms of action. Protalix’s initial commercial focus is on complex therapeutic proteins for the treatment of genetic disorders, such as Gaucher disease and Fabry disease.
Protalix is also advancing other recombinant biopharmaceutical drug development programs, including a Tumor Necrosis Factor (TNF) inhibitor for inflammatory diseases. TNF inhibitors work by blocking the action of TNF, a cytokine – a small cell-signaling protein molecule – in the body that causes inflammation. (Pubmed Health)
Tumor necrosis factors (or the TNF-family) refers to a group of cytokines family that can cause cell death (apoptosis). TNF blockers include infliximab (Remicade), etancercept (Enbrel), adalimumab (Humira), certolizumab pegol (Cimzia) and golimumab (Simponi). (Wikipedia)
Protalix’s lead product is Uplyso (taliglucerase alfa) for the treatment of Gaucher’s disease. The treatment has orphan drug status in both the EU and US. The product is partnered with Pfizer. Gaucher’s disease is a genetic disease in which a fatty (lipid) substance accumulates in cells and certain organs. Patients (males and females) have a hereditary deficiency of the enzyme glucocerebrosidase (also known as acid β-glucosidase). This deficiency causes lipids to accumulate in cells, especially those of the immune system. Uplyso is a plant-cell expressed recombinant form of glucocerebrosidase which Protalix is developing for the treatment of Gaucher disease." (Drugs.com)
About 1 in 100 people in the United States are carriers of the most common type of Gaucher disease. The carrier rate among Ashkenazi Jews is 8.9% while the birth incidence is 1 in 450 (remember this!).
In clinical trials, data from Uplyso showed that it is as efficacious as Genzyme’s Cerezyme and Shire’s Vpriv. (See comparison table at Gekkowire’s terrific site.) During Genzyme’s manufacturing issues in 2009, patients were switched over to Uplyso under a Life Savings Use Program from the Department of Health. As of November 2010, PLX submitted a Marketing Authorization Application (MAA) in the EU for Uplyso and the company is awaiting a ruling.
In February 2011, PLX…
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DiggTheravance – A GSK Love Affair
by Pharmboy - July 21st, 2011 9:30 pm
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(This post has been updated)
(THRX) is a biopharmaceutical company that is involved in discovering, developing and commercializing small molecule medicines for respiratory diseases, bacterial infections, and central nervous system (CNS) pain.
Figure 1. Theravance Stock Price the last year.
While THRX has a mixed product pipeline, a majority of its pipeline (and future) is tied up with GSK in the treatment of asthma/COPD.
Asthma comes from a Greek word meaning "panting." It is a common chronic inflammatory disease of the airways characterized by variable and recurring symptoms including wheezing, coughing, chest tightness, and shortness of breath. Doctors clinically classify asthma according to the frequency of symptoms, forced expiratory volume in 1 second (FEV1), and peak expiratory flow rate. Asthma can also be classified as atopic (extrinsic) or non-atopic (intrinsic).
Figure 2. Normal vs. asthmatic lungs/bronchi.
The causes of asthma are thought to be a combination of genetic and environmental factors (e.g., allergens). Treatment of acute symptoms is usually with an inhaled short-acting beta-2 agonist (such as salbutamol). Recent global data estimate the asthma market to be valued at $12.4 billion in 2009. It is expected to grow with a compound annual growth rate (CAGR) of approximately 1.5% from $12.4 billion to $14 billion by 2017.
Figure 3. Overlap of Obstructive Pulmonary Disease: Asthma, COPD, Bronchitis, Emphysema.
A brief description of the players in the asthma/COPD field are noted below, with GSK’s Advair and MRK’s Singulair owning a lion’s share of the market.
GlaxoSmithKline (GSK) – I wrote about GSK here. GSK owes its market-leading position in the global respiratory market to Ventolin for the treatment of asthma over 30 years ago. In 1990, the company developed and launched Serevent and Flixotide. A combination of these two compounds is sold under the brand name Seretide (Advair). The Advair combination drug consists of a corticosteroid (fluticasone) and a long-acting beta antagonist (salmeterol). It is the dominant drug in the market, with 2010 sales of $7.94B billion. It will likely remain the market leader in the current generation of asthma medication.
AstraZeneca (AZN) – Symbicort (budesonide/formoterol) is a new asthma drug that was launched in the US in mid-2007. The sales in 2010…
Accumulation – Time to Consider A Few Biotech Picks
by Pharmboy - July 4th, 2011 5:56 pm
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Seasonality stock rotation is nothing new to investors, and we at PSW have mainly stayed on the sideline in many biotech stocks for the past few months, knowing that late spring and early summer is not a time to start buying. Come July and August, though, it is time to consider accumulating scientifically sound companies for potential rewards later.
Figure 1. IBB 5 year weekly chart.
A few weeks ago, INCY and YMI were in a write-up and we jumped on the pullback in YMI’s stock price in member chat. While it is early for YMI, I still like their chances and one can buy in on the stock and selling the Jan12 $2.5 calls and puts for $1.40. INCY has a similar product that is further along, and the stock has had an impressive run up. Many think they could be a takeover by LLY, and it is not out of the realm of possibility. LLY has a horrible pipeline, and they need revenue…badly. Time will tell if a deal is done.
One of LLY’s biggest revenue generators is in the treatment of diabetes. The next pick is more of a speculative bet on a new mechanism of action for this very large medical need, and one that LLY could also take advantage of in the future.
Diabetes is a group of metabolic diseases in which a person has high blood sugar, either because the body does not produce enough insulin, or because cells do not respond to the insulin that is produced. This high blood sugar produces the classical symptoms of polyuria (frequent urination), polydipsia (increased thirst) and polyphagia (increased hunger).
There are three main types of diabetes:
- Type 1 diabetes: results from the body’s failure to produce insulin, and presently requires the person to inject insulin. (Also referred to as insulin-dependent diabetes mellitus, IDDM for short, and juvenile diabetes.)
- Type 2 diabetes: results from insulin resistance, a condition in which cells fail to use insulin properly, sometimes combined with an absolute insulin deficiency. (Formerly referred to as non-insulin-dependent diabetes mellitus, NIDDM for short, and adult-onset diabetes.)
- Gestational diabetes: is when pregnant women, who have never had diabetes before, have a high blood glucose level during pregnancy. It may precede development of type 2 DM.
Treatment…
The Change is Coming – Biotech Watchlist
by Pharmboy - June 18th, 2011 5:24 pm
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Over a year ago, I noted that the winds of change were coming for the pharmaceutical industry. This week, a very good article came out in the Guardian that stated two of the largest firms in the business, GSK and AZN are getting out of the neuroscience business. This is a huge blow to those that suffer from mental disorders such as bipolar, depression, anxiety, schizophrenia, OCD and the likes. The reason for this, according to David Nutt, a professor of Neuropsychopharmacology at Imperial College in London, is:
Despite the public health imperative, not only has EU research funding remained very low, but – even worse – big pharma is increasingly coming to see research into better neuropsychiatric drug targets as economically non-viable.
There are a number of reasons why companies are leaving the field, according to the report. Medicines for brain disorders take longer to develop than for other conditions – on average, 13 years – and there is a high failure rate.
Figure 1. Prevalence of Mental Disorders in the US from NIMH.
The brain is one of the last unknowns of medicine. It is a complex network of cells that interact to enable humans to hear, see, talk, feel, remember, experience emotion, and it controls our ‘biological clocks.’ I believe that the brain will be the ultimate frontier for personalized medicine. Mental disorders need to be treated on an individual basis. There is nothing worse than having a loved one or someone very close to you who suffers from these debilitating disorders. The patients require time with a psychiatrist/psychologist to be properly diagnosed. The varying treatment regimens take time, and in many cases patients will have to go on and off multiple medications until the right combination and dosages are attained. The drug combinations can take years to complete and the patient must want to get better and those around must provide support and compassion that the patient requires for successful treatment. Further, those close to the patient must try to understand that it is very hard for the patients to describe, with words, what they are experiencing.
The most important things are the hardest things to say. They are the things you get ashamed of, because
…
Robert Reich – What’s Wrong With the Economy in 2 Minutes
by Pharmboy - June 18th, 2011 12:54 pm
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By PragCap, with graph courtesy of Gordon T. Long
Robert Reich describes what’s wrong with the economy in 2 minutes. His bullet points (thanks to Stephanie Kelton):
- The economy doubles since 1980, but wages flat. Where did the money go…
- All (or most) of the gains went to the super rich. And…
- With money comes political power. Taxes on super rich slashed, revenues evaporate. This leads to…
- Huge budget deficits. Middle class agitated, fights for scraps…
- Middle class divided. Buying and borrowing slow. Resulting in:
- Anemic recovery/economy.
I would add one very important point that Mr. Reich misses here:
The real destruction has come in the growth of the financialization of the US economy. Since the 1970′s when the financialization of the USA began we have seen an increasing number of would-be entrepreneurs leave productive positions for Wall Street jobs where they largely help devise ways to help separate the middle class from their savings.
As the country grew more and more wealthy in the 1980′s and 1990′s (thanks to entrepreneurs like Bill Gates) the problem compounded because the demand for Wall Street’s services expanded (higher wealth meant higher demand for protecting that wealth). Wall Street convinces Main Street that the best way to protect their wealth is by giving it to them (2% at a time) and Main Street doesn’t know any better because they don’t (and still don’t) understand how the monetary system or the economic system in the USA actually works so they give their money to a trusted “expert”.
…Now we all suffer because of this cannibalization of capitalism.
Full article by PragCap here.
United Therapeutics – Growth Engine for Cardiovascular Diseases
by Pharmboy - May 31st, 2011 7:05 pm
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Here’s a company that Pharmboy wrote up in April – with several updates. The stock has been bouncing around the price it was trading at when Pharmboy first entered a long position, and he still likes this trade. Caveat: Phil likes the idea of waiting for the company to "screw up" and the stock to drop lower before entering a position.
That’s why there are buyers and sellers, but there are not promises.- Ilene
United Therapeutics (UTHR) is a biotech company developing and selling pharmaceutical products for the treatment of cardiovascular disease. It has also been expanding into the viral and cancer arenas. The company has been profitable since ’04, and the stock has had a nice run since the market crash in 2008, rising from $24/share to its current price of around $64.50 (as of May 31, 2011). (Figure 1)
Figure 1. 2 yr stock price of UTHR
I think the stock still has strong potential to go higher. The company has a market cap of almost $3.8B, a forward looking P/E value of 20, and a operating margin of 27%. Revenue growth as been a whopping 52% year over year, and UTHR’s mainstay product, Remodulin, used to treat pulmonary arterial hypertension (PAH), is making in-roads into a very competitive field.
As discussed in the company’s February earnings call, UTHR’s total revenue for the three months ended December 31, 2010, was $166.5 million, up from $108.9 million for the quarter ended December 31, 2009. Net income for the three months ended December 31, 2010, was $9.5 million or $0.17 per basic share, compared to a net loss of $3.3 million or $0.06 per basic share for the quarter ended December 31, 2009. For the year ended December 31, 2010 (Table 1), UTHR had net income of $105.9 million, or $1.89 per basic share and $1.78 per diluted share, compared to $19.5 million, or $0.37 per basic share and $0.35 per diluted share, for the year ended December 31, 2009. Bottom line: revenues and income are growing rapidly!
Table 1. 2009 vs. 2010 Revenues by Product
| 2009 | 2010 | |
| in $M | ||
| Remodulin | 332 | |
…
Personalized Medicine for the Masses
by Pharmboy - May 24th, 2011 10:39 pm
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Courtesy of Pharmboy at Phil’s Stock World
This article was originally published at Phil’s Stock World, and has been updated.
Costs of everything from food, oil and medicine have been increasing for American consumers. Health care is one expense that is increasing faster than inflation. Over the past 12 years, prescription drug prices have outpaced the two largest health care costs, hospital care and physician services (Figure 1). However, pharmaceuticals only make up 10% of all health care costs, while hospital and doctor costs make up 52% of the total $2.3T (Figure 2). More effective prescription drugs and vaccines have transformed health care over the last several decades and many health problems have been prevented, cured, or managed effectively through the use of these agents.1 In some cases, the use of prescription medicines have kept people from more expensive options such as hospitalization and/or surgery.
Figure 1. Percent increases of Prescription Drugs, Hospital Care and Physicians (2010, Kaiser)
Figure 2. Health Care Costs (2010, Kaiser)
Total = $2.3 Trillion
Source: Centers for Medicare and Medicaid Services, Office of the Actuary, National Health Statistics Group.
The pharmaceutical industry is headed for a patent expiration cliff, as I pointed out last year (here). The NY Times wrote covered this problem recently (here). Pharmaceuticals make up a big part of our life. In 2007, 90% of seniors and 58% of non-elderly adults relied on a prescription medicine on a regular basis.2 While the public demands safe, effective drugs, the costs to bring these medications to market keep increasing. Development costs have soared according to a recent analysis by Tufts. Tuffs’ research showed a 64% increase in the cost to discover and bring a new drug (not a reformulation or recombination of an existing drug) to market from $802M in 2000 to $1,320M in 2006. The Pharmaceutical industry must recoup the R&D costs for drugs that make it to market, as well as those that do not. Only one in five drugs that enter the clinical testing process receive FDA approval and are brought to market.3 The pharmaceutical industry must reinvent itself, and I think personalized medicine will be part of its reinvention and the next wave to take the industry by storm.
Personalized medicine is about devising a treatment plan as individualized…
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Philip R. Davis is a founder Phil's Stock World, a stock and options trading site that teaches the art of options trading to newcomers and devises advanced strategies for expert traders...
Ilene is editor and affiliate program
coordinator for PSW. She manages the Favorites backup site
(