drug development


Headline of the Month, Maybe the Year . . .

Journalists and politicians have been hitting pharma companies pretty hard and heavy these days over high drug prices.  But we think a recent headline at STAT news about drug effectiveness might be going too far.  

The following appeared in my email inbox:

Opinion:  Drug effectiveness should influence what doctors prescribe.

Proposed NIH Cuts, Undermining GINA, and Game Changing Drugs: March 2017 with Nathan and Laura

The largest cut to NIH budget ever, rolling back genetic non-discriminatory law—the bad news continues to roll from Washington. But there was great news this month as well.

Both Nathan and Laura are fuming about HR 1313, or a Republican bill to roll back GINA protections. Laura points out that the proposed law builds on an exemption in GINA for wellness programs—a category difficult to define. And Nathan reminds us that families and children could be hurt by the new bill. Theral asks since when did privacy become partisan? GINA passed in ’08 with a vote of 95-0 in the Senate, 414-1 in the House (Ron Paul playing the weirdo there), and it was signed by George Bush.

Then on to some “game changing” drugs for multiple sclerosis and eczema and a successful gene therapy trial for severe sickle cell anemia. Not only are there new therapies, drug manufacturers seem to be getting the message on pricing.

The Saga Continues: Ethan Perlstein, Indie Scientist, Part 3

Today Ethan Perlstein joins us on the program for a third time. His path as a ‘rogue scientist’ has become a bit of a case study here at Mendelspod as we look into alternate paths for scientists and for drug development.

When Ethan was first on the program, he’d just declared his independence, or break from the world of academia. He urged other scientists to do the same and to take to social media to upend a system that was creating the “postdocalypse.” And he didn’t just take a job working for big pharma. He wanted to carry on with the science that intrigued him: a new model of drug discovery.

The second time we featured Ethan he was set up at the QB3 incubator space in San Francisco as the Perlstein Lab, a hybrid of academic lab and regular biotech startup. He had secured some seed funding, and hired five scientists. It was then that he announced a new drug discovery platform using simple animal models (yeast, frutiflies, worms) that would provide proof of concept at a much lower price than what you see with traditional biotechs and pharma.

That was in 2014. Now, at the end of 2016 Ethan has some updates to share. Last month he announced a licensing and investment deal with the drug giant Novartis. He can boast of his first drug candidate, Per101. And he’s a graduate of the Silicon Valley’s business maker, the well known Y Combinator.

Ethan says the Novartis deal is more than just his first revenue generator, it validates the platform of using simple animal models. The deal also boosts what he calls his precision 'business development model.”

“People talk about precision medicine, but they don’t ever talk about precision business development. And that part of the discussion is critical, because you need to think about not only the science that gets the job done, but how the company practically gets put together.”

Working on rare diseases, Ethan is going directly to patients, albeit rich ones at first, with a low cost drug discovery method, creating a new business model that cuts past traditional funding partners and offers a somewhat more academic setting. Perlara, the newest name for Perlstein Lab, is a public benefit corporation.

What was Ethan's experience at the tech dominated Y Combinator? How has being a public benefit corp impacted his decisions this past year? And, come on, if he looks, smells, and acts like a regular old biotech entrepreneur, is he still a rogue scientist?

Here it is. The Indie Scientist, Part 3.

Flipping Drug Development Upside Down: Niven Narain, BERG Health

The promise of rational drug design has driven pharma companies for years. The history of the industry has been one of trial and error, or “guess and check”, as scientists often say. Companies have screened thousands and thousands of compounds looking for one that might work—the proverbial needle in the haystack. With the arrival of molecular profiling and an explosion in understanding of basic biology, many in our field have hoped to make drug development more predictable: to start with the biology, and with the aid of new computing power, design a drug to work in a specific biological setting. But instead we've been making the haystack bigger. When will we see a tipping point where more drugs than not are derived through logic rather than luck? Though there have been great examples—Merck’s Truspot being the first and Novartis’ Gleevec perhaps the best known—rational drug design still remains a mostly unfulfilled promise.

BERG Heatlh, a Boston based pharma company, is working to change that with their AI platform and a new “back to biology” approach. Today we talk with BERG CEO, Niven Narain, who also is a co-founder of the company.

“We’re flipping the entire [drug development] model upside down,” Niven says. "We’re going to ask the patient biology what has gone wrong and generate as much of the omics data as we can from that patient. We then correlate that to their histories and health records in a population based way and compare it to healthy individuals. Out of that analysis we derive potential drug candidates and biomarkers.”

BERG has secured a considerable amount of press with the attention grabbing headline, “company using AI to cure cancer.” Backed by billionaire Carl Berg, the company can’t be faulted for thinking small. But are they doing anything different than other pharma companies? In today’s interview, Niven says they are looking at 15 trillion data points on a single sample. And the scientific approach is perhaps more robust that we’ve seen in the past. Rather than relying on the latest biomarker studies, the company takes a non-biased approach by letting the patient’s data create it’s own picture of the biology.

Is the Future of Biology a Return to Chemistry? Carolyn Bertozzi, Stanford

Classes for the school year begin this week at Stanford University. New to the faculty is Carolyn Bertozzi, an American chemist who made her name across the bay at Berkeley and was wooed to Stanford by a chance to do research and teach chemistry in a new interdisciplinary institute known as ChEM-H. The institute will bring chemists, engineers, biologists and medical doctors together to understand life at a chemical level. We’ve often heard of biology and engineering institutes, or bringing bio and IT. This institute ups the ante and includes chemistry and medicine.

Carolyn is an outspoken scientist who feels that chemistry gets short shrift in a time when biology is considered the queen of the sciences. She points out that the National Insittues of Health tend to be lead and run by biologists. We usually call it biomedical research, not chemical-biomedical research. And yet, she argues, it is chemistry that will give us the answers going forward.

“This is a bit of semantics, but I’d say that what we don’t understand about biology is what happens at the level of molecules. What we don’t understand about biology is the chemistry of it. It is hard to see. You need a different set of tools and technology to see what happens at the molecular scale. And that is the chemistry,” says Carolyn in today’s interview.

Does Carolyn think there’s too much hype around genomics? Would she like to see a revival of chemistry?

As the editor-in-chief of a new open access journal, ACS Central Science, Carolyn will be publishing much more on the topic, making louder and prouder the voice of the chemist.

Returning to Old Biotech Model, OncoMed Boasts of Seven Drugs in Clinical Trials by Mid 2015

For many years, the trend in biotech was for drug development companies to pursue one or maybe two drug candidates, or assets, as they’re called in industry parlance. Go lean and attract the attention of big pharma or investors in the late stage trials. But today’s guest says there is more innovation when the biotech organization invests in a technology platform that produces multiple drug candidates. Paul Hastings is the CEO of OncoMed, a company developing drugs that target cancer stem cells. He’s built OncoMed in what he says was the old model. By mid-2015, the company will have seven drug candidates in the clinic.

"There was a period of time when investors were only interested in late stage products that they could put their arms around and measure,” he says. "But I think investors woke up. . . .They realized that the innovation part of what we were doing before was suffering from this model.”

OncoMed was one of the first IPOs of the current bull market. Has Paul changed his management style since going public? Does he buy into the virtual drug development model? And what does he see for the industry in 2015?

'A Good Year' with John LaMattina

Guest:

John LaMattina, Senior Partner, Pure Tech Ventures Bio and Contact Info

Listen (6:27) Comparing drug approvals: 2014 with 1996

Listen (7:10) More rational drug development?

Listen (1:58) Did public image of big pharma improve last year?

Listen (7:00) The Peter Bach argument

Listen (5:16) Who is winning the debate?

John LaMattina, the former president of Pfizer Global Research, is spending his semi-retirement cultivating the brand of public defender of pharma. As a contributor to Forbes, he showcases some of the great stories of drug development and offers counter punches to pharma’s detractors.

His recent column: FDA Approvals 1996 vs. 2014: The Two Most Prolific Years, But Stark Differences provides the context for today’s interview.

After a look at what is now seen as a banner year for drug development, LaMattina comments on highlights from last week’s JP Morgan Healthcare Conference in San Francisco. He's excited not only about all the business deals, but the new science as well.

The interview ends with a discussion about the debate over drug pricing, and who John thinks is winning it.

Janet Woodcock, FDA, on Biomarker Development and the Future of Clinical Trials

Guest:

Janet, Woodcock, MD, Director, CDER, FDA
Bio and Contact Info

Listen (4:41) No agency charged with better translational outcomes

Listen (2:46) How will recent FDA move on LDTs impact biomarker development?

Listen (4:45) Lung Map and the future of clinical trials

Listen (3:07) What about trials of one?

Listen (4:12) Translation not just about getting to Phase I

Listen (1:49) How important are biosampling issues?

Listen (2:50) Diagnosis the foundation of medicine

Listen (2:25) Thoughts on drug pricing debate

Since becoming the Director of the Center for Drug Evaluation and Research at the FDA, Janet Woodcock has been a strong advocate for better science. But the FDA’s job as regulator is to protect and promote health, not particularly to focus on improving the process of drug development. In fact, Janet points out, there is no agency “charged” with better translational outcomes.

For this reason, Janet worked to set up the Critical Path Initiative some years back. This would be a program to bring together a consortia of different organizations to primarily focus on better biomarker development as well as to modernize clinical trials.

Today we ask Janet for an update on the program. Are we getting better biomarkers? And what do the clinical trials of the future look like?

“In the future, we’ll be setting up trials around patients and disease rather than setting up a trial around a drug,” she says in today’s interview. “We have too many questions to answer about treating disease than can be done by serial trials, each one one only addressing a question about a single investigational drug.”

She’s particularly excited about a new Lung Map trial being run by the NCI. This trial is about finding a second line treatment for patients with squamous cell lung cancer. Patients are enrolled into one of five different strata based on the biomarkers of their tumors. This is a trial that can go on and on, where if a patient doesn’t respond to one treatment, this patient can be moved to a different therapy. This type of trial is known as an adaptive trial and has strong support from the FDA.

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What are Janet’s thoughts about "trials of one," where the health of the patient in the trial is more important than some future unknown patients as advocated by former guest on the program, Marty Tenenbaum?

And what does Janet think about the translational work of physician scientists who are enrolling their own patients in clinical trials based on those patients' biomarkers?

We explore with Janet the question of why diagnostics are valued so much lower than therapeutics in our culture. And at the end of the interview she offers some brave, if limited, comments about the current debate over drug pricing.

Podcast brought to you by: National Biomarker Development Alliance - Collaboratively creating standards for end-to-end systems-based biomarker development—to advance precision medicine

Biotech’s Gentleman Lawyer: Alan Mendelson

Guest: Alan Mendelson, Partner, Latham & Watkins

Bio and Contact Info

Alan Mendelson, a partner at Latham & Watkins, is the first service provider--as opposed to a scientist, entrepreneur, or venture capitalist--to receive one of BayBio’s prestigious Pantheon Lifetime Achievement Awards. We talk to him a month before the awards ceremony which will be held in San Francisco on December 11th, 2014.

Alan’s career took off back in the early 80’s when he incorporated one of the few new biotech companies coming on to the scene. The company: Amgen. Alan gives a great deal of credit to his “mentor”, George Rathman, the legendary first CEO of Amgen who inspired a generation of biotech entrepreneurs.

In the early 2000’s, Alan was one of the first to see signs of the Silicon Valley dot com bust which led to him leave his long time firm, Cooley and Associates, to join Latham & Watkins. In today's wide ranging interview, he shares this and other stories from his career as well as his thoughts on the current biotech marketplace.

“The award means a lot to me,” he says, "it’s been thirty-four years since I first incorporated Amgen. Working with biotech and life science companies, I can’t tell you how satisfying it is to feel that in a small way, I've helped improve the human condition. I’ve had cancer patients tell me that I saved their lives because I worked with Amgen. . . and this is, frankly, why I don’t want to retire.”

Chapters: (Advance the marker)

1:17 The “gentleman lawyer”

5:20 If they’re passing out cookies, take one

10:25 Importance of the Jobs Act

16:12 The Cooley “divorce”

25:34 What does this award mean to you?

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Sponsor: Today’s show is brought to you by the 11th Annual BayBio Pantheon Ceremony, presenting the 2014 DiNA Awards on December 5 in San Francisco. The Pantheon Awards Ceremony is a celebration of the contributions and achievements of the Bay Area, a moment to pause and reflect on the industry’s legacy over three decades.

The Basic Biology Lab Goes into the Cloud: Brian Frezza, Emerald Therapeutics

Guest: Brian Frezza, Co-Founder, Co-CEO, Emerald Therapeutics

Bio and Contact Info

Chapters: (Advance the marker)

0:50 What is the Emerald Cloud Lab?

4:16 Does this impact the scientific method?

10:00 Pulling the labor, not the scientist out

14:20 Much more data detail

19:52 A chance to improve reproducibility

24:06 The tools are slowing us down

We knew it was coming. Everything else has been going that direction--that virtual realm that offers humanity such hope, affectionately referred to as The Cloud.

Brian Frezza is a young entrepreneur quite fresh out of grad school. Brian and his co-founder, D.J. Kleinbaum, went to Carnegie Mellon and Stanford. They liked to think about the big picture when they were in school. What could we do, what product, what company could we work on that would drastically--not just incrementally--change the world of drug and diagnostic discovery? they'd ask themselves.

Four years into their commercial adventure, they've released what they think will make that big change--to use the popular term, be a disruption.

"The Emerald Cloud Lab--think of it as a remote laboratory that you're controlling via the internet, as if you were standing in front of the instruments themselves when you run your experiments," says Brian at the outset of today's interview.

Brian carries on with a cool evenness, but this is quite a mouthful. What? A scientist can have access to a full laboratory to run one of about forty experiments without having to invest in the equipment, space, and labor?

Brian says the biggest challenge to putting a lab in the cloud, no doubt, was in coding the language for the automation. This is automation on a scale we've never seen before.

Presenting The Emerald Cloud Lab.

Editor's note to our audience: As a scientist what is your view of this? Is this the best thing ever, or is it too giant a step? Does this degrade the scientific method or better enable it? Please give your feedback in the comment section below.

Podcast brought to you by: Chempetitive Group - "We love science. We love marketing. We love the idea of combining the two to make great things happen for your marketing communications."



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