The GLG Pharma STAT Blog

STAT3, Cancer and Genetics - GLG Pharma

Posted by Richard Gabriel on Wed, Dec 26, 2018 @ 11:59 AM

STAT or Signal Transducer and Activator of Transcription is a biological process in our normal cells that is as biologists like to say 'highly conserved'. At the Moffitt Cancer Center, the work of researchers funded by the NIH has led to the important discovery that certain compounds inhibit STAT at the phosphorylation, dimerization and also at the DNA binding site. Unlike the trans-membrane activated receptors which are 'up-stream' from STAT, STAT is a biological function that works ultimately on the DNA.

This has led some researchers to worry about the toxic effects of STAT inhibiting compounds and frankly it is a justified concern. STAT is an important step in the reproduction of individual cells in our bodies and because it is highly conserved it is turned on very few times in a cell's life. Mostly during the cell division stage.

cancer stat 3, cancer and stat 3, stat 3 inhibitors

Cancer cells however, especially when they are metastatic or rapidly reproducing, have been shown to have STAT turned on all the time. So in cancer therapy, it is always a treatment that works specifically on cancer cells because they are rapidly reproducing or they are continually robbing nutrients from normal tissue. STAT inhibitors offer promise for treating metastatic cancers that are essentially run-away cancer cell reproduction in combination with standard chemotherapy treatment. Moffitt researchers have shown STAT to varing degrees of activation in over 23 different cancer types, including breast, pancreatic and neural cancers.

Cancer and stat 3, stat 3, stat 3 inhibitors, stat and cancer

The reason that the Moffitt is so interested in STAT3 inhibiting technologies is because we also have the ability to test for the activation of STAT3 in cancer cell types. Along with a person's cancer cell genetics as well as their personal ancestral genetics, tailoring a treatment regime for the patient with lower toxicities and higher efficacy is one of the major goals of our program. That is also why the Moffitt Cancer Center is also an investor in GLG Pharma. 

In these difficult economic times, GLG Pharma has found many new sources of funding to develop its STAT inhibiting technologies so that they may begin clinical trials as soon as possible. It is clear to all of us that work in biotech and pharmaceutical companies that there has to be a better way to fund the development of these important diagnostics and therapeutics. All of our current animal studies have shown that our STAT inhibitors have low toxicities but inhibitory effects on cancer growth and when combined with traditional chemotherapy could provide the knock out punch to the metastatic cancer cells. GLG has received support from local, State and Federal agencies to help develop its products and technologies. If you want to learn more about GLG, contact us at GLG Pharma.

Tags: GLG Pharma, STAT3, STAT3 inhibitors, STAT

GLG Pharma STAT 3 Blog

Posted by Richard Gabriel on Wed, Dec 26, 2018 @ 11:57 AM

Welcome the first blog of our new website, GLG Pharma, LLC. We will try to cover a variety of topics related to STAT 3 inhibitors, cancer in general as well as other informative articles that we find on health, nutrition, exercise and cancer prevention and treatment.

Cancer cells, STAT3 and cancer, STAT 3, cancer treatment and STATs

GLG Pharma, LLC is a Florida based company, located in Jupiter, Florida, near the Scripps Institute, Florida Atlantic University and the Max Planck Institute. It has affiliations with and has private equity partnerships with the Moffitt Cancer Center in Tampa, Florida, the Town of Jupiter and the Paragon Foundation of Palm Beach County.

If you want to learn more about our company, give us a call or send us an inquiry. We are happy to answer most questions. Our development plan for our four targeted products is emerging and we will from time to time report on our progress in pre-clinical and clinical development of these novel and exciting compounds for the inhibition of the STAT 3 mechanism in cancer cell metabolism. 

Tags: STAT3, STAT 3 inhibitors, STAT3 cancer, cancer diagnosis, STAT

GLG Pharma Announces Presentation at Upcoming AACR, the Annual Cancer Research Meeting 2015 April 18-22, 2015

Posted by Richard Gabriel on Mon, Mar 23, 2015 @ 03:30 PM

Jupiter, FL - March 23, 2015 – GLG Pharma, LLC, focused on developing drugs to prevent/treat cancer as well as other diseases mediated by dysfunctional STAT3 signaling, announced a NCI data presentation at the 2015 American Association of Cancer Research (AACR) Annual Meeting being held April 18-22, 2015 at the Pennsylvania Convention Center in Philadelphia, PA. The study was sponsored by the PREVENT Cancer program of the National Cancer Institute of the NIH. The PREVENT Program is a National Cancer Institute-supported pipeline to bring new and novel cancer preventing interventions and biomarkers through preclinical development towards clinical trials.  

The details for the data presentation at AACR are as follows:

Poster Presentation

Abstract Number:


Presentation Title:

Targeting STAT3 for mammary cancer prevention in MMTV/Neu mice employing the antagonist GLG-302

Presentation Time:

Tuesday, Apr 21, 2015, 1:00 PM - 5:00 PM


Section 37

Poster Board Number:


Author Block:

Robert H. Shoemaker1, Michael W. Lovell2, John J. Whalen2, Fariba Moeinpour3, Clinton J. Grubbs3. 1National Cancer Institutes, Bethesda, MD; 2GLG Pharma, LLC, Jupiter, FL; 3University of Alabama at Birmingham, Birmingham, AL

About GLG-302

GLG-302 is an orally available compound designed to target dysfunctional STAT3 signaling in cancer and other diseases. In cancer, dysfunction STAT3 signaling is found in tumors, cancer stem cells and cells that suppress the immune system. Cancer stem cells are an underlying cause of tumor resistance to chemotherapy, recurrence and ultimate disease progression. Immune suppression is associated with ultimate disease progression. In pre-clinical studies, GLG-302 has been shown to suppress the growth of breast cancer and other tumors, in vivo, has been reported to suppress the growth of glioblastoma stem cells and has been reported to modulate the immune microenvironment of glioblastoma, as well as in the blood of cancer patients with urothelial and renal carcinomas, in vitro.  GLG-302 was well tolerated in pre-clinical toxicology studies.

About GLG Pharma

Founded in 2009 and located in Jupiter, Florida, GLG Pharma, LLC is a privately held, early stage, biotechnology company developing personalized therapies for patients with cancer and other proliferative diseases. GLG Pharma’s therapeutics are expected to aid in the treatment of a wide variety of cancers and address unmet needs in the multi-billion dollar anti-cancer market with potentially greater efficacy and fewer side effects than existing therapies. For more information on GLG Pharma visit:


For information on investing in GLG Pharma visit: for Accredited Investors following Regulation D 506(c).


The PREVENT Cancer Drug Development Program is a National Cancer Institute-supported pipeline to bring new cancer preventing interventions and biomarkers through preclinical development towards clinical trials.

PREVENT is an active NCI partnership with successful applicants to enable the milestone-driven progression of novel cancer preventive chemical or biological agents (singly or in combination) and biomarkers from the lab bench towards proof-of principle clinical testing and registration or validation.

PREVENT is a flexible entry-point system that will optimize and test an agent depending on the stage of development, with the goal of filing an Investigational New Drug Application with the U.S. Food and Drug Administration and entering it into clinical testing.




 Michael W. Lovell, PhD

Executive Vice President

GLG Pharma, LLC

601 Heritage Drive, Suite 223

Jupiter, FL 33458





 Hector J. Gomez, MD, PhD

President & Chief Executive Officer

GLG Pharma, LLC

601 Heritage Drive, Suite 223

Jupiter, FL 33458

Office 561 847-4926

Fax 562 365-6044





GLG Pharma at Rare Disease Day Tallahassee Florida!

Posted by Richard Gabriel on Wed, Feb 18, 2015 @ 04:47 PM


GLG Pharma is participating along with Rare Disease Advocates Commemorate Worldwide Awareness Day at the Florida State House 

Tallahassee, Florida, February 19, 2015----- Join rare disease patients, caregivers and other health care advocates as they share their stories on February 19, 2015, to observe Rare Disease Day in Florida. Rare Disease Day occurs each year on the last day of February, and on this day, millions of patients and their families will share their stories to focus a spotlight on rare diseases as a global public health concern. The day is observed in more than 70 nations. 

Patient advocates have joined with the National Organization for Rare Disorders (NORD), the national sponsor of the day in the US, to organize this special event for legislators, legislative staff, the public and the media. The event will be held in the State Capitol in the entry level of the Rotunda, from 9:00 am –11:00 am.  

During this event attendees will have the opportunity to meet/ hear from Florida Rep. Mike La Rosa District 42, David Dittman – Shriners Hospital, Tampa, Bella’s Fight, Felecia Peete adoptive mother of 3 boys who are affected with ALD, and Shannon Brown-Brinson – United For ALD, Inc. The topic we will be discussing will be common challenges that the rare disease community faces pertaining to care, diagnosis, and awareness. GLG Pharma will have a table, featuring its STAT3 inhibitors that may be used to combat rare diseases. GLG have identified over 45 rare diseases where the mechanism of STAT3 is turned on leading to excessive cell proliferation. Normally, STAT3 is only turned on when a cell in body divides. In cancer and proliferative diseases or diseases where cell proliferation is a product of the disease, STAT3 can be turned on up to 100% of the time. 

The purpose of this event is to raise awareness at the state level for the 1 in 10 individuals living with a rare disease and the challenges they face. Many important decisions related to rare diseases are made at the state level, and the implementation of the Affordable Care Act has highlighted the increasingly important role of state policies and programs in assuring that the healthcare needs of the American public are addressed. 

Issues of importance to the rare disease community that may be decided at the state level including newborn screening, support services for families coping with complex medical needs, an environment that promotes innovative medical research and product development, and insurance practices that assure patient access to medically necessary therapies. 

A rare disease is one that affects fewer than 200,000 Americans. There are nearly 7,000 such diseases affecting nearly 30 million Americans, according to the National Institutes of Health (NIH). Two-thirds of those affected by rare diseases are children, and the diseases tend to be serious and lifelong. Even so, most rare diseases have no approved treatment, and many are not even being studied by medical researchers. Often, research on rare diseases is funded by the families and friends of patients or by patient organizations. 

Participating Organizations Include: NORD, United For ALD, Inc., and Shriners Hospital in Tampa

Rare Disease Day was launched in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe. It is now observed in more than 65 nations, and is sponsored in the U.S. by NORD. 

For more information about Rare Disease Day in the U.S., go to For information about global activities, go to 

For more information about GLG Pharma visit our website at 

For more information if you are an accredited investor interested in helping GLG Pharma focus on rare diseases then visit and register. It’s safe, secure and confidential. There is a better way to fund Biotech!


Tags: Rare Diseases

Why Work on Treatment or Cures for Rare Diseases?

Posted by Richard Gabriel on Wed, Jan 28, 2015 @ 04:15 PM

“Highlighting Chronic Lymphocytic Leukemia (CLL)”


CLL, STAT3, GLG Pharma, STAT3 Inhibitors


The goal of every pharmaceutical and biotechnology scientist, physician and clinician is to save the patient’s life through an outright cure of the disease and if it can’t be saved then improve the quality of his/her life. Below are definitions of Rare Diseases, according to our friends at Evaluate Pharma[1]:

Evaluate Pharma Excerpt:

“The National Organization for Rare Disorders (NORD), which was instrumental in establishing the Act, currently estimates 30 million Americans suffer from 7,000 rare diseases. Prior to the 1983 Act, 38 orphan drugs were approved. To date, 468 indication designations covering 373 drugs have been approved. The success of the original Orphan Drug Act in the US led to it being adopted in other key markets, most notably in Japan in 1993 and in the European Union in 2000. Rare Disease Patient Populations are Defined in Law as:

  • USA: <200,000 patients (<6.37 in 10,000, based on US population of 314m)
  • EU: <5 in 10,000 (<250,000 patients, based on EU population of 506m)
  • Japan: <50,000 patients (<4 in 10,000 based on Japan population of 128m)

Financial Incentives by Law Include: Market Exclusivity

  • USA: 7 Years of marketing exclusivity from approval. Note: Majority of orphan drugs have a compound patent beyond 7 years. The market exclusivity blocks ‘same drug’ recombinant products, e.g. Fabrazyme (Genzyme, now Sanofi) vs. Replagal (Transkaryotic, now Shire). ‘Same drug’ exclusion can be overturned if clinically superior (mix of efficacy/ side effects), e.g. Rebif overturned Avonex’s orphan drug exclusivity (7 MAR 2002) 
  • EU: 10 Years of marketing exclusivity from approval.

Reduced R&D Costs:  

  • USA: 50% Tax Credit on R&D Cost
  • USA: R&D Grants for Phase I to Phase III Clinical Trials ($30m for each of fiscal years 2008-12)
  • USA: User fees waived (FFDCA Section 526: Company WW Revenues <$50m) 

Methodology on Classifying an Orphan Drug:  

"We, (Evaluate Pharma) have identified all products that have orphan drug designations filed in the US, EU or Japan. These are available as part of the core EvaluatePharma service. To further enhance analysis, we have defined a clean ‘Orphan’ sub-set of products following a number of criteria including:

  • First indication approved is for an orphan condition.
  • Products expected to generate more than 25% of sales from their orphan indications. 

This has led to the exclusion of drugs such as Avastin, Enbrel, Herceptin, Humira and Remicade, all of which have orphan designations for indications contributing less than 25% of sales.

  • Trial sizes, with smaller Phase III trials suggesting orphan status.
  • Drug pricing, higher prices were taken as an indicator of orphan status.
  • All sales analysis in the report is based on this clean ‘Orphan’ sub-set of products.” End of Evaluate Pharma excerpt. 

Chronic Lymphocytic Leukemia (CLL) is considered a ‘rare disease’. A great source of information on CLL[2] is the Leukemia & Lymphoma Society[3] which has over the years poured over $1 billion into research for Leukemia.  The information found in the PDF download (see reference) is excellent and is a foundation for understanding this disease and other leukemia’s as well.  

Some rare diseases have identified mechanisms of action that lay across the biological human horizon of diseases but aren’t as highly expressed or manifest themselves as a chronic lethal disease. One of the mechanisms sometimes associated with proliferative diseases that includes rare forms of cancer, is an abnormality in a major signaling pathway located downstream where many other pathways convey extracellular signals into the nucleus.  This is the case of the Signal Transduction and Activators of Transcription (STAT) and in particular, STAT3.  

At GLG Pharma we have focused on the STAT3 signaling pathway and its uncontrolled hyperactivity. Activation of STAT3 can be blocked at three different sites: 

  • Phosphorylation
  • Dimerization
  • DNA Binding

 STAT3 Inhibitor, STAT3, GLG Pharma, GLG-801, GLG-302

Three drugs are in the development pipeline. The first with the shortest path to the market is GLG-801. This is a repurposed drug and under US FDA rules for 505(b)(2) could be fast tracked for various indications and clinical trials might be initiated, as soon as funds become available, in patients with  rare diseases such as Chronic Lymphocytic Leukemia (CLL) and Gastro-intestinal Stromal Tumors (GIST). GLG-302, a new chemical entity (NCE) follows in the development cycle and is expected to be in the clinic in about 8 months from funding. GLG-202 is another NCE that will follow in the development cycle. GLG-801 inhibits DNA binding and both GLG-302 and GLG-202 inhibit dimerization of the STAT3 molecule, preventing its penetration of the nuclear membrane and the initiation of the transcription process and the continuation of the uncontrolled proliferation process.

Want to know more about GLG Pharma and Poliwogg? Raising awareness and helping us fight cancer! Then click on the Poliwogg picture and it will take you to the Poliwogg accredited investor site!

Poliwogg, GLG Pharma, STAT3 inhibitors, CLL, Chronic Lymphocytic Leukemia

Want to find out more about what compounds are in the clinic for CLL? Then click the button. Once you have signed up, we will email you the PDF document that provides you with compound structures and data on the activity of the compounds as well as their site of action on the leukemia cells!

 CLL in Clinic!

[1] Evaluate Pharma Report “Orphan Drugs 2014”



Tags: GLG Pharma, GLG, STAT3, STAT3 cancer, STAT3 inhibitors, Cancer, cancer diagnosis, STAT, cancer prevention, Alpha-1, Cancer Therapy, Rare Diseases


Posted by Richard Gabriel on Tue, Jan 06, 2015 @ 02:54 PM

(What's In It For Me?)

Many years ago Maureen Doerr at Doerr Associates[1], (pronounced Door) explained to me how to sell an idea to an unknown prospect. Notice that I said prospect and not customer because Maureen reasoned, rightly so, that a prospect becomes a customer when they buy something from your company. A prospect sometimes doesn’t know they want to buy something from you until you Advertise or Promote the ‘something’. Stick with me on this.  WIIFM is simply “What’s in it for me”? It has stuck with me ever since.


Maureen Doerr, WIIFM, Doerr Associates, Advertising, Marketing 

Maureen Doerr, President Doerr Associates 

This is a simple concept that should, theoretically, drive just about everything your organization does to make your products and services and your company a success, including research!  All facets of the business you are planning to build should be geared to answering the WIIFM of your prospects and also your continuing customers. And making sure that what is driving your research and development and ultimately your manufacturing, marketing and selling engine, is focused on answering the WIIFM for new prospects, new customers and loyal customers. The answers to each group may not necessarily be the same. Sometimes the products aren’t the same either, think Sonites and Vodites[2], for those of you that know what those two products are, you know what I’m talking about…for the rest of you, try googling the terms, its fascinating stuff! 

In health care, especially in disease oriented healthcare, our prospects are the patients, advocacy groups, healthcare insurers, government agencies, physicians, clinicians, families, institutions and the research and development community at large. Add to that the investment community which includes venture capital, angel capital, friends and family, private equity, institutional capital, research funds and grants, government funds plus the stock market; it’s a pretty complex scenario. It is so complex, that everyone tries to simplify it just in order to try and understand or attempt to understand just what the heck is going on in the market. I know I have that problem; this is way too complex for my puny brain.  

Oversimplification can at times add to the confusion and miss the WIIFM of our target prospect audience, new customers and existing customers. Sometimes these groups don’t want the same thing. Take me for instance, I love my Blackberry (still do) and everyone laughs at me. But I grew up with a Blackberry when it would only do email and within a company network, you could do something that today is called ‘texting’ from one person to the next, securely

If Blackberry had been paying attention to Apple, Microsoft, Google and others including Samsung, and the younger generation, they might still have the lion’s share of the market or at least a cougar’s share. But they didn’t pay attention to WIIFM's. So how did that happen? I don’t know and I’m not smart enough to guess. Only the ex-management team and employees of Blackberry (RIM) could tell us. Same thing happened to Xerox, IBM, Bell and Howell, Polaroid, Atari and countless others. The same process will happen to the many now ‘darlings’ in healthcare. It seems impossible for any of us to see past the ends’ of our noses and it seems almost impossible for any of us to predict what the next new technology breakthrough will be or where it will occur across the globe. Blackberry always satisfied my WIIFM’s because I am a loyal customer, stupid, stubborn but loyal. 

I’ve always tried to put myself in the shoes of the prospect that I want to talk to. Sometimes this works, but not always. The prospects I want to talk to are the 8.5 million qualified accredited investors[3] in the United States BEFORE the Securities and Exchange Commission shrinks that number down to about 2-3 million through added restrictions. Everyone wants to talk to them and so far, as a group, they are pretty clammed up. I don’t think we yet understand their WIIFM’s. Or at least I don’t. 

A really good summary of the Accredited Investor market was put out by the Angel Capital Association and addressed to the Honorable Mary Jo White Chairperson of the Securities and Exchange Commission[4] calling for White to NOT restrict Accredited Investor Requirements further as only about 200,000 of the 8.5 million Accredited Investors actually participate in Regulation D investments. Just to provide some perspectives, according to the article, there were 31,000 Reg D offerings issued in 2013 compared to 954 public offerings. In that same year, $980 billion was invested under Reg D (granted they were not all accredited investors because it includes businesses and institutional investors etc.) compared to $250 billion in the IPO market raised during the same time period.


SEC, Mary Jo White, Chairperson, Regulation D, Reg D 

The Honorable Mary Jo White, Chairperson of the SEC 

So let’s do some elementary simple math of averages:


Reg D, SEC, Regulation D 

Only 2.3% of the total Reg D market is composed of Accredited Angel Investors capital ($23billion) and they on average risk $115K of their average $2.5MM net worth or less than 4.6% of this AVAILABLE resource for investment! Looking at this issue from the far-out and somewhat unreliable perspective of averages, the total 8.5 million accredited investors who have an average net worth of, wait for it, at a Net Worth of $1.0mm represent about $8.5 trillion or some percentage of that number, to possibly invest or put at risk. And of that 8.5 million Accredited investors, the the current $23billion represents 0.27% of their average net worth! In other words, what makes the SEC think that suddenly 99.7% of the NON PARTICIPATING accredited investors are suddenly going to throw all of their assets to the wind and invest in Regulation D companies? 

So why is the SEC so focused on Restricting Accredited Investors? They hardly made a dent in the Regulation D market! Don’t get me wrong, if you or me as an entrepreneur, got funding from one of the 200,000 investors in 2013 we would be delighted! 

Reality is that nearly all of the Regulation D market is composed of SOPHISTICATED Institutional investors who are by the way, capturing all the upside of new companies that are now listing on the exchanges! 

Obviously, we as an investment seeking community of struggling lifescience entrepreneurs are seeking investments for our research and development endeavors to cure, treat or eradicate disease, don’t know the WIIFM of the other 8.3 million Accredited Investors, many of whom remain silent, unapproachable and generally disinterested in healthcare, until it’s too late, either for them as individuals or for a family or friend suffering from the sudden onset of a disease or chronic ailment. Then, sometimes, they may become motivated. 

So do the math. For 31,000 offerings that are on the Reg D filing there are probably another 10x that never make it to that stage, 300,000 plus good ideas that go virtually nowhere or flounder: in regione vivorum mortuis[5]. The Secretary of Energy, Steven Chu supposedly coined this term, ‘the valley of death’, apparently in 2009; but I swear I have heard it before[6]. This Valley of Death is the no-finance-available zone between idea and application and the finding of customers that are willing to pay for the discovery, whether it’s a product or a service. 

First and foremost, Reg D filings are NOT CROWD FUNDING investment opportunities. REGULATION D is presently reserved for the very rich as well as institutions, funds and capitalists who have gathered the wherewithal to fund new startups or advance a startup from a slow startup growth to accelerated high growth by providing capital for expanding manufacturing, marketing and sales to meet the new demand. So far, 8.3 million rich, accredited investors are not interested in participating in one of the largest investment opportunities in the history of the United States. Why is that? Well we haven’t figured out their WIIFM’s, now have we?

Interesed in GLG Pharma and our fight against cancer and proliferative diseases? Visit us: GLG Pharma
Want to find out more about Accredited Investor Funding to fight cancer? Visit: Polliwogg
poliwogg,, accredited investor, glg pharma, glg, cancer





[5] in regione vivorum mortuis: “In the Land of the Living Dead”


Tags: GLG Pharma, accredited investor, angel investor, angel investors, accredited investors, WIIFM, GLG

SEC Reviews Accredited Investor Requirements

Posted by Richard Gabriel on Fri, Jan 02, 2015 @ 12:33 PM

This is a short blog: The current debate at the SEC going on right now (December 17, 2014 and we have 60 days to comment on this as the general public) is about the definition of an accredited investor. There are two interesting presentations and anyone can down load by following the link we provide (click on Mary Jo White's picture).

Be your own judge if you think that tightening the restrictions will help or hurt our economy and the entrepreneurs starting new businesses or will it just help widen the income gap between the really rich and the not so really rich? Here are some facts:

  • There are an estimated 8.5 million accredited investors in the US
  • 400,000 to 500,000 tops, are active private equity investors in startup companies
  • Regulation D investment for 2014 will come in at about a total of $980 billion
  • Venture capital about $30-$50 billion (estimate)
  • Angel capital about $25-$35 billion (estimate)
  • Private equity - $350 billion+ (est.)
  • The rest ($550+ billion) is who knows?
  • The IPO market will be about $250-$300 billion (est.)

 SEC, Chairman, Accredited Investor

 Ms Mary Jo White

SEC Chairperson

Just click the image and it will take you to the SEC site where you can download the two presentations. You will see the link on the right hand side under the date: December 17, 2014.

If you are a visual person, then you can go to the following two links and if you have Windows Media player, you can actually hear who is saying what in the meeting!

SEC Recorded: Session 1 and 2



Thanks and good luck listening and reading. For entrepreneur's this is a vital discussion!  













Tags: accredited investor, angel investor, angel investors, accredited investors, sec, jobs act

Why Invest in Early Stage Drug Development for Rare Diseases?

Posted by Richard Gabriel on Mon, Dec 08, 2014 @ 02:54 PM


Ok, so in one of my past lives, as a member of our team at Pharm-eco Laboratories and with an excellent team at Vertex, together we worked on and helped advance Vertex’s Amprenavir (GSK’s Agenerase) which is now known as Phosamprenavir.   Amprenavir used to be known as VX-478 and we did scale up process development under cGMP and made a couple of tons of API. The discovery projects we worked on were mothballed or sent to someone else, who knows. 

Vertex bought Aurora in 2001 for an all-stock deal of $600MM and inherited the cystic fibrosis project in 2001. Aurora started in 2000 working on the project with the Cystic Fibrosis Foundation. The first capital investment by the CFF was $40MM to Vertex, the new landlord of a plethora of new screening technologies. 

The real point of this blog is this. $150MM invested in the Vertex technology over a nearly 13 year period, so about $11+ MM a year, average and the Cystic Fibrosis Foundation get back $3.3 Billion! 

So, we know the last two years sales for Vertex were $371MM and $470MM and assume that Vertex agreed to a 9% royalty (just a guess, it could be lower) to the Cystic Fibrosis foundation, then over a FIFTEEN YEAR (we added two years, one year on either end for negotiations etc.) period, giving the current payout, the Net Present Value of the $150MM investment; if you did the investment all at once with a discount of 8% over the period, is $915 million! Is that good enough for a VC firm? I don’t know but it sure is good enough for me! 

For more information on the deal, please go to an excellent article by Xconomy’s Deputy Biotech Editor Ben Fidler at or just click on the picture below (also from the article by Ben Fidler, Thanks!).

STAT3, GLG Pharma, cystic fibrosis, STAT3 inhibitors

So why pay $3.3 billion for a drug that has a small market potential? Or a portfolio of drugs with a small market potential? Well, because it isn’t a small market potential! The disease target is also present in a variety of sub, less toxic and less lethal diseases and the mechanism is spread across much wider populations around the globe! Vertex was formed when Josh Boger came out of Merck and having worked for Merck back when Roy Vagelos ran the company, he had and still has a very impressive personal activity in promoting drug discovery and development in New England and around the world. I remember visiting Vertex for the first time when there were only 40 employees. Most of them were ex-Merck and ex-Biogen…great team! 

The drug targeting philosophy that Vertex probably does maintain to this day  is identify mechanisms of targeted therapy that could have broader applications in other diseases and focus on the rare diseases to get the FDA’s attention, fast track, cut time to market and then get the drug approved sooner rather than later. Well Cystic Fibrosis took a lot longer but the payoff was worth it for the foundation. Why is this model important? Because the foundation now has enough cash to really pour some capital into finding a cure, which has always, been their goal as it is for many other foundations. $3.3 billion sets up a heck of an annuity stream! 

Bottom line message is that this is a great case study and business model for a new way of funding biotech and pharma and that could revolutionize our industry! It’s not just a ‘buyout’ it is a reward to the committed not-for-profit groups that slog day after day against the insurmountable odds of the disease to raise capital for research and development for new therapies, against diseases that are unforgiving, ruthless and terminal. But this time, ~ thirteen years later, they are delivering on their commitment! Two new drugs for cystic fibrosis patients and the foundation now has a ton of capital to use for protecting its objective from the vagaries of the markets and funding drives for the next fifteen years or more! Congratulations CFF and congratulations to the team at Vertex, congratulations Josh Boger! 

If you want to find out more about funding development of new pharma and biotech, visit and also go to Faster Cures at: 

GLG is developing a series of STAT3 inhibitors to treat orphan diseases particularly: Chronic Lymphocytic Leukemia (CLL), Polycystic Kidney Disease (ADPKD), A1- Antitrypsin deficiency and Gastrointestinal Stromal Tumors (GIST)! If you want to find out more about GLG Pharma and our pipeline of products, visit us at or email me at 

Cheers and as I have had the privilege to say to our soon to be former governor, Deval Patrick, ‘There is a better way to fund biotech’!

Poliwogg, STAT3, GLG Pharma, STAT3 Inhibitors

Tags: STAT3, STAT3 inhibitors, Cancer, Alpha-1, Rare Diseases, Alpha-1 Antitry, Rare Disease

GLG Pharma Attending Faster Cures Partnering Nov. 16-18 NYC

Posted by Richard Gabriel on Sat, Nov 15, 2014 @ 02:18 PM

GLG Pharma Attending Partnering For Faster Cure

GLG Pharma will be attending the Partnering for Faster Cures in New York City on November 16 through 18. The program is a matching of entrepreneurs looking for capital, investors and pharma companies looking for new ideas and technologies. The event is also peppered with researchers and patient advocacy groups as well as not-for-profits funding research organizations across a variety of diseases. Along with Richard Gabriel, COO of GLG Pharma, Greg Simon, CEO of Poliwogg will be attending.  In 2003, Mr. Simon along with Michael Milken founded Faster Cures  out the Milken Institute. 

Poliwogg, Greg Simon, GLG Pharma

Greg Simon, CEO; Poliwogg, Inc. 

Faster Cures’s goals are what the name implies, groups of patients, organizations, companies and entrepreneurs seeking a better way to fund Biotech and Pharma discovery programs so that they reach target patient populations sooner rather than later. 

GLG Pharma is raising funds using the new Poliwogg platform. Poliwogg offers private company investment on its site. This is an example of how accredited investors can now democratize their investments across a portfolio of new startup companies. 

“We are really excited about the Poliwogg platform as it offers investors direct access to startup companies such as ours” said Richard Gabriel; “Poliwogg’s platform uses standard SEC and FINRA rules and regulations for investment, it’s easy, secure and the valuations of the companies are in line with what previously only venture capital firms have had access to” Gabriel goes on to say. “For patient advocacy groups that want a direct opportunity to invest or for individuals that also want to invest directly in the development of a technology for a particular disease, Poliwogg offers an exciting and easy way to do it. Investors are able to spread their investments across multiple platforms and multiple technologies in multiple private companies.” 

Gabriel, GLG Pharma, STAT3 Inhibitors

Richard Gabriel, COO; GLG Pharma

Faster Cures features this event as a mean of accelerating drug discovery, development and funding. The new Poliwogg platform allows companies like GLG Pharma to raise capital and also provides a crucial first step to the potential public offerings. The valuation increases of a private company moving to a public offering will then go directly to the investors, employees and entrepreneurs participating in the private funding rounds.

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Click on the Poliwogg!

Tags: GLG Pharma, STAT3, STAT 3 inhibitors, STAT3 cancer, STAT3 inhibitors, Cancer, cancer diagnosis, Polycystic Kidney Disease, Cancer Stem Cells

The Alpha-1 Antitrypsin Deficiency Walk!

Posted by Richard Gabriel on Mon, Nov 10, 2014 @ 11:36 AM

The Alpha-1 Question

Alpha-1 Antitrypsin Deficiency (Alpha-1) is an inherited genetic disorder and about 19 million people in the US are carriers of the gene. The problem is when genetic mixing creates the perfect Alpah-1 storm. Although there are many kinds of Alpha-1 Trypsin disease the most common amongst global populations is called the S and Z genes. 

For persons with both ZZ genes, which represent an estimated 100,000 persons in the USA and are likely to get liver and lung complications that will lead to transplant surgery. Persons with the SZ combination are not as likely but will be susceptible to chronic diseases such as emphysema, liver disorders and chronic pulmonary obstructive disease (COPD). About 3% of the COPD patient population test positive for the Alpha-1 disease according to the Alpha-1 Foundation. 

Why is GLG Pharma working and participating in raising money for Alpha-1 research? Simple, there is no cure for Alpha-1 and The Alpha-1 Foundation has invested more than $50 million to support research and programs to speed the commercialization of therapies for the elimination of Chronic Obstructive Pulmonary Disease (COPD) and liver disease caused by Alpha-1. Activated STAT3 mediates several diseases of the lung and liver and this may also be the case in Alpha-1 mediated liver and lung diseases. 

We want you to help the Alpha-1 Foundation by sponsoring Michael Lovell and Hector Gomez for a November 15th walk. This money will help the foundation continue its work in looking for cures as well as helping new products reach the market sooner rather than later. Michael Lovell our Executive Vice President and Hector Gomez our CEO are walking so please support the Alpha-1 foundation by donating through their link. Just click on the photo of your choice and donate to the Alpha-1!

                                           Support Hector Gomez:                                      Support Michael Lovell:

                                          HJGomez, MD, PhD                            IMG 7828 resized 600

Tags: GLG Pharma, STAT3, STAT3 inhibitors, Cancer, Alpha-1, Cancer Therapy, Alpha-1 Antitry, Cancer Stem Cells, Chemotherapy, Alpha-1 Antitrypsin deficiency

STAT 3 publications are numerous and we hope that the following links will help you better understand it's importance in cancer cell metabolism and cancer cell death. Inhibiting STAT 3 is an important mechanism for encouraging cancer cell death. Using STAT 3 inhibitors with other traditional cancer chemotherapy should help improve patient outcomes. Linking a diagnostic with a STAT 3 inhibitor will also help reduce patient side effects as well as potentially improve patient outcomes.